Shares of Avidity Biosciences Inc. (NASDAQ:RNA) hit a 52-week high of $39.21 this morning after the company reported promising data from the first efficacy cohort of its phase I/II Fortitude study testing delpacibart braxlosiran (AOC-1020), an antibody oligonucleotide conjugate designed to target the underlying cause of facioscapulohumeral muscular dystrophy (FSHD). Results showed the therapy appeared to “connect the dots” from delivery to muscle cells to reduce DUX4 expression, which correlated with functional improvements. San Diego-based Avidity now aims to add registrational phase III cohorts to the ongoing trial, starting with a biomarker-focused phase III cohort, expected to begin in the second half of 2023, which, if positive, could lead to an accelerated approval filing.

Drug makers not sleeping on orexin in narcolepsy

The recent Sleep 2024 meeting in Houston further ratified interest in orexin as a neuropsychiatry target. A hand of developers are working with drugs that take aim at orexin, also known as hypocretin, in narcolepsy. The neuropeptide is understood to play a crucial role in regulating wakefulness, arousal, and appetite.

Nodthera's NLRP3 inflammasome inhibitor targets cardiometabolic space

After delivering animal data to show that inhibiting the NLRP3 inflammasome induces weight loss in mice, Nodthera Ltd. has now demonstrated this effect translates to humans. In a phase Ib/II study of NT-0796, an orally available, brain-penetrant drug, subjects in both the active and placebo arms all lost weight, due to the calorie-restricted diet they received in the 28-day trial. However, the most pronounced, placebo-adjusted reductions in body weight were among high risk subgroups of NT-0796 dosed subjects. Together with the mice data, this supports the anti-obesity profile of the drug, said Nodthera Chief Scientific Officer Alan Watt.

US court adopts FTC stance in Orange Book listing challenge

The U.S. FTC’s campaign against the Orange Book listing of patents claiming device components gained momentum this week when a federal judge ordered Teva Pharmaceuticals USA Inc. to delist device patents pertaining to its Proair HFA (albuterol sulfate) inhaler. The judge also refused Teva’s request to dismiss antitrust claims brought by Amneal Pharmaceuticals Inc. as part of a patent fight involving the combination drug-device product approved 20 years ago to treat or prevent bronchospasm. Teva was one of the first biopharmas targeted by the FTC last year over its listing of device patents in the FDA’s Orange Book.

Opthea raising AU$227M, enters final phase III stretch for OPT-302

Opthea Ltd. announced it plans to raise up to AU$227.3 million (US$150 million) to extend its cash runway through the data readout for its two phase III pivotal trials of sozinibercept (OPT-302) in wet age-related macular degeneration (AMD). The capital raise consists of an AU$10 million (US$6.6 million) placement and a partially underwritten entitlement offer to raise up to AU$217.3 million (US$143.4 million). “This institutional placement and entitlement offer will strengthen Opthea’s cash position, and we expect to fund operations through the anticipated top-line data readouts of both our pivotal trials, which have enrolled a total of 1,984 wet AMD patients,” said Opthea CEO Fred Guerard.

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60 Degrees, Actinium, Actuate, Alvotech, Alzheon, Annovis, Arcutis, Bright Peak, Capricor, Coya, Deciphera, Destiny, Dong-A St, Eli Lilly, Gannex, Ipsen, Neurona, Newcells, Nimble, Ono, Pfizer, Processa, Regenxbio, Sage, Shaperon, Stada, Tacalyx, Tiziana, Tracon, Treveni, T-Therapeutics, Ultimovacs, Vera, Viridian, Zai Lab