TPTX » Topics » We do not undertake to discuss matters relating to our ongoing clinical trials or our regulatory strategies beyond those which have already been made public or discussed herein.

This excerpt taken from the TPTX 10-K filed Mar 16, 2006.

          We do not undertake to discuss matters relating to our ongoing clinical trials or our regulatory strategies beyond those which have already been made public or discussed herein.

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PART I

Item 1.      Business

TABLE OF CONTENTS

 

 

 

 

A.

Recent Events

 

 

B.

Axonyx Business Strategy and Drug Development Programs

 

 

C.

Alzheimer’s Disease Overview

 

 

D.

Out-Licensed Technology

 

 

E.

Competition

 

 

F.

Government Regulation

 

 

G.

Strategic Alliances

 

 

H.

Marketing and Sales

 

 

I.

Patents, Trademarks, and Copyrights

 

 

J.

Employees

This excerpt taken from the TPTX 10-Q filed Aug 8, 2005.

          We do not undertake to discuss matters relating to our ongoing clinical trials or our regulatory strategies beyond those which have already been made public or discussed herein.

          We are a biopharmaceutical company, specializing in central nervous system (CNS) neurodegenerative diseases, engaged in the business of acquiring the patent rights to clinical stage compounds, compounds with strong proof of concept data and compounds ready for proof of concept validation with convincing scientific rationale. We further develop and add value to these compounds and then seek to out-license or partner them when we believe it business prudent. We have acquired worldwide exclusive patent rights to three main classes of therapeutic compounds designed for the treatment of Alzheimer’s disease (AD), Mild Cognitive Impairment, and related diseases.  We have acquired patent rights to a class of potential therapeutic compounds designed for the treatment of prion related diseases, which are degenerative diseases of the brain that are thought to be caused by an infectious form of a protein called a prion.  Prions, unlike viruses, bacteria and fungi, have no DNA and consist only of protein.  Such diseases include Creutzfeldt Jakob Disease, new variant in humans, Bovine Spongiform Encephalopathy (BSE or Mad Cow Disease) in cows, and Scrapies disease in sheep.  We have licensed these patent rights separately from New York University and from the National Institutes of Health/National Institute on Aging (via a sublicense).  We also have co-inventorship rights to a patent application regarding a therapeutic compound named Posiphen designed for the treatment of Alzheimer’s disease progression.

          Our mission continues to be the leading biopharmaceutical company that develops products and technologies to treat AD and other central nervous system disorders. Our current business strategy is to

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concentrate our financial resources primarily on three compounds in development for Alzheimer’s disease.
These are:

 

 

 

 

Phenserine – A symptomatic and disease progression treatment of mild to moderate AD.

 

 

 

 

Posiphen™ – A disease progression treatment for AD

 

 

 

 

BisNorCymcerine (BNC) – A symptomatic treatment of severe AD.

          Phenserine is an inhibitor of acetylcholinesterase, and is our lead drug candidate for the treatment of AD.  Acetylcholinesterase is an enzyme in the synapse that degrades the neurotransmitter acetylcholine in the brain and other tissues of the body. Acetylcholinesterase inhibitors are drugs designed to selectively inhibit acetylcholinesterase. Acetylcholine is a chemical substance that sends signals between nerve cells, called neurotransmission, and is therefore called a neurotransmitter.  Neurotransmitters are secreted by neurons, or nerve cells, into the space between neurons called the synapse.  Acetylcholine is a primary neurotransmitter in the brain, and is associated with memory and cognition.

          Posiphen is a compound that appears to decrease the formation of the beta amyloid precursor protein (beta-APP) with potential applications in the treatment of AD. Posiphen is the positive isomer of Phenserine. As such, it appears to affect the messenger RNA of beta-APP as well as inhibit beta secretase whereby beta amyloid levels, in preclinical animal models, are reduced.

          BisNorCymcerine is one of our butyrylcholinesterase inhibitors. Butyrylcholinesterase is found in high concentration in the plaques taken from individuals who have died from AD. Butyrylcholinesterase is an enzyme that is normally found widely in the body and butyrylcholine appears to play a relatively increasingly important role in advancing AD. Therefore inhibition of the enzyme may prove valuable in the treatment of severe AD. 

          The Company’s lead drug, Phenserine, is a third generation acetylcholinesterase inhibitor, which has progressed to late stage clinical trials. The results of the 1st Phase III trial were announced on February 7, 2005 and the interim results from the Phase IIb beta amyloid trial were announced on March 11, 2005. The Company announced a second interim analysis of the Phase IIb trial on July 26, 2005.

