Buoyed by block­buster Ver­tex pay­ments, the CF Foun­da­tion seeds new ad­vances with $500M fund

What does a large foun­da­tion do when they re­ceive a check for 10 times their an­nu­al rev­enue?

For five years now, the Cys­tic Fi­bro­sis Foun­da­tion has been a liv­ing ex­per­i­ment to that ques­tion. In 2014, the group saw its for­ay in­to ven­ture phil­an­thropy pay off with a $3.3 bil­lion check from Roy­al­ty Phar­ma for roy­al­ties to Ver­tex’s CF treat­ments. (The Eli Lil­ly heiress-en­dowed Po­et­ry Foun­da­tion has been a small­er long-run­ning ex­per­i­ment in the same ques­tion, for the lit­er­ary phar­ma nerds out there.)

To­day, CFF un­veiled their biggest project since Ver­tex, launch­ing a $500 mil­lion fund called Path to a Cure ded­i­cat­ed to sup­port­ing gene-based ther­a­pies to help the 10% of pa­tients who have been passed over by the new round of treat­ments that have re­made the dis­ease for many pa­tients since they first de­buted in 2012. The fund is in­tend­ed to last through 2025.

Mike Boyle

“This is a way of get­ting treat­ment for every­one,” Mike Boyle, who will be pro­mot­ed to CEO of the foun­da­tion on Jan­u­ary 1, told End­points News. “It’s a whole new tech­nol­o­gy.”

The new wave of CF treat­ments that earned the foun­da­tion its bil­lions – in­clud­ing the three-drug com­bo Trikaf­ta that the FDA ap­proved last week – work by mod­u­lat­ing the de­fec­tive CFTR pro­tein that caus­es the dis­ease, help­ing it get to the right lo­ca­tion and func­tion prop­er­ly there. But 10% of pa­tients ei­ther have pro­teins fold­ed in such a way they don’t re­spond to the mod­u­la­tors or they lack the pro­tein al­to­geth­er.

For those pa­tients, the foun­da­tion is look­ing to fund po­ten­tial gene edit­ing or gene trans­fer so­lu­tions that would fix cys­tic fi­bro­sis’s un­der­ly­ing ge­net­ic cause. CF is caused by mu­ta­tions in both copies of the CT­FR gene. An edit­ing so­lu­tion would fix the spe­cif­ic mis­spelled nu­cleotides, while a trans­fer would in­sert a com­plete func­tion­ing copy of the gene.

CFF will find them­selves with far more funds than they used to fu­el the rise of the pro­tein mod­u­la­tors. They gave Ver­tex $150 mil­lion over 12 years. This new fund will give out more than 3 times that in half the time, and the group said they’re ready to in­vest far more if a par­tic­u­lar­ly promis­ing so­lu­tion gains trac­tion.

Boyle said they would and have fund­ed pro­pos­als from all types of drug de­vel­op­ers. But he’s al­so hop­ing the large dol­lar fig­ure, along with the promise of the foun­da­tion’s ex­perts and clin­i­cal net­work, would at­tract biotechs who have had suc­cess in oth­er ar­eas to take a chance on CF.  CF is a more dif­fi­cult tar­get than mus­cles or oth­er tis­sues, he said, be­cause the im­mune sys­tem is stronger (to guard against the con­stant flow of air­borne virus­es) and cel­lu­lar turnover is high­er.

“We’ve been do­ing this [fund­ing] all along but this was an ef­fort to grab at­ten­tion,” Boyle said. “I want the world’s best re­search fo­cused on CF.”

In ad­di­tion to the Path fund, the foun­da­tion has re­cent­ly poured mil­lions in­to re­search on an­ti-in­flam­ma­to­ries and an­ti-in­fec­tives to curb some of the side ef­fects and risks CF pa­tients face, along with re­search for co-mor­bidi­ties now emerg­ing as pa­tients live longer.

But if the new wave of re­search is ef­fec­tive – and far more dol­lars than this have been pushed in­to ther­a­pies that have dead end­ed – it could have a greater im­pact than any of the foun­da­tion’s oth­er ven­tures. Al­though the gene ther­a­pies are first tar­get­ed at the un­treat­able forms of the dis­ease, if suc­cess­ful they hold the po­ten­tial to fix the dis­ease en­tire­ly.

“Over time every­one would ben­e­fit — which is an im­por­tant thing for com­pa­nies as they think about their mar­ket,” Boyle said. “It would have to be ef­fec­tive, though, and that will take time.”

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.

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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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