Operations Update LONDON, UNITED KINGDOM / ACCESSWIRE / June 17, 2024 / Hemogenyx Pharmaceuticals plc (LSE: HEMO), the clinical stage biopharmaceutical group developing treatments for cancers and viral diseases, is pleased to provide an update on its ongoing activities and progress. 1. HEMO-CAR-T Clinical Trials Progress: Additional Clinical Trial Site: The Company has added an additional prestigious US medical center to its list of clinical trial sites. This will enhance the scope and expedite the timeline for the implementation of clinical trials. Clinical Site Setup: The setup of clinical sites at the University of Pennsylvania ("PENN") and the new medical centre is underway. This multi-step process involves thorough internal reviews by the science review committee and the internal review board of both clinical sites, as well as budget negotiations and logistics planning. The Company continues to work with Prevail Infoworks, a contract research organization ("CRO"), to manage and oversee the planning for the clinical trials. Expansion into Pediatric Indications: Pediatric AML and ALL: The Company is seeking to expand the HEMO-CAR-T indication to include pediatric acute myeloid leukemia ("AML") and a subset of pediatric acute lymphoblastic leukemia ("ALL") patients. An amendment to the clinical protocol to include pediatric AML is currently under review by third party experts. If approved, the Company will seek to implement the clinical trials for pediatric AML at the newly established clinical site. This area is of particular concern, and there is an urgent need for effective treatments for these conditions, which the Company believes HEMO-CAR-T will be able to provide. 2. Chimeric Bait Receptor ("CBR") Platform The Company's CBR platform is an advanced immunotherapy designed to reprogram or redirect innate immune cells, such as macrophages, to prevent and combat infections from both existing and emerging viral threats, as well as to eliminate specific types of cancer. Development and Testing: CBR Constructs: Scientists at Hemogenyx Pharmaceuticals are developing and testing multiple CBR constructs to identify the best candidates for targeting rare cancers such as epithelial ovarian carcinoma. Selected candidates will undergo rigorous testing to advance them to investigational new drug (IND) enabling studies. mRNA-Based Delivery of CBR: Intranasal Delivery for Viral Infections: The Company continues to develop mRNA-based delivery of CBRs for treating airborne viral infections via intranasal administration. Recent improvements in the stability of mRNA-based CBRs are expected to enhance the effectiveness of this treatment. 3. CDX - Bispecific Antibody IND-Enabling Studies: Progress: The Company is advancing IND-enabling studies for CDX, a bispecific antibody designed for treating relapsed and/or refractory AML, a subset of ALL, and for conditioning in bone marrow transplants. Dr Vladislav Sandler, CEO & Co-Founder of Hemogenyx Pharmaceuticals, commented: "We are excited about the progress we are making across our various programs. The addition of a world-renowned medical center to our HEMO-CAR-T clinical trial sites marks a significant step forward in our mission to develop life-saving therapies. Our expansion into pediatric indications for HEMO-CAR-T highlights our commitment to addressing unmet medical needs in both adult and pediatric populations. "Furthermore, our advancements in the CBR platform and the development of mRNA-based delivery systems for treating airborne viral infections demonstrate our innovative approach to tackling complex diseases. The progress in our CDX bispecific antibody program also underscores our dedication to bringing effective treatments to patients with relapsed or refractory AML and other severe conditions. "We are diligently pursuing non-dilutive financing options to support these initiatives and remain focused on translating our scientific discoveries into clinical success. We look forward to updating our shareholders and the market as we continue to make strides in our development programs." Market Abuse Regulation (MAR) Disclosure Certain information contained in this announcement would have been inside information for the purposes of Article 7 of Regulation No 596/2014 (as it forms part of UK domestic law by virtue of the European Union (Withdrawal) Act 2018) until the release of this announcement. Enquiries: Hemogenyx Pharmaceuticals plc Dr Vladislav Sandler, Chief Executive Officer & Co-Founder headquarters@hemogenyx.com Peter Redmond, Director peter.redmond@hemogenyx.com SP Angel Corporate Finance LLP Tel: +44 (0)20 3470 0470 Matthew Johnson, Vadim Alexandre, Adam Cowl Peterhouse Capital Limited Tel: +44 (0)20 7469 0930 Lucy Williams, Duncan Vasey, Charles Goodfellow About Hemogenyx Pharmaceuticals plc Hemogenyx Pharmaceuticals is a publicly traded company (LSE: HEMO) headquartered in London, with its US operating subsidiaries, Hemogenyx Pharmaceuticals LLC and Immugenyx LLC, located in New York City at its state-of-the-art research facility. The Company is a clinical-stage biopharmaceutical group developing new medicines and treatments for life-threatening diseases. Hemogenyx Pharmaceuticals is developing several distinct and complementary product candidates, as well as a platform technology that it uses as an engine for novel product development. This information is provided by RNS, the news service of the London Stock Exchange. RNS is approved by the Financial Conduct Authority to act as a Primary Information Provider in the United Kingdom. Terms and conditions relating to the use and distribution of this information may apply. For further information, please contact rns@lseg.com or visit . SOURCE: Hemogenyx Pharmaceuticals PLC View the original press release on accesswire.com

