On May 28, 2025 Bristol Myers Squibb (NYSE: BMY) reported that the company will participate in the Goldman Sachs 46th Annual Global Healthcare Conference on Wednesday, June 11, 2025 (Press release, Bristol-Myers Squibb, MAY 28, 2025, View Source [SID1234653432]).

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The company will take part in a fireside chat beginning at 10:00 a.m. ET.

Investors and the general public are invited to listen to the session by visiting View Source An archived edition of the session will be available following its conclusion.

On May 28, 2025 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, reported that Raul Rodriguez, the company’s president and CEO, will present a company overview at the Jefferies Global Healthcare Conference on Wednesday, June 4, 2025 at 12:50 p.m. ET in New York, NY (Press release, Rigel, MAY 28, 2025, View Source [SID1234653449]).

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To access the live webcast or archived recording, visit the Investor Relations section of the company’s website at www.rigel.com. Please connect to Rigel’s website prior to the start of the live webcast to allow for any software downloads.

On May 28, 2025 Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; "Ono"), reported that the results from the open label Phase 2 PROSPECT Study of tirabrutinib is to be presented at the 2025 American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting (Press release, Ono, MAY 28, 2025, View Source [SID1234653465]). Patients in the U.S. with relapsed or refractory primary central nervous system lymphoma (R/R PCNSL) who received oral tirabrutinib as a monotherapy achieved an overall response rate (ORR) of 67%.2

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Tirabrutinib is a highly selective irreversible, second generation Bruton’s tyrosine kinase inhibitor discovered by Ono in Japan. In March 2023, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to investigational tirabrutinib for the treatment of PCNSL.3

"PCNSL is a rare and aggressive extranodal non-Hodgkin lymphoma with historically poor survival rates and no approved treatments in the U.S.," said Tracy Batchelor, MD, MPH, Mass General Brigham Chair of Neurology and Coordinating Investigator for the PROSPECT study. "The PROSPECT Study data show tirabrutinib provided a promising response rate in patients with R/R PCNSL, supporting its role as a potentially effective treatment option for patients living with this devastating disease."

Study Results
In the PROSPECT Study, 48 patients with R/R PCNSL received oral tirabrutinib as a monotherapy once daily.2,4 The primary endpoint was ORR, and secondary endpoints included duration of response (DOR), time to response (TTR), and safety.2,4

After a median follow-up of 11.5 months, ORR was 67%, with a complete response rate of 44%.2 Median DOR was 9.3 months, and median TTR was 1.0 months.2 Exploratory endpoints included median overall survival, which was not reached, and median progression-free survival, which was 6.0 months.2

Tirabrutinib demonstrated a generally favorable safety profile.2 At data cutoff, 13 patients (27%) remained on tirabrutinib treatment.2 The main reasons for discontinuation were disease progression (54.2%) and death (8.3%); one patient discontinued due to an adverse event (AE).2 Incidence of grade ≥3 treatment-emergent adverse events (TEAEs) was 56.3%.2 Any-grade treatment-related TEAEs were experienced by 75.0%, and most frequently included anemia (18.8%), rash maculo-papular (16.7%), fatigue (14.6%), neutrophil count decreased (14.6%), lymphocyte count decreased (14.6%), pruritus (14.6%), and rash (14.6%).2 Two patients died of TEAEs, which were considered unrelated to study treatment. 2

"We are deeply grateful to the study investigators and the patients who participated in this study for making this important research possible," said Thomas Lechner, MSc. Ph.D., VP, Medical Affairs, ONO PHARMA USA INC. "Tirabrutinib is approved for relapsed or recurring PCNSL in Japan, Taiwan, and South Korea and our hope is to bring this potential treatment to those living with this hard-to-treat disease in the U.S as soon as possible."

PROSPECT Study data will be included in a regulatory submission to the FDA in the near future.

About PCNSL
PCNSL is a rare and aggressive extra nodal non-Hodgkin lymphoma (NHL) that is confined to the brain parenchyma, spinal cord, eye, or leptomeninges, without systemic involvement. The annual incidence rate of PCNSL is approximately five cases per 1,000,000 people in the U.S. The rate can further increase among immunocompromised people aged 65 years and older. The signs and symptoms presented in patients with PCNSL vary depending on the neuroanatomical site of the lesion, and include cranial neuropathy, neuropsychiatric symptoms, symptoms associated with increased intracranial pressure, seizures, ocular symptoms, headache, dysmotility, cranial neuropathy, and radiculopathy. There are no therapeutic products approved for the treatment of PCNSL in the U.S., and data guiding therapeutic approaches are very limited. Despite recent progress resulting in the improvement of clinical outcomes in newly diagnosed patients with PCNSL after an induction treatment, approximately 20 to 30 percent of patients are refractory to the initial treatment, and up to 60 percent of patients will eventually relapse. To learn more about R/R PCNSL, please visit navigatingpcnsl.com.

About Tirabrutinib
Tirabrutinib, discovered and developed by Ono Pharmaceutical Co., Ltd., is a highly potent selective BTK inhibitor. Signaling through the B-cell receptor (BCR) regulates cellular proliferation and activation, and promotes survival, differentiation, and clonal expansion of B-cells. The BCR signaling pathway plays an important role in a number of B-cell malignancies. In Japan, tirabrutinib was approved in March 2020 for the treatment of relapsed or refractory PCNSL and launched under the tradename of Velexbru in May 2020. It was subsequently approved for the treatment of Waldenstrom macroglobulinemia and lymphoplasmacytic lymphoma in August 2020.Tirabrutinib was approved for the treatment of relapsed or refractory PCNSL in South Korea in November 2021 and in Taiwan in February 2022.

About the PROSPECT Study
The PROSPECT Study is a Phase 2 trial (NCT04947319) evaluating the safety and efficacy of an investigational oral medicine called tirabrutinib for the potential treatment of newly diagnosed or relapsed/refractory (R/R) primary central nervous system lymphoma (PCNSL), which is a type of cancer that either does not improve from treatment (refractory) or improves only for a limited time (relapsed). Current treatment options for R/R PCNSL are limited, and there are no medications specifically approved in the U.S. for the treatment of PCNSL. Learn more about the PROSPECT Study here: theprospectstudy.com.

On May 28, 2025 Invectys, Inc., a clinical-stage biotechnology company pioneering novel cancer immunotherapies, reported that MD Anderson Investigators Drs. Samer Srour and Aung Naing will present a Trial-in-Progress poster at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place May 30–June 3 in Chicago, Illinois (Press release, Invectys, MAY 28, 2025, View Source [SID1234653612]).

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The presentation will detail the ongoing Phase 1/2a clinical trial evaluating IVS-3001, Invectys’ first-in-human chimeric antigen receptor (CAR) T-cell therapy targeting HLA-G, a non-classical MHC class I molecule overexpressed in various solid tumors. HLA-G functions as a powerful immune checkpoint, enabling tumor cells to evade immune surveillance. By engineering CAR T cells to recognize and eliminate HLA-G–expressing cancer cells, IVS-3001 aims to restore the immune system’s ability to combat tumors.

Presentation Details:

Abstract Title: Safety and efficacy of HLA-G–targeted CAR T cells (IVS-3001) in patients with advanced HLA-G–positive solid tumors: Clinical trial in progress
Abstract Number: TPS2679
Session: Developmental Therapeutics—Immunotherapy
Session Type: Poster Session
Poster Board Number: 320b
Date and Time: Monday, June 2, 2025, 1:30 PM – 4:30 PM CDT
Location: McCormick Place Convention Center, Chicago, IL
The Phase 1/2a trial is designed to assess the safety, tolerability, and preliminary efficacy of IVS-3001 in patients with advanced solid tumors expressing HLA-G. The study also aims to evaluate the therapy’s pharmacokinetics and immunogenicity.

"We are excited to share our progress on IVS-3001 at ASCO (Free ASCO Whitepaper) 2025. Patients with solid tumors have profound unmet clinical need. HLA-G is an especially exciting target for cell therapy, as it is both a widely expressed tumor marker and a potent immune checkpoint. Thus, HLA-G directed therapies have the potential to treat a vast variety of solid tumors." said Dr. Jake Kushner, CEO of Invectys. "This study represents a key step forward in our mission to develop innovative immunotherapies that address unmet needs in oncology."

On May 28, 2025 Candel Therapeutics, Inc. (Candel or the Company) (Nasdaq: CADL), a clinical-stage biopharmaceutical company focused on developing multimodal biological immunotherapies to help patients fight cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to CAN-2409 (aglatimagene besadenovec), the Company’s biological immunotherapy lead candidate, for the treatment of newly diagnosed localized prostate cancer in patients with intermediate-to-high-risk disease (Press release, Candel Therapeutics, MAY 28, 2025, View Source [SID1234653433]). CAN-2409 was also previously granted FDA Fast Track designation for the same indication.

