Intravenous Immunoglobulin (IVIG) in the Treatment of Small Fiber Neuropathy Due to TS-HDS, FGFR-3, or Plexin D1 Antibodies: a Double Blinded Placebo-controlled Phase II Trial

This study will enroll patients with small fiber neuropathy (SFN). The study will look at an intravenous immunoglobulin (IVIG) called Panzyga. Panzyga is approved by the FDA as a therapy for Primary humoral immunodeficiency (PI) in patients 2 years of age and older; Chronic immune thrombocytopenia (ITP) in adults and Chronic inflammatory demyelinating polyneuropathy (CIDP) in adults. It has not been approved by the FDA for use in SFN.
There is mounting evidence that Intravenous Immunoglobulin (IVIG) can cause pain reduction and improve objective nerve fiber densities on skin biopsies in great numbers in SFN patients. The primary outcome is quantified improvement in intraepidermal nerve fiber density (IENFD) on repeat skin punch biopsy after 6 months of IVIG treatment.

Start Date15 Dec 2023

Sponsor / Collaborator

Henry Ford Health System

[+1]

NCT04929236 / RecruitingPhase 3

Multicenter, Prospective, Double-Blinded, Parallel Group, Randomized Phase III Study to Evaluate Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients

Safety and Efficacy of Different PANZYGA Dose Regimens in Pediatric Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Patients

Start Date01 Oct 2023

Sponsor / Collaborator

Octapharma AG

NCT04354129 / RecruitingNot ApplicableIIT

Safety, Tolerability, and Patient Satisfaction of 16.5% Subcutaneous Immunoglobulin (Cutaquig®) Treatment by Rapid Push in Patients With Primary or Secondary Immunodeficiency (PID or SID)

This study will assess the relative safety, tolerability, and participant satisfaction in participants using the rapid manual push method with Cutaquig®. The hypothesis being that treatment with Cutaquig® by rapid manual push method will improve the safety, tolerability and patient satisfaction of participants with PID or SID.
Cutaquig® by rapid push is already approved in Canada and has proven to be efficacious in preventing significant infection. However, relative safety, tolerability, and patient satisfaction have not been studied in these patients. The information gained from this study will improve the safety and tolerability knowledge database and will support the optimal use of Cutaquig® - thus benefitting both physicians and patients.

Start Date01 Apr 2023

Sponsor / Collaborator

University of Alberta

[+1]

100 Clinical Results associated with Immune Globulin Intravenous (Human) (Octapharma)

100 Translational Medicine associated with Immune Globulin Intravenous (Human) (Octapharma)

100 Patents (Medical) associated with Immune Globulin Intravenous (Human) (Octapharma)

Literatures (Medical) associated with Immune Globulin Intravenous (Human) (Octapharma)

01 Oct 2023·EClinicalMedicine

Efficacy of intravenous immunoglobulins (IVIg) in improving skin symptoms in patients with dermatomyositis: a post-hoc analysis of the ProDERM study

Article

Author: Kopasz, Norbert ; Charles-Schoeman, Christina ; Schessl, Joachim ; Aggarwal, Rohit ; Levine, Todd ; Werth, Victoria P ; Bata-Csörgő, Zsuzsanna ; Worm, Margitta

Background:

Dermatomyositis (DM) is a rare autoimmune disease characterized by skin involvement, with or without proximal muscle weakness. Recently, following the ProDERM study, intravenous immunoglobulin (IVIg) was approved for treatment of DM. Until ProDERM evidence from large, placebo-controlled studies supporting its use for dermatological symptoms, was lacking. Here we present efficacy data from ProDERM of IVIg versus placebo for treatment of the cutaneous aspect of DM.

Methods:

ProDERM was a double-blind, randomized, multicenter, Phase 3 study. In the First Period (Weeks 0-16), adults with active DM received 2.0 g/kg IVIg (Octagam 10%; Octapharma AG) or placebo every 4 weeks. In the open-label Extension Period (Weeks 16-40), all patients received IVIg for 6 additional cycles. Cutaneous disease was assessed using measures including modified cutaneous DM disease area and severity index activity (CDASI-A) and damage (CDASI-D) scores, and myositis disease activity assessment tool (MDAAT) including visual analogue scale (VAS). This trial is registered with ClinicalTrials.gov, NCT02728752.

Findings:

The study took place from February 2017 to November 2019. 95 patients received IVIg (N = 47) or placebo (N = 48) in the First Period. Together, 664 IVIg infusion cycles were administered (median dose, 2.0 g/kg). At Week 16, mean CDASI-A change from baseline was -9.36 (95% CI: -12.52, -6.19) in the IVIg group versus -1.16 (-3.32, 0.99) in placebo group (p < 0.0001). At the end of the Extension Period, mean changes from baseline were -10.44 (95% CI: -13.94, -6.94) and -10.03 (-13.12, -6.94), respectively. Similar changes were seen for CDASI-D and VAS of MDAAT. These observations were seen regardless of baseline disease severity.

Interpretation:

ProDERM is the first large prospective, randomized trial to demonstrate the efficacy of IVIg to improve the cutaneous manifestations of DM. IVIg treatment significantly improved dermatological symptoms in patients with DM, regardless of disease severity before treatment, suggesting that IVIg is effective for even the most severe cutaneous DM.

Funding:

This study was sponsored by Octapharma Pharmazeutika Produktionsges m.b.H.

01 Sep 2023·Drug safety

Safety and Tolerability of Intravenous Immunoglobulin in Chronic Inflammatory Demyelinating Polyneuropathy: Results of the ProCID Study.

Article

Author: Cornblath, David R ; Clodi, Elisabeth ; Merkies, Ingemar S J ; Hinterberger, Doris ; van Doorn, Pieter A ; Katzberg, Hans D ; Hartung, Hans-Peter

BACKGROUND AND AIMS:

The ProCID study evaluated the efficacy and safety of three doses of a 10% liquid intravenous immunoglobulin (IVIg) preparation (panzyga^®) in patients with chronic inflammatory demyelinating polyneuropathy (CIDP). This report describes the safety findings.

METHODS:

Patients were randomised to receive a 2.0 g/kg induction dose followed by maintenance doses of either 0.5, 1.0 or 2.0 g/kg IVIg every 3 weeks over 24 weeks.

RESULTS:

All 142 enrolled patients were included in the safety analyses. In total, 286 treatment-emergent adverse events (TEAEs) were reported in 89 patients, of which 173 (60.5%) were considered treatment-related. Most TEAEs were of mild severity. Eleven serious TEAEs were reported in 6 patients. Two serious TEAEs in one patient (headache and vomiting) were considered related to treatment, which resolved without study discontinuation. No treatment-related thrombotic events, haemolytic transfusion reactions or deaths occurred. One patient discontinued the study due to a TEAE (allergic dermatitis) probably related to IVIg. Headache was the only dose-dependent TEAE, with incidences ranging from 2.9 to 23.7%, the incidence of all other TEAEs was similar across treatment groups. Most TEAEs were associated with the induction dose infusion, and the rate of TEAEs decreased thereafter. The median (IQR) daily IVIg dose was 78 (64-90) g, and 94.4% of patients tolerated the maximal infusion rate of 0.12 ml/kg/min without pre-medication.

INTERPRETATION:

Infusions of 10% IVIg at doses up to 2.0 g/kg with high infusion rates were safe and well tolerated in patients with CIDP.

CLINICAL TRIAL NUMBERS:

EudraCT 2015-005443-14, NCT02638207.

01 Aug 2023·Journal of clinical immunology

Subcutaneous Immunoglobulin 16.5% (Cutaquig®) in Primary Immunodeficiency Disease: Safety, Tolerability, Efficacy, and Patient Experience with Enhanced Infusion Regimens.

Article

Author: Koterba, Alan ; Bridges, Tracy ; Manning, Michael ; Hoeller, Sonja ; Turpel-Kantor, Eva ; Walter, Jolan E. ; DeAngelo, James ; Gupta, Sudhir ; Bernstein, Jonathan A. ; Kreuwel, Huub ; Kobayashi, Roger H. ; Kobayashi, Ai-Lan ; Maltese, Joanna ; Melamed, Isaac ; Sublett, J. Wesley

PURPOSE:

To achieve reductions in infusion time, infusion sites, and frequency, a prospective, open-label, multicenter, Phase 3 study evaluated the safety, efficacy, and tolerability of subcutaneous immunoglobulin (SCIG) 16.5% (Cutaquig®, Octapharma) at enhanced infusion regimens.

METHODS:

Three separate cohorts received SCIG 16.5% evaluating volume, rate, and frequency: Cohort 1) volume assessment/site: up to a maximum 100 mL/site; Cohort 2) infusion flow rate/site: up to a maximum of 100 mL/hr/site or the maximum flow rate achievable by the tubing; Cohort 3) infusion frequency: every other week at twice the patient's weekly dose.

RESULTS:

For Cohort 1 (n = 15), the maximum realized volume per site was 108 mL/site, exceeding the currently labeled (US) maximum (up to 40 mL/site for adults). In Cohort 2 (n = 15), the maximum realized infusion flow rate was 67.5 mL/hr/site which is also higher than the labeled (US) maximum (up to 52 mL/hr/site). In Cohort 3 (n = 34), the mean total trough levels for every other week dosing demonstrated equivalency to weekly dosing (p value = 0.0017). All regimens were well tolerated. There were no serious bacterial infections (SBIs). Most patients had mild (23.4%) or moderate (56.3%) adverse events. The majority of patients found the new infusion regimens to be better or somewhat better than their previous regimens and reported that switching to SCIG 16.5% was easy.

