Emendo Biotherapeutics Inc. CEO David Baram told BioWorld his firm’s allele-specific gene-editing approach offers such an advantage over previous methods that “we decided to take the challenge of curing diseases that require the highest precision possible,” and the New York-based firm bears an impressive list of partners. “Doors opened immediately and collaborations formed very fast,” sometimes “even faster than we could digest,” he said.
With a label broadened by the FDA in December and two aspiring competitors apparently picked off via late-stage trial blowups, Amarin Corp. plc finds itself in strong position with fish oil therapy Vascepa (icosapent ethyl) – at least pending the outcome of court proceedings that involve challengers to patents for the cardiovascular (CV) drug.
Cambridge, Mass.-based Blueprint Medicines Corp.’s price tag and label for Ayvakit (avapritinib) caused some chagrin on Wall Street as observers continued to weigh the drug’s odds against ripretinib, the competing drug for which Deciphera Pharmaceuticals Inc. awaits regulatory action.
Philadelphia-based Aro Biotherapeutics Co. CEO Sue Dillon told BioWorld her firm has “incoming interest from other companies to apply Centyrins to other kinds of drug conjugates,” which could mean more deals with the platform like the one sealed with Ionis Pharmaceuticals Inc.
Applied Therapeutics Inc. (ATI) CEO Shoshana Shendelman said positive top-line findings with aldose reductase inhibitor AT-007 in galactosemia were “the data we needed, and we’re going to move as quickly as we can to get in and speak to the FDA about this, not only to firm up our NDA filing but also to get into the pediatric study.”
San Diego-based Atyr Pharma Inc. CEO Sanjay Shukla told BioWorld that the company plans to move into a registrational trial with lead candidate ATYR-1923 in pulmonary sarcoidosis (PS) if data from the ongoing phase Ib/IIa trial, due later this year, turn out positive. Releasing patients from steroid burdens “would be a real game-changer,” he said, noting that people with PS take as much as 25 mg of prednisone per day to control their coughs and shortness of breath.
Apellis Pharmaceuticals Inc.’s CEO, Cedric Francois, said his firm’s phase III study called Pegasus testing pegcetacoplan, or APL-2, in paroxysmal nocturnal hemoglobinuria (PNH) “finally established that there is an important unmet medical need in this disease.”
San Diego-based Atyr Pharma Inc. CEO Sanjay Shukla told BioWorld that the company plans to move into a registrational trial with lead candidate ATYR-1923 in pulmonary sarcoidosis (PS) if data from the ongoing phase Ib/IIa trial, due later this year, turn out positive. Releasing patients from steroid burdens “would be a real game-changer,” he said, noting that people with PS take as much as 25 mg of prednisone per day to control their coughs and shortness of breath.
With many on Wall Street transfixed by the three injectable calcitonin gene-related peptide (CGRP) therapies cleared in the prophylactic migraine market, Satsuma Pharmaceuticals Inc.’s prospects with STS-101 may have gone overlooked, at least until lately.
With many on Wall Street transfixed by the three injectable calcitonin gene-related peptide (CGRP) therapies cleared in the prophylactic migraine market, Satsuma Pharmaceuticals Inc.’s prospects with STS-101 may have gone overlooked, at least until lately.
