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

As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Sanofi brings in 4 new ex­ec­u­tives in con­tin­ued shake-up, as vac­cines and con­sumer health chief head out the door

In the middle of Sanofi’s multi-pronged race to develop a Covid-19 vaccine, David Loew, the head of their sprawling vaccines unit, is leaving – part of the final flurry of moves in the French giant’ months-long corporate shuffle that will give them new-look leadership under new CEO Paul Hudson.

The company also said today that Alan Main, the head of their consumer healthcare unit, is out, and they named 4 executives to fill new or newly vacated positions, 3 of whom come from both outside both Sanofi and from Pharma.

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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Ab­b­Vie wins an ap­proval in uter­ine fi­broid-as­so­ci­at­ed heavy bleed­ing. Are ri­vals My­ovant and Ob­sE­va far be­hind?

Women expel on average about 2 to 3 tablespoons of blood during their time of the month. But with uterine fibroids, heavy bleeding is typical — a third of a cup or more. Drugmakers have been working on oral therapies to try and stem the flow, and as expected, AbbVie and their partners at Neurocrine Biosciences are the first to make it across the finish line.

Known chemically as elagolix, the drug is already approved as a treatment for endometriosis under the brand name Orilissa. It targets the GnRH receptor to decrease the production of estrogen and progesterone.

David Chang, Allogene CEO (Jeff Rumans)

Head­ed to PhII: Al­lo­gene CEO David Chang com­pletes a pos­i­tive ear­ly snap­shot of their off-the-shelf CAR-T pi­o­neer

Allogene CEO David Chang has completed the upbeat first portrait of the biotech’s off-the-shelf CAR-T contender ALLO-501 at virtual ASCO today, keeping all eyes on a drug that will now try to go on to replace the first-wave personalized pioneers he helped create.

The overall response rate outlined in Allogene’s abstract for treatment-resistant patients with non-Hodgkin lymphoma slipped a little from the leadup, but if you narrow the patient profile to treatment-naïve patients — removing the 3 who had previous CAR-T therapy who didn’t respond, leaving 16 — the ORR lands at 75% with a 44% complete response rate. And 9 of the 12 responders remained in response at the data cutoff, offering a glimpse on durability that still has a long way to go before it can be completely nailed down.

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

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As­traZeneca’s $7B ADC suc­ceeds where Roche failed, im­prov­ing sur­vival in gas­tric can­cer

Another day, another win for Enhertu.

The antibody-drug conjugate AstraZeneca promised up-to $7 billion to partner on has had a quite a few months, beginning with splashy results in a Phase II breast cancer trial, a rapid approval and, earlier this month, breakthrough designations in both non-small cell lung cancer and gastric cancer.

Now, at ASCO, the British pharma and their Japanese partner, Daiichi Sankyo, have shown off the data that led to the gastric cancer designation, which they’ll take back to the FDA. In a pivotal, 187-person Phase II trial, Enhertu shrunk tumors in 42.9% of third-line patients with HER2-positive stomach cancer, compared with 12.5% in a control arm where doctors prescribed their choice of therapy. Progression-free survival was 5.4 months for Enhertu compared to 3.5 months for the control.

Once a gem, now just a rock, Take­da punts PhI­II IBD drug as ri­vals mus­cle ahead

Back in 2016, when then-Shire CEO Flemming Ørnskov picked up a promising clinical-stage IBD drug from Pfizer, the Boston-based biotech dubbed it SHP647 and moved it into the gem section of the pipeline, with rosy expectations of registration-worthy Phase III data ahead.

This was a drug that the EC wanted Takeda to commit to selling off before it gave their blessing to its acquisition of Shire, to settle some deep-seated concerns revolving around the potential market overlap with their blockbuster rival Entyvio. And Takeda, which took on a heavy debt load to buy Shire, clearly wanted the cash to pay down debt.