Jim Wilson at the WuXi Global Forum, JPM20

Gates Foun­da­tion backs gene ther­a­py pi­o­neer­s' sec­ond-gen Covid-19 vac­cine

The Gates Foun­da­tion has backed an­oth­er ear­ly-stage vac­cine ef­fort, hand­ing up to $2.1 mil­lion to a pre-clin­i­cal can­di­date from a pair of gene ther­a­py pi­o­neers they think can of­fer ben­e­fits over the first wave of Covid-19 vac­cines now rolling out around the globe.

Luk Van­den­berghe

The mon­ey will help ad­vance a vac­cine Luk Van­den­berghe and Jim Wil­son, di­rec­tors of the gene ther­a­py cen­ters at Mass­a­chu­setts Eye and Ear and the Uni­ver­si­ty of Penn­syl­va­nia, have been de­vel­op­ing over the past year based on the AAV vec­tors com­mon­ly used in gene ther­a­py. Now in non-hu­man pri­mate chal­lenge stud­ies, ear­ly da­ta sug­gest the vac­cine may work as a sin­gle dose and re­main sta­ble at room tem­per­a­ture, ad­dress­ing two of the dif­fi­cul­ties with rolling out the cur­rent gen­er­a­tion of vac­cines around the globe.

“When the da­ta came in that we had a num­ber of at­trib­ut­es that could be ben­e­fi­cial from a lo­gis­ti­cal glob­al de­ploy­ment per­spec­tive, we reached out to Gates,” Van­den­berghe told End­points News. These are “crit­i­cal at­trib­ut­es to get these vac­cines out, par­tic­u­lar­ly in ar­eas that are more chal­leng­ing from an in­fra­struc­ture per­spec­tive.”

Of course, Van­den­berghe is not the on­ly vac­cine de­vel­op­er to try to make a more scal­able and dis­trib­utable vac­cine; Mer­ck aimed for sim­i­lar goals with its two ex­per­i­men­tal in­oc­u­la­tions be­fore shut­ting down both pro­grams ear­li­er this month.

There are yet hun­dreds of vac­cine can­di­dates in ear­ly de­vel­op­ment, though, and the Gates Foun­da­tion has been back­ing a hand­ful it sees as par­tic­u­lar­ly promis­ing. That in­cludes a syn­thet­ic bi­ol­o­gy can­di­date from the Uni­ver­si­ty of Wash­ing­ton that may al­so be sin­gle-dose and shelf-sta­ble. And they’ve al­so giv­en a $20 mil­lion grant to the Coali­tion for Epi­dem­ic Pre­pared­ness, which is se­lect­ing three to six “sec­ond gen­er­a­tion” vac­cines to back.

Van­den­berghe’s vac­cine op­er­ates sim­i­lar­ly to the ade­n­ovirus-based Covid-19 vac­cines de­vel­oped by J&J and As­traZeneca, us­ing a harm­less virus to shut­tle a gene for the coro­n­avirus spike pro­tein in­to hu­man cells. Rather than ade­n­ovirus, though, they used a form of the AAV vec­tor com­mon in gene ther­a­py.

The AAV vari­ant they chose, Van­den­berghe said, should ex­press the gene in­side cells for about 2 to 3 months — or­ders of mag­ni­tude short­er than you would want for gene ther­a­py, but al­so longer than mR­NA or ade­n­ovirus vec­tor vac­cines. In the­o­ry, he said, that could pro­duce a more po­tent and long-last­ing im­mune re­sponse.

The Gates Foun­da­tion dol­lars, though, will on­ly push them through pre-clin­i­cal stud­ies. Van­den­berghe said they are search­ing for ad­di­tion­al back­ers or phar­ma part­ners to help bring what has large­ly been an aca­d­e­m­ic ef­fort in­to the clin­ic in the next cou­ple months.

They are al­so look­ing to ex­pand their man­u­fac­tur­ing ca­pa­bil­i­ty. So far, they’ve re­lied on No­var­tis, which has AAV fa­cil­i­ties for pro­duc­ing the SMA gene ther­a­py Zol­gens­ma. Be­cause their vac­cines re­quire about 1/10,000 of the dose need­ed for gene ther­a­py, Van­den­berghe said they should be able to ex­tract 100 mil­lion dos­es from cur­rent AAV fa­cil­i­ties even with­out the fa­cil­i­ty ex­pan­sions oth­er vac­cines have re­lied on.

