Four years af­ter it im­plod­ed at Sanofi, John Hood is res­ur­rect­ing the myelofi­bro­sis drug fe­dra­tinib

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.”

Kevin John­son

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.

Lessons for biotech and phar­ma from a doc­tor who chased his own cure

After being struck by a rare disease as a healthy third year medical student, David Fajgenbaum began an arduous journey chasing his own cure. Amidst the hustle of this year’s JP Morgan conference, the digital trials platform Medable partnered with Endpoints Studio to share Dr. Fajgenbaum’s story with the drug development industry.

What follows is an edited transcript of the conversation between Medable CEO Dr. Michelle Longmire and Dr. Fajgenbaum, and it is full of lessons for biotech executives charged with bringing the next generation of medicines to patients.

Kathy High (file photo)

Gene ther­a­py pi­o­neer Kathy High has left Spark af­ter com­plet­ing $4.3B union with Roche

Kathy High dedicated the past seven years of her life shepherding experimental gene therapies she’s developed at Children’s Hospital of Philadelphia toward the market as president and head of R&D at Spark Therapeutics. Now that the biotech startup is fully absorbed into Roche — with an FDA approval, a $4.3 billion buyout and a promising hemophilia program to boast — she’s ready to move on.

Roche confirmed her departure with Endpoints News and noted “she will take some well-deserved time off and then will begin a new chapter in a sabbatical at a university.”

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Deborah Dunsire

The fourth CGRP mi­graine drug is here. Time for Lund­beck to prove it's worth $2B

They may be late, but Lundbeck is now officially in the game for preventing migraine with CGRP drugs.

The FDA has OK’d eptinezumab, the prize in Lundbeck’s $2 billion acquisition of Alder. Like rival offerings from Amgen/Novartis, Eli Lilly and Teva, the antibody blocks the calcitonin gene-related peptide, which is believed to dilate blood vessels in the brain and cause pain.

It will now be sold as Vyepti. The company has yet to announce a price. Amgen and Novartis had set the wholesale acquisition cost of their pioneering Aimovig at $6,900 for a year’s supply before raising it slightly this year; Lilly and Teva had followed suit with Emgality and Ajovy.

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Tal Zaks (Moderna via YouTube)

For two decades, a new vac­cine tech­nol­o­gy has been slow­ly ap­proach­ing prime time. Now, can it stop a pan­dem­ic?

Two months before the outbreak, Moderna CMO Tal Zaks traveled from Cambridge, MA to Washington DC to meet with Anthony Fauci and the leaders of the National Institutes of Health.

For two years, Moderna had worked closely with NIH researchers to build a new kind of vaccine for MERS, one of the deadliest new viruses to emerge in the 21st century. The program was one test for a new technology designed to be faster, cheaper and more precise than the ways vaccines had been made for over a century. They had gathered evidence the technology could work in principle, and Fauci, the longtime head of the National Institute of Allergy and Infectious Diseases and a longtime advocate for better epidemic preparedness, wanted to see if it, along with a couple of other approaches, could work in a worst-case scenario: A pandemic.

“[We were] trying to find a test case for how to demonstrate if our technology could rapidly prepare,” Zaks told Endpoints News.

Zaks and Fauci, of course, wouldn’t have to wait to develop a new test. By year’s end, an outbreak in China would short circuit the need for one and throw them into 24/7 work on a real-world emergency. They also weren’t the only ones with new technology who saw a chance to help in a crisis.

An ocean away, Lidia Oostvogels was still on vacation and relaxing at her mother’s house in Belgium when her Facebook started changing. It was days after Christmas and on most people’s feeds, the news that China had reported a novel virus to the World Health Organization blurred into the stream of holiday sweaters and fir trees. But on Oostvogels’s feed, full of vaccine researchers and virus experts, speculation boiled: There was a virus in China, something contained to the country, but “exotic,” “weird,” and maybe having to do with animals. Maybe a coronavirus.

Lidia Oostvogels

“I was immediately thinking like, ‘Hey, this is something that if needed, we can play a role,'” Oostvogels told Endpoints.

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Christos Kyratsous (via LinkedIn)

He built a MERS treat­ment in 6 months and then the best Ebo­la drug. Now Chris­tos Kyrat­sous turns his sights on Covid-19

TARRYTOWN, NY — In 2015, as the Ebola epidemic raged through swaths of West Africa, Kristen Pascal’s roommates sat her down on their couch and staged an intervention.

“Are you sure this is what you want to be doing with your life?” she recalls them asking her.

Special report

Pascal, a research associate for Regeneron, had been coming home at 2 am and leaving at 6 am. At one point, she didn’t see her roommate for a week. For months, that was life in Christos Kyratsous’ lab as the pair led a company-wide race to develop the first drug that could effectively treat Ebola before the outbreak ended. For Pascal, that was worth it.

“I’m ok, I don’t have Ebola,” Pascal told them. “I see that death toll rising and I can’t not do something about it.”

