Q4 2023 Harmony Biosciences Holdings Inc Earnings Call
Operator: Good morning. My name is Brittany, and I will be your conference operator today. At this time, I would like to welcome everyone to the Harmony Biosciences fourth quarter and full year 2023 financial results conference call. All participant lines have been placed on mute to prevent any background noise.
Good morning, My name is Britney and I will be your conference operator today at this time I would like to welcome everyone to the harmony Biosciences fourth quarter and full year 'twenty 'twenty through 2023 financial results conference call.
Just a bit lines have been placed on mute to prevent any background noise. After the speakers' remarks tariff will be a question and answer session. If he would like to ask a question at that time. Please press star one on your telephone keypad. Please be advised for today's conference maybe recorded lastly, if you should require operator assistance. Please press star.
Operator: After the speaker's remarks, there will be a question and answer session. If you would like to ask a question at that time, please press star one on your telephone keypad. Please be advised that today's conference may be recorded. Lastly, if you should require operator assistance, please press star zero. I would now like to turn the call over to Louis Diné, head of investor relations. Please go ahead.
Zero.
Now I'd like to turn the call over to lose today head of Investor Relations. Please go ahead. Thank.
Louis Diné: Thank you, operator. Good morning, everyone, and thank you for joining us today as we review Harmony Biosciences' fourth quarter and full year 2023 financial results and provide a business update. Before we start, I encourage everyone to go to the investors section of our website to find the materials that accompany our discussion today, including the reconciliation of our GAAP to non-GAAP financial measures. At this stage of our life cycle, we believe non-GAAP financial results better represent the underlying business performance. Our speakers on today's call are Dr. Jeffrey Dano, President and CEO; Jeffrey Dirks, Chief Commercial Officer; Dr. Kumar Bidur, Chief Medical Officer; and Sandip Kapadia, Chief Financial Officer and Chief Administrative Officer. As a reminder, we will be making forward-looking statements today, which are based on our current expectations and beliefs. These statements are subject to certain risks and uncertainties; our actual results may differ materially, and we undertake no obligation to update these statements, even if circumstances change.
Thank you operator, good morning, everyone and thank you for joining US today as we review harmony Biosciences fourth quarter and full year 2023 financial results and provide a business update.
Before we start I encourage everyone to go to the investors section of our website to find the materials that accompany our discussion today, including a reconciliation of our GAAP to non-GAAP financial measures.
At this stage of our lifecycle, we believe non-GAAP financial results better represent the underlying business performance.
Our speakers on today's call are Dr. Jeffrey day, no president and CEO Jeffrey.
Jeffrey Dirks, Chief commercial officer plastic Kumar of a door Chief Medical Officer.
Sandeep could party, our chief financial Officer, and Chief administrative officer.
As a reminder, we will be making forward looking statements today, which are based on our current expectations and beliefs.
These statements are subject to certain risks and uncertainties. Our actual results may differ materially and we undertake no obligation to update these statements even if circumstances change.
Operator: We encourage you to consult the risk factors referenced in our SEC filings for additional details. I would now like to turn the call over to Dr. Jeffrey Dano.
We encourage you to consult the risk factors referenced in our SEC filings for additional details.
I would now like to turn the call over to Dr. Jeffrey Dana Jack.
Dr. Jeffrey Dano: Thank you, Lewis, and thanks, everyone, for joining our conference call today. Harmony continues to be a growth story, as demonstrated by our team's accomplishments throughout 2023. We delivered another strong year of performance across the organization, growing revenue and the average number of patients on WACIC. We advanced all our clinical development programs for patolisin, moved the next-generation or next-gen formulations of bitolacin into the clinic, and expanded our pipeline and diversified our portfolio with the acquisition of Zynerva and the ongoing phase three clinical trial in patients with Fragile X syndrome. Yesterday, we announced that FDA granted priority review for our Supplemental NDA, or SNDA, for WCAGs in pediatric narcolepsy with a BDUFA date of June 21. Earlier this week, we also shared that FDA granted orphan drug designation for patolacin for the treatment of Prader-Willi syndrome, or PWS, as we are preparing to initiate our pivotal phase 3 TEMPO study in patients with PWS down to age 6.
Thank you Louis and thanks, everyone for joining our conference call today.
Harmony continues to be a growth story as demonstrated by our team's accomplishments throughout 2023.
We delivered another strong year of performance across the organization growing revenue and average number of patients on <unk>.
We advanced all of our clinical development programs for pets Holliston.
Move to the next generation or next Gen formulations at the Tulsa and into the clinic.
And expanded our pipeline and diversified our portfolio with the acquisition of <unk> and the ongoing phase III clinical trial in patients with fragile X syndrome.
Yesterday, we announced that FDA granted priority review for our supplemental NDA for S. N da for wake kicks in pediatric narcolepsy with a producer date of June 21st.
Earlier. This week, we also shared that FDA granted orphan drug designation for <unk> for the treatment of prouder Willi syndrome or P. W. S. As we are preparing to initiate our pivotal phase III tempo study in patients with P Ws down to age six.
Dr. Jeffrey Dano: All of this reflects momentum across our organization from our commercial business with Wacox and Narcolepsy to our clinical development programs as well as positive interactions with FDA related to these programs. These outcomes also reflect excellence in execution and the dedication of our team to deliver on the strategy we have laid out and are executing on. In addition, we returned capital to shareholders via our Share Repurchase Program and will continue to take an opportunistic approach to this program throughout this year. I am very excited for the year ahead and believe that Harmony is poised to accelerate its growth in 2024. For the fourth quarter, we reported wages net revenue of $168.4 million, and full year net revenue was $582 million, representing growth of 31% and 33%, respectively.
All of this reflects momentum across our organization from our commercial business with wake kicks in narcolepsy to our clinical development programs as well as positive interactions with FDA related to these programs.
These outcomes also reflect excellence in execution and the dedication of our team to deliver on this strategy, we have laid out and are executing on.
In addition, we returned capital to shareholders via our share repurchase program and we'll continue to take an opportunistic approach to this program throughout this year.
I am very excited for the year ahead, and believe that harmony is poised to accelerate our growth in 'twenty to 'twenty four.
For the fourth quarter, we reported weight <unk> net revenue of $168 $4 million and full year net revenue was $582 million representing growth of 31% and 33% respectively.
Dr. Jeffrey Dano: This result demonstrates the significant underlying demand for Wacox and the durability of the brand going into year five in the market. Other factors that drive our confidence in our ability to grow WCAGs include its meaningfully differentiated product profile, which results in its broad clinical utility. Combining that with our ability to reach the broad prescriber universe of approximately 9,000 HCPs who see and treat almost 100% of the diagnosed narcolepsy patient opportunity, we have seen growth in patients on Wacax and prescribers every quarter since launch, even with the entrance of new branded and generic Oxivate treatment options coming into the market over the past few years. Finally, while there are several investigational agents in the clinic for narcolepsy, we do not see any new or novel mechanisms of action coming to the market that will impact the growth of WACCX until late during its life cycle in this polypharmacy market.
This result demonstrates the significant underlying demand for wake effects and the durability of the brand going into year five in the market.
Other factors that drive our confidence in our ability to grow way kicks include its meaningfully differentiated product profile, which results in its broad clinical utility.
Couple that with our ability to reach the broad prescriber universe of approximately 9000, hcp's, who see and treat almost 100% of the diagnosed narcolepsy patient opportunity.
Yeah.
We have seen growth in patients on way kicks 'em prescribers every quarter since launch even with the entrance of new branded and generic oxidate treatment options coming into the market over the past few years.
Finally, while there are several investigational agents in the clinic for narcolepsy, we do not see any new or novel mechanisms of action coming to the market that will impact the growth of wake ex until late during its lifecycle and this polypharmacy market.
Dr. Jeffrey Dano: Based on these reasons, along with the vast market opportunity that remains in narcolepsy, we believe that we can continue to grow the franchise for years to come and remain confident that WACIX represents a $1 billion plus opportunity in adult narcolepsy alone, and we are well on our way. This is evidenced by our net revenue guidance for 2024, ranging from $700 to $720 million. In addition to our strong commercial performance, we also advanced and expanded our clinical development enterprise on several fronts. Kumar will provide more details on our clinical development programs later in the call. But first, let me highlight some of our progress.
Based on these reasons along with the vast market opportunity that remains in narcolepsy. We believe that we can continue to grow the franchise for years to come and remain confident that wakes represents a 1 billion dollar plus opportunity in adult narcolepsy alone and we are well on our way.
This is evidenced by our net revenue guidance for 2024, ranging from $700 million to $720 million.
In addition to our strong commercial performance, we also advanced and expanded our clinical development enterprise on several fronts.
Omar will provide more details on our clinical development programs later in the call, but first let me highlight some of our progress.
Dr. Jeffrey Dano: Starting with pediatric narcolepsy, we are pleased that FDA granted priority review for our SNDA for WCAG. This expedites the review process, and FDA has set a PDUFA date of June 21. We look forward to collaborating with the agency to potentially introduce a new non-scheduled treatment option for pediatric patients living with narcolepsy.
Starting with pediatric narcolepsy.
We are pleased that FDA granted priority review for our S. N D. A for wake ex this expedites the review process and FDA has set a paducah date of June 21st.
We look forward to collaborating with the agency to potentially introduce a new nonscheduled treatment option for pediatric patients living with narcolepsy.
Turning to idiopathic hypersomnia or I H.
Dr. Jeffrey Dano: After completing our review of the full dataset, we continue to believe that, based on the totality of the data, along with pitolicin receiving orphan drug designation for IH, and against the backdrop of only one currently approved product, there is a strong case to be made regarding the overall benefit-risk proposition of Pitocin for patients with IH. We will be engaging with the agency with this goal in mind, have a meeting scheduled with them next month, and look forward to working with the agency on bringing a potential new non-scheduled treatment option to patients living with IH. Our current life cycle management programs for Patulsin, which include IH, Prader-Willi syndrome, and type 1 myotonic dystrophy, represent about 100,000 diagnosed patients in the U.S.
After completing a review of the full dataset.
We continue to believe that based on the totality of the data.
Along with <unk>, receiving orphan drug designation for IH.
And against the backdrop of only one currently approved product that there is a strong case to be made regarding the overall benefit risk proposition of <unk> for patients with IH.
