Q2 2024 Syndax Pharmaceuticals Inc Earnings Call
Good day everyone and welcome to the Syndax second quarter 2024 earnings conference call. Today's call is being recorded. All participants have been placed in a listen only mode.
Operator: If you would like to ask a question, please press star 5 on your telephone keypad to be placed into the Q&A box. You may also press star 5 again to remove your shelf.
You will have an opportunity to ask questions after today's presentation. If you would like to ask a question, please press star 5 on your telephone keypad to be placed into the queue.
You may also press star 5 again to remove yourself from the queue.
Sharon Klahre: At this time, I would like to turn the call over to Sharon Klahre, Head of Investor Relations at Syndax Pharmaceuticals.
Sharon Klahre: Thank you, Operator. Welcome, and thank you all for joining us today for a review of Syndax's second quarter 2024 financial and operating results. I'm Sharon Klahre, and with me this afternoon to provide an update on the company's progress and discuss financial results.
Our Michael Metzger, Chief Executive Officer, Dr. Neil Gallagher, President and Head of R&D, Steve Closter, Chief Commercial Officer, and Keith Goldan, Chief Financial Officer.
Speaker Change: Also joining us on the call today for the question and answer session are Dr. Peter Oerdemlich, Chief Scientific Officer, and Dr. Anjali Ganguli, Chief Business Officer.
Unknown Executive: This call is accompanied by a slide deck that has been posted on the investor page of the company's website. Now, you can turn to our forward-looking statements on slide two. Before we begin, I would like to remind you that any statements made during the call that are not historical are considered to be forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. However, 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 SAC.
Speaker Change: This call is accompanied by a slide deck that has been posted on the investor page of the company's website.
Speaker Change: You can now turn to our forward-looking statements on slide 2.
Speaker Change: Before we begin, I would like to remind you that any statements made during the call that are not historical are considered to be forward-looking statements within the meaning of the Private Securities 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 factors section in the company's most recent quarterly report on Form 10-Q .
Unknown Executive: Any forward-looking statements made represent our views as of today, August 1, 2024, only. A replay of this call will be available on the company's website, www.syndax.com, following its completion. With that, I'm pleased to turn the call over to Michael Metzger, Chief Executive Officer of Syndax Pharmaceuticals, Inc., for more information. Thank you, Sharon. Good afternoon, everyone, and thank you for joining us today.
Speaker Change: as well as other reports filed with the SAC. Any forward-looking statements made represent our views as of today, August 1st, 2024 only.
Speaker Change: A replay of this call will be available on the company's website, www.syndax.com following its completion. With that, I'm pleased to turn the call over to Michael Metzger, Chief Executive Officer of Syndax.
Michael Metzger: Thank you, Sharon. Good afternoon, everyone, and thank you for joining us today.
Michael Metzger: Starting with slide 3, this is a very exciting year for Syndax as we stand on the cusp of becoming a commercial stage company with the anticipated upcoming FDA approvals of Revumenib and Exotilamab, two first-in-class treatments for diseases with high unmet medical needs.
Michael Metzger: I'm pleased to report that we have achieved several clinical milestones in the second quarter that support our strategic priorities.
Michael Metzger: In June, we presented updated positive revumenib combination data from the BEAT-AML and AUGMENT-102 trials at the European Hematology Association, or EHA, Congress. These data, which Neil will review shortly, continue to support Revumentib's potential to enhance current standard of care agents. Reinforcing the previous organ response data that we have shared, this latest data set shows that responses were noted in all fibrosis-dominant organs that had clinical activity supported by clinician-reported and patient-reported changes in organ-specific symptoms, such as improvements in swallowing, shortness of breath, skin and joints, and sclerotic skin.
Michael Metzger: In June , we presented updated positive revumenib combination data from the BEAT AML and AUGMENT 102 trials at the European Hematology Association, or EHA, Congress.
Michael Metzger: The data, which Neil will review shortly, continues to support Revumentib's potential to enhance current standard of care agents.
Speaker Change: At EHA, we also presented additional positive data from the Agave 201 Pivotal Trial, evaluating exotilamab in patients with refractory chronic graft-versus-host disease, or GVHD.
Neil Gallagher: Reinforcing the previous organ response data that we had shared, this latest data set shows that responses were noted in all fibrosis-dominant organs that had clinical activity
Michael Metzger: Supported by clinician-reported and patient-reported changes in organ-specific symptoms, such as improvements in swallowing, shortness of breath, skin and joints, and sclerotic skin.
Michael Metzger: Looking ahead, we are on track for a historic year with the anticipated FDA approval of Revimenev and Oxetilimab coming soon.
Speaker Change: With Axitilumab, our anti-CSF1 antibody for patients with chronic GVHD, we have a PDUFA action date of August 28, 2024, and anticipate launching in the fourth quarter of this year with our partner Insight.
Michael Metzger: With RevuMenib, our menin inhibitor being reviewed by the FDA for patients with relapse or refractory KMT2A rearranged acute leukemias, we have a PDUFA action date of December 26, 2024. As we announced earlier this week, the FDA recently extended the PDUFA action date for RevuMenib from September 26 to December 26, 2024, a standard three-month extension to allow for additional time to conduct a full review of the supplemental information we provided in response to their request.
Speaker Change: With Revimenib, our menin inhibitor being reviewed by the FDA for patients with relapse or refractory KMT2A rearranged acute leukemias, we have a PDUFA action date of December 26, 2024.
Speaker Change: As we announced earlier this week, the FDA recently extended the PDUFA action date for revumenib to
Speaker Change: NDA from September 26 to December 26, 2024, a standard three-month extension to allow for additional time to conduct a full review of supplemental information we provided in response to their requests.
Speaker Change: Importantly, we are confident that the information we have provided supports approval and continues to demonstrate the meaningful benefit Revumented brings to patients.
Speaker Change: We look forward to continuing to closely engage with the FDA as they complete their review under the Real-Time Oncology Review Program, or RTOR, and we stand ready to launch RevuMed with strength as soon as we receive the anticipated approval.
Michael Metzger: Later in the call, Steve will provide some additional color on our preparations for these two anticipated launches. Positive results could support the future use of these agents in combination with standard of care medicines earlier in a patient's treatment and thereby expand their utility significantly. We are in a strong financial position with $455 million in cash as of June 30th, which we expect will provide sufficient capital through 2026.
Speaker Change: Later in the call, Steve will provide some additional color on our preparations for these two anticipated launches.
Steve Closter: We also have several other meaningful milestones approaching, such as the anticipated readout of pivotal top-line revumentum data in relapsor refractory MPM1-AML in the fourth quarter, which could serve as the basis for a supplemental NDA filing in the first half of 2025.
Speaker Change: Additionally, we expect to make further progress advancing our clinical trials of Revumenib and Axitilumab that are underway or planned to initiate in the coming months.
Speaker Change: Positive results could support the future use of these agents in combination with standard of care medicines earlier in a patient's treatment and thereby expand their utility significantly.
Speaker Change: We are in a strong financial position with $455 million in cash as of June 30th that we expect will provide sufficient capital through 2026.
Speaker Change: Our current balance sheet not only supports our planned commercial launches and clinical trials, but also sets us up to expand beyond our first registration indications and to pursue early stage business development opportunities.
Speaker Change: I will now ask Neil to review some of our recent data presentations and pipelines. Neil?
Neil Gallagher: Thanks Michael. Turning to slide 4 in Revumenib, we have a robust clinical development strategy underway that will support the use of Revumenib in KMT2A rearranged and re-content PM1 acute leukemias across the treatment paradigm.
Neil Gallagher: The development plan extends beyond the initial relapsed or refractory indications. It's trials of regimen have in combination with standards of care potentially support use of the front in the frontline relapsed or refractory and post-transplant maintenance settings.
