EDINBURGH, U.K.—Pheno Therapeutics, a new drug discovery company spun out from the University of Edinburgh, is investigating novel treatments that may reverse the effects of multiple sclerosis (MS). Backed by a Series A funding round of £5 million for its first three years, Pheno Therapeutics plans to search for new drugs that will help repair damage to the nervous system and significantly improve patients’ debilitating symptoms.
Pheno Therapeutics is supported by Advent Life Sciences, the London-based venture capital firm; the Scottish Investment Bank, with backing from the Scottish Government through the Scottish Growth Scheme; and independent medical research charity LifeArc. Together, they have committed to invest £5 million over three years, subject to Pheno Therapeutics meeting certain milestone conditions.
“I’m delighted to see this company launch with the support of such credible investors,” stated Dr. George Baxter, CEO of Edinburgh Innovations. “Everyone involved is focused on driving the science forward, and we look forward to supporting the team as momentum continues to build, ultimately offering the promise of new treatments.”
Building on original research by Prof. Siddharthan Chandran and Prof. Neil Carragher of the University of Edinburgh, Pheno Therapeutics aims to develop new therapies for MS by identifying novel molecules that cause the body to repair or replace the damaged myelin sheath surrounding nerve cells. This so-called remyelination process has the potential to slow or arrest the progressive disability caused by MS.
“There are no interventions for people with later-stage multiple sclerosis, which is a devastating and debilitating condition. The opportunity for this company is to bring new and repurposed therapeutics to clinical trials and, by doing so, meet an urgent and currently unmet need,” said Chandran.
“It is believed that the neurodegeneration characteristic of multiple sclerosis is a consequence of an age-related decline in remyelination, which may be due to a block in the differentiation of oligodendrocyte precursor cells (OPCs) to oligodendrocytes,” notes Jon Moore, CEO of Pheno Therapeutics. “The majority of Pheno Therapeutics’ current activities are to perform phenotypic screens to discover new leads compounds that promote OPC to oligodendrocytes. This field was opened up by repurposing studies: several existing drugs score as hits in such assays, and some of these have proved to have activity in vivo. However, we expect the ideal agents for the safe and effective modulation of this process are yet to be discovered.”
The key to the company’s potential impact in MS treatments is the University of Edinburgh’s advanced cell-based technology platform that enables the screening of large compound libraries on novel human cellular platforms, in addition to the founders’ and investors’ combination of clinical and drug discovery expertise. Pheno Therapeutics intends to optimize the leads emerging from its cutting edge phenotypic screens via medicinal chemistry to deliver new candidate compounds that will progress first through preclinical tests, and then proof-of-concept clinical trials.
“Edinburgh has a number of excellent labs studying myelination and remyelination and also substantial strengths in phenotypic screening. The screening platform has evolved over many years and marries sophisticated image analysis with transcriptomics and other analytic techniques,” Moore remarks. “We anticipate that this will enable us to rapidly compare and contrast new hits with positive controls, to group compounds of disparate chemical structure into shared biological actions and understand in depth the effect agents have on OPC differentiation. We already have some indication that Edinburgh’s platform can identify agents missed by others.”
MS affects more than 100,000 people in the United Kingdom and 2.5 million worldwide. The disease targets the nervous system, including the brain and spinal cord, and occurs when the body’s immune system attacks the protective layer surrounding nerve cells called the myelin sheath. This slows or disrupts the electrical signals traveling along the nerves. MS causes a wide range of symptoms, including problems with movement, vision, sensation and balance.
Current MS treatments mainly focus on the immune system aspects of the disease, and reduce the severity and frequency of relapses. There is a significant medical need for novel neuroprotective agents that halt the disease progression and prevent long-term disability.
“Multiple sclerosis patients are reasonably well served by a wide range of immunomodulatory agents that enable substantial control of the inflammatory aspects of the disease. But patients can still anticipate a poor long term prognosis and are keen to see the advance of therapies that would prevent their disability worsening,” adds Moore. “The concept of promoting remyelination by influencing oligodendrocyte production is perhaps the most prominent neuroprotective strategy, but there are others.”
Pheno Therapeutics was founded by Chandran, Carragher of Advent Life Sciences and Moore, who is also operating partner at Advent Life Sciences. The formation of Pheno Therapeutics has been supported by Edinburgh Innovations, the University of Edinburgh’s commercialization service, which helped bring together the scientific and clinical expertise to launch the company.
“At the Seed Fund, we look to use our translational expertise to invest in enterprises with a sound scientific concept and the potential to lead to new interventions that address patient needs. In the founders of Pheno Therapeutics and their research to induce myelin repair, we saw an appealing opportunity, particularly given the existing clinical needs in progressive MS,” added Dr. David Holbrook, head of LifeArc’s Seed Fund. “We are delighted to have reached an agreement to support Pheno Therapeutics translate their discoveries.”