Clinical Trials

Date: 2017-12-06

Type of information: Presentation of results at a congress

phase: 1-2

Announcement: presentation of results at the 59th American Society of Hematology (ASH) Annual Meeting in Orlando

Company: Spark Therapeutics (USA - PA)

Product: SPK-8011

Action mechanism:

• gene therapy. SPK-8011 was developed using Spark's proprietary technology platform and uses Spark200, a novel bio-engineered adeno-associated virus (AAV) capsid optimized for more efficient transduction of human hepatocytes, and contains an optimized B-domain deleted FVIII expression cassette. Spark retains global commercialization rights to its SPK-FVIII program for hemophilia A.

Disease: hemophilia A

Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases

Country:

Trial details:

Latest news:

• • On December 11, 2017, Spark Therapeutics announced that it has dosed seven participants in the Phase 1/2 clinical trial of SPK-8011 in hemophilia A. The first four participants, who have been followed at least 12 weeks post infusion, have reduced their overall annualized bleeding rate (ABR), calculated based on data after week four, by 100 percent (calculated based on data after week four; 82 percent based on data after infusion) to a mean of 0 (1) annualized bleeds as of the data cutoff, compared to a mean of 5.5 annualized bleeds before a single administration of SPK-8011. Similarly, their overall annualized infusion rate (AIR) was reduced approximately 98 percent (calculated based on data after week four; 96 percent based on data after infusion) to a mean of 1.2 (2.5) annualized infusions as of the data cutoff, compared to a mean of 57.8 annualized infusions before SPK-8011 administration. No serious adverse events have been observed to date. These data were presented by Lindsey A. George at the 59th American Society of Hematology (ASH) Annual Meeting and Exposition in Atlanta.
• As of the Dec. 6, 2017, data cutoff, two participants have received a single administration of SPK-8011 at an initial dose of 5 x 10¹¹ vector genomes (vg)/kg body weight and have now been followed for more than 40 weeks and 30 weeks. During this time, participant one has achieved sustained expression with a mean factor VIII activity level after week 12 of 10 percent (range as of the data cutoff: 7 to 11 percent).
• Participant two has shown different kinetics of factor VIII expression. His factor VIII level, as of the data cutoff, is 37 percent, and the mean factor VIII level since week 12 is 16 percent (6 to 37 percent).
• Two additional participants, infused at a dose of 1 x 10¹² vector genomes (vg)/kg body weight, have also achieved therapeutic levels of factor VIII. They have now been followed for 19 weeks and 14 weeks and have mean sustained factor VIII activity levels of 9 percent (7 to 12 percent) and 13 percent (7 to 24 percent) of normal, respectively, as of the data cutoff. Both participants have completed a precautionary tapering course of corticosteroids.
• Three more participants have been infused, one at the 1 x10¹² vector genomes (vg)/kg body weight dose and two at a dose of 2 x 10¹² vector genomes (vg)/kg body weight. Results for these three participants are too early to report as of the data cutoff.
• To date, none of the seven infused participants has reported a serious adverse event, including no factor VIII inhibitors, no thrombotic events and no factor VIII activity levels that may increase risk of thrombosis.

Is general: Yes