Imara at­tempts to spin weak da­ta in­to an 'en­cour­ag­ing' re­sult — but in­vestors aren't hav­ing it

Imara un­veiled da­ta from two sub­groups of a Phase IIa tri­al for its lead com­pound Wednes­day, aim­ing to con­jure up a pos­i­tive light de­spite chal­lenges tied to the Covid-19 pan­dem­ic. But in­vestors weren’t buy­ing it and sent the com­pa­ny’s shares in­to a tail­spin.

The ex­per­i­men­tal drug, dubbed IMR-687, is be­ing eval­u­at­ed for sick­le cell dis­ease as both a monother­a­py and in com­bi­na­tion with hy­drox­yurea. In the monother­a­py sub­group, Imara re­port­ed IMR-687 showed no mean­ing­ful changes in F-cells, fe­tal he­mo­glo­bin (HbF) lev­els or Hb lev­els from base­line af­ter 24 weeks.

Ad­di­tion­al­ly, in the com­bo sub­group, the can­di­date demon­strat­ed nu­mer­i­cal in­creas­es in F-cells and HbF lev­els from base­line, but Hb lev­els did not mean­ing­ful­ly change. But Imara was un­able to mea­sure the dif­fer­ence against a place­bo in both groups, as pa­tients in those arms could not be eval­u­at­ed due “in part” to missed study vis­its, the com­pa­ny said.

Ul­ti­mate­ly, on­ly one place­bo pa­tient in the com­bi­na­tion group had ap­pro­pri­ate bio­mark­er da­ta af­ter the 24-week pe­ri­od, and p-val­ues were not re­port­ed. Imara at­tempt­ed to spin those less-than-stel­lar re­sults in­to a win in a re­lease, but in­vestors saw through the ef­fort im­me­di­ate­ly.

“I am en­cour­aged by the in­cre­men­tal da­ta from this read­out, es­pe­cial­ly in light of the COVID-19 pan­dem­ic chal­lenges,” lead in­ves­ti­ga­tor Biree An­de­mari­am said in a state­ment. “This in­cludes a fa­vor­able safe­ty pro­file of IMR-687, low­er rate of VOCs/SCPCs and VOC-re­lat­ed hos­pi­tal­iza­tions in the Pop­u­la­tion A1 monother­a­py arm and im­prove­ments in sev­er­al bio­mark­er re­sults across both the monother­a­py and com­bi­na­tion groups.”

Imara $IM­RA shares were down 33% in ear­ly trad­ing Wednes­day.

The re­sults make up part of a 93-pa­tient Phase IIa tri­al, study­ing four dif­fer­ent dose lev­els across the monother­a­py and com­bi­na­tion reg­i­mens. Wednes­day’s monother­a­py da­ta come from a reg­i­men of a once-dai­ly dose of 100 mg through 4 weeks, which es­ca­lates to 200 mg through an ad­di­tion­al 20 weeks.

In the com­bi­na­tion group, pa­tients took a 50 mg dose once a day on top of stan­dard of care HU, with es­ca­la­tion af­ter 4 weeks to 100 mg for the re­main­ing 20 weeks. There were 18 to­tal in­di­vid­u­als in this monother­a­py co­hort and 14 in the com­bo por­tion.

Imara can still hang its hat on in­ter­im da­ta from the oth­er sub­groups in the study, which showed the high­er dose of the monother­a­py had sta­tis­ti­cal­ly sig­nif­i­cant in­creas­es in F-cells, as well as a dose-de­pen­dent in­crease in HbF lev­els in adults. There was al­so a mean in­crease from base­line of 1.7% in HbF per­cent­age in the 100 mg / 200 mg dose group through week 24, a fig­ure that SVB Leerink an­a­lyst Joseph Schwartz had deemed promis­ing.

Ahead of Wednes­day’s da­ta, Schwartz had been hop­ing to see a fur­ther rise in this spe­cif­ic da­ta point, re­gard­less of whether or not it reached the 3% lev­el the FDA set as the thresh­old for the Phase IIb study that launched in Au­gust. But the over­all per­cent change in monother­a­py re­port­ed Wednes­day was -1.1%, even though Imara not­ed an ab­solute, dose-de­pen­dent in­crease in HbF of 1.3% when pa­tients es­ca­lat­ed from 100 mg to 200 mg.

Schwartz wrote Wednes­day that he does not be­lieve the da­ta is a good in­di­ca­tor of the up­com­ing Phase IIb re­sults, and still be­lieves the 3% fig­ure is achiev­able. He al­so not­ed that the dos­es be­ing used in that study are much high­er — up to 400 mg — than those in Phase IIa, with signs point­ing to longer and high­er dos­es re­sult­ing in bet­ter ef­fi­ca­cy.

IMR-687 was one of a group of small mol­e­cule PDE9 in­hibitors that Imara had li­censed from Lund­beck. The the­o­ry is that block­ing PDE9 in­creas­es cyclic GMP lev­els, which is as­so­ci­at­ed with re­ac­ti­va­tion of fe­tal he­mo­glo­bin and thus re­store some func­tion­al­i­ty im­paired in blood dis­or­ders. Imara went pub­lic in March fol­low­ing a $75.2 mil­lion IPO raise.

This ar­ti­cle has been up­dat­ed to in­clude SVB Leerink’s Joseph Schwartz’s most re­cent analy­sis, pub­lished late Wednes­day morn­ing.

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Richard Pazdur (vis AACR)

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Karen Flynn, Catalent

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Yuval Cohen, Corbus CEO (Corbus via YouTube)

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Elizabeth Warren (Michael Brochstein/Sipa USA)(Sipa via AP Images)

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Two top Senate Finance committee senators on Thursday called for a hearing to examine the questions and challenges for Medicare arising from the FDA’s recent approval of Biogen’s Aduhelm, the controversial new drug approved to treat Alzheimer’s disease.

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