On April 21, 2025 Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; "Ono") reported that it has entered into a research collaboration agreement with Jorna Therapeutics (Headquarters: Cambridge, Massachusetts, USA; CEO: Chengwei Luo; "Jorna") in December 2024 aimed at drug discovery using Jorna’s proprietary ribonucleic acid (RNA) editing platform (Press release, Ono, APR 21, 2025, View Source [SID1234652004]). Having completed the validation of this platform, Ono has begun the search for nucleic acid sequences that could serve as drug candidates.

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Under the agreement, Jorna will design RNA editing drug sequences using its proprietary protein and RNA generative AI model, which is based on the quantum mechanics-based AI called SkyEngine. Ono will have an exclusive option right to discover, develop, and commercialize drug candidates generated by the sequences designed by Jorna worldwide. In addition to the upfront payment already made to Jorna, Ono will also pay for research funding and milestone payments based on the progress of the research.

"We highly value Jorna’s unique generative AI technologies, which combine large-scale amino acid sequence information and language models to design desired proteins," said Seishi Katsumata, Corporate Officer / Executive Director, Discovery & Research of Ono. "Through this partnership, we aim to accelerate drug development using RNA editing technology and provide new treatment options to patients around the world with unmet medical needs."

"We are delighted to collaborate with Ono to advance our shared vision of delivering groundbreaking therapies to patients. This partnership is expected to accelerate efforts to bring RNA editing-based therapies to market by leveraging Ono’s global resources and expertise," said Chengwei Luo, Founder and CEO of Jorna.

On April 21, 2025 Axcynsis Therapeutics that is at the forefront of developing cutting-edge Antibody Drug Conjugate (ADC) therapies, reported two upcoming poster presentations at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025 (Press release, Axcynsis Therapeutics, APR 21, 2025, View Source [SID1234651989]). This meeting will be held in Chicago from April 25th to 30th 2025.

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The company will present the following two assets:

AT65474 is a highly selective anti-CLDN6 ADC with a proprietary payload
Session Category: Experimental and Molecular Therapeutics

Session Title: Antibody-Based Cancer Therapeutics 4

Session Date and Time: April 30, 2025, 9:00 AM to 12:00 PM (Local Time)

Location: Poster Section 16

Poster Board Number: 7

Published Abstract Number: 6741

CLDN6 is a membrane-bound oncofetal protein and a promising target for cancers such as ovarian and testicular cancer. AT65474 is an antibody drug conjugate (ADC) candidate composed of a highly selective humanized anti-CLDN6 antibody conjugated to a proprietary payload, namely TCS132, with a drug-to-antibody (DAR) ratio of 4. The antibody is engineered to incorporate AxcynCYSTM technology for site-specific conjugation which can reproducibly achieve high DAR4 ratios of greater than 97% by HIC analysis.

The payload, TCS132, is derived from an FDA approved drug and when compared to the parent drug, demonstrates increased potency with broad sub-nanomolar activities across a wide range of cancer cell lines including those resistant to paclitaxel, DM1, MMAE and DXd. Additionally, TCS132 was chemically optimized for better PK properties and demonstrates a much-improved safety profile despite its increased potency over the parent drug.

AT2604 is a highly efficacious ADC targeting alkaline phosphatases ALPP and ALPPL2​
Session Category: Experimental and Molecular Therapeutics

Session Title: Antibody-Based Cancer Therapeutics 4

Session Date and Time: April 30, 2025, 9:00 AM to 12:00 PM (Local Time)

Location: Poster Section 16

Poster Board Number: 17

Published Abstract Number: 6751

Placental alkaline phosphatases, ALPP and ALPPL2, are highly homologous, membrane-bound proteins involved in fetal development. Despite having limited expression in normal tissues, they are highly differentiated in multiple tumor cells making them ideal targets for antibody drug conjugate (ADC) development.

AT2604 is an ADC incorporating an anti-ALPP/ALPPL2 antibody conjugated to monomethyl auristatin E (MMAE) with high DAR 4 homogeneity; and displays strong tumor growth inhibition at low doses in CDX models of gastric and pancreatic cancer. In patient-derived xenograft models of gastric cancer with moderate- and low-antigen expression, AT2604 achieves near-complete tumor regression at 3 and 6 mg/kg (QWx3), respectively. A preliminary toxicity assessment in non-human primates concluded a well-tolerated dose of 10 mg/kg (Q3Wx3) without exhibiting any non-reversible adverse effects and good plasma stability; implying that AT2604 possesses an improved safety profile over other vedotin-based ADCs.

