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

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.

(L-R) Dr. Robert Redfield, director of the Centers for Disease Control and Prevention; Dr. Anthony Fauci, director of the National Institute for Allergy and Infectious Diseases; Adm. Brett P. Giroir, Assistant Secretary for Health; and Dr. Stephen M. Hahn, commissioner of the Food and Drugs Administration prepare to testify at a hearing of the House Committee on Energy and Commerce on Capitol Hill on June 23, 2020 in Washington, DC. The committee is investigating the Trump administration's response to the COVID-19 pandemic. (Photo by Kevin Dietsch-Pool/Getty Images)

Trump neuters FDA's at­tempt to strength­en vac­cine guide­lines, call­ing it 'ex­treme­ly po­lit­i­cal'

Stephen Hahn went before a Senate committee Wednesday and declared he’s fighting. “Every one of the decisions we have reached has been made by career FDA scientists based on science and data, not politics,” he exclaimed, adding that “FDA will not permit any pressure from anyone to change that. I will fight for science.”

A few hours later, he was undermined by President Donald Trump when a reporter asked if he was okay with stricter vaccine guidelines that the FDA was said to be cooking up. “That has to be approved by the White House. We may or may not approve it. That sounds like a political move,” he decided.

CEO Markus Warmuth (Monte Rosa)

Monte Rosa rakes in $96M Se­ries B as it pre­pares 'mol­e­c­u­lar glue' plat­form for IND-en­abling stud­ies

About four months after completing an extension to its Series A, Monte Rosa Therapeutics is putting its next foot forward with another heap of cash.

The Boston-based biotech is back with $96 million in Series B financing with a goal to get its lead program ready for IND-enabling studies by the end of the year. Though Monte Rosa is keeping its specific target a secret for now, the company has been researching how to utilize its protein degradation technology in breast cancer and non-small cell lung cancer, among other areas.

Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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Jim Roberts and Brian Finrow (Lumen Bioscience)

With a $4M fed­er­al grant, Lu­men jumps in­to the Covid-19 treat­ment race

It’s been less than a month since Lumen Bioscience announced a $16 million Series B to engineer spirulina — a nutrient-packed super food — for diseases like traveler’s diarrhea, norovirus and C. difficile colitis. And now, the biotech has pulled in another $4 million to do the same for Covid-19.

The approach is quite similar to other gastrointestinal targets the company is pursuing, co-founders and Brian Finrow and Jim Roberts said. The Seattle-based company is working on a camelid antibody cocktail to combat GI infection common among Covid-19 patients. In a study published in the American Journal of Gastroenterology, a majority of Covid-19 patients showed GI and respiratory symptoms, and 25% had only GI symptoms.

FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

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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PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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