Pfizer advanced the cancer pipeline in 2019, but with the advent of the COVID-19 pandemic, the company threw its scientific strength at generating a vaccine against the novel coronovirus.

By Christiane Truelove • chris.truelove@medadnews.com

Pfizer Inc.

235 East 42nd Street
New York, NY 10017
Phone: 212-733-2323
Website: pfizer.com

FINANCIAL PERFORMANCE

(All figures are in millions of dollars, except EPS)

2019

Revenue $51,750 

Net income $16,273  

Diluted EPS $2.87         

R&D expense $8,650  

1H 2020

Revenue $23,829       

Net income $6,828  

Diluted EPS $1.22  

R&D expense $3,856  

BEST-SELLING Rx PRODUCTS

(All sales are in millions of dollars)

2019

Prevnar 13/Prevenar 13 $5,847 

Ibrance $4,961 

Eliquis* $4,220 

Lyrica  $3,321 

Xeljanz  $2,242 

Lipitor $1,973 

Enbrel $1,699            

Chantix/ Champix $1,107 

Norvasc $950 

Sutent $936 

Xtandi ** $838 

Premarin family $734 

Celebrex $719 

Sulperazon $684 

Inflectra/Remsima $625 

Xalkori $530  

1H 2020

Ibrance $2,598 

Eliquis* $2,572  

Prevnar 13/Prevenar 13  $2,566  

Xeljanz $1,086  

Lipitor $836  

Lyrica $706  

Enbrel $684    

Vyndaqel/Vyndamax $508  

Chantix/Champix $505  

Xtandi** $475  

Norvasc $419  

Sutent $414  

Inlyta $364  

Inflectra/Remsima $308  

Premarin family $304  

Celebrex $295  

Sulperazon $289 

Xalkori  $287  

Notes: 
* – Includes alliance revenue and direct sales
** – Includes alliance revenue

Outcomes Creativity Index Score: 23
Manny Awards – N/A
Cannes Lions – N/A
LIA: Health & Wellness – 4
Clio Health – 8
One Show: HW&P – N/A
MM&M Awards – 3
Global Awards – 8
Creative Floor Awards – N/A

For Pfizer, 2019 was a busy year, according to Dr. Albert Bourla, Pfizer’s chairman and CEO. The year was highlighted by solid financial performance, shareholder-friendly capital allocation, the strengthening of the company’s pipeline, as well as the formation of the consumer healthcare joint venture with GlaxoSmithKline. Pfizer also announced a definitive agreement to combine its Upjohn unit with Mylan to create a new global pharmaceutical company, Viatris, which Dr. Bourla says marks an important milestone in Pfizer’s evolution toward becoming a more focused, global leader in innovative medicines.

“Pfizer calls on all members of the innovation ecosystem – from large pharmaceutical companies to the smallest of biotech companies, from government agencies to academic institutions – to commit to work together in addressing this dire crisis,” says Alfred Bourla, CEO.

Upon releasing the company’s 2019 financial results at the end of January 2020, Dr. Bourla stated, “2020 is expected to be an exciting year for Pfizer with the close of the Upjohn-Mylan transaction anticipated by mid-year, leaving New Pfizer positioned to deliver revenue and adjusted diluted EPS growth that is expected to be among the industry leaders. New Pfizer will be a smaller, science-based company with a singular focus on innovation while also continuing to allocate significant capital directly to shareholders, primarily through dividends.”

But then came COVID-19, delaying the formation of Viatris, and suddenly Pfizer found itself square in the battle against coronavirus as the lockdown began in March. 

“In this troubling time, Pfizer is committed to doing all we can to respond to the COVID-19 pandemic,” Dr. Bourla said on March 13 when the company outlined a five-point plan to fight COVID-19. “Many companies, including Pfizer, are working to develop antiviral therapies to help infected patients fight this emerging virus as well as new vaccines to prevent infection and halt the further spread of this disease. Pfizer is working to advance our own potential antiviral therapies and is engaged with BioNTech on a potential mRNA coronavirus vaccine. We are committed to work as one team across the industry to harness our scientific expertise, technical skills and manufacturing capabilities to combat this evolving crisis.”

Pfizer promised to share tools and assets; marshal its people; apply the company’s drug-development expertise; offer its manufacturing capability; and improve future rapid response. 

