Aeglea BioTherapeutics Reports Second Quarter 2019 Financial Results and Corporate Highlights

On August 6, 2019 Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company that engineers next-generation human enzymes to provide solutions for diseases with unmet medical need, reported financial results for the second quarter ended June 30, 2019 and corporate highlights (Press release, Aeglea BioTherapeutics, AUG 6, 2019, View Source [SID1234538214]).

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"We made tremendous progress this quarter with pegzilarginase, our lead product candidate for the treatment of Arginase 1 Deficiency (ARG1-D)," said Anthony G. Quinn, M.B. Ch.B., Ph.D., president and chief executive officer of Aeglea. "We dosed our first patient in our global pivotal Phase 3 PEACE trial, which is a major milestone toward providing our therapy to patients. The U.S. Food and Drug Administration’s (FDA) breakthrough therapy designation highlights the clinical relevance of the emerging data from the Phase 1/2 and Phase 2 open-label extension clinical trials as well as the potential of pegzilarginase to provide meaningful clinical improvements over available therapy."

Recent Highlights

Aeglea dosed the first patient in the Company’s global pivotal Phase 3 PEACE trial. The pivotal trial is intended to further evaluate the efficacy and safety of pegzilarginase, the Company’s lead product candidate for the treatment of ARG1-D, a progressive disease presenting in early childhood that results in severe complications and early mortality. The Company expects to report topline data from the PEACE trial in the first quarter of 2021.

The FDA granted Breakthrough Therapy Designation (BTD) to the Company’s lead product candidate, pegzilarginase, for the treatment of ARG1-D. The FDA’s BTD is intended to expedite the development and review of new therapies that are aimed at treating a serious or life-threatening condition when preliminary clinical evidence demonstrates the therapy may have substantial improvement on at least one clinically significant endpoint over available therapy. The designation was based on data from the completed Phase 1/2 clinical trial and the ongoing Phase 2 open-label extension study. Aeglea expects to continue discussions with the FDA regarding the pegzilarginase program and the Company’s next steps in the fourth quarter of 2019.

Interim data from 35 patients in the Company’s Phase 1/2 combination trial of pegzilarginase and KEYTRUDA in extensive disease small cell lung cancer revealed to date one complete response, four partial responses and 11 patients with stable disease. The combination trial was well tolerated, and safety observations were consistent with prior studies of pegzilarginase in patients with cancer. The Company has concluded enrollment and intends on submitting the results for presentation or publication after the final dataset becomes available.
Upcoming Events

Aeglea will present at the following conferences, with details regarding the date and time of the presentations and webcasts to be announced prior to the events.

Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium, September 3-6, Rotterdam, Netherlands
H.C. Wainwright & Co. 21st Annual Global Investment Conference, September 8-10, New York, NY
Second Quarter 2019 Financial Results

As of June 30, 2019, Aeglea had available cash, cash equivalents, marketable securities and restricted cash of $107.0 million. Based on Aeglea’s current operating plan, management believes it has sufficient capital resources to fund anticipated operations through the first quarter of 2021.

Research and development expenses totaled $14.8 million for the second quarter of 2019, compared with $9.1 million for the second quarter of 2018. The increase was primarily due to expanded personnel-related expenses, clinical development activity, investment in manufacturing and pre-commercial activities for Aeglea’s lead product candidate, pegzilarginase, and a ramp-up in manufacturing activities for the Company’s AEB4104 program for homocystinuria.

General and administrative expenses totaled $3.8 million for the second quarter of 2019, compared with $2.9 million for the second quarter of 2018. This increase was primarily due to additional employee headcount and compensation to support company growth.

Net loss totaled $18.0 million and $9.4 million for the second quarter of 2019 and 2018, respectively, with non-cash stock compensation expense of $1.2 million and $1.0 million for the second quarter of 2019 and 2018, respectively.

