On Friday, Ionis Pharmaceuticals (NASDAQ:IONS) maintained its Buy rating and a price target of $60.00 from Needham, following the FDA's approval of TRYNGOLZA for the treatment of Familial Chylomicronemia Syndrome (FCS).
The approval, coinciding with the anticipated PDUFA date of December 19, 2024, was expected by the industry and did not present any unexpected conditions. The approved label for TRYNGOLZA, also known as olezarsen, reflects results from the BALANCE Phase 3 study, which demonstrated that 80 mg of the drug reduced triglycerides by 30.0% at the six-month mark, with a placebo-adjusted reduction of 42.5%.
Ionis Pharmaceuticals has set the annual Wholesale Acquisition Cost (WAC) for TRYNGOLZA at $595,000, a figure that aligns with projections for rare-disease drug pricing. Despite this, analysts at Needham anticipate a potential price reduction should the drug also receive approval for the broader Severe Hypertriglyceridemia (SHTG) indication. Results from the Phase 3 trials for the SHTG indication are expected in the second half of 2025.
The revenue forecast for TRYNGOLZA in 2025 stands at $27 million, which is slightly under the current consensus estimate of $28 million. This projection takes into account the current pricing strategy and the drug's status as a first-in-class treatment for FCS. The anticipation of future approval for the SHTG indication could significantly influence the drug's pricing and market impact.
The FDA's approval of TRYNGOLZA marks a significant milestone for Ionis Pharmaceuticals, as it brings a new treatment option to patients suffering from FCS, a rare and often debilitating condition. The drug's performance in the BALANCE Phase 3 study underpins the FDA's decision, offering clinical evidence of its efficacy in triglyceride reduction.
Needham's reiteration of the Buy rating suggests confidence in Ionis Pharmaceuticals' market position and the potential of TRYNGOLZA. With the pricing strategy set and the approval process moving forward, Ionis is poised to enter the rare-disease market with this newly approved therapy.
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