With dosing complete in its phase II trial testing Ra Pharmaceuticals Inc.'s RA-101495 in generalized myasthenia gravis (MG) and date likely to roll out near the end of this year, the world already has begun weighing odds of the compound in a space where the likes of Alexion Pharmaceuticals Inc. and Argenx SE are formidable players.
With dosing complete in its phase II trial testing Ra Pharmaceuticals Inc.'s RA-101495 in generalized myasthenia gravis (MG) and date likely to roll out near the end of this year, the world already has begun weighing odds of the compound in a space where the likes of Alexion Pharmaceuticals Inc. and Argenx SE are formidable players.
Full results are not due until the spring, but investors meanwhile didn't seem much enthused by Dublin-based Alkermes plc's positive top-line results from Enlighten-2, a pivotal phase III study of ALKS-3831 (olanzapine/samidorphan), a once-daily, oral atypical antipsychotic prospect for schizophrenia.
Cancer data typically steal the show at the American Society of Hematology (ASH) meeting, but hemophilia often gets plenty of play as well, and this time around could be especially compelling for investors in firms with therapies in the works for the clotting disorder.
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."
