Am­i­cus scraps a top rare dis­ease drug af­ter it flunks a piv­otal test — and shares spike

Just two months af­ter Am­i­cus Ther­a­peu­tics $FOLD got a rare re­prieve from the FDA, clear­ing the path for a new mar­ket­ing ap­pli­ca­tion for its lead drug mi­gala­s­tat, the biotech has been ham­mered by a se­vere set­back on its num­ber two drug de­vel­op­ment ef­fort. The com­pa­ny re­port­ed this morn­ing that SD-101 failed a Phase III study, miss­ing the co-pri­ma­ry and key sec­ondary end­points in treat­ing epi­der­mol­y­sis bul­losa.

John Crow­ley, Am­i­cus

There was no ev­i­dence of sta­tis­ti­cal su­pe­ri­or­i­ty in wound clos­ing — with the drug ac­tu­al­ly un­der­per­form­ing the place­bo on the key mea­sures — and Am­i­cus ex­ecs say they will scrap any oth­er de­vel­op­ment ef­forts, though pa­tients in the piv­otal can re­main on ther­a­py if they choose.

Am­i­cus shares dropped 14% soon af­ter the news hit, but then quick­ly — and re­mark­ably — turned back north in­to the green as in­vestors and an­a­lysts ral­lied be­hind the com­pa­ny.

This rare and dev­as­tat­ing ge­net­ic dis­ease is char­ac­ter­ized by pa­pery thin skin sub­ject to se­vere blis­ter­ing and tear­ing. Most pa­tients —and there are some 30,000 to 40,000 in the world’s ma­jor drug mar­kets — die be­fore the age of 30.  There are no ther­a­pies ap­proved for the ail­ment.

Am­i­cus CEO John Crow­ley arranged to buy the drug in an $847 mil­lion deal — $229 mil­lion up front — for Scio­derm back in 2015. At the time, he told me he felt cer­tain that the ther­a­py was on track to be­come a block­buster.

An­a­lysts, though, weren’t near­ly as en­thu­si­as­tic, which helps ex­plain the mut­ed in­vestor re­sponse. Leerink’s Joseph Schwartz put it this way:

We be­lieve the neg­a­tive news will cause nom­i­nal weak­ness of a dol­lar or so for the stock to­day, but based on al­ready cau­tious in­vestor sen­ti­ment for the EB pro­gram, we be­lieve this blip could be short-lived as in­vestors an­tic­i­pate ad­di­tion­al pos­i­tive Ph.1/2 Pompe da­ta, which is al­so ex­pect­ed in 3Q.

Crow­ley can still look to a re­newed ef­fort to gain an ap­proval for mi­gala­s­tat, which reg­u­la­tors at the FDA ini­tial­ly stopped in its tracks with a de­mand for a new study. That was be­fore Scott Got­tlieb took over as com­mis­sion­er, and Pres­i­dent Don­ald Trump voiced a full-throat­ed call for faster ac­tion at the agency.

Crow­ley said:

In seek­ing to de­vel­op nov­el, high qual­i­ty ther­a­pies for those liv­ing with dev­as­tat­ing rare dis­eases we may some­times fail. But we would rather be the first to fail, than the last to try. Our vi­sion at Am­i­cus re­mains stead­fast and is fo­cused on build­ing a lead­ing glob­al biotech­nol­o­gy com­pa­ny that de­liv­ers sig­nif­i­cant ben­e­fits for peo­ple liv­ing with rare dis­eases.

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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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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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.

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 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: 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.

As dis­as­ter struck, Ab­b­Vie’s Rick Gon­za­lez swooped in on Al­ler­gan with an of­fer Brent Saun­ders couldn’t say no to

Early March was a no good, awful, terrible time for Allergan CEO Brent Saunders. His big lead drug had imploded in a Phase III disaster and activists were after his hide — or at least his chairman’s title — as the stock price continued a steady droop that had eviscerated share value for investors.

But it was a perfect time for AbbVie CEO Rick Gonzalez to pick up the phone and ask Saunders if he’d like to consider a “strategic” deal.

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CEO Pascal Soriot via Getty Images

As­traZeneca's jug­ger­naut PARP play­er Lyn­parza scoops up an­oth­er dom­i­nant win in PhI­II as the FDA adds a 'break­through' for Calquence

AstraZeneca’s oncology R&D group under José Baselga keeps churning out hits.

Wednesday morning the pharma giant and their partners at Merck parted the curtains on a successful readout for their Phase III PAOLA-1 study, demonstrating statistically significant improvement in progression-free survival for women with ovarian cancer in a first-line maintenance setting who added their PARP Lynparza to Avastin. This is their second late-stage success in ovarian cancer, which will help stave off rivals like GSK.

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