Pfiz­er dumps a fad­ing pipeline star, giv­ing up on its PC­SK9 drug bo­co­cizum­ab

Paul M. Rid­ker, MD, SPIRE clin­i­cal tri­als pro­gram

You can scratch any hopes you might have had that Pfiz­er could mount a come-from-be­hind win and seize the lead from Am­gen and Sanofi/Re­gen­eron in the race of PC­SK9 cho­les­terol drugs. The phar­ma gi­ant has de­cid­ed to drop its can­di­date, bo­co­cizum­ab, cit­ing wan­ing ef­fi­ca­cy re­sults, a poor mar­ket re­cep­tion for the pi­o­neers, signs of an im­mune re­sponse to the drug and side ef­fects that would have like­ly marred its de­but.

Pfiz­er will now slam the brakes on two late-stage stud­ies — Spire-1 and Spire-2 — de­signed to test their drug in 32,000 pa­tients, a mas­sive­ly ex­pen­sive en­deav­or that went look­ing for the kinds of out­comes da­ta that could win over pay­ers. And it’s tak­ing a $0.04 R&D charge on the move.

Pfiz­er’s shares dropped 2.2% on the de­ci­sion in pre-mar­ket trad­ing Tues­day.

The move marks the ter­mi­na­tion of one of the phar­ma gi­ant’s biggest late-stage clin­i­cal ef­forts, leav­ing it still in the hunt for new drugs that can whip up some en­thu­si­asm from an­a­lysts.

Am­gen and Sanofi/Re­gen­eron sparked big hopes for their fran­chise plays in this field, bat­tling each oth­er for first-mover po­si­tion and vow­ing to jump out in front in the strug­gle to seize a mar­ket worth bil­lions. So far, though, the com­pa­nies have been bru­tal­ly dis­ap­point­ed by the ane­mic ini­tial de­mand for these ther­a­pies, as pay­ers kick back on the cost. That soft launch com­bined with ev­i­dence that Pfiz­er’s drug may not have mea­sured up well, ev­i­dent­ly played a key role in the de­ci­sion to kill it af­ter spend­ing hun­dreds of mil­lions of dol­lars on de­vel­op­ment.

Bar­clays dis­cussed it with Pfiz­er ex­ecs, and came away with this con­clu­sion:

Of note, the de­ci­sion to dis­con­tin­ue bo­co­cizum­ab was not in­flu­enced by any on­go­ing out­comes da­ta or feed­back from the da­ta mon­i­tor­ing com­mit­tee; the tri­als are still blind­ed. In our view, the cul­prit was the pres­ence of an­ti-drug an­ti­bod­ies (ADAs). Of note, from the pub­lished phase 2 da­ta from bo­co­cizum­ab, the ADA rate was ~7%.  It seemed to us that the pres­ence of ADAs in some pa­tients on bo­co­cizum­ab be­came prob­lem­at­ic, with a mi­nor­i­ty of pa­tients hav­ing a ‘sub­stan­tial lev­el’ of ADAs and a larg­er num­ber hav­ing a high­er back­ground lev­el. In our view, this like­ly wouldn’t have af­fect­ed the over­all prob­a­bil­i­ty of suc­cess in the SPIRE-1 or SPIRE-2, but it would present chal­lenges com­mer­cial­ly, where Am­gen’s Repatha or Re­gen­eron’s Pralu­ent haven’t seen an ADA rate in that range. And it does ex­plain an at­ten­u­a­tion of LDL-C ef­fect with bo­co­cizum­ab.

Pfiz­er had al­ready read out da­ta from the first 4 of 6 stud­ies in the late-stage pro­gram, reg­u­lar­ly tout­ing its ef­fi­ca­cy and pro­mot­ing its mar­ket­ing hopes in the field. Now The Med­i­cines Com­pa­ny, part­nered with Al­ny­lam, will move up to the third spot in this race with a PC­SK9 drug that a may on­ly need twice-year­ly dos­ing.

Pfiz­er has one of the weak­est late-stage pipelines in Big Phar­ma, and this set­back was not­ed by sev­er­al an­a­lysts, in­clud­ing Leerink’s Sea­mus Fer­nan­dez, who said: “This re­moves an im­por­tant Ph3 pipeline prod­uct for PFE while elim­i­nat­ing a ma­jor pot’l com­peti­tor for AMGN (MP) and SNY/REGN.”

Aside from the new­ly ac­quired PARP ta­la­zoparib, the check­point in­hibitor avelum­ab part­nered with Mer­ck KGaA and the SGLT2 di­a­betes drug er­tugliflozin part­nered with Mer­ck, Pfiz­er doesn’t have much that’s new in late-stage stud­ies. Much of its Phase III ef­fort is now giv­en to biosim­i­lars.

Pfiz­er promised to hus­tle out the da­ta on its de­funct PC­SK9 drug for fur­ther dis­cus­sion.

“To hon­or the al­tru­ism of tri­al par­tic­i­pants, and to max­i­mize the clin­i­cal and sci­en­tif­ic knowl­edge de­rived from the halt­ed tri­als, Pfiz­er has com­mit­ted to en­sur­ing that the da­ta will be made avail­able to study lead­ers for in­de­pen­dent analy­sis and prompt pub­lic pre­sen­ta­tion. We be­lieve the avail­able da­ta will al­low us to test the core sci­en­tif­ic ques­tions posed by the over­all pro­gram which is in the best in­ter­est of pa­tients who vol­un­teered in these clin­i­cal tri­als, and for pa­tients world­wide who suf­fer from heart dis­ease,” stat­ed Paul M. Rid­ker, MD, Co-chair Ex­ec­u­tive Com­mit­tee, SPIRE clin­i­cal tri­als pro­gram and di­rec­tor for Car­dio­vas­cu­lar Dis­ease Pre­ven­tion, Brigham and Women’s Hos­pi­tal.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

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: