Pfizer acquires Therachon and its dwarfism drug

Pfizer has announced it will pay $340 million upfront to snap up rare disease biotech Therachon, which is developing treatments for a form of dwarfism and short bowel syndrome (SBS).

There’s also an additional $470 million worth of payments on the table contingent on Therachon achieving certain milestones in the development and commercialisation of TA-46, a drug for the treatment of the rare genetic condition achondroplasia, the most common form of short-limbed dwarfism.

There are currently no approved treatments for achondroplasia, which can result in serious cardiovascular, neurological and metabolic complications and affects approximately 250,000 people worldwide – although BioMarin has also caught analysts’ eyes with its own investigational treatment for the disease, vosoritide.

TA-46 is a recombinant human fibroblast growth factor receptor 3 (FGFR3) decoy, a mechanism of action that is believed to normalise the overactive FGFR3 signaling pathways that underline bone development abnormalities associated with achondroplasia.

Therachon is developing the drug as a weekly subcutaneous injection for children and adolescents living with the condition. It has completed Phase 1 and has received Orphan Drug Designation from the EMA and FDA. It has also helped the company pull in $100 million of funding from investors, including Novo Holdings and OrbiMed.

Prior to the closing of the transaction, Therachon will spin-off its development programme for SBS drug apraglutide – a GLP-2 analogue – into a separate, independent company. Pfizer Ventures, the venture capital arm of Pfizer, currently holds a minority stake and will continue to hold an equity stake in the new company.

Therachon CEO Luca Santarelli said: “We are thrilled that the work we have done to discover and advance a novel and potentially life-transforming medicine for achondroplasia is now being continued by Pfizer.

“With its rare disease expertise and worldwide reach, Pfizer is well positioned to accelerate the development of TA-46 and fulfill Therachon’s vision of addressing the complications suffered by children with achondroplasia by targeting the molecular root causes of this condition.”

Pfizer said the drug will complement its existing research programmes for paediatric growth disorders.

The company is under pressure this year after announcing that revenue growth will be flat in 2019 because its blockbuster erectile dysfunction drug Viagra is due to lose US patent protection, along with big-selling painkiller Lyrica.