Speaker Change: On the next two slides, I will briefly review the data recently presented at EHA from the two combination trials that you see listed, BEAT-AML and AUGMENT-102.
Speaker Change: Slide 5. At IHA in June , we and our collaborators at the Leukemia and Lymphoma Society presented updated data from the BEAT AML trial of reviumatib in combination with venetoclaxonesis or venasa in patients newly diagnosed with mutant MPM1 or KMT2A rearranged AML.
Neil Gallagher: This updated data set, with a cutoff of the 1st of May 2024, includes 24 efficacy-evaluable patients.
Michael Metzger: 96% of patients had a composite complete response for CRC, and 92% had no residual disease and were therefore MRD negative. The Dose-Limiting Toxicity, or DLT, window for each was cleared last year in this trial, and both cohorts are currently being expanded in order to identify the dose-to-phase 3 developers. Of note, the rates of adverse events associated with the combination of reviumenib with venasa observed in the study are consistent with data for venasa from historical controls.
Neil Gallagher: 96% of patients had a composite complete response for CRC and 92% had no residual disease and were therefore MRD negative.
Neil Gallagher: As in all the combination trials conducted so far, two doses of Reviuminib are being investigated. 113 mg and 163 mg are administered every 12 hours.
Neil Gallagher: The Dose-Limiting Toxicity, or DLT, window for each was cleared last year on this trial, and both cohorts are currently being expanded in order to identify the dose for Phase 3 development.
Neil Gallagher: The combination is well tolerated and the safety profile reported at EHA is consistent with what has been previously reported.
Neil Gallagher: Of note, the rates of adverse events associated with the combination of Revumenov with Xanasa observed in the study are consistent with data for Xanasa from historical controls. For example, the addition of Revumenov to the doublet does not appear to exacerbate the minor suppression associated with Xanasa alone.
Michael Metzger: For example, the addition of reviumenib to the doublet does not appear to exacerbate the minor suppression associated with venasa alone. Additionally, the BEAT AML investigators concluded that the data suggest that adjusting the dose of venetoclax while maintaining the dose for every minute may be the most logical strategy for future use. Turning to slide six.
Neil Gallagher: Additionally, the BEAT AML investigators concluded that the data suggests that adjusting the dose of venetoclax while maintaining the dose per minute may be the most logical strategy for future development.
Speaker Change: As we've previously communicated, we intend to initiate a pivotal trial with this triplet combination in newly diagnosed patients' wavefront.
Neil Gallagher: Turning to slide 6...
Neil Gallagher: At EHA we also presented updated data from the Augment 102 trial of Revimenib in combination with Fludarbine, Cytaribine, or FLA in a predominantly pediatric population with relapsed refractory, mutant NPM1, NUP98 rearranged, or KMT2A rearranged ANL.
Neil Gallagher: As of the data cutoff of 15th of January 2024, 27 patients received flapless Reviminib at 113 mg or 163 mg.
Neil Gallagher: Patients included in the trial were heavily pre-treated with a median of three prior lines of therapy.
Neil Gallagher: 67% had prior FLA containing regimens. 52% of patients achieved composite complete remission or CRC. The MRD negative rate among CRC patients was 71%. Furthermore, 7 patients or 50% of responders proceeded to transplant once they had entered remission.
Michael Metzger: As with the Venazo triplet we discussed, Revumenib does not appear to alter the tolerability profile of this standard of care regimen. These two updated data sets presented at EHAF provide further support for combining Revumenib with different standards of care in newly diagnosed as well as relapsed or refractory patients. We are on track to report data in the fourth quarter. On this slide, you can see the results from 14 mutant MPM1 patients in the Phase 1 portion of Augment 101, which showed that 50% of patients achieved a response, and 36% achieved a complete remission or CR with partial hematological recovery.
Speaker Change: As with the VenAZER triplet, we just discussed Revumentiv does not appear to alter the tolerability profile of this standard of care regimen. These two updated data sets presented at EHAF provide further support for combining Revumentiv with different standards of care in the newly diagnosed as well as relapsed or refractory settings.
Speaker Change: Turning to slide 7, in March we completed enrollment in the pivotal cohort of the Augment 101 trial with the enrollment of 64 adults and up to 20 pediatric relapsed or refractory NPM1 mutant AML patients.
Speaker Change: We are on track, or acute leukemia patients, we are on track to report data in the fourth quarter.
Speaker Change: On this slide, you can see the results from 14 mutant NPM1 patients in the Phase 1 portion of Augment 101, which showed that 50% of patients achieved a response and 36% achieved a complete remission, or CR, with partial hematological recovery. Notably, all patients with a CR CRH were MRD negative.
Speaker Change: Consistent with the KMT2A population, Revumenib also enabled patients in remission to proceed to transplant with durable responses, despite many of the patients having failed prior venetoclax therapy and stem cell transplant.
Speaker Change: These data also indicate that Revumenib is well tolerated in patients with mutant MPM1 with an overall safety profile that is consistent with what has been previously reported in the KMT2A rearranged population.
Michael Metzger: Before I hand over to Steve, I'd like to provide a quick overview on slide eight of the preparations we've made on the medical side of the organization to support the upcoming anticipated launches. We have built a highly experienced medical affairs team that is driving deep scientific engagement and robust evidence. As you've seen from our participation with ASH and EHA, as well as various papers in top-tier journals such as Nature, we have an ambitious conference and publication plan underway to increase awareness of the compelling data from our two aims. Of note, once the top-line data for Mutant MPM1 AML are available, we plan to publish as soon as it's feasible to support potential guideline inclusion, following the approval of Revumanib and KM
Speaker Change: As you have seen from our participation with ASH and EHA as well as various papers in top-tier journals such as Nature, we have an ambitious conference and publication plan underway to increase awareness of the compelling data from our two agents.
Speaker Change: We also continue to prepare for future NCCN guideline submissions for every amendment that we anticipate will include the prescribing information, as well as peer-reviewed publications and Congress presentations.
Speaker Change: Above note, once the top-line data in Mutant MPM1 AML are available, we plan to publish as soon as it's feasible to support potential guideline inclusion, following the approval of RevuMenub in KM22A.
Speaker Change: Additionally, we have a team of seasoned medical science liaisons in the field.
Michael Metzger: They've made tremendous progress driving scientific exchange with physicians at major centers of excellent support. We've also built a health economics and real-world evidence team focused on supporting the value of a portfolio, and we continue to increase engagement with patient advocacy. Turning to Revumetib preparations and Slide 8.
Speaker Change: They've made a tremendous, they've made tremendous progress driving scientific exchange with physicians at major centers of excellence across the country.
Speaker Change: We've also built a health economics and real-world evidence team focused on supporting the value of a portfolio, and we continue to increase engagement with patient advocacy groups. With that, I'll now turn over to Steve to talk about our commercial launch plans and the market opportunities. Steve. Thank you, Anupam. Thank you, Anupam.
Steve Closter: Thank you, Neil, and thanks to everyone for joining us today. Really appreciate it. It's really a pleasure to be with you to talk about the extensive preparations we're making to position Syndax as a leading commercial stage organization serving patients and healthcare providers.
Steve Closter: We are well prepared to execute on the exciting opportunities in front of us.
Michael Metzger: Our goal simply is a strong launch, and we are ready to hit the ground running with Revumetib as soon as we receive the anticipated approval from FDA. We've hired and trained a sales team with extensive experience in pre-existing relationships in the hematology-oncology space and a demonstrated track record of success. Our customer-facing team has an average of 22 years of experience, primarily in hematology and oncology, and an average of six product launches each.
Speaker Change: Turning to Revumatib preparations and slide 8. Our goal simply is a strong launch and we are ready to hit the ground running with Revumatib as soon as we receive the anticipated approval from FDA.
Speaker Change: We've hired and trained a sales team with extensive experience and pre-existing relationships in the hematology-oncology space and a demonstrated track record of success.
