A small, ground­break­ing car­dio study push­es MyoKar­dia to a piv­otal show­down — shares soar

MyoKar­dia $MYOK has come up with the proof-of-con­cept ef­fi­ca­cy da­ta that the South San Fran­cis­co-based biotech was look­ing for in its small but po­ten­tial­ly ground­break­ing car­dio study. And now it will ex­plore by­pass­ing one el­e­ment in its de­vel­op­ment plan and see whether the FDA will sign up on a straight shot at a piv­otal study that could point the com­pa­ny down a straight path to the mar­ket­place.

Tas­sos Gi­anakakos

MyoKar­dia’s shares rock­et­ed up 83% as in­vestors took a look at the num­bers and its plans for its drug mava­camten.

The field is cur­rent­ly dom­i­nat­ed by a few ma­jor drugs look­ing to in­flu­ence the course of heart dis­ease for large num­bers of pa­tients. While rais­ing the bar on safe­ty and ef­fi­ca­cy in heart dis­ease is an ob­sta­cle course rarely nav­i­gat­ed by any­one out­side of gi­ant phar­ma, this biotech is fol­low­ing a game-chang­ing mol­e­c­u­lar strat­e­gy with a ri­fle-shot ap­proach at dis­ease.

MyoKar­dia’s ini­tial fo­cus on heart dis­ease cen­ters on an ail­ment called ob­struc­tive hy­per­trophic car­diomy­opa­thy, where a mu­ta­tion in heart pro­teins forces the heart to squeeze more, thick­en­ing heart mus­cles and cre­at­ing a cas­cade of ef­fects and symp­toms that can lead to afib­ril­la­tion and death.

“By tar­get­ing the mo­tor pro­tein in the heart, off­set­ting the mu­ta­tion, you’re bring­ing the force of the squeeze back to nor­mal,” MyoKar­dia CEO Tas­sos Gi­anakakos tells me. “The heart re­lax­es, that ob­struc­tion moves out of the way… By damp­en­ing the mu­ta­tion­al ef­fect, the heart re­mod­els. We’d love to see that heart look like a nor­mal heart.”

Out­comes in the field take a for­tune to nail down, but MyoKar­dia’s con­cen­trat­ed on post-ex­er­cise peak left ven­tric­u­lar out­flow tract, or LVOT, gra­di­ent from base­line to week 12, in 11 pa­tients who were re­cruit­ed for the study. One of those pa­tients was forced out due to episodes of atri­al fib­ril­la­tion pos­si­bly linked to the re­quire­ment that the pa­tients in the first co­hort wash out be­ta block­ers that are used to keep the con­di­tion

But for the 10 who com­plet­ed the dos­ing, the base­line mea­sure of 124.9 plunged to 18.9 — be­low the di­ag­nos­tic thresh­old for the dis­ease in 8 of 10 pa­tients — while peak VO2  dropped more than the 10% to 15% lev­el than the biotech had told an­a­lysts to watch out for.

“While it is 10 pa­tients,” notes the CEO, “the treat­ment is mas­sive.”

As a re­sult of the da­ta, and the ef­fi­ca­cy on peak VO2, which is the planned piv­otal end­point, Gi­anakakos is now plan­ning to meet with the FDA and see if he can by­pass a Phase IIb and go straight to a reg­is­tra­tion study, cut­ting as much as 18 months out of the de­vel­op­ment time­line.

I’ve cov­ered MyoKar­dia right from the Se­ries A with an idea of start­ing a biotech that would break things down in car­dio R&D on a ge­net­ic ba­sis. Gi­anakakos es­ti­mates that there are some 400,000 pa­tients with this par­tic­u­lar con­di­tion, about 65,000 of whom are symp­to­matic of the dis­ease. That’s the kind of tar­get that a small, fo­cused biotech com­pa­ny can hit with a small sales force. And he be­lieves that if the FDA signs off on his piv­otal plans, he can get start­ed lat­er this year on a 2-year ef­fort.

Amarin CEO John Thero discussing the company's plans for Vascepa, August 2019 — via Bloomberg

Amarin wins a block­buster ap­proval from the FDA. Now every­one can shift fo­cus to the patent

For all those people who could never quite believe that Amarin $AMRN would get an expanded label with blockbuster implications, the stress and anxiety on display right up to the last minute on Twitter can now end. But new, pressing questions will immediately surface now that the OK has come through.

On Friday afternoon, the FDA stamped its landmark approval on the industrial strength fish oil for reducing cardio risks for a large and well defined population of patients. The approval doesn’t give Amarin everything it wants in expanding its use, losing out on the primary prevention group, but it goes a long way to doing what the company needed to make a major splash. The approval was cited for patients with “elevated triglyceride levels (a type of fat in the blood) of 150 milligrams per deciliter or higher. Patients must also have either established cardiovascular disease or diabetes and two or more additional risk factors for cardiovascular disease.”

