Erythropoietic Protoporphyria (EPP) and Bitopertin
April 25, 2023
Disclaimer and FLS
This presentation includes "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These forward-looking statements include express or implied statements relating to Disc's management team's expectations, hopes, beliefs, intentions or strategies regarding the future. In addition, any statements that refer to projections, forecasts or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. The words "anticipate," "believe," "contemplate," "continue," "could," "estimate," "expect," "intends," "may," "might," "plan," "possible," "potential," "predict," "project," "should," "will," "would" and similar expressions may identify forward- looking statements, but the absence of these words does not mean that a statement is not forward-looking.Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on Disc's current beliefs, expectations and assumptions regarding the future of Disc's business, future plans and strategies, clinical results and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements.
Disc may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and investors should not place undue reliance on these forward-looking statements. These forward-looking statements involve a number of risks, uncertainties (some of which are beyond Disc's control) or other assumptions that may cause actual results or performance to be materially different from those expressed or implied by these forward-looking statements. These risks and uncertainties include, but are not limited to: the adequacy of Disc's capital to support its future operations and its ability to successfully initiate and complete clinical trials; the nature, strategy and focus of Disc; the difficulty in predicting the time and cost of development of Disc's product candidates; Disc's plans to research, develop and commercialize its current and future product candidates; the timing of initiation of Disc's planned preclinical studies and clinical trials; the timing of the availability of data from Disc's clinical trials; Disc's ability to identify additional product candidates with significant commercial potential and to expand its pipeline in hematological diseases; the timing and anticipated results of Disc's preclinical studies and clinical trials and the risk that the results of Disc's preclinical studies and clinical trials may not be predictive of future results in connection with future studies or clinical trials and may not support further development and marketing approval; the other risks and uncertainties described in the "Risk Factors" section of the Annual Report on Form 10-K filed with the SEC on March 31, 2023, and other documents filed by Disc from time to time with the SEC, as well as discussions of potential risks, uncertainties, and other important factors in Disc's subsequent filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. None of Disc, nor its affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forwardlooking statement, whether as result of new information, future events or otherwise, except as required by law.
Confidential
Agenda
01 | Welcome and Introduction |
John Quisel, J.D., PhD, CEO | |
02 | EPP Disease Background and Pathophysiology |
Bruce Wang, M.D., Professor of Gastroenterology, University of California San Francisco | |
03 | EPP Patient Experience and Unmet Need |
Jean-Charles Deybach, M.D., PhD, Professor of Medicine, Paris Diderot University | |
04 | Bitopertin Overview and Development Plan |
Will Savage, M.D., CMO | |
05 Q&A Session
Confidential
Disc is Building a Leading Company Dedicated to Treating Hematologic Diseases
Focus on
Hematologic
Disorders
Immense medical need
across a wide spectrum of
disorders
Predictive, objective
endpoints
Fundamental
- Validated Pathways
Fundamental to red blood cell biology: iron and heme
Clinical and genetic evidence of target mechanism in humans
Multiple
Clinical
Programs with Broad Potential
Bitopertin in Phase 2
DISC-0974 in Phase 1b/2
MWTX-003 is Phase 1-
Ready
Multiple
Near-Term
Catalysts
Data expected 2023:
Bitopertin in EPP
DISC-0974 in MF and NDD-
CKD
Initiate Ph 1 MWTX-003
EPP (Erythropoietic Protoporphyria); XLP (X-linked Protoporphyria); MF (myelofibrosis); NDD (non-dialysis dependent);CKD (chronic kidney disease)
Confidential
Targeting Fundamental Pathways that Impact the Biology of Red Blood Cells
Iron and heme | Critical points of |
formation play a | intervention across |
central role in | multiple hematologic |
erythropoiesis | diseases |
Wide Spectrum of Hematologic Diseases Addressable by Disc Portfolio
Severe Rare (000s) | Moderate Prevalence (100K+) | Widely Prevalent (MMs) | |||||||
Diamond-Blackfan | Erythropoietic | Beta- | Anemia of | Myelodysplastic | Sickle Cell | Polycythemia | Hereditary | IBD | CKD |
Anemia | Porphyrias | Thalassemia | Myelofibrosis | Syndromes | Disease | Vera | Hemochromatosis | Anemia | Anemia |
Confidential
Attachments
- Original Link
- Original Document
- Permalink
Disclaimer
Disc Medicine Inc. i published this content on 25 April 2023 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 25 April 2023 18:57:07 UTC.