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 IDEAYA Biosciences, Inc. (Nasdaq:IDYA), a precision medicine oncology company committed to the discovery and development of targeted therapeutics, reported a successful FDA Type D meeting on the Phase 3 registrational trial design that will assess the safety and efficacy of darovasertib for potential regulatory approval as neoadjuvant therapy for primary uveal melanoma (UM) (Press release, Ideaya Biosciences, APR 14, 2025, View Source [SID1234651919]).

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"The successful FDA Type D meeting provides darovasertib a registrational path as neoadjuvant therapy for UM, using primary clinical endpoints of eye preservation and proportion of patients with vision loss, with no detriment to EFS as a secondary endpoint required for both cohorts. Based on the promising clinical efficacy and safety observed with darovasertib in the neoadjuvant setting in over 90 patients and the recent Breakthrough Therapy Designation by the US FDA, we are excited to advance the darovasertib program into our second registrational trial," said Dr. Darrin Beaupre, M.D., Ph.D., Chief Medical Officer, IDEAYA Biosciences.

Darovasertib is a potent and selective protein kinase C (PKC) inhibitor being developed to broadly address primary and metastatic UM (MUM). Darovasertib has received U.S. FDA Breakthrough Therapy Designation as neoadjuvant therapy in enucleation recommended primary UM and Fast Track designation for darovasertib in combination with crizotinib in adult patients with metastatic uveal melanoma (MUM), where a Phase 2/3 registration-enabling trial of the darovasertib and crizotinib combination in 1L HLA-A2-negative MUM is ongoing. Darovasertib has also been designated as an Orphan Drug by the U.S. FDA in UM, including in MUM.

FDA Guidance from the Type D Meeting on the Phase 3 Neoadjuvant Darovasertib Registrational Trial Design for Potential Regulatory Approval in Primary UM

IDEAYA is targeting to initiate the Phase 3 randomized clinical trial evaluating neoadjuvant darovasertib in primary UM in the first half of 2025. The randomized Phase 3 clinical trial design incorporates guidance and feedback from the U.S. FDA following a recent Type D meeting.

In the Phase 3 clinical trial, we currently project approximately 520 patients will be randomized 2:1 to the darovasertib treatment versus control arm. There will be 2 cohorts enrolled: 1) 120 enucleation eligible UM patients, 2) 400 PB eligible UM patients. For the enucleation cohort, the randomization will be with or without darovasertib as neoadjuvant therapy. For the PB cohort, the randomization will be darovasertib followed by PB versus PB alone.

Key highlights of the Phase 3 registrational trial design in neoadjuvant UM, based on FDA guidance:

Eye preservation rate (exceed lower bound of 10% eye preservation rate with a 95% confidence interval) is the primary endpoint for the enucleation UM cohort
Proportion of patients with vision loss from the time of randomization and time of completion of PB is the primary endpoint for the plaque brachytherapy cohort. Vision detriment will be measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) Best-Corrected Visual Acuity (BCVA) of >15-letters lost
No detriment to Event-Free-Survival (EFS) is a secondary endpoint for both cohorts and is required for approval. No detriment is defined as overlapping confidence intervals
Additional secondary endpoints: Overall Response Rate (>20% ocular tumor shrinkage by product of diameters), proportion of patients with clinically significant macular edema, proportion of subjects with 20/200 vision loss or worse (legal blindness), proportion of subjects with reduction of radiation dose of >20% delivered to key eye structures
Potential to submit the enucleation cohort data for regulatory review earlier than the PB cohort pending the EFS data maturity in both cohorts
300mg BID darovasertib will be the move-forward dose for the registrational trial

On April 14, 2025 Bio-Thera Solutions Inc. (688177:SH), a commercial-stage biopharmaceutical company developing a pipeline of innovative therapies and biosimilars, reported that it will have an oral presentation in collaboration with medical centers at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting entitled "Safety and efficacy of BAT8006, a Folate Receptor α (FRα) antibody drug conjugate (ADC), in patients with platinum-resistant ovarian cancer: Update on the dose optimization/expansion cohort of BAT-8006-001-CR trial" (Press release, BioThera Solutions, APR 14, 2025, View Source [SID1234651918]). Three posters will be also presented in the poster session, covering TROP2-ADC BAT8008, HER2-ADC BAT8010, BAT1006 (an ADCC-enhanced anti-HER2 mAb with a different epitope from BAT8010) , and a novel PD-1 mAb BAT1308 in various solid tumors.

