You are here: Home ยป Archives for Clinical Study

Minovia Therapeutics Announces FDA Clearance of IND Application for a Phase Ib Clinical Trial of MNV-201 in Low Risk Myelodysplastic Syndrome

MNV–201 is a mitochondrial cell therapy product composed of autologous hematopoietic stem cells enriched with allogeneic mitochondria

In pre–clinical studies, MNV–201 demonstrated improved engraftment and bone marrow reconstitution potential of patient derived hematopoietic stem cells

In vitro data also demonstrated improved ability to differentiate to erythroid cells, supporting potential for improvement in biomarkers of anemia

HAIFA, Israel, Sept. 26, 2024 (GLOBE NEWSWIRE) — Minovia Therapeutics Ltd, a clinical stage biopharmaceutical company advancing mitochondrial cell therapies for primary and secondary mitochondrial diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for MNV–201, an autologous hematopoietic stem cell product augmented with allogeneic mitochondria. The IND supports the initiation of a Phase Ib dose exploration clinical trial of MNV–201 in patients with Low Risk Myelodysplastic Syndrome (MDS).

Anemia is a common and serious symptom in patients with low–risk myelodysplastic syndrome (LR–MDS), affecting almost 90% of cases and is often the primary characteristic of the disease. Anemia in MDS can have a negative impact on quality of life and may correlate with decreased progression–free survival and overall survival. 

“The FDA’s clearance of our IND marks an important achievement for Minovia, allowing us to clinically evaluate our allogeneic mitochondrial cell therapy approach and proceed with the Phase Ib clinical program for this first–in–class allogeneic mitochondrial therapy for low risk MDS patients,” said Natalie Yivgi Ohana, PhD, CEO of Minovia. “We are pleased to have safely dosed two MDS patients enrolled in an ongoing study under the Israeli Ministry of Health. We look forward to treating additional patients under this IND, as well as to learning about the potential of MAT to improve anemia in this patient population.”

The Phase Ib clinical trial is an open–label, dose exploration study to evaluate the safety and efficacy of MNV–201 in subjects with low risk MDS. This trial will continue our campaign to evaluate dose exploration and safety of single or repeat dosing of MNV–201. The trial will also enable assessment of efficacy in improving anemia and durability of response. The study is expected to enroll at least three patients each in the low, medium and high dose cohorts, and up to a total of 15 patients in total. For more information visit clinicaltrials.gov.

About MNV–201
MNV–201 is an autologous hematopoietic stem cell product enriched with allogeneic mitochondria. MNV–201 aims to restore mitochondrial function in low risk MDS patient hematopoietic stem cells, resulting in improved differentiation to erythroid lineage with the potential to improve anemia. Preclinical research suggests the potential for safe dosing with low immunogenicity risk and scalable manufacturing to address the significant number of patients who are potentially eligible for MNV–201 therapy.

About Minovia Therapeutics
Minovia Therapeutics Ltd. is a clinical stage biotechnology company advancing mitochondrial cell therapies for primary–genetic and age–related mitochondrial diseases. Minovia's clinical stage product candidate, MNV–201, is composed of mobilized peripheral blood, autologous CD34+ cells enriched with allogeneic, cryopreserved placental derived mitochondria, produced by Minovia's proprietary Mitochondrial Augmentation Technology (MAT). The enrichment of hematopoietic stem cells with healthy and functional mitochondria aims to restore stem cells function of patients suffering mitochondrial dysfunction, caused both by mtDNA mutations or deletions in pediatric patients suffering from primary mitochondrial diseases, or in adults with age–related bone marrow failure disorders. MNV–201 is currently in clinical studies for pediatric patients with single–large scale mtDNA deletion syndromes (Pearson Syndrome and Kearn Sayre Syndrome) with four patients successfully dosed; and in Low Risk Myelodysplastic Syndrome. For more information, please visit www.minoviatx.com or follow the Company LinkedIn.

About Low Risk Myelodysplastic Syndrome

Myelodysplastic syndromes (MDS) are a group of bone marrow failures that occur when the blood–forming cells in the bone marrow become abnormal leading to an abnormal differentiation and production of one or more blood cell types. Patients with MDS collectively have a high symptom burden and are also at risk of death from complications of cytopenias or progression to acute myeloid leukemia (AML). MDS is generally a disease that develops with aging; the median age at diagnosis of MDS is ~70 years.

