Fedratinib was one of the biggest clinical drug disasters that occurred at Sanofi under the tumultuous reign of CEO Chris Viehbacher. But after imploding with spectacular effect right at the threshold of a new drug application four years ago, one of the drug’s co-inventors — a scientist named John Hood — has patiently picked up all the shattered pieces and put it together into a new company that just scored a $22.5 million venture round.
Now Hood’s little biotech is going after that FDA/EMA approval that eluded the pharma giant.
If he’s right, Hood has scored one of the biggest biotech coups of the year. But let’s start this story with a little background.
Seven years ago, Sanofi scooped up a biotech called TargeGen in a $635 million deal, bagging a mid-stage JAK2 myelofibrosis drug — then dubbed TG 101348 — that had gathered promising early-stage human data. Hood, the former research chief at TargeGen and a co-inventor of the drug, moved on to co-found a biotech unicorn company called Samumed, focused on regenerative medicine with Hood taking the CSO job.
In 2013, the FDA dropped a clinical hold on fedratinib after a few patients began to develop Wernicke’s encephalopathy — a dangerous neurological condition tied to vitamin B deficiency. And Sanofi’s top team quickly put a bullet in it, scrapping a program that included seven ongoing clinical trials. The pharma giant shoved the mortally wounded program on the shelf, and that’s where it sat until early 2016, when Hood decided to start his own company, get the rights back and pursue transatlantic approvals.
To hear Hood tell it, after the clinical trials ended, the patients involved were shifted to standard of care and began to relapse.
“That’s when physicians and patients began to reach out to me,” Hood says, looking for some way to get the drug under compassionate use rules. That inspired him to go for it.
I happened to pick up word of what was happening at the time, and Hood and I started exchanging occasional messages around his progress. Wait for it, he would write, it was gradually coming together.
Sanofi gave up the drug for equity in Hood’s company, Impact Biomedicines in San Diego, which now has a lean-and-mean team of six. The package includes positive Phase III results (with a p-value of 0.0001 says a proud Hood) data from 877 patients in 18 studies.
“There was no out-of-pocket,” Hood tells me. Sanofi took an equity stake in the biotech for its share.
The FDA came through in August, dropping the clinical hold after Hood made the case that the side effects that so alarmed investigators and regulators in 2013 could be attributed to a small number of cases of thiamine depletion linked to the high metabolic rate of cancer patients and malnourishment.
Says Hood: “It was easy enough to mitigate.”
Hood’s backed by a globe-trotting team at Medicxi, where Kevin Johnson — a buoyant Cambridge grad and scientist who had been head of research at the legendary Cambridge Antibody Technology — put together the financing round to fund what comes next.
That next part still has to be worked out, the CEO tells me. I suggested that if anything, regulators have embraced speeded up development programs in oncology, which could put him right back at the FDA’s door, knocking for market entry.
After spending more than 18 months putting this all together, though, Hood is still taking it one step at a time. He’ll know more about how fast he can move after meeting with regulators to test their appetite for speed.
I asked Johnson what his first thoughts were when he and Hood talked it over.
My initial reaction to the story was that I couldn’t grasp the thought process that led to it being put on clinical hold and therefore not reaching the market. We pieced that all together at a later stage, and while it looks like a strange decision in retrospect, at the time, with those specific market conditions, it would have been a brave decision to keep going. Of course it’s all different now.On John himself, he really does think like that and I love it! It’s my experience that the top biotech and pharma entrepreneurs are driven by a need to do good, rather than just make money. In the end I guess it’s a belief system- do the right things for the right reasons and the money will follow as a consequence. It does tend to work out that way. He’s also what I call a handshake guy; you shake hands on something and that’s it. He could have played us but he didn’t. That too is consistent with his phenotype.
In most cases like this, Hood and his tiny band of staffers would be setting himself up to do a deal on the drug. But Hood isn’t jumping that far ahead either. Right now, he says, he’s working on the kind of financial package that would “allow us to take it the whole way.”
That may sound a bit far-fetched, but the whole story up to this point was improbable.
Hood’s just getting started again.
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