On February 11, 2021 Labcorp (NYSE: LH), a leading life sciences company, reported results for the fourth quarter and year ended December 31, 2020, and full year 2021 guidance (Press release, LabCorp, FEB 11, 2021, View Source [SID1234574894]).

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"Labcorp’s science and innovation throughout the pandemic led to strong performance in the quarter and for the full year. In addition to our leadership in testing, our capabilities are being leveraged to identify variants to the virus through genetic sequencing and to support biotechnology and pharmaceutical companies in developing new therapies and vaccines to fight COVID-19," said Adam Schechter, chairman and CEO, Labcorp.

"We are encouraged about the future of our base business. In the fourth quarter, our base diagnostics business continued to improve as more people focused on managing their health and chronic conditions. Our drug development business also grew and delivered strong quarterly performance, with a book-to-bill of 1.43 on a trailing twelve-month basis driven by strong demand across therapeutic areas.

I want to thank our over 75,000 employees worldwide who have tirelessly served our clients and patients during the pandemic. We are entering 2021 with a renewed commitment to advancing health on a global basis."

Labcorp made substantial progress on its strategic priorities in 2020, which was a transformational year for the company:

Accelerated growth through the power of our platform: Brought Labcorp’s combined science, technology and innovation capabilities to market quickly. Labcorp was the first to receive an FDA Emergency Use Authorization ("EUA") to launch several COVID-19 PCR tests, including the Pixel by Labcorp at-home COVID-19 specimen collection kit for PCR testing, which also received the first EUA to be available through retail without a prescription, and helped bring multiple treatments and vaccines to market in record time. Our science and technology are also being leveraged to genetically sequence thousands of virus samples each week to identify variants that can inform future vaccines and treatments.
Advanced our leadership position in oncology: Expanded Labcorp’s leadership through strategic partnerships in oncology testing, major customer wins in late-stage clinical trials and the addition of leaders in the field to further build our Enterprise Oncology team.
Integrated artificial intelligence, data, digitization and analytics across our business: Increased COVID-19 testing capacity through the use of robotics and technology, and developed proprietary insight and analytics capabilities for clients to improve their operations, guide patient care and support clinical trials across a larger number of programs and therapeutic areas.
Put customers at the center of all that we do: Broadened access of COVID-19 testing to millions of people through hospitals, pharmacies, employers, physicians, drive thrus, and our Pixel by Labcorp at-home collection kit; responded to customer demand by rapidly increasing testing capacity to 275,000 COVID-19 PCR tests per day; delivered test results quickly and more easily through our websites and patient apps; and made testing affordable, charging no upfront out-of-pocket costs to consumers who needed a COVID-19 test.
Evaluated and executed on high-growth opportunities: Focused on deploying shareholder capital to grow the company’s business in diagnostic testing through targeted lab acquisitions, and accelerated wins in clinical trials with the addition of digital and mobile technology capabilities through the acquisitions of GlobalCare and SnapIOT.
Consolidated Results

Fourth Quarter Results

Revenue for the quarter was $4.49 billion, an increase of 52.0% over $2.95 billion in the fourth quarter of 2019. The increase in revenue was due to organic growth of 50.1%, acquisitions of 0.9%, and favorable foreign currency translation of 1.0%. The 50.1% increase in organic revenue includes a 46.4% contribution from COVID-19 Testing and a 3.7% increase in the company’s organic Base Business. "Base Business" includes the company’s business operations except for PCR and antibody COVID-19 testing ("COVID-19 Testing"). The increase in organic Base Business includes the negative impact from lower Medicare and Medicaid pricing as a result of PAMA of (0.6%).

Operating income for the quarter was $1,293.2 million, or 28.8% of revenue, compared to $336.4 million, or 11.4%, in the fourth quarter of 2019. The increase in operating income and margin was primarily due to COVID-19 Testing, organic Base Business growth, acquisitions, and LaunchPad savings, partially offset by the negative impact from PAMA of ($18.3) million and higher personnel costs. The company recorded amortization, restructuring charges, and special items, which together totaled $136.3 million in the quarter, compared to $85.6 million during the same period in 2019. This increase was primarily due to COVID-19 related costs and increased amortization. Adjusted operating income (excluding amortization, restructuring charges, and special items) for the quarter was $1,429.5 million, or 31.8% of revenue, compared to $422.0 million, or 14.3%, in the fourth quarter of 2019.

Net earnings for the quarter were $938.3 million, compared to $227.1 million in the fourth quarter of 2019. Diluted EPS were $9.54 in the quarter, up from $2.32 in the same period in 2019. Adjusted EPS (excluding amortization, restructuring charges, and special items) were $10.56 in the quarter, up from $2.86 in the fourth quarter of 2019.

Operating cash flow for the quarter was $774.6 million, compared to $569.8 million in the fourth quarter of 2019. The increase in operating cash flow was due to higher cash earnings, partially offset by higher working capital to support growth. In addition, operating cash flow for the fourth quarter of 2020 was reduced by $132.1 million due to the company’s decision to return the Coronavirus Aid, Relief, and Economic Security Act (CARES Act) Provider Relief Funds that the company received during the second and third quarters of 2020. Capital expenditures totaled $99.4 million, down from $128.2 million a year ago, despite the expenditures associated with the increase in COVID-19 Testing capacity. As a result, free cash flow (operating cash flow less capital expenditures) was $675.2 million, up from $441.6 million in the fourth quarter of 2019.

At the end of the quarter, the company’s cash balance and total debt were $1.3 billion and $5.8 billion, respectively. During the quarter, the company invested $58.8 million on acquisitions. As of December 31, 2020, the company had $800.0 million of authorization remaining under its share repurchase program.

Full Year Results

Revenue was $13.98 billion, an increase of 21.0% over $11.55 billion last year. The increase in revenue was due to organic growth of 19.0%, acquisitions of 1.8%, and favorable foreign currency translation of 0.4%, partially offset by the disposition of a business of (0.2%). The 19.0% increase in organic revenue includes the 24.1% contribution from COVID-19 Testing, partially offset by the (5.1%) reduction in the company’s organic Base Business due to the pandemic. The decline in organic Base Business includes the negative impact from PAMA of (0.6%).

