On February 4, 2021 Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) reported financial results for the fourth quarter and full year of 2020 (Press release, Alexion, FEB 4, 2021, View Source [SID1234574612]). Total revenues for the full year of 2020 were $6,069.9 million, a 22 percent increase compared to the same period in 2019. The negative impact of foreign currency on total revenues year-over-year was 1 percent, or $52.0 million, inclusive of hedging activities. On a GAAP basis, diluted EPS for the full year of 2020 was $2.72, compared to $10.70 in the prior year. Full year 2020 includes impairment charges of $2,053.3 million primarily relating to the KANUMA intangible asset and a related deferred tax benefit of $377.3 million. Full year 2019 includes one-time tax benefits of $382.2 million related to intra-entity asset transfers of intellectual property. Non-GAAP diluted EPS for the full year of 2020 was $12.51, a 19 percent increase versus the prior year.
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
Total revenues in the fourth quarter were $1,591.8 million, a 15 percent increase compared to the same period in 2019. The positive impact of foreign currency on total revenues year-over-year was less than 1 percent, or $0.1 million, inclusive of hedging activities. On a GAAP basis, diluted EPS in the quarter was $2.42, compared to $4.00 in the prior year, inclusive of one-time tax benefits of $382.2 million related to intra-entity asset transfers of intellectual property in the fourth quarter of 2019. Non-GAAP diluted EPS for the fourth quarter of 2020 was $2.96, a 9 percent increase versus the fourth quarter of 2019.
"In 2020, we delivered on our LEAD-EXPAND-DIVERSIFY strategy – progressing our commercial portfolio with multiple global regulatory approvals, and further building our pipeline, which now spans more than 20 development programs. I am so proud of our team’s remarkable execution and perseverance amidst the uncertainties of COVID-19," said Ludwig Hantson, Ph.D., Chief Executive Officer of Alexion. "We enter 2021 with significant momentum, a strong foundation and a promising future. I am confident we are well positioned to build on our success and further advance our mission of delivering life-changing medicines to people with rare diseases and devastating conditions in the coming months and once we become part of AstraZeneca."
Full Year 2020 Financial Highlights
Net product sales were $6,069.1 million, compared to $4,990.0 million in 2019.
SOLIRIS net product sales were $4,064.2 million, compared to $3,946.4 million in 2019, representing a 3 percent increase.
ULTOMIRIS net product sales were $1,076.7 million, compared to $338.9 million in 2019, representing a 218 percent increase.
STRENSIQ net product sales were $731.8 million, compared to $592.5 million in 2019, representing a 24 percent increase.
KANUMA net product sales were $117.9 million, compared to $112.2 million in 2019, representing a 5 percent increase.
ANDEXXA/ONDEXXYA net product sales were $78.5 million.
GAAP cost of sales was $553.5 million, compared to $394.5 million in 2019. Non-GAAP cost of sales was $518.2 million, compared to $380.3 million in 2019.
GAAP R&D expense was $1,002.9 million, compared to $886.0 million in 2019. Non-GAAP R&D expense was $929.4 million, compared to $720.9 million in 2019.
GAAP SG&A expense was $1,399.9 million, compared to $1,261.1 million in 2019. Non-GAAP SG&A expense was $1,198.6 million, compared to $1,099.9 million in 2019.
GAAP impairment of intangible assets was $2,053.3 million primarily related to an impairment charge recorded during the second quarter 2020 related to the KANUMA intangible asset.
GAAP income tax benefit was $34.4 million, compared to $225.5 million in 2019. GAAP income tax benefit for 2020 includes a deferred tax benefit of $377.3 million associated with the impairment charge related to the KANUMA intangible asset. GAAP income tax benefit for 2019 includes one-time tax benefits of $382.2 million related to intra-entity asset transfers of intellectual property in 2019. Non-GAAP income tax expense was $512.8 million, compared to $359.4 million in 2019.
