-- Out-of-specification lot resulted from the presence of trace levels of DNA fragment in research-grade . . raw material plasmid sourced from third-party manufacturer --
-- Fragment fully characterized; preliminary testing and analysis indicates no safety signals --
-- Subject to FDA review of a corrective action plan, which will include the use of GMP-s plasmid for all . . . future production lots --
-- Clinical timeline to commence dosing of patients in pivotal trial by year-end 2018 remains on track --
-- Sarepta to host conference call on Wednesday, July 25, 2018 at 5:00 p.m. ET --
CAMBRIDGE, Mass., July 25, 2018 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, has been notified by the Research Institute at Nationwide Children’s Hospital (the Research Institute) that they have received a letter from the Food and Drug Administration (FDA) on July 24, 2018, stating that their Phase 1/2a Duchenne Muscular Dystrophy (DMD) Micro-Dystrophin Gene Therapy Trial has been placed on clinical hold due to the presence of a trace amount of DNA fragment in research-grade third-party supplied plasmid. Preliminary in-vivo testing performed by the Research Institute indicates that the trace fragment does not result in protein expression and is quickly cleared.
The Research Institute, working with Sarepta, has developed their action plan with immediate plans to submit for review by the FDA, which will include the use of GMP-s plasmid for the program. Subject to the FDA’s acceptance of the action plan, Sarepta does not anticipate any material delay in dosing patients as originally planned by year-end 2018.
“Patient safety is our top priority at Sarepta as we know it is for Nationwide Children’s Research Institute,” stated Doug Ingram, Sarepta’s president and chief executive officer. “We intend to rapidly respond to the FDA’s clinical hold letter, including a commitment to the Agency to only use GMP-s plasmid. Independently, we will also request a meeting with the Agency to discuss the micro-dystrophin program with the goal of commencing a pivotal trial by year-end 2018.”
Sarepta will host a conference call today, Wednesday, July 25, 2018 at 5:00 p.m. ET. The conference call may be accessed by dialing 844-534-7313 for domestic callers and +1-574-990-1451 for international callers. The passcode for the call is 8689739. Please specify to the operator that you would like to join the "Sarepta Conference Call." The conference call will be webcast live under the investor relations section of Sarepta's website at www.sarepta.com and will be archived there following the call for 90 days. Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.
About Sarepta Therapeutics
Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying Duchenne muscular dystrophy (DMD) drug candidates. For more information, please visit www.sarepta.com.
For more information, visit sareptatherapeutics.com