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Pfizer Invites Public to View and Listen to Webcast of July 28 Conference Call with Analysts

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Pfizer Invites Public to View and Listen to Webcast of July 28 Conference Call with Analysts

June 24, 2022: “Pfizer Inc. invites investors and the general public to view and listen to a webcast of a conference call with investment analysts at 10 a.m. EDT on Thursday, July 28, 2022.

The purpose of the call is to provide an update on Pfizer’s results, as reflected in the company’s Second Quarter 2022 Performance Report, to be issued that morning.

To view and listen to the webcast and view the Performance Report, visit our web site at www.pfizer.com/investors.

Information on accessing and registering for the webcast will be available at www.pfizer.com/investors beginning today. Participants are advised to register in advance of the conference call.

You can also listen to the conference call by dialing either 800-456-4352 in the United States and Canada or 785-424-1086 outside of the United States and Canada. The password is “PFEQ222”.

The transcript and webcast replay of the call will be made available on our web site atwww.pfizer.com/investors within 24 hours after the end of the live conference call and will be accessible for at least 90 days.

About Pfizer: Breakthroughs That Change Patients’ Lives

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives.

We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines.

Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time.

Consistent with our responsibility as one of the world’s premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world.

For more than 170 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.Pfizer.com.

In addition, to learn more, please visit us on www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

Disclosure Notice:The webcast may include forward-looking statements about, among other things, our anticipated operating and financial performance, reorganizations, business plans, strategy and prospects; expectations for our product pipeline, in-line products and product candidates, including anticipated regulatory submissions, data read-outs, study starts, approvals, clinical trial results and other developing data, revenue contribution, growth, performance, timing of exclusivity and potential benefits; strategic reviews; capital allocation objectives; dividends and share repurchases; plans for and prospects of our acquisitions, dispositions and other business development activities, and our ability to successfully capitalize on these opportunities; manufacturing and product supply; and our efforts to respond to COVID-19, including the Pfizer-BioNTech COVID-19 vaccine (Comirnaty) and our oral COVID-19 treatment (Paxlovid), that are subject to substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements.

A description of these risks and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the fiscal year ended December 31, 2021 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors” and “Forward-Looking Information and Factors That May Affect Future Results”, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at www.sec.gov and www.pfizer.com.

The forward-looking statements in the webcast speak only as of the original date of the webcast.

Pfizer assumes no obligation to update forward-looking statements contained in the webcast as the result of new information or future events or developments.

Sanofi-GSK first to report successful efficacy study against Omicron

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Sanofi-GSK first to report successful efficacy study against Omicron

June 24, 2022: “Sanofi and GSK announce positive data from their vaccine trial which evaluated an adjuvanted bivalent D614 and Beta (B.1.351) vaccine candidate. Sanofi-GSK’s vaccine is the first candidate to demonstrate efficacy in a placebo-controlled trial in an environment of high Omicron variant circulation.

The vaccine candidate showed a favorable safety and tolerability profile.

Earlier this month Sanofi reported positive data from two trials conducted with its new next-generation COVID-19 booster vaccine candidate modelled on the Beta variant antigen and including GSK’s pandemic adjuvant.

The data supporting this next-generation booster vaccine will be submitted to regulatory authorities and indicate the potential of Sanofi-GSK’s next-generation Beta-based booster to be a relevant response to public health needs.

Thomas Triomphe
Executive Vice President Vaccines, Sanofi
“Today’s results reinforce the strong potential for the Beta antigen to confer broad protection against multiple strains that cause COVID-19.

With the immunogenicity data from our Beta-booster vaccine, they support our belief that, in a largely seropositive world, a next-generation Beta booster vaccine could provide protection against variants like Omicron.

mRNA has proven speed to market; we are demonstrating here the efficacy that our recombinant protein platform can provide to the world. We look forward to completing our submissions to regulatory authorities and are ready to contribute to ongoing vaccination campaigns with our next-generation booster.”

Roger Connor
President of GSK Vaccines
“These positive data show efficacy of our protein-based, bivalent adjuvanted vaccine candidate in an environment of high Omicron variant circulation.

Our vaccine candidate has the potential to make an important contribution to public health as the pandemic evolves further.

We are looking forward to the discussions with regulatory authorities with the aim of making our vaccine candidate available later this year.”

In Stage 2 of the Phase 3 COVID-19 vaccine trial VAT08 of more than 13,000 participants 18 and above years of age, the Sanofi-GSK Beta-containing vaccine candidate demonstrated an efficacy of 64.7% (95% confidence interval [CI, 46.6, 77.2]) against symptomatic COVID-19 and 72% efficacy (95% confidence interval [CI, 45.8, 86.6]) in Omicron-confirmed symptomatic cases (sequencing was performed for 71 cases out of 121 total cases to date).

In previously seropositive populations, the Sanofi-GSK vaccine candidate demonstrates an overall efficacy of 75.1% (95% confidence interval [CI, 56.3, 86.6]) against symptomatic infection, and 93.2% (95% confidence interval [CI, 73.2, 99.2]) in Omicron-confirmed symptomatic cases, according to the sequencing analysis performed to date.

Throughout Stage 1 and Stage 2 of the VAT08 trial (~23,000 participants in total), the Sanofi-GSK vaccine demonstrated a favorable safety and tolerability profile.

These efforts are supported by federal funds from the Biomedical Advanced Research and Development Authority, part of the office of the Assistant Secretary for Preparedness and Response at the U.S. Department of Health and Human Services in collaboration with the U.S. Department of Defense Joint Program Executive Office for Chemical, Biological, Radiological and Nuclear Defense under Contract # W15QKN-16-9-1002 and the National Institute of Allergy and Infectious Diseases (NIAID).

About the Sanofi and GSK partnership
In the collaboration between the two companies, Sanofi provides its recombinant antigen and will be the marketing authorization holder.

GSK contributes with its pandemic adjuvant, both established vaccine platforms that have proven successful against influenza.”

https://www.sanofi.com/en/media-room/press-releases/2022/2022-06-24-05-29-02-2468538

Novartis receives positive CHMP opinion for Scemblix® for chronic myeloid leukemia

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Novartis receives positive CHMP opinion for Scemblix® for chronic myeloid leukemia

June 24, 2022: “Novartis announced that the Committee for Medicinal Products for Human Use (CHMP) of the EMA has adopted a positive opinion and recommended granting marketing authorization for Scemblix® (asciminib) for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP), previously treated with two or more tyrosine kinase inhibitors (TKIs). 

If approved, Scemblix will be the first CML treatment in Europe that works by specifically targeting the ABL myristoyl pocket (also known as a STAMP inhibitor in scientific literature), representing an important therapeutic advancement for patients who experience intolerance and/or resistance to currently available TKI therapies.

It is estimated that, every year, more than 6,300 people will be diagnosed with CML in Europe.

While many patients will benefit from available TKI therapies, a significant proportion may experience intolerance or resistance to these treatments.

In an analysis of patients with CML treated with two prior TKIs, approximately 55% reported intolerance to previous treatment; and a pooled analysis of patients in the second-line setting revealed that up to 70% are unable to achieve major molecular response (MMR) within two years of follow-up.

“Although CML treatments have advanced over the last 20 years, many patients continue to experience side effects and resistance to treatment, affecting their quality of life and putting them at risk of disease progression or even death,” says Dr. Andreas Hochhaus, Head of the Department of Hematology and Medical Oncology at Jena University Hospital in Germany.

“If approved, the novel mechanism of action of Scemblix brings us another option to combat these challenges faced by patients — offering new hope in the management of their disease.”

The positive CHMP opinion for Scemblix is based on results from the pivotal Phase III ASCEMBL trial, which showed a near doubling of MMR rate for patients treated with Scemblix vs. Bosulif®* (bosutinib) (25.5% vs. 13.2%) at 24-weeks, with a more than three times lower discontinuation rate due to adverse reactions (5.8% vs. 21.1%).

The most common (incidence ≥ 20%) adverse reactions reported in this analysis were thrombocytopenia (29.5%) and neutropenia (23.1%) in the Scemblix arm; and diarrhea (71.1%), nausea (46.1%), increased ALT (28.9%), vomiting (26.3%), rash (23.7%), increased AST (21.1%) and neutropenia (21.1%) in the Bosulif arm.

These results were confirmed in longer-term follow-up, where the MMR rate at week 96 was more than double with Scemblix (37.6%, 95% CI: 29.99-45.65) compared with Bosulif (15.8%, 95% CI: 8.43-25.96).

This data was shared at oral presentations during the  American Society for Clinical Oncology (ASCO) and the European Hematology Association (EHA) annual meetings in June 2022.

“We are pleased with the recommendation of Scemblix and hope to offer patients living with CML in Europe timely access to this innovative therapy, if approved,” said Haseeb Ahmad, President, Europe, Novartis.

“We’ve worked relentlessly to improve CML care over the past two decades, and must seize this opportunity to help patients in need achieve better outcomes.

With the strong clinical results seen to-date, we believe we have the potential to transform the standard of care in CML yet again with Scemblix.”

The CHMP recommended approval of Scemblix in CML will be referred to the European Commission (EC). The EC will review the CHMP recommendations and deliver a final decision in the coming months.

About Scemblix® (asciminib)
Scemblix is the first CML treatment that acts as a STAMP inhibitor, specifically targeting the ABL myristoyl pocket.

This novel mechanism of action may help address resistance in patients with CML previously treated with two or more TKIs and overcome mutations at the defective BCR::ABL1 gene, which is associated with the over-production of leukemic cells

Scemblix represents an important development for patients who experience resistance and/or intolerance to currently available TKI therapies, and it is being studied across multiple treatment lines for CML-CP, both as a monotherapy and in combination.

Specifically, the ASC4FIRST Phase III study (NCT04971226) evaluates Scemblix in newly diagnosed adult patients with Ph+ CML-CP vs. an investigator-selected TKI, with recruitment proceeding ahead of plan.

Novartis has initiated regulatory filings for Scemblix in multiple countries and regions across the globe.

In October 2021, the US FDA granted accelerated approval of Scemblix for adult patients with Ph+ CML-CP, previously treated with two or more TKIs based on MMR rate at 24 weeks, and full approval for adult patients with Ph+ CML-CP with the T315I mutation.

