On February 22, 2022 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and financial results for the fourth quarter and full year ending December 31, 2021 (Press release, PTC Therapeutics, FEB 22, 2022, View Source [SID1234608838]).

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"It is gratifying to see that after two years of investment in the innovation revenue cycle we see substantial progress across the robust pipeline and commercial revenues exceeding half a billion dollars," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "2022 is expected to be a transformational year for PTC and I look forward to continued growth."

Key 2021 Corporate Updates:

Strong revenue growth continued in the Duchenne muscular dystrophy (DMD) franchise, with total net product revenue of $423 million for Translarna (ataluren) and Emflaza (deflazacort) in 2021.
Translarna total net product revenue of $236 million was due to treatment of new patients, continued high compliance, and geographic expansion.
Emflaza total net product revenue of $187 million was driven by continued new prescriptions, continued high compliance, and more favorable access.
Evrysdi (risdiplam) 2021 revenue of over $500 million resulted in a $25 million sales-based milestone to PTC. Evrysdi is now the most prescribed Spinal Muscular Atrophy (SMA) therapy in the U.S. and growth continues in markets outside of the U.S. Evrysdi is a product of the SMA collaboration between PTC, the SMA Foundation and Roche.
Waylivra (volanesorsen) and Tegsedi (inotersen) both received Category 1 classification from Câmara de Regulação do Mercado de Medicamentos – CMED (Drug Market Regulation Chamber) in Brazil. This allows for pricing in line with international markets.
PTC submitted an application to the Brazilian Health Regulatory Agency, Agência Nacional de Vigilância Sanitária (ANVISA), for approval of Waylivra for the treatment of familial partial lipodystrophy (FPL). If approved, Waylivra would be the first treatment in the world approved for FPL.
PTC successfully advanced the clinical pipeline in 2021:
Phase 1 healthy volunteer trial of PTC518, the next compound from the validated splicing platform, which is being developed for Huntington’s disease (HD).
Phase 1 healthy volunteer trial of PTC857, the second compound from the Bio-e platform, which is being developed for amyotrophic lateral sclerosis (ALS).
Phase 1b studies of unesbulin in both leiomyosarcoma and diffuse intrinsic pontine glioma (DIPG), two rare oncology indications.
Enrollment is complete in the MOVE-FA registration-directed trial of vatiquinone in Friedreich ataxia, with results expected in the second quarter of 2023.
2022 Potential Value-Creating Milestones:

Results are expected mid-year for Translarna Study 041.
Results are expected from the Phase 3 APHENITY trial of PTC923 in phenylketonuria (PKU) by the end of 2022.
Results from the Phase 2/3 MIT-E study of vatiquinone in mitochondrial disease associated seizures are expected in the fourth quarter of 2022.
An opinion on our marketing authorization application (MAA) for PTC-AADC is expected in April 2022 from the European Medicine Agency’s Committee for Medicinal Products for Human Use (CHMP).
PTC remains on track to submit the biologics license application for PTC-AADC to the Food and Drug Administration in the second quarter of 2022.
PIVOT-HD, the Phase 2 study of PTC518 in HD is expected to initiate in the first quarter of 2022.
The CardinALS registration-directed Phase 2 study of PTC857 in ALS is expected to initiate in the second quarter of 2022.
Fourth Quarter and Full-Year 2021 Financial Highlights:

Total revenues were $165.2 million for the fourth quarter of 2021, compared to $118.9 million for the fourth quarter of 2020. Total revenues were $538.6 million for the full year 2021, compared to $380.8 million for the full year 2020.
Total revenue includes net product revenue across the commercial portfolio of $118.9 million for the fourth quarter of 2021 and $428.9 million for full year 2021, compared to $107.3 million for the fourth quarter of 2020 and $333.4 million for full year 2020. Total revenue also includes collaboration and royalty revenue of $46.3 million in the fourth quarter of 2021 and $109.7 million for the full year 2021, compared to $11.6 million for the fourth quarter of 2020 and $47.4 million for the full year 2020.
Translarna net product revenues were $69.7 million for the fourth quarter of 2021, compared to $69.4 million for the fourth quarter of 2020. Translarna net product revenues were $236.0 million for the full year 2021, compared to $191.9 million for the full year 2020. These results were driven by treatment of new patients, continued high compliance, and geographic expansion.
Emflaza net product revenues were $47.5 million for the fourth quarter of 2021, compared to $36.8 million for the fourth quarter of 2020. Emflaza net product revenues were $187.3 million for the full year 2021, compared to $139.0 million for the full year 2020. These results were driven by continued new prescriptions, continued high compliance, and more favorable access.
Roche reported Evrysdi full year 2021 sales of approximately CHF 602 million, resulting in full year 2021 royalty revenue of $54.6 million to PTC, as compared to $4.8 million for the full year 2020. In the first quarter of 2021 the first commercial sale of Evrysdi in the European Union triggered a $20 million milestone payment to PTC, in the third quarter of 2021 the first commercial sale of Evrysdi in Japan triggered a $10 million milestone payment from Roche to PTC, and in the fourth quarter of 2021 PTC recorded its first sales milestone of $25 million for the achievement of $500 million in worldwide annual net sales from Evrysdi. All of these 2021 achievements were reported as collaboration revenue. For the full the year 2020, the acceptance of the MAA filed by Roche for Evrysdi for the treatment of SMA triggered a $15 million milestone payment to PTC and the first commercial sale of Evrysdi in the U.S. triggered a $20 million milestone payment to PTC. Additionally, in October 2020, Chugai Pharmaceutical, Co. Ltd, a member of the Roche group, announced that a new drug application for Evrysdi for the treatment of SMA was filed in Japan. The filing in Japan triggered a $7.5 million milestone payment to PTC in the fourth quarter of 2020. PTC recognized $25 million of collaboration revenue associated with Roche milestone events in the fourth quarter of 2021 and $55 million for the full year 2021, as compared to $7.5 million in the fourth quarter of 2020 and $42.6 million for the full year 2020.
Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $149.8 million for the fourth quarter of 2021, compared to $118 million for the fourth quarter of 2020. GAAP R&D expenses were $540.7 million for the full year 2021, compared to $477.6 million for the full year 2020. The increase reflects additional investment in research programs and advancement of the clinical pipeline.
Non-GAAP R&D expenses were $136.4 million for the fourth quarter of 2021, excluding $13.4 million in non-cash, stock-based compensation expense, compared to $105.3 million for the fourth quarter of 2020, excluding $12.7 million in non-cash, stock-based compensation expense. Non-GAAP R&D expenses were $487.1 million for the full year 2021, excluding $53.6 million in non-cash, stock-based compensation expense, compared to $438.9 million for the full year 2020, excluding $38.7 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $86.5 million for the fourth quarter of 2021, compared to $75.5 million for the fourth quarter of 2020. GAAP SG&A expenses were $285.8 million for the full year 2021, compared to $245.2 million for the full year 2020. The increase reflects our continued investment to support commercial activities including expanding our commercial portfolio.
Non-GAAP SG&A expenses were $73.7 million for the fourth quarter of 2021, excluding $12.8 million in non-cash, stock-based compensation expense, compared to $66.8 million for the fourth quarter of 2020, excluding $8.7 million in non-cash, stock-based compensation expense. Non-GAAP SG&A expenses were $235.9 million for the full year 2021, excluding $49.9 million in non-cash, stock-based compensation expense, compared to $213.6 million for the full year 2020, excluding $31.6 million in non-cash, stock-based compensation expense.
Change in the fair value of deferred and contingent consideration was a gain of $12.1 million for the fourth quarter of 2021, compared to a loss of $6.3 million for the fourth quarter of 2020. Change in the fair value of deferred and contingent consideration was a gain of $0.5 million for the full year 2021, compared to a loss of $23.3 million for the full year 2020. The change in fair value of deferred and contingent consideration is related to the fair valuation of potential future consideration to be paid to former equity holders of Agilis Biotherapeutics, Inc. (Agilis) in connection with PTC’s acquisition of Agilis, which closed in August 2018.
Settlement of deferred and contingent consideration was $10.6 million for the full year 2020. The settlement of deferred and contingent consideration is related to a loss upon the settlement of the deferred and contingent consideration liabilities as a result of the rights exchange agreement with certain former shareholders of Agilis, whereby such former shareholders exchanged their pro rata share of specific future cash milestone payments in the aggregate amount of $225 million for a mixture of cash and equity of PTC. Under this agreement, which the former shareholders and PTC entered into on April 29, 2020, PTC has paid $36.9 million in cash and issued 2,821,176 shares of common stock in exchange for the cancellation and forfeiture of the participating shareholders’ rights to receive (i) $174.0 million, in the aggregate, of potential milestone payments based on the achievement of certain regulatory milestones and (ii) $37.6 million, in the aggregate, of $40.0 million in development milestone payments that would have been due upon the passing of the second anniversary of the closing of PTC’s merger with Agilis, regardless of whether the milestones are achieved.
Net loss was $143.3 million for the fourth quarter of 2021, compared to net loss of $74.4 million for the fourth quarter of 2020. Net loss was $523.9 million for the full year 2021, compared to net loss of $438.2 million for the full year 2020.
Cash, cash equivalents, and marketable securities were $773.4 million on December 31, 2021, compared to $1.1 billion at December 31, 2020.
Shares issued and outstanding as of December 31, 2021 were 70,828,226.
PTC Reaffirms 2022 Financial Guidance:

PTC anticipates total revenues for the full year 2022 to be between $700 and $750 million.
PTC anticipates net product revenues for the DMD franchise for the full year 2022 to be between $475 and $495 million.
PTC anticipates GAAP R&D and SG&A expense for the full year 2022 to be between $915 and $965 million.
PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2022 to be between $800 and $850 million, excluding estimated non-cash, stock-based compensation expense of $115 million.
Non-GAAP Financial Measures:

In this press release, the financial results and financial guidance of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management’s opinion, these non-GAAP financial measures are useful to investors and other users of PTC’s financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company’s future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the tables below.

