Patients were evaluated while receiving either placebo or one of three dose levels of arimoclomol three times daily for 12 weeks and during a 4-week follow-up period without drug.
“This is an important milestone in arimoclomol’s clinical development and moves us a step closer to our goal of commercializing drugs aimed at reducing human suffering,” said Steven Kriegsman, president and CEO of CytRx. “Now that we have successfully completed the clinical aspect of the trial we can begin the process of data analysis.”
The data will now be evaluated and analyzed under strict quality-controlled guidelines. CytRx expects to complete data analysis of the phase IIa trial and announce final results early in the fourth quarter of this year.
The FDA has granted Fast Track designation and Orphan Drug status to arimoclomol for the treatment of ALS. The drug is believed to function by stimulating a normal cellular protein repair pathway through the activation of “molecular chaperones.” Since damaged proteins called aggregates are thought to play a role in many diseases, CytRx believes that activation of molecular chaperones could have therapeutic efficacy for a broad range of diseases.