In accordance with the Accelerated Approval Program, continued approval for the first indication may be contingent upon verification and description of clinical benefit from confirmatory evidence. Further data has been shared with the FDA for evaluation.

Scemblix has received approval in several countries outside the US for adult patients with Ph+ CML-CP with resistance or intolerance to at least two or more previous therapies.”

https://www.novartis.com/news/media-releases/novartis-receives-positive-chmp-opinion-scemblix-novel-treatment-adult-patients-chronic-myeloid-leukemia

Novartis Tabrecta receives EC approval for METex14 skipping advanced nSCLC

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Novartis Tabrecta receives EC approval for METex14 skipping advanced nSCLC

June 22, 2022: “Novartis announced that the EC approved Tabrecta® (capmatinib) as a monotherapy for treatment of adults with advanced non-small cell lung cancer (NSCLC) harboring alterations leading to mesenchymal-epithelial-transition factor gene (MET) exon 14 (METex14) skipping who require systemic therapy following prior treatment with immunotherapy and/or platinum-based chemotherapy.

The approval follows a positive opinion issued in April by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) and is applicable to all 27 European Union member states plus Iceland, Norway and Liechtenstein.

“Patients with METex14 skipping alterations typically have a more advanced form of lung cancer that is often associated with a poor prognosis and limited response to standard therapy, including immunotherapy,” said Juergen Wolf, MD, from the Center for Integrated Oncology, University Hospital Cologne, Germany, and lead investigator of the GEOMETRY mono-1 trial.

“With the approval of Tabrecta in Europe, supported by advances in biomarker testing that can help doctors direct treatment more precisely, patients with this specific genomic profile have a new targeted treatment option that can lead to improved outcomes.”

The approval is based on results from the Phase II GEOMETRY mono-1 trial that demonstrated positive overall response rates (ORR) among adult patients with advanced NSCLC whose tumors had alterations leading to METex14 skipping.

In the study, among 31 patients who received Tabrecta as second- (n=30) or later-line (n=1) therapy in the METex14 skipping pretreated population, a confirmed ORR of 51.6% (95% CI, 33.1-69.8) was achieved, and the ORR across all 100 previously-treated patients, which included patients who received one or more prior lines of systemic therapy, was 44.0% (95% CI, 34.1-54.3).

The most common treatment-related adverse events (AEs) (incidence ≥20%) were peripheral oedema, nausea, fatigue, increased blood creatinine, vomiting, dyspnea, decreased appetite and back pain.

“As the leading cause of cancer-related deaths worldwide, lung cancer can be a devastating diagnosis for patients and their families,” said Marie-France Tschudin, President, Innovative Medicines International & Chief Commercial Officer, Novartis.

“With this new targeted therapy that treats a specific mutation driving cancer growth, we are delivering a much-needed treatment option and bringing hope to patients with this challenging disease.”

In the European Union, there are an estimated 291,000 patients with locally advanced or metastatic NSCLC.

METex14 skipping, a recognized oncogenic driver, occurs in approximately 3-4% of NSCLC cases.

About Tabrecta (capmatinib)
Tabrecta (capmatinib) is approved in several countries including the EU, US, Switzerland and Japan.

It is the number one prescribed targeted therapy for patients with advanced NSCLC with alterations leading to METex14 skipping globally.

Tabrecta is a kinase inhibitor that targets MET. Tabrecta was discovered by Incyte and licensed to Novartis in 2009.

Under the agreement, Incyte granted Novartis worldwide exclusive development and commercialization rights to capmatinib and certain back-up compounds in all indications.”

https://www.novartis.com/news/media-releases/novartis-receives-european-commission-approval-tabrecta-treatment-metex14-skipping-advanced-non-small-cell-lung-cancer

AZ’s NDA filing anticipated based on positive data from interim analysis

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AZ’s NDA filing anticipated based on positive data from interim analysis

FDA Announces Plans for Proposed Rule to Reduce Addictiveness of Tobacco Products

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FDA Announces Plans for Proposed Rule to Reduce Addictiveness of Tobacco Products

June 21, 2022: “The Biden-Harris Administration published plans for future potential regulatory actions that include the U.S. Food and Drug Administration’s plans to develop a proposed product standard that would establish a maximum nicotine level to reduce the addictiveness of cigarettes and certain other combusted tobacco products.

The goal of the potential rule would be to reduce youth use, addiction and death. 

Each year, 480,000 people die prematurely from a smoking-attributed disease, making tobacco use the leading cause of preventable disease and death in the United States.

Additionally, tobacco use costs nearly $300 billion a year in direct health care and lost productivity. 

While nicotine is not what makes smoking cigarettes so toxic, it’s the ingredient that makes it very hard to quit smoking.

Addiction to nicotine in combusted products is the main driver of sustained use of these products.

In fact, more than half of adult cigarette smokers make a serious quit attempt each year (quitting for at least a day), but most do not succeed due to the addictive nature of cigarettes.

Such a product standard, if proposed and then finalized after a thorough process, would make those products minimally- or non-addictive.

“Nicotine is powerfully addictive,” said FDA Commissioner Robert M. Califf, M.D.

