Sarepta Commits To Rapid, Thorough Pivotal Study For DMD Gene Therapy Based On Functional Improvements
The company presented encouraging data on functional assessments of four boys treated with a micro-dystrophin gene therapy at the World Muscle Society, furthering momentum around the program.
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Including upfront payments, half of development costs for SRP-9001, $1.7bn in milestone fees and royalties on sales outside the US, Sarepta pegs deal’s value at $10bn-plus.
Although only interim data from six treated patients, Capricor’s Phase II DMD study shows improvements in upper limb performance, grip strength and respiration. Cardiac performance shows positive trend, but not yet statistical significance.
Another setback for the novel therapy leaves rivals Sarepta and Pfizer in a stronger position in a potentially highly rewarding market.