Regeneron Reports First Quarter 2019 Financial and Operating Results

On May 7, 2019 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported financial results for the first quarter of 2019 and provided a business update (Press release, Regeneron, MAY 7, 2019, View Source [SID1234535804]).

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"In the first quarter, aggregate sales of all Regeneron-invented products, recorded by the Company and its collaborators, were $2.27 billion, an increase of 23% over the same period last year. This was driven by an 8% increase in EYLEA net sales, 185% increase in Dupixent net sales, and a strong initial launch for Libtayo in advanced cutaneous squamous cell carcinoma," said Leonard S. Schleifer, M.D., Ph.D., President and Chief Executive Officer of Regeneron. "We continue to unlock the full potential of Dupixent, which is now FDA-approved in atopic dermatitis and asthma in both adults and adolescents and is currently under Priority Review by the FDA for chronic rhinosinusitis with nasal polyps. Regeneron also continues to invest in a broad immuno-oncology portfolio. At the June European Hematology Association (EHA) (Free EHA Whitepaper) meeting, we look forward to presenting updated promising results of the Phase 1 study of REGN1979 in relapsed or refractory B-cell non-hodgkin lymphoma, including in patients who have failed previous CAR-T therapy."

Business Highlights

Key Pipeline Progress
Regeneron has twenty product candidates in clinical development, including five of the Company’s U.S. Food and Drug Administration (FDA) approved products for which it is investigating additional indications. Updates from the clinical pipeline include:
EYLEA (aflibercept) Injection

In April 2019, the Company resubmitted a supplemental Biologics License Application (sBLA) for EYLEA in a pre-filled syringe.

The EYLEA sBLA for the treatment of diabetic retinopathy has a target action date of May 13, 2019.

Dupixent (dupilumab)

In March 2019, the FDA approved Dupixent for adolescent patients 12 to 17 years of age with moderate-to-severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable.

The FDA accepted for priority review the sBLA for Dupixent as an add-on maintenance treatment for adults with inadequately controlled severe chronic rhinosinusitis with nasal polyps (CRSwNP), with a target action date of June 26, 2019. The Company and Sanofi have also submitted a European Marketing Authorization Application (MAA) for CRSwNP.

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for Dupixent, recommending it be approved for use in adults and adolescents 12 years and older as add-on maintenance treatment for severe asthma.

Initiated a Phase 3 study in chronic obstructive pulmonary disease (COPD).

Libtayo (cemiplimab)

The European Medicines Agency’s CHMP recommended conditional approval for Libtayo for the treatment of adult patients with metastatic or locally advanced cutaneous squamous cell carcinoma (CSCC) who are not candidates for curative surgery or curative radiation.

REGN1979, a bispecific antibody against CD20 and CD3

The Company expects to begin a potentially pivotal Phase 2 study in advanced follicular lymphoma this quarter, and a potentially pivotal Phase 2 study in diffuse large B-cell lymphoma (DLBCL) later this year.

At the June European Hematology Association (EHA) (Free EHA Whitepaper) meeting, the Company plans to present updated results of the Phase 1 study in relapsed or refractory B-cell non-hodgkin lymphoma, including in patients who have failed previous CAR-T therapy.

Praluent (alirocumab)

In March 2019, the European Commission approved a new indication for Praluent to reduce cardiovascular risk in adults with established atherosclerotic cardiovascular disease (ASCVD) by lowering low-density lipoprotein cholesterol (LDL-C) levels as an adjunct to correction of other risk factors.

In April 2019, the FDA approved a new indication for Praluent to reduce the risk of heart attack, stroke, and unstable angina requiring hospitalization in adults with established cardiovascular disease.

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Beginning in March 2019, Praluent was made available for both the 75 mg and 150 mg doses at a U.S. list price of $5,850 annually, a 60% reduction from the original price.

Business Development Update

In April 2019, the Company entered into a collaboration with Alnylam Pharmaceuticals, Inc. to discover, develop, and commercialize new RNA interference (RNAi) therapeutics for diseases of the eye and central nervous system, in addition to a select number of targets expressed in the liver. Under the terms of the agreement, the Company is obligated to make an up-front payment of $400 million and purchase $400 million of Alnylam common stock. In addition, the Company will provide Alnylam with a specified amount of funding at program initiation and at lead candidate designation, and Alnylam is eligible to receive up to $200 million in clinical proof-of-principle milestones.

