On February 23, 2021 Halozyme Therapeutics, Inc. (NASDAQ: HALO) ("Halozyme" or the "Company"), a leader in converting IV biologics to subcutaneous delivery, reported that it intends to offer, subject to market conditions and other factors, $500 million aggregate principal amount of convertible senior notes due 2027 (the "Convertible Notes") (Press release, Halozyme, FEB 23, 2021, View Source [SID1234575467]). The Convertible Notes are to be offered and sold to "qualified institutional buyers" pursuant to Rule 144A under the Securities Act of 1933, as amended (the "Securities Act"). The Company also expects to grant a 30-day option to the initial purchasers to purchase up to an additional $75 million aggregate principal amount of Convertible Notes.

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The Convertible Notes will be senior, unsecured obligations of the Company and will accrue interest payable semiannually in arrears. The Convertible Notes will mature on March 1, 2027, unless earlier redeemed, repurchased or converted in accordance with their terms. Prior to September 1, 2026, the Convertible Notes will be convertible only upon the satisfaction of certain conditions and during certain periods, and on and after September 1, 2026, at any time prior to the close of business on the scheduled trading day immediately preceding the maturity date, the Convertible Notes will be convertible regardless of these conditions. The Company will settle conversions in cash and, if applicable, shares of the Company’s common stock. The initial conversion rate, interest rate and other terms of the Convertible Notes will be determined at the time of pricing in negotiations with the initial purchasers of the Convertible Notes.

The Company expects to use a portion of the net proceeds of the offering to enter into privately negotiated agreements with certain holders of its outstanding 1.25% convertible senior notes due 2024 (the "Existing Convertible Notes") to exchange their Existing Convertible Notes for a combination of cash and shares of its common stock through privately negotiated transactions entered into concurrently with or shortly after the pricing of the proposed offering (the "Note Repurchases"). In addition, the Company plans to use up to $75 million of the net proceeds of the offering to repurchase shares of its common stock under the existing stock repurchase program described below (the "Share Repurchases"), concurrently with, or shortly after, the pricing of the offering in privately negotiated transactions or otherwise, which may be effected through one or more of the initial purchasers or any affiliate thereof.

These Note Repurchases and Share Repurchases could increase (or reduce the size of any decrease in) the market price of Halozyme common stock or the Convertible Notes. We also expect that some existing noteholders may purchase or sell shares of the Company’s common stock in the market to hedge their exposure in connection with these transactions. The Note Repurchases, Share Repurchases and any associated hedging by holders could affect the market price of the Company’s common stock prior to, concurrently with or shortly after the pricing of the Convertible Notes and could also result in a higher effective conversion price for the Convertible Notes.

The Company intends to use the remainder of the net proceeds from the offering for general corporate purposes, including other repurchases of the Company’s common stock from time to time under the existing stock repurchase program described below, working capital, capital expenditures, potential acquisitions and strategic transactions. If the initial purchasers exercise their option to purchase additional notes, the Company intends to use net proceeds from the sale of additional notes for general corporate purposes.

This press release is neither an offer to sell nor a solicitation of an offer to buy the Convertible Notes or the shares of the Company’s common stock issuable upon conversion of the Convertible Notes, if any, nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction. Any offer of these securities will be made only by means of a private offering memorandum.

The offer and sale of the Convertible Notes and the shares of the Company’s common stock issuable upon conversion of the Convertible Notes, if any, have not been registered under the Securities Act, or the securities laws of any other jurisdiction, and may not be offered or sold in the United States absent registration or an applicable exemption from registration requirements.

Existing Stock Repurchase Program

In November 2019, the Board of Directors of the Company authorized a capital return program to repurchase up to $550.0 million of outstanding common stock over a three-year period. As of December 31, 2020, $200 million of Halozyme’s common stock remained available for repurchase under this program.

On February 23, 2021 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported financial results for the full year and fourth quarter of 2020 and provided financial guidance for 2021 (Press release, Jazz Pharmaceuticals, FEB 23, 2021, View Source [SID1234575466]).

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"In 2020, we made meaningful progress toward our goal to significantly grow and diversify 2022 revenues from products launched since 2019, highlighted by the strong execution of our U.S. launches of both Zepzelca and Xywav. Despite the pandemic, we delivered important new treatment options for patients, ensured the well-being of employees and generated significant value for shareholders. We meaningfully increased revenues, executed three product launches, advanced early- and late-stage clinical trials and added multiple new novel product candidates to our expanding pipeline, all of which exemplifies our highly effective operational execution throughout 2020, while continuing our transformation as an innovative global biopharmaceutical company," said Bruce Cozadd, chairman and chief executive officer of Jazz Pharmaceuticals.

Bruce Cozadd continued, "We expect 2021 to be another catalyst-rich year as we focus on our ambitious set of objectives, including continued growth of our marketed products, especially our recent launches of Xywav and Zepzelca, the next phase of Sunosi growth, two U.S. planned product launches, and the close of the GW Pharmaceuticals (GW) acquisition. We are excited about this transformative opportunity, which will bring together two innovative biopharmaceutical companies, to create a global neuroscience leader. We have long admired what the GW team has done to revolutionize cannabinoid-based medicine and look forward to combining our highly complementary neuroscience expertise across sleep medicine, epilepsies, movement disorders and psychiatry. We expect the GW transaction to provide accelerated, double-digit revenue growth through the addition of a near-term potential blockbuster product, provide a robust pipeline of complementary programs, and deliver substantial value to both shareholders and patients."

Robert Iannone, M.D., M.S.C.E., executive vice president, research and development, added, "2020 was a year of significant advancements as we initiated FDA submissions for JZP-458 in acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) under Real-Time Oncology Review (RTOR) and JZP-258 in idiopathic hypersomnia. We also continued to expand our innovative oncology and neuroscience pipelines through both internal and external collaborations, with a focus on building a highly differentiated portfolio of products to drive long-term sustainable growth. We look forward to an exciting 2021, with the opportunity for additional regulatory approvals, initiation of mid- and late-stage clinical development studies across the neuroscience and oncology therapeutic areas and the addition of a new and innovative neuroscience pipeline with the anticipated closing of the GW acquisition."

The company successfully executed on its prioritized objectives across its business during 2020. Achievements include:

Launched Xywav in the U.S. in November 2020 for the treatment of cataplexy or excessive daytime sleepiness (EDS) in narcolepsy;
Launched Zepzelca in the U.S. for the treatment of metastatic small cell lung cancer (SCLC) on or after platinum based chemotherapy in July 2020, six months after acquiring the U.S. licensing rights;
Initiated the European rolling launch for Sunosi in May 2020 to reduce excessive daytime sleepiness (EDS) in narcolepsy and obstructive sleep apnea (OSA);
Announced positive top-line results in the JZP-258 Phase 3 study in idiopathic hypersomnia (IH) in October 2020, and subsequently, completed the rolling supplemental New Drug Application (sNDA) submission under Fast Track Designation to the Food and Drug Administration (FDA) in February 2021, with potential launch in the fourth quarter of 2021; and
Initiated a Biologics License Application (BLA) submission to FDA for JZP-458 in ALL and LBL in December 2020, with potential launch in mid-2021.
Business Updates

Corporate Development

On February 3, 2021, the company announced that it had entered into a definitive agreement to acquire GW for $220.00 per American Depositary Share, in the form of $200 in cash and $20 in Jazz ordinary shares, for a total value of approximately $7.2 billion, or $6.7 billion net of GW cash. The transaction, which has been unanimously approved by the Boards of Directors of both companies, is expected to close in the second quarter of 2021, subject to satisfaction or waiver of closing conditions including regulatory approvals and the approval of GW shareholders.

Corporate Updates

In December 2020, the company appointed Jennifer Cook and Mark D. Smith, M.D. to the company’s Board of Directors.

