On September 15, 2026 Elevar Therapeutics, Inc., a majority-owned subsidiary of HLB Co., Ltd. and a fully integrated biopharmaceutical company dedicated to elevating treatment experiences and outcomes for cancer patients, reported the submission of a marketing authorization application to the European Medicines Agency (EMA) for lirafugratinib as a second-line treatment for patients with advanced/metastatic cholangiocarcinoma (CCA) harboring FGFR2 fusions or rearrangements.

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The application is based on results of the Phase 1/2 ReFocus study, of which the primary endpoint is objective response rate (ORR). In advanced/metastatic FGFR2 fusion+ CCA patients after receiving a prior line of systemic therapy, lirafugratinib achieved an ORR of 46.5% as assessed by independent review committee. The median duration of response, a key secondary endpoint, was 11.8 months. Median progression-free survival was 11.3 months, while median overall survival was 22.8 months.

"Lirafugratinib is a novel drug candidate that has demonstrated meaningful clinical activity and a differentiated safety profile consistent with its high selectivity for FGFR2 and sustained inhibitory mechanism," said Dong-Gun Kim, chief executive officer of Elevar. "We will continue to advance the regulatory processes in the United States and European Union while broadening the therapeutic potential of lirafugratinib across multiple solid tumors through our tumor-agnostic clinical development program."

Lirafugratinib is an irreversible inhibitor that selectively targets FGFR2, differentiating it from existing pan-FGFR inhibitors that broadly inhibit FGFR1, FGFR3, and/or FGFR4 in addition to FGFR2. Consistent with its high selectivity, the rates of hyperphosphatemia and diarrhea – adverse events commonly associated with non-selective FGFR inhibitors – were 20.7% and 21.6%, respectively.

Lirafugratinib received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) in 2022 and Breakthrough Therapy Designation in 2023. Elevar submitted a New Drug Application for lirafugratinib to the FDA in January 2026, and the application was granted Priority Review. The FDA is currently reviewing the application, with a Prescription Drug User Fee Act target action date of Sept. 25, 2026.

Elevar is also conducting ReFocus202, a global Phase 2 study evaluating lirafugratinib in patients with various solid tumors harboring FGFR2 fusions or rearrangements. Patient enrollment and dosing are currently underway at clinical sites globally, including in the EU, U.K., Korea and U.S. Through this program, Elevar seeks to expand the therapeutic potential of lirafugratinib beyond CCA to a broader range of solid tumors with a FGFR2 fusion or rearrangement.

About Lirafugratinib

Lirafugratinib (aka RLY-4008) is a potent, selective and oral small molecule inhibitor of FGFR2, a receptor tyrosine kinase that is frequently altered in certain cancers. FGFR2 is one of four members of the FGFR family, a set of closely related proteins with highly similar protein sequences and properties. Lirafugratinib is currently being evaluated in a clinical trial to enroll additional patients with previously treated, advanced or metastatic solid tumors other than CCA harboring FGFR2 fusion or rearrangement, who have not been treated with prior FGFR inhibitors. Elevar has an exclusive license to lirafugratinib from Relay Therapeutics, Inc. for commercialization worldwide.

(Press release, Elevar Therapeutics, SEP 15, 2026, View Source [SID1234670882])

On September 15, 2026 Genexine, Inc. (KOSDAQ: 095700) reported that Jaehyun Choi, Ph.D., CEO and Head of R&D, presented new preclinical data for GX-BP1, the company’s SOX2-targeting bioPROTAC, at the 2026 World Conference on Lung Cancer (WCLC) in Seoul on September 13.

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The mini oral presentation, titled ‘Lung-Targeted mRNA-LNP Delivery of SOX2 bioPROTAC: A Comprehensive Strategy for LUAD, LUSC, and SCLC in Monotherapy and Combination Settings’ highlighted the potential of GX-BP1 to overcome treatment resistance and suppress tumor regrowth when combined with standard lung cancer therapies, including the EGFR-targeted therapy osimertinib and the TROP2 antibody-drug conjugate datopotamab deruxtecan (Dato-DXd).

In an HCC827 EGFR-mutant lung cancer xenograft model, osimertinib initially produced strong tumor control, but tumors began to regrow after treatment ended at day 20. The combination of osimertinib and Dato-DXd delayed regrowth, but tumors eventually progressed from day 40 and showed significant volume after day 70.

In contrast, osimertinib plus GX-BP1 combination therapy maintained durable tumor control for more than 100 days, with no substantial tumor regrowth observed during the study period. Most notably, the triple combination of GX-BP1, osimertinib and Dato-DXd achieved complete responses in all six animals (6/6 CR) with no regrowth observed.

Genexine also presented data from a Dato-DXd-resistant tumor model. GX-BP1 alone achieved 92.3% tumor growth inhibition (TGI), while the combination of GX-BP1 and Dato-DXd achieved 115.3% TGI, indicating tumor regression from baseline despite resistance to the ADC.

Targeting SOX2 to Address Recurrence and Drug Resistance

Genexine believes the antitumor activity of GX-BP1 is driven by degradation of SOX2, a transcription factor associated with cancer stemness, treatment resistance, recurrence and metastasis.

In EGFR-mutant lung cancer cells, treatment with osimertinib increased SOX2 expression in a dose-dependent manner. Similar increases were observed following treatment with other EGFR-targeted therapies, including lazertinib and amivantamab.

Osimertinib treatment also increased the expression of genes associated with drug efflux pumps, a resistance mechanism that reduces intracellular drug exposure by transporting therapeutic agents or cytotoxic compounds out of cancer cells. Such mechanisms may also reduce intracellular exposure to cytotoxic payloads delivered by ADCs.

By degrading SOX2, GX-BP1 is designed to target cancer stem cells and drug-persistent cancer cells while reducing mechanisms associated with treatment resistance.

"Our goal with GX-BP1 is to develop it as a combination backbone molecule with any standard therapies for lung cancer as an anti-resistant anti-cancer therapy" said Dr. Choi. "The WCLC data support the potential of GX-BP1 to improve the durability of response when combined with EGFR-targeted therapies and ADCs."

Dr. Choi added, "The observed effect on drug-efflux mechanisms generated significant interest during our WCLC presentation. We plan to further develop GX-BP1 in combination with established lung cancer therapies and actively pursue global licensing opportunities with companies developing EGFR-targeted agents and ADCs."

(Press release, Genexine, SEP 15, 2026, View Source [SID1234670881])

On September 15, 2026 Summit Therapeutics Inc. (Nasdaq: SMMT) reported that updated overall survival (OS) results from the global Phase III HARMONi clinical trial featuring the novel, potential first-in-class investigational bispecific antibody ivonescimab were presented today at the International Association for the Study of Lung Cancer (IASLC) 2026 World Conference on Lung Cancer (WCLC 2026) in Seoul, Republic of Korea.

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As Summit previously announced on July 22, 2026, ivonescimab plus platinum-doublet chemotherapy in the HARMONi trial continued to show a positive OS trend and a consistent efficacy and safety profile in Asian and western patients when compared to placebo plus chemotherapy.

"Updated results from the global Phase III HARMONi study show that ivonescimab combined with chemotherapy continued to demonstrate a consistent overall survival improvement compared with placebo plus chemotherapy in patients with EGFR-mutated non-small cell lung cancer following prior treatment with a third-generation EGFR TKI," said Antonio Passaro, M.D., Ph.D., Director of the Division of Thoracic Oncology, European Institute of Oncology (IEO) in Milan, Italy, and presenting author. "Importantly, with longer follow-up, the survival improvement observed in western patients was consistent with the global population, reinforcing the relevance of these results across geographic regions in a setting where patients continue to need additional treatment options after progression on EGFR-targeted therapy."

HARMONi Detailed Efficacy and Safety Results

The HARMONi study is evaluating ivonescimab combined with chemotherapy compared to placebo plus chemotherapy in patients with epidermal growth factor receptor (EGFR)-mutated, locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) who were previously treated with a third-generation EGFR tyrosine kinase inhibitor (TKI). The study demonstrated a statistically significant benefit in the primary analysis for progression-free survival (PFS), one of the study’s two primary endpoints, the other being OS.

In April 2025, the primary OS analysis was performed, whereby ivonescimab in combination with chemotherapy showed a positive trend without achieving a statistically significant benefit with a hazard ratio of 0.79 (95% CI: 0.62 – 1.01; p=0.057). Median OS was 16.8 months for those patients administered ivonescimab plus chemotherapy vs. 14.0 months for those receiving placebo plus chemotherapy. At the time of the primary analysis, median follow-up time for western patients was 9.2 months which was less than the median OS.

An additional analysis was performed with a data cut-off date in June 2026, whereby most western patients have discontinued or completed two years of treatment (median follow-up time 23.2 months for western patients). Median follow-up time for Asian patients was 32.7 months (this was reached in April 2025 and Asian patient data was locked at the time of that analysis). The updated June 2026 analysis continued to show consistent, favorable OS results, with an OS hazard ratio of 0.76 (95% CI: 0.61 – 0.95; nominal p=0.0151) in the global intention-to-treat (ITT) population. An OS hazard ratio of 0.76 was demonstrated in the western patient subgroup (95% CI: 0.52 – 1.10), which was consistent with the ITT population and Asian subgroup.

