Risk Factors Update Summary

• Litigation or proceedings could substantially increase operating losses and reduce resources. This could have a substantial adverse effect on the price of common stock.
• Uncertainty regarding potential changes in orphan drug regulations by the FDA and EMA could impact business.
• Accumulated deficit rose from $439 million to $653 million, a substantial increase of $214 million.
• Expanded product pipeline to include treatments for Graves' disease and Thyroid Eye Disease, addressing unmet medical needs.
• Positive Phase 1 data for CRN04894 showed dose-dependent reductions in cortisol levels with no serious adverse events.
• Patent litigation could prevent marketing of patented products and technologies, impacting market competitiveness.
• Proposed European Commission changes may make it harder to obtain orphan designation, affecting exclusivity.
• The patent for compound CRN04777 extended from expiring between 2036-2040 to 2044.
• The company plans to seek orphan drug designation in the EU for CRN04777.
• Increased number of full-time employees from 210 to 290 may impact operations and growth.
• The company increased full-time employees from 210 to 290, a significant 38% rise.
• Potential vulnerability to a single UPC-based revocation challenge could invalidate patents in multiple countries.
• Clinical trials for paltusotine increased from one to two Phase 3 trials.
• Initiated Phase 2 study for CRN04894 in CAH patients, with initial data expected in the second quarter of 2024.
• The FDA published new guidance on informed consent, impacting recruitment and retention in trials.
• Federal NOL carryforwards increased from $254 million to $311 million, enhancing tax benefits.
• Investment in commercial infrastructure increased significantly, with ongoing financial commitments.
• Cybersecurity risks include a wide range of threats, such as cyberattacks, data loss, and technological malfunctions.
• Future acquisitions may require additional financing, impacting the realization of full benefits.
• Failure to comply with new laws and regulations, like SEC's proposed climate-related reporting rules, could harm business.