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.”

Covid-19 roundup: Eu­rope pur­chas­es 80M dos­es of Mod­er­na's vac­cine; CO­V­AXX se­cures $2.8B in emerg­ing mar­ket pre-or­ders

With the announcement of its vaccine efficacy data last week, Moderna is starting to line up customers for its Covid-19 mRNA jabs.

The Massachusetts-based biotech announced Wednesday it has agreed to sell an initial round of 80 million doses to the European Commission, with the option to double the amount to 160 million. Once the member states rubber stamp the approval, the deal will be finalized.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,200+ biopharma pros reading Endpoints daily — and it's free.

Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,200+ biopharma pros reading Endpoints daily — and it's free.

Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan.

The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,200+ biopharma pros reading Endpoints daily — and it's free.

Vivek Ramaswamy (Jeff Rumans/JPM 2020)

Urovan­t's lead drug dis­ap­points in mid-stage study as first big FDA de­ci­sion looms

Just as Urovant gets ready for its first big FDA decision on vibegron, the drug has flopped in what would’ve been a follow-on indication.

In a Phase IIa trial involving women with abdominal pain due to irritable bowel syndrome, vibegron failed to meet the bar on improving “average worst abdominal pain” over 12 weeks, compared to placebo, among IBS-D patients.

There were actually slightly more responders in the placebo group than in the drug arm, with only 40.9% of those randomized to vigebron achieving at least a 30% decrease in “worst abdominal pain” in the past 24 hours. The trial enrolled 222 women but only 189 completed the study.

FDA hands Liq­uidia and Re­vance a CRL and de­fer­ral, re­spec­tive­ly, as Covid-19 cre­ates in­spec­tion chal­lenge

Two biotechs said they got turned away by the FDA on Wednesday, in part due to pandemic-related travel restrictions.

North Carolina-based Liquidia Technologies was handed a CRL for its lead pulmonary arterial hypertension drug, citing the need for more CMC data and on-site pre-approval inspections, which the FDA hasn’t been able to conduct due to travel restrictions. The agency also deferred its decision on Revance Therapeutics’ BLA for its frown line treatment, because it needs to inspect the company’s northern California manufacturing facility. The action, Revance emphasized, was not a CRL.

News brief­ing: FDA re­quests new tri­al for Reata's Friedre­ich's atax­ia pro­gram; J&J's Trem­fya picks up ex­pand­ed la­bel in Eu­rope

Three months after Reata Pharmaceuticals suggested its Friedreich’s ataxia program omaveloxolone could be delayed, the company revealed that is indeed going to be the case.

Reata $RETA shares took a nosedive Wednesday after the biotech revealed that the FDA said supplemental data for its pivotal trial did not strengthen the case for approval. As a result, the drug is likely to need another study before the FDA takes up the case.

Jef­frey Hat­field takes over from Diego Mi­ralles as CEO of Vi­vid­ion; Drag­on­fly scores a new ex­ec with COO Alex Lu­gov­skoy

→ San Diego protein degradation startup Vividion Therapeutics has made a change at the top with Jeffrey Hatfield taking the helm as CEO, replacing Diego Miralles six months after Roche forked over $135 million to collaborate with Vividion on their small molecule degraders. Hatfield is chairman of the board at miRagen Therapeutics and previously held the CEO job at Zafgen and Vitae Pharmaceuticals. He also had a series of leadership roles at Bristol Myers Squibb from 1996-2004, including SVP, immunology and virology divisions.

Chi­na opens the door for biotech in­vestors in Hong Kong to buy Shang­hai stocks, and vice ver­sa

When Shanghai’s STAR board began opening its doors to biotech, it was considered not just a rival to Nasdaq but also the stock exchange in Hong Kong. Those perceptions may take an amicable turn as China expands a mutual access program with the city.

The changes mean investors in mainland China will be able to own Hong Kong biotech chapter stocks, while those in Hong Kong — a much more internationally connected group — would have access to those listed on STAR. In effect, it turns the Shanghai market into a globally accessible exchange overnight while also broadening a key source of revenue for HKEX.

Bax­ter con­tin­ues on-shoring push with $50M In­di­ana ex­pan­sion

It’s been a banner year for the once humdrum business of manufacturing drugs, particularly vaccines. Billions have been spent ramping up facilities for Covid-19 jabs, while individual CDMOs have expanded their facilities, apparently anticipating demand or responding to a government-led push to onshore drug manufacturing.

Now Baxter Biopharma Solutions, the CDMO wing of the many-armed healthcare giant Baxter, is getting in on the game. On Tuesday, they announced plans to spend $50 million to expand their flagship, 600,000 square-foot facility in Bloomington, IN.