Mitochondrial Dysfunction in MDS: Scientific literature shows a correlation between mitochondrial dysfunction and MDS progression. It is known that ineffective hematopoiesis in MDS results from increased susceptibility of clonal myeloid progenitors to apoptosis. This may be triggered by intrinsic factors, such as mitochondrial polarization due to iron retention in ringed sideroblasts. A subset of MDS patients present with sideroblastic anemia, a phenotype common in Pearson Syndrome patients and which implicates mitochondrial dysfunction of HSPCs as part of the pathology of MDS.

Contact Information: Natalie Yivgi Ohana, Co–Founder and CEO

Phone: +972–74–7039954

Email: [email protected]


GLOBENEWSWIRE (Distribution ID 9237236)

Entera Bio Reports Q2 2024 Financial Results and Provides Business Updates

JERUSALEM , Aug. 09, 2024 (GLOBE NEWSWIRE) — Entera Bio Ltd. (NASDAQ: ENTX), (“Entera” or the “Company”) a leader in the development of orally delivered peptides and small therapeutic proteins, today reported financial results and key business updates for the quarter ended June 30, 2024.

“We continue to deliver strong execution with key milestones achieved during the second quarter of 2024 across each of our N–Tab™ oral peptide programs dedicated to patients with OBGYN/endocrinology, GI and metabolic diseases,” said Miranda Toledano, Entera’s CEO. “Importantly, we are now just five months away from FDA’s potential landmark ruling on the ASBMR–FNIH SABRE regulatory endpoint for osteoporosis drugs, which we view as a major catalyst for EB613. We are especially keen to start our pivotal study of EB613 in a much wider population where injectable anabolic drugs do not play a dominant role. Because of its potential dual mechanism of action, faster onset of action as an anabolic boosting agent and oral minitablet format, we believe EB613 is uniquely positioned to support earlier osteoanabolic intervention in post–menopausal women at high risk of fracture,” she added.

EB613: First Oral PTH(1–34) Daily Osteoanabolic Tablets for Osteoporosis

  • In April 2024, the Journal of Bone and Mineral Research (JBMR) published “Oral daily PTH(1–34) Tablets [EB613] in Postmenopausal Women with Low BMD or Osteoporosis: A Randomized, Placebo–Controlled, 6–Month, Phase 2 Study”
  • In May 2024, Entera welcomed Dr. Rachel Wagman as Key Clinical Advisor to lead EB613 clinical development. Wagman has successfully advanced the development of five molecules, including the osteoporosis products Forteo®, Prolia® and Evenity® through registration
  • In June 2024, the JMBR published an independent editorial titled “A Novel Oral hPTH(1–34) [EB613] Unveils the Promise of Modeling–Based Anabolism with No Increase in Bone Remodeling”
  • In July 2024, Entera announced that new comparative pharmacological data for its investigational agent EB613 vs. Forteo® was selected for presentation at the ASBMR September 2024 Annual Meeting in Toronto
  • In July 2024, Entera announced that the SABRE (Study to Advance BMD as a Regulatory Endpoint) is expected to provide an update at the ASBMR September 2024 Annual Meeting in Toronto

EB612: First Oral PTH(1–34) Peptide Replacement Therapy Tablets for Hypoparathyroidism

  • In June 2024, Entera presented Phase 1 clinical data for its hypoparathyroidism focused investigational program, EB612, at the Endocrine Society ENDO 2024 Annual Meeting. Entera showed that the data supports potentially moving the BID (twice–daily) tablet dose to Phase 2 development in patients with hypoparathyroidism
  • Entera continues to collaborate with a third party on the development of another PTH replacement treatment for hypoparathyroidism

First GLP–2 Peptide Tablets for Short Bowel Syndrome

  • In March 2024, Entera announced positive in vivo PK results from its program combining OPKO Health, Inc.’s (Nasdaq: OPK) long acting GLP–2 analogue with N–Tab™ technology. Pharmacology data is expected early in the second half of 2024

