Scholar Rock : Corporate Presentation June 2025
SRRK
Published on 06/02/2025 at 09:16
Company Overview | June 2025
To discover, develop, and deliver life-changing therapies by harnessing cutting-edge science
to create new possibilities for people living with serious diseases
1 COMMERCIALIZE
Execute a Successful
Commercial Launch*
2 EXPAND
Apitegromab Development
Program: Building a Pipeline in a Product
3 ADVANCE
Highly Innovative Anti-
Myostatin Program
Building off
successful Phase 3 SAPPHIRE results
BLA accepted under priority review & MAA validated
$2B+ Opportunity in SMA
Initiate earlier treatment
with OPAL study for patients under 2 years of age with SMA
Pursuing opportunities
for apitegromab in additional rare neuromuscular diseases
EMBRAZE
Phase 2 study results
expected in 2Q 2025
IND submission on track for 3Q 2025 for SRK-439, a highly innovative myostatin inhibitor
Selectivity Drives Success
Latent Growth Factor
RIGHT TARGET
Validated Biology
RIGHT TIME
Latent Form
Targeting the 'cage' before growth factor is released allows for exquisite selectivity
"Mature" Active Growth Factor
Challenging to target because of high homology across super-family
Industry-leading Anti-myostatin Programs
Our Differentiated Approach: Target Latent Growth Factor
THERAPEUTIC AREA
PRODUCT
(target)
Discovery/
Preclinical PHASE 1 PHASE 2 PHASE 3 Commercial
NEUROMUSCULAR
Apitegromab
(latent myostatin)
SPINAL MUSCULAR ATR
OPHY
(undisclosed)
*Indication
CARDIOMETABOLIC
Apitegromab
(latent myostatin)
OBESITY
SRK-439
(novel latent myostatin)
OBESITY
IMMUNO-ONCOLOGY
Linavonkibart (SRK-181)
(latent TGFβ1)
UC, ccRCC**
HEMATOLOGY
SRK-256
(RGMc)
ANEMIA
FIBROSIS
SRK-373
(LTBP1/3)
Fibrotic indications
Disclaimer
Scholar Rock Holding Corporation published this content on June 02, 2025, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on June 02, 2025 at 13:15 UTC.