Israel Lowy (Regeneron)

#ES­MO20: 'As good as any PD-1 out there': Re­gen­eron flash­es PD-(L)1 lung can­cer da­ta to ri­val Mer­ck

Re­gen­eron en­tered the PD-(L)1 game late, so they de­vised a two-pronged strat­e­gy to catch up with Big Phar­ma ri­vals: They would push it in­to can­cers where PD-1s had yet been test­ed, and they would prove that it’s as pow­er­ful in the big in­di­ca­tions as any oth­er on the mar­ket.

They cleared a hur­dle on the first goal Fri­day, show­ing a 31% re­sponse in pa­tients with the rare skin can­cer basal cell car­ci­no­ma. And with the da­ta they’re rolling out Mon­day, Re­gen­eron can­cer chief Is­rael Lowy is ready to de­clare suc­cess on the sec­ond.

“When we set out to de­vel­op a PD-1, we want­ed to be sure no one had any doubt that cemi­plimab was as good as any PD-1 out there,” Lowy told End­points News, us­ing the chem­i­cal name for Lib­tayo. “I think that’s what we can say with con­fi­dence to­day.”

On Mon­day at ES­MO, Re­gen­eron rolled out the full Phase III da­ta test­ing Lib­tayo as a monother­a­py in non-small cell lung can­cer. It’s the same set­ting where Mer­ck’s Keytru­da had one of its biggest suc­cess­es, and where Bris­tol My­ers Squibb’s Op­di­vo had one of its worst stum­bles.

Across 710 pa­tients, those who re­ceived Lib­tayo lived for a me­di­an of 22 months com­pared with 14 months on chemo. No­tably, that trans­lat­ed to a 32% re­duc­tion in risk of death in all pa­tients who ex­pressed PD-L1. And for pa­tients con­firmed to ex­press PD-L1 above 50%, there was a 43% re­duc­tion in risk of death.

Cross-tri­al com­par­isons are no­to­ri­ous­ly rough, but these num­bers are in line or high­er than what Mer­ck re­port­ed for Keynote 042, its long-term study on Keytru­da as a monother­a­py in NSCLC. In that study, the re­duc­tion in risk of death was 32% for those who had more than 50% PD-L1 ex­pres­sion and 19% for those with PD-L1 ex­pres­sion above 1%. Lowy al­so ar­gued the tri­al had a clos­er to re­al-world pa­tient pop­u­la­tion, with loos­er en­roll­ment cri­te­ria.

It’s not yet clear how much mar­ket share these num­bers will net for Re­gen­eron. Keytru­da has a firm group on the mar­ket in NSCLC and many pa­tients don’t re­ceive a check­point in­hibitor alone but rather a com­bi­na­tion of check­point and chemother­a­py. Lowy not­ed that some physi­cians will opt for monother­a­py, par­tic­u­lar­ly in pa­tients with high ex­pres­sion who don’t want to un­der­go the rig­ors of chemo, but the com­pa­ny al­so has a tri­al test­ing chemo and Lib­tayo to­geth­er vs. chemo alone that’s due to read out ear­ly next year.

More broad­ly, the new da­ta es­tab­lish Lib­tayo as a pow­er­ful PD-1 as Re­gen­eron looks to make up for lost time. And they boost the big biotech’s broad­er strat­e­gy to break new ef­fi­ca­cy ground with new com­bi­na­tions. Com­bos are the rage right now in check­point ther­a­py, but Re­gen­eron has out­lined a rel­a­tive­ly unique ap­proach to the mix and match game.

For one, there’s the bis­pecifics the com­pa­ny has spent years de­vel­op­ing and which they hope, in some cas­es, will work even bet­ter when mixed with Lib­tayo. They showed off one such com­bi­na­tion in mice in a  Sci­ence Trans­la­tion­al Med­i­cine cov­er sto­ry. Lowy al­so named com­bi­na­tions with on­colyt­ic virus­es and RNA and DNA can­cer vac­cines. Re­cent pacts with Vyr­i­ad and BioN­Tech will help push in that di­rec­tion.

A year from now, Lowy said, he wants to come in­to ES­MO show­ing off their proof-of-con­cept da­ta for the bis­pecifics and new da­ta on Lib­tayo in com­bi­na­tion.

