On April 15, 2025 Pilatus Biosciences, pioneering biologics targeting metabolic checkpoints, reported that it will present new research on its lead candidate, PLT012, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2025 Annual Meeting (Press release, Pilatus Biosciences, APR 15, 2025, View Source [SID1234651947]). The presentations will include an oral symposium by co-founder and Chair of the Scientific Advisory Board (SAB), Prof. Ping-Chih Ho, and a poster session by Lead Scientist, Dr. Yi-Ru Yu. These efforts highlight PLT012’s potential as a novel therapeutic approach for immune-cold solid tumors. PLT012 is currently in late-stage preclinical development and progressing toward its first-in-human clinical trial.

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Event Participation and Presentations

Pilatus Biosciences will participate at the AACR (Free AACR Whitepaper) 2025 Annual Meeting, held April 27-30, 2025, with the following presentations:

Oral presentation: Prof. Ping-Chih Ho, co-founder and Chair of the SAB, will present during the Major Symposium SY21 – Immunometabolism and Metabolic Fitness in Tumors. The talk, titled "Reprogramming the Tumor Microenvironment with a Single Punch – Our Journey from Bench to Bedside", is scheduled for April 28, 2025, at 1:15 PM.
Poster presentation: Dr. Yi-Ru Yu, Lead Scientist, will present Abstract #6077 in Section 37, on April 29, 2025, from 2:00 PM to 5:00 PM. The poster will detail PLT012’s efficacy in addressing unmet needs in immune-cold solid tumors.
CEO Statement

"We are pleased to present our latest research at AACR (Free AACR Whitepaper) 2025," said Dr. Raven Lin, CEO of Pilatus Biosciences. "PLT012 reflects a differentiated approach—one that reawakens the immune system by targeting metabolic pathways, with the goal of extending the power of immunotherapy to patients who currently don’t benefit from it."

"PLT012 has earned FDA Orphan Drug Designation for liver and intrahepatic bile duct cancers. Its ability to reprogram the tumor microenvironment has led us to explore synergistic combinations, including bispecific antibodies (BsAb) and antibody-drug conjugates (ADCs), in preclinical studies across various solid tumor models, addressing critical unmet needs in oncology."

About PLT012

PLT012, is a humanized anti-CD36 antibody with a dual mechanism of action (MOA). It simultaneously inhibits immunosuppressive cell populations and enhances effector T cell function. Preclinical studies as a monotherapy have demonstrated its efficacy in both immune-hot and immune-cold tumor models, with a significant increase in GzmB-expressing CD8+ T cells and reductions in intratumoral Tregs and pro-tumorigenic macrophages. Additionally, PLT012 reshapes the exhaustion profile of cytotoxic CD8+ T cells by expanding both progenitor exhausted (Texprog) and terminally-exhausted (Texterm) populations with rejuvenated effector functions, leading to enhanced tumoricidal immunity. These findings suggest that PLT012, functioning as a metabolic regulator, may provide therapeutic benefits in cancer treatment, either as a monotherapy or in combination with immune checkpoint inhibitors such as PD-1 or PD-L1 inhibitors. Other than oncology, the unique MOA as a metabolic regulator also shows the potential of reprogramming the metabolic environment with associated benefits, e.g. liver functional improvement, thus laying the groundwork for targeting a broader spectrum of metabolic and immunological diseases.

On April 15, 2025 HepaRegeniX GmbH ("HepaRegeniX"), a clinical-stage company advancing novel therapies to treat acute and chronic liver diseases, reported the second closing and completion of a €21.5 million financing round with the addition of Wellington Partners to its investor syndicate. HepaRegeniX plans to use the proceeds to complete its ongoing Phase Ib trial and to advance the Phase IIa clinical trial for HRX-215, the company’s lead clinical candidate in liver regeneration.

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"We continue to make significant progress in the clinical evaluation of our lead candidate, HRX-215, and the additional investment is a strong acknowledgment of our achievements and the impressive safety results of HRX-215 in the completed Phase I trials," said Elias Papatheodorou, Chief Executive Officer at HepaRegeniX. "HepaRegeniX has brought to light the significant therapeutic potential of Mitogen-Activated Protein (MAP) Kinase Kinase 4 (MKK4) in multiple indications of high medical need, and we remain committed to making a real difference in the lives of patients that need liver resections due to tumors, for patients in need of a liver transplant, and for patients with chronic and acute liver diseases. As we gain momentum in our clinical development strategy, we appreciate the continued support of our existing investors and the support of Wellington Partners at this important stage in our journey."

