On May 5, 2021 Unum Group (NYSE: UNM) reported net income of $153.0 million ($0.75 per diluted common share) for the first quarter of 2021, compared to net income of $161.0 million ($0.79 per diluted common share) for the first quarter of 2020 (Press release, Unum Therapeutics, MAY 5, 2021, View Source [SID1234579232]).

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Unum Group Reports First Quarter 2021 Results
Included in net income for the first quarter of 2021 are the net after-tax loss from the second phase of the Closed Block individual disability reinsurance transaction of $56.7 million ($0.27 per diluted common share), the after-tax amortization of the cost of reinsurance of $15.8 million ($0.08 per diluted common share), and a net after-tax realized investment gain on the Company’s investment portfolio, excluding the net realized investment gain associated with the completion of the second phase of the Closed Block individual disability reinsurance transaction, of $13.5 million ($0.06 per diluted common share). Included in net income for the first quarter of 2020 is a net after-tax realized investment loss on the Company’s investment portfolio of $113.1 million ($0.56 per diluted common share). Excluding the items above, after-tax adjusted operating income was $212.0 million ($1.04 per diluted common share) in the first quarter of 2021, compared to $274.1 million ($1.35 per diluted common share) in the first quarter of 2020.

"Our first quarter results reflect the dynamics of the world around us with good growth results, pressured by high, yet improving, levels of mortality," said Richard P. McKenney, president and chief executive officer. "With vaccines rolling out and a better economy, the current trends show continued improvement. Looking forward, our market leadership and financial underpinnings position us well to drive growth as the recovery builds in the second half of the year."

RESULTS BY SEGMENT

We measure and analyze our segment performance on the basis of "adjusted operating income" or "adjusted operating loss", which differ from income before income tax as presented in our consolidated statements of income due to the exclusion of net realized investment gains and losses, amortization of cost of reinsurance, and certain other items. These performance measures are in accordance with GAAP guidance for segment reporting, but they should not be viewed as a substitute for income before income tax or net income.

Unum US Segment

Unum US reported adjusted operating income of $115.7 million in the first quarter of 2021, a decrease of 55.8 percent from $261.8 million in the first quarter of 2020. Premium income for the segment was $1,525.8 million in the first quarter of 2021, which was generally consistent with the $1,527.7 million in the first quarter of 2020. Net investment income for the segment was $179.7 million in the first quarter of 2021, which was consistent with the $179.6 million in the first quarter of 2020.

Within the Unum US operating segment, the group disability line of business reported a 16.0 percent decrease in adjusted operating income to $64.1 million in the first quarter of 2021, compared to $76.3 million in the first quarter of 2020. Premium income for the group disability line of business increased 1.0 percent to $672.9 million in the first quarter of 2021, compared to $666.2 million in the first quarter of 2020 due to higher sales in our short-term disability product line. Net investment income increased 4.3 percent to $97.4 million in the first quarter of 2021, compared to $93.4 million in the first quarter of 2020, driven by higher miscellaneous investment income, partially offset by a decline in the yield on invested assets and a lower level of invested assets. The benefit ratio for the first quarter of 2021 was 74.8 percent, compared to 73.2 percent in the first quarter of 2020, due to higher claims incidence in the short-term disability product line, resulting from the impacts of COVID-19, partially offset by favorable claim recoveries in our group long-term disability product line. Group long-term disability sales were $31.1 million in the first quarter of 2021, which was generally consistent with the $31.4 million in the first quarter of 2020. Group short-term disability sales were $24.1 million in the first quarter of 2021, an increase of 69.7 percent from $14.2 million in the first quarter of 2020. Persistency in the group long-term disability product line was 90.7 percent for the first quarter of 2021, compared to 90.6 percent for the first quarter of 2020. Persistency in the group short-term disability product line was 86.1 percent for the first quarter of 2021, compared to 86.1 percent for the first quarter of 2020.

The group life and accidental death and dismemberment line of business reported an adjusted operating loss of $58.3 million in the first quarter of 2021, compared to adjusted operating income of $70.4 million in the first quarter of 2020. Premium income for this line of business decreased 1.1 percent to $451.4 million in the first quarter of 2021, compared to $456.2 million in the first quarter of 2020, driven primarily by lower prior period sales, partially offset by higher persistency. Net investment income decreased 3.1 percent to $24.9 million in the first quarter of 2021, compared to $25.7 million in the first quarter of 2020, due to decline in the yield on invested assets. The benefit ratio in the first quarter of 2021 was 98.8 percent, compared to 70.6 percent in the first quarter of 2020, due primarily to higher claims incidence in the group life product line resulting from the impacts of COVID-19. Sales of group life and accidental death and dismemberment products increased 7.5 percent in the first quarter of 2021 to $30.2 million, compared to $28.1 million in the first quarter of 2020. Persistency in the group life product line was 90.1 percent for the first quarter of 2021, compared to 88.4 percent for the first quarter of 2020. Persistency in the accidental death and dismemberment product line was 89.7 percent for the first quarter of 2021, compared to 87.9 percent for the first quarter of 2020.

The supplemental and voluntary line of business reported a decrease of 4.5 percent in adjusted operating income to $109.9 million in the first quarter of 2021, compared to $115.1 million in the first quarter of 2020. Premium income for the supplemental and voluntary line of business decreased to $401.5 million in the first quarter of 2021, compared to $405.3 million in the first quarter of 2020, with a decline in the voluntary benefits product line, mostly offset by growth in the individual disability and dental and vision product lines. Net investment income decreased 5.1 percent to $57.4 million in the first quarter of 2021, compared to $60.5 million in the first quarter of 2020, due to a decline in the yield on invested assets, partially offset by an increase in the level of invested assets. The benefit ratio for the individual disability product line was 42.4 percent for the first quarter of 2021, compared to 52.1 percent for the first quarter of 2020, due to lower claims incidence and higher mortality. The benefit ratio for the voluntary benefits product line was 39.3 percent in the first quarter of 2021, compared to 32.7 percent for the first quarter of 2020, due to higher claims incidence in the life product line, resulting from the impacts of COVID-19. The benefit ratio for the dental and vision product line was 73.2 percent for the first quarter of 2021, compared to 65.1 percent for the first quarter of 2020, due primarily to higher claims incidence. Relative to the first quarter of 2020, sales in the individual disability product line declined 25.1 percent in the first quarter of 2021 to $17.0 million. Sales in the voluntary benefits product line declined 21.5 percent in the first quarter of 2021 to $101.0 million. Sales in the dental and vision product line totaled $8.0 million for the first quarter of 2021, a decrease of 25.9 percent compared to the first quarter of 2020. Persistency in the individual disability product line was 90.2 percent for the first quarter of 2021, compared to 88.9 percent for the first quarter of 2020. Persistency in the voluntary benefits product line was 74.3 percent for the first quarter of 2021, compared to 72.4 percent for the first quarter of 2020. Persistency in the dental and vision product line was 87.4 percent for the first quarter of 2021, compared to 81.9 percent for the first quarter of 2020.

Unum International

The Unum International segment reported adjusted operating income of $26.4 million in the first quarter of 2021, an increase of 36.1 percent from $19.4 million in the first quarter of 2020. Premium income increased 6.0 percent to $174.4 million in the first quarter of 2021, compared to $164.6 million in the first quarter of 2020. Net investment income was $26.0 million in the first quarter of 2021, compared to $26.5 million in the first quarter of 2020. Sales decreased 2.9 percent to $23.2 million in the first quarter of 2021, compared to $23.9 million in the first quarter of 2020.

The Unum UK line of business reported adjusted operating income, in local currency, of £18.6 million in the first quarter of 2021, an increase of 35.8 percent from £13.7 million in the first quarter of 2020. Premium income was £110.5 million in the first quarter of 2021, a decrease of 2.9 percent from £113.8 million in the first quarter of 2020, driven by an increase in ceded premiums in the group life product line, partially offset by both higher sales and persistency in the group life product line and rate increases in the group long-term disability product line. Net investment income was £17.4 million in the first quarter of 2021, a decrease of 9.8 percent from £19.3 million in the first quarter of 2020, due to lower miscellaneous investment income and a lower yield on fixed-rate bonds, partially offset by higher investment income from inflation index-linked bonds. The benefit ratio in the first quarter of 2021 was 75.3 percent, compared to 80.5 percent in the first quarter of 2020, due primarily to favorable mortality experience and lower claims incidence in both the group long-term disability and group critical illness product lines, partially offset by higher claims incidence in the group life product line, resulting from the impacts of COVID-19. Sales decreased 13.3 percent to £14.3 million in the first quarter of 2021, compared to £16.5 million in the first quarter of 2020. Persistency in the group long-term disability product line was 87.0 percent for the first quarter of 2021, compared to 90.1 percent for the first quarter of 2020, resulting from pricing discipline. Persistency in the group life product line was 87.2 percent for the first quarter of 2021, compared to 86.3 percent for the first quarter of 2020. Persistency in the supplemental product line was 88.2 percent for the first quarter of 2021, compared to 91.2 percent for the first quarter of 2020.

Colonial Life Segment

Colonial Life reported a 9.6 percent decrease in adjusted operating income to $73.3 million in the first quarter of 2021, compared to $81.1 million in the first quarter of 2020. Premium income decreased 1.9 percent to $426.4 million in the first quarter of 2021, compared to $434.7 million in the first quarter of 2020, due to a decline in sales, partially offset by higher persistency. Net investment income was $37.7 million in the first quarter of 2021, which was consistent with the first quarter of 2020. The benefit ratio was 55.4 percent in the first quarter of 2021, compared to 52.4 percent in the first quarter of 2020, with unfavorable experience in the life product line, resulting from the impacts of COVID-19, partially offset by favorable experience in the accident, sickness, and disability line of business.

Sales decreased 9.2 percent to $90.2 million in the first quarter of 2021, compared to $99.3 million in the first quarter of 2020. Persistency in Colonial Life was 78.4 percent for the first quarter of 2021, compared to 76.8 percent for the first quarter of 2020.

