Biotech M&A is back. Ipsen set to fork out up to $1.3B for rare dis­ease spe­cial­ist Clemen­tia

Merg­er Mon­day is back in full swing. Along with Roche buy­ing Spark Ther­a­peu­tics $ONCE, Parisian biotech Ipsen (Eu­ronext: $IPN) is beef­ing up its rare dis­ease ar­se­nal by ac­quir­ing Cana­da’s Clemen­tia Phar­ma­ceu­ti­cals, less than two years af­ter the Mon­tre­al-based drug de­vel­op­er’s $120 mil­lion IPO.

Un­der the terms of the deal, Ipsen will pay $25 per Clemen­tia share — a rough­ly 68% pre­mi­um to its Fri­day close — for a to­tal of $1.04 bil­lion, in ad­di­tion to an­oth­er $6/share if Clemen­tia’s lead ex­per­i­men­tal drug, palo­varotene, can be used to treat a rare bone dis­ease called mul­ti­ple os­teo­chon­dro­mas (MO), which could trans­late to an­oth­er $263 mil­lion.

Clemen­tia’s shares $CM­TA sky­rock­et­ed 73.5% in pre­mar­ket trad­ing.

Palo­varotene has been re­pur­posed by Clemen­tia for a pair of rare dis­eases, af­ter Roche failed to show the retinoic acid re­cep­tor gam­ma (RARγ) se­lec­tive ag­o­nist could work against em­phy­se­ma, a form of chron­ic ob­struc­tive pul­monary dis­ease (COPD).

The Swiss drug­mak­er had plans to shelve the drug, but re­searchers un­der­scored the po­ten­tial of the class of drugs in pre­vent­ing bone for­ma­tion and fi­bro­sis (retinoic acid sig­nal­ing is be­lieved to play a role in mus­cle cell gen­er­a­tion). Clemen­tia took the drug off Roche’s shelf, for two bone dis­or­ders: fi­brodys­pla­sia os­si­f­i­cans pro­gres­si­va (FOP) and MO.

A mar­ket­ing ap­pli­ca­tion for the drug to treat episod­ic flare-ups as­so­ci­at­ed with FOP is ex­pect­ed to be sub­mit­ted to the FDA in the sec­ond half of this year — and if ap­proved the drug is set for launch mid-2020. For the FOP in­di­ca­tion, the FDA has laid out a red car­pet for the drug, with or­phan drug des­ig­na­tion, break­through ther­a­py sta­tus, fast track sta­tus and rare pe­di­atric dis­ease des­ig­na­tion, so the odds are in its fa­vor for an ac­cel­er­at­ed re­view, if the NDA is ac­cept­ed.

In an ini­ti­a­tion note pub­lished last month Baird an­a­lysts cheered the po­ten­tial of the drug: “(With) strik­ing ef­fi­ca­cy and a po­ten­tial ac­cel­er­at­ed NDA fil­ing in an ul­tra-rare con­di­tion, we have con­vic­tion that pa­lo can dom­i­nate the FOP ther­a­peu­tic land­scape.”

A Phase III study test­ing the treat­ment as a chron­ic dos­ing reg­i­men for FOP, a Phase II tri­al for MO, and a Phase I tri­al for dry eye dis­ease are al­so on­go­ing.

In a note on Mon­day, Leerink’s Joseph Schwartz said the on­go­ing phase III study test­ing palo­varotene in FOP pa­tients could fur­ther demon­strate and sup­ple­ment its ef­fi­ca­cy in the pa­tient pop­u­la­tion, es­ti­mat­ing peak sales of more than $400 mil­lion for the in­di­ca­tion by 2030.

Schwartz ex­pects in­ter­im da­ta from on­go­ing MO study in pe­di­atric pa­tients by the end of next year, and topline da­ta by 2021. On the ba­sis of a “sig­nif­i­cant­ly high­er pro­ject­ed preva­lence of MO”, he fore­cast peak sales of over $1 bil­lion by 2030.

The deal is ex­pect­ed to be con­sum­mat­ed in the sec­ond quar­ter, and will have a lim­it­ed di­lu­tive im­pact on Ipsen’s core op­er­at­ing mar­gin for 2019 and 2020, the com­pa­ny said on Mon­day.

'Our lega­cy mat­ter­s': Mer­ck maps out Keytru­da king­dom while spot­light­ing ad­vances in vac­cines, hos­pi­tal care

“You can for the mo­ment stop tak­ing notes. You can put down your pens and your pad. I have no slides. I have no sub­stan­tive da­ta. I have no pitch.”

So be­gan Roger Perl­mut­ter’s brief ap­pear­ance on­stage at Mer­ck’s first in­vestor day in five years, where he dived in­to the com­pa­ny’s his­to­ry dat­ing back to 1933. The first em­ploy­ees at Mer­ck Re­search Lab­o­ra­to­ries, hand­picked by founder George W. Mer­ck, were crit­i­cal to Mer­ck’s abil­i­ty to achieve clin­i­cal and com­mer­cial suc­cess.

Eli Casdin, Casdin Capital

Eli Cas­din backs Codex­is' plat­form tech with $50M eq­ui­ty buy

About a month af­ter Codex­is notched a deal with No­var­tis $NVS, the Cal­i­for­nia com­pa­ny $CDXS on Thurs­day said long-time in­vestor Cas­din Cap­i­tal is putting up $50 mil­lion in a pri­vate place­ment, which puts the New York-based in­vest­ment firm in con­trol of more than 5% of the pro­tein en­gi­neer­ing play­er’s stock.

Eli Cas­din start­ed his epony­mous in­vest­ment firm in 2012 and dates his re­la­tion­ship with Codex­is back to at least a decade. About three years ago, Cas­din Cap­i­tal be­gan in­vest­ing in the in­dus­tri­al biotech com­pa­ny, af­ter it piv­ot­ed its fo­cus to the life sci­ences — un­der the aus­pices of new chief John Nicols — away from the en­er­gy in­dus­try.

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.

In­vestors pony up $476M for the lat­est round of biotech IPOs to hit the Street

Three biotechs — and a genome se­quenc­ing play­er — have caught the lat­est tide to the Gold Coast of IPOs, round­ing out the first half of 2019 with 23 new drug de­vel­op­ers mak­ing it on Nas­daq.

Most of these com­pa­nies filed their IPOs al­most si­mul­ta­ne­ous­ly, though we’re still wait­ing on word of fel­low class­mate Bridge­Bio’s pric­ing af­ter CEO Neil Ku­mar set the terms at $14 to $16 a share on Mon­day in search of a $240 mil­lion (or so) wind­fall. If he’s suc­cess­ful, that would take the one-week haul past the $700 mil­lion mark, a fresh sign that in­vestors’ en­thu­si­asm for new­ly coined pub­lic biotechs hasn’t cooled.

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.

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.

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.

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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.
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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:

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