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Neogap receives approval to administer full dose in clinical trial – streamlining development of its personalised cell therapy

On May 28, 2025 Neogap Therapeutics AB, a Swedish clinical-stage biotechnology company developing personalised immunotherapy for cancer treatment, reported to have received approval from the Swedish Medical Products Agency to administer the full dose to all patients in its ongoing phase I/II clinical trial (Press release, Neogap Therapeutics, MAY 28, 2025, View Source;streamlining-development-of-its,c4156253 [SID1234653443]). The decision introduces a more flexible study design and contributes to a faster and more efficient development of the company’s cell therapy.

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According to the revised study protocol, the company is now authorised to treat each patient with the full dose, instead of starting with a low dose and gradually increasing it for subsequent patients. The amendment aligns with recently published data from other cell therapy studies and enables earlier analysis of treatment response.

"Being able to give the full dose from the outset is an important step in the development of our personalised cell therapy. It allows us to collect data earlier that may contribute to the evaluation of both the treatment’s safety and efficacy, while supporting a more efficient and flexible study design – to the benefit of both patients and our continued development efforts," says Samuel Svensson, CEO of Neogap Therapeutics.

The company has also received approval to extend the shelf life of both the starting material from the patient’s lymph nodes and the final frozen product. This provides greater flexibility in clinical operations, particularly when treatment needs to be scheduled around standard chemotherapy or temporary infections.

The treatment under evaluation is pTTL (personalised Tumour Trained Lymphocytes), Neogap’s personalised, cell-based immunotherapy designed to strengthen the patient’s own T cells to target solid tumours. It is based on Neogap’s patented technologies PIOR and EpiTCer, and is currently being investigated in a phase I/II clinical trial for advanced colorectal cancer. Patient recruitment is ongoing at several hospitals in Sweden.

iTeos Therapeutics Announces Its Intention to Wind Down Operations

On May 28, 2025 iTeos Therapeutics, Inc. (Nasdaq: ITOS), a clinical-stage biopharmaceutical company pioneering the discovery and development of a new generation of immuno-oncology therapeutics for patients, reported the intention to wind down its operations as part of a comprehensive review of strategic alternatives aimed at maximizing shareholder value (Press release, iTeos Therapeutics, MAY 28, 2025, View Source [SID1234653442]).

Following a thorough assessment of the Company’s development pipeline, business prospects, and financial position, the Company’s Board of Directors intends to wind down clinical and operational activities and focus on leveraging the Company’s cash balance to deliver near-term value to shareholders, including any proceeds from the potential sale of the Company’s intellectual property and assets such as EOS-984, EOS-215, and a preclinical obesity program targeting ENT1.

IMUNON Closes Up To $9.75 Million Private Placement Priced At-The-Market Under Nasdaq Rules

On May 28, 2025 IMUNON, Inc. (NASDAQ: IMNN), a clinical-stage company in Phase 3 development of its DNA-mediated immunotherapy, reported sale of an aggregate of 7,222,223 shares of its common stock (or pre-funded warrants in lieu therof) and short-term warrants to purchase up to an aggregate of 14,444,446 shares of common stock at a purchase price of $0.45 per share (or pre-funded warrant in lieu thereof) and accompanying short-term warrants in a private placement priced at-the-market under Nasdaq rules (Press release, IMUNON, MAY 28, 2025, View Source [SID1234653441]). The warrants are exercisable beginning on the effective date of stockholder approval of the issuance of the shares of common stock upon exercise of the warrants at an exercise price of $0.45 per share and will expire three years from the date of stockholder approval.

H.C. Wainwright & Co. acted as the exclusive lead placement agent for the offering. Brookline Capital Markets, a division of Arcadia Securities, LLC, acted as co-placement agent.

The aggregate gross proceeds to the Company from the private placement was approximately $3.25 million, before deducting placement agent fees and other offering expenses payable by the Company. The potential additional gross proceeds to the Company from the short-term warrants, if fully-exercised on a cash basis, will be approximately $6.5 million. No assurance can be given that any of such short-term warrants will be exercised. The Company intends to use the net proceeds from the offering for working capital and general corporate purposes.

The shares of common stock, pre-funded warrants and short-term warrants described above were offered in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the "Act") and Regulation D promulgated thereunder and, along with the shares of common stock underlying the pre-funded warrants and short-term warrants, have not been registered under the Act or applicable state securities laws. Accordingly, the shares of common stock, the pre-funded warrants, the short-term warrants and the shares of common stock underlying the pre-funded warrants and short-term warrants may not be offered or sold in the United States absent registration with the Securities and Exchange Commission ("SEC") or an applicable exemption from such registration requirements. The securities were offered only to accredited investors. Pursuant to a registration rights agreement, the Company has agreed to file one or more registration statements with the SEC covering the resale of the shares of common stock and the shares issuable upon exercise of the pre-funded warrants and short-term warrants.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

Geron Announces Presentations at ASCO and EHA Underscoring RYTELO® (imetelstat) Efficacy and Safety Across Range of LR-MDS Patients, and Showcasing Momentum of Myelofibrosis Program

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).

"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.

Genprex Collaborators Present Positive Preclinical Data on Diabetes Gene Therapy at the ASGCT 28th Annual Meeting

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]).

"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.