A Proof-of-concept study to investigate the Efficacy of Telbivudine Over Placebo in patients with Parvovirus-associated Inflammatory Cardiomyopathy - preTopic

Start Date06 Dec 2017

Sponsor / Collaborator

Charité - Universitätsmedizin Berlin

ChiCTR-OPN-17012206

/ SuspendedPhase 4IIT

Non-randomized, open clinical study of efficacy and safety of tenofovir combined with telbivudine in the treatment of HBeAg positive chronic hepatitis B patients

Start Date01 Sep 2017

Sponsor / Collaborator-

CTR20171290

/ CompletedNot Applicable

替比夫定片在健康受试者中的单剂量、空腹/餐后、随机、开放、两周期、两交叉生物等效性研究

[Translation] A single-dose, fasting/fed, randomized, open-label, two-period, two-crossover bioequivalence study of telbivudine tablets in healthy subjects

主要研究目的：本研究以四川科伦药业股份有限公司生产的替比夫定片（600mg/片）为受试制剂，原研Novartis Pharma GmbH生产的替比夫定片（600mg/片）（商品名：Sebivo）为参比制剂，评价受试制剂和参比制剂在空腹/餐后条件下给药时的生物等效性。次要研究目的：观察受试制剂和参比制剂在健康受试者中的安全性。

[Translation]

Primary study objective: This study used telbivudine tablets (600 mg/tablet) produced by Sichuan Kelun Pharmaceutical Co., Ltd. as the test preparation and telbivudine tablets (600 mg/tablet) (trade name: Sebivo) produced by the original Novartis Pharma GmbH as the reference preparation to evaluate the bioequivalence of the test preparation and the reference preparation when administered under fasting/postprandial conditions. Secondary study objective: To observe the safety of the test preparation and the reference preparation in healthy subjects.

Start Date09 Aug 2017

Sponsor / Collaborator

Sichuan Kelun Pharmaceutical Co., Ltd.

100 Clinical Results associated with Telbivudine

100 Translational Medicine associated with Telbivudine

100 Patents (Medical) associated with Telbivudine

1,665

Literatures (Medical) associated with Telbivudine

01 Nov 2025·Journal of Orthopaedics

Superior capsular reconstruction vs. Latissimus dorsi transfer for posterosuperior irreparable rotator cuff tears without arthritic change: Mid-term Crossover study

Article

Author: Baek, Chang Hee ; Kim, Seung Jin ; Kim, Bo Taek ; Lim, Chaemoon ; Kim, Jung Gon

Background:

Latissimus dorsi transfer (LDT) is a well-known tendon-transfer technique with long-term clinical follow-up results, whereas superior capsular reconstruction (SCR) has recently emerged as a treatment option for posterosuperior irreparable rotator cuff tears (PSIRCTs). However, only a few small short-term comparative studies have evaluated SCR and LDT. We evaluated the clinical and radiological outcomes of SCR and LDT in PSIRCTs without arthritic changes.

Methods:

This retrospective clinical comparative study evaluated patients who underwent SCR or LDT for PSIRCTs and had a mid-term (at least 5 years) follow-up. 28 patients in the SCR group and 31 in the LDT group were included. The visual analog scale (VAS) score, patient-reported clinical outcome scores, and active range of motion (aROM) were used for assessing clinical outcomes. The radiologic outcomes including progression of glenohumeral osteoarthritis and graft integrity were evaluated using Hamada grade and Sugaya classification, respectively.

Results:

Significant improvements in VAS scores and aROM were observed in both groups. The LDT group reported significant improvements in all patient-reported outcome scores. However, SCR group showed no significant improvement in patient-reported outcome scores except for American Shoulder and Elbow Surgeons score. In radiologic outcomes, AHD was significantly decreased, and the Hamada grade was significantly increased postoperatively in both groups. Moreover, the progression of arthritic change was significantly greater in the LDT group, while the rate of graft retear was markedly higher in the SCR group.

Conclusions:

Although both SCR and LDT improved clinical outcomes, LDT was superior to SCR in terms of patient-reported outcome scores. However, progression of osteoarthritis is more common in LDT whereas graft retear is more common in SCR. Therefore, SCR or LDT could be considered as a surgical treatment in PSIRCTs without arthritic changes, but clinical outcomes as well as complications (osteoarthritis and graft re-tear) must be considered.

Level of evidence:

Level of evidence, III.

01 May 2025·JOURNAL OF HEPATOLOGY

Real-world clinical data-driven modelling on the initiation time of antiviral prophylaxis among pregnant women with chronic hepatitis B infection

Article

Author: Wang, Huihui ; Chen, Tianyan ; Zhuang, Guihua ; Yao, Naijuan ; He, Shihao ; Wang, Jinyan ; Wang, Jing ; Li, Rui ; Shen, Mingwang ; Zhang, Lei ; Zhong, Wenting ; Xie, Li

BACKGROUND & AIMS:

The risk of mother-to-child transmission for pregnant women with chronic hepatitis B (CHB) still exists, especially for those with high HBV DNA levels. The guidelines for initiating prophylaxis for pregnant women with CHB vary across countries. We aimed to explore the latest prophylaxis initiation time for these women.

METHODS:

We collected the real-world clinical data of 328 pregnant women aged 20-49 with CHB, who were treated with telbivudine or tenofovir disoproxil fumarate, from July 2010 to December 2020 in China. A mathematical model was developed to describe the viral kinetics of HBV after prophylaxis. We calculated the time required to reduce viral load below the threshold value of 5.3 log₁₀ IU/ml. We derived the prophylaxis initiation time by subtracting the required time to threshold from the childbirth gestational week.

RESULTS:

The median time for 328 women to reduce HBV DNA levels below the threshold of 5.3 log₁₀ IU/ml was 4.2 (range: 0.2-12.8) weeks, corresponding to a prophylaxis initiation time of no later than 35.1 (25.2-41.4) weeks. Specifically, for women with viral loads >8.0 log₁₀ IU/ml, prophylaxis should be initiated before 33.9 (25.2-39.5) weeks, and even before the lower bound of 25.2 weeks, to maximize clinical safety. For women with viral load >7.0 to ≤8.0 log₁₀ IU/ml, prophylaxis should be initiated before 35.5 (28.6-39.8) weeks, and for women with viral load >5.3 to ≤7.0 log₁₀ IU/ml, prophylaxis should be initiated before 36.2 (28.3-41.4) weeks.

CONCLUSION:

Pregnant women with HBV DNA levels >5.3 to ≤8.0 log₁₀ IU/ml can initiate prophylaxis before 28 gestational weeks. However, women with HBV DNA >8.0 log₁₀ IU/ml could consider initiating prophylaxis before 25 weeks.

IMPACT AND IMPLICATIONS:

This study investigates how long it takes to decrease maternal viral load below a threshold (5.3 log₁₀ IU/ml) after receiving antiviral prophylaxis in pregnant women with different HBV DNA levels based on real-world clinical data and mathematical modelling, which provides quantitative evidence on the initiation time of antiviral prophylaxis. The results show that pregnant women with CHB infection at high HBV DNA levels (>8 log₁₀ IU/ml) should initiate antiviral prophylaxis earlier to decrease the risk of mother-to-child transmission of HBV. Physicians can determine when to begin antiviral prophylaxis for those women according to their maternal HBV DNA levels. Our findings justify the initiation time of antiviral prophylaxis recommended by the Chinese guidelines and will offer new insights for other international guidelines.

01 May 2025·EBioMedicine

A polytherapy approach demonstrates therapeutic efficacy for the treatment of SOD1 associated amyotrophic lateral sclerosis

Article

Author: McAlary, Luke ; Gorman, Jody ; Farrawell, Natalie E ; Donnelly, Paul S ; Chisholm, Christen G ; Bartlett, Rachael ; Crouch, Peter J ; Dux, Florian ; Yerbury, Justin J ; Dosseto, Anthony ; Brown, Mikayla L ; Lum, Jeremy S ; McInnes, Lachlan E ; Ecroyd, Heath

BACKGROUND:

SOD1 mutations are a significant contributor of familial amyotrophic lateral sclerosis (ALS) cases. SOD1 mutations increase the propensity for the protein to misfold and aggregate into insoluble proteinaceous deposits within motor neurons and neighbouring cells. The small molecule, CuATSM, has repeatedly shown in mouse models to be a promising therapeutic treatment for SOD1-associated ALS and is currently in Phase II/III clinical trials for the treatment of ALS. We have previously shown CuATSM stabilises various ALS-associated variants of the SOD1 protein, reducing misfolding and toxicity. Two additional FDA-approved small molecules, ebselen and telbivudine, have also been identified to reduce mutant SOD1 toxicity, providing additional potential therapeutic candidates that could be used in combination with CuATSM. Here, we aimed to investigate if CuATSM, ebselen and telbivudine (CET) polytherapy could improve on the therapeutic efficacy of CuATSM monotherapy for the treatment of SOD1-associated ALS.

METHODS:

We utilised a 3D checkerboard approach to investigate whether a matrix of different concentrations CuATSM, ebselen and telbivudine could provide therapeutic improvements on cell survival, SOD1 folding and aggregation in SOD1^G93A-transfected NSC-34 cells, compared to CuATSM alone. To progress the preclinical development of CET polytherapy, we evaluated the bioavailability and safety of in vivo polytherapy administration. Furthermore, we assessed and compared the effects of CET- and CuATSM-treatment on disease onset, motor function, survival and neuropathological features in SOD1^G93A mice.

