Sanofi, Re­gen­eron bur­nish their block­buster Dupix­ent fran­chise with pos­i­tive re­sults in chil­dren with un­con­trolled eczema

As a sta­ble of new play­ers sets out to top­ple Dupix­ent’s sta­tus as the reign­ing an­ti­body treat­ment for atopic der­mati­tis, the block­buster’s de­vel­op­ers are mov­ing the goal­posts.

Dupix­ent has aced a Phase III tri­al in­volv­ing 162 chil­dren be­tween 6 months and 5 years old, Sanofi and Re­gen­eron say, be­com­ing the first bi­o­log­ic drug to show ef­fi­ca­cy in this young pop­u­la­tion. When giv­en Dupix­ent on top of stan­dard of care top­i­cal cor­ti­cos­teroids, 28% of pa­tients achieved clear or al­most-clear skin com­pared to 4% of those on place­bo — thus meet­ing the pri­ma­ry end­point.

Sanofi and Re­gen­eron have bet big on the Dupix­ent fran­chise, and an­a­lysts have large­ly ex­pect­ed it to pay off, with Jef­feries once peg­ging peak sales at $12.5 bil­lion.

Core to the pitch for physi­cians will be the fact that even as it clears eczema, the drug doesn’t broad­ly sup­press the im­mune sys­tem, which could be par­tic­u­lar­ly dan­ger­ous for young in­fants.

By bind­ing to IL-4Rα, the drug is de­signed to block sig­nal­ing of the IL-4 and IL-13 path­ways.

“When a child is di­ag­nosed with mod­er­ate-to-se­vere atopic der­mati­tis in the first few months of life, many as­pects of their child­hood can be sig­nif­i­cant­ly im­pact­ed. Par­ents and care­givers are chal­lenged to find safe and ef­fec­tive treat­ment op­tions,” said John Reed, Sanofi’s head of R&D. “Cur­rent­ly, the stan­dard of care for this pa­tient pop­u­la­tion is top­i­cal steroids and oth­er im­muno­sup­pres­sive med­i­cines may be used which can dam­age del­i­cate skin and, if used long-term, po­ten­tial­ly im­pact growth.”

The drug, which is al­ready ap­proved for chil­dren aged 6 or above, al­so met the co-pri­ma­ry end­point out­side of the US, which is 75% or greater over­all dis­ease im­prove­ment from base­line. In the drug arm, 53% hit that thresh­old, ver­sus 11% with place­bo, good for a p-val­ue of p<0.0001.

In­ves­ti­ga­tors al­so spelled out stel­lar num­bers on a hand­ful of sec­ondary end­points, in ad­di­tion to “sig­nif­i­cant­ly im­proved mea­sures” in symp­toms such as sleep, skin pain and oth­er qual­i­ty of life met­rics.

  • 70% av­er­age im­prove­ment from base­line in EASI com­pared to 20% im­prove­ment with place­bo (p<0.0001)
  • 49% av­er­age im­prove­ment from base­line in itch com­pared to 2% im­prove­ment in place­bo (p<0.0001)

Dur­ing the 16-week treat­ment pe­ri­od, the com­pa­nies added, pa­tients on Dupix­ent were 50% less like­ly to get a skin in­fec­tion — 12% of them had at least one in­fec­tion, com­pared to 24% on place­bo. Over­all, there were 11 in­fec­tions in the drug arm and 34 in­fec­tions in the place­bo arm.

Eli Lil­ly, Pfiz­er, Am­gen and oth­ers are hot in pur­suit of the drug as they seek a slice of the eczema mar­ket for them­selves. But they will first have to crash a sol­id wall of da­ta that Sanofi and Re­gen­eron con­tin­ue to build.

Ed­i­tor’s Note: This sto­ry has been up­dat­ed to clar­i­fy the re­sults on skin in­fec­tions.

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His­toric drug pric­ing re­forms pass; Pfiz­er ac­quires GBT; The long search for non-opi­oid pain drugs; 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.

The Endpoints Weekly has officially crossed the 60,000 mark on subscribers — thanks to all of your support. As the editorial team grows, we’ve been able to do a lot more, with many of those on display this week. Be sure to check out Lei Lei Wu’s deep dive on pain R&D. If you missed it, you may also rewatch her companion panel here.

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Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

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No­var­tis re­ports two pa­tient deaths af­ter treat­ment with Zol­gens­ma

Two children with spinal muscular atrophy have died after receiving Novartis’ Zolgensma, a gene therapy designed as a one-time treatment for the rare fatal disease.

The deaths, which resulted from acute liver failure, occurred in Russia and Kazakhstan, Novartis confirmed in a statement to Endpoints News. Having notified health authorities across all the markets where Zolgensma is available, it will update the drug label “to specify that fatal acute liver failure has been reported,” a spokesperson wrote.

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Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

Sen­ate Fi­nance chair con­tin­ues his in­ves­ti­ga­tion in­to phar­ma tax­es with re­quests for Am­gen

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.

FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

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J&J to re­move talc prod­ucts from shelves world­wide, re­plac­ing with corn­starch-based port­fo­lio

After controversially spinning out its talc liabilities and filing for bankruptcy in an attempt to settle 38,000 lawsuits, Johnson & Johnson is now changing up the formula for its baby powder products.

J&J is beginning the transition to an all cornstarch-based baby powder portfolio, the pharma giant announced on Thursday — just months after a federal judge ruled in favor of its “Texas two-step” bankruptcy to settle allegations that its talc products contained asbestos and caused cancer. An appeals court has since agreed to revisit that case.

CSL is gathering its four business units under a unified brand identity strategy (Credit: CSL company site)

CSL brings Se­qirus, Vi­for un­der par­ent um­brel­la brand in iden­ti­ty re­vamp

CSL is gathering its brands under the family name umbrella, renaming its vaccine and newly acquired nephrology specialty businesses with the parent initials.

CSL Seqirus and CSL Vifor join CSL Plasma and CSL Behring as the four now uniformly branded business units of the global biopharma. The Seqirus vaccine division was formed in 2015 with the combination of bioCSL and its purchase of Novartis’ flu vaccine business. CSL picked up Vifor Pharma late last year in an $11.7 billion deal for the nephrology, iron deficiency and cardio-renal drug developer.

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Anirvan Ghosh, Unity CEO (via Facebook)

Uni­ty finds it­self with a fi­nite amount of cash as new da­ta pro­vide a ray of hope for in­vestors

It has not been an easy road for the anti-aging startup Unity Biotechnology. From staff cuts to a lead program loss, the company has run into several obstacles on its journey, but their latest news points to both the perils and potential ahead.

In the biotech’s Q2 results on Friday, the company revealed it has only enough cash to fund its operations through the first quarter of next year, with posted results showing that Unity had $64.5 million in cash and market securities at the end of the quarter. This is compared to the $79.2 million the biotech had at the end of March. The company also had an operating loss of $12.3 million for the quarter as well.