Vaxcyte, Inc. announced positive regulatory updates, including the United States Food and Drug Administration (FDA) clearance of the VAX-31 infant Investigational New Drug (IND) application and the FDA granting Breakthrough Therapy designation (BTD) for VAX-31 for the prevention of invasive pneumococcal disease (IPD) in adults. VAX-31, the broadest-spectrum PCV candidate in the clinic, is a potentially best-in-class investigational 31-valent pneumococcal conjugate vaccine (PCV) candidate being studied for the prevention of IPD in the adult and pediatric populations. VAX-31 is designed to cover approximately 94% of IPD in U.S. children under five and over 95% of IPD in U.S. adults, with the potential to offer much greater coverage relative to the standard-of-care PCVs against both currently circulating and historically prevalent strains.
VAX-31 Infant IND Clearance and Phase 2 Study Initiation: The FDA clearance of the VAX-31 infant IND application, supported by the positive topline safety, tolerability and immunogenicity results from the VAX-31 adult Phase 1/2 study, enables the initiation of a VAX-31 pediatric study that proceeds directly into healthy infants. The VAX-31 infant Phase 2 study will be a randomized, double-blind, active-controlled, dose-finding clinical study that will include a primary immunization series consisting of three doses at two, four and six months of age followed by a subsequent booster dose at 12-15 months of age concomitantly with routine pediatric vaccines. Despite the effectiveness of current vaccines, IPD remains a significant threat during the first years of life.
Approximately 300,000 children under the age of five worldwide die every year due to Streptococcus pneumoniae which is the leading cause of vaccine-preventable fatalities in this age group. The burden of disease in the pediatric population underscores the need for a broader-spectrum vaccine. VAX-31 was designed to cover approximately 94% of IPD and approximately 86% of acute otitis media in children under five years of age in the United States.
VAX-31 Adult Breakthrough Therapy Designation: The FDA?s decision to grant BTD to VAX-31 in the adult population for the prevention of IPD was informed by the positive topline results from the VAX-31 Phase 1/2 study in adults. Based on the strength of these study results, the Company selected VAX-31 to exclusively advance to an adult Phase 3 program. The FDA?s BTD process is designed to expedite the development and review of drugs that are intended to treat serious or life-threatening conditions.
The designation is based upon preliminary clinical evidence indicating that the drug or vaccine may demonstrate substantial improvement over available therapies on one or more clinically significant endpoints. With BTD, Vaxcyte will have access to all the elements of the FDA?s Fast Track program, as well as the ability to receive guidance and support from the FDA on an efficient drug development program and an organizational commitment from senior managers within the FDA. PCV Franchise Adult Indication: VAX-31: Following an FDA End-of-Phase 2 meeting, initiate a Phase 3 pivotal, non-inferiority study by mid-2025 and announce topline safety, tolerability and immunogenicity data in 2026.
Initiate remaining Phase 3 studies in 2025 and 2026. PCV Franchise Infant Indication: VAX-24: Announce topline safety, tolerability and immunogenicity data from the primary three-dose immunization series of the Phase 2 study, which is fully enrolled with 802 healthy infants, by the end of the first quarter of 2025, followed by topline data from the booster dose by the end of 2025. VAX-31: Initiate Phase 2 study by the end of January 2025.
Announce topline safety, tolerability and immunogenicity data from the VAX-31 infant Phase 2 study primary three-dose immunization series in mid-2026, followed by topline data from the booster dose approximately nine months later.