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.

Which brings me to Athi­ra, a lit­tle, Seat­tle-based biotech that caught my at­ten­tion with a heads-up that they’re an­nounc­ing an $85 mil­lion round to­day to fund their Phase II/III quest for Alzheimer’s. Athi­ra’s team has been on a long, 9-year jour­ney to get here, and they are acute­ly aware of the burned-out wreck­age and dis­as­ter head­lines cov­er­ing the route that they have been cruis­ing.

To get the mon­ey, they en­list­ed a long line of back­ers: Per­cep­tive Ad­vi­sors for the lead with par­tic­i­pa­tion from new in­vestors RTW In­vest­ments, Viking Glob­al In­vestors, Ven­rock Health­care Cap­i­tal Part­ners, Franklin Tem­ple­ton, Rock Springs Cap­i­tal, LifeSci Ven­ture Part­ners, Sur­vey­or Cap­i­tal (a Citadel com­pa­ny), High­side Cap­i­tal Man­age­ment, Lo­gos Cap­i­tal, funds man­aged by Janus Hen­der­son In­vestors, Sofinno­va In­vest­ments, Avid­i­ty Part­ners, and ex­ist­ing in­vestors in­clud­ing Rick and Suzanne Kayne and Sah­sen Ven­tures.

Joe Edel­man, who runs the well-con­nect­ed Per­cep­tive, is join­ing the board af­ter giv­ing the crew a thumbs up.

So far, Athi­ra’s back­ers have put up about $20 mil­lion to get to this point, so you can see how big a deal this is to the biotech team.

There are in­vestors out there who have nev­er been burned by Alzheimer’s and who are still ready to con­sid­er jump­ing in, says CEO Leen Kawas, who’s been mak­ing the rounds, and then there have been plen­ty which have.

“I’m proud we put to­geth­er a syn­di­cate that in­cludes both,” she tells me.

Edel­man and the rest are back­ing a drug — NDX-1017 — that by­pass­es the whole tox­ic plaque de­bate and fo­cus­es on an­oth­er fa­mil­iar strat­e­gy: the frayed synap­tic net­work in the brain that erodes with the dis­ease. Boost the net­work and you can stop, pos­si­bly re­verse, the dam­age that cor­rodes cog­ni­tion. For awhile.

Their small mol­e­cule is de­signed to tar­get he­pa­to­cyte growth fac­tor (HGF) and its re­cep­tor, MET, in an ef­fort to re­gen­er­ate af­flict­ed tis­sue. And that’s some­thing these in­vestors haven’t seen be­fore.

Athi­ra got this far by spot­light­ing da­ta they say back up the ef­fi­ca­cy pro­file they’re look­ing for. In a Phase Ib, Kawas says, re­searchers used a test to gauge pa­tients’ abil­i­ty to count odd tones pep­pered in a string of re­peat­ed tones. For pa­tients with de­men­tia, their nor­mal pat­tern of recog­ni­tion falls in a 400 to 450 mil­lisec­ond range, says the CEO. Their base­line in the study was 390. In the drug arm, just 7 pa­tients, that dropped to an av­er­age of 311. There was no change among 4 place­bo pa­tients, says Kawas.

Now, they’ll need to see if they can repli­cate a cog­ni­tive im­prove­ment in a much larg­er Phase II. And then they can move on to a piv­otal Phase III. The FDA re­quires pos­i­tive da­ta from 2 con­trolled stud­ies, and that might be it. But Kawas knows that if they can’t con­vince reg­u­la­tors — bal­anc­ing the da­ta against the moun­tain­ous un­met need — they’d need to go on to a sec­ond Phase III.

The odds against suc­cess here are sig­nif­i­cant. Every­thing fails in Alzheimer’s. Or has. But win­ning over a syn­di­cate of in­vestors able to pro­vide that kind of cash is al­so val­i­dat­ing — and no mean achieve­ment in this en­vi­ron­ment.

Right now, the Athi­ra team has spent a good chunk of their lives de­vot­ed to see­ing if this drug can work. “We’re be­liev­ers that this can be done,” says the CEO.

It’s a daunt­ing up­hill climb, but they in­tend to make be­liev­ers out of every­one else as well. As some­where down the trail, the plan is to float an IPO down Wall Street to see if they can win over an even big­ger group of be­liev­ers.

A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

Su­per-se­cre­tive an­ti-ag­ing biotech Cal­i­co tees up the first vis­i­ble clin­i­cal tri­al of an ex­per­i­men­tal drug. And it’s for can­cer?

Over the past 7 years, Calico has been so much more than your average, run-of-the-mill secretive biotech players. It’s a riddle, wrapped in a mystery, inside an enigma, to repurpose an old Winston Churchill line dating from the time he confronted the Iron Curtain surrounding Stalin’s thoughts.

Launched by industry legend Art Levinson of Genentech fame, with the infinitely deep pockets of Google for support, one of the few big headlines the anti-aging biotech has sparked focused on a major alliance with AbbVie — a giant outfit that conversely likes to show off its drug prospects whenever it can. Together, they’ve been focused on diseases that limit life span — quite an arc of ailments.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 91,900+ biopharma pros reading Endpoints daily — and it's free.

