- The first patient has been treated in a two-part Phase 2 clinical trial assessing Acceleron Pharma's (XLRN +0.7%) ACE-083 for the treatment of an inherited nerve disease called Charcot-Marie-Tooth (CMT).
- The first phase will evaluate escalating doses of ACE-083 in 18 CMT patients with muscle weakness in a lower leg muscle called the tibialis anterior (involved in raising the foot at the ankle). The second phase will compare ACE-083 against placebo in 24 patients over a three-month treatment period.
- According to ClinicalTrials.gov, the estimated study completion date is July 2019.
- ACE-083, based on a naturally occurring protein called follistatin, binds to (inhibits) certain proteins that negatively regulate muscle growth (activins and myostatin). The company says untreated muscles or other organs are unaffected thereby reducing the risk of unwanted systemic side effects.
- Now read: 3 Things In Biotech You Should Learn Today: June 28, 2017
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