Core Viewpoint - Celcuity Inc. announced that the FDA accepted its New Drug Application for gedatolisib in HR+/HER2- advanced breast cancer for review under the Real-Time Oncology Review program, facilitating an earlier evaluation process [1][2] Group 1: FDA Review and Application - The FDA's acceptance of the NDA for gedatolisib is part of the RTOR program, which allows for earlier submission of topline efficacy and safety results [1][3] - Celcuity plans to initiate a rolling submission of its NDA in September 2025, targeting completion by Q4 2025 [1][2] Group 2: Clinical Trial Results - The NDA submission is based on positive topline results from the PIK3CA wild-type cohort of the Phase 3 VIKTORIA-1 trial [2] - The gedatolisib-triplet (gedatolisib, fulvestrant, and palbociclib) reduced the risk of disease progression or death by 76% compared to fulvestrant, with a median progression-free survival (PFS) of 9.3 months versus 2.0 months for fulvestrant, an improvement of 7.3 months [2] - The gedatolisib-doublet (gedatolisib and fulvestrant) reduced the risk of disease progression or death by 67%, with a median PFS of 7.4 months compared to 2.0 months for fulvestrant, an improvement of 5.4 months [2] Group 3: Company Overview - Celcuity is a clinical-stage biotechnology company focused on developing targeted therapies for various solid tumors, with gedatolisib as its lead candidate [4] - Gedatolisib is a potent pan-PI3K and mTORC1/2 inhibitor, differentiating its mechanism of action from other therapies targeting PI3Kα, AKT, or mTORC1 [4] - The company is conducting multiple clinical trials, including the ongoing Phase 1/2 trial for metastatic castration-resistant prostate cancer and the Phase 3 VIKTORIA-2 trial for first-line treatment in HR+/HER2- ABC [4]

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 - 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]