- Nido Biosciences, a startup focused on developing drugs for brain diseases, emerged from stealth Monday having raised $109 million in funding to date.
- The company already has one drug candidate for spinal and bulbar muscular atrophy, or SBMA, in early-stage clinical testing. Also known as Kennedy’s disease, the condition is rare, genetic and characterized by muscle weakness and atrophy.
- Started in 2018 through 5AM Ventures’ incubator, Nido is backed by investors that include Eli Lilly, Bioluminescence Ventures and Bessemer Venture Partners. It has been operating in stealth, completing seed and Series A financing, and starting a Series B round.
When markets are tight, as they have been for biotech over the past year, investors look for safer bets.
Private biotechnology companies with more advanced drug programs, rather than early and unproven platforms, may have an edge. Industry veterans interviewed by BioPharma Dive have suggested that startups with drugs approaching or in clinical testing are more likely to successfully raise funding in the current climate.
For instance, companies that recently raised large sums via initial public offerings, such as Acelyrin and Structure Therapeutics, benefited from having drugs in clinical studies.
Nido, with a drug in early testing and a syndicate of investors that have now supported multiple financing rounds, appears to fit that profile.
The research behind Nido’s lead program comes from J. Paul Taylor, a Howard Hughes Medical Institute investigator and St. Jude Children's Research Hospital researcher who co-founded the company.
Nido’s drug, dubbed NIDO-361, takes what CEO Jeremy Springhorn calls a “corrector” approach to the genetic dysfunction behind SBMA, also known as Kennedy’s disease. Patients with SBMA have a mutation in a gene that contains blueprints for making a protein that binds to androgen, a hormone involved in male sexual development. The mutated protein affects motor neurons in the spinal cord that control muscles, eventually leading to the cells’ death.
Fewer than 1 in 150,000 men have the disease, which is linked to the X chromosome, according to the National Institutes of Health. SBMA progresses slowly, and Nido believes its drug can help before a patient loses their muscle movement.
“The problem from a drug development perspective is that the neurodegeneration happens so quickly, the best you can hope for with therapeutic intervention is stable disease,” Springhorn said. “We're hoping that we'll do better than that simply because the neurons haven't died yet.”
Nido aims to finish its Phase 1 study of the drug this year, he said, and then advance it into studies that can show whether treatment can slow, or reverse, disease progression.
Springhorn joined Nido, which is headquartered in Watertown, Massachusetts, in early 2021. Previously, he was an executive at Syros Pharmaceuticals, worked at Flagship Pioneering and spent more than a decade at Alexion Pharmaceuticals.
The company was founded through 5AM Ventures’ in-house company creation program, the 4:59 Initiative. Nido started, but hasn’t closed, fundraising for its Series B round, Springhorn said, which is being led by Bioluminescence Ventures.
Nido expects to begin narrowing down compounds to identify a second drug candidate this year. The company’s pipeline includes research programs for neurodegenerative and inflammatory conditions, though it has not disclosed specific disease targets.