Morphogen-IX Raises $23.2 Million to Develop PAH Therapy Candidate MGX292

Biotechnology company Morphogen-IX has raised $23.2 million to advance development of its lead drug candidate, MGX292, for the treatment of pulmonary arterial hypertension (PAH).

The investment, led by Medicxi with contributions from Cambridge Enterprise and Cambridge Innovation Capital, will support preclinical development and initiation of clinical trials, planned for 2021. Morphogen-IX, based at the University of Cambridge, United Kingdom, is a spin-out from Cambridge Enterprise that specializes in bone morphogenetic proteins for the treatment of PAH.

MGX292 is a protein-engineered variant of bone morphogenetic protein 9 (BMP9), which has demonstrated efficacy and safety in preventing and reverting PAH in preclinical models of the disease, Morphogen-IX said.

Normally, BMP9 circulates in the blood and works to maintain the integrity of cells that line blood vessels in the lungs, known as endothelial cells. But in certain conditions, such as idiopathic and heritable PAH, the levels of BMP9 are insufficient to protect the lung endothelium.

An estimated 25 percent of PAH patients carry genetic mutations in components of the BMP9 signaling pathway, reducing its protective function.

The idea of targeting the BMP9 pathway to treat PAH came from the the initial work of Nicholas Morrell, MD, PhD, and his team at the University of Cambridge, with funding from the British Heart Foundation.

Morrell is now chief executive officer at Morphogen-IX, together with co-founders Wei Li and Paul Upton.

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“This major investment, following closely on the nomination of MGX292 as our drug candidate, will support the critical next steps of preclinical development and take us all the way through to completion of Phase 2 studies over the next [three to four years]. MGX292 has major disease-modifying capability that is badly needed for patients suffering from pulmonary arterial hypertension,” Morrell said in a press release.

David Grainger, chairman of Morphogen-IX’s board and chief scientific adviser at Medicxi, said:  “Morphogen-IX [is] 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.”