Multiple sclerosis (MS) is a common chronic neurological disorder affecting approximately 1 in 1000 Caucasians, particularly persons of Northern European origin who reside in temperate climates. Although there is still no cure for MS, several drugs can delay the progression of clinical worsening of patients with relapsing-remitting MS (RRMS). However, no treatment is available for primary and secondary progressive MS (PPMS and SPMS). For more than 10 years, we have focused our research on the role of CD24 in MS. Our studies in animal models established that the primary function of CD24 is to regulate the persistence of autoreactive T cells after they have reached the CNS. The significance of this checkpoint for MS is supported by genetic studies that demonstrate that a polymorphism of CD24 controls risk and/or progression of MS. Based on these novel observations, we have devoted more than 10 years of effort to develop therapeutics that target CD24 for the treatment of MS. We have completed manufacture, efficacy, and toxicity studies in animal models of EAE and in non-human primates. We have recently received FDA approval of our IND for a first-in- human study. Here we propose a randomized, double-blind, placebo-controlled, single ascending dose study to assess the safety, tolerability, immunogenicity and pharmacokinetics of CD24Fc in healthy adult subjects. The phase I studies will be carried out by one of the most respected CROs in the nation with strong track record in clinical drug testing. The phase I studies proposed herein will provide crucial safety information for a go-no-go decision of the phase II studies. The human PK and immunogenicity data will be valuable for the design of phase II studies.

Public Health Relevance

The main goal of the study is to develop a drug for the treatment of multiple sclerosis. The phase I studies proposed herein will provide crucial safety information for a go-no-go decision for phase II studies. The human pharmacokinetics and immunogenicity data will be valuable for the design of phase II studies.

National Institute of Health (NIH)
National Institute of Neurological Disorders and Stroke (NINDS)
Research Project (R01)
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National Institute of Neurological Disorders and Stroke Initial Review Group (NSD)
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Conwit, Robin
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Oncoimmune, Inc.
United States
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Mills, Elizabeth A; Mao-Draayer, Yang (2018) Aging and lymphocyte changes by immunomodulatory therapies impact PML risk in multiple sclerosis patients. Mult Scler 24:1014-1022
Mills, Elizabeth A; Mao-Draayer, Yang (2018) Understanding Progressive Multifocal Leukoencephalopathy Risk in Multiple Sclerosis Patients Treated with Immunomodulatory Therapies: A Bird's Eye View. Front Immunol 9:138
Mills, Elizabeth A; Begay, Joel A; Fisher, Caitlyn et al. (2018) Impact of trial design and patient heterogeneity on the identification of clinically effective therapies for progressive MS. Mult Scler :1352458518800800
Mirza, Ali; Mao-Draayer, Yang (2017) The gut microbiome and microbial translocation in multiple sclerosis. Clin Immunol 183:213-224
Du, Fanny Huynh; Mills, Elizabeth A; Mao-Draayer, Yang (2017) Next-generation anti-CD20 monoclonal antibodies in autoimmune disease treatment. Auto Immun Highlights 8:12
Soltys, John; Wang, Qin; Mao-Draayer, Yang (2017) Optical coherence tomography and T cell gene expression analysis in patients with benign multiple sclerosis. Neural Regen Res 12:1352-1356
Mao-Draayer, Yang; Sarazin, Jeffrey; Fox, David et al. (2017) The sphingosine-1-phosphate receptor: A novel therapeutic target for multiple sclerosis and other autoimmune diseases. Clin Immunol 175:10-15
Koshy Cherian, Ajeesh; Parikh, Vinay; Wu, Qi et al. (2017) Hemicholinium-3 sensitive choline transport in human T lymphocytes: Evidence for use as a proxy for brain choline transporter (CHT) capacity. Neurochem Int 108:410-416
Himedan, Mai; Camelo-Piragua, Sandra; Mills, Elizabeth A et al. (2017) Pathologic Findings of Chronic PML-IRIS in a Patient with Prolonged PML Survival Following Natalizumab Treatment. J Investig Med High Impact Case Rep 5:2324709617734248
Wu, Qi; Wang, Qin; Mao, Guangmei et al. (2017) Dimethyl Fumarate Selectively Reduces Memory T Cells and Shifts the Balance between Th1/Th17 and Th2 in Multiple Sclerosis Patients. J Immunol 198:3069-3080

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