Wel­come Imfinzi: As­traZeneca crash­es the check­point par­ty late with a green light for dur­val­um­ab

And then there were 5.

The FDA hand­ed out its lat­est ap­proval for a PD-L1 check­point in­hibitor on Mon­day af­ter­noon, giv­ing a green light to As­traZeneca to start sell­ing dur­val­um­ab as Imfinzi as a sec­ond-line ther­a­py for metasta­t­ic urothe­lial car­ci­no­ma.

Pas­cal So­ri­ot

The reg­u­la­to­ry OK comes in the wake of ap­provals for Mer­ck, Bris­tol-My­ers Squibb, Roche/Genen­tech and Pfiz­er/Mer­ck KGaA. The bi­ol­o­gy of these new check­point drugs is well un­der­stood now, and the FDA is­sued its ap­proval af­ter a rel­a­tive­ly small, sin­gle-arm study.

Reg­u­la­tors waved this one through af­ter giv­ing dur­val­um­ab a break­through drug des­ig­na­tion and pri­or­i­ty re­view, even though its the third ap­proval for a check­point ther­a­py in blad­der can­cer.

Dur­val­um­ab’s longterm suc­cess is cru­cial to the fu­ture of As­traZeneca and CEO Pas­cal So­ri­ot. Billed as a block­buster-to-be, fol­low­ing a com­mer­cial trail al­ready clear­ly laid out, the big show­down for As­traZeneca comes lat­er in the year, when it rolls out late-stage da­ta on a com­bi­na­tion of its check­point com­bo in its MYS­TIC study, which match­es dur­val­um­ab with treme­li­mum­ab, a CT­LA-4 sim­i­lar to Yer­voy, for lung can­cer.

The ju­ry is still out, though, on how well a CT­LA-4 drug — with all its at­ten­dant tox­i­c­i­ty — will do in this field. That’s one rea­son why Bris­tol-My­ers Squibb re­cent­ly inked a deal with Cy­tomX on a next-gen CT­LA-4 that might prove far bet­ter for pa­tients.

Sea­mus Fer­nan­dez at Leerink not­ed some mod­est ex­pec­ta­tions on this first ap­proval. The up­side lies fur­ther down the road.

Al­though this in­di­ca­tion rep­re­sents a rel­a­tive­ly small op­por­tu­ni­ty for AZN (we fore­cast Imfinzi cap­tur­ing 10% of our es­ti­mat­ed ~$2.3B WW blad­der can­cer mar­ket), the ap­proval will al­low the agent to be­come more fa­mil­iar with on­col­o­gists and should help fa­cil­i­tate fu­ture sBLAs for drug. The ma­jor in­di­ca­tion for Imfinzi re­mains first-line (1L) non-small cell lung can­cer (NSCLC) and we await top-line da­ta from the Phase 3 MYS­TIC tri­al in com­bi­na­tion with treme­li­mum­ab (an­ti-CT­LA-4) ex­pect­ed in mid-2017.

As­traZeneca, mean­while, raised a red flag last week when it de­layed its third-line read­out in the ARC­TIC study of the duo, spurring some sus­pi­cions that it was on track to a trou­bling fail­ure that would have raised se­ri­ous doubts about its fu­ture in the field.

The oth­er ques­tion that many of us have is how many of these PD-1/PD-L1 check­points can be ap­proved be­fore they start slic­ing and dic­ing this mar­ket in­to ever small­er bites. A range of sec­ond-wave check­points are in de­vel­op­ment now, with every­one that’s fi­nanced well enough and in­ter­est­ed in it go­ing af­ter one of their own.

That group in­cludes In­cyte, which has been part­ner­ing with the main­stream check­points in nonex­clu­sive arrange­ments. And the main play­ers, like Mer­ck and Bris­tol-My­ers, are al­ready well along with their own com­bi­na­tion tri­als. Hun­dreds of them.

