Ophthalmic therapy and device developer Alcon SA, of Geneva, further strengthened its portfolio by buying Aerie Pharmaceuticals Inc. for about $770 million in equity. Aerie’s financial guidance for its glaucoma franchise puts net product revenue at $130 million to $140 million for all of 2022.
It’s a step forward and a step back at Pfizer Inc. Even as Pfizer and its partner Biontech SE finished filing an emergency use authorization application with the U.S. FDA seeking to field an updated booster dose of their omicron COVID vaccine, the regulator has made a request of its own, for more data on the company's oral antiviral, Paxlovid (nirmatrelvir + ritonavir), for treating COVID-19. An Aug. 5, 2022, letter from the FDA stipulates post-EUA requirements for Paxlovid, including that Pfizer conduct a trial in patients with a relapse of COVID symptoms, longhand for a “rebound,” after an initial Paxlovid treatment course.
A year from the U.S. FDA’s August 2021 citation of deficiencies in the labeling for Axsome Therapeutics Inc.’s major depressive disorder therapy, Auvelity (dextromethorphan + bupropion), the agency has approved the drug for use by adults. The New York-based company’s stock (NASDAQ:AXSM) took flight on the news, with shares rising 40% to close at $59.55 Aug. 19, surpassing their previous 52-week high of $48.82 and starkly contrasting with a same-period low of $19.38.
Bluebird Bio Inc. isn’t giving out much of the information on the margins for the cost of its newly approved cell-based gene therapy for treating adult and pediatric patients with beta-thalassemia. The numbers will, the company said, come into better focus when another Bluebird drug is approved and launched.
Blue Water Vaccines Inc. is ready to try its hand at developing a monkeypox vaccine. The company is launching an exploratory program to develop a vaccine attempting to present antigens within its norovirus shell and protrusion virus-like particle (VLP) platform.
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.
