SMALL CAP IDEA: Why investors should add Hemogenyx Pharmaceuticals to their watchlist

In a recent article, I said at least 20 micro-cap life sciences stocks were waiting to pop once interest returned to the sector.

Investors can now add Hemogenyx Pharmaceuticals to the watch list after it pulled off the near impossible by raising $4.2million at a time when investors have largely gone into hibernation.

This feat of resourcefulness, which will fund the initial stages of a first-in-human study of the drug developer’s lead asset, HEMO-CAR-T, only tells part of the story.

The pedigree of the scientific team backing the efforts of Hemogenyx is impressive.

At the same time, the interest in the next generation of CAR-T – the type of therapy that Hemogenyx is developing – amongst big pharma has seen the likes of AstraZeneca, Johnson & Johnson, Abbvie and Novartis top the billion-dollar threshold in milestone and royalty payments.

The pedigree of the scientific team backing the efforts of Hemogenyx is impressive

Upfront payments, usually a good litmus test of ‘real’ interest from these titans of the world of drugs, have been eye-watering; in some cases up to $100million.

The reason? CAR-T offers something that the healthcare industry rarely comes up with – a curative treatment, showing the world has truly moved on from the era of me-too, once-a-day pills such as statins.

Anyway, before we delve into the nuts and bolts of the Hemogenyx investment case, it is probably worth explaining just what CAR-T therapy is and how it might revolutionise the treatment of cancers such as acute myeloid leukaemia (AML), which is the particular area of interest of this UK biotech.

CAR-T, or chimeric antigen receptor T-cell therapy, redefines the battle against the disease by reprogramming the body’s own natural defences, called T-cells, to recognise and kill cancer cells.

This advanced form of immunotherapy involves extracting T-cells from a patient, genetically engineering them to target cancer cells, and then reintroducing these enhanced cells into the patient’s bloodstream.

CAR-T has demonstrated remarkable success in tackling certain blood cancers, offering new avenues for combating previously untreatable cancers.

Despite its potential, much of the research is now focused on CAR-T therapy’s significant side effects, while broadening its applicability to a wider array of cancers.

Hemogenyx will use the newly raised funds to take HEMO-CAR-T into the clinic to treat AML later this year.

The open-label study will test for any potential side effects from treating people with blood-borne diseases and assess how the therapy interacts with the body.

A secondary goal or ‘endpoint’ will be whether any of the critically ill AML patients chosen for the trial respond to the CAR-T infusion.

Under the strict guidelines laid down by the US Food & Drug Administration, patients will be dosed individually one month apart, recognising people are being administered with a potentially toxic cells.

Hemogenyx hopes to have the results from ‘two, or even three patients’ in time for the American Society of Hematology conference in early December.

Success at this stage would be a 30% response rate, says Dr Vladislav Sandler, chief executive and co-founder of Hemogenyx.

Investigational work will be carried out by a team from the University of Pennsylvania, led by Professor Nolle Frey, who has headed a number of important CAR-T clinical trials.

The entre was provided by Professor Carl June, the ‘father’ of CAR-T, and his colleague, Dr Saar Gill, who was involved in the preclinical research on HEMO-CAR-T.

On the university’s involvement, Sandler said: ‘This isn’t a situation where we’ve paid [UPenn] to carry out the work. They don’t work like that. All along it been data driven.’

While no guarantee of success, it provides validation for the science behind the efforts of Hemogenyx.

So expensive is the process of creating individual CAR-T treatments, that cash raised in February may be enough to dose three patients.

Hemogenyx will look to further non-dilutive funding to assess a cohort of around 18 AML sufferers, which will take it to the phase II stage of the clinical evaluation.

This is key, as it is the point that big pharma has weighed into the sector with the deals mentioned above.

If all of this suggests that Hemogenyx is a one-trick pony, then that would be misleading.

Before moving into the area of CAR-T, its team together with Eli Lilly, developed a breakthrough that enhances bone marrow and stem cell transplant procedures.

The approach focuses on the initial critical stages of eradicating diseased cells and replacing them with healthy ones, using a CDX bi-specific antibody that is both safer and more effective than traditional chemotherapy.

‘We decided to do something that other people wouldn’t touch at that time,’ says Sandler. This has been a theme with all of Hemogenyx’s research – attempting the difficult.

As such, the company has created and is a leader in the field of chimeric bait receptors. Put simply, CBRs are designed to attract and catch harmful cells, like cancer cells, by pretending to be something the harmful cells want to attach to.

Once the bad cells latch onto these bait receptors, the immune system can easily find and destroy them.

Its research is being deployed to combat brain cancers such as glioblastoma and neurodegenerative diseases where delivering therapeutics across the blood-brain barrier has long been problematic.

At the same time, it is working on intranasal delivery of CBRs to tackle airborne viral infections, an area of research germane to the civil defence industry and the military.

While Sandler wouldn’t be drawn on exactly who he is talking to, there are discussions with parties interested in helping develop the viral infections defense deployment, the CEO confirmed.

‘We are talking to institutions and agencies,’ Sandler says. ‘There is a lot of interest and we are moving these conversations along.’

The Ice Age-like big freeze across the small-cap market in the UK means none of company’s potential has been captured in the current share price, which values Hemogenyx at a bargain basement £21million.

If we accept this is a cyclical anomaly, the rebound could be dramatic – not just for the sector, but individual stocks such as Hemogenyx.