Acor­da writes off an­oth­er R&D pro­gram as Ampyra flops in stroke study

Ron Co­hen

Six months af­ter Acor­da was forced to write off one of its drug can­di­dates in the wake of a tri­al flop, the biotech is back with more bad news for in­vestors. Its fran­chise drug Ampyra (dal­fam­pri­dine) failed a piv­otal study for help­ing stroke vic­tims walk bet­ter. And Acor­da is writ­ing the ef­fort off as a los­er.

A lit­tle more than three years ago Acor­da $ACOR was stoked by ev­i­dence of suc­cess in a small study of the same pa­tient pop­u­la­tion. But an analy­sis of the da­ta this time showed that the drug es­sen­tial­ly lined up with a place­bo when put to a ma­jor test.

Acor­da’s shares were dent­ed again by the news, drop­ping 13% in pre-mar­ket trad­ing. Their shares have lost half their val­ue over the course of the past 12 months.

Eval­u­at­ing da­ta from 368 pa­tients in the study, 23 of 121 pa­tients tak­ing 10 mg dal­fam­pri­dine, 17 of 121 pa­tients tak­ing 7.5 mg of the drug and 17 of 126 tak­ing a place­bo demon­strat­ed at least a 20% im­prove­ment in a two-minute walk test. That may have re­flect­ed a slight drug ef­fect, but it wasn’t near­ly good enough for an ex­pand­ed in­di­ca­tion.

Back in May Acor­da was al­so forced to fi­nal­ly punt the epilep­sy pro­gram for Plumi­az, re­ject­ed in 2013, af­ter the nasal spray for­mu­la­tion for di­azepam failed a bioe­quiv­a­lence study. Plumi­az was one of the most ad­vanced R&D ef­forts at Acor­da. And while it was con­sid­ered less po­ten­tial­ly lu­cra­tive than the in­haled lev­odopa ther­a­py CVT-301 ($500 mil­lion peak sales es­ti­mate by the com­pa­ny) and tozadenant ($400 mil­lion peak), the biotech had made it a key part of its $1 bil­lion-plus plan to boost rev­enue by a block­buster mea­sure of suc­cess.

Acor­da CEO Ron Co­hen, BIO’s chair, ac­knowl­edged the most re­cent fail­ure and then used it as an ex­am­ple of the high-risk na­ture of drug de­vel­op­ment — a com­mon theme of his as the in­dus­try or­ga­ni­za­tion looks to steer the cur­rent de­bate over drug pric­ing to­ward fa­vor­able treat­ment for biotech in­no­va­tion.

“We are dis­ap­point­ed by this out­come. The study in­di­cat­ed there was ac­tiv­i­ty re­lat­ed to walk­ing in peo­ple with PSWD, as sug­gest­ed by the pri­or Phase 2 study, but over­all this was not suf­fi­cient­ly clin­i­cal­ly mean­ing­ful. I want to ex­press our grat­i­tude to the study par­tic­i­pants, their care part­ners and clin­i­cians, who gave their time and com­mit­ment to this re­search. This out­come un­der­scores the risks that com­pa­nies in the bio­phar­ma­ceu­ti­cal in­dus­try must take in or­der to de­vel­op in­no­v­a­tive med­i­cines. Over the past three years, we have suc­cess­ful­ly di­ver­si­fied our pipeline port­fo­lio to ac­count for this risk. We plan to fo­cus R&D re­sources on de­vel­op­ing our promis­ing late-stage Parkin­son’s dis­ease ther­a­pies, CVT-301 and tozadenant, as well as ad­vanc­ing our ear­li­er stage as­sets, CVT-427 in mi­graine, SYN120 in Parkin­son’s dis­ease de­men­tia, and rHIgM22 in MS.”

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
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Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

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Over the last four years, we’ve honored 80 women whose extraordinary accomplishments have changed the game in biopharma R&D. You can now nominate someone to be highlighted in this year’s special report. Details are here.

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No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

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Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

Stuart Peltz, former PTC Therapeutics CEO

Stu­art Peltz re­signs as PTC Ther­a­peu­tics CEO af­ter 25 years

Stuart Peltz, the longtime CEO of PTC Therapeutics who’s led the rare disease drug developer since its founding 25 years ago, is stepping down.

Succeeding him in the top job is Matthew Klein, who joined PTC in 2019 and was promoted to chief operating officer in 2022. In a call with analysts, he said the CEO transition has been planned for “quite some time” — in fact, as part of it, he gave the company’s presentation at the JP Morgan healthcare conference earlier this year.

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