Rallybio announces positive phase 1 results for drug treating PNH and gMG

muscle weakness

Affibody’s licensee Rallybio Corporation has announced positive topline results from a phase 1 single ascending dose (SAD) study in healthy participants of RLYB116.

RLYB116 is a novel, potentially long-acting, subcutaneously administered inhibitor of complement factor 5, or C5, in development for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) and generalized myasthenia gravis (gMG). 

RLYB116 is an Affibody molecule that has the potential to drive the rapid, complete, and sustained inhibition of C5 with a subcutaneous injection. The molecule was initially developed by Affibody under a collaboration with Sobi.

Study details

In the ongoing study, all participants that were administered a single 1 mL subcutaneous injection of 100mg of RLYB116 demonstrated a reduction in free C5 greater than 99% within 24 hours of dosing. 

The terminal elimination half-life of RLYB116 was greater than 300 hours. Subcutaneously administered RLYB116 was observed to be generally well-tolerated at the 100mg dose, with mild or moderate adverse events and no drug-related serious adverse events reported. 

The ongoing dose escalation study was initiated in February 2022 and is designed to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of single subcutaneous dose RLYB116 in healthy participants and includes five dose cohorts (2, 10, 30, 100, 300 mg RLYB116) with each enrolling eight participants. Post-treatment/study follow-up is expected to continue for 10 weeks.

PNH and gMG

Paroxysmal nocturnal hemoglobinuria is a rare, acquired, life-threatening disease of the blood. The disease is characterized by destruction of red blood cells, blood clots, and impaired bone marrow function. It is closely related to aplastic anemia.

Myasthenia gravis is characterized by weakness and rapid fatigue of any muscle under voluntary control. It is caused by a breakdown in the communication between nerves and muscles.

While the disease can affect people of any age, it is more common in women younger than 40 and in men older than 60.

About RallyBio

Rallybio is a clinical-stage biotechnology company identifying and accelerating the development of therapies for patients with severe and rare diseases. Since its launch in January 2018, it has built a portfolio of promising product candidates, which are now in development to address rare diseases in the areas of hematology, immuno-inflammation, maternal fetal health, and metabolic disorders. 

Rallybio is headquartered in New Haven, Connecticut, with an additional facility at the University of Connecticut’s Technology Incubation Program in Farmington, Connecticut. 

About Affibody

Affibody is a Swedish clinical stage biopharmaceutical company with a broad product pipeline focused on developing innovative bi- and multi-specific next generation biopharmaceuticals based on its technology platforms: Affibody molecules and Albumod.

Affibody AB is a holding of Patricia Industries.

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