On February 23, 2019 Xynomic Pharma, a clinical stage US-China oncology drug development company, and Bison Capital Acquisition Corp. (Nasdaq: BCAC), reported that jointly announced the dosing of the first South Korean patient at the Asan Medical Center in South Korea in the on-going global pivotal Phase 3 trial of Xynomic’s abexinostat combined with pazopanib as a first- or second-line therapy against renal cell carcinoma (RCC) (Press release, Xynomic Pharmaceuticals, FEB 23, 2019, View Source [SID1234533611]). According to US International Trade Administration, South Korea is the third largest pharmaceutical market in Asia and the 13th largest globally. Furthermore, Xynomic plans to roll out this multi-national trial, currently ongoing in the United States, in Europe and China in the first half of 2019.

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In addition, to support its on-going Phase 3 trial and potential submissions for new drug approval of its lead drug candidate abexinostat, Xynomic has appointed Dr. Sophia Paspal as the Vice President, Regulatory Affairs and Quality Assurance. Dr. Paspal assumes overall responsibility to strengthen Xynomic’s regulatory compliance and quality assurance functions. Dr. Paspal brings 20 years of relevant global industry experience. From 2017 to January 2019, Dr. Paspal worked at Capricor Therapeutics, Inc. and Cellics Therapeutics, Inc., holding the same title. From 2015 to 2017 Dr. Paspal worked as the Director of Regulatory Affairs, Oncology, at Halozyme Therapeutics Inc. From 2014 to 2015 Dr. Paspal worked as Associate Director of Regulatory Affairs, Neurology, for Dart NeuroScience LLC. Prior to 2014, Dr. Paspal worked for companies such as Shire PLC, Allergan, Inc., and Pfizer in developing and implementing regulatory strategies and obtaining and maintaining regulatory approvals. Dr. Paspal holds Regulatory Affairs Certification (RAC) and Drug Development Certification from Temple University RA and QA Program. Dr. Paspal holds a Bachelor of Science in Chemistry and Ph.D. in Pharmaceutics from the University of Minnesota, Twin Cities in Minnesota.

On February 23. 2019 Bausch Health Companies Inc. (NYSE/TSX: BHC) ("Bausch Health," the "Company" or the "Offeror") reported that it has increased the maximum aggregate purchase amount of its outstanding notes listed in the table below (collectively, the "Notes") that it may purchase pursuant to the previously announced cash tender offers (collectively, the "Tender Offers," and each offer to purchase a series of notes individually, a "Tender Offer") upon terms and subject to the conditions set forth in the Offers to Purchase and Consent Solicitation Statement dated Feb. 22, 2019 (the "Statement"), as supplemented by this release, and the related Letter of Transmittal and Consent (Press release, Valeant, FEB 23, 2019, View Source [SID1234533596]). The Company announced that it has increased the maximum aggregate purchase price of the Notes that may be purchased pursuant to the Tender Offers from $1,250,000,000 to $1,500,000,000 (the "Aggregate Maximum Purchase Amount") and it has increased the maximum aggregate purchase price of the 5.50% Senior Notes due 2023 (the "5.50% Notes") and 5.875% Senior Notes due 2023 (the "5.875% Notes" and, together with the 5.50% Notes, the "2023 Notes") that may be purchased pursuant to the Tender Offers from $550,000,000 to $800,000,000 (the "Tender Cap"). The Company also announced that the Tender Offers and the Solicitation are subject to, among other conditions, the completion of concurrent private offerings by Bausch Health and/or its subsidiaries of at least $1,500 million aggregate principal amount of senior notes, an increase from $1,250 million.

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The Company is also soliciting consents (the "Solicitation," and, together with the Tender Offers, the "Tender Offers and Solicitation") to certain proposed amendments to the indenture governing its 5.625% Senior Notes due 2021 (the "2021 Notes") to eliminate substantially all of the restrictive covenants and events of default and related provisions contained in the indenture under which the 2021 Notes were issued (the "Proposed Amendments"). No amendments to the indenture governing the 2023 Notes are being sought. All other terms and conditions of the Tender Offers and Solicitation will remain the same.