Speaker Change: This team is in the field right now profiling accounts and understanding the patient treatment journey so we can meet the needs of the different stakeholders involved in their care. This robust pre-launch preparation will let us rapidly begin meeting patient needs upon the anticipated approval.
Speaker Change: Our customer-facing team has an average of 22 years of experience, primarily in hematology, oncology, and an average of six product launches each.
Speaker Change: With an efficient field force footprint in the range of 30-50 individuals, we believe that we can effectively reach the relevant academic and community-based centers and meet the needs of physicians and patients.
Speaker Change: We plan to call in approximately 2,000 centers.
Speaker Change: With roughly 200 of those accounts representing more than two-thirds of the opportunity, enabling a concentrated effort. With our plan, we believe we should reach centers where 98% or more of potential relapsed refractory KNT2AR patients receive treatment.
Speaker Change: Now another important thing we've done in preparation for the launch is develop advanced data mining capabilities to appropriately identify patients in need.
Speaker Change: Because these are high-risk patients that require rapid identification of their treatment options, we've developed capabilities that will help us initiate very targeted physician engagement based on where our tools indicate there are appropriate patients.
Michael Metzger: Now, with respect to market access, we built an accomplished team with extensive experience working with payers and other trade partners to facilitate access to new products. Together with the medical affairs team, our payer field team continues its pre-approval information exchange work with payers, and we're on track to reach plans covering more than 90% of all covered lives, both commercial and Part D, prior to the anticipated approval. Payers tell us that they recognize the unmet need and appreciate the value that Revumena provides.
Speaker Change: Now with respect to market access, we built an accomplished team with extensive experience working with payers and other trade partners to facilitate access to new products.
Speaker Change: Together with the medical affairs team, our payer field team continues their pre-approval, information exchange work with payers, and we're on track to reach plans covering more than 90% of all covered lives, both commercial and Part D, prior to the anticipated approval.
Speaker Change: Payers tell us that they recognize the unmet need and appreciate the value that Revumenta provides. We believe plans will make their formulary decisions within 6-12 months of approval and will work with them to expedite the review when possible.
Michael Metzger: We believe plans will make their formulary decisions within 6 to 12 months of approval, and we'll work with them to expedite the review when possible. Importantly, given the urgent patient need, we expect that plans will provide patients access to the product at launch through the medical exception process. To support providers and patients, we've partnered with leading, best-in-class specialty pharmacies who are well-recognized for their ability to help providers and patients navigate access to new oncology medicines.
Speaker Change: Importantly, given the urgent patient need, we expect that plans will provide patients access to the product at launch through the medical exception process.
Speaker Change: To support providers and patients, we've partnered with leading, best-in-class specialty pharmacies who are well-recognized for their ability to help providers and patients navigate access to new oncology medicines.
Michael Metzger: Through a network of specialty pharmacies and specialty distributors, we're prepared to have product and channel very quickly, right after we receive approval. We're also ready to launch a dedicated patient support program that will provide a level of support on par with what you see from leading oncology. Turn to slide 9 and the markup.
Speaker Change: Through a network of specialty pharmacies and specialty distributors, we're prepared to have product and channel very quickly right after we receive approval.
Speaker Change: We're also ready to launch a dedicated patient support program that will provide a level of support on par with what you see from leading oncology companies.
Speaker Change: KFC2AR and NPM1 acute leukemias represent up to 40% of all AML patients. There are no FDA approved targeted therapies for this population.
Michael Metzger: We believe relapse or refractory KMT2AR acute leukemia alone represents a totable, addressable market opportunity of approximately $750 million in the U.S. The annual incidence of KMT2AR acute leukemia is about 2,600 patients, with the majority of these patients, about 2,000, experiencing relapse or refractory disease. We estimate a median duration of therapy across the treated population of approximately nine months, and we believe the clinical data supports pricing competitively to other targeted therapies in AML, such as split-3 or IDH inhibitors.
Speaker Change: We believe relapse or refractory KNT2AR acute leukemia alone represents a totable, addressable market opportunity of approximately $750 million in the U.S.
Speaker Change: The annual incidence of KNP2AR acute leukemia is about 2,600 patients with the majority of these patients about 2,000 experiencing relapsed or refractory disease.
Speaker Change: We estimate a median duration of therapy across the treated population of approximately nine months, and we believe the clinical data supports pricing competitively to other targeted therapies in AML, such as split 3 or IDH inhibitors.
Speaker Change: Physicians we've spoken with indicate an eagerness to prescribe Revumetam early during an eligible patient's treatment journey to bring more patients to transplant, and then extend responses by continuing with Revumetam monotherapy following transplant engraftment.
Michael Metzger: We expect that our first-mover advantage and the early experience physicians will gain treating patients with Revumetib will be vitally important to the long-term success of our brand. We estimate that the two distinct market segments, in acute leukemias, AMT2AR and MPM1, equal a combined accessible population of 5,000 to 6,500 patients in the relapsed or refractory setting and an addressable market opportunity that approaches $2 billion in the To pave the way for a successful launch, Insight is continuing to drive engagement with payers and raise awareness of the distinct pathway that Axotilimab targets and the compelling outcomes observed in the UGAVI-201 trial, which we recently detailed at the 2023 ASHCON. $29.1 million of selling general and administrative expenses. For the third quarter, the company expects research and development expenses to be between $70 and $75 million.
Speaker Change: We expect that our first mover advantage and the early experience physicians will gain treating patients with Revumetib will be vitally important to the long-term success of our brands.
Speaker Change: Our significant market share is likely to extend meaningfully beyond KMT-2AR, especially as we will be the first to deliver meaningful pivotal data and other indications such as NPM-1AML.
Speaker Change: We estimate that the two distinct market segments...
Speaker Change: in acute leukemias.
Speaker Change: ANSI2AR and MPM1 equal a combined accessible population of 5,000 to 6,500 patients in the relapsed or refractory setting and an addressable market opportunity that approaches $2 billion in the U.S.
Speaker Change: During the various meetings that I've had the chance to participate in with clinicians from advisory boards to field visits and more, I've seen tremendous excitement and support for Revumentive, creating a momentum that I look forward to building on through our commitment to delivering an absolute best-in-class experience for healthcare providers and patients.
Speaker Change: Moving to AXA TILMAB in slide 10.
Speaker Change: The anticipated commercialization of Axatilumab will be led by the Insight team and will benefit from the deep experience and long-standing relationships that they have established through their work building the GVHD market with Jackify.
Speaker Change: We'll provide 30% of the sales effort, leveraging our own field force that we anticipate will carry two products with highly overlapping call points.
Speaker Change: To pave the way for a successful launch, Insight is continuing to drive engagement with payers and raise awareness of the distinct pathway that Axotilumab targets and the compelling outcomes observed in the UGAVI-201 trial, which we recently detailed at the 2023 ASH Congress.
Speaker Change: Turning to slide 11.
Speaker Change: We estimate there are approximately 17,000 patients on treatment for chronic GVHD at any one time, the majority of whom are refractory, and cycle through therapies for better symptom control as their disease progresses.
Speaker Change: We believe there are approximately 6,500 patients progressing to later lines of treatment after two previous lines of treatment, which would be our target population for our first indication and represents an attractive initial opportunity.
Speaker Change: For instance, in the three years since the launch of Reziroc, another drug with a third line indication, net sales continue to grow, and they're annualizing at nearly $500 million.
Speaker Change: We estimate that the total addressable market for third-line treatment in the U.S. is between $1.5 to $2 billion, which assumes that patients will remain on therapy for over 12 months.
Speaker Change: Assuming Axatilumab is priced at a premium to approved agents for chronic GVHD based on its product profile and Part B reimbursement.
Speaker Change: Beyond the third-line setting, we plan to study axotilamab in earlier-line settings for chronic GVHD and other diseases where we believe its antifibrotic and anti-inflammatory mechanism is relevant, such as IPFs, which represents a large opportunity.