ImmunotherapyClinical Study

09 Feb 2024

·www.biospace.com

Hemogenyx Pharmaceuticals PLC Announces FDA Consents to Phase I Trials of HEMO-CAR-T

FDA Consents to Phase I Trials of HEMO-CAR-T LONDON, UK / ACCESSWIRE / February 9, 2024 / Hemogenyx Pharmaceuticals plc (LSE:HEMO) is pleased to announce that it has been informed by the U.S. Federal Food and Drug Administration ("FDA") that it has lifted the clinical hold on the Investigational New Drug (IND) application for HEMO-CAR-T for the treatment of acute myeloid leukemia ("AML"). The FDA confirmed that the Company had addressed all issues identified in its prior clinical hold letter satisfactorily and consents to the Company proceeding with its Phase I clinical study of HEMO-CAR-T. Dr Vladislav Sandler, CEO & Co-Founder of Hemogenyx Pharmaceuticals, commented: "We are extremely pleased with the FDA's decision to lift the clinical hold. We now look forward to accelerating clinical development of HEMO-CAR-T and to offering patients a potentially life-saving treatment. The removal of the clinical hold was made possible by the hard work and dedication of the entire Hemogenyx Pharmaceuticals team and its Board of Directors and advisors." About AML and CAR-T Therapy AML, the most common type of acute leukemia in adults, has poor survival rates (a five-year survival rate of less than 30% in adults) and is currently treated using chemotherapy, rather than the potentially more benign and effective form of therapy being developed by Hemogenyx Pharmaceuticals. The successful development of a new therapy for AML would have a major impact on treatment and survival rates for the disease. CAR-T therapy is a treatment in which a patient's own T-cells, a type of immune cell, are modified to recognize and kill the patient's cancer cells. The procedure involves: isolating T-cells from the patient; modifying the isolated T-cells in a laboratory using a CAR gene construct (which allows the cells to recognize the patient's cancer); amplifying (growing to large numbers) the newly modified cells; and re-introducing the cells back into the patient. Market Abuse Regulation (MAR) Disclosure Certain information contained in this announcement would have been inside information for the purposes of Article 7 of Regulation No 596/2014 (as it forms part of UK domestic law by virtue of the European Union (Withdrawal) Act 2018) until the release of this announcement. The person responsible for arranging for the release of this announcement on behalf of Hemogenyx Pharmaceuticals plc is Dr Vladislav Sandler, Chief Executive Officer & Co-Founder. Enquiries: Hemogenyx Pharmaceuticals plc Dr Vladislav Sandler, Chief Executive Officer & Co-Founder headquarters@hemogenyx.com Peter Redmond, Director Peter.redmond@hemogenyx.com SP Angel Corporate Finance LLP Tel: +44 (0)20 3470 0470 Matthew Johnson, Vadim Alexandre, Adam Cowl Peterhouse Capital Limited Tel: +44 (0)20 7469 0930 Lucy Williams, Duncan Vasey, Charles Goodfellow About Hemogenyx Pharmaceuticals plc Hemogenyx Pharmaceuticals is a publicly traded company (LSE:HEMO) headquartered in London, with its US operating subsidiaries, Hemogenyx Pharmaceuticals LLC and Immugenyx LLC, located in New York City at its state-of-the-art research facility. The Company is a pre-clinical stage biopharmaceutical group developing new medicines and treatments to treat blood and autoimmune disease and to bring the curative power of bone marrow transplantation to a greater number of patients suffering from otherwise incurable life-threatening diseases. Hemogenyx Pharmaceuticals is developing several distinct and complementary product candidates, as well as a platform technology that it uses as an engine for novel product development. This information is provided by RNS, the news service of the London Stock Exchange. RNS is approved by the Financial Conduct Authority to act as a Primary Information Provider in the United Kingdom. Terms and conditions relating to the use and distribution of this information may apply. For further information, please contact rns@lseg.com or visit . SOURCE: Hemogenyx Pharmaceuticals PLC View the original press release on accesswire.com

