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Genprex Collaborators to Present Positive Preclinical Data on Diabetes Gene Therapy for Type 2 Diabetes at the 2026 American Society of Gene and Cell Therapy Annual Meeting

On April 28, 2026 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 will present positive preclinical data on the Company’s diabetes gene therapy drug candidate at the upcoming 2026 American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting, taking place May 11-15, 2026 in Boston, Mass. The collaborators will present preclinical data demonstrating that the diabetes gene therapy (Pdx1/MafA gene therapy, PM or GPX-002) can reverse hyperglycemia in Type 2 diabetic (T2D) mouse models.

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"Our collaborators’ preclinical data to be presented at ASGCT (Free ASGCT Whitepaper) 2026 demonstrate the ability of our diabetes gene therapy to reverse hyperglycemia in T2D models, representing a significant advancement toward a novel therapeutic paradigm for T2D patients," said Ryan Confer, President and Chief Executive Officer at Genprex. "The compelling evidence from the preclinical studies, which achieved complete rescue of HFD-induced hyperglycemia at four weeks post-treatment via direct intrapancreatic infusion, suggests the technical translatability of our diabetes gene therapy approach to human application, potentially through endoscopic retrograde cholangiopancreatography, offering a potentially promising avenue for long-term glycemic control in T2D."

The featured Genprex-supported abstract and poster at the 2026 ASGCT (Free ASGCT Whitepaper) Annual Meeting:

Title: "Pancreatic Delivery of AAV-Pdx1/MafA Reverses Hyperglycemia in a Preclinical Model of Type 2 Diabetes"
Abstract ID: 2419
Topic: Gene-Based Therapies in Pre-Clinical Models of Genetic Disease
Poster Presentation Date: Wednesday, May 13, 2026
Poster Presentation Time: 5-6:30 p.m. ET

In this study, eight-week-old male C57BL/6 mice were maintained on a regular diet (RD) or high fat diet (HFD) for 24 weeks. HFD mice then either remained unoperated or underwent intrapancreatic infusion of adeno-associated virus (AAV-8) encoding Pdx1 and MafA (PM) cassettes under the CMV promoter (global–islet cell targeting) or the rat insulin promoter (RIP) (β-cell–specific targeting) or received a control virus. The diet remained unchanged after surgery. At two and/or four weeks after surgery, researchers performed intraperitoneal glucose tolerance testing (IPGTT), insulin tolerance testing (ITT), glucose-stimulated insulin secretion (GSIS), calculated HOMA-IR and assessed glucagon secretion. Mice were then euthanized for pancreatic histology, quantification of β- and α-cell mass, electron microscopy (EM), and islets were isolated for ex-vivo glucose-stimulated insulin secretion (GSIS) and single-cell RNA sequencing. The results at four weeks showed major improvements in the control of diabetes.

At four weeks after surgery, ex-vivo GSIS showed that islets isolated from HFD+CMV-PM-GFP treated mice had insulin secretion similar to islets from RD mice, and both groups had increased insulin secretion compared to islets from the control HFD groups, indicating improved β-cell function with PM treatment.

Similarly, and importantly, treatment of HFD mice with RIP-PM-GFP, which selectively targets β-cells, reversed hyperglycemia and improved ex-vivo GSIS. In addition, EM imaging showed that PM treatment in HFD mice increased the number of total and mature insulin granules and decreased the number of immature insulin granules compared with HFD controls. Furthermore, transcriptomic pseudotime analysis demonstrated a shift in β-cells from an immature state toward a more mature state after PM treatment.

PM gene therapy reverses hyperglycemia, likely in large part by specifically enhancing β-cell function and maturation. This approach is technically translatable to humans using endoscopic retrograde cholangiopancreatography to deliver PM gene therapy to the pancreas.

(Press release, Genprex, APR 28, 2026, View Source [SID1234664855])

BioLineRx and Hemispherian Announce First Patient Dosed in Phase 1/2a Study of GLIX1 for the Treatment of Glioblastoma (GBM)

On April 28, 2026 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical development stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases, and Hemispherian AS, a clinical-stage oncology company developing novel small molecule therapeutics, reported that the first patient has been dosed in the first-in-human, Phase 1/2a study of GLIX1 for the treatment of recurrent and progressive glioblastoma (GBM) and other high-grade gliomas. The GLIX1 program is being conducted under a collaboration between BioLineRx and Hemispherian announced in September 2025.

