Novo powers rare disease startup Therachon to $60M round

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Therachon has raised $60 million (€52 million) to take a phase 1 rare disease drug deeper into the clinic. The Novo Holdings-led mezzanine round comes six months after Therachon began testing its FGFR3 ligand trap in healthy volunteers.

Swiss biotech Therachon thinks the candidate, TA-46, may restore normal bone growth in people with achondroplasia, a form of short-limbed dwarfism. The idea is built on evidence that most cases of the condition stem from a mutation in the gene that encodes for FGFR3, a protein involved in the regulation of bone formation. The mutation causes excessive signaling that suppresses bone growth.

Therachon’s plan is to prevent the excessive signaling through the administration of a soluble human form of FGFR3. The protein will serve as a decoy receptor that stops FGF from binding to the mutant form of FGFR3.

Preclinical studies suggest the approach has potential, emboldening Therachon to start testing the drug in healthy volunteers in February. Therachon followed up with a natural history study designed to gather data to inform endpoint selection and other aspects of the clinical development program. The next step is to trial weekly subcutaneous injections of TA-46 in children with achondroplasia.

The financing tees Therachon up to start its first trial in patients next year. New investors Cowen Healthcare Investments, Pfizer Ventures and Tekla Capital Management joined lead financier Novo in the round. Existing backers Versant Ventures, OrbiMed, Bpifrance and Inserm Transfert contributed, too.

Raising the money secures the near-term financial future of TA-46 while also providing Therachon with the means to expand its activities. 

“With this financing, we will be able to devote more resources to our lead clinical program in achondroplasia. Additionally, we look forward to exploring additional pipeline opportunities in the rare disease space,” Therachon CEO Luca Santarelli, M.D., said in a statement.