Q4 2024 Zevra Therapeutics Inc Earnings Call
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Speaker Change: Good afternoon, and thank you for joining <unk> therapeutics fourth quarter and full year 2024 financial results and corporate update conference call. Today's call is being recorded and will be available via the Investor Relations section of the company's website later today the host for todays calls Nichol Ochsner Zabriskie, Vice President of Investor Relations.
Communications.
Speaker Change: Thank you and welcome to those who are joining US today, we will provide an overview of our accomplishments in the fourth quarter and full year of 2024, followed by a review of financial results I encourage you to read our financial results news release, which we distributed this after.
Speaker Change: And is available in the investors section of our website.
Speaker Change: Before we begin please note that certain information shared today will include forward looking statements actual results may differ materially from those stated or implied by any forward looking statements due to risks and uncertainties associated with Cerberus business.
Speaker Change: Forward looking statements are not promises or guarantees and are inherently subject to risks uncertainties and other important factors that may lead to actual results differ materially from the projections made these forward looking statements should be evaluated together with the cautionary statements contained in.
Speaker Change: The risk factors section in our most recent quarterly report on Form 10-Q, our annual reports on Form 10-K, and our other filings with the FCC.
Speaker Change: I'm pleased to welcome servers management team members participating in today's call.
Speaker Change: Neil Mcfarlane, Zephyrus, President and Chief Executive Officer.
Speaker Change: <unk> Clifton, our Chief Financial Officer, and Josh Schaffer, our Chief commercial officer, and EVP of business development.
Speaker Change: Our Chief Medical Officer, Adrian portal will also be available for today's question and answer session. Now, it's my pleasure to hand, the call over to Neil.
Neil McFarlane: Thank you Nicole and thank you for joining us this afternoon.
Neil McFarlane: As Nicole mentioned today, we will provide our fourth quarter and fiscal year 2024 financial results and share progress on the execution of our priorities for 2025.
Neil McFarlane: Our priorities are guided by Zebra strategic plan.
Neil McFarlane: During our third quarter call and categorized under four actionable pillars commercial excellence pipeline and innovation.
Neil McFarlane: And culture and corporate Foundation.
Neil McFarlane: 2024 was a transformational year for zebra and bringing my play for people living with Niemann pick disease type C. N. P. C was the crowning achievement.
Neil McFarlane: In the past year, we became a commercial stage company I've had the opportunity to positively impact the lives of people living with rare diseases.
Neil McFarlane: Approval of my pipe for last year was an outcome of our longstanding collaboration with the NPC community and included contributions from more than 270, Niemann pick disease type C patients who participate in globally and our pivotal studies open label extension studies and expanded access programs.
Neil McFarlane: The early days of the my playful launch have exceeded our expectations in the fourth quarter. We received 109 patient enrollment forms, which we believe reflected the anticipation and unmet need in the MPC community.
Neil McFarlane: Additionally by the end of Q4, all active U S. EAP participants submitted an enrollment form and our entire organization has been activated to ensure smooth.
Neil McFarlane: In addition to might play for commercial products.
Neil McFarlane: As previously reported we anticipate that the U S. EAP will close by the end of Q2.
Neil McFarlane: Going forward our goal is to expand availability of my play for outside of the U S to provide access to as many people living with MPC as possible.
Neil McFarlane: With this goal in mind, we are progressing our regulatory submissions with the focus on submitting a marketing authorization application in Europe. During the second half of 2025, where we estimate 1100 people are living with MPC.
Neil McFarlane: Additionally, we will maintain our global expanded access program that currently supports 70 to 80 MPC patients and we look forward to providing updates in the coming quarters as we seek to expand access to my playbook.
Neil McFarlane: We're leveraging our infrastructure for both urea cycle disorders, or your Cds and M. P C leading to efficiencies that drive commercial excellence.
Neil McFarlane: The launch of our approval in early 2024th facilitated key learnings as we built our commercial capabilities.
Neil McFarlane: During the fourth quarter, we received for all proven enrollments as announced in our Q3 call. We refined our strategy to reach the adult onset population for whom will prove us portability and ease of administration may provide a benefit with a focus on patients receiving payer pushback.
Neil McFarlane: So we'll provide more details related to the performance of our commercial products later in the call.
Neil McFarlane: Moving to pipeline and innovation, which refers to our ambition to develop products that address significant unmet need.
Neil McFarlane: Deliberate wall is our phase III investigational clinical candidate for the treatment of vascular Eilers, Danlos syndrome, or <unk>, which is an inherited connective tissue disorder caused by the call three a one gene mutation that can result in spontaneous arterial aneurysms and hollow, Oregon ruptures.
Neil McFarlane: There are currently no treatments approved in the U S where there are approximately 7500 people living with this disease and the prevailing treatment paradigm is only reactive surgical interventions.
Neil McFarlane: Although <unk> is not approved for beds in any country. It is utilized as a standard of care in several European countries.
