Positive news due for for Hemogenyx Pharmaceuticals ?
“…there seems no reason to suppose the clarification is anything other than another step in the long and winding process of securing approval for Phase I trials to begin…”
Hemogenyx Pharmaceuticals (LSE:HEMO), a biopharmaceutical company pioneering new therapies and treatments for blood diseases, is on the cusp of gaining permission from US regulators for Phase I clinical trials of its HEMO-CAR-T therapy, a groundbreaking treatment for acute myeloid leukaemia (AML).
The company has also advanced its proprietary Chimeric Bait Receptor (CBR) platform technology, which uses synthetic biology and artificial intelligence to program immune cells to eliminate viral infections by destroying the viruses that cause them.
HEMO’s research focuses on the development of accessible and efficient treatments for blood cancers such as AML and lymphoma, and severe autoimmune diseases like multiple sclerosis, aplastic anemia and systemic lupus erythematosus.
Traditional treatments require patients to undergo tough conditioning processes involving the use of radiation that can have severe side effects, including damage to the heart or lungs, thyroid problems, fertility problems, bone damage, and the nurture of other cancers. Many who would benefit from effective treatments like bone marrow transplants are too weak to withstand these preparatory phases.
HEMO is pioneering techniques that would facilitate a much gentler treatment process. The company is developing a proprietary HEMO-CAR-T immune therapy, a treatment in which a patient’s own ’T cells’, a type of immune cell, are modified to recognise and kill cancer cells. The therapy promises to be a key potential treatment for patients suffering from advanced stage AML. HEMO is also developing CD3-FLT3 CDX cellsbispecific antibody redirecting a patient’s immune cells to eliminate unwanted leukaemic and blood stem cells, better preparing patients for bone marrow transplantation. Use of CDX bispecific antibody would eliminate AML cells and the need for chemotherapy or radiation..
The company is seeking permission from the US Federal Drugs Administration (FDA) to proceed to Phase I clinical trials for both HEMO-CAR-T and CDX antibodies. Its work is based on groundbreaking research by co-founder and CEO Dr Vladislav Sandler, a widely published stem cell scientist who also holds a professorship at the State University of New York. Last summer HEMO opened a custom-designed laboratory in the city, a state-of-the-art research facility allowing the company to manufacture cell therapies in-house, accelerating and simplifying the research and commercialisation of its product candidates. The new facility plugs Hemogenyx Pharmaceuticals -into a rich local life sciences ecosystem including Columbia University and the City College of New York.
HEMO-CAR-T in focus
As noted in our previous coverage of HEMO, the company’s primary focus over the past couple of years has been preparing HEMO-CAR-T for its IND application, a process HEMO has acknowledged has been even ‘more complex and hence longer and more intensive than we had anticipated’. Preparations have included the design and conduct of numerous IND-enabling studies , feedback and guidance from a pre-IND submission to the FDA to guide the final application, and advice from a committee of experts in the treatment of leukaemia. During the past few months the internal and third-party tests necessary to complete the detailed IND submission pack have been completed, including development runs of the end-to-end process for the manufacture of HEMO-CAR-T cells, and exhaustively documented engineering runs under real-world conditions. These cell manufacturing trials were followed by analytical release tests conducted by the company and a third party to ensure that the manufactured HEMO-CAR-T cells comply with a set of required quality attributes, notably the viability, potency and sterility of the resulting cells.
HEMO was finally able to submit the application in May, and earlier this month received a notice from the FDA requesting further information. In the meantime HEMO’s work on the product has been put on ‘clinical hold’, with ‘an FDA letter to be received within 30 days setting out additional information required to be provided by the Company.’ Dr Sandler commented: ‘We are confident that we will be able to address the FDA’s questions and concerns regarding the IND. AML has poor survival rates and we are eager to resolve this hold and continue down the treatment development pathway toward saving lives.’
CBR and CBX progress
While pressing ahead with HEMO-CAR-T the company has made significant progress towards developing its CBR technology. The platform was evolved in the course of HEMO’s cell cancer research, which indicated how cell therapy approaches might be used to treat major existing and emerging viral infections.
CBR identifies protective cells and molecules including macrophages that can be activated to kill virus infected cells and protect against viruses including SARS-CoV-2, Dengue, Ebola, Marburg, Zika and Chikungunya. Earlier this year HEMO announced that further research had identified a further efficiency, a target protein that can be incorporated into a single multipurpose CBR-based therapeutic capable of treating multiple viruses that belong to different viral families, instead of having to make a separate CBR construct for every virus. The platform would allow off-the-shelf cell therapies against infectious disease, and the development biopharmaceuticals for eliminating viruses that might develop in the course of infection, or for use as preventive treatments.
HEMO says: ‘When fully developed, we would be able to create front-line treatments that may prevent the development of the next pandemic. Moreover, these new therapeutic tools can be used to protect against bio-terrorism, potentially rendering a universe of viral bio-weapons ineffective.’ A big claim: but the company’s belief in the significance and broad applicability of the proprietary technology led it to file a seminal provisional patent application last year.
HEMO has also continued developing its other major pipeline asset, the CDX antibody the company hopes will provide an alternative means of treating AML and of conditioning patients for bone marrow transplants. Early last year it entered into a service agreement with Selexis to develop a ‘master cell line’ that will be used to produce CDX antibodies for future clinical trials and patient treatments. The partners will use Selexis’ SUREtechnology Platform, a suite of cell line development tools and technologies that reduces the time, effort and cost in developing high-performance mammalian cell lines. Described by HEMO as ‘an important step in moving CDX towards clinical trials’ the platform facilitates the rapid, stable, and cost-effective production of recombinant proteins and vaccines, ensuring seamless integration of the development continuum from discovery to commercialisation. HEMO is exploring ways to finance and further the IND-enabling testing of CDX, participating in ‘early-stage conversations with potential development partners.’
The company has also engaged in fundraising. HEMO’s final results to 31 December 2022 stated a loss of £3,986,982 (2021: £5,108,310 loss), including an increased operating loss of £3,997,548 (31 December 2021: £2,702,754) marking ‘the increasing volume of work and need to engage external service providers as our assets are taken towards the crucial clinical trial stage of their development’. A placing this January raised a gross £4,056,250, which will be used to facilitate progression of the company’s HEMO-CAR-T product candidate into clinical trials and to enable continued development of product candidates for the treatment of viral infections based on the CBR platform.
Outlook
The FDA’s request for further information from HEMO earlier this month knocked the company’s share price, which currently stands at 1.7p, down from 2.3p, taking its market cap to £19.5m. But there seems no reason to suppose the clarification is anything other than another step in the long and winding process of securing approval for Phase I trials to begin. Speaking to TMS earlier this month, Dr Sandler said: ‘[W]e are optimistic as ever and unlike the market we don’t recoil when faced with hiccups. Basically, we double down our efforts and power through. That’s what we have been doing since we went into operations over eight years ago and it’s the keystone of our company’s values and culture.’ It’s worth noting that HEMO’s share price was nearly 4p as recently as January: positive news regarding the application would surely see the company’s value bounce again.
