Bio­gen, Io­n­is win FDA OK for spinal mus­cu­lar at­ro­phy drug Spin­raza

Bio­gen $BI­IB and Io­n­is $IONS got an ear­ly Christ­mas present Fri­day evening as the FDA an­nounced that it had pro­vid­ed an ac­cel­er­at­ed ap­proval for Spin­raza (nusin­ersen), their new drug — and the first — for rare cas­es of spinal mus­cu­lar at­ro­phy.

Peak sales es­ti­mates hov­er around $2 bil­lion, with some of the new es­ti­mates com­ing in over $3 bil­lion by 2026.

“Our pre­vi­ous fore­cast in­clud­ed sales of $75mm in 2017 grow­ing to $2.8bn by 2026,” not­ed Leerink’s Ge­of­frey Porges in ear­ly No­vem­ber, with the ar­rival of more Phase III da­ta. “We now as­sume a launch in Q1 2017 from Q2 2017 pre­vi­ous­ly and our cur­rent fore­cast is for sales of $92mm in 2017 in­creas­ing to $1.56bn by 2020 and then $3.2bn by 2026.”

Bio­gen says that it will be ready to ship the drug in a week.

Shares of Io­n­is shot up 10% and Bio­gen saw its stock climb 2% on the news.

The ap­proval comes a lit­tle less than two months af­ter re­searchers post­ed a fresh round of promis­ing da­ta on the drug. Us­ing the Ham­mer­smith Func­tion­al Mo­tor Scale Ex­pand­ed re­port card for pa­tients’ mo­tor func­tions, the drug arm wit­nessed a mean 4.0 im­prove­ment on scores while the place­bo arm dropped by 1.9 points, a 5.9 point over­all spread in the drug’s fa­vor.

Io­n­is CEO Stan­ley Crooke

Both com­pa­nies bad­ly need­ed this win af­ter a year of up­heavals. The ap­proval will help set the stage for Bio­gen’s new CEO, Michel Vounatsos, to take the helm. Io­n­is, mean­while, can boast of a year-end vic­to­ry af­ter set­backs in the clin­ic. And an­a­lysts were im­pressed by the broad la­bel ap­plied by the FDA.

“La­bel­ing is not re­strict­ed to any spe­cif­ic SMA sub­type, in­di­cat­ing that it will like­ly be broad­ly pre­scribed for type 1, 2, and 3 pa­tients and while the epi­demi­ol­o­gy in SMA is not en­tire­ly de­fin­i­tive, a la­bel mak­ing the drug avail­able to all pa­tient types rep­re­sents sig­nif­i­cant ex­pan­sion po­ten­tial to the ini­tial mar­ket op­por­tu­ni­ty (though giv­en the un­met need in SMA, this pos­i­tive de­vel­op­ment is not en­tire­ly un­sur­pris­ing),” notes Jef­feries Bri­an Abra­hams.

The ap­proval al­so comes with a rare dis­ease pri­or­i­ty vouch­er, which can be used to ac­cel­er­ate re­view of an­oth­er pro­gram, or sold on the open mar­ket.

“There has been a long-stand­ing need for a treat­ment for spinal mus­cu­lar at­ro­phy, the most com­mon ge­net­ic cause of death in in­fants, and a dis­ease that can af­fect peo­ple at any stage of life,” said Bil­ly Dunn, MD, di­rec­tor of the Di­vi­sion of Neu­rol­o­gy Prod­ucts in the FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search. “As shown by our sug­ges­tion to the spon­sor to an­a­lyze the re­sults of the study ear­li­er than planned, the FDA is com­mit­ted to as­sist­ing with the de­vel­op­ment and ap­proval of safe and ef­fec­tive drugs for rare dis­eases and we worked hard to re­view this ap­pli­ca­tion quick­ly; we could not be more pleased to have the first ap­proved treat­ment for this de­bil­i­tat­ing dis­ease.”

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 Hudson, Getty Images

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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