CONCLUSIONS:

SCIG 16.5% (Cutaquig®), infusions are efficacious, safe, and well tolerated with reduced infusion time, fewer infusion sites, and reduced frequency. Further, the majority of patients found the new infusion regimens to be better or somewhat better than their previous regimens.

News (Medical) associated with Immune Globulin Intravenous (Human) (Octapharma)

10 Jun 2024

·www.prnewswire.com

Octapharma's continuing dedication to improving the lives of people with rare bleeding disorders to be showcased at ISTH 2024

Octapharma to present new clinical and scientific findings for wilate® and Nuwiq® at the upcoming ISTH 2024 Congress New data and clinical study developments to be showcased in seven oral presentations, two posters and two Supported Symposia LACHEN, Switzerland, June 10, 2024 /PRNewswire/ -- The most recent developments from Octapharma's Haematology portfolio will be presented at the 32nd Congress of the International Society on Thrombosis and Haemostasis (ISTH) on June 22–26, 2024, in Bangkok, Thailand. The data will be featured in seven oral presentations, two poster presentations and two Supported Symposia during this key international meeting. Octapharma is a proud Silver Supporter of the ISTH 2024 Congress. Octapharma will also present the offer in modern solutions for acute bleeding and coagulation management in critical and intensive care. Bleeding disorders, such as haemophilia A and von Willebrand disease (VWD), carry the risk of recurrent and prolonged bleeds, which can not only be life-threatening but also take a heavy toll on patients' quality of life and mental health. The presentations at ISTH 2024 reflect Octapharma's long-term commitment to improving patient care and addressing unmet clinical needs. "Octapharma is excited to present our latest findings and study results from across our haematology and critical care portfolios at ISTH 2024." said Olaf Walter, Board Member at Octapharma. "Bleeding and coagulation disorder patients continue to face many challenges in their day-to-day care and bleeding management. Here at Octapharma we remain dedicated to frontline research towards improving patients' lives." Oral Presentations Although prophylaxis is the standard of care for severe haemophilia A, it remains underutilised in VWD. WIL-31, the largest study in VWD prophylaxis, demonstrated the safety and efficacy of prophylaxis with a plasma-derived von Willebrand factor/factor VIII concentrate (wilate®) in patients with VWD. The lead investigator, Dr Robert F. Sidonio Jr., will present sub-analyses of data from the study which explore differences in bleed sites across VWD types and age groups. Prophylaxis was well-tolerated across all age groups in the 22 VWD type 3 patients who were included in the analysis population. MOTIVATE is an ongoing, international, investigator-initiated study supported through funding from Octapharma AG, evaluating the management of haemophilia A patients with inhibitors. Dr Carmen Escuriola-Ettingshausen, one of the study's coordinating investigators, will present an interim analysis of the study. Haemophilic arthropathy is a serious complication of haemophilia A and early diagnosis is critical to minimise its long-term impact. A proof-of-concept study has identified a signature of several circulating small non-coding microRNAs, which may be used as biomarkers to assist in the diagnosis of haemophilic arthropathy. Future work will investigate microRNAs associated with earlier stages of the condition in a sub-study of the PROVE clinical trial. Altered microvascular endothelial functionality has been observed in haemophilia A patients. Indeed, impaired tubulogenesis, migration and permeability of endothelial cells from haemophilia A patients has been observed, compared to those from healthy patients. New data will be presented showing that Nuwiq® can bind to endothelial cell surfaces, improving their functionality. FVIII physically interacts with platelets following vascular injury to ensure efficient clot formation. Different FVIII modifications may impact platelet binding and downstream signalling. Data will be presented showing that Nuwiq® exhibits significantly higher binding to platelets compared to comparator products, and that these differences in platelet binding may impact the efficacy of different rFVIII products in the treatment of haemophilia A. The oral presentations are scheduled at the following times: Sunday June 23 OC 27.5: Impact of differential binding of recombinant factor VIII concentrates to platelets on platelet clotting functionality. Presenting author: Viola Vogel. 15:45–16:00 ICT, Room 210 A-D Monday June 24 OC 35.1: Factor VIII regulates extracellular matrix proteins to stimulate angiogenesis and vessel stability. Presenting author: Alessia Cucci. 9:30–9:45 ICT, Room 110 A-C OC 36.4: miRNA expression profiles as a potential biomarker of joint and bone health in haemophilia A. Presenting author: Yesim Dargaud. 10:15–10:30 ICT, Room 111 A-C OC 36.5: Factor VIII is a regulator of angiogenesis and a promoter of endothelial barrier stability. Presenting author: Antonia Follenzi. 10:30–10:45 ICT, Room 111 A-C Tuesday June 25 OC 63.4: Bleeding Sites in von Willebrand Disease Patients on Prophylaxis with a Plasma-derived von Willebrand Factor/Factor VIII Concentrate – A Sub-Analysis of Data from the WIL-31 Study. Presenting author: Robert F Sidonio Jr. 15:30–15:45 ICT, Ballroom B3 OC 61.4: Immune tolerance induction (ITI) strategies in haemophilia A patients with inhibitors – Interim analysis from the MOTIVATE study. Presenting author: Carmen Escuriola-Ettingshausen. 