Biotech spin­out grabs $46M round, jumps straight in­to PhI­II with a drug/de­vice com­bo for rare lung con­di­tion

Jens Stege­mann, CEO

The Ger­man aerosol ther­a­py com­pa­ny PARI Phar­ma­ceu­ti­cals is spin­ning out a Phase III ef­fort to de­vel­op a drug/de­vice com­bo that match­es an in­haled for­mu­la­tion of an im­muno­sup­pres­sant with a re­mote­ly mon­i­tored de­liv­ery de­vice to treat a rare lung con­di­tion. And it’s re­cruit­ed a trio of Eu­ro­pean ven­ture groups, in­clud­ing Sofinno­va Part­ners in Paris, to back a €43.5 mil­lion (about $46 mil­lion) round to boot it up to reg­u­la­tors on both sides of the At­lantic.

The idea is sim­ple. In­ves­ti­ga­tors will use an aerosolized ver­sion of li­po­so­mal cy­closporine A, de­liv­ered through a neb­u­liz­er with re­mote mon­i­tor­ing tech­nol­o­gy from PARI to eval­u­ate its ef­fect on Bron­chi­oli­tis Oblit­er­ans Syn­drome, or BOS, a lethal or­phan res­pi­ra­to­ry dis­ease in­volv­ing lung trans­plant re­jec­tion. This ver­sion of the im­muno­sup­pres­sant is de­signed to con­cen­trate the ther­a­py in the lung, where it’s need­ed to pre­vent graft re­jec­tion.

The work is based on re­search al­ready con­duct­ed at PARI — which has been do­ing re­search work with in­haled li­po­so­mal cy­closporine A for years — along with guid­ance from lung trans­plan­ta­tion ex­pert Al­do Ia­cono at the Uni­ver­si­ty of Mary­land. The com­pa­ny will be based in Mu­nich and Frank­furt.

Gimv and Sofinno­va Part­ners co-led the round for Breath Ther­a­peu­tics and were joined by Gilde Health­care.

Graziano Seghezzi, a part­ner at Sofinno­va, says the deal fits in­to their sweet spot for new in­vest­ments. It aims straight at a crit­i­cal stage of de­vel­op­ment un­der an ex­pe­ri­enced team that has de­vel­oped da­ta on the pro­gram, some of which is about to be pub­lished. Right now, he tells me, it’s too ear­ly to say just how long it will take to com­plete Phase III, as the biotech is still “deep in con­ver­sa­tions” with the FDA. And, he adds, it’s pos­si­ble that the com­pa­ny may go on to work on ad­di­tion­al projects, some of which are cur­rent­ly un­der re­view.

PARI busi­ness de­vel­op­ment ex­ec Jens Stege­mann is tak­ing the helm as CEO of the spin­out, which plans to go straight in­to Phase III stud­ies in Eu­rope and the US, with an eye to tak­ing the piv­otal da­ta to the FDA and EMA.

Said Stege­mann:

Our strat­e­gy is to de­posit high con­cen­tra­tions of an im­muno­sup­pres­sive agent di­rect­ly in­to the small air­ways of the lung. With this top-tier group of in­vestors and the best in­ter­na­tion­al lung trans­plan­ta­tion cen­tres sup­port­ing us, we now are in an ex­cel­lent po­si­tion to achieve ground-break­ing im­prove­ments in the com­bat of BOS.

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.

Robert Forrester, Verastem

Ve­rastem CEO For­rester steps to the ex­it as the board hunts com­mer­cial-savvy ex­ec for the be­lea­guered biotech

Robert For­rester is step­ping down as CEO of Ve­rastem On­col­o­gy $VSTM just 8 months af­ter the com­pa­ny nabbed an ap­proval for du­velis­ib, a PI3K drug with a sto­ried past — and what ap­pears as not much of a fu­ture.

The biotech put out word this morn­ing that For­rester will take an ad­vi­so­ry role with Ve­rastem while COO Dan Pa­ter­son steps up to take charge of the lead­er­ship team and the board looks around for a new CEO.

Ken Frazier appears before the Senate Committee on Finance for a hearing on prescription drug pricing on Capitol Hill in Washington, DC, February 26, 2019. Chris Kleponis for CNP via AP Images

Who’s next in line to suc­ceed Ken Fra­zier as CEO of the Keytru­da-blessed Mer­ck?

When Merck waved off a looming forced retirement for Ken Frazier last September, the board cited flexibility in CEO transition as a key factor in the decision. Having Frazier — who’s also chairman of the company — around beyond his 65th birthday in 2019 would ensure they install the best person at the best time, they said.

The board has evidently begun that process with a clear preference for internal candidates, sources told Bloomberg. CFO Robert Davis, chief marketing officer Michael Nally, and chief commercial officer Frank Clyburn are all in the running, according to an insider.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,100+ biopharma pros reading Endpoints daily — and it's free.

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 53,100+ biopharma pros reading Endpoints daily — and it's free.

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.

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 53,100+ biopharma pros reading Endpoints daily — and it's free.

In starved an­tibi­ot­ic field, Melin­ta soars as FDA grants speedy drug re­view

Such is the state of af­fairs in an­tibi­ot­ic land that the FDA agree­ing to pri­or­i­ty re­view an ap­pli­ca­tion to ex­pand the use of an an­tibi­ot­ic can rock­et up a stock more than two-fold.

On Wednes­day, Melin­ta Ther­a­peu­tics said its ap­proved an­tibi­ot­ic Baxdela had been grant­ed pri­or­i­ty re­view for use in com­mu­ni­ty-ac­quired bac­te­r­i­al pneu­mo­nia (CAPB). The FDA is ex­pect­ed to make its de­ci­sion by Oc­to­ber 24. Shares of the Con­necti­cut drug­mak­er $ML­NT cat­a­pult­ed, clos­ing up near­ly 224% at $6.41.

Brent Saunders at an Endpoints News event in 2017 — File photo

An­a­lyst call with Al­ler­gan ex­ecs stokes an­tic­i­pa­tion of a plan to split the com­pa­ny in ‘a month or two’

So what’s up at Al­ler­gan?

Ear­li­er this week the ubiq­ui­tous Ever­core ISI an­a­lyst Umer Raf­fat was on the line with com­pa­ny ex­ec­u­tives to probe in­to the lat­est on the num­bers as well as CEO Brent Saun­ders’ re­cent de­c­la­ra­tion that he’d be do­ing some­thing de­fin­i­tive to help long-suf­fer­ing in­vestors who have watched their shares dwin­dle in val­ue.

He came away with the im­pres­sion that a sig­nif­i­cant com­pa­ny split is on the way. And not on some dis­tant time hori­zon.

Partners Innovation Fund

David de Graaf now has his $28.5M launch round in place, build­ing a coen­zyme A plat­form in his lat­est start­up

Long­time biotech ex­ec David de Graaf has the cash he needs to set up the pre­clin­i­cal foun­da­tion for his coen­zyme A me­tab­o­lism com­pa­ny Comet. A few high-pro­file in­vestors joined the ven­ture syn­di­cate to sup­ply Comet with $28.5 mil­lion in launch mon­ey — enough to get it two years in­to the plat­form-build­ing game, with­in knock­ing dis­tance of the clin­ic.

Canaan jumped in along­side ex­ist­ing in­vestor Sofinno­va Part­ners to co-lead the round, with par­tic­i­pa­tion by ex­ist­ing in­vestor INKEF Cap­i­tal and new in­vestor BioIn­no­va­tion Cap­i­tal.