Neil McFarlane: In Q4, we enrolled eight patients in our phase III discover trial, bringing the total number of enrolled patients to 27.
Neil McFarlane: As an event driven trial the rate of participant enrollment is integral to the program's success.
Neil McFarlane: As a result, we are investing in tactics to accelerate enrollment among genetically confirmed patients with providers and clinics that treat call three eight <unk> positive patients.
Neil McFarlane: In addition, we have support from the Vets Foundation professional advisory board on our path forward.
Neil McFarlane: Moving on to <unk> 77.
Neil McFarlane: As outlined on our Q3 call and following a successful end of phase II meeting with the FDA, we are exploring strategic alternatives to advance clinical development and future commercialization.
Neil McFarlane: We are focused on maximizing the value of this phase III ready asset that has regulatory feedback for the potential development in both idiopathic hypersomnia and narcolepsy patients.
Neil McFarlane: In 2024, we completed a comprehensive review of our IP portfolio to identify areas for optimization.
Neil McFarlane: This review led to a decision to rationalize our in house discovery efforts and outsource those activities to focus our investments where we have the greatest potential for impact.
Neil McFarlane: Execute our strategic plan and deliver on our key priorities, it's paramount that we develop a high performing culture and attract and retain talent.
Neil McFarlane: In the second quarter of 2024, we expanded our executive leadership team with the appointment of Rahsaan Thompson as Chief Legal Officer, Secretary and compliance Officer, and Alison Peters as Chief people Officer.
Neil McFarlane: Additionally, during the fourth quarter, we consolidated our development and scientific functions under Adrian <unk>, Our Chief Medical Officer. We believe these changes optimize zebra for success with continuity of leadership and the appropriate span of control to achieve our objectives.
Neil McFarlane: An important element of executing against our strategy is to build and maintain our position of financial strength and disciplined capital allocation that we refer to as our corporate foundation.
Neil McFarlane: This underpins all other pillars and allows us to responsibly invest in our long term transformation.
Neil McFarlane: In 2024th we refinanced existing debt with a new credit facility and completed a modest secondary offering which provided financial stability.
Neil McFarlane: Recently, we entered into an agreement to monetize our priority review voucher for $150 million, which upon closing we will provide non dilutive capital to support our future growth.
Neil McFarlane: Building on the momentum from 2024, we are pleased to start 2025 from a position of strength across our four strategic pillars execute on the tremendous opportunity we have to serve people living with rare diseases.
Neil McFarlane: I'll now turn the call over to Josh who will provide an update on our commercial products.
Josh: Thank you Neil.
Josh: 2024 was a year of significant milestones for zebra and the rare disease community.
Josh: In particular, the Fda's approval of my place Mark the first product in the U S for the treatment of MPC.
Josh: At that time, we immediately began receiving patient enrollment forms and initiated the benefits verification process.
Josh: During the fourth quarter product was available in the last five weeks of the year.
Josh: As a reminder, today and on future calls while discussing our commercial products, we will provide patient enrollments the percent of covered lives and net revenue for the quarter.
Neil McFarlane: As Neil noted the launch of <unk> has gone exceptionally well and carry significant momentum into 2025.
Neil McFarlane: From the time of approval through December 31, we received 109 total prescription enrollment forms.
Neil McFarlane: And as at the end of the year, we had enrolled all of the active U S EAP patients.
Neil McFarlane: As a reminder, an enrollment is a prescription submitted to our specialty pharmacy initiating the benefits investigation process, leading to a decision on reimbursement and paid dispense which is a 30 day supply it might play for us.
Neil McFarlane: In the fourth quarter early enrollees may have received both an initial dispense and the first refill depending on the time of their enrollment.
Neil McFarlane: We estimate that there are approximately 900 people with MPC in the U S of whom an estimated 300 to 350 <unk> had been diagnosed.
Neil McFarlane: But there is some perspective, our fourth quarter performance resulted in approximately one third of diagnosed patients submitting an enrollment to receive my place.
Neil McFarlane: NPC is this neuro degenerative progressive and fatal lysosomal storage disorder caused by lipid buildup, leading to cell death, and ultimately, Oregon dysfunction in the spleen liver and brain.
Neil McFarlane: Cause symptom presentation and age of onset or heterogeneous disease progression is primarily assessed using the only clinically validated endpoint. The niemann pick disease type C clinical severity score, including measurements of ambulation fine motor speech and swallow abilities.
Neil McFarlane: The data included in our label shows might play for in combination with <unk> halted disease progression through 12 months demonstrated by more than a two point improvement in patients receiving this combination compared to those receiving <unk> sat alone.
Neil McFarlane: We're only a one point improvement is needed to demonstrate a clinically meaningful difference.
Neil McFarlane: In a disease like NPC.
Neil McFarlane: Access to data covering at 12 months duration is critical to establishing treatments as disease modifying.