Today’s Conference Call and Webcast Reminder:

PTC will host a conference call to discuss the fourth quarter and full year of 2021 corporate updates and financial results today at 4:30 pm ET and can be access by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 6884452. A live, listen-only webcast of the conference call can be accessed on the investor relations section of the PTC website at www.ptcbio.com. A webcast replay of the call will be available approximately two hours after completion of the call and will be archived on the company’s website for 30 days following the call.

On February 22, 2022 Fresenius Medical Care, reported Throughout 2021, COVID-19 has significantly impacted our patients’ and employees’ lives as well as Fresenius Medical Care as a company (Press release, Fresenius, FEB 22, 2022, View Source [SID1234608787]). Although excess mortality among our patients in the fourth quarter has moderated, the accumulated impact of COVID-19 weighed heavier on our earnings development than we had anticipated in the beginning of 2021. Given these challenging headwinds, we are proud that we have continuously been there for our patients and, on the back of this, were able to deliver on our 2021 guidance. Despite the ongoing impact of the pandemic and an increasingly inflationary cost environment, we target to return to earnings growth this year. At the same time, we will continue to execute on the key priorities of our Strategy 2025, advancing on our transformational FME25 program as well as progressing on our Sustainability agenda."

1 2021: costs related to the FME25 program; 2020: impairment of goodwill and trade names in the Latin America Segment
2 Net income attributable to shareholders of Fresenius Medical Care AG & Co. KGaA

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Implementing strategic priorities

Based on its Strategy 2025, Fresenius Medical Care aims to capture further growth potential along the Renal Care Continuum and beyond – by leveraging its core competencies and offering sustainable solutions with innovative products and services of the highest quality and at reliable costs. To this end, the company is continuing to steadily drive forward its strategic priorities.
Fresenius Medical Care aims to build sustainable partnerships with payors worldwide to support the transition from a fee-for-service to a pay-for-performance system. In Value-based Care models, the Company contributes to creating medical value while ensuring that care remains affordable.

Fresenius Medical Care´s longstanding experience in Value-based Care and the largest database of CKD (Chronic Kidney Disease) patients in the industry enables the use of proprietary predictive models to slow kidney disease progression and reduce hospital admissions. In 2022, the Company expects to grow the number of its ESRD (End-Stage Renal Disease) and CKD patients receiving care in Value-based Care arrangements from more than 20,0000 patients at the end of 2021 to around 80,000. The significant increase compared to the previous year will be mainly driven by the start on 1 January 2022 of the Comprehensive Kidney Care Contracting options (CKCC) of the Centers for Medicare and Medicaid Services (CMS) in the U.S. Across all Value-based Care arrangements, Fresenius Medical Care expects to be managing risk for more than USD 6 billion in medical expenses in 2022.

To further enhance the quality of life for its patients and increase their choice of available treatment options, Fresenius Medical Care remains committed to further expanding its home dialysis offerings. In 2021, the Company provided around 15% of its dialysis treatments in the U.S. in a home setting and has thus already achieved its target originally set for 2022.
Based on its strategic business planning, Fresenius Medical Care has set a new aspirational target for the expansion of home dialysis: By 2025, the Company aims to perform 25% of all treatments in the U.S. at a patient’s home.

Fresenius Medical Care’s commitment to Sustainability is not only incorporated in its vision and mission but also reflected in its strategy. To shape operations, integrate sustainability principles into business activities and assume even greater accountability, the Company is consistently advancing its global sustainability activities. In 2021, Fresenius Medical Care reached further important milestones in focus areas such as patients and employees, environmental protection, combating bribery and corruption, and respect for human rights. More than ever, Fresenius Medical Care continues to actively drive progress of its Sustainability agenda.

As part of the FME25 program, Fresenius Medical Care is transforming its operating model into a simplified structure with two global operating segments – Care Enablement and Care Delivery, thus providing the basis for the acceleration of innovation for our patients and future sustainable profitable growth.

Helen Giza, Chief Financial Officer and Chief Transformation Officer of Fresenius Medical Care, said: "We continue to move at pace on the overall implementation and execution of our transformation program, focusing on unlocking shareholder value, creating the basis for future sustainable profitable growth and shaping the organization to the new operating model. An important step in that direction was the recent naming of the next level of leaders in the new organization."

The Company is committed to implementing necessary changes in a socially responsible way, and to following applicable consultation procedures with works councils and other workplace representative bodies in good faith.

Fresenius Medical Care expects to implement its new global operating model around 2023 and the savings initiative to be largely completed by 2024. With the transformation of the operating model, the annual cost base shall be reduced by EUR 500 million by the end of 2025. One-time costs of FME25 are expected to amount to approximately EUR 450-500 million. In 2021, costs incurred for FME25 amounted to EUR 63 million in the program.

For 2022, the Company expects FME25-related one-time costs of approximately EUR 175 to 245 million and has included EUR 40 to 70 million of sustainable EBIT savings in its outlook.

