On March 31, 2025 Halozyme Therapeutics, Inc. (NASDAQ: HALO) (Halozyme) reported that Bristol Myers Squibb received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency, recommending approval of a new Opdivo (nivolumab) subcutaneous formulation developed with ENHANZE, Halozyme’s proprietary recombinant human hyaluronidase enzyme (rHuPH20), across multiple previously approved adult solid tumors as monotherapy, monotherapy maintenance following completion of nivolumab plus Yervoy (ipilimumab) combination therapy, or in combination with chemotherapy or cabozantinib (Press release, Halozyme, MAR 31, 2025, View Source [SID1234651692]).

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The CHMP opinion will now be reviewed by the European Commission, which has the authority to approve medicines in the European Union. A decision on the European extension of marketing authorization for the subcutaneous formulation of Opdivo is expected by June 2, 2025.

"We are delighted that the subcutaneous formulation of Opdivo developed with Halozyme’s ENHANZE drug delivery technology was recommended for approval in the European Union. Subcutaneous delivery of Opdivo would provide cancer patients a faster and more flexible treatment option and may help alleviate pressure on healthcare system resources," said Dr. Helen Torley, president and chief executive officer of Halozyme.

The CHMP positive opinion is supported by positive results from the Phase 3 CheckMate -67T trial. For more information on the study and its findings, please view Bristol Myers Squibb’s press release issued on March 28, 2025.

On December 27, 2024, nivolumab and hyaluronidase-nvhy, marketed under the brand name Opdivo Qvantig, was approved by the U.S. Food and Drug Administration.

On March 31, 2025 Theriva Biologics (NYSE American: TOVX), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, reported that a second Independent Data Monitoring Committee (IDMC) review of data from the VIRAGE Phase 2b clinical trial in newly-diagnosed metastatic pancreatic ductal adenocarcinoma (PDAC) found that that VCN-01 was well tolerated in combination with standard-of-care chemotherapy (gemcitabine/nab-paclitaxel) and the adverse event (AE) profile was as expected for the patient population and the medications being studied (Press release, Theriva Biologics, MAR 31, 2025, View Source [SID1234651691]).

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The IDMC, composed of key opinion leaders in pancreatic cancer and oncolytic viruses, reviewed clinical data from the complete safety population of 101 patients enrolled across 5 sites in the U.S. and 9 sites in Spain. The VCN-01 AE profile was consistent with that observed in prior clinical trials. The most common VCN-01 related AEs (pyrexia, flu-like illness, vomiting, nausea, and elevated transaminases) were transient and reversible. These AEs were observed to be less frequent and of reduced CTCAE grade after the second VCN-01 dose (administered on day 92) compared to the first VCN-01 dose (administered on day 1). The IDMC noted that the overall type and number of AEs in the VCN-01 treatment group was as expected for the pancreatic cancer population, the duration of treatment, and the administration of an oncolytic virus. VIRAGE patient enrollment was completed in September 2024 and topline clinical outcomes data are anticipated in Q2 2025.

"This second positive IDMC review of VCN-01 safety data from a larger number of patients affirms the feasibility of repeated VCN-01 dosing in metastatic PDAC patients" said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. "We are now working towards the release of topline clinical outcomes data in Q2 2025. If positive, these data, in combination with the previously reported feedback from the FDA and EMA, will guide the design of a potential Phase 3 registrational trial for discussion with regulatory agencies later this year."

About VIRAGE
VIRAGE is a two-arm, Phase 2b open-label, randomized, controlled, multicenter clinical trial in patients with histologically confirmed, newly-diagnosed metastatic PDAC. Patients have been enrolled at 5 sites in the U.S> and 9 sites in Spain. In both the control and VCN-01 treatment arms, patients receive gemcitabine/nab-paclitaxel standard-of-care chemotherapy in repeated 28-day cycles until disease progression. In the VCN-01 treatment arm only, patients are also administered intravenous VCN-01 seven-days prior to starting the first and fourth cycles of gemcitabine/nab-paclitaxel treatment (study days 1 and ~92 respectively). Primary endpoints for the trial include overall survival and VCN-01 safety/tolerability. Additional endpoints include progression free survival, objective response rate, and measures of VCN-01 biodistribution, replication, and immune response. More information about the trial is available on Clinicaltrials.gov (NCT05673811), through the Spanish Clinical Trials Registry and European Union Drug Regulating Authorities Clinical Trials Database (EudraCT Number: 2022-000897-24).