Operating income was $2.45 billion, or 17.5% of revenue, compared to $1.33 billion, or 11.5%, in 2019. The increase in operating income and margin was primarily due to COVID-19 Testing and LaunchPad savings, partially offset by the negative impact from the pandemic (including the impairments to goodwill and other assets), higher personnel costs, and ($72.1) million from PAMA. The company recorded amortization, restructuring charges, special items, and impairments, which together totaled $886.5 million, compared to $380.6 million in 2019. This increase includes the $437.4 million of COVID-19 related impairments on goodwill and other assets that the company recorded in the first quarter of 2020. In addition, the company recorded $63.9 million of other COVID-19 related costs. Adjusted operating income (excluding amortization, restructuring charges, special items, and impairments) was $3.33 billion, or 23.8% of revenue, compared to $1.71 billion, or 14.8%, in 2019. The $1.62 billion increase in adjusted operating income and 900 basis point increase in adjusted operating margin were primarily due to the increase in COVID-19 Testing and LaunchPad savings, partially offset by the reduction in the Base Business (due to the pandemic) and higher personnel costs.

Net earnings in 2020 were $1,556.1 million, or $15.88 per diluted share, compared to $823.8 million, or $8.35 per diluted share, in 2019. Adjusted EPS (excluding amortization, restructuring charges, special items, and impairments) were $23.94, an increase of 111.5% over $11.32 in 2019.

Operating cash flow was $2.14 billion, compared to $1.44 billion in 2019. The increase in operating cash flow was due to higher cash earnings, partially offset by higher working capital to support growth. Capital expenditures totaled $381.7 million, down from $400.2 million in 2019, despite the expenditures associated with the increase in COVID-19 Testing capacity. As a result, free cash flow (operating cash flow less capital expenditures) was $1.75 billion, compared to $1.04 billion in 2019.

During the year, the company invested $267.6 million on acquisitions, repurchased $100.0 million of stock representing approximately 0.6 million shares, and paid down $412.2 million of debt.

Fourth Quarter Segment Results

The following segment results exclude amortization, restructuring charges, special items, and unallocated corporate expenses. The segments that are now referred to as Diagnostics and Drug Development were formerly referred to as LabCorp Diagnostics and Covance Drug Development.

Diagnostics

Revenue for the quarter was $3.15 billion, an increase of 79.5% over $1.76 billion in the fourth quarter of 2019. The increase in revenue was due to organic growth of 78.5%, acquisitions of 0.9%, and favorable foreign currency translation of 0.1%. The 78.5% increase in organic revenue was due to a 78.0% contribution from COVID-19 Testing and a 0.5% increase in the Base Business, which includes the negative impact from PAMA of (1.0%). This represents the first quarter of 2020 with organic Base Business revenue growth versus prior year.

Total volume (measured by requisitions) increased by 33.9% as organic volume increased by 33.3% and acquisition volume contributed 0.6%. The organic volume growth was due to the 41.0% contribution from COVID-19 Testing demand, partially offset by a (7.7%) reduction in the Base Business volume due to the pandemic. Price / mix increased by 45.5% primarily due to COVID-19 Testing of 37.0% and organic Base Business of 8.2%, which includes the negative impact from PAMA of (1.0%). In the fourth quarter, Diagnostics achieved its highest contribution from COVID-19 Testing volume, as well as the strongest year-over-year volume in the Base Business since the pandemic began in March 2020.

Adjusted operating income for the quarter was $1,234.4 million, or 39.1% of revenue, compared to $277.1 million, or 15.8%, in the fourth quarter of 2019. The $957.3 million increase in adjusted operating income and 2,340 basis point increase in adjusted operating margin were primarily due to the increase in COVID-19 Testing and LaunchPad savings, partially offset by the negative impact from PAMA of ($18.3) million and higher personnel costs. The company remains on track to deliver approximately $200 million of net savings from its three-year Diagnostics LaunchPad initiative by the end of 2021. The company continues to develop and execute new LaunchPad programs to support profitable growth in Diagnostics.

Drug Development

Revenue for the quarter was $1.40 billion, an increase of 16.4% over $1.20 billion in the fourth quarter of 2019. The increase in revenue was due to organic growth of 13.1%, acquisitions of 0.9%, and favorable foreign currency translation of 2.3%. The 13.1% increase in organic revenue was due to an 8.4% increase in the Base Business and a 4.7% contribution from COVID-19 Testing performed through its Central Laboratories business. While continuing to be negatively impacted by the pandemic, Drug Development achieved its highest quarter of organic Base Business growth in 2020, benefiting from broad-based demand across all service lines, including COVID-19 vaccine and therapeutic work.

Adjusted operating income for the quarter was $248.4 million, or 17.8% of revenue, compared to $183.2 million, or 15.2%, in the fourth quarter of 2019. The $65.2 million increase in adjusted operating income and 250 basis point increase in adjusted operating margin were primarily due to COVID-19 Testing, organic Base Business demand, and LaunchPad savings, partially offset by higher personnel costs. The company achieved its goal to deliver approximately $150 million of net savings from its three-year Drug Development LaunchPad initiative. While the target was achieved, the company continues to develop and execute new LaunchPad programs to support profitable growth in Drug Development.

Net orders and net book-to-bill during the trailing twelve months were $6.96 billion and 1.43, respectively. Backlog at the end of the quarter was $13.76 billion, compared to $12.46 billion last quarter, and the company expects approximately $4.5 billion of its backlog to convert into revenue in the next twelve months.

Outlook for 2021

The following guidance assumes foreign exchange rates effective as of December 31, 2020 for the full year. Enterprise level guidance includes the estimated impact from currently anticipated capital allocation, including acquisitions and share repurchases.

(1) 2021 Guidance includes a benefit from foreign currency translation of 0.9%.

(2) Enterprise level revenue is presented net of intersegment transaction eliminations, including Drug Development COVID-19 Testing revenue.

(3) 2021 Guidance includes a benefit from foreign currency translation of 0.1%.

(4) 2021 Guidance includes a benefit from foreign currency translation of 2.2%.

(5) Free Cash Flow consists of operating cash flow less capital expenditures.

Use of Adjusted Measures

The company has provided in this press release and accompanying tables "adjusted" financial information that has not been prepared in accordance with GAAP, including adjusted net income, adjusted EPS (or adjusted net income per share), adjusted operating income, adjusted operating margin, free cash flow, and certain segment information. The company believes these adjusted measures are useful to investors as a supplement to, but not as a substitute for, GAAP measures, in evaluating the company’s operational performance. The company further believes that the use of these non-GAAP financial measures provides an additional tool for investors in evaluating operating results and trends, and growth and shareholder returns, as well as in comparing the company’s financial results with the financial results of other companies. However, the company notes that these adjusted measures may be different from and not directly comparable to the measures presented by other companies. Reconciliations of these non-GAAP measures to the most comparable GAAP measures and an identification of the components that comprise "special items" used for certain adjusted financial information are included in the tables accompanying this press release.

The company today is providing an investor relations presentation with additional information on its business and operations, which is available in the investor relations section of the company’s website at View Source Analysts and investors are directed to the website to review this supplemental information.

