May 7 (Reuters) – Entrada Therapeutics’ drug for children with Duchenne muscular dystrophy fell well short of analyst expectations in an early-to-mid stage trial, sending its shares down more than 50% in premarket trading on Thursday.
• The company tested the drug, ENTR-601-44, in ambulatory patients aged six to 17 who have a form of Duchenne caused by a gene mutation that can be treated by skipping exon 44.
• The Boston-based company reported that treated patients showed an increase in dystrophin, a key muscle protein missing in people with Duchenne, of 2.36 percentage points from a baseline of 4%.
• William Blair analyst Myles Minter, in an April client note, had expected a placebo-adjusted dystrophin increase of at least 10% as its base case for the drug, while the firm’s own modelling estimating around 11%.
• The data also pales against del-zota, Avidity Biosciences’ rival exon-44 skipping drug, which reported placebo-adjusted dystrophin increases of around 25% in its early-to-mid stage trial – a benchmark Oppenheimer analysts’ had set as the “bar for success” for Entrada.
• Minter had cited muscle tissue delivery as the “primary risk” for the drug ahead of the readout, as Entrada later reported less of the drug reached the bloodstream in children than expected compared to adults.
• Duchenne is an inherited condition that mostly affects boys and causes muscles to weaken steadily over time.
• Treated patients also showed a statistically significant improvement in how fast they could rise from the floor to a standing position, a widely used test of muscle strength that doctors use to track disease progression and predict when a patient might lose the ability to walk.
• Entrada said it has begun dosing a second group of patients at double the original dose and results are expected by the end of 2026.
(Reporting by Kamal Choudhury in Bengaluru; Editing by Shailesh Kuber)
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