Novartis had one eye on the struggles its peers have encountered in the muscular dystrophy space when it decided to acquire Kate Therapeutics, according to the Big Pharma’s CEO.
The Swiss drugmaker signed off on $1.1 billion in a combination of upfront cash and potential milestone payments to buy the gene therapy biotech, it announced today. San Diego-based Kate is focused on preclinical programs aimed at Duchenne muscular dystrophy (DMD), facioscapulohumeral dystrophy (FSHD) and myotonic dystrophy type 1 (DM1).
These programs have been developed using the biotech’s Directed Evolution of AAV Capsid Leveraging In Vivo Expression of Transgene RNA (DELIVER) platform to produce “liver de-targeted muscle-tropic capsids, which transduce both skeletal muscle and cardiac tissue with potency and selectivity in vivo, while avoiding the liver,” according to a Nov. 21 release.
This year has been a stark reminder of what tricky indications muscular dystrophies can be. Pfizer suffered a $230 million financial hit over the summer when its DMD gene therapy flunked a phase 3 trial while Fulcrum Therapeutics’ losmapimod encountered its own late-stage failure just four months after Sanofi bet $80 million on the FSHD candidate.
Explaining the thinking behind the Kate acquisition during his opening presentation at the Meet Novartis Management event in London this morning, CEO Vas Narasimhan, M.D., said the pharma is “very interested” in FSHD, DM1 and the other muscular dystrophies.
Novartis is “really thinking about, how can we learn from the experiences of the first wave of companies that have had failures and perhaps some success with targeting these diseases,” he added.
Kate has “really solved some of the challenges of delivering the right targets or the right genes for patients with FSHD and DM1, and we'll see how we can also broaden that capability to help our own pipeline also advance in the neuromuscular space,” Varasimhan explained.
Novartis—which markets the gene therapy Zolgensma for spinal muscular atrophy—has been working in neuroscience for “many years,” the CEO pointed out in his presentation, namechecking multiple sclerosis, neuromuscular disease and neurodegeneration.
“We are now, I think, at a place where we think our gene therapy capabilities and the capabilities we brought in-house with the various vectors from Regenxbio and other partners will allow us to solve the biology that we did solve with Zolgensma, but then struggled to solve for other monogenic or polygenic neuromuscular diseases,” he added.