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SAN DIEGO--( BUSINESS WIRE )-- Onchilles Pharma , a private biotech company pioneering pan-cancer therapeutics that leverage the ELANE pathway, today announced the presentation of new preclinical data from its NEU-002 program at the 39th Annual Meeting of the Society for Immunotherapy of Cancer (SITC). The event is being held virtually and at the George R. Brown Convention Center in Houston, Texas from November 6-10, 2024. The ELANE pathway is a novel innate immune mechanism that enables potent and selective immunogenic cell death of cancer cells irrespective of immunotype, genotype, anatomical origin while sparing healthy tissue. Onchilles Pharma has leveraged this pathway to develop N17350 and NEU-002, two leading therapeutic candidates designed for intratumoral and intravenous (IV) administration, respectively. N17350 is on track to start Phase 1 clinical trials in 2025 to validate the ELANE pathway in head & neck, skin, breast, and lung cancers. At SITC 2024, Onchilles Pharma presented new preclinical data on NEU-002, which allows targeting the ELANE pathway through systemic delivery. NEU-002 addresses the challenges of systemic delivery by overcoming inhibition from serine protease inhibitors and enhancing substrate specificity, thereby extending its potential to treat a broad range of solid tumor types that are well-suited for systemic administration. Key Preclinical Data Highlights: Enhanced Specificity and Activity : NEU-002 retains full enzymatic activity in plasma and shows improved substrate specificity, effectively cleaving CD95, its therapeutic target, while sparing known off-target substrates. Selective Cancer Cell Killing : The NEU-002 candidates selectively induce immunogenic cell death in primary cancer cells derived from ovarian cancer patients, validating their selective killing properties in primary human tissue. Durable Responses : In preclinical studies using the CT26 tumor mouse model, NEU-002 treatment generated complete responses with resistance to tumor rechallenge, suggesting the potential for immune memory formation. "The data presented today mark a significant advancement in the systemic targeting of the ELANE pathway," said Lev Becker, Ph.D. , Scientific Founder and Chief Scientific Officer of Onchilles Pharma. "The ability to selectively target cancer cells while inducing a sustained immune response could provide durable clinical benefits across a wide range of tumor types." Court R. Turner, J.D. , Co-Founder & Chief Executive Officer, added, "N17350 and NEU-002 represent a transformative approach in oncology, with the potential to redefine the standard of care. We are excited about advancing N17350 into a Phase 1 trial in 2025, providing the clinical proof-of-concept for targeting the ELANE pathway and delivering innovative cancer treatments to patients." About the NEU-002 Program and the ELANE Pathway Onchilles Pharma's NEU-002 program stems from groundbreaking research by its scientific founder, Dr. Lev Becker, who identified the ELANE pathway as a novel innate immune mechanism where neutrophil elastase (ELANE) selectively kills cancer cells while sparing healthy cells. Published in Cell in 2021, these findings have laid the foundation for developing NEU-002, which is tailored for systemic IV delivery, and N17350, designed for intratumoral administration. By leveraging the ELANE pathway, these candidates offer a unique approach to treat cancer regardless of genetic background, anatomical origin, or immunotype, positioning them as potential game-changers in cancer therapy. Looking Ahead: Clinical Validation in 2025 Onchilles Pharma plans to initiate a Phase 1 trial of its lead candidate, N17350, in 2025 to validate the ELANE pathway in humans. This trial will further explore the safety and efficacy of this innovative mechanism, representing a significant step toward realizing the potential of ELANE-based therapies. About Onchilles Pharma Onchilles Pharma is a global drug discovery and development company focused on harnessing the ELANE pathway to create next-generation, pan-cancer therapeutics. These first-in-class medicines hold the potential to revolutionize cancer treatment by selectively targeting tumors, independent of genetic background, anatomical origin, or immune status. The company's pipeline includes biologic drug candidates aimed at treating various cancers, such as head & neck, skin, breast, and lung cancers, with plans to begin first-in-human trials in 2025. For more information, visit www.onchillespharma.com .

SAN MATEO, Calif.--( BUSINESS WIRE )--BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global oncology company, today announced it will share new data across a range of B-cell malignancies and assets, including best-in-class Bruton’s tyrosine kinase (BTK) inhibitor BRUKINSA ® (zanubrutinib), at the 66 th ASH Annual Meeting and Exposition in San Diego, December 7-10. BeiGene has 21 abstracts accepted at ASH 2024, with four selected for oral presentation. “In the five years since its initial approval, BRUKINSA has become a standard of care for patients facing many B-cell malignancies, and our data featured at ASH demonstrated how long-term follow-up of treatment with BRUKINSA elicited deep and durable responses, including in patients with chronic lymphocytic leukemia and Waldenström’s macroglobulinemia,” said Mehrdad Mobasher, M.D., M.P.H., Chief Medical Officer, Hematology at BeiGene. “BRUKINSA is just the starting point – pipeline data for our BTK degrader BGB-16673 and BCL2 inhibitor sonrotoclax showcase our continued leadership across the hematology landscape and our commitment to bringing innovative medicines to as many people with cancer as possible.” Presentations Highlight Sustained Progression-Free Survival and Deepening Durable Responses for Patients Treated with BRUKINSA in Treatment-Naïve and Relapsed/Refractory (R/R) Settings Five-year follow-up results from Cohort 1 of the Phase 3 SEQUOIA study showed sustained progression-free survival (PFS) benefit with BRUKINSA in patients with treatment-naïve chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), with no new safety signals observed. Results from the LTE (long-term extension rollover) study of patients with treatment-naïve and R/R CLL also showed that treatment with BRUKINSA as a single agent or as an investigational treatment in combination with obinutuzumab achieved high overall and complete response rates. With a median follow-up of up to 6.5 years, the responses were sustained, and no new safety signals were observed. Results from an LTE study of patients with Waldenström macroglobulinemia (WM) from the Phase 3 ASPEN study, with a median follow-up of up to 5.8 years, demonstrated that treatment with BRUKINSA monotherapy remained durable and the safety/tolerability profile remained favorable. Data from a Phase 2 study showed patients with prior intolerance to acalabrutinib were able to safely and effectively switch to BRUKINSA, with the majority of patients not experiencing recurrence of prior acalabrutinib-intolerance events while maintaining or deepening responses. Pipeline Data Show Early Safety and Efficacy Across Multiple B-cell Malignancies First-in-human Phase 1/2 CaDAnCe-101 presentations (two oral, one poster) highlighted generally manageable safety and promising efficacy results for BTK degrader, BGB-16673, in patients with R/R CLL/SLL, WM, and R/R indolent non-Hodgkin’s lymphoma. BGB-16673, which induces BTK degradation, is the first and most advanced asset from BeiGene’s chimeric degradation activation compound (CDAC) platform. Oral presentation of the BGB-11417-101 Phase 1 study demonstrated B-cell lymphoma 2 (BCL2) inhibitor sonrotoclax in combination with BRUKINSA continued to show promising efficacy and was generally well-tolerated in patients with treatment-naïve CLL/SLL; this combination is being evaluated in the Phase 3 CELESTIAL-TNCLL study ( NCT06073821 ). BeiGene Presentations During ASH 2024 Abstract Title Presentation Details Lead Author BRUKINSA Prospective patient preference study for Bruton tyrosine kinase inhibitor (BTKi) treatment attributes and factors affecting patient shared decision-making CLL/SLL in the USA Publication #2265 Poster Dec. 7, 5:30-7:30 p.m. S. Ailawadhi Real-world Bruton tyrosine kinase inhibitor (BTKi) utilization and clinical outcomes among patients with chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) Publication #2353 Poster Dec. 7, 5:30-7:30 p.m. J. Hou Long-term clinical outcomes in patients with Waldenström macroglobulinemia (WM) who received zanubrutinib in the phase 3 