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The FDA’s RMAT designation is intended to expedite the development and review of regenerative medicine therapies intended to treat, modify, reverse, or cure serious or life-threatening diseases where preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition. The designation provides opportunities for intensive FDA guidance and organizational commitment to potentially support and expedite drug development. The designation also offers eligibility for mechanisms designed to speed Biologics License Application (BLA) review and approval, with potential opportunities for rolling review and priority review.

The RMAT designation was granted on the basis of the positive data from Candel’s phase 3 randomized, placebo-controlled clinical trial evaluating the efficacy and safety of CAN-2409 plus valacyclovir (prodrug), together with standard of care (SoC) external beam radiation therapy, in newly diagnosed, localized, intermediate-to-high-risk prostate cancer.

Data announced by Candel in December 2024 showed that the phase 3 trial met its primary endpoint and demonstrated statistically significant improvement in disease-free survival (DFS) (p=0.0155) with a 30% reduction (HR 0.70) in the risk for prostate cancer recurrence or death due to any cause in patients who received CAN-2409 plus prodrug, combined with SoC radiotherapy (n=496), compared with patients who received placebo combined with SoC radiotherapy (n=249). CAN-2409 improved prostate-specific DFS with a 38% risk reduction compared with placebo (HR 0.62; p=0.0046). There was also a significant increase in the proportion of patients achieving a prostate-specific antigen (PSA) nadir of <0.2 ng/ml in the CAN-2409 treatment arm compared to the placebo arm (67.1% vs. 58.6%, respectively; p=0.0164). Furthermore, the data showed an 80.4% pathological complete response in the 2-year post-treatment biopsies after CAN-2409 administration compared to 63.6% in the control arm (p=0.0015). The safety profile of CAN-2409 was generally consistent with previous studies, with no new safety signals identified. Key aspects of the study design, including the primary endpoint, were agreed with the FDA under a Special Protocol Assessment (SPA).

"Receiving the FDA’s RMAT designation underscores the critical unmet need in patients with early, localized prostate cancer and validates the promising clinical activity observed with CAN-2409. This designation further supports the design of our phase 3 study, including the DFS primary endpoint agreed upon with the FDA during the SPA negotiation," stated Paul Peter Tak, MD, PhD, FMedSci, President and Chief Executive Officer of Candel.

Dr. Tak continued, "We look forward to collaborating with the FDA to pursue an expeditious approval of CAN-2409 once we submit our BLA—currently anticipated at the end of 2026. Our aim is to introduce a new treatment option for patients at the early stages of prostate cancer, a disease that has seen minimal innovation over the past two decades. We expect the RMAT designation to facilitate the BLA filing process and bring us closer to achieve this objective."

About CAN-2409

CAN-2409 (aglatimagene besadenovec), Candel’s most advanced multimodal biological immunotherapy candidate, is an investigational, off-the-shelf, replication-defective adenovirus designed to deliver the herpes simplex virus thymidine kinase (HSV-tk) gene to a patient’s specific tumor. HSV-tk is an enzyme that locally converts orally administered valacyclovir into a nucleotide analog that kills nearby cancer cells. Together, this regimen is designed to induce an individualized and specific CD8+ T cell-mediated response against the injected tumor and uninjected distant metastases for broad anti-tumor activity, based on in situ immunization against a variety of tumor antigens. Because of its mechanism of action, CAN-2409 has pan solid tumor treatment potential. Encouraging monotherapy activity as well as combination therapy activity with standard of care radiotherapy, surgery, chemotherapy, and immune checkpoint inhibitor (ICI) treatment, have previously been shown in several preclinical and clinical settings. More than 1,000 patients have been dosed with CAN-2409 with a favorable tolerability profile to date, supporting the potential for combination with standard of care, when indicated.

Candel has recently completed a successful phase 3 clinical trial of CAN-2409 in localized prostate cancer and positive phase 2a clinical trials of CAN-2409 in non-small cell lung cancer (NSCLC) and borderline resectable pancreatic ductal adenocarcinoma (PDAC). CAN-2409 plus prodrug (valacyclovir) has been granted Fast Track Designation by the FDA for the treatment of PDAC, stage III/IV NSCLC in patients who are resistant to first line PD-(L)1 inhibitor therapy and who do not have activating molecular driver mutations or have progressed on directed molecular therapy, and localized primary prostate cancer. CAN-2409, plus prodrug, has also been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA for the treatment of newly diagnosed localized prostate cancer in patients with intermediate-to-high-risk disease. Candel’s pivotal phase 3 clinical trial in prostate cancer was conducted under a SPA agreed with the FDA. The FDA has also granted Orphan Drug Designation to CAN-2409 for the treatment of PDAC.