Decrease in COVID-19-related excess mortality

Despite the global spread of the Omicron variant, which resulted in a significant increase in infection rates in many countries, COVID-19-related excess mortality among Fresenius Medical Care’s patients decreased in the fourth quarter of 2021 and amounted to around 1,800 (Q1 2021: ~3,200; Q2 2021: ~1,900; Q3 2021: ~2,9003). Thus, excess mortality accumulated to approximately 9,800 patients in FY 2021 and approximately 20,100 since the start of the pandemic.

The overall estimated adverse effect of accumulated COVID-19-related excess mortality on organic growth in the Health Care Services business amounted to around 290 basis points in the fourth quarter and 280 basis points in the full year 2021.

As of year-end 2021, approximately 81% (U.S.: ~80%) of Fresenius Medical Care’s patients had been at least partially vaccinated; 58% (U.S.: ~57%) of all fully vaccinated patients had already received a booster vaccination.

3 Historical excess mortality updated for late entries

Revenue and earnings development affected by COVID-19

Revenue in the fourth quarter increased by 6% to EUR 4,647 million (+3% at constant currency, +2% organic).

Health Care Services revenue increased by 6% to EUR 3,621 million (+3% at constant currency, +2% organic). This was mainly driven by a positive exchange rate effect as well as organic growth, which was achieved despite the adverse impact of COVID-19 and a lower reimbursement for calcimimetics.

Health Care Products revenue increased by 3% to EUR 1,026 million (+1% at constant currency, +1% organic), mainly driven by a positive exchange rate effect as well as higher sales of machines for chronic treatment, home hemodialysis products and in-center disposables, partially offset by lower sales of products for acute care treatments.

In the full year, revenue declined by 1% to EUR 17,619 million (+2% at constant currency, +1% organic). Health Care Services revenue decreased by 2% to EUR 13,876 million (+2% at constant currency, +1% organic), mainly due to a negative exchange rate effect, partially offset by organic growth, which was achieved despite COVID-19 and a lower reimbursement for calcimimetics, as well as higher contributions from acquisitions. Health Care Products revenue remained stable and amounted to EUR 3,743 million (+2% at constant currency, +2% organic). Higher sales of machines for chronic treatment, home hemodialysis products and renal pharmaceuticals were offset by a negative exchange rate effect and lower sales of products for acute care treatments.

Operating income decreased by 3% to EUR 449 million in the fourth quarter (-7% at constant currency), resulting in a margin of 9.7% (Q4 2020: 10.5%). Operating income excluding special items, i.e. the impairment of goodwill and trade names in the Latin America Segment in 2020 and costs incurred for FME25 in 2021, declined by 25% to EUR 492 million (-28% at constant currency), resulting in a margin of 10.6% (Q4 2020: 14.9%). The decline was mainly due to a remeasurement effect on the fair value of investments, higher labor costs, the adverse COVID-19-related net effects and inflationary materials cost increases. These effects were only slightly mitigated by an improved U.S. payor mix, in particular due to an increased number of patients with Medicare Advantage coverage.

In the full year, operating income decreased by 20% to EUR 1,852 million (-17% at constant currency), resulting in a margin of 10.5% (FY 2020: 12.9%). Operating income excluding special items declined by 23% to EUR 1,915 million (-21% at constant currency), resulting in a margin of 10.9% (FY 2020: 14.0%). This was mainly due to adverse COVID-19-related net effects, inflationary materials cost increases, higher labor costs, and the remeasurement effect on the fair value of investments. These effects were slightly mitigated by an improved U.S. payor mix, in particular due to an increased number of patients with Medicare Advantage coverage.

Net income2 increased by 29% to EUR 229 million in the fourth quarter (+23% at constant currency). Excluding special items, net income declined by 29% to EUR 263 million (-32% at constant currency), mainly due to the mentioned negative effects on operating income.

In the full year, net income decreased by 17% to EUR 969 million (-14% at constant currency). Net income excluding special items declined by 25% to EUR 1,018 million (-23% at constant currency).

Basic earnings per share (EPS) increased by 29% to EUR 0.78 (+23% at constant currency) in the fourth quarter. EPS excluding special items declined by 29% to EUR 0.90 (-32% at constant currency).

In the full year, EPS decreased by 16% to EUR 3.31 (-14% at constant currency). EPS excluding special items declined by 25% to EUR 3.48 (-23% at constant currency).

Cash flow development

In the fourth quarter, Fresenius Medical Care generated EUR 669 million of operating cash flow (Q4 2020: EUR 584 million), resulting in a margin of 14.4% (Q4 2020: 13.3%). The increase was mainly due to improved working capital including contributions from FME25 and U.S. federal relief funding, partially offset by continued recoupment of the U.S. government’s payments received in 2020 under the CARES Act and lower tax payments related to COVID-19 reliefs in the prior year. In the full year, operating cash flow amounted to EUR 2,489 million (FY 2020: EUR 4,233 million), resulting in a margin of 14.1% (FY 2020: 23.7%). The decline was mainly due to the U.S. government’s advanced payments received in 2020, the partial recoupment of these payments in 2021 as well as other COVID-19 relief, including lower tax payments in North America segment in the previous year.

