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Kairos Pharma Ltd. Announces Completed Enrollment of the Safety Arm of Its Phase 2 Clinical Trial of ENV105 in Metastatic, Castration-Resistant Prostate Cancer

On March 31, 2025 Kairos Pharma, Ltd. (NYSE American: KAPA), a clinical-stage biopharmaceutical company (the "Company’"), reported the completion of the safety lead-in of its Phase 2 clinical trial of ENV105 for the treatment of metastatic, castration-resistant prostate cancer (Press release, Kairos Pharma, MAR 31, 2025, View Source [SID1234651703]). The trial, titled, "Phase II study of Apalutamide with Carotuximab (ENV105) in Metastatic, Castration Resistant Prostate Cancer," began with the safety lead-in, which combined apalutamide, a standard of care for prostate cancer, with the Company’s compound ENV105.

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The second part of the study, which randomizes patients to receive either apalutamide alone or in combination with ENV105 is ongoing. The Company expects to announce the safety and efficacy data readout from the safety arm of the trial beginning in the first half of 2025.

"This is an important milestone in this trial as it helps to establish the safety and preliminary efficacy of the combination therapy with ENV105, which is essential in order to proceed with the randomized portion of the trial. We are very pleased with the rate of our progress thus far and are grateful to our participating centers for their continued dedication to the trial," said John Yu, M.D., Kairos CEO.

The Company is also validating biomarkers throughout the study to best identify those patients believed to have the most benefit for the combination therapy. In the meantime, the Company continues to enroll patients for the randomization arm of the study at key cancer centers in Los Angeles and Salt Lake City. The trial is supported by Kairos Pharma Ltd. and a grant from the National Cancer Institute (NCI).

Adicet Bio to Participate in 2025 Canaccord Genuity Horizons in Oncology Virtual Conference

On March 31, 2025 Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer, reported that Chen Schor, President and Chief Executive Officer, will participate in a panel at the Canaccord Genuity Horizons in Oncology Virtual Conference being held April 7, 2025 (Press release, Adicet Bio, MAR 31, 2025, View Source [SID1234651702]).

Details of the event are as follows:
Panel: "CAR T Approaches in the Autoimmune Space"
Date: Monday, April 7, 2025
Time: 3:00 p.m. ET

OS Therapies Provides Regulatory Update on Rare Pediatric Cancer Immunotherapy Candidate OST-HER2 for Human Osteosarcoma

On March 31, 2025 OS Therapies (NYSE-A: OSTX) ("OS Therapies" or "the Company"), a clinical-stage immunotherapy and Antibody Drug Conjugate (ADC) biopharmaceutical company, reported a regulatory update for its OST-HER2 listeria monocytogenes (Lm) immunotherapeutic cancer biologic drug candidate in the prevention or delay of fully-resected, recurrent, lung metastatic osteosarcoma (Press release, OS Therapies, MAR 31, 2025, View Source [SID1234651701]).

"We are making rapid progress in putting together an appropriate data package to achieve accelerated approval for OST-HER2 given the feedback we’ve received to date from the FDA," said Paul Romness, MHP, Chairman & CEO of OS Therapies. "We know one of the US government’s stated priorities is to treat deadly childhood cancers, and we believe that OST-HER2 for osteosarcoma aligns well with that mission."

Regulatory Timelines

The Company initiated regulatory correspondence with the US Food & Drug Administration (FDA) and the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) in the first quarter of 2025. The Company intends to initiate regulatory interaction with the European Medicines Agency (EMA) and EMA National Competent Authorities in the second quarter of 2025.

FDA

Q1/25: Regulatory communication regarding endpoints for accelerated approval
Q2/25: End of Phase 2 Meeting
Q3/25: Initiation of rolling BLA submission
Q4/25: Conditional BLA via Accelerated Approval Program
MHRA

Q1/25: Scientific Advice Meeting requested and granted
Q3/25: Scientific Advice Meeting with MHRA and ILAP application submission
Q4/25: Application for joint Scientific Advice Meeting with MHRA and National Institute for Health and Care Excellence (NICE)
Q4 25: MHRA Conditional Marketing Authorisation application.
EMA

Q2/25: EMA National Competent Authority Scientific Advice Meeting Request (Medicines Evaluation Board, Netherlands)
Q3/25: EMA PRIME and EMA Orphan Designation applications
Q4/25: EMA-FDA Parallel Scientific Advice application
Q1 26: EMA Conditional Marketing Authorisation application.
The Company has recently buttressed its regulatory and clinical strategy & operations infrastructure with the addition of key consulting agencies with significant track records in rare pediatric cancer, Priority Review Voucher (PRV) program approvals, and worldwide Conditional Market Access Applications including in the EU and UK.

The Company reiterates its commitment to seeking to obtain FDA approval for OST-HER2 in the rare pediatric cancer osteosarcoma in 2025. The Company is eligible to receive a Priority Review Voucher (PRV) if OST-HER2 is approved under its Rare Pediatric Disease Designation (RPDD) by September 30, 2026.

