Struggling Marinus gets a bump out of a positive PhII snapshot

A lit­tle more than six months since its stock price was crushed by a failed late-stage pro­gram for its lead­ing — and only — drug, tiny biotech mi­cro­cap Mar­i­nus $MRNS saw its shares shoot up this morn­ing after it of­fered a pos­i­tive Phase II snap­shot on a hand­ful of pa­tients who suf­fer from a rare ge­netic ail­ment.

In­ves­ti­ga­tors flipped the first card of its Phase II hand for ganax­olone in treat­ing CDKL5, which trig­gers se­vere seizures and neu­ro­log­i­cal dam­age among the chil­dren af­flicted by it. One of four pa­tients had to drop out from a lack of re­sponse, but in­ves­ti­ga­tors say that the three oth­ers ex­pe­ri­enced a re­duc­tion in seizures rang­ing from 52% to 88%.

Pre­sent­ing data at the an­nual meet­ing of the Amer­i­can Epilepsy So­ci­ety, re­searchers also added seizure re­sponses and EEG mea­sure­ments for three pa­tients — one with CDKL5 and two with PCD­H19 pe­di­atric epilepsy — un­der­scor­ing a pos­i­tive re­sponse in cut­ting the rate of seizures from base­line.

In­vestors liked what they saw, dri­ving up shares about 40% in morn­ing trad­ing. It was a dif­fer­ent story last June, when the com­pany’s shares dived on the news that ganax­olone had failed a late-stage study for adult focal onset seizures. Its mar­ket cap at the end of day on Fri­day was a frag­ile $22 mil­lion.

It’s early days yet for this study, which is de­signed to en­roll up to 10 pa­tients with each of CDKL5 dis­or­der, Lennox Gas­taut Syn­drome and PCD­H19 pe­di­atric epilepsy.

The drug de­vel­op­ment pro­gram here is sim­i­lar to what Sage has been work­ing on, with a focus on sta­tus epilep­ti­cus as well as post­par­tum de­pres­sion.

Michael G. Chez, the di­rec­tor of Pe­di­atric Neu­rol­ogy Re­search and Pe­di­atric Epilepsy, Sut­ter Neu­ro­science In­sti­tute, said:

“I am im­pressed with the re­spon­der rate and mag­ni­tude of seizure con­trol seen with ganax­olone in the ini­tial CDKL5 pa­tients. The CGIs (clin­i­cal global im­pres­sion scales) are con­sis­tent with seizure con­trol, with re­spon­ders show­ing ‘much im­proved’ under this scale. I look for­ward to fur­ther eval­u­at­ing these chil­dren and see­ing the final re­sults.”

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