SAN FRANCISCO, Aug. 21, 2018 /PRNewswire/ -- Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing innovative gene therapy products for patients living with serious, life-threatening rare diseases, today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to AT132 for the treatment of X-linked Myotubular Myopathy (XLMTM).
"We are pleased that the FDA has granted RMAT designation to AT132, an important regulatory milestone that highlights the transformative potential of AT132 as a therapy to treat XLMTM, a rare, congenital disease characterized by extreme muscle weakness, respiratory failure and early death," stated Mary S. Newman, Senior Vice President, Regulatory Affairs at Audentes. "This news follows our recent announcement of the Priority Medicines, or PRIME, designation received from the European Medicines Agency, and we look forward to collaborating closely with the FDA, EMA, our XLMTM clinical experts and the patient community as we seek to rapidly advance the AT132 development program toward global regulatory approvals."
Established under the 21st Century Cures Act, RMAT designation is a program designed to expedite the development and approval of regenerative medicine products, including gene therapy products. An investigational therapy is eligible for the RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates a potential to address unmet medical needs for that disease or condition. The designation includes all the benefits of the FDA's Fast Track and Breakthrough Therapy designations, and enables the ability to work more closely and frequently with the FDA to discuss surrogate or intermediate endpoints to support the potential acceleration of approval and satisfy post-approval requirements.
RMAT designation for AT132 was granted based on positive interim clinical data from ASPIRO, the ongoing Phase 1/2 clinical study of AT132 for XLMTM. These data show encouraging initial evidence of safety and efficacy in the first dose cohort, including significant improvements in neuromuscular function as assessed by the CHOP-INTEND scale, increased respiratory function as demonstrated by reductions in ventilator dependence and gains in maximal inspiratory pressure (MIP), a measure of respiratory muscle strength. In addition to these functional outcome measures, muscle biopsy results from the first three patients treated in the study at the 24-week timepoint demonstrate highly efficient tissue transduction as indicated by vector copy number, robust myotubularin protein expression as assessed by western blot, and significant improvement in histology.
About AT132 for X-Linked Myotubular Myopathy AT132 is the Audentes product candidate being developed to treat XLMTM, a rare monogenic disease characterized by extreme muscle weakness, respiratory failure and early death, with an estimated 50 percent mortality rate by 18 months of age. XLMTM is caused by mutations in the MTM1 gene, which encodes the protein myotubularin. Myotubularin plays an important role in the development, maintenance and function of skeletal muscle cells. AT132 is comprised of an AAV8 vector containing a functional copy of the MTM1 gene. In August 2018, Audentes reported promising safety, efficacy and muscle biopsy data out to the 24-week timepoint from the first dose cohort of ASPIRO, a multicenter, ascending dose Phase 1/2 clinical study to evaluate the safety and preliminary efficacy of AT132 in approximately 12 XLMTM patients less than five years of age. The preclinical development of AT132 was conducted in collaboration with Genethon (www.genethon.fr).
AT132 has been granted Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric Disease, Fast Track and Orphan Drug designations by the FDA, and Priority Medicines (PRIME) and Orphan Drug designations by the European Medicines Agency (EMA).
About Audentes Therapeutics, Inc. Audentes Therapeutics (Nasdaq: BOLD) is a biotechnology company focused on developing and commercializing innovative gene therapy products for patients living with serious, life-threatening rare diseases. We are currently conducting Phase 1/2 clinical studies of our lead product candidates AT132 for the treatment of X-linked myotubular myopathy (XLMTM) and AT342 for the treatment of Crigler-Najjar syndrome. We have two additional product candidates in development, including AT982 for the treatment of Pompe disease, and AT307 for the treatment of the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia (CASQ2-CPVT). We are a focused, experienced and passionate team committed to forging strong, global relationships with the patient, research and medical communities.
Forward Looking Statements This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: the transformative potential of AT132 to treat XLMTM, and the ability of the company to work with regulatory agencies to rapidly advance the development and potential approval of AT132. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. Although the company believes that the expectations reflected in such forward-looking statements are reasonable, the company cannot guarantee future events, results, actions, levels of activity, performance or achievements, and the timing and results of biotechnology development and potential regulatory approval is inherently uncertain. Forward-looking statements are subject to risks and uncertainties that may cause the company's actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to the company's ability to advance its product candidates, obtain regulatory approval of and ultimately commercial its product candidates, the timing and results of preclinical and clinical trials, the company's ability to fund development activities and achieve development goals, the company's ability to protect intellectual property and other risks and uncertainties described under the heading "Risk Factors" in documents the company files from time to time with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and the company undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof.