Roche has acquired exclusive commercial rights outside the U.S. to Sarepta Therapeutics’ lead gene therapy pipeline candidate SRP-9001 (AAVrh74.MHCK7.micro-dystrophin) for Duchenne muscular dystrophy (DMD), through a licensing agreement that could generate more than $2.85 billion for Sarepta, the companies said today.
SRP-9001, currently in Phase II clinical development, is designed to deliver the micro-dystrophin-encoding gene directly to the muscle tissue for the targeted production of the micro-dystrophin protein.
Sarepta is recruiting patients for the two-part Phase II SRP-9001-102 trial (NCT03769116), a 40-patient study designed to assess the safety and efficacy of SRP-9001 in a 48-week randomized, double-blinded, placebo-controlled period (Part 1), and a 96-week, double-blinded extension period (Part 2). The study has an estimated primary completion date of October 10, 2022.
The trial has two sites: one at Nationwide Children’s Hospital led by Jerry R. Mendell, MD, and a second at UCLA led by Perry B. Shieh, MD, PhD. “Both sites are actively dosing patients and we remain on target to complete our dosing by year-end,” Sarepta President and CEO Douglas Ingram told analysts November 7 on the company’s third-quarter conference call, according to a transcript published by Seeking Alpha.
SRP-9001 will also be evaluated in an upcoming clinical study, referred to as study 103 or 301, and set to start in mid-2020. Ingram told analysts the multi-center, multi-country, placebo-controlled trial will use commercial process material from the company’s hybrid manufacturing model with Brammer Bio (now Thermo Fisher Scientific) and Paragon Gene Therapy, a unit of Catalent Biologics. The main study will include DMD patients ages 4 to 7, with a separate study planned for older and non-ambulatory patients as well.
“This licensing agreement instills added confidence in SRPT [Sarepta]’s gene therapy programs and removes any remaining financial overhang for SRPT, in our view,” Joseph P. Schwartz, managing director, rare diseases with SVB Leerink, and two of the firm’s equity research associates, Dae Gon Ha, PhD, and Joori Park, PhD, concluded today in an investor note.
“To the extent that RHHBY [Roche] likely critically evaluated SRPT’s manufacturing and clinical probability of success, the deal may bode well for the ability to produce enough SRP-9001 via SRPT’s hybrid adherent-based approach and SRP-9001’s likelihood to succeed in the ongoing ‘102 and upcoming ‘103 studies.”
“A Suitable Partner”
“We see RHHBY as a suitable partner that will be able to extend SRP-9001’s reach globally,” Schwartz, Ha, and Park added.
The licensing agreement is intended to accelerate Sarepta’s ex-U.S. development of SRP-9001 through Roche’s global reach, commercial presence and regulatory expertise, Roche and Sarepta said.
“This collaboration will not only increase the speed with which SRP-9001 could benefit DMD patients outside the United States, but will also greatly expand the scope of territories within which we could potentially launch SRP-9001 and improve and save lives,” Ingram said in a statement.
Sarepta has agreed to continue overseeing the global development plan and manufacturing buildout for SRP-9001. Through its leading hybrid manufacturing platform, Sarepta said, it will remain responsible for manufacturing of clinical and commercial supplies.
Sarepta said it has also granted Roche an option to acquire ex-U.S. rights to unspecified future DMD-specific programs, in exchange for separate milestone and royalty considerations, and cost sharing.
“By working together to provide SRP-9001 to patients, we hope to fundamentally transform the lives of patients and families living with this devastating disorder for which there are currently only limited treatment options,” added James Sabry, Head of Roche Pharma Partnering.
Roche has agreed to pay Sarepta at least $1.15 billion, to consist of $750 million cash upfront and a $400 million equity investment; up to $1.7 billion in payments tied to achieving regulatory and sales milestones; and royalties on net sales, anticipated to be in the mid-teens.
In addition, Roche and Sarepta said, they will equally share global development expenses.
The licensing agreement is anticipated to close in the first quarter of 2020, subject to the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and other customary conditions.
Sarepta retains all rights to SRP-9001 in the U.S.
“In addition to the validation that comes from joining forces with Roche, this licensing agreement—one of the most significant ex-U.S. licensing transactions in biopharma—will provide Sarepta with the resources and focus to accelerate our gene therapy engine and, if successful, bring SRP-9001 to patients as quickly as possible, potentially transforming the lives of countless DMD patients across the globe,” Ingram added.