About Pegzilarginase in Arginase 1 Deficiency

Pegzilarginase is an enhanced human arginase that enzymatically depletes the amino acid arginine. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency, a rare debilitating disease presenting in childhood with persistent hyperargininemia, severe progressive neurological abnormalities and early mortality. Pegzilarginase is intended for use as an enzyme replacement therapy in patients to reduce elevated blood arginine levels. Aeglea’s Phase 1/2 and Phase 2 open-label extension data evaluating pegzilarginase in patients with Arginase 1 Deficiency demonstrated clinical improvements and sustained lowering of plasma arginine. Aeglea is currently recruiting patients for its single, global pivotal Phase 3 PEACE trial designed to assess the effects of treatment with pegzilarginase versus placebo over 24 weeks with a primary endpoint of plasma arginine reduction.

Blueprint Medicines to Present at 2019 Wedbush PacGrow Healthcare Conference

On August 6, 2019 Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported that Andy Boral, M.D., Ph.D., Blueprint Medicines’ Chief Medical Officer, will present a company overview at the 2019 Wedbush PacGrow Healthcare Conference in New York, NY on Tuesday, August 13, 2019 at 9:45 a.m. ET (Press release, Blueprint Medicines, AUG 6, 2019, View Source [SID1234538213]).

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A live webcast of the presentation will be available by visiting the Investors & Media section of Blueprint Medicines’ website at View Source A replay of the webcast will be archived on Blueprint Medicines’ website for 30 days following the presentation.

Oncternal Therapeutics Announces Opening of Randomized Phase 2 Study of Cirmtuzumab in Combination with Ibrutinib

On August 6, 2019 Oncternal Therapeutics, Inc., (Nasdaq: ONCT) a clinical-stage biotechnology company developing potential first-in-class product candidates for cancers with critical unmet medical need, reported that it has opened for enrollment its randomized Phase 2 study of cirmtuzumab, a ROR1-targeted monoclonal antibody, combined with ibrutinib in patients with chronic lymphocytic leukemia (CLL) (Press release, Oncternal Therapeutics, AUG 6, 2019, View Source [SID1234538212]). The decision to open Phase 2 of the company’s ongoing Phase 1/2 CIRLL (Cirmtuzumab and Ibrutinib targeting ROR1 for Leukemia and Lymphoma) clinical trial was triggered by favorable outcomes from the Part 1 dose-finding and Part 2 dose-confirming cohorts of the clinical trial, including an observed interim objective response rate (ORR) of 100% for the first 9 CLL patients with evaluable data receiving the recommended dosing regimen who have completed 12 weeks of cirmtuzumab plus ibrutinib treatment in Part 2, and a well-tolerated safety profile consistent with that seen with ibrutinib treatment alone.

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In June, the company presented data at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting, reporting that results from the first 12 patients with CLL treated in Part 1 of the Phase 1 portion of the study showed an observed interim objective response rate (ORR) of 91.7% for the combination of cirmtuzumab plus ibrutinib, including three patients with clinical or confirmed complete responses, and a well-tolerated safety profile consistent with that seen for ibrutinib treatment alone.

Included in the results presented at ASCO (Free ASCO Whitepaper) were preliminary results from six patients with mantle cell lymphoma (MCL), who were treated in a separate cohort of the CIRLL study. Data from this cohort will be presented at a future medical conference. One patient with MCL who had relapsed following an allogeneic stem cell transplant experienced a confirmed complete response (CR) after 3 months of cirmtuzumab plus ibrutinib treatment, including complete resolution of a large mediastinal mass. This CR appears to be durable, and has been confirmed after 6, 9 and 11 months of cirmtuzumab plus ibrutinib treatment.

The CIRLL clinical trial is supported by a grant from the California Institute for Regenerative Medicine (CIRM) and is being conducted in collaboration with the University of California at San Diego (UC San Diego).

"We are very pleased to be opening the randomized Phase 2 portion of the CIRLL study for patients with CLL and continue to be encouraged by the interim results from the study for both patients with CLL and patients with mantle cell lymphoma," said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO.

