On February 29, 2016 Kite Pharma, Inc. (Nasdaq:KITE), a clinical-stage biopharmaceutical company focused on developing engineered autologous cell therapy (eACT) products for the treatment of cancer, reported a corporate update and reported full-year and fourth quarter 2015 financial results for the period ended December 31, 2015 (Press release, Kite Pharma, FEB 29, 2016, View Source [SID:1234509308]).

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"2015 was a year of solid execution and transformation for Kite," noted Arie Belldegrun, M.D., FACS, Chairman, President, and Chief Executive Officer. "The development of KTE-C19, our breakthrough immunotherapy candidate for patients with refractory hematological malignancies who have limited or no treatment options, is currently advancing in four Kite-sponsored multi-center clinical studies. We expect interim pivotal data from ZUMA-1, our lead study of KTE-C19, in the second half of this year, positioning us to submit our registrational filing with the FDA by year end. Construction of our commercial manufacturing facility is complete, and commercial planning is currently progressing under the leadership of our Chief Commercial Officer, Shawn Tomasello, and her capable team."

Dr. Belldegrun continued, "We are advancing what we believe is the most robust clinical pipeline of chimeric antigen receptors and T cell receptors for both solid and hematological oncology. This effort is supported by our ongoing collaborations with distinguished academic and industry leaders in immuno-oncology. We expect that our pipeline progress in 2016 will include additional human proof of concept data supporting the potential of our TCRs for the treatment of solid tumors."

Full Year 2015 and Recent Highlights

KTE-C19 Program

Initiated Kite’s lead multi-center study (ZUMA-1) for KTE-C19 for the treatment of aggressive, refractory diffuse large B cell lymphoma (DLBCL), primary mediastinal B cell lymphoma (PMBCL), and transformed follicular lymphoma (TFL). DLBCL is the most common form of non-Hodgkin Lymphoma (NHL).

Announced positive data from the Phase 1 portion of ZUMA-1 and advanced the study to the pivotal phase. The overall safety, efficacy, and biomarker data were generally consistent with previously published data from the National Cancer Institute (NCI).

Initiated three additional pivotal multi-center studies of KTE-C19 for the treatment of relapsed/refractory mantle cell lymphoma (MCL), adult relapsed/refractory acute lymphoblastic leukemia (ALL), and pediatric relapsed/refractory ALL.

Received Breakthrough Therapy Designation for KTE-C19 for the treatment of patients with refractory DLBCL, PMBCL, and TFL.

Secured Orphan Drug Designations in the EU for KTE-C19 for all of Kite’s hematological indications.

Completed Kite’s clinical manufacturing facility that is manufacturing KTE-C19 and that will manufacture future product candidates.

Completed construction of Kite’s commercial manufacturing facility, which is on track for qualification and validation later this year.

Acquisition and Strategic Collaborations

Established European operations with the acquisition of T-Cell Factory, now known as Kite Pharma EU. Through this acquisition, Kite also gained the proprietary TCR-GENErator discovery platform developed by Ton Schumacher, Ph.D., Chief Scientific Officer of Kite Pharma EU.

Expanded clinical and research partnership with the NCI including:
An enhanced Cooperative Research and Development Agreement (CRADA) with the NCI’s Surgery Branch to advance multiple CAR and TCR programs led by Steven Rosenberg, M.D., Ph.D.
A separate CRADA with the NCI’s Experimental Transplantation and Immunology Branch to advance the development of a fully human anti-CD19 CAR product candidate. James Kochenderfer, M.D. is leading the Phase 1 study.

Expanded agreement with the Netherlands Cancer Institute for the exclusive option to license multiple TCR gene sequences for the development and commercialization of immunotherapy candidates targeting solid tumors.

Entered into a research and license agreement with Leiden University Medical Center in the Netherlands to identify and develop TCR product candidates targeting solid tumors that are associated with the human papillomavirus (HPV) type 16 infection.

Partnered with Amgen to develop and commercialize the next generation of novel CAR T cell immunotherapies.

Entered into a collaboration with bluebird bio to advance second-generation TCR cell therapy products to treat HPV-associated cancers.

Secured an exclusive license to Alpine Immune Sciences’ transmembrane immunomodulatory protein (TIPTM) technology for eACT-based products.

Corporate Milestones

Strengthened the organization with the addition of more than 100 new hires across all functions.

