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.

Bio­mark­er 'roadmap­s' and the fu­ture of can­cer R&D; Cur­tain rais­es on #AS­CO22; Pfiz­er, No­var­tis tack­le drug ac­cess; 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.

While this was not a week for earth-shattering news, there were certainly a lot of interesting tidbits. If you found this recap helpful, please recommend it to your friends and colleagues. We’ll see you on the other side of the long weekend.

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Keep­ing pres­sure on Am­gen, Mi­rati draws mixed re­views on lat­est cut of KRAS da­ta

As the close runner-up to Amgen’s Lumakras in the KRAS race, any data cut from Mirati’s adagrasib continues to draw scrutiny from analysts. And the latest batch of numbers from ASCO is a decidedly mixed bag.

While a quick comparison suggests that adagrasib spurred slightly more responses and led to a longer overall survival than Lumakras among a group of non-small cell lung cancer patients, its duration of response appears shorter and the safety profile continues to spark concern.

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Switzer­land to de­stroy over 600,000 ex­pired dos­es of Mod­er­na Covid vac­cine

As concerns related to uptake and distribution continue to linger, Switzerland is among the first countries that plans to destroy hundreds of thousands of expired and unused Covid-19 vaccine doses.

The European country said it plans to destroy more than 600,000 doses of Moderna’s Spikevax Covid-19 vaccine as the doses have reached their expiration date.

However, Moderna CEO Stéphane Bancel told the World Economic Forum in Davos, Switzerland that he’s in the process of throwing 30 million doses in the garbage, exclaiming, “We have a big demand problem.”

Ann is one of ViiV Healthcare's newest spokespeople as the retired school administrator speaks up about her HIV status.

GSK's Vi­iV de­buts next evo­lu­tion in HIV med Dova­to cam­paign with new spokes­peo­ple and new mes­sage

When Ann saw the first TV commercials for HIV medicine Dovato, she didn’t see herself represented. So the 74-year-old retired school administrator who’s been living with HIV since 1998, reached out to GSK’s ViiV Healthcare and asked why not?

Now Ann is one of three people starring in ViiV’s latest Dovato campaign called “Detect This.” The next-step evolution in the branded campaign plays on the word “detect” — often used in describing HIV status under control as undetectable — but in this case, uses the word as a directive for people to understand they can use fewer medicines.

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Tran­si­tion to new Eu­ro­pean clin­i­cal tri­als in­fo sys­tem starts slow­ly

At the end of January, the European Medicines Agency officially launched its new clinical trials info system (CTIS), although the migration to the new platform has only really just begun, and sponsors have until the end of January 2023 before all initial trial applications must be submitted through CTIS.

Overall, 56 clinical trial applications have been submitted in CTIS during the first 3 months since the launch of the system on Jan. 31, according to new data posted by the EMA. By comparison, about 4,000 new trials are authorized each year across Europe.

Covid-19 roundup: CDC study shows good pro­tec­tion from mR­NA boost­er plus J&J's sin­gle-dose vac­cine; White House launch­es test-to-treat Paxlovid site

Concerns about rare but life-threatening blood clots have limited the use of J&J’s Covid-19 vaccine — once pitched as the only one-shot option in the mix — with the FDA cutting it off except in limited circumstances. Yet there’s some good news for those who did receive it: A single booster dose of an mRNA vaccine for recipients of a single priming dose of Ad26.COV2.S (the J&J vaccine) provided protection close to that of a three-dose mRNA vaccine regimen.

Nassim Usman, Catalyst Biosciences CEO

Af­ter $60M Ver­tex deal, group of Cat­a­lyst share­hold­ers claims biotech could’ve sold as­sets three years ago

Catalyst Biosciences was down to five employees in March, and the biotech needed to do something after two rounds of layoffs, a nixed collaboration and a culling of its hemophilia program.

In came Vertex, with $60 million to buy up the South San Francisco biotech’s preclinical complement drugs, which target the system that bridges the body’s innate and adaptive immune response and a class most known for Ultomiris and Soliris. The deal includes CB 2782-PEG, the dry AMD drug that Biogen no longer wanted in March.

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Lina Khan, FTC chair (Graeme Jennings/Pool via AP Images)

Pile-on over PBMs con­tin­ues with FTC com­ments and a new bi­par­ti­san Sen­ate bill

More than 500 stakeholders sent comments to the FTC on whether the commission should look further into pharma middlemen, known as PBMs, with many of the commenters calling for more federal oversight.

Similar to the critical open comment period in a deadlocked FTC session last February, pharmacies and pharmacy groups are continuing to call out the lack of transparency among the top 3 PBMs, which control about 80% of the market.

Pharma brands are losing their shine with US consumers who are now thinking about the economy and inflation instead of Covid. (Credit: Shutterstock)

Phar­ma brands fade in an­nu­al Har­ris con­sumer vis­i­bil­i­ty poll: Mod­er­na drops off and Pfiz­er dips

As Covid-19 concerns are fading in the US, so is biopharma visibility. The annual Axios Harris Poll survey to determine and rank the 100 most top-of-mind brands in the US finds Moderna, which was No. 3 last year, not on the list at all for 2022, and Pfizer sinking 37 spots.

However, it’s not that Moderna or Pfizer did anything wrong, it’s just that Americans have moved on to other worries beyond Covid.

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