Dive Brief:

• An experimental drug from Agios Pharmaceuticals succeeded in the first of two Phase 3 trials in a rare form of anemia known as pyruvate kinase deficiency, or PKD.

• The Agios drug, mitapivat, led to a sustained boost in hemoglobin levels — at least 1.5 gram per deciliter of blood (g/dL), the study’s main goal — in 40% of PKD patients randomized to receive treatment, while none on placebo met that threshold. Agios also reported success on several secondary measures, and a safety profile “generally consistent” with previous results, though it didn’t provide specifics.

• Mitapivat could be the first approved treatment for PKD and is a critical drug for Agios. The company earned notoriety for quickly developing two cancer medicines, but both drugs aren’t big sellers and Agios hasn’t become profitable yet. Mitapivat, which is being studied in multiple rare blood diseases, could change the company’s trajectory if it’s successful.

Dive Insight:

Agios is one of the more unusual stories in biotech. Founded in 2008, it quickly formed a wide-ranging alliance with Celgene before having any drugs even close to human testing. That partnership helped fuel a 2013 initial stock offering and a fast development track for two leukemia drugs, Idhifa and Tibsovo. Shares climbed as high as $132 apiece in early 2015 as the two programs progressed, and both were approved for genetic forms of acute myeloid leukemia a few years later.

Yet Agios hasn’t been able to turn that success into consistent profitability. Last quarter the company lost nearly $100 million on roughly $35 million in revenue, largely from Tibsovo. Until a recent deal with Royalty Pharma, Agios drew royalties from Idhifa, which is now owned by Bristol Myers Squibb following its acquisition of Celgene. Shares entered Monday at about $46 apiece, roughly right where they were six years ago.

Agios could soon generate more revenue for Tibsovo, thanks to a recent positive result in a type of bile duct cancer. But the company’s most important growth driver is mitapivat. Unlike Idhifa and Tibsovo, the latter of which was once owned by Celgene, mitapivat has been wholly owned by Agios from the start.

The drug, a pill meant to boost patients’ levels of oxygen-carrying hemoglobin, was once only being tested for PKD, a rare condition in which patients are lacking a key enzyme that turns sugar into energy in red blood cells. But Agios has since begun tests in more common, chronic blood diseases like thalassemia and sickle cell disease. Each represent larger revenue opportunities, but also more competitive treatment landscapes.

The preliminary Phase 3 trial results announced Tuesday, then, are a stepping stone for Agios. Before they were disclosed, SVB Leerink analyst Andrew Berens noted a positive outcome “could validate the overall drug profile” of mitapivat and show it might be useful in treating other diseases.

The data come from a study of 80 patients with PKD who don’t require blood transfusions. Agios provided only top-line results; specifics will be disclosed at a medical meeting next year. But it said that mitapivat led to sustained increases in hemoglobin concentration at two or more assessments at 16, 20 and 24 weeks, for 40% of treated patients, versus none on placebo. Twenty-six of 52 patients (50%) in a single-arm Phase 2 trial responded to mitapivat, though the bar for success in that trial was lower than in the ACTIVATE study. The data were published in the New England Journal of Medicine last year.

The Phase 3 results are “largely consistent” with what was seen in previous testing, wrote Piper analyst Tyler Van Buren. “The bottom line is that there is no standard of care for PKD patients and mitapivat is likely to establish it,” he added.

A similar, second trial, in PKD patients who need regular transfusions, should produce results in early 2021. The company is also running some long-term safety studies: Agios scrapped a previous, similar type of molecule for PKD due to safety concerns. It hasn’t seen the same issues with mitapivat, however. The most common side effects in Phase 2 were headache, insomnia and nausea, and Agios said the Phase 3 safety results were similar to what it’s seen before.

Agios aims to file for approval of mitapivat in PKD next year, which is also when Phase 3 studies in sickle cell and thalassemia should begin. Agios disclosed initial results from a National institutes of Health-sponsored study in sickle cell in June, and more will be revealed at the American Society of Hematology’s virtual meeting next week.

Forma Therapeutics is developing a similar, rival drug for sickle cell that should also enter late-stage tests in 2021.

Agios shares climbed about 6% in early trading Tuesday.