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The Food and Drug Administration has granted fast-track designation to CX-01 as a treatment for patients older than 60 years receiving induction therapy for newly diagnosed acute myeloid leukemia (AML).

CX-01 also has orphan drug designation from the FDA.

CX-01 is a polysaccharide derived from heparin thought to enhance chemotherapy by disrupting leukemia cell adhesion in bone marrow. Cantex Pharmaceuticals is conducting a randomized, phase 2b study to determine whether CX-01 can improve the efficacy of frontline chemotherapy in patients with AML.

This study builds upon results of a pilot study, which were published in Blood Advances (Blood Adv. 2018 Feb 27;2[4]:381-9). The pilot study enrolled 12 adults with newly diagnosed AML who received CX-01 as a continuous infusion for 7 days, along with standard induction chemotherapy (cytarabine and idarubicin).


A total of 11 patients achieved morphological complete remission after one cycle of induction. This included two patients who did not complete induction. All patients received subsequent therapy – consolidation, salvage, or transplant – on or off study.

At a median follow-up of 24 months, eight patients were still alive. Two patients died of transplant-related complications, one died of infectious complications, and one died of cerebral hemorrhage. The median disease-free survival was 14.8 months, and the median overall survival was not reached.

There were five serious adverse events in five patients; most were considered unrelated to CX-01, but a case of grade 4 sepsis was possibly related.

The FDA’s fast-track development program is designed to expedite clinical development and submission of applications for products with the potential to treat serious or life-threatening conditions and address unmet medical needs.

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The Food and Drug Administration has granted fast-track designation to CX-01 as a treatment for patients older than 60 years receiving induction therapy for newly diagnosed acute myeloid leukemia (AML).

CX-01 also has orphan drug designation from the FDA.

CX-01 is a polysaccharide derived from heparin thought to enhance chemotherapy by disrupting leukemia cell adhesion in bone marrow. Cantex Pharmaceuticals is conducting a randomized, phase 2b study to determine whether CX-01 can improve the efficacy of frontline chemotherapy in patients with AML.

This study builds upon results of a pilot study, which were published in Blood Advances (Blood Adv. 2018 Feb 27;2[4]:381-9). The pilot study enrolled 12 adults with newly diagnosed AML who received CX-01 as a continuous infusion for 7 days, along with standard induction chemotherapy (cytarabine and idarubicin).


A total of 11 patients achieved morphological complete remission after one cycle of induction. This included two patients who did not complete induction. All patients received subsequent therapy – consolidation, salvage, or transplant – on or off study.

At a median follow-up of 24 months, eight patients were still alive. Two patients died of transplant-related complications, one died of infectious complications, and one died of cerebral hemorrhage. The median disease-free survival was 14.8 months, and the median overall survival was not reached.

There were five serious adverse events in five patients; most were considered unrelated to CX-01, but a case of grade 4 sepsis was possibly related.

The FDA’s fast-track development program is designed to expedite clinical development and submission of applications for products with the potential to treat serious or life-threatening conditions and address unmet medical needs.

 

The Food and Drug Administration has granted fast-track designation to CX-01 as a treatment for patients older than 60 years receiving induction therapy for newly diagnosed acute myeloid leukemia (AML).

CX-01 also has orphan drug designation from the FDA.

CX-01 is a polysaccharide derived from heparin thought to enhance chemotherapy by disrupting leukemia cell adhesion in bone marrow. Cantex Pharmaceuticals is conducting a randomized, phase 2b study to determine whether CX-01 can improve the efficacy of frontline chemotherapy in patients with AML.

This study builds upon results of a pilot study, which were published in Blood Advances (Blood Adv. 2018 Feb 27;2[4]:381-9). The pilot study enrolled 12 adults with newly diagnosed AML who received CX-01 as a continuous infusion for 7 days, along with standard induction chemotherapy (cytarabine and idarubicin).


A total of 11 patients achieved morphological complete remission after one cycle of induction. This included two patients who did not complete induction. All patients received subsequent therapy – consolidation, salvage, or transplant – on or off study.

At a median follow-up of 24 months, eight patients were still alive. Two patients died of transplant-related complications, one died of infectious complications, and one died of cerebral hemorrhage. The median disease-free survival was 14.8 months, and the median overall survival was not reached.

There were five serious adverse events in five patients; most were considered unrelated to CX-01, but a case of grade 4 sepsis was possibly related.

The FDA’s fast-track development program is designed to expedite clinical development and submission of applications for products with the potential to treat serious or life-threatening conditions and address unmet medical needs.

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