Gedatolisib

Evaluate forecast that gedatolisib sales will reach $2.1 billion by 2032.\n A phase 3 trial of Celcuity’s pan-PI3K/mTOR inhibitor has hit its primary endpoint, clearing the biotech to file a second submission for a molecule already under review by the FDA. Celcuity previously filed for FDA approval of the drug candidate, gedatolisib, in PIK3CA-wild-type breast cancer. The agency is set to rule on the application by July 17. In parallel, Celcuity continued to study the candidate in the PIK3CA-mutant cohort of the phase 3 study, dubbed Viktoria-1, pitting a gedatolisib-based regimen against a doublet featuring Novartis’ PI3K inhibitor Piqray.Progression-free survival (PFS) was significantly longer in people who received gedatolisib, AstraZeneca’s Faslodex and Pfizer’s Ibrance than in patients who took Piqray and Faslodex, achieving the trial’s primary endpoint. The study also hit a secondary endpoint that compared gedatolisib and Faslodex as a doublet to the combination of Piqray and the AstraZeneca drug.Celcuity shares rose about 15% to $144 a piece in premarket trading as of publication time.Celcuity plans to submit the data to the FDA as a supplemental new drug application. If everything goes to plan, the FDA will approve gedatolisib in PIK3CA-wild-type breast cancer and then quickly expand the label to cover PIK3CA-mutant patients.  Celcuity plans to seek approval in Europe in the fourth quarter, CEO Brian Sullivan said on an earnings call in March. After filing for approval in Europe, Celcuity will step up efforts to find a partner for the drug in the region and in other parts of the world, Sullivan said. Evaluate included gedatolisib in its top 10 drug launches for 2026, reflecting the forecast that sales will reach $2.1 billion by 2032. Sales in the same ballpark as that forecast would represent a dramatic turnaround for gedatolisib, which Pfizer let go for $10 million upfront in 2021. Pfizer terminated another PI3K/mTOR drug over tolerability, and Eli Lilly dropped a program after seeing clinical data. How high gedatolisib sales peak will depend on as-yet-undisclosed aspects of the phase 3 data. Celcuity plans to share detailed data in a late-breaking session at the 2026 ASCO Annual Meeting, which will take place 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 gedatolisib presentation was posted by ASCO Friday, after the organization released initial late-breaking titles in April, Leerink Partners analysts noted Monday. The late inclusion, possibly after ASCO’s original abstract submission deadline, bodes well for the magnitude of gedatolisib’s benefit, the Leerink team said.The PIK3CA-wild-type data suggested gedatolisib causes lower rates of hyperglycemia than Piqray, which Novartis linked (PDF) to grade 3 cases in one-third of 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. Relay Therapeutics’ mutant‑selective PI3K-alpha inhibitor could compete with gedatolisib.

Celcuity’s breast cancer drug wins again: The company said two regimens with its experimental drug gedatolisib succeeded in PIK3CA mutant patients as part of its Phase 3 trial. Last year, Celcuity reported that the drug staved off cancer progression in patients without PIK3CA mutations, and the company is awaiting a July FDA approval in that setting. The company is studying its drug as a second-line treatment for patients with HR-positive, HER2-negative breast cancer. The Minneapolis-based company said Friday that gedatolisib plus two other drugs —fulvestrant and Pfizer’s Ibrance — beat a combination of Novartis’ Piqray and fulvestrant, meeting the study’s primary endpoint. Another secondary analysis also showed that gedatolisib plus fulvestrant — without Ibrance — also beat Piqray plus fulvestrant. Celcuity is now headed to the FDA to ask for approval of gedatolisib in PIK3CA mutant patients as well. Unlike Piqray or Roche’s Itovebi, which are only approved for patients with a PIK3CA mutation, gedatolisib is meant to hit multiple parts of the key cancer pathway. As a result, Celcuity is aiming for a broad approval encompassing both wildtype and mutant forms of the cancer. — Lei Lei Wu 🚀 Odyssey charts $205M IPO: The biotech plans to raise that amount in net proceeds from its proposed Nasdaq listing as $ODTX . The autoimmune and inflammatory disease startup previously attempted an IPO in 2025 but pulled back and went with a Series D instead. Now, it seeks to sell 13.24 million shares at $16 to $18 apiece after Kailera Therapeutics, Avalyn Pharma, Seaport Therapeutics and Hemab Therapeutics opened the IPO window heading into the summer. — Kyle LaHucik 💸 Gene therapy biotech gets $43M: Philadelphia startup Latus Bio has extended its 2024 Series A to a total of $97 million as it looks to begin two clinical trials this year. The company will test AAV-based gene therapies for Huntington’s disease and CLN2 disease. Its backers include 8VC, DCVC Bio and others. — Kyle LaHucik MacroGenics expands royalty deal: The biotech will get $60 million upfront and up to $20 million more based on a 2026 sales milestone for Zynyz. Sagard Healthcare Partners and MacroGenics formed the initial deal around the Incyte-commercialized PD-1 inhibitor last year. — Kyle LaHucik Mirum scores Phase 2b win for liver drug: The biotech said volixibat hit its primary endpoint in the trial, which enrolled patients with a rare liver condition called primary sclerosing cholangitis. Individuals with the chronic disease have inflammation in the bile ducts, leading to liver damage, according to the Mayo Clinic. Volixibat is designed to treat a common itching symptom (cholestatic pruritus) that comes with the disease. Mirum plans an FDA submission in the second half of 2026. — Max Gelman Indivior begins $175M share repurchase: The move comes after the company said last week that it would punt on Phase 3 studies for a program called INDV-6001, and that a Phase 2 trial for INDV-2000 in opioid use disorder had failed. — Max Gelman