Mer­ck just dashed the last re­main­ing hopes for its in­dus­try-lead­ing BACE drug for Alzheimer's

The Alzheimer’s field  is keep­ing its per­fect record for fail­ure in piv­otal Phase III stud­ies.

Mer­ck to­day scrapped the last re­main­ing Phase III study of its lead­ing BACE ther­a­py verube­ce­s­tat, the sec­ond of two col­lapsed stud­ies, af­ter in­de­pen­dent mon­i­tors con­clud­ed that the drug wasn’t work­ing for ear­ly-stage, pro­dro­mal pa­tients.

Roger Perl­mut­ter

Back in Feb­ru­ary Mer­ck sig­naled its lack of suc­cess with the drug, which moves up­stream to re­duce the pro­duc­tion of amy­loid be­ta, when it punt­ed its Phase III tri­al in mild-to-mod­er­ate pa­tients.

Once one of Mer­ck’s top late-stage drugs in the pipeline, its fail­ure here will like­ly cast a dark shad­ow over oth­er BACE drugs, in­clud­ing the one that Eli Lil­ly ob­tained from As­traZeneca as the UK gi­ant backed out of CNS dis­ease R&D.

Mer­ck had lit­tle to say in de­tail Tues­day evening. A spokesper­son not­ed that the com­pa­ny had just re­ceived the news and it would now have to move to shut­ter the work and be­gin the process of an­a­lyz­ing the re­sults. And now the an­a­lysts are writ­ing it off. Not­ed Leerink’s Sea­mus Fer­nan­dez:

While a dis­ap­point­ing de­vel­op­ment, we had al­ways viewed this tri­al as a high risk en­deav­or giv­en the pre­vi­ous sus­pen­sion of the Phase 2/3 EPOCH study in mild-to-mod­er­ate AD pa­tients, along with his­toric fail­ures of oth­er drugs in this dis­ease. Nev­er­the­less, we had in­clud­ed $750M in peak 2026E prob­a­bil­i­ty-ad­just­ed sales for the drug in our mod­el. Re­mov­ing this brings our price tar­get to $66/shr (from $67/shr).

Alzheimer’s re­search has been an un­re­lent­ing dis­as­ter zone for 15 years, with noth­ing avail­able to treat this dis­ease aside from some mar­gin­al symp­to­matic ther­a­pies. Pfiz­er just aban­doned neu­ro­sciences en­tire­ly, drop­ping its Alzheimer’s work along the way. Yet Mer­ck and most of the rest of the play­ers aren’t step­ping back. J&J, which had one of the biggest fail­ures in the field, is forg­ing ahead with gene ther­a­pies and oth­er tech­nolo­gies. De­nali pulled off a record IPO last year promis­ing a new ap­proach to con­quer Alzheimer’s. And Mer­ck is press­ing ahead on tau, the oth­er tox­ic pro­tein of­ten fig­ured as a trig­ger for the mem­o­ry-wast­ing ail­ment.

“We are dis­ap­point­ed with this out­come, es­pe­cial­ly giv­en the lack of treat­ment op­tions for pa­tients suf­fer­ing from Alzheimer’s dis­ease,” said Roger Perl­mut­ter, pres­i­dent, Mer­ck Re­search Lab­o­ra­to­ries. “We are grate­ful to the pa­tients and care­givers who par­tic­i­pat­ed in this study, and de­spite this out­come, Mer­ck re­mains com­mit­ted to de­vel­op­ing nov­el ther­a­pies for the treat­ment of Alzheimer’s and oth­er neu­rode­gen­er­a­tive dis­eases.”

UP­DAT­ED: Roche bags 'break­through' an­ti-fi­bro­sis drug in $1.4B biotech buy­out deal

Roche is snapping up a “breakthrough” anti-fibrotic drug in a $1.4 billion buyout.

The pharma giant announced Friday that it is acquiring Promedior, primarily to get its hands on PRM-151, a recombinant form of human pentraxin-2 (PTX-2) protein that has nailed down mid-stage clinical data on idiopathic pulmonary fibrosis and demonstrating its potential for a range of fibrotic conditions.

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Amarin emerges from an ex­pert pan­el re­view with a clear en­dorse­ment for Vas­cepa and high odds of suc­cess when the FDA weighs in for­mal­ly

Several FDA experts who gathered Thursday to consider the landmark approval of Vascepa to reduce cardio events in an at-risk population voiced their unease about various aspects of the efficacy and safety data, or ultimately the population it should be used to treat. But the overwhelming belief that the data pointed to the drug’s benefit and clearly outweighed risks carried the day for Amarin.

The panel voted unanimously (16 to 0) to support the company’s positive data presentation — backing an OK for expanding the label to include reducing cardio risk. The vote points Amarin $AMRN down a short path to a formal decision by the FDA, with the odds heavily in its favor. Chances are the rest of the questions about the future of this drug will be hashed out in the label’s small print.

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Federal Trade Commission commissioner Rohit Chopra testifies on Capitol Hill (AP Photo/Susan Walsh)

FTC clears Bris­tol-My­ers’ $74B deal to buy Cel­gene — but Dems sig­nal a po­ten­tial hard shift against Big Phar­ma M&A

Bristol-Myers Squibb’s record $74 billion takeover of Celgene is a done deal. And it will all be over — except for the lingering complaints from die-hard Celgene investors — on Wednesday.

Like much else that’s going on in Washington these days, the vote among the 5 FTC commissioners split along party lines, with the 3 Republicans voting to clear the way and the 2 Democrats steamed over what they see as a major M&A move that will lessen competition and innovation. And that split has big implications for the M&A side of the business if the Dems take the White House in 2020.

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No­var­tis scores its lat­est FDA OK — this time for a new sick­le cell dis­ease drug picked up in a $665M deal

Novartis’ decision to buy Oklahoma-based biotech Selexys 3 years ago for up to $665 million has paid off with an FDA approval today.

Blessed with the FDA’s breakthrough drug designation for a speedy review, the pharma giant has pinned down an approval for crizanlizumab, a new therapy designed to reduce the frequency of painful incidents of vaso-occlusive crises among sickle cell disease patients 16 or older.

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No­var­tis spin­out’s first an­ti-ag­ing PhI­II is a flop, so now they’ll turn to Parkin­son’s chal­lenge as shares wilt

Novartis spinout resTORbio is grappling with the collapse of its lead clinical program this morning — an anti-aging R&D failure that will badly damage their rep in the field.

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BeiGene CEO John Oyler at an Endpoints event in Shanghai, October 2018 (Credit: Endpoints News/PharmCube)

UP­DAT­ED: In a first, FDA green-lights use of a Chi­nese built can­cer ther­a­py — and more are com­ing

Weeks after Amgen took a $2.7 billion stake in BeiGene, the Beijing-based biotech has secured its first-ever FDA approval for zanubrutinib, a BTK inhibitor, months ahead of schedule.

BeiGene’s drug, branded as Brukinsa, has secured accelerated approval for adult patients with mantle cell lymphoma (MCL) — a typically aggressive, rare, form of blood cancer — who have received at least one prior therapy.

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What does $62B buy you these days? A lot, says Take­da ex­ecs as the phar­ma play­er promis­es a block­buster R&D fu­ture

First comes the $62 billion buyout. Then comes the asset auction and reorganization to pay down debt. Now comes the detailed pledge of a bigger, brighter future in drug development.

That’s where Takeda finds itself on R&D day today, about 11 months after closing on their Shire acquisition. R&D chief Andy Plump is joining CEO Christophe Weber and other top members of the team to outline a new set of priorities in the greatly expanded pipeline at Takeda, which has jumped into the top ranks of the world’s pharma giants in the wake of the Shire deal.

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GSK's asth­ma bi­o­log­ic Nu­cala scores in rare blood dis­or­der study

GlaxoSmithKline’s asthma drug Nucala, which received a resounding FDA rejection for use in chronic obstructive pulmonary disease (COPD) last year, has shown promise in a rare blood disorder.

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Mer­ck buys a fledg­ling neu­rode­gen­er­a­tive biotech spawned by an old GSK dis­cov­ery al­liance. What’s up with that?

Avalon Ventures chief Jay Lichter has a well-known yen for drug development programs picked up in academia. And what he found in Haoxing Xu’s lab at the University of Michigan pricked his interest enough to launch one of his umbrella biotechs in San Diego.

Xu’s work laid the foundation for Avalon to launch Calporta, which has been working on finding small molecule agonists of TRPML1 (transient receptor potential cation channel, mucolipin subfamily, member 1) for lysosomal storage disorders. And that pathway, they believe, points to new approaches on major market neurodegenerative diseases like Parkinson’s, ALS and Alzheimer’s.

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