On February 23, 2021 Myriad Genetics, Inc. (NASDAQ: MYGN), a leader in genetic testing and precision medicine, reported financial results for its quarter ended Dec. 31, 2020 and provided an update on recent business performance (Press release, Myriad Genetics, FEB 23, 2021, View Source [SID1234575465]).

Financial Highlights:
Myriad Genetics delivered total revenue in the quarter of $154.6 million which declined 21% year-over-year but increased 6% sequentially from the fiscal quarter ending Sep. 30, 2020, with improved commercial execution and a more stable pricing environment.

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Total test volumes of 224,000 declined 5% year-over-year but increased 7% sequentially. Average revenue per test declined 1% sequentially, demonstrating an increasingly stable pricing environment for the company’s products and was driven by mix shift increases from non-hereditary cancer product and the reduction of zero pay tests.
During the quarter, revenue was impacted by $5.3 million due to changes associated with an increase in our payer reserve, predominantly associated with hereditary cancer revenue. The Company also had immaterial net positive adjustments to revenues related to prior periods.
GAAP gross margin was 69.5% and non-GAAP gross margin was 70.1%. Adjusted gross margin improved 30 basis points sequentially, impacted positively by test volume growth and negatively by increased revenue mix from prenatal testing.
GAAP total operating expenses were $155.7 million. Total adjusted operating expenses declined $7.9 million year-over-year to $119.6 million in the December 2020 quarter.
GAAP operating loss in the quarter was ($48.2) million; non-GAAP operating loss of ($11.3) million.
GAAP earnings per share (EPS) were ($0.59) and adjusted EPS were ($0.12) which improved by $0.03 sequentially.
Free cash flow in the quarter was ($20.4) million, and cash utilization improved by 66% sequentially. The company ended the quarter with $171.7 million in cash and equivalents and investments. In February, Myriad received a tax refund from the U.S. government providing an additional $89 million in cash.
The company negotiated an amendment to its credit facility providing increased financial flexibility. Under the new amendment certain financial covenants are waived until the June 2022 quarter and there is added flexibility around future deployment of capital. The company ended the December quarter with $224.8 million drawn on the $300 million facility. Near-term plans include using proceeds from business unit divestitures to pay down the existing credit facility balance.
Business Performance and Highlights

Women’s Health
The Myriad Women’s Health business — which serves women who are assessing their risk of cancer, and women who are pregnant or planning a family — recorded revenue of $56.3 million in the quarter, a decline of 33% year-over-year. Elective testing for hereditary cancer has been negatively impacted by the COVID-19 pandemic due to delayed elective office visits. The COVID-19 surge in the December quarter led many women to postpone elective testing. The company’s prenatal business continued to demonstrate improving fundamentals with test volumes increasing 15% year-over-year and 7% sequentially.

Myriad myRisk Hereditary Cancer
myRisk Hereditary Cancer test volumes for the Women’s Health business declined 30% year-over-year but increased 6% relative to the September 2020 quarter.
Myriad Foresight Carrier Screen
Technological enhancements to Myriad’s Foresight carrier screen test in the December quarter increased the detection rate for alpha thalassemia inherited blood conditions from 90% to >99% in high-risk ethnicities such as Hispanic patients where the risk of alpha thalassemia can be 200 times greater than the risk of cystic fibrosis. These changes reduced the risk of a false negative by 10 times and improved the accuracy of the Foresight test for ethnic minority populations.
Myriad Prequel Prenatal Screen
The scientific journal Genetics in Medicine published a study demonstrating that Myriad’s proprietary AMPLIFY technology increases the accuracy of the Prequel prenatal screen for five common microdeletions by an average of 9 times. For these microdeletions the Prequel test demonstrated 97.2% sensitivity and 99.8% specificity.
Oncology
The Myriad Oncology business provides hereditary cancer testing for patients who have cancer, and products such as the EndoPredict breast cancer prognostic test, the Prolaris prostate cancer test, and it’s myChoice CDx and BRACAnalysis CDx products for predicting response to PARP inhibitors. The Oncology business delivered total revenue of $60.4 million, down 6% relative to revenue in the December quarter of last year.

