RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (Australia), Overseas New Drugs Urgently Needed in Clinical Settings (China), Orphan Drug (United Kingdom)

Structure/Sequence

Sequence Code 35284H

Source: *****

Clinical Trials associated with Caplacizumab-YHDP

NCT06358703

/ Not yet recruitingNot ApplicableIIT

Impact of Acute ITTP Therapies on Long Term Neurologic and Cognitive Outcomes in ITTP Survivors

1. We expect to find that the silent cerebral infarct (SCI) rate is two fold higher in patients treated without caplacizumab. We also expect to find that the rate of mild and major cognitive impairment in patients treated with caplacizumab within 3 days of starting plasma exchange will be lower than patients treated without caplacizumab.
2. We expect that the differences in cognitive impairment in cases (caplacizumab) versus controls (no caplacizumab) will persist on serial evaluation 1 year later. We also expect that there will be differences in these groups even after adjusting for time since episode and severity of presentation.
3. We expect to find that SCI and cognitive impairment is associated with worse scores on the health related quality of life instrument (SF-36)
4. Based on studies in non-TTP populations, we expect to find that the rate of incident stroke over the period of follow up is at least 2 fold higher in patients that have SCI compared with patients who do not have SCI

Start Date01 Dec 2024

Sponsor / Collaborator-

NCT05876221

/ RecruitingNot ApplicableIIT

Platelet Response to Caplacizumab in the Treatment of Acquired Thrombotic Thrombocytopenic Purpura

The interpretation of platelet counts has to be revaluated in the light of caplacizumab. By effectively blocking platelet binding sites on VWF-multimers, the nanobody leads to a rapid normalization of the platelet count within 3 to 4 days. Most importantly, caplacizumab uncouples platelet counts from ADAMTS13 activity and thereby launches unprecedented thrombocyte dynamics, with potential pitfalls for over- and undertreatment.
A relevant number of patients responds to caplacizumab with a brisk increase in platelet count, followed by a marked dip of platelets (patient on the left). This may mislead treating physicians into re-intensifying therapy, with a respective risk for adverse side-effects and complications. Taken together, these observations call for reliable descriptions and the identification of predictive parameters to predict the platelet response upon administration of caplacizumab in a large patient cohort. Here, PREDICT-2020 is designed as a retrospective study to specifically address the following aspects:
* Identifying and describing clusters of platelet responses to caplacizumab
* Identifying potential pitfalls for treating physicians
* Predicting the individual thrombocyte response
* Correlating platelet responses with individual patient outcome

Start Date30 Oct 2023

Sponsor / Collaborator

Universities of Cologne

NCT05468320

/ CompletedPhase 3

An Open-label, Single-arm, Multicenter Study to Evaluate the Efficacy and Safety of Caplacizumab and Immunosuppressive Therapy Without Firstline Therapeutic Plasma Exchange in Adults With Immune-mediated Thrombotic Thrombocytopenic Purpura

This is a single group, treatment, Phase 3, open-label, single-arm study to evaluate the efficacy and safety of caplacizumab and immunosuppressive therapy (IST) without firstline therapeutic plasma exchange (TPE) with primary endpoint of remission in male and female participants aged 18 to 80 years with immune-mediated thrombotic thrombocytopenic purpura (iTTP).
The anticipated study duration per participant without a recurrence while on therapy is maximum 24 weeks (ie, approximately 1 day for screening + maximum 12 weeks of treatment for the presenting episode + 12 weeks of follow-up). Participants will have daily assessments during hospitalization and weekly visits for assessments during ongoing treatment with caplacizumab and IST. There will be 3 outpatient visits for assessments during the follow-up period. There will be two additional follow-up visits for participants who do not have ADAMTS13 activity levels of ≥50% at the time of caplacizumab discontinuation.

