New potential treatment for pulmonary arterial hypertension

Cambridge University research has led to the development of spin-out Morphogen-IX, a Cambridge-based biotech that’s working on a novel treatment for pulmonary arterial hypertension (PAH). This severe and life-limiting disease affects the blood vessels in the lungs, leading to heart failure, and leaves sufferers feeling breathless and exhausted.

This potential treatment is based on the culmination of 15 years of work by Professor Nick Morrell and his team at the University of Cambridge’s Department of Medicine at Addenbrooke’s Hospital and could provide the first disease-modifying approach to this serious, life-limiting condition.

PAH affects about 6,500 people in the UK, mostly women in their 30s though it can affect people of any age. It is caused by a narrowing of the blood vessels that lead from the heart to the lungs. As these arteries narrow, it becomes harder for the heart to pump blood through to the lungs, leading to breathlessness and heart failure. Current therapies may alleviate some of the symptoms but there are no medical options that modify the underlying disease process or outcome of the disease. Given the relatively young average age of diagnosis and the high mortality rate associated with PAH, it has a devastating impact on both sufferers and their families. The treatment has the potential to save hundreds of lives each year, and that’s just in the UK. There could also be potential for use of the new drug in other heart and lung diseases, with implications for thousands of patients each year.

We are delighted that our BHF funded research has led to the discovery of a new potential treatment for this rare but important disease. Our new company, Morphogen-IX, is the most efficient vehicle to take this exciting approach forward rapidly into clinical development.

Nick Morrell

Professor Morrell leads the British Heart Foundation (BHF) Centre of Research Excellence at the University of Cambridge and is the Research Director of the National Pulmonary Hypertension Service at Papworth Hospital. The research began in 2000, when genetic mutations were identified in patients – in the gene that codes for the bone morphogenetic protein type 2 receptor, BMPR2. Ten years were then spent finding out how these BMPR2 mutations cause the disease, followed by five years identifying ways to target this fundamental pathway. This complex, highly specialised and challenging research in a rare disease is often best tackled by academic research teams to the point at which a potential new therapeutic agent can be identified. It was at this point that the team needed to step out of the research lab and into the drug development world. To achieve this, and to take development of the drug forward, the spin-out route provided an effective route to the clinic, allowing the small team to remain flexible and make decisions easily.

Morphogen-IX has worked closely with BHF and Cambridge Enterprise, the commercialisation arm of the University of Cambridge, to license the intellectual property arising from Professor Morrell’s research. Morphogen-IX raised £1.5m in seed funding in January 2016, led by Index Ventures (now Medicxi Ventures), along with Cambridge Innovation Capital and Cambridge Enterprise Seed Funds.

This year, the company is confirming the best drug candidate, based on the bone morphogenetic protein that selectively targets BMPR2, to take forward into full clinical development over the following two years.

Professor Morrell said, “We are delighted that our BHF funded research has led to the discovery of a new potential treatment for this rare but important disease. Our new company, Morphogen-IX, is the most efficient vehicle to take this exciting approach forward rapidly into clinical development.”

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