On May 28, 2025 InnoCare Pharma (HKEX: 09969; SSE: 688428), a leading biopharmaceutical company focusing on the treatment of cancer and autoimmune diseases, reported the approval of the Investigational New Drug (IND) by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) to conduct the clinical trial of B-cell lymphoma-2 (BCL2) inhibitor mesutoclax (ICP-248) in combination with azacitidine for the treatment of myeloid malignancies, including but not limited to myelodysplastic syndromes (MDS) (Press release, InnoCare Pharma, MAY 28, 2025, View Source [SID1234653471]).

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!

Mesutoclax is a novel, orally bioavailable BCL2 selective inhibitor. BCL2 is an important regulatory protein in the apoptosis pathway, and its abnormal expression is associated with the development of various hematologic malignancies. Mesutoclax exerts anti-tumor activity by selectively inhibiting BCL2 and restoring the normal apoptosis process in cancer cells.

Myelodysplastic syndrome (MDS) is a group of heterogeneous clonal myeloid diseases characterized by the abnormal proliferation of hematopoietic stem cells, recurrent genetic abnormalities, myelodysplasia, ineffective hematopoiesis, peripheral-blood cytopenia, and a high risk of progression to acute myeloid leukemia (AML). The annual incidence of myelodysplastic syndromes is about 4 cases/100,000 people/year (reaching 40–50/100,000 in patients aged ≥ 70 years).

Mesutoclax has been granted Breakthrough Therapy Designation (BTD) by the CDE for the treatment of BTKi-treated relapsed or refractory mantle cell lymphoma (R/R MCL). This marks the first BCL2 inhibitor to receive BTD recognition in China. The Company is accelerating patient enrollment of a Phase III registrational trial of mesutoclax in combination with orelabrutinib as a first line therapy for the treatment of chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), as well as a clinical trial of mesutoclax for the treatment of AML.

Dr. Jasmine Cui, the Co-founder, Chairwoman, and CEO of InnoCare, said, "Mesutoclax is an important global asset of our company in the field of hematology. We are delighted to receive approval to initiate the clinical trial for the fourth indication of our BCL2 inhibitor. We will accelerate the clinical development of mesutoclax across multiple indications in China and globally to bring benefits to patients as early as possible."

On May 28, 2025 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported that its research collaborators presented positive preclinical data and research from studies of GPX-002, the Company’s diabetes gene therapy drug candidate, in an oral presentation at the American Society of Gene and Cell Therapy’s (ASGCT) (Free ASGCT Whitepaper) 28th Annual Meeting which took place May 13-17, 2025 in New Orleans, Louisiana (Press release, Genprex, MAY 28, 2025, View Source [SID1234653439]).

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!

"We are pleased with the preclinical studies evaluating our novel gene therapy in diabetes, and we are proud to have been selected to present this data before an audience of innovators in cell and gene therapy," said Ryan Confer, President and Chief Executive Officer at Genprex. "We believe GPX-002 offers a promising opportunity for curative therapy in diabetes, which could impact the millions of patients suffering from this debilitating disease."

The oral presentation details for the Genprex-supported abstract:

Abstract Title: Immune Modulation Sustains Alpha Cell Reprogramming and Mitigates Immune Responses to AAV in a Diabetic Non-Human Primate Model

Session Title: Challenges in Immunological Responses to Therapeutic Interventions

Presenter: Hannah Rinehardt, MD, University of Pittsburgh Medical Center

Presentation Date: May 16, 2025

Presentation Time: 4:15 – 4:30 p.m. CT

Location: Room 278-282

In the oral presentation, Dr. Rinehardt discussed GPX-002, the diabetes gene therapy, which uses intraductal infusion of recombinant adeno-associated virus (rAAV) to deliver the Pdx1 and MafA genes. The gene therapy converts alpha cells into beta-like cells that secrete insulin physiologically, reversing diabetes in mouse models, where immunosuppression was not necessary. Researchers evaluated the immune response to direct infusion of rAAV into the pancreatic duct of Non-Human Primates (NHPs) with streptozotocin-induced diabetes and evaluated how to best manage immune responses.