Formed Kite’s integrated commercial leadership team, led by Shawn Tomasello, Kite’s Chief Commercial Officer.

Appointed Dr. Franz B. Humer, former Chairman and Chief Executive of Roche Holding Ltd., to Kite’s Board of Directors.

Strengthened Kite’s Scientific Advisory Board with the addition of James Allison, Ph.D. and Padmanee Sharma, M.D., Ph.D., recognized immunotherapy leaders from MD Anderson Cancer Center.

Raised $272.6 million in net proceeds from a public offering in December 2015.

2016 Goals

Announce interim Phase 2 pivotal data from the ZUMA-1 study for the first 50 patients with 90-day follow-up in the second half of 2016.

Submit the KTE-C19 registration filing to the U.S. Food and Drug Administration (FDA) based on interim data from ZUMA-1 study by the end of 2016.

Expand KTE-C19 clinical program to Europe in preparation for a European registration filing in 2017.

Complete the qualification and validation testing of the commercial manufacturing facility in anticipation of pre-approval inspection by the FDA.

Initiate a second series of KTE-C19 studies for additional indications and earlier lines of therapy in DLBCL patients.

Present longer term follow-up KTE-C19 data in patients with refractory aggressive DLBCL from the Phase 1 portion of ZUMA-1.

File investigational new drug (IND) application with the FDA for a TCR product candidate that targets a MAGE antigen expressed on solid tumors.

Advance the Kite-Amgen collaboration resulting in the filing of an IND application with the FDA for a next generation CAR T cell immunotherapy candidate, the first of multiple INDs expected to originate from the collaboration.

Fourth Quarter and Full Year 2015 Financial Results

Revenue was $4.9 million for the fourth quarter of 2015 compared to $0 for the fourth quarter of 2014, and $17.3 million for the full year of 2015, compared to $0 for the full year of 2014. The increase was primarily due to revenue recognized under the Kite-Amgen collaboration.

Research and development expenses were $28.8 million for the fourth quarter of 2015, compared to $7.9 million for the fourth quarter of 2014, and $76.4 million for the full year of 2015 compared to $23.1 million in 2014. The full-year increase of $53.3 million was primarily attributable to a $31.5 million increase in research and development and manufacturing expenses supporting the advancement of KTE-C19 and other programs, $11.8 million in expenses related to increased personnel and consulting costs, and $10.0 million of non-cash stock-based compensation expense.

General and administrative expenses were $14.1 million for the fourth quarter of 2015, compared to $5.4 million for the fourth quarter of 2014, and $44.2 million for the full year of 2015, compared to $13.6 million in 2014. The full-year increase of $30.6 million was primarily attributable to an $8.2 million increase in personnel related expenses, $7.8 million for public company expenses and license obligations, and $14.6 million of non-cash stock-based compensation.

Net loss attributable to common stockholders was $38.2 million, or $0.85 per share, for the fourth quarter of 2015, compared to $13.0 million, or $0.33 per share, for the fourth quarter of 2014. For the full year of 2015, the net loss was $101.7 million, or $2.33 per share, compared to $43.7 million, or $1.91 per share, in 2014.

Non-GAAP net loss attributable to common stockholders for the fourth quarter of 2015 was $24.5 million, or $0.54 per share, which excludes non-cash stock-based compensation expense of $13.8 million for the fourth quarter of 2015. Non-GAAP net loss attributable to common stockholders for the full year of 2015 was $61.0 million, or $1.40 per share, which excludes non-cash stock-based compensation expense of $40.7 million for the full year of 2015.

As of December 31, 2015, Kite had $614.7 million in cash, cash equivalents, and marketable securities, compared to $367.0 million as of December 31, 2014.

Kite expects the full year 2016 net cash burn to be $235 to $250 million dollars, which includes approximately $20 million in capital expenditures, but excludes any inflows or outflows from business development activities. The estimated full year 2016 net cash burn is primarily driven by an estimated net loss of $295 to $310 million, which includes an estimated $80 million of non-cash stock-based compensation expense.

On February 29, 2016 Puma Biotechnology, Inc. (NYSE: PBYI), a biopharmaceutical company, reported financial results for the fourth quarter and year ended December 31, 2015 (Filing, Q4/Annual, Puma Biotechnology, 2015, FEB 29, 2016, View Source [SID:1234509300]).

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Unless otherwise stated, all comparisons are for the fourth quarter and full year 2015 compared to the fourth quarter and full year 2014, respectively.