About the Study
The CIRLL Study (CIRM-0001) is a Phase 1/2 clinical trial evaluating cirmtuzumab in combination with ibrutinib in patients with CLL or MCL. Part 1 of the study was a Phase 1 dose-finding portion designed to determine the Phase 2 dose, or recommended dosing regimen (RDR). Part 2 was a Phase 1b expansion cohort to confirm the RDR. Interim analyses were specified for Part 1 and Part 2. Part 3 of the study, which is now open for enrollment, is a Phase 2 study in which approximately 90 patients with CLL will be randomized to receive either ibrutinib alone or ibrutinib plus cirmtuzumab, with a primary endpoint of complete response rate. An interim assessment of the first 12 patients with CLL enrolled in Part 1 of the study was presented as a poster at the 2019 ASCO (Free ASCO Whitepaper) Annual Meeting. Additional information about the CIRM-0001 study and other clinical studies of cirmtuzumab may be accessed at ClinicalTrials.gov.

About Cirmtuzumab
Cirmtuzumab is an investigational, potentially first-in-class monoclonal antibody targeting ROR1, or Receptor tyrosine kinase-like Orphan Receptor 1. Cirmtuzumab is currently in a Phase 1/2 clinical trial in combination with ibrutinib for the treatment of CLL and MCL, in a collaboration with the University of California San Diego School of Medicine and the California Institute for Regenerative Medicine. In addition, an investigator-initiated Phase 1 clinical trial of cirmtuzumab in combination with paclitaxel for women with metastatic breast cancer is being conducted at the UC San Diego School of Medicine. CIRM has also provided funding to support development programs for cirmtuzumab and a CAR-T product candidate that targets ROR1, which is currently in preclinical development as a potential treatment for hematologic cancers and solid tumors.

When expressed by hematologic malignancies such as CLL and MCL, ROR1 acts as a receptor for the tumor growth factor Wnt5a. Researchers at the UC San Diego School of Medicine discovered that targeting a critical epitope on ROR1 was key to inhibiting Wnt5a activation, specifically targeting ROR1 expressing tumors, and this finding led to the discovery of the potent and highly selective antitumor activity of cirmtuzumab observed in preclinical studies. Oncternal believes ROR1 is an attractive target for cancer therapy because it is an oncofetal antigen – a protein not normally expressed in adults, but which confers a survival and fitness advantage when reactivated and expressed by tumor cells. Preclinical data indicate that when cirmtuzumab binds to ROR1, it blocks Wnt5a signaling, induces differentiation of the tumor cells, and inhibits tumor cell proliferation, migration and survival. Cirmtuzumab is in clinical development and has not been approved by the U.S. Food and Drug Administration for any indication.

Can-Fite to Treat Advanced Liver Cancer Patients with Namodenoson Under Compassionate Use Setting in Israel

On August 6, 2019 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE:CFBI), a biotechnology company with a pipeline of proprietary small molecule drugs that address cancer, liver and inflammatory diseases, reported that a supply of Namodenoson has been manufactured and is ready for use in the treatment of advanced liver cancer patients under compassionate use at the Rabin Medical Center in Israel (Press release, Can-Fite BioPharma, AUG 6, 2019, View Source [SID1234538211]).

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Compassionate use allows doctors and their patients the option of early access to investigational new drugs, under closely controlled and monitored circumstances, when a patient who is facing serious illness has exhausted all available treatment options.

Salomon M. Stemmer, MD, the Principal Investigator of the Company’s prior Phase II liver cancer study said, "Given the evidence of clinical benefit of Namodenoson in patients with hepatocellular carcinoma and Child Pugh B7 to whom there is no accepted well established treatment, I plan to offer Namodenoson to certain HCC CPB7 patients in the compassionate use setting."

Can-Fite recently announced results from its Phase II study of Namodenoson in the treatment of advanced liver cancer. Namodenoson was found to increase overall survival in HCC patients with Child Pugh B7, the largest subpopulation of the study, as compared to placebo, even though the trial did not meet its primary endpoint.

An end of Phase II meeting with the U.S. Food and Drug Administration to review study data and to present the design of a Phase III clinical trial is expected soon. The FDA has granted Namodenoson both Orphan Drug and Fast Track status providing a pathway for accelerated approval based on unmet need in the treatment of advanced liver cancer. Fast Track designation offers advantages including more frequent meetings with the FDA and rolling review, which provides the opportunity to submit parts of its New Drug Application (NDA) for review prior to completing the entire application for commercialization. Orphan Drug designation includes 7-year market exclusivity following marketing approval, FDA assistance during the drug development process, and exemption of application fees.

