Fac­ing a pay­er back­lash over stick­er shock, Bio­gen fields pos­i­tive da­ta on Spin­raza for chil­dren 2-12

Fight­ing an up­hill bat­tle to get pay­ers to cov­er its $750,000 first-year price tag on Spin­raza (nusin­ersen), Bio­gen is post­ing an­oth­er round of stel­lar da­ta to back its use among old­er chil­dren with rare cas­es of spinal mus­cu­lar at­ro­phy.

Re­searchers test­ed the drug in 126 SMA pa­tients who were be­tween the ages of 2 and 12 and gen­er­al­ly suf­fered from Type 2 or Type 3 forms of the neu­ro­mus­cu­lar dis­ease. As we’ve al­ready seen with in­fants un­der 6 months suf­fer­ing ear­ly-on­set dis­ease, there was a clear re­sponse, with a 3.9 point im­prove­ment at month 15 in the Ham­mer­smith Func­tion­al Mo­tor Scale Ex­pand­ed (HFMSE) score, com­pared to a 1.0 point drop in pa­tients in the place­bo arm.

Al San­drock, Bio­gen

“In CHER­ISH, most chil­dren with lat­er-on­set SMA treat­ed with Spin­raza saw im­prove­ments in mo­tor func­tion and sta­bi­liza­tion or slow­ing of dis­ease pro­gres­sion,” said Dr. Richard Finkel, chief of neu­rol­o­gy, Nemours Chil­dren’s Hos­pi­tal, Or­lan­do, Flori­da. “As a physi­cian who has spent 37 years treat­ing chil­dren with SMA, it’s in­cred­i­bly en­cour­ag­ing to see some pa­tients on Spin­raza achieve mile­stones such as crawl­ing and stand­ing with as­sis­tance with­in the clin­i­cal tri­al. These kinds of clin­i­cal­ly mean­ing­ful im­prove­ments are un­prece­dent­ed and give new hope to in­di­vid­u­als with SMA and their fam­i­lies.”

But it’s pay­ers that Bio­gen most wants to im­press now.

“We’re hope­ful that when [health plans] see this da­ta, they will up­date their poli­cies” to cov­er the drug for old­er chil­dren, Bio­gen Chief Med­ical Of­fi­cer Al San­drock told the home­town news­pa­per Boston Globe.

Bio­gen, part­nered with Io­n­is, stunned some an­a­lysts with its price, which drops to half that $750,000 in the sec­ond year, or $1.5 mil­lion for the first three years of treat­ment. Leerink’s Ge­of­frey Porges not­ed re­cent­ly that the price could turn Spin­raza in­to the “So­val­di of rare dis­ease drugs,” com­par­ing it to the Gilead hep C cure that sparked an out­cry over the $1,000-per-pill cost.

Bio­gen bad­ly needs to make a suc­cess of Spin­raza. Its flag­ship drug Tec­fidera has been flag­ging as new com­peti­tors hit the MS mar­ket, and it has a thin pipeline of CNS drugs that most an­a­lysts would like to see fat­tened through new deals. Along those lines Bio­gen lined up a $300 mil­lion up­front deal a few days ago to grab an an­ti-tau drug from Bris­tol-My­ers Squibb. And more deals may be on the way.

“We’re mov­ing, and we’re back in terms of aug­ment­ing the pipeline,” re­search and de­vel­op­ment head Michael Ehlers told Bloomberg on Fri­day.

Pres­i­dent Trump, mean­while, has been cas­ti­gat­ing the in­dus­try for as­tro­nom­i­cal drug prices, though rare dis­ease drugs have gen­er­al­ly avoid­ed the spot­light among all the price con­tro­ver­sies that have erupt­ed in the last cou­ple of years.

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

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.

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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.

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.

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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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