First in Human Study to Test the Safety and Preliminary Efficacy of PPSGG in Patients With Anti-MAG Neuropathy
First in Human Study to Test the Safety and Preliminary Efficacy of PPSGG in Patients With Anti-MAG Neuropathy
In this study, the new drug called PPSGG (PN-1007) will be tested. Preliminary studies
 conducted in animals suggest PPSGG (PN-1007) might be a good treatment for reducing levels of
 anti-MAG antibodies in patients with anti-MAG neuropathy.

 This is the first research of PPSGG (PN-1007) in people and its main purpose is to test its
 safety and acceptability in patients. In this study it will be examined how the drug is
 changed by and removed from the body and checked for signs that the drug may be truly
 effective against anti-MAG neuropathy. PPSGG (PN-1007) will be tested at several different
 doses.
PPSGG (PN-1007) is intended to bind anti-MAG IgM autoantibodies, the underlying cause of
 anti-MAG neuropathy, in a highly selective manner, resulting in their neutralization and
 removal from the circulation. This allows specific targeting of anti-MAG IgM in the
 circulation and circumvents unspecific immunosuppression associated with current treatment
 strategies.

 This is a Phase I/IIa, First in Human (FiH), multicenter, single and multiple ascending dose
 escalation trial of PPSGG (PN-1007), an antibody scavenger of pathogenic anti-MAG
 immunoglobulin M (IgM) autoantibodies for treatment of anti-MAG neuropathy. The aim of the
 study is to assess the safety and tolerability, pharmacokinetics (PK), pharmacodynamics (PD),
 and preliminary efficacy of PPSGG (PN-1007) in a SAD and a MAD phase in an adaptive trial in
 anti-MAG neuropathy patients.
Inclusion Criteria:

 -  Patients with a confirmed diagnosis of monoclonal IgM associated with MGUS with
 anti-MAG activity (titer of > 10'000 BTU) and demyelinating neuropathy defined by
 electrophysiological criteria according to EFNS/PNS PDN guideline, 2010.

 -  Clear clinical signs of disability

 -  Adequate hepatic and renal function

 Exclusion Criteria:

 -  Patients with total serum IgM levels >30 g.

 -  Hematological malignancy, prior malignancy of any organ system (except BCC)

 -  Prior immunosuppression: No IVIG in previous 3 months, no previous cyclophosphamide or
 biologicals in prior 6 months.

 -  Other neurological, neuromuscular, rheumatologic or orthopedic condition with
 significant impact on the capabilities of walk preventing evaluation of neurological
 scores