We will be engaging with the agency with this goal in mind.
Have a meeting scheduled with them next month and look forward to working with the agency on bringing a potential new nonscheduled treatment option to patients living with IH.
Our current lifecycle management programs for <unk>, which include I H.
<unk> Willi syndrome, and type one my atonic dystrophy represent about 100000 diagnosed patients in the U S.
Dr. Jeffrey Dano: So, if successful, these new indications could contribute up to an additional $1 billion of revenue to the WACIX franchise. Given Pitolacin's novel mechanism of action and the success of Wacox in the market, we've been working on next-gen formulations of Pitolacin with the goal to generate new IP and extend the Pitolacin franchise out beyond 2040. These programs entered the clinic last quarter, and we are on track to report PK data in the first half of this year. Another key component of our growth strategy is business development to expand our pipeline beyond WACIX and diversify our portfolio beyond SleepWake. The Zynerva acquisition, which closed last October, represented an important step in that direction and was an excellent strategic fit for Harmony, bringing in two late-stage development programs focused on orphan rare neuropsychiatric disorders with significant unmet medical needs.
So if successful these new indications could contribute up to an additional $1 billion of revenue to the wake X franchise.
Given <unk> since novel mechanism of action and the success of wake acts in the market. We've been working on next Gen formulations of <unk> with the goal to generate new IP and extend the pit tolleson franchise out beyond 2040.
These programs entered the clinic last quarter and we are on track to report PK data in the first half of this year.
Another key component of our growth strategy is business development to expand our pipeline beyond <unk> and diversify our portfolio beyond sleep wake.
This I never acquisition, which closed last October represented an important step in that direction and it was an excellent strategic fit for harmony, bringing into late stage development programs focused on orphan rare neuropsychiatric disorders with significant unmet medical needs.
Dr. Jeffrey Dano: And we are not stopping with the Zynerva acquisition and remain very active in business development, continually assessing the BD landscape with a focus on rare neurological disease assets and other rare disease assets with unmet medical needs, where we can leverage our existing infrastructure and synergies across our organizations. We are looking for assets across a range of development stages with a preference for late-stage assets but open to early-stage assets where there is a strategic fit. We have approximately $426 million in cash, cash equivalents, and investments at year-end. We are in a solid financial position to execute on business development opportunities, which is a strategic priority for us. In conclusion, I am proud of our team's accomplishments in 2023 and what we were able to do to help patients living with narcolepsy while advancing our development programs and other rare disease patient populations.
And we are not stopping with this <unk> acquisition and remain very active in business development continually assessing the BD landscape with a focus on rare neurological disease assets and other rare disease assets with unmet medical needs, where we can leverage our existing infrastructure and synergies.
Across our organization.
We are looking for assets across a range of development stages with a preference for late stage assets, but open to early stage assets, where there was a strategic fit.
With approximately $426 million in cash cash equivalents and investments at yearend.
We are in a solid financial position to execute on BD opportunities, which is a strategic priority for us.
In conclusion.
I am proud of our team's accomplishments in 2023, and what we were able to do to help patients living with narcolepsy.
While advancing our development programs and other rare disease patient populations.
Dr. Jeffrey Dano: We are experiencing solid and durable growth in our core commercial business, strong momentum in our clinical development programs, and expansion and diversification of our pipeline assets. Based on the dedication and commitment across our organization, I am very excited for our opportunities in 2024, as we remain focused on developing and commercializing innovative treatments for patients living with rare neurological diseases who have unmet medical needs. I will now turn the call over to Jeffrey Dirks, our Chief Commercial Officer, to provide more details on our commercial performance. Jeff?
We experienced solid and durable growth in our core commercial business.
Strong momentum in our clinical development programs and expansion and diversification of our pipeline assets.
Based on the dedication and commitment across our organization I am very excited for our opportunities in 2024, as we remain focused on developing and commercializing innovative treatments for patients living with rare neurological diseases, who have unmet medical needs.
I will now turn the call over to Jeffrey Darks, our Chief commercial officer.
To provide more details on our commercial performance Jeff.
Okay.
Jeffrey Dirks: Thank you, Jeff. 2023 was a strong year of growth for WACACS and year four of our commercialization for adult narcolepsy. The fourth quarter represented the strongest revenue quarter in our history with continued growth and momentum and our underlying business fundamentals and top line performance metrics. Net revenue for the fourth quarter was $168.4 million, representing 31% growth from the same quarter a year ago and our second consecutive quarter of over $150 million in net revenue. Full year 2023 net revenue was $582 million, a 33% increase from full year 2022.
Thank you Jeff.
2023 was a strong year of growth for wake X in year four of our commercialization in adult narcolepsy.
The fourth quarter represented the strongest revenue quarter in our history with continued growth and momentum in our underlying business fundamentals and topline performance metrics.
Net revenue for the fourth quarter was $168 $4 million, representing 31% growth from the same quarter prior year, and our second consecutive quarter of over $150 million in net revenue.
Full year 2023, net revenue was $582 million or 33% increase from full year 2022.
Jeffrey Dirks: We continue to see strong double-digit growth in net revenue for WACIX, heading into year five of our commercialization, reflecting continued high interest in WACIX in the narcolepsy market. The solid performance and continued growth reinforces our long-term belief that WACACS represents a potential billion-dollar-plus opportunity in adult narcolepsy alone. I'd like to share a few key highlights from our performance in the fourth quarter on slide five. The average number of patients on WACIX in the fourth quarter increased to approximately 6,150, an increase of approximately 350 average patients sequentially from what we reported last quarter.
We continue to see strong double digit growth in net revenue per week X heading into year five of our commercialization, reflecting continued high interest of wake extend the narcolepsy market.
The solid performance and continued growth reinforces our long term belief that <unk> represents a potential billion dollar plus opportunity in adult narcolepsy alone.
I'd like to share a few key highlights from our performance in the fourth quarter on slide five.
The average number of patients on <unk> in the fourth quarter increased to approximately 6150, an increase of approximately 350 average patients sequentially from what we reported last quarter.
Jeffrey Dirks: The impressive growth in average patients in the fourth quarter was driven by strong top-line demand and new patient starts and speaks to continued product adoption, and more importantly, it highlights the remaining large diagnosed patient opportunity that we continue to tap into each quarter as the market allows. In addition to the strong growth in the average number of patients on Wacax, we also saw continued product adoption of Wacax by the narcolepsy healthcare community, both new and existing prescribers. The number of unique prescribers of WACACS increased again in the fourth quarter, and importantly, we continue to see growth in product adoption of WACACS in the prescriber base beyond Oxybate REMS-enrolled healthcare professionals. Of the approximately 5,000 healthcare professionals not enrolled in the Oxybate REMS program, more than 30% of them have prescribed Wacax to date, up from 25% reported on our last earnings call.
The impressive growth in average patients in the fourth quarter was driven by strong topline demand and new patient starts and speaks to continued product adoption.
And more importantly, it highlights the remaining large diagnosed patient opportunity that we continue to tap into each quarter as the market allows.
In addition to the strong growth in average number of patients on <unk>. We also saw continued product adoption of wake X by the narcolepsy health care community, both in new and existing prescribers.
The number of unique prescribers of <unk> increased again in the fourth quarter and importantly, we continue to see growth in product adoption of wakes and the prescriber base beyond ox debate Rems enrolled health care professionals.
Of the approximately 5000 health care professionals not enrolled in the oxalate Rems program more than 30% of them have prescribed wakes to date up from 25% reported on our last earnings call.
In addition to the continued growth of our prescriber base. We also saw strong growth in the depth of prescribing within the approximately 4000, Oxidate Rems enrolled health care professionals.
Our ability to reach and educate the broadened arc lessee treating health care professional universe allows us to access the full diagnosed adult narcolepsy patient opportunity and gives us confidence and continued growth for wake effects.
Jeffrey Dirks: In addition to the continued growth of our prescriber base, we also saw strong growth in the depth of prescribing within the approximately 4,000 octobate REMS-enrolled healthcare professionals. Our ability to reach and educate the broad narcolepsy-treating healthcare professional universe allows us to access the full-diagnosed adult narcolepsy patient opportunity and gives us confidence in continued growth for WIC. The availability of new and generic Oxibates hasn't impacted patient or prescriber growth or existing strong payer coverage for Wacox, given its meaningfully differentiated product profile. Wakeish remains the first and only FDA-approved treatment for EDS and cataplexy and narcolepsy that is not scheduled as a controlled substance, an attribute that continues to appeal to our broader narcolepsy healthcare professional audience and patient population and is a driver of brand growth.
The availability of new and generic oxidase, hasnt impacted patient or prescriber growth or existing strong payer coverage for <unk>, given its meaningfully differentiated product profile.
Wake each remains the first and only FDA approved treatment for eds, and cataplexy and narcolepsy that is not scheduled as a controlled substance and attribute the continues to appeal to a broader narcolepsy health care professional audience and patient population and as a driver of brand growth.
In summary, 2023 was another year of strong commercial performance for <unk> full year net revenue of $582 million, 33% growth versus the full year 2022.
The fourth quarter represented the strongest revenue quarter for harmony to date with over $168 million in net revenue.
The average number of patients on <unk> increased to approximately 6150.
The waitress prescriber base within and beyond the oxalate Rems enrolled health care professional audience continued to grow and lastly, payer coverage remained strong even with the availability of new in generic oxidate options.
Jeffrey Dirks: In summary, 2023 was another year of strong commercial performance for Wacom. Full-year net revenue of $582 million, 33% growth versus the full-year 2022. The fourth quarter represented the strongest revenue quarter for Harmony to date, with over $168 million in net revenue.
Waitkus continues to demonstrate durable growth in the narcolepsy market, we've seen growth in patients and prescribers every quarter since launch even with branded and generic treatment availability and new launches during its first four years on the market.
Looking ahead to 2024, we expect continued growth in the underlying business fundamentals for <unk> with net revenue is expected to be between 700 and $720 million.
We anticipate a similar quarterly rhythm to our business in 2024 with what we've experienced in previous years traditional seasonal payer dynamic headwinds that impact the entire industry as a whole in Q1.
