预约演示

Roche drops antisense drug after seeing efficacy data, leaving Biogen, Ionis and Ultragenyx to duke it out

2023-06-23

临床1期寡核苷酸

Evercore ISI analysts said Roche’s decision is good overall for Ultragenyx.

Roche has bowed out of the Rocheto develop an antisense oligonucleotide (ASO) for Angelman syndrome. After getting a look at early efficacy data, the Swiss drugmaker opted against advancing the candidate, switching its Angelman focus to another asset and leaving the ASO space to Biogen, Ionis Pharmaceuticals and Ultragenyx.

Roche-based Roche was one of a small band of drug developers to hit on the idea ofAngelman syndromeost expression of UBE3A, the neuronal E3 ligase that is at the root of the severe neurodevelopmental disorder. A phase 1 clinical trial of Roche’s ASO, rugonersen, began in 2020, buBiogen iIonis PharmaceuticalscandiUltragenyxget under the Swiss drugmaker.

“Roche has tRochethe decision not to progress rugonersen into the next stage of clinical development,” a spokespersonUBE3A. “The molecule was well tolerated and had an acceptable saneurodevelopmental disorderitted to finding the best possRochesolution for rugonersen and people living with Angelman syndrome.”

NRochef Roche’s decision emerged via the patierugonersenty, with FAST, an Angelman advocacy group, putting out a blog post after learning of the revised development plan for rugonersen. In the poRocheAST said Roche saw “encouraging effects” from electroencephalogram testing but the changesAngelman syndromes criteria for taking the asset forward. Roche will continue an open-label extension study and is looking to partner the molecule.

After heRoche the news, analysts at Evercore ISI spoke to Ultragenyx and, in a note to investors, said “they always thought Roche’s ASO didn't have enough potency and in our recent meeting with them wondered aRoche[the] future of the molecule.” Patients are already contacting Ultragenyx about switching to a trial of its ASO, according to the analRoche and transferring “should be okay with the right washout period.”

Ultragenyx’s GTX-102 is one of two UBE3A ASO options open Ultragenyxn patients who want to switch from Roche’s rugonersen toRochemilar molecule. BIIB121, an Ionis candidate that Biogen has the option to license, is the other ASO. The candidates are in phase 1 trials that are sUltragenyxo wrap up in 2025, years later than originally planned for both GTX-102 and BIIB121, also known as ION582.

Ultragenyxhe GTX-102e ISI analysts UBE3A ASOe’s decision is good for Ultragenyx because it means less cRocheition, but they acknowledged it is imBIIB121e to rule out that the effBiogenlimitations of rugonersen are a classwide problem that will affect GTX-102 and BIIB121. Ultragenyx and GeneTx, its partner on GTX-102, shared some data on their ASO laGTX-102, butBIIB121lease left many ION582ons unanswered.

At Roche, Angelman attention now turns tRochegabat, a GABA modulator Ultragenyx into phase 2 late last year. Roche, which currently lists one recruiting location, is activating sites, according to FArugonersen GTX-102 BIIB121 Ultragenyx GeneTx GTX-102

更多内容，请访问原始网站

文中所述内容并不反映新药情报库及其所属公司任何意见及观点，如有版权侵扰或错误之处，请及时联系我们，我们会在24小时内配合处理。

机构