Shire’s multi­bil­lion-dol­lar gam­ble on lanadelum­ab pays off with stel­lar PhI­II da­ta, quick mar­ket­ing ap­pli­ca­tion

Flem­ming Orn­skov

When Shire bought out Dyax for up to $6.5 bil­lion in 2015, the big prize was a new drug for hered­i­tary an­gioede­ma dubbed DX-2930 which posed a di­rect threat to Cin­ryze, a fran­chise drug for the rare dis­ease play­er.

Thurs­day morn­ing, Shire CEO Flem­ming Orn­skov says their mul­ti-bil­lion dol­lar bet paid off with stel­lar Phase III da­ta for the drug, now called lanadelum­ab (SHP643).

A 300 mg dose of the drug twice a month de­liv­ered an 87% re­duc­tion in mean HAE at­tack fre­quen­cy, com­pared to 0% in the place­bo group. It hit the pri­ma­ry end­point with “high­ly” sta­tis­ti­cal­ly sig­nif­i­cant re­sults as well as all the sec­on­daries.

Al­ready des­ig­nat­ed a break­through ther­a­py with or­phan drug sta­tus, Shire says it will now race to the FDA lat­er this year or ear­ly next with a bi­o­log­ics ap­pli­ca­tion, with plans to cap­i­tal­ize on the re­sults as soon as pos­si­ble.

Shares of the $55 bil­lion biotech $SH­PG surged 6% on the news in pre-mar­ket trad­ing.

Many an­a­lysts be­lieve that Shire, which swelled in size af­ter it com­plet­ed the Bax­al­ta and Dyax ac­qui­si­tions, is on a short path to block­buster rev­enue. Shire it­self set a goal of $2 bil­lion in peak sales for this drug. And Eval­u­atePhar­ma re­cent­ly picked it as a top-10 or­phan drug bound for a boom­ing mar­ket with 6-fig­ure price tags.

“The pos­si­bil­i­ty of a new way to ad­dress the un­der­ly­ing cause of HAE to pre­vent at­tacks could trans­form how we treat the dis­ease in the fu­ture,” said Pro­fes­sor Mar­cus Mau­r­er, M.D., Char­ité –Uni­ver­sitätsmedi­zin Berlin, Ger­many and clin­i­cal tri­al in­ves­ti­ga­tor. “Pa­tients with HAE want to live in­de­pen­dent­ly and with­out fear of an an­gioede­ma at­tack.”

Shire paid close to $6 bil­lion for Dyax, large­ly on the promise of the next-gen HAE ther­a­py. And Orn­skov added a $4 CVR worth $656 mil­lion if the drug is ap­proved. That pay­off could be loom­ing in 2018 if the da­ta pass­es muster with reg­u­la­tors.

The Phase III re­sults mark an­oth­er big win for CEO Flem­ming Orn­skov, who was de­ter­mined to trans­form the com­pa­ny, based in Lex­ing­ton, MA., af­ter he took over. Shire went on to ex­pe­ri­ence some bumps along the road, but in the longterm his strate­gic plan and will­ing­ness to bet big on new drugs has been pay­ing off.

“We are ex­treme­ly en­cour­aged by these topline Phase III re­sults,” said Orn­skov in a state­ment, “We have near­ly a decade of ex­pe­ri­ence and a strong port­fo­lio and pipeline in HAE and be­lieve these da­ta demon­strate high po­ten­tial for trans­form­ing the way pa­tients liv­ing with this con­di­tion are treat­ed.”

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.