The Future is in Sight Again

At Impact Biomedicines, our late-stage pipeline is centered around fedratinib, a selective, oral small molecule Janus kinase 2 (JAK2) inhibitor, which is currently under clinical investigation for the treatment of myelofibrosis (MF).

That Future is Selective

Myeloproliferative neoplasms (MPN), including MF, are characterized by the mutations of JAK2, MPL or CALR genes, but the mutational activation of JAK2 is the primary driver of disease. Current research suggests that many patients with MF are unable to tolerate, do not respond or become refractory to current JAK1/JAK2 inhibitor therapy. Therefore, selective inhibition of JAK2 may represent a promising treatment strategy.

Responding to Unmet Patient Needs

The fedratinib program is approaching final regulatory filings after it was prematurely stopped in 2013.

In 2013, the U.S. Food and Drug Administration (FDA) imposed a clinical hold based on reports of eight potential cases of Wernicke encephalopathy (WE), an acute neurological condition associated with vitamin B (thiamine) deficiency, out of 877 patients from 18 completed studies.

Encouraged by otherwise promising clinical results, Impact obtained worldwide rights to fedratinib and submitted documentation to the FDA to support lifting the hold. After reviewing these data, the FDA agreed to lift the clinical hold and allow Impact to continue development.

Impact is now working with global regulatory authorities to initiate registration activities. The clinical package for fedratinib includes results from the JAKARTA Phase 3 trial in MF and a Phase 2 study (JAKARTA-2) assessing fedratinib in patients unresponsive to other therapies that have been published.1,2

“The decision to discontinue the development of fedratinib was heartbreaking for the patients who were experiencing positive responses while in clinical trials. There are very limited therapeutic options for these conditions and fedratinib was active in most patients when nothing else had worked. Because of the very high unmet medical need in MF, Impact acquired fedratinib and worked diligently with the FDA to investigate and successfully address their concerns,” said John Hood, Ph.D. Chief Executive Officer and Founder, Impact Biomedicines.

“We are now focused on completing a regulatory filing based on the robust fedratinib data set.”

Responding to Unmet Needs of Patients with MPN

The safety and efficacy of fedratinib has been explored in a large clinical development program that includes 18 completed studies that enrolled a total of 877 patients. The results of these trials have been published in leading peer-reviewed journals including The Lancet Haematology and JAMA Oncology.

Impact will present additional retrospective data on the safety of fedratinib at the 59th American Society of Hematology (ASH) Annual Meeting, taking place on December 9-12, 2017 in Atlanta, GA.

The details of the presentation are as follows:

Session: 634 – Myeloproliferative Syndromes: Clinical: Poster III
Poster Title: Case Series of Potential Wernicke’s Encephalopathy in Patients treated with Fedratinib* Presenter: John Hood, Ph.D.
Date: Monday, December 11, 2017
Presentation time: 6:00-8:00 PM ET
Location: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2

Article Sources

1 Pardanani A, Harrison C, Cortes JE, et al. Safety and efficacy of fedratinib in patients with primary or secondary myelofibrosis. A randomized clinical trial. JAMA Oncol. 2015; 1(5): 643-651.
2 Harrison CN, Schaap N, Vannucchi AM, et al. Janus kinase-2 inhibitor fedratinib in patients with myelofibrosis previously treated with ruxolitinib (JAKARTA-2): a single-arm, open-label, non-randomised, phase 2, muticentre study. Lancet Haematol. 2017 Jul;4(7): e317-e324. doi: 10.1016/S2352-3026(17)30088-1. Epub 2017 Jun 8

The Future is in Sight Again

At Impact Biomedicines, our late-stage pipeline is centered around fedratinib, a selective, oral small molecule Janus kinase 2 (JAK2) inhibitor, which is currently under clinical investigation for the treatment of myelofibrosis (MF).

That Future is Selective

Myeloproliferative neoplasms (MPN), including MF, are characterized by the mutations of JAK2, MPL or CALR genes, but the mutational activation of JAK2 is the primary driver of disease. Current research suggests that many patients with MF are unable to tolerate, do not respond or become refractory to current JAK1/JAK2 inhibitor therapy. Therefore, selective inhibition of JAK2 may represent a promising treatment strategy.

Responding to Unmet Patient Needs

The fedratinib program is approaching final regulatory filings after it was prematurely stopped in 2013.

In 2013, the U.S. Food and Drug Administration (FDA) imposed a clinical hold based on reports of eight potential cases of Wernicke encephalopathy (WE), an acute neurological condition associated with vitamin B (thiamine) deficiency, out of 877 patients from 18 completed studies.

Encouraged by otherwise promising clinical results, Impact obtained worldwide rights to fedratinib and submitted documentation to the FDA to support lifting the hold. After reviewing these data, the FDA agreed to lift the clinical hold and allow Impact to continue development.

Impact is now working with global regulatory authorities to initiate registration activities. The clinical package for fedratinib includes results from the JAKARTA Phase 3 trial in MF and a Phase 2 study (JAKARTA-2) assessing fedratinib in patients unresponsive to other therapies that have been published.1,2

“The decision to discontinue the development of fedratinib was heartbreaking for the patients who were experiencing positive responses while in clinical trials. There are very limited therapeutic options for these conditions and fedratinib was active in most patients when nothing else had worked. Because of the very high unmet medical need in MF, Impact acquired fedratinib and worked diligently with the FDA to investigate and successfully address their concerns,” said John Hood, Ph.D. Chief Executive Officer and Founder, Impact Biomedicines.

