The following is an update on the Spark and Roche gene therapy program.
“Together, Spark and Roche remain fully committed in our objective to meet the needs of the hemophilia community by developing gene therapies for hemophilia A that, with the lowest effective dose and optimal regimen to manage the immune system response, demonstrate safety, efficacy, durability and predictability, similar to the encouraging results seen with SPK-9001 in hemophilia B currently in Phase 3 clinical development.
In order to achieve this objective, we are continuing to optimize the dose and immune management regimen for both SPK-8011 and SPK-8016 in Phase 1/2 before proceeding to Phase 3. As a result of our ongoing activities to select the optimal dose and immune management regimen, we now expect to dose patients in a Phase 3 clinical study starting in 2021.
We have been particularly encouraged with the progress of participants treated with SPK-8011 in our Phase 1/2 clinical study in the first two cohorts (at the 5 x 1011 vg/Kg and 1 x 1012 vg/Kg doses). Spark has submitted an abstract and hopes to present these data at ISTH virtually in July.
As part of our program efforts, over the past two years Spark has also expanded its state-of-the-art gene therapy manufacturing capabilities to the scale required to meet clinical and potential commercial demand for hemophilia A.
Spark鈥檚 commitment to the hemophilia community extends beyond the development of potential gene therapies in hemophilia. We believe strongly in the importance of providing gene therapy education that empowers patients, caregivers, and advocates. For the past three-plus years we have been dedicated to creating and disseminating unbiased and balanced gene therapy education, as well as supporting organizations with this common goal in mind. Our dedicated advocacy team continues to work closely with the community and we invite you to visit www.hemophiliaforward.com to advance your knowledge about gene therapy, be informed and feel empowered.”