On March 1,2018 Nektar Therapeutics (Nasdaq: NKTR) reported its financial results for the fourth quarter and year ended December 31, 2017 (Press release, Nektar Therapeutics, MAR 1, 2018, View Source [SID1234524309]

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Cash and investments in marketable securities at December 31, 2017 were $353.2 million as compared to $389.1 million at December 31, 2016. This does not include $1.85 billion in upfront payments from the new Bristol-Myers Squibb collaboration, which was announced on February 14, 2018.

"This past year was truly transformational for Nektar as we achieved a number of successes with Nektar medicines across our three key therapeutic areas of immuno-oncology, immunology and pain," said Howard W. Robin, President and Chief Executive Officer of Nektar. "In the area of pain, we completed a successful Phase 3 program for NKTR-181 in over 2,100 patients and healthy volunteers that will comprise our NDA submission in the second quarter of this year. In immunology, we entered into a major partnership with Eli Lilly for NKTR-358, a potential first-in-class T regulatory resolution therapeutic, which will be developed to treat a broad range of auto-immune disorders. Finally, in immuno-oncology, the clinical success we achieved with NKTR-214 led to a groundbreaking collaboration with Bristol-Myers Squibb that now enables us to broadly and rapidly advance NKTR-214 into over 20 registrational trials in up to 15,000 patients."

Summary of Financial Results

Revenue for the fourth quarter of 2017 was $95.5 million as compared to $37.5 million in the fourth quarter of 2016. Revenue in the fourth quarter of 2017 included a total of $60.0 million of non-recurring revenue related to a new sublicense agreement, a contract settlement agreement and the recognition of deferred revenue from several collaboration agreements.

Revenue for the year ended December 31, 2017 was $307.7 million as compared to $165.4 million in 2016. Revenue in 2017 included recognition of $130.1 million of the $150.0 million upfront payment from Nektar’s collaboration with Eli Lilly & Company for the development and commercialization of NKTR-358.

Total operating costs and expenses in the fourth quarter of 2017 were $119.5 million as compared to $69.6 million in the fourth quarter of 2016. Total operating costs and expenses increased primarily as a result of higher research and development (R&D) expense. Total operating costs and expenses for the year ended December 31, 2017 were $367.4 million as compared to $278.3 million in 2016.

R&D expense in the fourth quarter of 2017 was $81.4 million as compared to $50.2 million for the fourth quarter of 2016. R&D expense for the year ended December 31, 2017 was $268.5 million as compared to $203.8 million in 2016. R&D expense was higher in 2017 as compared to 2016 primarily because of expenses for our pipeline programs, including the completion of Phase 3 clinical studies for NKTR-181, Phase 1/2 clinical studies of NKTR-214 and NKTR-358, and IND-enabling activities for NKTR-262 and NKTR-255.

General and administrative (G&A) expense was $12.3 million in the fourth quarter of 2017 as compared to $12.8 million in the fourth quarter of 2016. G&A expense for the year ended December 31, 2017 was $52.4 million as compared to $44.3 million in 2016.

Net loss for the fourth quarter of 2017 was $33.8 million or $0.21 loss per share as compared to a net loss of $42.2 million or $0.28 loss per share in the fourth quarter of 2016. Net loss for the year ended December 31, 2017 was $96.7 million or $0.62 loss per share as compared to a net loss of $153.5 million or $1.10 loss per share in 2016.

2017 and Year-to-Date Business Highlights

● In February 2018, Nektar and Bristol-Myers Squibb entered into a global development and commercialization agreement to evaluate the full potential of NKTR-214 plus Opdivo (nivolumab) in more than 20 indications in 9 tumor types including melanoma, renal cell carcinoma, non-small cell lung cancer, bladder and triple negative breast cancer. The first pivotal studies in melanoma and renal cell carcinoma are expected to be initiated in mid-2018.
● In December 2017, Nektar submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for NKTR-262, a small molecule agonist that targets toll-like receptors (TLRs) found on innate immune cells in the body. The REVEAL Phase 1/2 study will evaluate the safety, tolerability and anti-tumor effect of NKTR-262, administered in combination with NKTR-214 (doublet) and in combination with NKTR-214 and nivolumab (triplet), in patients with locally advanced or metastatic cancers. The company plans to enroll the first patients in the REVEAL study in March of 2018.
● In November 2017, Nektar announced positive data from the dose-escalation stage of the PIVOT-02 study of NKTR-214 in combination with Opdivo at the 2017 SITC (Free SITC Whitepaper) conference. Results showed compelling response rates and favorable safety data in both PD-L1 negative and PD-L1 positive patients with melanoma, renal cell carcinoma and non-small cell lung cancer.
● In September 2017, Nektar initiated the PROPEL clinical study to evaluate the efficacy and safety of NKTR-214 in combination with approved checkpoint inhibitors, TECENTRIQ(atezolizumab) and KEYTRUDA (pembrolizumab) in patients with bladder and non-small cell lung cancer. Data from the PROPEL study is expected in the second half of 2018.
● In July 2017, Nektar and Eli Lilly announced a strategic collaboration to develop and commercialize NKTR-358, a potential first-in-class resolution therapeutic, that addresses an underlying immune system imbalance in patients with auto-immune conditions. A Phase 1 singe-ascending dose study is underway and a Phase 1/2 multiple-ascending dose study of NKTR-358 in patients with lupus is planned to begin in the second quarter of 2018.
● In July 2017, Nektar announced positive results from a Human Abuse Potential (HAP) study of NKTR-181, a first-in-class opioid analgesic.
● In May 2017, Nektar announced a new research collaboration with Takeda to explore the combination of NKTR-214 with five oncology compounds from Takeda’s cancer portfolio including a SYK-inhibitor and a proteasome inhibitor.
● In March 2017, Nektar announced positive results from the SUMMIT-07 Phase 3 efficacy study of NKTR-181 in over 600 patients with chronic low back pain. The primary efficacy endpoint of the study demonstrated significantly improved chronic back pain relief with NKTR-181 compared to placebo (p=0.0019). Key secondary endpoints of the study also achieved high statistical significance. The study demonstrated that NKTR-181 had a favorable safety profile and was well tolerated.

