AVG-001 is a novel, inhaled gene therapy designed to promote localised production of alpha-1 antitrypsin to treat patients with AATD. The Company is aiming to progress this candidate towards clinical development over the next 2-3 years.
AATD is a rare inherited disorder in which patients produce reduced levels of alpha-1 antitrypsin, a protective plasma protein that safeguards the lungs from inflammation and tissue damage caused by infection and inhaled irritants. AATD affects at least 100,000 people in the US and a similar number in Europe and is a major genetic risk factor for progression to emphysema.
The condition remains substantially underdiagnosed and it can take several years until a patient with AATD is identified. There is no available cure for patients with AATD and the current standard of care is either symptomatic treatment, or in some countries weekly IV infusions of human plasma-derived functional alpha-1 antitrypsin.
Recently, a new at-home diagnostic test (AlphaID™ at Home; Grifols, Spain) has been approved by the US Food & Drug Administration and is now commercially available. It is anticipated that this product will improve the rates of early diagnosis substantially and help identify the more than 90% of people with AATD believed to be undiagnosed, and who might benefit from treatment.
Current commercial estimates put the market for AATD at circa $1Bn rising to $3Bn with improved disease awareness and diagnosis. Transforming the treatment paradigm would be of major beneficial impact to patients and healthcare outcomes alike.
AVG-002 uses the same proprietary lentiviral vector as that used for AVG-001 to deliver the relevant transgene to the lung. AlveoGene believes that AVG-002 could transform the current treatment paradigm and provide a lasting solution to this very rare respiratory disease that affects newborns/infants.
SP-B deficiency is caused by inherited mutations in the SP-B gene (SFTPB) on chromosome 2, which leads to a partial or complete absence of surfactant protein B. It is an autosomal recessive condition. Infants present shortly after birth with respiratory distress and failure, despite assisted ventilation and surfactant replacement therapy. The diagnosis is made by genetic testing for the mutation in the child and both parents. SP-B deficiency carries a poor prognosis and children with this disorder are very unlikely to survive beyond the first few months of life. Presently, the only effective treatment is lung transplantation with the availability of suitable organs being very limited.
The pulmonary surfactant market is dominated by their use in pre-term infants. Whilst it is forecast to reach approximately $800m by 2030 with a CAGR of around 4.5%, their use is not regarded as a viable solution for the treatment of SP-B deficiency.
With AVG-002 we believe we will be creating a first and best-in-class, life-changing product together with a new high value market opportunity, providing a lifelong solution where presently there is none.
AVG-003 uses the same proprietary lentiviral vector for lung gene delivery as that used for AVG-001 and AVG-002. AlveoGene believes that AVG-003 could transform the current treatment paradigm and provide a lasting solution to this very rare respiratory disease that affects newborns/infants.
ABCA3 surfactant protein disorder is an autosomal recessive condition caused by inherited mutations in the ABCA3 gene (ABCA3) on chromosome 16. This results in the loss or defective function of the phospholipid transporter required to produce pulmonary surfactant.
Individuals with ABCA3 deficiency may present at birth, with severe respiratory distress similar to SP-B deficiency, or in older children where the disease may be milder but with chronic breathing difficulties and failure to thrive. The prognosis is variable, depending on the severity of the disease. In the most severe cases children require lung transplantation to survive, with only limited availability of suitable organs.
With AVG-003 we believe we will be creating a first and best-in-class, life-changing product together with a new high value market opportunity, providing a lifelong solution where presently there is none.
AVG-004 uses the same proprietary lentiviral vector that is used for both AVG-001, AVG-002 and AVG-003 to deliver the relevant transgene to the lung.
AlveoGene believes that AVG-004 could transform the current treatment paradigm for IPF, by slowing the progression of IPF and improving lung function to extend patient lifespan and improve quality of life.
Idiopathic pulmonary fibrosis (IPF) is a serious chronic disease that affects the tissue surrounding the air sacs, or alveoli, in the lungs. This condition develops when lung tissue becomes thick and stiff for unknown reasons. Over time, these changes can cause permanent scarring in the lungs, called fibrosis, making it progressively more difficult to breathe.
Family history, smoking and increasing age are risk factors for developing IPF. The most common symptoms of IPF are shortness of breath and cough. Some people may not have symptoms at first, but symptoms can develop and get worse as the disease progresses.
The way that IPF progresses varies from person to person, and scarring may happen slowly or quickly. In some people, the disease stays the same for years. In other people, the condition quickly gets worse. Many people with IPF also experience acute exacerbations, where symptoms suddenly become much more serious. Other complications of IPF include pulmonary hypertension and respiratory failure, which happen when the lungs cannot deliver enough oxygen into the bloodstream without support. This prevents the brain and other organs from getting the oxygen they need. There is currently no cure for IPF.
Third-party analyses estimate a market size of approximately $6B by 2030 with a CAGR of around 7%.4,
