# Targeted Therapy for Myasthenia Gravis

> **NIH NIH R41** · ARC BIOTECHNOLOGY, LLC · 2020 · $232,839

## Abstract

Myasthenia gravis is a severe neuromuscular disease primarily caused by complement
activating autoantibodies directed towards the skeletal muscle acetylcholine receptor. Present
treatments are ineffectiveand uniformly have adverse effects, which negatively impact quality of
life. Despite being a rare disease, myasthenia gravis is a costly one with a hospitalization
expense of half a billion dollars in 2013. The investigative team through extensive basic and
preclinical assessment have demonstrated the complement system as a valid therapeutic
target, which has been further confirmed by the recent FDA approval of a first generation
complement inhibitor as treatment for generalized myasthenia gravis. We have shown in proof-
of-concept investigations the ability to target a complement inhibitor to the neuromuscular
junction and moderate disease severity in rodent models of myasthenia gravis. The work led to
issuance of U.S. Patent No. 8,961,981 for targeting a therapeutic to the neuromuscular junction
and our establishment of ARC BioTechnology, LLC to develop the technology for human use. If
we are successful in fulfilling the expectations of our candidate drug profile, we will produce a
drug with limited toxicity associated with systemic complement inhibition, prolonged
duration of action, and enhanced efficacy compared with presently available drugs and
those in development. In Phase 1 of the STTR we propose three mile-stone driven specific
aims in order to develop a lead compound for phase 2 development. Aim 1 will achieve small
batch synthesis of a series of constructs with a complement inhibitor domain and a domain
targeting the compound to proteins concentrated at the neuromuscular junction. Aim 2 will
identify lead compound (s) through validated in vitro assays of complement inhibitory activity
and affinity for target proteins. Aim 3 assesses lead compound(s) for moderation of severity in
validated animal models of myasthenia gravis produced by administration of antibody directed
against the acetylcholine receptor or immunization with purified acetylcholine receptor.
Successful completion of the milestones will trigger progression to Phase 2 to assess efficacy
and pharmacodynamics of the lead compound.

## Key facts

- **NIH application ID:** 10004729
- **Project number:** 5R41NS110331-02
- **Recipient organization:** ARC BIOTECHNOLOGY, LLC
- **Principal Investigator:** HENRY J KAMINSKI
- **Activity code:** R41 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $232,839
- **Award type:** 5
- **Project period:** 2019-09-01 → 2022-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10004729

## Citation

> US National Institutes of Health, RePORTER application 10004729, Targeted Therapy for Myasthenia Gravis (5R41NS110331-02). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10004729. Licensed CC0.

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