Avalo Therapeutics Announces First Patient Dosed in the AVTX-803 Pivotal Trial (LADDER) for the Treatment of Leukocyte Adhesion Deficiency Type II (LAD II)
Topline Pivotal trial results expected 1H2023
WAYNE, Pa. and ROCKVILLE, Md., Aug. 02, 2022 (GLOBE NEWSWIRE) -- Avalo Therapeutics, Inc. (Nasdaq: AVTX), announced the first patient has been dosed in the pivotal LADDER trial (A Phase 3, Randomized, Double-blind, Two-period, Crossover, Withdrawal Study to Assess the Efficacy and Safety of AVTX-803 in Subjects with Leukocyte Adhesion Deficiency Type II (LAD II) ER). AVTX-803 is a therapy developed and studied for the treatment of leukocyte adhesion deficiency type II (LAD II, also known as SLC35C1-CDG). AVTX-803 has been granted Rare Pediatric Disease Designation (RPDD), Orphan Drug Designation (ODD) and Fast Track Designation (FTD).
“We are excited to announce the first patient was dosed in the LADDER pivotal trial. This substantially advances the program toward an approved treatment option for patients and also a potential PRV,” said Garry A. Neil, MD, President and Chief Executive Officer, Avalo Therapeutics.
The active pharmaceutical ingredient (API) in AVTX-803 is L-fucose. L-fucose is a plant-derived, naturally occurring monosaccharide with high solubility in water and is isolated as a white crystalline powder. AVTX-803 is an oral formulation of L-fucose that enhances fucosylation of proteins in the absence of a functioning GDP-fucose transporter, partially restoring protein function. AVTX-803 was granted FTD, ODD and RPDD, making it potentially eligible for a Priority Review Voucher (PRV).
The LADDER trial is a pivotal, 16-week randomized, double-blind, two-period crossover, withdrawal study to assess the efficacy and safety of AVTX-803 in patients with LAD II (n=2). The primary endpoint is the comparison of leukocyte function as determined by sialyl Lewis-X (SLx) antigen expression on leukocytes between treatment periods. The trial will conclude with the End of Study/Early Termination Visit at which time subjects will be permitted to enroll into a long-term open-label, safety and efficacy study.
LAD II is an ultra-rare, inherited congenital disorder of glycosylation (CDG), previously known as CDG-IIc and also referred to as SLC35C1-CDG. The disease is autosomal recessive and is characterized by reduced or absent fucosylation of certain glycoproteins. Patients with LAD II present with a multi-system phenotype that may include: leukocytosis, neutrophilia, recurrent infections, reduced selectin expression and binding, Bombay blood group, hypotonia, poor feeding, short stature, coarse facial features, global developmental delay and psychomotor retardation. In some cases, these symptoms lead to death, and in most LAD II patients, the disorder negatively impacts quality of life.
About Avalo Therapeutics
Avalo Therapeutics is a leading clinical-stage precision medicine company that discovers, develops, and commercializes targeted therapeutics for patients with significant unmet clinical need in immunology and rare genetic diseases. The Company has built a diverse portfolio of innovative therapies to deliver meaningful medical impact for patients in urgent need. The Company’s clinical candidates commonly have a proven mechanistic rationale, biomarkers and/or an established proof-of-concept to expedite and increase the probability of success.
For more information about Avalo, please visit www.avalotx.com.
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Released August 2, 2022