Pfizer continues to make headlines with its COVID-19 initiatives, but the company prides itself in all of its scientific achievements and the financial muscle to back them up.
By Christiane Truelove • [email protected]
235 East 42nd Street NY, NY 10017
(All figures are in millions of dollars, except EPS)
Net income $9,616
Diluted EPS $1.71
R&D expense $9,405
Net income $10,440
Diluted EPS $1.84
R&D expense $4,473
(All sales are in millions of dollars)
Prevnar 13/Prevenar 13 $5,850
Premarin family $680
Prevnar 13/Prevenar 13 $2,524
Premarin family $271
Product Sales Notes:
BNT162b2 = Direct sales and alliance revenue
Eliquis = Direct sales and alliance revenue
Enbrel = Sales outside U.S. and Canada
Xtandi = Alliance revenue
Outcomes Creativity Index Score: 28
Manny Awards — N/A
Cannes Lions — 19
Clio Health — N/A
Creative Floor Awards — 8
MM+M Awards — N/A
One Show — 1
2020 was a year like none other in Pfizer’s history – defined by bold decisions, even bolder actions and incredible results,” says Pfizer CEO Albert Bourla.
After spinning off the Upjohn business and combining it with Mylan to form Viatris in November 2020, Pfizer became 20 percent smaller “but more focused than ever on delivering first-in-class science for the benefit of patients,” Bourla says.
“To the outside world, it may have appeared that COVID-19 was the only thing we were working on in 2020, but that could not be further from the truth. While we invested significant time, resources and brainpower to find medical solutions to the pandemic, tens of thousands of Pfizer colleagues continued to advance equally important work across all of our therapeutic areas – recognizing that the needs of those suffering from other diseases were no less urgent.”
Bourla says the completion of the Upjohn-Mylan transaction was the culmination of a bold, decade-long transformation of Pfizer from a large, diversified enterprise to a smaller, science-driven, innovative biopharma company.
“The new Pfizer is all about two things: science and patients,” Bourla says. “By uniting transformational technology and cutting-edge science, we are pioneering biopharmaceutical innovations to do more than just treat difficult diseases – we want to cure and prevent them. Our pipeline currently includes 95 potential new therapies or indications. That’s 95 potential opportunities to change the lives of patients around the world.
In 2021, Pfizer launched a new corporate brand identity, with a new logo and the slogan: Science Will Win.
The company also opened a merchandise store on its website, with sales of branded clothing (such as T-shirts and rain jackets) and accessories (including mugs, water bottles, canvas cooler bags, and even golf balls) going to charity.
“Our new emblem is a digital-first expression of our commitment to the transformative power of science,” Bourla says. “It’s a dynamic reflection of our purpose: Breakthroughs that change patients’ lives. And it’s a clear signal that the new Pfizer is about daring more courageously, inquiring more deeply and advocating more passionately to make what was once unimaginable, reality.”
Performance & Outlook
Despite the challenging business environment created by the pandemic and the significant amount of resources the company devoted to developing a safe and effective COVID-19 vaccine, Pfizer generated 8 percent operational revenue growth for the year from its biopharmaceutical product portfolio, excluding the results of the divested Upjohn business, the revenue impact from Consumer Healthcare, and $154 million of sales from the Pfizer and BioNTech COVID-19 vaccine. The 8 percent operational growth included a negative 2 percent impact due to the slowdown in macroeconomic and healthcare activity resulting from the pandemic.
The company generated revenue of $41.91 billion in 2020, growth of 2 percent from the previous year. Net income was $9.62 billion compared with $16.27 billion in 2019, and diluted earnings per share were $1.71 compared with $2.87.
Pfizer’s lead product in sales in 2020 was the vaccine Prevnar 13, which generated $5.85 billion, up 1 percent versus the previous year. Sales in the first half of 2021 were $2.52 billion, 2 percent less than in first-half 2020.
Coming in at No. 2 was the HR-positive and HER2-negative breast cancer drug Ibrance, with $5.39 billion, 9 percent more than in 2019. In first-half 2021, Ibrance sales totaled $2.66 billion, 2 percent more than in the same period last year.
The anticoagulant Eliquis accounted for direct sales and alliance revenue totaling $4.95 billion, 18 percent more than in 2019. The first-half 2021 total reached $3.12 billion, 19 percent more than in first-half 2020.
Pfizer’s rheumatoid arthritis, psoriatic arthritis, and ulcerative colitis treatment Xeljanz produced 2020 sales of $2.44 billion, growing 9 percent from the previous year. First-half 2021 sales grew 2 percent over first-half 2020, to $1.12 billion.
The rheumatoid and psoriatic arthritis drug Enbrel generated $1.35 billion in 202o sales outside of the United States and Canada compared to $1.7 billion in 2019. Biosimilars continued to erode sales in the first half of 2021, at $605 million compared with $684 million in the same period of 2020.
