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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‘PAIX Data Centres’ expands capacity to boost Ghana’s digital economy


PAIX Data Centres, a leading provider of Data Center Solutions, has launched the expansion of its facility in Accra to 1.2 MW intended to boost the digital economy of Ghana.

The expansion was to help meet the demand for digital infrastructure as data consumption rapidly outstrips supply in Ghana and Africa at large.

Mr Bright Tawiah, Managing Director, PAIX Data Centres, Ghana, said, the data centre would help Internet Service Providers (ISPs), cloud providers, and enterprises take full advantage of robust digital infrastructure and improved connectivity, to create online businesses such as e-commerce that would thrive in the digital era.

He said the expansion was crucial and pivotal as the demand for reliable and scalable data centers in Africa is expected to exceed supply by 300 per cent over the next two years.

He said the current installed capacity of 250 MW would need to be significantly increased to 1,200 MW by 2030 to meet the rapidly increasing demand for data, with consumption expected to rise by
40 per cent each year until 2025, according to industry estimates.

Mr Tawiah said the upgraded facility boasts state-of-the-art infrastructure and robust security measures would ensure optimal performance and reliability for mission-critical applications and services.

He said the data center also featured advanced cooling and waste management systems, and the increased integration of renewable energy as a power source, to improve environmental impact.

‘This expansion reaffirms our dedication to providing best-in-class data center solutions to our customers in Accra and beyond. As one of Africa’s digital economy hotspots, Accra plays a vital role in driving innovation and growth across various industries, ‘ he added.

He said they were proud to be at the forefront of the transformation, enabling businesses to thrive in today’s increasingly connected world’, he added.

The Managing Director, said the strategic location in Accra offered low latency connectivity to all major regional and international network
routes and subsea cables, further enhancing the overall efficiency and performance of its services.

Mr Raza Hasnani, Managing Director, Head of Infrastructure Investments of Africa50 and Board Member of PAIX said, data centers were essential to support the creation of businesses that can thrive in the digital era, leading to increased employment opportunities and economic transformation.

He said this significant enhancement not only supports their mission to deliver reliable and high-speed internet services but also reinforces our commitment to fostering digital growth in Ghana.

‘The increased capacity and advanced infrastructure provided by PAIX would enable us to better serve our customers, drive innovation, and contribute to the overall digital transformation of the region,’ he added.

He said Africa50 was committed to supporting PAIX for such significant growth, saying, an amount of $30 million capital was paid to PAIX to support the company’s expansion into a new markets and to upgrade its current fac
ilities.

PAIX Data Centres acquired its first facility in Accra, 2018, and constructed its second data center in Kenya in 2020. However, other centres were being built across other countries.

The company’s mission is to drive digital transformation and foster innovation across Africa, providing world-class data centre solutions that enable businesses to thrive in a digital age.

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

Chief Justice declares July 15 to 19 as ADR Week


Justice Mrs Gertrude Sackey Torkornoo, the Chief Justice, has declared 15 to 19 July 2024 as Alternative Dispute Resolution (ADR) week nationwide.

It would be the second of the three series observed thrice yearly, apart from March and November.

In a press release signed by Mr Alex Nartey, National Coordinator of ADR, and copied to the Ghana News Agency, it said ‘We wish to bring to the attention of the general public that the Chief Justice, has declared Monday, 15th to Friday, 19th July 2024 as ADR Week for this Legal Year Term.’

It gave the theme for the week as, ‘Building the Pillars of Justice through Alternative Dispute Resolution (ADR).’

‘The rationale for the ADR week is to afford the ADR Directorate of the Judicial Service the opportunity to inform the citizenry of the presence of ADR within the court system, its importance in seeking justice and how to take advantage of such an important process for a meaningful access to justice especially, the poor and the vulnerable,’ it added.

‘It is also to
afford court users whose cases are pending before the courts, the benefit of using ADR during the week under the ‘Mass Mediation Exercise.’

The release said a total of 138 Courts, comprising 35 Circuit Courts and 103 District Courts shall participate by devoting the whole week for settlement of Court cases with ADR across the country.

There would be courtroom education on ADR to deepen knowledge of ADR among the litigating public by Judges, Magistrates and ADR officials throughout the week, it noted.