First GLP–1/Glucagon Agonist (Oxyntomodulin) Peptide Tablets for Obesity

  • Collaborative work is ongoing combining N–Tab™ with OPKO’s long–acting Oxyntomodulin (OXM) analogues for potential treatment for obesity and other metabolic diseases. PK data for the oral OXM tablet are expected early in the second half of 2024

Financial Results for the Quarter Ended June 30, 2024

As of June 30,2024, Entera had cash and cash equivalents of $9.1 million. The Company expects that its existing cash resources are sufficient to meet its projected operating requirements into the third quarter of 2025.

Research and development expenses for the three months ended June 30, 2024 were $1.1 million, as compared to $1.2 million for the three months ended June 30, 2023. The decrease of $0.1 million was primarily due to a decrease of $0.3 million in clinical expenses for our Phase 1 PK study related to our new generation platform and new formulations for EB612, which completed its first stage in 2023.

General and administrative expenses for both the three months ended June 30, 2024 and 2023 were $1.1 million.

Operating expenses for the period ended June 30, 2024 were $2.2 million, as compared to $2.3 million for the quarter ended June 30, 2023.

Net loss was $2.1 million, or $0.06 per ordinary share (basic and diluted), for the quarter ended June 30, 2024, as compared to $2.3 million, or $0.08 per ordinary share (basic and diluted), for the quarter ended June 30, 2023.

About Entera Bio

Entera is a clinical stage company focused on developing oral peptide or protein replacement therapies for significant unmet medical needs where an oral tablet form holds the potential to transform the standard of care. The Company leverages on a disruptive and proprietary technology platform (N–Tab™) and its pipeline includes five differentiated, first–in–class oral peptide programs, expected to enter the clinic (Phase 1 to Phase 3) by 2025. The Company’s most advanced product candidate, EB613 (oral PTH (1–34)), is being developed as the first oral, osteoanabolic (bone building) once–daily tablet treatment for post–menopausal women with low BMD and high–risk osteoporosis, with no prior fracture. A placebo controlled, dose ranging Phase 2 study of EB613 tablets (n= 161) met primary (PD/bone turnover biomarker) and secondary endpoints (BMD). Entera is preparing to initiate a Phase 3 registrational study for EB613 pursuant to the FDA’s qualification of a quantitative BMD endpoint which is expected to occur by January 2025. The EB612 program is being developed as the first oral PTH(1–34) tablet peptide replacement therapy for hypoparathyroidism. Entera is also developing the first oral oxyntomodulin, a dual targeted GLP1/glucagon peptide, in tablet form for the treatment of obesity; and first oral GLP–2 peptide tablet as an injection–free alternative for patients suffering from rare malabsorption conditions such as short bowel syndrome in collaboration with OPKO Health. For more information on Entera Bio, visit www.enterabio.com or follow us on LinkedIn, Twitter, Facebook, Instagram.

Cautionary Statement Regarding Forward Looking Statements

Various statements in this press release are “forward–looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. All statements (other than statements of historical facts) in this press release regarding our prospects, plans, financial position, business strategy and expected financial and operational results may constitute forward–looking statements. Words such as, but not limited to, “anticipate,” “believe,” “can,” “could,” “expect,” “estimate,” “design,” “goal,” “intend,” “may,” “might,” “objective,” “plan,” “predict,” “project,” “target,” “likely,” “should,” “will,” and “would,” or the negative of these terms and similar expressions or words, identify forward–looking statements. Forward–looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties. Forward–looking statements should not be read as a guarantee of future performance or results and may not be accurate indications of when such performance or results will be achieved.

