Ancain (omfiloctocog alfa) / Sinocelltech - LARVOL DELTA

Evaluation of Omfiloctocog alfa (SCT800) Efficacy in Real-World Treatment of Severe or Moderate Hemophilia A in China: Final Analysis from the SURPASS Study (EAHAD 2025) - No abstract available

Clinical • HEOR • Real-world • Real-world evidence • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Evaluating the Stability and Efficacy of SCT800 in Perioperative Management of Orthopedic Procedures for Patients with Hemophilia A: A Preliminary Analysis (EAHAD 2025) - No abstract available

Clinical • Hematological Disorders • Hemophilia • Hemophilia A • Orthopedics • Rare Diseases

The lower double negative T cells proportion predicted the better high-titer inhibitors eradication outcomes of immune tolerance induction (ISTH 2024) - " From January 2022 to September 2023, a prospective single-center cohort study was conducted on SHA children age ≤8 years, with peak historical inhibitor-titer 5 – 200 Bethesda Units (BU)/mL, received ITI treatment [Factor (F)VIII 50-100IU/kg every other day-every day] using recombinant FVIII (SCT800)... Total 27 patients were enrolled, including 19 (70.4%) in success (S)-group, and rest in non-success (NS)-group. There was no difference in the clinical baseline variables and ITI regimens between S-group and NS-group. As shown in table 1, only the proportion of T cell receptor (TCR)-αβ+ CD4− CD8− double negative T cells (DNTs) in total lymphocyte was lower in the S-group compared to that in the NS-group (1.6% vs."

Hematological Disorders • Hemophilia • Rare Diseases • CD4 • CD8

Stability of Omfiloctocog alfa (SCT800) for Continuous Infusion in Hemophilia A Treatment (ISTH 2024) - "Stability experiments revealed that the reconstituted product remains stable for at least 48 hours when stored at room temperature or even at 37℃, enabling continuous administration (Fig. 1). A patient undergoing continuous infusion post-surgery had the diluted SCT800 solution syringe changed every 6 hours, maintaining steady FVIII levels as depicted in Figure 2."

Hematological Disorders • Hemophilia • Rare Diseases

Evaluation of Omfiloctocog alfa (SCT800) Efficacy in Real-World Treatment of Severe Hemophilia A in China: Insights from the SURPASS Study (ISTH 2024) - "The interim analysis incorporated data from 295 enrolled patients, including 15 children and 56 adolescents. Before SCT800, 45.7% of patients received on-demand therapy, while the remainder had irregular or low-dose prophylaxis. Post-switch, all patients were prescribed SCT800 regular prophylaxis."

Clinical • HEOR • Real-world • Real-world evidence • Hematological Disorders • Hemophilia • Rare Diseases

ITI Using SCT800 Alone or Combining Daratumumab in Hemophilia A Adolescents and Adults With High Titer Inhibitor (clinicaltrials.gov) - P4 | N=50 | Recruiting | Sponsor: Institute of Hematology & Blood Diseases Hospital, China | Initiation date: Aug 2023 ➔ Nov 2023

Trial initiation date • Hematological Disorders • Hemophilia • Rare Diseases

Chinese Severe Hemophilia a Children with High-Titer Inhibitors Obtain More Benefit from Intermediate-Dose Immune Tolerance Induction: The Interim Result of Lome Part a (ASH 2023) - "ObjectivesConduct the RCT to compare the efficacy, safety and cost of LD- to MD-ITI strategy using rFVIII (Omfiloctocog alfa, SCT800) for SHA children with HTI in China...This included the cost of rFVIII and rituximab for ITI, PCC and rFVIIa for treatment of breakthrough bleeding, and IVIG for infection prevention in IS patients...On the premise of similar cost, shorter time to success, less bleedings were observed in MD-ITI strategy. PICC implantation is a safe option to conduct ITI."

