A Multinational, Prospective, Open-label, Roll-over Study (LIBERTY) to Provide Post-trial Access to Treatment for Patients With Severe Haemophilia A Who Have Completed a Previous Trial With Efanesoctocog Alfa

This is a multinational, prospective, open-label, roll-over study in patients with severe haemophilia A, ≥6 years of age, who have completed participation in any of the parental studies with efanesoctocog alfa; XTEND-ed study (LTS16294), FREEDOM study (Sobi.BIVV001-001), or PK comparison study (Sobi.BIVV001-003). The aim of the study is to provide patients with continuous benefit from efanesoctocog alfa treatment and to further continue clinical monitoring for safety and efficacy until efanesoctocog alfa is commercially available in each patient's respective country (or until March 2027, whichever comes first).
The study starts with the Baseline Visit, which will be done in connection to the End of Study visit (or equivalent) in the respective parent study. Subsequent study visits (on site or phone call) will be done approximately every 13 weeks until End of Treatment. An End of Study safety phone call will be done 14 (+7) days after the End of Treatment Visit.

Start Date01 Jan 2025

Sponsor / Collaborator

Swedish Orphan Biovitrum AB

[+1]

NCT06684314 / RecruitingNot Applicable

PROphylactic Efanesoctocog Alfa Therapy Evaluated for Critical Joint Health in Hemophilia A Treatment: The PROTECT-ALT Study

This is a national, multicenter, retrospective/prospective, observational study in Taiwan designed to assess effectiveness, safety, and usage of efanesoctocog alfa prophylaxis treatment in hemophilia A participants. The data related to efanesoctocog alfa effectiveness, safety and usage will be recorded prospectively during routine visits for up to 5 years following enrollment initiation and the retrospective data will be collected at least 12 months and up to 24 months prior to efanesoctocog alfa initiation. Joint imaging data will be collected in centers performing Joint U/S and/or MRI (≥6 years old). At least 12 months of retrospective data will also be collected from medical records, as available. Prospectively collected data will be recorded at routine clinical visits during a five-year follow-up period. The end of study is defined as the last participant's last visit. No intervention will be administered, and no study related visits are required.

Start Date11 Nov 2024

Sponsor / Collaborator

Sanofi

NCT06579144 / Not yet recruitingPhase 1

An Open-label, Multicentre Study to Compare the Pharmacokinetics of Efanesoctocog Alfa Versus rFVIII Products, Damactocog Alfa Pegol or Turoctocog Alfa Pegol, After a Single Intravenous Dose of 50 IU/kg in a Fixed Sequence in Previously Treated Adults With Severe Haemophilia A.

Sobi.BIVV001-003 is an open-label, 2-period, fixed sequence study for intra-participant comparison of the PK profiles of efanesoctocog alfa and the extended half-life rFVIII products damactocog alfa pegol or turoctocog alfa pegol after a single i.v. injection in previously treated males, 18-65 years of age, with severe haemophilia A.
Participants who are receiving treatment with damoctocog alfa pegol (n
12) or turoctocog alfa pegol (n
12) will be enrolled in the study. The study will start with a screening period (up to 28 days), including a wash-out period prior to start of the actual study period.
During the the first visit, a single dose of damactocog alfa pegol or turoctocog alfa pegol (corresponding to the participant's pre-study treatment) will be administered. A PK sampling period will follow over 7 visits. Following completion of the PK sampling of the original treatment regimen, the patients will be given a single dose of efanesoctocog alfa at visit 8, after which a new PK sampling period will follow (visit 8-15).
The primary objective for the study is to compare the half-life of efanesoctocog alfa with that of the two comparator drugs after a single iv. injections.
Secondary objectives include comparison of area under the curve for efanesoctocog alfa vs. the two comparator drugs, characterization of PK parameters for all three drugs as well as well as to evaluate safety and tolerability of a single iv. injection of efanesoctocog alfa.