With sales of $1.29 billion in 2021 compared with $473 million in 2019, Vyndaqel/Vyndamax is one of Pfizer’s best-selling drugs. The treatment for transthyretin amyloid cardiomyopathy generated $953 million in first-half 2021 compared with $508 million in the same period last year.
The prostate cancer treatment Xtandi posted 2020 alliance revenue of $1.02 billion, 22 percent more than in 2019. Alliance revenue in the first six months of 2021 grew 20 percent to $570 million.
Sales during 2020 for the anti-smoking drug Chantix/Champix decreased 17 percent to $919 million. Sales continued to decline in the first half of this year, by 22 percent to $401 million.
The oncology medicine Sutent declined in sales during 2020 to $819 million from $936 million in the previous year. First-half 2021 sales were 8 percent less than in first-half 2020, at $394 million.
The kidney cancer drug Inlyta’s sales increased to $787 million from $477 million in 2019. Sales in the first half of this year were also higher than in the same period of 2020, at $486 million compared with $364 million.
The Premarin family of hormone replacement therapies generated $680 million in 2020, down from $734 million during 2019. First-half 2021 sales were 11 percent less than in first-half 2020, at $271 million.
Sulperazon 2020 sales amounted to $618 million compared with $684 million during the previous year. The broad-spectrum antibiotic produced 7 percent operational growth in the first half of 2021 compared with the same period last year, totaling $334 million.
Pfizer’s non-small cell lung cancer drug Xalkori generated $544 million during 2020 versus $530 million in 2019. First-half 2021 sales declined to $255 million, 15 percent less operationally than in first-half 2020.
For the first half of 2021, Pfizer’s revenue was $33.56 billion compared with $19.95 billion in the same period last year. Net income was $10.44 billion compared with $6.84 billion in first-half 2020, and diluted earnings per share were $1.84 compared with $1.22.
In commenting on the company’s performance in the first half of 2021, Bourla says the second quarter was remarkable in a number of ways. “Most visibly, the speed and efficiency of our efforts with BioNTech to help vaccinate the world against COVID-19 have been unprecedented, with now more than a billion doses of BNT162b2 having been delivered globally. In addition, we are equally proud of the second-quarter performance of our business excluding BNT162b2, which posted 10 percent operational revenue growth.”
Bourla adds, “Looking forward, we remain highly confident in our ability to achieve at least a 6 percent compound annual growth rate through 2025 and intend to build upon our recent successes by continuing to follow the science, trust in our people and remain focused on delivering breakthroughs for the patients we serve.”
Pfizer & COVID-19
Pfizer collaborated with the German biotechnology company BioNTech SE to jointly develop a COVID-19 vaccine using BioNTech’s messenger RNA (mRNA) vaccine program while simultaneously making self-funded investments to scale up Pfizer’s manufacturing capacity and distribution infrastructure. The goal was to bring a potential vaccine to the world faster than ever, but with Pfizer’s same scientific rigor and commitment to quality and safety. “In less than a year, we made the impossible possible, delivering in record time a breakthough COVID-19 vaccine granted a conditional marketing authorization, Emergency Use Authorization or temporary authorization in more than 50 countries worldwide. And we did it, without ever losing sight of the integrity, quality or safety of our work.”
Bourla maintains that “Our ability to move at such extraordinary speed – while always maintaining our focus on quality and safety – was the first powerful display of what the new Pfizer is capable of.”
“While we never imagined a pandemic of this magnitude, every action we have taken over the past several years has been to transform Pfizer into an agile, scientific powerhouse capable of addressing the world’s most devastating diseases.”
FDA gave full approval to the vaccine, now known as Comirnaty, in August. It is the first COVID vaccine to be given full approval by FDA. The vaccine has been available in the United States under Emergency Use Authorization (EUA) since December 11, 2020 (as the Pfizer-BioNTech COVID-19 Vaccine). The EUA permitted essential rollout of vaccine doses across the United States.
For FDA approval, Pfizer and BioNTech submitted a comprehensive data package that included longer-term follow-up data from the Phase III trial, where the vaccine’s high efficacy and favorable safety profile were observed up to six months after the second dose. The BLA submission package also included the manufacturing and facilities data required for licensure. Pfizer and BioNTech completed submission of the BLA in May 2021, and the BLA was granted Priority Review in July 2021.
“Based on the longer-term follow-up data that we submitted, today’s decision by the FDA affirms the efficacy and safety profile of our vaccine at a time when it is urgently needed,” Bourla says. “About 60 percent of eligible Americans are fully vaccinated, and infection, hospitalization and death rates continue to rise rapidly among unvaccinated populations across the country. I am hopeful this approval will help increase confidence in our vaccine, as vaccination remains the best tool we have to help protect lives and achieve herd immunity.
“Hundreds of millions of doses of our vaccine already have been administered in the U.S. since December 2020, and we look forward to continuing to work with the U.S. government to reach more Americans now that we have FDA approval.”