Myriad myRisk Hereditary Cancer
myRisk Hereditary Cancer test volumes for the Oncology business declined 16% year-over-year but increased 5% relative to the September 2020 quarter.
Myriad Prolaris Prostate Cancer
Received a new local coverage determination (LCD) for the Prolaris prostate cancer test from Palmetto GBA and CGS Administrators, LLC, two of the administrative contractors for the Centers for Medicare & Medicaid Services, which took effect December 6th. The new LCD expands benefit entitlements for patients with unfavorable intermediate and high-risk prostate cancer and was partially responsible for the 31% sequential growth in Prolaris revenue.
Received acceptance for a new study publication in The Prostate demonstrating high accuracy for Prolaris in predicting metastases and disease specific mortality in men following radical prostatectomy.
A new study in Genitourinary Cancer demonstrated that the Prolaris test can accurately predict which patients will benefit from multi-modality therapy. Using the newly established threshold, 27% of men with newly diagnosed high-risk disease and 73% with unfavorable intermediate-risk disease could avoid multimodality therapy.
Myriad BRACAnalysis CDx
Saw significant increases in BRACAnalysis CDx test volume in Japan with total revenue from the country increasing 167% year-over-year to $8.8 million.
Myriad myChoice CDx
Announced a strategic collaboration with Illumina, Inc. for Illumina to create a kit-based version of the myChoice companion diagnostic (CDx) test for select international markets.
Received new reimbursement for the myChoice diagnostic system in Japan effective January 1, 2021.
Myriad EndoPredict
Received new public reimbursement for EndoPredict in Germany which will take effect between March and June 2021. Currently almost half of European EndoPredict volumes come from Germany.
Mental Health
Myriad’s Mental Health business — which consists of the GeneSight Psychotropic test that helps physicians understand how genetic alterations impact response to antidepressant and other psychotropic medications — saw revenue of $18.0 million in the quarter compared to $22.5 million in the same period last year. Total revenue for GeneSight was up 51% sequentially and test volumes increased 13% sequentially.

Myriad GeneSight
Saw a strong increase in new ordering providers with over 2,100 physicians ordering GeneSight for the first time in the quarter. Strong growth in new ordering physicians was a contributor to the 13% sequential growth in test volume despite COVID-19 related headwinds.
Had two important publications on GeneSight. The first was a new publication in Psychiatry Research demonstrated that the GeneSight combinatorial test was superior to single gene testing using the Clinical Pharmacogenetics Implementation Consortium (CPIC) guidelines. In a sub-analysis utilizing the GUIDED study data, only the GeneSight combinatorial approach was able to accurately predict variations in outcomes for patients with depression and statistically significantly predicted remission, response, and symptom improvement. The second was a meta-analysis of 1,556 patients based upon four prospective controlled clinical trials and published in Pharmacogenomics. The meta-analysis demonstrated statistically significant improvements in remission, response and symptom improvement. Myriad is actively in discussions with commercial payers based upon these positive data-sets.
Autoimmune
Myriad’s Autoimmune business — which consists of the Vectra test for measuring disease activity in rheumatoid arthritis — generated revenue of $8.9 million in the quarter compared to $10.3 million in the same period last year.

Vectra
Shared new data at the American College of Rheumatology annual meeting further demonstrating that Vectra testing and three additional biomarkers, combined with traditional risk factors, can predict the risk of cardiovascular events in patients with rheumatoid arthritis. The study, which evaluated over 44,000 patients, found that a one-point increase in the Vectra score was associated with being approximately four times greater risk of having a cardiovascular event.
Announced the decision to pursue strategic alternatives for the Myriad Autoimmune business units as part of the company’s transformation and growth plan.
Other
Other revenue – comprised of Myriad RBM contract research services for the pharmaceutical industry and the myPath Melanoma diagnostic test in dermatology — was $11.0 million in the December quarter versus $14.3 million in the same period in the prior year. The decline in revenue is entirely attributable to the previously announced sale of Myriad’s German clinic which occurred at the beginning of calendar year 2020.

Announced the decision to pursue strategic alternatives for the Myriad RBM and Dermatology business units as part of the company’s transformation and growth plan.
In-Network Provider with Anthem Blue Cross Blue Shield

Further expanding coverage for its genetic tests in the United States, Myriad recently signed a contract with the majority of the affiliated commercial health plans of Anthem Blue Cross Blue Shield, the second largest commercial payer in the country. The contract returns all Myriad products to in-network status, including hereditary cancer testing. While the new agreement is not expected to materially impact revenue in fiscal year 2021, it will aid in providing easier access to testing for patients and providers, and reducing non-payment on tests across all the company’s product lines.
Investor Day

Myriad plans to host an investor day to provide an update on its strategic transformation and growth plan on May 4th. The investor day will be a virtual event hosted on the company’s website.
Financial Guidance
Given the continued unpredictability surrounding the COVID-19 pandemic and the impact it has had on the healthcare environment, customer behavior and the ability to market tests to physicians, the company will not provide financial guidance for the quarter ending March 31, 2021 or fiscal year 2021.