Jeffrey Dirks: The average number of patients on WACIX increased to approximately 6,150. The Wakex Prescriber Base, within and beyond the Octobate REMS-enrolled healthcare professional audience, continued to grow. And lastly, payer coverage remained strong, even with the availability of new and generic Octobate options. WAKIS continues to demonstrate durable growth in the narcolepsy market. We've seen growth in patients and prescribers every quarter since launch, even with branded and generic treatment availability and new launches during its first four years on the market. Looking ahead to 2024, we expect continued growth in the underlying business fundamentals for WACIX, with net revenues expected to be between $700 and $720 million.
Tailwind coming out of Q1, and Q2 with stronger prescription demand tipping.
Typical seasonal headwinds in Q3 with lower patient visits that are common for all products in diseases that are chronically managed and tailwind in the fourth quarter as we close out the year.
With no new competition expected in 2024 and limited differentiation in generic and branded products in the near term pipeline, coupled with the large remaining diagnose patient opportunity unmet need and the polypharmacy nature of the narcolepsy market weight geeks is well positioned for continued growth.
Jeffrey Dirks: We anticipate a similar quarterly rhythm for our business in 2024 with what we've experienced in previous years. Traditional seasonal payer dynamic headwinds that impact the entire industry as a whole in Q1, and tailwinds coming out of Q1 and Q2 with stronger prescription demands. Typical seasonal headwinds in Q3 with lower patient visits that are common for all products and diseases that are chronically managed, and tailwinds in the fourth quarter as we close out the year.
Jeffrey Dirks: With no new competition expected in 2024 and limited differentiation in generic and branded products in the near-term pipeline, coupled with the large remaining diagnosed patient opportunity, unmet need, and the polypharmacy nature of the narcolepsy market, Wacox is well-positioned for continued growth. Our ability to educate the broad REMS and non-REMS narcolepsy treating healthcare professional audience and ability to tap into the full diagnosed patient opportunity gives us confidence in the long-term potential for the brand to represent a billion-dollar plus opportunity in adult narcolepsy alone. Our commercial business has been resilient, and I appreciate the dedication and impact of the entire commercial team and the passion that they have for the narcolepsy patient community. I would now like to turn the presentation over to Kumar Bhadur, our Chief Medical Officer, to provide an update on our clinical development pipeline. Kumar?
Good portfolio.
Somebody with a strong experienced and dedicated Orange organization ready to take on new opportunities as we continue to build on existing portfolio.
We have a business development.
I'm proud of the work.
Every day we.
We look forward to evaluating potential new treatment options for patients with America with heightened mcneese.
Oh for clinical development pipeline is shown on slide number seven.
Starting with pediatric narcolepsy.
Submitted a supplemental NDA to the FDA for an indication in pediatric narcolepsy in the four quarters and we are pleased with F. D. As decision to grant a priority to view.
This is deficient highlights the need for a new treatment option for the approximately 4000 pediatric patients living with multiple let's see.
<unk> date of June 21st.
Dr. Kumar Bidur: Thank you. Last year was a defining year for Harmony RA. We made great progress in advancing, expanding, and diversifying our portfolio, working on nine different development programs across five different assets, several of which are in late-stage development. It is not just the numbers but also the complexity of the program as we target a broad set of indications in rare disease patient populations with significant unmet medical needs. As we grew our portfolio, we also built a strong, experienced, and dedicated R&D organization ready to take on new opportunities as we continue to build our existing portfolio with new assets via business development. I'm proud of the work that our team does every day as we look forward to delivering potential new treatment options for patients with rare diseases with high unmet needs.
Forward to working with the FDA potentially bring a nonscheduled treatment option administered once a day in the morning for pediatric patients with narcolepsy.
Moving onto our development programming idiopathic hypersomnia.
We completed our review of the fruit dot effect and submitted an SDA meeting request in the fourth quarter to discuss the path forward and the meeting escape for March.
We are optimistic that we'll be able to find an efficient path forward to bring the total assigned to patients with idiopathic hypothermia.
Our optimism.
Based on the totality of the data with strongly support.
If he can see in patients with idiopathic hypersomnia.
Specially in the context of high end would need.
Only one pickup fraud with this keyboard plus three controlled substance with the Rams broke up and the off label use of cluster <unk> with significant safety issue.
The data from the open label part of this study double blind randomized withdrawal part of this study and the ongoing long term extension study in conjunction with 59 safety profiling offer a strong benefit proposition in favor of <unk> patient idiopathic hypersomnia.
Dr. Kumar Bidur: We look forward to working with the FDA to potentially bring a non-scheduled treatment option administered once a day in the morning for pediatric patients with narcolepsy. We are optimistic that we will be able to find an efficient path forward to bring petrolocine to patients with idiopathic hyperplasia. Our optimism is based on the totality of the data, which strongly supports the adolescent's efficacy in patients with idiopathic hypersomnia, especially in the context of high and mid-need, with only one drug approved, a scheduled class III control substance with a REMS program, and the off-label use of class II control stimulants with significant safety issues.
We look forward to engaging with ESP in the meeting scheduled next month and making a strong case for a positive benefit profile <unk> type of <unk>.
Provide further updates on the IH broker mixed.
Mixed earnings call.
What private Willy's syndrome, we are on track to any safety phase three temple study in the first quarter of 50 years.
This will be a global double blind randomized placebo controlled study that could.
Randomize, approximately 134 patients to either <unk> or a placebo.
<unk> two one ratio.
The duration of the double bind treatment beat it is 11 weeks.
Dr. Kumar Bidur: We look forward to engaging with the FDA at the meeting scheduled next month and making a strong case for a positive beneficiary profile for pitot-lacentine idiopathic hypersomnia. We will provide further updates on the IH program at our next earnings call. This will be a global, double-blind, randomized placebo-controlled study that will randomize approximately 134 patients to either pitolacin or placebo in a 1-to-1
The age range is 60% orders.
Primary eight point is change in Cvs the effects of the beta and sleepiness.
Measured by <unk> F. R. I T score from baseline for the end of the double blind treatment idiot compared to placebo.
Based on the promising data from the phase two proof of concept study. We are also evaluating irritable and disruptive behaviors, which have commenting tw.
Phase three registration study.
Not just to meet the requirements for an indication in T. W. S. But also fulfilling one of the two requirement to gain pediatric exclusivity.
Dr. Kumar Bidur: Based on the promising data from the Phase II Proof of Concept study, we are also evaluating the irritable and disruptive behaviors which are common in PWS. Based on our discussion with the FDA, we have a clear line of sight on the requirements for a PWR, which include data from pediatric narcolepsy patients and data from the phase 3 study in PWL. The Pediatric Narcolepsy SNDS submission in the fourth quarter and the initiation of the Phase III PWS study this quarter are designed to support our efforts to gain pediatric exclusivity for vacancies. In myotonic dystrophy type 1, or DM1, we were pleased to report positive top-line results from the phase 2 proof of concept study, where we saw clinically meaningful improvements in not just excessive daytime sleepiness, as measured by the daytime sleepiness scale, but also in fatigue, which was assessed using the fatigue severity scale.
We remain confident uncommitted to gaining pediatric exclusivity for <unk>, which will offer an additional six month irregularity exclusively on the back end of the longest Patrick slept to go into effect.
Based on the discussion 50, FDA, we have a clear line of sight on the requirements for a PW loss, which include data pediatric narcolepsy patient and.
Data from the Phase three study in T. W.
We are making steady progress to meet this requirement.
<unk> S N D a submission in the fourth quarter.
Imitation of space CPW 30 this quarter.
<unk> are designed to support our efforts to gain pediatric exclusivity for vehicles.
And my Attorney Dystrophy type, one or D. M. One we will feast to report positive pop into it from.
From the phase two proof of concept study <unk> clinically meaningful improvement in not just excessive daytime sleepiness asthma shaped by the daytime sleepiness scale, but also fatigue, which was assessed using.
Using <unk> to scale and this data is summarized on slide nine.
Dr. Kumar Bidur: And these data are summarized on slide 9. In addition, the safety profile of this patient population is consistent with the established safety profile of the hospital. We are currently reviewing the complete data set to assess the opportunity and inform our next steps. As we have noted in the past, this is an indication that could be pivoted to the next 10 petroleum-based formulations, which will have a much longer patent on them. We expect to launch NG-WAN within the VXLight cycle.
It's important to note that these two symptoms.
Data and sleepiness and fatigue.
Present in approximately 80% to 90% of patients be on file.
While at these symptoms.
<unk> of course.
Symptoms of my twenties and broken muscle weakness.
A clear and consistent doses pump was also demonstrated with a higher dose showing greater.
<unk> across the study points.
In addition, the safety profile in this patient population is consistent with the established 50 profile of it all the time.
Dr. Kumar Bidur: This formulation will have new IP, a full clinical development program, and is expected to be launched towards the end of the vacancy life cycle. We were also pleased to expand and diversify our pipeline last quarter with the acquisition of ZynerPath. There are approximately 80,000 patients diagnosed with Fartherleck syndrome in the U.S. with no approved treatments and significant unmet medical needs.
We are currently reviewing the complete that effect to assist the opportunity and inform our next steps.
As we have noted in the past this is an indication which could be pure day to the next and Pitocin based formulation, which will have a much longer pattern for the day.
He continued to make good progress on the next <unk> based formulation M C. One and D. Two without a partner <unk> with a goal of generating new IP extending the <unk> beyond 2040.
Dr. Kumar Bidur: DYM002 was also studied in an open-level phase 2 proof-of-concept study in patients with 22q deletion syndrome, known as the INSPIRE trial, which generated promising data. This represents another market opportunity for approximately 80,000 patients in the U.S., and we have been interacting with FDA about a Phase III program in 2022. To conclude, we have made great progress at Harmony in advancing, expanding, and diversifying our portfolio, resulting in a pipeline with multiple late-stage programs. Our financial performance is also shown on slides 10 through 13, which show $582 million in annual WCAG net revenue.
The new treatment options for people living with <unk>.
Please note we have painted the naming conventions for these two formulation. According to this effective market entry timing.
<unk> one R M T. One.
Modified formulation Mcdonald with a potential for clinical different <unk> at a fast to market is trying to be based on the demonstration of equal level.
<unk> <unk>, we can debate lifecycle.
Next thing to do.
<unk> is an enhanced formulation of <unk> to.