Important factors that could cause actual results to differ materially from those reflected in Entera’s forward–looking statements include, among others: changes in the interpretation of clinical data; results of our clinical trials; the FDA’s interpretation and review of our results from and analysis of our clinical trials; unexpected changes in our ongoing and planned preclinical development and clinical trials, the timing of and our ability to make regulatory filings and obtain and maintain regulatory approvals for our product candidates; the potential disruption and delay of manufacturing supply chains; loss of available workforce resources, either by Entera or its collaboration and laboratory partners; impacts to research and development or clinical activities that Entera may be contractually obligated to provide; overall regulatory timelines; the size and growth of the potential markets for our product candidates; the scope, progress and costs of developing Entera’s product candidates; Entera’s reliance on third parties to conduct its clinical trials; Entera’s expectations regarding licensing, business transactions and strategic collaborations; Entera’s operation as a development stage company with limited operating history; Entera’s ability to continue as a going concern absent access to sources of liquidity; Entera’s ability to obtain and maintain regulatory approval for any of its product candidates; Entera’s ability to comply with Nasdaq’s minimum listing standards and other matters related to compliance with the requirements of being a public company in the United States; Entera’s intellectual property position and its ability to protect its intellectual property; and other factors that are described in the “Cautionary Statements Regarding Forward–Looking Statements,” “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” sections of Entera’s most recent Annual Report on Form 10–K filed with the SEC, as well as the company’s subsequently filed Quarterly Reports on Form 10–Q and Current Reports on Form 8–K. There can be no assurance that the actual results or developments anticipated by Entera will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, Entera. Therefore, no assurance can be given that the outcomes stated or implied in such forward–looking statements and estimates will be achieved. Entera cautions investors not to rely on the forward–looking statements Entera makes in this press release. The information in this press release is provided only as of the date of this press release, and Entera undertakes no obligation to update or revise publicly any forward–looking statements, whether as a result of new information, future events or otherwise, except to the extent required by law.

ENTERA BIO LTD.
CONSOLIDATED BALANCE SHEETS
(U.S. dollars in thousands)
 
  June 30,   December 31,
  2024   2023
  (Unaudited)   (Audited)
   
Cash and cash equivalents 9,056   11,019
Accounts receivable and other current assets 539   238
Property and equipment, net 76   100
Other assets, net 364   408
Total assets 10,035   11,765
       
       
Accounts payable and other current liabilities 1,294   1,091
Total non–current liabilities 219   288
Total liabilities 1,503   1,379
Total shareholders' equity 8,532   10,386
       
Total liabilities and shareholders' equity 10,035   11,765
 

ENTERA BIO LTD.
CONSOLIDATED STATEMENTS OF OPERATIONS
(U.S. dollars in thousands, except share and per share data)
(Unaudited)
 
  Three Months Ended
June 30,
  2024   2023  
REVENUES 57   –  
COST OF REVENUES 48   –  
GROSS PROFIT 9   –  
OPERATING EXPENSES:    
Research and development 1,086   1,209  
General and administrative 1,088   1,135  
Other income –   (14)  
TOTAL OPERATING EXPENSES 2,174   2,330  
OPERATING LOSS 2,165   2,330  
FINANCIAL INCOME, NET (20)   (5)  
NET LOSS 2,145   2,325  
     
LOSS PER SHARE BASIC AND DILUTED 0.06   0.08  
WEIGHTED AVERAGE NUMBER OF SHARES OUTSTANDING         
USED IN COMPUTATION OF BASIC AND DILUTED LOSS PER SHARE 37,090,160   28,812,375  
     


GLOBENEWSWIRE (Distribution ID 9202470)

Sabin Vaccine Institute Begins Phase 2 Clinical Trial for Sudan Ebolavirus Vaccine

Laboratory at Makerere University Walter Reed Project, where Sabin’s Phase 2 Sudan ebolavirus vaccine clinical trial begins this month.

WASHINGTON, July 15, 2024 (GLOBE NEWSWIRE) — Sabin Vaccine Institute launched a Phase 2 clinical trial for its vaccine against Sudan ebolavirus, with healthy volunteers receiving the single–dose vaccine at Makerere University Walter Reed Project (MUWRP) in Uganda. There are currently no approved vaccines for this strain of ebolavirus, which saw an outbreak end just last year. Ebolavirus disease kills on average half the people infected, according to the World Health Organization.

Sudan ebolavirus is a filovirus, in the same family as Marburg virus disease and Zaire ebolavirus, which killed 11,325 in one outbreak in West Africa from 2014–16. Ebolavirus disease spreads between people via direct contact with the blood or other bodily fluids of infected people and is highly virulent, causing hemorrhagic fever.

