In wake of a CAR-T re­struc­tur­ing, No­var­tis says it’s ready to hunt a pi­o­neer­ing OK for lead pro­gram

Bruno St­rig­i­ni, CEO No­var­tis On­col­o­gy

No­var­tis is still in the race to win the world’s first ap­proval for a pi­o­neer­ing CAR-T treat­ment.

The phar­ma gi­ant $NVS pulled back the veil from their Phase II study of their CAR-T CTL019, reg­is­ter­ing an im­pres­sive 82% com­plete re­mis­sion rate among the 50 chil­dren and young adults with B-cell acute lym­phoblas­tic leukemia. And with no deaths, though sig­nif­i­cant ev­i­dence of tox­i­c­i­ty, No­var­tis says it will be ready to file for an ap­proval ear­ly on in a loom­ing 2017.

Forty-one of 50 pa­tients achieved com­plete re­mis­sion or com­plete re­mis­sion with in­com­plete blood count re­cov­ery three months af­ter an in­fu­sion of CTL019, which reengi­neers pa­tients’ T cells in­to a can­cer ther­a­py.

Close to half of the pa­tients ex­pe­ri­enced a grade 3 or 4 case of cy­tokine re­lease syn­drome, not un­com­mon in the field. And 15% per­cent of pa­tients ex­pe­ri­enced grade 3 neu­ro­log­i­cal and psy­chi­atric events in­clud­ing en­cephalopa­thy and delir­i­um. But there were no grade 4 events and no one died.

That’s a sol­id enough ben­e­fit/safe­ty pro­file to take to reg­u­la­tors as No­var­tis hunts up an ac­cel­er­at­ed ap­proval.

No­var­tis man­aged to stun prac­ti­cal­ly every­one in the field when it abrupt­ly dis­band­ed its cell and gene ther­a­py unit three months ago, lay­ing off 120 and in­te­grat­ing an­oth­er 280 back in­to its reg­u­lar R&D ranks as CAR-T chief Us­man ‘Oz’ Azam hit the ex­it. That was wide­ly re­gard­ed as a se­ri­ous set­back that could af­fect fu­ture pro­grams, even if more ad­vanced ef­forts like this one at the Uni­ver­si­ty of Penn­syl­va­nia stayed on track.

Juno has been thrown in­to ut­ter dis­ar­ray with mul­ti­ple deaths de­rail­ing their lead ef­fort for JCAR015. And Kite re­cent­ly not­ed that it would need a lit­tle more time to com­plete a rolling sub­mis­sion for their top ther­a­py, which should now be com­plet­ed in Q1.

“This first-of-its-kind tri­al rep­re­sents ex­cit­ing progress to­ward our goal of help­ing chil­dren and young adults with re­lapsed or re­frac­to­ry B-cell ALL, a pa­tient pop­u­la­tion with an ur­gent need for new treat­ment op­tions,” said Bruno St­rig­i­ni, CEO, No­var­tis On­col­o­gy. “We are com­mit­ted to ad­vanc­ing CTL019 and look for­ward to work­ing close­ly with the FDA and EMA in the com­ing months.”

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

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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