Phase IV Multi-Center, Prospective, Interventional, Post-Marketing Study in Hemophilia B Patients in India Receiving RIXUBIS as On-demand or Prophylaxis Under Standard Clinical Practice

The purpose of this study is to characterize the safety and describe the effectiveness of RIXUBIS in routine clinical practice.

Start Date07 Dec 2018

Sponsor / Collaborator

Baxalta, Inc.

[+1]

CTRI/2018/07/014754 / Active, not recruitingPhase 4

Phase IV Multi-center, Prospective, Interventional, Post-marketing study in Hemophilia B Patients in India receiving RIXUBIS as on-demand or Prophylaxis under standard clinical practice - None

Start Date05 Jul 2018

Sponsor / Collaborator

Baxalta Innovations GmbH

[+1]

NCT02922231 / CompletedNot Applicable

Post-Marketing Surveillance (Usage Results Study) of RIXUBIS in Adult and Pediatric Patients With Haemophilia B in South Korea

Primary: To characterize the safety of RIXUBIS when used under normal clinical care in South Korea.
Secondary: To describe hemostatic effectiveness in subjects receiving RIXUBIS under normal clinical care in South Korea.

Start Date06 Jan 2017

Sponsor / Collaborator

Baxalta, Inc.

100 Clinical Results associated with Coagulation Factor IX (Recombinant)

100 Translational Medicine associated with Coagulation Factor IX (Recombinant)

100 Patents (Medical) associated with Coagulation Factor IX (Recombinant)

308

Literatures (Medical) associated with Coagulation Factor IX (Recombinant)

01 Oct 2024·CPT-Pharmacometrics & Systems Pharmacology

PBPK modeling of recombinant factor IX Fc fusion protein (rFIXFc) and rFIX to characterize the binding to type 4 collagen in the extravascular space

Article

Author: Lenting, Peter. J. ; Cnossen, Marjon H. ; Cloesmeijer, Michael E. ; Mathôt, Ron A. A. ; Koopman, Sjoerd F. ; Sjögren, Erik

Abstract:

Patients with severe and sometimes moderate hemophilia B are prophylactically treated with factor IX concentrates to prevent bleeding. For some time now, various extended terminal half‐life (EHL) recombinant factor IX concentrates are available allowing less frequent administration during prophylaxis in comparison to standard half‐life recombinant FIX (rFIX). Especially, recombinant FIX‐Fc fusion protein (rFIXFc; Alprolix®) exhibits a rapid distribution phase, potentially due to binding to type IV collagen (Col4) in the extravascular space. Studies suggest that the presence of extravascular rFIXFc is protective against bleeding as without measurable FIX activity in plasma, and no extra bleeding seems to occur. The physiologically based pharmacokinetic (PBPK) model for rFIXFc which we describe in this study, is able to accurately predict the observed concentration‐time profiles of rFIXFc in plasma and is able to quantify the binding of rFIXFc to Col4 in the extravascular space after an intravenous dose of 50 IU/kg rFIXFc in a male population. Our model predicts that the total AUC of rFIXFc bound to Col4 in the extravascular space is approximately 19 times higher compared to the AUC of rFIXFc in plasma. This suggests that rFIXFc present in the extravascular compartment may play an important role in achieving hemostasis after rFIXFc administration. Further studies on extravascular distribution of rFIXFc and the distribution profile of other EHL‐FIX concentrates are needed to evaluate the predictions of our PBPK model and to investigate its clinical relevance.

01 Jul 2024·JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Characterization of recombinant factor IX fusion proteins enabling subcutaneous administration

Article

Author: Seyfert-Brandt, Waltraud ; Rezvani-Sharif, Alireza ; Schön, Kathrin ; Herzog, Eva ; Pestel, Sabine ; Claar, Philipp ; Riedesel, Julia ; Nolte, Marc W ; Ponnuswamy, Padmapriya

BACKGROUND:

The X-linked bleeding disorder Hemophilia B, caused by mutation(s) in the coagulation factor IX (FIX) gene, leads to partial or total loss of its function requiring lifelong FIX replacement therapy. Although new recombinant FIX (rIX) therapeutics like albumin-fusion proteins (rIX-FP) enable longer plasma half-life and thus less frequent administration, the complexity of intravenous (IV) injection remains.

