Q3 2023 Zevra Therapeutics Inc Earnings Call
Speaker 1: Good morning, everyone. Thank you for joining these ever therapeutic third quarter 2023 corporate updates and financial results call.
Good morning, everyone. Thank you for joining these ever therapeutics third quarter 2023, corporate updates and financial results call.
Speaker 1: Today's call is being recorded and will be made available on the company's website following the conclusion of the call.
Today's call is being recorded and will be made available on the company's website. Following the conclusion of the call.
Speaker 1: With that, I will now turn the call over to Nicole Oshner, Vice President of Investor Relations and Corporate Communications for Zephyra Therapeutics. Please go ahead.
With that I will now turn the call over to Nicole Ocean Air Vice President of Investor Relations and corporate communications for separate Therapeutics. Please go ahead.
Speaker 2: Good morning and thank you for joining us today to review the ever therapeutics progress in the third quarter of 2023, outlining our clinical advances, operational achievements, and financial results.
Good morning, and thank you for joining us today to review its ever Therapeutics progress in the third quarter of 2023 outlining our clinical advances operational achievements and financial results.
Speaker 2: Before we get started, let me take a moment to provide some important information. First, I encourage you to access the press release, which was published this morning, and is available in the Investor section of the ZEVER website.
Before we get started let me take a moment to provide some important information first I encourage you to access the press release, which was published this morning and is available in the investors section does ever website.
Speaker 2: while we will not be using slides on today's call and updated corporate presentation will be made available on our website later today.
Well, we will not be using slides on todays call an updated corporate presentation will be made available on our website later today.
Speaker 2: As we move forward, it's important to highlight that the company's discussions will include forward the team statement.
As we move forward, it's important to highlight that the company's discussion will include forward looking statements.
Speaker 2: Forward-looking statements are not promises or guarantees and are inherently subject to risks on certainties and other significant factors that may lead to actual results differing materially from the projections made. For a comprehensive understanding of these factors, please refer to the risk factors section in our most recent quarterly report on Form 10Q and our annual report on Form 10K.
Forward looking statements are not promises or guarantees and are inherently subject to risks uncertainties and other significant factors that may lead to actual results differ materially from the projections made.
For a comprehensive understanding of these factors. Please refer to the risk factors section in our most recent quarterly report on Form 10-Q, and our annual report on Form 10-K.
Speaker 2: I am pleased to welcome Zebra's Management Team members participating in today's call. I'm joined by Neil McFarlane, President and Chief Executive Officer, Ligwain Quiston, our Chief Financial Officer, Joshua Schaefer, our Chief Commercial Officer and EVP of Business Development, and Crystal Mico, our Chief Development Officer, with that, I'll turn the call over to Neil.
I am pleased to welcome <unk> management team members participating in today's call I'm joined by Niall Macfarlane, President and Chief Executive Officer.
<unk> Clifton, our Chief Financial Officer, Joshua Schaffer, our Chief commercial officer, and EVP of business development and Crystal Nickle, Our Chief development officer with that I'll turn the call over to Neil.
Speaker 3: Thank you Nicole, and thank you all for making the time to join us today.
Thank you Nicole and thank you all for making the time to join us today.
Speaker 3: It's truly an honor for me to have the opportunity to lead Zebra at this critical stage of our growth. And to build on the company's solid foundation, as we work towards achieving multiple near-term clinical and regulatory milestones, along with accelerating our rare disease, commercial capabilities, to bring therapies to patients.
Truly an honor for me to have the opportunity to lead Zephyr at this critical stage of our growth and to build on the company's solid foundation as we work towards achieving multiple near term clinical and regulatory milestones along with accelerating our rare disease commercial capabilities to bring therapies to patients.
Speaker 3: What I'd like to do this morning is to provide a brief update on my transition and a few corporate updates. Then I'll speak briefly on our key development programs and the acquisition of ASER. After that, I'll turn it over to Le Dwayne to provide the financial highlights and open the call for questions.
What I'd like to do this morning is to provide a brief update on my transition and a few corporate updates.
Speak briefly on our key development programs and the acquisition of Acer.
After that I'll turn it over to Lynn Duane to provide the financial highlights and open the call for questions.
Speaker 3: Since joining last month, I've been meeting with an actively listening to our stakehold.
Since joining last months I've been meeting with and actively listening to our stakeholders consistent with what I understood before joining the company zephyrus transformation from an organization built on prodrug platform technology into one intensely focused on delivering promising therapeutic candidates to people living with rare diseases.
Speaker 3: consistent with what I understood before joining the company. Zervers transformation from an organization built on pro-drug platform technology into one intensely focused on delivering promising therapeutic candidates to people living with rare diseases is well on its way.
It's well on its way.
Speaker 3: The mission of the company, with its deep focus on people living with rare diseases, is very much aligned with my own beliefs. And I've been impressed with the unwavering commitment of the team to build on the past to accelerate the future.
The mission of the company with a deep focus on people living with rare diseases is very much aligned with my own beliefs and I've been impressed with the unwavering commitment of the team to build on the path to accelerate the future.
Speaker 3: I would like to thank Crystal for her work as interim president and CEO . She kept our programs moving forward while leading the company during the transition.
I would like to thank crystal for her work as interim President and CEO. She kept our programs moving forward, while leading the company during the transition Chris.
Speaker 3: Crystal has now returned to her role as chief development officer and will continue to advance our clinical program.
Crystal has now returned to her role as Chief Development Officer, and will continue to advance our clinical programs.
Speaker 3: Joe Saluri also recently retired from the board. We thank him for his dedication and service to the company's growth and success.
<unk> also recently retired from the board we thank him for his dedication and service to the company's growth and success.
Speaker 3: I would also like to take the opportunity to thank our shareholders that have remained committed to Zebra on its journey to becoming a leading rare disease company. I'm now pleased to report that there is stability within the board and management team along with the clarity to execute our plan. Let me now start with our close.
