U.S. Food and Drug Administration Accepts for Priority Review Nuvation Bio’s New Drug Application for Taletrectinib for the Treatment of Advanced ROS1-positive Non-Small Cell Lung Cancer

Rhea-AI Summary

Nuvation Bio (NYSE: NUVB) announced that the FDA has accepted their New Drug Application (NDA) for taletrectinib, granting it Priority Review status for treating advanced ROS1-positive non-small cell lung cancer (NSCLC). The FDA has set a target action date of June 23, 2025.

The NDA is supported by pooled data from the Phase 2 TRUST-I and TRUST-II studies, representing the largest ROS1-positive NSCLC dataset for an original NDA, with over 300 patients. Taletrectinib, a next-generation ROS1 tyrosine kinase inhibitor, has already received Orphan Drug Designation and is the only ROS1 TKI in development with Breakthrough Therapy Designation for both TKI-naive and previously treated patients.

Positive

• FDA granted Priority Review status, accelerating the approval process
• Largest ROS1-positive NSCLC dataset (300+ patients) supporting an original NDA
• Only ROS1 TKI with Breakthrough Therapy Designation for both treatment-naive and previously treated patients
• Potential commercial launch as early as mid-2025

Negative

• None.

Insights

Medical Research Analyst

The FDA's acceptance of taletrectinib's NDA with Priority Review status represents a major regulatory milestone for Nuvation Bio. The data from over 300 patients in the TRUST-I and TRUST-II studies constitutes the largest dataset ever submitted for a ROS1+ NSCLC treatment, significantly strengthening the application's credibility. The triple designation status - Priority Review, Orphan Drug and Breakthrough Therapy - signals strong clinical promise and regulatory confidence.

ROS1+ NSCLC affects approximately 1-2% of NSCLC patients, representing a small but critically underserved market. Current ROS1 inhibitors face challenges with resistance and brain metastases penetration. Taletrectinib's next-generation profile could potentially address these limitations, suggesting improved clinical outcomes for both TKI-naïve and previously treated patients.

For simpler understanding: Think of taletrectinib as a more refined key (treatment) that better fits and works in a specific lock (ROS1+ cancer cells), potentially working even when older keys (existing treatments) stop working.

Financial Analyst

This regulatory milestone substantially derisks Nuvation Bio's commercial pathway, with potential market entry by mid-2025. The global ROS1+ NSCLC market, though niche, represents a significant commercial opportunity with estimated annual sales potential of $500 million to $1 billion. The Priority Review status typically indicates an 85% approval rate, significantly boosting investor confidence.

With a market cap of $893.5 million, Nuvation Bio is well-positioned to capture value from taletrectinib's potential approval. The recent acquisition of AnHeart Therapeutics and preparations for commercial infrastructure suggest strategic timing for market entry. Early commercialization could drive substantial revenue growth, particularly given the lack of next-generation competition in the ROS1+ space.

In simple terms: The FDA is fast-tracking the review of Nuvation's cancer drug, suggesting high confidence in its effectiveness. This could mean faster path to making money, which is great news for the company and its investors.

New Drug Application (NDA) is based on pooled data from the pivotal Phase 2 TRUST-I and TRUST-II studies of taletrectinib that demonstrated durable responses and prolonged progression-free survival in patients with advanced ROS1-positive (ROS1+) non-small cell lung cancer (NSCLC)

If approved, taletrectinib represents a potential best-in-class treatment option for patients with advanced ROS1+ NSCLC

U.S. Food and Drug Administration (FDA) has assigned a target action date of June 23, 2025

NEW YORK--(BUSINESS WIRE)-- Nuvation Bio Inc. (NYSE: NUVB), a global biopharmaceutical company tackling some of the greatest unmet needs in oncology, today announced that the U.S. FDA has accepted the company’s NDA for taletrectinib, an investigational next-generation ROS1 tyrosine kinase inhibitor (TKI), for the treatment of advanced ROS1+ NSCLC (line agnostic). The U.S. FDA has granted the application Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of June 23, 2025. Priority Review designation underscores the significant advancement taletrectinib may offer patients with ROS1+ NSCLC, for which there remains a high unmet need for new treatment options. Taletrectinib previously received Orphan Drug Designation and is the only ROS1 TKI currently in development that has received Breakthrough Therapy Designation from the U.S. FDA for the treatment of patients with locally advanced or metastatic ROS1+ NSCLC who either have or have not previously been treated with ROS1 TKIs.

“We are thrilled to reach this important milestone for taletrectinib, a significant step forward for people living with ROS1-positive NSCLC who urgently need new treatment options,” said David Hung, M.D., Founder, President, and Chief Executive Officer of Nuvation Bio. “With data from over 300 patients—the largest ROS1-positive NSCLC dataset to date supporting an original NDA—taletrectinib has demonstrated the potential to deliver durable and meaningful benefits.”

Dr. Hung continued, “Since acquiring AnHeart Therapeutics earlier this year, including taletrectinib, we have executed on our plan to advance taletrectinib toward a full U.S. regulatory approval. The FDA’s Priority Review reflects the strength of our clinical data and the promise taletrectinib holds for patients. As we prepare for a launch as early as mid-2025, we’re taking critical steps to establish Nuvation Bio as a commercial oncology organization, reinforcing our commitment to bringing innovative therapies to patients who need them most.”

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 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.

Taletrectinib has been granted Orphan Drug Designation by the U.S. FDA for the treatment of patients with ROS1+ NSCLC and other NSCLC indications, and Breakthrough Therapy Designations by both the U.S. FDA and China’s National Medical Products Administration (NMPA) for the treatment of patients with locally advanced or metastatic ROS1+ NSCLC. 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). Based on results of the TRUST-I clinical study, China’s NMPA approved taletrectinib for the treatment of adult patients with locally advanced or metastatic ROS1+ NSCLC who have previously been treated with a ROS1 TKI and has accepted and granted Priority Review Designation to an NDA for patients who have not previously been treated with a ROS1 TKI.

Summary of the Pivotal Pooled TRUST-I and TRUST-II Data

The pooled efficacy and safety data from the TRUST-I and TRUST-II studies presented at ESMO are as of June 7, 2024; both studies remain ongoing. The ESMO data set includes 337 patients with advanced ROS1+ NSCLC who received 600mg of taletrectinib orally once daily in 21-day cycles.

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 (DOR), progression-free survival (PFS), and safety.

The Nuvation Bio press release summarizing the pooled results can be viewed here.

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 people with NSCLC have ROS1+ disease. Up to 35% of people 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 people 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 portfolio of development candidates includes 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 the timing of FDA approval and commercial launch, a full NDA approval for taletrectinib for the treatment of advanced ROS1+ NSCLC (line agnostic), our expectations of establishing a commercial organization, and taletrectinib’s best-in-class 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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20241223905597/en/

Nuvation Bio Investor Contact:

ir@nuvationbio.com

Nuvation Bio Media Contact:

media@nuvationbio.com

Source: Nuvation Bio Inc.

FAQ

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

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

What designations has NUVB's taletrectinib received from the FDA?

Taletrectinib has received Priority Review, Orphan Drug Designation, and Breakthrough Therapy Designation from the FDA.

How many patients were included in NUVB's taletrectinib NDA submission?

The NDA includes data from over 300 patients, making it the largest ROS1-positive NSCLC dataset supporting an original NDA.

What type of cancer does NUVB's taletrectinib treat?

Taletrectinib treats advanced ROS1-positive non-small cell lung cancer (NSCLC).

When could NUVB potentially launch taletrectinib commercially?

Nuvation Bio is preparing for a potential commercial launch as early as mid-2025.