AveXis is repeating previous preclinical work and adding additional studies under new leadership to ensure new quality controls to support the planned IND, she noted.
Rett syndrome (RTT) is a genetic brain disorder that typically becomes apparent after 6 to 18 months of age in females. Symptoms include problems with language, coordination and repetitive movements.
There will be a “better understanding of these studies” in the middle of 2020, and Novartis plans to provide an update on the programme at that time, she said. The IND had been planned for submission in 1Q19, according to Novartis’ 4Q18 presentation made on 30 January 2019. A subsequent 1Q19 presentation referred to the program as being in “early clinical development,” and Novartis’ 2Q19 US Securities and Exchange Commission (SEC) filing on 18 July described the programme as in Phase I.
Novartis completed its acquisition of AveXis in May 2018 for $8.7bn and successfully brought AveXis’ most advanced asset, Zolgensma (onasemnogene abeparvovec-xioi), past US Food and Drug Administration (FDA) approval on 24 May 2019 for spinal muscular atrophy.
Recently, though, the $2.1m gene therapy has been in the headlines again after the FDA announced on 6 August that Novartis had alerted the agency to manipulation of preclinical data in the BLA, which the FDA started investigating. The FDA statement noted it stands behind Zolgensma’s approval but may take action against Novartis in the form of civil or criminal penalties.
On 14 August, Novartis announced the appointment of Page Bouchard as senior vice-president of research and chief scientific officer of AveXis as of 5 August. These positions were previously held by Allan Kaspar and Brian Kaspar, respectively, who were dismissed in May 2019.
As a gene therapy, AVXS-201 has the potential to be a disease-modifying treatment, while only symptomatic treatments are available now. It was granted orphan drug designation on 17 April, according to the FDA database. Existing preclinical data, presented in May at the American Academy of Neurology 2019 annual meeting, showed that multiple doses of the AAV9 gene therapy expressing methyl CpG-binding protein 2 improved survival in MeCP2-knockout mice by over 300% (Power, et al, AAN 2019 S51.002). Novartis has a market capitalisation of $ 222.3bn.
by Shuan Sim in New York
Shuan Sim is a Senior Reporter for Clinical Trials Arena parent company GlobalData’s investigative journalism team. A version of this article originally appeared on the Insights module of GlobalData’s Pharmaceutical Intelligence Center. To access more articles like this, visit GlobalData.