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 programme 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.
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