Risk Factors Update Summary

• RM-718 now forms a drug-PL co-precipitate, demonstrating significant weight reduction (-12.9%) in obese rats.
• Health Canada approved IMCIVREE for weight management in patients with obesity due to BBS or genetic deficiencies, expanding market access to 14 countries.
• Added material weakness in internal controls over financial reporting, potentially impacting financial statements.
• Addition of a Medicare drug price negotiation program subject to legal challenges.
• RM-718 was well tolerated in rats and monkeys up to 30 mg/kg, with no systemic toxicity.
• Revenue from product sales increased significantly to approximately $97.0 million from no significant revenue.
• The company raised approximately $791 million through underwritten offerings, increasing net proceeds from $742 million.
• IMCIVREE approved for chronic weight management in patients with monogenic or syndromic obesity.
• Increase in MDRP rebate liability cap from 100% to 100% of AMP.
• IMCIVREE is being developed globally, with approvals in the US, EU, Canada, and other regions.
• Expanded market access for IMCIVREE to 14 countries for BBS or POMC and LEPR deficiencies.
• Acquisition of Xinvento for congenital hyperinsulinism (CHI) aims to develop treatments and initiate clinical development in 2024.
• Introduction of new EU-US Data Privacy Framework with implications for data transfers.
• The accumulated deficit increased from $710 million to $894 million, impacting financial position and long-term profitability.
• Licensing agreements with LG Chem for LB54640 include Phase 2 studies and royalty agreements.
• Health Canada approved IMCIVREE for weight management in pediatric patients, expanding the target age group to 6 years and older.
• Changes in UK regulations post-Brexit may impact medicinal product approvals and market access.
• Genetic sequencing efforts expanded to almost 80,000 individuals, supporting research on rare genetic diseases.
• Initiated Phase 3 trial for RM-718, a more selective MC4R agonist, with plans for Phase 1 trials in 2024.
• The company's net losses increased from $69.6 million to $181.1 million, affecting financial performance for the years ended December 31, 2021 and 2022.
• Identification of a material weakness in internal controls over financial reporting.
• Increased DNA database samples to almost 80,000, aiming to improve understanding of rare genetic diseases.
• The company continues to collaborate with authorities to achieve market access in additional countries for IMCIVREE, indicating a focus on global expansion.
• Increased focus on environmental sustainability initiatives may lead to higher costs.
• Discontinued development of weekly setmelanotide formulation in favor of advancing RM-718.
• The company may need to raise additional funds to pursue additional indications and commercialize setmelanotide, impacting financial stability and growth potential.
• Potential risks from short sellers manipulating the market price of common stock.
• Advanced LB54640, an oral MC4R agonist, with positive efficacy and safety results in overweight adults.