On May 5, 2021 Eli Lilly and Company (NYSE: LLY) reported that it will participate in the Bank of America Securities 2021 Health Care Conference on Tuesday, May 11, 2021 (Press release, Eli Lilly, MAY 5, 2021, View Source [SID1234579176]). Michael Mason, senior vice president, president of Lilly Diabetes, will participate in a virtual fireside chat at 9:30 a.m., Eastern Time.

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A live audio webcast will be available on the "Webcasts & Presentations" section of Lilly’s Investor website at View Source A replay of the presentation will be available on this same website for approximately 90 days.

On May 5, 2021 MorphoSys AG (FSE:MOR; NASDAQ:MOR) reported its financial results for the first quarter of 2021 (Press release, MorphoSys, MAY 5, 2021, View Source [SID1234579187]).

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"For 2021, our focus is on three key areas: executing on the Monjuvi launch; rapidly advancing the tafasitamab backbone strategy through additional clinical studies; and expanding our pipeline," said Jean-Paul Kress, M.D., Chief Executive Officer of MorphoSys. "While we experienced headwinds from the pandemic in the first quarter, we are cautiously optimistic that the COVID-19 impact in the U.S. will start to diminish in the second half of 2021. We are confident in the potential of Monjuvi given its broad second-line label and overall profile in the r/r DLBCL setting. We are also making important progress initiating key trials for both tafasitamab and felzartamab this year."

Tafasitamab Highlights

Monjuvi(R) (tafasitamab-cxix) U.S. net product sales of € 12.9 million (US$ 15.5 million).
Monjuvi was granted a product-specific HCPCS J-Code, effective April 1, 2021.
On January 5, 2021, MorphoSys and Incyte announced that the Swiss Agency for Therapeutic Products (Swissmedic) had accepted the marketing authorization application (MAA) for tafasitamab and on January 12, 2021, MorphoSys and Incyte announced that Health Canada had accepted the New Drug Submission (NDS) for tafasitamab. Both applications seek approval for tafasitamab, in combination with lenalidomide, followed by tafasitamab monotherapy, for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), including DLBCL arising from low grade lymphoma, who are not eligible for, or refuse, autologous stem cell transplantation (ASCT).
Pipeline Highlights

Felzartamab:

M-PLACE study of felzartamab in autoimmune membranous nephropathy ongoing: the safety run-in phase was completed and the full enrollment phase opened.
In February 2021, the first patient with autoimmune membranous nephropathy was dosed with felzartamab in the New-PLACE study, a phase 2 study evaluating different treatment schedules to identify the regimen for the pivotal study.
Otilimab:

On March 2, 2021, we announced that our partner GSK reported preliminary results of the OSCAR study using otilimab for the treatment of severe pulmonary COVID-19 related disease. Given these data suggest an important clinical benefit in a pre-defined sub-group of high-risk patients and the urgent public health need, GSK has amended the OSCAR study to expand this cohort to confirm these potentially significant findings. The dosing of the first patient in the expanded study triggered milestone payments totaling € 16 million to MorphoSys.
MOR210:

On January 25, 2021, MorphoSys and I-Mab announced that the first patient has been dosed in a phase 1 dose escalation study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of MOR210/TJ210 monotherapy in patients with relapsed or refractory advanced solid tumors in the United States.
Corporate Updates

On January 6, 2021, MorphoSys announced the appointment of Sung Lee as Chief Financial Officer, effective as of February 2, 2021.
Significant Events After The Reporting Period

On April 19, 2021, MorphoSys and Incyte announced that the first patient has been dosed in the placebo-controlled Phase 3 inMIND study evaluating the efficacy and safety of tafasitamab or placebo in combination with lenalidomide and rituximab in patients with relapsed or refractory follicular lymphoma (FL) or marginal zone lymphoma (MZL).

First Quarter 2021 Financial Results (IFRS)

Total revenues for the quarter ended March 31, 2021 were € 47.2 million compared to € 251.2 million for the comparable period in 2020. The year-over-year decline was driven by the upfront payment of the collaboration and license agreement with Incyte in the first quarter 2020 for the out-licensing of tafasitamab outside the USA.

Cost of Sales: In the first three months of 2021, cost of sales increased to € 5.0 million (3M 2020: € 3.3 million).

