Feasibility Study Agreement

Study to evaluate targeted lipid nanoparticle delivery of Hemogenyx’s CAR mRNA - a potential route to an off-the-shelf, in vivo CAR-T therapy

Hemogenyx Pharmaceuticals plc (LSE: HEMO), the clinical-stage biopharmaceutical group announces that it has entered into a Feasibility Study Agreement (the “Agreement”) with Nitto Denko Corporation (“Nitto”) (TSE: 6988), the Japan-based manufacturer of high-performance materials.

Under the Agreement, the parties will evaluate whether Nitto’s cell-targeted lipid nanoparticle (“ctLNP”) drug delivery technology can deliver Hemogenyx’s messenger RNA (“mRNA”) encoding its FLT3-targeting chimeric antigen receptor (“CAR”) to human T cells (the “Study”).

Highlights

• The Study is the Company’s first step towards an in vivo CAR-T approach, in which a patient’s T cells are reprogrammed inside the body by an injectable medicine rather than engineered individually in a manufacturing facility.
• If the approach proves successful, it could allow Hemogenyx’s CAR-T technology to be converted into a drug-like, off-the-shelf product that is significantly easier to manufacture, store and distribute than traditional autologous CAR-T therapy.
• The Study combines Hemogenyx’s proprietary FLT3-CAR mRNA with Nitto’s ctLNP technology; each party retains ownership of its own technology.
• The Study is an in vitro evaluation, is expected to take approximately four months.
• The Company’s ongoing Phase I clinical trial of HG-CT-1 in relapsed/refractory acute myeloid leukemia (“R/R AML”) is unaffected and remains its principal focus.

Strategic rationale

HG-CT-1, the Company’s lead product candidate, is an autologous CAR-T cell therapy: each dose is made from the individual patient’s own T cells, which are collected, genetically engineered and expanded outside the body before being returned to that patient. While autologous CAR-T therapies have transformed the treatment of certain blood cancers, this patient-by-patient manufacturing model is complex, time-consuming and costly, and it limits the number of patients and treatment centers that can be served.

An in vivo approach seeks to remove that constraint. mRNA encoding the CAR is packaged in lipid nanoparticles designed to seek out T cells; once administered, the nanoparticles deliver the mRNA to the patient’s T cells, which then produce the CAR themselves. The therapeutic product is therefore not a living, patient-specific cell preparation but a defined pharmaceutical formulation that could, in principle, be manufactured in large batches, held in inventory and administered to any eligible patient without cell collection or a bespoke manufacturing run for each individual. Because mRNA-driven CAR expression is transient, such a product may also allow CAR-T cell activity to be controlled through dosing.

The Company believes that, if technically feasible, an in vivo product built on the Company’s FLT3-CAR could materially broaden the reach and commercial potential of Hemogenyx’s CAR-T technology, complementing the autologous HG-CT-1 program currently in the clinic.

Dr Vladislav Sandler, Chief Executive Officer & Co-Founder of Hemogenyx Pharmaceuticals, commented:

“Autologous CAR-T therapy works, but every dose is a bespoke manufacturing campaign for a single patient. In vivo CAR-T offers a fundamentally different model: a drug-like product, made at scale and available off the shelf, that instructs the patient’s own T cells to become CAR-T cells inside the body. This Study with Nitto, a recognized leader in nucleic acid medicine and drug delivery, opens that opportunity for Hemogenyx. It allows us to test, at modest cost, whether our FLT3-CAR can be delivered by targeted lipid nanoparticles, and so whether the technology behind HG-CT-1 can be translated into a product that is significantly easier to manufacture and to deliver to patients. Our priority remains the HG-CT-1 Phase I trial; this work runs alongside it and, if successful, could considerably extend the value of our CAR-T platform.”

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About Nitto Denko Corporation

Since its founding in 1918, Nitto has globally provided a wide range of products and services as a high-performance materials manufacturer, including optical materials used in display devices such as smartphones and automotive displays, advanced semiconductor materials, high density printed circuit boards for data centers, medical-related products and services, and decarbonization solutions, based on its core technologies. Under its brand slogan, “Innovation for Customers,” Nitto aims to create indispensable value for society and contribute to a sustainable global environment and a prosperous human society.