The company also announced upcoming presentations at the following scientific congresses during the first half of 2018:

American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2018, Chicago, IL:

● Abstract 3755/Poster 5: "Comprehensive antitumor immune activation by a novel TLR7/8 targeting agent NKTR-262 combined with CD122-biased immunostimulatory cytokine NKTR-214", Kivimae, S., et al.
○ Session: Immunology: Immunomodulatory Agents and Interventions 1
○ Session Date and Time: Tuesday, April 17, 2018, 8:00 a.m. – 12:00 p.m. Central Time
○ Location: McCormick Place South, Exhibit Hall A, Poster Section 32
● Abstract 2755/Poster 17: "NKTR-262: Prodrug pharmacokinetics in mice, rats, and dogs", Lee, M., et al.
○ Session: Immunology: Immune Mechanisms Invoked by Therapies 1
○ Session Date and Time: Monday, April 16, 2018, 1:00 p.m. – 5:00 p.m. Central Time
○ Location: McCormick Place South, Exhibit Hall A, Poster Section 33
● Abstract 123/Poster 13: "Enhanced anti-tumor activity of the combination of entinostat and NKTR-214 in renal and colon cancer tumor models", Wang, L., et al.
○ Session: Tumor Biology: Role of the Innate Immune System in Tumorigenesis
○ Session Date and Time: Sunday, April 15, 2018, 1:00 p.m. – 5:00 p.m. Central Time
○ Location: McCormick Place South, Exhibit Hall A, Poster Section 5

● Abstract 3566/Poster 4: "Enhanced expansion and tumor targeting of adoptively transferred T cells with NKTR-214", Parisi, G., et al.
○ Session: Clinical Research: Adoptive Cell Therapy 3
○ Session Date and Time: Tuesday, April 17, 2018, 8:00 a.m. – 12:00 p.m. Central Time
○ Location: McCormick Place South, Exhibit Hall A, Poster Section 24

Antigen-Specific Immune Tolerance Drug Development Summit 2018, Boston, MA:

● Preclinical Data Presentation: "NKTR-358: A Selective Regulatory T Cell Inducing Agent for the Treatment of Autoimmune and Inflammatory Diseases"
○ Presenter: Jonathan Zalevsky, Ph.D., Nektar Therapeutics
○ Date and Time: Wednesday, April 25, 2018, 4:20 p.m. Eastern Time

American Academy of Pain Medicine 34th Annual Meeting, Vancouver, BC:

● Poster: "Efficacy, safety, and tolerability of NKTR-181 in patients with moderate to severe chronic low-back pain: A Phase 3 study"
○ Presenter: John Markman, M.D., University of Rochester Medical Center
○ Session: Poster Session 2
○ Date and Time: Friday, April 27, 2018, 6:00 p.m. Pacific Time
● Poster: "Measuring withdrawal in a phase 3 study of a new analgesic, NKTR-181, in subjects with moderate-to-severe chronic low-back pain"
○ Presenter: Jack Henningfield, Ph.D., Pinney Associates
○ Session: Poster Session 2
○ Date: Friday, April 27, 2018, 6:00 p.m. Pacific Time

Treg Directed Therapy for Autoimmune Disorders Meeting, Boston, MA:

● Preclinical Data Presentation: "NKTR-358: An IL-2 Pathway Agonist that Selectively Expands and Activates Regulatory T cells for the Treatment of Allergy and Autoimmune Disease"
○ Presenter: Jonathan Zalevsky, Ph.D., Nektar Therapeutics
○ Session: Enhanced Treg-based therapy with the use of IL-2
○ Date and Time: Wednesday, May 23, 2018, 3:40 p.m. Eastern Time

3rd Annual Advances in Immuno-Oncology Congress, London, U.K.:

● Presentation: "Accessing The Potential Of An Immunotherapeutic Agent"
○ Presenter: Jonathan Zalevsky, Ph.D., Nektar Therapeutics
○ Session: Translational Immuno-Oncology
○ Date and Time: Thursday, May 24, 2018, 5:40 p.m. London Time

College on Problems of Drug Dependence 80th Annual Scientific Meeting, San Diego, CA:

● Oral Presentation: "Neuropharmacodynamic Profile of NKTR-181: Correlation to Low Abuse Potential"
○ Presenter: Laurie VanderVeen, Ph.D., Nektar Therapeutics
○ Session: Preclinical Opioid
○ Date and Time: Tuesday, June 12, 2018, 10:15 a.m. – 10:30 a.m. Pacific Time

● Oral Presentation: "Assessment of Drug Abuse-Related Events with MADDERS in SUMMIT-07: A Phase-3 Study of NKTR-181 in Patients With Moderate to Severe Chronic Low-Back Pain"

○ Presenter: Ryan K. Lanier, Ph.D., Analgesic Solutions
○ Session: Pain
○ Date and Time: Wednesday, June 13, 2018, 1:30 p.m. – 1:45 p.m. Pacific Time

Conference Call to Discuss Fourth Quarter and Year-End 2017 Financial Results
Nektar management will host a conference call to review the results beginning at 5:00 p.m. Eastern Time/2:00 p.m. Pacific Time today, Thursday, March 1, 2018.

This press release and a live audio-only Webcast of the conference call can be accessed through a link that is posted on the home page and Investors section of the Nektar website: View Source The web broadcast of the conference call will be available for replay through Monday, April 2, 2018.

To access the conference call, follow these instructions:

Dial: (877) 881.2183 (U.S.); (970) 315.0453 (international)
Passcode: 6299239 (Nektar Therapeutics is the host)

In the event that any non-GAAP financial measure is discussed on the conference call that is not described in the press release, or explained on the conference call, related information will be made available on the Investors page at the Nektar website as soon as practical after the conclusion of the conference call..

Cash and investments in marketable securities at December 31, 2017 were $353.2 million as compared to $389.1 million at December 31, 2016. This does not include $1.85 billion in upfront payments from the new Bristol-Myers Squibb collaboration, which was announced on February 14, 2018.

"This past year was truly transformational for Nektar as we achieved a number of successes with Nektar medicines across our three key therapeutic areas of immuno-oncology, immunology and pain," said Howard W. Robin, President and Chief Executive Officer of Nektar. "In the area of pain, we completed a successful Phase 3 program for NKTR-181 in over 2,100 patients and healthy volunteers that will comprise our NDA submission in the second quarter of this year. In immunology, we entered into a major partnership with Eli Lilly for NKTR-358, a potential first-in-class T regulatory resolution therapeutic, which will be developed to treat a broad range of auto-immune disorders. Finally, in immuno-oncology, the clinical success we achieved with NKTR-214 led to a groundbreaking collaboration with Bristol-Myers Squibb that now enables us to broadly and rapidly advance NKTR-214 into over 20 registrational trials in up to 15,000 patients."

Summary of Financial Results

Revenue for the fourth quarter of 2017 was $95.5 million as compared to $37.5 million in the fourth quarter of 2016. Revenue in the fourth quarter of 2017 included a total of $60.0 million of non-recurring revenue related to a new sublicense agreement, a contract settlement agreement and the recognition of deferred revenue from several collaboration agreements.

Revenue for the year ended December 31, 2017 was $307.7 million as compared to $165.4 million in 2016. Revenue in 2017 included recognition of $130.1 million of the $150.0 million upfront payment from Nektar’s collaboration with Eli Lilly & Company for the development and commercialization of NKTR-358.

Total operating costs and expenses in the fourth quarter of 2017 were $119.5 million as compared to $69.6 million in the fourth quarter of 2016. Total operating costs and expenses increased primarily as a result of higher research and development (R&D) expense. Total operating costs and expenses for the year ended December 31, 2017 were $367.4 million as compared to $278.3 million in 2016.

R&D expense in the fourth quarter of 2017 was $81.4 million as compared to $50.2 million for the fourth quarter of 2016. R&D expense for the year ended December 31, 2017 was $268.5 million as compared to $203.8 million in 2016. R&D expense was higher in 2017 as compared to 2016 primarily because of expenses for our pipeline programs, including the completion of Phase 3 clinical studies for NKTR-181, Phase 1/2 clinical studies of NKTR-214 and NKTR-358, and IND-enabling activities for NKTR-262 and NKTR-255.

General and administrative (G&A) expense was $12.3 million in the fourth quarter of 2017 as compared to $12.8 million in the fourth quarter of 2016. G&A expense for the year ended December 31, 2017 was $52.4 million as compared to $44.3 million in 2016.

Net loss for the fourth quarter of 2017 was $33.8 million or $0.21 loss per share as compared to a net loss of $42.2 million or $0.28 loss per share in the fourth quarter of 2016. Net loss for the year ended December 31, 2017 was $96.7 million or $0.62 loss per share as compared to a net loss of $153.5 million or $1.10 loss per share in 2016.