Start Date01 Nov 2024

Sponsor / Collaborator

Swedish Orphan Biovitrum AB

[+1]

100 Clinical Results associated with Efanesoctocog alfa

100 Translational Medicine associated with Efanesoctocog alfa

100 Patents (Medical) associated with Efanesoctocog alfa

Literatures (Medical) associated with Efanesoctocog alfa

01 Dec 2024·CLINICAL THERAPEUTICS

Patient Experience With Efanesoctocog Alfa for Severe Hemophilia A: Results From the XTEND-1 Phase 3 Clinical Study Exit Interviews

Article

Author: Pan-Petesch, Brigitte ; Willemze, Annemieke ; DiBenedetti, Dana ; Wilson, Amanda ; Wynn, Tung ; Neme, Daniela ; Kragh, Nana

PURPOSE:

Hemophilia A is a rare bleeding disorder that leads to recurrent hemarthrosis, which can ultimately result in reduced mobility and poor quality of life. Qualitative exit interviews provide insights into patient perspectives and support the interpretation of quantitative trial data, such as patient-reported outcome measures. In the Phase 3 XTEND-1 study (NCT04161495) of efanesoctocog alfa in participants with severe hemophilia A, exit interviews were conducted to understand pre- and post-study experiences with pain and physical functioning and to evaluate participants' treatment experiences.

METHODS:

In XTEND-1, participants (≥12 years old) received once-weekly efanesoctocog alfa prophylaxis 50 IU/kg for 52 weeks (Arm A) or on-demand efanesoctocog alfa 50 IU/kg for 26 weeks followed by 26 weeks once-weekly prophylaxis (50 IU/kg; Arm B). Optional qualitative exit interviews were conducted using a semi-structured guide in a subset of participants following study completion. Interviews included open-ended questions about participants' pre- and post-study experiences with hemophilia A and targeted questions relating to improvements in patient-reported outcomes assessed during XTEND-1, including the Haemophilia Quality of Life Questionnaire for Adults Physical Health subscale (Haem-A-QoL PH). Content validity of the Patient-Reported Outcomes Measurement Information System (PROMIS) Pain Intensity 3a measure was also assessed, particularly the worst pain item.

FINDINGS:

Exit interviews were conducted with 29 of 159 patients enrolled in XTEND-1 (mean [range] age 40 [16-73] years). Of 17 participants enrolled in Arm A, 13 (76.5%) reported a "wearing off" feeling with pre-study treatment, including more aches/pain, breakthrough bleeds, and limited physical activities. Joint pain was the most reported pre-study symptom (96.6%; n = 28/29), followed by a reduced ability to move without pain (89.7%, n = 26/29). Improvements following efanesoctocog alfa prophylaxis in ≥1 Haem-A-QoL PH domain were reported by 89.7% (n = 26/29) of participants, with improvements in joint pain, the ability to move without pain, and painful swellings reported by at least 21 (84%) participants. Participants reported that the PROMIS Pain Intensity 3a items were relevant, clear, and easy to answer. Most participants (96.6%) were "quite satisfied" or "very satisfied" with efanesoctocog alfa prophylaxis. All participants preferred efanesoctocog alfa over pre-study treatment.

IMPLICATIONS:

The exit interviews demonstrated that once-weekly efanesoctocog alfa prophylaxis resulted in patient-relevant and meaningful improvements in pain and physical functioning, consistent with the quantitative findings from XTEND-1. These results support the validity of the Haem-A-QoL PH and PROMIS Pain Intensity 3a assessed during XTEND-1, demonstrating the potential for change with efficacious treatment.

TRIAL REGISTRY:

ClinicalTrials.gov TRIAL REGISTRATION NUMBER: NCT04161495 REGISTRY URL: https://clinicaltrials.gov/study/NCT04161495.

01 Aug 2024·PEDIATRIC BLOOD & CANCER

Practical challenges associated with efanesoctocog alfa (ALTUVIIIO) prophylaxis in a 19‐month‐old male with severe hemophilia A

Letter

Author: Español, Maria G. ; Christ, Dane C. ; Tarantino, Michael D. ; Mistretta, Jessica N. ; Roberts, Jonathan C. ; Jones, Maureen D.