Conference Call and Webcast
A conference call will be held today, Tuesday, Feb. 23, 2021, at 5 p.m. EST to discuss Myriad’s financial results for the December quarter and business developments. The dial-in number for domestic callers is 1-800-584-2088. International callers may dial 1-212-231-2924. All callers will be asked to reference reservation number 21990097. An archived replay of the call will be available for seven days by dialing 1-800-633-8284 and entering the reservation number above. The conference call along with a slide presentation will be available through a live webcast at www.myriad.com.

On February 23, 2021 bluebird bio, Inc. (NASDAQ:BLUE) reported financial results and business highlights for the fourth quarter and full year ended December 31, 2020 and shared recent operational progress (Press release, bluebird bio, FEB 23, 2021, View Source [SID1234575464]).

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"For over ten years, bluebird bio has been pioneering gene therapies for patients with rare diseases and cancer, and our work continues with the analysis of the recent safety events identified in our HGB-206 study in patients with sickle cell disease. We have seen the transformative benefit of these therapies and are conducting a scientific and medical investigation to best inform our path forward on behalf of the patients we hope to serve with LentiGlobin," said Nick Leschly, chief bluebird. "While this work is ongoing, we remain focused on the upcoming PDUFA action due date for ide-cel for multiple myeloma next month, the potential EU approval of eli-cel for CALD mid-year and the planned US filings for β-thalassemia and CALD later in the year. Through these challenges, we are keeping patients at the center and were very excited to reach a major milestone of the first commercial infusion of ZYNTEGLO in Germany earlier this month."

RECENT HIGHLIGHTS

COMPANY

CLINICAL HOLD OF PHASE 1/2 AND PHASE 3 STUDIES OF LENTIGLOBIN GENE THERAPY FOR SICKLE CELL DISEASE AND TEMPORARY SUSPENSION OF MARKETING OF ZYNTEGLO IN EUROPE – On February 16, 2021, bluebird bio announced that due to a Suspected Unexpected Serious Adverse Reaction (SUSAR) of acute myeloid leukemia and a SUSAR of myelodysplastic syndrome in our HGB-206 clinical study, the FDA has placed our clinical studies of LentiGlobin for SCD on clinical hold. We are investigating these events and plan to continue to work closely with the FDA in their review of these events. In addition, we are also engaged with the EMA in discussions regarding our proposed development plans for LentiGlobin for SCD in Europe. In light of these SUSARs, we have temporarily suspended marketing of ZYNTEGLO in Europe. Additionally, the EMA has paused the renewal procedure for ZYNTEGLO’s conditional marketing authorization while the EMA’s pharmacovigilance risk assessment committee reviews the risk-benefit assessment for ZYNTEGLO and determines whether any additional pharmacovigilance measures are necessary.
INTENT TO SEPARATE – On January 11, 2021, bluebird bio announced its intent to separate its severe genetic disease and oncology businesses into two independent, publicly traded companies (bluebird bio and "Oncology Newco"). The separation is expected to be completed by year-end 2021 and it is anticipated that the spin out of Oncology Newco is to be tax-free to shareholders, subject to receipt of a favorable Internal Revenue Service (IRS) ruling.
Following the separation, bluebird bio intends to focus on delivery of its Core 3 therapies in β-thalassemia, cerebral adrenoleukodystrophy and sickle cell disease, expand access and reimbursement for our commercial product, ZYNTEGLO, in Europe and continue to explore innovative tools and technologies to ultimately bring these transformative medicines to more patients.
Following the separation, Oncology Newco plans to support commercial success of ide-cel and continued development of bb21217, deliver on the oncology pipeline of cellular therapies with a focus on non-Hodgkin’s lymphoma, acute myeloid leukemia, next-generation multiple myeloma and solid tumors and advance next generation product cycling engine with an overarching goal of 1-2 investigational new drugs (INDs) in each of the years 2021 and 2022.
At the time of separation, bluebird bio plans to capitalize each business with sufficient capital to achieve value creating milestones and intends to provide additional financial details closer to the date of separation.
TRANSFUSION DEPENDENT β-THALASSEMIA

