Ax­o­vant shares blast­ed as Vivek Ra­maswamy's pre­mier drug proves worth­less, sec­ond drug miss­es key goal

Vivek Ra­maswamy

Vivek Ra­maswamy’s first big bet on bring­ing a drug back from Big Phar­ma’s dis­card list is a com­plete bust. And an­oth­er set­back in the pipeline has con­spired to scut­tle its stock price this morn­ing.

The biotech fi­nancier’s Ax­o­vant, float­ed in an IPO that fund­ed an ex­pen­sive quest to put in­tepir­dine back in­to the clin­ic for neu­ro con­di­tions, re­port­ed out this morn­ing that both dos­es of the ther­a­py test­ed for de­men­tia with Lewy bod­ies failed to sig­nif­i­cant­ly im­prove symp­toms for pa­tients. The drug has al­so failed spec­tac­u­lar­ly for Alzheimer’s, leav­ing Ra­maswamy’s op­er­a­tion at Roivant with no choice but to kill the pro­gram.

The da­ta ac­tu­al­ly high­light­ed a wors­en­ing in dis­ease scores for two mea­sures used to eval­u­ate the low dose in the study while the top dose con­sis­tent­ly fell short of the mark.

In­vestors bailed on the news, send­ing the stock down 49% in pre-mar­ket trad­ing.

David Hung

Said Ax­o­vant CEO David Hung in a state­ment:

“Based on the to­tal­i­ty of in­tepir­dine da­ta to date, there is no ev­i­dence to sup­port its fur­ther de­vel­op­ment.”

Ra­maswamy was able to in-li­cense the failed Alzheimer’s drug from GSK for on­ly $5 mil­lion. But he sold it to in­vestors in a record-set­ting IPO back in 2015, haul­ing in $315 mil­lion dur­ing a big bull mar­ket for biotech. Since then he’s gath­ered about $2 bil­lion to­geth­er for a set of star­tups that have been busi­ly scoop­ing up drugs off of bio­phar­ma’s shelves. But every miss in the clin­ic will raise new ques­tions about the val­ue of his strat­e­gy.

Ra­maswamy bad­ly needs a win.

In an ef­fort to try and take some of the sting out of the lead fail­ure, Ax­o­vant spot­light­ed some hope­ful signs seen in a post hoc analy­sis on a sep­a­rate study of nelotanserin. But that drug al­so failed the key ef­fi­ca­cy end­point for de­men­tia pa­tients with vi­su­al hal­lu­ci­na­tions.

In a pre­spec­i­fied ITT analy­sis, nelotanserin treat­ment ver­sus place­bo (n=27) re­sult­ed in a 3.12 point im­prove­ment in the (Uni­fied Parkin­son’s Dis­ease Rat­ing Scale) Part III with a pos­i­tive trend (p=0.075, un­ad­just­ed). No­tably, in a pre­spec­i­fied analy­sis of the DLB pa­tient sub­set (n=19), nelotanserin im­proved the UP­DRS Part III by 4.00 points (p=0.041, un­ad­just­ed).

About a year ago Ax­o­vant tout­ed pos­i­tive signs from the first stage of the study, but al­so con­ced­ed a key miss on the sec­on­daries, fail­ing to im­prove the fre­quen­cy of vi­su­al hal­lu­ci­na­tions. Nev­er­the­less, the com­pa­ny head­ed straight in­to Phase III to un­der­score Ra­maswamy’s com­mit­ment to rapid-fire clin­i­cal test­ing.

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Law pro­fes­sors call for FDA to dis­close all safe­ty and ef­fi­ca­cy da­ta for drugs

Back in early 2018 when Scott Gottlieb led the FDA, there was a moment when the agency seemed poised to release redacted complete response letters and other previously undisclosed data. But that initiative never gained steam.

Now, a growing chorus of researchers are finding that a dearth of public data on clinical trials and pharmaceuticals means industry and the FDA cannot be held accountable, two law professors from Yale and New York University write in an article published Wednesday in the California Law Review.

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Novavax CEO Stanley Erck at the White House in 2020 (Andrew Harnik, AP Images)

As fears mount over J&J and As­traZeneca, No­vavax en­ters a shaky spot­light

As concerns rise around the J&J and AstraZeneca vaccines, global attention is increasingly turning to the little, 33-year-old, productless, bankruptcy-flirting biotech that could: Novavax.

In the now 16-month race to develop and deploy Covid-19 vaccines, Novavax has at times seemed like the pandemic’s most unsuspecting frontrunner and at times like an overhyped also-ran. Although they started the pandemic with only enough cash to last 6 months, they leveraged old connections and believers into $2 billion and emerged last summer with data experts said surpassed Pfizer and Moderna. They unveiled plans to quickly scale to 2 billion doses. Then they couldn’t even make enough material to run their US trial and watched four other companies beat them to the finish line.

FDA of­fers scathing re­view of Emer­gent plan­t's san­i­tary con­di­tions, em­ploy­ee train­ing af­ter halt­ing pro­duc­tion

The FDA wrapped up its inspection of Emergent’s troubled vaccine manufacturing plant in Baltimore on Tuesday, after halting production there on Monday. By Wednesday morning, the agency already released a series of scathing observations on the cross contamination, sanitary issues and lack of staff training that caused the contract manufacturer to dispose of millions of AstraZeneca and J&J vaccine doses.

Brad Bolzon (Versant)

Ver­sant pulls the wraps off of near­ly $1B in 3 new funds out to build the next fleet of biotech star­tups. And this new gen­er­a­tion is built for speed

Brad Bolzon has an apology to offer by way of introducing a set of 3 new funds that together pack a $950 million wallop in new biotech creation and growth.

“I want to apologize,” says the Versant chairman and managing partner, laughing a little in the intro, “that we don’t have anything fancy or flashy to tell you about our new fund. Same team, around the same amount of capital, same investment strategy. If it ain’t broke, don’t fix it.”

But then there’s the flip side, where everything has changed. Or at least speeded into a relative blur. Here’s Bolzon:

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Emma Walmsley, GlaxoSmithKline CEO (Kevin Dietsch/Pool via CNP/Alamy)

Glax­o­SmithK­line hus­tles the 7th PD-1 past the fin­ish line with Jem­per­li. But how big will up­take be?

Everything came up sevens for GlaxoSmithKline on Thursday as the pharma notched the seventh PD-1 approval seven years after the first such drugs were OK’ed in Keytruda and Opdivo. But will it bring GSK good fortune?

The FDA granted accelerated approval to dostarlimab, to be branded Jemperli, to treat recurrent or advanced endometrial cancer in a specific subset of patients following platinum-based chemo. It’s a drug that came to GSK through its buyout of Tesaro, which it snapped up for $5.1 billion back in December 2018.

JP Gabriel, Ocugen

JP Gabriel watched from the bleach­ers as the pan­dem­ic raged. Now head of sup­ply chain at Ocu­gen, he's ready to bat

The world was in the middle of the most pressing public health risk his generation had ever seen, and JP Gabriel felt like he was sitting on the sidelines. As a VP of biologics and mRNA manufacturing at Ultragenyx, Gabriel watched from the sidelines as players like Pfizer/BioNTech and Moderna used mRNA tech to chase their own Covid-19 vaccines.

This month, Gabriel got the chance to get his hands dirty against the pandemic — but it won’t be with mRNA.

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Sen. Patty Murray (D-WA) (Graeme Sloan/Sipa USA/Sipa via AP Images)

Sen­a­tors to NIH: Do more to pro­tect US bio­med­ical re­search from for­eign in­flu­ence

Although Thursday’s Senate health committee hearing was focused on how foreign countries and adversaries might be trying to steal or negatively influence biomedical research in the US, the only country mentioned by the senators and expert witnesses was China.

Committee chair Patty Murray (D-WA) made clear in her opening remarks that the US cannot “let the few instances of bad actors” overshadow the hard work of the many immigrant researchers in the US, many of which have won Nobel prizes for their work. But she also said, “There is more the NIH can be doing here.”

Jenny Rooke (Genoa Ventures)

Ear­ly Zymer­gen in­vestor Jen­ny Rooke re­flects on 'chimeras' in biotech, what it takes to spot a $500M gem

When Jenny Rooke first heard of Zymergen back in 2014, she knew she was looking at something different and exciting. The Emeryville, CA biotech held the promise of blending biology and technology to solve a huge unmet need for cost-effective chemicals — of all things — and a stellar founding team to boot.

But back then, West Coast venture capitalists didn’t see in Zymergen the one thing they were looking for in a winning biotech: therapeutic potential. Rooke, however, saw an opportunity and made her bets. Seven years later, that bet is paying off in a big way.

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