“In recent years, the biopharmaceutical industry has brought forward some of the most impactful medical breakthroughs known to society, from therapies for HIV and cancer that have extended millions of lives to novel gene therapies that are seeing cure-like outcomes for some the most devastating rare diseases,” Dr. Bourla says. “Pfizer calls on all members of the innovation ecosystem – from large pharmaceutical companies to the smallest of biotech companies, from government agencies to academic institutions – to commit to work together in addressing this dire crisis. With our combined efforts we know that there is no health challenge that we cannot overcome.”

In September, Dr. Bourla joined the CEOs of eight other pharmaceutical companies developing COVID-19 vaccines to continue to make the safety and well-being of vaccinated individuals the top priority. The other CEOs signing the pledge are from AstraZeneca, BioNTech, GlaxoSmithKline, Johnson & Johnson, Merck, Moderna, Novavax, and Sanofi.

The companies pledged to always make the safety and well-being of vaccinated individuals their top priority; continue to adhere to high scientific and ethical standards regarding the conduct of clinical trials and the rigor of manufacturing processes; only submit for approval or emergency use authorization after demonstrating safety and efficacy through a Phase III clinical study that is designed and conducted to meet requirements of expert regulatory authorities such as FDA; and work to ensure a sufficient supply and range of vaccine options, including those suitable for global access. 

“We believe this pledge will help ensure public confidence in the rigorous scientific and regulatory process by which COVID-19 vaccines are evaluated and may ultimately be approved,” the CEOs stated.

The German phase of the COVID-19 vaccine trial with BioNTech began in April. By May, the U.S. phase of the trial started, with the first patients being dosed.

By July, the two companies had announced early positive data from a Phase I/II trial for BNT162b1, an MRNA-based vaccine candidate. At day 28 (seven days after dose two), all subjects who received 10 or 30 µg of BNT162b1 had significantly elevated RBD-binding IgG antibodies with geometric mean concentrations (GMCs) of 4,813 and 27,872 units/ml, which are eight and 46.3-times, respectively, the GMC of 602 units/ml in a panel of 38 sera of convalescent patients who had contracted SARS-CoV-2

At day 28 (seven days after dose two), all subjects who received 10 or 30 µg of BNT162b1 had SARS-CoV-2 neutralizing antibodies with geometric mean titers (GMTs) of 168 and 267, which are 1.8- and 2.8-times, respectively, the GMT of the convalescent serum panel.

Local reactions and systemic events after immunization with 10 µg and 30 µg of BNT162b1 were dose-dependent, generally mild to moderate, and transient. No serious adverse events were reported.

FDA granted Fast Track Designation to BNT162b1 and the candidate was advanced into global clinical studies in July. Pfizer and BioNTech also pledged to provide 20 million doses of the vaccine to the United Kingdom, 120 million to Japan, 200 million to the EU, and 600 million to the United States.

In September, Pfizer and BioNTech proposed an expansion of their Phase III pivotal COVID-19 vaccine trial to up to 44,000 participants, which allows for the enrollment of new populations. The proposed expansion would allow the companies to further increase trial population diversity, and include adolescents as young as 16 years of age and people with chronic, stable HIV (human immunodeficiency viruses), hepatitis C or hepatitis B infection, as well as provide additional safety and efficacy data. 

Based on infection rates, at the time, the companies expected that a conclusive readout on efficacy was likely by the end of October 2020.

During August, Pfizer announced a multi-year agreement with Gilead Sciences Inc. to manufacture and supply Gilead’s investigational antiviral remdesivir, as one of multiple external manufacturing organizations supporting efforts to scale up supply of the investigational treatment for COVID-19. Under the terms of the agreement, Pfizer is providing contract manufacturing services at Pfizer’s McPherson, Kansas facility to manufacture and supply remdesivir for Gilead.

“From the beginning it was clear that no one company or innovation would be able to bring an end to the COVID-19 crisis. Pfizer’s agreement with Gilead is an excellent example of members of the innovation ecosystem working together to deliver medical solutions,” Dr. Bourla said on Aug. 7. “Together, we are more powerful than alone. As one of the largest manufacturers of vaccines, biologics and sterile injectables, it is a privilege to offer our expertise and infrastructure to help fight this pandemic. In that spirit, we are pleased that Gilead is using our manufacturing capacity to help facilitate supply of this medicine to patients as quickly as possible.”

Financial & Product Performances

Pfizer generated revenue of $51.75 billion in 2019, 4 percent less than in 2018. The decreased in revenue was attributed to the impact of the closing of the consumer health joint venture with GlaxoSmithKline, GSK Consumer Healthcare, in the third quarter of 2019. Pfizer owns a 32 percent equity stake in the joint venture and GSK owns 68 percent.

