Summary of Syndax Pharmaceuticals Conference Call Company Overview - **Company**: Syndax Pharmaceuticals - **Key Products**: RevuForge (Menin inhibitor for AML) and Nictimvo (for chronic GVHD) - **Market Potential**: - RevuForge: Over $5 billion market opportunity - Nictimvo: Over $3 billion market opportunity - **Current Status**: Both products are generating revenue and have FDA approval, with early launches showing positive results [3][4][7]. Core Points and Arguments RevuForge - **Product Profile**: First-in-class Menin inhibitor with a significant first-mover advantage in the market [3]. - **Clinical Data**: - Phase 1b study confirmed a dose of 160 mg in combination with Venaza, showing good tolerability and a 67% complete response (CR) rate compared to 37% historical control [11][13]. - 100% of evaluable patients achieved minimal residual disease (MRD) negativity, indicating strong efficacy [14]. - **Market Position**: Anticipated inclusion in NCCN guidelines ahead of approval, which could enhance market penetration [24][44]. Nictimvo - **Clinical Data**: - Updated data to be presented at EHA, showing a 26% CR rate and a 48% overall response rate in a larger patient population [21][22]. - Rapid symptom improvement across organ systems in chronic GVHD patients [47][48]. - **Sales Performance**: Strong initial sales with $13.6 million in net revenue for the first two months post-launch [49]. Additional Important Insights - **Regulatory Strategy**: Plans to use CR and MRD negativity as potential endpoints for accelerated approval in clinical trials [35][36]. - **Launch Metrics**: - RevuForge generated $20 million in its first full quarter, with 90% of sales from KMT2A patients and expectations for growth as NPM1 reimbursement is secured [37][42]. - High coverage rates with 72% of managed care lives covered, indicating strong market access [39]. - **Competitive Landscape**: Syndax is currently the only approved Menin inhibitor, with a best-in-class profile that physicians favor [29][30][31]. Conclusion Syndax Pharmaceuticals is well-positioned in the oncology market with its innovative products, strong clinical data, and strategic regulatory plans. The company is focused on expanding its market presence and enhancing its product offerings, which could lead to significant growth in the coming years [5][6][34].

Summary of Syndax Pharmaceuticals Conference Call Company Overview - **Company**: Syndax Pharmaceuticals - **Industry**: Biotechnology, specifically focused on oncology and hematology treatments Key Points and Arguments Product Launches - **REVUFORGE**: - Indicated for KMT2A acute leukemia in adults and pediatrics - First in market as a menin inhibitor with a best-in-class profile - Early launch results are encouraging, with outpacing expectations in the first quarter [3][5] - **Nyktymbo**: - A CSF-1R antibody for chronic graft-versus-host disease (GVHD) - First of its kind, launched this year with a new mechanism of action - Partnership with Insight for promotion and development [4][5] Market Performance - **REVUFORGE** has seen a rapid increase in prescriber base, reaching 50% of Tier one and Tier two prescribers by March [25] - Formulary coverage has improved significantly, with nearly complete coverage reported [27] - 80% of patients are initiated on the drug within a week, which is critical for this urgent patient population [29] Clinical Data and Studies - **EHA Conference**: - Syndax will present 10 abstracts, including updates on KMT2A and NPM1 data [14][12] - The BEAT AML study shows a 67% complete response rate when combining REVUFORGE with standard therapies, significantly higher than the historical benchmark of 37% [18][19] - **NPM1 Indication**: - An sNDA has been submitted, with a 26% complete response rate reported in the relevant population [40][44] - Anticipation of rapid regulatory approval due to the existing market presence of REVUFORGE [45][48] Future Growth and Strategy - **KMT2A Launch**: - Expected to penetrate 50% of the KMT2A population in the first year, with additional growth anticipated from the NPM1 indication [32][34] - **Combination Therapies**: - Ongoing studies in both relapse refractory and frontline settings, with a focus on KMT2A and NPM1 patients [52][56] - Plans to establish two randomized studies for KMT2A and NPM1 patients to confirm benefits of combination therapies [56] Nyktymbo's Market Potential - Initial launch focused on fourth-line GVHD patients, with potential to expand into earlier lines of treatment and other indications like idiopathic pulmonary fibrosis (IPF) [70][71] - The drug's unique profile and mechanism of action position it well for future growth opportunities [70] Additional Important Insights - The company is well-positioned to leverage its first-mover advantage in the menin inhibitor market, with a strong focus on patient outcomes and innovative treatment approaches [41][44] - Regulatory support for accelerated approvals is seen as a gradual shift, with the agency open to using surrogate endpoints for efficacy [64][65] This summary encapsulates the key discussions and insights from the conference call, highlighting Syndax Pharmaceuticals' strategic direction, product performance, and market opportunities.

