Spinal muscular atrophy is a neuromuscular disease that affects about 20,000 people in Europe and the United States, and is the leading cause of infant deaths.
The disease is characterized by muscle weakness which leads to severe physical disability and loss of life due to respiratory problems.
The drug, known as RG3039, which was licensed from the patient advocacy organization called Families of Spinal Muscular Atrophy in 2009, had been administered to 32 healthy volunteers in a blinded single dose trial.
RG3039 is the first clinical-stage drug to treat decreased levels of the survival motor neuron protein, a key protein necessary for normal neuromuscular function.
Repligen said the phase 1 study aimed to evaluate RG3039’s safety profile and pharmacokinetics, which is what happens to a drug once it enters the body.
Additionally, data revealed a dose-related drug response, resulting in 90 percent inhibition of the target enzyme.
It also noted that there were no adverse events reported. Repligen intends to present the data at the 64th Annual Meeting of the American Academy of Neurology.
"The safety and PK outcomes from our Phase 1 study are encouraging," chief executive Walter Herlihy said in a statement.
"We look forward to initiating the next steps for this drug candidate in alignment with guidance from the U.S. Food and Drug Administration."
The health regulator has already granted Orphan Drug and Fast Track designations to the medicine, as there is an unmet medical need that exists for patients with spinal muscular atrophy.
Orphan designation grants exclusive marketing rights for seven years in the U.S. and 10 years in Europe. Rare diseases are defined as those affecting fewer than 200,000 Americans.
Repligen, which is a biopharmaceutical company, has two central nervous system rare disease programs in phase 1 clinical trials.
Shares of the company traded higher by 11.29 percent, climbing to $7.08 apiece in trade on the Nasdaq Wednesday afternoon.