Impact Biomedicines to develop fedratinib to treat myelofibrosis and polycythemia vera.

Impact Biomedicines announced its launch to pioneer the development of life-changing treatments for patients with myeloproliferative neoplasms and other cancers. The Company�s pipeline is centered around fedratinib, a potent and highly selective oral small molecule JAK2 kinase inhibitor that is being developed initially for the treatment of myelofibrosis (MF) and polycythemia vera (PV). In conjunction with this launch, Impact is pleased to share that the FDA has removed the clinical hold placed on fedratinib and that the company has received $22.5 million from Medicxi through a Series A financing.

Impact was formed in 2016 after acquisition of Sanofi�s full rights for the global development and commercialization of fedratinib. The majority equity holders in Impact include the co-founders and Medicxi, with Sanofi retaining a minority stake. In 2013, the development of fedratinib was discontinued by Sanofi after the FDA issued a clinical hold subsequent to reports of a few potential cases of Wernicke�s encephalopathy (WE), an acute neurological condition usually indicative of a vitamin B deficiency, in patients participating in fedratinib clinical trials. Following a Type A meeting and review of additional data, the FDA concluded that Impact provided the necessary documentation to support lifting the clinical hold � opening the path for Impact�s continued development of fedratinib.

The clinical package for fedratinib includes data from a total of 18 studies completed in 877 subjects. In JAKARTA-1, a completed international Phase III pivotal trial for the treatment of myelofibrosis, fedratinib met its primary and secondary endpoints by reducing spleen size in 47% of patients by greater than 35% at 24 weeks (p<0.0001) and improving symptom score in 36 of patients by greater than 50 at 24 weeks p>< 0.0001). Comparable responses were seen in patients with normal or low platelet counts and thrombocytopenia was similar between placebo and the target dose of 400mg. In JAKARTA-2, a follow-on study in patients who were unresponsive to all other available therapies, including patients who were either Jakafi (ruxolitinib) resistant or intolerant, fedratinib showed similar activity. In that study, 55% of patients who had failed or were intolerant to ruxolitinib experienced a spleen size reduction of >35% with fedratinib. Notably, responses were noted in 63% of patients intolerant to ruxolitinib and 61% of patients who had lost ruxolitinib response.

Currently, ruxolitinib is the only drug approved by the FDA to treat patients with MF and PV. The most common adverse events for fedratinib were hematological (anemia) and gastrointestinal (nausea, diarrhea and vomiting). The results of these trials have been published in leading peer reviewed journals.