BBRM2 (Intrathecal Azythromycin)

BBRM2 (Intrathecal Azythromycin) for the treatment of Spinal Muscular Atrophy

Dr. Gliko-Kabir

5th December 2015

BioBlast Pharma Ltd. brought their proposal BBRM2 (intrathecal Azithromycin) for the treatment of spinal muscular atrophy to TACT. Based on their preclinical data they proposed to start a phase 1b open label study to assess the safety, tolerability and pharmacokinetics of single intrathecal administration of BBrm02 in paediatric patients with SMA type2. Azithromycin is an approved antibiotic and potential read-through agent for the stop codon located at exon 8 of the SMNĪ”7 protein. The compound has a well established risk/benefit profile and therefore is an interesting candidate for this indication. It was pointed out by TACT that the compound is not currently approved for continuous administration, which is likely required in the treatment of SMA, which has potential safety and also regulatory implications.

TACT welcomed the proposal but advised that more pre-clinical work would be beneficial before moving into clinic to ensure successful clinical trials. Advice was given on suitable animal models and relevant pre-clinical dosing experiments and readouts that should inform the design of the clinical study.

12 Apr 2017