Deep in talks with J&J, Acte­lion’s flag­ship drug cruis­es past a piv­otal tri­al fail­ure

Pro­fes­sor Naz­zareno Gal­iè, Uni­ver­si­ty of Bologna

Deep in­to ne­go­ti­a­tions with J&J over a prospec­tive buy­out, Acte­lion (SIX: ATLN) re­port­ed to­day that its up-and-com­ing flag­ship ther­a­py Op­sum­it flunked a piv­otal test for treat­ing pul­monary ar­te­r­i­al hy­per­ten­sion due to Eisen­menger Syn­drome. And while the tri­al fail­ure could put a crimp in Acte­lion’s — as well as J&J’s — plan to con­tin­ue to rapid­ly boost sales of Op­sum­it as Tr­a­cleer los­es ground to gener­ic com­pe­ti­tion, an­a­lysts seemed ready to write it off and move on.

In­ves­ti­ga­tors for the Swiss biotech say they tracked the kind of bio­mark­er im­pact they were look­ing for, but Op­sum­it (maci­ten­tan) did not im­prove pa­tients’ six-minute walk tests when com­pared to a place­bo. And they added that it was like­ly an un­usu­al place­bo re­sponse that caused the un­ex­pect­ed set­back.

In fact, the drug arm ac­tu­al­ly per­formed worse than the place­bo group.  Place­bo pa­tients gained an av­er­age of 19.7 me­ters from base­line com­pared to an in­fe­ri­or 18.3 me­ter gain in the Op­sum­it arm. If you wash out three pa­tients with miss­ing 6-minute re­sults, the im­prove­ment in the drug arm goes up to 30.2 me­ters.

Op­sum­it is al­ready ap­proved to treat PAH. But Acte­lion ex­ecs felt they had a good shot at boost­ing their mar­ket reach by go­ing af­ter the sick­est pa­tient pop­u­la­tion in PAH. These pa­tients suf­fer from ad­vanced PAH along with con­gen­i­tal heart dis­ease. And the pop­u­la­tion in­cludes a large num­ber of peo­ple suf­fer­ing from Down Syn­drome.

Jef­feries’ Pe­ter Welford, who es­ti­mates peak sales at $2.3 bil­lion, shrugged off the fail­ure. He not­ed:

We did not specif­i­cal­ly as­cribe sales or an NPV to Op­sum­it for this in­di­ca­tion but be­lieve the ad­verse im­pact is rel­a­tive­ly mi­nor. Es­ti­mates sug­gest up to 15k Eisen­menger pa­tients WW, <5% of the PAH pop­u­la­tion. Tr­a­cleer is ap­proved in Eu­rope to treat PAH due to Eisen­menger af­ter demon­strat­ing a sig­nif­i­cant +53m ben­e­fit on the 6-minute walk dis­tance (6MWD).

Welford al­so doubts that J&J will be se­ri­ous­ly put off by the study re­sults. The phar­ma gi­ant has been qui­et­ly en­gaged in ex­clu­sive talks with Acte­lion af­ter ini­tial­ly walk­ing away from ne­go­ti­a­tions.

In­vestors ap­par­ent­ly agreed with that as­sess­ment. Acte­lion’s shares moved up this morn­ing in an­tic­i­pa­tion of a deal with J&J.

Pro­fes­sor Naz­zareno Gal­iè, head of the Pul­monary Hy­per­ten­sion Cen­ter at the In­sti­tute of Car­di­ol­o­gy, Uni­ver­si­ty of Bologna and steer­ing com­mit­tee mem­ber for the MAE­STRO study, com­ment­ed:

The re­sults of the MAE­STRO study are very dif­fi­cult to in­ter­pret. We have seen en­cour­ag­ing pos­i­tive ef­fects of maci­ten­tan in the re­sponse of N-ter­mi­nal pro b-type na­tri­uret­ic pep­tide plas­ma lev­els and he­mo­dy­nam­ic mea­sures. Al­though the re­sults point to­wards a ben­e­fit of treat­ment with maci­ten­tan, we do not see a sig­nif­i­cant treat­ment ef­fect on the pri­ma­ry end­point of ex­er­cise ca­pac­i­ty as mea­sured in the 6 minute walk test. I be­lieve this has been in­flu­enced by an un­ex­pect­ed im­prove­ment in the place­bo arm of the study, which is un­usu­al in a pre­dom­i­nant­ly un­treat­ed PAH pop­u­la­tion. In fact, we have not seen such a per­sis­tent place­bo ef­fect in the mul­ti­ple stud­ies pub­lished so far in PAH. We need to ful­ly an­a­lyze the da­ta to un­der­stand what could have caused this phe­nom­e­non.

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Arie Belldegrun (Photo: Jeff Rumans for Endpoints News)

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Paul Biondi (File photo)

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After 17 years with Bristol-Myers Squibb, Paul Biondi, Head of Strategy and Business Development, decided to leave the company to pursue an external opportunity. The company wishes him well in his new endeavors. Bristol-Myers Squibb  is actively searching for Paul’s successor, and will make an announcement, as appropriate.

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Arie Belldegrun at UKBIO 2019. Shai Dolev for Endpoints News

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