An­oth­er PhI­II RSV fail­ure thumps be­lea­guered No­vavax, but ex­ecs claim there's a way for­ward

Back in No­vem­ber 2016, when No­vavax was pick­ing up the scraps from a Phase III crash of its RSV vac­cine in old­er adults, the com­pa­ny point­ed to the Gates Foun­da­tion-backed pro­gram to test the vac­cine in in­fants as a “sig­nif­i­cant com­mer­cial op­por­tu­ni­ty.” It went on to be­come the lead pro­gram as No­vavax mount­ed an ar­du­ous come­back cam­paign for RSV-F vac­cine.

But to­day, ex­ecs con­ced­ed that the in­fant tri­al has suf­fered the same late-stage fate.

Stan­ley Er­ck

In­vestors showed lit­tle pa­tience for a com­pa­ny that’s been quick with ex­pla­na­tions but slow to de­liv­er. The stock $NVAX tanked more than 65% in pre-mar­ket trad­ing to $2.13.

As it turned out, im­mu­niz­ing moth­ers with ResVax while they are preg­nant did not pre­vent med­ical­ly sig­nif­i­cant low­er res­pi­ra­to­ry tract in­fec­tions caused by RSV in in­fants for the first three months of their lives — the pri­ma­ry end­point.

To be sure, No­vavax is op­er­at­ing in a tough field lit­tered with set­backs, in­clud­ing a flop from Re­gen­eron (which has since dropped its RSV an­ti­body). In a rare win, As­traZeneca re­cent­ly se­cured ex­pe­dit­ed re­views in the US and EU for a dif­fer­ent ap­proach — a sin­gle dose long-act­ing drug for a broad swath of new­borns.

What the treat­ment did achieve — and this is the sil­ver lin­ing that ex­ecs are hold­ing on­to as they try to beat down a path to ap­proval — was pro­tect­ing in­fants from “some of the most se­ri­ous con­se­quences of RSV, in­clud­ing RSV LR­TI hos­pi­tal­iza­tions and RSV LR­TI with se­vere hy­pox­emia,” CEO Stan­ley Er­ck said in a state­ment.

In oth­er words, ex­ecs elab­o­rat­ed on a con­fer­ence call, while the vac­cine was not deemed ef­fec­tive for com­mon — but less se­vere — man­i­fes­ta­tions of the res­pi­ra­to­ry syn­cy­tial virus, it ap­pears to pro­tect the small group of in­fants who get the worst at­tacks.

They al­so sug­gest­ed that vac­ci­nat­ing moth­ers at an ear­li­er stage of ges­ta­tion (from 28 to 33 weeks) might in­crease ef­fi­ca­cy — po­ten­tial­ly a key point in their pitch to reg­u­la­tors.

No­vavax of­fered a peek on the da­ta, which it plans to un­veil at a med­ical meet­ing. Re­spec­tive­ly, the ef­fi­ca­cy rates of ResVax in per-pro­to­col in­fants were as fol­lows::

  • 39% against med­ical­ly sig­nif­i­cant RSV LR­TI (97.5%CI, -1% to 64%)
  • 44% against RSV LR­TI hos­pi­tal­iza­tions (95%CI, 20% to 62%)
  • 48% against RSV LR­TI with se­vere hy­pox­emia (95%CI, -8% to 75%)

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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Matt Gline, Roivant Sciences CEO (Photo by John Sciulli/Getty Images for GLG)

Pfiz­er and Roivant team up again for an­oth­er 'Van­t', set­ting up an­ti-in­flam­ma­to­ry show­down with Prometheus

Pfizer and Roivant are teaming up to launch a new ‘Vant’ aimed at bringing a mid-stage anti-inflammatory drug to market, the pair announced Thursday.

There’s no name for the startup yet, nor are there any employees. Thus far, the new company and Roivant can be considered “one and the same,” Roivant CEO Matt Gline tells Endpoints News. But Pfizer is so enthusiastic about the target that it elected to keep 25% of equity in the drug rather than take upfront cash from Roivant, Gline said.

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Roche HQ in Basel, Switzerland. (Image credit: Kyle LaHucik/Endpoints News)

As com­peti­tors near FDA goal­post, Roche spells out its re­peat Alzheimer's set­back

Before Roche can turn all eyes on a new version of its more-than-once-failed Alzheimer’s drug gantenerumab, the Big Pharma had to flesh out data on the November topline failure at an annual conference buzzier than in years past thanks to hotly watched rivals in the field: Eisai and Biogen’s lecanemab, and Eli Lilly’s donanemab.

There was less than a 10% difference between Roche’s drug and placebo at slowing cognitive decline across two Phase III trials, which combined enrolled nearly 2,000 Alzheimer’s patients. In its presentation at the conference Wednesday, Roche said it saw less sweeping away of toxic proteins than it had anticipated. For years, researchers and investors have put their resources behind the idea that more amyloid removal would equate to reduced cognitive decline.

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SQZ Biotech slash­es head­count by 60% as founder/CEO hits ex­it — while Syn­log­ic lays off 25%

It’s a tough time for early-stage companies developing highly promising, but largely unproven, new technologies.

Just ask SQZ Biotechnologies and Synlogic. The former is bidding farewell to its founder and CEO and slashing the headcount by 60% as it pivots from its original cell therapy platform to a next-gen approach; the latter — a synthetic biology play founded by MIT’s Jim Collins and Tim Lu — is similarly “optimizing” the company to focus on lead programs. The resulting realignment means 25% of the staffers will be laid off.

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Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

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Paul Hudson, Sanofi CEO (Romuald Meigneux/Sipa via AP Images)

Sanofi and DN­Di aim to elim­i­nate sleep­ing sick­ness in Africa with promis­ing Ph II/III re­sults for new drug

The Drugs for Neglected Diseases initiative (DNDi) and Sanofi today said that their potential sleeping sickness treatment saw success rates of up to 95% from a Phase II/III study investigating the safety and efficacy of single-dose acoziborole.

The potentially transformative treatment for sleeping sickness would mainly be targeted at African countries, according to data published today in The Lancet Infectious Diseases medical journal. The clinical trial was led by DNDi and its partners in the Democratic Republic of the Congo (DRC) and Guinea, with the authors noting:

Lynn Baxter, Viiv Healthcare's head of North America

Vi­iV dri­ves new cor­po­rate coali­tion in­clud­ing Uber, Tin­der and Wal­mart, aimed at end­ing HIV

ViiV Healthcare is pulling together an eclectic coalition of consumer businesses in a new White House-endorsed effort to end HIV by the end of the decade.

The new US Business Action to End HIV includes pharma and health companies — Gilead Sciences, CVS Health and Walgreens — but extends to a wide range of consumer companies that includes Tinder, Uber and Walmart.

ViiV is the catalyst for the group, plunking down more than half a million dollars in seed money and taking on ringmaster duties for launch today on World AIDS Day, but co-creator Health Action Alliance will organize joint activities going forward. ViiV and the alliance want and expect more companies to not only join the effort, but also pitch in funding.

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