Core Insights - Syndax Pharmaceuticals announced the acceptance of multiple abstracts for clinical data presentations of Revuforj® (revumenib) and Niktimvo™ (axatilimab-csfr) at the 30th European Hematology Association (EHA) Annual Congress Meeting scheduled for June 12-15, 2025, in Milan, Italy [1][2] Group 1: Revuforj® (revumenib) - Revuforj is a first-in-class selective menin inhibitor approved for treating relapsed or refractory acute leukemia with KMT2A translocation in patients aged one year and older [10][11] - The BEAT AML frontline trial data will be presented, showcasing the combination of revumenib with venetoclax and azacitidine in patients with mNPM1 and KMT2Ar AML [2][6] - Compelling results in acute leukemia across various genetic populations, including mNPM1 and KMT2Ar, will be highlighted [5][6] Group 2: Niktimvo™ (axatilimab-csfr) - Niktimvo is a first-in-class CSF-1R-blocking antibody approved for chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy [12][35] - The AGAVE-201 trial data will be presented, demonstrating robust responses in different organs and patient subgroups with chronic GVHD [5][8] - Ongoing studies include a Phase 2 combination trial with ruxolitinib and a Phase 3 trial with steroids for chronic GVHD [14][35]

Core Insights - Syndax Pharmaceuticals has appointed Dr. Nicholas Botwood as the new Head of Research and Development and Chief Medical Officer, bringing over 25 years of experience in oncology drug development [1][2] - Dr. Botwood previously held senior roles at Bristol Myers Squibb, where he led medical oncology and was responsible for the medical strategy in the U.S. [2][3] - The company aims to leverage Dr. Botwood's expertise to accelerate the growth of its oncology franchises and enhance its R&D strategy [2][3] Company Overview - Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company focused on innovative cancer therapies [4] - The company's pipeline includes FDA-approved products such as Revuforj® (revumenib) and Niktimvo™ (axatilimab-csfr) [4] - Syndax is committed to advancing cancer care through ongoing clinical trials and aims to unlock the full potential of its product pipeline [4]

Core Viewpoint - Syndax Pharmaceuticals (SNDX) shows significant upside potential with a mean price target of $35.23, indicating a 220% increase from the current price of $11.01 [1] Price Targets and Estimates - The mean estimate consists of 13 short-term price targets with a standard deviation of $10.34, suggesting variability among analysts [2] - The lowest estimate of $17 indicates a 54.4% increase, while the highest estimate predicts a surge of 363.2% to $51 [2] - A low standard deviation indicates a high degree of agreement among analysts regarding price movement [9] Analyst Sentiment and Earnings Estimates - Analysts have shown increasing optimism about SNDX's earnings prospects, with a positive trend in earnings estimate revisions [11] - The Zacks Consensus Estimate for the current year has risen by 5.6% over the past month, with four estimates increasing and no negative revisions [12] - SNDX holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimates [13] Caution on Price Targets - While price targets are commonly referenced, they can mislead investors, as empirical research shows they rarely indicate actual stock price movements [7][10] - Analysts may set overly optimistic price targets due to business incentives, which can inflate expectations [8]

Core Insights - Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company focused on innovative cancer therapies [2] - The company has FDA-approved products in its pipeline, including Revuforj® (revumenib) and Niktimvo™ (axatilimab-csfr) [2] - Syndax is actively conducting clinical trials to maximize the potential of its pipeline [2] Upcoming Events - Michael A. Metzger, CEO of Syndax, and the management team will participate in investor conferences [1] - A fireside chat at the Bank of America Merrill Lynch Vegas Health Care Conference is scheduled for May 15, 2025, at 9:20 a.m. PT/12:20 p.m. ET [3] - A virtual fireside chat at the TD Cowen 6th Annual Oncology Innovation Summit will take place on May 28, 2025, at 10:30 a.m. ET [3]

