The U.S. Food and Drug Administration has given its approval to Nuvation Bio’s new oral medication Ibtrozi for treating metastatic non-small cell lung cancer patients whose tumors test positive for ROS1 alterations, a genetic marker found in approximately 2% of cases.
Clinical trials demonstrated impressive efficacy, with response rates of 90% and 85% in treatment-naive patients across two separate studies. For those previously treated with tyrosine kinase inhibitors, the drug showed response rates of 52% and 62%. The duration of response reached up to 47 months in one trial and 30 months in the other.
The approval comes with certain caveats, as the FDA’s prescribing information highlights potential risks including cardiac rhythm abnormalities, liver complications, and pulmonary inflammation. According to Nuvation, 7% of trial participants discontinued treatment due to adverse effects. Following the announcement, the company’s stock value declined by 10%.
Nuvation Bio, led by former Medivation CEO David Hung, emerges from a challenging period marked by multiple setbacks. The company abandoned its initial drug program in 2022 and faced disappointment with a second candidate two years later. Like many biotech firms, Nuvation has weathered significant market challenges, with its share price dropping more than 75% since 2020.
The company’s fortunes changed following its acquisition of AnHeart Therapeutics, which brought taletrectinib (now Ibtrozi) into its portfolio. The drug has already secured approval in China before its U.S. clearance.
Ibtrozi enters a competitive landscape dominated by established pharmaceutical giants, including Pfizer’s Xalkori, Roche’s Rozlytrek, and Bristol Myers Squibb’s Augtyro. Despite high-value acquisitions behind Roche and Bristol Myers’ entries, sales have been modest, with Rozlytrek generating 134 million Swiss francs in 2024 and Augtyro recording just $38 million in revenue.
Nevertheless, Nuvation remains optimistic about Ibtrozi’s potential. CEO David Hung emphasized the drug’s exceptional response rates and sustained efficacy, particularly highlighting its ability to treat patients whose cancer has spread to the brain, a common progression pathway for the disease.
Financial analysts have mixed views on the drug’s prospects. RBC Capital Markets analyst Leonid Timashev projects peak annual sales could reach nearly $640 million, suggesting Wednesday’s stock decline may have been an overreaction to launch and labeling concerns. Jefferies analyst Michael Yee noted that Ibtrozi’s clinical results appear superior to competing treatments, though success will depend on Nuvation’s execution in the marketplace.
The approval represents a potential turning point for Nuvation Bio, which emerged from the legacy of Medivation – known for developing the successful prostate cancer treatment Xtandi before being acquired by Pfizer. However, market skepticism remains about the commercial viability of drugs targeting ROS1-positive lung cancers, given the limited success of previous entries in this space.
Despite these challenges, Nuvation positions Ibtrozi as a potential new benchmark in targeted therapy for ROS1-positive lung cancer. The drug’s demonstrated efficacy, particularly in patients with brain metastases, could help differentiate it in a competitive market where existing treatments have struggled to gain significant commercial traction.
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