23 Jul 2024·EXPERT OPINION ON PHARMACOTHERAPY

Factor VIII stimulants and other novel therapies for the treatment of von Willebrand disease: what’s new on the horizon?

Review

Author: Sidonio, Robert F. ; Regling, Katherine

INTRODUCTION:

Von Willebrand disease (VWD) is the most common inherited bleeding disorder, affecting about 0.6% to 1.3% of the population, and is characterized primarily by mucocutaneous bleeding secondary to defective platelet adhesion and aggregation. Current therapeutic options for those with severe disease are limited and require frequent intravenous infusions.

AREAS COVERED:

This review discusses the current and recently completed clinical trials involving pathways to FVIII augmentation for the treatment of VWD. Clinical trials registered on clinicaltrials.gov and published data via PubMed searches through June 2024 were included.

EXPERT OPINION:

Available treatment options to those with VWD are limited in part due to limited clinical trials, the complexity of VWD types, and the pharmacokinetics of current treatment options. The development of therapeutic options that reduce treatment burden is necessary to improve quality of life and reduce bleeding complications and in recent years there has been an increased interest from industry to apply novel therapeutics for VWD. The FVIII mimetic, emicizumab, has demonstrated early success in patients with severe VWD and is a promising treatment option for those who require prophylaxis. Furthermore, products like efanesoctocog alfa (Altuviiio®) and BT200 have achieved enhanced VWF/FVIII half-life extension could expand the current treatment landscape while concurrently minimizing treatment burden.