ASPEN study: A report from the zanubrutinib extension study Publication #3031 Poster Dec. 8, 6-8 p.m. S. D’Sa Patient medication preferences in follicular lymphoma (FL) in the United States (USA): A discrete choice experiment (DCE) Publication #3655 Poster Dec. 8, 6-8 p.m. S. Gaballa Evaluating reasons for differences in real-world (RW) clinical outcomes among patients with R/R MCL on covalent BTKis Publication #3732 Poster Dec. 8, 6-8 p.m. T. Phillips Sustained superiority of zanubrutinib vs bendamustine + rituximab in treatment-naive chronic lymphocytic leukemia/small lymphocytic lymphoma (TN CLL): 5-year follow-up of cohort 1 from the SEQUOIA study Publication #3249 Poster Dec. 8, 6-8 p.m. M. Shadman Deep and sustained responses in patients with CLL treated with zanubrutinib or zanubrutinib + obinutuzumab in phase 1/2 AU-003 and phase 1b GA-101 studies: A report from the zanubrutinib extension study Publication #3255 Poster Dec. 8, 6-8 p.m. C. Tam Comparative efficacy of zanubrutinib plus obinutuzumab versus last prior treatment in relapsed/refractory follicular lymphoma: Growth modulation index analysis from ROSEWOOD study Publication #3029 Poster Dec. 8, 6-8 p.m. J. Trotman Impact of novel therapies (NTs) on real-world (RW) clinical outcomes of patients (pts) with relapsed/refractory (R/R) mantle-cell lymphoma (MCL) by race/ethnicity and TP53 mutation status Publication #5097 Poster Dec. 9, 6-8 p.m. T. Phillips Zanubrutinib is well tolerated and effective in acalabrutinib-intolerant patients with B-cell malignancies Publication #4632 Poster Dec. 9, 6-8 p.m. M. Shadman Long-term impact of dose interruptions (DIs) of Bruton tyrosine kinase inhibitors (BTKis) on change in IgM levels and clinical outcomes in Waldenström macroglobulinemia (WM) Publication #4412 Poster Dec. 9, 6-8 p.m. J. Trotman Final analysis of a phase 1 study of zanubrutinib plus lenalidomide in patients with relapsed/refractory diffuse large B-cell lymphoma Publication #986 Oral Dec. 9, 4:45 p.m. Z. Song Treatment with zanubrutinib in patients with chronic lymphocytic leukemia or small lymphocytic lymphoma who were previously treated with another Bruton tyrosine kinase inhibitor in a US community oncology setting Publication #7763 Online D. Andorsky BGB-16673 (BTK CDAC) Preliminary efficacy and safety of the Bruton tyrosine kinase degrader BGB-16673 in patients with relapsed or refractory (R/R) indolent NHL: Results from the phase 1 CaDAnCe-101 study Publication #1649 Poster Dec. 7, 5:30-7:30 p.m. C. Tam Preliminary efficacy and safety of the Bruton tyrosine kinase degrader BGB-16673 in patients with relapsed or refractory Waldenström macroglobulinemia: Results from the phase 1 CaDAnCe-101 study Publication #860 Oral Dec. 9, 3 p.m. J. Seymour Preliminary efficacy and safety of the Bruton tyrosine kinase degrader BGB-16673 in patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma: Results from the phase 1 CaDAnCe-101 study Publication #885 Oral Dec. 9, 3:15 p.m. M. Thompson BGB-16673, a selective BTK degrader, exhibits deeper inhibition of cancer cell signaling pathways and better efficacy in MCL models Publication #5833 Online H. Wang Sonrotoclax (BCL2 Inhibitor) Sonrotoclax and zanubrutinib as frontline treatment for CLL demonstrates high MRD clearance rates with good tolerability: Data from an ongoing phase 1/1b study BGB-11417-101 Publication #1012 Oral Dec. 9, 5:15 p.m. J. Soumerai CELESTIAL-TNCLL: An ongoing, open-label, multiregional, phase 3 study of sonrotoclax (BGB-11417) + zanubrutinib vs venetoclax + obinutuzumab for treatment-naive CLL Publication #6807 Online P. Patten BGB-11417-203, an ongoing, phase 2 study of sonrotoclax (BGB-11417), a next-generation BCL2 inhibitor, in patients with Waldenström macroglobulinemia Publication #6289 Online H. Lee Cell Therapy Research iPSC-derived CAR-γδT with SOCS1/CISH/BIM/FAS combinatorial KO demonstrated extended longevity and profound anti-tumor efficacy without cytokine support in preclinical studies Publication #4790 Poster Dec. 7, 5:30-7:30 p.m. J. Yu Select Investigator-Initiated Trial Multicenter Phase II trial of zanubrutinib, obinutuzumab, and venetoclax (BOVen) in treatment-naïve chronic lymphocytic leukemia: 5-year follow up, retreatment outcomes, and impact of MRD kinetics (ΔMRD400) Publication #1867 Poster Dec. 7, 5:30-7:30 