American Society of Hematology annual meeting on December 9
Imetelstat Development in Hematologic Malignancies
Geron will be reporting the results of a phase 2 study of single agent imetelstat in patients with essential thrombocythemia (ET) in an oral presentation at the American Society of Hematology annual meeting on December 9. The ET study was designed to provide proof-of-concept for the potential use of imetelstat as a treatment for various hematologic myeloid malignancies, including myelofibrosis, myelodysplastic syndromes and acute myelogenous leukemias. JAK2 mutations are quantified during the trial to evaluate the effect of the drug on the malignant progenitor cells driving the disease. The top-line results from 14 patients enrolled in this study, all of whom were refractory to or intolerant of conventional therapies, showed a hematologic response rate of 100% (with 93% of patients achieving a complete response) and a molecular response rate of 86% among the seven patients who had a JAK2 mutation. These hematologic and molecular response rates, which exceeded the company's expectations, suggest a selective inhibition of the malignant progenitor cells responsible for the patients' malignancy, and therefore potential disease-modifying activity by imetelstat that may be applicable in other hematologic myeloid malignancies.
Market for myelofibrosis
From the following article:
Jefferies Equity Research Report
Cowen & Company Biotechnology Report
Ruben A. Mesa, Uma Yasothan & Peter Kirkpatrick
Nature Reviews Drug Discovery 11, 103-104 (February 2012)
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Analysing the market for myelofibrosis is Uma Yasothan, IMS Health, London, UK.
It is estimated that ~100,000 patients are afflicted with primary myelofibrosis, polycythaemia vera (PV) and essential thrombocythaemia (ET) in the United States alone2. Ruxolitinib (Jakafi; Incyte/Novartis), an oral Janus kinase inhibitor, is the first drug to be approved specifically to treat patients with intermediate or high-risk myelofibrosis, including primary myelofibrosis, post-PV myelofibrosis and post-ET myelofibrosis. It is launching with a fairly broad label into a disease area with a high unmet need and with plenty of opportunity to grow. Initial perceptions of physicians on efficacy and/or safety are also likely to change with the release of additional long-term safety data and survival data, which — if favourable — could contribute to increased uptake. Potential barriers to its adoption include its high cost; Incyte states that ruxolitinib will cost US$7,000 a month, or $84,000 per year, for insured patients.
Novartis holds the rights to ruxolitinib outside the United States, and its experience with imatinib (Gleevec) for chronic myeloid leukaemia is expected to be valuable in establishing a launch platform. Additional Phase II studies are currently underway with ruxolitinib for PV and ET, and other potential indications include rheumatoid arthritis. Analysts' projections for ruxolitinib sales reflect its promising clinical profile; sales estimates range from $56 million to $67 million in 2012, with projected worldwide peak sales of more than $1 billion by 2015 (Wei, T. et al. Jefferies Equity Research Report. 7 Nov 2011; Schmidt, E. & Bishop, N. Cowen & Company Biotechnology Report. 8 Dec 2011).