I would also like to take the opportunity to thank our shareholders that have remained committed to as ever on its journey to becoming a leading rare disease company.
I'm now pleased to report that there is stability within the board and management team along with the clarity to execute our plan.
Let me now start with our clinical development updates.
Speaker 3: Aramoklamol is poised to be the first drug approved in the US for the treatment of Neiman Guillewen
A remarkable is poised to be the first drug approved in the U S for the treatment of Niemann pick disease type C or N P C.
Speaker 3: which is a rare genetic, progressive, and potentially fatal, neurologic disease, which currently has no approved therapy in the US, creating an urgent need for people who are awaiting the treatment off.
Which is a rare genetic progressive and potentially fatal neurologic disease, which currently has no approved therapy in the U S, creating an urgent need for people who are waiting the treatment option.
Speaker 3: Today, Arab Montlomall has been studied in more than 10 different clinical trials, evaluating its safety and efficacy across over 500 subjects.
To date, our remarkable has been studied in more than 10 different clinical trials evaluating safety and efficacy of crossover 500 subjects.
Speaker 3: Since we acquired the asset in 2022, we've had productive interactions with the FDA and have been steadily working to address their feedback raised in the complete response letter.
Since we acquired the asset in 2022, we've had productive interactions with the FDA and have been steadily working to address their feedback raised in the complete response letter.
Speaker 3: We are confident that each of the deficiencies raised are being addressed in the resubmission. Specifically, additional evidence is being provided to support the use of the NPC clinical severity scale, as well as the inclusion of an FDA preferred primary analysis.
We are confident that each of the deficiencies raised are being addressed and the resubmission specifically additional evidence is being provided to support the use of the MPC clinical severity scale as well as the inclusion of an SBA preferred primary analysis.
Speaker 3: New data will also be included in the resubmission from multiple non-clinical studies, natural history comparisons, and real world data generated from the ongoing early access programs in the US and the EU.
New data will also be included in the Resubmission from multiple non clinical studies natural history comparisons and our real world data generated from the ongoing early access programs in the U S and the EU.
Speaker 3: In addition, data from the four-year open label extension of the Phase 2, 3 clinical trial will be included in the submission.
In addition data from the four year open label extension of the Phase II III clinical trial will be included in the submission.
Speaker 3: Further demonstrating that aromachemal may reduce the long term progression of N.
Further demonstrating the Aramark the mall may reduce the long term progression of M. P C.
Speaker 3: We remain on track for the resubmission of the Aremotomal NDA by the end of the year, and as expected to be classified as a Class II submission, which will be subject to a six month review period by the FDA, placing the potential paedophidate in mid-2024.
We remain on track for the Resubmission of the Aramark them all NDA by the end of the year and is expected to be classified as a class two submission, which will be subject to a six month review period by the FDA, placing the potential <unk> date in mid 2024.
Speaker 3: We're also actively laying the groundwork for the commercial launch of Arab Mothamol in the US to make this new therapy accessible to people as soon as possible.
We're also actively laying the groundwork for the commercial launch of Aramark. The mall in the U S to make this new therapy accessible to people as soon as possible.
Speaker 3: We are developing the NPC market by raising awareness of the heterogeneous presentation, which includes visceral, neurological, and psychiatric symptoms, making it difficult to identify and diagnose.
We are developing the MPC market by raising awareness of the heterogeneous presentation, which includes visceral neurological and psychiatric symptoms.
It's difficult to identify and diagnose as.
Speaker 3: As we progress towards the potential approval and launch, we will continue to focus on advancing genetic testing programs, creating early diagnosis tools, and supporting the evolution of treatment guidelines to support families and reduce the time to treat.
As we progressed towards the potential approval and launch.
We will continue to focus on advancing genetic testing programs, creating early diagnosis tools and supporting the evolution of treatment guidelines to support families and reduce the time to treatment.
Speaker 3: As a trusted and committed partner, we've worked alongside people living with NPC to elevate their voice.
As a trusted and committed partner, we've worked alongside people living with MPC to elevate their voice.
Speaker 3: Their input has been instrumental in building awareness of the need for approved treatments for this debilitating condition.
Their input has been instrumental in building awareness of the need for approved treatments for this debilitating condition.
Speaker 3: We continue to work together to develop patient services that will provide access and a positive treatment experience.
We continue to work together to develop patient services that will provide access and a positive treatment experience.
Speaker 3: Now I'd like to turn your attention to KP 1077. Our clinical candidate, being developed as a treatment for idiopathic hypersomnia or I-8.
Now I would like to turn your attention to K P. 10, 77, our clinical candidate being developed as a treatment for idiopathic hypersomnia or IH. It is.
Speaker 3: It is estimated that approximately 37,000 people in the United States are currently diagnosed with IA.
Estimated at approximately 37000 people in the United States are currently diagnosed with IH.
Speaker 3: With one FDA-proofed treatment, there remains an unmet need for treatments with different mechanisms of action to address key unmet needs of excessive daytime sleepiness, sleep inertia, and cognitive dysfunction.
With one FDA approved treatment there remains an unmet need for treatments with different mechanisms of action to address key unmet needs of excessive daytime sleepiness sleep inertia and cognitive dysfunction.
Speaker 3: Our development program for rear sleep disorders continues to make meaningful progress. Last month, we reported interim data on the open label, those tight titration portion of the Phase II clinical trial. These results demonstrated that KP1077 is well-polarated at all those levels and regi-
Our development program for rare sleep disorders continues to make meaningful progress last month, we reported interim data on the open label dose titration portion of the phase III clinical trial.
These results demonstrated that <unk> 77 is well tolerated at all dose levels and regimens.
Speaker 3: We are also encouraged by the interim results and believe the unique PK profile may be well suited to address the unmet.
