Bio­gen, Ei­sai BACE drug cuts amy­loid be­ta in the brain, but there’s no hard da­ta to show it does any­thing im­por­tant for cog­ni­tion

Just af­ter Mer­ck $MRK re­search raised se­ri­ous doubts about the BACE ap­proach to tack­ling Alzheimer’s dis­ease, re­searchers for Bio­gen $BI­IB and Ei­sai are fight­ing against the tide this week. And they have a long way to go be­fore they start per­suad­ing they may have some­thing of val­ue for pa­tients.

Lynn Kramer

The team fo­cused on the oral BACE drug elen­be­ce­s­tat says the drug is do­ing what it’s in­tend­ed to do, cut­ting down on the amount of amy­loid be­ta in the brains of pa­tients with a mild form of the dis­ease and pro­duc­ing a nu­mer­i­cal de­cline in the rate at which pa­tients’ cog­ni­tion spi­rals down­ward.

But …. the re­searchers were un­able to track any­thing in the 18-month Phase II study that qual­i­fied as a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment on cog­ni­tion scores. Ac­tu­al da­ta were be­ing held back, so we don’t know why ex­act­ly the team con­cludes that the re­sponse sug­gests they may have seen “a de­lay of clin­i­cal symp­tom de­cline in ex­plorato­ry end­points.”

Re­searchers used both the Clin­i­cal De­men­tia Rat­ing Sum of Box­es as well as a new as­sess­ment they’ve been work­ing on.

This wouldn’t be the first time re­searchers tout­ed/hyped a pos­si­ble tie to an im­proved cog­ni­tive score. Eli Lil­ly did it with great suc­cess on the sec­ond piv­otal fail for solanezum­ab, set­ting up a third late-stage study that com­plete­ly failed to show an im­prove­ment in cog­ni­tion or func­tion. Now they’re try­ing it in pre-symp­to­matic pa­tients. And Bio­gen it­self has reg­u­lar­ly stoked hopes for ad­u­canum­ab, its top late-stage drug in the Alzheimer’s field, where noth­ing has worked out in more than a decade.

Sig­nif­i­cant­ly, the re­searchers found no se­ri­ous safe­ty is­sues that could stop the pro­gram at this stage.

The lat­est set­back for Mer­ck al­so un­der­scored fears that the en­tire BACE field is head­ed for the grow­ing scrap heap of drugs — as far as symp­to­matic pa­tients are con­cerned. Mer­ck re­searchers con­clud­ed that their first Phase III tri­al al­so proved that their drug hit its tar­get, with­out sig­nif­i­cant­ly help­ing pa­tients. There have been a num­ber of oth­er BACE fail­ures, but those set­backs have been blamed on the drug’s weak­ness­es, rather than prob­lems with the tar­get.

“It is high­ly en­cour­ag­ing that Study 202 con­firmed elen­be­ce­s­tat’s treat­ment ef­fect in re­duc­ing amy­loid in the brain and sug­gest­ed a slow­ing of clin­i­cal de­cline. Ei­sai and Bio­gen will con­tin­ue to work to­geth­er to ad­vance the on­go­ing Phase III pro­gram (MIS­SION AD) in or­der to con­tribute a new po­ten­tial treat­ment op­tion to Alzheimer’s dis­ease pa­tients as soon as pos­si­ble,” said Lynn Kramer, the CMO for Ei­sai’s Neu­rol­o­gy Busi­ness Group. 

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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