Speaker Change: I'll now turn the call over to Keith to review our financial results.
Keith Goldan: And thank you, Steve.
Keith Goldan: Turn to slide 12.
Keith Goldan: As Michael mentioned earlier, the $455 million in cash, equivalents, and short and long-term investments on our balance sheet as of June 30th is expected to provide runway through 2026.
Speaker Change: Our financial strength allows us to fund the anticipated commercialization of two drugs and appropriately invest to continue to realize the value of our pipeline.
Speaker Change: Turning to the income statement, operating expenses in the second quarter were $77.7 million and included $48.7 million of research and development expense.
Keith Goldan: and $29.1 million of selling general and administrative expense.
Keith Goldan: We'd like to provide financial guidance for the third quarter and full year 2024.
Keith Goldan: For the third quarter, the company expects research and development expense to be $70 to $75 million, and total operating expenses to be $105 to $110 million.
Keith Goldan: For the full year of 2024, there is no change to the existing guidance.
Keith Goldan: And the company expects research and development expenses to be $240 to $260 million, and total operating expenses to be between $355 to $375 million.
Keith Goldan: Please note that the guidance range for operating expenses for the full year includes an estimated $43 million of non-cash stock compensation expense.
Keith Goldan: Research and Development Expense Guidance includes any milestones owed to our partners upon potential drug approvals.
Keith Goldan: Ahead of the upcoming launch of Exit Tillamab, I want to briefly review how we will recognize revenue for that partner in product.
Keith Goldan: Slide 13 provides an illustrative example of accounting for sales of Exit TeleMab and is not intended to provide any margin or any other guidance.
Keith Goldan: We will record 50% of the commercial profit defined as net product revenue minus the cost of sales and commercial expenses.
Keith Goldan: During a period where there is net commercial profit for hexatillumab, as in the top example of the slide, a 50% share of the net profit will be recognized on a P&L as collaboration revenue.
Keith Goldan: During a period where there is a net commercial loss for ectopilimab, as in the example on the bottom of the slide,
Keith Goldan: Our 50% share of the net commercial loss would be included in operating expenses, designated as a separate line item called share of collaboration loss.
Keith Goldan: The milestone revenue from various global, commercial, and regulatory milestones that we received from Insight
Keith Goldan: will be recorded as milestone revenue on our income statement.
Keith Goldan: As a reminder, research and development expenses under our partnership, including regulatory and CMC expenses, are shared 55-45 in the U.S., and our 45% share is included in the income statement as part of our R&D expense.
Keith Goldan: Outside of the United States, Insight is responsible for 100% of the development and regulatory expenses, and we are entitled to receive milestones plus a double-digit royalty on ex-U.S. sales.
Keith Goldan: With that, let me turn the call back over to Michael.
Michael Metzger: During a period where there is net commercial profit for Exetil and ABB, as in the top example of the slide, a 50% share of the net profit will be recognized on the P&L as collaboration revenue. Thank you, Keith. As you heard during our call, we have made tremendous progress over the past few months and are well prepared for the transformational period ahead of us with the anticipated FDA approval and launch of our first two medicines.
Michael Metzger: Thank you, Keith. As you heard during our call, we have made tremendous progress over the past few months and are well prepared for the transformational period ahead of us with the anticipated FDA approval and launch of our first two medicines.
Speaker Change: With the momentum we have built and our robust clinical development strategy designed to explore the full potential of Revumenib and Exotilamab, we are excited about the path ahead and the opportunity we have to make a major impact for patients.
Speaker Change: On slide 14, you can see a recap of our upcoming anticipated milestones that we believe we will continue to fuel our momentum and drive value.
Speaker Change: As always, I want to express my gratitude to the Syndax team and our partners for their hard work and dedication to our mission.
Michael Metzger: Most importantly, I want to thank all of the patients, families, trial sites, and investigators who have participated in our trials and inspire us with their tenacity and commitment to finding new ways to improve the lives of patients. With that, I would like to open the call to questions. Operator?
Speaker Change: Most importantly, I want to thank all of the patients, families, trial sites, and investigators who have participated in our trials and inspire us with their tenacity and commitment to finding new ways to improve the lives of patients with cancer.
Speaker Change: I'd also like to thank our committed long-term investors who continue to share in our vision and support our work building Syndax. With that, I would like to open the call for questions. Operator?
Operator: Our first question comes from Anupam Rama from J.P. Morgan. Your line is open, feel free to unmute. Can you remind us of the size and scope of the field force, and then what sort of medical education efforts will you have ongoing ahead of the potential ACTU-TIL-NAD and REV-U-MED approvals? Our next question comes from Chris Shibutani from Goldman Sachs. Your line is now open. Right.
Speaker Change: Hi, thanks for taking the question. This is actually Malcolm Kuno for Anupam.
Malcolm Kuno: Can you remind us of the size and scope of the field force, and then what sort of medical education efforts will you have ongoing ahead of the potential ACTU-TIL and ADD and REV-E-MAN approvals?
Speaker Change: Thanks, Malcolm, for the question. In terms of size, I think my comments in the opening was, you know, roughly 30 to 50. Sorry for the wide range. We haven't given an exact number, but it's adequate to cover the audience. We've got overlapping field teams. I mean, it's a complex treatment journey for patients.
Malcolm Kuno: They've got treatment centers that have physicians and nursing staff and lab and pathology and formulary. So we've got different customer-facing teams to cover that. We'll cover, I think I said, 98% of the opportunity. So it's in place. The field team is.
Malcolm Kuno: is activating. I think your other question was around education and what are we doing. I'd say for the field...
Malcolm Kuno: Right now, as an organization, we really kind of do two things. One of them is profiling accounts. I mentioned the complex treatment journey.
Malcolm Kuno: We've been to, probably at this point, 85% of them, and in some of them, we've made multiple calls, so that's one thing we're doing, and that's profiling accounts, and the goal is to, at the time of launch, know everything that we need to know in order to get patients on drug and treated.
Malcolm Kuno: We're clearly not talking about the drug at least from a commercial standpoint, but are raising awareness of the mechanism. We're the only one that's going to be out. The awareness level should be high by the time it gets a launch. So between profiling, raising awareness, we'll be in great shape once the drug gets approved to pull it through.
Malcolm Kuno: Great, thank you.
Malcolm Kuno: Hi, this is Kevin Stragon for Chris. Thanks for taking my question.
Kevin Stragon: And then also for the BEAT AML trial, can you talk about where you are with respect to establishing a recommended phase 2 dose there? And then what the rate limiting steps are to start that pivotal trial by the end of the year? And then potentially how many doses you could bring forward into that trial? Thanks.
Unknown Executive: Maybe I'll address that. So, for the BEAT AML trial, the BEAT AML trial, I'll start there, is, as you know, we're looking for our recommended phase 2 dose. We've made tremendous progress, and at EHA, we presented the data on both of those doses. We are, in the process of filling out at least those two doses, just filling out the expected number of patients, and we're going to be making a choice at some point soon between the two doses, which is the recommended phase 2 dose. We'll be working through that over the next week. This is in relapsed refractory patients, a combination of VEN and COVI plus revumenib.
Speaker Change: At least those two doses, just filling out the expected number of patients, and we're going to be making a choice at some point soon between the two doses, which is the recommended phase two dose.
Malcolm Kuno: working through that over the next weeks. So, we're in very good shape, ultimately, to start a trial, we believe, by the end of this year, a pivotal trial. And so, stay tuned for additional updates on that as we get through the year. In the SAVE trial, we
Unknown Executive: We presented data. There was data presented last year at EHA, sorry, at ASH. So, the investigators told us that there will be some updated presentations in the latter half of this year. But, of course, we're not at liberty to say exactly where that is at this time.
Speaker Change: Again, this is Dr. Gus Issa's trial at MD Anderson. This is in relapsed refractory patients.