Closed Block Segment

The Closed Block segment reported adjusted operating income of $97.0 million in the first quarter of 2021, which excludes the impacts from the second phase of the Closed Block individual disability reinsurance transaction of $139.3 million and the amortization of cost of reinsurance related to the Closed Block individual disability reinsurance transaction of $20.0 million, compared to $29.7 million in the first quarter of 2020. Premium income for this segment increased 3.0 percent to $251.7 million in the first quarter of 2021, compared to $244.4 million in the first quarter of 2020, due to premium rate increases on certain in-force business in the long-term care line of business, partially offset by continued policy terminations and maturities in the individual disability line of business. Net investment income decreased 11.6 percent to $297.2 million in the first quarter of 2021, compared to $336.1 million in the first quarter of 2020, due to a decrease in the level of invested assets supporting individual disability resulting from the reinsurance transaction, partially offset by higher miscellaneous investment income, primarily related to increases in the net asset values on our private equity partnerships, and an increase in the level of invested assets supporting long-term care. In connection with the completion of the second phase of the Closed Block individual disability reinsurance transaction, we recognized a net realized investment gain of $67.6 million.

The interest adjusted loss ratio for the long-term care line of business was 77.7 percent in the first quarter of 2021, compared to an interest adjusted loss ratio of 81.0 percent in the first quarter of 2020, driven primarily by higher claimant mortality, partially offset by higher submitted claims. The interest adjusted loss ratio for long-term care for the rolling twelve months ended March 31, 2021, excluding the update of assumptions during the fourth quarter of 2020, was 68.1 percent which is significantly below our long-term expected range. The interest adjusted loss ratio for the individual disability line of business, excluding the impacts from the reinsurance transaction, was 68.9 percent in the first quarter of 2021, compared to 84.5 percent in the first quarter of 2020, driven primarily by lower submitted claims.

Corporate Segment

The Corporate segment reported an adjusted operating loss of $38.9 million in the first quarter of 2021 compared to an adjusted operating loss of $45.9 million in the first quarter of 2020, resulting primarily from an increase in the level of invested assets and lower operating expenses.

OTHER INFORMATION

Shares Outstanding

The Company’s weighted average number of shares outstanding, assuming dilution, was 204.7 million for the first quarter of 2021, compared to 203.4 million for the first quarter of 2020. Shares outstanding totaled 204.2 million at March 31, 2021. The Company did not repurchase shares during the first quarter of 2021.

Capital Management

At March 31, 2021, the weighted average risk-based capital ratio for the Company’s traditional U.S. insurance companies was approximately 370 percent, and cash and marketable securities in the holding companies equaled $1,664 million.

Book Value

Book value per common share as of March 31, 2021 was $51.77, compared to $48.21 at March 31, 2020.

Outlook

As previously announced, the Company expects a modest decline in after-tax adjusted operating income per share for full-year 2021 compared to full-year 2020. The Company also anticipates a strong recovery in after-tax adjusted operating income per share in the second half of 2021 as the expected impacts of the COVID-19 pandemic subside.

NON-GAAP FINANCIAL MEASURES

We analyze our performance using non-GAAP financial measures. A non-GAAP financial measure is a numerical measure of a company’s performance, financial position, or cash flows that excludes or includes amounts that are not normally excluded or included in the most directly comparable measure calculated and presented in accordance with GAAP. The non-GAAP financial measure of "after-tax adjusted operating income" differs from net income as presented in our consolidated operating results and income statements prepared in accordance with GAAP due to the exclusion of net realized investment gains and losses and amortization of cost of reinsurance as well as certain other items as specified in the reconciliations in the Financial Highlights section below. We believe after-tax adjusted operating income is a better performance measure and better indicator of the profitability and underlying trends in our business.

Realized investment gains or losses depend on market conditions and do not necessarily relate to decisions regarding the underlying business of our segments. Our investment focus is on investment income to support our insurance liabilities as opposed to the generation of realized investment gains or losses. Although we may experience realized investment gains or losses which will affect future earnings levels, a long-term focus is necessary to maintain profitability over the life of the business since our underlying business is long-term in nature, and we need to earn the interest rates assumed in calculating our liabilities.

As previously discussed, we have exited a substantial portion of our Closed Block individual disability product line through the two phases of the reinsurance transaction that were executed in December 2020 and March 2021, respectively. As a result, we exclude the amortization of the cost of reinsurance that was recognized upon the exit of the business related to the ceded reserves for the cohort of policies on claim status. We believe that the exclusion of the amortization of the cost of reinsurance provides a better view of our results from our ongoing businesses.

We may at other times exclude certain other items from our discussion of financial ratios and metrics in order to enhance the understanding and comparability of our operational performance and the underlying fundamentals, but this exclusion is not an indication that similar items may not recur and does not replace net income or net loss as a measure of our overall profitability.

CONFERENCE CALL INFORMATION

Members of Unum Group senior management will host a conference call on Thursday, May 6, at 8:00 a.m. (Eastern Time) to discuss the results of operations for the first quarter. Topics may include forward-looking information, such as the Company’s outlook on future results, trends in operations, and other material information.

The dial-in number for the conference call is (866) 652-5200 for U.S. and Canada (Please ask to be joined to the Unum Group call). For international, the dial-in number is (412) 317-6060 (Please ask to be joined to the Unum Group call). A live webcast of the call will also be available at www.investors.unum.com in a listen-only mode. It is recommended that webcast viewers access the "Investors" section of the Company’s website and opt-in to the webcast approximately 5-10 minutes prior to the start of the call. A replay of the webcast will be available on the Company’s website. A replay of the call will also be available through Thursday, May 13 by dialing (877) 344-7529 (U.S.), (855) 669-9658 (Canada), or (412) 317-0088 (International) – pass code 10154518.