OBJECTIVES:

The study aims to characterize rIX-FP variants with anticipated enhanced specific activity, which would leverage rIX-FP's superior pharmacokinetic (PK) profile with beneficial characteristics for subcutaneous (SC) administration.

METHODS:

Two rIX-FP variants, R338L ("Padua-variant") and R338L/E410K, were characterized in vitro. PK profiles of FIX antigen and activity levels were evaluated in FIX-deficient mice after IV and SC administration of these variants (dosing based on antigen levels). The efficacy of the most promising variant was tested after IV and SC administration (dosing based on activity) in a tail-clip bleeding model. A marketed wildtype (WT) rIX-FP product served as the comparator.

RESULTS:

Both rIX-FP variants showed a 4- to 5-fold increase in specific activity in vitro compared to rIX(WT)-FP, whilst FXIa-mediated activation was the fastest for rIX(WT)-FP and rIX(R338L)-FP. Compared to rIX(WT)-FP and rIX(R338L/E410K)-FP, rIX(R338L)-FP exhibited higher FIX activity exposure after IV and SC administration, and demonstrated comparable efficacy towards rIX(WT)-FP in reducing bleeding time and blood loss in FIX-deficient mice requiring ∼4 times lower protein amount.

CONCLUSIONS:

rIX(R338L)-FP was shown to be a promising candidate for SC administration, exhibiting increased specific activity combined with higher activity-based exposure, and indicating efficacy at lower protein dose.

01 Jun 2024·Advances in therapy

Cost-Effectiveness of Recombinant Factor IX Fc Prophylaxis and Recombinant Factor IX On-Demand Treatment in Patients with Haemophilia B Without Inhibitors

Article

Author: Eriksson, Daniel ; Pochopien, Michal ; Martone, Nicoletta ; Tytuła, Anna ; Hakimi, Zalmai ; Toumi, Mondher ; Falk, Aletta

INTRODUCTION:

Recombinant factor IX (rFIX) and recombinant FIX Fc fusion protein (rFIXFc) are standard half-life and extended half-life FIX replacement therapies, respectively, and represent established treatment options indicated for adults and children with haemophilia B. These FIX replacement therapies can be administered as prophylaxis (to prevent bleeding) or 'on-demand' (to stop bleeding). This analysis aimed to estimate the cost-effectiveness of once-weekly prophylaxis with rFIXFc versus on-demand treatment with rFIX in patients with haemophilia B without inhibitors in the Italian healthcare setting.

METHODS:

A Markov model was developed to assess a hypothetical cohort of adolescent or adult male patients (≥ 12 years) with haemophilia B (FIX level of ≤ 2 IU/dL) without inhibitors. Model inputs were derived from the pivotal phase 3 clinical studies for rFIXFc and rFIX, published literature and assumptions when published data were unavailable. The model employed a lifelong time horizon with 6-monthly transitions between health states, and it estimated total costs, total quality-adjusted life years (QALYs), number of bleeds, number of surgeries and incremental cost-effectiveness ratio.

RESULTS:

rFIXFc prophylaxis was associated with lower total costs per patient (€5,308,625 versus €6,564,510) and greater total QALYs per patient (15.936 versus 11.943) compared with rFIX on-demand; rFIXFc prophylaxis was therefore the dominant treatment strategy. The model also demonstrated that rFIXFc prophylaxis was associated with fewer incremental bleeds (- 682.29) and surgeries (- 0.39) compared with rFIX on-demand.

CONCLUSIONS:

rFIXFc prophylaxis provides improved health outcomes and lower costs, and represents a cost-effective treatment option compared with rFIX on-demand for adolescent and adult male patients with haemophilia B. This comparative assessment of cost-effectiveness should help to inform both clinicians and healthcare policy makers when making treatment decisions for patients with haemophilia B.

