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.”

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

The End­points 11: They've got mad mon­ey and huge am­bi­tions. It's time to go big or go home

These days, selecting a group of private biotechs for the Endpoints 11 spotlight begins with a sprint to get ahead of IPOs and the M&A teams at Big Pharma. I’ve had a couple of faceplants earlier this year, watching some of the biotechs on my short list choose a quick leap onto Nasdaq or into the arms of a buyer.

Vividion, you would have been a great pick for the Endpoints 11. I’m sorry I missed you.

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Dave Lennon, former president of Novartis Gene Therapies

So what hap­pened with No­var­tis Gene Ther­a­pies? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

FDA+ roundup: Bs­U­FA III ready for show­time, court tells FDA to re-work com­pound­ing plan, new guid­ance up­dates and more

The FDA has now spelled out what exactly will be included in the third iteration of Biosimilar User Fee Act (BsUFA) from 2023 through 2027, which similarly to the prescription drug deal, sets fees that industry has to pay for submitting applications, in exchange for firm timelines that the agency must meet.

This latest deal includes several sweeteners for the biosimilar industry, which has yet to make great strides in the US market, with shorter review timelines for safety labeling updates and updates to add or remove an indication that does not contain efficacy data.

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Joshua Liang, Clover Biopharmaceuticals CEO

With world still in sore need of dos­es, Clover says its Covid-19 vac­cine is 67% ef­fec­tive in Phase III

With concerns about the Delta variant rising and much of the world still in desperate need of vaccine doses, a Chinese biotech announced Wednesday that a new shot has shown positive results in a large trial against Covid-19, including new variants.

Clover Biopharmaceuticals announced Wednesday that its vaccine candidate showed 79% efficacy against the Delta variant in a Phase II/III trial dubbed Spectra, and 67% effective against Covid-19 overall.

Jean Bennett (Brent N. Clarke/Invision/AP Images)

Lux­tur­na in­ven­tor Jean Ben­nett starts a new gene ther­a­py com­pa­ny to tack­le rare dis­eases left be­hind by phar­ma, VCs

A few years ago Jean Bennett found herself in a surprising place for a woman who invented the first gene therapy ever approved in the United States: No one, it seemed, wanted her work.

Bennett, who designed and co-developed Luxturna, approved in 2018 for a rare form of blindness, had kept building new gene therapies for eye diseases at her University of Pennsylvania lab. But although the results in animals looked promising, pharma companies and investors kept turning down the pedigreed ophthalmology professor.

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Maureen Hillenmeyer, Hexagon Bio CEO

Hexa­gon Bio rais­es $61M to con­tin­ue ef­forts to turn fun­gi in­to drugs

A year after raising a $47 million launch round, the fungi-loving drug hunters at Hexagon Bio have more than doubled their coffers.

Hexagon announced today that it raised another $61 million for its efforts to design cancer and infectious disease drugs based on insights mined from the DNA in millions of species of fungi. The new financing brings Hexagon’s committed funding to over $108 million.