The company’s decision is supported by previous preclinical studies that have shown the potential of MGX292, a signaling protein analogue, as a safe and effective therapy for PAH.
“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,” Nick Morrell, CEO of Morphogen-IX, said in a press release.
“This drug has real potential to become a new, disease-modifying therapy for patients suffering from pulmonary arterial hypertension,” he added. “We look forward to the next stage of development leading to clinical trials by 2021.”
Inactivating mutations in the bone morphogenetic protein type II receptor (BMPR-II) are the most common cause of PAH. BMPR-II is a membrane protein that binds to the protein BMP9. The signaling pathway that follows BMP9 binding is important for protecting cells lining the blood vessels of the lungs and preventing cell death.
It is estimated that 25% of patients with idiopathic and familial PAH have mutations in players involved in the BMP9 signaling pathway.
While current PAH therapies focus mainly on reversing pulmonary vasoconstriction of blood vessels, MGX292 is an engineered variant of BMP9 that can potentially restore the protective signaling pathway important for cell integrity and protection from cell death in blood vessels.
The development of MGX292 is the result of a multidisciplinary approach including research in human genetics, structural biology, and protein engineering. Previous in vitro and in vivo studies showed that the agent was safe and has the potential to halt and even reverse PAH.
“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 adviser at Medicxi, which, along with Cambridge Innovation Capital and Cambridge Enterprise, provided financial backing for the founding of Morphogen-IX in 2015.
Morphogen-IX, a biotechnology company based in Cambridge, United Kingdom, is a spin-out from Cambridge Enterprise that specializes in bone morphogenetic proteins for the treatment of PAH.
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