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Presentation details are as follows:

Oral presentation:

Abstract Title: Safety and efficacy of BAT8006, a folate receptor α (FRα) antibody drug conjugate, in patients with platinum-resistant ovarian cancer: Update on the dose optimization/expansion cohort of BAT-8006-001-CR trial.
Abstract ID: 5517
Type: Rapid Oral Abstract-Gynecologic Cancer
Time: 6/3/2025, 8:00 AM-9:30 AM CDT
Poster 1:

Abstract Title: BAT8008, a TROP-2 antibody-drug conjugate (ADC), in patients with advanced solid tumor: Results from a phase 1 study.
Abstract ID: 3024
Type: Poster Session
Time: 06/02/2025 1:30 PM-4:30 PM CDT
Poster 2:

Abstract Title: A phase Ⅰb/Ⅱa study of BAT8010+BAT1006, an anti-HER2 monoclonal antibody-exatecan conjugate combined with an ADCC-enhanced HER2 mAb in patients with advanced solid tumors.
Abstract ID: 1027
Type: Poster Session
Time: 6/2/2025 9:00 AM-12:00 PM CDT
Poster 3:

Abstract Title: Primary efficacy and safety results of BAT1308, a PD-1 inhibitor, + chemotherapy ± bevacizumab in phase 2 trial for persistent, recurrent, or metastatic cervical cancer.
Abstract ID: 2611
Type: Poster Session
Time: 6/2/2025 1:30 PM-4:30 PM CDT

On April 14, 2025 Accord BioPharma, Inc., the U.S. specialty division of Intas Pharmaceuticals, Ltd., focused on the development of oncology, immunology, and critical care therapies, reported that Intas Pharmaceuticals has completed its acquisition of the UDENYCA (pegfilgrastim-cbqv) business from Coherus BioSciences, Inc (Press release, Coherus Biosciences, APR 14, 2025, View Source [SID1234651917]). UDENYCA, a biosimilar to Neulasta (pegfilgrastim), expands the company’s portfolio of FDA-approved products and accelerates Accord BioPharma’s growth in the biosimilar industry.

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By acquiring UDENYCA, Accord BioPharma will continue to make this treatment available to decrease the incidence of infection, as manifested by febrile neutropenia, in patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs. UDENYCA’s three administration options — autoinjector (AI), on-body injector (OBI), and prefilled syringe (PFS) — offer flexibility for patients to receive their treatment at home, on-the-go, or in the office. Since the brand’s commercial launch, more than 300,000 patients are estimated to have been treated with UDENYCA and over 1.4 million units have been sold.

The acquisition not only expands Accord BioPharma’s product offerings, but also brings a wealth of talent from Coherus BioSciences. Key Coherus employees across multiple functions, including Sales, Marketing, Finance, Supply Chain, and Quality and Manufacturing, will join Accord BioPharma, playing a crucial role in supply and services continuity/transition and expanding the company’s work around UDENYCA.

"The completion of the UDENYCA acquisition marks a pivotal moment for Accord BioPharma, as it not only strengthens our market presence but broadens our capabilities as we endeavor to innovate and expand in the biosimilar space," said Chrys Kokino, U.S. President of Accord. "We are delighted to welcome talented employees from Coherus, whose historical knowledge and expertise will complement that of our existing team, making us stronger and better equipped to achieve our goals with UDENYCA."

"We are energized by this addition to Accord BioPharma’s portfolio because it exemplifies our commitment to improving patient access to high-quality treatments that effectively meet the needs of both patients and healthcare providers," said Binish Chudgar, Executive Chairman and Managing Director of Intas Pharmaceuticals. "We are pleased with the successful completion of this agreement and are excited about the future opportunities it brings for Accord BioPharma in the U.S. market."

