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.

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,700+ biopharma pros reading Endpoints daily — and it's free.

Credit: Shutterstock

How Chi­na turned the ta­bles on bio­phar­ma's glob­al deal­mak­ing

Fenlai Tan still gets chills thinking about the darkest day of his life.

Three out of eight lung cancer patients who received a tyrosine kinase inhibitor developed by his company, Betta Pharma, died in the span of a month. Tan, the chief medical officer, was summoned to Peking Union Medical College Hospital, where the head of the clinical trial department told him that the trial investigators would be conducting an autopsy to see if the patients had died of the disease — they were all very sick by the time they enrolled — or of interstitial lung disease, a deadly side effect tied to the TKI class that’s been reported in Japan.

No­var­tis dumps AveX­is pro­gram for Rett syn­drome af­ter fail­ing re­peat round of pre­clin­i­cal test­ing

Say goodbye to AVXS-201.

The Rett syndrome gene therapy drug made by AveXis — the biotech that was bought, kept separate, then renamed and finally absorbed by Novartis into its R&D division — has been dropped by the biopharma.

In Novartis’ third quarter financial report, the pharma had found that preclinical data did not support development of the gene therapy into IND-enabling trials and beyond. The announcement comes a year after Novartis told the Rett Society how excited it was by the drug — and its potential benefits and uses.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,700+ biopharma pros reading Endpoints daily — and it's free.

Vas Narasimhan, Novartis CEO (Simon Dawson/Bloomberg via Getty Images)

With San­doz con­tin­u­ing to drag on No­var­tis, Vas Narasimhan says he may fi­nal­ly be ready for a sale or spin­off

After years of rehab work aimed at getting Sandoz in fighting trim to compete in a market overshadowed by declining prices, CEO Vas Narasimhan took a big step toward possibly selling or spinning off the giant generic drug player.

The pharma giant flagged plans to launch a strategic review of the business in its Q3 update, noting that “options range from retaining the business to separation.”

Analysts have been poking and prodding Novartis execs for years now as Narasimhan attempted to remodel a business that has been a drag on its performance during most of his reign in the CEO suite. The former R&D chief has made it well known that he’s devoted to the innovative meds side of the business, where they see the greatest potential for growth.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,700+ biopharma pros reading Endpoints daily — and it's free.

FDA is much worse than its reg­u­la­to­ry peers at proac­tive­ly dis­clos­ing da­ta, re­searchers find

The European Medicines Agency and Health Canada continue to outpace the FDA when it comes to proactively releasing data on drugs and biologics the agency has reviewed, leading to further questions of why the American agency can’t be more transparent.

In a study published recently in the Journal of Law, Medicine, & Ethics, Yale and other academic lawyers and researchers found that between 2016 and April 2021, the EMA proactively released data for 123 unique medical products, while Health Canada proactively released data for 73 unique medical products between 2019 and April 2021. What’s more, the EMA and Health Canada didn’t proactively release the same data on the same drugs. In stark contrast, the FDA in 2018 only proactively disclosed data supporting one drug that was approved that year.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,700+ biopharma pros reading Endpoints daily — and it's free.

Ugur Sahin, AP Images

As pres­sure to share tech­nol­o­gy mounts, BioN­Tech se­lects Rwan­da for lat­est vac­cine site

BioNTech’s first mRNA-based vaccine site in Africa will call Rwanda home, and construction is set to start in mid-2022, the company announced Tuesday at a public health forum.

The German company signed a memorandum of understanding, after a meeting between Rwanda’s Minister of Health, Daniel Ngamije, Senegal’s Minister of Foreign Affairs Aïssata Tall Sall, and senior BioNTech officials. Construction plans have been finalized, and assets have been ordered. The agreement will help bring end-to-end manufacturing to Africa, and as many as several hundred million doses of vaccines per year, though initial production will be more modest.

UP­DAT­ED: Eli Lil­ly toss­es a mar­quee pain drug and hits the gas on Alzheimer’s — as Bio­gen’s suf­fer­ing opens mar­ket to ri­vals

The furious chorus of critics that brought sales of Biogen’s ultra controversial Alzheimer’s drug aducanumab (sold as Aduhelm) to a near halt is opening up some big opportunities for a major league rival that has long sought the lead role in this largely untapped megamarket.

In its Q3 update today, Eli Lilly — noted for its dogged persistence in attempting for years to get solanezumab across the FDA finish line — said that it has begun a rolling submission of its rival Alzheimer’s drug donanemab in search of an accelerated approval. Anne White, senior VP of Lilly’s neuroscience unit, acknowledged during the investor call the challenges Biogen has faced with uptake and noted Lilly may face similar hurdles.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,700+ biopharma pros reading Endpoints daily — and it's free.

Peter Nell, Mammoth Biosciences CBO

UP­DAT­ED: Jen­nifer Doud­na spin­out inks a Mam­moth CRISPR deal with Ver­tex worth near­ly $700M

When a company gets its start in gene editing pioneer Jennifer Doudna’s lab, it’s bound to make headlines. But three years in, the fanfare still hasn’t died down for Mammoth Biosciences. Now, the Brisbane, CA-based company is cheering on its first major R&D pact.

Mammoth unveiled a nearly $700 million deal with Vertex on Tuesday morning, good for the development of in vivo gene therapies for two mystery diseases. The stars of the show are Mammoth’s ultra-small CRISPR systems, including two Cas enzymes licensed from Doudna’s lab over the past couple years, Cas14 and Casɸ.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,700+ biopharma pros reading Endpoints daily — and it's free.