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
RILUTEK® (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 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.
"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."
About AEN-100
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.
RILUTEK® (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.