Research and Development (R&D) Expenses: In the first three months of 2021, research and development expenses were € 33.3 million (3M 2020: € 21.5 million). Growth over 2020 reflects the increased investment to support the advancement of proprietary programs and consisted primarily of expenses for external laboratory services and personnel expenses.

Selling, General and Administrative (SG&A) Expenses: Selling expenses increased in the first three months of 2021 to € 28.2 million (3M 2020: € 12.8 million) and general and administrative expenses remained almost unchanged at € 10.3 million (3M 2020:€ 10.1 million). The year-over-year increase in selling expenses was primarily driven by the full quarter impact of the expenses for services provided by Incyte as part of the joint U.S. marketing activities for Monjuvi.

Operating Loss: Operating loss amounted to € 29.6 million in the first three months of 2021 (3M 2020: operating profit of € 203.5 million).

Consolidated Net Loss: For the first three months of 2021, consolidated net loss was € 41.6 million (3M 2020: consolidated net profit of € 195.5 million).

Cash and Investments: As of March 31, 2021, the Company had cash and investments of € 1,215.0 million compared to € 1,244.0 million on December 31, 2020.

Number of shares: The number of shares issued remained unchanged since year-end 2020 and totaled 32,890,046.

Financial Guidance and Operational Outlook for 2021

*Group revenues includes the announced € 16 million milestone payments from GSK, but excludes other potential significant milestones from development partners and/or licensing partnerships. This revenue guidance is subject to a number of uncertainties including the potential for variability from the first full year of the Monjuvi product launch, the limited visibility that MorphoSys has on the Tremfya royalty stream as well as the ongoing COVID-19 pandemic and the impact on our as well as our partner’s business operations.

**Operating expenses is comprised of R&D and SG&A, inclusive of Incyte’s share of Monjuvi selling costs in the USA.

MorphoSys expects for Tafasitamab the following events and activities in 2021:

Continuation of the phase 1b trial with tafasitamab in previously untreated DLBCL (firstMIND);
Initiation of a pivotal phase 3 trial of tafasitamab in previously untreated DLBCL (frontMIND);
Continuation of the phase 3 inMIND trial of tafasitamab in patients with relapsed or refractory follicular lymphoma (FL) or marginal zone lymphoma (MZL);
Investigation of tafasitamab, plamotamab and lenalidomide in patients with relapsed or refractory DLBCL, first-line DLBCL and relapsed or refractory follicular lymphoma (r/r FL) jointly with Incyte and Xencor;
Continuation of the L-MIND study of tafasitamab and evaluate the long-term efficacy and safety data;
Continuation of the phase 3 B-MIND study of tafasitamab in combination with bendamustine for r/r DLBCL;
Presentation of data from the 3-year follow up of L-MIND as well as other abstracts at several scientific conferences (e.g. ASCO (Free ASCO Whitepaper), EHA (Free EHA Whitepaper));
Decision on the European Marketing Authorization Application (MAA), seeking approval of tafasitamab in combination with lenalidomide, followed by tafasitamab monotherapy, for the treatment of adult patients with r/r DLBCL which is currently under review;
Support of Incyte in submitting marketing authorization applications in other markets.
MorphoSys will hold its conference call and webcast tomorrow, May 6, 2021, to present the results for the first quarter of 2021 and the further outlook for 2021.

A live webcast and slides will be made available at the Media and Investors section under Conferences on MorphoSys’ website at View Source and after the call, a slide-synchronized audio replay of the conference will be available at the same location.

The interim statement for the first quarter of 2021 (IFRS) is available online:
View Source/Reports

About tafasitamab
Tafasitamab is a humanized Fc-modified cytolytic CD19 targeting monoclonal antibody. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb(R) engineered Fc domain, which mediates B-cell lysis through apoptosis and immune effector mechanism including antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP).

Monjuvi(R) (tafasitamab-cxix) is approved by the U.S. Food and Drug Administration in combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

In January 2020, MorphoSys and Incyte entered into a collaboration and licensing agreement to further develop and commercialize tafasitamab globally. Monjuvi(R) is being co-commercialized by Incyte and MorphoSys in the United States. Incyte has exclusive commercialization rights outside the United States.

