The U.S. FDA has approved the first cell-based gene therapy for treating adult and pediatric patients with beta-thalassemia requiring frequent red blood cell transfusions. The $2.8 million wholesale acquisition cost for the one-time I.V. infusion will make it one of the most expensive drugs in the U.S.
Positive data from Unity Biotechnology Inc.’s phase II study of UBX-1325, a senolytic BCL-XL inhibitor for treating diabetic macular edema, boosted the stock out of the penny range. Shares of the South San Francisco-based company’s stock (NASDAQ:UBX) rose sharply, 54% on Aug. 12, to $1.31 each.
Top-line data from Verona Pharma plc’s phase III ENHANCE-2 study show its inhaled ensifentrine hit the primary and secondary endpoints in treating chronic obstructive pulmonary disease (COPD). Next up: data from the companion phase III, ENHANCE-1, which is expected by the end of 2022.
Pfizer Inc. plans to pay about $5.8 billion – total equity value – for Global Blood Therapeutics Inc. (GBT) and its oral sickle cell disease (SCD) treatment Oxbryta (voxelotor). The company reported the enterprise value as $5.4 billion, which includes debt and net cash. If completed, the GBT buy would be the second largest M&A in 2022 after Pfizer’s $6.7 billion buyout of Arena Pharmaceuticals Inc. Oxbryta, which netted about $195 million in sales in 2021, gained U.S. FDA approval in November 2019 for the treatment of SCD in adults and pediatric patients ages 12 and up. The FDA later expanded Oxbryta’s approved uses to SCD patients 4 years of age and older in December 2021.
Now that the U.S. Department of Health and Human Services (HHS) has declared monkeypox a public health emergency, nearly two weeks after a similar declaration from the World Health Organization, the way is cleared for a coordinated response and emergency use authorizations to address supply challenges that could limit the availability of currently approved vaccines. It also has several companies ready to leap into the fray if their preclinical studies show a path to approval. HHS said it just shipped more than 602,000 doses of the Jynneos vaccine to states and jurisdictions, an increase of 266,000 in the past week.
Amgen Inc. is paying $3.7 billion in cash to buy Chemocentryx Inc. The deal, with Amgen paying $52 per share for Chemocentryx stock, brings Amgen Tavneos (avacopan), a first-in-class medicine for treating antineutrophil cytoplasmic antibody-associated vasculitis, which destroys small blood vessels.
Genentech Inc. is paying Kiniksa Pharmaceuticals Ltd. $100 million in up-front and near-term payments for the development and commercialization rights to vixarelimab, a fully human monoclonal antibody. Kiniksa also could receive up to approximately $600 million in certain clinical, regulatory and sales-based milestones, as well as royalties on annual net sales.
After spending a lot of money and putting in seven years of hard work, Astrazeneca plc is discontinuing its phase III Interlink-1 study of monalizumab. Results from an interim futility analysis and an independent data monitoring committee’s recommendation convinced the company to bring it to a halt.
Top-line results from Vistagen Therapeutics Inc.’s Palisade-1 phase III study of PH-94B for treating social anxiety disorder showed the therapy fell shy of the primary endpoint. The South San Francisco-based company’s stock (NASDAQ:VTGN) fell dramatically on the results July 22 as shares closed 86% lower at 15 cents each.