Clinical • Cardiovascular • Hematological Disorders • Hemophilia • Infectious Disease • Rare Diseases • Thrombosis

ITI Using SCT800 Alone or Combining Daratumumab in Hemophilia A Adolescents and Adults With High Titer Inhibitor (clinicaltrials.gov) - P4 | N=50 | Recruiting | Sponsor: Institute of Hematology & Blood Diseases Hospital | Not yet recruiting ➔ Recruiting

Enrollment open • Hematological Disorders • Hemophilia • Rare Diseases

LOME PILOT STUDY: A MULTICENTER CONTROLLED RANDOMIZED CLINICAL TRIAL OF LOW- VERSUS INTERMEDIATE-DOSE IMMUNE TOLERANCE INDUCTION WITH RECOMBINANT FACTOR VIII (OMFILOCTOCOG ALFA, SCT800) IN CHINA (EHA 2023) - "Compared with LD-ITI (with rFVIII), SHA children with high-titer inhibitors with MD-ITI (with rFVIII) obtained success/partial success more quickly and had less breakthrough bleedings within ITI period. The comparison of ITI success/partial success rates between MD-ITI and LD-ITI groups required longer ITI treatment time to evaluate. Hemophilia A"

Clinical • Hematological Disorders • Hemophilia • Rare Diseases

ITI Using SCT800 Combining Daratumumab or SCT800 Alone in Hemophilia A Adolescents and Adults With High Titer Inhibitor (clinicaltrials.gov) - P4 | N=50 | Not yet recruiting | Sponsor: Institute of Hematology & Blood Diseases Hospital

New P4 trial • Hematological Disorders • Hemophilia • Rare Diseases

LONG TERM SAFETY AND EFFICACY OF RECOMBINANT HUMAN COAGULATION FACTOR VIII (SCT800) IN PREVIOUSLY TREATED PATIENTS WITH SEVERE HEMOPHILIA A (EHA 2022) - P3, P4 | "Improved joint function was observed in 67 patients with a mean total HJHS scores decreased by 3.4. Conclusion The interim data demonstrate favorable long-term safety of SCT800 and the maintenance of low ABR in patients with severe hemophilia A."

Clinical • Hematological Disorders • Hemophilia • Rare Diseases

BASELINE ANALYSIS OF SURPASS STUDY, A PROSPECTIVE, NON-INTERVENTIONAL, MULTI-CENTER STUDY ON REAL-WORLD EFFECTIVENESS AND USAGE OF RECOMBINANT FACTOR VIII (SCT800, OMFILOCTOCOG ALFA) IN HEMOPHILIA A (EAHAD 2023) - "Interim baseline data from the ongoing SURPASS study provides insights into the demographics and clinical characteristics of HA patients treated with SCT800. There were many patients switching from pdFVIII and beginning prophylaxis treatment. The majority of patients initiated SCT800 on a regimen three times a week or QoD, which may indicate that adolescent and adult patients still had a strong willingness to get better outcomes."

Clinical • Real-world • Real-world effectiveness • Real-world evidence • Hematological Disorders • Hemophilia • Rare Diseases

A SINGLE-CENTER, RETROSPECTIVE, OBSERVATIONAL STUDY OF THE FIRST CHINESE RFVIII (SCT800, OMFILOCTOCOG ALFA) IN REAL-WORLD PRACTICE (EAHAD 2023) - "This retrospective analysis shows that many patients chose SCT800 for its affordability and accessibility, and more of them could receive prophylaxis and the factor consumption increased dramatically. Treatment options for HA have never been better with the approval of these new recombinant FVIII products that are affordable and safe. Additional factor products still in development will add to the choices that we will have in the treatment of HA patients."

Observational data • Real-world • Real-world evidence • Retrospective data • Hematological Disorders • Hemophilia • Rare Diseases

EFFICACY AND SAFETY EVALUATION OF RECOMBINANT HUMAN COAGULATION FACTOR VIII (OMFILOCTOCOG ALFA, SCT800) DURING ORTHOPEDIC PROCEDURES IN SEVERE HEMOPHILIA A PATIENTS: A SINGLE CENTER RETROSPECTIVE STUDY (EAHAD 2023) - "The data indicate that SCT800 is effective and with a favorable safety profile for perioperative management of orthopedic procedures in patients Hemophilia A."

