Ab­b­Vie en­lists pro­tein degra­da­tion ex­perts at Mis­sion for a new bet on Alzheimer's, Parkin­son's

As a group of pre­clin­i­cal biotechs fo­cused on pro­tein degra­da­tion steadi­ly gath­ers steam, Ab­b­Vie is jump­ing on the band­wag­on al­beit steer­ing it to a some­what sur­pris­ing di­rec­tion: Alzheimer’s and Parkin­son’s dis­eases.

Mis­sion Ther­a­peu­tics, its cho­sen part­ner, has been con­cen­trat­ing much of its ef­forts on hit­ting USP30 and USP10 — two of over 100 deu­biq­ui­ty­lat­ing en­zymes, or DUBs, in the hu­man body that col­lec­tive­ly serve as a nat­ur­al “garbage dis­pos­al” sys­tem for cells — with one USP30 pro­gram in Parkin­son’s and neu­rode­gen­er­a­tion. Ab­b­Vie, though, is more in­ter­est­ed in iden­ti­fy­ing new DUB tar­gets and get­ting Mis­sion’s dis­cov­ery plat­form to churn out be­spoke com­pounds.

Anker Lun­demose

The col­lab­o­ra­tion cov­ers ex­clu­sive rights to up to four se­lect­ed tar­gets — each to come with its own up­front li­cense fee and mile­stones for the Cam­bridge, UK-based biotech — if Ab­b­Vie ex­er­cis­es its op­tion. Fi­nan­cial terms were kept un­der wraps.

The the­o­ry is that by elim­i­nat­ing and pre­vent­ing the ac­cu­mu­la­tion of mis­fold­ed, tox­ic pro­teins — the hall­marks of both Alzheimer’s and Parkin­son’s — they can ad­dress the func­tion im­pair­ment and brain nerve cell deaths that come with the dis­eases.

Two of these pro­teins, tau and amy­loid be­ta, re­main the two key tar­gets in Alzheimer’s, even af­ter a se­ries of ma­jor set­backs in the field raise fresh ques­tions about how much we know and don’t know about this dev­as­tat­ing dis­eases.

While this marks the first ma­jor pact for Mis­sion in its sev­en years ac­cord­ing to CEO Anker Lun­demose, it’s one of sev­er­al ear­ly bets for Ab­b­Vie, which in Feb­ru­ary had al­so hand­ed Voy­ager Ther­a­peu­tics $69 mil­lion to pur­sue a gene ther­a­py ap­proach to Alzheimer’s.

Mis­sion’s peers in the pro­tein degra­da­tion field, mean­while, are main­ly fo­cused on on­col­o­gy, from C4 Ther­a­peu­tics, Arv­inas to Kymera, which raised $65 mil­lion just days ago.

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

Brian Stuglik (file photo)

Turn­ing fo­cus to clin­i­cal work, Ve­rastem ax­es 31 jobs, scales back can­cer drug pro­mo­tion af­ter dis­ap­point­ing sales

Months after taking the helm at Verastem Oncology, Brian Stuglik has a plan to take the biotech in a “new strategic direction” — but not before some layoffs.

Left out of an upbeat press release spelling out its clinical plans, and buried below news of a $100 million private placement in an SEC filing, is a planned restructuring that will claim 31 jobs. Alongside some other cost-saving measures, Verastem expects to cut expenses down by $70 million to $80 million per year.

Olivier Brandicourt (AP Images)

Ex-Sanofi chief Olivi­er Brandi­court, cur­rent Black­stone ad­vi­sor, jumps on Al­ny­lam board

Former Sanofi chief Olivier Brandicourt, who departed his post with an unexpected early retirement last year, has made his move — as most C-suite executives inevitably do — to become a director on the board of a biopharma company.

RNAi player Alnylam is Brandicourt’s destination. Meanwhile, the Cambridge, Massachusetts-based drugmaker — which pioneered the first approval in the field — also disclosed the retirement of Alnylam co-founder Dr. Paul Schimmel from its board.

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.

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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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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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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.

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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