FDA slams J&J’s block­buster con­tender sirukum­ab on ‘im­por­tant safe­ty risks’

Just days af­ter Glax­o­SmithK­line dumped its part­ner­ship with J&J on the late-stage rheuma­toid arthri­tis drug sirukum­ab, the FDA helped il­lus­trate why the phar­ma gi­ant was ready to bail at this late date.

In an in­ter­nal re­view out two days ahead of a sched­uled pan­el re­view, reg­u­la­tors spot­light­ed a trou­bling round of safe­ty da­ta for the IL-6 drug, which has pro­vid­ed on­ly mixed ef­fi­ca­cy re­sults so far. Ac­cord­ing to the re­view, there was a clear im­bal­ance in deaths and ma­lig­nan­cies among the pa­tients stud­ied over a 52-week pe­ri­od.

From the re­view:

Wide con­fi­dence in­ter­vals around treat­ment com­par­isons for se­ri­ous rare events such as death, ma­lig­nan­cy, and MACE in­di­cate that the im­bal­ances could be due to chance but al­so that rel­a­tive­ly large in­creas­es in risks on sirukum­ab can­not be ruled out based on the da­ta alone. Such im­bal­ances raise con­cern re­gard­ing these im­por­tant safe­ty risks. Ad­di­tion­al safe­ty sig­nals re­lat­ed to events of GI per­fo­ra­tion and hy­per­sen­si­tiv­i­ty were al­so ob­served.

Omi­nous­ly, the re­view al­so high­lights al­ter­na­tive RA ther­a­pies, rais­ing ad­di­tion­al ques­tions why the agency would green-light a drug with po­ten­tial safe­ty is­sues for a huge mar­ket.

High­light­ing the agency’s stance on rheuma­toid arthri­tis drugs, Eli Lil­ly re­vealed last week that the FDA had re­ject­ed its big block­buster con­tender baric­i­tinib af­ter a hand­ful of blood clots were seen in the drug arms of the com­par­a­tive stud­ies done.

In the mean­time, Re­gen­eron and Sanofi have strut­ted ahead to win a de­layed OK on sar­ilum­ab while Ab­b­Vie works on its own suc­ces­sor to Hu­mi­ra, ABT-494. And Gilead is mov­ing con­fi­dent­ly ahead with a mas­sive Phase III pro­gram for fil­go­tinib.

J&J, though, isn’t about to give up at this point. In a state­ment, the com­pa­ny said:

Pa­tient well-be­ing is our high­est pri­or­i­ty at Janssen and we take the safe­ty of all pa­tients par­tic­i­pat­ing in our clin­i­cal pro­grams very se­ri­ous­ly.  We rec­og­nize the com­plex­i­ty and se­ri­ous­ness of a dis­ease like rheuma­toid arthri­tis (RA) and the tremen­dous needs that re­main for new treat­ment al­ter­na­tives as pa­tients liv­ing with RA of­ten cy­cle through mul­ti­ple ther­a­pies un­til find­ing one that may work for them.  We are con­fi­dent in the ben­e­fit-to-risk of sirukum­ab and look for­ward to meet­ing with the Arthri­tis Ad­vi­so­ry Com­mit­tee to re­view ef­fi­ca­cy and safe­ty da­ta sup­port­ing the pro­posed in­di­ca­tion for the treat­ment of mod­er­ate­ly to se­vere­ly ac­tive RA.

We’ll find out more at the pan­el re­view on Wednes­day.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 72,400+ biopharma pros reading Endpoints daily — and it's free.

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

UP­DAT­ED: 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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

UP­DAT­ED: 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.

Lars Fruergaard Jørgensen, chief executive officer of Novo Nordisk A/S, (via Getty Images)

The list of the 11 block­busters-to-be in line for a 2020 launch high­light agony and ec­sta­sy of drug R&D

For all the talk about unmet medical need and patients first and so on, the key criteria investors watch for any new drug in the pipelines is peak sales projection. Are you going to hit the blockbuster mark, at $1 billion-plus, or are you going to be an also-ran in the sales department?

Of course, analysts’ peak sales projections by themselves are of limited value in many cases. When the PCSK9 drugs started arriving 5 years ago, Repatha was billed as a $2.5 billion peak earner. They’re nowhere near that, with new competition threatening current levels. And if Biogen’s controversial Alzheimer’s drug aducanumab (submission planned but not on the list) is approved, per chance, will payers cover it?

Maybe not. And then those $10 billion in peak sales assumptions would go straight down the drain.

But, analysts are analysts, and peak sales projections have to be factored in when assessing the top experimental drugs up for a launch in the year ahead.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 72,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 72,400+ biopharma pros reading Endpoints daily — and it's free.