Hepcidin becomes the Protagonist for a second biotech

In the space of 12 months hepcidin has provided a major catalyst for two small biotechs, though for the first this proved short-lived. Last week Protagonist Therapeutics nearly doubled in value after revealing proof-of-concept results with its hepcidin mimetic PTG-300, less than a year after La Jolla experienced a similar jump on a trial of its synthetic hepcidin LJPC-401. According to EvaluatePharma these are the only two industry projects using this mechanism of action. Protagonist’s data came in seven subjects with the myeloproliferative disorder polycythemia vera, six of whom became free of phlebotomy, a blood-removal procedure that is often the first treatment for the condition; the seventh missed a dose owing to the Covid-19 pandemic, and had to undergo a single phlebotomy. Protagonist has designated polycythemia vera the primary target use for PTG-300, with hereditary haemochromatosis, in which a phase II trial reads out next year, the second indication. Though a beta-thalassemia study will yield data shortly, Protagonist has discontinued development in this indication. It was hereditary haemochromatosis in which La Jolla’s LJPC-401 scored last year, but the group later said it was unconvinced by the dataset and would “reassess” LJPC-401; the stock’s earlier gains fell away.

The duelling hepcidin assets
  Protagonist La Jolla
  PTG-300 (hepcidin mimetic) LJPC-401 (synthetic hepcidin)
Polycythemia vera Topline ph2 (NCT04057040) data reported (Not in development)
Hereditary haemochromatosis Ph2 (NCT04202965) data in 2021 Topline ph2 (NCT03395704) data reported; development under review
Beta thalassaemia Ph2 (NCT04054921) publication Q2 2020; development discontinued Ph2 (NCT03381833) failed at interim analysis
Source: EvaluatePharma & clinicaltrials.gov.

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