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.”

Biogen CEO Michel Vounatsos (via Getty Images)

With ad­u­canum­ab caught on a cliff, Bio­gen’s Michel Vounatsos bets bil­lions on an­oth­er high-risk neu­ro play

With its FDA pitch on the Alzheimer’s drug aducanumab hanging perilously close to disaster, Biogen is rolling the dice on a $3.1 billion deal that brings in commercial rights to one of the other spotlight neuro drugs in late-stage development — after it already failed its first Phase III.

The big biotech has turned to Sage Therapeutics for its latest deal, close to a year after the crushing failure of Sage-217, now dubbed zuranolone, in the MOUNTAIN study.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,300+ biopharma pros reading Endpoints daily — and it's free.

Pascal Soriot (AP Images)

As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,300+ biopharma pros reading Endpoints daily — and it's free.

Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan.

The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,300+ biopharma pros reading Endpoints daily — and it's free.

The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,300+ biopharma pros reading Endpoints daily — and it's free.

Bob Nelsen (Photo by Michael Kovac/Getty Images)

Bob Nelsen rais­es $800M and re­cruits a star-stud­ded board to build the 'Fox­con­n' of biotech

Bob Nelsen spent his pandemic spring in his Seattle home, talking on the phone with Luciana Borio, the scientist who used to run pandemic preparedness on the National Security Council, and fuming with her about the dire state of American manufacturing.

Companies were rushing to develop vaccines and antibodies for the new virus, but even if they succeeded, there was no immediate supply chain or infrastructure to mass-produce them in a way that could make a dent in the outbreak.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,300+ biopharma pros reading Endpoints daily — and it's free.

Carl Hansen, AbCellera CEO (University of British Columbia)

From a pair of Air Jor­dans to a $200M-plus IPO, Carl Hansen is craft­ing an overnight R&D for­tune fu­eled by Covid-19

Back in the summer of 2019, Carl Hansen left his post as a professor at the University of British Columbia to go full time as the CEO at a low-profile antibody shop he had founded called AbCellera.

As biotech CEOs go, even after a fundraise Hansen wasn’t paid a whole heck of a lot. He ended up earning right at $250,000 for the year. His compensation package included a loan — which he later paid back — and a pair of Air Jordan tennis shoes. His newly-hired CFO, Andrew Booth, got a sweeter pay packet than that — which included his own pair of Air Jordans.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,300+ biopharma pros reading Endpoints daily — and it's free.

Vipin Suri, Catamaran Bio CSO

Cata­ma­ran Bio sails in­to the CAR-NK wa­ters with a $42M launch round

Catamaran Bio’s founding members decided to jump into the CAR-NK game last December over drinks at a trendy bar in Boston.

They were sitting around a table, discussing an MD Anderson study which provided some of the first clinical proof that natural killer (NK) cells can be reengineered to attack tumors, much like CAR-T therapies. It was a “long and lively” discussion, COO Mark Boshar recalls. And by the time it was over, they had a starting point to launch a company.