Q1 2025 Syndax Pharmaceuticals Inc Earnings Call
Speaker Change: Good day everyone and welcome to the Syndax First Quarter 2025 earnings conference call. Today's call is being recorded. If you would like to ask a question following the company's prepared remarks, please press star 5 during the call. At this time, I would like to turn the call over to Sharon Clary, head of investor relations at Syndax Pharmaceuticals.
Speaker Change: Great, thank you, operator. Welcome, and thank you all for joining us today for a review of Syndax's first quarter, 2025 Financial and Operating Results. I'm Sharon Clare and with me this afternoon to provide an update on the company's progress and discuss financial results.
Speaker Change: Arm Michael Metzger, Chief Executive Officer, Dr. Neil Gallagher, President and Head of R&D, Steve Closter, Chief Financial Officer.
Speaker Change: Also joining us on the call today for the question and answer session are Dr. Peter Ordentlich, Chief Scientific Officer, Dr. Anjali Ganguli
Chief Strategy Officer
Speaker Change: Who's called the company by a slide deck that's posted on the investor page at a company's website?
Speaker Change: Can now turn to a forward-looking statement on slide two
Speaker Change: Before we begin, I'd like to remind you that any statements made during this call that are not historical are considered to be four-looking statements within the meaning of the Private Security's litigation reform act of 1995
Speaker Change: Actual results may differ materially from those indicated by the statements, as a result of various important factors, including those discussed in the risk factor section in the company's most recent quarterly report on Form 10-Q , as well as other reports filed with the SEC. Thank you.
Speaker Change: Any four-looking statements made represent our views as of today, May 5th, 2025, only. A replay of this call will be available on the company's website www.syndax.com, following its completion.
Speaker Change: With that, I am pleased to turn the call over to Michael Metzger, Chief Executive Officer of Syndex.
Speaker Change: Thank you Sharon, good afternoon everyone and thank you for joining us today, starting with slides three
Speaker Change: and pleased to report that Syndax delivered another outstanding quarter of execution as a commercial company with two first and best-in-class medicines on the market and a robust pipeline of clinical development programs designed to unlock the multi-billion dollar opportunities for both of our medicines.
Starting with the progress on the commercial front.
Speaker Change: The launches of Revue Forage and Nictimbo are both off to very strong starts.
Speaker Change: In the first quarter of 2025, Syndax recorded $20 million in Revy Forage Net Revenue from the first full quarter of the launch, and our partner, Insight, recorded $13.6 million in net revenue from the first two months of our joint launch of Nick Timvov
Speaker Change: Later on the call, Keith will break down the numbers we were reporting for our 50% share of the commercial Nick Timbo contribution
Speaker Change: This combined $34 million in net sales from Revy Forge and Nictimbo reflects the high unmet need, the compelling profiles of our medicine, and outstanding execution across our entire organization.
Speaker Change: These results also underscore the significant commercial opportunities we have with both our products and the benefits of being first to market. Importantly, we are well funded to deliver on the exciting opportunities in front of us with 602.1 million in cash and equivalence as of March 31st.
Speaker Change: In addition to the progress we have made with the two product launches, we have also continued to advance our position as a pioneer in men in inhibition with the initiation of the first quarter of evolved two, the first pivotal front line trial of a men in inhibitor [inaudible]
Speaker Change: The evolved two trial of revumantive in combination with benediclacks and asus cytodine is enrolling newly diagnosed mutant NPM1 and KMT2A rearranged AML patients who are unfit for intensive chemotherapy, a patient population with a high unmet medical need.
Speaker Change: In parallel with ongoing trial recruitment, we will be amending the protocol and analysis plan to include complete remission and overall survival as dual primary
We've also made other major progress advancing our pipeline.
Speaker Change: In April , we completed the submission of a supplemental new drug application, or SNDA, to the FDA, seeking priority review for the approval of Revy Forge for the treatment of relapse or refractory mutant NPM-1 AML.
Speaker Change: If priority review is granted, it would provide a target FDA review period of six months from the date of submission of the SNDA, a two month advantage compared to the timeline with an NDA.
Speaker Change: We see it as a significant advantage that our submission builds off of our Rev. Forge NDA which was recently approved by the FDA in November of 2024.
Speaker Change: Like our successful NDA in relapse or refractory acute leukemia with a K&T2A translocate location, the SNDA will be reviewed under the FDA's real-time oncology review or RTOR program, which aims to ensure that safe and effective treatments are available to patients as early as possible.
Speaker Change: The program provides for frequent, iterative communication with the FDA and a rolling submission which we recently completed.
Speaker Change: Since 2018, when the program was created, the SNDA applications reviewed under our tour have had a strong track record of approval. As expected, thus far we have continued to experience robust and timely engagement with the FDA on our SNDA filing and programs overall.
Speaker Change: Before I hand the call over to the team to provide more color on our recent progress, I want to take a moment on slide four to reflect in the journey that brought us to this point.
Speaker Change: From the start, Syndax has been focused on generating clinical data that validates promising scientific ideas and research, and this is exactly what we have done with both Revy Forge and Nectimbo.
Speaker Change: Looking at the history of Revy Forges as an example, in 2020, Syndax was the first to deliver clinical data that validated the therapeutic benefit of men in inhibition. Since then we have achieved many other important firsts for the field and most importantly, patients.
Speaker Change: We were the first to deliver positive pivotal data in relapse or refractory acute leukemia patients .
Speaker Change: with K&T2A rearrangements, or MPM1 mutations, the first to achieve FDA approval for a men inhibitor, the first to have a men inhibitor listed in the AML and ALL treatment guidelines, and the first to start a pivotal frontline trial of a men inhibitor. [inaudible]
Speaker Change: And today, here we stand, having just delivered the largest full quarter of sales for any targeted AML drug in the US to date.