Diabetes was induced with streptozotocin (STZ) in cynomolgus macaques, a type of NHP. NHPs received retrograde intraductal infusion of rAAV via laparotomy for precise delivery to the pancreas. rAAV capsids were chosen based on tropism for endocrine cells, and pre-existing neutralizing antibody titers (NAbs) were negative. Blood work, including serum C peptide and IV glucose tolerance tests, were serially obtained to monitor therapeutic efficacy. Immune response monitoring was performed for up to 4 months post-infusion and included serial NAbs, ELISpot assays, and immunophenotyping. Pancreatic tissues were analyzed using IHC and RNA-scope for beta cell markers, as well as single-cell RNA transcriptomics.

Expression of viral proteins occurs for a limited period of time after rAAV infection, since the infection doesn’t produce new AAV virus. One-month post-infusion, NHPs showed improved glucose tolerance and reduced insulin requirements. In the following months, using steroid-sparing regimens, increases in pancreatic B and T lymphocyte populations were noted on scRNA sequencing. Temporary immunosuppression (IS), using a combination of rituximab, rapamycin, and steroids for a 3-month course is largely effective at preventing anti-viral immunity. However, discontinuation of IS at 3 months post-infusion led to an immune response afterwards, indicating that IS in NHPs may need to be continued longer.

In conclusion, the novel rAAV gene therapy demonstrated that infusion of rAAV directly into the pancreatic duct of NHPs improves glucose tolerance but induces an anti-viral immune response which can degrade the improvement in glucose tolerance. The anti-viral immune response in NHPs can be largely prevented by administration of a multi-agent IS that leads to sustained therapeutic effects.

Researchers are continuing preclinical studies of GPX-002 therapy in NHP models of both Type 1 and Type 2 diabetes to generate additional data, and present studies are evaluating viral efficacy after six months of immunosuppression.

For more in-depth preclinical data supporting GPX-002, please review Genprex’s Corporate Presentation.

About GPX-002

GPX-002, which has been exclusively licensed from the University of Pittsburgh, is currently being developed using the same construct for the treatment of both Type 1 diabetes (T1D) and Type 2 diabetes (T2D). The same general novel approach is used in each of T1D and T2D whereby an adeno-associated virus (AAV) vector containing the Pdx1 and MafA genes is administered directly into the pancreatic duct. In humans, this can be done with a routine endoscopy procedure. In T1D, GPX-002 is designed to work by transforming alpha cells in the pancreas into functional beta-like cells, which can produce insulin but may be distinct enough from beta cells to evade the body’s immune system. In vivo, preclinical studies show that GPX-002 restored normal blood glucose levels for an extended period of time in T1D mouse models. In T2D, where autoimmunity is not at play, GPX-002 is believed to rejuvenate and replenish exhausted beta cells.

On May 28, 2025 Bexion Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company developing a novel class of biologic therapy to treat solid tumor cancers and chemotherapy-induced peripheral neuropathy (CIPN), reported that two abstracts were accepted at the upcoming 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place May 30, 2025 – June 3, 2025, in Chicago, Illinois (Press release, Bexion, MAY 28, 2025, View Source [SID1234653456]). Details of the abstracts are included below.

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!

Abstract Details:

Title: Effect of BXQ-350, a novel sphingolipid metabolism modulator, on neuronal environments through modulation of gene pathways
Abstract Number: e24058
Author: Michael Gazda, Tariq Arshad, Jim Beach, Nikhil Wilkins, Adam Creighbaum, Timothy Stephens
Session Title: Publication Only: Symptom Science and Palliative Care

Title: A phase 1 study to evaluate the safety and tolerability of BXQ-350, a novel sphingolipid metabolism modulator, in pediatric diffuse intrinsic pontine glioma and diffuse midline glioma
Abstract Number: e14044
Author: Tariq Arshad, Michael Gazda, Jim Beach, Kathleen Dorris, Margot Lazow, Trent Hummel, Richard Curry III, Adam Creighbaum
Session Title: Publication Only: Central Nervous System Tumors

The full abstracts are currently available in the ASCO (Free ASCO Whitepaper) digital program.

About BXQ-350
Bexion’s lead drug candidate is BXQ-350, a first-in-class biologic containing the multifunctional sphingolipid activator protein, Saposin C, and a phospholipid. Multiple Phase 1 clinical trials in adult and pediatric patients have demonstrated a robust safety profile for BXQ-350 with evidence of single agent activity across multiple solid tumor types. Additionally, other clinical and non-clinical data suggest BXQ-350 has activity in chemotherapy-induced peripheral neuropathy, an area of high unmet medical need in patients treated with oxaliplatin and other chemotoxic agents.

On May 28, 2025 bioAffinity Technologies, Inc. (Nasdaq: BIAF; BIAFW), a biotechnology company advancing noninvasive diagnostics and targeted therapeutics for cancer, reported the U.S. Patent and Trademark Office (USPTO) has issued a new patent covering a novel composition and method for selectively killing cancer by targeting the CD320 and LRP2 receptors on the cell membrane (Press release, BioAffinity Technologies, MAY 28, 2025, View Source [SID1234653472]).

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 patent, "Compositions and Methods for Treating Cancer," (Patent No. 12,305,171) provides for use of the novel therapeutic approach with cancers of the lung, breast, prostate, brain and skin. Research has begun toward development of a topical treatment for cutaneous malignancies and neoplasms of the skin.

The therapeutic patent is an important addition to bioAffinity Technologies’ patent portfolio, which includes 19 awarded U.S. and foreign patents and 36 pending patent applications related to its diagnostic platform and cancer treatment therapeutics.

The invention uses small interfering RNAs (siRNAs) to suppress the expression of CD320 and LRP2 proteins. In vitro studies demonstrated that the double knockdown of the proteins using siRNAs is selectively cytotoxic to human cancer cells but does not kill normal cells under the same treatment.

"The cancer cells killed by this discovery were not selected on the basis of any characteristic or biomarker, and the effect appears to be quite general," said William Bauta, PhD, Chief Science Officer at bioAffinity Technologies. "We believe our research has discovered a fundamental vulnerability of cancer that could be leveraged for new, highly selective therapies."

In vitro studies demonstrated that when only one of the receptors was down regulated, there was an increase in protein expression in the other receptor. The double knockdown of CD320 and LRP2 is required to kill cancer cells or significantly inhibit their proliferation.

"This patent marks a significant advancement in our therapeutic pipeline as well as our intellectual property portfolio," said Maria Zannes, President and CEO of bioAffinity Technologies. "This discovery is a continuation of the scientific curiosity that led to our CyPath Lung diagnostic for early-stage lung cancer and opens a potential new path to precision treatment across multiple cancer types. Our first target is developing a topical treatment for skin cancer."

About CyPath Lung

CyPath Lung uses proprietary advanced flow cytometry and artificial intelligence (AI) to identify cell populations in patient sputum that indicate malignancy. Automated data analysis helps determine if cancer is present or if the patient is cancer-free. CyPath Lung incorporates a fluorescent porphyrin that is preferentially taken up by cancer and cancer-related cells. Clinical study results demonstrated that CyPath Lung had 92% sensitivity, 87% specificity and 88% accuracy in detecting lung cancer in patients at high risk for the disease who had small lung nodules less than 20 millimeters. Diagnosing and treating early-stage lung cancer can improve outcomes and increase patient survival. For more information, visit www.cypathlung.com.

On May 28, 2025 Geron Corporation (Nasdaq: GERN), a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer, reported presentations on RYTELO (imetelstat) at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and the European Hematology Association (EHA) (Free EHA Whitepaper) 2025 Congress (Press release, Geron, MAY 28, 2025, View Source [SID1234653440]). Together, the presentations reinforce the potential benefits of the first-in-class oligonucleotide telomerase inhibitor RYTELO for a range of patients with lower-risk myelodysplastic syndromes (LR-MDS) with transfusion-dependent anemia and showcase the progress Geron is making with the ongoing IMpactMF and IMproveMF trials of imetelstat in myelofibrosis (MF).

