Watch out Al­ny­lam/Io­n­is: Pfiz­er’s pos­i­tive PhI­II tafamidis da­ta makes drug a new ri­val

Pfiz­er $PFE re­port­ed out some good news from a late-stage tri­al test­ing its drug tafamidis against a rare con­di­tion that leads to heart fail­ure. The pos­i­tive re­sults of the Phase III study have both Al­ny­lam $AL­NY and Io­n­is $IONS in­vestors squea­mish, as it could be com­pe­ti­tion for patisir­an and in­ot­ersen.

Bren­da Coop­er­stone

The Pfiz­er tri­al, called AT­TR-ACT, test­ed its drug tafamidis in pa­tients with transthyretin car­diomy­opa­thy, an in­her­it­ed con­di­tion in which pro­teins don’t fold in­to their nor­mal shapes cor­rect­ly. The mis­fold­ing makes them build up and cause dam­age to the nerves, heart, and oth­er or­gans. In the tri­al, the drug met its pri­ma­ry end­point, Pfiz­er said, and demon­strat­ed a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion in the com­bi­na­tion of all-cause mor­tal­i­ty and fre­quen­cy of car­dio­vas­cu­lar-re­lat­ed hos­pi­tal­iza­tions com­pared to place­bo at 30 months.

Fol­low­ing news on the da­ta, Al­ny­lam stock fell 5%, po­ten­tial­ly on fear of an up­com­ing ri­val as the phar­ma com­pa­ny is al­so pur­su­ing a car­diomy­opa­thy ap­pli­ca­tion for patisir­an. And Io­n­is, which owns a ma­jor­i­ty stake in its spin­out Akcea (which is de­vel­op­ing in­ot­ersen for the same con­di­tion), al­so sank 5%.

Pfiz­er land­ed tafamidis when it ac­quired Fol­dRx in a buy­out back in 2010 as it pushed in­to the rare dis­ease field. Like oth­er phar­ma com­pa­nies, Pfiz­er was lured by the prospect of big re­turns for drugs that meet an ur­gent need in tiny pa­tient pop­u­la­tions.

Pfiz­er test­ed the drug in a rare neu­rode­gen­er­a­tive dis­ease called TTR-FAP, but got re­ject­ed by the FDA in 2012 af­ter try­ing to gain ap­proval with on­ly one tri­al as proof of the drug’s ef­fi­ca­cy. Tafamidis was ap­proved in 40 coun­tries — in­clud­ing the EU in 2011 — how­ev­er. It sells un­der the brand name Vyn­daquel.

In a state­ment ear­li­er this year, Pfiz­er said it’s still work­ing on a way for­ward in the US for tafamidis in TTR-FAP.

“As a leader in TTR amy­loi­do­sis, Pfiz­er Rare Dis­ease con­tin­ues to part­ner with the FDA re­gard­ing a po­ten­tial path to ap­proval for tafamidis for TTR-FAP, as we hope to achieve the ob­jec­tive of pro­vid­ing TTR-FAP pa­tients liv­ing in the Unit­ed States with the same treat­ment op­tion as those pa­tients liv­ing in many oth­er parts of the world.”

For now, tack­ling transthyretin car­diomy­opa­thy gives the drug a new av­enue for rev­enue gen­er­a­tion in the US. Bren­da Coop­er­stone, SVP and chief de­vel­op­ment of­fi­cer in Pfiz­er’s rare dis­ease unit, had this to say about the re­cent Phase III da­ta:

These topline re­sults are im­por­tant for peo­ple with transthyretin car­diomy­opa­thy and bring us one step clos­er to re­al­iz­ing the po­ten­tial for a new treat­ment for those in des­per­ate need. Pfiz­er Rare Dis­ease has been at the fore­front of im­prov­ing the un­der­stand­ing of transthyretin car­diomy­opa­thy, and we thank the pa­tients who par­tic­i­pat­ed in the tri­al and their fam­i­lies, as well as the physi­cians and in­ves­ti­ga­tion­al sites that con­tributed to this im­por­tant study. We look for­ward to shar­ing the de­tailed re­sults of the study with the car­dio­vas­cu­lar com­mu­ni­ty and dis­cussing these da­ta with health au­thor­i­ties to de­ter­mine an ap­pro­pri­ate reg­u­la­to­ry path for­ward.

 

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.

Big week for Alzheimer’s da­ta; As­traZeneca buys cell ther­a­py start­up; Dig­i­tal ther­a­peu­tics hits a pay­er wall; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

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Am­gen, years be­hind ri­vals, says PhI obe­si­ty drug shows dura­bil­i­ty signs

While NBC ran “The Biggest Loser” for 17 seasons, deemed toxic by critics for the reality show’s punishing exercise and diet upheavals, researchers in pharmaceutical labs have been attempting to create prescription drugs that induce weight loss — and one pharma betting it can require less frequent dosing is out with a new crop of data.

Amgen was relatively late to the game compared to its approved competitor Novo Nordisk and green light-approaching rival Eli Lilly. But early data suggested Amgen’s AMG 133 led to a 14.5% weight reduction in the first few months of dosing, buoying shares earlier this fall, and now the California pharma is out with its first batch of durability data showing that figure fell slightly to 11.2% about 150 days after the last dose. Amgen presented at the 20th World Congress on Insulin Resistance, Diabetes & Cardiovascular Disease on Saturday afternoon.

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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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US month­ly costs for biosim­i­lars 'sub­stan­tial­ly high­er' than Ger­many or Switzer­land, JA­MA re­search finds

As the global biologics market is expected to hit nearly the half-trillion-dollar mark this year, new JAMA research points to the importance of timely biosimilar entry, particularly as fewer biosimilars are entering the US than in Europe, and as monthly treatment costs for biosimilars were “substantially higher” in the US compared with Germany and Switzerland.

Among the three countries, biosimilar market share at launch was highest in Germany, but increased at the fastest rate in the US, the authors from the University of Zurich’s Institute of Law wrote in JAMA Network Open today.

Kirk Myers is shown in a still image from a new film series showcasing the efforts of HIV advocates funded by Gilead.

Gilead spot­lights HIV projects and the com­mu­ni­ty lead­ers dri­ving them in new mi­ni-doc­u­men­tary films

Gilead is going behind the scenes of some of the HIV initiatives it funds through grants in a new film series narrated by the people helming the projects.

The first four films and leaders come from across the US — Arianna Lint in Florida and Puerto Rico, Cleve Jones in San Francisco, June Gipson in Mississippi and Kirk Myers in Texas. Their HIV-focused efforts range from addressing unmet needs of the transgender community to delivering social services and high-quality health care in underserved communities.

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EMA pulls an opi­oid from the 1950s used to treat dry cough

The European Medicines Agency said Friday that it’s pulling from all European markets pholcodine-containing medicines, which are an opioid used in adults and children for the treatment of dry cough and in combo with other drugs as a treatment for cold and flu.

The decision to pull the medicines comes as the EMA points to the results from the recent ALPHO study, which show that use of pholcodine during the 12 months preceding anesthesia is linked to a risk of an anaphylactic reaction related to the neuromuscular blocking agents (NMBAs) used (with an adjusted OR of 4.2, and a 95% confidence interval of 2.5 to 6.9).

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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