FIRST PATIENT TREATED WITH ZYNTEGLO GENE THERAPY – bluebird bio is announcing today that a patient was treated earlier this month with the first commercial infusion of ZYNTEGLO [betibeglogene autotemcel (beti-cel)]. The patient was treated at a Qualified Treatment Center (QTC) in Germany.
TDT DATA AT ASH (Free ASH Whitepaper) – On December 5, 2020, bluebird bio presented new data showing that all patients that achieved transfusion independence continue to remain free from transfusions up to six years in the ongoing long-term follow-up study (LTF-303) of beti-cel gene therapy in patients with TDT at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. The company also presented updated efficacy and safety results for pediatric patients in the Phase 3 HGB-207 (Northstar-2) and HGB-212 (Northstar-3) studies.
SICKLE CELL DISEASE

FIRST PATIENT TREATED IN CONFIRMATORY HGB-210 STUDY –bluebird bio is announcing today that within the first quarter, the first patient was infused in HGB-210, a Phase 3 confirmatory study of LentiGlobin gene therapy (bb1111) for adults and pediatric subjects ≥2 and ≤50 years of age with sickle cell disease (SCD).
SCD DATA AT ASH (Free ASH Whitepaper) – On December 7, 2020, bluebird bio presented new data showing a complete elimination of severe vaso-occlusive events (VOEs) through 24 months of follow-up in patients who had a history of at least four severe VOEs and at least six months follow-up in Group C of its ongoing Phase 1/2 HGB-206 study of bb1111 for patients with SCD at the 62nd ASH (Free ASH Whitepaper) Annual Meeting.
MAGENTA COLLABORATION – On December 4, 2020, bluebird bio and Magenta Therapeutics announced an exclusive clinical trial collaboration to evaluate the utility of MGTA-145, in combination with plerixafor, for mobilization and collection of stem cells in patients with SCD. The combination approach has the potential to achieve safe, rapid and reliable mobilization of sufficient quantities of high-quality stem cells to ultimately improve outcomes associated with stem cell transplantation. The companies will co-fund the clinical trial and Magenta will retain all rights to its product candidate.
MULTIPLE MYELOMA

CRB-401 DATA AT ASH (Free ASH Whitepaper) – On December 5, 2020, bluebird bio and Bristol-Myers Squibb presented longer-term results showing ongoing deep and durable responses in the original Phase 1 study (CRB-401) of the companies’ investigational B-cell maturation antigen (BCMA) directed chimeric antigen receptor (CAR) T cell therapy, idecabtagene vicleucel (ide-cel), in patients with relapsed and refractory multiple myeloma at the 62nd ASH (Free ASH Whitepaper) Annual Meeting.
BB21217 DATA AT ASH (Free ASH Whitepaper) – On December 5, 2020, bluebird bio and Bristol-Myers Squibb presented updated safety and efficacy results showing promising response rates and durability from the ongoing Phase 1 study (CRB-402) of bb21217, an investigational BCMA-directed CAR T cell therapy in patients with relapsed and refractory multiple myeloma at the 62nd ASH (Free ASH Whitepaper) Annual Meeting. The company announced the study has completed enrollment and follow-up is ongoing as data continue to mature and the durability of response at the recommended phase 2 dose is assessed.
UPCOMING ANTICIPATED MILESTONES

Regulatory Outlook

Multiple Myeloma: The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 27, 2021 for the approval of ide-cel (bb2121) in patients with relapsed and refractory multiple myeloma.
SCD: The company is investigating the recently-reported safety events and plans to continue to work closely with the FDA in their review of these events to provide an update on the Company’s development plan and timeline for submission for regulatory approval.
TDT: The company is on track to complete its rolling BLA submission to the U.S. FDA for beti-cel in mid-2021, contingent upon successful resolution of any U.S. FDA concerns applicable to the program arising out of the recently-reported safety events in the SCD program. This submission is anticipated to include patients with transfusion dependent β-thalassemia across all genotypes (including non-β0/β0 genotypes and β0/β0 genotypes).
CALD: The company is on track to complete its BLA submission to the U.S. FDA for elivaldogene autotemcel (eli-cel) in mid-2021. The company plans to receive European approval for eli-cel in patients with cerebral adrenoleukodystrophy (CALD) in mid-2021.
Clinical Updates and Milestones

Updated data from ongoing clinical study in patients with SCD by the end of 2021.
Updated data from ongoing clinical studies in patients with TDT in mid-2021.
Updated data from ongoing clinical studies in patients with CALD in mid-2021.
bb21217 clinical data from the ongoing CRB-402 study in patients with multiple myeloma by the end of 2021.
Submission of 1 – 2 investigational new drug (IND) applications by the end of 2021.
FOURTH QUARTER AND FULL YEAR 2020 FINANCIAL RESULTS