Start Date21 Nov 2022

Sponsor / Collaborator

Sanofi

100 Clinical Results associated with Caplacizumab-YHDP

100 Translational Medicine associated with Caplacizumab-YHDP

100 Patents (Medical) associated with Caplacizumab-YHDP

297

Literatures (Medical) associated with Caplacizumab-YHDP

01 Feb 2025·JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Bortezomib for rituximab-refractory immune-mediated thrombotic thrombocytopenic purpura in the caplacizumab era: an Italian multicenter study

Article

Author: Napolitano, Mariasanta ; Miri, Syna ; Grimaldi, Daniele ; Scarpa, Elisabetta ; Massaia, Massimo ; Cuccaro, Annarosa ; Bocchia, Monica ; Artoni, Andrea ; Salutari, Prassede ; Giannotta, Juri Alessandro ; Agosti, Pasquale ; Truma, Addolorata ; Molteni, Alfredo ; Mancini, Giorgia ; Prezioso, Lucia ; Carpenedo, Monica ; Peyvandi, Flora ; Mancini, Ilaria ; De Leo, Pasqualina ; Ferrari, Barbara ; Rinaldi, Ermina ; Condorelli, Annalisa

BACKGROUND:

Immune-mediated thrombotic thrombocytopenic purpura (iTTP) patients are not responsive to standard rituximab in approximately 10% to 15% of cases, and oral immunosuppressants showed controversial results with significant toxicity. Targeting plasma cells with bortezomib appears promising, but the available evidence is scarce and stems only from isolated reports in the precaplacizumab era.

OBJECTIVES:

To evaluate the safety and efficacy of bortezomib in rituximab-refractory iTTP patients.

METHODS:

We conducted a retrospective observational multicenter study among 13 Italian iTTP treating centers, collecting data from May 2017 to May 2023 (caplacizumab was licensed in Italy in January 2020).

RESULTS:

Bortezomib was effective in 10/17 patients (59%). Eleven were treated in the acute phase (9/11 responders, 82%, allowing discontinuation of caplacizumab in 5/6 treated patients), and 7 during clinical remission (2/7 responders, 28%). Responses occurred at a median time of 30 days, but 3 patients responded after 4 months. The median duration of response was 22 months (IQR, 10-38), still ongoing in 6 patients at the time of data cutoff. Responders had fewer previous acute iTTP episodes than nonresponders (median [IQR], 1 [1,2] vs 5.5 [2-7]; P = .03). Eight subjects (47%) reported toxicities, mostly in those treated with ≥2 cycles.

CONCLUSION:

Durable responses to bortezomib were registered in about 60% of multirefractory iTTP patients with mild to moderate toxicities. The occurrence of late responses (ie, after 30 days) suggests a "watchful waiting" approach after bortezomib treatment.

01 Feb 2025·TRENDS IN BIOTECHNOLOGY

A high-performance protein preparation approach in a single column-free step

Article

Author: Lin, Zhanglin ; Huang, Yuan ; Zhang, Yuanyuan ; Yang, Xiaofeng

Protein purification remains a formidable and costly technical obstacle in biotechnology. Here, we present a new column-free method, utilizing the cleavable self-aggregating tag 2.0 (cSAT2.0) scheme, to streamline protein production in Escherichia coli, yielding high quantities with exceptional purity. In shake-flask experiments using lysogeny broth (LB) medium, the cSAT2.0 scheme successfully produced one peptide and five proteins, with yields ranging from 24 mg/l to 89 mg/l, and purity levels exceeding 98%. The cSAT2.0 scheme also enabled high-throughput protein preparation on microplates. Furthermore, we scaled up the fermentation process for caplacizumab, achieving 1.4 g/l of highly purified protein in a 5-l fermenter. Our results demonstrate that the cSAT2.0 scheme can serve as an economical and robust platform for protein production from microplate to fermenter scales.

01 Feb 2025·Journal of immunotherapy and precision oncology

A Rare But Fatal Toxicity: Immune Checkpoint Inhibitor–Related Acquired Thrombotic Thrombocytopenic Purpura

Review

Author: Yikilmaz, Aysun Senturk ; Hernandez, Cristhiam M. Rojas

ABSTRACT:

Thrombotic thrombocytopenic purpura (TTP) is characterized by thrombotic microangiopathy resulting from decreased activation of the von Willebrand factor–cleaving protease (ADAMTS13). TTP can cause organ damage and is often fatal if the appropriate treatment is not started immediately. Although primary immune TTP is the most common form of TTP, secondary immune etiologies, including complications from immune checkpoint inhibitors (ICIs), have also been reported. ICIs are used as neoadjuvant and adjuvant therapy for metastatic and nonmetastatic solid tumors and hematologic cancers. ICIs stimulate the T-cell–mediated antitumor response, and the subsequent upregulation of the immune system can cause ICI-related adverse events (AEs). ICI-associated AEs may result in various hematological outcomes. Therefore, TTP, as a rare ICI-related AE, requires awareness. TTP has been mentioned as a rare ICIrAE in a few case reports. When using ICIs, the differential diagnosis of TTP should be considered if hemolytic anemia is accompanied by thrombocytopenia. Low ADAMTS13 activity can be used to diagnose TTP and support the need for plasma exchange. This review will assess the approach for ICI-related acquired TTP by scanning a limited number of reported case series in the literature. Low ADAMTS13 activity can be used to diagnose TTP and support the need for plasma exchange. Treatment in the cases that have been published includes combinations of rituximab and caplacizumab, corticosteroids, and plasma exchange. Furthermore, acquired TTP associated with ICI is encountered during the initial and subsequent cycles of ICI treatment. It is essential to detect ICI-related acquired TTP early, a highly fatal AE of ICIs, and to increase awareness of TTP, which will likely be encountered more frequently with the use of new ICI agents.

News (Medical) associated with Caplacizumab-YHDP

19 Mar 2025

·www.prnewswire.com

CABLIVI Market Reports Positive Growth - Strong Momentum Expected to Continue | DelveInsight

