Prize fight in Paris: Al­ny­lam and Io­n­is slug it out over PhI­II da­ta in prepa­ra­tion for a block­buster show­down

This morn­ing Al­ny­lam $AL­NY rolled out a bat­tery of stel­lar Phase III da­ta on their block­buster con­tender patisir­an, which promis­es to go on to be­come the com­pa­ny’s first ap­proved and mar­ket­ed drug af­ter 15 long years of RNAi dis­cov­ery and de­vel­op­ment work. And as of now, it looks like they’ll be go­ing head-to-head with the an­ti­sense ex­perts at Io­n­is $IONS, the un­der­dogs in what promis­es to be a long run­ning fight for a small group of pa­tients suf­fer­ing from rare cas­es of hered­i­tary AT­TR (hAT­TR) amy­loi­do­sis.

John Maraganore

For Al­ny­lam, the key points in the APOL­LO da­ta un­veiled to­day in Paris fo­cus on an im­pres­sive 34-point mean dif­fer­ence in the mod­i­fied neu­ropa­thy im­pair­ment score (mNIS+7) at 18 months, with a 6-point im­prove­ment for pa­tients which un­der­scores that they got some­what bet­ter over that pe­ri­od. There was al­so a 21.1-point mean im­prove­ment in the qual­i­ty of life score, with a 6.7-point im­prove­ment to show that the pa­tients al­so felt bet­ter.

Just hours ear­li­er, it was Io­n­is that took cen­ter stage, adding to its Phase III da­ta roll­out with a 19.73-point mean change in dis­ease scale at 15 months, and an 8.69-point ben­e­fit at 8 months. Their qual­i­ty of life scores reg­is­tered an 11.68-point mean im­prove­ment, with a 6.4-point ben­e­fit that showed their pa­tients al­so re­spond­ed bet­ter with treat­ment.

“We beat them on both,” Al­ny­lam CEO John Maraganore tells me flat­ly, “with the caveat that these aren’t com­par­a­tive (head-to-head) stud­ies. We clear­ly have a more sub­stan­tial treat­ment ef­fect.”

“This is a big win,” adds the CEO. “It’s a nice way to de­but the ad­vent of this tech­nol­o­gy in med­i­cine.”

The mar­ket agreed with Maraganore. Al­ny­lam shares shot up 13%, with Io­n­is down 8% af­ter the matchup.

Stan­ley Crooke. Im­age: OTS

Maraganore — who’s stuck with RNAi through the good years and the bad as Big Phar­ma dropped out or came in­to the emerg­ing field — hasn’t made his own peak sales pro­jec­tions here, but he al­so isn’t shy of not­ing an­a­lysts’ pro­jec­tions rang­ing up to $2 bil­lion worth of year­ly rev­enue. And he’s al­ready been ramp­ing up com­mer­cial ac­tiv­i­ties in the US and Eu­rope, while their big part­ner Sanofi $SNY takes on the rest of the world.

Io­n­is CEO Stan Crooke has heard the an­a­lysts cheer­ing Al­ny­lam. And he’s not about to roll over and play dead now.

“We think we’ll win in the mar­ket­place,” Crooke told me in the lead-up to to­day’s da­ta re­veal, adding that “there are peo­ple who think oth­er­wise.”

So what does Io­n­is have that Al­ny­lam doesn’t? Crooke — who hadn’t seen the Al­ny­lam re­sults when we talked ahead of Thurs­day’s ses­sions — says there’s lots on the ta­ble.

First, he can point to 8-month and 15-month re­sults which demon­strate sig­nif­i­cant suc­cess ahead the 18-month mark at Al­ny­lam. He’s not talk­ing price yet — no one does at this stage — but “the cost of ther­a­py will be sub­stan­tial­ly bet­ter.” And he’s heard Al­ny­lam hint­ing about Soliris style prices, which could land it on the list of the top 10 most ex­pen­sive ther­a­pies.

“We’ll win be­cause we think in­ot­ersen is easy to use,” he adds, adding point­ed­ly that this isn’t their first time out on the mar­ket. Where the Al­ny­lam ther­a­py has to be in­fused, ex­pos­ing pa­tients to re­ac­tions that can leave them un­able to work, he’ll be sell­ing a treat­ment that can be self-ad­min­is­tered at home.

Io­n­is, though, al­so had to deal with the death of a pa­tient from throm­bo­cy­tope­nia dur­ing its study, throw­ing in an added safe­ty fac­tor that could play to Al­ny­lam’s ad­van­tage — as many an­a­lysts would be will­ing to tell you. Since that case oc­curred, though, the biotech added a more care­ful screen­ing process to pre­vent any new in­ci­dents.

“I think both drugs will be very suc­cess­ful,” Crooke sums up. But any­one who counts Io­n­is out now, he adds, is in for a big sur­prise.

The sales num­bers will pro­vide the score on who’s win­ning.

Brent Saunders [Getty Photos]

UP­DAT­ED: Ab­b­Vie seals $63B deal to buy a trou­bled Al­ler­gan — spelling out $1B in R&D cuts

Brent Saunders has found his way out of the current fix he’s in at Allergan $AGN. He’s selling the company to AbbVie for $63 billion in the latest example of the hot M&A market in biopharma.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,600+ biopharma pros reading Endpoints daily — and it's free.