FINDINGS:

CET polytherapy reduced inclusion formation and increased cell survival of NSC-34 cells overexpressing SOD1^G93A compared to higher concentrations of CuATSM monotherapy. In addition, CET administration was bioavailable and tolerable in mice. CET treatment in SOD1^G93A mice delayed disease onset, reduced motor impairments, and increased survival compared to vehicle- and CuATSM-treated mice. In line with these findings, biochemical analysis of lumbar spinal cords showed CET administration improved SOD1 folding, decreased misfolded SOD1 accumulation, and reduced motor neuron loss.

INTERPRETATION:

These findings support CET polytherapy as an advantageous alternative compared to CuATSM monotherapy and highlight the potential of utilising small molecules targeting SOD1 as a polytherapy avenue for the treatment of SOD1-associated ALS.

FUNDING:

This work was supported by a FightMND Drug Development Grant, an Australian National Health and Medical Research Council (NHMRC) Investigator Grant (No. 1194872) and a Motor Neuron Disease Research Institute of Australia Bill Gole Postdoctoral Fellowship.

News (Medical) associated with Telbivudine

19 Mar 2025

·www.prnewswire.com

New data on investigational therapy for thymidine kinase 2 deficiency presented at Muscular Dystrophy Association (MDA) 2025 Conference

