FDA ush­ers in a new era in can­cer treat­ment with 'his­toric' CAR-T ap­proval for No­var­tis

The FDA has ap­proved the world’s first CAR-T ther­a­py, giv­ing a green light to No­var­tis for Kym­ri­ah (ti­s­agen­le­cleu­cel) in what reg­u­la­tors them­selves de­scribe as an his­toric event.

The ear­ly ap­proval — about a month ahead of the PDU­FA date — came through for cer­tain pe­di­atric and young adult pa­tients with a form of acute lym­phoblas­tic leukemia.

No­var­tis set the price for the one-time treat­ment at $475,000, right in line with low-end es­ti­mates and one that will put pres­sure on Gilead to rein in its own price for a ri­val ther­a­py ex­pect­ed to be ap­proved in the near fu­ture.

Scott Got­tlieb, FDA Com­mis­sion­er

None oth­er than FDA com­mis­sion­er Scott Got­tlieb did the hon­ors in the agency’s state­ment, not­ing:

We’re en­ter­ing a new fron­tier in med­ical in­no­va­tion with the abil­i­ty to re­pro­gram a pa­tient’s own cells to at­tack a dead­ly can­cer. New tech­nolo­gies such as gene and cell ther­a­pies hold out the po­ten­tial to trans­form med­i­cine and cre­ate an in­flec­tion point in our abil­i­ty to treat and even cure many in­tractable ill­ness­es. At the FDA, we’re com­mit­ted to help­ing ex­pe­dite the de­vel­op­ment and re­view of ground­break­ing treat­ments that have the po­ten­tial to be life-sav­ing.

A group of bio­phar­ma com­pa­nies have been rac­ing to this break­through point. Kite Phar­ma $KITE, new­ly bought out in a $12 bil­lion deal by Gilead $GILD, is com­ing in a close sec­ond with its own ap­pli­ca­tion for axi­cel.

No­var­tis’ in­ves­ti­ga­tors reg­is­tered a game-chang­ing 83% re­mis­sion rate in its piv­otal study.

CAR-T, though, is al­so as­so­ci­at­ed with se­vere and po­ten­tial­ly dead­ly side ef­fects, in­clud­ing lethal in­stances of cy­tokine re­lease syn­drome with some pa­tients dy­ing from brain swelling in sep­a­rate stud­ies from the No­var­tis drug.

Reg­u­la­tors not­ed that they will re­quire spe­cial train­ing for any­one in­volved in de­liv­er­ing this ther­a­py, while ex­pand­ing the ap­proval of Actem­ra (tocilizum­ab) to treat CAR T-cell-in­duced se­vere or life-threat­en­ing CRS in pa­tients 2 years of age or old­er. “In clin­i­cal tri­als in pa­tients treat­ed with CAR-T cells,” the FDA re­port­ed, “69% of pa­tients had com­plete res­o­lu­tion of CRS with­in two weeks fol­low­ing one or two dos­es of Actem­ra.”

This ther­a­py, which is made us­ing a pa­tient’s own im­mune cells, won’t be cheap.

By its own reck­on­ing, the UK’s tough watch dog on drug pric­ing has said that these drugs would be worth up to $649,000 a year, giv­en the young pa­tients it’s tar­get­ing first. An­a­lysts, mean­while, have pegged the price at any­where from $400,000 to $750,000.

In a call with an­a­lysts, No­var­tis ex­ecs said it would keep the price well be­low the high end, set­ting the cost at $475,000 for a drug that will be avail­able in 20 cen­ters with­in one month, and 35 soon af­ter.

In a state­ment the com­pa­ny said it “is col­lab­o­rat­ing with CMS to make an out­comes-based ap­proach avail­able to al­low for pay­ment on­ly when pe­di­atric and young adult ALL pa­tients re­spond to Kym­ri­ah by the end of the first month. Fu­ture po­ten­tial in­di­ca­tions would be re­viewed for the most rel­e­vant out­comes-based ap­proach.”

Joe Jimenez

Said Joseph Jimenez, CEO of No­var­tis:

Five years ago, we be­gan col­lab­o­rat­ing with the Uni­ver­si­ty of Penn­syl­va­nia and in­vest­ed in fur­ther de­vel­op­ing and bring­ing what we be­lieved would be a par­a­digm-chang­ing im­muno­cel­lu­lar ther­a­py to can­cer pa­tients in dire need. With the ap­proval of Kym­ri­ah, we are once again de­liv­er­ing on our com­mit­ment to change the course of can­cer care.

This ap­proval marks a ma­jor shift in on­col­o­gy, with a tru­ly rev­o­lu­tion­ary ap­proach to treat­ing can­cer. That hasn’t es­caped the no­tice of big play­ers and small, with a host of de­vel­op­ers look­ing to do much bet­ter, more safe­ly, with new drugs in the pipeline.

It’s a big day in the biotech world, but it’s just the of­fi­cial start date for a new era.

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Pfiz­er's big block­buster Xel­janz flunks its post-mar­ket­ing safe­ty study, re­new­ing harsh ques­tions for JAK class

When the FDA approved Pfizer’s JAK inhibitor Xeljanz for rheumatoid arthritis in 2012, they slapped on a black box warning for a laundry list of adverse events and required the New York drugmaker to run a long-term safety study.

