SIG-001 / Eli Lilly - LARVOL DELTA

Preclinical pharmacokinetic modeling and clinical translation of SIG001, a novel anti-cancer therapeutic antibody targeting on cancer-derived sialylated IgG. (PubMed, Front Pharmacol) - "The preclinical investigations delineate the PK profile and antitumor activity of SIG001. The quantitative translation of animal data into human PK and exposure-response expectations provides a rational foundation for clinical development and supports informed decision-making in the design of early-phase trials."

First-in-human • Journal • PK/PD data • Preclinical • Oncology

A single-arm, open-label, dose-escalating and expansion Phase I clinical study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of SIG001 in patients with advanced solid tumors (ChiCTR) - P1 | N=66 | Sponsor: Peking University Cancer Hospital; Guangzhou Aisaiji Biomedical Technology Co., Ltd.

New P1 trial • Oncology • Solid Tumor

The Phase I Study of SIG001 Antibody on Cancer Therapy. (clinicaltrials.gov) - P1 | N=66 | Not yet recruiting | Sponsor: Peking University

New P1 trial • Breast Cancer • Oncology • Solid Tumor

[VIRTUAL] First-in-Human Phase 1/2 Clinical Trial of SIG-001, an Innovative Shielded Cell Therapy Platform, for Hemophilia Α (ASH 2020) - "SIG-001 produces hFVIII with in vitro and in vivo functionality. SIG-001-121, first-in-human clinical trial of SIG-001 in HA, is targeted to open in 2020."

Clinical • P1/2 data • Fibrosis • Hematological Disorders • Hemophilia • Rare Diseases • Rheumatology

Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A (clinicaltrials.gov) - P1/2 | N=3 | Terminated | Sponsor: Sigilon Therapeutics, Inc. | N=18 ➔ 3 | Trial completion date: Sep 2026 ➔ Oct 2022 | Suspended ➔ Terminated | Trial primary completion date: Sep 2026 ➔ Oct 2022; SIG-001 programme terminated

Enrollment change • Trial completion date • Trial primary completion date • Trial termination • Hematological Disorders • Hemophilia • Rare Diseases

Sigilon Therapeutics Announces Strategic Reprioritization (Businesswire) - "Sigilon Therapeutics, Inc...today announced a strategic reprioritization to enable the Company to focus on MPS-1 and diabetes....'There have been key learnings in our Phase 1/2 trial of SIG-001 for Hemophilia A. While we continue to investigate the findings from our SIG-001 study to help inform our development of the platform, following a review of our programs, we have made the strategic decision to refocus our pipeline. We will be prioritizing MPS-1—a rare lysosomal disease—with our product candidate..."

Trial status • Genetic Disorders • Hemophilia

Sigilon Therapeutics Announces Update on SIG-001 Phase 1/2 Study in Hemophilia A (GlobeNewswire) - "Sigilon Therapeutics, Inc...today reported that fibrosed spheres were observed during a retrieval procedure in a patient in its Phase 1/2 study of SIG-001 in severe or moderately severe hemophilia A....This patient underwent a laparoscopic procedure prescribed by the investigator to retrieve implanted spheres. Upon inspection, it was determined that the spheres placed in the patient had fibrosed and that cells within the spheres were no longer viable. While the Company investigates the fibrosed spheres in this patient, all three patients enrolled in the SIG-001 trial will continue to be followed per study protocol....The status of Sigilon’s clinical hold investigation, including these findings, will be reviewed by the Safety Review Committee for SIG-001 at its next meeting in December."

P1/2 data • Trial status • Genetic Disorders • Hemophilia

Sigilon Therapeutics Reports Third Quarter 2021 Financial Results and Business Highlights (GlobeNewswire) - "Company expects to complete its investigation of the clinical hold on its Phase 1/2 trial of SIG-001 in hemophilia A by year-end...Sigilon Therapeutics, Inc...today reported financial results for the third quarter ended September 30, 2021 as well as certain other business highlights....The Company expects to provide up to 12 months of follow-up data for three patients from the Phase 1/2 safety and dose-ranging study of SIG-001 in severe to moderate-severe hemophilia A in the fourth quarter of 2021."

P1/2 data • Genetic Disorders • Hemophilia

Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A (clinicaltrials.gov) - P1/2; N=18; Suspended; Sponsor: Sigilon Therapeutics, Inc.; Recruiting ➔ Suspended

Clinical • Trial suspension • Hematological Disorders • Hemophilia • Rare Diseases

Sigilon Therapeutics Announces Clinical Hold on SIG-001 Phase 1/2 Study in Hemophilia A (GlobeNewswire) - "Sigilon Therapeutics...today reported that the U.S. Food and Drug Administration (FDA) has notified the Company that its Phase 1/2 study of SIG-001 in patients with severe or moderately severe hemophilia A, has been placed on clinical hold. The clinical hold was initiated following the Company’s submission of a serious adverse event (SAE) and temporary enrollment halt to the FDA and other regulatory agencies....The status of the SAE investigation will continue to be reviewed by the Safety Review Committee for SIG-001 and Sigilon will provide additional data when available."

