Talazoparib Tosylate

iStock, JHVEPhoto The FDA’s independent advisors found Pfizer’s data lacked the precision needed to establish the efficacy of Talzenna in patients with prostate cancer who do not carry HRR mutations. The FDA’s Oncologic Drugs Advisory Committee unanimously declined to endorse a label expansion for Pfizer ’s PARP inhibitor Talzenna, which the pharma is proposing as a combination regimen with its drug Xtandi for first-line treatment of patients with metastatic castration-resistant prostate cancer carrying a certain biomarker profile. Voting 8–0 against the proposal, the external experts said Wednesday that Pfizer had not provided enough evidence to establish the efficacy of the Talzenna combo in men with non-homologous recombination repair mutated (non-HRRm) prostate cancer. “Precision oncology demands precision trials,” Neil Vasan, assistant professor at the Columbia University Medical Center, said at the end of the meeting explaining his no vote. “The study was not powered to test the efficacy in the patients without HRR mutations,” which in turn “weakened the strength of the conclusions that we can draw.” This problem extends “across all oncology” as well, Vasan added. “As a field, we need to commit to formally evaluating whether targeted therapies benefit patients with and without the biomarker.” To back its latest application, which seeks a broader prostate cancer label for Talzenna, Pfizer drew data from the Phase III TALAPRO-2 trial. The company announced in October 2024 that Talzenna plus Xtandi significantly and meaningfully improved overall survival (OS) versus Xtandi alone—an effect that was observed not only in those with HRR mutation but in all participants regardless of biomarker status. At the time, Roger Dansey, former chief oncology officer at Pfizer, said that Talzenna is the “first and only PARP inhibitor” to demonstrate a significant survival benefit, when used with an androgen receptor blocker, in mCRPC patients, “regardless of mutation status.” But in a briefing document published ahead of the advisory committee meeting, the FDA’s internal reviewers challenged Dansey’s claims. “While the statistically significant OS improvement in the all-comers population provided the impetus for this application, interpretation of the OS result and its applicability to current U.S. standard of care are unclear,” they wrote. “Consideration of the all-comers OS result may be misleading when interpreting efficacy in the non-HRRm population.” Talzenna, an oral drug, was first approved by the FDA in October 2018 for HER2-negative, germline BRCA-mutated breast cancer. Nearly five years later, in June 2023, the agency handed Talzenna a label expansion , this time in combination with Xtandi, an oral androgen receptor blocker, for patients with metastatic castration-resistant prostate cancer (mCRPC) who have HRR mutations.

Today, a brief rundown of news involving Pfizer and Sarepta Therapeutics, as well as updates from UroGen Pharma, Altos Labs and BioNTech that you may have missed.A Food and Drug Administration advisory panel on Wednesday voted unanimously against expanding use of Pfizers Talzenna in prostate cancer. All eight committee members concluded Pfizers data werent strong enough to support Talzennas benefits in metastatic prostate cancer patients who dont have a specific set of genetic alterations called homologous recombination repair mutations. Talzenna, a so-called PARP inhibitor, was approved for use alongside Xtandi in that particular group of prostate cancer patients in 2023. Pfizer has been hoping survival data accrued since then might convince the FDA to clear treatment in all people newly diagnosed with metastatic disease. Ben FidlerAgency panelists also declined to endorse a drug Urogen Pharma has been developing for a form of bladder cancer. Urogenis seeking clearance of the therapy, a formulation of the chemotherapy mitomycin, in a recurrent type of non-muscle invasive bladder cancer. But by a slim, 5-4 margin, FDA advisers determined the drugs benefits didnt outweigh the risks of treatment. UroGen shares fell by more than 50% following the vote. The FDA will issue a verdict by June 13. Ben FidlerHealth authorities in the U.K. are allowing Sarepta Therapeutics to continue dosing in a trial called Envision, which is testing the companys gene therapy Elevidys in people with Duchenne muscular dystrophy who either cant walk or who still can, but are older. In March, Sarepta revealed a boy who received Elevidysdied from acute liver failure, which led to a pause in testing in Europe. Writing in a Wednesday note to clients,Brian Skorney, an analyst at Baird, described the decision by U.K. regulators as a favorable signal for how the Food and Drug Administration and the European Medicines Agency may view Elevidys. Ned PagliaruloBioNTech will invest up to 1 billion, or about $1.34 billion, in drug research in the U.K. over the next decade, the company said Tuesday. Alongside that commitment, BioNTech will receive as much as 129 million in grant funding from the U.K. government over 10 years, one of the largest grants the country has handed to a pharmaceutical company. That money will help BioNTech establish two new R&D centers, one of which will be based in Cambridge, England and focus on genomics, oncology and other research priorities. BioNTech will build a U.K. headquarters in London as well. Ben FidlerAltos Labs, a buzzy startup backed by billions of dollars in venture investment, has put some of its funds to use buying Dorian Therapeutics, a small privately held company based in San Carlos, California. In a LinkedIn post, Dorian CEO Maddalena Adorno wrote that Dorians cellular rejuvenation research will continue at Altos, which is studying the basis of aging and how that translates to disease. Dorians research started at the Stanford University School of Medicine years ago,Adorno wrote. Ned Pagliarulo '