Scholar Rock : ASCO Investor Call Presentation

Published: 2024-06-04 07:50:09 ET SRRK

Investor Call to Discuss Updated Results from SRK-181 Phase 1 DRAGON Study

June 4, 2024

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.

Welcome

Jay Backstrom, M.D., MPH

President and Chief Executive Officer

Company Speakers

Jay Backstrom, M.D., MPH

Jing Marantz, M.D., Ph.D.

President & Chief

Chief Medical Officer

Executive Officer

Expert Speaker

Toni Choueiri, M.D.

Director of the Lank Center for Genitourinary (GU) Oncology at Dana-Farber Cancer Institute (DFCI)

Today's Agenda

Topic

Speaker

 Welcome

Jay Backstrom, President & Chief Executive Officer

 SRK-181 Data Update

Jing Marantz, Chief Medical Officer

 Discussion of SRK-181 Data with

Jay Backstrom, President & Chief Executive Officer

Toni Choueiri, M.D.

Toni Choueiri, M.D., Dana-Farber Cancer Institute

Jay Backstrom, President & Chief Executive Officer

 Q&A

Ted Myles, Chief Operating Officer and Chief Financial Officer

Jing Marantz, Chief Medical Officer

Toni Choueiri, M.D., Dana-Farber Cancer Institute

 Closing Remarks

Jay Backstrom, President & Chief Executive Officer

Q&A -

SRK-181 Data Update

Jing Marantz, M.D., Ph.D.

Chief Medical Officer

Phase 1 study (DRAGON) of SRK-181 (linavonkibart), a latent TGFβ1 inhibitor, combined with pembrolizumab in anti-PD1 resistant patients with advanced solid tumors: Updated results of expansion phase

Ulka Vaishampayan1, Randy F. Sweis2, Deepak Kilari3, Ahmad Tarhini4, Justin F. Gainor5, Minal Barve6, Guru Sonpavde7, Meredith Mckean8, David Park9, Sunil Babu10, Yawen Ju11, Lan Liu11, Susan Henry11, Lu Gan11, Timothy A. Yap12

1University of Michigan, Ann Arbor, MI; 2University of Chicago, Chicago, IL; 3Medical College of Wisconsin, Milwaukee, WI; 4Moffitt Cancer Center Magnolia Campus, Tampa, FL; 5Massachusetts General Hospital Harvard Medical School, Boston, MA; 6Mary Crowley Cancer Research, Dallas, TX; 7AdventHealth Medical Group, Orlando, FL; 8Sarah Cannon Research Institute, Nashville, TN; 9St Jude Crosson Cancer Institute/Providence Medical Foundation, Fullerton, CA; 10Fort Wayne Medical Oncology and Hematology, Fort Wayne, IN; 11Scholar Rock, Inc., Cambridge, MA; 12The University of Texas MD Anderson Cancer Center, Houston, TX

Mechanism of Action

SRK-181, a Selective Anti-TGFβ1 Antibody, Overcomes CPIs Resistance

1.Batlle E, et al. Immunity. 2019; 50(4):924-940.

CPI, checkpoint inhibitor; GLP, good laboratory practice; MDSC, myeloid derived suppressor cells; TGFβ1, transforming growth factor beta-1.

Phase 1 Clinical Trial Overview

Dose Escalation (3+3)

Part A1: SRK-181 Single Agent

(80-3000 mg q3w/2000 mg q2w)

Dose Expansion

Part B: SRK-181 (1500mg q3w) + Pembrolizumab

n=up to 40/cohort

All advanced solid tumor

n=19

Part A2: SRK-181 + anti-PD-(L)1

(SRK-181:240-2400mg q3w)

Advanced solid tumor non-

responders to prior anti-PD-(L)1

n= 15

Key Eligibility Criteria

Cohort ccRCC

Cohort HNSCC

Cohort MEL

Cohort UC

Cohort NSCLC

Cohort Any Other*

Study Endpoints

Primary:

Secondary:

Exploratory:

*Cohort Any Other was terminated early and HNSCC was added.

ADA, anti drug antibody; BOR, best overall response; ccRCC, clear cell renal cell carcinoma; DCR, disease control rate; DoR, duration of response; ECOG, eastern cooperative oncology group; HNSCC, head and neck squamous cell carcinoma; MEL, melanoma; NSCLC, non-small cell lung cancer; ORR, objective response rate; OS, overall survival; PD, progressive disease; PD-1, programmed cell death protein 1; PD-L1, programmed cell death ligand 1; PD-(L)1, PD- 1/PD-L1; PFS, progression-free survival; PK, Pharmacokinetic; q2w, every 2 weeks; q3w, every 3 weeks; RECIST, response evaluation criteria in solid tumors; UC, urothelial carcinoma.

Disclaimer

Scholar Rock Holding Corporation published this content on 04 June 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 04 June 2024 11:49:01 UTC.