Neuroscience

Oxybate (Xyrem and Xywav):

Following the launch of Xywav in the fourth quarter of 2020, the company will provide certain oxybate business performance metrics on a combined basis throughout the Xywav launch. Net product sales will be reported on both a combined and individual product level.

Net product sales for the combined oxybate business increased 7% to $1,757.0 million in 2020 and increased 4% to $454.5 million in the fourth quarter of 2020 compared to the same periods in 2019. Oxybate revenue bottle volume increased 4% in 2020 and 2% in the fourth quarter of 2020 compared to the same periods in 2019. Average active oxybate patients on therapy was approximately 15,300 in the fourth quarter of 2020, an increase of 2% compared to the same period in 2019.

Xyrem (sodium oxybate) oral solution:

Xyrem net product sales increased 6% to $1,741.8 million in 2020 and increased 1% to $439.3 million in the fourth quarter of 2020 compared to the same periods in 2019.
Xywav (calcium, magnesium, potassium, and sodium oxybates) oral solution:

Xywav net product sales were $15.3 million in the fourth quarter of 2020.
There were approximately 1,900 active patients on Xywav at the end of the fourth quarter of 2020, following the U.S. launch in November.
The company has entered into agreements that provide coverage for two of the three largest pharmacy benefit managers, with total commercial coverage now exceeding 60% of lives and remains on track to deliver broad commercial payor coverage within the first six to nine months following launch.
JZP-258

The company completed the rolling submission of an sNDA for JZP-258 for the treatment of IH in February 2021, with an objective of launching in the fourth quarter of 2021.
The company expects the Phase 3 results to be presented at an upcoming medical meeting in the second quarter of 2021.
Sunosi (solriamfetol):

Sunosi net product sales were $28.3 million in 2020 and $8.7 million in the fourth quarter of 2020, compared to $3.7 million and $2.7 million in the same periods of 2019 following the U.S. launch in July 2019.
In the fourth quarter of 2020, U.S. prescriptions increased 9% compared to the third quarter of 2020.
JZP-385:

JZP-385, a highly selective modulator of T-type calcium channels, is in clinical development for the potential treatment of essential tremor.
The company expects to initiate a Phase 2b trial in mid-2021.
JZP-150:

JZP-150, a fatty acid amide hydrolase (FAAH) inhibitor, is in clinical development for the potential treatment of post-traumatic stress disorder (PTSD).
The company expects to initiate a Phase 2 study in late 2021.
Oncology

Zepzelca (lurbinectedin):

Zepzelca net product sales were $90.4 million in 2020 and $53.4 million in the fourth quarter of 2020. Zepzelca launched in the U.S. in July 2020.
The company anticipates the 2021 initiation of a Phase 3 study evaluating immunotherapy plus lurbinectedin maintenance therapy, compared to immunotherapy alone, in patients with extensive-stage SCLC after induction chemotherapy.
The company continues to engage with FDA regarding the confirmatory data package.
In December 2020, the company initiated a New Drug Submission for Zepzelca in SCLC with Health Canada’s Therapeutic Products Directorate.
Erwinaze / Erwinase (asparaginase Erwinia chrysanthemi):

Erwinaze/Erwinase net product sales decreased 17% to $147.1 million in 2020 compared to $177.5 million in 2019 due to ongoing supply and manufacturing issues at the owner and sole manufacturer of the product, Porton Biopharma Limited (PBL). Erwinaze/Erwinase net product sales increased 3% to $56.6 million in the fourth quarter of 2020 compared to $54.9 million for the same period in 2019 due to timing and availability of supply. The company continues to expect inter-quarter variability in Erwinaze net product sales due to timing and availability of supply.
The company’s agreement with PBL terminated on December 31, 2020. The company has the right to sell certain Erwinaze inventory post-termination and expects to distribute this Erwinaze inventory during the first half of 2021. Once sales of available inventory are complete, the company will cease recording net sales of Erwinaze.
JZP-458 (recombinant Erwinia asparaginase):