Speaker Change: We look forward to building off this momentum and achieving additional important first as we continue to rapidly advance Revy Forge our best in class medicine.
Speaker Change: In the relative near term, we expect that Revy Forge will be the first men in inhibitor, including clinical guidelines for the treatment of relapse or refractory mutant NPM-1 AML [inaudible]
Speaker Change: Today we announce that our manuscript detailing our positive pivotal data in mutant MPM1 patients has been accepted by a high impact journal and we expect this paper will publish imminently.
Speaker Change: As soon as the paper is published, we intend to submit the paper to the NCCN Guidelines for consideration.
Speaker Change: Further, with our recent SNDA submission building off our previously approved NDA, and the benefits afforded by our tour, we expect that Revy Forge will be the first drug to receive FDA approval in Relapse or Refractory Mutant NPM1 AMO.
Speaker Change: Looking to the future, Revy Forge is well positioned to be the first men and inhibitor approved in the front line setting.
Speaker Change: With the evolved two trial now underway in close partnership with the Hovan Network, a leading clinical trial group with an outstanding track record of successfully advancing transformative therapies for blood cancers, we are confident that we will continue to lead the development of this new therapeutic class that holds such tremendous promise for patients.
Speaker Change: With that, I will now turn the call over to Steve to discuss our commercial progress in more detail. Steve!
Steve: Thank you, Michael. I'm pleased to report that the launches of Revy Forge in Nictimvo are both off to fantastic starts of all the early launch metrics either meeting or exceeding our high expectations
Starting with Revy Forge on Slide 5. [inaudible]
Steve: As Michael stated, in the first quarter of 2025, the first full quarter of the launch, we delivered an impressive $20 million in Revy Forge and that revenue. These early results bolster our confidence in the number of acute leukemia patients with K&T-2A translocations, and highlight this significant commercial opportunity that we have with Revy Forge. [inaudible]
Steve: The rapid adoption reflects multiple factors, such as major unmet patient need, the strength of our clinical date and product profile, favorable formula recovered in the high caliber of our customer facing team.
Steve: Together, these factors are driving the expansion of prescribing, as well as strong numbers of new patient starts and robust refill rates with what we would expect to see roughly 5 months into the launch. [inaudible]
Diving into a bit more detail on slide six. [inaudible]
Steve: Position feedback and review forages, clinical profile, any overall ease of access to the medicine has been very favorable.
Steve: This positive experience has reflected in the expanding breadth and depth of Revy Forge prescribing.
Steve: with 44% of our high priority tier one and tier two accounts, ordering as of the end of March. That's up from one third of accounts at the end of February , and continuing to grow into the second quarter. That's up from the end of February , and continuing to grow into the third quarter.
Steve: Now these tier one and tier two accounts are the centers of excellence and the medium to large academic institutions which represent two thirds of the patient opportunity [inaudible]
Steve: Compared to benchmarks, we have achieved orders at more top tier accounts over a shorter time frame than all other targeted ANL products.
Steve: While we have made outstanding progress in these priority accounts in the first few months of launch, there is still significant room for further growth and upside as the remaining roughly 55% of high priority accounts identified the right patients and initiate treatment.
Steve: I will also note that we are calling on an entire universe of 2,000 accounts, and accounts beyond Tier 1 and Tier 2 are ordering review for each, including academic centers of all sizes, as well as community practices. [inaudible]
Steve: Notably, among all the accounts that have ordered, two-thirds have ordered multiple times as of the end of March.
Steve: Physicians are prescribing wherever you forge to a mix of K&T2AR patients, Witcher Flux are broad label, encompassing adults and children one year and older, with any lineage of relapse or refractory Achilles leukemia, with a K&T2A translocation, including ALL, ALL, and MPAL.
Steve: We are anecdotally hearing a review forage being prescribed to canty-to-wear patients across the treatment continuum including patients experiencing their first relapse as well as those with very advanced disease
Steve: All indicators suggest that the use of revenue forage will move towards first relapse and rapidly become the standard of care for the population within our current label.
Steve: Encouraging, we're also hearing of several patients going on to receive stem cell transplants after getting into remission with Revy Forge, which is the treatment goal for these patients as transplants are the only potentially curative treatment option
Steve: We expect, based on feedback from physicians, that most of these patients, if not all, will ultimately go back and review for Post and Graphmon [inaudible]
Steve: As the launch progresses and the relevant data sets mature, we look forward to providing more color and metrics and revenue for usage, usage including in the proposed transplant setting.
Steve: Turning to market access. Formular coverage of Revy Forge continues to build very nicely due to the extensive work our market access and medical teams have done educating payers and the rapid inclusion of the product in NCCM guidelines.
Steve: As of the end of March, formal coverage policies were in place for approximately 72% of all managed care lives, which includes commercial, Medicare, Medicaid lives up from 53% at the end of February .
Steve: We have also continued to achieve high payer approval rates with the vast majority of prescriptions reimbursed. We are in a strong position and very pleased with how favourably our formula recovered is tracking compared to the launches of other AML therapies.
Steve: We also continue to see the benefits of the limited distribution model we have established to provide patients and clinicians with the best experience possible.
Steve: Thanks to the infrastructure we have built combined with the deep experience of our team and all the work we've done with payers patients are getting approved for coverage quickly and many patients receive their medication within a few days of receiving a prescription well ahead of industry benchmarks .