A marketing authorization application (MAA) seeking the approval of tafasitamab in combination with lenalidomide in the EU has been validated by the European Medicines Agency (EMA) and is currently under review for the treatment of adult patients with relapsed or refractory DLBCL, including DLBCL arising from low grade lymphoma, who are not candidates for ASCT.

Tafasitamab is being clinically investigated as a therapeutic option in B-cell malignancies in a number of ongoing combination trials.

Monjuvi(R) is a registered trademark of MorphoSys AG.
XmAb(R) is a registered trademark of Xencor, Inc.

On May 5, 2021 Replimune Group, Inc. (Nasdaq: REPL), a biotechnology company developing oncolytic immuno-gene therapies derived from its Immulytic platform, reported a clinical development update for CERPASS, its registration-directed global randomized Phase 2 clinical trial of RP1 in combination with Libtayo (cemiplimab) versus Libtayo for the treatment of cutaneous squamous cell carcinoma (CSCC), and for IGNYTE, its registration-directed single arm clinical trial in combination with Opdivo (nivolumab) for the treatment of anti-PD1 failed melanoma (Press release, Replimune, MAY 5, 2021, View Source [SID1234579212]).

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CERPASS

"Based on the depth and durability of responses and the manageable safety profile we have seen in patients with non-melanoma skin cancers treated with RP1 in combination with Opdivo to date, we are amending the clinical trial protocol for our Phase 2 CERPASS clinical trial to include complete response (CR) rate as a primary endpoint in addition to overall response rate (ORR), and to reduce target enrollment to 180 patients," said Robert Coffin, Ph.D., President and Chief Research and Development Officer of Replimune. "Incorporating CR as an independent additional primary endpoint should ensure a robust assessment of the clinical meaningfulness of adding RP1 to Libtayo in the primary data analysis upon which a BLA submission and FDA’s assessment for approval will be based."

Under the modified clinical trial protocol for CERPASS, Replimune plans to add CR rate as an additional independent primary endpoint, in addition to ORR, and to reduce target enrollment from 240 patients to 180 patients. Secondary endpoints will continue to include duration of response, progression-free survival (PFS), and overall survival (OS). Replimune is maintaining its guidance to expect initial data in 2022.

Replimune plans to submit the amended CERPASS clinical trial protocol to the U.S. Food and Drug Administration (FDA) in the first half of the year.

Libtayo is a registered trademark of Regeneron.

IGNYTE

Replimune recently held a Type B meeting with the FDA to discuss the design of the 125-patient, single-arm cohort in the IGNYTE clinical trial which is currently enrolling patients with anti-PD1 failed cutaneous melanoma, with registrational intent. The FDA expressed that while a randomized controlled trial would always be preferred to support licensure, that in this population if the data generated were sufficiently compelling, then the data could be considered for submission by the FDA under the accelerated approval pathway. The FDA also confirmed that a confirmatory trial would be needed as is required under the accelerated approval pathway. The design of the confirmatory trial is intended to be discussed with the FDA prior to a Biologics License Application (BLA) submission.

Dr. Coffin commented: "The discussion with the FDA at the Type B meeting provides clarity on the appropriate regulatory pathway to potential approval for RP1 in combination with Opdivo in anti-PD1 failed melanoma based on the data intended to be generated from our ongoing clinical trial. We look forward to releasing initial data from this study during 2022."

Opdivo is a registered trademark of Bristol Myers Squibb.

About CERPASS
CERPASS is Replimune’s registration-directed randomized, global Phase 2 clinical study to compare the effects of Libtayo alone versus a combination of Libtayo and Replimune’s investigational oncolytic immunotherapy RP1. The clinical trial will enroll 180 patients with locally advanced or metastatic cutaneous squamous cell carcinoma (CSCC) who are naïve to anti-PD1 therapy. The trial will evaluate complete response (CR) rate and objective response rate (ORR) as its two primary efficacy endpoints as assessed by independent review, as well as duration of response, progression-free survival (PFS), and overall survival (OS) as its secondary endpoints. The study is being run under a clinical trial collaboration agreement with Regeneron in which the costs of the trial are shared and full commercial rights retained by Replimune. Libtayo is being jointly developed by Regeneron and Sanofi.

