On October 30, 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 third quarter ended September 30, 2019 and provided a business update (Press release, Insmed, OCT 30, 2019, View Source [SID1234550019]).

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"We remain very pleased with the continued strong performance of the US launch of ARIKAYCE (amikacin liposome inhalation suspension). "Insmed remains guided by our desire to have a positive impact on patients’ lives and is powered by our shared sense of purpose to deliver therapies to small patient populations experiencing big health problems," commented Will Lewis, Chairman and Chief Executive Officer of Insmed. "We are also progressing well with our global expansion efforts including filing for regulatory approval of ARIKAYCE in the EU last quarter and advancing our planned regulatory filings in Japan in first half of 2020. We are also looking forward to the topline results from the WILLOW study which we expect in the first quarter of 2020."

Third Quarter 2019 Financial Results

·Total revenue for the third quarter ended September 30, 2019 was $38.9 million, comprising U.S. net sales of $37.8 million and ex-U.S. net sales of $1.1 million. The ex-U.S. net product sales include $0.9 million from the Temporary Authorization for Use (Autorisation Temporaire d’Utilisation or ATU) program in France and $0.2 million from the named patient program in Germany, both compassionate use programs.
·Cost of product revenues (excluding amortization of intangible assets) was $6.4 million for the third quarter of 2019.
·Research and development expenses were $34.3 million for the third quarter of 2019, compared with $39.5 million for the third quarter of 2018.
·Selling, general and administrative expenses for the third quarter of 2019 were $53.3 million, compared with $44.4 million for the third quarter of 2018. The increase was primarily due to milestone payments and other external expenses related to ARIKAYCE.

·For the third quarter of 2019, Insmed reported a GAAP net loss of $60.7 million, or $0.68 per share, compared with a GAAP net loss of $87.7 million, or $1.14 per share, for the third quarter of 2018.

Recent Corporate Developments & Program Highlights

WILLOW Study

Insmed completed enrollment in the six-month Phase 2 WILLOW study of INS1007 for patients with non-cystic fibrosis (CF) bronchiectasis during the 2nd quarter of 2019 and continues to expect top-line data in the first quarter of 2020.

ARIKAYCE Launch and Lifecycle Management

Insmed continues to advance the post-approval confirmatory clinical trial for ARIKAYCE and the Company has initiated efforts to develop an appropriate patient reported outcome (PRO) tool that will enable the assessment of therapies for the treatment of NTM lung disease. Insmed plans to conduct the confirmatory study of ARIKAYCE in a frontline setting of patients with MAC lung disease as well as a separate study in patients with NTM lung disease caused by Mycobacterium abscessus.

Insmed Appoints Chief Medical Officer

Martina Flammer, M.D., M.B.A., has been appointed Chief Medical Officer at Insmed, effective mid-December 2019. Dr. Flammer has more than 17 years of experience in both medical and commercial roles. She has launched global brands and managed pipeline portfolios across therapeutic areas and geographies, including the U.S., Europe, Japan and China. Dr. Flammer was most recently Head of Corporate Division Customer Value, Senior Vice President at Boehringer Ingelheim International. She has previously held various roles at Boehringer Ingelheim, including Vice President Clinical Development & Medical Affairs, Specialty Care Business Unit, and Chief Medical Officer, Vice President of Medicine, Regulatory Affairs & Pharmacovigilance, Boehringer Ingelheim Canada. Prior to that, Dr. Flammer held commercial and medical roles at Pfizer. She holds a medical degree from the University of Vienna Medical School, Austria and a Master of Business Administration degree from New York University Stern School of Business.

Financial Guidance and Balance Sheet

As of September 30, 2019, Insmed had cash and cash equivalents of $535.6 million. The Company’s total costs and expenses for the third quarter of 2019 were $95.4 million, compared with total costs and expenses for the third quarter of 2018 of $84.0 million. Cash-based operating expenses, as defined below, for the third quarter of 2019 were $72.6 million, compared with cash-based operating expenses for the third quarter of 2018 of $75.1 million.

The Company now expects full-year 2019 total revenue for ARIKAYCE to be in the range of $133 million to $138 million.

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

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

(ii)clinical trials including (a) the development and verification of a PRO for NTM lung disease as a pivotal step toward initiating a confirmatory clinical study of ARIKAYCE, (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 pre-commercial activities in those regions and potential regulatory filings in Japan; and

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

Insmed continues to expect cash-based operating expenses to be in the range of $140 million to $155 million for the second half of 2019. In addition, the Company continues to expect capital expenditures, including those related to its 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 $20 million to $30 million for the second 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 (888) 317-6003 (domestic) or (412) 317-6061 (international) and referencing conference ID number 6042526. 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 November 6, 2019 by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and referencing replay access code 10136134. 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.

