On October 23, 2025 Partner Therapeutics, Inc. (PTx), a private, fully-integrated biotechnology company, reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation (BTD) to zenocutuzumab-zbco for the treatment of adults with advanced unresectable or metastatic cholangiocarcinoma harboring a neuregulin 1 (NRG1) gene fusion. The designation was supported by results from the ongoing investigational Phase 2 eNRGy trial. These findings will be presented as both an oral and poster presentation at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) in Boston, MA and will serve as the basis for a supplemental Biologics License Application (sBLA) that will be submitted to FDA in 2026.

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BTD is granted by the FDA to expedite the development and review of therapies that may demonstrate substantial improvement over existing treatments for serious or life-threatening conditions. The designation conveys all of the fast track program features, more intensive FDA guidance on an efficient drug development program, an organizational commitment, and eligibility for rolling review and priority review. For adult patients with advanced unresectable or metastatic NRG1+ cholangiocarcinoma, a rare and aggressive cancer with limited treatment options, this designation underscores the urgent need for effective therapies and recognizes the potential of zenocutuzumab-zbco to help address this significant unmet medical need.

The designation was supported by new interim data presented at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) meeting which evaluated zenocutuzumab-zbco in patients with advanced NRG1+ cholangiocarcinoma in the eNRGy trial. In 19 evaluable patients, the results demonstrated an investigator-assessed overall response rate of 37%, a median duration of response of 7.4 months, and a median time to response of 1.9 months. Progression-free survival was 9.2 months and clinical benefit rate, defined as partial/complete response or stable disease for ≥24 weeks, was 58%. Among patients with evaluable CA 19-9 data, all experienced a decline in serum levels, including a >50% reduction in 69% of patients. The safety profile was consistent with the overall eNRGy trial population, with most adverse events grade 1 or 2. Five patients (23%) experienced serious adverse events, none considered treatment related, and no patients discontinued therapy due to treatment-related toxicity.

Dr. Alison M. Schram, Principal Investigator of the eNRGy trial and medical oncologist from Memorial Sloan Kettering Cancer Center, commented: "NRG1 fusions represent a rare but actionable driver in cholangiocarcinoma, and the data from the eNRGy trial continue to highlight the potential of zenocutuzumab to offer meaningful clinical benefit for these patients. I’m honored to present these findings at AACR (Free AACR Whitepaper)-NCI-EORTC, where we can advance the dialogue around targeted therapies in hard-to-treat cancers."

"Patients with cholangiocarcinoma face an aggressive disease with limited standard therapy options," said Juan W. Valle, MD, Chief Medical Officer of the Cholangiocarcinoma Foundation. "The eNRGy trial results are encouraging, and they underscore how critical comprehensive molecular testing, notably tissue-based RNA NGS, is to ensure that patients with rare drivers such as NRG1 fusions are identified and can potentially have access to targeted treatments."

"In December 2024, zenocutuzumab-zbco (tradename BIZENGRI) received accelerated approval for the treatment of adults with advanced unresectable or metastatic non-small cell lung cancer (NSCLC) and pancreatic adenocarcinoma harboring a neuregulin 1 (NRG1) gene fusion with disease progression on or after prior systemic therapy. The new data from the eNRGy trial highlight the potential of zenocutuzumab-zbco as a promising treatment option for patients with NRG1 fusion–positive cholangiocarcinoma," said Pritesh J. Gandhi, PharmD, Chief Development Officer of Partner Therapeutics. "With the growing number of genomic alternations and gene fusions that are now actionable, it is imperative that oncologists order upfront tissue-based RNA next generation sequencing to ensure that gene fusions, of which NRG1 is just one, are not missed."

A copy of the poster and presentation will be available on the Partner Therapeutics website under the research tab following the conference. Additional meeting information can be found on the AACR (Free AACR Whitepaper)-NCI-EORTC website.

These indications are approved under accelerated approval based on overall response rate and duration of response. Continued approval for these indications may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

For more information on the eNRGy trial and zenocutuzumab-zbco, please visit www.partnertx.com.

Dr. Schram has financial interests related to Partner Therapeutics.

