On October 7, 2026 CHARM Therapeutics ("CHARM", "The Company"), a clinical-stage biotechnology company addressing resistance in acute myeloid leukemia (AML) with a best-in-class menin inhibitor, reported it has initiated dosing and fully enrolled the first cohort in its Phase I/II clinical trial of CHM-029 in patients with AML. This is the first example of a molecule designed using a protein-ligand co-folding model to progress into clinical studies.

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!

AML affects approximately 20,000 people annually in the United States and remains one of the most challenging blood cancers to treat with a five-year survival rate of less than 30%. Menin inhibitors have emerged as an important breakthrough in AML treatment, targeting the protein-protein interaction between menin and KMT2A, a key driver of leukemic cell growth. However, despite first-generation menin inhibitors demonstrating promising clinical activity, they are limited by the rapid emergence of resistance mutations in the menin protein that reduce efficacy and lead to disease progression. Further, the efficacy of some first generation menin inhibitors may be limited by safety risks including QTc prolongation and poor pharmaceutical properties such as the potential for drug-drug interactions and requirement for high doses.

CHARM has used its proprietary AI platform DragonFoldTM to specifically design CHM-029 to address this issue by closely mimicking the binding of KMT2A to menin, enabling the differentiation and cell death of otherwise cancerous cells. CHM-029 has demonstrated potent activity against all publicly described resistance mutations, robust dose-dependent tumor regression in both wild-type and mutant pre-clinical models, and a favorable preclinical safety profile.

Gary D. Glick, Ph.D., Interim Chief Executive Officer of CHARM Therapeutics, said: "Initiation of this Phase I/II clinical trial represents a pivotal moment for CHARM and, most importantly, for patients with AML who urgently need more durable treatment options. We progressed the program rapidly with less than 24 months elapsed between the first synthesis of CHM-029 and the first patient dosed. We look forward to continuing to advance CHM-029 through clinical development and believe our next-generation approach has the potential to transform outcomes for patients living with this devastating disease."

Professor Paresh Vyas, Principal Investigator, said: "Despite advances in targeted therapies for AML, outcomes remain poor for many patients. Dosing the first patient in this trial using a novel menin inhibitor that has been designed using AI to avoid drug resistance marks significant progress in efforts to deliver deeper more durable responses for patients, and we look forward to evaluating CHM-029 in the clinic."

The Phase I/II clinical trial (NCT07751991) will evaluate the safety and efficacy of CHM-029 in patients with relapsed or refractory AML with an NPM1 mutation, KMT2A rearrangement, or NUP98 rearrangement.

(Press release, CHARM Therapeutics, OCT 7, 2026, https://www.globenewswire.com/news-release/2026/10/07/3376361/0/en/charm-therapeutics-announces-first-patient-dosed-and-first-cohort-fully-enrolled-in-its-phase-i-ii-clinical-trial-of-chm-029-for-acute-myeloid-leukemia.html [SID1234673273])

On October 7, 2026 MAIA Biotechnology, Inc. (NYSE American: MAIA) ("MAIA", the "Company"), a clinical-stage biopharmaceutical company focused on developing targeted immunotherapies for cancer, reported that it has reached an enrollment milestone with 150 patients treated with ateganosine sequenced with an immune checkpoint inhibitor (CPI) in the ongoing Phase 2 and pivotal Phase 3 clinical trials of its novel telomere-targeting agent ateganosine as a treatment for non-small cell lung cancer (NSCLC).

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!

Enrollment is complete for the Part C of Phase 2 trial, THIO-101, which evaluates ateganosine sequenced with the CPI cemiplimab. MAIA recently announced 90.5% interim disease control (DCR) for the combination therapy in the efficacy evaluable population who had at least one tumor scan after starting treatment. Ateganosine’s measures of efficacy are close to triple the reported outcome for standard-of-care treatment with chemotherapy.

THIO-104 is MAIA’s pivotal Phase 3 trial evaluating ateganosine in sequence with a CPI in third line (3L) NSCLC patients whose disease has progressed following chemotherapy and CPI treatment. MAIA announced the first patient dosed in December 2025, and enrollment and dosing continues to advance at a strong pace. To date, 65 patients have been enrolled. The trial is targeting 100 patients enrolled and dosed by year-end.

