Sanofi faces a make-or-break year, say­ing it’s ready to put a 3-prong R&D strat­e­gy to a piv­otal test

With the help of some key de­vel­op­ment part­ners, Sanofi set out to­day to try and prove to an in­creas­ing­ly skep­ti­cal group of in­vestors that it can con­trol its own des­tiny with a late-stage pipeline bristling with block­buster con­tenders.

Oliv­er Brandi­court, Sanofi CEO

But with a decade-long track record large­ly ab­sent of big, in-house suc­cess sto­ries and its “block­buster” Deng­vax­ia in a sham­bles, caught up in a glob­al safe­ty cri­sis that has crushed its po­ten­tial, that’s no easy task.

Un­der CEO Olivi­er Brandi­court and chief sci­en­tist Elias Zer­houni, the phar­ma gi­ant has pur­sued a three-prong R&D strat­e­gy for two years: Shift from small mol­e­cules to bi­o­log­ics; ad­vance from a monother­a­py ap­proach to com­bos; and drop the re­liance on li­cens­ing with a new em­pha­sis on de­vel­op­ing their own, whol­ly-owned as­sets.

That’s a work in progress, though, even as Sanofi touts a shot at 9 reg­u­la­to­ry sub­mis­sions in 18 months with the launch of 10 piv­otal tri­als by the end of 2018. The out­line al­so in­cludes one shelved pro­gram and key part­ner­ships with Re­gen­eron, Al­ny­lam, Myokar­dia and Prin­cip­ia.

The key ques­tion here is whether a big op­er­a­tion like Sanofi, which has re­lied on al­liances to pro­duce in­no­va­tion, can ac­tu­al­ly do some­thing im­por­tant on its own. And this af­ter for­mer CEO Chris Viehbach­er con­clud­ed that it was too big and too slow to ac­com­plish any­thing sig­nif­i­cant with­out lots of help. In­vestors, mean­while, have been grum­bling through­out CEO Olivi­er Brandi­court’s rein that the com­pa­ny’s best hope for fast progress lies in M&A, which has yet to ma­te­ri­al­ize.

Elias Zer­houni

Sanofi’s R&D pre­sen­ta­tion starts with a boast about the Phase III po­ten­tial of Dupix­ent — part­nered with Re­gen­eron — for COPD. Sanofi al­so has high hopes for an­oth­er an­ti-in­flam­ma­to­ry, the an­ti-IL-33 drug SAR440340, part­nered with Re­gen­eron. And they sep­a­rate­ly her­ald­ed the pos­i­tive piv­otal da­ta for their PD-1 can­cer check­point drug cemi­plimab, al­so part­nered with Re­gen­eron.

The pipeline re­view notes that Sanofi is tak­ing this treat­ment in­to a piv­otal pro­gram for front­line lung can­cer — a field that has at­tract­ed the in­tense in­ter­est of every ma­jor check­point play­er.

Here’s the rest of the line­up now in the spot­light, with a break­down by dis­ease fo­cus:

On­col­o­gy

  • Isat­ux­imab, a home grown an­ti-CD38 an­ti­body, is in the clin­ic for mul­ti­ple myelo­ma and will be stud­ied as a com­bi­na­tion ther­a­py with their check­point cemi­plimab, with in­ves­ti­ga­tors pur­su­ing signs that the add-on could have an ef­fect on PD-1 re­sis­tance. This drug rep­re­sents some re­newed ex­pec­ta­tions in on­col­o­gy, close to three years af­ter a re­or­ga­ni­za­tion on the can­cer front fol­low­ing some sig­nif­i­cant fail­ures and the de­par­ture of Tal Zaks.

Mul­ti­ple scle­ro­sis

  • Lem­tra­da — ac­quired in the Gen­zyme buy­out — is be­ing stud­ied for the pri­ma­ry, pro­gres­sive form of mul­ti­ple scle­ro­sis. An­oth­er drug, GLD-52, is be­ing “de­pri­or­i­tized” to keep the spot­light on Lem­tra­da. And there’s a BTK drug for MS part­nered with Prin­cip­ia.

Rare dis­eases

  • There’s a drug called venglu­s­tat for rare cas­es of au­to­so­mal dom­i­nant poly­cys­tic kid­ney dis­ease. And the phar­ma gi­ant sees po­ten­tial here for Fab­ry Dis­ease, Gauch­er Dis­ease Type 3 and GBA Parkin­son’s Dis­ease.
  • Two oth­er rare dis­ease drugs — olipu­dase and aval­glu­cosi­dase al­fa — are get­ting the spot­light treat­ment to­day.
  • And the fo­cus on rare dis­eases fin­ish­es with the al­liance with Al­ny­lam, which is do­ing the de­vel­op­ment work for patisir­an and fi­tusir­an.

Di­a­betes and car­dio

  • There’s a once-week­ly GLP-1 drug look­ing to join the field. It’s called ef­pe­gle­natide.
  • An oral dual ag­o­nist of GLP-1/GCG is get­ting a shoutout for a Phase III in obe­si­ty next year, along with a mid-stage pro­gram for NASH.
  • The big part­ner­ship hope here lies at Myokar­dia, which is al­lied with Sanofi on mava­camten, their lead drug for ge­net­i­cal­ly de­fined cas­es of car­diomy­opa­thy.

Vac­cines

  • Af­ter two huge dis­as­ters in vac­cines in the last few days, Sanofi high­lights its work on RSV, but it’s still in Phase II.

R&D chief Zer­houni summed it up:

“2018 will be an im­por­tant year as we ex­pect mul­ti­ple mile­stones for Sanofi’s late-stage pipeline, made pos­si­ble through the pri­or­i­ti­za­tion prin­ci­ples we have con­sis­tent­ly ap­plied to our ear­ly-stage re­search pro­grams.”

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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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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'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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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.

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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