Fresenius Medical Care generated EUR 400 million of free cash flow4 (Q4 2020: EUR 283 million) in the fourth quarter, resulting in a margin of 8.6% (Q4 2020: 6.4%). In the full year, free cash flow amounted to EUR 1,660 million (FY 2020: EUR 3,197 million), resulting in a margin of 9.4% (FY 2020: 17.9%).

4 Net cash provided by / used in operating activities, after capital expenditures, before acquisitions, investments, and dividends

Regional developments

In North America, revenue increased by 6% to EUR 3,156 million in the fourth quarter (+1% at constant currency, 0% organic). A positive exchange rate effect was largely offset by the adverse COVID-19 impact on both the Health Care Services and Health Care Products businesses along with associated downstream effects. In the full year, revenue decreased by 3% to EUR 12,088 million (stable at constant currency, 0% organic).

Operating income in North America decreased by 25% to EUR 402 million in the fourth quarter (-29% at constant currency), resulting in a margin of 12.7% (Q4 2020: 17.9%). The decline was mainly due to a remeasurement effect on the fair value of investments, higher personnel expense, the adverse net impact of COVID-19 and costs related to FME25. This was only partially offset by an improved U.S. payor mix, in particular due to an increased number of patients with Medicare Advantage coverage. In the full year, operating income declined by 22% to EUR 1,644 million (-20% at constant currency), resulting in a margin of 13.6% (FY 2020: 17.0%).

Revenue in the EMEA region increased by 2% to EUR 732 million in the fourth quarter (+2% at constant currency, +2% organic), mainly driven by organic growth in the Health Care Services business, which was achieved despite the negative impact of COVID-19. In the full year, revenue remained stable and amounted to EUR 2,765 million (+1% at constant currency, +1% organic).
Operating income in EMEA decreased by 42% to EUR 77 million in the fourth quarter (-42% at constant currency), resulting in a margin of 10.6% (Q4 2020: 18.7%). The decline was mainly due to a favorable impact from equity method investees in the previous year, inflationary cost increases, adverse COVID-19-related effects and costs related to FME25. In the full year, operating income decreased by 25% to EUR 309 million (-25% at constant currency), resulting in a margin of 11.2% (FY 2020: 14.9%).

In Asia-Pacific, revenue increased by 7% to EUR 552 million in the fourth quarter (+5% at constant currency, +4% organic). This was mainly driven by organic growth in both the Health Care Services and Health Care Products business as well as a positive exchange rate effect. In the full year, revenue grew by 6% to EUR 2,010 million (+7% at constant currency, +7% organic), thus exceeding EUR 2 billion for the first time.

Operating income decreased by 12% to EUR 94 million in the fourth quarter (-12% at constant currency), resulting in a margin of 17.0% (Q4 2020: 20.6%). The decline was mainly due to inflationary cost increases and adverse COVID-19-related effects. In the full year, operating income grew by 2% to EUR 350 million (+3% at constant currency), resulting in a margin of 17.4% (FY 2020: 18.1%).

Despite a significant headwind from exchange rates and the adverse impact of COVID-19, Latin America revenue increased by 10% to EUR 195 million in the fourth quarter (+17% at constant currency, +20% organic). This was mainly driven by strong organic growth in both the Healthcare Services and Healthcare Products business. In the full year, revenue grew by 3% to EUR 703 million (+16% at constant currency, +15% organic).

Due to the impairment of goodwill and trade names in the Latin America Segment that impacted earnings in the previous year, operating income for the region improved by 99% to EUR -2 million in the fourth quarter (+99% at constant currency), resulting in a margin of -0.8% (Q4 2020: -105.0%). In the full year, operating income increased to EUR 12 million, resulting in a margin of 1.7%.

Patients, clinics and employees

As of December 31, 2021, Fresenius Medical Care treated 345,425 patients in 4,171 dialysis clinics worldwide. At the end of 2021, the Company had 122,909 employees (full-time equivalents) worldwide, compared to 125,364 employees as of December 31, 2020.
 
25th consecutive dividend increase to be proposed

Consistent with Fresenius Medical Care’s commitment to drive shareholder returns while striving for dividend continuity, the Company proposes a dividend of EUR 1.35 per share to the Annual General Meeting in May 2022. This proposal would result in the 25th consecutive dividend increase. The Company believes that the fundamental drivers of its business and growth remain unchanged, despite the unprecedented but temporary effects of the COVID-19 pandemic.

