On April 15, 2025 Toragen, Inc., a San Diego-based clinical-stage biotechnology company focused on developing uniquely selective drugs targeting cancers caused by viruses, reported positive safety data from its Phase 1 trial of TGN-S11, a small molecule inhibitor of the human papillomavirus (HPV) E5 oncogene protein, in patients with cancers associated with HPV (Press release, Toragen, APR 15, 2025, View Source [SID1234651951]).

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This Phase 1 trial was an open-label, non-randomized study in multiple cohorts of patients with relapsed, resistant, or metastatic HPV-associated cancers. The study has been conducted in two parts: Part 1 was escalating doses of TGN-S11 as monotherapy and Part 2 was TGN-S11 in combination with Keytruda (pembrolizumab), a PD-1 checkpoint inhibitor. The dose escalation part consisted of four Cohorts of three to six patients on monotherapy with increasing doses of TGN-S11. The Keytruda combination part consisted of three Levels of three to six patients with increasing doses of TGN-S11 and the standard dose of Keytruda.

In Part 1, 18 patients were treated with TGN-S11 as monotherapy in 4 dose cohorts with no serious adverse events identified. In the 4th dose cohort, 2 patients developed non-serious dose-related toxicities; therefore, the maximum tolerated dose (MTD) was determined to be 300mg/day. One-third of the patients who received TGN-S11 monotherapy showed drug activity with decreases in tumor size and decreases in Tumor Tissue Modified Viral (TTMV) HPV DNA as measured by the NavDx test. In addition, 10 patients were treated in Part 2 in 3 dose levels evaluating TGN-S11 in combination with Keytruda with no safety issues. Half of these patients in this portion of the study showed drug activity with decreases in tumor size and/or decreases in TTMV-HPV DNA score. One of these patients had a 92% reduction in TTMV-HPV DNA score and 3 patients remain on treatment.

This Phase 1 trial met both primary endpoints of proving safety and reaching the maximum tolerated dose of TGN-S11. In addition, our data also shows that 53% of patients that reached at least 2 months of treatment showed drug activity.

Dr. Neil Clendeninn, CMO of Toragen, said, "TGN-S11 was well-tolerated as monotherapy and in combination with Keytruda and we are very excited to see evidence of activity based on the reduction in TTMV-HPV DNA score in this study in advanced cancer patients."

Based on these positive results, Toragen plans to proceed with the development of its second-generation inhibitor of the E5 oncogene protein, TGN-S15, which has demonstrated increased efficacy and decreased side effects in preclinical testing. TGN-S15 will advance to IND-enabling studies to support a first in human study in healthy volunteers and Phase 2 clinical trials targeting HPV-associated cancers.

On April 15, 2025 Illumina Inc. (NASDAQ: ILMN) and Tempus AI, Inc. (NASDAQ: TEM), a technology company leading the adoption of AI to advance precision medicine and patient care, reported a collaboration to accelerate clinical adoption of next-generation sequencing tests through novel evidence generation (Press release, Illumina, APR 15, 2025, View Source [SID1234651950]). The collaboration will combine leading Illumina AI technologies with Tempus’s comprehensive multimodal data platform to train genomic algorithms and ultimately accelerate clinical adoption of molecular testing for patients.

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"In the era of true precision medicine, every patient who is battling complex disease should be routed to the optimal therapy based on molecular insights," said Everett Cunningham, chief commercial officer of Illumina. "We envision a world where the full range of molecular profiling is available as part of the standard of care—not just in cancer, but in cardiology, neurology, immunology, and every other category of disease."

Today, patients frequently miss the benefit of precision medicine because molecular profiling is not yet standard across disease areas and regions. This collaboration will leverage Tempus multimodal data to further improve Illumina’s AI-driven molecular analysis technologies and generate new insights supporting the clinical value of sequencing. These insights will be used to build evidence packages needed to standardize use of comprehensive genomic profiling and other molecular testing across all major diseases.

"By expanding our collaboration with Illumina, we are combining our strengths in technology and data analytics with their strengths in developing new sequencing technologies to drive forward innovation and advance precision medicine," said Terron Bruner, chief commercial officer of Tempus.

The program builds on a long-standing collaboration between the companies, which has focused on developing tools and assays to address gaps in testing needs from preemptive screening through therapy selection, health economics, and bioinformatics pipelines to improve patient outcomes and research.

On April 15, 2025 Accent Therapeutics, a clinical-stage biopharmaceutical company focused on novel, targeted, small molecule cancer therapeutics, reported that the first patient has been dosed in a first-in-human Phase 1/2 clinical trial evaluating the safety and tolerability of ATX-295, a potential best-in-class oral KIF18A inhibitor (Press release, Accent Therapeutics, APR 15, 2025, View Source [SID1234651949]). In addition, the company has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for ATX-295 for the treatment of adult patients with advanced/metastatic platinum-resistant or refractory ovarian cancer, and for ATX-559, a first-in-class potent and selective inhibitor of DHX9, for the treatment of adult patients with unresectable/metastatic dMMR/MSI-H colorectal cancer post checkpoint inhibitor treatment.

