About Avalo Therapeutics

Avalo Therapeutics, Inc. (Avalo), a clinical stage biotechnology company, focuses on the treatment of immune dysregulation by developing therapies that target the LIGHT-signaling network. LIGHT (Lymphotoxin-like, exhibits Inducible expression, and competes with HSV Glycoprotein D for Herpesvirus Entry Mediator (HVEM), a receptor expressed by T lymphocytes; also referred to as TNFSF14) is an immunoregulatory cytokine. LIGHT and its signaling receptors, HVEM (TNFRSF14), and lymphotoxin ß receptor (TNFRSF3), form an immune regulatory network with two co-receptors of herpesvirus entry mediator, checkpoint inhibitor B and T Lymphocyte Attenuator (BTLA), and CD160 (collectively, the LIGHT-signaling network or the LIGHT network). AVTX-002 is a fully human mAb, directed against human LIGHT. There is increasing evidence that the dysregulation of the LIGHT-signaling network which includes LIGHT, its receptor HVEM and LTßR and the downstream checkpoint BTLA, is a disease-driving mechanism in autoimmune and inflammatory reactions in barrier organs. Avalo is developing AVTX-002 for the treatment of non-eosinophilic asthma (NEA). The company’s Phase 2 PEAK Trial (A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Safety and Efficacy of AVTX-002 for the treatment of Poorly Controlled Non-Eosinophilic Asthma K) is fully enrolled and the company expects to release topline data in the second quarter of 2023. AVTX-002 previously demonstrated proof-of-concept in COVID-19 acute respiratory distress syndrome (ARDS), including reduction in mortality and respiratory failure. AVTX-002 also showed clinically meaningful mucosal healing in a Phase 1b study in Crohn’s Disease. Avalo will assess the next stage of development for these indications, as well as potentially other acute and inflammatory disorders, upon or close to the data readout of the PEAK Trial in NEA. On March 25, 2021, the company entered into a license agreement with Kyowa Kirin Co., Ltd. (KKC) for exclusive worldwide rights to develop, manufacture and commercialize AVTX-002 for all indications (the KKC License Agreement). The KKC License Agreement replaced the Amended and Restated Clinical Development and Option Agreement between the company and KKC dated May 28, 2020. Patents exclusively licensed from KKC may provide exclusivity in the United States through 2028 absent any extension, and additional patent applications filed by the company covering certain methods of using AVTX-002, if issued and properly maintained, should provide additional exclusivity through 2043, absent any extension. Additionally, if the company receives marketing approval, it expects to receive biologics data exclusivity in the United States, which would provide twelve years of data exclusivity in the United States from the date of FDA approval. AVTX-008 is a fully human BTLA agonist fusion protein. Avalo has identified a lead molecule and is evaluating several immune dysregulation disorders to pursue. Avalo has initiated activities for the preparation of an investigational new drug application (IND), with a target IND submission date in 2024. On June 21, 2021, the company entered into an Exclusive Patent License Agreement with Sanford Burnham Prebys Medical Discovery Institute (the Sanford Burnham Prebys License Agreement or the SBP License Agreement) under which Avalo obtained an exclusive license to a portfolio of issued patents and patent applications covering AVTX-008. Patents exclusively licensed from Sanford Burnham Prebys Medical Discover Institute may provide exclusivity in the United States through 2036 absent any extension. Additionally, if the company receives marketing approval, it expects to receive biologics data exclusivity in the United States, which may provide twelve years of data exclusivity in the United States from the date of FDA approval. AVTX-803 is a fucose replacement for leukocyte adhesion deficiency type II (LAD II, also known as SLC35C1-CDG). LAD II is a congenital disorder of glycosylation (CDG). Oral administration of AVTX-803 replenishes critical metabolic intermediates that are reduced or absent due to genetic mutations, overcoming single enzyme defects in respective CDGs to support glycoprotein synthesis, maintenance, and function. 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 (LADD) (ER)) is ongoing. AVTX-803 was granted ODD by the FDA. As a result, at a minimum, following marketing approval, the company plans to rely on seven-year marketing exclusivity in the United States. The company has one marketed product, Millipred, an oral prednisolone indicated across a wide variety of inflammatory conditions and indications. Prednisolone is a man-made form of a natural substance (corticosteroid hormone) made by the adrenal gland. It is used to treat conditions, such as arthritis, blood disorders, immune system disorders, skin and eye conditions, respiratory disorders, cancer, and severe allergies. Prednisolone decreases an individual's immune response to various diseases to reduce symptoms, such as pain, swelling and allergic-type reactions. Millipred is supplied in 5mg tablets. Millipred tablets primarily compete in the generic prednisolone market. Millipred utilizes the proprietary double taste-masking technology to provide a pleasant grape taste with no bitterness, which makes the product easier to administer to children. The company has a license and supply agreement for the Millipred product with a wholly owned subsidiary of Teva Pharmaceutical Industries Ltd. (Teva), which expires on September 30, 2023. Strategy The key elements of the company’s strategy include advancing its pipeline of compounds through development and to regulatory approval; developing the go-to-market strategy to quickly and effectively market, launch, and distribute each of its compounds that receive regulatory approval; opportunistically out-licensing rights to indications or geographies; and acquiring or licensing rights to targeted, complementary differentiated preclinical and clinical stage compounds. Sales and Marketing For its clinical stage pipeline assets, the company may retain or partner in the United States with third parties on the commercialization rights and develop sales and marketing capabilities when needed. If the company develops its own United States sales force it may complement it with co-promotion agreements with partners in and outside the United States. The company may also seeks to commercialize any of its approved products outside of the United States and may do so either through an expansion of its sales force or through collaboration with third parties. Government Regulations AVTX-803 was granted Orphan Drug Designation (ODD) by the United States Food and Drug Administration (the FDA). The company relies on contract manufacturing organizations to produce its drug candidates in accordance with applicable provisions of the FDA’s current good manufacturing practices (cGMP) regulations for use in its clinical studies. History The company was founded in 2011. It was incorporated in 2011 in Delaware. The company was formerly known as Ceregen Corporation and changed the name to Cerecor Inc. in March 2011 and to Avalo Therapeutics, Inc. in August 2021.