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.


Lessons for biotech and phar­ma from a doc­tor who chased his own cure

After being struck by a rare disease as a healthy third year medical student, David Fajgenbaum began an arduous journey chasing his own cure. Amidst the hustle of this year’s JP Morgan conference, the digital trials platform Medable partnered with Endpoints Studio to share Dr. Fajgenbaum’s story with the drug development industry.

What follows is an edited transcript of the conversation between Medable CEO Dr. Michelle Longmire and Dr. Fajgenbaum, and it is full of lessons for biotech executives charged with bringing the next generation of medicines to patients.

Christos Kyratsous (via LinkedIn)

He built a MERS treat­ment in 6 months and then the best Ebo­la drug. Now Chris­tos Kyrat­sous turns his sights on Covid-19

TARRYTOWN, NY — In 2015, as the Ebola epidemic raged through swaths of West Africa, Kristen Pascal’s roommates sat her down on their couch and staged an intervention.

“Are you sure this is what you want to be doing with your life?” she recalls them asking her.

Pascal, a research associate for Regeneron, had been coming home at 2 am and leaving at 6 am. At one point, she didn’t see her roommate for a week. For months, that was life in Christos Kyratsous’ lab as the pair led a company-wide race to develop the first drug that could effectively treat Ebola before the outbreak ended. For Pascal, that was worth it.

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Left to right, top to bottom: Carl Gordon, Adam Stone, Peter Moglia, David Schenkein, Robert Nelsen, Carol Gallagher; Srinivas Akkaraju, Ray Debbane, Jim Flynn, Peter Kolchinsky, Thilo Schroeder, Brad Bolzon

The top 100 bio­phar­ma ven­ture in­vestors at the mega­bil­lions deal ta­ble

The VC crowd took a step back last year, but nevertheless maintained a furious pace of new investments in therapeutic tech platforms and biotech startups. And the top 100 players completely dominated the megabillions game.

Just looking at the number of deals done by each of the top 100, OrbiMed came in at the top, with 20, followed by Alexandria (18), Perceptive (16) and the ubiquitous RA Capital at 16. It’s impossible to say exactly how much they invested in total — those numbers are only rarely provided — but it is clear from the numbers assembled by Chris Dokomajilar at DealForma who’s most likely to be found sitting at the table during the go-go days of biotech investing.

Dokomajilar tracked $14.06 billion in biotech venture investing last year, a dip from the frenzied pace of $16.02 billion in 2018 and more than $10 billion higher than he recorded for 2010, as the economy was recovering from a profound economic crisis.

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Rahul Ballal, Imara

As sick­le cell pa­tients find new op­tions, NEA-found­ed Imara pitch­es mid-stage al­ter­na­tive for $86M IPO

November 2019 proved to be a fruitful month for patients with blood disorders known as hemoglobinopathies. Within days, the FDA ushered two drugs for sickle cell disease and another for beta thalassemia to the market — livening up a barren field.

Imara, a relatively young plower, is riding on that enthusiasm as it shoots for an $86.25 million IPO.

Imara emerged from New Enterprise Associates’ orphan drug accelerator Cydan in 2016 as a single-product company. $77.3 million in private financing later IMR-687 remains the sole asset in its pipeline; the difference is the drug is now in Phase II for sickle cell disease, with topline data slated for later this year and two other mid-stage beta thalassemia studies lined up.

RA joins glob­al syn­di­cate to back a $98M round for CAN­bridge

A Beijing-based rare disease and oncology player has raised $98 million to help fund the expansion of its pipeline as well as a commercial portfolio.

CANbridge put out word Tuesday that the global private equity player General Atlantic joined forces with Chinese CRO Wuxi AppTec to lead the Series D, with both ready to chip in an extra $10 million each under the right conditions. The syndicate includes RA Capital Management, Hudson Bay Capital Management, YuanMing Prudence Fund and Tigermed.

Carol Robinson, Professor Dame Carol Robinson Research Group

UP­DAT­ED: Drug dis­cov­ery in HD: Ox­ford spin­of­f's mass spec­trom­e­try ap­proach scores fresh fund­ing

The technology used to detect explosives at airports — mass spectrometry — is being piloted as an engine for drug discovery.

Mass spectrometry is a tool designed to measure with profound accuracy the mass of a single molecule. Typically, mass spectrometers can be used to identify unknown compounds, to quantify known compounds, and to determine the structure and chemical properties of molecules.

UP­DAT­ED: Chi­na ap­proves flu drug be­ing tout­ed as a po­ten­tial coro­n­avirus treat­ment amid a rush of clin­i­cal stud­ies

One of the three drugs that China’s Ministry of Science and Technology has tapped as potential COVID-19 treatments to watch has notched its first Chinese OK — for the flu.

While there’s no proof yet that fapilavir, or favipiravir, is the cure that patients and physicians are yearning for, it stands out for a unique constellation of qualities. It’s been commercially available in Japan for several years (unlike Gilead’s experimental remdesivir) yet it’s new to China (unlike the malaria drug chloroquine phosphate). Perhaps more importantly, a domestic biotech — Zhejiang Hisun Pharma — owns the rights to manufacture and market the drug, preempting any concerns about patents.

FDA goes on high alert as coro­n­avirus rais­es threat to drug man­u­fac­tur­ing and clin­i­cal tri­als grind to a halt

The FDA isn’t quite sure just what the coronavirus outbreak in China will mean for the US pharma industry, but it has the potential to trigger a host of troublesome issues around the supply chain the country is directly plugged into.

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Warren Buffett, AP Images

War­ren Buf­fett gets a dou­ble take as the in­vest­ment pow­er­house set­tles on its first biotech in­vest­ment

Coke. American-Express. Apple. And Biogen?

Warren Buffet’s Berkshire Hathaway, which made itself into a symbol of rock-solid investment strategy, has revealed a stake in the big biotech as it takes on one of the biggest gambles in the industry.

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