Q3 2024 Zevra Therapeutics Inc Earnings Call
Please stand by, we're about to begin.
Thank you and welcome to those who are joining US today, we will provide an overview of our accomplishments in the third quarter and recent weeks followed by a review of financial results I encourage you to read our financial results news release, which we distribute this afternoon and is available in the investors section.
<unk> of our website.
Before we begin the call. 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 severance business.
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 projections made these forward looking statements should be evaluated together with the cautionary statements contained in the risk factors section of.
Our most recent quarterly report on Form 10-Q, our annual report on Form 10-K, and other filings with the SEC.
I'm pleased to welcome the <unk> management team members participating in today's call.
Speaker Change: Neil Macfarlane <unk>, President and Chief Executive Officer, let Dwayne Clifton.
Speaker Change: Our Chief Financial Officer, and Josh Schaffer, our Chief commercial officer, and EVP of business development.
Speaker Change: Also joining us today for the question and answer session is Adrian Foretell, our Chief Medical Officer now, it's my pleasure to hand, the call over to Neil.
Neil Macfarlane: Thank you Nicole.
Neil Macfarlane: Thank you for joining us this afternoon.
Speaker Change: On the call today, we will provide an update on the company's progress towards achieving our 2024 goals review the milestones achieved in the third quarter and share with you our priorities going forward.
Speaker Change: I would like to start by reflecting on the progress we made over the last few quarters, where the statement I made in the annual shareholder letter earlier this year.
Speaker Change: I mentioned, how we must execute as one zebra focus our resources on high impact opportunities and drive innovation by investing in programs that clearly address unmet medical need.
Speaker Change: Thus far the pace of Zephyrus transformation has been tremendous and while we celebrate our success. We know there is more to do to achieve our ultimate goals.
Speaker Change: Today I'm pleased to report on our progress in the third quarter, which was rich with milestones. Our crowning achievement was the approval and launch of my <unk>. The first FDA approved therapy for the treatment of Niemann pick disease type seat or N. P C.
Speaker Change: This accomplishment was an outcome of our close collaboration with the NPC community and we are delighted to celebrate this victory with them.
The launch of my <unk> has exceeded our expectations with 90 prescription enrollment forms submitted as of October 31.
This is largely due to the extensive prelaunch preparations and the deep relationships, we have built with MPC prescribers.
Speaker Change: Additionally, we expect to have my <unk> available for patient shipment in line with our previous guidance.
Speaker Change: We also announced the receipt of a rare pediatric disease priority review voucher or <unk> with the approval of my playbook, and we intend to monetize the voucher to support our future growth with non dilutive capital.
Speaker Change: Let's turn to approval, our commercial product for the treatment of certain urea cycle disorders or do you see these.
Speaker Change: Our launch has been impacted by low patient awareness and reimbursement hurdles for new products and the UCD market.
Speaker Change: With only three prescription enrollment forms received in the third quarter. The launch is underperforming and we have taken actions to remedy this.
Speaker Change: To better understand the challenges we face we conducted a comprehensive external market and brand assessment that provided us with feedback from the UCD community.
Speaker Change: Based on this we adjusted to focus our efforts on patient segments that we believe have the greatest potential to benefit from approval and to revitalize the launch.
Speaker Change: Turning briefly to our pipeline, we achieved several clinical milestones in the quarter as well.
Speaker Change: We conducted an end of phase II meeting with the FDA at the end of September.
Speaker Change: And have clear direction for our phase III path forward regarding $10 77 in idiopathic hypersomnia or IH.
Speaker Change: In addition, we met our goal of dosing new patients and the recently re initiated phase III program for select below I.
Speaker Change: I will talk more about these programs after Josh is update on the commercial launches.
Speaker Change: As we approached the end of the year I can report that we've completed our portfolio assessment and developed our strategic plan for 2025.
Speaker Change: This plan builds on our many accomplishments while taking into consideration external market factors and our internal capabilities, along with an exhaustive evaluation of scenarios on how best to drive stakeholder value.
Speaker Change: Looking to the future we have a plan to drive continued transformation and have organized our priorities around four key pillars that you'll hear me refer to today and on future calls.
Marshall Excellence pipeline and innovation.
Speaker Change: Talent and culture and corporate Foundation.
Speaker Change: To start we must demonstrate commercial excellence through strong execution.
Speaker Change: Our business model is built to bring new therapies to highly targeted patients and health care professional communities.
Speaker Change: We will seek to maximize the potential of our current and future products by leveraging our expertise and infrastructure investment.
Speaker Change: Tangible outcome is our sense of urgency to make my playful available globally with pursuit of a regulatory submission in Europe as our top priority.
Speaker Change: Pipeline and innovation refers to ensuring that we develop products that address a significant unmet need within the rare disease community.