In conjunction with today’s earnings announcement, the Company’s Statistical Supplement for the first quarter of 2021 is available on the "Investors" section of the Company’s website.

On May 5, 2021 CTI BioPharma Corp. (CTI BioPharma) (NASDAQ: CTIC) reported that management plans to report its first quarter 2021 financial results on Tuesday, June 1, 2021, after the close of the U.S. financial markets (Press release, CTI BioPharma, MAY 5, 2021, View Source [SID1234579147]). Following the announcement, members of the management team will host a conference call and webcast to discuss the results and provide a general corporate update at 4:30 p.m. ET (1:30 p.m. PT).

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To access the live call by phone please dial (877) 735-2860 (domestic) or (602) 563-8791 (international); the conference ID is 9343326. A live audio webcast of the event may also be accessed through the "Investors" section of CTI’s website at www.ctibiopharma.com. A replay of the webcast will be available for 30 days following the event.

On May 5, 2021 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) reported financial results for the first quarter ended March 31, 2021, including sales of TAVALISSE (fostamatinib disodium hexahydrate) tablets, for the treatment of adults with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment (Press release, Rigel, MAY 5, 2021, View Source [SID1234579180]).

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"We continued to make significant progress in achieving important corporate and clinical milestones in the first quarter, which is a true testament to the dedication of our team to bring meaningful treatment options to those patients who need them the most," said Raul Rodriguez, Rigel’s president and CEO. "Following our recent announcement of positive topline data from the Phase 2 trial of fostamatinib in hospitalized patients with COVID-19 and our partnership with Eli Lilly for our RIP1 inhibitor program, we are excited by the prospects that the rest of 2021 holds for Rigel."

Business Update
In April 2021, Rigel reported positive topline data from the multi-center, Phase 2 clinical trial evaluating the safety of fostamatinib, Rigel’s oral spleen tyrosine kinase (SYK) inhibitor, for the treatment of hospitalized patients with COVID-19. The trial met its primary endpoint of safety, and showed broad and consistent improvement in numerous efficacy endpoints including mortality, ordinal scale assessment, and number of days in the ICU. The trial was conducted in collaboration with the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health (NIH), and Inova Health System. The NHLBI is expected to publish a full analysis of the trial data in a peer-reviewed journal. Rigel is discussing these results with health authorities, including the U.S. Food and Drug Administration (FDA), and intends to apply for Emergency Use Authorization (EUA) for fostamatinib for the treatment of hospitalized COVID-19 patients.

Rigel’s Phase 3 clinical trial to further evaluate fostamatinib in hospitalized patients with COVID-19 is currently enrolling. Rigel was awarded $16.5 million from the U.S. Department of Defense’s (DOD) Joint Program Executive Office for Chemical, Biological, Radiological and Nuclear Defense (JPEO-CBRND) to support this Phase 3 clinical trial. The study is designed to evaluate fostamatinib for prevention of progression to severe disease in hospitalized patients with COVID-19 without respiratory failure that have certain high-risk prognostic factors. This multi-center, double-blind, placebo-controlled study will randomly assign patients to either fostamatinib plus standard of care (SOC) or matched placebo plus SOC (1:1). Treatment will be administered orally twice daily for 14 days. There will be a follow-up period to day 60. The primary endpoint of this study is the proportion of subjects who progress to severe/critical disease within 29 days. In addition, Rigel’s COVID-19 program includes an investigator-sponsored trial currently being conducted by Imperial College London.

Rigel’s FORWARD study, a Phase 3 pivotal trial of TAVALISSE in patients with warm autoimmune hemolytic anemia (wAIHA), has enrolled 72 of 90 patients as of May 5, 2021. If approved, TAVALISSE has the potential to be the first to market therapy for patients with wAIHA. The FDA has granted Fast Track designation as well as Orphan Drug designation to TAVALISSE for the treatment of wAIHA.

In the first quarter of 2021, 1,599 bottles of TAVALISSE were shipped to patients and clinics, an increase of 14% year over year. Net product sales for the first quarter decreased 2% year over year to $12.4 million. During the quarter, the company experienced typical first quarter reimbursement issues such as the resetting of co-pays and the Medicare donut hole, along with physician and patient access issues created by the COVID-19 pandemic. Incrementally, the company’s net product sales were negatively impacted by a significant 235 bottle decrease in bottles remaining in its distribution channels compared to Q4 2020.

In April 2021, Rigel received a $125 million upfront cash payment from its collaboration with Eli Lilly and Company (Lilly), following the expiration of the waiting period under the Hart-Scott Rodino Antitrust Improvements Act of 1976. In February 2021, Rigel and Lilly entered into a global exclusive license agreement and strategic collaboration to develop and commercialize Rigel’s R552, a receptor-interacting serine/threonine-protein kinase 1 (RIP1) inhibitor, for all indications including autoimmune and inflammatory diseases. Rigel and Lilly are currently focused on the planning for the initiation of a Phase 2 clinical trial. Pursuant to the collaboration, Lilly will also lead all clinical development of central nervous system (CNS) penetrating RIP1 inhibitors. Rigel is eligible to receive up to an additional $835 million in potential development, regulatory and commercial milestone payments, as well as tiered royalties that will vary depending upon Rigel’s clinical development investment.

