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.

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,100+ biopharma pros reading Endpoints daily — and it's free.

Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,100+ biopharma pros reading Endpoints daily — and it's free.

Gen­mab ax­es an ADC de­vel­op­ment pro­gram af­ter the da­ta fail to im­press

Genmab $GMAB has opted to ax one of its antibody-drug conjugates after watching it flop in the clinic.

The Danish biotech reported Tuesday that it decided to kill their program for enapotamab vedotin after the data gathered from expansion cohorts failed to measure up. According to the company:

While enapotamab vedotin has shown some evidence of clinical activity, this was not optimized by different dose schedules and/or predictive biomarkers. Accordingly, the data from the expansion cohorts did not meet Genmab’s stringent criteria for proof-of-concept.

Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

Vas Narasimhan's 'Wild Card' drugs: No­var­tis CEO high­lights po­ten­tial jack­pots, as well as late-stage stars, in R&D pre­sen­ta­tion

Novartis is always one of the industry’s biggest R&D spenders. As they often do toward the end of each year, company execs are highlighting the drugs they expect will most likely be winners in 2021.

And they’re also dreaming about some potential big-time lottery tickets.

As part of its annual investor presentation Tuesday, where the company allows investors and analysts to virtually schmooze with the bigwigs, Novartis CEO Vas Narasimhan will outline what he thinks are the pharma’s “Wild Cards.” The slate of five experimental drugs are those that Novartis hopes can be high-risk, high-reward entrants into the market over the next half-decade or so, and cover a wide range of indications.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,100+ biopharma pros reading Endpoints daily — and it's free.

The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Bahija Jallal (file photo)

TCR pi­o­neer Im­muno­core scores a first with a land­mark PhI­II snap­shot on over­all sur­vival for a rare melanoma

Bahija Jallal’s crew at TCR pioneer Immunocore says they have nailed down a promising set of pivotal data for their lead drug in a frontline setting for a solid tumor. And they are framing this early interim readout as the convincing snapshot they need to prove that their platform can deliver on a string of breakthrough therapies now in the clinic or planned for it.

In advance of the Monday announcement, Jallal and R&D chief David Berman took some time to walk me through the first round of Phase III data for their lead TCR designed to treat rare, frontline cases of metastatic uveal melanoma that come with a grim set of survival expectations.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,100+ biopharma pros reading Endpoints daily — and it's free.

News brief­ing: Gilead part­ner Gala­pa­gos sells off CRO for $37M; Polyphor bags $3.3M from CF Foun­da­tion

Close Gilead ally Galapagos is selling off one of its contract research organizations to a Polish pharma company.

Galapagos has agreed to sell 100% of the outstanding shares in the CRO Fidelta to Selvita, in a deal worth roughly $37 million expected to close in the first week of January. The acquisition is expected to nearly double Selvita’s revenues, the company says, as well as expand its drug discovery efforts.