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

John Hood [file photo]

UP­DATE: Cel­gene and the sci­en­tist who cham­pi­oned fe­dra­tinib's rise from Sanofi's R&D grave­yard win FDA OK

Six years after Sanofi gave it up for dead, the FDA has approved the myelofibrosis drug fedratinib, now owned by Celgene.

The drug will be sold as Inrebic, and will soon land in the portfolio at Bristol-Myers Squibb, which is finalizing a deal to acquire Celgene.

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UP­DAT­ED: AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

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Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

The top 10 fran­chise drugs in bio­phar­ma his­to­ry will earn a to­tal of $1.4T (tril­lion) by 2024 — what does that tell us?

Just in case you were looking for more evidence of just how important Amgen’s patent win on Enbrel is for the company and its investors, EvaluatePharma has come up with a forward-looking consensus estimate on what the list of top 10 drugs will look like in 2024.

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ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

ICER has some strong words for PTC, Sarepta and the FDA as the US drug price watchdog concludes that as currently priced, their respective new treatments for Duchenne muscular dystrophy are decidedly not cost-effective.

The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.

The key dates for KRAS watch­ers through the end of the year — the trail is nar­row and risks are ex­treme

There’s nothing quite like a big patent win when it comes to burnishing your prospects in the pipeline. And for Amgen, which seems to have rescued Enbrel for a run to 2029, the cheering section on Wall Street is now fixed on AMG 510 and a key rival.

And it didn’t take much data to do it. 

There was the first snapshot of a handful of patients, with a 50% response rate. Then came word that Amgen researchers are also tracking responses in different cancers, at least one in colorectal cancer and appendiceal too. 

Bain's Or­ly Mis­han joins Pfiz­er's neu­ro spin­out Cerev­el; On­colyt­ic virus biotech taps Sil­la­Jen ex­ec He­le­na Chaye as CEO

→ Bain Capital is deploying one of its top investors to Cerevel Therapeutics, steering a $350 million-plus neuro play carved out of Pfizer. Orly Mishan — a co-founder and principal of Bain’s life sciences unit — was involved in the partnership that birthed the biotech spinout in the first place. As Cerevel’s first chief business officer, she is tasked with corporate development, program management as well as technical operations. 

UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

The executive team at Alector $ALEC has a bone to pick with scientific co-founder Asa Abeliovich. Their latest quarterly rundown has this brief note buried inside:

On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

There’s no explicit word in the filing on what kind of confidential info was involved, but the proceeding got started 2 days ahead of Abeliovich’s IPO.

Abeliovich, formerly a tenured associate professor at Columbia, is a top scientist in the field of neurodegeneration, which is where Alector is targeted. More recently, he’s also helped start up Prevail Therapeutics as the CEO, which raised $125 million in an IPO. And there he’s planning on working on new gene therapies that target genetically defined subpopulations of Parkinson’s disease. Followup programs target Gaucher disease, frontotemporal dementia and synucleinopathies.

But this time Abeliovich is the CEO rather than a founding scientist. And some of their pipeline overlaps with Alector’s.

Abeliovich and Prevail, though, aren’t taking this one lying down.

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Chi­na has be­come a CEO-lev­el pri­or­i­ty for multi­na­tion­al phar­ma­ceu­ti­cal com­pa­nies: the trend and the im­pli­ca­tions

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.