Roche Drops Pompe Gene Therapy Program
The company acquired the program, along with Spark Therapeutics’ pipeline, in December 2019.
Roche has discontinued development of its investigational gene therapy SPK-3006, which was being evaluated in the phase 1/2 RESOLUTE trial (NCT04093349) in participants with late-onset Pompe disease.1
Roche adopted the program into its pipeline along with its acquisition of Spark Therapeutics in December 2019.2 The trial dosed its first participant in February 2021 and no data updates have been shared from the trial since.3
The quiet discontinuation comes a year after the company made a similar move with another of its acquired programs from Spark, SPK-8016 gene therapy, which was also being evaluated in a phase 1/2 trial (NCT03734588) for participants with hemophilia A and Factor VIII inhibitors.4 Spark now has 1 gene therapy remaining in its pipeline, SPK-8011 (dirloctocogene samoparvovec), which previously showed some promise in participants with hemophilia A in a phase 1/2 trial (NCT03003533) and which is currently being evaluated in the phase 3 KEYSTONE 1 trial (NCT06297486).5 Notably, Spark also collaborated on the development of fidanacogene elaparvovec, Pfizer’s gene therapy approved for treating moderate to severe hemophilia B in April 2024.6
READ MORE: News and Expert Insights in Pompe Disease Gene Therapy for International Pompe Day
“With up to 5 years of follow-up after a single infusion of SPK-8011 we see a result of durable FVIII expression with the majority of participants experiencing levels in the mild hemophilia range. Investigations are ongoing to overcome the challenge of managing a presumed capsid immune response to further reduce variability in FVIII expression following AAV-mediated gene transfer for hemophilia A,” Stacy Croteau, MD, MMS, Medical Director, Boston Hemophilia Center, and Assistant Professor of Pediatrics, Harvard Medical School, said while presenting the phase 1/2 trial data on SPK-8011.5
Roche’s withdrawal from the Pompe space leaves behind active players such as Astellas Pharma and Actus Bio, which are developing the gene therapies AT845 and ACTUS-101, respectively.
AT845 is currently being evaluated in the phase 1/2 FORTIS clinical trial (NCT04174105), in which 4 participants with LOPD have demonstrated stable forced vital capacity over time following treatment with AT845, including after enzyme replacement therapy (ERT) withdrawal in 3 participants as of February 2023.7
ACTUS-101 is being evaluated for the same indication in another phase 1/2 clinical trial (NCT03533673), the most recent data from which were presented in May 2022. All participants met criteria for ERT withdrawal at week 24 and continued to meet criteria to remain off ERT at weeks 52 and 104, although 1 participant chose to resume ERT at week 97.8