We're also encouraged by the interim results and believe the unique PK profile may be well suited to address the unmet need.
Speaker 3: As the program progresses, we are continuing enrollment at over 30 sites across the US and remain on track to report top-lying data in the first half of 2024.
As the program progresses, we are continuing enrollment at over 30 sites across the U S and remain on track to report topline data in the first half of 'twenty 'twenty four.
Speaker 3: These results will inform the design of the Phase III clinical trial in IH, which is expected to be initiated by the end of 2024.
These results will inform the design of the phase III clinical trial, NIH, which is expected to be initiated by the end of 2024.
Speaker 3: In addition to the progress being made on the Phase 2 trial, I'm pleased to share that our Phase 1 study under the NARTELEPSE IND is complete. And the data will be analyzed alongside the IH data to support the clinical development of both NARTELEPSE and IH program.
In addition to the progress being made on the phase II trial I'm pleased to share that our phase one study under the narcolepsy.
Is complete and the data will be analyzed alongside the IH data to support the clinical development of both narcolepsy and IH programs.
Speaker 3: Moving to our proposed acquisition of ACER. Upon closing, we will acquire complementary rare disease assets that accelerate our transition into a commercial cup.
Moving to our proposed acquisition of Pacer.
Upon closing, we will acquire complementary rare disease assets that accelerate our transition into a commercial company.
Speaker 3: This is a natural fit with zebra's mission, mission of to bring life-changing therapies to people with rare disease.
This is a natural fit with zebras mission mission to bring life changing therapies to people with rare diseases.
Speaker 3: Oprova indicated for the treatment of UCDs, which are a group of rare genetic disorders that can cause harmful ammonia to build up in the blood, potentially resulting in neurocognitive impairment, brain damage, and in some cases, even coma.
Oh prove are indicated for the treatment of Ucd's, which are a group of rare genetic disorders that can cause harmful ammonia to buildup in the blood potentially resulting in neuro cognitive impairment brain damage and in some cases, even coma.
Speaker 3: UCDs can be both symptomatic or asymptomatic, making it difficult to identify people who have not yet received a confirmatory diagnosis.
You see these can be both symptomatic or asymptomatic, making it difficult to identify people who have not yet received a confirmatory diagnosis.
Speaker 3: There are approximately 2,000 people in the United States of which roughly half are diagnosed and treated.
Approximately 2000 people in the United States of which roughly half are diagnosed and treated.
Speaker 3: The U.C.D. market and the U.S. is estimated at approximately $400 million annually.
Did you see any market in the U S is estimated at approximately $400 million annually.
Speaker 3: This is an attractive opportunity for Zebra, not only to build our commercial capabilities, but it's also a good strategic fit with Aramoklamal as there's a high degree of overlap between UCDs and NP.
This is an attractive opportunity for zebra not only to build our commercial capabilities, but it's also a good strategic fit with aramark them, all as Theres a high degree of overlap between the <unk> and N. P. C. Both our genetic disorders diagnosed by clinical geneticist and metabolic specialists and while other specialists are involved with the <unk>.
Speaker 3: Both are genetic disorders, diagnosed by clinical geneticists and metabolic specialists, and while other specialists are involved with the ongoing treatment, such as pediatric neurologists in the case of NPC, the majority of these experts work within a concentrated number of centers of X-
Ongoing treatment such as pediatric neurologists in the case of MPC. The majority of these experts work within a concentrated number of centers of excellence.
Speaker 3: This close proximity will allow us to reach the majority of prescribers and to realize synergies and scale with an efficient customer facing team.
This close proximity will allow us to reach the majority of prescribers and the realized synergies and scale with an efficient customer facing team.
Speaker 3: Acer shareholder meeting scheduled for tomorrow, November 8th. During which time of vote, to approve the deal will take place. This vote will mark a decisive moment for Acer and gives Zephyr the opportunity to build on the highly complementary programs that impact the lives of people with serious serious behaviour related to education and 250 projects that were a significant concern. Acer has also believed that arguing with the project duo was the handful for failure of decision-making, the project was a significant concern. Acer has also believed that arguing with the project duo was the handful for failure of decision-making,
Acer shareholder meeting scheduled for Tomorrow November 8th.
During which time a vote to approve the deal will take place. This vote will market decisive moment for Acer and gives us the opportunity to build on the highly complementary programs that impact the lives of people with serious rare diseases.
Speaker 3: Now hand the call over to Latouane, who will provide an update on our financial results and outlook. Thank you.
Now I'll hand, the call over to <unk>, who will provide an update on our financial results and outlook.
Thank you and good morning, everyone.
Speaker 3: As you can see from the updates the Neal has provided, Q3 2023 has been a time of incredible progress in our quest to make therapies available to people with rare disease.
As you can see from the updates that Neil has provided Q3 2023 has been a time of incredible progress in our quest to make therapies available to people with rare diseases.
Speaker 4: Our financial results for the quarter reflect our steady progress and continued investments in advancing our development programs and building out our commercial capabilities where they focus on patient services and advocacy.
Our financial results for the quarter reflect our steady progress and continued investments in advancing our development programs and building out our commercial capabilities with a focus on patient services and advocacy.
Speaker 4: For Q3 2023, we reported revenue of $2.9 million compared to the same amount for the same quarter in prior year.
For Q3, 2023, we reported revenue of $2 $9 million compared to the same amount for the same quarter in prior year.
Speaker 4: The U3-20023 revenue was comprised of $2 million in reimbursements from the French Early Access Program for Aremoclomo and royalties from the Astaris license of $900,000.
Q3, 2023 revenue was comprised of $2 million in reimbursements from the French and early access program for Aramark them all.
And royalties from the historic license of $900000.
Speaker 4: Compared to Q3 of last year, a Staris royalty set grown by $600,000.
