Spark Therapeutics Inc. told BioWorld there was "not much more we can say beyond the press release" and the company wasn't offering executive interviews, but the buyout by Roche Holding AG generated vigorous buzz on Wall Street as pundits tried to guess which gene therapy firm might be next for a takeover in the space.
During 2017 and 2018, the FDA put life aplenty into the AML space by taking action to clear or reapprove no fewer than eight drugs for acute myeloid leukemia (AML), including Agios Pharmaceuticals Inc.'s Tibsovo (ivosidenib) to treat relapsed or refractory disease with an isocitrate dehydrogenase 1 (IDH1) mutation who are not eligible for standard therapy and Idhifa (enasidenib), cleared for patients with IDH2 mutations. And the indication only looks to grow hotter.
When a woman in Cork, Ireland, cutting off her own right index finger to relieve her pain made headlines recently, the mainstream media brought to popular knowledge the condition with which she had been diagnosed: chronic regional pain syndrome (CRPS), a devilish affliction being pursued by the likes of New York-based Axsome Therapeutics Inc. and the Grunenthal GmbH, of Aachen, Germany.
After excitement on Wall Street early in the day about pivotal phase III data with obeticholic acid (OCA) in patients with liver fibrosis due to nonalcoholic steatohepatitis (NASH), shares of New York-based Intercept Pharmaceuticals Inc. (NASDAQ:ICPT) settled to close at $117.57, up $6.75.
Cambridge, Mass.-based Mersana Therapeutics Inc. laid out in early January development plans that included the discontinuation of XMT-1522, an antibody-drug conjugate (ADC) taking aim at the popular HER2 target, partnered with Takeda Pharmaceutical Co. Ltd.
After having donated the benzimidazole therapy to the World Health Organization (WHO) since 2005 as a way of helping to treat about 2 million patients in more than 30 countries, Novartis AG won approval by the FDA of Egaten (triclabendazole) for fascioliasis, or liver-fluke disease, in patients 6 years of age and older.
Tim Dyer, CEO of Geneva-based Addex Therapeutics SA, told BioWorld that Indivior plc's decision to stop research with addiction candidate ADX-71441 and focus work on other GABAB positive allosteric modulator (PAM) therapies is "not the end of the world. The most important thing for us is that we've got a committed partner. Shifting from a lead asset to a backup – that's the business we're in," he said.
CEO Shawn Singh of Vistagen Therapeutics Inc. called the favorable vote for ketamine derivative esketamine by a joint meeting of FDA advisory panels "the writing of a bright new chapter in the history of neuropsychiatry," telling BioWorld that the "next transformative step" will involve developing more convenient, at-home options. Esketamine is given under medical supervision.
Trouble surfaced early in the joint FDA panel meeting to deliberate the drug-device combo esketamine 28-mg single-use nasal spray from Johnson & Johnson unit Janssen Pharmaceuticals Inc. when an agency official said U.S. gatekeepers didn't necessarily agree about the efficacy offered to support the ketamine-derived therapy for treatment-resistant depression (TRD).
