Decades in the mak­ing, Ky­owa Hakko Kirin fi­nal­ly wins FDA ap­proval for add-on Parkin­son's ther­a­py

More than two decades af­ter Ky­owa Hakko Kirin kicked off the clin­i­cal eval­u­a­tion of its Parkin­son’s dis­ease drug, the Japan­ese phar­ma group has fi­nal­ly se­cured FDA ap­proval.

The drug, istrade­fylline, tar­gets the adeno­sine A2A re­cep­tor, which is lo­cat­ed in the basal gan­glia — un­der­stood to play a key role in con­trol­ling vol­un­tary move­ment. It was ap­proved by the FDA as an add-on treat­ment to lev­odopa/car­bidopa on Tues­day. The drug was cleared for use in Japan back in 2013.

The drug, brand­ed as Nouri­anz, was sanc­tioned to treat Parkin­son’s pa­tients on the ba­sis of four 12-week place­bo-con­trolled clin­i­cal stud­ies that in­clud­ed a to­tal of 1,143 par­tic­i­pants. In each tri­al, pa­tients giv­en Nouri­anz ex­pe­ri­enced a sta­tis­ti­cal­ly sig­nif­i­cant de­crease from base­line in dai­ly ‘off’ time com­pared to those on a place­bo, the FDA said.

How­ev­er, its path to the fin­ish line with the FDA was not smooth sail­ing — Ky­owa Hakko Kirin had orig­i­nal­ly ap­plied for ap­proval more than a decade pri­or and was hand­ed a re­jec­tion by the agency in 2008. But the Japan­ese group elect­ed to plow ahead with its de­vel­op­ment pro­gram, al­though da­ta was not al­ways pris­tine. For ex­am­ple, in late 2016, a 12-week Phase III tri­al test­ed two dos­es of the drug against a place­bo, show­ing a trend to­wards ef­fi­ca­cy in Parkin­son’s pa­tients — but the re­sults were not sta­tis­ti­cal­ly sig­nif­i­cant.

The class of drugs Nouri­anz be­longs to has a tur­bu­lent past. In 2013, late-stage da­ta on Mer­ck’s adeno­sine A2A re­cep­tor ag­o­nist, preladenant, sug­gest­ed the drug was no bet­ter than a place­bo — which caused the US drug­mak­er to walk away from the mol­e­cule. Acor­da spent $363 mil­lion to swal­low Bi­otie, and its adeno­sine A2A re­cep­tor an­tag­o­nist — tozadenant — in 2016, but de­cid­ed to shelve its de­vel­op­ment the year af­ter, in re­sponse to un­sa­vory safe­ty da­ta that blight­ed a late-stage tri­al. Late last year, how­ev­er, Acor­da fi­nal­ly scored ap­proval for its in­haled lev­odopa ther­a­py, In­bri­ja.

The first line of ther­a­peu­tic de­fense for Parkin­son’s dis­ease pa­tients is typ­i­cal­ly lev­odopa/car­bidopa. How­ev­er, the ef­fec­tive­ness of oral lev­odopa is lim­it­ed by its short half-life — it re­mains in plas­ma for on­ly up to four hours fol­low­ing a sin­gle dose, re­quir­ing pa­tients to take mul­ti­ple dos­es dai­ly to fight ‘off’ pe­ri­ods of de­cline in mo­tor and non-mo­tor func­tion. Ex­ces­sive/in­ter­mit­tent oral dos­es of lev­odopa of­ten lead to in­vol­un­tary move­ments, or dysk­i­ne­sia, in some pa­tients.

Near­ly one mil­lion Amer­i­cans will be liv­ing with Parkin­son’s by 2020 — more than the com­bined num­ber of peo­ple di­ag­nosed with mul­ti­ple scle­ro­sis, mus­cu­lar dy­s­tro­phy and Lou Gehrig’s dis­ease, es­ti­mates the non-prof­it Parkin­son’s Foun­da­tion.