According to Ugur Sahin, M.D., CEO and co-founder of BioNTech, at the time of the full approval, Pfizer and BioNTech had shipped more than one billion doses worldwide.
The companies are continuing to explore innovative plans to increase the number of doses that they are able to produce globally by the end of 2021.
“Based on the updated six-dose labeling and subject to continuous process improvements, expansion at current facilities and adding new suppliers and contract manufacturers, we now believe we can potentially manufacture at least two billion doses in total by year’s end,” Bourla says.
Additionally, Pfizer and BioNTech have engaged governments and global health organizations around the world, and have supply agreements, or are in talks with, more than 100 countries and supranational organizations for the supply of the vaccine. Doses have also been allocated for supply to low-income countries at a not-for-profit price.
During September, Pfizer and BioNTech expanded their agreement with the U.S. government by providing an additional 500 million doses of the companies’ COVID-19 vaccine at a not-for-profit price for donation to low-income and lower-middle-income countries and the organizations that support them. This expanded agreement brings the total number of doses to be supplied to the U.S. government for donation to these countries to one billion.
While the full approval was for people 16 years and older, Pfizer and BioNTech continue to run trials for other age groups as well as investigate the need for boosters.
On Sept. 22, 2021, the FDA authorized for emergency use a booster dose of the Pfizer-BioNTech vaccine for individuals 65 years of age and older, individuals 18 through 64 years of age at high risk of severe COVID-19, and individuals 18 through 64 years of age whose frequent institutional or occupational exposure to SARS-CoV-2 puts them at high risk of serious complications of COVID-19 including severe COVID-19.
According to the companies, the booster dose is to be administered at least six months after completion of the primary series, and is the same formulation and dosage strength as the doses in the primary series.
“This first FDA authorization of a COVID-19 vaccine booster is a critical milestone in the ongoing fight against this disease,” Bourla stated. “Over the last year and a half, we have aimed to stay vigilant as the pandemic has evolved – including evaluating the impact of a booster dose. We believe boosters have an important role to play in addressing the continued threat of this disease, alongside efforts to increase global access and uptake among the unvaccinated. Today’s FDA action is an important step in helping the most vulnerable among us remain protected from COVID-19.”
The FDA based this EUA on the totality of scientific evidence shared by Pfizer and BioNTech and reviewed by the FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC), including data from the companies’ clinical program assessing the safety, tolerability and immunogenicity of a booster dose of the COVID-19 vaccine.
A booster dose of the vaccine elicited significantly higher neutralizing antibody titers against the initial SARS-CoV-2 virus (wild type), as well as the Beta and Delta variants, versus the levels observed after the two-dose primary series.
Research has shown that the reactogenicity profile within seven days after the booster dose was typically mild to moderate, and the frequency of reactions was similar to or lower than after dose two.
During August, Pfizer and BioNTech announced that they plan to seek licensure of a booster dose of Comirnaty in individuals 16 years of age and older via a supplemental BLA. The companies also intend to submit a supplemental BLA to support potential full FDA approval of the vaccine in individuals 12 through 15 years of age, once the required data out to six months after the second vaccine dose are available. In the meantime, the vaccine remains available to 12- to 15-year-olds under the Emergency Use Authorization (EUA) granted by the FDA on May 10, 2021.
Conditional Marketing Authorization (CMA) for Comirnaty in the European Union was expanded during May 2021 to include individuals 12 to 15 years of age. This follows the European Medicines Agency’s Committee for Medicinal Products for Human Use positive opinion to authorize Comirnaty for this age group. The extended indication for the CMA for the vaccine is valid in all 27 EU member states.
Comirnaty represents the first COVID-19 vaccine to receive EU authorization and is the first to have its CMA extended to the adolescent population.
For individuals at least 12 years of age who have undergone solid organ transplantation, or who are diagnosed with conditions that are considered to have an equivalent level of immunocompromise, a third dose of the vaccine also remains available under EUA following an amendment by the FDA on August 12.
In September, Pfizer submitted initial data to FDA from its pivotal trial of children ages 5 to under 12. Positive results from the trial were announced earlier in the month. In the trial, which included 2,268 participants 5 to less than 12 years of age, the vaccine demonstrated a favorable safety profile and elicited robust neutralizing antibody responses using a two-dose regimen of 10 µg doses. Company executives say these results, the first from a pivotal trial of any COVID-19 vaccine in this age group, were comparable to those recorded in a previous Pfizer-BioNTech study in people 16 to 25 years of age, who were immunized with 30 µg doses.
Management says topline immunogenicity and safety readouts for the other two age cohorts from the trial – children 2 to less than 5 years of age and children 6 months to less than 2 years of age – are expected as soon as the fourth quarter of this year.
In August, Pfizer and BioNTech signed a letter of intent with Eurofarma Laboratórios SA, a Brazilian biopharmaceutical company, to manufacture Comirnaty for distribution within Latin America.
