Spark Therapeutics Stock Plunges On Hemophilia Therapy Data
Zacks Investment Research | Dec 11, 2017 10:15PM ET
Spark Therapeutics’ (NASDAQ:ONCE) shares plunged 35% on Dec 11 as investors were disappointed with the data from a phase I/II study evaluating its gene therapy candidate, SPK-8011, for treating hemophilia A.
Data on SPK-8011 looked less competitive than BioMarin Pharmaceutical Inc.’s (NASDAQ:BMRN) hemophilia A candidate, valoctocogene roxaparvovec (formerly BMN 270), which achieved better factor VIII levels. BioMarin had announced data on its candidate last week.
Shares of Spark Therapeutics have lost 4.4% so far this year, underperforming the industry ’s rise of 2.6% in that period.
The dose ranging phase I/II study evaluated doses of 5e11 vector genomes per kg (vg/kg) of body weight, 1e12 vg/kg and 2e12 vg/kg of SPK-8011
The data announced form the phase I/II study showed impressive reduction in overall annualized bleeding rate (“ABR”) and overall annualized infusion rate (“AIR”). The ABR was reduced by 100% at week four to 0 from 5.5 annualized bleeds before initiating administration of SPK-8011. Similarly, AIR was reduced to 1.2 annualized infusions from 57.8 before SPK-8011 administration, a reduction of 96%.
However, factor VIII levels achieved were not impressive and also not consistent. Two patients infused with 5e11 vg/kg achieved mean factor VIII activity levels of 10% and 16% after 12 weeks. Moreover, two patients infused with a dose of 1e12 vg/kg of SPK-8011. One of them achieved mean factor VIII activity level of 13% after 14 weeks and another patient achieved 9% after 19 weeks.
BioMarin’s valoctocogene roxaparvovec achieved mean factor VIII activity levels of 49% and 89% for two tested doses of the candidate. However, BioMarin tested higher doses of its candidate – 4e13 vg/kg and 6e13 vg/kg.
In a separate press release, Spark Therapeutics along with Pfizer Inc. (NYSE:PFE) announced data from another phase I/II study evaluating SPK-9001 for the treatment of patients with hemophilia B. Data from this study showed impressive reduction in ABR and AIR. Moreover, patients who were administered SPK-9001 had discontinued their regular factor IX concentrates infusions. They also showed sustained steady-state factor IX activity levels.
Hemophilia A is rare genetic disorder characterized by mutation in factor VIII gene, which is responsible for clotting of blood in the human body. The deficiency of factor VIII increases the risk of bleeding that can become potentially life threatening even in case of modest injuries.
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