One business day earlier than expected, rare disease specialist Amicus Therapeutics Inc. secured a long-sought FDA approval for its oral Fabry disease therapy, Galafold (migalastat), on Friday, the company's first U.S.-approved medicine. The initial U.S. list price will be $315,000 per year.
For the first time in more than 18 years, the FDA has approved a new drug for malaria prevention. Washington-based 60 Degrees Pharmaceuticals LLC (60P), which got a green light to market Arakoda (tafenoquine) tablets for adults, said the approval marks the culmination of more than 30 years of R&D with the U.S. Army Medical Research and Materiel Command, starting with the discovery of tafenoquine at the Walter Reed Army Institute of Research.
More than two decades after founding Berlin Heart, the company's former CEO, Johannes Müller – a doctor, researcher and inventor behind that company's pioneering ventricular assist device – is leading a new company, Berlin Cures Holding AG, to attack a root cause of a common type of heart failure with a new DNA aptamer-based compound, BC-007. It enters phase II testing this summer. The trial is expected to read out by the middle of 2020.
Spark Therapeutics Inc. said its experimental hemophilia A therapy, SPK-8011, reduced the annualized need for infusions across all 12 participants in its phase I/II study by 97 percent. But negative immune responses in two of 12, one of which sent a patient to the hospital, sent company shares (NASDAQ:ONCE) down about 28 percent on Tuesday. Plans for phase III trials of the treatment are set to begin in the fourth quarter. Meanwhile, shares of competitor Biomarin Pharmaceutical Inc. (NASDAQ:BMRN), which is developing its own gene therapy for the disorder, climbed, rising about nearly 8 percent.
Samumed LLC, a small company developing drugs to harness the Wnt pathway to treat multiple conditions, has closed a $438 million equity round at what it said was a pre-money valuation of $12 billion, marking one of the biggest disclosed private company financings of the year to date.
California's new Consumer Privacy Act, the toughest data privacy law in the nation, will impose stringent new requirements on the state's high-profile tech companies in 2020. Its impacts on the life sciences industry, though, remain unclear.
Shares of Epizyme Inc. (NASDAQ:EPZM) fell 23.9 percent to $9.90 on Thursday after the company said it would delay submitting a new drug application for its lead candidate, tazemetostat, in epithelioid sarcoma (ES) until mid-2019 and quit testing the drug as a monotherapy in diffuse large B-cell lymphoma (DLBCL) after an interim assessment suggested the phase II study wasn't worth continuing.
Laekna Inc., a Shanghai-based company that recently completed an $18.5 million series A financing led by Orbimed Asia, has licensed rights to develop and commercialize two cancer-focused oral Akt kinase inhibitors from Novartis AG. The deal gives the Swiss drugmaker an equity stake in the Chinese company as well as undisclosed up-front and development milestone payments, plus potential royalties on future sales. Laekna, which has indicated plans to seek regulatory approvals in China and beyond, said that it has "mapped out several clear registration paths for NDA approval" for the drugs.
Two pivotal trials testing the ability of a topical suspension of silver particles to improve the efficacy of laser therapy for acne have both failed to surface additional benefit from the candidate, SNA-001, sending shares of the regimen's developer, Sienna Biopharmaceuticals Inc. (NASDAQ:SNNA) down as far as $12.77 before ending the day at $15 Monday, down 70 cents. The probability of success in a soon-to-finish third pivotal trial with a different laser now seems lower, Sienna's president and CEO, Frederick Beddingfield, said.
The lead program at Tonix Pharmaceuticals Holding Corp., a medicine aimed at delivering bedtime relief from symptoms of military-related post-traumatic stress disorder (PTSD), failed to adequately separate from placebo in an interim analysis of 12-week phase III data. News of the setback, the second pivotal miss for the drug, sent company shares (NASDAQ:TNXP) down 65.8 percent to $1.35 on Friday, as Tonix pursued hopes that the FDA might judge a future study by a data cut more favorable to the drug.