p.m. J. Soumerai About BRUKINSA ® (zanubrutinib) BRUKINSA is an orally available, small molecule inhibitor of Bruton’s tyrosine kinase (BTK) designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared with other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease-relevant tissues. BRUKINSA has the broadest label globally of any BTK inhibitor and is the only BTK inhibitor to provide the flexibility of once or twice daily dosing. The global BRUKINSA clinical development program includes about 6,000 patients enrolled in 30 countries and regions across more than 35 trials. BRUKINSA is approved in more than 70 markets, and more than 100,000 patients have been treated globally. U.S. Indications and Important Safety Information for BRUKINSA (zanubrutinib) INDICATIONS BRUKINSA is a kinase inhibitor indicated for the treatment of adult patients with: Chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). Waldenström’s macroglobulinemia (WM). Mantle cell lymphoma (MCL) who have received at least one prior therapy. Relapsed or refractory marginal zone lymphoma (MZL) who have received at least one anti-CD20-based regimen. Relapsed or refractory follicular lymphoma (FL), in combination with obinutuzumab, after two or more lines of systemic therapy. The MCL, MZL and FL indications are approved under accelerated approval based on overall response rate and durability of response. Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trials. IMPORTANT SAFETY INFORMATION Warnings and Precautions Hemorrhage Fatal and serious hemorrhage has occurred in patients with hematological malignancies treated with BRUKINSA. Grade 3 or higher hemorrhage including intracranial and gastrointestinal hemorrhage, hematuria, and hemothorax was reported in 3.8% of patients treated with BRUKINSA in clinical trials, with fatalities occurring in 0.2% of patients. Bleeding of any grade, excluding purpura and petechiae, occurred in 32% of patients. Bleeding has occurred in patients with and without concomitant antiplatelet or anticoagulation therapy. Coadministration of BRUKINSA with antiplatelet or anticoagulant medications may further increase the risk of hemorrhage. Monitor for signs and symptoms of bleeding. Discontinue BRUKINSA if intracranial hemorrhage of any grade occurs. Consider the benefit-risk of withholding BRUKINSA for 3-7 days before and after surgery depending upon the type of surgery and the risk of bleeding. Infections Fatal and serious infections (including bacterial, viral, or fungal infections) and opportunistic infections have occurred in patients with hematological malignancies treated with BRUKINSA. Grade 3 or higher infections occurred in 26% of patients, most commonly pneumonia (7.9%), with fatal infections occurring in 3.2% of patients. Infections due to hepatitis B virus (HBV) reactivation have occurred. Consider prophylaxis for herpes simplex virus, pneumocystis jirovecii pneumonia, and other infections according to standard of care in patients who are at increased risk for infections. Monitor and evaluate patients for fever or other signs and symptoms of infection and treat appropriately. Cytopenias Grade 3 or 4 cytopenias, including neutropenia (21%), thrombocytopenia (8%) and anemia (8%) based on laboratory measurements, developed in patients treated with BRUKINSA . Grade 4 neutropenia occurred in 10% of patients, and Grade 4 thrombocytopenia occurred in 2.5% of patients. Monitor complete blood counts regularly during treatment and interrupt treatment, reduce the dose, or discontinue treatment as warranted. Treat using growth factor or transfusions, as needed. Second Primary Malignancies Second primary malignancies, including non-skin carcinoma, have occurred in 14% of patients treated with BRUKINSA. The most frequent second primary malignancy was non-melanoma skin cancers (8%), followed by other solid tumors in 7% of the patients (including melanoma in 1% of patients) and hematologic malignancies (0.7%). Advise patients to use sun protection and monitor patients for the development of second primary malignancies. Cardiac Arrhythmias Serious cardiac arrhythmias have occurred in patients treated with BRUKINSA. Atrial fibrillation and atrial flutter were reported in 4.4% patients treated with BRUKINSA, including Grade 3 or higher cases in 1.9% of patients. Patients