About Namodenoson

Namodenoson is a small orally bioavailable drug that binds with high affinity and selectivity to the A3 adenosine receptor (A3AR). Namodenoson is being evaluated in Phase II trials for two indications, as a second line treatment for hepatocellular carcinoma, and as a treatment for non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). A3AR is highly expressed in diseased cells whereas low expression is found in normal cells. This differential effect accounts for the excellent safety profile of the drug.

BIO-TECHNE RELEASES FOURTH QUARTER FISCAL 2019 RESULTS

On August 6, 2019 Bio-Techne Corporation (NASDAQ:TECH) reported its financial results for the fourth quarter ended June 30, 2019 (Press release, Bio-Techne, AUG 6, 2019, View Source [SID1234538210]).

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Fourth Quarter FY2019 Snapshot

Fourth quarter organic growth of 7% (6% reported) to $191.7 million. Full year organic growth of 10% (11% reported) to $714.0 million.
GAAP EPS was $0.42 vs. $1.08 one year ago. Delivered adjusted earnings per share (EPS) of $1.25 vs. $1.34 one year ago. Full year GAAP EPS was $2.47 vs $3.31 one year ago. Full year adjusted EPS was $4.51 vs $4.54 in the prior year despite foreign currency exchange headwinds negatively impacting fiscal year results by $0.14 or 3%.
Protein Sciences Segment delivered 9% organic growth in FY19 Q4. The segment achieved full year organic growth of 13%.
The U.S. Food and Drug Administration granted breakthrough device designation to ExoDx Prostate IntelliScore (EPI) making it the first exosome-based liquid biopsy test to receive a breathrough device designation.
Acquired B-MoGen Biotechnologies Inc. with technology specializing in solving the most complex gene editing problems with proprietary, cutting edge non-viral gene editing and delivery tools.
The company’s consolidated financial statements are prepared in accordance with accounting principles generally accepted in the United States (GAAP). Adjusted dilutive EPS, adjusted net earnings, adjusted gross margin, adjusted operating income, and adjusted operating margin are non-GAAP measures that exclude certain items detailed later in this press release under the heading "Use of non-GAAP Adjusted Financial Measures." A reconciliation of GAAP to non-GAAP financial measures is included in this press release.

"We are pleased to deliver strong results in the fourth quarter and finish a phenomenal fiscal year 2019 with 10% organic growth," said Chuck Kummeth, President and Chief Executive Officer of Bio-Techne. "Our teams executed well all year on both the top and bottom-line, exceeding market growth in our portfolio of research reagents and instruments."

Kummeth added, "While our Diagnostics tools division was adversely impacted this quarter by the timing of OEM order flow, our Genomics division continues to be in full recovery from earlier in the year and again, topped 20% organic growth in Q4. We also received exciting news that our EPI liquid-biopsy test for prostate cancer was granted breakthrough device designation by the U.S. FDA. To date, over 25,000 EPI tests have already been conducted. Now with FDA support and the draft Local Coverage Decision for EPI issued in May by the National Government Services (NGS), ExosomeDx is in strong position for a full commercialization ramp in FY20."

Kummeth concluded, "All in all, a solid quarter and a truly outstanding year for the company. I am delighted with the performance of our entire Bio-Techne team."

Fourth Quarter Fiscal 2019

Revenue

Net sales for the fourth quarter increased 6% to $191.7 million. Organic growth was 7%, with currency translation having an unfavorable impact of 2% and acquisitions contributing 1% to revenue growth.

GAAP Earnings Results

GAAP EPS was $0.42 per diluted share, versus $1.08 in the same quarter last year. The decrease was driven by changes in the fair value of our CCXI investment, which impacted year over year GAAP EPS by $0.91 per share, partially offset by a gain on our historical investment in B-MoGen of $0.09, which was recognized upon acquisition. GAAP operating income for the fourth quarter of fiscal 2019 increased 1% to $43.3 million, compared with $42.9 million in the fourth quarter of fiscal 2018. GAAP operating margin was 22.6%, compared to 23.8% in the fourth quarter of fiscal 2018. GAAP operating margin compared to prior year was impacted by negative margin acquisitions, namely ExosomeDx.

