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.

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.

Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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Jackie Fouse, Agios CEO

Agios scores its sec­ond pos­i­tive round of da­ta for its lead pipeline drug — but that won't an­swer the stub­born ques­tions that sur­round this pro­gram

Agios $AGIO bet the farm on its PKR activator drug mitapivat when it recently decided to sell off its pioneering cancer drug Tibsovo and go back to being a development-stage company — for what CEO Jackie Fouse hoped would be a short stretch before they got back into commercialization.

On Tuesday evening, the bellwether biotech flashed more positive topline data — this time from a small group of patients in a single-arm study. And the executive team plans to package this with its earlier positive results from a controlled study to make its case for a quick OK.

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Adeno-associated virus-1 illustration; the use of AAVs resurrected the gene therapy field, but companies are now testing the limits of a 20-year-old technology (File photo, Shutterstock)

Af­ter 3 deaths rock the field, gene ther­a­py re­searchers con­tem­plate AAV's fu­ture

Nicole Paulk was scrolling through her phone in bed early one morning in June when an email from a colleague jolted her awake. It was an article: Two patients in an Audentes gene therapy trial had died, grinding the study to a halt.

Paulk, who runs a gene therapy lab at the University of California, San Francisco, had planned to spend the day listening to talks at the American Association for Cancer Research annual meeting, which was taking place that week. Instead, she skipped the conference, canceled every work call on her calendar and began phoning colleagues across academia and industry, trying to figure out what happened and why. All the while, a single name hung in the back of her head.

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George Yancopoulos (L) and Len Schleifer (Regeneron)

Re­gen­eron touts pos­i­tive pre­lim­i­nary im­pact of its Covid an­ti­body cock­tail, pre­vent­ing symp­to­matic in­fec­tions in high-risk group

Regeneron flipped its cards on an interim analysis of the data being collected for its Covid-19 antibody cocktail used as a safeguard against exposure to the virus. And the results are distinctly positive.

The big biotech reported Tuesday morning that their casirivimab and imdevimab combo prevented any symptomatic infections from occurring in a group of 186 people exposed to the virus through a family connection, while the placebo arm saw 8 of 223 people experience symptomatic infection. Symptomatic combined with asymptomatic infections occurred in 23 people among the 223 placebo patients compared to 10 of the 186 subjects in the cocktail arm.

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Hal Barron, GSK via YouTube

What does $29B buy you in Big Phar­ma? In Glax­o­SmithK­line’s case, a whole lot of un­com­fort­able ques­tions about the pipeline

Talk about your bad timing.

A little over a week ago, GSK R&D chief Hal Barron marked his third anniversary at the research helm by taking a turn at the virtual podium during JP Morgan to make the case that he and his team had built a valuable late-stage pipeline capable of churning out more than 10 blockbusters in the next 5 years.

And then, just days later, one of the cancer drugs he bet big on as a top prospect — bintrafusp, partnered with Merck KGaA — failed its first pivotal test in non-small cell lung cancer.

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Vir's CMO says he's sur­prised that a low dose of their he­pati­tis B drug ap­pears promis­ing in ear­ly slice of da­ta — shares soar

Initial topline data from a Phase I study of a new therapeutic for chronic hepatitis B virus was so promising that it surprised even the CMO of the company that produces it.

Vir Biotechnology on Tuesday announced that its VIR-3434 molecule reduced the level of virus surface antigens present in a blinded patient cohort after eight days of the trial with just a single 6 mg dose. Six of the eight patients in the cohort were given the molecule, and the other two a placebo—all six who received the molecule saw a mean antigen reduction of 1.3 log10 IU/mL, Vir said.

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David Marek, Myovant

My­ovant beefs up da­ta pack­age in NDA #3, boost­ing its case for longterm dos­ing of Pfiz­er-part­nered re­l­u­golix

When Pfizer handed over $650 million in cash to partner on Myovant’s relugolix, the pharma giant made clear that the deal — valued at $4.2 billion total — was just as much about the approved indication of prostate cancer as the two women’s health conditions the drug could treat.

A month later, the two companies are offering another glimpse of the therapy’s longterm potential in endometriosis.

Look­ing to win over some skep­ti­cal an­a­lysts, Rhythm beats the drum on in­ter­im da­ta in PhII bas­ket study for ad­di­tion­al in­di­ca­tions

Rhythm Pharmaceuticals has been working toward expanding the FDA approval they received just two months ago for three rare genetic disorders that result in obesity. In December, their Phase III cut of data saw mixed reactions from analysts, but new interim results released Tuesday may provide more excitement.

In an ongoing Phase II study for setmelanotide across individuals with one of three distinct rare genetic diseases of obesity, 65 patients had reached the Dec. 17 cutoff date for evaluation. Among patients who met the primary endpoint of at least 5% weight loss over three months, Rhythm saw an average reduction of no less than 7.1% in any of the groups.