News (Medical) associated with Coagulation Factor IX (Recombinant)

04 Jan 2024

·www.biospace.com

Be Biopharma Expands Leadership Team as Company Transitions to Clinical Stage Organization

Angus Smith joins as Chief Financial Officer Wing-Yen Wong, M.D., joins as interim Chief Medical Officer CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Be Biopharma, Inc. (“Be Bio”), a company pioneering the development of engineered B Cell Medicines (BCMs), today announced the appointment of Angus Smith as Chief Financial Officer (CFO) and Wing-Yen Wong, M.D., as interim Chief Medical Officer (CMO). Mr. Smith brings more than 15 years of experience in the biotechnology and life sciences industry, having held C-level positions in several public companies, including his most recent role as the CFO of Affimed N.V. and co-President of the company’s U.S. subsidiary, where he was responsible for implementing a new business plan and for raising over $400M in capital to support clinical development efforts. Mr. Smith also brings nearly a decade of investment banking experience, providing strategic and financial advice across the healthcare sector. Dr. Wong brings over 35 years of experience as a practicing clinician and professor at Children’s Hospital of Los Angeles, and leading development efforts in several companies including BioMarin, Biogen, and Baxter. Most recently Dr. Wong was Group Vice-President, Head of Global Medical Affairs and Scientific Strategy at BioMarin where she was involved in the development of the most recently approved hemophilia gene therapy. “We are thrilled to welcome Angus and Wing to Be Bio,” said Joanne Smith-Farrell, Ph.D., Chief Executive Officer. “We are rapidly transitioning from a research-based company to one focused on delivering innovative medicines to patients, with our first program in hemophilia B entering the clinic this year. These accomplished and experienced leaders will further strengthen our leadership team. Angus’s long track record of financial operating experience and fundraising will be important as we advance multiple programs to the clinic, while Wing’s expertise in drug development and as a clinical scientist is particularly important as we pioneer a new class of cellular medicines, Engineered B Cell Medicines (BCMs), to dramatically improve the lives of patients.” Angus Smith Mr. Smith brings more than 15 years of experience in the biotechnology and life sciences industry, including his most recent role as the CFO of Affimed N.V. and, prior to that, at Rockwell Medical. At Affimed, Mr. Smith led all finance, accounting, and investor relations functions, implemented a new business plan and financing strategy, and raised more than $400 million. Mr. Smith began his career in healthcare investment banking, serving most recently as a Director in the Healthcare Investment Banking Group at Cantor Fitzgerald. During his nearly decade-long investment banking tenure, Mr. Smith provided strategic and financial advice to life sciences and healthcare companies. He has worked on a substantial number of transactions across the healthcare sector with an aggregate transaction value of more than $15 billion. Mr. Smith holds a B.A. in Mathematical Economics from Colgate University. Wing-Yen Wong, M.D. Dr. Wong brings over 35 years of experience as a practicing clinician and working in the biotechnology industry with a focus in the treatment and management