Small molecules (Roche – risdiplam)

About small Molecules

Small molecules are chemicals or compounds that are capable of treating or even curing a disease. These potential drugs may be used to increase the amount of SMN protein made by SMN2.

A multi-step process is used to convert small molecules into drugs:

A number of companies and academic laboratories have small molecule programmes that are attempting to identify and develop compounds that target the SMN2 gene.

Ongoing small molecule trials for SMA

Small molecules to treat SMARoche’s risdiplam (RG7916)

Recruitment for three phase 2 clinical trials started in December 2016 in SMA types 1, 2 & 3 through trials called Firefish, Sunfish and Jewelfish. RG7916 is an orally available drug that aims to correct the splicing of SMN2, the “backup gene.”

For more information, please read our dedicated page.

A new update concerning development results of risdiplam in Type 1 SMA (Firefish study) has been provided (July 2021). Roche has a dedicated website for these SMA trials, where more information can be found.

Discontinued small molecule trials for SMA

Small molecules to treat SMANovartis’ Branaplam (LMI070)

This was an open-label, multi-part, first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy. The purpose of this study was to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and efficacy after 13 weeks; and to estimate the Maximum Tolerated Dose (MTD) and optimal dosing regimen of orally administered LMI070 in patients with Type 1 SMA.