France's NASH con­tender Gen­fit leaps on­to Nas­daq with $135M IPO

As it gears up for the late-stage read­out of its lead ex­per­i­men­tal NASH drug, France’s Gen­fit is vault­ing on­to the Nas­daq on Wednes­day with a $135 mil­lion IPO un­der the sym­bol $GN­FT.

The Lille-based com­pa­ny, which is al­ready list­ed on Eu­ronext Paris al­so as $GN­FT, spelled out the terms of the of­fer­ing on Tues­day.

The com­pa­ny de­cid­ed it want­ed a US list­ing to ex­pand its base in Boston, ac­cess the US tal­ent pool — con­sid­er­ing the ge­og­ra­phy is go­ing to be a large mar­ket for the drug (elafi­bra­nor) — as well a forge a clos­er con­nec­tion with US in­vestors, Gen­fit COO Dean Hum told End­points News.

NASH, which is typ­i­cal­ly as­so­ci­at­ed with obe­si­ty and di­a­betes, is set to eclipse he­pati­tis C as the lead­ing rea­son for liv­er trans­plants by 2020.

Gen­fit is of­fer­ing 6.15 mil­lion Amer­i­can De­posi­tary Shares priced $20.32 per ADS, and has con­cur­rent­ly se­cured a pri­vate place­ment of 500,000 or­di­nary shares in Eu­rope at €18.00 per share. In to­tal, the to­tal of­fer­ing is val­ued at rough­ly $135.1 mil­lion, be­fore com­mis­sions and ex­pens­es.

Gen­fit’s fu­ture hinges on the reg­u­la­to­ry fate of elafi­bra­nor for two main liv­er dis­or­ders: NASH and pri­ma­ry bil­iary cholan­gi­tis (PBC).

The big­ger, more lu­cra­tive in­di­ca­tion is NASH, an un­treat­ed fat­ty liv­er dis­ease that has rav­aged the de­vel­oped world and sparked a flur­ry of drug de­vel­op­ment from bio­phar­ma firms big and small. It is char­ac­ter­ized by a buildup of ex­cess fat in the liv­er that in­duces chron­ic in­flam­ma­tion and even­tu­al­ly cul­mi­nates in scar­ring that can lead to cir­rho­sis, liv­er fail­ure, can­cer and death.

While oth­er ma­jor NASH con­tenders — Gilead $GILD (fail in Phase III) and In­ter­cept $ICPT (mixed win in Phase III) — have dis­closed the top line num­bers of their late-stage tri­als, Gen­fit is ex­pect­ed to come out with its Phase III in­ter­im re­sults by the end of 2019.

When com­pared to its main ri­val, In­ter­cept, Hum as­sert­ed that elafi­bra­nor’s po­ten­tial in NASH res­o­lu­tion and re­duc­ing CV risk along with a be­nign safe­ty and tol­er­a­bil­i­ty pro­file set it apart.

Late-stage da­ta on In­ter­cept’s drug — obeti­cholic acid (OCA) — showed it in­duced a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in fi­bro­sis, but not NASH res­o­lu­tion, and a high­er-than-ex­pect­ed num­ber of pa­tients dropped out due to the pesky side-ef­fect of itch­ing.

“If you think about an­oth­er liv­er dis­ease, like say vi­ral he­pati­tis. The virus…dri­ves the fi­bro­sis for­ward in the liv­er. The way to treat vi­ral he­pati­tis is you get rid of the virus, and the fi­bro­sis gets bet­ter by it­self. So NASH is not any dif­fer­ent, what you want to is to treat the un­der­ly­ing cause of the on­set of pro­gres­sion of fi­bro­sis and that’s what elafi­bra­nor was able to demon­strate (in the phase IIb),” Hum said.

Up­on the read­out of OCA’s piv­otal study last month, In­ter­cept said it would ap­ply for mar­ket­ing ap­proval based on the dataset. Al­though Gen­fit’s piv­otal read­out comes at the end of the year, which will mean that its mar­ket­ing ap­pli­ca­tion (as­sum­ing the tri­al is pos­i­tive) will cer­tain­ly come af­ter In­ter­cept. But Hum is not wor­ried.

“The drug is well po­si­tioned to be­come stan­dard-of care,” he said, em­pha­siz­ing that elafi­bra­nor has the abil­i­ty to low­er CV risk (the lead­ing cause of mor­tal­i­ty in NASH pa­tients are CV events) and that its mech­a­nism of ac­tion is such that it could treat the widest NASH pa­tient pop­u­la­tion — from ad­vanced late-stage pa­tients to ear­ly-stage pa­tients with less fi­bro­sis.

Dubbed the silent dis­ease, it is hard to di­ag­nose in the ear­ly stages, mak­ing it dif­fi­cult to es­ti­mate its preva­lence, but stud­ies show that it af­flicts up to 12% of the adult pop­u­la­tion in de­vel­oped coun­tries. Al­though there are no ap­proved drugs for the dis­ease, the size of the NASH mar­ket is ex­pect­ed to cross $20 bil­lion by 2025.

