Andrea Pfeifer (AC Immune)

AC Im­mune has 're­mark­able' Alzheimer's vac­cine da­ta. Just don't ask to see them

AC Im­mune an­nounced it has “re­mark­able,” “un­prece­dent­ed” re­sults on an Alzheimer’s vac­cine. You’re just not al­lowed to see the da­ta.

On Thurs­day, the J&J-part­nered Swiss biotech said that its vac­cine had gen­er­at­ed “high” lev­els of an­ti­bod­ies against tau, a tan­gled pro­tein com­mon­ly im­pli­cat­ed in Alzheimer’s, in every pa­tient in their Phase I/II tri­al for the neu­rode­gen­er­a­tive dis­ease. The stock $ACIU in­stant­ly dou­bled in pre-mar­ket trad­ing, ris­ing from $7.30 to $14.70, and briefly mak­ing AC Im­mune once again a $1 bil­lion com­pa­ny.

There were two caveats, though. For one, the study was tiny by Alzheimer’s stan­dards: just 32 pa­tients across two dose co­horts.

And, per­haps more no­tably, the da­ta re­lease was sur­pris­ing­ly de­void of da­ta. Al­though AC Im­mune said that they in­creased an­ti­body lev­els to “po­ten­tial­ly ther­a­peu­tic lev­els” in 100% of pa­tients, they didn’t de­fine what they be­lieved to be the po­ten­tial ther­a­peu­tic lev­el — whether that was a 5% in­crease or a 90% in­crease. And they didn’t say what the av­er­age or me­di­an in­crease was across the study, al­though they said it was high­er than their ref­er­ence da­ta for pa­tients who re­ceived in­fused an­ti­bod­ies.

In a field that’s seen near­ly every Phase III study fail, no mat­ter how pos­i­tive bio­mark­ers were in ear­ly tri­als, the an­nounce­ment earned de­ri­sion from some in­vestors on Twit­ter. The stock cooled down to $10 once trad­ing opened.

No­tably, the re­lease comes af­ter an­oth­er Alzheimer’s biotech has surged dra­mat­i­cal­ly off of sun­ny press re­leas­es. Cas­sa­va Sci­ences, an Austin biotech that sold for less than $3 a share 6 months ago, now trades just un­der $50 af­ter re­leas­ing non-sta­tis­ti­cal­ly sig­nif­i­cant da­ta from open la­bel tri­als that CEO Re­mi Bar­bi­er said they “could not be more pleased with.” The stock has caught on with re­tail in­vestors, in­clud­ing the Wall­Street­Bets sub­red­dit made fa­mous by the GameStop phe­nom­e­non.

AC Im­mune CEO An­drea Pfeifer sim­i­lar­ly praised their non-dis­closed da­ta as “re­mark­able” and “un­prece­dent­ed.” By con­trast, af­ter Eli Lil­ly an­nounced re­sults last month from an Alzheimer’s study that en­rolled over 200 pa­tients and in­clud­ed a de­tailed break­down of sta­tis­ti­cal­ly sig­nif­i­cant da­ta on both cog­ni­tive de­cline and brain bio­mark­ers, CSO Dan Skrovon­sky called it “tan­ta­liz­ing, but de­bat­able.”

In an email, an AC Im­mune spokesper­son said they wouldn’t re­lease the da­ta, but that high titers of an­ti-tau an­ti­body were crit­i­cal, as it can be dif­fi­cult to get old­er pa­tients to gen­er­ate a strong im­mune re­sponse to a vac­cine.

“We now have ev­i­dence that ACI-35.030 is able to reach the lev­els of an­ti­body that were ther­a­peu­tic in pre­clin­i­cal mod­els, in 100% of pa­tients, with no safe­ty is­sues,” he said. “This re­sult war­rants ad­vanc­ing the pro­gram in­to phase 2/3.”

The re­lease is par­tic­u­lar­ly no­table giv­en AC Im­mune’s re­cent his­to­ry. Af­ter the wreck­age of the amy­loid hy­poth­e­sis, biotech and phar­ma have in­creas­ing­ly turned to tar­get tau as a po­ten­tial path to slow­ing Alzheimer’s pa­tients’ de­cline. That shift has land­ed AC Im­mune big-mon­ey col­lab­o­ra­tions with Eli Lil­ly, Genen­tech and J&J.

Yet the com­pa­ny has re­cent­ly seen how dif­fi­cult it can be to trans­late ear­ly da­ta. The com­pa­ny’s stock col­lapsed in Sep­tem­ber af­ter Genen­tech an­nounced their co-de­vel­oped tau-clear­ing an­ti­body failed to im­prove cog­ni­tive per­for­mance on three dif­fer­ent met­rics, de­spite Phase I re­sults that the com­pa­nies ev­i­dent­ly deemed promis­ing enough. (AC Im­mune has yet to an­nounce whether the drug cleared tau in the study.)

AC Im­mune was al­so dealt a small blow at the end of the last month, when Eli Lil­ly an­nounced in their Q4 they would stop de­vel­op­ing for Alzheimer’s a mol­e­cule they had li­censed from the biotech. The AC Im­mune spokesper­son said de­vel­op­ment will con­tin­ue in oth­er tauopathies, a group of rare neu­rode­gen­er­a­tive con­di­tions.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

So what hap­pened with No­var­tis' gene ther­a­py group? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Volker Wagner (L) and Jeff Legos

As Bay­er, No­var­tis stack up their ra­dio­phar­ma­ceu­ti­cal da­ta at #ES­MO21, a key de­bate takes shape

Ten years ago, a small Norwegian biotech by the name of Algeta showed up at ESMO — then the European Multidisciplinary Cancer Conference 2011 — and declared that its Bayer-partnered targeted radionuclide therapy, radium-223 chloride, boosted the overall survival of castration-resistant prostate cancer patients with symptomatic bone metastases.

In a Phase III study dubbed ALSYMPCA, patients who were treated with radium-223 chloride lived a median of 14 months compared to 11.2 months. The FDA would stamp an approval on it based on those data two years later, after Bayer snapped up Algeta and christened the drug Xofigo.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,900+ biopharma pros reading Endpoints daily — and it's free.

Mi­rati tri­umphs again in KRAS-mu­tat­ed lung can­cer with a close­ly watched FDA fil­ing now in the cards

After a busy weekend at #ESMO21, which included a big readout for its KRAS drug adagrasib in colon cancer, Mirati Therapeutics is ready to keep the pressure on competitor Amgen with lung cancer data that will undergird an upcoming filing.

In topline results from a Phase II cohort of its KRYSTAL-1 study, adagrasib posted a response rate of 43% in second-line-or-later patients with metastatic non-small cell lung cancer containing a KRAS-G12C mutation, Mirati said Monday.

When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,900+ biopharma pros reading Endpoints daily — and it's free.

Ted White, Verrica CEO

Ver­ri­ca hits an­oth­er bump in the road with CMO re­lat­ed let­ter from FDA

The FDA has rejected Verrica’s new drug application for VP-102 again, with the company pinning the CRL on problems at a CMO that it was partnered with, the company announced Monday.

The FDA didn’t raise issues that directly relate to the manufacturing of VP-102, the company said, but raised “general quality issues” at the CMO’s facility. There were also no clinical concerns, it said, or need to collect more data.