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.

As­traZeneca trum­pets the good da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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The Advance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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Bryan Roberts, Venrock

Ven­rock sur­vey shows grow­ing recog­ni­tion of coro­n­avirus toll, wan­ing con­fi­dence in ar­rival of vac­cines and treat­ments

When Venrock partner Bryan Roberts went to check the results from their annual survey of healthcare leaders, what he found was an imprint of the pandemic’s slow arrival in America.

The venture firm had sent their form out to hundreds of insurance and health tech executives, investors, officials and academics on February 24 and gave them two weeks to fill it out. No Americans had died at that point but the coronavirus had become enough of a global crisis that they included two questions about the virus, including “Total U.S. deaths in 2020 from the novel coronavirus will be:”.

Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

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David Hoey (Vaxxas)

In for the long vac­cine game, Mer­ck buys in­to patch de­liv­ery tech with pan­dem­ic po­ten­tial

When Merck dived into the R&D fray for a Covid-19 vaccine earlier this week, execs made it clear that they’re not necessarily looking to be first — with CEO Ken Frazier throwing cold water on the hotly-discussed 12- to 18-month timelines. But when it does emerge from behind, the pharma giant clearly expects to play a significant part.

Part of that will depend on next-generation delivery technology that reshapes the world’s imagination of a vaccine.

No­var­tis jumps in­to Covid-19 vac­cine hunt, as Big Phar­ma and big biotech com­mit to bil­lions of dos­es

After spending most of the pandemic on the sidelines, Novartis is offering its aid in the race to develop a Covid-19 vaccine.

AveXis, the Swiss pharma’s gene therapy subsidiary, has agreed to manufacture the vaccine being developed by Massachusetts Eye and Ear and Massachusetts General Hospital. The biotech will begin manufacturing this month, while the vaccine undergoes further preclinical testing. They’ve agreed to provide the vaccine for free for clinical trials beginning in the second half of 2020, but have not disclosed financials for after.

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