So far, he said, they’ve pro­duced four lots of the vac­cine — a small num­ber com­pared to ma­jor de­vel­op­ers, but sig­nif­i­cant for an aca­d­e­m­ic ef­fort boot-strapped in the mid­dle of a pan­dem­ic.

“This is not in line with larg­er funds and we cer­tain­ly are not the fron­trun­ners here,” Van­den­berghe said. “The goal is to ad­dress some of the short­com­ings of the first wave of vac­cines.”

For a look at all End­points News coro­n­avirus sto­ries, check out our spe­cial news chan­nel.

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; 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.

With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

Trodelvy notch­es a win in most com­mon form of breast can­cer

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.

Raymond Stevens, Structure Therapeutics CEO

Be­hind Fri­day's $161M IPO: A star sci­en­tist, GPCR drug dis­cov­ery and a plan to chal­lenge phar­ma in di­a­betes

What does it take to pull off a $161 million biotech IPO these days?

In Structure Therapeutics’ case, it means having a star scientist co-founder paired with the computational drug discovery company Schrödinger, $198 million in private funding from blue-chip investors, almost six years of research work on G protein-coupled receptors and a slate of oral, small-molecule drugs, with an eye on the huge and growing diabetes and weight-loss market.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

Af­ter 13 years, Ramy Mah­moud steps in­to CEO seat at Opti­nose; Ru­pert Vessey set to ex­it Bris­tol My­ers in Ju­ly

After 13 years as president and COO at Optinose, Ramy Mahmoud has stepped into a new role as its CEO. He is taking the place of Peter Miller, who stepped down earlier this week, though Miller is still staying with the company as a consultant.

In 2010, the two business partners joined Optinose to take it in a new direction, transforming it from a delivery platform to product company. They previously worked together at Johnson & Johnson, when Miller was president at Janssen and Mahmoud headed medical affairs. Miller said after he learned about Optinose, “I did what I always do, which is find people smarter than me to talk with about the idea. And the first person I called was Ramy … and I said, ‘Hey, Ramy, what do you think of this technology?’”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

Photo: Julia Weeks/AP Images

FDA ax­es re­quire­ment for pos­i­tive Covid test be­fore Paxlovid use

FDA announced today that doctors and pharmacists can now prescribe Paxlovid to patients without a positive test for Covid-19.

CDER Director Patrizia Cavazzoni reissued Paxlovid’s authorization letter Wednesday, saying it has revised the authorization to “no longer require positive results of direct SARS-CoV-2 viral testing.” The EUA now requires instead that adults and kids 12 years of age and older have a “current diagnosis of mild-to-moderate COVID-19.”

Te­va drops out of in­dus­try trade group PhRMA

Following in AbbVie’s footsteps, Teva confirmed on Friday that it’s dropping out of the industry trade group Pharmaceutical Research and Manufacturers of America (PhRMA).

Teva didn’t give a reason for its decision to leave, saying only in a statement to Endpoints News that it annually reviews “effectiveness and value of engagements, consultants and memberships to ensure our investments are properly seated.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

Sanofi CFO Jean-Baptiste de Chatillon (L) and CEO Paul Hudson (Romuald Meigneux/Sipa via AP Images)

Sanofi sees downtick in flu sales as it preps for launch of RSV an­ti­body

Sanofi expects its RSV antibody jointly developed with AstraZeneca will be available next season, executive VP of vaccines Thomas Triomphe announced on the company’s quarterly call.

Beyfortus, also known as nirsevimab, was approved in the EU back in November and is currently under FDA review with an expected decision coming in the third quarter of this year. The news comes as the FDA plans to hold advisory committee meetings over the next couple months to review RSV vaccines from Pfizer and GSK.

Christophe Weber, Takeda CEO (Photographer: Shoko Takayasu/Bloomberg via Getty Images)

Take­da fo­cus­es on ‘di­verse’ pipeline prospects on heels of two ac­qui­si­tions

After a whopping $4 billion asset buy from Nimbus Therapeutics, along with a $400 million deal with Hutchmed for a colorectal cancer drug, Takeda executives touted pipeline optimism on its latest earnings call this week.

That’s because the TYK2 inhibitor for psoriasis Takeda is getting from Nimbus, along with the Hutchmed fruquintinib commercialization outside of China, are just two of what it reports are 10 late-stage development programs of promising candidates.