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.”

Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

Tes­la chief Elon Musk teams up with Covid-19 play­er Cure­Vac to build 'R­NA mi­cro­fac­to­ries'

Elon Musk has joined the global tech crusade now underway to revolutionize vaccine manufacturing — now aimed at delivering billions of doses of a new mRNA vaccine to fight Covid-19. And he’s cutting right to the front.

In a late-night tweet Wednesday, the Tesla chief announced:

Tesla, as a side project, is building RNA microfactories for CureVac & possibly others

That’s not a lot to go on. But the tweet comes a year after Tesla’s German division in Grohmann and CureVac filed a patent on a “bioreactor for RNA in vitro transcription, a method for RNA in vitro transcription, a module for transcribing DNA into RNA and an automated apparatus for RNA manufacturing.” CureVac, in the meantime, has discussed a variety of plans to build microfactories that can speed up the whole process for a global supply chain.

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George Yancopoulos (Regeneron)

Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Dan Gold, MEI Pharma CEO

De­vel­op­ment part­ners at MEI, Helsinn dump a high-risk PhI­II AML study af­ter con­clud­ing it would fail sur­vival goal

Four years after Switzerland’s Helsinn put $25 million of cash on the table for an upfront and near-term milestone to take MEI Pharma’s drug pracinostat into a long-running Phase III trial for acute myeloid leukemia, the partners are walking away from a clinical pileup.

The drug — an HDAC inhibitor — failed to pass muster during a futility analysis, as researchers concluded that pracinostat combined with azacitidine wasn’t going to outperform the control group in the pivotal.

Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

Pfizer and their partners at the mRNA specialist BioNTech have published the first glimpse of biomarker data from an early-stage study spotlighting the “robust immunogenicity” triggered by their Covid-19 vaccine, which is one of the leaders in the race to vanquish the global pandemic.

Researchers selected 45 healthy volunteers 18-55 years of age for the study. They were randomized to receive 2 doses, separated by 21 days, of 10 µg, 30 µg, or 100 µg of BNT162b1, “a lipid nanoparticle-formulated, nucleoside-modified, mRNA vaccine that encodes trimerized SARS-CoV-2 spike glycoprotein RBD.” Their responses were compared against the effect of a natural, presumably protective defense offered by a regular infection.

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No­var­tis los­es biosim­i­lar ap­peal as court up­holds a 31-year mo­nop­oly by Am­gen's En­brel

A new court ruling has strengthened Amgen’s grip on the IP estate around Enbrel, keeping biosimilars of the autoimmune and inflammatory drug at bay until 2029.

Novartis, the patent challenger, isn’t throwing in the towel yet. In a statement noting the failed appeal, its generics division Sandoz noted its reviewing options, “including potential appeal to US Supreme Court.”

It’s been almost four years since the FDA approved Erelzi, Sandoz’s copycat version of Enbrel. While sales of the Pfizer-partnered drug in the US — the market Amgen is in charge of — have dipped slightly during that time, it remains a solid megablockbuster with 2019 revenue slightly above $5 billion.

Douglas Love, Annexon CEO (Annexon)

IPO bound? A Bay Area biotech grabs a mega-round on the road to a piv­otal neu­rode­gen­er­a­tion pro­gram

South San Francisco-based Annexon has added $100 million to its cash reserves, along with a new roster of marquee investors backing their play on the classical complement pathway involved in neurodegeneration. And that may well fit the profile for an IPO — though right now everything seems to be working on that score.

Eighteen months after Bain and their syndicate partners put up $75 million to fuel clinical work, Annexon is back at the trough. And this time they’re adding Redmile Group for the lead role, with supporting investments from these new arrivals: BlackRock, Deerfield Management Company, Eventide Asset Management, Farallon Capital Management, Janus Henderson Investors and Logos Capital.

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Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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An ex­pe­ri­enced biotech is stitched to­geth­er from transpa­cif­ic parts, with 265 staffers and a fo­cus on ‘new bi­ol­o­gy’

Over the past few years, different teams at a pair of US-based biotechs and in labs in Japan have labored to piece together a group of cancer drug programs, sharing a single corporate umbrella with research colleagues in Japan. But now their far-flung operations have been knit together into a single unit, creating a pipeline with 10 cancer drug development programs — going from early-stage right into Phase III — and a host of discovery projects managed by a collective staff of some 265 people.

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