Re the discussion on Breakthrough Therapy Designation (BTD):
A study of drug candidates awarded BTD found, on average, that data was presented for 222 patients. Phase 2 data for NNZ-2591in PMS is limited to just 18 patients.
There is precedent for BTD designation based on data in a very small patient population - bimagrumab for the treatment of sporadic Inclusion Body Myositis (sIBM). The supporting clinical evidence came from a Phase 2a study in just 14 patients. However, this was a 24 week randomized, placebo-controlled, double-blind, parallel arm study. The Phase 2 study of NNZ-2591 in PMS was open-label and of 13 weeks duration.
As an aside, unfortunately for drug sponsor Novartis, bimagrumab failed to succeed in its subsequent Phase 2b/3 trial in siBM. Novartis sold bimagrumab in 2021 for just US$70m to a US start-up, Versanis. Versanis repurposed the drug for weight loss and while the drug was still in Phase 2, Versanis was acquired by Eli Lilly in 2023 for US$1.93bn.
Breakthrough therapy designation applies to a drug in a specific indication. Hence, supporting efficacy clinical data for any BTD application for NNZ-2591 in PMS would be restricted to that specific indication. The flip side of the coin though is that it is possible to apply for, and be granted, multiple BTDs for multiple indications of the one drug.
Priority Review Vouchers are unrelated to Breakthrough Therapy Designation and it is only possible to receive one per drug.
Nusinersen (Spinraza) did not receive BTD from the FDA. An application was made but was denied on 9/11/14.
Reata applied for BTD for omoveloxolone (SkyClarys) but was denied on 15/02/18. But it didn’t stop Biogen acquiring Reata for SkyClarys last year for US$7.3bn!
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