Speaker Change: We conducted a comprehensive review of our pipeline assets and as a result, we've halted some development activities, most notably <unk>.
Speaker Change: We also made the difficult but necessary decision to discontinue our in house pro drug discovery activities and close our facilities in Iowa and Virginia.
Speaker Change: With the intent to outsource lab efforts for programs that move forward.
Speaker Change: Additionally, we are actively seeking to extract value from our legacy intellectual property portfolio through active outreach with long term success in mind, we will re prioritize those resources to late stage programs that maximize our research and development capabilities, allowing us to expand our portfolio with.
Speaker Change: Mentally assets.
Speaker Change: Achieving great things for patients requires zebra to develop great talent and culture.
As the needs of our business and portfolio evolve, we will look to expand our internal capabilities relative to future growth.
Speaker Change: We can only achieve the ambitions of our strategic plan by shaping and engaging a high performing team and living and Unapologetically patient centric culture.
Speaker Change: Our corporate foundation refers to financial strength and disciplined capital allocation to execute our key priorities.
Speaker Change: This underpins all other pillars facilitates growth and allows us to responsibly invest in our long term transformation.
Speaker Change: Part of our future growth will be inorganic and we plan to establish zebra as a partner of choice for companies with late stage development or commercial rare disease assets now.
Speaker Change: Now I'll turn the call over to Josh who will provide an update on our commercial launches for my playful and I'll prove up Josh.
Josh Schaffer: Thanks Neil.
We believe the FDA approvals might play for was one of monumental importance to those living with MPC, along with their family members patient advocacy groups and clinicians.
Josh Schaffer: It also marked an extraordinary milestone for zebra as we advance our commercial efforts to bring new treatments for rare diseases.
Speaker Change: As Neil mentioned, we received 90 prescription enrollment forms as of October 31 <unk>.
Speaker Change: Exceeding our internal expectations.
These enrollment forms came from both the patients who are in our U S expanded access program or EAP as well as patients who are not previously on my Plaza.
As a reminder, an enrollment is a prescription submitted to our specialty pharmacy, which initiated the benefits investigation process ultimately leading to a decision on reimbursement and paid dispenses.
Speaker Change: As I mentioned in our September call a top launch priority is to convert EAP patients to my place.
Speaker Change: And at the time of FDA approval the program had grown to 83 patients.
Speaker Change: As of the end of October 69 of those patients had submitted an enrolment form.
Speaker Change: Given the speed of this transition we anticipate closing the EAP program in the second quarter of next year well ahead of the expected 12 months post launch timeframe. We previously reported with our intent to leave no patient behind.
Speaker Change: Our patient services team has been working through the benefits investigation process for each submitted enrollment.
Speaker Change: As of the end of October approximately 30% of the enrollment forms received had been approved for reimbursement and are ready for fulfillment upon drug availability in the channel, which will be within our previous guidance of eight to 12 weeks post launch.
Speaker Change: We are pleased with the early progress with regards to enrollments and on future calls, we intend to provide metrics on market access and revenue.
Speaker Change: To recap the prevalence and market opportunity for MPC estimates show that approximately 900 people in the U S are living with the disease. However, only about a third of this population are between 300 and 350 patients have been diagnosed and treated.
Speaker Change: Data show that of those treated approximately 80% are currently receiving niglet stat.
Speaker Change: And as a reminder, my place is approved and indicated for the use in combination with Mega stat for the treatment of neurological manifestations of MPC and adult and pediatric patients two years of age and older.
Speaker Change: The robust demand that we've seen for my place as a direct response to the clinical benefits of physicians and patients have experienced through our long term EAP and open label extension studies.
Speaker Change: And we are confident that might play for in combination with <unk> have the potential.
Speaker Change: <unk> to become the cornerstone treatment for MPC.
Speaker Change: Our position is bolstered through physician feedback on the clinical data that supported the FDA approval of my playbook.
Speaker Change: That data showed that in our pivotal clinical trial my place in combination with <unk> hull to disease progression through 12 months.
This was demonstrated by more than a two point improvement in patients receiving my place in <unk> compared to those receiving Midwest stat alone.
Speaker Change: These data were assessed using the re scored four domain NPC clinical severity scale, which is the clinically relevant endpoint for evaluating treatment effect on MPC disease progression.
Speaker Change: Additionally, 41 patients from the pivotal trial were then enrolled into the open label extension study, where 34 of the 41 were treated with my play for for more than two years and 17 of those continued treatment with might play for more than five years, demonstrating its long term value.
Speaker Change: Adrian stated on our September conference call that a one point difference on this scale could mean the difference in one's ability to feed oneself versus the need for a caregiver or.
Speaker Change: Or could mean the difference between walking on one zone versus the need for a wheelchair.