About VCN-01
VCN-01 is a systemically administered oncolytic adenovirus designed to selectively and aggressively replicate within tumor cells and degrade the tumor stroma that serves as a significant physical and immunosuppressive barrier to cancer treatment. This unique mode-of-action enables VCN-01 to exert multiple antitumor effects by (i) selectively infecting and lysing tumor cells; (ii) enhancing the access and perfusion of co-administered chemotherapy products; and (iii) increasing tumor immunogenicity and exposing the tumor to the patient’s immune system and co-administered immunotherapy products. Systemic administration enables VCN-01 to exert its actions on both the primary tumor and metastases. VCN-01 has been administered to 142 patients in clinical trials of different cancers, including PDAC (in combination with chemotherapy), head and neck squamous cell carcinoma (with an immune checkpoint inhibitor), ovarian cancer (with CAR-T cell therapy), colorectal cancer, and retinoblastoma (by intravitreal injection).

On March 31, 2025 Oncotelic Therapeutics, Inc. (OTCQB:OTLC) ("Oncotelic," the "Company," or "We"), a leader in RNA-based therapeutics, reported the publication of its latest research paper, titled, "Positive Prognostic Overall Survival Impacts of Methylated TGFB2 and MGMT in Adult Glioblastoma Patients (Press release, Oncotelic, MAR 31, 2025, View Source [SID1234651690])." The paper, authored by Sanjive Qazi, Michael Potts, Scott Myers, Stephen Richardson, and Vuong Trieu, is available online at: View Source

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To facilitate deeper exploration and discussion of this research by the research community, Oncotelic is introducing its proprietary communication platform powered by PDAOAI. PDAOAI enables users to query this paper and dozens of referenced articles through a single interactive interface. Scientists and clinicians are invited to engage at: View Source

A Simple Summary

Glioblastoma (GBM) is one of the most aggressive brain tumors in adults. It is well established that methylation of the O-6-methylguanine-DNA methyltransferase (MGMT) gene is predictive of overall survival (OS) benefits in patients receiving standard temozolomide and radiotherapy. Transforming growth factor beta (TGFB) is a family of cytokines involved in vital cellular processes and the regulation of growth factors.

The study’s novel discovery demonstrates that high TGFB2 gene methylation correlates with an improved OS risk, surpassing the predictive value of MGMT and TGFB1 methylation when controlling for age and sex. Several genes and pathways linked to TGFB2 methylation, including immune mechanisms such as T-cell activation, antigen processing, and Toll-like receptor pathways, were identified as improving survival outcomes in GBM patients. Of note, mucosa-associated lymphoid tissue lymphoma translocation protein, also referred to as MALT1, mRNA negatively impacted survival rates, suggesting a potential avenue for targeted therapies.

"The complexity of the publication was simplified into a concise statement by our PDAOAI platform, demonstrating the power of this platform for scientific communication: The findings underscore the importance of TGFB2 methylation as a prognostic marker in GBM treatment. High levels of TGFB2 methylation are associated with improved overall survival, particularly in young adult males. This suggests that TGFB2 methylation could be a valuable biomarker for risk stratification and therapeutic targeting in GBM, potentially guiding treatment decisions and improving patient outcomes." – Dr. Vuong Trieu, CEO of Oncotelic and co-author of the study.

