Nine months af­ter launch­ing a $500 mil­lion hunt for a cure for cys­tic fi­bro­sis, the Cys­tic Fi­bro­sis Foun­da­tion said it will use a por­tion of those funds to do some­thing it has nev­er done be­fore: help launch new com­pa­nies.

The CF Foun­da­tion, whose ven­ture phil­an­thropy ef­forts helped fund Ver­tex’s line of pow­er­ful CF drugs, is team­ing with Long­wood Fund to cre­ate a CF in­cu­ba­tor. The in­cu­ba­tor will iden­ti­fy new com­pa­nies with plat­forms or tech­nolo­gies that can be ap­plied in the rare ge­net­ic con­di­tion. The part­ners can then fi­nance ear­ly de­vel­op­ment in ex­change for a com­mit­ment from the com­pa­nies to fo­cus on ap­pli­ca­tions in cys­tic fi­bro­sis.

CF Foun­da­tion has com­mit­ted up to $20 mil­lion to the ef­fort, al­though the com­pa­nies se­lect­ed could al­so re­ceive fund­ing through Long­wood’s tra­di­tion­al ear­ly-stage VC ef­forts.

David Stein­berg

“This is new, so we’ll have to see how it plays out in prac­tice,” Long­wood part­ner David Stein­berg told End­points News. 

The idea for the in­cu­ba­tor emerged last Oc­to­ber at Har­vard Med­ical School, where Long­wood was hold­ing their bian­nu­al meet­ing with non-prof­its and pa­tient groups. At the time, the CF Foun­da­tion was prepar­ing to launch its $500 mil­lion Path to a Cure ini­tia­tive. Trikaf­ta, the third of Ver­tex’s three small-mol­e­cule CF drugs, was about to hit mar­ket, but al­though the new drug meant 90% of CF pa­tients could now be treat­ed, that still left 10% un­treat­ed. Armed with roy­al­ties from Ver­tex, the Path ini­tia­tive would try to back tech­nolo­gies that could treat that last group of pa­tients and ul­ti­mate­ly, per­haps, lead to a cure.

The CF Foun­da­tion had long backed aca­d­e­m­ic work and par­tic­u­lar pro­grams at es­tab­lished com­pa­nies — Ver­tex kept up their ear­ly CF work in part be­cause the foun­da­tion was fund­ing it — but it had nev­er tried to found new ones. But such an ear­ly-stage ap­proach made par­tic­u­lar sense for what the foun­da­tion was now try­ing to ac­com­plish, foun­da­tion CEO Mike Boyle told End­points.

Mike Boyle

Cys­tic fi­bro­sis in­volves a mal­func­tion­ing trans­port pro­tein in the lungs, called CFTR. Ver­tex’s drugs ei­ther help that pro­tein fold in the right way or guide it to the right place. The re­main­ing 10% of pa­tients, though, ei­ther have a pro­tein mis­fold­ed in such a way it doesn’t re­spond to ex­ist­ing small mol­e­cules or they sim­ply don’t have the pro­tein at all.

That meant pa­tients would have to be treat­ed with tech­nolo­gies that can tar­get the gene: gene ther­a­py, gene edit­ing, and RNA drugs, among oth­ers. Boyle said the foun­da­tion is in ear­ly talks with 17 biotechs, most­ly small, with such plat­forms. Many larg­er com­pa­nies with these plat­forms are fo­cused in oth­er ar­eas.

“One of the chal­lenges right now is that there are eas­i­er tar­gets for gene ther­a­py than CF,” Boyle said.

It’s dif­fi­cult to get gene ther­a­py in­to the lungs, where the im­mune re­sponse is unique and cells turn over at a high­er rate than in oth­er parts of the body. But the in­cu­ba­tor could al­low Long­wood and the foun­da­tion to steer com­pa­nies that are just start­ing out to this chal­lenge in ex­change for the fund­ing they need to get off the ground. Those star­tups would al­so have ac­cess to the foun­da­tion’s sci­en­tif­ic ad­vi­sors, their CF lab in Boston and their CF clin­i­cal tri­al net­work.

In ad­di­tion to spe­cif­ic ther­a­pies, the in­cu­ba­tor may al­so in­vest in drug de­liv­ery tech­nol­o­gy, such as ex­o­somes, that could make de­liv­er­ing DNA, RNA or oth­er types of drugs to the lungs.

There is prece­dent for the ap­proach. The Gates Foun­da­tion’s ven­ture arm has long in­vest­ed in ear­ly-stage im­munol­o­gy plat­forms in ex­change for a com­mit­ment from the com­pa­nies to fo­cus part of their work on in­fec­tious dis­ease and not just can­cer or au­toim­mune dis­or­ders.

Boyle said they’re hop­ing to launch com­pa­nies with­in a cou­ple years, but they’re al­so con­tin­u­ing to so­lic­it part­ner­ships with aca­d­e­m­ic cen­ters and more es­tab­lished biotechs. The in­cu­ba­tor’s $20 mil­lion is just a small por­tion of Path’s $500 mil­lion vault.

“This is the be­gin­ning of the process,” Boyle said. “We are open for busi­ness.”

So­cial im­age: Mike Boyle, Cys­tic Fi­bro­sis Foun­da­tion via YouTube

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

President Donald Trump, who seems intent on announcing a COVID-19 vaccine before Election Day, could legally authorize a vaccine over the objections of experts, officials at the FDA and even vaccine manufacturers, who have pledged not to release any vaccine unless it’s proved safe and effective.

In podcasts, public forums, social media and medical journals, a growing number of prominent health leaders say they fear that Trump — who has repeatedly signaled his desire for the swift approval of a vaccine and his displeasure with perceived delays at the FDA — will take matters into his own hands, running roughshod over the usual regulatory process.

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

For the blockbuster potential of Novartis’ Tafinlar and Pfizer’s Braftovi, all the BRAF inhibitors on the market so far only target V600 mutations — which accounts for roughly 50% of patients.

Israeli biotech Novellus now has $57 million to develop a drug that they say can help the other 50% who have everything else.

The Series C will fund a Phase II trial for PLX-8394, a “paradox breaker” that could block RAF without activating MAPK signaling. In a Phase I trial, a patient with a BRAF fusion saw their tumor go away after taking the drug, allowing Novellus to hit the ground running.

A little under two years after a company rebranding, Immune Regulation is taking an even bigger step toward advancing its goals.

Formerly known as Peptinnovate, the British biotech announced a $53.4 million Series B early Monday morning, helping to further advance two clinical programs in rheumatoid arthritis and asthma. Though those are the two initial indications the company is focusing on, CEO Jonathan Rigby told Endpoints News he hopes the candidates can be applied to a broad swath of autoimmune disorders.

William B Crews had been a public affairs specialist at the NIH’s National Institute of Allergy and Infectious Diseases.

That ended today when he informed the agency of his decision to retire, after he was identified as the managing editor at RedState, a prominent Trump loyalist website.

Crews’ RedState duties are performed under the alias streiff. While enjoying the benefits of pseudonymity, he disparaged and worked against NIAID with an incendiary level of rhetoric in the midst of a pandemic.