Cambridge, UK-based Healx has pulled in $47 million in a Series C round and has nabbed IND clearance from the FDA to run its first Phase 2 clinical trial with an oral drug in neurofibromatosis type 1, a rare genetic disorder.
The funding will be used to advance the company’s pipeline of medicines for rare cancers and renal and neurodevelopmental disorders, as well as push the lead program, HLX-1502, through a Phase 2 study, which is expected to start by the end of this year.
Healx, which was co-founded by Tim Guilliams and Viagra co-inventor David Brown, raised $10 million in a Series A round in 2019 and then another $56 million in a Series B that same year. With an AI drug discovery algorithm custom-built for rare diseases, the company seeks to repurpose and optimize existing compounds that have been “clinically de-risked.”
The rare disease biotech is first targeting neurofibromatosis type 1. Patients with the disease have a predisposition to develop tumors, including plexiform neurofibromas and cutaneous neurofibromas. Plexiform tumors currently only have one treatment option available and it’s only approved for children; these tumors are aggressive and can lead to disfigurement and pain. Cutaneous tumors are benign but lead to cosmetic issues and currently have no treatment options.
According to Guilliams, who’s also CEO, HLX-1502 “is a nerve-specific molecule that accumulates in nerve tissue where it modulates metabolism and inhibits mitochondrial function,” adding that the drug “disrupts the energy metabolism of NF1 tumor cells, which are particularly vulnerable due to their altered metabolic state. This leads to reduced tumor cell viability and inhibits tumor growth and Schwann cell proliferation.”
Guilliams added that it’s the only drug in development for the disease that has a different mechanism of action, as other treatments in development are MEK/ERK inhibitors.
The FDA has granted HLX-1502 orphan drug and rare pediatric disease designations for this indication.
Per an investment agreement, the Children’s Tumor Foundation is also supporting the neurofibromatosis type 1 program through milestone-based payments.
“We are also generating important preclinical data for multiple additional compounds identified using our novel generative AI drug discovery engine,” Guilliams wrote in a statement. “Those compounds target rare diseases with significant unmet needs, including additional nerve-related tumor disorders, autosomal dominant polycystic kidney disease and neurodevelopmental disorders such as Angelman syndrome.”
According to Healx’s website, it has other early-stage assets in a rare form of kidney disease, Angelman syndrome, fragile X syndrome and neurofibromatosis type 2.
Silicon Valley-based R42 Group and Atomico co-led the funding round, which included participation from Balderton, Jonathan Milner, Global Brain, btov, Ayana Capital, o2h and VU Venture Partners.