Based on the same cAd3 platform as its Marburg vaccine candidate, Sabin’s single–dose investigational Sudan ebolavirus vaccine was found to be promising in Phase 1 clinical and non–clinical studies, with results showing it to be safe, while eliciting rapid and robust immune responses that lasted up to 12 months.

This is Sabin’s second Phase 2 clinical trial partnership with MUWRP, based in Uganda’s capital, Kampala. A Phase 2 trial for a Marburg vaccine is already underway, having recently completed enrollment. Initial results from the Marburg trial are expected later this year.

Dr. Betty Mwesigwa, deputy executive director of MUWRP, is once again the principal investigator (PI) for the Sabin–sponsored trial for the Sudan ebolavirus vaccine. In the coming weeks, participants will also be enrolled at the Kenya Medical Research Institute in Siaya, Kenya, with Dr. Videlis Nduba serving as PI for that site. In all, 125 volunteers will participate in the trial across the two countries.

“We are delighted to advance a vaccine candidate that can thwart a deadly and devastating disease, especially one that caused a fairly recent outbreak and for which no approved treatments exist,” says Amy Finan, Sabin’s Chief Executive Officer. “Sabin’s vaccine candidate is backed by strong safety and immunogenicity data, and we hope this trial will yield further evidence to move the vaccine closer to licensure.”

The most recent outbreak of Sudan ebolavirus occurred in the fall of 2022 in Uganda, after six suspicious deaths in the Mubende district. That outbreak ultimately resulted in 55 deaths. Sabin’s vaccine was the first to arrive in Uganda during that outbreak after the World Health Organization included it as one of three vaccines for possible use in an outbreak trial. The outbreak ended on January 11, prior to vaccine being deployed for use.

“Makerere University Walter Reed Project is pleased to partner with the Sabin Vaccine Institute once again,” says Dr. Mwesigwa. “Uganda has the most experience with Sudan ebolavirus outbreaks so we understand the importance of testing and researching an effective Sudan ebolavirus vaccine that could be used in the event of an outbreak.”

The Phase 2 clinical trial will evaluate safety and immunogenicity for the vaccine among a larger group of individuals than in previous trials. This is a randomized, placebo–controlled, double–blind study, meaning that neither the participants nor the researchers will know whether trial participants receive a vaccine dose or a placebo dose until after the trial is over, an approach used to help reduce experimental bias.

Participants in the clinical trial will be monitored for a full year and will include both younger (18–50 years) and older age groups (51–70 years). Interim results are expected next year. In addition to the current trial in Uganda and Kenya, Sabin plans to conduct a similar Phase 2 clinical trial for Sudan ebolavirus vaccine in the U.S.

The Sudan ebolavirus vaccine trials are supported by the Biomedical Advanced Research and Development Authority (BARDA), part of the Administration for Strategic Preparedness and Response within the U.S. Department of Health and Human Services, under multi–year contracts between the organizations.

To date, Sabin has received around $216 million in contract awards from BARDA for furthering vaccine research and development against Sudan ebolavirus and Marburg virus diseases. BARDA and Sabin began working together in September 2019 to develop the two monovalent vaccine candidates.

This project has been supported in whole or in part with federal funds from the Department of Health and Human Services; Administration for Strategic Preparedness and Response; Biomedical Advanced Research and Development Authority (BARDA), under contract numbers 75A50119C00055 and 75A50123C00010.

About the Sabin Vaccine Institute

The Sabin Vaccine Institute is a leading advocate for expanding vaccine access and uptake globally, advancing vaccine research and development, and amplifying vaccine knowledge and innovation. Unlocking the potential of vaccines through partnership, Sabin has built a robust ecosystem of funders, innovators, implementers, practitioners, policy makers and public stakeholders to advance its vision of a future free from preventable diseases. As a non–profit with three decades of experience, Sabin is committed to finding solutions that last and extending the full benefits of vaccines to all people, regardless of who they are or where they live. At Sabin, we believe in the power of vaccines to change the world. For more information, visit www.sabin.org and follow us on X, @SabinVaccine.

