Savara Inc.'s stock (NASDAQ:SVRA) plummeted 75.2% Thursday as top-line data from Impala, the phase III trial of Molgradex, showed the study failed to meet its primary endpoint in rare lung disease autoimmune pulmonary alveolar proteinosis.
Swedish Orphan Biovitrum AB (Sobi) shed some old skin to grow some new by expanding its Novimmune SA deal by acquiring emapalumab (NI-0501, branded as Gamifant) and all its related assets. Sobi also gets all emapalumab employees involved in the drug's clinical and biopharmaceutical development.
Deciphera Pharmaceuticals Inc. and Zai Lab Ltd. agreed to develop and commercialize ripretinib in mainland China, Hong Kong, Macau and Taiwan for patients with gastrointestinal stromal tumors (GIST).
Artizan Biosciences Inc. has kept a low profile since its creation in 2016, quietly raising money and forging a path to finding a cure for certain diseases involving human intestinal microbiota. It stepped out Monday when it announced completion of a series A funding round totaling $12 million.
Vertex Pharmaceuticals Inc. plans to up its gene editing game by expanding an existing collaboration with Crispr Therapeutics AG and acquiring privately held Exonics Therapeutics Inc. to develop therapies for Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1).
Axovant Sciences Ltd. has terminated its $187.5 million license and collaboration agreement with Benitec Biopharma Ltd., a Sydney-based gene therapy company, regarding AXO-AAV-OPMD (formerly BB-301) for treating oculopharyngeal muscular dystrophy (OPMD).
The FDA has approved Indianapolis-based Eli Lilly and Co.'s Emgality (galcanezumab-gnlm) solution (300 mg) for injection for treating episodic cluster headache in adults, making it the first and only calcitonin gene-related peptide (CGRP) inhibitor that reduces the frequency of these attacks.
Arvinas Inc. and Bayer AG will leverage Arvinas' PROTAC (PROteolysis TArgeting Chimeras) protein degrader platform to develop therapeutics for patients with cardiovascular, oncological and gynecological diseases.
Ridgeback Biotherapeutics LP said the FDA has granted orphan drug status to mAb-114, an experimental treatment for Ebola. It’s a step forward for the 4-year-old biotech focused on pediatric orphan and infectious diseases with limited or no treatment options for patients in the developing world.
Struggling Mereo Biopharma Group plc disclosed six-month data from the open-label arm of its phase IIb dose-ranging study in adults with type I, III or IV osteogenesis imperfecta treated with BPS-804 (setrusumab). Denise Scots-Knight, Mereo's CEO, said results from the Asteroid study were in line with company expectations and she expects 12-month headline data from blinded arms of the study in the second half of this year.
