Look­ing for an ear­ly suc­cess in PhII Alzheimer’s study, Bio­gen and Ei­sai just racked up the lat­est set­back

Chalk up an­oth­er big set­back for the amy­loid be­ta the­o­ry and an­oth­er failed at­tempt at blaz­ing a clin­i­cal path to suc­cess.

In­ves­ti­ga­tors for Bio­gen $BI­IB and Ei­sai say that their an­ti-amy­loid be­ta an­ti­body BAN2401 flunked the 12-month pri­ma­ry end­point in their Phase II study. They had hoped that us­ing Bayesian analy­sis meth­ods would set them up for an ear­ly win, based on a new goal for suc­cess us­ing a com­pos­ite dis­ease score with the pa­tients en­rolled. Now they will go on to the full 18 month mark and then run a check of bio­mark­ers for the dis­ease — which has proven to be a re­mark­ably un­re­li­able as­sess­ment for clin­i­cal ef­fi­ca­cy.

Bio­gen shares dropped more than 4% this morn­ing.

Lynn Kramer, Ei­sai

Still, the biotechs aren’t giv­ing up, hold­ing out hope that they can move in­to late-stage test­ing — a mine field for com­pa­nies in the space which no one has suc­cess­ful­ly nav­i­gat­ed in the past 15 years.

The no­tion that elim­i­nat­ing the tox­ic tan­gles seen in many, though not all, pa­tients di­ag­nosed with this mem­o­ry-wast­ing dis­ease has cost bil­lions of dol­lars of re­search cash in re­cent years. Eli Lil­ly tried and failed with solanezum­ab three times, and still has work on­go­ing. Mer­ck re­cent­ly failed on the first step with their BACE ap­proach and Vivek Ra­maswamy’s Ax­o­vant tanked af­ter they took an­oth­er failed ap­proach on a dif­fer­ent strat­e­gy for amp­ing cog­ni­tion.

With the po­ten­tial earn­ings from any suc­cess gen­er­al­ly rank­ing in the bil­lions, though, don’t look for any­one in the field to slow down or stop.

Bio­gen and Ei­sai are al­lied on three dif­fer­ent ther­a­pies for Alzheimer’s, with the late-stage ad­u­canum­ab and the BACE drug E2609 in­clud­ed in the pack­age.

For it to be con­sid­ered a suc­cess at 12 months, the da­ta would need to demon­strate a greater than 80% chance of suc­cess for see­ing a greater than 25% re­duc­tion in the dis­ease score. Ever­cor­eISI’s Umer Raf­fat says that we won’t re­al­ly know about the re­sult un­til we get to 18 months, and then he ex­pects to see it move ahead in­to late-stage stud­ies.

“By us­ing Bayesian sta­tis­tics in this unique­ly-de­signed tri­al we had hoped that it would en­able us to demon­strate clin­i­cal suc­cess faster than more tra­di­tion­al study de­signs. We now await the fi­nal study analy­sis which will be con­duct­ed af­ter 18 months of treat­ment, which rep­re­sents an amount of treat­ment time that is con­sid­ered as ap­pro­pri­ate for as­sess­ing ef­fi­ca­cy in dis­ease mod­i­fy­ing agents for Alzheimer’s dis­ease,” said Lynn Kramer, Ei­sai’s R&D chief for neu­rol­o­gy.

UP­DAT­ED: Mer­ck pulls Keytru­da in SCLC af­ter ac­cel­er­at­ed nod. Is the FDA get­ting tough on drug­mak­ers that don't hit their marks?

In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders?

Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.

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Paul Sekhri

The next big biotech su­per­star? Paul Sekhri has some thoughts on that

It occasionally occurs to Paul Sekhri that if they pull this off, his company will be on the front page of the New York Times and a lead story in just about every major news outlet on the planet. He tries not to dwell on it, though.

“I just want to be laser-focused on getting to that point,” Sekhri says, before acknowledging, “Yes, it absolutely crossed my mind.”

Sekhri, a longtime biopharma executive with tenures at Sanofi and Novartis, is now entering year three as CEO of eGenesis, the biotech that George Church protégé Luhan Yang founded to genetically alter pigs so that they can be used for organ transplants. He led them through one megaround and has just closed another, raising $125 million from 17 different investors to push the first-ever (humanized) pig to human transplants into the clinic.

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Amit Munshi, Arena

One of Are­na's top drugs flops in a PhI­Ib study for IBS pain. But re­searchers tease out a pos­si­ble path for­ward as CEO ex­plores 's­trate­gic op­tion­s'

Four years ago, when Arena CEO Amit Munshi cut its ties to a troubled weight drug and doubled down on the pipeline, a cannabinoid receptor 2 agonist figured prominently in the biotech’s future. On Tuesday evening, however, Munshi’s high hopes for the drug took a nasty hit after it failed a Phase IIb study for patients with irritable bowel syndrome pain.

