Marvel planning trial to test fragile X treatment in healthy volunteers
New funding, guidance help advance MB-204 to human study
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Marvel Biosciences plans to launch an early clinical trial in the coming months to test MB-204, its lead therapy candidate for fragile X syndrome and other neurological conditions, in healthy volunteers.
The Phase 1 trial will be possible due to funding and expert guidance from 5 Horizons Ventures, a U.S.-based life sciences firm that provides capital and strategic assistance to advance potentially innovative therapies.
“By securing participation in our clinical development costs from a group deeply embedded within the global CRO [contract research organization] ecosystem, we are not only strengthening our capital efficiency but also aligning with partners who bring strategic insight into clinical execution and financing pathways,” Rod Matheson, Marvel’s CEO, said in a company press release. CROs are companies that assist in clinical trials and related services for pharmaceutical drug development.
Marvel, which is based in Calgary, Canada, also recently announced that it will run the Phase 1 study in collaboration with Australia-based Novotech as its CRO.
“Having previously worked with Novotech on multiple clinical programs, I am pleased to be working with Novotech again,” Mark Williams, PhD, Marvel’s chief scientific officer, said in a separate company press release. “Australia is an attractive place to conduct first-in-man studies owing to [its] favourable regulatory environment and tax credits.”
Under the companies’ agreement, 5 Horizons will cover about 15% of the CRO costs for the upcoming trial. 5 Horizons will also help evaluate intellectual property, checking that MB-204 is unique and protectable, and support partnerships that can move the treatment forward faster.
“We’ve been conducting due diligence with the Marvel team over the past year, and we’re excited to provide an investment for the Phase 1 trial of MB-204” said Aaron Ray, managing partner of 5 Horizons. “We believe the combination of the Marvel team, pre-clinical data, and multiple indications in both rare disease and the central nervous system positions the company for success.” The central nervous system is comprised of the brain and spinal cord, and plays a central role in fragile X.
The agreement builds on the momentum of recent funding from the National Research Council of Canada Industrial Research Assistance Program and Alberta Innovates CarE. Additionally, the company’s intellectual property has been bolstered by newly granted patents in major markets, including the U.S. and Japan, according to Marvel.
MB-204 is a version of a Parkinson’s treatment
Fragile X is caused by mutations in the FMR1 gene, which provides instructions for producing a protein that regulates proteins involved in how nerve cells communicate. FMR1 mutations disrupt how well the brain develops and are a leading genetic cause of autism, a neurodevelopmental condition that may affect communication and social interaction.
MB-204, being developed by Marvel’s wholly owned subsidiary Marvel Biotechnology, is a modified version of istradefylline, a medication approved in the U.S. under the brand name Nourianz for Parkinson’s disease.
It works by blocking the activity of adenosine A2a receptor proteins, which normally mediate signals from adenosine, a chemical in the brain. Abnormal adenosine signaling has been implicated in a number of neurological and neurodevelopmental conditions.
“According to internal and third-party analyses, MB-204 has the potential to address significant unmet needs in rare neurodevelopmental disorders, where current treatment options remain limited and often burdened by tolerability challenges,” the release stated.
The rare neurodevelopmental disorders that Marvel is developing MB-204 for include fragile X and Rett syndrome, a disease marked by developmental regression and difficulties communicating.
In preclinical studies, MB-204 lessened social and behavioral deficits in a mouse model of Rett. Data on the therapy’s preclinical efficacy in fragile X models are expected by year’s end, per the company.
Marvel has said it’s pursuing orphan drug designation for MB-204 from the U.S. Food and Drug Administration for both indications. That status is meant to accelerate the clinical development and regulatory review of treatments for rare diseases in the U.S.
The upcoming Phase 1 trial will evaluate MB-204’s safety, tolerability, and pharmacokinetics, or its movement into, through, and out of the body. If the results are positive, Marvel said it plans to advance to Phase 2 and 3 trials in people with fragile X and Rett syndrome.