News (Medical) associated with Efanesoctocog alfa

03 Dec 2024

·www.prnewswire.com

Sobi's strength in haematology to be showcased at ASH 2024

STOCKHOLM, Dec. 3, 2024 /PRNewswire/ -- New data from Sobi® and partners will be presented at the 66th Annual Meeting of the American Society of Hematology (ASH) in San Diego, CA (USA) from the 7th - 10th of December 2024. During the meeting several analyses will be presented on haemophilia A, haemophagocytic lymphohistiocytosis (HLH), myelofibrosis, paroxysmal nocturnal haemoglobinuria (PNH), and immune thrombocytopenia (ITP). "Haemophilia A has a lasting impact on joint health and quality of life. At ASH 2024, three oral presentations will reveal important new data about long-term outcomes with ALTUVOCT® prophylaxis," said Lydia Abad-Franch, MD, MBA, Head of Research, Development, and Medical Affairs (RDMA), and Chief Medical Officer at Sobi. "We will also present new data spanning different severe and debilitating rare diseases, including an oral presentation on the real-world effectiveness of Doptelet® in treating people with ITP, demonstrating our broader commitment to advancing innovative treatments for people with rare haematological diseases. We look forward to sharing these findings in San Diego." Key data to be presented at ASH 2024 About ALTUVOCT® ALTUVOCT® (efanesoctocog alfa) [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein] (formerly BIVV001) is the first high-sustained FVIII replacement therapy with the potential to deliver near-normal factor activity levels for a significant part of the week, improving bleed protection in a once-weekly dose for people with haemophilia A. ALTUVOCT builds on the established Fc fusion technology by innovatively adding a region of von Willebrand factor and XTEN® polypeptides to extend its time in circulation. It is the only therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on current factor VIII therapies. The European Commission granted Orphan Drug designation in June 2019. It is approved and marketed as ALTUVOCT by Sobi in Europe. It is approved and marketed as ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl] by Sanofi in the United States, Japan, and Taiwan. About the Sanofi and Sobi collaboration Sobi and Sanofi collaborate on the development and commercialisation of Alprolix® and Elocta®/Eloctate®. The companies also collaborate on the development and commercialisation of ALTUVOCT® (efanesoctocog alfa), or ALTUVIIIO™ in the US. Sobi has final development and commercialisation rights in the Sobi territory (essentially Europe, North Africa, Russia, and most Middle Eastern markets). Sanofi has final development and commercialisation rights in North America and all other regions in the world excluding the Sobi territory. About Gamifant® Gamifant (emapalumab) is an anti-interferon gamma (IFNγ) monoclonal antibody that binds to and neutralises IFNγ. In the USA, Gamifant is indicated for the treatment of adult and paediatric (newborn and older) patients with primary haemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Primary HLH is a rare syndrome of hyperinflammation that usually occurs within the first year of life and can rapidly become fatal unless diagnosed and treated. The FDA approval is based on data from the phase 2/3 studies (NCT01818492 and NCT02069899). Gamifant is indicated for administration through intravenous infusion over one hour twice per week until haematopoietic stem cell transplantation (HSCT). About Vonjo® Vonjo (pacritinib), a JAK-1 sparing JAK2/IRAK1/ACVR1 inhibitor, is approved for the treatment of adults with intermediate- or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L in the United States. This indication is approved under FDA accelerated approval based on spleen volume reduction. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). Currently, a Phase 3 study (PACIFICA) is being conducted to assess pacritinib in patients with myelofibrosis and severe thrombocytopenia as a post-marketing requirement. About Aspaveli®/ Empaveli® Aspaveli/Empaveli (pegcetacoplan) is a targeted C3 and C3b inhibitor designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is approved for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) as Aspaveli/Empaveli in the United States, European Union, and other countries globally. Currently, a Phase 3 trial (VALIANT) is being conduted to assess the efficacy and safety of pegcetacoplan in patients with C3G and primary IC-MPGN. Aspaveli/Empaveli is also under investigation for several other rare diseases across haematology and nephrology. About the Sobi and Apellis collaboration Sobi and Apellis have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialisation rights for systemic pegcetacoplan, and Apellis has exclusive U.S. commercialisation rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy. About Doptelet® Doptelet (avatrombopag) is an orally administered thrombopoietin receptor agonist (TPO-RA) that mimics the biologic effects of TPO in stimulating the development and maturation of megakaryocytes, resulting in increased platelet count. It is approved in over 30 countries worldwide, including the EU and the US, for the treatment of severe thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo an invasive procedure, and for the treatment of thrombocytopenia in adult patients with primary chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. Chronic ITP is a rare autoimmune bleeding disorder characterised by low number of platelets. The incidence of primary ITP in adults is 3.3/100,000 adults per year with a prevalence of 9.5 per 100,000 adults. About Sobi® Sobi® is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology, and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia, and Australia. In 2023, revenue amounted to SEK 22.1 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information was brought to you by Cision The following files are available for download: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Drug ApprovalPhase 3ASHOrphan DrugLicense out/in

28 Oct 2024

·www.echemi.com

Merck's RSV Antibody Faces Major Challenges vs. Sanofi's Beyfortus Exhibiting 83% Efficacy in Reducing Hospitalization

