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.”

Com­mu­ni­cat­ing the val­ue of pre­ci­sion med­i­cine

By Natasha Cowan, Content Marketing Manager at Blue Latitude Health.
Many stakeholders are confused by novel precision medicines, including patients and healthcare professionals. So, how can industry help them to navigate this complexity?

Precision medicine represents a new paradigm in healthcare. It embodies the shift from treating many patients with the same therapy, to having the tools to identify the best treatment for every patient.

Spe­cial re­port: Twen­ty ex­tra­or­di­nary women in bio­phar­ma R&D who worked their way to the top

What differentiates a woman leader in biopharma R&D from a man?

Not much, except there are fewer of them in senior posts. Data suggest women are not more risk-averse, family-oriented or less confident than their male counterparts — indeed the differences between the two sexes are negligible. But a glance at the top R&D positions in Big Pharma leaves little doubt that upward migration in the executive ranks of biopharma R&D is tough.

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Ed­i­tas and Cel­gene sub Juno are tack­ling hottest im­munother­a­py cells

As the first CRISPR-edited cancer patients watch their treatments unfold, one of the first CRISPR companies is rejigging a major oncology deal.

Editas Medicine is amending its long-running collaboration with Celgene and their subsidiary Juno Therapeutics. The new deal will expand the focus of their work to cover a subset of immune cells that have become an increasingly hot target for immunotherapy: gamma-delta cells.

FDA Vas­cepa re­view spot­lights new safe­ty sig­nals, pos­si­ble min­er­al oil spoil­er as Amarin hunts a block­buster ap­proval

An in-house FDA review of Amarin’s Vascepa raises a set of hurdles the biotech will have to clear if the biotech expects to get the long-awaited FDA approval that could set it on a path to superstar status. But it appears that Amarin has survived another potential setback without introducing a major new threat to its prospects.
The stakes don’t get much higher, with analysts saying a win this week for Amarin could lead to billions in new sales — provided the agency stamps it with an OK. And investors liked what they say in the FDA review this morning, bumping the stock $AMRN 17%.
The insider take at the agency includes a note on two new safety signals seen in the big cardio outcomes study of the omega-3 fatty acid drug that shocked many analysts with a solid set of efficacy data. There’s a key concern over whether the use of mineral oil in the placebo skewed LDL levels in such a way that tilted the data in Amarin’s favor.
The FDA overview was written by John Sharretts, the acting deputy director in the Division of Metabolism and Endocrinology Products. 
On the safety side, the internal review focused on a 3.1% versus 2.1% rate of adjudicated events of atrial fibrillation or atrial flutter requiring hospitalization. But they also say a-fib shouldn’t confound the benefit-safety of the drug — given the improvement on MACE — or prevent its use. And then there was also a higher rate of bleeding events in the drug arm.

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Prakash Raman. Flagship

Flag­ship woos No­var­tis top deal­mak­er Prakash Ra­man in move to get the BD ball rolling ear­ly

Flagship Pioneering likes to be ahead of its times — so far ahead, perhaps, that it is often challenging to find partners for their startups while the scientific scaffolding is underway. But Prakash Raman is here to change that.

Raman, who most recently headed up business development at the Novartis Institutes for BioMedical Research, became Flagship’s first chief business development officer two weeks ago. By acting as a “central resource” for the 100 companies in the venture fund’s portfolio, he hopes to help entrepreneurs and management teams strategize about dealmaking to capture value beyond the near-term validation of their platform technologies, Raman told Endpoints News.

FDA puts Sol­id Bio’s lead gene ther­a­py pro­gram on hold — again — af­ter an­oth­er pa­tient is hurt by SGT-001

Solid Biosciences continues to be plagued by safety issues.

Close to 18 months after the gene therapy biotech was able to quickly shed an FDA hold on their lead Duchenne muscular dystrophy program for SGT-001, regulators have stepped back in to force another halt after another patient was hit hard by a set of serious adverse events remarkably similar to the first set.

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Bill Haney, Skyhawk

Cel­gene ex­ecs shell out $92M cash for a pair of R&D deals that will fit per­fect­ly in their new home at Bris­tol-My­ers

With Bristol-Myers Squibb’s Celgene buyout all but complete, the BD teams are working in perfect synchrony now. The Celgene side is going back to Skyhawk, a darling of the crowd that set out to drug RNA, and they’re adding a suite of new programs that mesh perfectly with the new regime in charge.

Celgene is shelling out $80 million in a cash upfront to add oncology, immuno-oncology and autoimmune diseases to the initial roundup of neurological targets mapped early in Skyhawk’s existence.

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Reata's bar­dox­olone of­fers promise in pa­tients with rare kid­ney dis­or­der

After surprising Wall Street with positive data on its drug, omaveloxolone, in patients suffering from a notoriously hard-to-treat degenerative neuromuscular disorder last month, Reata Pharma on Monday unveiled pivotal results from a trial testing another drug, bardoxolone, in patients with a rare, genetic form of chronic kidney disease for which there exist no approved therapies.

Bardoxolone, like Reata’s other lead drug — omaveloxolone — is a small molecule engineered to bind to a gene called Keap1 to enhance the activity of the protein Nrf2 in order to defuse inflammation.

Kad­mon wax­es rhap­sod­ic on cGVHD re­sults as race with Jakafi heats up

A year ago, Kadmon piqued cautious interest and sent its stock up 20% when it announced positive results from a tiny proof-of-concept study on a new, chronic graft-versus-host-disease treatment. Now interim results are out on the pivotal, and not just the biotech’s executives are gushing about it.

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