Compared to Q3 of last year as far as royalties have grown by $600000.
Speaker 4: R&D expenses during the period were $12.3 million, which was an increase compared to $5.4 million for the same quarter in prior year.
R&D expenses during the period were $12 $3 million, which was an increase compared to five point, so $4 million for the same quarter in prior year.
Speaker 4: The increases due primarily to the ongoing phase 2 clinical trial for KP1077, as well as ongoing work to prepare the AERA-Mocomal NDA for resubmission later this year.
The increase is due primarily to the ongoing phase II clinical trial for K P $10 77 as.
As well as ongoing work to prepare the Aramark mall NDA for Resubmission later this year.
Speaker 4: GNA expenses were $5.8 million for Q3 2023 compared to $4 million in Q3 of last year.
G&A expenses were $5 8 million for Q3, 2023 compared to $4 million in Q3 of last year.
Speaker 4: This was consistent with our expectations and is driven by our business development activities and our ongoing investments into our commercial capability.
This was consistent with our expectations and is driven by our business development activities and our ongoing investments into our commercial capabilities.
Speaker 4: For Q3 2023, that loss was $14 million or $40 per basic and diluted chair compared to a net loss of $6.6 million or 19 cents per basic and diluted chair for the same period in 2022.
For Q3, 2023, net loss was $14 million or <unk> 40 per basic and diluted share compared to a net loss of $6 $6 million or <unk> 19 per basic and diluted share for the same period in 2022.
Speaker 4: Total cash, cash equivalents and securities were $83.4 million as a September 30, 2023, which was a decrease of $4 million compared to the prior quarter.
Total cash cash equivalents and securities were $83 $4 million as of September 32023, which was a decrease of $4 million compared to the prior quarter.
Speaker 4: The $5 million milestone payment earned under the Astaurus license agreement during Q2 2023 was received during Q3 and also at the use of cash during the quarter of $9 million, which was driven by the ongoing KP 1077 development program, investments in our commercial capabilities, and business development activities.
$5 million milestone payment earned under the <unk> license agreement. During Q2 2023 was received during Q3 and offset the use of cash during the quarter of $9 million, which was driven by the ongoing K P. 10, 77 development program investments in our commercial capabilities and business Development Act.
<unk>.
Speaker 4: As of September 30, 2023, total offstanding shares was $36.2 million, and fully diluted shares outstanding was $51.6 million.
As of September 32023, total outstanding shares was $36 2 million.
Fully diluted shares outstanding was $51 6 million.
Speaker 4: Our balance she remains strong and is expected to support our forecasted operating cash runway in to 2026.
Our balance sheet remains strong and is expected to support our forecasted operating cash runway into 2026.
Speaker 4: It is important to note that our forecast does not include commercial revenue from Aramakamal, which would follow potential FDA approval or the sale of the priority review voucher or any revenue from salesable proof of.
It is important to note that our forecast does not include commercial revenue from <unk>, which would follow potential FDA approval or.
Or the sale of the priority review voucher or any revenue from sales of approval.
Speaker 4: As you can see, there are many reasons for our optimism. We are focused on executing against our plan with the goal of creating long-term value for our shareholders.
As you can see there are many reasons for our optimism.
We are focused on executing against our plan with the goal of creating long term value for our shareholders.
We will now return the call to the operator for questions.
Speaker 1: At this time, if you wish to ask a question, please press star one on your telephone keypad.
Yeah.
At this time, if you wish to ask a question.
Press Star one on your telephone keypad.
Speaker 1: You may remove yourself from the cue by pressing star 2.
You may remove yourself from the queue by pressing star two.
Speaker 1: Once again that star 1 2 ask a question. Our first question will come from Louise Chin with Cantor Fitzgerald. Please go ahead.
Once again Thats star one to ask a question. Our first question will come from Louise Chen with Cantor Fitzgerald. Please go ahead.
Speaker 5: Hi, thanks for taking my questions and congratulations on all the progress this quarter. I wanted to ask you on Alperva, Alperva, and basically how you plan to commercialize this product and how you think about peak sales potential. And then for the Aramokomal NDA, was the additional or, you know, the larger amount of data that you said would take some time to review something that was requested by the FDA, or is it something that you wanted to provide? Thank you.
Hi, Thanks for taking my questions and congratulations on all the progress this quarter I wanted to ask you on a per box.
And basically how you plan to commercialize this product and how you think about peak sales potential and then for the <unk> NDA would be additional or you know against larger amount of data that you said would take some time to review something that was a question about the FDA or is it something that you wanted to provide thank you.
Speaker 3: Thank you, Luis. Alaska just to take the approval, commercialization questions, and then Alaska going to pass it off to Crystal to discuss the NDA resubmission.
Thank you Louise I'll ask Josh to take the old prove a commercialization questions and then I'll ask him to pass it off to.
Crystal to discuss the NDA resubmission.
Speaker 6: Thanks, Louise. With regards to the commercialization for Alpruva, we're excited for the deal to close, hopefully, any day now. Upon closure, we'll take over responsibility for commercializing this, and we see the opportunity to have a more full-sum lodge for the drug. Really targeting the first quarter to put full resources behind the commercialization.
Thanks, Louise with regards to the commercialization for it'll prove up we're excited for the deal to close hopefully.
Any day now.
Upon closure will take over responsibility for commercializing this and we see the opportunity to have a more fulsome launch for for the drug.
Targeting the first quarter to put full resources behind.
Behind the commercialization. So we anticipate and are putting in place a sales team medical liaisons, we have a particular focus on patient advocacy and working with the patient community to bring <unk> to those living with UCD as well as the prescribing community and we're building out a full suite of <unk>.
Speaker 6: So we anticipate and are putting in place a sales team medical A's on's. We have a particular focus on patient advocacy and working with the patient community to bring all proof of it to those living with UCD, as well as...