A $800,000,000 Tender Cap applies to the aggregate purchase price of the 5.50% Notes and the 5.875% Notes.

Per $1,000 principal amount of Notes validly tendered and accepted for purchase in the applicable Tender Offer (exclusive of any Accrued Interest, which will be paid by the Offeror in addition to the Tender Offer Consideration or the Total Consideration, as applicable, to, but not including, the applicable Settlement Date). References to aggregate purchase price herein exclude Accrued Interest, if any.

Includes the Applicable Premium, if any.

The Tender Offers and Solicitation will expire at 11:59 p.m., New York City time, on March 21, 2019 (such date and time with respect to a Tender Offer and the Solicitation, as it may be extended for such Tender Offer and the Solicitation, the "Expiration Date"). No tenders will be valid if submitted after the Expiration Date. Tendered Notes may be withdrawn from the applicable Tender Offer and, with respect to the 2021 Notes, the related consents may be revoked at or prior to, but not after, 5:00 p.m., New York City time, on March 7, 2019 (such date and time with respect to a Tender Offer and the Solicitation, as it may be extended for such Tender Offer and the Solicitation, the "Withdrawal Deadline"). Holders of Notes who tender their Notes after the Withdrawal Deadline, but prior to the Expiration Date, may not withdraw their tendered Notes or, with respect to the 2021 Notes, revoke their delivered consents, except for certain limited circumstances where additional withdrawal rights are required by law. Holders may not tender their 2021 Notes without delivering their consents to the Proposed Amendments to the indenture under which the 2021 Notes were issued and may not deliver their consents to the Proposed Amendments without tendering their 2021 Notes pursuant to the applicable Tender Offer.

Subject to the terms and conditions of the Tender Offers, the consideration for each $1,000 principal amount of Notes validly tendered (and not validly withdrawn) and accepted for purchase pursuant to the Tender Offers and the Solicitation will be the tender offer consideration for the applicable series of Notes set forth in the table above (with respect to each series of Notes, the "Tender Offer Consideration"). Holders of Notes that are validly tendered (and not validly withdrawn) at or prior to 5:00 p.m., New York City time, on March 7, 2019 (such date and time with respect to a Tender Offer, as it may be extended for such Tender Offer, the "Early Tender Date") and accepted for purchase pursuant to the Tender Offers and the Solicitation will receive the applicable Tender Offer Consideration plus the early tender premium, if any, for the applicable series of Notes as set forth in the table above (with respect to each series of Notes, the "Applicable Premium" and, together with the applicable Tender Offer Consideration, the "Total Consideration"). Holders of Notes validly tendered after the Early Tender Date, but at or prior to the Expiration Date, and accepted for purchase pursuant to the Tender Offers and the Solicitation will receive the applicable Tender Offer Consideration, but not the Applicable Premium for the applicable series of Notes. No tenders will be valid if submitted after the Expiration Date.

In addition to the Tender Offer Consideration or the Total Consideration, as applicable, all Holders of Notes accepted for purchase pursuant to the Tender Offers and the Solicitation will, on the Early Settlement Date (as defined below) or the Final Settlement Date (as defined below), as applicable, also receive accrued and unpaid interest on those Notes from the last interest payment date with respect to those Notes to, but not including, the Early Settlement Date or the Final Settlement Date, as applicable (the "Accrued Interest").

If the Company receives valid consents of the holders of a majority in aggregate principal amount of the outstanding 2021 Notes (the "Requisite Consents"), the Company will execute a supplemental indenture effecting the Proposed Amendments. The Proposed Amendments will not become operative, however, unless and until the Company accepts for purchase and pays the Total Consideration with respect to validly tendered 2021 Notes and validly delivered consents representing a majority in aggregate principal amount of the outstanding 2021 Notes.