Two biotech uni­corns swell pro­posed IPOs, eye­ing a $600M-plus wind­fall

We’ve been wait­ing for the ar­rival of Bridge­Bio’s IPO to top off the wave of new biotech of­fer­ings sweep­ing through Nas­daq at the end of H1. And now we learn that it’s been sub­stan­tial­ly up­sized.

Ini­tial­ly pen­ciled in at a uni­corn-sized $225 mil­lion, the KKR-backed biotech has spiked that to the neigh­bor­hood of $300 mil­lion, look­ing to sell 20 mil­lion shares at $14 to $16 each. That’s an added 5 mil­lion shares, re­ports Re­nais­sance Cap­i­tal, which fig­ures the pro­posed mar­ket val­u­a­tion for Neil Ku­mar’s com­pa­ny at $1.8 bil­lion.

No­var­tis holds back the copy­cat brigade's at­tack on its top drug fran­chise — for now

A fed­er­al judge has put a gener­ic chal­lenge to No­var­tis’ block­buster mul­ti­ple scle­ro­sis drug Gilenya on hold while a patent fight plays out in court.

Judge Leonard P. Stark is­sued a tem­po­rary in­junc­tion ear­li­er this week, forc­ing My­lan, Dr. Red­dy’s Lab­o­ra­to­ries and Au­robindo Phar­ma to shelve their launch plans to al­low the patent fight to pro­ceed. He ruled that al­low­ing the gener­ics in­to the mar­ket now would per­ma­nent­ly slash the price for No­var­tis, even if it pre­vails. 

Af­ter rais­ing $158M, this up­start's founders have star back­ers and plans to break new ground in gene ther­a­py

Back in 2014, Stephanie Tagliatela opted to take an early exit out of her PhD program after working in Mark Bear’s lab at MIT, where she specialized in the synaptic connections between neuronal cells in the brain. She never finished that PhD, but she and fellow MIT student Kartik Ramamoorthi — who was on the founding team at Voyager — came away with some ideas for a gene therapy startup.

Today, fully 5 years later, she and Ramamoorthi are taking the wraps off of a $104 million mega-round designed to take the cumulative work of their preclinical formative stage for Encoded Therapeutics into human studies. They’ve now raised $158 million since starting out in Illumina’s incubator in the Bay Area, and they believe they are firmly on track to do something unique in gene therapy.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,600+ biopharma pros reading Endpoints daily — and it's free.

Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

Sanofi/Re­gen­eron mus­cle ahead of a ri­val No­var­tis/Roche team, win first ap­proval in key rhi­nos­i­nusi­tis field

Re­gen­eron and their part­ners at Sanofi have beat the No­var­tis/Roche team to the punch on an­oth­er key in­di­ca­tion for their block­buster an­ti-in­flam­ma­to­ry drug Dupix­ent. The drug team scored an ac­cel­er­at­ed FDA ap­proval for chron­ic rhi­nos­i­nusi­tis with nasal polyps, mak­ing this the first such NDA for the field.

An­a­lysts have been watch­ing this race for awhile now, as Sanofi/Re­gen­eron won a snap pri­or­i­ty re­view for what is now their third dis­ease in­di­ca­tion for this treat­ment. And they’re not near­ly done, build­ing up hopes for a ma­jor fran­chise.

FDA re­jects Ac­er's rare dis­ease drug, asks for new tri­al — shares crater

Ac­er Ther­a­peu­tics’ bid to re­pur­pose celipro­lol — a be­ta-block­er on the mar­ket for hy­per­ten­sion — as a treat­ment for a rare, in­her­it­ed con­nec­tive tis­sue dis­or­der has hit a se­vere set­back. The New­ton, Mass­a­chu­setts-based com­pa­ny on Tues­day said the FDA re­ject­ed the drug and has asked for an­oth­er clin­i­cal tri­al.

The com­pa­ny’s shares $AC­ER cratered near­ly 77% to $4.47 in Tues­day morn­ing trad­ing.

Richard Gonzalez testifying in front of Senate Finance Committee, February 2019 [AP Images]

Ab­b­Vie's $63B buy­out spot­lights the re­turn of ma­jor M&A deals — de­spite the back­lash

Big time M&A is back. But for how long?

Over the past 18 months we’ve now seen three major buyouts announced: Takeda/Shire; Bristol-Myers/Celgene and now AbbVie/Allergan. And with this latest deal it’s increasingly clear that the sharp fall from grace suffered by high-profile players which have seen their share prices blasted has created an opening for the growth players in big pharma to up their game — in sharp contrast to the popular bolt-on deals that have been driving the growth strategy at Novartis, Merck, Roche and others.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,600+ biopharma pros reading Endpoints daily — and it's free.

Top an­a­lyst finds a sil­ver lin­ing in Ab­b­Vie’s $63B Al­ler­gan buy­out — but there’s a catch

Af­ter get­ting beat up on all sides from mar­ket ob­servers who don’t much care for the lat­est mega-deal to ar­rive in bio­phar­ma, at least one promi­nent an­a­lyst now is start­ing to like what he sees in the num­bers for Ab­b­Vie/Al­ler­gan.

But it’s go­ing to take some en­cour­age­ment if Ab­b­Vie ex­ecs want it to last.

Ab­b­Vie’s mar­ket cap de­clined $20 bil­lion on Tues­day as the stock took a 17% hit dur­ing the day. And SVB Leerink’s Ge­of­frey Porges can see a dis­tinct out­line of an up­side af­ter re­view­ing the fun­da­men­tals of the deal.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,600+ biopharma pros reading Endpoints daily — and it's free.