On April 25, 2025 Rgenta Therapeutics, a clinical-stage biotechnology company pioneering the development of a new class of oral small molecules targeting RNA and RNA regulation for oncology and neurological disorders, reported that preclinical data from its lead program, RGT-61159 were presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2025 Annual Meeting, which is being held from April 25-30, 2025, in Chicago, IL (Press release, Rgenta Therapeutics, APR 25, 2025, View Source [SID1234652159]). The data demonstrate robust anti-tumor activity of RGT-61159 in several cell-derived xenograft (CDX) models of AML and synergistic benefit when combined with standard of care for AML highlighting the potential of RGT-61159 to treat a broad AML patient population.

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"The data presented at the AACR (Free AACR Whitepaper) meeting highlight the ability of RGT-61159 to reduce levels of both MYB RNA transcripts and correspondingly MYB protein in a dose dependent fashion, which translates to potent killing activity in cells that overexpress MYB," said Travis Wager, Ph.D. co-founder and chief scientific officer. "Importantly, we see robust antitumor activity with RGT-61159 across a range of AML models that carry genetic alterations that are common in patients with AML highlighting its potential to treat a broad AML patient population."

"Using RGT-61159, which specifically acts at the RNA level, we are able to address MYB which has been shown to function as an oncogenic driver in a variety of cancers including AML, colorectal cancer (CRC) and adenoid cystic carcinoma (ACC), and until now, has been a difficult target to drug," said Simon Xi, Ph.D., cofounder and chief executive officer of Rgenta. "Our ongoing Phase 1a/b clinical trial of RGT- 61159 in patients with relapsed or refractory ACC or CRC is advancing well and we look forward to broadening that program and initiating a new Phase 1/2 study of RGT-61159 in adults with AML/high risk myelodysplastic syndromes the second half of 2025."

In a poster titled RGT-61159, Best-in-class Oral Small Molecule Inhibitor of MYB via Selective RNA Splicing Alteration, Synergistic Anti-Tumor Activity When Combined with Standards of Care in Leukemia Disease Models Harboring AML Common Genetic Lesions, data were presented highlighting the close correlation between the elimination of MYB RNA and protein by RGT-61159 and its potent killing activity against leukemia and lymphoma cancer cell lines that over express MYB, demonstrating on-target therapeutic activity of this potent, orally available small molecule. RGT-61159 also inhibited AML cell proliferation and downregulated the expression of master oncogenes controlled by MYB, including MYC, BCL2, FLT3 and IDH1. Data from several CDX models of AML with genetic alterations seen commonly in AML, demonstrated the robust anti-tumor activity of tolerated doses of RGT-61159 and synergistic activity both in vitro and in vivo when administered in combination with agents used in AML standard of care.

About RGT-61159
RGT-61159 is an orally available small molecule designed to specifically modulate splicing of the transcription factor MYB resulting in the inhibition of the oncogenic MYB protein and potential cell death of the cancer cells that overexpress the MYB protein. MYB acts as a master regulator of cell proliferation, self-renewal, and differentiation processes and its aberrant expression has been demonstrated in multiple forms of human cancer including adenoid cystic carcinoma (ACC), acute myeloid leukemia (AML), T-cell acute lymphoblastic leukemia (T-ALL), colorectal cancer (CRC), small cell lung cancer (SCLC) and breast cancer. Rgenta is evaluating RGT-61159 in an ongoing multi-center, open-label Phase 1a/b clinical trial in patients with advanced relapsed or refractory ACC or CRC. The Phase 1a/b study is designed to evaluate safety, tolerability, pharmacokinetics, target engagement and clinical efficacy of RGT-61159 in patients with ACC or CRC. Additional information about the Phase 1a/b clinical trial can be accessed at ClinicalTrials.gov (NCT06462183).

On April 25, 2025 Tempus AI, Inc. (NASDAQ: TEM), a technology company leading the adoption of AI to advance precision medicine and patient care, reported that 18 abstracts, including one oral presentation, have been accepted for presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025, on April 25 – 30 in Chicago (Press release, Tempus, APR 25, 2025, View Source [SID1234652176]). Tempus researchers will showcase scientific and clinical research that highlight the transformative impact of AI on oncology treatment and patient outcomes.