Based on accounting principles generally accepted in the United States (GAAP), Puma reported a net loss applicable to common stock of $61.7 million, or $1.90 per share, for the fourth quarter of 2015, compared to a net loss of $47.5 million, or $1.57 per share, for the fourth quarter of 2014. Net loss applicable to common stock for the full year 2015 was $239.3 million, or $7.45 per share, compared to $142.0 million, or $4.73 per share, for the full year 2014.

Non-GAAP adjusted net loss was $40.0 million, or $1.23 per share, for the fourth quarter of 2015, compared to non-GAAP adjusted net loss of $31.1 million, or $1.03 per share, for the fourth quarter of 2014. Non-GAAP adjusted net loss for the full year 2015 was $144.3 million, or $4.49 per share, compared to $102.8 million, or $3.43 per share, for the full year 2014. Non-GAAP adjusted net loss excludes stock-based compensation expense, which represents a significant portion of overall expense and has no impact on the Company’s cash position. For a reconciliation of GAAP net loss to non-GAAP adjusted net loss and GAAP net loss per share to non-GAAP adjusted net loss per share, please see the financial tables at the end of this news release.

Net cash used in operating activities for the fourth quarter of 2015 was $33.3 million. Net cash used in operating activities for the full year 2015 was $154.5 million. At December 31, 2015, Puma had cash and cash equivalents of $31.6 million and marketable securities of $184.3 million, compared to cash and cash equivalents of $38.5 million and marketable securities of $102.8 million at December 31, 2014. Puma’s current level of cash and cash equivalents and marketable securities includes net proceeds of approximately $205.1 million from a public offering of the Company’s common stock, which was completed in January 2015.

"During the fourth quarter of 2015, Puma accomplished several clinical and regulatory milestones that contributed significant value to the shareholders of the Company," said Alan H. Auerbach, Chairman, Chief Executive Officer and President of Puma. "From the clinical perspective, in December we reported additional data from our Phase III ExteNET trial at the San Antonio Breast Cancer Symposium (SABCS), which continued to demonstrate that after 3 years of follow up, treatment with neratinib resulted in a statistically significant benefit in disease-free survival in patients with early stage HER2 positive breast cancer who had completed one year of adjuvant trastuzumab therapy. In addition, at SABCS we presented interim data from the Phase II trial of neratinib in patients with HER2-negative breast cancer who have a HER2 mutation that showed compelling benefit with neratinib, both as a monotherapy and in combination with fulvestrant. We also announced the interim data from our Phase II trial of neratinib monotherapy in patients with early stage HER2- positive breast cancer who had completed treatment with adjuvant trastuzumab, which demonstrated that using the loperamide prophylaxis with neratinib monotherapy treatment resulted in a lower rate of overall diarrhea, specifically grade 3 diarrhea, and resulted in diarrhea that was short term in duration and self-limiting. From the regulatory perspective, in November we announced that based on our Marketing Authorization Application (MAA) pre-submission meeting with the European Medicines Agency (EMA), at which the EMA assessed that there were no critical concerns that would prevent us from submitting a complete MAA for European centralized review in support of neratinib for the extended adjuvant treatment of HER2-positive early stage breast cancer in patients who have previously been treated with a trastuzumab-containing regimen, that we anticipate submitting an MAA for neratinib in this indication in the first half of 2016.

"We look forward to continuing to contribute value to shareholders with neratinib during 2016. We anticipate (i) submitting a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) during the first quarter of 2016 and submitting the MAA to the EMA during the first half of 2016 for neratinib for the extended adjuvant treatment of HER2-positive early stage breast cancer based on the positive ExteNET Phase III trial; (ii) reporting additional data from the Phase II trial of neratinib as an extended adjuvant treatment in HER2-positive early stage breast cancer using loperamide prophylaxis during the first half of 2016; (iii) reporting additional Phase II data from the FB-7 neoadjuvant HER2-positive breast cancer trial in the subgroup of patients who are MammaPrint High, during the first half of 2016; (iv) reporting Phase II data from an investigator sponsored trial of neratinib in patients with HER2-negative breast cancer who have a HER2 mutation in mid-2016; (v) reporting data from the Phase III trial of neratinib in third-line HER2-positive metastatic breast cancer patients in either the fourth quarter of 2016 or the first quarter of 2017; (vi) reporting data from the Phase II trial of neratinib in metastatic breast cancer patients with brain metastases during the fourth quarter of 2016; and (vii) reporting data from the Phase II trial of neratinib plus fulvestrant in patients with HER2 non-amplified breast cancer that has a HER2 mutation during the fourth quarter of 2016."

