BioAge Labs (BIOA) investor relations material

Key clinical data and program updates

• Announced first clinical results for a brain-penetrant NLRP3 inhibitor, showing target coverage in brain and periphery at 60 mg and complete IL-1 beta shutoff at 120 mg, with good safety and tolerability.

• Initial phase 1 included SAD and two MAD cohorts in healthy volunteers, with 60 mg and 120 mg doses; 120 mg achieved full IL-1 beta suppression.

• Three individuals with elevated CRP in MAD cohorts saw an average 79.5% CRP reduction.

• Two new MAD cohorts in obese patients with high CRP will read out in the first half of next year, followed by a 12-week study starting mid-next year.

• No dose-limiting toxicity observed; safety margins over 40-fold at 60 mg, with only mild to moderate AEs.

Strategic focus and indication prioritization

• Next proof-of-concept (POC) study will focus on CRP reduction in obese individuals with cardiovascular risk, not obesity as a primary endpoint.

• Indication selection will target areas where the company can advance alone through phase 3, leveraging unique brain and retina penetration.

• Additional indications, including neurodegenerative and ocular diseases, are under consideration, with plans to announce next year.

• Differentiation from competitors includes a novel NLRP3 binding site, best-in-class potency, and brain penetration.

• Portfolio includes a follow-on brain-penetrant compound and potent peripherally restricted NLRP3 inhibitors.

Financial and operational outlook

• Cash balance of ~$300 million and runway of about three years, covering planned studies and APJ programs through phase 1.

• Strategy includes advancing some indications independently and partnering for larger indications like ASCVD and Alzheimer's.

• Discovery platform leveraged DNA-encoded library screening and structural biology collaborations, resulting in novel compounds.