A conference call discussing Labcorp’s quarterly results will be held today at 9:00 a.m. ET and is available by dialing 877-898-8036 (720-634-2811 for international callers). The conference ID is 7378794. A telephone replay of the call will be available through Feb. 25, 2021, and can be heard by dialing 855-859-2056 (404-537-3406 for international callers). The conference ID for the replay is 7378794. A live online broadcast of Labcorp’s quarterly conference call on Feb. 11, 2021, will be available at Labcorp Investor Relations website beginning at 9:00 a.m. ET. This webcast will be archived and accessible through Jan. 28, 2022.

On February 11, 2021 Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-driven biopharmaceutical group, reported its financial results for the full year 2020 (Press release, Ipsen, FEB 11, 2021, View Source [SID1234574893]).

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Solid full year 2020 financial results in a COVID-19 environment
Group sales of €2,592 million, growing by 3.0%1 at constant currency or 0.6% as reported, driven by Specialty Care sales growth of 5.9%1, reflecting a resilient oncology portfolio, while Consumer Healthcare sales were down 21.3%1 mostly due to the impact of COVID-19.
Core Operating margin at 32.0% of the sales, up 1.6 points. IFRS Operating margin at 20.1% of the sales, up 21.4 points.
Core consolidated net profit of €610.5 million, with fully diluted Core EPS growing by 8.4% to reach €7.31. IFRS Consolidated net profit showing a gain of €548.9 million, with IFRS net earnings per share of €6.57.
Sound financial structure, with a closing Net Debt of €525.3 million and a Net Debt to EBITDA ratio of 0.6x. Strong Free Cash Flow at €646.4 million, up 38%, mainly driven by higher Operating Cash Flow.
Advancing solid pipeline in H2 2020
Cabometyx (cabozantinib) in combination with nivolumab for 1L renal cell carcinoma (RCC) filing with EMA based on successful Phase 3 CheckMate -9ER;
Onivyde (irinotecan liposome injection) received Fast Track designation from the FDA for 2L Small Cell Lung Cancer (SCLC);
Palovarotene on track for NDA and MAA submissions for fibrodysplasia ossificans progressiva (FOP).
Proposed dividend of €1.00 per share2 for the 2020 financial year, consistent with the prior year.
2021 guidance3 of Group sales growth greater than 4.0% at constant currency and Core Operating margin greater than 30.0% of the sales.
Executing on new strategy presented in December 2020: Together. For patients and society.

David Loew, Chief Executive Officer of Ipsen, stated: "I am truly proud of Ipsen’s performance in 2020. We met our financial objectives, delivering steady top-line growth, significant core operating margin expansion and strong cash flow generation to fund our external innovation strategy. The fact these achievements took place against the backdrop of the pandemic is remarkable and speaks to the dedication and patient-centricity of our highly motivated employees. We made encouraging pipeline progress, especially in Oncology. In December we announced our new strategy which will drive long-term value for all our stakeholders: Focus. Together. For patients & society. While the world continues to face uncertain business conditions in 2021, I am confident that Ipsen will build on its strong foundations and execute on its strategy to deliver another successful year."

New Strategy
Ipsen is executing on the four key pillars of its new strategy presented in December 2020:

The Group is focused on maximizing the value of its current Specialty Care product portfolio through commercial excellence and geographic expansion. It aims to maximize its core brands and capture the full potential of its innovative oncology products. A strategic review of the Consumer Healthcare business is proceeding.
Ipsen’s priority is to build a sustainable pipeline to drive long-term growth. Recent initiatives have prioritized the pipeline on the highest potential opportunities and progressed the transformation of the R&D organization. Ipsen is strengthening its external innovation efforts by targeting differentiated medicines in its three core therapeutics areas of Oncology, Rare Disease and Neuroscience, with a broader disease and modality scope than previously defined, and across all stages of clinical development.
The company is committed to generating efficiencies through a focused and agile operating model. Leveraging smart spending, streamlined operations, manufacturing efficiencies and optimizing digitalization, the Group will be able to reinvest in R&D and external innovation to fuel future growth.
Patients and society are at the core of Ipsen’s mission, starting with fully engaged employees and a culture of accountability to perform and compete in the long term. Ipsen is highly committed to its corporate social responsibility (CSR) initiatives which are centered around employees, community and the environment, as reflected throughout the organization and in the inclusion of responsibility metrics in management compensation.

2021 Financial guidance
The Group has set the following financial targets for the current year, assuming a progressive recovery from COVID-19 by H2 2021:

Group sales growth year-on-year greater than 4.0% at constant currency1, with an expected negative 3.0% impact of currency based on the level of exchange rates at the end of January 2021;
Core Operating margin greater than 30.0% of the sales, excluding any potential impact of incremental investments from external innovation.
This guidance assumes a phased launch of lanreotide generic in Europe by mid-2021 and a limited impact in case of a potential launch of octreotide or lanreotide generics in the U.S.

Group sales reached €2,591.6 million, up 3.0%1 year-on-year.

Specialty Care sales reached €2,381.1 million, up 5.9%1, driven by the continued strong growth of Somatuline (lanreotide) and Cabometyx. Somatuline growth of 13.1%1 was driven by continued positive momentum in North America with a double-digit growth (17.0%1) and solid performance throughout Europe despite the availability of the octreotide generic. Dysport (botulinum toxin type A) down by 3.4%1, was impacted in most geographies by the closure of treatment centers resulting from COVID-19 despite a faster recovery in the aesthetics market. Decapeptyl (triptorelin) sales reflected good volume growth across Major European countries offset by lower volumes in China.

Consumer Healthcare sales reached €210.6 million, down 21.3%1, mainly due to a decline in Smecta (diosmectite) sales impacted by COVID-19, the implementation of hospital central procurement in China and lower performance in France.

Core Operating Income reached €829.3 million in 2020, compared to €782.6 million in 2019, a growth of 6.0%, driven by sales growth, Group-wide efficiencies and costs savings with less travel, medical and marketing expenses due to COVID-19 partially offset by continued investment in R&D to advance key programs in Oncology, Rare Disease and Neuroscience.

Core Operating margin reached 32.0% of the sales, up 1.6 points compared to 2019.

Core consolidated net profit was €610.5 million in 2020, an increase of 8.4% versus €563.4 million in 2019, driven by higher Core Operating Income.

Fully diluted Core EPS (earnings per share) grew by 8.4% to reach €7.31, compared to €6.74 in 2019.

IFRS Fully diluted EPS was a net profit per share amounting to €6.57 versus a net loss of €0.61 in 2019.

Free Cash Flow reached €646.4 million, up by €178.7 million, mainly driven by higher Operating Cash Flow thanks to a lower level of capital expenditures and working capital partly offset by higher cash out from restructuring costs, financial result and current income tax.

