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.

A New Fron­tier: The In­ner Ear

What happens when a successful biotech venture capitalist is unexpectedly diagnosed with a chronic, life-disrupting vertigo disorder? Innovation in neurotology.

That venture capitalist was Jay Lichter, Ph.D., and after learning there was no FDA-approved drug treatment for his condition, Ménière’s disease, he decided to create a company to bring drug development to neurotology. Otonomy was founded in 2008 and is dedicated to finding new drug treatments for the hugely underserved community living with balance and hearing disorders. Helping patients like Jay has been the driving force behind Otonomy, a company heading into a transformative 2020 with three clinical trial readouts: Phase 3 in Ménière’s disease, Phase 2 in tinnitus, and Phase 1/2 in hearing loss. These catalysts, together with others in the field, highlight the emerging opportunity in neurotology.
Otonomy is leading the way in neurotology
Neurotology, or the treatment of inner ear neurological disorders, is a large and untapped market for drug developers: one in eight individuals in the U.S. have moderate-to-severe hearing loss, tinnitus or vertigo disorders such as Ménière’s disease.1 With no FDA-approved drug treatments available for these conditions, the burden on patients—including social anxiety, lower quality of life, reduced work productivity, and higher rates of depression—can be significant.2, 3, 4

Af­ter 4 years of furor, the FTC and New York state ac­cuse Mar­tin Shkre­li of run­ning a drug mo­nop­oly. And this time they plan to squash it

Pharma bro Martin Shkreli was jailed, publicly pilloried and forced to confront some lawmakers in Washington riled by his move to take an old generic and move the price from $17.50 per pill to $750. But through 4 years of controversy and public revulsion, his company never backed away from the price — left uncontrolled by a laissez faire federal policy on a drug’s cost.

Now the FTC and the state of New York plan to pry his fingers off the drug once and for all and open it up to some cheap competition.

Patrik Jonsson, the president of Lilly Bio-Medicines

Who knew? Der­mi­ra’s board kept watch as its stock price tracked Eli Lil­ly’s se­cret bid­ding on a $1.1B buy­out

In just 8 days, from December 6 to December 14, the stock jumped from $7.88 to $12.70 — just under the initial $13 bid. There was no hard news about the company that would explain a rise like that tracking closely to the bid offer, raising the obvious question of whether insider info has leaked out to traders.

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Short at­tack­er Sahm Ad­ran­gi draws crosshairs over a fa­vorite of Sanofi’s new CEO — with PhII da­ta loom­ing

Sahm Adrang Kerrisdale

Kerrisdale chief Sahm Adrangi took a lengthy break from his series of biotech short attacks after his chief analyst in the field pulled up stakes and went solo. But he’s making a return to drug development this morning, drawing crosshairs over a company that’s one of new Sanofi CEO Paul Hudson’s favorite collaborators.

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UP­DAT­ED: Ac­celeron of­fers thumbs up on a PhII suc­cess for would-be block­buster drug — and shares rock­et up

There’s no public data yet, but Acceleron $XLRN says that its first major trial readout of 2020 is a success.

In a Phase II study of 106 patients with pulmonary arterial hypertension (PAH), Acceleron’s experimental drug sotatercept hit its primary endpoint: a significant reduction in pulmonary vascular resistance. The drug also met three different secondary endpoints, including the 6-minute walking test.

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Civi­ca and Blue Cross Blue Shield launch new ven­ture to low­er gener­ic prices

Five years after Martin Shkreli put a smug face to the volatile prices companies can charge even for generic drugs, payers and governments are coming up with outside-the-box solutions.

The latest fix is a new venture from the Blue Cross Blue Shield Association, 18 of its members and Civica, the generics company founded in 2018 by hospitals fed up with high prices for drugs that had long-since lost patent protection. While Civica focused on drugs that hospitals purchased, the new company will aim to lower prices on drugs that, like Shkreli’s Daraprim, are purchased by individuals.

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Merck Invests in State-Of-The-Art Biotech Development Facility in Switzerland

Mer­ck KGaA match­es lofty R&D goals with €250M in­vest­ment in­to a new clin­i­cal man­u­fac­tur­ing site in Switzer­land

As Merck KGaA strives to prove itself as a capable biopharma R&D player, it has begun construction on a €250 million facility dedicated to developing and manufacturing drugs for use in clinical trials.

The German drugmaker chose a location at Corsier-sur-Vevey, Switzerland, where it already has a commercial manufacturing site, in order to “bridge together research and manufacturing.”

“This investment in the Merck Biotech Development Center reflects our commitment to speed up the availability of new medicines for patients in need, and confirms the importance of Switzerland as our prime hub for the manufacturing of biotech medicines,” CEO Stefan Oschmann said at the groundbreaking ceremony, according to a statement.

Breast can­cer ap­proval in tow, As­traZeneca, Dai­ichi armed an­ti­body scores in key gas­tric can­cer study

AstraZeneca kicked off Monday with a flurry of good news. Apart from unveiling positive results on its stroke trial testing its clot-fighter Brilinta, and welcoming its experimental IL-23 inhibitor brazikumab back from Allergan — the British drugmaker also disclosed some upbeat gastric cancer data on its HER2-positive oncology therapy it is collaborating on with Daiichi Sankyo.

Buoyed by the performance of its oncology drugs, last March AstraZeneca chief Pascal Soriot bet big to partner with Daiichi on the cancer drug, with $1.35 billion upfront in a deal worth up to roughly $7 billion. Roughly 8 months later, as 2019 drew to a close, the FDA swiftly approved the drug — trastuzumab deruxtecan — for use in breast cancer, months ahead of the expected decision date.

Sor­ren­to shrugs off an anony­mous pri­vate eq­ui­ty group’s $1B of­fer to buy the com­pa­ny

San Diego-based Sorrento Therapeutics isn’t going the M&A route — at least not today.

The biotech caused quite a stir when it put out word a few weeks ago that an unidentified private equity group was bidding a billion dollars-plus for the company. The news drove a quick spike in the company’s share price as investors hooked up for the ride — that didn’t happen.

The update sparked a 5% drop in the share price $SRNE ahead of the bell. It’s now trading just above $4, without any evidence that the $7 price looked like it was firm.