Tell you, what an optimist VK profile and a higher dosage determined.
Formulation will have a new IP.
<unk> program and is expected to be launched towards the end of making slight cycle.
Sandip S. Kapadia: Overall, we remain well positioned to execute on our growth strategy moving forward. For the fourth quarter of 2023, we reported our strongest revenue quarter in company history, with net revenues of $168.4 million compared to $128.3 million in the prior quarter, representing a growth of 31%. In the fourth quarter of 2023, operating expenses were $85.1 million, compared to $53.8 million in the prior quarter.
Both formulation N T one and T to enter in the clinic in the fourth quarter.
Data on both of them in the first half of this year.
We were also pleased to expand and diversify our pipelines last quarter with the acquisition sign up.
This brought in Z Y N 002.
Pharmaceutical you produced synthetic cannot be deal.
B y b at fee for <unk>.
Like <unk>.
Another portfolio product opportunities.
We are currently enrolling patients and <unk> and <unk>.
Really excited about this opportunity.
Sandip S. Kapadia: The higher operating expenses were primarily driven by the advancement of our clinical development programs, including ZYN002, and one times the Narba transaction related cost of $9.8 million. Non-GAAP-adjusted net income for the fourth quarter of 2023 was $42.8 million, or $0.73 per diluted share. The balance reflects continued strong cash generation, with approximately $77 million in cash from operations in the fourth quarter. Offset, but is the Nurbot acquisition cost and share repurchase activities in the quarter? For the full year of 2023, we generated $219 million in cash from operations.
There are approximately 80000 patients diagnosed with <unk> syndrome in the U S.
Two treatments and significant unmet medical need.
We had an opportunity to assess the timeline of the <unk> connect hot and expect a complete patient enrollment in the first quarter of 2025 with toppling data in print 895.
<unk> was ultra studied in an open liberal phase two proof of concept study in patient 22, <unk> control known that inspired trial, which is generated promising data.
This represents another market opportunity of approximately 80000 patients being <unk> and we have been interacting with.
About 53 program in 22 Q.
It is worth noting that <unk>, who is a global opportunity for harmony and we look forward to exploring S D with opportunities to win.
The smaller treatment for people living with <unk> and 22 Q around the world.
Sandip S. Kapadia: During the fourth quarter, we returned capital to shareholders and repurchased approximately 1.8 million shares of common stock for $50 million. A strong balance sheet allows us not only to execute on the return of capital but also maintain financial flexibility to execute on business development. We received approximately $26 million of cash and Deferred Tax Assets from the acquisition and assumed approximately $14 million in liability.
To conclude.
We have made great progress at harmony in advancing expanding.
Fangled portfolio, there's anything in the pipeline with multiple <unk> protocols.
I look forward to sharing additional space as we continue to make progress on a clinical development program.
On behalf of <unk> I would like to thank all the patient and family what participating in our <unk>.
Sandip S. Kapadia: Turning to 2024 guidance for the full year, we expect net revenues of $700 to $720 million. It also highlights that we are well on our way towards the $1 billion-plus opportunity in Delta narcolepsy alone. We have consistently communicated and remain confident that we are well on our way towards the $1 billion-plus opportunity. And finally, a comment on seasonality as you think about the phasing of revenue for the first quarter of 2024. We expect to see the typical seasonal dynamics that the industry as a whole experiences each year in Q1. Higher gross net deductions due to insurance plans being reset and higher copay obligations, along with a drawdown in trade image.
Clinical investigators on site personnel for that effort and commitment in helping us Bradford Elephant Pro cloud.
I have not done the call over towards vehicle Sunday <unk> for an update on our financial apartment Sunday.
Thank you to come or and good morning, everyone. This morning, we issued our fourth quarter earnings release, and father 10-K, or you'll find the details of our fourth quarter and full year of 2023 financial and operating results.
Our financial performance is also shown on slides 10 13.
We finish the year with strong momentum across the business, helping us deliver solid growth across several key metrics.
Reported $582 million in annual Wake X net revenue.
<unk>, our third year of profitability, along with robust cash generation.
Dr. Jeffrey Dano: In conclusion, we are very pleased with our strong financial performance in 2023 and remain well positioned to continue growth in 2024 and beyond. We look forward to leveraging our financial strength, continuing to expand and diversify the portfolio while also returning capital to shareholders via our share repurchase program. And with that, I'd like to turn the call back to Jeff for his closing remarks.
In addition, we advance our <unk> and lifecycle management program and executed on business development with the acquisition of <unk>.
We also deployed capital towards our share repurchase program.
Overall, we remain well positioned to execute on our growth strategy moving forward.
So let me take a moment to review our financial results in more detail.
For the fourth quarter of 2023 reported our strongest revenue quarter and company history with net revenues of $168 $4 million compared to $128.3 million in the prior quarter.
Dr. Jeffrey Dano: Looking ahead, we see durability in our core business, strong momentum in our development programs, and remain focused on advancing our pipeline across both our Pitocin and ZYN002 clinical development programs, and Deploying Capital to Maximize Shareholder Value through our share repurchase program and business development opportunities. I am excited for what is to come this year and believe that we are poised to accelerate Harmony's growth in 2024. This concludes our planned remarks for today. Thank you for joining our call, and I will now turn the call back over to the operator to facilitate the Q&A session. Operator, can you please open the call to questions? Thank you. At this time, if you would like to ask a question, please press star one on your telephone keypad. If you wish to remove yourself from the queue, you may do so by pressing star two.
Presenting it broke up 31%.
Performance and a quarter reflects the strong continued underlying demand for <unk>.
And the fourth quarter of 2023, operating expenses or $85.1 million compared to $53.8 million in the prior year quarter.
Higher operating expenses were primarily driven by our advancement of our clinical development programs, including Z Y N 002 <unk>.
Expenses related to the commercialization of wake X and one times and Narva transaction related costs of $9 $8 million.
Operating income for the fourth quarter of 2023 with $42 million compared to $47 $6 million in the prior year quarter.
non-GAAP adjusted net income for the fourth quarter of 2023 was $42.8 million or 73 cents per diluted share.
Compared to $61.9 million or one dollar and one cents per diluted share in the prior year quarter.
We believe non-GAAP adjusted net income better reflects the underlying business performance.
Operator: We remind you to please pick up your handset and to please limit yourself to one question and one follow-up. We'll take our first question from Amy Farrada with Neumann Company. Your line is now open.
Please refer to our press release for a reconciliation of gap and non-GAAP results.
We ended the year with $425 $6 million cash cash equivalents and investment securities on the balance sheet.
Unidentified: Good morning. Thanks for taking my question, and thank you for giving all the updates on the pipeline. First, I have two questions. First, can you talk about the two new petroleum sand formulations on which you're going to be sharing data in the first half? What should we expect to see from the data? And, just to set expectations for investors, will we be able to get visibility into how these formulations may be differentiated from WCAGs based on that data? And then the second question is regarding idiopathic hypersomnia.
The balance reflects continued strong cash generation with approximately $77 million in cash from operations in the fourth quarter.
That that is a nearby acquisition costs and share repurchase activities and a quarter.
For the full year of 2023, we generated $219 million in cash for operations.
During the fourth quarter, we return capital to shareholders and repurchased approximately 1.8 million shares of common stock for $50 million, bringing.
Bringing the full your total of repurchase activities to 3.2 million shares of common stock for $100 million.
Moving forward, we expect to continue to opportunistically repurchase shares under the reminding share repurchase program authorization of $150 million.
Unidentified: Could you share any additional color on the analysis that you've put together for your meeting with the FDA and could you comment on whether there's any possibility of generating any additional data from the open-label portion of the study to support an approval? Thank you. Yeah, Ami, good morning. Thank you for your questions, and I'll ask Kumar to comment on, you know, the next-gen formulations and, you know, some visibility there, as well as our preparation for the FDA meeting on IH. Kumar?
A strong balance sheet allows us not only to execute on return of capital, but also maintain financial flexibility to execute on business development.
And the fourth quarter, we closed the acquisition of <unk>, which was treated as an asset acquisition for accounting purposes.
As previously disclosed we paid approximately $60 million in cash for the closing of the transaction.
We received approximately $26 million of cash and $45 million in deferred tax asset from the acquisition and assumed approximately $14 million in liabilities.
Dr. Kumar Bidur: Yeah, good morning, Ami. Thanks for the question. In terms of next-gen formulations, as we mentioned, we made progress on next-gen formulations last year, and we were really pleased to bring both of those two formulations into the clinic in the fourth quarter, and, as we previously said, we will provide data from the next-gen formulation in the first half of this year. In terms of next-gen one, or NT1, it's a modified pitolosan formulation with the Potential for Clinical Different The nature of the clinical study and the potential differentiation that will be offered will be discussed at the next earnings call along with the PK date. With the NexGen2 or NG2 formulation, it's an enhanced formulation of Pitola's band designed to deliver optimized decay and also an ability to go to a higher dose strength.
That's when we recorded $2.3 million in I T R&D charge related to the acquisition along with $7.5 million in one time restructuring costs.
As a result, we were able to acquire too late stage programs at very attractive deal terms.
Going forward, we will we will recognize considerable operating synergies and expect ongoing incremental costs from the dinar about development program of approximately $30 million for 2024.
Turning to 2024 guidance for the full year, we expect net revenues of $700 million to $720 million.
Sprained reflects are thoughtful and balanced approach to providing guidance for the first time in company history.
It also highlights than we are well on our way towards the 1 billion plus opportunity an adult narcolepsy alone that we have consistently communicated and remain confident in.
And finally, a comment on seasonality as you think about the pacing of revenue for the first quarter of 2024.
We expect to see the typical seasonal dynamics that the industry as a whole the experiences each year in Q1 higher breast and that deductions due to insurance plans to reset and higher copay obligations, along with a drawn out and train inventories.
Dr. Kumar Bidur: This is the formulation that will have a full development program, a new IP, and it will extend the Bitolus franchise well beyond 2040. And we plan to provide the PK data from this formulation at our next earnings call. Regarding your question about idiopathic hypersomnia, Ami, the totality of the data from the open-label study, from the randomized treatment period of the study, and the long-term extension study supports pituitary efficacy in patients with idiopathic hypersomnia. And in terms of the arguments for the discussions that we'll be having with the FDA, it's the totality of the data, the Especially in the context of only one trust, that happens to be a Scheduled 3 controlled substance, and the off-label use of Class 2 controlled substance trusts that have their own space to compile.
