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.”

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

So — that pig-to-hu­man trans­plant; Po­ten­tial di­a­betes cure reach­es pa­tient; Ac­cused MIT sci­en­tist lash­es back; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

We’re incredibly excited to welcome Beth Bulik, seasoned pharma marketing reporter, to the team. You can find much of her work in our new Marketing channel — and in her weekly newsletter, Endpoints PharmaRx, which will launch in early November. Add it to your subscriptions here.

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NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

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How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data are messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data are exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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David Livingston (Credit: Michael Sazel for CeMM)

Renowned Dana-Far­ber sci­en­tist, men­tor and bio­phar­ma ad­vi­sor David Liv­ingston has died

David Livingston, the Dana-Farber/Harvard Med scientist who helped shine a light on some of the key molecular drivers of breast and ovarian cancer, died unexpectedly last Sunday.

One of the senior leaders at Dana-Farber during his nearly half century of work there, Livingston was credited with shedding light on the genes that regulate cell growth, with insights into inherited BRCA1 and BRCA2 mutations that helped lay the scientific foundation for targeted therapies and earlier detection that have transformed the field.

No­vo CEO Lars Fruer­gaard Jør­gensen on R&D risk, the deal strat­e­gy and tar­gets for gen­der di­ver­si­ty

 

I kicked off our European R&D summit last week with a conversation involving Novo Nordisk CEO Lars Fruergaard Jørgensen. Novo is aiming to launch a new era of obesity management with a new approval for semaglutide. And Jørgensen had a lot to say about what comes next in R&D, how they manage risk and gender diversity targets at the trendsetting European pharma giant.

John Carroll: I’m here with Lars Jørgensen, the CEO of Novo Nordisk. Lars, it’s been a really interesting year so far with Novo Nordisk, right? You’ve projected a new era of growing sales. You’ve been able to expand on the GLP-1 franchise that was already well established in diabetes now going into obesity. And I think a tremendous number of people are really interested in how that’s working out. You have forecast a growing amount of sales. We don’t know specifically how that might play out. I know a lot of the analysts have different ideas, how those numbers might play out, but that we are in fact embarking on a new era for Novo Nordisk in terms of what the company’s capable of doing and what it’s able to do and what it wants to do. And I wanted to start off by asking you about obesity in particular. Semaglutide has been approved in the United States for obesity. It’s an area of R&D that’s been very troubled for decades. There have been weight loss drugs that have come along. They’ve attracted a lot of attention, but they haven’t actually ever gained traction in the market. My first question is what’s different this time about obesity? What is different about this drug and why do you expect it to work now whereas previous drugs haven’t?

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Bris­tol My­ers pledges to sell its Ac­celeron shares as ac­tivist in­vestors cir­cle Mer­ck­'s $11.5B buy­out — re­port

Just as Avoro Capital’s campaign to derail Merck’s proposed $11.5 billion buyout of Acceleron gains steam, Bristol Myers Squibb is leaning in with some hefty counterweight.

The pharma giant is planning to tender its Acceleron shares, Bloomberg reported, which add up to a sizable 11.5% stake. Based on the offer price, the sale would net Bristol Myers around $1.3 billion.

To complete its deal, Merck needs a majority of shareholders to agree to sell their shares.

Hedge fund jumps in with Avoro ac­tivists in an at­tempt to de­rail Mer­ck­'s $11B Ac­celeron buy­out

Avoro Capital, which made its bones blowing up the Seagen-Immunomedics deal and then selling the smaller biotech for $21 billion, is getting an assist in its quest to derail Merck’s $11 billion buyout of Acceleron $XLRN.

Wednesday morning one of Acceleron’s biggest investors joined the opposition. Darwin Global Management, a hedge fund which owns about 4% of Acceleron, blasted the Merck deal, saying the Big Pharma is getting the company for billions less than what it’s worth. Earlier, Holocene Advisers, reportedly a top-20 investor in Acceleron, said it would not tender its stock after criticizing the $180-per-share deal.

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