Nido Biosciences Shuts Down Following Phase 2 Trial Disappointment in Kennedy's Disease
Nido Biosciences, a promising neuro-focused biotech startup, has announced its closure after a disappointing phase 2 clinical trial for its lead candidate targeting Kennedy's disease. The company, which emerged in 2023 with substantial financial backing, will wind down operations in early 2026, marking a setback in the pursuit of treatments for rare neurological disorders.
Trial Failure Leads to Company Closure
Nido's lead asset, NIDO-361, failed to show significant efficacy in a global phase 2 trial for Kennedy's disease, also known as spinal and bulbar muscular atrophy (SBMA). The small molecule drug was designed to correct transcriptional dysregulation and restore healthy cell function in patients with this rare inherited neuromuscular disorder.
CEO Jeremy Springhorn, Ph.D., expressed the difficulty of this outcome in a LinkedIn post, particularly noting the dashed hopes for SBMA patients and their families. The closure will affect "just a handful of employees," according to a company spokesperson.
A Promising Start Cut Short
Founded in 2020 as part of 5AM Ventures' 4:59 Initiative, Nido Biosciences made its public debut in 2023 with an impressive $109 million in funding. The company garnered support from pharmaceutical giant Eli Lilly and biotech venture capital firm Bioluminescence Ventures, among others.
At its launch, Nido also highlighted an unnamed program targeting a novel mechanism with potential applications across neurodegenerative and peripheral inflammatory diseases. The company's approach was built on a functional genomics discovery platform using human cell lines to identify new therapeutic targets for conditions such as amyotrophic lateral sclerosis and frontotemporal disorders.
Implications for Rare Disease Research
The closure of Nido Biosciences underscores the challenges faced by biotechnology companies focused on rare diseases. Despite initial promise and substantial financial backing, the failure of NIDO-361 in clinical trials highlights the high-risk nature of drug development, particularly in the field of neurological disorders.
As the company prepares to fully shutter in early 2026, the broader implications for research into Kennedy's disease and related neurological conditions remain to be seen. The setback may prompt a reevaluation of approaches to treating these rare disorders within the pharmaceutical industry.
References
- Neuro biotech Nido closes after phase 2 study fails to move needle in rare disease
After five years, Nido Biosciences has reached the end of the road, closing its doors after its lead neurological disease candidate disappointed in a midstage study.
Explore Further
What were the key reasons behind the failure of NIDO-361 in the phase 2 clinical trial for Kennedy's disease?
What other companies or research teams are currently developing treatments for Kennedy's disease, and what stages are their programs in?
What was the specific mechanism of action for NIDO-361, and how did it compare to other therapeutic approaches in the field of rare neurological disorders?
How might the closure of Nido Biosciences impact future investment in biotech companies focusing on rare disease research?
What lessons can be learned from the NIDO-361 trial failure to improve the development of future therapeutics for neurodegenerative disorders like Kennedy's disease?