Core Insights - Revumenib, in combination with venetoclax and azacitidine, shows promising clinical activity in older patients with newly diagnosed mNPM1 and KMT2Ar AML, achieving a 67% complete remission (CR) rate and an 88% overall response rate (ORR) [1][2][6] - The ongoing Phase 3 EVOLVE-2 trial is evaluating the efficacy of this combination in newly diagnosed mNPM1 AML patients who are unfit for intensive chemotherapy [1][2] Group 1: Clinical Trial Results - The BEAT AML trial reported a 67% CR rate (29 out of 43 patients) and an 88% ORR (38 out of 43 patients) among newly diagnosed older adults with mNPM1 or KMT2Ar AML [1][6] - Among 37 patients assessed for measurable residual disease (MRD), 100% were found to be MRD negative [6] - The median duration of composite complete remission (CRc) was 12.0 months [6] Group 2: Patient Demographics and Safety - As of September 2024, 43 patients were enrolled, with 79% having mNPM1 AML and 21% KMT2Ar AML; the median age was 70 years [4][5] - Revumenib was generally well tolerated, with common non-hematologic treatment-emergent adverse events including nausea (60%), constipation (53%), and QTc prolongation (44%) [5][6] Group 3: Comparative Outcomes - The observed median overall survival (OS) was 15.5 months, with a CRc rate of 77% in mNPM1 patients and 89% in KMT2Ar patients [7][8] - Historical data indicated lower CRc rates and median OS for patients treated with venetoclax and azacitidine alone, highlighting the potential of revumenib in improving outcomes [8] Group 4: Future Developments - Revumenib is being developed for the treatment of relapsed or refractory AML with mNPM1 mutations, with a supplemental NDA submitted in April 2025 [10] - The company is conducting multiple trials of revumenib in combination with standard-of-care agents across various treatment settings [10][25]

Core Insights - Syndax Pharmaceuticals announced new data from the pivotal AUGMENT-101 trial of Revuforj (revumenib), a first-in-class menin inhibitor, for patients with relapsed or refractory (R/R) mutant NPM1 (mNPM1) and NUP98-rearranged (NUP98r) acute myeloid leukemia (AML) [1][2][16] Efficacy Results - In the efficacy-evaluable pivotal R/R mNPM1 AML population, the complete remission plus complete remission with partial hematologic recovery (CR+CRh) rate was 26% (20/77) and the overall response rate (ORR) was 48% (37/77) [1][5][4] - Robust responses were observed across various subgroups, regardless of co-mutations, number of prior lines of therapy, or prior venetoclax exposure [7][2] - Among patients with R/R NUP98r AML, 60% (3/5) achieved morphological remission [10] Survival Data - The median overall survival (OS) for all efficacy-evaluable Phase 2 R/R mNPM1 AML patients was 4.8 months, while responders had a median OS of 23.3 months [6][6] Safety Profile - Revumenib was generally well-tolerated, with only 4.8% (4/84) of patients discontinuing treatment due to treatment-related adverse events [8] - The safety profile was consistent with previously reported data, with differentiation syndrome occurring in 29% of patients [19][24] Future Developments - The company submitted a supplemental NDA for Revuforj for R/R mNPM1 AML, building on the FDA approval for R/R acute leukemia with a KMT2A translocation [2][16] - Ongoing and planned trials include combinations of revumenib with standard-of-care agents in various AML populations [11][16]

