{"id":394791,"date":"2020-12-07T16:33:19","date_gmt":"2020-12-07T21:33:19","guid":{"rendered":"http:\/\/www.marketnewsdesk.com\/?p=394791"},"modified":"2020-12-07T16:33:19","modified_gmt":"2020-12-07T21:33:19","slug":"agios-announces-updated-data-from-phase-1-study-of-mitapivat-first-in-class-pkr-activator-in-sickle-cell-disease","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/agios-announces-updated-data-from-phase-1-study-of-mitapivat-first-in-class-pkr-activator-in-sickle-cell-disease\/","title":{"rendered":"Agios Announces Updated Data from Phase 1 Study of Mitapivat, First-in-Class PKR Activator, in Sickle Cell Disease"},"content":{"rendered":"
\n

\n \u2013<\/em>
\n Treatment with Mitapivat <\/em>
\n Induced <\/em>
\n Hemoglobin Increase of <\/em>
\n \u22651.0 g\/dL<\/em>
\n in 6 of 11 (55%) Efficacy Evaluable Patients<\/em>
\n ,<\/em>
\n Decreased Markers of Hemolysis, <\/em>
\n Reduced 2,3-DPG<\/em>
\n and<\/em>
\n Increased ATP<\/em>
\n \u2013<\/em>
\n \n <\/p>\n

\n \u2013 Safety Profile <\/em>
\n Generally <\/em>
\n Consistent with Previous<\/em>
\n ly Presented<\/em>
\n Data in Patients with Pyruvate Kinase Deficiency<\/em>
\n and Thalassemia <\/em>
\n \u2013<\/em>\n <\/p>\n

\n \u2013 Data Support Advancement of Mitapivat to\u00a0Pivotal Development\u00a0in Sickle Cell Disease<\/em>
\n ; <\/em>
\n Company Expects to Initiate <\/em>
\n Pivotal Program in 2021 <\/em>
\n \u2013<\/em>\n <\/p>\n

\n \u2013 Company to Host Investor Event and Webcast <\/em>
\n Tomorrow<\/em>
\n , December 8, at\u00a08:00 a.m. ET\u00a0\u2013<\/em>\n <\/p>\n

CAMBRIDGE, Mass., Dec. 07, 2020 (GLOBE NEWSWIRE) — Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today reported updated data from a Phase 1 trial of mitapivat, the company\u2019s first-in-class pyruvate kinase R (PKR) activator, in patients with sickle cell disease. The study is being conducted in collaboration with the National Institutes of Health (NIH) as part of a cooperative research and development agreement. Data from the study were featured in an oral presentation at the American Society of Hematology (ASH) Annual Meeting, which is being held virtually. Mitapivat is an investigational, oral, small molecule allosteric activator of wild-type and a variety of mutated PKR enzymes.<\/p>\n

As of the October 6, 2020 data cut-off, six of 11 efficacy evaluable patients (55%) achieved a hemoglobin increase of \u22651.0 g\/dL from baseline. The mean maximal hemoglobin increase among all efficacy evaluable patients was 1.3 g\/dL, and the mean maximal hemoglobin increase among responders was 1.9 g\/dL. Treatment with mitapivat was associated with decreases in hemolytic markers such as bilirubin, lactate dehydrogenase (LDH) and reticulocytes. As expected, dose-dependent decreases in 2,3-diphosphoglycerate (2,3-DPG) and increases in adenosine triphosphate (ATP) levels were observed, consistent with the proposed mechanism of action. Adverse events (AEs) reported during the study were generally consistent with those previously reported in pyruvate kinase (PK) deficiency and thalassemia studies.<\/p>\n

\u201cThe hemoglobin improvement, in conjunction with improvements in markers of hemolysis, induced by mitapivat in sickle cell disease patients is highly encouraging, particularly given the short duration patients are on treatment at the higher dose levels,\u201d said Swee Lay Thein, M.B.B.S., F.R.C.P., F.R.C.Path., D.Sc., chief of the Sickle Cell Branch of the National Heart, Lung, and Blood Institute, NIH, and the principal investigator of the study. \u201cI believe mitapivat may be a promising sickle cell disease treatment option with a well-tolerated safety profile and convenient oral administration. I look forward to further investigating the long-term effects of mitapivat in an ongoing sickle cell disease extension study and to working with Agios to continue to advance mitapivat on behalf of this patient community, a historically under-served population in tremendous need of new treatment options.\u201d<\/p>\n

\u201cWe are pleased to continue collaborating with Dr. Thein and her colleagues at the NIH to further elucidate mitapivat\u2019s potential to improve red blood cell health, energy and longevity for people living with sickle cell disease, a debilitating, inherited, lifelong red blood cell disorder,\u201d said Chris Bowden, M.D., chief medical officer at Agios. \u201cWe are excited by the updated results of this study and plan to build on our findings by initiating a pivotal study of mitapivat in adults with sickle cell disease next year.\u201d<\/p>\n

\n Updated Data from the <\/strong>
\n M<\/strong>
\n itapivat Phase 1 Trial in Sickle Cell Disease<\/strong>
\n
The ongoing Phase 1 study, which can enroll up to 25 patients, is evaluating the efficacy, safety, pharmacokinetics and pharmacodynamics of treatment with mitapivat in adults with sickle cell disease. As of the data cut-off, 12 patients were dosed and 11 were evaluable for efficacy. One patient discontinued within the first week due to a pre-existing condition and was subsequently lost to follow-up, one patient discontinued prior to completing the 100 mg BID dose level, one patient is ongoing and nine had completed all planned dose levels. All 11 efficacy evaluable patients have received three ascending dose levels of mitapivat (5 mg BID, 20 mg BID, 50 mg BID) for two weeks\u2019 duration, respectively, and of these, three patients have received an additional ascending dose of 100 mg BID for two weeks. All underwent a 12- or 15-day drug taper after completing the dosing.<\/p>\n

\n
\n Safety Data<\/em>
\n <\/strong>
\n
A safety analysis conducted for all 12 patients as of the data cut-off demonstrated:<\/p>\n