Outlook

Fresenius Medical Care expects revenue and net income to grow at low to mid-single digit percentage rates in FY 2022.5
These targets are based on the following assumptions:

COVID-19
accumulated excess mortality to impact operating income by EUR 100 million compared to the level of 2021
staff shortages are anticipated not to cause significant disruptions in production, distribution and dialysis operations
Macro-economic inflation and supply chain costs to impact operating income by EUR 50 million
Labor costs for 2022 are expected to be around EUR 100 million in excess of the 3% base wage inflation assumption
Any potential further government support is assumed to be applied to manage the unprecedented labor market situation if costs exceed the above labor costs assumption.
FME25 savings are expected to contribute EUR 40 to 70 million to operating income
Remeasurement effects on the fair value of investments are expected to be volatile but neutral on a full year basis
Based on current projections, Fresenius Medical Care confirms its mid-term targets that are based on the Company’s mid-term strategy. Until 2025 the Company expects compounded annual average increases in the mid-single-digit percentage range for revenue and in the high-single-digit percentage range for net income.6 Fresenius Medical Care expects FME25 savings to mitigate the ongoing negative effects of COVID-19.

5 These targets are based on the 2021 results excluding the costs related to FME25 of EUR 49 million (for Net Income). They are based on the outlined assumptions, in constant currency and exclude special items. Special items include further costs related to FME25 and other effects that are unusual in nature and have not been foreseeable or not foreseeable in size or impact at the time of giving guidance.

6 These targets are in constant currency and exclude special items.

Press conference

Fresenius Medical Care will hold a virtual press conference to discuss the results of the fourth quarter and full year 2021 on February 22, 2022 at 10:00 a.m. CET / 4:00 a.m. ET. The press conference will be webcasted on the Company’s website www.freseniusmedicalcare.com in the "Media" section. A replay will be available shortly after the conference.

Conference call

Fresenius Medical Care will host a conference call to discuss the results of the fourth quarter and full year 2021 on February 22, 2022 at 3:30 p.m. CET / 9:30 a.m. ET. Details will be available on the Fresenius Medical Care website www.freseniusmedicalcare.com in the "Investors" section. A replay will be available shortly after the call.

Please refer to our statement of earnings included at the end of this news and to the attachments as separate PDF files for a complete overview of the results of the fourth quarter and full year 2021. Our 20-F disclosure provides more details.

On February 22, 2022 NuCana plc (NASDAQ: NCNA) reported it has completed enrollment of the number of patients in the ongoing Phase III NuTide:121 study required to conduct the second interim analysis, which is expected to occur in the second half of 2022 after the 644th patient has completed 28 weeks of follow-up (Press release, Nucana BioPharmaceuticals, FEB 22, 2022, View Source [SID1234608805]). The NuTide:121 study is comparing Acelarin combined with cisplatin to the global standard of care, gemcitabine plus cisplatin, as a first-line treatment for patients with advanced biliary tract cancer. As previously announced, NuCana expects to conduct the first interim analysis after the 418th patient has completed 28 weeks of follow-up, which is expected to occur in the first half of 2022.

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NuCana believes that a statistically significant improvement in the Objective Response Rate (ORR) at either of the first two interim analysis, accompanied by positive trends in other endpoints, has the potential to allow for accelerated approval of a new drug application (NDA) for Acelarin in the United States. At the first interim analysis, an improvement in ORR of approximately 14% or more in the Acelarin plus cisplatin arm compared to the gemcitabine plus cisplatin arm would be statistically significant. Due to the larger number of patients included in the second interim analysis, an improvement in ORR of approximately 9% or more would be statistically significant. Recruitment in the NuTide:121 study, which is intended to enroll up to 828 patients, is ongoing and NuCana believes subsequent analyses could provide the confirmatory data to support full (regular) approval.

"We are delighted with the pace of enrollment in the NuTide:121 study especially against the backdrop of the Covid pandemic," said Hugh S. Griffith, NuCana’s Founder and Chief Executive Officer. "With the enrollment of the 644th evaluable patient, we are now in position to complete two pre-specified interim analyses in 2022."

Mr. Griffith continued: "In the ABC-08 study of first-line patients with biliary tract cancer, Acelarin plus cisplatin achieved an ORR of 44% among the evaluable population. This compared favorably to the ORR of 26% achieved among evaluable patients treated with gemcitabine plus cisplatin in the ABC-02 study, which established this regimen as the global standard of care. Biliary tract cancer is a devastating disease and we believe Acelarin could represent a new option to help address the significant unmet need for more effective medicines."

About NuTide:121

NuTide:121 is a global, multi-center, 1:1 randomized Phase 3 study comparing Acelarin, a ProTide transformation of gemcitabine, in combination with cisplatin, to gemcitabine in combination with cisplatin in up to 828 patients with advanced biliary tract cancer who have not previously received treatment for advanced disease. The primary endpoints of NuTide:121 are Overall Survival (OS) and Objective Response Rate (ORR) and the FDA-approved protocol includes three interim analyses. Based on the statistical analysis plan, and subject to any further regulatory guidance, the Company believes that a statistically significant improvement in ORR at either of the first two interim analyses, accompanied by positive trends in other endpoints, has the potential to allow for an accelerated approval of a new drug application (NDA) for Acelarin in the United States. Under this scenario, the NuTide:121 study would continue and the Company believes it could use the data from subsequent analyses as the confirmatory data required to support full (regular) approval. There are currently no agents approved for the first-line treatment of patients with biliary tract cancer.