Steve: We and our trade partners are keenly aware that these patients are facing a life-threatening diagnosis and we're deeply committed to ensuring that every appropriate patient can quickly gain access to review forage. [inaudible]
Moving to slide seven. [inaudible]
Steve: Our current review forage indication provides us with the opportunity to target an estimated 2,000 patients in the U.S. with relapse or refractory leukemia with a K-2H translocation. A population which represents a $750 million market opportunity.
Steve: We have high conviction that we'll be able to penetrate a major portion of this market and possibly expand the opportunity given the widespread testing for KT2A rearrangements, strong enthusiasm for review forage, and the absence of any other targeted therapies for these patients today or in the foreseeable future.
Steve: Importantly, based on feedback from clinicians and other data points, we believe the majority of revenue revenue date is from on label use that we have just started to penetrate the CANTY-2A opportunity with a lot of room to still grow.
Steve: Additionally, we're excited about the opportunity for significant future growth, including with the potential inclusion of review forged in the clinical guidelines for the treatment of relapse or refractory mutant NPM1 AML, followed by the expected approval of our SNDA in this population. [inaudible]
Steve: We have commercial preparations well underway for anticipated expansion into the second population, a launch that will be boosted by a significant head start into the market with the KT2A approval and physicians rapidly growing familiarity with Revy Forge
Steve: To ensure that Revy Forge is positioned for near-term and long-term success, we're laser focused on strong execution against our strategic launch imperatives they originally outlined on our Revy Forge approval call in November .
Steve: Each day we are focused on ensuring that we leave no appropriate patient behind, engaging all key stakeholders and delivering a best-in-class experience for patients and clinicians
Steve: Our strategy is designed to first and foremost meet the needs of patients and secondly to build long-term, competitive immunity ahead of
Moving to Slide 8
Steve: In late January of 2025, we, an Insight, Launching a Tempo in the U.S. for patients with chronic graft versus host disease or GVHD, after failure of at least two prior lines of systemic
Steve: From the first two months of the launch, Insider reported an Ximpo net product revenue of $13.6 million. This is a very encouraging result which highlights this significant and sometimes underappreciated commercial opportunity that we have with Ximpo. [inaudible]
Steve: The robust uptake was driven by multiple factors, including high unmet need, a unique product profile, very strong product awareness, the benefit of co-commercializing with the leader in GVHD and the commercial synergies that Nectimvo has with both companies, product portfolios. [inaudible]
Steve: Turning to Slide 9 and some of the early launch metrics.
Steve: We're pleased to report that an estimated more than 1,250 infusions of Nictimvo have been administered a year to date an approximately 95% of top accounts and more than 70% of all bone marrow transplant centers have ordered as of the end of March.
Steve: The rapid progress we have made in these accounts highlights the advantages of launching into an established market with well-identified patients, as well as the benefits of insights longstanding relationships with the roughly 200 accounts in the US that perform stem cell transplants.
Steve: At this early point in the launch, we are hearing of some accounts using Nintendo in the third line setting, but typically they are fourth line plus patients who have already cycled through several treatments and are in need of a new option.
Steve: Feedback from physicians has been very positive regarding the responses they are seeing in their patients, and we expect this first-hand experience along with their educational efforts will drive more usage in the third-line setting [inaudible]
Steve: On the market access front, payers recognize the value of Nintendo, and the drug is being reimbursed. Additionally, a permanent J-code was assigned by CMS Effect of April 1, an important milestone that helps facilitate efficient billing and reimbursement for IV products.
Moving to slide 10
Steve: Our current Attimbo indication provides us with the opportunity to target the 6500 Chronic GVHD patients in the US who require three or more lines of therapy which represents a one and a half to $2 billion total addressable market.
Steve: are the dressing the needs of this patient population and is an attractive commercial opportunity. For instance, in the three-year since the launch of Reserock, another drug indicated for the same line of treatment as Nick Timbo, net sales continue to grow and suggest that the drug is now annualizing, but over $500 million in US sales.
Steve: Together with insight, we are making excellent progress executing on our strategic launch imperatives for Nintendo and are very excited to continue advancing the launch of this much needed new option for patients suffering from this debilitating and sometimes life-threatening disease.
Steve: With that, I'll hand the call over to Neil to discuss the progress we're making across our development programs, Neil. Thanks, Steve. It's a pleasure to be able to provide you with an update on development today. Turning to slide 11 and our pipeline, we are aggressively advancing a thoughtful and robust clinical development plan that aims to bring both our assets into additional important populations.
Neil: Joining with the latest updates on the REVY Menoprogram, we have multiple ongoing and planned technical trials across the acute leukemia treatment continuum, including in combination with standards of care therapies in the frontline setting.
Speaker Change: Our combination trials build off the positive results from the beat AML trial being conducted by the leukemia and the phoma society, and the safe trial being conducted by investigators from MD Anderson Cancer Center.
Speaker Change: Repleased to have recently initiated a valve two, the pivotal frontline trial of revue menub and combination with synetic laxonase aside of them, in newly diagnosed patients with mutant MPM1, or KMT2A rearranged AML, who are ineligible or unfit to receive intensive chemotherapy. . . . . . .
Speaker Change: Evolved to is a safe, three randomized double blind placebo controlled trial that we expect will enroll approximately 415 patients [inaudible]
Speaker Change: As Michael mentioned, in parallel with ongoing trial recruitment, we will be amending the evolves to protocol and analysis plan to include complete remission and overall survival as dual primary endpoints to support potential US accelerated approval and full approval respectively. Thank you very much.
Speaker Change: While the trial is open to both MPM1 and came to 2A patients, the primary efficacy analysis will be based on the MPM1 population. This is a population that is more commonly ineligible for intensive chemotherapy due to advanced age or other comorbidities.
Speaker Change: Unlike the came-to-twey population which tends to be younger and eligible are fit for intensive keen of therapy [inaudible]
Speaker Change: We are excited to partner with the Hova Network and this trial, considering their extensive network of leading research institutions across the EU and globally.
Speaker Change: Turning to the newly diagnosed fit population, we're planning to initiate two randomized placebo control trials of revumative and combination with intensive chemotherapy followed by a maintenance phase, one trial for patients with an MPM-1 mutation and one for patients with came-to-toe rearrangements.
Speaker Change: We believe this is most efficient approach to establish efficacy across these two populations with different expected long-term outcomes and different current treatment goals. We've named these trials reveal ND or newly diagnosed trials.
Speaker Change: In line with our standard approach we will share more details on these studies once we have posted them on ClinicalTrials.gov which we anticipate during later this year.
Speaker Change: In the fifth frontline setting, we also look forward to reporting data from a phase one trial of Revented in combination with intensive chemotherapy in the fourth quarter of this year.
Speaker Change: With our frontline strategy, we are well positioned to generate comprehensive data that could support accelerated approvals, full approvals, and transform the treatment paradigm for the 40% of AML patients with MPN1 mutations are came to to a rearrangements.
Turning to the bottom half of the slide and exit to Lamand.
Insight: We and Insight are very excited about accidental nubs potential in earlier lines of chronic HD and other fibiotic diseases starting with ITF.
Insight: Together, we are advancing several important trials, including two ongoing trials of exotilumab in combination with standards of care and newly diagnosed client GVHT patients.
Insight: One is a phase two trial studying accidental amount of incommunation with Rox Lippner, and the other is a phase three placebo controlled registration directed trial investigating accidental amount incommination with steroids.
Insight: Beyond chronic GVHD, we have an ongoing phase two placebo control trial called Max Bar, which is investigating acetylomob in IPF.
Insight: Enrollment is proceeding well and we expect to complete enrollment this year with top line data anticipated in the second half of 2026.
Insight: We inter-clinical collaborators are very encouraged by the three clinical evidence which shows a significant reduction in markers of lung fibrosis with CSF-1R inhibition. Results which are bolstered by the positive results we observed in patients with BOS or bronchialitis obliterant syndrome in the Yigave-2-1 trial.
Keith: With that, I will hand the call over to Keith to discuss our fundamentals. [inaudible]
Keith: For the first quarter of 2025, we reported a revenue-forged net revenue of $20 million in dollars.
Keith: The vast majority of the revenue is driven by real demand with approximately just two to three weeks of inventory in the channel at the end of the quarter consistent with levels at December 31st and consistent with what is typical for a specialty medicine.
Keith: We expect channel inventory levels to remain at two to three weeks moving forward.
Keith: Moving to Nick Timbo, for the first quarter of 2025, Nick Timbo achieved $13.6 million in that revenue.
Keith: As a reminder, insight records net revenue and Syndax report 50% of the Nektivo net commercial property loss, which is defined as net revenue less the cost of sales and commercial expenses.
For this quarter, a partial quarter, [inaudible]
Keith: We encourage a report that our share of the net commercial loss is only $200,000, as you can see recorded in a line item called collaboration loss.
Keith: Given the strong results from the first two months of launch, we expect that Nick Tinville will quickly convert to a positive revenue contribution to a positive revenue contribution.
Learn more at www.plastics-car.com
Keith: In periods where there is an activity on the commercial profit, you will see our 50% share in the revenue section of our income statement under collaboration revenue.
Keith: I will also note that any future milestone revenue we receive from insight for various commercial and regulatory milestones will be in a separate revenue line under milestone and license revenue.
Keith: As a reminder, the synthetic oiled royalty, we owe to royalty-farmer, is not one of the expenses deducted from the top-line dictum vote in that revenue.
Keith: The upfront $350 million that received from World Informa is liability classified.
Keith: The capped 13.8% royalty payments that are due are based on U.S. net revenue of Nick Timbo, and the payments we make will reduce the associated liability on the balance sheet.
Keith: You will also see on our income statement a new line called royalty interest expense which reflects the interest expense calculated using the effective interest rate method [inaudible]
associated with the amortization of the liability principle.
Keith: With regard to our guidance on expenses, you can find our guidance for the second quarter of 2025 and full year on the slide and then the press release we issued earlier today.
Keith: Turn to the ballot sheet, we continue to maintain a strong financial position with $602.1 million in cash, equivalence, and short and long-term investments as of March 31st
Keith: We expect that our cash, combined with anticipated Revy Forge gross margin contribution, collaboration revenue from Nikimbo, and an interest income, will enable the company to reach profitability.
Michael Metzger: But that, let me now turn the call back over to Michael [inaudible]
Michael Metzger: Thank you, Keith. As you heard today, we have continued to make outstanding progress as a commercial company and Syndax is well positioned for long-term success.
Speaker Change: We are in the enviable position of having two de-risk medicines with high, with multi-billion dollar potential along with the capital and the right strategy and people to execute on these opportunities [inaudible]
Speaker Change: Looking ahead, we remain sharply focused on fulfilling our commitment to patients and shareholders to achieve the multiple upcoming milestones that you can see on slide 13
Speaker Change: As always I want to close by thanking everyone who has made it possible for us to reach this transformational point, including our dedicated Syndax employees and our long-term investors
Speaker Change: I especially want to extend my gratitude to the patients and families who have chosen to participate in our clinical trials
Speaker Change: On slide 14, you can see a picture of Lila, one of the young children with acute leukemia who participated in our study. Hearing remarkable stories like Lila is what drives our entire organization to deliver for patients every day. With that, I would like to open the call for questions, operator.
Thank you.
