Five months after taking a hit on its decision to shelve a mid-stage program for their lead therapy as a monotherapy for paroxysmal nocturnal hemoglobinuria, Achillion $ACHN is back with more encouraging proof-of-concept data for the drug as a therapy for the anemia that can beset patients taking the industry standard drug Soliris.
Investigators say they tracked a sharp drop in the need for transfusions among Soliris patients suffering from anemia. The group needed just 1 transfusion with 2 units of blood during the 24-week study period, compared to the 34 transfusions and 58 units needed in the 24 weeks before they began treatment with the add-on therapy. They also noted increases in mean hemoglobin of approximately 2 g/dL at week 4 (n=11).
Investors liked the change of tune, early as it may be in the development process. Achillion shares surged 12% in pre-market trading as the biotech plotted a pivotal course ahead.
“After completion of regulatory discussions, we hope to initiate a Phase III PNH combination trial of ACH-4471 with C5 inhibitors in the first half of 2020,” stated Steven Zelenkofske, the chief medical officer at Achillion.
Longtime industry observers will recall that Achillion was once a star player in the hep C field, tapped by the punters as a likely takeover target. Then Gilead came up with its painless cure, cleaned up with billions of revenue and then watched the market shrivel as the patient pool dwindled.
That left Achillion looking for greener fields, with a pivot to ACH-4471 as its new pipeline hopeful.
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