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."

On April 14, 2025 Aulos Bioscience, an immuno-oncology company working to revolutionize cancer care through development of immune-activating antibody therapeutics, reported dosing of its first patient with a combination of AU-007, the anti-PD-1 antibody nivolumab and low-dose, subcutaneous aldesleukin in a Phase 2 expansion cohort focused on second-line treatment of melanoma (Press release, Aulos Bioscience, APR 14, 2025, View Source [SID1234651905]). This new cohort is part of Aulos’ Phase 1/2 clinical trial of AU-007 in patients with unresectable locally advanced or metastatic cancer.

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Preliminary Phase 2 data presented in November at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 39th Annual Meeting showed that a combination of AU-007 and low-dose, subcutaneous aldesleukin is clinically active in patients with melanoma, with durable objective responses achieved. The additional Phase 2 cohort in melanoma now allows the nivolumab combination portion of this study to progress.

"We are excited that the first patient is receiving treatment in this new Phase 2 cohort evaluating AU-007 in combination with nivolumab," said Aron Knickerbocker, Aulos Bioscience’s president and chief executive officer. "Given its unique mechanism of action and the positive data presented to date on AU-007 and low-dose, subcutaneous aldesleukin, we believe that AU-007 holds real promise as a novel immuno-oncology treatment in combination with checkpoint inhibitors in multiple cancer types. These include non-small cell lung cancer, for which we initiated a Phase 2 cohort with the anti-PD-L1 antibody avelumab in November, and now melanoma."

AU-007 is the first human monoclonal antibody designed with the assistance of artificial intelligence to enter a human clinical trial. The antibody harnesses the power of interleukin-2 (IL-2) by binding precisely to the portion of IL-2 that binds to CD25, which prevents IL-2 from binding to high-affinity IL-2 receptors on Tregs, vasculature, pulmonary tissue and eosinophils. This redirects IL-2 to medium-affinity receptors on effector T cells (Teffs) and natural killer (NK) cells, which expand and kill tumor cells.

Aulos anticipates presenting preliminary data from the Phase 2 cohort evaluating AU-007, nivolumab and low-dose, subcutaneous aldesleukin as a second-line treatment for melanoma in the second half of 2025. The company will present new Phase 2 data for AU-007 and low-dose, subcutaneous aldesleukin without a checkpoint inhibitor as a second-line treatment for melanoma at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting later this month.

To learn more about the AU-007 clinical trial program, please visit ClinicalTrials.gov (identifier: NCT05267626). For patients and providers in the U.S., please visit www.solidtumorstudy.com. For patients and health professionals in Australia, please visit www.solidtumourstudy.com.

About AU-007
AU-007 is a human IgG1 monoclonal antibody designed by leveraging artificial intelligence that is highly selective to the CD25-binding portion of IL-2. With a mechanism of action unlike any other IL-2 therapeutic in development, AU-007 redirects IL-2 to reinforce anti-tumor immune effects. This is achieved by preventing IL-2, either exogenous or secreted by effector T cells, from binding to trimeric receptors on regulatory T cells while still allowing IL-2 to bind and expand effector T cells and NK cells. This prevents the negative feedback loop caused by other IL-2-based treatments and biases the immune system toward activation over suppression. AU-007 also prevents IL-2 from binding to CD25-containing receptors on eosinophils, as well as vasculature and pulmonary endothelium, which may significantly reduce the vascular leak syndrome and pulmonary edema associated with high-dose IL-2 therapy.

On April 14, 2025 Applied Therapeutics, Inc. (Nasdaq: APLT) (the "Company"), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, reported financial results for the fourth quarter and full year ended December 31, 2024 (Press release, Applied Therapeutics, APR 14, 2025, View Source [SID1234651931]).

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"We remain focused on preparing for potential regulatory interactions regarding govorestat in both Classic Galactosemia and Sorbitol Dehydrogenase ("SORD") Deficiency," said Les Funtleyder, Interim CEO and CFO of Applied Therapeutics. "As we continue to optimize our strategy for our late-stage programs, we have also made key senior appointments across regulatory, medical and quality affairs functions to bolster our capabilities. We are confident in the promise of govorestat across indications and remain committed to our mission of addressing the unmet needs of patients with rare diseases."

Recent Highlights

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Appointed John H. Johnson as Executive Chairman and Les Funtleyder as Interim Chief Executive Officer. In December 2024, the Company appointed John H. Johnson as Executive Chairman of its Board of Directors. Mr. Johnson is a biopharmaceutical industry veteran with 40 years of transformational leadership experience at global healthcare organizations, including Johnson & Johnson, Eli Lilly & Company, ImClone, and Pfizer, Inc. In connection with Mr. Johnson’s appointment, the Company appointed Les Funtleyder, Chief Financial Officer of the Company, as Interim Chief Executive Officer.

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Appointed Todd F. Baumgartner, MD, MPH as Chief Regulatory Officer and Reena Thomas Colacot as Vice President and Head of Quality. In March 2025, the Company appointed Todd F. Baumgartner, MPD, MPH as Chief Regulatory Officer to lead the Company’s global regulatory strategy. Dr. Baumgartner joins the Company with over 35 years of experience in senior regulatory, clinical development, and medical affairs roles. In January 2025, the Company appointed Reena Thomas Colacot as Vice President and Head of Quality, where she is responsible for overseeing all quality matters, including Good Manufacturing Practices, Good Laboratory Practices, and Good Clinical Practices. Ms. Colacot brings over 25 years of quality leadership experience across the biopharmaceutical and medical device industries.

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Continued Review of Govorestat Development Programs for Classic Galactosemia and SORD Deficiency. As previously disclosed, the Company received a Complete Response Letter ("CRL") from the U.S. Food and Drug Administration ("FDA") for the New Drug Application ("NDA") submitted for govorestat for the treatment of Classic Galactosemia. The Company continues to evaluate its response to the CRL, including any meeting request to discuss appropriate next steps with the FDA regarding the path forward for govorestat for the treatment of Classic Galactosemia. The Company also continues to closely examine the ongoing govorestat development program for the potential treatment of SORD Deficiency and will continue to work with the FDA on the data needed to support an appropriate regulatory pathway, including ongoing work to provide the FDA with support for the potential use of the accelerated approval pathway for SORD Deficiency.

Financial Results

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Cash and cash equivalents totaled $79.4 million as of December 31, 2024, compared with $49.9 million at December 31, 2023.

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Research and development expenses for the year ended December 31, 2024, were $48.7 million, compared to $53.9 million for the year ended December 31, 2023. The decrease of approximately $5.2 million was primarily related to a decrease in clinical, pre-clinical and drug manufacturing and formulation costs, offset by an overall increase in regulatory, personnel and stock-based compensation expenses.

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General and administrative expenses were $56.0 million for the year ended December 31, 2024, compared to $20.6 million for the year ended December 31, 2023. The increase of approximately $35.4 million was primarily related to an increase in commercial, legal and professional, data storage, personnel and stock-based compensation expense, offset by an overall decrease in insurance expense.

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Net loss for the year ended December 31, 2024, was $105.6 million, or $0.76 per basic and diluted common share, compared to a net loss of $119.8 million, or $1.42 per basic and diluted common share, for the year ended December 31, 2023.