Tag Archives: neuroscience

Myrobalan wins grant to advance MS remyelination candidate MRO-002

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multiplesclerosisnewstoday.com/news-posts/2025/03/18/myrobalan-wins-grant-advance-ms-remyelination-candidate-mro-002/

March 18, 2025

Myrobalan Therapeutics has been awarded a grant of more than $850,000 from the National Multiple Sclerosis Society to advance its new oral candidate MRO-002 for treating progressive forms of multiple sclerosis (MS).

The funding was made through the society’s Fast Forward program, which seeks to bridge the gap between preclinical and clinical development for new therapies and diagnostic tools that address disease progression and progressive forms of MS.

Myrobalan‘s experimental therapy MRO-002 is a GPR17 antagonist, or inhibitor, that’s designed to promote myelin repair in people with MS and other demyelinating conditions. With the grant, the company will conduct additional preclinical experiments to determine if it may have therapeutic benefits in progressive MS.

“We are excited to receive the Fast Forward grant from the National MS Society and appreciate their confidence in our drug development approach,” Jing Wang, PhD, CEO and co-founder of Myrobalan, said in a company press release. “We believe that MRO-002 has the potential to promote myelin repair in neurodegenerative diseases, demyelinating disorders, and conditions involving recovery from [central nervous system] injury.”

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March 10, 2025 News by Marisa Wexler, MS

Slowly expanding MS lesions linked to myelin damage in brain

Finding ways to aid myelin repair

In MS, the immune system mistakenly attacks the myelin sheath, a fatty coating around nerve cells. Like an insulator on electrical wires, myelin protects nerve fibers and ensures the efficient transmission of nerve signals. When it’s lost, patients experience a range of symptoms related to disrupted nerve signaling.

Myelin is produced by oligodendrocytes, specialized cells in the brain and spinal cord. While existing oligodendrocytes have some ability to repair damaged myelin or restore lost myelin, the remyelination process isn’t very efficient in MS due to ongoing inflammation. There’s also insufficient maturation of oligodendrocyte precursor cells (OPCs) into fully functional oligodendrocytes that are able to produce myelin.

A key challenge in MS treatment is the repair of damaged myelin. While available disease-modifying therapies help reduce relapses and slow disease progression, their ability to repair myelin and reverse disability is limited.

Finding therapies that can improve remyelination has been a longstanding goal in MS research. One promising approach involves targeting GPR17, a receptor in OPCs that prevents the cells from maturing into fully functional oligodendrocytes, thereby limiting myelin repair.

MRO-002 is a highly selective GPR17 antagonist that can cross the blood-brain barrier, the protective membrane that restricts the passage of most substances from the bloodstream into the brain. This ability lets it reach the brain and remove this brake from OPCs, causing them to mature and boosting the production of new myelin.

Preclinical studies on human-derived oligodendrocytes and animal models of demyelination have shown encouraging results. MRO-002 significantly boosted the maturation of oligodendrocytes in both models, resulting in enhanced remyelination in the brains of the animals.

With support from the National MS Society, the company hopes to conduct additional studies to support MRO-002’s development toward clinical trials.

“Having a therapy that promotes myelin repair would have tremendous implications for people living with MS,” said Walter Kostich, PhD, associate vice president of translational research at the National MS Society. “We are happy to support Myrobalan Therapeutics’ preclinical testing of MRO-002 and look forward to learning whether it has translational potential for people with MS.”

First published by: multiplesclerosisnewstoday.com/news-posts/2025/03/18/myrobalan-wins-grant-advance-ms-remyelination-candidate-mro-002/

March 18, 2025

REMYELIUNATION THERAPY TRIAL

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First clinical trial of remyelination therapy PTD802 cleared in the UK

Pheno Therapeutics’ candidate is inhibitor of GPR17 receptor

The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has cleared Pheno Therapeutics to initiate a first-in-human Phase 1 clinical trial to test its oral candidate PTD802 in healthy volunteers.

The therapy is a selective small molecule antagonist, or inhibitor, of the GPR17 receptor and is designed to restore the damaged and lost myelin — a process known as remyelination — in people with multiple sclerosis (MS) and potentially other demyelinating conditions.

“We are delighted to have received approval from the MHRA to progress our PTD802 program to a Phase 1 trial, a major milestone, marking our transition to a clinical stage organization. As the first company to carry out dosing of a selective GPR17 antagonist in healthy humans, we are leading the way in the race to develop GPR17-targeting remyelination therapeutics,” Fraser Murray, PhD, Pheno Therapeutics’ CEO, said in a company press release. “With this first-in-human program we are moving closer to our goal of delivering transformational drugs for the treatment of neurological diseases associated with demyelination.”

MS is caused by the immune system erroneously targeting myelin, a protective layer that surrounds nerve fibers and is important for nerve signals to be efficiently transmitted. As myelin is lost, nerve cells also become damaged and patients experience a range of symptoms related to impaired nerve signaling.

An ‘urgent need’ for remyelination therapies

The search for therapies that promote myelin repair has been a key focus in MS research for many years. There are a number of treatments that reduce inflammation and prevent or slow further damage, but none can reverse the damage that’s already occurred.

“Current treatments for MS focus mainly on the immune aspects of the disease, reducing severity and frequency of relapses. There is an urgent and unmet need for effective therapeutics that limit disability progression in MS, with remyelination offering a promising neuroprotective treatment,” said Siddharthan Chandran, MD, PhD, co-founder of Pheno Therapeutics.

The GPR17 receptor works like a natural brake that slows the development of oligodendrocytes, the cells that produce myelin in the brain and spinal cord. PTD802 is designed to boost remyelination by releasing this brake. Pheno believes it may be used with existing MS therapies to better control disease progression and possibly help patients regain some lost function.

“Whilst GPR17 antagonists have potential utility beyond MS, PTD802 is a hugely promising first-in-class oral remyelination agent, which we believe will be the next step in devising combinatorial approaches to preventing MS progression,” Chandran said.

by Patricia Inacio, PhD | January 21, 2025

Reposted from Multiple Sclerosis News Today