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Scholar Rock Announces Option Exercised by Janssen in Immuno-Oncology Collaboration Focused on Supracellular Activation
Company generates unique monoclonal antibodies that selectively inhibit the supracellular activation of latent TGF-beta 1 in the immune system

CAMBRIDGE, Mass., January 7, 2016 – Scholar Rock, a biotechnology company focused on discovering and developing drugs that selectively target supracellular activation of growth factors in the disease microenvironment, announced today that Janssen Biotech, Inc. (Janssen) has exercised an option as part of the existing immuno-oncology modulator collaboration. Scholar Rock’s successful generation of antibodies that selectively inhibit the supracellular activation of the growth factor TGF-beta 1 in the immune system by targeting its supracellular activation represents a novel approach in immuno-oncology therapeutics, and triggered the decision.

Under the terms of the original collaboration agreement, facilitated by Johnson & Johnson Innovation and announced in January 2014, Scholar Rock granted Janssen options to obtain worldwide exclusive licenses to pursue development and commercialization of supracellular activation targeted therapeutics intended to selectively modulate TGF-beta 1 activation in the immune system.

“Starting with a novel pharmacological hypothesis, within two short years we have been able to discover drug candidates that represent a unique immuno-oncology opportunity,” said Nagesh Mahanthappa, PhD, President and Chief Executive Officer of Scholar Rock. “This key milestone, taken together with our own nomination this past September of SRK-015 as our first proprietary development candidate for the treatment of muscle diseases, clearly demonstrates the power and productivity of our supracellular activation platform.”

“Our foundation of Scholar Rock stemmed from unique structural biology insights into how growth factors are activated or kept latent in vivo that suggested novel therapeutic modalities. Indeed, Scholar Rock’s preclinical data suggests that disease can be treated earlier than previously thought possible, at the step before harmful growth factors are released. Moreover, we have now achieved this for two different growth factor programs, one proprietary and the other in our collaboration with Janssen,” said Timothy A. Springer, PhD, co-founder of Scholar Rock and Latham Family Professor at Harvard Medical School and Professor of Medicine at Children’s Hospital Boston. “Scholar Rock’s innovative supracellular activation platform is well on its way toward generating an entirely new class of therapeutics with novel mechanisms of action.”

The research collaboration between Scholar Rock and Janssen has been conceived to focus on the discovery and development of novel antibody therapeutics that modulate supracellular activation, and thereby selectively inhibit TGF-beta 1 in the immune system for immuno-oncology applications, or selectively activate TGF-beta 1 in the immune system for the treatment of autoimmune diseases. The collaboration between Scholar Rock and Janssen to identify supracellular activation targeted therapeutics for the treatment of autoimmune diseases is continuing.

About Supracellular Activation
Scholar Rock was founded on unique insights into the mechanisms of supracellular activation of growth factors, a newly elucidated mechanism by which nature regulates growth factor activation in complex disease microenvironments. Through modulation of supracellular activation, highly specific and localized efficacy at the site of disease can be achieved, overcoming the challenges that have limited the traditional approach of inhibiting growth factors systemically. Scholar Rock is the first company to exploit this mechanism to discover and develop a pipeline of potential best-in-class therapeutic candidates.

About Scholar Rock
Scholar Rock is a biotechnology company focused on discovering and developing a novel class of biologic therapies that target supracellular activation of growth factors in the disease microenvironment. The Company’s initial proprietary and partnered drug discovery programs target specific growth factors which are present in the microenvironments of significant diseases, including fibrosis, musculoskeletal diseases, immuno-oncology and autoimmune diseases. Scholar Rock was founded upon discoveries made by its scientific founders, Professors Timothy Springer, PhD, and Leonard Zon, MD, of Boston Children's Hospital and Harvard Medical School, related to the molecular mechanisms of supracellular activation. The company is backed by leading investors, including Polaris Partners, Timothy Springer, ARCH Venture Partners, Fidelity Investments, EcoR1 Capital, The Kraft Group, and Cormorant Asset Management.

Media Contact:
Kathryn Morris
The Yates Network
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kathryn@theyatesnetwork.com

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Stern Investor Relations, Inc.
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stephanie@sternir.com