Build­ing an or­phan pipeline, Hori­zon Phar­ma bags PhI­II-ready rare dis­ease drug in $145M buy­out

Tim­o­thy P. Wal­bert

Eight months af­ter a lit­tle-known sub­sidiary group named Riv­er Vi­sion De­vel­op­ment Corp. won a break­through ther­a­py des­ig­na­tion for its lead drug for a rare eye dis­ease, Hori­zon Phar­ma $HZNP has swooped in to pluck its sole drug as­set in a buy­out deal pow­ered with a $145 mil­lion up­front.

The com­pa­ny is a sub­sidiary of Nar­row Riv­er, a Rock­e­feller Cen­ter-based biotech which was set up by biotech vet­er­an David Mad­den, the chair­man at Dicer­na and a board mem­ber of Nav­i­tor. Mad­den got Riv­er Vi­sion start­ed with a $17 mil­lion round backed by GSK’s SR One along with Lund­beck­fond in 2012. Then he fol­lowed up with a li­cens­ing deal with Roche on tepro­tu­mum­ab (RV001), which had been part­nered with Gen­mab, which had dis­cov­ered the treat­ment.

Hori­zon is pay­ing for an an­ti­body tar­get­ing rare cas­es of thy­roid eye dis­ease, an au­toim­mune in­flam­ma­to­ry dis­or­der. In the Phase II tri­al the drug looked pos­i­tive in the in­tent-to-treat group: 29 of 42 pa­tients who re­ceived tepro­tu­mum­ab (69%), as com­pared with 9 of 45 pa­tients who re­ceived place­bo (20%), had a re­sponse at week 24.

Hori­zon is well known for han­dling some of the high­est cost meds in the in­dus­try, with two of the top 10 most ex­pen­sive drugs on the plan­et — both or­phan ther­a­pies. And they see this lat­est pur­chase as a key ex­am­ple of how they plan to build a pipeline of rare dis­ease treat­ments.

The deal comes with an un­spec­i­fied set of mile­stones.

Hori­zon’s in­vestors, though, were more fo­cused on the com­pa­ny’s dis­ap­point­ing Q1 per­for­mance this morn­ing, with a big cut to the com­pa­ny’s 2017 fore­cast, which drove down the val­ue of its shares by 22% in pre-mar­ket trad­ing.

Their new drug in­hibits in­sulin-like growth fac­tor type 1 re­cep­tor (IGF-1R), and Hori­zon be­lieves it has the po­ten­tial to go on to an or­phan ap­proval and more than $250 mil­lion in an­nu­al sales. The BTD ti­tle was won on hu­man da­ta that the com­pa­ny snagged in its TED01RV tri­al.

“This ac­qui­si­tion is an im­por­tant step in our strat­e­gy of pur­su­ing and ac­quir­ing de­vel­op­ment-stage med­i­cines tar­get­ing rare dis­eases,” said Tim­o­thy P. Wal­bert, chair­man, pres­i­dent and chief ex­ec­u­tive of­fi­cer, Hori­zon Phar­ma.  “With no ap­proved med­i­cines to treat Thy­roid Eye Dis­ease, there is a sig­nif­i­cant un­met treat­ment need among the ap­prox­i­mate­ly 10,000 pa­tients in the Unit­ed States with mod­er­ate to se­vere dis­ease and we look for­ward to be­gin­ning the piv­otal study with tepro­tu­mum­ab in the sec­ond half of this year.”

Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

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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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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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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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