Speaker 6: And we're building out a full suite of patient services as well to ensure the patients have access to them.
Patient services as well too to ensure that patients have access to approval and we anticipate that all of that will be in place by the end of the year to really optimize the.
Speaker 6: And we anticipate that all of that will be in place by the end of the year to really optimize the commercial opportunity crop.
The commercial opportunity for all prove out with regards to revenue projections, we really haven't yet.
Speaker 6: With regards to revenue projections, we really haven't yet, we really haven't yet disclosed that and we'd like to get a couple of months of commercial activity behind us before we're ready to do that. But the market size, as you probably know, is about 400 million with several products in that market now and we see that we would have a...
We really haven't yet.
You don't disclose that and we'd like to get a couple of months of commercial activity behind us before we're ready to do that.
But the market size as you probably know is about 400 million with several products in that market now and we see that we would have.
Speaker 6: a differentiated product that would have the opportunity to take considerable share within that market.
A differentiated product that would have the opportunity to take considerable share within that market.
Passage of Crystal.
Speaker 7: So, Hi, Louise. Thanks for that question regarding the FDA and whether or not they request specific studies or they were just studies that we conducted ourselves. So throughout this process, we have had several interactions with the FDA. The CRL had three.
So hi, Louise Thanks for that question regarding the FDA and whether or not they requested specific studies or they were just studies that we we conducted ourselves.
Throughout this process, we have had several interactions with the FDA.
<unk> had.
Three.
Speaker 7: three concerns that they had and we feel like we have sufficiently addressed those concerns.
Three concerns that they had and we feel like we have sufficiently address those concerns.
Speaker 7: Again, all of that comes with a collaborative working relationship with the FDA. So as we decided on studies to conduct and got results for those studies, then we would meet with the FDA to discuss whether or not those were adequate. Again, most of those, it's all going to be review issues for the FDA. But we are confident that we have addressed what the FDA has asked us to.
Again, all of that comment with a collaborative working relationship with the FDA. So as we are.
Decided on studies to take.
To conduct and that results for those studies than we would meet with the FDA to discuss whether or not those were.
Adequate again most of those its all going to be reveal issues for the FDA, but we are confident that we have addressed what the FDA has had that that's down.
Thank you.
Speaker 1: Thank you once again if you would like to ask a question please press star one. Our next question comes from Jonathan Ashoff with Roth MKM. Please go ahead.
Thank you once again, if you would like to ask a question. Please press star one or.
Our next question comes from Jonathan Aschoff with Roth.
Please go ahead.
Speaker 8: Hi, good morning. Thank you. What were your exact stars?
Hi, guys. Good morning, and thank you what was your exact stars.
Speaker 9: World sees and he still expect 10 million in milestones and you know to be earned in this quarter We're actually 15 million Yeah, good morning Jonathan
A L T's and do you still expect $10 million in milestones.
You know to be earned in the in this quarter.
Were actually $15 million.
So Duane yeah. Good morning, Jonathan Thank you for the question.
Speaker 4: The exact was, I believe, was $880,000, so we rounded up to 900.
The exact was due I believe is 880000, so we rounded up to 900000 and with regard to the $10 million milestone we are definitely on track too.
Speaker 4: And with regard to the $10 million milestone, we are definitely on track to earn that milestone. And with regard to the $10 million milestone, we are definitely on track to earn that milestone.
Earned that milestone during Q4.
Speaker 4: And then that of course that cash would be received in Q1.
And then of course that cash will be received in Q1.
Speaker 8: And so that was one milestone, not a five and a 10 this quarter, just at just the 10.
And so that was one milestone not a five <unk> this quarter just it just the tests.
Speaker 4: The 5 was earned in Q2 and then the cash was received in Q3.
The five was earned in Q2 and then the cash was received in Q3 and so then looking ahead the $10 million would be earned in Q4, we believe and then <unk>.
Speaker 4: So then looking ahead, the 10 million would be earned in Q4, we believe, and then received.
<unk> in Q1.
Speaker 8: Okay, I have about this R&D spike. What does that look like going forward?
Okay I've got this R&D spike what is that look like going forward.
Speaker 4: The increase in R&D was certainly because we're in the right in the middle of this phase 2 trial. And so we're continuing to work through that. You can expect a similar trend going in the Q4. And then that top line data for the...
The increase in R&D was certainly because we're in the right in the middle of this phase two trial and so we're continuing to work through that you can expect a similar trend going into Q4, and then that top line data for the for the <unk> 77 phase II is expected in the first half of 'twenty four so as the click.
Speaker 4: for the K-P1077 Phase II is expected in the first half of 24. So as the clinical phase kind of winds down toward the beginning of that first half, you know, for Q1, then you'll see a moderate a bit.
Nicole phase kind of winds down towards the beginning of that first half for Q1, then youll see it moderate a bit.
Speaker 4: As you look into the end of 2024, that we intend to initiate the Phase III for KP1077.
As you look into the end of Q.
2024.
That we intend to initiate the phase III for <unk> 77, and so then maybe towards the end of next year you begin to see additional ramp up in R&D spend as that begins at the clinical phase there for the phase III.
Speaker 4: So then maybe toward the end of next year, you begin to see an additional ramp up. And R&D spend is that begins at the clinical phase there for the phase.
Speaker 8: Okay, in the US, how would you quantify that low hanging error mark will morph through? The identified patients who are eager to take something for NPC.
Okay.
How would you quantify that low hanging fruit.
Food together the idea identified patients who are eager to take.
Something for N P C.
Speaker 6: My Jonathan, this is Josh. I'd like to remind you that we have this ongoing expanded access program here in the U.S., which has roughly seven patients who are already...
Hi, Jonathan this is Josh.
