PTC Therapeutics (PTCT) investors are expected to remain focused on the Sephience rollout for phenylketonuria, with early indicators showing strong uptake and potential near-term consensus beats, as well as upcoming US Food and Drug Administration actions in Huntington's disease, RBC Capital Markets said in a note Wednesday.
The brokerage noted that the company's recent "R&D day" highlighted a broad slate of early-stage programs, including splicing modulators, ferroptosis, and inflammation, which could provide potential long-term strategic value.
RBC said most programs remain at the preclinical stage, with commercial or regulatory outcomes still uncertain, but PTC's growing experience in splicing modulators could provide a competitive advantage as these programs advance. Next-generation splicing modulators are expected to enter the clinic in late 2026, though regulatory pathways remain unclear.
The firm also highlighted early ferroptosis and DHODH programs, noting that preclinical data showed preliminary therapeutic signals, but translatability to humans remains uncertain.
RBC has a sector perform rating on PTC Therapeutics with a price target of $91.
Shares of PTC Therapeutics were down more than 6% in recent trading.
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