FDA approves BB Biotech backed Agios’ AML drug Tibsovo
Agios Pharmaceuticals (NASDAQ:AGIO), a top 10 holding for Switzerland’s BB Biotech (BBB), has received US FDA approval for Tibsovo for relapsed or refractory acute myeloid leukaemia (r/r AML) with a specific IDH1 mutation. Agios, which has seen its shares rise by ~50% this year, accounted for around 5.6% of BB Biotech’s NAV, based on data at 30 June. Agios is also held by the UK’s International Biotechnology Trust (IBT).
The approval was based on data from a 174-patient Phase I/II study, which showed 32.8% rate of complete remission or complete remission with partial haematologic recovery and a median duration of responses of 8.2 months. Additionally, 37.3% of patients who started the trial dependent on red blood cell or platelet transfusions became independent during any 56-day post-baseline period.
Consensus estimates suggest Tibsovo could achieve sales approaching $850m/year by 2024. The drug is wholly owned by Agios, with rights in China, Hong Kong, Macau and Taiwan licensed to CStone Pharmaceuticals. Agios intends to commercialise the product itself in the US and potentially other territories and plans to submit a marketing authorisation application in Europe in the fourth quarter. The drug will be priced in the US at $26,115/month.
Tibsovo is the first product to be approved for AML patients with the IDH1 mutation, which occurs in 6-10% of cases, but the second drug invented by Agios to obtain approval. Almost one year ago, the Agios-derived Idhifa was approved for r/r AML with IDH2 mutation, which occurs in 8-19% of cases. Agios licensed global rights to Idhifa to Celgene in 2010, but retained those to the compound that is now Tibsovo. Agios, in collaboration with Celgene, now intends to initiate a registration-enabling Phase III trial codenamed HO150 where Tibsovo or Idhifa will be given with standard induction/consolidation chemotherapy for frontline AML patients with an IDH1 or IDH2 mutation in the fourth quarter.