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Page last updated: 7th August 2023



30th June: Scholar Rock announced the latest data from their TOPAZ trial.

27th October: Scholar Rock presented the latest data from their Phase 2 TOPAZ trial of apitegromab at the 3rd International Scientific Congress on SMA in Barcelona, Spain (22nd October).

20th September: Scholar Rock announced the opening of the initial European clinical trial sites for the Phase 3 SAPPHIRE study in Spain, with additional sites to be announced.

23rd June: Phase 2 trial (TOPAZ) extension data, presented at the recent Cure SMA Research & Clinical Care Meeting.


2nd December: Scholar Rock announce design of Phase 3 clinical trial SAPPHIRE

7th July: Scholar Rock received Fast Track Designation in the US from the FDA for Apitegromab for the treatment of SMA.

8th April: Scholar Rock announces results from TOPAZ Phase 2 trial in SMA Types 2 and 3.


28th October: Scholar Rock announces encouraging interim data from the TOPAZ Phase 2 trial

13th May: Scholar Rock reports delays in TOPAZ interim trial results due to Covid-19


25th November: Scholar Rock presents the preliminary pharmacokinetic and pharmacodynamic analysis of the TOPAZ Phase 2 trial

18th March: Positive interim results from Phase 1 TOPAZ trial announced by Scholar Rock


4th December: European Medicines Agency (EMA) gives SRK-015 Orphan Drug Designation for SMA status.

Scholar Rock also recently published some pre-clinical data on the effects of an early-generation variant of SRK-015, called SRK-015P, in SMA mouse models which has a very similar mode of action and is likely to have a comparable impact on muscles as SRK-015. The publication in Human Molecular Genetics, showed that co-administration of SRK-015 with a drug that increases SMN levels called SMN-C1 (click here for more information) provided greater benefit than SMN-C1 treatment on its own. The combination treatment resulted in increased size and strength of muscles, as well as improvements in bone deficiencies that have previously been reported in SMA mice.

Scholar Rock pre-clinical SRK-015 publication >

8th June: Dosing of the first cohort completed in TOPAZ Phase I trial to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of intravenous injections of SRK-015 in healthy adult volunteers.

3rd May: The US Food and Drug Administration (FDA) granted Orphan Drug Designation to SRK-015 for the treatment of SMA.