Axoltis teaming with InSilicoTrials on MS candidate NX210c

Effort will explore effects on brain health, biomarker levels, safety

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by Steve Bryson, PhD |

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NX210c, an investigational therapy for multiple sclerosis (MS) and other neurodegenerative diseases from Axoltis Pharma, will have its clinical development boosted as part of a collaboration with InSilicoTrials.

The companies will use InSilicoTrials’ simulation platform to replicate the brain and spinal cord characteristics of people with neurological disorders such as MS, Parkinson’s disease, and amyotrophic lateral sclerosis (ALS) to assess NX210c’s effectiveness in virtual patients with the conditions.

“This partnership represents an exciting synergy between cutting-edge simulation technologies and innovative biotechnological advancements,” Luca Emili, CEO of InSilicoTrials, said in a company press release. “Together, we aim to unlock new insights and develop groundbreaking treatments that will positively impact the lives of patients with neurologic impairments worldwide.”

InSilicoTrials’ platform uses both artificial intelligence (AI) and computer modeling tools to accelerate potential therapies’ development more efficiently by minimizing the costs associated with traditional clinical trials, which can be time-consuming and expensive.

Leveraging preclinical and Phase 1b study data, the collaboration will investigate multiple NX210c dosing regimens and their effects on brain health, biomarker levels, and safety.

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“We are very delighted to initiate this collaboration with InSilicoTrials and have the opportunity to optimize and accelerate our next clinical steps,” Yann Godfrin, PhD, Axoltis’ CEO, said. “Integrating their industry-leading AI and computational modeling solutions in the development plan of our promising disease-modifying drug will allow us to address the pressing challenges faced by individuals suffering from neurological disorders.”

Neurodegenerative diseases often share features such as leakage of the blood-brain-barrier, a highly selective and protective membrane that controls which substances from the blood can reach the brain and spinal cord. Other common characteristics include impaired communication between nerve cells and neuronal death.

A feature of MS is an abnormal immune attack on the brain and spinal cord, triggering a wide range of symptoms such as fatigue, loss of balance, numbness and tingling, and muscle spasms or stiffness. The entry of the overactive immune cells into the brain and spinal cord through the blood-brain barrier contributes to inflammation and nerve cell damage in MS.

NX210c is a lab-made small protein that corresponds to the active part of SCO-spondin, a protein that plays key roles in neuronal survival, maturation, and growth. According to Axoltis, NX210c improves blood-brain-barrier integrity, boosts nerve cell communication, and prevents cell death.

Multiple increasing doses of the candidate therapy are being tested in healthy people, ages 55 and older, in a Phase 1 study (NCT05827653). The trial’s second half is expected to enroll people with Alzheimer’s disease. Single ascending doses of a peptide related to NX210c (NX210) were well tolerated in 39 healthy people in a previous Phase 1 trial.

The therapy was granted orphan drug status in the U.S. for ALS and in Europe for spinal cord injury. The designation should help expedite NX210c’s development and streamline the regulatory review process.

“We are thrilled to collaborate with Axoltis in our shared mission to advance therapeutic solutions for neurological disorders,” Emili said.