While Merck & Co. 's infant respiratory syncytial virus (RSV) antibody clesrovimab is nearing approval, sanofi also won approval last year for its RSV prevention drug beyforts in partnership with astrazeneca. This news undoubtedly brings new hope to the global healthcare industry, especially in the field of RSV vaccines. "We would very much welcome having more participants on board because it increases the scale of the competition," Thomas Triomphe, Sanofi's executive vice president for vaccines, said of the upcoming RSV competition during an analyst call on Friday. He stressed that more players mean more innovation and better healthcare solutions, which is good for the industry as a whole and for patients. Triomphe noted that Merck still has more data on clesrovimab after announcing phase 2b/3 clinical results last week. In the meantime, Sanofi remains "very confident" in the Beyfortus numbers it has released so far, he said. He mentioned that the protection data provided by Beyfortus in six months is very encouraging, as the effect has not diminished. "After six months, we were still seeing 83 percent hospitalization efficacy," he said, adding that he believed "the duration of protection from Beyfortus was significantly more favorable compared to clesrovimab." This data suggests that Beyfortus has a significant advantage in preventing RSV infection. In Merck's infant RSV study, clesrovimab successfully reduced RSV-related hospitalizations and RSV-related lower respiratory tract infection hospitalizations by 84 percent and 91 percent, respectively, over five months compared to placebo. This result is impressive and shows the great potential of clesrovimab in preventing RSV infection. At the trial's primary endpoint, clesrovimab reduced the incidence of RSV-related medical lower respiratory tract infections by 60 percent compared to placebo. This data provides further evidence of the effectiveness of clesrovimab in preventing RSV infection. Sanofi Chief Financial Officer franois-xavier Roger noted in a conference call on Friday that the inherent uncertainty of cross-trial comparisons suggested that existing Beyfortus data showed a reduction of about 75 percent in the incidence of lower respiratory tract infections with clesrovimab compared to 60 percent with Clesrovimab. This data further reinforces Beyfortus' advantage in preventing RSV infection. And, whether or not Merck brings clesrovimab into the 2025-2026 RSV season as planned, "2025 will be a year of growth for Beyfortus," the CFO stressed. He is confident that Beyfortus' sales will grow significantly as it gradually gains market share. Since it was approved last July, Beyfortus has gradually built up its sales profile. In the third quarter of 2024, sales of this preventive antibody reached 645 million euros ($699), an increase of 382% compared to the same period in 2023. Overall, sales of the drug have reached 845 million euros since the beginning of the year, which is close to the best-selling threshold. Sanofi said at the time that Beyfortus quickly fell into a supply crunch shortly after going public last year due to "higher than expected demand." To counter this, Sanofi and its partner Astrazeneca have moved quickly to ensure there is no repeat of the supply crunch in 2024. Recently they added a new filling line and shipped antibody doses ahead of the 2024 RSV season. "There are 20 markets and we don't see any capacity constraints," Triumph said on Sanofi's conference call. "We will increase supply next year and the year after to make sure there are no supply constraints." This initiative demonstrates Sanofi's confidence in the future of Beyfortus and its commitment to meeting the needs of the global market. The comments by Sanofi executives came as the French pharmaceutical company reported a 15.7 per cent rise in third-quarter sales. This growth was mainly driven by the excellent performance of its immunology product Dupixent, which brought in sales of nearly 3.5 billion euros (about $3.8 billion), an increase of about 24 percent over the third quarter of 2023. Sanofi confirmed DuPont's full-year sales target of about 13 billion euros. Elsewhere, newly launched drugs rose 67.1 percent to 727 million euros ($787), led by hemophilia drug Altuviiio, Pompeii disease treatment Nexviazyme and Rezurock for graft-versus-host disease. The strong performance of these new drugs further strengthens Sanofi's position in the field of innovative medicines. Meanwhile, Sanofi's consumer health division, Opella, saw revenue rise 7.9 percent. This growth shows that Sanofi is also making significant progress in consumer health. Earlier this week, Sanofi confirmed it had entered exclusive talks with U.S. private equity firm Clayton, Dubilier & Rice to sell a controlling 50 percent stake in its consumer healthcare business, which is valued at around 16 billion euros. This move will further strengthen Sanofi's financial strength and provide additional financial support for its future expansion.