That study has since become a consistent headache for Pfizer and their blockbuster molecule. Last year, Pfizer dropped the entire high dose cohort after an independent monitoring board found more patients died in that group than in the low dose arm or a control arm of patients who received one of two TNF inhibitors, Enbrel or Humira.

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Steve Harr (L) and Hans Bishop

One of the most am­bi­tious start­up teams in biotech just out­lined plans for a $400M IPO and a val­u­a­tion of about $4B

The executive team at Sana Biotechnology has sketched out more details about the full scope of its ambitions as the new unicorn to watch. They amended their S-1 today to include a price range of $20 to $23 a share — which puts them in reach of pulling in around $400 million on the high end with a market value starting right around $4 billion.

That’s not bad for a preclinical biotech with no drugs yet in human studies, but it squares with its ambitions to remake the cell therapy field with a slate of in-house platforms. The biotech raised $705 million — primarily from ARCH (44 million shares) and Flagship (34.2 million shares) — to get to this stage.

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Top gene ther­a­py deals, M&A pacts in 2020 high­light an­oth­er big year in one of the hottest fields in bio­phar­ma

Chris Dokomajilar at DealForma has been crunching the numbers on gene therapy deals over the last 2 years and came away with a few key observations.

Both the upfront cash and deal totals last year backed off a bit from the record high hit in 2019, but the totals are still running well ahead of anything we’ve seen in the years prior to 2019/2020.
2020 R&D partnerships came in at 23 deals, with $1.1 billion in disclosed upfront cash and equity and more than $8.5 billion in total deal value. Looking at 2019-2020 M&A, Dokomajilar found: 9 Acquisitions, with over $11.1 billion in disclosed upfront cash and equity and more than $13.4 billion in total M&A value.

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Lil­ly at­tempts to re­vive P2X7 re­cep­tor an­tag­o­nists for pain, li­cens­ing PhI pro­gram from Japan’s Asahi Ka­sei Phar­ma

Eli Lilly is fronting some new cash in a space they’re quite familiar with.

The company is partnering with Japan’s Asahi Kasei Pharma on an experimental drug for chronic pain, acquiring the rights for the P2X7 receptor antagonist program dubbed AK1780. Lilly will shell out a pretty penny for the program, promising up to $410 million total should each milestone payment come to pass.

Asahi Kasei will receive an upfront sum of $20 million for the candidate. In addition, Lilly is on the hook for up to $210 million in development and regulatory milestones and another potential $180 million in sales milestones. Asahi Kasei can also obtain royalties ranging from the mid-single to low-double digits should an approved product come out of the deal.

Ther­mo Fish­er plat­form seeks to ex­pe­dite donor cell cul­ti­va­tion for al­lo­gene­ic cell ther­a­pies

One of the world’s leading CDMOs has launched a new technology it says will expedite a quickly-growing sect of biotech drug development: off-the-shelf, allogeneic cell therapies.

It’s been nearly a decade since the FDA approved the first use of the method that uses healthy donor cells to create a master cell bank, which is then used for specific therapies — a cord blood allogeneic treatment called Hemacord. In the years since, the use of allogeneic cells has taken off in research circles, most notably in the use of T cell therapies to target solid tumor cancers.

Bob Nelsen (Michael Kovac/Getty Images)

ARCH an­nounces largest fund yet, rais­ing $1.85B to back men­tal health, cell and gene edit­ing ap­proach­es

Nearly a year ago, as the pandemic encroached and the stock market cratered, Flagship and ARCH Venture announced three mega-funds worth a combined $2.6 billion. They wanted, ARCH’s Bob Nelsen said, to restore confidence “that there was money out there and a lot of it” to invest in biotech.

Since then, the stock market has returned — almost frighteningly so — and Nelsen has kept raising and spending cash. On Thursday, he announced a new fund, worth $1.85 billion. It’s the largest pot yet for a VC famous for its deep pockets.

Covid-19 roundup: EU and As­traZeneca trade blows over slow­downs; Un­usu­al unions pop up to test an­ti­bod­ies, vac­cines

After coming under fire for manufacturing delays last week, AstraZeneca’s feud with the European Union has spilled into the open.

The bloc accused the pharma giant on Wednesday of pulling out of a meeting to discuss cuts to its vaccine supplies, the AP reported. AstraZeneca denied the reports, saying it still planned on attending the discussion.

Early Wednesday, an EU Commission spokeswoman said that “the representative of AstraZeneca had announced this morning, had informed us this morning that their participation is not confirmed, is not happening.” But an AstraZeneca spokesperson later called the reports “not accurate.”

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Janet Woodcock (AP Images)

Ad­vo­ca­cy groups don't want Janet Wood­cock to head the FDA, blast­ing ‘reg­u­la­to­ry fail­ures’ in opi­oid cri­sis

It turns out the controversies around Janet Woodcock’s regulatory legacy weren’t limited to Sarepta’s eteplirsen.

A coalition of advocacy groups dedicated to the opioid crisis urged Norris Cochran and Xavier Becerra — the acting and designated HHS secretary, respectively — to keep her reign as interim FDA chief a “very short transition.” During her lengthy tenure as CDER, they add, Woodcock presided over “one of the worst regulatory agency failures in U.S. history.”

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