Bernat Olle, Vedanta Biosciences CEO

Vedan­ta brings home a win for lead mi­cro­bio­me pro­gram, pass­ing a PhII test and clinch­ing BAR­DA in­vest­ment

Vedan­ta Bio­sciences wrapped up a Se­ries D about two months ago to ad­vance its mi­cro­bio­me re­search, but it’s not con­tent to rest on its lau­rels.

The Cam­bridge, MA-based biotech claimed a win in a Phase II study treat­ing Clostrid­ioides dif­fi­cile in­fec­tion, say­ing its lead pro­gram achieved a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion in re­cur­rences af­ter eight weeks com­pared with place­bo. It’s a re­sult that os­ten­si­bly im­pressed the US gov­ern­ment as the study re­sults trig­gered a $23.8 mil­lion op­tion from BAR­DA.

“We were al­ready get­ting BAR­DA sup­port, but the ad­di­tion­al sup­port we’re get­ting now, the $23.8 mil­lion is more­so go­ing to­ward the Phase III study,” CEO Bernat Olle told End­points News in an in­ter­view.

Vedan­ta still has to sit down with the FDA to agree on a study de­sign, Olle added, but not­ed it will like­ly in­volve the same pa­tient pop­u­la­tion.

Olle and his team had been work­ing on the can­di­date known as VE303, an oral drug com­pris­ing eight dif­fer­ent bac­te­ria strains de­signed to pro­vide re­sis­tance to C. dif­fi­cile. The Phase II study en­rolled 79 pa­tients and ran­dom­ized them be­tween a low dose, high dose and place­bo group.

The high dose achieved the pri­ma­ry end­point. In the ac­tive arm, pa­tients saw a 13.8% C. dif­fi­cile re­cur­rence rate com­pared to 45.5% in the place­bo group, with re­searchers us­ing a com­bi­na­tion of mea­sure­ment tools to de­tect in­fec­tion. The re­sult proved a greater than 80% re­duc­tion in the odds of a re­cur­rence, good for a p-val­ue of p=0.0077.

Olle high­light­ed that the dif­fer­ent tools used in the pri­ma­ry are the norm for these kinds of stud­ies. Rather than on­ly use tox­in test­ing, Olle said Vedan­ta al­so looked at PCR test­ing in its end­point analy­sis. And when pa­tient sam­ples were un­avail­able for test­ing, Vedan­ta re­lied on a clin­i­cian’s di­ag­no­sis.

Re­searchers de­sired a broad­er de­f­i­n­i­tion of what con­sti­tutes a re­cur­rence be­cause tox­in test­ing is in­her­ent­ly in­ac­cu­rate, Olle said. The tests can miss be­tween 20% to 50% of pos­i­tive cas­es if pa­tient sam­ples are not im­me­di­ate­ly frozen in trans­fer.

“The sam­ple starts de­grad­ing right away,” Olle said. “Those are pa­tients that have C. dif­fi­cile, but … by the time you get the re­sult you on­ly cap­ture a por­tion of them.”

To ham­mer this point home, Vedan­ta al­so mea­sured the pro­por­tion of pa­tients re­main­ing re­cur­rence-free af­ter eight weeks us­ing on­ly the tox­in test­ing. An analy­sis look­ing at the dif­fer­ence be­tween the 86.2% of those in the high-dose co­hort who did not see a re­cur­rence, and the 54.5% of place­bo pa­tients, did not prove sta­tis­ti­cal­ly sig­nif­i­cant.

The re­sult is the “broad­est and most com­pre­hen­sive” de­f­i­n­i­tion of C. dif­fi­cile in­fec­tion re­cur­rence, Olle said.

Vedan­ta is con­tin­u­ing to build out a man­u­fac­tur­ing fa­cil­i­ty to pre­pare for Phase III and po­ten­tial com­mer­cial­iza­tion, re­main­ing on track to com­plete it by the end of 2021. Should the Phase III study val­i­date what re­searchers saw with Tues­day’s re­sults, Olle says it will hope­ful­ly be enough for an FDA ap­proval.

At that point, Vedan­ta could be pro­duc­ing about 1 mil­lion dos­es per year. With sup­port from BAR­DA, Olle ex­pects some gov­ern­ment in­vest­ment to build the na­tion­al se­cu­ri­ty stock­pile in case of an out­break.

Af­ter many in­vestors fled the mi­cro­bio­me space fol­low­ing a Seres Ther­a­peu­tics flop in 2016, cash has slow­ly start­ed com­ing back to the field. In May 2020, Re­bi­otix pre­sent­ed pos­i­tive da­ta from a place­bo-con­trolled study for its own C. dif­fi­cile trans­plant ther­a­py, and in Ju­ly, af­ter Seres found promis­ing re­sults with a new tack, Nestlé dropped $525 mil­lion to fund de­vel­op­ment for their lead mi­cro­bio­me treat­ment.

Pfiz­er al­so got in on the ac­tion, hav­ing in­vest­ed $25 mil­lion in Vedan­ta back in Jan­u­ary to help ad­vance its slate of IBD pro­grams. With the biotech now past a Se­ries D and hav­ing a pos­i­tive Phase II re­sult, ques­tions about an IPO will abound. Olle left the door open but didn’t give away many hints to his plans.

“At the stage that we are at, we’re open to all kinds of fi­nanc­ing,” Olle said.

Cor­rec­tion: This ar­ti­cle has been up­dat­ed to cor­rect in­for­ma­tion about the na­ture of test­ing for C. dif­fi­cile. A com­bi­na­tion ap­proach us­ing tox­in test­ing, PCR test­ing and clin­i­cian di­ag­no­sis is the norm. 

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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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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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.

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.

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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