Speaker Change: To reiterate in our study my pleasure and made this that combination so more than two point difference through the 12 month pivotal trial.
Speaker Change: It also showed that might playful was well tolerated compared to placebo and adverse events were generally mild to moderate severity and very few led to withdrawal of treatment.
Speaker Change: Wrapping up with my place. So we're pleased with the early indicators of the launch progress and look forward to sharing more details in future calls.
Speaker Change: Turning to all through the as Neil mentioned, we have refined our strategy to raise patient awareness and improved payer pull through.
So we have patients continuing on all prove out.
Speaker Change: We saw new enrollments decreased to three in the third quarter from 9% in the second quarter and revenue was de Minimis.
Speaker Change: We know that certain patients struggle to remain compliant on therapy and there is a need to raise awareness of the symptoms of UCD and to treat this indication persistently.
Speaker Change: Another dynamic of the UC market is that most patients are on nitrogen scavengers and receive annual paid authorizations.
Speaker Change: Prior authorizations are being reviewed by payers as we approach the end of the year and the market leader is increasingly being moved to the exclusion list many commercial insurance plans.
Speaker Change: Our market access for <unk> is currently 76% of covered lives and over the quarter, we've improved our formulary position on a number of accounts to preferred as payers look for differentiated lower cost treatment.
Speaker Change: Also in the quarter, we reassessed the market opportunity considering all prove is positioned relative to other approved products.
Speaker Change: The outcome was a data driven shifts to target specific patient segments that will receive the greatest benefit from our proven and face fewer reimbursement hurdles.
We focus our resources and promotional efforts in the third quarter to drive patient identification and to improve pull through with payers.
Speaker Change: Specifically, we're targeting patients seeking greater lifestyle independents, who could benefit from the ammonia control on the go.
Speaker Change: <unk> offers.
Speaker Change: Our field team has been working with clinicians to identify appropriate patients across the various centers of excellence that we call on.
Speaker Change: We've also teamed with the National urea cycle disorders Foundation to highlight the critical importance of recognizing the signs and symptoms of hyper ammonia and the need to treat early and persistently.
Speaker Change: As a reminder, there is a high overlap and clinicians who treat UCD an NPC.
Speaker Change: Our targeted efforts for approval should allow us to maintain a share of voice in the UCD market, while leveraging our field teams and other resources across our commercial portfolio. Two established zebra has a committed partner for those living with rare diseases.
Speaker Change: I'll now turn the call back over to Neil to discuss our pipeline assets Neil.
Neil Macfarlane: Thanks, Josh.
Starting with <unk> 77 start ex methylphenidate for the treatment of idiopathic hypersomnia.
Speaker Change: I would like to remind you the need and opportunity for new treatments for IH.
Speaker Change: This rare sleep disorder affects approximately 37000 people in the U S and is characterized by excessive daytime sleepiness and difficulty waking also known as sleeping Harsha.
Speaker Change: Which is still an unmet need.
Speaker Change: Currently there is only one FDA approved treatment and we believe the profile of <unk> 77, uniquely positioned as a product candidate to address the unmet need.
Speaker Change: At the end of the third quarter, we completed our end of phase II meeting in which the FDA agreed that our proposed phase III design could proceed and indicated that a single pivotal study with appropriate confirmatory evidence will be sufficient to submit an NDA.
Speaker Change: In parallel we completed the business case evaluation for <unk> $10 77 within the context of our strategic plan.
Speaker Change: Based on the analysis, we have concluded that the appropriate path forward to maximize the value of <unk> 77 is to explore strategic alternatives to advance clinical development and future commercialization.
Speaker Change: Furthermore, while the K P $10 77, Narcolepsy program was previously de prioritize to focus on IH regulatory flexibility remains to develop the program for narcolepsy concurrently with IH.
Speaker Change: Turning to slip a law, which is in late stage clinical development for the treatment of vascular eilers danlos syndrome or events.
Speaker Change: There are approximately 7500 people in the U S where there is no approved treatment.
While slipper law was approved in Europe for hypertension is commonly used off label for <unk>.
Speaker Change: As previously reported in the second quarter, our decision to restart recruitment in the discover trial has been welcomed by people suffering from vets soon.
Speaker Change: Since we restarted recruitment in the second quarter, we met our goal of dosing patients in Q3, bringing the total number of patients enrolled at the end of the third quarter to 19.
Speaker Change: We continue to have significant interest from patients and physicians to participate in this decentralized trial and in the future. We intend to report total patient enrollment.
Speaker Change: Additionally, we continue to build on our existing partnerships with patient advocacy groups as well as with major treatment centers and key opinion leaders.
With this progress we are refining the business case to inform the value proposition and we intend to provide an update on our next call in early 2025.
Speaker Change: In summary, I am proud of the collaborative effort of team zebra that delivered the accomplishments in the third quarter and throughout 2024.
