Re­gen­eron hands off ex-US rights for cho­les­terol dis­or­der drug to rare dis­ease spe­cial­ist Ul­tragenyx

Re­gen­eron has held high hopes for evinacum­ab, a rare cho­les­terol dis­or­der drug that earned a first-in-class nod from the FDA ear­ly last year, as a cen­ter­piece of its car­diometa­bol­ic fran­chise. Now, the com­pa­ny is tap­ping an ul­tra-rare spe­cial­ist to take evinacum­ab to the next lev­el abroad.

Re­gen­eron will re­ceive $30 mil­lion in up­front cash and a po­ten­tial $63 mil­lion in down­stream mile­stones from Ul­tragenyx for ex-US li­cens­ing rights to Evkeeza (evinacum­ab), an ANGPTL3 block­er with an FDA ap­proval to treat a rare cho­les­terol dis­or­der along­side LDL-C low­er­ing ther­a­py and di­et, the com­pa­nies said Fri­day.

Ul­tragenyx, a small phar­ma known best for its ul­tra-rare dis­ease drugs, will pick up de­vel­op­ment and com­mer­cial­iza­tion costs for evinacum­ab in pa­tients with the con­di­tion known as ho­mozy­gous fa­mil­ial hy­per­c­ho­les­terolemia (HoFH), which is be­lieved to af­fect any­where be­tween 1 in 160,000 and 300,000 peo­ple world­wide.

In ad­di­tion, Re­gen­eron and Ul­tragenyx have set pre­lim­i­nary terms for an ex-US li­cens­ing deal for one of Re­gen­eron’s in­ves­ti­ga­tion­al drugs, an an­ti­body de­signed to treat the ul­tra-rare dis­ease fi­brodys­pla­sia os­si­f­i­cans pro­gres­si­va (FOP). Terms for that po­ten­tial part­ner­ship have yet to be set.

Evinacum­ab was ap­proved last Feb­ru­ary at the hefty price tag of an av­er­age of $450,000 per year. It was the first drug ap­proved to in­hib­it ANGPTL3, or an­giopoi­etin-like 3, a pro­tein in­volved in lipid me­tab­o­lism.

In piv­otal da­ta back­ing the ap­proval, pa­tients tak­ing evinacum­ab saw a 47% re­duc­tion in LDL lev­els from base­line, com­pared to a 2% in­crease in the con­trol. The drug arm al­so saw sig­nif­i­cant re­duc­tions in lev­els of apolipopro­tein B, an in­di­ca­tor of vas­cu­lar dis­ease, as well as triglyc­erides and over­all cho­les­terol.

Evinacum­ab was en­vi­sioned as a sort of nat­ur­al fol­low-up for Re­gen­eron’s Pralu­ent, a part­nered PC­SK9 block­er with Sanofi ap­proved to low­er LDL-C. That drug has been locked in a years­long pric­ing bat­tle with Am­gen’s Repatha in what has ef­fec­tive­ly be­come a war of at­tri­tion over price de­creas­es in the pricey PC­SK9 class.

Ul­tragenyx, mean­while, scored its first FDA ap­proval some five years ago with Mep­se­vii, a drug ap­proved to treat an ul­tra-rare ge­net­ic en­zyme dis­or­der called MPS VII. Since then, the drug­mak­er has earned its stature as an ul­tra-rare spe­cial­ist, bring­ing two ad­di­tion­al drugs on­to the mar­ket in Crysvi­ta, ap­proved to treat two forms of hy­pophos­phatemia, and Do­jolvi, an en­gi­neered medi­um-chain fat­ty acid de­signed as a sup­ple­ment for pa­tients with mol­e­c­u­lar­ly con­firmed long-chain fat­ty acid ox­i­da­tion dis­or­ders.