Trial suspension • Hemophilia

Sigilon Therapeutics Reports First Quarter 2021 Financial Results and Business Highlights (GlobeNewswire) - "The Phase 1/2 safety and dose-ranging study of SIG-001 in severe to moderate-severe hemophilia A remains ongoing, with sites initiated in the United Kingdom and United States....Research and development expenses were $16.0 million for the first quarter of 2021...Stock-based compensation expense increased to $0.8 million from $0.2 million for the three months ended March 31, 2021 and 2020, respectively. These increases were partially offset by a reduction of $0.7 million associated with our SIG-001 due to the timing of manufacturing activities in the first quarter of 2020."

Commercial • Trial status • Hemophilia

[VIRTUAL] A Scaled and Semi-Automated Cell Encapsulation Process for a Shielded Cell-Based Platform for Chronic Diseases (ASGCT 2021) - P1/2 | "The increased sphere formation rate achievable in this system allowed an order of magnitude increase in the volume of spheres that can be produced while maintaining a short residence time in the crosslinking bath, without impact to sphere strength, viability, or potency. The semi-automated manufacturing method described above is being used for the ongoing first-in-human clinical trial of SIG-001 in hemophilia A. There is continuing work to further scale and automate the manufacturing process for future manufacturing for the hemophilia A program, and for our pipeline programs in other rare blood disorders, lysosomal storage disorders, type 1 diabetes, and other serious chronic illnesses."

Diabetes • Fibrosis • Hematological Disorders • Hemophilia • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases • Type 1 Diabetes Mellitus

Sigilon Therapeutics Reports Fourth Quarter and Full Year 2020 Financial Results and Business Highlights (GlobeNewswire) - "Anticipated Milestones: Phase 1/2 safety and dose-ranging study of SIG-001 in severe to moderate-severe hemophilia: Sigilon expects to disclose up to 9 months of follow up data for 3-4 patients in the third quarter of 2021; and...Sigilon expects to complete enrollment of the study in the second half of 2021."

Enrollment status • P1/2 data • Hemophilia

[VIRTUAL] ASH Poster Walk on Clinical Trials in Progress: Live Q&A (ASH 2020) - "The following abstracts will be featured during this session: Adore: A Randomized, Open-Label, Phase 1/2 Open-Platform Study Evaluating Safety and Efficacy of Novel Ruxolitinib Combinations in Patients with Myelofibrosis, Andrew Perkins First-in-Human Phase 1/2 Clinical Trial of SIG-001, an Innovative Shielded Cell Therapy Platform, for Hemophilia Α, Pasi An Adaptive, Randomized, Placebo-Controlled, Double-Blind, Multi-Center Study of Oral FT-4202, a Pyruvate Kinase Activator in Patients with Sickle Cell Disease (PRAISE), Kenneth Wood Fostamatinib for the Treatment of Warm Antibody Autoimmune Hemolytic Anemia (wAIHA): A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Global Study, Nichola Coper Phase 1 and Dose Expansion Study of APR-246 in Combination with Ibrutinib or Venetoclax-Based Therapy in Subjects with TP53-Mutant Relapsed and/or Refractory Non-Hodgkin Lymphomas (NHL) Including Chronic Lymphocytic Leukemia (CLL) and Mantle Cell Lymphoma (MCL), Meghan..."

Clinical • Acute Myelogenous Leukemia • Chronic Lymphocytic Leukemia • Complement-mediated Rare Disorders • Genetic Disorders • Hematological Disorders • Hematological Malignancies • Hemophilia • Leukemia • Lymphoma • Mantle Cell Lymphoma • Myelofibrosis • Non-Hodgkin’s Lymphoma • Oncology • Rare Diseases • Sickle Cell Disease • CD123 • TP53

IPO Preview: Sigilon Therapeutics Commences U.S. IPO Effort (The Street) - "Sigilon Therapeutics (SGTX) intends to raise $100 million in an IPO of its common stock, according to an S-1 registration statement....Management says it will use the net proceeds from the IPO as follows: to progress our Phase 1/2 clinical trial and ongoing development for SIG-001 for treatment of Hemophilia A; to continue to scale our GMP manufacturing processes for our lead candidates SIG-001..."

Financing • Genetic Disorders • Hemophilia

Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A (clinicaltrials.gov) - P1/2; N=18; Recruiting; Sponsor: Sigilon Therapeutics, Inc.

Clinical • New P1/2 trial • Hematological Disorders • Hemophilia • Rare Diseases

[VIRTUAL] SIG-001 for Hemophilia A: Preclinical Safety and Efficacy Studies Conducted in Mice and Non-Human Primates (ISTH 2020) - "These safety studies, combined with the in vivo efficacy data reported previously, indicate that SIG-001 has a clear safety/toxicology profile and results in sustained, long-lasting FVIII levels. The first-in-human trial of SIG-001 is planned to open in Q2-2020."

Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases