PIPELINE
Nuvation Bio is focused on treating patients with the most difficult-to-treat cancers for which conventional therapies have failed. Our programs include taletrectinib (ROS1 inhibitor), safusidenib (mIDH1 inhibitor), NUV-1511 (drug-drug conjugate), and NUV-868 (BET inhibitor).
Program
Potential Indication(s)
Current Stage of Development
Preclinical
Phase 1
Phase 2
Pivotal
Anticipated Milestones & Recent Updates
Taletrectinib1
(ROS1)
Advanced ROS1+ NSCLC
(treatment line agnostic)
• NDA accepted by U.S. FDA for priority review (line agnostic)
• Approved by China’s NMPA for advanced ROS1+ NSCLC55
• Pooled data from pivotal TRUST-I & TRUST-II studies
presented at ESMO in September 2024
Safusidenib2
(mIDH1)
Diffuse IDH1-mutant glioma
• Entering pivotal studies in 2025
• Phase 2 study ongoing
NUV-1511
(DDC)
Advanced solid tumors 3
• Phase 1/2 dose escalation study ongoing
NUV-868
(BET)
Currently under internal evaluation 4
• Completed Phase 1 monotherapy and Phase 1b
combination studies in advanced solid tumors
NUV-868
(BET)
Advanced solid
tumors3
NUV-868 +
olaparib
Phase 1b dose escalation study ongoing
BET: Bromodomain and Extra-Terminal motif; ESMO: European Society of Medical Oncology Congress; mIDH1: mutant isocitrate dehydrogenase 1; NSCLC: Non-small cell lung cancer; PDUFA: Prescription Drug User Fee Act; ROS1+: c-ros oncogene 1-positive; 1. Taletrectinib has been granted Orphan Drug Designation from 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 NMPA for the treatment of patients with locally advanced or metastatic ROS1+ NSCLC; worldwide development and commercial rights in-licensed from Daiichi Sankyo; rights to taletrectinib have been out-licensed in China and Japan. 2. Worldwide development and commercial rights in-licensed from Daiichi Sankyo, excluding Japan where Daiichi Sankyo retains development and commercial rights. 3. Includes patients with advanced solid tumors who previously received and progressed on or after treatment with Enhertu® and/or Trodelvy® per approved U.S. FDA labeling, human epidermal growth factor receptor 2-negative (HER2-) metastatic breast cancer, metastatic castration-resistant prostate cancer (mCRPC), advanced pancreatic cancer, and platinum-resistant ovarian cancer. 4. Nuvation Bio has decided not to initiate a Phase 2 study of NUV-868 as a monotherapy or in combination with olaparib or enzalutamide in the advanced solid tumor indications that were part of the Phase 1 and Phase 1b study designs. The Company is evaluating next steps for the NUV-868 program, including further development in combination with approved products for indications in which BD2-selective BET inhibitors may improve outcomes for patients. 5. 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 or have not previously been treated with ROS1 TKIs.
ROS1
Taletrectinib is an oral, potent, central nervous system-active, selective, next-generation ROS1 inhibitor specifically designed for the treatment of patients with ROS1-positive (ROS1+) non-small cell lung cancer (NSCLC). In December 2024, the U.S. FDA accepted for priority review the NDA for taletrectinib for the treatment of advanced ROS1-positive NSCLC (PDUFA goal date of June 23, 2025).
The U.S. FDA has 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. 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). In addition, in January 2025, China’s NMPA approved taletrectinib for the treatment of locally advanced or metastatic 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.
mIDH1
Safusidenib is a novel, oral, potent, brain penetrant, targeted inhibitor of mutant IDH1 (mIDH1).
Safusidenib is being evaluated in a Phase 2 study for the treatment of patients with diffuse IDH1-mutant glioma. Safusidenib has shown high blood-brain barrier penetration in both pre-clinical and clinical studies and demonstrated anti-tumor activity and tolerability in a Phase 1 clinical study. Glioma is the most common type of adult brain cancer, and IDH1 mutations are present in the majority of low-grade diffuse gliomas. There are currently no targeted treatments approved for IDH-mutant glioma; current options include surgery, radiation, and chemotherapy.
DDC
NUV-1511, our first clinical-stage drug-drug conjugate (DDC), fuses a targeting agent to a widely used chemotherapy agent.
Our proprietary, small molecule DDC’s leverage a novel therapeutic approach within the drug-conjugate class of anti-cancer therapies. The platform is designed to selectively deliver potent anti-cancer therapeutics to cancer cells to exert greater toxicity against these target tumor cells than against healthy non-target tissues.
Utilizing this technology, we are able to design potent oncology-focused chimeric small molecules which combine tumor-targeting specificity with anti-cancer activity of known oncology agents. We believe our DDC technology will be broadly applicable and can be replicated across many existing therapies to transform the standard-of-care in multiple oncology indications.
In March 2024, we treated the first patient in a Phase 1/2 study of NUV-1511. The study will initially evaluate safety and tolerability, pharmacokinetic profile, and assess for signs of clinical activity in patients with advanced solid tumors who previously received and progressed on or after treatment with Enhertu® and/or Trodelvy® per approved U.S. FDA labeling, human epidermal growth factor receptor 2-negative (HER2-) metastatic breast cancer, mCRPC, advanced pancreatic cancer, and platinum-resistant ovarian cancer (PROC). The dose escalation portion of the study employs a flexible design that allows for the potential to explore two dosing schedules for NUV-1511, with the goal of establishing the recommended Phase 2 dose.
BET
NUV-868 is a BD2-selective, oral, small molecule bromodomain and extra-terminal (BET) inhibitor that inhibits BRD4. BRD4 is a key member of the BET family, which epigenetically regulates proteins that control tumor growth and differentiation.
NUV-868 is almost 1,500 times more selective for BD2 than BD1 and is designed to alleviate the therapeutic limiting toxicities observed by other non-BD2 selective BET inhibitors.
We have completed a Phase 1 monotherapy study and a Phase 1b study of NUV-868 in combination with olaparib or enzalutamide. We are evaluating next steps for the NUV-868 program, including further development in combination with approved products for indications in which BD2-selective BET inhibitors may improve outcomes for patients.
