Rare cancers can hurt every bit as much as more common cancers, but as it happens, they’re often too rare to present a target market for big pharmaceuticals.
One of these rare cancers is myelogenous leukemia and a drug being put to the test against it is Bisantrene by biotech Race Oncology (ASX: RAC).
Bisantrene was abandoned after dozens of clinical studies during the 1980s and 1990s, not because it didn’t work but because pharmaceutical mergers left the entities wanting large opportunities only.
Race’s CEO Peter Molloy told Stockhead he knows this first-hand. In his career he experienced two mergers, and drugs targeting a smaller field of patients are left behind.
“Each [merger] there’s a severe rationalisation of the research and development portfolio – 2+2 has to be 2, not 4,” he said. “So drugs that only target a modest market opportunity like AML get deprioritised and they receive no further development even though they have good clinical data and a purpose.”
Their loss, our gain
But big pharma’s loss is a small cap biotech’s gain. Often, the big company has done all the hard work for the little ones.
“You have a lot of clinical data so they risk mitigate and it allows you to start from 3rd base instead of the home play,” said Molloy, “because it has already been done and the risk is being removed.”
“The task for us is take the data we have and seek FDA’s approval to do a final clinical trial to demonstrate the drug meets their approval requirements. We filed that in March and it is one of our goals.”
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But an Israeli law that allows drugs to be made available on a named patient basis is also Race’s gain. The company is undertaking a 12-person clinical trial in patients with this cancer.
The trial is being conducted, “to show it [Bisantrene] is a useful drug and this should be used”.
News of the first patient’s treatment sent this stock up 33 per cent last Friday.
How do you find dormant drugs?
But how do you find “dormant drugs” in the first place? Molloy told Stockhead he credited a US doctor who read old medical literature. He was specifically looking for assets that had a useful medical purpose but were overlooked.
One problem was if a past patent had expired. If so, in Molloy’s own words, “it’s a dead duck”. However, he said there was capacity to “renovate the runway” by filing for new patents.
Myelogenous leukemia is the cancer Race Oncology is targeting – a rare bone cancer.
“It [the blood stream] makes too many white blood cell precursors and these cells, a kind of stem calls called blasts, have no activity,” said Molloy.
“They’re supposed to differentiate into white blood cells. They pleliforate and crowd out bone marrow and prevent white blood cells from developing.
“And they enter the blood stream and crowd out red blood cells so you have unusual bleeding, bruising, capacity to get infections and it rapidly progresses.”
While only 20,000 people have it, over 90 per cent die within five years even with treatment.
“It’s so awful, I think its still about 90% plus of people die in five years even with treatment’, he said. ‘As a result, it’s never going to be a billion dollar opportunity, but it could be a $200-$300 million sales opportunity.”