arimoclomol maleate (BRX-220) / LadRx - LARVOL DELTA

"CytRx Highlights Orphazyme’s Acceleration of Pre-Launch Activities for Arimoclomol https://t.co/nrBMvdGVu5" (@NewsFromBW)

Cellular Chaperones As Therapeutic Targets in ALS to Restore Protein Homeostasis and Improve Cellular Function. (PubMed, Front Mol Neurosci) - "Initial clinical investigations of Arimoclomol have shown promising results. Therefore, it is possible that the long series of unsuccessful clinical trials for ALS may soon be reversed, as optimal targeting of proteostasis in ALS may now be possible, and may deliver clinical benefit to patients."

Journal • Review • Biosimilar • Complement-mediated Rare Disorders • Immunology • Myositis • Pain

Arimoclomol in Amyotropic Lateral Sclerosis (clinicaltrials.gov) - P3; N=231; Active, not recruiting; Sponsor: Orphazyme; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed

"Yes!! And what about levosimendan?! And ASO?! And Arimoclomol?!" (@jimiLdorado)

Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type C (clinicaltrials.gov) - P2/3; N=50; Active, not recruiting; Sponsor: Orphazyme; Trial primary completion date: Oct 2020 ➔ Jun 2018

Clinical • Trial primary completion date

Study of Arimoclomol in Inclusion Body Myositis (IBM) (clinicaltrials.gov) - P2; N=150; Active, not recruiting; Sponsor: Orphazyme; Recruiting ➔ Active, not recruiting

Enrollment closed

Arimoclomol in Amyotropic Lateral Sclerosis - Open Label Extension Trial (clinicaltrials.gov) - P3; N=121; Not yet recruiting; Sponsor: Orphazyme

Clinical • New P3 trial