Cash Position: Cash, cash equivalents and marketable securities as of December 31, 2020 and December 31, 2019 were $1.27 billion and $1.24 billion, respectively. The increase in cash, cash equivalents and marketable securities is primarily a result of proceeds received from the May 2020 public offering of the Company’s common stock and a one-time upfront payment received in connection with the Company’s amended collaboration with BMS, partially offset by cash used in support of ordinary course operating and commercial-readiness activities.
Revenues: Total revenues were $10.7 million for the three months ended December 31, 2020 compared to $10.0 million for the three months ended December 31, 2019. Total revenues were $250.7 million for the twelve months ended December 31, 2020 compared to $44.7 million for the twelve months ended December 31, 2019. The increase for the three-month period was primarily driven by an increase in royalty and other revenue. The increase for the twelve-month period was primarily driven by the amended BMS collaboration and monetization for ex-U.S. milestones and royalties from ide-cel and bb21217, with the majority of the revenue recognized relating to ide-cel license and manufacturing services.
R&D Expenses: Research and development expenses were $137.1 million for the three months ended December 31, 2020 compared to $161.8 million for the three months ended December 31, 2019. Research and development expenses were $588.0 million for the twelve months ended December 31, 2020 compared to $582.4 million for the twelve months ended December 31, 2019. The decrease for the three-month period was primarily driven by a decrease in manufacturing costs, partially offset by increased costs incurred through the amended BMS collaboration. The increase for the twelve-month period was primarily driven by an overall increase in costs incurred to advance and expand the company’s pipeline, partially offset by a decrease in manufacturing costs.
SG&A Expenses: Selling, general and administrative expenses were $77.0 million for the three months ended December 31, 2020 compared to $76.2 million for the three months ended December 31, 2019. Selling, general and administrative expenses were $286.9 million for the twelve months ended December 31, 2020 compared to $271.4 million for the twelve months ended December 31, 2019. The increase for both periods was largely attributable to increased headcount and costs incurred to support the Company’s ongoing operations and growth of its pipeline, partially offset by a decrease in costs related to commercial readiness activities due to delays during 2020 as a result of the COVID-19 pandemic.
Net Loss: Net loss was $199.9 million for the three months ended December 31, 2020 compared to $223.3 million for the three months ended December 31, 2019. Net loss was $618.7 million for the twelve months ended December 31, 2020 compared to $789.6 million for the twelve months ended December 31, 2019.

On February 23, 2021 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of drugs to treat severe metabolic, oncologic and psychiatric disorders by modulating the effects of the hormone cortisol, reported its results for the quarter- and year-ended December 31, 2020 (Press release, Corcept Therapeutics, FEB 23, 2021, https://ir.corcept.com/news-releases/news-release-details/corcept-therapeutics-announces-fourth-quarter-and-full-year-2020 [SID1234575463]).

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Corcept’s 2020 revenue was $353.9 million, compared to $306.5 million in 2019. Fourth quarter revenue was $85.7 million, compared to $87.9 million in the fourth quarter of 2019. The company reiterated its 2021 revenue guidance of $375 – 405 million.

GAAP net income was $106.0 million for the year and $26.0 million in the fourth quarter of 2020, compared to $94.2 million for the year and $29.4 million in the fourth quarter of 2019.

Excluding non-cash expenses related to stock-based compensation and the utilization of deferred tax assets, together with related income tax effects, non-GAAP net income was $34.7 million in the fourth quarter, compared to $40.3 million in the fourth quarter of 2019. For the full-year, non-GAAP net income was $145.6 million, compared to $133.3 million in 2019. A reconciliation of GAAP to non-GAAP net income is included below.

Cash and investments increased by $32.7 million in the fourth quarter, to $476.9 million at December 31, 2020. At December 31, 2019, the balance of cash and investments was 315.3 million.

The company spent $9.7 million in the fourth quarter repurchasing 458,769 shares of common stock pursuant to its stock repurchase program. Under the currently authorized terms of that program, $190.3 million remains available for the repurchase of shares.

"Corcept’s financial and clinical accomplishments in 2020 lay the foundation for significant progress this year," said Joseph K. Belanoff, MD, Corcept’s Chief Executive Officer. "As the COVID-19 pandemic is brought under control, the easing of public health restrictions and greater willingness of patients to visit their doctors will allow more physicians to diagnose and optimally treat patients with Cushing’s syndrome. We expect revenue next year of $375 – 405 million.

