Disc Medicine, Inc. · NASDAQ:IRON
Disc Medicine — Corporate Update (November 2024)
16 slides from Disc Medicine's corporate update, Nov 4, 2024
Slide 1: Bitopertin End of Phase 2 Meeting Feedback
Bitopertin End of Phase 2 Meeting Feedback November 4, 2024 Exhibit 99.2
Slide 2: Disclaimer and FLS
Disclaimer and FLS This presentation contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, express or implied statements regarding Disc’s expectations with respect to its proposed APOLLO clinical trial of bito
Slide 3: Agenda
Agenda 01 Introduction and Summary John Quisel, J.D., PhD, Chief Executive Officer 02 Detailed Review of End of Phase 2 Feedback Steve Caffé, M.D., Chief Regulatory Officer Proposed APOLLO Study Parameters Will Savage, M.D., PhD, Chief Medical Officer 03 Closing Remarks John Quisel, J.D., PhD, Chief
Slide 4: Alignment with the FDA on all proposed study parameters
Alignment with the FDA on all proposed study parameters FDA acknowledged that EPP is a serious and potentially life-threatening disease with significant unmet medical need FDA agreed that average monthly time in sunlight without pain at the end of a 6-month treatment period can be used as a primary
Slide 5: EPP Phase 2 Development Program: BEACON, AURORA, and HELIOS Studies
EPP Phase 2 Development Program BEACON, AURORA, and HELIOS Studies Successful end of Phase 2 meeting with the FDA acknowledging the high unmet need in EPP and supporting our chosen trial parameters; clear development path to registration EPP and XLP; N = 26 (22 adults, 4 adolescents) Australia Open-
Slide 6: Summary of AURORA Results
Significant reductions in PPIX 40% reduction vs baseline Time-dependent, improvements in pain-free time in sunlight vs placebo 2x more light time vs baseline Significant 75% reduction in rate of phototoxic reactions vs placebo Phototoxic reaction-free in last 60 days Significant improvement in PGIC
Slide 7: Agenda
Agenda 01 Introduction and Summary John Quisel, J.D., PhD, Chief Executive Officer 02 Detailed Review of End of Phase 2 Feedback Steve Caffé, M.D., Chief Regulatory Officer Proposed APOLLO Study Parameters Will Savage, M.D., PhD, Chief Medical Officer 03 Closing Remarks John Quisel, J.D., PhD, Chief
Slide 8: Detailed FDA Feedback
Detailed FDA Feedback FDA agreed to all study parameters Disc requested prior to the meeting CONFIDENTIAL Primary Endpoint Average monthly total time in sunlight without pain between 10:00 and 18:00 after 6 months of treatment is clinically meaningful and can serve as a primary endpoint Additional
Slide 9: Accelerated Approval
Accelerated Approval CONFIDENTIAL FDA acknowledged PPIX reduction, as demonstrated in AURORA, may serve as surrogate endpoint to support accelerated approval An accelerated NDA package would include data from BEACON, AURORA, HELIOS, and a >4,000 participant safety database Will meet with FDA to fina
Slide 10: Agenda
Agenda 01 Introduction and Summary John Quisel, J.D., PhD, Chief Executive Officer 02 Detailed Review of End of Phase 2 Feedback Steve Caffé, M.D., Chief Regulatory Officer Proposed APOLLO Study Parameters Will Savage, M.D., PhD, Chief Medical Officer 03 Closing Remarks John Quisel, J.D., PhD, Chief
Slide 11: Primary Endpoint: Average Monthly Time in Light without Pain After 6 Months
Primary Endpoint: Average Monthly Time in Light without Pain After 6 Months of Treatment Robust Endpoint Longitudinal analysis leverages robust model that demonstrated significance in AURORA Accounts for time-dependent PPIX lowering effects with bitopertin and for waning of a placebo effect Endpoint
Slide 12: Proposed APOLLO Study Design
APOLLO Study Parameters N Size ~150 patients across sites in the US and Europe Trial Duration 6-month treatment period Trial Design Randomized 1:1, double-blind, placebo-controlled Trial Population EPP and XLP patients ages 12+, stratified by baseline light tolerance and geography Dose 60 mg Primary
Slide 13: Agenda
Agenda 01 Introduction and Summary John Quisel, J.D., PhD, Chief Executive Officer 02 Detailed Review of End of Phase 2 Feedback Steve Caffé, M.D., Chief Regulatory Officer Proposed APOLLO Study Parameters Will Savage, M.D., PhD, Chief Medical Officer 03 Closing Remarks John Quisel, J.D., PhD, Chief
Slide 14: Key Takeaways from Meeting
Key Takeaways from End of Phase 2 Meeting Endpoint Alignment Alignment with FDA on clinically meaningful endpoints Target Population Included Study can be conducted in adults and adolescents (ages 12+) with EPP and XLP Desired Dose Endorsed Endorsement of 60 mg dose for 6-month study Accelerated App
Slide 15: Next Steps and Upcoming Catalysts
ASN: Presented positive Phase 1 SAD data for DISC-0974 in CKD Additional data updates by EOY Bitopertin: Additional analyses from BEACON and AURORA DISC-0974: Complete Phase 1b data in MF anemia DISC-3405: Phase 1 HVOL MAD data Q4 2024 Catalysts Discussion of confirmatory trial design with FDA, with
Slide 16: Agenda
Agenda 01 Introduction and Summary John Quisel, J.D., PhD, Chief Executive Officer 02 Detailed Review of End of Phase 2 Feedback Steve Caffé, M.D., Chief Regulatory Officer Proposed APOLLO Study Parameters Will Savage, M.D., PhD, Chief Medical Officer 03 Closing Remarks John Quisel, J.D., PhD, Chief