An RNA drug Novartis acquired as part of its $12 billion bet on Avidity Biosciences succeeded in a trial of patients with a rare neuromuscular disease, Novartis said Thursday.
Called delpacibart braxlosiran, the experimental drug met its primary biomarker endpoint in a Phase 1/2 study evaluating the treatment in a muscle-wasting disease known as facioscapulohumeral muscular dystrophy, or FSHD. The drug, a so-called "antisense oligonucleotide conjugate" or AOC, is designed to restore muscle function and help slow progression of the disease.
Del-brax targets a gene called DUX4, which is expressed incorrectly in patients with FSHD.
A subgroup of FSHD patients receiving del-brax showed lowered biomarker levels in two measures that indicate "strong target engagement and reduction in muscle damage,” according to the company. The 90-patient trial had three cohorts that evaluated two different doses of the drug versus placebo, as well as how del-brax affected levels of the biomarker KHDC1L, which is regulated by DUX4, and creatine kinase, a biomarker of muscle damage.
The study results build on data previously revealed by Avidity, indicating del-brax increased mobility and strength among people with FSHD.
“We are now evaluating the totality of the biomarker and clinical data and look forward to discussions with global regulatory agencies as we work with urgency to advance the development of del-brax for patients in need,” Nazem Atassi, Novartis’ global head of neuroscience and gene therapy development, said in a statement.
The Swiss pharma is now enrolling patients in a Phase 3 trial which will measure muscle strength and their progression on a 10-meter walk and run test.
The del-brax data “at least partially validate[s] Novartis’ decision to acquire Avidity,” Jefferies analyst Michael Leuchten wrote in a note to clients. The trial data could also lead to a path for accelerated approval.
Still, investors and analysts are keeping an eye on two other AOC drugs Novartis picked up as part of the acquisition. Study data from Novartis’ trial of del-desiran, an experimental treatment for a form of myotonic dystrophy, is expected in the second half of this year. Meanwhile, its other AOC candidate, del-zota, is in mid-stage development for Duchenne muscular dystrophy.
Shares in Novartis ticked up a little under 3% on Thursday morning.