Neil McFarlane: Beyond the pivotal trial might Playfirst safety profile and long term treatment effect had been evaluated in an open label extension study and through multiple center eap's with some patients having as many as five to seven years of experience on therapy.
Neil McFarlane: We are pleased with my places data and its indication for use in combination with Mega staff and our market intelligence suggests that health care providers prefer multi therapeutic approach to treating this heterogeneous disease.
Neil McFarlane: Based on early market experience and feedback we believe providers will use a disease modifying treatment such as my place plus megawatts that as the cornerstone of therapy, and then potentially at therapies or supplements to provide symptomatic treatment if needed.
Neil McFarlane: Many commercial plans have not yet formalized reimbursement coverage.
Neil McFarlane: So it is too early in the launch to provide a meaningful market access and reimbursement update in terms of the percentage of covered lives.
Neil McFarlane: However, our team is actively engaged with payers and we have been addressing coverage hurdles to gain reimbursement through either direct formulary coverage via the medical exception process thus far.
Neil McFarlane: We will continue meeting with payers and presenting clinical data to support my places us as the cornerstone of therapy for MPC and we will report progress in future calls.
Neil McFarlane: As we look forward to 2025, our focus will be to grow enrollments of currently diagnosed and untreated patients.
Neil McFarlane: We recognize the impact of prompt diagnosis and treatment to halt the progression of disease for people living with MPC and have invested in educational resources and tactics drive patient identification and early intervention.
Neil McFarlane: As an example, we commenced a media campaign working with local outlets and key opinion leaders to educate about early signs and symptoms of MPC.
Neil McFarlane: This program led to the identification of three new patients who had not been previously treated.
Neil McFarlane: We are also using advanced analytics to understand where recently diagnosed MPC patients were treated and to ensure new patients have an opportunity to receive treatment.
Neil McFarlane: Our rare disease sales and reimbursement specialists are actively engaged in raising awareness of MPC diagnosis and available treatments with my play for us.
Neil McFarlane: Our efforts to increase disease awareness and are multifaceted and on February 28 of this year timed with rare disease day, we launched a disease state awareness campaign entitled learn NPC read between the signs to educate health care professionals unfamiliar with MPC.
To support the identification and diagnosis of people living with MPC.
Neil McFarlane: In addition, we entered into an agreement with a leading diagnostic company to provide genetic testing options for individuals with suspected lysosomal storage disorders.
Neil McFarlane: Including MPC.
Neil McFarlane: We believe this program will lead to the identification of new patients and will allow us to support earlier diagnosis and treatment.
Neil McFarlane: Our sales and reimbursement teams regularly meet with prescribers to provide education on the strength of <unk> data and to support the reimbursement processes.
Neil McFarlane: And currently under the direction of Adrian <unk>, our Chief Medical Officer, our medical liaisons are engaging with key opinion leaders and patient advocacy groups to build the body of evidence demonstrating the need to treat patients early to delay progression and to position <unk> as the cornerstone of treatment.
Neil McFarlane: Additionally, our medical affairs team is prioritizing our publication strategy.
Neil McFarlane: Further drive awareness of the need to treat MPC patients for example, during the 20 <unk> annual World Symposium.
Neil McFarlane: That represented eight posters, including new data assessing the safety and Tolerability of my place in MPC patients six months to 24 months as evaluated and a pediatric sub study of the pivotal phase III trial.
Neil McFarlane: In conclusion for my place we're encouraged by the initial signs of the launch and are excited to provide future updates on our progress in reaching patients who can benefit the most.
Neil McFarlane: Now, let's turn to approval, our commercial product for the treatment of certain ucd's.
Neil McFarlane: Do you see these are a group of rare inherited metabolic disorders caused by a defect in one of the six enzymes or two transporters in the urea cycle, leading to an accumulation of ammonia.
Neil McFarlane: Elevated ammonia or hyper ammonia can be toxic leading to neuro cognitive damage or even death.
Neil McFarlane: I'll prove as a nitrogen scavenger that removes excess ammonia, thus reducing harmful accumulation.
Speaker Change: The approval launch has progressed slower than we would like for this differentiated product.
Speaker Change: During the fourth quarter, we saw new enrollments increased to four as compared to three in the third quarter and our market access for approval remains consistent at 76% of covered lives.
Speaker Change: As ever launched a prove a little over a year ago into a mature UCD market.
Speaker Change: At that time, we were able to build initial demand with clinicians and patients looking for alternative therapies that can improve adherence and better control ammonia levels.
However, limited patient pull through and reimbursement support experienced early in the launch necessitated a change to improve the patient experience and we transitioned to a new specialty pharmacy.
Speaker Change: We also refined our strategy to target specific patient segments, who will receive the greatest benefit from <unk> and who may face fewer reimbursement hurdles.
Speaker Change: We believe that the adult female OTC deficient carrier population as well as those UCD patients who may seek additional lifestyle independents would receive the greatest benefit from the ammonia control on the go that all prove it provides.
