On April 23, 2025 Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; "ONO") reported that it will present positive results from the Phase 2 PROSPECT study of tirabrutinib (ONO-4059) for the treatment of relapsed or refractory primary central nervous system lymphoma during an oral presentation at the Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) being held May 30 to June 3, 2025, in Chicago, Illinois (Press release, Ono, APR 23, 2025, View Source [SID1234652073]). The company will also present data on ONO-4578, an investigational EP4 antagonist, for the treatment of locally advanced resectable rectal cancer, during a poster presentation.

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"We are fully committed to developing innovative drugs for cancer patients. This announcement is an important step forward in demonstrating that we can potentially provide new value to patients," said Tatsuya Okamoto, Corporate Officer / Executive Director, Clinical Development at ONO. "We aim to offer new treatment options in cancer care globally and contribute to improving patients’ quality of life."

Details for both studies and data to be presented are as follows:

Tirabrutinib (ONO-4059)
Abstract Title: Tirabrutinib for the treatment of relapsed or refractory primary central nervous system lymphoma: Efficacy and safety from the phase II PROSPECT study.
Abstract Submission ID: 487376
Session Type and Title: Rapid Oral Abstract – Central Nervous System Tumors
Session Date and Time: May 31, 2025, 3:00-4:30 PM CDT
Presenter: Lakshmi Nayak, MD, Dana-Farber Cancer Institute

ONO-4578
Abstract Title: Neoadjuvant ONO-4578, an EP4 antagonist, in combination with nivolumab after chemoradiation therapy in locally advanced resectable rectal cancer.
Session Type and Title: Poster Session Gastrointestinal Cancer – Colorectal and Anal
Abstract Number for Publication: 3588
Poster Board: 257
Session Date and Time: May 31, 2025, 9:00 AM-12:00 PM CDT
Presenter: Yusuke Takahashi, Lower Gastrointestinal Surgery, National Hospital Organization Osaka National Hospital

On April 23, 2025 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported it will host a conference call and live webcast at 8:30 a.m. ET on Monday, April 28, 2025, to review updated safety and efficacy data from the ongoing Phase 2 open-label ADVANCED-2 trial of TARA-002 in patients with non-muscle invasive bladder cancer (NMIBC), including data from patients who have reached the 12-month evaluation timepoint (Press release, Protara Therapeutics, APR 23, 2025, https://ir.protaratx.com/news-releases/news-release-details/protara-therapeutics-host-conference-call-and-webcast-review [SID1234652042]). The data will be featured during an interactive poster session at the American Urological Association 2025 Annual Meeting on Saturday, April 26, 2025, at 7:00 a.m. PT.

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The live call can be accessed by registering as a participant here. Upon registration, participants will receive conference call dial-in information. A live webcast of the event can be accessed by visiting the Events and Presentations section of the Company’s website: View Source A replay of the webcast will be archived for a limited time following the event.

About ADVANCED-2

ADVANCED-2 (NCT05951179) is a Phase 2 open-label trial assessing intravesical TARA-002 in NMIBC patients with carcinoma in situ or CIS (± Ta/T1) who are Bacillus Calmette-Guérin (BCG)-unresponsive (n≈100) and BCG-Naïve (n=31). The BCG-Unresponsive cohort has been designed to be registrational in alignment with the U.S. Food and Drug Administration’s 2024 BCG-Unresponsive Non-muscle Invasive Bladder Cancer: Developing Drugs and Biological Products for Treatment, Draft Guidance for Industry.

About TARA-002

TARA-002 is an investigational cell therapy in development for the treatment of NMIBC and of LMs, for which it has been granted Rare Pediatric Disease Designation by the U.S. Food and Drug Administration. TARA-002 was developed from the same master cell bank of genetically distinct group A Streptococcus pyogenes as OK-432, a broad immunopotentiator marketed as Picibanil in Japan by Chugai Pharmaceutical Co., Ltd. Protara has successfully shown manufacturing comparability between TARA-002 and OK-432.

