Alglucosidase Alfa

Roche decided to close two TIGIT studies. 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.”

Maze Therapeutics looks to have found another partner for its investigational Pompe disease drug MZE001 after a deal last year with Sanofi fell foul of antitrust regulators. In an agreement announced on Friday, Maze will receive $150 million upfront from Shionogi and is eligible for additional milestones payments and tiered sales royalties.Sanofi abandoned its plans to license the Maze molecule in December under a deal potentially worth $750 million, including $150 million upfront, after the US Federal Trade Commission (FTC) raised competition concerns. The French drugmaker already markets the Pompe disease enzyme replacement therapies Lumizyme (alglucosidase alfa), also known as Myozyme, and Nexviazyme (avalglucosidase alfa), which are both administered via biweekly intravenous infusions.Meanwhile, Shionogi is looking to strengthen its rare diseases portfolio as part of its revised medium-term business strategy. “The science behind MZE001 is differentiated and promising,” with the potential to serve as “both mono and add-on therapy to enzyme replacement therapies,” remarked Shionogi CEO Isao Teshirogi. MZE001, an oral inhibitor of GYS1, is designed to restrict glycogen accumulation in patients with Pompe disease. The drug’s Phase I data released in February last year confirmed target engagement, prompting Maze to finalise mid-stage development. As part of the deal for MZE001, Shionogi will also have global rights to related programmes and intellectual property.