Top 10 pipeline blowups, set­backs and sna­fus in H1 2017

As Big Phar­ma and the grow­ing biotech sec­tor shift­ed their R&D sights over the past decade — fo­cus­ing more on game-chang­ing drugs and shun­ning the me-too va­ri­ety that don’t fare well with pay­ers — the usu­al pit­falls, set­backs and sna­fus have grown more spec­tac­u­lar with each pass­ing year. You have to break it down in­to 6-month al­lot­ments just to keep a han­dle on the con­se­quences of ma­jor break­downs.

This list, though, al­so rais­es ques­tions about the role the FDA has come play in rais­ing risks for pa­tients in can­cer stud­ies. Two of my 10 drugs on the hit list are on­col­o­gy drugs that were put on hold — briefly — on­ly to get yanked again af­ter more deaths hit the drug arm of the piv­otal study. Is the FDA do­ing its job prop­er­ly in eval­u­at­ing the risks as­so­ci­at­ed with ex­per­i­men­tal can­cer drugs? Or has the rush to get new ap­provals caused reg­u­la­tors to low­er safe­ty stan­dards too far?

The FDA has re­ceived con­sid­er­able praise for the way it’s carved a short path­way for ac­cel­er­at­ed can­cer drug ap­provals. So it’s no sur­prise that no one is even ask­ing that ques­tion.

I’ve added a new cat­e­go­ry of a bio­phar­ma im­plo­sion: the self-in­flict­ed pric­ing scan­dal. It did in one com­pa­ny on this list, and the same pit­fall awaits oth­ers. And I’m al­so adding a line on how the com­pa­ny’s stock did year-to-date. The Dow Jones, of­fered in con­text, is up 9% from the be­gin­ning of this year.

The nasty sur­pris­es out­lined be­low are an­oth­er re­minder of the big gam­bles every drug de­vel­op­er is en­gaged in. This isn’t a job for the weak of heart. And too much hubris is even worse.

1
Eli Lil­ly
Baric­i­tinib goes from block­buster con­tender to dog­house ten­ant in a sud­den and dev­as­tat­ing fall from grace

Based: In­di­anapo­lis, IN
$LLY:+3% YTD
CEO: David Ricks

The scoop: By all ac­counts, Eli Lil­ly’s NDA for its block­buster con­tender baric­i­tinib looked like a slam dunk. The phar­ma gi­ant had high­light­ed sol­id ef­fi­ca­cy da­ta and a safe­ty pro­file with­out set­ting off any alarms. So when the FDA’s re­jec­tion was an­nounced in April, the news took every­one by sur­prise. It took awhile, but Lil­ly even­tu­al­ly re­vealed that ev­i­dence of blood clots in two of sev­en stud­ies — Lil­ly is noth­ing if not com­plete­ly thor­ough in its de­vel­op­ment work — forced reg­u­la­tors to throw the red flag. The de­lay on new safe­ty da­ta could now take three or four years to nail down, leav­ing Lil­ly look­ing at a very late come­back at­tempt af­ter a new round of en­tries from its ri­vals. It’s a big de­ci­sion, and an aw­ful place to be in for new CEO Dave Ricks, who’s been si­mul­ta­ne­ous­ly re­jig­ging the pipeline. Lil­ly, though, has a habit of stick­ing with its late-stage drugs if there’s any chance of suc­cess. Lil­ly’s R&D group ran three failed late-stage stud­ies on their Alzheimer’s drug solanezum­ab, and they still have clin­i­cal work un­der­way on it. The phar­ma gi­ant went through one of the worst dry spells in the in­dus­try, which forced a pledge on a string of new ap­provals. And re­cent­ly they got hand­ed a beat­ing in a Phase III head-to-head be­tween its new di­a­betes drug Trulic­i­ty and No­vo’s semaglu­tide. The last thing Ricks needs now is a re­turn to the old, bad days of R&D set­backs.

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