GAAP diluted EPS was $2.72, compared to $10.70 in 2019. GAAP diluted EPS for 2020 includes impairment charges of $2,053.3 million primarily relating to the KANUMA intangible asset, offset by a deferred tax benefit of $377.3 million associated with the KANUMA impairment charge. GAAP diluted EPS for 2019 includes one-time tax benefits of $382.2 million related to intra-entity asset transfers of intellectual property in 2019. Non-GAAP diluted EPS was $12.51, compared to $10.53 in 2019.
Fourth Quarter 2020 Financial Highlights
Net product sales were $1,591.7 million in the fourth quarter of 2020, compared to $1,384.2 million in the fourth quarter of 2019.
SOLIRIS net product sales were $1,023.5 million, compared to $1,013.1 million in the fourth quarter of 2019, representing a 1 percent increase.
ULTOMIRIS net product sales were $313.5 million, compared to $170.2 million in the fourth quarter of 2019, representing an 84 percent increase.
STRENSIQ net product sales were $185.9 million, compared to $166.8 million in the fourth quarter of 2019, representing an 11 percent increase.
KANUMA net product sales were $29.2 million, compared to $34.1 million in the fourth quarter of 2019, representing a 14 percent decrease.
ANDEXXA/ONDEXXYA net product sales were $39.6 million in the fourth quarter of 2020.
GAAP cost of sales was $152.2 million, compared to $114.3 million in the fourth quarter of 2019. Non-GAAP cost of sales was $138.0 million, compared to $110.8 million in the fourth quarter of 2019.
GAAP R&D expense was $295.0 million, compared to $269.6 million in the fourth quarter of 2019. Non-GAAP R&D expense was $269.8 million, compared to $226.7 million in the fourth quarter of 2019.
GAAP SG&A expense was $444.4 million, compared to $381.0 million in the fourth quarter of 2019. Non-GAAP SG&A expense was $384.6 million, compared to $340.0 million in the fourth quarter of 2019.
GAAP income tax expense was $54.8 million, compared to income tax benefit of $287.0 million in the fourth quarter of 2019, inclusive of one-time tax benefits related to intra-entity asset transfers of intellectual property in the fourth quarter of 2019. Non-GAAP income tax expense was $111.0 million, compared to $85.8 million in the fourth quarter of 2019.
GAAP diluted EPS was $2.42, compared to $4.00 in the fourth quarter of 2019, inclusive of one-time tax benefits related to intra-entity asset transfers of intellectual property in the fourth quarter of 2019. Non-GAAP diluted EPS was $2.96, compared to $2.71 in the fourth quarter of 2019.
COVID-19
We continue to take steps to proactively respond to the evolving COVID-19 pandemic and to plan for related uncertainties. We remain focused on continuing to serve patients, protecting the health and safety of our employees and the communities in which we live and work, and supporting patients in clinical trials. We are also focused on minimizing potential interactions that could contribute to the spread of the virus and put additional strain on healthcare systems through the use of innovative virtual means where possible.
Clinical Trials: We have implemented a pandemic response business continuity plan designed to protect patients and site staff safety while continuing our clinical trials with limited interruption to the extent we are able. The COVID-19 impact has varied by study and program, but there has been little timing impact on fully-enrolled trials. By the third quarter of 2020, we had successfully re-initiated the majority of studies that had been temporarily paused earlier in the year. However, increasing COVID-19 cases have had further effects on the timing of healthy volunteer studies in particular. In addition, there has been, and may continue to be, an impact to the timing of trials that are enrolling patients and activating sites, or have not yet started to do so, based on local dynamics where these studies are being conducted.
Business Impact: We continue to take proactive measures designed to mitigate the risk of potential interruptions in supply and/or access to patients’ customary site-of-care locations. Treatment compliance rates across all our medicines have remained strong. We have also seen the predicted slowing of new patient initiations and delays in treatment starts, and we are continuing to closely monitor this environment as the pandemic continues.
Research and Development
PHASE 3/4
SOLIRIS – Guillain-Barre Syndrome (GBS): SOLIRIS in GBS has been granted SAKIGAKE designation by Japan’s Ministry of Health, Labour and Welfare (MHLW). Alexion plans to initiate a Phase 3 study of SOLIRIS in GBS in Japan in the first half of 2021.