Developed by Sanofi, CABLIVI is the first FDA-approved nanobody therapy for this condition. Increasing awareness, improved diagnostic rates, and rising demand for targeted therapies in hematology drive the market growth. With its niche indication and high treatment costs, CABLIVI holds a strong position in the rare disease therapeutics segment. LAS VEGAS, March 19, 2025 /PRNewswire/ -- DelveInsight's " CABLIVI Market Size, Forecast, and Market Insight Report" highlights the details around CABLIVI, a von Willebrand factor (vWF)-directed antibody fragment. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of CABLIVI. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Ablynx NV's CABLIVI (caplacizumab-yhdp) Overview CABLIVI is an antibody fragment that targets von Willebrand factor (vWF) and is used to treat adult patients with acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange and immunosuppressive therapy. It works by binding to the A1-domain of vWF, preventing its interaction with platelets, which helps reduce vWF-mediated platelet adhesion and consumption. CABLIVI is approved in both the US and EU for treating aTTP. In 2024, CABLIVI sales reached €249 million, reflecting a 9.7% increase, driven by a rise in the number of patients identified for suitable treatment in the US, as well as new launches in Europe and other regions. The recommended CABLIVI dosing schedule is as follows: Day 1: Administer an 11 mg intravenous bolus injection at least 15 minutes before plasma exchange, followed by an 11 mg subcutaneous injection after the exchange on the same day. During Plasma Exchange Therapy: Administer 11 mg subcutaneously once daily after each plasma exchange session. After Plasma Exchange Therapy: Continue with 11 mg subcutaneous injections once daily for 30 days after the final plasma exchange. If signs of persistent underlying disease, such as low ADAMTS13 activity, are still present, treatment can be extended for up to 28 additional days. CABLIVI treatment should be discontinued if the patient experiences more than two recurrences of aTTP while on the medication. Learn more about CABLIVI projected market size for thrombocytopenia @ CABLIVI Market Potential Thrombocytopenia is a medical condition where the platelet count in the blood drops below the normal level (<150,000/µL). Common causes include heparin-induced thrombocytopenia, chemotherapy-induced thrombocytopenia, thrombocytopenia linked to chronic liver disease, immune thrombocytopenia (ITP), and thrombotic thrombocytopenic purpura (TTP). Among the 7MM, the United States reported the highest number of thrombocytopenia cases, accounting for about 40% of the total cases in 2023, according to DelveInsight. Treatment for thrombocytopenia varies based on its cause and severity, with the primary goal of preventing bleeding-related complications and improving patient outcomes. Approved therapies for thrombocytopenia in the US include MULPLETA, DOPTELET, TAVALISSE, PROMACTA, NPLATE, CABLIVI, GAMMAPLEX, OCTAGAM, RHOPHYLAC, and PRIVIGEN. Additionally, danaparoid, argatroban, and lepirudin are approved for treating thrombosis in patients with heparin-induced thrombocytopenia. For severe thrombocytopenia related to chronic liver disease, treatment options include platelet transfusion, splenic artery embolization, splenectomy, and transjugular intrahepatic portosystemic stent shunt (TIPSS) placement. The US holds approximately 60% of the thrombocytopenia market share, surpassing the combined market size of the EU4, the UK, and Japan. Market growth is expected to be driven by an increasing patient population, the introduction of new therapies, and deeper market penetration within the 7MM. Discover more about the thrombocytopenia market in detail @ Thrombocytopenia Market Report Emerging Competitors of CABLIVI The potential therapies that can mark a significant change in the thrombocytopenia treatment landscape and give tough competition to CABLIVI include Rilzabrutinib (Sanofi), Ianalumab (Novartis), Mezagitamab (Takeda), PF-06835375 (Pfizer), Povetacicept (Vertex), Cevidoplenib (Genosco/Oscotec), and Efgartigimod (ARGX-113) (Argenx), among others. In December 2024, Sanofi shared encouraging Phase III results at ASH for rilzabrutinib, its investigational oral BTK inhibitor, in patients with ITP. In June 2024, Takeda reported late-breaking Phase IIb data on Mezagitamab, highlighting its potential to revolutionize the treatment of Primary ITP. In Novartis' Q2 2024 update, the company forecasted key trial results: VAYHIT1 (NCT05653349) for first-line therapy, expected in 2026, and VAYHIT2 (NCT05653219) for second-line therapy, anticipated in 2025. Novartis also aims to file for ianalumabin approval in both first- and second-line ITP treatment by 2027 or