Roche scores an un­usu­al ‘break­through’ at the FDA for an autism drug

The team at Roche’s Basel-based un­der­dog R&D group pRED has some­thing to cheer about to­day. The FDA has des­ig­nat­ed their autism drug RG7314 (balo­vap­tan) as a “break­through” ther­a­py wor­thy of VIP treat­ment at the agency.

John Reed

Over­shad­owed by Genen­tech (gRED) over the past 9 years, Roche $RHH­BY has been pump­ing mon­ey in­to the Basel group for years, with lit­tle to show for it af­ter a ma­jor re­struc­tur­ing that left John Reed in charge.

The FDA, though, says that balo­vap­tan — a V1A va­so­pressin re­cep­tor an­tag­o­nist — had of­fered enough hu­man and an­i­mal da­ta to demon­strate its po­ten­tial for help­ing im­prove the so­cial be­hav­ior of peo­ple with autism spec­trum dis­or­ders.

Autism in gen­er­al has been a hard tar­get in drug R&D, one of many tough chal­lenges in the neu­ro­sciences field, where Roche main­tains a ma­jor com­mit­ment af­ter many of its big phar­ma peers bowed out.

In its pipeline out­line Roche says it ex­pects to file for an ap­proval some­time around 2020 or lat­er. A break­through drug des­ig­na­tion may help ac­cel­er­ate that process as reg­u­la­tors main­tain their com­mit­ment to hur­ry­ing along drugs with great promise. Roche’s pRED, though, is not known for rapid de­vel­op­ment pro­grams. This drug was in Phase II back in 2014, when Roche an­nounced its lat­est set­backs in neu­ro­sciences with a fail­ure for Frag­ile X.

Part club, part guide, part land­lord: Arie Bellde­grun is blue­print­ing a string of be­spoke biotech com­plex­es in glob­al boom­towns — start­ing with Boston

The biotech industry is getting a landlord, unlike anything it’s ever known before.

Inspired by his recent experiences scrounging for space in Boston and the Bay Area, master biotech builder, investor, and global dealmaker Arie Belldegrun has organized a new venture to build a new, 250,000 square foot biopharma building in Boston’s Seaport district — home to Vertex and a number of up-and-coming biotech players.

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Novotech CRO Ex­pands Chi­na Team as Biotech De­mand for Clin­i­cal Tri­als In­creas­es up to 79%

An increase in demand of up to 79% for clinical trials in China has prompted Novotech the Asia-Pacific CRO to rapidly expand the China team, appointing expert local clinical executives to their Shanghai and Hong Kong offices. The company is planning to expand their team by 30% over the next quarter.

Novotech China has seen considerable demand recently which is borne out by research from GlobalData:
A global migration of clinical research is occurring from high-income countries to low and middle-income countries with emerging economies. Over the period 2017 to 2018, for example, the number of clinical trial sites opened by biotech companies in Asia-Pacific increased by 35% compared to 8% in the rest of the world, with growth as high as 79% in China.
Novotech CEO Dr John Moller said China offers the largest population in the world, rapid economic growth, and an increasing willingness by government to invest in research and development.
Novotech’s 23 years of experience working in the region means we are the ideal CRO partner for USA biotechs wanting to tap the research expertise and opportunities that China offers.
There are over 22,000 active investigators in Greater China, with about 5,000 investigators with experience on at least 3 studies (source GlobalData).

H1 analy­sis: The high-stakes ta­ble in the biotech deals casi­no is pay­ing out some record-set­ting win­nings

For years the big trend among dealmakers at the major players has been centered on ratcheting down upfront payments in favor of bigger milestones. Better known as biobucks for some. But with the top 15 companies competing for the kind of “transformative” pacts that can whip up some excitement on Wall Street, with some big biotechs like Regeneron now weighing in as well, cash is king at the high stakes table.

We asked Chris Dokomajilar, the head of DealForma, to crunch the numbers for us, looking over the top 20 deals for the past decade and breaking it all down into the top alliances already created in 2019. Gilead has clearly tipped the scales in terms of the coin of the bio-realm, with its record-setting $5 billion upfront to tie up to Galapagos’ entire pipeline.