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"Cancers with high chromosomal instability, such as in certain ovarian, breast, and lung cancers, collectively affect a large patient population but have limited treatment options. With ATX-295 entering the clinic, we are excited to translate multiple years of KIF18A research into the development of a potentially best-in-class program," said Jason Sager, M.D., Chief Medical Officer of Accent Therapeutics. "With the launch of our Phase 1/2 clinical trial of ATX-295, we now have two investigational drugs in the clinic, bringing us closer to achieving our mission of transforming cancer care. Additionally, receiving FDA Fast Track designation for both of our lead assets underscores the power of our approach and the potential for these investigational drugs to urgently address high unmet medical needs."

ATX-295 is a selective inhibitor of KIF18A, a mitotic kinesin motor protein critical for cell division in select tumors with chromosomal instability, but not in healthy cells. Accent has demonstrated that its novel, potent, and selective small molecule KIF18A inhibitor displays selective dose-dependent tumor growth inhibition in preclinical models, including in high grade serous ovarian cancer and triple negative breast cancer, supporting its advancement to the clinic.

The ATX-295 Phase 1/2 open-label, dose-escalation and expansion study (NCT06799065) is designed to evaluate the molecule’s safety profile at multiple dose levels, assessing the tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of orally administered ATX-295. The trial is enrolling patients with locally advanced or metastatic solid tumors, including high-grade serious ovarian cancer.

The initiation of the Phase 1/2 ATX-295 study follows closely after Accent’s initial clinical asset, ATX-559, a first-in-class oral inhibitor of DHX9, entered clinical evaluation in late 2024. Both clinical assets have been granted Fast Track status by the FDA. Fast Track designation is designed to facilitate the development and expedite the review of novel drug candidates that address serious conditions marked by unmet medical need, with the aim of accelerating patient access to novel treatment options.

Accent will present new preclinical data supporting the continued clinical assessment of ATX-295 and ATX-559 at the 2025 AACR (Free AACR Whitepaper) Annual Meeting taking place April 25-30 in Chicago, Illinois. Accent will also present a trial-in-progress update on the ATX-559 Phase 1/2 clinical trial at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting taking place May 30 – June 3 in Chicago, Illinois.

About ATX-559
ATX-559 is a first-in-class potent and selective inhibitor of DHX9, a novel and previously undrugged RNA and DNA/RNA helicase, shown to play a critical role in tumors with high levels of replication stress (including breast, ovarian, colorectal, endometrial, gastric, and others), representing large patient populations with significant unmet medical need. DHX9 has been reported to play important roles in replication, transcription, translation, RNA splicing, RNA processing, and maintenance of genomic stability, making it a compelling novel oncology target. In addition to exploiting key tumor vulnerabilities in DNA repair deficient backgrounds (e.g., BRCA) and hyper-mutated states (e.g., MSI-H/dMMR), Accent is exploring the sensitivity of other tumor types to DHX9 inhibition, and the potential to combine DHX9 inhibitors with other cancer treatments to maximize its full potential for helping patients. Accent retains full worldwide rights to ATX-559, currently being evaluated in a Phase 1/2 clinical trial (NCT06625515), and the DHX9 program.

About ATX-295
Accent’s second lead program, ATX-295, is a potential best-in-class inhibitor for KIF18A which may address a large patient population across several cancer indications, including ovarian and triple negative breast cancer (TNBC). KIF18A is a mitotic kinesin motor protein critical for cell division in select tumors with chromosomal instability, but not in healthy cells. KIF18A inhibitor treatment results in rapid cell death for cancers with an abnormal number of chromosomes (aneuploid) in vitro and in vivo, while cells with normal numbers of chromosomes (euploid) are unaffected. Accent retains full worldwide rights to the KIF18A program, currently being evaluated in a Phase 1/2 clinical trial enrolling solid tumor patients (NCT06799065).

On April 15, 2025 Leap Therapeutics, Inc. (Nasdaq: LPTX), a biotechnology company focused on developing targeted and immuno-oncology therapeutics, reported it will host a virtual key opinion leader (KOL) event featuring Zev A. Wainberg, MD, Professor of Medicine at University of California, Los Angeles (UCLA) and co-director of the UCLA GI Oncology Program, on Wednesday, April 23, 2025 at 2:30 p.m. ET (Press release, Leap Therapeutics, APR 15, 2025, View Source [SID1234651948]).

