Neu­ro­crine breaks out proof-of-con­cept da­ta for CAH drug; Ake­bia, MT­PC work to­ward Japan­ese NDA af­ter ane­mia drug scores in twin PhI­I­Is

→ More than a month af­ter its big tick­et deal with Voy­ager Ther­a­peu­tics $VY­GR, Neu­ro­crine Bio­sciences $NBIX an­nounced pos­i­tive in­ter­im da­ta from a Phase II proof-of-con­cept study eval­u­at­ing its ex­per­i­men­tal drug, NBI-74788, in adults with a ge­net­ic dis­or­der that re­sults in an en­zyme de­fi­cien­cy that al­ters the pro­duc­tion of adren­al steroids called clas­sic con­gen­i­tal adren­al hy­per­pla­sia (CAH). Da­ta showed a re­duc­tion of at least 50% from base­line in 17-hy­drox­yprog­es­terone (17-OHP) — used as a di­ag­nos­tic bio­mark­er in CAH — in more than half the treat­ed pa­tients. “But what does a 50% ‘re­spon­der’ rate mean in clin­i­cal terms? This study wasn’t re­al­ly de­signed to an­swer this ques­tion, and while we don’t know the pa­tients’ 17-OHP base­line val­ues in this study, our guess from look­ing at oth­er CAH tri­als (ahead of the da­ta to­day) is that it was prob­a­bly very high. Thus, it is un­clear to us to­day how these bio­mark­er changes will or will not trans­late in­to an im­prove­ment in pa­tient out­comes,” Stifel’s Paul Mat­teis wrote.

Ake­bia, which merged with Au­ryx­ia mak­er Keryx last year, put out pos­i­tive late-stage da­ta from twin Phase III Japan­ese stud­ies eval­u­at­ing its lead ex­per­i­men­tal drug vadadu­s­tat as an ane­mia treat­ment for CKD pa­tients. Part­ner Mit­subishi Tan­abe Phar­ma Corp (MT­PC) ex­pects to sub­mit a Japan­ese mar­ket­ing ap­pli­ca­tion in 2019. Ake­bia $AK­BA is in a fierce race with Fi­bro­Gen $FGEN in both Japan and the Unit­ed States to get their re­spec­tive ane­mia drugs on the mar­ket. Fi­bro­Gen and its part­ner Astel­las re­port­ed pos­i­tive 4th Phase III study of their ri­val rox­adu­s­tat in Japan in 2018.

→ UK’s Ox­ford Bio­med­ica is join­ing forces with tech ma­jor Mi­crosoft in a re­search and de­vel­op­ment col­lab­o­ra­tion to de­vel­op next-gen gene ther­a­py vec­tors us­ing the cloud and ma­chine learn­ing.

Lipocine pro­vid­ed up­dat­ed da­ta from its “liv­er fat” study eval­u­at­ing its ex­per­i­men­tal oral drug — LPCN 1144 — which is be­ing de­vel­oped for NASH. In Jan­u­ary, the com­pa­ny said the drug was be­ing eval­u­at­ing in 36 hy­pog­o­nadal males in a 16-week study. Sev­en of the nine sub­jects had at least 10% base­line liv­er fat, which the com­pa­ny said is in­dica­tive of sub­jects with NAFLD with the po­ten­tial to have NASH. Base­line mean liv­er fat of these sev­en sub­jects was 21.0%, and af­ter treat­ment there was an av­er­age ab­solute mean re­duc­tion from base­line of 7.6% in liv­er fat af­ter 8 weeks of treat­ment. On Tues­day, Lipocine said 62% of the of the eval­u­at­ed sub­jects had NAFLD, de­fined as base­line liv­er fat of at least 5%. At the end, 48% of the treat­ed NAFLD sub­jects had NAFLD res­o­lu­tion, de­fined as liv­er fat <5% post treat­ment for 16 weeks. The com­pa­ny’s shares $LPCN were up 7.3% in morn­ing trad­ing.

On a glob­al romp, Boehringer BD team picks up its third R&D al­liance for Ju­ly — this time fo­cused on IPF with $50M up­front

Boehringer Ingelheim’s BD team is on a global deal spree. The German pharma company just wrapped its third deal in 3 weeks, going back to Korea for its latest pipeline pact — this time focused on idiopathic pulmonary fibrosis.

They’re handing over $50 million to get their hands on BBT-877, an ATX inhibitor from Korea’s Bridge Biotherapeutics that was on display at a science conference in Dallas recently. There’s not a whole lot of data to evaluate the prospects here.