Malcolm Kuno: A combination of Venn and Covey, plus Revumentib. We presented data, there was data presented last year at EHA, sorry, at ASH.
Speaker Change: So the investigators told us that there will be some updated presentation in the latter half of this year, but of course, we're not at liberty to say exactly where that is at this time, but we do expect it to be more patient, additional follow-up.
Unknown Executive: But we do expect it to be more patients, and additional follow-up. And, as you know, it was exciting data, so we're eagerly anticipating that as well. The next question is from the line of Phil Nadeau from TD Cowen. Your line is now open. In terms of the pre-launch period, do they wish they had less time or more time in advance of an approval? I think the answer is always that they wish they had more time.
Malcolm Kuno: And, as you know, it was exciting data, so we're eagerly anticipating that as well.
Speaker Change: Great, thank you.
Ernie Rodriguez: I think this is Ernie Rodriguez for Phil. Thanks for taking our questions. I have two quick ones. The first one is, given the overlap in the target treatment centers between Revu and Axan, all the commercial prep work that you've been doing,
Malcolm Kuno: Did you see any benefit to the now sort of like delay or longer time between the two launches? Like can you see perhaps that...
Ernie Rodriguez: Maybe any benefit or any learnings that you can use to later accelerate the
Speaker Change: The launch came to a
Ernie Rodriguez: Relative to what your internal estimates were. And then the second question is,
Speaker Change: Thanks for the questions. First I'll ask Neil to address the trial question that you asked and then Steve will address your commercial question.
Neil Gallagher: Thanks. I think you're asking about the Intercept Trial. So you're right, the Intercept Trial has been ongoing for some time. The trial is progressing well. As you are also aware, it is a third-party trial and therefore we don't control disclosure of data, but our understanding is that
Speaker Change: That recruitment is going relatively well. And, and, you know, we expect that the investigators will present data at a medical meeting in the future, but we don't have an exact timing on that. And with that, I'll pass over to Steve for your first question, actually.
Steve Closter: So just to reiterate, the first question was really, was there a benefit in an extra time, particularly the overlap of the two compounds. So I think just speaking and really speaking from experience, there's never been a launch where
Steve Closter: Somewhat, you know, in terms of the pre-launch period, do they wish they had less time or more time in advance of an approval? I think the answer is always they wish they had more time. So we obviously have more time in revumentum than we would have otherwise anticipated.
Unknown Executive: We obviously have more time in regulatory than we would otherwise have anticipated. The activity that I described before will enable us, with all the profiling and the mechanisms of disease and disease state awareness work we're doing, we'll put to good use over however long it takes us to get approval. And the 2,000 accounts that I mentioned, there's a good chance we will be at all of them. It's not most of them, and we'll be there multiple times.
Speaker Change: The activity that I described before will enable us, you know, the profiling and the mechanisms of disease and disease state awareness work we're doing.
Speaker Change: We'll put to good use over the however long it takes us to get to approval, and the 2,000 accounts that I mentioned, there's a good chance we will be at all of them, if not most of them, and we'll be there multiple times, so I think that
Unknown Executive: So I think that we'll be able to leverage all that time. And I think the point made is that there is tremendous overlap with the accessibility opportunity. I don't think I shared the number of treatment centers for chronic GVHD, but we essentially cover them all by covering all of the revumentum opportunities.
Unknown Executive: And what I mean by that is, there are 2,000 centers, both academic and community, that we're covering for AML and revumentum, and there are under 200 that you cover for transplant to support axopilumab. So we're already in those, essentially. And we're profiling those accounts as well.
Speaker Change: We essentially cover them all by covering all of the revenue opportunities. What I mean by that there's
Speaker Change: centers, both academic and community, that we're covering for AML and RevuMed. There's under 200 that you cover for transplant to support axopitilumab. So we're already in those, essentially, so we're profiling those accounts as well. There's a lot of connectivity between hemogs.
Unknown Executive: There's a lot of connectivity between HEMOX and transplanters, and there are actually some advantages with REV, particularly as patients hopefully move to our transplant and potentially go on continued treatment. So we've always seen that, strategically, both drugs are highly aligned to what we're trying to do as an organization. Same call point.
Unknown Executive: So we can really accomplish both. And we'll, like I said, make use of and leverage the time we have from now till either product comes to market. Thank you, guys. That's helpful.
Speaker Change: It's the same call point, so we can really accomplish both, and we'll, like I said, make use and leverage the time we have from now until either product comes to market.
Speaker Change: Thank you. That's helpful.
Operator: Thank you. Our next question comes from the line of Peter Lawson from Barclays. Your line is now open. Hey, good afternoon.
Speaker Change: Thank you.
Unknown Executive: This is Alex on behalf of Peter from Barclays. Thanks for taking our questions. Just a quick clarification for me on the BEAT AML study. Do we expect more data from this study later this year? And then a separate question is, do we see, could we see initial phase one data from your seven plus three combination in 2024? Alex, thanks for the question. So for BEAT AML, I think we obviously mentioned that this was not our trial.
Speaker Change: Hey, good afternoon. This is Alex on for Peter from Barclays. Thanks for taking our questions. Just a quick clarification for me on the BAML study.
Speaker Change: Do we see more data from this study later this year? And then a separate question is, could we see initial Phase I data from your 7 plus 3 combination in 2024?
Unknown Executive: We are collaborating closely with the sponsor, but the last update was done at EHA, so that was a pretty comprehensive update. We don't have knowledge yet when that's going to be updated. It's possible it could come in the second half of this year at one of the medical meetings, but we don't have perfect information there. So stay tuned on that. And then for the 7 plus 3 phase 1 that we've initiated, we haven't given guidance yet as to when we're going to be putting together the initial data for that. So stay tuned. I wouldn't expect that we would have it in 2024.
Speaker Change: Alex, thanks for the question. So for B2AML,
Speaker Change: I think we had obviously mentioned that this is this is not our trial. We are collaborating closely with this with the sponsor but the
Speaker Change: The last update was done at EHA, so that was a pretty comprehensive update. We don't have knowledge yet when that's going to be updated. It's possible it could come in the second half of this year at one of the medical meetings.
Speaker Change: But we don't have we don't have perfect information there. So stay tuned on that. And then for the seven plus three phase one that we've initiated, we haven't given guidance yet as to when we were going to be.
Speaker Change: Putting together the initial data for that. So stay tuned. I wouldn't expect that we would have it in 2024. That's likely to come in 2025.
Unknown Executive: That's likely to come in 2025. Okay, great. And just, if I may, a quick follow-up. Do you have to enroll sort of, you know, adverse risk patients here initially? Or, you know, what types of patients are being enrolled in Phase I? Thank you. Yeah, thanks, Brad.
Speaker Change: Okay, great. And just, if I may, a quick follow-up. Do you have to enroll sort of, you know, adverse risk patients here initially? Or, you know, what types of patients are being enrolled in the Phase I? Thank you.
Speaker Change: Thank you.
Speaker Change: Our next question comes from the line of Brad Canino from Stiefel. Your line is now open.
Brad Canino: Oh hi, thank you, this is Brad.
Speaker Change: With our tour and the close FDA collaboration that that provides, plus your rampant commercial and vocal about it, that you had really good visibility about what you needed to get this across the line, either at or well before the PDUFA. So, can you help us understand a bit of what happened here?
Unknown Executive: And I think we were able to catch up with many of you earlier in the week when the news came out. Look, I think this was unfortunate in the sense that we were in receipt of a few RFIs, and this is an ongoing process under RTOR that's been going on since last year, when we submitted our NDA. Since then, we've been receiving requests for information from the agency. The two, I'd say, two latest RFIs resulted in quite a bit of information going back to the agency.
Brad Canino: Yeah, thanks, Brad.
