Scholar Rock Receives $25M for Continued Research in Potential Therapies for Fibrotic Diseases

Scholar Rock Receives $25M for Continued Research in Potential Therapies for Fibrotic Diseases

Biopharmaceutical company Scholar Rock received $25 million from Gilead Sciences to continue the development of potential therapies to treat the progression of fibrotic diseases, including pulmonary fibrosis (PF).

This funding is a milestone payment, based on Scholar Rock’s preclinical studies in animals showing the effectiveness of therapeutic compounds that can block the activation of a factor called transforming growth factor beta (TGF-beta).

In patients with PF, TGF-beta is a key player in the abnormal wound-healing process that happens in the lungs. Once TGF-beta becomes activated, it causes the deposition of collagen in the lungs and the proliferation of fibroblasts — a type of cell that leads to the excessive buildup of extracellular matrix proteins. The combination of collagen deposition and accumulation of extracellular matrix proteins are key components for the development of fibrosis in the lungs.

The collaboration between Scholar Rock and Gilead started in December 2018, with Scholar Rock receiving an initial $80 million for its research. The goal of the collaboration is to identify and develop potent and highly specific inhibitors of TGF-beta for the treatment of fibrotic diseases.

With the successful completion of preclinical tests for Scholar Rock’s inhibitors of TGF-beta activation, Gilead has now awarded the company another $25 million.

“Achieving this important first milestone at the one year mark of the collaboration is indicative of the progress we are making together towards unlocking the potential of this novel approach to TGF-beta inhibition in the treatment of patients suffering from fibrotic diseases,” Nagesh Mahanthappa, PhD, president and CEO of Scholar Rock, said in a press release.

Scholar Rock is a company focused on the identification and development of therapies for the treatment of medical conditions in which protein growth factors have a key role, as is the case with TGF-beta in PF. 

At present, the company is developing three programs focused on the discovery and research of compounds able to block the activation of TGF-beta. Depending on the successful achievement of certain research, development, regulatory, and commercialization milestones, it may receive up to $1.425 billion in future funding from Gilead across its three TGF-beta-based programs. 

“This accomplishment further validates the broad applicability of our platform by targeting the activation of latent growth factors to overcome historic challenges with selectivity and to discover and develop safe and effective therapeutics to address a wide range of serious diseases, including neuromuscular disorders, cancers, fibrosis, and anemia,” Mahanthappa said.

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Patrícia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.
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