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.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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Ivan Cheung, Eisai US chairman and CEO

Bio­gen, Ei­sai re­fresh amy­loid hy­poth­e­sis with PhI­II show­ing Alzheimer's med slows cog­ni­tive de­cline

In the first look at Phase III data for lecanemab, Eisai and Biogen’s follow-up Alzheimer’s drug to the embattled Aduhelm launch, results show the drug passed with flying colors on a test looking at memory, problem solving and other dementia metrics.

One of the most-watched Alzheimer’s therapies in the clinic, lecanemab met the study’s primary goal on the CDR-SB — Clinical Dementia Rating-Sum of Boxes — giving the biotech the confidence to ask for full approval in the US, EU and Japan by next March 31. The experimental drug reduced clinical decline on the scale by 27% compared to placebo at 18 months, the companies said Tuesday night Eastern time and Wednesday morning in Japan.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Some­one old, some­one new: Mod­er­na pro­motes CTO, raids No­var­tis for re­place­ment amid pipeline push

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

Gilead names 'k­ing­pin­s' in coun­ter­feit HIV med law­suit

Gilead is mounting its counterfeit drug lawsuit, naming two “kingpins” and a complex network of conspirators who allegedly sold imitation bottles of its HIV meds, some of which ended up in US pharmacies.

The pharma giant on Wednesday provided an update on what it called a “large-scale, sophisticated counterfeiting conspiracy,” accusing two new defendants of “leading and orchestrating” a scheme to sell hundreds of millions of dollars in illegitimate drugs posing as meds such as Biktarvy and Descovy.

Vlad Coric, Biohaven CEO (Photo Credit: Andrew Venditti)

As Amy­lyx de­ci­sion waits in the wings, Bio­haven’s ALS drug sinks (again) in plat­form tri­al

The FDA’s decision on Amylyx’s ALS drug is set to come out sometime Thursday. In a space with few drugs, any approval would be a major landmark.

But elsewhere in the ALS field, things are a bit more tepid.

Thursday morning, Biohaven announced that its drug verdiperstat failed its arm of an ALS platform trial led by Massachusetts General Hospital. According to a press release, the drug did not meet its primary endpoint — improvement on an ALS functional status test — or any key secondary endpoints at 24 weeks. The trial had enrolled 167 patients, giving them either verdiperstat or placebo twice a day.

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Tar­sus looks to raise aware­ness of eye­lid mite dis­ease in cam­paign aimed at eye­care spe­cial­ists

Eyelid mite disease may be “gross” but it’s also fairly common, affecting about 25 million people in the US.

Called demodex blepharitis, it’s a well-known condition among eyecare professionals, but they often don’t always realize how common it is. Tarsus Pharmaceuticals wants to change that with a new awareness campaign called “Look at the Lids.”

The campaign and website debut Thursday — just three weeks after Tarsus filed for FDA approval for a drug that treats the disease.

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Marcelo Bigal, Ventus Therapeutics CEO

No­vo Nordisk joins No­var­tis, Roche in NL­RP3 are­na, bet­ting $70M cash on NASH, car­diometa­bol­ic us­es

As a drug target, the NLRP3 inflammasome has drawn serious interest from Big Pharma, inspiring a series of M&A deals from Novartis and Roche on top of venture investments by others. Now Novo Nordisk is jumping on the bandwagon — and the Danish pharma giant is taking the target where it knows best.

Novo Nordisk is getting its NLRP3 inhibitors from Ventus Therapeutics, a Versant-backed startup that set out to make some of the best NLRP3 drugs out there by incorporating new insights into the structure of the target complex.

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