The Bar (lawyers), disputants, the Media and the public are therefore encouraged to participate fully and cooperate with the Judicial Service to make this Legal Year Term’s ADR Week a success, the press release appealed.

It said ADR had been adopted by the Judicial Service of Ghana as part of its adjudication process dubbed, ‘Court-Connected ADR.’

Source: Ghana News Agency

NASPA has not declared any strike


The National Service Personnel Association (NASPA), the official representative body of national service personnel, says the Association has not declared any strike.

NASPA is, therefore, urging the public to disregard the statement from a group calling itself the ‘ Concerned NSS Personnel Association of Ghana’ which has declared a strike for National Service Personnel over delayed payment of monthly allowances.

In a statement issued by the National President of NASPA, Mr Gideon Osei, in Accra said the Association was actively engaged with the management of the National Service Scheme to address the delayed payment of monthly allowances for National Service Personnel.

‘We urge the public to disregard the statement from the ‘Concerned NSS Personnel Association of Ghana,’ (CONSAG) as they are not a recognised organisation.

‘NASPA is actively engaged with the National Service Scheme (NSS) management to address the recent delays in allowance payments.

‘We are pleased to inform all personnel that allowances fo
r April 2024 have been processed and paid and will be reflected in your accounts by Friday, July 5, 2024.

‘Allowances for May 2024 will be paid next week, by Friday, July 12, 2024,’ the statement added.

It said NASPA remained committed to the well-being of service personnel and is working diligently with the management of the Scheme to prevent future delays and ensure the timely reflection of allowances in their e-zwich accounts.

‘We appreciate your patience and understanding during this time. Please remain calm and continue your valuable service to our nation.

‘We assure you that NASPA is actively addressing your concerns for a smooth and supported National Service experience,’ the statement read.

Source: Ghana News Agency

CLOGSAG strike: NRCC offices closed


Offices at the Northern Regional Coordinating Council (NRCC) have been closed due to a nationwide strike initiated by members of the Civil and Local Government Staff Association of Ghana (CLOGSAG).

When the Ghana News Agency visited the Regional Budget, Records Management, Accounts and Internal Audit offices, it observed that the offices were locked.

Some departments, which did not belong to CLOGSAG, were seen opened to business and actively working.

The workers explained that they belonged to different associations and had no authority from their mother unions to join in the strike.

CLOGSAG announced a nationwide strike starting Wednesday, July 3, 2024, citing the government’s failure to implement a new salary structure for its members.

Source: Ghana News Agency

The Beige Group spent over GHC1.8million on publicity – witness


Mr Michael Djanie, the 15th witness in the ongoing trial of Michael Nyinaku, former Chief Executive Officer (CEO) of the collapsed Beige bank, says the Beige Group (TBG) spent GHC1,899.273.83 on publicity.

He said it was as part of activities marking the transition of the Beige Capital Savings and Loans (BCSL) to the Beige Bank (TBB).

Mr Djanie, Head of the brands and communications department of the TBB, made these known during his evidence-in-chief at the High Court presided over by Mrs Afia Serwaa Asare-Botwe, Court of Appeal Judge with additional responsibility at the High Court.

Giving the breakdown, the witness said GHC5,904.92 was paid to the Daily Graphic on August 8, 2017, to inform the public about the new status of BCSL into a universal bank.

He explained that the advertisement cost GHC6,307.53 but payment was made minus withholding tax, adding that the advertisement was placed in the newspaper on July 25, 2017.

A part payment of GHC11,325.60 was made to the Daily Guide, specifically for the f
ront page called ‘strip advertisement,’ explaining that the money was paid on August 16, 2017, for the advert to last for a month and the entire publication was estimated at GHC24,195.60.

Again, out of the GHC38,275.5 budgeted on office branding, GHC11,000.00 was spent as honourarium for media engagement, GHC1,150.00 as Staff reward and the rest on a painting of some agencies under the group, among others, Mr Djanie said.

The witness said a part payment of GHC13,200.00 was made on December 11, 2017, to Websoft Solutions, for the design and development of a new site to replace the BCSL website with that of TBB, saying, the entire amount was GHC17,625.00.

To get a live Facebook streaming of the launch of TBB for the worldwide audience, part payment of GHC2,775.00 was paid to Echohouse Ghana Limited on December 19, 2017, though the entire activity was to cost GHC3,000.00, the Court heard.

He said a total of GHC285,257.06 was allotted for the fabrication and installation of signages and branding for certain b
ranches in the country.

A sum of GHC2,400.00, for instance, from GHC443,651.75 set aside for the production and installation of community facial signages in parts of the country, was paid to the Shama District Assembly for a permit for erecting them, the Court heard.

He told the Court that ‘Think Mohogany Limited,’ an event planning organisation was also paid GHC9,409.32, instead of the entire GHC34,849.35, for rental logistics and decorations work done to make the event venue attractive and colourful.

Mr Djanie also told the Court that one Mr George Nii Lantey, on December 15, 2017, and January 9, 2018, paid GHC45,056.50, each, for the production of branded souvenirs which were distributed to all customers, guests and participants that graced the occasion and to show appreciation to all customers and patrons who visited any of the bank’s branches nationwide.

In all the transactions with the various companies, vendors of TBB automatically had to open an account with the bank to support the growth of TBG.

O
n the other hand, a witness with the brand’s department informed the Court that GHC964,255.32 out of the over GH1.8 million was used for billboard advertising.

He explained that all the five payments made to DDP Outdoor Limited, for rental of billboards on different occasions were used for the advertisements at various locations nationwide.

Breaking it down, he said between February 28, 2017 and April 25, 2018, GHC74,893.62, GHC93,617.02, GHC280,851.06, GHC514,893.62 and GHC35,106.36 were paid to DPP Outdoor Limited.

He added that the GHC35,106.36 paid on April 25, 2018, was a refund of amounts withheld from previous payments made to it in respect of rental billboards, adding that those amounts when withheld were paid to Ghana Revenue Authority (GRA) as withholding taxes.

‘However, GRA allows such funds to be paid to the institutions to whom the funds were due as long as the institutions were able to present evidence of them being exempted from withholding tax,’ he stated.

He said the refund was part of
advertising expenditure incurred on behalf of and for the benefit of the Bank.

Nyinaku has been charged with stealing, money laundering and fraudulent breach of trust.

He has denied all charges, and he is currently on bail pending trial.

Source: Ghana News Agency

GWR cook-a-thon saga: Chefs Association clarifies stance on controversy


The Chefs Association of Ghana (CAG) says it has no hand in the organisation of the recent cook-a-thon event involving one Ebenezer Smith, whose outcome has generated controversy.

Chef Smith announced at a press conference on July 02, 2024, that he had set a new record with a cooking marathon, lasting 820 hours and 25 minutes, producing a certificate purportedly issued by the Guinness World Records (GWR) officials to validate his claim.

However, the GWR has denounced the claim, objecting to the authenticity of the said certificate.

The current record is held by Irishman Alan Fisher, with a time of 119 hours, 57 minutes and 16 seconds.

‘We would like to clarify that Chef Smith is not currently a registered member of the Chefs Association of Ghana.

‘Therefore, his recent activities, including the record-breaking cook-a-thon attempt, were not under the Association’s mandate or supervision,’ the CAG noted in a statement signed by Isaac Sackey, the President.

The statement indicated that the attention of the
Association was drawn during the cooking marathon in February 2024.

‘At that time, we noticed a logo in his materials that closely resembled our own.

‘In the spirit of cooperation, we approached Chef Smith to discuss this and recommended that he register with our Association to align his efforts with our standards and support.’

The statement explained that, despite the leadership’s encouragement, ‘Chef Smith has not yet registered with the Association’.

‘We remain open and supportive of his potential membership and hope he will join us to benefit from our professional community,’ the statement said.

The Association, affirming its commitment to promote the positive image of the Ghanaian chef profession, said it would continue to uplift high professional standards and foster a positive environment for all chefs in the country.

‘While there have been misunderstandings regarding the presentation of a certificate at a recent press conference, we believe that such situations can be learning opportunities for
all involved.

‘We continue to support the development and recognition of Ghanaian chefs who achieve excellence in their craft,’ the statement noted.

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