2017 and Year-to-Date Business Highlights

● In February 2018, Nektar and Bristol-Myers Squibb entered into a global development and commercialization agreement to evaluate the full potential of NKTR-214 plus Opdivo (nivolumab) in more than 20 indications in 9 tumor types including melanoma, renal cell carcinoma, non-small cell lung cancer, bladder and triple negative breast cancer. The first pivotal studies in melanoma and renal cell carcinoma are expected to be initiated in mid-2018.
● In December 2017, Nektar submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for NKTR-262, a small molecule agonist that targets toll-like receptors (TLRs) found on innate immune cells in the body. The REVEAL Phase 1/2 study will evaluate the safety, tolerability and anti-tumor effect of NKTR-262, administered in combination with NKTR-214 (doublet) and in combination with NKTR-214 and nivolumab (triplet), in patients with locally advanced or metastatic cancers. The company plans to enroll the first patients in the REVEAL study in March of 2018.
● In November 2017, Nektar announced positive data from the dose-escalation stage of the PIVOT-02 study of NKTR-214 in combination with Opdivo at the 2017 SITC (Free SITC Whitepaper) conference. Results showed compelling response rates and favorable safety data in both PD-L1 negative and PD-L1 positive patients with melanoma, renal cell carcinoma and non-small cell lung cancer.
● In September 2017, Nektar initiated the PROPEL clinical study to evaluate the efficacy and safety of NKTR-214 in combination with approved checkpoint inhibitors, TECENTRIQ(atezolizumab) and KEYTRUDA (pembrolizumab) in patients with bladder and non-small cell lung cancer. Data from the PROPEL study is expected in the second half of 2018.
● In July 2017, Nektar and Eli Lilly announced a strategic collaboration to develop and commercialize NKTR-358, a potential first-in-class resolution therapeutic, that addresses an underlying immune system imbalance in patients with auto-immune conditions. A Phase 1 singe-ascending dose study is underway and a Phase 1/2 multiple-ascending dose study of NKTR-358 in patients with lupus is planned to begin in the second quarter of 2018.
● In July 2017, Nektar announced positive results from a Human Abuse Potential (HAP) study of NKTR-181, a first-in-class opioid analgesic.
● In May 2017, Nektar announced a new research collaboration with Takeda to explore the combination of NKTR-214 with five oncology compounds from Takeda’s cancer portfolio including a SYK-inhibitor and a proteasome inhibitor.
● In March 2017, Nektar announced positive results from the SUMMIT-07 Phase 3 efficacy study of NKTR-181 in over 600 patients with chronic low back pain. The primary efficacy endpoint of the study demonstrated significantly improved chronic back pain relief with NKTR-181 compared to placebo (p=0.0019). Key secondary endpoints of the study also achieved high statistical significance. The study demonstrated that NKTR-181 had a favorable safety profile and was well tolerated.

The company also announced upcoming presentations at the following scientific congresses during the first half of 2018:

American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2018, Chicago, IL:

● Abstract 3755/Poster 5: "Comprehensive antitumor immune activation by a novel TLR7/8 targeting agent NKTR-262 combined with CD122-biased immunostimulatory cytokine NKTR-214", Kivimae, S., et al.
○ Session: Immunology: Immunomodulatory Agents and Interventions 1
○ Session Date and Time: Tuesday, April 17, 2018, 8:00 a.m. – 12:00 p.m. Central Time
○ Location: McCormick Place South, Exhibit Hall A, Poster Section 32
● Abstract 2755/Poster 17: "NKTR-262: Prodrug pharmacokinetics in mice, rats, and dogs", Lee, M., et al.
○ Session: Immunology: Immune Mechanisms Invoked by Therapies 1
○ Session Date and Time: Monday, April 16, 2018, 1:00 p.m. – 5:00 p.m. Central Time
○ Location: McCormick Place South, Exhibit Hall A, Poster Section 33
● Abstract 123/Poster 13: "Enhanced anti-tumor activity of the combination of entinostat and NKTR-214 in renal and colon cancer tumor models", Wang, L., et al.
○ Session: Tumor Biology: Role of the Innate Immune System in Tumorigenesis
○ Session Date and Time: Sunday, April 15, 2018, 1:00 p.m. – 5:00 p.m. Central Time
○ Location: McCormick Place South, Exhibit Hall A, Poster Section 5

● Abstract 3566/Poster 4: "Enhanced expansion and tumor targeting of adoptively transferred T cells with NKTR-214", Parisi, G., et al.
○ Session: Clinical Research: Adoptive Cell Therapy 3
○ Session Date and Time: Tuesday, April 17, 2018, 8:00 a.m. – 12:00 p.m. Central Time
○ Location: McCormick Place South, Exhibit Hall A, Poster Section 24

Antigen-Specific Immune Tolerance Drug Development Summit 2018, Boston, MA:

● Preclinical Data Presentation: "NKTR-358: A Selective Regulatory T Cell Inducing Agent for the Treatment of Autoimmune and Inflammatory Diseases"
○ Presenter: Jonathan Zalevsky, Ph.D., Nektar Therapeutics
○ Date and Time: Wednesday, April 25, 2018, 4:20 p.m. Eastern Time

American Academy of Pain Medicine 34th Annual Meeting, Vancouver, BC:

● Poster: "Efficacy, safety, and tolerability of NKTR-181 in patients with moderate to severe chronic low-back pain: A Phase 3 study"
○ Presenter: John Markman, M.D., University of Rochester Medical Center
○ Session: Poster Session 2
○ Date and Time: Friday, April 27, 2018, 6:00 p.m. Pacific Time
● Poster: "Measuring withdrawal in a phase 3 study of a new analgesic, NKTR-181, in subjects with moderate-to-severe chronic low-back pain"
○ Presenter: Jack Henningfield, Ph.D., Pinney Associates
○ Session: Poster Session 2
○ Date: Friday, April 27, 2018, 6:00 p.m. Pacific Time

