Lyn­parza se­cures third EU nod; Pro­tein degra­da­tion biotech grabs $16M launch round from Chi­nese VCs; Wave shares tum­ble on tri­al de­lay

Lyn­parza, the first PARP in­hibitor from As­traZeneca $AZN to win US ap­proval in 2014, has se­cured its third EU ap­proval. The drug won the EMA nod for use pa­tients with germline BR­CA1/2-mu­ta­tions and who have hu­man epi­der­mal growth fac­tor re­cep­tor 2 (HER2)-neg­a­tive lo­cal­ly ad­vanced or metasta­t­ic breast can­cer, part­ner Mer­ck $MRK said on Wednes­day.

→ A new pro­tein degra­da­tion biotech has raised a $16 mil­lion launch round from a pair of Chi­nese ven­ture groups. San Diego-based Cull­gen says it plans to put the mon­ey to use on its tech plat­form us­ing ubiq­ui­tin-me­di­at­ed, small mol­e­cule-in­duced pro­tein degra­da­tion to go af­ter can­cer, in­flam­ma­to­ry, and au­toim­mune dis­eases. Se­quoia Cap­i­tal Chi­na and High­light Cap­i­tal pro­vid­ed the cash. “Since our found­ing in ear­ly 2018 we have made sig­nif­i­cant ad­vance­ment of our tar­get­ed pro­tein degra­da­tion plat­form which has di­rect­ly led to the iden­ti­fi­ca­tion of sev­er­al pre-clin­i­cal as­sets that we are rapid­ly ad­vanc­ing to­wards the clin­ic,” notes Ying Luo, chair­man and pres­i­dent of Cull­gen.

→ Shares of Wave Life Sci­ences $WVE tum­bled 15% Wednes­day morn­ing af­ter the biotech re­vealed that it has to de­lay a top-line read­out from their Phase Ib/IIa study of 2 new Hunt­ing­ton’s dis­ease ther­a­pies un­til the end of the year. Re­searchers blamed the de­lay on slow­er than ex­pect­ed pa­tient re­cruit­ment.

→ Pfiz­er $PFE has joined forces with Con­cer­to HealthAI to col­lab­o­rate on us­ing re­al world da­ta to help de­vel­op as well as use can­cer ther­a­pies. Con­cer­to gath­ers da­ta from elec­tron­ic health records and in­sur­ance claims to cre­ate their data­base. This is the sec­ond new pact for Con­cer­to in a mat­ter of days, fol­low­ing an ear­li­er al­liance with Bris­tol-My­ers Squibb $BMY.

→ The transPa­cif­ic biotech Edi­GENE has raised $15 mil­lion in the first tranche of its B round. The com­pa­ny is work­ing with CRISPR on new gene edit­ing pro­grams for ge­net­ic dis­eases caused by mal­func­tion­ing pro­teins. UTokyo In­no­va­tion Plat­form joined a syn­di­cate that in­cludes SBI In­vest­ment, Fast Track Ini­tia­tive, SM­BC Ven­ture Cap­i­tal, Mizuho Cap­i­tal, CareNet Group. The com­pa­ny says it has de­vel­oped CRISPR-GNDM (Guide Nu­cleotide Di­rect­ed Mod­u­la­tion) to nor­mal­ize lev­els of gene ex­pres­sion with­out cut­ting the DNA or RNA. 

Roger Perlmutter. Merck via webcast

'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.

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.

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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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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.

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.