In December 2020, the company initiated the submission of a BLA to FDA for JZP-458 for use as a component of a multi-agent chemotherapeutic regimen for the treatment of ALL or LBL in adult and pediatric patients who have developed hypersensitivity or silent inactivation to E. coli-derived asparaginase. The BLA will be reviewed under the RTOR pilot program, an initiative of the FDA’s Oncology Center of Excellence designed to expedite the delivery of safe and effective cancer treatments to patients.
The company continues to prioritize development of JZP-458 with the objective of ensuring that ALL/LBL patients have access to a reliable, high-quality recombinant asparaginase.
Enrollment in the pivotal Phase 2/3 trial continues.
The company is targeting a mid-2021 launch in the U.S., subject to anticipated FDA approval.
Defitelio (defibrotide sodium) / defibrotide:

Defitelio/defibrotide net product sales increased 13% to $195.8 million in 2020 and increased 16% to $55.5 million in the fourth quarter of 2020 compared to the same periods in 2019.
Vyxeos (daunorubicin and cytarabine) liposome for injection:

Vyxeos net product sales of $121.1 million in 2020 were in line with 2019. In the fourth quarter of 2020, net sales decreased 2% to $31.0 million compared to the same period in 2019. Vyxeos net product sales in 2020 and the fourth quarter of 2020 were negatively impacted by recommendations to increase use of oral oncology products to avoid hospitalizations and use of intensive care beds during the COVID-19 pandemic.

Commencing in 2020, following consultation with the staff of the Division of Corporation Finance of the U.S. Securities and Exchange Commission, the company no longer excludes upfront and milestone payments from the company’s non-GAAP adjusted net income, its line item components and non-GAAP adjusted EPS. For purposes of comparability, non-GAAP adjusted financial measures for the three months and year ended December 31, 2019 have been updated to reflect this change. See "Non-GAAP Financial Measures" below.

GAAP net income for 2020 was $238.6 million, or $4.22 per diluted share, compared to $523.4 million, or $9.09 per diluted share, for 2019. GAAP net income for the fourth quarter of 2020 was $133.4 million, or $2.33 per diluted share, compared to $74.0 million, or $1.29 per diluted share, for the fourth quarter of 2019.

Non-GAAP adjusted net income for 2020 was $704.0 million, or $12.46 per diluted share, compared to $885.2 million, or $15.38 per diluted share, for 2019. Non-GAAP adjusted net income for the fourth quarter of 2020 was $228.7 million, or $4.00 per diluted share, compared to $253.2 million, or $4.42 per diluted share, for the fourth quarter of 2019. Reconciliations of applicable GAAP reported to non-GAAP adjusted information are included at the end of this press release.

Total revenues increased 9% in 2020 and 14% in the fourth quarter of 2020 compared to the same periods in 2019.

Neuroscience net product sales in 2020 increased 8% to $1,785.4 million compared to 2019 led by continued strong growth in Xyrem net product sales, which increased by $99.2 million and a $24.6 million increase in Sunosi net product sales. Neuroscience net product sales in the fourth quarter of 2020 increased 6% to $463.2 million compared to the same period in 2019 led by the launch of Xywav in November 2020 and a $6.0 million increase in Sunosi net product sales.
Oncology net product sales in 2020 increased 18% to $554.5 million compared to 2019 led by strong post-launch Zepzelca net product sales of $90.4 million and a $22.9 million increase in Defitelio net product sales, partially offset by a decrease in Erwinaze net product sales of $30.3 million. Oncology net product sales in the fourth quarter of 2020 increased 46% to $196.5 million compared to the same period in 2019 led by strong Zepzelca net product sales of $53.4 million and a $7.7 million increase in Defitelio net product sales.
Operating expenses changed over the prior year periods primarily due to the following:

Selling, general and administrative (SG&A) expenses increased in 2020 and in the fourth quarter of 2020 compared to the same periods in 2019 on a GAAP and on a non-GAAP adjusted basis primarily due to increased investment in sales, marketing and launch activities related to the launches of Zepzelca and Xywav in the U.S., and the continuation of the launch of Sunosi in the U.S., as well as an increase in other expenses related to the expansion of the company’s business.
Research and development (R&D) expenses increased in 2020 compared to 2019, on a GAAP and on a non-GAAP adjusted basis, primarily due to an increase in expenses related to the progress made on the company’s clinical programs, including JZP-458 and JZP-385, partially offset by a decrease in milestone expense of $25.0 million. R&D expenses decreased in the fourth quarter of 2020 compared to the same period in 2019, on a GAAP and on a non-GAAP adjusted basis, primarily due to milestone expense of $15.0 million in the fourth quarter of 2019, partially offset by an increase in expenses related to the progress made on the company’s clinical programs, including JZP-458 and JZP-385.
Acquired in-process research and development (IPR&D) expense in 2020 on a GAAP and on a non-GAAP adjusted basis primarily related to a $200.0 million upfront payment to PharmaMar for the exclusive U.S. commercialization and development rights to Zepzelca and a $35.0 million upfront payment to SpringWorks Therapeutics, Inc., in the fourth quarter, for a FAAH inhibitor program. Acquired IPR&D expense in 2019 on a GAAP and on a non-GAAP adjusted basis included an upfront payment of $56.0 million to Codiak BioSciences, Inc. under a collaboration agreement. Acquired IPR&D expenses in 2019 on a GAAP basis also included $48.3 million related to the acquisition of Cavion, Inc. (Cavion).
In 2020, the company recorded an impairment charge of $136.1 million on a GAAP basis following the company’s decision to stop enrollment in its Phase 3 clinical study of defibrotide for the prevention of VOD due to an Independent Data Monitoring Committee determination that it is highly unlikely that the study will reach its primary endpoint.
The effective tax rate for 2019 on a GAAP basis included a one-time tax benefit of $112.3 million resulting from an intra-entity intellectual property asset transfer. Excluding this effect, the increase in the effective tax rate for 2020 on both a GAAP and on a non-GAAP adjusted basis compared to 2019 was primarily due to the benefit recognized in 2019 from the application of the Italian patent box incentive regime and the impact in 2020 of the disallowance of certain interest deductions and a provision for a proposed settlement reached with the French tax authorities.

Cash Flow and Balance Sheet

As of December 31, 2020, cash, cash equivalents and investments were $2.1 billion, and the outstanding principal balance of the company’s long-term debt was $2.4 billion.

In 2020, the company generated $899.6 million of cash from operations, made upfront and milestone payments totaling $301.0 million to PharmaMar under a license agreement and used $146.5 million to repurchase ordinary shares under the company’s share repurchase program.

In 2020, the company repurchased approximately 1.2 million ordinary shares under the company’s share repurchase program at an average cost of $121.98 per ordinary share. As of December 31, 2020, the remaining amount authorized for share repurchases under the company’s share repurchase program was $431.2 million.

Conference Call Details

Jazz Pharmaceuticals will host an investor conference call and live audio webcast today at 4:30 p.m. EST (9:30 p.m. GMT) to provide a business and financial update and discuss its 2020 full year and fourth quarter results and provide 2021 financial guidance. The live webcast may be accessed from the Investors section of the company’s website at www.jazzpharmaceuticals.com. Please connect to the website prior to the start of the conference call to ensure adequate time for any software downloads that may be necessary. Investors may participate in the conference call by dialing +1 855 353 7924 in the U.S., or +1 503 343 6056 outside the U.S., and entering passcode 3093553.

A replay of the conference call will be available through March 2, 2021 by dialing +1 855 859 2056 in the U.S., or +1 404 537 3406 outside the U.S., and entering passcode 3093553. An archived version of the webcast will be available for at least one week in the Investors section of the company’s website at www.jazzpharmaceuticals.com.

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)."