When TARA-002 is administered, it is hypothesized that innate and adaptive immune cells within the cyst or tumor are activated and produce a pro-inflammatory response with release of cytokines such as tumor necrosis factor (TNF)-alpha, interferon (IFN)-gamma IL-6, IL-10, IL-12. TARA-002 also directly kills tumor cells and triggers a host immune response by inducing immunogenic cell death, which further enhances the antitumor immune response.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

Bladder cancer is the 6th most common cancer in the United States, with NMIBC representing approximately 80% of bladder cancer diagnoses. Approximately 65,000 patients are diagnosed with NMIBC in the United States each year. NMIBC is cancer found in the tissue that lines the inner surface of the bladder that has not spread into the bladder muscle.

On April 23, 2025 Mersana Therapeutics, Inc. (NASDAQ: MRSN), a clinical-stage biopharmaceutical company focused on discovering and developing a pipeline of antibody-drug conjugates (ADCs) targeting cancers in areas of high unmet medical need, reported that the following presentations related to emiltatug ledadotin (Emi-Le; XMT-1660), Mersana’s B7-H4-directed Dolasynthen ADC, will be given at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2025 Annual Meeting taking place May 30-June 3, 2025 at McCormick Place, Chicago, IL (Press release, Mersana Therapeutics, APR 23, 2025, View Source [SID1234652058]):

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Oral Presentation Details

Title: Initial Phase 1 Dose Escalation Data for Emiltatug Ledadotin (Emi-Le), a Novel B7-H4-Directed Dolasynthen Antibody-Drug Conjugate
Rapid Oral Session Title: Developmental Therapeutics
Date and Time: Monday, June 2, 2025 from 8:00-9:30 a.m. CT
Abstract Number: 3009
Presenter: Erika Hamilton, M.D., Director Breast Cancer Research Program, Sarah Cannon Research Institute in Nashville, Tennessee
This presentation will include clinical data from dose escalation and backfill cohorts across tumor types from the ongoing Phase 1 clinical trial of Emi-Le.

Poster Presentation Details

Title: Emiltatug Ledadotin (Emi-Le): A B7-H4-Directed Dolasynthen Antibody-Drug Conjugate (ADC) Being Investigated in Phase 1 Dose Expansion in Patients with Triple Negative Breast Cancer Who Received at Least One Prior Topoisomerase-1 Inhibitor ADC
Poster Session Title: Breast Cancer – Metastatic
Date and Time: Monday, June 2, 2025 from 9:00-12:00 p.m. CT
Abstract Number: TPS1141
Presenter: Hyo Han, M.D., H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida
This "trial-in-progress" presentation will focus on the design of the ongoing expansion portion of Mersana’s Phase 1 clinical trial of Emi-Le that is actively enrolling patients with triple negative breast cancer (TNBC) who have received one to four prior treatment lines, including at least one topoisomerase-1 inhibitor ADC.

Additionally, as previously announced, an oral presentation regarding Emi-Le will be given at the ESMO (Free ESMO Whitepaper) Breast Cancer 2025 Annual Congress in Munich, Germany on Thursday, May 15, 2025. This presentation will focus on TNBC clinical data from dose escalation and backfill cohorts from the Phase 1 clinical trial of Emi-Le.

About Emi-Le
Emi-Le is a B7-H4-directed Dolasynthen ADC with a precise, target-optimized drug-to-antibody ratio (DAR 6) and a proprietary auristatin payload with controlled bystander effect. This candidate is being investigated in a Phase 1 dose escalation and expansion trial in patients with solid tumors, including breast, endometrial and ovarian cancers as well as adenoid cystic carcinoma type 1. In the initial clinical data that were reported as of a December 13, 2024 data cutoff, Emi-Le was observed to be generally well tolerated with differentiated safety and tolerability profile. Additionally, confirmed objective responses were observed across all enrolled tumor types, including in patients with triple negative breast cancer (TNBC) who had previously been treated with a topoisomerase-1 inhibitor (topo-1) ADC.