"Improving conditions should also permit more rapid progress in our clinical development programs, many of which have been significantly slowed by the pandemic," he added. We are currently evaluating our proprietary, selective cortisol modulators as potential treatments for patients with metastatic ovarian and pancreatic cancer, castration-resistant prostate cancer, adrenal cancer, Cushing’s syndrome, antipsychotic-induced weight gain and non-alcoholic steatohepatitis. We will have topline data from our ovarian and pancreatic cancer trials in the second quarter. In the fourth quarter, we plan to initiate a Phase 2 trial in patients with amyotrophic lateral sclerosis (ALS)."

On February 23, 2021 Acacia Pharma Group plc ("Acacia Pharma" or the "Company") (EURONEXT: ACPH), a commercial stage biopharmaceutical company focused on developing and commercializing novel products to improve the care of patients undergoing serious medical treatments such as surgery, invasive procedures, or chemotherapy, reported that, further to the announcements on 18 February 2021 and 19 February 2021 regarding a placing of new ordinary shares in the Company (the "New Ordinary Shares") by means of an accelerated bookbuild offering (the "Placing"), 10,000,000 New Ordinary Shares have been issued and admitted to trading on the regulated market of Euronext Brussels (Press release, Acacia Pharma, FEB 23, 2021, View Source [SID1234575462]).

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The New Ordinary Shares issued pursuant to the Placing have the same rights and benefits as, and rank pari passu in all respects with, the existing Ordinary Shares.

Following issuance of the New Ordinary Shares, as per 23 February 2021, the Company’s total issued share capital amounts to £1,993,789.02, represented by 99,689,451 ordinary shares with one voting right per share. The Company does not hold any ordinary shares in treasury and has not issued any other voting securities. Therefore, the total number of voting rights in the Company is 99,689,451. This number represents the denominator for purposes of notifications under transparency regulations.

On the basis of this information, shareholders of the Company can verify whether they are above or below one of the thresholds of 5%, 10%, 15%, 20% and so on, in multiples of five, of the total voting rights, and whether there is therefore an obligation to disclose that they have reached, exceeded or fallen below any such threshold in accordance with the Belgian Transparency Act of 2 May 2007. Notifications of major shareholdings must be sent by email to Acacia Pharma, for the attention of Ali Elsley at [email protected], as well as to the Belgian Financial Services and Markets Authority (FSMA), at [email protected].

The Company has in total 2,437,960 outstanding options and 2,861,500 Restricted Stock Units, for the benefit of current and former staff and the managers of the group, conferring entitlement to subscribe for a maximum total of 5,299,460 new ordinary shares, each conferring one voting right.

On February 23, 2021 Xencor, Inc. (NASDAQ:XNCR), a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies and cytokines for the treatment of cancer and autoimmune diseases, reported financial results for the fourth quarter and full year ended December 31, 2020 and provided a review of recent business and clinical highlights (Press release, Xencor, FEB 23, 2021, View Source [SID1234575461]).

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"Throughout 2020, we advanced multiple clinical programs, introduced several new XmAb bispecific technologies, and saw the progress of many partnered programs, including U.S. regulatory approval for tafasitamab, the second antibody with XmAb technology to achieve commercialization. Internally, early-stage clinical data have guided our decisions to advance several candidates into new studies scheduled for 2021. These include a potentially registrational study to evaluate the chemotherapy-free, triple combination of plamotamab, tafasitamab and lenalidomide for patients with relapsed or refractory DLBCL and a study of XmAb717 in patients with prostate cancer, a population with high unmet need and in whom we have seen encouraging activity to date," said Bassil Dahiyat, Ph.D., president and chief executive officer at Xencor. "To continue enriching our pipeline, we have entered into new collaborations with partners who identify novel tumor targets that we can engineer XmAb bispecific antibodies against. This includes our recently started partnership with Janssen Biotech, where we are engineering CD28 bispecific antibodies against a prostate tumor target and can also access their leading prostate cancer therapeutics portfolio for combination clinical studies with our growing prostate cancer pipeline."

Dr. Dahiyat added, "Looking ahead, we will continue to present maturing data from our clinical-stage programs and soon expect to initiate a Phase 1 study for XmAb564, our wholly owned IL-2 cytokine that we engineered to preferentially activate regulatory T cells, an emerging mechanism for treating patients with autoimmune diseases. In addition, we plan to submit an IND by year-end for XmAb819, an ENPP3 x CD3 bispecific antibody for renal cell carcinoma. Notably, this program is engineered with our multi-valent XmAb 2+1 format, which enables CD3 bispecific antibodies with greater tumor selectivity and against an expanded set of novel tumor antigens."

