Taletrectinib

Approval follows Priority Review and is supported by the robust TRUST clinical program, in which IBTROZI treatment demonstrated high, durable response rates and brain-penetrant efficacy across different lines of therapy The safety and tolerability of IBTROZI have been well established in the pivotal program, with one of the largest safety datasets in ROS1+ NSCLC showing a favorable and consistent profile Company to host conference call tomorrow, June 12 at 7:30 a.m. EDT NEW YORK, NY, USA I June 12, 2025 I Nuvation Bio Inc. (NYSE: NUVB), a global oncology company focused on tackling some of the toughest challenges in cancer treatment, today announced that the U.S. Food and Drug Administration (FDA) has approved IBTROZI™ (taletrectinib) for the treatment of adult patients with locally advanced or metastatic ROS1-positive (ROS1+) non-small cell lung cancer (NSCLC). IBTROZI is a highly selective, next-generation oral ROS1 tyrosine kinase inhibitor (TKI) designed to address some of the outstanding challenges of treating ROS1+ NSCLC. It has demonstrated high response rates with durable benefit and intracranial activity and is generally well tolerated, providing a new treatment option for patients with advanced ROS1+ NSCLC. “The FDA approval of IBTROZI marks a major milestone in the evolution of targeted therapy for advanced ROS1-positive NSCLC,” said David Hung, M.D., Founder, President and Chief Executive Officer of Nuvation Bio. “We believe one of the greatest threats to ROS1-positive lung cancer patients is disease progression, especially in the first-line setting. In pivotal trials, IBTROZI delivered high response rates with sustained durability—truly meaningful benefits for patients. With its clinically proven efficacy and safety profile, we believe IBTROZI has the potential to become a new standard for what targeted therapies can achieve in this type of lung cancer. With approvals for IBTROZI now in the U.S. and China, and additional global filings underway, we remain committed to delivering innovative therapies that help patients stay ahead of their disease.” ROS1+ NSCLC is a rare and aggressive form of lung cancer, accounting for approximately 2% of new NSCLC cases, or about 3,000 new diagnoses of advanced disease annually in the U.S. The median age at diagnosis for patients with this type of lung cancer is approximately 50 years old, and the disease is more likely to occur in people who have never smoked. Brain metastases are common and a leading cause of disease progression and mortality in this population. “For people living with advanced ROS1-positive lung cancer, who tend to be diagnosed at a younger age, having another treatment option can make a real difference for them and their loved ones,” said Janet Freeman-Daily, Co-Founder and President of The ROS1ders. “The approval of this new targeted therapy is a meaningful step forward for the advanced ROS1+ lung cancer community and offers hope for patients facing the added challenge of cancer spreading to the brain.” The FDA approval of IBTROZI is supported by one of the largest global clinical trial programs in ROS1+ NSCLC to date, with over 300 patients enrolled in the pivotal TRUST-I and TRUST-II studies. In TRUST-I, IBTROZI achieved a confirmed overall response rate (cORR) of 90% in TKI-naïve patients. These findings were reinforced by the TRUST-II results, with a cORR of 85% in TKI-naïve patients. The median duration of response (DOR) was not yet reached for either trial, based on a cutoff date that is nearly five months later than that of the pooled TRUST-I and TRUST-II analysis published in April in the Journal of Clinical Oncology . For TRUST-I, with a median follow-up for responses of 40 months, the longest DOR was observed at 46.9 months and ongoing. For TRUST-II, with a median follow-up for responses of 19 months, the longest DOR was observed at 30.4 months and ongoing as of October 2024. Given the single-arm nature of the TRUST clinical studies, median progression-free survival (PFS) is not provided in the label. Across the pivotal studies, consistent results were also observed among patients who were previously treated with a ROS1 TKI (TKI-pretreated). In TRUST-I, treatment with IBTROZI achieved a cORR of 52% and median DOR of 13.2 months for TKI-pretreated patients, with median follow-up for responses of 33 months. In TRUST-II, treatment with IBTROZI achieved a cORR of 62%, and as of October 2024 the median DOR was 19.4 months in these patients, with a median follow-up for responses of 19 months. Brain metastases are among the most common and devastating complications in advanced ROS1+ NSCLC. IBTROZI was designed to penetrate the central nervous system (CNS) and has demonstrated consistent intracranial responses in patients with measurable brain metastases at baseline. An intracranial response was achieved in 73% of TKI-naive patients (11/15) and 63% of TKI-pretreated patients (15/24). “Patients living with advanced ROS1+ non-small cell lung cancer and their healthcare providers are in need of new treatment options,” added Nathan Pennell, M.D., Ph.D., TRUST study investigator and Professor of Medicine at the