later. To know more about the number of competing drugs in development, visit @ CABLIVI Market Positioning Compared to Other Drugs Key Milestones of CABLIVI In June 2022, at the 27th Annual European HematologyAssociation (EHA) Congress, Sanofi presented long-term safety and efficacy results from the post-HERCULES trial (NCT02878603), a three-year prospective follow-up study of patients with acquired thrombotic thrombocytopenic purpura (aTTP) who completed the Phase 3 HERCULES trial. In February 2019, the FDA approved CABLIVI (caplacizumab-yhdp) injection as the first therapy specifically indicated for treating adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), a rare and life-threatening blood clotting disorder. It is to be used in combination with plasma exchange and immunosuppressive therapy. In September 2018, the European Commission approved CABLIVI (caplacizumab) for marketing, authorizing its use in treating adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), a rare blood-clotting disorder. CABLIVI is the first therapy specifically designed for aTTP treatment. In July 2017, Ablynx announced a research collaboration and global exclusive licensing agreement with Sanofi, aiming to develop and commercialize Nanobody-based therapies for treating various immune-mediated inflammatory diseases. Discover how CABLIVI is shaping the thrombocytopenia treatment landscape @ CABLIVI FDA Approval CABLIVI Market Dynamics The primary driver of CABLIVI's market growth is the increasing recognition and diagnosis of aTTP, coupled with improved access to specialized treatments. Growing awareness among healthcare providers and advancements in diagnostic capabilities have facilitated early and accurate diagnosis, thereby boosting the demand for targeted therapies like CABLIVI. The drug's ability to prevent microthrombi formation, shorten the duration of plasma exchange, and reduce the risk of relapses has positioned it as a preferred option in the treatment landscape for aTTP. Moreover, Sanofi's robust commercial infrastructure and strategic marketing efforts have further contributed to market penetration and adoption. Despite its therapeutic benefits, CABLIVI faces challenges related to its high cost and reimbursement complexities. The specialized nature of aTTP treatment, combined with the requirement for hospital-based administration and close monitoring, adds to the overall cost burden on healthcare systems and patients. Additionally, competition from alternative therapies, including plasma exchange and immunosuppressive treatments, may limit its market expansion. Regulatory hurdles and the need for long-term safety data could also impact the drug's market trajectory. The CABLIVI market is expected to grow steadily, driven by ongoing clinical research and potential label expansions into related thrombotic disorders. Increased investments in rare disease treatments and orphan drug incentives are likely to support market growth. Moreover, the rise in strategic collaborations and partnerships to improve patient access and streamline reimbursement processes could enhance market uptake. However, balancing pricing pressures with broader patient access will remain a key factor in sustaining long-term market performance. Dive deeper to get more insight into CABLIVI's strengths & weaknesses relative to competitors @ CABLIVI Market Drug Report Table of Contents Related Reports Acquired Thrombotic Thrombocytopenic Purpura Pipeline Acquired Thrombotic Thrombocytopenic Purpura Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key acquired thrombotic thrombocytopenic purpura companies, including Sanofi, TargED Biopharmaceuticals, Takeda, Genentech, Alexion Pharmaceuticals, among others. Thrombocytopenia Market Thrombocytopenia Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key thrombocytopenia companies including Sanofi, Principia Biopharma, Baxalta, Takeda, Argenx, Millennium Pharmaceuticals, Biotest, GC Pharma, Genosco (Subsidiary of Oscotec), Rigel Pharmaceuticals, Kissei Pharmaceutical, Shionogi & Co., Ltd, Amgen, Novartis, Zenyaku Kogyo, among others. Thrombocytopenia Pipeline Thrombocytopenia Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key thrombocytopenia companies, including Takeda, Argenx, Keros Therapeutics, Principia Biopharma, Momenta Pharmaceuticals, Veralox Therapeutics, Novartis Pharmaceuticals, Pfizer, HUTCHMED, Principia Biopharma, Genosco, UCB, among others. Immune Thrombocytopenia Market Immune Thrombocytopenia Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key immune thrombocytopenia companies including Rigel Pharmaceuticals, Kissei Pharmaceutical, Sobi (Dova Pharmaceuticals), Amgen, Argenx, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultLicense out/inPhase 3Drug Approval