Jean-Paul Clozel, Idorsia CEO (Patrick Straub/Keystone via AP Images)

Idor­si­a's brain bleed drug flunks PhI­II tri­al, a decade af­ter pre­vi­ous flop

Idorsia’s long journey with clazosentan came to an abrupt “unexpected result” Monday morning with a Phase III flop.

The Swiss biopharma said the drug did not meet the main goal of the late-stage REACT study, conducted in the US, Canada and Europe since early 2019.

The 409-patient trial tested the intravenous drug’s ability to prevent complications due to delayed cerebral ischemia following aneurysmal subarachnoid hemorrhage (aSAH), in which blood vessels in the brain narrow and blood accumulates around the brain’s surface, which then dials up the pressure on the brain.

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Kenji Yasukawa, Astellas CEO (Photographer: Akio Kon/Bloomberg via Getty Images)

Astel­las taps chief strat­e­gy of­fi­cer as next CEO to 'go on the ag­gres­sive'

Five years into its big R&D revamp, Astellas says it’s time for a changing of the guard.

Kenji Yasukawa, who took over as president and CEO in 2018, will step down to become chairman of the board in April, making room for Naoki Okamura to take over. Okamura joined the company in 1986 and has served in a variety of finance, business and strategy roles, including most recently as chief strategy officer.

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Clin­i­cal tri­al di­ver­si­ty da­ta show mis­match be­tween en­roll­ment and dis­ease preva­lence, GSK says

A lack of diversity in clinical trials has persisted despite decades of initiatives to try to turn the tide.

In a recent review of 17 years of clinical trials, drugmaker GSK found that there were some mismatches between the demographics of its US-based trials and how prevalent diseases were in those populations.

The results, the company says, will help GSK and others design studies that better represent epidemiological rates within races and ethnicities.

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; 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.

With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

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Goldfinch Bio CEO Tony Johnson (L) and Karuna Therapeutics CEO Bill Meury

Karuna li­cens­es Goldfinch as­sets to com­pete with Boehringer In­gel­heim in neu­ro­science

Karuna Therapeutics is looking to compete with Boehringer Ingelheim on depression and anxiety with a new license to Goldfinch Bio’s assets, starting with $15 million to the shuttered biotech.

Karuna steps into an arena already being tested by Boehringer in multiple Phase II studies — the two are targeting transient receptor potential canonical 4 and 5, or TRPC4/5, which is thought to have a role in neuroscience indications. Goldfinch’s asset went through a Phase II in kidney diseases, but Karuna’s sights are set on mood and anxiety disorders for now.

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Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

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Trodelvy notch­es a win in most com­mon form of breast can­cer

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.

Raymond Stevens, Structure Therapeutics CEO

Be­hind Fri­day's $161M IPO: A star sci­en­tist, GPCR drug dis­cov­ery and a plan to chal­lenge phar­ma in di­a­betes

What does it take to pull off a $161 million biotech IPO these days?

In Structure Therapeutics’ case, it means having a star scientist co-founder paired with the computational drug discovery company Schrödinger, $198 million in private funding from blue-chip investors, almost six years of research work on G protein-coupled receptors and a slate of oral, small-molecule drugs, with an eye on the huge and growing diabetes and weight-loss market.

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Af­ter 13 years, Ramy Mah­moud steps in­to CEO seat at Opti­nose; Ru­pert Vessey set to ex­it Bris­tol My­ers in Ju­ly

After 13 years as president and COO at Optinose, Ramy Mahmoud has stepped into a new role as its CEO. He is taking the place of Peter Miller, who stepped down earlier this week, though Miller is still staying with the company as a consultant.

In 2010, the two business partners joined Optinose to take it in a new direction, transforming it from a delivery platform to product company. They previously worked together at Johnson & Johnson, when Miller was president at Janssen and Mahmoud headed medical affairs. Miller said after he learned about Optinose, “I did what I always do, which is find people smarter than me to talk with about the idea. And the first person I called was Ramy … and I said, ‘Hey, Ramy, what do you think of this technology?’”

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