Treg Directed Therapy for Autoimmune Disorders Meeting, Boston, MA:

● Preclinical Data Presentation: "NKTR-358: An IL-2 Pathway Agonist that Selectively Expands and Activates Regulatory T cells for the Treatment of Allergy and Autoimmune Disease"
○ Presenter: Jonathan Zalevsky, Ph.D., Nektar Therapeutics
○ Session: Enhanced Treg-based therapy with the use of IL-2
○ Date and Time: Wednesday, May 23, 2018, 3:40 p.m. Eastern Time

3rd Annual Advances in Immuno-Oncology Congress, London, U.K.:

● Presentation: "Accessing The Potential Of An Immunotherapeutic Agent"
○ Presenter: Jonathan Zalevsky, Ph.D., Nektar Therapeutics
○ Session: Translational Immuno-Oncology
○ Date and Time: Thursday, May 24, 2018, 5:40 p.m. London Time

College on Problems of Drug Dependence 80th Annual Scientific Meeting, San Diego, CA:

● Oral Presentation: "Neuropharmacodynamic Profile of NKTR-181: Correlation to Low Abuse Potential"
○ Presenter: Laurie VanderVeen, Ph.D., Nektar Therapeutics
○ Session: Preclinical Opioid
○ Date and Time: Tuesday, June 12, 2018, 10:15 a.m. – 10:30 a.m. Pacific Time

● Oral Presentation: "Assessment of Drug Abuse-Related Events with MADDERS in SUMMIT-07: A Phase-3 Study of NKTR-181 in Patients With Moderate to Severe Chronic Low-Back Pain"

○ Presenter: Ryan K. Lanier, Ph.D., Analgesic Solutions
○ Session: Pain
○ Date and Time: Wednesday, June 13, 2018, 1:30 p.m. – 1:45 p.m. Pacific Time

Conference Call to Discuss Fourth Quarter and Year-End 2017 Financial Results
Nektar management will host a conference call to review the results beginning at 5:00 p.m. Eastern Time/2:00 p.m. Pacific Time today, Thursday, March 1, 2018.

This press release and a live audio-only Webcast of the conference call can be accessed through a link that is posted on the home page and Investors section of the Nektar website: View Source The web broadcast of the conference call will be available for replay through Monday, April 2, 2018.

To access the conference call, follow these instructions:

Dial: (877) 881.2183 (U.S.); (970) 315.0453 (international)
Passcode: 6299239 (Nektar Therapeutics is the host)

In the event that any non-GAAP financial measure is discussed on the conference call that is not described in the press release, or explained on the conference call, related information will be made available on the Investors page at the Nektar website as soon as practical after the conclusion of the conference call.

On March 1, 2018 Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, today reported financial results for the three and twelve months ended December 31, 2017 (Press release, Sarepta Therapeutics, MAR 1, 2018, View Source [SID1234524329]).

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"In 2017, Sarepta advanced our ambitious vision to make a profound difference in the lives of those with Duchenne muscular dystrophy (DMD) and furthered our position as an important global genetic medicine company in rare disease. We accelerated our industry-leading DMD pipeline composed of some 16 programs across our RNA-targeted, gene therapy and gene-editing platforms; raised substantial resources to invest in our programs; and delivered on our commitment to execute a successful launch for EXONDYS 51, our first genetic medicine therapy for DMD," said Douglas Ingram, Sarepta’s president and chief executive officer.

Mr. Ingram continued, "The exceptional 2017 performance of EXONDYS 51 and the advancement of our multi-platform pipeline reflects our leadership position in DMD and our commitment to turning our vision into reality. As we track into 2018, our commitment to the DMD community is unwavering, exemplified by our continued focus on the launch of EXONDYS 51, multiple important data read outs over the year, and our progress in bringing new therapies to the DMD community with a sense of urgency."

Financial Results

For the fourth quarter of 2017, on a GAAP basis, Sarepta reported a net loss of $24.0 million, or $0.37 per share, compared to a net loss of $88.5 million for the same period of 2016, or $1.62 per share. On a non-GAAP basis, the net loss for the fourth quarter of 2017 was $18.0 million, or $0.28 per share, compared to a net loss of $38.6 million for the same period of 2016, or $0.71 per share.

For the year ended December 31, 2017, on a GAAP basis, Sarepta reported a net loss of $50.7 million, or $0.86 per share, compared to a net loss of $267.3 million for the same period of 2016, or $5.49 per share. On a non-GAAP basis, the net loss for 2017 was $88.7 million, or $1.51 per share, compared to a net loss of $191.9 million for the same period of 2016, or $3.94 per share.

Net Revenues

For the three and twelve months ended December 31, 2017, the Company recorded net product revenues of $57.3 million and $154.6 million, respectively, which reflects sales from EXONDYS 51 compared to net revenues of $5.4 million for fourth quarter of 2016. The Company did not achieve any product revenues for the first three quarters of 2016. The increase primarily reflects increasing demand for EXONDYS 51 in the U.S.