The Offeror reserves the right, in its sole discretion, to increase or decrease the amount of Notes purchased in any Tender Offer at any time such that the Aggregate Maximum Purchase Amount and the Tender Cap may be increased or decreased without extending the Early Tender Date or the Withdrawal Deadline or otherwise reinstating withdrawal rights for any Tender Offer, subject to compliance with applicable law, which could result in the Offeror purchasing a greater or lesser amount of Notes in the Tender Offers. If the Aggregate Maximum Purchase Amount or the Tender Cap changes, the Offeror does not expect to extend the Withdrawal Deadline, subject to applicable law.

The Offeror reserves the right, in its sole discretion, at any point following the Early Tender Date and prior to the Expiration Date, to accept for purchase any Notes validly tendered (and not validly withdrawn) at or prior to the Early Tender Date (the date of such acceptance and purchase, the "Early Settlement Date"), subject to the Aggregate Maximum Purchase Amount, the Tender Cap, the Acceptance Priority Levels and proration as described in the Statement. The Early Settlement Date will be determined at the Offeror’s option, assuming the conditions to the Tender Offers and the Solicitation have been either satisfied or waived by the Offeror at or prior to the Early Settlement Date. If the Offeror elects to have an Early Settlement Date, it will accept Notes validly tendered at or prior to the Early Tender Date, subject to the Aggregate Maximum Purchase Amount, the Tender Cap, the Acceptance Priority Levels and proration as described in the Statement. Irrespective of whether the Offeror chooses to exercise its option to have an Early Settlement Date, it will purchase any remaining Notes that have been validly tendered at or prior to the Expiration Date and accepted for purchase, subject to all conditions to the Tender Offers and the Solicitation having been either satisfied or waived by the Offeror, promptly following the Expiration Date (the date of such acceptance and purchase, the "Final Settlement Date"; the Final Settlement Date and the Early Settlement Date each being a "Settlement Date"), subject to the Aggregate Maximum Purchase Amount, the Tender Cap, the Acceptance Priority Levels and proration as described in the Statement. The Final Settlement Date is expected to occur on the second business day following the Expiration Date, assuming the conditions to the Tender Offers and the Solicitation have been either satisfied or waived by the Offeror at or prior to the Expiration Date and Notes having an aggregate purchase price (exclusive of Accrued Interest) equal to the Aggregate Maximum Purchase Amount are not purchased on the Early Settlement Date.

Subject to the Aggregate Maximum Purchase Amount, the Tender Cap, the Acceptance Priority Levels and proration as described in the Statement, all Notes validly tendered at or prior to the Early Tender Date having the highest Acceptance Priority Level will be accepted for purchase before any Notes validly tendered at or prior to the Early Tender Date having the lowest Acceptance Priority Level are accepted for purchase, and all Notes validly tendered after the Early Tender Date having the highest Acceptance Priority Level will be accepted for purchase before any Notes validly tendered after the Early Tender Date having the lowest Acceptance Priority Level are accepted for purchase. However, even if the Tender Offers are not fully subscribed as of the Early Tender Date, subject to the Aggregate Maximum Purchase Amount and the Tender Cap, Notes validly tendered at or prior to the Early Tender Date will be accepted for purchase before any Notes validly tendered after the Early Tender Date are accepted for purchase, even if such Notes validly tendered after the Early Tender Date have the highest Acceptance Priority Level than Notes validly tendered at or prior to the Early Tender Date. Therefore, if the aggregate purchase price of Notes validly tendered at or prior to the Early Tender Date equals or exceeds the Aggregate Maximum Purchase Amount, the Offeror will not accept for purchase any Notes tendered after the Early Tender Date, and if the aggregate purchase price of 2023 Notes validly tendered at or prior to the Early Tender Date equals or exceeds the Tender Cap, the Offeror will not accept for purchase 2023 Notes tendered after the Early Tender Date, in each case unless the Offeror increases the Tender Cap and/or Aggregate Maximum Purchase Amount. Additional information about the application of the Aggregate Maximum Purchase Amount, Acceptance Priority Levels, Tender Cap and proration is set forth in the Statement.

