Nido Biosciences emerges from stealth mode announcing phase 1 clinical trial to treat a rare and debilitating neuromuscular disease with technology originating from the University of British Columbia and Simon Fraser University.
Nido Biosciences is a licensee of UBC-SFU that is advancing a compound, NIDO-361, to treat Spinal and Bulbar Muscular Atrophy (SBMA) with initial chemical matter that was invented by teams led by Dr. Artem Cherkasov and Dr. Bob Young. Dr. Cherkasov is a professor in the UBC Department of Urologic Sciences and a senior research scientist at Vancouver Prostate Centre, which is hosted by the Vancouver Coastal Health Research Institute and UBC. Dr. Young is a professor of Chemistry at Simon Fraser University.
SBMA, also known as Kennedy’s disease, is a rare inherited neuromuscular disorder that results in the loss of skeletal muscle and motor neuron function. It is caused by a genetic mutation of the androgen receptor and manifests in men, causing progressive weakness and wasting of limb, facial and esophageal muscles, which results in impaired mobility, speech, and swallowing. The initial tool compound was invented by Cherkasov and Young’s teams and is a small molecule that binds to the androgen receptor site in such a way that it can restore healthy cell function. Nido Bio’s current phase 1 clinical trial of NIDO-361 is an important step towards enabling an improved quality of life for those impacted by this disease, which currently has no meaningful treatment. Results from this trial are anticipated later this year.
The tool compound was invented at UBC in 2008 as a potential prostate cancer treatment. The potential for more advanced analogues for treating SBMA was identified in 2018 by Dr. Paul Taylor at St. Jude Children’s Research Hospital, who used an early version of the molecule in a mouse model for SBMA. Nido Bio was formed through the 4:59 Initiative at 5AM Ventures (a notable venture capital firm) in 2018 on the premise that breakthroughs in human genetics and the understanding of neurological diseases could be leveraged to develop novel and transformative precision medicines. Nido Bio took an interest in the work and funded sponsored research in the program. In 2019, they licensed the technology in a deal negotiated by UBC’s University-Industry Liaison Office.
Since this time and prior to their announcement on May 15, 2023, Nido Biosciences has been operating in “stealth mode.” While in stealth, it has engaged in sponsored research with UBC researchers, led by Dr. Nathan Lack at the Vancouver Prostate Centre, to further bioanalytical assays in support of NIDO-361’s advancement into preclinical studies. The company, based in the Boston area, also announced that they have raised a combined $109 million (USD) during this period through seed, Series A and B financings.
“This opportunity is a great example of collaborative efforts across the Vancouver life science research environment. Drug design was by UBC’s Cherkasov lab and assay design at UBC’s Lack lab, both at Vancouver Prostate Centre at Vancouver General Hospital, and expert medicinal chemistry was provided by the Young lab at SFU,” said Brad Wheeler, the technology transfer manager at the UBC UILO who led the licensing negotiation. “UBC’s UILO has negotiated the licensing deals for the approximately 250 spin-off companies based on UBC research, and Nido Bio certainly stands out in terms of the amount of early investor support it has received.”