Dokomajilar notes:

We’re going to need a ‘three comma club’ for the deals with over $1 billion in total upfront cash and equity. The $100 million-plus club is getting crowded at 164 deals in the last decade with new deals being added towards the top of the chart. 2019 already has 14 deals with at least $100 million in upfront cash and equity for a total year-to-date of over $9 billion. That beats last year’s $8 billion and sets a record.

Add upfronts and equity payments and you get $11.5 billion for the year, just shy of last year’s record-setting $11.8 billion.

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Takeda's trans­la­tion­al cell ther­a­py group revs up for a race to the clin­ic with off-the-shelf CAR-T

Four years after Takeda launched a wide-ranging induced pluripotent stem cell project with the researchers at Nobel prize-winning Shinya Yamanaka’s lab at the University of Kyoto, the pharma company is taking delivery of the first of what it hopes will be a whole pipeline of iPS cell-derived therapies that can deliver on the promise of off-the-shelf CAR-T therapies.

From here, Stefan Wildt — the head of pharma sciences and translational cell therapy at Takeda — and his group of 100-plus scientists will be charged with steering their way to the clinic as they build out the manufacturing and support work for this pipeline-in-the-making. 

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Hal Barron [File photo]

Hal Bar­ron's team at GSK scores a win with pos­i­tive Ze­ju­la PhI­II front­line study — now comes the hard part

Score one for Hal Barron and the new R&D team steering GlaxoSmithKline’s pipeline.

The pharma giant reported this morning that its recently acquired PARP, Zejula (niraparib), hit the primary endpoint on progression-free survival in a frontline maintenance setting for women suffering ovarian cancer — following chemo and regardless of their BRCA status.

GSK bet $5 billion on the Tesaro buyout primarily to get this drug, drawing the shaking heads of biopharma. Why pay a big premium for a drug like this when AstraZeneca was going from strength to strength with Lynparza, ran the argument, having won a hugely important accelerated approval to jump out ahead — way ahead — of the rest of the PARP players? Lynparza — now co-owned by a powerhouse cancer team at Merck — won the first approval in frontline maintenance in ovarian cancer.

Bill Haney, Skyhawk

Genen­tech inks its lat­est dis­cov­ery deal — this time turn­ing to RNA up­start Sky­hawk

Roche’s Genentech has just completed the latest in a string of discovery deals with a lineup of much, much smaller companies looking to do new things in drug development.

Right on the heels of the news of Sosei’s coup bringing Genentech on board as a partner for its GPCR platform, the prolific dealmakers at Skyhawk — run by Bill Haney — have just added the storied biotech subsidiary as its latest partner in the world of drugging RNA.

AM-Phar­ma gath­ers €116M for PhI­II pro­gram on kid­ney drug Pfiz­er passed on

Back in 2015, Pfizer put a spotlight on Dutch biotech AM-Pharma and its drug candidate for sepsis-associated acute kidney injury, anteing up $87.5 million for a minority stake in addition to an option to gobble the whole operation up. The Phase II trial that the giant partner had bet on ended up delivering a mixed win, and Pfizer walked away from the deal citing “internal strategic reasons” — leaving the small player on its own to pull off a large Phase III study and ponder commercialization.

Pe­ter Kolchin­sky and Raj Shah raise a $300M fund de­vot­ed to biotech star­tups

Peter Kolchinsky and Raj Shah have another $300 million-plus to play with on the biotech venture side of their investment business. 

The two announced Monday morning that they’ve put together their first pure-play venture fund at RA Capital Management, which has been known to bet on just about every angle in healthcare investing — from rounds to follow-on investments at public companies. This new fund of theirs arrives well into a go-go era of new startup financing, with a particular focus on building new biotechs.

Boehringer buys Swiss biotech in its lat­est M&A deal, go­ing the next-gen can­cer vac­cine route

Boehringer Ingelheim has snapped up a Swiss biotech startup and added their group as a new platform for the oncology pipeline. 

The German biopharma company has bagged Geneva-based AMAL Therapeutics, paying out an unspecified upfront in a $358 million deal — cash, milestones and everything else, all in. Plus there’s 100 million euros on the line for commercial milestones.

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