Theratechnologies has entered into an agreement with Ionis Pharmaceuticals to license two investigational RNA-targeted medicines, olezarsen and donidalorsen.
Olezarsen is intended for the treatment of familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (sHTG), while donidalorsen targets hereditary angioedema (HAE).
The agreement focuses on the development and commercialisation of these investigational therapies in Canada.
The US Food and Drug Administration (FDA) has accepted the new drug application (NDA) for olezarsen, seeking a priority review for its use in FCS treatment.
The regulator has designated olezarsen as an orphan drug, with a prescription drug user fee act (PDUFA) action date set for 19 December 2024.
Theratechnologies plans to submit the drug candidate for review by Health Canada in 2025, potentially making it the first approved treatment for FCS in Canada.
Additionally, US-based Ionis has completed enrolment in Phase 3 clinical trials of olezarsen in sHTG. In September 2023, the biotechnology firm announced that olezarsen has met the primary endpoint in a Phase 3 FCS patient trial.
The FDA has accepted the NDA for donidalorsen, evaluating it for the treatment of hereditary angioedema (HAE). Regulatory submissions for donidalorsen are also underway in Europe.
The drug received orphan drug designation from the FDA in 2023 and from the European Commission in 2024. Theratechnologies plans to seek donidalorsen’s approval in HAE from Health Canada in 2025.
Last December, Otsuka Pharmaceutical licensed donidalorsen for HAE patients in Europe.
Theratechnologies president and CEO Paul Lévesque said: “The agreement with Ionis is a testament to our team’s capabilities to advance innovation across North America and reaffirms our commitment to be a commercially focused company that delivers sustained top- and bottom-line growth and value for shareholders.”
As per the agreement, Ionis will receive a $10m upfront payment upon the agreement’s execution, along with milestone payments of up to $12.75m based on regulatory, reimbursement, and sales targets.
Ionis will also earn tiered double-digit royalties on annual net sales of both medicines.
Theratechnologies is responsible for filing, obtaining, and maintaining regulatory approval for olezarsen and donidalorsen in Canada.
The American biotechnology firm will manufacture and supply the products to Theratechnologies, with the latter being granted limited rights to manufacture in certain situations.
Ionis CEO Brett Monia said: “Ionis is well-positioned to deliver these medicines to the US market as we rapidly approach our first anticipated independent commercial launches, assuming regulatory approvals of olezarsen in FCS and donidalorsen in HAE.”