Retrospective data • Hematological Disorders • Hemophilia • Musculoskeletal Diseases • Musculoskeletal Pain • Oncology • Orthopedics • Pain • Rare Diseases • Rheumatology

DESIGN OF A RANDOMIZED, CONTROLLED TRIAL OF CONTINUOUS VERSUS BOLUS INFUSIONS UNDER PREOPERATIVE PHARMACOKINETIC-GUIDED BY RECOMBINANT HUMAN COAGULATION FACTOR VIII (OMFILOCTOCOG ALFA, SCT800) IN HAEMOPHILIA A (EAHAD 2023) - "N/A"

Clinical • PK/PD data • Hematological Disorders • Hemophilia • Musculoskeletal Diseases • Orthopedics • Rare Diseases

PHASED ANALYSIS OF LOME: A RANDOMIZED TRIAL OF LOW- VERSUS INTERMEDIATE-DOSE IMMUNE TOLERANCE INDUCTION WITH RECOMBINANT FACTOR VIII (OMFILOCTOCOG ALFA, SCT800) IN CHINA (EAHAD 2023) - "Compared with LD group, patients in MD group tended to achieved ph1- or ph2-success faster. Because of relatively short duration of ITI, we couldn't compare the success rate between LD group and MD group now. The study is ongoing."

Clinical • Hematological Disorders • Hemophilia • Rare Diseases

Protocol and Primary Analysis of Lome: A Randomized Trial of Low- Versus Intermediate-Dose Immune Tolerance Induction with Recombinant Factor VIII (Omfiloctocog alfa, SCT800) in China (ASH 2022) - "If (a) there is an upward trend in titer over 3-6 months period; or (b) fall in chromogenic Bethesda titer of less than 20% in 6 months period after initial peak inhibitor titer; or (c) titer ≥ 200BU/ml during ITI therapy; or (d) spontaneous bleeding > 1 monthly over 3-6 months period, modify the regimen as below: In the LD arm, increase to 100IU/kg daily; In the MD arm, rituximab 375 mg/m2 weekly × 4 weeks (maximum 600 mg) and prednisone 2 mg/kg daily × 1 month (maximum 60 mg, tapering over 3 months) would be added. It is planned to enroll 40 patients in Part A and 60 patients in Part B, who will be followed for up to 2 years. The study is ongoing and, currently, 10/40 (25%, in Part A) PwHI from 4 HTCs have been included. LOME study is the first, multicenter, prospective, randomized trial of LD vs MD ITI with domestic rFVIII (Omfiloctocog alfa, SCT800) in Chinese PwHI."

Clinical • Hematological Disorders • Hemophilia • Infectious Disease • Rare Diseases

Efficacy, Safety and Pharmacokinetics of Recombinant Human Coagulation Factor VIII (SCT800) in Previously Treated Children with Severe Hemophilia A. (ISTH 2022) - "Sixty-eight (68) patients were enrolled (34 each for age group of 0-5yr and 6-11) and the mean exposure to SCT800 was 78.9 days. The median ABRs of the two groups were similar, 2.21 vs 2.17(0-5yr vs 6-11yr. The median ABRs for spontaneous bleeds were 0.00 for both groups."

Clinical • PK/PD data • Hematological Disorders • Hemophilia • Pediatrics • Rare Diseases

Efficacy, safety and pharmacokinetics of recombinant human coagulation factor VIII (omfiloctocog alfa) in previously treated Chinese children with severe hemophilia A. (PubMed, Haemophilia) - "Omfiloctocog alfa was efficacious and well tolerated for the prevention and treatment of bleeding in Chinese pediatric PTPs with severe hemophilia A."