of patients with hemophilia, sickle cell disease (SCD) and pediatric hematology/oncology. Most recently Dr. Wong was Group Vice-President, Head of Global Medical Affairs and Scientific Strategy at BioMarin and Vice President, Global Medical, Hematology and Immunology at Biogen. Prior to Biogen, Dr. Wong was the Head of Clinical Research and Global Senior Medical Director of Hemophilia/Hematology at Baxter Healthcare Corporation where she directed medical and clinical functions, including global medical support of licensed products, clinical development and new product approvals. During her tenure at Baxter, Dr. Wong successfully directed the BLA submission and launch of multiple hemophilia products including RIXUBIS®, FEIBA®, OBIZUR and ADVATE®. Prior to her work in industry, Dr. Wong spent over 15 years as a practicing clinician and professor at Children’s Hospital Los Angeles and the Los Angeles County (LAC) and University of Southern California (USC) Medical Center. As a clinician, Dr. Wong presented and published numerous studies and papers on her research in hemophilia and hematology and has served as a reviewer for medical journals including the American Journal of Hematology, Hemophilia and the Lancet. In 2003, Dr. Wong was appointed by the Secretary of Health and Human Services as a member of the Advisory Committee on Blood Safety and Availability. Dr. Wong earned her M.D. at the USC, Keck School of Medicine and a BA at Occidental College. She served a Fellowship at the LAC and USC Medical Center in pediatric hematology-oncology and was appointed Associate Professor of Pediatrics at the University of Southern California. About Engineered B Cell Medicines – A New Class of Cellular Medicines The B cell is a powerful cell that produces thousands of proteins per cell per second at constant levels, over decades. Precision genome editing can now be used to engineer B Cells that produce therapeutic proteins of interest, driving a new class of cellular medicines – Engineered B Cell Medicines (BCMs) – with the potential to be durable, allogeneic, redosable and administered without pre-conditioning. The promise of BCMs could transform therapeutic biologics with broad application – across protein classes, patient populations and therapeutic areas. About Be Biopharma Be Biopharma (“Be Bio”) is pioneering Engineered B Cell Medicines (BCMs) to dramatically improve the lives of patients who are living with Hemophilia B and other genetic diseases, cancer, and other serious conditions. With eyes locked on the patient, our team of purpose-driven scientists, technologists, manufacturing experts and business builders collaborate to create a bold new class of cell therapies. Be Bio was founded in October 2020 by B cell engineering pioneers David Rawlings, M.D., and Richard James, Ph.D., from Seattle Children’s Research Institute. Be Bio is backed by ARCH Venture Partners, Atlas Venture, RA Capital Management, Alta Partners, Longwood Fund, Bristol Myers Squibb, Takeda Ventures, Seattle Children’s Research Institute and others. Since our founding, Be Bio’s investors have committed over $180 million to enable the Company to re-imagine medicine based on the power of B cell therapy. For more information, please visit us at Be.Bio and our LinkedIn page. View source version on businesswire.com: Contacts Investor Contact: ir@be.bio Source: Be Biopharma, Inc. View this news release online at:

Executive ChangeASHCell Therapy

18 Aug 2023

·www.biospace.com

Medexus Study Supports Efficacy and Safety of IXINITY in Pediatric Patients with Severe or Moderately Severe Hemophilia B

Poster presentation at National Hemophilia Foundation (NHF) Bleeding Disorders Conference 2023 highlights effective prophylaxis, control of bleeding episodes, and consistent safety pro > Phase 3/4 data presentation follows FDA acceptance for review of IXINITY® supplemental Biological License Application for pediatric patients Toronto, Ontario and Chicago, Illinois--(News - August 17, 2023) - Medexus Pharmaceuticals (TSX: MDP) (OTCQX: MEDXF) today announced the presentation of data from a Phase 3/4 study that evaluated the pharmacokinetics (PK), safety, and efficacy of IXINITY, an intravenous recombinant factor IX therapeutic, as a prophylactic treatment in previously treated pediatric patients under 12 years of age with severe or moderately severe hemophilia B, a hereditary bleeding disorder characterized by a deficiency of clotting factor IX in the blood. In a poster presentation at the National Hemophilia Foundation (NHF) Bleeding Disorders Conference 2023 in National Harbor, Maryland, researchers reported that prophylaxis with IXINITY was associated with low annualized bleeding rates (ABRs), effective control of bleeding episodes, consistent PK, and a consistent safety profile. See "About the study" below for additional information about the study's methods, results, and conclusions. IXINITY is currently approved for use in patients 12 years of age or older with hemophilia B. "This study demonstrated the efficacy of IXINITY in the prevention and control of bleeding episodes in pediatric subjects while providing a favorable safety profile," commented Johnny Mahlangu, BSc, MBBCh, MMed, FCPath, the study's principal investigator. "The results bolster the overall utility of this recombinant factor IX agent in the treatment of individuals with hemophilia B." "The data presented at BDC 2023 add to the growing body of evidence of IXINITY's efficacy and safety," said Mark Fosdal, DHSc, PA-C, Director of Scientific Communications at Medexus and a coauthor of the study. "Subject to FDA approval of the pediatric indication, the accumulated data should give clinicians greater confidence in administering IXINITY to maintain reliably high factor IX levels long-term, for all patients of all ages with hemophilia B." About the study The Phase 3/4 study was a single-arm, open-label clinical trial evaluating the PK, safety, and efficacy of IXINITY prophylaxis in 21 previously treated patients less than 12 years of age with severe (factor IX activity <1%; n = 13) or moderately severe (factor IX activity 1% to <2%; n = 8) hemophilia B. The prophylaxis dose was based on IXINITY recovery from PK assessments, after a single intravenous infusion dose of 75 ± 5 IU/kg, and was within the recommended prophylaxis dose of 35-75 IU/kg, twice weekly. The hemostatic efficacy of IXINITY was determined by calculating the ABR while on prophylaxis to prevent bleeding episodes, the number of infusions used to treat bleeding episodes, and overall ratings of efficacy by participants and investigators. Safety was evaluated by assessment of adverse events (AEs), physical examinations, vital signs, and laboratory assessments. The mean ABR for the entire study was 2.34 ± 4.226 (median = 0.86, range = 0-18.7) for all bleeds and 0.63 ± 1.257 (median = 0, range = 0-4.7) for spontaneous bleeds. One-third (33.3%) of participants had no bleeds and more than sixty percent (61.9%) had zero spontaneous bleeds. Participants rated the hemostatic efficacy of IXINITY as "excellent" or "good" in 78.8% of all episodes. Overall, investigators rated IXINITY prophylaxis as "effective"; there were three ratings of "partially effective" during the study. All investigator ratings for control over bleeding episodes were "effective". Of the bleeding episodes that required treatment, 83.8% resolved after one or two infusions. There appeared to be no clinically important differences in results for participants younger than 6 years of age compared to those between 6 and 12 years of age. The younger age group experienced lower mean incremental recovery of clotting factor (0.731 vs 0.849 IU/dL:IU/kg) accompanied by a higher mean clearance (7.3 vs 6.1 mL/[kg*hr]), consistent with the PK pattern of factor IX in children aged 12 and younger when compared to use in adolescents and adults. Overall, 76.2% of participants had at least one AE; the most frequently reported AEs were nasopharyngitis (23.8%) and bronchitis (14.3%). Only one AE was considered possibly treatment-related: a non-serious, moderate hypersensitivity reaction that led to the participant's withdrawal from the study. About IXINITY® IXINITY® [coagulation factor IX (recombinant)] Lyophilized Powder for Solution for Intravenous Injection is a coagulation factor IX (recombinant) indicated in adults and children ≥12 years of age with hemophilia B for on-demand treatment and control of bleeding episodes, perioperative management, and for routine prophylaxis to reduce the frequency of bleeding episodes. The most common adverse reaction observed in >2% of patients in clinical trials was headache. For more information about IXINITY®, including important safety information, see the full prescribing information, which is available on the product's website at: . IXINITY® is approved by the FDA for sale