FDA slaps a par­tial hold on one of As­traZeneca's cru­cial dur­val­um­ab pro­grams

Shares of As­traZeneca $AZN start­ed to slide this morn­ing as the phar­ma gi­ant con­firmed that the FDA has placed a par­tial clin­i­cal hold on a Phase III re­search pro­gram in­volv­ing its check­point in­hibitor dur­val­um­ab, halt­ing new en­roll­ment.

The par­tial hold was in­sti­tut­ed af­ter in­ves­ti­ga­tors tracked bleed­ing events in a Phase III com­bo study for dur­val­um­ab, its top in­ves­ti­ga­tion­al drug, with treme­li­mum­ab for head and neck can­cer. But the com­pa­ny cau­tions that all oth­er stud­ies are pro­ceed­ing as planned, not­ing that such bleed­ing events are not un­usu­al in head and neck can­cer.

That said, As­traZeneca can ill af­ford any de­lays in the de­vel­op­ment of dur­val­um­ab now. The com­pa­ny al­ready de­layed its de­vel­op­ment time­line for the check­point in­hibitor to al­low for com­bi­na­tion stud­ies, which As­traZeneca is count­ing on to make a splash with a late ar­rival in the field.

A set­back here will raise fresh ques­tions for As­traZeneca as it at­tempts to play catch-up to the mar­ket lead­ers in check­point in­hibitors. Dur­val­um­ab is now a dis­tant fourth — or fifth — in the mar­ket, like­ly to fol­low Roche’s $RHH­BY Tecen­triq and well be­hind Mer­ck ($MRK/Keytru­da) and Bris­tol-My­ers Squibb $BMY, which has racked up block­buster sales with Op­di­vo.

As­traZeneca’s shares are down 5%.

The news about the safe­ty is­sue has been per­co­lat­ing in­side As­traZeneca for more than a month.

In a fol­lowup to a query, As­traZeneca said that they took the first step in paus­ing en­roll­ment in mid-Sep­tem­ber, more than a month ago, then fol­lowed up with no­tices to reg­u­la­tors and in­ves­ti­ga­tors. Ac­cord­ing to a spokesper­son:

The FDA re­cent­ly con­firmed their agree­ment with our ac­tion and placed a par­tial clin­i­cal hold on re­cruit­ment of new pa­tients while we com­plete our analy­sis. We sent an up­date to Clin­i­cal­Tri­als.gov with­in the usu­al time­frames. Our analy­sis is now com­plete and has been sub­mit­ted to FDA and un­der their re­view; we hope to re­sume [en­roll­ment] as soon as pos­si­ble.

Here’s the full ini­tial state­ment sent to End­points News:

Fol­low­ing the re­cent up­date on clin­i­cal­tri­als.gov, As­traZeneca con­firms that the US FDA has placed a par­tial clin­i­cal hold on the en­roll­ment of new pa­tients with head and neck squa­mous cell car­ci­no­ma (HN­SCC) in clin­i­cal tri­als of dur­val­um­ab as monother­a­py and in com­bi­na­tion with treme­li­mum­ab or oth­er po­ten­tial med­i­cines. All tri­als are con­tin­u­ing with ex­ist­ing pa­tients.

The par­tial clin­i­cal hold on new pa­tient [en­roll­ment] re­lates on­ly to head and neck can­cer. Tri­als for dur­val­um­ab in dif­fer­ent can­cer types, as monother­a­py or in com­bi­na­tion with treme­li­mum­ab or oth­er po­ten­tial med­i­cines, are pro­gress­ing as planned, with piv­otal da­ta in lung can­cer an­tic­i­pat­ed in the first half of 2017.

The FDA’s de­ci­sion fol­lows vol­un­tary ac­tion by As­traZeneca to pause [en­roll­ment] of new HN­SCC pa­tients while a de­tailed analy­sis is con­duct­ed of ad­verse events re­lat­ed to bleed­ing that were ob­served as part of rou­tine safe­ty mon­i­tor­ing of the Phase III KESTREL and EA­GLE tri­als. Bleed­ing is a known com­pli­ca­tion in treat­ments of head and neck can­cers pri­mar­i­ly due to the na­ture of the un­der­ly­ing dis­ease, the prox­im­i­ty of tu­mours to ma­jor blood ves­sels and use of pri­or can­cer ther­a­pies, which may in­volve surgery and ra­di­a­tion.

As­traZeneca has sub­mit­ted its analy­sis of the ob­served bleed­ing events to the FDA for re­view and is work­ing close­ly with the Agency, pro­vid­ing the re­quired in­for­ma­tion to re­sume new pa­tient [en­roll­ment] as soon as pos­si­ble.

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.

Thomas Gad, Y-mAbs Therapeutics founder and interim CEO

FDA re­jects Y-mAbs’ neu­rob­las­toma drug af­ter tak­ing is­sue with clin­i­cal tri­al de­sign

Uncertainty about clinical trial evidence has led the FDA to hand down a complete response letter for Y-mAbs’ neuroblastoma drug, casting a cloud on the future of a candidate that had gone through a long development journey in a rare pediatric cancer.

Y-mAbs said it’s disappointed “but not surprised” given that the agency’s oncology drug advisory committee had voted 16-0 against its drug’s approval a few weeks ago.

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.

Tim Pearson, Carrick Therapeutics CEO

Pfiz­er backs $60M in­fu­sion in­to Car­rick, teams up on breast can­cer treat­ment

In a big week for Carrick Therapeutics, the company announced $60 million in funding for its lead breast cancer drug and development of a second program, as well as a collaboration with Pfizer for combo development.

The $35 million from Pfizer comes with an agreement under which Pfizer will support Carrick’s Phase II study of samuraciclib in combination with Pfizer’s Faslodex for advanced breast cancer. Along with the investment, Adam Schayowitz, vice president and development head of breast cancer, colorectal cancer and melanoma at Pfizer global product development, will join Carrick’s scientific advisory board.

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.

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.

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.

Paul Hudson, Sanofi CEO (Romuald Meigneux/Sipa via AP Images)

Sanofi and Am­gen are 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.” And Amgen CEO Robert Bradway came right in behind him, filing a statement on the London Stock Exchange overnight that any offer they may make will “likely” be in cash as well.

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.

Philip Tagari switch­es Am­gen's dis­cov­ery lab for in­sitro's ma­chine learn­ing tools; CEO Joaquin Du­a­to to chair J&J's board

In February, Philip Tagari will take a few days of retirement and then immediately return to industry. He won’t be leading the therapeutics discovery unit for a large biopharma, though.

He’ll trade in his Amgen hat for chief scientist at a machine learning startup that has reeled in hundreds of millions in capital to lay the groundwork for a much-hyped new model of drug discovery that aims to speed up the time to new clinical assets.

Raul Rodriguez, Rigel Pharma CEO

Rigel Phar­ma scores FDA ap­proval for leukemia, kick­ing off show­down with Servi­er in IDH1

When Rigel Pharma bought olutasidenib from Forma Therapeutics, it acquired a drug that already secured a PDUFA date at the FDA — for February 2023. But regulators are ready to give their OK sooner than that.

The FDA has approved the IDH1 inhibitor as a treatment for adult patients with relapsed or refractory acute myeloid leukemia who have a susceptible IDH-1 (isocitrate dehydrogenase-1) mutation as detected by an FDA-greenlit test. Rigel will market it as Rezlidhia.

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.