Harmony Biosciences (HRMY) investor relations material

Leadership and company overview

• Leadership team has extensive experience in both large pharma and biotech, emphasizing a preference for the dynamic biotech environment.

• Company launched in 2017, focusing on growth through commercial success and pipeline expansion.

• Pipeline includes late-stage, catalyst-rich assets in orphan and rare neurological disorders.

Pipeline and clinical development

• Three main franchises: sleep/wake (Wakix and next-gen pitolisant), rare epilepsy (EPX-100 in phase 3 for Dravet and Lennox-Gastaut), and neurobehavioral (ZYN002 for Fragile X, phase 3 data imminent).

• Pipeline includes eight assets across 13 programs, with up to six in phase 3 by year-end; each franchise targets $1–2 billion peak sales.

• ZYN002 is a synthetic, transdermal gel targeting the endocannabinoid system for Fragile X; phase 3 Reconnect trial top-line data expected soon.

• Phase 3 Reconnect trial focuses on patients with complete methylation of the FMR1 gene, aiming for a clinically meaningful and statistically significant improvement in social avoidance.

• Positive data could lead to the first approved therapy for Fragile X and support expansion into related indications like 22q deletion syndrome.

Regulatory and market strategy

• Consistent, positive interactions with the FDA and EMA, with global rights to ZYN002.

• Plans to file for approval in both the US and Europe if phase 3 data are positive.

• Market opportunity for Fragile X estimated at 80,000 US patients, with 60–70% fully methylated.