“Making cigarettes and other combusted tobacco products minimally addictive or non-addictive would help save lives.

The U.S. Surgeon General has reported that 87 percent of adult smokers start smoking before age 18, and about two-thirds of adult daily smokers began smoking daily by 18 years of age.

Lowering nicotine levels to minimally addictive or non-addictive levels would decrease the likelihood that future generations of young people become addicted to cigarettes and help more currently addicted smokers to quit.” 

A paper published by the FDA in the New England Journal of MedicineExternal Link Disclaimer in 2018 projected that by year 2100, a potential nicotine product standard could result in more than 33 million people not becoming regular smokers, a smoking rate of only 1.4%, and more than 8 million fewer people dying from tobacco-related illnesses. The current smoking rate is 12.5%.

The Spring 2022 Unified Agenda of Regulatory and Deregulatory actions published today provides a report on the actions administrative agencies are considering issuing in the near and long term and currently lists several planned potential regulatory actions related to tobacco products; however, the dates in the Unified Agenda are not intended to be a precise estimate of when the work necessary to complete a proposed rule will be finished nor a final decision regarding whether a rule will be proposed.  

The FDA also remains focused on its regulatory oversight of e-cigarettes and other electronic nicotine delivery systems (ENDS).

Thus far, FDA has taken action on approximately 99% of the nearly 6.7 million products for which applications were received by the Sept. 9, 2020, deadline, including issuing marketing denial orders for more than 1 million ENDS products.

The FDA has also issued warning letters to ENDS product manufacturers and retailers who continue to sell products that are illegally on the market. 

The agency is focused on expeditiously completing the review of the remaining applications we received by the Sept. 9 deadline with a focus on those products with large market share.

In addition, the FDA has made a significant investment in a multimedia e-cigarette public education campaign aimed at the nearly 10.7 million youth aged 12-17 who have ever used e-cigarettes or are open to trying them highlighting information about the potential risks of e-cigarette use.”

https://www.fda.gov/news-events/press-announcements/fda-announces-plans-proposed-rule-reduce-addictiveness-cigarettes-and-other-combusted-tobacco

FDA Authorizes Moderna and Pfizer-BioNTech COVID-19 Vaccines for Children Down to 6 Months of Age

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FDA Authorizes Moderna and Pfizer-BioNTech COVID-19 Vaccines for Children Down to 6 Months of Age

June 17, 2022: “The U.S. FDA authorized emergency use of the Moderna COVID-19 Vaccine and the Pfizer-BioNTech COVID-19 Vaccine for the prevention of COVID-19 to include use in children down to 6 months of age. 

  • For the Moderna COVID-19 Vaccine, the FDA amended the emergency use authorization (EUA) to include use of the vaccine in individuals 6 months through 17 years of age. The vaccine had been authorized for use in adults 18 years of age and older.
  • For the Pfizer-BioNTech COVID-19 Vaccine, the FDA amended the EUA to include use of the vaccine in individuals 6 months through 4 years of age. The vaccine had been authorized for use in individuals 5 years of age and older. 

Key points:

  • The FDA’s evaluation and analysis of the safety, effectiveness and manufacturing data of these vaccines was rigorous and comprehensive, supporting the EUAs.
  • The agency determined that the known and potential benefits of the Moderna and Pfizer-BioNTech COVID-19 vaccines outweigh the known and potential risks in the pediatric populations authorized for use for each vaccine.
  • Prior to making the decision to authorize these vaccines for the respective pediatric populations, the FDA’s independent Vaccines and Related Biological Products Advisory Committee was consulted and voted in support of the authorizations. 

“Many parents, caregivers and clinicians have been waiting for a vaccine for younger children and this action will help protect those down to 6 months of age.  As we have seen with older age groups, we expect that the vaccines for younger children will provide protection from the most severe outcomes of COVID-19, such as hospitalization and death,” said FDA Commissioner Robert M. Califf, M.D. “Those trusted with the care of children can have confidence in the safety and effectiveness of these COVID-19 vaccines and can be assured that the agency was thorough in its evaluation of the data.” 

The Moderna COVID-19 Vaccine is administered as a primary series of two doses, one month apart, to individuals 6 months through 17 years of age. The vaccine is also authorized to provide a third primary series dose at least one month following the second dose for individuals in this age group who have been determined to have certain kinds of immunocompromise. 

The Pfizer-BioNTech COVID-19 Vaccine is administered as a primary series of three doses in which the initial two doses are administered three weeks apart followed by a third dose administered at least eight weeks after the second dose in individuals 6 months through 4 years of age. 

Information about each vaccine is available in the fact sheets for healthcare providers administering vaccine and the fact sheets for recipients and caregivers.

“As with all vaccines for any population, when authorizing COVID-19 vaccines intended for pediatric age groups, the FDA ensures that our evaluation and analysis of the data is rigorous and thorough,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. “In addition to making certain the data for these vaccines met FDA’s rigorous standards, the agency’s convening of an advisory committee was part of a transparent process to help the public have a clear understanding of the safety and effectiveness data supporting the authorization of these two vaccines for pediatric populations.” 

