FDA rolls out an ear­ly and his­toric OK for Spark's pi­o­neer­ing gene ther­a­py -- now let's talk price

Spark Ther­a­peu­tics CSO Kather­ine High and CEO Jeff Mar­raz­zo. Spark


Spark Ther­a­peu­tics $ONCE  has scored an his­toric FDA ap­proval of Lux­tur­na, the world’s first such AAV-de­liv­ered gene ther­a­py de­signed to cure a rare eye dis­ease trig­gered by a ge­net­ic mu­ta­tion.

The drug is OK’d for RPE65 mu­ta­tion linked reti­nal dy­s­tro­phy. The treat­ment us­es a vi­ral vec­tor to in­sert the cor­rect copy of a gene reti­nal cells need to cre­ate a pro­tein that turns light in­to elec­tric sig­nals which can re­store vi­sion lost to the dis­ease.

Scott Got­tlieb

As with the ear­li­er pi­o­neer­ing ap­proval of the world’s first CAR-T, FDA com­mis­sion­er Scott Got­tlieb did the hon­ors in rec­og­niz­ing the im­por­tance of this ap­proval. And he says the agency will make sure that the reg­u­la­to­ry path is straight and clear for the rest of the field look­ing to fol­low­ing Spark’s foot­steps.

“We’re at a turn­ing point when it comes to this nov­el form of ther­a­py and at the FDA, we’re fo­cused on es­tab­lish­ing the right pol­i­cy frame­work to cap­i­tal­ize on this sci­en­tif­ic open­ing,” Got­tlieb not­ed. “Next year, we’ll be­gin is­su­ing a suite of dis­ease-spe­cif­ic guid­ance doc­u­ments on the de­vel­op­ment of spe­cif­ic gene ther­a­py prod­ucts to lay out mod­ern and more ef­fi­cient pa­ra­me­ters — in­clud­ing new clin­i­cal mea­sures — for the eval­u­a­tion and re­view of gene ther­a­py for dif­fer­ent high-pri­or­i­ty dis­eases where the plat­form is be­ing tar­get­ed.”

“This one-time gene ther­a­py for an in­her­it­ed dis­ease rep­re­sents a first-of-its-kind break­through that may lay the ground­work for the de­vel­op­ment of gene ther­a­pies for oth­er con­di­tions that are not ad­e­quate­ly ad­dressed to­day,” said Jef­frey Mar­raz­zo, CEO at Spark Ther­a­peu­tics, in a state­ment. “We of­fer our sin­cere grat­i­tude to the pa­tients and their fam­i­lies as well as the ex­pert in­ves­ti­ga­tors who con­tin­ue to par­tic­i­pate in Lux­tur­na’s clin­i­cal de­vel­op­ment pro­gram.”

The next big step in this process? Mar­raz­zo can tell us how much it will cost. The biotech has been hint­ing that the tick­et will come in at about $1 mil­lion, stir­ring a long run­ning de­bate over a new kind of drug de­signed to last a life­time, but proven to work for on­ly a lim­it­ed amount of time.

Peak sales es­ti­mates tend to hov­er around the $500 mil­lion a year mark.

A Spark spokesper­son said the com­pa­ny wouldn’t re­lease the drug’s price un­til Jan­u­ary, but that Lux­tur­na is ex­pect­ed to be avail­able in se­lect treat­ment cen­ters in Q1 2018.

Spark al­so has a close­ly watched he­mo­phil­ia B gene ther­a­py in the clin­ic, though its mixed da­ta from their he­mo­phil­ia A pro­gram un­der­scored how many hur­dles are left for the lead de­vel­op­ers in the field.

The ap­proval marks yet an­oth­er quick de­ci­sion for the FDA, which had a PDU­FA date for this treat­ment in mid-Jan­u­ary. The agency has been rolling out new drugs this year at a fast pace, look­ing to sur­pass 2015, when 45 new drugs were ap­proved. And the agency seems de­ter­mined to hit that goal post, if not surge past.

Paul Hudson, Getty Images

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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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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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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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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What does $6.9B buy these days in on­col­o­gy R&D? As­traZeneca has a land­mark an­swer

Given the way the FDA has been whisking through new drug approvals months ahead of their PDUFA date, AstraZeneca and their partners Daiichi Sankyo may not have to wait until Q2 of next year to get a green light on trastuzumab deruxtecan (DS-8201).

The pharma giant this morning played their ace in the hole, showing off why they were willing to commit to a $6.9 billion deal — with $1.35 billion in a cash upfront — to partner on the drug.

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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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