Re­gen­eron, Sanofi are ready to de­but the 6th PD-1/L1. Is it still pos­si­ble to stand out from the grow­ing crowd?

Re­gen­eron $REGN and Sanofi $SNY rolled out an up­date on their PD-1 check­point cemi­plimab this morn­ing, demon­strat­ing that the promis­ing ear­ly da­ta they had seen on a high re­sponse rate for a dead­ly skin can­cer re­mained in­tact for close to half of all the pa­tients en­rolled in the piv­otal Phase II tri­al.

In­ves­ti­ga­tors came up with a 46.3% ORR for ad­vanced cu­ta­neous squa­mous cell car­ci­no­ma in the study, which is be­ing tak­en to reg­u­la­tors in search of a quick OK for what will like­ly be the world’s sixth PD-1/L1 ther­a­py to hit the mar­ket. Ac­cord­ing to re­cent re­search from the Can­cer Re­search In­sti­tute, there are an­oth­er 49 PD-1/L1 check­points in clin­i­cal de­vel­op­ment, with hun­dreds more in pre­clin­i­cal re­search.

Is­rael Lowy, Re­gen­eron

This one, though, has been held up by the re­search team as po­ten­tial­ly su­pe­ri­or to much of the rest of the field. Top in­ves­ti­ga­tors in the pro­gram have high­light­ed their be­lief that PD-1 is a fun­da­men­tal­ly su­pe­ri­or ap­proach than PD-L1 — though they have yet to prove that. The two big part­ners are al­so once again re­ly­ing on Re­gen­eron’s fa­mous an­ti­body fac­to­ry to stand out from the crowd with a check­point that won the FDA’s break­through des­ig­na­tion.

There’s still no fi­nal take on the du­ra­tion of the re­sponse, with Sanofi say­ing that 32 of 38 re­spons­es among the 82 pa­tients en­rolled are con­tin­u­ing.

It’s no co­in­ci­dence that Sanofi arranged to de­clare the piv­otal da­ta to­day, right at the be­gin­ning of its an­nu­al R&D day. For the phar­ma gi­ant it’s a chance to demon­strate that it has the R&D fire­pow­er need­ed to come up with im­por­tant new ther­a­pies — even if much of the in­no­va­tion is still com­ing from its part­ners at Re­gen­eron.

Skep­tics, though, aren’t see­ing any­thing new with to­day’s up­dates. Still, Sanofi can use this PD-1 to cre­ate its own com­bos as it sets the stage for a re­newed R&D ef­fort on the on­col­o­gy front, af­ter some se­ri­ous set­backs forced an ear­li­er re­or­ga­ni­za­tion. But Baird’s Bri­an Sko­r­ney says that at this point no-one should think a launch will be easy, or quick­ly prof­itable. He notes;

Sales of Keytru­da and Op­di­vo are on track for $3.8B and $4.8B, re­spec­tive­ly, this year. Tecen­triq, the third to mar­ket two years lat­er, is on track for about $500M, while Baven­cio and Imfinzi, both ap­proved this year, have sold close to noth­ing. Com­pet­ing in the ma­jor PD-(L)1 in­di­ca­tions such as melanoma, NSCLC, etc. will there­fore like­ly be very dif­fi­cult. Though we don’t dis­agree that front-line NSCLC is a huge op­por­tu­ni­ty, the pletho­ra of dis­ap­point­ing da­ta in NSCLC with ap­proved agents presents an un­re­al­is­ti­cal­ly high hur­dle for man­age­ment to ex­pect to eas­i­ly cross.

Is­rael Lowy, Re­gen­eron’s lead sci­en­tist on the pro­gram, had this to say:

This is the largest prospec­tive study ever con­duct­ed in this dis­ease, and we are pleased that many peo­ple were able to achieve deep and durable re­spons­es with cemi­plimab monother­a­py. The high and durable re­sponse rates seen in this study are par­tic­u­lar­ly no­table giv­en that the study en­rolled pa­tients re­gard­less of bio­mark­er sta­tus.

The rolling sub­mis­sion will be wrapped in Q1.

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.

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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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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Stéphane Bancel, Getty

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.

Paul Hastings, Nkarta CEO

With no up­front pay­ment or mile­stones on the line, Nkar­ta and CRISPR join forces on CAR-NK search

Most deals in biotech come with hefty upfront payments attached, and the promise of big biobucks if a program works out. Not this one.

Nkarta has struck what CEO Paul Hastings calls a “real collaboration” with CRISPR Therapeutics to co-develop and commercialize two CAR-NK therapies, in addition to an NK+T program. The duo will split all R&D costs — and any worldwide profits — 50/50, Hastings said.

Brent Saunders (Richard Drew, AP Images)

OcuWho? Star deal­mak­er turned aes­thet­ics czar Brent Saun­ders flips back in­to biotech. But who’s he team­ing up with now?

Brent Saunders went on a tear of headline-blazing deals building Allergan, merging and rearranging a variety of big companies into one before an M&A pact with Pfizer blew up and sent him on a bout of biotech drug deals. That didn’t work so well, so under pressure, he got his buyout at AbbVie — which needed a big franchise like Botox. And it was no big surprise to see him riding the SPAC wave into a recent $1 billion-plus deal that left him in the executive chairman’s seat at an aesthetics outfit — now redubbed The Beauty Health Company — holding a big chunk of the equity.

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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.

'Chang­ing the whole game of drug dis­cov­ery': Leg­endary R&D vet Roger Perl­mut­ter leaps back in­to work as a biotech CEO

Roger Perlmutter needs no introduction to anyone remotely involved in biopharma. As the R&D chief first at Amgen and then Merck, he’s built a stellar reputation and a prolific career steering new drugs toward the market for everything from cancer to infectious diseases.

But for years, he’s also held a less known title: science partner at The Column Group, where he’s regularly consulted about the various ideas the VCs had for new startups.

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