Rigel is also advancing the development of its IRAK1/4 program, where it intends to pursue both hematology/oncology and rare immune disease opportunities. Rigel has begun discussions with the FDA regarding initiating a Phase 2 clinical trial in low-risk myelodysplastic syndrome (MDS) and is also in discussions regarding academic medical collaborations in this indication. In rare immune diseases, the company is exploring opportunities including palmoplantar pustulosis (PPP), hidradenitis suppurativa (HS), and others.

Rigel’s partner Kissei Pharmaceutical Co., Ltd. (Kissei) has completed enrollment of its Phase 3 clinical trial of fostamatinib in adult Japanese patients with chronic ITP. In October 2018, Rigel and Kissei entered into an exclusive agreement to develop and commercialize fostamatinib in all current and potential indications in Japan, China, Taiwan, and the Republic of Korea.

Financial Update
For the first quarter of 2021, Rigel reported net income of $39.5 million, or $0.23 per basic share and $0.22 per diluted share, compared to net income of $21.2 million, or $0.13 per basic and diluted share, for the same period of 2020.

In the first quarter of 2021, total revenues were $81.0 million, consisting of $12.4 million in TAVALISSE net product sales, $65.6 million in contract revenues from collaborations and $3.0 million in government contract revenue.

Contract revenues from collaborations of $65.6 million for the first quarter of 2021 consisted of $60.6 million in revenue related to Rigel’s license agreement with Lilly, $4.0 million in revenue related to the grant of non-exclusive license of a certain patent to an unrelated third-party company, and $1.0 million in revenue for the delivery of drug supply under its collaboration agreement with Grifols. Government contract revenue was related to the income that Rigel recognized pursuant to the agreement it entered into in January 2021 with the DOD to support Rigel’s ongoing Phase 3 clinical trial of fostamatinib in hospitalized patients with COVID-19.

Rigel reported total costs and expenses of $39.3 million in the first quarter of 2021, compared to $34.7 million for the same period in 2020. The increase in costs and expenses was primarily due to increases in personnel-related costs, stock-based compensation expense, and research and development costs related to its ongoing Phase 3 clinical trial in hospitalized patients with COVID-19 and development of its IRAK 1/4 inhibitor program, partially offset by the decrease in research and development costs due to the completion of a Phase 1 clinical trial for its RIP1 inhibitor program.

As of March 31, 2021, Rigel had cash, cash equivalents and short-term investments of $39.3 million, compared to $57.3 million as of December 31, 2020. Cash, cash equivalents and short-term investments as of March 31, 2021, does not include the $125.0 million upfront payment received from Lilly in April 2021.

Conference Call and Webcast with Slides Today at 4:30pm Eastern Time
Rigel will hold a live conference call and webcast today at 4:30pm Eastern Time (1:30pm Pacific Time).

Participants can access the live conference call by dialing (877) 407-3088 (domestic) or (201) 389-0927 (international). The conference call and accompanying slides will also be webcast live and can be accessed from the Investor Relations section of the company’s website at www.rigel.com. The webcast will be archived and available for replay after the call via the Rigel website.

About ITP
In patients with ITP (immune thrombocytopenia), the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising and bleeding. People suffering with chronic ITP may live with an increased risk of severe bleeding events that can result in serious medical complications or even death. Current therapies for ITP include steroids, blood platelet production boosters (TPO-RAs) and splenectomy. However, not all patients respond to existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP.

About AIHA
Autoimmune hemolytic anemia (AIHA) is a rare, serious blood disorder in which the immune system produces antibodies that result in the destruction of the body’s own red blood cells. AIHA affects approximately 45,000 adult patients in the U.S. and can be a severe, debilitating disease. To date, there are no disease-targeted therapies approved for AIHA, despite the unmet medical need that exists for these patients. Warm antibody AIHA (wAIHA), the most common form of AIHA, is characterized by the presence of antibodies that react with the red blood cell surface at body temperature.

About COVID-19 & SYK Inhibition
COVID-19 is the infectious disease caused by Severe Acute Respiratory Syndrome Coronavirus-2 (SARS-CoV-2). SARS-CoV-2 primarily infects the upper and lower respiratory tract and can lead to acute respiratory distress syndrome (ARDS). Additionally, some patients develop other organ dysfunction including myocardial injury, acute kidney injury, shock resulting in endothelial dysfunction and subsequently micro and macrovascular thrombosis.1 Much of the underlying pathology of SARS-CoV-2 is thought to be secondary to a hyperinflammatory immune response associated with increased risk of thrombosis.2

SYK is involved in the intracellular signaling pathways of many different immune cells. Therefore, SYK inhibition may improve outcomes in patients with COVID-19 via inhibition of key Fc gamma receptor (FcγR) and c-type lectin receptor (CLR) mediated drivers of pathology, such as inflammatory cytokine release by monocytes and macrophages, production of neutrophil extracellular traps (NETs) by neutrophils, and platelet aggregation.3,4,5 Furthermore, SYK inhibition in neutrophils and platelets may lead to decreased thromboinflammation, alleviating organ dysfunction in critically ill patients with COVID-19.