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!

"Transfusion independence is an important goal for LR-MDS patients, but one that historically has not been achievable for many," said Rami S. Komrokji, M.D., Vice Chair, Malignant Hematology Department, Moffitt Cancer Center. "While imetelstat is already playing a vital role in LR-MDS, these new analyses being presented at ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) reinforce its potential to give a broad range of patients more time without transfusions and should also give clinicians confidence to add it to their toolkit as a second-line option for eligible patients."

Presentations at ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) include:

New pooled, post-hoc analyses expand on the pivotal IMerge results across challenging LR-MDS subpopulations

Analysis of patients with ring sideroblast negative (RS-) disease, showing that these difficult-to-treat patients appeared to experience clinical benefit with RYTELO, including ≥8-week, ≥24-week and ≥1-year red blood cell transfusion independence (RBC-TI), duration of RBC-TI, and hemoglobin rise in patients who achieved RBC-TI, consistent with prior findings from the overall Phase 3 IMerge population.
Analysis showing clinical benefit with RYTELO regardless of baseline serum erythropoietin (sEPO) level, supporting the use of RYTELO in the frontline setting for LR-MDS patients ineligible for erythropoiesis-stimulating agents (ESAs), and in the second-line setting after ESAs regardless of sEPO.
Analysis showing clinical benefit with RYTELO in ESA-ineligible or relapsed/refractory patients regardless of prior treatment with luspatercept or lenalidomide. Patients who had prior treatment with a hypomethylating agent (HMA) showed modest clinical activity with RYTELO.
Patient-centric outcome measures offering deeper insight on RYTELO impact in new post-hoc analyses from Phase 3 IMerge trial

Analysis showing that certain RYTELO-treated patients experienced sustained improvements in health-related quality-of-life (QOL) compared with placebo, as measured by certain categories in the patient-reported QOL in Myelodysplasia Scale (QUALMS) instrument.
Exploratory analysis suggesting that certain patients treated with RYTELO experienced a longer mean duration of time without transfusion reliance or relapse (TWiTR), compared with placebo.
New updates on ongoing trials in MF

Recently updated preliminary data from the dose escalation portion of the Phase 1/1b IMproveMF trial showing the combination of imetelstat and ruxolitinib was generally well-tolerated, with no dose-limiting toxicities observed as of the data cutoff date, and encouraging early dose-dependent efficacy data suggesting the potential of the combination for patients with intermediate-1 (INT-1), intermediate-2 (INT-2) or high-risk (HR) MF.
Trial-in-progress (TiP) update on the Phase 3 IMpactMF trial investigating imetelstat in relapsed/refractory MF, reporting that the trial has met approximately 80% of its enrollment target as of February 2025.
"At ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper), we’re excited to present new analyses on how RYTELO can deliver meaningful benefit across a range of LR-MDS patients who otherwise may have had limited options, whether due to their ring sideroblast status, baseline EPO level, or their treatment history," said Faye Feller, M.D., Executive Vice President and Chief Medical Officer of Geron. "We believe the depth and breadth of our data reinforce the potential of RYTELO to address critical unmet needs in LR-MDS and, combined with the advances we are making with our myelofibrosis program, underscore our confidence in telomerase inhibition as a potentially transformative clinical strategy and our commitment to exploring its full potential."

See below for a full schedule of presentations at ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper).

ASCO 2025 Presentations

Presentation Title

Author

Abstract Number

Presentation Details

IMproveMF update: phase 1/1B trial of imetelstat (IME)+ruxolitinib (RUX) in patients (pts) with intermediate (INT)-1, INT-2, or high-risk (HR) myelofibrosis (MF)

John O. Mascarenhas, M.D.

