ImetelChat

Where does this leave Geron/Imetelstat? Looks scary or don't I get it?



Janssen's Eprex effective in late-stage MDS study
Jun 11 2016, 08:58 ET | By: Douglas W. House, SA News Editor

A Phase 3 clinical trial, EPOANE 3021, evaluating Janssen's (NYSE:JNJ) EPREX (epoetin alfa) for the treatment of anemia in adult patients with low or intermediate-1 risk myelodysplastic syndromes (MDS) met its primary endpoint. The results were presented at the 21st Annual Congress of the European Hematology Association in Copenhagen.

EPOANE 3021 randomized 130 subjects to receive either epoetin alfa or placebo over 24 weeks. Patients receiving epoetin alfa experienced a statistically significantly higher erythroid response rate (31.8%) compared to placebo (4.4%) (p<0.001). Also, significantly less patients in the treatment group required a transfusion (24.7% versus 54.1%). Time to first transfusion favored epoetin alfa as well.

The data have been submitted to the regulatory agency in France, the reference health authority, seeking an expanded label for EPREX in the EU to include this new use. A decision should be made in the next few months.

EPREX is currently approved for the treatment of anemia in patients with renal insufficiency/failure, those with non-myeloid malignancies receiving chemo, adults with mild-to-moderate anemia who scheduled for elective surgery and to augment autologous blood collection in anemic adults who are scheduled for major elective surgery.

Anemia affects the vast majority of MDS sufferers but there are no erythropoiesis stimulating agents approved for this patient population.

Interesting post, Jayfish. For me it doens't look scary as it is a drug that stimulates the production of blood cells. It doesn't look like a silver bullet as the compound doesn't seem to target the underlying disease. Although the Mayo study (see below) didn't observe changes on the mutations, imetelstat clearly had an effect on various major disease markers other than transfusion dependence, such as leukocytosis, thrombocytosis and enlarged spleen.

Conclusions from the Mayo MDS study "Imetelstat therapy in refractory anemia with ring sideroblasts with or without thrombocytosis":
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Three (38%) of eight transfusion-dependent patients became transfusion-independent at a median time of 11 weeks (range, 9–14) and their response lasted for a median of 28 weeks (range, 9–37; Figure 1); one of the three patients also had resolution of leukocytosis and thrombocytosis. A fourth patient experienced >50% decrease in palpable spleen size (16 cm at baseline) and a decrease in transfusion need. Two additional patients with thrombocytosis and leukocytosis experienced normalization of counts.
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The current study suggests drug activity in imetelstat-treated patients with RARS or RARS-T.
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Link: http://www.nature.com/bcj/journal/v6/n3 ... 1613a.html

As long as this EPOANE 3021 looks like a palliative drug with a response rate of 31,8% (compare imetelstat with 38% and a deeper response), I see it as a complementary treatment.

Thanks, Fishermangents. Thanks what I was thinking, but without the knowledge. It seems this drug treats one of the possible side affects of Imetelstat treatment and/or a symptom of MDS, but not the underlying MDS disease.