“NASH by and large is the liv­er man­i­fes­ta­tion of liv­er dis­ease — it is a mul­ti­fac­to­r­i­al dis­ease with dif­fer­ent facets to it…like oth­er meta­bol­ic dis­eases — type II di­a­betes for ex­am­ple…NASH is very sim­i­lar, I think NASH will prob­a­bly be op­ti­mal­ly man­aged clin­i­cal­ly by com­bi­na­tion ther­a­py,” Hum said, adding that if ap­proved, elafi­bra­nor could eas­i­ly serve as the back­bone ther­a­py in fu­ture NASH com­bi­na­tion reg­i­mens.

Gen­fit is cur­rent­ly work­ing on get­ting its com­mer­cial op­er­a­tions ready in an­tic­i­pa­tion of the ap­proval. The com­pa­ny plans to re­tain the com­mer­cial rights to elafi­bra­nor in the key Eu­ro­pean coun­tries, and will like­ly pur­sue part­ner­ships with a glob­al phar­ma play­er in larg­er mar­kets like North and South Amer­i­ca, Hum said, adding that the com­pa­ny is in dis­cus­sions with dif­fer­ent groups about pric­ing.

“You’ve heard num­bers of $10,000 per year, here in the Unit­ed States — I think those num­bers are rea­son­able,” he said.

Mean­while, elafi­bra­nor is be­ing test­ed for use in oth­er liv­er in­di­ca­tions. In the com­ing weeks, the com­pa­ny is re­cruit­ing pa­tients for a study eval­u­at­ing the drug in pe­di­atric NASH pa­tients. Pos­i­tive mid-stage da­ta on the use of the drug in PBC — a rare and pro­gres­sive liv­er dis­ease — were an­nounced last De­cem­ber.

An­oth­er NASH hope­ful, the Mer­ck-part­nered NGM Bio­phar­ma, on Mon­day broke out the terms of its IPO. The com­pa­ny, which plans to list on the Nas­daq un­der the sym­bol $NGM, is of­fer­ing about 6.7 mil­lion shares priced be­tween $14 to $16, ac­cord­ing to the fil­ing.

The top 10 block­buster drugs in the late-stage pipeline — Eval­u­ate adds 6 new ther­a­pies to heavy-hit­ter list

Vertex comes in for a substantial amount of criticism for its no-holds-barred tactical approach toward wresting the price it wants for its commercial drugs in Europe. But the flip side of that coin is a highly admired R&D and commercial operation that regularly wins kudos from analysts for their ability to engineer greater cash flow from the breakthrough drugs they create.

Both aspects needed for success in this business are on display in the program backing Vertex’s triple for cystic fibrosis. VX-659/VX-445 + Tezacaftor + Ivacaftor — it’s been whittled down to 445 now — was singled out by Evaluate Pharma as the late-stage therapy most likely to win the crown for drug sales in 5 years, with a projected peak revenue forecast of $4.3 billion.

The latest annual list, which you can see here in their latest world preview, includes a roster of some of the most closely watched development programs in biopharma. And Evaluate has added 6 must-watch experimental drugs to the top 10 as drugs fail or go on to a first approval. With apologies to the list maker, I revamped this to rank the top 10 by projected 2024 sales, instead of Evaluate's net present value rankings.

It's how we roll at Endpoints News.

Here is a quick summary of the rest of the top 10:

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.

How small- to mid-sized biotechs can adopt pa­tient cen­tric­i­ty in their on­col­o­gy tri­als

By Lucy Clos­sick Thom­son, Se­nior Di­rec­tor of On­col­o­gy Pro­ject Man­age­ment, Icon

Clin­i­cal tri­als in on­col­o­gy can be cost­ly and chal­leng­ing to man­age. One fac­tor that could re­duce costs and re­duce bar­ri­ers is har­ness­ing the pa­tient voice in tri­al de­sign to help ac­cel­er­ate pa­tient en­roll­ment. Now is the time to adopt pa­tient-cen­tric strate­gies that not on­ly fo­cus on pa­tient needs, but al­so can main­tain cost ef­fi­cien­cy.

John Reed at JPM 2019. Jeff Rumans for Endpoints News

Sanofi's John Reed con­tin­ues to re­or­ga­nize R&D, cut­ting 466 jobs while boost­ing can­cer, gene ther­a­py re­search

The R&D reorganization inside Sanofi is continuing, more than a year after the pharma giant brought in John Reed to head the research arm of the Paris-based company.
Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.

John Chiminski, Catalent CEO - File Photo

'It's a growth play': Catal­ent ac­quires Bris­tol-My­er­s' Eu­ro­pean launch pad, ex­pand­ing glob­al CD­MO ops

Catalent is staying on the growth track.

Just two months after committing $1.2 billion to pick up Paragon and take a deep dive into the sizzling hot gene therapy manufacturing sector, the CDMO is bouncing right back with a deal to buy out Bristol-Myers’ central launchpad for new therapies in Europe, acquiring a complex in Anagni, Italy, southwest of Rome, that will significantly expand its capacity on the continent.

