Although Wall Street hammered shares of Solid Biosciences Inc. (NASDAQ:SLDB) down to $15.15, or 67.8 percent, to close them Thursday at $7.19, company officials told investors of a thesis based on preclinical work that could lead to better results with SGT-001 microdystrophin gene transfer for Duchenne muscular dystrophy (DMD)
Analysts during a conference call with Macrogenics Inc. characterized themselves as "dead wrong" and called the company's phase III victory "especially sweet" with margetuximab (marge) that was "written off by so many" in HER2-positive metastatic breast cancer. The news sent shares (NASDAQ:MGNX) up 130.4 percent, or $14.49, to close Wednesday at $25.60.
Luciano Rossetti, head of global R&D for Merck KGaA's biopharma branch EMD Serono, told BioWorld that "a very elegant molecule, fascinating from a protein-engineering point of view in addition to the biology" lies at the heart of the potential €3.7 billion (US$4.2 billion) deal with Glaxosmithkline plc (GSK).
Sunovion Pharmaceuticals Inc. stayed mostly mum on details regarding the FDA's complete response letter (CRL) related to the NDA for apomorphine sublingual film (APL-130277) to treat "off" episodes in Parkinson's disease (PD).
Details are yet to come about Evolus Inc.'s "user-centric" focus regarding Jeuveau (prabotulinumtoxinA-xvfs) for injection, as the product goes up against Botox (onabotulinumtoxinA, Allergan plc) as well as Dysport (abobotulinumtoxinA, Galderma Laboratories LP) in treating severe glabellar (frown) lines associated with corrugator and/or procerus muscle activity in adults. There's also Xeomin (botulinum toxin type A, Merz Pharma GmbH & Co. KGaA).
Epizyme Inc. recently charmed investors by disclosing its brisker path forward with EZH2 inhibitor tazemetostat in follicular lymphoma (FL), and drew more attention to the subtype of non-Hodgkin lymphoma (NHL).
Meiragtx Holdings plc CEO Alexandria Forbes told BioWorld that the "very strong financial position" conferred by her firm's deal with New Brunswick, N.J.-based Johnson & Johnson arm Janssen Pharmaceuticals Inc. means that more money can go to the Parkinson's disease (PD) program. "We haven't given guidance about how long our cash is going to last," she said, but the London-based firm now has "multiple years" worth of resources.
Sunovion Pharmaceuticals Inc. stayed mostly mum on details regarding the FDA's complete response letter (CRL) related to the NDA for apomorphine sublingual film (APL-130277) to treat "off" episodes in Parkinson's disease (PD).
If there's a small hitch in the phase II/III data rolled out by Orphazyme A/S with arimoclomol against the lysosomal storage disease Niemann-Pick type C (NPC), it's that the trial missed a co-primary endpoint chosen by the FDA, and the firm plans to take the results with the heat-shock protein (Hsp) amplifier to regulators in the U.S. and EU in the first half of next year.
Toronto-based Triphase Accelerator Corp. and its majority shareholder, Fight Against Cancer Innovation Trust (FACIT), disclosed a new epigenetics deal with Celgene Corp. for what's described as a first-in-class preclinical therapeutic targeting the WDR5 protein for the treatment of blood cancers, including leukemia.