Evaluation of the Moderna COVID-19 Vaccine for Individuals 6 Months through 17 Years of Age

Effectiveness

The effectiveness and safety data evaluated and analyzed by the FDA for the Moderna COVID-19 Vaccine to support the EUA for these pediatric populations were generated in two ongoing, randomized, blinded, placebo-controlled clinical trials in the United States and Canada which enrolled infants, children and adolescents.

  • Children 6 months through 5 years of age: Immune responses of a subset of 230 children 6 through 23 months and a subset of 260 children 2 through 5 years of age who received a two-dose primary series of the Moderna COVID-19 Vaccine at 25 micrograms (mcg) of messenger RNA (mRNA) per dose were compared to immune responses among 290 adults 18 through 25 years who received two higher doses of the vaccine in a previous study which determined the vaccine to be effective in preventing COVID-19. In these FDA analyses, the immune response to the vaccine, of both age groups of children, was comparable to the immune response of the adults.  

    An analysis of cases of COVID-19 occurring at least 14 days after the second dose among approximately 5,400 children in this age group without evidence of prior infection with SARS-CoV-2 was conducted during the time period in which the omicron variant was the predominant circulating strain. In this analysis, among participants 6 through 23 months of age, 64% of whom had blinded follow-up for more than two months after the second dose, the vaccine was 50.6% effective in preventing COVID-19. Among participants 2 through 5 years of age, 72% of whom had blinded follow-up for more than two months after the second dose, the vaccine was 36.8% effective in preventing COVID-19.
  • Children 6 years through 11 years of age: Immune responses of a subset of 320 children in this age group who received a two-dose primary series of the Moderna COVID-19 Vaccine at 50 mcg of mRNA per dose were compared to immune responses among 295 adults 18 through 25 years who received two higher doses of the vaccine in a previous study which determined the vaccine to be effective in preventing COVID-19. In the FDA analysis, the immune response of the children to the vaccine was comparable to the immune response of the adults. An additional analysis pertaining to the occurrence of COVID-19 cases was determined not to be reliable due to the low number of COVID-19 cases that occurred in study participants. 
  • Adolescents 12 through 17 years of age: Immune responses of a subset of 340 adolescents in this age group who received a two-dose primary series of the Moderna COVID-19 Vaccine at 100 mcg of mRNA per dose were compared to immune responses among 296 adults 18 through 25 years who received two equivalent doses of the vaccine in a previous study which determined the vaccine to be effective in preventing COVID-19. In this analysis, the immune response of adolescents was comparable to the immune response of the older participants. 

    An analysis was also conducted of cases of COVID-19 occurring at least 14 days after the second dose among approximately 3,000 adolescents in this age group without evidence of prior infection with SARS-CoV-2, in which approximately 42% of participants had two or more months of blinded follow-up after the second dose. In this analysis, among participants 12 through 17 years of age, the vaccine was 93.3% effective in preventing COVID-19. The data for this analysis were obtained before the omicron variant became the predominant circulating strain.”
  • https://www.fda.gov/news-events/press-announcements/coronavirus-covid-19-update-fda-authorizes-moderna-and-pfizer-biontech-covid-19-vaccines-children

NICE to evaluate Genedrive® MT-RNR1 Test

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NICE to evaluate Genedrive® MT-RNR1 Test

June 16, 2022: “Genedrive plc, the near patient molecular diagnostics company, announces that the UK’s National Institute for Health and Clinical Excellence (‘NICE’) has started an evaluation of the Genedrive® MT-RNR1 test via their Diagnostics Assessment Programme (‘DAP’).

DAP evaluations are designed to provide robust recommendations on the use of new products, which is presented in the form of NICE guidance, and to promote rapid and consistent adoption of clinically innovative and cost-effective diagnostic technologies in the NHS.


An independent advisory committee considers the evidence provided, makes draft recommendations for public consultation and ultimately makesfinal recommendations for publication in NICE guidance.

The guidance produced is used by NHS commissioners, practitioners, healthcare operational managers and purchasing and procurement organisations.


genedrive’s assay is the world’s first rapid point of care test to screen infants in an urgent care setting for a genetic variant that will cause life-long hearing loss when carriers of the variant are given certain antibiotics.

Those that carry the variant can then be given alternative treatments following detection of the variant by the Genedrive® MTRNR1 test.


David Budd, CEO of genedrive plc, said: “We are grateful to NICE for their engagement and interest in our innovative technology and pleased that the Genedrive® MT-RNR1 test was selected for this programme following successful publication of the NICE Medtech innovation briefing (“MIB290”) in March.

The NICE guidance is an important element required to drive uptake and adoption of the test in the NHS by demonstrating the cost-saving efficiencies.

The application of Genedrive’s technology shows how a rapid, affordable, point-of-care test could impact patients’ treatment and quality of life.”

http://www.genedriveplc.com/press-releases/gdr_-nice(16.06.22).pdf

Jazz and Redx Announce Pan-RAF Inhibitor to Enter Clinical Development

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Jazz and Redx Announce Pan-RAF Inhibitor to Enter Clinical Development

June 15, 2022: “Jazz Pharmaceuticals and Redx announce the U.S. FDA has cleared the Investigational New Drug (IND) application for JZP815, a pan-RAF inhibitor for the treatment of solid tumors and hematologic malignancies that contain mutations in the MAPK pathway, enabling Jazz to proceed with initiating a clinical trial for JZP815.

