JPM24: Novartis 'pretty aggressive and active' in neuroscience BD, gene editing not so much, research chief says

Novartis’ R&D chief Fiona Marshall, Ph.D., says the company has been “pretty aggressive and active” in neuroscience-focused business development, looking for a boost to one of the company’s four key therapeutic pillars.

The president of biomedical research delved a bit deeper into how the company weighs internal research with external opportunities in an interview with Fierce Biotech Monday on the sidelines of the J.P. Morgan Healthcare Conference in San Francisco.

Within neuroscience, Novartis’ priority is on multiple sclerosis and neurodegeneration, not as much in neuropsychiatric, Marshall explained. The latter category of neuroscience has been front and center following Big Pharma M&A deals for schizophrenia biotechs Cerevel and Karuna.

“I think the harder challenge is to get the differentiation over standard of care,” Marshall said. 

headshot of Novartis NIBR head Fiona Marshall
Novartis NIBR chief Fiona Marshall (Novartis)

Marshall also spoke about the opportunities and hurdles that remain for the psychedelic field after Lykos Therapeutics submitted its new drug application in December for an MDMA-assisted therapy to treat PTSD. Marshall said an outstanding challenge is patient stratification, saying the field doesn’t quite know yet how to select the best patients for psychedelic or psychoactive treatments. 

Novartis’ existing neuroscience pipeline is buoyed by spinal muscular atrophy treatment Zolgensma and relapsed multiple sclerosis med Kesimpta. The former works by replacing the SMN1 gene that’s dysfunctional in patients with a new and improved version. But Marshall said Novartis is not keen on widening its gene therapy unit to include gene editing anytime soon, specifically as questions remain around the delivery of editors. 

“I wouldn't say it's at the top of our deal list at this conference, put it that way,” she said. 

Instead, Marshall says Novartis' internal research is centered on the expanded use of RNA, including siRNA and antisense oligonucleotides, and the delivery of those therapies outside of the liver. 

Marshall’s comments come as Novartis sprinted out of the gates of this year’s major investor conference. The Swiss pharma announced the acquisition of autoimmune biotech Calypso for $250 million with $170 million in biobucks available. The smaller startup spun out of Merck KGaA in 2013. Novartis is also paying $185 million upfront for ex-China rights to two RNAi cardiovascular candidates from Shanghai Argo Biopharmaceutical. 

The Wall Street Journal reported Monday that Novartis was also in late-stage talks to buy Cytokinetics after the heart disease biotech showed in late December that its late-stage cardiovascular drug improved patients’ exercise capacity in a phase 3 trial. Marshall would not comment on the potential deal.