RIP amy­loid be­ta the­o­ry? Nope. Bio­gen part­ner launch­es a new PhI­II be­fore ad­u­canum­ab's corpse turned cold

A day af­ter Bio­gen rat­tled the bio­phar­ma world with the news that its lead late-stage ther­a­py ad­u­canum­ab proved worth­less in treat­ing Alzheimer’s — a dis­as­ter that may dri­ve a stake through the heart of the amy­loid be­ta the­o­ry once and for all — the big biotech’s part­ners at Ei­sai have come up with their next big move.


Right in the wake of a 35% plunge in their stock val­ue, Ei­sai $ESALY is start­ing a Phase III study in­volv­ing 1,566 Alzheimer’s pa­tients with mild cog­ni­tive im­pair­ment for the con­tro­ver­sial Alzheimer’s drug BAN2401. The an­ti-amy­loid an­ti­body was the cen­ter of a firestorm of crit­i­cism over a tardy re­veal that re­searchers had pulled high-risk pa­tients out of their last study, po­ten­tial­ly warp­ing the pos­i­tive re­sults that were claimed, leav­ing that drug un­der a dark cloud.

“We still be­lieve that amy­loid be­ta hy­poth­e­sis is po­ten­tial­ly the right ap­proach for the treat­ment of Alzheimer’s dis­ease,” an Ei­sai spokesman told Reuters.

That’s a po­si­tion that Bio­gen ex­ecs will find tough to jus­ti­fy to­day. A whole slate of ma­jor de­vel­op­ers — Eli Lil­ly and As­traZeneca, Mer­ck and Roche — have re­port­ed out de­ci­sive late-stage fail­ures over the last year that all point to one con­clu­sion: Tar­get­ing amy­loid be­ta alone in symp­to­matic pa­tients may hit your bio­mark­ers on ef­fect, but it doesn’t de­lay the ruth­less march of the dis­ease.

Ei­sai and Bio­gen may not have re­ceived the memo, but a whole host of an­a­lysts have writ­ten off BAN2401 as a los­er. As for this new move, don’t ex­pect any sup­port from Leerink’s Ge­of­frey Porges, who es­sen­tial­ly be­lieves any new work like this is dam­ag­ing to in­vestors and pa­tients. He not­ed this morn­ing:

We as­sume that the com­pa­ny (Bio­gen) takes the re­spon­si­ble de­ci­sion to ter­mi­nate all fur­ther in­vest­ments in be­ta amy­loid-di­rect­ed med­i­cines (which has not oc­curred), and saves their in­vestors the cash and saves pa­tients and in­ves­ti­ga­tors from the bur­den of such stud­ies. If Bio­gen does not make this de­ci­sion, then our ad­just­ed ex­pense fore­cast could be too low, with fur­ther neg­a­tive ef­fects on our val­u­a­tion even com­pared to our new PT.

What will be ahead for Bio­gen? Porges is tak­ing a hard line. It’s worth quot­ing him at length.

In our view, Bio­gen finds it­self in the un­en­vi­able po­si­tion of be­ing a wound­ed cash cow (which we are sur­prised man­age­ment and the board did not con­sid­er as a po­ten­tial out­come and pre­pare for). The next few months are like­ly to con­sist of a mix­ture of re­crim­i­na­tions, ex­pla­na­tions, ne­go­ti­a­tions, and pos­si­bly ter­mi­na­tions and lit­i­ga­tion. We ex­pect the com­pa­ny to be dis­tract­ed and ham­pered by those over­hangs for a cou­ple of quar­ters at least. Con­trary to pop­u­lar be­lief we don’t be­lieve that their board or man­age­ment will have the lat­i­tude to im­me­di­ate­ly piv­ot to ma­jor ac­qui­si­tions that would al­ter the com­pa­ny’s out­look ma­te­ri­al­ly (de­spite our sug­ges­tions in the past that such in­vest­ments were ad­vis­able). To­ward the end of this year, af­ter the dust of this dis­ap­point­ment has like­ly set­tled, we ex­pect Bio­gen to ex­plore both as­set sales and as­set pur­chas­es (af­ter oth­er changes have been made, or im­posed by in­vestors). We be­lieve that the case for re­struc­tur­ing and di­vesti­tures will be as com­pelling as the case for ac­qui­si­tions, par­tic­u­lar­ly in the con­text of such poor re­turns from the com­pa­ny’s re­cent cap­i­tal al­lo­ca­tion de­ci­sions.

Bio­gen shares $BI­IB tum­bled 29% on Thurs­day, wip­ing out $18 bil­lion in mar­ket cap. They won’t get any of that back based on the launch of the BAN2401 tri­al. To the con­trary. At a time they need to re­build con­fi­dence, there’s no sign that the part­ners learned any­thing this week.

