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STAT+: PepGen says early trial for muscular dystrophy drug looks promising

By · Via STAT Health · Published by Everything Blog

In short

PepGen evaluated its experimental myotonic dystrophy type 1 treatment in an early study involving six patients over 12 weeks. The findings emerge as competing programs experience contrasting fortunes in clinical development. While Dyne Therapeutics nears the completion of a pivotal trial for its candidate, Novartis recently suffered a Phase 3 setback regarding muscle control with a treatment gained from its $12 billion acquisition of Avidity Biosciences.

Key points

  • PepGen generated positive early clinical data by a key measure for its muscle disorder dr…: PepGen generated positive early clinical data by a key measure for its muscle disorder drug candidate.
  • The competitive landscape remains active as Dyne Therapeutics advances toward finishing i…: The competitive landscape remains active as Dyne Therapeutics advances toward finishing its pivotal trial for the condition.
  • Clinical development challenges persist in the space following a recent Phase 3 failure a…: Clinical development challenges persist in the space following a recent Phase 3 failure announced by Novartis.

Sources checked by our analysis pipeline: 1

The biotech PepGen said Tuesday that its experimental drug had shown, by one key measure, the most promising early data yet in the race to treat a muscle disorder that affects over 100,000 people in the U.S. and Europe.

PepGen is a latecomer to the fight to treat myotonic dystrophy type 1 (DM1), a condition that typically robs patients of muscle control and strength beginning around adolescence. Tuesday’s data come from six patients, who were treated for 12 weeks.

Dyne Therapeutics is close to completing a pivotal trial for a DM1 drug. Novartis just announced that a DM1 drug it acquired in its $12 billion buyout of Avidity Biosciences failed to improve a key measure of muscle control in a Phase 3 trial, casting a pall over the field.

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