- Inherited SP-B deficiency is an ultra-rare monogenic cause of fatal respiratory distress syndrome in newborn infants with few, if any, options for long-term survival
- Highly encouraging preclinical data highlight the possibility of lifelong treatment from a single administration of AVG-002 with preparations for clinical development underway
- Follows recent grant of Rare Pediatric Disease Designation from FDA
Inherited SP-B deficiency is an ultra-rare monogenic cause of fatal respiratory distress syndrome in newborn infants with very limited treatment options. This genetic disorder, affecting approximately 1 in 1 million newborns in the US and Europe, results from mutations in the SP-B gene, which is essential for lung function and survival.
Current treatments are mainly supportive, including mechanical ventilation and surfactant replacement, however these interventions only provide temporary relief and once genetic diagnosis of SP-B deficiency is confirmed, treatment is usually withdrawn and patients die. The only definitive treatment, lung transplantation, is often not accessible owing to the scarcity of donor organs for newborns and the risks involved, leaving few if any viable options for long-term survival.
David Hipkiss, Executive Chair of AlveoGene, said: "Receiving Orphan Drug Designation from FDA for AVG-002 represents another important milestone for AlveoGene, following the recent grant of Rare Pediatric Disease Designation for the same candidate. Both awards highlight the significant unmet need for novel therapies targeting SP-B deficiency in newborn infants, which is invariably fatal within a few months and for which there are very limited treatment options. AVG-002, a novel gene therapy, has been designed using our InGenuiTy® technology to reach deep into the affected lung tissues to restore lung function and could provide a lifelong treatment from a single administration. We are totally committed to advancing AVG-002 as quickly as possible to provide a transformative, life-saving treatment for patients.”
AVG-002 is being developed by AlveoGene using its InGenuiTy® platform, which employs a unique pseudotyped lentiviral vector to deliver a functional SP-B gene directly to the neonatal deep lung alveolar region with high efficiency and efficacy via respiratory instillation.
Preclinical data in SP-B gene knock-out murine models demonstrate that a single dose of AVG-002 extends survival substantially longer when compared with reported data of other SP-B deficiency candidates in development, offering the possibility of a lifelong treatment from a single administration. These data are further reinforced by findings that confirm the restoration of normal lung histology and function following AVG-002 treatment in disease-induced lung tissues. Based on these highly encouraging findings, AlveoGene is advancing its preparations for the clinical development of AVG-002 in lethal neonatal SP-B deficiency with the possibility of filing for marketing authorisation by 2028.
FDA's Office of Orphan Drug Products grants Orphan Drug Designation (ODD) to support the development of medicines for rare disorders that affect fewer than 200,000 people in the US. Under the Orphan Drug Act, ODD qualifies a company for incentives, including tax credits, exemptions from certain FDA fees for clinical trials, and the potential for seven years of market exclusivity following drug approval.
ENDS
About AlveoGene
AlveoGene is a lung-targeted, inhaled gene therapy company aiming to transform the standard of care in respiratory disease and providing therapeutic solutions where few, if any, effective options exist.
The Company is advancing a pipeline of novel inhaled gene therapies with enhanced safety, efficacy, durability and improved targeting to lung tissues, based on its unique InGenuiTy® technology.
- AVG-002 and AVG-003 are potentially first-in-class disease-modifying therapies for newborns with rare lethal Surfactant Protein B (SP-B) and ABCA-3 deficiencies, respectively. AVG-002 is being developed under Rare Pediatric Disease Designation and Orphan Drug Designation with preparations for its clinical development underway.
- AVG-001 is designed to promote localised production of alpha-1 antitrypsin in the lung to treat patients with Alpha-1 Antitrypsin Deficiency (AATD) lung disease from a single treatment providing an alternative to standard-of-care therapy (weekly IV protein replacement). Preparations for clinical development are underway.
About GTC
The UK Respiratory Gene Therapy Consortium (GTC) comprises world-leading researchers at Imperial College London and the Universities of Oxford and Edinburgh who have worked together for more than 20 years with a focus on translating gene therapy for respiratory diseases. The GTC developed the novel pseudotyped lentiviral vector, which underpins AlveoGene's InGenuiTy® platform and pipeline, and which is relevant to both Cystic Fibrosis (licensed to and now being developed by Boehringer Ingelheim in the Lenticlair™ clinical trial) and multiple rare and other lung conditions.
Further details and commentary can be found at www.respiratorygenetherapy.org.uk/
Contacts
AlveoGene
David Hipkiss, Executive Chair - [email protected]
MEDiSTRAVA (for media enquiries)
Mark Swallow, Sandi Greenwood, Erica Hollingsworth - [email protected]
