Clinical Trials

Novartis Pharmaceuticals has sponsored multiple clinical trials evaluating the therapeutic application of Branaplam (LMI070). These Phase 1 and Phase 2 investigations encompass safety, efficacy, and pharmacokinetic evaluations in infants with Type 1 spinal muscular atrophy, adults with early manifest Huntington's disease, and healthy volunteers. Depending on the target indication, recruitment statuses across these studies include both completed and terminated trials.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT05111249 TERMINATED
Early Manifest Huntington Disease
Novartis Pharmaceuticals
 2021-12-08 PHASE2
NCT02268552 COMPLETED
Spinal Muscular Atrophy
Novartis Pharmaceuticals
 2015-04-02 PHASE1; PHASE2
NCT05330286 TERMINATED
Healthy Volunteers
Novartis Pharmaceuticals
 2022-04-13 PHASE1
NCT05111249 Completed
Early Manifest Huntington Disease
Novartis Pharmaceuticals|Novartis
 2021-12-08 Phase 2

(data from https://clinicaltrials.gov, updated on 2025-05-16)

Check the Branaplam (LMI070) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Branaplam (LMI070) functions as a highly selective small-molecule splicing modulator of survival motor neuron-2 (SMN2) pre-mRNA with an EC50 of 0.02 μM, promoting correct splicing and boosting full-length functional protein expression in target cells. By altering RNA splicing processes to restore essential protein production and alter pathogenic gene expression, it provides therapeutic potential for neurodegenerative conditions evaluated in clinical trials, including spinal muscular atrophy and Huntington's disease.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

Jump to

Note: Technical data last updated: Sep 1, 2026.