15:45–16:00 ICT, Ballroom B1 Wednesday June 26 OC 73.5: Efficacy and Safety of Prophylaxis with a Plasma-derived von Willebrand Factor/Factor VIII Concentrate in Type 3 Patients with von Willebrand Disease – A Sub-Analysis of Data from the WIL-31 Study. Presenting author: Robert F Sidonio Jr. 11:15–11:30 ICT, Ballroom B4 Poster Presentations Poster presentations are scheduled at the following times: Sunday June 23 PB 0251 - Indirect comparison of prophylaxis efficacy between simoctocog alfa and efanesoctocog alfa in severe hemophilia A and their cost in the United States. Presenting author: Craig M. Kessler. 13:45–14:45 ICT, Exhibition Hall Tuesday June 25 PB 1102 - Covalently linked complexes of coagulation factor VIII and von Willebrand factor fragment display increased stability and half-life in animal models and retain their function. Presenting author: Barbara Solecka-Witulska. 13:45–14:45 ICT, Exhibition Hall Supported Symposia Two Supported Symposia during the Congress will share new clinical and scientific data, providing updates on ongoing and new studies in the fields of haemophilia A and VWD. Learn About Tomorrow's Advances Today – Breakthrough in All-Round Bleed Protection Sunday June 23, 12:15–13:30 ICT, Room 208 Chair: Guy Young, Children's Hospital Los Angeles and Keck School of Medicine, Los Angeles, California, USA Octapharma's Symposium on haemophilia A this year is dedicated to cutting-edge research committed to bleed protection. Speakers will present the first results from the MOTIVATE study interim analysis and introduce the PROVE study investigating bone and joint health. This symposium will also explore the critical role of FVIII binding to platelets and endothelial cells in haemophilia A. Next Level Insights on Prophylaxis in von Willebrand Disease – Are You Addressing Your Patient's Needs? Monday June 24, 12:15–13:30 ICT, Room 210 Chair: Craig Kessler, Georgetown University Medical Center, Washington DC, USA This symposium will explore the critical role of prophylaxis in severe VWD. Topics presented will include the importance of prophylaxis and how to identify patients who may benefit from prophylactic treatment. Key lessons from the WIL-31 VWD prophylaxis study will be shared as well as the important role for prophylaxis in the management of heavy menstrual bleeding in women with VWD. Optimising care during pregnancy will also be discussed and the VIP and EMPOWER studies will be introduced. Both symposia will be open to congress participants in Bangkok and will be live streamed for participants not directly attending the Congress. An opportunity to ask questions will be available at both symposia. Larisa Belyanskaya, SVP and Head of IBU Haematology at Octapharma commented: "We are excited to present new insights from the WIL-31 data, and will take a deep dive into the importance of prophylaxis for specific patient groups, as well as discussing important clinical and scientific questions with the goal of improving the quality of life for people with von Willebrand disease and haemophilia A." About Nuwiq® Nuwiq® (simoctocog alfa) is a 4th generation recombinant factor VIII (rFVIII) protein, produced in a human cell line without chemical modification or fusion with any other protein1. It is cultured without additives of human or animal origin, is devoid of antigenic non-human protein epitopes and has a high affinity for von Willebrand factor1. Nuwiq® treatment has been assessed in nine1 2 3 completed clinical trials which included 201 previously treated patients (190 individuals)1 and 108 previously untreated patients2 with severe haemophilia A. Nuwiq® is available in 250 IU, 500 IU, 1,000 IU, 1,500 IU, 2,000 IU, 2,500 IU, 3,000 IU and 4,000 IU presentations4. Nuwiq® is approved for use in the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital FVIII deficiency) across all age groups4. About wilate® wilate® is a high-purity human von Willebrand factor/factor VIII (VWF/FVIII) concentrate, that undergoes two virus inactivation steps during its production1. No albumin is added as a stabiliser5. The purification processes result in a 1:1 ratio of VWF to FVIII that is similar to normal plasma1. wilate® contains a VWF triplet structure and content of large high molecular weight multimers similar to normal human plasma1. wilate® is exclusively derived from large pools of human plasma collected in approved plasma donation centres6. wilate® is available in 500 IU and 1000 IU presentations. wilate® is indicated for the prevention and treatment of haemorrhage or surgical bleeding in von Willebrand disease (VWD), when desmopressin (DDAVP) alone is ineffective or contra-indicated, and for the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency)6. About Octapharma Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein manufacturers in the world, developing and producing human proteins from human plasma and human cell lines. Octapharma employs nearly 12,000 employees worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: Immunotherapy, Hematology and Critical Care. Octapharma has seven R&D sites and five state-of-the-art manufacturing facilities in Austria, France, Germany and Sweden, and operates more than 195 plasma donation centers across Europe and the US. "Octapharma press releases are specifically for health specialist/medical media and are not for consumer press." [1] Lissitchkov T et al. Ther Adv Hematol 2019; 10:2040620719858471. [2]Liesner RJ et al. Thromb Haemost 2021; 121:1400–8. [3] Octapharma AG, data on file. [4] Nuwiq® Summary of Product Characteristics [5] Stadler M et al. Biologicals 2006; 34:281-8. [6] wilate® Summary of Product Characteristics. Logo: SOURCE Octapharma AG

Drug Approval

27 Feb 2024

·www.businesswire.com

Sepsis Drug Pipeline Research Report 2024: Insights from 30 Companies and 35 Pipeline Drugs - ResearchAndMarkets.com

DUBLIN--( BUSINESS WIRE )--The "Sepsis - Pipeline Insight, 2024" clinical trials has been added to ResearchAndMarkets.com's offering. This report provides comprehensive insights about 30+ companies and 35+ pipeline drugs in Sepsis pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space. The report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Sepsis pipeline landscape is provided which includes the disease overview and Sepsis treatment guidelines. The assessment part of the report embraces, in depth Sepsis commercial assessment and clinical assessment of the pipeline products under development In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Sepsis collaborations, licensing, mergers and acquisition, funding, designations and other product related details. Report Highlights The companies and academics are working to assess challenges and seek opportunities that could influence Sepsis R&D. The therapies under development are focused on novel approaches to treat/improve Sepsis. Sepsis Emerging Drugs Chapters This segment of the Sepsis report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases. Sepsis Emerging Drugs Thymosin alpha 1: SciClone Pharmaceuticals Thymosin alpha 1, referred to as thymalfasin, is a synthetic 28-amino acid peptide with multiple biological activities primarily directed towards immune response enhancement. SciClone developed and launched Thymosin alpha 1, under the trade name Zadaxin, for the treatment of hepatitis B virus (HBV) infection and hepatitis C virus (HCV) infections. The drug is also being developed for the treatment of COVID 2019 infections. Scientific and clinical studies have shown that Thymosin alpha 1 helps to stimulate and direct the body's immune response to eradicate infectious diseases, such as HBV, HCV, bacterial, and fungal infections; to fight certain cancers such as melanoma and liver cancer; and to enhance response to vaccines. Clinical trials have shown that Thymosin alpha 1 improves survival in patients in intensive care units being treated for sepsis from severe bacterial infections. The therapy is being evaluated in Phase III stage to treat Sepsis. Currently, the drug is in Phase III stage of its development for the treatment of sepsis. Enibarcimab: AdrenoMed AG Enibarcimab (HAM8101; former name: Adrecizumab) a clinical-stage, first-in-class drug targeting loss of vascular integrity. The strong rationale for Enibarcimab is supported by the elegance of its mode of action, a monoclonal antibody that on binding to its target Adrenomedullin preserves its functionality as regulator of vascular integrity. Enibarcimab targets Adrenomedullin (ADM), a vasoprotective peptide hormone. ADM that remains in the bloodstream, however, has a different effect, one that helps mitigate sepsis: promoting stability of the endothelial barrier by restoring the cell junctions between endothelial cells that ordinarily regulate molecule transport and leakage. In health, levels of ADM in the bloodstream and extravascular space are in equilibrium so that endothelial barrier integrity is maintained and blood pressure remains normal. Currently, the drug is in Phase II stage of its development for the treatment of sepsis. M 6229: Matisse Pharmaceuticals Matisse's platform technology is based on the discovery that in many patients suffering from sepsis, proteins called histones are released by the innate immune system and dying cells into the blood stream, where they are toxic to other cells. Due to a self-enforcing cascade, people may die from organ failure within one or two days. Preclinical results have shown that by neutralizing the toxic histones with Matisse's product M6229, the negative cascade is terminated by neutralization of cationic histones by anionic M6229. Matisse claims to have identified an elegant solution for treating one of the major complications in sepsis by using a non-anticoagulant fraction of heparin called M6229 to neutralize toxic circulatory histones .Currently, the drug is in Phase I stage of its development for the treatment of sepsis. SNIPR 001: SNIPR Biome SNIPR001 is a novel, orally-administered antibiotic that is designed to precisely target difficult-to-treat bacterial infections. It is developed using SNIPR Biome's CRISPR-Guided VectorsT (CGVT Technology), which is designed to deliver CRISPR reagents into target bacterial cells. SNIPR001 is designed to target certain E. coli bacteria in the gut and thus prevent their translocation to the bloodstream, without affecting beneficial bacteria in the microbiome. It contains four CRISPR-armed phages that selectively target and eliminate E. coli strains that are resistant to fluoroquinolone, with demonstrated efficacy in animal disease models. Currently, the drug is in preclinical stage of its development for the treatment of sepsis. Sepsis: Pipeline Development Activities The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Sepsis therapeutic drugs key players involved in developing key drugs. Pipeline Development Activities The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Sepsis drugs. Sepsis: Therapeutic Assessment Phases Late stage products (Phase III) Mid-stage products (Phase II) Early-stage product (Phase I) along with the details of Pre-clinical and Discovery stage candidates Discontinued & Inactive candidates Route of Administration Products have been categorized under various ROAs such as Oral Intravenous Subcutaneous Parenteral Topical Molecule Type Products have been categorized under various Molecule types such as Recombinant fusion proteins Small molecule Monoclonal antibody Peptide Polymer Gene therapy Product Type Sepsis Report Insights Sepsis Pipeline Analysis Therapeutic Assessment Unmet Needs Impact of Drugs Sepsis Report Assessment Pipeline Product Profiles Therapeutic Assessment Pipeline Assessment Inactive drugs assessment Unmet Needs Key Players SciClone Pharmaceuticals AdrenoMed AG Matisse Pharmaceuticals SNIPR Biome Tianjin Chase Sun Pharmaceutical Beijing Scitech-Mq Pharmaceuticals Recce Pharmaceuticals Inotrem Octapharma Shaperon Shionogi Pharmazz Seres Therapeutics Key Products Thymosin alpha 1 Enibarcimab M 6229 SNIPR 001 Kukoamine B ST-1830 RECCE 327 Nangibotide OctaplasLG HY209 Cefiderocol Centhaquine SER 155 For more information about this clinical trials report visit https://www.researchandmarkets.com/r/ixi7k5 About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

