Morphogen-IX Ltd, a biotechnology company focused on the development of bone morphogenetic proteins (BMPs) for the treatment of pulmonary arterial hypertension (PAH), today announced the nomination of its clinical development candidate and initiation of formal preclinical development. The agent, MGX292, is a protein-engineered variant of BMP9 that has proven highly efficacious and safe in extensive preclinical studies.

Morphogen-IX CEO, Nick Morrell, said, “We are delighted to announce MGX292 as our drug candidate. It is the result of 3 long years of preclinical research and development by our outstanding team. This drug has real potential to become a new, disease-modifying therapy for patients suffering from pulmonary arterial hypertension. We look forward to the next stage of development leading to clinical trials by 2021.”

PAH is a rare but devastating disease in which the blood vessels in the lung narrow and close, raising the blood pressure in the lungs, leading to heart failure and death. In many patients the disease has a genetic cause that reduces the protective function of the native BMP9 protein in the blood. MGX292 is the first agent to enter development that can safely restore that protective function for the blood vessels in the lungs.

November is Pulmonary Hypertension Awareness Month, raising public awareness of the debilitating and eventually fatal condition.

“The combination of powerful human genetic approaches to identify the importance of BMP9 in PAH and structure-driven engineering of the protein to create a form that can be safely administered has put Morphogen-IX in a world-leading position to develop the first agent capable of halting, or even reversing, the progress of this terrible disease. We are working with many of world’s leading experts in PAH to get our proprietary MGX292 into the clinic as quickly as possible” said David Grainger, Chairman of the Morphogen-IX Board and Chief Scientific Advisor at Medicxi, who led the Series A investment in Morphogen-IX Ltd in 2015.

About Morphogen-IX Ltd.Morphogen-IX has developed protein-engineered forms of bone morphogenetic protein 9 (BMP9), conferring potent protection of the blood vessels in the lung, whilst lacking the bone-inducing properties of the native protein. BMP9 has shown strong preclinical efficacy in the reversal of PAH. This approach is based on targeting the central pathway underlying PAH identified in human genetic studies, pioneered in the laboratory of Professor Nick Morrell at the University of Cambridge, who is now CEO at Morphogen-IX, together with co-founders Dr Wei Li and Dr Paul Upton. The company, founded in 2015, is backed by investment from Medicxi, Cambridge Innovation Capital and Cambridge Enterprise, and operates a virtual research and development model from its headquarters on the Babraham Research Campus, south of Cambridge, UK.

About pulmonary arterial hypertension.PAH is a rare disease characterized by narrowing and obliteration of small pulmonary arteries, resulting in a severe elevation of pulmonary artery pressure and ultimately right heart failure. Patients typically present with progressive breathlessness on exertion. The mortality remains high despite currently licensed therapies that target vasoconstriction. Approximately 25% of patients with idiopathic and familial PAH carry mutations in components of the BMP9 signaling pathway.

Source: Morphogen-ix Ltd