A phase 3 trial of a pan-PI3K/mTOR inhibitor, Celcuity, has met its primary endpoint, and the biotech is now free to submit a second filing of a molecule already under review by the FDA.
Previously, Celcuity had submitted an FDA application to approve the drug candidate, gedatolisib, in PIK3CA-wild-type breast cancer. The agency will decide on the application by July 17. Simultaneously, Celcuity was still studying the candidate in the PIK3CA-mutant cohort of the phase 3 study, dubbed Viktoria-1, which pitted a gedatolisib-based regimen against a doublet featuring Novartis PI3K inhibitor Piqray.
The progression-free survival (PFS) was significantly higher in individuals who received gedatolisib, Faslodex, developed by AstraZeneca and Pfizer, and Ibrance, than in those who were treated with Piqray and Faslodex and achieved the primary endpoint of the trial. Another secondary endpoint that was met in the study compared gedatolisib and Faslodex as a doublet to the combination of Piqray and the AstraZeneca drug.
The stock of Celcuity increased around 15 percent to $144 in after-market trading by the time of publication. Celcuity will file the data with the FDA as an optional new drug application. Provided all things go well, the FDA will approve gedatolisib in PIK3CA-wild-type breast cancer and will, in turn, fast-track the label to cover PIK3CA-mutant patients.
Celcuity plans to seek approval in Europe in the fourth quarter, CEO Brian Sullivan said on the earnings call in March. With the drug having been filed to be approved in Europe, Celcuity will increase efforts to find a partner for the drug in the region and in other parts of the world, Sullivan said.
It is expected to rank gedatolisib as one of its top 10 drug launches by 2026, in reflection of the forecast that sales would hit $2.1 billion by 2032. Sales in the same ballpark as that forecast would be a dramatic turnaround of gedatolisib, which Pfizer sold off in 2021. Pfizer terminated another PI3K/mTOR drug due to tolerability issues, and Eli Lilly dropped a program upon seeing clinical results.
How high the gedatolisib sales peak will depend on as-yet-undisclosed aspects of the phase 3 data. Celcuity will present detailed information in a late-breaking session at the 2026 ASCO Annual Meeting, which will be held in late May and early June. The magnitude of the PFS win and details of the safety and tolerability profile of gedatolisib will be areas of focus.
The PIK3CA-wild-type data indicated that gedatolisib causes lower incidences of hyperglycemia than Piqray, which Novartis linked (PDF) to grade 3 cases in one-third of the patients during development. However, “high rates” of stomatitis are a safety concern for gedatolisib, TD Cowen analysts said in a note to investors in December. The mutant selective PI3K-alpha inhibitor developed by Relay Therapeutics might be a competitor to gedatolisib.
Celcuity has reported strong Phase 3 results showing that Gedatolisib outperforms Novartis’s Piqray in treating certain advanced cancers. The findings position Gedatolisib as a next-generation targeted therapy with the potential to reshape treatment standards in oncology.
The Phase 3 study was structured as a randomized, controlled trial enrolling patients with advanced HR-positive, HER2-negative breast cancer who had previously received endocrine therapy. Participants were divided into treatment arms receiving the investigational therapy in combination with standard endocrine therapy versus the comparator regimen.
The trial incorporated stratification factors such as prior treatment history, genetic mutation status (including PI3K pathway alterations), and geographic region. This design ensured that outcomes reflected a broad and diverse patient population, increasing the reliability and applicability of the findings.
Biomarker and Precision Medicine Insights
An important component of the study was the evaluation of biomarkers to identify which patients derived the greatest benefit. Researchers observed that patients with specific PI3K pathway mutations responded particularly well, reinforcing the growing importance of precision medicine in oncology.
These findings suggest that future treatment strategies may rely more heavily on genetic profiling to guide therapy selection. Tailoring treatment based on tumor biology not only improves outcomes but also reduces unnecessary exposure to less effective therapies.
Clinical Significance of Gedatolisib
The success of Gedatolisib in Phase 3 trials marks a major milestone for Celcuity. By outperforming an established therapy like Piqray, Gedatolisib demonstrates its potential to become a new standard of care.
For oncologists, Gedatolisib offers a more effective and potentially safer alternative, particularly for patients who have developed resistance to existing treatments.
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