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.”

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

FDA chief Stephen Hahn on Capitol Hill earlier this week (Getty Images)

As FDA’s work­load buck­les un­der the strain, Trump again ac­cus­es the agency of a po­lit­i­cal hit job

Peter Marks appeared before a virtual SVB Leerink audience yesterday and said that his staff at FDA’s CBER is on the verge of working around the clock. Manufacturing inspections, policy work and sponsor communications have all been pushed down the to-do list so that they can be responsive to Covid-related interactions. And the agency’s objective right now? “To save as many lives as we can,” Marks said, likening the mortality on the current outbreak as equivalent to “a nuclear bomb on a small city.”

Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

Play-by-play of Gilead­'s $21B Im­munomedics buy­out de­tails a fren­zied push — and mints a new biotech bil­lion­aire

Immunomedics had not really been looking for a buyout when the year began. Excited by its BLA for Trodelvy, submitted to the FDA in late 2019, executive chairman Behzad Aghazadeh started off looking for potential licensing deals and zeroed in on four potential partners, including Gilead, following January’s JP Morgan Healthcare Conference in San Francisco. Such talks advanced throughout the year, with discussions advancing to the second round in mid-August.

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David Berry (Flagship)

Flag­ship's next big tech­no­log­i­cal bet? The cloud

Earlier this month, Flagship announced their big bet on the software half the industry is talking about, launching the AI and machine learning startup. Now, they and a couple other investors are gambling $100 million on a software that much of the public generally thinks of as a cool, IT afterthought: cloud computing.

The idea, says founder and Flagship partner David Berry, is one of scale: The sheer magnitude of biological data that you can store on cloud technology is unprecedented. And that size, when leveraged properly, can allow you to ask questions and form insights that are similarly unprecedented.

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Patrick Enright, Longitude co-founder (Longitude)

As its biotechs hit the pan­dem­ic ex­it, Lon­gi­tude rais­es $585M for new neu­ro, can­cer, ag­ing and or­phan-fo­cused fund

The years have been kind to Longitude Capital. This year, too.

A 2006 spinout of Pequot Capital, its founders started their new firm just four years before the parent company would go under amid insider trading allegations. Their first life sciences fund raised $325 million amid the financial crisis, they added a second for $385 million and then in, 2016, a third for $525 million. In the last few months, the pandemic biotech IPO boom netted several high-value exits from those funds, as Checkmate, Vaxcyte, Inozyme and Poseida all went public.

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The win­dow is wide open as four more biotechs join the go-go IPO class of 2020

It’s another day of hauling cash in the biopharma world as four more IPOs priced Friday and a fifth filed its initial paperwork.

The biggest offering comes from PMV Pharma, an oncology biotech focusing on p53 mutations, which raised $211.8 million after pricing shares at $18 apiece. Prelude Therapeutics, developing PRMT5 inhibitors for rare cancers, was next with a $158 million raise, pricing shares at $19 each. Graybug Vision raised $90 million after pricing at $16 per share for its wet AMD candidates, and breast cancer biotech Greenwich Lifesciences brought up the rear with a small, $7 million raise after pricing shares at $5.75.

J&J of­fers PhI/IIa da­ta show­ing its sin­gle-dose vac­cine can stir up suf­fi­cient im­mune re­sponse

Days after J&J dosed the first participants of its Phase III ENSEMBLE trial, the pharma giant has detailed the early-stage data that gave them confidence in a single-dose regimen.

Testing two dose levels either as a single dose or in a two-dose schedule spaced by 56 days in, the scientists from Janssen, the J&J subsidiary developing its vaccine, reported that the low dose induced a similar immune response as the high dose. The interim Phase I/IIa results were posted in a preprint on medRxiv.

President Donald Trump reacts after signing an executive order following his remarks on his healthcare policies yesterday in Charlotte, North Carolina (Getty Images)

Op-ed: Will phar­ma re­al­ly pay for Trump’s lat­est law­less promise to 33 mil­lion Medicare ben­e­fi­cia­ries? Not like­ly

Sitting atop the executive branch, President Donald Trump is the ultimate authority at the FDA. He can fast track any vaccine to approval himself. If it came to that, of course.

What he can’t do is unilaterally order the legislative branch to loosen the Treasury’s coffers for $6.6 billion. Nor can he command pharmaceutical companies to pay for $200 vouchers sent to 33 million Medicare beneficiaries for prescription drugs before the election.

Pal­la­dio bags $20 mil­lion Se­ries B to top­ple a prob­lem­at­ic kid­ney dis­ease drug

Palladio Biosciences just took one step further in its quest to topple Otsuka’s Samsca with its own — it hopes safer — autosomal dominant polycystic kidney disease (ADPKD) drug.

The Pennsylvania-based biotech announced a $20 million Series B on Friday, which will fund a 10-person Phase III trial of its vasopressin V2 receptor agonist, lixivaptan. CEO Alex Martin expects to read out data in the first half of next year, then launch straight into a larger pivotal Phase III study with about 1,200 participants.