The Tender Offers are not conditioned upon a minimum amount of Notes of any series, or a minimum amount of Notes of all series, being tendered. However, the Tender Offers and the Solicitation are subject to, and conditioned upon, the satisfaction or waiver of certain conditions described in the Statement, including completion of concurrent private offerings by Bausch Health and/or its subsidiaries of at least $1,500 million aggregate principal amount of senior notes to finance the payment of the Tender Offer Consideration and the Total Consideration. While none of the Tender Offers are subject to receipt of the Requisite Consents, the Solicitation is conditioned on receipt of the Requisite Consents.

If, following the consummation of the Tender Offers and the Solicitation, any 2021 Notes remain outstanding, the Offeror intends to promptly redeem such 2021 Notes in accordance with terms of the 2021 Notes and the indenture under which the 2021 Notes were issued. In addition, if the aggregate purchase price of 2023 Notes validly tendered by the Expiration Date and accepted for purchase does not exceed the Tender Cap, the Offeror intends to retire or repay existing debt in a purchase price equal to the excess of the Tender Cap over the aggregate purchase price of 2023 Notes validly tendered and accepted for purchase in the Tender Offers.

J.P. Morgan is acting as the dealer manager and solicitation agent in the Tender Offers and Solicitation. Global Bondholder Services Corporation has been retained to serve as both the depositary and the information agent for the Tender Offers and Solicitation. Persons with questions regarding the Tender Offers and Solicitation should contact J.P. Morgan Securities LLC at (collect) (212) 834-3260 or (toll free) (866) 834-4666. Requests for copies of the Statement, the related Letter of Transmittal and Consent and other related materials should be directed to Global Bondholder Services Corporation at (toll-free) (866) 470-4200 or (collect) (212) 430-3774.

None of the Offeror, its board of directors or officers, the dealer managers and solicitation agents, the depositary, the information agent or the trustee with respect to the Notes, or any of their respective affiliates, makes any recommendation that holders tender or refrain from tendering all or any portion of the principal amount of their Notes, and no one has been authorized by any of them to make such a recommendation. Holders must make their own decision as to whether to tender their Notes and, if so, the principal amount of Notes to tender. The Tender Offers and Solicitation are made only by the Statement and related Letter of Transmittal and Consent. This news release is neither an offer to purchase nor a solicitation of an offer to sell any notes in the Tender Offers nor a solicitation of consents with respect to the Notes or any other securities. The Tender Offers and the Solicitation are not being made to holders of Notes in any jurisdiction in which the making or acceptance thereof would not be in compliance with the securities, blue sky or other laws of such jurisdiction. In any jurisdiction in which the Tender Offers and the Solicitation are required to be made by a licensed broker or dealer, the Tender Offers and the Solicitation will be deemed to be made on behalf of the Offeror by the dealer manager and solicitation agent or one or more registered brokers or dealers that are licensed under the laws of such jurisdiction.

Any securities issued pursuant to the financing transactions described above will not be registered under the Securities Act of 1933, as amended (the "Securities Act"), or any state securities law and may not be offered or sold in the United States absent registration or an applicable exemption from registration under the Securities Act and applicable state securities laws. Such securities have not been and will not be qualified for sale to the public by prospectus under applicable Canadian securities laws and, accordingly, any offer and sale of the securities in Canada will be made on a basis which is exempt from the prospectus requirements of such securities laws.

This news release is being issued pursuant to Rule 135c under the Securities Act and shall not constitute an offer to sell or the solicitation of an offer to sell or the solicitation of an offer to buy any securities that may be issued pursuant to the financing transactions described above. Further, nothing contained herein shall constitute a notice of redemption of the Notes.