About NRG1 Gene Fusions

NRG1 fusions are unique cancer drivers that create oncogenic chimeric ligands rather than the more widely described chimeric receptors (NTRK, RET, ROS1, ALK, and FGFR fusions). The chimeric ligands bind to HER3, triggering HER2/HER3 heterodimerization and activate downstream signaling pathways that cause cancer cells to grow and proliferate. Zenocutuzumab-zbco is a bispecific antibody that blocks HER2/HER3 dimerization and NRG1 fusion interactions with HER3, resulting in the suppression of these pathways. Comprehensive molecular testing, notably tissue-based RNA next generation sequencing, is essential to identify rare and actionable gene fusions like NRG1.

About BIZENGRI (zenocutuzumab-zbco)

INDICATIONS

BIZENGRI is indicated for the treatment of adults with advanced unresectable or metastatic non-small cell lung cancer (NSCLC) harboring a neuregulin 1 (NRG1) gene fusion with disease progression on or after prior systemic therapy.

BIZENGRI is indicated for the treatment of adults with advanced unresectable or metastatic pancreatic adenocarcinoma harboring a neuregulin 1 (NRG1) gene fusion with disease progression on or after prior systemic therapy.

These indications are approved under accelerated approval based on overall response rate and duration of response. Continued approval for these indications may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Important Safety Information

BOXED WARNING: EMBRYO-FETAL TOXICITY

Embryo-Fetal Toxicity: Exposure to BIZENGRI during pregnancy can cause embryo-fetal harm. Advise patients of this risk and the need for effective contraception.

WARNINGS AND PRECUATIONS

Infusion-Related Reactions/Hypersensitivity/Anaphylactic Reactions

BIZENGRI can cause serious and life-threatening infusion-related reactions (IRRs), hypersensitivity and anaphylactic reactions. Signs and symptoms of IRR may include chills, nausea, fever, and cough.

In the eNRGy study, 13% of patients experienced IRRs, all were Grade 1 or 2; 91% occurred during the first infusion.

Administer BIZENGRI in a setting with emergency resuscitation equipment and staff who are trained to monitor for IRRs and to administer emergency medications. Monitor patients closely for signs and symptoms of infusion reactions during infusion and for at least 1 hour following completion of first BIZENGRI infusion and as clinically indicated. Interrupt BIZENGRI infusion in patients with ≤ Grade 3 IRRs and administer symptomatic treatment as needed. Resume infusion at a reduced rate after resolution of symptoms. Immediately stop the infusion and permanently discontinue BIZENGRI for Grade 4 or life-threatening IRR or hypersensitivity/anaphylaxis reactions.

Interstitial Lung Disease/Pneumonitis

BIZENGRI can cause serious and life-threatening interstitial lung disease (ILD)/pneumonitis.

In the eNRGy study, ILD/pneumonitis occurred in 2 (1.1%) patients treated with BIZENGRI. Grade 2 ILD/pneumonitis (Grade 2) resulting in permanent discontinuation of BIZENGRI occurred in 1 (0.6%) patient. Monitor for new or worsening pulmonary symptoms indicative of ILD/pneumonitis (e.g., dyspnea, cough, fever). Immediately withhold BIZENGRI in patients with suspected ILD/pneumonitis and administer corticosteroids as clinically indicated.

Permanently discontinue BIZENGRI if ILD/pneumonitis ≥ Grade 2 is confirmed.

Left Ventricular Dysfunction

BIZENGRI can cause left ventricular dysfunction.

Left ventricular ejection fraction (LVEF) decrease has been observed with anti-HER2 therapies, including BIZENGRI. Treatment with BIZENGRI has not been studied in patients with a history of clinically significant cardiac disease or LVEF less than 50% prior to initiation of treatment.

In the eNRGy study, Grade 2 LVEF decrease (40%-50%; 10 – 19% drop from baseline) occurred in 2% of evaluable patients. Cardiac failure without LVEF decrease occurred in 1.7% of patients, including 1 (0.6%) fatal event.

Before initiating BIZENGRI, evaluate LVEF and monitor at regular intervals during treatment as clinically indicated. For LVEF of less than 45% or less than 50% with absolute decrease from baseline of 10% or greater which is confirmed, or in patients with symptomatic congestive heart failure (CHF), permanently discontinue BIZENGRI.