About IGNYTE
IGNYTE is Replimune’s multi-cohort Phase 1/2 trial of RP1 plus Opdivo. There are 4 tumor specific cohorts currently enrolling in this trial including a 125-patient extension cohort of RP1 combined with Opdivo in anti-PD-1 failed cutaneous melanoma. This cohort was initiated after completing enrollment in a prior Phase 2 cohort in the same trial of approximately 30 patients with melanoma. The additional thirty patient cohorts are studying RP1 in combination with Opdivo in non-melanoma skin cancers which includes both naïve and anti-PD-1 failed CSCC, in microsatellite instability high, or MSI-H/dMMR tumor types and anti-PD-1 failed non-small cell lung cancer, or NSCLC. This trial is being done under a collaboration and supply agreement with Bristol Myer Squibb.

About RP1
RP1 is Replimune’s lead Immulytic product candidate and is based on a proprietary new strain of herpes simplex virus engineered to maximize tumor killing potency, the immunogenicity of tumor cell death and the activation of a systemic anti-tumor immune response through the expression of a GALV-GP R- fusogenic protein and GM-CSF.

On May 5, 2021 US Medical Innovations, LLC (USMI) reported that it is developing the first robotic delivery system used for cancer surgery (Press release, US Medical Innovations, MAY 5, 2021, View Source [SID1234579229]).

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The new Canady Surgical System (CSS) is a space-efficient, cost-effective, stand-alone solution for Robotic Assisted Surgery during open, laparoscopic/mini-invasive, endoscopic, thoracoscopic and trans-oral surgical procedures.

Features of the new Canady Surgical System include Voice Command technology which frees up the surgeon and assistant to focus on the surgical procedure and perform other tasks. The system includes both Canady Hybrid Plasma (CHP) and Canady Helios Cold Atmospheric Plasma (CHCAP) as well as a flexible disposable scope and camera system.

Preloaded in the CSS are treatment-dosage protocols for over 30 different cancer types and a robotic assisted image-guided navigational mapping system that delivers personalized and targeted CHCAP cancer treatments directly to the surgical site and ablating any remaining cancer cells after the tumor is removed with the CHP.

According to Jerome Canady, MD, and CEO of USMI, "Our CHCAP technology actually ablates cancer cells without damaging surrounding healthy tissue. We recently completed a Phase 1 FDA Clinical Trail using Canady Helios Cold Atmospheric Plasma (CHCAP) for the treatment of recurrent and stage 4 solid tumors. We see this novel technology, delivered with the precision of robotic assisted technology, as the most exciting new form of personalized and targeted medicine being explored today."

CHCAP has many advantages. It is performed during the surgical procedure. It is highly selective because it only targets cancer cells. And thirdly, the CHCAP treatment is personalized for specific cancer types (i.e., breast, ovarian, sarcoma, lung, colon, cervical, pancreas and other solid cancerous tumors).

The organization expects to have the new Canady Surgical System in clinical trials later this year. Protocols and site locations are being explored at this time.

On May 5, 2021 Horizon Therapeutics plc (Nasdaq: HZNP) reported first-quarter 2021 financial results (Press release, Horizon Pharma, MAY 5, 2021, View Source [SID1234579256]). The Company updated its full-year 2021 net sales guidance and its adjusted EBITDA guidance to incorporate Viela Bio, Inc., which was acquired on March 15, 2021.

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"We continued to advance our position as one of the fastest-growing biotechs, completing the milestone acquisition of Viela with its strong biologics pipeline, R&D capabilities and rare disease medicine, UPLIZNA," said Tim Walbert, chairman, president and chief executive officer, Horizon. "We were very pleased with our performance in a quarter challenged by the supply disruption of TEPEZZA due to U.S. government-mandated COVID-19 vaccine orders. With the resumption of TEPEZZA supply, our expanded pipeline and our strong ability to execute, we are well positioned to continue to drive significant value for our patients, shareholders and all of our stakeholders."