Cost and Operating Expenses

Cost of sales (excluding amortization of in-licensed rights)

For the three and twelve months ended December 31, 2017, cost of sales (excluding amortization of in-licensed rights) were $3.5 million and $7.4 million, respectively, compared to $0.1 million for the same periods of 2016. The increase primarily reflects royalty payments to BioMarin Pharmaceuticals (BioMarin) as a result of the execution of the settlement and license agreements with BioMarin in July 2017 as well as higher inventory costs related to increasing demand for EXONDYS 51 during 2017. Prior to the approval of EXONDYS 51, the Company expensed related manufacturing and material costs as research and development expenses.

Research and development

Research and development expenses were $44.4 million for the fourth quarter of 2017, compared to $70.7 million for the same period of 2016, a decrease of $26.3 million. The decrease was primarily driven by an up-front payment of $40.0 million to Summit (Oxford) Ltd. (Summit) in the fourth quarter of 2016 partially offset by increased patient enrollment in the Company’s on-going late stage clinical trials and a ramp up of preclinical studies for the Company’s PPMO platform and other follow-on exons. Non-GAAP research and development expenses were $41.8 million for the fourth quarter of 2017, compared to $27.8 million for the same period of 2016, an increase of $14.0 million. The increase was primarily due to increased patient enrollment in the Company’s on-going late-stage clinical trials and a ramp up of preclinical studies for the Company’s PPMO platform and other follow-on exons.

Research and development expenses were $166.7 million for 2017, compared to $188.3 million for the same period of 2016, a decrease of $21.6 million. The decrease was primarily driven by lower manufacturing expenses due to the capitalization of inventory following the approval of EXONDYS 51 and $40.0 million and $7.0 million up-front payments, respectively, to Summit and University of Western Australia in 2016. The decreases were partially offset by a $22.0 million payment to Summit in 2017 as a result of achieving the milestone of the last patient being dosed in the safety arm cohort to the PhaseOut DMD study, increased patient enrollment in the Company’s ongoing late-stage clinical trials and a ramp up of preclinical studies for the Company’s PPMO platform and other follow-on exons and increases in professional fees and compensation and other personnel expenses. Non-GAAP research and development expenses were $136.0 million for 2017, consistent with the same period of 2016.

Selling, general and administration

Selling, general and administrative expenses were $32.2 million for the fourth quarter of 2017, compared to $22.9 million for the same period of 2016, an increase of $9.3 million. Non-GAAP selling, general and administrative expenses were $27.3 million for the fourth quarter of 2017, compared to $16.1 million for the same period of 2016, an increase of $11.2 million. The year-over-year increases for both GAAP and non-GAAP selling, general and administrative expenses for the fourth quarter were primarily driven by increases in professional services due to global expansion and compensation and other personnel expenses.

Selling, general and administrative expenses were $122.7 million for 2017, compared to $83.7 million for the same period of 2016, an increase of $39.0 million. Non-GAAP selling, general and administrative expenses were $97.9 million for 2017, compared to $60.7 million for the same period of 2016, an increase of $37.2 million. The year-over-year increases for both GAAP and non-GAAP selling, general and administration expenses for the full year were primarily driven by increases in professional services due to global expansion, legal expenses and compensation and other personnel expenses.

EXONDYS 51 litigation and license charges and amortization of in-licensed rights

As a result of the execution of the settlement and license agreements with BioMarin in July 2017, the Company recorded $28.4 million in litigation and license charges. Additionally, the Company recognized an amortization of in-licensed rights of $1.1 million during 2017, primarily due to the BioMarin transactions.

Other Income (Loss)

Gain from sale of Priority Review Voucher

In connection with the completion of the sale of the Priority Review Voucher (PRV) in March 2017, the Company recorded a gain of $125.0 million from sale of PRV for 2017.

Interest expense and other, net

For the three and twelve months ended December 31, 2017, the Company recorded $2.7 million and $2.0 million, interest expense and other, net, respectively, compared to less than $0.1 million and $0.5 million, respectively, for the same periods of 2016. The year-over-year increases for both periods were primarily driven by accrued interest expense related to the convertible note that the Company issued in November 2017.

Cash, Cash Equivalents, Restricted Cash and Investments

The Company had $1.1 billion in cash, cash equivalents, restricted cash and investments as of December 31, 2017 compared to $329.3 million as of December 31, 2016, an increase of $760.5 million. The increase is primarily driven by the net proceeds from the Company’s equity and debt offerings, proceeds from the sale of the Company’s PRV and collection of accounts receivable related to EXONDYS 51 sales offset by up-front payments of $35.0 million related to the Company’s license and settlement agreements with BioMarin and a milestone payment of $22.0 million to Summit Therapeutics, and the use of cash to fund the Company’s ongoing operations.

Use of Non-GAAP Measures

In addition to the GAAP financial measures set forth in this press release, the Company has included certain non-GAAP measurements: non-GAAP research and development expenses, non-GAAP selling, general and administrative expenses, non-GAAP other income adjustments, non-GAAP income tax expense, non-GAAP net loss, and non-GAAP basic and diluted net loss per share, which present operating results on a basis adjusted for stock-based compensation, restructuring expenses, and other items.

1. Stock-based compensation expenses

Stock-based compensation expenses represent non-cash charges related to equity awards granted by Sarepta. Although these are recurring charges to operations, management believes the measurement of these amounts can vary substantially from period-to-period and depend significantly on factors that are not a direct consequence of operating performance that is within management’s control. Therefore, management believes that excluding these charges facilitates comparisons of the Company’s operational performance in different periods.