          Overall, the results from each trial did not show statistically significant improvements over placebo for the protocol’s primary endpoints following 26 weeks of treatment. We have evaluated the results, and following recommendations from our Scientific Advisory Board and Safety Steering Committee, we have modified the planned Phenserine clinical program going forward. The Phenserine results to date demonstrate a consistent positive trend on all primary and secondary domains in the symptomatic management of Alzheimer’s disease. However, there was no statistically significant efficacy in cognition, global function and activities of daily living, that may in part have been caused by the fact that there was minimal decline over the 26 weeks in the placebo group. Our conclusion from analyzing the data is that we need to investigate the reformulation of Phenserine to a sustained or extended release formulation rather than the immediate release formulation used in the recently completed trials. The reformulation should reduce the pharmacokinetic peak experienced with the immediate release formulation and enable more drug to work longer over the dosing interval in the patients. Therefore, we have halted additional patient recruitment to our second and third Phase III clinical trials that were commenced in June 2004 and September 2004 respectively. Those patients already enrolled in the trial will receive 12 weeks of treatment.

          The March 11, 2005 interim statistical analysis of the results from our Phase IIb beta amyloid trial, while not meant to show significance, showed a consistent trend towards beta amyloid reduction with the 15mg doses. While the second interim analysis announced on July 26, 2005 appeared again to confirm that Phenserine may have an impact on the levels of beta amyloid, there is still insufficient evidence due to the variability of the data to draw definitive conclusions.  Further detailed analysis as well as additional laboratory bio assay activities are being undertaken by the Company, its scientific advisors and bio- statisticians to investigate the variability in the data.  Mean while, the enrollment in the phase IIb trial will continue at the 15mg dose level and placebo with a target of 150 patients.   

          Posiphen has been shown to lower beta-amyloid precursor protein (beta-APP) levels in pre-clinical studies. The primary mechanism of action results in a dose dependent reduction of beta amyloid, which may

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result in slowing AD progression. The initial pre-clinical side effect rates potentially allow for higher clinical doses. On August 1, 2005 the Company announced that the US Food and Drug Administration (FDA) has approved its investigational new drug (IND) application allowing phase I clinical testing of Posiphen.  The first Phase I clinical study is expected to begin shortly and will primarily evaluate the safety of Posiphen in healthy volunteers. 

          BisNorCymcerine (BNC) is a highly selective butyrylcholinesterase inhibiter. Butyrylcholinesterase is found in high concentration in the plaques taken from individuals who have died from AD. Butyrylcholinesterase appears to have an increasing role with advancing Alzheimer’s disease and its primary mechanism of action results in a dose dependent reduction of Acetylcholine. The initial pre-clinical side effect rate potentially allows higher clinical doses. A secondary mechanism of action is associated with dose dependent reductions of beta APP and amyloid beta. BNC, the lead compound from our butyrylcholinesterase family, is currently in full pre-IND development and we plan an IND submission in fourth quarter 2005 followed by the initiation of Phase I clinical studies in first quarter 2006.

          In December 2000, the Company incorporated Axonyx Europe BV, a wholly owned subsidiary, in the Netherlands.  Gosse Bruinsma, M.D., currently the President and Chief Executive Officer of Axonyx Inc., is also the President of Axonyx Europe BV. The majority of our clinical development activities and a significant amount of our pre-clinical development activities are carried out in Europe. The Axonyx Europe BV office manages, directs, and controls these activities. Axonyx Europe BV explores and pursues in-licensing and out-licensing opportunities for the Company’s licensed technologies and facilitates communication with the Company’s European shareholders and Serono.

          In June 2005, the Company appointed Paul Feuerman as its General Counsel.  Mr. Feuerman is a founding member of Pharm Advisors LLC, a consulting firm serving pharmaceutical and biopharmaceutical companies. Formerly, he was Executive Vice President and General Counsel of Schein Pharmaceutical Inc., a New York Stock Exchange listed specialty pharma/generics company.

          We have incurred negative cash flows from operations since the inception of the Company in 1997.  Our net losses for the three fiscal years ended 2002, 2003 and 2004 were $6,256,000, $8,106,000 and $28,780,000 respectively, and our net loss for the six months ended June 30, 2005 was $18,612,000.  We have no products available for sale and we do not expect to have any products commercially available for several years, if at all.

          Axonyx Inc. was incorporated in Nevada on July 29, 1997.  Our principal executive offices are located at 500 Seventh Avenue, 10th Floor, New York, New York 10018, and our telephone number is (212) 645-7704.

EXCERPTS ON THIS PAGE:

10-K
Mar 16, 2006
10-Q
Aug 8, 2005
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