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.”

BY­OD Best Prac­tices: How Mo­bile De­vice Strat­e­gy Leads to More Pa­tient-Cen­tric Clin­i­cal Tri­als

Some of the most time- and cost-consuming components of clinical research center on gathering, analyzing, and reporting data. To improve efficiency, many clinical trial sponsors have shifted to electronic clinical outcome assessments (eCOA), including electronic patient-reported outcome (ePRO) tools.

In most cases, patients enter data using apps installed on provisioned devices. At a time when 81% of Americans own a smartphone, why not use the device they rely on every day?

Chris Gibson (Photo By Vaughn Ridley/Sportsfile for Web Summit via Getty Images)

Re­cur­sion founders gin for­tunes as IPO back­ers show­er $436M on one of the biggest boasts in AI -- based on some very small deals

In the AI drug development world, boasting often comes with the territory. Yet few can rival Recursion when it comes to claiming the lead role in what company execs like to call the industrialization of drug development, with promises of continued exponential growth in the number of drugs it has in the pipeline.

On Friday, the Salt Lake City-based biotech translated its unicorn-sized boasts into a killer IPO, pricing more than 24 million shares at the high end of its range and bringing in $436 million — with a large chunk of that promised by some deep-pocket backers.

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Covid-19 vac­cine halt drags on, an FDA ap­point­ment at long last, the great CRO con­sol­i­da­tion, and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Conference season is upon us, and while we’d much prefer to be wandering down the hallways and presentation rooms in person, the team is ready to cover the most consequential data coming out of these scientific meetings. Get in touch early if you have news to share.

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Eli Lil­ly asks FDA to re­voke EUA for Covid-19 treat­ment

Eli Lilly on Friday requested that the FDA revoke the emergency authorization for its Covid-19 drug bamlanivimab, which is no longer as effective as a combo therapy because of a rise in coronavirus variants across the US.

“With the growing prevalence of variants in the U.S. that bamlanivimab alone may not fully neutralize, and with sufficient supply of etesevimab, we believe now is the right time to complete our planned transition and focus on the administration of these two neutralizing antibodies together,” Daniel Skovronsky, Lilly’s CSO, said in a statement.

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Ex­clu­sive in­ter­view: Pe­ter Marks on why full Covid-19 vac­cine ap­provals could be just months away

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, took time out of his busy schedule last Friday to discuss with Endpoints News all things related to his work regulating vaccines and the pandemic.

Marks, who quietly coined the name “Operation Warp Speed” before deciding to stick with his work regulating vaccines at the FDA rather than join the Trump-era program, has been the face of vaccine regulation for the FDA throughout the pandemic, and is usually spotted in Zoom meetings seated in front of his wife’s paintings.

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Near­ly a year af­ter Au­den­tes' gene ther­a­py deaths, the tri­al con­tin­ues. What hap­pened re­mains a mys­tery

Natalie Holles was five months into her tenure as Audentes CEO and working to smooth out a $3 billion merger when the world crashed in.

Holles and her team received word on the morning of May 5 that, hours before, a patient died in a trial for their lead gene therapy. They went into triage mode, alerting the FDA, calling trial investigators to begin to understand what happened, and, the next day, writing a letter to alert the patient community so they would be the first to know. “We wanted to be as forthright and transparent as possible,” Holles told me late last month.

The brief letter noted two other patients also suffered severe reactions after receiving a high dose of the therapy and were undergoing treatment. One died a month and a half later, at which point news of the deaths became public, jolting an emergent gene therapy field and raising questions about the safety of the high doses Audentes and others were now using. The third patient died in August.

“It was deeply saddening,” Holles said. “But I was — we were — resolute and determined to understand what happened and learn from it and get back on track.”

Eleven months have now passed since the first death and the therapy, a potential cure for a rare and fatal muscle-wasting disease called X-linked myotubular myopathy, is back on track, the FDA having cleared the company to resume dosing at a lower level. Audentes itself is no more; last month, Japanese pharma giant Astellas announced it had completed working out the kinks of the $3 billion merger and had restructured and rebranded the subsidiary as Astellas Gene Therapies. Holles, having successfully steered both efforts, departed.

Still, questions about precisely what led to the deaths of the 3 boys still linger. Trial investigators released key details about the case last August and December, pointing to a biological landmine that Audentes could not have seen coming — a moment of profound medical misfortune. In an emerging field that’s promised cures for devastating diseases but also seen its share of safety setbacks, the cases provided a cautionary tale.

Audentes “contributed in a positive way by giving a painful but important example for others to look at and learn from,” Terry Flotte, dean of the UMass School of Medicine and editor of the journal Human Gene Therapy, told me. “I can’t see anything they did wrong.”

Yet some researchers say they’re still waiting on Astellas to release more data. The company has yet to publish a full paper detailing what happened, nor have they indicated that they will. In the meantime, it remains unclear what triggered the events and how to prevent them in the future.

“Since Audentes was the first one and we don’t have additional information, we’re kind of in a holding pattern, flying around, waiting to figure out how to land our vehicles,” said Jude Samulski, professor of pharmacology at UNC’s Gene Therapy Center and CSO of the gene therapy biotech AskBio, now a subsidiary of Bayer.

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As­traZeneca-Alex­ion merg­er slides through FTC re­view af­ter sup­posed M&A crack­down pos­es no bar­ri­ers

The AstraZeneca-Alexion megamerger received a good sign Friday, despite warning signs of the tides turning against large M&A pharma deals.

US regulators at the FTC have cleared the acquisition for approval, AstraZeneca announced, all but signing off on the deal to go through once it officially closes in the third quarter. AstraZeneca originally said it was planning to buy out Alexion back in December for $39 billion.

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J&J faces CDC ad­vi­so­ry com­mit­tee again next week to weigh Covid-19 vac­cine risks

The CDC’s Advisory Committee on Immunization Practices punted earlier this week on deciding whether or not to recommend lifting a pause on the administration of J&J’s Covid-19 vaccine, but the committee will meet again in an emergency session next Friday to discuss the safety issues further.

The timing of the meeting likely means that the J&J vaccine will not return to the US market before the end of next week as the FDA looks to work hand-in-hand with the CDC to ensure the benefits of the vaccine still outweigh the risks for all age groups.

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David Stack, Pacira Biosciences CEO

In high­ly un­usu­al move, Paci­ra sues med­ical jour­nal for li­bel over its non-opi­oid painkiller

A New Jersey biotech whose only approved drug is used as a painkiller after surgeries is suing a scientific journal, its editors and a handful of authors for libel after the publication printed numerous papers and editorials that the company says discredited the drug.

Pacira Biosciences filed the complaint against the American Society of Anesthesiologists in the US District Court for New Jersey on Wednesday afternoon. A February issue of the group’s journal Anesthesiology printed three articles and other content full of “bias” that “seriously disparaged” the drug Exparel, Pacira claimed.