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.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

Stéphane Bancel, Moderna CEO

'This is not go­ing to be good': Mod­er­na CEO Ban­cel warns of a 'ma­te­r­i­al drop' in vac­cine ef­fi­ca­cy as Omi­cron spreads

Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

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Ap­peals court puts the fi­nal nail in the cof­fin for Tec­fidera patent, adding to Bio­gen's bur­geon­ing set­backs

In another setback for Biogen, the big biotech lost its appeal to revive a patent for the once-blockbuster drug Tecfidera, marking a likely conclusion to the case.

The US Court of Appeals for the Federal Circuit issued the ruling Tuesday morning, saying Biogen failed to satisfy the “written description” requirement for patent law. As a result, Mylan-turned-Viatris will be able to sell its multiple sclerosis generic without fear of infringement and Biogen will have to find a new revenue driver elsewhere.

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Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.

Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

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In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

Tillman Gerngross (Adagio)

Till­man Gern­gross on Omi­cron: 'It is a grim sit­u­a­tion...we’re go­ing to see a sig­nif­i­cant drop in vac­cine ef­fi­ca­cy'

Tillman Gerngross, the rarely shy Dartmouth professor, biotech entrepreneur and antibody expert, has been warning for over a year that the virus behind Covid-19 would likely continue to mutate, potentially in ways that avoid immunity from infection and the best defenses scientists developed. He spun out a company, Adagio, to build a universal antibody, one that could snuff out any potential mutation.

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In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.

Mar­ket­ingRx roundup: Ab­b­Vie’s Hu­mi­ra TV turns fo­cus to HS skin con­di­tion; Sanofi amps par­ent­ing pol­i­cy

After years as the top spending pharma TV advertiser, AbbVie’s Humira brand finally downshifted earlier this year, ceding much of its marketing budget to up-and-coming sibling meds Skyrizi and Rinvoq. However, now Humira is back on TV with ads for another condition — Hidradenitis suppurativa (HS).

The chronic and painful skin condition results in lumps and abscesses caused by inflammation or infection of sweat glands, most often in the armpits or groin. Humira was first approved to treat HS in 2015 and remains the only FDA-approved drug for the condition. Two TV ads both note more than 30,000 people with HS have been prescribed Humira.

As lead drug runs in­to a wall, De­ci­phera slims down its pipeline, puts 140 jobs on the chop­ping block

Barely a month after disappointing data shattered hopes for a major label expansion for the GI tumor drug Qinlock, Deciphera is making a major pivot — scrapping development plans for that drug and discarding another while it hunkers down and focuses on two remaining drugs in the pipeline.

As a result, 140 of its staffers will be laid off.

The restructuring, which claims the equivalent of 35% of its total workforce, will take place across all departments including commercial, R&D as well as general and administrative support functions, Deciphera said, as it looks to streamline Qinlock-related commercial operations in the US while concentrating only on a “select number of key European markets.”

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