Closing net debt reached €525.3 million at the end of 2020, as compared to closing net debt in 2019 of €1,115.6 million.

Conference call
Ipsen will hold a conference call Thursday, 11 February 2021 at 2:30 p.m. (Paris time, GMT+1). Participants should dial in to the call approximately five to ten minutes prior to its start. No reservation is required to participate in the conference call.

On February 11, 2021 Pulmatrix, Inc. (NASDAQ: PULM), a clinical stage biopharmaceutical company developing innovative inhaled therapies to address serious pulmonary and non-pulmonary disease using its patented iSPERSE technology, reported that it has entered into definitive agreements with healthcare-focused institutional investors for the purchase and sale of 20,000,000 shares of the Company’s common stock, at a purchase price of $2.00 per share, in a registered direct offering priced at-the-market under Nasdaq rules (Press release, Pulmatrix, FEB 11, 2021, View Source [SID1234574892]). The closing of the offering is expected to occur on or about February 16, 2021, subject to the satisfaction of customary closing conditions.

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H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering.

The gross proceeds to Pulmatrix from this offering are expected to be $40,000,000, before deducting the placement agent’s fees and other offering expenses. The Company intends to use the net proceeds from this offering for working capital and general corporate purposes.

The shares of common stock (are being offered by Pulmatrix pursuant to a "shelf" registration statement on Form S-3 (File No. 333-230225) previously filed with the Securities and Exchange Commission (the "SEC") on March 12, 2019 and declared effective by the SEC on March 15, 2019. The offering of the securities will be made only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement. A final prospectus supplement and accompanying prospectus relating to the shares of common stock being offered will be filed with the SEC. Electronic copies of the final prospectus supplement and accompanying prospectus may be obtained, when available, on the SEC’s website at or by contacting H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, NY 10022, by phone at (646) 975-6996 or e-mail at .

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

On February 11, 2021 Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, reported its consolidated financial results for the fourth quarter and full year ended December 31, 2020 and reviewed recent business highlights (Press release, Alnylam, FEB 11, 2021, View Source [SID1234574890]).

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"We’re very pleased with the 2020 commercial performance for our marketed products, and expect steady and continued growth in 2021 across our product portfolio, driven by improved disease awareness, new patient finding, and geographic expansion. In the fourth quarter we celebrated the regulatory approvals of our third and fourth commercial medicines: OXLUMO, in the EU and U.S.; and Leqvio, in the EU. We also reported positive topline results from the HELIOS-A Phase 3 study of vutrisiran, our fifth investigational medicine to achieve that important milestone," said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "Having exceeded our "Alnylam 2020" vision and goals, we now look to the next chapter of our Company’s story, and are excited to have recently launched our new five-year vision, ‘Alnylam P5x25.’ With Alnylam P5x25, we aim to bring transformative medicines in both rare and common diseases to patients around the world, while advancing a robust and high-yielding pipeline of first and/or best-in-class product candidates from our organic research engine, and delivering exceptional financial performance."

Fourth Quarter 2020 and Recent Significant Corporate Highlights

Commercial Performance

ONPATTRO

Achieved global net product revenues for the fourth quarter and full year 2020 of $90 million and $306 million, respectively, representing 9.5% quarterly growth compared to Q3 – including 10.4% growth in the U.S. market segment driven by new patient demand – and over 80% annual growth from full year 2019.
Attained approximately 1,350 patients worldwide on commercial ONPATTRO treatment as of December 31, 2020.
Continued global expansion with achievement of regulatory approval in Taiwan and market access now achieved in over 20 countries.
GIVLAARI

Achieved global net product revenues for the fourth quarter and full year 2020 of $22 million and $55 million, respectively, representing over 30% quarterly growth compared to Q3.
Attained approximately 200 patients worldwide on commercial GIVLAARI treatment as of December 31, 2020.
Continued strong progress toward establishing value-based agreements (VBAs), with over 10 VBAs finalized to date – including the first state-level VBA with MassHealth – and confirmed access for 94% of covered U.S. lives.
Continued progress with market access efforts across the CEMEA region, with an ongoing launch in Germany, Temporary Authorization for Use (ATU) supply in France, and named patient sales in other countries.
The Company announces today that it has achieved market access in Italy.
OXLUMO

For the period following EMA and FDA approval of OXLUMO in late November 2020, observed strong initial U.S. demand with 8 Start Forms received through December 31, 2020.
Achieved global net product revenues for the fourth quarter of approximately $0.3 million representing initial patient demand in Europe.
The Company has actively engaged with U.S. and EU payers toward establishing VBAs, including both Patient Need Adjustment and Prevalence-Based Adjustment features.
The Company announces today that it has completed its first OXLUMO VBA with a major commercial payer, with several more under negotiation.
Achieved significant progress with market access efforts across the CEMEA region, with an ongoing launch in Germany, and ATU supply in France.
R&D Highlights

Patisiran (the non-proprietary name for ONPATTRO), in development for the treatment of the cardiomyopathy of both hereditary and wild-type ATTR amyloidosis

Continued enrollment in the APOLLO-B Phase 3 study in ATTR amyloidosis patients with cardiomyopathy, with plans to complete enrollment in early 2021.
Vutrisiran, a subcutaneously administered investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis

Reported positive topline results from the HELIOS-A Phase 3 study in hATTR amyloidosis patients with polyneuropathy, with plans to file a New Drug Application (NDA) with the FDA in early 2021.
Continued enrollment in the HELIOS-B Phase 3 study in ATTR amyloidosis patients with cardiomyopathy.
Lumasiran (the non-proprietary name for OXLUMO), for the treatment of primary hyperoxaluria type 1 (PH1)

Presented positive complete results from ILLUMINATE-B, a global Phase 3 pediatric study of lumasiran in PH1 patients less than six years of age, including infants, with preserved renal function.
Completed enrollment in the ILLUMINATE-C Phase 3 study of lumasiran for the treatment of advanced PH1 in patients of all ages.
Leqvio (inclisiran), for the treatment of hypercholesterolemia

Alnylam’s partner Novartis received marketing authorization for Leqvio from the European Commission in December 2020.
Novartis received a Complete Response Letter from the FDA in December 2020, due to unresolved facility inspection-related conditions at a third-party manufacturing facility in Europe. Novartis is working closely with the third-party manufacturer and the FDA to obtain approval as soon as possible, and has guided for a resubmission of its NDA in Q2-Q3 2021.
Fitusiran, in development for the treatment of hemophilia A or B with and without inhibitors

Alnylam’s partner, Sanofi, resumed dosing and enrollment in the ATLAS Phase 3 program.
Sanofi announced that fitusiran was granted Fast Track designation by the FDA for all indications.
Early- and mid-stage RNAi therapeutic pipeline programs

