• A Phase 1/2a clinical trial is underway in children ages six to 59 months of age

Blue Lake Biotechnology, Inc., a clinical-stage intranasal vaccine company developing PIV5-vectored vaccines that harness the full breadth of the immune system to protect against serious infectious diseases, announced the publication of positive Phase 1 clinical trial results of its intranasal RSV vaccine candidate BLB201 in Science Advances. Data generated during the first-in-human study of BLB201 showed that BLB201 was well-tolerated and stimulated RSV-specific antibody and cell mediated immune responses in adults ages 33 to 75 years following a single intranasal dose.

"In the ever-evolving landscape of vaccine development, our Phase 1 study of BLB201 showcases the potential of the PIV5 vector platform,” said Blue Lake’s chief executive officer, Biao He, Ph.D. “The positive results not only position BLB201 well in the race for an effective RSV vaccine but also pave the way for using the PIV5 platform in developing other novel vaccines. We are excited about the future possibilities and the impact this could have on global health."

BLB201 was found to increase RSV-specific serum antibody levels as well as RSV-specific mucosal antibodies following a single dose in adults and elderly, all of whom had evidence of prior exposure to RSV. In addition, administration of BLB201 was associated with increases in RSV F antigen-specific CD8⁺ cytotoxic T cells as well as Th1 CD4⁺ T cells (helper T cells), highlighting its potential to provide longer-lasting immunity against RSV. Of all intranasal RSV vaccine candidates that have so far been evaluated in adults, BLB201 has generated the most robust immune responses. The vaccine was well-tolerated. Side effects were generally mild, short-lived and self-limited.

"The findings from this phase 1 study of BLB201 represent a significant leap forward in the quest to provide children with a safe and easy to administer vaccine that can provide durable protection,” said Paul Spearman, MD, Professor of Infectious Diseases at Cincinnati Children’s Hospital Medical Center and principal investigator of the study. “We are especially excited to see strong T cell immune responses after intranasal administration, which are important for maintaining long-lasting immunity.”

RSV causes significant mortality and morbidity in the elderly and in infants. It has been difficult to develop an RSV vaccine that can be used in both populations, one which can overcome reduced immune responsiveness in the elderly while avoiding safety issues in infants. The Phase 1 results show that BLB201 is a promising intranasal vaccine candidate for the elderly. In addition, since live vaccines are believed to be safer for infant RSV vaccine development (i.e., unlikely to trigger a phenomenon called “enhanced RSV disease”), BLB201 may also be a safe and effective RSV vaccine for infants. To explore this potential, Blue Lake is now actively enrolling a Phase 1/2a study in infants and children between the ages of six to 59 months in the U.S.

About BLB201

BLB201 is an RSV vaccine candidate that is currently in Phase 1/2a clinical testing in children ages six 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 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 5 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 costly antibody drugs approved for use in infants that provide temporary passive immunity against RSV, there is no approved vaccine for generating prophylactic immunity against RSV. Such a vaccine is needed to protect populations at the highest risk from severe RSV disease, including the elderly as well as infants, to reduce the substantial health and economic burdens of RSV infection in the US and worldwide.

About CyanVac and Blue Lake Biotechnology

CyanVac LLC and its affiliate, Blue Lake Biotechnology, Inc., 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 high efficacy and durability with few vaccine-related side effects.

Learn more at Blue Lake Biotechnology

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