First-Quarter and Recent Company Highlights

Completed Acquisition of Viela: On March 15, 2021, the Company completed the acquisition of Viela, representing a significant step forward in advancing the Company’s strategy to expand its pipeline to accelerate long-term sustainable growth. The acquisition advances the Company’s strategy in four ways: it adds a deep, mid-stage biologics pipeline with four candidates in nine development programs; it expands the capabilities of the Company’s R&D organization with Viela’s early-stage research and translational capabilities and deep scientific knowledge in autoimmune and severe inflammatory diseases; it supports the Company’s global expansion strategy; and it diversifies the Company’s on-market medicine portfolio with the addition of UPLIZNA, an infused biologic medicine indicated for the rare disease NMOSD.
Resumed TEPEZZA Supply: In March 2021, the U.S. Food and Drug Administration approved a prior approval supplement giving the Company authorization to manufacture an increased scale of TEPEZZA drug product, resulting in a greater number of vials per manufacturing slot at its third-party manufacturer, Catalent. The Company resumed supplying the market in April following a supply disruption that began in December 2020 due to U.S. government-mandated COVID-19 vaccine orders. The Company continues to make progress on adding a second drug product manufacturer by the end of 2021.
Advancing the Company’s Global Expansion with European UPLIZNA Launch Preparations: Today, the Company announced additional plans for its global expansion, preparing to build out its European infrastructure to support the potential approval by the first quarter of 2022 of UPLIZNA for NMOSD, which has been granted orphan designation by the European Commission. The Company is adding key infrastructure to support the potential launch of UPLIZNA in Europe in the near term as well as to support the potential launch of additional medicines over the long term. The Company anticipates building a global presence over time in several other markets outside of Europe, including Japan, one of the markets the Company is pursuing for TEPEZZA, where the Company is engaging with the Pharmaceutical and Medical Devices Agency and the Japanese medical community.
UPLIZNA Approved in Japan for NMOSD: In March 2021, the Company’s strategic partner Mitsubishi Tanabe Pharma Corporation received manufacturing and marketing approval of UPLIZNA for the prevention of relapses of NMOSD from the Japanese Ministry of Health, Labour and Welfare.
Presented New UPLIZNA Data at Medical Meetings: New UPLIZNA data were presented at the American Academy of Neurology’s 73rd Annual Meeting in April, including end-of-study data from the open-label extension period (OLP) of the pivotal N-MOmentum trial in patients with NMOSD. The data showed UPLIZNA was generally well-tolerated for at least four years, and that long-term UPLIZNA treatment provided a sustained reduction in NMOSD attack risk from baseline, regardless of when treatment was initiated. Data from the OLP were also recently presented at the American Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) 2021 Forum, including a poster featuring data demonstrating the safety and efficacy of UPLIZNA in those with previous rituximab exposure. Additionally, a new analysis of the N-MOmentum trial demonstrating that the medicine consistently reduced the risk of worsening disability in people living with NMOSD was published in the May issue of Neurology Neuroimmunology & Neuroinflammation.
Initiated Enrollment in KRYSTEXXA Monthly Dosing Trial: In March 2021, the first patient was enrolled in an open-label trial to evaluate a monthly dosing regimen of KRYSTEXXA with methotrexate to treat people with uncontrolled gout. The goal of the trial is to explore whether a monthly dosing regimen can provide similar outcomes as the current dosing schedule, which is every other week.
Completed Enrollment in KRYSTEXXA PROTECT Trial: In January 2021, the Company completed enrollment in its PROTECT open-label trial. The trial, which is evaluating KRYSTEXXA to improve the management of uncontrolled gout for adults with a kidney transplant, enrolled a total of 20 patients. Results are expected in the fourth quarter of 2021.
Progressed the TEPEZZA Subcutaneous Administration Clinical Program: The Company completed dosing in late March for its first trial exploring a subcutaneous (SC) formulation of TEPEZZA. The trial is a small, single-dose Phase 1 pharmacokinetic trial which includes evaluating the use of the Halozyme ENHANZE drug-delivery technology for a SC formulation, which could potentially shorten drug administration time, reducing healthcare practitioner time and offering additional flexibility and convenience for patients.
Received Multiple Best Workplace Awards: The Company continued to be recognized as a best workplace. In March 2021, the Company ranked No. 1 on the "Fortune Best Workplaces in Biopharma 2021" list and was named to the "Best Workplaces in Ireland 2021" list. In April 2020, the Company was named one of Fortune’s "100 Best Companies to Work For" in the United States and placed on Crain’s Chicago Business’ 2021 "Best Places to Work in Chicago" list for the sixth consecutive year.
Key Clinical Development Programs