Presented positive interim results from the Phase 1 study of ALN-AGT, in development for the treatment of hypertension.
Initiated dosing in the Phase 1 study of ALN-HSD, in development for the treatment of non-alcoholic steatohepatitis (NASH).
Continued enrollment and dosing in the Phase 2 study of cemdisiran monotherapy in IgA nephropathy, and initiated dosing in a Phase 1 study of combination therapy with pozelimab, an anti-C5 monoclonal antibody, in collaboration with Regeneron.
Alnylam’s partner Vir Biotechnology continues enrollment and dosing in a Phase 2 combination trial of ALN-HBV02 (VIR-2218) with pegylated interferon-alpha (PEG-IFN-α).
Continued progress with investigational RNAi therapeutics for CNS and ocular diseases, including advancement of ALN-APP, in development for the treatment of cerebral amyloid angiopathy (CAA) and autosomal dominant Alzheimer’s Disease (ADAD), with an expected CTA filing in mid-2021.
Additional Business Updates

Launched Alnylam P5x25 strategy.
Received recognition from Science Magazine as a Top Biopharma Employer.
Issued the second annual Patient Access Philosophy Report.
Announced new senior leadership appointments:
Tolga Tanguler, Executive Vice President, Chief Commercial Officer
Kasha Witkos, Senior Vice President, Head of CEMEA
Salil Patel, Ph.D., Senior Vice President, Head of Medical Affairs
Agnieszka Gallagher, Senior Vice President, Chief Ethics and Compliance Officer
Upcoming Events

Alnylam announces today that it intends to present complete 9-month results from the HELIOS-A Phase 3 study of vutrisiran at the American Academy of Neurology (AAN) 2021 Virtual Meeting being held April 17-22, 2021.
In addition, in early 2021, Alnylam intends to:

File an NDA for vutrisiran.
Complete enrollment in the APOLLO-B Phase 3 study of patisiran in patients with ATTR amyloidosis with cardiomyopathy.
Initiate study of biannual dosing regimen with vutrisiran.
Achieve regulatory approval for OXLUMO in Brazil.
Financial Results for the Quarter and Year Ended December 31, 2020

"We saw very strong performance from our commercial products in 2020, even with headwinds from the COVID-19 pandemic. ONPATTRO demonstrated more than 80% growth from 2019 and GIVLAARI achieved $55 million in full year revenues, an excellent start for this important medicine in its launch year. In 2021, we expect continued growth from both products, and look forward to now leveraging our global commercial capabilities for the launch of OXLUMO, which was recently approved in the U.S. and EU," said Jeff Poulton, Chief Financial Officer of Alnylam. "Today, we are guiding that we expect to achieve between $610 million and $660 million in combined net product revenues for full-year 2021 across our three wholly owned commercial brands, representing over 75% growth at the midpoint of the range as compared with our 2020 results. In addition, we intend to continue to demonstrate disciplined investment in our operations as we transition toward a self-sustainable financial profile."

Combined net product revenues were $112.8 million in the fourth quarter 2020 representing 102% growth from the fourth quarter 2019. The strong growth primarily resulted from an increase in patients on therapy driven by growth in established markets and continued global expansion of ONPATTRO and GIVLAARI, as well as initial net product revenues from OXLUMO following regulatory approval in the fourth quarter of 2020.
Combined net product revenues were $361.5 million in the full-year 2020 representing 117% growth from the full-year 2019. The strong growth was driven by the continued global expansion of ONPATTRO and the global launch of GIVLAARI.
Net Revenues from Collaborations

Net revenues from collaborations were $50.7 million in the fourth quarter 2020, an increase from $15.7 million in the fourth quarter 2019, primarily due to the achievement of a $15.0 million milestone related to Leqvio approval in Europe under the Novartis collaboration agreement, as well as an increase in revenue recognized in connection with our collaboration agreement with Regeneron.
Net revenues from collaborations were $131.3 million for the full-year 2020, an increase from $53.2 million in 2019, primarily due to increases in revenue recognized from our collaborations with Regeneron, Vir, and Novartis.
Research & Development (R&D) and Selling, General & Administrative (SG&A) Expenses

R&D expenses decreased on a GAAP basis in the fourth quarter 2020 compared to the same period in 2019, primarily due to a decrease in manufacturing activity for clinical drug and a decrease in stock-based compensation as a result of the achievement of certain performance-based milestones in 2019. R&D expenses decreased on a non-GAAP basis in the fourth quarter 2020 compared to the same period in 2019 primarily due to decreases in manufacturing activity for clinical drug.
For the full-year 2020, R&D expenses were relatively consistent on a GAAP basis and increased on a non-GAAP basis, compared to the same period in 2019, primarily due to increased expenses associated with clinical and preclinical activities, personnel, and facilities as we continue to support our long-term strategic growth offset by decreased license fees associated with regulatory filings.
SG&A expenses increased in the fourth quarter 2020 compared to the same period in 2019 on a GAAP and non-GAAP basis primarily due to increased investment in commercial and medical affairs activity to support the ongoing launches of ONPATTRO and GIVLAARI and launch activities for OXLUMO.
For the full-year 2020, SG&A expenses increased on a GAAP and non-GAAP basis, compared to the same period in 2019, primarily due to increased investment in commercial and medical affairs activity to support the ongoing launches of ONPATTRO and GIVLAARI and initial launch of OXLUMO. SG&A expenses on a GAAP basis also increased for the full-year 2020, compared to the same period in 2019, due to a change in estimate of contingent liabilities related to our arbitration with Ionis.
Cash and Investments

Cash, cash equivalents, and marketable debt and equity securities were $1.87 billion at the end of the fourth quarter 2020 compared to $1.54 billion at the end of 2019. The increase was primarily due to $800.0 million in proceeds from the sale of future royalties, the issuance of common stock and the initial draw down under our credit agreement in each case associated with our strategic financing collaboration with The Blackstone Group Inc. and certain of its affiliates, partially offset by cash used in our operations to advance our pipeline and support overall growth.
A reconciliation of GAAP to non-GAAP results for the current quarter is included in the tables of this press release.

Use of Non-GAAP Financial Measures

This press release contains non-GAAP financial measures, including expenses adjusted to exclude certain non-cash expenses and non-recurring gains outside the ordinary course of the Company’s business. These measures are not in accordance with, or an alternative to, GAAP, and may be different from non-GAAP financial measures used by other companies.