Speaker Change: And I think we were able to catch up with many of you earlier in the week when the news came out. Look, I think this was unfortunate in the sense that we were in receipt of
Speaker Change: A few RFIs, and this is an ongoing process under RTOR that's been going on since last year, where we submitted our NDA and since then we've been receiving requests for information from the agency. The two, I'd say, two latest RFIs were
Unknown Executive: Once they received that information, they let us know as of last Friday, late last Friday, that that would require them to actually spend more time on the supplementary information, and they were going to extend the clock under a major amendment. That's a three-month delay.
Speaker Change: Resulted in quite a bit of information going back to the agency. Once they received that information, they let us know as of last Friday, late last Friday, that that
Unknown Executive: So that was, and you're right, we were actually quite collaborative for many months with the agency and have been working very closely with them. The information that we provided could have been reviewed within the time of the PDUFA, and so for us, we were quite confident that that was the process that we were following. We were a little bit surprised that the agency would extend the clock, but they did. That's up to them. That's their decision.
Speaker Change: That was, and you're right, we were actually, you know, quite collaborative over many months with the agency and have been working very closely with them.
Speaker Change: The information that we provided, we believe, could have been reviewed within the time of the PDUFA, and so for us, we were quite confident that that was the process that we were following. We were a little bit surprised that the agency would extend the clock, but they did. That's up to them. That's their decision.
Unknown Executive: So, look, this is information that, as I previously said, Yeah, maybe just to follow on that, because I think many of us are now looking at a situation where there's a potential for heightened asymmetry of information. You've got the FDA potentially looking at data that we haven't been able to vet. I wonder, what assurance can you give that the overall profile that we know publicly from Revumentib is consistent and that prior data cut remains reliable? Thank you.
Speaker Change: So look, this is information that, as I previously said, this is information that...
Speaker Change: that we did not have in the NDA. It was not requested of us at the time of the submission of the NDA.
Speaker Change: It was new information, new clinical information that they wanted to review, and so we provided that.
Speaker Change: This is something that we were prepared for, but they didn't ask for it, and we provided it when they did ask for it.
Speaker Change: The only unfortunate part of this is that they, not that they asked for it, but we...
Speaker Change: learned about it sort of late in the review cycle. We had gone through our mid-cycle and our late-cycle review.
Speaker Change: And it was, you know...
Speaker Change: without events, so we were feeling quite confident.
Speaker Change: In everything that we have not only been through with the agency, but what we've submitted and heard back.
Speaker Change: Those are the sequence of events, and we feel very confident that this drug will get approved.
Speaker Change: On the December timeline, as they've extended the date, so we're in, I think, a very good position to launch the drug before the end of the year.
Speaker Change: There's a potential for heightened asymmetry of information. You've got the FDA potentially looking at data that we haven't been able to vet.
Speaker Change: I guess, what assurance can you give that the overall profile that we know publicly from Revumentiv is consistent and that prior data cut remains reliable? Thank you.
Speaker Change: Yeah, thanks Brad. Look, I think the information, and we don't get into the specifics of any one RFI and what's being asked of us in an ongoing review, but what I can say at the
Speaker Change: At the late cycle review, we did learn that there's going to be no adcom for the product.
Speaker Change: This is not information that changes the profile of the risk-benefit at all. And so we feel, actually, it's even more robust of a package than as previously.
Speaker Change: worked on is basically what the agency had asked for, and they asked for supplemental information. So ...
Speaker Change: We feel like it doesn't change anything at all having to do with the profile or the risk-benefit.
Speaker Change: And for that reason, that's why we're so confident that the draws will not only get approved, but it'll get approved on the timeline as proposed. So I think that's as detailed as I could get for you, Brad.
Operator: The next question comes from the line of Michael Schmidt from Guggenheim. Please go ahead. Yeah, Michael, thanks for the question. It's not unprecedented, for even with the extensions, we've seen a couple of Artur products, Resiroc being one that got approved under Artur, got extended, actually got approved about six weeks earlier than their extended timeframe, or their extended PDUFA.
Speaker Change: The next question comes from the line of Michael Schmidt from Guggenheim. Please go ahead.
Michael Schmidt: Hey guys, thanks for taking my question. Um, maybe just a quick follow-up. So, given that the Revumanib NDA obviously is still under RTOR review and
Speaker Change: with the extended PDUFA date until the end of September , now giving ample time for the FDA.
Speaker Change: Just wondering what your view is on the potential for an approval to come ahead of that extended PDUFA date. Is that still on the table in your opinion? And then I had an unrelated follow-up.
Speaker Change: under our tour got extended actually got approved about six weeks earlier than their extended time frame or their extended PDUFA. So we've seen
Unknown Executive: So we've seen Artur products get extended and get approved. Many products under priority review, same thing. Most of the time they go the distance, meaning that they're approved on or around their extended PDUFA date. It's very hard to know if that will or will not be the case for us.
Unknown Executive: We do expect it to be on or before, of course, but we can't, it's very hard to judge whether that's, you know, very removed from the extended date itself. So our expectation is that it will come on or before, on or around the date that they've, in terms of the percentage of patients who go on to transplant and then potentially even go back on drugs post-transplant, even though they happen to be a slightly older patient population.
Speaker Change: It's very hard to know if that will or will not be the case for us. We do expect it to be on a report, of course, but we can't. It's very hard to judge whether that's.
Michael Schmidt: you know, very removed from the extended date itself. So our expectation is that it will come on or before, on or around the date that they've specified.
Speaker Change: Unrelated follow-up, just thinking about the upcoming registration data in MNPM1 patients later this year.
Speaker Change: We've seen in the phase one trial looks very similar.
Speaker Change: [inaudible]
Speaker Change: We've also seen a good proportion of those patients get transplanted.
Unknown Executive: So I think that's, to the best of our knowledge, based on information that we've seen from phase one and now in our pivotal trial for cancer. Great, thank you. Thanks, Michael. Our next question comes from the line of Kelly Shee from Jefferies. Your line is now open. Hi, this is Clara. I'm for Kelly.
Unknown Executive: Thanks for taking our question. Now that you will likely have pivotal data readout in MPM-1 that had approval in KMT-2A, I was wondering how that possibly would change your discussion with the NCCN Committee for Guideline Inclusion? And maybe, you know, one step forward, also, what will the conversation with payers be like? as well.
Unknown Executive: Thank you. Yeah, Claire, thanks for the question. Look, I think the cadence of events here in our minds seems to be that we will have NPM1 data now in our pivotal trial ahead of our KMT2A approval. They'll be in close proximity to one another, but it's no longer the other way around.
Speaker Change: Clare, thanks for the question. Look, I think the cadence of events here in our mind seems to be that we will have NPM1 data now on our pivotal trial ahead of our KMT2A approval.
Speaker Change: They'll be in close proximity to one another, but it's no longer the other way around. So, having NPM1 data first are...
Unknown Executive: So having NPM1 data first, our priority will be to get that data published as quickly as we can. And once the drug is approved in KMT2A, we can, you know, proceed with potential guideline inclusion for NPM1. So that's the sort of cadence here, and there will be some urgency to move in order to affect the fact that we have a drug on the market and then another indication in NPM1, which is moving through the SNDA process quickly at the same time but could be included in guidelines ahead of approval.
Speaker Change: The priority will be to get that data published as quickly as we can, and once the drug is approved in KMT2A, we can proceed with potential guideline inclusion for MPM1. So that's the sort of cadence here, and there'll be some urgency to move.
Speaker Change: in order to affect the fact that we have we have a drug on the market and then another indication in NPM1 which is moving through quickly through an SNDA process at the same time but could be included in in guidelines ahead of ahead of approval. So there will be there will be, you know
Unknown Executive: So there will be, you know, quite a bit of activity related to not only getting the drug approved but also moving guidelines through publications and conversations with thought leaders who work, you know, on an ad hoc basis, if you will, to get new drugs included in guidelines. Maybe I'll turn it over to Steve to talk about the other.