#6515

Rapid Oral, Fri. May 30, 1:00-2:30 PM CDT

Effect of prior treatment (tx) on the clinical activity of imetelstat (IME) in transfusion-

dependent (TD) patients (pts) with erythropoiesis-stimulating agent (ESA), relapsed or

refractory (R/R)/ineligible lower-risk myelodysplastic syndromes (LR-MDS)

Rami S. Komrokji, M.D.

#6569

Poster, Sun. June 1, 9:00 AM-12:00 PM CDT

IMpactMF, randomized, open-label, phase 3 trial of imetelstat (IME) versus best available therapy (BAT) in patients (pts) with intermediate-2 (INT-2) or high-risk (HR) myelofibrosis (MF) relapsed or refractory (R/R) to Janus kinase inhibitors (JAKi)

John O. Mascarenhas, M.D.

#TPS6588

Poster, Sun. June 1, 9:00 AM-12:00 PM CDT

EHA 2025 Presentations

Presentation Title

Author

Abstract Number

Presentation Details

Increased Duration of Time Without Transfusion Reliance (TWiTR) For Patients with LR-MDS Treated with Imetelstat Versus Placebo in the IMerge Trial

Mikkael A. Sekeres, M.D.

#PF646

Poster, Fri. June 13, 18:30-19:30 CEST

IMproveMF Update: Phase 1/1B Trial of Imetelstat + Ruxolitinib in Patients with INT-1, INT-2, or High-Risk MF

John O. Mascarenhas, M.D.

#PF830

Poster, Fri. June 13, 18:30-19:30 CEST

IMpactMF: Randomized, Open-Label, Phase 3 Trial of Imetelstat vs Best Available Therapy in Patients with INT-2 or High-Risk MF Relapsed/Refractory to JAK Inhibitors

John O. Mascarenhas, M.D.

#PF841

Poster, Fri. June 13, 18:30-19:30 CEST

Health-related Quality of Life Outcomes in Patients with Lower-Risk Myelodysplastic Syndromes Treated with Imetelstat in the IMerge Trial

María Díez-Campelo, M.D., Ph.D.

#PS1639

Poster, Sat. June 14, 18:30-19:30 CEST

Outcomes of Imetelstat Therapy in RS-Negative LR-MDS from the Pooled IMerge Study Populations

Valeria Santini, M.D.

#PS1622

Poster, Sat. June 14, 18:30-19:30 CEST

Outcomes with Imetelstat by Serum Erythropoietin Levels in Patients with LR-MDS Who Were Treatment Naïve or Who Had Prior Treatment with Erythropoiesis-Stimulating Agents

Rami S. Komrokji, M.D.

#PS1640

Poster, Sat. June 14, 18:30-19:30 CEST

Characterization and Management of Transient Cytopenias Associated with Imetelstat in LR-MDS from the IMerge Trial

Amer M. Zeidan, M.D.

#PB2760

Publication-only

Please see the full presentations for important qualifications and limitations on these post-hoc analyses.

About RYTELO (imetelstat)

RYTELO is an oligonucleotide telomerase inhibitor approved in the U.S. for the treatment of adult patients with low-to-intermediate-1 risk myelodysplastic syndromes (LR-MDS) with transfusion-dependent anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESAs). It is indicated to be administered as an intravenous infusion over two hours every four weeks.

In addition, RYTELO is approved in the European Union as a monotherapy for the treatment of adult patients with transfusion-dependent anemia due to very low, low or intermediate risk myelodysplastic syndromes without an isolated deletion 5q cytogenetic (non-del 5q) abnormality and who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy.

RYTELO is a first-in-class treatment that works by inhibiting telomerase enzymatic activity. Telomeres are protective caps at the end of chromosomes that naturally shorten each time a cell divides. In LR-MDS, abnormal bone marrow cells often express the enzyme telomerase, which rebuilds those telomeres, allowing for uncontrolled cell division. Developed and exclusively owned by Geron, RYTELO is the first and only telomerase inhibitor approved by the U.S. Food and Drug Administration and the European Commission.