INDICATION
UDENYCA (pegfilgrastim-cbqv) is a leukocyte growth factor indicated to:

Decrease the incidence of infection, as manifested by febrile neutropenia, in patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with a clinically significant incidence of febrile neutropenia.
Increase survival in patients acutely exposed to myelosuppressive doses of radiation (Hematopoietic Subsyndrome of Acute Radiation Syndrome).
Limitations of Use
UDENYCA is not indicated for the mobilization of peripheral blood progenitor cells for hematopoietic stem cell transplantation.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATION: Patients with a history of serious allergic reactions to pegfilgrastim products or filgrastim products. Reactions have included anaphylaxis.

WARNINGS AND PRECAUTIONS:

Fatal splenic rupture: Evaluate patients who report left upper abdominal or shoulder pain for an enlarged spleen or splenic rupture.
Acute respiratory distress syndrome (ARDS): Evaluate patients who develop fever, lung infiltrates, or respiratory distress. Discontinue UDENYCA in patients with ARDS.
Serious allergic reactions, including anaphylaxis: The majority of reported events occurred upon initial exposure. Allergic reactions, including anaphylaxis, can recur within days after the discontinuation of initial anti-allergic treatment. Permanently discontinue UDENYCA in patients with serious allergic reactions.
Allergies to Acrylics (UDENYCA ONBODY only): The on-body injector (OBI) for UDENYCA uses acrylic adhesive. For patients who have reactions to acrylic adhesives, use of this product may result in a significant reaction.
Sickle cell crises: Severe and sometimes fatal crises have occurred. Discontinue UDENYCA if sickle cell crisis occurs.
Glomerulonephritis: The diagnoses were based upon azotemia, hematuria (microscopic and macroscopic), proteinuria, and renal biopsy. Generally, events resolved after dose reduction or discontinuation. Evaluate and consider dose-reduction or interruption of UDENYCA if causality is likely.
Leukocytosis: White blood cell (WBC) counts of 100 x 109/L or greater have been observed in patients receiving pegfilgrastim products. Monitoring of complete blood count (CBC) during UDENYCA therapy is recommended.
Thrombocytopenia: Thrombocytopenia has been reported in patients receiving pegfilgrastim. Monitor platelet counts.
Capillary Leak Syndrome: Has been reported after G-CSF administration, including pegfilgrastim products, and is characterized by hypotension, hypoalbuminemia, edema, and hemoconcentration. Episodes vary in frequency, severity and may be life-threatening if treatment is delayed. If symptoms develop, closely monitor and give standard symptomatic treatment, which may include a need for intensive care.
Potential for Tumor Growth Stimulatory Effects on Malignant Cells: The granulocyte colony stimulating factor (G-CSF) receptor through which pegfilgrastim products and filgrastim products act has been found on tumor cell lines. The possibility that pegfilgrastim products act as a growth factor for any tumor type, including myeloid malignancies and myelodysplasia, diseases for which pegfilgrastim products are not approved, cannot be excluded.
Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML) in Patients with Breast and Lung Cancer: MDS and AML have been associated with the use of pegfilgrastim in conjunction with chemotherapy and/or radiotherapy in patients with breast and lung cancer. Monitor patients for signs and symptoms of MDS/AML in these settings.
Potential Device failures (UDENYCA ONBODY only): Missed or partial doses have been reported for products administered via on-body injectors due to the device not performing as intended. In the event of a missed or partial dose, patients may be at increased risk of events such as neutropenia, febrile neutropenia and/or infection than if the dose had been correctly delivered. Instruct patients using the OBI to notify their healthcare professional immediately to determine the need for a replacement dose of UDENYCA if they suspect that the device may not have performed as intended.
Aortitis: Has been reported in patients receiving pegfilgrastim products, occurring as early as the first week after start of therapy. Manifestations may include generalized signs and symptoms such as fever, abdominal pain, malaise, back pain, and increased inflammatory markers (e.g., c-reactive protein and white blood cell count). Consider aortitis when signs and symptoms develop without known etiology. Discontinue UDENYCA if aortitis is suspected.
Nuclear Imaging: Increased hematopoietic activity of the bone marrow in response to growth factor therapy has been associated with transient positive bone imaging changes. Consider when interpreting bone imaging results.
ADVERSE REACTIONS: Most common adverse reactions (≥ 5% difference in incidence compared to placebo) are bone pain and pain in extremity.

