IB1001-201 (NPC): NOV. 2019 RECRUITMENT UPDATE

IntraBio Ltd. is pleased to share that the IB1001-201 clinical trial has screened two-thirds ( 2 / 3 ) the target number of patients. The study, which investigates N-acetyl-L-leucine (IB1001) for the treatment of Niemann-Pick disease Type C (NPC), will enroll a total of approximately 30 patients across all international sites. Since recruitment commenced in September 2019, twenty (20) patients have been screened across nine (9) international clinical trial sites. Recruitment is expected to continue rapidly and be completed by December 2019. To ensure all patients have the opportunity to participate, interested patients are encouraged to contact their nearest study center as soon as possible to schedule a screening visit before enrollment is complete. Information on each site, including contact details, can be found below. GERMANY Ludwig Maximilian University of Munich Patients aged 6 Years + Principal Investigator: Susanne Schneider, MD Contact: Anita Hennig, MD Email: [email protected] Tel: +49(0)89 4400 76777 University of Giessen Patients aged 6 Years + Principal Investigator: Andreas Hahn, MD Contact: Kyriakos Martakis, MD Email: [email protected] Tel: +49 (0)641 985 43543 SLOVAKIA Comenius University in Bratislava Patients aged 6 -17 Years Principal Investigator: Miriam Koníková, MUDr, PhD Sub-Investigator: Tomáš Foltán, MUDr Contact: Katarína Cichovská Email: [email protected] Tel: +421 2 59371 274 SPAIN Bellvitge University Hospital Patients aged 6 Years + Principal Investigator: Jordi Gascon Bayarri, MD Email: [email protected] Tel: +34 932 607500 ext 2958 UNITED KINGDOM Great Ormond Street Hospital Patients aged 6 – 17 Years Principal Investigator: Paul Gissen, MD Contact: Olivia Rosie-Wilkinson, Senior Research Coordinator Email: [email protected] Tel: +44 (0)2074 059200 ext. 47267 Royal Manchester Children’s Hospital Trial Site for Patients Aged 6 – 17 Years Principal Investigator: Simon Jones, MD Contact: Laura Crowther, Clinical Trials Manager Email: [email protected] Tel: +44 (0)161 701 9137 Royal Free London NHS Foundation Trust Trial Site for Patients Aged 13 Years + Principal Investigator: Uma Ramaswami, MD Contact: Masoud Kazemi, Senior Research Lead Email: [email protected] Tel: +44 (0) 207 830 2814 Salford Royal NHS Foundation Trust Trial Site for Patients Aged 18 Years + Principal Investigator: Reena Sharma, MD Contact: Marie Meehan, Senior Clinical Research Practitioner/ Team Manager Email: [email protected] Tel: +44 (0)161 206 4192 UNITED STATES The Mayo Clinic (Rochester, MN) Patients 18 Years + Principal Investigator: Anhar Hassan, MD Contact: Sandra Looney, Clinical Research Coordinator Email: [email protected] Tel : +1 (507) 538-4107 About IB1001-201 IB1001-201 investigates N-acetyl-L-leucine (IB1001) for the treatment of Niemann-Pick disease Type C (NPC). This international trial is being run in clinical centers in the United States, the United Kingdom, Germany, Spain, and Slovakia. In Europe, patients aged 6 and older with a confirmed diagnosis of NPC may be eligible for recruitment. The trial will consist of three study phases: a baseline period, a 6-week treatment period, and a 6-week post-treatment washout period followed by a planned one-year extension phase. Patients (and a caregiver) will be reimbursed for reasonable out-of-pocket expenses incurred for participating in the trial, such as travel and parking. Additional information on Clinical Trial Study IB1001-201 can be found via ClinicalTrials.Gov(NCT03759639) .

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