Speaker Change: Another resource we have added includes field reimbursement managers, who work with physician teams to assist with reimbursement challenges.
Speaker Change: We've also identified payer plans that have moved other therapies to the exclusion list and are working with payers to provide a smoother reimbursement process for all prove out.
Speaker Change: These efforts are in early stage and we look forward to reporting our progress in future calls.
Speaker Change: Finally, we are proud to highlight our sponsorship of the national urea cycle disorders Foundation.
Speaker Change: Pneumonia campaign to raise awareness of the critical importance of recognizing the signs and symptoms of hyper ammonia anemia, and the need to treat early and persistently.
Speaker Change: Patients remain at the center of what we do in supporting the rare disease community through patient advocacy organizations like end use C. D. F is one of our highest priorities.
Speaker Change: I'll now turn the call over to Lynn Duane to discuss the fourth quarter and fiscal year 2024 financial results.
Lynn Duane: Thank you, Josh and good afternoon, everyone.
Lynn Duane: In addition to the financial details included in todays call. We encourage you to refer to <unk> annual report on Form 10-K for more detailed information, which we intend to file within the next few days.
Lynn Duane: In the fourth quarter of 2024, we reported net revenue of $12 million, which includes $10 1 million and <unk> revenue 0.1, and approve our revenue.
Lynn Duane: 1.1, and net reimbursements from the French EAP for Aramark, Limo, and 0.7 of royalties and other reimbursements under the <unk> license.
Lynn Duane: This was a record level of quarterly product revenue for zebra.
Lynn Duane: For our commercial products might play for Endo prove up we recognize revenue when shipments are received by the specialty pharmacy.
Lynn Duane: Or my FIFO Q4 was a partial period and revenue included initial patient dispenses refills for some early enrollees and target supply levels at the specialty pharmacy.
Lynn Duane: French EAP reimbursements were reduced during Q4 due to a true up in program access fees and we continue to expect net reimbursements will continue to be approximately $2 one per quarter.
Lynn Duane: Our operating expenses for the fourth quarter were $24 $5 million, which includes noncash stock compensation expense of four.
Lynn Duane: Of which $2 1 million was accelerated vesting expense related to severance arrangements and severance expenses of $1 six recognized within R&D expenses.
Lynn Duane: R&D expenses for Q4, 2024 were $8 4 million, which was a decrease of $3 million compared to Q4 2023, due primarily to a decrease in third party costs. Upon completion of the K P. 10, 77 phase two trial offset in part by an increase.
Lynn Duane: And personnel related costs.
Lynn Duane: SG&A expenses were $16 one for Q4, 2024, which was an increase of one four.
Lynn Duane: Compared to $14 seven for Q4 2023 do.
Lynn Duane: Due primarily to an increase in personnel related costs as our full team was in place and actively engaged in commercial launch activities for the entire quarter.
Lynn Duane: Net loss for the fourth quarter 2024, it was $35 7 million or <unk> 67.
Lynn Duane: Per basic and diluted share.
Lynn Duane: Compared to $19 6 million or <unk> 51 per basic and diluted share for the same quarter a year ago.
Lynn Duane: For our full year 2024 results net revenue was $23 $6 million, which.
Lynn Duane: <unk> 10, one of my plan for net revenue 0.1 of all prove a net revenue 9.1, and net reimbursements from the French EAP for Aramark, Hummel, and $4, three and royalties and other reimbursements under the <unk> license agreement.
Lynn Duane: Our full year operating expenses were $97 million, which includes $14 nine of noncash stock compensation expense.
Lynn Duane: R&D expenses were $42 1 million, which was an increase of $2 three compared to full year 2023, due to an increase of $6 three and personnel related costs offset by a decrease of three six and third party costs, primarily as a result of the completion of the K P 10 77.
Lynn Duane: Phase III trial.
Lynn Duane: SG&A expenses were $54 $9 million for 2024 compared to 34 three for 2023 and this reflects the commercial team fully in place and actively engaged in our launch activities.
Lynn Duane: For fiscal year, 2024, net loss was $105 $5 million or $2 28 per basic and diluted share.
Lynn Duane: Compared to net loss of $46 million or $1 30 per basic and diluted share for 2023.
Lynn Duane: As of December 31, 2020 for total cash cash equivalents and investments were $75 $5 million, which was a decrease of $20 million compared to the end of Q3 2024.
Lynn Duane: Total long term debt was approximately $60 million.
Lynn Duane: And as announced on February 27th we have entered into an agreement to sell the priority review voucher, we received with the my place of approval for $150 million.
Lynn Duane: Upon closing, we expect to receive net proceeds of approximately $148 $3 million net of fees and our pro forma balance of cash cash equivalents and investments will be approximately $223 $8 million.
Lynn Duane: This non dilutive capital adds a significant strength to execute on our strategic priorities with our immediate focus on executing the commercial launches of <unk> and <unk> <unk>.