The U.S. Food and Drug Administration has granted two Fast Track designations to Emi-Le for the treatment of 1) adult patients with advanced or metastatic triple-negative breast cancer, and 2) advanced or metastatic breast cancer in patients with human epidermal growth factor receptor 2 (HER2) low (IHC 1+ or IHC 2+/ISH–) or HER2-negative (IHC 0) disease, including TNBC, who have received a prior topo-1 ADC. For more information about Mersana’s ongoing Phase 1 trial of Emi-Le, please visit clinicaltrials.gov (NCT05377996).

On April 23, 2025 Arcus Biosciences, Inc. (NYSE:RCUS), a clinical-stage, global biopharmaceutical company focused on developing differentiated molecules and combination therapies for people with cancer, reported that data from the ARC-20 study will be presented in an oral session at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting taking place May 30 – June 3, 2025 (Press release, Arcus Biosciences, APR 23, 2025, View Source [SID1234652074]).

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The oral presentation will highlight the first safety and efficacy data for the ARC-20 expansion cohort evaluating the HIF-2a inhibitor casdatifan plus cabozantinib, an anti-vascular endothelial growth factor receptor 2 tyrosine kinase inhibitor (VEGFR2-TKI), in patients with ccRCC that had previously been treated with immunotherapy alone or in combination with anti-VEGFR2-TKI therapies.

"We are thrilled to be presenting the first combination data evaluating our HIF-2a inhibitor, casdatifan, in combination with cabozantinib in an oral presentation at ASCO (Free ASCO Whitepaper)," said Terry Rosen, Ph.D., chief executive officer of Arcus. "The data being presented support our rapidly advancing and differentiated development program for casdatifan, which includes the initiation of our Phase 3 trial, PEAK-1, that will evaluate the same combination as this cohort. The development plan also includes our clinical collaboration to evaluate casdatifan in combination with AstraZeneca’s PD-1/CTLA-4 bispecific antibody, volrustomig, in immuno-oncology-naive ccRCC patients. We look forward to presenting additional data from the ARC-20 study throughout the year."

Study

Title

Abstract Number

Session Type & Title

Session Date & Time

Casdatifan (HIF-2a Inhibitor)

ARC-20

Combination Casdatifan Plus Cabozantinib Expansion Cohort of Phase 1 ARC-20 Study in Previously Treated Patients with Clear Cell Renal Cell Carcinoma

4506

Oral Abstract Session – Genitourinary Cancer—Kidney and Bladder

6/01/2025,

9:45 AM – 12:45 PM CT

Investors may dial in to the conference call at +1 404 975 4839 (local) or +1 833 470 1428 (toll-free) using Conference ID: 446724 on Monday, June 2, 2025, at 5:00 AM PT / 7:00 AM CT. Participants may also register for the call online using the following link: View Source To access the live webcast and accompanying slide presentation, please visit the "Investors & Media" section of the Arcus Biosciences website at www.arcusbio.com. A replay will be available following the live event.

About RCC

According to the American Cancer Society, kidney cancer is among the top 10 most commonly diagnosed forms of cancer among both men and women in the U.S., and an estimated 80,980 Americans will be diagnosed with kidney cancer in 2025. Clear cell RCC is the most common type of kidney cancer in adults. If detected in its early stages, the five-year survival rate for RCC is high; for patients with advanced or late-stage metastatic RCC, however, the five-year survival rate is only 18%. In 2022, approximately 32,200 patients with advanced kidney cancer required systemic therapy in the U.S., with over 20,000 patients receiving first-line treatment.

About Casdatifan (AB521)

Casdatifan is a small-molecule inhibitor of HIF-2a, a transcription factor responsible for activating multiple tumor growth pathways in hypoxic and pseudo-hypoxic tumor environments. By selectively binding HIF-2⍺, casdatifan is designed to shut down hypoxic oncogenesis, which blocks tumor growth and key oncogenic pathways, leading to cancer cell death. Clear cell RCC (ccRCC) is almost universally associated with HIF-2a dysregulation. Casdatifan is currently being evaluated in ARC-20, a Phase 1/1b study in renal cell carcinoma and other cancers.