First Quarter 2019 Financial Results

Product Revenues: Net product sales were $1.104 billion in the first quarter of 2019, compared to $988 million in the first quarter of 2018. EYLEA net product sales in the United States were $1.074 billion in the first quarter of 2019, compared to $984 million in the first quarter of 2018. Overall distributor inventory levels for EYLEA in the United States remained within the Company’s one-to-two-week targeted range.

Total Revenues: Total revenues, which include product revenues described above, increased by 13% to $1.712 billion in the first quarter of 2019, compared to $1.512 billion in the first quarter of 2018. Total revenues include Sanofi and Bayer collaboration revenues(6) of $523 million in the first quarter of 2019, compared to $437 million in the first quarter of 2018. The increase in Sanofi collaboration revenue in the first quarter of 2019 was primarily due to the Company’s share of lower losses of collaboration antibodies, primarily driven by higher net product sales of Dupixent. This increase was partly offset by a decrease in reimbursement of research and development costs under the Immuno-oncology Discovery and Development Agreement with Sanofi, as the amended agreement narrowed the scope of reimbursable activities to the BCMAxCD3 and MUC16xCD3 programs.

Refer to Table 4 for a summary of collaboration and other revenue.

Research and Development (R&D) Expenses: GAAP R&D expenses were $642 million in the first quarter of 2019, compared to $499 million in the first quarter of 2018. The higher R&D expenses in the first quarter of 2019 were principally due to additional costs incurred in connection with our earlier-stage pipeline, an increase in Libtayo development expenses, higher clinical manufacturing costs, and higher headcount and headcount-related costs. In the first quarter of 2019, R&D-related non-cash share-based compensation expense was $59 million, compared to $41 million in the first quarter of 2018.

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Selling, General, and Administrative (SG&A) Expenses: GAAP SG&A expenses were $411 million in the first quarter of 2019, compared to $331 million in the first quarter of 2018. The higher SG&A expenses in the first quarter of 2019 were primarily due to higher headcount and headcount-related costs, higher contributions to independent not-for-profit patient assistance organizations, and an increase in commercialization-related expenses for Dupixent. In the first quarter of 2019, SG&A-related non-cash share-based compensation expense was $44 million, compared to $35 million in the first quarter of 2018.

Cost of Collaboration and Contract Manufacturing (COCM): GAAP COCM expenses were $108 million in the first quarter of 2019, compared to $46 million in the first quarter of 2018. The increase in COCM was primarily due to higher expenses in connection with process validation at our Limerick manufacturing facility, higher inventory write-offs and reserves, and the recognition of manufacturing costs associated with higher sales of Dupixent.

Other Income (Expense): GAAP other income (expense), net, in the first quarter of 2019 and 2018 includes the recognition of $43 million and $9 million, respectively, of net gains on equity securities.

Income Taxes: In the first quarter of 2019, GAAP income tax expense was $85 million and the effective tax rate was 15.6%, compared to $107 million and 18.3% in the first quarter of 2018. The effective tax rate for the first quarter of 2019 was positively impacted, compared to the U.S. federal statutory rate, primarily by the federal tax credit for research activities, stock-based compensation, the foreign-derived intangible income deduction, and income earned in foreign jurisdictions with tax rates lower than the U.S. federal statutory rate.

GAAP and Non-GAAP Net Income(2): GAAP net income was $461 million, or $4.23 per basic share and $3.99 per diluted share, in the first quarter of 2019, compared to GAAP net income of $478 million, or $4.44 per basic share and $4.16 per diluted share, in the first quarter of 2018.

Non-GAAP net income was $518 million, or $4.75 per basic share and $4.45 per diluted share, in the first quarter of 2019, compared to non-GAAP net income of $537 million, or $4.99 per basic share and $4.67 per diluted share, in the first quarter of 2018.

A reconciliation of the Company’s GAAP to non-GAAP results is included in Table 3 of this press release.

Regeneron records net product sales of EYLEA in the United States. Outside the United States, EYLEA net product sales comprise sales by Bayer in countries other than Japan and sales by Santen Pharmaceutical Co., Ltd. in Japan under a co-promotion agreement with an affiliate of Bayer. The Company recognizes its share of the profits (including a percentage on sales in Japan) from EYLEA sales outside the United States within "Bayer collaboration revenue" in its Statements of Operations.

