Bio­gen hy­pes a fresh set of ear­ly, up­beat re­sults for Alzheimer’s drug

New da­ta from an ear­ly-stage study of Bio­gen’s Alzheimer’s drug ad­u­canum­ab has demon­strat­ed a clear im­pact on de­posits of amy­loid be­ta, the tox­ic tan­gles in the brain sus­pect­ed of trig­ger­ing the mem­o­ry-wast­ing ail­ment. And while they couldn’t pre­cise­ly prove it, the re­searchers spot­light­ed ad­di­tion­al ev­i­dence that the drug might al­so be slow­ing the steady cog­ni­tive de­cline of the peo­ple tak­ing the drug.

Al San­drock, Bio­gen Chief Med­ical Of­fi­cer

The ev­i­dence — a de­tailed re­tread of re­sults that have al­ready been re­viewed — im­me­di­ate­ly trig­gered the kind of re­lent­less hype that has sur­round­ed ad­u­canum­ab from the first ear­ly signs of its abil­i­ty to have an im­pact on the dis­ease. But there are some im­por­tant caveats that en­thu­si­asts nev­er­the­less need to keep in mind.

First, while amy­loid be­ta de­posits are a well de­fined tar­get, it’s al­so not uni­ver­sal­ly ac­cept­ed as the sole cause of Alzheimer’s. There are some who be­lieve it may not be in­volved at all, point­ing to pa­tients who have the clus­ters in their brains but no symp­toms of de­men­tia. In ad­di­tion, the re­searchers care­ful­ly not­ed that the study with 165 pa­tients was not pow­ered to pro­vide clear ev­i­dence of ef­fi­ca­cy.

The en­thu­si­asm among the ex­ecs and re­searchers in­volved, though, was pal­pa­ble. And Bio­gen, which bad­ly needs to whip up some ex­cite­ment for the com­pa­ny’s pipeline, was not in the least bit re­luc­tant to pump the lat­est da­ta set that has now been pub­lished in Na­ture. Chief Med­ical Of­fi­cer Al San­drock re­marked:

“These ear­ly stud­ies of ad­u­canum­ab show its ef­fec­tive­ness in re­mov­ing amy­loid plaque from the brain as well as its po­ten­tial ef­fect on the slow­ing of cog­ni­tive de­cline in pa­tients suf­fer­ing from Alzheimer’s dis­ease. Pub­li­ca­tion in Na­ture is an achieve­ment we share with the many sci­en­tists and clin­i­cal in­ves­ti­ga­tors who con­duct­ed this re­search as well as the pa­tients who vol­un­teered to par­tic­i­pate in our clin­i­cal tri­al; we are grate­ful to all of them.”

There is, how­ev­er, a long way to go from a suc­cess­ful Phase Ib study to a piv­otal win, which is where Bio­gen is now fo­cused.

Shares of Bio­gen re­mained down slight­ly for the day.

In ad­di­tion to the pos­i­tive signs that the high dose was most ef­fec­tive, there were al­so some con­cerns.

Check­ing on safe­ty, 38% of the pa­tients in the high, 10 mg kg arm of the study dropped out, many cit­ing side ef­fects, com­pared to 25% of the place­bo arm.

Bio­gen’s progress on Alzheimer’s has helped whip up fresh en­thu­si­asm for the amy­loid be­ta hy­poth­e­sis, even spurring Roche to keep one of its failed drugs, gan­tenerum­ab, in the pipeline. Sev­er­al late-stage drugs, in­clud­ing solanezum­ab at Eli Lil­ly, al­so at­tack a-be­ta, look­ing to prove their ef­fec­tive­ness in very ear­ly-stage pa­tients, be­fore the dis­ease caus­es se­ri­ous brain dam­age. New imag­ing tech­niques are al­so bet­ter able to iden­ti­fy pa­tients who are most like­ly to ben­e­fit.

Lil­ly is ex­pect­ed to de­liv­er piv­otal da­ta from its sec­ond Phase III pro­gram lat­er in the year. But it’s al­so been back­ing away from a key end­point on dai­ly func­tion, sig­nal­ing that it ex­pects to be able to prove an im­pact on cog­ni­tion.