Last August, Regeneron learned they had succeeded: In a large trial across West Africa, their drug, REGN-EB3, was vastly more effective than the standard treatments. It was surprise news for the company, coming just 10 months into a trial they thought would take several years and a major victory in the global fight against a deadly virus that killed over 2,000 in 2019 and can carry a mortality rate of up to 90%.

For Kyratsous and Pascal, though, it brought only fleeting reprieve. Just four months after the NIH informed them REGN-EB3 worked, Kyratsous was back in his office reading the New York Times for updates on a new outbreak on another continent, and wondering alongside Pascal and senior management whether it was time to pull the trigger again.

In late January, as the death toll swelled and the first confirmed cases outside China broke double digits, they made a decision. Soon they were back on the phone with the multiple government agencies and their coronavirus partners at the University of Maryland’s Level 3 bio lab. The question was simple: Can Kyratsous and his team use a process honed over two previous outbreaks, and create a treatment before the newest epidemic ends? Or worse, if, as world health experts fear, it doesn’t vanish but becomes a recurrent virus like the flu?

“Christos likes things immediately,” Matt Frieman, Regeneron’s coronavirus collaborator at the University of Maryland, told Endpoints. “That’s what makes us good collaborators: We push each other to develop things faster and faster.”

Kristen Pascal (Regeneron)

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The first time Regeneron tried to respond to a global outbreak, it was something of a systems test, Kyratsous explains from his office at Regeneron’s Tarrytown headquarters. Kyratsous, newly promoted, has crammed it with photos of his family, sketches of viral vectors and a shark he drew for his 3-year-old son. He speaks rapidly – an idiosyncrasy his press person says has only been aggravated this afternoon by the contents of his “Regeneron Infectious Diseases”-minted espresso glass – and he gesticulates with similar fluidity, tumbling through antibodies, MERS, the novel coronavirus, Ebola-infected monkeys.

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Tim Mayleben (file photo)

Es­pe­ri­on's goldilocks cho­les­terol fight­er wins FDA ap­proval — will its 'tra­di­tion­al' pric­ing ap­proach spur adop­tion?

It’s more effective than decades-old statins but not as good as the injectable PCSK9 — the goldilocks treatment for cholesterol-lowering, bempedoic acid, has secured FDA approval.

Its maker, Esperion Therapeutics, is betting that their pricing strategy — a planned list price of between $10 to $11 a day — will help it skirt the pushback the PCSK9 cholesterol fighters, Repatha and Praluent, got from payers for their high sticker prices.

The sky-high expectations for the pair of PCSK9 drugs that were first approved in 2015 quickly simmered — and despite a 60% price cut, coupled with data showing the therapies also significantly cut cardiovascular risk, sales have not really perked up.

Esperion is convinced that by virtue of being a cheaper oral therapy, bempedoic acid will hit that sweet spot in terms of adoption.

“We’re kind of like the old comfortable shoe,” Esperion’s chief commercial officer Mark Glickman remarked in an interview with Endpoints News ahead of the decision date. “It’s an oral product, once-daily and nontitratable — these are things that just resonate so true with patients and physicians and I think we’ve kind of forgotten about that.”

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James Collins, Broad Institute via Youtube

UP­DAT­ED: A space odyssey for new an­tibi­otics: MIT's ma­chine learn­ing ap­proach

Drug development is complex, expensive and comes with lousy odds of success — but in most cases, if you make it across the finish line brandishing a product with an edge (and play your cards right) it can be a lucrative endeavor.

As it stands, the antibiotic market is cursed — it harbors the stink of multiple bankruptcies, a dearth of innovation, and is consequently barely whetting the voracious appetites of big pharma or venture capitalists. Enter artificial intelligence — the biopharma industry’s cure-all for the pesky process of making a therapeutic, including data mining, drug discovery, optimal drug delivery, and addressable patient population.

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Gilead los­es two more patent chal­lenges on HIV pill, set­ting up court­room fight in Delaware

Gilead sustained two more losses in their efforts to rid themselves of an activist-backed patent lawsuit from the US government over a best-selling HIV pill.

Urged on by activists seeking to divert a portion of Gilead’s revenue to clinics and prevention programs, the Department of Health and Human Services made a claim to some of the patents for the best-selling HIV prevention drug, Truvada, also known as PrEP. Gilead responded by arguing in court that HHS’s patents were invalid.

Today, the US Patent and Trademark Office ruled that Gilead was likely to lose the last two of those challenges as well. The USPTO ruled against Gilead on the first two patents earlier this month.

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Jim Scholefield via PR Newswire

Mer­ck los­es its chief dig­i­tal of­fi­cer, spot­light­ing tal­ent hunt for the hottest ti­tle in Big Phar­ma

Over the last few years we’ve seen the chief digital officer title become one of the hottest commodities in Big Pharma as global organizations hunt the best talent to sharpen the cutting edge of their tech platforms.

But Merck just discovered how hard it may be to keep them focused on pharma.

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