The hearing has returned for the first person who has received gene therapy for treating genetic hearing loss in the U.S. Results from Akouos Inc.’s phase I/II study show that within 30 days of receiving AK-OTOF-101, pharmacologic hearing was restored to an 11-year-old who had profound hearing loss from birth. In October 2022, Eli Lilly and Co. acquired Akouos, which specializes in developing AAV hearing loss treatments due to mutations in the otoferlin gene. There are no approved therapies for congenital hearing loss, which is estimated to affect about 1.7 of every 1,000 children born in the U.S., and for those patients, hearing aids and cochlear implants offer limited benefits.

Annovis phase III Parkinson’s report delayed, stock nipped

Shares of Annovis Bio Inc. (NYSE:ANVS) sunk 21%, or $2.48, to trade midday at $9.18 on word that Parkinson’s disease data from the phase III trial with buntanetap are delayed. The Malvern, Pa.-based company said it needs more time for data cleaning. Once known as Posiphen or ANVS-401, buntanetap is designed to attack neurodegeneration by reducing neurotoxic proteins to improve synaptic transmission and axonal transport.

With series A funds, IMU Bio’s Cytatlas identifies cell types and immune signatures

IMU Biosciences Ltd. has raised £11.5 million (US$14.7 million) in a series A round to further develop and commercialize profiling technology that can identify from a blood sample which of more than 2,000 cell types are present in an individual’s immune system. Among other applications, the technology called Cytatlas, is being used to identify immune signatures that are predictive of how donor cells will respond when they are expanded, significantly improving consistency and quality control in the manufacture of cell therapies.

US senators demand answers about FTC’s PBM investigation

What’s the status of the U.S. FTC’s investigation into pharmacy benefit managers’ role in prescription drug prices? Enquiring minds in the Senate really want to know, especially since it’s been more than 18 months since that investigation began. “We urge the FTC to complete its 6(b) study in a timely manner,” 14 senators, led by Sens. Chuck Grassley (R-Iowa) and Maria Cantwell (D-Wash.), wrote in a letter this week to FTC Chair Lina Khan. “In the interim, we request that you publicly release a progress report on the status of your investigation.”

New effort to reform patent eligibility in play in US Senate

The ongoing controversy over the types of inventions that are eligible for patent protection just gained new momentum thanks to a Jan. 23 hearing in the U.S. Senate, during which a bipartisan pair of senators once again pushed the argument that current jurisprudence for subject matter eligibility is hampering life science companies based in the U.S. However, Sen. Chris Coons (D-Del.) also made the case that a failure to reform U.S. patent law would be a signal to innovators that the U.S. is no longer open for business for novel medical diagnostics and biotech therapies among others, a problem he said must be fixed if the American economy is to continue to flourish in the 21st Century.

Sciwind moves to once-weekly dosing after oral GLP-1 delivers strong phase I obesity data

Sciwind Biosciences Co. Ltd.’s oral ecnoglutide (XW-004) produced strong weight loss results after short-term treatments, up to a 6.8% body weight decrease in obese and overweight participants after once-daily dosing for six weeks, according to data from the first four cohorts of the phase I trial. A long-acting, cAMP signaling biased glucagon-like peptide-1 (GLP-1) analog, ecnoglutide is being developed for type 2 diabetes and obesity. Sciwind President and Chief Strategy Officer Weidong Zhong told BioWorld that the weight loss effect seen in the XW-004 phase I trial appears “quite strong compared to other oral GLP-1 drugs.” The oral compound showed good oral bioavailability and plasma exposure, “which made us believe that the drug could be dosed less frequently.“

UK further restricts use of fluoroquinolones

Making them an antibiotic of last resort, the U.K.’s Medicines and Healthcare Products Regulatory Agency (MHRA) introduced new restrictions on the use of fluoroquinolones due to the risk of potentially long-term or irreversible side effects. Effective immediately, the MHRA said fluoroquinolone antibiotics given systemically can only be administered when no other antibiotics are appropriate for use – when other recommended antibiotics have failed, won’t work due to resistance or are unsafe to use in an individual patient.

Novartis slapped with first OPDP letter of the year

Quality-of-life claims in a TV ad for breast cancer drug Kisqali (ribociclib) earned Novartis AG the first untitled letter of 2024 from the U.S. FDA’s Office of Prescription Drug Promotion (OPDP). According to the letter, the ad overstated the efficacy of the drug, “which could lead patients with advanced or metastatic breast cancer, an incurable condition whose treatment involves serious risks, to believe that Kisqali has been shown to be more effective in treating their condition and symptoms … than was actually demonstrated.”

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