Eurofarma will perform manufacturing activities within Pfizer’s and BioNTech’s global COVID-19 vaccine supply chain and manufacturing network, which now spans four continents and includes more than 20 manufacturing facilities. To facilitate Eurofarma’s involvement in the process, technical transfer, on-site development, and equipment installation activities began immediately. Per the agreement, Eurofarma will obtain drug product from facilities in the United States, and manufacturing of finished doses will commence in 2022.
At full operational capacity, the annual production is expected to exceed 100 million finished doses annually. All doses will exclusively be distributed within Latin America.
“Everyone – regardless of financial condition, race, religion or geography – deserves access to lifesaving COVID-19 vaccines,” Bourla says. “Our new collaboration with Eurofarma expands our global supply chain network to another region – helping us continue to provide fair and equitable access to our COVID-19 vaccine. We will continue to explore and pursue opportunities such as this to help ensure that vaccines are available to all who are in need.”
Beyond the vaccine, Pfizer has also been investigating other ways of fighting SARS-CoV-2. In September, the company launched a global Phase II study, EPIC-PEP (Evaluation of Protease Inhibition for COVID-19 in Post-Exposure Prophylaxis), to evaluate the novel protease inhibitor PF-07321332, co-administered with a low dose of ritonavir, for prevention of illness in adults living in the same household as someone with COVID-19.
“With the continued impact of COVID-19 around the world, we believe that tackling the virus will require effective treatments for people who contract, or have been exposed to, the virus, complementing the impact that vaccines have had in helping quell infections,” says Mikael Dolsten, M.D., Ph.D., chief scientific officer and president, Worldwide Research, Development and Medical of Pfizer. “If successful, we believe this therapy could help stop the virus early – before it has had a chance to replicate extensively – potentially preventing symptomatic disease in those who have been exposed and inhibiting the onset of infection in others.”
Dr. Dolsten stated, “Given the continued emergence and evolution of SARS-CoV-2 variants and their immense impact, we continue to work diligently to develop and study new ways that our investigational oral antiviral candidate could potentially lower the impact of COVID-19, not only on patients’ lives, but also the lives of their families and household members.”
The Phase II/III EPIC-PEP trial is a randomized, double-blind, placebo-controlled study and will enroll up to 2,660 healthy adult participants aged 18 and older. Participants will be randomly assigned (1:1:1) to receive PF-07321332/ritonavir or placebo orally twice daily for 5 or 10 days. The primary objective will assess safety and efficacy for the prevention of confirmed SARS-CoV-2 infection and its symptoms through Day 14. PF-07321332 is an oral antiviral SARS-CoV-2-3CL protease inhibitor, which has an encouraging pre-clinical profile, including potent in vitro antiviral SARS-CoV-2 and broad coronavirus activity. Results from the Phase 1 clinical trial demonstrated that PF-07321332 was safe and well tolerated.
In addition to this study, the global EPIC program consists of multiple ongoing clinical trials, including one in SARS-CoV-2 infected patients who are at high risk of severe illness (including hospitalization or death), which began in July 2021, and another in infected patients who are at standard risk (i.e., do not have risk factors for severe illness), which began in August 2021.
Other R&D Progress
Company executives say Pfizer’s pipeline has been driven by “incredible” improvements in clinical success rates.
“For example, our Phase II success rate on a five-year rolling average more than tripled from 15 percent five years ago to 52 percent as of year-end 2020 – which is almost double the 2019 industry benchmark of 29 percent,” Bourla says. “Significantly, most of these successes are either first-in-class assets or innovations built on established mechanisms with novel scientific designs.
“In addition, our end-to-end success rate more than quadrupled over the same time frame from 5 percent to 21 percent – almost triple the 2019 industry benchmark of 8 percent. We believe these metrics demonstrate that through our science, we are selecting assets to move through the R&D process that have the best chance of benefiting patients.”
In September, FDA accepted for review a supplemental New Drug Application (sNDA) for Myfembree (relugolix 40 mg, estradiol 1 mg, and norethindrone acetate 0.5 mg) for the management of moderate to severe pain associated with endometriosis.
The FDA set a target action date of May 6, 2022 for this sNDA under the Prescription Drug User Fee Act (PDUFA).
The sNDA submission in endometriosis is supported by results from the Phase III SPIRIT program, which included two multinational, replicate pivotal clinical studies (SPIRIT 1 and SPIRIT 2) in over 1,200 women with pain associated with endometriosis for 24 weeks, and an open-label extension study for eligible women who completed either SPIRIT 1 or SPIRIT 2 through one year.
“The submission of the sNDA for Myfembree to treat endometriosis pain reflects our commitment to addressing areas of significant unmet need in women’s health,” says James Rusnak, M.D., Ph.D., senior VP, chief development officer, Internal Medicine and Hospital, Global Product Development at Pfizer. “We look forward to potentially bringing this important new treatment option to women with endometriosis.”