As a result, a milestone payment of USD $5 million from Jazz payable to Redx has been triggered. 

The milestone payment was triggered under the Agreement in which Jazz acquired Redx’s pan-RAF inhibitor programme, announced on 10 July 2019.

Redx carried out development activities up to the completion of IND-enabling studies.

Today’s milestone is on top of USD $6.5 million already received under the collaboration and Redx remains entitled to development, regulatory and commercial milestone payments as well as incremental tiered royalties in mid-single digit percentages, based on any future net sales.

Preclinical data from this pan-RAF programme was recently presented at the American Association for Cancer Research (AACR) conference in March.

JZP815 is a precision pan-RAF inhibitor with a differentiated mechanism of action, and Jazz expects to assess its utility in treating several types of difficult-to-treat solid tumours where there remains significant unmet patient needs.

Jazz expects to advance JZP815 into a Phase 1 clinical programme and, when initiated, JZP815 will be the fifth compound discovered by Redx to enter the clinic.

Lisa Anson, Chief Executive Officer of Redx, commented: “I am delighted that the IND application for the pan-RAF inhibitor, JZP815, has been accepted.

When Jazz commence the clinical programme this will become the fifth drug candidate discovered by Redx to enter the clinic, further validating our world-class research and development capabilities.

We value the strong relationship we have built with Jazz Pharmaceuticals and look forward to continuing our work together.”

Rob Iannone, M.D., M.S.C.E., Executive Vice President, Global Head of Research and Development of Jazz Pharmaceuticals, commented: “We’re excited to advance JZP815, a precision pan-RAF inhibitor with a differentiated mechanism of action, into a clinical trial programme.

JZP815 may represent a significant advancement in the pan-RAF inhibitor class by not inducing paradoxical pathway activation that can stimulate the growth of certain cancers.

The JZP815 programme exemplifies our continued progress in expanding our early-stage oncology pipeline, and in developing therapies with the potential to address unmet patient need. 

Redx has an exceptional team of research and development scientists and together we have formed an outstanding collaboration, leveraging the strengths of both companies.”

Jazz and Redx also have a separate collaboration agreement to discover and develop drug candidates in the RAS-RAF-MAP kinase (MAPK) pathway, where Redx is again responsible for research and preclinical development activities up to IND application to the FDA.

About Pan-RAF inhibitors
Mutations leading to uncontrolled signalling in the RAS-RAF-MAPK pathway are seen in around one third of all cancers.

The Company’s pan-RAF inhibitor programme aims to overcome resistance mechanisms associated with clinically approved B-RAF selective drugs.

The RAF kinases A-RAF, B-RAF and C-RAF are an integral part of the RAS-RAF-MAPK pathway, with B-RAF mutations commonly seen in the clinic. Although most B-RAFV600E/K mutant skin cancers are initially sensitive to approved B-RAF selective drugs, treatment resistance often develops leading to disease progression.

Moreover, B-RAFV600E mutant colorectal cancers are surprisingly insensitive to these B-RAF selective drugs as single agents due to the compensatory functions of other RAF family members.

Importantly, B-RAF selective therapies fail to show clinical benefit against the more prevalent RAS-mutated tumours.

About JZP815
JZP815 is an investigational, pre-clinical stage pan-RAF kinase inhibitor that was discovered and developed using state-of-the-art screening methodologies and medicinal chemistry.

JZP815 targets specific components of the mitogen-activated protein kinase (MAPK) pathway that, when activated by oncogenic mutations, can be a frequent driver of human cancer.

JZP815 potently inhibits both monomer- and dimer-driven RAF signaling (e.g., RAS-induced), prevents paradoxical pathway activation induced by BRAF selective inhibition, and is active against class 1, class 2, and class 3 BRAF mutants, as well as BRAF fusions and CRAF mutants.

JZP815 is not currently approved for use anywhere in the world. JZP815 is part of Jazz’s growing early-stage R&D pipeline focused on solid tumours and targeted therapy.”

https://investor.jazzpharma.com/news-releases/news-release-details/jazz-pharmaceuticals-and-redx-announce-pan-raf-inhibitor-jzp815

Bristol Myers Squibb Announces Dividend

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Bristol Myers Squibb Announces Dividend

June 15, 2022: “Bristol Myers Squibb announced that its Board of Directors has declared a quarterly dividend of fifty-four cents ($0.54) per share on the $.10 par value common stock of the company.

The dividend is payable on August 1, 2022 to stockholders of record at the close of business on July 1, 2022.

In addition, the Board of Directors has declared a quarterly dividend of fifty cents ($0.50) per share on the company’s $2.00 convertible preferred stock, payable September 1, 2022, to stockholders of record at the close of business on August 9, 2022.

