ANN ARBOR, MI , a specialty pharmaceutical company developing
innovative late-stage drug candidates for the treatment of
neurologic and fibrotic diseases, announced today that it has
received notice from the FDA that its New Drug Application (NDA)
for oral tetrathiomolybdate (COPREXA) for the treatment of
initially presenting neurologic Wilson's disease has not been
accepted by the FDA for further review as submitted.
A conference call has been scheduled for 8:30am ET today. In
order to participate in the conference call, please call toll free:
866-300-7687 (US); international dial-in: 416-641-6142. The call
will be archived and available for replay for at least 30 days by
accessing the company's website at www.pipexinc.com.
Pipex believes that the clinical and nonclinical deficiencies
cited by the FDA were already discussed, resolved and agreed upon
with the FDA during one of the two pre-NDA meetings held with a
first review division of the FDA in August of last year. Pipex
relied upon and believes it honored those commitments in the NDA it
filed. Pipex was notified by the FDA that the NDA was transferred
from the initial review division in which the pre-NDA meetings took
place, to a new review division in late December. According to the
FDA, the FDA had determined that a medical group in the new review
division had expertise in diseases resulting from inborn metabolic
errors and would be a more appropriate group for the review of this
application. Pipex believes that this recent transfer may have
resulted in a loss of continuity of discussion and detailed
understanding and may have contributed to the FDA's decision deny
further review of the application as filed.
The FDA communicated a refusal to file ("RTF") under the sixty
(60) day statutory period of 21 C.F.R. 314.101. It is Pipex's
position that the main issues cited by the FDA are substantive
review items and therefore should be considered upon review of the
application, as opposed to in the context of an RTF. In response to
this RTF, Pipex immediately requested a meeting with the FDA to
discuss the perceived deficiencies in further detail with the
expectation of either reaching agreement to file the NDA as
submitted or reach agreement on what modifications are necessary in
order for the application to be filed. The FDA, in accordance with
regulations, has agreed to schedule this meeting within 30 days.
After this meeting with the FDA, Pipex plans to provide further
details.
The eight RTF issues cited by the FDA include:
-- the adequacy of the analytical methodology to characterize the active
pharmaceutical ingredient of COPREXA;
-- a request to conduct an additional short term reproductive drug safety
study in animals;
-- 4 formatting and presentation items contained in the NDA; and
-- 2 preliminary assessments concerning the adequacy of the clinical
evidence of safety and efficacy of COPREXA considered by Pipex to have been
agreed upon with the FDA during two pre-NDA meetings.
Dr. Kenneth King, the lead regulatory consultant to Pipex
involved in the preparation of the COPREXA NDA commented, "The FDA
is bound by their previous decisions, regardless of who made them,
unless new evidence suggests the previous decisions or
recommendations should be altered. We worked hard with the FDA to
facilitate the transfer of the agreements made in our pre-NDA
meetings. I believe the issues raised by the RTF decision are in
large part the issues Pipex had discussed and reached agreement
with the FDA. We will, of course, work with the FDA to resolve any
misunderstandings and reach agreement on the best way forward to
initiate the review of this application and these comments received
will assist us in re-submitting the application and securing the
requisite approvals."
Dr. Charles L. Bisgaier, Pipex's President, commented, "During
our pre-NDA meetings with the prior interacting division, we
believed we adequately addressed the substance and content of the
planned NDA. Our regulatory submission was based on these
agreements."
"We are obviously disappointed with the FDA's decision not to
accept the COPREXA NDA filing for further review at this time. My
hope is that upon further consideration within the next thirty 30
days the Agency, whose Orphan Products Division generously funded
the 20 years of clinical testing of this important new treatment
option for this desperate group of Wilson's disease patients, will
be satisfactorily convinced that our New Drug Application should be
accepted for filing and approved," said Steve H. Kanzer, CPA, Esq.,
Pipex's Chairman & Chief Executive Officer.
About COPREXA
COPREXA is an oral, small-molecule, anti-copper agent that is
highly specific for the reduction of free copper in serum, the most
toxic form of copper in the body, and is thus suited for the
treatment of central nervous system (CNS) diseases in which
abnormal serum and CNS copper homeostasis are implicated. COPREXA
has completed two clinical trials in initially presenting
neurologic Wilson's disease patients, the results of which have
been previously published(1)(2). Pipex is also developing COPREXA
for fibrotic disorders based upon the rationale that the fibrotic
disease process is dependent upon the availability of free copper
in the body. COPREXA has demonstrated the ability to inhibit
fibrosis in a number of well established animal models through the
sequestration of available copper and inhibition of key fibrotric
cytokines, including secreted protein acid rich in cysteine
(SPARC), NFKB, TGF-B, FGF-2, IL-1, IL-6, IL-8, and connective
tissue growth factor (CTGF). COPREXA has been designated as an
"Orphan Drug" by the FDA for the treatment of initially presenting
neurologic Wilson's Disease.
COPREXA has completed a 20-patient, one year, open label, phase
I/II clinical trial for the treatment of refractory idiopathic
pulmonary fibrosis (IPF), a fatal respiratory disease. The results
of this trial were presented in May of this year at the annual
meeting of the American Thoracic Society (ATS).
About Wilson's Disease
Wilson's disease is an autosomal recessive genetic disease
attributable to mutations of the ATP7B gene. Worldwide, it is
estimated that there are between 10 million and 30 million carriers
of the heterozygous mutated gene. These mutations lead to an
inability to properly clear excess free copper from the body via
the liver into the bile and stool. As a result, copper accumulates
in the liver and elevated levels of toxic free copper enter the
systemic circulation, cross the blood brain barrier, and enter the
cerebral spinal fluid (CSF) and brain. These increased levels of
free copper cause significant neurologic damage, resulting in
tremors, impaired speech, impaired coordination, and
Parkinson's-like dystonia.
Psychiatric symptoms of neurologically-presenting Wilson's
patients will generally precede neurologic symptoms by months or
years and may include loss of emotional control, temper tantrums,
emotional outbursts, bouts of crying, severe depression, suicidal
ideation, loss of inhibitions, delusions, hallucinations and loss
of ability to focus on tasks. Neurologic symptoms later develop as
a result of neurodegeneration in the basal ganglia of the brain and
include impaired speech, tremor, dystonia, incoordination and
dysphasia. Crippling movement disorders may ultimately occur.
Without proper treatment, Wilson's disease is usually fatal by the
age of 30. However, if treatment is begun early enough, symptomatic
recovery is usually complete and a life of normal length and
quality can be expected. For more information about Wilson's
disease, please visit or contact the Wilson's Disease Association
at www.wilsonsdisease.org.
About Pipex Pharmaceuticals, Inc.
Pipex Pharmaceuticals, Inc. ("Pipex") is a specialty
pharmaceutical company that is developing proprietary, late-stage
drug candidates for the treatment of neurologic and fibrotic
diseases. Pipex's strategy is to exclusively in-license
proprietary, clinical-stage drug candidates and complete the
further clinical testing, manufacturing and regulatory requirements
sufficient to seek marketing authorizations via the filing of New
Drug Applications (NDAs) with the FDA in the US and Marketing
Application Authorizations (MAAs) with the European Medicines
Evaluation Agency (EMEA). For further information please visit
www.pipexinc.com.
(1) Brewer, G.J., et al., Treatment of Wilson disease with ammonium
tetrathiomolybdate: III. Initial therapy in a total of 55 neurologically
affected patients and follow-up with zinc therapy. Arch Neurol. 2003 Mar;
60(3):379-85.
(2) Brewer, G.J., Askari, F., Lorincz, M.T., Carlson, M., Schilsky, M.,
Kluin, K.J., Hedera, P., Moretti, P., Fink, J.K., Tankanow, R., et al.
2006. Treatment of Wilson disease with ammonium tetrathiomolybdate: IV.
Comparison of tetrathiomolybdate and trientine in a double-blind study of
treatment of the neurologic presentation of Wilson disease. Arch Neurol
63:521-527.
This press release contains forward-looking statements, within
the meaning of Section 21E of the Securities Exchange Act of 1934,
that reflect Pipex Pharmaceuticals, Inc. and subsidiaries ("we" or
"our") current expectations about its future results, performance,
prospects and opportunities, including statements regarding the
potential use of COPREXA as well as the resolution of any of the
deficiencies raised by the FDA noted in this announcement,
including the potential delay in potential approval as well as the
cost and expense of conducting any additional testing, development
and/or clinical trials required by the FDA, the prospects for any
regulatory filings in the treatment of neurologic Wilson's disease,
including and/or that the FDA will agree with our analysis of data
supporting the safety, clinical efficacy, manufacturing, stability
and other regulatory requirements necessary for COPREXA to be
approved for use in neurologically presenting Wilson's disease or
that even if approved for initial indication, that we will be able
to conduct and complete necessary initial and registration clinical
trials required to support and receive FDA approval for a
Supplemental New Drug Application to market COPREXA for the
treatment of other disease indications, such as, idiopathic
pulmonary fibrosis, Alzheimer's disease and Huntington's disease,
for example. Where possible, the Company has tried to identify
these forward-looking statements by using words such as
"anticipates," "believes," "intends," or similar expressions. These
statements are subject to a number of risks, uncertainties and
other factors that could cause actual events or results in future
periods to differ materially from what is expressed in, or implied
by, these statements, including risks set forth in our filings with
the Securities and Exchange Commission. We cannot assure you that
we will be able to successfully develop or commercialize products
based on our technologies, including COPREXA(TM), TRIMESTA(TM),
zincmonocysteine, SOLOVAX(TM), EFFIRMA(TM) or Anti-CD4 802-2,
particularly in light of the significant uncertainty inherent in
developing, manufacturing and conducting preclinical and clinical
trials of new pharmaceuticals, and obtaining regulatory approvals,
that our technologies will prove to be safe and effective, that our
cash expenditures will not exceed projected levels, that we will be
able to obtain future financing or funds when needed, that product
development and commercialization efforts will not be reduced or
discontinued due to difficulties or delays in clinical trials or
due to lack of progress or positive results from research and
development efforts, that we will be able to successfully obtain
any further grants and awards, maintain our existing grants which
are subject to performance, that we will be able to patent,
register or protect our technology from challenge and products from
competition or maintain or expand our license agreements with our
current licensors, or that our business strategy will be
successful. All forward-looking statements made in this press
release are made as of the date hereof, and the Company assumes no
obligation to update the forward-looking statements included in
this news release whether as a result of new information, future
events, or otherwise, other than as required by law.
For Further Information Contact: Steve H. Kanzer, CPA, Esq.
Chairman and Chief Executive Officer (734) 332-7800 Thomas
Redington (Investor Relations) Redington, Inc. (203) 222-7399
Meet Kevin Pricing Power... (AMEX:PP)
Historical Stock Chart
From Jan 2025 to Feb 2025
Meet Kevin Pricing Power... (AMEX:PP)
Historical Stock Chart
From Feb 2024 to Feb 2025
