On April 25, 2025 Leap Therapeutics, Inc. (Nasdaq:LPTX), a biotechnology company focused on developing targeted and immuno-oncology therapeutics, reported it will present preclinical data of FL-501 in a poster presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting taking place April 25-30 in Chicago, Illinois (Press release, Leap Therapeutics, APR 25, 2025, View Source [SID1234652153]).

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FL-501 is a potential best-in-class monoclonal antibody targeting growth differentiation factor 15 (GDF-15), a cytokine that is implicated in multiple diseases and therapeutic areas, including cancer cachexia.

"Cancer cachexia is a devasting and potentially life-threatening condition characterized by significant weight loss, muscle wasting, fatigue, and severely reduced quality of life. It is a major contributor to cancer-related mortality, and unfortunately there are no effective treatment options available to patients," said Jason Baum, PhD, Chief Scientific Officer of Leap. "These data not only demonstrate that FL-501 is a novel and potential best-in-class anti-GDF-15 antibody, but also capable of fully restoring body composition in preclinical models that is comparable or better than other, clinical-stage antibodies. We look forward to progressing the development of FL-501 and bringing the asset into the clinic in 2026."

Key Findings:

In humanized FcRn mouse studies, FL-501 demonstrated a 2-3-fold longer half-life and 50% reduced clearance compared to its wild-type precursor and ponsegromab
In mouse cachexia models using GDF-15-overexpressing colorectal cancer cells, FL-501 fully restored body composition, comparably or better than clinical-stage antibodies visugromab and ponsegromab
In a non-small cell lung cancer patient-derived xenograft model, FL-501 effectively countered cisplatin-induced weight loss, restoring body weight, composition, and condition scores
These findings confirm GDF-15’s role in cachexia and support FL-501’s advancement in development
Poster Details:

Title: FL-501 is a potential best in class GDF-15 inhibitor with extended half-life and potent anti-cachexia activity in preclinical models
Presenter: Roma Kaul, PhD, Leap Therapeutics
Session Category: Experimental and Molecular Therapeutics
Session Title: New and Emerging Cancer Drug Targets
Date and Time: Tuesday, April 29, 2025, 9:00 a.m. – 12:00 p.m. CT
Poster Board Number: 15
Published Abstract Number: 4258

About FL-501
FL-501 is a potential best-in-class monoclonal antibody in preclinical development that targets growth differentiation factor-15 (GDF-15), a cytokine that is produced at elevated levels in response to various stresses, including chronic inflammation, obesity, cardiovascular diseases, cancers, and chemotherapy treatment. High GDF-15 expression is associated with cancer cachexia including loss of appetite, nausea and weight loss. FL-501 was engineered for higher affinity to GDF-15 and longer plasma half-life compared to competing therapies. In addition to cachexia, FL-501 may be able to reverse immunosuppression in cancers where elevated GDF-15 is correlated with poor survival, as well as play a role in treating other GDF-15-related diseases. FL-501 is being developed through a collaboration agreement with Adimab.

On April 25, 2025 Repare Therapeutics Inc. ("Repare" or the "Company") (Nasdaq: RPTX), a leading clinical-stage precision oncology company, reported the acceptance of two abstracts for mini-oral presentation and the acceptance of four abstracts for poster presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, taking place April 25-30, 2025, in Chicago, Illinois (Press release, Repare Therapeutics, APR 25, 2025, View Source [SID1234652170]).

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Mini-Oral Presentation Details:
Title: Efficacy and safety of the combination PKMYT1-inhibitor lunresertib and ATR-inhibitor camonsertib in patients with ovarian and endometrial cancers: Phase I MYTHIC study (NCT04855656)
Presenter: Alison M. Schram, MD, Memorial Sloan Kettering Cancer Center
Session: Innovative Approaches to Key Molecular Targets
Session Date and Time: Tuesday, April 29 from 2:30-4:30 p.m. CT
Location: Room S406 (Vista Ballroom)
Abstract Number: CT262

Title: The PLK4 inhibitor RP-1664 drives centriole modulation and single agent tumor regressions in preclinical neuroblastoma models
Presenter: John M. Maris, MD, Children’s Hospital of Philadelphia
Session: Advancing the Science of Childhood Cancers: From Bench to Bedside
Session Date and Time: Sunday, April 27 from 3:00-5:00 p.m. CT
Location: Room E353 C
Abstract Number: 1201

Poster Presentation Details:

Title: A dual mechanism of sensitivity to PLK4 inhibition by RP-1664 in neuroblastoma
Presenter: Michal Zimmermann, PhD, Repare Therapeutics
Session: Cell Cycle Effects of Anticancer Drugs
Session Date and Time: Sunday, April 27 from 2:00-5:00 p.m. CT
Location: Poster Section 17
Poster Number: 9
Abstract Number: 365

Title: RP-1664: A potent and selective PLK4 inhibitor causing tumor regressions in TRIM37-high xenograft models of solid tumors
Presenter: Anne Roulston, PhD, Repare Therapeutics
Session: Kinase and Phosphatase Inhibitors 1
Session Date and Time: Monday, April 28 from 9:00 a.m-12:00 p.m. CT
Location: Poster Section 21
Poster Number: 9
Abstract Number: 1734

Title: Pan-cancer analysis of TRIM37 copy-number and development of fit-for-screening in situ hybridization tools
Presenter: Isabel Soria-Bretones, PhD, Repare Therapeutics
Session: Diagnostic Biomarkers 2
Session Date and Time: Sunday, April 27 from 2:00-5:00 p.m. CT
Location: Poster Section 31
Poster Number: 2
Abstract Number: 717

Title: Targeting CCNE1 amplification in gastric cancer
Presenter: Sung Joo Jang, Columbia University Irving Medical Center
Session: Protein Kinases and Phosphatases as Targets for Therapy
Session Date and Time: Wednesday, April 30 from 9:00 a.m.-12:00 p.m. CT
Location: Poster Section 24
Poster Number: 4
Abstract Number: 6942

A copy of each poster presentation is available on the Scientific Resources page of the Repare Therapeutics website and a copy of each mini-oral presentation will be available on the Scientific Resources page of the Repare Therapeutics website at the start of each mini-oral session.

On April 24, 2025 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported its financial results for the first quarter of fiscal year 2025 (Press release, Chugai, APR 24, 2025, View Source [SID1234652101]).

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"The first quarter of 2025 got off to a strong start with increased revenue and profit year-on-year. In Japan, despite the impact of NHI drug price revisions and generic penetration, our new products Phesgo and PiaSky, along with our mainstay product Vabysmo, performed well. Overseas sales increased, with exports of our mainstay products Hemlibra and Actemra growing significantly, driving overall growth. In development pipeline progress, LUNSUMIO, in-licensed from Roche, has been launched in Japan for the treatment of relapsed or refractory follicular lymphoma. For in-house projects, NEMLUVIO (nemolizumab), out-licensed to Galderma received approval in Europe, while orforglipron, out-licensed to Eli Lilly, met its primary endpoint in a Phase III clinical trial for type 2 diabetes, each making significant progress. NXT007, one of our most important development projects achieved Proof of Concept (PoC)* for hemophilia A, and we are accelerating preparations for the earliest possible initiation of Phase III trials. Additionally, three in-house projects of Enspryng, RAY121, and MINT91 initiated new clinical trials. We will continue to strongly promote our unique and innovative drug development to deliver new value to patients worldwide," said Dr. Osamu Okuda, Chugai’s President and CEO.

Chugai reported an increase in revenue of 21.8% and in operating profit of 36.6% year-on-year for the first quarter of 2025 (Core-basis), primarily driven by significant growth in overseas sales.

Regarding revenue, domestic sales remained nearly flat with a slight decrease of 0.2% year-on-year. In the oncology field, sales decreased by 5.3% compared to the previous year. While our new product Phesgo performed well, this was offset by the decline in Perjeta and Herceptin along with the market penetration of Phesgo which includes the same active pharmaceutical ingredients. Additionally, products including our mainstay product Avastin were affected by NHI drug price revisions and biosimilars. In the specialty field, sales increased by 6.2% year-on-year, driven by the strong performance of our mainstay product Vabysmo and successful market penetration of our new product PiaSky. Overseas sales increased significantly by 54.7% year-on-year, driven by a substantial increase in exports of Hemlibra and Actemra to Roche. Other revenue decreased by 11.7%, despite the increase in royalty income related to Hemlibra, mainly due to a decrease in one-time income, etc.

Cost to sales ratio improved by 1.8 percentage points year-on-year to 33.7%, mainly due to a change in the product mix. Research and development expenses and selling, general and administration expenses remained at levels comparable to the previous year. Other operating income (expense) resulted in a gain of ¥0.3 billion. As a result, Core operating profit totaled ¥139.5 billion (+36.6%).

Chugai made good progress in both early and late-stage development activities.

