The drum­beat of Alzheimer’s PhI­II fail­ures con­tin­ues as vTv drug is out­per­formed by a place­bo, shares crash

The ex­ecs at vTv $VTVT have put an­oth­er nail in the cof­fin of re­cy­cled Alzheimer’s drug pro­grams.

The biotech, which float­ed an IPO on the back of an idea that it could take a failed drug from Pfiz­er and make it work based on some ret­ro­spec­tive da­ta analy­sis (sound fa­mil­iar?), re­port­ed Mon­day af­ter­noon that their lead ther­a­py azeli­ragon failed a Phase III study for mild to mod­er­ate Alzheimer’s.

The fail­ure was com­plete, flop­ping on both cog­ni­tion and func­tion, the cur­rent gold stan­dard for an ap­proval that has re­mained elu­sive in this field. The drug arm ac­tu­al­ly did worse than the place­bo group. The com­pa­ny state­ment not­ed:

The azeli­ragon treat­ed group in Part A had a 4.4 point de­cline from base­line in ADAS-Cog and a 1.6 point de­cline from base­line in CDR-sb com­pared to a place­bo de­cline of 3.3 and 1.6 re­spec­tive­ly.

Un­ex­pect­ed? To the con­trary. Prac­ti­cal­ly every­thing thrown at Alzheimer’s over the last 15 years has failed in piv­otal stud­ies. And the lat­est com­pa­ny to try this tack with a dis­card, Ax­o­vant, was crushed by its own re­cent fail­ure. The ex­ecs at vTv can now join that crowd. The biotech’s shares crashed Mon­day evening, evis­cer­at­ing val­ue in a 66% plunge.

The biotech went pub­lic close to three years ago at $15 a share, rais­ing $117 mil­lion to pay for this Phase III. It’s been floun­der­ing ever since, end­ing Mon­day evening at $1.14 a share in af­ter-mar­ket trad­ing.

Their drug tar­gets the re­cep­tor for ad­vanced gly­ca­tion end­prod­ucts — “RAGE” — and about 800 pa­tients were en­rolled for the Phase III pro­gram un­der a spe­cial pro­to­col as­sess­ment struck with the FDA.

The biotech ac­tu­al­ly isn’t fin­ished yet, hop­ing that it can find some sign of suc­cess. It’s un­like­ly any­one will pay much at­ten­tion if they do.

“We will con­tin­ue to an­a­lyze the datasets and trends with­in sub­groups from both Part A and Part B to de­ter­mine if there are po­ten­tial ben­e­fits or fu­ture us­es and ap­pli­ca­tions for azeli­ragon,” said vTv CEO Steve Hol­combe in a state­ment.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

CDC’s Robert Redford, NIAID’s Anthony Fauci, Admiral Brett Giroir at HHS, and FDA’s Stephen Hahn prepare to testify at a House hearing on June 23 (Getty)

'Ex­treme­ly po­lit­i­cal' — Trump neuters FDA's at­tempt to strength­en vac­cine EUA, ques­tions need to length­en process

Stephen Hahn went before a Senate committee Wednesday and declared he’s fighting. “Every one of the decisions we have reached has been made by career FDA scientists based on science and data, not politics,” he exclaimed, adding that “FDA will not permit any pressure from anyone to change that. I will fight for science.”

A few hours later, he was undermined by President Donald Trump when a reporter asked if he was okay with stricter vaccine guidelines that the FDA was said to be cooking up. “That has to be approved by the White House. We may or may not approve it. That sounds like a political move,” he decided.

CEO Markus Warmuth (Monte Rosa)

Monte Rosa rakes in $96M Se­ries B as it pre­pares 'mol­e­c­u­lar glue' plat­form for IND-en­abling stud­ies

About four months after completing an extension to its Series A, Monte Rosa Therapeutics is putting its next foot forward with another heap of cash.

The Boston-based biotech is back with $96 million in Series B financing with a goal to get its lead program ready for IND-enabling studies by the end of the year. Though Monte Rosa is keeping its specific target a secret for now, the company has been researching how to utilize its protein degradation technology in breast cancer and non-small cell lung cancer, among other areas.

Chair of FDA's vac­cine ad­comm — who's al­so a lead in­ves­ti­ga­tor of Mod­er­na's vac­cine — re­cus­es her­self from Covid-19 talks

When the FDA’s Vaccines and Related Biological Products Advisory Committee meets next month to discuss the development and authorization of Covid-19 vaccines, the chairwoman won’t be there.

Hana El Sahly has recused herself from the expert panel’s review of the topic, citing her role as a lead investigator in Moderna’s Phase III trial, Reuters reported. An associate professor of virology and microbiology at Baylor College of Medicine in Houston, El Sahly was appointed the chairwoman last year.

David Berry (Flagship)

Flag­ship's next big tech­no­log­i­cal bet? The cloud

Earlier this month, Flagship announced their big bet on the software half the industry is talking about, launching the AI and machine learning startup. Now, they and a couple other investors are gambling $100 million on a software that much of the public generally thinks of as a cool, IT afterthought: cloud computing.

The idea, says founder and Flagship partner David Berry, is one of scale: The sheer magnitude of biological data that you can store on cloud technology is unprecedented. And that size, when leveraged properly, can allow you to ask questions and form insights that are similarly unprecedented.

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Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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Jim Roberts and Brian Finrow (Lumen Bioscience)

With a $4M fed­er­al grant, Lu­men jumps in­to the Covid-19 treat­ment race

It’s been less than a month since Lumen Bioscience announced a $16 million Series B to engineer spirulina — a nutrient-packed super food — for diseases like traveler’s diarrhea, norovirus and C. difficile colitis. And now, the biotech has pulled in another $4 million to do the same for Covid-19.

The approach is quite similar to other gastrointestinal targets the company is pursuing, co-founders and Brian Finrow and Jim Roberts said. The Seattle-based company is working on a camelid antibody cocktail to combat GI infection common among Covid-19 patients. In a study published in the American Journal of Gastroenterology, a majority of Covid-19 patients showed GI and respiratory symptoms, and 25% had only GI symptoms.

Covid-19 roundup: J&J be­gins piv­otal Phase III tri­al for vac­cine; Con­tro­ver­sial hu­man chal­lenge tri­als to be­gin in Lon­don — re­port

Johnson & Johnson announced it’s beginning a pivotal Phase III trial for its Covid-19 candidate, JNJ-78436735 — the first single-dose vaccine in this stage.

The Phase III trial, dubbed ENSEMBLE, will enroll 60,000 patients worldwide, making it the largest Phase III study of a Covid-19 vaccine to date. J&J said the candidate achieved positive interim results in a Phase I/IIa study, which will be published “imminently.” There’s a possibility that the first batches will be ready for potential emergency use in early 2021, according to the company.

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