Daniel Barber, Aquestive Therapeutics CEO

Aque­s­tive touts PhII da­ta — and speed — in po­ten­tial EpiPen re­place­ment can­di­date

A New Jer­sey biotech spe­cial­iz­ing in drug de­liv­ery via oral film is re­port­ing new Phase II da­ta, set­ting a count­down to have it be­fore the FDA by end of 2023.

Aque­s­tive Ther­a­peu­tics an­nounced new da­ta Tues­day for its can­di­date AQST-109, an oral film us­ing a pro­drug of ep­i­neph­rine to treat ana­phy­lac­tic shock from an al­ler­gic re­ac­tion.

Per the biotech, a read­out of the EPIPHAST II tri­al was to com­pare one dose of AQST-109 — ba­si­cal­ly a patch — to a dose of an EpiPen at 0.3 mg, and an in­tra­mus­cu­lar in­jec­tion of 0.3mg of ep­i­neph­rine that wasn’t EpiPen.

In a sin­gle dose test, the me­di­an Tmax (the time it takes for a drug to reach max­i­mum con­cen­tra­tion in the blood af­ter ad­min­is­tra­tion) was 12 min­utes for AQST-109, com­pared to 22.5 min­utes for EpiPen and 45 min­utes for the gener­ic in­jec­tion.

Ken Tru­itt

Aque­s­tive CMO Ken Tru­itt tells End­points News the “stan­dard” way of de­liv­er­ing ep­i­neph­rine — through the mus­cle — takes time to ac­tu­al­ly have a ther­a­peu­tic ef­fect af­ter en­ter­ing the blood­stream and reach­ing or­gans.

“If you com­pare us to the tried-and-true stan­dard of just reg­u­lar old IM (in­tra­mus­cu­lar) ep­i­neph­rine, you can see some room for im­prove­ment in the old stan­dard pro­file. It can get in­to the body, but then there’s a de­lay time be­fore they re­al­ly start get­ting in­to the blood­stream and start hav­ing phar­ma­co­dy­nam­ic ef­fects,” Tru­itt added.

Aque­s­tive said the re­sults for AQST-109 were “in line with ex­pec­ta­tions,” re­port­ing no ma­jor ad­verse events. As for why a patch un­der the tongue, the CMO said that area is high­ly vas­cu­lar and al­lows for faster ab­sorp­tion of the pro drug.

Shares of $AQST went up about 5% for the near pen­ny stock play­er, cur­rent­ly float­ing around $1.18 each.

CEO Daniel Bar­ber added that now with the da­ta in hand, the biotech has al­ready sub­mit­ted the in­fo to the FDA, and he told End­points the com­pa­ny will be meet­ing with the fed­er­al agency be­fore the end of the year. The end goal so far, ac­cord­ing to Bar­ber, is to get three re­quired stud­ies up and run­ning and com­plet­ed over the next year, ready to file by the end of 2023.

Those three stud­ies, ac­cord­ing to the CEO, are a piv­otal study, a small pe­di­atric study, and a hu­man fac­tor study.

This is one of the more re­cent de­vel­op­ments for the biotech un­der the new CEO, who re­placed for­mer chief ex­ec­u­tive Kei­th Kendall sev­er­al months ago af­ter sev­en and a half years at the helm. While Aque­s­tive said that Kendall would re­main a con­sul­tant through the end of 2022, the com­pa­ny didn’t give an ex­act rea­son for the de­par­ture at the time, on­ly not­ing that it was “un­re­lat­ed” to strat­e­gy, op­er­a­tions nor fi­nances.

AQST-109 is not the on­ly film in the biotech’s reper­toire: Oth­er films in­clude opi­oid ad­dic­tion med­i­cine com­bo buprenor­phine/nalox­one or Parkin­son’s symp­tom drug apo­mor­phine, used to treat “off episodes” in PD pa­tients.

Aque­s­tive has al­so been down a long road for its oral film to treat seizures. Orig­i­nal­ly sub­mit­ted in late 2019, the drug can­di­date was first re­ject­ed in Sep­tem­ber 2020 due to what the FDA said was low­er-than-de­sired ex­po­sure lev­els in cer­tain weight groups, per Aque­s­tive.

Af­ter the com­pa­ny re­sub­mit­ted its ap­pli­ca­tion in 2021, re­ceiv­ing a PDU­FA date for that De­cem­ber which was punt­ed again, it fi­nal­ly re­ceived ten­ta­tive ap­proval in the US just last month.

“Liber­vant has met all re­quired qual­i­ty, safe­ty, and ef­fi­ca­cy stan­dards for ap­proval but, due to an ex­ist­ing FDA reg­u­la­to­ry grant of or­phan drug mar­ket ex­clu­siv­i­ty for Val­to­co, a di­azepam nasal spray prod­uct, Liber­vant is not yet el­i­gi­ble for mar­ket­ing in the Unit­ed States,” Aque­s­tive said in a state­ment.

The FDA can­not give fi­nal ap­proval to Liber­vant un­til the or­phan drug mar­ket ex­clu­siv­i­ty pe­ri­od is over, ei­ther by nat­u­ral­ly ex­pir­ing or through oth­er op­tions like a court or­der. The com­pa­ny ex­pects to launch the prod­uct in the US mar­ket by 2027, ac­cord­ing to Aque­s­tive’s web­site.

In the mean­time, Aque­s­tive is look­ing for a mar­ket­ing part­ner to take Liber­vant out­side the US.

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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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.

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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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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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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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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AbCellera founder and CEO Carl Hansen (L) and Rallybio CEO Martin Mackay

Rally­bio, Ab­Cellera form new part­ner­ship around an­ti­bod­ies for rare dis­ease

Two biotechs that have been working heavily on different stages of antibody candidate development over the past several years are looking to work together to find potential candidates for rare diseases.

Canadian-based AbCellera and Connecticut-based Rallybio have entered a strategic partnership to find, develop and commercialize antibodies primarily for rare diseases. The multi-year, multi-target deal will seek to combine AbCellera’s antibody “discovery engine” with Rallybio’s expertise in rare diseases. However, the dollar amount for the deal was not disclosed.