ULTOMIRIS – 100 mg/mL: In November 2020, the ULTOMIRIS 100 mg/mL formulation for PNH and aHUS was approved in the EU. An application for approval is under review in Japan. This higher concentration formulation is designed to reduce infusion time by more than 60 percent to approximately 45 minutes.
ULTOMIRIS – Subcutaneous: The Phase 3 study of weekly subcutaneous (SC) ULTOMIRIS demonstrated PK-based non-inferiority versus intravenous ULTOMIRIS. Pending collection of 12-month safety and drug-device combination data, Alexion plans to file for approval in the U.S. and EU for the ULTOMIRIS SC formulation and device combination in PNH and aHUS in the third quarter of 2021.
ULTOMIRIS – gMG: In November 2020, Alexion completed enrollment in the Phase 3 study of ULTOMIRIS in adults with gMG. Study results are expected in the second half of 2021.
ULTOMIRIS – NMOSD: A Phase 3 study of ULTOMIRIS in NMOSD is underway.
ULTOMIRIS – Amyotrophic Lateral Sclerosis (ALS): As completion of full enrollment nears, screening of new patients has closed for the Phase 3 study of ULTOMIRIS in ALS.
ULTOMIRIS – Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy (HSCT-TMA): Dosing is underway in Phase 3 studies of ULTOMIRIS in adults and children with HSCT-TMA.
ULTOMIRIS – Complement Mediated Thrombotic Microangiopathy (CM-TMA): In January 2021, Alexion submitted an Investigational New Drug (IND) application for ULTOMIRIS in CM-TMA and plans to initiate a Phase 3 study in the first half of 2021.
ULTOMIRIS – Severe COVID-19: In January 2021, Alexion paused further enrollment in a Phase 3 trial of ULTOMIRIS in adults hospitalized with severe COVID-19 requiring mechanical ventilation, due to lack of efficacy, pending further analysis of the data. This decision was made based on the recommendation of an independent data monitoring committee, following their review of data from a pre-specified interim analysis. Alexion continues to provide ULTOMIRIS for the ongoing TACTIC-R platform study led by Cambridge University Hospitals NHS Foundation Trust, which is evaluating the potential of earlier immune modulatory treatment (hospitalized patients not requiring mechanical ventilation) in preventing progression of the virus.
ULTOMIRIS – Dermatomyositis (DM): Alexion plans to initiate a Phase 2/3 study of ULTOMIRIS in DM in the second half of 2021, pending regulatory feedback.
ALXN1840 – Wilson Disease: Enrollment is complete in a Phase 3 study of ALXN1840 in Wilson disease. Study results are expected in the first half of 2021.
CAEL-101 – Caelum Biosciences: Alexion and Caelum Biosciences are conducting the Cardiac Amyloid Reaching for Extended Survival (CARES) Phase 3 clinical program to evaluate CAEL-101, a first-in-class amyloid fibril targeted therapy, in combination with standard-of-care therapy in AL amyloidosis. Dosing is underway in two parallel Phase 3 studies – one in patients with Mayo stage IIIa disease and one in patients with Mayo stage IIIb disease.
ALXN2060 (AG10): Alexion holds an exclusive license to develop and commercialize ALXN2060 (AG10) in Japan. Eidos is currently evaluating AG10 in two Phase 3 studies in the U.S. and Europe – one for ATTR cardiomyopathy (ATTR-CM) and one for ATTR polyneuropathy (ATTR-PN). In October 2020, Alexion initiated a Phase 3 bridging study of ALXN2060 for patients with ATTR-CM in Japan and dosing is underway.
ALXN2040 (Danicopan) – PNH with Extravascular Hemolysis (EVH): In December 2020, Alexion initiated a Phase 3 study of ALXN2040 as an add-on therapy for PNH patients with EVH and dosing is underway.
