The AI-driven drug repurposing company Healx has teamed up with the global Children’s Tumor Foundation (CTF) to develop new treatments for the genetic disease neurofibromatosis type 1, right after the FDA approved the first-ever drug for the condition.
In the collaboration, the UK biotech will use its AI-driven pharmacology expertise to speed a candidate drug combination towards phase I. In turn, the non-profit CTF organization will use its disease data as well as patient and research networks to help with the clinical testing of the repurposed drugs.
Neurofibromatosis type 1 is a debilitating and disfiguring genetic disorder where patients — often infants — develop deformed bones and sometimes tumors around their nerves. While surgery and chemotherapy is sometimes used to remove the tumors in neurofibromatosis type 1, there hadn’t been any approved treatments for the condition until very recently.
Last week, the FDA approved Koselugo — a drug developed by AstraZeneca and MSD Research Laboratories — for the treatment of inoperable tumors caused by neurofibromatosis type 1 in patients aged two years and over. Specifically, the small molecule drug blocks proteins called mitogen-activated protein kinase kinase enzymes, which help tumors around the nerves to grow.
“We were delighted to hear news of the first drug approved for neurofibromatosis,” said Simone Manso, Healx’ new Head of Neurofibromatosis (NF) Strategic Partnerships. “However, it’s worth noting that this drug has been approved for a subset of patients and for one specific phenotype.”
“Neurofibromatosis is a highly heterogeneous disease and at Healx we are working to find treatments that tackle other disease phenotypes too.”
Manso added that combining two or more drugs together — Healx’s core strength — could make a more effective treatment for the condition than a single drug alone.
Healx has been able to get rare disease treatments to phase I in around 24 months before — a shorter time than traditional drug discovery, which can take several years. However, Manso told me that the current coronavirus pandemic makes it harder to say when they expect to get this program to phase I.
“Many labs and research centers around the world have had to close temporarily due to social distancing regulations,” Manso said.
“This means that we will most likely have to carry out preclinical testing on our predictions at a later date than we would have planned to under normal circumstances.”
Healx has a strong record of working with patient groups to develop therapies for rare diseases. In its most advanced program, the company is planning to enter phase I with a drug combination for the rare developmental disorder fragile X syndrome in collaboration with the US patient group FRAXA Research Foundation.
To fund the development of its AI-driven drug development, Healx raised a €51M Series B round in October 2019. Around €20M of Healx’s cash went to the launch of a program aimed at new patient group collaborations repurposing drugs for rare diseases.
In addition to rare diseases, Healx is one of the latest AI-based drug discovery companies to search for a treatment for coronavirus disease, also known as Covid-19. Others in the race include the UK companies Exscientia and BenevolentAI, and the German company Innoplexus.
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