Positive Clinical Study Results Reported by Adeona’s Oral Zinc for ALS Collaborator
ANN ARBOR, Mich., Nov. 30, 2011 /PRNewswire/ — Adeona Pharmaceuticals, Inc. (NYSE Amex: AEN), a developer of innovative disease-modifying medicines for serious illnesses, announced today that the Company’s clinical collaborator for amyotrophic lateral sclerosis (ALS), PNA Center for Neurological Research (PNA), reported top-line 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 on Wednesday, November 30, 2011 at 6:00 p.m. (Wednesday, November 30, 2011 at 2:00 a.m. Eastern Standard Time), by David S. Saperstein, M.D., and Nicole C. Hank, M.H.S.M., from PNA.
Ten patients diagnosed with sporadic ALS and on stable doses of RILUTEKÃ‚® (riluzole) 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 (IND) and was registered at http://clinicaltrials.gov/ct2/show/NCT01259050. The rate of disease progression was measured by the ALS Functional Rating Scale-Revised (ALSFRS-R), 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 ten 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.[i] 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 underway 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 RILUTEKÃ‚® (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.
“We are pleased to report that the use of zinc is safe in ALS patients, and we are also encouraged to observe that this small group of ALS patients demonstrated a reduced rate of change in their ALSFRS-R scores while taking zinc, suggesting a slower rate of disease progression,” 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.”
“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,” said James S. Kuo, M.D., M.B.A., Chief Executive Officer of Adeona. “We are pleased to be working with the dedicated neurologists at PNA to evaluate the potentially revolutionary therapeutic benefit of AEN-100 for this devastating and fatal progressive neurological disease.”
AEN-100 is Adeona’s patent-pending gastroretentive, sustained-release oral zinc drug candidate intended for indications in which high dose zinc therapy may be appropriate. Based upon prior studies conducted by Adeona, the Company believes that AEN-100 provides far superior gastrointestinal tolerability and once-daily dosing convenience compared to existing zinc therapy products. Gastrointestinal tolerability (namely, nausea and vomiting) represents a major dose limiting factor of oral high dose zinc therapy. Adeona also intends to file for orphan drug protection with the Food & Drug Administration (FDA) in the U.S. and European Medicines Agency (EMEA) in the E.U, which may provide for marketing exclusivity for a period of seven and ten years, respectively, following approval. In ALS, there is a demonstrated zinc binding defect of the ALS-implicated protein known as copper/zinc superoxide dismutase (SOD-1) as well as a demonstrated sequestration of zinc in the Lewy body-like hyaline inclusions that are characteristic of ALS.[ii]
About Amyotrophic Lateral Sclerosis (ALS)
ALS is a devastating progressive neurodegenerative disease that affects the motor nerve cells in the brain and the spinal cord of predominantly older people of both sexes. It is estimated that as many as 30,000 Americans may have the disease at any given time. The progressive degeneration of the motor neurons in ALS eventually leads to the death of the patient. Motor neurons reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. When motor neurons die, the ability of the brain to initiate and control muscle movement is lost. With voluntary muscle action progressively affected, patients in the later stages of the disease may become totally paralyzed.
While non-invasive ventilation and gastrostomy tubes prolong life by 6-12 months, the average lifespan from time of symptom onset is 2-5 years. Currently, RILUTEK is the only FDA-approved drug for ALS. RILUTEK is an NMDA receptor antagonist and has been shown to prolong life in patients with ALS by 3 months. Presently, there is no cure for ALS, nor is there a known cause. For more information on ALS, please visit the ALS Association website at www.alsa.org.
About PNA Center for Neurological Research
PNA Center for Neurological Research (PNA) is an independent, not-for-profit organization based in Phoenix, Arizona. PNA was established by five of the neurologists from Phoenix Neurological Associates, Ltd., who are dedicated to discovering new treatments for patients with neurological diseases. PNA’s goal is to be a hub where philanthropists, advocates, organizations, family and friends of patients with a neurological illness could make donations that can support investigator-initiated clinical trials. PNA hopes to optimize proper treatments in order to improve outcomes for patients with neurological diseases. For more information about PNA, please visit its website at www.pnaresearch.org. For more information about Phoenix Neurological Associates, Ltd., please visit its website at www.phoenixneurology.com.
About Adeona Pharmaceuticals, Inc.
Adeona is a pharmaceutical company focused on the development of innovative disease-modifying medicines for serious illnesses. Adeona is developing, or has partnered the development of, drug product candidates to treat pulmonary arterial hypertension, relapses in multiple sclerosis, cognitive dysfunction in multiple sclerosis, fibromyalgia, amyotrophic lateral sclerosis (ALS) and Alzheimer’s disease. For more information, please visit Adeona’s website at www.adeonapharma.com.
RILUTEKÃ‚® (riluzole) is a registered trademark of sanofi-aventis U.S. LLC.
This release includes forward-looking statements on Adeona’s current expectations and projections about future events. In some cases forward-looking statements can be identified by terminology such as “may,” “could,” “potential,” “positions,” “continue,” “expects,” “anticipates,” “intends,” “plans,” “believe,” “estimates,” and similar expressions. These statements are based upon current beliefs, expectations and assumptions and are subject to a number of risks and uncertainties, many of which are difficult to predict and include statements regarding the effects of zinc with regard to ALS, the intent to file for orphan drug protection and the anticipated trial enrollment for the planned Phase ll/III clinical trial. The forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those set forth or implied by any forward-looking statements. Important factors that could cause actual results to differ materially from those reflected in Adeona’s forward-looking statements include, among others, our failure to obtain regulatory approval or market approval of the use of AEN-100 as an orphan drug or in the treatment of ALS, failure to fully enroll patients in the Phase II/III clinical trial, failure to obtain approval to conduct the trial under the IND, failure of the clinical trial evaluating AEN-100 to have favorable result such as a failure to show benefits of zinc therapy in ALS patients, a failure of the treatment group to meet the planned primary and secondary endpoints, acceptable and superior levels of tolerability and efficacy of AEN-100 and as compared to existing over-the-counter zinc products such as that used in the present study, and other factors described in Adeona’s report on Form 10-K for the year ended December 31, 2010 and any other filings with the SEC. The information in this release is provided only as of the date of this release, and Adeona undertakes no obligation to update any forward-looking statements contained in this release on account of new information, future events, or otherwise, except as required by law.
[i]Miller RG, Moore DH, et. al., Phase II screening trial of lithium carbonate in amyotrophic lateral sclerosis: examining a more efficient trial design., Neurology. 2011 Sep 6;77(10):973-9. Epub 2011 Aug 3., Appendix e-1.
[ii]Chattopadhyay M, Valentine JS., Aggregation of copper-zinc superoxide dismutase in familial and sporadic ALS, Antioxid Redox Signal. 2009 Jul;11(7):1603-14.
SOURCE Adeona Pharmaceuticals, Inc.