Stargazer Pharmaceuticals Initiates a Phase 2a Clinical Trial for its Stargardt Disease Drug

Stargazer Pharmaceuticals, Inc., a Boston-based biopharmaceutical company developing treatments for rare eye diseases, has initiated a Phase 2a clinical trial of STG-001, an indirect visual cycle modulator, in Stargardt disease patients. The clinical study is evaluating two doses of STG-001 with regard to safety, the drug’s biodistribution in the body, and its mechanism of action. The company successfully completed a Phase 1 safety study of STG-001 in healthy volunteers.

Stargazer also announced it has completed Series A financing totaling $57 million from leading investors Novo Holdings, venBio Partners, Canaan Partners, and Pontifax. The Foundation Fighting Blindness also participated in the Series A financing via its venture arm, the Retinal Degeneration Fund (RD Fund), launched in 2018 with $72 million in total assets. The Foundation is also providing Stargazer with consultative support and access to resources for clinical development.

“We at Stargazer are grateful to the Foundation for helping identify potential patients and clinical trial sites for the STG-001 study,” says Gary Sternberg, MD, chief executive officer at Stargazer. “In addition, data gleaned from the Foundation’s ProgStar study, the largest natural history study ever conducted in Stargardt patients, is greatly informing our STG-001 clinical trial design.”

Stargardt disease is the leading form of inherited macular degeneration, affecting 30,000 people in the US and tens of thousands more around the world. There are no treatments for this condition. The hallmark of Stargardt disease is the accumulation of lipofuscin, containing harmful byproducts from vitamin A processing in the retina that lead to retinal cell degeneration and central vision loss over time. Though vitamin A is necessary for vision, its metabolites are toxic if not properly processed, which is what occurs in Stargardt disease.

STG-001 is designed to reduce blood concentrations of RBP4, a protein that delivers vitamin A to the retina. By reducing the uptake of vitamin A in the retina, researchers believe that STG-001 can potentially reduce the accumulation of retinal toxins for people with Stargardt disease and prevent retinal degeneration and subsequent vision loss.

“Stargazer is bringing a promising molecule and a strong clinical development plan to the STG-001 clinical trial,” says Rusty Kelley, PhD, MBA, senior vice president, investments & alliances at the Foundation Fighting Blindness and Stargazer board observer. “We are excited by the potential for STG-001 to address a critical unmet need by preserving vision for Stargardt disease patients.”

Read more in the press release here.