Af­ter thy­roid eye drug scores in PhI­II test, Hori­zon Phar­ma read­ies mar­ket­ing ap­pli­ca­tion

Less than two years af­ter Hori­zon Phar­ma $HZNP pro­cured phase III-ready thy­roid eye dis­ease (TED) drug tepro­tu­mum­ab for $145 mil­lion up­front by ac­quir­ing a unit of Nar­row Riv­er, biotech vet­er­an David Mad­den’s in­vest­ment firm — the bet has paid off, as the au­toim­mune in­flam­ma­to­ry dis­or­der drug passed its late-stage test on Thurs­day.

Tim Wal­bert, Hori­zon CEO

In TED pa­tients, the eye mus­cles and fat­ty tis­sue be­hind the eye be­come in­flamed, caus­ing the eyes to ap­pear to be ‘bulging’ and eye­lids to be­come swollen and red, even­tu­al­ly cul­mi­nat­ing in eye mis­align­ment, dou­ble vi­sion and in some cas­es even blind­ness — but there is lim­it­ed win­dow of up to three years dur­ing which the con­di­tion can be treat­ed with­out sur­gi­cal in­ter­ven­tion.

Tepro­tu­mum­ab is a mon­o­clon­al an­ti­body de­signed to in­hib­it in­sulin-like growth fac­tor 1 re­cep­tor (IGF-1R). In the 83-pa­tient phase III tri­al, 82.9% of pa­tients treat­ed with tepro­tu­mum­ab ex­pe­ri­enced a mean­ing­ful im­prove­ment in prop­to­sis or bulging of the eye (a 2 mm or more re­duc­tion (p<0.001)) com­pared to 9.5% of pa­tients on the place­bo — meet­ing the main goal of the study. All the sec­ondary end­points were al­so met, the com­pa­ny said.

The rare dis­ease drug­mak­er is work­ing on sub­mit­ting a mar­ket­ing ap­pli­ca­tion for tepro­tu­mum­ab by the mid­dle of 2019, it said. The FDA has grant­ed the ex­per­i­men­tal TED treat­ment — which was orig­i­nal­ly de­vel­oped by Gen­mab and sub­se­quent­ly li­censed from Roche — all the bells and whis­tles: break­through ther­a­py, or­phan drug and fast track des­ig­na­tions that should make a speedy re­view like­ly.

Ray­mond Dou­glas

“If ap­proved, tepro­tu­mum­ab would give physi­cians the first med­i­cine shown to re­duce prop­to­sis dur­ing ac­tive thy­roid eye dis­ease, in ad­di­tion to treat­ing oth­er painful symp­toms,” said Ray­mond Dou­glas, the study’s co-prin­ci­pal in­ves­ti­ga­tor and di­rec­tor of the or­bital and thy­roid eye dis­ease pro­gram at the Cedars-Sinai Med­ical Cen­ter, in a state­ment.

If the BLA is ac­cept­ed, the drug is po­si­tioned for launch in the first half of next year — giv­en the pos­si­bil­i­ty of pri­or­i­ty re­view, Jef­feries an­a­lyst David Stein­berg wrote in a note, adding that there are rough­ly 15,000 to 20,000 ac­tive TED US pa­tients el­i­gi­ble for treat­ment.

Stein­berg es­ti­mat­ed $500 mil­lion in peak sales of the drug by 2024, but added that Hori­zon man­age­ment has fore­cast $750 mil­lion, giv­en the drug has se­cured or­phan sta­tus and is dis­ease-mod­i­fy­ing — po­ten­tial­ly paving the way for ro­bust pric­ing.

Hori­zon cur­rent­ly sells the drug with the high­est price tag in the Unit­ed States — Ac­tim­mune.

“Giv­en the strong ex­e­cu­tion in the re­launch of Krys­texxa (a drug for chron­ic gout) and now on the cusp of in­tro­duc­ing an­oth­er long du­ra­tion or­phan drug with tepro, we in­creas­ing­ly see HZNP be­com­ing a leader in the rare dis­ease area,” Stein­berg wrote.

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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With dust set­tled on ac­tivist at­tack, Lau­rence Coop­er leaves Zio­pharm to a new board

Laurence Cooper has done his part.

In the five years since he left a tenured position at Houston’s MD Anderson Cancer Center to become CEO of Boston-based Ziopharm, he’s steered the small-cap immunotherapy player through patient deaths in trials, clinical holds, short attacks and, most recently, an activist attack on the board.

So when the company has “fantastic news” like an IND clearance for a TCR T cell therapy program, he’s ready to pass on the baton.

Doug Ingram (file photo)

Why not? Sarep­ta’s third Duchenne MD drug sails to ac­cel­er­at­ed ap­proval

Sarepta may be running into some trouble with its next-gen gene therapy approach to Duchenne muscular dystrophy. But when it comes to antisense oligonucleotides, the well-trodden regulatory path is still leading straight to an accelerated approval for casimersen, now christened Amondys 45.

We just have to wait until 2024 to find out if it works.

Amondys 45’s approval was unceremonious, compared to its two older siblings. There was no controversy within the FDA over approving a drug based on a biomarker rather than clinical benefit, setting up a powerful precedent that still haunts acting FDA commissioner Janet Woodcock as biotech insiders weighed her potential permanent appointment; no drama like the FDA issuing a stunning rejection only to reverse its decision and hand out an OK four months later, which got more complicated after the scathing complete response letter was published; no anxious tea leaf reading or heated arguments from drug developers and patient advocates who were tired of having corticosteroids as their loved ones’ only (sometimes expensive) option.

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Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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Tal Zaks, Moderna CMO (AP Photo/Rodrique Ngowi, via still image from video)

CMO Tal Zaks bids Mod­er­na a sur­prise adieu as biotech projects $18.4B in rev­enue, plots post-Covid ex­pan­sion

How do you exit a company after six years in style? Developing one of the most lucrative and life-saving products in pharma history is probably not the worst way to go.

Tal Zaks, Moderna’s CMO since 2015, will leave the mRNA biotech in September, the biotech disclosed in their annual report this morning. The company has already retained the recruitment firm Russell Reynolds to find a replacement.

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Glax­o­SmithK­line re­thinks strat­e­gy for Covid-19 an­ti­body — not the Vir ones — af­ter tri­al flop. Is there hope in high-risk pa­tients?

In the search for a better Covid-19 therapeutic, GlaxoSmithKline and Vir have partnered up on two antibodies they hope have a chance. GSK is also testing its own in-house antibody, and early results may have shut the door on its widespread use.

A combination of GSK’s monoclonal antibody otilimab plus standard of care couldn’t best standard of care alone in preventing death and respiratory failure in hospitalized Covid-19 patients after 28 days, according to data from the Phase IIa OSCAR study unveiled Thursday.

Mod­er­na's Stéphane Ban­cel plans to dou­ble down on vac­cine pro­duc­tion, new vari­ants as mR­NA rules in pan­dem­ic fight

Stéphane Bancel thought he’d be sleeping more by now.

The 48-year-old Moderna CEO figured that by 2021 he’d have his vaccine through the clinic, authorized, and in mass production — that the hard part would be over. Instead, he’s still working Saturdays and Sundays, talking with his lab and manufacturing teams and fielding calls with two to three world leaders a day to answer their concerns about supply and emerging new variants.

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