The items included in GAAP presentations but excluded for purposes of determining non-GAAP financial measures for the periods presented in this press release are stock-based compensation expenses, unrealized losses (gains) on marketable equity securities, costs associated with our strategic financing collaboration, loss/net loss on contractual settlement, change in estimate of contingent liabilities and a gain on the change in fair value of a liability obligation. The Company has excluded the impact of stock-based compensation expense, which may fluctuate from period to period based on factors including the variability associated with performance-based grants for stock options and restricted stock units and changes in the Company’s stock price, which impacts the fair value of these awards. The Company has excluded the impact of the costs associated with our strategic financing collaboration, loss/net loss on contractual settlement, change in estimate of contingent liabilities and a gain on the change in fair value of a liability obligation because the Company believes these items are non-recurring transactions outside the ordinary course of the Company’s business. The Company has excluded the impact of the unrealized losses (gains) on marketable equity securities because the Company does not believe these adjustments accurately reflect the performance of the Company’s ongoing operations for the period in which such gains or losses are reported as their sole purpose is to adjust amounts on the balance sheet.

The Company believes the presentation of non-GAAP financial measures provides useful information to management and investors regarding the Company’s financial condition and results of operations. When GAAP financial measures are viewed in conjunction with non-GAAP financial measures, investors are provided with a more meaningful understanding of the Company’s ongoing operating performance and are better able to compare the Company’s performance between periods. In addition, these non-GAAP financial measures are among those indicators the Company uses as a basis for evaluating performance, allocating resources and planning and forecasting future periods. Non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. A reconciliation between GAAP and non-GAAP measures is provided later in this press release.

Conference Call Information

Management will provide an update on the Company and discuss fourth quarter and year-end 2020 results as well as expectations for the future via conference call on Thursday, February 11, 2021 at 8:30 am ET. To access the call, please dial 877-312-7507 (domestic) or +1-631-813-4828 (international) five minutes prior to the start time and refer to conference ID 6180123. A replay of the call will be available beginning at 11:30 am ET on the day of the call. To access the replay, please dial 888-859-2056 (domestic) or +1-404-537-3406 (international) and refer to conference ID 6180123.

A live audio webcast of the call will be available on the Investors section of the Company’s website at www.alnylam.com/events. An archived webcast will be available on the Alnylam website approximately two hours after the event.

About ONPATTRO (patisiran)

ONPATTRO is an RNAi therapeutic that was approved in the United States and Canada for the treatment of the polyneuropathy of hATTR amyloidosis in adults. ONPATTRO is also approved in the European Union, Switzerland and Brazil for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and in Japan for the treatment of hATTR amyloidosis with polyneuropathy. ONPATTRO is an intravenously administered RNAi therapeutic targeting transthyretin (TTR). It is designed to target and silence TTR messenger RNA, thereby blocking the production of TTR protein before it is made. ONPATTRO blocks the production of TTR in the liver, reducing its accumulation in the body’s tissues in order to halt or slow down the progression of the polyneuropathy associated with the disease. For more information about ONPATTRO, visit ONPATTRO.com.

ONPATTRO Important Safety Information

Infusion-Related Reactions

Infusion-related reactions (IRRs) have been observed in patients treated with ONPATTRO (patisiran). In a controlled clinical study, 19% of ONPATTRO-treated patients experienced IRRs, compared to 9% of placebo-treated patients. The most common symptoms of IRRs with ONPATTRO were flushing, back pain, nausea, abdominal pain, dyspnea, and headache.

To reduce the risk of IRRs, patients should receive premedication with a corticosteroid, acetaminophen, and antihistamines (H1 and H2 blockers) at least 60 minutes prior to ONPATTRO infusion. Monitor patients during the infusion for signs and symptoms of IRRs. If an IRR occurs, consider slowing or interrupting the infusion and instituting medical management as clinically indicated. If the infusion is interrupted, consider resuming at a slower infusion rate only if symptoms have resolved. In the case of a serious or life-threatening IRR, the infusion should be discontinued and not resumed.

Reduced Serum Vitamin A Levels and Recommended Supplementation

ONPATTRO treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking ONPATTRO. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with ONPATTRO, as serum levels do not reflect the total vitamin A in the body.

Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g. night blindness).

Adverse Reactions

The most common adverse reactions that occurred in patients treated with ONPATTRO were upper respiratory tract infections (29%) and infusion-related reactions (19%).

For additional information about ONPATTRO, please see the full Prescribing Information.

About GIVLAARI (givosiran)

GIVLAARI is an RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) approved in the United States and Brazil for the treatment of adults with acute hepatic porphyria (AHP). GIVLAARI is also approved in the European Union for the treatment of AHP in adults and adolescents aged 12 years and older. In the pivotal study, givosiran was shown to significantly reduce the rate of porphyria attacks that required hospitalizations, urgent healthcare visits or intravenous hemin administration at home compared to placebo. GIVLAARI is Alnylam’s first commercially available therapeutic based on its Enhanced Stabilization Chemistry ESC-GalNAc conjugate technology to increase potency and durability. GIVLAARI is administered via subcutaneous injection once monthly at a dose based on actual body weight and should be administered by a healthcare professional. GIVLAARI works by specifically reducing elevated levels of aminolevulinic acid synthase 1 (ALAS1) messenger RNA (mRNA), leading to reduction of toxins associated with attacks and other disease manifestations of AHP. For more information about GIVLAARI, visit GIVLAARI.com.

GIVLAARI Important Safety Information

Contraindications

GIVLAARI is contraindicated in patients with known severe hypersensitivity to givosiran. Reactions have included anaphylaxis.

Anaphylactic Reaction

Anaphylaxis has occurred with GIVLAARI treatment (<1% of patients in clinical trials). Ensure that medical support is available to appropriately manage anaphylactic reactions when administering GIVLAARI. Monitor for signs and symptoms of anaphylaxis. If anaphylaxis occurs, immediately discontinue administration of GIVLAARI and institute appropriate medical treatment.

Hepatic Toxicity

Transaminase elevations (ALT) of at least 3 times the upper limit of normal (ULN) were observed in 15% of patients receiving GIVLAARI in the placebo-controlled trial. Transaminase elevations primarily occurred between 3 to 5 months following initiation of treatment.

Measure liver function tests prior to initiating treatment with GIVLAARI, repeat every month during the first 6 months of treatment, and as clinically indicated thereafter. Interrupt or discontinue treatment with GIVLAARI for severe or clinically significant transaminase elevations. In patients who have dose interruption and subsequent improvement, reduce the dose to 1.25 mg/kg once monthly. The dose may be increased to the recommended dose of 2.5 mg/kg once monthly if there is no recurrence of severe or clinically significant transaminase elevations at the 1.25 mg/kg dose.

