AGIO

Scotiabank analyst Greg Harrison lowered the firm's price target on Agios Pharmaceuticals to $71 from $74 and keeps an Outperform rating on the shares. The firm believes Agios is on track to unlock Pyrukynd's "blockbuster potential," the analyst tells investors. However, the firm is lowering its price target on the stock to reflect Q1 reporting.

Agios Pharmaceuticals presented positive results from the Phase 3 ENERGIZE-T study investigating mitapivat, an oral, small molecule PK activator, in adults with transfusion-dependent alpha- or beta-thalassemia. These findings were shared in an oral presentation at the 66th American Society of Hematology Annual Meeting and Exposition in San Diego, California. In the ENERGIZE-T trial, mitapivat demonstrated a statistically significant reduction in transfusion burden compared to placebo in patients with transfusion-dependent alpha- or beta-thalassemia, achieving its primary endpoint. Additionally, the ENERGIZE-T study met all the key secondary endpoints, with mitapivat demonstrating a statistically significant reduction in additional measures of transfusion reduction response compared to placebo. In June 2024, Agios also presented positive results from the Phase 3 ENERGIZE study, which evaluated mitapivat in adults with non-transfusion-dependent alpha- or beta-thalassemia. The study's primary endpoint of transfusion reduction response was defined as a greater than or equal to 50% reduction in transfused red blood cell units with a reduction of greater than or equal to2 units of transfused RBCs in any consecutive 12-week period through Week 48 compared with baseline. A TRR was achieved by 30.4% of patients in the mitapivat arm compared to 12.6% of patients in the placebo arm. Additionally, mitapivat demonstrated statistically significant reductions in transfusion burden compared with placebo as measured by the three key secondary endpoints of transfusion reduction response reflective of durability of response up to 36 weeks during the 48-week double-blind period. The key secondary endpoint TRR2, defined as a greater than or equal to 50% reduction in transfused RBC units in any consecutive 24-week period through Week 48 compared with baseline, was achieved in 13.5% versus 2.3% of patients in the mitapivat and placebo arms, respectively. The key secondary endpoints TRR3 and TRR4 were defined as a greater than or equal to 33% and greater than or equal to 50% reduction in transfused RBC units, respectively, from Week 13 through Week 48 compared with baseline. TRR3 was achieved in 14.6% versus 1.1% of patients in the mitapivat and placebo arms, respectively, and TRR4 was achieved in 7.6% versus 1.1% of patients in the mitapivat and placebo arms, respectively. The results for the primary and key secondary endpoints were not driven by any of the individual prespecified subgroups, including but not limited to genotype and baseline transfusion burden, highlighting the overall robustness of the efficacy results. Further, 17 patients in the mitapivat arm compared with one patient in the placebo arm achieved the secondary endpoint of transfusion independence. Three patients in the mitapivat arm did not receive any transfusions during the 48-week double-blind period. Overall, during the 48-week double-blind period, incidence of adverse events was similar across the mitapivat and placebo arms. The proportion of patients with any treatment-emergent adverse events was 90.1% in patients on mitapivat and 83.5% in patients on placebo. The TEAEs leading to discontinuation of mitapivat, each of which occurred in one patient, were diarrhea, paresthesia oral, concurrent anxiety and insomnia, initial insomnia, supraventricular tachycardia, fatigue, hypertransaminasemia, hepatitis C, hepatic cancer, and renal mass. The TEAE that led to discontinuation of the one patient on placebo was blood creatine phosphokinase increased.

Scotiabank raised the firm's price target on Agios Pharmaceuticals to $75 from $53 and keeps an Outperform rating on the shares. The firm increased its price target on the stock given the promising results presented for its phase 3 ENERGIZE-T study as well as its announcement it filed for the regulatory approval of mitapivat for the treatment of thalassemia in four regions, the analyst tells investors.

Scotiabank raised the firm's price target on Agios Pharmaceuticals to $53 from $51 and keeps an Outperform rating on the shares. The company has a strong quarter marked by continued achievement of important clinical development milestones, the analyst tells investors. With a favorable cash position, the firm believes the company is well-equipped to support the advancement of mitapivat in sickle cell disease, thalassemia, and its earlier clinical pipeline.