Lynn Duane: Investments in <unk> and our other development programs and building our corporate foundation, while we earned the right to make disciplined growth investments in the future.
Lynn Duane: Based on our execution to date, our Form 10-K will not include a going concern disclosure.
And our current operating forecast combined with existing resources extends our cash runway into 2029.
Lynn Duane: Our cash run rate guidance does not include the potential proceeds from the sale of the <unk>, but it does include anticipated net revenue from <unk> and <unk> sales.
Lynn Duane: Net reimbursements from the French EAP for are marketable.
Lynn Duane: Royalties under the <unk> license agreement and continued investments into our development pipeline programs.
Lynn Duane: Our financial results for 2024 reflect the solid execution of our strategic plan and demonstrates our commitment to our mission to serve patients and generate meaningful value for shareholders. We.
Lynn Duane: We are pleased with the opportunities we have in 2025 and beyond to drive value creation through disciplined investments, where we can win.
Lynn Duane: Now, let's open the call to questions operator.
Speaker Change: Thank you at this time, if you wish to ask a question. Please press star one on your telephone keypad you may refer to are all yourself from the queue by pressing star too while we wait for that <unk> to build will take our first question from Kristen <unk> with Cantor. Please go ahead.
Hi, everyone. Congrats on a really strong star out of the gate, especially around the holidays I think speak to how much. These patients really wanted that therapy. So congratulations a few questions from me I think this is the first time that you've provided us more specific guidance.
Speaker Change: On the European filing timelines. So can you give us a sense of the last dialogue that you've had with the agency there in the plan and then when we think about the European market, which is more mature partially due to the fact that <unk> been there for over a decade, how do you think that drug <unk>.
Speaker Change: Available and used for patients and how that identified more patients over time can translate to some of the efforts in the U S. Now that you have the approval there.
Speaker Change: Thanks, Chris and thanks for the comments, it's been a great.
Speaker Change: 2024, and transformational for sure, but as we mentioned in the.
Speaker Change: The remarks, the prepared remarks.
Speaker Change: In my play for the patients with Niemann pick C is a its definitely a crowning achievement, let me quickly touch base on a little bit of the market dynamics for Europe, and then I'll ask Adrian to talk a little bit about briefly about our regulatory interactions.
Speaker Change: In Europe, we see about a 100 patients in Europe based on our prevalence number and <unk> has been approved there for well over a decade. So so are our current label in the U S. And we are still working through what this filing would look like for our MAA in Europe as you know have my pipe.
Speaker Change: And megawatts stat in combination.
Speaker Change: In order to be able to halt the progression of the disease versus <unk> stat alone, having an approximate two point progression over 12 months based on our clinical study. So based on the fact that that there has been <unk> in the market. We believe the European market is more mature there are more patients diagnosed and treated because of decades worth of work that.
Speaker Change: It has been done and now bringing a product that has the potential in Europe under the MAA filing two actually.
Speaker Change: Halt the progression of the disease based on the label that we have and the data we had in our phase III program. We feel like this is a great opportunity to go after in Europe in regards to the European filing.
Speaker Change: Probably not ready to talk too much about all of the intricacies of the European filing but to be able to state that 2020 by the second half of 2025, we believe that we're going to be able to file Adrian will talk a little bit about our last interactions. Yes. It allows interaction with EMA obviously happened.
Speaker Change: Via offers I mean about four years ago.
Speaker Change: Through the application based upon the complete response letter that we received from the FDA and some of the comments that were received.
Speaker Change: From the EMA.
Speaker Change: In regards to defining never had in Europe, and obviously in a much better position with long term data available.
Speaker Change: Our currently consulting with couple of consultancy group to our <unk>.
Speaker Change: To specifically address some of the ones that they had.
Speaker Change: During the original filing and as said we are planning to file in the second half of this year and we're confident that.
Speaker Change: The filing will be accepted by the EMA.
Speaker Change: Okay. Thanks, and then thanks for providing the cash runway guidance, which I understand excludes the PRD for now but can you just maybe high level give us a sense of like how conservative are you being with launch projections projections into this does this include.
Speaker Change: <unk>, if you do get an approval in Europe, adding that territory. There just any high level would be really helpful. Thank you again.
Speaker Change: Yes of course, yes, we did not include the <unk> proceeds to yourself and I would just say we have been conservative as we looked at that cash runway. We have to continue executing and continue into 'twenty five with the plans that Josh and the team have got in place and so that's definitely where our focus is but yes. It is.
Speaker Change: Safe to call that a conservative appropriate conservative estimate.
Speaker Change: Thanks, everyone.
Speaker Change: Thank you and we'll next go to Jason Jason Butler with citizens JMP. Please go ahead.
Jason Butler: Alright, Thanks for taking my questions and let me add my congrats on the Florida as oil price thoughts on the buy side for launch.
Jason Butler: Can you maybe give us any more quantitative color on the <unk> sales during the quarter in terms of the supply amounts to 10.