Phase 2Phase 3Phase 1

22 Jan 2024

·www.prnewswire.com

Publication of data from WIL-31 study marks a milestone in improving long-term care for patients with severe von Willebrand disease (VWD)

WIL-31 is the largest prospective prophylaxis study in VWD WIL-31 is the only study in VWD with a prospective on-demand run-in study as an intra-individual comparator wilate® prophylaxis is highly effective at reducing bleeding rates in children and adults with all types of VWD vs prior on-demand treatment LACHEN, Switzerland, Jan. 22, 2024 /PRNewswire/ -- Octapharma announced today the publication of the results from the clinical study WIL-31, which investigated the efficacy and safety of prophylaxis with wilate ®, a von Willebrand factor/factor VIII (VWF/FVIII) concentrate, in patients with all types of von Willebrand disease (VWD). The results have been published in the leading medical journal Blood Advances [Sidonio RF Jr et al. "Von Willebrand factor/factor VIII concentrate (wilate®) prophylaxis in children and adults with von Willebrand disease"] The lead author and Principal Investigator of the WIL-31 study, Robert F. Sidonio Jr, M.D. (Medical Director of Hemophilia at the Aflac Cancer & Blood Disorders Center with Children's Healthcare of Atlanta and Associate Professor of Pediatrics at Emory University School of Medicine), commented: "WIL-31 is the largest prospective study to date examining the efficacy and safety of regular prophylaxis in patients with VWD. Importantly, the study included a diverse patient population, including young children and adults, males and females, and all types of VWD. The findings from WIL-31 provide strong evidence to support the use of VWF prophylaxis and have led to the recent addition of wilate ® prophylaxis as a therapeutic indication for VWD in the US, widening its access to the US patient population." The WIL-31 study (NCT04052698) was a prospective, multinational, open-label, non-controlled phase III study assessing wilate ® prophylaxis in preventing bleeds in VWD. Efficacy was assessed in 33 adult and pediatric VWD patients aged 6 years and older from 14 centers in eight countries, with patients receiving wilate ® prophylaxis two or three times weekly for 12 months. All patients had taken part in a prospective 6-month run-in study (WIL-29) during which they received on-demand treatment with any available VWF concentrate, allowing assessment of the efficacy of prophylaxis at an intra-individual level. The primary endpoint of the study, >50% reduction in mean total annualized bleeding rate (ABR) during wilate® prophylaxis compared with prior on-demand treatment, was met. Mean total ABR during wilate® prophylaxis was 5.2 compared with 33.4 during on-demand treatment, representing a decrease of 84%. Mean spontaneous ABR decreased by 87% during wilate® prophylaxis compared with on-demand treatment. Reductions in total and spontaneous bleeding were comparable across age groups, bleeding sites and VWD disease types, and between males and females. The majority of patients were dosed twice weekly and the median weekly dose of wilate® was relatively low (58 IU/kg) compared to the dose range recommended in the prescribing information. Seven women with child-bearing potential were included in the study. This allowed the investigators to gain insights into the effect of wilate® prophylaxis on heavy menstrual bleeding, which is a major problem in women with VWD as there are limited clinical data on effective treatments. Although the data are based on a small sample size, they suggest that wilate® prophylaxis has a positive impact in reducing heavy menstrual bleeding, with 71% (n=5) patients not experiencing heavy menstrual bleeding during wilate® prophylaxis. Prophylaxis with wilate® was well tolerated across VWD types, in children and adults, and in males and females. During the 12 months of wilate® prophylaxis, there were no serious adverse events related to wilate® and no thrombotic events or FVIII accumulation. "In contrast to haemophilia A, prophylaxis is not