Swap­ping stock and cash, Io­n­is turns to close­ly-con­trolled Akcea for a deal on spurned drug in­ot­ersen

Last year, af­ter Glax­o­SmithK­line un­cer­e­mo­ni­ous­ly dumped its part­ner­ship with Io­n­is on in­ot­ersen and a fol­low-up rare dis­ease ther­a­py, the chief busi­ness of­fi­cer at the biotech said that she was in talks with mul­ti­ple po­ten­tial part­ners in­ter­est­ed in tak­ing GSK’s place.

Sarah Boyce

To­day, Io­n­is un­veiled the win­ning play­er at the bar­gain­ing ta­ble: Akcea, an af­fil­i­ate of Io­n­is, which owns a con­trol­ling in­ter­est in its stock. And Sarah Boyce, the CBO in charge of the talks at Io­n­is, is now trans­fer­ring over to be­come pres­i­dent of Akcea as it preps a like­ly com­mer­cial launch in the US and Eu­rope.

Akcea is al­so get­ting Io­n­is’ sec­ond-gen drug for hered­i­tary TTR amy­loi­do­sis, the ex­per­i­men­tal IO­N­IS-TTR-LRx.

To­geth­er, they will most prob­a­bly square off against Al­ny­lam’s ri­val patisir­an, which a line­up of an­a­lysts be­lieve is poised to hand­i­ly carve out the largest share of the mar­ket.

Mon­ey or stock is ex­chang­ing hands — in a man­ner of speak­ing, as Io­n­is car­ries Akcea on its books, ac­cord­ing to its re­cent an­nu­al re­port. Akcea is pay­ing Io­n­is $150 mil­lion and Io­n­is is buy­ing $200 mil­lion in Akcea shares, boost­ing its stake in the com­pa­ny from 68% to 75% as the mon­ey stays in the fam­i­ly.

Ap­provals in the US and Eu­ro­pean mar­kets will trig­ger pay­ments of $50 mil­lion and $40 mil­lion to Io­n­is. Akcea can use stock in lieu of cash, if it choos­es, to pay for the up­front and fees, which would fur­ther swell Io­n­is’ stake in the com­pa­ny. And there’s an­oth­er $1.3 bil­lion in prospec­tive mile­stones out­lined for mar­ket­ing goals, while Io­n­is gets the li­on’s share of a 60/40 split with its af­fil­i­ate.

In­vestors clear­ly saw it as a boon for Akcea and a dis­ap­point­ment for Io­n­is. Akcea’s stock went up 23% and Io­n­is slumped 6% on the news. But in this case, Io­n­is wins ei­ther way.

Just how much their deal is worth, though, will de­pend a lot on the prospec­tive launch of Al­ny­lam’s patisir­an, which has bet­ter ef­fi­ca­cy da­ta. Akcea, where Io­n­is CEO Stan­ley Crooke sits on the board, is adopt­ing their mar­ket­ing stance from Io­n­is.

Paula Soteropou­los, Akcea CEO

As part of their due dili­gence for the deal, Akcea CEO Paula Soteropou­los told me, she came to the con­clu­sion that in­ot­ersen is “a very trans­for­ma­tive drug for pa­tients. In the clin­i­cal tri­al, in­ot­ersen showed a sig­nif­i­cant im­pact in the dis­ease and im­pact on life.” Pa­tients can dose them­selves at home, any­time and any­place.

“It gives them con­trol of their life back,” she adds, “the dis­ease robs pa­tients of their abil­i­ty to lead a nor­mal life.” And that will dif­fer­en­ti­ate the drug from patisir­an.

Af­ter I queried Soteropou­los on the close ties be­tween the two com­pa­nies dur­ing an in­ter­view, a spokesper­son fol­lowed up with a state­ment.

Io­n­is con­duct­ed a thor­ough process to eval­u­ate po­ten­tial part­ners and types of trans­ac­tion for in­ot­ersen.  As you might ex­pect, in­ter­est in in­ot­ersen took a va­ri­ety of forms, from larg­er com­pa­nies that want­ed glob­al rights with no Io­n­is par­tic­i­pa­tion to more re­gion­al trans­ac­tions; from com­pa­nies with sub­stan­tial rare dis­ease ex­per­tise to those with less.  We had strong in­ter­est from a range of com­pa­nies and re­ceived mul­ti­ple of­fers.

Af­ter a thought­ful eval­u­a­tion of all the pos­si­bil­i­ties, we ar­rived at the con­clu­sion that this trans­ac­tion with Akcea met all three of our key part­ner­ing goals: to rapid­ly de­liv­er in­ot­ersen to the pa­tients who des­per­ate­ly need this treat­ment, to max­i­mize the com­mer­cial suc­cess of in­ot­ersen, and to op­ti­mize our com­mer­cial par­tic­i­pa­tion in our TTR fran­chise.  The trans­ac­tion has the added ben­e­fit of be­ing trans­for­ma­tive for Akcea which is now po­si­tioned to launch two drugs for sig­nif­i­cant rare dis­eases this year, sub­stan­tial­ly in­creas­ing the breadth of Akcea’s op­er­a­tions and ca­pa­bil­i­ties.