Jason Butler: The specialty pharmacies or what proportion of patients, Florida Refills and then my second question is how should we think about the <unk>.
Jason Butler: Launch from here in terms of net patient adds you've obviously successfully converted the EAP patients, but should we expect consistent steady net patient adds or could it be more volatile from month to month. Thank you.
Speaker Change: Yes, Jason I'm going to see if I can parse that to let duane to be able to answer the original questions around.
Duane: Inventory and we're not going to get into specifics, but he'll be able to answer that and then I will hand, it off to Josh to be able to talk a little bit about that.
This bolus of patients that we've seen and what the future may look like.
Duane: Yes, so with my plasma revenue again, we reported $10 1 million in Q4.
Duane: That would have included.
Duane: Product to handle the <unk>.
Duane: Initial patient dispenses. Some early enrollees may have received a second refill late in December and then we did allow for an appropriate level of stock to continue to service patients. It's actually at the target level that we would expect to maintain at this point.
Duane: And so Josh alternate view, yes, Jason your question about.
Duane: New patients going forward I think it's really important for us to kind of reflect on.
Duane: How quickly we were able to get to 109 patients and how quickly we were able specifically to convert those patients who are in our expanded access program.
Duane: To enroll incident might play phone. So we had all of those patients plus some patients who had previously not been exposed to my place.
Duane: All within the fourth quarter and so we were really pleased with how quickly we were able to do that I think it speaks to the demand that there is in the marketplace. I think it also speaks to the execution of the team and.
Duane: And as we look forward into 2025, our focus now is really to be able to find those patients who have been diagnosed that arent currently treated as well as those patients who have not yet been diagnosed and so that's really going to be the focus as we continue into this year.
Duane: Alright, thank you.
Speaker Change: Next we will take our next question from Sumit Kulkarni with Canaccord. Please go ahead.
Sumit Kulkarni: Great to see all the progress and thanks for taking our questions. My first one is a bit of a follow up on the last question.
Sumit Kulkarni: In terms of your sequential Delta in patient enrollment forms for my best buy that was 19.
Sumit Kulkarni: What are the key variables that we should keep in mind that might influence the trajectory as we look to the next quarter versus this one.
Sumit Kulkarni: So youre absolutely right we went from.
Sumit Kulkarni: Excuse me <unk>.
Sumit Kulkarni: <unk> 90 at the end of October to 109 at the end of December.
Sumit Kulkarni: This really reflects many of these patients were really sort of queued up ready to go from our expanded access program.
Sumit Kulkarni: As we move forward into this quarter as I just mentioned, we're really focused on trying to.
Sumit Kulkarni: Expand beyond those expanded access program sites.
Sumit Kulkarni: To where we know that these patients are being treated have been diagnosed.
Sumit Kulkarni: We're going to continue to to to deploy our sales teams into those offices.
Sumit Kulkarni: As we continue to do on each of these quarters will be providing enrollment numbers or percent of covered lives and net revenue and you.
Sumit Kulkarni: You can expect that we'll continue to do that as we as we move forward into subsequent quarters.
Neil: It's Neil here I think it's important.
Neil: As we try to answer this question with a follow up also to the previous.
Neil: Analyst questions as well.
Neil: When we talked about the transition of our EAP patients traditionally we would see that over about a 12 month period.
Neil: And the team based on all of the work they did prior to launch.
Neil: <unk> launch and then a combination I've been able to exceed every expectation we had internally on the conversion of those EAP patients not over a 12 month period, but actually over a one quarter period.
Neil: And I think that is a true testament to as we mentioned previously too.
Neil: The built up demand.
Neil: And as well as the unmet need in this patient population. So as we go through this trajectory and it has been a a rocket ship trajectory, we expect that over the next phase of launch for us to be able to then take this this momentum into Q1 into Q2 executing on all of the tactics and the <unk>.
Speaker Change: The approach that Josh.
Neil: In his prepared remarks so.
Speaker Change: I think there's a there's a.
An important perspective here for us to be able to show that this has been an outstanding performance on the conversion and on the launch trajectory. We know we're going to take this into executing phase II.
Speaker Change: Point, well made been taken.
Speaker Change: So on the 300 to 350 or so diagnosed and treated patients with MPC roughly how many centers.
Speaker Change: Centers of excellence versus not.
Speaker Change: Yes.
Speaker Change: Many of these patients have some diagnosis that takes place at these centers of excellence, but keep in mind.
Speaker Change: They had previously been no treatments approved for Niemann pick. So many of these patients would be diagnosed and then go back to their local neurologists to continue being monitored we're finding now that those patients are coming back to the centers of excellence as they are made aware of my Piper.
Speaker Change: Being approved and so the vast majority of them have some connection either being treated at the centers of excellence or some sort of a referral to the centers of excellence.
Speaker Change: And last one before I hop into the queue back.
Speaker Change: Roughly how many quarters are you, giving yourselves that the new patient start getting started to see a potential inflection.
