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.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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So what hap­pened with No­var­tis' gene ther­a­py group? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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FDA hands ac­cel­er­at­ed nod to Seagen, Gen­mab's so­lo ADC in cer­vi­cal can­cer, but com­bo stud­ies look even more promis­ing

Biopharma’s resident antibody-drug conjugate expert Seagen has scored a clutch of oncology approvals in recent years, finding gold in what are known as “third-gen” ADCs. Now, another of their partnered conjugates is ready for prime time.

The FDA on Monday handed an accelerated approval to Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv, or “TV”) in second-line patients with recurrent or metastatic cervical cancer who previously progressed after chemotherapy rather than PD-(L)1 systemic therapy, the companies said in a release.

The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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Lat­est news: It’s a no on uni­ver­sal boost­ers; Pa­tient death stuns gene ther­a­py field; In­side Tril­li­um’s $2.3B turn­around; and more

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