"Our findings present an actionable opportunity to improve GBM patient outcomes by integrating sophisticated predictive analytical platforms and tools with clinical data. By uncovering insightful methylation patterns and elucidating gene-expression profiles at the biochemical pathway level, we expand our capacity to identify potential therapeutic targets. This approach supports the development of tailored treatment strategies through our nano-technology drug delivery platform. Ultimately, these insights enhance innovation in targeted therapies, driving improved clinical efficacy and patient survival rates in this devastating disease." – Dr. Sanjive Qazi, lead researcher

"This is the first paper we have published that utilized our proprietary AI technology. We are excited to see our technology being applied in real-world scenarios, and we look forward to the advancements it can potentially bring in the future." – Scott Myers, Product Manager

On March 31, 2025 Revolution Medicines, Inc. (Nasdaq: RVMD), a late-stage clinical oncology company developing targeted therapies for patients with RAS-addicted cancers, reported that Mark A. Goldsmith, M.D., Ph.D., the company’s chief executive officer and chairman, will participate in in two upcoming investor conferences (Press release, Revolution Medicines, MAR 31, 2025, View Source [SID1234651685]).

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Details of the company’s participation are as follows:

Needham 24th Annual Virtual Healthcare Conference
Fireside Chat: Monday, April 7 at 2:15 p.m. ET
Stifel 2025 Virtual Targeted Oncology Forum
Fireside Chat: Wednesday, April 9 at 1:00 p.m. ET

To listen to a live webcast of any of these events, or access archived webcasts, please visit: View Source Following the live webcasts, replays will be available on the company’s website for at least 14 days.

On March 31, 2025 Phio Pharmaceuticals Corp. (Nasdaq: PHIO) is a clinical-stage biotechnology company developing therapeutics that use its INTASYL siRNA gene silencing technology designed to make the body’s immune cells more effective in killing cancer cells, reported its financial results for the year ended December 31, 2024, and provided a business update (Press release, Phio Pharmaceuticals, MAR 31, 2025, View Source [SID1234651684]).

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Recent Corporate Updates

PH-762 Clinical Progress

PH-762 is currently being evaluated in a U.S. multi-center Phase 1b dose-escalating clinical trial through the intratumoral injection of PH-762 for the treatment of patients with cutaneous squamous cell carcinoma, melanoma and Merkel cell carcinoma. The trial (NCT 06014086) is designed to evaluate the safety and tolerability of neoadjuvant use of intratumorally injected PH-762 in up to 30 patients to assess tumor response and determine the dose or dose range for continued study of PH-762 in future trials. In May and December 2024, respectively, a Safety Monitoring Committee (SMC) reviewed data from the first and second dose cohorts treated with PH-762, and in both instances recommended escalation to the next dose concentration. A total of 7 patients with cutaneous carcinomas have been enrolled in dose cohorts 1 and 2. The second cohort enrolled a total of 4 patients, all of whom were diagnosed with cutaneous squamous cell carcinoma. At Day 36 (tumor excision), a complete response (100% tumor clearance) was reported for 2 patients, a partial response (90% tumor clearance) was reported for 1 patient and 1 patient had stable disease, having not progressed. Patients in the first cohort maintained stable disease.

To date, intratumoral injection of PH-762 has been well tolerated in all enrolled patients. There were no dose-limiting toxicities or clinically relevant treatment-emergent adverse effects in the patients receiving intratumoral PH-762. The third dose cohort is fully enrolled and patients in this cohort are currently in the treatment or follow-up phase of the study. Phio expects to complete enrollment of all patients in the study in the third quarter of 2025.

Capital Sourcing

In December 2024 and January 2025, Phio raised an aggregate of approximately $9.2 million in registered direct offerings and concurrent private placements, before deduction of commissions and other expenses. Additional gross proceeds of approximately $2.9 million were raised from the exercise of warrants previously issued on July 12, 2024. With these proceeds, the Company now believes it has sufficient capital to complete the treatment phase of the Phase 1b trial.

Cost Rationalization

In 2023, Phio commenced a cost rationalization program resulting from its strategy to transition from discovery research to a product development focus. In combination with headcount reductions, Phio did not renew its building lease in Marlborough, MA., the lease for which expired on March 31, 2024. The Company has established a smaller research footprint in the Massachusetts Biomedical Initiatives in Worcester, MA, where the Company leases laboratory space. Expense reductions have been redirected to funding the Phase 1b clinical trial for PH-762.