On February 22, 2019 Clovis Oncology, Inc. (Nasdaq: CLVS) reported that its Chief Executive Officer and President, Patrick J. Mahaffy, will present at the 8th Annual SVB Leerink Global Healthcare Conference on Thursday, February 28, 2019 at 11:00 a.m. Eastern time (Press release, Clovis Oncology, FEB 22, 2019, View Source [SID1234533579]). The conference will be held at the Lotte New York Palace in New York City.

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A live webcast of the presentation and Q&A session can be accessed through the investor relations section of the Company’s website at www.clovisoncology.com. Following the live presentation, a replay of the webcast will be available on the Company’s website for 30 days.

On February 22, 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 fourth quarter and full year ended December 31, 2018 and provided a business update (Press release, Insmed, FEB 22, 2019, View Source [SID1234533599]).

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"2018 was a pivotal year for Insmed, with the U.S. approval and launch of ARIKAYCE (amikacin liposome inhalation suspension), the first and only FDA-approved treatment for patients with refractory Mycobacterium avium complex (MAC) lung disease. We are very pleased with the strong momentum we’ve seen since launch, including a wide breadth of prescribers, steady additions of new patients, and positive reimbursement trends," commented Will Lewis, Chairman and Chief Executive Officer of Insmed. "In 2019, we are focused on continuing our efforts to execute a successful U.S. launch while pursuing our strategic priorities, including completing the design and protocol of the confirmatory study required for the full U.S. approval of ARIKAYCE, which is intended to support use of ARIKAYCE in a front-line setting; accelerating our global expansion to support potential regulatory filings for ARIKAYCE in Europe and Japan; and advancing our pipeline to bring other potential therapies to market for patients with serious and rare diseases."

Fourth Quarter and Full-Year 2018 Financial Results

· Total revenue for the fourth quarter and full year ended December 31, 2018 was $9.8 million, comprising U.S. net sales of $9.2 million and ex-U.S. net sales of $0.6 million. The ex-U.S. net sales reflect utilization from the Temporary Authorization for Use (Autorisation Temporaire d’Utilisation or ATU) program in France.

· Cost of product revenues (excluding amortization of intangible assets) was $2.4 million for the fourth quarter of 2018. Prior to the approval of ARIKAYCE, the company expensed manufacturing and material costs as research and development expenses.

· Research and development expenses were $39.9 million for the fourth quarter of 2018, compared with $33.9 million for the fourth quarter of 2017. For the full year of 2018, research and development expenses were $145.3 million compared with $109.7 million for the full year of 2017. The increase was primarily due to an increase in external manufacturing expenses and higher compensation and related expenses due to an increase in headcount.

· Selling, general and administrative expenses for the fourth quarter of 2018 were $54.0 million, compared with $31.4 million for the fourth quarter of 2017. For the full year of 2018, selling, general and administrative expenses were $168.2 million compared with $79.2 million for the full year of 2017. The increase was primarily due to higher compensation and related expenses due to an increase in headcount and an increase in expenses relating to pre-commercial planning activities in preparation for the launch of ARIKAYCE.

· For the fourth quarter of 2018, Insmed reported a net loss of $91.6 million, or $1.19 per share, compared with a net loss of $65.4 million, or $0.85 per share, for the fourth quarter of 2017. For the full year of 2018, Insmed reported a net loss of $324.3 million, or $4.22 per share, compared with a net loss of $192.6 million, or $2.89 per share, for the full year of 2017.

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 MAC lung disease as part of a combination antibacterial drug regimen for adult patients who have limited or no alternative treatment options. As previously reported, as of December 31, 2018, more than 500 patients in the U.S. had initiated treatment with ARIKAYCE and approximately 600 physicians in the U.S had written at least one prescription for the therapy.

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.

Global Expansion Under Way

The Company 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. In January 2019, Insmed opened a new office in Tokyo to support the growth of the workforce in Japan.

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

Enrollment Remains on Track for WILLOW Study

Insmed continues to advance the development of INS1007 for patients with non-CF bronchiectasis and expects to complete enrollment in the six-month Phase 2 WILLOW study in mid-2019.

