Casey Lynch, Cortexyme CEO

Up­dat­ed: Cor­texyme's un­ortho­dox Alzheimer's ap­proach tripped up by FDA hold

The FDA has thrown a mi­nor wrench in Cor­texyme’s un­ortho­dox plan to treat Alzheimer’s.

On Mon­day, the Bay Area biotech an­nounced that the agency had put one of the stud­ies in their piv­otal pro­gram on hold af­ter the com­pa­ny re­port­ed that pa­tients ex­pe­ri­enced liv­er prob­lems while tak­ing their lead drug. The is­sues were re­versible and had no known long-term ef­fects, the com­pa­ny said.

The hold will not af­fect the Phase II/III, blind­ed, ran­dom­ized con­trolled study they hope to read out to­wards the end of this year. In­stead, the agency has de­cid­ed to end, for the time be­ing, an open-la­bel ex­ten­sion study that had been mea­sur­ing their mol­e­cule’s long-term ef­fects on pa­tients who al­ready com­plet­ed the ini­tial 48-week study. No new pa­tients will be en­rolled and pa­tients cur­rent­ly in the tri­al will be dis­con­tin­ued.

“Cor­texyme’s high­est pri­or­i­ty is the safe­ty of study par­tic­i­pants,” Casey Lynch, Cor­texyme’s CEO, co-founder, and chair said in a state­ment.

The hold took out of a quar­ter of Cor­texyme’s $CRTX mar­ket cap, drop­ping shares from $47.47 to $36.

Cor­texyme, which has been backed by Pfiz­er and Take­da Ven­tures, emerged from stealth mode in 2018 to pur­sue an al­ter­na­tive ap­proach to Alzheimer’s. While most com­pa­nies tried to di­rect­ly cleave the amy­loid plaques that build up in pa­tients’ brains, gen­er­al­ly floun­der­ing in the process, Lynch pur­sued an idea tout­ed by a hand­ful of rene­gade neu­ro­sci­en­tists: that Alzheimer’s was caused by an in­fec­tious agent and the body’s in­flam­ma­to­ry re­sponse to that agent.

Specif­i­cal­ly, Cor­texyme has gone af­ter P. gin­gi­valis, one of the bac­te­ria that caus­es gum dis­eases. The bac­te­ria has been found in the brain of Alzheimer’s pa­tients and shown to lead to Alzheimer’s in mice mod­els.  Their drug, COR388, blocks a pro­tease se­cret­ed P. gin­gi­valis, in­hibit­ing their abil­i­ty to dam­age cells and slow­ly starv­ing them by pre­vent­ing them from get­ting food from those cells.

Al­though the hold marks a sig­nif­i­cant set­back, the big­ger test will come at the end of the year, when Cor­texyme plans to an­nounce whether COR388 slowed cog­ni­tive de­cline in a Phase II/III tri­al. The study, which the com­pa­ny be­lieves will be piv­otal, is al­ready ful­ly en­rolled with 643 pa­tients.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

Up­dat­ed: Al­ny­lam re­in­forces APOL­LO-B patisir­an da­ta be­fore head­ing to the FDA

Weeks after uncorking some mostly positive data for patisiran in transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, Alnylam is bolstering its package with new exploratory and subgroup data before shipping it off to regulators.

The RNAi drug maintained “generally consistent” benefits in efficacy and quality of life across several prespecified subgroups at month 12, Alnylam announced on Friday afternoon, including age, baseline tafamidis use, ATTR amyloidosis type, baseline six-minute walk test score and others.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Jerome Durso, Intercept Pharmaceuticals CEO

In­ter­cep­t's OCA fails a PhI­II NASH tri­al, rais­ing fresh doubts about its years­long quest for an OK

Intercept Pharmaceuticals has run into another big setback in its yearslong quest to win an approval for OCA in NASH. The biotech put out word Friday morning that its Phase III REVERSE study failed the primary endpoint for the liver disease, sending its share price into a tailspin.

There was no significant improvement in fibrosis among the patients suffering from cirrhosis who were treated with obeticholic acid, with investigators hunting for a minimum 1-stage histological improvement in the disease after 18 months of therapy.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

Bo Cumbo, new Solid Bio CEO

Sol­id Bio gets a new CEO, $75M cash and drops lead drug as big in­vestors ju­ry-rig a merg­er deal

Three months after Endpoints News broke the story that gene therapy outfit AavantiBio had gutted its CMC group in a reorganization, the biotech’s CEO has helped engineer a merger with struggling penny stock player Solid Bio. And he’s going to remain in charge of the combined operation, as Solid founder Ilan Ganot steps aside.

The merger news this morning features some high-profile investors.

Perceptive Advisors, RA Capital Management and Bain Capital Life Sciences are leading a $75 million raise to add to the pool of cash Solid will have after the tie-up. That will leave Solid $215 million in cash as Bain’s Adam Koppel jumps on the board — enough to pay for ops and get through some key data milestones on their way into 2025.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.