Fresh from its drawn-out takeover of Spark Therapeutics, Roche has signed another gene therapy deal, licensing Sarepta’s Duchenne muscular dystrophy (DMD) candidate SRP-9001.
The agreement – which consists of $750m in cash and $400m in equity – gives Roche the rights to SRP-9001 outside the US, with Sarepta also eligible to receive up to $1.7bn in regulatory and sales milestones, plus royalties on net sales.
Roche and Sarepta will equally share global development expenses, according to the terms of the agreement.
DMD – a rare muscle-wasting disease that mostly affects boys and typically is fatal by age 30 – is caused by a defect in the gene coding for dystrophin, a protein found in muscles that is critical for movement.
SRP-9001 is one of a clutch of therapies coming through development that are designed to deliver a gene sequence coding for a truncated ‘micro-dystrophin’ protein, restoring some of the functionality of the mutated protein and avoiding the contraction-induced muscle injury that characterises DMD.
Sarepta is considered to be in the lead in the race to bring this type of therapy to market, particularly after main rival Solid Biosciences had a clinical hold placed on its DMD gene therapy SGT-001 last month because of safety concerns. The hold is still in place, according to an update from the company last week.
Others working on micro-dystrophin gene therapies include Pfizer’s PF-06939926, which was also placed on an FDA hold earlier this year after adverse reactions including kidney injury were seen in one patient. The company has said it intends to press on with phase 3 testing.
Earlier readouts from a clinical trial were deemed to be spectacular, with levels of micro-dystrophin expression well above what Sarepta has said should be sufficient to affect the course of disease progression.
The Swiss pharma group said the new licensing deal “demonstrates Roche’s commitment to gene therapy and its transformational potential for patients.”
Roche has also been trying to develop conventional pharmaceutical therapies for DMD, but suffered a setback last month when it abandoned development of RG6206, a myostatin inhibitor, after disappointing results in clinical trials.