Global ITT Overall Survival Analyses at Each Data Cut-Off

DCO: Apr 2025

(Primary Analysis)

DCO: Sept 2025*

DCO: June 2026*

Ivonescimab + Chemo

Placebo + Chemo

Ivonescimab + Chemo

Placebo + Chemo

Ivonescimab + Chemo

Placebo + Chemo

(n=219)

(n=219)

(n=219)

(n=219)

(n=219)

(n=219)

Median OS, ITT

16.8 mos

14.0 mos

16.8 mos

14.0 mos

16.8 mos

14.0 mos

Hazard Ratio, ITT

0.79

(95% CI: 0.62 – 1.01;
p=0.057)

0.78

(95% CI: 0.62 – 0.98; nominal p=0.0332)

0.76

(95% CI: 0.61 – 0.95; nominal p=0.0151)

DCO = data cut-off; ITT = intention-to-treat population; mos = months; chemo = chemotherapy

*DCO for Asian patients was Apr 2025 for both Sept 2025 and June 2026 analyses

With longer follow-up, western patients replicated the survival improvement seen in Asian patients, further reinforcing the regional consistency of the efficacy results observed in the global HARMONi study.

Western Subgroup of Overall Survival Analyses at Each Data Cut-Off

DCO: Apr 2025

DCO: Sept 2025

DCO: June 2026

Ivonescimab + Chemo

Placebo + Chemo

Ivonescimab + Chemo

Placebo + Chemo

Ivonescimab + Chemo

Placebo + Chemo

(n=83)

(n=82)

(n=83)

(n=82)

(n=83)

(n=82)

Median OS, Western Patients

Not Reached

14.0 mos

17.0 mos

14.0 mos

17.5 mos

14.0 mos

Hazard Ratio, Western Patients

0.98

(95% CI: 0.55 – 1.73)

0.84

(95% CI: 0.53 – 1.32)

0.76
(95% CI: 0.52 – 1.10)

Median follow-up, Western Patients

9.2 mos

13.7 mos

23.2 mos

DCO = data cut-off; ITT = intention-to-treat population; mos = months; chemo = chemotherapy

In this most recent analysis, ivonescimab continued to demonstrate an acceptable and manageable safety profile that was consistent with previous Phase III data of ivonescimab plus chemotherapy. No additional safety signals were noted in this latest HARMONi data cut.

"The updated HARMONi overall survival analysis presented at WCLC 2026 provides important additional evidence of the consistency of ivonescimab’s clinical profile across patient populations, with western patients showing consistent survival improvement to what was observed in Asian patients," stated Dr. Maky Zanganeh, President and Co-Chief Executive Officer of Summit. "Together with the continued acceptable and manageable safety profile, these data reinforce our confidence in the HARMONi results as we work toward the potential approval of ivonescimab in the U.S."

"What continues to distinguish ivonescimab is not only the strength of these HARMONi results, but the expanding clinical data sets emerging across studies and tumor types, including recent positive results from HARMONi-2 and HARMONi-GI1 in biliary tract cancer," added Robert W. Duggan, Chairman and Co-Chief Executive Officer of Summit. "Together, these data reinforce our belief in the potential breadth of ivonescimab’s differentiated bispecific design as an important new therapeutic approach in oncology, beginning with EGFR-mutated non-small cell lung cancer and extending across our broader ambition to address serious needs in solid tumors where patients urgently need better options."

Summit’s Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) is based on the results from the HARMONi trial and has a Prescription Drug User Fee Act (PDUFA) goal action date of November 14, 2026.

HARMONi-2 Detailed OS Efficacy and Safety Results

Previously, key findings from the HARMONi-2 primary OS analysis were announced by Summit’s partner, Akeso Inc. Today, the full detailed results were presented at WCLC 2026, with highlights provided below. HARMONi-2 (AK112-303) is a single-region, multi-center Phase III study conducted in China and sponsored by Akeso, with all relevant data exclusively generated, managed, and analyzed by Akeso.

In this protocol-specified interim analysis of OS, a secondary endpoint in the HARMONi-2 study, ivonescimab monotherapy demonstrated a statistically significant and clinically meaningful improvement compared to pembrolizumab monotherapy, achieving a hazard ratio (HR) of 0.73 (95% CI: 0.57, 0.95; p=0.009). A clinically meaningful benefit was demonstrated across important clinical subgroups, including those with PD-L1 low expression (PD-L1 Score 1-49%) and PD-L1 high expression (PD-L1 Score ≥ 50%), along with those with squamous and non-squamous histologies.

