Spark adds fresh ev­i­dence that pi­o­neer­ing gene ther­a­py could be a game chang­er for pa­tients

Spark Ther­a­peu­tics has added an­oth­er im­pres­sive round of late-stage da­ta demon­strat­ing that its gene ther­a­py for an in­her­it­ed eye dis­ease could be a game chang­er for pa­tients. This lat­est read­out in­clud­ed pos­i­tive out­comes for 9 crossover pa­tients who re­ceived voreti­gene nepar­vovec (SPK-RPE65) a year ago, along with promis­ing longer term re­sults on the orig­i­nal group of 20 pa­tients.

Nine pa­tients with in­her­it­ed reti­nal dis­ease caused by mu­ta­tions in the RPE65 gene were put in the con­trol arm of the study, and all vol­un­teered for the gene ther­a­py af­ter in­ves­ti­ga­tors con­clud­ed that the orig­i­nal treat­ment group had ben­e­fit­ed marked­ly with sig­nif­i­cant gains in vi­sion. A year af­ter treat­ment, Spark says that 8 of the 9 “demon­strat­ed the max­i­mum im­prove­ment mea­sur­able on the pri­ma­ry end­point at one year.” And the first 20 pa­tients who were treat­ed with their gene ther­a­py have demon­strat­ed a sus­tained ben­e­fit in vi­sion, speak­ing to the dura­bil­i­ty of the change.

But it wasn’t all up­beat for Spark to­day. An­a­lysts not­ed that the biotech ex­pects a sig­nif­i­cant de­lay in its reg­u­la­to­ry fil­ing, cit­ing CMC is­sues.

“ONCE al­so re­port­ed that the BLA sub­mis­sion will be de­layed un­til ear­ly 2017 due to in­com­plete CMC da­ta,” not­ed Wed­bush an­a­lyst David Nieren­garten. “(W)e have pushed back the ex­pect­ed launch from 4/1/17 to 10/1/17 in the US, which re­duces our PT by $1.” In­vestors didn’t over­look the un­ex­pect­ed set­back, send­ing Spark’s shares down slight­ly.

Voreti­gene nepar­vovec is a lead­ing gene ther­a­py in a di­verse field that has pro­duced re­mark­ably mixed re­sults. Wide­ly billed as the first gene ther­a­py like­ly to go to the FDA in search of mar­ket­ing ap­proval, it’s be­come a test case for dis­cus­sions on pric­ing a po­ten­tial one-time treat­ment. And it’s al­so helped un­der­score the sim­ple fact that not all gene ther­a­pies are equal, as de­vel­op­ers test new and bet­ter de­liv­ery ve­hi­cles and cor­rec­tive genes.

Spark wants to be the leader, and it’s been mak­ing its case with da­ta like this.

Look­ing for ev­i­dence of a func­tion­al im­prove­ment in vi­sion, Spark re­port­ed that the 8 ad­di­tion­al pa­tients hit the top rung in the tracked change in bi­lat­er­al mo­bil­i­ty test­ing. On light sen­si­tiv­i­ty test­ing, the eight al­so im­proved, “with an av­er­age im­prove­ment of near­ly 200-fold, com­pared to the more than 100-fold im­prove­ment av­er­age seen in the 301 study sub­jects. Sub­jects in the (crossover) study demon­strat­ed an av­er­age vi­su­al acu­ity im­prove­ment of 4.5 let­ters, av­er­aged across both eyes, com­pared to an av­er­age im­prove­ment of eight let­ters by the same analy­sis in the (orig­i­nal) 301 study.”

Spark al­so added some fresh ev­i­dence that its pi­o­neer­ing treat­ment could live up to its stock sym­bol {$ONCE), with ev­i­dence that the treat­ment was equal­ly ef­fec­tive two years af­ter treat­ment as one. Ac­cord­ing to Spark:

The mean dura­bil­i­ty of ben­e­fit was sus­tained at 1.9 lux lev­els af­ter two years, com­pared with the im­prove­ment of 1.9 lux lev­els af­ter one year. The more than 100-fold im­prove­ment av­er­age seen in the orig­i­nal in­ter­ven­tion group at one year sim­i­lar­ly was main­tained through at least two years.

Spark—a spin­out of Chil­dren’s Hos­pi­tal of Philadel­phia—has a mar­ket cap of $1.8 bil­lion.

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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