An experimental medicine that Novartis acquired in a $12 billion buyout has failed a key clinical trial, an outcome at least one analyst believes could sow doubt not only about the technology platform behind the treatment, but Novartis’ dealmaking strategy as well.
Without providing many details, Novartis on Tuesday said the medicine failed to hit the main goal of a late-stage study that evaluated it in people with a muscle-weakening disease known as myotonic dystrophy type 1. The disease — “DM1,” for short — stems from genetic mutations that cause toxic buildup of certain RNA molecules, which then keeps proteins critical for muscle relaxation and organ health from doing their job.
Hallmark symptoms of DM1 include muscles that are locked, fatigued or wasting, as well as cataracts, stomach issues and heart irregularities. According to Novartis, its drug, which is called del-desiran, was not significantly better than a placebo on a test that measures a patient’s ability to relax hand muscles.
The Swiss pharmaceutical giant said researchers did observe “evidence of clinical activity” on secondary goals and in “exploratory analyses.” It now plans to “engage with health authorities to determine the most appropriate development path for del-desiran.”
