Zevra Therapeutics Q3 2023 Earnings Call Transcript

Quartr
Provided by Quartr
November 7, 2023

There are 9 speakers on the call.

Operator

Good morning, everyone. Thank you for joining the Zevra Therapeutics Third Quarter 2023 Corporate Updates and Financial Results Call. Today's call is being recorded and will be made available on the company's website following the conclusion of the call. With that, I will now turn the call over to Nicole Ochsner, Vice President of Investor Relations and Corporate Communications for Zephyrra Therapeutics. Please go ahead.

Speaker 1

Good morning and thank you for joining us today to review Deborah Therapeutics' progress and the Q3 of 2023 outlining our clinical advances, operational achievements and financial results. 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 of the Zevr website. While we will not Using slides on today's call, an updated corporate presentation will be made available on our website later today. As we move forward, it's important to highlight that the company's discussions will include forward looking statements.

Speaker 1

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 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 10 Q and our annual report on Form 10 ks. I am pleased to welcome Zebra's management team members participating in today's call. I'm joined by Neil MacFarlane, President and Chief Executive Officer LaDuane Clifton, our Chief Financial Officer Joshua Schafer, our Chief Commercial Officer and EVP of Business Development and Crystal Mickle, our Chief Development Officer. With that, I'll turn the call over to Neil.

Speaker 2

Thank you, Nicole, and thank you all for making the time to join us today. It's truly an honor for me to have the opportunity to lead Zevra 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. 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 Acer. After that, I'll turn it over to LeDuane The candidates for people living with rare diseases is well on its way.

Speaker 2

The mission of the company with a deep focus on people living with rare diseases It's 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. I would like to thank Crystal for her work as Interim President and CEO. She kept our programs moving forward while leading the company The transition. Crystal has now returned to her role as Chief Development Officer and will continue to advance our clinical programs. Joe Saluri also recently retired from the Board.

Speaker 2

We thank him for his dedication and service to the company's growth and success. I would also like to take the opportunity to thank our shareholders that have remained committed to Zevra on its journey to becoming a leading rare disease 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. Aromoclonal is poised to be the 1st drug approved in the U. S.

Speaker 2

For the treatment of Niemann Pick Disease Type which currently has no approved therapy in the U. S, creating an urgent need for people who are awaiting the treatment option. To date, aramol has been studied in more than 10 different clinical trials evaluating its safety and efficacy across over 500 subjects. 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. We are confident that each of the deficiencies raised are being addressed in the resubmission.

Speaker 2

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. 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 U. S. And the EU. In addition, data from the 4 year open label extension of the Phase twothree clinical trial will be included in the submission, further demonstrating that aramoxamol may reduce the long term progression of NPC.

Speaker 2

We remain on track for the resubmission of the aramaxamol NDA by the end of the year and is expected to be classified as a Class II submission, which will be subject to a 6 month review period by the FDA, placing the potential PDUFA date in mid-twenty 24. We are also actively laying the groundwork for the commercial launch of aramoftimol in the U. S. To make this new therapy accessible to people as soon as possible. We are developing the MPC market by raising awareness of the heterogeneous presentation, which includes visceral, neurological and psychiatric making it difficult to identify and diagnose.

Speaker 2

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 treatment. As a trusted and committed partner, we've worked alongside people living with NPC to elevate their voice. Their input has been instrumental in building awareness of the need for approved treatments for the debilitating condition. We continue to work together to develop patient services that will provide access and a positive treatment experience. Now I'd like to turn your attention to KP-ten seventy seven, our clinical candidate being developed as a treatment for idiopathic hypersomnia or IH.

Speaker 2

It is estimated that approximately 37,000 people in the United States are currently diagnosed with IH. 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. 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 2 clinical trial. These results demonstrated that KP-ten seventy seven is well tolerated at all dose levels and regimens.

Speaker 2

We are also encouraged by the interim results and believe the unique PK profile may be well suited to address the unmet need. As the program progresses, we are These results will inform the design of the Phase 3 clinical trial in IH, which is expected to be initiated by the end of 2024. In addition to the progress being made on the Phase II trial, I'm pleased to share that our Phase I study under the narcolepsy IND is complete And the data will be analyzed alongside the IH data to support the clinical development of both narcolepsy and IH programs. Moving to our proposed acquisition of Acer. Upon closing, we will acquire complementary rare disease assets that accelerate our transition into a This is a natural fit with Zevra's mission to bring life changing therapies to people with rare diseases.