Renal Toxicity

Increases in serum creatinine levels and decreases in estimated glomerular filtration rate (eGFR) have been reported during treatment with GIVLAARI. In the placebo-controlled study, 15% of patients receiving GIVLAARI experienced a renally-related adverse reaction. The median increase in creatinine at Month 3 was 0.07 mg/dL. Monitor renal function during treatment with GIVLAARI as clinically indicated.

Injection Site Reactions

Injection site reactions were reported in 25% of patients receiving GIVLAARI in the placebo-controlled trial. Symptoms included erythema, pain, pruritus, rash, discoloration, or swelling around the injection site. One (2%) patient experienced a single, transient, recall reaction of erythema at a prior injection site with a subsequent dose administration.

Drug Interactions

Concomitant use of GIVLAARI increases the concentration of CYP1A2 or CYP2D6 substrates, which may increase adverse reactions of these substrates. Avoid concomitant use of GIVLAARI with CYP1A2 or CYP2D6 substrates for which minimal concentration changes may lead to serious or life-threatening toxicities. If concomitant use is unavoidable, decrease the CYP1A2 or CYP2D6 substrate dosage in accordance with approved product labeling.

Adverse Reactions

The most common adverse reactions that occurred in patients receiving GIVLAARI were nausea (27%) and injection site reactions (25%).

For additional information about GIVLAARI, please see full Prescribing Information.

About OXLUMO (lumasiran)

OXLUMO is an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients. HAO1 encodes glycolate oxidase (GO), an enzyme upstream of the disease-causing defect in PH1. OXLUMO works by degrading HAO1 messenger RNA and reducing the synthesis of GO, which inhibits hepatic production of oxalate – the toxic metabolite responsible for the clinical manifestations of PH1. In the pivotal ILLUMINATE-A study, OXLUMO was shown to significantly reduce levels of urinary oxalate relative to placebo, with the majority of patients reaching normal or near-normal levels. Injection site reactions (ISRs) were the most common drug-related adverse reaction. In the ILLUMINATE-B pediatric Phase 3 study, OXLUMO demonstrated an efficacy and safety profile consistent to that observed in ILLUMINATE-A. OXLUMO utilizes Alnylam’s Enhanced Stabilization Chemistry (ESC)-GalNAc conjugate technology designed to increase potency and durability. OXLUMO is administered via subcutaneous injection once monthly for three months, then once quarterly thereafter at a dose based on actual body weight. For patients who weigh less than 10 kg, ongoing dosing remains monthly. OXLUMO should be administered by a healthcare professional. For more information about OXLUMO, visit OXLUMO.com.

OXLUMO Important Safety Information

Adverse Reactions

The most common adverse reaction that occurred in patients treated with OXLUMO was injection site reaction (38%). Symptoms included erythema, pain, pruritus, and swelling.

Pregnancy and Lactation

No data are available on the use of OXLUMO in pregnant women. No data are available on the presence of OXLUMO in human milk or its effects on breastfed infants or milk production. Consider the developmental and health benefits of breastfeeding along with the mother’s clinical need for OXLUMO and any potential adverse effects on the breastfed child from OXLUMO or the underlying maternal condition.

For additional information about OXLUMO, please see the full Prescribing Information.

About LNP Technology

Alnylam has licenses to Arbutus Biopharma LNP intellectual property for use in RNAi therapeutic products using LNP technology.

About RNAi

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines, known as RNAi therapeutics, is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

On February 11, 2021 Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical company with expertise in protein dysregulation and a pipeline of investigational therapeutics for rare peripheral amyloid and neurodegenerative diseases, reported financial results for the fourth quarter and full year 2020 (Press release, Prothena, FEB 11, 2021, View Source [SID1234574889]). In addition, the Company provided 2021 financial guidance and an update on its R&D programs.

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"We continue to see positive momentum in our pipeline with the recent announcement of the confirmatory, registration-enabling Phase 3 AFFIRM-AL study of birtamimab in AL amyloidosis and positive clinical findings reported in 2020 from both the Phase 2 study of prasinezumab in Parkinson’s disease and Phase 1 study of PRX004 in ATTR amyloidosis," said Gene Kinney, Ph.D., President and Chief Executive Officer of Prothena. "We are particularly pleased that our unique protein dysregulation platform has now resulted in the translation of our preclinical findings into clinical benefit for patients across multiple programs in our portfolio. We look forward to a number of significant milestones in 2021, with the planned initiation of 3 late-stage clinical studies, including the birtamimab AFFIRM-AL study, the prasinezumab Phase 2b study and the PRX004 Phase 2/3 study. In addition to these late-stage programs, we have also advanced the three targets under our global neuroscience collaboration with Bristol-Myers Squibb and expect to file an IND for PRX005 this year. Our strong cash position and potential partner payments provide a foundational capital position to fund the company through value-creating milestones as we transition to a fully integrated commercial company."

Full Year 2020 and Recent Developments:

Birtamimab, a potential treatment for AL amyloidosis, is a humanized monoclonal antibody designed to directly neutralize soluble toxic aggregates and promote clearance of amyloid that causes organ dysfunction and failure

Based on significant survival benefit observed in the previous VITAL study in Mayo Stage IV patients (HR=0.413, p=0.025, over 9 months) and multiple in-depth discussions with the U.S. Food and Drug Administration (FDA), Prothena announced plans in February 2021 to advance birtamimab into the confirmatory Phase 3 AFFIRM-AL study in Mayo Stage IV patients with AL amyloidosis. AFFIRM-AL is a global, registration-enabling Phase 3 study that will be conducted with a primary endpoint of all-cause mortality at p≤0.10 under a Special Protocol Assessment (SPA) agreement with FDA.
Prasinezumab, a potential treatment for Parkinson’s disease, is a humanized monoclonal antibody designed to target key epitopes within the C-terminus of alpha-synuclein and is the focus of the worldwide collaboration with Roche

Presented results from Phase 2 PASADENA study showing prasinezumab significantly slows progression on pre-specified clinical measures of Parkinson’s disease in September 2020 at the International Parkinson and Movement Disorder Society’s 2020 Congress. Prasinezumab is the first potentially disease-modifying, anti-alpha-synuclein antibody to demonstrate signals of efficacy on multiple pre-specified secondary and exploratory clinical endpoints in patients with early Parkinson’s disease.
Announced that Roche and Prothena will advance prasinezumab into a late-stage Phase 2b study in patients with early Parkinson’s disease. The study will be designed to further assess the efficacy of prasinezumab by expanding upon the patient population enrolled in PASADENA to include patients with early Parkinson’s disease on stable levodopa therapy. Prasinezumab is the first anti-alpha synuclein antibody to advance into late-stage development.
PRX004, a potential treatment for ATTR amyloidosis, is a humanized monoclonal antibody designed to deplete the pathogenic, non-native forms of the TTR protein

