Novel antibody therapeutics
The promise of precision patient-centric care.
Mabwell Therapeutics is developing a broad portfolio of antibody drugs to help address unmet medical needs. Our disciplined, science-driven R&D regimen in target selection, lead molecule discovery, and antibody engineering improves the developability of drug candidates.
Scientific rigor is the foundation of our successful therapeutic development.
Our programs are driven by scientific rationale, informed by high-quality research and deep biological understanding.
We optimize the efficiency, effectiveness, and value of our R&D through adherence to stringent governing principles:
- Prioritizing the selection of targets with compelling genetic and experimental evidence
- Applying rigorous, data-driven science to the discovery of lead molecules to enhance the developability of drug candidates
- Leveraging innovative engineering techniques to ensure that each therapeutic candidate has maximum potential for meaningful impact
Our proprietary targeted delivery platform
In the fight against rare and debilitating diseases, we’ve developed MyoDock™, a proprietary targeted delivery platform engineered to achieve optimal drug exposure at the neuromuscular junction (NMJ), the critical synapse between motor neurons and skeletal muscle fibers. This platform is specifically designed to improve outcomes for individuals with neuromuscular disorders.
The MyoDock platform utilizes monoclonal antibodies that selectively bind to a receptor expressed on the surface of skeletal muscle cells. Notably, expression of this receptor is significantly elevated in diseased muscle tissue, enabling dynamic, precision delivery of therapeutic molecules directly to the site of pathology. By concentrating drugs at the NMJ while minimizing systemic distribution, MyoDock enhances therapeutic index, allowing for reduced dosing and mitigating dose-limiting toxicities associated with systemic administration.
The versatile MyoDock platform can be used with a variety of therapeutic payloads, including peptides, proteins, and oligonucleotides. It enables the development of novel treatments for a broad range of neuromuscular diseases, including congenital myasthenic syndromes, myasthenia gravis, amyotrophic lateral sclerosis, spinal muscular atrophy. It also supports therapeutic innovation in various forms of muscle atrophy/dystrophy, such as myotonic dystrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and disuse-induced muscle atrophy.
By enabling molecular-level targeting at the site of disease, the MyoDock delivery platform represents a foundational technology in our mission to transform the treatment landscape for rare neuromuscular disorders.
Our pipeline
Mabwell Therapeutics Pipeline
MWTX-003 (anti-TMPRSS6 mAb)
Indication: Polycythemia vera, β-Thalessmia
The problem: Patients with iron overload disorders, such as polycythemia vera, beta thalassemia, myelodysplastic syndrome, and hereditary hemochromatosis, have limited therapeutic options against iron toxicity imposed by excessive iron loading. Significant morbidity and mortality can result, severely impacting one’s daily life.
The promise: MWTX-003 is a monoclonal antibody inhibitor of TMPRSS6 (Transmembrane Serine Protease 6, also known as Matriptase-2). Through increasing the production of hepcidin, the master regulator of iron homeostasis, MWTX-003 has demonstrated efficacy in improving iron toxicity in animal models of polycythemia vera and beta thalassemia.
- MWTX-003 has completed Phase I clinical trials in both US and China, demonstrating good safety and tolerability. Potent and durable suppression of serum iron was observed in healthy volunteers, align with expected PK/PD responses.
- MWTX-003 was out-licensed to Disc Medicine in January 2023 with global rights excluding Greater China and Southeast Asia.
Scientific Presentations
US Patent # 12215170 B2
MWTX-3011 (anti-C1s mAb)
Indication: Complement-mediated disorders
The problem: The complement system is an important component of innate immunity and a critical mechanism in maintaining tissue homeostasis. Aberrant activation of complement may result in tissue damage and fuel systemic inflammation, contributing to the pathogenesis of many diseases.
The promise: MWTX-3011 is a monoclonal antibody inhibitor of C1s, designed to modulate the activation cascade of the classical complement pathway (CP). By targeting CP activation driven by the engagement of autoantibody with self-antigen, it has the potential to address a broad range of rare autoimmune disorders. These include immune thrombocytopenia purpura (a rare autoimmune bleeding disorder), neuromyelitis optica (a debilitating autoimmune disease affecting the optic nerves and spinal cord), among other indications. Due to its greatly improved pharmacokinetic profile, MWTX-3011 is predicted to provide a Best-in-Class opportunity based on improved clinical dosing regimen relative to sutimlimab, the first FDA approved anti-C1s antibody for Cold Agglutinin Disease.
- MWTX-3011 is currently in the IND-enabling phase of development.
