Acor­da preps FDA fil­ing af­ter Parkin­son’s drug hits the pri­ma­ry end­point in PhI­II


Acor­da’s $525 mil­lion gam­ble on a Phase III Parkin­son’s drug has paid off. The biotech $ACOR an­nounced ear­ly Thurs­day that the late-stage study of an in­haled res­cue for­mu­la­tion of lev­odopa was a suc­cess, open­ing the door to a fast-ap­proach­ing FDA ap­pli­ca­tion for mar­ket­ing ap­proval.

Acor­da is re­port­ing this morn­ing that CVT-301 hit the pri­ma­ry end­point in the study, demon­strat­ing a sig­nif­i­cant im­prove­ment in mo­tor skills over a place­bo. CEO Ron Co­hen scooped up the drug in an all-cash deal to ac­quire Civ­i­tas in late 2014.

The ther­a­py will tar­get Parkin­son’s pa­tients dur­ing OFF pe­ri­ods they ex­pe­ri­ence dur­ing the reg­u­lar course of their oral car­bidopa / lev­odopa reg­i­men, when Parkin­son’s symp­toms reemerge.

Acor­da’s shares jumped 21% by the close on Thurs­day.

The pri­ma­ry end­point of the study was the change at Week 12 in the Uni­fied Parkin­son’s Dis­ease Rat­ing Scale-Part 3 – UP­DRS III-  score rel­a­tive to place­bo at 30 min­utes post-treat­ment for the 84 mg dose. And it scored: UP­DRS III change for the 84 mg dose was -9.83 com­pared to -5.91 for place­bo (p=0.009), Acor­da re­port­ed. In­ves­ti­ga­tors, though, are stay­ing mum for now about the sec­ondary end­points, with the full da­ta set slat­ed for re­lease lat­er in the year.

The Phase III pri­ma­ry end­point da­ta are a sig­nif­i­cant drop from the Phase IIb re­sults, which saw a least-squares mean change in UP­DRS Part III score fa­vor CVT-301 by 7.0 points (P < 0.001) at 4 weeks. But Acor­da says it’s good enough.

The plan now is to hus­tle along an FDA ap­pli­ca­tion in Q2, af­ter in­ves­ti­ga­tors get long-term safe­ty da­ta in Q1, with an EMA pitch to fol­low be­fore the end of the year. That’s a lit­tle be­hind sched­ule. Acor­da had ini­tial­ly mapped out a plan to file by the end of 2016. But that’s a rel­a­tive­ly small hitch at this stage of the game.

The Phase III suc­cess is al­so good news for Alk­er­mes, which picked up $60 mil­lion of the buy­out cash along with a roy­al­ty stream. Alk­er­mes, which once had worked on in­haled in­sulin, pro­vid­ed a sig­nif­i­cant piece of the tech­nol­o­gy for CVT-301, which is de­liv­ered by an in­haler.

At the time of the buy­out, ISI’s Mark Schoenebaum was en­thu­si­as­tic about Acor­da’s prospects, high­light­ing the nat­ur­al fit for a com­pa­ny that has been mar­ket­ing the mul­ti­ple scle­ro­sis drug Ampyra.

“It’s an ob­vi­ous strate­gic fit (spe­cial­ty neu­ro),” he not­ed when the deal was an­nounced. “In­vestors were “prepped” for such a deal by ACOR mgmt, the med­ical need for the new prod­uct is clear­ly re­al, Phase III cost should be man­age­able, deal is like­ly to be high­ly ac­cre­tive (even­tu­al­ly) even at sales lev­els that fall short of com­pa­ny’s peak $500M US sales guid­ance, and the da­ta look sol­id.”

“We are great­ly en­cour­aged by the ef­fi­ca­cy and safe­ty re­sults of this tri­al, which val­i­date the pos­i­tive Phase IIb re­sults,” said Burkhard Blank, the chief med­ical of­fi­cer of Acor­da. “We would like to ex­press our grat­i­tude to the study vol­un­teers and clin­i­cal in­ves­ti­ga­tors who par­tic­i­pat­ed in this tri­al to ad­vance our un­der­stand­ing of this po­ten­tial­ly im­por­tant ther­a­py for peo­ple with Parkin­son’s.”

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 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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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)

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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This image shows a lab technician measuring the zone of inhibition during an antibiotic sensitivity test, 1972. The zone of inhibition is measured and compared to a standard in order to determine if an antibiotic is effective in treating the bacterial infection. (Gilda Jones/CDC via Getty Images)

Bio­phar­ma has aban­doned an­tibi­ot­ic de­vel­op­ment. Here’s why we did, too.

Timing is Everything
When we launched Octagon Therapeutics in late 2017, I was convinced that the time was right for a new antibiotic discovery venture. The company was founded on impressive academic pedigree and the management team had known each other for years. Our first program was based on a compelling approach to targeting central metabolism in the most dangerous bacterial pathogens. We had already shown a high level of efficacy in animal infection models and knew our drug was safe in humans.

Shehnaaz Suli­man dives back in­to Alzheimer's at Alec­tor; Pyx­is re­cruits Spring­Works founder Lara Sul­li­van as CEO

Amid Shehnaaz Suliman’s lengthy resume it could be easy to miss her stint leading early-stage Alzheimer’s R&D at Genentech, where she oversaw a program for the ill-fated crenezumab and initiated one of the first prevention studies around the devastating neurodegenerative disease. But it is this experience that she — after thinking long and hard about her next career move over the past months — will be leaning heavily on as the first president and COO of Alector.

PhII fail­ure in rare neu­rode­gen­er­a­tive dis­ease? No mat­ter, Bio­gen will mo­tor on in Alzheimer's

Biogen’s fierce focus on disorders of the brain has hit another roadblock.

On Friday, the US drugmaker — which recently resurrected its amyloid-targeting Alzheimer’s drug, aducanumab — said its anti-tau drug, gosuranemab, failed a mid-stage study in patients with progressive supranuclear palsy (PSP), a rare brain disorder that results from deterioration of brain cells that control movement and thought.

A USP­TO le­gal ad­vis­er is off con­tro­ver­sial Gilead HIV case af­ter ac­tivists al­lege tweets show bias

Last week, a top legal adviser in the US Patent and Trademark Office working on the high-profile Gilead HIV PrEP case tweeted at Sen Bernie Sanders (I-VT) “What proof????” and then at activists “Do facts even matter to you?”

Now, STAT reports, she’s off of the case.

Activists in the coalition PrEP4All filed a petition to the USPTO on December 9 asking longtime senior legal advisor Mary Till be removed from the Gilead case, saying her tweets showed a bias toward Gilead. PrEP4All requested earlier this month the agency reject Gilead’s three-year patent extension for TAF (tenofovir alafenamide), a component of one of the HIV prevention regimens often referred to as PrEP. They allege the pharma giant delayed developing the drug in order to “game” the system and hold off generics.