Lee Landenberger

The U.S. FDA has greenlit the first steps of Uniqure NV’s accelerated approval pathway for gene therapy AMT-130 to treat Huntington’s disease. The agency said data from the ongoing phase I/II studies compared to natural history external control are muscular enough to get the process going without having to dive into additional studies.

Sound Pharmaceuticals Inc.’s lead candidate, the anti-inflammatory SPI-1005 (ebselen), has produced positive pivotal phase III study data in treating Meniere’s disease, an inner-ear disorder that can lead to vertigo and deafness.

After Keros Therapeutics Inc.’s voluntary halt of dosing in two arms of its phase II study in pulmonary arterial hypertension, the company’s stock crumpled after a year of muscular performance and its recent and massive deal with Takeda Pharmaceutical Co. Ltd.

Mixed results for three of Bicycle Therapeutics plc’s zelenectide pevedotin development programs preceded a strong drop in the company’s stock. Bicycle shares (NASDAQ:BCYC) dropped 31.3% on Dec. 13 to close at $13.81 each, the stock’s lowest price in the past 12 months.

While Spruce Biosciences Inc.’s tildacerfont missed its phase II primary endpoint in classic congenital adrenal hyperplasia (CAH) last March, the U.S. FDA approved Crenessity (crinecerfont) from Neurocrine Biosciences Inc. for treating pediatric and adult CAH patients. The nod could lead the drug to become a blockbuster, analysts said.

Released from a clinical hold by the U.S. FDA in late September, Biomea Fusion Inc.’s menin inhibitor, icovamenib, has charged ahead to produce positive top-line data from a phase II study in type 2 diabetes (T2D). The data, however, didn’t stop the stock from dropping to near its lowest level of the past 12 months.

Merck & Co. Inc. has turned to Asia for a second time to get into the GLP-1 market, this time to Shanghai-based Hansoh Pharmaceutical Group Co. Ltd. for its investigational preclinical oral small-molecule GLP-1 receptor agonist. Hansoh is getting $112 million up front and could bring in another $1.9 billion in milestone payments.

Sangamo Therapeutics Inc.’s second large, worldwide licensing deal for its capsid technology in the past five months is with Astellas Pharma Inc. The California-based company is getting $20 million up front and the chance to bring in up to $1.3 billion in fees and milestone payments in an agreement spanning five potential disease targets for gene therapies to treat neurological diseases.

The first acellular tissue engineered vessel for adults with vascular trauma in a bodily extremity has been approved by the U.S. FDA. Symvess, which comes from regenerative tissue developer Humacyte Inc., can be used to repair an arterial injury that could be life threatening and require immediate attention.

With Alhemo (concizumab-mtci) from Novo Nordisk A/S, a third hemophilia drug in the past eight months has been approved by the U.S. FDA. The once-daily injectable prophylaxis is to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or B with inhibitors.