Mastocytosis Treatment: Masitinib Enters Phase 3 of Clinical Study

Thank you to GlobeNewswire for allowing the sharing of the press release below!

 

AB Science: Final Results of Phase 3 Mastocytosis Study Expected by End of 2014

Final results of phase 3 mastocytosis study expected by end of 2014.

Indolent Systemic Mastocytosis is an orphan disease with no approved treatment so far.

Up to 20,000 treatable adult patients annually throughout countries paying for medication.

Long term follow up from phase 2 studies shows sustainability of response with masitinib.

PARIS, Nov. 12, 2013 (GLOBE NEWSWIRE) — AB Science SA (NYSE Euronext – FR0010557264 – AB), a pharmaceutical company specialized in research, development and marketing of protein kinase inhibitors (PKIs), following the press release from November 11^th that announced the phase 3 study’s success in the futility analysis, further specifies important points concerning availability of phase 3 data, number of patients treatable and sustainability of response generated by masitinib observed in phase 2.

The read-out of the phase 3 study in mastosytosis is expected by end of 2014. Phase 3 study was accelerated and expanded recently outside of Europe and the USA, in Russia, India and Latin America.

Indolent Systemic Mastocytosis is an orphan disease with no approved drug currently registered. Masitinib received orphan drug status designation in mastocytosis, both from EMA and FDA.

Mastocytosis is an orphan disease characterized by an abnormal proliferation of mast cells either in bone marrow only or in several tissues. Mastocytosis comes in two main forms: indolent and aggressive. Indolent mastocytosis can be either cutaneous or systemic. The prevalence of Indolent Systemic Mastocytosis (ISM) is estimated at between 1/40,000 and 1/20,000^[1] of the general population. The symptoms and handicaps are severe in about one third of the patients, hence an estimated target population for masitinib ranging from 1/120,000 to 1/60,000 of the general population.

Since the prevalence of Indolent Systemic Mastocytosis is reputed to be comparable across countries, the target population for masitinib could reach 20,000 adult patients in the world annually.

The fact that the phase 3 study in mastocytosis passed futility analysis is in line with phase 2 results.

Two phase 2 studies enrolled 46 patients suffering from systemic mastocytosis. Masitinib decreased the flush frequency by 54%, decreased the pruritus score by 45%, improved the depression status by 40% and decreased the fatigue score by 52% from baseline. The two studies generated results consistent with each other despite the fact that the first one enrolled patients without the c-kit 816 mutation and the second one with this mutation, suggesting that masitinib acts by inhibiting not only c-Kit but also Lyn, in blocking the release of the mediators by the mast cell.

In addition, long term follow up data of the two phase 2 studies showed sustainability of responses generated by masitinib. Two-thirds of the patients decided to enroll in the extension phase of the study ; 61% patients were treated for more than 1 year and 25% were still under masitinib after 5 years.

About masitinib

Masitinib is a new orally administered tyrosine kinase inhibitor that targets mast cells, important cells for immunity, as well as a limited number of kinases that play key roles in various cancers. Owing to its novel mechanism of action, masitinib can be developed in a large number of conditions in oncology, in inflammatory diseases, and in certain diseases of the central nervous system. Through its activity of inhibiting certain kinases that are essential in some oncogenic processes, masitinib may have an effect on tumor regression, alone or in combination with chemotherapy. Through its activity on the mast cell and certain kinases essential to the activation of the inflammatory cells and fibrosing tissue remodeling, masitinib can have an effect on the symptoms associated with some inflammatory and central nervous system diseases.

About AB Science

Founded in 2001, AB Science is a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), a new class of targeted molecules whose action is to modify signaling pathways within cells. Through these PKIs, the Company targets diseases with high unmet medical needs (cancer, inflammatory diseases, and central nervous system diseases), in both human and veterinary medicines.

AB Science has developed a proprietary portfolio of molecules and the Company’s lead compound, masitinib, has already been registered for veterinary medicine in Europe and in the USA, and is pursuing ten on-going phase 3 studies in human medicine in GIST, metastatic melanoma expressing JM mutation of c-Kit, multiple myeloma, mastocytosis, severe persistent asthma, rheumatoid arthritis, Alzheimer’s disease, progressive forms of multiple sclerosis, and in Amyotrophic Lateral Sclerosis. The company is headquartered in Paris, France, and listed on Euronext Paris (ticker: AB).

Further information is available on AB Science website: www.ab-science.com.