Pfizer’s 2019 net income was $16.27 billion, 46 percent more than in 2018. Diluted earnings per share were $2.87 compared with $1.87 in the prior year.

For the first half of 2020, revenue was $23.83 billion, 10 percent less than in the previous year’s period. Net income was $6.83 billion compared with $8.93 billion in first-half 2019. Diluted earnings were share for the 2020 first half were $1.22, 22 percent less than in first-half 2019. Pfizer attributes the declines to the impact of COVID-19 on the company’s sales and marketing activities due to widespread restrictions on in-person meetings with healthcare professionals and the refocused attention of the medical community on fighting the pandemic. 

“Access to prescribers for sales force colleagues during second-quarter 2020 was mixed, with those in most international markets able to meet with healthcare professionals for most of the quarter, while those in the U.S. were unable to meet in-person with doctors for nearly all of the quarter,” Pfizer executives stated. “As a result of the lower number of in-person meetings with prescribers and restrictions on patient movements due to government-mandated work-from-home or shelter-in-place policies, the rate of new prescriptions for certain products and of vaccination rates for most vaccines slowed in certain markets, including the U.S., which negatively impacted second-quarter 2020 financial results. These declines were partially offset by certain Pfizer medicines and vaccines that saw increased demand in certain markets compared to the prior-year quarter, including Prevenar 13 for streptococcus pneumoniae in adults in international markets as well as certain sterile injectable products utilized in the intubation and ongoing treatment of mechanically ventilated COVID-19 patients.”

Pfizer’s top-selling franchise in 2019 for the fifth consecutive year was the Prevnar 13 vaccine family, which generated $5.85 billion, 1 percent more than in 2018. In first-half 2020, Prevnar 13/Prevenar 13 sales totaled $2.57 billion, down 4 percent versus same-period 2019.

The oncology drug Ibrance hopped into Pfizer’s No. 2 sales spot in 2019, generating $4.96 billion, 20 percent more than in 2018. In the first six months of 2020, Ibrance sales were $2.6 billion, 9 percent more than in first-half 2018.

Next in the 2019 sales line was the blood thinner Eliquis, for which Pfizer recorded alliance revenue and direct sales of $4.22 billion, representing 23 percent growth from 2018. During first-half 2020 the drug generated $2.57 billion in alliance revenue and direct sales for Pfizer, 23 percent more than in the first half of 2019.

No. 4 was Lyrica, which posted sales of $3.32 billion, 33 percent less than in 2019. Sales in the first half of 2020 totaled $706 million compared with $2.36 billion in the same period of the previous year.

Pfizer’s fifth best seller was the rheumatoid arthritis, psoriatic arthritis and ulcerative colitis drug Xeljanz, which made $2.24 billion in 2019, 26 percent more than in 2018. First-half 2020 sales were $1.09 billion, up 5 percent versus first-half 2019.

Once again at No. 6 in sales was the cholesterol drug Lipitor, generating $1.97 billion, 4 percent less than in 2018. Sales for first-half 2020 were $836 million, 19 percent less than in the first six months of 2019.

The rheumatoid arthritis drug Enbrel slipped to No. 7 in Pfizer sales for 2019, at $1.7 billion, 20 percent less than in 2018. In the first half of 2020, the company’s sales amounted to $684 million, a decline of 21 percent compared with first-half 2019. 

Coming in at No. 8 was the smoking cessation drug Chantix/Champix, with $1.11 billion, an increase of 2 percent from 2018. Sales in the first half of 2020 were $505 million, 8 percent less than in first-half 2019.

At No. 9 was the hypertension drug Norvasc, with sales of $950 million, 8 percent less than in 2018. In the first half of 2020, sales were $419 million, 19 percent less than in the same period of the prior year.

The oncology drug Sutent placed No. 10 in sales in 2019, at $936 million, 11 percent less than in 2018. During the first half of 2020, Sutent sales declined 14 percent from first-half 2019 to $414 million. 

Pfizer reported alliance revenue of $838 million for the oncology drug Xtandi in 2019, growing from $699 in 2018. First-half 2020 alliance revenue amounted to $475 million, 29 percent more than in first-half 2018.

The Premarin family of hormone replacement products was the 12th best seller in 2019, at $734 million, a decline of 12 percent. Sales in the first six months of 2020 were $304 million, 16 percent less than in the same period of 2019.