Speaker Change: Yes, we haven't really.
Speaker Change: But any any timeframe on that we know that all proven can provide benefit to patients we have revised our strategy to be able to.
Speaker Change: To bring that to specific patients who will benefit from the portability and the personalized dose and these are the adult patients and in particular, those where theres some reimbursement challenges. So we're continuing to.
Speaker Change: To monitor this it's early in kind of.
Speaker Change: Assessing the impact of that but we also know that there is great synergy within our sales team within our broader commercial team and having both of these products. It's opening a lot of doors for my Piper, it's allowing us to foster relationships across the entire commercial spectrum.
Speaker Change: We're watching this very closely.
Speaker Change: We're allowing some time to see the impact of this change in our strategy.
Speaker Change: <unk>.
Speaker Change: We will take our next question from Oren <unk> with H C. Wainwright. Please go ahead.
Speaker Change: Thanks.
Oren: My pleasure.
Can you just characterize that initial bolus of 90 patient referrals that came in really fast in that first month.
Oren: Can you get the lion's share of MPC patients in a concentrated group of centers and so now youre more gradually expanding to a broader set of treaters and centers to penetrate those.
Oren: Sure.
Oren: Was that 90, including I guess just.
Oren: A fraction of the patients at a broader.
Oren: Set of sites. If you understand my question just trying to figure out like what.
Oren: But the heavy lifting is here going forward and also.
Oren: I think on the last call you had mentioned that 30% of those initial 90 had already been quote unquote approved which.
Oren: Which is really fast in the first month, either I assume via some formal coverage on more likely medical attention.
Oren: Do you have any updates on those percentages for the initial wave continuing.
Oren: Continuing approval rates or the cumulative accrual rates for the entire 109 to December and I did thanks.
Oren: Yeah. So I'll take your question about the 90, so as we reported.
Oren: In our last call of those 90 69 of them were from the expanded access program and 21 were from other other sites that were naive to <unk>.
My wife or had not previously received <unk>.
Oren: Any of those patients were still treated within one of those centers of excellence.
Oren: As we move forward now to 109, we've gotten the remainder of those EAP patients. We are also.
Oren: Expanded beyond the centers of excellence to find other prescribers and patients and we're going to continue to do that as we move into this next phase of launch.
Oren: Your second question was around the percent of patients that had received authorization for payment and at the time of our last call. It was 30%.
Oren: We're continuing to see improvement in that end.
Oren: And what we're seeing is that.
Oren: Many commercial payers have not yet made decisions around policy.
Oren: But the vast majority of our patients are able to get reimbursed either through direct formulary or through a medical exception process and importantly, our team is out in front of these payers talking about the clinical benefits of my wife.
Oren: Sharing with them the new data that's been generated since the approval and so we expect that's going to continue to improve as we are.
Speaker Change: It moved down the launch.
Oren: And as you have these.
Speaker Change: <unk> conversations.
Speaker Change: Can you just maybe give us more color on.
Speaker Change: How that's going if there's any pushback at the obviously ultra rare so I would assume that's somewhat limited, but obviously, we're talking about both too expensive drugs in combination. So just maybe a little more color. There and are you able to give us a sense of what your I guess average net.
Speaker Change: Dollars per patient per year expectation is going forward.
Speaker Change: So in terms of the conversations that we're having with payers right now.
Speaker Change: We are really getting in there and we're talking about the benefits that might play for offers to these patients where we're talking about the the halting of progression of disease. After 12 months.
Speaker Change: We are the only product that has been approved with based on a clinically meaningful endpoints and the $2 one.
Speaker Change: <unk> in in that endpoint, so thats really where the conversations are taking us and we're continuing to drive that message home and as I mentioned.
Speaker Change: Most of these patients are getting covered either through formulary or through some sort of medical exception process and thats really continue and so that's really the tone.
Speaker Change: A lot of additional doors are being opened for continued clinical conversations.
Speaker Change: As it relates to the.
Lynn Duane: Net pricing I'm going to turn that over to Duane here.
Speaker Change: Or and I would just say, we don't generally discuss or comment too much on the gross to net details but at this point in time I would say our strategy is still underway in terms of how we get the data story in front of payers, it's not clear yet if we need to even be that aggressive in that concept.
Speaker Change: It's early days, there, but I expect the gross to net is going to be.
Speaker Change:
Speaker Change: We're going to have.
Speaker Change: Oh I'm sorry, gross to net is going to be in a solid place and I don't expect we'll be very aggressive there at this point.
Speaker Change: Okay. Thank you so much.
Speaker Change: Our next question comes from Sami Corwin with William Blair. Please go ahead.
Sami Corwin: Hi, there congrats on the great quarter and thank you for taking our question.
Speaker Change: I guess I was curious.
Sami Corwin: Average time, Youre kind of thing from the us.
Sami Corwin: Starting with <unk> approval of reimbursement and then as you're kind of thinking about expanding your targeted population.
