The FDA has cleared a Phase 2 trial of Lucid-MS, allowing the experimental oral therapy to be tested in people with multiple sclerosis for the first time.
The investigational treatment is designed to protect and repair myelin, the fatty sheath surrounding nerve fibres that is damaged in multiple sclerosis.
An earlier Phase 1 study involving healthy adults found the treatment was safe and well tolerated.
Quantum Biopharma received clearance from the US Food and Drug Administration for its investigational new drug application, allowing the mid-stage clinical study to go ahead.
The application submitted to the regulator ran to approximately 15,000 pages, according to the company.
Andrzej Chruscinski, vice president of scientific and clinical affairs at Quantum, said: “The randomised, double-blind, placebo-controlled Phase 2 trial is intended to evaluate efficacy using clinical and radiological measures relevant to disability progression and disease biology, while also assessing safety and tolerability.”
Lucid-MS, also known as Lucid-21-302, is designed to halt or potentially reverse MS disability progression by protecting myelin.
MS occurs when the immune system mistakenly attacks and damages the myelin sheath.
This causes myelin loss, known as demyelination, and inflammation that gradually damages nerve cells and leads to symptoms.
Approved MS treatments primarily focus on suppressing the acute immune attacks that cause myelin damage. H
owever, they cannot restore myelin that has already been lost and have more limited effects on the chronic inflammation linked to ongoing nerve damage and worsening disability in progressive MS.
Lucid-MS takes a different approach by blocking a group of enzymes that alter myelin proteins in ways believed to make the protective sheath more vulnerable to autoimmune attacks.
By interfering with this process, the treatment is intended to prevent further demyelination and promote myelin repair, potentially slowing disease progression and restoring some lost function.
The Phase 2 study will be the first time the therapy has been given to people with MS.
Trial preparations, including the selection of study sites, are under way, with enrolment and dosing expected to begin as soon as possible.
Quantum is working with Allucent, a global clinical research organisation with experience conducting studies in neurodegenerative diseases.
Preclinical studies in mouse models of MS found that Lucid-MS could prevent myelin loss and promote its repair.
Researchers also reported reduced symptom severity and improved motor function to levels comparable with healthy controls.
In Phase 1 testing involving healthy adults, the treatment was found to be safe and well tolerated.
The forthcoming Phase 2 trial will assess its efficacy using clinical and radiological measures related to disability progression and disease biology, as well as its safety and tolerability.
Zeeshan Saeed, founder and CEO of QuantumSaeed said: “Lucid-MS represents a unique patented first-in-class, disease-modifying therapeutic opportunity in multiple sclerosis by targeting demyelination, particularly in progressive forms of the disease where unmet medical needs remain today.”