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Part club, part guide, part land­lord: Arie Bellde­grun is blue­print­ing a string of be­spoke biotech com­plex­es in glob­al boom­towns — start­ing with Boston

The biotech industry is getting a landlord, unlike anything it’s ever known before.

Inspired by his recent experiences scrounging for space in Boston and the Bay Area, master biotech builder, investor, and global dealmaker Arie Belldegrun has organized a new venture to build a new, 250,000 square foot biopharma building in Boston’s Seaport district — home to Vertex and a number of up-and-coming biotech players.

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Servi­er scoots out of an­oth­er col­lab­o­ra­tion with Macro­Gen­ics, writ­ing off their $40M

Servier is walking out on a partnership with MacroGenics $MGNX — for the second time.

After the market closed on Wednesday MacroGenics put out word that Servier is severing a deal — inked close to 7 years ago — to collaborate on the development of flotetuzumab and other Dual-Affinity Re-Targeting (DART) drugs in its pipeline.

MacroGenics CEO Scott Koenig shrugged off the departure of Servier, which paid $20 million to kick off the alliance and $20 million to option flotetuzumab — putting a heavily back-ended $1 billion-plus in additional biobuck money on the table for the anti-CD123/CD3 bispecific and its companion therapies.

Den­mark's Gen­mab hits the jack­pot with $500M+ US IPO as small­er biotechs rake in a com­bined $147M

Danish drugmaker Genmab A/S is off to the races with perhaps one of the biggest biotech public listings in decades, having reaped over $500 million on the Nasdaq, as it positions itself as a bonafide player in antibody-based cancer therapies.

The company, which has long served as J&J’s $JNJ key partner on the blockbuster multiple myeloma therapy Darzalex, has asserted it has been looking to launch its own proprietary product — one it owns at least half of — by 2025.

FDA over­rides ad­comm opin­ions a fifth of the time, study finds — but why?

For drugmakers, FDA advisory panels are often an apprehended barometer of regulators’ final decisions. While the experts’ endorsement or criticism often translate directly to final outcomes, the FDA sometimes stun observers by diverging from recommendations.

A new paper out of Milbank Quarterly put a number on that trend by analyzing 376 voting meetings and subsequent actions from 2008 through 2015, confirming the general impression that regulators tend to agree with the adcomms most of the time — with discordances in only 22% of the cases.

Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

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Novotech CRO Ex­pands Chi­na Team as Biotech De­mand for Clin­i­cal Tri­als In­creas­es up to 79%

An increase in demand of up to 79% for clinical trials in China has prompted Novotech the Asia-Pacific CRO to rapidly expand the China team, appointing expert local clinical executives to their Shanghai and Hong Kong offices. The company is planning to expand their team by 30% over the next quarter.

Novotech China has seen considerable demand recently which is borne out by research from GlobalData:
A global migration of clinical research is occurring from high-income countries to low and middle-income countries with emerging economies. Over the period 2017 to 2018, for example, the number of clinical trial sites opened by biotech companies in Asia-Pacific increased by 35% compared to 8% in the rest of the world, with growth as high as 79% in China.
Novotech CEO Dr John Moller said China offers the largest population in the world, rapid economic growth, and an increasing willingness by government to invest in research and development.
Novotech’s 23 years of experience working in the region means we are the ideal CRO partner for USA biotechs wanting to tap the research expertise and opportunities that China offers.
There are over 22,000 active investigators in Greater China, with about 5,000 investigators with experience on at least 3 studies (source GlobalData).

UP­DAT­ED: With loom­ing ‘apoc­a­lypse of drug re­sis­tance,’ Mer­ck’s com­bi­na­tion an­tibi­ot­ic scores FDA ap­proval on two fronts

Merck — one of the last large biopharmaceuticals companies in the beleaguered field of antibiotic drug development — on Wednesday said the FDA had sanctioned the approval of its combination antibacterial for the treatment of complicated urinary tract and intra-abdominal infections.

To curb the rise of drug-resistant bacteria and maintain the efficacy of the therapy, Recarbrio (and other antibacterials) — the drug must be used to treat or prevent infections that are proven or strongly suspected to be caused by susceptible gram-negative bacteria, Merck $MRK said.

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John McHutchison in 2012. Getty Images

The $1.1M good­bye: Gilead CSO John McHutchi­son is out as Daniel O’Day shakes up the se­nior team

Just a little more than a year after John McHutchison grabbed a promotion to become CSO at Gilead in the wake of Norbert Bischofberger’s exit, he’s out amid a shakeup of the senior team that is also triggering the departure of two other top execs.

Gilead stated that McHutchison “has decided to step down” from the job as of August 2nd. And their SEC filing notes that he’ll be getting a $1.1 million check to settle up on his contract.

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