In autumn 2017, Audentes Therapeutics announced it had commenced dosing of patients in ASPIRO, a Phase 1/2 clinical trial of AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM). ASPIRO is a multicenter, multinational, open-label, ascending dose study to evaluate the safety and preliminary efficacy of AT132 in approximately twelve x-linked myotubular myopathy patients less than five years of age.
Suyash Prasad, M.D., Senior Vice President and Chief Medical Officer said “We are grateful to the many expert collaborators, patient advocacy organizations and parents who have supported Audentes and helped guide our efforts. Most of all we are thankful to the children and families affected by XLMTM for their participation in ASPIRO. We look forward to working together with our partners to advance AT132 as a potentially transformative therapy to treat this devastating disease.”
In addition to ASPIRO, the clinical development program for AT132 includes RECENSUS, a retrospective medical chart review, for which Audentes has previously announced data from an initial analysis of 112 male subjects. This analysis confirmed and expanded upon the understanding of the significant disease burden of XLMTM on patients, families and the healthcare system. Audentes is also conducting INCEPTUS, a prospective natural history run-in study. The primary objectives of INCEPTUS are to characterise the clinical condition of children with XLMTM, identify subjects for potential enrollment in ASPIRO, and serve as a longitundinal baseline and within-patient control for ASPIRO. Audentes plans to announce preliminary data from INCEPTUS in the third quarter of 2017.
In November Audentes announced that AT132 had been well-tolerated by all patients with no significant treatment-related safety signals to date and the plan was to report preliminary clinical data from ASPIRO in early January 2018.
Matthew R. Patterson, President and Chief Executive Officer said “I am pleased with the excellent progress we have made to advance our pipeline toward key value inflection milestones, including the recent completion of dosing for the first cohort in ASPIRO, the Phase 1 / 2 clinical study of AT132 for the treatment of XLMTM. While we are only weeks into the study, we are encouraged by the progress to date and look forward to sharing preliminary safety and efficacy data from the first cohort of patients in early January 2018. This will be the first of many catalysts over the coming months as we execute on our ambitious goal of creating the world’s leading gene therapy company focused on developing a multi-product pipeline to treat serious, life-threatening rare diseases with high unmet medical need.”
Rare Pediatric Disease and fast track designations for AT132
Audentes also announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease and Fast Track designations for AT132, the company’s gene therapy product candidate being developed to treat XLMTM. In addition to these two new designations, AT132 has also received Orphan Drug designation from both the FDA and European Medicines Agency.
Matthew R. Patterson stated “The incentives provided by the Rare Pediatric Disease and Fast Track designations are significant and include opportunities to work with FDA to expedite the development of AT132 and the potential to obtain a valuable Rare Pediatric Disease Priority Review Voucher upon approval. We are eager to leverage these benefits and to work closely with the FDA and XLMTM patient community as we pursue our goal of developing AT132 to treat this devastating rare disease.”
A Rare Pediatric Disease designation may be granted by the FDA to drugs and biologics intended to treat orphan diseases affecting fewer than 200,000 patients in the United States, primarily age 18 years or younger. The designation provides incentives to advance the development of rare disease drugs, including access to the FDA’s expedited review and approval programs.
In addition, under the FDA’s Rare Pediatric Disease Priority Review Voucher program, a sponsor that receives approval for a biologics license application for a rare pediatric disease may be eligible to receive a voucher for a priority review of a subsequent marketing application for a different product.
The priority review voucher may be used by the sponsor, sold or transferred. The Fast Track program was created by the FDA to facilitate the development and expedite the review of new drugs which show promise in treating a serious or life-threatening disease and address an unmet medical need. Drugs that receive this designation benefit from more frequent communications and meetings with FDA to review the drug’s development plan including the design of the proposed clinical trials, use of biomarkers and the extent of data needed for approval. Drugs with Fast Track Designation may qualify for priority review to expedite the FDA review process, if relevant criteria are met.