In early-stage development of in-house products that will drive mid to long-term growth, Enspryng (for Duchenne muscular dystrophy) has initiated Phase II clinical trials. Additionally, RAY121 and MINT91 (for solid tumors) have each entered Phase I clinical trials. Furthermore, NXT007 (for hemophilia A) has achieved an important milestone by obtaining PoC. In-house products out-licensed to third parties excluding Roche also progressed steadily. Orforglipron, an oral GLP-1 agonist out-licensed to Eli Lilly, has shown favorable results in a Phase III clinical trial for type 2 diabetes. NEMLUVIO, being developed overseas by Galderma, has received approval in Europe for moderate-to-severe atopic dermatitis and prurigo nodularis.
For products in-licensed from Roche, LUNSUMIO has been launched in Japan for the treatment of relapsed or refractory follicular lymphoma. Tecentriq has received approval for an expanded indication for alveolar soft part sarcoma, while Vabysmo and Evrysdi have received approvals for new formulations. Trontinemab, under development for Alzheimer’s disease, has shown favorable results in Phase I/II clinical trials.

* Proof of Concept (A demonstration that the therapeutic effect conceived in the research stage is effective in humans)

[2025 first quarter results]

Billion JPY 2025
Jan – Mar 2024
Jan – Mar % change
Core results
　Revenue 288.5 236.9 +21.8%
　　Sales 259.7 204.5 +27.0%
　　Other revenue 28.7 32.5 -11.7%
　Operating profit 139.5 102.1 +36.6%
　Net income 99.2 76.0 +30.5%
IFRS results
　Revenue 288.5 236.9 +21.8%
　Operating profit 136.7 99.9 +36.8%
　Net income 97.2 74.4 +30.6%
[Sales breakdown]

Billion JPY 2025
Jan – Mar 2024
Jan – Mar % change
Sales 259.7 204.5 -29.8%
　Domestic sales 103.0 103.2 -0.2%
　　Oncology 53.1 56.1 -5.3%
　　Specialty 49.9 47.0 +6.2%
　Overseas sales 156.7 101.3 +54.7%
[Oncology field (Domestic) Top5-selling medicines]

Billion JPY 2025
Jan – Mar 2024
Jan – Mar % change
　Tecentriq 13.8 14.5 -4.8%
　Polivy 7.5 7.4 +1.4%
　Alecensa 7.5 6.6 +13.6%
　Phesgo 6.8 3.2 +112.5%
　Avastin 6.1 8.7 -29.9%
[Specialty field (Domestic) Top5-selling medicines plus Ronapreve]

Billion JPY 2025
Jan – Mar 2024
Jan – Mar % change
　Hemlibra 12.6 12.5 +0.8%
　Actemra 10.9 10.2 +6.9%
　Enspryng 6.1 5.8 +5.2%
　Vabysmo 5.4 4.0 +35.0%
　Evrysdi 3.4 3.4 +0.0%
[Progress in R&D activities from Jan 30th, 2025 to Apr 24th, 2025]

2024 Q1 R&D Progress
About Core results

Chugai discloses its results on a Core basis from 2013 in conjunction with its decision to apply IFRS. Core results are the results after adjusting Non-Core items to IFRS results. Chugai’s recognition of non-recurring items may differ from that of Roche due to the difference in the scale of operations, the scope of business and other factors. Core results are used by Chugai as an internal performance indicator, for explaining the underlying business performance both internally and externally, and as the basis for payment-by-results such as a return to shareholders.

Trademarks used or mentioned in this release are protected by law.

On April 24, 2025 Akeso, Inc. (9926.HK) ("Akeso" or the "Company") reported that the U.S. Food and Drug Administration (FDA) has approved its differentiated PD-1 monoclonal antibody, penpulimab-kcqx, in combination with cisplatin or carboplatin and gemcitabine for the first-line treatment of adult recurrent or metastatic non-keratinizing nasopharyngeal carcinoma (NPC) (Press release, Akeso Biopharma, APR 24, 2025, View Source [SID1234652118]). FDA also approved penpulimab-kcqx as a single agent for adults with metastatic non-keratinizing NPC with disease progression on or after platinum-based chemotherapy and with least one other prior line of therapy. Penpulimab-kcqx was developed independently by Akeso, with further development and commercialization managed through a joint venture with Chia Tai-Tianqing Pharmaceutical Group.

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This milestone marks penpulimab-kcqx as Akeso’s first internally developed innovative biologic to receive FDA approval. The approval underscores the robust clinical research behind penpulimab-kcqx and marks Akeso’s successful entry into the United States regulatory system for the first time. This achievement highlights the company’s innovative drug development capabilities and its commitment to adhering to the highest international standards in pharmaceutical quality management.

The FDA’s approval of penpulimab-kcqx validates Akeso’s international drug development strategy and expansion capabilities. This approval lays a strong foundation for Akeso’s continued clinical development efforts in the global therapeutics markets.

Penpulimab-kcqx has been approved in China for two indications: 1. first-line treatment of advanced NPC, and 2. second or later line treatment of advanced NPC. The recent FDA approval of penpulimab-kcqx offers a new, immunotherapy option for advanced NPC patients in the US.

