Novartis announces lift of partial clinical trial hold and plans to initiate a new, pivotal Phase 3 study of intrathecal OAV-101 in older patients with SMA

Ad hoc announcement pursuant to Art. 53 LR

  • FDA concluded that OAV-101 intrathecal (IT) clinical program may proceed based on data from nonclinical toxicology study

  • New Phase 3 STEER study will evaluate efficacy, safety, and tolerability of OAV-101 IT in treatment-naïve patients with SMA Type 2 aged between 2 and 18 years old, the first to study gene therapy in this patient population

  • STEER will build upon the OAV-101 IT STRONG study which demonstrated significant increases in HFMSE scores and a clinically meaningful response in patients with SMA Type 2 aged between =2 years and <5 years old

  • OAV-101 IT under investigation as a one-time, single-dose, treatment option for older patients with SMA


Basel, August 3, 2021 - Novartis today announced that the U.S. Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. The decision to lift the hold was based on data from Novartis’ comprehensive nonclinical toxicology study in non-human primates (NHP) that addressed all issues identified, including questions of dorsal root ganglia (DRG) injury following IT administration.

Following this decision and input from the FDA and European Medicines Agency (EMA), Novartis now plans to initiate STEER, a global pivotal Phase 3 registration-enabling study to evaluate the clinical efficacy, safety, and tolerability of OAV-101 IT in treatment naïve patients who are between two and 18 years of age, able to sit, but have never walked. While disease progression is slower in patients with later-onset SMA, there are significant unmet needs.

"We are very pleased that our comprehensive nonclinical data package has addressed all issues identified related to DRG toxicity and the FDA has reached the decision that we may proceed with our OAV-101 IT clinical trial program and initiate the STEER trial," said Shephard Mpofu, M.D., SVP, Chief Medical Officer, Novartis Gene Therapies. "We believe that all patients diagnosed with SMA should be able to benefit from the transformative impact of gene therapy and we remain confident that investigational OAV-101 IT is a viable potential treatment path for older patients who often have ongoing unmet needs, and for whom a one-time treatment could be especially compelling."

STEER will build upon the Phase 1/2 STRONG study which showed that treatment with OAV-101 IT led to significant increases in Hammersmith Functional Motor Scale-Expanded (HFMSE) scores and a clinically meaningful response in older patients between

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