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.

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

A lab technician works during research on coronavirus at Johnson & Johnson subsidiary Janssen Pharmaceutical in Beerse, Belgium, Wednesday, June 17, 2020. (Virginia Mayo/AP Images)

End­points News ranks all 28 play­ers in the Covid-19 vac­cine race. Here's how it stacks up to­day

The 28 players now in or close to the clinical race to get a Covid-19 vaccine over the finish line are angling for a piece of a multibillion-dollar market. And being first — or among the leaders — will play a big role in determining just how big a piece.

Endpoints News writer Nicole DeFeudis has posted a snapshot of all the companies, universities and hospital-based groups now racing through the clinic, ranking them according to their place in the pipeline as well as the latest remarks available on timelines. And we’ll keep this lineup updated right through the end of the year, as the checkered flags start to fall, possibly as early as October.

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Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

Bayer's Marianne De Backer with Endpoints founder John Carroll, Endpoints@JPM20 (Jeff Rumans for Endpoints News)

UP­DAT­ED: Hunt­ing a block­buster, Bay­er forges an $875M-plus M&A deal to ac­quire women’s health biotech

Bayer has dropped $425 million in cash on its latest women’s health bet, bringing a UK biotech and its non-hormonal menopause treatment into the fold.

KaNDy Therapeutics had its roots in GlaxoSmithKline, which spun out several neuroscience drugs into NeRRe Therapeutics back in 2012. Five years later the team created a new biotech to focus solely on NT-814 — which they considered “one of the few true innovations in women’s health in more than two decades.”

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Eric Shaff (Seres)

UP­DAT­ED: Af­ter a 4-year so­journ, strug­gling mi­cro­bio­me pi­o­neer Seres claims a break­out PhI­II come­back. And shares re­spond in fren­zied spike

Almost exactly 4 years ago, Seres Therapeutics $MCRB experienced one of those soul-crunching failures that can raise big questions about a biotech’s future. Out front in their pursuit of a gut punch to C. difficile infection (CDI), the Phase II test was a flat failure, and investors wiped out a billion dollars of equity value that never returned in the years that followed.

Seres, though, pressed ahead, changing out CEOs a year ago — bidding Merck vet Roger Pomerantz farewell from the C suite — and pushing through a Phase III, hoping that amping up the dosage would make the key difference. And this morning, they unveiled a claim that they had aced the Phase III and positioned themselves for a run at a landmark FDA OK.

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Michel Vounatsos, Biogen CEO (via YouTube)

Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

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Robert Gould, Fulcrum Therapeutics CEO

Ful­crum stum­bles in PhII of old GSK drug, send­ing shares tum­bling

Investors are selling off shares of Fulcrum Therapeutics $FULC after their lead drug failed in a Phase II trial.

The company, founded three years ago on new research techniques such as CRISPR screening, isolated a gene called DUX4 they believed to have a central role in facioscapulohumeral muscular dystrophy, where patients’ muscle dies and is replaced by fat. And to target it, they licensed a GlaxoSmithKline drug that had failed as a cardio drug.

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Eisai moves to 200 Metro Blvd. by late 2021 (ON3)

Ei­sai is cre­at­ing a new US cor­po­rate, R&D HQ in Roche’s old Nut­ley, NJ cam­pus

Eight years after Roche pulled up stakes from Nutley, NJ in a major R&D reorganization, Japan’s Eisai is moving its US corporate and research hub into their old campus.

Now the ON3 property, Eisai — a longtime Biogen partner focused on neurodegenerative disorders like Alzheimer’s — will bring together a staff of up to 1,200 employees. And execs are pitching the move to the New Jersey campus as a cultural game-changer.

Lig­and scoops up Pfenex for up to $516M, adding pro­teins to their an­ti­body chick­ens and de­liv­ery tech

The technology hunting folks over at Ligand Pharmaceuticals have picked up a new one from across town, for a significant price.

Ligand has acquired fellow San Diego-based biotech Pfenex and their protein expression platform for $438 million cash, plus $78 million in contingent value agreements should an undisclosed milestone be hit before the end of next year.  The deal pays $12 per share, or $4.34 more than what Pfenex had been trading at before the announcement.