All in: Gilead maps a $3.15B gene edit­ing al­liance with Sang­amo for off-the-shelf CAR-T drugs

In a block­buster en­dorse­ment of its gene-edit­ing tech­nol­o­gy, Gilead $GILD has tapped Sang­amo’s zinc fin­ger nu­cle­ase ap­proach to tai­lor­ing its next-gen pro­grams for off-the-shelf as well as per­son­al­ized ther­a­pies. And Gilead, which seized a lead­ing spot in the CAR-T world with its ac­qui­si­tion of Kite, is fronting the deal with $150 mil­lion in cash and slight­ly more than $3 bil­lion in mile­stones.

The deal gives Gilead an ex­clu­sive po­si­tion with Sang­amo $SG­MO, which has been work­ing with its ZFN gene edit­ing tech for years. While CRISPR/Cas9 and TAL­ENs have fig­ured promi­nent­ly in gene edit­ing, with ri­val off-the-shelf CAR-T de­vel­op­er Cel­lec­tis ini­tial­ly pre­fer­ring TAL­ENs, Sang­amo has been mak­ing some dra­mat­ic ad­vances in re­cent months.

Gilead, mean­while, sig­naled with its ac­qui­si­tion of Cell De­sign that it was se­ri­ous about in­vest­ing big in CAR-T 2.0, and few pro­grams are as am­bi­tious as the move to use healthy donor cells to cre­ate a sim­pler, less ex­pen­sive al­ter­na­tive to the pricey per­son­al­ized ther­a­pies now hit­ting the mar­ket from No­var­tis and Gilead.

And it’s will­ing to con­tin­ue to in­vest heav­i­ly through the de­vel­op­ment process. In a fil­ing with the SEC to­day, Sang­amo not­ed:

Of this amount (for mile­stones), ap­prox­i­mate­ly $1.26 bil­lion re­lates to the achieve­ment of spec­i­fied re­search, clin­i­cal de­vel­op­ment and first com­mer­cial sale mile­stones, and ap­prox­i­mate­ly $1.75 bil­lion re­lates to the achieve­ment of spec­i­fied com­mer­cial sales-based mile­stones if an­nu­al world­wide net sales of Li­censed Prod­ucts reach spec­i­fied lev­els. Each de­vel­op­ment- and sales-based mile­stone pay­ment is payable (i) on­ly once for each Li­censed Prod­uct, re­gard­less of the num­ber of times that the as­so­ci­at­ed mile­stone event is achieved by such Li­censed Prod­uct, and (ii) on­ly for the first ten times that the as­so­ci­at­ed mile­stone event is achieved, re­gard­less of the num­ber of Li­censed Prod­ucts that may achieve such mile­stone event. In ad­di­tion, Sang­amo will be en­ti­tled to re­ceive es­ca­lat­ing, tiered roy­al­ty pay­ments with a per­cent­age in the sin­gle dig­its based on po­ten­tial fu­ture an­nu­al world­wide net sales of Li­censed Prod­ucts. These roy­al­ty pay­ments will be sub­ject to re­duc­tion due to patent ex­pi­ra­tion, en­try of biosim­i­lar prod­ucts to the mar­ket and pay­ments made un­der cer­tain li­cens­es for third-par­ty in­tel­lec­tu­al prop­er­ty.

Now both No­var­tis $NVS — work­ing with CRISPR tech from In­tel­lia Ther­a­peu­tics and Cari­bou Bio­sciences — and Cel­gene $CELG, which just bought out Juno, have been served no­tice that the next-gen tech race in CAR-T is in full swing. How will they re­spond?

The deal marks the sec­ond big tech en­dorse­ment for Sang­amo in a lit­tle more than a month. Pfiz­er start­ed the year by ink­ing an al­liance with the gene edit­ing crew on ALS, build­ing on their orig­i­nal pact for he­mo­phil­ia A. Pfiz­er had ear­li­er al­lied it­self with Spark in a move to get in­to the lead of the gene ther­a­py field, work­ing on he­mo­phil­ia B.

