French biotech In­ven­ti­va's lead drug stum­bles in sys­temic scle­ro­sis study, ahead of NASH read­out

France’s In­ven­ti­va (Eu­ronext: $IVA) has suf­fered its first big set­back fol­low­ing its pub­lic list­ing in 2017. The com­pa­ny’s lead ex­per­i­men­tal drug, lan­i­fi­bra­nor, failed a mid-stage tri­al in pa­tients with a form of sys­temic scle­ro­sis (SSc) — a rare, chron­ic life-threat­en­ing dis­or­der in which the im­mune sys­tem at­tacks its own or­gans and is char­ac­ter­ized by a buildup of scar tis­sue — prompt­ing the Daix-based drug de­vel­op­er to aban­don the pro­gram for the in­di­ca­tion.

The Phase IIb tri­al eval­u­at­ed two dos­es of lan­i­fi­bra­nor (800 mg, 1200 mg) against a place­bo in 145 pa­tients with dif­fuse cu­ta­neous sys­temic scle­ro­sis, which ac­count for rough­ly 35% of the SSc pop­u­la­tion. Pa­tients were giv­en lan­i­fi­bra­nor in ei­ther two dos­es of 400mg per day or two dos­es of 600mg per day over 48 weeks in ad­di­tion to stan­dard of care, which typ­i­cal­ly in­clud­ed im­muno­sup­pres­sive ther­a­py.

Com­pared to the place­bo, nei­ther dose of the drug in­duced a sta­tis­ti­cal­ly sig­nif­i­cant change in the mod­i­fied Rod­nan Skin Score — a scale mea­sur­ing the evo­lu­tion of skin fi­bro­sis, which is cor­re­lat­ed with in­ter­nal scar­ring — miss­ing the main goal of the study. None of the sec­ondary end­points, in­clud­ing changes in pul­monary func­tion mea­sured via forced vi­tal ca­pac­i­ty and over­all pro­gres­sion of the dis­ease, were met, In­ven­ti­va said on Mon­day.

There are no ap­proved ther­a­pies for SSc, a dis­ease that dis­pro­por­tion­ate­ly af­fects women. Ac­cord­ing to an­a­lysts at H.C. Wain­wright, the dif­fuse cu­ta­neous form of the dis­ease trans­lates to an ad­dress­able mar­ket of about 23,500 pa­tients in the Eu­rope and 31,500 in the Unit­ed States.

Lan­i­fi­bra­nor is an oral small mol­e­cule de­signed to spark an­tifi­brot­ic, an­ti-in­flam­ma­to­ry changes by spurring three iso­forms — α, δ and γ — of PPAR (per­ox­i­some pro­lif­er­a­tor-ac­ti­vat­ed re­cep­tor), which are nu­clear re­cep­tor pro­teins that reg­u­late gene ex­pres­sion. There are oth­er PPAR ag­o­nists on the mar­ket (in­clud­ing di­a­betes drug pi­ogli­ta­zone) and in de­vel­op­ment (ex­per­i­men­tal NASH drugs such as Gen­fit’s elafi­bra­nor and CymaBay’s se­ladel­par) that tar­get on­ly one or two PPAR iso­forms for ac­ti­va­tion, but lan­i­fi­bra­nor is a pan-PPAR ag­o­nist like bezafi­brate, which In­ter­cept ac­quired last month to de­vel­op in com­bi­na­tion with its NASH con­tender obeti­cholic acid.

In­ven­ti­va is al­so test­ing lan­i­fi­bra­nor for NASH — a fat­ty liv­er dis­ease af­fect­ing mil­lions that has no ap­proved ther­a­pies — which has thus cap­ti­vat­ed a pletho­ra of drug de­vel­op­ers, in­clud­ing Gilead $GILD, In­ter­cept $ICPT and Gen­fit (Eu­ronext: $GN­FT). In­ven­ti­va’s Phase IIb tri­al for lan­i­fi­bra­nor in NASH pa­tients is ex­pect­ed to read­out in the first half of 2020.

In a note ear­li­er this month, H.C. Wain­wright an­a­lysts sug­gest­ed that pos­i­tive SSc da­ta would bode well for In­ven­ti­va’s NASH pro­gram: “(Grow­ing) clin­i­cal ev­i­dence points to a mech­a­nis­tic link be­tween fi­brot­ic NASH and cer­tain in­flam­ma­to­ry skin con­di­tions, like pso­ri­a­sis…  an­oth­er an­ti-fi­brot­ic agent in de­vel­op­ment for NASH, Galectin Ther­a­peu­tics’ GR-MD-02, has sep­a­rate­ly re­port­ed sig­nif­i­cant im­prove­ment in pa­tients across both plaque pso­ri­a­sis and cir­rhot­ic NASH.”

Last year, In­ven­ti­va raised raised about $44 mil­lion from a group of ven­ture back­ers, in­clud­ing Paris-based Sofinno­va, af­ter go­ing pub­lic in 2017 in a $51 mil­lion IPO. The com­pa­ny, which has pro­grams part­nered with Ab­b­Vie and Boehringer In­gel­heim, was spun out of Ab­bott in 2012.

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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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House pass­es his­toric drug pric­ing re­forms, lin­ing up decades-in-the-mak­ing win for Biden and De­moc­rats

The US House of Representatives today voted along party lines (all Dems voted for it), 220-207 to pass new, wide-ranging legislation that will allow Medicare drug price negotiations for the first time ever, and cap seniors’ drug expenses to $2,000 per year and seniors’ insulin costs at $35 per month.

Setting up a major victory for President Joe Biden, representatives returned from their summer recess to pass the Inflation Reduction Act, even as many noted the bill would only modestly reduce inflation.

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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