Gilead and Scholar Rock target fibrosis

The partners will focus on the potential of TGFβ inhibitors

Kristen Smith
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FOSTER CITY, Calif.—Gilead Sciences Inc., a research-based biopharmaceutical company that specializes in therapies focused on unmet medical need, and Scholar Rock Holding Corp., focused on finding treatments for serious diseases in which signaling by protein growth factors plays a fundamental role, have formed a new strategic partnership. The collaboration focuses on the discovery and development of highly specific inhibitors of transforming growth factor beta (TGFβ) activation for the treatment of fibrotic diseases.
Per this agreement, Scholar Rock is responsible for antibody discovery and preclinical research through product candidate nomination, after which, upon exercising the option for a program, Gilead will be responsible for the program’s preclinical and clinical development and commercialization.
Gilead is committed to developing innovative therapies that address a range of fibrotic diseases, and under the terms of the collaboration, it has the exclusive option to license worldwide rights to product candidates from three of Scholar Rock’s specific TGFβ programs: inhibitors that target activation of latent TGFβ1 with high affinity and specificity, inhibitors that selectively target latent TGFβ1 localized to connective tissue, and a third undisclosed TGFβ discovery program—all of which represent unique approaches to targeting pro-fibrotic signaling.
“Gilead is committed to developing innovative therapies that address a range of fibrotic diseases, including non-alcoholic steatohepatitis and diabetic kidney disease,” said Dr. John McHutchison, chief scientific officer and head of Research and Development at Gilead Sciences. “We are excited to work with Scholar Rock to investigate this novel approach to TGFβ inhibition as an important aspect of our research programs in fibrotic diseases.”
Fibrosis refers to the pathological accumulation of extracellular matrix (ECM) proteins in the liver, lungs, kidneys and a number of other tissues, which results in scarring and thickening of the affected tissue. It is essentially an exaggerated wound healing response which interferes with normal organ function. TGFβ1 in the connective tissue microenvironment has been firmly established as a central driver in the manifestation of fibrosis. Fibrotic disorders afflict over 40 million people, and are estimated to be responsible for up to 50 percent of all deaths in the United States.
This collaboration capitalizes on Scholar Rock’s unique ability to locate the precursor form of growth factors and then develop monoclonal antibodies that locally and selectively target these signaling proteins at the cellular level, resulting in potent, durable and highly selective options for suppressing pro-fibrotic signaling in multiple organs. They have found that selectively inhibiting the activity of TGFβ1 at the source of disease in the tissue microenvironment by blocking supracellular activation can treat fibrosis while avoiding known toxicities caused by less selective inhibition of signaling mediated by a broader set of TGFβ superfamily members.
“We are very excited about this strategic collaboration, which draws on Gilead’s expertise and commitment to fibrosis and Scholar Rock’s highly selective approach to inhibiting the activation of TGFβ to develop novel therapies for the treatment of fibrotic diseases,” Dr. Nagesh Mahanthappa, president and CEO of Scholar Rock, commented in a press release. “This collaboration allows Scholar Rock to maximize the value of candidates from our TGFβ program and also emphasizes the tremendous potential of our broad pipeline of highly specific growth factors modulators, which we are developing for the treatment of a wide range of serious diseases, including neuromuscular disorders, cancer and anemia.”
The partnership allows Scholar Rock to maximize the value of their proprietary platform, by bringing in $80 million in upfront payments ($50 million cash and $30 million in equity), $25 million in a preclinical milestone, and an additional $1.425 billion in clinical, regulatory and commercial milestones. Scholar Rock is also eligible for high single- to low double-digit royalties on future sales. Importantly, the partnership is structured so that Scholar Rock retains exclusive worldwide rights to discover, develop and commercialize certain TGFβ inhibitors for oncology and cancer immunotherapy. 
“Gilead’s commitment to developing innovative therapies for fibrotic diseases makes the company an ideal partner to maximize the value of candidates from our TGFβ program,” noted Mahanthappa. “This collaboration also emphasizes our belief in the tremendous potential of Scholar Rock’s broad pipeline of highly specific modulators targeting the TGFβ superfamily, with potential applications in a wide range of serious diseases, including neuromuscular disorders, cancer, fibrosis and anemia. We believe that our platform has broad applicability and partnerships, such as this one with Gilead, are a logical way to maximize the value of our platform as we continue to build our pipeline.”

Kristen Smith

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