About Bristol Myers Squibb Company

Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop, and deliver innovative medicines that help patients prevail over serious diseases.”

https://news.bms.com/news/corporate-financial/2022/Bristol-Myers-Squibb-Announces-Dividend-06f4e09a1/default.aspx

GSK to present bepirovirsen in chronic hepatitis B at International Liver Congress 2022

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GSK to present bepirovirsen in chronic hepatitis B at International Liver Congress 2022

June 15, 2022: “GSK plc will present 12 abstracts at the European Association for the Study of the Liver’s International Liver Congress 2022, taking place on June 22-26 in London.

GSK’s presence will focus on two novel investigational specialty medicines, bepirovirsen, an antisense oligonucleotide for chronic hepatitis B (CHB), and linerixibat, an ileal bile acid transporter (IBAT) inhibitor for cholestatic pruritus in primary biliary cholangitis (PBC).

Chris Corsico, SVP, Development, GSK said: “New data being shared at this year’s International Liver Congress support our ambition to deliver novel medicines for patients with significant unmet medical need, such as those who suffer from chronic hepatitis B and cholestatic pruritus in primary biliary cholangitis.

We look forward to engaging with the global hepatology community to share the exciting progress we have made for these patients.”

Updates from the bepirovirsen clinical trial programme

New interim analysis data to be shared from the randomised B-Clear phase IIb trial evaluating the efficacy and safety of bepirovirsen in patients with chronic hepatitis B virus infection on and off stable nucleos(t)ide analogue therapy.

Data from this trial will be presented as part of the Late Breaker oral presentation session on June 25 (ILC oral # LB004A, LB004B).

Additional presentations from the bepirovirsen development programme include:

  • Preclinical evidence that bepirovirsen harbours intrinsic immunostimulatory activity via Toll-like receptor 8 (TLR8), correlating with clinical efficacy from the study (ILC abstract #3635/ SAT439)
  • Mechanistic pharmacokinetic and pharmacodynamic modelling of the simultaneous effects of bepirovirsen on hepatitis B surface antigen (HBsAg) and alanine transaminase (ALT) changes in CHB (ILC abstract #3592/ SAT441).

Data from these trials reflect GSK’s ambition to contribute to the development of a functional cure for people living with CHB and reduce the disease burden for patients.

Updates from the linerixibat clinical trial programme

New analysis to be presented from the GLIMMER phase II trial comparing the health-related quality of life of patients with cholestatic pruritus in PBC to several more common diseases, using EQ-5D, a standardised measure of health utility.

The data from this analysis demonstrate that severe cholestatic pruritus in PBC has a negative impact on health utility that is similar to severe Parkinson’s disease (ILC abstract #1916/ THU470).

Full list of GSK’s presentations at the European Association for the Study of the Liver’s International Liver Congress 2022:

Bepirovirsen

Abstract NamePresenterPresentation details
Efficacy and safety of bepirovirsen in patients with chronic hepatitis B virus infection: interim results from the randomised phase 2b B-Clear study Man-Fung Yuen Oral (LB004A, LB004B)
Efficacy and safety of bepirovirsen in patients with chronic hepatitis B virus infection not on stable nucleos(t)ide analogue therapy: interim results from the randomised phase 2b B-Clear study Seng-Gee LimPoster (3595/ SAT452)
Efficacy and safety of bepirovirsen in patients with chronic hepatitis B virus infection on stable nucleos(t)ide analogue therapy: interim results from the randomised phase 2b B-Clear study Man-Fung Yuen Poster (3629/ SAT453)
Evaluation Of Quantitative HBsAg Levels In Chronic Hepatitis B – A Targeted Literature Review Vera Gielen Poster (1850/ SAT405)
Characterisation of baseline complement values in patients with chronic hepatitis B virus infection in the phase IIb B-Clear study Jennifer Cremer  Poster (1994/ THU390)
Characteristics and renal function in patients with chronic hepatitis B virus infection: baseline data from the phase 2b B-Clear study Seng-Gee Lim Poster (1879/ THU389)
Distribution of patients by guideline-defined disease phase and/or grey zones in B-Clear, an international multi-centre clinical trial Seng-Gee Lim  Poster (2127/ THU393)
Bepirovirsen, antisense oligonucleotide (ASO) against hepatitis B virus (HBV), harbors intrinsic immunostimulatory activity via Toll-like receptor 8 (TLR8) preclinically, correlating with clinical efficacy from the Phase 2a study Shihyun You Poster (3635/ SAT439)
Mechanistic Pharmacokinetics/Pharmacodynamics modelling of the simultaneous effects of bepirovirsen on Hepatitis B surface antigen (HBsAg) and alanine transaminase (ALT) changes in Chronic Hepatitis B (CHB) patients: Analysis of Phase 2b study to inform Phase 3 decision-making Ahmed Nader Poster (3592/ SAT441)
Combination treatment of a PAPD5/7 inhibitor with an antisense oligonucleotide bepirovirsen with concurrent dosing shows additive HBsAg decreases in the AAV-HBV mouse modelMartin LeiversPoster (3590/ SAT451)

Linerixibat

Abstract NamePresenterPresentation details
More than just an itch: Impact of cholestatic pruritus in primary biliary cholangitis (PBC) on health-related quality of life (HRQoL) Helen Smith Poster (1916/ THU470)
Linerixibat dose-response analysis of C4 concentrations as a quantitative approach to predict gastrointestinal tolerability Fernando CarreñoPoster (2056/ THU472)
Investigation of linerixibat 40mg BID for cholestatic pruritus of primary biliary cholangitis (PBC); further data from the Phase 2b GLIMMER study to support the Phase 3 GLISTEN study James Fettiplace Poster (3606/ THU485)

About chronic hepatitis B

Hepatitis B is a viral infection of the liver, caused by the hepatitis B virus (HBV), it can cause both acute and chronic liver disease.