HARMONi-2 ITT (n=398)

Median Follow-up: 36.0 mos

Ivonescimab

(n=198)

Pembrolizumab

(n=200)

Median OS

30.8 mos

(95% CI: 25.4, 37.8)

22.6 mos

(95% CI: 17.8, 26.5)

OS Stratified HR

0.73

(95% CI: 0.57, 0.95; p=0.009)

24-Month KM OS Rate

57.9%

48.0%

36-Month KM OS Rate

45.0%

33.1%

ITT = intention-to-treat population; mos = months; CI = confidence interval, KM= Kaplan Meier method

HARMONi-2 Subgroup Analyses

Descriptive, not formally powered

Ivonescimab vs. Pembrolizumab

PD-L1 High (PD-L1 Score ≥50%)

HR = 0.58 (95% CI: 0.38, 0.89)

n=168

Median OS: NR vs. 23.2 mos

PD-L1 Low (PD-L1 Score 1-49%)

HR = 0.85 (95% CI: 0.61, 1.18)

n=230

Median OS: 28.5 mos vs. 22.1 mos

Squamous Histology

HR = 0.65 (95% CI: 0.45, 0.95)

n=181

Median OS: 30.5 mos vs. 19.3 mos

Non-Squamous Histology

HR = 0.79 (95% CI: 0.55, 1.14)

n=217

Median OS: 33.6 mos vs. 25.6 mos

Age <65

HR = 0.75 (95% CI: 0.51, 1.11)

n=182

Median OS: 32.8 mos vs. 25.0 mos

Age ≥65

HR = 0.72 (95% CI: 0.51, 1.01)

n=216

Median OS: 30.2 mos vs. 22.1 mos

Male

HR = 0.74 (95% CI: 0.56, 0.98)

n=333

Female

HR = 0.69 (95% CI: 0.38, 1.25)

n=65

NR = not reached; mos = months; CI=confidence interval; n = number

In this analysis, ivonescimab continued to demonstrate an acceptable and manageable safety profile in the HARMONi-2 study, which was consistent with previous Phase III studies of ivonescimab. No additional safety signals were noted in the HARMONi-2 study in this current data cut with longer treatment duration (median of 14 cycles of ivonescimab vs 10 cycles of pembrolizumab) compared to the previous data cut.

Treatment-Related Adverse Events

Median follow-up: 36.0 mos

Ivonescimab (n=198)

Pembrolizumab (n=200)

Serious TRAEs, n (%)

59 (29.9)

43 (21.6)

TRAEs Leading to Discontinuation, n (%)

8 (4.1)

10 (5.0)

TRAEs Leading to Death, n (%)

1 (0.5)

3 (1.5)

TRAEs = treatment-related adverse events; n = number; mos = months

About Ivonescimab

Ivonescimab, known as SMT112 in Summit’s license territories, North America, South America, Europe, the Middle East, Africa, and Japan, and as AK112 outside of Summit’s license territories, is a novel, potential first-in-class investigational bispecific antibody combining the effects of immunotherapy via a blockade of PD-1 with the anti-angiogenesis effects associated with blocking VEGF into a single molecule. By design, ivonescimab displays unique cooperative binding to each of its intended targets with multifold higher affinity to PD-1 when in the presence of VEGF.

This design is intended to differentiate ivonescimab as there is potentially higher expression (presence) of both PD-1 and VEGF in tumor tissue and the tumor microenvironment (TME) as compared to normal tissue in the body. Summit believes ivonescimab’s specifically engineered tetravalent structure (four binding sites) enables higher avidity (accumulated strength of multiple binding interactions) in the TME (Zhong, et al, iScience, 2025). This tetravalent structure, the intentional novel design of the molecule, and bringing these two targets into a single bispecific antibody with cooperative binding qualities have the potential to direct ivonescimab to the tumor tissue versus healthy tissue. The intent of ivonescimab’s design, together with a half-life of 6 to 7 days after the first dose (Zhong, et al, iScience, 2025) and increasing to approximately 10 days at steady state dosing, is to improve upon previously established efficacy thresholds, side effects, and safety profiles associated with prior approved drugs to these targets.

Ivonescimab was engineered by Akeso Inc. (HKEX Code: 9926.HK) and is currently utilized in multiple Phase III clinical trials. Over 4,000 patients have been treated with ivonescimab in clinical studies globally and over 70,000 patients when considering those treated in a commercial setting in China, as noted by Akeso.

