Paula Cannon (Credit: Richard Carrasco)

Re­searchers teamed up to de­vel­op a 'three in one' HIV treat­ment — and the NIH is throw­ing in $14.6M

The NIH is pitch­ing $14.6 mil­lion in­to a “three for one” HIV re­search pro­gram led by USC and the Fred Hutchin­son Can­cer Re­search Cen­ter that aims to strike the need for dai­ly med­ica­tion — or even achieve a “home run” cure.

The five-year grant will back pre­clin­i­cal stud­ies that com­bine gene edit­ing with tech­nol­o­gy to im­prove bone mar­row trans­plants. The po­ten­tial ther­a­py would en­gi­neer a pa­tient’s own stem cells to fight HIV, and stim­u­late them to pro­duce new im­mune cells once rein­tro­duced to the pa­tient.

“A home run would be that we com­plete­ly cure peo­ple of HIV,” Paula Can­non, a USC pro­fes­sor of mol­e­c­u­lar mi­cro­bi­ol­o­gy and im­munol­o­gy and co-di­rec­tor of the pro­gram, said in a state­ment. “What I’d be fine with is the idea that some­body no longer needs to take an­ti-HIV drugs every day be­cause their im­mune sys­tem is keep­ing the virus un­der con­trol, so that it no longer caus­es health prob­lems and, im­por­tant­ly, they can’t trans­mit it to any­body else.”

Hans-Pe­ter Kiem

Hans-Pe­ter Kiem, the Stephanus Fam­i­ly En­dowed Chair for Cell and Gene Ther­a­py at Fred Hutch, is the co-di­rec­tor. Har­vard Uni­ver­si­ty pro­fes­sor David Scad­den and Ma­gen­ta Ther­a­peu­tics are al­so col­lab­o­rat­ing on the project.

The ap­proach was in­spired by three pa­tients who ap­pear to have been cured of the virus — all of whom re­ceived blood stem cell trans­plants from donors who car­ried a mu­ta­tion in the CCR5 gene. One of them, dubbed the “Berlin pa­tient,” has been off an­ti­retro­vi­ral drugs since 2007.

“I think of the Berlin pa­tient as proof of prin­ci­ple that re­plac­ing the im­mune sys­tem with one that’s HIV-re­sis­tant by re­mov­ing CCR5 is a pos­si­ble way to treat some­body,” Can­non said.

The pro­gram will study the use of gene edit­ing to re­move CCR5 from pa­tients’ stem cells — a process which is al­ready in clin­i­cal tri­al for HIV treat­ment at City of Hope Na­tion­al Med­ical Cen­ter in Duarte, CA. The stem cells will al­so be en­gi­neered to re­lease an­ti­bod­ies and an­ti­body-like mol­e­cules that block HIV.

In ad­di­tion, the grant will fund a Fred Hutch team’s en­deav­or to adapt CAR-T cell ther­a­py to cre­ate stem cells whose prog­e­ny tar­get HIV-in­fect­ed cells.

As for prepar­ing a pa­tient for the trans­plant, Ma­gen­ta is work­ing on an­ti­body-drug con­ju­gates to re­place mild chemother­a­py or ra­dio­ther­a­py typ­i­cal­ly giv­en be­fore the pro­ce­dure. And Scad­den is re­search­ing an in­jectable gel that could help im­mune cells re­pop­u­late more quick­ly, avoid­ing a de­lay.

HIV in­fec­tion, which cur­rent­ly af­fects about 1.2 mil­lion Amer­i­cans, has proved to be ex­ceed­ing­ly dif­fi­cult to cure. In Ju­ly, Mer­ck and Dew­point inked a deal that al­lows the phar­ma to use the Boston-based biotech’s bio­mol­e­c­u­lar con­den­sate tech­nol­o­gy to de­vel­op treat­ments, and po­ten­tial­ly a cure, for the HIV virus. The NIH-fund­ed group is hop­ing to at least con­trol the virus enough to elim­i­nate the need for dai­ly meds. But at best, they’re al­so eye­ing a long sought-af­ter cure.

“This grant funds a team with an over­ar­ch­ing goal of de­vel­op­ing what our per­fect HIV gene ther­a­py would look like,” Can­non said. “All of these pieces could hap­pen sep­a­rate­ly, but the fact that the NIH has fund­ed us as a team means that the sum will be so much big­ger than the parts.”

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.

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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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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.

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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