and use in the United States only and is not intended for export outside the United States. Medexus makes no representation that IXINITY® is appropriate for, or authorized for sale to or use by, persons who are not located in the United States. About hemophilia B Hemophilia B is a genetic bleeding disorder caused by a deficiency of coagulation factor IX, a protein needed to produce blood clots to stop bleeding. The clinical spectrum may include spontaneous or trauma-induced bleeding into joints, muscles, and soft tissues, resulting in joint damage, reduction in mobility, and severe arthritis, all of which negatively impact health-related quality of life. The primary aim of care is to prevent and treat bleeding by replacing the deficient clotting factor. About Medexus Medexus is a leading specialty pharmaceutical company with a strong North American commercial platform and a growing portfolio of innovative and rare disease treatment solutions. Medexus's current focus is on the therapeutic areas of oncology, hematology, rheumatology, auto-immune diseases, allergy, and dermatology. For more information about Medexus and its product portfolio, please see the company's corporate website at and its filings on SEDAR+ at . Contacts Ken d'Entremont | CEO, Medexus Pharmaceuticals Tel: 905-676-0003 | Email: ken.dentremont@medexus.com Marcel Konrad | CFO, Medexus Pharmaceuticals Tel: 312-548-3139 | Email: marcel.konrad@medexus.com Forward-looking statements Certain statements made in this news release contain forward-looking information within the meaning of applicable securities laws (forward-looking statements). The words "anticipates", "believes", "expects", "will", "plans", "potential", and similar words, phrases, or expressions are often intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words, phrases, or expressions. Specific forward-looking statements contained in this news release include, but are not limited to, statements regarding the potential benefits of IXINITY®, the occurrence, timing, and expected outcome of the FDA review process for Medexus's supplemental Biological License Application for IXINITY®, and expectations regarding the product's prospects including if approved by the FDA. These statements are based on factors or assumptions that were applied in drawing a conclusion or making a forecast or projection, including assumptions based on historical trends, current conditions, and expected future developments, and, in particular, Medexus's interpretation and assessment of the data presented by Medexus as summarized in this news release. Since forward-looking statements relate to future events and conditions, by their very nature they require making assumptions and involve inherent risks and uncertainties. Medexus cautions that although it is believed that the assumptions are reasonable in the circumstances, these risks and uncertainties give rise to the possibility that actual results may differ materially from the expectations set out in the forward-looking statements. Material risk factors include, but are not limited to, those set out in Medexus's materials filed with the Canadian securities regulatory authorities from time to time, including Medexus's most recent annual information form and management's discussion and analysis. In addition, specific risks and uncertainties relevant to the content of this news release include, among other things, the uncertainties inherent in research initiatives (including the possibility of unfavorable new data and further analyses of existing data); the risk that data are subject to differing interpretations and assessments by regulatory authorities and/or other relevant third parties; and whether regulatory authorities and/or other relevant third parties will be satisfied with the design and methodology of and results from the relevant study, which will depend on many factors, including determinations as to whether the product's benefits outweigh its known risks. Accordingly, undue reliance should not be placed on these forward-looking statements, which are made only as of the date of this news release. Other than as specifically required by law, Medexus undertakes no obligation to update any forward-looking statements to reflect new information, subsequent or otherwise. Additional notes IXINITY®, as discussed in this news release, is a trademark of Medexus. Solely for convenience, trademarks may appear without the "™", "®", or similar symbols. Each such reference should be read as though it appears with the relevant symbol. Any such references are not intended to indicate, in any way, that the holder or holders of the relevant intellectual property rights will not assert those rights to the fullest extent under applicable law. The information in this news release is provided for informational purposes to investors in Medexus securities. Medexus sponsored the publication referenced in this news release, and Medexus employees and paid consultants participated in the authoring and editing of the publication. Uniform resource locators, or website addresses, that appear in this news release are intended to be provided as inactive textual references only. Information contained on or accessible through these website addresses is not a part of this news release and is not incorporated by reference into this news release or any of Medexus's public filings. To view the source version of this press release, please visit

