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.

Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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Donald and Melania Trump watch the smoke of fireworks from the South Lawn of the White House on July 4, 2020 (via Getty)

Which drug de­vel­op­ers of­fer Trump a quick, game-chang­ing ‘so­lu­tion’ as the pan­dem­ic roars back? Eli Lil­ly and Ab­Cellera look to break out of the pack

We are unleashing our nation’s scientific brilliance and will likely have a therapeutic and/or vaccine solution long before the end of the year.

— Donald Trump, July 4

Next week administration officials plan to promote a new study they say shows promising results on therapeutics, the officials said. They wouldn’t describe the study in any further detail because, they said, its disclosure would be “market-moving.”

— NBC News, July 3

Something’s cooking. And it’s not just July 4 leftovers involving stale buns and uneaten hot dogs.

Over the long weekend observers picked up signs that the focus in the Trump administration may swiftly shift from the bright spotlight on vaccines being promised this fall, around the time of the election, to include drugs that could possibly keep patients out of the hospital and take the political sting out of the soaring Covid-19 numbers causing embarrassment in states that swiftly reopened — as Trump cheered along.

So far, Gilead has been the chief beneficiary of the drive on drugs, swiftly offering enough early data to get remdesivir an emergency authorization and into the hands of the US government. But their drug, while helpful in cutting stays, is known for a limited, modest effect. And that won’t tamp down on the hurricane of criticism that’s been tearing at the White House, and buffeting the president’s most stalwart core defenders as the economy suffers.

We’ve had positive early-stage vaccine data, most recently from Pfizer and BioNTech, playing catchup on an mRNA race led by Moderna — where every little sign of potential trouble is magnified into a lethal threat, just as every advance excites a frenzy of support. But that race still has months to play out, with more Phase I data due ahead of the mid-stage numbers looming ahead. A vaccine may not be available in large enough quantities until well into 2021, which is still wildly ambitious.

So what about a drug solution?

Trump’s initial support for a panacea focused on hydroxychloroquine. But that fizzled in the face of data underscoring its ineffectiveness — killing trials that aren’t likely to be restarted because of a recent population-based study offering some support. And there are a number of existing drugs being repurposed to see how they help hospitalized patients.

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In­vestors give ail­ing Unum a lease on life and a whole new suite of ex­per­i­men­tal can­cer drugs

Investors, it seems, are willing to give Unum Therapeutics one last shot — or at least one last shot to a company of that name.

The ailing cancer biotech, beset by a series of clinical holds and multiple failed lead programs, announced today that they’ve acquired Kiq LLC and that investors are putting in $104 million to advance Kiq’s pipeline of kinase inhibitors. Unum shareholders will now own only 16.2% of the company and CEO Chuck Wilson indicated that the cell therapies the biotech has worked on since its founding may be on their way out, saying Unum will “explore strategic options” for those products.

RA Cap­i­tal dou­bles down on Sid­dhartha Mukher­jee's vi­sion for a new cell en­gi­neer­ing ap­proach, lead­ing Vor's $110M Se­ries B

Vor Biopharma is muscling up.

CEO Robert Ang, who was reluctant to divulge the headcount when discussing his move from Neon Therapeutics to Vor last August, readily offered that the team has grown from 6 to 50 in less than a year. The biotech is moving to a larger office on Cambridge Parkway Drive in weeks, giving it more space to complete the IND-enabling work and manufacturing scale-up — conducted by a CDMO partner — in preparation for clinical trials planned for the first half of 2021.

Covid-19 roundup: Left out no longer, No­vavax se­cures largest Warp Speed deal yet: $1.6B

It looks like Novavax won’t be left out of Operation Warp Speed after all.

A month after the Gaithersburg, MD biotech saw its shares tumble when it was left off the first reported list of finalists for the White House’s Covid-19 vaccine accelerator, HHS and the Department of Defense have announced a $1.6 billion deal to scale up their Covid-19 candidate. It is the largest deal HHS has announced yet, eclipsing the $1.2 billion deal the administration reached with AstraZeneca in May.

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Cel­lec­tis slammed af­ter pa­tient dies and FDA slaps a hold on their tri­al for an off-the-shelf CAR-T for mul­ti­ple myelo­ma

Cellectis was slammed after the market close on Monday as the biotech reported that the FDA demanded it hit the brakes on their MELANI-01 trial for their off-the-shelf cell therapy UCARTCS1A after one of the patients in the study died of treatment-related cardiac arrest.

The multiple myeloma patient had previously been treated unsuccessfully with various therapies, noted the biotech, and had been given dose level two (DL2) of their allogeneic CAR-T.

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Shoshanna Shendelman, Applied Therapeutics CEO (Applied Therapeutics)

A lit­tle biotech slaps back at a 'crim­i­nal' short at­tack, vow­ing to pur­sue a pros­e­cu­tion of their case

As short attacks go, Biotech Research Partners’ assault on Applied Therapeutics’ “cherry picked” data and a variety of so-called red flags didn’t cause a whole lot of damage. Ahead of the July 4 holiday, its shares $APLT were dinged and showed signs of quick recovery.

But that didn’t stop an incendiary response, as the biotech swung into action bright and early Monday morning.

Applied Therapeutics accused the authors of the short report of manipulating graphs and figures, misrepresenting data and included factual misrepresentations — all of which added up, in their view, to fraud.

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Bill Haney, Dragonfly CEO (Dave Pedley/Getty Images for SXSW)

A boom­ing Drag­on­fly is tak­ing its TriN­KETs to Copen­hagen as the lat­est Bris­tol My­ers pact spurs ex­pan­sion plans — out­side the US

Bristol Myers Squibb is making a habit out of collaborating with the crew at Dragonfly, adding their 3rd deal in a series that now will take them into newly charted R&D territory. And the fast-growing team at the Cambridge-based biotech is adding a facility in Copenhagen for its next growth spurt, where the government is making it easy to recruit scientists internationally as the U.S. throttles back.

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Covid-19 roundup: Teamed up with NIH, Re­gen­eron launch­es PhI­II pre­ven­tion tri­al for an­ti­body cock­tail

As Regeneron moves its antibody cocktail into Phase II/III trials testing REGN-COV2 as a treatment for both hospitalized and non-hospitalized patients with Covid-19, the biotech is also starting a Phase III in the prevention setting.

The National Institute of Allergy and Infectious Diseases — which orchestrated the large, randomized study for remdesivir that produced positive results — will jointly run the study.

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