Axovia Therapeutics Presents New Preclinical Data for AXV-101 for the Treatment of Blindness Associated with BBS Mutations at ASGCT

/EIN News/ -- -  Data support planned dosing regimen and protocol for FIH study to initiate in mid-2025

LONDON, May 13, 2025 (GLOBE NEWSWIRE) -- Axovia Therapeutics Ltd., a biotechnology company developing therapies to address the genetic causes of blindness and obesity, announced the presentation of new preclinical data in two poster presentations at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting, which is being held from May 13-17, 2025 in New Orleans, LA. The two poster presentations include data that support the dosing regimen and protocol for the subretinal Phase 1/2 study for the company’s lead program for Bardet-Biedl Syndrome (BBS), AXV-101, which is expected to initiate in mid-2025.

“As we draw closer to the initiation of our Phase 1/2 first-in-human study mid-year, these presentations at ASCGT highlight critical data supporting our planned dosing regimen and trial protocol,” said Dr. Victor Hernandez, Co-Founder and Chief Scientific Officer. “We believe that our novel gene therapy, AXV-101, can modify the underlying disease of BBS, including prevention of vision loss by halting retinal degeneration and we look forward to generating clinical data from our study, which was thoughtfully designed based on a robust preclinical data package.”

Poster details:

“A new dosing regimen to treat Bardet-Biedl Syndrome 1 (BBS1) retinal degeneration with AXV-101 (AAV9-BBS1) improves histological and functional photoreceptor survival in Bbs1 M390R mice. Finding the right balance between bleb number, dose per bleb and total dose per eye.”

Data highlights demonstrate the enhanced efficacy of a multi-bleb strategy, with lower viral titer per bleb but higher total titer per eye as follows:

• Double bleb of AXV-101 halts retinal degeneration and preserves ONL thickness, while maintaining safety with higher doses per retina
• Longitudinal analysis demonstrates enhanced functional rescue of photoreceptors with double bleb of AXV-101 achieving up to 70% of wild-type retinal function
• AXV-101 restores photoreceptor number and arrestin-C localization up to 6 months post-treatment
  

“Development of a ddPCR Assay to Quantify AXV-101 Levels for a Mouse Biodistribution Study.”

Data demonstrates AXV-101 has a biodistribution restricted to the dosing tissue (eye), with minimal transfer to only nearby neuronal tissues and no distribution to any organ outside the CNS or into the germline as follows:

• The highest copy numbers of AXV-101 were seen in the eye samples from high and then medium dose group animals, with lower levels also detected in CNS tissues (optic nerve and regions of the brain)
• Clear dose dependent distribution in the eye, with the high dose averaging 6344 copies, medium dose 2220 copies and low dose 235 copies
• In the high dose group, there is a small, expected distribution in the optic nerve and brain tissues. The medium dose shows a small distribution in the optic nerve and frontal cortex. In the medium dose group, 4 animals show frontal cortex expression averaging 1300 copies
• Biodistribution throughout the rest of the analyzed organs was minimal, and comparable to controls, showing no spread of the AXV-101 vector outside neuronal tissue

“These preclinical findings are very encouraging and provide important support for the planned dosing regimen and protocol for the Phase 1/2 study,” said Dr. Shehla Mohammed, Clinical Lead for the BBS specialized service at St Thomas’ Hospital, London. “These results represent a critical step forward as Axovia advances this therapy into clinical development, with the ultimate goal of offering the BBS community a much-needed treatment that can address retinal degeneration.”

About Bardet-Biedl Syndrome (BBS)
Bardet-Biedl Syndrome (BBS) is an autosomal recessive disorder associated with primary cilia dysfunction, presenting with retinal degeneration and morbid obesity, amongst other clinical features. There is no curative treatment for BBS. BBS affects between one in 70,000 - one in 100,000 in Europe and North America, with up to 10 times that prevalence in certain populations in the Middle East. In Europe and N. America, BBS1 is the most commonly mutated gene found in BBS patients, with the missense BBS1 M390R mutation being the most common allele.

About Axovia Therapeutics Ltd.
Axovia Therapeutics is leading the development of therapies that address the genetic causes of blindness and obesity syndromes that are driven by cilia dysfunction. Ciliopathies are a group of more than 55 rare inherited genetic diseases linked to more than 950 genes that impact the function of cilia which are critical for protein transport and cellular signaling. The company plans to initiate a clinical study to treat retinal degeneration for its lead program for Bardet-Biedl Syndrome (BBS), AXV-101, in mid-2025, based on robust preclinical efficacy and toxicological data with established scaled GMP manufacturing and patient registries. AXV-101 has achieved U.S. Food and Drug Administration Orphan Drug Designation and Rare Pediatric Disease Designation. The company is developing its second program, AXV-201, for genetic obesity caused by MC4R mutations. Axovia is backed by ALSA Ventures and was formed following decades of work on ciliopathies at University College London by co-founders Professor Phil Beales and Dr. Victor Hernandez. For further information, please visit https://axoviatherapeutics.com.

Contacts:

Professor Phil Beales
Chief Executive Officer
investors@axovia.com