Bio­gen, mean­while, is see­ing off CEO George Scan­gos at a time the biotech com­pa­ny has been un­der heavy pres­sure to do some­thing now to prove it has im­por­tant new drugs in the pipeline that can make up for flag­ging sales of its flag­ship drug, Tec­fidera. Still some ways from their piv­otal read out, and un­able so far to come up with the kind of deal that can gen­er­ate some cheers from Wall Street an­a­lysts, Bio­gen will set­tle for trum­pet­ing an­oth­er ear­ly snap­shot for ad­u­canum­ab.

Jef­feries’ Bri­an Abra­hams not­ed:

Much of the clin­i­cal de­tails, sub-analy­ses and pre­clin­i­cal da­ta have all been ex­ten­sive­ly pre­sent­ed, and we didn’t de­tect any­thing ma­te­ri­al­ly new in the ar­ti­cle; it did cite cau­tion in in­ter­pret­ing cog­ni­tive end­points giv­en lack of pow­er­ing for these mea­sures and no im­pu­ta­tion for miss­ing val­ues, and point­ed out the pos­si­bil­i­ty the need for MRIs in pa­tients de­vel­op­ing ARIA could have un­blind­ed the study. Still, the dose-de­pen­dent plaque re­duc­tions and cor­re­la­tion be­tween plaque clear­ance and cog­ni­tive ben­e­fits con­tin­ue to look most com­pelling to us, and we con­tin­ue to see a 45% prob­a­bil­i­ty of ul­ti­mate­ly gen­er­at­ing $2B+ rev­enues to BI­IB by 2025.

 

From left to right: Lilian Kim, Associate Director Business Development; John Moller, CEO; Yooni Kim, Executive Director, Asia Operations; Michelle Park, Director South Korea Operations.

Novotech CRO sees 26% growth in Asia tri­al ac­tiv­i­ty from biotechs, but still plen­ty of ca­pac­i­ty

As the Asia-Pacific clinical trials sector continues to grow rapidly, Novotech the Asia-Pacific-based CRO is seeing biotech clinical activity up by 26%. But says there is still plenty of capacity in the region that features advanced medical facilities, supportive regulatory environments, and more than 2.3 billion people, largely treatment naïve, living in urban areas.

China, South Korea and Australia have the most studies registered as recruiting or about to recruit according to ClinicalTrials.Gov.

Pfizer, South San Francisco — Jeff Rumans for Endpoints News

UP­DAT­ED: Pfiz­er takes aim at a flag­ship fran­chise at Sanofi and Re­gen­eron — and scores a few di­rect hits

Count Pfizer in as a top player in the blockbuster game of JAK1 inhibitors.

Over the weekend the pharma giant posted some stellar Phase III efficacy data for their heavyweight contender abrocitinib in atopic dermatitis (eczema) that lines up ahead of a booming Dupixent (dupilumab), a blockbuster in the portfolios of Regeneron and Sanofi. And they put some real distance ahead of Eli Lilly’s trailing Olumiant, which made a delayed initial arrival on the market for rheumatoid arthritis after the FDA hobbled it with some additional hurdles on safety concerns.

JADE-MONO-1 scores well for Pfizer, teeing up what will be an intensely followed breakdown of the JADE MONO-2 data, which the pharma giant recently top-lined as “similar” to the first Phase III when tested against a placebo — a control group that has been easily outclassed by all the drugs in this market niche.

As of now, Pfizer looks to be equipped to run into the review stage — advantaged by a breakthrough therapy designation that is intended to speed up the regulatory process.

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A preda­tor's world? Top an­a­lyst sees the 'haves' and the 'haven't­s' di­verge as biotech bub­bles form — and col­lapse

Josh Schimmer

We’ve all seen the deluge of cash that’s been pouring into biotech from every angle: VCs, IPOs and follow-ons have generated billions in capital for new and emerging drug developers with ready access to some powerful new tech. But Evercore ISI’s Josh Schimmer is asking where we’re headed from here.