Speaker Change: Quite a bit of activity related to not only getting the drug approved, but also moving guidelines through publications and conversations with
Speaker Change: with thought leaders who work on an ad hoc basis, if you will, to get new drugs included in guidelines. Maybe I'll turn it over to Steve to talk about the other questions.
Unknown Executive: Yeah, I think part two is, you know, what is the impact on payers of NPM1? So, you guys, in my opening comments, talked about our activity with payers, which has been extensive. You know, we'll have talked to payers that cover 90% of managed care lives in both commercial and Part D. I think payers recognize the unmet need in KNP2AR and they recognize it in NPM1. They like what they've seen with Revumentib and, you know, given the price point in the space of roughly $27,000 to $32,000 a month, we believe we'll be at a slight premium to those agents. You know, there is an obvious connection between NCCM guidelines and payers. Payers like to see what's in the public domain.
Steve Closter: Yeah, I think part two is, you know, what is the impact on payers of NPM1? So you guys, in my opening comments, had talked about our activity with payers, which has been extensive.
Steve Closter: We'll have talked to payers that cover 90% of managed care lives in both commercial and Part D.
Speaker Change: I think payers, they recognize the unmet need in KMT-2AR and they recognize it in NPM-1. They like what they've seen with Revumentib and, you know, given the price point in the space of roughly $27,000 to $32,000 a month, we believe we'll be at a
Speaker Change: You know, it's an obvious connection between NCCM guidelines and payers. Payers like to see what's in the public domain. They want to see, you know, data published. They want to see, you know, the guidelines. Clearly, the entree point is KNC2AR. That's where we'll set price.
Unknown Executive: They want to see, you know, data published. They want to see, you know, the guidelines. Clearly, the entry point is KNC2AR.
Unknown Executive: That's where we'll set the price. I think payers, you know, want to serve as patients. You know, they'll produce, you know, some hurdles and obstacles upfront and barriers, which they always do with products like this. You know, we've got, you know, fantastic specialty pharmacy partners and distributors and a patient portal when needed to usher patients through the early days of a launch when products are not yet on formulary. So we understand the medical exception process, as well as our preferred pharmacy partners, as well as, you know, doctors and institutions.
Unknown Executive: So we think either way, we'll be able to set the right price, and we'll be able to get patients on the drug, you know, with some challenge. But we'll get there, and we're prepared to do it. Thanks, Claire. Next question comes from Yigal Nochomovitz from Citi. Your line is now open. Hi guys, this is Ashiq Mubarack.
Speaker Change: Thank you, that was very helpful.
Speaker Change: The next question comes from Yigal Nochomovitz from Citi. Your line is now open.
Unknown Executive: I'm free to go. Thanks for taking my questions. I just wanted to ask one on Axatilumab. Sounds like everything is on track with the PDUFA, which is coming up pretty soon. I just wanted to know if you could share any color on maybe where things are in the sort of BLA process. Have you reached label negotiations? Was there anything of value with maybe a mid cycle review?
Speaker Change: Hi guys, this is Ashiq Mubarack. I'm free to go. Thanks for taking my questions. I just wanted to ask one on axotilamide.
Ashif Mubarak: Sounds like everything is on track with the PDUFA, which is coming up pretty soon. I just wondered if you could share any color on...
Speaker Change: Maybe where things are in the sort of BLA process. Have you reached label negotiations? Was there anything of value with the maybe a mid-cycle review? Anything to sort of confirm that that BLA is on track would be very helpful. Thank you.
Unknown Executive: Anything to sort of confirm that the BLA is on track would be very helpful. Thank you. Thank you for the question. We don't comment on any specific part of our negotiations or discussions with the agency during the process.
Speaker Change: Any specific part of our negotiations or discussions with the agency during the process.
Speaker Change: It is under priority review, as you remember.
Speaker Change: As we said along, it's going well. We do expect the drug to be approved around the PDUFA date, which is the end of August .
Speaker Change: Nothing that we've encountered in the review process, you know, changes that point of view. I won't comment specifically around whether we are or we are not in label discussions, but we are feeling very confident that the drug will be approved and will be approved on its timeline.
Unknown Executive: It is under priority review, as you remember. As we said earlier, it's going well. We do expect the drug to be approved around the PDUFA date, which is the end of August. Nothing that we've encountered in the review process changes that point of view.
Speaker Change: Okay, maybe one follow-up on that then. Should we sort of expect the commercial launch to take place maybe immediately after a potential approval of Axitelimab, or is there a reason to sort of think there might be a little bit of a lag, maybe waiting for Revimanib, so there's some synergy with that commercial launch as well? Thanks.
Unknown Executive: Right, so no, thanks for the follow-up, Ashiq. We said that the launch is expected in the fourth quarter. So that's our guidance. We're working with our partner to get ready to do that. And we'll launch the drug. Got it.
Speaker Change: Thanks for the follow-up, Ashiq. We said that the launch is expected in the fourth quarter, so that's our guidance. We're working with our partner to get ready to do that, and we'll launch the drug when it's ready.
Unknown Executive: Thank you very much. Thank you. Our next question comes from the line of Kalpit Patel from B Riley. Your line is now open.
Speaker Change: The KMT-2AR regulatory filing here.
Speaker Change: That could be beneficial for the NPM1 filing and help streamline that. I know the timeline says first half 25 instead of maybe first quarter 25. Is there a reason for that? And then I have a follow-up.
Speaker Change: Yeah, thanks, Cal. Let me turn it over to Neil to maybe take a crack at that.
Unknown Executive: Yeah, thanks for the question. So, in the NDA, we do, you know, a lot of the heavy lifting is done, and any subsequent application, supplementary application, is much more straightforward. So, you know, we're obviously excited and looking forward to getting the NPM1 data during the fourth quarter, and you can be assured that we will be, we will be filing or submitting those data as expeditiously as possible. I, I, there was a part of your question I missed. Yeah, yeah.
Neil Gallagher: Yeah, thanks for the question. So,
Neil Gallagher: The straightforward answer to your question is yes, right? I think that we've learned a lot about the process, and of course, it's important to draw the distinction between the NDA and the SMDA.
Neil Gallagher: So in the NDA we do, you know, a lot of the heavy lifting is done and any subsequent application, supplementary application is much more straightforward.
Neil Gallagher: So, you know, we're obviously excited and looking forward to getting the NPM1 data during the fourth quarter, and you can be assured that we will be, we will be filing or submitting those data as expeditiously as possible. There was a part of your question I missed.
Neil Gallagher: Did I address all of your questions or was there something I missed?
Speaker Change: Yeah, it just it says first half 25 instead of first quarter 25 for filing for the NPM1 cohort.
Neil Gallagher: Yeah, we haven't been that specific with our guidance yet, but you can be assured that we will be working pretty hard at it. With that, I'll pass it back to Michael.
Unknown Executive: So, and then the second question is sort of related here. Was there any information that was requested by the FDA? I guess was any part of the NPM1 cohort data set that was sent to the FDA for this update? Okay, thanks for taking the question. Thank you. Justin, thanks for the question. I don't think you should assume for a second that this is going to be approved in any other way other than full approval.
Michael: Is that your second question?
al: The second question is sort of related here. Was there any information that was requested by the FDA? I guess was any part of the NPM1 cohort data set that was sent to the FDA for this update?
Michael: Kalpit, I would actually just focus on the KMT2A submission. That's what we're working with the FDA on now. NPM1 data hasn't even been, you know, we're not even through the trial. All of it hasn't been done yet. So when that trial is available or when the data is available, we'll publish it.
Michael: And that will be in the fourth quarter. So you should, the information that we're focused on with the agency relates to KMT2A.
Speaker Change: Okay, thanks for taking the questions.
Speaker Change: Thank you.