On October 30, 2019 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers, reported it will report financial results for the third quarter of 2019 after the close of U.S. markets on Wednesday, November 6, 2019 (Press release, Bellicum Pharmaceuticals, OCT 30, 2019, View Source [SID1234550018]). Management will host a webcast and conference call at 5 p.m. ET / 2 p.m. PT that day to discuss the financial results and provide a corporate update.

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The live call may be accessed by dialing 877-407-3103 (U.S. domestic) and 201-493-6791 (international) at least 10 minutes prior to the start of the call. The event will be webcast live and can also be accessed in the Events & Presentations section of bellicum.com. An archived version of the webcast will be available for replay in the Investors & Media section of the Bellicum website following the call.

On October 30, 2019 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for the treatment of cancer, reported that it will report third quarter 2019 financial results after the close of U.S. financial markets on Tuesday, November 5, 2019 (Press release, Kura Oncology, OCT 30, 2019, View Source [SID1234550017]). Kura’s management will host a webcast and conference call at 4:30 p.m. ET / 1:30 p.m. PT that day to discuss the financial results and provide a corporate update.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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The live call may be accessed by dialing 877-516-3514 for domestic callers and 281-973-6129 for international callers and entering the conference code: 5159117. A live webcast and archive of the call will be available online from the investor relations section of the company website at www.kuraoncology.com.

On October 30, 2019 ArQule, Inc. (Nasdaq: ARQL) reported its financial results for the third quarter of 2019 (Press release, ArQule, OCT 30, 2019, View Source [SID1234550016]).

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For the quarter ended September 30, 2019, the Company reported a net loss of $10.7 million, or $0.09 per basic share, compared with net loss of $5.6 million, or $0.05 per basic share, for the quarter ended September 30, 2018.

As of September 30, 2019, the Company had a total of approximately $174.1 million in cash, cash equivalents, and marketable securities.

Key Highlights from Q3, 2019

ARQ 531, our potent and reversible dual inhibitor of both wild-type and C481-mutant BTK:
Completed recruitment of the phase 1 dose escalation trial and determined a recommended phase 2 dose (RP2D) of 65 mg once a day
Submitted American Society of Hematology (ASH) (Free ASH Whitepaper) abstract in July, which is scheduled to be made public at 9:00 a.m. ET, November 6, 2019
Began dosing phase 2 expansion cohorts in multiple B cell malignancies at the RP2D
Requested and received a meeting date with the FDA to discuss registrational trial design
Miransertib, our potent and selective first-generation AKT inhibitor:
Initiated multiple additional sites in our previously described registrational trial, MOSAIC (Miransertib in Overgrowth Syndromes in Adults and Children), for both Proteus syndrome (PS) and PIK3CA-related Overgrowth Spectrum (PROS)
Recently announced the first patient dosed under the MOSAIC protocol at Texas Children’s Hospital (TCH) Vascular Anomalies Center
ARQ 751, our highly potent and selective next-generation AKT inhibitor:
Continued the signal generation work in genetically defined solid tumors
Derazantinib, our FGFR inhibitor, partnered with Basilea and Sinovant, in a registrational trial for intrahepatic cholangiocarcinoma:
Continued to interact with our partners, Basilea and Sinovant
Paolo Pucci, Chief Executive Officer of ArQule, commented, "We are very excited to present the final phase 1 data set for ARQ 531 at ASH (Free ASH Whitepaper) in December. These data will include meaningful updates on clinical activity and durability that provide a more complete picture of the potential of ARQ 531 and will reinforce its position as the leading reversible BTK inhibitor."

"The tremendous progress ARQ 531 has made in such a short period has allowed us to provide first ever proof of concept with a reversible BTK inhibitor in the emerging unmet medical need of C481-mutant CLL patients. We are now actively recruiting patients in the multi-arm phase 2 trial," commented Dr. Brian Schwartz, Chief Medical Officer of ArQule. "It is also very gratifying to have begun patient enrollment in our registrational MOSAIC trial in Proteus syndrome and PROS."

Revenues and Expenses

Revenues for the third quarter, 2019, were $0.2 million compared with revenues of $5.0 million for the third quarter, 2018.

