Al­ny­lam kicks off #JPM18 with a re­struc­tured Sanofi deal, grab­bing glob­al rights to patisir­an

Four years ago Al­ny­lam $AL­NY jazzed the start of the JP­Mor­gan con­fer­ence with news that Sanofi had stepped up to pay $700 mil­lion for a chunk of its stock and the right to ex­pand its com­mer­cial rights to the biotech’s lead drug, patisir­an. But with patisir­an on the verge of win­ning like­ly reg­u­la­to­ry ap­proval this year, the com­pa­nies have once again re­struc­tured their deal in a way that gives Al­ny­lam glob­al con­trol over its fu­ture.

John Maraganore

Al­ny­lam will now han­dle the glob­al mar­ket­ing for patisir­an and ALN-TTRsc02, with the ex­pect­ed launch com­ing for AT­TR amy­loi­do­sis. In re­turn, Sanofi is land­ing glob­al rights to fi­tusir­an for he­mo­phil­ia A and B.

The rest of the deal terms re­main in place, with the two col­lab­o­ra­tors ex­chang­ing roy­al­ties on sales.

The move un­der­scores Al­ny­lam CEO John Maraganore’s con­fi­dence that the biotech can score big on patisir­an, which is head­ed in­to a com­mer­cial ri­val with Io­n­is $IONS, which has al­so racked up pos­i­tive da­ta for its hAT­TR amy­loi­do­sis drug. Most an­a­lysts, though, give Al­ny­lam a dis­tinct edge af­ter re­view­ing the piv­otal da­ta be­ing hus­tled to the FDA and EMA.

Al­ny­lam says it ex­pects to land an OK from the FDA in mid-2018, with a green light from the EMA fol­low­ing soon af­ter.

The new deal comes just weeks af­ter reg­u­la­tors lift­ed a clin­i­cal hold on fi­tusir­an, which was put in place fol­low­ing the death of a pa­tient due to a blood clot. Sanofi had paid $100 mil­lion to opt in on fi­tusir­an 10 months be­fore the hold was clamped down on the late-stage study.

Notes Leerink’s Paul Mat­teis:

Un­der the new agree­ment, AL­NY gains ROW com­mer­cial­iza­tion rights for patisir­an while re­tain­ing their rights to the US, Cana­da, and west­ern EU. SNY now stands to re­ceive tiered roy­al­ties of up to 25% on ROW sales for patisir­an, with the caveat that in Japan, SNY will re­ceive a flat roy­al­ty rate (not tiered) of 25%. AL­NY gain­ing ROW rights to patisir­an in ex­change for the roy­al­ty is like­ly to have a more mod­est im­pact on our mod­el. More im­pact­ful, the new agree­ment gives AL­NY full glob­al de­vel­op­ment and com­mer­cial rights to their fol­low-on TTR prod­uct ALN-TTRsc02 and AL­NY will pay SNY tiered roy­al­ties of 15%-30% on glob­al net sales. Fi­nal­ly, SNY will ob­tain full glob­al de­vel­op­ment and com­mer­cial­iza­tion rights to fi­tusir­an (he­mo­phil­ia). Per the agree­ment, AL­NY stands to re­ceive tiered roy­al­ties of 15%-30% on glob­al net sales of fi­tusir­an.

“This strate­gic re­struc­tur­ing en­ables stream­lined de­vel­op­ment and an op­ti­mized ap­proach to bring­ing in­no­v­a­tive med­i­cines to pa­tients with AT­TR amy­loi­do­sis and he­mo­phil­ia around the world, max­i­miz­ing the com­mer­cial op­por­tu­ni­ties for these pro­grams,” said Maraganore. “For Al­ny­lam, this pro­vides strate­gic clar­i­ty and op­er­a­tional align­ment with re­gard to the de­vel­op­ment and com­mer­cial­iza­tion of patisir­an and ALN-TTRsc02. This will al­low us to de­vel­op both prod­ucts in a com­pre­hen­sive man­ner, po­ten­tial­ly ad­dress­ing the full spec­trum of transthyretin-me­di­at­ed amy­loi­do­sis dis­ease treat­ment and pre­ven­tion. At the same time, we will con­tin­ue to sup­port and ben­e­fit – via roy­al­ties – from the fi­tusir­an op­por­tu­ni­ty through Sanofi’s sig­nif­i­cant de­vel­op­ment and com­mer­cial lead­er­ship.”

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at with any issues.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.