There are currently 16 Phase III clinical studies that are either announced, ongoing, or have been completed studying ivonescimab, five of which are Summit-sponsored global studies, one of which is a multiregional study sponsored by a cooperative group, and 10 of which are being or have been conducted in China by Akeso. Summit began its clinical development of ivonescimab in NSCLC, commencing enrollment in 2023 in two multiregional Phase III clinical trials, HARMONi and HARMONi-3. In 2025, Summit began enrolling patients in HARMONi-7. Summit expanded its Phase III clinical development program into colorectal cancer (CRC) in the fourth quarter of 2025 by initiating enrollment in HARMONi-GI3. In 2026, Summit announced initiation of HARMONi-GU1, a Phase II/III study in urothelial carcinoma (bladder cancer) with global clinical trial site activations planned to begin by the fourth quarter of 2026.

HARMONi is a Phase III clinical trial evaluating ivonescimab combined with chemotherapy compared to placebo plus chemotherapy in patients with EGFR-mutated, locally advanced or metastatic non-squamous NSCLC who were previously treated with a third-generation EGFR TKI (e.g., osimertinib). Detailed results of the study were provided in September 2025, and a Biologics License Application (BLA) was submitted to the United States Food and Drug Administration (FDA) for marketing authorization, which the FDA accepted for filing in January 2026; the goal Prescription Drug User Fee Act (PDUFA) date is November 14, 2026.

HARMONi-3 is a Phase III clinical trial evaluating ivonescimab combined with chemotherapy compared to pembrolizumab combined with chemotherapy in patients with first-line metastatic, squamous or non-squamous NSCLC, irrespective of PD-L1 expression. The clinical trial is evaluating the two histologies as individual, separately powered cohorts with independent statistical powering.

HARMONi-7 is a Phase III clinical trial evaluating ivonescimab monotherapy compared to pembrolizumab monotherapy in patients with first-line metastatic NSCLC whose tumors have high PD-L1 expression.

HARMONi-GI3 is a Phase III clinical trial evaluating ivonescimab in combination with chemotherapy compared with bevacizumab plus chemotherapy in patients with first-line unresectable metastatic CRC.

HARMONi-GU1 is a Phase II/III clinical trial evaluating ivonescimab plus the antibody drug conjugate (ADC) enfortumab vedotin (EV) compared to pembrolizumab plus EV as first-line therapy in patients with previously untreated locally advanced or metastatic urothelial carcinoma (la/mUC).

ILLUMINE is a Phase III study being conducted by GORTEC, a cooperative group dedicated to Head and Neck Oncology, in recurrent / metastatic head and neck squamous cell carcinoma (r/m HNSCC). ILLUMINE is a three-arm Phase III clinical trial designed to evaluate ivonescimab monotherapy, as well as ivonescimab in combination with ligufalimab, Akeso’s proprietary anti-CD47 monoclonal antibody, compared to monotherapy pembrolizumab in patients with PD-L1 positive r/m HNSCC.

Five Phase III ivonescimab clinical trials have read out to date, all five with positive data. Four of these five studies are in NSCLC, and one is in biliary tract cancer (BTC). In addition to Summit’s positive HARMONi study, Akeso has had positive read-outs in three single-region (China), randomized Phase III clinical trials, HARMONi-A, HARMONi-2, and HARMONi-6, for ivonescimab in NSCLC, including a statistically significant overall survival benefit in all three studies from China. Akeso has also reported a statistically significant OS benefit in the single-region (China), randomized Phase III HARMONi-GI1 trial in advanced BTC.

HARMONi-A was a Phase III clinical trial which evaluated ivonescimab combined with chemotherapy compared to placebo plus chemotherapy in patients with EGFR-mutated, locally advanced or metastatic non-squamous NSCLC who have progressed after treatment with an EGFR TKI.

HARMONi-2 is a Phase III clinical trial evaluating monotherapy ivonescimab against monotherapy pembrolizumab in patients with locally advanced or metastatic NSCLC whose tumors have positive PD-L1 expression.

HARMONi-6 is a Phase III clinical trial evaluating ivonescimab in combination with platinum-based chemotherapy compared with tislelizumab, an anti-PD-1 antibody, in combination with platinum-based chemotherapy in patients with locally advanced or metastatic squamous NSCLC, irrespective of PD-L1 expression.

HARMONi-GI1 is a Phase III clinical trial evaluating ivonescimab in combination with chemotherapy compared with durvalumab plus chemotherapy as a first-line treatment for patients with advanced BTC.