In conclusion, we're very pleased with our strong financial performance in 2023 and remained well positioned to continue growth in 2024 and beyond.
We look forward to leveraging our financial strength as we continue to expand and diversify the portfolio. While also returning capital shareholders B R. A share repurchase program.
And with that I'd like to turn the call back to Jeff for his closing remarks, Jeff.
Thank you Sunday.
In summary, harmony continues to be a gross story as evidenced by the significant progress our team made in 2023.
Looking ahead, we see durability and our core business strong momentum in our development programs.
And remained focused on <unk>.
Continuing to grow our wake X business, and helping even more adult patients living with narcolepsy.
Advancing our pipeline across both are <unk> N Z Y N 002 clinical development programs.
Dr. Kumar Bidur: So all these factors offer a strong benefit-risk proposition to bring pitolacin for patients with idiopathic hypothermia, and we hope to have good discussions with the FDA and find an efficient path forward to bring pitolacin to patients with idiopathic hypothermia as soon as possible. Thank you. Your line is open.
Working with our partner bio <unk> on next Gen formulations to extend that to tell us in franchise to help even more patients living with rare neurological diseases.
Continuing to build out our pipeline through business development to create a robust portfolio of rare disease assets covering all stages of development.
And deploying capital to maximize shareholder value.
Through our share repurchase program and business development opportunities.
Unidentified: Yeah. Hey, good morning, Jeff and team. Congratulations on a good year in 23.
I am excited for what is to come this year and believe that we are poised to accelerate harmonies growth in 2024.
Unidentified: Thanks for taking our questions. I have one commercial question and then a pipeline question to follow. So regarding the commercial question, I absolutely appreciate the 25 revs guide. Looks good to me. I note that you've added about 350 patients on WCAG per quarter for the last three quarters. And that seems to be just a really consistent number.
This concludes our plan remarked for today.
Thank you for joining our call and I will now turn the call back over to the operator to facilitate the Q&A session. Operator can you. Please open the call to questions.
Thank you at this time, if you would like to ask a question. Please press the star one on your telephone keypad.
If you wish to remove yourself from the queue. You may do so by pressing star team. We remind you to please pick up your handset and can please limit yourself to one question and one follow up we'll take our first question from Amy Sky.
Unidentified: And I guess I'm wondering if you could speak to anything in the market dynamics or prescriber access that results in that 350, or is that just serendipity? Thanks. Good morning, Charles.
With the only company your line is now okay.
Good morning, Thanks for taking my question and.
Unidentified: Thank you for your question. Yeah, I think Jeff Dirks can provide some color on the patient ads and the continued market opportunity for Wacox.
Thank you for getting all the updates on the pipeline Firstly I have two questions first can you talk about the two new <unk> formulation.
Jeffrey Dirks: Good morning, Charles, and thanks for the question. Yeah, we've been extremely pleased with the durable growth that we've seen in the average number of patients on Wacox, and I think some of the things that we're seeing, Charles, is given the unique and meaningfully differentiated product profile, Wacox has the ability, and our sales team has the ability, to engage with the broad 9,000 approximate healthcare professionals that see and treat the narcolepsy patients, and what that does is that affords us the opportunity to tap into the full diagnosed patient opportunity, both those individuals and healthcare professionals outside the Oxybate REMS program, which we continue to see meaningful growth in the number of unique writers every quarter, but even within the Oxybate REMS enrolled healthcare professionals, even with the availability of generic and once nightly Oxybate launches this year, we're seeing meaningful growth, excuse me, in terms of depth of prescribing in that audience, so I think really what we're seeing is the unique nature of the meaningfully differentiated product profile, the non-scheduled status, the broad clinical utility that Jeff Dano spoke about, that really affords us the ability to continue to tap into that broad diagnosed patient opportunity as the market allows around, you know, the traditional quarterly, you know, dynamics that we speak about each earnings call, but Charles, we're extremely pleased with what we're seeing as I'm hearing from you as well, and we're confident in continued growth in the average number of patients as we move into 24. Okay, that's helpful. I added color, Jeff.
Gonna be sharing data in the first half what should we expect to see from the data and just just set up expectations for investors.
Would you be able to get disability and to have the foundations, maybe differentiated from <unk> based on that data.
And then the second question is regarding idiopathic hypersomnia, if you could shed any additional color on the analysis that you've put together for your meeting with the F. D. A N.
And if you could comment on whether there's any possibility of generating any additional data from the open David portion of the study.
<unk>. Thank you.
[noise] yeah good.
Good morning, Thank you for your questions and I'll ask him or to yeah to comment on the next gen formulations, and some visibility there and as well as our preparation for the F. D. A meeting on IH.
Yeah good.
Good morning, <unk>. Thanks for the question.
In terms of mixed in formulation asked we mention we made.
<unk> formulation last year, and we were really pleased doping both of those two formulations seemed to the clinic in the fourth quarter and ask you previously said we.
We will provide.
Data from the next information in the first half of 50 years.
Most of next Gen. One R N T. One.
It's a modified for developing formulation.
<unk> for clinical differentiation crossed the market started the by demonstrating equal then how.
Alongside with clinical study that will offer a meaningful clinical defense decent location.
The nature of the clinical study and a potential defense stations that could be offered will be discussed that'd be mixed earnings call along with the PK data.
With the next June two N T. Two formulation, it's an enhanced formulation of the total thing designed.
Unidentified: Thanks. A quick question on the pipeline. I'm wondering when you would anticipate data, how rapidly you think that study can enroll given some competing programs, and then the NDA, and SNDA timing. I assume it would be gated by the open-label extension, or would you file perhaps before that's completed? And then the second part of the question is regarding pediatric narcolepsy and priority review. It seems to say something about the agency's perspective on the safety of the product, Yeah, Charles, first, let me say that we're excited to be initiating the phase three, you know, TEMPO study in patients with Prader-Willi. I'll have Kumar comment on that, and then I'll circle back on some thoughts about the priority review for the pediatric narcolepsy SNDA. Kumar?
Designed to tell you what they might be good.
Also.
And the ability to go to a higher strength.
This is the formulation that will have a fruit eloquent broke up a new IP and it will extend that mcdonalds franchise, well beyond 2040 <unk>.
We plan to provide the speaker data from these formulation, but our next call.
Regarding your question about that idiopathic hypersomnia homey, the totality of the from the open label study from the randomized.
Don't period of the study and the long term extension study.
<unk> <unk>, if he could be in patients with idiopathic hyper Columbia and in terms of the.
The arguments of the discussions that we will be having with the F. D. A is the totality of the data.
The Nonscheduled startup dollar thing.
The ease of administration.
<unk> nine safety profile.
Especially in the context of <unk> <unk> that it happens.
<unk> <unk> <unk> three controlled substance.
Off label use of plus two controlled substance drug that how that will be.
Dr. Jeffrey Dano: Yeah. Hey, good morning, Charles. Thanks for the question. Regarding the PWA study, first of all, we are very pleased to have the orphaned designation from the FDA for the PWS program. In terms of recruitment, we anticipate to complete enrollment in about two years. It is true, Charles, that there are multiple programs in this space, but then we are the only ones who are looking at excessive daytime sleepiness and behavioral symptoms in PWS. The other programs are targeting more towards the hyperphagia in PWS. And, as I mentioned previously, more than half of the patients with PWS have excessive daytime sleepiness, and almost all of these patients have behavioral disorders.
So all of this factor offered straw.
Strong benefit proposition to bring the total of plan for patients with idiopathic hypersomnia and we hope to have good discussions with the F D a and find an efficient wildwood.
<unk> patients with the beauty of other <unk> from you as soon as possible.
Thank you.
I think our next question from Charles Tucker with Cantor Fitzgerald. Your line is okay [noise].
Yeah, Hey, good morning, Jeff and team congratulations on a good year and twenty-three.
Thanks for taking our questions I have one commercial question and then and then a pipeline question to follow so regarding the commercial question absolutely appreciate the 25 Red Sky.
It looks it looks good to me I know that you've added about 350 patients on wake X per quarter for the last three quarters.
Dr. Kumar Bidur: And from our proof-of-concept study, we showed strong data supporting pitolescent efficacy, both in excessive data and sleepiness. Yeah, and Charles, can you repeat that second question about the PEEJ and Oracle FC, SNBA? Yeah, I mean, the pediatric narcolepsy, you know, PDUFA, and rapid time to respond, you know, just seems to say something about the safety of the product or the agency's perspective on that. And not speaking for the agency, of course, but that combined with the priority review, I guess, do you have any thoughts about the agency's perspective on that? Yeah, Charles, I mean, what I would say, you know, I think that, as we've shared this week, we're very pleased with the interactions we've had with FDA with regard to the orphan drug designation for Pitocin for Prader-Willi and then followed by the decision on priority review for the pediatric narcolepsy SNDA.
And that seems to be just a really consistent number and I guess I'm wondering if you could speak to anything in the market dynamics are prescribed or <unk>.
Access that results in that 350 or is that just serendipity. Thanks [noise].
[noise] good morning, Charles Thank you for your question. Yeah. I think you know <unk> can provide some color on the patient ads and the continued.
Market opportunity for wake, it's sure good.
Good morning, Charles and thanks for the question Yeah, we'd been extremely pleased with the durable growth that we've seen in the average number of patients on <unk> and I think some of the things that we're seeing Charles is given the unique and meaningfully differentiated product profile wake X has the ability and our sales team has the ability to engage with abroad 9000 approximate healthcare.
<unk> the narcolepsy patients and what that does is that affords us the opportunity to tap into the full diagnosed patient opportunity both those individuals and healthcare professionals outside the Octavate Rems program, which we continue to see meaningful growth in the number of unique writers every quarter, but even within the ox abate rems enrolled hell.
Dr. Kumar Bidur: You know, I think that, you know, it reflects, you know, with regard to the overall benefit-risk profile of Pitocin and what we have said all along. And advancing the development programs, we don't have any concern with regard to, you know, that the safety profile has been consistent from the original pivotal development program in narcolepsy and on through the life cycle management programs that we've been conducting with, you know, no real change in the overall safety profile and the favorable benefit-risk profile. I think that reflects the truth.