Embryo-Fetal Toxicity

Based on its mechanism of action, BIZENGRI can cause fetal harm when administered to a pregnant woman. No animal reproduction studies were conducted with BIZENGRI. In post marketing reports, use of a HER2-directed antibody during pregnancy resulted in cases of oligohydramnios manifesting as fatal pulmonary hypoplasia, skeletal abnormalities, and neonatal death. In animal models, studies have demonstrated that inhibition of HER2 and/or HER3 results in impaired embryo-fetal development, including effects on cardiac, vascular and neuronal development, and embryolethality. Advise patients of the potential risk to a fetus. Verify the pregnancy status of females of reproductive potential prior to the initiation of BIZENGRI. Advise females of reproductive potential to use effective contraception during treatment with BIZENGRI and for 2 months after the last dose.

ADVERSE REACTIONS

NRG1 Gene Fusion Positive Unresectable or Metastatic NSCLC

Serious adverse reactions occurred in 25% of patients with NRG1 gene fusion positive NSCLC who received BIZENGRI. Serious adverse reactions in ≥ 2% of patients included pneumonia (n=4) dyspnea and fatigue (n=2 each). Fatal adverse reactions occurred in 3 (3%) patients and included respiratory failure (n=2), and cardiac failure (n=1). Permanent discontinuation of BIZENGRI due to an adverse reaction occurred in 3% of patients. Adverse reactions resulting in permanent discontinuation of BIZENGRI included dyspnea, pneumonitis and sepsis (n=1 each).

In patients with NRG1 gene fusion positive NSCLC who received BIZENGRI, the most common (>20%) adverse reactions, including laboratory abnormalities, were decreased hemoglobin (35%), increased alanine aminotransferase (30%), decreased magnesium (28%), increased alkaline phosphatase (27), decreased phosphate (26%), diarrhea (25%), musculoskeletal pain (23%), increased gamma-glutamyl transpeptidase (23%), increased aspartate aminotransferase (22%), and decreased potassium (21%).

NRG1 Gene Fusion Positive Unresectable or Metastatic Pancreatic Adenocarcinoma

Serious adverse reactions occurred in 23% of patients with NRG1 gene fusion positive pancreatic adenocarcinoma who received BIZENGRI.

There were 2 fatal adverse reactions, one due to COVID-19 and one due to respiratory failure.

In patients with NRG1 gene fusion positive pancreatic adenocarcinoma who received BIZENGRI the most common (≥20%) adverse reactions, including laboratory abnormalities, were increased alanine aminotransferase (51%), diarrhea (36%), increased aspartate aminotransferase (31%), increased bilirubin (31%), decreased phosphate (31%), increased alkaline phosphatase (28%), decreased sodium (28%), musculoskeletal pain (28%), decreased albumin (26%), decreased potassium (26%), decreased platelets (26%), decreased magnesium (24%), increased gamma-glutamyl transpeptidase (23%), decreased hemoglobin (23%), vomiting (23%), nausea (23%), decreased leukocytes (21%), and fatigue (21%).

Please see full Prescribing Information, including Boxed Warning

(Press release, Partner Therapeutics, OCT 23, 2025, View Source [SID1234656963])

On October 23, 2025 Jacobio Pharma (1167.HK) reported that it presented the pre-clinical data of its internally discovered pan-KRAS inhibitor JAB-23E73 in a poster presentation at the 2025 AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper).

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The pre-clinical results demonstrated that JAB-23E73 is a highly potent pan-KRAS (ON/OFF) inhibitor with strong selectivity that spares HRAS and NRAS inhibition. The compound exhibits superior antitumor activity across multiple cancer types harboring different KRAS driver mutations or amplification.

In KRAS-driven mouse tumor models, JAB-23E73 induced tumor regression without causing significant body weight loss, indicating good tolerability and a wide therapeutic window. The compound also showed a favorable pharmacokinetic profile for oral administration and exhibited plasma drug concentration-dependent intratumoral p-ERK inhibition.

Phase I clinical trials of JAB-23E73 are currently ongoing in both China and the United States for patients with advanced solid tumors harboring KRAS gene alterations.

(Press release, Jacobio Pharmaceuticals, OCT 23, 2025, View Source [SID1234656962])

On October 23, 2025 HanchorBio Inc. (TPEx: 7827), a global clinical-stage biotechnology company developing next-generation immunotherapies, reported that its manuscript describing the discovery and preclinical development of HCB101, an engineered SIRPα-Fc fusion protein, has been published in the Journal of Hematology & Oncology (SCI Impact Factor 40.4; for reference, leading journals in the field of immuno-oncology include Journal of Clinical Oncology (impact factor 41.9), The Lancet Oncology (35.9), and Nature Cancer (28.5)).