To report SUSPECTED ADVERSE REACTIONS, contact 1-800-4-UDENYCA (1-800-483-3692) or notify the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

UDENYCA Prefilled Syringe: 6 mg/0.6 mL in a single-dose prefilled syringe for manual use only.
UDENYCA Autoinjector: 6 mg/0.6 mL in a single-dose prefilled autoinjector.
UDENYCA ONBODY: 6 mg/0.6 mL in a single-dose prefilled syringe co-packaged with the on-body injector for UDENYCA.

Full Prescribing Information, including Patient Information and Instructions For Use, is available at www.UDENYCA.com.

Advisors
Smith, Anderson, Blount, Dorsett, Mitchell & Jernigan, L.L.P. acted as legal counsel to Accord and Intas with respect to the Transaction.

On April 14, 2025 Sumitomo Pharma America, Inc. (SMPA) reported it has entered into a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI), part of the U.S. National Institutes of Health (NIH), to evaluate enzomenib, an investigational, oral, small molecule designed to inhibit the menin and KMT2A protein interaction present in certain difficult to treat cancers (Press release, Sumitomo Pharmaceuticals, APR 14, 2025, View Source [SID1234651916]).

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This unique partnership aims to harness the combined expertise of SMPA and the NCI to further expand the scientific evaluation of enzomenib as a potential therapeutic and explore additional cancers to address unmet patient needs.

Enzomenib is an investigational, oral, small-molecule currently being clinically evaluated by SMPA in Phase 1/2 clinical trials for relapsed or refractory acute leukemia. Enzomenib is designed to target the menin and mixed-lineage leukemia protein interaction, a key interaction for acute leukemia and other cancer cell growth. In preclinical studies, enzomenib demonstrated selective growth inhibition in human acute leukemia cell lines with KMT2A (MLL) rearrangements or NPM1 mutations – cancers with particularly high mortality rates and limited treatment options.1,3

As part of the collaboration, enzomenib will be explored in MyeloMATCH (Myeloid Malignancies Molecular Analysis for Therapy Choice), an NCI precision medicine clinical trial designed to improve treatment outcomes for individuals diagnosed with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). Enzomenib will also be explored with other important NCI programs.

"We are highly encouraged by the preliminary data showing promising clinical activity of enzomenib in patients with relapsed/refractory acute leukemia, and with the support and partnership of the National Cancer Institute, we are poised to continue this positive momentum by expanding exploration of its application across additional cancers," said Jatin Shah, M.D., Chief Medical Officer, Oncology, SMPA. "We’re honored to partner with the National Cancer Institute whose scientific expertise, resources and commitment to advancing oncological research will enable an even broader pursuit of scientific innovation on behalf of patients with urgent and unmet needs."

About Enzomenib
Enzomenib is an investigational, oral, small molecule inhibitor of the menin and mixed-lineage leukemia (MLL) protein interaction, a key interaction in acute leukemia and other tumor cell proliferation and growth. Menin is a scaffold nuclear protein which plays key roles in gene expression and protein interactions involved in many biological pathways, including cell growth, cell cycle, genomic stability, and hematopoiesis.1,2 In preclinical studies, enzomenib has shown selective growth inhibition in human acute leukemia cell lines with KMT2A (MLL) rearrangements or NPM1 mutations.1,3 Enzomenib reduced the expression of the leukemia-associated genes HOXA9 and MEIS1, and increased the expression of the differentiation gene CD11b in human acute leukemia cell lines with MLL rearrangements and NPM1 mutation.4,5 The safety and efficacy of enzomenib is currently being clinically evaluated in a Phase 1/2 dose escalation/dose expansion study in patients with relapsed or refractory acute leukemia (NCT04988555). The FDA granted Orphan Drug Designation for enzomenib for the indication of acute myeloid leukemia in June 2022. The FDA granted Fast Track Designation for enzomenib for the indication of relapsed or refractory acute myeloid leukemia with MLLr or NPM1m in June 2024. Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) granted Orphan Drug Designation for enzomenib for the indication of relapsed or refractory acute myeloid leukemia with MLLr or NPM1m in September 2024.