“The emer­gence of gene edit­ing as a tool to ed­it im­mune cells holds promise in the de­vel­op­ment of ther­a­pies with po­ten­tial­ly im­proved safe­ty, ef­fi­ca­cy and ef­fi­cien­cy,” said Gilead CEO John Mil­li­gan in a state­ment. “We be­lieve Sang­amo’s zinc fin­ger nu­cle­as­es pro­vide the op­ti­mal gene edit­ing plat­form, and we look for­ward to work­ing with Sang­amo to ac­cel­er­ate our ef­forts to de­vel­op next-gen­er­a­tion au­tol­o­gous cell ther­a­pies, as well as al­lo­gene­ic treat­ments that can be ac­cessed more con­ve­nient­ly in the hos­pi­tal set­ting for peo­ple liv­ing with can­cer.”

Im­age: Gilead CEO John Mil­li­gan. bloomberg via get­ty im­ages

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.

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.

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.

In­vestor day prep at Mer­ck in­cludes a new strat­e­gy to pick up the pace on M&A — re­port

Mer­ck’s re­cent deals to buy up two bolt-on biotechs — Ti­los and Pelo­ton — weren’t an aber­ra­tion. In­stead, both ac­qui­si­tions mark a new strat­e­gy to beef up its dom­i­nant can­cer drug op­er­a­tions cen­tered on Keytru­da while look­ing to ad­dress grow­ing con­cerns that too many of its eggs are in the one I/O bas­ket for their PD-1 pro­gram. And Mer­ck is go­ing af­ter more small- and mid-sized buy­outs to calm those fears.

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.

Dave Barrett, Brian Chee, Amir Nashat, Amy Schulman. Polaris

Bob Langer's first port of call — Po­laris Part­ners — maps $400M for ninth fund

Health and tech ven­ture group Po­laris Part­ners, which counts Alec­tor, Al­ny­lam and Ed­i­tas Med­i­cine as part of its port­fo­lio, is set­ting up its ninth fund, rough­ly two years af­ter it closed Po­laris VI­II with $435 mil­lion in the bank, sur­pass­ing its tar­get by $35 mil­lion.

The Boston-based firm, in an SEC fil­ing, said it in­tends to raise $400 mil­lion for the fund. Po­laris — which rou­tine­ly backs com­pa­nies mold­ed out of the work done in the lab of pro­lif­ic sci­en­tist Bob Langer of MIT  — typ­i­cal­ly in­vests ear­ly, and sticks around till com­pa­nies are in the green. Like its peers at Flag­ship and Third Rock, Po­laris is all about cham­pi­oning the lo­cal biotech scene with a steady flow of start­up cash.

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.

Right back at you, Pfiz­er: BeiGene and a Pfiz­er spin­out launch a new­co to de­vel­op a MEK/BRAF in­hibitor that could ri­val $11.4B com­bo

A day af­ter Pfiz­er bought Ar­ray and its ap­proved can­cer com­bo, BeiGene and Pfiz­er spin­out Spring­Works have part­nered in launch­ing a new biotech that has an eye on the very same mar­ket the phar­ma gi­ant just paid bil­lions for. And they’re plan­ning on us­ing an ex-Pfiz­er drug to do it.

In a nut­shell, Chi­na’s BeiGene is toss­ing in a pre­clin­i­cal BRAF in­hibitor — BGB-3245, which cov­ers both V600 and non-V600 BRAF mu­ta­tions — for a big stake in a new, joint­ly con­trolled biotech called Map­Kure with Bain-backed Spring­Works.

Sanofi aligns it­self with Google to stream­line drug de­vel­op­ment

Tech­nol­o­gy is bleed­ing in­to health­care, and big phar­ma is rid­ing the wave. Sanofi $SNY ap­point­ed its first chief dig­i­tal of­fi­cer this Feb­ru­ary, fol­low­ing the foot­steps of its peers. By May, the French drug­mak­er and some of its big phar­ma com­pa­tri­ots joined forces with Google par­ent Al­pha­bet’s Ver­i­ly unit to aug­ment clin­i­cal tri­al re­search. On Tues­day, the Parisian com­pa­ny tied up with Google to ac­cess its cloud com­put­ing and ar­ti­fi­cial in­tel­li­gence tech to spur the de­vel­op­ment of new ther­a­pies.