But Roche Pharma CEO Teresa Graham denied Fierce\u2019s suggestion that the issues around Elevidys have altered how the company views gene therapy as a modality.<\/p>\n
\u201cIt doesn\u2019t, actually,\u201d Graham told Fierce in an interview at Roche\u2019s headquarters in Basel, Switzerland on Thursday.<\/p>\n
\u201cThe reality is that gene therapy will be an important modality in the treatment of human health,\u201d Graham continued. \u201cThere is a lot of promise in gene therapy. I think what the entire industry is learning is that, scientifically, it’s just a little harder than we might have thought.\u201d<\/p>\n
<\/a><\/p>\n Roche<\/p>\n Fierce attending Roche’s earnings event at the company’s headquarters in Basel, Switzerland. Even before the latest difficulties<\/a> with Elevidys, Roche had announced a \u201cfundamental reorganization\u201d of Spark Therapeutics, the gene therapy unit the Swiss pharma bought for $4.3 billion in 2019.\u00a0That overhaul closely followed Roche\u2019s decision to end work<\/a> on a hemophilia A gene therapy candidate, which was a focus of the Spark acquisition.<\/p>\n Roche\u2019s move came against a backdrop of wider industry retreat from gene therapy, with Pfizer pulling its FDA-approved hemophilia B treatment Beqvez. Bluebird bio, a gene therapy pioneer once valued at $10 billion at its peak,\u00a0was sold for just $49 million<\/a>\u00a0upfront to private equity firms.<\/p>\n But Roche still sees potential in the modality, according to Graham.<\/p>\n \u201cI am firmly convicted that over time we will crack gene therapy and we will be able to deliver really meaningful treatments to patients,\u201d she said.<\/p>\n Graham spoke to Fierce on the sidelines of the company\u2019s first-half earnings event, where Roche CEO Thomas Schinecker reminded journalists that 760 boys have successfully received Elevidys in the ambulatory setting.<\/p>\n \u201cI’m really hopeful that we can find a path forward for this medicine,\u201d Graham said in the interview on Thursday. \u201cWe clearly need to talk to regulators; we clearly need to have some good conversations about the safety\u2014but we do firmly believe in the risk-benefit of this product.\u201d<\/p>\n Not all regulators sound ready to be convinced, with the European Medicines Agency (EMA) announcing Friday morning<\/a> that it won’t recommend Elevidys for approval in the ambulatory setting. Roche said in a statement that it \u201cplans to continue to work with the EMA to explore a potential path forward.\u201d Mixed results from emerging pipeline Another drug that Roche is keeping faith in is the Parkinson\u2019s disease prospect prasinezumab. The future of the Prothena Biosciences\u2013partnered asset looked in doubt after the\u00a0anti-alpha-synuclein antibody failed a phase 2 study<\/a> in December 2024.<\/p>\n But Roche is giving prasinezumab a second chance, with plans to launch a phase 3 trial by the end of the year off the back of an analysis of data from the failed study. Specifically, the pharma pointed to longer-term follow-up data<\/a> from an open-label portion of the study suggesting a clinical benefit on top of symptomatic treatment in early-stage Parkinson\u2019s.<\/p>\n \u201cGiven the dramatic unmet need in Parkinson’s, it does feel like it would be a miss not to explore this,\u201d Graham explained.<\/p>\n \u201cThere’s only really been minor improvements in treatments for these patients over the last 50 years, and, in every conversation I’ve had with [key opinion leaders], they have really encouraged us to take this forward,\u201d she said.<\/p>\n \u201cThere’s a little bit of a finger feel that there’s something there, and we’re only really ever going to be able to pull the signal out in a phase 3,\u201d Graham added.<\/p>\n Roche had less faith in CT-173, an early-stage obesity drug picked up as part of its $2.7 billion acquisition<\/a> of Carmot Therapeutics\u00a0in 2023. The Swiss pharma had previously touted<\/a> the long-acting PYY analog\u2019s potential to drive weight loss past the GLP-1 plateau, but Graham announced at the earnings event<\/a> that Roche would no longer take CT-173 into human trials after assessing its \u201cdevelopability and competitiveness.\u201d<\/p>\n Despite this early-stage setback, Graham still feels that combinations offer Roche a path to carving out its own corner of the increasingly crowded obesity space.<\/p>\n \u201cObesity is inherent in something like 200 other diseases,\u201d she said. \u201cSo the ability to actually look at where we believe our portfolio in combination with other drugs can really help address some of these comorbidities is one of the more exciting pieces.\u201d<\/p>\n \u201cThis is an incredibly big market,\u201d Graham added. \u201cIt is very diverse. There are a lot of scientific pathways to explore here.\u201d<\/p>\n
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\n (James Waldron)<\/p>\n
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