Montreal firm making world’s only therapy for E. coli complication.
This is Chipotle’s big problem. Now for the solution …
Therapy targets kidney failure
MONTREAL • The most recent outbreak of E. coli in the U. S. sickened 52 people i n nine states who ate at Chipotle restaurants and, although company’s shares have lost nearly US$ 3 billion in market value, for those who contracted the toxic bacteria the outcome could have been much more devastating.
While Chipotle works to reassure customers that its food is safe, Montreal drugmaker, Bellus Health Inc., is the only company in the world developing a treatment for a sometimes fatal complication to the bacteria and will be taking the treatment to the FDA to begin U. S. studies next year.
Normally, E. coli causes diarrhea, abdominal cramping, fever, nausea and vomiting for roughly a week, and in the most recent Chipotle outbreak the people who contracted this bacteria have recovered.
Still, in recent history there have been other strains that had far worse results.
In five to 10 per cent of cases of E. coli produce the Shiga toxin and cause Hemolytic Uremic Syndrome ( sHUS), which affects the kidneys, often leading to the need for dialysis, and in certain cases chronic renal disease and death, primarily in children under five.
That’s where Bellus’ Shigamab antibody therapy would come in, stopping the sHUS when kidney function starts to drop.
In 2011, nearly 4,000 people were infected with E. coli in Germany causing 857 sHUS cases and 48 deaths.
Bellus estimates there are 2,000 to 3,000 annual cases of sHUS in developed countries, with a market opportunity between $150 and $200 million.
“This falls really right in our niche. It’s both a rare disease product, which is an area that we strongly believe in developing drugs for, there’s a very high unmet medical need there and there’s also a good potential for a strong return for our shareholders,” said Bellus CEO Roberto Bellini.
“We decided to take it on because it’s exactly in the area we are in, in terms of our business model.”
Bellus bought the rights to Shigamab ( originally developed by the U. S. Army because the Shiga toxin is a Level 2 biohazard) when it acquired Montreal- based Thallion Pharmaceuticals Inc. in 2013.
Before then there was little progress being made to develop any sort of treatment for this severe complication.
“When there is a drop- off in your kidney function those doctors would then put you on dialysis, hooking you up to a machine to help your kidneys,” Bellini said.
As a small company of nine employees, Bellus doesn’t actually conduct the initial research or the later stage commercialization.
Bellini says the company is focused mostly on the strategic level: clinical trial design, oversight of the projects, business development, finding new opportunities in the rare-disease area and then eventually finding the big pharma partners that will commercialize these products.
“We’re f ocused on developing drugs for rare diseases and don’t want to do research or stuff that’s too early stage because we don’t think that’s a good use of our money and our time,” Bellini said.
Analyst David Martin says there has been a rise in developing treatments for rare diseases because a lot of the low-hanging fruit in medicine has been picked by the big pharma companies who commercialize treatments for the most common illnesses.
“It’s looking at these rare diseases where you can actually make an impact in medicine and the value of what you’re developing,” said Martin.
Also, because a lot of the “blockbuster” drugs that came out in the past three decades have gone off patent it’s created room to pay for drugs for rare, serious diseases.
“There’s more of a need for companies developing drugs for rare, currently untreated, diseases than there is for just about any other area of medicine right now,” Martin said.
To make a significant profit, treatments for rare diseases typically don’t come cheap.
Bellus’ flagship product, Kiacta, which is used for treating another rare kidney disease, will be on its way to gaining regulatory approval by the middle of next year and has been modelled by Zacks Small Cap Research to be priced at an estimated $ 200,000 per year in the U.S.
“I think it’s accepted that it just costs so much to develop drugs these days and if you’ve got 10 million people who take your drug it’s pretty easy to recoup your cost, but when you’ve got 5,000 people who take your drug it’s got to be priced high,” Martin said.
Bellus will be going to the FDA with Shigamab sometime in the first half of 2016, and Bellini is hoping to begin running a study in the patient population by 2017 at the latest. Bellini says that, because Shigamab could qualify for Breakthrough Therapy designation, it could be fasttracked and available to the public by 2019.