This press release uses non-GAAP net income, non-GAAP net income per share, non-GAAP unreimbursed R&D, and non-GAAP SG&A, which are financial measures that are not calculated in accordance with U.S. Generally Accepted Accounting Principles ("GAAP"). These non-GAAP financial measures are computed by excluding certain non-cash and other items from the related GAAP financial measure. Non-GAAP adjustments also include the estimated income tax effect of reconciling items.

The Company makes such adjustments for items the Company does not view as useful in evaluating its operating performance. For example, adjustments may be made for items that fluctuate from period to period based on factors that are not within the Company’s control (such as the Company’s stock price on the dates share-based grants are issued or changes in the fair value of the Company’s equity investments) or items that are not associated with normal, recurring operations (such as changes in applicable laws and regulations). Management uses these non-GAAP measures for planning, budgeting, forecasting, assessing historical performance, and making financial and operational decisions, and also provides forecasts to investors on this basis. Additionally, such non-GAAP measures provide investors with an enhanced understanding of the financial performance of the Company’s core business operations. However, there are limitations in the use of these and other non-GAAP financial measures as they exclude certain expenses that are recurring in nature. Furthermore, the Company’s non-GAAP financial measures may not be comparable with non-GAAP information provided by other companies. Any non-GAAP financial measure presented by Regeneron should be considered supplemental to, and not a substitute for, measures of financial performance prepared in accordance with GAAP. A reconciliation of the Company’s historical GAAP to non-GAAP results is included in Table 3 of this press release.

The Company’s 2019 financial guidance does not assume the completion of any significant business development transactions not completed as of the date of this press release (other than the collaboration with Alnylam Pharmaceuticals, Inc. discussed above).

Unreimbursed R&D represents R&D expenses reduced by R&D expense reimbursements from the Company’s collaborators and/or customers.

The Company’s collaborators provide it with estimates of the collaborators’ respective sales and the Company’s share of the profits or losses from commercialization of products for the most recent fiscal quarter. The Company’s estimates for such quarter are reconciled to actual results in the subsequent fiscal quarter, and the Company’s share of the profit or loss is adjusted on a prospective basis accordingly, if necessary.

Conference Call Information

Regeneron will host a conference call and simultaneous webcast to discuss its first quarter 2019 financial and operating results on Tuesday, May 7, 2019, at 8:30 AM. To access this call, dial (800) 708-4539 (U.S.) or (847) 619-6396 (International). A link to the webcast may be accessed from the "Investors and Media" page of Regeneron’s website at www.regeneron.com. A replay of the conference call and webcast will be archived on the Company’s website and will be available for 30 days.

Pacira BioSciences to Present at Two Healthcare Conferences in May

On May 7, 2019 Pacira BioSciences, Inc. (NASDAQ: PCRX) reported that it will present at the following two healthcare conferences (Press release, Pacira Pharmaceuticals, MAY 7, 2019, View Source;p=RssLanding&cat=news&id=2397477 [SID1234535803]):

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Bank of America Merrill Lynch 2019 Healthcare Conference in Las Vegas, on Tuesday, May 14 at 3:00 PM PT (6:00 PM ET)
2019 RBC Capital Markets Global Healthcare Conference in New York, on Wednesday, May 22 at 10:30 AM ET
Live audio of the presentations can be accessed by visiting the "Events" page of the company’s website at investor.pacira.com. A replay of the webcasts will also be available for two weeks following the events.

Onconova Therapeutics, Inc. to Provide Corporate Update and First Quarter 2019 Financial Results

On May 7, 2019 Onconova Therapeutics, Inc. (NASDAQ: ONTX), a Phase 3 stage biopharmaceutical company focused on discovering and developing novel small molecule drug candidates to treat cancer, with a primary focus on Myelodysplastic Syndromes (MDS), reported that the Company will release its first quarter 2019 financial results on Tuesday, May 14, 2019 before the market opens (Press release, Onconova, MAY 7, 2019, View Source [SID1234535802]). The Company will host a conference call on Tuesday, May 14, 2019, at 9 a.m. Eastern Time to discuss these results. Interested parties may access the call by dialing toll-free (855) 428-5741 from the U.S. or (210) 229-8823 internationally and using conference ID 4275108. The call will also be webcast live. Please click here to access the webcast on the Investor Relations page of the Company’s website. A replay will be available for 90 days.

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About Myelodysplastic Syndromes

Myelodysplastic syndromes (MDS) are conditions that can occur when the blood-forming cells in the bone marrow become dysfunctional and thus produce an inadequate number of circulating blood cells. It is frequently associated with the presence of blasts or leukemic cells in the marrow. This leads to low numbers of one or more types of circulating blood cells, and to the need for blood transfusions. In MDS, some of the cells in the bone marrow are abnormal (dysplastic) and may have genetic abnormalities associated with them. Different cell types can be affected, although the most common finding in MDS is a shortage of red blood cells (anemia). Patients with higher-risk MDS may progress to the development of acute leukemia.

Insmed Reports First Quarter 2019 Financial Results and Provides Business Update

On May 7, 2019 Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, reported financial results for the first quarter ended March 31, 2019 and provided a business update (Press release, Insmed, MAY 7, 2019, View Source [SID1234535801]).

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"We are very excited about the progress this quarter with the U.S. launch of ARIKAYCE (amikacin liposome inhalation suspension), the first and only FDA-approved treatment for patients with refractory MAC lung disease, including steady increases in new patients and prescribers, strong anecdotal feedback from the MAC lung disease community, and positive payer trends," commented Will Lewis, Chairman and Chief Executive Officer of Insmed. "In line with our strategic priorities, we are continuing to work with the FDA on the design of a post-marketing confirmatory study of ARIKAYCE, which is required for the full U.S. approval of ARIKAYCE and will be conducted in a frontline setting; continuing our global expansion to support potential regulatory filings for ARIKAYCE in Europe and Japan; and building and advancing a promising pipeline with the potential to bring much-needed therapies to patients with serious and rare diseases."

First Quarter 2019 Financial Results

·Total revenue for the first quarter ended March 31, 2019 was $21.9 million, comprising U.S. net sales of $21.0 million and ex-U.S. net sales of $0.9 million. The ex-U.S. net product sales include $0.8 million from the Temporary Authorization for Use (Autorisation Temporaire d’Utilisation or ATU) program in France and $0.1 million from the named patient program in Germany, both compassionate use programs.

· Cost of product revenues (excluding amortization of intangible assets) was $4.2 million for the first quarter of 2019.

Research and development expenses were $31.2 million for the first quarter of 2019, compared with $30.1 million for the first quarter of 2018.

· Selling, general and administrative expenses for the first quarter of 2019 were $54.8 million, compared with $32.7 million for the first quarter of 2018. The increase was primarily due to an increase in headcount, including the hiring of our field force, and

higher expenses related to commercial activities for ARIKAYCE, including non-branded disease awareness, patient support activities, and field operations.

· For the first quarter of 2019, Insmed reported a net loss of $74.2 million, or $0.96 per share, compared with a net loss of $68.5 million, or $0.89 per share, for the first quarter of 2018.

Recent Corporate Developments & Program Highlights

Strong Start to U.S. ARIKAYCE Launch

ARIKAYCE was granted accelerated approval by the U.S. Food and Drug Administration (FDA) on September 28, 2018, for the treatment of refractory Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen for adult patients who have limited or no alternative treatment options.

The Company expects to complete the design and protocol of the confirmatory clinical study during the first half of 2019 required for the full U.S. approval of ARIKAYCE by the FDA, which is intended to support the use of ARIKAYCE in a front-line setting for patients with MAC lung disease.

Data from Phase 3 CONVERT Study Accepted as Late-Breaker at ATS

Insmed announced in April that data on the sustainability and durability of culture conversion from the Phase 3 CONVERT Study of ARIKAYCE will be presented in a late-breaking oral session at the American Thoracic Society (ATS) International Conference on May 20, 2019. Additional data on ARIKAYCE as well as on Insmed’s pipeline candidates will also be presented at the meeting, taking place May 17-22 in Dallas.

Additional U.S. Patent Granted for ARIKAYCE

In April, the U.S. Patent and Trademark Office issued its 10th patent to Insmed for ARIKAYCE in MAC lung disease. The claims of the patent relate in part to methods for treating MAC lung disease via administration of ARIKAYCE to patients previously unresponsive to MAC therapy. This is the second U.S. patent for ARIKAYCE that provides intellectual property protection through 2035.