About Biliary Tract Cancer

Biliary tract cancer, including cholangiocarcinoma, gallbladder and ampullary carcinoma, are a group of cancers originating in the biliary tract. The biliary tract is comprised of the gallbladder and interconnecting ducts responsible for the transport of bile from the liver to the gallbladder and small intestine. Approximately 178,000 new cases of biliary tract cancer are diagnosed each year worldwide, with more than 16,000 of those diagnoses in the United States. There are currently no agents approved for the first-line treatment of patients with advanced biliary tract cancer; however, the worldwide standard of care in these patients is the combination of gemcitabine and cisplatin. Patients receiving this regimen have a median overall survival of 11.7 months.

On February 22, 2022 Poseida Therapeutics, Inc. (NASDAQ: PSTX), a clinical-stage biopharmaceutical company utilizing proprietary gene engineering platform technologies to create cell and gene therapeutics with the capacity to cure, reported that Brent Warner has joined the Company as President, Gene Therapy effective February 21, 2022 (Press release, Poseida Therapeutics, FEB 22, 2022, View Source [SID1234608823]).

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"Brent brings deep pharmaceutical expertise and a strong track record of leadership in gene therapy and rare genetic diseases to Poseida, and I am excited to welcome him to our team," said Mark Gergen, Chief Executive Officer of Poseida. "Alongside our top scientists, he will lead the execution of drug development programs in gene therapy, including our recent research collaboration with Takeda. It’s an exciting time at Poseida, and I look forward to working with Brent as we continue to advance our innovative gene therapy programs."

Mr. Warner has more than 15 years of biotech and pharmaceutical experience, most recently serving as Vice President, Gene Therapy and Rare Disease at Novartis, where he was responsible for building a division to commercialize multiple gene therapies overseeing three therapeutic areas. Prior roles included serving as a U.S. commercial leader in Hemophilia A at BioMarin Pharmaceutical Inc. and a variety of strategic operational roles at Biogen. Before that, he held commercial launch roles in blood disorders at Baxalta, then a biopharmaceutical division spun off by Baxter International and now a wholly owned subsidiary of Takeda. Warner has an MBA with an emphasis in marketing and finance and a Bachelor of Business Administration degree, both from Northwood University in Midland, Mich.

"I am thrilled to join Poseida, which has built an unparalleled platform to deliver novel cell and gene therapies for patients with high unmet need," Warner said. "Together with Poseida’s outstanding team, I look forward to the opportunity to accelerate potential single-treatment cures for the rare disease community."

On February 22, 2022 Halozyme Therapeutics, Inc. (NASDAQ: HALO) reported financial results for the fourth quarter and full year ended December 31, 2021, and provided an update on its recent corporate activities and outlook (Press release, Halozyme, FEB 22, 2022, View Source [SID1234608839]).

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"Our performance in the fourth quarter closed out a year of strong performance across our business highlighted by record full year revenue from royalties, growing 130% from last year driven by the continued adoption of our wave 2 partner products subcutaneous DARZALEX and Phesgo (pertuzumab, trastuzumab, and hyaluronidase-zzxf)," said Dr. Helen Torley, president and chief executive officer. "In 2022, we look forward to building on 2021 momentum and anticipate 50% growth in our high-margin, recurring royalty revenues driven by further adoption of wave 2 products. We project further expansion and maturing of our pipeline, with 4 new targets entering Phase 1 development and at least 5 products advancing to Phase 2 or Phase 3 development. We are also excited to be starting development of a higher yield ENHANZE API as well as a new rHuPH20 with extended room temperature stability and patent protection to 2032 in Europe and 2034 in the U.S."

Fourth Quarter Partner Highlights:

In December 2021, Janssen received FDA approval for DARZALEX FASPRO (daratumumab and hyaluronidase – fijh) in combination with Kyprolis (carfilzomib) and dexamethasone for patients with relapsed or refractory multiple myeloma who have received one to three prior lines of therapy.

In December 2021, ViiV initiated enrollment of a Phase 1 study arm to evaluate cabotegravir administered subcutaneously with ENHANZE.

In December 2021, Janssen initiated a Phase 1 clinical trial combining Janssen’s rilpivirine and ENHANZE.

In November 2021, Roche initiated a Phase 1 study combining an undisclosed therapy with ENHANZE for a new exclusive ENHANZE target.

In October 2021, Janssen’s DARZALEX FASPRO was approved by the China National Medical Products Administration for the treatment of primary light chain amyloidosis, in combination with bortezomib, cyclophosphamide and dexamethasone in newly diagnosed patients.

In October 2021, Takeda initiated a Phase 1 single-dose, single-center, open-label, three-arm study to assess the tolerability and safety of immune globulin subcutaneous (human), 20% solution with ENHANZE (TAK-881) at various infusion rates in healthy adult subjects.
Fourth Quarter Corporate Highlights:

In December 2021, the Company announced its second share repurchase program, to repurchase up to $750 million of its outstanding common stock over a three-year period. As part of the new share repurchase program, in December 2021, the Company entered into an accelerated share repurchase agreement to repurchase $150 million of common stock.