They’ll look to “be­lie this no­tion that an­ti­bod­ies are com­modi­ties,” he said.”Any­body can make an an­ti­body, but mak­ing a damn good an­ti­body is not triv­ial.”

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

Philipp Spycher

Promis­ing bet­ter link­er tech to ADC field, Araris has 'very, very am­bi­tious' plans for the clin­ic

A couple months after raising CHF 2.5 million ($2.76 million) in initial seed funding, one-year-old Araris Biotech is topping off the round with another CHF 12.7 million ($14 million).

The Paul Scherrer Institute and ETH Zurich spinout now has CHF 15.2 million to work with, and CEO Philipp Spycher has big plans. He hopes to bring one of the company’s antibody-drug conjugates (ADC) to the clinic by late 2022 or early 2023. “It’s very, very ambitious, but we are very optimistic that we actually can make it,” he said.

Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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UP­DAT­ED: CRISPR Ther­a­peu­tics gets a snap­shot of off-the-shelf CAR-T suc­cess in B-cell ma­lig­nan­cies — marred by the death of a pa­tient

Just days after scientific founder Emmanuelle Charpentier shared the Nobel prize for her work on CRISPR/Cas9, CRISPR Therapeutics $CRSP is showing off a snapshot of success in their early-stage study for an off-the-shelf CAR-T approach to CD19+ B cell malignancies — a snapshot marred by the death of a patient who had been given a high dose of the treatment.

Using their gene editing tech, researchers for CRISPR engineered cells from healthy donors into an attack vehicle aimed at cancer, something that has been achieved with great success using patients’ own cells — the autologous approach. But autologous CAR-T is hampered by the more complex vein-to-vein requirement that delays treatment, and now CRISPR Therapeutics along with other players like Allogene are determined to replace the pioneers with CAR-T 2.0.

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RBC's Bri­an Abra­hams holds a mock ad­comm on Bio­gen's iffy ad­u­canum­ab da­ta — and most of these ex­perts don't see a path to an ap­proval

As catalysts go, few loom larger than the aducanumab adcomm slated for Nov. 6.

With its big franchise under assault, Biogen is betting the ranch that its mixed late-stage Alzheimer’s data can squeak past the experts and regulators and get onto the market. And the topic — after a decade of Alzheimer’s R&D disasters in what still represents the El Dorado of drug markets — remains in the center ring of discussions around late-stage pipeline prospects.

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David Hung (file photo)

Mas­ter deal­mak­er David Hung re­tools a SPAC sedan in­to a fi­nanc­ing mus­cle ve­hi­cle that leaves his can­cer start­up with $850M and a place on Wall Street

It’s only right that one of the industry’s top dealmakers just completed one of the biggest SPAC-related deals in the pipeline.

David Hung, of Medivation fame, has completed a back flip into the market, merging with EcoR1 Capital’s SPAC Panacea and landing neatly on Wall Street with an $NUVB stock ticker after filling out the blank check in his name. In addition to the $144 million held in the SPAC — provided none of the investors opt out — Hung is getting ahold of $500 million more being chipped in by a slate of institutional investors who feel that Hung could have the keys to another Medivation-style success.

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Years af­ter a ma­jor tri­al set­back, No­var­tis switch­es gears with SMA drug. This time they're try­ing it for Hunt­ing­ton's

Four years after a Phase I/II setback in spinal muscular atrophy (SMA), Novartis is hoping its drug branaplam will find more success in a new neurological indication: Huntington’s disease.

The decision was announced a year after the head of research, Jay Bradner, said he did not see a “big opportunity” in SMA, according to Reuters. Novartis says it has preclinical data showing that branaplam reduces levels of mutant huntingtin protein, and SMA data showing patients on the drug had reductions in huntingtin mRNA. The FDA gave branaplam their orphan drug designation, and Novartis plans to move forth with a Phase IIb trial next year.

Glax­o­SmithK­line's vac­cines group aims for a first as it kicks off PhI­II RSV stud­ies

One of GlaxoSmithKline’s big projects at its global vaccine R&D center in Rockville, MD is set to enter Phase III after passing early-stage tests with flying colors.

Eyeing the wide-open respiratory syncytial virus (RSV) space, GSK is pushing two different vaccine candidates: GSK3888550A is designed to confer protection to infants via maternal immunization, while GSK3844766A is meant for the elderly.