Today, 21 August, Audentes Therapeutics announced that they have been granted RMAT, or Regenerative Medicine Advanced Therapy designation to AT132 for the treatment of x-linked myotubular myopathy. Both RMAT, and it’s EU equivalent known as PRIME designation which was granted to A132 earlier this month, are very similar in that they provide organisational commitment from regulators to drive envelopment as quickly as possible.

In terms of the RMAT designation announced today:

– Designation granted based on positive interim data from ASPIRO clinical trial

– RMAT designation enables closer and more frequent multidisciplinary interaction with FDA and includes all of the benefits of breakthrough therapy designation

– Plan to meet with FDA to gain alignment on AT132 development plan

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.

For more information regarding Audentes, please visit www.audentestx.com.

Click here to read the full press release on Audentes Therapeutic’s website.