As­traZeneca's star res­pi­ra­to­ry drug runs in­to an­oth­er wall with COPD, but it's not giv­ing up — yet

As­traZeneca’s at­tempt to push its res­pi­ra­to­ry bi­o­log­ic Fasen­ra (ben­ral­izum­ab) in­to the chron­ic ob­struc­tive pul­monary dis­ease field has been foiled by a Phase III flop.

Sean Bo­hen

In the 56-week mul­ti-cen­ter tri­al, Fasen­ra failed to spur a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion of ex­ac­er­ba­tions in pa­tients with mod­er­ate to very se­vere COPD, the pri­ma­ry end­point, com­pared to place­bo.

Cur­rent­ly ap­proved as an add-on treat­ment for se­vere eosinophilic asth­ma in the US, EU, Japan and sev­er­al oth­er coun­tries, Fasen­ra was billed as a block­buster in the mak­ing, with CEO Pas­cal So­ri­ot es­ti­mat­ing peak sales po­ten­tial at $2 bil­lion (Jef­feries an­a­lysts gave it a more mod­est $1.5 bil­lion).

To achieve that, As­traZeneca need­ed to nav­i­gate a mar­ket al­ready staked by Glax­o­SmithK­line’s first-in-class Nu­cala, Te­va’s Cinqair, and No­var­tis’ Xo­lair. It’s too ear­ly to tell whether its com­bi­na­tion of low­er pric­ing, dos­ing fre­quen­cy and de­liv­ery method was enough to start win­ning over large num­bers of physi­cians and pa­tients, and its more en­trenched ri­vals are plan­ning moves of their own. Last Sep­tem­ber, GSK fol­lowed up with an NDA for Nu­cala in COPD — though the case was al­so built up­on shaky da­ta heav­i­ly re­liant on a bio­mark­er.

As­traZeneca went in­to the Phase III Galathea study (as well as an­oth­er one dubbed Ter­ra­no­va) de­spite con­ced­ing de­feat in a PhI­Ia study back in 2014, where Fasen­ra not on­ly failed to im­prove the rate of acute ex­ac­er­ba­tions for se­vere COPD pa­tients but al­so saw a high­er rate of treat­ment-emer­gent ad­verse events. Ex­ecs at the UK phar­ma gi­ant and its bi­o­log­ics unit Med­Im­mune were bet­ting that bet­ter tri­al de­sign and pa­tient se­lec­tion would de­liv­er a des­per­ate­ly need­ed win in this key dis­ease tar­get, but it proved elu­sive.

That said, no new safe­ty or tol­er­a­bil­i­ty is­sues were re­port­ed in this tri­al — per­haps the on­ly good news in the an­nounce­ment.

While the team is still crunch­ing the num­bers for an up­com­ing med­ical meet­ing, CMO Sean Bo­hen has this to say:

COPD is a de­bil­i­tat­ing dis­ease with sig­nif­i­cant un­met need among pa­tients whose dis­ease re­mains un­con­trolled de­spite treat­ment with ex­ist­ing in­haled ther­a­pies. We will now await the re­sults of TER­RA­NO­VA and a full eval­u­a­tion of both tri­als to de­ter­mine next steps for Fasen­ra in COPD.

Ter­ra­no­va is sched­uled to read out this quar­ter.

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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Paul Hudson, Sanofi CEO (ROMUALD MEIGNEUX/Sipa via AP Images)

Sanofi's Paul Hud­son is bring­ing cash to cov­er the ta­ble stakes for the Hori­zon M&A game

With the market cap on Horizon Therapeutics $HZNP pushed up to the $23 billion mark today, one of the Big Pharmas in the hunt for a major league buyout deal signaled it’s playing the M&A game with cash.

Paris-based Sanofi, where CEO Paul Hudson has been largely focused on some risky biotech acquisitions to win some respect for its future pipeline prospects, issued a statement early Friday — complying with rule 2.12 of the Irish takeover rules — making clear that while the certainty or size of an offer can’t be determined, any offer “will be solely in cash.”

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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: