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. 

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.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 73,300+ biopharma pros reading Endpoints daily — and it's free.

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

Jim Wilson's gene ther­a­py start­up Pas­sage Bio bucks mar­ket sen­ti­ments, rais­ing up­sized $216M IPO

A coronavirus fear-induced bloodbath on the Nasdaq has not stopped Passage Bio from making a public debut — and an exuberant one.

By pricing an upsized offering at $18, the top of the range, the gene therapy biotech bagged $216 million from its IPO, 72% more than it’s originally penciled in.

The proceeds likely reflected confidence in Jim Wilson, who gathered all the tools he’s built over decades of gene therapy research to assemble the startup and teamed up with Frazier and OrbiMed to hone its focus on rare, monogenic disorders of the central nervous system. Just before the IPO, Deerfield partner Bruce Goldsmith took over from OrbiMed’s Stephen Squinto as CEO.

Dan O'Day (AP Images)

UP­DAT­ED: 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 acquisition, 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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 73,300+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 73,300+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 73,300+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 73,300+ biopharma pros reading Endpoints daily — and it's free.

'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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 73,300+ biopharma pros reading Endpoints daily — and it's free.

Vlad Coric (Photo Credit: Andrew Venditti)

UP­DAT­ED: 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.