“We are now focused on completing a regulatory filing based on the robust fedratinib data set.”

Responding to Unmet Needs of Patients with MPN

The safety and efficacy of fedratinib has been explored in a large clinical development program that includes 18 completed studies that enrolled a total of 877 patients. The results of these trials have been published in leading peer-reviewed journals including The Lancet Haematology and JAMA Oncology.

Impact will present additional retrospective data on the safety of fedratinib at the 59th American Society of Hematology (ASH) Annual Meeting, taking place on December 9-12, 2017 in Atlanta, GA.

The details of the presentation are as follows:

Session: 634 – Myeloproliferative Syndromes: Clinical: Poster III
Poster Title: Case Series of Potential Wernicke’s Encephalopathy in Patients treated with Fedratinib* Presenter: John Hood, Ph.D.
Date: Monday, December 11, 2017
Presentation time: 6:00-8:00 PM ET
Location: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2

Article Sources

1 Pardanani A, Harrison C, Cortes JE, et al. Safety and efficacy of fedratinib in patients with primary or secondary myelofibrosis. A randomized clinical trial. JAMA Oncol. 2015; 1(5): 643-651.
2 Harrison CN, Schaap N, Vannucchi AM, et al. Janus kinase-2 inhibitor fedratinib in patients with myelofibrosis previously treated with ruxolitinib (JAKARTA-2): a single-arm, open-label, non-randomised, phase 2, muticentre study. Lancet Haematol. 2017 Jul;4(7): e317-e324. doi: 10.1016/S2352-3026(17)30088-1. Epub 2017 Jun 8

The Future is in Sight Again

At Impact Biomedicines, our late-stage pipeline is centered around fedratinib, a selective, oral small molecule Janus kinase 2 (JAK2) inhibitor, which is currently under clinical investigation for the treatment of myelofibrosis (MF).

That Future is Selective

Myeloproliferative neoplasms (MPN), including MF, are characterized by the mutations of JAK2, MPL or CALR genes, but the mutational activation of JAK2 is the primary driver of disease. Current research suggests that many patients with MF are unable to tolerate, do not respond or become refractory to current JAK1/JAK2 inhibitor therapy. Therefore, selective inhibition of JAK2 may represent a promising treatment strategy.

Responding to Unmet Patient Needs

The fedratinib program is approaching final regulatory filings after it was prematurely stopped in 2013.

In 2013, the U.S. Food and Drug Administration (FDA) imposed a clinical hold based on reports of eight potential cases of Wernicke encephalopathy (WE), an acute neurological condition associated with vitamin B (thiamine) deficiency, out of 877 patients from 18 completed studies.

Encouraged by otherwise promising clinical results, Impact obtained worldwide rights to fedratinib and submitted documentation to the FDA to support lifting the hold. After reviewing these data, the FDA agreed to lift the clinical hold and allow Impact to continue development.

Impact is now working with global regulatory authorities to initiate registration activities. The clinical package for fedratinib includes results from the JAKARTA Phase 3 trial in MF and a Phase 2 study (JAKARTA-2) assessing fedratinib in patients unresponsive to other therapies that have been published.1,2

“The decision to discontinue the development of fedratinib was heartbreaking for the patients who were experiencing positive responses while in clinical trials. There are very limited therapeutic options for these conditions and fedratinib was active in most patients when nothing else had worked. Because of the very high unmet medical need in MF, Impact acquired fedratinib and worked diligently with the FDA to investigate and successfully address their concerns,” said John Hood, Ph.D. Chief Executive Officer and Founder, Impact Biomedicines.

“We are now focused on completing a regulatory filing based on the robust fedratinib data set.”

Responding to Unmet Needs of Patients with MPN

The safety and efficacy of fedratinib has been explored in a large clinical development program that includes 18 completed studies that enrolled a total of 877 patients. The results of these trials have been published in leading peer-reviewed journals including The Lancet Haematology and JAMA Oncology.

Impact will present additional retrospective data on the safety of fedratinib at the 59th American Society of Hematology (ASH) Annual Meeting, taking place on December 9-12, 2017 in Atlanta, GA.

The details of the presentation are as follows:

Session: 634 – Myeloproliferative Syndromes: Clinical: Poster III
Poster Title: Case Series of Potential Wernicke’s Encephalopathy in Patients treated with Fedratinib* Presenter: John Hood, Ph.D.
Date: Monday, December 11, 2017
Presentation time: 6:00-8:00 PM ET
Location: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2

Article Sources

1 Pardanani A, Harrison C, Cortes JE, et al. Safety and efficacy of fedratinib in patients with primary or secondary myelofibrosis. A randomized clinical trial. JAMA Oncol. 2015; 1(5): 643-651.
2 Harrison CN, Schaap N, Vannucchi AM, et al. Janus kinase-2 inhibitor fedratinib in patients with myelofibrosis previously treated with ruxolitinib (JAKARTA-2): a single-arm, open-label, non-randomised, phase 2, muticentre study. Lancet Haematol. 2017 Jul;4(7): e317-e324. doi: 10.1016/S2352-3026(17)30088-1. Epub 2017 Jun 8