Step 3: Sanofi com­mits up to $100M to opt-in on Al­ny­lam’s PhI­II RNAi he­mo­phil­ia tri­al

John Maraganore, Al­ny­lam

Al­ny­lam is step­ping up to the start­ing line for a Phase III study of its he­mo­phil­ia drug fi­tusir­an with Sanofi on board as a part­ner. The phar­ma com­pa­ny lined up the opt-in rights un­der their ear­li­er deal to buy a mi­nor­i­ty stake in the biotech for $700 mil­lion. And Sanofi is now com­mit­ted to pro­vid­ing $75 mil­lion in de­vel­op­ment and reg­u­la­to­ry mile­stones fol­low­ing a $25 mil­lion up­front due when Phase III starts in ear­ly 2017.

Cam­bridge, MA-based Al­ny­lam $AL­NY and its col­lab­o­ra­tor Sanofi $SFY will now part­ner up on de­vel­op­ment of the RNAi pro­gram with the prospect of a mar­ket­ing al­liance in the US, Cana­da and West­ern Eu­rope, while Sanofi Gen­zyme in Boston will have rights to de­vel­op the drug for he­mo­phil­ia and rare bleed­ing dis­or­ders in the rest of the world.

Ex­ecs from both sides her­ald­ed the move as an­oth­er big step for­ward for an al­liance an­nounced with great fan­fare in ear­ly 2014, which great­ly ex­pand­ed an ex­ist­ing re­la­tion­ship. For Sanofi, the Al­ny­lam deal re­flect­ed their en­thu­si­asm for a sim­i­lar re­la­tion­ship they have with Re­gen­eron, which has been their most pro­duc­tive pact.

Sanofi Gen­zyme, though, is leav­ing an­oth­er pro­gram on the side­line at Al­ny­lam. There will be no opt-in for ALN-AS1, an­oth­er RNAi drug aimed at acute he­pat­ic por­phyr­ias. Sanofi bowed out af­ter tak­ing a look at the da­ta from the first two stages of a Phase I study. A third stage is com­ing up for re­view as Al­ny­lam plans to pro­ceed on its own.

The opt-in on he­mo­phil­ia comes as a boon to Al­ny­lam, which had to scrap the num­ber two drug in its pipeline — re­vusir­an — af­ter track­ing a dis­pro­por­tion­ate num­ber of deaths on the treat­ment arm of a late-stage pro­gram. On a brighter note, Al­ny­lam part­ner The Med­i­cines Com­pa­ny en­joyed a clin­i­cal suc­cess with their PC­SK9 drug.

Al­ny­lam CEO John Maraganore had this to say:

“This de­ci­sion al­lows us to broad­en the glob­al reach and ac­cel­er­ate the com­mer­cial de­vel­op­ment of fi­tusir­an, a po­ten­tial­ly trans­for­ma­tive ap­proach to the treat­ment of he­mo­phil­ia and rare bleed­ing dis­or­ders, with a well-es­tab­lished part­ner in Sanofi Gen­zyme. We look for­ward to con­tin­ued col­lab­o­ra­tion with Sanofi Gen­zyme to ad­vance fi­tusir­an as Al­ny­lam tran­si­tions to­ward the com­mer­cial stage with our late-stage pipeline.”

Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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As­traZeneca trum­pets the good da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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The Avance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

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Bryan Roberts, Venrock

Ven­rock sur­vey shows grow­ing recog­ni­tion of coro­n­avirus toll, wan­ing con­fi­dence in ar­rival of vac­cines and treat­ments

When Venrock partner Bryan Roberts went to check the results from their annual survey of healthcare leaders, what he found was an imprint of the pandemic’s slow arrival in America.

The venture firm had sent their form out to hundreds of insurance and health tech executives, investors, officials and academics on February 24 and gave them two weeks to fill it out. No Americans had died at that point but the coronavirus had become enough of a global crisis that they included two questions about the virus, including “Total U.S. deaths in 2020 from the novel coronavirus will be:”.

David Hoey (Vaxxas)

In for the long vac­cine game, Mer­ck buys in­to patch de­liv­ery tech with pan­dem­ic po­ten­tial

When Merck dived into the R&D fray for a Covid-19 vaccine earlier this week, execs made it clear that they’re not necessarily looking to be first — with CEO Ken Frazier throwing cold water on the hotly-discussed 12- to 18-month timelines. But when it does emerge from behind, the pharma giant clearly expects to play a significant part.

Part of that will depend on next-generation delivery technology that reshapes the world’s imagination of a vaccine.

No­var­tis jumps in­to Covid-19 vac­cine hunt, as Big Phar­ma and big biotech com­mit to bil­lions of dos­es

After spending most of the pandemic on the sidelines, Novartis is offering its aid in the race to develop a Covid-19 vaccine.

AveXis, the Swiss pharma’s gene therapy subsidiary, has agreed to manufacture the vaccine being developed by Massachusetts Eye and Ear and Massachusetts General Hospital. The biotech will begin manufacturing this month, while the vaccine undergoes further preclinical testing. They’ve agreed to provide the vaccine for free for clinical trials beginning in the second half of 2020, but have not disclosed financials for after.

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