Research and development expenses in the third quarter, 2019 were $8.3 million compared with $7.3 million for the third quarter, 2018.

General and administrative expenses in the third quarter, 2019 were $3.2 million compared with $3.4 million for the third quarter, 2018.

2019 Financial Guidance

For 2019, ArQule expects revenue to range between $2 and $5 million. Net loss is expected to range between $40 and $43 million, and net loss per share to range between $(0.35) and $(0.37) for the year. ArQule expects to end 2019 with approximately $160 million in cash and marketable securities which will support the current business plan into 2022.

Conference Call and Webcast

ArQule will hold its second quarter financial results call today, October 30, 2019 at 9:00 a.m. ET. The live webcast can be accessed in the "Investors and Media" section of our website, www.arqule.com, under "Events & Presentations." You may also listen to the call by dialing (877) 868-1831 within the U.S. or (914) 495-8595 outside the U.S. and entering the conference ID: 4289763. A replay will be available two hours after the completion of the call and can be accessed in the "Investors & Media" section of our website, www.arqule.com, under "Events and Presentations."

On October 30, 2019 Bristol-Myers Squibb Company (NYSE:BMY) ("Bristol-Myers Squibb") reported the extension of the expiration date of the offers to exchange (the "Exchange Offers") notes (the "Celgene Notes") issued by Celgene Corporation (NASDAQ:CELG) ("Celgene") for up to $19,850,000,000 aggregate principal amount of new notes to be issued by Bristol-Myers Squibb Company (the "Bristol-Myers Squibb Notes") and cash and the related consent solicitations (the "Consent Solicitations") being made by Bristol-Myers Squibb on behalf of Celgene to adopt certain proposed amendments (the "Amendments") to the indentures governing the Celgene Notes (Press release, Bristol-Myers Squibb, OCT 30, 2019, View Source;3 [SID1234550015]). Bristol-Myers Squibb hereby extends such expiration date from 5:00 p.m., New York City time, on October 31, 2019, to 5:00 p.m., New York City time, on November 4, 2019 (as the same may be further extended, the "Expiration Date").

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On the early participation date of May 1, 2019, requisite consents were received and supplemental indentures were executed, eliminating substantially all restrictive covenants and certain events of default and other provisions in each of the indentures governing the Celgene Notes. Such supplemental indentures will only become operative upon the settlement date of the Exchange Offers.

The Exchange Offers and Consent Solicitations are being made pursuant to the terms and subject to the conditions set forth in the confidential offering memorandum and consent solicitation statement dated April 17, 2019 and the related letter of transmittal hereby, each as amended by the press releases dated May 1, 2019, May 24, 2019, June 28, 2019, September 23, 2019, October 8, 2019, October 18, 2019 and as amended hereby, and are conditioned upon the closing of Bristol-Myers Squibb’s acquisition of Celgene (the "Merger"), which condition may not be waived by Bristol-Myers Squibb, and certain other conditions that may be waived by Bristol-Myers Squibb.

The settlement date for the Exchange Offers is expected to occur promptly after the Expiration Date and on or about the closing date of the Merger. The closing of the Merger is expected to occur by the end of 2019. As a result, the Expiration Date may be further extended one or more times. Bristol-Myers Squibb will provide notice of any such extension in advance of the Expiration Date.

Except as described in this press release, all other terms of the Exchange Offers and Consent Solicitations remain unchanged.

As of 5:00 p.m., New York City time, on October 29, 2019, the principal amounts of Celgene Notes set forth in the table below had been validly tendered and not validly withdrawn:

Documents relating to the Exchange Offers and Consent Solicitations will only be distributed to eligible holders of Celgene Notes who complete and return an eligibility form confirming that they are either a "qualified institutional buyer" under Rule 144A or not a "U.S. person" and outside the United States under Regulation S for purposes of applicable securities laws. Except as amended by the press releases dated May 1, 2019, May 24, 2019, June 28, 2019, September 23, 2019, October 8, 2019, October 18, 2019 and as amended hereby, the complete terms and conditions of the Exchange Offers and Consent Solicitations are described in the confidential offering memorandum and consent solicitation statement dated April 17, 2019 and the related letter of transmittal, copies of which may be obtained by contacting Global Bondholder Services Corporation, the exchange agent and information agent in connection with the Exchange Offers and Consent Solicitations, at (866) 470 3900 (U.S. toll-free) or (212) 430 3774 (banks and brokers). The eligibility form is available electronically at: View Source