Blood cancers are some of the most difficult to treat forms of the killer disease, but Hemogenyx Pharmaceutical PLC (LON:HEMO), a small, recently-listed biotech, claims to have made a major breakthrough.
While the UK biotech sector is littered with skeletons of companies promising the next big thing, Dr Vladislav Sandler, Hemogenyx's founder and chief executive, believes clinical proof may be available in the not-so-distant future.
Three major forms
Blood cancers take three major forms: Leukaemia, Lymphoma and Myeloma, all of which involve problems in a person’s bone marrow, the source of the cells in our blood - red, white or platelets.
The current standard of care treatment involves chemotherapy and radiotherapy and ultimately, in recurring cases, a bone marrow transplant.
Two major problems
All current treatments are dangerous, highly toxic and debilitating, with serious side effects.
Indeed, so traumatic is the transplant option that many patients are rejected on the grounds that they will not survive the conditioning process.
After that comes the problem of finding a donor, with 2,000 people in the UK alone in need of a bone marrow or stem cell transplant every year according to Anthony Nolan, the UK’s leading blood cancer charity.
Hemogenyx has developed products that deal with both problems and offer the possibility of transforming the current treatments for blood cancers, says Sandler.
Partially based around work he undertook at Cornell University, which is now a shareholder in Hemogenyx, they target a market estimated to be currently worth between US$8bn - US$9bn.
Hemogenyx believes it can solve problems
CDX antibodies, the first product, tackle the pre-transplant, conditioning stage.
A more benign alternative to the current standard of care, it will eliminate chemotherapy and radiotherapy by targeting and killing only “unwanted” cells (not everything) and allowing many more people who would otherwise be deemed unfit enough to receive a transplant.
Pre-clinical trials on humanised animals have already proved it as a concept, says Sandler.
A Phase I clinical trial is scheduled for 18 months and though only a safety study, it will give a good indication of its potential.
Trial patients will only have a few months to live and they will either survive after treatment or not.
“It will be a binary result“
Hemogenyx’s second product effectively replaces a patient’s cancer-infected blood with a new, clean, supply generated from their own stem cells.
As these blood stem cells have not been through the body’s circulatory system, they have not accumulated the mutations or chromosome rearrangements that cause the cancer.
“In effect, they reset blood generation back to zero” says Sandler.
These blood stem-like cells can also be found in everybody’s liver, he adds.
Phase I funding in place
Hemogenyx has licensed the IP for the Hu-PHEC therapy from Cornell, but it owns the rights to this CDX antibodies technology.
The company's main market listing raised £2mln, which following a pre-IPO round of financing, has raised enough funds to carry it through to the Phase 1 trial of the CDX antibodies.
Four more pieces of pre-clinical work need to be carried out before then, says Sandler, after which it will also apply for US orphan drug status to give it additional commercial protection for seven years.
Work on the Hu-PHEC therapy will be focused on the proof of principle on cells taken from human livers, though orphan drug status for this treatment will also be sought eventually.
Hemogenyx is valued currently at £10mln (at 2.9p) but the price tag will not stay that low for long if the research and clinical work continues to impress.
Buoyant market for I/O firms
US immuno-oncology (I/O) companies recently have been changing hands for anything between US$500mln and US$1bn even before they reach the clinic.
A line on value may also be available through another US company, Actinium Pharmaceuticals, which is currently carrying out a Phase IIb trial for a first line treatment for acute myeloid leukaemia.
Actinium is a lot further down the development road and is valued at US$55mln currently. It is working in a similar field to CDX, with a treatment based around a radioactive isotope.
While in theory a competitor, a good trial result might also boost interest generally in bone marrow research.
Hemogenyx says it needs the help of a partner to get through the clinical process.
Intriguingly, Sandler hints at big pharma interest but says it is too early to give any details.
In eighteen months he may be spoilt for choice if it all goes to plan.