In a first for deuter­at­ed drugs, FDA (fi­nal­ly) OKs Te­va’s would-be Hunt­ing­ton’s block­buster Auste­do

Ten months af­ter the FDA put Te­va’s would-be block­buster deutetra­benazine on hold af­ter rais­ing some sus­pi­cions re­gard­ing cer­tain metabo­lites found in pa­tients, the FDA has giv­en the drug an OK for Hunt­ing­ton’s chorea.

Michael Hay­den, Te­va

This is a first FDA ap­proval in the world of deuter­at­ed drugs, which tweaks ther­a­pies so that they break down more slow­ly in pa­tients. That way you can use a low­er dose to greater ef­fect. In this case the drug is a small mol­e­cule in­hibitor of vesic­u­lar monoamine 2 trans­porter, or VMAT2, which is de­signed to reg­u­late the lev­els of dopamine in the brain.

The drug will be sold as Auste­do. Te­va shares $TE­VA surged 2.2% Mon­day evening.

A star-crossed Te­va paid $3.5 bil­lion to ac­quire Aus­pex for this drug two years ago, sup­pos­ed­ly set­ting up a slap shot at the first reg­u­la­to­ry OK. That ap­proval was very slow com­ing, but the pay­off has fi­nal­ly ar­rived.

The big draw­back in the ap­proval is that it came with a black box warn­ing on de­pres­sion and sui­ci­dal­i­ty. But­Te­va al­so wast­ed no time in tack­ling the mar­ket with an ag­gres­sive price point. Ja­son Ger­ber­ry at Leerink not­ed:

While physi­cians will titrate Auste­do, the av­er­age dosage is ex­pect­ed to be 24mg per day which TE­VA priced at $60,000 for a year of ther­a­py and is be­low the list price of (Valeant’s) brand Xe­nazine ($152K) and gener­ic tetra­benazine ($96K). We view tonight’s up­date as a pos­i­tive, elim­i­nat­ing any lin­ger­ing con­cerns around deep­er is­sues with the CRL (com­plete re­sponse let­ter) that de­layed Auste­do ap­proval. We cur­rent­ly fore­cast $850m in 2023E Auste­do sales.

Sev­er­al new drugs have now been re­leased with dis­count pric­ing, un­der­scor­ing a new, harsh­er en­vi­ron­ment on high drug prices.

Te­va has al­so been push­ing this drug along for tar­dive dysk­i­ne­sia, though its mixed batch of late-stage da­ta spurred some an­a­lysts to sing the prais­es of a com­pet­ing drug from Neu­ro­crine. Eval­u­atePhar­ma has tagged this drug as a po­ten­tial block­buster, with a shot at earn­ing slight­ly more than a bil­lion dol­lars a year — though that kind of cash won’t come eas­i­ly.

Still, the FDA ap­proval marks a big win for Te­va, which has been un­der the gun for years now. Cur­rent­ly be­ing re­struc­tured, the CEO de­part­ed re­cent­ly as Te­va’s gener­ic busi­ness is as­sault­ed by low­er prices and its brand­ed di­vi­sion en­dured a lengthy drought in R&D.

The ap­proval al­so marks a big plus for Con­cert Phar­ma­ceu­ti­cals $CNCE, which has been la­bor­ing at deuter­at­ing drugs with the heavy hy­dro­gen for years now.

Hunt­ing­ton’s, a lethal neu­rode­gen­er­a­tive dis­ease, is char­ac­ter­ized by harsh, repet­i­tive twist­ing and writhing as­so­ci­at­ed with chorea.

“Chorea is a ma­jor symp­tom for many liv­ing with Hunt­ing­ton dis­ease. It im­pacts pa­tients’ func­tion­al­i­ty and ac­tiv­i­ties of dai­ly liv­ing, and there have been lim­it­ed treat­ment op­tions for these pa­tients,” said Michael Hay­den, the CSO at Te­va. “Based on the re­sults demon­strat­ed in the clin­i­cal de­vel­op­ment pro­gram which sup­port­ed the ap­proval of AUSTE­DO and our on­go­ing com­mit­ment to pa­tients, we feel unique­ly po­si­tioned to bring this treat­ment op­tion for­ward.”

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

Re­gen­eron's Evkeeza shows promise in curb­ing high triglyc­erides, but will ge­net­ic dis­par­i­ties lim­it use?

When Regeneron scored an early approval for lipid lowering antibody Evkeeza back in February, the drugmaker cracked open a new pathway to lower abnormally high cholesterol levels. Now, Regeneron is chasing high triglycerides as well with some promising mid-stage data — but will genetic restrictions limit the drug’s use?

Regeneron’s Evkeeza (evinacumab) cut median triglyceride levels by more than 800 mg/dL (57%) in patients with a rare disorder causing abnormally high triglyceride levels compared with an overall increase of 50 mg/dL (1.8%) in participants on placebo, according to Phase II data presented Sunday at the virtual American College of Cardiology meeting.

