Agios Pharmaceuticals Inc Stock Price, News & Analysis

Agios Pharmaceuticals announced on August 17, 2021, that the FDA has accepted its New Drug Application (NDA) for mitapivat, aimed at treating adults with pyruvate kinase deficiency. This NDA received a Priority Review designation, expediting the review process to a PDUFA date of February 17, 2022. Mitapivat is being positioned as a potential first disease-modifying therapy for this chronic condition, with the FDA’s designation indicating significant improvements in treatment.

Agios Pharmaceuticals (AGIO) recently completed regulatory submissions for mitapivat, targeting adults with Pyruvate Kinase deficiency in the U.S. and EU. The company presented positive Phase 3 study results for mitapivat at the EHA Virtual Congress, showing strong efficacy, including an 80% hemoglobin increase in thalassemia patients. Q2 2021 R&D expenses rose to $62 million, while net loss decreased to $86.2 million. Agios reported $1.7 billion in cash reserves, sufficient for ongoing operations and future developments, including two planned Phase 3 trials and a Phase 2/3 trial in sickle cell disease.

Agios Pharmaceuticals (NASDAQ: AGIO) has scheduled a conference call for July 29, 2021, at 8:00 a.m. ET to discuss its second quarter 2021 financial results and business highlights. The call will be accessible via telephone or live webcast through the company’s website. Agios specializes in developing medicines for genetically defined diseases, with its lead drug candidate, mitapivat, targeting hemolytic anemias.

Agios Pharmaceuticals (NASDAQ: AGIO) has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency for mitapivat, aimed at treating adults with pyruvate kinase deficiency. This follows a recent New Drug Application (NDA) to the FDA. Mitapivat is designed to offer a first-in-class therapy for this chronic hemolytic anemia, characterized by serious complications and no approved treatments currently available. The application is backed by pivotal study results and ongoing studies to evaluate long-term efficacy.

Agios Pharmaceuticals (NASDAQ: AGIO) has announced the launch of myAgios® patient support services for individuals with pyruvate kinase deficiency. This program connects patients and caregivers with dedicated Patient Support Managers, offering tailored support and educational resources. The initiative responds to the unique challenges faced by this rare disease community, aiming to improve care management. Additionally, a virtual webinar series will start on June 23, 2021, focusing on PK deficiency, enhancing community engagement and awareness.

Agios Pharmaceuticals (NASDAQ: AGIO) has submitted a New Drug Application (NDA) to the FDA for mitapivat, targeting adults with pyruvate kinase (PK) deficiency, a chronic hemolytic anemia with serious complications. This marks a pivotal step as no therapies currently exist for PK deficiency. The NDA is based on two pivotal studies, ACTIVATE and ACTIVATE-T, showcasing long-term safety and efficacy. Agios aims to address significant unmet needs in this patient community. Mitapivat is not yet approved for any use.

Agios Pharmaceuticals announced positive results from its Phase 2 study of mitapivat in adults with non-transfusion dependent α- or β-thalassemia. The primary endpoint was met, with 80% of patients showing a hemoglobin increase of ≥1.0 g/dL during Weeks 4-12. Mitapivat was well tolerated, with a safety profile consistent with previous studies. The trial involved 20 patients—5 with α-thalassemia and 15 with β-thalassemia. The company plans to initiate two Phase 3 studies in the second half of 2021, focusing on treatment options for those with thalassemia.

Agios Pharmaceuticals (NASDAQ: AGIO) announced its plan to file for regulatory approval for mitapivat, a therapy for pyruvate kinase (PK) deficiency, in the U.S. this quarter and in the EU by mid-2021. Results from the Phase 3 ACTIVATE and ACTIVATE-T studies indicate mitapivat significantly improved hemoglobin levels and reduced transfusion burdens in adults with PK deficiency. With a favorable safety profile, the findings highlight mitapivat's potential as the first disease-modifying treatment for this condition. An investor webcast is scheduled for later today.

Agios Pharmaceuticals will present clinical data on its drug mitapivat at the European Hematology Association Annual Congress from June 9-17, 2021. Significant findings will be shared from Phase 3 studies on adults with pyruvate kinase deficiency, both transfusion-dependent and non-transfusion-dependent. Two oral presentations and several poster presentations will be made, highlighting the drug's effectiveness. An investor webcast is scheduled for June 11, 2021, to discuss these findings in detail, emphasizing Agios' commitment to addressing genetically defined diseases.