Put through a randomized pace with 273 patients, researchers said it flat failed the primary endpoint among the large group with abdominal pain. But they quickly went on to highlight subgroup data, always a tricky and controversial ploy, where they spotlighted a positive p value for patients with moderate to severe pain who received the high dose of the drug — one of 3 provided in the study.

Bob Nelsen (Photo by Michael Kovac/Getty Images)

With stars aligned and cash in re­serve, Bob Nelsen's Re­silience plans a makeover at 2 new fa­cil­i­ty ad­di­tions to its drug man­u­fac­tur­ing up­start

Bob Nelsen’s new, state-of-the-art drug manufacturing initiative is taking shape.

Just 3 months after gathering $800 million of launch money, a dream team board and a plan to shake up a field where he found too many bottlenecks and inefficiencies for the era of Covid-19, Resilience has snapped up a pair of facilities now in line for a retooling.

The company has acquired a 310,000-square-foot plant in Boston from Sanofi along with a 136,000-square-foot plant in Ontario to add to a network which CEO Rahul Singhvi says is just getting started on building his company’s operations up. The Sanofi deal comes with a contract to continue manufacturing one of its drugs.

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CEO Marco Taglietti (Scynexis)

'N­ev­er been more ur­gent:' Scynex­is looks to tack­le su­per­bug cri­sis with late-stage read­out for an­ti­fun­gal hope­ful

As the superbug crisis heats up around the world, Scynexis says it has new data from two interim analyses that prove its antifungal has the potential to treat a broad range of infections.

“The need for new anti-infectives capable of fighting the most resistant pathogens has never been more urgent as we confront the ongoing COVID-19 global pandemic,” CEO Marco Taglietti said in a statement.

A spot­light schiz­o­phre­nia drug in Neu­ro­crine's $2B Take­da deal flunks its first ma­jor test. But it's not giv­ing up yet

When Takeda spun out a pipeline of experimental psychiatry drugs to Neurocrine in a $2 billion deal amid a post-merger shakeout, R&D chief Andy Plump described the therapies as “very interesting but still difficult.”

On Tuesday, we got some idea of how difficult.

San Diego-based Neurocrine revealed that one of the three spotlight clinical programs they’d acquired failed the primary endpoint in a Phase II trial for schizophrenia, registering a negative outcome on the change from baseline in the positive and negative syndrome scale/negative symptom factor score (PANSS NSFS).

Af­ter bail­ing on Covid-19 vac­cines, Mer­ck will team up with J&J to pro­duce its shot as part of un­usu­al Big Phar­ma pact

Merck took a big gamble when it opted to jump into the Covid-19 vaccine race late, and made an equally momentous decision to back out in late January. Now, looking to chip in on the effort, Merck reportedly agreed to team up with one of the companies that has already crossed the finish line.

President Joe Biden on Tuesday is expected to announce a partnership between drugmakers Merck and Johnson & Johnson to jointly produce J&J’s recombinant protein Covid-19 vaccine that received the FDA’s emergency use authorization Saturday, the Washington Post reported.

Ab­b­Vie tees up a biotech buy­out af­ter siz­ing up their Parkin­son's drug spun out of Ke­van Shokat's lab

AbbVie has teed up a small but intriguing biotech buyout after looking over the preclinical work it’s been doing in Parkinson’s disease.

The company is called Mitokinin, a Bay Area biotech spun out of the lab of UCSF’s Kevan Shokat, whose scientific explorations have formed the academic basis of a slew of startups in the biotech hub. One of Shokat’s PhD students in the lab, Nicholas Hertz, co-founded Mitokinin using their lab work on PINK1 suggesting that amping up its activity could play an important role in regulating the mitochondrial dysfunction contributing to Parkinson’s disease pathogenesis and progression.

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Fi­bro­Gen shares skid low­er as a sur­prise ad­comm rais­es risks on roxa OK

FibroGen will likely have to delay its US rollout for roxadustat once again.

In an unexpected move, the FDA is convening its Cardiovascular and Renal Drugs Advisory Committee to review the NDA in an advisory committee meeting. The date is yet to be confirmed.

Just a few weeks ago, SVB Leerink analyst Geoffrey Porges predicted that the roxa approval could come ahead of the PDUFA date on March 20 — effusive despite already being let down once by the FDA’s extension of its review back in December. AstraZeneca, which is partnered with FibroGen on the chronic kidney disease-related anemia drug, disclosed regulators had requested further clarifying analyses of clinical data.