FDA Approves ASCENIV(formerly referred to as RI-002), A Novel Intravenous Immune Globulin

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About Primary Immunodeficiency Disease (PI)

PI is a class of inherited genetic disorders that causes an individual to have a deficient or absent immune system due to either a lack of necessary antibodies or a failure of these antibodies to function properly. PI patients are more vulnerable to infections and more likely to suffer complications from these infections. According to the World Health Organization, there are over 250 different presentations of PI. As patients suffering from PI lack a properly functioning immune system, they typically receive monthly, outpatient infusions of IVIG therapy. Without this exogenous antibody immune support, these patients would be susceptible to a wide variety of infectious diseases. PI has an estimated prevalence of 1:1,200 in the United States, or approximately 250,000 people.

Rationale for the potential Evaluation in RSV-Infected Patients

RSV is a common virus that ordinarily leads to mild, cold-like symptoms in healthy adults and children. In high-risk groups, such as the PI population and the other immunocompromised populations, RSV can lead to a more serious infection and may even cause death.

Additional Research Findings

We previously conducted a randomized, double-blind, placebo-controlled Phase II clinical trial to evaluate RI-001, RI-002’s predecessor product candidate, in immunocompromised, RSV-infected patients. This trial was conducted with 21 patients in the United States, Canada, Australia, and New Zealand. The Phase II dose-ranging trial demonstrated a statistically significant improvement in the change from baseline RSV titers to day 18 in the high-dose and low-dose treatment groups when compared with placebo (P=0.0043 and P=0.0268, respectively). The mean fold increase in anti-RSV neutralizing antibody in the high dose group was 9.24 (95% CI 4.07, 21.02) and the observed mean fold increase in the low dose group was 4.85 (95% CI 2.22, 10.59). The mean fold change for placebo treated patients was 1.42 (95% CI 0.64, 3.17). In addition, more patients in the high dose (85.7%) and low dose (42.9%) groups experienced greater than a 4-fold increase from baseline to day 18 in RSV titer levels compared to placebo (0%). There were no serious drug-related adverse events reported during the trial.

From April 2009 to February 2011, RI-001 was also administered to 15 compassionate use patients where physicians requested access to the product for treating their patients with documented lower respiratory tract RSV infections due to the fact that these patients had failed conventional therapeutic interventions. Serum samples were obtained from 13 patients. Samples showed that patients demonstrated a 4-fold or greater rise in RSV antibody titers from baseline. Serum samples were not obtained from 2 patients that received palivizumab. All patients (11) who received RI-001 within 4.2 days after the onset of the diagnosis of RSV survived. The drug was well-tolerated in all 15 patients and there were no reports of serious adverse events attributable to RI-001. Data from our Phase II trial, compassionate use experience, and data obtained from the evaluation of RI-002 in the infected cotton rat animal model have been presented at various conferences during 2014, 2015, and 2016.

Based on these results, we look forward to working with the FDA and the immunology and infectious disease community on developing a clinical investigation to evaluate use of ASCENIV™ in immune-compromised patients infected with or at-risk for RSV infection.