Core Insights - Syndax Pharmaceuticals announced positive results from the pivotal Phase 2 portion of the AUGMENT-101 trial for revumenib in relapsed or refractory mutant NPM1 acute myeloid leukemia (mNPM1 AML) patients, achieving nearly 50% overall response rate [1][2] - The company submitted a supplemental New Drug Application (sNDA) for revumenib in April 2025 under the FDA's Real-Time Oncology Review (RTOR) program [1][2] Group 1: Trial Results - The primary efficacy endpoint was met with a complete remission (CR) plus CR with partial hematological recovery (CRh) rate of 23% among the first 64 adult patients [5] - The overall response rate (ORR) was reported at 47%, with 17% of responders proceeding to hematopoietic stem cell transplant (HSCT) while in remission [6] - The median overall survival (OS) for all patients was 4.0 months, while responders had a median OS of 23.3 months [7] Group 2: Patient Demographics and Treatment Background - The efficacy-evaluable population had a median age of 65, with 36% having received three or more prior lines of therapy [4] - 75% of patients were previously treated with venetoclax, indicating a heavily pretreated population [4] Group 3: Safety Profile - The safety population included 84 patients, with treatment-emergent serious adverse events occurring in ≥5% of patients, including febrile neutropenia (21%) and differentiation syndrome (13%) [8] - The safety profile of revumenib was consistent with previously reported data, with 12% of patients experiencing dose reductions due to adverse events [8] Group 4: Background on mNPM1 AML - Mutations in the NPM1 gene are the most common genetic alteration in adult AML, observed in approximately 30% of cases [9] - There are currently no approved targeted therapies specifically for mNPM1 AML, highlighting a significant unmet medical need [9] Group 5: Revumenib Overview - Revumenib is an oral, first-in-class selective menin inhibitor, previously approved for R/R acute leukemia with KMT2A translocation [10][11] - The drug is in development for R/R mNPM1 AML, with ongoing trials planned for combination therapies [11]

Core Viewpoint - Syndax Pharmaceuticals reported a quarterly loss of $0.98 per share, which was better than the Zacks Consensus Estimate of a loss of $1.04, indicating an earnings surprise of 5.77% [1] Financial Performance - The company posted revenues of $20.04 million for the quarter ended March 2025, surpassing the Zacks Consensus Estimate by 36.69%, compared to zero revenues a year ago [2] - The current consensus EPS estimate for the upcoming quarter is -$0.99 on revenues of $17.81 million, and for the current fiscal year, it is -$3.99 on revenues of $81.74 million [7] Stock Performance - Syndax shares have increased by approximately 3.9% since the beginning of the year, while the S&P 500 has declined by 3.3% [3] - The stock currently holds a Zacks Rank 3 (Hold), indicating it is expected to perform in line with the market in the near future [6] Industry Outlook - The Medical - Biomedical and Genetics industry, to which Syndax belongs, is currently in the top 34% of over 250 Zacks industries, suggesting a favorable outlook [8] - Empirical research indicates a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can impact stock performance [5]

Financial Data and Key Metrics Changes - Syndax reported net revenue of $20 million for RevuForge in Q1 2025, marking the first full quarter of its launch [8][35] - Nictimvo generated $13.6 million in net revenue for the first two months of its launch, with Syndax reporting a collaboration loss of only $200,000 for this period [8][36] - The company maintained a strong financial position with $602.1 million in cash and equivalents as of March 31, 2025 [9][38] Business Line Data and Key Metrics Changes - RevuForge's launch has seen strong adoption, with 44% of tier one and tier two accounts ordering the product as of March, up from one-third in February [16][17] - Nictimvo has been administered in over 1,250 infusions year-to-date, with approximately 95% of top accounts ordering the product [25][26] Market Data and Key Metrics Changes - The current indication for RevuForge targets an estimated 2,000 patients in the U.S. with relapsed or refractory acute leukemia, representing a market opportunity of $750 million [22] - The total addressable market for Nictimvo is estimated to be between $1.5 billion to $2 billion, targeting 6,500 chronic GVHD patients in the U.S. [27][10] Company Strategy and Development Direction - Syndax aims to position RevuForge as the first menin inhibitor included in clinical guidelines for treating relapsed or refractory mutant NPM1 AML [13] - The company is focused on executing strategic launch imperatives to ensure long-term competitive immunity ahead of potential market entrants [23] - The EVOLVE-two trial is a pivotal frontline study for Rebuminav, aiming for accelerated approval and full approval based on dual primary endpoints [29][30] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong commercial opportunities for both RevuForge and Nictimvo, highlighting the unmet medical needs and the compelling profiles of their medicines [8][10] - The company anticipates that RevuForge will be the first menin inhibitor approved in the frontline setting, with ongoing engagement with the FDA on their submissions [11][14] Other Important Information - The company has submitted a supplemental new drug application (sNDA) for RevuForge, seeking priority review for treating relapsed or refractory mutant NPM1 AML [10] - The FDA's real-time oncology review program is expected to facilitate quicker approvals for their submissions [11] Q&A Session Summary Question: What are you seeing regarding repeat prescribers for RevuForge? - Management noted that 44% of tier one and tier two accounts have ordered, with about 80% of those ordering more than once, indicating a growing user base [43][46] Question: Are you seeing a similar pace of patients receiving transplants with RevuForge as in clinical trials? - Management stated that anecdotal information suggests patients are being taken to transplant, but it is too early to provide definitive data [52][53] Question: Can you share any color on month-over-month trends for new patient adds for RevuForge in Q1? - Management indicated that while they are not providing specific numbers yet, they are happy with the steady stream of new patients and refill rates [58][60] Question: What portion of the $20 million revenue stems from refill dynamics versus new patient starts? - Management mentioned that both refill and new patient dynamics are building, but specific data is still maturing [101][102]