Speaker 2

Opruva 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. 2,000 people in the United States, of which roughly half are diagnosed and treated. The UCD market in the U. S. Is estimated at approximately $400,000,000 annually.

Speaker 2

This is an attractive opportunity for Zebra not only to build our commercial capabilities, but it's also a good strategic fit with Aramarkamal There's a high degree of overlap between UCDs and NPC, 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 excellence. This close proximity will allow us to reach the majority of prescribers and to realize synergies and scale with an efficient customer facing team. Acer shareholder meeting is scheduled for tomorrow, November 8, during which time a vote to approve the deal will take place. This vote will mark a decisive moment for Acer and give Zebra the opportunity to build on Highly complementary programs that impact the lives of people with serious rare diseases. Now I'll hand the call over to LaDuane, who will provide an update on our financial results and outlook.

Speaker 2

Thank you, and good morning, everyone. As you can see from the update that Neil has provided, Q3 2023 has been a time of incredible progress and our quest to make therapies available to people with rare diseases. 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. For Q3 2023, we reported revenue of $2,900,000 compared to the same amount for the same quarter in prior year. Q3 2023 revenue was comprised of $2,000,000 in reimbursements from the French early access program for Aramarkimel and royalties from the Astaris license of $900,000 Compared to Q3 of last year, Astar's royalties have grown by $600,000 R and D expenses during the period were $12,300,000 which was an increase compared to $5,400,000 for the same quarter and prior year.

Speaker 2

The This is due primarily to the ongoing Phase 2 clinical trial for KP-ten seventy seven as well as ongoing work to prepare the aramofimol NDA for resubmission later this year. G and A expenses were $5,800,000 for Q3 2023 compared to $4,000,000 in Q3 of last year. This was consistent with our expectations and is driven by our business development activities and our ongoing investments into our commercial capabilities. For Q3 2023, net loss was $14,000,000 or $0.40 per basic and diluted share compared to a net loss of $6,600,000 or $0.19 per basic and diluted share for the same period in 2022. Total cash, cash equivalents and securities were $83,400,000 As of September 30, 2023, which was a decrease of $4,000,000 compared to the prior quarter, the 5 and offset the use of cash during the quarter of $9,000,000 which was driven by the ongoing KP-ten seventy seven development program, investments in our commercial capabilities and business development activities.

Speaker 2

As of September 30, 20 20 3 total outstanding shares was 36,200,000 and fully diluted shares outstanding was 51,600,000. Our balance sheet remains strong and is expected to support our forecasted operating cash runway into 2026. It is important to note that our forecast does not include commercial revenue from Aramocimel, which would follow potential FDA approval or the sale of the priority review voucher or any revenue from sales of review voucher or any revenue from sales of Opruva. 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.

Operator

Our first question will come from Louise Chen with Cantor Fitzgerald. Please go ahead.

Speaker 3

Hi, thanks for taking my questions and congratulations on all the progress this quarter. I wanted to ask you on Apruva, and basically how you plan to commercialize this product and how you think about peak sales potential? And then for the Aramokamal NDA, Was the additional or I guess larger amount of data that you said would take some time to review something that was Question about the FDA or is it something that you wanted to provide? Thank you.

Speaker 2

Thank you, Luis. I'll ask Josh to take the ALFRUVA commercialization And then I'll ask him to pass it off to Crystal to discuss the NDA resubmission.

Speaker 4

Great. Thanks, Louise. With regards to the commercialization for Ultruva, 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 fulsome launch For the drug, really targeting the Q1 to put full resources behind Commercialization. So we anticipate and are putting in place a sales team, medical liaisons.

Speaker 4

We have a particular focus on And we're building out a full suite of patient services as well to ensure that patients have access to Opruva. And we anticipate that all of that will be in place by the end of the year to really optimized the commercial opportunity for UltralProvo. With regards to revenue projections, we really haven't yet We 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 $400,000,000 with several products in that market now, and we see that we would have A differentiated product that would have the opportunity to take considerable share within that market. I'll pass it to Crystal.

Speaker 5

So hi Louise, thanks for that question regarding the FDA and whether or not they requested Studies, or they were just studies that we conducted ourselves. So throughout this process, we have We've had several interactions with the FDA. The CRL had 3 concerns that they had, and we Chip 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 for the FDA, but we are confident that we have addressed what the FDA has asked us to.

Speaker 1

Thank you.