TEPEZZA: an insulin-like growth factor type 1 receptor (IGF-1R) antagonist monoclonal antibody.
Chronic Thyroid Eye Disease (TED) Trial: Phase 4 randomized, placebo-controlled trial evaluating TEPEZZA in chronic TED expected to initiate in mid-2021.
Subcutaneous Administration: Phase 1 pharmacokinetic trial exploring SC administration of TEPEZZA.
Diffuse Cutaneous Systemic Sclerosis (dcSSc) Exploratory Trial: Phase 1 exploratory trial in dcSSc expected to initiate in mid-2021.
KRYSTEXXA: a recombinant uricase enzyme that converts urate into a water-soluble liquid, allantoin, that can be easily excreted from the body.
MIRROR Randomized Controlled Trial: Phase 4 randomized, placebo-controlled trial evaluating KRYSTEXXA with methotrexate to increase the complete response rate in patients with uncontrolled gout.
PROTECT Trial: Phase 4 open-label trial evaluating KRYSTEXXA to improve management of uncontrolled gout in kidney transplant patients.
Shorter Infusion Duration Trial: Phase 4 open-label trial evaluating the impact of administering KRYSTEXXA with methotrexate over a shorter infusion duration in patients with uncontrolled gout.
Monthly Dosing Trial: Phase 4 open-label trial evaluating monthly dosing of KRYSTEXXA with methotrexate in patients with uncontrolled gout.
Retreatment Trial: Phase 4 open-label trial evaluating KRYSTEXXA with methotrexate in patients who have previously failed KRYSTEXXA monotherapy; expected to initiate in the second quarter of 2021.
UPLIZNA: an anti-CD19 humanized monoclonal antibody that depletes B cells and the pathogenic cells that produce autoantibodies.
Myasthenia Gravis Trial: Phase 3 randomized, placebo-controlled trial evaluating UPLIZNA in patients with myasthenia gravis, a chronic, rare, autoimmune neuromuscular disease that affects voluntary muscles, especially those that control the eyes, mouth, throat and limbs.
IgG4-Related Disease Trial: Phase 3 randomized, placebo-controlled trial evaluating UPLIZNA in patients with IgG4-related disease, which is a group of disorders marked by tumor-like swelling and fibrosis of affected organs, such as the pancreas, salivary glands and kidneys.
Kidney Transplant Desensitization Trial: Phase 2 open-label trial evaluating UPLIZNA, HZN-4920, or both in highly-sensitized patients waiting for a kidney transplant, which is paused due to COVID-19.
HZN-825: an oral lysophosphatidic acid receptor 1 (LPAR1) antagonist that prevents gene activation.
dcSSc Trial: Pivotal Phase 2b trial in dcSSc expected to initiate in the second quarter of 2021.
Interstitial Lung Disease Trial: Pivotal Phase 2b trial in idiopathic pulmonary fibrosis, the most common form of interstitial lung disease, expected to initiate in mid-2021.
HZN-4920: a CD40 ligand antagonist that blocks T cell interaction with the CD40-expressing B cells, disrupting the overactivation of the CD40 ligand co-stimulatory pathway. Several autoimmune diseases are associated with the overactivation this pathway.
Sjögren’s Syndrome Trial: Phase 2b randomized, placebo-controlled trial evaluating HZN-4920 in patients with Sjögren’s syndrome, a chronic, systemic autoimmune condition that impacts exocrine glands, including the salivary and tear glands.
Rheumatoid Arthritis Trial: Phase 2 randomized, placebo-controlled trial evaluating HZN-4920 in patients with rheumatoid arthritis.
Kidney Transplant Rejection Trial: Phase 2 open-label trial evaluating HZN-4920 in kidney transplant rejection patients.
HZN-7734: an anti-ILT7 human monoclonal antibody that depletes certain dendritic cells. Depleting these cells may interrupt the cycle of inflammation that causes tissue damage in diseases such as lupus, dermatomyositis and a variety of other autoimmune conditions.
Systemic Lupus Erythematosus (SLE) Trial: Phase 2 trial in SLE, a disease in which the body’s immune system attacks its own tissues and organs, expected to initiate in mid-2021.
COVID-19-Related Acute Lung Injury Trial: Phase 1 trial in COVID-19-related acute lung injury.
HZN-1116 Autoimmune Disease Trial: Phase 1 trial in autoimmune diseases expected to initiate in mid-2021.
First-Quarter Financial Results

Note: For additional detail and reconciliation of non-GAAP financial measures to the most directly comparable GAAP financial measures, please refer to the tables at the end of this release.