Clinical data highlight survival benefits and improvement in functional motor outcomes associated with treatment with doxecitine (dC) and doxribtimine (dT) - an investigational pyrimidine nucleoside therapy - in people living with thymidine kinase 2 deficiency (TK2d).1,2,3 Additional patient experience data emphasize the profound physical challenges and severe psychological strain that come with living with TK2d, and the heavy emotional and physical burden experienced by caregivers.4,5 Across all study populations, pyrimidine nucleoside and/or nucleotide therapy was generally well tolerated.1,2,3 TK2d is an ultra-rare, life-threatening, genetic mitochondrial disease with no currently approved treatment options.6,7,8,9,10,11 BRUSSELS, March 19, 2025 /PRNewswire/ -- UCB, a global biopharmaceutical company, today announced positive data from studies involving its investigational pyrimidine nucleoside therapy, doxecitine (dC) and doxribtimine (dT), in people living with thymidine kinase 2 deficiency (TK2d), at this year's MDA Clinical and Scientific Conference, Dallas, Texas, March 16-19, 2025. The data show that in individuals with TK2d who were aged 12 years or less when their symptoms first appeared, treatment with pyrimidine nucleoside and/or nucleotide therapy significantly decreased mortality and increased survival.2,3 In addition, treatment also improved functional outcomes irrespective of age of onset, including retaining or regaining motor milestones, and helped stabilize ventilatory and feeding support use.1,2,3 Across all study populations, pyrimidine nucleoside and/or nucleotide therapy was generally well tolerated with diarrhea being the most common treatment emergent adverse event.1,2,3 Thymidine kinase 2 deficiency is an ultra-rare, life-threatening, genetic mitochondrial disease characterized by progressive and severe muscle weakness (myopathy), which can impact the ability to walk, eat, and breathe independently.6,7,8,9,10 "There are no approved therapies or international clinical guidelines for the management of TK2d, therefore we are very excited to share this data with the medical community at MDA," said Donatello Crocetta, Chief Medical Officer at UCB. "The data highlight the positive impact this investigational treatment could have on the lives of people living with this debilitating and life-threatening condition." Data from participants treated in the doxecitine (dC) and doxribtimine (dT) clinical program were pooled from retrospective and prospective sources and a company-supported expanded access program (EAP). Data from untreated participants were pooled from literature reviews of case series and reports, and a retrospective chart review study. Subgroups were stratified by age of TK2d symptom onset categories and reported for participants with age of TK2d symptom onset ≤12 years and >12 years. Doxecitine and doxribtimine is currently under regulatory review by US and EU regulatory authorities. The safety and efficacy have not been established, and doxecitine and doxribtimine has not been approved by the US Food and Drug Administration (FDA) nor the European Medicines Agency (EMA). Key findings from the studies Improvement in survival: Among people living with TK2d with an age of symptom onset 12 years or less, treatment with nucleoside and/or nucleotide therapy reduced risk of death by 92–94% (hazard ratio=0.06–0.08; p<0.0001) and 87–95% (hazard ratio=0.05–0.13; p<0.0001) in the time from symptom onset and starting treatment, respectively.3 Enhanced functional outcomes: Among people living with TK2d who were aged 12 years or less when their symptoms first appeared, following treatment with pyrimidine nucleoside and/or nucleotide therapy, 75.0% (30/40) regained at least one previously lost motor milestone, with 22.5% (9/40) regaining four or more motor milestones.2 In addition, ventilatory support dependency decreased, with 16.1% (5/31) of patients reducing usage time and 16.1% (5/31) discontinuing ventilatory support altogether after treatment.2 Safety profile: Across all study populations, pyrimidine nucleoside and/or nucleotide therapy was generally well tolerated. Diarrhea (range from 84.6%-90.9%) was the most reported treatment-emergent adverse event.1,2,3 Impact on quality of life on individuals and caregivers: In addition to the data presented on nucleoside and/or nucleotide therapy, patient experience data presented highlight the debilitating physical impacts and severe psychological strain associated with living with TK2d, as well as its impact on caregivers. Many individuals reported the 'extreme' impact the condition has on their quality of life including walking, breathing and eating/swallowing difficulties. Caregivers reported that the constant demands of caregiving and minimal support/respite caused persistent stress and emotional burnout.4,5 Key data presented at MDA About thymidine kinase 2 deficiency (TK2d) Thymidine kinase 2 deficiency is an ultra-rare, life-threatening, genetic mitochondrial disease characterized by progressive and severe muscle weakness (myopathy), which can impact the ability to walk, eat, and breathe independently.6,7,8,9,10 It is estimated that the worldwide prevalence of TK2d is 1.64 [0.5, 3.1] cases per 1,000,000 people.12 The age of TK2d symptom onset is categorized as ≤12 years and >12 years.6,7,8 TK2d profoundly affects multiple health, physical, quality-of-life, and psychosocial domains, as children struggle to achieve developmental milestones or lose them, and adults lose functional independence with challenges in breathing, eating, and walking.11,13 About doxecitine and doxribtimine Doxecitine and doxribtimine is a nucleoside therapy that supports mitochondrial DNA replication resulting in increased or stabilized mitochondrial function in patients with TK2d. Pre-clinical data suggest the primary mechanism of action of doxecitine and doxribtimine is the incorporation of nucleosides deoxycytidine (dC) and deoxythymidine (dT) into skeletal muscle mitochondrial deoxyribonucleic acid (mtDNA), which has the potential to restore mitochondrial DNA copy number and improve skeletal muscle function in patients with TK2d.14,15,16 In the U.S., the application has been granted a priority review, Breakthrough Therapy Designation and Rare Pediatric Disease Designation.17,18 For further information, contact UCB: US Rare Diseases Communications Daphne Teo T: +1 770.880.7655 [email protected] Global Communications Nick Francis T: +44 7769 307745 [email protected] Corporate Communications, Media Relations Laurent Schots T +32.2.559.92.64 [email protected] Investor Relations Antje Witte T +32.2.559.94.14 [email protected] About UCB UCB, Brussels, Belgium () is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. With more than 9 000 people in approximately 40 countries, the company generated revenue of € 6.1 billion in 2024. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCB_news Forward-looking statements This press release may contain forward-looking statements including, without limitation, statements containing the words "believes", "anticipates", "expects", "intends", "plans", "seeks", "estimates", "may", "will", "continue" and similar expressions. These forward-looking statements are based on current plans, estimates and beliefs of management. All statements, other than statements of historical facts, are statements that could be deemed forward-looking statements, including estimates of revenues, operating margins, capital expenditures, cash, other financial information, expected legal, arbitration, political, regulatory or clinical results or practices and other such estimates and results. By their nature, such forward-looking statements are not guarantees of future performance and are subject to known and unknown risks, uncertainties and assumptions which might cause the actual results, financial condition, performance or achievements of UCB, or industry results, to differ materially from those that may be expressed or implied by such forward-looking statements contained in this press release. Important factors that could result in such differences include: the global spread and impact of COVID-19, changes in general economic, business and competitive conditions, the inability to obtain necessary regulatory approvals or to obtain them on acceptable terms or within expected timing, costs associated with research and development, changes in the prospects for products in the pipeline or under development by UCB, effects of future judicial decisions or governmental investigations, safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, product liability claims, challenges to patent protection for products or product candidates, competition from other products including biosimilars, changes in laws or regulations, exchange rate fluctuations, changes or uncertainties in tax laws or the administration of such laws, and hiring and retention of its employees. There is no guarantee that new product candidates will be discovered or identified in the pipeline, will progress to product approval or that new indications for existing products will be developed and approved. Movement from concept to commercial product is uncertain; preclinical results do not guarantee safety and efficacy of product candidates in humans. So far, the complexity of the human body cannot be reproduced in computer models, cell culture systems or animal models. The length of the timing to complete clinical trials and to get regulatory approval for product marketing has varied in the past and UCB expects similar unpredictability going forward. Products or potential products, which are the subject of partnerships, joint ventures or licensing collaborations may be subject to differences disputes between the partners or may prove to be not as safe, effective or commercially successful as UCB may have believed at the start of such partnership. UCB's efforts to acquire other products or companies and to integrate the operations of such acquired companies may not be as successful as UCB may have believed at the moment of acquisition. Also, UCB or others could discover safety, side effects or manufacturing problems with its products and/or devices after they are marketed. The discovery of significant problems with a product similar to one of UCB's products that implicate an entire class of products may have a material adverse effect on sales of the entire class of affected products. Moreover, sales may be impacted by international and domestic trends toward managed care and health care cost containment, including pricing pressure, political and public scrutiny, customer and prescriber patterns or practices, and the reimbursement policies imposed by third-party payers as well as legislation affecting biopharmaceutical pricing and reimbursement activities and outcomes. Finally, a breakdown, cyberattack or information security breach could compromise the confidentiality, integrity and availability of UCB's data and systems. Given these uncertainties, you should not place undue reliance on any of such forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labelling in any market, or at any particular time, nor can there be any guarantee that such products will be or will continue to be commercially successful in the future. UCB is providing this information, including forward-looking statements, only as of the date of this press release and it does not reflect any potential impact from the evolving COVID-19 pandemic, unless indicated otherwise. UCB is following the worldwide developments diligently to assess the financial significance of this pandemic to UCB. UCB expressly disclaims any duty to update any information contained in this press release, either to confirm the actual results or to report or reflect any change in its forward-looking statements with regard thereto or any change in events, conditions or circumstances on which any such statement is based, unless such statement is required pursuant to applicable laws and regulations. Additionally, information contained in this document shall not constitute an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any offer, solicitation or sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such jurisdiction. References: Scaglia F, et al. Survival and Functional Outcomes in Patients with Thymidine Kinase 2 Deficiency Aged >12 Years at Symptom Onset Who Received Pyrimidine Nucleos(t)ides. . Accessed February 2025. Garone C, et al. Functional Outcomes in Patients with Thymidine Kinase 2 Deficiency Aged ≤12 Years at Symptom Onset Who Received Pyrimidine Nucleos(t)ide Therapy. . Accessed February 2025. Hirano M, et al. Survival Analyses in Patients with Thymidine Kinase 2 Deficiency Aged ≤12 Years at Symptom Onset Who Received Pyrimidine Nucleos(t)ide Therapy. . Accessed February 2025. Yeske P, et al. Patients' Lived Experience of Thymidine Kinase 2 Deficiency (TK2d): Results from the Assessment of TK2d Patient Perspectives (ATP) Study. . Accessed February 2025. Yeske P, et al. Burden and impact of caring for those with thymidine kinase 2 deficiency (TK2d): results from the Assessment of TK2d Patient Perspectives (ATP) study. . Accessed February 2025. Berardo A, et al. Advances in Thymidine Kinase 2 Deficiency: Clinical Aspects, Translational Progress, and Emerging Therapies. J Neuromuscul Dis. 2022;9(2):225-35. Wang J, et al. TK2-Related Mitochondrial DNA Maintenance Defect, Myopathic Form. 2018. In: Adam MP, et al. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2025. Garone C, et al. Retrospective natural history of thymidine kinase 2 deficiency. J Med Genet. 2018;55(8):515-21. Domínguez-González C, et al. Late-onset Thymidine Kinase 2 Deficiency: A Review of 18 cases. Orphanet J Rare Dis. 2019;14(1):100. National Institutes of Health. TK2-related mitochondrial DNA depletion syndrome, myopathic form. . Accessed February 2025. U.S. FDA TK2d Patient Listening Session. . Accessed February 2025. Ma Y. Prevalence Estimation of Thymidine Kinase 2 Deficiency: An Ultra-Rare Autosomal Recessive Mitochondrial Disease. Poster presented at: ISPOR Europe; 2023, 12-15 November; Denmark. Amtmann D, et al. The impact of TK2 deficiency syndrome and its treatment by nucleoside therapy on quality of life. Mitochondrion. 2023;68:1-9. Lopez-Gomez C, et al. Deoxycytidine and Deoxythymidine Treatment for Thymidine Kinase 2 Deficiency. Ann Neurol. 2017;81(5):641-52. Lopez-Gomez C, et al. Bioavailability and cytosolic kinases modulate response to deoxynucleoside therapy in TK2 deficiency. EBioMedicine. 2019;46:356-367. National Library of Medicine. A Study of the Efficacy and Safety of MT1621 in Thymidine Kinase 2 (TK2) Deficiency (Treatment naïve). . Accessed February 2025. U.S. FDA. Orphan Drug Designations and Approvals. . Accessed February 2025. UCB Press Release. . Accessed February 2025. ©2025 UCB, Inc., Smyrna, GA 30080. All rights reserved. US-MT-2500041 SOURCE UCB WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