Speaker Change: Still theres more work for us to do to realize our mission of becoming a leading rare disease company.
Speaker Change: I'm confident that we can get there with our strong commitment to execute focus and innovate.
Speaker Change: I'll now hand, the call over to La Duane who will provide an update on our financial results with Duane.
Speaker Change: Thank you Neil and good afternoon.
La Duane: While I will provide a detailed financial overview during today's call.
Encourage you to review <unk> quarterly report on Form 10-Q for more detailed information, which we intend to file post market Tomorrow November 13 2024.
La Duane: In the third quarter, we reported net revenue of $3 $7 million, which includes $2 6 million and net reimbursements from the French EAP for <unk> and.
La Duane: And $1 $1 million of royalties and other reimbursements under the <unk> license.
La Duane: As a reminder, for our commercial products <unk> and <unk> approval.
La Duane: We recognized commercial product revenue when shipments are received by the specialty pharmacy.
La Duane: In the third quarter net revenue for our approval was de Minimis and we expect to have our initial shipment for my place.
La Duane: In Q4.
La Duane: Our R&D expenses for the third quarter were $10 9 million, which was an increase of 4% compared to the second quarter of this year.
La Duane: Selling general and administrative expenses were $16 $2 million during the third quarter, representing an increase of 28, 6% from the second quarter of this year.
La Duane: This includes a noncash stock compensation expense of $6 $1 million during the quarter.
La Duane: It's $2 $5 million was performance based vesting upon the approval of my Python.
La Duane: Overall this increase is in line with our projections as we transition to full commercial stage operations.
La Duane: The net loss for the third quarter was $33 $2 million or <unk> 69 per basic and diluted share <unk>.
La Duane: Compared to $10 4 million or <unk> 30 per basic and diluted share in the same quarter a year ago.
La Duane: In the third quarter, we completed an underwritten public offering of more than $10 6 million shares of our common stock at a price of $6 50 per share.
La Duane: The total net proceeds were approximately $64 5 million after deducting underwriting discounts commissions and offering expenses.
La Duane: The proceeds from the offerings support our commercial ankle clinical development operations with an emphasis on the launch activities for our commercial products and pipeline.
As of September 30th total cash cash equivalents and investments were $95 $5 million, which was an increase of $46 2 million compared to June 30 of this year.
La Duane: Use of cash during the period was $18 2 million.
La Duane: Total long term debt was $58 $9 million.
La Duane: Based on our current operating forecast existing resources extend our cash runway into 2027 subject to continued compliance with debt covenants.
La Duane: Our cash runway guidance is based on our current operating plan available cash cash equivalents and investments and includes revenue from <unk> sales reimbursements from the French EAP.
La Duane: Royalties under the <unk> license agreement.
La Duane: And continued investments into our development pipeline programs.
La Duane: It does not include potential proceeds from <unk> sale.
La Duane: As we wind down the year, we are pleased with our strong financial footing and our continued ability to be capital efficient with our operations and deliver value to shareholders.
Speaker Change: As Neil said earlier, we are well on our way to becoming a leading patient focused rare disease Therapeutics company.
Speaker Change: Now we will open the call to questions operator.
Speaker Change: Thank you Mr. Clifton, ladies and gentlemen at this time, if you would like to ask a question. Please press star one and if you do find your question has already been addressed you may remove yourself from the queue by pressing star to once again star one for questions and we will go first this afternoon to Jason Butler of citizen JMP.
Jason Butler: Hi, Thanks for taking the questions and congrats on all the progress in the quarter just a couple from me on my place.
Jason Butler: Can you maybe just give us a sense of what the remaining steps are for patients who have received reimbursement approval to get drug shipped to them is everything.
Jason Butler: I guess maybe from a.
Jason Butler: Manufacturing fill finish perspective as their drug.
Speaker Change: Are you ready have drug in the channel yet just what are the next steps there and then I know, it's early but it seems like you've been able to achieve reimbursement approvals quickly looking forward as you kind of more patients come in for the.
Final that where there are new to drive value and not in the IP, how should we think about the average time to you.
The submitted.
Speaker Change: Prescription for him to reimbursement approval. Thank you.
Speaker Change: Jason. Thank you for the question why don't I address the first part of your question, which was around some.
Speaker Change: On the manufacturing.
Speaker Change: Drug and channel timeframe, which we quoted is eight to 12 weeks post approval.
Speaker Change: We are well on our way in regards to getting the drug in channel and believe that the eight to 12 week timeframe is still is appropriate we're coming up close to the eight week timeframe here at the end of the month and we believe that we.
Speaker Change: Scott got bottles in.
Speaker Change: In boxes, and and are ready to be able to move forward.
Speaker Change: Jay you want to and there are no other hurdles by the way Jason to be able to get the drug in the channel except for just a.
Speaker Change: The logistics, but at this point I'll ask Josh to talk about where we go from the 30% of the 90 patients enrolled in that and.
Josh Schaffer: Moving forward.
Josh Schaffer: Yes.
Speaker Change: Jason just as a reminder, as we said we currently have 90 enrollments and I think your question was specifically around.
Speaker Change: What are some of the reimbursements.
Speaker Change: Steps for patients who are not in the EAP program and of those 90, only 69 are from the AP program, meaning that we do have a handful that have already.
Speaker Change: Been enrolled who are not in the EAP program and have not had prior exposure to might play for us and we're working our way through each of those enrollments kind of independent of whether or not that patient was from an EAP program or naive to Tim My place. So there are no additional hurdles as we see them towards getting.
Speaker Change: Reimbursement, regardless of whether that patient was previously in our on our EAP or not.
Speaker Change: Okay, great. Thanks for taking my questions and congrats again on the progress.
Speaker Change: Thank you we'll go next now to Louise Chen of Cantor.
Louise Chen: Hi, congratulations on all the progress and thanks for taking my question here. So just an additional question here on my place how do we think about modeling the sales in the fourth quarter.
Louise Chen: 24, all 27 of those patients actually getting your product or is that going to be staged.
Louise Chen: Into the following quarter and then for the EU you talked about.
Louise Chen: Extending the product there and what are your next steps with title here in the last question here is on your PRP any sense of the timing and value of what that's worth okay.
Speaker Change: Thanks, Luis I'll see what I can do to get these started in seafood Duane wants to add anything else. There in terms of modeling sales in Q4, as we talked about in our prepared remarks, we plan to have our first <unk>.
Louise Chen: Matt.
Louise Chen:
Louise Chen: Our drug to our specialty pharmacy and have revenue in Q4.
Louise Chen: In regards to the question that came previously from Jason We expect that these 30% of the benefits investigations that have already been approved as of October 31, we will actually get the first shipment of product to patients as soon as the drug hits the specialty pharmacy. So those are done and again thats as of <unk>.
Louise Chen: Over 31, so we expect that number to be higher as we work through the benefits investigation process for the remaining 90 enrolled as of October and the additional enrollments that we will get in the next month and moving forward.
Louise Chen: In regards to the EU timing it is a priority for us as I mentioned in our in the prepared remarks, we are gone through our extensive portfolio of review.
Louise Chen: We have made some very difficult decisions to reallocate resources towards those areas that are we feel are compelling to drive value not only for patients, but for our shareholders and in the EU as a priority project for us.
Louise Chen: That being said, we can't give you timing at this point because we're in the process of putting the data.
Louise Chen: That we received that the robust package from the FDA and then now bringing that data to.
Louise Chen: The dialogue with the EMEA, so you'll have to stay tuned on the timing, we expect to give you that.
Louise Chen: And the first of the year.
Louise Chen: And then on the <unk> side Youre question is timely we have seen a number of <unk> that are now transacted at a much higher price than the previous.
Louise Chen: Approximately $100 million, we've now seen two print in the 150 range.
Louise Chen: And we are actively monitoring the prs market and hope to be able to monetize the PRP and drive non dilutive capital into the balance sheet.
Speaker Change: Do you have anything else you want to add.
Speaker Change: I would just say that the recent offering can be completed during this quarter provided the flexibility we have now to watch the market and find the right time to monetize.
Speaker Change: Okay.
Speaker Change: Thank you.
Speaker Change: Okay.
Speaker Change: Thank you the next now to Sami Corwin at William Blair.
Sami Corwin: Hey, guys. Congrats on the progress this quarter and thank you for taking our question.
Sami Corwin: I was wondering if you could provide more detail on the status of the remaining 60 patients that have started the enrollment form.
Sami Corwin: Are you just still working through those forums has there been any denials.
And then could you provide any stats as to how many of those.
Speaker Change: 90 patient comes limited to start enrollment forms that are that had already been on neglect that thank you.
Speaker Change: I'll ask Josh to talk about it but I think it's an important perspective Sami that the 90 enrollments were as of October 31. So that's not a final number for the quarter as of today. That's as of October 31, where we put a cutoff I'll ask Josh to talk about the specifics.
Speaker Change: To that point.
Josh Schaffer: As of October 31, we had been able to process and complete the benefits investigation process for a third or 30 of those of those 90.
Josh Schaffer: And we.
We continue to just work through those enrollments and and we will continue to do so our patient services team is working very diligently to make sure that all of those patients have the opportunity to.
Josh Schaffer: Hopefully get it approved.
Shipment of my place.
Josh Schaffer: Your other question was how many of those have are on <unk>.
Speaker Change: We know that the majority of the patients on our EAP about 80% to 85% of them are on <unk> and as they're going through the enrollment forum.
Speaker Change: We're assuming that the same number of those patients are on <unk>.
Speaker Change: Great. Thank you and to clarify you haven't seen anything yet.
Speaker Change: Jamie I'll take that question you see denials and do you also see the benefits investigation process.
Speaker Change: Everything we do that being said, we've been able to process, 30% or approximately 30 patients so far and getting them to approval status at some of those patients were denied originally and then gotten through to approval status. So we're working through the same specialty pharmacy benefits investigation process that any specialty pharmacy product with <unk>.
Speaker Change: Go through we have a high level of confidence, though that 30% and a very short period of time of those patients have been approved that will be successful and the remaining 90 and again that was through October 31, we've continued to see enrollments through but we wanted to be able to provide an early.
Speaker Change: Number four for the street at this point through October.
Speaker Change: Got it I appreciate the clarity.
Thank you we'll go next now to Eddie Hickman at Guggenheim Securities.
Eddie Hickman: Hey, good afternoon, and thanks for taking my questions. Congrats on the nice progress. So far can you talk about maybe looked at us in the U S and what steps physicians currently have to take to get an MPC patient on that product and reimbursed and maybe compare that to whats required for an improved drive now like my Plaza what are the main hurdles to broadening that coverage and then given your label.
Is increasing mega lift that used a leading indicator for my play for US are we seeing any spikes in prescriptions or payer claims that coincide with your ongoing launch thanks.
Speaker Change: Thanks, Eddie I think Youre asking me to answer some questions. So we don't have very good answers to your first question was in regards to the <unk> approval process.
Speaker Change: If I understood it correctly I cant really opine on how physicians get megawatts that are what I can tell you, though is that those patients.
Speaker Change: So far in our EAP about 80% of those patients were on megawatts stat.
Speaker Change: As you can see the 83 patients we left.
Speaker Change: Less.
EAP when it was closed when we got approval 69 of those patients have already been.
<unk> had an enrollment form in our enrollment form actually states.
Speaker Change: Here's the dose of my <unk> have you been on megawatts that are on <unk>, and then where are you in our EAP program. Those patients who have had <unk> approved so far have also gotten my apply for approved and our early experience in the 30% of those patient enrollment forms out of that process. So far so I can't comment on specifics.
What the processes for <unk>, but I can tell you that those patients that were on or have been on megawatts that are also getting approved through the benefits investigation process.
Speaker Change: And we've seen success, so far and this is very early in <unk> and.
Speaker Change: And we're pretty excited about the success we've had so far.
Speaker Change: The next question you asked me was in regards to US if there is a increasing.
Speaker Change: Increasing use of megawatts that use and if that's a leading indicator again <unk> is not approved in the U S for MPC and its uses are approved for <unk> disease type one.
Speaker Change: And it's highly generic size there are a number of generic players out there. So I think it's hard for us to say whether or not the total megawatts that utilization is actually representative of anemia exceed disease type patient.
Speaker Change: I Hope I tried my best to answer your questions here, but tell me. If you have got to have a follow up.
Speaker Change: Yes, I guess I guess, a simpler way to ask maybe as of the 20% of patients that are not Omega Scott how many of them aren't on it because they can't be on it or how many are on it because they just arent on it yet because I guess I'm wondering if we'll see that number increase at all now that the label includes that language.
Speaker Change: Yes, so feedback from physicians and I think the experience that <unk> had as most patients have been on <unk> at one point in there.
Speaker Change: Disease journey and what we've seen is is that it really is dependent on side effects as well as payer approvals. So access for <unk> again has been about 80% of those patients. We've had in our EAP that have been able to get it but tolerability is also another one of those areas. So.
I hope that that helps to answer your question.
Speaker Change: Yes, I appreciate it thank you.
Speaker Change: Thank you and just a quick reminder, ladies and gentlemen star one. Please for questions. Today. We'll go next now to <unk> Kulkarni at Canaccord.
Speaker Change: Good afternoon, thanks for taking our questions.
Speaker Change: On <unk>, you said that 69 of the patients that have a prescription enrollment funds.
Speaker Change: In the expanded access program, but could you provide that ratio for the 30% of the 90 prescriptions that are approved for reimbursement and what are you seeing in terms of competition from the jumbo product Thats also approved for MPC.
Speaker Change: Yes, thanks, so much.
Speaker Change: So we have not really parse the.
<unk> paid authorizations were going through them one by one.
Speaker Change: Of course, we're very excited to see that as many of the EAP patients come through is as we have with nearly 70 of the 83.
Speaker Change: But we are not we're not having any sort of.
Speaker Change: Differential processing of those whether they come through the EAP or whether they're new to my place. So we're not really in a position to give those those those numbers.
Speaker Change: And when it.
Speaker Change: And in regards to your question around what we're seeing with the <unk>.