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"Tempus is proud to showcase a comprehensive collection of scientific research this year, highlighting the impact of our multimodal dataset and AI-enabled diagnostic solutions on cancer research," said Kate Sasser, PhD, Chief Scientific Officer at Tempus. "AACR stands as a leading forum for cancer research, and we look forward to presenting our findings alongside our collaborators in Tempus’ home city of Chicago."

Research highlights include:

Oral Presentation: Investigating the clinical landscape and biological impact of SF3B1 hotspot mutations in breast cancer
Date/Time: April 27, 2025; 4:40 PM – 4:45 PM CT
Location: To be announced

Overview: This study examines the implications of SF3B1 hotspot mutations in breast cancer, focusing on genetic profile, survival outcomes, and biological impacts, by analyzing de-identified data from Tempus’ multimodal real-world database consisting of 420 breast cancer patients with SF3B1 mutations. Innovative genome editing in isogenic breast cell lines revealed that SF3B1 mutations negatively impact cell growth and tumor development. The findings support the utility of SF3B1 mutations as potential therapeutic targets and underscore the importance of understanding their role in cancer biology, with ongoing research aimed at uncovering the mechanisms behind hotspot-specific effects.
Poster Presentation: Genetic and clinical landscape of NUTM1 structural variants
Date/Time: April 28, 2025; 2:00 PM – 5:00 PM CT
Location: Section 34
Overview: Within Tempus’ multimodal real-world database, researchers identified 59 patients with a primary diagnosis of NUT carcinoma—an aggressive cancer—81% of whom had a confirmed NUTM1 fusion. Notably, there were 106 additional patients who had a NUTM1 fusion without a corresponding initial NUT carcinoma diagnosis, suggesting a potentially significant underdiagnosis rate. The study found a variety of fusion gene partners, with certain cancer types showing enrichment of specific fusions. With a median overall survival of just over 5 months, the findings suggest that certain cancer types with a high enrichment of NUTM1 fusions may benefit from universal next-generation sequencing to ensure accurate diagnosis and potentially improve outcomes for patients with high-risk cancer types.
Poster Presentation: A longitudinal, circulating tumor molecular response biomarker as a predictor of clinical outcomes in a real-world cohort of patients with advanced solid tumors treated with tyrosine kinase inhibitors
Date/Time: April 29, 2025; 9:00 AM – 12:00 PM CT

Location: Section 45

Overview: In a study analyzing advanced cancer patients, researchers evaluated the prognostic value of changes in circulating tumor DNA tumor fraction (ctDNA TF) during tyrosine kinase inhibitor (TKi) therapy. The study, which consisted of 109 patients from Tempus’ multimodal real-world database, found that molecular responders had significantly longer real-world overall survival (rwOS) than molecular non-responders across various cancer types. The findings suggest that ctDNA TF may serve as a biomarker to predict molecular response to TKi therapy, potentially guiding treatment decisions and improving patient outcomes in a real-world setting.
Collaborator-led Poster Presentation: Enhancing TCR-T with a Fas-based switch receptor boosts T cell engraftment, persistence, and anti-tumor activity in models of hard-to-treat PRAME solid tumor indications
Date/Time: April 29, 2025; 9:00 AM – 12:00 PM CT

Location: Section 39

Overview: T-knife Therapeutics is developing a FAS-based switch receptor (FAS-TNFR) to target PRAME-positive solid tumors, designed to enhance T cell activity and overcome the hostile tumor microenvironment. Utilizing Tempus multi-modal data, T-Knife analyzed a large database of tumor samples to identify the inhibitory ligands most frequently found in PRAME-expressing indications and to understand in depth the pattern of expression of PRAME and inhibitory ligands in different patient populations. These insights provided by Tempus were crucial for T-knife to select the optimal switch receptor from their armoring toolbox and determine appropriate target populations for their upcoming clinical trials (Figures 1A, 2D, and 3A-C present Tempus-driven data and insights).

On April 24, 2025 Pasithea Therapeutics Corp. (NASDAQ: KTTA) ("Pasithea" or the "Company"), a clinical-stage biotechnology company developing PAS-004, a next-generation macrocyclic MEK inhibitor, for the treatment of neurofibromatosis type 1 (NF1) and other MAPK pathway driven indications, reported the acceptance of an abstract for a poster prenstation at the Annual Meeting of the American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) taking place May 30 – June 3, 2025, in Chicago, Illinois (Press release, Pasithea Therapeutics, APR 24, 2025, View Source [SID1234652108]).

