Posted on July 21st, 2015
by Arnold Kling
As the President and Director of Protalex, Inc., I would like to introduce our clinical-stage biopharmaceutical company that is focused on developing a novel class of drugs for treating autoimmune and inflammatory diseases. Our lead product is PRTX-100, which is derived from a bacterial protein known to modify aspects of the human immune system, potentially making it useful for treating autoimmune diseases, such as rheumatoid arthritis (RA) and immune thrombocytopenia (ITP), both effectively and safely.
The current autoimmune market represents a significant opportunity with global sales over $23 billion. In fact, the leading RA biological agents alone, including the TNF inhibitors Humira, Enbrel, and Remicade, account for over $18 billion in sales. Our initial clinical indication for PRTX-100 is RA, which is the largest portion of the auto-immune market. We will also shortly begin enrolling a multi-center clinical study of PRTX-100 in the treatment of patients with ITP, a rare bleeding disorder in which the immune system destroys platelets, a type of blood cell that are necessary for blood-clotting. Our decision to initiate trials in ITP follows a series of provocative findings in sophisticated animal models of the disease; these studies will be presented at scientific symposia in the coming months.
Although there are numerous treatments for RA, we believe current options do not balance the patients’ needs for safety and efficacy. With about 1.5 million people living with RA in the U.S. alone, patients have to choose between less effective but generally safe drugs, against the leading biologics with significantly increased efficacy, but also significantly increased risks of severe adverse events. It should be noted however, even the biologics are not cures or even long term options as patients often cycle through treatments as the benefits decline over time.
We believe that PRTX-100 may have the ability to deliver biologic-like efficacy without the significant drug-related safety issues. PRTX-100 is a highly purified formulation of Staphylococcal protein A (SpA). In human blood, SpA forms small complexes with certain human antibodies. These complexes can induce ‘alternatively activated’ or ‘regulatory’ macrophages, which have an anti-inflammatory effect, thereby addressing one of the underlying causes of perhaps all autoimmune diseases.
In two Phase Ib trials involving patients suffering from active RA, we saw promising effects on measures of disease activity, achieving an 83.3% ACR20 response at some points. Reassuringly, these effects were accomplished without any drug related serious adverse events to the patients. The most common adverse events were constitutional and included some temporary worsening of the arthritic condition. While still early in the clinical process, these results are encouraging and highlight what we believe may be compelling attributes for an exploratory RA treatment: biologic-like efficacy without biologic-like toxicity. In fact, PRTX-100 has not had a single drug-related severe adverse event so far.
We are very excited for the second half of 2015, as we expect it to be a period of significant progress as we work diligently to develop PRTX-100 efficiently and effectively along several fronts. Earlier this year we initiated enrollment for the PRTX-100-105 Continuation Study which is open to former patients in the 104 Study; eight patients have enrolled in this study to date. In the coming months we will submit the final results of the U.S. PRTX-100-104 Study to the FDA. Finally as noted earlier, we will be initiating a Phase I/II trial of PRTX-100 in ITP, an orphan indication.in the third quarter of 2015. We believe that the next 12 months will be a period of both confirmation of the safety and efficacy of PRTX-100 as well as one of expansion of its potential indications and uses.
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