Net Sales: First-quarter 2021 net sales were $342.4 million. First-quarter 2020 net sales were $355.9 million.
Gross Profit: Under U.S. GAAP, the first-quarter 2021 gross profit ratio was 70.7 percent compared to 72.6 percent in the first quarter of 2020. The non-GAAP gross profit ratio in the first quarter of 2021 was 90.9 percent compared to 90.0 percent in the first quarter of 2020.
Operating Expenses: Research and development (R&D) expenses were 16.8 percent of net sales and selling, general and administrative (SG&A) expenses were 97.0 percent of net sales. Non-GAAP R&D expenses were 14.3 percent of net sales, and non-GAAP SG&A expenses were 63.8 percent of net sales.
Income Tax Expense: On a GAAP basis in the first quarter of 2021, income tax benefit was $47.8 million. First-quarter non-GAAP income tax expense was $25.8 million.
Net (Loss) Income: On a GAAP basis, first-quarter 2021 net loss was $123.4 million. First-quarter 2021 non-GAAP net income was $7.4 million.
Adjusted EBITDA: First-quarter 2021 adjusted EBITDA was $45.8 million.
(Loss) Earnings per Share: On a GAAP basis, diluted loss per share in the first quarter of 2021 and 2020 were $0.55 and $0.07, respectively. Non-GAAP diluted earnings per share in the first quarter of 2021 and 2020 were $0.03 and $0.40, respectively. Weighted average shares outstanding used for calculating GAAP and non-GAAP diluted earnings per share in the first quarter of 2021 were 223.9 million and 234.1 million, respectively.
First-Quarter Segment Results

Management uses net sales and segment operating income to evaluate the performance of the Company’s two segments, the orphan segment and the inflammation segment. While segment operating income contains certain adjustments to the directly comparable GAAP figures in the Company’s consolidated financial results, it is considered to be prepared in accordance with GAAP for purposes of presenting the Company’s segment operating results.

Orphan Segment

First-quarter 2021 net sales of the orphan segment, the Company’s strategic growth segment, were $257.5 million, an increase of 5 percent over the prior year’s quarter, driven by strong performance of KRYSTEXXA, RAVICTI, PROCYSBI and ACTIMMUNE. First-quarter 2021 net sales include a partial quarter of UPLIZNA net sales. First-quarter 2021 TEPEZZA net sales were negatively impacted by a short-term supply disruption due to U.S. government-mandated COVID-19 vaccine orders.
First-quarter 2021 net sales of the inflammation segment were $84.9 million, and segment operating income was $42.7 million.
Cash Flow Statement and Balance Sheet Highlights

On a GAAP basis in the first quarter of 2021, cash used in operating activities was $3.7 million. Non-GAAP operating cash flow was $63.5 million.
As of March 31, 2021, the Company had cash and cash equivalents of $811.6 million.
As of March 31, 2021, the total principal amount of debt outstanding was $2.618 billion. As of March 31, 2021, the gross-debt-to-last-12-months adjusted EBITDA leverage ratio was 2.8 times.
2021 Guidance

The Company now expects full‐year 2021 net sales to range between $2.75 billion and $2.85 billion, updated from the previous range of $2.70 billion and $2.80 billion. Full-year 2021 adjusted EBITDA is now expected to range between $1.02 billion and $1.06 billion, updated from the previous guidance of $1.14 billion and $1.18 billion. The updated guidance ranges incorporate Viela, which was acquired on March 15, 2021. The Company continues to expect TEPEZZA full-year 2021 net sales of greater than $1.275 billion and KRYSTEXXA full-year 2021 net sales of greater than $500 million.

Webcast

At 8 a.m. EST / 1 p.m. IST today, the Company will host a live webcast to review its financial and operating results and provide a general business update. The live webcast and a replay may be accessed at View Source Please connect to the Company’s website at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast. A replay of the webcast will be available approximately two hours after the live webcast.