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.

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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])

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

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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])

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").

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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])

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.

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"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])

On April 28, 2026 Asgard Therapeutics, a privately held biotech company pioneering in vivo direct cell reprogramming for cancer immunotherapy, reported it will present advanced preclinical data on its lead asset AT-108, a first-in-class, off-the-shelf cancer immunotherapy, in an oral presentation at the ASGCT (Free ASGCT Whitepaper) Annual Meeting, held from 11-15 May, 2026, in Boston, Massachusetts, US.

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Fábio Rosa, Co-founder and VP and Head of Research at Asgard Therapeutics, who will give the oral presentation, said: "These data demonstrate the ability of AT-108 to reprogram tumor cells in situ into antigen-presenting cells with high efficiency, systematically driving activation of tumor-specific immune responses within the tumor microenvironment. This work provides clear preclinical evidence supporting our in vivo cell reprogramming platform and represents another milestone for Asgard as we advance AT-108 toward the clinic."

Shane Olwill, Chief Development Officer at Asgard Therapeutics, said: "We are delighted to have been selected for an oral presentation at ASGCT (Free ASGCT Whitepaper), which will highlight the progress we have made advancing AT-108 – our first-in-class, off-the-shelf gene-based immunotherapy designed to trigger powerful, personalized anti-cancer responses. Our pioneering approach to tackling cancer is potentially transformative and we are excited by AT-108’s broad potential across multiple cancer types."

Asgard had previously demonstrated that intratumoral delivery of AT-018 reprograms tumor cells into cDC1-like antigen-presenting cells and works synergistically with immune checkpoint blockade (ICB) to elicit powerful anti-tumor activity.

This new study, being presented at ASGCT (Free ASGCT Whitepaper) 2026, advances AT-108 across four key dimensions. It demonstrates AT-108’s ability to induce systemic anti-tumor immunity in mouse models leading to abscopal effect and regression on non-treated tumors in the same animals; it establishes AT-108 as the optimized clinical candidate following screening of >25 cassette variants; it defines dosing and treatment parameters required for durable responses; and it identifies pharmacodynamic biomarkers associated with reprogramming and immune activation.

Further highlights of the study include that:

In combination with ICB, Asgard’s approach induced abscopal effects and long-term tumor-free survival in the B16 mouse syngeneic model. These results were associated with increased T cells and NK cells, and reduced regulatory T cells, in both injected and non-injected tumors.
When used as a monotherapy, AT-108 doubled median survival in B16 and YUMM1.7 mouse models, and induced regression in ID8 ascites mouse model.
When used in combination with ICB, AT-108 achieved 50% complete response (CR) in B16, and extended survival in the PANC02 immunosuppressed mouse model.
Cell transduction peaked 1-2 days post injection and persisted for up to 9-15 days, supporting re-dosing every two days to sustain transduction. A three-dose lead cycle was required to achieve CRs, with maintenance dosing improving durability.
Regarding biomarkers, tumor and peripheral blood analyses identified pharmacodynamic signatures associated with AT-108 activity, including increased cytotoxic T cells, expansion of follicular helper T cells and enrichment of dendritic cells.
The abstract is available to view via the ASGCT (Free ASGCT Whitepaper) interactive program here.

The positive study results come as Asgard progresses AT-108 toward clinical development with a focus on solid tumors, by advancing IND-enabling studies and CMC development.

Details of the oral presentation are as follows:

Presentation title: Optimized in situ tumor-to-dendritic cell reprogramming by AT-108 adenoviral vector drives local and systemic antitumor immunity

Presenter: Fabio Rosa, Asgard Therapeutics

Presentation session: Cancer vaccines and oncolytic viruses I

Session date and time: 13 May 2026, 03:30 PM – 05:00 PM EDT

Location: MCEC Room 162AB (Level 1)

Presentation Time: 04:30 PM – 04:45 PM EDT

(Press release, Asgard Therapeutics, APR 28, 2026, View Source [SID1234664848])