Speaker Change: At this time, I would like to remind everyone in order to ask a question, press star, then the number 5 on your telephone keypad. If you would like to withdraw your question, press star 5 once again.
We'll pause for a moment to compile the Q&A roster.
And great.
Speaker Change: All right, our first question comes from Anupam Rama from JP Morgan. Your line is now open.
Speaker Change: Hi, guys. This is Priyanka, Anupam. Congratulations on the amazing progress. Our question is, what are you seeing in regards to the repeat prescribers for registrations, other certain centers of excellence that have viewed high repeat prescribers, or are those prescribers more spread out? Thank you.
Speaker Change: Great, thank you so much for the question and I'll actually ask Steve to address that regarding repeat discrepters. Yeah, hey Brad, good question I think in the prepared comments we've talked about a significantly size user base 44% of our tier ones and tier two is have written in
Speaker Change: About 80% of those folks have actually ordered more than once, whether that was for another patient or for a refold prescription.
Steve: I wouldn't say it's concentrated in only those groups. We know about two-thirds of the business is coming from that group. I think what's very encouraging about the launch is that we're seeing usage well outside the Tier 1 and Tier 2s.
Steve: We do call on 2,000 treatment centers. There's oncology practices that if they haven't already prescribed, they refer to patients into other academic centers and we've seen a number of
Steve: Small Academic Centers prescribed as well. So I would say that it's a good foundation to start. I think physicians like the pro-phobia, like what they're seeing in patients, and that user base is growing, and those that are using more than once is increasing. We know the first prescription is often the hardest, and then once we've seen that happen that...
Steve: A muffled memory of how to prescribe diagnosed and treat comes after that pretty quickly.
Thank you so much.
Speaker Change: Thank you. Our next question comes from Kelly Sheeth from Jeffries. Kelly, your line is open.
Clara Elm: Hi guys, this is Clara on for Kelly, congrats on the great progress. Let's go.
Clara Elm: For the patients receiving rebel forged, could you comment on whether you are seeing a similar pace of patients receiving transplant to what you're seeing in clinical trials and maybe whether you're seeing any patients use rebel forged as a maintenance therapy? And how do you expect this dynamic to evolve over time? Thank you
Yigal, thanks for the question, so...
Clara Elm: Your question relates to patients receiving every forage and whether we're seeing transplants consistent with clinical trials. I could just say that.
Right now it's anecdotal information, I think we're...
Clara Elm: We do know from physicians who we speak to reporting that they are taking patients to transplant and we do expect patients to go back on posting craftsman in the maintenance setting. I think it's a little early to comment too much on that and we'll see that.
Clara Elm: kind of go throughout the year as more and more patients do go to transplant, but it's very encouraging what we're seeing so far relative to transplant. We're just mostly added total information at this point.
Okay, got it, thanks.
Speaker Change: Thanks for the question. Thank you. Our next question comes from Ellen Horst from TV Kelly. Your line is now open.
Ellen Horst: Hi, guys. Thanks for taking the question and congratulations on an awesome Q1. I'm wondering if you can share any caller on the month-over-month trends for new patient ads for Revy foraging Q1 as it was patient closed studies through the quarter or did you see some acceleration throughout? And...
Speaker Change: Then, can you share any details on the pre-drug rate and whether you expect that to continue in Q2 and beyond?
Speaker Change: Sure, thanks so much for the question. So, Steve, on month-to-month trends, do you want to talk about that?
Steve: We didn't get a wide range of patients at the launch all the way from first relapse to patients that were on hospice. So we're moving into steady stakes on, I think drugs like this, you're going to have to wait, probably at least two full quarters, really to see the run rate but we're happy with what we see, we're happy with the
Speaker Change: The Net Sales gain from Q4, Partial Quarter into our first quarter into Q1, and we haven't reported on what we're seeing this month in April , but as you can imagine, user bases is increasing, you know, Michael answered the question on transplant, we're seeing patients already on their fifth refill, in some case, which is very encouraging. I think another part of your question was just around free drug. Thank you very much.
Speaker Change: So we believe every appropriate patient should be put on drug. We've made every effort obviously to get drug paid which we've had a very successful rate as formula recovered has improved. Our patient assistance program is very specific. It's really for uninsured or underinsured patients.
Speaker Change: Sometimes through the first of the year, you'll see some changes in terms of the ductables resetting. Medicaid plans tend to open up a first full quarter after the drug had launched, so that opened up the April 1st.
Speaker Change: So the rate of free drug right now is very low. It's in the single digits and that's just due to execution and a great trade team where levels that you typically see two to three years into a launch. So we feel great about where we are and all patients being treated. Thank you.
Thank you [inaudible]
Thanks for the question.
Speaker Change: Great, our next question comes from Peter Lawson, from Barclays. Your line is now open.
Peter Lawson: Hi, thanks so much, thanks for taking the questions. I wonder if you could speak about any trends that you'll see in GVHD in particular of any of the subgroups.
Thank you [inaudible]
Peter Lawson: Great, Peter, thanks for the question. So we've already trends in GVHC in terms of subgroups. I assume your question is, you know, the patients that are actually getting the drug and what their, what their specific clinical manifestation is. GVHC, is that the question? Yes, yeah, exactly. And then if there's any kind of age differences between those that, again, it versus not any, any details of it would be great.
Thanks for watching.
Speaker Change: Yeah, I'd maybe turn the question over to Peter any kind of information relative to that. I don't know that we've reported any of that yet, but Peter, I don't know if it must have said it. No, I think it's a little bit early for us to be able to comment on that. I know that the team's collecting the...
Speaker Change: Real-world evidence is coming in, but I think it was just sort of partial quarter so far. I don't think we have that much information. Yeah, catch it. Okay. Thanks, Peter, to me.
