Scholar Rock Sees Phase III Path For SMA Drug
Apitegromab Being Studied As Monotherapy And In Combination
While the drug may be well-positioned for registrational development, some important caveats remain after mixed data in Phase II study cohorts.
You may also be interested in...
Phase III study under way may lead to filing of drug meant to address muscular component of the rare disease, with existing therapies such as Spinraza targeting neurological aspects.
While spinal muscular atrophy therapies Evrysdi and Zolgensma have gained ground at the expense of pioneer Spinraza, emerging myostatin inhibitors promise a complementary mechanism that could enhance functional outcomes.
With Nurtec and zavegepant in hand from Bristol, Biohaven tries for another success with a spinal muscular atrophy asset. It will move into epilepsy indications with the buyout of Channel, a subsidiary of Knopp.