Non-GAAP Earnings Results

Adjusted EPS was $1.25 per diluted share, versus $1.34 in the same quarter last year. Adjusted operating margin for the fourth quarter of fiscal 2019 decreased to 35.1%, compared with 39.5% in the fourth quarter of fiscal 2018. Adjusted operating margin compared to prior year was impacted by negative margin acquisitions, namely ExosomeDx.

Full Year Fiscal 2019

Revenue

Net sales for the full year fiscal 2019 increased 11% to $714.0 million. Organic growth was 10%, with currency translation having an unfavorable impact of 1% and acquisitions contributing 2% to revenue growth.

GAAP Earnings Results

GAAP EPS decreased to $2.47 per diluted share, versus $3.31 last fiscal year. GAAP operating income for full year fiscal 2019 increased 8% to $146.7 million, compared with $136.2 million in the full year fiscal 2018. GAAP operating margin was 20.5%, compared to 21.2% in the full year fiscal 2018. GAAP operating margin compared to prior year was impacted by unfavorable foreign currency exchange rates and negative margin acquisitions, namely ExosomeDx.

Non-GAAP Earnings Results

Adjusted EPS was $4.51 per diluted share, versus $4.54 in full fiscal year 2018. Adjusted operating margin for full fiscal year 2019 decreased to 34.1%, compared with 37.1% in full year fiscal 2018. Adjusted operating margin compared to prior year was impacted impacted by unfavorable foreign currency exchange rates and negative margin acquisitions, namely ExosomeDx.

Segment Results

Management uses adjusted operating results to monitor and evaluate performance of the Company’s business segments, as highlighted below. Since these results are used for this purpose, they are also considered to be prepared in accordance with GAAP.

Protein Sciences Segment

The Company’s Protein Sciences segment is one of the world’s leading suppliers of specialized proteins such as cytokines and growth factors, immunoassays, antibodies and reagents, to the biotechnology community. Additionally, the segment provides an array of platforms useful in various areas of protein analysis. Protein Sciences segment’s fourth quarter fiscal 2019 net sales were $143.4 million, an increase of 7% from $133.9 million for the fourth quarter of fiscal 2018. Organic growth for the segment was 9%, with currency translation having an unfavorable impact of 2% on revenue growth. Protein Sciences segment’s operating margin was 45.4% in the fourth quarter of fiscal 2019 compared to 44.5% in the fourth quarter of fiscal 2018. Segment operating margin compared to the prior year was positively impacted by volume leverage and operational productivity.

Protein Sciences segment’s full year fiscal 2019 net sales were $543.2 million, an increase of 13% from $482 million for fiscal 2018. Organic growth for the segment was 13% for the fiscal year, with currency translation having an unfavorable impact of 2% on revenue growth and acquisitions contributing 2%. Protein Sciences segment’s operating margin was 44.4% in fiscal 2019 compared to 43.5% in fiscal 2018. The higher operating margin was driven by strong volume leverage and operational productivity.

Diagnostics and Genomics Segment

The Company’s Diagnostics and Genomics segment provides blood chemistry and blood gas quality controls, hematology instrument controls, diagnostics immunoassays and other bulk and custom reagents for the in vitro diagnostic market. The Diagnostics and Genomics segment also develops and provides in situ hybridization products as well as exosome-based diagnostics for various pathologies, including prostate cancer. The Diagnostics and Genomics segment’s fourth quarter fiscal 2019 net sales were $48.5 million, an increase of 4% from $46.6 million for the fourth quarter of fiscal 2018. Organic growth for the segment was 2%, with currency translation having an unfavorable impact of 1% on revenue growth and acquisitions contributing 3% to revenue growth. The Diagnostics and Genomics segment operating margin was 10.3% in the fourth quarter of fiscal 2019 compared to 27.2% in the fourth quarter of fiscal 2018. Segment operating margin compared to the prior year was impacted by negative operating margins for acquisitions made in the segment, namely ExosomeDx.