Clinical ResultDrug Approval

06 Jul 2021

·www.globenewswire.com

Catalyst Biosciences Announces Presentations at the International Society on Thrombosis and Haemostasis

SOUTH SAN FRANCISCO, Calif., July 06, 2021 (GLOBE NEWSWIRE) -- Catalyst Biosciences, Inc. (NASDAQ: CBIO) today announced four poster presentations on marzaptacog alfa (activated) (MarzAA) and dalcinanonog alfa (DalcA) at the upcoming International Society on Thrombosis and Haemostatis (ISTH) Virtual Congress being held from July 17-21, 2021. Poster TitleDose selection for subcutaneous marzaptacog alfa (activated) in subjects with Factor VII deficiency using population pharmacometrics clinical trial simulationsAuthorTom Knudsen, D.V.M., Ph.D., vice president, corporate development, Catalyst Biosciences Poster titleDose selection of marzaptacog alfa (activated) in children with Hemophilia: a population pharmacokinetic exposure matching strategyAuthorTom Knudsen, D.V.M., Ph.D., vice president, corporate development, Catalyst Biosciences Poster titleSubcutaneous marzaptacog alfa (activated) is effective treatment of bleeding in FVII deficient ratsAuthorTom Knudsen, D.V.M., Ph.D., vice president, corporate development, Catalyst Biosciences Poster titleMitigation of injection site reactions after subcutaneous administration of dalcinanonog alfa (DalcA) in Hemophilia B using preclinical modelsAuthorNatacha Le Moan, senior director, translational research, Catalyst Biosciences A copy of the presentation materials can be accessed on the Events and Presentations section of the Catalyst website once the congress begins. About Catalyst Biosciences, the Protease Medicines companyCatalyst is a research and clinical development biopharmaceutical company focused on addressing unmet medical needs in rare disorders of the complement and coagulation systems. Our protease engineering platform has generated two late-stage clinical programs, including MarzAA, a SQ-administered next-generation engineered rFVIIa for the episodic treatment of bleeding in subjects with rare bleeding disorders and dalcinonacog alfa (DalcA) a SQ-administered next-generation engineered rFIX for prophylaxis of bleeding in subjects with Hemophilia B. Our complement pipeline includes a preclinical C3-degrader program licensed to Biogen for dry age-related macular degeneration, an improved complement factor I protease for SQ replacement therapy in patients with CFI deficiency, and C4b-degraders designed to target disorders of the classical complement pathway, as well as other complement programs in discovery. Forward-Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties. Forward-looking statements include statements about the potential benefits of products based on Catalyst's engineered protease platform. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially, including, but not limited to, the risk that the one or both of the clinical trials of MarzAA may be delayed or terminated as a result of COVID-19, competitive products and other factors, that trials may not have satisfactory outcomes, that additional human trials will not replicate the results from earlier trials, that potential adverse effects may arise from the testing or use of MarzAA, including the generation of neutralizing antibodies, the risk that costs required to develop or manufacture the Company's products will be higher than anticipated, including as a result of delays in trial enrollment, development and manufacturing resulting from COVID-19 and other factors, competition and other risks described in the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on May 6, 2021, and in other filings with the Securities and Exchange Commission. The Company does not assume any obligation to update any forward-looking statements, except as required by law. Contact:Ana KaporCatalyst Biosciences, Inc.investors@catbio.com

Antibody

100 Deals associated with Coagulation Factor IX (Recombinant)

External Link

KEGG	Wiki	ATC	Drug Bank
D10538	-	B02BD04	DB00100

R&D Status

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Hemophilia B	AU	Takeda Pharmaceuticals Australia Pty Ltd.	05 Feb 2014
Influenza B virus infection	US	Takeda Pharmaceuticals U.S.A., Inc.	26 Jun 2013

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03565237 (CTgov)	Phase 4	Hemophilia B	25	RIXUBIS	qfbglvmhyl(ripovtkkqw) = ygwrtbkvha gnoilbzjqr (isgtcwodon, syrdixfwlz - tpxqopnttv) View more	-	30 Aug 2022
ISTH2019	Not Applicable	Hemophilia B	-	recombinant factor IX	ddgdholdze(pdqzronxkb) = vgkehzoroq lomunxmqgq (tjjwjzyoiy ) View more	-	10 Jun 2019
NCT01488994 (Pubmed \| Haemophilia) Manual	Phase 2/3	Hemophilia B	23	BAX326	esvfweuauv(sndrqvbsvi) = flxxsmygnb ajazbeqqio (qqafuoufnn, 3.14) View more	Positive	01 Mar 2015

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