Celcuity's Gedatolisib Gains Momentum with FDA's RTOR Program and Promising Phase 3 Trial Results

Celcuity's gedatolisib has gained momentum with FDA's RTOR program and promising Phase 3 trial results, showing a significant improvement in median progression-free survival for patients with advanced/metastatic ER+ breast cancer. The FDA's acceptance of the NDA under the RTOR program indicates an expedited review process. LifeSci Capital maintains a Buy rating with a $65.00 price target, while Stifel Nicolaus also maintains a Buy rating with a $68.00 price target.

Celcuity Inc. (Nasdaq: CELC), a clinical-stage biotechnology company, has made significant progress with its lead therapeutic candidate, gedatolisib. The U.S. Food and Drug Administration (FDA) has agreed to accept the company's New Drug Application (NDA) for gedatolisib in HR+/HER2- advanced breast cancer (ABC) for review under the Real-Time Oncology Review (RTOR) program. This decision signifies an expedited review process, potentially bringing the drug to market sooner than through standard channels.

The RTOR program is designed to facilitate a more efficient review process for drugs, ensuring that safe and effective treatments are available to patients as early as possible. To be considered for RTOR, submissions must demonstrate substantial improvement over available therapies, easily interpreted clinical trial endpoints, and no aspects likely to require a longer review time. Celcuity's NDA submission, based on the Phase 3 VIKTORIA-1 trial, meets these criteria [1].

The VIKTORIA-1 trial results for the PIK3CA wild-type cohort showed unprecedented efficacy. The gedatolisib-triplet therapy (gedatolisib, fulvestrant, and palbociclib) reduced the risk of disease progression or death by 76% compared to fulvestrant alone, with a median progression-free survival (PFS) of 9.3 months versus 2.0 months. The gedatolisib-doublet (gedatolisib and fulvestrant) demonstrated a 67% risk reduction with a median PFS of 7.4 months versus 2.0 months. These hazard ratios and improvements in median PFS are remarkable, representing some of the most substantial improvements ever seen in HR+/HER2- advanced breast cancer trials [2].

The FDA's acceptance of Celcuity's NDA under the RTOR program is a significant regulatory milestone. The drug's earlier Breakthrough Therapy and Fast Track designations further underscore its potential significance. Gedatolisib addresses an unmet medical need for patients with HR+/HER2- advanced breast cancer who have progressed after CDK4/6 inhibitor treatment. With submission initiation in September and completion targeted for Q4 2025, Celcuity is positioning gedatolisib for potential approval in 2026, assuming standard FDA review timelines following a complete submission.

Investors have shown optimism, with LifeSci Capital maintaining a Buy rating with a $65.00 price target and Stifel Nicolaus also maintaining a Buy rating with a $68.00 price target [2].

References:
[1] https://www.morningstar.com/news/globe-newswire/9519366/celcuity-to-initiate-nda-submission-of-gedatolisib-in-pik3ca-wild-type-cohort-in-hrher2-advanced-breast-cancer-under-fdas-real-time-oncology-review-program
[2] https://www.stocktitan.net/news/CELC/celcuity-to-initiate-nda-submission-of-gedatolisib-in-pik3ca-wild-u9uvrrmp5z07.html