Global Expansion Under Way

Insmed is continuing its global expansion efforts to support potential regulatory filings for ARIKAYCE in Europe in mid-2019 and in Japan in the first half of 2020. The Company has hired Neil Hughes as Head of Europe, Middle East and Africa, effective June 1, to advance our launch preparations in Europe. Mr. Hughes joins Insmed from Shire, where he most recently served as general manager of endocrinology.

The Company is also progressing with the buildout of its new, state-of-the-art corporate headquarters in Bridgewater, NJ, with an anticipated move during the second half of 2019.

Insmed continues to invest in the buildout of an additional third-party manufacturing facility with Patheon UK Limited to increase the long-term production capacity for ARIKAYCE commercial inventory.

Enrollment Target Reached for WILLOW Study

Insmed has reached its enrollment target of 240 patients in the six-month Phase 2 WILLOW study of INS1007 for patients with non-CF bronchiectasis. The Company also plans to advance INS1007 into Phase 2 trials for the potential treatment of granulomatosis with polyangiitis (GPA).

Financial Guidance and Balance Sheet

As of March 31, 2019, Insmed had cash and cash equivalents of $420.2 million. The Company’s total costs and expenses for the first quarter of 2019 were $91.4 million, compared with total costs and expenses for the first quarter of 2018 of $62.8 million. The cash-based operating expenses for the first quarter of 2019 were $78.0 million, compared with cash-based operating expenses for the first quarter of 2018 of $56.3 million.

The Company now expects full-year 2019 revenues for ARIKAYCE to be in the range of $90 million to $105 million.

The Company plans to continue to invest in the following key activities in 2019:

(i)continued support of the U.S. launch and commercialization of ARIKAYCE;

(ii)clinical trials including (a) the ARIKAYCE post-marketing confirmatory study, which will be conducted in a front-line setting, (b) the six-month Phase 2 WILLOW study of INS1007 in patients with non-CF bronchiectasis, and (c) the advancement of other pipeline programs including INS1009 and our earlier-stage research pipeline;

(iii)global expansion in Europe and Japan to support potential regulatory filings and pre-commercial activities in those regions; and

(iv) buildout of an additional third-party manufacturing facility to increase long-term production capacity for ARIKAYCE and a new corporate headquarters facility.

As a result of these activities, Insmed continues to expect cash-based operating expenses to be in the range of $150 million to $170 million for the first half of 2019. In addition, the Company expects capital expenditures, including spend related to the buildout of a new corporate headquarters facility as well as payments classified within other assets for the future right-of-use asset related to the buildout of an additional third-party manufacturing facility, to be in the range of $25 million to $35 million for the first half of 2019.

Conference Call

Insmed will host a conference call beginning today at 8:30 AM Eastern Time. Shareholders and other interested parties may participate in the conference call by dialing 1-888-317-6003

(domestic) or 1-412-317-6061 (international) and referencing conference ID number 6243203. The call will also be webcast live on the Company’s website at www.insmed.com.

A replay of the conference call will be accessible approximately one hour after its completion through May 14, 2019 by dialing 1-877-344-7529 (domestic) or 1-412-317-0088 (international) and referencing replay access code 10131112. A webcast of the call will also be archived for 90 days under the Investor Relations section of the Company’s website at www.insmed.com.

Non-GAAP Financial Measures

In addition to the U.S. generally accepted accounting principles (GAAP) results, this earnings release includes cash-based operating expenses, a non-GAAP financial measure, which Insmed defines as total costs and expenses excluding cost of product revenues, stock-based compensation expense, depreciation and amortization of intangibles. A reconciliation of this non-GAAP financial measure to its most directly comparable GAAP financial measure is presented in the table attached to this press release.

Management believes that this non-GAAP financial measure is useful to both management and investors in analyzing our ongoing business and operating performance. Management believes that providing this non-GAAP information to investors, in addition to the GAAP presentation, allows investors to view our financial results in the way that management views financial results. Management does not intend the presentation of this non-GAAP financial measure to be considered in isolation or as a substitute for results prepared in accordance with GAAP. In addition, this non-GAAP financial measure may differ from similarly named measures used by other companies.

