A Phase 1 Study of ATORM-C in Patients With Crohn's Disease

This is an open-label, sequential dose-escalation Phase 1 study to evaluate the safety, tolerability, and exploratory efficacy of ATORM-C across three dose levels in patients with Crohn's disease. ATORM-C is manufactured by culturing and expanding autologous adult stem cell-derived intestinal organoids isolated from each participant's intestinal tissue. Participants will receive a single administration of ATORM-C directly applied to a target intestinal ulcer during colonoscopy. Primary evaluations will focus on safety and tolerability, with exploratory assessments of ATORM-C's effect on intestinal ulcer healing.

Inclusion criteria

• Aged 19 years or older and younger than 80 years at the time of screening (male or female).
• Diagnosed with Crohn's disease at least 6 months prior to screening, confirmed by imaging, tissue sampling (biopsy), and/or colonoscopy.
• Have had an inadequate response to at least 6 months of standard treatment for Crohn's disease (such as anti-inflammatory drugs, oral corticosteroids, immunosuppressants, or biologic agents). Inadequate response means failure to achieve ulcer-free endoscopy, defined as an SES-CD Ulcerated Surface Score of 0 or 1.
• Have active daily symptoms at screening and right before treatment, defined as either an average of 4 or more loose/liquid stools per day or an average daily abdominal pain score of 2 or more, based on the corresponding CDAI components.
• Meet all of the following colonoscopy findings at screening:

• Have at least one active ulcer in an accessible area of the intestine (such as the colon or ileocecal region).
• Have at least one ulcer with a longest diameter of 5 mm or larger.
• Have an active ulcer segment with an endoscopic severity score (SES-CD) of 6 or higher, with an ulcer size score of 2 or higher and no narrowing or blockage (stricture score of 0) in that area.
• Meet all required blood test standards for cell therapy donor eligibility.
• Voluntarily agree to participate and sign the written informed consent form after receiving a full explanation of the study.

Exclusion criteria

• Other Inflammatory Bowel Conditions: Diagnosed with ulcerative colitis or unclassified colitis.
• Surgical Needs: Require, or are expected to require, surgery for Crohn's disease or its complications.
• Severe Co-existing Conditions: Have a severe autoimmune disease other than Crohn's disease, or an active abdominal/perianal condition (such as an undrained abscess or active fistula).
• Recent Surgeries: Had bowel resection within 6 months or intra-abdominal surgery within 3 months prior to screening.
• Procedure Inability: Unable to safely undergo bowel preparation, colonoscopy, or sedation, or have a known allergy/contraindication to medications used during these procedures.
• Uncontrolled Medical Issues: Have a clinically significant or poorly controlled medical condition, such as severe heart, bleeding, kidney, or liver disease, or uncontrolled diabetes.
• Active Severe Infection: Have an active, severe infection requiring ongoing medical treatment.
• Cancer History: Have a history of cancer (malignancy) within 5 years before screening, except for specific low-risk cancers permitted by the protocol.
• Allergies to Study Treatment: Have a known severe allergy (hypersensitivity) to ATORM-C, its ingredients, or materials used in its manufacturing.
• Prohibited Therapies or Transplants: Are taking prohibited medications, have previously received any cell therapy product, or have received an organ transplant (except corneal transplant).
• Significant Blood Test Abnormalities: Have severe laboratory test abnormalities, including liver function (AST or ALT ≥3 times the upper limit of normal), kidney function (eGFR <30 mL/min/1.73 m²), bleeding markers (PT INR >1.5), or low blood platelets (<50,000/mm³).
• Pregnancy, Breastfeeding, or Contraception: Are pregnant, breastfeeding, or unwilling to follow the protocol-specified contraception requirements during the study.
• Recent Clinical Trials: Received another investigational drug or device within 4 weeks prior to screening.
• Investigator Judgment: Deemed unsuitable for participation by the study investigator for any other safety reason.