There are no terms being offered, but this is no small deal. The Anagni campus employs some 700 staffers, and Catalent is planning to go right in — once the deal closes late this year — with a blueprint to build up the operations further as they expand on oral solid, biologics, and sterile product manufacturing and packaging.

This is an uncommon deal, Catalent CEO John Chiminski tells me. But it offers a shortcut for rapid growth that cuts years out of developing a green fields project. That’s time Catalent doesn’t have as the industry undergoes unprecedented expansion around the world.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.

Arc­turus ex­pands col­lab­o­ra­tion, adding $30M cash; Ku­ra shoots for $100M raise

→  Rare dis­ease play­er Ul­tragenyx $RARE is ex­pand­ing its al­liance with Arc­turus $ARCT, pay­ing $24 mil­lion for eq­ui­ty and an­oth­er $6 mil­lion in an up­front as the two part­ners ex­pand their col­lab­o­ra­tion to in­clude up to 12 tar­gets. “This ex­pand­ed col­lab­o­ra­tion fur­ther so­lid­i­fies our mR­NA plat­form by adding ad­di­tion­al tar­gets and ex­pand­ing our abil­i­ty to po­ten­tial­ly treat more dis­eases,” said Emil Kakkis, the CEO at Ul­tragenyx. “We are pleased with the progress of our on­go­ing col­lab­o­ra­tion. Our most ad­vanced mR­NA pro­gram, UX053 for the treat­ment of Glyco­gen Stor­age Dis­ease Type III, is ex­pect­ed to move in­to the clin­ic next year, and we look for­ward to fur­ther build­ing up­on the ini­tial suc­cess of this part­ner­ship.”

UP­DAT­ED: Chica­go biotech ar­gues blue­bird, Third Rock 'killed' its ri­val, pi­o­neer­ing tha­lassemia gene ther­a­py in law­suit

Blue­bird bio $BLUE chief Nick Leschly court­ed con­tro­ver­sy last week when he re­vealed the com­pa­ny’s be­ta tha­lassemia treat­ment will car­ry a jaw-drop­ping $1.8 mil­lion price tag over a 5-year pe­ri­od in Eu­rope — mak­ing it the plan­et’s sec­ond most ex­pen­sive ther­a­py be­hind No­var­tis’ $NVS fresh­ly ap­proved spinal mus­cu­lar at­ro­phy ther­a­py, Zol­gens­ma, at $2.1 mil­lion. A Chica­go biotech, mean­while, has been fum­ing at the side­lines. In a law­suit filed ear­li­er this month, Er­rant Gene Ther­a­peu­tics al­leged that blue­bird and ven­ture cap­i­tal group Third Rock un­law­ful­ly prised a vi­ral vec­tor, de­vel­oped in part­ner­ship with the Memo­r­i­al Sloan Ket­ter­ing Can­cer Cen­ter (MSK), from its grasp, and thwart­ed the de­vel­op­ment of its sem­i­nal gene ther­a­py.

Neil Woodford. Woodford Investment Management via YouTube

Wood­ford braces po­lit­i­cal storm as UK fi­nan­cial reg­u­la­tors scru­ti­nize fund sus­pen­sion

The shock of Neil Wood­ford’s de­ci­sion to block with­drawals for his flag­ship fund is still rip­pling through the rest of his port­fo­lio — and be­yond. Un­der po­lit­i­cal pres­sure, UK fi­nan­cial reg­u­la­tors are now tak­ing a hard look while in­vestors con­tin­ue to flee.

In a re­sponse let­ter to an MP, the Fi­nan­cial Con­duct Au­thor­i­ty re­vealed that it’s opened an in­ves­ti­ga­tion in­to the sus­pen­sion fol­low­ing months of en­gage­ment with Link Fund So­lu­tions, which tech­ni­cal­ly del­e­gat­ed Wood­ford’s firm to man­age its funds.

Gilead baits new al­liance with $45M up­front, div­ing in­to the busy pro­tein degra­da­tion field

Gilead is jump­ing on board the pro­tein degra­da­tion band­wag­on. And they’re turn­ing to a low-pro­file Third Rock start­up for the ex­per­tise. But if you were look­ing for a trans­for­ma­tion­al deal to kick up fresh en­thu­si­asm for Gilead, you’ll have to re­main pa­tient.

This one will have a long way to go be­fore they get in­to the clin­ic.

The big biotech said Wednes­day morn­ing that it is pay­ing $45 mil­lion up­front and re­serv­ing a whop­ping $2.3 bil­lion in biotech bucks if San Fran­cis­co-based Nurix can point the way to new can­cer ther­a­pies, as well as drugs for oth­er, un­spec­i­fied dis­eases.

A new num­ber 1 drug? Keytru­da tapped to top the 10 biggest block­busters on the world stage by 2024

Analysts may be fretting about Keytruda’s longterm prospects as a host of rival therapies elbow their way to the market. But the folks at Evaluate Pharma are confident that last year’s $7 billion earner is headed for glory, tapping it to beat out the current #1 therapy Humira as AbbVie watches that franchise swoon over the next 5 years.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.