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.”

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Law pro­fes­sors call for FDA to dis­close all safe­ty and ef­fi­ca­cy da­ta for drugs

Back in early 2018 when Scott Gottlieb led the FDA, there was a moment when the agency seemed poised to release redacted complete response letters and other previously undisclosed data. But that initiative never gained steam.

Now, a growing chorus of researchers are finding that a dearth of public data on clinical trials and pharmaceuticals means industry and the FDA cannot be held accountable, two law professors from Yale and New York University write in an article published Wednesday in the California Law Review.

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Novavax CEO Stanley Erck at the White House in 2020 (Andrew Harnik, AP Images)

As fears mount over J&J and As­traZeneca, No­vavax en­ters a shaky spot­light

As concerns rise around the J&J and AstraZeneca vaccines, global attention is increasingly turning to the little, 33-year-old, productless, bankruptcy-flirting biotech that could: Novavax.

In the now 16-month race to develop and deploy Covid-19 vaccines, Novavax has at times seemed like the pandemic’s most unsuspecting frontrunner and at times like an overhyped also-ran. Although they started the pandemic with only enough cash to last 6 months, they leveraged old connections and believers into $2 billion and emerged last summer with data experts said surpassed Pfizer and Moderna. They unveiled plans to quickly scale to 2 billion doses. Then they couldn’t even make enough material to run their US trial and watched four other companies beat them to the finish line.

FDA of­fers scathing re­view of Emer­gent plan­t's san­i­tary con­di­tions, em­ploy­ee train­ing af­ter halt­ing pro­duc­tion

The FDA wrapped up its inspection of Emergent’s troubled vaccine manufacturing plant in Baltimore on Tuesday, after halting production there on Monday. By Wednesday morning, the agency already released a series of scathing observations on the cross contamination, sanitary issues and lack of staff training that caused the contract manufacturer to dispose of millions of AstraZeneca and J&J vaccine doses.

Brad Bolzon (Versant)

Ver­sant pulls the wraps off of near­ly $1B in 3 new funds out to build the next fleet of biotech star­tups. And this new gen­er­a­tion is built for speed

Brad Bolzon has an apology to offer by way of introducing a set of 3 new funds that together pack a $950 million wallop in new biotech creation and growth.

“I want to apologize,” says the Versant chairman and managing partner, laughing a little in the intro, “that we don’t have anything fancy or flashy to tell you about our new fund. Same team, around the same amount of capital, same investment strategy. If it ain’t broke, don’t fix it.”

But then there’s the flip side, where everything has changed. Or at least speeded into a relative blur. Here’s Bolzon:

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Emma Walmsley, GlaxoSmithKline CEO (Kevin Dietsch/Pool via CNP/Alamy)

Glax­o­SmithK­line hus­tles the 7th PD-1 past the fin­ish line with Jem­per­li. But how big will up­take be?

Everything came up sevens for GlaxoSmithKline on Thursday as the pharma notched the seventh PD-1 approval seven years after the first such drugs were OK’ed in Keytruda and Opdivo. But will it bring GSK good fortune?

The FDA granted accelerated approval to dostarlimab, to be branded Jemperli, to treat recurrent or advanced endometrial cancer in a specific subset of patients following platinum-based chemo. It’s a drug that came to GSK through its buyout of Tesaro, which it snapped up for $5.1 billion back in December 2018.

JP Gabriel, Ocugen

JP Gabriel watched from the bleach­ers as the pan­dem­ic raged. Now head of sup­ply chain at Ocu­gen, he's ready to bat

The world was in the middle of the most pressing public health risk his generation had ever seen, and JP Gabriel felt like he was sitting on the sidelines. As a VP of biologics and mRNA manufacturing at Ultragenyx, Gabriel watched from the sidelines as players like Pfizer/BioNTech and Moderna used mRNA tech to chase their own Covid-19 vaccines.

This month, Gabriel got the chance to get his hands dirty against the pandemic — but it won’t be with mRNA.

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Sen. Patty Murray (D-WA) (Graeme Sloan/Sipa USA/Sipa via AP Images)

Sen­a­tors to NIH: Do more to pro­tect US bio­med­ical re­search from for­eign in­flu­ence

Although Thursday’s Senate health committee hearing was focused on how foreign countries and adversaries might be trying to steal or negatively influence biomedical research in the US, the only country mentioned by the senators and expert witnesses was China.

Committee chair Patty Murray (D-WA) made clear in her opening remarks that the US cannot “let the few instances of bad actors” overshadow the hard work of the many immigrant researchers in the US, many of which have won Nobel prizes for their work. But she also said, “There is more the NIH can be doing here.”

Jenny Rooke (Genoa Ventures)

Ear­ly Zymer­gen in­vestor Jen­ny Rooke re­flects on 'chimeras' in biotech, what it takes to spot a $500M gem

When Jenny Rooke first heard of Zymergen back in 2014, she knew she was looking at something different and exciting. The Emeryville, CA biotech held the promise of blending biology and technology to solve a huge unmet need for cost-effective chemicals — of all things — and a stellar founding team to boot.

But back then, West Coast venture capitalists didn’t see in Zymergen the one thing they were looking for in a winning biotech: therapeutic potential. Rooke, however, saw an opportunity and made her bets. Seven years later, that bet is paying off in a big way.

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