In the United States, Myfembree is available for the management of heavy menstrual bleeding associated with uterine fibroids in premenopausal women, with a treatment duration of up to 24 months. The FDA approved Myfembree for this indication on May 26, 2021, based on data from the Phase III LIBERTY program. Myovant Inc. and Pfizer are jointly developing and commercializing Myfembree in the United States.
In another September pipeline advancement for Pfizer, the UK Medicines and Healthcare products Regulatory Agency (MHRA) granted Great Britain marketing authorization for Cibinqo (abrocitinib), an oral, once-daily, Janus kinase 1 (JAK1) inhibitor, for the treatment of moderate-to-severe atopic dermatitis (AD) in adults and adolescents aged 12 years and over, who are candidates for systemic therapy. Abrocitinib is licensed in Great Britain in recommended doses of 100mg and 200mg. This is the first marketing authorization worldwide for this treatment.
“We welcome the MHRA’s authorization of abrocitinib to treat people with moderate to severe atopic dermatitis,” says Angela Hwang, group president, Pfizer Biopharmaceuticals Group. “This is an important development for people in Great Britain who have moderate to severe disease and need innovative treatment options. Following marketing authorization, our priority now is to work with NICE and the Scottish Medicines Consortium (SMC) to ensure routine access so that patients with moderate to severe AD can benefit from this important treatment.”
Last year, abrocitinib received a Promising Innovative Medicine (PIM) designation from the MHRA. In January of this year, abrocitinib was granted a positive scientific opinion for an Early Access to Medicines Scheme (EAMS) from the MHRA for people with severe atopic dermatitis requiring treatment with systemic therapy and who have had inadequate response or have lost response to licensed systemic therapies, or who are ineligible or intolerant of licensed systemic therapies. This enabled healthcare professionals to prescribe the treatment prior to marketing authorization, based on clinical factors for patients with a clear unmet need.
Regulatory applications for abrocitinib have been submitted to countries around the world for review, including the United States, Australia, Japan, and the European Union.
The FDA notified Pfizer In July that the PDUFA goal dates would not be met for the New Drug Application for abrocitinib for the treatment of adults and adolescents with moderate to severe atopic dermatitis and the supplemental New Drug Application for Xeljanz/Xeljanz XR (tofacitinib) for treating adults with active ankylosing spondylitis. The U.S. health regulator cited its ongoing review of Pfizer’s post-marketing safety study, ORAL Surveillance, assessing tofacitinib in rheumatoid arthritis patients, as a factor for the extensions.
Also in September, Pfizer announced the initiation of RENOIR (RSV vaccine Efficacy study iNOlder adults Immunized against RSV disease), a Phase III clinical trial evaluating the efficacy, immunogenicity and safety of a single dose of its respiratory syncytial virus (RSV) bivalent prefusion F subunit investigational vaccine candidate (RSVpreF) in adults ages 60 years or older.
“RSV is a significant cause of severe respiratory disease in older adults, and it can cause disability and death. There is an important unmet medical need for an effective vaccine that can help protect older adults against this highly-contagious disease,” says Kathrin U. Jansen, Ph.D., senior VP and head of Vaccine Research & Development at Pfizer. “The start of this Phase III study is an important step forward towards our goal of comprehensive immunization against RSV disease, which includes developing a potential first vaccine to help prevent RSV disease in adults as well as the ongoing efforts to help protect infants through maternal immunization, subject to regulatory approval of the candidate vaccine.”
The Phase III RENOIR trial of RSVpreF is a global, randomized, double-blind, placebo-controlled study that expects to enroll approximately 30,000 participants 60 years and older. The primary objectives of the study will assess safety and efficacy for the prevention of moderate to severe lower respiratory tract illness (msLRTI-RSV) during the first RSV season.
Valneva SE and Pfizer during September announced further positive Phase II results, including booster response, for the Lyme disease vaccine candidate VLA15. The investigational multivalent protein subunit vaccine uses an established mechanism of action for a Lyme disease vaccine that targets the outer surface protein A (OspA) of Borrelia burgdorferi, the bacteria that results in Lyme disease, and covers the six OspA serotypes prevalent in North America and Europe. OspA is one of the most dominant surface proteins expressed by the bacteria when present in a tick. VLA15 has shown strong immunogenicity and safety data in pre- clinical and clinical studies so far. The clinical development program was granted Fast Track designation by the U.S. FDA during July 2017.
The Phase II trial, VLA15-202, is testing the immunogenicity and safety of VLA15 in a Month 0-2-6 vaccination schedule. The clinical trial enrolled 246 healthy adults 18 to 65 years of age in the United States. As announced during October 2020, the trial met its primary endpoint of showing that VLA15 was immunogenic across all dose groups tested and elicited high antibody responses across all serotypes (ST1 – ST6) at one month after completion of the primary vaccination series. Continued assessment at Month 18 demonstrated that antibody titers decreased thereafter across all groups, remaining above baseline but confirming the need for a booster strategy.
According to Valneva and Pfizer, VLA15 was safe and well-tolerated across all doses and age groups tested. VLA15 represents the only active Lyme disease vaccine candidate in clinical development.