On April 21, 2025 BostonGene, a leader in AI-driven molecular and immune profiling that accelerates drug development and personalizes patient care, reported that four abstracts have been selected for presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025, held from April 25 – 30, at McCormick Place Convention Center in Chicago, IL (Press release, BostonGene, APR 21, 2025, View Source [SID1234652005]). BostonGene will be exhibiting at booth #2452.

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Ferran Prat, PhD, JD, BostonGene’s Chief Commercial Officer, will also speak at the AACR (Free AACR Whitepaper) educational session, Academic Entrepreneurship: Getting Your Discovery to Patients on April 26 from 8:00 AM – 9:30 AM. The session will address key topics such as understanding intellectual property rights and patents, raising capital and developing commercialization pathways. By bridging these knowledge gaps, the session seeks to empower researchers to move innovations from the laboratory to patient care.

BostonGene is also proud to partner with the American Cancer Society to host the BrightEdge Entrepreneurs (BEE) Dinner at AACR (Free AACR Whitepaper) 2025, held on April 28 in Chicago. The event will spotlight eight innovative cancer-focused startups from the BEE Program, which empowers underrepresented scientific entrepreneurs with mentorship, investor access and early-stage funding.

In collaboration with leading academic institutions, BostonGene will present new research that highlights the power of its AI-enabled, integrated multiomic platform to drive precision oncology. By leveraging comprehensive genomic, transcriptomic and immune system profiling, the platform enables high-resolution tumor characterization, molecular subtyping, and immune landscape reconstruction—delivering clinical and mechanistic insights to optimize drug trials and clinical care. From refining transcriptional subtypes in small cell lung cancer and predicting treatment response in triple-negative breast cancer to improving germline variant interpretation and advancing biomarker detection in pan-cancer analyses, these studies demonstrate the platform’s utility in both clinical care and translational research. In addition, the studies also highlight its role in accelerating biomarker-informed drug development.

Details of BostonGene’s poster presentations at the AACR (Free AACR Whitepaper) Annual Meeting are below:

Abstract number: 1101
Title: A novel machine learning classifier for SCLC transcriptional subtypes
Date and time: Sunday, April 27 | 2:00 PM – 5:00 PM
Presenter: Carl Gay, MD, PhD, MD Anderson Cancer Center

In this study, BostonGene applied gradient-boosting machine learning models to refine the small cell lung cancer (SCLC) subtypes developed by Gay et al. (The University of Texas MD Anderson Cancer Center). The model demonstrated high performance metrics during validation, reinforcing its potential clinical impact.

Research done in collaboration with MD Anderson Cancer Center

Abstract number: 5308
Title: Pan-cancer analysis of TRIM37 copy-number and development of fit-for-screening in situ hybridization tools
Date and time: Sunday, April 27 | 2:00 PM – 5:00 PM
Presenter: J.D. Schonhoft, PhD, Repare Therapeutics

BostonGene’s WES platform was used to examine the copy numbers and RNA levels of TRIM37 across over 12,000 adult solid tumor samples. Newly developed DNA- and RNA-based in situ hybridization (ISH) approaches uncovered high TRIM37 expression in many patients, underscoring the need for sensitive ISH methods for examining clinical biomarkers.

Research done in collaboration with Repare Therapeutics

Abstract number: 2036
Title: Transcriptomic immune signatures and reconstructed immune cell types associated with pathological complete response to neoadjuvant chemotherapy in triple-negative breast cancer
Date and time: Monday, April 28 | 9:00 AM – 12:00 PM
Presenter: Tomohiro Oshino, MD, Hokkaido University Hospital

BostonGene’s tumor microenvironment (TME) subtyping and cell deconvolution were used for TME reconstruction in triple-negative breast cancer (TNBC). The findings revealed patients with immune-enriched TME were more likely to achieve a pathological complete response following neoadjuvant chemotherapy, underscoring the potential of TME-based analytical tools to predict treatment response in TNBC.