So­cial im­age: Ky­owa Hakko Kirin

Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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Atul Deshpande, Harbour BioMed chief strategy officer & head, US operations (Harbour BioMedO

An­oth­er biotech IPO set-up? Multi­na­tion­al biotech leaps from round to round, scoop­ing up cash at a blis­ter­ing pace

A short four months after announcing a $75 million haul in Series B+ fundraising, the multinational biotech Harbour BioMed pulled in another round of investments and eclipsed the nine-digit mark in the process.

Harbour completed its Series C financing, the company announced Thursday morning, raising $102.8 million and bringing its total investment sum to over $300 million since its founding in late 2016. The biotech plans to use the money to transition early-stage candidates from the discovery phase, fund candidates already in the clinic, and prep late-stage candidates for commercialization.

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Covid-19 roundup: Af­ter promis­ing US its sum­mer sup­ply, Gilead in talks with EU over remde­sivir ac­cess; First in­haled remde­sivir study un­der­way

It wasn’t lost on European journalists or European doctors that the “amazing deal” the Trump Administration said it signed with Gilead to buy up remdesivir meant that they would have severely limited access to one of only two drugs proven to treat Covid-19. “This is the first major approved drug, and where is the mechanism for access?” Andrew Hill, a research fellow at Liverpool University, told The Guardian. “Once again we’re at the back of the queue.”

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UP­DAT­ED: Bio­gen shares spike as ex­ecs com­plete a de­layed pitch for their con­tro­ver­sial Alzheimer's drug — the next move be­longs to the FDA

Biogen is stepping out onto the high wire today, reporting that the team working on the controversial Alzheimer’s drug aducanumab has now completed their submission to the FDA. And they want the agency to bless it with a priority review that would cut the agency’s decision-making time to a mere 6 months.

The news drove a 10% spike in Biogen’s stock $BIIB ahead of the bell.

Part of that spike can be attributed to a relief rally. Biogen execs rattled backers and a host of analysts earlier in the year when they unexpectedly delayed their filing to the third quarter. That delay provoked all manner of speculation after CEO Michel Vounatsos and R&D chief Al Sandrock failed to persuade influential observers that the pandemic and other factors had slowed the timeline for filing. Actually making the pitch at least satisfies skeptics that the FDA was not likely pushing back as Biogen was pushing in. From the start, Biogen execs claimed that they were doing everything in cooperation with the FDA, saying that regulators had signaled their interest in reviewing the submission.

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Ed Engleman, (Stanford Blood Center)

Stan­ford star on­col­o­gy sci­en­tist Ed En­gle­man helped cre­ate the im­munother­a­py field. Now he wants to shake up neu­rode­gen­er­a­tion R&D

Over the last generation of drug R&D, Ed Engleman has been a standout scientist.

The Stanford professor co-founded Dendreon and provided the scientific insights needed to develop Provenge into a pioneering — though not particularly marketable — immunotherapy. He’s spurred a slate of startups, assisted by his well-connected perch as a co-founder of Vivo Capital, and took the dendritic cell story into its next chapter at a startup called Bolt.

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FDA bars the door — for now — against Mer­ck’s star can­cer drug af­ter Roche beat them to the punch

Merck has been handed a rare setback at the FDA.

After filing for the accelerated approval of a combination of their star PD-1 drug Keytruda with Eisai’s Lenvima as a first-line treatment for unresectable hepatocellular carcinoma, the FDA nixed the move, handing out a CRL because Roche beat them to the punch on the same indication by a matter of weeks.

According to Merck:

Ahead of the Prescription Drug User Fee Act action dates of Merck’s and Eisai’s applications, another combination therapy was approved based on a randomized, controlled trial that demonstrated overall survival. Consequently, the CRL stated that Merck’s and Eisai’s applications do not provide evidence that Keytruda in combination with Lenvima represents a meaningful advantage over available therapies for the treatment of unresectable or metastatic HCC with no prior systemic therapy for advanced disease. Since the applications for KEYNOTE-524/Study 116 no longer meet the criteria for accelerated approval, both companies plan to work with the FDA to take appropriate next steps, which include conducting a well-controlled clinical trial that demonstrates substantial evidence of effectiveness and the clinical benefit of the combination.

