Nuvation Bio Announces Expanded Access Program in the U.S. for Taletrectinib in Advanced ROS1-positive Non-Small Cell Lung Cancer

Rhea-AI Summary

Nuvation Bio (NYSE: NUVB) has launched an Expanded Access Program (EAP) in the United States for taletrectinib, targeting patients with advanced ROS1-positive non-small cell lung cancer (NSCLC) who lack alternative treatment options. The EAP, authorized by the FDA, enables patients with serious or life-threatening ROS1+ NSCLC to access taletrectinib outside the ongoing Phase 2 TRUST-II study.

This initiative follows the FDA's acceptance of Nuvation Bio's New Drug Application (NDA) for taletrectinib in December 2024. The application received Priority Review status with a PDUFA goal date of June 23, 2025. The NDA is supported by combined results from the Phase 2 TRUST-I and TRUST-II studies, which were presented at the ESMO Congress in September 2024.

Positive

• FDA granted Priority Review status for taletrectinib NDA
• EAP program provides additional access route for patients
• PDUFA date set for June 23, 2025, indicating near-term potential approval

Negative

• None.

Insights

Oncology Research Analyst

The announcement of Nuvation Bio's Expanded Access Program for taletrectinib marks a strategically significant milestone that could accelerate market penetration ahead of potential FDA approval. ROS1+ NSCLC, affecting approximately 1-2% of NSCLC patients, represents a specialized market with significant unmet needs.

The FDA's acceptance of the NDA with Priority Review status is particularly noteworthy, as it indicates both the drug's potential therapeutic value and the agency's recognition of the urgent need in this space. The June 23, 2025 PDUFA date, combined with Priority Review, suggests a high-confidence regulatory pathway.

Three key factors make this development particularly impactful for investors:

• The EAP program will help build physician familiarity and real-world experience with taletrectinib before potential commercial launch, potentially accelerating market uptake
• The line-agnostic treatment approach in the NDA submission indicates a broader potential patient population, expanding the commercial opportunity
• The involvement of patient advocacy groups like The ROS1ders suggests strong community support, which could influence adoption patterns

The timing of the EAP launch, coinciding with the NDA review period, demonstrates sophisticated commercial strategy, as it allows Nuvation to build presence in the ROS1+ NSCLC treatment landscape while navigating the regulatory process. This approach could provide valuable real-world data to support potential post-approval market expansion.

NEW YORK--(BUSINESS WIRE)-- Nuvation Bio Inc. (NYSE: NUVB), a global biopharmaceutical company tackling some of the greatest unmet needs in oncology, today announced it has initiated an Expanded Access Program (EAP) for taletrectinib in the U.S. for the treatment of patients with locally advanced or metastatic ROS1-positive (ROS1+) non-small cell lung cancer (NSCLC) when no comparable or satisfactory alternative therapy options are available.

“This EAP reflects our unwavering dedication to patients with ROS1-positive NSCLC,” said David Hung, M.D., Founder, President, and Chief Executive Officer of Nuvation Bio. “We are committed to working with the ROS1-positive NSCLC community, and the healthcare providers who serve them, to ensure this EAP provides eligible patients in the U.S. with efficient access to taletrectinib outside of our clinical trials.”

The EAP, as authorized by the U.S. Food and Drug Administration (FDA), allows patients with serious or immediately life-threatening ROS1+ NSCLC to access taletrectinib outside of the ongoing pivotal Phase 2 TRUST-II study.

“ROS1-positive NSCLC is a rare form of lung cancer with a unique patient journey to navigate,” said Janet Freeman-Daily, Co-Founder and President of The ROS1ders. “At The ROS1ders, we are constantly working toward our goal of improving survival and quality of life for the ROS1-positive cancer community by encouraging the research, development, and commercialization of effective treatments. Despite recent progress, there are times when approved therapies have not provided sufficient benefit or when patients are not eligible for a clinical trial. EAPs represent an opportunity for patients and their families to access investigational drugs that would not have otherwise been available to them.”

In December 2024, the U.S. FDA accepted Nuvation Bio’s New Drug Application (NDA) for taletrectinib for the treatment of advanced ROS1+ NSCLC (line agnostic). The U.S. FDA granted the application Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of June 23, 2025. The NDA is based on the pooled results from the pivotal Phase 2 TRUST-I and TRUST-II studies of taletrectinib, which were presented at the European Society of Medical Oncology (ESMO) Congress in September 2024.

About Expanded Access to Taletrectinib

EAPs are intended to serve as a potential pathway for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medical treatment outside of clinical trials and before it is approved by the U.S. FDA. The EAP for taletrectinib may be available for patients with locally advanced or metastatic ROS1+ NSCLC who, in the opinion of the treating clinician, are not appropriate candidates for approved ROS1 targeted therapies and ongoing taletrectinib clinical studies. For additional information on Nuvation Bio’s clinical trials and EAP, please visit https://www.nuvationbio.com/for-patients or contact earlyaccess@nuvationbio.com.