with cardiac risk factors, hypertension, and acute infections may be at increased risk. Grade 3 or higher ventricular arrhythmias were reported in 0.3% of patients. Monitor for signs and symptoms of cardiac arrhythmias (e.g., palpitations, dizziness, syncope, dyspnea, chest discomfort), manage appropriately, and consider the risks and benefits of continued BRUKINSA treatment. Hepatotoxicity, Including Drug-Induced Liver Injury Hepatotoxicity, including severe, life-threatening, and potentially fatal cases of drug-induced liver injury (DILI), has occurred in patients treated with Bruton tyrosine kinase inhibitors, including BRUKINSA. Evaluate bilirubin and transaminases at baseline and throughout treatment with BRUKINSA. For patients who develop abnormal liver tests after BRUKINSA, monitor more frequently for liver test abnormalities and clinical signs and symptoms of hepatic toxicity. If DILI is suspected, withhold BRUKINSA. Upon confirmation of DILI, discontinue BRUKINSA. Embryo-Fetal Toxicity Based on findings in animals, BRUKINSA can cause fetal harm when administered to a pregnant woman. Administration of zanubrutinib to pregnant rats during the period of organogenesis caused embryo-fetal toxicity, including malformations at exposures that were 5 times higher than those reported in patients at the recommended dose of 160 mg twice daily. Advise women to avoid becoming pregnant while taking BRUKINSA and for 1 week after the last dose. Advise men to avoid fathering a child during treatment and for 1 week after the last dose. If this drug is used during pregnancy, or if the patient becomes pregnant while taking this drug, the patient should be apprised of the potential hazard to a fetus. Adverse Reactions The most common adverse reactions (≥30%), including laboratory abnormalities, in patients who received BRUKINSA (N=1729) are decreased neutrophil count (51%), decreased platelet count (41%), upper respiratory tract infection (38%), hemorrhage (32%), and musculoskeletal pain (31%). Drug Interactions CYP3A Inhibitors: When BRUKINSA is co-administered with a strong CYP3A inhibitor, reduce BRUKINSA dose to 80 mg once daily. For coadministration with a moderate CYP3A inhibitor, reduce BRUKINSA dose to 80 mg twice daily. CYP3A Inducers: Avoid coadministration with strong or moderate CYP3A inducers. Dose adjustment may be recommended with moderate CYP3A inducers. Specific Populations Hepatic Impairment: The recommended dose of BRUKINSA for patients with severe hepatic impairment is 80 mg orally twice daily. Please see full U.S. Prescribing Information including U.S. Patient Information . About BeiGene BeiGene is a global oncology company that is discovering and developing innovative treatments that are more affordable and accessible to cancer patients worldwide. With a broad portfolio, we are expediting development of our diverse pipeline of novel therapeutics through our internal capabilities and collaborations. We are committed to radically improving access to medicines for far more patients who need them. Our growing global team of more than 10,000 colleagues spans five continents. To learn more about BeiGene, please visit www.beigene.com and follow us on LinkedIn , X (formerly known as Twitter), Facebook and Instagram . Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding BeiGene’s continued leadership in hematology and commitment to bringing innovative medicines to as many people with cancer as possible; the safety and efficacy of BeiGene’s pipeline assets; and BeiGene’s plans, commitments, aspirations, and goals under the heading “About BeiGene.” Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeiGene’s ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing, and progress of clinical trials and marketing approval; BeiGene’s ability to achieve commercial success for its marketed medicines and drug candidates, if approved; BeiGene’s ability to obtain and maintain protection of intellectual property for its medicines and technology; BeiGene’s reliance on third parties to conduct drug development, manufacturing, commercialization, and other services; BeiGene’s limited experience in obtaining regulatory approvals and commercializing pharmaceutical products; BeiGene’s ability to obtain additional funding for operations and to complete the development of its drug candidates and achieve and maintain profitability; and those risks more fully discussed in the section entitled “Risk Factors” in BeiGene’s most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in BeiGene’s subsequent filings with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of this press release, and BeiGene undertakes no duty to update such information unless required by law.