Recent Business and Clinical Highlights

Plamotamab (CD20 x CD3): In November 2020, the Company entered into a clinical collaboration with MorphoSys AG and Incyte Corporation to investigate the chemotherapy-free triple combination of plamotamab, tafasitamab and lenalidomide in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), first-line DLBCL and relapsed or refractory follicular lymphoma. Plamotamab, which redirects T cells to tumors, and tafasitamab, a CD19-directed XmAb antibody, combine powerful and distinct immune pathways, and this collaboration is designed to generate new clinical insights and accelerate development timelines for the program. MorphoSys and Incyte will provide tafasitamab for the studies, which Xencor will sponsor and fund. Tafasitamab is co-commercialized in the U.S. by MorphoSys and Incyte and is marketed as Monjuvi. Monjuvi, the second product with XmAb technology to be approved for commercial marketing, was approved by the U.S. FDA in July 2020.
XmAb717 (PD-1 x CTLA-4): In November 2020, the Company presented updated interim data from the Phase 1 study of XmAb717 (formerly XmAb20717) in patients with multiple types of advanced solid tumors at the SITC (Free SITC Whitepaper) Annual Meeting. XmAb717 was generally well-tolerated. As of the September 2020 data cutoff, a complete response was observed in a patient with melanoma, and partial responses were observed in multiple tumor types, including melanoma, renal cell carcinoma (RCC), non-small cell lung cancer (NSCLC), castration-resistant prostate cancer (CRPC) and ovarian cancer. In the first half of 2021, the Company plans to initiate a Phase 1b study of XmAb717 for patients with certain molecular subtypes of CRPC, as a monotherapy or in combination depending on the subtype, as these patients represent a high unmet medical need.
Vibecotamab (CD123 x CD3): In December 2020, the Company presented updated data from the Phase 1 study of vibecotamab in patients with relapsed or refractory acute myeloid leukemia (AML) at the ASH (Free ASH Whitepaper) Annual Meeting. The most common adverse event was cytokine release syndrome, the majority of which was observed in the first dose, and it was generally manageable with premedication. The efficacy and biomarker analyses indicated that responses appeared to be associated with lower baseline disease burden. The Company is continuing the dose escalation study and is reviewing data with Novartis in planning additional studies of vibecotamab.
XmAb564 (IL2-Fc Cytokine): XmAb564 (formerly XmAb27564) is a wholly owned, engineered IL2-Fc fusion that the Company is developing for the treatment of patients with autoimmune diseases. In January 2021, an investigational new drug (IND) application for XmAb564 was allowed by the FDA, and the Company plans to initiate a Phase 1 study in healthy volunteers in early 2021.
Select New Collaborations and Progress Across Partnered Programs

Janssen Biotech: In November 2020, Xencor entered into an agreement with Janssen Biotech, Inc., focused on the discovery of XmAb bispecific antibodies against CD28, an immune co-stimulatory receptor on T cells, and an undisclosed prostate tumor target, for the potential treatment of patients with prostate cancer. Additionally, the Company has a right to access select, predefined agents from Janssen’s portfolio of clinical-stage drug candidates and commercialized medicines to evaluate potential combination therapies in prostate cancer with agents in the Company’s own pipeline, subject to some limitations. Janssen has the same right with Xencor’s portfolio to evaluate potential combination therapies in prostate cancer. The Company received a $50 million upfront payment from Janssen and is eligible for future potential milestone and royalty payments.
MD Anderson: In December 2020, Xencor entered into a new agreement with The University of Texas MD Anderson Cancer Center to develop novel CD3 bispecific antibody therapeutics for the potential treatment of patients with cancer. MD Anderson will work to identify and develop promising antibodies, and the Company will apply its XmAb Fc bispecific technology to create therapeutic candidates. MD Anderson will then conduct and fund all preclinical activities to advance candidates toward clinical studies. Xencor has certain exclusive options to license worldwide rights to develop and commercialize potential new medicines arising from the collaboration.
Viridian Therapeutics: In December 2020, Xencor entered into a technology license agreement with MiRagen Therapeutics, Inc., which received a non-exclusive license to Xtend Fc technology and an exclusive license to apply Xtend Fc technology to antibodies targeting IGF-1R. MiRagen subsequently changed its name to Viridian Therapeutics, Inc. The Company received an upfront payment of common stock valued at $6 million.
In November 2020, Xencor entered into a product license agreement with a newly formed, privately held biotechnology company which received the exclusive worldwide rights to develop and commercialize three preclinical-stage Fc-engineered drug candidates for autoimmune disease—XmAb6755, XPro9523 and XmAb10717—programs incorporating an Xtend Fc Domain, a Cytotoxic Fc Domain, or both. The Company received a 15% equity interest in the company and is eligible to receive royalties on net sales of approved products in the mid-single digit to mid-teen percentage range.
Ultomiris (Alexion): Alexion’s Ultomiris uses Xtend Fc technology for longer half-life, and it has received marketing authorizations from regulatory agencies in the U.S., Europe and Japan for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) and for patients with atypical hemolytic uremic syndrome (aHUS). Alexion is also evaluating Ultomiris in a broad late-stage development program across many indications in neurology and nephrology. In 2020, the Company earned $16.2 million in royalties and, in the fourth quarter of 2020, received a $10 million sales-based milestone payment from Alexion.
Monjuvi is a registered trademark of MorphoSys AG. Ultomiris is a registered trademark of Alexion Pharmaceuticals, Inc.

