Ultragenyx Pharmaceutical Inc. anticipates a meeting later this year with the U.S. FDA to discuss a BLA filing for gene therapy DTX-401 as the first potential medical treatment for glycogen storage disease type 1a (GSD1a) after the phase III study hit its primary endpoint and two key secondary endpoints.
After shares climbed 36% on May 24 following the release of an American Society of Clinical Oncology abstract detailing an impressive phase II overall response rate in first-line head and neck cancer with bispecific antibody petosemtamab in combination with pembrolizumab, Merus NV is raising $400.2 million in an upsized follow-on offering.
The American Society of Clinical Oncology (ASCO) begins its 2024 annual meeting at the cavernous and labyrinthine McCormick Place convention center in Chicago Friday, May 31. It’s one of the world’s largest cancer research conferences and can be daunting to follow. More than 400 organizations will participate this year, with about 200 sessions ready to convene. The vast majority of the 5,000 abstracts that cover all aspects of cancer treatment have already been released, and they will be scrutinized by the more than 40,000 attendees from around the world.
Interim data from Biohaven Ltd.’s phase I study of BHV-1300, an IgG degrader using an ASGRP-bispecific, produced dose-dependent results in its first four cohorts, though not enough to satisfy investors.
Neuren Pharmaceuticals Ltd.’s NNZ-2591 met the primary endpoints in a phase II trial in children with Pitt Hopkins syndrome, with improvements seen in communication, social interaction, cognition and motor abilities, according to top-line results.
Insmed Inc.’s phase III study of brensocatib in treating noncystic fibrosis bronchiectasis hit its primary and multiple secondary endpoints, impressing investors with statistically significant results. The positive data could lead to the first drug approval for the treating the deadly lung disease.
Shares of Redwood City, Calif.-based Rezolute Inc., and Korean parent company Handok Inc., rose May 22 on positive results of a phase II proof-of-concept study for its investigative oral diabetic macular edema (DME) drug, RZ-402. RZ-402 is an oral small-molecule plasma kallikrein inhibitor designed to block vascular leakage and inflammation for treating chronic DME.
Innovent Biologics Inc.’s picankibart (IBI-112) met all primary endpoints and key secondary endpoints in the phase III registrational Clear-1 trial in Chinese subjects with moderate to severe plaque psoriasis.
Researchers affiliated with the U.K.’s University Hospitals Birmingham NHS Foundation Trust filed for protection of a device for treating simple or complex anal or rectovaginal fistulas which is less invasive and more effective than existing treatments. The device combines a bioresorbable scaffold to heal the fistula and a thin comfortable seton to achieve drainage.
As the company began a rolling NDA submission to the U.S. FDA for its drug combo in low-grade serous ovarian cancer, Verastem Oncology Inc. popped the lid off phase I/II data in pancreatic tumors, but Wall Street seemed uncertain about the news. Boston-based Verastem disclosed upbeat outcomes from the Ramp 205 study testing the RAF/MEK clamp avutometinib when paired with focal adhesion kinase inhibitor defactinib in combination with gemcitabine plus nab-paclitaxel as first-line therapy for metastatic pancreatic cancer.
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