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.
Astellas Pharma Inc.’s early 2018 buyout of Universal Cells Inc. (UC) may have laid the groundwork for longer-range steps in allogeneic CAR T-cell therapy, but Xyphos Biosciences Inc. CEO James Knighton told BioWorld Asia that the buyout of his firm provides the Tokyo-based giant for now with “an incredibly elegant solution that has tremendous potential.”
Shares of Cambridge, Mass.-based Wave Life Sciences Ltd. (NASDAQ:WVE) lost 49.5% of their value, or $7.82, to close at $7.99 as investors learned of top-line data from the ongoing phase Ib/IIa Precision HD2 trial testing WVE-120102 in Huntington’s disease.