Research done in collaboration with Hokkaido University Hospital

Abstract number: 5047
Title: Enhancing germline variant calling: Adjustment with tumor samples to filter low-confidence variants from clonal hematopoiesis
Date and time: Tuesday, April 29 | 9:00 AM – 12:00 PM
Presenter: Artur Baisangurov, MS, BostonGene

Leveraging whole-exome sequencing (WES) analysis for 2,078 cancer patients, BostonGene developed a filtering method to improve the accuracy of germline reports. This novel approach identified artifacts caused by molecular and biological variations to streamline quality control of genetic reports for cancer patients.

The abstracts will be published in an online-only Proceedings supplement to the AACR (Free AACR Whitepaper) journal Cancer Research.

On April 21, 2025 ImmunityBio, Inc. (NASDAQ: IBRX), a leading immunotherapy company, reported new clinical findings for ANKTIVA (nogapendekin alfa inbakicept-pmln) in non-muscle invasive bladder cancer carcinoma in situ (NMIBC CIS) and updated data on papillary disease without CIS at the American Urological Association Annual Meeting (AUA 2025) in Las Vegas, April 26-29 (Press release, ImmunityBio, APR 21, 2025, View Source [SID1234651990]). The company will also host an educational and peer-networking event to discuss ImmunityBio’s approach to treatment of NMIBC CIS and papillary disease and how they have impacted patients.

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"ImmunityBio has made remarkable strides in 2025, marked by groundbreaking long-term efficacy data for ANKTIVA and the launch of our Expanded Access Program to help end the BCG shortage," said Dr. Patrick Soon-Shiong, Founder, Executive Chairman, Global Chief Scientific & Medical Officer of ImmunityBio. "With these critical advancements, we are eager to connect, collaborate, and drive meaningful discussions with the urology community at AUA 2025, shaping the future of bladder and prostate cancer care together. In addition, ImmunityBio has applied for Expanded Access for the use of ANKTIVA as a ‘BioShield’ in patients receiving chemotherapy, radiation, and immunotherapy to enable access to ANKTIVA across the country for patients in need."

Oral Presentations by Key Opinion Leaders:

Presented research will continue to demonstrate long-term duration of response with ANKTIVA + BCG in an updated analysis of the pivotal QUILT 3.032 trial in CIS and papillary BCG-Unresponsive NMIBC.

An Update on QUILT 3.032: Complete Responses to N-803 plus BCG Therapy in BCG-Unresponsive Bladder Carcinoma in Situ (CIS) With or Without Ta/T1 Papillary Disease (PD12-12)
Chang S. Oral Podium Presentation. Session: Bladder Cancer: Non-Invasive II, Saturday, April 26, 3:30 pm – 5:30 pm PDT, Galileo 1001, The Venetian Convention & Expo Center
Keynote Events by Dr. Soon-Shiong at AUA

Patrick Soon-Shiong, M.D., will participate in a series of presentations showcasing the power and potential of immunotherapy in shaping the future of urological cancers.

Embracing the Future: The Power of Innovation in a Changing World
Dr. Patrick Soon-Shiong, Keynote. AUA Innovation Nexus Conference, Friday, April 25, 1:00-1:45 pm PDT, The Venetian Convention & Expo Center
The Science of the Triangle Offense: NMIBC Patient and Prostate Cancer Experience
Educational and peer-networking event with Drs. Patrick Soon-Shiong, ImmunityBio Chief Medical Officer Sandeep "Bobby" Reddy, and Senior Vice President of Medical Affairs Bruce Brown, Saturday, April 26, 6:30 pm – 9:00 pm PDT, The Venetian Convention & Expo Center
The Missing Link: Overcoming Lymphopenia through NK & Memory T Cells to Achieve Durable Responses in Urological Diseases – ALC Matters, Duration Matters, the Immune System Matters
Dr. Patrick Soon-Shiong, ImmunityBio Product Theater, Sunday, April 27, 1:30 pm – 2:30 pm PDT, Science & Technology Hall, The Venetian Convention & Expo Center
About ANKTIVA

The cytokine interleukin-15 (IL-15) plays a crucial role in the immune system by affecting the development, maintenance, and function of key immune cells—NK and CD8+ killer T cells—that are involved in killing cancer cells. By activating NK cells, ANKTIVA overcomes the tumor escape phase of clones resistant to T cells and restores memory T cell activity with resultant prolonged duration of complete response.