Casdatifan is an investigational molecule. Approval from any regulatory authority for its use has not been received, and its safety and efficacy have not been established.

On April 23, 2025 AB Science SA (Euronext – FR0010557264 – AB) reported that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) granted orphan drug status to AB8939 for the treatment of acute myeloid leukemia (AML) (Press release, AB Science, APR 23, 2025, View Source [SID1234652043]).

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AB8939 had already obtained orphan drug designation from the US Food and Drug Administration (FDA) in AML.

Granting of this orphan drug status in the EU is a significant milestone because it means that the COMP considered that AB8939 offers a significant benefit to people suffering from this condition in addition to existing treatments.

Indeed, the criteria to obtain orphan drug designation at EMA differ from those at FDA and are very stringent for the following reasons :

-There must be no satisfactory method of diagnosis, prevention or treatment of the condition or, if such a method exists, the medicinal product must offer a significant benefit to patients. -Because the application is based on an assumption of significant benefit, a comparison with authorized treatments is required.

-Significant benefit means that a medicine produces a clinically relevant advantage or makes a major contribution to patient care, as compared with existing methods to treat the condition. Thus, orphan designation is given to a product that will improve patients’ current treatment, having considered what else is available.

-To follow the spirit of the orphan legislation, which makes it clear that an orphan application may bemade at any stage of the development, significant benefit will be based on the available evidence at the stage of designation.

In support of the significant benefit of AB8939 in AML, AB Science has released preclinical data from mouse models demonstrating a significant benefit of AB8939 treatment over current therapies, such as cytarabine, azacitidine (Vidaza) and venetoclax (Venclexta). This included:

-Efficacy on resistant cells: AB8939 manages to have an effect on the cancer cells (blasts) of AML patients, even when these cells are resistant to other drugs such as vincristine or cytarabine. For example, 45% of vincristine-resistant cells and 66% of cytarabine-resistant cells still respond to AB8939, including in severe cases with complex genetic mutations (MECOM, TP53).

-Convincing results in xenograft models derived from refractory AML patients: In these mouse models which mimick human AML, AB8939 reduces tumors and prolongs survival, even when cells areresistant to cytarabine. -Additive effect with reference treatments: When used with other treatments (cytarabine, Vidaza or venetoclax), AB8939 further improves results. For example, with venetoclax, it eliminates cancer cells in the blood, spleen and bone marrow, without serious side effects

-Furthermore, unlike venetoclax, AB8939 does not cause blood toxicity (hematotoxicity) and appears to act synergistically with other treatments, reinforcing its efficacy.

AB Science also presented preliminary efficacy and safety data from phase 1 of AB8939 as a monotherapy, with a 3-day treatment cycle (stage 1 of phase 1) and a 14-day treatment cycle (stage 2 of phase 1).

Professor Olivier Hermine, President of AB Science’s Scientific Committee, member of the French Academy of Sciences and Head of the Hematology Department at Necker Hospital, commented: "This designation testifies to the potential of AB8939 for the treatment of AML. Indeed, AB8939 has shown activity as a monotherapy on Ara-C-resistant patient lines, including in unfavorable genetic situations (MECOM, TP53 mutations) that have resisted all treatments administered to date, as well as a synergistic effect with the reference treatments Vidaza and Venclexta. The ongoing Phase 1 trial will now evaluate the combination of AB8939 with these reference treatments in refractory patients".

About AB8939

AB8939 is a new synthetic molecule which jointly targets cancer cells, by destabilizing the microtubules essential for cell division, and cancer stem cells, by inhibiting enzymes (ALDH1A1 and ALDH2) essential for maintaining their physiological state and survival. The molecule ‘1-{4-[2-(5-ethoxymethyl-2-methylphenylamino)-oxazol-5-yl]phenyl}imidazolidin-2-one’ is the chemical name of AB8939. The intellectual property of AB8939 is 100% owned by AB Science.