ImmunotherapyPhase 1INDCell Therapy

09 Feb 2024

·www.pharmaceutical-technology.com

Hemogenyx gets reprieve as FDA lifts clinical hold on AML CAR-T therapy

The FDA placed a clinical hold on the trial for HEMO-CAR-T in June, requesting additional information from Hemogenyx. Credit: JHVEPhoto via Shutterstock. Hemogenyx has received the all-clear from the US Food and Drug Administration (FDA) to begin a Phase I clinical trial of its acute myeloid leukaemia (AML) chimeric antigen receptor (CAR) T-therapy in the US. In June 2023, the US FDA placed a clinical hold on the trial for HEMO-CAR-T, requesting additional information from Hemogenyx after a splicing deficiency during manufacture of the lentivirus used to produce CAR-T cells. Hemogenyx produced a plan, supported by laboratory tests to address FDA’s concerns in August 2023, which was accepted the following month. In January, the London, UK-based biopharma issued a complete response to the agency to lift the hold. Hemogenyx’s stock price has increased by 62% following the announcement of the lifting of the hold today (9 February) since the market close yesterday. CAR-T therapies have been hitting the headlines recently after the FDA launched an investigation into the incidence of potentially associated secondary malignancies in November. A month later, Johnson & Johnson and Legend Biotech added a boxed warning to jointly-developed therapy Carvykti (ciltacabtagene autoleucel; cilta-cel) after Phase II data from the CARTITUDE-1 trial (NCT03548207) showed that myeloid neoplasms occurred in 10 out of 97 patients post treatment. Last month, the FDA added similar boxed warnings on additional CAR-T therapies . Despite this, the CAR-T cell therapy market is still expected to grow, with many professionals arguing that the benefits outweigh the risks associated with the therapies. According to a report on GlobalData’s Pharma Intelligence Center, the total market value for both cell therapies and established/traditional AML therapies, is expected to increase from $3.1bn in 2021 to $6.62bn in 2031 globally. See Also: Why patient centricity is the answer to the clinical trial enrolment gap Pfizer and Saama expand deal to expedite clinical research GlobalData is the parent company of Pharmaceutical Technology. AML, a cancer that affects the bone marrow and blood, is characterised by the rapid growth of abnormal white blood cells, which interfere with the production of normal blood cells. The current standard of care for AML is chemotherapy, with the cancer generally having a bad prognosis. HEMO-CAR-T comprises T cells genetically engineered to express chimeric antigen receptors, targeting cells expressing Fms-like receptor tyrosine kinase 3 (FLT3). T cells are extracted from the patient, modified to target specific proteins on cancer cells, and then infused back into the patients’ bloodstream. In the announcement accompanying the announcement, CEO of Hemogenyx Vladislav Sandler said: “We are extremely pleased with the FDA’s decision to lift the clinical hold. We now look forward to accelerating clinical development of HEMO-CAR-T and to offering patients a potentially life-saving treatment.” Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Cell and gene therapies: Pipe dream to pipeline The cell and gene industry is gaining momentum, with a new wave of therapies promising to transform the way doctors treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead. By Cytiva Thematic By downloading this case study, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

Cell TherapyImmunotherapyGene TherapyPhase 2Phase 1

100 Deals associated with HEMO-CAR-T cell therapy (Hemogenyx)

R&D Status

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Acute Myeloid Leukemia	Phase 1	US	HemoGenyx Pharmaceuticals Plc	-

Clinical Result

Indication

Phase

Evaluation

View All Results

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Chat with Hiro

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis


No Data

HEMO-CAR-T cell therapy (Hemogenyx)

Overview

Basic Info

Related

R&D Status

Clinical Result

Translational Medicine

Deal

Core Patent

Clinical Trial

Approval

Regulation