I'd like to remind you that we have this ongoing expanded access program here in the U S, which has roughly 70 patients who are already receiving aramark them, all and it's our intention that upon approval. Our focus is really going to be to convert those patients who were receiving free clinical drug now to paid.
Speaker 6: air moccal mall and it's our intention that upon approval our focus is really going to be to convert those patients who are receiving free clinical drug now to paid patients on air moccal mall. So we see that as kind of a bolus of patients who will become commercial while at the same time being able to.
<unk> on air Mark them, all so we see that as kind of a bolus of patients who will become.
Commercial while at the same time being.
Naval too.
Speaker 6: Try and reach those other patients with NPC who might not yet be on air month.
Try and reach those other patients with MPC, who might not yet be on air Mark them all.
Speaker 6: I also remind you, you know, as we mentioned in our prepared remarks.
I'd also remind you as we mentioned in our prepared remarks.
Speaker 6: I'm sure you know, Aramachemal has a high degree of awareness within the prescribed community right now. So we've been working with the patient advocacy community and with the thought leaders to ensure that one Aramachemal is commercially available and approved that we'll be able to bring it to those patients.
I'm sure you know.
<unk> has a high degree of awareness within the prescribing community right now so.
We've been working with the patient advocacy community and with the thought leaders to to ensure that one aramark. The mall is commercially available and approved that we'll be able to bring bring it to those patients with with MPC now and they're roughly about $3 to 400 patients who are actively treated today.
Speaker 6: Now, they're roughly about three to 400 patients who are actively treated today.
Speaker 8: Okay, that's helpful. So how many additional sales and marketing heads do you think you'll bring on for both Alproofa and Aramaka, I'm all assuming approval in the middle of this next year? You know, buy the end of this.
Okay. That's helpful.
So how many additional sales and marketing head do you think you'll bring on for both all prove AR Antero Mako them, all assuming approval in the middle of next year.
Bye.
24, yeah.
Speaker 6: Yeah, I don't think we're yet prepared to give you a precise number, but we are planning to use the same team to commercialize both O'Proof and Air Mock them all. And given the fact that both markets are very concentrated and largely, these patients are treated within the same centers of excellence of which they're...
Yeah.
We are yet prepared to give you a precise number.
But we are planning to use a very use the same team to commercialize both approved and aramark them, all and given the fact that both markets are very concentrated and largely these patients are treated within the same centers of excellence of which there are roughly 40 or 50 across the country.
Speaker 6: roughly 40 or 50 across the country, we believe that we'll be able to reach the majority of physicians who diagnose and treat these patients with a very small team focused on those centers of excellence. And again, that team will be...
We believe that we'll be able to reach the majority of physicians, who diagnose and treat these patients with a very small team focused on those centers of excellence and again that team will be comprised of.
Speaker 6: sales team, marketing, account managers, talking with pairs and insurance.
Sales team marketing account managers talking with payers and ensuring that there's sufficient access for these patients.
Speaker 6: the patient access for these patients as well as the medical.
As well as a medical team out there as well.
Speaker 8: Yeah, because you had mentioned 90s and 20 for Aramaical, do you think 30 for everything or is it still something where it's that same number, 90 and 20?
Because you had mentioned.
Plenty quiet for Aramark of all these things.
30 for everything or is it still something where it's the same number guidance for 'twenty.
Speaker 6: Yeah, it's probably in that range, you know, in terms of the customer facing team, but then we'll also have patient services and a robust, you know, how that will provide support for those patients as they embark on their treatment journey.
Yes, it's probably in that range.
In terms of the customer facing team, but then we'll also have patient services and a robust.
Hub that will provide support for those patients as they embark on their treatment journey.
I think I think within that range is probably a good direction.
Speaker 8: Okay, and lastly, do you have any sense of timing for approve a label expansion? How aggressively you might go into maple syrup or anything else? Or are you also looking for additional orphan acquisition?
Okay, and lastly, do you have any sense of timing for.
All through the label expansion, how aggressively you might go into maple syrup or.
Or anything else or more still looking for additional acquisitions.
Speaker 3: Jonathan, Mrs. Neal, that's a great question. We're gonna, and what I'm doing as part of my listening towards really understanding what are the things that we need to focus on and have clear lines to execute on today. That is...
Jonathan This is Neil.
It's a great question.
We're going to and what I'm doing as part of my listening tour is really understanding what are the things that we need to focus on and have clear line to execute on today.
That is our ASER a program that is the our muscle program in that 10, 77, I think when I'm looking for the team right now to do is to evaluate the entire portfolio and find out how we're going to be able to make those decisions based on.
Speaker 3: Our ACER program, that is the ARMAPO program, and that's 1077.
Speaker 3: I think what I'm looking for the team right now to do is to evaluate the entire portfolio and find out how we're going to be able to make those decisions.
Speaker 3: based on prior to and prioritized those areas of development. So I think you got to stay tuned on that one. Give me another quarter and let's have that conversation next time.
Priority and prioritize those areas of development. So I think you got to stay tuned on that one give me another quarter and let's have that conversation next time.
Okay.
<unk>.
Speaker 1: Thank you. Our next question will come from Sumam Kokarni with Canacourt. Please go ahead.
Thank you. Our next question will come from <unk> Kulkarni with Canaccord. Please go ahead.
Speaker 10: Thanks for taking my questions and nice to see all the updates. So you mentioned several interactions with the FDA, but could you specify how long ago was the last?
Thanks for taking my questions and nice to see all the updates. So you mentioned several interactions with the FDA, but could you specify how long ago was the last injection.
Okay.
Speaker 7: Hi, Simont. Thanks for the question. Our last interaction was the FDA meeting at the beginning of August .
Hi, Mike Thanks.
Thanks for the question I'll ask interaction with the FDA meeting at the beginning of August.
