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.

Brent Saunders [Getty Photos]

UP­DAT­ED: Ab­b­Vie seals $63B deal to buy a trou­bled Al­ler­gan — spelling out $1B in R&D cuts

Brent Saunders has found his way out of the current fix he’s in at Allergan $AGN. He’s selling the company to AbbVie for $63 billion in the latest example of the hot M&A market in biopharma.

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Top an­a­lyst of­fers a rare, up­beat in­ter­pre­ta­tion of Ab­b­Vie’s $63B Al­ler­gan deal — but there’s a catch

Af­ter get­ting beat up on all sides from mar­ket ob­servers who don’t much care for the lat­est mega-deal to ar­rive in bio­phar­ma, at least one promi­nent an­a­lyst now is start­ing to like what he sees in the num­bers for Ab­b­Vie/Al­ler­gan.

But it’s go­ing to take some en­cour­age­ment if Ab­b­Vie ex­ecs want it to last.

Ab­b­Vie’s mar­ket cap de­clined $20 bil­lion on Tues­day as the stock took at 17% hit dur­ing the day. And SVB Leerink’s Ge­of­frey Porges can see a dis­tinct out­line of an up­side af­ter re­view­ing the fun­da­men­tals of the deal.

While Ako­rn works to re­vive its for­tunes, the FDA hits it with an­oth­er warn­ing let­ter

Ako­rn just can’t dig it­self out of its hole.

The spe­cial­ty gener­ic drug­mak­er has re­ceived yet an­oth­er warn­ing let­ter from the FDA this year. With­out dis­clos­ing any specifics, the Lake For­est, Illi­nois-based drug­mak­er on Wednes­day said the US reg­u­la­tor had is­sued the let­ter, cit­ing an in­spec­tion of its Som­er­set, New Jer­sey man­u­fac­tur­ing fa­cil­i­ty in Ju­ly and Au­gust of 2018. The com­pa­ny’s shares $AKRX dipped about 1.7% to $4.65 be­fore the bell.

Af­ter rais­ing $158M, this up­start's founders have star back­ers and plans to break new ground in gene ther­a­py

Back in 2014, Stephanie Tagliatela opted to take an early exit out of her PhD program after working in Mark Bear’s lab at MIT, where she specialized in the synaptic connections between neuronal cells in the brain. She never finished that PhD, but she and fellow MIT student Kartik Ramamoorthi — who was on the founding team at Voyager — came away with some ideas for a gene therapy startup.

Today, fully 5 years later, she and Ramamoorthi are taking the wraps off of a $104 million mega-round designed to take the cumulative work of their preclinical formative stage for Encoded Therapeutics into human studies. They’ve now raised $158 million since starting out in Illumina’s incubator in the Bay Area, and they believe they are firmly on track to do something unique in gene therapy.

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FDA re­jects Ac­er's rare dis­ease drug, asks for new tri­al — shares crater

Ac­er Ther­a­peu­tics’ bid to re­pur­pose celipro­lol — a be­ta-block­er on the mar­ket for hy­per­ten­sion — as a treat­ment for a rare, in­her­it­ed con­nec­tive tis­sue dis­or­der has hit a se­vere set­back. The New­ton, Mass­a­chu­setts-based com­pa­ny on Tues­day said the FDA re­ject­ed the drug and has asked for an­oth­er clin­i­cal tri­al.

The com­pa­ny’s shares $AC­ER cratered near­ly 77% to $4.47 in Tues­day morn­ing trad­ing.

Richard Gonzalez testifying in front of Senate Finance Committee, February 2019 [AP Images]

Ab­b­Vie's $63B buy­out spot­lights the re­turn of ma­jor M&A deals — de­spite the back­lash

Big time M&A is back. But for how long?

Over the past 18 months we’ve now seen three major buyouts announced: Takeda/Shire; Bristol-Myers/Celgene and now AbbVie/Allergan. And with this latest deal it’s increasingly clear that the sharp fall from grace suffered by high-profile players which have seen their share prices blasted has created an opening for the growth players in big pharma to up their game — in sharp contrast to the popular bolt-on deals that have been driving the growth strategy at Novartis, Merck, Roche and others.

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Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

Suf­fer­ing No­var­tis part­ner Cona­tus grabs the ax and packs it in on NASH af­ter a se­ries of set­backs

The NASH par­ty is over at No­var­tis-backed Cona­tus. And this time they’re turn­ing off the lights.

More than 2 years af­ter No­var­tis sur­prised the biotech in­vest­ment com­mu­ni­ty with its $50 mil­lion up­front and promise of R&D sup­port to part­ner with the lit­tle biotech on NASH — ig­nit­ing a light­ning strike for the share price — Cona­tus $CNAT is back with the lat­est bit­ter tale to tell about em­ri­c­as­an, which once in­spired con­fi­dence at the phar­ma gi­ant.

Bet­ter than Am­bi­en? Min­er­va soars on PhI­Ib up­date on sel­torex­ant for in­som­nia

A month af­ter roil­ing in­vestors with what skep­tics dis­missed as cher­ry pick­ing of its de­pres­sion da­ta, Min­er­va is back with a clean slate of da­ta from its Phase IIb in­som­nia tri­al.

In a de­tailed up­date, the Waltham, MA-based biotech said sel­torex­ant (MIN-202) hit both the pri­ma­ry and sev­er­al sec­ondary end­points, ef­fec­tive­ly im­prov­ing sleep in­duc­tion and pro­long­ing sleep du­ra­tion. In­ves­ti­ga­tors made a point to note that the ef­fects were con­sis­tent across the adult and el­der­ly pop­u­la­tions, with the lat­ter more prone to the sleep dis­or­der.