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Donald and Melania Trump watch the smoke of fireworks from the South Lawn of the White House on July 4, 2020 (via Getty)

Which drug de­vel­op­ers of­fer Trump a quick, game-chang­ing ‘so­lu­tion’ as the pan­dem­ic roars back? Eli Lil­ly and Ab­Cellera look to break out of the pack

We are unleashing our nation’s scientific brilliance and will likely have a therapeutic and/or vaccine solution long before the end of the year.

— Donald Trump, July 4

Next week administration officials plan to promote a new study they say shows promising results on therapeutics, the officials said. They wouldn’t describe the study in any further detail because, they said, its disclosure would be “market-moving.”

— NBC News, July 3

Something’s cooking. And it’s not just July 4 leftovers involving stale buns and uneaten hot dogs.

Over the long weekend observers picked up signs that the focus in the Trump administration may swiftly shift from the bright spotlight on vaccines being promised this fall, around the time of the election, to include drugs that could possibly keep patients out of the hospital and take the political sting out of the soaring Covid-19 numbers causing embarrassment in states that swiftly reopened — as Trump cheered along.

So far, Gilead has been the chief beneficiary of the drive on drugs, swiftly offering enough early data to get remdesivir an emergency authorization and into the hands of the US government. But their drug, while helpful in cutting stays, is known for a limited, modest effect. And that won’t tamp down on the hurricane of criticism that’s been tearing at the White House, and buffeting the president’s most stalwart core defenders as the economy suffers.

We’ve had positive early-stage vaccine data, most recently from Pfizer and BioNTech, playing catchup on an mRNA race led by Moderna — where every little sign of potential trouble is magnified into a lethal threat, just as every advance excites a frenzy of support. But that race still has months to play out, with more Phase I data due ahead of the mid-stage numbers looming ahead. A vaccine may not be available in large enough quantities until well into 2021, which is still wildly ambitious.

So what about a drug solution?

Trump’s initial support for a panacea focused on hydroxychloroquine. But that fizzled in the face of data underscoring its ineffectiveness — killing trials that aren’t likely to be restarted because of a recent population-based study offering some support. And there are a number of existing drugs being repurposed to see how they help hospitalized patients.

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Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

A new study points to $6.5B in pub­lic sup­port build­ing the sci­en­tif­ic foun­da­tion of Gilead­'s remde­sivir. Should that be re­flect­ed in the price?

By drug R&D standards, Gilead’s move to repurpose remdesivir for Covid-19 and grab an emergency use authorization was a remarkably easy, low-cost layup that required modest efficacy and a clean safety profile from just a small group of patients.

The drug OK also arrived after Gilead had paid much of the freight on getting it positioned to move fast.

In a study by Fred Ledley, director of the Center for Integration of Science and Industry at Bentley University in Waltham, MA, researchers concluded that the NIH had invested only $46.5 million in the research devoted to the drug ahead of the pandemic, a small sum compared to the more than $1 billion Gilead expected to spend getting it out this year, all on top of what it had already cost in R&D expenses.

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Roger Tung, Concert Pharmaceuticals CEO (Concert)

Con­cert gets BTD for alope­cia drug, set­ting up a late-stage show­down with gi­ant ri­val Pfiz­er

Concert Pharmaceuticals’ path to developing a drug that treats alopecia areata has been bumpy, but the pharma company scored a win Wednesday.

The FDA granted Concert a Breakthrough Therapy Designation (BTD) for its oral Janus kinase inhibitor, named CTP-543, paving the way for a Phase III study of the drug to begin in the fourth quarter of 2020. The news follows positive Phase II results from last September, which saw the drug meet its primary endpoint in both 8 mg and 12 mg twice-daily doses.