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.

Hal Barron, GSK

Break­ing the death spi­ral: Hal Bar­ron talks about trans­form­ing the mori­bund R&D cul­ture at GSK in a crit­i­cal year for the late-stage pipeline

Just ahead of GlaxoSmithKline’s Q2 update on Wednesday, science chief Hal Barron is making the rounds to talk up the pharma giant’s late-stage strategy as the top execs continue to woo back a deeply skeptical investor group while pushing through a whole new R&D culture.

And that’s not easy, Barron is quick to note. He told the Financial Times:

I think that culture, to some extent, is as hard, in fact even harder, than doing the science.

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Aca­dia is mak­ing the best of it, but their lat­est PhI­II Nu­plazid study is a bust

Acadia’s late-stage program to widen the commercial prospects for Nuplazid has hit a wall. The biotech reported that their Phase III ENHANCE trial flat failed. And while they $ACAD did their best to cherry pick positive data wherever they can be found, this is a clear setback for the biotech.

With close to 400 patients enrolled, researchers said the drug flunked the primary endpoint as an adjunctive therapy for patients with an inadequate response to antipsychotic therapy. The p-value was an ugly 0.0940 on the Positive and Negative Syndrome Scale, which the company called out as a positive trend.

Their shares slid 12% on the news, good for a $426 million hit on a $3.7 billion market cap at close.

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Some Big Phar­mas stepped up their game on da­ta trans­paren­cy — but which flunked the test?

The nonprofit Bioethics International has come out with their latest scorecard on data transparency among the big biopharmas in the industry — flagging a few standouts while spotlighting some laggards who are continuing to underperform.

Now in its third year, the nonprofit created a new set of standards with Yale School of Medicine and Stanford Law School to evaluate the track record on trial registration, results reporting, publication and data-sharing practice.

Busy Gilead crew throws strug­gling biotech a life­line, with some cash up­front and hun­dreds of mil­lions in biobucks for HIV deal

Durect $DRRX got a badly needed shot in the arm Monday morning as Gilead’s busy BD team lined up access to its extended-release platform tech for HIV and hepatitis B.

Gilead, a leader in the HIV sector, is paying a modest $25 million in cash for the right to jump on the platform at Durect, which has been using its technology to come up with an extended-release version of bupivacaine. The FDA rejected that in 2014, but Durect has been working on a comeback.

In­tec blitzed by PhI­II flop as lead pro­gram fails to beat Mer­ck­'s stan­dard com­bo for Parkin­son’s

Intec Pharma’s $NTEC lead drug slammed into a brick wall Monday morning. The small-cap Israeli biotech reported that its lead program — coming off a platform designed to produce a safer, more effective oral drug for Parkinson’s — failed the Phase III at the primary endpoint.

Researchers at Intec, which has already seen its share price collapse over the past few months, says that its Accordion Pill-Carbidopa/Levodopa failed to prove superior to Sinemet in reducing daily ‘off’ time. 

Cel­gene racks up third Ote­zla ap­proval, heat­ing up talks about who Bris­tol-My­ers will sell to

Whoever is taking Otezla off Bristol-Myers Squibb’s hands will have one more revenue stream to boast.

The drug — a rising star in Celgene’s pipeline that generated global sales of $1.6 billion last year — is now OK’d to treat oral ulcers associated with Behçet’s disease, a common symptom for a rare inflammatory disorder. This marks the third FDA approval for the PDE4 inhibitor since 2014, when it was greenlighted for plaque psoriasis and psoriatic arthritis.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors.

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Apotex, though, has been a disaster ground. The manufacturer voluntarily yanked the ANDAs on 31 drugs — in late 2017 — after the FDA came across serious manufacturing deficiencies at their plants in India. A few days ago, the FDA made it official.

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