Cell and gene therapy companies are the beneficiaries of positive changes along the regulatory path that the U.S. FDA is paving for them, according to a panel of executives who spoke at the BioFuture 2024 conference in New York earlier today. The agency is trying to set up cell and gene companies for success and that’s a very difference agency than what it was years ago, said Paul Bresge, CEO of Ray Therapeutics Inc. Parastoo Khoshakhlagh, the CEO and co-founder GC Therapeutics Inc., agreed that the FDA has worked to open pathways that were not previously there. She added that Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, has been open minded and led a huge change that has occurred since she worked there years ago.

Tharimmune surges on EMA feedback for PBC pruritus drug

Shares of Tharimmune Inc. shot up more than 100% in early trading on the latest update for TH-104, a transdermal buccal film version of nalmefene, set to start phase II testing in pruritus associated with primary biliary cholangitis (PBC). Following a scientific advice meeting with the EMA, Tharimmune said it received positive feedback from the agency for the upcoming phase II study, expected to assess safety and tolerability of TH-104, as well as the change from baseline in itch scores. Top-line data are expected in 2025.

Lexeo dips on early data with gene therapy in Alzheimer’s

Lexeo Therapeutics Inc. offered positive interim results from the phase I/II study of LX-1001 for the treatment of APOE4-associated Alzheimer’s disease (AD). Treatment with the adeno-associated viral vector gene therapy led to dose-dependent increases in APOE2 protein expression and improvements in AD-associated tau biomarkers, measures that have been closely correlated with cognitive outcomes, the New York-based company noted. LX-1001 also turned up a favorable safety profile with no reports of amyloid-related imaging abnormalities. Shares (NASDAQ:LXEO) were trading at $8.38, down $1.59, or 16%.

Biogen, Neomorph pact worth $1.45B for molecular glue degraders

Two days after Monte Rosa Therapeutics Inc. signed a molecular glue degrader deal with Novartis AG, two other companies, Biogen Inc. and Neomorph Inc., are moving forward in the same space in a partnership worth up to $1.45 billion. Cambridge, Mass.-based Biogen and San Diego-based Neomorph will develop molecular glue degraders (MGDs) for priority targets in Alzheimer’s, rare neurological and immunological diseases, using Neomorph’s MGD platform to identify and validate novel small-molecule protein degraders.

Synox extends series B by $17B to advance emactuzumab

Synox Therapeutics Ltd. extended its series B by a further $17 million as it announced the first patients have been dosed in a phase III study of emactuzumab in the treatment of tenosynovial giant cell tumor (TGCT), a rare condition in which benign tumors grow in the soft tissue lining of joints and tendons. The new investment, led by new investor Gilde Healthcare, brings the total for the round to $92 million. The fresh funding gives Oxford, U.K.-based Synox “extra optionality” in terms of its strategy, said Ray Barlow, CEO. “We are planning for success and it is always helpful to have capital available to support the successful registration and commercialization of emactuzumab,” he told BioWorld.

Shionogi finds positive data on ensitrelvir for COVID-19

Phase III Scorpio-PEP study results showed Shionogi & Co. Ltd.’s oral antiviral ensitrelvir fumaric acid (Xocova) reduced the risk of symptomatic COVID-19 infection in subjects who were exposed to the virus by infected household members. Scorpio-PEP is the first to show that an oral, once-daily antiviral drug could be used as a post-exposure prophylactic (PEP) therapy to block the onset of COVID-19 symptoms in subjects when ensitrelvir was administered within 72 hours of exposure, Osaka, Japan-based Shionogi said.

Also in the news

Abeona, Aion, Allogene, Aprinoia, Biora, Biovie, Cantargia, Chimagen, Clover, Coya, Curis, Cytodyn, Dr. Falk, Eledon, Fina, GC, Gemvax, Glycomimetics, GSK, Hopo, Innovent, Invivyd, Kind, Kynos, Maia, Monopar, Ncage, Nektar, Neuvivo, Nkgen, Novartis, Novelty Nobility, Pathos, Protega, Purdue, Regeneration, Remegen, Ryvu, Shorla, Tikkunlev, Valanx, Vaxinano, Vaxxas, Zai Lab