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20 Jun 2024

·www.biospace.com

Precision BioSciences Expands Hepatitis Scientific Advisory Board with Addition of World-Class Clinical Investigators Mark Sulkowski, M.D. and Jordan Feld, M.D., MPH, as PBGENE-HBV Program Nears Clinical Readiness

DURHAM, N.C.--(BUSINESS WIRE)-- Precision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for sophisticated gene edits, including gene elimination, insertion, and excision, today announced the appointment of Mark Sulkowski, M.D. and Jordan Feld, M.D., M.P.H. to its Hepatitis Scientific Advisory Board (SAB). Dr. Sulkowski and Dr. Feld will join Raymond Schinazi, Ph.D., DSc, inaugural member of Precision’s Hepatitis SAB, to provide counsel and deepen the Company’s scientific and clinical expertise ahead of Precision’s anticipated Investigational New Drug (IND) and/or Clinical Trial Application (CTA) submission of PBGENE-HBV for the treatment of chronic hepatitis B in the second half of 2024. “We are thrilled and privileged to welcome Dr. Sulkowski and Dr. Feld as members of our SAB as we rapidly advance the development of PBGENE-HBV towards the clinic,” said Michael Amoroso, President and Chief Executive Officer of Precision BioSciences. “Together, they bring immense expertise as investigators in hepatitis clinical trials, which will be invaluable as we expect to submit the IND and/or CTA applications for PBGENE-HBV later this year, bringing our first wholly owned ARCUS nuclease into clinical trials. We look forward to leveraging the SAB’s input to guide the development of PBGENE-HBV and the broader potential of our ARCUS platform.” “Dr. Sulkowski and Dr. Feld are welcome additions to the SAB as Precision nears key inflection points,” said Dr. Raymond Schinazi, a pioneer of treatments for hepatitis and member of Precision’s Hepatitis Scientific Advisory Board. “Mark’s and Jordan’s contributions to the existing hepatitis B body of knowledge establishes them as leaders in the field, and we are delighted to have advisors of their caliber coming aboard.” Precision BioSciences Hepatitis Scientific Advisory Board includes: Mark Sulkowski, M.D. is a Professor of Medicine at the Johns Hopkins University School of Medicine and the Director of the Division of Infectious Diseases at Johns Hopkins Bayview Medical Center. He also serves as the Medical Director of the Viral Hepatitis Center in the Divisions of Infectious Diseases and Gastroenterology /Hepatology in the Department of Medicine and is the Senior Associate Dean for Clinical Trials. Dr. Sulkowski has been the principal investigator for more than 120 clinical trials related to the management of viral hepatitis B and C and has published over 300 peer reviewed articles with works in Annals of Internal Medicine, New England Journal of Medicine, JAMA, Clinical Infectious Diseases, Journal of Hepatology, and Hepatology. Jordan Feld, M.D., M.P.H. is the Site Lead for Clinical Research at Toronto General Hospital. He is also Professor of Medicine and the R. Phelan Chair in Translational Liver Research at the University of Toronto, where he serves as Research Director in Gastroenterology with the Francis Family Liver Clinic at Toronto Western Hospital. Dr. Feld is a Hepatitis B Special Interest Group Executive of AASLD, a member of the AASLD Viral Hepatitis Elimination Task Force, the ASCO Provisional Clinical Opinion on Hepatitis B Reactivation Working Group, and the Hepatitis Steering Committee of the AIDS Clinical Trials Group Network Coordinating Center/Hepatitis Transformative Science Group. Dr. Feld is deeply involved in clinical and laboratory-focused translational research and has published extensively on prevention of viral hepatitis transmission, long-term nucleotide analogue therapy in chronic hepatitis B, and hepatitis B vaccination. Raymond Schinazi, Ph.D., DSc is the Frances Winship Walters Professor of Pediatrics and Director of the Laboratory of Biochemical Pharmacology at Emory University and Co-Director of the HIV Cure Scientific Working Group for the NIH-sponsored Emory University Center for AIDS Research. Dr. Schinazi has authored over 550 peer-reviewed papers, seven books and holds over 100 issued US patents, which have resulted in 22 New Drug Applications. A world leader in nucleoside chemistry, Dr. Schinazi is best known for his pioneering work on HIV, HBV, and HCV drugs d4T (stavudine), 3TC (lamivudine), FTC (emtricitabine), LdT (telbivudine), and sofosbuvir (Sovaldi), which are all approved by the US FDA and the EMEA. More than 94% of HIV-infected individuals in the US on combination therapy take at least one of the drugs he invented. About Hepatitis B and PBGENE-HBV: Hepatitis B is a leading cause of morbidity in the US and death globally, with no curative options currently available for patients. In 2019, despite the availability of approved antiviral therapies, an estimated 300 million people globally and more than 1 million people in the US were estimated to have chronic hepatitis B infection. An estimated 15% to 40% of patients with HBV infections may develop complications, such as cirrhosis, liver failure, or liver cancer (hepatocellular carcinoma), which account for the majority of HBV-related deaths. Chronic hepatitis B infection is primarily driven by persistence of HBV cccDNA and integration of HBV DNA into the human genome in liver cells, the primary source of HBsAg in late-stage disease. Current treatments for patients with HBV infection include agents that result in long-term viral suppression as indicated by reduction of circulating HBV DNA, but these therapies do not eradicate HBV cccDNA, rarely lead to functional cure, and require lifelong administration. PBGENE-HBV is a highly specific, novel therapeutic approach to treating patients with chronic HBV infection. It’s designed to directly eliminate cccDNA and inactivate integrated HBV DNA with high specificity, potentially leading to functional cures. About Precision BioSciences, Inc. Precision BioSciences, Inc. is an advanced gene editing company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform that differs from other technologies in the way it cuts, its smaller size, and its simpler structure. Key capabilities and differentiating characteristics may enable ARCUS nucleases to drive more intended, defined therapeutic outcomes. Using ARCUS, the Company’s pipeline is comprised of in vivo gene editing candidates designed to deliver lasting cures for the broadest range of genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit . The ARCUS® platform is being used to develop in vivo gene editing therapies for sophisticated gene edits, including gene insertion (inserting DNA into gene to cause expression/add function), elimination (removing a genome e.g. viral DNA or mutant mitochondrial DNA), and excision (removing a large portion of a defective gene by delivering two ARCUS nucleases in a single AAV). Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the clinical development and expected safety, efficacy and benefit of our product candidates and gene editing approaches including editing efficiency; the design of PBGENE-HBV to directly eliminate cccDNA and inactivate integrated HBV DNA with high specificity, potentially leading to functional cures; the suitability of ARCUS nucleases for gene elimination, insertion and excision and differentiation from other gene editing approaches due to its small size, simplicity and distinctive cut; the expected timing of regulatory processes (including filings such as IND’s and CTA’s and studies for PBGENE-HBV); expectations about operational initiatives, strategies, and further development of our programs; expectations about achievement of key milestones; and anticipated timing of clinical data. In some cases, you can identify forward-looking statements by terms such as “aim,” “anticipate,” “approach,” “believe,” “contemplate,” “could,” “designed,” “estimate,” “expect,” “goal,” “intend,” “look,” “may,” “mission,” “plan,” “possible,” “potential,” “predict,” “project,” “pursue,” “should,” “strive,” “target,” “will,” “would,” or the negative thereof and similar words and expressions. Forward-looking statements are based on management’s current expectations, beliefs and assumptions and on information currently available to us. These statements are neither promises nor guarantees, and involve a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to, our ability to become profitable; our ability to procure sufficient funding to advance our programs; risks associated with our capital requirements, anticipated cash runway, requirements under our current debt instruments and effects of restrictions thereunder, including our ability to raise additional capital due to market conditions and/or our market capitalization; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the progression and success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; the risk that other genome-editing technologies may provide significant advantages over our ARCUS technology; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities and preclinical and clinical studies, including clinical trial and investigational new drug applications; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, and biotechnology fields; our or our collaborators’ or other licensees’ ability to identify, develop and commercialize product candidates; pending and potential product liability lawsuits and penalties against us or our collaborators or other licensees related to our technology and our product candidates; the US and foreign regulatory landscape applicable to our and our collaborators’ or other licensees’ development of product candidates; our or our collaborators’ or other licensees’ ability to advance product candidates into, and successfully design, implement and complete, clinical trials; potential manufacturing problems associated with the development or commercialization of any of our product candidates; delays or difficulties in our and our collaborators’ and other licensees’ ability to enroll patients; changes in interim “top-line” and initial data that we announce or publish; if our product candidates do not work as intended or cause undesirable side effects; risks associated with applicable healthcare, data protection, privacy and security regulations and our compliance therewith; our or our licensees’ ability to obtain orphan drug designation or fast track designation for our product candidates or to realize the expected benefits of these designations; our or our collaborators’ or other licensees’ ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; the rate and degree of market acceptance of any of our product candidates; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate executives and personnel; effects of system failures and security breaches; insurance expenses and exposure to uninsured liabilities; effects of tax rules; effects of any pandemic, epidemic, or outbreak of an infectious disease; the success of our existing collaboration and other license agreements, and our ability to enter into new collaboration arrangements; our current and future relationships with and reliance on third parties including suppliers and manufacturers; our ability to obtain and maintain intellectual property protection for our technology and any of our product candidates; potential litigation relating to infringement or misappropriation of intellectual property rights; effects of natural and manmade disasters, public health emergencies and other natural catastrophic events; effects of sustained inflation, supply chain disruptions and major central bank policy actions; market and economic conditions; risks related to ownership of our common stock, including fluctuations in our stock price; our ability to meet the requirements of and maintain listing of our common stock on Nasdaq or other public stock exchanges; and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2024, as any such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SEC’s website at and the Investors page of our website under SEC Filings at investor.precisionbiosciences.com. All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