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The Company will present updated interim clinical data from its onging Phase 1 clinical trial of PAS-004 in patients with MAPK pathway driven advanced solid tumors.

"We are pleased to present interim clinical data of PAS-004 through cohort 4A and 4B, that to date has demonstrated clinical activity, target engagement, and a favorable safety profile," said Dr. Tiago Reis Marques, Chief Executive Officer of Pasithea. "We believe PAS-004’s emerging profile may achieve the sweet spot between PK, PD and tolerability and may make PAS-004 an ideal candidate for the treatment of NF1 related cutaneous and plexiform neurofibromas as well as a potential candidate for treatment of various cancers and MAPK pathway driven diseases."

Presentation and poster details

Title: Phase 1 dose-escalation study of the safety and pharmacokinetics of PAS-004, a macrocyclic MEK inhibitor, for the treatment of patients with MAPK pathway–driven advanced solid tumors
Session: Poster Session – Developmental Therapeutics – Molecularly Targeted Agents and Tumor Biology
Poster Board: 440
Date and Time: 6/2/2025, 1:30 – 4:30 PM CDT

The full abstract will be available on the ASCO (Free ASCO Whitepaper) website on May 22, 2025, at 5:00 p.m. ET.

The ongoing Phase 1 clinical trial is a multi-center, open-label, dose escalation 3+3 study design to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and preliminary efficacy of PAS-004 in patients with MAPK pathway driven advanced solid tumors with a documented RAS, NF1 or RAF mutation or patients who have failed BRAF/MEK inhibition (NCT06299839).

On April 24, 2025 Debiopharm Research & Manufacturing S.A. (Debiopharm, www.debiopharm.com), a Swiss-based global biopharmaceutical company aiming to cure cancer and infectious diseases, and Oncodesign Services (www.oncodesign-services.com), a leading CRO specialized in drug discovery and preclinical services, reported the execution of a license agreement for the use of the AbYlink technology for preclinical services (Press release, Debiopharm, APR 24, 2025, View Source [SID1234652124]).

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AbYlink is a regio-selective bioconjugation technology ideally suited for preparing conjugates for use in therapeutic and non-invasive diagnostic applications. Oncodesign Services will use this cutting-edge technology to prepare antibody chelator conjugates for use in preclinical studies to gain insights into the predictive effectiveness of novel treatment approaches in Molecular Radiotherapy, and particularly Radioimmunotherapy in the context of cancer. A joint poster will be presented at the AACR (Free AACR Whitepaper) Annual Meeting 2025 to demonstrate the use of the non-invasive technology in Radioimmunotherapy, targeting animal models bearing HER2+ tumors.

"We have demonstrated the effectiveness of this powerful technology to rapidly and covalently conjugate any off-the-shelf antibody in a single step with an imaging agent. Our strategic collaboration with Oncodesign Services allows broader access to and usage of non-invasive imaging applications and provides an innovative solution to biotech and pharma partners to integrate imagery in the development of new antibody- and ADC-based therapeutics," commented Frédéric Lévy, Chief Scientific Officer at Debiopharm.

Aidan Synnott, CEO of Oncodesign Services, said, "We are pleased to integrate this cutting-edge technology into our portfolio for our clients. The AbYlink technology gives the advantage of generating reproducible batches of bioconjugated antibodies and ADCs that secure specificity and efficacy of targeted radiotherapy."

About AbYlink

AbYlink is a versatile and rapid regio-selective chemical conjugation technology for use to prepare diagnostic or therapeutic conjugates. This one-step method results in stable conjugation at defined and invariable sites on the Fc domain of an antibody or the like, with no impact on antigen-binding regions. It enables a seamless and reproducible conjugation of payloads (e.g., a chelator for radiolabeling, a fluorescent dye or a drug) to antibodies or ADCs. The universal applicability of the technology has been demonstrated for various antibody isotypes and payloads.

On April 24, 2025 Merck & Co reported its First-Quarter 2025 Financial Results (Presentation, Merck & Co, APR 24, 2025, View Source [SID1234653383]).

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