Alzheon files for $80M IPO to take once-failed Alzheimer's drug in­to PhI­II

A com­pa­ny based just out­side Boston is join­ing the pa­rade to the pub­lic mar­kets, hop­ing to raise $80.5 mil­lion to de­vel­op an Alzheimer’s drug that’s a true Hail Mary, con­sid­er­ing the field’s re­cent im­plo­sions.

Mar­tin To­lar

The drug­mak­er, Alzheon, filed a no­tice with the SEC sig­nal­ing its up­com­ing IPO, but the com­pa­ny might need more cash than it can raise in this round. Back in 2016, Alzheon’s CEO Mar­tin To­lar told End­points News the com­pa­ny would need more like $100 mil­lion to pay for two Phase III stud­ies for its lead drug — and that would be a bar­gain com­pared to most late-stage Alzheimer’s pro­grams.

To­lar — the biggest share­hold­er with 44% of the stock — al­so needs the cash. He on­ly had a lit­tle more than $6 mil­lion on hand at the end of De­cem­ber af­ter burn­ing through about $24 mil­lion. He’s com­mit­ted to pay­ing a roy­al­ty stream to FB Health, which out-li­censed the rights to the drug, in the event they can win an ap­proval.

Alzheon is de­vel­op­ing an amy­loid-block­ing drug called ALZ-801 (tramiprosate), which it li­censed from Mon­tre­al-based Neu­rochem back in 2013. Since the drug flopped in Neu­rochem’s tri­als, Alzheon made some tweaks to the once-dai­ly pill that the com­pa­ny hopes will get the drug to its tar­get more quick­ly while re­duc­ing side ef­fects in the gut.

In its S-1, the com­pa­ny tells in­vestors that they met with the FDA and went over the da­ta, point­ing to a post hoc analy­sis of the da­ta in­di­cat­ing that a 150 mg dose of the drug among APOE4/4 ho­mozy­gous pa­tients reg­is­tered an im­pact on cog­ni­tion and dai­ly func­tion, the gold stan­dard that has de­feat­ed vir­tu­al­ly every­thing thrown at it over the last 15 years. By stick­ing with a clear­ly de­fined group of pa­tients, the biotech be­lieves it can avoid re­cruit­ing pa­tients for its study who don’t ac­tu­al­ly have the dis­ease. But FDA of­fi­cials say the da­ta were on­ly from a small sub­set of pa­tients and didn’t prove ef­fi­ca­cy. Fur­ther­more, the agency told the com­pa­ny they might have to run a sec­ond Phase III tri­al to prove any pos­i­tive re­sults they gained from the first one.

Alzheon is fo­cus­ing on a strat­e­gy very sim­i­lar to the one adopt­ed by Ax­o­vant $AX­ON: tak­ing a failed drug and re­ly­ing on ex­ist­ing da­ta spied in a post hoc analy­sis to prove it’s both safe and po­ten­tial­ly ef­fec­tive for a spe­cif­ic pop­u­la­tion of pa­tients. Of course, the ef­fort proved fu­tile for Ax­o­vant, which watched its stock plum­met near­ly 80% when its Alzheimer’s drug crashed and burned in a Phase III study.

It doesn’t help Alzheon’s odds that its pur­su­ing the amy­loid be­ta hy­poth­e­sis, which has de­feat­ed every drug thrown at it so far in a piv­otal study. Eli Lil­ly $LLY tried this route for treat­ing Alzheimer’s with solanezum­ab, but scrapped the pro­gram for treat­ing symp­to­matic pa­tients a cou­ple years ago thanks to a se­ries of Phase III fail­ures. The drug is cur­rent­ly be­ing stud­ied to see if it can de­lay the de­vel­op­ment of pre­clin­i­cal pa­tients.

