Cidara Therapeutics Inc. won U.S. FDA approval March 22 of once-weekly Rezzayo (rezafungin for injection) for candidemia and invasive candidiasis in adults with limited or no alternatives. The go-ahead landed days after the CDC warned of an emerging Candida strain, C. auris, that has begun to threaten more people. Rezzayo is the first new option to reach the market for the two indications in more than a decade and is expected to launch in July. Shares of the firm (NASDAQ:CDTX), which had been rising ahead of the March 22 PDUFA date, were trading midday at $1.63, down 27 cents, or 14%.
US FDA approves Incyte’s PD-1 inhibitor Zynyz for Merkel cell carcinoma
Incyte Corp.’s retifanlimab-dlwr received its first regulatory nod on March 22, with the U.S. FDA granting accelerated approval for the PD-1 inhibitor to treat adults with the rare form of skin cancer, advanced Merkel cell carcinoma (MCC). Branded Zynyz, it was approved based on data from Podium-201, an open-label, multiregional, single-arm study of 65 patients with advanced disease who had not received prior systemic therapy.
Almost, but not quite: Adcom is mixed on Biogen’s ALS drug
Tofersen’s development is progressing in fits and starts. That was evident at the U.S. FDA’s March 22 meeting of the Peripheral and Central Nervous System Drugs Advisory Committee, which unanimously agreed Biogen Inc.’s failed phase III study did predict a clinical benefit in treating amyotrophic lateral sclerosis that includes the rare SOD1 component. However, the adcom was not convinced the study data was strong enough for the agency to grant it full approval, suggesting accelerated approval may be the best route for tofersen to take. The FDA has set an April 25 PDUFA date. The adcom members were torn by the lack of data, citing the need for an effective treatment for a disease that is usually fatal yet only affects about 330 people in the U.S.
Biohaven acquires early stage TYK2/JAK1 inhibitor from Highlightll in $970M deal
Biohaven Ltd. has acquired global rights, excluding China, from Highlightll Pharmaceutical Co. Ltd. to develop and commercialize a dual inhibitor of tyrosine kinase 2 (TYK2) and JAK1, BHV-8000 (TLL-041), for the treatment of brain disorders in a deal worth up to $970 million. Highlightll will receive $10 million in up-front cash and $10 million in Biohaven equity. The Chinese company is also eligible to obtain up to $950 million in development and commercial milestone payments, as well as mid-single-digit to lower teens percentages of tiered royalties.
Aurion victorious with Vyznova cell therapy approval in Japan
Japan’s PMDA has approved Aurion Biotech Inc.’s cell therapy, Vyznova, for the treatment of bullous keratopathy of the cornea, making it the first-ever approval of a cell therapy to treat corneal endothelial disease. The company’s technology was developed by ophthalmic surgeon and research scientist Shigeru Kinoshita and his colleagues at Kyoto Prefecture University of Medicine in Japan. The method involves taking healthy corneal cells from donor corneas and culturing them to produce off-the-shelf, fully differentiated corneal endothelial cells. An intracameral injection deposits the cells into the eye, which repopulate the cornea and start removing fluid from it, thereby reducing the edema.
China greenlights CSPC’s mRNA COVID-19 vaccine for emergency use
CSPC Pharmaceutical Group Ltd.’s COVID-19 mRNA vaccine, SYS-6006, has been included for emergency use by the NMPA, the company said on March 22, marking the first domestically developed COVID-19 mRNA vaccine approved for use in China. Independently developed by CSPC, SYS-6006 covers the omicron subvariant BA.5’s core mutation at the spike mutation positions. It entered emergency clinical trial approval in April 2022 and has finished phase I, II and heterologous booster vaccination clinical studies in China with more than 5,500 participants.
Mbiomics raises $14M to pivot to microbiome-based therapeutic development
Mbiomics GmbH raised €13 million (US$14.2 million) in a first close of a series A round that will enable it to pivot from being a microbiome analytics firm to a therapeutics developer. The firm is bringing a new high-throughput, low-cost technique, based on fluorescent microscopy, to bear on the complex questions of analyzing and quantifying the gut microbiome, in an effort to add more precision to an area that has been sorely lacking in it to date.