Celcuity Expands Gedatolisib Trial to 90,000 Patients in First-Line Breast Cancer

Celcuity Inc. is expanding its Phase 3 trial for gedatolisib to include nearly all first-line patients with HR+/HER2- advanced breast cancer, a move that positions its multi-target inhibitor against established therapies in a market of approximately 90,000 new patients annually.

"Developing the gedatolisib triplet for nearly all patients in the first-line setting, irrespective of their endocrine sensitivity or PIK3CA status, offers the potential to advance the standard of care," said Brian Sullivan, CEO and co-founder of Celcuity.

The expansion into endocrine-sensitive patients is backed by Phase 1b data where a gedatolisib combination showed a median progression-free survival of 48.6 months and an objective response rate of 79 percent, results the company notes compare favorably to current standard-of-care regimens.

The amended VIKTORIA-2 trial now pits gedatolisib directly against Novartis AG’s ribociclib, a standard therapy, in two separate studies for endocrine-resistant and endocrine-sensitive patients. Success could disrupt the treatment paradigm for a disease where resistance to single-target inhibitors remains a primary challenge.

Pivoting to a Broader First-Line Setting

The VIKTORIA-2 trial will now consist of two independent studies. The first will evaluate gedatolisib with palbociclib and fulvestrant in approximately 440 endocrine-resistant patients. The second, larger study will enroll about 740 endocrine-sensitive patients to receive gedatolisib with palbociclib and letrozole. This strategic shift, finalized after a Type B meeting with the U.S. Food and Drug Administration, significantly widens the drug's potential first-line indication.

Gedatolisib is a pan-PI3K/mTOR inhibitor, a mechanism that differentiates it from currently approved single-target inhibitors of the PI3K/AKT/mTOR (PAM) pathway, a key driver of HR+/HER2- breast cancer. By targeting all four class I PI3K isoforms as well as mTORC1 and mTORC2, the drug aims to prevent the adaptive resistance that often develops with single-node inhibitors. The trial’s control arms will use ribociclib, marketed by Novartis as Kisqali, establishing a high bar for demonstrating clinical superiority. Pfizer Inc.'s palbociclib (Ibrance) is also part of the combination therapy being tested.

Subcutaneous Formulation Signals Long-Term Ambitions

In a concurrent move to bolster its long-term strategy, Celcuity has filed its first patent for a subcutaneous formulation of gedatolisib. This would allow for a simple injection instead of an intravenous infusion, a critical factor for patient convenience and adherence in therapies that may last for several years.

"As we initiate pivotal studies for indications that may offer several years of progression-free survival benefit, development of a subcutaneous formulation of gedatolisib would particularly benefit patients," Sullivan said. The company is working to demonstrate clinical equivalence to the current intravenous version. This life-cycle extension strategy comes as Celcuity awaits an FDA decision on gedatolisib for a different subset of breast cancer patients, with a PDUFA goal date set for July 17, 2026, for its application in second-line PIK3CA wild-type tumors.

This article is for informational purposes only and does not constitute investment advice.