Akeso is actively conducting additional Phase III clinical studies in settings outside of NSCLC and biliary-tract cancer, including triple-negative breast cancer, head and neck squamous cell carcinoma, small cell lung cancer, colorectal cancer, and pancreatic cancer.

Ivonescimab is an investigational therapy that is not approved by any regulatory authority in Summit’s license territories, including the United States and Europe. Ivonescimab was initially approved for marketing authorization in China in May 2024.

(Press release, Summit Therapeutics, SEP 15, 2026, View Source [SID1234670880])

On September 15, 2026 BostonGene, developer of the leading AI model for tumor and immune biology, reported an expansion of its scientific collaboration with Dana-Farber Cancer Institute to deepen the understanding of the complex biological mechanisms driving BRCA1- and BRCA2-associated breast cancers. The collaboration applies BostonGene’s foundation model of tumor and immune biology and multimodal AI to place individual tumors into a much broader biological context, enabling systematic discovery of the mechanisms that distinguish disease subtypes and potentially drive therapeutic response and resistance.

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Focusing on BRCA1/2-associated ER-positive breast cancer (BRCA ER+), TP53-mutant ER-positive breast cancer (TP53 ER+), and triple-negative breast cancer (TNBC), the initiative investigates why biologically similar-looking cancers behave differently, what drives response and resistance to targeted therapies, and how those insights can directly de-risk pipeline development by informing biomarker strategy and patient selection.

"Patients with hereditary BRCA1 and BRCA2 mutations face unique biological challenges, particularly across distinct disease presentations like ER+ breast cancer and TNBC," said Filipa Lynce, MD, Senior Physician at Dana-Farber Cancer Institute. "By combining detailed genomic and transcriptomic profiling with advanced analytics, this collaboration allows us to uncover critical molecular signatures underlying hereditary breast cancer to pave the way for more tailored, effective therapeutic strategies."

"Unraveling the crosstalk between DNA repair deficiencies and hormone signaling is fundamental to advancing precision oncology," said Nathan Fowler, MD, Chief Medical Officer at BostonGene. "The opportunity is to move beyond describing these tumors by mutation or clinical subtype and understand the biology that actually differentiates them. By placing each patient into a much broader biological context, we can identify mechanisms that may explain disease behavior and ultimately translate those findings into better biomarker, patient-selection and therapeutic strategies."

By uncovering how DNA damage response pathways interact with hormone signaling, the collaboration demonstrates the capabilities of BostonGene’s AI-powered multimodal analytics in establishing high-resolution biological frameworks. The collaboration provides a model for how biologically grounded AI can turn relatively small, deeply characterized patient cohorts into insights that inform drug development, patient selection, and future clinical strategy.

(Press release, BostonGene, SEP 15, 2026, View Source [SID1234670879])

On September 15, 2026 Nuvation Bio Inc. (NYSE: NUVB), a global oncology company focused on tackling some of the toughest challenges in cancer treatment, reported new subgroup analyses from the pivotal TRUST-I and TRUST-II studies evaluating IBTROZI (taletrectinib) in both TKI- naïve and TKI-pretreated patients with advanced ROS1-positive (ROS1+) non-small cell lung cancer (NSCLC). The data, presented at the IASLC 2026 World Conference on Lung Cancer (WCLC) in Seoul, Republic of Korea, demonstrated that IBTROZI delivered consistent efficacy regardless of prior chemotherapy exposure or ROS1 fusion partner, providing additional evidence supporting its use across a broad range of patients with advanced ROS1+ NSCLC.

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"The most important step for a patient with newly diagnosed non-small cell lung cancer is to conduct comprehensive biomarker testing to identify a targetable driver and start the right targeted therapy as early as possible," said Jorge Nieva, M.D., medical oncologist at USC Norris Comprehensive Cancer Center. "Guidelines recommend holding treatment until biomarker testing comes back, which can create pressure to begin chemotherapy first. These analyses offer reassurance that patients who do start chemotherapy before a ROS1 fusion is identified can still respond well once they transition to taletrectinib. We hope this gives clinicians added confidence to move promptly to targeted therapy as soon as biomarker testing confirms a ROS1 fusion."

"We’ve already been impressed by the durable efficacy of IBTROZI, with many responses lasting over four years, and now these new data answer critical questions about its consistency," said David Hung, M.D., Founder, President and Chief Executive Officer of Nuvation Bio. "These analyses show that patients derive a similar, powerful benefit from IBTROZI across multiple subgroups, which we hope gives clinicians even greater confidence in selecting IBTROZI for any patient with advanced ROS1+ NSCLC."