In October 2021, the Company repurchased 0.3 million shares of common stock for $10.3 million, completing its initial three-year $550 million share repurchase program that began in November 2019 one year early. Under the program the Company repurchased a total of 22.3 million shares for $550.0 million at an average price per share of $24.72.
Fourth Quarter and Full Year 2021 Financial Highlights

Revenue for the fourth quarter was $102.0 million compared to $121.7 million for the fourth quarter of 2020. The year-over-year decrease was primarily driven by a decrease in revenue under collaboration agreement and product sales, partially offset by an increase in royalty revenue primarily attributable to subcutaneous DARZALEX (daratumumab). Revenue for the quarter included $62.6 million in royalties, an increase of 96% compared to $32.0 million in the prior year period.

Total revenues for the full year were $443.3 million, compared with $267.6 million in 2020, representing growth of 66% year over year.

Cost of product sales for the fourth quarter was $21.6 million, compared to $26.3 million for the fourth quarter of 2020. The year-over-year decrease was primarily driven by lower product sales, principally the sales of bulk rHuPH20 to the Company’s partners.

Cost of product sales for the full year was $81.4 million, compared to $43.4 million in 2020, primarily driven by higher product sales, principally the sales of bulk rHuPH20 to the Company’s partners.

Research and development expenses for the fourth quarter were $10.1 million, compared to $7.4 million for the fourth quarter of 2020. The increase is primarily due to an increase in compensation expense, including stock-based compensation, for personnel to support additional ENHANZE targets entering clinical development.

Research and development expenses for the full year were $35.7 million, compared with $34.2 million in 2020.

Selling, general and administrative expenses for the fourth quarter were $13.8 million, compared to $10.4 million for the fourth quarter of 2020. The increase was primarily due to an increase in compensation expense, including stock-based compensation, for personnel to support additional ENHANZE targets.

Selling, general and administrative expenses for the full year were $50.3 million, compared with $45.7 million in 2020.

Operating Income: On a GAAP basis in the fourth quarter of 2021, operating income was $56.5 million, compared to an operating income of $77.6 million in the fourth quarter of 2020.

Operating income for the full year was $275.9 million, compared to $144.3 million in 2020.

Net Income: On a GAAP basis in the fourth quarter of 2021, net income was $66.8 million, compared with net income of $73.2 million in the fourth quarter of 2020. Non-GAAP net income was $61.3 million in the fourth quarter of 2021, compared with Non-GAAP net income of $80.9 million in the fourth quarter of 2020.1

Net income for the full year on a GAAP basis was $402.7 million or $2.74 per share, compared to a net income of $129.1 million or $0.91 per share in 2020. This year over year comparison is impacted by the one-time tax benefit of $1.05 per share in 2021 as a result of the tax valuation allowance release.

Net income for the full year on a non-GAAP basis was $294.1 million compared with $160.4 million in 2020.

Earnings per Share: On a GAAP basis in the fourth quarter of 2021, diluted earnings per share was $0.46, compared with $0.50 in the fourth quarter of 2020. On a Non-GAAP basis diluted earnings per share was $0.42, compared with diluted earnings per share of $0.56 in the fourth quarter of 2020.1

GAAP diluted earnings per share for the full year was $2.74 compared with $0.91 per share in 2020. These GAAP results included the one-time recognition of a non-cash income tax benefit during 2021 of $1.05 per share. Non-GAAP diluted earnings per share for the full year was $2.00 per share, compared to $1.12 per share in 2020.

Cash, cash equivalents and marketable securities were $740.9 million on December 31, 2021, compared to $368.0 million on December 31, 2020.
Financial Outlook for 2022

The Company is reiterating its financial guidance for 2022 first provided on January 10, 2022. For the full year 2022, the Company expects:

Total revenue of $530 million to $560 million, representing growth of 20%-26% over 2021 total revenue. The Company expects revenue from royalties to increase approximately 50% over revenue from royalties in 2021 to approximately $300 million.

GAAP operating income of $350 million to $380 million, representing growth of 27%-38% over 2021 GAAP operating income, resulting in operating margins greater than 65%.

GAAP net income of $270 million to $295 million; and non-GAAP net income of $290 million to $315 million.1 The Company notes that 2022 will be the first full fiscal year in which Halozyme will record income tax expense as part of its income statement.

Webcast and Conference Call

Halozyme will webcast its Quarterly Update Conference Call for the fourth quarter of 2021 today, Tuesday, February 22, 2022 at 4:30 p.m. ET/1:30 p.m. PT. Dr. Torley will lead the call, which will be webcast live through the "Investors" section of Halozyme’s corporate website and a replay will be available following the close of the call. To register for this conference call, please use this link: View Source After registering, you will receive an email confirmation that includes dial in details and unique conference call codes for entry. Registration is open through the live call. However, to ensure you are connected for the full call, please register a day in advance or at minimum 10 minutes before the start of the call.