How to assure screening for patients in whom just-approved Vitrakvi (larotrectinib) most likely will help is something "we've been working very hard for the last nine months" to establish, Joseph Germino, vice president of medical affairs for oncology with Bayer AG, told BioWorld. "The drug is so effective – it doesn't work in everybody [but] it works in most people – that it would be a shame if somebody were not able to get it because nobody checked" for eligibility.
Edge Therapeutics Inc.'s hunt for a "strategic alternative" after the failure of its flagship candidate, EG-1962 (nimodipine microparticles), ended with a stock-for-stock merger deal with privately held immuno-oncology (I-O) specialist Protein Delivery Sciences Biotechnology Corp. (PDS).
With a "cosmetically elegant" topical therapy, Pellepharm Inc. CEO Sanuj Ravindran told BioWorld that his firm is hoping to exploit the upside of hedgehog inhibition demonstrated so well by Erivedge (vismodegib, Roche Holding AG) while avoiding the downside of a systemic therapy with that mechanism of action in basal cell carcinoma (BCC).
Arena Pharmaceuticals Inc. CEO Amit Munshi said the $800 million up-front, worldwide licensing deal with United Therapeutics Corp. centered on prostacyclin (IP) receptor agonist ralinepag in pulmonary arterial hypertension (PAH) "allows us a broader push in the GI [gastrointestinal] space" with S1P receptor modulator etrasimod, which has reached the planning stage for phase III trials in ulcerative colitis (UC).
Founder and Chief Technology Officer Nello Mainolfi told BioWorld that Kymera Therapeutics Inc. will disclose more in the months ahead about its lead compound, which sits at "the juncture between oncology and inflammation" and has been widely implicated in hematological cancers as well as other conditions. Meanwhile, the Cambridge, Mass.-based firm's $65 million series B round will push that prospect into the clinic and advance the pipeline of other candidates in cancer and immunology. So far, the Pegasus protein degradation platform has yielded preclinical data packages that support drug-like properties of Kymera's assets as well as differentiated pharmacology, Kymera said.
Harpoon Therapeutics Inc.'s series C financing of $70 million plus cash on hand "basically enables all four of [our] development programs" to enter the clinic, CEO Gerald McMahon told BioWorld. "Near-term, we'll have clinical data on the first two in the 2019-2020 time frame."
Cabaletta Bio Inc. CEO Steven Nichtberger noted that his firm is "not the first to develop chimeric T cells as therapeutics, [but] we are the first to develop them in a way that leverages the potential of T cells to specifically eliminate only the B cells that cause autoimmune disease, thus sparing the healthy cells," and he told BioWorld that the lead indication – mucosal pemphigus vulgaris (PV) – represents a "hallmark for potential efficacy in a whole host of B-cell-mediated autoimmune diseases."
During a recent conference call by Oppenheimer & Co. to advise investors about what's ahead and point out valuable names in the sector, analyst Hartaj Singh took a high-altitude view, calling this "a weird time" for raising money when compared to trends in previous eras.
On the regulatory front Friday, one pain drug company, Trevena Inc., lost and another, Acelrx Pharmaceuticals Inc., won, as the FDA – just for good measure – also cleared a biosimilar product from Coherus Biosciences Inc. in a busy afternoon of notifications.
As expected, Dublin-based Alkermes plc encountered rough waters in the joint meeting of the FDA's Psychopharmacologic Drugs Advisory Committee and Drug Safety and Risk Management Advisory Committee, which deliberated over ALKS-5461 sublingual tablets for the adjunctive treatment of major depressive disorder (MDD).
