RARE Daily

Avalo Raises $27.5 Million to Advance Pipeline of Cancer and Rare Genetic Disease Therapies

September 15, 2021

Avalo Therapeutics, formerly Cerecor, said it has entered into an agreement with private investors to provide it with $27.5 million in new capital to advance its clinical stage pipeline of precision medicines for patients with significant unmet clinical need in immunology, immuno-oncology, and rare genetic diseases.

The underwriters agreed to purchase 12.5 million shares of common stock of Avalo at $2.20 per share. Avalo has also granted the underwriters a 30-day option to purchase up to an additional 1.9 million shares of common stock at public offering price. Avalo intends to use the net proceeds of the offering for working capital and other general corporate purposes.

Avalo Therapeutics’ precision medicine approach de-risks programs by reducing development cost and targeting the right patients who may benefit from the therapies, increasing the probability of clinical and regulatory success. It does this by identifying accessible molecular targets that have strong associations to diseases with substantial unmet need, verifying the role of the potential target in disease pathology and establish its utility as a biomarker, investigating novel therapeutics aimed at altering the identified molecular targets, then progressing these targeted therapeutics through a proof-of-concept study to demonstrate efficacy and safety, accelerating the time to clinical development.

The company’s rare genetic disease pipeline includes experimental therapies for lymphatic malformations and congenital disorders of glycosylation.

Complex lymphatic malformations are rare, noncancerous masses thought to be caused by the abnormal development of the lymphatic system and are usually present at birth or by 2 years of age. Regardless of size, lymphatic malformations may cause functional impairment of nearby structures or organs and disfigurement of affected areas.

Complex lymphatic malformations often have activating variants along the PI3K/AKT/mTOR pathway, leading to local proliferation of lymphatic endothelial cells and perturbation of lymph flow. Currently, the main therapeutic options for treating lymphatic malformations are percutaneous drainage, surgery, sclerotherapy, laser therapy, radiofrequency ablation, and medical therapy. The single mTORC1 inhibitor sirolimus has demonstrated clinical utility in lymphatic malformations. Avalo is testing its experimental candidate AVTX-006, a dual mTOR inhibitor, for its potential to improve upon both the safety and efficacy of sirolimus in lymphatic malformations.

Avalo also has three pipeline candidates that are a therapeutic dose of monosaccharide therapy in development for three different ultra-rare CDGs: PGM1-CDG, MPI-CDG, and LAD II.

Congenital disorders of glycosylation (CDG) is an umbrella term for a rapidly expanding group of more than 130 rare genetic, metabolic disorders due to defects in a complex chemical process known as glycosylation, a process by which sugar molecules are attached to proteins and fats. Glycosylation involves many different genes, encoding many different proteins such as enzymes. A deficiency or lack of one of these enzymes can lead to a variety of symptoms potentially affecting multiple organ systems. CDG can affect any part of the body and there is nearly always an important neurological component. CDG can be associated with a broad variety of symptoms and can vary in severity from mild to severe, disabling or life-threatening. CDG are usually apparent from infancy. Individual CDG are caused by changes (mutations) in a specific gene. Most CDG are inherited as autosomal recessive conditions, but some are X-linked or dominant. Others may arise spontaneously.

Author: Rare Daily Staff

Stay Connected

Sign up for updates straight to your inbox.