Pfizer and OPKO Health Inc. announced in September that the FDA extended the review period for the Biologics License Application (BLA) for somatrogon. The once-weekly long-acting recombinant human growth hormone is intended for the treatment of growth hormone deficiency (GHD) in pediatric patients. The PDUFA goal date was extended by three months to January 2022, as a result of Pfizer’s filing of additional data to the original BLA.
Somatrogon has been granted Orphan Drug designation in the United States and the EU for treating children and adults with growth hormone deficiency.
In August, FDA approved Ticovac (tick-borne encephalitis (TBE) vaccine) for active immunization to prevent TBE in individuals 1 year of age and older. Ticovac is the only FDA-approved vaccine to help protect U.S. adults and children against the TBE virus when visiting or living in TBE endemic areas. Following the approval, the U.S. Centers for Disease Control and Prevention’s (CDC) Advisory Committee on Immunization Practices (ACIP) is expected to discuss recommendations on the safe and appropriate use of Ticovac.
“We are proud to deliver the first vaccine to help protect people in the U.S. against TBE, if they are traveling to any risk areas,” says Nanette Cocero, Ph.D., global president, Vaccines, Pfizer. “This vaccine has helped to protect millions of people in TBE endemic regions since its first approval outside the U.S. 45 years ago. This authorization helps to ensure that people from the U.S. are also able to receive this vaccination if needed, reflecting our commitment to provide health for all.”
TBE is a viral infection of the brain and spine, which can be transmitted to humans through the bite of an infected tick. Although TBE is not endemic in the United States, to date, it has been identified in more than 35 countries across Europe and Asia. The European Centre for Disease Prevention and Control (ECDC) currently recommends TBE vaccination for people who live in or are traveling to risk areas.
Another August approval came in the form of Xeljanz receiving European Commission (EC) clearance for the treatment of active polyarticular juvenile idiopathic arthritis (JIA) and juvenile psoriatic arthritis (PsA) in patients 2 years of age and older who have responded inadequately to previous therapy with disease modifying antirheumatic drugs (DMARDs). Two formulations were approved by the EC, a tablet and a new oral solution (weight-based dosing). Xeljanz represents the first JAK inhibitor approved in Europe for treating polyarticular JIA and juvenile PsA, and has received regulatory clearance in four indications in the European Union, the most of any JAK inhibitor.
The New England Journal of Medicine during June published positive findings from the STOP-COVID trial (NCT04469114) assessing the efficacy and safety of oral tofacitinib in 289 hospitalized adult patients with COVID-19 pneumonia who were not on ventilation. The study shows cumulative incidence of death or respiratory failure through day 28 was 18.1 percent (26 of 144) with tofacitinib versus 29 percent (42 of 145) with placebo, in hospitalized patients with COVID-19 pneumonia. The multi-center, randomized, double-blind, placebo-controlled study was performed across 15 sites in Brazil. As this magazine went to publication, tofacitinib had not been approved or authorized for use by any regulatory authority for treating COVID-19.
Also in August, Pfizer announced positive top-line results from the Phase IIb/III ALLEGRO trial evaluating oral once-daily ritlecitinib in patients with alopecia areata, an autoimmune disease driven by an immune attack on the hair follicles that causes hair loss on the scalp and can also affect the face and body. Ritlecitinib 50 mg and 30 mg achieved the primary efficacy endpoint of the study, namely the proportion of patients with less than or equal to 20 percent scalp hair loss after six months of treatment versus placebo.
Ritlecitinib is the first in a new investigational class of covalent kinase inhibitors that have high selectivity for Janus kinase 3 (JAK3) and members of the tyrosine kinase expressed in hepatocellular carcinoma (TEC) kinase family. In laboratory studies, ritlecitinib has been shown to block the activity of signaling molecules and immune cells believed to contribute to loss of hair in people with alopecia areata. Granted Breakthrough Therapy designation from the U.S. FDA for the treatment of alopecia areata in September 2018, ritlecitinib is also being evaluated for vitiligo, rheumatoid arthritis, Crohn’s disease and ulcerative colitis.
In June, Pfizer received news about another vaccine, this time for FDA’s approval of Prevnar 20 (Pneumococcal 20-valent Conjugate Vaccine) for the prevention of invasive disease and pneumonia caused by the 20 Streptococcus pneumoniae (pneumococcus) serotypes in the vaccine in adults ages 18 years and older. The U.S. Centers for Disease Control and Prevention’s ACIP is expected to meet in October to discuss and update recommendations on the safe and appropriate use of pneumococcal vaccines in adults.
Prevnar 20 includes capsular polysaccharide conjugates for the 13 serotypes (1, 3, 4, 5, 6A, 6B, 7F, 9V, 14, 18C, 19A, 19F and 23F) already included in Prevnar 13. The vaccine also contains capsular polysaccharide conjugates for seven additional serotypes (8, 10A, 11A, 12F, 15B, 22F and 33F) that cause invasive pneumococcal disease and have been associated with high case-fatality rates, antibiotic resistance, and/or meningitis.