Drug ApprovalClinical ResultVaccine

24 Oct 2024

·www.prnewswire.com

Sobi Q3 2024 report: Strong growth and significant pipeline momentum

STOCKHOLM, Oct. 24, 2024 /PRNewswire/ -- Swedish Orphan Biovitrum AB (publ) (Sobi®) today announced its report for the third quarter 2024 Third Quarter 2024 Total revenue increased 33 per cent, 39 per cent at constant exchange rates, (CER)1, to SEK 6,894 M (5,168) Haematology revenue increased 18 per cent at CER to SEK 4,000 M (3,484), mainly driven by strong sales of Doptelet® of SEK 1,039 M (650), sales of Aspaveli®/Empaveli® of SEK 270 M (169) and launch sales of Altuvoct® of SEK 129 M (—) Immunology revenue increased 96 per cent at CER to SEK 2,583 M (1,400), driven by strong Beyfortus® royalty of SEK 1,478 M (263) and sales of Kineret® of SEK 699 M (600) Revenue from medicines in the strategic portfolio* grew by 113 per cent at CER to SEK 3,830 M (1,924) The adjusted EBITA margin1,2 was 43 per cent (30), excluding items affecting comparability (IAC)2. EBITA was SEK 2,923 M (1,443), corresponding to a margin of 42 per cent (28). EBIT was SEK 2,038 M (547) Earnings per share (EPS) before dilution was SEK 4.27 (0.30). Adjusted EPS before dilution1 was SEK 4.36 (0.54). Cash flow from operating activities was SEK 1,201 M (1,058) In August, Sobi and Apellis Pharmaceuticals announced positive topline results from the Phase 3 VALIANT study of pegcetacoplan in C3G and primary IC-MPGN Outlook 2024 - Updated Revenue is anticipated to grow by a mid-teens percentage at CER (previously low double-digit) The adjusted EBITA margin is anticipated to be in the mid-30s percentage of revenue (unchanged) Click here for the Financial Summary. Alternative Performance Measures (APMs). Items affecting comparability (IAC). * The strategic portfolio includes Sobi's medicines Altuvoct, Aspaveli/Empaveli, Doptelet, Gamifant®, Vonjo® and Zynlonta®, and royalty on Sanofi's sales of Altuviiio® and Beyfortus. Investors, analysts, and the media are invited to a conference call on the same day at 14:00 CEST, 13:00 BST, and 08:00 EDT. The call will include a presentation of the results and a Q&A session. The presentation can be followed live here or afterwards on sobi.com. The slides will be made available on sobi.com before the conference call. To participate in the conference call, please use the following dial-in details: Sweden: +46 8 5051 0031 United Kingdom: +44 207 107 06 13 United States: +1 631 570 56 13 For other countries, please find the details here. Sobi Sobi® is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia and Australia. In 2023, revenue amounted to SEK 22.1 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information is information that Sobi is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out below, at 08:00 CEST on 24 October 2024. Gerard Tobin Head of Investor Relations This information was brought to you by Cision The following files are available for download: SOURCE Swedish Orphan Biovitrum AB WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Financial StatementClinical ResultPhase 3Biosimilar

100 Deals associated with Efanesoctocog alfa

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Hemophilia A	US	Bioverativ, Inc.	22 Feb 2023

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Hemorrhage	Phase 3	US	Biogen, Inc.	23 Feb 2021
Hemorrhage	Phase 3	US	Sanofi (China) Investment Co. Ltd.	23 Feb 2021
Hemorrhage	Phase 3	US	Bioverativ Therapeutics, Inc.	23 Feb 2021
Hemorrhage	Phase 3	BE	Sanofi (China) Investment Co. Ltd.	23 Feb 2021
Hemorrhage	Phase 3	BE	Bioverativ Therapeutics, Inc.	23 Feb 2021
Hemorrhage	Phase 3	BE	Biogen, Inc.	23 Feb 2021
Hemorrhage	Phase 3	BG	Bioverativ Therapeutics, Inc.	23 Feb 2021
Hemorrhage	Phase 3	BG	Sanofi (China) Investment Co. Ltd.	23 Feb 2021
Hemorrhage	Phase 3	BG	Biogen, Inc.	23 Feb 2021
Hemorrhage	Phase 3	CA	Bioverativ Therapeutics, Inc.	23 Feb 2021