The patient was dosed at NYU Langone Health under the supervision of Dr. Alexandra Miller, Chief of Neuro-Oncology & Co-Director of Brain and Spine Tumor Center, Perlmutter Cancer Center. Northwestern University, led by lead investigators Dr. Roger Stupp and Dr. Ditte Primdahl, and Moffit Cancer Center, led by Dr. Patrick Grogan, will also be participating in the study.

GLIX1 is an oral, first-in-class, small molecule with a novel mechanism of action, designed to activate TET2 and drive tumor DNA damage. By restoring TET2 activity, GLIX1 induces DNA damage selectively in cancer cells, representing a differentiated approach to targeting the DNA damage response with potential applicability across a broad range of tumors. Glioblastoma was selected as the initial indication due to its highly suppressed TET2 activity and significant unmet medical need. GBM remains one of the most aggressive and treatment-resistant cancers, and there is an urgent need for breakthrough innovation and more effective treatment options.

In multiple pre-clinical models, including in-vivo GBM models, GLIX1 demonstrated potent anti-tumor activity, excellent blood-brain-barrier penetration, and a favorable safety profile.

"The dosing of the first patient in our Phase 1/2a study of GLIX1 is an important milestone for BioLineRx and, more importantly, for patients battling glioblastoma, a very challenging tumor where there has been very little innovation over the past 20 years," said Philip Serlin, Chief Executive Officer of BioLineRx. "We believe GLIX1 has the potential to offer a novel therapeutic approach in this cancer indication, as well as in multiple other cancer indications, where DNA damage repair is critical for cancer survival. We are excited to advance GLIX1 development into this first-in-human clinical trial and look forward to initial data in the first half of 2027."

Zeno Albisser, Chief Executive Officer of Hemispherian, added, "The initiation of patient dosing in this important study represents a watershed event for Hemispherian and GLIX1. This is the culmination of years of focused scientific and operational work, and an important step toward bringing a new therapeutic approach to patients with glioblastoma. We are encouraged by our preclinical data and look forward to generating initial clinical insights as the trial progresses."

Phase 1/2a clinical trial design (NCT07464925)

The Phase 1 part of the trial is a dose escalation part where patients receive GLIX1 daily as monotherapy. This part is expected to recruit up to 30 patients with recurrent and progressive GBM and other high-grade gliomas. The objective is to establish a maximum tolerated dose (MTD) and/or a recommended dose based on safety, PK/PD and preliminary efficacy. Data from the Phase 1 part of the trial are anticipated in H1 2027.

The Phase 2a expansion part of the trial is planned to include additional indications, including newly diagnosed GBM, as well as select cancers with/without standard of care (e.g., in combination with PARP inhibitors). These cohorts are expected to identify preliminary efficacy, PD assessments and dose optimization data, serving as the basis for a rapid and effective advanced clinical development plan.

(Press release, BioLineRx, APR 28, 2026, View Source [SID1234664852])

BioCryst to Present at Upcoming Investor Conferences

On April 28, 2026 BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) reported that the company plans to present at the following conferences:

Bank of America Health Care Conference in Las Vegas on Tuesday, May 12, 2026, at 4:40 p.m. ET.
Jefferies Global Healthcare Conference in New York on Wednesday, June 3, 2026, at 3:45 p.m. ET.

Links to the live audio webcasts and replays of the presentations may be accessed in the Investors & Media section of BioCryst’s website at www.biocryst.com.

(Press release, BioCryst Pharmaceuticals, APR 28, 2026, View Source [SID1234664851])

Azitra, Inc. Announces Poster Presentation at ASGCT 2026 Highlighting ATR-01 Program for Ichthyosis Vulgaris

On April 28, 2026 Azitra, Inc. (NYSE American: AZTR), a clinical stage biopharmaceutical company focused on developing innovative therapies for precision dermatology, reported the presentation of new preclinical data from its ATR-01 program at the 2026 Annual Meeting of the American Society of Gene & Cell Therapy ("ASGCT 2026").

The poster highlights Azitra’s engineered live biotherapeutic candidate ATR01-616, which is designed to treat ichthyosis vulgaris (IV) by delivering recombinant human filaggrin directly into the skin using a modified Staphylococcus epidermidis strain. IV is a common genetic skin disorder caused by filaggrin deficiency, leading to impaired skin barrier function and increased trans-epidermal water loss (TEWL).

