Structure Therapeutics Inc.’s stock (NASDAQ:GPCR) climbed to $49.90, up 33% in early trading Sept. 29 following what analysts call “competitive” and “exceptional” phase Ib data at 28 days of oral small-molecule glucagon-like peptide 1 (GLP-1) receptor agonist GSBR-1290 in healthy overweight or obese individuals. At the same time as the data readout, the company agreed to a $300 million private placement with several large health care institutional and mutual fund investors, extending Structure’s runway through the end of 2026. Data from the multiple ascending-dose (MAD) phase Ib study demonstrated significant weight loss supporting once-daily dosing, as well as an encouraging safety and tolerability profile. The MAD study included 24 individuals who were randomized 3-to-1 to GSBR-1290 or placebo across three dose cohorts, 30 mg, 60 mg or 90 mg. The reduction in mean body weight ranged up to 4.9 kg compared to baseline and up to 4.9% when adjusted for placebo.
Amicus’ Pompe disease treatment gets FDA approval
After a nearly year-long delay pegged to COVID-19 travel restrictions, the U.S. FDA has approved Amicus Therapeutics Inc.’s Pompe disease drug. Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) 65-mg capsules for adults with late-onset Pompe disease will be available immediately at a list price of around $650,000. In November 2022, the FDA had deferred action on the BLA due to being unable to inspect the manufacturing site in China. The company’s stock sputtered some on the approval, possibly to a label restriction on the patient’s weight, something that doesn’t appear on the treatment’s EU approval. Shares (NASDAQ:FOLD) were trading about 3% higher at midday at about $12 each.
Korean biosimilars locked in ‘fierce’ battle to box out competition in US
National support for the biosimilar sector and the domestic industry’s efforts to increase production and sales may not be enough for South Korean biosimilar firms to box out competition in the everchanging regulatory court of the U.S. “Competition in U.S. negotiations and rebates are fierce,” Choi Sung-ho, chairman of the Korean Society for Bioeconomy said. “Even if you get listed, it is crucial to be placed in an advantageous class to lower out-of-pocket costs.”
BioWorld tracks 2,314 phase I-III clinical trial updates from Jan-Aug
January-August 2023 saw clinical trial data up 1.22% compared to the same time period last year. In the first eight months of this year, BioWorld reported on 2,314 drugs in phase I-III, compared to 2,286 in the same time period in 2022. The number of trial updates is down 13.24% from the 2,667 in 2021 and down from 2020’s 2,363, but up 20.46% from 2019’s 1,921.
US ARPA-H program prepared to go live as Congress mulls FY 2024 appropriations
The Advanced Research Projects Agency for Health (ARPA-H) reported the opening of a series of regional offices that focus on both administrative and research assignments, a development that may bolster cures and treatments for tough-to-treat conditions. However, the U.S. House and Senate have different ideas about how much money the agency will receive in 2024, casting a cloud of uncertainty over how many projects the agency will be able to finance in the 12 months that will start on Oct. 1, 2023.
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