$DNA is once again on NYSE; FDA clears Soliris chal­lenger for the mar­ket; Flag­ship’s think­ing big again with eR­NA; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

I still remember the uncertainty in the air last year when nobody was sure whether ASCO would cancel their in-person meeting. But it’s now back again for the second virtual conference, and Endpoints News is here for it. Check out our 2-day event reviewing the landscape of cancer R&D and send news our way.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 105,400+ biopharma pros reading Endpoints daily — and it's free.

As­traZeneca's Farx­i­ga missed big on Covid-19 study, but it's tak­ing SGLT2 safe­ty da­ta as a sil­ver lin­ing

AstraZeneca hasn’t seen many setbacks in recent months for SGLT2 inhibitor Farxiga, which broke ground in heart failure and kidney disease regardless of diabetes diagnosis. But the British drugmaker had to admit defeat in taking Farxiga into Covid-19, but follow-up results add a bit of a silver lining to that trial’s safety data.

Of hospitalized Covid-19 patients dosed with AstraZeneca’s Farxiga, 11.2% experienced an organ failure or died after 30 days of therapy compared with 13.8% of those given placebo, according to follow-up data from the DARE-19 study revealed Sunday at the virtual American College of Cardiology meeting.

Pfiz­er, Bris­tol My­er­s' Eliquis flops in post-heart surgery pa­tients, spurring an 'un­ex­plained sig­nal' in cer­tain deaths

Pfizer and Bristol Myers Squibb’s non-warfarin blood thinner Eliquis has raced out to become the most prescribed drug of its class on the market — even overtaking warfarin’s long-time lead. But in tricky-to-treat patients after a valve replacement, an investigator-sponsored study couldn’t turn up benefit and raised a troubling safety signal.

Eliquis failed to show benefit over standard of care in preventing serious clinical outcomes after a transaortic valve replacement (TAVR) and was linked to an “unexplained signal” in a subset of populations with a higher rate of non-CV deaths who did not need blood thinners apart from the surgery, according to data presented Saturday at the virtual American College of Cardiology meeting.

Vas Narasimhan (Photographer: Simon Dawson/Bloomberg via Getty Images)

No­var­tis whiffs on En­tresto study af­ter heart at­tacks — but that does­n't mean it's go­ing down qui­et­ly

If Novartis learned one thing from its interaction with the FDA over its latest heart failure approval for Entresto, it was that missing a primary endpoint may not be the nail in the coffin. Now, Entresto has missed again on a late-stage study in high-risk heart patients, and it’s already sowing the seeds for a path forward regardless.

Novartis’ Entresto couldn’t best standard-of-care ramipril in staving off a composite of deaths and heart failure events in patients with left ventricular systolic dysfunction and/or pulmonary congestion who have had a prior heart attack, according to topline data from the Phase III PARADISE-MI study revealed Saturday at the virtual American College of Cardiology meeting.

Michael Dell (Richard Drew, AP Images)

'Dude, you're get­ting a Del­l' — as a new deep-pock­et biotech in­vestor

What happens when you marry longtime insiders in the global biotech VC game with the family fund of tech billionaire Michael Dell, a synthetic biology legend out of MIT and Harvard and the former director of the NCI?

Today, the answer is a newly financed, $200 million biotech SPAC now cruising the industry for a top player interested in finding a short cut to Nasdaq.

Orion Biotech Opportunities priced their blank check company today, raising $200 million with Dell’s multibillion-dollar MSD group’s commitment on investing another $20 million in a forward-purchase agreement.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Gene ther­a­py from Bio­gen's $800M buy­out flops in mid-stage study, deal­ing blow to new am­bi­tions

The #2 candidate from Biogen’s $800 million ocular gene therapy buyout has failed in a mid-stage trial, dealing an early blow to the big biotech’s plans to revitalize its pipeline with new technologies.

Biogen announced that the candidate, an experimental treatment for a rare and progressive form of blindness called X-linked retinitis pigmentosa (XLRP), failed to sufficiently improve vision in patients’ treated eye — patients only received an injection in one eye — after a year, on a standard scale, compared to their untreated eye. The company said they saw “positive trends” on several secondary endpoints, including visual acuity, but declined to say whether the trial actually hit any of those endpoints.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 105,400+ biopharma pros reading Endpoints daily — and it's free.

In­cyte’s PD-(L)1 in­hibitor head­ed for an ODAC show­down next month

The FDA’s Oncologic Drugs Advisory Committee will spend a half day on June 24 reviewing Incyte’s PD-(L)1 inhibitor retifanlimab as a treatment for locally advanced or metastatic squamous carcinoma of the anal canal (SCAC) for those who have progressed on or who are intolerant of platinum-based chemotherapy.

The eighth PD-(L)1 entrant in January nabbed a priority review and an orphan designation from the FDA, which sets the agency’s final decision date as July 25.