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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GFA signs four-year partnership agreement with 5 Star Energy Drink


The Ghana Football Association (GFA) has signed a four-year partnership with Multi Pac Limited, manufacturers of 5 Star energy drink as its official beverage partner.

Multi Pac Limited as part of the agreement, would provide the GFA with products and cash on annual basis.

The football association had over the years gained the trust of corporate Ghana following its commitment in developing sports.

Mr. Kurt Edwin Simeon-Okraku, President of the GFA speaking at the ceremony expressed excitement having reached an agreement with one of the leading beverage companies in the country.

He said the deal was a testament of the growing confidence corporate Ghana had in the football association.

‘The support from 5-star energy drink would undoubtedly energize our players and teams, contributing to their performance on and off the field. We look forward to a fruitful and mutually beneficial relationship,’ he said.

The GFA President said the new achievement not only benefit their partners, but also boost the energy le
vels of the various national teams.

Mr. Okraku said he was hopeful the partnership would yield positive results in the coming years.

He thanked Multi Pac Limited for supporting the GFA’s vision of developing sports in Ghana.

Mr. Rabih Bourji, General Manager of 5-Star Energy Drink said he was excited with the unveiling of the partnership which had been in the pipeline for long.

‘We are thrilled to partner with the Ghana Football Association. Football is a significant part of our culture, and supporting the GFA aligns with our commitment to promoting sports and healthy living.’

He said the sponsorship deal would go a long way to create great synergies and bring immense value to both the GFA and 5-Star Energy Drink.

Present at the ceremony was Mr. Mark Addo, GFA Vice President, Mr. Eugene Noel Nobel Executive Council Member, and John Paintsil, Black Stars deputy coach.

Source: Ghana News Agency

President applauds Parliament for initiating the maiden Democracy Cup match


The President, Nana Addo Dankwa Akufo-Addo has applauded the Parliament of Ghana for initiating the ‘Democracy Cup’ competition as part of the activities marking 30 years of Parliamentary Democracy in Ghana.

‘I am excited with this special and excellent initiative, because it would deepen and entrench the culture of democracy in the country,’ the President said this on Thursday, July 4, when the Speaker of Parliament, Mr. Alban S.K. Bagbin presented the special trophy to him at the Jubilee House.

‘I am in support of this initiative and i declare my total support for it. I hope it would remain on schedules for years to come.

‘A football match to celebrate 30 years of parliamentary democracy would be an opportunity to bring the youth closer to works of parliament and also imbibe in them the tenets of democracy,’ he added.

The President noted that, the fourth republic had proven to be the most enduring and successful hence the need to celebrate the achievements under it.

On his part, Mr. Bagbin said the pur
pose of the Democracy Cup match was to use football to engage the youth to join in the celebration.

He added that it was a platform to preach peace and unity ahead of the 2024 elections and also promote democratic culture in Ghana.

Mr. Bagbin called on the President to lead the campaign to make democracy relevant to the citizens and took the opportunity to invite him to the match.

Accra Hearts of Oak and Asante Kotoko would on Wednesday, July 17, battle each other in the maiden edition of the Democracy Cup match at the Accra Sports Stadium on Wednesday, July 17.

There would also be special curtain raiser between ex Black Stars players and Members of Parliament.

Source: Ghana News Agency

Ghana gets tough draw in AFCON 2025 qualifiers, set to face Sudan, Angola


The Black Stars of Ghana have been paired against Sudan, Angola, and Niger in Group F of the qualifiers for the 2025 African Cup of Nations (AFCON).

The top two teams in each group would qualify for the 2025 AFCON, to be held in Morocco from December 2025 to January 2026.

The Black Stars of Ghana would begin their AFCON 2025 qualification campaign in September 2024, aiming for their 25th appearance at the continental tournament.

Ghana, who have had terrible outings in the last two editions of the AFCON, would be aiming to qualify for the 35th edition of the biennial competition.

The four-time African Champions would be looking to end their long trophy drought, having last won it back in 1982.

Coach Otto Addo, who has had a tremendous start to his second spell with the Black Stars in the 2026 World Cup qualifiers, would be seeking to replicate the same form in the 2025 AFCON qualifiers.

Source: Ghana News Agency

HIV breakthrough: drug trial shows injection twice a year is 100 per cent effective against infection


A large clinical trial in South Africa and Uganda has shown that a twice-yearly injection of a new pre-exposure prophylaxis drug gives young women total protection from HIV infection.

The trial tested whether the six-month injection of lenacapavir would provide better protection against HIV infection than two other drugs, both daily pills. All three medications are pre-exposure prophylaxis (or PrEP) drugs.

Physician-scientist Linda-Gail Bekker, principal investigator for the South African part of the study, tells Nadine Dreyer what makes this breakthough so significant and what to expect next.

Tell us about the trial and what it set out to achieve

The Purpose 1 trial with 5,000 participants took place at three sites in Uganda and 25 sites in South Africa to test the efficacy of lenacapavir and two other drugs.

Our mission is to share knowledge and inform decisions.

Lenacapavir (Len LA) is a fusion capside inhibitor. It interferes with the HIV capsid, a protein shell that protects HIV’s genetic material
and enzymes needed for replication. It is administered just under the skin, once every six months.

The randomised controlled trial, sponsored by the drug developers Gilead Sciences, tested several things.

The first was whether a six-monthly injection of lenacapavir was safe and would provide better protection against HIV infection as PrEP for women between the ages of 16 and 25 years than Truvada F/TDF, a daily PrEP pill in wide use that has been available for more than a decade.

Secondly, the trial also tested whether Descovy F/TAF, a newer daily pill, was as effective as F/TDF. The newer F/TAF has superior pharmacokinetic properties to F/TDF. Pharmacokinetic refers to the movement of a drug into, through, and out of the body. F/TAF is a smaller pill and is in use among men and transgender women in high-income countries.

The trial had three arms. Young women were randomly assigned to one of the arms in a 2:2:1 ratio (Len LA: F/TAF oral: F/TDF oral) in a double blinded fashion. This means neither the part
icipants nor the researchers knew which treatment participants were receiving until the clinical trial was over.

In eastern and southern Africa, young women are the population who bear the brunt of new HIV infections. They also find a daily PrEP regimen challenging to maintain, for a number of social and structural reasons.

During the randomised phase of the trial none of the 2,134 women who received lenacapavir contracted HIV. There was 100 percent efficiency.

By comparison, 16 of the 1,068 women (or 1.5%) who took Truvada (F/TDF) and 39 of 2,136 (1.8%) who received Descovy (F/TAF) contracted the HIV virus.

The results at a recent independent data safety monitoring board review led to the recommendation that the trial’s ‘blinded’ phase should be stopped and all participants should be offered a choice of PrEP.

This board is an independent committee of experts who are put in place at the start of a clinical trial. They see the unblinded data at stipulated times during the trial to monitor progress and saf
ety. They ensure that a trial does not continue if there is harm or a clear benefit in one arm over others.

What is the significance of these trials?

This breakthrough gives great hope that we have a proven, highly effective prevention tool to protect people from HIV.

There were 1.3 million new HIV infections globally in the past year. Although that’s fewer than the 2 million infections seen in 2010, it is clear that at this rate we are not going to meet the HIV new infection target that UNAIDS set for 2025 (fewer than 500,000 globally) or potentially even the goal to end Aids by 2030.

PrEP is not the only prevention tool.

PrEP should be provided alongside HIV self-testing, access to condoms, screening and treatment for sexually transmitted infections and access to contraception for women of childbearing potential.

In addition, young men should be offered medical male circumcision for health reasons.

But despite these options, we haven’t quite got to the point where we have been able to stop new infect
ions, particularly among young people.

For young people, the daily decision to take a pill or use a condom or take a pill at the time of sexual intercourse can be very challenging.

HIV scientists and activists hope that young people may find that having to make this ‘prevention decision’ only twice a year may reduce unpredictability and barriers.

For a young woman who struggles to get to an appointment at a clinic in a town or who can’t keep pills without facing stigma or violence, an injection just twice a year is the option that could keep her free of HIV.

What happens now?

The plan is that the Purpose 1 trial will go on but now in an ‘open label’ phase. This means that study participants will be ‘unblinded’: they will be told whether they have been in the ‘injectable’ or oral TDF or oral TAF groups.

They will be offered the choice of PrEP they would prefer as the trial continues.

A sister trial is also under way: Purpose 2 is being conducted in a number of regions including some sites in Africa amon
g cisgender men, and transgender and nonbinary people who have sex with men.

It’s important to conduct trials among different groups because we have seen differences in effectiveness. Whether the sex is anal or vaginal is important and may have an impact on effectiveness.

How long until the drug is rolled out?

We have read in a Gilead Sciences press statement that within the next couple of months the company will submit the dossier with all the results to a number of country regulators, particularly the Ugandan and South African regulators.

The World Health Organization will also review the data and may issue recommendations.

We hope then that this new drug will be adopted into WHO and country guidelines.

We also hope we may begin to see the drug being tested in more studies to understand better how to incorporate it into real world settings.

Price is a critical factor to ensure access and distribution in the public sector where it is badly needed.

Gilead Sciences has said it will offer licences to co
mpanies that make generic drugs, which is another critical way to get prices down.

In an ideal world, governments will be able to purchase this affordably and it will be offered to all who want it and need protection against HIV.

Source: Ghana News Agency

Bono Region records highest HIV prevalence rate in 2023 – GAC


The Bono Region recorded the highest HIV prevalence rate of 2.12 per cent in 2023, exceeding the national prevalence rate of 1.53 per cent, Mr Ahmed Ibrahim Bimbilla, the Bono, Bono East and Ahafo Regional Technical Coordinator of the Ghana AIDS Commission (GAC), has said.

He said the region now had 18,564 Persons Living with HIV, and advised the people to be mindful of their sexual behaviours, avoid unprotected sex and multiple sex partners.

Mr Bimbilla gave the advice at a stakeholder’s meeting organised by the GAC in Sunyani, and called for concerted efforts to intensify the HIV/ AIDS campaign to prevent the spread of the disease.

The meeting was attended by some heads of departments and agencies, traditional authorities and civil society organisations and actors.

Giving the breakdown, Mr Bimbilla said Greater Accra had the second highest prevalence rate of 1.98 per cent, Eastern Region 1.95 per cent, Western North, 1.82 per cent, Ahafo, 1.79 per cent, and Ashanti 1.70 per cent in that order.

The Nort
h -East and Northern regions recorded the lowest prevalence rate of 0.43 percent and 0.38 per cent respectively, he added.

Madam Justina Owusu-Banahene, the Bono Regional Minister, said HIV and AIDS remained a public health threat in the region, and called on everybody to support the campaign against the spread of the virus.

She expressed concern over the poor parental care in the area, which was contributing to promiscuous lifestyles among the youth, thereby exposing them to HIV and related Sexually Transmitted Infections.

The Regional Minister, therefore, called on parents and guardians to endeavour to monitor the movements of their children, draw them closer, identify their needs and help them address them.

Source: Ghana News Agency

Stigmatisation hampering fight against HIV – AIDS Commissions


Mr Dramani Yakubu, the Western Regional Technical Coordinator of the Ghana AIDS Commission (GAC), says stigmatisation and discrimination of persons living with HIV are a major challenge to the country’s objective of ending HIV/AIDS as a public health threat by 2030.

He said stigma prevented people with the virus from voluntarily accessing HIV care, adherence support and other essential services, thus hampering progress in the national response against those infections.

Mr Yakubu was speaking at a Western Regional stakeholders’ coordination forum, organised by the GAC, to discuss critical issues on continuity of care, adherence support and other services for persons living with HIV and key population.

The forum, held at Sekondi, was attended by members of the Regional Committee of GAC, representatives from Civil Society Organisations (CSOs) working around HIV/AIDS, and Ghana Health Service among other stakeholders.

It provided a platform for participants to understand the diverse efforts of partners and le
arn and share experiences in the implementation of programmes towards the fight against HIV infections.

‘The badmouthing and pointing fingers at persons who are living with HIV is not the best, because if it happens like that, such persons go into hiding without accessing the care they need,’ Mr Yakubu said.

‘… And when that also happens it means they can infect other people, which will hold back the progress we are making to achieve the national target of ending HIV/AIDS as a public health threat by 2030.’

The Technical Coordinator, therefore, called on the citizenry to unite and support efforts to end stigma and enhance the progress in curbing the infection.

Mr Kwabena Okyere Darko-Mensah, the Western Regional Minister, in a speech read on his behalf, said despite the challenges, the region had made significant strides in the response against HIV/AIDS over the past years.

‘Our collective efforts have resulted in notable achievements such as expanding access to HIV services, and the number of facilities
offering HIV treatment services has increased from 26 to 76, bringing essential care closer to many in our communities,’ he said.

However, he expressed worry over the increase in new infections in the region from 1,101 in 2022 to 1,235 in 2023, saying: ‘This data prompts us to question why our efforts are not yielding greater impact.’

Mr Darko-Mensah, also the Chairman of the Western Regional Committee of the Ghana AIDS Commission, called for effective collaboration between stakeholders to ensure a successful implementation of sustainable programmes towards achieving the desired outcomes.

The CSOs at the forum took turns to update participants on the projects and activities being undertaken towards the response against HIV/AIDS in the region.

Source: Ghana News Agency

Africa CDC calls for subsidised charges on scientific publications


The Africa Centres for Disease Control and Prevention (Africa CDC) has advocated for reduced article processing charges to facilitate the publication of scientific findings by more African countries and researchers.

This would provide additional resources to address some of the key pillars of the new public health order, Professor Nicaise Ndembi, Senior Advisor Africa CDC and Editor-In-Chief of the Journal of Public Health in Africa (JPHIA) has said.

He made the call at a meeting organised by the Journal of Public Health in Africa and the National Vaccine Institute, Ghana in Accra on Thursday.

The meeting, which was organized for researchers from Ghana and other African countries, aimed to Africanize scientific excellence and promote the Africa CDC journal.

It was also aimed at building the capacities of African scientists in writing and publishing findings from their own research and implementation science.

Prof Ndembi said more research was urgently required because Africa was the most vulnerable regio
n to infectious disease threats.

He noted that every year, 100 health emergencies are documented, with infectious diseases accounting for 90 per cent and zoonotic diseases responsible for 70 per cent, adding that ‘no country is well prepared for an infectious disease outbreak, and Africa is even worse.’

Prof Ndembi said that in constructing the global health architecture, African countries must adhere to the Africa CDC’s new five main pillars of public health order.

The new public health order calls for stronger public health institutions, a larger public health workforce, greater vaccine, diagnostic, and therapeutic production, more domestic health-care investment, and respectful action-oriented partnerships.

Prof Ndembi called on African countries to prioritize research findings to enhance public health systems.

Speaking on Knowledge Hubs and the Publishing Landscape in Africa, Dr Loice Cushny of the Africa CDC, called for collaboration and cross-learning among different stakeholders and jurisdictions.

This will serve as a platform for information exchange, and knowledge sharing, minimise duplication and time waste in inventing the wheel, she added.

Prof Willam Kwabena Ampofo, the Chief Executive Officer of the National Vaccine Institute, called on scientists to document their works to create the enabling environment for research development and manufacturing of vaccines in Ghana.

He said that the JPHIA had been in existence for five years and that it was time for African researchers to publish in the journal on the different high-quality vaccines being developed.

Dr Wisdom Atiwoto, Director of Research Statistics and Information Management at the Ministry of Health, asked researchers to work with the Ministry to ensure the successful application of research findings.

During the panel discussion, scientists advocated for robust mentorship programmes that would allow scientific students to get guidance throughout their practice.

They also appealed for the necessary resources and time to be able to publi
sh their scientific findings for proper public health system implementation.

Dr. Gifty Dufie Ampofo, a representative from the University of Allied Health Sciences (UHAS) Ho, urged scientific students to understand the need of publishing as early as possible to develop interest.

Source: Ghana News Agency