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Sarep­ta was stunned by the re­jec­tion of Vyondys 53. Now it's stun­ning every­one with a sur­prise ac­cel­er­at­ed ap­proval

Sarepta has a friend in the FDA after all. Four months after the agency determined that it would be wrong to give Sarepta an accelerated approval for their Duchenne MD drug golodirsen, regulators have executed a stunning about face and offered the biotech a quick green light in any case.

It was the agency that first put out the news late Thursday, announcing that Duchenne MD patients with a mutation amenable to exon 53 skipping will now have their first targeted treatment: Vyondys 53, or golodirsen. Having secured the OK via a dispute resolution mechanism, the biotech said the new drug has been priced on par with their only other marketed drug, Exondys 51 — which for an average patient costs about $300,000 per year, but since pricing is based on weight, that sticker price can even cross $1 million.

Sarepta shares $SRPT surged 23% after-market to $124.

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UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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Paul Biondi (File photo)

Paul Biondi's track record at Bris­tol-My­ers cov­ered bil­lions in deals of every shape and size. Here's the com­plete break­down

Paul Biondi was never afraid to bet big during his stint as business development chief at Bristol-Myers Squibb. And while the gambles didn’t all pay out, by any means, his roster of pacts illustrates the broad ambitions the pharma giant has had over the last 5 years — capped by the $74 billion Celgene buyout.

On Thursday, we learned that Biondi had exited the company. And Chris Dokomajilar at DealForma came up with the complete breakdown on every buyout, licensing pact and product purchase Bristol-Myers forged during his tenure in charge of the BD team at one of the busiest companies in biopharma.

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Arie Belldegrun (Photo: Jeff Rumans for Endpoints News)

Ju­ry finds Gilead li­able for $585M and big roy­al­ties in Kite CAR-T patent case

A Kite deal that’s already become a burden on Gilead’s back just got heavier as a California jury has ruled Gilead must pay Bristol-Myers Squibb and Sloan Kettering $585 million plus a 27.6% royalty for patent infringement committed by its subsidiary. The ruling is almost certain to be appealed.

Kite Pharma — founded by Arie Belldegrun, now focused on a next-gen CAR-T company — has been facing a lawsuit since the day its first CAR–T therapy won approval in October, 2017. Juno Therapeutics and Sloan Kettering filed a complaint saying Kite had copied its technology. Gilead acquired Kite in June of that year for $11.9 billion.  Juno was acquired the following year by Celgene for $9 billion, before Celgene was acquired by Bristol-Myers Squibb in 2019.

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FDA ex­pert pan­el unan­i­mous­ly rec­om­mends ap­proval for Hori­zon Ther­a­peu­tics eye drug

An FDA advisory committee noted with concern a small safety database but unanimously endorsed a Horizon Therapeutics drug for a rare eye autoimmune disease that can blind patients: teprotumumab for thyroid eye disease (TED).

“It was a pretty easy vote,” said Erica Brittain, an NIH biostatistician and one of the 12 panelists on FDA’s Dermatologic and Ophthalmic Drugs Advisory Committee.

Paul Biondi (File photo)

Bris­tol-My­er­s' strat­e­gy, BD chief Paul Bion­di ex­it­ed the com­pa­ny — just ahead of the $74B Cel­gene deal close

Paul Biondi, who orchestrated billions of dollars in deals for Bristol-Myers Squibb over the 5 years he’s run their business development team, has exited the company. Biondi left last month, according to a company spokesperson, in pursuit of another — unspecified — external opportunity.

After 17 years with Bristol-Myers Squibb, Paul Biondi, Head of Strategy and Business Development, decided to leave the company to pursue an external opportunity. The company wishes him well in his new endeavors. Bristol-Myers Squibb  is actively searching for Paul’s successor, and will make an announcement, as appropriate.

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Arie Belldegrun at UKBIO 2019. Shai Dolev for Endpoints News

Kite Phar­ma's ex-CEO con­tra­dicts founder as CAR-T patent tri­al heats up, with con­flict­ing val­u­a­tions

Two days after Kite Pharma founder Arie Belldegrun told a federal courtroom that a meeting he had with a Memorial Sloan Kettering executive wasn’t about licensing their immunotherapy patent, Kite’s ex-CEO Aya Jakobovits said it was.

The admission came Tuesday during cross-examination in a patent infringement case that features two of the biggest cancer biotechs and some of the most well-known names in American medicine.

Jakobovits initially said she was not in attendance, didn’t know it was going to happen and didn’t know what took place, according to Law360. But then the plaintiff’s lawyer handed her a document – whose contents were not publicly revealed – and asked again if she learned after-the-fact that the meeting involved a potential patent license.

“Yes,” Jakobovits eventually said.

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On the heels of promis­ing MCL da­ta, Kite hus­tles its 2nd CAR-T to the FDA as the next big race in the field draws to the fin­ish line

Three days after Gilead’s Kite subsidiary showed off stellar data on their number 2 CAR-T KTE-X19 at ASH, the executive team has pivoted straight to the FDA with a BLA filing and a shot at a near-term approval.

In a small, 74-patient Phase II trial reported out at the beginning of the week, investigators tracked a 93% response rate with two out of three mantle cell lymphoma patients experiencing a complete response.

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