Financial Guidance and Balance Sheet

As of December 31, 2018, Insmed had cash and cash equivalents of $495.1 million. The Company’s total costs and expenses for the fourth quarter of 2018 were $97.6 million and for the full year of 2018 were $317.2 million. The cash-based operating expenses for the fourth quarter of 2018 were $86.9 million and for the full year of 2018 were $283.7 million.

The Company expects full-year 2019 revenues for ARIKAYCE to be in the range of $80 million to $90 million.

The Company is investing 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 regulatory and pre-commercial activities in those regions; and

(iv) build-out 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 expects 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 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 5106939. 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 two hours after its completion through March 1, 2019 by dialing 1-877-344-7529 (domestic) or 1-412-317-0088 (international) and referencing conference ID number 10128650. 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.

On February 22, 2019 Momenta Pharmaceuticals, Inc. (Nasdaq: MNTA), a biotechnology company focused on discovering and developing novel biologic therapeutics to treat rare immune-mediated diseases, reported its financial results for the fourth quarter and full year ended December 31, 2018 (Press release, Momenta Pharmaceuticals, FEB 22, 2019, View Source [SID1234533600]).

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"In 2018 we transformed our company to focus on discovering and developing new therapies to treat rare immune-mediated disorders. We have since made important progress, as we have commenced two Phase 2 clinical trials of our anti-FcRn antibody, M281, and dosed the first healthy volunteer in part 1 of a Phase 1 / 2 clinical trial of our hypersialylated IgG candidate M254. Meanwhile, our legacy business continues to provide value in the form of revenue from the Glatopa franchise and we continue to work with Mylan to advance the Phase 3 clinical trial of M710, our biosimilar EYLEA candidate," said Craig A. Wheeler, President and Chief Executive Officer of Momenta Pharmaceuticals.

"Looking forward, as we focus on operational execution in the year ahead, we remain confident in the ability of our differentiated platforms to produce best-in-class immune-mediated therapeutics and are well-capitalized to execute on our development plans," Wheeler continued.

Fourth Quarter 2018 Highlights, Recent Events and Anticipated Upcoming Milestones

Novel Therapeutics Pipeline:

M281 (anti-FcRn): a fully human anti-neonatal Fc receptor (FcRn) aglycosylated immunoglobulin G (IgG1) monoclonal antibody (mAb)

The Company has commenced two Phase 2 clinical studies of M281 in gMG and HDFN with topline results anticipated in 2020 and 2021, respectively. The Company also plans to initiate a third study of M281 in an additional autoimmune indication in 2019.

In February 2019, the Company presented research at the Society for Maternal-Fetal Medicine 39th Annual Pregnancy Meeting in Las Vegas, NV highlighting the ability of M281 to inhibit transfer of immunoglobulin G from maternal to fetal circulation in an ex vivo placental perfusion model.

· In November 2018, the Company announced the publication of results from a Phase 1 clinical trial of M281 in healthy volunteers in the peer-reviewed journal Clinical Pharmacology & Therapeutics.

M254 (hsIgG): a hypersialylated immunoglobulin designed as a high potency alternative for intravenous immunoglobulin (IVIg)

· In January 2019, the Company announced that the first subject was dosed in the Phase 1/2 clinical trial in ITP. The multi-part study will first enroll healthy volunteers and includes single and multiple dose studies, and a randomized cross-over study comparing M254 to IVIg. Preliminary clinical data is expected in 2020.

M230 (CSL730): a recombinant Fc multimer being developed in collaboration with CSL

·The Phase 1 clinical trial in healthy volunteers to evaluate the safety and tolerability of M230 continues. Momenta’s partner, CSL expects to complete the Phase 1 study by the end of 2019.

Legacy Products:

Glatopa 20 mg and 40 mg: FDA approved generic versions of COPAXONE 20 mg and 40 mg, developed and commercialized in collaboration with Sandoz

·In the fourth quarter of 2018, Momenta recorded $10.8 million in product revenue from Sandoz’s sales of Glatopa products.

