Stargazer Pharmaceuticals Raises $57 Million for Rare Eye Disease Therapies

Rare Daily Staff

Stargazer Pharmaceuticals completed a $57 million Series A financing to advance development of novel treatments for rare eye diseases.

Novo Holdings, VenBio Partners, Canaan Partners and Pontifax participated in the financing, which will be used to develop Stargazer’s STG-001 for treating Stargardt disease.

Stargardt disease is a rare, inherited retinal degenerative disease that leads to progressive central visual loss and legal blindness. It is the most common juvenile macular dystrophy and the leading cause of childhood blindness with an estimated prevalence of 50,000-80,000 patients in the United States and major European countries combined. It occurs in 1 in 8,000 to 1 in 10,000 people making it the most common juvenile macular dystrophy.

Typical onset of disease is generally between 10-20 years of age and there are no treatments currently available.

The visual cycle is fueled by uptake of vitamin A (retinol) from the circulation that is transported by retinol binding protein 4 (RBP4), a key protein that binds retinol in the systemic circulation and delivers it to the eye. STG-001 is an indirect visual cycle modulator. By reducing plasma concentrations of RBP4 and vitamin A, STG-001 modulates the visual cycle and the accumulation of cytotoxic retinoids in the eye, potentially reducing the rate of damage to the retina and preserving vision in Stargardt disease patients. STG-001 has received Orphan Drug designation from both the U.S. and European regulatory agencies.

Novo Ventures launched Stargazer in 2018, along with Stargazer CEO Gary Sternberg and Chief Operating Officer Silvia Ragno, following licensing of STG-001 from Takeda Pharmaceuticals.

Stargazer has successfully completed a phase 1 single- and multiple-ascending dose clinical study of STG-001 in healthy volunteers and is planning to initiate a pivotal efficacy study in 2021.

The series A financing enables Stargazer to advance development of STG-001 through a pivotal clinical efficacy study potentially leading to a new therapy for Stargardt disease. The company is collaborating with Foundation Fighting Blindness (FFB) for the development of STG-001. FFB also participated in the financing through its Retinal Degeneration Fund.

“This financing from leading investors, as well as the support from FFB, is a testament to the potential of our lead compound, STG-001. Initiation of the phase 2a trial in Stargardt disease patients is an important milestone for us and we look forward to further development of STG-001 for the benefit of Stargardt Disease patients,” said Sternberg, CEO of Stargazer Pharmaceuticals.