Nationwide Children’s Hospital spinout Celenex could fetch as much as $452m upon completion of milestones after progressing rare disease therapies conceived at the hospital’s collaborative program with Ohio State University.
Celenex, a US-based gene therapy developer spun out of Nationwide Children’s Hospital, was acquired by rare disease treatment developer Amicus Therapeutics today for an upfront cash consideration of $100m.
The spinout’s investors could receive up to $15m on the fulfilment of development milestones and up to $337m for certain regulatory and sales milestones, though Amicus expects to pay $75m at most over the next four years.
Through the acquisition, Amicus has bought development and commercial rights to ten gene therapy programs started at Center for Gene Therapy, part of research institute OSU Center for Clinical Translational Science (CCTS), itself a collaboration between Nationwide Children’s and Ohio State University.
The programs include Celenex’s lead therapies targeting the CLN6, CLN3, and CLN8 subtypes of Batten disease, a rare and fatal inherited disorder of the central nervous system (CNS) that often presents symptoms from early childhood.
The drugs are injected into the patient’s spinal canal, passing into targeted cells via an adeno-associated virus (AAV) vector previously exploited by other clinical-stage CNS therapies.
Gene therapies often exploit AAA viruses as a delivery mechanism because they are not thought to carry disease and can therefore safely transport genetic material into the body.
Celenex’s underlying thesis is based on the findings of Kathrin Meyer, a principal investigator at Nationwide Children’s Hospital, as well as Brian Kaspar, principal investigator at the Center for Gene Therapy, and Arthur Burghes, professor at Ohio State’s Department of Biological Chemistry and Pharmacology.
Amicus will not pay any royalties to Celenex on the assets and will finance the deal through a $150m debt loan agreed with investment fund BioPharma Credit.
Celenex had raised more than $2m of seed funding, according to the Wall Street Journal, though further details could not be ascertained. Its investors include VC firm Rev1 Ventures, which launched a $22m fund in 2016 with limited partners including Ohio State University.