In the United States, more than half of all cases of invasive pneumococcal disease – which include bacteremia and meningitis – in adults ages 65 or older are due to the 20 serotypes in Prevnar. In the United States, these 20 serotypes are estimated to cause up to 250,000 cases of IPD (including bacteremia and meningitis) and community-acquired pneumonia and more than 10,000 deaths in adults ages 18 or older. Overall, the seven additional serotypes in Prevnar 20 account for approximately 40 percent of all pneumococcal disease cases and deaths in the United States.
Pfizer reported positive top-line results during September 2021 from a Phase III trial (B7471004) assessing the safety and immunogenicity of Prevnar 20 in adults 65 years of age or older when administered at the same time as the seasonal influenza vaccine (SIIV, Fluad Quadrivalent [adjuvanted], 2020/2021 strains). According to Pfizer, responses elicited by Prevnar 20 for all 20 serotypes and by seasonal influenza vaccine when given together were noninferior (the clinical trial’s primary immunogenicity objectives) to those elicited by the vaccines when administered one month apart. The safety profile of Prevnar 20 was similar when the vaccines were jointly administered as compared to when each vaccine was administered separately, one month apart.
“We are encouraged by these results showing that these two vaccines can be administered at the same time without affecting the immune protection provided by either vaccine or changing the safety profile,” Jansen stated. “This study adds to the body of evidence further supporting that pneumococcal conjugate vaccines may be coadministered with influenza vaccines, this time studied with the adjuvanted influenza vaccine. We are committed to vaccine development to help address needs across many respiratory diseases.”
The mRNA technology developed with BioNTech is being explored in other vaccine uses. During September, Pfizer announced that the first participants were dosed in a Phase I clinical trial to evaluate the safety, tolerability, and immunogenicity of a single-dose quadrivalent mRNA vaccine against influenza in healthy adults. Pfizer’s mRNA influenza vaccine program is the first in a planned wave of programs leveraging mRNA technology for influenza. Beyond influenza, the company plans to explore mRNA in other respiratory viruses, including medically appropriate vaccines combinations that could provide protection against more than one respiratory virus, as well as expand to develop mRNA technology in oncology, and genetic diseases.
The Phase I randomized study will take place in the United States and will start by evaluating the safety, tolerability, and immunogenicity of a single dose of an influenza mRNA vaccine in healthy adults 65-85 years of age, with an FDA-approved standard quadrivalent influenza vaccine as a control.
“Since 2018, we have been working to develop a potential mRNA influenza vaccine, driven by our deep understanding of infectious diseases and our extensive experience in researching, developing and implementing new vaccine technologies to help prevent them,” Jansen says. “The COVID-19 pandemic allowed us to deliver on the immense scientific opportunity of mRNA. Influenza remains an area where we see a need for vaccines which could result in improved efficacy in any given season, and we believe mRNA is the ideal technology to take on this challenge to transform global health outcomes.”
Conventional seasonal influenza vaccines are generally developed by growing the virus in chicken eggs or mammalian cells, which are inactivated and processed to be made into a vaccine. This process faces multiple challenges, including producing immunogenic antigens, keeping up with virus strain changes, and alterations in the vaccine antigens during production. Even when the vaccine strains match circulating influenza virus strains well, current seasonal vaccines typically confer 40 percent to 60 percent protection against circulating strains, with even lower protection in years with poor matching of strains. Influenza causes approximately 5 million cases of severe illness and up to 650,000 deaths worldwide every year.
mRNA-based influenza vaccine design requires only the genetic sequence of the virus. The flexibility of mRNA technology and its rapid manufacturing could potentially allow better strain match, greater reliability of supply, and the potential opportunity to improve upon the efficacy of current flu vaccines. Furthermore, in a pandemic influenza situation, mRNA technology could allow rapid, large-scale manufacturing of effective vaccines.
A supplemental Biologics License Application (sBLA) for Panzyga (Immune Globulin Intravenous [Human] – ifas 10% Liquid Preparation) was approved by the FDA in February to treat adults with a rare neurological disease of the peripheral nerves called chronic inflammatory demyelinating polyneuropathy (CIDP). Panzyga is the first intravenous immunoglobulin (IVIg) with two FDA-approved maintenance dosing options for CIDP.
FDA approval was granted in January 2021 for a new indication for Xalkori (crizotinib): for treating pediatric patients 1 year of age and older and young adults with relapsed or refractory, systemic anaplastic large cell lymphoma (ALCL) that is anaplastic lymphoma kinase (ALK)-positive.
EMD Serono and Pfizer announced in January that the European Commission approved Bavencio (avelumab) as monotherapy for the first-line maintenance treatment of adult patients with locally advanced or metastatic urothelial carcinoma (UC) who are progression-free following platinum-based chemotherapy. Bavencio is the first immunotherapy to show a significant overall survival benefit in the first-line setting in a Phase III study.