The FDA approval is based on the international Phase III clinical trial AK105-304 and the pivotal AK105-202 study, which supported the two Biologics License Application (BLA) for penpulimab-kcqx. These studies demonstrated the drug’s clinical benefits and favorable safety profile across two stages of treatment for metastatic NPC. AK105-304 is a randomized, double-blind, international Phase III trial that enrolled NPC patients of diverse ethnicities. The data will be presented at the 2025 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. Previously, the FDA granted penpulimab-kcqx Breakthrough Therapy Designation (BTD), Orphan Drug Designation (ODD), and Fast Track Designation (FTD) for NPC treatment, highlighting the critical unmet need for this therapy.

According to the WHO 2020 Global Cancer Statistics, over 133,000 new NPC cases are diagnosed annually worldwide, with more than 70% of the patients presented with locally advanced disease. Recurrent or metastatic NPC has a poor prognosis and limited survival. Penpulimab-kcqx’s FDA approval will expand the number of NPC patients that can benefit from its treatment.

Prof. Chaosu Hu, Principal Investigator of penpulimab-kcqx from Fudan University Shanghai Cancer Center, commented: "This milestone enhances international treatment guidelines for advanced NPC and extends the benefits of China’s innovations to global patients, ultimately reshaping the treatment landscape for metastatic NPC worldwide."

Prof. Xiaozhong Chen, Investigator of penpulimab-kcqx from Zhejiang Cancer Hospital, added: "The FDA approval of penpulimab-kcqx confirms its high efficacy and low toxicity, positioning China’s innovative drug development in alignment with international standards."

Dr. Yu Xia, Founder, Chairwoman, President & CEO of Akeso, expressed: "We are very excited by the approval of penpulimab-kcqx’s approval in the US FDA for first line and later line NPC. Beyond reaching our first international regulatory milestone, this approval also provides an important immunotherapy treatment option for patients with NPC in the United States. The FDA approval of penpulimab-kcqx not only highlights the quality of our innovation but also underscores Akeso’s focus on delivering treatments for difficult to treat cancers for patients around the world. We are deeply grateful to all the researchers, participants, and patients who have contributed to this success. Akeso will continue to advance first and best in class therapies, including bispecific antibodies and CD47 inhibitors, challenge global standards of care and unlocking the full potential of our pipeline for cancer patients everywhere."

On April 24, 2025 BriaCell Therapeutics Corp. (Nasdaq: BCTX, BCTXW) (TSX: BCT) ("BriaCell" or the "Company"), a clinical-stage biotechnology company developing novel immunotherapies to transform cancer care, reported the sustained complete resolution of the lung metastasis, first reported in February 2025 , two months after initial treatment in the ongoing Phase 1/2 Bria-OTS study (Press release, BriaCell Therapeutics, APR 24, 2025, View Source [SID1234652431]). The latest data at four months also demonstrates stable disease elsewhere.

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The patient, a 78-year-old woman with metastatic breast cancer (hormone receptor positive, HER2 negative), had failed several prior lines of therapy and received the lowest dose level in the Phase 1/2a Bria-OTS study. At enrollment on November 21, 2024, she had extensive metastases including bone, lymph node and lung involvement. Following Bria-OTS intradermal injections every 2 weeks for six weeks (4 total doses), and subsequent dosing every 3 weeks, the lung metastasis completely resolved with stable disease elsewhere. This response is now confirmed and shows the potentially promising activity of the Bria-OTS platform as monotherapy.

"Despite recent advancements with Antibody-drug-conjugates (ADCs) and immune check point inhibitors (CPIs), many patients, including those with HR+ disease, like BriaCell’s first OTS patient, have very few options," stated Neal S. Chawla MD, Director at the Sarcoma Oncology Center, Santa Monica, Ca., and Principal Investigator for the Bria-OTS study. "We are thrilled with our initial data with single agent Bria-OTS showing rapid and strong anti-tumor activity in an HR+ patient and look forward to continuing this novel approach in patients with MBC, and other cancers."

"This unprecedented anti-cancer response in the first patient dosed with Bria-OTS is an important milestone for us and provides early validation of BriaCell’s personalized immunotherapy approach," stated Dr. William V. Williams, BriaCell’s President and CEO.

Bria-OTS is a personalized off-the-shelf immunotherapy, currently under investigation in a Phase 1/2a dose escalation study (ClinicalTrials.gov identifier: NCT06471673 ) in metastatic recurrent breast cancer. Bria-OTS represents a personalized, next generation, advancement of BriaCell’s lead candidate Bria-IMT which is currently in a pivotal Phase 3 study for metastatic breast cancer. The Phase 1/2a clinical trial in metastatic breast cancer is a dose escalation study initially evaluating the safety and efficacy of Bria-OTS as monotherapy and will be followed by Bria-OTS in combination with an immune checkpoint inhibitor.