10126469Development of a Novel Treatment Option for Hypertrophic CardiomyopathyClosedCollaborative R&DInnovate UKSignaCor Therapeutics are in the advanced stages of research into the repurposing of an old chemotherapy drug for treatment of heart disease. Specifically, this drug has potential to offer improved clinical outcomes for patients with a form of heart disease called hypertrophic cardiomyopathy (HCM). The prevalence of HCM in the general population depends on whether a patient has subclinical or overt disease. While the prevalence of asymptomatic HCM ranges between 1 in 200 and 1 in 500 people, symptoms are noted in fewer than 1 in 3000 people. Currently, there are limited treatment options for patients with severe (symptomatic) HCM and these treatments only relieve symptoms, they do not target the underlying cause. The drug being researched at SignaCor Therapeutics is a first-in-class treatment that can reverse damage to the heart as well as preventing further deterioration of heart function. From the research conducted to date with various animal models, there is sufficient evidence to proceed with bringing this drug into a clinical trial in human patients with HCM. Over the next 12 months, SignaCor Therapeutics will be conducting a phase 2a clinical trial to determine the safety and tolerance of this drug in humans and select the maximum tolerated dose of treatment. With Innovate UK funding additional data will be collected on the efficacy of the drug at reducing damage to the heart. This will be important for moving onto the next clinical trial phase and reducing time-to-market for a much-needed new treatment option for HCM. Additional research and development will also be performed as part of this Innovate-UK funded project to reformulate the drug from a subcutaneous injection to an oral capsule. At the end of this 12-month project, a novel treatment option for HCM will be closer to the clinic and the company will be in the advanced stages of producing an oral form of the drug, which would further minimise the burden of disease and treatment on patients.