Relay Therapeutics Pauses Lirafugratinib Rare Cancer Plans Due to IRA

Pause button/iStock

Pictured: Finger pressing pause button/iStock, cagkansayin

Relay Therapeutics announced plans Thursday to shift gears, pausing its push for rare cancer and switching focus to the larger tumor-agnostic market. The Boston-based biotech is pointing to the Inflation Reduction Act as a driving factor of its decision. 

Relay’s FGFR2 inhibitor has been engaged in a Phase I/II trial since August 2020 for patients with unresectable or metastatic cholangiocarcinoma (CCA). Considered rare, an estimated 8,000 people in the U.S. are diagnosed each year with CCA, or bile duct cancer, with only around 1,000 individuals having the FGFR2-fusion.

Due to the impacts of the Inflation Reduction Act (IRA) signed into law in August 2022, Relay is switching gears to focus lirafugratinib in patients with FGFR2-altered solid tumors, a population for which the biotech estimates a much higher 20,000 a year diagnosed in the U.S.  

Initial clinical data of lirafugratinib in patients across several sub-groups, including FGFR2-fusion tumors and patients with FGFR2-altered HR+/HER2- breast cancer, are showing promise. Relay reported a 35% overall response rate in patients with the former and a 40% ORR in the latter. Of those responders, 63% with FGFR2 fusions had a duration of at least six months as of the data cutoff. In the breast cancer group, all responders had a duration of at least six months.  

With this early evidence in hand, Relay is looking to take lirafugratinib to the clinic for this broader patient population. The biotech will continue enrollments into three tumor agnostic cohorts with additional data and a regulatory update expected in 2024.  

The decision to pause the CCA indication will help extend Relay’s cash runway into the second half of 2026, according to the company. 

Relay’s move is in line with what some experts expected might happen after the IRA was signed into law. BioSpace previously reported Meenakshi Datta, a partner at Chicago-based law firm Sidley Austin, argued that the IRA might also adversely affect orphan drug development. In reading the statute and guidance, Datta contends that the initial date of approval for first indication is what would start the clock counting down toward price negotiations. 

The law is expected by some analysts to discourage companies from targeting a rare population first, with plans to expand indications to broader use at a later point. Datta noted at the time that some companies have already abandoned additional orphan indication programs due to the IRA, as research budgets focus on programs with higher likelihood of success. 

Kate Goodwin is a freelance life science writer based in Des Moines, Iowa. She can be reached at and on LinkedIn.   

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