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NICE recommends offering PrEP to people at high risk of HIV for first time

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NICE recommends offering PrEP to people at high risk of HIV for first time

 December 16, 2021: “The announcement (Wednesday 15 December 2021) comes as NICE launches a consultation on its draft guideline on reducing sexually transmitted infections.

NICE’s recommendation is backed up by the government’s HIV Action Plan to hit zero new transmissions of HIV by 2030.

PrEP is a pill which prevents infection with HIV by stopping the virus from crossing into the healthy cells and replicating

Evidence from the UK PROUD study reported that PrEP reduced the risk of HIV infection by 86% for men who have sex with men.

Since October 2020, the NHS in England has prescribed PrEP to more than 26,000 people most of whom are gay and bisexual men via specialist sexual health services.

Those people who are taking PrEP must also be supported to get regular HIV testing and STI screening every three months.

Dr Paul Chrisp, director of the centre for guidelines at NICE, said: “Thankfully the number of cases of HIV being recorded each year has fallen considerably and the number of people dying from this virus is also receding in the UK.

“Recommending specialist sexual health clinics offer PrEP to those at the highest risk is another step forward in helping to reduce HIV transmission and could have a transformative effect on helping the UK reach zero HIV transmissions by 2030.

“If you are a person in one of the highest risk groups, I would urge you to speak with your local sexual health clinic to find out if PrEP is an option for you.”

The new NICE guideline has identified key groups in which promotion of PrEP should be targeted in the hope it will make a significant impact in reducing HIV transmission in these groups.

Those at the highest risk, according to BHIVA/BASHH guidelines, include:

  • HIV negative men who have condomless sex with other men.
  • HIV-negative heterosexual men and women having condomless sex with partners who are HIV positive
  • HIV-negative trans women who are identified as being at elevated risk of HIV acquisition through condomless sex

Only specialist sexual health services are authorised to prescribe PrEP on the NHS in England.

The independent NICE guideline committee has recommended awareness should be raised in health professionals who work in primary care, community settings and gender identity clinics about which groups of people are eligible for PrEP

It is also recommended that services that do not provide PrEP should connect people who are interested in PrEP, and eligible for it, to a service that can prescribe it.

Other areas in the guideline are:

  • Improving uptake and increasing the frequency of STI testing by offering a range of STI testing options based on local need, including remote self-sampling, in-person attendance at specialist clinics or in primary care, and outreach services
  • Increasing the uptake of HPV and hepatitis A and B vaccination in gay, bisexual and other men who have sex with men.

A consultation on the recommendations within the draft guideline is open until Monday 31 January 2021 via nice.org.uk”

https://www.nice.org.uk/news/article/nice-recommends-offering-prep-to-people-at-high-risk-of-hiv-for-first-time

Only antibody authorised in the US for pre-exposure prophylaxis of COVID-19

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Only antibody authorised in the US for pre-exposure prophylaxis of COVID-19

December 16, 2021: “AstraZeneca’s Evusheld (tixagevimab co-packaged with cilgavimab), a long-acting antibody combination for the prevention of COVID-19, retained  neutralising activity against the Omicron SARS-CoV-2 variant (B.1.1.529), according to new preclinical data.

In this study, Evusheld’s Inhibitory Concentration 50 (IC50), a measure of neutralising potency of an antibody, was 171 ng/ml and 277 ng/ml in two confirmatory tests, which is within the range of neutralising titres found in someone who has been previously infected with COVID-19. 

Evusheld’s IC50 for the original strain of SARS-CoV-2, previously referred to as the Wuhan strain, was approximately 1.3 ng/ml and 1.5 ng/ml, respectively.

The early data, generated by pseudovirus testing of the full Omicron variant spike against the combination of tixagevimab with cilgavimab, the antibodies that comprise Evusheld, add to the growing body of preclinical evidence demonstrating that Evusheld retains activity against all tested variants of concern to date.

The study was performed independently by investigators at the US Food and Drug Administration (FDA), Center for Biologics Evaluation and Research. The work was supported by US government research funds.

Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca, said: “This study shows Evusheld retains neutralisation activity against the Omicron variant.

By combining two potent antibodies with different and complementary activities against the virus, Evusheld was designed to evade potential resistance with the emergence of new SARS-CoV-2 variants.

Evusheld is the first long-acting antibody to receive emergency use authorisation in the US for pre-exposure prophylaxis of COVID-19, in addition to authorisations in other countries, and we are working with regulators on applications for the use of Evusheld in treating COVID-19.”

The Omicron variant was not in circulation during the Evusheld clinical trials.

The Company is continuing to collect further data to better understand the implications of this observation in clinical practice.

Additional analyses to evaluate Evusheld against the Omicron variant are being conducted by AstraZeneca and third-party laboratories, with data anticipated very soon.

Evusheld received Emergency Use Authorization (EUA) in the US in December 2021 for pre-exposure prophylaxis (prevention) of COVID-19 in people with moderate to severe immune compromise due to a medical condition or immunosuppressive medications and who may not mount an adequate immune response to COVID-19 vaccination, as well as those individuals for whom COVID-19 vaccination is not recommended.