ANKTIVA is a first-in-class IL-15 agonist IgG1 fusion complex, consisting of an IL-15 mutant (IL-15N72D) fused with an IL-15 receptor alpha, which binds with high affinity to IL-15 receptors on NK, CD4+, and CD8+ T cells. This fusion complex of ANKTIVA mimics the natural biological properties of the membrane-bound IL-15 receptor alpha, delivering IL-15 by dendritic cells and drives the activation and proliferation of NK cells with the generation of memory killer T cells that have retained immune memory against these tumor clones. The proliferation of the trifecta of these immune killing cells and the activation of trained immune memory results in immunogenic cell death, inducing a state of equilibrium with durable complete responses. ANKTIVA has improved pharmacokinetic properties, longer persistence in lymphoid tissues, and enhanced anti-tumor activity compared to native, non-complexed IL-15 in-vivo.

ANKTIVA was approved by the FDA in 2024 for BCG-unresponsive non-muscle invasive bladder cancer CIS with or without papillary tumors. For more information, visit ImmunityBio.com (Founder’s Vision) and Anktiva.com.

On April 21, 2025 Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) reported that China’s National Medical Products Administration (NMPA) has accepted the supplemental New Drug Application (sNDA) for repotrectinib for the treatment of adult patients with solid tumors that harbor a neurotrophic tyrosine receptor kinase (NTRK) gene fusion (Press release, Zai Laboratory, APR 21, 2025, View Source [SID1234652006]). The application is intended for patients whose disease is locally advanced or metastatic, or where surgical resection is likely to result in severe morbidity, and who have either progressed following prior therapies or have no satisfactory alternative treatment options.

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"NTRK fusion-positive tumors represent a significant therapeutic challenge, particularly in the setting of acquired resistance to existing TRK tyrosine kinase inhibitors (TKIs)," said Dr. Rafael Amado, M.D., President, Head of Global Research and Development at Zai Lab. "There are no approved treatments for NTRK-positive cancers for both TKI-naïve and TKI-pretreated patients in China. Repotrectinib has the potential to become a next-generation TKI that can be used across a broad range of NTRK fusion-positive solid tumors in both settings."

In February 2025, China’s NMPA granted priority review to repotrectinib for the treatment of adult patients with advanced solid tumors that have an NTRK gene fusion.

About Repotrectinib

Repotrectinib is a next-generation tyrosine kinase inhibitor targeting the ROS1 and NTRK oncogenic drivers. Patients with solid tumors, including NSCLC, harboring ROS1 and NTRK gene fusions treated with approved targeted therapies often develop resistance mutations that limit binding of these drugs to their target. Ultimately, this leads to shortened duration of response and tumor progression. Repotrectinib is the first next-generation ROS1 and TRK TKI uniquely designed to improve durability of benefit, including in the brain, and to address acquired resistance.

In June 2024, AUGTYRO (repotrectinib) was approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult and pediatric patients 12 years of age and older with solid tumors that have a NTRK gene fusion, are locally advanced or metastatic or where surgical resection is likely to result in severe morbidity, and have progressed following treatment or have no satisfactory alternative therapy.

In May 2024, AUGTYRO was approved by the NMPA for the treatment of adult patients with locally advanced or metastatic ROS1-positive non-small-cell lung cancer (NSCLC). It was approved by the FDA for this indication in November 2023.

Zai Lab has an exclusive license agreement with Turning Point Therapeutics, Inc. (a Bristol Myers Squibb company) to develop and commercialize AUGTYRO in Greater China (mainland China, Hong Kong, Taiwan, and Macau, collectively).

About NTRK-Positive Solid Tumors

NTRK-positive advanced malignancies are life-threatening with poor prognoses and represent an area of significant unmet medical need in adult and pediatric patients. Existing targeted therapies have demonstrated clinical benefits but are limited by the duration of response due to the emergence of acquired resistance mutations.1 In China, there was no approved treatment option for NTRK-positive cancers that was studied in both TKI-naïve and TKI-pretreated patients across solid tumors.

1 Harada G, Santini FC, Wilhelm C, Drilon A, et al. NTRK fusions in lung cancer: From biology to therapy. Lung Cancer. 2021;161:108-113.