RBC's Bri­an Abra­hams holds a mock ad­comm on Bio­gen's iffy ad­u­canum­ab da­ta — and most of these ex­perts don't see a path to an ap­proval

As catalysts go, few loom larger than the aducanumab adcomm slated for Nov. 6.

With its big franchise under assault, Biogen is betting the ranch that its mixed late-stage Alzheimer’s data can squeak past the experts and regulators and get onto the market. And the topic — after a decade of Alzheimer’s R&D disasters in what still represents the El Dorado of drug markets — remains in the center ring of discussions around late-stage pipeline prospects.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 91,900+ biopharma pros reading Endpoints daily — and it's free.

Af­ter near­ly a year of de­bate, the Covid-19 vac­cine chal­lenge tri­als are of­fi­cial­ly com­ing

After nearly a year of public advocacy and often rancorous ethical debate, human challenge trials for Covid-19 vaccines are getting off the ground in London.

The UK government’s Covid-19 Vaccine Taskforce and the contract research firm Open Orphan announced today £10 million ($13 million) plan to test experimental Covid-19 vaccines in volunteers whintentionally exposed to the novel coronavirus. The studies, which won’t launch until early 2021, come after 9 months of debate over whether such studies were safe and would actually hasten vaccine development, and they follow a long history of researchers using challenge models to study other respiratory viruses, including flu and the coronaviruses that cause the common cold.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 91,900+ biopharma pros reading Endpoints daily — and it's free.

Derek Chalmers, Cara Ther

Cara lines up a $440M deal for US rights to its late-stage drug for se­vere itch, with $150M cash on the ta­ble

With plans afoot to file an NDA for what could be its first approved drug, Cara Therapeutics is pivoting its focus to commercialization. And Swiss company Vifor Pharma is willing to surrender up to $440 million to market the candidate in the US.

Cara $CARA CEO Derek Chalmers said an NDA submission is coming this quarter for their intravenous drug Korsuva in chronic kidney disease-associated pruritus (CKD-aP), a severe itching condition. The Stamford, CT-based biotech read out positive topline data from a Phase III pivotal study back in April, and announced plans to approach EMA regulators shortly after filing with the FDA.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 91,900+ biopharma pros reading Endpoints daily — and it's free.

Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

Gilead feels the heat as close al­ly Gala­pa­gos re­ports a big set­back on one of their top ex­per­i­men­tal drugs

The bad news keeps stacking up at Galapagos — which quite likely just lost control of a billion-dollar deal — and by extension their close partners at Gilead.

The biotech $GLPG reported after the bell Thursday that GLPG1972, one of their top development programs, flat failed a mid-stage study for osteoarthritis, flunking the primary and all secondary endpoints.

Testing 3 different doses of their drug, which relies on ADAMTS-5 inhibition, investigators concluded that none of them triggered a statistically significant response — as measured by cartilage thickness.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 91,900+ biopharma pros reading Endpoints daily — and it's free.

Covid-19 roundup: Pars­ing Bourla, a top an­a­lyst sees im­proved chances for Pfiz­er vac­cine; Fau­ci: No sur­prise that Trump was hit by Covid-19

With a medley of adverse events hobbling the late-stage development of vaccines and drugs, Pfizer CEO Albert Bourla’s latest — extended — timeline for the mRNA approach they’re working on with BioNTech is giving some top analysts added confidence that the pharma giant can come up with the regulatory goods next month.

Parsing Bourla’s language in his comments last week, SVB Leerink’s Geoffrey Porges notes that Bourla’s decision to say they “may” be able to nail down the positive efficacy of their vaccine in a matter of days — a big change from his earlier certainty — may also indicate a delay on that to early November.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 91,900+ biopharma pros reading Endpoints daily — and it's free.

CAR-plus: Irish biotech re­cruits Kite alum Chris Now­ers to prep dual-tar­get­ing NK cell ther­a­py for the clin­ic

Soon after Chris Nowers left Cell Medica — freshly rebranded Kuur Therapeutics — in February, the Kite Pharma alum was introduced to another cell therapy player.

The basic idea of building an off-the-shelf allogeneic platform with a CAR-NK approach was familiar to him, riding on the same wave as Takeda, J&J-backed Fate, Nkarta and others. But then there was something else that stood out: a membrane-bound TNF related apoptosis inducing ligand variant, or TRAIL variant, that’s also engineered onto the NK cell for a dual-targeted attack.

Mer­ck touts new da­ta for Keytru­da com­bos in NSCLC at North Amer­i­can con­fer­ence

Merck marched out new data from two studies on Friday to back king Keytruda — the drug that made the Big Pharma $11.1 billion last year — in advanced non-small cell lung cancer (NSCLC).

At the IASLC 2020 North America Conference on Lung Cancer, Merck read out long-term data from Cohort G of its Keynote-021 study, which assessed Keytruda in combination with chemotherapy. It also touted results from a Phase I/II study testing Keytruda and quavonlimab, its anti-CTLA-4 therapy, as a first-line therapy.