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The publication, titled "HCB101: A Novel Potent Ligand-Trap Fc-fusion Protein Targeting the CD47-SIRPα Pathway with High Safety and Preclinical Efficacy for Hematological and Solid Tumors," describes the rational protein engineering that enabled HCB101 to restore macrophage-mediated phagocytosis and bridge innate and adaptive immune responses while reducing red blood cell binding. In preclinical studies, HCB101 showed broad activity across more than 80 xenograft and PDX tumor animal models with a safety profile that differs from first- and second-generation CD47-targeting agents.

Notably, a US competitor focusing on CD47/SIRPα published findings in JHO in November 2020, reporting on a third-generation anti-CD47–SIRPα therapy. This underscores the journal’s strong recognition within the field. The continued publication of anti-CD47 biologics in JHO highlights both the scientific importance of ongoing advancements and the journal’s role as a leading international platform for preclinical and translational research on anti-CD47 therapies.

"Publication in the Journal of Hematology & Oncology affirms the scientific rigor and innovation behind HCB101 and emphasizes its differentiated preclinical foundation," said Scott Liu, PhD, Chairman, CEO, and Founder of HanchorBio. "Importantly, these insights are being directly translated into clinical studies: combined with standard-of-care, HCB101 has demonstrated a clear dose-dependent efficacy profile in 2L gastric cancer, culminating in a nearly 90% partial response rate at 5.12 and 8.0 mg/kg. In parallel, we have now escalated monotherapy dosing to 30 mg/kg without safety concerns. These milestones highlight HCB101’s potential to serve as a backbone immunotherapy across solid tumors and hematologic malignancies and lay the groundwork for expanding into autoimmune indications where CD47-SIRPα biology is also potentially useful in creating a new B-cell depletion therapy."

Clinical Progress Strengthens Differentiated Best-in-Class Profile
HCB101 is currently being evaluated in multinational Phase 1 and Phase 1b/2a trials:

Monotherapy (HCB101-101, NCT05892718):
The Safety Review Committee (SRC) has reviewed the safety data up to 30 mg/kg weekly and found no dose-limiting toxicities, confirming a wide therapeutic window. Early signs of efficacy included two confirmed partial responses (PRs) in head and neck squamous cell carcinoma and marginal zone lymphoma, along with stable disease (SD) in nine patients, including over 40 weeks of disease control in platinum-resistant ovarian cancer.
Combination (HCB101-201, NCT06771622):
In the triplet regimen for 2L-gastric cancer (HCB101 + ramucirumab + paclitaxel), emerging activity was first observed at 2.56 mg/kg, where evaluable patients achieved SD with modest tumor shrinkage, averaging a 6% reduction in tumor size in the dose cohort. At 5.12 mg/kg, all three evaluable patients achieved confirmed PRs (33%–46% tumor reductions). At 8.0 mg/kg, all three evaluable patients have now achieved confirmed PRs (tumor shrinkage up to -78%) after extended follow-up. Together, these results represent a nearly 90% confirmed response rate (6 of 7 patients) at ≥ 5.12 mg/kg dose level — a striking outcome in a setting where the typical overall response rate (ORR) for the SOC is only 26.5%.
"The data now published in JHO validate the design strategy that made HCB101 possible – balancing high efficacy with a clean safety margin where earlier CD47-targeting agents failed," added Wenwu Zhai, PhD, Chief Scientific Officer of HanchorBio. "Seeing those preclinical insights translate into durable monotherapy activity and remarkable combination dose-dependent responses in gastric cancer provides confidence not only for oncology but also for new areas such as autoimmunity. HCB101’s differentiated mechanism and safety profile open the door to rational combinations and next-generation applications well beyond cancer."

About HCB101: A Differentiated CD47-SIPRα Blockade
HCB101 is a 3.5th-generation, affinity-optimized SIRPα-Fc fusion protein with intact IgG4 effector function, developed using HanchorBio’s proprietary FBDB platform. Engineered for selective CD47 blinding on tumors with low affinity for red blood cells, HCB101 avoids the hematologic toxicities commonly associated with anti-CD47 monoclonal antibodies, while preserving strong antibody-dependent cellular phagocytosis (ADCP) and innate-to-adaptive immune bridging.

