ATHENS, Ga. & SAN JOSE, Calif.--(BUSINESS WIRE)--Blue Lake Biotechnology, Inc., a clinical-stage intranasal vaccine company developing parainfluenza virus 5 (PIV5)-vectored vaccines that harness the full breadth of the immune system to protect against serious infectious diseases, today announced positive data from a preliminary analysis of the first 63 infants and young children enrolled in the company’s Phase 1/2a clinical study of BLB201, an intranasal vaccine candidate against RSV.
BLB201 has been well-tolerated by the participants in this double-blind placebo-controlled study, with no vaccine-related medically-attended adverse events or serious adverse events reported. Eight symptomatic RSV infections have been reported, all of which have been mild or moderate, with no severe cases or cases requiring hospitalization. Among the 63 children enrolled so far in the study (of which over one third were seronegative for RSV at baseline), a preliminary analysis of partially unblinded data showed that participants who received BLB201 have been more than 80% less likely to contract symptomatic RSV infections than participants who received placebo (p<0.05). Consistent with the immune responses seen in Blue Lake Biotechnology’s prior clinical studies, BLB201 has elicited moderate serum antibody responses and stimulated mucosal and cellular immune responses which are believed to be responsible for the vaccine’s protective activity.
“Historically, the biggest challenge that has stymied the development of infant RSV vaccines for decades is the risk of vaccine-associated enhanced respiratory disease, or VAERD,” said Henry Radziewicz, M.D., chief medical officer of Blue Lake Biotechnology. “In addition to the overall excellent tolerability and safety of BLB201 in infants and young children so far, it is reassuring that we have not detected any safety signals of VAERD.”
“The preliminary analysis showing a greater than 80% reduction in the risk of symptomatic RSV infection underscores the promise of BLB201 as a safe and effective vaccine in preventing symptomatic RSV disease in infants and young children,” added Biao He, Ph.D., founder and CEO of Blue Lake Biotechnology. “We believe these data strongly suggest that our intranasal BLB201 vaccine has a distinct mechanism of protection which is different from the immunity generated by injectable formalin-inactivated and mRNA-based RSV vaccines which have been associated with VAERD. Such vaccines elicit very high levels of serum antibody responses but have not conferred protection against symptomatic RSV infection in infants.”
The Phase 1/2a study (NCT05655182) is a double-blind, placebo-controlled study that is to enroll up to 137 RSV seropositive and seronegative infants and young children between eight and 59 months old in an adaptive design. To date, a total of 38 children have received either a single low dose or a single high dose of BLB201, and another 25 children have received placebo. Primary and secondary endpoints include assessments of safety, tolerability, and immunogenicity, and exploratory endpoints include assessment of the proportion of participants diagnosed with symptomatic RSV infection. The trial is ongoing, and some participants are still within the 12-month follow-up period of the trial.
About RSV
Respiratory syncytial virus (RSV) is a highly contagious and common respiratory virus and one of the leading causes of acute respiratory disease, infecting more than 64 million people worldwide per year. For most people, it causes symptoms similar to the common cold, but for infants, the immunocompromised, people with chronic heart or lung disease, and older adults, it can cause severe illness and be life-threatening. Repeat infections with RSV are believed to be common, so even people who contracted RSV when they were younger adults and healthy can be at risk from RSV infection later in life. According to the Centers for Disease Control, up to 80,000 children under five years old and up to 160,000 older adults are hospitalized per year because of RSV infection in the US, with up to 10,000 deaths each year.
While there are approved antibody drugs and a maternal RSV vaccine that can provide passive immunity against RSV to infants, there is no approved vaccine for generating prophylactic immunity against RSV in infants and children. Such a vaccine is needed to fully protect young populations at significant risk from severe RSV disease.
About BLB201
BLB201 is an RSV vaccine candidate that is currently in Phase 1/2a clinical testing in children ages eight to 59 months of age. It has received Fast Track designation from the US Food and Drug Administration for the prevention of RSV-associated acute respiratory disease in adults (>60 years) and pediatric populations (<2 years). BLB201 encodes the RSV F protein and uses a proprietary PIV5 vector, which is not known to cause human disease. PIV5 has been commonly administered to dogs as part of combination distemper/kennel cough vaccines for decades. Blue Lake Biotechnology is developing BLB201 as an intranasal vaccine to prevent acute and severe disease associated with RSV infection. Phase 1 clinical trial results demonstrate BLB201's safety and ability to induce antibody and cell-mediated immune responses, positioning it as a promising candidate in the fight against RSV.
About Blue Lake Biotechnology
Blue Lake Biotechnology, Inc. and its affiliate, CyanVac LLC, are developing intranasal vaccines that harness the full breadth of the immune system to keep people healthy, prevent serious infectious diseases, and protect the health of vulnerable populations. Our platform uses a proprietary parainfluenza virus 5 vector into which a foreign gene from a targeted pathogen is inserted. We have generated a robust clinical-stage pipeline of best-in-class vaccines designed to overcome the limitations of existing vaccine technologies. Our lead product candidates have demonstrated potential for efficacy following a single intranasal dose with few vaccine-related side effects.
Learn more at Blue Lake Biotechnology.
