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.

Pfiz­er's big block­buster Xel­janz flunks its post-mar­ket­ing safe­ty study, re­new­ing harsh ques­tions for JAK class

When the FDA approved Pfizer’s JAK inhibitor Xeljanz for rheumatoid arthritis in 2012, they slapped on a black box warning for a laundry list of adverse events and required the New York drugmaker to run a long-term safety study.

That study has since become a consistent headache for Pfizer and their blockbuster molecule. Last year, Pfizer dropped the entire high dose cohort after an independent monitoring board found more patients died in that group than in the low dose arm or a control arm of patients who received one of two TNF inhibitors, Enbrel or Humira.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 99,000+ biopharma pros reading Endpoints daily — and it's free.

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Top gene ther­a­py deals, M&A pacts in 2020 high­light an­oth­er big year in one of the hottest fields in bio­phar­ma

Chris Dokomajilar at DealForma has been crunching the numbers on gene therapy deals over the last 2 years and came away with a few key observations.

Both the upfront cash and deal totals last year backed off a bit from the record high hit in 2019, but the totals are still running well ahead of anything we’ve seen in the years prior to 2019/2020.
2020 R&D partnerships came in at 23 deals, with $1.1 billion in disclosed upfront cash and equity and more than $8.5 billion in total deal value. Looking at 2019-2020 M&A, Dokomajilar found: 9 Acquisitions, with over $11.1 billion in disclosed upfront cash and equity and more than $13.4 billion in total M&A value.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 99,000+ biopharma pros reading Endpoints daily — and it's free.

Bob Nelsen (Michael Kovac/Getty Images)

ARCH an­nounces largest fund yet, rais­ing $1.85B to back men­tal health, cell and gene edit­ing ap­proach­es

Nearly a year ago, as the pandemic encroached and the stock market cratered, Flagship and ARCH Venture announced three mega-funds worth a combined $2.6 billion. They wanted, ARCH’s Bob Nelsen said, to restore confidence “that there was money out there and a lot of it” to invest in biotech.

Since then, the stock market has returned — almost frighteningly so — and Nelsen has kept raising and spending cash. On Thursday, he announced a new fund, worth $1.85 billion. It’s the largest pot yet for a VC famous for its deep pockets.

Take­da earns win for its TKI in­hibitor in tiny lung can­cer group — but GI side ef­fects could be an ear­ly red flag

Japanese drugmaker Takeda has made a big push in recent years to build a hand in oncology, particularly in the next-gen cancer space. One of those candidates, tyrosine kinase inhibitor (TKI) mobocertinib, recently earned the FDA’s interest in a small section of untreated lung cancer patients, but will severe GI side effects be a roadblock?

Takeda’s oral mobocertinib posted clinically significant objective response rates in a Phase I/II adaptive trial drugging metastatic non-small cell lung cancer patients with EGFR exon 20 gene mutations who had previously undergone platinum-based chemotherapy, according to data presented Thursday at the virtual World Conference on Lung Cancer.

Vas Narasimhan (AP Images)

BeiGene's PD-1 scores PhI­II win in esophageal can­cer — a 'key in­di­ca­tion' tapped by No­var­tis in $650M deal

Days after bringing BeiGene’s PD-1 into its portfolio, Novartis is adding a feather to its tislelizumab cap.

BeiGene reported that in the second-line setting, the checkpoint inhibitor extended overall survival for patients with esophageal squamous cell carcinoma, a key indication identified by Novartis. It was the primary endpoint in this Phase III trial.

“This is our fourth positive Phase 3 readout for tislelizumab and the first from our large Phase 3 program in gastrointestinal cancers that also include liver, stomach cancers as well as esophageal cancer,” BeiGene’s CMO in immuno-oncology, Yong Ben, said in a statement.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 99,000+ biopharma pros reading Endpoints daily — and it's free.

Covid-19 roundup: EU and As­traZeneca trade blows over slow­downs; Un­usu­al unions pop up to test an­ti­bod­ies, vac­cines

After coming under fire for manufacturing delays last week, AstraZeneca’s feud with the European Union has spilled into the open.

The bloc accused the pharma giant on Wednesday of pulling out of a meeting to discuss cuts to its vaccine supplies, the AP reported. AstraZeneca denied the reports, saying it still planned on attending the discussion.

Early Wednesday, an EU Commission spokeswoman said that “the representative of AstraZeneca had announced this morning, had informed us this morning that their participation is not confirmed, is not happening.” But an AstraZeneca spokesperson later called the reports “not accurate.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 99,000+ biopharma pros reading Endpoints daily — and it's free.

Janet Woodcock (AP Images)

Ad­vo­ca­cy groups don't want Janet Wood­cock to head the FDA, blast­ing ‘reg­u­la­to­ry fail­ures’ in opi­oid cri­sis

It turns out the controversies around Janet Woodcock’s regulatory legacy weren’t limited to Sarepta’s eteplirsen.

A coalition of advocacy groups dedicated to the opioid crisis urged Norris Cochran and Xavier Becerra — the acting and designated HHS secretary, respectively — to keep her reign as interim FDA chief a “very short transition.” During her lengthy tenure as CDER, they add, Woodcock presided over “one of the worst regulatory agency failures in U.S. history.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 99,000+ biopharma pros reading Endpoints daily — and it's free.

Dean Li kicks off Mer­ck­'s post-Roger Perl­mut­ter era by team­ing with Arti­va and its off-the-shelf CAR-NK tech

Even though Dean Li has now officially taken over for Roger Perlmutter as R&D chief, Merck’s appetite for dealmaking continues to be ravenous.

Li struck his first big deal at the helm Thursday morning, hammering out a collaboration with Artiva Biotherapeutics that could earn the biotech nearly $1.9 billion when all is said and done. It’s a quick rise and validation for Artiva, which just last June launched with a $78 million Series A.