MyoKar­dia re­claims US roy­al­ty rights to heart drugs, com­plet­ing con­scious un­cou­pling from Sanofi

In Jan­u­ary, MyoKar­dia dis­closed that Sanofi was walk­ing away from their heart drug part­ner­ship forged in 2014. On Thurs­day, the San Fran­cis­co-based biotech said it had re­gained the US roy­al­ty rights to two prod­ucts from the French drug­mak­er for $50 mil­lion up­front.

MyoKar­dia $MYOK will al­so pay an ad­di­tion­al $30 mil­lion next year for the rights to the ex­per­i­men­tal drugs: mava­camten and MYK-224.  Un­der the terms of the for­mer deal with Sanofi $SNY, the Parisian com­pa­ny was el­i­gi­ble to re­ceive tiered roy­al­ties rang­ing from 5% to 10% on US sales of the two drugs.

MyoKar­dia man­aged to get a good price for the rights, “which ap­pears to be the re­sult of Sanofi be­ing a mo­ti­vat­ed sell­er (as op­posed to Sanofi hav­ing con­cerns about the mava­camten mar­ket op­por­tu­ni­ty),” Cred­it Su­isse an­a­lysts wrote in a note. “We spoke with MYOK man­age­ment who re­port­ed there were sev­er­al oth­er par­ties who tried to ob­tain the roy­al­ty rights, in­clud­ing an­oth­er par­ty who re­port­ed­ly had a sig­nif­i­cant­ly high­er bid.”

MyoKar­dia’s lead drug, mava­camten, is ex­pect­ed to break new ground in heart dis­ease — a field mo­nop­o­lized by large phar­ma­ceu­ti­cal com­pa­nies large­ly due to the long, ar­du­ous and ex­pen­sive tri­als that are com­mon­place in heart drug de­vel­op­ment. Un­like oth­er de­vel­op­ers fo­cus­ing on com­mon heart dis­or­ders, MyoKar­dia’s treat­ment is al­so tar­get­ing a so-far un­tapped con­di­tion — ob­struc­tive hy­per­trophic car­diomy­opa­thy — in which a heart pro­tein mu­ta­tion forces the or­gan to squeeze more, thick­en­ing heart mus­cles and cre­at­ing a cas­cade of con­se­quences that can cul­mi­nate in death.

In the sec­ond tranche of the drug’s Phase II tri­al in a small group of pa­tients re­port­ed in March, the com­pa­ny said its low dose ap­proach was large­ly suc­cess­ful across a crop of end­points, ex­cept one for peak VO2 (ex­er­cise ca­pac­i­ty mea­sured by an in­crease in oxy­gen con­sump­tion). Last June, MyoKar­dia launched its keen­ly an­tic­i­pat­ed piv­otal tri­al for the drug — and on Thurs­day brought for­ward its ex­pect­ed read­out to the sec­ond quar­ter of next year, from the pre­vi­ous­ly fore­cast­ed sec­ond half of 2020.

MyoKar­dia’s ap­proach to re­search is to de­vel­op drugs for ge­net­i­cal­ly de­fined pa­tient groups, which is al­so re­flect­ed in its sec­ond pro­gram, MYK-491, un­der de­vel­op­ment for di­lat­ed car­diomy­opa­thy. The Sanofi part­ner­ship in­volved the de­vel­op­ment of up to three pro­grams through dis­cov­ery and in­to clin­i­cal de­vel­op­ment for the treat­ment of hy­per­trophic car­diomy­opa­thy (HCM) and di­lat­ed car­diomy­opa­thy (DCM). In Jan­u­ary, MyoKar­dia re­gained the glob­al rights to all the pro­grams un­der the re­search and col­lab­o­ra­tion agree­ment with Sanofi, which will not be ex­tend­ed be­yond the ini­tial re­search term, which end­ed on De­cem­ber 31, 2018.

The price MyoKar­dia paid for the US roy­al­ty rights to mava­camten (and MYK-224) is right, giv­en the $3 bil­lion-plus op­por­tu­ni­ty es­ti­mat­ed for the drug in ob­struc­tive and nonob­struc­tive hy­per­trophic car­diomy­opa­thy, BMO Cap­i­tal Mar­kets an­a­lysts wrote in a note.

So­cial im­age: Shut­ter­stock

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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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.

FDA asks why No­var­tis took two months to launch for­mal in­ter­nal probe, af­ter AveX­is flagged da­ta ma­nip­u­la­tion

And the plot thickens. Novartis $NVS officials are reportedly now scrambling to explain to the FDA why it took them two months to open an internal investigation into data discrepancies for their $2.1 million gene-therapy for spinal muscular dystrophy — the world’s most expensive drug.

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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