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.”

Andre Kalil, AP Images

A 9/11-era Om­a­ha fa­cil­i­ty, an old Ebo­la drug, and the ubiq­ui­tous Dr. Fau­ci: In­side the first US nov­el coro­n­avirus tri­al

The first 11 coronavirus patients who arrived in Omaha last week, airlifted across the globe after two weeks quarantined on a cruise ship, showed only minor symptoms or none at all. And then one of them — or one of the couple of Americans who arrived later — got worse. He developed pneumonia, a life-threatening complication for coronavirus patients.

In a biocontainment room at the University of Nebraska Medical Center on Friday, doctors infused him with an experimental Gilead drug once developed for Ebola, called remdesivir. Or they gave him a placebo. For the first time in the US, neither he nor the doctors knew.

The first US novel coronavirus trial was underway and with it, a mad dash for an answer. Sponsored by the NIH, the study marked a critical point in the epidemic. Since the start of the outbreak, the agency had helped lead a global effort to contain the virus. Now, as it spread worldwide and the CDC issued warnings the US could see a major internal outbreak, they were looking at home.

“We don’t have too much time,” Andre Kalil, the trial’s lead investigator, told Endpoints News. “Everything’s moving really fast.”

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Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Take­da swoops in to buy lit­tle biotech part­ner and its celi­ac drug poised to 'change stan­dard of care'

Having spent three years carefully grooming PvP Biologics and its drug for celiac disease, Takeda is happy enough with the proof-of-concept data to buy it all.

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Dan O'Day (AP Images)

A name emerges out of the Gilead M&A ru­mor mill, and it’s a can­cer biotech

After months of questions and speculation about when and if Gilead will make a major acquisitions, a name has emerged.

The California-based drugmaker has approached Forty Seven Inc, a cancer biotech, with a takeover offer, Bloomberg News reports. With Forty Seven’s market cap at $2.3 billion, an acquisition would likely be Gilead’s largest since they acquired Kite Pharma for $11.9 billion in 2017.

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Biogen head of R&D Al Sandrock, Sangamo CEO Sandy Macrae

UP­DAT­ED: Bio­gen makes an­oth­er bold Alzheimer’s bet, drop­ping $350M up­front to part­ner with genome-edit­ing fo­cused Sang­amo

While the fate of Biogen’s resurrected Alzheimer’s drug aducanumab remains uncertain, the Cambridge, MA-based drugmaker is joining forces with genome editing company Sangamo Therapeutics to develop therapies for neurological conditions.

Sangamo is set to receive a meaty $350 million upfront in cash and stock and is eligible to receive up to $2.37 billion in milestone payments, in addition to royalties. In return, Biogen gets the rights to two Sangamo preclinical compounds: ST-501 (for use in tauopathies including Alzheimer’s disease) and ST-502 (for synucleinopathies including Parkinson’s disease).

“The partnership represents a lower-cost way to expand its work in neurologic disease,” Credit Suisse’s Evan Seigerman said in a note, referring to Biogen.

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Spark los­es an­oth­er top ex­ec in the wake of $4.3B takeover by Roche — re­port

Days after bidding farewell to co-founder Kathy High, Spark Therapeutics — now operating under Roche — has one more opening on its C-suite.

Kathy Reape

Kathy Reape, who joined the Philadelphia-based biotech in 2016 as head of clinical R&D and became chief medical officer in 2018, is reportedly set to leave.

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'The head­lines are the head­lines, but': Bio­Marin talks up po­ten­tial sav­ings as he­mo­phil­ia gene ther­a­py launch looms

BioMarin execs are still staying tight-lipped about their pricing plans for what is poised to be the world’s first hemophilia gene therapy. But as the company enters the final regulatory stretch and approaches a potential launch this summer, they are also dropping more hints to get investors ready.

First thing to know: They really, really don’t expect an advisory committee to be convened for valrox, which is under priority review, to pop up before its PDUFA date on August 21.

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Vlad Coric (Photo Credit: Andrew Venditti)

Bio­haven scores CGRP OK for acute mi­graine — can the com­mer­cial team catch up with Al­ler­gan on its de­but?

Seven years after spinning out of Yale, Biohaven has entered the ranks of commercial-stage biotechs.

The FDA handed down an OK for its CGRP drug, rimegepant, as an acute treatment. Dubbed Nurtec, the orally dissolving pill will join Allergan’s (soon to be AbbVie’s) Ubrelvy and Lilly’s Reyvow on the market amid a new wave of migraine therapies reshaping the disease space.

In a pivotal Phase III trial, Nurtec hit the co-primary endpoints on pain freedom and freedom from most bothersome symptoms at two hours post dose, proving superior to placebo.

Mi­cro­bio­me Q&A: New study maps the vagi­na's 'op­ti­mal mi­cro­bio­ta' — and its im­pli­ca­tions for bio­phar­ma

The widely-held notion that the “optimal” vaginal microbiota is dominated by one strain of lactic-acid producing bacteria has now been challenged in a new paper, published in Nature Communications on Wednesday, which used advanced gene sequencing methods to map out the most comprehensive gene catalog of the human vaginal microbiome.

Things have changed in the more than 50 years since the concept of vaginal microbiota transplants was proposed and subsequently tainted by a Texas-based gynecologist who transplanted the vaginal fluid of women who had bacterial vaginosis into healthy females, suspecting he had isolated the bacteria responsible for the condition.

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