Novartis suffers third drug trial setback in a week as del-desiran fails

Novartis suffered its third clinical-trial setback in a week after del-desiran, an experimental treatment for the rare muscle-wasting disorder myotonic dystrophy type 1 (DM1), failed to meet the main goal of a late-stage study.

The setback adds to pressure on the Swiss drugmaker as it looks for new sources of growth ahead of what is expected to be its biggest patent cliff.

Del-desiran was the centrepiece of Novartis’ $12 billion acquisition of Avidity Biosciences and had been viewed as a potential blockbuster. Chief Executive Officer Vas Narasimhan told Bloomberg TV in July that the drug had “$5 billion plus peak sales potential”, adding that a successful trial would make it the first drug to demonstrate efficacy in DM1.

However, patients treated with del-desiran did not show a statistically significant improvement in how quickly they could open their hands compared with those given a placebo, Novartis said on Tuesday, September 8.

The company said it will analyse the full trial data and consult regulators before deciding whether, and how, to continue developing the drug.

Analyst sees setback for Avidity pipeline

The failed trial has also raised questions over the broader pipeline Novartis acquired through Avidity.

The result is a “major pipeline setback” that “has not helped our confidence” in the other therapies obtained through the acquisition, Vontobel analyst Stefan Schneider told Bloomberg, while maintaining a hold rating on Novartis shares.

Del-desiran is designed to target the genetic cause of DM1, an inherited disorder that causes progressive muscle weakness and stiffness.

Third setback in a week

The failed DM1 trial comes after two other major developments for Novartis in the past week.

The company halted trials of an experimental cell therapy for autoimmune diseases after three patients died. Its heart drug pelacarsen also failed in a closely watched study.

Novartis did receive positive results from a separate trial of an experimental multiple sclerosis treatment during the week, providing some relief amid the broader string of setbacks.

Novartis shares fell as much as 5.1% on Tradegate early Tuesday, compared with their close in Zurich.

Also read: Novartis suffers third drug trial setback in a week as del-desiran fails