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.”

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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Michael Gladstone, partner at Atlas Venture

At­las rais­es new $400M fund amid spree of VC rais­es. Here’s what they’ll spend it on

You can add another few hundred million to the now Montana-sized reservoir of cash biotech VCs have raised since the WHO declared Covid-19 a pandemic.

Atlas Venture, the prominent Kendall Square incubator, has raised $400 million for its twelfth biotech fund, their first in 3 years. After a string of mammoth new raises from other major VCs in April and May, the total pot now stands between $5 billion and $6 billion, depending on how you slice it.

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President Donald Trump (left) and Moncef Slaoui, head of Operation Warp Speed (Alex Brandon, AP Images)

UP­DAT­ED: White House names fi­nal­ists for Op­er­a­tion Warp Speed — with 5 ex­pect­ed names and one no­table omis­sion

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.

FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

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Gilead bol­sters its case for block­buster hope­ful fil­go­tinib as FDA pon­ders its de­ci­sion

Before remdesivir soaked up the spotlight amid the coronavirus crisis, Gilead’s filgotinib was the star experimental drug tapped to rake in billions competing with other JAK inhibitors made by rivals including AbbVie and Eli Lilly.

Now, long term data on the drug — discovered by Gilead’s partners at Galapagos and posted as part of a virtual medical conference — have solidified the durability and safety of filgotinib in patients with rheumatoid arthritis, spanning data from three late-stage trials. An FDA decision on the drug is expected this year.