Into The Future
Looking ahead, we remain focused on being nimble and investing in our R&D organization, so we can build on the strong improvement in key metrics we’ve seen over the past five years,” Bourla says. “We continue to expect a revenue CAGR of at least 6 percent, on a risk-adjusted basis, through the end of 2025, as well as double-digit growth on the bottom line. We remain very confident in our ability to achieve these growth rates because of the strength of both our current product portfolio and our R&D pipeline.”
At the same time, Pfizer will continue to pursue business development opportunities with the potential to enhance its long-term (post-2025) growth prospects.
“Through it all, our Purpose Blueprint will remain our roadmap for success, our commitment to patients will be our North Star, and the power of science will be the engine that drives us forward,” Bourla says.
In another step for its business development plans, in August, Aamir Malik joined the company as executive VP and chief business innovation officer. Malik became a member of Pfizer’s executive leadership team reporting to Bourla. Malik joined Pfizer from McKinsey & Company, where he most recently served as the managing partner responsible for the firm’s U.S. operations. Previously, he led the firm’s Global Pharmaceuticals & Medical Products practice.
Malik succeeded John Young, executive VP and chief business officer, who announced his intent to retire after a 34-year career at Pfizer. Malik began his role on August 30, 2021 and worked with Young to ensure a seamless transition.
At Pfizer, Malik is overseeing the company’s strategy, business development, portfolio management, pipeline prioritization, and formation of new business ventures, as well as the advancement of innovative access partnerships with payers and governments around the world.
“Aamir brings with him 25 years of experience developing innovative growth strategies, guiding mergers and acquisitions, and implementing high-impact programs to improve patients’ lives and transform performance for life science companies,” Bourla says. “He is the ideal leader to drive these efforts.”
One of these efforts was announced in August, when Pfizer acquired Trillium Therapeutics Inc., a clinical-stage immuno-oncology company developing innovative therapies for the treatment of cancer.
Pfizer is acquiring all outstanding shares of Trillium not already owned by Pfizer for an implied equity value of $2.26 billion, or $18.50 per share, in cash. This represents a 118 percent premium to the 60-day weighted average price for Trillium.
Trillium’s portfolio includes biologics that are designed to enhance the ability of patients’ innate immune system to detect and destroy cancer cells. The company’s two lead molecules, TTI-622 and TTI-621, block the signal-regulatory protein α (SIRPα)–CD47 axis, which is emerging as a key immune checkpoint in hematological malignancies.
TTI-622 and TTI-621 are novel, potentially best-in-class SIRPα-Fc fusion proteins that are undergoing Phase Ib/II development across several indications, with a focus on hematological malignancies.
“Today’s announcement reinforces our commitment to pursue scientific breakthroughs with the addition of potentially best-in-class molecules to our innovative pipeline,” says Andy Schmeltz, global president and general manager, Pfizer Oncology. “The proposed acquisition of Trillium builds on our strong track record of leadership in oncology, enhancing our hematology portfolio as we strive to improve outcomes for people living with blood cancers around the globe. Our deep experience in understanding the science of blood cancers, along with the diverse knowledge base we have developed across our growing hematology portfolio of eight approved and investigational therapies, provide us with a foundation to advance these important potential medicines to patients who need them.”
Hematological malignancies are cancers that affect the blood, bone marrow, and lymph nodes. This classification includes various types of leukemia, multiple myeloma, and lymphoma.
More than 1 million people worldwide were diagnosed with a blood cancer in 2020, representing almost 6 percent of all cancer diagnoses globally. In 2020, more than 700,000 people worldwide died from a form of blood cancer.
In September 2020, as part of the Pfizer Breakthrough Growth Initiative (PBGI), Pfizer invested $25 million in Trillium and Jeff Settleman, senior VP and chief scientific officer of Pfizer’s Oncology Research & Development Group, was named to Trillium’s Scientific Advisory Board.
Established in June 2020, PBGI’s goal is to provide funding for scientific research as well as access to Pfizer’s experts to ensure the continuity of clinical programs that could be of potential strategic interest for Pfizer. Pfizer has committed to providing up to $500 million in total funding to the PBGI.
In another 2021 acquisition for Pfizer, during April the company purchased Amplyx Pharmaceuticals Inc. A privately held company, Amplyx is dedicated to the development of therapies for debilitating and life-threatening diseases that affect people with compromised immune systems. The company’s lead compound, fosmanogepix (APX001), is a novel investigational asset under development for treating invasive fungal infections.
In addition to fosmanogepix, Pfizer has secured ownership of Amplyx’s early-stage pipeline that includes potential antiviral (MAU868) and antifungal (APX2039) therapies. The acquisition follows an initial equity investment by Pfizer during December 2019 as part of Amplyx’s Series C financing.
Management says the Amplyx acquisition represents an opportunity to advance Pfizer’s expertise and deep heritage in infectious disease.