Executive ChangeOrphan DrugGene Therapy

100 Deals associated with Telbivudine

External Link

KEGG	Wiki	ATC	Drug Bank
D06675	Telbivudine	J05AF11	DB01265

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Hepatitis B	China	Novartis Pharma AG	20 Jan 2008
Hepatitis B, Chronic	United States	Novartis Pharmaceuticals Corp.	25 Oct 2006

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Complete atrioventricular block	Phase 3	United States	Novartis AG	01 Dec 2006
Fibrosis	Phase 3	United States	Novartis Pharmaceuticals Corp.	01 Dec 2003
Fibrosis	Phase 3	China	Novartis Pharmaceuticals Corp.	01 Dec 2003
Fibrosis	Phase 3	Australia	Novartis Pharmaceuticals Corp.	01 Dec 2003
Fibrosis	Phase 3	Canada	Novartis Pharmaceuticals Corp.	01 Dec 2003
Fibrosis	Phase 3	France	Novartis Pharmaceuticals Corp.	01 Dec 2003
Fibrosis	Phase 3	Germany	Novartis Pharmaceuticals Corp.	01 Dec 2003
Fibrosis	Phase 3	India	Novartis Pharmaceuticals Corp.	01 Dec 2003
Fibrosis	Phase 3	Israel	Novartis Pharmaceuticals Corp.	01 Dec 2003
Fibrosis	Phase 3	Latvia	Novartis Pharmaceuticals Corp.	01 Dec 2003

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT01521975 (Pubmed \| BMC Infect Dis)	Phase 4	Hepatitis B, Chronic	108	Telbivudine (LdT)	nmgckdwqvh(jzubasxifu) = otipciuskp vizcugsqir (jdfqirstrx ) View more	-	07 Dec 2020
NCT02058108 (CTgov)	Phase 3	Hepatitis B, Chronic	53	Telbivudine (Telbivudine)	pyfrwqyhpn = ygetwoqhiz nyhfyuqkvz (lkqyefivee, sdxpdplqya - szzzkmknsm) View more	-	10 Sep 2019
				Placebo (Placebo)	pyfrwqyhpn = bmbvzignop nyhfyuqkvz (lkqyefivee, hfruyzdeof - yzqnvuryms) View more
NCT01379508 (CTgov)	Phase 4	Hepatitis B, Chronic	241	Ldt+TDF (LdT Overall)	eqipkphier = bmznpnvjwb ombblnyffs (faabaoyztc, fsexemmdvt - sozwleqxvw) View more	-	05 Nov 2018
				TDF (TDF Overall)	eqipkphier = zxnnzeowbm ombblnyffs (faabaoyztc, kqspwiuhbe - aljgnnjaxg) View more
NCT01637844 (Pubmed \| J Viral Hepat)	Phase 4	pregnancy late	328	Telbivudine	moljnnnajt(lhvanlxnwp) = bzyrxlvwnv nlmoucxwcp (njfkmfrzqk )	-	01 Apr 2018
				telbivudine (Control (no antiviral agent))	wbeazxmmrw(uupuegnmvh) = ziolyrvsem nvmfzuexbt (mtoyvjxbbo ) View more
NCT02253485 (Pubmed \| J Viral Hepat)	Not Applicable	Hepatitis B	397	Group A	kibebheijj(pspgdnvnja) = xqqjhwdgqk xqavppzjwr (dckfstdlbr )	-	01 Nov 2017
				Group B	kibebheijj(pspgdnvnja) = grnvimeuvv xqavppzjwr (dckfstdlbr ) View more
NCT00939068 (Pubmed \| J Viral Hepat)	Phase 4	-	214	Telbivudine	sdqlmrlzpu(wpzuevqsux) = pifjqcvskq afylkivqmy (xcpnajomnn ) View more	-	01 Jun 2017
NCT01595685 (TERESA, Pubmed \| BMC Gastroenterol) Manual	Phase 3	Hepatitis B	97	Telbivudine	zrgctrrxbr(zzvvxcpdnp) = tzclwyuncz vawdzxzjlq (llzhpsxbwm ) View more	Negative	19 Jan 2017
				entecavir	zrgctrrxbr(zzvvxcpdnp) = mqiryytequ vawdzxzjlq (llzhpsxbwm ) View more
NCT02447705 (Pubmed \| Biomed Res Int)	Phase 2/3	Chronic Kidney Diseases	120	Lamivudine-continuous	kjzwukkrpy(fmodfohlrd) = Eleven (18.3%) patients with telbivudine developed polyneuritis during the trial and post hoc following-up lpmaiaajwh (hnnvfccrqe )	-	01 Jan 2017
				Telbivudine-replacement
NCT00877149 \| NCT00646503 (Pubmed \| Adv Ther)	Phase 4	Hepatitis B, Chronic	57	Telbivudine 600 mg/day	twldnjeypj(avfqacnvch) = wltbxbieec bitdnpfmlk (csgfzdnsoy ) View more	-	01 Aug 2015
				Lamivudine 100 mg/day + Telbivudine	twldnjeypj(avfqacnvch) = frmvshhdkl bitdnpfmlk (csgfzdnsoy )
NCT00412750 (Pubmed \| J Hepatol)	Phase 3	Hepatitis B, Chronic	159	Telbivudine plus PegIFN alpha-2a	kflgrlaopn(llleejsvrm) = Peripheral neuropathy occurred in 7/50, 1/54, and 0/54 patients in the three groups of safety populations, respectively xlllopnogk (vjrzdxfoay )	Negative	01 Jan 2015
				Telbivudine monotherapy

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