Financial Data and Key Metrics Changes - Syndax Pharmaceuticals reported net revenue of $20 million for RevuForge in Q1 2025, marking the first full quarter of its launch [6][35] - Nictimvo achieved net revenue of $13.6 million in the first two months of its launch, with Syndax reporting a collaboration loss of only $200,000 for this period [6][36] - The company maintained a strong financial position with $602.1 million in cash and equivalents as of March 31, 2025 [7][39] Business Line Data and Key Metrics Changes - RevuForge's launch has seen strong adoption, with 44% of tier one and tier two accounts ordering the product as of March, up from one-third in February [16] - Nictimvo has been administered in over 1,250 infusions year-to-date, with approximately 95% of top accounts ordering the product [24] Market Data and Key Metrics Changes - The current indication for RevuForge targets an estimated 2,000 patients in the U.S. with relapsed or refractory acute leukemia with a KMT2A translocation, representing a $750 million market opportunity [20] - The total addressable market for Nictimvo is estimated to be between $1.5 billion to $2 billion, targeting 6,500 chronic GVHD patients in the U.S. [10][26] Company Strategy and Development Direction - Syndax aims to position RevuForge as the first menin inhibitor included in clinical guidelines for treating relapsed or refractory mutant NPM1 AML, with plans for a supplemental new drug application (sNDA) to the FDA [8][11] - The company is focused on executing strategic launch imperatives to ensure long-term competitive immunity ahead of potential me-too products entering the market [22] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong commercial opportunities for both RevuForge and Nictimvo, highlighting the high unmet medical need and the compelling profiles of their medicines [5][6] - The company anticipates that RevuForge will be the first menin inhibitor approved in the frontline setting, with ongoing trials expected to support accelerated approval [11][12] Other Important Information - The EVOLVE-two trial, a pivotal frontline trial for a menin inhibitor, has been initiated, targeting newly diagnosed patients with high unmet medical needs [7][28] - The company has seen robust engagement with the FDA regarding its sNDA filing and overall programs [9] Q&A Session Summary Question: What are you seeing regarding repeat prescribers for RevuForge? - Management noted that 44% of tier one and tier two accounts have ordered RevuForge, with about 80% of those having ordered more than once, indicating a growing user base [44][46] Question: Can you comment on patients receiving RevuForge and their transplant rates? - Management stated that anecdotal information suggests patients are being taken to transplant, and they expect patients to return to RevuForge post-engraftment [52][53] Question: What are the month-over-month trends for new patient adds for RevuForge? - Management indicated that while they are not providing specific numbers yet, they are happy with the steady stream of new patients and refill rates [57][60] Question: Can you provide insights on trends in GVHD and any subgroup details? - Management mentioned that it is still early to provide detailed information on GVHD subgroups, but they are collecting real-world evidence [67][70] Question: What portion of the $20 million revenue for RevuForge stems from refill dynamics? - Management stated that both new patient starts and refill dynamics are building, but specific numbers are not available yet [92][95]