Am­gen/UCB hit an­oth­er Eveni­ty hur­dle, as Eu­ro­pean reg­u­la­tors fail to back the os­teo­poro­sis drug

Am­gen $AMGN and its Bel­gian part­ners at UCB may have won over the FDA, but the EMA is still not con­vinced by their bone-build­ing os­teo­poro­sis drug ro­mosozum­ab.

On Fri­day, the com­pa­nies dis­closed that the Eu­ro­pean reg­u­la­tor had adopt­ed a neg­a­tive opin­ion on the drug, brand­ed Eveni­ty, for the treat­ment of os­teo­poro­sis in men and in post­menopausal women at in­creased risk of frac­ture. The part­ners plan to ap­peal the de­ci­sion.

Eveni­ty was test­ed in three late-stage stud­ies: the 7,180-pa­tient place­bo-con­trolled FRAME study in post­menopausal women with os­teo­poro­sis; the 4,093-pa­tient ARCH study in post­menopausal women in os­teo­poro­sis, which test­ed the drug against Mer­ck’s Fos­amax; and the 245-pa­tient place­bo-con­trolled BRIDGE study in men with os­teo­poro­sis.

All three piv­otal stud­ies showed the mon­o­clon­al an­ti­body was ef­fec­tive, but se­ri­ous CV ad­verse events were ob­served in the ARCH tri­al, which prompt­ed the FDA to orig­i­nal­ly is­sue a com­plete re­sponse let­ter to de­ny ap­proval in post­menopausal women with os­teo­poro­sis. The man­u­fac­tur­ers then re-sub­mit­ted an ap­pli­ca­tion to mar­ket the drug last year, but for a nar­row­er pa­tient pop­u­la­tion: post­menopausal women with os­teo­poro­sis who car­ry a high risk of frac­ture, or pa­tients who have failed or are in­tol­er­ant to ex­ist­ing os­teo­poro­sis ther­a­pies.

In April, the US health reg­u­la­tor ap­proved Eveni­ty with a black box warn­ing, high­light­ing the po­ten­tial­ly height­ened risk of heart at­tack, stroke and car­dio­vas­cu­lar death. The once-month­ly drug car­ries an an­nu­al list price of $21,900 in the Unit­ed States. The in­jectable treat­ment has al­so been cleared for use in Japan, South Ko­rea and Cana­da.

“Fol­low­ing reg­u­la­to­ry chal­lenges in the US (UCB has pre­vi­ous­ly dis­cussed this pos­si­bil­i­ty with in­vestors), to­day’s news was not com­plete­ly un­ex­pect­ed – the part­ners view EU as the least valu­able re­gion in terms of Eveni­ty, and we es­ti­mate that the EU op­por­tu­ni­ty is worth $1-2 per share,” Cred­it Su­isse an­a­lyst Evan Seiger­man wrote in a note.

Eveni­ty could still win Eu­ro­pean ap­proval with its ap­peal, but Eu­ro­pean sales are ex­pect­ed to be mod­est, he added.

The drug func­tions pre­dom­i­nant­ly as a bone an­a­bol­ic agent that stim­u­lates bone growth by in­hibit­ing a pro­tein called scle­rostin, which ceas­es the pro­duc­tion of bone and en­hances its break­down. Pro­lia, Am­gen’s $AMGN old­er and Eu­rope-ap­proved os­teo­poro­sis treat­ment, is in­tend­ed for chron­ic treat­ment. It works by in­creas­ing bone mass for as long as the pa­tient re­ceives it. Eveni­ty is in­tend­ed for short term use —  12 months — to rapid­ly in­crease bone min­er­al den­si­ty and re­duce frac­ture risk in pa­tients with im­mi­nent risk of frac­ture, it is then fol­lowed by an an­tire­sorp­tive agent such as Pro­lia.

So­cial im­age: Kristof­fer Trip­plaar, AP Im­ages

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.

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

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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For­mer No­var­tis ex­ec pleads guilty to gener­ic price fix­ing

On Friday, Hector Armando Kellum — a former senior executive at Novartis’ Sandoz unit plead guilty to charges of conspiring to fix the prices of products, including eczema and psoriasis treatment clobetasol and anti-fungal therapy nystatin triamcinolone, the Justice Department said.

His alleged partners-in-crime included Ara Aprahamian, a former sales and marketing executive at Taro Pharmaceutical Industries, who was also indicted in Philadelphia earlier this month for price-fixing and bid-rigging.