standard of care in VWD and less than 10% of patients with severe VWD receive prophylaxis", said Sigurd Knaub, SVP CR&D Haematology. "This is due in part to the scarcity of data from prospective trials. The results of the WIL-31 study help to fill this gap, providing strong evidence for the benefits of a prophylactic strategy in patients with severe VWD. Increasing the adoption of prophylaxis in VWD is expected to lead to improved outcomes and reduced disease burden for people suffering from VWD." Octapharma would like to thank all participating centers, as well as the patients and their caregivers, for their contribution to the study. Further publications on other analyses from the WIL-31 study are planned. About wilate® wilate® is a high-purity human von Willebrand factor/factor VIII (VWF/FVIII) concentrate, that undergoes two virus inactivation steps during its production1. No albumin is added as a stabiliser1. The purification processes result in a 1:1 ratio of VWF to FVIII that is similar to normal plasma1. wilate® contains a VWF triplet structure and content of large high molecular weight multimers similar to normal human plasma1. wilate® is available in 500 IU and 1,000 IU presentations2. wilate® is indicated for the prevention and treatment of haemorrhage or surgical bleeding in von Willebrand disease (VWD), when desmopressin (DDAVP) alone is ineffective or contra-indicated, and for the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency)2. The indication for prophylaxis in the US is detailed in the updated prescribing information available here: wilate Full Prescribing Information (wilateusa.com) About Octapharma Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein manufacturers in the world, developing and producing human proteins from human plasma and human cell lines. Octapharma employs more than 11,000 people worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: Immunotherapy, Hematology, and Critical Care. Octapharma has seven R&D sites and five state-of-the-art manufacturing facilities in Austria, France, Germany and Sweden, and operates more than 190 plasma donation centers across Europe and the US. Octapharma has 40 years of experience in patient care. Octapharma press releases are specifically for health specialist/medical media and are not for consumer press. Contact: Ivana Spotakova Global Communications Manager Ivana.Spotakova@octapharma.com +41 79 3474607 References Stadler M et al. Biologicals 2006; 34:281–8. wilate® Summary of Product Characteristics 2023. Logo :

Clinical ResultPhase 3

100 Deals associated with Immune Globulin Intravenous (Human) (Octapharma)

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Polyradiculoneuropathy, Chronic Inflammatory Demyelinating	US	Octapharma Pharmazeutika Produktionsgesellschaft Mbh	02 Aug 2018
Primary Immunodeficiency Diseases	US	Octapharma Pharmazeutika Produktionsgesellschaft Mbh	02 Aug 2018
Dermatomyositis	US	Octapharma Pharmazeutika Produktionsgesellschaft Mbh	21 May 2004
Purpura, Thrombocytopenic, Idiopathic	US	Octapharma Pharmazeutika Produktionsgesellschaft Mbh	21 May 2004

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Pediatric Acute-Onset Neuropsychiatric Syndrome	Phase 3	US	Octapharma AG	30 Jun 2021
Pediatric Acute-Onset Neuropsychiatric Syndrome	Phase 3	IT	Octapharma AG	30 Jun 2021
Pediatric Acute-Onset Neuropsychiatric Syndrome	Phase 3	SE	Octapharma AG	30 Jun 2021
Pediatric Autoimmune Neuropsychiatric Disorders Associated With Streptococcal Infections	Phase 3	US	Octapharma AG	30 Jun 2021
Pediatric Autoimmune Neuropsychiatric Disorders Associated With Streptococcal Infections	Phase 3	IT	Octapharma AG	30 Jun 2021
Pediatric Autoimmune Neuropsychiatric Disorders Associated With Streptococcal Infections	Phase 3	SE	Octapharma AG	30 Jun 2021
Chronic Lymphocytic Leukemia	Phase 3	US	Octapharma AG	05 Oct 2020
Chronic Lymphocytic Leukemia	Phase 3	CZ	Octapharma AG	05 Oct 2020
Chronic Lymphocytic Leukemia	Phase 3	DK	Octapharma AG	05 Oct 2020
Chronic Lymphocytic Leukemia	Phase 3	DE	Octapharma AG	05 Oct 2020

