ANN ARBOR — Adeona Pharmaceuticals (NYSE Amex: AEN) Wednesday said that its clinical collaborator for amyotrophic lateral sclerosis (ALS), PNA Center for Neurological Research, reported results from its pilot Phase I/II open label, three month safety study of oral high dose zinc therapy in ALS, also known as Lou Gehrig’s disease.
The clinical study met its primary outcome as no safety issues related to zinc therapy were observed. In addition, an average decrease in the monthly rate of disease progression was observed in the ALS patients on zinc therapy, compared to published historical controls, as well as compared to the average monthly rate of disease progression of the subjects prior to enrollment in the study.
PNA’s clinical study data was presented at the 22nd International Symposium on ALS/Motor Neurone Disease in Sydney, Australia Wednesday. from PNA.
Ten patients diagnosed with sporadic ALS and on stable doses of Rilutek were enrolled in the open label, three month study of oral high dose zinc therapy. The study was conducted under an Investigational New Drug application and was registered at http://clinicaltrials.gov/ct2/show/NCT01259050. The rate of disease progression was measured by the ALS Functional Rating Scale-Revised, a widely used, validated rating scale that assesses the progression of disability in patients with ALS, revised to also incorporate assessments of respiratory function. At baseline, the average ALSFRS-R score of these patients was 33 and the average time from symptom onset was one year.
Patients were administered pills containing 90mg of elemental zinc per day, as well as 2 mg of copper every other day to prevent potential copper depletion. Eight out of the 10 patients enrolled completed three months of zinc therapy. Two patients dropped out within the first month for reasons unrelated to the zinc therapy. All patients reported taste disturbance (metallic taste) and two of eight patients reported nausea (both of whom were able to complete the study after reducing their dose to 60mg of zinc per day).
On average, the eight patients who completed the study lost 0.37 ALSFRS-R points per month during the three months of therapy. This represents a lower rate of monthly disease progression compared to the average 0.89 ALSFRS-R monthly rate of disease progression in ALS based on historical controls. Prior to enrolling in the study, seven of the eight patients for whom previous ALSFRS-R scores were available lost an average of 0.61 ALSFRS-R points per month.
Based on these findings, the neurologists at PNA hypothesize that high doses of zinc may slow disease progress in ALS and that a larger controlled clinical trial of zinc therapy in ALS patients is warranted. Preparations are currently under way to evaluate the safety and efficacy of Adeona’s proprietary drug candidate, AEN-100, a gastroretentive, sustained-release, zinc tablet, in an adaptively designed, multi-center, double-blind, placebo-controlled Phase II/III clinical trial in ALS patients to be conducted under an IND.
It is anticipated that the trial will enroll approximately 65 ALS patients, who will continue on Rilutek (riluzole) as the standard of care treatment, and that the patients will be randomized into two treatment groups and one matching placebo group. They will receive clinical trial medications for at least six months with periodic monitoring.
A small Phase I pharmacokinetic clinical trial of AEN-100 is planned for completion prior to initiating the multi-center clinical trial. It is anticipated that Adeona will provide the study medications and fund the clinical trials, which will be led by the neurology team at PNA.
Said Todd D. Levine, M.D., president of PNA, assistant clinical professor at the University of Arizona, co-director of the Banner Samaritan ALS Center in Phoenix, Arizona and lead principal investigator of Adeona’s planned clinical trial: “We look forward to initiating this larger clinical trial in ALS patients and to providing Adeona’s proprietary zinc-based therapy that has already demonstrated clinical evidence of being very well tolerated by patients and of providing superior bioavailability.”
Added James S. Kuo, M.D., Adeona CEO: “Given the clinical results PNA presented today at an international symposium suggesting a safe and therapeutic role for zinc in ALS, we believe it supports our planned Phase II/III clinical trial of AEN-100 in ALS patients.”