The data being presented at ASGCT (Free ASGCT Whitepaper) 2026 highlight ATR01-616’s mechanism of action and translational potential, including its ability to elicit robust secretion of a recombinant human filaggrin domain, with peak production observed 6–8 hours following application. In an ex vivo pig skin model, ATR01-616 significantly reduced transepidermal water loss across all dose levels (p < 0.001), with levels returning near baseline within 20 hours. In parallel, studies in reconstructed human epidermis showed restoration of key structural features such as increased filaggrin levels and co-localization with keratin proteins, supporting functional integration into the skin barrier.

"These data provide compelling validation of our ATR-01 program and its potential to address the underlying cause of ichthyosis vulgaris," said Francisco Salva, Chief Executive Officer of Azitra. "By using the skin’s natural bacteria to deliver functional filaggrin domains deeper into the epidermis, we are advancing a differentiated approach designed to restore skin barrier function and target the protein to where it is needed to address this genetically driven disease. The findings presented at ASGCT (Free ASGCT Whitepaper) build on our previously reported positive preclinical data for ATR-01 and further support the program’s advancement toward IND-enabling studies and a first-in-human clinical trial in patients with ichthyosis vulgaris."

ATR01-616 is a topical formulation containing a genetically engineered S. epidermidis strain designed as an auxotroph for controlled growth and optimized to secrete therapeutic filaggrin fragments. This approach enables localized, sustained delivery of protein therapeutics directly to affected skin, potentially overcoming limitations of existing treatments that do not address underlying disease biology and positioning ATR-01 as a novel, microbiome-based modality within dermatology.

Poster Details

Title: An Engineered Human Filaggrin Secreting Staphylococcus epidermidis Strain for the Topical Treatment of Ichthyosis Vulgaris
Presenter: Roger Léger, Ph.D., Vice President of Chemistry, Formulation and Development, Azitra, Inc.
Abstract Number: 2691
Session: Gene Addition: Non-Viral Vectors
Meeting: ASGCT (Free ASGCT Whitepaper) 2026 Annual Meeting

(Press release, Azitra, APR 28, 2026, View Source [SID1234664850])

Avenzo Therapeutics Advances the Combination of CDK4 Selective Inhibitor (AVZO-023) with CDK2 Selective Inhibitor (AVZO-021) in Ongoing Phase 1/2 ORION-1 Study in HR+/HER2- Breast Cancer

On April 28, 2026 Avenzo Therapeutics, Inc. ("Avenzo"), a clinical-stage biotechnology company developing next-generation oncology therapies, reported initiation of the combination cohort evaluating AVZO-023, its potential best-in-class cyclin-dependent kinase 4 (CDK4) selective inhibitor, in combination with AVZO-021, its potential best-in-class cyclin-dependent kinase 2 (CDK2) selective inhibitor, with fulvestrant in patients with advanced or metastatic hormone receptor-positive (HR+)/human epidermal growth factor receptor 2-negative (HER2-) breast cancer in the ongoing Phase 1 portion of the ORION-1 Phase 1/2 clinical study.

"Hyperactivation of CDK2 has emerged as a key resistance mechanism to CDK4/6 inhibitors, and we believe addressing both CDK4 and CDK2 with selective inhibitors represents a rational approach to improving outcomes for patients with HR+/HER2- breast cancer," said Antonio Giordano, M.D., Ph.D., Clinical Director, Center for Cancer Therapeutic Innovation, Dana-Farber Cancer Institute. "The combination of AVZO-023 and AVZO-021 is designed to optimize CDK4 target coverage while simultaneously targeting CDK2-driven resistance, and the selective profiles of both agents may enable a tolerability advantage over less selective approaches."

The Phase 1/2 first-in-human, open-label ORION-1 clinical study is designed to assess the safety, tolerability, and preliminary clinical activity of AVZO-023 with endocrine therapy as well as the combination of AVZO-023 and AVZO-021 with endocrine therapy. The combination cohort will evaluate AVZO-023 and AVZO-021 with fulvestrant in patients with HR+/HER2- metastatic breast cancer. AVZO-021 is currently being studied in a separate Phase 1/2 clinical study in HR+/HER2- metastatic breast cancer and other advanced solid tumors, and the company plans to present updated safety and efficacy results from the Phase 1 portion of the ongoing study at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.

"We are encouraged by the early emerging data from AVZO-023 and believe the combination of our potential best-in-class CDK4 and CDK2 selective inhibitors has the potential to meaningfully improve outcomes for patients with HR+/HER2- breast cancer," said Mohammad Hirmand, M.D., Co-founder, and Chief Medical Officer of Avenzo Therapeutics. "We look forward to advancing this program and exploring the full potential of this novel combination."

(Press release, Avenzo Therapeutics, APR 28, 2026, View Source [SID1234664849])