"Wellington Partners is very excited to join and further strengthen the HepaRegeniX investor base in this financing round," commented Dr. Rainer Strohmenger, Managing Partner at the Munich-based life science venture capital fund. "HepaRegeniX’ approach is highly differentiated and has demonstrated efficacy in several in vivo models for liver diseases with extraordinarily high unmet medical need. We look forward to supporting the generation of meaningful clinical efficacy data in human patients in the near future."

HRX-215 is a first-in-class, orally available small molecule inhibitor designed to target Mitogen-Activated Protein (MAP) Kinase Kinase 4 (MKK4), a master regulator of liver regeneration. By selectively inhibiting MKK4, HRX-215 enhances the liver’s natural ability to regenerate. Extensive preclinical studies in mice and pigs have demonstrated that HRX-215 enhances liver regeneration of healthy and diseased livers. Impressively, HRX-215 helps prevent liver failure after extended liver resection. A Phase I clinical trial in healthy participants has further validated its favorable safety and pharmacokinetic profile. HRX-215 represents a new therapeutic approach for indications requiring rapid liver repair, offering a new possibility beyond conventional treatments.

(Press release, HepaRegeniX, APR 15, 2025, View Source [SID1234666458])

On April 14, 2025 Pheast Therapeutics, a private biotechnology company developing novel therapies to unleash the power of macrophages on aggressive, difficult-to-treat cancers, reported that the first patient has been treated in its Phase 1 clinical trial evaluating PHST001, an anti-CD24 macrophage checkpoint inhibitor, in patients with advanced solid tumors (Press release, Pheast Therapeutics, APR 14, 2025, View Source [SID1234651920]).

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"The start of this Phase 1 trial is an important milestone for Pheast and for the advancement of next generation macrophage checkpoint therapies," said Roy Maute, Ph.D., Cofounder and CEO, Pheast Therapeutics. "We believe PHST001 has the potential to provide new treatment options for patients, particularly in cancers where other immunotherapies have not been effective."

The multicenter, open-label Phase 1 study will enroll up to 80 patients with advanced relapsed and/or refractory solid tumors (ClinicalTrials.gov Identifier: NCT06840886). The study’s primary objectives include evaluating safety and tolerability of PHST001 and establishing the recommended Phase 2 dose, with secondary endpoints assessing pharmacokinetics and early signs of anti-tumor activity.

Raphaël Rousseau, M.D., Ph.D., Chief Medical Officer, Pheast Therapeutics, said, "PHST001 has demonstrated robust activity across tumor types and a favorable safety profile in preclinical studies. Furthermore, these data suggest that PHST001 may overcome tumor immune evasion through a differentiated approach to macrophage activation. We are pleased to have initiated this study as a first step in understanding the clinical potential of PHST001 to improve patient outcomes across multiple cancer types."

Irving Weissman, M.D., scientific co-founder of Pheast Therapeutics added, "CD24 is a key mechanism that tumors use to evade macrophage-mediated immune responses. PHST001 was developed based on foundational research into this pathway, and I’m encouraged to see this science progressing into the clinic. It’s a meaningful step toward harnessing the full potential of the innate immune system in cancer."

Preclinical data presented at SITC (Free SITC Whitepaper) 2024 highlight PHST001’s potential across multiple cancer types, and its differentiation as a novel macrophage checkpoint inhibitor uniquely designed to target all glyco-variants of CD24. By binding CD24 with high affinity and specificity, PHST001 promotes macrophage-induced phagocytosis in a variety of cancer cell types and significantly shrinks tumors in in vivo models. Additionally, PHST001 has a favorable pharmacokinetic profile in non-human primates and does not induce immune-mediated toxicity in in vitro studies.

About CD24

CD24 is a cell surface protein that plays a key role in tumor immune evasion by engaging Siglec-10, an inhibitory receptor on macrophages. This interaction suppresses macrophage-mediated clearance of cancer cells, allowing tumors to escape destruction by the innate immune system. CD24 was identified as a novel macrophage checkpoint through foundational work by Dr. Amira Barkal, principal founder of Pheast. Along with other co-founders, Drs. Irving Weissman, Ravi Majeti, and Roy Maute, Pheast’s research opened the door to therapeutic strategies targeting CD24 to drive innate immune responses against cancer.

About PHST001

PHST001 is an anti-CD24 macrophage checkpoint inhibitor designed to overcome immune suppression in the tumor microenvironment. CD24 is highly expressed by many human cancers, including ovarian and triple negative breast cancer (TNBC), and high expression of CD24 is a negative prognostic factor in multiple cancer indications. Pheast has engineered PHST001 to be a best in class antibody designed to induce macrophages to phagocytose cancer cells and initiate a powerful immune response.

