Ionis Pharmaceuticals (IONS) investor relations material

Commercial performance and product launches

• INGREZZA continues to deliver strong, durable revenue with 2024 guidance of $2.7–$2.8 billion and LOE in 2038, maintaining growth nine years post-launch.

• CRENESSITY, approved in December 2024 for classic congenital adrenal hyperplasia, achieved over $150 million in Q1 sales and rapid patient uptake, with over 10% of patients on therapy within five quarters.

• VYKAT XR, acquired via Soleno Therapeutics, is expected to be a durable asset with $95 million in Q1 sales and strategic fit in the rare endocrine segment.

• The company’s commercial base is expanding, with Q1 aggregate sales from three products exceeding $900 million.

• Paltusotine and CRENESSITY are positioned as blockbusters, with peak sales potential in the $1.3–$1.4 billion range and strong adoption rates.

Pipeline and R&D outlook

• Nearly 20 clinical-stage pipeline programs are active, with lead phase III assets osavampator (major depressive disorder) and dereclidine (schizophrenia) expected to read out in 2027–2028.

• Early-stage CRF2 agonist for obesity is in the clinic, with phase I data expected in late 2027.

• The pipeline is set for a data-rich period starting in 2027, with multiple late-stage readouts anticipated.

• Internal next-generation molecules are being developed to maintain a high bar for efficacy, safety, and tolerability.

Market strategy and competitive positioning

• INGREZZA maintains a dominant position in tardive dyskinesia, with only 1 in 10 diagnosed patients on a VMAT2 inhibitor, indicating significant room for growth.

• Direct-to-consumer campaigns and increased HCP engagement are driving diagnosis and prescription rates.

• Competitive landscape for rare endocrine and psychiatric franchises is favorable, with high barriers for new entrants due to established efficacy and safety profiles.

• The company is leveraging synergies in rare endocrine sales and expects rapid path to peak sales for new launches compared to established products.