Chronic hepatitis B (CHB) is a long-lasting infection and occurs when the body’s immune system is unable to fight off the virus and it persists in the blood and liver.

It is estimated that there are 296 million people globally living with CHB.1 Even when treated, CHB can progress to liver complications including cirrhosis and liver cancer, which results in nearly 900,000 deaths per year.

Viral suppression is the current goal for treatment of CHB. However, viral replicative activity may return upon cessation of treatment requiring lifelong therapy to prevent viral rebound.

The concept of functional cure of CHB aims to eliminate the virus from circulating in the blood and prevent any disease activity in the liver.

As only a limited number of patients currently treated for CHB achieve HBsAg loss, considered the hallmark for achieving functional cure, development of therapeutic approaches to reach functional cure are needed.”

https://www.gsk.com/en-gb/media/press-releases/gsk-to-present-new-data-from-the-b-clear-phase-iib-trial-for-bepirovirsen-in-chronic-hepatitis-b-at-the-international-liver-congress-2022/

FDA Approves First Systemic Treatment for Alopecia Areata

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FDA Approves First Systemic Treatment for Alopecia Areata

June 13, 2022: “The U.S. FDA approved Olumiant (baricitinib) oral tablets to treat adult patients with severe alopecia areata, a disorder that often appears as patchy baldness and affects more than 300,000 people in the U.S. each year.

Today’s action marks the first FDA approval of a systemic treatment (i.e. treats the entire body rather than a specific location) for alopecia areata.

“Access to safe and effective treatment options is crucial for the significant number of Americans affected by severe alopecia,” said Kendall Marcus, M.D., director of the Division of Dermatology and Dentistry in the FDA’s Center for Drug Evaluation and Research.

“Today’s approval will help fulfill a significant unmet need for patients with severe alopecia areata.”

Alopecia areata, commonly referred to as just alopecia, is an autoimmune disorder in which the body attacks its own hair follicles, causing hair to fall out, often in clumps.

Olumiant is a Janus kinase (JAK) inhibitor which blocks the activity of one or more of a specific family of enzymes, interfering with the pathway that leads to inflammation.

The efficacy and safety of Olumiant in alopecia areata was studied in two randomized, double-blind, placebo-controlled trial (Trial AA-1 and Trial AA-2) with patients who had at least 50% scalp hair loss as measured by the Severity of Alopecia Tool for more than six months.

Patients in these trials received either a placebo, 2 milligrams of Olumiant, or 4 milligrams of Olumiant every day.

The primary measurement of efficacy for both trials was the proportion of patients who achieved at least 80% scalp hair coverage at week 36.

In Trial AA-1, 22% of the 184 patients who received 2 milligrams of Olumiant and 35% of the 281 patients who received 4 milligrams of Olumiant achieved adequate scalp hair coverage, compared to 5% of the 189 patients who received a placebo.

In Trial AA-2, 17% of the 156 patients who received 2 milligrams of Olumiant and 32% of the 234 patients who received 4 milligrams of Olumiant achieved adequate scalp hair coverage, compared to 3% of the 156 patients who received a placebo.  

The most common side effects associated with Olumiant include: upper respiratory tract infections, headache, acne, high cholesterol (hyperlipidemia), increase of an enzyme called creatinine phosphokinase,  urinary tract infection,  liver enzyme elevations, inflammation of hair follicles (folliculitis), fatigue, lower respiratory tract infections, nausea, genital yeast infections (Candida infections), anemia, low number of certain types of white blood cells (neutropenia), abdominal pain, shingles (herpes zoster) and weight increase.  

Olumiant is not recommended for use in combination with other JAK inhibitors, biologic immunomodulators, cyclosporine or other potent immunosuppressants.

Olumiant comes with warnings and precautions including recommending close monitoring for the development of signs and symptoms of infection during and after treatment; evaluating patients for active tuberculosis infection and testing for latent tuberculosis prior to treatment with Olumiant; and the potential for viral reactivation.

In addition, other warnings and precautions include hypersensitivity (allergic reactions), gastrointestinal perforations (tears in stomach or intestine), and laboratory abnormalities including low white and red blood cell counts, liver enzyme elevations and lipid elevations.

Olumiant comes with a boxed warning for serious infections, mortality, malignancy, major adverse cardiovascular events and thrombosis.

Olumiant received priority review and breakthrough therapy designations for this indication.

Olumiant was originally approved in 2018.

It is approved as a treatment for certain adult patients with moderately to severely active rheumatoid arthritis.

Olumiant is also approved for the treatment of COVID-19 in certain hospitalized adults.  

The FDA granted the approval of Olumiant to Eli Lilly and Company.”

https://www.fda.gov/news-events/press-announcements/fda-approves-first-systemic-treatment-alopecia-areata
 

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