The deal gives Akcea a new drug to launch along­side their drug volane­sors­en, part of the pipeline the biotech got when it spun out of Io­n­is. And the CEO says that adding in­ot­ersen to that sched­ule makes sense, giv­en the com­mer­cial in­fra­struc­ture they’ve been build­ing. She added that a rare dis­ease drug like in­ot­ersen would typ­i­cal­ly re­quire a sales group of about 100 in the US, with Eu­ro­pean staffing break­ing out on an as need­ed, coun­try-by-coun­try ba­sis.

Io­n­is’ PDU­FA date is Ju­ly 6, a lit­tle more than a month ahead of the patisir­an ac­tion date.

Hal Barron, GSK

Break­ing the death spi­ral: Hal Bar­ron talks about trans­form­ing the mori­bund R&D cul­ture at GSK in a crit­i­cal year for the late-stage pipeline

Just ahead of GlaxoSmithKline’s Q2 update on Wednesday, science chief Hal Barron is making the rounds to talk up the pharma giant’s late-stage strategy as the top execs continue to woo back a deeply skeptical investor group while pushing through a whole new R&D culture.

And that’s not easy, Barron is quick to note. He told the Financial Times:

I think that culture, to some extent, is as hard, in fact even harder, than doing the science.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Aca­dia is mak­ing the best of it, but their lat­est PhI­II Nu­plazid study is a bust

Acadia’s late-stage program to widen the commercial prospects for Nuplazid has hit a wall. The biotech reported that their Phase III ENHANCE trial flat failed. And while they $ACAD did their best to cherry pick positive data wherever they can be found, this is a clear setback for the biotech.

With close to 400 patients enrolled, researchers said the drug flunked the primary endpoint as an adjunctive therapy for patients with an inadequate response to antipsychotic therapy. The p-value was an ugly 0.0940 on the Positive and Negative Syndrome Scale, which the company called out as a positive trend.

Their shares slid 12% on the news, good for a $426 million hit on a $3.7 billion market cap at close.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Some Big Phar­mas stepped up their game on da­ta trans­paren­cy — but which flunked the test?

The nonprofit Bioethics International has come out with their latest scorecard on data transparency among the big biopharmas in the industry — flagging a few standouts while spotlighting some laggards who are continuing to underperform.

Now in its third year, the nonprofit created a new set of standards with Yale School of Medicine and Stanford Law School to evaluate the track record on trial registration, results reporting, publication and data-sharing practice.

Busy Gilead crew throws strug­gling biotech a life­line, with some cash up­front and hun­dreds of mil­lions in biobucks for HIV deal

Durect $DRRX got a badly needed shot in the arm Monday morning as Gilead’s busy BD team lined up access to its extended-release platform tech for HIV and hepatitis B.

Gilead, a leader in the HIV sector, is paying a modest $25 million in cash for the right to jump on the platform at Durect, which has been using its technology to come up with an extended-release version of bupivacaine. The FDA rejected that in 2014, but Durect has been working on a comeback.

In­tec blitzed by PhI­II flop as lead pro­gram fails to beat Mer­ck­'s stan­dard com­bo for Parkin­son’s

Intec Pharma’s $NTEC lead drug slammed into a brick wall Monday morning. The small-cap Israeli biotech reported that its lead program — coming off a platform designed to produce a safer, more effective oral drug for Parkinson’s — failed the Phase III at the primary endpoint.

Researchers at Intec, which has already seen its share price collapse over the past few months, says that its Accordion Pill-Carbidopa/Levodopa failed to prove superior to Sinemet in reducing daily ‘off’ time. 

Cel­gene racks up third Ote­zla ap­proval, heat­ing up talks about who Bris­tol-My­ers will sell to

Whoever is taking Otezla off Bristol-Myers Squibb’s hands will have one more revenue stream to boast.

The drug — a rising star in Celgene’s pipeline that generated global sales of $1.6 billion last year — is now OK’d to treat oral ulcers associated with Behçet’s disease, a common symptom for a rare inflammatory disorder. This marks the third FDA approval for the PDE4 inhibitor since 2014, when it was greenlighted for plaque psoriasis and psoriatic arthritis.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors.

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Apotex, though, has been a disaster ground. The manufacturer voluntarily yanked the ANDAs on 31 drugs — in late 2017 — after the FDA came across serious manufacturing deficiencies at their plants in India. A few days ago, the FDA made it official.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.