Summary of Syndax's Conference Call Company Overview - **Company**: Syndax - **Industry**: Healthcare, specifically oncology and hematology - **Products**: Two approved drugs - RevuForge (for acute AML and ALL) and Niktimbo (for chronic GVHD) [4][5][99] Key Points and Arguments Product Launch and Market Position - Syndax has two approved products addressing large markets, with a strong capital position to execute growth strategies [4][5] - RevuForge launched late last year, targeting acute leukemia with a focus on KMT2A translocations, which affects about 10% of AML and ALL patients [5][8] - Niktimbo is aimed at chronic GVHD, with a significant patient population of approximately 6,500 in the line plus and 15,000 in the US frontline and later [101] Clinical Data and Efficacy - RevuForge has shown a response rate of two-thirds in clinical trials, with many patients able to undergo stem cell transplants, which is a critical treatment goal [9][11] - The drug is positioned as a best-in-class menin inhibitor, with a unique profile that allows for treatment across various patient demographics, including pediatric patients [9][10] - Niktimbo has demonstrated early and durable responses in patients, with a high percentage remaining on the drug for extended periods [100][101] Sales and Market Dynamics - Early sales data indicated $7.7 million in Q4 and $20 million in Q1, with a strong execution team driving growth [13][19] - Formulary coverage for RevuForge is over 90% within six to seven months of launch, indicating strong payer acceptance [16] - The company expects to achieve over 50% market penetration for KMT2A by the end of the year, with ongoing patient inflow [27] Financial Outlook - Syndax is confident in achieving profitability within the next few years, driven by the high margins of Niktimbo and the ramp-up of RevuForge sales [109][110] - The company does not anticipate significant increases in operating expenses, allowing for a clearer path to cash flow positivity [110] Future Development and Market Expansion - Syndax is focused on expanding into earlier lines of therapy, particularly for unfit populations, with ongoing clinical trials [76][90] - The total addressable market (TAM) for both KMT2A and NPM1 is estimated at $2 billion annually, with potential for growth as treatment duration and patient penetration increase [70][71] Competitive Landscape - Syndax's drugs are positioned as the most efficacious in their respective categories, with a significant advantage over competitors in terms of overall response rates and tolerability [42][46] - The company is actively engaging with healthcare providers to build familiarity and confidence in using their products [60][66] Additional Important Insights - The company is committed to generating real-world data to support the use of their drugs in maintenance settings, which is crucial for physician adoption [36][39] - The management team emphasizes the importance of educating healthcare providers and building relationships to facilitate drug utilization [60][66] - Syndax is exploring additional assets for future growth but is currently focused on maximizing the potential of its existing products [112][113]

Syndax Pharmaceuticals (SNDX) 2025 Conference June 05, 2025 01:25 PM ET Speaker0 Good afternoon, everyone. Thank you for attending Jefferies Global Healthcare Conference. My name is Baozhi, a senior biotech analyst biotech team here. And please join me and welcome CEO, Mr. Michael Metzger CMO, Mr. Nick Batwood and COO, Mr. Steve Closter from Syndax Pharmaceuticals for this fireside chat session. Welcome. Speaker1 Thank you, Kelly. Speaker0 Maybe let's start with a high level question. Last year 2024 was a v ...

Group 1 - Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company focused on innovative cancer therapies [2] - The company's pipeline includes FDA-approved therapies such as Revuforj (revumenib), a menin inhibitor, and Niktimvo™ (axatilimab-csfr), a monoclonal antibody targeting the CSF-1 receptor [2] - Syndax is committed to advancing cancer care and is conducting several clinical trials across various treatment stages [2] Group 2 - Michael A. Metzger, CEO of Syndax, and the management team will participate in investor conferences, including the Jefferies Global Healthcare Conference on June 5, 2025, and the Goldman Sachs 46th Annual Global Healthcare Conference on June 11, 2025 [1][3] - A live webcast of the fireside chats will be available on the company's website, with replays accessible for a limited time [1]

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]

Syndax Pharmaceuticals (SNDX) closed the last trading session at $11.01, gaining 4.7% over the past four weeks, but there could be plenty of upside left in the stock if short-term price targets set by Wall Street analysts are any guide. The mean price target of $35.23 indicates a 220% upside potential.The mean estimate comprises 13 short-term price targets with a standard deviation of $10.34. While the lowest estimate of $17 indicates a 54.4% increase from the current price level, the most optimistic analys ...