Back in the year 2000, a major mutation in familial pulmonary arterial hypertension (PAH) was discovered in the receptor for BMP9 — and for years scientists investigated how this mutation causes PAH and whether that information could be used to treat the debilitating disease. This culminated in a manuscript in Nature Medicine in 2015, and served as the foundation for the company Morphogen-IX — which on Tuesday said it had raised £18.4 million ($23.2 million) in a Series B round of financing.
The name Morphogen-IX (pronounced morphogenix) emanates from BMP9, which is a bone morphogenetic protein. The money will be used to support formal preclinical development of the company’s lead candidate — dubbed MGX292 — and the initiation of human studies by 2021, the company said.
MGX292 is a protein engineered variant of the naturally occurring BMP9. The approach was pioneered by the British Heart Foundation-funded laboratory of Professor Nick Morrell at the University of Cambridge, who now serves as the company’s CEO.
“The genetic validation for the use of BMP9 in PAH is overwhelming. We also discovered that mutations in BMP9 itself cause PAH earlier this year,” Morrell told Endpoints News.
PAH is a rare, progressive disease characterized by narrowing and obliteration of small pulmonary arteries, resulting in a severe elevation in arterial pressure and ultimately right heart failure. Mortality rates remain high despite currently licensed therapies that target vasoconstriction. In certain patients, the disease has a genetic cause that reduces the protective function of the BMP9 protein in blood, and some 25% of patients with idiopathic and familial PAH carry mutations in components of the BMP9 signaling pathway, the company estimated.
“The problem is that the disease is not characterised by vasoconstriction — it is the result of profound endothelial dysfunction and structural alterations in the vascular wall. The mortality in PAH remains 50% at 5 years despite combinations of the available drugs. There is thus a huge unmet medical need for drugs that are truly disease modifying that target the molecular and cellular pathways involved in the disease, exemplified by BMP9,” Morrell said.
The biotech, which was spun out of Cambridge Enterprise, raised £1.5 million in a Series A round back in 2015. This latest round of funding was led by Medicxi and included the participation of Cambridge Innovation Capital and Cambridge Enterprise.
“The power of human genetics identifying the centrality of BMP9 in PAH, together with structure-driven engineering to create a protein that can be administered safely, places 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 MGX292, already the subject of patent applications, into the clinic as quickly as possible,” said David Grainger, chairman of the Morphogen-IX board and chief scientific advisor at Medicxi, in a statement.
For Morrell, the transition from scientist to CEO has been interesting. “I am 50% CEO and 50% academic. As company CEO I get to lead science that really matters to patients with PAH, as well as leading on BD and commercial stuff that I find really fascinating (and challenging!)” he said.
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