The Diagnostics segment’s full year fiscal 2019 net sales were $171.7 million, an increase of 7% from $161.2 million for fiscal 2018. Organic growth for the segment was 4%, with acquisitions contributing 3% to revenue growth. The Diagnostics segment’s operating margin was 5.9% in fiscal 2019 compared to 22.0% in fiscal 2018. Operating margin was impacted by negative operating margins for acquisitions made in the segment, namely ExosomeDx.

Conference Call

Bio-Techne will host an earnings conference call today, Tuesday, August 6, 2019, at 8:00 a.m. CST. To listen, please dial 1-888-394-8218 or 1-323-701-0225 for international callers, and reference conference ID 9336552. A recorded rebroadcast will be available for interested parties unable to participate in the live conference call. To access the replay, U.S. callers should dial 1-844-512-2921 or international callers should dial 1-412-317-6671, and enter the replay access code 9336552. The replay can also be accessed by going to: View Source

The replay will be available from 11:00 a.m. CDT on Tuesday, August 6, 2019, until 11:00 p.m. CDT on Friday, September 6, 2019.

Use of non-GAAP Adjusted Financial Measures:

This press release contains financial measures that have not been calculated in accordance with accounting principles generally accepted in the U.S. (GAAP). These non-GAAP measures include:

Organic Growth
Adjusted diluted earnings per share
Adjusted net earnings
Adjusted gross margin
Adjusted operating income
Adjusted operating margin
We provide these measures as additional information regarding our operating results. We use these non-GAAP measures internally to evaluate our performance and in making financial and operational decisions, including with respect to incentive compensation. We believe that our presentation of these measures provides investors with greater transparency with respect to our results of operations and that these measures are useful for period-to-period comparison of results.

Our non-GAAP financial measure of organic growth represents revenue growth excluding revenue from acquisitions within the preceding 12 months as well as the impact of foreign currency. Excluding these measures provides more useful period-to-period comparison of revenue results as it excludes the impact of foreign currency exchange rates, which can vary significantly from period to period, and revenue from acquisitions that would not be included in the comparable prior period.

Our non-GAAP financial measures for adjusted gross margin, adjusted operating margin, and adjusted net earnings, in total and on a per share basis, exclude the costs recognized upon the sale of acquired inventory, amortization of acquisition intangibles, and acquisition related expenses. The Company excludes amortization of purchased intangible assets and purchase accounting adjustments, including costs recognized upon the sale of acquired inventory and acquisition-related expenses, from this measure because they occur as a result of specific events, and are not reflective of our internal investments, the costs of developing, producing, supporting and selling our products, and the other ongoing costs to support our operating structure. Additionally, these amounts can vary significantly from period to period based on current activity.

The Company’s non-GAAP adjusted operating margin and adjusted net earnings, in total and on a per share basis, also excludes stock-based compensation expense, which is inclusive of the employer portion of payroll taxes on those stock awards, restructuring, impairments of equity method investments, gain and losses from investments, and certain adjustments to income tax expense. Stock-based compensation is excluded from non-GAAP adjusted net earnings because of the nature of this charge, specifically the varying available valuation methodologies, subjective assumptions, variety of award types, and unpredictability of amount and timing of employer related tax obligations. Impairments of equity investments are excluded as they are not part of our day-to-day operating decisions. Additionally, gains and losses from other investments that are either isolated or cannot be expected to occur again with any predictability are excluded. Costs related to restructuring activities, including reducing overhead and consolidating facilities, are excluded because we believe they are not indicative of our normal operating costs. The Company independently calculates a non-GAAP adjusted tax rate to be applied to the identified non-GAAP adjustments considering the impact of discrete items on these adjustments and the jurisdictional mix of the adjustments. In addition, the tax impact of other discrete and non-recurring charges which impact our reported GAAP tax rate are adjusted from net earnings. We believe these tax items can significantly affect the period-over-period assessment of operating results and not necessarily reflect costs and/or income associated with historical trends and future results.

Investors are encouraged to review the reconciliations of adjusted financial measures used in this press release to their most directly comparable GAAP financial measures as provided with the financial statements attached to this press release.