Journal • PK/PD data • Hematological Disorders • Hemophilia • Pediatrics • Rare Diseases

DESIGN OF A REAL-WORLD STUDY TO EVALUATE THE EFFECTIVENESS AND SAFETY OF OMFILOCTOCOG ALFA IN PATIENTS RECEIVING PROPHYLACTIC TREATMENT FOR MODERATE OR SEVERE HEMOPHILIA A (EAHAD 2022) - "This real-world study will provide additional long-term effectiveness, safety and health economics data on prophylactic Omfiloctocog alfa in a large number of Chinese patients. These real-world date may be helpful in the goal of achieving universal access to prophylactic treatment in China."

Clinical • Real-world evidence • Hematological Disorders • Hemophilia • Rare Diseases • Rheumatology

DESIGN OF A REAL-WOLRD STUDY TO EVALUATE THE EFFECTIVENESS AND SAFETY OF OMFILOCTOCOG ALFA IN PATIENTS RECEIVING PROPHYLACTIC TREATMENT FOR MODERATE OR SEVERE HEMOPHILIA A (EAHAD 2022) - "This real-world study will provide additional long-term effectiveness, safety and health economics data on prophylactic Omfiloctocog alfa in a large number of Chinese patients. These real-world date may be helpful in the goal of achieving universal access to prophylactic treatment in China."

Clinical • Hematological Disorders • Hemophilia • Rare Diseases • Rheumatology

Beijing Shenzhou Cell Biotechnology Group Co., Ltd. Voluntary Disclosure Announcement on Acceptance of Supplementary Application for Recombinant Human Coagulation Factor VIII for New Children's Indications [Google translation] (Sina Corp) - "Recently, China Cell Engineering Co., Ltd...a holding subsidiary of Beijing China Cell Biotechnology Group Co., Ltd. The 'Notice of Acceptance' issued by the supplementary application for the proposed new pediatric indications for the company's product recombinant human coagulation factor VIII for injection (trade name: Ancain®, product code: SCT800) has been accepted by the State Food and Drug Administration."

Non-US regulatory • Genetic Disorders • Hemophilia

Guojin Securities: Maintain the “Buy” rating of Shenzhou Cell (688520.SH) and get the approval. The first fixed increase on the Science and Technology Innovation Board helps the commercialization and internationalization process [Google translation] (jrj.com) - "Approved for the first fixed increase on the Science and Technology Innovation Board, the 2.2 billion new drug R&D fundraising plan will help the company's many products to accelerate the commercialization and internationalization process. According to the company’s fixed-increased fundraising prospectus, all of the 2.2 billion raised in this fixed-increasing financing will be used to supplement the cost of new drug research and development projects; the total investment of the project is nearly 2.5 billion, and about 450 million will be used for preclinical research such as virus vaccines, about 20.5 Yijiang will invest in SCT800..."

Financing • Genetic Disorders • Hemophilia

The first domestic recombinant coagulation factor Ⅷ product was approved for the control and prevention of bleeding in adolescents and adults with hemophilia A [Google translation] (Eastmoney.com) - "A few days ago, the official website of the National Food and Drug Administration (NMPA) announced that recombinant human coagulation factor VIII (SCT800) for Shenzhou cell injection has been officially approved. The approved indication for this product is: suitable for adolescents and adults with hemophilia A (Control and prevention of bleeding in patients with congenital factor VIII deficiency). At present, SCT800 has completed the Phase 3 on-demand treatment study for patients with hemophilia A ≥ 12 years old, and is currently conducting a Phase 3 preventive treatment study for patients with hemophilia A ≥ 12 years old. It is also targeting 3 patients with hemophilia A who are younger than 12 years old."

Non-US regulatory • Genetic Disorders • Hemophilia

Pharmacokinetic, efficacy and safety evaluation of B-domain-deleted recombinant FVIII (SCT800) for prophylactic treatment in adolescent and adult patients with severe haemophilia A. (PubMed, Haemophilia) - "SCT800 has robust PK characteristics, and is safe and efficacious for the prophylaxis and treatment of bleeding episodes in previously treated adolescent and adult patients with severe haemophilia A."

Clinical • Journal • PK/PD data • Hematological Disorders • Hemophilia • Rare Diseases