<unk>.
Speaker Change: Again, we're not commenting on what.
Speaker Change: What is going on with <unk> I think what is important is to really reiterate the differentiated data around my place in.
Speaker Change: And the fact that we are the only product out there with.
Data that has demonstrated the halting of disease progression through 12 months.
Speaker Change: Demonstrated a two point improvement on a score which is used.
To really measure the disease modification.
Speaker Change: The re scored four domain Neiman tic severity score.
Speaker Change: And we've got long term data that shows that patients have seen clinical benefits up to five years through our EAP. An open label study. So that's really what we're focused on is our data and <unk> and the great success in.
Speaker Change: The.
Speaker Change: What we're seeing already with the enrollments that we've we've seen today.
Speaker Change: Got it and then 77 is there a preference for the types of strategic alternatives you might be contemplating.
Speaker Change: <unk> I'll take that question.
Speaker Change: Where we looked at the ability for us to drive value with a single pivotal trial the ability to also in parallel look at our narcolepsy indication.
Speaker Change: And actually through the data that we got through the phase II. It really allowed us to then inform this business case right the opportunity for us to know what would it take us to succeed to commercialize this program and the infrastructure needs that are necessary to be able to successfully commercialize a much broader footprint with a much larger organization.
Speaker Change: With a differentiated with success of differentiated product that hits, an unmet need is a very different.
Speaker Change: Call point to what we are specializing in and really leaning into today, which is a very targeted call point for <unk> for patients in this rare genetic and metabolic area as well as for physicians that are in this.
Speaker Change: In these centers of excellence, so I can't say what type of strategic alternatives, we are lean to one versus another but what we absolutely see is is that there is value to unlocked by working.
Speaker Change: Where the with partners to try and figure out where the best fits got.
Speaker Change: Got it and then my last question is on <unk>, given there aren't too many alternatives even put products in China.
Speaker Change: <unk>.
Speaker Change: How would you characterize the demand from a patient perspective.
Speaker Change: Okay.
Speaker Change: So this has been one of the big surprises in 2024 for us.
Speaker Change: <unk>.
Speaker Change: The excitement around the ability to screen patients and what we'd heard prior to <unk>.
Speaker Change: <unk> and restarting the trial, we couldnt really tell whether or not that would translate itself into enrollments and I think what we've seen is is that with no approved treatments for events in the U S.
Speaker Change: And that prevalence, we see at about 7500 patients.
Speaker Change: For us it is an opportunity to reestablish enrollment and see if we can actually now target what.
Speaker Change: Enrollment timeframe, we might be looking at to be able to then think about our interim analysis and our final analysis and trying to.
Speaker Change: To deliver then that commercially this is an important data point for us in regards to restarting the trial getting our first patient enrolled in queues in the first few patients enrolled in Q3 now it is really up for us in Q4 to understand what that ramp looks like and then early next year be able to get back to the market to let them know what we believe the time.
Speaker Change: Frame is and how we how we move towards unlocking the value but early.
Yes.
Speaker Change: Our early indicators are strong that we've seen these patients and and then the desire to get into the trial quite high now we're working through that.
Speaker Change: To refine the case.
<unk>.
Speaker Change: Thank you we take our next question now from Oren <unk> at H C. Wainwright.
Speaker Change: Yeah.
Speaker Change: I appreciate that.
Speaker Change: You guys are adding.
Speaker Change: Diagnostic to wear my patients are coming from whether they're in the EAP or outside but I'm really curious about what your view is on the level of awareness and excitement.
Speaker Change: Outside of the EAP program, obviously thats the majority of treated patients out there. So if that is already high that would be really positive indicators I am curious what you can add there.
Speaker Change: And just with regards to the.
Speaker Change: Yes.
Speaker Change: Ram obviously, youre not giving guidance now I'm curious if you have any intention.
Speaker Change: You hadn't been launched I just have to ask do you have any intentions given the visibility you do have in the limited population.
Speaker Change: Trade promotion to get potentially any guidance in early 2025.
Speaker Change: Our next year's sales and I do have a follow up thanks.
Speaker Change: Yeah.
Speaker Change: Great. Thanks, Oren there is no intention for us.
Speaker Change: In the early phases of a launch to provide you with guidance on.
Speaker Change: Where we are but it is important where we see the product going into the next few quarters. It is important though that we reiterate that we believe there are about 900 patients in the U S and about 300 to 300 of those patients.
Speaker Change: <unk> had been identified and diagnosed.
Speaker Change: This ramp that we're on right now I do believe.
Speaker Change: Is it is it.
Speaker Change: Good metric for us to understand how we can continue to go after not only those patients who have been diagnosed but also.
Speaker Change: The greater than the 350 patients that get us to that 900 patient number a lot of times in these rare diseases you see that once you have a therapy available.