About MAC Lung Disease

Mycobacterium avium complex (MAC) lung disease is a rare and serious disorder that can significantly increase morbidity and mortality. Patients with MAC lung disease can experience a range of symptoms that often worsen over time, including chronic cough, dyspnea, fatigue, fever, weight loss, and chest pain. In some cases, MAC lung disease can cause severe, even permanent damage to the lungs, and can be fatal.

MAC lung disease is an emerging public health concern worldwide with significant unmet needs. Current guideline-based treatment involves the use of multi-drug regimens that are not specifically approved for MAC lung disease. The course of treatment is often two years or more and is inadequate in treating the disease in many patients.

About ARIKAYCE (amikacin liposome inhalation suspension)

ARIKAYCE is the first and only FDA-approved therapy indicated for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen for adult patients with limited or no alternative treatment options. ARIKAYCE is a novel, inhaled, once-daily formulation of amikacin, an established antibiotic that was historically administered intravenously and associated with severe toxicity to hearing, balance, and kidney

function. Insmed’s proprietary PULMOVANCE liposomal technology enables the delivery of amikacin directly to the lungs, where liposomal amikacin is taken up by lung macrophages where the infection resides. This approach prolongs the release of amikacin in the lungs while limiting systemic exposure. ARIKAYCE is administered once daily using the Lamira Nebulizer System manufactured by PARI Pharma GmbH (PARI).

About PARI Pharma and the Lamira Nebulizer System

ARIKAYCE (amikacin liposome inhalation suspension) is delivered by a novel inhalation device, the Lamira Nebulizer System, developed by PARI. Lamira is a quiet, portable nebulizer that enables efficient aerosolization of liquid medications, including liposomal formulations such as ARIKAYCE, via a vibrating, perforated membrane. Based on PARI’s 100-year history working with aerosols, PARI is dedicated to advancing inhalation therapies by developing innovative delivery platforms and new pharmaceutical formulations that work together to improve patient care.

IMPORTANT SAFETY INFORMATION

WARNING: RISK OF INCREASED RESPIRATORY ADVERSE REACTIONS

ARIKAYCE has been associated with an increased risk of respiratory adverse reactions, including hypersensitivity pneumonitis, hemoptysis, bronchospasm, and exacerbation of underlying pulmonary disease that have led to hospitalizations in some cases.

Hypersensitivity Pneumonitis has been reported with the use of ARIKAYCE in the clinical trials. Hypersensitivity pneumonitis (reported as allergic alveolitis, pneumonitis, interstitial lung disease, allergic reaction to ARIKAYCE) was reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (3.1%) compared to patients treated with a background regimen alone (0%). Most patients with hypersensitivity pneumonitis discontinued treatment with ARIKAYCE and received treatment with corticosteroids. If hypersensitivity pneumonitis occurs, discontinue ARIKAYCE and manage patients as medically appropriate.

Hemoptysis has been reported with the use of ARIKAYCE in the clinical trials. Hemoptysis was reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (17.9%) compared to patients treated with a background regimen alone (12.5%). If hemoptysis occurs, manage patients as medically appropriate.

Bronchospasm has been reported with the use of ARIKAYCE in the clinical trials. Bronchospasm (reported as asthma, bronchial hyperreactivity, bronchospasm, dyspnea, dyspnea exertional, prolonged expiration, throat tightness, wheezing) was reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (28.7%) compared to patients treated with a background regimen alone (10.7 %). If bronchospasm occurs during the use of ARIKAYCE, treat patients as medically appropriate.

Exacerbations of underlying pulmonary disease has been reported with the use of ARIKAYCE in the clinical trials. Exacerbations of underlying pulmonary disease (reported as

chronic obstructive pulmonary disease (COPD), infective exacerbation of COPD, infective exacerbation of bronchiectasis) have been reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (14.8%) compared to patients treated with background regimen alone (9.8%). If exacerbations of underlying pulmonary disease occur during the use of ARIKAYCE, treat patients as medically appropriate.