For more information on Rigel’s comprehensive clinical program in COVID-19, go to: View Source

About R552
The investigational candidate, R552, is an orally available, potent and selective inhibitor of receptor-interacting serine/threonine-protein kinase 1 (RIP1). RIP1 is believed to play a critical role in necroptosis. Necroptosis is a form of regulated cell death where the rupturing of cells leads to the dispersion of their inner contents, which induces immune responses and enhances inflammation. In preclinical studies, R552 prevented joint and skin inflammation in a RIP1-mediated murine model of inflammation and tissue damage. The safety and efficacy of R552 has not been established by the FDA or any healthcare authority.

About R835
The investigational candidate, R835, is an orally available, potent and selective inhibitor of IRAK1 and IRAK4 that has been shown preclinically to block inflammatory cytokine production in response to toll-like receptor (TLR) and the interleukin-1 receptor (IL-1R) family signaling. TLRs and IL-1Rs play a critical role in the innate immune response, and dysregulation of these pathways can lead to a variety of inflammatory pathological conditions. R835 treatment demonstrates amelioration of clinical symptoms in multiple rodent models of inflammatory disease including psoriasis, arthritis, lupus, multiple sclerosis and gout. The safety and efficacy of R835 has not been established by the FDA or any healthcare authority.

About TAVALISSE
Indication
TAVALISSE (fostamatinib disodium hexahydrate) tablets is indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

Important Safety Information
Warnings and Precautions

Hypertension can occur with TAVALISSE treatment. Patients with pre-existing hypertension may be more susceptible to the hypertensive effects. Monitor blood pressure every 2 weeks until stable, then monthly, and adjust or initiate antihypertensive therapy for blood pressure control maintenance during therapy. If increased blood pressure persists, TAVALISSE interruption, reduction, or discontinuation may be required.
Elevated liver function tests (LFTs), mainly ALT and AST, can occur with TAVALISSE. Monitor LFTs monthly during treatment. If ALT or AST increase to >3 x upper limit of normal, manage hepatotoxicity using TAVALISSE interruption, reduction, or discontinuation.
Diarrhea occurred in 31% of patients and severe diarrhea occurred in 1% of patients treated with TAVALISSE. Monitor patients for the development of diarrhea and manage using supportive care measures early after the onset of symptoms. If diarrhea becomes severe (≥Grade 3), interrupt, reduce dose or discontinue TAVALISSE.
Neutropenia occurred in 6% of patients treated with TAVALISSE; febrile neutropenia occurred in 1% of patients. Monitor the ANC monthly and for infection during treatment. Manage toxicity with TAVALISSE interruption, reduction, or discontinuation.
TAVALISSE can cause fetal harm when administered to pregnant women. Advise pregnant women the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for at least 1 month after the last dose. Verify pregnancy status prior to initiating TAVALISSE. It is unknown if TAVALISSE or its metabolite is present in human milk. Because of the potential for serious adverse reactions in a breastfed child, advise a lactating woman not to breastfeed during TAVALISSE treatment and for at least 1 month after the last dose.
Drug Interactions

Concomitant use of TAVALISSE with strong CYP3A4 inhibitors increases exposure to the major active metabolite of TAVALISSE (R406), which may increase the risk of adverse reactions. Monitor for toxicities that may require a reduction in TAVALISSE dose.
It is not recommended to use TAVALISSE with strong CYP3A4 inducers, as concomitant use reduces exposure to R406.
Concomitant use of TAVALISSE may increase concentrations of some CYP3A4 substrate drugs and may require a dose reduction of the CYP3A4 substrate drug.
Concomitant use of TAVALISSE may increase concentrations of BCRP substrate drugs (eg, rosuvastatin) and P-Glycoprotein (P-gp) substrate drugs (eg, digoxin), which may require a dose reduction of the BCRP and P-gp substrate drug.
Adverse Reactions

Serious adverse drug reactions in the ITP double-blind studies were febrile neutropenia, diarrhea, pneumonia, and hypertensive crisis, which occurred in 1% of TAVALISSE patients. In addition, severe adverse reactions occurred including dyspnea and hypertension (both 2%), neutropenia, arthralgia, chest pain, diarrhea, dizziness, nephrolithiasis, pain in extremity, toothache, syncope, and hypoxia (all 1%).
Common adverse reactions (≥5% and more common than placebo) from FIT-1 and FIT-2 included: diarrhea, hypertension, nausea, dizziness, ALT and AST increased, respiratory infection, rash, abdominal pain, fatigue, chest pain, and neutropenia.

On May 5, 2021 bluebird bio, Inc. (NASDAQ: BLUE) reported financial results and business highlights for the first quarter ended March 31, 2021 and shared recent operational progress (Press release, bluebird bio, MAY 5, 2021, View Source [SID1234579190]).

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"Undoubtedly the highlight of last quarter at bluebird was the approval of Abecma, the first and only CAR T therapy approved for the treatment of relapsed or refractory multiple myeloma," said Nick Leschly, chief bluebird. "We and our colleagues at BMS are now full speed ahead and on track to begin treating patients this quarter. It has been an amazing journey and in many ways, we’re just getting started. While the oncology team has been delivering on Abecma, the severe genetic disease team met the moment. We quickly completed an investigation of the SUSAR of AML in our HGB-206 study of LentiGlobin gene therapy for SCD and determined that it was highly unlikely to be due to BB305 lentiviral vector. With these data and the other event changed from an MDS diagnosis to transfusion-dependent anemia, we are now quickly moving to engage with regulators with a goal of lifting the clinical holds in mid-2021. Amidst these challenges and work towards the planned separation, I want to commend and thank all birds for truly demonstrating anti-fragility and continuing to keep patients at the center of everything we do."

