Med­i­cines Co, Al­ny­lam gen­er­ate pos­i­tive buzz with ev­i­dence of a durable, 6-month LDL re­sponse to PC­SK9 ri­val

Clive Mean­well, CEO

The Med­i­cines Com­pa­ny $MD­CO and its part­ner Al­ny­lam $AL­NY post­ed an im­pres­sive set of 6-month re­sults for the first batch of pa­tients to reach the an­nu­al halfway mark on their new and per­haps much eas­i­er PC­SK9 drug reg­i­men. A sin­gle 300 mg in­jec­tion of their RNAi drug — now dubbed in­clisir­an — reg­is­tered a mean 43% re­duc­tion in bad LDL cho­les­terol af­ter 180 days, trig­ger­ing some ex­cit­ed buzz among the an­a­lysts fol­low­ing this pro­gram.

The Med­i­cines Com­pa­ny has at­tract­ed some con­sid­er­able en­thu­si­asm for a cho­les­terol drug that might on­ly be need­ed to be dosed two or three times a year—high­light­ing a drug that could well slap down ap­proved ther­a­pies from Am­gen and Sanofi/Re­gen­eron. So when they turned up with a late-break­er at the Amer­i­can Heart As­so­ci­a­tion Sci­en­tif­ic Ses­sions 2016 in New Or­leans, an­a­lysts were ready to clap at the first sol­id sign of dura­bil­i­ty from the first 189 sub­jects in the 497-pa­tient study.

Two 300 mg in­jec­tions of the drug trig­gered a 57% mean drop in LDL on day 120 and 52% by day 180.

“To­geth­er these da­ta sup­port fea­si­bil­i­ty for tri-an­nu­al and po­ten­tial­ly bi-an­nu­al dos­ing,” wrote Gena Wang, an an­a­lyst at Jef­feries.

Umer Raf­fat from Ever­core ISI not­ed:

Ph 2 PC­Sk9 da­ta pre­sent­ed at AHA just now looks very con­sis­tent and durable through day 180 (pts get­ting sin­gle in­jec­tion had -43% LDL re­duc­tion at day 180).

I am get­ting sev­er­al ques­tions on 1 death in the 500 mg arm:  it was in a pa­tient with ex­ten­sive CAD his­to­ry and mul­ti­ple CV events in the past (note that ex­clu­sion cri­te­ria on­ly se­lect­ed out pts with MACE event with­in last 6 months).  Al­so, 1 case of ALT>3ULN oc­curred in a pa­tient es­ca­lat­ing dose of statin.”

Both Am­gen as well as the team from Sanofi/Re­gen­eron have been forced to lay out dis­ap­point­ing launch­es for their PC­SK9 of­fer­ings, both tapped as like­ly block­busters in the mak­ing. The cost, as well as ques­tions of the un­der­ly­ing health ben­e­fits, have con­spired to lim­it their ini­tial use.

The dream sce­nario from Clive Mean­well, the CEO:

“We will fo­cus our re­sources on in­clisir­an for ag­gres­sive Phase 3 de­vel­op­ment to en­sure that this promis­ing agent is in­ves­ti­gat­ed thor­ough­ly and rapid­ly in Phase III, sub­mit­ted to world­wide reg­u­la­to­ry agen­cies and, if ap­proved, made avail­able to mil­lions of at-risk, of­ten non-ad­her­ent, pa­tients world­wide who con­tin­ue to grap­ple with the re­al­i­ties and risks of high LDL-C.”

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.

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.