The first doses are expected to become available within days.

About 2% of the global population is considered at increased risk of an inadequate response to a COVID-19 vaccine.

Emerging evidence indicates that protecting vulnerable populations from getting COVID-19 could help prevent viral evolution that is an important factor in the emergence of variants.

Additionally, the TACKLE Phase III outpatient treatment trial of Evusheld showed it reducedthe risk of developing severe COVID-19 or death (from any cause) by 50% compared to placebo in non-hospitalised patients with mild to moderate COVID-19 who had been symptomatic for seven days or less.”

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2021/evusheld-long-acting-antibody-combination-retains-neutralising-activity-against-omicron-variant-in-independent-fda-study.html

10th Global Conference on Health Promotion charters a path for creating ‘well-being societies

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10th Global Conference on Health Promotion charters a path for creating ‘well-being societies

December 15, 2021: “Past epidemics showed us the importance of resilient health systems. The COVID-19 pandemic brought into sharp focus the importance of resilient societies.

The 10th Global Conference on Health Promotion on 13-15 December 2021 marked the start of a global movement on the concept of well-being in societies.

A focus on well-being encourages different sectors to work together to address global challenges and help people take control over their health and lives.

Over 4500 participants of the Global Conference, who met virtually and in Geneva, Switzerland, agreed on a Geneva Charter for Well-being.

The charter builds on the Ottawa Charter for Health Promotion and the legacy of nine global conferences on health promotion. It highlighted the need for global commitments to achieve equitable health and social outcomes now and for future generations, without destroying the health of our planet.

This charter will drive policy-makers and world leaders to adopt this approach and commit to concrete action.

“Health does not begin in a hospital or clinic. It begins in our homes and communities, with the food we eat and the water we drink, the air we breathe, in our schools and our workplaces,” said Dr Tedros Adhanom Ghebreyesus, WHO Director General.

“We have to fundamentally change the way that leaders in politics, the private sector, and international institutions think about and value health, and to promote growth that is based on health and well-being for people and the planet, for countries in all income levels.”

The Charter outlines the necessary elements of a ‘well-being society’ and what needs to be done in order to better prevent and respond to the multiple health and ecological crises we face globally. It identifies key action areas and offers instruments for implementation.

The document encourages five key actions:  

  • Design an equitable economy that serves human development within planetary boundaries;
  • Create public policy for the common good;
  • Achieve universal health coverage;
  • Address the digital transformation to counteract harm and disempowerment and to strengthen the benefits; and
  • Value and preserve the planet.

“It is time to look at how the economy can support the societal goal of well-being, as an investment that is the foundation of productive, resilient and inclusive economies,” said Dr Rüdiger Krech, WHO Director for Health Promotion. 

“We cannot – we must not – go back to the same exploitative patterns of production and consumption, the same disregard for the planet that sustains all life, the same cycle of panic and neglect, and the same divisive politics that fueled this pandemic.”

To change the global development landscape, both the well-being of people and the planet must become central to defining humanity’s progress.

This Charter calls upon nongovernmental and civic organizations, academia, business, governments, international organizations and all concerned to work in society-wide partnerships for decisive implementation of strategies for health and well-being.

These will drive the transformation towards well-being societies in all countries, centering around the most marginalized populations.

Moving forward countries must prioritize health as part of a larger ecosystem that encompasses environmental, social, economic, and political factors.

Universal health coverage, based on strong primary health care, must be at the core of all our efforts, as the cornerstone of social, economic and political stability.

And the narrative around health should be reframed, not as a cost, but as an investment in our common future.”

https://www.who.int/news/item/15-12-2021-10th-global-conference-on-health-promotion-charters-a-path-for-creating-well-being-societies

Novartis initiates new USD 15 billion share buyback highlighting confidence in growth and pipeline

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Novartis initiates new USD 15 billion share buyback highlighting confidence in growth and pipeline

December 16, 2021: “Novartis announced that it will initiate a share buyback of up to USD 15 billion to be executed by the end of 2023.

The buyback is funded through the proceeds from the recent sale of 53.3 million Roche bearer shares.

The Novartis Board of Directors and Executive Committee are confident in the company’s short, medium and long-term growth profile based on strong in-market portfolio, a robust pipeline with up to 20 assets with significant potential, and advanced technology platforms.

With strong operational performance, prospects for earnings growth and the proceeds from the recent sale of the Roche stake, Novartis retains the flexibility to return value to shareholders through the planned USD 15bn share buyback, without compromising the company’s capacity for value-creating bolt-on M&A, whilst providing a strong, growing dividend and reinvesting in the business.

The share buy-back will be executed on the 2nd trading line, is expected to commence in the coming days and to conclude by the end of 2023.

A proposal for an additional CHF 10bn buyback will be presented to the the forthcoming Annual General Meeting on March 4, 2022 to cover the amount exceeding the CHF 8.8bn still available under the existing shareholder authority granted in 2021.

Further information on the growing dividend and growth outlook will be provided at the company’s full year 2021 results on February 2nd, 2022.”

https://www.novartis.com/news/media-releases/novartis-initiates-new-usd-15-billion-share-buyback-highlighting-confidence-growth-and-pipeline

Gilles SCHNEPP takes over as Chairman of the Appointments, Governance and CSR Committee of Sanofi

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Gilles SCHNEPP takes over as Chairman of the Appointments, Governance and CSR Committee of Sanofi

December 16, 2021 – On December 15, 2021, the Board of Directors appointed Gilles Schnepp, an independent director of Sanofi, as Chairman of the Appointments, Governance and CSR Committee.

Gilles Schnepp leaves his position on the Audit Committee.

This appointment follows Serge Weinberg’s decision to step down as Chairman of the Appointments, Governance and CSR Committee, as he will no longer be an independent director in December 2021, after 12 years of service on the Board of Directors of the group, in accordance with the Afep-Medef Code.

As Serge Weinberg’s term of office will expire at the end of the 2023 General Meeting, this decision will also enable the Group to continue preparing his succession in the best possible way.”

https://www.sanofi.com/en/media-room/press-releases/2021/2021-12-16-08-00-00-2353238

FDA Approves First Drug to Prevent Graft Versus Host Disease

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FDA Approves First Drug to Prevent Graft Versus Host Disease

December 15, 2021: “The U.S. Food and Drug Administration approved Orencia (abatacept) for the prophylaxis (prevention) of acute graft versus host disease (aGVHD), a condition that occurs when donor bone marrow or stem cells attack the graft recipient, in combination with certain immunosuppressants.

Orencia may be used in adults and pediatric patients two years of age or older undergoing hematopoietic stem cell transplantation (commonly known as bone marrow transplantation or stem cell transplantation) from an unrelated donor. 

This is the first FDA drug approval for aGVHD prevention and incorporates real world evidence (RWE) as one component of the determination of clinical effectiveness.

RWE is clinical evidence regarding the usage and potential benefits, or risks, of a medical product derived from analysis of real world data – i.e., data relating to patient health status and/or the delivery of health care routinely collected data from a variety of sources, including registry data.

There are significant ongoing efforts at the FDA to incorporate use of high-quality RWE to support regulatory decision-making.

“Acute graft versus host disease can affect different parts of the body and become a serious post-transplant complication,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research.

“By potentially preventing the disease, more patients may successfully undergo bone marrow or stem cell transplantation with fewer complications.”

Acute GVHD is a potentially fatal complication that can occur after stem cell transplantation when the donor’s immune cells (the graft) view the recipient’s body (the host) as foreign, and the donated cells attack the body.

The chances of developing aGVHD increase when the donor and recipient are not related or are not a perfect match. 

The safety and efficacy of Orencia in combination with immunosuppressant therapy in patients age six years and older who underwent stem cell transplantation from a matched or mismatched unrelated donor were evaluated in two separate studies. 

One study, GVHD-1, was a double-blind, placebo-controlled trial of 186 patients who underwent stem cell transplantation from a matched unrelated donor and randomly received Orencia or a placebo in combination with the immunosuppressive drugs.

The study measured severe (grade III-IV) aGVHD-free survival, overall survival and moderate-severe (grade II-IV) aGVHD-free survival six months after transplantation. While severe aGVHD-survival was not significantly improved in patients who received Orencia (87%) compared to patients who received a placebo (75%), patients who received Orencia saw a 97% overall survival rate compared to 84% for patients who received a placebo.

For moderate-severe aGVHD-free survival, patients who received Orencia saw a 50% rate compared to 32% for patients who received a placebo.

Additional evidence of effectiveness was provided by GVHD-2, a registry-based clinical study conducted using real world data from the Center for International Blood and Marrow Transplant Research in patients who underwent stem cell transplantation from a mismatched unrelated donor.

This study analyzed outcomes of 54 patients treated with Orencia for the prevention of aGVHD, in combination with standard immunosuppressive drugs, versus 162 patients treated with standard immunosuppressive drugs alone.

The study measured overall survival six months after transplantation. Patients who received Orencia saw a 98% overall survival rate compared to 75% for patients who received standard immunosuppression alone.

The most common side effects of Orencia for prevention of aGVHD include anemia, hypertension, cytomegalovirus (CMV) reactivation/CMV infection, fever, pneumonia, nosebleed, decreased levels of specific white blood cells called CD4 lymphocytes, increased levels of magnesium in the blood and acute kidney injury.

Patients who receive Orencia should be monitored for Epstein-Barr virus reactivation in accordance with institutional practices and receive preventative medication for Epstein-Barr virus infection before starting treatment and for six months post-transplantation.

Patients should also be monitored for CMV infection/reactivation for six months post-transplant. 

Orencia received Breakthrough, Orphan Drug and Priority Review designations for this indication.

Development of this product was partially supported by the FDA’s Orphan Products Grants Program, which provides grants for clinical studies on safety and efficacy of products for use in rare diseases or conditions.   

Orencia was originally approved by the FDA in 2005 for the treatment of adult rheumatoid arthritis. Orencia is also approved for the treatment of polyarticular juvenile idiopathic arthritis and adult psoriatic arthritis.  

The FDA granted approval of Orencia to Bristol Myers Squibb. 

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence.

Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners.

For this review, the FDA collaborated with Health Canada, Swissmedic and MOH (Israel’s Ministry of Health). The application reviews are ongoing at the other regulatory agencies.”

https://www.fda.gov/news-events/press-announcements/fda-approves-first-drug-prevent-graft-versus-host-disease

Sanofi and GSK announce positive preliminary booster data for their COVID-19 vaccine candidate

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Sanofi and GSK announce positive preliminary booster data for their COVID-19 vaccine candidate

December 15 2021: “Sanofi and GSK announced that a single booster dose of their recombinant adjuvanted COVID-19 vaccine candidate delivered consistently strong immune responses.

Preliminary results from the VAT0002 clinical trial investigating the safety and immunogenicity of the booster showed neutralizing antibodies increased 9- to 43-fold regardless of the primary vaccine received (AstraZeneca, Johnson & Johnson, Moderna, Pfizer/BioNTech) and for all age groups tested.

The booster was well tolerated, with a safety profile similar to currently approved COVID-19 vaccines. This is the most comprehensive booster trial to date to explore boosting across different vaccine technologies used for primary vaccination.

The ongoing global Phase 3 trial, VAT0008, includes regular reviews by an independent Data Safety Monitoring Board (DSMB).

During its last review, the DSMB identified no safety concerns and recommended the trial to continue into early 2022 to accrue more data.

Regulatory authorities require Phase 3 efficacy to be demonstrated in “naive” populations, i.e. participants who have never been infected by the COVID-19 virus (seronegative).

The Phase 3 trial recruited most participants in Q3 2021, coinciding with a significant increase in the number of people infected by the COVID-19 virus globally due to the Delta variant.

To provide the necessary data to regulatory authorities for the booster vaccine submission, the trial will continue to accrue the number of events needed for analysis, with results expected in Q1, 2022.

“These preliminary data show we have a strong booster candidate, whatever primary vaccine you have received.” said Thomas Triomphe, Executive Vice President, Sanofi Pasteur. 

“This is consistent with our efforts to provide relevant responses to evolving public health needs.

While pursuing a phase 3 trial is a challenge in a quickly shifting pandemic environment, we look forward to seeing the results to support submissions of our booster vaccine as quickly as possible.”

Roger Connor, President of GSK Vaccines, added: “As the pandemic threat continues with the current dominant Delta variant and Omicron rapidly gaining ground, booster vaccines will continue to be needed to help protect people over time.

The initial booster data are promising, and we await the phase III results to determine the next steps on making protein-based adjuvanted COVID-19 vaccines available.”

In parallel, Sanofi continues its contribution to global public health needs with the manufacturing of up to half a billion doses from BioNTech/Pfizer, Moderna, and Johnson & Johnson vaccines.

About the booster trial (VAT0002)

The VAT0002 extension trial is the most comprehensive heterologous booster trial conducted to date.

In the first cohort of this trial, the four most-widely approved COVID-19 primary vaccines using mRNA and adenovirus vector technologies were boosted with the Sanofi/GSK protein-based adjuvanted vaccine candidate after full primary vaccination to assess its safety profile and immunogenicity.

Participants in the first cohort (n=521) had previously been vaccinated with the approved dosing schedule of an authorized COVID-19 mRNA vaccine (Moderna, Pfizer/BioNTech,) or adenovirus vector vaccine (AstraZeneca, Johnson & Johnson,).

This preliminary analysis includes data from trial participants who received one 5µg booster dose of the adjuvanted recombinant protein vaccine targeting the D614 parent virus, between four and ten months after a complete primary vaccination schedule.

The trial is ongoing across sites in multiple countries, including the U.S., France, and the UK. To address the emergence of COVID-19 variants of concern, additional trial cohorts are assessing the boosting potential of monovalent and bivalent vaccine formulations also containing the Beta (B.1.351) variant.

More data from this trial are expected during the first half of 2022. The Omicron variant was not circulating during the trial.

Using sera from booster trial participants, testing is underway to establish the ability of the vaccine candidate to cross-neutralize against Omicron.

About the Phase 3 efficacy trial (VAT0008)

The primary endpoint of this ongoing Phase 3, randomized, double-blind, placebo-controlled trial is the prevention of symptomatic COVID-19 in SARS-CoV-2 naïve adults, with secondary endpoints of preventing severe COVID-19 disease and infection.

Stage one of the trial is assessing the efficacy of a vaccine formulation containing the spike protein against the original D614 (parent) virus in more than 10,000 participants >18 years of age, randomized to receive two doses of 10µg vaccine or placebo at day 1 and day 22 across sites in the US, Asia, Africa and Latin America.

A second stage in the trial is evaluating a second bivalent formulation, adding the spike protein of the B.1.351 (Beta) variant.”

https://www.sanofi.com/en/media-room/press-releases/2021/2021-12-15-07-30-00-2352255

Coronavirus (COVID-19) Update: December 14, 2021

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Coronavirus (COVID-19) Update: December 14, 2021

December 14, 2021: “The U.S. Food and Drug Administration announced the following actions taken in its ongoing response effort to the COVID-19 pandemic:

  • Today, the FDA is announcing revisions to the Janssen COVID-19 Vaccine Fact Sheet for Heath Care Providers Administering Vaccine (Vaccination Providers) and the Fact Sheet for Recipients and Caregivers.

    The fact sheets will now include a cContraindication to the administration of the Janssen COVID-19 Vaccine to individuals with a history of thrombosis with thrombocytopenia following the Janssen COVID-19 Vaccine or any other adenovirus-vectored COVID-19 vaccine, and to update the information about the risk of thrombosis with thrombocytopenia syndrome or TTS following vaccination.

    Cases of TTS following administration of the Janssen COVID-19 Vaccine have been reported in males and females 18 years of age and older, with the highest reporting rate of approximately 1 (one) case per 100,000 doses administered in females 30-49 years of age; overall, approximately 1 out of 7 cases has been fatal.

    The FDA and CDC continue to investigate the level of potential excess risk. The FDA continues to find that the known and potential benefits of the Janssen COVID-19 Vaccine outweigh its known and potential risks in individuals 18 years of age and older.

    Individuals should speak to their health care provider to determine which COVID-19 vaccine is most appropriate for their own situation.
  • Testing updates:
    • As of today, 421 tests and sample collection devices are authorized by the FDA under emergency use authorizations (EUAs).

      These include 291 molecular tests and sample collection devices, 89 antibody and other immune response tests and 41 antigen tests. There are 67 molecular authorizations and one antibody authorization that can be used with home-collected samples. “

      There is one EUA for a molecular prescription at-home test, three EUAs for antigen prescription at-home tests, 11 EUAs for antigen over-the-counter (OTC) at-home tests and three EUAs for molecular OTC at-home tests.
    • The FDA has authorized 22 antigen tests and nine molecular tests for serial screening programs. The FDA has also authorized 707 revisions to EUA authorizations.”

https://www.fda.gov/news-events/press-announcements/coronavirus-covid-19-update-december-14-2021

Pfizer To Acquire Arena Pharmaceuticals

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Pfizer To Acquire Arena Pharmaceuticals

December 13, 2021: ” Pfizer Inc. and Arena Pharmaceuticals, Inc. announced that the companies have entered into a definitive agreement under which Pfizer will acquire Arena, a clinical stage company developing innovative potential therapies for the treatment of several immuno-inflammatory diseases.

Under the terms of the agreement, Arena Pharmaceuticals

The boards of directors of both companies have unanimously approved the transaction.

Arena’s portfolio includes diverse and promising development-stage therapeutic candidates in gastroenterology, dermatology, and cardiology, including etrasimod, an oral, selective sphingosine 1-phosphate (S1P) receptor modulator currently in development for a range of immuno-inflammatory diseases including gastrointestinal and dermatological diseases.

“The proposed acquisition of Arena complements our capabilities and expertise in Inflammation and Immunology, a Pfizer innovation engine developing potential therapies for patients with debilitating immuno-inflammatory diseases with a need for more effective treatment options,” said Mike Gladstone, Global President & General Manager, Pfizer Inflammation and Immunology.

“Utilizing Pfizer’s leading research and global development capabilities, we plan to accelerate the clinical development of etrasimod for patients with immuno-inflammatory diseases.”

Arena has built a robust development program for etrasimod, including two Phase 3 studies in ulcerative colitis (UC), a Phase 2/3 program in Crohn’s Disease, a planned Phase 3 program in atopic dermatitis, and ongoing Phase 2 studies in eosinophilic esophagitis and alopecia areata.

In UC, the randomized, placebo-controlled, dose-ranging, Phase 2 study (OASIS) evaluated the efficacy and safety of etrasimod in moderate to severe UC patients over 12 weeks versus placebo.

In the study, most patients who achieved clinical response, clinical remission, or endoscopic improvement at week 12 experienced sustained or improved effects up to week 46 with etrasimod 2 mg in the open-label extension.

Etrasimod also demonstrated a favorable benefit/risk profile, consistent with safety findings reported in the double-blind portion of OASIS.

The findings are encouraging as there remains significant unmet need for safe and effective oral therapies in UC for patients with inadequate response, loss of response, or intolerance to conventional or advanced therapies.

The OASIS trial supported the advancement of the ELEVATE UC 52 and UC 12 trials, which are currently fully enrolled, and for which data are expected in 2022.

In addition, Arena’s pipeline includes two development-stage cardiovascular assets, temanogrel and APD418. Temanogrel is currently in Phase 2 for the treatment of microvascular obstruction and Raynaud’s phenomenon secondary to systemic sclerosis. APD418 is currently in Phase 2 for acute heart failure.

“We’re delighted to announce Pfizer’s proposed acquisition of Arena, recognizing Arena’s potentially best in class S1P molecule and our contribution to addressing unmet needs in immune-mediated inflammatory diseases,” said Amit D. Munshi, President and Chief Executive Officer of Arena.

“Pfizer’s capabilities will accelerate our mission to deliver our important medicines to patients. We believe this transaction represents the best next step for both patients and shareholders.”

Pfizer expects to finance the transaction with existing cash on hand.

Under the terms of the merger agreement, Pfizer will acquire all of the outstanding shares of Arena common stock for $100 per share in cash.

The proposed transaction is subject to customary closing conditions, including receipt of regulatory approvals and approval by Arena’s stockholders.

Pfizer’s financial advisors for the transaction are BofA Securities and Centerview Partners LLC, with Ropes & Gray and Arnold & Porter Kaye Scholer LLP acting as its legal advisors.

Guggenheim Securities, LLC and Evercore Group LLC served as Arena’s financial advisors, while Cooley LLP served as its legal advisor.”

https://www.pfizer.com/news/press-release/press-release-detail/pfizer-acquire-arena-pharmaceuticals

WHO and St. Jude to dramatically increase global access to childhood cancer medicines

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WHO and St. Jude to dramatically increase global access to childhood cancer medicines

December 13, 2021: “The World Health Organization and St. Jude Children’s Research Hospital announced plans to establish a platform that will dramatically increase access to childhood cancer medicines around the world.

The Global Platform for Access to Childhood Cancer Medicines, the first of its kind, will provide an uninterrupted supply of quality-assured childhood cancer medicines to low- and middle-income countries.

St. Jude is making a six-year, US$ 200 million investment to launch the platform, which will provide medicines at no cost to countries participating in the pilot phase. This is the largest financial commitment for a global effort in childhood cancer medicines to date. 

“Close to nine in ten children with cancer live in low- and middle-income countries,” said Dr Tedros Adhanom Ghebreyesus, WHO Director-General. “Survival in these countries is less than 30%, compared with 80% in high-income countries.

This new platform, which builds on the success of the Global Initiative for Childhood Cancer launched with St. Jude in 2018, will help redress this unacceptable imbalance and give hope to many thousands of parents faced with the devastating reality of a child with cancer.” 

Affordable, good quality and uninterrupted cancer medicines for children

Each year, an estimated 400 000 children worldwide develop cancer. The majority of children living in low- and middle-income countries are unable to consistently obtain or afford cancer medicines. As a result, nearly 100 000 children die each year. 

The new platform aims to provide safe and effective cancer medicines to approximately 120 000 children between 2022 and 2027, with the expectation to scale up in future years.

This platform will provide end-to-end support  ̶  consolidating global demand to shape the market; assisting countries with the selection of medicines; developing treatment standards; and building information systems to track that effective care is being provided and to drive innovation. 

“St. Jude was founded on the mission to advance research and treatment of childhood cancer and other catastrophic pediatric diseases. Nearly 60 years later, we stand with the World Health Organization, partner organizations and our Global Alliance collaborators to expand that promise for children worldwide,” said James R. Downing, M.D., president and CEO of St. Jude. “With this platform, we are building the infrastructure to ensure that children everywhere have access to safe cancer medicines.”

This innovative approach will open a new chapter in access to cancer care by addressing medicine availability in low- and middle-income countries that is often complicated by higher prices, interruptions in supply and out-of-pocket expenditures that result in financial hardship.

According to a WHO Noncommunicable Disease Country Capacity survey published in 2020, only 29% of low-income countries report that cancer medicines are generally available to their populations compared to 96% of high-income countries.

By consolidating the needs of children with cancer globally, the new platform will curtail the purchasing of sub-standard and falsified medicines that results from unauthorized purchases and the limited capacity of national regulatory authorities.

“Unless we address the shortage and poor quality of cancer medicines in many parts of the world, there are very few options to cure these children,” said Carlos Rodriguez-Galindo, M.D., executive vice president and chair of the St. Jude Department of Global Pediatric Medicine and director of St. Jude Global.

“Health-care providers must have access to a reliable source of cancer medicines that constitute the current standard of care. We at St. Jude, with our co-founding partners at WHO and many vital partners around the world, can help achieve that.” 

“WHO, St Jude and partners will spare no efforts to get children’s access to cancer medicines on track,” added Dr Bente Mikkelsen, Director of the Department of Noncommunicable Diseases at WHO.

“WHO is on the ground, working with governments to deliver support and services to ensure that all children have access to the best cancer treatment possible.”

Pilot phase in 12 countries

During an initial two-year pilot phase, medicines will be purchased and distributed to 12 countries through a process involving governments, cancer centers and nongovernmental organizations already active in providing cancer care.

Discussions are already ongoing with governments to determine the countries which will participate in this pilot phase. By the end of 2027, it is expected that 50 countries will receive childhood cancer medicines through the platform.  

Kathy Pritchard-Jones, president of the International Society of Paediatric Oncology, said; “We look forward to working with St. Jude and WHO on this journey to ensure all children, everywhere, have access to quality cancer medicines. The platform is bringing forth a dream of our more than 2600 global members.” 

João Bragança, president of Childhood Cancer International, added: “Cancer should not be a death sentence, no matter where a child lives. By developing this platform, St. Jude is helping families get access to lifesaving medicines for their children. Working together, we can change the outcome for cancer-afflicted children around the world.” A continuing collaboration

The World Health Organization and St. Jude Children’s Research Hospital first collaborated in 2018, when St. Jude became the first WHO Collaborating Centre for Childhood Cancer and committed US$ 15 million for the creation of the Global Initiative for Childhood Cancer.

This Initiative supports more than 50 governments in building and sustaining local cancer programmes and aims to increase survival to 60% by 2030.

The platform synergizes with the Global Initiative, with activities implemented through this new effort expected to contribute substantially to the achievement of the Initiative’s goals.

The Global Platform for Access to Childhood Cancer Medicines is part of the Six-Year St. Jude Strategic Plan focused on accelerating progress on catastrophic childhood diseases on a global scale through the institution’s largest investment in research and patient care.”

https://www.who.int/news/item/13-12-2021-who-and-st.-jude-to-dramatically-increase-global-access-to-childhood-cancer-medicines

FDA Publishes Discussion Paper and Seeks Public Input on 3D Printing of Medical Devices at Point of Care

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FDA Publishes Discussion Paper and Seeks Public Input on 3D Printing of Medical Devices at Point of Care

December 10, 2021: “The following quote is attributed to CDRH’s William Maisel, M.D.,M.P.H., director of the Office of Product Evaluation and Quality and Ed Margerrison, Ph.D., director of the Office of Science and Engineering Laboratories.

“The 3D printing of medical devices is at the forefront of innovation and health care.

3D printing at hospitals and other patient-care settings enables health care professionals to quickly create patient-matched devices and anatomical models for surgical planning, as well as many other uses that can help health care facilities rapidly respond to patient needs.

The discussion paper we’re sharing today provides insight into our perspective of the benefits and challenges of 3D printing at hospitals and other points of care and presents a potential approach for regulatory oversight under various scenarios to inform future policy development.

Importantly, the release of this discussion paper is intended to foster discussion and solicit feedback from the public.

This feedback will help build the foundation for an appropriate regulatory approach for 3D printing at the point of care, personalized care for patients and new innovations in this area.”

Additional Information

  • Today, the U.S. Food and Drug Administration published a discussion paper regarding 3D printing medical devices at the point of care (PoC), such as hospitals and doctor’s offices.

    The discussion paper does not constitute guidance; instead, its purpose is to gather feedback from the public to inform future policy development.
  • The discussion paper:
    • Provides relevant background, including terminology, a brief overview of FDA regulation of devices and 3D printing, and how capabilities at a 3D printing facility factor into device safety and effectiveness;
    • Identifies challenges presented by 3D printed medical devices at the PoC and presents a potential approach for regulatory oversight under various scenarios to inform future policy development; and
    • Poses questions to facilitate public comment.”

      https://www.fda.gov/news-events/press-announcements/fda-brief-fda-publishes-discussion-paper-and-seeks-public-input-3d-printing-medical-devices-point

Novartis announces T-Charge™ as first-in-human data at ASH 2021

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December 13, 2021: “Novartis announced the introduction of T-Charge™, the company’s next-generation CAR-T platform that will serve as the foundation for various new investigational CAR-T cell therapies in the Novartis pipeline.

At the 63rd American Society of Hematology Annual Meeting & Exposition (ASH) 2021, Novartis will present early clinical data from ongoing Phase I clinical trials with YTB323 (anti-CD19) and PHE885 (anti-BCMA), the first Novartis CAR-T cell therapies developed using this platform.

Notably, initial efficacy data show a complete response rate of 73% (95% CI: 44.9, 92.2) at month three for the 15 patients with diffuse large B-cell lymphoma (DLBCL) who received dose level two of YTB323.

For the 11 patients with multiple myeloma who received the two highest doses of PHE885, the best overall response was 100%.

The T-Charge platform preserves T cell stemness, the ability to self-renew and mature, which results in a product containing greater proliferative potential and fewer exhausted T cells.

With T-Charge, CAR-T cell expansion occurs primarily within a patient’s body (in-vivo), eliminating the need for an extended culture time outside of the body (ex-vivo).

These unique characteristics of the T-Charge platform may lead to better and more durable responses, improved long-term outcomes and a reduced risk of severe adverse events.

“With T-Charge, we aim to build on the vast knowledge gleaned from early investment in CAR-T research and trials.

Our ambition now is to go beyond incremental advances, to further reimagine CAR-T cell therapy and give patients a higher likelihood of durable responses with the ultimate potential for a cure,” said Jay Bradner, President of the Novartis Institutes for BioMedical Research.

“We are encouraged by these promising early clinical data from the first CAR-T cell therapies produced using the T-Charge platform as we look to accelerate their development and delivery to patients.”

Phase I YTB323 clinical study
YTB323, an investigational, autologous CD19-directed CAR-T cell therapy developed using the T-Charge platform, showed promising results in the diffuse large B-cell lymphoma arm of a first-in-human, multicenter, Phase I dose-escalation study.

Patients received a single treatment of YTB323 at two dose levels (DL). The median administered doses were 2.5×106 CAR+ cells (DL1; n=4) and 12.5×106 CAR+ cells (DL2; n=16).

Of the 15 patients who received YTB323 treatment at DL2 at least three months prior to the data cut-off, the complete response (CR) rate was 73% (95% CI: 44.9, 92.2), including two patients who were in CR prior to treatment with YTB323.

For the 20 patients evaluable for safety, there were no new safety signals beyond those previously known to be related to CD19-directed CAR-T cell therapy. All AEs were reported regardless of the study drug relationship.

Six patients experienced CRS including five of grade 1/2 and one of grade 4. Five patients had neurological adverse reactions (AR), of which two events were considered serious (both at DL2; one experienced grade 3 immune effector cell-associated neurotoxicity syndrome (ICANS) and the other experienced grade 2 seizure that resulted in a grade 3 ICANS).

Recruitment for this trial is ongoing. These data will be presented in an oral session at the ASH annual meeting (Abstract #740; Monday, December 13, 3:00 PM EST).

“The introduction of CAR-T cell therapy led to unprecedented efficacy results for patients facing limited treatment options and a poor prognosis.

Unfortunately, some patients with late-stage B-cell malignancies relapse or do not respond after initial response when treated with traditional CAR-T cell therapies,” said principal investigator Ian W. Flinn, MD, PhD, Director of the Lymphoma Research Program at Sarah Cannon Research Institute in Nashville.

“As part of a community of researchers, physicians and patient advocates, I am hopeful about the promise of novel and next-generation CAR-T cell therapies.”

Phase I PHE885 clinical study
PHE885, an investigational, autologous BCMA-directed CAR-T cell therapy developed using the T-Charge platform, demonstrated promising results in patients with relapsed or refractory multiple myeloma in a first-in-human Phase I, multicenter, dose-escalation study.

Fifteen patients were evaluated for efficacy and safety and the fixed doses received were 2.5×106 (n=4), 5×106 (n=10) and 14.3×106 CAR-T cells (n=1).

Although the follow-up period was brief, PHE885 shows encouraging initial clinical activity with a best overall response of 100% for patients receiving the 5×106 or 14.3×106 CAR-T cell dose, with responses deepening over time.

With a median follow-up of 3.5 months, eight of 15 patients had ongoing responses at the time of data cutoff. Of the evaluable patients at three months post administration, 34% (2/6) were MRD-negative by next-generation sequencing (NGS) at 10-6; 43% (3/7) were MRD-negative at 10-5.

All patients experienced CRS with two patients experiencing grade ≥3 CRS. No patients experienced grade 4 or 5 CRS. All neurotoxicity events (n=4) were nonserious, grade 1-2, reversible, and temporally associated with CRS.

Recruitment for this trial is ongoing.

These data will be presented in a poster presentation session at the ASH annual meeting (Abstract #3864; Monday, December 13, 6:00 PM EST).

Results from pre-clinical studies of YTB323 and PHE885 that served as the scientific rationale to initiate these Phase I clinical trials will also be presented at the meeting (Abstracts #2848 and #2770).

About T-Charge™1-4
T-Charge is a next-generation CAR-T platform, innovated at the Novartis Institutes for BioMedical Research (NIBR), that will serve as the foundation for various new investigational CAR-T cell therapies in the Novartis pipeline.

By implementing the T-Charge platform, we aim to revolutionize CAR-T cell therapy with new products that have the potential to offer patients a higher likelihood of better and more durable responses, improved long-term outcomes and a reduced risk of severe adverse events.

The T-Charge platform preserves T cell stemness (T cell ability to self-renew and mature), an important T cell characteristic closely tied to its therapeutic potential, which results in a product containing greater proliferative potential and fewer exhausted T cells.

With T-Charge, CAR-T cell expansion occurs primarily within the patient’s body (in-vivo), eliminating the need for an extended culture time outside of the body (ex-vivo).

The T-Charge platform, which implements important process efficiencies, will be rapid, compared with traditional CAR-T, and reliable, through simplified processes and streamlined quality control. Multiple CAR-T therapies, including YTB323 and PHE885, are being developed using the Novartis T-Charge platform.”

https://www.novartis.com/news/media-releases/novartis-announces-t-chargetm-next-generation-car-t-platform-first-human-data-ash-2021

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