His answer is neither apocalyptic nor universally blissful, but if he’s right — and this is a discussion we’re hearing much, much more about at a time of growing economic and industry uncertainty — we may well be at a crossroads that could affect valuations, M&A and the entire global industry that has formed over the past 5 years.

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US mulls tar­iffs on Swiss drug ex­ports, weigh­ing on No­var­tis and Roche –  re­port

The leading Swiss newspaper has reported that the US is considering placing tariffs on pharmaceuticals from Switzerland. Roche and Novartis stock each fell 1% after the news broke.

Neue Zürcher Zeitung reported that US Trade Representative Robert Lighthizer told pharmaceutical representatives the Trump administration was considering the move. Tariffs do not appear to be in the immediate offing, but they would potentially affect Swiss giants Novartis and Roche along with other companies that manufacture in Switzerland, including Merck KGaA and US biotech Biogen, which is currently constructing a new facility in the country.

Neil Kumar, Endpoints

Bridge­Bio drops bid to re­claim Ei­dos af­ter di­rec­tors spurn 3 of­fers

A couple of months ago a newly public BridgeBio turned some heads by disclosing that it had made a bid for subsidiary Eidos Therapeutics in hopes of gobbling up the 34% stake that it doesn’t already own. Two offers later, the parties are calling it off.

A special committee of independent directors at the smaller biotech led by RA Capital’s Rajeev Shah and ex-Portola CEO William Lis first rejected the parent company’s initial offer — which would swap 1.3 BridgeBio shares for each Eidos share — on September 12. In the latest announcement, BridgeBio revealed that it eventually raised the offer to 1.5 shares and made $110 million available for all-cash or mixed consideration options, but Eidos still wasn’t interested.

Mark Foley, Revance

HR vi­o­la­tion push­es Re­vance co-founder out, vault­ing for­mer Zel­tiq chief to the helm

Months after Revance amended the terms of its Botox biosimilar collaboration with Mylan, the Newark, California-based drug developer disclosed its co-founder Dan Browne is stepping down, in what appears to be mysterious circumstances.

The company — which is also developing a rival to Allergan’s formidable Botox franchise — on Monday said Browne is departing “due to misjudgment in handling an employee matter,” that has also culminated in his resignation from Revance’s board of directors.

In-house FDA re­view flags a sus­pi­cious im­bal­ance in deaths as Sh­iono­gi hunts an OK for an­tibi­ot­ic

Shionogi has some big questions to answer if they plan to win an FDA panel’s backing for their new antibiotic.

While investigators have provided positive efficacy data for their new product to treat cases of complex urinary tract infections, an FDA review has flagged an imbalance of deaths between the antibiotic and a control arm. And they want the agency’s outside advisers to take a good hard look at that when they meet on Wednesday.

Cell ther­a­py start­up rais­es $16 mil­lion to fund its quest for the Holy Grail in re­gen­er­a­tive med­i­cine

In 2006, Shinya Yamanaka shook stem cell research with his discovery that mature cells can be converted into stem cells, relieving a longstanding political-ethical blockage and throwing open medical research on everything from curbing eye degeneration to organ printing.

But that process still has pitfalls, including in risk and scalability, and some researchers are exploring another way first hinted at years ago: new technology to convert mature cells directly into other mature cells without the complex and time-consuming process of first making them into stem cells.

Eye­ing $86M, Galera leads a pack of three mod­est biotech IPOs push­ing past high pro­file stum­bles

Exactly one year after kicking off a pivotal Phase III study for its lead drug — a companion for cancer patients receiving radiotherapy — Galera is looking to the Nasdaq for some new cash to complete the clinical work and fuel its commercial drive.

CEO Mel Sorensen has penciled in an $86 million ask, which was filed on the same day as liver disease company 89bio and rare disease diagnostics shop Centogene. The trio marks the first batch of IPO filings in the wake of two highly anticipated but ultimately disappointing public debuts by BioNTech and Vir, signaling dwindling biotech fervor on Wall Street. 89bio and Centogene are seeking $70 million and $69 million, respectively.