M923: a fully-owned proposed biosimilar to HUMIRA (adalimumab)

· In November 2018, the Company announced license agreements with AbbVie, providing worldwide rights for the launch of M923. Under the terms of the agreements, and subject to approval by health regulatory authorities, Momenta may launch M923 worldwide based on agreed-to launch dates, including in the United States (U.S.) in November 2023.

M710: a proposed biosimilar to EYLEA (aflibercept) candidate being developed in collaboration with Mylan

· In August 2018, Mylan initiated a pivotal clinical trial in patients with diabetic macular edema to compare safety, efficacy and immunogenicity of M710 with EYLEA. In November 2018, Momenta provided formal notice of termination for all other biosimilar candidates previously subject to the collaboration agreement with Mylan.

Corporate:

·In December 2018, Momenta announced the closing of a public offering of 20.0 million shares of its common stock at the price of $11.50 per share. Aggregate gross proceeds from the offering were $230.0 million.

Fourth Quarter and Full Year 2018 Financial Results

Revenue: In the fourth quarter of 2018, the Company recorded $10.8 million in product revenue from Sandoz’s sales of Glatopa, compared to $13.1 million for the same period in 2017. For the year ended December 31, 2018, the Company recorded $39.7 million in product revenue from Sandoz’s sales of Glatopa, compared to $66.8 million for the same period in 2017. The decrease in product revenue of $2.3 million, or 17.6% from the fourth quarter of 2017 to the fourth quarter of 2018 was primarily due to continued competition. The decrease in product revenue of $27.2 million, or 41.3% from the year ended 2017 to the year ended 2018 was primarily due to continued competition and reflects a $9.8 million deduction of our 50% share of GLATOPA 40 mg/mL inventory written off by Sandoz.

Research and development revenue for the fourth quarter of 2018 was $32.1 million, compared to $51.2 million in the same quarter in 2017. The decrease in research and development revenue of $19.2 million, or 37.3%, was primarily due to the recognition of an upfront payment of $50.0 million from CSL, offset in part by an increase of revenue recognized related to Mylan’s upfront payment of $28.4 million due to the partial termination of the Mylan collaboration agreement and the resulting determination that certain performance obligations under the agreement have been partially satisfied. For the year ended December 31, 2018, research and development revenue was $35.9 million compared to $72.1 million recorded in the same period in 2017. The decrease in research and development revenue of $36.2 million, or 50.2%, was primarily due to recognition of the $50.0 million upfront payment from CSL and the $10.0 million commercial milestone from Sandoz recognized in 2017 that was non-recurring in 2018. This was offset in part by an increase of revenue recognized related to Mylan’s upfront payment of $28.4 million due to the partial termination of the Mylan collaboration agreement and the resulting determination that certain performance obligations under the agreement have been partially satisfied.

Total revenue for the fourth quarter of 2018 was $42.8 million compared to $64.6 million for the same period in 2017. For the year ended December 31, 2018, total revenue was $75.6 million compared to $138.9 million for the same period in 2017.

Operating Expenses: Total GAAP operating expenses were $52.5 million in the fourth quarter of 2018. For the year ended December 31, 2018, total GAAP operating expenses were $256.9 million.

Research and development expenses for the fourth quarter of 2018 were $28.7 million, compared to $36.1 million for the same period in 2017. The decrease of $7.4 million, or 20.5%, was primarily due to decreased spending on the Company’s biosimilar programs. For the year ended December 31, 2018, research and development expenses were $124.0 million, compared to $149.2 million for the same period in 2017. The decrease of $25.2 million, or 16.9%, was due to decreased spending on the Company’s biosimilar programs of $43.5 million. These decreases were partially offset by increases in spend of $23.9 million on the Company’s novel therapeutic programs.