Alzheon, which will list on the Nas­daq un­der the sym­bol $ALZH, plans to start a Phase III tri­al of ALZ-801 in the US and in­ter­na­tion­al­ly in 2018, ac­cord­ing to the com­pa­ny’s SEC state­ment. First, though, it will have to over­come some scathing re­views.

Hal Barron, GSK

Break­ing the death spi­ral: Hal Bar­ron talks about trans­form­ing the mori­bund R&D cul­ture at GSK in a crit­i­cal year for the late-stage pipeline

Just ahead of GlaxoSmithKline’s Q2 update on Wednesday, science chief Hal Barron is making the rounds to talk up the pharma giant’s late-stage strategy as the top execs continue to woo back a deeply skeptical investor group while pushing through a whole new R&D culture.

And that’s not easy, Barron is quick to note. He told the Financial Times:

I think that culture, to some extent, is as hard, in fact even harder, than doing the science.

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Aca­dia is mak­ing the best of it, but their lat­est PhI­II Nu­plazid study is a bust

Acadia’s late-stage program to widen the commercial prospects for Nuplazid has hit a wall. The biotech reported that their Phase III ENHANCE trial flat failed. And while they $ACAD did their best to cherry pick positive data wherever they can be found, this is a clear setback for the biotech.

With close to 400 patients enrolled, researchers said the drug flunked the primary endpoint as an adjunctive therapy for patients with an inadequate response to antipsychotic therapy. The p-value was an ugly 0.0940 on the Positive and Negative Syndrome Scale, which the company called out as a positive trend.

Their shares slid 12% on the news, good for a $426 million hit on a $3.7 billion market cap at close.

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Some Big Phar­mas stepped up their game on da­ta trans­paren­cy — but which flunked the test?

The nonprofit Bioethics International has come out with their latest scorecard on data transparency among the big biopharmas in the industry — flagging a few standouts while spotlighting some laggards who are continuing to underperform.

Now in its third year, the nonprofit created a new set of standards with Yale School of Medicine and Stanford Law School to evaluate the track record on trial registration, results reporting, publication and data-sharing practice.

Busy Gilead crew throws strug­gling biotech a life­line, with some cash up­front and hun­dreds of mil­lions in biobucks for HIV deal

Durect $DRRX got a badly needed shot in the arm Monday morning as Gilead’s busy BD team lined up access to its extended-release platform tech for HIV and hepatitis B.

Gilead, a leader in the HIV sector, is paying a modest $25 million in cash for the right to jump on the platform at Durect, which has been using its technology to come up with an extended-release version of bupivacaine. The FDA rejected that in 2014, but Durect has been working on a comeback.

In­tec blitzed by PhI­II flop as lead pro­gram fails to beat Mer­ck­'s stan­dard com­bo for Parkin­son’s

Intec Pharma’s $NTEC lead drug slammed into a brick wall Monday morning. The small-cap Israeli biotech reported that its lead program — coming off a platform designed to produce a safer, more effective oral drug for Parkinson’s — failed the Phase III at the primary endpoint.

Researchers at Intec, which has already seen its share price collapse over the past few months, says that its Accordion Pill-Carbidopa/Levodopa failed to prove superior to Sinemet in reducing daily ‘off’ time. 

Cel­gene racks up third Ote­zla ap­proval, heat­ing up talks about who Bris­tol-My­ers will sell to

Whoever is taking Otezla off Bristol-Myers Squibb’s hands will have one more revenue stream to boast.

The drug — a rising star in Celgene’s pipeline that generated global sales of $1.6 billion last year — is now OK’d to treat oral ulcers associated with Behçet’s disease, a common symptom for a rare inflammatory disorder. This marks the third FDA approval for the PDE4 inhibitor since 2014, when it was greenlighted for plaque psoriasis and psoriatic arthritis.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors.

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Apotex, though, has been a disaster ground. The manufacturer voluntarily yanked the ANDAs on 31 drugs — in late 2017 — after the FDA came across serious manufacturing deficiencies at their plants in India. A few days ago, the FDA made it official.

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