UM171, the life-saving molecule developed at Université de Montréal that boosts the effectiveness of stem-cell transplants in cancer patients, is now officially on the commercial market.
Today the European Medicines Agency (EMA) approved UM171's retail formulation, Zemcelpro, as the first and only authorized treatment for adults with blood cancer getting transplants when no suitable donor can be found.
Zemcelpro contains stem cells from a donor's umbilical cord blood, some of which have been grown and multiplied using the UM171 molecule, otherwise known as dorocubicel.
By increasing the number of cells, UM171 makes the stem cells from a small unit of cord blood more effective.
"Now that we have official approval, a lot of things will happen," said Dr. Guy Sauvageau, the UdeM hematologist who developed and patented UM171 with UdeM medical chemist Dr. Anne Marinier via the biotech start-up ExCellThera Inc.
"First, ExCellThera can begin to commercialize the product in jurisdictions across Europe; second, negotiations can begin in each country on the price they or their country's insurance companies will decide to pay for it," said Sauvageau.
"In short, as of Aug. 26, a doctor grafting a patient anywhere in Europe can call us up and say 'We want the product,' and ExCellThera can say 'Yes, you can have it, on early access, subject to the price negotiated by the country you're in."
France comes first
One exception is France: that country will immediately pay the price ExCellThera sets. However, if within 12 months a lower price is negotiated, the buyers can retroactively demand to be reimbursed the difference.
"One advantage of this for us is that we get Zemcelpro into France first, in 2025, rather than wait until 2026 when the final price gets worked out," said Sauvageau, ExCellThera's chair and scientific director.
Canada is next, he added.
"Normally, Health Canada waits until Phase 3 trials have begun before fast-tracking a treatment for approval," said Sauvageau. "But Canada does have an early-access program like Europe's, so approval there bodes well for here."
In fact, 60 days after being informed of pending European approval, Canada can grant early access. ExCellThera gave that notice in early August, so as soon as early October any hospital or foundation or person can pay to get the treatment.
The U.S. is also interested, Sauvageau said.
"We're meeting with the Food and Drug Administration (FDA) in mid-September to decide if we're okay to apply for approval, after which would come commercialization and negotiation of a price."
Discovered at UdeM's Institute for Research in Immunology and Cancer (IRIC), UM171 was financially supported by IRICoR, a non-profit organization focused on commercializing research.
120 patients treated
Over the last eight years, UM171 has been used experimentally to treat 80 patients in Canada and 40 in the U.S. and Europe. Of the 120, about 7 out of every 10 were cancer-free after two years and so can be said to be cured.
Initially, when they were recruited for the treatment, each was only given a 40-per-cent chance of recovery from their disease via the treatment. Simply put, UM171 exceeded expectations.
"It's almost double the success we anticipated, which is incredible," said Sauvageau.
One example: François Lucas Alzate, a young Montrealer. Diagnosed with acute myeloid leukemia in 2014, when he was only 23, and successfully grafted two years later at Maisonneuve-Rosemont Hospital, he remains disease-free.
Alzate's testimonial can be viewed on the Hema-Québec website.
Results like his - and the capacity of governments and insurers to pay for them to be extended to more patients - are what will ultimately determine the commercial value of UM171.
For EMA approval, it took 50 experts one year to plow through 20,000 pages of supporting evidence to discuss the merits of Zemcelpro therapy and arrive at a positive decision.
Next comes the commercial part: figuring out its value on the open market. "Approval comes, and governments and insurers say 'Wait a minute, do I have the budget for this and how much am I going to pay for it?'" said Sauvageau.
'Tremendous pride'
"Our molecule is not a drug strictly speaking - it's part of a graft, a molecule that modifies the cell graft and makes it much more effective," said Marinier, one of ExCellThera's six administrators.
"I feel tremendous personal pride that we've come this far. There aren't many in the pharmaceutical industry who can say they helped bring a drug successfully to market."
She added: "I've been at UdeM 17 years and this is the first time, to the best of my knowledge, that a molecule is approved commercially. It's a big moment not just for us and our teams, but for the university, too."
And there's more good news to come.
"We can certainly say that, thanks to UM171 we've found a new mechanism of action that can be spun off into new applications, especially anti-aging ones, and that can be taken orally," said Sauvageau.
To that end, in late 2020 the duo launched a new biotech start-up called RejuvenRX.
"That's what we're working on now, just as scientists at major universities such as Harvard University and the University of Oxford are doing; UM171 is their model for pursuing new research," said Marinier.
Anything else on the horizon?
"It took two years," said Sauvageau, "but we finally got the FDA's go-ahead to test-trial a much wider application of UM171: on people with high-risk leukemia, who represent 35 per cent of all stem-cell grafts, close to 5,000 a year in the U.S."
To start that Phase 3 trial, $30 million must first be raised.
"Calling all investors!" Sauvageau said with a laugh.
