Io­n­is and Akcea get their shot at com­pet­ing with Al­ny­lam for a rare dis­ease group — but it’s not ex­pect­ed to be pret­ty

Io­n­is $IONS and Akcea $AK­CA have been giv­en a green light to go head-to-head in­to a mar­ket­ing fight with Al­ny­lam for treat­ing ex­treme­ly rare cas­es of TTR amy­loi­do­sis — with one arm tied be­hind their back on ef­fi­ca­cy and both com­mer­cial feet hob­bled by a wor­ri­some safe­ty is­sue.

Akcea — a spin­out from Io­n­is, which still con­trols a ma­jor­i­ty of the com­pa­ny — put out the word of the de­layed OK for Tegse­di (in­ot­ersen) on Fri­day night, which comes af­ter a reg­u­la­to­ry de­lay that put them one step be­hind the com­pe­ti­tion. 

It’s not the de­lay that presents the biggest prob­lem, though. That hon­or goes to the black box warn­ing about the drug’s side ef­fects, which in­cludes the risk of throm­bo­cy­tope­nia — low platelet counts that car­ry the risk of se­ri­ous bleed­ing. Pa­tients will need to be care­ful­ly mon­i­tored for the side ef­fects, while Al­ny­lam’s drug comes with a much clean­er safe­ty pro­file that will be clear­ly pre­ferred by the pa­tient pop­u­la­tion and their doc­tors.

Steve Miller

The ap­proval did come with an up­beat com­ment from Ex­press Scripts CMO Steve Miller, who spot­light­ed the week­ly in­jec­tion reg­i­men — a slight ad­van­tage in Akcea’s fa­vor — and the chance for some head-to-head price dis­counts.

“Tegse­di is a week­ly self-ad­min­is­tered in­jec­tion, that should re­duce ad­min­is­tra­tion costs and drug wastage,” not­ed an ap­prov­ing Miller. “By in­tro­duc­ing com­pe­ti­tion in­to the mar­ket­place this should dri­ve down the to­tal cost of care.”

The com­pa­nies, though, start out at ex­act­ly the same price of $450,000 a year whole­sale, which Al­ny­lam set. Any dis­count­ing on price will now be worked out with pay­ers, and Al­ny­lam is vow­ing to get ag­gres­sive to stay on top, hop­ing to prove that its shift to mar­ket­ing can be ac­com­plished with ex­per­tise. Al­ny­lam has es­ti­mat­ed the net price at $345,000 with in­di­vid­ual deals be­ing worked out on a pay-for-per­for­mance ba­sis.

Not to be dis­count­ed in all this is Pfiz­er $PFE, which is hus­tling along tafamidis af­ter scor­ing pos­i­tive da­ta on transthyretin amy­loid car­diomy­opa­thy. Pfiz­er is al­ready rolling out an ex­pand­ed ac­cess pro­gram that will sign up pa­tients with the dis­ease as they shoot for a quick OK of their own.

This dis­ease is trig­gered by a mis­fold­ing pro­tein which then builds up in or­gans, threat­en­ing pa­tients with an ear­ly death. 

The Akcea/Io­n­is team have had plen­ty of set­backs to deal with this year. Most no­tably the FDA re­cent­ly re­ject­ed volane­sors­en, their drug for reg­u­lat­ing plas­ma triglyc­eride for pa­tients with rare cas­es of fa­mil­ial chy­lomi­crone­mia syn­drome, even af­ter an ex­pert pan­el re­view gave the com­pa­nies a thumbs up on mar­ket­ing. The re­jec­tion forced the com­pa­ny to re­struc­ture, lay­ing off 10% of its work­force as they plan a come­back.

Top­ping it all off, Io­n­is CEO Stan­ley Crooke opt­ed to jump off the Akcea board Mon­day, de­part­ing a com­pa­ny he cre­at­ed. He’ll be re­placed by Io­n­is BD chief Damien McDe­vitt.

Akcea’s stock dropped 7% Mon­day morn­ing. Io­n­is dropped 4%. Al­ny­lam was down too, just un­der 2%.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

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Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

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José Basel­ga finds promise in new class of RNA-mod­i­fy­ing can­cer tar­gets, lock­ing in 3 pre­clin­i­cal pro­grams with $55M

Having dived early into some of the RNA breakthroughs of the last decades — betting on Moderna’s mRNA tech and teaming up with Silence on the siRNA front — AstraZeneca is jumping into a new arena: going after proteins that modify RNA.

Their partner of choice is Accent Therapeutics, which is receiving $55 million in upfront payment to steer a selected preclinical program through to the end of Phase I. After AstraZeneca takes over, the Lexington, MA-based startup has the option to co-develop and co-commercialize in the US — and collect up to $1.1 billion in milestones in the long run. The deal also covers two other potential drug candidates.

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UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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