TGD001 Treatment in Thrombotic Microangiopathies

In Part A, dose escalation of TGD001 is conducted in participants with suspicion or clinical diagnosis of immune thrombotic thrombocytopenic purpura (iTTP) in conjunction with their respective standard of care to identify a tolerated dose(s) with a pharmacological/clinical response to be evaluated in Part B.

Once a recommended dose of TGD001 is established in Part A, the dose expansion/basket part of the trial will commence. In Part B, "basket" cohorts of participants with iTTP and other Thrombotic Microangiopathies (TMAs) will receive single or repeat doses of TGD001 in conjunction with their respective standard of care to determine the TGD001 dose and treatment regimen with clinical effect in reducing the initial thrombus burden.

Baskets will include up to 20 participants each and may be combined based on emerging safety and efficacy findings. Baskets may be further expanded with the TGD001 dose(s) and regimen(s) that displayed the best clinical response as assessed by the Data Safety Management Committee up to a total of approximately 60 participants in Part B.

Key Inclusion Criteria:

• 18 years old or older
• Willing to sign an informed consent form
• Willing to refrain from sexual intercourse or must use a contraceptive method that is highly effective for 90 days after the last dose of TGD001
• Symptomatic acute TMA episode
• Accessible to follow-up

Key Exclusion Criteria:

• Diagnosis other than TMA, which could account for the findings of thrombocytopenia and hemolytic anemia
• Diagnosis of Shiga-toxin induced HUS
• Known bone marrow/graft failure
• Diagnosis of ongoing severe, uncontrolled Graft versus Host Disease
• Received therapeutic plasma exchange (PEX) within 7 days prior to screening
• Use of an anticoagulant and/or thrombolytics
• Active internal bleeding
• Any major bleeding episode within the past 30 days, or diagnosis of chronic bleeding conditions
• Known gastrointestinal ulcer
• Severe, uncontrolled hypertension, renal impairment requiring dialysis, or liver impairment, or active infection indicated by sepsis
• Life expectancy less than 3 months independent of TMA disorder
• Pregnant or breastfeeding