Sami Corwin: And going beyond the 300 to 350.
Sami Corwin: We are currently seeking treatment.
Sami Corwin: What extent do you think you can further expand and penetrate into the estimated 900 pipeline population. Thank you.
Sami Corwin: Hey, Sami, it's Neal I'm going to I'm going to kick. This one off because I think we're really pleased so far with what we've seen not just in those patients who are diagnosed but in our in our prepared remarks, we talked about the fact that one of the areas. We've been investing in has been media.
Sami Corwin: And those three patients that we've talked about we're actually patients who were.
Sami Corwin: Diagnosed but not treated and this gets back down to this expanding the market population.
Sami Corwin: And we believe that as we continue to get a treatment in the market and multiple treatments in the market for that matter.
Sami Corwin: Physicians that actually have MPC or have other diseases that will start to think about MPC in their diagnostic cascade. In these rare diseases that will really allow us to be able to then increase that number from the 300 to 350 in the 900.
Sami Corwin: And then if Europe as a marketplace of the 1100 patients from a prevalence perspective, and the <unk> stack growth that's happened over a decade, plus we think it's a great information its great information for us to inform what our market possibility is in the U S. So jocelyn will talk a little bit about <unk>.
Speaker Change: Pullman tends to reimbursement and those things sure.
Speaker Change: And I was just going to add to this that as we mentioned in our prepared remarks.
Speaker Change: <unk> employed a number of other tactics to really identify these patients including some.
Speaker Change: Collaboration with that.
That genetic testing.
Speaker Change: <unk> around the niemann pick all of which is going to lead to some market expansion in terms of the average time from enrollment to reimbursement.
Speaker Change: It's too early in the launch to be able to really give you specifics on that but I would mention again that in the fourth quarter in those five weeks from the time that we shipped drug until the end of the year. We did have a number of patients who not only receive their first fill but also refill.
Speaker Change: In that period of time, so I think that really speaks to how quickly our team was able to get some of these patients reimbursed and reauthorized.
Speaker Change: We anticipate that those.
It's just going to continue as as we work our way through those are those patients who are pending approval now.
Speaker Change: Great. That's very helpful and a quick follow up on how have you had any patients that have submitted an enrollment one bad debt ultimately denied and do you have any clarity as to what the rationale behind I mean, I'll take a mile denial list.
Speaker Change: Yes. So of course, you know in any rare disease. There are some initial denials and we work through those with patients to make sure that we can help them navigate in.
Speaker Change: And worked through those those denials through appeals and other.
Speaker Change: Parts of that.
Speaker Change: Pathway through the medical exception pathway.
Speaker Change: There we've only had a couple of patients who have who have been denied but again, we continue to support those patients we're continuing to to help them work through the reimbursement challenges in any patient who is experiencing those challenges we have a very robust patient services plan and patient assistance plants, so that no pay.
Speaker Change: <unk>.
Speaker Change: Woods go without drug.
Speaker Change: Got it thank you.
Speaker Change: Perfect and as a reminder, ladies and gentlemen, if you would like to ask a question. It is star one on your telephone keypad. We'll next go to Eddie Hickman with Goldman <unk> Securities. Please go ahead.
Eddie Hickman: Hi, Good afternoon, I'll add my congratulations on a really nice quarter.
Eddie Hickman: What can you tell us about how we should model discontinuation rate. So far and then going forward do you expect the rate to meaningfully change as we get further outside of the EAP and then of the sort of current bolus of patients like how many of them do you expect to retain sort of in the long term. Thanks.
Eddie Hickman: Yes.
Eddie Hickman: Thanks for that question I think it's just it's way too early in the launch to be able to to address or answer that question yes.
Yes, Eddie maybe I'll give you a comp though that you can actually think about it.
Eddie Hickman: We look at our EAP data and we think about the patients and the continuity we have patients that remain in our EAP five.
Eddie Hickman: Five years and patients went through OLED to EAP now on to commercial product 567 years, you heard that also in the and the open public Forum during our Advisory Committee. So what we've seen and continue to see also in our European expanded access program is a really high level.
Eddie Hickman: Continuity of care once patients are on <unk> that again.
Just a few months in where we're reporting on five weeks of.
Patient shipments and in Q4, so it's going to be a little early to talk about discontinuation rates, but if history repeats itself. Once patients are on the side effect profile that my pie five megawatts that have we see longevity.
Eddie Hickman: Great I appreciate it I appreciate all that color. Thanks, guys.
Speaker Change: Thank you I'd now like to turn the call back over to Neil Mcfarlane for any closing remarks. Please.
Neil McFarlane: Thank you operator, thanks for joining us today to review what was a transformational 2024, we're pleased to start 2025 from a position of strength and look forward to executing on our mission to serve people living with rare diseases have a great day.
Speaker Change: Thank you and this does conclude today's program. We thank you for your participation you may disconnect at any time.
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