Investor Day
May 22, 2024
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© 2024 Scholar Rock, Inc. All rights reserved.
Welcome
Rushmie Nofsinger
Vice President
Investor Relations & Corporate Affairs
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© 2024 Scholar Rock, Inc. All rights reserved.
Forward-Looking Statements
Various statements in this presentation concerning the future expectations, plans and prospects of Scholar Rock Holding Corporation and Scholar Rock, Inc. (collectively, "Scholar Rock"), including without limitation, Scholar Rock's expectations regarding its strategy, its product candidate selection and development timing, including timing for the initiation of and reporting results from its preclinical studies and clinical trials for SRK-439, apitegromab, SRK-181, and other product candidates and indication selection and development timing, its cash runway, the ability of any product candidate to perform in humans in a manner consistent with earlier nonclinical, preclinical or clinical trial data, and the potential of its product candidates and proprietary platform. The use of words such as "may," "could," "might," "will," "should," "expect," "plan," "anticipate," "believe," "estimate," "project," "intend," "future," "potential," or "continue," and other similar expressions are intended to identify such forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, without limitation, that preclinical and clinical data, including the results from the Phase 2 trial of apitegromab or Part A or Part B of the Phase 1 trial of SRK-181, are not predictive of, may be inconsistent with, or more favorable than, data generated from future or ongoing clinical trials of the same product candidate, including the Phase 3 clinical trial of apitegromab in SMA and Part B of the Phase 1 clinical trial of SRK-181, respectively, Scholar Rock's ability to provide the financial support, resources and expertise necessary to identify and develop product candidates on the expected timeline, the data generated from Scholar Rock's nonclinical and preclinical studies and clinical trials, information provided or decisions made by regulatory authorities, competition from third parties that are developing products for similar uses, Scholar Rock's ability to obtain, maintain and protect its intellectual property, the success of Scholar Rock's current and potential future collaborations, Scholar Rock's dependence on third parties for development and manufacture of product candidates including, without limitation, to supply any clinical trials, Scholar Rock's ability to manage expenses and to obtain additional funding when needed to support its business activities and establish and maintain strategic business alliances and new business initiatives, as well as those risks more fully discussed in the section entitled "Risk Factors" in Scholar Rock's Form 10-K for the year ended December 31, 2023, and Quarterly Report on Form 10-Q for the quarter ended March 31, 2024, as well as discussions of potential risks, uncertainties, and other important factors in Scholar Rock's subsequent filings with the Securities and Exchange Commission. Any forward-looking statements represent Scholar Rock's views only as of today and should not be relied upon as representing its views as of any subsequent date. All information in this press release is as of the date of the release, and Scholar Rock undertakes no duty to update this information unless required by law.
This presentation may also contain estimates and other statistical data made by independent parties and by us relating to market size and growth and other data about our industry. This data involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such estimates. In addition, projections, assumptions, and estimates of our future performance and the future performance of the markets in which we compete are necessarily subject to a high degree of uncertainty and risk.
Apitegromab and SRK-181 are investigational drug candidates under evaluation. Apitegromab, SRK-181, and SRK-439 have not been approved for any use by the FDA or any other regulatory agency and the safety and efficacy of apitegromab, SRK-181 and SRK-439 have not been established.
© 2024 Scholar Rock, Inc. All rights reserved.
Company Speakers
Jay Backstrom, M.D., MPH | Jing Marantz, M.D., Ph.D. | Tracey Sacco | Mo Qatanani, Ph.D. |
President & Chief | Chief Medical Officer | Chief Commercial Officer | Chief Scientific Officer |
Executive Officer |
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© 2024 Scholar Rock, Inc. All rights reserved.
Expert Speakers
Diana Castro, M.D. | Ania Jastreboff, M.D., Ph.D. |
Founder of Neurology & Neuromuscular Care Center | Associate Professor of Medicine (Endocrinology), Yale School |
and Neurology Rare Disease Center | of Medicine |
Former Associate Professor of Pediatrics, Neurology | Director, Yale Obesity Research Center (Y-Weight) |
and Neurotherapeutics, University of Texas | |
Southwestern, Director of the Neuromuscular Program | Co-Director, Yale Center for Weight Management |
& Fellowship and Director of the Pediatric Muscular | |
Dystrophy Association Clinic at Children's Health | Member of Board of Directors for the American Board of |
Obesity Medicine |
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© 2024 Scholar Rock, Inc. All rights reserved.
Today's Agenda
Topic | 8:30 - 12:00 | Speaker | ||
Welcome | Rushmie Nofsinger, VP of IR & Corporate Affairs | |||
Vision & Strategic Overview | Jay Backstrom, President & Chief Executive Officer | |||
SMA: The Patient Journey | Diana Castro, M.D., Neurology & Neuromuscular Care Center | |||
and Neurology Rare Disease Center | ||||
Apitegromab: Development Program | Jing Marantz, Chief Medical Officer | |||
Apitegromab: Commercial Readiness | Tracey Sacco, Chief Commercial Officer | |||
Q&A | ||||
10-minute Break | ||||
Obesity: Muscle Matters | Ania Jastreboff, M.D., Ph.D., Yale School of Medicine | |||
SRK-439: Differentiated Approach | Mo Qatanani, Chief Scientific Officer | |||
Cardiometabolic Development Program | Jing Marantz, Chief Medical Officer | |||
Q&A | ||||
Closing Remarks | Jay Backstrom, President & Chief Executive Officer | |||
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© 2024 Scholar Rock, Inc. All rights reserved.
Building a Fully
Integrated Biopharma
Company
Jay Backstrom
President & Chief Executive Officer
© 2024 Scholar Rock, Inc. All rights reserved.
We are a global leader in harnessing the life-changing potential of TGFβ biology
OUR MISSION
To discover, develop, and deliver life-changing therapies by harnessing cutting-edge science to create new possibilities for people living with serious diseases
TGFβ=Transforming growth factor-beta.
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WHAT YOU WILL HEAR TODAY
Advancing Our Journey Towards Commercialization
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Selectivity is the Key
The hallmark of our differentiated platform is unparalleled selectivity
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Large Unmet Needs
SMA and obesity represent high value markets offering significant potential revenue opportunities
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Positioned for Success
Experienced team executing on strategy and goals
Next 12 - 24 months of execution is expected to be transformative for our company
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© 2024 Scholar Rock, Inc. All rights reserved.
AGENDA
Evolving into a Fully Integrated Biopharma Company
Leveraging our proprietary platform
Building a muscle-targeted franchise
The road ahead - value drivers
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© 2024 Scholar Rock, Inc. All rights reserved.
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Disclaimer
Scholar Rock Holding Corporation published this content on 22 May 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 22 May 2024 16:20:04 UTC.