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Pharma Update: FDA Eases CGT Path, Tharimmune Surges on EMA Feedback, Lexeo Dips on Alzheimer's Data

• The FDA is creating a more accessible regulatory environment for cell and gene therapy companies, according to executives at BioFuture 2024. • Tharimmune's stock soared following positive feedback from the EMA regarding its phase II study of TH-104 for pruritus associated with primary biliary cholangitis (PBC). • Lexeo Therapeutics experienced a stock dip despite releasing positive interim results from its phase I/II study of LX-1001 for APOE4-associated Alzheimer’s disease. • Biogen and Neomorph have entered into a partnership worth up to $1.45 billion to develop molecular glue degraders for neurological and immunological diseases.

Cell and gene therapy companies are seeing a more streamlined regulatory pathway thanks to the FDA, according to discussions at the BioFuture 2024 conference. Executives noted a significant shift in the agency's approach, aiming to foster success in the field.

Tharimmune's TH-104 Shows Promise in PBC-Associated Pruritus

Tharimmune Inc. witnessed a significant surge in its stock value after receiving encouraging feedback from the European Medicines Agency (EMA) regarding its TH-104. TH-104, a transdermal buccal film formulation of nalmefene, is slated for phase II testing to alleviate pruritus linked to primary biliary cholangitis (PBC). The upcoming phase II trial will primarily assess the safety and tolerability of TH-104, alongside monitoring changes in itch scores from baseline. Top-line data from this study are anticipated in 2025.

Lexeo's LX-1001 for Alzheimer's Shows Early Promise, Market Hesitation

Lexeo Therapeutics Inc. presented interim data from its phase I/II trial of LX-1001, a gene therapy targeting APOE4-associated Alzheimer’s disease (AD). The adeno-associated viral vector gene therapy demonstrated dose-dependent increases in APOE2 protein expression and improvements in AD-related tau biomarkers, which correlate with cognitive outcomes. LX-1001 also exhibited a favorable safety profile, with no amyloid-related imaging abnormalities reported. Despite the positive data, Lexeo's shares experienced a downturn, trading at $8.38, a decrease of $1.59, or 16%.

Biogen and Neomorph Collaborate on Molecular Glue Degraders

Biogen Inc. and Neomorph Inc. are partnering to develop molecular glue degraders (MGDs) for Alzheimer’s, rare neurological, and immunological diseases. The collaboration, valued at up to $1.45 billion, will leverage Neomorph’s MGD platform to identify and validate novel small-molecule protein degraders for priority targets.

Synox Extends Series B Funding for Emactuzumab in TGCT

Synox Therapeutics Ltd. has extended its series B funding by $17 million, coinciding with the initiation of a phase III trial of emactuzumab for tenosynovial giant cell tumor (TGCT). The additional investment, led by Gilde Healthcare, brings the total series B funding to $92 million. Emactuzumab is being developed to treat this rare condition characterized by benign tumors in the soft tissue lining of joints and tendons.

Shionogi's Ensitrelvir Shows Positive Prophylactic Data Against COVID-19

Results from the phase III Scorpio-PEP study indicate that Shionogi & Co. Ltd.’s oral antiviral, ensitrelvir fumaric acid (Xocova), reduces the risk of symptomatic COVID-19 infection in individuals exposed to the virus by infected household members. The study demonstrated that ensitrelvir, administered within 72 hours of exposure, could effectively block the onset of COVID-19 symptoms as a post-exposure prophylactic (PEP) therapy.
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Reference News

[1]
BioFuture 2024: FDA eases the way for cell and gene therapy companies | BioWorld
bioworld.com · Oct 31, 2024

FDA's regulatory changes benefit cell and gene therapy companies; Tharimmune's TH-104 receives positive EMA feedback; Le...

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