Noxopharm's Chemotherapy Program

The significance of the announcement on Friday 7 May, 2021 lies in the Company putting on the public record its formal commitment to its 4-pillars oncology strategy. We have mentioned this strategy in recent times, but the Company’s recent commitment to the CEP-2 trial with the appointment of an overseeing contract research organisation, formalises the strategy.

Behind this strategy is an aim to see Veyonda become a standard way of making most common forms of cancer therapy work better.

Such an aim is about as big as you can have in oncology. It’s one that we are not aware of anyone trying before, but that is probably because no-one has a drug with the unique ability of idronoxil (the active ingredient in Veyonda) to restore cancer-fighting immune function to tumours. Restoring that immune function increasingly is being recognised as a pre-condition to all forms of effective anti-cancer therapy. In a field of very few competing technologies, the Company is confident, based on public disclosures, that Veyonda is the only technology that specifically addresses the mechanism responsible for expelling immune cells from tumours.**

4-pillars strategy

This strategy involves using Veyonda across the current 3 mainstays of cancer therapy, along with an emerging 4th form of therapy - injectable radiopharmaceuticals:

Therapy NOX Program
Chemotherapy CEP
Checkpoint inhibitor therapy IONIC
Externally-delivered radiotherapy DARRT
Radiopharmaceuticals  LuPIN

Value creation crossed with risk mitigation

The bigger the aim, the bigger the potential pay-off, but also the bigger the risk. The 4-pillars concept is designed to address that risk-reward trade-off.

Value creation.  Success in any one of the 4 programs has the potential to put Veyonda on the world map; success in 2, 3 or all 4 programs would be worth …..???  I will leave it to the market and the pharmaceutical industry to put a number on that one.

Risk mitigation - only about 4% of oncology drugs make it from Phase 1 to marketing approval, with most failure occurring in Phase 3. That is, only 1 in 25 anti-cancer drugs that start off in a Phase 1 clinical trial, end up making it to market. And this is across the full spectrum of the industry, from the small cap companies to the big end of town, with big pharma being just as likely to succeed or fail as smaller biotechs.

So, the more eggs you have in the basket, the better.

Chemotherapy market

We used the announcement to put our 4-pillars strategy on the public record because the Company last week made a formal commitment to its fourth pillar –  its CEP or Chemotherapy Enhancement Program.

In very broad terms, the current global values of the 4 market sectors are:

  US$ billions
Chemotherapy 50
Checkpoint inhibitor therapy 20
Externally-delivered radiotherapy 7
Radiopharmaceuticals  3

Chemotherapy is the highest value sector because chemotherapy remains the backbone of cancer therapy. That makes it the biggest source of income in the oncology sector for large pharma companies such as Roche, Novartis, Bristol Myers Squibb, Johnson and Johnson, Merck, GSK and AstraZeneca. Apart from jostling for a piece of the action in a very crowded field, most of these companies are facing running out of patent protection in the near future on some of their star products.

If you put the size of the chemotherapy market, together with some of the biggest companies in the world searching for a competitive edge, then you can understand why Noxopharm believes it is so important to include chemotherapy as a target for the treatment-boosting properties of Veyonda.

It’s worth re-emphasising that statistically, as a small biotech company, we have as much chance of success as any of the major pharmas. If that turns out to be the case… then our potential success could well be their success too.

Veyonda + doxorubicin + soft tissue sarcomas

With CEP back on the agenda, the next task was to settle on the appropriate drug/cancer combination. With over 40 chemotherapy drugs and over 200 different types of cancer to choose from, the permutations are enormous.

Previous pre-clinical studies had shown that idronoxil boosts the cancer-killing effects of all commonly used chemotherapies – eg., platinums, taxanes, gemcitabine, doxorubicin etc. Hence our confidence that we could make a range of drug/cancer type combinations work in the clinic.

We were looking for a single combination that would provide unequivocal proof-of-principle, and ‘unequivocal’ meant using a cancer/chemotherapy combination that everyone accepted as the best there was, but ‘the best’ being a poor response rate. Soft tissue sarcomas fitted in that category, with only about 1 in 7 cases responding to chemotherapy in any meaningful way.

Chemotherapy for soft tissue sarcomas typically involves combinations of chemotherapy drugs in an effort to find one that might work, but central to the treatment of soft tissue sarcomas is the chemotherapy drug, doxorubicin.

Doxorubicin (sold under the brand name Adriamycin), is almost 50 years old and remains widely used in the treatment of cancers of the breast, bladder, lung, stomach, ovaries, thyroid, and multiple myeloma, lymphoma and certain leukaemias. And, of course, soft tissue sarcomas.

Potentially showing that Veyonda boosts the response rate to doxorubicin in soft tissue sarcomas provides a rationale for believing it might do the same for doxorubicin in other cancers, and even further on, to providing a basis for believing that Veyonda might do the same for other commonly used chemotherapies in a broad range of cancers. 


CEP-2 is a multi-national study that we are looking to involve both U.S. and Australian sites. It will involve 40 patients with soft tissue sarcomas who have not previously received anthracycline therapy (the class of drug doxorubicin belongs to), effectively making the Veyonda + doxorubicin a first-line treatment. In contrast to the CEP-1 trial which used a maximum Veyonda dose of 800 mg, CEP-2 will uses doses up to 1800 mg.

** Idronoxil is a selective inhibitor of sphingosine-1-phosphate (S1P) in tumour cells. Excess S1P levels drive cancer growth, cancer cell migration, multi-drug resistance mechanisms, and the expulsion of immune cells from tumours.