ANDEXXA – Acute Intracranial Hemorrhage (ICH): The Phase 4 ANNEXA-I study – designed to provide clinical data supporting full approval – is underway to assess ANDEXXA compared to usual standard of care in patients presenting with acute intracranial hemorrhage while taking an oral Factor Xa inhibitor. In December 2020, Alexion submitted a supplemental Biologics License Application (sBLA) to the U.S. FDA to enable the addition of edoxaban and enoxaparin to the U.S. label. Alexion plans to file for regulatory approval of ANDEXXA in Japan in the first quarter of 2021.
PHASE 1/2
ULTOMIRIS – Renal Diseases: In November 2020, Alexion initiated a proof-of-concept study of ULTOMIRIS in patients with IgA nephropathy and lupus nephritis.
ALXN1830: Due to COVID-19, Alexion discontinued the Phase 2 study of ALXN1830, administered intravenously, in warm autoimmune hemolytic anemia (WAIHA) and the Phase 1 study of a subcutaneous formulation of ALXN1830 in healthy volunteers. In January 2021, a Clinical Trial Application (CTA) for a new Phase 1 study of subcutaneous ALXN1830 was approved in New Zealand; study initiation is planned for the first quarter of 2021. Following successful completion of the Phase 1 study, Alexion plans to initiate Phase 2 studies of subcutaneous ALXN1830 in gMG and WAIHA in 2021, pending regulatory feedback.
ALXN2040 – Geographic Atrophy (GA): Alexion plans to initiate a Phase 2 study of ALXN2040 in GA in the second half of 2021.
ALXN2040 – COVID-19: Alexion has agreed to provide ALXN2040 to the U.S. National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, for the ACTIV-5 Big Effect Trial in adults hospitalized with COVID-19. This Phase 2 platform trial is comparing different investigational therapies to a common control arm with the intent of identifying promising treatments to enter a more definitive study.
ALXN2050 – PNH: Alexion has paused additional enrollment in the Phase 2 study of ALXN2050 monotherapy in PNH patients, pending the receipt of further Phase 1 data (expected in the second quarter of 2021) that will allow for dose escalation in the Phase 2 study.
ALXN2050 – Renal Diseases: Alexion plans to initiate a proof-of-concept study of ALXN2050 in patients with various renal diseases in 2021, pending regulatory feedback.
ALXN1720: The Phase 1 healthy volunteer study of ALXN1720, a novel anti-C5 albumin-binding bi-specific mini-body that is designed to bind and prevent activation of human C5, has been paused for a second time due to COVID-19. Additional cohorts have been added to the study to explore higher doses and enable the initiation of a Phase 3 study in gMG, pending successful completion of the Phase 1 study as has been agreed with the FDA. Data from the Phase 1 study are expected in the second half of 2021. Alexion also plans to initiate a study of ALXN1720 in DM.
ANDEXXA – Urgent Surgery: ANDEXXA is currently being evaluated in a single-arm, open-label Phase 2 study in patients taking apixaban, rivaroxaban, edoxaban, or enoxaparin who require urgent surgery. The results of this study will inform the design of a randomized controlled Phase 3 clinical trial to expand the label in this population.
ALXN2075 (cerdulatinib): Acquired as part of the Portola acquisition, ALXN2075 is a dual spleen tyrosine kinase and janus kinase (SYK/JAK) inhibitor being evaluated in a Phase 1/2a study in patients with relapsed/refractory chronic lymphocytic leukemia or B-cell or T-cell non-Hodgkin lymphoma. Data are expected in the first half of 2021.
ALXN1820: In January 2021, Alexion initiated a Phase 1 study of ALXN1820, its bi-specific anti-properdin mini-body, in healthy volunteers.
ALXN1850 – Hypophosphatasia (HPP): In November 2020, Alexion submitted an IND application to the FDA for ALXN1850, its next generation asfotase alfa asset. Initiation of a Phase 1 study in adults with HPP is planned for the second quarter of 2021.
Conference Call/Earnings Materials:
Given the recently announced agreement for Alexion to be acquired by AstraZeneca, Alexion will not be hosting a conference call. Earnings materials are available publicly on the Investor Relations page of our website at View Source Questions may be directed to the Investor Relations team via e-mail at [email protected] or the contact information below.