Funds will advance the development of ONL1204 Ophthalmic Solution with the initiation of a Phase 2 global study for the treatment of geographic atrophy (GA) associated with dry age-related macular degeneration (AMD)ANN ARBOR, Mich., Sept. 13, 2024 (GLOBE NEWSWIRE) -- ONL Therapeutics, Inc., a clinical-stage biopharmaceutical company developing novel therapies for protecting the vision of patients with retinal disease, today announced that it has secured $65M in Series D financing. Johnson & Johnson Innovation – JJDC, Inc. led the round that was backed by a consortium of investors that included Bios Partners, Novartis Venture Fund, and Visionary Ventures, amongst others. “We are grateful to our new and returning investors who are making it possible for the company to achieve its vision of helping patients see the future,” commented David Esposito, chief executive officer of ONL Therapeutics. “This new round of funding provides an exciting opportunity for our company to advance the clinical development program for ONL1204.” ONL Therapeutics will continue to work alongside Johnson & Johnson’s Specialty Ophthalmology research and development team to provide a forum for sharing input to the company’s ongoing clinical development plans. ONL Therapeutics is the first and only clinical-stage company focused on the unique mechanism of action (MOA) preventing Fas-mediated death of retinal cells and inflammatory signaling pathways, the root causes of vision loss and blindness. In a Phase 1b clinical trial of ONL1204 Ophthalmic Solution in patients with GA associated with dry AMD, patients treated with ONL1204 showed reductions in the rate of growth of the GA lesion with either a single injection or two injections (90 days apart) after 6 months of treatment compared to sham patients. A consistent treatment effect was seen when comparing treated eyes versus fellow eyes. “This new round of funding builds on the University of Michigan’s support of ground-breaking biomedical research that will enable us to further advance our unique and differentiated clinical program in GA, a disease that is responsible for nearly one in five cases of blindness caused by macular degeneration,” said David Zacks, M.D., Ph.D., co-founder and chief scientific officer of ONL Therapeutics. “With the unique MOA of ONL1204 addressing neuroprotection and our compelling Phase 1 clinical data, we are excited for the potential to bring a new innovation to GA patients around the world.” About ONL1204 Ophthalmic SolutionONL1204 is a novel, first-in-class small molecule Fas inhibitor designed to protect key retinal cells, including photoreceptors, from cell death that occurs across a range of retinal diseases and conditions. Death of these retinal cells, through both direct and inflammatory signaling pathways, is the root cause of vision loss and the leading cause of blindness. The company’s later-stage clinical development program for ONL1204 includes a Phase 2 study in the U.S. for the treatment of macula-off retinal detachment (NCT05730218), a condition for which the compound has been granted orphan drug designation by the United States Food and Drug Administration (FDA). The company has also conducted a Phase 1b clinical trial in patients with geographic atrophy (GA) associated with age-related macular degeneration (AMD) (NCT04744662) and a Phase 1b clinical trial in patients with progressing open-angle glaucoma (NCT05160805) at sites in Australia and New Zealand. About ONL TherapeuticsONL Therapeutics (ONL) is a clinical-stage biopharmaceutical company committed to developing first-in-class therapeutics to protect and improve the vision of patients with retinal disease. By advancing a breakthrough technology designed to protect key retinal cells from Fas-mediated cell death, ONL is pioneering a new approach to preserving vision. For more information about ONL Therapeutics, please visit www.onltherapeutics.com.