Good.
Peter Lawson: Go ahead, I would just like to find a question just around MSS, CRC, kind of what's a go-forward signal you need to see about?
Thank you. Thank you. Thank you.
Peter Lawson: Right, Peter, so thanks for the question and you're asking about colorectal cancer and I think we've...
Peter Lawson: We continue to follow patients and I think that's where I'll continue as we haven't given an update as of yet [inaudible]
Peter Lawson: But I think what we were looking for was stable, stable disease, prolonged stable disease over a certain period of time and we're looking to follow up on that sometime later this year.
Thanks so much. Thank you.
Speaker Change: Great Peter, thanks. Our next question comes from Michael Schmidt from Guggenheim, your line is now open.
Alright guys, congrats on the first quarter result. Thank you very much.
Michael Schmidt: Could you just comment on to what degree off-label use in NPM1 patients may have contributed to one
Michael Schmidt: And then, yeah, I had a clinical question also on the comments that were made around the change in the design of the Olaf II study where you now have the CR endpoint included. And yeah, could you just comment on how that.
Michael Schmidt: May impact potential timing to a completion of the trial, and it sounds like you may have had some of discussions around the plant studies in the fit patient population as well. Just curious if you could share some of the feedback that you've received from the agency. Thanks so much.
Michael Schmidt: Great. Thanks, Michael, for the question. So in terms of off-label contribution for MPM1, I think what we're hearing again anecdotally, I think, and based on some of the things that we can get our own data, this looks so far.
Michael Schmidt: Primarily to be a Camt2A set of patients. I think there's been some...
Michael Schmidt: Physicians reported to us that they are prescribing the drug for MPM1, they're prescribing in combination, monotherapy, and also potentially even earlier lines of therapy. But I think the vast majority of what we're seeing in this quarter is unlabeled KMT2A.
And then...
Michael Schmidt: And in terms of your your your question about the clinical comments about our our unsit our unsit trial
Michael Schmidt: And that's what we're calling now the evolved trial. From the start, the trial was designed to collect CR.
Michael Schmidt: and we're amending the analysis plan and raising CR in the hierarchy of endpoints, and so it's pretty straightforward.
So these are dual primary endpoints. [inaudible]
Michael Schmidt: Independent Success Criteria for both so we can win on either. So it's a, I think an advantage set up for potential accelerated approval. Let's go.
Michael Schmidt: you know, we have alignment there to go forward. So there's no impact. There's no impact to, you know, trial, because the trial will simply go forward when we start it. But in terms of accelerated approval and how that potentially could be an early readout, we haven't given guidance on the timing there. But we do expect...
Michael Schmidt: As we are the first to start a trial in the front line setting, we do expect to be the first
Michael Schmidt: You know, men inhibitor approved in the front line hopefully by a good margin so that's a positive development and then on the fit in the fit. [inaudible]
Michael Schmidt: Side of the equation with the trials will be starting there. We're not commenting as we generally don't on FDA interactions and our conversations there, but we are moving in a positive direction on all fronts including in the fit setting with additional trials. [inaudible]
Speaker Change: Great, thanks for your question, Michael. Our next question comes from David Day from UBS. David, your line is open.
David Day: Great, thanks for taking my questions and congrats on the quarter. I also just want to focus on evolve to a trial in the onset population. So understand that you just got FDA buy-in to use the dual from the endpoint. So I wonder if it's possible for you to provide some additional color around powering for the trial. Thank you very much.
David Day: Yeah, David, thanks for the question. We haven't talked about the powering of the trial. I mean, it's a other than the fact that, of course, it's the number of patients, 415 patients. It is a randomized trial. It's powered with MPM, you know, based on MPM1 population versus KMT2A, because MPM1 is far greater patient population. And essentially that's the... [inaudible]
David Day: So, it'll be both popularly included, but it'll be powered off of MPN1, but specific statistics we haven't talked about publicly at this point.
Thank you.
Thank you.
Speaker Change: Thanks for your question David. Our next question comes from Yigal Nochomovitz from City. Your line is now open.
Eagle Notromovitz: Yeah, hi, thank you very much. I had a question on the strategy around the fit studies. Could you just elaborate as to why it seems like you're pursuing two separate trials for MPM1 and KM22A versus in the unfitted, it's all lumped together. Is there a specific reason for that? No, I don't think so.
Speaker Change: Is that a stone or is that something that's still subject to discussion with the FDA?
Speaker Change: No, I think thanks for the question, Yigal. I think the simple answer is that we're there are different populations of patients and I think we we're having a trial that's specifically for MP1, a trial specifically for KMT2A. I think the outcomes, the
Speaker Change: The standard care, the work that we're doing in each population is specific, or the trial design is specific to, you know, trying to achieve the best outcomes in both of these populations. And so, um,
Speaker Change: Knowing that there are differences we design trials that would be most appropriate for these populations so that's the driving force behind the strategy and we think that obviously we'll give us the best chances of success. Thank you very much.
Speaker Change: Okay, and also on the comments around the use of revenue forage.
Speaker Change: and the transplant patients that you mentioned. I'm just curious, did some of those, did they achieve CR CRH? Are you seeing in the commercial setting that the physicians are willing to go to transplant before seeing CR CRH with Ravi Forge? If you know, I know that's quite detailed.
Speaker Change: Yeah, thanks Yigal. We don't actually know. I mean, I think that's that's a physician choice, right? They treat their first their first priority is to get the patient to a
Speaker Change: to a remission and then they make a determination based on that individual patient, whether they have an MRD negative CR or just a CR that's completely blind to us that's there in their own domain. So we do expect that we'll learn more as we go, but as it's today we don't have that information.
