Black­stone fi­nal­izes R&D pact with Al­ny­lam as part of ear­li­er $2 bil­lion in­vest­ment

Back in April, Black­stone an­nounced a mas­sive, multi­bil­lion dol­lar in­vest­ment in Al­ny­lam Phar­ma­ceu­ti­cals, but not all the R&D de­tails were fi­nal­ized. On Mon­day, we got a glimpse at how some of those de­tails end­ed up look­ing.

The new­ly-ce­ment­ed R&D agree­ment calls for $150 mil­lion to be paid to Al­ny­lam for the de­vel­op­ment of two pipeline prod­ucts, vutrisir­an and ALN-AGT. $70 mil­lion will go to­ward sup­port­ing Phase III vutrisir­an tri­als in AT­TR amy­loi­do­sis-re­lat­ed car­diomy­opa­thy, while the oth­er $80 mil­lion will ad­vance Phase II and III stud­ies for ALN-AGT in hy­per­ten­sion ther­a­py.

Mon­day’s cash is part of the $2 bil­lion fund­ing Black­stone Life Sci­ences doled out to Al­ny­lam’s RNAi ma­chin­ery a few months ago. The deal was ul­ti­mate­ly struc­tured in four parts: $1 bil­lion for roy­al­ties on in­clisir­an, up to $750 mil­lion in loans and $100 mil­lion in eq­ui­ty in­vest­ment, in ad­di­tion to Mon­day’s $150 mil­lion for the R&D fund.

Yvonne Green­street

Al­ny­lam COO Yvonne Green­street al­so said at the time Black­stone will re­ceive de­vel­op­ment and reg­u­la­to­ry mile­stones over 2 to 4 years on both pro­grams and a 1% roy­al­ty on world­wide vutrisir­an sales.

In­clisir­an is cur­rent­ly un­der re­view by the FDA and ex­pect­ed to be ap­proved at some point this year. The drug works sim­i­lar­ly to PC­SK9 in­hibitors pa­tients cur­rent­ly take to treat high cho­les­terol, but rather than block the pro­tein, it us­es RNA in­ter­fer­ence to pre­vent the body from pro­duc­ing it. Cortel­lis an­a­lysts have pre­dict­ed peak sales to reach $1.16 bil­lion.

Short­ly af­ter the April deal was dis­closed, vutrisir­an al­so re­ceived fast track des­ig­na­tion from the FDA in a sep­a­rate in­di­ca­tion — polyneu­ropa­thy of hAT­TR amy­loi­do­sis. At the time, Black­stone hint­ed it would help sup­port those tri­als but not re­ceive roy­al­ties. Phase III re­sults for this study are ex­pect­ed in ear­ly 2021.

Black­stone’s in­vest­ment comes as part of the largest-ever life sci­ences fund, in which it raised a to­tal of $4.6 bil­lion. The deal with Al­ny­lam makes up the biggest chunk of that fund. Ex-Sanofi CEO Olivi­er Brandi­court, who now works as a se­nior ad­vi­sor at Black­stone, joined Al­ny­lam’s board in Feb­ru­ary but the in­vest­ment firm said at the time he wasn’t in­volved in the trans­ac­tion.

Sanofi and Al­ny­lam had worked to­geth­er pre­vi­ous­ly, but they cut off ties in May 2019. The two broke it off as Al­ny­lam built on an ex­ist­ing col­lab­o­ra­tion with Re­gen­eron, net­ting Al­ny­lam $1 bil­lion-plus. Re­gen­eron ac­quired an ex­clu­sive re­la­tion­ship with Al­ny­lam on treat­ments for eye and CNS dis­eases, while Al­ny­lam got ac­cess to Re­gen­eron’s ge­net­ics re­search.

Thus far, Black­stone has al­so com­mit­ted $350 mil­lion to Rea­ta on its late-stage work for Al­port syn­drome treat­ment, and an­oth­er $337 mil­lion to Medtron­ic for di­a­betes R&D.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

David Baker working with a student on their protein design (Jason Mast)

Sci­en­tists are fi­nal­ly learn­ing how to de­sign pro­teins from scratch. Drug de­vel­op­ment may nev­er be the same

SEATTLE — It’s a cloudy Thursday afternoon in mid-July and David Baker is reclining into the futon in his corner office at the University of Washington, arms splayed out like a daytime talk show host as he coaches another one of his postdocs through the slings and arrows of scientific celebrity.