"Our Phase 2 THIO-101 trial is on track to be the first completed clinical study of a telomere-targeting agent in the field of cancer drug discovery and treatment," said Vlad Vitoc, M.D., Founder and CEO of MAIA. "Our strategic focus on third-line NSCLC addresses a critical treatment gap for patients who have progressed after immunotherapy and chemotherapy, with no established standard of care today. Clinical data to date supports ateganosine’s potential to define a new treatment category for this difficult-to-treat population.

"For THIO-104, we have reached 65% of our enrollment target of 100 patients by year-end 2026, and remain on track with our target to conduct an interim analysis in 2027," Dr. Vitoc added.

The U.S. Food and Drug Administration (FDA) granted Fast Track designation for ateganosine for the treatment of NSCLC in July 2025. The designation allows for more frequent FDA communication, potential rolling review, and eligibility for Accelerated Approval and Priority Review. If approved, ateganosine will hold FDA New Chemical Entity (NCE) five-year marketing exclusivity. An NCE is a small molecule drug with a novel active ingredient that has not been previously approved or marketed.

About Ateganosine

Ateganosine (THIO, 6-thio-dG or 6-thio-2’-deoxyguanosine) is a first-in-class investigational telomere-targeting agent currently in clinical development to evaluate its activity in non-small cell lung cancer (NSCLC). Telomeres, along with the enzyme telomerase, play a fundamental role in the survival of cancer cells and their resistance to current therapies. The modified nucleotide 6-thio-2’-deoxyguanosine induces telomerase-dependent telomeric DNA modification, DNA damage responses, and selective cancer cell death. Ateganosine-damaged telomeric fragments accumulate in cytosolic micronuclei and activates both innate (cGAS/STING) and adaptive (T-cell) immune responses. The sequential treatment of ateganosine followed by PD-(L)1 inhibitors resulted in profound and persistent tumor regression in advanced, in vivo cancer models by induction of cancer type–specific immune memory. Ateganosine is presently developed as a second or later line of treatment for NSCLC for patients that have progressed beyond the standard-of-care regimen of existing checkpoint inhibitors.

About THIO-101 Phase 2 Clinical Trial

THIO-101 is a multicenter, open-label, dose finding Phase 2 clinical trial. It is the first trial designed to evaluate ateganosine’s anti-tumor activity when followed by PD-(L)1 inhibition. The trial is testing the hypothesis that low doses of ateganosine administered prior to cemiplimab (Libtayo) will enhance and prolong immune response in patients with advanced NSCLC who previously did not respond or developed resistance and progressed after first-line treatment regimen containing another checkpoint inhibitor. The trial design has two primary objectives: (1) to evaluate the safety and tolerability of ateganosine administered as an anticancer compound and a priming immune activator (2) to assess the clinical efficacy of ateganosine using Overall Response Rate (ORR) as the primary clinical endpoint. The expansion of the study will assess overall response rates (ORR) in advanced NSCLC patients receiving third line (3L) therapy who were resistant to previous checkpoint inhibitor treatments (CPI) and chemotherapy. Treatment with ateganosine followed by cemiplimab (Libtayo) has shown an acceptable safety profile to date in a heavily pre-treated population. For more information on this Phase II trial, please visit ClinicalTrials.gov using the identifier NCT05208944.

About THIO-104 Phase 3 Clinical Trial

THIO-104 is a multicenter, open-label, randomized Phase 3 clinical trial, designed to evaluate ateganosine’s telomere-targeting anti-tumor activity when followed by PD-(L)1 inhibition in patients with advanced third-line NSCLC who previously did not respond or developed resistance to treatment regimens containing checkpoint inhibitor and/or chemotherapy and have progressed. The trial has two primary objectives: (1) to assess the clinical efficacy of ateganosine compared to investigator’s choice of chemotherapy, using median Overall Survival (OS) as the primary clinical endpoint (2) to evaluate the safety and tolerability of ateganosine in sequential combination with a checkpoint inhibitor. For more information on this Phase 3 trial, please visit ClinicalTrials.gov using the identifier NCT06908304.

(Press release, MAIA Biotechnology, OCT 7, 2026, View Source [SID1234673272])

On October 7, 2026 RenovoRx, Inc. ("RenovoRx" or "the Company") (Nasdaq: RNXT), a life sciences company developing innovative targeted oncology therapies and commercializing RenovoCath, a patented, FDA-cleared drug-delivery device, reported that the first patient has enrolled in PRISM-TAMP, "PDAC Regression and Intraoperative Surgical Margin With Neoadjuvant TAMP," an investigator-initiated trial (IIT) sponsored by the University of Vermont (UVM). One week after the IIT began, a second patient is also progressing toward TAMP treatment.

