Precision Therapies for Rare Diseases

Product Pipeline

We are expanding our pipeline to include new antibody therapies for both rare and non-rare diseases, focusing on identifying rare diseases with a dysregulated complement system. Our therapies target Hematological, Renal, Inflammatory, and Ocular disorders. We have received regulatory approvals from U.S. and international agencies, including Orphan Drug Designation from the FDA for PNH. We are also preparing an IND application for Geographic Atrophy (GA) and other Inflammatory Ocular Disorders (e.g., Uveitis). Our approach encompasses inventing new therapies capable of targeting multiple diseases with a single drug, while creating multiple drugs to address a range of indications.

NM5072 : Preclinical animal model studies have shown efficacy across hematology, inflammatory, and ocular disease models, and the drug has been extensively studied in serum and blood samples from PNH patients. The drug has received Orphan Drug Designation (ODD), and Phase II INDs have been approved for hematological and renal indications.

NM8074 : The drug has received  Orphan Drug Designation. Multiple Phase II INDs have been  approved by the FDA for PNH, IgAN, aHUS, C3G, AAV, and DM. As Phase II is closing, Phase III regulatory documentation is being prepared for submission. Based on the compelling data, a Breakthrough Therapy Designation filing is planned.

NM8070: NM8070 is in preclinical development for Geographic Atrophy. It differentiates itself from Apellis’ C3 blocker by selectively targeting the alternative pathway. Full ocular toxicology studies have been completed, and an ocular IND submission is planned to support a Phase II GA study.

NM4651: NM4651 has been developed as a screening tool to identify additional complement-mediated rare diseases. As a result, we can generate a more extensive portfolio of rare diseases treatable with our drugs.

Our strategy is to align with our partners' business objectives, which may involve licensing one or more antibodies for multiple indications. Our experience has confirmed that therapeutic efficacy varies between targets and antibodies, leading us to continuously invent new antibodies and develop new intellectual property. We are actively pursuing regulatory approvals for additional indications as we advance our pipeline to new heights.

Complement-Mediated Diseases