On April 3, 2025 Phio Pharmaceuticals Corp. (NASDAQ: PHIO) is a clinical-stage siRNA biotechnology company developing therapeutics using its proprietary INTASYL gene silencing technology to eliminate cancer, reported that podium presentations will be given on its two lead INTASYL product candidates, PH-762 and PH-894 (Press release, Phio Pharmaceuticals, APR 3, 2025, View Source [SID1234651795]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The ITOC conference provides a unique platform for the exchange of latest findings in immunotherapy of cancer to advance oncology drug development and delivery.

Melissa Maxwell, Phio’s Director of Research and Program Management, was awarded two speaker presentations, as follows:

Plenary Session 3: New Targets and New Leads
Date and Time: April 3, 2025, 16.30 – 16.45 PM (CEST)

Title: Targeting PD-1 with self-delivering RNAi: preclinical advances and ongoing clinical evaluation in intratumoral Immunotherapy

PH-762, an INTASYL siRNA compound targeting PD-1, is being evaluated in an ongoing clinical trial (NCT 06014086) as a neoadjuvant intratumoral therapy for cutaneous malignancies, demonstrating promising preclinical efficacy, favorable tolerability, and the potential to enhance immune responses while minimizing systemic side effects.

Plenary Session 11: What Cellular Therapies Need to Work
Date and Time: April 5, 2025, 12.15 – 12.30 PM (CEST)

Title: Silencing BRD4 to increase NK cell activity for adoptive cell therapy: a novel self- delivering RNAi approach

PH-894, an INTASYL siRNA compound, selectively silences BRD4, a key regulator of gene expression. PH-894 enhances NK cell activation and proliferation without off-target effects, presenting a promising strategy to improve adoptive cell therapy.

On April 3, 2025 Akiram Therapeutics, a Swedish biotech company specializing in targeted radiotherapy, reported the launch of PRE-CISE, a Eurostars-funded research collaboration with Danish PreTT and TetraKit Technologies (Press release, Akiram Therapeutics, APR 3, 2025, View Source [SID1234651780]). The aim of the project is to develop a new generation of targeted alpha-therapeutics by expanding Akiram’s proprietary CD44v6-targeting antibody platform using advanced pre-targeting strategies and radiolabeling chemistry.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The PRE-CISE project combines Akiram’s CD44v6-targeting antibody platform with PreTT’s expertise in pre-targeting technology and TetraKit’s radiolabeling chemistry. Using a two-step approach—where the antibody first binds to the tumor, followed by the separate delivery of a radioactive payload—the collaboration aims to unlock access to alpha-emitting radionuclides that cannot typically be used with conventional antibody-based therapies. This strategy may significantly improve treatment precision while minimizing damage to surrounding healthy tissue.

"By separating the targeting step from the therapeutic phase, we open up new possibilities for designing more precise and effective cancer treatments," says Marika Nestor, CEO of Akiram Therapeutics. "This innovative therapeutic modality builds on our CD44v6 platform and represents an opportunity to expand our pipeline beyond beta-emitting radiotherapies."

"Participating in this project presents a remarkable opportunity to further validate our pretargeting approach, which leverages the targeting properties of antibodies for radioligand therapy. This advancement allows us to broaden the scope of potential treatments for various types of cancer," says Francesco Sergi-Lindell, CEO of PreTT. "Collaborating with the consortium will enable us to enhance our platform technology, as well as expand our pipeline."

"This collaboration will enable us to further develop our TetraKit platform and expand its application into new and highly exciting areas," says Andreas Jensen, CEO of TetraKit Technologies.

The PRE-CISE collaboration is co-funded by the Eurostars program with a total grant of EUR 1 million. Preclinical results are expected in 2026.

About Akiram’s CD44v6 platform and lead candidate
PRE-CISE builds on the CD44v6-targeting platform used in Akiram’s lead candidate, 177Lu-AKIR001, a beta-emitting radiopharmaceutical currently being evaluated in clinical trials.

Developed through antibody phage display and affinity maturation targeting the CD44v6 cancer marker, 177Lu-AKIR001 combines the radiation component lutetium-177 with a targeted molecule. Preclinical studies have demonstrated its potential as a promising, first-in-class radiopharmaceutical therapy for cancers with high CD44v6 expression.