About Taletrectinib

Taletrectinib is an oral, potent, central nervous system-active, selective, next-generation ROS1 inhibitor specifically designed for the treatment of patients with advanced ROS1+ NSCLC. Taletrectinib is being evaluated for the treatment of patients with advanced ROS1+ NSCLC in two Phase 2 single-arm pivotal studies: TRUST-I (NCT04395677) in China, and TRUST-II (NCT04919811), a global study.

Based on pooled results of the TRUST-I and TRUST-II clinical studies, the U.S. FDA has accepted for Priority Review Nuvation Bio’s NDA for taletrectinib for the treatment of patients with advanced ROS1+ NSCLC (line agnostic, full approval) and assigned a PDUFA goal date of June 23, 2025. The U.S. FDA previously granted taletrectinib Breakthrough Therapy Designation for the treatment of patients with locally advanced or metastatic ROS1+ NSCLC who either have or have not previously been treated with ROS1 TKIs, and Orphan Drug Designation for the treatment of patients with ROS1+ NSCLC and other NSCLC indications. In January 2025, China’s National Medical Products Administration (NMPA) approved taletrectinib for the treatment of adult patients with locally advanced or metastatic ROS1+ NSCLC.

About ROS1+ NSCLC

Each year, more than one million people globally are diagnosed with NSCLC, the most common form of lung cancer. It is estimated that approximately 2% of patients with NSCLC have ROS1+ disease. Up to 35% of patients newly diagnosed with metastatic ROS1+ NSCLC have tumors that spread to their brain, increasing up to 55% for those whose cancer has progressed following initial treatment. Despite recent progress for patients with ROS1+ NSCLC, there remains a need for more effective and tolerable treatment options.

About Nuvation Bio

Nuvation Bio is a global biopharmaceutical company tackling some of the greatest unmet needs in oncology by developing differentiated and novel product candidates. Nuvation Bio’s programs include taletrectinib (ROS1), safusidenib (mIDH1), NUV-1511 (DDC), and NUV-868 (BET). 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, please visit www.nuvationbio.com or follow us on LinkedIn and X (@nuvationbioinc).

Forward Looking Statements

Certain statements included in this press release that are not historical facts are forward-looking statements for purposes of the safe harbor provisions under the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements are sometimes accompanied by words such as “believe,” “may,” “will,” “estimate,” “continue,” “anticipate,” “intend,” “expect,” “should,” “would,” “plan,” “predict,” “potential,” “seem,” “seek,” “future,” “outlook” and similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These forward-looking statements include, but are not limited to, our expectations regarding taletrectinib’s therapeutic potential in advanced ROS1+ NSCLC. These statements are based on various assumptions, whether or not identified in this press release, and on the current expectations of the management team of Nuvation Bio and are not predictions of actual performance. These forward-looking statements are subject to a number of risks and uncertainties that may cause actual results to differ from those anticipated by the forward-looking statements, including but not limited to the challenges associated with conducting drug discovery and initiating or conducting clinical studies due to, among other things, difficulties or delays in the regulatory process, enrolling subjects or manufacturing or acquiring necessary products; the emergence or worsening of adverse events or other undesirable side effects; risks associated with preliminary and interim data, which may not be representative of more mature data; and competitive developments. Risks and uncertainties facing Nuvation Bio are described more fully in its Form 10-Q filed with the SEC on November 6, 2024, under the heading “Risk Factors,” and other documents that Nuvation Bio has filed or will file with the SEC. You are cautioned not to place undue reliance on the forward-looking statements, which speak only as of the date of this press release. Nuvation Bio disclaims any obligation or undertaking to update, supplement or revise any forward-looking statements contained in this press release.

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Nuvation Bio Investor:

ir@nuvationbio.com

Nuvation Bio Media:

media@nuvationbio.com

Source: Nuvation Bio Inc.

FAQ

When is the PDUFA date for Nuvation Bio's (NUVB) taletrectinib NDA?

The FDA assigned a PDUFA goal date of June 23, 2025, for Nuvation Bio's taletrectinib NDA review.

What is the purpose of Nuvation Bio's (NUVB) Expanded Access Program for taletrectinib?

The EAP allows patients with serious ROS1+ NSCLC to access taletrectinib outside of clinical trials when no alternative therapy options are available.

When did the FDA accept Nuvation Bio's (NUVB) NDA for taletrectinib?

The FDA accepted Nuvation Bio's New Drug Application for taletrectinib in December 2024.

What clinical trials support Nuvation Bio's (NUVB) taletrectinib NDA?

The NDA is supported by pooled results from the Phase 2 TRUST-I and TRUST-II studies, presented at ESMO Congress in September 2024.