This document contains prospective information. No guarantee can be given as for the realization of these forecasts, which are subject to those risks described in documents deposited by the Company to the Authority of the financial markets, including trends of the economic conjuncture, the financial markets and the markets on which AB Science is present.

[1] http://www.orpha.net  (Indolent systemic mastocytosis)

AB Science EN: http://hugin.info/155655/R/1742623/585910.pdf

 

Reposted with permission. Original article can be found:

http://www.globenewswire.com/news-release/2013/11/12/589193/10057632/en/AB-Science-Final-Results-of-Phase-3-Mastocytosis-Study-Expected-by-End-of-2014.html?parent=588800

Clinical Study of New Drug for Mastocytosis Treatment

The following is a news release that was recently published regarding a new drug that is currently in clinical study for Mastocytosis treatment. Thank you to GlobeNewswire for graciously allowing us to repost the release here!

AB Science: Masitinib successfully passes futility analysis in pivotal clinical study in Mastocytosis

PARIS, Nov. 11, 2013 (GLOBE NEWSWIRE) — AB Science SA (NYSE Euronext – FR0010557264 – AB), a pharmaceutical company specialized in research, development and marketing of protein kinase inhibitors (PKIs), announces that the independent Data and Safety Monitoring Board (DSMB) created as part of the company pivotal clinical study evaluating masitinib in the treatment of mastocytosis has recommended continuation of the study, based upon completion of the futility analysis included in the study protocol.

The objective in this phase 3 study is to compare the safety and efficacy of masitinib to placebo in adult patients with Indolent Systemic Mastocytosis with severe handicap at baseline.

The futility analysis was included in the clinical study design to ensure that the study could be stopped early if it appeared that the study would be eventually unable to demonstrate the efficacy of masitinib in the target population of patients with Indolent Systemic Mastocytosis with severe handicap at baseline.

The analysis was performed with two-third of the target population to recruit. The futility analysis was performed by the independent Data and Safety Monitoring Board. It consisted in testing the possibility of masitinib to demonstrate superiority over placebo on the primary analysis set in the protocol, based on the EMA guideline on clinical trials in small populations (CHMP/EWP/83561/2005).

Alain Moussy, co-founder and CEO, stated “since the data remain blinded to AB Science, the efficacy results will not be known until the study is completed. However, we are very pleased that this pivotal clinical study has passed this important step successfully since the placebo effect was unknown as there has been no previous placebo controlled clinical study carried-out in this orphan disease”.

About masitinib

Masitinib is a new orally administered tyrosine kinase inhibitor that targets mast cells, important cells for immunity, as well as a limited number of kinases that play key roles in various cancers. Owing to its novel mechanism of action, masitinib can be developed in a large number of conditions in oncology, in inflammatory diseases, and in certain diseases of the central nervous system. Through its activity of inhibiting certain kinases that are essential in some oncogenic processes, masitinib may have an effect on tumor regression, alone or in combination with chemotherapy. Through its activity on the mast cell and certain kinases essential to the activation of the inflammatory cells and fibrosing tissue remodeling, masitinib can have an effect on the symptoms associated with some inflammatory and central nervous system diseases.

About AB Science

Founded in 2001, AB Science is a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), a new class of targeted molecules whose action is to modify signaling pathways within cells. Through these PKIs, the Company targets diseases with high unmet medical needs (cancer, inflammatory diseases, and central nervous system diseases), in both human and veterinary medicines.
AB Science has developed a proprietary portfolio of molecules and the Company’s lead compound, masitinib, has already been registered for veterinary medicine in Europe and in the USA, and is pursuing ten on-going phase 3 studies in human medicine in GIST, metastatic melanoma expressing JM mutation of c-Kit, multiple myeloma, mastocytosis, severe persistent asthma, rheumatoid arthritis, Alzheimer’s disease, progressive forms of multiple sclerosis, and in Amyotrophic Lateral Sclerosis. The company is headquartered in Paris, France, and listed on Euronext Paris (ticker: AB).

Further information is available on AB Science website: www.ab-science.com.

This document contains prospective information. No guarantee can be given as for the realization of these forecasts, which are subject to those risks described in documents deposited by the Company to the Authority of the financial markets, including trends of the economic conjuncture, the financial markets and the markets on which AB Science is present.

AB Science EN: http://hugin.info/155655/R/1742219/585574.pdf

 

Reposted with permission. The original article can be found at:

http://www.globenewswire.com/news-release/2013/11/11/588800/10057365/en/AB-Science-Masitinib-successfully-passes-futility-analysis-in-pivotal-clinical-study-in-Mastocytosis.html