Vandria SA published phase I data on a potential new mechanism of action in treating Alzheimer’s disease, demonstrating its orally available and brain-penetrant mitochondria-boosting compound, VNA-318, engages its intended target. In addition to passing safety and tolerability assessments, the pharmacokinetic readout for VNA-318 indicates its half-life is suitable for daily dosing and that there is a dose-dependent change in a biomarker of target engagement. The results were presented at the Clinical Trials in Alzheimer’s Disease meeting in San Diego.
Jasper posts phase Ib results in allergic asthma with briquilimab, concludes Beacon probe
Jasper Therapeutics Inc. made public positive data from the phase Ib study called Etesian with KIT-targeting briquilimab in adults with allergic asthma. A single subcutaneous 180-mg dose yielded substantial reductions in sputum eosinophils at six and 12 weeks, as well as improvements over baseline in forced expiratory volume in early plus late asthmatic response. Separately, Jasper said the investigation is finished into the phase Ib/II Beacon study with briquilimab in chronic spontaneous urticaria. The lack of efficacy observed in certain U.S. patients, reported in July, was “not the result of any issues with drug product” and “appears to be the result of patient selection issues.” Jasper shares (NASDAQ:JSPR), having first dropped from .11 to .80 – a reduction of more than 15% – were trading at .83, up 11 cents.
Hightide’s HTD-1801 outperforms Farxiga in type 2 diabetes
Hightide Therapeutics Inc.’s berberine ursodeoxycholate (HTD-1801) met the primary endpoint showing superior improvements in key cardiometabolic markers in patients with type 2 diabetes compared to Astrazeneca plc’s SGLT2 inhibitor, Farxiga (dapagliflozin), in a head-to-head phase III trial. Developed in-house, lead candidate HTD-1801 is a potentially first-in-class new molecular entity that targets the residual risks underlying cardiovascular-kidney-metabolic diseases. It is an orally delivered, anti-inflammatory metabolic modulator with a dual mechanism of action that activates AMP kinase and inhibits the NLRP3 inflammasome to mitigate metabolic dysfunction.
Subcutaneous biologics maker Bao Pharma prices 28M IPO
Bao Pharmaceuticals Co. Ltd., a developer of subcutaneous biologic drugs, priced its IPO at HK6.38 on Dec. 2, aiming to raise about HK billion (US28 million). Trading on the Hong Kong Stock Exchange is set to begin Dec. 10 under the ticker “2659.” The sale of about 37.91 million H shares is expected to render a market capitalization of HK billion. Citic Securities and Guotai Haitong Securities are joint bookrunners. Bao expects net proceeds of HK21.5 million after expenses, which will fund its “two-anti” strategy – developing both antibody and antibiotic drugs worldwide, mainly in China, the U.S. and Europe.
UK/US deal fixes import tariff on drugs at 0% for next 3 years
The U.K. government has secured a deal guaranteeing there will be no tariffs on the £6.6 billion (US.7 billion) of pharmaceutical products exported annually to the U.S., as part of a package of measures under which the U.K. will pay more for patented drugs. The 0% rating will be in force for three years. In return, the price at which the health technology assessment body, the National Institute for health and Care Excellence assesses the cost effectiveness of new drugs will rise from £20,000 to £30,000 per quality adjusted life year, to £25,000 to £35,000.
One-experiment studies open new paths for science publishing
“I love the idea of ‘micropublications’ (preparing one now),” the neurobiologist Oded Rechavi commented on social media in July. The term clearly suggests a short article, and although the publishing model has been around for more than a decade, not everyone is familiar with this type of scientific communication. What are they? What’s their impact? Rechavi, a professor at the School of Biochemistry, Neurobiology and Biophysics at Tel Aviv University, was pointing to an emerging discussion among scientists, the search for alternative formats for their work.
Last-minute changes made ahead of ACIP meeting
U.S. Health and Human Services (HHS) Secretary Robert Kennedy continued his last-minute musical chairs ahead of this week’s meeting of CDC’s Advisory Committee for Immunization Practices (ACIP) when he named Kirk Milhoan as the new chair of the panel. A pediatric cardiologist, Milhoan was first named as an ACIP member in Kennedy’s last round of musical chairs played out three days before the panel’s September meeting. This week’s move came on the heels of Kennedy appointing the former ACIP chair, Martin Kulldorff, as chief science officer for the Office of the Assistant Secretary for Planning and Evaluation at HHS.
US pediatric voucher making moves in Congress
The next stop on the comeback tour for the U.S. FDA’s Rare Pediatric Disease Priority Review Voucher (PPRV) program is the Senate, after the House unanimously passed the Mikaela Naylon Give Kids a Chance Act, H.R. 1262, yesterday. Intended to increase access to innovative treatments for children, the legislation would reauthorize the PPRV through 2029. The program expired a year ago despite several congressional attempts to continue it. Applauding the House passage of the bill, the Biotechnology Innovation Organization called on the Senate “to get this essential legislation across the finish line and reauthorize the PPRV program.” It credited the voucher program with bringing hope to the families of the sickest children by incentivizing the development of treatments for rare pediatric diseases at no cost to taxpayers.
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