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.

Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Sanofi out­lines big API plans as coro­n­avirus out­break re­port­ed­ly threat­ens short­age of 150 drugs

As the world becomes increasingly dependant on Asia for the ingredients of its medicines, Sanofi sees business to be done in Europe.

The French drugmaker said it’s creating the world’s second largest active pharmaceutical ingredients (API) manufacturer by spinning out its six current sites into a standalone company: Brindisi (Italy), Frankfurt Chemistry (Germany), Haverhill (UK), St Aubin les Elbeuf (France), Újpest (Hungary) and Vertolaye (France). They have mapped out €1 billion in expected sales by 2022 and 3,100 employees for the new operations headquartered in France.

UP­DAT­ED: NGM Bio takes leap for­ward in crowd­ed NASH field

South San Francisco-based NGM Bio may have underwhelmed with its interim analysis of a key cohort from a mid-stage NASH study last fall — but stellar topline data unveiled on Monday showed the compound induced significant signs of antifibrotic activity, NASH resolution and liver fat reduction, sending the company’s stock soaring.

There are an estimated 50+ companies focused on developing drugs for non-alcoholic steatohepatitis, or NASH, a common liver disease that has long flummoxed researchers. The first wave of NASH drug developers struggled with efficacy as well as safety — and companies big and small have crashed and burned.

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Mickey Kertesz, KidsandArtOrg via YouTube

Soft­Bank's newest, $165M biotech in­vest­ment looks for in­fec­tious traces in the blood

SoftBank has found its newest biotech investment.

The Japanese bank has invested $165 million into Karius, a company that uses blood tests to diagnose infectious diseases, as part of its new Vision Fund 2. The full scope of the new fund has yet to be announced, but the first and newly-beleaguered Vision Fund poured $100 billion into technology companies, including the biotechs Vir Biotechnology and Roivant and the sequencing company 10x Genomics.

Methicillin-resistant Staph aureus (Shutterstock)

FDA grants ‘break­through’ sta­tus to an­tibi­ot­ic al­ter­na­tive as Con­tra­Fect rush­es to join fight against su­per­bug

An experimental drug that promises to be the first anti-infective agent to prove superior to vancomycin — an antibiotic approved in 1958 — has notched the FDA’s “breakthrough” status.

ContraFect said the designation was based on Phase II data in which exebacase was tested against a superbug known as methicillin-resistant Staph aureus, or MRSA. In a subgroup analysis, the clinical responder rate at day 14 was 42.8% higher than that among those treated with standard of care, the company said (p=0.010).

Zhong Nanshan, CGTN via YouTube

Har­vard joins coro­n­avirus fight with $115 mil­lion and a high-pro­file Chi­nese part­ner

For two months, as the novel coronavirus swelled from a few early cases tied to a Wuhan market to a global epidemic, most of the world’s focus and dollars have flowed toward emergency initiatives: building vaccines at a record pace, plucking experimental antivirals out of freezers to see what sticks and immunizing mice for new antibodies.

Now a new and well-funded collaboration between Harvard and a top Chinese research institute will play the long game. In a 5-year, $115 million initiative backed by China Evergrande Group, researchers from the Harvard Medical School, Harvard T.H. Chan School of Public Health and Guangzhou Institute for Respiratory Health will study the virus in an effort to develop therapies against infections by the novel coronavirus, known as SARS–CoV-2, and to prevent new ones.

No­var­tis gets a boost in block­buster mul­ti­ple scle­ro­sis race with Roche

In the first step of what’s likely to be a long and uphill battle for the drugmaker, the FDA has accepted Novartis’s BLA submission for a new multiple sclerosis drug and given it priority review. The PDUFA date for the potential blockbuster drug is in June.

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Juergen Horn

An­i­mal health vet Juer­gen Horn makes new an­ti­body play for pets, rak­ing $15M in Se­ries A haul

Zoetis forked over $85 million in 2017 to acquire Nexvet Biopharma and its pipeline of monoclonal antibodies. Juergen Horn, Nexvet’s former chief product development officer, has now secured $15 million for his own biologic company for animals: Invetx.

Buoyed by emerging advances in gene therapies for humans, scientists have started looking at harnessing the technology for animals setting up companies such as Penn-partnered Scout Bio and George Church-founded Rejuvenate Bio. But akin to Nexvet, Invetx is working on leveraging the time-tested science of monoclonal antibodies to treat chronic diseases that afflict man’s best friend.

As coro­n­avirus out­break reach­es 'tip­ping point,' GSK lends ad­ju­vant tech to Chi­nese part­ner armed with pre­clin­i­cal vac­cine

As the coronavirus originating out of Wuhan spreads to South Korea, Italy and Iran, stoking already intense fears of a pandemic, GlaxoSmithKline has found another pair of trusted hands to place its adjuvant system. China’s Clover Biopharmaceuticals will add the adjuvant to its preclinical, protein-based vaccine candidate against SARS-CoV-2.

Clover, which is based in the inland city of Chengdu, boasts of a platform dubbed Trimer-Tag that produces covalently-trimerized fusion proteins. Its candidate, COVID-19 S-Trimer, resembles the viral spike (S)-protein found in the virus.