Scot Roberts, Altimmune CSO

Why mu­cos­al im­mu­ni­ty may be re­quired to end the pan­dem­ic

Biotech Voices is a collection of exclusive opinion editorials from some of the leading voices in biopharma on the biggest industry questions today. Think you have a voice that should be heard? Reach out to Amber Tong.

Fol­low­ing the re­cent emer­gency use au­tho­riza­tions of mul­ti­ple vac­cines for COVID-19, the first wave of peo­ple have al­ready be­gun the vac­ci­na­tion process — a tru­ly re­mark­able mile­stone. As some­one who has ded­i­cat­ed his ca­reer to drug de­vel­op­ment and dis­cov­ery fo­cused on vi­ral vec­tors and an­tivi­ral ther­a­pies, I can tell you that the sci­ence has moved fast, and we have every rea­son to cel­e­brate. The da­ta on these vac­cines are good. They are well stud­ied and their po­ten­tial for pub­lic health is promis­ing.

How­ev­er, I can al­so tell you that this is like­ly on­ly the be­gin­ning. I wor­ry that the roll­out of the cur­rent vac­cines will cre­ate a false sense of se­cu­ri­ty among a large pop­u­la­tion of peo­ple. There is a sce­nario that we need to be pre­pared for and one that could pos­si­bly de­rail our fight against this virus: These first-gen­er­a­tion vac­cines will be able to ef­fec­tive­ly block dis­ease, but not the trans­mis­sion of the SARS-CoV-2 virus.

Pfiz­er, Mod­er­na and near­ly every oth­er phar­ma­ceu­ti­cal com­pa­ny are pro­duc­ing in­jectable vac­cines. Their pro­tec­tive pow­er re­sults from sys­temic im­mu­ni­ty, mean­ing that they gen­er­ate an­ti­bod­ies that cir­cu­late in the blood to oth­er parts of the body. They were au­tho­rized based on their abil­i­ty to block the de­vel­op­ment of dis­ease, which is not the same as the abil­i­ty to in­hib­it vi­ral in­fec­tion or trans­mis­sion.

The best way to block both vi­ral in­fec­tion and trans­mis­sion is by in­duc­ing a spe­cial type of im­mune re­sponse called mu­cos­al im­mu­ni­ty. Mu­cos­al im­mu­ni­ty pro­tects the parts of the body that are in con­tact with the en­vi­ron­ment, and in the case of a res­pi­ra­to­ry pathogen, that means the nasal cav­i­ty and the lungs. Be­cause it is a lo­cal­ized type of re­sponse, a vac­cine needs to be de­liv­ered to the ap­pro­pri­ate com­part­ment if mu­cos­al im­mu­ni­ty is to be stim­u­lat­ed. In the case of a res­pi­ra­to­ry pathogen like SARS-CoV-2 that means in­tranasal vac­cine ad­min­is­tra­tion.

With­out mu­cos­al im­mu­ni­ty, the virus may con­tin­ue to thrive in a per­son’s nasal cav­i­ty al­low­ing for easy trans­mis­sion. This is why wear­ing a mask, even af­ter vac­ci­na­tion with an in­tra­mus­cu­lar vac­cine, will re­main crit­i­cal.

Thank­ful­ly, sci­en­tists, in­clud­ing our team, are work­ing on next gen­er­a­tion vac­cines that can elic­it mu­cos­al im­mu­ni­ty, in­clud­ing in­tranasal vac­cines that have the po­ten­tial to elic­it an im­mune re­sponse in the nose as well as sys­tem­i­cal­ly. This type of broad­er im­mune re­sponse could de­fend against in­fec­tion, dis­ease and trans­mis­sion. In fact, an in­tranasal vac­cine could al­so be used in com­bi­na­tion with an in­tra­mus­cu­lar vac­cine to elic­it mu­cos­al im­mu­ni­ty and serve as a boost­er for sys­temic im­mu­ni­ty.

We have a prece­dent. Flu­Mist is a live but weak­ened in­fluen­za vac­cine de­liv­ered by in­tranasal spray and this type of mu­cos­al vac­cine has been shown to de­crease the trans­mis­sion of in­fluen­za to close con­tacts.

Be­cause of their po­ten­tial to block trans­mis­sion, vac­cines that stim­u­late nasal mu­cos­al im­mu­ni­ty may be es­sen­tial to bring the pan­dem­ic to an end. How­ev­er, it will take a lit­tle more time to de­vel­op and test these new­er vac­cines as we can’t sac­ri­fice ef­fi­ca­cy and safe­ty for speed.

Un­til then, the pub­lic must take pre­cau­tions to keep this virus un­der con­trol. Even af­ter vac­ci­na­tion, our best weapon is so­cial dis­tanc­ing, test­ing and masks. They must re­main a core part of our pub­lic health strat­e­gy even as the first-gen­er­a­tion vac­cines roll out.

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.

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.

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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Work taking place in the clean rooms at Vor (Credit: Vor)

Vor Bio opts to keep man­u­fac­tur­ing op­er­a­tions in-house for de­vel­op­ing stem cell, CAR-T ther­a­pies

While it is not uncommon for a biotech to go down the route of having the product manufactured by a contract organization, one small biotech is looking to keep its card close to its chest.

Vor Biopharma has started manufacturing operations at an in-house facility at its HQ in Cambridge, MA after beginning construction last summer.

According to the biotech, the facility aims to develop Vor’s hematopoietic stem cells (eHSCs) and CAR-T therapies for patients with blood cancers. The site will initially manufacture a clinical supply of its candidate VCAR33allo to support its IND, which is slated to be submitted in the first half of next year. It also plans to transfer the production of VOR33 to the facility. Vor is getting to work quickly as engineering runs for VCAR33allo has started this week.

Aim­ing for fourth nod, Sarep­ta files an­oth­er DMD gene ther­a­py to FDA; Ax­some head­ed to­ward mi­graine re­sub­mis­sion

Sarepta Therapeutics has filed the data needed for an FDA accelerated approval, which would be the biotech’s fourth if granted by the agency.

The biotech has yet to complete confirmatory trials for those first three conditional nods. The filing for its fourth Duchenne muscular dystrophy treatment, disclosed Thursday, is not a surprise. Sarepta said in late-July it would do so after releasing positive results for the Roche-partnered gene therapy.

Phillip Gomez, Siga Technologies CEO

Siga nabs $10.7M from the US gov­ern­ment in deal for its mon­key­pox an­tivi­ral

The US government is all set to buy $10.7 million worth of Siga Technologies’ monkeypox oral antiviral, the company announced Thursday.

Of the total doses, $5.1 million worth of oral antivirals called Tpoxx (tecovirimat) will be delivered this year, with the US Department of Defense having the option of buying the rest at a later point.

The new contract follows an earlier one in which the government had purchased $7.4 million worth of Tpoxx from the company.

Renhong Tang, Simcere co-CEO

Almi­rall part­ners up with Sim­cere in po­ten­tial $500M+ deal — with plans to take IL-2 can­di­date glob­al

A Chinese pharma is looking to go international with one of its preclinical candidates, and it’s teaming up with a Spanish company in a new pact potentially worth half a billion dollars to do just that.

Simcere and Almirall announced Thursday that the two companies had reached a deal for Simcere’s IL-2 mutant fusion protein drug candidate, called SIM0278. According to a statement, Almirall gets an exclusive right to develop and commercialize the drug candidate in all indications and markets outside of China, Hong Kong, Taiwan and Macau.

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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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