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 gene alterations, a condition affecting approximately 2% of cases.
Clinical trials demonstrated impressive efficacy, with response rates reaching 90% and 85% in two separate studies among patients who hadn’t previously received tyrosine kinase inhibitor treatment. For those who had undergone prior therapy, the response rates were 52% and 62%. The durability of these responses proved significant, extending up to 47 months in one trial and 30 months in the other.
The drug’s approval comes with several safety considerations, including warnings about potential cardiac rhythm abnormalities, liver complications, and pulmonary inflammation. According to Nuvation’s data, adverse effects led to treatment discontinuation in 7% of patients. Following the announcement, the company’s stock value declined by approximately 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. Their acquisition of AnHeart Therapeutics last year provided a new direction through taletrectinib, now marketed as Ibtrozi.
The drug enters a competitive landscape dominated by established pharmaceutical giants. Existing treatments for ROS1-positive lung cancers include Pfizer’s Xalkori, Roche’s Rozlytrek, and Bristol Myers Squibb’s Augtyro. Despite high-value acquisitions to obtain these medicines, sales have been modest. Rozlytrek generated 134 million Swiss francs in 2024, while Augtyro’s revenue reached only $38 million.
Despite market skepticism, Nuvation remains optimistic about Ibtrozi’s potential. CEO David Hung emphasized the drug’s exceptional response rates and sustained effectiveness, particularly highlighting its ability to treat patients whose cancer has spread to the brain, a common progression pathway.
Financial analysts have offered mixed perspectives on the drug’s future. RBC Capital Markets analyst Leonid Timashev projects potential peak annual sales approaching $640 million, suggesting the market’s negative reaction to the approval might be excessive. He indicates that Ibtrozi could potentially outperform Augtyro based on its clinical profile.
Jefferies analyst Michael Yee characterized the drug’s trial results as potentially best-in-class, while acknowledging the challenge Nuvation faces as a smaller company competing against industry giants.
The approval marks a significant milestone for Nuvation Bio, which has experienced substantial market challenges since its Wall Street debut through a special purpose acquisition company merger five years ago. The company’s stock value has declined by more than 75% since 2020, reflecting broader biotechnology sector struggles.
This development follows successful regulatory approval in China and represents a crucial opportunity for Nuvation to establish itself in the oncology market. The company’s success will largely depend on its ability to differentiate Ibtrozi from existing treatments and effectively market its advantages, particularly its demonstrated efficacy in treating brain metastases and its sustained response duration.
The FDA’s decision adds another treatment option for patients with ROS1-positive non-small cell lung cancer, potentially offering new hope for those affected by this rare but serious condition. However, Nuvation’s ability to capture market share from established
competitors remains to be seen in this specialized therapeutic area.
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