About Sabin’s Vaccine R&D Using the cAd3 Platform

In August 2019, Sabin announced exclusive agreements with GSK for Sabin to advance the development of the prophylactic candidate vaccines against the deadly Zaire ebolavirus, Sudan ebolavirus and Marburg virus. The three candidate vaccines were initially developed collaboratively by the U.S. National Institutes of Health and Okairos, which was acquired by GSK in 2013. The candidate vaccines, based on GSK’s proprietary cAd3 platform, were further developed by GSK, including the Phase 2 development for the Zaire ebolavirus vaccine. Under the agreements between GSK and Sabin, Sabin exclusively licensed the technology for all three candidate vaccines and acquired certain patent rights specific to these vaccines.

About the Makerere University Walter Reed Project

MUWRP is a non–profit biomedical research organization with a mission to mitigate disease threats through quality research, health care and disease surveillance. The project’s scope includes, among others; clinical research in infectious and non–infectious diseases such as HIV, Ebola, Marburg, COVID–19, Influenza and Influenza–like illnesses, and neglected tropical diseases such as Schistosomiasis, among others. A major part of the clinical research are clinical trials, where the MUWRP has conducted more than 12 phase 1 and 2 vaccine clinical trials including the first Ebola vaccine trial in Africa.

Media Contact:
Monika Guttman
Media Relations Specialist
Sabin Vaccine Institute
+1 (202) 662–1841
[email protected]

A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/7ea30f47–768e–4cd0–b0a3–19adff04b2ab


GLOBENEWSWIRE (Distribution ID 9177893)

CSL Behring Announces First Two Patients Treated with HEMGENIX® (etranacogene dezaparvovec) Gene Therapy for Hemophilia B in Europe

MARBURG, Germany, July 04, 2024 (GLOBE NEWSWIRE) — Global biotechnology leader CSL Behring (ASX: CSL) today announced that two hemophilia B patients were treated with the gene therapy HEMGENIX® (etranacogene dezaparvovec) at Hemophilia Treatment Centers in France. This milestone achievement makes HEMGENIX® the first gene therapy administered as a treatment in a real–world setting for hemophilia B in Europe.

HEMGENIX® is the first one–time gene therapy approved in Europe for the treatment of adults with severe and moderately severe hemophilia B, an inherited bleeding disorder caused by the lack of Factor IX (a protein needed to produce blood clots to stop bleeding). It is used in adults without a history of Factor IX inhibitors.1

Following European Commission approval, HEMGENIX® was the first ever therapy to be granted Direct Access in France2, thus enabling the first patients to be treated in Europe outside of the clinical program.

Though effective, current therapies can be time intensive and require regular treatment that can have a substantial impact on a patient’s daily life.3 HEMGENIX® offers a one–time treatment, allowing people living with hemophilia B to produce their own Factor IX, which can lower the risk of bleeding.4

“Only a few decades ago, gene therapy for hemophilia was a distant concept, which has now become reality. Accordingly, the first two patients treated with HEMGENIX® since receiving European approval is a major accomplishment and a testament to the joint commitment of the hemophilia B community, as well as the access and reimbursement authorities, in bringing innovative therapies to patients,” said Dr Lutz Bonacker SVP and General Manager, CSL Behring Commercial Operations Europe. “This milestone has been made possible by the innovative Direct Access scheme adopted in France, allowing patients to benefit from early access to pioneering treatments. We are encouraged to see increasing access to gene therapies in European countries and are fully committed to ensuring that access to potentially life–changing treatment continues.”

HEMGENIX® was granted conditional marketing authorisation by the European Commission (EC) for the European Union and European Economic Area in February 2023, following approval from the U.S. Food and Drug Administration (FDA) in November 2022. It has also been approved by Health Canada, the United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA), Switzerland’s Swissmedic and Australia’s Therapeutic Goods Administration (TGA).

The multi–year clinical development of HEMGENIX® was led by uniQure and sponsorship of the clinical trials transitioned to CSL after it licensed global rights to commercialise the treatment.

About Hemophilia B

Hemophilia B is a life–threatening rare disease. People with the condition are particularly vulnerable to bleeds in their joints, muscles, and internal organs, leading to pain, swelling, and joint damage. Current treatments for moderate to severe hemophilia B include life–long prophylactic infusions of factor IX to temporarily replace or supplement low levels of the blood–clotting factor.  

About HEMGENIX®

HEMGENIX® is a gene therapy that reduces the rate of abnormal bleeding in eligible people with hemophilia B by enabling the body to continuously produce factor IX, the deficient protein in hemophilia B. It uses AAV5, a non–infectious viral vector, called an adeno–associated virus (AAV). The AAV5 vector carries the Padua gene variant of Factor IX (FIX–Padua) to the target cells in the liver, generating factor IX proteins that are 5x–8x more active than normal. These genetic instructions remain in the target cells, but generally do not become a part of a person’s own DNA. Once delivered, the new genetic instructions allow the cellular machinery to produce stable levels of factor IX.

About the Pivotal HOPE–B Trial

The pivotal Phase III HOPE–B trial is an ongoing, multinational, open–label, single–arm study to evaluate the safety and efficacy of HEMGENIX®. Fifty–four adult hemophilia B patients classified as having moderately severe to severe hemophilia B and requiring prophylactic factor IX replacement therapy were enrolled in a prospective, six–month or longer observational period during which time they continued to use their current standard of care therapy to establish a baseline Annual Bleeding Rate (ABR). After the six–month lead–in period, patients received a single intravenous administration of HEMGENIX® at the 2×10^13 gc/kg dose. Patients were not excluded from the trial based on pre–existing neutralizing antibodies (NAbs) to AAV5.

A total of 54 patients received a single dose of HEMGENIX® in the pivotal trial, with 52 patients completing at least three years of follow–up. The primary endpoint in the pivotal HOPE–B study was ABR 52 weeks after achievement of stable factor IX expression (months 7 to 18) compared with the six–month lead–in period. For this endpoint, ABR was measured from month seven to month 18 after infusion, ensuring the observation period represented a steady–state factor IX transgene expression. Secondary endpoints included assessment of factor IX activity.

No serious treatment–related adverse reactions were reported. One death resulting from urosepsis and cardiogenic shock in a 77–year–old patient at 65 weeks following dosing was considered unrelated to treatment by investigators and the company sponsor. A serious adverse event of hepatocellular carcinoma was determined to be unrelated to treatment with HEMGENIX® by independent molecular tumour characterization and vector integration analysis. No inhibitors to factor IX were reported. 

Long–term three–year data presented at the 17th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) 2024 continue to reinforce the potential long–lasting efficacy and safety of HEMGENIX® and the ongoing benefit of this treatment for people living with hemophilia B.

About CSL
CSL (ASX:CSL; USOTC:CSLLY) is a global biotechnology company with a dynamic portfolio of lifesaving medicines, including those that treat hemophilia and immune deficiencies, vaccines to prevent influenza, and therapies in iron deficiency and nephrology. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL – including our three businesses: CSL Behring, CSL Seqirus and CSL Vifor – provides lifesaving products to patients in more than 100 countries and employs 32,000 people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest. For inspiring stories about the promise of biotechnology, visit CSL.com/Vita. For more information about CSL, visit CSL.com.

Media Contacts
Stephanie Fuchs
Mobile: +49 151 584 388 60
Email: [email protected]

References

1 European Medicines Agency. First Gene therapy to treat haemophilia B. Available at: https://www.ema.europa.eu/en/news/first–gene–therapy–treat–haemophilia–b. [Accessed May 2024].
2 Republique Française. Légifrance: Article 62 of Law No. 2021–1754. Available at: https://www.legifrance.gouv.fr/jorf/id/JORFTEXT000048551003 [Accessed May 2024].
3 Leebeek, F & Miesbach, W. (2021) Gene therapy for haemophilia: a review on clinical benefit, limitations, and remaining issues. Blood. Vol 138, Issue 11. pp923–931.
4 Coppens M et al. Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE–B): 24–month post–hoc efficacy and safety data from a single–arm, multicentre, phase 3 trial. The Lancet Haematology 2024; 11(4):E265–E275.


GLOBENEWSWIRE (Distribution ID 1000969961)