Previously at AACR (Free AACR Whitepaper) 2026, the pooled analysis of all TKI-naïve patients in the TRUST-I and TRUST-II studies presented showed a confirmed objective response rate (ORR) of 89.8% and a median duration of response (DOR) of 49.7 months. At WCLC, one of the new analyses presented in the poster revealed consistent response rates regardless of prior chemotherapy exposure.

Among TKI-naïve patients:

ORR was 90.0% in those with prior chemotherapy (n=30) and 89.8% in those with no prior chemotherapy (n=127).
Median DOR was 48.3 months and 54.3 months, respectively.
While this analysis shows that patients can respond to IBTROZI following prior chemotherapy, treatment guidelines continue to recommend waiting to begin treatment until biomarker results come back and initiating targeted therapy as soon as a ROS1 fusion is confirmed. These findings reinforce the importance of early biomarker testing and prompt transition to targeted therapy.

Additionally, both TKI-naïve and TKI-pretreated patients consistently benefited from IBTROZI regardless of ROS1 fusion partner. A fusion partner is the gene that merges with ROS1, resulting in a hybrid, cancer-driving protein that fuels tumor growth. The most common partner is CD74, which accounts for up to half of all ROS1 fusions in NSCLC.

Among TKI-naïve patients:

ORR was 89.5% in those with CD74 fusions (n=19) and 88.9% in those with non-CD74 fusions (n=18).
Median DOR was comparable between the two groups at 44.8 and 43.3 months, respectively, as was median progression-free survival (PFS) at 46.1 and 44.6 months.
With long-term follow-up, IBTROZI continued to demonstrate a manageable safety profile consistent with previous reports, with no new safety signals identified. The most common adverse events in the overall TRUST-I and TRUST-II pooled safety analyses were increased AST and ALT, diarrhea, nausea and vomiting, which were mostly low grade.

To review the poster, visit the Publications section of Nuvation Bio’s website.

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 central nervous system (CNS) metastases.

About IBTROZI
IBTROZI is an oral, potent, CNS-active, selective, next-generation ROS1-inhibitor therapy. On June 11, 2025, following Priority Review and Breakthrough Therapy designations for both TKI-naive and TKI-pretreated disease, the U.S. Food and Drug Administration (FDA) approved taletrectinib for the treatment of adult patients with locally advanced or metastatic ROS1+ NSCLC. Learn more about taletrectinib in the U.S. at IBTROZI.com.

About the TRUST Clinical Program
The TRUST clinical program comprises three registrational studies evaluating the safety and efficacy of IBTROZI. TRUST-I (NCT04395677) and TRUST-II (NCT04919811) are Phase 2 single-arm studies evaluating IBTROZI for the treatment of adults with advanced ROS1+ NSCLC in China (N=173) and globally (N=189), respectively. The primary endpoint of both studies is confirmed objective response rate (cORR) as assessed by an independent review committee. TRUST-IV (NCT07154706) is a Phase 3 placebo-controlled study evaluating IBTROZI for the adjuvant treatment of adults with resected early-stage ROS1+ NSCLC. The study will enroll approximately 180 patients in the U.S., Canada, Europe, Japan and China. The primary endpoint is disease-free survival as determined by investigator, and the primary completion date is estimated to be in 2030. Nuvation Bio is also sponsoring TRUST-III (NCT06564324), a confirmatory randomized Phase 3 study evaluating IBTROZI versus crizotinib in 194 patients in China with advanced ROS1+ NSCLC who have not previously received ROS1 TKIs.

U.S. Indication
IBTROZI is indicated for the treatment of adult patients with locally advanced or metastatic ROS1+ non-small cell lung cancer (NSCLC).

IMPORTANT SAFETY INFORMATION FOR IBTROZI (taletrectinib)

WARNINGS AND PRECAUTIONS

Hepatotoxicity: Hepatotoxicity, including drug-induced liver injury and fatal adverse reactions, can occur. 88% of patients experienced increased AST, including 10% Grade 3/4. 85% of patients experienced increased ALT, including 13% Grade 3/4. Fatal liver events occurred in 0.6% of patients. Median time to first onset of AST or ALT elevation was 15 days (range: 3 days to 20.8 months).

Increased AST or ALT each led to dose interruption in 7% of patients and dose reduction in 5% and 9% of patients, respectively. Permanent discontinuation was caused by increased AST, ALT, or bilirubin each in 0.3% and by hepatotoxicity in 0.6% of patients.

