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.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Big week for Alzheimer’s da­ta; As­traZeneca buys cell ther­a­py start­up; Dig­i­tal ther­a­peu­tics hits a pay­er wall; and more

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Am­gen, years be­hind ri­vals, says PhI obe­si­ty drug shows dura­bil­i­ty signs

While NBC ran “The Biggest Loser” for 17 seasons, deemed toxic by critics for the reality show’s punishing exercise and diet upheavals, researchers in pharmaceutical labs have been attempting to create prescription drugs that induce weight loss — and one pharma betting it can require less frequent dosing is out with a new crop of data.

Amgen was relatively late to the game compared to its approved competitor Novo Nordisk and green light-approaching rival Eli Lilly. But early data suggested Amgen’s AMG 133 led to a 14.5% weight reduction in the first few months of dosing, buoying shares earlier this fall, and now the California pharma is out with its first batch of durability data showing that figure fell slightly to 11.2% about 150 days after the last dose. Amgen presented at the 20th World Congress on Insulin Resistance, Diabetes & Cardiovascular Disease on Saturday afternoon.

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Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls

Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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Matt Gline, Roivant Sciences CEO (Photo by John Sciulli/Getty Images for GLG)

Pfiz­er and Roivant team up again for an­oth­er 'Van­t', set­ting up an­ti-in­flam­ma­to­ry show­down with Prometheus

Pfizer and Roivant are teaming up to launch a new ‘Vant’ aimed at bringing a mid-stage anti-inflammatory drug to market, the pair announced Thursday.

There’s no name for the startup yet, nor are there any employees. Thus far, the new company and Roivant can be considered “one and the same,” Roivant CEO Matt Gline tells Endpoints News. But Pfizer is so enthusiastic about the target that it elected to keep 25% of equity in the drug rather than take upfront cash from Roivant, Gline said.

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US month­ly costs for biosim­i­lars 'sub­stan­tial­ly high­er' than Ger­many or Switzer­land, JA­MA re­search finds

As the global biologics market is expected to hit nearly the half-trillion-dollar mark this year, new JAMA research points to the importance of timely biosimilar entry, particularly as fewer biosimilars are entering the US than in Europe, and as monthly treatment costs for biosimilars were “substantially higher” in the US compared with Germany and Switzerland.

Among the three countries, biosimilar market share at launch was highest in Germany, but increased at the fastest rate in the US, the authors from the University of Zurich’s Institute of Law wrote in JAMA Network Open today.

Kirk Myers is shown in a still image from a new film series showcasing the efforts of HIV advocates funded by Gilead.

Gilead spot­lights HIV projects and the com­mu­ni­ty lead­ers dri­ving them in new mi­ni-doc­u­men­tary films

Gilead is going behind the scenes of some of the HIV initiatives it funds through grants in a new film series narrated by the people helming the projects.

The first four films and leaders come from across the US — Arianna Lint in Florida and Puerto Rico, Cleve Jones in San Francisco, June Gipson in Mississippi and Kirk Myers in Texas. Their HIV-focused efforts range from addressing unmet needs of the transgender community to delivering social services and high-quality health care in underserved communities.

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EMA pulls an opi­oid from the 1950s used to treat dry cough

The European Medicines Agency said Friday that it’s pulling from all European markets pholcodine-containing medicines, which are an opioid used in adults and children for the treatment of dry cough and in combo with other drugs as a treatment for cold and flu.

The decision to pull the medicines comes as the EMA points to the results from the recent ALPHO study, which show that use of pholcodine during the 12 months preceding anesthesia is linked to a risk of an anaphylactic reaction related to the neuromuscular blocking agents (NMBAs) used (with an adjusted OR of 4.2, and a 95% confidence interval of 2.5 to 6.9).