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Agios, seeking a new strategy, touts positive data and marketing deal

Agios Pharmaceuticals on Friday announced a marketing deal worth hundreds of millions of dollars, as well as positive data from two studies of a closely watched experimental medicine the company is developing for several blood diseases.The deal, with New York-based Royalty Pharma, has Agios selling both its royalty rights and its remaining milestone rights to the leukemia drug Idhifa for $255 million. Agios developed Idhifa and then sold most of the drug’s rights to Celgene, now a part of Bristol Myers Squibb. Agios said it will continue to co-promote Idhifa alongside Bristol Myers.The new data, meanwhile, provide early evidence that Agios’ drug mitapivat could help treat sickle cell disease and two types of thalassemia. So far, Agios has mostly focused on testing the drug in patients with a rare condition known as pyruvate kinase deficiency. But with the new clinical results, Agios is now readying pivotal programs for sickle cell and thalassemia.

Founded in 2008, Agios quickly gained attention for bringing two targeted cancer drugs to market unusually quickly. The company notched its first approval in 2017 with Idhifa, eights years after discovering the leukemia drug. It followed up a year later with another, similar medicine called Tibsovo.

Despite the regulatory wins, both Idhifa and Tibsovo are used in a very small group of leukemia patients. Revenues have reflected this, rising slowly as the company ended the first quarter with a $40 million net loss.

Agios has since made a push to aim toward higher-revenue drug markets, relying on mitapivat, a drug it had originally been developing only for pyruvate kinase deficiency. Sickle cell and thalassemia, though both rare diseases, represent larger opportunities. But treatment for both is quickly changing, which could make it more difficult for the company to find a place for its medicine.

Agios reported results from studies of mitapavit, which regulates a type of enzyme vital to the health of red blood cells, at the European Hematology Association’s virtual meeting on Friday.

The Phase 2 thalassemia trial enrolled 20 patients, who had either the alpha or beta forms of the disease.

By March 3, 13 of them had been in the trial long enough to be evaluated on its main goal: having an increase in hemoglobin concentration of at least 1 g/dL during at least one assessment between week 4 and week 12. Results showed 12 of those 13 patients hit the goal, with a mean hemoglobin change of 1.34 g/dL over weeks 4 through 12.

Two patients experienced a serious side effect during the trial, though Agios said neither was deemed related to mitapivat treatment. There was a third serious adverse event, renal dysfunction, after the data cutoff, which was judged to be related to the Agios drug. Agios said the patient recovered after stopping treatment.

Agios expects to finalize a development plan in both alpha and beta thalassemia, as well as in transfusion-dependent and in non-transfusion dependent patients, by the end of year. Pivotal programs could begin next year.

Additionally, Agios reported positive data from a small, proof-of-concept study that tested various doses of mitapivat in patients with sickle cell disease. Kennen MacKay, an analyst at RBC Capital Markets, said it was these results which were “the most significant data update” from Friday’s disclosures.

Of the eight patients who completed all the planned dose levels, seven showed some amount of hemoglobin increase, with five achieving at least a 1 g/dL increase. Agios said that one patient experienced a vaso-occlusive crisis during the drug tapering. Though such incidents are typical for sickle cell disease patients, investigators deemed the event possibly related to the drug.

The results indicate, for the first time, that PKR enzyme activation “has the potential to address chronic hemolytic anemia and impact markers of sickling in sickle cell disease patients,” Swee Lay Thein, chief of the Sickle Cell Branch of the National Heart, Lung, and Blood Institute said in a statement, referencing the drug’s mechanism of action. Thein also served as the principal investigator of the study.

Tyler Van Buren, an analyst at Piper Sandler, noted that the data “compares favorably” to what’s been reported for other sickle cell drugs. But genetic medicine approaches, which hold the potential to dramatically change the course of the disease, are advancing too, with new data also presented at EHA.

Agios said the Phase 1 data supports its decision to push mitapivat into pivotal development.