Concurrent elevations in AST or ALT ≥3 times the ULN and total bilirubin ≥2 times the ULN, with normal alkaline phosphatase, occurred in 0.6% of patients.

Interstitial Lung Disease (ILD)/Pneumonitis: Severe, life-threatening, or fatal ILD or pneumonitis can occur. ILD/pneumonitis occurred in 2.3% of patients, including 1.1% Grade 3/4. One fatal ILD case occurred at the 400 mg daily dose. Median time to first onset of ILD/pneumonitis was 3.8 months (range: 12 days to 11.8 months).

ILD/pneumonitis led to dose interruption in 1.1% of patients, dose reduction in 0.6% of patients, and permanent discontinuation in 0.6% of patients.

QTc Interval Prolongation: QTc interval prolongation can occur, which can increase the risk for ventricular tachyarrhythmias (e.g., torsades de pointes) or sudden death. IBTROZI prolongs the QTc interval in a concentration-dependent manner.

In patients who received IBTROZI and underwent at least one post baseline ECG, QTcF increase of >60 msec compared to baseline and QTcF >500 msec occurred in 13% and 2.6% of patients, respectively. 3.4% of patients experienced Grade ≥3. Median time from first dose of IBTROZI to onset of ECG QT prolongation was 22 days (range: 1 day to 38.7 months). Dose interruption and dose reduction each occurred in 2.8% of patients.

Significant QTc interval prolongation may occur when IBTROZI is taken with food, strong and moderate CYP3A inhibitors, and/or drugs with a known potential to prolong QTc. Administer IBTROZI on an empty stomach. Avoid concomitant use with strong and moderate CYP3A inhibitors and/or drugs with a known potential to prolong QTc.

Hyperuricemia: Hyperuricemia can occur and was reported in 14% of patients, with 16% of these requiring urate-lowering medication without pre-existing gout or hyperuricemia. 0.3% of patients experienced Grade ≥3. Median time to first onset was 2.1 months (range: 7 days to 35.8 months). Dose interruption occurred in 0.3% of patients.

Myalgia with Creatine Phosphokinase (CPK) Elevation: Myalgia with or without CPK elevation can occur. Myalgia occurred in 10% of patients. Median time to first onset was 11 days (range: 2 days to 10 months).

Concurrent myalgia with increased CPK within a 7-day time period occurred in 0.9% of patients. Dose interruption occurred in 0.3% of patients with myalgia and concurrent CPK elevation.

Skeletal Fractures: IBTROZI can increase the risk of fractures. ROS1 inhibitors as a class have been associated with skeletal fractures. 3.4% of patients experienced fractures, including 1.4% Grade 3. Some fractures occurred in the setting of a fall or other predisposing factors. Median time to first onset of fracture was 10.7 months (range: 26 days to 29.1 months). Dose interruption occurred in 0.3% of patients.

Embryo-Fetal Toxicity: Based on literature, animal studies, and its mechanism of action, IBTROZI can cause fetal harm when administered to a pregnant woman.

ADVERSE REACTIONS

Among patients who received IBTROZI, the most frequently reported adverse reactions (≥20%) were diarrhea (64%), nausea (47%), vomiting (43%), dizziness (22%), rash (22%), constipation (21%), and fatigue (20%).

The most frequently reported Grade 3/4 laboratory abnormalities (≥5%) were increased ALT (13%), increased AST (10%), decreased neutrophils (5%), and increased creatine phosphokinase (5%).

DRUG INTERACTIONS

Strong and Moderate CYP3A Inhibitors/CYP3A Inducers and Drugs that Prolong the QTc Interval: Avoid concomitant use.
Gastric Acid Reducing Agents: Avoid concomitant use with PPIs and H2 receptor antagonists. If an acid-reducing agent cannot be avoided, administer locally acting antacids at least 2 hours before or 2 hours after taking IBTROZI.
OTHER CONSIDERATIONS

Pregnancy: Please see important information in Warnings and Precautions under Embryo-Fetal Toxicity.
Lactation: Advise women not to breastfeed during treatment and for 3 weeks after the last dose.
Effect on Fertility: Based on findings in animals, IBTROZI may impair fertility in males and females. The effects on animal fertility were reversible.
Pediatric Use: The safety and effectiveness of IBTROZI in pediatric patients has not been established.
Photosensitivity: IBTROZI can cause photosensitivity. Advise patients to minimize sun exposure and to use sun protection, including broad-spectrum sunscreen, during treatment and for at least 5 days after discontinuation.

(Press release, Nuvation Bio, SEP 15, 2026, View Source [SID1234670878])