Operator

Thank you. Our next question comes from Jonathan Nashoff with ROTH MKM. Please go ahead.

Speaker 6

Hi, guys. Good morning. Thank you. What were your Back to Astara's royalties and do you still expect $10,000,000 in milestones to be earned in this quarter We're actually $15,000,000

Speaker 2

Yes. Good morning, Jonathan. Thank you for the question. The exact was, I believe it's $880,000 So we rounded up to $900,000 And with regard to the $10,000,000 milestone, We are definitely on track to earn that milestone during Q4. And then that of course that cash would be received in Q1.

Speaker 6

And so that was one milestone, not a 5 and a 10 this quarter, just the 10?

Speaker 2

The $5,000,000 was earned in Q2 and then the cash was received in Q3. And so then looking ahead, The $10,000,000 would be earned in Q4, we believe, and then received in Q1.

Speaker 6

Okay. How about this R and D spike? What does that look like going forward?

Speaker 2

The increase in R and D was certainly because we're in the right in the middle of this Phase 2 trial. 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 KP-ten seventy seven Phase 2 is expected in 1st half of 'twenty four. So as the clinical phase kind of winds down towards the beginning of that first half For Q1, then you'll see it moderate a bit.

Speaker 2

As you look into the end of 2024, that we intend to Initiate the Phase 3 for KP-ten seventy seven. And so then maybe toward the end of next year, you'll begin to see additional ramp up in R and D spend as that begins at the clinical phase

Speaker 6

Okay. In the U. S, how would you quantify that low hanging Aramach LaMol Foods, the identified patients who are eager to take something for NPC.

Speaker 4

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 aramaxamol. 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 Hermoqamil.

Speaker 4

So We see that as kind of a bolus of patients who will become commercial, while at the same time being able to Try and reach those other patients with NPC who might not yet be on aramaximol. I'd also remind you, as we've mentioned in Prepared remarks and as I'm sure you know, aramaximol has a high degree of awareness within the prescribing community So we've been working with the patient advocacy community and with the thought leaders to ensure that 1 Aramacamole Is commercially available and approved that we'll be able to bring it to those patients with MPC now. There are roughly about 300 to 400 patients who are actively treated today.

Speaker 6

Okay. That's helpful. So how many additional sales and marketing hit do you think you'll bring on for both ALprova and Aramarko Mall assuming approval in the middle of next year by the end of 'twenty four?

Speaker 4

Yes. I don't think we're yet prepared to give you a precise number. But we are planning to use a very use the same team to commercialize both Opruva And it's Aramark Namal. And given the fact that both markets are very concentrated and largely these patients are treated within Same centers of excellence of which there are roughly 40 or 50 across the country, we believe that we'll be able to reach the majority of physicians Diagnose and treat these patients with a very small team focused on those centers of excellence. And again, that team will be Comprised of sales team, marketing, account managers talking with payers and ensuring that there's sufficient access For these patients as well as the medical team out there as well.

Speaker 6

Yes, because you had mentioned 90 20. Why for Aramaklobal? Do you think 30 for everything? Or is it still something where it's that same number, not even 20?

Speaker 4

Yes, it's probably in that range in terms of the customer facing team, but then we'll also have patient Services and robust hub that will provide support for those patients as they embark And their treatment journey. So I think within that range is probably a good direction.

Speaker 6

Okay. And lastly, do you have any sense of timing for ALPUVA label expansion? How aggressively you might go into maple syrup Or anything else? Or are you more so looking for additional orphan acquisition?

Speaker 2

Jonathan, this is Neil. That'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 And have clear line to execute on today. That is our ACER program, that is the ARM model program, that's 10/70 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 based on And prioritize those areas of development. So I think you got to stay tuned on that one.

Speaker 2

Give me another quarter, and let's have that conversation next

Speaker 6

Okay. Thank you.

Operator

Thank you. Our next question will come from Sumant Kulkarni with Canaccord. Please go ahead.

Speaker 7

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 interaction?

Speaker 5

Yes. Hi, Sumant. Thanks for the question. Our last interaction was the FDA meeting at the beginning of August.

Speaker 8

Got it.

Speaker 7

And then assuming the company had both Artoova and Aramocimol to commercialize next year, do you think the organization will be at with selling 2 important products for some time or do you think the infrastructure could support additional acquired rare disease products? And along those lines, How would you characterize the landscape of available rare disease products today, especially given how markets have treated small and mid cap biotech?

