Al­ny­lam shoots for a speedy FDA OK af­ter their next shot at field­ing a pricey rare dis­ease drug hits PhI­II goal

Al­ny­lam $AL­NY is go­ing for it.

Af­ter rack­ing up a pos­i­tive set of da­ta for an in­ter­im re­view of their piv­otal study for givosir­an, ex­ecs at the RNAi pi­o­neer say they’ll hus­tle ahead with their sec­ond shot at a new drug ap­proval for a rare dis­ease, just weeks af­ter gain­ing a his­toric green light for their lead drug patisir­an.

Ak­shay Vaish­naw.

We’re not get­ting any hard da­ta in the state­ment out Thurs­day morn­ing, just con­fir­ma­tion that re­searchers hit their marks on sta­tis­ti­cal sig­nif­i­cance in an in­ter­im look at a key bio­mark­er. And that leaves them on track to gain a po­ten­tial ac­cel­er­at­ed ap­proval, es­pe­cial­ly as new man­age­ment at the FDA is quick­er than ever in push­ing out new drugs for rare dis­eases and can­cer ahead of fi­nal ef­fi­ca­cy and safe­ty da­ta.

We saw ear­ly-stage da­ta on this drug ear­li­er in the year, when Al­ny­lam tracked a 75% drop in the rate of acute he­pat­ic por­phyr­ia at­tacks, do­ing even bet­ter with a 90% drop in an ex­ten­sion study rel­a­tive to the run-in base­line rate ahead of dos­ing, en­cour­ag­ing them to see a ris­ing rate of ef­fi­ca­cy with longterm dos­ing. There was al­so a big drop in lev­els of aminole­vulin­ic acid, be­lieved to be the pri­ma­ry dis­ease trig­ger for the ul­tra-rare dis­ease.

Full top line re­sults are due in ear­ly 2019, with an ap­pli­ca­tion com­ing in ahead dur­ing Q4.

Al­ny­lam spent years in the clin­ic ad­vanc­ing lead­ing RNAi drugs through a se­ries of ups and downs. But now that it’s be­gun to roll out new drugs, the biotech has big plans to keep the drug ap­pli­ca­tions com­ing. These won’t be cheap, with ul­tra-rare con­di­tions of­ten sold for sub­stan­tial six-fig­ure sums.

“With these in­ter­im re­sults in hand, we plan to meet with the FDA to dis­cuss the re­sults and the over­all ben­e­fit-risk pro­file for a po­ten­tial NDA sub­mis­sion at or around year-end in sup­port of an Ac­cel­er­at­ed Ap­proval. In the mean­time, with en­roll­ment in EN­VI­SION com­plet­ed ahead of sched­ule, we look for­ward to re­port­ing topline re­sults for the full study ear­ly next year,” says Al­ny­lam R&D chief Ak­shay Vaish­naw. “If clin­i­cal ef­fi­ca­cy and ac­cept­able safe­ty are con­firmed in the full study, we be­lieve givosir­an has the po­ten­tial to trans­form the lives of pa­tients liv­ing with an AHP.”

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,600+ biopharma pros reading Endpoints daily — and it's free.

Top an­a­lyst finds a sil­ver lin­ing in Ab­b­Vie’s $63B Al­ler­gan buy­out — 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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,600+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,600+ biopharma pros reading Endpoints daily — and it's free.

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.