Look­ing for an on­col­o­gy re­boot, Sanofi, Re­gen­eron shoot for a fast and flashy FDA OK of a promis­ing new PD-1

There’s an­oth­er PD-1 drug head­ed to­ward the mar­ket. And this fast-mov­ing — and of­ten over­looked — pro­gram is due to ar­rive at the FDA for mar­ket­ing re­view in just a mat­ter of months.

In an out­line of Sanofi’s R&D work for the Q2 call this week, re­search chief Elias Zer­houni was clear­ly ex­cit­ed by the “fast-to-mar­ket strat­e­gy in the PD-1 space” that has been set up with its big de­vel­op­ment part­ner Re­gen­eron $REGN.

Here’s what Zer­houni had to say:

At AS­CO, we pre­sent­ed pos­i­tive clin­i­cal da­ta from a Phase I study, which showed that in pa­tients with ad­vanced CSCC (cu­ta­neous squa­mous cell car­ci­no­ma — the sec­ond dead­liest form of skin can­cer be­hind melanoma), our PD-1 was as­so­ci­at­ed with a 46% over­all re­sponse rate, a 69% dis­ease con­trol rate, and was gen­er­al­ly well tol­er­at­ed. So, we look for­ward to the re­sults of our reg­is­tra­tion study and are plan­ning to sub­mit to the FDA in metasta­t­ic cu­ta­neous squa­mous cell car­ci­no­ma in Q1 2018.

We al­so start­ed sev­er­al stud­ies of our PD-1 in oth­er in­di­ca­tions this past quar­ter, and these in­clude a Phase 3 in first line non-small cell lung can­cer and a Phase 2 in metasta­t­ic and lo­cal­ly ad­vanced basal cell car­ci­no­ma.

Is­rael Lowy

With 5 PD-(L)1 drugs on the mar­ket and hun­dreds of stud­ies un­der­way to ad­vance new ap­provals, the con­ver­sa­tion in can­cer cir­cles these days tends to fo­cus on whether these mul­ti­ply­ing check­point in­hibitors are be­com­ing swift­ly com­modi­tized.

That’s not the way Sanofi and Re­gen­eron are ap­proach­ing this. At all.

As Re­gen­eron’s top re­searcher on the pro­gram Is­rael Lowy told me at AS­CO last sum­mer, REGN2810 has con­vinced him PD-1 is a sig­nif­i­cant­ly bet­ter way to tack­le the check­point hur­dle than PD-L1. And the part­ners be­lieve they can quick­ly start by leapfrog­ging the PD-L1s from As­traZeneca, Roche and Pfiz­er/Mer­ck KGaA.

If he’s right, the im­pli­ca­tions in this megablock­buster field are enor­mous.

Elias Zer­houni

Zer­houni — who was diplo­mat­ic enough to thrive as head of the NIH — wasn’t will­ing to go that far right now. But for some­one who’s look­ing to this drug to help re­boot what has been a dis­ap­point­ing for­ay in­to on­col­o­gy for years, he may be work­ing up to it. Pressed on the point, he added:

Now, we, as I told you, be­lieve that PD-1 is the right se­lec­tion, the right tar­get based on many da­ta that we have gen­er­at­ed our­selves and our part­ner, Re­gen­eron. We do be­lieve that there is a po­ten­tial for PD-1 to be su­pe­ri­or. We do not know that at this point. I wouldn’t ven­ture to say that this is proven at this point. There are hints that in­deed tell us that, and we’ve made a choice and this is the choice we’re mak­ing here. We’re go­ing af­ter PD-1. We do not have a PD-L1 in de­vel­op­ment.

A suc­cess here would set up Sanofi as a play­er in on­col­o­gy, with a com­bo ap­proach that could well help over­come the sting of past set­backs. Sanofi’s in­ter­nal pipeline has been unin­spired at best for years, with Re­gen­eron lend­ing a big hand to pro­vide the kind of re­mark­able in­no­va­tion that is need­ed for a re­boot. That part­ner­ship has al­ready land­ed sev­er­al land­mark drug OKs, and now it’s lin­ing up an­oth­er.

Look for the num­ber 6 PD-(L)1 to hit the reg­u­la­to­ry re­view path soon. It isn’t an­gling for last place.

John Hood [file photo]

UP­DATE: Cel­gene and the sci­en­tist who cham­pi­oned fe­dra­tinib's rise from Sanofi's R&D grave­yard win FDA OK

Six years after Sanofi gave it up for dead, the FDA has approved the myelofibrosis drug fedratinib, now owned by Celgene.

The drug will be sold as Inrebic, and will soon land in the portfolio at Bristol-Myers Squibb, which is finalizing a deal to acquire Celgene.

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UP­DAT­ED: AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

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Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

The top 10 fran­chise drugs in bio­phar­ma his­to­ry will earn a to­tal of $1.4T (tril­lion) by 2024 — what does that tell us?

Just in case you were looking for more evidence of just how important Amgen’s patent win on Enbrel is for the company and its investors, EvaluatePharma has come up with a forward-looking consensus estimate on what the list of top 10 drugs will look like in 2024.

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ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

ICER has some strong words for PTC, Sarepta and the FDA as the US drug price watchdog concludes that as currently priced, their respective new treatments for Duchenne muscular dystrophy are decidedly not cost-effective.

The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.

The key dates for KRAS watch­ers through the end of the year — the trail is nar­row and risks are ex­treme

There’s nothing quite like a big patent win when it comes to burnishing your prospects in the pipeline. And for Amgen, which seems to have rescued Enbrel for a run to 2029, the cheering section on Wall Street is now fixed on AMG 510 and a key rival.

And it didn’t take much data to do it. 

There was the first snapshot of a handful of patients, with a 50% response rate. Then came word that Amgen researchers are also tracking responses in different cancers, at least one in colorectal cancer and appendiceal too. 

Bain's Or­ly Mis­han joins Pfiz­er's neu­ro spin­out Cerev­el; On­colyt­ic virus biotech taps Sil­la­Jen ex­ec He­le­na Chaye as CEO

→ Bain Capital is deploying one of its top investors to Cerevel Therapeutics, steering a $350 million-plus neuro play carved out of Pfizer. Orly Mishan — a co-founder and principal of Bain’s life sciences unit — was involved in the partnership that birthed the biotech spinout in the first place. As Cerevel’s first chief business officer, she is tasked with corporate development, program management as well as technical operations. 

UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

The executive team at Alector $ALEC has a bone to pick with scientific co-founder Asa Abeliovich. Their latest quarterly rundown has this brief note buried inside:

On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

There’s no explicit word in the filing on what kind of confidential info was involved, but the proceeding got started 2 days ahead of Abeliovich’s IPO.

Abeliovich, formerly a tenured associate professor at Columbia, is a top scientist in the field of neurodegeneration, which is where Alector is targeted. More recently, he’s also helped start up Prevail Therapeutics as the CEO, which raised $125 million in an IPO. And there he’s planning on working on new gene therapies that target genetically defined subpopulations of Parkinson’s disease. Followup programs target Gaucher disease, frontotemporal dementia and synucleinopathies.

But this time Abeliovich is the CEO rather than a founding scientist. And some of their pipeline overlaps with Alector’s.

Abeliovich and Prevail, though, aren’t taking this one lying down.

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Chi­na has be­come a CEO-lev­el pri­or­i­ty for multi­na­tion­al phar­ma­ceu­ti­cal com­pa­nies: the trend and the im­pli­ca­tions

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.