Cleveland Clinic. “IBTROZI’s durability of response and ability to effectively penetrate the brain, coupled with a well-characterized and manageable safety profile, further addresses these critical needs for patients. I believe this now-approved therapy offers providers and patients a promising new option for the treatment of advanced ROS1+ non-small cell lung cancer.” Dr. Pennell is a compensated member of Nuvation Bio’s advisory committee. IBTROZI was generally well-tolerated, with most adverse events being low grade, transient and manageable. Patients infrequently (7%) discontinued treatment due to treatment-emergent adverse events (TEAEs). The most common adverse reactions (≥20%) included diarrhea (64%), nausea (47%), vomiting (43%), dizziness (22%), rash (22%), constipation (21%), and fatigue (20%). Overall, the majority of CNS events were mild to moderate (~90%) and resolved within days, and dose modifications due to these events were low (~5%). Approximately 90% of reported cases of dizziness were Grade 1 (mild) and transient. Liver enzyme elevations (AST 87%/ALT 85%) and QT prolongation (19%) were manageable with standard monitoring and dose modifications. IBTROZI is approved as a 600 mg once-daily oral dose, supported by a half-life of approximately 66 hours and broad tissue distribution, including the brain, enabling sustained systemic and CNS exposure. Nuvation Bio also announced the launch of NuvationConnect, a program designed to support patients prescribed IBTROZI. The program will offer financial assistance, access to resources and personalized support for eligible patients. Prescribers can learn more at NuvationConnect.com or by calling 1-877-NUV-CON1 (1-877-688-2661). Conference Call & Business Update Nuvation Bio will host a conference call on June 12, 2025 at 7:30 a.m. EDT / 4:30 a.m. PDT, where company executives will provide an overview of the FDA approval of IBTROZI and our plans to now bring this medicine to patients. As a reminder to investors, the approval, together with the first commercial sale, makes the funding secured from Sagard Healthcare Partners in March fully available to the company. Nuvation Bio expects that this finances the launch of IBTROZI in full and provides further resources to continue advancing our pipeline in areas of unmet need. Investors and the general public are invited to register and listen to a live webcast of the event through the company website at https://investors.nuvationbio.com . Those unable to register can access the live conference call by dialing +1 833-470-1428 (U.S. toll-free) and entering access code 783971. A replay of the event will be available shortly after the conclusion. About ROS1+ NSCLC Each year, more than one million people globally are diagnosed with non-small cell lung cancer (NSCLC), the most common form of lung cancer. It is estimated that approximately 2% of patients with NSCLC have ROS1+ disease. About 35% of patients newly diagnosed with metastatic ROS1+ NSCLC have tumors that have spread to their brain. The brain is also the most common site of disease progression, with about 50% of previously treated patients developing CNS metastases. Despite recent progress for patients with ROS1+ NSCLC, there remains a need for more effective and tolerable treatment options. About IBTROZI IBTROZI is an oral, potent, central nervous system-active, selective, next-generation ROS1 inhibitor therapy approved for the treatment of adult patients with advanced ROS1-positive non-small cell lung cancer. Learn more at IBTROZI.com. About the TRUST Clinical Program The TRUST clinical program evaluating IBTROZI for the treatment of adult patients with advanced ROS1+ NSCLC included two Phase 2 single-arm pivotal studies: TRUST-I (NCT04395677) in China, which enrolled 173 patients, and TRUST-II (NCT04919811), a global study, which enrolled 164 patients. The primary endpoint of these registrational studies is confirmed objective response rate (cORR) as assessed by an independent review committee (IRC). Key secondary endpoints include intracranial cORR, duration of response, progression-free survival, and safety. Indication IBTROZI is indicated for the treatment of adult patients with locally advanced or metastatic ROS1+ non-small cell lung cancer (NSCLC). About Nuvation Bio Nuvation Bio is a global oncology company focused on tackling some of the toughest challenges in cancer treatment by developing therapies that create a profound, positive impact on patients’ lives. Our diverse pipeline includes IBTROZI (taletrectinib), a next-generation ROS1 inhibitor; safusidenib, a brain-penetrant IDH1 inhibitor for glioma; NUV-1511, an innovative drug-drug conjugate (DDC) designed for targeted cancer treatment; and NUV-868, a BD2-selective BET inhibitor. Nuvation Bio was founded in 2018 by biopharma industry veteran David Hung, M.D., who previously founded Medivation, Inc., which brought to patients one of the world’s leading prostate cancer medicines. Nuvation Bio has offices in New York, San Francisco, Boston, and Shanghai. For more information, visit www.nuvationbio.com or follow the company on LinkedIn and X (@nuvationbioinc). SOURCE: Nuvation Bio