Speaker 10: correct. And then assuming the company has both all proven and are more to more to commercialize next year, do you think the organization will be at capacity with selling two important products for some time or do you think the infrastructure could support additional acquired red disease products? And along those lines, how would you characterize the landscape of available red disease products today, especially given how markets have treated small and mid-cap biotech?
Got it and then.
Assuming the company had <unk> added more commodity commercialized next year you think.
Additionally, we had capacity with two important products for some time or do you think the infrastructure could support additional acquired entities products along those lines. How would you characterize the landscape of Beatable ended these startups debate, especially given how the markets are small and mid cap biotech.
Thank you so much.
Speaker 3: The opportunity for us to be able to accelerate our commercial platform and capabilities by bringing the talent together with Acer and Okrava and then Ara-Mokamal really is a platform. This is a step in the direction of us becoming a leading rare disease company.
The opportunity for us to be able to accelerate our commercial platform and capabilities by bringing the talent together with Acer and.
In <unk> and then a remarkable really is a platform. This is this is a step in the direction of us becoming a leading rare disease company. Our how we execute on that I think we will earn the right to be able to do this again and again and again so from a standpoint of will we be at capacity I think are clearly executing on what.
Speaker 3: how we execute on that, I think we'll earn the right to be able to do this again and again and again. So from a standpoint of will we be at capacity, I think clearly executing on what we have to do will allow us to be able to make that decision at the time as to bringing additional assets under the umbrella.
We have to do will allow us to be able to make that decision at the time as to bringing additional assets under the umbrella.
Speaker 6: You want to take the network? Yes, absolutely. And just to add on to what Neil said.
John do you want to take the necessary, yeah, absolutely and just to add onto what Neil said.
Speaker 6: We are building the commercial capability in a way that it can be scalable for that purpose.
We are building the commercial capability in a way that it can be scalable for that purpose and we're building. It today with the intention to execute against our approval plans, but scalable to then at Aramark them, all and potentially other rare disease products.
Speaker 6: building it today with the intention to execute against our approved plans but scalable to then add air and maca mull and potentially other rare disease products.
Speaker 6: effect comes, but that's the intent. And in terms of other rare disease products in the landscape, there are a number of very attractive other opportunities out there, whether they're in late stage clinical developments or...
If that comes but that's the intent and in terms of other rare disease products and the landscape. There are a number of very attractive other opportunities out there whether they are in late stage clinical development or or.
Speaker 6: already commercialized on the market. And so we are constantly scanning the the horizon for other opportunities. But our focus right now is really on building the commercial team and executing against the plants that we have in place.
Already commercialized on the market and so we are constantly scanning.
The horizon for other opportunities, but our focus right now is really on building the commercial team in executing against the plans that we have in place.
Got it thanks.
Speaker 1: Thank you our next question will come from Orrin Libnet with H.C. Rainlight. Please go ahead.
Thank you. Our next question will come from Orin Loopnet with H C. Wainwright. Please go ahead.
Speaker 11: Thanks for taking my questions. I think all of them are kind of touched on already, but just to build out some more, firstly, I don't follow, Acer, already. Can you just help us understand what is the current state of all proof of commercialization, so to speak already in terms of existing support awareness. You know, obviously you're going to, thematically, resource that product, early next year, but where is that at now?
Thanks for taking my questions.
I think all of them are kind of touched on it already but just to build out some more firstly I don't follow answer already can you just help us understand what is the current state Apple prove.
Commercialization so to speak already in terms of existing support awareness, obviously, you are going to dramatically.
Sorry, Scott products to eat out early next year, but where is that at now.
Speaker 6: or now Alaska just to hit on that. Yeah, Hey, Warren.
Yeah.
For now I'll ask just to hit on that.
Hey, Ron.
Speaker 6: So, approval was approved in late 2022, but didn't actually make it into the market and into the channels until July of this year due to some of the financial challenges that the company was faced. And so, consequently, there isn't a tremendous amount of awareness of approving the market.
So approval was was approved in late 2022, but didn't actually make it into the market and into the channels until July of this year due to some of the financial challenges that the company was faced and so consequently, there isn't a tremendous amount of.
<unk> of awareness of our proven in the market today.
Speaker 6: And the team at Acer is a pretty small team. They've been, I think, really effective, given the limited resources that they have.
And the team at Ace or is it.
Is it a pretty small team they've been I think really effective given the limited resources that they have.
Speaker 6: They don't have a sales force out there talking with physicians. They have a very small count team talking with payers. So we see this as a real opportunity to put appropriate resources behind it and to really drive the awareness of all proof and the benefits that it confers to our patients.
They don't have a sales force out there talking with physicians.
They have a very small account team talking with payers. So we see this as a real opportunity to put the appropriate resources behind it to <unk>.
Drive the awareness of <unk> and the benefits that it confers to our patients and we think with that concerted efforts in the combined efforts of our two companies coming together, we can really drive awareness and demand for for approval.
Speaker 6: And we think with that, certain efforts and the combined efforts of our two companies coming together, we can really drive awareness and demand for the forum.
Speaker 11: Okay, and I guess big our picture conceptually.
Okay, and I guess bigger picture conceptually.
Speaker 11: As you sort of lay a commercial groundwork now well ahead of potential air moclemall approval, which is a new development in the story versus if he wants to go, how do you think about
Are you sort of lay a commercial groundwork now well ahead of potential Aramark Lam all approval, which is.
New development in the story versus a few months ago.
How do you think about that.
Speaker 11: potential return on investment and shareholder evaluation with old Prova alone versus needing Aramaquimal to lay on top of that infrastructure. I guess another to put it, do you think, do you need Aramaquimal in addition to Old Prova to really...
The potential return on investment and shareholder value creation with <unk> alone.
Versus needing aramark homology to layer on top of that infrastructure I guess another to put it or do you think do you need Aramark I'm. All in addition to all prove out to.
Really.
