- Inozyme Pharma (NASDAQ:INZY) traded higher on Tuesday after Jefferies assumed coverage with a buy rating and a $17 price target, citing three rare disease opportunities for the company’s flagship asset, INZ-701.
- Enzyme replacement therapy is currently undergoing clinical trials. Studies on ENPP1 deficiency, calcification, and pseudoexfoliative ectodermal tumor (PXE), a trio of rare disorders caused by deficiency of inorganic pyrophosphate (PPi), a regulator of cell function.
- INZ-701, which is designed to boost PPi, could generate $2.5 billion in peak revenue, said Jefferies analyst Murray Rycroft. The experimental treatment could boost PPi to normal levels in these disease areas, the analyst said.
- Additionally, Rycroft highlighted upcoming catalysts for INZ-701, including a Phase 1 readout in calcifications and a top-line readout from a pivotal pediatric trial for ENPP1 deficiency scheduled for Q4 and H2 2025, respectively.
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