US-based biotechnology company Rallybio has reported results from a Phase I first-in-human clinical study of its drug candidate RLYB116.
A single dose of the drug at 100mg and 300mg resulted in exposure levels of more than 1µM and 3µM respectively, which led to a more than 99% reduction in free C5 concentrations.
RLYB116 is a potentially long-acting, subcutaneously injected C5 inhibitor that is under development to treat patients with complement-mediated diseases.
It has the potential to treat several complement dysregulation diseases.
The drug's subcutaneous administration at either a 100mg or 300mg dose was generally well-tolerated, causing mild to moderate adverse events and no serious adverse events.
Rallybio RLYB116 programme lead Eric Watsky said: “These single-dose data suggest that RLYB116 could be a highly innovative C5 inhibitor with the potential to address significant unmet medical need for patients.
“We remain on track to report initial data from the Phase I multiple ascending dose study of RLYB116 and disclosing our indication strategy in the fourth quarter of 2023.”
The company’s other clinical-stage programme is RLYB212, an anti-HPA-1a antibody to prevent foetal and neonatal alloimmune thrombocytopaenia (FNAIT).
Its pipeline also includes other programmes currently in clinical development.
Based in Connecticut, Rallybio is focused on the development and commercialisation of advanced therapies for patients with severe and rare diseases.
The company has a diverse portfolio of product candidates focused on addressing unmet medical needs in haematology, complement dysregulation, maternal foetal health and metabolic disorders.
Last month, it reported results from a Phase Ib study of RLYB212, in which the drug candidate produced a dose-dependent, rapid and complete elimination of transfused HPA-1a positive platelets in HPA-1a negative patients.
Both of the trial's two dose groups met the pre-specified proof-of-concept criteria of a 90% or greater reduction in mean platelet elimination half-life.