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04161495 (XTEND-1, Pubmed \| Clin Ther)	Phase 3	Hemophilia A	159	Efanesoctocog alfa prophylaxis 50 IU/kg for 52 weeks	alvoyluvgx(acqupglien) = rcmpcrmaqv zolnryjtfy (jrxfixrhns ) View more	Positive	01 Dec 2024
NCT04759131 (XTEND-Kids, PRNewswire) Manual	Phase 3	Hemophilia A	73	ALTUVOCT	hdpbtvxkax(itwriivjnl) = lkusouvrup osxtxfaole (mzxgvylzmm, 0.42–0.90) View more	Positive	18 Jul 2024
NCT04759131 (XTEND-Kids, Pubmed)	Phase 3	Hemophilia A	74	Efanesoctocog alfa	dcyvyipwki(vievikpxhp) = Most adverse events were nonserious. No serious adverse events that were assessed by the investigator as being related to efanesoctocog alfa were reported. ssfobylnez (xwfnikpron )	Positive	18 Jul 2024
NCT05042440 (Phase 1 sequential pharmacokinetic study, EHA2024) Manual	Phase 1	Hemophilia A FVIII	13	Octocog alfa	iegokuynov(wghwvrbsat) = ufsxrzssch chkkycvlwh (mgoqwqmpzs ) View more	Positive	14 May 2024
	Phase 1	Hemophilia A FVIII	13	Rurioctocog alfa pegol	iegokuynov(wghwvrbsat) = ulezxreprn chkkycvlwh (mgoqwqmpzs ) View more	Positive	14 May 2024
NCT04759131 (XTEND-Kids, CTgov)	Phase 3	Hemophilia A	74	efanesoctocog alfa (BIVV001) (BIVV001: Participants Aged <6 Years)	tmaboqovyl(yxtlsstxkl) = fwisoeregw tapjtcezje (aqqzfnssat, fmybndhxip - ndbuormvod) View more	-	13 Feb 2024
NCT04759131 (XTEND-Kids, CTgov)	Phase 3	Hemophilia A	74	efanesoctocog alfa (BIVV001) (BIVV001: Participants Aged 6 to <12 Years)	tmaboqovyl(yxtlsstxkl) = gmuxonplvw tapjtcezje (aqqzfnssat, tmzntjsbvc - prnfqhpybg) View more	-	13 Feb 2024
XTEND-1 (ISTH2023)	Phase 3	Hemophilia A	78	Standard-of-care FVIII prophylaxis	qszppnmqzn(xmnnvyzivi) = jjoejmdlbo wmrdgdxhhi (ddsugzykun )	-	24 Jun 2023
XTEND-1 (ISTH2023)	Phase 3	Hemophilia A	78	Efanesoctocog alfa 50 IU/kg prophylaxis	qszppnmqzn(xmnnvyzivi) = mxfpifbyxc wmrdgdxhhi (ddsugzykun )	-	24 Jun 2023
NCT04161495 (XTEND-1, ASH2022) Manual	Phase 3	Pain \| Hemophilia A	159	Efanesoctocog alfa (Arm A)	thjvinofiy(xwlwdxeyde) = was improved or maintained (change from baseline≤0) for most patients (81.5% [97/119]) at Week 52 apqjkydyrk (icdasqhafu )	Positive	15 Nov 2022
NCT04161495 (XTEND-1, ASH2022) Manual	Phase 3	Pain \| Hemophilia A	159	Efanesoctocog alfa (Arm B)		Positive	15 Nov 2022
NCT04161495 (XTEND-1, ASH2022) Manual	Phase 3	Hemophilia A	159	Efanesoctocog alfa	nmhfampopa(ajenydhrxp) = gqlmvmtbcy jqtepnblvg (eyreoyboap ) View more	Positive	15 Nov 2022
NCT04161495 (XTEND-1, Corporate Publications) Manual	Phase 3	Hemorrhage	150	Efanesoctocog alfa	qoldwhtksj(bysdjzcxnq) = zfyghhrwbm pgozjomuje (iqdfhhfdhv )	Positive	11 Jul 2022
XTEND-1 (ISTH2022)	Phase 3	Hemophilia A	217	Efanesoctocog Alfa (BIVV001)	tfhddsigrb(urqlqjjewt) = dfiuuohwlq hbsgntnsby (gwsazdholh )	-	09 Jul 2022

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