The anti-inflammatory drug Celebrex moved to N0. 13 on Pfizer’s 2019 best sellers list, with sales of $719 million, 5 percent more from the previous year. First-half 2020 sales were $295 million, 15 percent less than in first-half 2019.

No. 14 on Pfizer’s 2019 sales ladder was the combination antibiotic Sulperazon, which is used in the hospital setting. The product generated $684 million, 12 percent more than in 2018. First-half 2020 sales were $289 million, 16 percent less than in the same period of 2019.

N0. 15 in 2019 sales was the immunosuppressive drug Inflectra/Remsima at $625 million, 3 percent less than in 2018. Sales in the first six months of 2020 were $308 million, up 6 percent from first-half 2019.

No. 16 was the oncology drug Xalkori at $530 million, up 1 percent versus the 2018 amount. Sales in the first half of 2020 reached $287 million, 12 percent more than in first-half 2019.

R&D and the pipeline

Pfizer had a lot of R&D plans at the beginning of 2020. When presenting the company’s 2019 results, Dr. Bourla stated that in the first half of 2020, the company expected to report pivotal top-line results for the JADE Compare study for abrocitinib (PF-04965842), Pfizer’s Janus kinase-1 (JAK1) inhibitor for moderate-to-severe atopic dermatitis (AD); for three Phase III trials of PF-06482077, a 20-valent pneumococcal conjugate vaccine candidate in adults aged 18 and older; and for Xeljanz in ankylosing spondylitis, in addition to the potentially registration-enabling Phase II ANCHOR study evaluating the combination of Braftovi, Mektovi and cetuximab for the first-line treatment of BRAFV600E-mutant metastatic colorectal cancer. 

The company also expected data in the first half of 2020 for promising earlier-stage opportunities, including proof-of-concept readouts for PF-06939926, a mini-dystrophin gene therapy candidate for Duchenne muscular dystrophy; for PF-06928316, a prophylactic vaccine candidate for the prevention of respiratory syncytial virus infection; and for PF-06700841, an investigational topical TYK2/ JAK1 dual inhibitor for psoriasis and AD.

In the second half of 2020, the company expected top-line results for the Phase III PENELOPE-B study of Ibrance in early-stage breast cancer, as well as for proof-of-concept readouts for PF-06651600, a dual JAK3/TEC inhibitor as a potential treatment for vitiligo; for PF-06700841 for the potential treatment of psoriatic arthritis (PsA); and for PF-06826647, an investigational TYK2 inhibitor for psoriasis. 

COVID-19, however, threw some of these plans awry with what company executives described as a brief pause in the recruitment portion of certain ongoing clinical studies and a delay to most new study starts, However, in late April 2020, Pfizer restarted recruitment across the development portfolio, including new study starts. 

“Pfizer continues to work closely with clinical trial sites to understand their needs and is performing remote monitoring where appropriate to oversee study conduct,” management says. “In addition, processes to enable tele-health and home healthcare are being utilized where appropriate to continue the data collection process and support patient safety.”

Pfizer kicked off 2020 with an announcement with development partner EMD Serono, the pharmaceuticals business of Merck KGaA, that the Phase III JAVELIN Bladder 100 study met its primary endpoint of overall survival (OS) at the planned interim analysis. In this study, patients with previously untreated locally advanced or metastatic urothelial carcinoma (UC) whose disease did not progress on induction chemotherapy and who were randomized to receive first-line maintenance therapy with Bavencio (avelumab) and best supportive care (BSC) lived significantly longer than those who received BSC only.

“Bavencio is the first immunotherapy to demonstrate in a clinical trial a statistically significant improvement in overall survival as a first-line treatment for patients with advanced urothelial carcinoma,” says Chris Boshoff, M.D., Ph.D., chief development officer, Oncology, Pfizer Global Product Development. “These latest positive data from the JAVELIN clinical development program add to the body of evidence for Bavencio in the treatment of genitourinary cancers, and we look forward to discussing these results with health authorities.”

In February, Astellas Pharma Inc. and Pfizer announced results of the final overall survival ß(OS) analysis from the Phase III PROSPER trial, which evaluated Xtandi (enzalutamide) plus androgen deprivation therapy (ADT) versus placebo plus ADT in men with non-metastatic castration-resistant prostate cancer (nmCRPC).

The results demonstrated a statistically significant improvement in OS in patients with nmCRPC who were treated with Xtandi plus ADT. OS was a key secondary endpoint of the trial. In a preliminary analysis, adverse events were generally consistent with those previously reported from PROSPER. 