The Company has sufficient cash on hand to operate into 2026.

Potential Treatment Response Biomarkers from Canine Osteosarcoma Program

The Company’s canine osteosarcoma program recently made significant progress in understanding which biomarkers likely drive anti-tumor activity in prevention of recurrence of metastases, metastasis treatment (lung resection is not standard of care in canines) and primary tumor treatment to achieve limb sparing in dogs. The data generated creates the potential for the eventual expansion of the uses of OST-HER2 in osteosarcoma into treatment of unresectable lung metastases and limb sparing prior to surgical resection.

The Company intends to evaluate these biomarkers as potentially predictive of treatment response in humans in preparation for regulatory discussions with FDA, MHRA and EMA. Canine equivalent biomarkers have previously been used as surrogate endpoints for rare diseases in Comparative Oncology, which is the study of diseases that occur in humans and animals and have significant genetic homology. Human and Canine osteosarcoma have 96% genetic homology.

The Company is preparing to initiate regulatory correspondence with the United States Department of Agriculture (USDA) with the Company’s updated manufacturing process which incorporates key improvements covered under a patent that was recently granted a notice of allowance by the United States Patent & Trademark Office (USPTO).

Shelter Me is a film that was produced to highlight the important role of human and canine Comparative Oncology disease research. The movie will premiere on April 3, 2025 at AMC Century City in Los Angeles and will later be available to stream through the PBS app and on PBS.org.

OS Therapies Reports Full Year 2024 Financial Results and Provides Business Update

On March 31, 2025 OS Therapies Inc. (NYSE-A: OSTX) ("OS Therapies" or "the Company"), a clinical-stage cancer immunotherapy and antibody drug conjugate biotechnology company, reported full-year 2024 financial results ended December 31, 2024 and provided a business update (Press release, OS Therapies, MAR 31, 2025, View Source [SID1234651700]).

"2024 was a transformative year for OS Therapies as we completed our initial public offering and finalized the treatment phase of our Phase 2b clinical trial in the prevention of recurrence in fully resected, lung metastatic osteosarcoma," said Paul Romness, MPH, Chairman & CEO of OS Therapies. "In parallel, the Company entered into an agreement to acquire all of the listeria monocytogenes (Lm)-based assets from Ayala Pharmaceuticals, supported the development of additional data for OST-HER2 in canines and positioned the Company’s tunable antibody drug conjugate (tADC) program for partnering. The Company remains primarily focused on gaining a Biologics Licensing Authorization (BLA) for OST-HER2 in osteosarcoma that would trigger the issuance of a Priority Review Voucher (PRV) that the Company expect to then divest in order to capitalize with non-dilutive capital to allow the Company to fully exploit the OST-HER2 immunotherapeutic candidate and the rest of the Lm platform."

"While historically, from 2021 to 2024, there were significant operating losses associated with the initiation, prosecution, and completion of the OST-HER2 clinical trial in recurrent, fully resected, metastatic osteosarcoma that used significant cash resources, going forward we expect a significantly reduced outlays beginning in the second quarter of 2025 now that one-time study costs were recognized in the first quarter of 2025," said Chris Acevedo, Chief Financial Officer of OS Therapies. "We have reduced our burn rate substantially such that we expect cash on hand to last the Company into 2026, through our projected BLA milestone and the related issuance of a PRV by the FDA that we intend to sell."

OS Therapies’ lead product candidate, OST-HER2, is a cancer immunotherapy biologic drug candidate comprised of HER2 bioengineered form of the Lm that mimics a listeria infection of HER2 overexpressing cancer cells and triggers a strong, immune response against cancer cells expressing HER2. This off-the-shelf immunotherapy is designed to prevent metastasis, delay recurrence, kill primary tumors expressing HER2 and increase overall survival.

Full-Year 2024 Corporate Highlights:

Completed treatment phase for Phase 2b clinical trial of OST-HER2 in recurrent, fully resected, lung metastatic osteosarcoma, a rare pediatric indication
Announced Phase 1 adult safety data of OST-HER2, primarily in breast cancer patients
Formed Scientific, Commercial and Patient Advocacy advisory boards
Completed $6 million initial public offering (IPO) & simultaneously converted all then-outstanding debt to equity
Completed $6 million private placement, providing sufficient capital into 2026 as a result of significantly reduced projected 2025 spend
Developed 2 new tunable ADC therapeutic candidates
Accepted into Johnson & Johnson – JLABS
2025 Progress to Date and Future Milestones

Progress to Date:

Positive Phase 2b data for OST-HER2 clinical trial in recurrent, fully resected, lung metastatic osteosarcoma
Agreement to acquire all LM-based Immuno-Oncology programs and IP assets from Ayala Pharmaceuticals, adding Phase 2 lung cancer and Phase 1 prostate cancer programs to pipeline, subject to closing conditions
Initiated commercial-ready manufacturing of OST-HER2 in preparation for BLA submission
Formed subsidiary OS Drug Conjugates and initiated review of strategic options for tunable Drug Conjugates (tDC) and tADC platform
Scheduled meeting with United Kingdom’s Medicines and Healthcare products Regulatory Agency’s (MHRA) Scientific Advice Meeting in the third quarter of 2025 for review of OST-HER2 immunotherapy candidate for osteosarcoma
Remaining Expected 2025 Milestones:

Presentation of full clinical data from OST-HER2 Phase 2b osteosarcoma clinical trial, including new synthetic control and biomarker data
End of Phase 2 Meeting with FDA for OST-HER2 osteosarcoma program, subsequent BLA submission and potential approval
Summer 2025 Scientific Advice Meeting (SAM) with MHRA for OST-HER2 osteosarcoma program, ILAP application submission and MHRA Conditional Marketing Authorisation application & decision
EMA National Competent Authority Scientific Advice Meeting Request (Medicines Evaluation Board, Netherlands) for OST-HER2 osteosarcoma program, EMA PRIME, EMA-FDA Parallel Scientific Advice application and EMA Conditional Marketing Authorisation application & decision
USDA meeting for OST-HER2 canine osteosarcoma program, conditional approval and initiation of pivotal clinical studies in preventive and therapeutic applications of OST-HER2 in osteosarcoma
Loss from Operations:

The Company recorded a net operating loss of $10.886 million in the year ended 2024 compared with a net operating loss of $7.916 million in 2023. The increase in net loss was largely due to the expenses associated with our IPO. Net loss per share in the full year 2024 was $0.88 on 12.377 million weighted average shares outstanding compared to full year 2023 where the Company delivered a loss of $1.46 per share on 5.429 million weighted average shares outstanding.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy any securities.

IDEAYA Biosciences Receives US FDA Breakthrough Therapy Designation for Darovasertib Monotherapy in Neoadjuvant Uveal Melanoma

On March 31, 2025 IDEAYA Biosciences, Inc. (NASDAQ: IDYA), a precision medicine oncology company committed to the discovery and development of targeted therapeutics, reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation (BTD) for darovasertib, a potential first-in-class protein kinase C (PKC) inhibitor, for the neoadjuvant treatment of adult patients with primary uveal melanoma (UM) for whom enucleation has been recommended (Press release, Ideaya Biosciences, MAR 31, 2025, View Source [SID1234651699]).

"We are pleased to receive FDA Breakthrough Therapy designation as we prepare to advance neoadjuvant darovasertib into a potential Phase 3 registrational trial in patients with primary UM. This designation highlights the potential of monotherapy darovasertib in a patient population with significant unmet medical need where there are currently no FDA-approved systemic therapies," said Dr. Darrin Beaupre, M.D., Ph.D., Chief Medical Officer of IDEAYA Biosciences. "We are targeting to present the updated Phase 2 clinical data in neoadjuvant UM that was provided as part of the BTD application at multiple medical conferences in 2025," said Yujiro S. Hata, President and Chief Executive Officer, IDEAYA Biosciences.

This U.S. FDA BTD designation, follows the Fast Track designation granted by the U.S. FDA for evaluation of darovasertib in combination with crizotinib in adult patients being treated for 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, entitling IDEAYA to certain potential tax credits, exemptions from user fees, and statutory marketing exclusivity.

The BTD application was supported by updated interim clinical data from an ongoing Phase 2 open-label trial (NCT05907954) evaluating darovasertib monotherapy in the neoadjuvant setting for localized UM. IDEAYA presented interim clinical data demonstrating an 82% ocular tumor shrinkage rate and a 61% eye preservation rate in UM patients in September 2024 (press release), and neoadjuvant UM data was also presented as an oral presentation at ASCO (Free ASCO Whitepaper) 2024. Updated clinical data in neoadjuvant UM, including efficacy, safety, radiation reduction, eye preservation, and vision preservation / improvement on treatment, were submitted as part of the BTD application that we plan to present at medical conferences in 2025. Multiple clinical data updates in neoadjuvant UM and MUM, including median overall survival (mOS) from the Phase 2 study (IDE196-001), are targeted to be presented at medical conferences in mid-year 2025 and the second half of 2025. A median progression free survival (mPFS) readout for the Phase 2/3 registration-enabling trial of the darovasertib and crizotinib combination in 1L HLA-A2-negative MUM is targeted by year-end 2025. The Company also intends to initiate a Phase 3 randomized registrational trial in neoadjuvant UM in the first half of 2025.

A potential Phase 3 registrational study would evaluate neoadjuvant darovasertib in primary UM patients who are eligible for enucleation (Cohort 1) or plaque brachytherapy (Cohort 2). Neoadjuvant UM has a projected annual incidence for North America, Europe, and Australia of approximately 12,000 patients, and is a high unmet medical with no FDA approved systemic therapies.

BTD is designed to expedite the development and regulatory review of promising therapies for serious or life-threatening conditions where preliminary clinical evidence suggests substantial improvement over existing treatments. The designation facilitates more intensive FDA guidance, cross-disciplinary collaboration, and eligibility for rolling submission and priority review.