Bio­gen shares tum­ble af­ter key ex­ec notes a change­up to piv­otal ad­u­canum­ab study due to 'vari­abil­i­ty'

With news of Mer­ck’s flag­ship BACE drug go­ing down to com­plete fail­ure in a late-stage Alzheimer’s study still re­ver­ber­at­ing through a dispir­it­ed R&D field, Bio­gen is now pitch­ing in to make things even worse.

Al San­drock, Bio­gen

Shares of Bio­gen $BI­IB tum­bled about 7% Wednes­day af­ter­noon af­ter their CMO Al San­drock shocked some at­ten­dees at the Leerink Health­care Con­fer­ence by say­ing that the com­pa­ny had de­cid­ed to add 510 pa­tients to its piv­otal study for the close­ly-watched drug ad­u­canum­ab.

San­drock told at­ten­dees that they need­ed to add hun­dreds of pa­tients to the study due to some “vari­abil­i­ty” they were see­ing in the da­ta from the first round of pa­tients to reach the pri­ma­ry end­point.

Bio­gen couldn’t have picked a worse time for the dis­clo­sure.

The drum­beat of neg­a­tive re­sults for every late-stage ef­fort aimed at amy­loid be­ta has be­gun to shake a once-sol­id be­lief that the clus­ters of tox­ic pro­tein found in many — though not all — pa­tients had to be the cul­prit for a dis­ease that af­flicts mil­lions of peo­ple the world over.

Ge­of­frey Porges, who fol­lows the drug close­ly, not­ed:

The ex­pan­sion in en­roll­ment de­ci­sion was made by the com­pa­ny af­ter they an­a­lyzed blind­ed re­sults for the per­for­mance of the pri­ma­ry end­point (18 month CDR-SB) in the pa­tients who have reached the pri­ma­ry end­point so far. These ad­di­tion­al pa­tients are re­quired to main­tain 90% pow­er for the tri­als due to “vari­abil­i­ty”. Bio­gen has not seen the da­ta by arm, but to main­tain the pow­er­ing need­ed to add the ad­di­tion­al en­rollees giv­en the vari­ance seen so far. Bio­gen in­di­cat­ed that be­cause of the ac­cel­er­at­ed ear­ly en­roll­ment in the tri­al they could still main­tain ap­prox­i­mate­ly the same tim­ing for full en­roll­ment, and sug­gest­ed that they ex­pect full en­roll­ment “in the sum­mer” and ex­pect fi­nal re­sults now in the ear­ly part of 2020. The com­pa­ny al­so com­ment­ed that the drop out rate had been low­er than ex­pect­ed, and that the rate of se­ri­ous ad­verse events, and ARIA-E in par­tic­u­lar, had been with­in their ex­pec­ta­tions. In the con­text of Mer­ck’s (MP) re­cent dis­ap­point­ment with the Mer­ck BACE in­hibitor verube­ce­s­tat, Bio­gen’s ad­u­canum­ab is one of what is now on­ly a hand­ful of Alzheimer’s med­i­cines still ac­tive in piv­otal tri­als.

The pos­i­tive da­ta that Bio­gen had dis­closed from small­er stud­ies had gone a long way to rais­ing hopes that Bio­gen could ac­tu­al­ly make a break­through here, even as se­ri­ous safe­ty is­sues arose. Any­thing that rais­es doubts about ef­fi­ca­cy can’t help Bio­gen, even as it in­sists that the com­pa­ny re­mains con­fi­dent in the amy­loid be­ta the­o­ry.

Michael Yee at Jef­feries not­ed this af­ter­noon that the study da­ta re­mains blind­ed and is even less like­ly to read out sig­nif­i­cant da­ta points be­fore it’s com­plet­ed way out in 2020. He sees to­day’s event as a wake up call for Bio­gen to get busy and buy some­thing the mar­ket won’t fret about.

Mar­ket will swing back to un­cer­tain­ty on the Alzheimer’s pen­du­lum based on tri­al changes, and giv­en da­ta is not like­ly un­til 2020 (in our view) and an in­ter­im seems un­like­ly giv­en dis­clo­sures about vari­abil­i­ty, while we main­tain a pos­i­tive stance on da­ta in 2020, we think co needs to go out and buy de-risked neu­ro/or­phan com­pa­nies to “change the nar­ra­tive” to be­ing a bi­na­ry Alzheimer’s com­pa­ny to one with prod­ucts even if Alzheimer’s doesn’t work.

Good luck with that.

Oxitec biologist releases genetically modified mosquitoes in Piracicaba, Brazil in 2016 [credit: Getty Images]

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When the hysteria of Zika transmission sprang into the American zeitgeist a few years ago, UK-based Oxitec was already field-testing its male Aedes aegypti mosquito, crafted to possess a gene engineered to obliterate its progeny long before maturation.

But when a group of independent scientists evaluated the impact of the release of these genetically-modified mosquitoes in a trial conducted by Oxitec in Brazil between 2013 and 2015, they found that some of the offspring had managed to survive — prompting them to speculate what impact the survivors could have on disease transmission and/or insecticide resistance.

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There are two updates on the lawsuit against Purdue Pharma over its role in fueling the opioid epidemic, as the Sackler family threatens to walk away from their pledge to pay out $3 billion if a bankruptcy judge does not stop outstanding state lawsuits against them. At the same time, the company has asked permission to pay millions in bonuses to select employees.

Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

Genen­tech sub­mits a plan to near­ly dou­ble its South San Fran­cis­co foot­print

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

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Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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David Grainger [file photo]

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While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

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The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

Jason Kelly. Mike Blake/Reuters via Adobe

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UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.