Adamas wins its first FDA OK, set­ting up mar­ket launch for a re­for­mu­lat­ed Parkin­son’s drug

More than a year af­ter Adamas $ADMS post­ed pos­i­tive re­sults from a piv­otal Phase III study of ADS-5102 — an ex­tend­ed-re­lease ver­sion of the gener­ic Parkin­son’s treat­ment aman­ta­dine — the com­pa­ny got a green light from the FDA to sell the ther­a­py.

The drug will be sold as Go­cov­ri.

Gre­go­ry Went, CEO

Prob­a­bly not un­ex­pect­ed­ly, the drug was able to beat out a place­bo in sig­nif­i­cant­ly re­duc­ing the side ef­fects of Parkin­son’s. And the Emeryville, CA-based biotech boasts that this is the first and on­ly drug ap­proved for “dysk­i­ne­sia in pa­tients with Parkin­son’s dis­ease re­ceiv­ing lev­odopa-based ther­a­py” — an or­phan in­di­ca­tion. The ther­a­py is de­signed to help pa­tients who suf­fer from the sharp, in­vol­un­tary move­ment dis­or­der brought on by lev­odopa, com­mon­ly used to con­trol the dis­ease.

In­vestors em­braced the news, send­ing shares rock­et­ing up 57%.

The gener­ic drug, though, is al­ready used off la­bel for dysk­i­ne­sia, which may well in­ter­fere with the com­pa­ny’s sales ex­pec­ta­tions. Walid Gel­lad, an as­so­ciate pro­fes­sor of med­i­cine at the Uni­ver­si­ty of Pitts­burgh, high­light­ed the off la­bel use in a Tweet.

The ap­proval marks a ma­jor shift for Adamas, which has been lin­ing up job of­fers for 59 sales ex­ecs they be­lieve can reach the vast ma­jor­i­ty of the physi­cians and spe­cial­ists who han­dle the bulk of the cas­es. And the team there has been lay­ing the ground­work with pay­ers to help roll out the drug.

What Adamas doesn’t have right now is a price. “We an­tic­i­pate a list price for Go­cov­ri around the range of the pre­vi­ous dis­clo­sure: $10K – $30K,” the com­pa­ny told me in an email. The fi­nal price, they say, will be heav­i­ly in­flu­enced by pa­tient ac­cess. In the US, whole­sale prices are typ­i­cal­ly heav­i­ly dis­count­ed for ma­jor pay­ers.

The gener­ic drug is sold by on­line phar­ma­cies, of­ten for the flu or Parkin­son’s, for a lit­tle more than $2 per 100 mg cap­sule.

Adamas ran two Phase III stud­ies for the drug, demon­strat­ing that it trig­gered a 37% re­duc­tion in Uni­fied Dysk­i­ne­sia Rat­ing Scale (UDysRS) to­tal score vs. 12 per­cent for place­bo at week 12. The re­sults were con­firmed in the sec­ond study, with Go­cov­ri achiev­ing a 46% re­duc­tion in UDysRS vs. 16% for place­bo.

The drug launch starts in Jan­u­ary, ac­cord­ing to Adamas, which can now wrap up its pre-launch ef­forts.

“To­day’s ap­proval is a tremen­dous mile­stone for Adamas and for the Parkin­son’s dis­ease com­mu­ni­ty,” said Adamas CEO Gre­go­ry T. Went. “Go­cov­ri has the po­ten­tial to help peo­ple with Parkin­son’s dis­ease suf­fer­ing from dysk­i­ne­sia by fi­nal­ly pro­vid­ing physi­cians with an ef­fec­tive tool to ad­dress this long-stand­ing un­met med­ical need.”

John Hood [file photo]

UP­DATE: Cel­gene and the sci­en­tist who cham­pi­oned fe­dra­tinib's rise from Sanofi's R&D grave­yard win FDA OK

Six years after Sanofi gave it up for dead, the FDA has approved the myelofibrosis drug fedratinib, now owned by Celgene.

The drug will be sold as Inrebic, and will soon land in the portfolio at Bristol-Myers Squibb, which is finalizing a deal to acquire Celgene.

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Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

The top 10 fran­chise drugs in bio­phar­ma his­to­ry will earn a to­tal of $1.4T (tril­lion) by 2024 — what does that tell us?

Just in case you were looking for more evidence of just how important Amgen’s patent win on Enbrel is for the company and its investors, EvaluatePharma has come up with a forward-looking consensus estimate on what the list of top 10 drugs will look like in 2024.

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UP­DAT­ED: AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

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UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

The executive team at Alector $ALEC has a bone to pick with scientific co-founder Asa Abeliovich. Their latest quarterly rundown has this brief note buried inside:

On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

There’s no explicit word in the filing on what kind of confidential info was involved, but the proceeding got started 2 days ahead of Abeliovich’s IPO.

Abeliovich, formerly a tenured associate professor at Columbia, is a top scientist in the field of neurodegeneration, which is where Alector is targeted. More recently, he’s also helped start up Prevail Therapeutics as the CEO, which raised $125 million in an IPO. And there he’s planning on working on new gene therapies that target genetically defined subpopulations of Parkinson’s disease. Followup programs target Gaucher disease, frontotemporal dementia and synucleinopathies.

But this time Abeliovich is the CEO rather than a founding scientist. And some of their pipeline overlaps with Alector’s.

Abeliovich and Prevail, though, aren’t taking this one lying down.

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Chi­na has be­come a CEO-lev­el pri­or­i­ty for multi­na­tion­al phar­ma­ceu­ti­cal com­pa­nies: the trend and the im­pli­ca­tions

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.

ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

ICER has some strong words for PTC, Sarepta and the FDA as the US drug price watchdog concludes that as currently priced, their respective new treatments for Duchenne muscular dystrophy are decidedly not cost-effective.

The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.

The key dates for KRAS watch­ers through the end of the year — the trail is nar­row and risks are ex­treme

There’s nothing quite like a big patent win when it comes to burnishing your prospects in the pipeline. And for Amgen, which seems to have rescued Enbrel for a run to 2029, the cheering section on Wall Street is now fixed on AMG 510 and a key rival.

And it didn’t take much data to do it. 

There was the first snapshot of a handful of patients, with a 50% response rate. Then came word that Amgen researchers are also tracking responses in different cancers, at least one in colorectal cancer and appendiceal too. 

Bain's Or­ly Mis­han joins Pfiz­er's neu­ro spin­out Cerev­el; On­colyt­ic virus biotech taps Sil­la­Jen ex­ec He­le­na Chaye as CEO

→ Bain Capital is deploying one of its top investors to Cerevel Therapeutics, steering a $350 million-plus neuro play carved out of Pfizer. Orly Mishan — a co-founder and principal of Bain’s life sciences unit — was involved in the partnership that birthed the biotech spinout in the first place. As Cerevel’s first chief business officer, she is tasked with corporate development, program management as well as technical operations.