US Patent # 11958899 B2
MWTX–4611 (anti-Amyloid β mAb)
Indication: Alzheimer's Disease
The problem: Alzheimer’s disease (AD) is one of the most prevalent, burdensome and lethal diseases worldwide, ranked as the sixth leading cause of death in the United States. The economic burden of AD and other dementias is expected to grow to $17 trillion by 2050. AD patients eventually lose their ability to carry out daily activities due to memory loss, changes in behavior, and other disabilities. Despite recent therapeutic advances which have demonstrated the importance of amyloid beta (Aβ) in disease progression, AD remains poorly treated and an area of significant medical need.
The promise: MWTX-4611 is a fully human monoclonal antibody that selectively targets Aβ protofibrils, the neurotoxic form of amyloid beta implicated in Alzheimer’s disease. In a preclinical model of AD, MWTX-4611 significantly reduces protofibril levels and plaque burden in a dose-dependent manner. The antibody has also demonstrated a favorable toxicology and CMC profile, supporting its advancement into clinical development.
Importantly, MWTX-4611 is clearly differentiated from currently approved anti-Aβ therapies such as Leqembi™ (lecanemab). It exhibits approximately 5-fold higher selectivity for Aβ protofibrils over monomers, which effectively overcomes the “peripheral sink” effect associated with systemic administration and potentially enabling lower effective doses and extended dosing intervals.
- MWTX-4611 has received clearance from both FDA and NMPA to initiate clinical trials in the United States and China.
Scientific Presentations
US Patent # 12084492 B2
MWTX-04 (anti-MuSK mAb)
Myasthenia Gravis, Congenital myasthenic syndrome
The problem: The neuromuscular junction (NMJ) is a highly specialized synapse that enables communication between motor neurons and muscle fibers, converting electrical signals into muscle contraction. Disruption of NMJ function impairs neuromuscular transmission and leads to debilitating and potentially life-threatening muscle weakness, as seen in a range of neuromuscular diseases.
The promise: MWTX-04 is a fully human monoclonal antibody that selectively targets Muscle-specific kinase (MuSK), a receptor tyrosine kinase essential for the formation and maintenance of the NMJ. In preclinical models of congenital myasthenic syndrome (CMS), MWTX-04 demonstrated robust efficacy in preventing early postnatal lethality and restoring neuromuscular function in a dose-dependent manner, including reversal of disease relapse.
Importantly, MWTX-04 has broad clinical application across multiple neuromuscular disorders. In addition to diseases directly linked to impaired MuSK signaling and dysfunctional NMJ, such as CMS and Myasthenia Gravis (MG), MWTX-04 may benefit patients with conditions where impaired NMJ is a contributing factor, including amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA), potentially improving muscle function and overall quality of life.
- MWTX-04 is currently in the IND-enabling phase of development.
MWTX-05 Undisclosed
Congenital myasthenic syndrome
The problem: Congenital myasthenic syndrome (CMS) are a group of rare, inherited neuromuscular disorders caused by genetic defects that impair signal transmission at the neuromuscular junction (NMJ). To date, more than 25 genes have been implicated in the etiology of the CMS. Current therapies for CMS are largely symptom-based and depend on the underlying genetic subtype. A drug that benefits one CMS subtype can be harmful in another, making precision targeting essential. Some subtypes still lack any effective treatment options.
The promise: MWTX-05 is a first-in-class biotherapeutic designed to treat a specific CMS subtype for which no treatment option currently exists. Leveraging Mabwell’s proprietary NMJ-targeted delivery platform, MWTX-05 is directly delivered and concentrated at the NMJ. This precision-targeting approach increases local drug exposure, enhances efficacy and reduces off-target side effects typically associated with conventional systemic administration.
- Lead candidates have been identified and are being evaluated for efficacy in animal models.
MWTX-06 Undisclosed
Muscle atrophy/dystrophy
The problem: Muscle atrophy/dystrophy are hallmark features of many muscle diseases. While numerous therapies have aimed to promote muscle growth, most have failed to deliver meaningful functional improvement. Insufficient stimulation or formation of the neuromuscular junction (NMJ) may underlie the disconnect between muscle mass increase and functional improvement.
The promise: MWTX-06 is a first-in-class bifunctional therapeutic designed to address both muscle wasting/degeneration and NMJ dysfunction. It combines two synergistic mechanisms: one component drives muscle growth, while the other promotes NMJ maturation and maintains neuromuscular function. MWTx-06 is also enabled by Mabwell’s proprietary dynamic muscle-targeting delivery platform, which ensures selective payload delivery to atrophic or diseased muscle while sparing healthy tissue and off-target organs, minimizing systemic side effects.
With its dual-targeted design and precision delivery, MWTX-06 has the potential to treat both muscle atrophy/dystrophy and associated weakness across a broad spectrum of muscle diseases.