FDA chief Stephen Hahn on Capitol Hill earlier this week (Getty Images)

As FDA’s work­load buck­les un­der the strain, Trump again ac­cus­es the agency of a po­lit­i­cal hit job

Peter Marks appeared before a virtual SVB Leerink audience yesterday and said that his staff at FDA’s CBER is on the verge of working around the clock. Manufacturing inspections, policy work and sponsor communications have all been pushed down the to-do list so that they can be responsive to Covid-related interactions. And the agency’s objective right now? “To save as many lives as we can,” Marks said, likening the mortality on the current outbreak as equivalent to “a nuclear bomb on a small city.”

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

The win­dow is wide open as four more biotechs join the go-go IPO class of 2020

It’s another day of hauling cash in the biopharma world as four more IPOs priced Friday and a fifth filed its initial paperwork.

The biggest offering comes from PMV Pharma, an oncology biotech focusing on p53 mutations, which raised $211.8 million after pricing shares at $18 apiece. Prelude Therapeutics, developing PRMT5 inhibitors for rare cancers, was next with a $158 million raise, pricing shares at $19 each. Graybug Vision raised $90 million after pricing at $16 per share for its wet AMD candidates, and breast cancer biotech Greenwich Lifesciences brought up the rear with a small, $7 million raise after pricing shares at $5.75.

J&J of­fers PhI/IIa da­ta show­ing its sin­gle-dose vac­cine can stir up suf­fi­cient im­mune re­sponse

Days after J&J dosed the first participants of its Phase III ENSEMBLE trial, the pharma giant has detailed the early-stage data that gave them confidence in a single-dose regimen.

Testing two dose levels either as a single dose or in a two-dose schedule spaced by 56 days in, the scientists from Janssen, the J&J subsidiary developing its vaccine, reported that the low dose induced a similar immune response as the high dose. The interim Phase I/IIa results were posted in a preprint on medRxiv.

Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

Play-by-play of Gilead­'s $21B Im­munomedics buy­out de­tails a fren­zied push — and mints a new biotech bil­lion­aire

Immunomedics had not really been looking for a buyout when the year began. Excited by its BLA for Trodelvy, submitted to the FDA in late 2019, executive chairman Behzad Aghazadeh started off looking for potential licensing deals and zeroed in on four potential partners, including Gilead, following January’s JP Morgan Healthcare Conference in San Francisco. Such talks advanced throughout the year, with discussions advancing to the second round in mid-August.

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President Donald Trump reacts after signing an executive order following his remarks on his healthcare policies yesterday in Charlotte, North Carolina (Getty Images)

Op-ed: Will phar­ma re­al­ly pay for Trump’s lat­est law­less promise to 33 mil­lion Medicare ben­e­fi­cia­ries? Not like­ly

Sitting atop the executive branch, President Donald Trump is the ultimate authority at the FDA. He can fast track any vaccine to approval himself. If it came to that, of course.

What he can’t do is unilaterally order the legislative branch to loosen the Treasury’s coffers for $6.6 billion. Nor can he command pharmaceutical companies to pay for $200 vouchers sent to 33 million Medicare beneficiaries for prescription drugs before the election.

New York governor Andrew Cuomo (AP Images)

An­drew Cuo­mo says New York will un­der­take its own vac­cine re­view process, and wouldn’t rec­om­mend trust­ing the fed­er­al gov­ern­ment

The concerns keep mounting over President Donald Trump’s politicization of the FDA and other federal agencies guiding the development of a safe and effective vaccine. And today, the telegenic New York governor Andrew Cuomo appeared to introduce even more politics into the matter — latest in an ongoing series of incidents that have cast the proudly independent FDA in starkly political terms.

During his daily press conference Cuomo said that the state will review any coronavirus vaccines approved by the federal government, citing a lack of trust in the Trump administration. The announcement comes one day after Trump accused the FDA of making an “extremely political” move in proposing stricter vaccine guidance.

President Trump walks past HHS secretary Alex Azar (Getty Images)

Azar falls in line un­der Trump again. Ex­perts say he's re­in­forc­ing a dark sig­nal sent to the FDA

In the latest incident where Alex Azar has steadfastly taken the side of President Donald Trump over that of the FDA, the HHS secretary was noncommittal this morning when asked if he supports the attempt by his subordinates at the FDA to strengthen guidelines for a vaccine EUA.

Appearing on NBC’s Today Show, the HHS secretary muddied the waters, stating that the guidance that matters is the one that is “actually already out there.”

Covid-19 roundup: Op­er­a­tion Warp Speed's 7th vac­cine is live at­ten­u­at­ed; Small biotech touts big suc­cess where gi­ants have failed

Operation Warp Speed is stacking its vaccine portfolio with a “TBD” new candidate: a live attenuated vaccine that can be administered in a single dose, potentially as an oral formulation rather than an injection.

Sound familiar?

That could be because the unannounced candidate appears to match the profile of an inoculation being developed by Merck, according to Bloomberg, which first reported the development based on a presentation by Moncef Slaoui.

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