Group 1 - Shares of Syndax Pharmaceuticals (SNDX) have increased by 2.2% over the past four weeks, closing at $9.57, with a mean price target of $32.33 indicating a potential upside of 237.8% [1] - The mean estimate consists of 12 short-term price targets with a standard deviation of $10.46, where the lowest estimate of $17.00 suggests a 77.6% increase, and the highest estimate of $45.00 indicates a potential surge of 370.2% [2] - Analysts show strong agreement in revising earnings estimates higher, which correlates with potential stock price movements, as the Zacks Consensus Estimate for the current year has increased by 0.5% over the past month [11][12] Group 2 - SNDX holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimates, suggesting a strong potential upside [13] - While consensus price targets may not be reliable indicators of actual stock gains, they can provide a directional guide for price movement [14]

Company Overview - Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company focused on advancing innovative cancer therapies [3] - 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 [3] Upcoming Financial Results - Syndax Pharmaceuticals will report its second quarter 2025 financial results and provide a business update on August 4, 2025 [1] - A conference call and live audio webcast will be hosted by the management at 4:30 p.m. ET on the same day [1] Accessing the Conference Call - The live audio webcast and accompanying slides can be accessed through the Events & Presentations page on the company's website [2] - Domestic and international dial-in numbers are provided for the conference call, with a specific Conference ID: Syndax2Q25 [2] - A replay of the conference call will be available approximately 24 hours after the event and will remain accessible for 90 days [2]

Group 1 - The article emphasizes that investing is a learning process, where failures serve as tuition and successes contribute to lessons learned [1] - The author has approximately five years of focused research on various stocks, with a primary emphasis on healthcare stocks in recent years [1] Group 2 - The author has a beneficial long position in the shares of Syndax, indicating a personal investment interest [2] - The article expresses the author's own opinions and is not influenced by any business relationships with companies mentioned [2]

Revuforj (revumenib) - Revuforj generated $20 million in net revenue in 1Q25[3] - Syndax initiated a pivotal frontline trial of revumenib with ven/aza in mNPM1 and KMT2Ar patients unfit for intensive chemo in 1Q25[3] - Syndax completed the submission of Revuforj sNDA for R/R mNPM1 AML in April 2025[3] - In the AUGMENT-101 trial, Revuforj showed a CR+CRh rate of 212% (22/104) with a median duration of 64 months[29] - In the Phase 2 portion of AUGMENT-101, revumenib demonstrated a CR/CRh of 23% (15/64) in R/R mNPM1 AML patients[47] Niktimvo (axatilimab-csfr) - Niktimvo generated $136 million in net revenue in 1Q25 (reported by Incyte)[3] - In the AGAVE-201 trial, Niktimvo showed an overall response rate of 75% (59/79) through 6 months[78] - 60% of responders maintained a response for at least 12 months[78] Financial Position - Syndax had $6021 million in cash and cash equivalents as of March 31, 2025, expected to fund the company to profitability[3] - Syndax's total revenue for 1Q25 was $20 million[102]

Core Insights - The FDA has granted Priority Review for Syndax Pharmaceuticals' supplemental New Drug Application (sNDA) for Revuforj® (revumenib) targeting relapsed or refractory (R/R) mutant NPM1 (mNPM1) acute myeloid leukemia (AML) with a PDUFA action date set for October 25, 2025 [1][5] - Revuforj is positioned to be the first and only menin inhibitor approved for both R/R mNPM1 AML and R/R KMT2Ar acute leukemia, expanding its indication from its initial approval in 2024 [2][5] - The sNDA is supported by positive pivotal data from the AUGMENT-101 trial, with results published in the journal Blood and presented at the European Hematology Association (EHA) Annual Congress Meeting in June 2025 [2][5] Company Overview - Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company focused on innovative cancer therapies, with Revuforj being a first-in-class oral menin inhibitor [4][22] - The company aims to lead in the therapeutic class of menin inhibitors, supported by compelling data and established relationships with clinicians and payers [2][22] Industry Context - Mutant NPM1 (mNPM1) AML is characterized by mutations in the NPM1 gene, occurring in approximately 30% of adult AML cases, and is associated with high relapse rates and poor prognosis [3] - There are currently no approved therapies that selectively target the underlying mechanisms of mNPM1 AML, highlighting a critical unmet need in the treatment landscape [3]

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]

Financial Data and Key Metrics Changes - The company achieved $20 million in sales for the first quarter, surpassing expectations [8][9] - The sales for Niktinvo marked $13.6 million, indicating strong demand and a good start for the product [61] Business Line Data and Key Metrics Changes - RAV4's launch has seen a broad and growing user base, with approximately 44% of tier one and tier two institutions having written prescriptions by March, expected to exceed 50% [10][12] - Formulary coverage has improved significantly, rising from just above 70% in March to over 90% [12] Market Data and Key Metrics Changes - The company is positioned well in the U.S. market with robust supply chains and minimal impact from external factors such as tariffs [6][7] - The KMT2A population is the primary focus, with expectations to achieve over 50% market penetration in the first year [14] Company Strategy and Development Direction - The company aims to expand beyond relapsed-refractory markets and build long-lasting franchises with its first and best-in-class medicines [5] - There is a strong focus on moving into frontline combinations and addressing both fit and unfit patient populations through randomized control studies [51][52] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's positioning amidst evolving policies on drug pricing and tariffs, stating that the supply chains are robust [6][7] - The company anticipates significant growth opportunities in the coming year, particularly with the expected approval of the sNDA for the NPM1 patient population [70][71] Other Important Information - The company is actively enrolling patients in the EVOLVE study, which is a large multi-site international study with dual primary endpoints [55] - The company expects to present updated data at the EHA Congress, which will include Kaplan Meier's around overall survival [59] Q&A Session Summary Question: What are the dynamics of the RAV4 launch? - The launch has exceeded expectations with a broad user base and improved formulary coverage, leading to strong sales momentum [9][12] Question: How is the company managing safety concerns with Revuforge? - Physicians are well-equipped to manage side effects, and early experiences have been positive with no significant hindrances to patient treatment [24][29] Question: What is the timeline for the NPM1 supplementary NDA review? - The supplementary NDA was submitted in April, with a six-month priority review expected to conclude around October [32] Question: How does the company plan to position Revuforge against competitors? - The company believes it has a compelling profile and is the only approved menin inhibitor, with strong efficacy data supporting its use [41][42] Question: What milestones should investors look for in the next twelve months? - Key milestones include data presentations at EHA, expected approval of the sNDA, and continued growth in KMT2A and NPM1 franchises [70][71]

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]