Speaker 2

Thank you, Sumant. The opportunity for us to be able to accelerate our commercial platform and capabilities by bringing the talent together with Acer and Olpruva and then Aramatamal really is a platform. This is a step in the direction of us becoming a leading rare 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 bring in additional assets under the umbrella. Yes, do you want to take the next one?

Speaker 4

Yes, absolutely. And just to add on to what Neil said, 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 If that 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 development or already commercializing on the market. And We are constantly scanning the horizon for other opportunities.

Speaker 4

But our focus right now is really on building the commercial team and executing against The plans that we have in place.

Speaker 7

Got it. Thanks.

Operator

Thank you. Our next We'll come from Oren Lipmat with H. C. Wainwright. Please go ahead.

Speaker 8

Thanks for taking my questions. I think all of Mark 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 Opuva commercialization, so to speak, already in terms of existing support Obviously, you're going to dramatically resource that product early next year, but where is that at now?

Speaker 2

Warren, I'll ask Josh to hit on that.

Speaker 4

Yes. Hey, Lauren. So, ALPRUVA 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 Opruva in the And the team at Acer is a pretty small team. They've been, I think, really effective given The limited resources that they have, that they don't have a sales force out there talking with physicians.

Speaker 4

They have a very small account 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 And we think with that concerted efforts and the combined efforts of our 2 companies coming together, We can really drive awareness and demand for Ultralfirma.

Speaker 8

Okay. And I guess bigger picture conceptually, as you sort of lay commercial groundwork now well ahead Arameklamal approval, which is a new development in the story versus a few months ago. How do you think about The potential return on investment and shareholder value creation with Ultruva alone Versus needing Aramaicomole to layer on top of that infrastructure, I guess, another input is, do you think Do you need Aramakimal in addition to Ultralpa to really reach the rewards of this infrastructure you're going Build out or is Ultralb a big enough opportunity alone in your view and Aramaklamal essentially, I guess, so to speak, gravy, All great on top of that.

Speaker 2

Yes. Thanks, Oren. I'm going to ask Josh to take that as one of the lead architects of the acquisition team. Yes. And Oren, thanks

Speaker 7

again for

Speaker 4

that. Yes. And Oren, thanks again for that question. And one of the reasons why this acquisition and bringing the two Together was so attractive for us was that we see really a standalone potential of Alprova to support the build out of a commercial team. Keep in mind, as I mentioned earlier, this is going to be a very focused An efficient team where we're while we're putting appropriate resources behind it, we're being very prudent about how we're doing that in a way It's the right investment for that opportunity and then we can scale it as appropriate as bermaklamole and other products come on board.

Speaker 4

But the deal was structured in a way that it took into consideration the possible Scenario that we were only commercializing Aeromak LaMalle and we believe that the investment that we're putting behind it will have an appropriate return given The deal structure that we struck.

Speaker 8

All right. If I could just change gears to Asterias, I've covered you guys for a long time. So a lot of time thinking about that and it's ironic now that that product has, I guess, really taken off. It's not really the focus 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 dilutive financing now going and sort of cut bait on that product is focused entirely on the orphan business or is that Something that you want to maintain an ongoing relationship with Coriant.

Speaker 2

Yes. Thanks, Oren. We're pleased with the performance and the acceleration That's happening in the Asterisk program for sure with our partners. In regards to financing strategies and what we might do moving forward, I'll pass that off Hello, Duane. Yes, Warren, right now, I think we're pleased in watching The progress of Coriant, as Neil mentioned, and so we're excited to continue following those trends.

Speaker 2

Our financing strategy is a bit of a broader question than just a single part of our And I think that 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 that ultimately, we 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. All right.

Speaker 8

I think that's all I got for now. Thank you. Good luck.

Speaker 2

Thank you.

Operator

Thank you. That does conclude our question and Session for today, I would now like to turn the call back over to Neil McFarlane for any additional or closing remarks.

Speaker 2

Thank you, Todd. As we look to the end of 2023 and the end of 2024, we're focused on 3 key priorities. 1st, To close the Acer acquisition and commercialize Olprufa for patients. 2nd, to resubmit the aramaxamol NDA and make that product available. And 3rd, to complete the Phase 2 trial in idiopathic hypersomnia and prepare to advance KP-ten seventy seven with a differentiated profile into Phase 3.

Speaker 2

Thank you all for your time today, and we look forward to speaking with you in the future.

Operator

This does conclude today's Zebra Therapeutics 3rd quarter 2023 results conference call.

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