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.

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

CDC’s Robert Redford, NIAID’s Anthony Fauci, Admiral Brett Giroir at HHS, and FDA’s Stephen Hahn prepare to testify at a House hearing on June 23 (Getty)

'Ex­treme­ly po­lit­i­cal' — Trump neuters FDA's at­tempt to strength­en vac­cine EUA, ques­tions need to length­en process

Stephen Hahn went before a Senate committee Wednesday and declared he’s fighting. “Every one of the decisions we have reached has been made by career FDA scientists based on science and data, not politics,” he exclaimed, adding that “FDA will not permit any pressure from anyone to change that. I will fight for science.”

A few hours later, he was undermined by President Donald Trump when a reporter asked if he was okay with stricter vaccine guidelines that the FDA was said to be cooking up. “That has to be approved by the White House. We may or may not approve it. That sounds like a political move,” he decided.

CEO Markus Warmuth (Monte Rosa)

Monte Rosa rakes in $96M Se­ries B as it pre­pares 'mol­e­c­u­lar glue' plat­form for IND-en­abling stud­ies

About four months after completing an extension to its Series A, Monte Rosa Therapeutics is putting its next foot forward with another heap of cash.

The Boston-based biotech is back with $96 million in Series B financing with a goal to get its lead program ready for IND-enabling studies by the end of the year. Though Monte Rosa is keeping its specific target a secret for now, the company has been researching how to utilize its protein degradation technology in breast cancer and non-small cell lung cancer, among other areas.

Chair of FDA's vac­cine ad­comm — who's al­so a lead in­ves­ti­ga­tor of Mod­er­na's vac­cine — re­cus­es her­self from Covid-19 talks

When the FDA’s Vaccines and Related Biological Products Advisory Committee meets next month to discuss the development and authorization of Covid-19 vaccines, the chairwoman won’t be there.

Hana El Sahly has recused herself from the expert panel’s review of the topic, citing her role as a lead investigator in Moderna’s Phase III trial, Reuters reported. An associate professor of virology and microbiology at Baylor College of Medicine in Houston, El Sahly was appointed the chairwoman last year.

David Berry (Flagship)

Flag­ship's next big tech­no­log­i­cal bet? The cloud

Earlier this month, Flagship announced their big bet on the software half the industry is talking about, launching the AI and machine learning startup. Now, they and a couple other investors are gambling $100 million on a software that much of the public generally thinks of as a cool, IT afterthought: cloud computing.

The idea, says founder and Flagship partner David Berry, is one of scale: The sheer magnitude of biological data that you can store on cloud technology is unprecedented. And that size, when leveraged properly, can allow you to ask questions and form insights that are similarly unprecedented.

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Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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Jim Roberts and Brian Finrow (Lumen Bioscience)

With a $4M fed­er­al grant, Lu­men jumps in­to the Covid-19 treat­ment race

It’s been less than a month since Lumen Bioscience announced a $16 million Series B to engineer spirulina — a nutrient-packed super food — for diseases like traveler’s diarrhea, norovirus and C. difficile colitis. And now, the biotech has pulled in another $4 million to do the same for Covid-19.

The approach is quite similar to other gastrointestinal targets the company is pursuing, co-founders and Brian Finrow and Jim Roberts said. The Seattle-based company is working on a camelid antibody cocktail to combat GI infection common among Covid-19 patients. In a study published in the American Journal of Gastroenterology, a majority of Covid-19 patients showed GI and respiratory symptoms, and 25% had only GI symptoms.

Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

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