Quarterly eNewsletter – June 2022 Edition

Welcome to our Q2 newsletter. As you’ll see below, we have lots of terrific news to share.

I'd like to start with news from our Biologics and Drug Delivery business. As we cautiously move into a post-pandemic reality, many of our pharmaceutical partners have begun actively enrolling patients into clinical trials in several geographies and achieved some exciting milestones:

• In May, our partner Lysogene shared a clinical update for their AAVance phase 2/3 clinical trial for gene therapy LYS-SAF302 for the treatment of MPS IIIA. The promising efficacy data showed a persistent increase or stabilization in 3 main developmental domains (cognition, motor and language) in all patients enrolled under the age of 30 months.
• Also last month,  PTC Therapeutics received a positive opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommending its gene therapy treatment Upstaza™ (eladocagene exuparvovec) for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. ClearPoint’s proprietary CE Marked SmartFlow® Neuro Ventricular Cannula is included in the submission for minimally invasive infusion of the Upstaza gene therapy. This significant milestone marks the first ever positive regulatory recommendation anywhere in the world for direct injection of a gene therapy to the brain.
• Last week uniQure announced encouraging safety and biomarker data from 10 patients enrolled in their Phase I/II clinical trial of AMT-130 for the treatment of Huntington's Disease.
• And finally, yesterday, Neurona Therapeutics announced the dosing of their initial patient in their Phase 1/2 clinical trial to treat drug-resistant mesial temporal lobe epilepsy.

Our team has been executing relentlessly to support both our neurosurgery and pharmaceutical customers across the globe, We recently announced receipt of Medical Device Single Audit Program certification (MDSAP). MDSAP allows a single regulatory audit of our quality management system to satisfy the regulatory requirements of multiple countries: Australia, Brazil, Canada, Japan and the United States. MDSAP, in addition to the 10 additional offers of employment we extended this quarter, will continue to power our global expansion, supporting our innovation pipeline, and commercial approvals beyond the US and EU.

Thank you for your continued support of our vision and our team. If you have any questions for me, please do not hesitate to reach out.