In May 2024, Phio terminated its Clinical Co-Development Agreement with AgonOx, Inc. (AgonOx). The Clinical Co-Development Agreement was entered into to conduct a Phase 1 clinical trial of PH-762 in combination with Agonox’s "double positive" tumor infiltrating lymphocytes (DP TIL) in patients with advanced melanoma and other advanced solid tumors. The primary trial objectives were to evaluate the safety and to study the potential for enhanced therapeutic benefit from the administration of PH-762 treated DP TIL. AgonOx had enrolled three patients. The first two patients were treated with DP TIL only, and a third patient was treated with a combination of DP TIL and PH-762. Clinical results for the single patient who received a combination of DP TIL and PH-762. Clinical results for the single patient who received a combination of DP TIL and PH-762 showed tumor size reductions of 65%, 100% and 81%, respectively, in three melanoma lesions. In assessing patient enrollment delays and the cost to continue the trial, management chose to redirect funding and focus on its self-directed phase 1b clinical trial with PH-762.

Patent Portfolio Enhancement

Phio’s portfolio includes 77 issued patents, 69 of which cover its INTASYL technology. There are 19 patent families broadly covering both the composition and methods of use of the Company’s self-delivering INTASYL technology and uses of INTASYL compounds targeting immune checkpoints for cancer therapy, cellular differentiation and metabolism targets for Adoptive Cell Therapy immunotherapies.

Scientific News

During 2024, Phio presented new data on its INTASYL self-delivering siRNA technology applications at several conferences including American Academy of Cancer Research (AACR) (Free AACR Whitepaper), Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper), American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) and at the Annual Oligonucleotide Therapeutics Society (OTS). Most recently, the Company was invited to present its phase 1b clinical trial results to date at the American Academy of Dermatology (AAD) in the Late-Breaking Research Session. The Company’s INTASYL PH-804 compound was highlighted in the peer reviewed journal, Cancer Immunology, Immunotherapy in an article entitled, "INTASYL Self Delivering RNAi Decreases TIGIT Expression, Enhancing NK Cell Cytotoxicity: A Potential Application to Increase the Efficacy of NK Adoptive Cell Therapy Against Cancer", authored by M. Maxwell et al.

Financial Results

Cash Position

At December 31, 2024, the Company had cash of approximately $5.4 million as compared with approximately $8.5 million at December 31, 2023.

During the year ended December 31, 2024, the Company completed multiple financings and received total net proceeds of approximately $4.0 million after deducting placement agent fees and offering expenses.

Subsequent to year-end, the Company completed additional financings and received total net proceeds of approximately $6.8 million after deducting placement agent fees and offering expenses.

Research and Development Expenses

Research and development expenses for the year ended December 31, 2024 decreased approximately $2.7 million, or 42%, as compared with the year ended December 31, 2023. The decrease in research and development expenses was primarily driven by the Company’s cost rationalization measures in transitioning from a research company to a product development company. These actions resulted in a decrease of approximately $1.0 million of expense due to the wind-down of preclinical studies, a reduction of approximately $0.8 million in salary-related costs including stock-based compensation expense, and approximately $0.2 million in lab supplies associated with the reduction in headcount. Additionally, the Company experienced a reduction in clinical consulting fees and clinical trial-related fees of approximately $0.4 million incurred in connection with its IND filing for PH-762 and its former PH-762 trials in ACT and European clinical trial, as well as a decrease of approximately $0.2 million in manufacturing fees for PH-762 compared with 2023.

General and Administrative Expenses

General and administrative expenses were approximately $3.7 million for the year ended December 31, 2024 as compared with approximately $4.4 million for the year ended December 31, 2023, a decrease of 14%. The decrease was primarily due to decreases in professional fees for a total of approximately $0.4 million primarily related to legal and patent expenses and in the Company’s D&O insurance premium of approximately $0.9 million as compared to the prior year.

Net Loss

Net loss was approximately $7.2 million, or ($9.08) per share, for the year ended December 31, 2024 as compared with a net loss of approximately $10.8 million, or $46.76 per share, for the year ended December 31, 2023. The decrease in net loss was primarily due to the changes in research and development expenses, as described above.