Operating Expenses
Operating expenses were $62.1 million for the fourth quarter of 2015, compared to $47.6 million for the fourth quarter of 2014. Operating expenses for the full year 2015 were $239.3 million compared to $142.3 million for the full year 2014.

General and Administrative Expenses:
General and administrative expenses were $9.6 million for the fourth quarter of 2015, compared to $8.1 million for the fourth quarter of 2014. General and administrative expenses for the full year 2015 were $31.8 million compared to $19.4 million for the full year 2014. Approximately $8.0 million of the year-over-year increase resulted from an increase in stock-based compensation expense, with the majority of the remaining increase made up of approximately $2.2 million in professional fees, $0.9 million in payroll and related expenses and $0.6 million in facility and equipment costs. These increases reflect our corporate growth.

Research and Development Expenses:
Research and development expenses were $52.5 million for the fourth quarter of 2015, compared to $39.5 million for the fourth quarter of 2014. Research and development expenses for the full year 2015 were $208.5 million, compared to $122.9 million for the full year 2014. Approximately $47.8 million of the year-over-year increase resulted from an increase in stock-based compensation expense, with the majority of the remaining increase made up of approximately $22.9 million in clinical trial expense, $9.0 million in internal costs such as payroll and related expenses, and $5.9 million in expenses related to consultants and contractors. These increases reflect our increased clinical trial activity.

On February 29, 2016 Exelixis, Inc. (Nasdaq: EXEL) reported financial results for the fourth quarter and full year of 2015 and provided an overview of key 2016 corporate objectives and clinical development milestones (Filing, Q4/Annual, Exelixis, 2015, FEB 29, 2016, View Source [SID:1234509290]).

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Corporate Updates and Key Priorities for 2016
In 2016, Exelixis will continue to focus its development efforts and financial resources on the opportunities for cabozantinib in advanced renal cell carcinoma (RCC) and advanced hepatocellular carcinoma (HCC). With regulatory applications under review for advanced RCC in the United States and European Union (EU), Exelixis is actively preparing for the potential commercialization of cabozantinib as a treatment for patients with advanced RCC and will soon be launch-ready for this indication should a positive regulatory decision come in the United States.
At the same time, Exelixis is working with its partner Genentech, a member of the Roche Group, to co-promote COTELLICTM (cobimetinib) in the United States. COTELLIC received U.S. approval in November 2015 as a treatment for patients with a BRAF V600E or V600K mutation-positive advanced melanoma, in combination with vemurafenib, also known as Zelboraf. Exelixis is entitled to an initial equal share of U.S. profits and losses, which will decrease as sales increase, and currently shares equally in U.S. marketing and commercialization costs. COTELLIC is also approved in the EU, Canada and Switzerland, and Exelixis will receive low double-digit royalties based upon sales outside the United States.

Cabozantinib Highlights
METEOR Trial of Cabozantinib in Advanced Renal Cell Carcinoma Delivers Positive Overall Survival Results. On February 1, 2016, Exelixis announced that a second interim analysis for overall survival (OS), a secondary endpoint of the METEOR pivotal trial, showed a highly statistically significant and clinically meaningful increase in OS for patients randomized to cabozantinib as compared to everolimus. As a result, among all the existing agents evaluated in large pivotal trials in patients with advanced RCC, including nivolumab, cabozantinib is the first and only therapy to unequivocally demonstrate robust and statistically-significant improvements in all three key efficacy parameters of OS, progression-free survival (PFS), and objective response rate (ORR). Exelixis has shared these data with U.S. and EU regulators and intends to present the results at a major medical meeting this year.

Additional Positive Data Presented from Subgroup Analyses from METEOR Trial. In January 2016, Exelixis announced positive results from subgroup analyses of the METEOR trial. This analysis contributed important details to the previously-released results conducted at the time of primary endpoint, demonstrating that the PFS and ORR benefits derived from cabozantinib treatment were consistent across various prespecified and post-hoc analysis subgroups. Importantly, observed benefits were independent of the location and number of organ metastases, tumor burden, the type, duration and number of prior VEGF receptor TKI therapies, and prior PD-1/PD-L1 therapy. These data were presented on January 9, 2016 at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2016 Genitourinary Cancers Symposium in San Francisco, CA.

