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.”

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.

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.