On April 14, 2025 Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, reported that it has received a Notice of Allowance from the United States Patent and Trademark Office (USPTO) for a new patent application covering its chimeric antigen receptor-T cell (CAR-T) technology (Press release, Anixa Biosciences, APR 14, 2025, View Source [SID1234651904]).

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The allowed claims in this patent encompass core methods and compositions that are fundamental to Anixa’s innovative CAR-T approach. Anixa’s CAR-T platform is specifically designed to address the long-standing challenges of applying CAR-T therapies to solid tumors, positioning the program as a potential breakthrough in immuno-oncology. This patent, along with others, was granted to The Wistar Institute and exclusively licensed to Anixa Biosciences. Anixa’s CAR-T technology is currently in a clinical trial at Moffitt Cancer Center, treating recurrent ovarian cancer patients.

Dr. Amit Kumar, Chairman and CEO of Anixa Biosciences, stated, "This Notice of Allowance further strengthens our growing intellectual property portfolio and reinforces the potential of our robust CAR-T program. Securing foundational patent protection is a vital step in supporting the program’s future success and in driving the development of next-generation immunotherapies with the potential to deliver transformative outcomes for patients."

On April 14, 2025 REVEAL GENOMICS and Ona Therapeutics reported a strategic collaboration aimed at accelerating the clinical development of ONA-255, a next-generation ADC designed to enhance the precision and efficacy of cancer treatment across multiple tumor types (Press release, Ona Therapeutics, APR 14, 2025, View Source [SID1234651921]).

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ONA-255 is positioned to be a first-in-class molecule against a novel ADC target present in multiple, prevalent solid tumor types. Ona has privileged insight of the target as a hallmark of molecular adaptation in advanced diseases and its unique function as a universal driver of resistance to various therapies. ONA-255 is an ADC tailor-made to match the design of the molecule to the extraordinary biology of the target to deliver exceptionally potent drug against recalcitrant tumors, especially resistant clones for deeper and more durable responses.

At the heart of this collaboration is Dr. Aleix Prat, Co-Founder and Chief Scientific Officer of REVEAL GENOMICS. A globally recognized expert in precision oncology and drug development, Dr. Prat played a key role in identifying the ONA-255 drug target and generating, in collaboration with Ona Therapeutics and Dr. Roger Gomis, the critical preclinical data necessary for its clinical advancement. He also serves as the head of Ona Therapeutics’ advisory board.

As a leader in genomic innovation, REVEAL GENOMICS will conduct a comprehensive molecular analysis of tumor and blood samples from the ONA-255 phase 1-2 clinical trial as part of this strategic collaboration. Leveraging proprietary technologies and advanced computational algorithms, the company will decode key molecular and genomic biomarkers to reveal insights into tumor biology, the immune microenvironment, and response mechanisms to ONA-255. This pioneering work will help define patient populations, identify predictive biomarkers, and deepen the understanding of ONA-255’s mechanism of action—accelerating its clinical development and opening new therapeutic avenues.

To advance this partnership, Ona Therapeutics—working in collaboration with the U.S. subsidiary of REVEAL GENOMICS—has been awarded a grant from CDTI (Centro para el Desarrollo Tecnológico y la Innovación), a public entity under the Spanish Ministry of Science, Innovation, and Universities that promotes technological innovation and development. The grant is funded by the Plan de Recuperación, Transformación y Resiliencia – Funded by the European Union NextGenerationEU.

Valerie Vanhooren, Co-Founder and CEO of Ona Therapeutics, added, "ADCs have transformed cancer treatment; however, the technology has been applied to a limited number of tumor targets. These limitations restrict the number of patients who can benefit from treatments and highlight the critical need to identify new broadly expressed tumor targets. Ona’s tailor-designed ADCs have the potential to define new treatment paradigms and patient populations, resulting in a clear benefit for patients with cancer. This partnership with REVEAL GENOMICS combines Ona’s ADC experience with cutting-edge diagnostics with the goal to transform the treatment landscape for aggressive solid tumors."

Patricia Villagrasa, Co-Founder and CEO of REVEAL GENOMICS, stated, "Ona Therapeutics is at the forefront of developing next-generation cancer therapies. This collaboration marks a key milestone in combining advanced diagnostics with innovative treatments, reinforcing the strategic value of integrating biomarker science early in drug development. It also underscores REVEAL GENOMICS’s leadership in driving biomarker strategy and our commitment to shaping new business models that bring precision oncology closer to patients."

Dr. Aleix Prat commented, "I am thrilled about this collaboration, which bridges groundbreaking therapies with precision medicine. By leveraging our combined expertise, we can gain deeper insights into ONA-255’s efficacy, paving the way for more targeted and impactful cancer treatments."