Reported results from the Phase 1 study of PRX004, the first anti-amyloid immunotherapy designed to deplete amyloid to demonstrate efficacy in ATTR amyloidosis. In the first report of clinical results with this depleter mechanism of action, PRX004 showed favorable results as demonstrated by slowing of neuropathy progression for all 7 evaluable patients at 9 months, including improvement in neuropathy in 3 of the 7 patients, and improved cardiac systolic function for all 7 patients. In this Phase 1 study, PRX004 was found to be generally safe and well tolerated across all dose levels.
PRX012, a potential treatment for Alzheimer’s disease, is monoclonal antibody targeting key epitopes within the N-terminus of Aβ

Presented data from next generation anti-Aβ antibodies for subcutaneous administration to improve patient access at the 13th Clinical Trials on Alzheimer’s Disease (CTAD) Conference in November 2020
Multi-immunogen vaccine, for the potential prevention and treatment of Alzheimer’s disease, is a multi-immunogen Aβ-tau vaccine

Presented data on a multi-immunogen vaccine that targets key Aβ and tau epitopes, the two main pathological proteins involved in the cause and progression of Alzheimer’s disease, at the CTAD Conference in November 2020
Corporate

Appointed Brandon Smith as Chief Business Officer, responsible for leading Prothena’s business development activities, portfolio strategic planning and alliance management activities. Mr. Smith joined Prothena after serving as Chief Operating Officer at Iconic Therapeutics
Upcoming Milestones:

Birtamimab

Phase 3 AFFIRM-AL study initiation expected mid-2021
VITAL study 9-month results expected to be presented at medical conference in 2021
Prasinezumab

New pre-specified exploratory subgroup analyses from Part 1 of the Phase 2 PASADENA study to be presented at the 15th International Conference for Alzheimer’s and Parkinson’s Diseases in March 2021 (ADPD 2021)
$60 million clinical milestone payment to be achieved upon first patient dosed in late-stage Phase 2b study in patients with early Parkinson’s disease; further details expected in 2Q 2021
Results from Part 2 of the PASADENA study expected to be presented at an upcoming medical conference
PRX004

Phase 1 study results expected to be presented at medical conference in 2021
Phase 2/3 study in patients with ATTR-cardiomyopathy expected to initiate 4Q 2021
PRX005

Preclinical data to be presented in March at ADPD 2021
IND filing expected 3Q 2021
$80 million potential payment from Bristol-Myers Squibb upon exercising their US license option in 2021
PRX012

IND filing expected in 1Q 2022
Upcoming Investor Conferences

Members of the senior management team will present and participate in investor meetings at the following upcoming investor conferences:

H.C. Wainwright Global Life Sciences Conference on Tuesday and Wednesday, March 9-10, 2021, virtual presentations will be available on demand both days
Oppenheimer 31st Annual Healthcare Conference on Wednesday March 17, 2021 at 10:00 AM ET
Stifel’s 3rd Annual CNS Day on Thursday April 1, 2021 at 8:00 AM ET
Fourth Quarter and Full Year of 2020 Financial Results and 2021 Financial Guidance

For the fourth quarter and full year of 2020, Prothena reported a net loss of $30.7 million and $111.1 million, respectively, as compared to a net loss of $21.6 million and $77.7 million for the fourth quarter and full year of 2019, respectively. Net loss per share for the fourth quarter and full year of 2020 was $0.77 and $2.78, respectively, as compared to a net loss per share of $0.54 and $1.95 for the fourth quarter and full year of 2019, respectively.

Prothena reported total revenue of $0.4 million and $0.9 million for the fourth quarter and full year of 2020, respectively, primarily due to license revenue in the fourth quarter and collaboration revenue for the full year as compared to total revenue of $0.3 million and $0.8 million for the fourth quarter and full year of 2019, respectively, primarily due to Roche collaboration revenue.

Research and development (R&D) expenses totaled $20.8 million and $74.9 million for the fourth quarter and full year of 2020, respectively, as compared to $15.5 million and $50.8 million for the fourth quarter and full year of 2019, respectively. The increase in R&D expense for the fourth quarter and full year of 2020 compared to the same periods in the prior year was primarily due to higher manufacturing costs primarily related to our PRX005, birtamimab and PRX012 programs and to a lesser extent PRX004, higher collaboration expense with Roche related to the prasinezumab program and higher R&D consulting expense. R&D expenses included non-cash share-based compensation expense of $2.1 million and $8.2 million for the fourth quarter and full year of 2020, respectively, as compared to $2.0 million and $8.1 million for the fourth quarter and full year of 2019, respectively.

General and administrative (G&A) expenses totaled $9.9 million and $38.7 million for the fourth quarter and full year of 2020, respectively, as compared to $8.1 million and $35.7 million for the fourth quarter and full year of 2019, respectively. The increase in G&A expenses for the fourth quarter and full year of 2020 compared to the same periods in the prior year was primarily related to higher costs for our director and officer insurance premiums. G&A expenses included non-cash share-based compensation expense of $3.2 million and $13.8 million for the fourth quarter and full year of 2020, respectively, as compared to $3.3 million and $15.5 million for the fourth quarter and full year of 2019, respectively.

Total non-cash share-based compensation expense was $5.2 million and $22.0 million for the fourth quarter and full year of 2020, respectively, as compared to $5.3 million and $23.6 million for the fourth quarter and full year of 2019, respectively.

As of December 31, 2020, Prothena had $298.1 million in cash, cash equivalents and restricted cash and no debt.

As of February 5, 2021, Prothena had approximately 39.9 million ordinary shares outstanding.

The Company expects the full year 2021 net cash used in operating and investing activities to be $51 to $74 million, which includes an expected $60 million milestone payment from Roche upon first patient dosed in the late-stage Phase 2b study of prasinezumab and expects to end the year with approximately $235 million in cash, cash equivalents and restricted cash (midpoint). The estimated full year 2021 net cash used in operating and investing activities is primarily driven by an estimated net loss of $79 to $111 million, which includes an estimated $20 million of non-cash share-based compensation expense.

Conference Call Details

Prothena management will discuss these results and its 2021 financial guidance during a live audio conference call today, Thursday, February 11, 2021, at 8:30 AM ET. The conference call will be made available on the Company’s website at www.prothena.com under the Investors tab in the Events and Presentations section. Following the live audio webcast, a replay will be available on the Company’s website for at least 90 days.

To access the call via dial-in, please dial (877) 887-5215 (U.S. and Canada toll free) or (315) 625-3069 (international) five minutes prior to the start time and refer to conference ID number 5677514. A replay of the call will be available until February 25, 2021 via dial-in at (855) 859-2056 (U.S. toll free) or (404) 537-3406 (international), Conference ID Number 5677514.