CF Foun­da­tion, Long­wood team on new in­cu­ba­tor for com­pa­nies with cut­ting-edge CF treat­ments

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

Late Fri­day ap­proval; Trio of biotechs wind down; Stem cell pi­o­neer finds new fron­tier; Biotech icon to re­tire; 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.

I hope your weekend is off to a nice start, wherever you are reading this email. As for me, I’m trying to catch the tail of the Lunar New Year festivities.

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Pfiz­er lays off em­ploy­ees at Cal­i­for­nia and Con­necti­cut sites

Pfizer has laid off employees at its La Jolla, CA, and Groton, CT sites, according to multiple LinkedIn posts from former employees.

The Big Pharma confirmed to Endpoints News it has let go of some employees, but a spokesperson declined to specify how many workers were impacted and the exact locations affected. Earlier this month, the drug developer had confirmed to Endpoints it was sharpening its focus and doing away with some early research on areas such as rare disease, oncology and gene therapies.

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Rodney Rietze, iVexSol CEO

Bris­tol My­ers, Charles Riv­er join Se­ries A fund­ing for iVex­Sol

Massachusetts-based iVexSol has secured funding to the tune of $23.8 million in its latest Series A round. The new investors include Bristol Myers Squibb, manufacturer Charles River Laboratories and Asahi Kasei Medical.

iVexSol is a manufacturer of lentiviral vectors (LVV), used in making gene therapies, and this latest round of fundraising brings its total Series A total over $39 million, which will be used to recruit more employees and bolster its technology.

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Jake Van Naarden, Loxo@Lilly CEO

Lil­ly en­ters ripe BTK field with quick FDA nod in man­tle cell lym­phoma

Eli Lilly has succeeded in its attempt to get the first non-covalent version of Bruton’s tyrosine kinase, or BTK, inhibitors to market, pushing it past rival Merck.

The FDA gave an accelerated nod to Lilly’s daily oral med, to be sold as Jaypirca, for patients with relapsed or refractory mantle cell lymphoma.

The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

Filip Dubovsky, Novavax CMO

No­vavax gets ready to take an­oth­er shot at Covid vac­cine mar­ket with next sea­son plans

While mRNA took center stage at yesterday’s FDA vaccine advisory committee meeting, Novavax announced its plans to deliver an updated protein-based vaccine based on new guidance.

Vaccines and Related Biological Products Advisory Committee (VRBPAC) members voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all future vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

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Steve Harr, Sana Biotechnology CEO

Four years in, Sana gets first FDA go-ahead to bring can­cer treat­ment in­to the clin­ic

Sana Biotechnology is finally headed to the clinic.

Thursday afternoon, the biotech announced the FDA had cleared its application to start a clinical trial for its allogeneic, or “off-the-shelf,” CAR-T cell therapy targeting the antigen CD19 for patients with B-cell lymphomas and leukemias. Sana said its therapy, dubbed SC291, was designed to evade the immune system, which could help cell therapy produce a more durable response in patients, a concern that has followed such off-the-shelf therapies that use donor cells as opposed to a patient’s own cells.

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Ying Huang, Legend CEO

J&J, Leg­end say Carvyk­ti beat stan­dard ther­a­py in ear­li­er-line blood can­cer

J&J and Legend Biotech’s next step in turning their CAR-T therapy Carvykti into a potential megablockbuster has succeeded, the companies said Friday.

Carvykti achieved the primary endpoint — progression-free survival — in an open-label Phase III study testing the treatment in second- to fourth-line multiple myeloma patients. The CARTITUDE-4 trial, for which there aren’t any hard data yet, represents the biggest development for Carvykti’s ability to compete with Bristol Myers Squibb’s Abecma since its approval last February.

Eliot Forster, F-star CEO (Rachel Kiki for Endpoints News)

F-star gets down to the wire with $161M sale to Chi­nese buy­er as na­tion­al se­cu­ri­ty con­cerns linger

With the clock ticking on F-star Therapeutics’ takeover by a Chinese buyer, the companies are still scrambling to remove a hold on the deal from the US government’s Committee on Foreign Investment in the United States.

F-star and invoX Pharma said they are “actively negotiating” with CFIUS “about the terms of a mitigation agreement to address CFIUS’s concerns regarding potential national security risks posed by the transaction.”

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CBER Director Peter Marks (Susan Walsh/AP Images)

FDA ad­vi­so­ry com­mit­tee votes unan­i­mous­ly in fa­vor of bi­va­lent Covid shots re­plac­ing pri­ma­ry se­ries

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all current vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

The vote marks an effort to clear up confusion around varying formulations and dosing schedules for current primary series and booster vaccines, as well as “get closer to the strains that are circulating,” according to committee member Paul Offit, professor of pediatrics at the Children’s Hospital of Philadelphia.