2. Restructuring expenses

Restructuring expenses have been excluded as the Company believes that adjusting for these items more closely represents the Company’s ongoing operating performance and financial results.

3. Other items

Management evaluates other items of expense and income on an individual basis. It takes into consideration quantitative and qualitative characteristics of each item, including (a) nature, (b) whether the items relates to the Company’s ongoing business operations, and (c) whether the Company expects the items to continue on a regular basis. These other items include the aforementioned gain from the sale of the Company’s PRV and associated income taxes, upfront license and milestone payments to Summit, EXONDYS 51 litigation and license charges and amortization of in-licensed rights.

The Company uses these non-GAAP measures as key performance measures for the purpose of evaluating operational performance and cash requirements internally. The Company also believes these non-GAAP measures increase comparability of period-to-period results and are useful to investors as they provide a similar basis for evaluating the Company’s performance as is applied by management. These non-GAAP measures are not intended to be considered in isolation or to replace the presentation of the Company’s financial results in accordance with GAAP. Use of the terms non-GAAP research and development expenses, non-GAAP selling, general and administrative expenses, non-GAAP other income adjustments, non-GAAP income tax expense, non-GAAP net loss, and non-GAAP basic and diluted net loss per share may differ from similar measures reported by other companies, which may limit comparability, and are not based on any comprehensive set of accounting rules or principles. All relevant non-GAAP measures are reconciled from their respective GAAP measures in the attached table "Reconciliation of GAAP to Non-GAAP Net Loss."

Recent Corporate Developments

— Sarepta Therapeutics Pre-Announces Fourth Quarter 2017 Revenue and Provides Full-Year 2018 Revenue Guidance for EXONDYS 51 (eteplirsen), Representing Approximately 100 Percent Year-over-Year Growth

— Sarepta Therapeutics Announces Publication of Long-Term Pulmonary Function of Eteplirsen-Treated Patients Compared to Natural History of Duchenne Muscular Dystrophy in The Journal of Neuromuscular Diseases

— Sarepta Therapeutics Elects Biopharmaceutical Veteran, Michael W. Bonney, to its Board of Directors

— Sarepta Therapeutics Announces Exercise of Initial Purchasers’ Option to Purchase Additional Convertible Senior Notes Due 2024

— Sarepta Therapeutics Prices $475 Million of Convertible Senior Notes Due 2024

— Sarepta Therapeutics Announces Proposed Offering of $375 Million of Convertible Senior Notes Due 2024

— Sarepta Therapeutics Announces FDA Clearance of IND for the Company’s PPMO Exon 51 Candidate, SRP-5051

— Sarepta Therapeutics and Nationwide Children’s Hospital Announce FDA Clearance of IND for Micro-Dystrophin Gene Therapy Program for the Treatment of Duchenne Muscular Dystrophy

— Sarepta Therapeutics and Nationwide Children’s Hospital Announce U.S. Food and Drug Administration (FDA) Clearance of the IND Application for the GALGT2 Gene Therapy Program

— Sarepta Therapeutics Signs Exclusive Global Collaboration with Duke University for Gene Editing CRISPR/Cas9 Technology to Develop New Treatments for Duchenne Muscular Dystrophy (DMD)

Conference Call

The Company will be hosting a conference call at 4:30 p.m. Eastern Time, to discuss these financial results and provide a corporate update. The conference call may be accessed by dialing 844-534-7313 for domestic callers and +1-574-990-1451 for international callers. The passcode for the call is 7396848. Please specify to the operator that you would like to join the "Sarepta Fourth Quarter and Full-Year 2017 Earnings Call". The conference call will be webcast live under the investor relations section of Sarepta’s website at www.sarepta.com and will be archived there following the call for 90 days. Please connect to Sarepta’s website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.

About EXONDYS 51

EXONDYS 51 uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene. EXONDYS 51 is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein.

Important Safety Information About EXONDYS 51

Hypersensitivity reactions, including rash and urticaria, pyrexia, flushing, cough, dyspnea, bronchospasm, and hypotension, have occurred in patients who were treated with EXONDYS 51. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy.

Adverse reactions in DMD patients (N=8) treated with EXONDYS 51 30 or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 25% more than placebo (N=4) (Study 1, 24 weeks) were (EXONDYS 51, placebo): balance disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most common adverse reactions were balance disorder and vomiting. Because of the small numbers of patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is not recommended.

In the 88 patients who received ³30 mg/kg/week of EXONDYS 51 for up to 208 weeks in clinical studies, the following events were reported in ³10% of patients and occurred more frequently than on the same dose in Study 1: vomiting, contusion, excoriation, arthralgia, rash, catheter site pain, and upper respiratory tract infection.

For further information, please see the full Prescribing Information.