Key Differentiators of HCB101:

Enhanced safety: Low RBC binding minimizes anemia and thrombocytopenia risk.
Robust immune activation: Engineered to enhance ADCP and innate-to-adaptive bridging.
Broad tumor applicability: Demonstrated activity in >80 PDX/CDX preclinical models.
Clinical translation: Early efficacy as monotherapy with durable disease control, and 100% ORR and disease control rate (DCR) at the middle dose cohort in combination with standard-of-care for 2L-gastric cancer.

(Press release, Hanchor Bio, OCT 23, 2025, View Source;oncology-302592241.html [SID1234656961])

On October 23, 2025 BridGene Biosciences, Inc., a leader in the discovery of small molecule drugs for traditionally "hard-to-drug" targets, announced today that three abstracts have been accepted for presentation at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper), taking place October 22-26, 2025, in Boston, Massachusetts. The presentations showcase new discoveries from BridGene’s proprietary IMTAC (Isobaric Mass Tagged Affinity Characterization) chemoproteomics platform, including the identification of a novel covalent FGFR3 inhibitor, a first-in-class PAX8 inhibitor, and data illustrating the breadth and precision of IMTAC in mapping covalent ligandable sites across the proteome.

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BridGene will present findings from its study titled "Discovery of a Highly Potent and Selective Covalent FGFR3 Inhibitor," which details the identification and preclinical evaluation of a selective FGFR3 inhibitor discovered using the company’s IMTAC chemoproteomics platform. The compound demonstrated irreversible binding to a previously uncharacterized cysteine residue in FGFR3, resulting in sustained inhibition of FGFR3 phosphorylation and downstream ERK signaling. In cellular models harboring FGFR3 alterations, the inhibitor exhibited nanomolar potency and strong antiproliferative effects, while maintaining high selectivity over other FGFR family members. These data support the potential of covalent FGFR3 inhibition as a novel therapeutic strategy for FGFR3-driven cancers and further validate BridGene’s chemoproteomic approach for uncovering druggable sites in challenging targets.

BridGene will present findings from its study titled "IMTAC: A Proteome-Wide Live-Cell Screening Platform for Discovering Covalent Binders to Diverse Targets Including GPCRs, Phosphatases, and More." The research showcases how BridGene’s proprietary IMTAC platform integrates a highly diverse covalent small molecule library with advanced live-cell chemical proteomics and quantitative mass spectrometry to identify direct, on-target binding events under native cellular conditions. The platform enables proteome-wide selectivity profiling, minimizes false positives, and reveals transient, novel binding pockets that are undetectable in traditional assays.

Using IMTAC, BridGene identified covalent binders across multiple challenging target classes, including GPCRs, phosphatases, and kinases. In GPCR studies, BGP-1951 inhibited serotonin-induced calcium influx through 5-HT₂A while avoiding common CNS toxicity profiles, and BGP-2992 acted as a positive allosteric modulator of CXCR4 signaling with therapeutic potential in neuroinflammation. Among phosphatases, BGP-15341 inhibited ENPP1 enzymatic activity at nanomolar potency, and BGP-1900 selectively bound PTPN2, offering a path toward targeted degradation strategies. In kinase assays, BGP-21172 showed strong selectivity for CDK7, and two ADK binders—BGP-1892 and BGP-13486—demonstrated nanomolar activity in target engagement assays.

BridGene will present findings from "Discovery of a Covalent Inhibitor Targeting PAX8-Driven Ovarian Cancer" highlighting the identification of BGP-31609, a covalent small molecule inhibitor discovered through the IMTAC platform. BGP-31609 binds irreversibly to a single cysteine residue within the DNA-binding domain of PAX8, disrupting transcriptional activity and reducing the expression of downstream oncogenic targets including FGF18 and CCNA2. In biochemical and cellular assays, the compound showed dose-dependent inhibition of DNA binding in EMSA and selective suppression of PAX8-driven luciferase activity with minimal off-target effects. Importantly, BGP-31609 inhibited proliferation of PAX8-high OVCAR3 ovarian cancer cells while sparing PAX8-negative A549 cells, demonstrating target selectivity. These results establish BGP-31609 as a validated covalent binder to an historically undruggable transcription factor and a promising lead for treating PAX8-dependent malignancies.

"We’re excited to share these new discoveries that further validate the strength and versatility of our IMTAC chemoproteomics platform," stated Ping Cao, Ph.D., CEO and co-founder of BridGene Biosciences. "The data we’re presenting at AACR (Free AACR Whitepaper)-NCI-EORTC illustrate how IMTAC can uncover previously hidden binding sites and enable the development of covalent inhibitors against some of the most challenging oncology targets. These findings reflect our commitment to expanding what’s possible in small molecule drug discovery and to translating this science into meaningful therapies for patients."