Speaker 6: reaper awards of this infrastructure going to build out or is all proof of a big enough opportunity alone in your view and air macklemmall essentially I guess so to speak gravy all gravy on top of that. Yeah thanks, thanks, Lauren. I'm going to ask Josh to take that as one of the lead architects of the acquisition team. Yeah, and Lauren thinks again for that question.
Regional rewards of this infrastructure youre going to build out or is all true.
Big enough opportunity alone in your view and are marking them all essentially I guess so to speak gravy.
On top of that.
That's it.
Thanks, Lauren I'm going to ask Josh to take that as one of the lead architects of our of.
The acquisition team yeah.
Or and thanks again for that question.
Speaker 4: One of the reasons why this acquisition and bringing the two companies together was so attractive for us was that we see really a standalone potential of all Prova to support the build-out of a commercial team.
One of the reasons why this acquisition of <unk>.
Bringing the two companies together was so attractive for US was that we see.
Really a standalone potential of all prove a to support the build out of our commercial team keep.
Speaker 6: Keep in mind, as I mentioned earlier, this is going to be a very focused and efficient team where we're putting appropriate resources behind it. We're being very prudent about how we're doing that in a way that...
Keep in mind as I mentioned earlier this is going to be a very.
Focused and efficient team, where we're what we're putting appropriate resources behind it we're being very prudent about how we're doing that in a way that it's the right investment for for that opportunity and then we can scale it as appropriate as Irma ophthalmology and other products come on board, but the deal was structured.
Speaker 4: the right investment for that opportunity, and then we can scale it as appropriate as Air Machuimal and other products come on board. But the deal was structured in a way that it took into.
In a way that.
It took into.
Speaker 6: took into consideration the possible scenarios that we were only commercializing our mock-comal and we believe that the investment that we're putting behind it will have an appropriate return given the deal starts.
Took into consideration the possible scenarios that we were only commercializing neuromuscular mall and we believe that the investment that we're putting behind it we.
We will have an appropriate return given given the deal structure that we that.
We stroke.
Speaker 11: All right, and if I could just change gears to a stair, I've covered you guys for a long time. And so a lot of times thinking about that, and it's ironic now that that product, I guess really taken off, it's not really the focus of your story. So as you look at that product going forward, it's doing really well and throwing off meaningful milestones and growing royalties now, is that something that you would look to potentially monetize for some non-deludive financing now? Financing now going, you know, eh.
Alright, if I could just change gears to us there I see that I've covered you guys for a long time and so a lot of time thinking about that.
Ironic now that that product I guess, it really taken off.
That's not really the focus of your story. So as you look at that product going forward.
It's doing really well and throwing off meaningful milestones and growing royalty is now is that something that you would look to potentially monetize or some non dilutive financing now Glenn.
Speaker 11: and sort of cut bait on that product and focus entirely on the orphan business or is that something you wanna maintain an ongoing relationship with a coreamp.
And sort of cut bait on that product to focus entirely on the orphan business or is that something you want to maintain an ongoing relationship with corium.
Speaker 3: Yeah, thanks, Lauren. We're pleased with the performance and the acceleration that's happening in the Starris program for sure with our partners in regards to financing strategies and what we might do moving forward. I'll pass that off to LeDoin.
Yes. Thanks Oren, we're we're pleased with the performance and the acceleration that's happening as the austerity program for sure with our with our partners in regards to financing strategies and what we might do moving forward I'll I'll pass that off to to let Dwayne.
Speaker 4: You know, Warren, right now, I think, you know, we're pleased in watching the progress of COREM, as Neal mentioned, and so we're excited to continue following those trends. You know, our financing strategy is a bit of a broader question than just a single part of our portfolio. And I think that, you know, we'll be evaluating all those things to make sure we have the capital we need to support Josh's efforts, to make sure each of our catalysts are met. And then ultimately, we...
Yes, right now I think we're pleased and watching the progress of corium as Neil mentioned and so we're excited to continue following those trends.
Our financing strategy is a bit of a broader question, Jim just a single part of our portfolio and I think that we.
We'll be evaluating all of those things to make sure we have the capital we need to support Josh as efforts to make sure each of our catalysts are met and that ultimately.
Speaker 9: Candlely, we want to get to a point where we're cash positive as a company and we're going to continue working toward that.
Candidly, we want to get to a point, where we're cash flow positive as a company and we can we're going to continue working toward that.
Alright, I think Thats all I got for now thank you.
Good luck.
Thank you.
Speaker 1: Thank you. That does conclude our question and answer session for today. I would now like to turn the call back over to Neil McFarley for any additional closing remarks.
Thank you that does conclude our question and answer session for today I would now like to turn the call back over to Neil Mcfarlane for any additional or closing remarks.
Thank you Todd.
Speaker 3: You know, as we look to the end of 2023 and in the 2024, we're focused on three key priorities. First, to close the AACSRAC physician and commercialize or approve of for patients. Second, to resubmit the ARMAXAMOL NDA and make that product available. And third, to complete the space to trial in idiopathic hypersomnia and prepare an advance KP1077 with a differentiated profile in the face.
As we look to the end of 2023 and the end of 2024, we're focused on three key priorities first to close the <unk> acquisition and commercialize all approval for patients second to resubmit, the <unk> NDA and make that product available and third to complete the phase II trial in idiopathic hypersomnia and.
Prepare to advance <unk> <unk> 77, with a differentiated profile in the phase III.
Speaker 3: Thank you all for your time today and we look forward to speaking with you.
Thank you all for your time today, and we look forward to speaking with you in the future.
Speaker 1: This does conclude today's EverThera Putus 3rd quarter 2023 results conference call. You may disconnect your line at this time and have a wonderful day.
This does conclude today's separate therapeutics third quarter 2023 results conference call.
You may disconnect your lines at this time and have a wonderful day.
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