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.”

Alice Shaw, Lung Cancer Foundation of America

Top ALK ex­pert and can­cer drug re­searcher Al­ice Shaw bids adieu to acad­e­mia, hel­lo to No­var­tis

Jay Bradner has recruited a marquee oncology drug researcher into the ranks of the Novartis Institutes for BioMedical Research. Alice Shaw is jumping from prestigious posts intertwined through Mass General, Harvard and Dana-Farber to take the lead of NIBR’s translational clinical oncology group.

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Med­ical an­i­ma­tion: Mak­ing it eas­i­er for the site and the pa­tient to un­der­stand

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
Improved accessibility to visualisation
Visualisation is a core component of our ability to understand a concept. Ask 10 people to visualise an apple, and each will come up with a slightly different image, some apples smaller than others, some more round, some with bites taken. Acceptable, you say, we can move on to the next part of the story. Now ask 10 people to visualise how HIV’s capsid protein gets arranged into the hexamers and pentamers that form the viral capsid that holds HIV’s genetic material. This request may pose a challenge even to someone with some virology knowledge, and it is that inability to effectively visualise what is going on that holds us back from fully understanding the rest of the story. So how does medical animation help us to overcome this visualisation challenge?

Hal Barron, GSK's president of R&D and CSO, speaks to Endpoints News founder and editor John Carroll in London at Endpoints' #UKBIO19 summit on October 8, 2019

[Video] Cel­e­brat­ing tri­al fail­ures, chang­ing the cul­ture and al­ly­ing with Cal­i­for­nia dream­ers: R&D chief Hal Bar­ron talks about a new era at GSK

Last week I had a chance to sit down with Hal Barron at Endpoints’ #UKBIO19 summit to discuss his views on R&D at GSK, a topic that has been central to his life since he took the top research post close to 2 years ago. During the conversation, Barron talked about changing the culture at GSK, a move that involves several new approaches — one of which involves celebrating their setbacks as they shift resources to the most promising programs in the pipeline. Barron also discussed his new alliances in the Bay Area — including his collaboration pact with Lyell, which we covered here — frankly assesses the pluses and minuses of the UK drug development scene, and talks about his plans for making GSK a much more effective drug developer.

This is one discussion you won’t want to miss. Insider and Enterprise subscribers can log-in to watch the video.

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Flu Virus (Source: CDC)

FDA ex­pands Xofluza ap­proval as Roche strug­gles to catch loom­ing flu mar­ket

As a potentially powerful flu season looms, so does a big test for Roche and its new flu drug, Xofluza. The Swiss giant just got a small boost in advance of that test as the FDA expanded Xofluza’s indication to include patients at high risk of developing flu-related complications.

Xofluza (baloxavir marboxil) was approved last October in the US, the first landmark flu drug approval in 20 years and a much-needed green light for a company that had watched its leading flu drug Tamiflu get eaten alive by generics. Like its predecessor, the pill offered a reduction in flu symptoms but not a cure.

EMA backs sev­en ther­a­pies, in­clud­ing Mer­ck­'s Ebo­la vac­cine

The first-ever Ebola vaccine is on the precipice of approval after the European Medicine’s Agency (EMA) backed the Merck product in this week’s roster of recommendations.

The drugmaker $MRK began developing the vaccine, christened Ervebo, during the West African outbreak that occurred between 2014 and 2016, killing more than 11,000.

The current outbreak in the Democratic Republic of Congo (DRC) has shown case fatality rates of approximately 67%, the agency estimated. Earlier this year, the WHO declared the outbreak — which so far has infected more than 3,000 people — a public health emergency of international concern.

Ronald Herb­st fol­lows Med­Im­mune ex­o­dus to Pyx­is CSO post; Jeff God­dard to suc­ceed CEO of AIT Bio­science

→ The outflow of top execs from MedImmune continues to fill the leadership ranks of smaller biotechs. The latest to take off is Ronald Herbst, the head of oncology research, who’s assuming the CSO post at Pyxis Oncology.  

Herbst was part of the old MedImmune organization AstraZeneca CEO Pascal Soriot restructured earlier this year, reorganizing the company and eliminating the storied subsidiary as a separate organization.

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Mer­ck is tak­ing the ax to its US op­er­a­tions, cut­ting 500 jobs in its lat­est re­or­ga­ni­za­tion

Merck is cutting 500 jobs in its US sales and headquarters commercial teams in its latest effort to find new ways to streamline the operation.

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UP­DAT­ED: J&J's Xarel­to, Amar­in's Vas­cepa are cost-ef­fec­tive, not bud­get friend­ly — ICER

ICER, an increasingly influential cost-effectiveness watchdog in the United States, has concluded in its review of treatments for cardiovascular disease that while the cost of J&J’s Xarelto and Amarin’s Vascepa meet its benchmark for value pricing — the two treatments will not likely treat as many patients as hoped without surpassing the annual budget threshold calculated by ICER for each therapy.

Mi­rati preps its first look at their KRAS G12C con­tender, and they have to clear a high bar for suc­cess

If you’re a big KRAS G12C fan, mark your calendars for October 28 at 4:20 pm EDT.

That’s when Mirati $MRTX will unveil its first peek at the early clinical data available on MRTX849 in presentations at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston.

Mirati has been experiencing the full effect of a rival’s initial success at targeting the G12C pocket found on KRAS, offering the biotech some support on the concept they’re after — and biotech fans a race to the top. Amgen made a big splash with its first positive snapshot on lung cancer, but deflated sky-high expectations as it proved harder to find similar benefits in other types of cancers.

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