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!

PRISM-TAMP is evaluating the approved chemotherapy gemcitabine delivered through RenovoRx’s Trans-Arterial Micro-Perfusion (TAMP) platform, enabled by RenovoCath, as neoadjuvant treatment before surgery in patients with borderline Pancreatic Ductal Adenocarcinoma (PDAC). PRISM-TAMP is the first RenovoRx supported IIT to advance to patient enrollment. UVM plans to enroll approximately 10 patients and is registered on ClinicalTrials.gov (NCT07477418). The primary focus of PRISM-TAMP is to assess PDAC tumor regression, the impact on intraoperative surgical margins following neoadjuvant (pre-surgical) treatment, and overall safety and tolerability.

In borderline resectable pancreatic cancer, the tumor involves nearby blood vessels to a degree that can make complete surgical removal difficult. Patients typically receive chemotherapy, with or without radiation, before surgery, and typically receive such treatment systemically, rather than administered locally near the tumor site as TAMP is designed to do.

"For patients with borderline resectable pancreatic cancer, treatment before surgery can influence both whether surgery is possible and how completely the tumor can be removed," said Conor O’Neill, MD, Assistant Professor at the UVM Larner College of Medicine and surgical oncologist at the UVM Medical Center, and principal investigator of PRISM-TAMP. "PRISM-TAMP will assess whether delivering chemotherapy directly near the tumor through TAMP before surgery can improve local therapeutic response and surgical outcomes. We are pleased to have enrolled our first patient in this important study."

"As TAMP becomes more well known in the marketplace, physicians have asked how TAMP could be used in borderline resectable pancreatic cancer and other settings beyond locally advanced pancreatic cancer, which is the focus of our ongoing Phase III clinical trial," said Leesa Gentry, Chief Clinical Officer of RenovoRx. "Enrolling the first PRISM-TAMP patient marks an important step for our IIT program as it moves from concept to clinical execution. These capital-efficient trials enable independent investigators to generate safety and tolerability data of new applications for TAMP, while we remain focused on completing the Phase III TIGeR-PaC trial and reporting topline data in the second half of 2027."

UVM is evaluating RenovoRx’s novel drug-device combination oncology product candidate intra-arterial gemcitabine delivered via RenovoCath (known as IAG) within the PRISM-TAMP study.

About RenovoCath

Based on its FDA clearance, RenovoCath is intended for the isolation of blood flow and delivery of fluids, including diagnostic and/or therapeutic agents, to select sites in the peripheral vascular system. RenovoCath is also indicated for temporary vessel occlusion in applications including arteriography, preoperative occlusion, and chemotherapeutic drug infusion. For further information regarding our RenovoCath Instructions for Use ("IFU"), please see: IFU-10004-Rev.-H-Universal-IFU.pdf.

(Press release, Renovorx, OCT 7, 2026, View Source [SID1234673271])

On October 7, 2026 Vaccinex, Inc. a clinical-stage biotechnology company pioneering a differentiated approach to treating Cancer and Alzheimer’s disease (AD) through the inhibition of Semaphorin 4D (SEMA4D), reported the publication in Nature Communications of results from an Emory University and Winship Cancer Institute-sponsored pilot study evaluating pepinemab in patients with metastatic melanoma. The publication represents an important milestone in an almost decade-long scientific partnership between Vaccinex and Winship investigators, focused on advancing the understanding of SEMA4D biology and its potential application in cancer immunotherapy. The findings provide new evidence in human cancer supporting Vaccinex’s strategy of targeting SEMA4D to reshape the tumor immune environment and promote conditions that support recognition of cancer cells and a coordinated antitumor immune response that may complement checkpoint inhibitors and other immunotherapies.

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 evaluated pepinemab in combination with the checkpoint inhibitors nivolumab and/or ipilimumab before surgery in patients with resectable metastatic melanoma. Consistent with its biomarker-driven design, the study focused on measuring treatment-associated changes in tumor immunity. The predefined primary biomarker endpoint was met; the trial was not powered to establish clinical efficacy.