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03939533 (CTgov)	Phase 3	Primary Immunodeficiency Diseases	64	CUTAQUIG (Increased Volume Cohort - Cohort 1)	llwmarjhxl(cqsztttckt) = hshgabakor tzsxdmvzsq (pvzaowspey, aaqrxujuzt - forxarqvfl) View more	-	07 Nov 2023
				CUTAQUIG (Increased Infusion Rate Cohort - Cohort 2)	llwmarjhxl(cqsztttckt) = rwzqnukrai tzsxdmvzsq (pvzaowspey, jdvyeujycm - kczyiaxdha) View more
NCT02728752 (ProDERM, Pubmed \| Br Dent J) Manual	Phase 3	Dermatomyositis	95	Intravenous Immune Globulin	hrivladgyh(pcqgultzrg) = hhuwobjmbg hvpalkilaj (gnmwdjcuws )	Positive	06 Oct 2022
				Placebo	hrivladgyh(pcqgultzrg) = gyvhvnuzhk hvpalkilaj (gnmwdjcuws )
NCT03686969 (SCGAM-02, CTgov)	Phase 3	Dermatomyositis	1	Octanorm (Octanorm)	ckseerqfwb(ziplysftbo) = mlixkwdtdb noyizwngct (djgnqlvwds, nrfbsytdwg - ihvyeltxkb) View more	-	23 Apr 2021
				Placebo (Placebo)	ckseerqfwb(ziplysftbo) = bjbhhqqfpg noyizwngct (djgnqlvwds, blvhijqvud - taspiypkly) View more
NCT02728752 (CTgov)	Phase 3	Dermatomyositis	95	placebo (Placebo)	jdvjjpbngm(qxsdhftnhc) = vvquwvnfeb iuvevmekvj (lwgmzeeqtr, svbdaknhlk - pvhbyqnlwx) View more	-	21 Apr 2021
				Octagam 10% (Octagam10%)	jdvjjpbngm(qxsdhftnhc) = zfehphmefj iuvevmekvj (lwgmzeeqtr, htjnvshuzz - xzmgavlyrp) View more
NCT02638207 (CTgov)	Phase 3	Polyradiculoneuropathy, Chronic Inflammatory Demyelinating	142	NewGam (1.0 g/kg NewGam)	ewkyjretpz(cejlqeagmn) = nrqhisnkbr dowfbqcxfv (kxhysmgkvl, ocgnbomtiw - orbhzdjjzx) View more	-	16 Feb 2021
				NewGam (0.5 g/kg NewGam)	ljlmhsbwgw(xpwxvhaxuc) = nxnhfaajif ckoqutvjbq (xeuoawqbni, raexmebruv - mjwdpxzeja) View more
NCT02728752 (ProDERM Study, ACR2020) Manual	Phase 3	Dermatomyositis	95	Octagam 10%	fxxqeyvexn(miyuhyknus) = vonfyyucnb jgsriaxxyd (hombcousvx ) View more	Positive	07 Nov 2020
				Placebo	fxxqeyvexn(miyuhyknus) = iojsmpprmr jgsriaxxyd (hombcousvx ) View more
NCT03593902 (CTgov)	Phase 2/3	Scleroderma, Systemic	9	Autologous Stem Cells+Mesna+Methylprednisolone+Fludarabine+CYCLOPHOSPHAMIDE+rATG+G-CSF+IVIg+Rituximab	depawamqvp(kithrarygg) = yzelthzavp talxjabojz (ksabmhhkct, gbjsramdal - htbyzorgxl) View more	-	12 Aug 2020
NCT03319810 (CTgov)	Phase 2	Alzheimer Disease \| Mild cognitive disorder	5	Octagam 10%	szbaeomyjs(fwxguuulti) = ogbqxzypee zelkshyvts (wrhuqdfykp, heliipbwhz - fylbdtjutv) View more	-	16 Apr 2019
NCT01349790 (Pubmed \| Transfus Med)	Phase 3	Chronic thrombocytopenia First line	40	IVIG 10%	dtgqlyoeuw(pdpxgftqro) = iwegpjzlcb qjllsxzsgr (iygndrgewo ) View more	-	01 Feb 2019
NCT00811174 (CTgov)	Phase 3	Primary Immunodeficiency Diseases	5	Octagam 10%	eezsgynjtk(pgxzbwgwzk) = qlkvmixjun ryytyljjdn (vsfujpsvxq, tenzgpiukt - dgezmegxzm) View more	-	14 Jul 2017

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Immune Globulin Intravenous (Human) (Octapharma)

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