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.

BY­OD Best Prac­tices: How Mo­bile De­vice Strat­e­gy Leads to More Pa­tient-Cen­tric Clin­i­cal Tri­als

Some of the most time- and cost-consuming components of clinical research center on gathering, analyzing, and reporting data. To improve efficiency, many clinical trial sponsors have shifted to electronic clinical outcome assessments (eCOA), including electronic patient-reported outcome (ePRO) tools.

In most cases, patients enter data using apps installed on provisioned devices. At a time when 81% of Americans own a smartphone, why not use the device they rely on every day?

Image: Shutterstock

Eli Lil­ly asks FDA to re­voke EUA for Covid-19 treat­ment

Eli Lilly on Friday requested that the FDA revoke the emergency authorization for its Covid-19 drug bamlanivimab, which is no longer as effective as a combo therapy because of a rise in coronavirus variants across the US.

“With the growing prevalence of variants in the U.S. that bamlanivimab alone may not fully neutralize, and with sufficient supply of etesevimab, we believe now is the right time to complete our planned transition and focus on the administration of these two neutralizing antibodies together,” Daniel Skovronsky, Lilly’s CSO, said in a statement.

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Osman Kibar (Samumed, now Biosplice)

Os­man Kibar lays down his hand at Sa­mumed, step­ping away from CEO role as his once-her­ald­ed an­ti-ag­ing biotech re­brands

Samumed made quite the entrance back in 2016, when it launched with some anti-aging programs and a whopping $12 billion valuation. That level of fanfare was nowhere to be found on Thursday, when the company added another $120 million to its coffers and quietly changed its name to Biosplice Therapeutics.

Why the sudden rebrand?

“We did that for obvious reasons,” CFO and CBO Erich Horsley told Endpoints News. “The name Biosplice echoes our science much more than Samumed does.”

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Mer­ck scraps their $425M Covid-19 drug in lat­est pan­dem­ic set­back

Seven months after paying $425 million cash to acquire it, Merck is scrapping a Covid-19 drug they hoped could provide one of the only treatments for severe hospitalized patients.

Merck’s decision comes after they faced significant and unexpected regulatory delays in getting the drug, known as MK-7110 or CD24Fc, across the finish line. The Big Pharma licensed the drug under the belief that it had already shown sufficient benefit in severe patients and they could help scale it up far faster than OncoImmune, its former owner, could. But in February, the company reported that the FDA insisted Merck run a new trial before seeking authorization.

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Severin Schwan, Roche CEO (Georgios Kefalas/Keystone via AP Images)

Look­ing to ce­ment its lead in packed MS mar­ket, Roche's Ocre­vus un­corks new da­ta in ear­ly-stage pa­tients

Among a positively jam-packed multiple sclerosis market, Roche’s Ocrevus has managed to stand out for what the Swiss drugmaker is calling the most successful launch in its long history. But in order to press its advantage, Ocrevus is looking to earlier-stage patients, and new interim data should help build its case there.

After 48 weeks on Roche’s Ocrevus, 85% of newly diagnosed primary progressing or relapsing MS patients without a history of disease modifying therapy posted no disease activity, including disease progression or relapse, according to interim data set to be presented this weekend at the virtual American Academy of Neurology meeting.

J&J faces CDC ad­vi­so­ry com­mit­tee again next week to weigh Covid-19 vac­cine risks

The CDC’s Advisory Committee on Immunization Practices punted earlier this week on deciding whether or not to recommend lifting a pause on the administration of J&J’s Covid-19 vaccine, but the committee will meet again in an emergency session next Friday to discuss the safety issues further.

The timing of the meeting likely means that the J&J vaccine will not return to the US market before the end of next week as the FDA looks to work hand-in-hand with the CDC to ensure the benefits of the vaccine still outweigh the risks for all age groups.

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Ex­clu­sive in­ter­view: Pe­ter Marks on why full Covid-19 vac­cine ap­provals could be just months away

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, took time out of his busy schedule last Friday to discuss with Endpoints News all things related to his work regulating vaccines and the pandemic.

Marks, who quietly coined the name “Operation Warp Speed” before deciding to stick with his work regulating vaccines at the FDA rather than join the Trump-era program, has been the face of vaccine regulation for the FDA throughout the pandemic, and is usually spotted in Zoom meetings seated in front of his wife’s paintings.

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Near­ly a year af­ter Au­den­tes' gene ther­a­py deaths, the tri­al con­tin­ues. What hap­pened re­mains a mys­tery

Natalie Holles was five months into her tenure as Audentes CEO and working to smooth out a $3 billion merger when the world crashed in.

Holles and her team received word on the morning of May 5 that, hours before, a patient died in a trial for their lead gene therapy. They went into triage mode, alerting the FDA, calling trial investigators to begin to understand what happened, and, the next day, writing a letter to alert the patient community so they would be the first to know. “We wanted to be as forthright and transparent as possible,” Holles told me late last month.

The brief letter noted two other patients also suffered severe reactions after receiving a high dose of the therapy and were undergoing treatment. One died a month and a half later, at which point news of the deaths became public, jolting an emergent gene therapy field and raising questions about the safety of the high doses Audentes and others were now using. The third patient died in August.

“It was deeply saddening,” Holles said. “But I was — we were — resolute and determined to understand what happened and learn from it and get back on track.”

Eleven months have now passed since the first death and the therapy, a potential cure for a rare and fatal muscle-wasting disease called X-linked myotubular myopathy, is back on track, the FDA having cleared the company to resume dosing at a lower level. Audentes itself is no more; last month, Japanese pharma giant Astellas announced it had completed working out the kinks of the $3 billion merger and had restructured and rebranded the subsidiary as Astellas Gene Therapies. Holles, having successfully steered both efforts, departed.

Still, questions about precisely what led to the deaths of the 3 boys still linger. Trial investigators released key details about the case last August and December, pointing to a biological landmine that Audentes could not have seen coming — a moment of profound medical misfortune. In an emerging field that’s promised cures for devastating diseases but also seen its share of safety setbacks, the cases provided a cautionary tale.

Audentes “contributed in a positive way by giving a painful but important example for others to look at and learn from,” Terry Flotte, dean of the UMass School of Medicine and editor of the journal Human Gene Therapy, told me. “I can’t see anything they did wrong.”

Yet some researchers say they’re still waiting on Astellas to release more data. The company has yet to publish a full paper detailing what happened, nor have they indicated that they will. In the meantime, it remains unclear what triggered the events and how to prevent them in the future.

“Since Audentes was the first one and we don’t have additional information, we’re kind of in a holding pattern, flying around, waiting to figure out how to land our vehicles,” said Jude Samulski, professor of pharmacology at UNC’s Gene Therapy Center and CSO of the gene therapy biotech AskBio, now a subsidiary of Bayer.

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Alex Leech, Alchemab CEO (SV Health Investors)

Alchemab bags fresh round of in­vestor for tar­get-ag­nos­tic an­ti­body de­vel­op­ment for Hunt­ing­ton's, Covid-19

With a “target-agnostic” approach to antibody development, the UK’s Alchemab has used lessons learned from patients with resistance to certain diseases to chase after conditions as far apart as Huntington’s and Covid-19. Now, investors are jumping on board the concept with an $86 million Series A.

The proceeds will go toward advancing the company’s target-agnostic drug discovery program, a release said. That approach looks at the antibody repertoires of patients who show resistance to typically destructive diseases regardless of genetic disposition.