Researchers found that pepinemab-containing regimens were associated with broad changes in the tumor microenvironment consistent with enhanced antitumor immunity. These changes included stronger biomarker signals associated with tumor recognition and antigen presentation, recruitment and activation of immune cells, and the development of highly organized immune structures known as mature lymphoid aggregates.

Importantly, greater abundance of mature lymphoid aggregates was associated with longer recurrence-free survival. This observation provides an encouraging link between the immune remodeling identified in the study and patient outcomes; however, the small biomarker-focused trial was not designed to demonstrate a clinical benefit from pepinemab. Confirmation in larger, randomized studies will be needed.

The publication, "Neoadjuvant pepinemab in combination with nivolumab and/or ipilimumab in resectable metastatic melanoma: a pilot biomarker-driven trial," is the result of a longstanding scientific collaboration between Vaccinex and investigators at Winship Cancer Institute of Emory University led by Michael C. Lowe, MD, MA, Gregory B. Lesinski, PhD, MPH, and Chrystal M. Paulos, PhD with additional contributions from The University of Texas MD Anderson Cancer Center led by Jennifer Wargo, MD, MMSc. The investigator-initiated trial was sponsored by Emory University and Winship Cancer Institute, with Vaccinex providing pepinemab together with scientific and research support. Vaccinex Research leaders included Elizabth Evans, PhD, Senior Vice President Discovery and Translational Medicine, and Crystal Mallow, Senior Research Scientist, who developed novel immunohistochemical methods for this study.

Key Findings

Pepinemab was associated with broad remodeling of the tumor immune environment. SEMA4D blockade was associated with changes across several components of antitumor immunity, including biological programs involved in antigen presentation, immune-cell recruitment and activation, together with changes in myeloid cells that can influence whether the tumor environment suppresses or supports an immune response.

Pepinemab-containing regimens were associated with highly organized immune structures within tumors. Mature lymphoid aggregates contained distinct populations of immune cells arranged in defined regions that enable local immune interactions. Some aggregates displayed features resembling tertiary lymphoid structures (TLS), specialized sites where multiple components of an immune response can come together within or near tumors.

More mature immune structures were associated with longer recurrence-free survival. Patients with greater abundance of mature lymphoid aggregates tended to remain recurrence-free longer. Additional analyses found evidence of expanded T- and B-cell populations within these structures, supporting the concept that they represented sites of active immune responses. Because the trial was designed to study biological effects and included a relatively small number of patients, these clinical associations are exploratory and require confirmation in larger studies.

About the Study

The investigator-sponsored pilot trial (NCT03769155) evaluated pepinemab in combination with nivolumab and/or ipilimumab in patients with resectable metastatic melanoma. Patients received two doses of treatment before planned surgery, allowing investigators to directly examine how the treatments affected the immune environment within subsequently resected tumors.

Thirty-eight patients participated in the study, and neoadjuvant treatment did not prevent or postpone curative-intent surgical resection. No grade 4 or 5 adverse events occurred during treatment before or after surgery.

The trial represents one component of a scientific partnership between Vaccinex and Winship Cancer Institute of Emory University, advancing SEMA4D research from mechanistic studies toward clinical evaluation in cancer. Vaccinex supplied pepinemab and supported multiple aspects of the research program, including scientific and operational oversight, generation and analysis of study data, and development of the scientific publication. Bristol Myers Squibb provided nivolumab and ipilimumab.

Together, these findings provide evidence from human tumors supporting the proposed mechanism of pepinemab and Vaccinex’s strategy of targeting SEMA4D as a means of reshaping the tumor immune environment. By promoting conditions associated with improved antigen presentation, immune-cell recruitment and organized adaptive immunity, SEMA4D blockade may offer a complementary approach to checkpoint inhibition and other cancer immunotherapies. The results support continued evaluation of pepinemab-containing combinations in melanoma and other solid tumors.

Publication

Ruffin, A.T., Mallow, C.L., Olson, B.M. et al. Neoadjuvant pepinemab in combination with nivolumab and/or ipilimumab in resectable metastatic melanoma: a pilot biomarker-driven trial. Nat Commun (2026). View Source

Clinical trial: NCT03769155

About Pepinemab

Pepinemab is a humanized IgG4 monoclonal antibody designed to block SEMA4D, which can otherwise bind to plexin-B1 receptors to trigger collapse of the actin cytoskeleton in cells and lead to loss of homeostatic functions of astrocytes and other glial cells in the brain and of dendritic cells in immune tissue. Pepinemab appears to be well-tolerated with a favorable safety profile in multiple clinical trials in different neurological and cancer indications.