General and administrative expenses for the fourth quarter of 2018 were $21.5 million, compared with $15.8 million for the same period in 2017. The increase of $5.7 million, or 36.1%, was primarily driven by increased legal costs and depreciation. For the year ended December 31, 2018, general and administrative expenses were $85.1 million, compared to $82.2 million for the same period in 2017. The increase of $2.9 million, or 3.5%, was driven by net of increased rent expense of $3.3 million related to occupancy of new premises, one-time costs related to our strategic review of $3.2 million and depreciation of $3.6 million as the Company evaluated estimates of the useful lives of depreciable assets, partially offset by decreases of $4.6 million of legal costs relating to our ongoing litigation and personnel salaries of $2.3 million due in part to the recent workforce reduction.

Fourth quarter non-GAAP operating expense was $47.2 million, within the range of previously provided guidance of $45 – $55 million. Full year 2018 non-GAAP operating expense was $219.2 million. Non-GAAP operating expense is total operating expenses, less stock-based compensation expense, restructuring expense and collaborative reimbursement revenue. See "Non-GAAP Financial Information and Other Disclosures" and the table below entitled "Reconciliation of GAAP Results to Non-GAAP Financial Measures" for a reconciliation of GAAP operating expense to non-GAAP operating expense.

Net Income (Loss): The Company reported a net loss of $8.2 million, or $0.10 per share for the fourth quarter of 2018 compared to a net income of $13.8 million, or $0.18 per share for the same period in 2017. For the year ended December 31, 2018, the Company reported a net loss of $176.1 million, or $2.26 per share compared to a net loss of $88.1 million, or $1.20 per share for 2017.

Cash Position: At December 31, 2018, Momenta had $449.4 million in cash, cash equivalents and marketable securities compared to $281.6 million at September 30, 2018, reflecting the December 2018 common stock financing.

2019 Financial Guidance

Momenta provides non-GAAP operating expense guidance, which it believes can enhance an overall understanding of its financial performance when considered together with GAAP financial measures. Refer to the section of this press release below entitled "Non-GAAP Financial Information and Other Disclosures" for further discussion of this subject.

Non-GAAP operating expense is total operating, less stock-based compensation expense, restructuring expense and collaborative reimbursement revenue. Momenta is providing quarterly non-GAAP operating expense guidance of $45 – $55 million for 2019.

Non-GAAP Financial Information and Other Disclosures

Momenta uses a non-GAAP financial measure, non-GAAP operating expense, to provide operating expense guidance. Momenta believes this non-GAAP financial measure is useful to investors because it provides greater transparency regarding Momenta’s operating performance as it excludes non-cash stock compensation expense, restructuring expense and collaborative reimbursement revenue. This non-GAAP financial measure should not be considered a substitute or an alternative to GAAP total operating expense and should not be considered a measure of Momenta’s liquidity. Instead, non-GAAP operating expense should only be used to supplement an understanding of Momenta’s operating results as reported under GAAP. Momenta has not provided GAAP reconciliation for its forward-looking non-GAAP annual or quarterly operating expense because Momenta

cannot reliably predict without unreasonable efforts the timing or amount of the factors that substantially contribute to the projection of stock compensation expense, which is excluded from the forward-looking non-GAAP financial measure. The Company has provided the estimated reconciling information that is available without unreasonable effort in the section of this press release above entitled "2019 Financial Guidance."

Conference Call Information

Management will host a conference call and webcast today at 8:30 am ET to discuss these results and provide an update on the Company. A live webcast of the conference call may be accessed on the "Investors" section of the Company’s website, www.momentapharma.com. Please go to the site at least 15 minutes prior to the call in order to register, download, and install any necessary software. An archived version of the webcast will be posted on the Momenta website approximately two hours after the call.

To access the call you may also dial (877) 224-9084 (domestic) or (720) 545-0022 (international) prior to the scheduled conference call time and provide the access code 7484068. A replay of the call will be available approximately two hours after the conclusion of the call and will be accessible through 7484068. To access the replay, please dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and provide the access code 7484068.