Novartis announced it will present 52 abstracts from across its unique neurological portfolio and pipeline at the upcoming 71stAmerican Academy of Neurology (AAN) meeting May 4 – 10 in Philadelphia, Pennsylvania, USA. The breadth and depth of the data highlights the company’s commitment to decoding the science behind neurological diseases such as spinal muscular atrophy (SMA), multiple sclerosis (MS) and migraine.
“Our presence at AAN this year is a testament to how we are reimagining medicine across a broad spectrum of neurological diseases, for many patients of all ages,” said Danny Bar-Zohar, Global Head, Neuroscience Development for Novartis Pharmaceuticals. “Nothing is more rewarding than combining cutting-edge science, advanced algorithms and technology with a relentless ambition to bring life-changing solutions to people who need them.”
Novartis highlights at AAN include:
Spinal muscular atrophy[1]-[12]:
- Four new scientific abstracts spanning the clinical development program for the investigational gene therapy Zolgensma® (onasemnogene abeparvovec-xioi; AVXS-101) show positive results in the treatment of SMA.
- Interim data presented for the first time from the Phase I STRONG trial demonstrated encouraging results via the intrathecal delivery in patients with SMA Type 2.
- New interim data from the Phase III STR1VE study demonstrated survival and motor function improvements that continue to parallel the Phase I START study.
- Preliminary data from the Phase III SPR1NT trial demonstrated significant motor function improvements in pre-symptomatic SMA patients.
- Data from the ongoing long-term follow-up study for the Phase I START trial show no waning of motor milestone achievements in patients with SMA Type 1.
- Three health economics studies demonstrated improved survival and milestone achievement with Zolgensma.
- New data supporting the potential use of serum neurofilament light chain as biomarker for SMA Type 1 disease activity and therapy response under branaplam (LMI070), an oral, once-weekly RNA splicing modulator.
Zolgensma was granted Priority Review for the treatment of SMA Type 1 by the FDA and regulatory action is anticipated in May 2019.
Multiple Sclerosis[12],[13], [23]-[38]:
- New analyses from the Phase III EXPAND study show Mayzent® (siponimod) had a sustained benefit on cognition in secondary progressive MS (SPMS) patients. Impaired cognitive function is a key aspect of disability associated with MS. It substantially affects the social and professional lives of people with MS and their families. Mayzent was approved by US Food and Drug Administration (FDA) in March 2019 for the treatment of adults across the spectrum of relapsing multiple sclerosis, including SPMS with active disease*. It is the only FDA-approved treatment for active SPMS, based on a positive pivotal study in a typical SPMS population.
- Presented for the first time, the full results from the Phase III ASSESS study, a direct head-to-head trial comparing Gilenya® (fingolimod) vs. glatiramer acetate patients with RRMS. Top line results announced in October 2018 showed treatment with Gilenya 0.5mg demonstrated superior efficacy in significant reduction of annualized relapse rate, with nearly 41% fewer relapses versus glatiramer acetate, as well beneficial effects on other key measures of disease activity.
- MSProDiscussTM, the first-of-its-kind algorithm-based tool developed side-by-side with renowned MS researchers, healthcare professionals and patients, to facilitate doctor-patient discussions around MS progression.
- Scientific explorations of ofatumumab, the first fully human anti-CD20 monoclonal antibody with a monthly self-administered subcutaneous dosing regimen tailored for RMS, allowing for the potential preservation of immune function.
- Furthermore, new analyses supporting neurofilaments as an increasingly used biomarker in MS clinical trials will be highlighted. Utilizing blood neurofilaments as an easy-to-use biomarker for MS could revolutionize the way MS treatments are assessed in clinical trials and ultimately, in clinical practice. Novartis is leading the field with 18 data analyses presented so far.
*Mayzent was approved by the US FDA in March 2019 for the treatment of adults with relapsing forms of multiple sclerosis, including SPMS with active disease, relapsing remitting multiple sclerosis (RRMS) and clinically isolated syndrome (CIS). Clinically isolated syndrome (CIS) is defined as a first episode of neurologic symptoms that lasts at least 24 hours and is caused by inflammation or demyelination in the central nervous system.
Migraine[14]-[22]:
- Nine new scientific abstracts on Aimovig® (erenumab), reinforcing the extensive long term safety and efficacy profile of the drug across the spectrum of migraine, including chronic, episodic and difficult to treat migraine patients.
- A new set of data shows that a majority of chronic migraine patients on Aimovig converted to episodic migraine. In a separate study, almost two thirds of patients with episodic migraine reported a sustained reduction of migraine days at one year.
Aimovig has been studied extensively in a broad clinical program involving more than 3,000 patients. Since launch in May 2018, Aimovig has been used to treat an estimated 220,000 patients worldwide, making it the most prescribed anti-CGRP therapy worldwide.