Care professionals, even with the availability of generic and once nightly octavate launches. This year, we're seeing meaningful growth <unk> excuse me in terms of depth of prescribing in that audience. So I think really what we're seeing is unique nature of the meaningfully differentiated product profile. The nonscheduled status the broad clinical utility that Jeff Daniels spoke about that.
Really affords us the ability to continue to tap into that broad diagnose patient opportunity as the market allows around you know the traditional quarterly you know dynamics that we speak about each earnings call, but Charles were extremely pleased with what we're seeing as I'm hearing from you as well and we're confident and continued growth in the average number of patients as we move into 24.
Okay. That's helpful added color chess. Thanks quick question on the pipeline.
Unidentified: The positive interactions we've had with the agency, the decisions that we've received, and we remain focused on the execution and advancement of those development programs to bring potential new treatment options to those patient populations. Very good. Thanks for the added info. Congratulations on a good decision. Thanks, Charles.
Actually a multi part question sorry about that and that is appreciate the phase III tempo study data or not data excuse me details I'm wondering when you would anticipate data how rapidly do you think that study Kenneth Royall, given some competing program.
Unidentified: Thank you. We'll take our next question from Francois Brisbra with Oppenheimer. Your line is now open. Hi, can you hear me okay?
And then is the N D. A S N D. A timing I assume that would be jaded by the open label extension or would you file perhaps the four that's completed and then the second part of the question is regarding the Paediatric narcolepsy and priority review it seems to say.
Unidentified: Thank you for taking the question. So I was just wondering, in terms of the guidance, can you help us understand what the thought process was behind giving guidance for the first time here, and how should we be thinking about revenue per patient in 2024? Thanks Frank. Sandip?
Something about the agencies perspective on the safety of the product. So I'm wondering if you could provide any any <unk> and recent pretty review being <unk>.
Sandip S. Kapadia: Yeah, sure. Look, Frank. Thanks for the question. I mean, our guidance represents what I'd say is a thoughtful and balanced approach, especially the first year that we're given guidance. You know, I think we certainly took opportunities to provide an update, you know, as we go throughout the year with respect to the guidance. How we should think about, you know, pricing or maybe revenues. Yeah, I think what we did with our price increase earlier this year was about seven percent. The portion of that usually falls through the bottom line. So I think, you know, generally, you'll see an impact from that as we go throughout the year. Obviously, in the first quarter, there's usually headwinds regarding, you know, gross pressure and normal things that happen typically during seasonality.
Yeah, Charles first let me say you know I I think that.
We're excited to be initiating the phase three temper study in patients with a crowd of Willie I'll have Kumar comment on that and then I'll circle back on some thoughts about the prior to review for the pediatric Narcolepsy <unk> Kumar Yeah. Good morning to authentic the question regarding the <unk>.
PW with 31st of all we are really pleased to.
To have the often practiced technician from the F D. A fluffy there'll be with the program.
Recruitment.
Anticipate to complete and <unk>.
Two years.
It is true Charles but there are multiple programs in this space, but then we have the only one who are <unk>.
Looking at the excessive daytime sleepiness and behavioral symptoms T. W. S. The other programs targeting more towards the hype of <unk> in T. W. At Yahoo.
Sandip S. Kapadia: You generally don't see as much of that in the first quarter, but generally, as we go throughout the year, you'll see the benefit. It's very similar to the pattern that we've had over the last, you know, three, four years. Great, thank you very much. Thanks, Frank.
I mentioned previously more than half of the patients with T. W. A topic 50 per cent sleepiness and almost all of the patient of behaving just opened.
Promote proof of concept study resort strong data supporting vitolo, if it could be important exit through data and sleepiness.
Unidentified: Thank you. We'll take our next question from David Amsellem with Piper Sandler. Your line is now open. Yeah, thanks. So just a couple.
Yeah, and Charles can you repeat that second question about the the piece narcolepsy Sandia yeah.
Yeah, I mean I I.
Unidentified: First, regarding business development, wanted to get more detailed thoughts on the extent to which you do a larger-scale transaction versus something smaller-scale along the lines of Zainerba. Just philosophically, you know, how are you thinking about acquisitions in general? And tied to that question is also, would you lever up and lever up significantly to do something?
The pediatric narcolepsy.
You know <unk> and am rapid rapid time to to respond you know just seems to say something about the safety of the product or the agency's perspective on that and not speaking for the agency of course, but that combined with the priority review I guess do you have any thoughts about.
Unidentified: So that's number one. And then, number two, I wanted to pick your brain on the orexins. We've seen some data from Takeda, from Alchemies. But there are others, of course.
The prospective stomach.
Yeah, Charles I mean, what I would say you know I think that as.
As we shared this week you know we're very pleased with the interactions we've had with FDA with regards to the orphan drug designation for <unk> for proud of Willie and then followed by the decision priority review for the pediatric narcolepsy us in da.
Unidentified: Wanted to get your view on the potential long-term impact of orexin receptor agonists to the extent one or more reach the market. Thank you. Thanks David, good morning. I think with regard to, you know, business development, we are, you know, assessing the landscape very actively, and we're open to sort of all opportunities. With regard to a deal similar to, you know, Zainerba, that's a good strategic fit, as well as something more, you know, transformational. We were in a strong, sort of, you know, financial position to transact. So, we first look for good strategic fit, prefer sort of late-stage assets but are open to earlier stage, and those that can launch both during the WCAG life cycle and after. In terms of capacity, I'll ask Sandip to, you know, comment on that. Thanks, Jeff. Yeah, look, we have good financial strength and flexibility, you know, to execute on business development. We also have access to debt, if appropriate, as well as the capital markets.
I think that.
It reflects with regards to the overall benefit risk profile and <unk> and what we have said all along and advancing the development programs.
We don't have any concern with regards to the.
Safety profile has been consistent from the original pivotal development program in narcolepsy.
And on through the lifecycle management programs that we've been conducting with no real change in the overall safety profile and the favorable benefit risk profile I think that reflects.
The positive interactions you've had with the agency the decisions that we've received and we remain focused on the execution and advancement of those you know development programs to bring potential need treatment options to those patient populations.
Very good thanks for the added info congrats on a great deal.
Thanks, Charles Charles.
Thank you we'll take our next question from Francois prescribed with Oppenheimer and I just now open.
Hi can you hear me okay.
Yep, that's right Yep, Okay. Thanks for taking the question Uhm just so I was just wondering in terms of the guidance can you help us understand what the thought process was behind giving them guidance for the first time here and how should we be thinking about the revenues for patience Uhm in 2024. Thank you.
Thanks, Frank Sunday.
Yeah sure look.
Frank Thanks for the question I mean, our guidance represents what I'd say, it's a thoughtful and balanced approach, especially the for the first year, they're giving guidance I think we certainly opportunities to provide an update is.
We go throughout the year.
Sandip S. Kapadia: As we mentioned, we had over, you know, $425 million of cash and cash equivalents available. We're generating significant positive cash flow. Last quarter, we generated about $77 million in cash flow generation, as well as almost over $219 million last year in cash generation.
With with respect to the guidance.
How we should think about you know pricing or maybe revenues per day I'm sorry.
Yeah, Yeah, I think like we we did take our price increase earlier. This year was about 7% a good portion of that usually follows through the bottom line. So I think you know generally.
See an impact from from that because we go throughout the year, obviously in the first quarter, there's usually headwinds regarding gross to that pressure and normal you know things that happen typically in the seasonality that happened to you generally don't see as much of that in the first quarter, but generally as we go throughout the year, you'll <unk>, you'll see the benefits is very similar to the pattern.
Sandip S. Kapadia: So, I think we have good flexibility, as Jeff mentioned, to look at, you know, both small add-on transactions as well as potentially something that's larger and transformative. But I think the important thing is the filter that Jeff mentioned up front, in terms of making sure it's a good strategic fit for the company. And I think from a capital perspective, we have broad flexibility. Thanks, Sandip.
That we've had over the last three or four years.
Great. Thank you very much.
No. Thanks right.
Thank you we'll take our next question from David Ameslan with Piper Sandler Your line is now okay.
Yeah headaches. So just a couple first regarding business development wanted to get more detailed thoughts on the extent to which you do a larger scale transaction versus something smaller scale along the lines of <unk>.
Dr. Jeffrey Dano: And, David, turning to a question about orexin agonists, I mean, you know this space well, as do we, and we've been following it closely. So, you know, obviously, a lot of attention lately, given some of the data that has come out. I think at a high level, you know, the programs are still early, and they're obviously advancing. I think questions are being generated, you know, both on the safety side and the efficacy side, you know, based on the data that has been coming out. Obviously, the target is interesting, you know, in terms of the next potential novel mechanism of action for narcolepsy and other central disorders of hypersomnolence. So, I think we are following the space closely as the data come out, as more questions are generated. And, you know, I think that they will likely come to market as successful towards the end of the decade, towards the end of the WACIC life cycle.
Just philosophically.
How are you thinking about acquisitions in general and tied to that question is also would you <unk> and liver up significantly to do something so that's number one and a number two wanted to pick your brain on the erections, we've seen some some data from Takeda.
From Alchemy is there are others of course wanted to get your view on the potential long term impact of the redfin to reset their agnes to the extent one or more reached the market. Thank you.
Yeah. Thanks, David Good morning, Uhm, I think with regards to business development. We are we are assessing the landscape very actively and we're open to sort of all opportunity with regards to the deal similar to.
Zeiner about that's a good strategic fit.
As well as something more transformational we are in a strong sort of you know financial position to transact. So first look for good strategic fit prefer sort of late late H assets, but open to earlier stage.
Dr. Jeffrey Dano: But importantly, it's our belief that narcolepsy will continue to be a polypharmacy market. New entrants can only help the space and more share a voice, and more education. So an interesting novel target.
And those that could launch both during the Waco lifecycle N and after.
In terms of capacity I'll ask some deep to comment on that.
Dr. Jeffrey Dano: I think that waits to be seen as these development programs advance. And we'll continue to be a polypharmacy market. And we'll follow them as things go forward. All right, thanks.
Thanks, Yeah look we have a good financial strength and flexibility to execute on business development. You know, we also have access to that if appropriate as well as the capital markets.
