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.

Un­pack­ing the Aduhelm de­ci­sion, Ver­tex's half full glass, a $525M J&J breakup, 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.

By now you have surely read about the FDA’s controversial approval of Biogen’s Alzheimer’s drug and all its reverberations. But I’d still recommend checking out the meaty recap below to make sure you didn’t miss all the angles that the Endpoints team has covered. If you’d rather look ahead, look no further than our three-day virtual panels next week at BIO, where we will discuss what the new normal means for every part of the industry.

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What does a clear ma­jor­i­ty of the bio­phar­ma in­dus­try think of the FDA ap­proval of ad­u­canum­ab? 'Hor­ri­fy­ing' 'Dan­ger­ous' 'Con­fus­ing' 'Dis­as­ter'

Over the years, we’ve become used to seeing a consensus emerge early in our industry polls at Endpoints News. And when we took the pulse of drug hunters on the heels of a controversial FDA approval for aducanumab this week, it became immediately apparent that the vast majority of our readers — heavily concentrated among biopharma staffers and execs — were incensed by what they had just witnessed.

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Aaron Kesselheim (Scott Eisen/AP Images for AIDS Healthcare Foundation)

Har­vard’s Aaron Kessel­heim re­signs from ex­pert pan­el in wake of ad­u­canum­ab OK, blast­ing FDA for ‘worst drug ap­proval de­ci­sion in re­cent U.S. his­to­ry'

A third member of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee has resigned in the wake of Biogen’s controversial Aduhelm approval, slamming the agency as he left and further deepening the controversy surrounding the decision.

Harvard University professor Aaron Kesselheim quit in protest Thursday afternoon, calling the Aduhelm OK “probably the worst drug approval decision in recent U.S. history.” Kesselheim follows both Joel Perlmutter, a neurologist from Washington University in St. Louis, and David Knopman, a neurologist from the Mayo Clinic, out the door.

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David Knopman (Mayo Clinic via YouTube)

A sec­ond ad­comm mem­ber aban­dons his post in af­ter­math of con­tro­ver­sial ad­u­canum­ab de­ci­sion

As the fallout from the FDA’s approval of Alzheimer’s med aducanumab grows, a second member of the adcomm overseeing that drug’s review has walked away. But even with two experts now having resigned from that committee in protest, is there enough broad-level outrage to prevent another aducanumab from getting approved?

The FDA on Wednesday lost another member of its Peripheral and Central Nervous System Drugs Advisory Committee as Mayo Clinic neurologist David Knopman hit the exit over the agency’s decision to approve Biogen’s Alzheimer’s drug Aduhelm despite the committee’s near-unanimous vote against it.

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FDA au­tho­rizes about 10M J&J vac­cine dos­es, trash­es 60M more from trou­bled Emer­gent plant

The FDA on Friday released about 10 million doses of J&J’s vaccine for use, and disposed of another 60 million doses that were manufactured at the now-shuttered Emergent BioSolutions facility in Baltimore where cross-contamination occurred.

The agency said it’s not yet ready to allow the Emergent plant to be included in the J&J EUA, but that may occur soon. FDA came to the decision to authorize some of the doses after reviewing facility records and quality testing results.

As it ex­pands its foot­print, Mod­er­na reach­es deal to man­u­fac­ture Covid-19 vac­cine dos­es in Mid­dle East

While the UAE leads the world with the highest percentage of residents vaccinated, neighboring Saudi Arabia — home to nearly 35 million people — has lagged behind significantly. On Friday, Moderna announced that it has partnered with the Saudi pharmaceutical company Tabuk to manufacture its jab and future variant-specific boosters in the country.

Tabuk will hold marketing authorization for the vaccine in Saudi Arabia, and the agreement gives them the possibility of distributing future Moderna mRNA products.

Covid-19 roundup: 60 mil­lion J&J dos­es from Emer­gent to be tossed; EMA up­dates As­traZeneca vac­cine warn­ings

After a two-month hold on millions of doses of the J&J vaccine, the FDA will announce that it is releasing 10 million doses and trashing 60 million that were made at a troubled Baltimore plant, The New York Times scooped on Friday morning.

Emergent BioSolutions reported that the doses were contaminated after drug material intended for the AstraZeneca vaccine came in close contact with media prepared for a J&J run. As a result, the doses were held from distribution, causing a shakeup in early availability.

Janet Woodcock, acting FDA commissioner, at Thursday's Senate Appropriations hearing (Bill Clark/CQ Roll Call via AP Images)

Sen­a­tors lam­bast new Alzheimer’s drug’s price but give Janet Wood­cock a free pass on the ap­proval de­ci­sion

Senate Finance Democrats took aim at Biogen’s pricey new Alzheimer’s drug on Thursday, but members on both sides of the aisle at a separate appropriations hearing didn’t question acting FDA commissioner Janet Woodcock on the approval.

“I was appalled that Biogen priced their Alzheimer’s drug approved by the FDA at $56,000 per year — I’m not going to debate whether this is effective or not, but it’s double the household median income for Michiganders over the age of 65,” Sen. Debbie Stabenow (D-MI) said at the finance hearing.

Reshma Kewalramani, Vertex CEO (BIO via YouTube)

UP­DAT­ED: Ver­tex strikes out on its lat­est big shot at a rare ge­net­ic dis­ease. But they're go­ing to keep on swing­ing

It’s been several months since Vertex culled one of its small molecules for alpha-1 antitrypsin deficiency (AATD), taking a big hit after evidence of liver damage surfaced in a key Phase II trial. Now we learned that the company has whiffed on its second shot, and there’s nothing left in the clinic to treat the rare genetic disease — but that won’t stop it from trying.

Despite avoiding the safety issues that plagued the last candidate, Vertex $VRTX is taking the axe to VX-864 after Phase II results revealed the magnitude of the drug’s response is “unlikely to translate into substantial clinical benefit.” As a result of the news, the company’s stock fell 12.5% after hours.

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