On March 1, 2018 Idera Pharmaceuticals, Inc. (NASDAQ:IDRA), a clinical-stage biopharmaceutical company developing toll-like receptor and RNA therapeutics for patients with rare cancers and rare diseases, reported the start of a Phase 3 global, multi-center, open-label clinical trial to evaluate the efficacy and safety of intratumoral IMO-2125 in combination with ipilimumab (Yervoy) versus ipilimumab alone in subjects with anti-PD-1 refractory melanoma (NCT03445533) (Press release, Idera Pharmaceuticals, MAR 1, 2018, View Source [SID1234524649]). The initiation of the Phase 3 trial follows the announcement that IMO-2125 was granted Fast-Track designation by the U.S. Food and Drug Administration, a designation designed to expedite the development and review of drugs with the potential to treat serious or life-threatening conditions such as refractory metastatic melanoma.

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Results presented at the 2017 European Society for Medical Oncology Congress (ESMO) (Free ESMO Whitepaper) meeting from the completed phase 1 portion of the ongoing Phase 1/2 clinical trial of intratumoral IMO-2125 in combination with ipilimumab (NCT02644967) show the combination to be well-tolerated over the entire range of IMO 2125 doses tested, with biopsy evidence for dendritic cell activation followed by infiltration of tumor specific immune cells. These results included 9 patients treated with the Recommended Phase 2 Dose (RP2D) of 8mg of which 4 (44%) achieved RECIST v1.1 responses, including one durable Complete Response (CR), with 6 of 9 (67%) patients experiencing disease control (CR, PR, or SD ≥ 12 weeks).

"For our advanced melanoma patients who have not benefited from anti-PD-1 therapy and in BRAF mutant melanoma, BRAF targeted therapy, there are very few, limited options available today," stated Ahmad Tarhini, M.D., Ph.D, Director, Melanoma and Skin Cancer Program, Cleveland Clinic Taussig Cancer Institute. "I am encouraged by the data that has been demonstrated to date with IMO-2125 and am hopeful that this Phase 3 trial offers hope to this large group of patients whose treatment options are very limited."

"We are excited to initiate the ILLUMINATE 301 Trial, grateful to our advisors who have worked with us to design this pivotal trial and very encouraged by the enthusiasm of investigators to participate in the study. We look forward to working together to conduct and complete this trial, so that we may bring IMO-2125 to the market as soon as possible for patients who are not benefiting from anti-PD-1 therapy," stated Joanna Horobin, M.B., Ch.B., Idera’s Chief Medical Officer.

ILLUMINATE 301 Trial Design
ILLUMINATE 301 is planned in approximately 80 sites, across 10 countries, and expected to enroll approximately 300 subjects with advanced melanoma who have confirmed disease progression while on nivolumab (Opdivo) or pembrolizumab (Keytruda). Subjects will be randomized into two treatment arms (IMO-2125 in combination with ipilimumab vs. ipilimumab monotherapy). Subjects must have histologically confirmed metastatic melanoma with measurable (by Response Evaluation Criteria in Solid Tumors [RECIST] v1.1), Stage III (lymph node or in transit lesions) or Stage IVA, IVB, or IVC disease, and at least one lesion that is accessible for injection.

This Phase 3 study is being performed to provide definitive evidence for superiority of the IMO-2125 and ipilimumab combination over ipilimumab. The reported overall response rate for ipilimumab monotherapy following anti-PD-1 therapy is 13%1. The primary endpoint family includes overall survival and overall response rate. For more information on the study and patient qualifications, visit www.ideraclinicaltrials.com.

About IMO-2125
IMO-2125 is a toll-like receptor (TLR) 9 agonist that received orphan drug designation from the US Food and Drug Administration (FDA) in 2017 for the treatment of melanoma Stages IIb to IV. It signals the immune system to create and activate cancer-fighting cells (T-cells) to target solid tumors in refractory melanoma patients. Currently approved immuno-oncology treatments for patients with metastatic melanoma, specifically check-point inhibitors, work for some but not all, as many patients’ immune response is missing or weak and thus they do not benefit from the checkpoint therapy making them so-called "refractory." The combination of ipilimumab and IMO-2125 appears to activate an immune response in these patients who have exhausted all options. Intratumoral injections with IMO-2125 are designed to selectively enable the T-cells to recognize and attack cancers that remained elusive and unrecognized by the immune system exposed to checkpoint inhibitors alone, while limiting toxicity or impact on healthy cells in the body.

About Metastatic Melanoma
Melanoma is a type of skin cancer that begins in a type of skin cell called melanocytes. As is the case in many forms of cancer, melanoma becomes more difficult to treat once the disease has spread beyond the skin to other parts of the body such as the lymphatic system (metastatic disease). Because melanoma occurs in younger individuals, the years of life lost to melanoma are also disproportionately high when compared with other cancers. Although melanoma is a rare form of skin cancer, it comprises over 75% of skin cancer deaths. The American Cancer Society estimates that there were approximately 76,000 new invasive melanoma cases and 10,000 deaths from the disease in the USA in 2016. Additionally, according to the World Health Organization, about 132,000 new cases of melanoma are diagnosed around the world every year.

Nektar Therapeutics has filed a 10-K – Annual report [Section 13 and 15(d), not S-K Item 405] with the U.S. Securities and Exchange Commission (Filing, 10-K, Nektar Therapeutics, 2018, MAR 1, 2018, View Source [SID1234524281]).

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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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Nektar Therapeutics has filed a 10-K – Annual report [Section 13 and 15(d), not S-K Item 405] with the U.S. Securities and Exchange Commission .

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!