On April 3, 2025 Scientists at the Nano Life Science Institute (WPI-NanoLSI), Kanazawa University and colleagues reported to have developed a promising new approach to cancer treatment (Press release, Kanazawa University, APR 3, 2025, View Source [SID1234651796]). By using tiny, naturally occurring particles called extracellular vesicles (EVs), they have created a way to boost the body’s immune system to fight tumors more effectively. This breakthrough could lead to more targeted cancer therapies with fewer side effects.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The study, published in Journal of Extracellular Vesicles, was led by Rikinari Hanayama. The research team developed a special type of engineered EVs, called antigen-presenting extracellular vesicles (AP-EVs), that activate immune cells inside tumors. These AP-EVs carry key immune-boosting signals, helping T cells—the body’s natural cancer fighters—recognize and attack tumors more effectively. This new approach enhances immune responses while reducing harmful side effects often seen in traditional cancer treatments.

Background

Many modern cancer treatments, such as immune checkpoint inhibitors and cytokine therapies, help the immune system fight cancer. However, these treatments can sometimes harm healthy tissues and cause severe side effects. Extracellular vesicles (EVs) are tiny, bubble-like structures naturally produced by cells to communicate with each other. Scientists have recently explored their potential as a targeted drug delivery system. In this study, researchers successfully modified EVs to directly deliver immune-activating molecules to tumor-fighting T cells, improving treatment effectiveness while reducing risks.

How the Research Was Conducted

The researchers tested their engineered vesicles using cell culture experiments and mouse models, analyzing their effects with imaging, flow cytometry, and molecular assays to track immune responses and tumor growth. By observing how immune cells reacted to AP-EVs, they confirmed that these vesicles could selectively stimulate T cells and enhance their tumor-fighting ability. Advanced imaging techniques also showed that AP-EVs accumulated in tumors, making them a promising tool for targeted cancer therapy.

Key Findings

Stronger Immune Response: AP-EVs helped immune cells grow and attack tumors more effectively.
Changing the Tumor Environment: The treatment made tumors more visible to the immune system, turning ‘cold’ tumors into ‘hot’ ones that are easier to attack.
Better Treatment When Combined: AP-EVs worked even better when combined with an existing immune checkpoint inhibitor (anti-PD-1 therapy).
Potential for Human Use: Researchers successfully tested human-compatible versions of AP-EVs, showing promise for future cancer therapies.
A New Approach to Cancer Treatment

Unlike traditional immunotherapy, which can cause broad immune activation and unwanted side effects, AP-EVs provide precise targeting of tumor-fighting T cells, leading to tumor eradication without harming healthy tissue or causing other unwanted side effects. AP-EVs also have the potential to improve the effectiveness of immune checkpoint inhibitors, and other T-cell therapies.

"This discovery is an important step toward using natural biological tools to improve cancer treatment," says Hanayama, lead author of the study. "By using extracellular vesicles, we can enhance immune responses with fewer side effects, potentially leading to better outcomes for patients."

Next Steps

The research team is now working on optimizing AP-EVs for clinical trials and exploring their use for other types of cancer and personalized medicine.

On April 3, 2025 BeiGene, Ltd. (NASDAQ: ONC; HKEX: 06160; SSE: 688235), a global oncology company that intends to change its name to BeOne Medicines Ltd., reported the discontinuation of its clinical development program for ociperlimab (BGB-A1217), an anti-TIGIT antibody, as a potential treatment for lung cancer (Press release, BeiGene, APR 3, 2025, View Source [SID1234651781]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The Independent Data Monitoring Committee recommended terminating the ongoing Phase 3 AdvanTIG-302 trial (NCT04746924) based on its findings as part of a pre-planned futility analysis. The overall efficacy and safety data assessment suggested that the study was unlikely to meet the primary endpoint of overall survival. No new safety signals were observed. After thorough deliberation, the Company has made the decision to terminate the trial.

"We evaluate our clinical programs to focus our resources on the most promising clinically differentiated candidates while thoughtfully de-prioritizing others. Our commitment remains steadfast: to discover and develop innovative treatments that are more affordable and accessible to cancer patients worldwide," said Mark Lanasa, M.D., Ph.D., Chief Medical Officer, Solid Tumors at BeiGene. "We thank the investigators, their patients, and support staff whose participation and dedication made this research possible."

Results from this study will be shared at a later date to help advance science and the understanding of anti-TIGIT activity.

On April 3, 2025 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, reported that management will participate in the 24th Annual Needham Virtual Healthcare Conference taking place on April 8, 2025 (Press release, Rocket Pharmaceuticals, APR 3, 2025, View Source [SID1234651797]). Gaurav Shah, M.D., Chief Executive Officer, will take part in a fireside chat at 8:45 a.m. ET on Tuesday, April 8.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

A webcast of the fireside chat will be available here and on the Investors section of the Company’s website. An archived replay of the webcast will be available for approximately 30 days following the event.