Also we mentioned we had over four.
Operator: Thank you. Thank you. We'll take our next question from Jason Gerberry with Bank of America. Your line is open. Hey guys, thanks for taking our questions. This is Tava on the floor with Jason.
$425 million in cash and cash per month available, we're generating significant positive cashflow last quarter was about $777 million in cash flow generation as well, it's almost over $219 million last year in cash generation. So I think we have a good flexibility as Jeff mentioned to look at both.
Unidentified: Our first question is with regard to the Zygel asset. Where are you at with enrollment for the Phase 3 Fragile X Syndrome study? And any added clarity as to...
Small add on transactions as well as potentially something that uhm or larger and transform it but I think the important thing is the filter that John mentioned upfront in terms of making sure that good strategic fifth for the company and I think from a capital perspective, we have broad flexibility.
Unidentified: Shape Your View on mid-2025 readout timing. And then, the second commercial question: what are your assumptions for average revenue per week expansion? Is that a meaningful tailwind for revenue growth in 2024 in your guidance, or is growth mainly driven by volume? Great, thanks for your questions.
Thanks Sunday.
And David turning to question about the Redfin agonists I mean, you know this space well as as Dewey and we following it closely so obviously a lot of attention lately.
Given some of the data that has come out.
Think at a high level. The programs are still are still early and they're obviously advancing I think questions are being generated.
Dr. Jeffrey Dano: I'll ask more to comment on the ZYN002 phase 3 ReConnect trial. Very exciting opportunity for us and can provide, you know, kind of some more color on where we are there. Yeah, yeah, thank you Jeff.
Both on the safety side in the efficacy side, you know based on the data that has been coming out obviously the target is is interesting you know in terms of the next potential.
Novel mechanism of action or for narcolepsy, and other central disorders of hyper somnolent. So I think we <unk> following a space closely.
Dr. Kumar Bidur: So yeah, we looked at the timeline, as we mentioned previously that we would, and based on the historical recruitment trend and the anticipated recruitment in this particular patient population, we estimated that we would complete enrollment in the first quarter of 2025, and the top line would be in mid-2025. Now that the asset is within half, obviously, we will bring all the additional resources and the expertise not just from a clinical development perspective but also from an advocacy group perspective as well to try and accelerate the recruitment. Thanks, Kumar.
As the data come out as more questions have been generated and uhm.
You know I think that.
<unk> likely.
Come to market is successful towards the end of the decade towards the end of the way could play cycle, but importantly.
It's our belief that narcolepsy will continue to be a polypharmacy market.
Mmm, new entrants can only help you know the space and more share a voice more education. So interesting novel target I think wait to be seen as the development programs advance and.
Will continue to be a polypharmacy market and Wohlfeil then as things go forward.
Alright. Thanks.
Yeah. Thanks, David.
Thank you we'll take our next question from Jason Carberry with Bank of America. Your line is okay.
Yeah. Thanks for taking your questions <unk> on for Jason. Our first question is regarding with regards to the <unk> where are you at with enrollment for the phase three fragile X syndrome study and any added clarity as to assumptions shape. Your view on mid 2025 read out timing.
Sandip S. Kapadia: In terms of revenue per patient, Jeff Dirks, do you want to comment on that? Sure. So, in looking ahead in 24, you know, questions in terms of just price, volume, or average revenue per patient, as Sandip shared, we took a 7% price increase in the beginning of the year, and as with previous years, we would expect to capture most of that price increase. And as you're looking at our guidance, right from 700 to 720, you can see that we're expecting 20-plus percent growth. So, you can see that the vast majority of the growth we're expecting in 24 is driven by volume.
And then a second commercial question what are your assumptions for average revenue per week. Each station is that a meaningful tailwind for revenue growth in 2024 in your guidance or is growth mainly driven by volume.
Thank you.
Great. Thanks for your questions, which we all have somewhere to comment on the <unk> Z Y N 002 phase three reconnect trial very exciting opportunity for us and can provide you know kind of.
Some more color on where we are there yeah.
Thank you, Jeff So we looked at the timeline.
Previously the female.
Jeffrey Dirks: You know, we've talked a lot about the large remaining diagnosed patient opportunity and the meaningfully differentiated product profile this week. So, we see significant opportunity and room for growth, but hopefully, that provides a little bit of context and helps you think about price versus volume assumptions and the average revenue per patient in 24. Okay.
Based on the historical recruitment trying and be anticipated groups 19. This particular patient population <unk> that we will complete enrollment in the first quarter of 2025 and the phone line will be.
2025.
No that'd be that'd.
That'd be <unk>.
Obviously, we will being on the additional resources <unk> not just from a clinical <unk>.
Unidentified: Thanks, Jeff. And if I could have one follow-up question, what does the appetite for share buybacks in 2024 look like relative to the hundred million in share buybacks that you guys did in 2020? You know, as we said, we're looking to take an opportunistic approach, and Sandip, do you want to comment further? Yeah, look, we were very pleased last year that we were able to do approximately $100 million of share repurchase activities over the last couple of quarters. And you know, and I think it's really not a question of either or.
Also the advocacy group.
Two of <unk>.
Yep, Thanks to more in terms of revenue for patient <unk>, you want to comment on that sure. So and looking ahead. In 24 question in terms of just price volume or average revenue per patient is sandeep shared we took a 7% price increase in the beginning of the year and as with previous years, we would expect to capture most of that price increase and as you're looking at our.
Guidance right from 700 to 720, you can see that we're expecting 20 plus per cent growth. So you could see that the vast majority of the growth. We're expecting in 24 is driven by volume.
Yeah, we've talked a lot about the large remaining diagnose patient opportunity the meaningfully differentiated product profile for week. So we see significant opportunity of room for growth, but hopefully that provides a little bit of context and helps you think about price versus volume assumptions and the average revenue per patient of 24.
Sandip S. Kapadia: We have the flexibility to do both, and you know, and with respect to business development, we obviously talked about our strategy there. And then in terms of the share buyback, again, we'd look at it more opportunistically, and at the appropriate levels, we would certainly be in a great cash position to be able to accomplish that. And we have approximately $150 million of capacity still remaining.
<unk> Thanks, Jeff.
And if I could have one follow up question what is the appetite for share buybacks in 2024 looked like relative to the hundred million dollars and share buybacks that you guys did in 2023.
I think as we said look into taken opportunistic approach and Sunday.
<unk> comment further yeah look we were very pleased last year that we were able to do approximately $100 million a share repurchase activities over the last a couple of quarters and you know and I think it's really not a question of either or we have the flexibility to do both and you know and <unk> and respected business development. We obviously talks about our strategy there.
Unidentified: Thank you. Thank you. Thank you. We'll take our next question from Danielle Brill with Raymond James. Your line is now open.
And then in terms of share buyback again, we'd look at it more opportunistically and at the appropriate levels, we would certainly.
Unidentified: Hey guys, good morning. Thanks for the question. I was also hoping you could provide some more color on the assumptions that went into your 24 guidance, specifically around expectations for patient ads. It looks like you're expecting a slowdown in that patient ad each quarter. Are you just being conservative here, or is growth starting to moderate?
Great cash position to be able to execute.
And we have approximately $150 million of capacity still remaining.
Thank you alright.
Thank you.
Thank you we'll take our next question from being <unk> with Raymond James Your line is open.
Hi, guys. Good night and thanks for the question I would also hoping you can provide some more color on the assumption that went into your 24 guidance.
Unidentified: And then, as a follow-up, can you remind us what impact label expansion into the pediatric narcolepsy population will have on the overall TAM for WCAG? Thank you. Thanks, Danielle, and good morning.
Typically around expectation for patient ads, it looks like you're expecting a slowdown in that patient at each corner or are you just being conservative here are as Chris starting to moderate and then as a follow up can you remind us what impact the labour expansion into the pediatric narcolepsy population will have any overall tam for <unk>. Thank you.
Unidentified: Yeah, with regard to, you know, I think that the revenue guidance and, Sandip, do you want to, you know, Yeah, look, I think, as we mentioned, we've taken, you know, a thoughtful and balanced approach in terms of providing guidance for us for the first year. We certainly look to provide an update as we go throughout the year. We're not in any way indicating that.
Yeah, Thanks, Danielle and good morning, Yeah with regards to you know I I think that the in terms of revenue in the revenue guidance and.
Sunday, if you want.
I don't know I think as we mentioned we've taken you know a thoughtful and balanced approach in terms of providing guidance for us for the first year and I would certainly look to provide an update as we go throughout the year, we're not in any way, indicating we see sprite momentum coming out of the fourth quarter, and we expect that momentum to sort of carry through for the year I mean, Jeff <unk>.
Sandip S. Kapadia: We see great momentum coming out of the fourth quarter, and we expect that momentum to sort of carry through for the year. I mean, Jeff, Dirk, do you want to comment at all on just overall growth and what you're seeing? Sure, and Danielle, in terms of looking at average patient growth and sort of the assumptions in the 24 guidance, I mean, if you've seen, we've seen demonstrated durable growth in a narcolepsy market, and we've grown average patients every quarter since launch, even with availability, but within that durable growth, that growth has evolved every year, and entering our year five of our rare orphan launch, we're going to expect that So you'll see traditional payer headwinds in the first quarter that impact all brands and specialty products. You'll see the traditional, you know, fewer patient visits associated with chronically managed conditions and diseases in Q3.
<unk> at all in terms of just overall.
What you're saying sure and Danielle in terms of looking at it average patient growth and sort of your assumptions and the 24 guidance I mean, if you seen we've seen demonstrated durable growth in in narcolepsy market and we've grown average patients every quarter since launch even with availability, but within that durable growth that growth has evolved every year and entering our <unk>.
Five of a rare often lunch, we're gonna expect that growth to continue to evolve but follow the previous year's cadence of the quarterly growth as we tap into that large diagnose patient opportunity. So you'll see the traditional pair headwinds in the first quarter that impact all brands of specialty products, you'll see the traditional you know fewer patient visits associated with chronically manage conditions and diseases in Q3.
