Avalo Therapeutics Announces First Patient Dosed in the AVTX-803 Pivotal Trial (LADDER) for the Treatment of Leukocyte Adhesion Deficiency Type II (LAD II)

Rhea-AI Summary

Avalo Therapeutics has announced that the first patient has been dosed in the pivotal LADDER trial for their drug AVTX-803, aimed at treating Leukocyte Adhesion Deficiency Type II (LAD II). This Phase 3 trial is randomized, double-blind, and designed to evaluate the drug's efficacy and safety over 16 weeks. AVTX-803 has received multiple designations, including Rare Pediatric Disease and Fast Track. Results are expected in 1H 2023, potentially leading to regulatory approval and a Priority Review Voucher.

Positive

• First patient dosed in pivotal LADDER trial for AVTX-803, advancing towards potential approval.
• AVTX-803 received Rare Pediatric Disease, Orphan Drug, and Fast Track Designation, facilitating expedited FDA review.

Negative

• Trial completion and results are contingent on patient enrollment, which may be affected by external factors like the COVID-19 pandemic.

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.

AVTX-803
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).

Pivotal Trial
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
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.

Forward-Looking Statements
This press release may include forward-looking statements made pursuant to the Private Securities Litigation Reform Act of 1995. Forward-looking statements are statements that are not historical facts. Such forward-looking statements are subject to significant risks and uncertainties that are subject to change based on various factors (many of which are beyond Avalo’s control), which could cause actual results to differ from the forward-looking statements. Such statements may include, without limitation, statements with respect to Avalo’s plans, objectives, projections, expectations and intentions and other statements identified by words such as “projects,” “may,” “might,” “will,” “could,” “would,” “should,” “continue,” “seeks,” “aims,” “predicts,” “believes,” “expects,” “anticipates,” “estimates,” “intends,” “plans,” “potential,” or similar expressions (including their use in the negative), or by discussions of future matters such as: the development of product candidates or products; timing and success of trial results and regulatory review; potential attributes and benefits of product candidates; the future financial and operational outlook; and other statements that are not historical. These statements are based upon the current beliefs and expectations of Avalo’s management but are subject to significant risks and uncertainties, including: drug development costs, timing and other risks, including reliance on investigators and enrollment of patients in clinical trials, which might be slowed by the COVID-19 pandemic; Avalo's cash position and the potential need for it to raise additional capital; reliance on key personnel, including as a result of recent management changes; regulatory risks; general economic and market risks and uncertainties, including those caused by the COVID-19 pandemic and tensions in Ukraine; and those other risks detailed in Avalo’s filings with the SEC. Actual results may differ from those set forth in the forward-looking statements. Except as required by applicable law, Avalo expressly disclaims any obligations or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in Avalo’s expectations with respect thereto or any change in events, conditions or circumstances on which any statement is based.

For media and investor inquiries

Christopher Sullivan, CFO
Avalo Therapeutics, Inc.
ir@avalotx.com
410-803-6793

or

Chris Brinzey
ICR Westwicke
Chris.brinzey@westwicke.com
339-970-2843

FAQ

What is the LADDER trial for AVTX-803?

The LADDER trial is a pivotal Phase 3 study assessing the efficacy and safety of AVTX-803 in patients with Leukocyte Adhesion Deficiency Type II.

When are the results of the LADDER trial expected?

Topline results from the LADDER trial are expected in the first half of 2023.

What designations has AVTX-803 received?

AVTX-803 has received Rare Pediatric Disease Designation, Orphan Drug Designation, and Fast Track Designation.

What condition does AVTX-803 aim to treat?

AVTX-803 is developed to treat Leukocyte Adhesion Deficiency Type II (LAD II), a rare genetic disorder.

Who announced the dosing of the first patient in the LADDER trial?

Avalo Therapeutics announced the dosing of the first patient in the LADDER trial.