Fourth Quarter and Full Year Ended December 31, 2020 Financial Results

Cash, cash equivalents and marketable securities totaled $604.0 million as of December 31, 2020, compared to $601.3 million on December 31, 2019. During the year, the Company received upfront payments, milestone payments and royalties from partners of $165 million, which offset spending on operations and resulted in an increase in the year-end cash balance.

Revenues for the fourth quarter ended December 31, 2020 were $41.9 million, compared to $3.5 million for the same period in 2019. Revenues for full year 2020 were $122.7 million, compared to $156.7 million in 2019. Revenues for the three-month period ended December 31, 2020 were earned primarily from the licensing of XmAb technologies and drug candidates and a sales-based milestone payment from Alexion, compared to revenues from the same period in 2019, which were primarily Alexion royalties. Total revenues earned in 2020 included royalties and milestones from the MorphoSys and Alexion agreements and the licensing of XmAb technologies and drug candidates, compared to revenue earned from Xencor’s Genentech and Astellas collaborations in 2019.

Research and development expenditures for the fourth quarter ended December 31, 2020 were $47.9 million, compared to $27.3 million for the same period in 2019. Research and development expenditures were $169.8 million for the full year ended December 31, 2020, compared to $118.6 million in 2019. Research and development spending for the fourth quarter and full year ended December 31, 2020 was greater than expenditures incurred over the comparable periods in 2019, primarily due to increased spending on Xencor’s bispecific antibody and cytokine candidates and technologies.

General and administrative expenses for the fourth quarter ended December 31, 2020 were $7.6 million, compared to $6.7 million in the same period in 2019. General and administrative expenses were $29.7 million in the full year 2020, compared to $24.3 million in 2019. Additional general and administrative spending for the full year ended December 31, 2020 over the comparable period in 2019 reflects increased staffing, professional expenses and spending on intellectual property.

Non-cash, share based compensation expense for the year ended December 31, 2020 was $31.6 million, compared to $31.9 million for the year ended December 31, 2019.

Net loss for the fourth quarter ended December 31, 2020 was $13.7 million, or $(0.24) on a fully diluted per share basis, compared to a net loss of $26.9 million, or $(0.47) on a fully diluted per share basis, for the same period in 2019. For the full year ended December 31, 2020, net loss was $69.3 million, or $(1.21) on a fully diluted per share basis, compared to a net income of $26.9 million, or $0.46 on a fully diluted per share basis, for the full year ended December 31, 2019. The lower net loss reported for the three months ended December 31, 2020 compared to the same period in 2019 is primarily due to higher revenue reported in the three months ended December 31, 2020, while the net loss reported for 2020 compared to the net income reported for 2019 is primarily due to higher research and development expenses and lower licensing and milestone revenue reported in 2020.

The total shares outstanding were 57,873,444 as of December 31, 2020, compared to 56,902,301 as of December 31, 2019.

Financial Guidance

Based on current operating plans, Xencor expects to have cash to fund research and development programs and operations into 2024. Xencor expects to end 2021 with between $425 million and $475 million in cash, cash equivalents and marketable securities.

Conference Call and Webcast

Xencor will host a conference call today at 4:30 p.m. ET (1:30 p.m. PT) to discuss these fourth quarter and full year 2020 financial results and provide a corporate update.

The live call may be accessed by dialing (877) 359-9508 for domestic callers or +1 (224) 357-2393 for international callers and referencing conference ID number 1163598. A live webcast of the conference call will be available online from the Investors section of the Company’s website at www.xencor.com. The webcast will be archived on the company’s website for 30 days.