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: Stephanie.Fuchs@cslbehring.com

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.

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Foreign Minister Receives Copies of Credentials from Five Ambassador Designates


Sierra Leone’s Minister of Foreign Affairs and International Cooperation, Alhaji Timothy Musa Kabba, welcomed five Ambassador Designates to Sierra Leone over the course of Tuesday and Wednesday at his office in Tower Hill, Freetown.

His Excellency Simeon Moriba, the designated Secretary General of the Mano River Union (MRU), presented his Letter of Credence, emphasizing the MRU’s commitment to peace, security, and regional cooperation since its establishment in 1973.

Minister Kabba acknowledged the MRU’s role and expressed optimism about leveraging its potential for economic development in the sub-region.

Angel Antonio Carrascal Gutierrez, Spain’s Ambassador designate, congratulated Sierra Leone on its recent seat in the UN Security Council and discussed areas for enhanced cooperation, including technology, digitalization, and tourism.

Minister Kabba praised Spain’s support in multilateral forums and encouraged deeper investment partnerships with African nations.

Ambassador Designate Alberto Efrain Caste
llar Padilla of Venezuela conveyed greetings from his country’s Foreign Minister and highlighted Venezuela’s interest in bolstering bilateral ties with Sierra Leone in commerce, agriculture, oil, and education.

Minister Kabba welcomed Venezuela’s commitment to education and agriculture cooperation, urging increased scholarships for Sierra Leonean students.

Ambassador Designate Mrs. Ursula Fahringer of Austria noted the longstanding bilateral relations spanning four decades and Austria’s support for projects in Sierra Leone, particularly in girls’ education and judicial institutions.

Minister Kabba expressed gratitude for Austria’s hospitality to President Bio at the OPEC Summit and emphasized continued collaboration for mutual development.

The Ambassador Designate from Sri Lanka highlighted opportunities for economic diplomacy, focusing on energy and mining sectors.

Minister Kabba underscored the historical ties and shared Commonwealth membership between Sierra Leone and Sri Lanka, outlining potential ar
eas for collaboration.

Deputy Minister of Foreign Affairs and International Cooperation, Mrs. Francess Piegie Alghali, extended a warm welcome to the Ambassador Designates on behalf of the Ministry, assuring them of full cooperation during their tenure.

The event was attended by senior officials from the Ministry’s Americas, Europe, and Africa directorates.

Source: Sierra Leone News Agency

CLOGSAG strike enters day two as more offices remain closed

The nationwide strike by members of the Civil and Local Government Staff Association, Ghana (CLOGSAG) has entered day two with most offices in the Ministries closed as the Association presses on Government to implement a new salary structure for the members.

A visit by the Ghana News Agency on Thursday saw empty offices in most of the Ministries with a few workers, particularly security persons, present at some of the offices.

In cases where the premises were opened, the security officers explained that they shared the place with other workers who did not belong to CLOGSAG so the place had to be made accessible to those non-members.

A few workers present also indicated that they were complying with the strike directive, but came to the office to ‘sort out personal issues’.

The whole ministerial enclave still has red bands on tress, fence walls, main entrances, and office doors.

Some of the places the GNA visited were the Ministry of Transport, Office of the Head of the Local Government Service, Ministry
of Gender, Children and Social Protection, Office of the Attorney-General and Ministry of Justice, Office of the Head of Civil Service, Ministry of Food and Agriculture, Ministry of Employment and Labour Relations, and CLOGSAG Secretariat.

The leadership of CLOGSAG on July 2, declared strike over Government’s failure to implement a new salary structure due for implementation January 2023.

Source: Ghana News Agency

MTN supports Elmina Bakatue Festival with GHS10,000 cheque

Telecommunications giant, MTN Ghana, has donated a cheque for GHS10,000 and a drink hamper to the Edina Traditional Council to support this year’s Bakatue Festival.

The gesture, which has been an annual ritual, was a demonstration of the company’s commitment to promoting the rich cultural heritage of the people across the country through festivals.

Mr David Woasey, the Area Sales Manager, Western and Central regions, said MTN was committed to protecting and promoting the African heritage and found festivals as a critical avenue to do so.

The Network was anticipating pressure on the existing capacity given the huge number of people in town, he said.

Consequently, beyond the donation, it will also install a mobile Base Transceiver Station (BTS) to augment the network’s capacity and improve service delivery.

‘We will also set up in various communities to market our product and services and excites our customers in many ways,’ he said.

Nana Kodwo Conduah VI, Omanhen of the Edina Traditional Area, received t
he donation on behalf of the Traditional Council and the festival’s planning committee.

The 2024 Bakatue Festival, which was launched in March, officially commenced on Tuesday, July 2, with the annual rituals at the banks of Benya Lagoon to lift the ban on fishing and noise making.

The rituals, led by Nana Conduah and the Chief Priest, were witnessed by President Nana Addo Dankwa Akufo-Addo and a host of dignitaries, indigenes, revellers and tourists.

The weeklong celebration is on the theme: ‘Patronising Made-in-Ghana Goods and Services to Enhance Economic Development: The Role of the People of Anomansa’.

It will be climaxed with a grand durbar on Saturday, July 6.

Nana Conduah applauded MTN for the continuous support to Elmina and officially gave them the greenlight to advertise and do business during the festival.

Reiterating his argument that the festival was business oriented because of its economic benefits, he indicated that the collaboration between MTN and the Traditional Council was great to f
urther enhance that business venture for mutually benefits.

He urged the company to brand parts of the newly built palace to strengthen the relationship between MTN and the traditional area.

Source: Ghana News Agency

Nandom NCCE fosters police-community collaboration for peacebuilding

The National Commission for Civic Education (NCCE) has taken measures to foster strong collaboration between the Ghana Police Service and the civilian community in the Nandom Municipality towards promoting peace and security in the area.

A dialogue session in the Bu Community was, therefore, organised by the Nandom Municipal Directorate of the NCCE to enhance the trust needed between the security agency and community members for effective law enforcement and community safety.

It was under the European Union (EU) funded: ‘Preventing and Containing Violent Extremism Action in the Northern Regions of Ghana (PCVE).’

Mr Issahaque Tivengnang Angme, the Nandom Municipal Director of the NCCE, said the dialogue was to discuss effective ways of maintaining peace in that community before, during and after the 2024 general election.

‘This activity is aimed at creating platforms where officials of the Ghana Police Service and the communities will interact to defuse suspicion and build trust to improve police-community
relations and confidence,’ he said.

‘By fostering mutual trust, understanding, and cooperation, this approach can address the root causes of radicalisation, enhance community resilience, and create a safer, more inclusive society.’

Inspector Ankora Enoch Delali of the Nandom Municipal Police Command, said violent extremism posed a major threat to national security, community stability and individual safety and stressed the need for strong collaboration among stakeholders at all levels of society to prevent it from plaguing the nation.

He indicated that fear, mistrust and division among others were breeding grounds for violent extremism and should be jealously guided against.

‘Traditional policing methods alone are insufficient to counter issues. The relationship between the police and the communities is critical in fostering a cooperative environment essential for identifying and mitigating extremist threats,’ Inspector Delali said.

Reverend Dr. Stephen A. Koya, the Chairperson of the Nandom Municipal IP
DC, stated that violent extremism was a threat to national security, sanity, stability and safety.

He, therefore, reiterated the need to take measures to facilitate and sustain meaningful police and community trust, cooperation and collaboration in providing early warning signs of violent extremism for the necessary action to be taken to foil it.

These measures should include building trust between the police and the community, ensuring community-friendly police services and increasing attitudinal change, mutual recognition, acceptance and respect of the dignity and integrity of the police and citizens among others.

He encouraged the community members and groups to serve as watchdogs and ambassadors of peace to ensure peace prevailed before, during and after the 2024 general election.

‘As much as possible and practical, we have to resort to genuine alternative dispute resolution methods in all challenges.’

These are inbuilt into and stem from our culture, tradition and history as a people,’ Rev. Dr. Koya
indicated.

The forum brought together traditional and religious leaders and the people of Bu Community to interact with the Police Service on promoting peace and security in the community.

Source: Ghana News Agency

Bricklayer on bail over alleged window damage, theft

The Amasaman Circuit Court has granted GHC50,000.00 bail with two sureties to a bricklayer who allegedly damaged his neighbour’s window and stole her money and cell phone.

David Adjei Nortey, 25, denied causing unlawful damage to the window and the charge of stealing levelled against him.

The sureties are to deposit their valid national identity cards with the Court’s registry.

The court presided over by Ms Enid Marful-Sau, ordered Nortey to make his next appearance on August 6, 2024, for a case management conference.

The court has also asked the prosecution to serve Nortey with the necessary documents before the next adjourned date.

Police Chief Inspector Frederick Awuah-Ansah told the court that Madam Juliana Coffie, the complainant, is a trader who lives in Akramaman, a suburb of Amasaman.

Nortey, who was not known to the complainant also resides in the same neighbourhood.

He said on June 27, 2024, Madam Coffie apprehended Nortey, took him to the Amasaman Police Station, and filed a report alleging
that he had damaged her window, and stolen her GHC9,000.00 and a Tecno Spark 20 mobile phone.

At that point, the Court heard, an investigation found that Madam Coffie and Nortey were neighbours.

Chief Inspector Awuah-Ansah said Nortey’s place of abode was searched and the complainant’s child’s tablet as well as GHC540.00 was found.

Nortey was cautioned, charged and put before the court.

Source: Ghana News Agency

Maritime actors and academia urged to collaborate to safeguard marine domain

Madam Ophelia Mensah Hayford, Minister of Environment, Science, Technology, and Innovation (MESTI), has urged maritime actors to use modern technologies and the strengths of academic institutions to protect the marine domain.

‘Our academic institutions play a pivotal role in driving innovation and building capacity for

maritime security and sustainability. By taking advantage of their research capabilities, we can develop innovative technologies and strategies that are specifically designed to address the maritime challenges we face,’ she said.

The Minister gave the advice on Thursday at a high-level meeting on Maritime Domain Awareness co-organised by the Ghana Navy and University of Ghana (UG) in Accra.

It was on the theme: ‘Maritime Domain Awareness: Role of Naval Forces and Academia’. 

In a speech read on her behalf by Mr Cephas Adjei Mensah, Director of Research, Statistics and Information Management, MESTI, she said Ghana’s maritime resources were under constant threat from illegal activities, envi
ronmental degradation, and over-exploitation.

She suggested the adoption of a comprehensive approach to Maritime Domain Awareness (MDA) to counter those threats.

The Minister said effective MDA allowed maritime authorities to monitor and manage activities, detect and deter illegal activities, and respond swiftly to environmental hazards.

She said such prompt reactions required collaborative and cross-cutting engagements,

partnerships and cooperation.

Madam Hayford said the Ghana Navy played an indispensable role in safeguarding the sovereignty of the state, securing maritime assets, and protecting Ghanaians.

She said the integration of satellites developed by the European Space Agency and European Operational Meteorological Satellite Agency into Ghana’s maritime strategy would provide the necessary insights to make informed decisions and implement proactive measures. 

The Minister expressed gratitude to the European Union Commission for their unwavering support in providing critical technologies.

Rear
Admiral Issah Adam Yakubu, Chief of the Naval Staff said the ‘cross fertilisation of ideas’ between Academia and security agencies was critical to finding cutting edge technological solutions to deal with emerging complex maritime security challenges.

He said the Gulf of Guinea possessed resources that can significantly contribute to the blue economy with industries such as fisheries, oil and gas, maritime transportation and tourism that boosted economic development.

The World Bank defines ‘blue economy’ as the ‘sustainable use of ocean resources for economic growth, improved livelihoods, and jobs while preserving the health of ocean ecosystem.’

The Chief of the Naval Staff stated that piracy and armed robbery at sea, illegal, unreported, and unregulated (IUU) fishing, pollution, and smuggling, among other things, continue to impede the full potential of our blue economy.

He said information sharing among Gulf of Guinea nations through national and regional monitoring mechanisms had made a positive impact
in the maritime environment.

Rear Admiral Yakubu said the availability of some equipment in the maritime operation centres had also greatly improved maritime situational awareness.

‘The Ghana Navy has gone further to explore the use of drone technology to improve our capabilities. Additionally, the Ghana Maritime Authority in collaboration with the Ghana Navy will soon take delivery of state-of-the-art Coastal Surveillance System comprising Over-the-Horizon-Radars, Coastal Radars and Electro-optical cameras which would further extend our ability to gain maritime situational awareness as far as our Exclusive Economic Zone.

‘This hi-tech comprehensive system will be the second of its kind in our Region, after Nigeria’s Falcon Eye system which has enabled them to effectively deal with maritime crimes in their maritime domain. This is a great example of the importance of collaboration and the need to further strengthen such collaborations,’ he said

Prof. Boateng Onwona-Agyeman, Provost of College of Basic and
Applied Sciences, UG, said the institution, through previous initiatives, had provided training support to the Navy in geospatial data analysis and interpretation, and introductory experiences with tools for handling, manipulating and interpreting data such as MATLAB and Python.

He said the Navy had also been instrumental in supporting students and various projects, especially those from the Department of Marine and Fisheries Sciences, by offering sea-going experiences, equipment deployment at sea, and swimming training.

Prof. Onwona-Agyemang said the Navy’s collaboration with the UG would enable personnel to utilise web applications developed for ship and oil spill detections and the monitoring of illegal fishing activities.

As part of the event, a Memorandum of Understanding was signed by the Ghana Navy and the University of Ghana to strengthen collaboration between both institutions.

Source: Ghana News Agency

GIZ hands over vehicles, equipment to Employment Ministry

Gesellschaft fr Internationale Zusammenarbeit (GIZ), with the European Union (EU) through the joint action for Jobs, Migration and Development, is supporting the Labour Department of the Ministry of Employment and Labour Relations, with five pick-up vehicles, furniture, office and IT equipment.

These actions, targeted at improving employment services in five regions across Ghana, aimed to improve workplace quality and services delivery, reinforcing the department’s capacity to support jobseekers and employers alike.

The five regions include Ashanti, Bono, Ahafo, Bono East and Greater Accra Regions of Ghana.

The handing over ceremony, held in Accra, marked a pivotal moment in the collaborative efforts of the EU and the GIZ to enable better access to remote areas for job counselling, employer engagements and job canvassing.

Mr. Wilhelm Hugo, Coordinator, GIZ’s Ghana’s Sustainable Economic Cluster, emphasised the importance of the support in strengthening national, regional and local stakeholder capacities.

H
e said they were enhancing the operational capabilities of the department and promoting employment opportunities and economic growth across Ghana, adding that it demonstrated their strong partnership and shared vision for a robust labour market.

‘We are committed to supporting the Labour Department in delivering efficient and effective employment services’

‘We believe these efforts will pave the way for sustainable development and prosperity for all Ghanaians,’ he said.

Mr. Ignatius Baffour Awuah, the Minister of Employment and Labour Relations, expressed gratitude for the partnership.

He said the support would greatly enhance their operational efficiency, enabling them to better serve the needs of both workers and employers.

The Minister said the vehicles, IT equipment, and furniture would facilitate various activities, including job canvassing, career guidance, and counseling sessions, thereby creating a more conducive environment for both staff and clients.

‘Additionally, the refurbishments are set t
o enhance the overall functionality and aesthetics of the Public Employment Centers, promoting a professional, and welcoming atmosphere,’ he added.

Source: Ghana News Agency