First-In-Human Clinical Study of the C3 Complement Inhibitor AMY- 101 in Healthy Male Volunteers
First-In-Human Clinical Study of the C3 Complement Inhibitor AMY- 101 in Healthy Male Volunteers
Safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of a Single Ascending
 Dose (SAD) and a Multiple Dose (MD) of the complement inhibitor AMY-101. A prospective,
 single-center, open-label, First-In-Human (FIH) clinical study in healthy male volunteers.
AMYNDAS is developing a novel peptidic complement inhibitor AMY-101, based on the
 third-generation compstatin analogue Cp40. AMY-101 is a selective inhibitor of complement
 activation in humans and in NHP. It binds to the complement component C3, the central
 "functional hub" that controls the upstream activation/amplification and downstream effector
 functions of complement. By binding to C3, AMY-101 inhibits the cleavage of native C3 to its
 active fragments C3a and C3b. As a consequence, the deposition of C3b, amplification via the
 alternative pathway and all downstream complement responses are prevented. AMY-101 is being
 developed to treat complement-mediated diseases, which are largely driven by aberrant C3
 activation.

 This first-in-human study of the C3-targeting complement inhibitor AMY-101 investigates the
 safety and PK/PD profile of AMY-101 in healthy male volunteers after Single Ascending Dose
 (SAD) and Multiple Doses (MD) using subcutaneous (SQ) or intravenous (IV) administration. The
 study is a prospective, single-center, open-label evaluation in healthy male volunteers.
Inclusion Criteria:

 1. Willing and able to give written informed consent for participation in the study.

 2. Healthy male subject aged 18-60 years, inclusive at the time of signing the informed
 consent.

 3. Body Mass Index (BMI) ≥ 18 and ≤ 30 kg/m2 and weight at least 50 kg.

 4. Clinically normal medical history, physical findings, vital signs, ECG and laboratory
 values at the time of screening, as judged by the Investigator.

 5. Willing to use condom and contraceptive methods with a failure rate of < 1% to prevent
 pregnancy and drug exposure of a partner and to refrain from donating sperm from the
 date of dosing until three (3) months after dosing of the IMP.

 6. Willing and able to complete all procedures according to the Protocol.

 Exclusion Criteria:

 1. History of any clinically significant disease or disorder which, in the opinion of the
 Investigator, may either put the subject at risk because of participation in the
 study, or influence the results or the subject's ability to participate in the study.

 2. History of any Neisseria meningitidis infection

 3. History of unexplained, recurrent infection, or infection requiring treatment with
 systemic antibiotics within the last 60 days prior to dosing.

 4. History of latent or active tuberculosis, as assessed by the investigator based on
 chest X-ray and positive Quantiferon-TB Gold test.

 5. History of complement deficiency.

 6. History of any type of malignancy. However, completely resolved minor malignancies, at
 the discretion of the Investigator, may not be an exclusion criterion (for example:
 Non-Melanoma Skin Cancer).

 7. Diagnosis of autoimmune, immunologic or rheumatologic disease (eg, systemic lupus
 erythematosus, rheumatoid arthritis).

 8. Any clinically significant illness, medical/surgical procedure or trauma within four
 (4) weeks of the administration of IMP.

 9. High CRP at screening (> 0.5 mg/dL).

 10. Current evidence or history of bacterial, viral or fungal infection within 14 days
 prior to (first) dosing or longer according to the judgment of the investigator for
 e.g. viral infections including herpes simplex or herpes zoster.

 11. Any current condition or risk, which, in the opinion of the Investigator, may
 interfere with the subject's participation in the study, poses an added risk for the
 subject, or confounds the assessment of the subject or outcome of the study

 12. Any clinically significant abnormality in clinical chemistry, hematology, or
 urinalysis results at the time of screening, as judged by the Investigator.

 13. Any positive result on screening for serum hepatitis B surface antigen, hepatitis C
 antibody and/or Human Immunodeficiency Virus (HIV).

 14. After 10 minutes supine rest abnormal vital signs defined as any of the following:

 -  Systolic BP > 140 mm Hg

 -  Diastolic BP > 95 mm Hg

 -  HR < 40 or > 90 beats per minute

 15. Prolonged QTcF (>450 ms), cardiac arrhythmias or any clinically significant
 abnormality in the resting ECG, as judged by the Investigator.

 16. Any history of any allergy/hypersensitivity or anaphylactic reaction or on-going
 allergy/hypersensitivity. History of hypersensitivity to antibiotics or drugs with a
 similar chemical structure or class to AMY-101.

 17. Use of any prescribed or non-prescribed medication including antacids, analgesics,
 herbal remedies, vitamins and minerals within two (2) weeks prior to the
 administration of IMP, except the occasional intake of paracetamol/acetominophen
 (maximum 2000 mg/day; and not exceeding 3000 mg/week) or nasal decongestant without
 cortisone or antihistamine, at the discretion of the Investigator.

 18. Administration of another new chemical entity (defined as a compound that has not been
 approved for marketing) or having participated in any other clinical study that
 included drug treatment within 30 day or 5 half lives of the last administration of
 the other IMP. Subjects consented and screened but not dosed in previous phase I
 studies are not excluded.

 19. Immunization with a live-attenuated vaccine one (1) month prior to the first
 administration of IMP.

 20. Current smokers or users of nicotine products. Irregular use of nicotine (e.g.
 smoking, snuffing, chewing tobacco) less than three (3) times per week is allowed
 before screening visit.

 21. History of alcohol abuse or excessive intake of alcohol, as judged by the
 Investigator.

 22. Positive screen for drugs of abuse at screening or on admission to the unit or
 positive screen for alcohol at screening or on admission to the unit prior to
 administration of the IMP.

 23. Use of anabolic steroids.

 24. Plasma donation within one (1) month of screening or any blood donation/blood loss >
 450 mL during the three (3) months prior to screening.

 25. Investigator considers the subject unlikely to comply with study procedures,
 restrictions and requirements.