19 Dec 2024

·www.prnewswire.com

TargED Biopharmaceuticals doses first participant in Phase 1 clinical trial of TGD001, a groundbreaking thrombolytic in acute ischemic stroke (AIS) and TTP

TGD001 is being developed to offer patients with clotting disorders a faster, safer and more effective treatment than today's standard of care – saving lives and reducing disability Following the Phase 1 safety study, TargED intends to further develop TGD001 in patients with AIS and iTTP (a rare micro-thrombotic disorder) in the second half of 2025 UTRECHT, the Netherlands, Dec. 19, 2024 /PRNewswire/ -- TargED Biopharmaceuticals ("TargED"), a private biotechnology company focused on developing improved treatments for thrombotic diseases, today announces the start of clinical development with the dosing of the first participant with TGD001. TGD001 is a groundbreaking thrombolytic designed to break down clots of all sizes and compositions – from large clots which cause AIS, down to 'micro-clots' which cause conditions such as the rare, life-threatening disorder immune-mediated thrombotic thrombocytopenic purpura (iTTP). The first study participant was successfully dosed yesterday [Dec 18] at a Phase 1 clinic in Germany. The study's design is a randomized, double-blind, placebo-controlled single-ascending dose study to evaluate TGD001's safety, tolerability and pharmacokinetics in healthy volunteers (EU CT ID number: 2024-514931-63-00). First safety read-out from the Phase 1 study is expected by mid-2025. TargED plans to continue clinical development with two proof-of-concept trials in the second half of 2025: a Phase 2a trial in AIS patients and a Phase 1b trial in iTTP patients, for which TargED received EU Orphan Drug Designation in July 2024 from the European Commission. Kristof Vercruysse, Chief Executive Officer and co-founder of TargED, said: "The dosing of the first participant with our lead compound TGD001 marks a watershed in the treatment of thrombotic disorders. Rapid treatment with highly effective thrombolytics that are well tolerated and don't exaggerate the risk of bleeding is essential to reduce the risk of serious disability or even death from thrombotic disorders. Today's thrombolytics show efficacy but their significant drawbacks greatly limit their safety and effectiveness, and result in restricted utility. TGD001 is specifically designed to address these shortcomings. Its unique mode of action allows for blood clots of all sizes and compositions to be targeted and rapidly dissolved without increasing bleeding risk, resulting in clinical benefit for a broad population of patients." Steven de Maat, Chief Scientific Officer and co-founder of TargED, said: "Acute ischemic stroke is one of the biggest causes of preventable death and disability in the world today, but up to 80% of AIS patients cannot be treated with currently available thrombolytics such as intravenous alteplase (tPA). Meanwhile, there is significant room for improvement in the treatment of iTTP. Despite recent advances, 15% of iTTP patients who receive treatment do not survive, and half of those who live still suffer long term health impairment. TGD001 has the potential to greatly improve outcomes in both these indications and, we believe, in other thrombotic indications as well." TGD001 is a first-in-class 'fusion protein' drug with a unique two-step mode of action. It targets clots via an antibody fragment that binds to a non-functional domain of von Willebrand Factor (VWF), a protein present in all types of thrombi. Then, in the immediate vicinity of the thrombus, it activates the endogenous enzyme system that degrades both VWF and fibrin, two key clotting ingredients which form the structural bond in the thrombus. This mode of action enables rapid and effective thrombolysis without systemic activation of the lysis cascade and the resulting bleeding risk. TGD001 is administered as short or bolus injection, making it a suitable therapeutic for the emergency, in-patient and first-response setting. About TargED Biopharmaceuticals TargED Biopharmaceuticals B.V. is a Netherlands based biotechnology company developing first-in-class biological drugs to improve treatment of thrombosis. TargED stands for Targeted Enzyme Delivery. TargED's biological drugs are unique by using antibody fragments ("VhH") to deliver enzymes to sites of thrombosis, enabling 'targeted' thrombolysis. Its lead compound TGD001 is currently under development for the treatment of immune-mediated thrombotic thrombocytopenic purpura (iTTP) and acute ischemic stroke (AIS). The objective is to accelerate thrombolysis in all forms of thrombosis, irrespective of the thrombus composition. TargED, a spin-off of the University Medical Center Utrecht, was founded in July 2020. Since then, TargED raised more than €45 million of funding, supporting its growth to become a clinical-stage company. For more information please visit Acute Ischemic Stroke (AIS), which occurs when one or more clots block blood flow to part of the brain, accounts for around 90% of all strokes. Worldwide, around 18 million AIS incidents take place every year, resulting in 5 million deaths and 5 million people permanently disabled. A delay in diagnosis and treatment can lead to loss of brain tissue, causing permanent disability or death. Standard of care thrombolysis must be initiated within 4.5 hours of ischemic stroke, since after this time the risk of tPA-induced bleeding outweighs the potential benefit. Due to their limitations, up to 80% of AIS patients are ineligible for current thrombolytics like tPA. Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening blood disorder characterized by sudden episodes of clot formation in small blood vessels throughout the body. Blood flow to organs such as the kidney, heart, and brain is disrupted, causing functional failure. There are about 7,500 incidents of iTTP a year worldwide and it particularly affects young women, sometimes after pregnancy. Without treatment, up to 90% of cases are fatal. Treatment in specialized centers cuts mortality to 15%; however, of the survivors, half still suffer long-term complications. Current treatment is complex, involving blood plasma exchange, immune suppression and administration of the drug caplacizumab, which stops new clot formation. To date, no thrombolytic – i.e. a drug that clears existing thrombi – is licensed in iTTP due to the perceived inherent bleeding risk of patients with thrombotic microangiopathies and the systemic thrombolytic activity of currently licensed thrombolytics. TGD001 has the potential to resolve iTTP attacks very rapidly, saving organ tissue and preventing disability and death. SOURCE TargED WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 1Orphan Drug

