Incyte Corp.’s Zynyz (retifanlimab-dlwr) has received another U.S. FDA approval and put some distance between it and a 2021 complete response letter (CRL). The FDA gave the humanized monoclonal antibody targeting PD-1 the nod for first-line treatment of adults with inoperable locally recurrent or metastatic squamous cell carcinoma of the anal canal (SCAC). That makes it the only approved first-line treatment for this cancer. Zynyz already was approved in March 2023 for adults with the rare skin cancer advanced Merkel cell carcinoma. In July 2021, the FDA issued a CRL for the NDA tied to treating adults with locally advanced or metastatic SCAC. The new FDA approval also allows Zynyz to be used for adults with locally recurrent or with metastatic SCAC with disease progression on or intolerance to platinum-based chemotherapy.

Drug developer stocks fall as companies navigate clinical roadblocks

The BioWorld Drug Developers Index underperformed both the Nasdaq Biotechnology Index and the Dow Jones Industrial Average throughout February and March. However, it showed some recovery by the end of April, finishing the month down 4.67%. The largest decliner so far this year is Ironwood Pharmaceuticals Inc. (down 79%), which faced a setback regarding apraglutide’s development for short bowel syndrome, with the U.S. FDA now requiring a confirmatory phase III trial before considering approval.

Female C-suite representation stalling, says BIA report

Even before the Trump administration’s executive order wiping out the concept of diversity, equity and inclusion, the statistics on female representation in the biotech industry painted a story of stagnating progress. But for Jane Wall, managing director of the U.K. Bioindustry Association (BIA), the available statistics also begged a question: Where was the U.K. specific data that could inform attempts to change things? There was “much frustration around stats being recycled without context or background – and very little our community could relate to,” Wall said. “We were challenged by our women in biotech advisory group to take a look under the bonnet and find out what was really happening,” she told a meeting in Edinburgh, May 14, held to discuss the findings.

Therini Bio locks in another $39M for its fibrin-targeting immunotherapies

Therini Bio raised an additional $39 million in its series A financing round, bringing the total for the round to $75 million. The Sacramento, Calif.-based company is developing THN-391, an antibody targeting fibrin, for the treatment of neurodegenerative diseases. The company plans to use the capital to run two concurrent phase Ib studies in patients with Alzheimer’s disease and diabetic macular edema (DME). The capital will also be used to support the development of a bispecific antibody targeting fibrin and VEGF that Therini plans to move into DME if the fibrin-specific antibody is successful in the phase Ib study.

Biomarin snags potential ‘first-in-disease’ ERT in $270M Inozyme buy

On the heels of a positive interim readout for enzyme replacement therapy (ERT) candidate INZ-701 in the rare disease ENPP1 deficiency, Inozyme Pharma Inc. closed out the week with an acquisition deal from Biomarin Pharmaceuticals Inc. valued at about $270 million. The all-cash transaction is priced at $4 per share, a 181% increase over Inozyme’s (NASDAQ:INZY) May 15 closing price and a 267% increase over the closing price May 13, prior to the release of interim data from the phase III Energy 3 trial. Biomarin, which expects full top-line data from Energy in the first part of 2026 and potential regulatory approval in 2027, said INZ-701 fits well with the firm’s existing ERT business and could become Biomarin’s sixth “first-in-disease” treatment.

ASGCT 2025: Overcoming kidney complexity in gene and cell therapy

Gene and cell therapies can target the kidney to treat congenital, acute or chronic diseases affecting this organ. However, its complex structure poses a challenge for these technologies. To be precise and effective in the long term, new approaches should circumvent the specificities of renal tissue, with novel methods of delivery and gene transfer to offer new therapeutic options for patients who lack them. “The kidney could be described as one of the world’s most sophisticated filters,” said Alice Brown at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in New Orleans during the session, “Harnessing cell and gene therapies: new frontiers in kidney treatment.” Brown is the chief scientific officer of Purespring Therapeutics Ltd., a U.K.-based gene therapy company specializing in kidney diseases and co-founded by Moin Saleem, who is a professor of pediatric renal medicine at the University of Bristol.

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ADC, Amneal, Arcellx, Artiva, Atara, Atossa, Aura, Biocryst, Biohaven, Bioinvent, Brainchild, BRC, Cabaletta, Cellarity, Cure Rare Disease, Enliven, GT, Immutep, Imunon, Inozyme, Ips, Janux, Kaerus, Mendus, Merck, Mesoblast, Nicox, Pathos, Plus, , Poltreg, Quoin, Quralis, Rybodyn, Sanofi, Sernova, Silo, Spur, Takeda, Telomir, Therini, Tiziana, Trilink, Vaxart, Xspray