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Dr. Wainberg will connect with Leap’s Chief Medical Officer, Cynthia Sirard, MD, to discuss the unmet need and how sirexatamab (DKN-01) may improve upon the current treatment landscape for previously treated patients with advanced microsatellite stable (MSS) colorectal cancer (CRC).

The event will focus on reviewing the positive data from Part B of the Phase 2 DeFianCe study of sirexatamab in second-line patients with advanced MSS CRC. Sirexatamab, Leap’s most advanced clinical program, is a humanized monoclonal antibody targeting the Dickkopf-1 (DKK1) protein.

A live Q&A will follow the discussion. A replay of the event will be available for a limited time on the Investors page of the Company’s website at View Source

About Zev A. Wainberg, MD
Zev A. Wainberg, MD, is the Professor of Medicine at UCLA and co-director of the UCLA GI Oncology Program. He was trained in medical oncology and hematology at UCLA. He completed his residency training at Albert Einstein College of Medicine and received his MD from the Sackler School of Medicine, New York Program at Tel Aviv University. His research involves a variety of clinical trials in multiple gastrointestinal cancers including pancreas, colon, gastric, and esophageal. Dr. Wainberg’s laboratory-based research efforts involve the testing of novel therapeutics against all gastrointestinal cancers. Currently, he is the recipient of several grants focused on the targeting of cancer stem cells and in molecular classification of gastrointestinal cancers.

On April 15, 2025 Pilatus Biosciences, pioneering biologics targeting metabolic checkpoints, reported that it will present new research on its lead candidate, PLT012, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2025 Annual Meeting (Press release, Pilatus Biosciences, APR 15, 2025, View Source [SID1234651947]). The presentations will include an oral symposium by co-founder and Chair of the Scientific Advisory Board (SAB), Prof. Ping-Chih Ho, and a poster session by Lead Scientist, Dr. Yi-Ru Yu. These efforts highlight PLT012’s potential as a novel therapeutic approach for immune-cold solid tumors. PLT012 is currently in late-stage preclinical development and progressing toward its first-in-human clinical trial.

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Event Participation and Presentations

Pilatus Biosciences will participate at the AACR (Free AACR Whitepaper) 2025 Annual Meeting, held April 27-30, 2025, with the following presentations:

Oral presentation: Prof. Ping-Chih Ho, co-founder and Chair of the SAB, will present during the Major Symposium SY21 – Immunometabolism and Metabolic Fitness in Tumors. The talk, titled "Reprogramming the Tumor Microenvironment with a Single Punch – Our Journey from Bench to Bedside", is scheduled for April 28, 2025, at 1:15 PM.
Poster presentation: Dr. Yi-Ru Yu, Lead Scientist, will present Abstract #6077 in Section 37, on April 29, 2025, from 2:00 PM to 5:00 PM. The poster will detail PLT012’s efficacy in addressing unmet needs in immune-cold solid tumors.
CEO Statement

"We are pleased to present our latest research at AACR (Free AACR Whitepaper) 2025," said Dr. Raven Lin, CEO of Pilatus Biosciences. "PLT012 reflects a differentiated approach—one that reawakens the immune system by targeting metabolic pathways, with the goal of extending the power of immunotherapy to patients who currently don’t benefit from it."

"PLT012 has earned FDA Orphan Drug Designation for liver and intrahepatic bile duct cancers. Its ability to reprogram the tumor microenvironment has led us to explore synergistic combinations, including bispecific antibodies (BsAb) and antibody-drug conjugates (ADCs), in preclinical studies across various solid tumor models, addressing critical unmet needs in oncology."

About PLT012

PLT012, is a humanized anti-CD36 antibody with a dual mechanism of action (MOA). It simultaneously inhibits immunosuppressive cell populations and enhances effector T cell function. Preclinical studies as a monotherapy have demonstrated its efficacy in both immune-hot and immune-cold tumor models, with a significant increase in GzmB-expressing CD8+ T cells and reductions in intratumoral Tregs and pro-tumorigenic macrophages. Additionally, PLT012 reshapes the exhaustion profile of cytotoxic CD8+ T cells by expanding both progenitor exhausted (Texprog) and terminally-exhausted (Texterm) populations with rejuvenated effector functions, leading to enhanced tumoricidal immunity. These findings suggest that PLT012, functioning as a metabolic regulator, may provide therapeutic benefits in cancer treatment, either as a monotherapy or in combination with immune checkpoint inhibitors such as PD-1 or PD-L1 inhibitors. Other than oncology, the unique MOA as a metabolic regulator also shows the potential of reprogramming the metabolic environment with associated benefits, e.g. liver functional improvement, thus laying the groundwork for targeting a broader spectrum of metabolic and immunological diseases.