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.

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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Tom Barnes (Orna)

The mR­NA era is here. MPM be­lieves the fu­ture be­longs to oR­NA — and Big Phar­ma wants a seat at the ta­ble

If the ultra-fast clinical development of Covid-19 vaccines opened the world’s eyes to the promises of messenger RNA, the subsequent delays in supply offered a crash course on the ultra-complex process of producing them. Even before the formulation and fill-finish steps, mRNA is the precious end product from an arduous journey involving enzyme-aided transcription, modification and purification.

For Bristol Myers Squibb, Novartis Institutes for Biomedical Research, Gilead’s Kite and Astellas, it’s time to rethink the way therapeutic RNA is engineered.

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Tal Zaks, Moderna CMO (AP Photo/Rodrique Ngowi, via still image from video)

CMO Tal Zaks bids Mod­er­na a sur­prise adieu as biotech projects $18.4B in rev­enue, plots post-Covid ex­pan­sion

How do you exit a company after six years in style? Developing one of the most lucrative and life-saving products in pharma history is probably not the worst way to go.

Tal Zaks, Moderna’s CMO since 2015, will leave the mRNA biotech in September, the biotech disclosed in their annual report this morning. The company has already retained the recruitment firm Russell Reynolds to find a replacement.

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Glax­o­SmithK­line re­thinks strat­e­gy for Covid-19 an­ti­body — not the Vir ones — af­ter tri­al flop. Is there hope in high-risk pa­tients?

In the search for a better Covid-19 therapeutic, GlaxoSmithKline and Vir have partnered up on two antibodies they hope have a chance. GSK is also testing its own in-house antibody, and early results may have shut the door on its widespread use.

A combination of GSK’s monoclonal antibody otilimab plus standard of care couldn’t best standard of care alone in preventing death and respiratory failure in hospitalized Covid-19 patients after 28 days, according to data from the Phase IIa OSCAR study unveiled Thursday.

Mod­er­na's Stéphane Ban­cel plans to dou­ble down on vac­cine pro­duc­tion, new vari­ants as mR­NA rules in pan­dem­ic fight

Stéphane Bancel thought he’d be sleeping more by now.

The 48-year-old Moderna CEO figured that by 2021 he’d have his vaccine through the clinic, authorized, and in mass production — that the hard part would be over. Instead, he’s still working Saturdays and Sundays, talking with his lab and manufacturing teams and fielding calls with two to three world leaders a day to answer their concerns about supply and emerging new variants.

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S&P ex­pects steady ero­sion in Big Phar­ma's cred­it pro­file in 2021 as new M&A deals roll in — but don't un­der­es­ti­mate their un­der­ly­ing strength

S&P Global has taken a look at the dominant forces shaping the pharma market and come to the conclusion that there will be more downgrades than upgrades in 2021 — the 8th straight year of steady decline.

But it’s not all bad news. Some things are looking up, and there’s still plenty of money to be made in an industry that enjoys a 30% to 40% profit margin, once you factor in steep R&D expenses.

Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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