Epizyme won accelerated FDA approval Thursday for a drug developed to treat the rare cancer epithelioid sarcoma (ES). The regulatory decision for the Epizyme (NASDAQ: EPZM) drug, tazemetostat (Tazverik), covers patients 16 or older whose ES has advanced or spread and is not eligible for surgical treatment. The drug is now the first FDA-approved treatment […]

[Updated 7:25 p.m. See below.] Safety concerns stymied Sarepta Therapeutics’ attempt to win FDA approval last August for golodirsen (Vyondys 53), its second Duchenne muscular dystrophy drug. But an appeal of the decision led to a surprise reversal by the regulator last month, with little explanation why. Details are now surfacing about the safety risks […]

Halozyme suffered a setback in November when its investigational pancreatic drug failed a Phase 3 clinical trial, but its CEO says the biotech’s drug delivery technology will make the company profitable as early as the second quarter of this year. The San Diego-based company’s technology, called Enhanze, allows some drugs traditionally given intravenously over hours […]

Cancer research has revealed proteins known to play a role in the disease, but with no known way of stopping what they do. In pharmaceutical parlance, they’re “undruggable.” Revolution Medicines is trying to drug the undruggable with small molecules that block the signaling pathways these proteins use to help cancer grow and thrive. With one […]

BioCryst Pharmaceuticals (NASDAQ: BCRX) has promoted Charles Gayer to chief commercial officer. Gayer joined the Durham, NC-based drug developer in 2015 as vice president of global strategic marketing. His experience also includes positions at Talecris and GlaxoSmithKline (NYSE: GSK). BioCryst is preparing for the expected commercial launch of berotralstat, a drug that has been submitted […]

Disarm Therapeutics has appointed Scott Holmes to serve as its chief financial officer, the same position he held most recently at Kiadis Pharma. Before working at Kiadis, Holmes was the CFO of Keryx Biopharmaceuticals. Cambridge, MA-based Disarm is developing drugs neurodegenerative disease drugs that target a protein called SARM1.

Daré Bioscience launched in 2015 with the aim of assembling a portfolio of novel women’s health products intended to pique interest from major pharmaceutical companies. Now the San Diego-based company has inked its first deal with Big Pharma, signing an agreement with German biopharma Bayer for its lead asset, a monthly non-hormonal form of birth […]

Neurocrine is doubling down on epilepsy, striking its second agreement in as many months with a new deal that grants it the option to license an investigational treatment for a rare form of the disease. The San Diego-based drug developer last month struck a deal with Xenon Pharmaceuticals (NASDAQ: XENE) that added an experimental treatment […]

RNA medicines can treat some diseases by hitting targets otherwise considered “undruggable” by small molecules and biologics, but protecting the nucleic acids from degradation and getting the therapies to where they are needed in the body can be difficult. San Diego startup DTx Pharma has raised $10.6 million to advance technology it has developed that […]

Food gurus tout the benefits of eating spinach and kale, but some people are better off leaving them on the plate. A molecule in these leafy greens contributes to hyperoxaluria, a condition characterized by kidney stones at first and serious organ damage later. Novome Biotechnologies is developing a gut microbiome treatment for hyperoxaluria. The company […]

Novo Holdings is repositioning the strategic focus of its REPAIR Fund in order to provide later-stage support to its portfolio companies, as the struggling antibiotics industry faces a dearth of investment. The Danish investor launched its REPAIR Impact Fund in 2018. An acronym for “Replenishing and Enabling the Pipeline for Anti-Infective Resistance,” the fund has […]

In the past seven years BioIntervene has been working to develop “ultra-selective” compounds to target an adenosine receptor associated with pain relief. Now the San Diego-based biotech has $30 million and a chief scientific officer, Charles Cohen, to guide its lead compound, BIO-205, into human testing for neuropathic pain. The company announced the Series A […]