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CPI-0610 receives fast track designation for MF

Micrograph showing myelofibrosis

The U.S. Food and Drug Administration (FDA) has granted fast track designation to CPI-0610 for the treatment of myelofibrosis (MF).

CPI-0610 is a BET inhibitor being developed by Constellation Pharmaceuticals, Inc.

The company said results from preclinical studies and translational insights from the first-in-human study of CPI-0610 led to the prioritization of CPI-0610 as a potential treatment for MF.

The company is now enrolling patients in the phase 2 portion of the phase 1/2 MANIFEST trial (NCT02158858).

In this trial, researchers are testing CPI-0610 alone or in combination with ruxolitinib in the following patient groups:

  • Transfusion-dependent or -independent MF patients who have previously received a JAK inhibitor and are ineligible for or cannot tolerate additional JAK inhibitor therapy, lost response to a JAK inhibitor, or are resistant or refractory to JAK inhibition
  • MF patients who are transfusion-dependent or -independent whose disease is not being adequately controlled by ruxolitinib
  • MF patients who are anemic and have not previously received a JAK inhibitor.

According to Constellation Pharmaceuticals, CPI-0610 demonstrated clinical activity in the first four patients treated on this trial, all of whom previously received a JAK inhibitor.

The researchers observed reductions in spleen volume and improvements in hemoglobin levels in patients who received monotherapy (n=2) or CPI-0610 plus ruxolitinib (n=2).

One patient treated with the combination experienced resolution of thrombocytosis and became transfusion-independent.

The company said proof-of-concept data from this trial are expected in mid-2019.

About fast track designation

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.

Fast track designation facilitates frequent interactions with the FDA review team, including meetings to discuss the product’s development plan and written communications about issues such as trial design and use of biomarkers.

Products that receive fast track designation may be eligible for accelerated approval and priority review if relevant criteria are met. Such products may also be eligible for rolling review, which allows a developer to submit individual sections of a product’s application for review as they are ready, rather than waiting until all sections are complete.

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Micrograph showing myelofibrosis

The U.S. Food and Drug Administration (FDA) has granted fast track designation to CPI-0610 for the treatment of myelofibrosis (MF).

CPI-0610 is a BET inhibitor being developed by Constellation Pharmaceuticals, Inc.

The company said results from preclinical studies and translational insights from the first-in-human study of CPI-0610 led to the prioritization of CPI-0610 as a potential treatment for MF.

The company is now enrolling patients in the phase 2 portion of the phase 1/2 MANIFEST trial (NCT02158858).

In this trial, researchers are testing CPI-0610 alone or in combination with ruxolitinib in the following patient groups:

  • Transfusion-dependent or -independent MF patients who have previously received a JAK inhibitor and are ineligible for or cannot tolerate additional JAK inhibitor therapy, lost response to a JAK inhibitor, or are resistant or refractory to JAK inhibition
  • MF patients who are transfusion-dependent or -independent whose disease is not being adequately controlled by ruxolitinib
  • MF patients who are anemic and have not previously received a JAK inhibitor.

According to Constellation Pharmaceuticals, CPI-0610 demonstrated clinical activity in the first four patients treated on this trial, all of whom previously received a JAK inhibitor.

The researchers observed reductions in spleen volume and improvements in hemoglobin levels in patients who received monotherapy (n=2) or CPI-0610 plus ruxolitinib (n=2).

One patient treated with the combination experienced resolution of thrombocytosis and became transfusion-independent.

The company said proof-of-concept data from this trial are expected in mid-2019.

About fast track designation

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.

Fast track designation facilitates frequent interactions with the FDA review team, including meetings to discuss the product’s development plan and written communications about issues such as trial design and use of biomarkers.

Products that receive fast track designation may be eligible for accelerated approval and priority review if relevant criteria are met. Such products may also be eligible for rolling review, which allows a developer to submit individual sections of a product’s application for review as they are ready, rather than waiting until all sections are complete.

Micrograph showing myelofibrosis

The U.S. Food and Drug Administration (FDA) has granted fast track designation to CPI-0610 for the treatment of myelofibrosis (MF).

CPI-0610 is a BET inhibitor being developed by Constellation Pharmaceuticals, Inc.

The company said results from preclinical studies and translational insights from the first-in-human study of CPI-0610 led to the prioritization of CPI-0610 as a potential treatment for MF.

The company is now enrolling patients in the phase 2 portion of the phase 1/2 MANIFEST trial (NCT02158858).

In this trial, researchers are testing CPI-0610 alone or in combination with ruxolitinib in the following patient groups:

  • Transfusion-dependent or -independent MF patients who have previously received a JAK inhibitor and are ineligible for or cannot tolerate additional JAK inhibitor therapy, lost response to a JAK inhibitor, or are resistant or refractory to JAK inhibition
  • MF patients who are transfusion-dependent or -independent whose disease is not being adequately controlled by ruxolitinib
  • MF patients who are anemic and have not previously received a JAK inhibitor.

According to Constellation Pharmaceuticals, CPI-0610 demonstrated clinical activity in the first four patients treated on this trial, all of whom previously received a JAK inhibitor.

The researchers observed reductions in spleen volume and improvements in hemoglobin levels in patients who received monotherapy (n=2) or CPI-0610 plus ruxolitinib (n=2).

One patient treated with the combination experienced resolution of thrombocytosis and became transfusion-independent.

The company said proof-of-concept data from this trial are expected in mid-2019.

About fast track designation

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.

Fast track designation facilitates frequent interactions with the FDA review team, including meetings to discuss the product’s development plan and written communications about issues such as trial design and use of biomarkers.

Products that receive fast track designation may be eligible for accelerated approval and priority review if relevant criteria are met. Such products may also be eligible for rolling review, which allows a developer to submit individual sections of a product’s application for review as they are ready, rather than waiting until all sections are complete.

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