Roche has punted a Pompe disease gene therapy from its pipeline after weighing up the competitive landscape. The Swiss drugmaker made the change in a second-quarter update that also featured news of TIGIT trial closures and a commitment not to make “huge cuts” to the organization.
Spark Therapeutics began enrolling patients in a phase 1/2 trial of SPK-3006 shortly after becoming part of Roche. At one point, Spark was aiming to enroll 30 people in the study. However, the company halted enrollment in 2022 and later revealed only four patients joined the study. Roche tossed the program out of its pipeline as part of its second-quarter clear-out.
“The decision to discontinue the trial is not due to safety concerns, it was made as part of a strategic portfolio prioritization within Spark,” a spokesperson for Roche told Fierce Biotech via email. “An established standard of care exists for Pompe disease, and while there are no currently approved gene therapies for Pompe disease, investigational treatments are being evaluated by different organizations.”
Astellas and AskBio, a part of Bayer, have Pompe gene therapies in clinical trials. Enzyme replacement therapies such as Sanofi’s Lumizyme and Nexviazyme are the standard of care in the rare genetic disease.
SPK-3006, also known as RG6359, was the most advanced new molecular entity culled this quarter. The Swiss drugmaker also defenestrated three phase 1 programs. Anti-CD25 Treg depleter RG6292 landed on the discard pile. Roche previously stopped a monotherapy clinical trial but continued to study RG6292 in combination with its checkpoint inhibitor Tecentriq.
The Swiss drugmaker also stopped studying CD19xCD28 prospect RG6333 in combination with its approved bispecific Columvi in non-Hodgkin lymphoma as well as confirmed its previously disclosed retreat from a SHP2 inhibitor.
Roche used a call with the media to share an update on the fallout from the failure of its TIGIT drug candidate in a phase 2/3 non-small cell lung cancer (NSCLC) trial. Based on the setback, the company has decided to close two other studies in NSCLC, namely the phase 3 SKYSCRAPER-15 trial and the phase 2 SKYSCRAPER-05 study.
“The remaining tiragolumab studies have either already completed, are already fully enrolled or are almost fully enrolled. Those studies that are obviously already quite down the path, we're going to allow those to close out,” Roche Pharmaceuticals CEO Teresa Graham said.
Graham also shared an update on Roche’s work on obesity, noting the positive data the company has reported on from assets acquired from Carmot Therapeutics in recent weeks and looking forward to the start of phase 2 trials of injectable and oral candidates in 2024 and 2025, respectively.
Roche CEO Thomas Schinecker fielded a question about the cost-cutting initiatives underway at some other drugmakers. Schinecker said Roche’s head count has remained stable and, while resources could be shifted from less to more promising areas, no big changes are incoming.
“There will be no announcement of any big restructurings,” Schinecker said. “We don't believe in making huge cuts in the organization because we want our team to be focused and diligent on developing medicines, and any such distraction would not be something that we would want in the organization.”