BUSINESS SEPARATION UPDATE

Today, bluebird bio is providing additional detail regarding the company’s planned business separation, which is targeted for completion by year-end 2021.

bluebird bio

bluebird bio is announcing that Tom Klima will join the company as chief commercial officer. Tom brings a track record of success across multiple commercial roles in oncology and rare diseases, most recently at Gamida Cell Ltd., where he served as chief commercial officer.

Additional members of the bluebird bio leadership team focused on severe genetic diseases will include:

Andrew Obenshain, chief executive officer (previously-disclosed)
Jason Cole, chief business officer
Rich Colvin, interim chief medical officer
Anne-Virginie Eggiman, senior vice president, regulatory science
"As we move towards separation, I’m pleased to have key leadership team members in place that are poised to bring bluebird bio to its next phase of success," said Andrew Obenshain. "With the addition of Tom Klima to lead our commercial efforts, we are rounding out our team of experts with a deep level of expertise from early stage clinical development to commercial delivery. I’m excited about our path forward and look forward to continuing to expand the bluebird team to bring gene therapy to patients with severe genetic diseases."

Oncology NewCo – 2seventy bio

Today, the company is announcing that Oncology NewCo will be named 2seventy bio and members of the leadership team will include:

Nick Leschly, chief executive officer (previously-disclosed)
Chip Baird, chief financial officer
Philip Gregory, chief scientific officer
Nicola Heffron, chief operating officer
"Two hundred seventy miles per hour is the maximum speed of human thought," said Nick Leschly, chief executive officer. "The name 2seventy was selected to signify this speed and our team’s translation of thought to action as we advance our next generation pipeline of transformative cell therapies to help cancer patients urgently in need."

RECENT HIGHLIGHTS

MULTIPLE MYELOMA

ABECMA FDA APPROVAL – On March 26, 2021, bluebird bio and Bristol-Myers Squibb announced that the U.S. Food and Drug Administration (FDA) approved Abecma (idecabtagene vicleucel; ide-cel) as the first B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy for the treatment of adult patients with relapsed or refractory multiple myeloma (RRMM) after four or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody. Abecma is a personalized immune cell therapy approved as a one-time infusion with a recommended dose range of 300 to 460 x 106 CAR-positive T cells.
KARMMA NEJM PUBLICATION– On February 24, 2021, bluebird bio and Bristol-Myers Squibb announced that results from the pivotal Phase 2 KarMMa study were published in The New England Journal of Medicine. The KarMMa study met its primary endpoint of overall response rate and key secondary endpoint of complete response rate. The data from the study demonstrates deep and durable responses with ide-cel treatment in triple-class exposed RRMM patients (n=128).
SICKLE CELL DISEASE

CLINICAL STUDIES UPDATE – On April 20, 2021, bluebird bio announced a revised diagnosis for the previously reported case of myelodysplastic syndrome (MDS) in its Phase 1/2 study of LentiGlobin for sickle cell disease (SCD) (bb1111). Upon further assessment, the treating investigator concluded this is not a case of MDS and revised the diagnosis to transfusion-dependent anemia. In addition, on March 10, 2021, bluebird bio reported that it is very unlikely the suspected unexpected serious adverse reaction (SUSAR) of acute myeloid leukemia (AML) reported in the HGB-206 study of LentiGlobin for SCD was related to the BB305 lentiviral vector (LVV). bluebird bio continues to work with regulators to resume its clinical studies in sickle cell disease as well as to remove the clinical hold for HGB-207 and HGB-212 clinical studies of beti-cel for β-thalassemia, with potential lift of all clinical holds in mid-2021.
CEREBRAL ADRENOLEUKODYSTROPHY

ELI-CEL DATA AT EBMT– On March 15, 2021, bluebird bio presented new data suggesting durability of response and a strong safety profile post elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy in patients with cerebral adrenoleukodystrophy (CALD) at the 47th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT 2021). Long-term results from the Phase 2/3 Starbeam study of eli-cel, showed that ninety percent of patients (27/30) are alive and free of major functional disabilities (MFDs) at 24 months or more of follow-up. In the 51 patients treated with eli-cel in clinical studies (ALD-102/LTF-304 and ALD-104) there were no reports of graft failure, graft rejection, GVHD, replication competent lentivirus or insertional oncogenesis.
PIPELINE

BILL & MELINDA GATES FOUNDATION GRANT TO EXPLORE IN-VIVO LVV APPLICATIONS – bluebird bio is announcing today that it has received a grant from the Bill & Melinda Gates Foundation to explore new, potentially transformative in vivo treatments for SCD using the Company’s proprietary lentiviral vector (LVV) platform. The funding will support the research and development of novel LVVs that target hematopoietic stem cells (HSCs) for in vivo administration to bring gene-based therapies for SCD and other potential indications to patients around the world who may have limited access to ex vivo and other emerging therapies. This research may also enable the application of in-vivo LVV approaches in other severe genetic diseases.
PSIOXUS PRE-CLINICAL DATA – On April 14, 2021, bluebird bio and PsiOxus Therapeutics presented preclinical data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021. The results showed synergistic activity between PsiOxus’ T-SIGn vector and bluebird bio’s CAR-T therapy in primary and metastatic solid tumors. A single IV cycle of PsiOxus’ T-SIGn vector enabled an otherwise non-effective dose of CAR-T cell therapy to clear primary and metastatic tumors in vivo.
COMPANY