All right, thank you [inaudible]
Thanks Yigal.
Speaker Change: Thanks for your question. Our next question comes from Kaltit Patel from B-Rionie Security's, Your Line is Now Open.
Thank you. Thank you.
Kalpit Patel: Yeah, hey, good afternoon and congrats on the net sales for both of these assets. I had a couple questions for Revy Forge. Do you have any color on what portion of the $20 million revenue? Thank you.
Kalpit Patel: Stemmed from refill dynamics, from those fourth quarter patients, versus the new patient starts in the first quarter, and then as a follow-up, do you have an early sense of the median duration of therapy for those patients that were enrolled or treated in the fourth quarter?
Speaker Change: Yeah, thanks, Calpit. Thanks for the question. So the first question regarding refill dynamics, let me ask Steve to comment on that.
Speaker Change: Yigal, good question. I think both are building right now. I'm not going to give a lot of call. I think the data set is, is uh...
Speaker Change: is still new. I think we talked about this before. We have decent visibility until maybe less than half to our specialty pharmacies. [inaudible]
Speaker Change: and our hub, not so much to the other side, which is a specially distributed side, which is why we...
Speaker Change: are not able with a lot of accuracy to answer some of these questions, but both are filling. I mean, I think from a refill rate perspective is we talked about getting to transplant. I mean, we've got patients now on their fourth refill. Obviously, there's more patients on over time. We're able to track that at least monthly from the data that we can see.
Speaker Change: We like what we're seeing, we do also obviously know when patients are dropping out of treatment it doesn't mean that it's been a treatment failure We're hoping that they go to transplant because that's really the only curative option that they have
Speaker Change: You know, in terms of patients and new patients, it's been a steady stream month to month and that's been building over time. So I'd say the fundamentals and the business are good. We're exactly where we want to be, April's the big month and obviously this coming quarter is going to be big as well.
Um, Um,
Speaker Change: And then your second question, median duration of therapy, so it's early. I think that we know we get this question a lot. That is going to take time for the data set to mature just the dynamics of patients going off.
Speaker Change: Off-drug, hopefully to a remission, getting to transplant and graftment, usually two to three months later, they may be back on treatment [inaudible]
Speaker Change: But what we can't see in the data from a compliance perspective, we like what we're seeing, right? You're going to see a decay month to month over time for a variety of reasons, but we think in, let's say a couple of quarters from now, we'll have to give a real definitive answer on duration of treatment and we like where we're at right now from the data that we can't see. [inaudible]
Okay, thank you very much.
Thanks, Calpit.
Speaker Change: Thanks for your question. Our next question comes from Jeet Mukherjee from BTIG. Your line is now open.
Speaker Change: Hey, congrats on the quarter and thanks for taking the question. Perhaps in line with the previous question just...
Jeet Mukherjee: Any color on the month-to-month trend for new patient starts for NICTIMVO, or alternatively the greater than 1250 infusions year-to-date, you know, what proportion of that is new patients and just any color on refill rate for NICTIMVO. Thanks.
Speaker Change: Thank you, Jeet. Another question for Steve here on the Kimbo. Yeah, really early. Even the 12 of the 15th, and it's more than 12 of the 15th, I know that's what inside reporter earlier this week. And that's really an estimate that they can take from a specialty distributor data because that's how they distribute their drug. [inaudible]
Thank you.
Speaker Change: And, you know, some of those patients that there's an EAP, I don't think they've given color to how big it is, but there are obviously some patients on the EAP. There were some patients that were waiting at centers. As, you know, the drug was initially approved in August . Ultimately, new vials out late January . So there was some, we'll call it some pent up demand. [inaudible]
Speaker Change: You know, largely new patients now since it's only a couple of months in, it was activated against all the pretty much all the important centers in the country. I think the statistic we gave was 95% of the top centers. That was as of I think March that
Speaker Change: Pardon that today, there's other centers bringing the drug on board, so it's been a smooth onboarding.
Speaker Change: High patient to man, I think the drug is being received well, I think we have to...
Speaker Change: Someone asked earlier just about the types of patients it's probably fourth line which is fine to get started patients and physicians will get experience like what we're seeing it'll take obviously another quarter and then even a little bit beyond that to see the true run rate as as this sort of these warehouse patients work through the AP gets exhausted. [inaudible]
But off to a great start, no doubt.
Great, thank you [inaudible]
Thank you.
Speaker Change: Thanks for the question, Chief. Our next question comes from Selim Sayer from Hizuhul. Your line is now open.
Salim Syed: Hey, guys, good afternoon. Thanks for the question and congrats on the quarter. I guess a couple from us, just one on Ready Forge and one on the Nictinville launch. On Ready Forge, is there any color you can provide just on the relative skis split between the 25-meg, 110-meg and the 160-meg just...
Salim Syed: So we can understand what your blended whack is per patient and then on the on the Nick Timvo launch just to clear on your slide nine here the 1250 infusion fear to date that's as of
Speaker Change: I want to say April when inside reported versus March 31 is want to be clear and that's not that's actually infusions and not unique patience correct thank you
Speaker Change: Thanks for the question. So maybe I'll turn it to Steve. The first question is related to Rev on the sort of breakdown of the SKUs because we do have three different SKUs, 25, 110, and 160.
Speaker Change: We do, we're not going to give a specific breakdown yet, I think guys like other things, the data sets mature, and we know the majority of prescriptions are going to be up to 110 to 160 to 25 is...
Steve: is new, we launched that in March. But the dosage varies, right? It's going to vary based on patient weight, also on concomit, use of a strong 3-4. The bottles are their 30s, it's twice a day, so essentially it's, you know, two bottles.