In May, the companies shared final results from this trial, which showed that Xtandi plus ADT reduced the risk of death by 27 percent (n=1,401; hazard ratio [HR]=0.73; [95% confidence interval [CI]: 0.61-0.89]; p=0.001) compared to placebo plus ADT. The median OS was 67.0 months (95 percent CI: 64.0 to not reached) for men who received Xtandi plus ADT compared to 56.3 months (95 percent CI: 54.4 to 63.0) with placebo plus ADT. 

These data were simultaneously published online in the New England Journal of Medicine and presented during the virtual scientific program of the 2020 American Society of Clinical Oncology (ASCO) Annual Meeting. 

In March, FDA accepted for review a Biologics License Application (BLA) for tanezumab 2.5 mg administered subcutaneously (SC), which is being evaluated for patients with chronic pain due to moderate-to-severe osteoarthritis who have experienced inadequate pain relief with other analgesics. Tanezumab is a monoclonal antibody that is part of an investigational class of non-opioid chronic pain medications known as nerve growth factor (NGF) inhibitors.

The Prescription Drug User Fee Act (PDUFA) goal date for the FDA to make a decision on the tanezumab application is in December 2020.

Though good news came in January for one of the Bavencio trials, in March EMD Serono and Pfizer Inc. announced that they had accepted the recommendation of the independent data monitoring committee to terminate the Phase III JAVELIN Head and Neck 100 study. The trial was evaluating avelumab in addition to chemoradiotherapy (CRT) versus standard-of-care CRT in patients with untreated locally advanced squamous cell carcinoma of the head and neck (LA SCCHN). The recommendation to terminate was made because the study is unlikely to show a statistically significant improvement in the primary endpoint of progression-free survival (PFS) based on a preplanned interim analysis. 

In April, EMD Serono and Pfizer announced completion of the submission of a supplemental Biologics License Application (sBLA) to FDA for Bavencio for first-line maintenance treatment of patients with locally advanced or metastatic urothelial carcinoma. The FDA granted Breakthrough Therapy Designation to Bavencio for this indication, and the sBLA was being reviewed by the FDA under its Real-Time Oncology Review (RTOR) pilot program.

The application is based on the positive results from an interim analysis of the Phase III JAVELIN Bladder 100 trial, preliminary results of which were announced during January. The interim analysis found that the trial met the primary endpoint of OS. Bavencio plus best supportive care as first-line maintenance therapy significantly extended the survival of patients with previously untreated locally advanced or metastatic UC whose disease did not progress on induction chemotherapy, compared with BSC only. A statistically significant improvement was demonstrated in both co-primary populations: all randomized patients and patients with PD-L1–positive tumors. 

According to Dr. Boshoff, “Participation in the Real-Time Oncology Review program, coupled with the Breakthrough Therapy Designation, reflect the potential impact of Bavencio in this patient setting and provide the opportunity to work towards bringing this treatment option to patients as quickly as possible.”

The RTOR program is intended to create a more efficient review process to bring safe and effective treatments to patients earlier, including drugs that are likely to demonstrate substantial improvements over currently available therapy. The program allows the FDA to review clinical trial data from certain applications before the complete application is formally submitted. 

FDA approved in June the sBLA for Bavencio in this indication. The agency previously approved Bavencio under the accelerated approval program in 2017 for the treatment of patients with locally advanced or metastatic UC who have disease progression during or following platinum-containing chemotherapy, or who have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy, based on tumor response rate and duration of response. Continued approval was contingent upon verification of clinical benefit, which was demonstrated in JAVELIN Bladder 100. The FDA converted the accelerated approval to full approval.

Pfizer announced in May that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency adopted a positive opinion for the Hedgehog pathway inhibitor Daurismo. The CHMP recommended marketing authorization for Daurismo (glasdegib), in combination with low-dose cytarabine, a type of chemotherapy, for the treatment of newly diagnosed (denovo or secondary) acute myeloid leukemia in adult patients who are not candidates for standard chemotherapy. The CHMP’s opinion is being reviewed by the European Commission.