Speaker Change: Our next question comes from the line of Justin Zelin from BTIG. Your line is now open.
Justin Zelin: Thanks for taking our question. Michael, I'd like to clarify regarding your confidence on the revimentum approval by the end of the year. Should we continue to expect a traditional approval here or could there be a scenario in the review cycle where revimentum could be approved under an accelerated approval basis?
Michael: Justin, thanks for the question. I don't think you should assume for a second that this is going to be approved in any other way other than
Unknown Executive: All of the precedent drugs that have gotten approved in AML as monotherapy targeted therapies have gone through the exact same process. And so that's never been discussed with the agency that we would have an accelerated approval pathway, and that's not, that's not the pathway we're pursuing here. So I would not assume that that would change at all.
Speaker Change: precedent drugs that have gotten approved in AML as monotherapy targeted therapies have been
Unknown Executive: And we have high confidence that this is just a delay of three months. It doesn't change the process at all in terms of what we're filing for, and what we're likely to have at a. Great. Thanks for clarifying and taking our question. The next question comes from the line of Jason Zemansky from Bank of America. Your line is open. Good afternoon, and this is Cameron Bozdog on behalf of Jason.
Justin: Great. Thanks for clarifying and taking our question.
Justin: Thanks, Justin.
Speaker Change: The next question comes from the line of Jason Zemansky from Bank of America. Your line is open.
Unknown Executive: Congratulations on the progress, and thanks so much for taking our question. So looking ahead to the potential near-term launch of Axitelimab, can you maybe comment on your internal market analysis and what you've been hearing from prescribers? I mean, do you expect uptake, at least initially, to be largely driven by academic prescribers centered at transplant centers versus community docs, especially when you think about the route to administration? Great, thanks for the question.
Jason Zemansky: Do you expect uptake, at least initially, to be largely driven by academic prescribers centered at transplant centers versus community docs, especially when you think about the route to administration?
Unknown Executive: I'm going to turn it over to Steve. Yeah, thanks for the question, Kim. You know, I'd start off saying it's a pretty small audience. I mean, there's a lot of high science and experts in the field of transplantation. So, from a treatment center standpoint, I think I may have provided the numbers earlier, 10% of the centers are under 200. So, it is, it's all driven by that. I think the part of who the drivers are, academic versus, you know, community, it's the biggest centers that are going to drive all this.
Speaker Change: Great. Thanks for the question. I'm going to turn it over to Steve to address it.
Steve Closter: It's from a treatment center standpoint. I think I may have provided the numbers early. It's 10% of the centers. It's under 200 So it is it all it's all driven by that I think in the part of who the drivers academic versus, you know community, it's the biggest centers are going to drive all this
Unknown Executive: For the most part, certainly at launch, there'll be some awareness outside of that. And even within the 200 centers, there's probably 35 that see half the patients in the country. So, it is as, you know, I wouldn't say it's the smallest physician audience to call, but it's one of the smallest.
Speaker Change: And even within the 200th center, there's probably 35 that see half the patients in the country.
Michael: It is as, you know, I wouldn't say it's the smallest physician audience to call in, but it's one of the smallest.
Unknown Executive: And it's a tight community. I think, speaking to market opportunity, there's a lot of business here. There's a lot of unmet need and dissatisfaction in the current treatment for the GVHD population. They want more; they need more agents. It's a really insidious disease.
Unknown Executive: So, it'll be, you know, I'd say early uptake and a group of patients probably that are waiting for something like this. It's not a huge number, but there are some numbers of patients waiting. So, there'll be some form of bullets, but the market is ready and will be ready shortly, too, and I think it'll be well received by physicians and patients. And our final question comes from the line of George Farmer from Scotiabank. Your line is open. Hi, this is Chloe on behalf of George. Can you hear me?
Operator: Okay. I hear you, Chloe. Thank you. Yeah, so two from us. So, based on your initial data package for review minutes, you said that FDA did its thing and ODAQ was necessary. Do you still believe that to be the case now?
Michael: Hi, this is Chloe Yonker-George. Can you hear me okay?
Unknown Executive: How confident are you that the additional information you submitted that prompted the proof extension will not be subject of an ODAQ review in the next case? And the second question is, Michael, you mentioned it was a prepared remark, so I'm just curious. What types of BD deals you have in mind, any examples of areas of interest or types of assets you're eyeing at the moment, both as a buyer or even a target, and if you could maybe put it in context to be the insight relationship for us and tell us kind of how you see that evolving over time. Chloe, thanks for the question.
Chloe Yonker-George: Yeah, so choose from us.
Speaker Change: At the moment, both as a buyer or even a target, and if you could maybe put it in context to give the insight relationship for us and tell us kind of how you see that evolving over time.
Unknown Executive: So first, in terms of ODAC, I think there is a high degree of confidence that an ODAC will not be required. The agency, on more than one occasion, has told us this. The data that we submitted, as I referenced earlier, is only, in our minds, and very clearly supportive of approval and only augments the package that we've submitted. So there's really, in our minds, and based on what we've heard from the agency, there doesn't seem to be any risk of an ODAC that we know of. So I feel quite confident about that.
Speaker Change: Chloe, thanks for the question. So first in terms of ODAC, I think
Speaker Change: The package that we've submitted so there's really there is in our minds and based on what we've heard from the agency There is doesn't seem to be any risk of an ODAC and that we know of so I feel quite confident about that
Unknown Executive: And in terms of BD, which is an interesting question, it essentially relates to how we grow the pipeline and continue doing what we do well at Syndax, in part, which is in licensing or acquiring new molecules to add to the pipeline. As we've said in the past, and we continue to work on adding or backfilling the pipeline as early-stage molecules in the targeted therapy space in oncology, focused on oncology, we don't speak specifically about which mechanisms we're going after or the status of any of these discussions and what, when we conclude our deals, we usually bring them forward and talk about them publicly.
Speaker Change: in Oncology, he's focused on oncology. We don't speak specifically about which mechanisms we're going after or the status of any of these discussions and what ...
Unknown Executive: But at this stage, we are very actively looking and feel quite good about the potential. But at this point, it's hard to speculate on business development transactions until they're done and closed. But whatever we do will be, it seems, of an earlier variety. So we're not looking to take on late-stage projects. Earlier projects are the type of inflection of what we feel like we can accommodate at this time with all the other interesting things we have going on in our pipeline for axotilumab and for revimenib. It's a balanced strategy and one that we are actively pursuing. Okay, thanks.
Unknown Executive: With respect to the partnership with Insight, do you have any thoughts on how you see that evolving in the future? Our partnership with Insight is a good one. We conceptualized it back in 2021 when we announced it.
Unknown Executive: They've been good partners to us, and as Steve outlined, and we've talked about, we're keen to launch that, get that product approved and launched, and certainly expand the use of Axotelimab in a variety of indications. Earlier in the treatment course for GVHD, we're pursuing IPF, so that's, you know, this is a global partnership with Insight, and so it's gone quite well, and we, you know, continue to believe that it will expand and continue in perpetuity in the way we envisioned it to start.
Speaker Change: Our partnership with Insight is a good one. We conceived it back in 2021 when we announced that. They've been good partners to us.
Unknown Executive: We're very happy with that. Thank you. Thank you, Klahre. This concludes the question and answer session. I'll now turn the floor over to Mr. Michael Metzger for any additional comments or closing remarks. Yeah, thank you. Thank you all. We appreciate you tuning in today to discuss the progress we have made and the exciting milestones ahead for the company. We look forward to seeing you at our planned investor events, including the upcoming BTIG conference in August. And with that, I wish you a good day. Thank you so much.
Chloe: Thank you, Chloe.
Speaker Change: Thank you all. We appreciate you all tuning in today to discuss the progress we have made and the exciting milestones ahead for the company. We look forward to seeing you at our planned investor events, including the upcoming BTIG conference in August . And with that, we wish you a good day. Thank you so much.