AlveoGene’s Novel, Inhaled Gene Therapy AVG-002 Receives Orphan Drug Designation from FDA for Lethal Neonatal Surfactant Protein B (SP-B) Deficiency

• 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

Oxford, UK – 3 December 2024 – AlveoGene (or ‘the Company’, www.alveogene.com), a company focused on transforming rare respiratory disease outcomes using inhaled gene therapy, announces that the US Food & Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to AVG-002, the Company’s novel, inhaled gene therapy for lethal neonatal Surfactant Protein B (SP-B) deficiency. This award follows the recent grant of Rare Pediatric Disease Designation for AVG-002 from FDA in November.

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only provide temporary relief and once genetic diagnosis of SP-B deficiency is confirmed, treatment is usually withdrawn and patients die.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.