BUSINESS OPERATIONS – On April 20, 2021 bluebird bio announced its decision to withdraw ZYNTEGLO (betibeglogene autotemcel, beti-cel) for transfusion-dependent β-thalassemia (TDT) from the German market because reimbursement negotiations in Germany did not result in a price for ZYNTEGLO that reflects the value of this one-time gene therapy with potential life-long benefit for people living with TDT. Due in part to this decision, the company also announced a targeted reshaping of its workforce intended to enable the company to advance its late-stage gene therapy programs. This reduction and reallocation of resources will allow the company to focus on priority European markets and streamline global operations going forward to ensure its ability to deliver gene therapies to patients.
UPCOMING ANTICIPATED MILESTONES

Regulatory Outlook

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 by year end.
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 adult, adolescent and pediatric 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 eli-cel in mid-2021. The company plans to receive European approval for eli-cel in patients with 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 clinical data from the ongoing pivotal Phase 2 KarMMa study of Abecma (ide-cel, bb2121) in patients with relapsed and refractory multiple myeloma to be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2021 (ASCO21) Virtual Scientific Program on June 4.
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.
Commercial and Foundation Building

Abecma first commercial patients treated in the first half of 2021.
Company

bluebird bio anticipates the separation of its severe genetic disease and oncology businesses into two independent, publicly traded companies (bluebird bio and 2seventy bio) to be completed by the end of 2021.
FIRST QUARTER 2021 FINANCIAL RESULTS

Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2021 and December 31, 2020 were $1.09 billion and $1.27 billion, respectively. The decrease in cash, cash equivalents and marketable securities is primarily related to cash used in support of ordinary course operating activities.
Revenues: Total revenues were $12.8 million for the three months ended March 31, 2021 compared to $21.9 million for the three months ended March 31, 2020. The decrease was primarily driven by a decrease in ide-cel license and manufacturing services revenue and a decrease in revenue recognized in connection with treating patients in the Phase 1 CRB-402 study of bb21217 under our agreements with BMS.
R&D Expenses: Research and development expenses were $154.5 million for the three months ended March 31, 2021 compared to $154.1 million for the three months ended March 31, 2020. The increase was primarily driven by increased costs incurred through the amended BMS collaboration as well as an increase in employee compensation, benefit, and other headcount related expenses. These increased costs were partially offset by a decrease in manufacturing costs.
SG&A Expenses: Selling, general and administrative expenses were $86.9 million for the three months ended March 31, 2021 compared to $73.2 million for the three months ended March 31, 2020. The increase was primarily driven by increased employee compensation, benefit, and other headcount related expenses, as well as an increase in consulting fees associated with the ongoing project to separate the Company’s severe genetic disease and oncology programs into two independently traded companies. These increased costs were partially offset by a decrease in costs related to commercial readiness activities due to delays in commercialization as a result of the COVID-19 pandemic and in light of safety events in the HGB-206 study of LentiGlobin gene therapy for SCD.
Net Loss: Net loss was $205.8 million for the three months ended March 31, 2021 compared to $202.6 million for the three months ended March 31, 2020.

On May 5, 2021 Crown Bioscience (CrownBio), a JSR Life Sciences company and leader in preclinical services that help biopharmaceutical companies accelerate new drug development programs reported that it has acquired OcellO B.V., a privately owned contract research organization located in the Netherlands (Press release, Crown Bioscience, MAY 5, 2021, View Source [SID1234579216]). Through the acquisition, CrownBio will expand its portfolio of in vitro services, integrating OcellO’s expertise in high content imaging alongside CrownBio’s in vitro and in vivo screens and immunotherapy assessment services. Financial terms are not being disclosed.

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"CrownBio and OcellO believe that the key to improving clinical research success is to utilize more patient-relevant models and assays that can generate reliable efficacy data as early as possible in the drug development process," said Armin Spura, PhD, Chief Executive Officer of CrownBio. "This acquisition brings together the complementary strengths of our two companies, enhancing our in vitro services portfolio and offering customers the ability to better evaluate multiple drug combinations and targets, across a wider range of tumor and cancer types, with improved predictability and clinical relevance moving from in vitro to in vivo phases of drug discovery."

The acquisition establishes CrownBio as the only global source licensed to offer customers specialized Hubrecht Organoid Technology (HUB) for drug development programs. Under the terms of the agreement, Leo Price, PhD, OcellO’s Chief Executive Officer and Chief Scientific Officer, will join CrownBio as Senior Vice President, In Vitro, to lead the Company’s in vitro business in the US and Europe.

"I am delighted to join CrownBio and help grow the company’s in vitro portfolio," said Dr. Price. "We will continue our shared heritage of innovation and dedication to customer service to serve complex needs in early-stage cancer drug development. Our combined expertise is particularly suited for disease-relevant large-scale screening, allowing our customers to evaluate multiple drug combinations in a rapid, scientifically rigorous, and cost-effective way."