(Press release, Vaccinex, OCT 7, 2026, View Source [SID1234673270])

On October 7, 2026 Rivercell, a Paris-based biotechnology and AI company building its own data infrastructure on how human cells respond to any treatment and training a world model of the cell that can predict those responses, reported a $25 million seed round led by HV. HCVC, Alven and Bpifrance Digital Venture also participated in the round, which will fund the scale-up of Rivercell’s proprietary data generation platform, the expansion of its wet lab in Paris (a lab working with chemicals and biological samples), and the launch of its AI Virtual Cell program. Being indication-agnostic, Rivercell’s world model can drive groundbreaking discovery across any cell behavior that leads to disease because it learns the underlying rules of how human cells respond to drugs and genetic changes, not the features of a single disease.

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!

Rivercell was started in summer 2025 by CEO Yann Fleureau, the engineer-turned-entrepreneur behind AI company Cardiologs, joined in 2026 by CSO Eric Durand, an AI scientist and former C-Level executive at AI drug discovery unicorn Owkin. Rivercell builds a proprietary platform that generates massive, high-quality pre-training datasets to train its "AI virtual cell" (AIVC) model. AIVC is a world model designed to predict how human cells respond to a drug or genetic change in silico, reducing the overall need for physical wet-lab experiments significantly. Rivercell’s long-term goal is to fundamentally change drug discovery by accelerating and redesigning it. Developing a single drug can take years and be costly, with the industry investing nearly $300 billion in R&D each year.

Realizing this potential depends on data. AlphaFold revolutionized protein structure prediction because it could draw on decades of publicly available protein structure data. Building a comparable model of the cell is a far greater challenge: it requires diverse, multimodal, time-resolved data on how cells respond to perturbations. No such dataset exists at the scale needed today, so Rivercell is creating that foundation. The company generates a large-scale biological dataset and uses it to train its AI virtual cell. Because today’s laboratory instruments were not designed for this application, Rivercell is developing an innovative, purpose-built platform capable of capturing the data at the scale required to pre-train its world model. The company describes this platform as the "GPU for the bio data center" of the future.

"A world model of the cell is the opportunity of the century in medicine. The missing piece is data: how cells change over time and under treatment, seen through multiple lenses and at multiple layers, captured at scale. Building that data engine in Europe is strategic, not only for Rivercell but for the continent’s place in AI and biology," said Yann Fleureau, co-founder and CEO of Rivercell.

Engineer and serial entrepreneur Yann Fleureau previously co-founded and led AI diagnostics company Cardiologs, raising several funding rounds before its nine-figure acquisition by Philips in 2021. Data scientist Eric Durand was director of oncology data science at Novartis and served as Chief Data Science Officer at Owkin before co-founding bio foundation model company Bioptimus, which raised $76 million.

"AI is already rewriting how we understand the chemistry of life, from protein structure to molecular design. The next frontier is predicting how cells behave, and that requires a full-stack approach that combines a novel platform, automated wet labs, and world models. Yann and Eric bring exactly this combination of expertise, and with its footing in both Paris’s AI ecosystem and Basel’s pharma cluster, Rivercell is uniquely positioned to lead it," said Maxi Pethö-Schramm, Principal at HV.

Rivercell is also backed by scientific advisors including Dr. Charlotte Bunne and Prof. Dr. Dr. Fabian Theis and angel investors including founders and executives from Twist Bioscience, DNA Script, Agilent and Abivax/Ipsen.

"AI virtual cells will transform how we understand and engineer biology. Rivercell’s ambition to unite large-scale perturbation experiments with AI simulation is why I’m excited to join its Scientific Advisory Board," said Dr. Charlotte Bunne, Assistant Professor, Artificial Intelligence in Molecular Medicine at EPFL.

"Understanding how cells respond to perturbations, over time rather than in snapshots, is one of the big open problems in biology. Rivercell is tackling it at scale, with both the data and the AI models to match. I’m thrilled to join its Scientific Advisory Board," closed Prof. Fabian Theis, Director of the Computational Health Center at Helmholtz Munich, Scientific Director of Helmholtz AI and Professor of Mathematical Modeling of Biological Systems at the Technical University of Munich.

(Press release, Rivercell, OCT 7, 2026, View Source [SID1234673269])