After selling off Tibsovo and entire cancer pipeline, Agios nets first approval for new 'anchor product'
Agios’ long-term plan is starting to come into focus, as the biotech won its first FDA approval since pivoting away from oncology Thursday.
The FDA approved Agios’ mitapivat in a rare blood disorder known as pyruvate kinase deficiency, which manifests as red blood cells being destroyed faster than they can be made. Agios won the OK in adult patients and touted mitapivat, to be branded as Pyrukynd, as the condition’s first approved disease-modifying therapy.
In a Friday morning conference call, Agios announced the average annual price (WAC) of the drug would be $334,880 and won’t be raised for five years.
Thursday’s approval marks a first step toward Agios making good on a promise that mitapivat will become the biotech’s “anchor product,” as CEO Jackie Fouse told Endpoints News in December 2020. It follows Agios’ decision to sell off its entire oncology portfolio, including the pioneering drug Tibsovo, to Servier for $1.8 billion cash upfront.
Soon after the deal closed, Agios began immediately buying up its outstanding shares, repurchasing a nearly $350 million stake from Bristol Myers Squibb last April — good for about 10%.
The pivot to mitapivat had been a risky proposition in the eyes of some analysts, as the PKR activator appeared to be less impressive than other competitors in the space. Notably, the drug’s ASH data from 2020 underwhelmed in sickle cell disease, a view reaffirmed in 2021, with a rival from Forma seemingly proving more efficacious (insert your favorite cross-trial comparison caveat here).
Sickle cell is likely to be the biggest selling indication for each of these candidates, though SVB Leerink noted Agios’ peak sales could hit $1.7 billion combined in SCD, pyruvate kinase deficiency and thalassemia. The two companies are taking different paths to the clinic in SCD, with Forma shooting for accelerated approval and Agios aiming at regular approval, according to a report from Evaluate Vantage.
Questions surrounding the pivot could remain until the rest of the SCD data play out, and Agios is currently studying mitapivat in a Phase II/III study for the condition. There are also two Phase III trials being conducted in thalassemia patients who are and are not regularly transfused, and two more Phase III studies in children with PK deficiency are expected to launch in mid-2022.
The approval was based on two Phase III studies looking at PK deficiency in adults who regularly receive blood transfusions and those who do not. Sixteen of 40 non-regularly transfused patients achieved a hemoglobin response, compared to zero in the placebo arm.
In addition, in those undergoing frequent transfusions, nine of 27 patients achieved at least a 33% reduction in transfusion burden during the 24-week treatment period. Figures were compared to personal historical data rather than placebo.
Due to a sourcing error, a previous version of this article incorrectly stated the number of patients enrolled and the number of responders in Agios’ PK deficiency studies. In non-regularly transfused patients, 16 of 40 patients saw a response, while in regularly transfused patients, nine of 27 patients achieved at least a 33% reduction during the treatment period, not six of 16 and three of nine, respectively.