The uncertainty around the future of Elevidys hasn’t destroyed Roche’s belief in the potential of gene therapies, the Swiss company’s head of pharma has told Fierce Biotech, as the pharma continues to expand its research reach outside of its traditional oncology focus.
Roche and its partner Sarepta Therapeutics both paused trials of Elevidys back in April following the death of a 16-year-old patient in the U.S. Since then, Roche—which markets the drug outside of the U.S.—has stopped all ex-U.S. distribution of the drug in the commercial setting for non-ambulatory patients, while the FDA requested that Sarepta halt Elevidys shipments in the U.S. Earlier this week, Roche paused shipments in all patients in countries where Elevidys’ approvals referenced the FDA.
But Roche Pharma CEO Teresa Graham denied Fierce’s suggestion that the issues around Elevidys have altered how the company views gene therapy as a modality.
“It doesn’t, actually,” Graham told Fierce in an interview at Roche’s headquarters in Basel, Switzerland on Thursday.
“The reality is that gene therapy will be an important modality in the treatment of human health,” Graham continued. “There 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.”
Even before the latest difficulties with Elevidys, Roche had announced a “fundamental reorganization” of Spark Therapeutics, the gene therapy unit the Swiss pharma bought for $4.3 billion in 2019. That overhaul closely followed Roche’s decision to end work on a hemophilia A gene therapy candidate, which was a focus of the Spark acquisition.
Roche’s 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, was sold for just $49 million upfront to private equity firms.
But Roche still sees potential in the modality, according to Graham.
“I am firmly convicted that over time we will crack gene therapy and we will be able to deliver really meaningful treatments to patients,” she said.
Graham spoke to Fierce on the sidelines of the company’s first-half earnings event, where Roche CEO Thomas Schinecker reminded journalists that 760 boys have successfully received Elevidys in the ambulatory setting.
“I'm really hopeful that we can find a path forward for this medicine,” Graham said in the interview on Thursday. “We clearly need to talk to regulators; we clearly need to have some good conversations about the safety—but we do firmly believe in the risk-benefit of this product.”
Not all regulators sound ready to be convinced, with the European Medicines Agency (EMA) announcing Friday morning that it won't recommend Elevidys for approval in the ambulatory setting. Roche said in a statement that it “plans to continue to work with the EMA to explore a potential path forward.”
Mixed results from emerging pipeline
Another drug that Roche is keeping faith in is the Parkinson’s disease prospect prasinezumab. The future of the Prothena Biosciences–partnered asset looked in doubt after the anti-alpha-synuclein antibody failed a phase 2 study in December 2024.
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 from an open-label portion of the study suggesting a clinical benefit on top of symptomatic treatment in early-stage Parkinson’s.
“Given the dramatic unmet need in Parkinson's, it does feel like it would be a miss not to explore this,” Graham explained.
“There'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,” she said.
“There'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,” Graham added.
Roche had less faith in CT-173, an early-stage obesity drug picked up as part of its $2.7 billion acquisition of Carmot Therapeutics in 2023. The Swiss pharma had previously touted the long-acting PYY analog’s potential to drive weight loss past the GLP-1 plateau, but Graham announced at the earnings event that Roche would no longer take CT-173 into human trials after assessing its “developability and competitiveness.”
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.
“Obesity is inherent in something like 200 other diseases,” she said. “So 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.”
“This is an incredibly big market,” Graham added. “It is very diverse. There are a lot of scientific pathways to explore here.”
One of those pathways is amylin, a hormone involved in the regulation of food intake. In March, Roche paid Zealand Pharma $1.6 billion for the long-acting amylin analog petrelintide. Graham sees the drug’s potential growing as the obesity market segments among patients “looking for maybe different depths of weight loss or different tolerability profiles.”
Meanwhile, Roche has yet to make a final call on whether to take the GLP-1/GIP receptor agonist CT-388—another Carmot asset—into a phase 3 obesity trial, but Graham said the company is “right on the cusp” of taking that program forward.
“We plan to make those decisions later this year, and, once we get rolling here, I think you're really going to see us flesh out our [obesity] strategy,” she said.
When it comes to bringing new drugs into Roche’s pipeline, Graham was tight-lipped on which of the company’s five core focus areas—neurology, oncology/hematology, immunology, ophthalmology and CVRM (cardiovascular renal metabolic)—the company is prioritizing for M&A.
“Clearly, within our five therapeutic areas, there are definitely places where we would like to bring in additional assets,” she told Fierce. “As Thomas [Schinecker] said, we look at an unbelievably large number of deals every year, and are constantly on the hunt, not only for things that will fit that strategy but things that are truly transformational.”
With such a multifaceted pipeline, what are the readouts Graham is most excited about?
“It's like trying to choose your favorite child,” she said, before name-checking the recent “super exciting” phase 2 results for the BTK inhibitor fenebrutinib in relapsing multiple sclerosis (MS). “It could be a really huge step forward for MS patients,” she explained.
There’s also the “high-risk, high-reward bet” on the Parkinson’s drug prasinezumab, said Graham, who ended by referencing the IgG1 monoclonal antibody afimkibart.
Along with CT-388 and the Alzheimer’s disease prospect trontinemab, afimkibart was one of the assets Roche fast-tracked as part of its reallocation of R&D resources.
“I'm an immunology girl at heart—it’s where I started my career,” Graham explained. “There's so much opportunity in the immunological space and so many diseases that we could potentially tackle with afimki.”