Speaker Change: A little more on the top of mind, the physicians as they work through their differential diagnoses for us to be able to educate and all the work that we will do to be able to raise the awareness of MPC, but.
Speaker Change: And then obviously attract that does more than 300 to 350 patients that can benefit from from Piper I'll ask Josh to talk a little bit about the ramp in a little bit about the awareness.
Speaker Change: And what.
Josh Schaffer: He has been seen in the marketplace yeah. So.
Josh Schaffer: As you know we've been engaged with the patient advocacy community for some time and I think that long standing relationship is really helping too.
Josh Schaffer: Heightened the awareness of my place.
Josh Schaffer: In addition, our sales team has been out in the market interacting with many of these these prescribers through their their connections.
Josh Schaffer: And calling on them for for <unk> one.
Josh Schaffer: One of the one of the benefits and strategic rationale for having these two products is that there is a high degree of overlap in the.
Josh Schaffer: In the call points and the prescribing physicians and so we're and this is quite high with two patient advocacy and our MSL activities with thought leaders as well as.
All of the.
Josh Schaffer: The work that we're doing in the offices to really build awareness not only for approval.
Josh Schaffer: Excuse me for my place, but also just around <unk>.
Josh Schaffer: Disease awareness to help identify new patients who might be diagnosed with mtc.
Speaker Change: Alright, and did I hear or did I understand you correctly that this increase in SG&A already this quarter is that a pretty good indication of a fully loaded.
Right size investment rate for this launch or is that going to keep ramping up into the fourth quarter and.
Speaker Change: With this launch.
Speaker Change: Hello, Duane you want to answer that one.
Duane: Yes. Thank you alright, I think that it does reflect our full commercial activities and patient services being in place.
Duane: But it is important to note too that it included non cash stock comp stock compensation of $6 1 million.
Which included a onetime best thing of about $2 $5 million, which was performance based on the approval of my Piper So that inflated the number just a little bit there so yes.
Duane: With that adjustment it does reflect our full infrastructure in place.
Speaker Change: Okay and last one I'll prove I appreciate you guys being transparent.
Speaker Change: Transparent on that.
Are you able to tell us how many patients are actually getting therapy, regardless of paid or not just in terms of just how many.
Speaker Change: People are out there getting the drug and are we not seeing any revenue because there are patients that are just not reimbursed.
Speaker Change: Or is that possibly that youre working through some inherited inventory and just I guess to keep it real straightforward. Obviously most of US are focused on my cliff.
Speaker Change: We just until further notice assume that thats going to may.
Speaker Change: De minimis until proven otherwise on the Oklahoma Frank.
Speaker Change: Yeah.
Hey, Lauren can you ask that last part of the question and I'm going to pass it off to Josh but can you ask the last part of the question we lost that last one.
Oh, sorry.
Speaker Change: Yes, just to be conservative do you think until proven otherwise that you can really generate awareness in our footprint with all proven that we should just assume that that's going to remain.
Speaker Change: So to speak until proven otherwise.
Speaker Change: Okay.
Yeah. So in answer to your first question, we do have a number of patients who are continuing on active treatment, but we're not.
Speaker Change: Position now to give you those numbers.
I would like to remind you the way that we book revenue is when shipments are received at the SP and so it isn't necessarily a direct correlation between the number of patients who are receiving ongoing therapy and when we realize revenue just given kind of the lumpiness of that.
Speaker Change: And we remain.
Speaker Change: Committed to <unk> as we talked about in our prepared remarks, we've really refined our strategy to go after those patients you're going to receive the most benefit while at the same time working really hard to improve our formulary positions with as many commercial plans as possible.
Speaker Change: Continue to drive revenue and drive.
A man there and make sure that as many patients can benefit.
Speaker Change: And I think that that would.
Speaker Change: That's the way, we're thinking about it and we'll continue to give you updates in future quarters.
Speaker Change: Alright, I appreciate it and good luck with the launch.
Speaker Change: Thank you.
Speaker Change: Thank you and gentlemen, it appears we have no further questions. This afternoon, Mr. Macfarlane and I'd like to turn things back to you Sir for any closing comments.
Neil Macfarlane: Thank you.
Neil Macfarlane: I'd like to thank all of our stakeholders for helping us get to this point and for your continued commitment we.
Neil Macfarlane: We're at the beginning of our next phase of growth and our team is ready to execute focus and innovate to create value for patients and shareholders and we're looking forward to updating you on our next call. Thank you very much have a good day.
Speaker Change: Thank you and again, everyone that does conclude today's conference call again, thanks, so much for joining us and we wish you all a great remainder of your day Goodbye.
Neil Macfarlane: Okay.
Neil Macfarlane: Okay.
Neil Macfarlane: Thanks, Bob.
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