The data will be presented during a platform presentation at the 19th Annual WORLDSymposium in
'There is tremendous need for new treatment approaches in progressive, debilitating, genetic diseases like LOPD that move beyond standard of care enzyme replacement therapy (ERT),' said
FORTIS is an ongoing multicenter, open-label, ascending dose Phase 1/2 first-in-human clinical trial to determine the safety, tolerability and exploratory efficacy of AT845 in adults with LOPD. As of the
Each participant's infusion of AT845 was generally well-tolerated, and most treatment-emergent adverse effects were mild (grade 1) and considered to be unrelated to study treatment. A possible infusion-related reaction occurred in one participant and resolved with oral diphenhydramine and acetaminophen. Three participants developed transient transaminitis and deemed possibly related to AT845. In all cases, the event resolved with modifications to immune suppression. A grade 2 peripheral sensory polyneuropathy event was reported in one participant in the 6x1013 vg/kg cohort, which led to an FDA clinical hold in
'We are excited to share these new findings from the ongoing FORTIS clinical study of AT845. These data, along with our recent announcement of the clinical hold lift of the FORTIS clinical trial, are very positive developments for the program,' said
About Pompe Disease
Pompe disease is a rare, severe, autosomal recessive metabolic disease characterized by progressive neuromuscular degeneration. The overall incidence is estimated to be approximately 1 in 40,000 births1, although frequency and disease progression varies with age of onset, ethnicity and geography2. The disease is caused by mutations in the acid alpha-glucosidase (GAA) gene that prevent the production and function of a protein called acid alpha-glucosidase (GAA). GAA is responsible for metabolizing glycogen, and dysfunction or absence of this protein results in the accumulation of glycogen in tissues, primarily in the skeletal and cardiac muscles, where it causes damage to tissue structure and function. Currently, the only approved treatment for Pompe is enzyme replacement therapy (ERT), which is a chronic treatment delivered in bi-weekly infusions and relies solely on tissue uptake of GAA from plasma.
About AT845 for the treatment of Late-Onset Pompe Disease (LOPD)
Astellas is developing AT845, a novel gene replacement therapy using an AAV8 vector under a muscle-specific promotor to deliver a functional copy of the GAA gene, for the treatment of adult LOPD. AT845 is being investigated to determine whether it can deliver a functional GAA gene that is efficiently transduced to express GAA directly in tissues affected by the disease, including skeletal and cardiac muscle.
About FORTIS
FORTIS (NCT04174105) is a multicenter, open-label, ascending dose Phase 1/2 first-in-human clinical trial to determine if AT845 is safe and tolerable in adults with Late-Onset Pompe Disease (LOPD). The primary endpoints of the trial are safety and tolerability, as well as efficacy measures, including change in muscle GAA protein expression and enzyme activity from baseline. Secondary endpoints evaluate improvements in respiratory, endurance and quality of life measures.
About Astellas
About Astellas Gene Therapies
Astellas Gene Therapies is an
Astellas Cautionary Notes
In this press release, statements made with respect to current plans, estimates, strategies and beliefs and other statements that are not historical facts are forward-looking statements about the future performance of Astellas. These statements are based on management's current assumptions and beliefs in light of the information currently available to it and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in general economic conditions and in laws and regulations, relating to pharmaceutical markets, (ii) currency exchange rate fluctuations, (iii) delays in new product launches, (iv) the inability of Astellas to market existing and new products effectively, (v) the inability of Astellas to continue to effectively research and develop products accepted by customers in highly competitive markets, and (vi) infringements of Astellas' intellectual property rights by third parties. Information about pharmaceutical products (including products currently in development) which is included in this press release is not intended to constitute an advertisement or medical advice.
Contact:
Tel: +1-347-226-1459
Email: Sara.zelkovic@astellas.com
(C) 2023 Electronic News Publishing, source