The ap­proval, though, marks a big win for As­traZeneca, which has made sig­nif­i­cant progress on the on­col­o­gy front in the last few years. They had to have this one to re­main a cred­i­ble ri­val. And they got it.

The FDA has been on a drug ap­proval spree over the last few days. This is the fifth OK for a new chem­i­cal en­ti­ty in the last three work­ing days, bring­ing the year-to-date to­tal to 19. Last year, which saw a big dip in ap­provals, the FDA ap­proved a to­tal of 22 new drugs.

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

Ron DePinho (file photo)

A 'fly­over' biotech launch­es in Texas with four Ron De­Pin­ho-found­ed com­pa­nies un­der its belt

In his 13 years at Genzyme, Michael Wyzga noticed something about East Coast drugmakers. Execs would often jet from Boston or New York to San Francisco to find more assets, and completely miss the work being done in flyover states, like Texas or Wisconsin.

“If it doesn’t come out of MGH or MIT or Harvard, probably not that interesting,” he said of the mindset.

Now, he and some well-known industry players are looking to change that, and they’ve reeled in just over $38 million to do it.

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Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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Ad­comm splits slight­ly in fa­vor of FDA ap­prov­ing Chemo­Cen­tryx’s rare dis­ease drug

The FDA’s Arthritis Advisory Committee on Thursday voted 10 for and 8 against the approval of ChemoCentryx’s $CCXI investigational drug avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

The vote on whether the FDA should approve the drug was preceded by a split vote of 9 to 9 on whether the efficacy data support approval, and 10 to 8 that the safety profile of avacopan is adequate enough to support approval.

Drug pric­ing watch­dog joins the cho­rus of crit­ics on Bio­gen's ad­u­canum­ab: What about charg­ing $2,560 per year?

As if Biogen’s aducanumab isn’t controversial enough, the researchers at drug pricing watchdog ICER have drawn up the contours of a new debate: If the therapy does get approved for Alzheimer’s by June, what price should it command?

Their answer: At most $8,290 per year — and perhaps as little as $2,560.

Even at the top of the range, the proposed price is a fraction of the $50,000 that Wall Street has reportedly come to expect (although RBC analyst Brian Abrahams puts the consensus figure at $11.5K). With critics, including experts on the FDA’s advisory committee, making their fierce opposition to aducanumab’s approval loud and clear, the pricing pressure adds one extra wrinkle Biogen CEO Michel Vounatsos doesn’t need as he orders full-steam preparation for a launch.

Biden ad­min­is­tra­tion backs a po­lar­iz­ing pro­pos­al to waive IP for all Covid-19 vac­cines

In a surprise U-turn, the Biden administration said Wednesday that it will support a proposal at the World Trade Organization to temporarily waive intellectual property protections on Covid-19 vaccines.

The proposal, backed by South Africa and India at the WTO, seeks to help developing countries with limited vaccine supplies. The US and Europe historically opposed the proposal, saying IP should be protected because it incentivizes new drug and vaccine development.

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FDA ex­tends re­search agree­ment with MIT-li­censed or­gan-on-chip sys­tems

The FDA on Wednesday extended its four-year agreement with CN Bio, a developer of single- and multi-organ-on-chip systems used for drug discovery, for another three years.

CN Bio said the scope of the research performed by the FDA’s Center for Drug Evaluation and Research has expanded to include the exploration of the company’s lung-on-a-chip system to help with the agency’s evaluation of inhaled drugs, in addition to the agency’s work on its liver model.

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In quest to meet user fee goals, FDA’s per­for­mance con­tin­ues down­ward trend

A recent update to the FDA’s running tally of how it’s meeting its user fee-related performance goals during the pandemic shows an agency that is not out of the woods yet.

The latest numbers reveal that for a second straight quarter in 2021, the FDA has met its user fee goal dates for 93% of original new drug applications, which compares with 94% and 98% for the previous two quarters in 2020, respectively.

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