Jeffrey Dirks: And then, typically, in previous years, we expect to have the traditional tailwinds in the second and the fourth quarter with stronger prescription demand. You know, again, but I think the takeaway is we're extremely pleased with what we're seeing, confident continued growth, and we really believe that Wacox is well positioned for future growth in 24. Thanks. And, Daniel, your next question on... It was on pediatric TAM and how that expands the WCAG opportunity. Yeah, I think Jeff can speak to that. I think obviously there are positive signals from the agency on the importance of that indication in pediatric narcolepsy patients. So, Jeff, you know, what does the opportunity look like?
Three and then typically in previous years, we expect to have the traditional tailwinds and the second in the fourth quarter was stronger prescription demand you know again, but I think the the takeaways. We're extremely pleased with what we're seeing I'm confident continued growth and we really believe it wakes as well positioned for future growth in 24.
Thanks, and then your next question on.
It was on pediatric Tam and how that expanse.
<unk>, Yeah, Yeah, I think Jeff can speak to that I think obviously, you know positive signals from the agency of the importance of identification in pediatric narcolepsy patients. So Jeff what what's the opportunity looked like sure. So I mean, Daniel we estimate there's about 4000 pediatric narcolepsy patients in the in the U S.
Dr. Jeffrey Dano: Sure. So, Daniel, we estimate there are about 4,000 pediatric narcolepsy patients in the U.S. And although it's not a large opportunity, it's an important one. And it's a very underserved patient population, you know, with only one FDA-approved treatment option being sodium oxidate, which is a Schedule III REMS product right now.
And although it's not a large opportunity it's an important one and it's a very underserve patient population with only one F. D. A approved treatment option being sodium <unk> being a schedule three <unk> product right now and I think we're very excited about the potential if successful to bring a nonscheduled treatment option given what we've seen the impact of of narco.
Dr. Jeffrey Dano: And I think we're very excited about the potential, if successful, to bring a non-scheduled treatment option given what we've seen, the impact of narcolepsy on these individuals' lives. Thanks, Jeff. And, Daniel, let me just also add with regard to, you know, the pediatric narcolepsy, that SNDA. It's also important that, you know, those data; we're also pursuing pediatric exclusivity, which is obviously an important commercial opportunity with regard to an additional, you know, six months of regulatory exclusivity. So, those data are one component of that, along with, you know, the data that we'll be generating in the Phase III, you know, Prader-Willi, TEMPO study. So, you know, both of these components are important, and we are advancing both of them and making progress in terms of pediatric narcolepsy data through that submission and then the initiation of the Phase III trial in Prader-Willi syndrome. And both of those components are sort of what would be required as we pursue pediatric exclusivity and an additional six months of protection. Tse, thank you so much.
I've seen on these individuals' lives yeah, thanks, Sir and Danielle Let me let me just also add with regards to the pediatrics <unk>.
<unk>. It's also important that you know those data we're also pursuing pediatric exclusivity, which is obviously an important.
Commercial opportunity with regards to an additional six months of regulatory exclusivity. So those data are one component of that along with the data that will be generating in the phase three proud of Whiley tempo study. So both of these components are important and we are.
Are advancing both of these and making progress in terms of pediatric narcolepsy data through that submission and then the initiation of the phase three trial and Potter Willi syndrome.
And both of those components are what would be required as we pursue pediatric exclusivity an additional six months of protection.
Okay. Thank you so much.
Unidentified: Thank you. Thank you. We'll take our next question from Greg Sabanovich with Mizzou.
Thanksgiving.
Thank you we'll take our next question from <unk> <unk>. Your line is open.
Unidentified: Your line is open. Hi, this is Avantika on behalf of Greg. I just have a question about the one about the Fragile X opportunity. I know you said that the timelines have been pushed a little bit due to enrollment, but were there any changes in the trial design as there originally were, which also caused delays? And then also on the pediatric opportunity, I know you said it's a pretty small opportunity, but do you anticipate growing the WIC Excel course at all if approved? Thank you for your questions with regard to the Fragile X study, Sumar. Yeah, thank you for the question, Avantika. With the Fragile X syndrome, you know, the timelines were not pushed out.
Hi, This is <unk> I just have a question about the one about the lack of opportunities I know you said that the timeline different pushed a little bit can you two enrollment.
Were there any changes in your childhood <unk> <unk>, originally where which also constantly and then also on the pediatric opportunity and then you said, it's a pretty small opportunity, but uhm do you anticipate growing the week accent of course at all if approved.
Alright. Thank you for your questions with regards to the fragile X.
<unk>.
Yeah. Thank you for the question I want to go with the <unk> the timeline pushed out.
Dr. Kumar Bidur: We mentioned that we would evaluate the timeline once the asset was in-house, and we had an opportunity to evaluate the timeline, and based on the historical recruitment trend and the anticipated recruitment in this patient population, we arrived at completing enrollment in the first quarter of 2025, with the timeline anticipated in mid-2025, and there are no changes in the studies. Yeah, and I would just add that the opportunity is for, you know, for Harmony, for us to, you know, put our resources forward to drive the Phase 3 ReConnect trial forward, with regards to the experience of the clinical development team, more capacity from an operational perspective, and really engaging with the patient community through our patient advocacy efforts as well, to drive that forward towards a significant market opportunity of 80,000 diagnosed patients in the U.S. Turning to pediatric narcolepsy, we keep hearing about sort of a small market opportunity, but an important one, and I think Jeff Zirks alluded to this, an important one in terms of potential new non-scheduled treatment options for pediatric patients living with narcolepsy, rather than Schedule II stimulants.
We had mentioned that'd be available.
This time by <unk>, and we had an opportunity to I'd rather be at the timeline and based on the historical recruitment trying.
The anticipated recruitment <unk> completing the enrollment be the first quarter of 2035 top line.
<unk> 20th May be fine.
<unk> no change in the study design.
Yeah, and I and I would just add I think that the opportunity is for harmony for us to put our resources toward driving the phase III reconnect trial forward with regards to the experience.
Of the clinical development team more capacity from an operational perspective, and really engaging with the patient community through our patient advocacy efforts as well to drive that forward toward a significant market opportunity of 80000 diagnose patients in the U S.
Turning to pediatric narcolepsy.
We keep hearing sort of small market opportunity, but put an important one I think Jeff started for alluded to this and.
An important one in terms of potential new nonscheduled treatment options for pediatric patients with narcolepsy.
Rather than scheduled to stimulant. So we are looking forward to working with the agency towards that and then with regards to.
Dr. Jeffrey Dano: So we are looking forward to working with the agency towards that goal. And then, with regard to the impact on the market opportunity, Jeff? Sure. So in looking at, you know, the Salesforce coverage for pediatric narcolepsy, we believe that we're optimized for the narcolepsy opportunity with our existing Salesforce footprint. There will be some new prescribers that we'll be adding to our target list if successful, but we believe that our current team, which is, you know, very excited about the potential to bring this forward, is optimized to be able to take on this new opportunity with these additional patients and with, you know, the addition of a handful of healthcare professionals. There's a high overlap between adult narcolepsy prescribing and pediatric narcolepsy prescribing, so we believe we've got, you know, relationships established with a good portion of those prescribers, but we are absolutely optimized with our team, and if successful, very excited to bring this new indication forward.
You know the the impact on the market opportunity just sure. So in looking at you know the the sales force covers for pediatric narcolepsy. Yeah. We believe that were optimized for the narcolepsy opportunity with our existing salesforce footprint, there will be some new prescribers it will be adding to our our target list of successful, but we believe that our current team that is.
You know very excited about the potential to bring this forward is optimized to be able to take on this new opportunity with these additional patients and with you know. The addition of a handful of health care professionals, there's a high overlap between adult narcolepsy prescribing in pediatric narcolepsy prescribing. So we believe we've got you know relationships established with a good portion of those prescribers.
But we are absolutely optimize with our team and if successful very excited to bring this new indication forward.
Jeffrey Dirks: Thanks, Jeff. Thank you. And can you just squeeze in one more? Sorry.
Alright, thanks, Sir thank you.
Thank you squeeze in one more sorry.
Unidentified: For Gross Connect, I know you said that there will be headwinds in the first quarter, but can you just quantify what you think they'll be for the full year? Thanks for the question. As I mentioned, typically in the first quarter, across the industry, there are general headwinds from just higher copay obligations and insurance plans receding in the first quarter. So we'll see a couple of percentage points impact from that, typically, which is what we've seen in the past in the first quarter. And that sort of improves as we go throughout the year, generally.
For cause I know you said that that'll be headlines in the first quarter, but can you just want to buy what you think it'll be for the full year.
Mmm Sunday.
Yeah, Hi, thanks, Thanks for the question as I mentioned typically in the first quarter across the industry you know their their general headwinds from just higher copay obligations and insurance plans resets in the first quarter. So we will see a couple of percentage points impact from that typically what we've seen in the past in the first quarter and that sort of <unk>.
Improves as we go throughout the year generally.
Sandip S. Kapadia: Thank you. Thank you. I am showing no further questions.
Thank you [laughter].
Thank you I am showing no further questions I would now like to turn the call back to management for closing remarks.
Operator: I would now like to turn the call back to management for closing remarks. Thank you, Brittany, and thank you everyone for joining our call today and for your interest in Harmony. As you heard from us this morning, our strong execution in 2023 and solid momentum going into this year positions Harmony Well for continued growth in 2024. We look forward to providing updates as we execute on our growth strategy. Thank you and have a great day. This does conclude today's Harmony Biosciences fourth quarter and full year 2023 financial results conference call. You may now disconnect your line and have a wonderful day. Hope you enjoyed it! www. kokosget.com bum bum bum, bum bum bump, Thank you, yeah thank you, and Humming, Paul Elmore Nicholas Sam Pegg, Eh! Alizade Na and Gus Offer S2 ESO, USDT Transcription by ESO-U Earlier today Transcription by ESO ESO, USDT Transcription by ESO Edited by ESO Transcription by ESO Transcription by ESO
[noise]. Thank you Brittany and thanks, everyone for joining our call today and for your interest in harmony.
As you heard from US. This morning are strong execution in 2023 and solid momentum going into this year physicians harmony well for continued growth in 2024.
We look forward to providing updates as we execute on our growth strategy.
Thank you and have a great day.
This does conclude today's harmony biosciences fourth quarter and full year 2023 financial results Conference call. He may now disconnect your line and have a wonderful day.
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