“Be jealous of your time,” he says, before plotting ways of sneaking her out of Zooms. “It’s this horrible cost to science that you’re tied up in some stupid meeting.”

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Pre­sent­ing a live End­points News event: Man­ag­ing a biotech in tur­bu­lent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

Amidst R&D reshuf­fle, Ver­tex ex­pands its pres­ence in Boston, aim­ing to be­come num­ber one

Vertex Pharmaceuticals has been one of the buzzier names in the bustling Boston biotech scene, but now the company is looking to vault to number one status — at least in terms of physical footprint.

At a ribbon cutting on Tuesday for its new Jeffrey Leiden Center for Cell and Genetic Therapies at the Boston Seaport, Vertex announced it would embark on a new project: The company will build a 344,000 square foot facility in the seaport to accommodate the company’s growing R&D needs, especially in its cell and gene therapies program.

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Patty Murray, D-WA (Graeme Sloan/Sipa USA)(Sipa via AP Images)

Sen­ate user fee reau­tho­riza­tion bill omits ac­cel­er­at­ed ap­proval re­forms, shows wide gaps with House ver­sion

The Senate health committee on Tuesday released its first version of the bill to reauthorize all the different FDA user fees. But unlike the House version, there are only a few controversial items in the Senate’s version, which does not address either accelerated approval reforms or clinical trial diversity (as the House did).

While it’s still relatively early in the process of finalizing this legislation (the ultimate statutory deadline is the end of September), the House and Senate, at least initially, appear to be starting off in different corners on what should be included.

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Warren Buffett, Berkshire Hathaway CEO

Berk­shire Hath­away pulls out of Ab­b­Vie, Bris­tol My­ers Squibb in­vest­ments

It looks like Warren Buffett is sticking to ice cream and railroads for the moment.

The billionaire CEO of Berkshire Hathaway backed out of two major holdings in the pharma industry, Forexlive first reported, including a $410 million investment in AbbVie and a $324.4 million stake in Bristol Myers Squibb.

The move comes after Berkshire abandoned its Teva shares just last quarter, Bloomberg reported.

Long-ex­pect­ed UK lay­offs im­mi­nent for No­var­tis fol­low­ing sale

Nearly a year ago, more than 200 workers at Novartis’ Grimsby, UK, facility were able to hang on to their jobs after the pharma closed a Switzerland site as a part of its workforce restructuring plan. Now, it looks like those employees’ time is up, as the site has been sold, Grimsby Telegraph reported today.

The manufacturing site has been sold to Humber Industrials, a subsidiary of International Process Plants. None of the current staff members will be working with the new owners, however.

Cheng Zhang, CEO of Character Biosciences (Character)

Ge­nomics biotech un­der­goes a facelift, re­brand­ing from Clover to Char­ac­ter Bio­sciences

Though Clover Therapeutics has a new identity, CEO Cheng Zhang is making one thing clear: the genomics outfit’s goals and mission remain the same. Simply put, “we’re doubling down on it,” Zhang tells Endpoints News.

The San Francisco biotech, originally focused on analyzing reams of healthcare and imaging data to tackle “diseases of aging,” announced its new name late Tuesday morning: Character Biosciences. On top of that, the biotech announced $18 million in new funding via a Series A round.

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Clay Siegall (Photo by Dimitrios Kambouris/Getty Images for Gabrielle's Angel Foundation)

UP­DAT­ED: Clay Sie­gall re­signs from Seagen amid in­ves­ti­ga­tion in­to do­mes­tic vi­o­lence claims

A week after Seagen revealed that longtime CEO Clay Siegall was on leave due to an allegation of domestic violence, he has resigned.

Since that shocking revelation, more details about the claims have emerged into the public eye. As Endpoints News reported, Siegall was arrested on April 23. A police report about that night and a subsequent temporary restraining order described a pattern of abusive behavior against his wife and a physical altercation that left her with multiple bruises. Siegall denied the claims.

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