Trendy Mod­er­na's sin­gle-as­set CF pact with Ver­tex marks a new phase of deal­mak­ing

Just a few days af­ter Mod­er­na wrapped up a $200 mil­lion-up­front re­search deal with Mer­ck, the Boston biotech is back with an­oth­er pact – this time ty­ing up with crosstown biotech Ver­tex $VRTX on a cys­tic fi­bro­sis de­vel­op­ment agree­ment.

Ver­tex is start­ing out with what amounts to a rel­a­tive­ly small up­front for Mod­er­na, which has struck the moth­er lode of deals with its promise – as yet un­proven in any hu­man tri­als – that your cells can be made in­to drug pro­duc­tion units. Mod­er­na gets $40 mil­lion up­front with half of that com­ing as a con­vert­ible note in­vest­ment. And there’s an­oth­er $275 mil­lion up for grabs in the form of mile­stone pay­ments.

Stéphane Ban­cel, Mod­er­na CEO

The deal marks a dis­tinct shift in Mod­er­na’s deal-mak­ing strat­e­gy. This is a one-as­set play, Mod­er­na CEO Stéphane Ban­cel tells me via a di­rect mes­sage, not like the big port­fo­lio deals the com­pa­ny has done with the likes of As­traZeneca ($240 mil­lion up­front for 40 prod­ucts), Alex­ion ($125 mil­lion for 10) and Mer­ck. And he says that Mod­er­na is in the process of strik­ing more of these sin­gle-as­set plays, where these num­bers work fine.

In the Ver­tex deal, Mod­er­na will use its mR­NA tech over the next three years to see if it can get lung cells to pro­duce func­tion­al copies of the cys­tic fi­bro­sis trans­mem­brane con­duc­tance reg­u­la­tor (CFTR) pro­tein, which is de­fec­tive among CF pa­tients. The part­ners will al­so ex­plore pul­monary mR­NA de­liv­ery.

Mod­er­na is in charge of dis­cov­ery, with Ver­tex han­dling every­thing pre­clin­i­cal and on. Ver­tex will al­so cov­er the ex­pens­es re­lat­ed to the work.

For Ver­tex, it’s a chance to go deep­er in­to cys­tic fi­bro­sis af­ter win­ning a land­mark ap­proval for Orkam­bi, a $259,000 drug. For Mod­er­na, it’s the lat­est in a long se­ries of col­lab­o­ra­tions that has put the com­pa­ny in the lab with a who’s who in drug de­vel­op­ment.

This isn’t the on­ly ef­fort in this field, though. Two years ago Shire aligned with the Cys­tic Fi­bro­sis Foun­da­tion to de­vel­op a new CFTR treat­ment for cys­tic fi­bro­sis.

“This col­lab­o­ra­tion is il­lus­tra­tive of our con­sis­tent strat­e­gy to part­ner with world class ther­a­peu­tic ex­perts in or­der to max­i­mize the po­ten­tial of lever­ag­ing mR­NA as a nov­el ther­a­peu­tic ap­proach across a spec­trum of se­ri­ous dis­eases,” said Ban­cel in a state­ment. “We’re par­tic­u­lar­ly ex­cit­ed to ex­plore the po­ten­tial of pul­monary de­liv­ery as a new modal­i­ty to bring trans­for­ma­tive mR­NA med­i­cines to pa­tients and ad­dress un­met needs.”

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 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.

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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.