Steve: for most patients. And the majority of patients, that's exactly what they're going to get. If they're not on a strong sip, it's possible they may have to, you know, add on a dose. You know, at this point, we're, you know, our guidance is that majority scripts are going to be at that one 10 and 160 dose. [inaudible]
Speaker Change: I'm in for Nick Timbo, I think we're clarifying. Yeah, 12-5, I think it's end of March, was what the data was through and I think the question was it's not patients, that's infusions. So in the time period since launch, it's very likely that some of those were for the same patient, meaning a couple of infusions. It's every two weeks and we don't have the mix yet of new patients versus continuing patients. [inaudible]
Speaker Change: Okay, got it, and just quick follow up, if you use two vials, that's still one infusion, correct?
Speaker Change: Yeah, how they calculated this is, again, it's the SD Channel, so they just know how much they shipped out to customers. They don't know if it's a new patient and existing patient, so the way you calculate it is they'll use an average toast per patient based on the clinical trials, and then they would apply that to the product that they sold to come up with the number of institutions. [inaudible]
Got it. Okay, super helpful. Thanks so much, guys.
Thank you, Lin.
Speaker Change: Thanks for your question. And once again, if you have a question, you may press star 5 on your telephone keypad. Our next question comes from George Farmer, from Scotia Bank. Your line is now open.
George Farmer: Hi, good afternoon. Thanks for taking my questions. Given the strength of your Q1 review forage result, do you think that the total addressable market is accurately reflected in our model? So you're getting a sense of...
George Farmer: of how many patients might be out there, maybe it's greater than what we've estimated. And also, can you comment, is post-transplant use reimbursed under the current J-code? Yeah, that's good.
Thank you, George. Thanks for the question. And so...
Just-
And Steve may chime in here, but total addressable market.
George Farmer: I think when you have a new market or a new drug that addresses a targetable population, you're often experiencing dynamics where you're exposing
or bringing new patients. [inaudible]
George Farmer: to, you know, into the market or be, have them be able to be treated. I think that's-
George Farmer: That could be true for review forages. There wasn't anything available for K&T2A or the...
George Farmer: The target of population was, as we said, every left is referred to about 2,000 patients It could be larger than 2,000 patients I think we'll have to see how that unfolds over time
Speaker Change: But we are, as Steve said, making great progress, not only identifying patients but getting them on drug and getting on drug quickly. So it's, we're off to a great start, wouldn't surprise us if the market were bigger than the 2000 patients,
Second question.
In terms of post transplant.
Speaker Change: Yep, post-transparent being reimbursed. We have only evidence and there's evidence of other drugs being reimbursed for
Post-Rexinal Maintenance, I think it's this. [inaudible]
Physicians say these are important. [inaudible]
Speaker Change: It's important to get patients back on drug after engrapment in order to maintain them in remission. And I think that is more and more accepted by pairs as well. These are high risk patients. And so we expect
Speaker Change: That post-transplant maintenance will be reimbursed, you know, generally. And so that's that is our that is our understanding.
Speaker Change: Okay, great. And maybe you mentioned this regarding the S-N-D-A or M-T-M-1. Is that going to be based on the 77 patients in the efficacy analysis or is there a larger data set?
Speaker Change: Yeah, so we updated the data set at our Ashe event last year. The efficacy of valuable patients were 77 patients and that's in fact we've submitted all the data to the FDA and the SNDA and so they have access to the 77 patients, they have access to every data set that we've pulled together. But
Speaker Change: But we do expect that they'll look at the 77 page. I can't tell you what will ultimately be part of the final data set that they analyze and include in the link.
Okay, thanks very much [inaudible]
Thank you.
Speaker Change: Thanks George, and our final question comes from Jason Zemansky from Bank of America. Your line is now open.
Jason Zemanski: Great. Good afternoon. Congratulations on the quarter and really appreciate you fitting us in with our question.
Jason Zemanski: In terms of the NCCN guidelines, it looks like the AML panel meets May 19th. Do you think you could realistically get in front of them with your publication by then? Alternatively, if not, is there potential for the committee to meet Ed Hock? Okay.
Speaker Change: So, Jason, thanks for the question. Exciting development, as we've announced today that we have acceptance of our manuscript in a very good journal. I think...
The idea is that we would...
Speaker Change: submitted to guidelines quickly, whether or not we can get it in for the...
Speaker Change: for the May 19th Guideline Review is an open question. I think we're optimistic, we'll see.
And then of course, we've had experience where...
Speaker Change: Guideline Committee is met on an ad-hoc basis, so we do think that for practice changing an important new medicine that.
Speaker Change: that they would meet in order to accommodate that. So I think we have both opportunities and we do expect to be included relatively rapidly as the year goes on. So we're I think in good shape either way.
Speaker Change: Perfect, so it's fair to say probably near a term rather than longer term. [inaudible]
Speaker Change: Yes, the air term is our expectation. We had said second quarter as our guidance so that's and of course we don't control that fully but realizing that we're you know where we are on the calendar I think we have a we have a good shot at that. [inaudible]
Understand, thanks for the color. [inaudible]
Great, thank you [inaudible]
Speaker Change: Thank you Jason. Alright, this concludes our question and answer session. I will now turn the floor over to Mr. Michael Metzger for any additional comments or closing remarks
Speaker Change: Great. Thank you all. We really appreciate you tuning in today to discuss our recent progress and the exciting milestones ahead.
Speaker Change: You look forward to seeing many of you at several upcoming investor conferences, including the Bank of America Conference, Bank of America Conference, later this month, and Jeffries in early June , as well as other important medical conferences this quarter. And with that, I'd say have a great day everyone.
Sharon, do as you please.