Ototoxicity has been reported with the use of ARIKAYCE in the clinical trials. Ototoxicity (including deafness, dizziness, presyncope, tinnitus, and vertigo) were reported with a higher frequency in patients treated with ARIKAYCE plus background regimen (17 %) compared to patients treated with background regimen alone (9.8%). This was primarily driven by tinnitus (7.6% in ARIKAYCE plus background regimen vs 0.9% in the background regimen alone arm) and dizziness (6.3% in ARIKAYCE plus background regimen vs 2.7% in the background regimen alone arm). Closely monitor patients with known or suspected auditory or vestibular dysfunction during treatment with ARIKAYCE. If ototoxicity occurs, manage patients as medically appropriate, including potentially discontinuing ARIKAYCE.

Nephrotoxicity was observed during the clinical trials of ARIKAYCE in patients with MAC lung disease but not at a higher frequency than background regimen alone. Nephrotoxicity has been associated with the aminoglycosides. Close monitoring of patients with known or suspected renal dysfunction may be needed when prescribing ARIKAYCE.

Neuromuscular Blockade: Patients with neuromuscular disorders were not enrolled in ARIKAYCE clinical trials. Patients with known or suspected neuromuscular disorders, such as myasthenia gravis, should be closely monitored since aminoglycosides may aggravate muscle weakness by blocking the release of acetylcholine at neuromuscular junctions.

Embryo-Fetal Toxicity: Aminoglycosides can cause fetal harm when administered to a pregnant woman. Aminoglycosides, including ARIKAYCE, may be associated with total, irreversible, bilateral congenital deafness in pediatric patients exposed in utero. Patients who use ARIKAYCE during pregnancy, or become pregnant while taking ARIKAYCE should be apprised of the potential hazard to the fetus.

Contraindications: ARIKAYCE is contraindicated in patients with known hypersensitivity to any aminoglycoside.

Most Common Adverse Reactions: The most common adverse reactions in Trial 1 at an incidence >5% for patients using ARIKAYCE plus background regimen compared to patients treated with background regimen alone were dysphonia (47% vs 1%), cough (39% vs 17%), bronchospasm (29% vs 11%), hemoptysis (18% vs 13%), ototoxicity (17% vs 10%), upper airway irritation (17% vs 2%), musculoskeletal pain (17% vs 8%), fatigue and asthenia (16% vs 10%), exacerbation of underlying pulmonary disease (15% vs 10%), diarrhea (13% vs 5%), nausea (12% vs 4%), pneumonia (10% vs 8%), headache (10% vs 5%), pyrexia (7% vs 5%), vomiting (7% vs 4%), rash (6% vs 2%), decreased weight (6% vs 1%), change in sputum (5% vs 1%), and chest discomfort (5% vs 3%).

Drug Interactions: Avoid concomitant use of ARIKAYCE with medications associated with neurotoxicity, nephrotoxicity, and ototoxicity. Some diuretics can enhance aminoglycoside toxicity by altering aminoglycoside concentrations in serum and tissue. Avoid concomitant use of ARIKAYCE with ethacrynic acid, furosemide, urea, or intravenous mannitol.

Overdosage: Adverse reactions specifically associated with overdose of ARIKAYCE have not been identified. Acute toxicity should be treated with immediate withdrawal of ARIKAYCE, and baseline tests of renal function should be undertaken. Hemodialysis may be helpful in removing amikacin from the body. In all cases of suspected overdosage, physicians should contact the Regional Poison Control Center for information about effective treatment.

INDICATION

LIMITED POPULATION: ARIKAYCE is indicated in adults, who have limited or no alternative treatment options, for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen in patients who do not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. As only limited clinical safety and effectiveness data for ARIKAYCE are currently available, reserve ARIKAYCE for use in adults who have limited or no alternative treatment options. This drug is indicated for use in a limited and specific population of patients.

This indication is approved under accelerated approval based on achieving sputum culture conversion (defined as 3 consecutive negative monthly sputum cultures) by Month 6. Clinical benefit has not yet been established. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Limitation of Use: ARIKAYCE has only been studied in patients with refractory MAC lung disease defined as patients who did not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. The use of ARIKAYCE is not recommended for patients with non-refractory MAC lung disease.

Patients are encouraged report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1 (800) FDA 1088. You can also call the Company at 1 (844) 4 INSMED.

Heat Biologics, Inc. Corporate Presentation

On May 7, 2019 Heat Biologics presented the corporate presentation (Presentation, Heat Biologics, MAY 7, 2019, View Source [SID1234535800]).

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