Also in May, Pfizer announced updated Phase 1b clinical data on PF-06939926, an investigational gene therapy being developed to treat Duchenne muscular dystrophy (DMD). The preliminary data from 9 ambulatory boys with DMD, aged 6 to 12 (mean age: 8 years) indicate that the intravenous administration of PF-06939926 was well-tolerated during the infusion period, with encouraging efficacy and manageable safety events, even when considering those adverse events that were more severe in nature. The treatment provided durable and statistically significant improvements across multiple efficacy-related endpoints measured at 12 months post-infusion, including sustained levels of mini-dystrophin expression and improvements on the North Star Ambulatory Assessment (NSAA) rating scale, which is a validated measure of muscle function. Three serious adverse events (SAEs) were recorded, two of which reflected likely complement activation. While these two SAEs were severe in nature, all three events fully resolved within two weeks, providing encouragement that close monitoring and early intervention can help mitigate the effects of complement activation. This new dataset, which includes updated 12-month results on safety, dystrophin expression, and exploratory functional endpoints for three additional boys, was presented for the first time at the American Society of Gene & Cell Therapy Annual Meeting.

“Based on the encouraging preliminary efficacy data and manageable safety events from our Phase 1b study, we believe we may have a potential breakthrough therapy for boys with Duchenne muscular dystrophy, a devastating disease for which there remains a significant medical need,” says Seng Cheng, Ph.D., chief scientific officer, Pfizer Rare Disease Research Unit. “We are advancing our Phase III program as quickly as possible and plan to begin dosing patients in the second half of 2020 pending regulatory approval. Our program has the potential to be the first DMD gene therapy Phase III trial start using a commercial-scale manufacturing process. If the program is successful, this manufacturing capability is expected to help position us to deliver this medicine to patients quickly following regulatory approval.”

Pfizer announced on Oct. 1, 2020, that PF-06939926 received Fast Track designation from the FDA for DMD.

Although Pfizer was expected to complete the PALLAS trial in early-stage breast cancer during early 2021, the month of May instead brought bad news. The independent data monitoring committee determined that the clinical trial is unlikely to show a statistically significant improvement in the primary endpoint of invasive disease-free survival (iDFS). The PALLAS trial compared palbociclib plus standard adjuvant endocrine therapy to standard adjuvant endocrine therapy alone in women and men with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) early (stage 2 and 3) breast cancer.

In September, FDA accepted and granted priority review to the supplemental New Drug Application (sNDA) for Xalkori (crizotinib) for the treatment of pediatric patients with relapsed or refractory systemic anaplastic large cell lymphoma (ALCL) that is anaplastic lymphoma kinase (ALK)-positive. Xalkori received Breakthrough Therapy Designation (BTD) for the ALK-positive ALCL indication in May 2018 and if approved, would be the first biomarker-driven therapy for this type of pediatric lymphoma. The PDUFA goal date for a decision by the FDA is January 2021.

“Despite high survival rates for children with ALK-positive anaplastic large cell lymphoma, many will relapse, requiring novel treatment approaches,” Dr. Boshoff says, adding that the sNDA filing exemplifies Pfizer’s commitment to broadening the use of biomarker-driven therapies in areas with significant needs, such as rare, pediatric cancers. 

“Given Xalkori’s proven efficacy in ALK-positive lung cancer and activity seen in clinical trials investigating relapsed or refractory ALK- and ROS-1 positive anaplastic large cell lymphoma, if approved, Xalkori could represent an important step toward improving outcomes for children with this type of cancer,” Boschoff says.

In August, Pfizer announced that the CROWN study of Lorbrena (lorlatinib) in people with previously untreated advanced ALK-positive NSCLC met its primary endpoint by demonstrating significantly improved PFS, as compared to Xalkori. The results were reviewed by an independent data monitoring committee at a planned interim analysis. The safety profile for lorlatinib and crizotinib were consistent with what has been previously seen in clinical trials. 

“Almost a decade ago, we pioneered the first biomarker-driven medicine for ALK-positive non-small cell lung cancer, which transformed treatment of this disease,” Dr. Boshoff says. “These top-line results of the CROWN study reinforce the significant benefit of Lorbrena demonstrated in later-line settings, and we are excited to share these data soon with physicians and other healthcare providers, as well as engage with global regulatory authorities to potentially provide people with previously untreated metastatic non-small cell lung cancer this third-generation ALK inhibitor.”

In 2018, FDA approved Lorbrena for the treatment of patients with ALK-positive metastatic NSCLC whose disease has progressed on crizotinib and at least one other ALK inhibitor for metastatic disease; or whose disease has progressed on alectinib or ceritinib as the first ALK inhibitor therapy for metastatic disease. 

The FDA on Sept. 25, 2020, approved Xeljanz for treating children and adolescents 2 years and older with active polyarticular course juvenile idiopathic arthritis (pcJIA). Two formulations were approved, a tablet and an oral solution, which are dosed based upon weight. Xeljanz is the first FDA-approved JAK inhibitor for treating pcJIA.