10 Jul 2024

·www.biospace.com

Antonin (Tony) de Fougerolles appointed as new Chair of etherna

Brings foundational experience in mRNA and LNP therapeutics given prior executive leadership roles at Moderna & Alnylam Will take an active role in expanding partnerships with biopharma Integrated partnership offering to customers remains etherna’s key differentiator – Proprietary customized mRNA chemistry and lipid nanoparticles (customized LNPs) expertise combined with GMP process knowhow Niel, Belgium, July 09, 2024 – etherna (“the Company”), a leading mRNA technologies company, today announced the appointment of Antonin (Tony) de Fougerolles as Chair. Tony has served as an Independent Board Member since January 2023. Outgoing Chair, Marijn Dekkers will remain on the Board. In addition to his role as Chair, Tony will also take an active consultancy role for the Company. Bernard Sagaert, etherna CEO said: “I am absolutely thrilled to be able to work more closely with Tony. His vast experience, connections and strategic vision in the field will further accelerate the development and success of the Company. I also want to thank Marijn for the close collaboration over the last 8 months, which has been inspirational.” Tony de Fougerolles, etherna Chair added: “Over the last 18 months I have seen first-hand how the cLNPs and mRNA manufacturing expertise that etherna has developed is world leading and impressive in its quality, breadth, and novelty. The recently announced partnership with Almirall is clear recognition of this. I am excited to accept the role of Chair and look forward to actively helping Bernard and his talented team take full advantage of the exciting opportunities to help our partners bring novel mRNA-based therapeutics to patients. Marijn Dekkers, etherna Independent Board Member noted: “Tony has already proven to be an extremely valued addition to the Board and therefore I am delighted to welcome him as my successor. His combination of scientific and entrepreneurial excellence as well as clear strategic vision will enable him, I believe, to steer etherna successfully through the next stage of its development.” Tony de Fougerolles has foundational experience in both mRNA therapeutics and LNPs specifically through his roles at Moderna and Alnylam. He has nearly 25 years of biotech R&D experience in building out drug pipelines, and he has played a key role in developing and successfully advancing 3 new drug modalities to the market (mRNA, RNAi, single domain antibodies) and in helping build several multi-billion-dollar companies from start-up stage. As founding Chief Scientific Officer (CSO) at Moderna Therapeutics, he pioneered modified mRNA as a new therapeutic and vaccine drug modality and the mRNA chemistry and delivery systems that form the basis of the approved COVID-19 mRNA vaccines. Prior to that, as VP of Research at Alnylam, he helped develop RNA interference (RNAi) as a new drug modality, including overseeing development of the 1st approved LNP-based RNAi delivery system and the development of several approved RNAi drugs including inclisiran. Author of over 60 scientific publications and an inventor on over 110 issued US patents, Tony has also a ‘Belgian’ connection, from his time as CSO at Ablynx (now Sanofi) where he played a key role in the approval of the 1st single domain antibody drug, caplacizumab. Most recently, Tony was CEO of Evox Therapeutics where he built out an exosome-focused biotech, raised over $140m in equity financing, and entered into pharma partnerships with combined upfronts > $60m and total deal value >$2 billion. Tony obtained his PhD in Immunology from Harvard University. -ends- For further information, please contact: etherna Bernard Sagaert media@etherna.be RHA Communications Richard@rhacomms.eu Notes to Editors About etherna etherna is an mRNA technology platform company with fully integrated capabilities including mRNA construct design and optimization, coupled with specialized expertise in designing and manufacturing customized lipid nanoparticles (cLNP) formulations tailored for the prevention and treatment of various pathological states, providing end-to-end solutions for next generation mRNA therapeutics. The company offers this knowledge and know-how in a partnership model to drive discovery and solve challenges for partners across LNP formulation and RNA chemistry to enable delivery of cost-effective, differentiated and efficacious RNA therapeutics.

Executive ChangeVaccinemRNADrug Approval

100 Deals associated with Caplacizumab-YHDP

External Link

KEGG	Wiki	ATC	Drug Bank
-	Caplacizumab-YHDP	B01AX07	DB06081

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Purpura, Thrombotic Thrombocytopenic	Canada	Sanofi-Aventis Canada, Inc.	04 Sep 2020
Thrombotic Thrombocytopenic Purpura, Acquired	European Union	Ablynx NV	30 Aug 2018
Thrombotic Thrombocytopenic Purpura, Acquired	Iceland	Ablynx NV	30 Aug 2018
Thrombotic Thrombocytopenic Purpura, Acquired	Liechtenstein	Ablynx NV	30 Aug 2018
Thrombotic Thrombocytopenic Purpura, Acquired	Norway	Ablynx NV	30 Aug 2018

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Angina, Stable	Phase 2	Austria	Ablynx NV	01 Sep 2009
Angina, Stable	Phase 2	Belgium	Ablynx NV	01 Sep 2009
Angina, Stable	Phase 2	Czechia	Ablynx NV	01 Sep 2009
Angina, Stable	Phase 2	Germany	Ablynx NV	01 Sep 2009
Angina, Stable	Phase 2	Israel	Ablynx NV	01 Sep 2009
Angina, Stable	Phase 2	Poland	Ablynx NV	01 Sep 2009
Angina, Stable	Phase 2	Switzerland	Ablynx NV	01 Sep 2009
Angina, Unstable	Phase 2	Austria	Ablynx NV	01 Sep 2009
Angina, Unstable	Phase 2	Belgium	Ablynx NV	01 Sep 2009
Angina, Unstable	Phase 2	Czechia	Ablynx NV	01 Sep 2009