The three poster presentations will be available on the AACR (Free AACR Whitepaper)-NCI-EORTC conference website following the sessions. BridGene’s scientific team will be available to discuss the data and the continued advancement of the company’s IMTAC platform in driving discovery of covalent small molecule drugs for previously undruggable targets.

Abstract Title:

Discovery of a Highly Potent and Selective Covalent FGFR3 Inhibitor

Session:

Poster Session B

Date and Time:

Friday, October 24, 12:30-4pm

Location:

Hynes Convention Center, Boston – Level 2, Exhibit Hall D

Abstract Title:

IMTAC: A Proteome-Wide Live-Cell Screening Platform for Discovering Covalent Binders to Diverse Targets Including GPCRs, Phosphatases, and More

Session:

Poster Session C

Date and Time:

Saturday, October 25, 12:30-4pm

Location:

Hynes Convention Center, Boston – Level 2, Exhibit Hall D

Abstract Title:

Discovery of a Covalent Inhibitor Targeting PAX8-Driven Ovarian Cancer

Session:

Poster Session B

Date and Time:

Friday, October 24, 12:30-4pm

Location:

Hynes Convention Center, Boston – Level 2, Exhibit Hall D

On October 23, 2025 Inhibrx Biosciences, Inc. (Nasdaq: INBX) ("Inhibrx" or the "Company"), a clinical-stage biopharmaceutical company focused on developing therapeutics for oncology and rare diseases, reported positive topline results from the registrational ChonDRAgon study (n= 206) investigating ozekibart (INBRX-109) as a single agent versus placebo in patients with advanced or metastatic, unresectable chondrosarcoma. The Company also provided updates on the ongoing expansion cohorts investigating ozekibart in combination with FOLFIRI in late-line colorectal cancer and in combination with irinotecan and temozolomide in refractory Ewing sarcoma.

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Chondrosarcoma

The ChonDRAgon study met its primary endpoint of a statistically significant and clinically meaningful median progression-free survival (PFS) for patients with advanced or metastatic chondrosarcoma treated with ozekibart compared to placebo. Ozekibart achieved a 52% reduction in the risk of disease progression or death compared to placebo (stratified Hazard Ratio [HR] 0.479; 95% CI: 0.33, 0.68); P<0.0001), more than doubling median PFS to 5.52 months versus 2.66 months for placebo. Importantly, ozekibart is the first investigational therapy to demonstrate a significant PFS benefit in a randomized trial for chondrosarcoma, a disease with no approved systemic options.

The benefit of ozekibart was consistent across all pre-specified subgroups, including patients with IDH-wild-type and IDH-mutant tumors. Other key secondary endpoints, including disease control rate (54% vs 27.5%), and delay to deterioration in pain and physical function, further supported the clinical benefit observed with ozekibart.

Ozekibart was generally well tolerated, with a manageable safety profile. The most common treatment-related adverse events were fatigue, constipation, and nausea. Hepatotoxicity, a known risk for this mechanism of action, occurs during the first treatment cycle and is in patients with underlying hepatic impairment. One hepatotoxicity-related fatal event occurred early in the study, prior to the implementation of mitigation measures. Over the course of the ChonDRAgon study, this risk was effectively mitigated by excluding patients with severe liver impairment and by implementing close monitoring during early treatment cycles, allowing for prompt management of liver enzyme elevations. This approach resulted in a low overall incidence of treatment-related hepatic adverse events, 11.8% compared to 4.5% in the placebo arm, the majority of which were Grade 1 or 2 in severity.

"I am very encouraged and enthusiastic about ozekibart and the impact I have seen on my sarcoma patients," said Dr. Robin Jones, head of the sarcoma unit at The Royal Marsden Hospital in London, United Kingdom. "With no approved treatments available, we have observed that ozekibart helps to keep the cancer from growing, improves how patients feel, and restores a sense of hope for my patients."

Detailed results from this trial will be presented at the Connective Tissue Oncology Society (CTOS) Annual Meeting on November 14, 2025.

Colorectal Cancer

Based on initial results from the Phase 1 trial of ozekibart in combination with FOLFIRI for the treatment of advanced or metastatic, unresectable colorectal cancer (CRC), Inhibrx initiated an expansion cohort enrolling 44 patients, as a fourth line of therapy for approximately 70% of patients and as a third line of therapy for approximately 30% of patients. 80% of patients had been previously treated with regimens containing irinotecan. Efficacy, based on RECIST v1.1 criteria, was assessed in 26 evaluable patients to date who had at least one post-baseline scan. The results show a 23% overall response rate (ORR) and an overall disease control rate of 92%. These outcomes are highly encouraging in a heavily pretreated CRC population, where responses are rare (5-6%) and outcomes are generally poor with the current standard of care.

Ozekibart, in combination with FOLFIRI, was well tolerated. The most common treatment-emergent adverse events included anemia, diarrhea, nausea, and fatigue, with the majority being low-grade and consistent with the known safety profile of FOLFIRI.

Ewing Sarcoma

Based on initial results from the Phase 1 trial of ozekibart in combination with irinotecan and temozolomide (IRI/TMZ) for advanced or metastatic, unresectable, relapsed, or refractory Ewing sarcoma, Inhibrx initiated an expansion cohort which is expected to enroll up to 50 patients. Of the 33 patients recruited to date, more than half were third or fourth line patients. Among the 25 evaluable patients to date, Inhibrx observed a 64% overall response rate (ORR), and a disease control rate of 92%, with the majority of patients experiencing measurable tumor reduction. These outcomes are highly encouraging in this difficult-to-treat patient population as compared to the response rate typically observed with standard IRI/TMZ (15-30%).

Overall, ozekibart in combination with IRI/TMZ was well tolerated. The most common adverse events were diarrhea, nausea, anemia, and fatigue, all consistent with the known safety profile of IRI/TMZ.

"We are excited by these results which suggest the potential of ozekibart to expand not only in sarcomas but also in high unmet need solid tumor indications," said Mark Lappe, CEO and Co-Founder of Inhibrx. "We look forward to working with the FDA to deliver ozekibart to patients as swiftly as possible."

The Company will host a live webcast presentation today, October 23rd, 2025, at 1:30 p.m. Pacific Time to further discuss the results.

About the Conference Call

Investors may join via the web: View Source or may listen to the call by dialing (1-888-880-3330). Please refer to Inhibrx Biosciences, Inc. or the conference ID 9577647 when calling in. Following the webcast, the presentation may be accessed through a link on the investors section of Inhibrx’s website at View Source The webcast will be available for 60 days following the event. Following the presentation, Inhibrx will update its corporate presentation within the "Investors" section of its website at www.inhibrx.com.

About ozekibart (INBRX-109)

Ozekibart is a precision-engineered, tetravalent death receptor 5 (DR5) agonist antibody designed to exploit the tumor-biased cell death induced by DR5 activation. In January 2021, the FDA granted Fast Track designation to ozekibart for the treatment of patients with metastatic or unresectable conventional chondrosarcoma, and, in November 2021, the FDA granted orphan drug designation to ozekibart for chondrosarcoma.

In June 2021, Inhibrx initiated a randomized, blinded, placebo-controlled, registrational trial of ozekibart in metastatic, unresectable conventional chondrosarcoma. The trial enrolled a total of 206 patients across 67 different sites worldwide. The primary objective of the trial was the evaluation of the efficacy of ozekibart as measured by median PFS, assessed by central real-time independent radiology review per RECIST 1.1. Secondary objectives were the evaluation of overall survival, median PFS by investigator assessment, quality of life, objective response rate, duration of response, disease control rate, safety and tolerability, pharmacokinetics and anti-drug antibodies to ozekibart.

Key enrollment criteria in order for patients to qualify for inclusion in the trial were grade 2 or 3 unresectable or metastatic conventional chondrosarcoma. Patients received either ozekibart or placebo every three weeks at a randomization of 2:1, stratified by the line of therapy, grade and IDH1/2 mutation status.

Patients randomized to the placebo arm were allowed to crossover to receive ozekibart upon confirmation of progression as reported by central independent radiology review.

In addition to the registrational trial, Inhibrx is advancing ongoing expansion cohorts, evaluating ozekibart in combination with irinotecan-based regimens in Ewing sarcoma and colorectal cancer. Encouraging early signals support further exploration of ozekibart’s potential in these difficult-to-treat tumor types with high unmet medical need.

(Press release, Inhibrx, OCT 23, 2025, View Source [SID1234656959])