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04985318 (REACT-2020, EHA2024)	Not Applicable	Purpura, Thrombotic Thrombocytopenic	200	Caplacizumab-treated iTTP patients	nlowhrhonu(slhfkbjoxj) = sawkdauqjy hsvukgpurc (ziqclqujye ) View more	Positive	14 May 2024
ASH2023	Not Applicable	Thrombotic Thrombocytopenic Purpura, Acquired	33	Caplacizumab (Conventional treatment alone)	pxmcmnugtb(tjqkrzrfiu) = sigzxrufen gccsejdnjs (dyxesjkxai ) View more	Positive	10 Dec 2023
ASH2023	Not Applicable	Thrombotic Thrombocytopenic Purpura, Acquired	33	Conventional treatment + Caplacizumab	pxmcmnugtb(tjqkrzrfiu) = nzsekzyxbc gccsejdnjs (dyxesjkxai )	Positive	10 Dec 2023
ISTH2023	Not Applicable	Purpura, Thrombotic Thrombocytopenic	505	Caplacizumab	pidbunaxfb(ddjdvpfaph) = Our study shows that bleeding is a common complication of Caplacizumab, which decreases the VWF levels and increases bleeding risk lghxerhsoe (mwovfameba )	-	24 Jun 2023
EHA2023	Not Applicable	Thrombotic Thrombocytopenic Purpura, Acquired	60	CAPLACIZUMAB (Patients ≥60 years)	ioeqayldoi(hyhrnslqmr) = one patient ≥60 years died on day 12 after diagnosis due to multiorgan failure that was not considered caplacizumab-related, but progression of iTTP unihpgavrk (cyvqbmioul ) View more	-	08 Jun 2023
EHA2023	Not Applicable	Thrombotic Thrombocytopenic Purpura, Acquired	60	CAPLACIZUMAB (Patients <60 years)		-	08 Jun 2023
NCT04985318 (Pubmed \| J Thromb Haemost)	Not Applicable	Thrombotic Thrombocytopenic Purpura, Acquired First line	-	Caplacizumab	aflcgieetw(dkltqnoddp) = bsrhscgfwv mcvzmifbxf (qwxiqhgyro ) View more	-	22 Dec 2022
NCT04074187 (Pubmed)	Phase 2/3	Thrombotic Thrombocytopenic Purpura, Acquired	21	Caplacizumab 10mg	gcbrhsedzx(lxuehwgxlo) = iqfbveedlq yntxyoujmj (celonvjsga )	Positive	24 Nov 2022
NCT04985318 (REACT-2020, ASN2022)	Not Applicable	Thrombotic Thrombocytopenic Purpura, Acquired First line ADAMTS13 activity below 10 percent \| relapsing thrombotic microangiopathy	-	Caplacizumab	ebxhpfomul(muomwwqddw) = slcfcngmta opwlnvsayo (zsjdhqzyfy )	Positive	03 Nov 2022
NCT04985318 (REACT-2020, ASN2022)	Not Applicable		-	Standard of care without Caplacizumab	ebxhpfomul(muomwwqddw) = hdoojmwngm opwlnvsayo (zsjdhqzyfy )	Positive	03 Nov 2022
NCT02878603 (HERCULES, Pubmed)	Phase 3	Thrombotic Thrombocytopenic Purpura, Acquired	104	Caplacizumab+TPE+IST	ndmcdkidvq(kucoxmtwus) = vbdeghnnrz xiyivnckrf (avksfnoxtj ) View more	Positive	22 Sep 2022
NCT02878603 (HERCULES, Pubmed)	Phase 3	Thrombotic Thrombocytopenic Purpura, Acquired	104	Placebo+TPE+IST	ndmcdkidvq(kucoxmtwus) = wdzjqhsoxz xiyivnckrf (avksfnoxtj )	Positive	22 Sep 2022
Milan TTP Registry (ISTH2022)	Not Applicable	Thrombotic Thrombocytopenic Purpura, Acquired	26	Caplacizumab	rolhqrpbtt(pjlxvyqmjx) = mnsnplvucy zbbeslmdlt (itileiokkk )	Positive	09 Jul 2022
NCT04074187 (ASH 12021 \| ASH 22021) Manual	Phase 2/3	Thrombotic Thrombocytopenic Purpura, Acquired	21	Caplacizumab	sjiycjchaq(jsyeejlgfx) = zjwipftglj slqzobwycn (ggycnhdmtb ) View more	Positive	05 Nov 2021

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