U.S. Food and Drug Administration Accepts Filing for Advanced RCC, Grants Priority Review, and Assigns Action Date. In late January 2016, the U.S. Food and Drug Administration (FDA) deemed Exelixis’ New Drug Application (NDA) for cabozantinib as a treatment for patients with advanced RCC who have received one prior therapy to be sufficiently complete to permit a substantial review. The FDA also granted Priority Review designation to the filing and assigned a Prescription Drug User Fee Act action date of June 22, 2016. The NDA was considered filed on February 20, 2016, sixty days following submission.

European Medicines Agency Validates Advanced RCC Regulatory Filing, Grants Accelerated Assessment. On January 28, 2016, Exelixis announced that the European Medicines Agency (EMA) has accepted for review the company’s Marketing Authorization Application (MAA) for cabozantinib as a treatment for patients with advanced RCC who have received one prior therapy. With accelerated assessment, the MAA is eligible for a 150-day review, versus the standard 210 days (excluding clock stops when information is requested by the EMA).

Enrollment in CELESTIAL Continues; Data Anticipated in 2017. Exelixis continues to make progress in enrollment in CELESTIAL, a phase 3 pivotal trial comparing cabozantinib to placebo in patients with advanced HCC who have previously been treated with sorafenib. The study was initiated in September 2013. The trial is designed to enroll 760 patients at approximately 200 sites. Patients are being randomized 2:1 to receive 60 mg of cabozantinib daily or placebo. The primary endpoint for CELESTIAL is OS, and the secondary endpoints include PFS and ORR. Exelixis continues to anticipate top-line results from CELESTIAL in 2017. At this time, there is no approved treatment for HCC patients who progress following sorafenib treatment, the current standard of care.

Broad Cabozantinib Development Program Continues to Expand through NCI and Independent Investigators. While Exelixis pursues cabozantinib’s late-stage development in advanced RCC and advanced HCC, earlier-stage investigation continues through the company’s collaboration with the National Cancer Institute’s Cancer Therapy Evaluation Program (NCI-CTEP), and its ongoing Investigator-Sponsored Trial (IST) program. Through these two programs, there are more than 45 ongoing or planned studies including trials in advanced RCC, bladder cancer, colorectal cancer, non-small cell lung cancer, and endometrial cancer. Results are expected from the following clinical studies this year:

• CABOSUN, the randomized phase 2 trial comparing cabozantinib to sunitinib in the treatment of first-line intermediate or poor risk RCC patients, which completed enrollment in early 2015. CABOSUN is being conducted by The Alliance for Clinical Trials in Oncology as part of Exelixis’ collaboration with the NCI-CTEP;

• A phase 1b trial of cabozantinib plus nivolumab alone, or in combination with ipilimumab, in patients with genitourinary tumors, including bladder cancer and RCC; and

• A phase 2 trial evaluating single agent cabozantinib in recurrent endometrial cancer.

Cobimetinib Highlights
Regulatory Approvals for COTELLIC Granted in the United States, European Union and Canada. In November 2015, Exelixis announced that the FDA approved COTELLIC as a treatment for patients with unresectable or metastatic melanoma with a BRAF V600E or V600K mutation, in combination with vemurafenib.

Also that month, Exelixis announced that the European Commission approved COTELLIC for use in combination with vemurafenib for the treatment of adult patients in the EU with unresectable or metastatic melanoma with a BRAF V600E or V600K mutation. Additionally, in February 2016, Health Canada approved COTELLIC in combination with vemurafenib for the treatment of patients with unresectable or metastatic melanoma with a BRAF V600 mutation.

Positive Overall Survival Data for COTELLIC in Combination with Vemurafenib in Advanced Melanoma. In October 2015, Exelixis announced that the phase 3 coBRIM trial of COTELLIC in combination with vemurafenib met its secondary endpoint of demonstrating a statistically significant and clinically meaningful increase in OS for patients with unresectable locally advanced or metastatic melanoma carrying the BRAF V600E or V600K mutation. These data were the subject of a presentation at the Society for Melanoma Research 2015 Congress.

2016 Financial Guidance
The Company anticipates that operating expenses for the full year 2016 will be between $240 million and $270 million, including approximately $30 million of non-cash items related to stock-based compensation expense.

"Exelixis began 2016 with significant momentum as a result of the major milestones that occurred during and shortly after the fourth quarter," said Michael M. Morrissey, Ph.D., president and chief executive officer of Exelixis. "Most notably, we now have a more complete picture of cabozantinib’s clinical activity and potential in advanced renal cell carcinoma, a patient population greatly in need of new treatment options. With the announcement of positive overall survival data earlier this month, cabozantinib is now the only therapy to demonstrate in a phase 3 trial statistically significant improvements as compared to an active comparator, everolimus, in the three key efficacy parameters of overall survival, progression-free survival, and objective response rate in previously-treated patients with advanced renal cell carcinoma. As regulators continue to review our submitted applications, we are on track to be commercially ready in the United States by April 1, should we receive a regulatory decision in advance of the June 22 PDUFA date. And finally, with this afternoon’s announcement, in Ipsen we now have the ideal partner to maximize the potential for cabozantinib to have a positive impact on the treatment of cancer on a global basis."

"Our second Exelixis-discovered compound, cobimetinib, also saw numerous milestones in the fourth quarter, including regulatory approval in the United States and European Union, as well as the presentation of overall survival results in advanced melanoma. Approval in Canada was also obtained this month. The collective progress in advancing both of these compounds sets the company up for an impactful year, and we remain grateful for the support of our stakeholders as we continue to make progress in our mission to meaningfully improve the care and outcomes for people with cancer."

Fourth Quarter and Full Year 2015 Financial Results
Net revenues for the quarter ended December 31, 2015 were $9.9 million, and consisted almost entirely of net product revenue from the sale of COMETRIQ. This is compared to $7.4 million for the comparable period in 2014.

For the year ended December 31, 2015, net revenues were $37.2 million, compared to $25.1 million for the comparable period in 2014. Net revenues for the year ended December 31, 2015 included $3.0 million of contract revenues for a milestone payment received from Merck in the third quarter of 2015 related to their worldwide license of our PI3K-delta program as well as the net product revenue related to the sale of COMETRIQ.

Research and development expenses for the quarter ended December 31, 2015 were $23.5 million, compared to $39.7 million for the comparable period in 2014; and for the year ended December 31, 2015 were $96.4 million, compared to $189.1 million for the comparable period in 2014. The decreases for both the quarter and year ended December 31, 2015 were primarily related to a net decrease in clinical trial costs related to COMET, the Company’s phase 3 trial in metastatic castration-resistant prostate cancer and METEOR, the Company’s phase 3 trial in advanced RCC, and to a lesser degree, decreases in personnel related expenses resulting from an overall reduction in headcount. Those decreases were partially offset by an increase in stock-based compensation expense for performance-based stock-options tied to the positive top-line data received from the METEOR trial and the anticipated acceptance of our NDA filing with the FDA.

Selling, general and administrative expenses for the quarter ended December 31, 2015 were $17.1 million, compared to $9.8 million for the comparable period in 2014; and for the year ended December 31, 2015 were $57.3 million, compared to $50.8 million for the comparable period in 2014. The increases for both the quarter and year ended December 31, 2015 were primarily related to stock-based compensation expense due to the vesting of performance-based stock-options as a result of the positive top-line data received from the METEOR trial and the anticipated acceptance of our NDA filing with the FDA and higher marking expenses. Our 2015 selling, general and administrative expenses include a portion of COTELLIC commercialization expenses allocated to the collaboration which are under discussion between Exelixis and Genentech.

The overall selling, general and administrative expenses increases were partially offset by a decrease in facilities costs and consulting and outside services. For the year ended December 31, 2015, there were also decreases in personnel related expenses resulting from an overall reduction in headcount and patent defense costs as compared to the comparable period in 2014.

Other income (expense), net for the quarter ended December 31, 2015 was a net expense of ($12.0) million compared to ($11.9) million for the comparable period in 2014. Other income (expense), net for the year ended December 31, 2015 was a net expense of ($48.3) million compared to $(44.3) million for the comparable period in 2014. The net expense is comprised primarily of interest expense which includes $7.1 million and $28.9 million, respectively of non-cash expense related to the accretion of the discounts on both the 4.25% Convertible Senior Subordinated Notes due 2019 and the Company’s indebtedness under the Deerfield Notes for the quarter and year ended December 31, 2015, as compared to $7.7 million and $29.5 million for the comparable periods in 2014.

Net loss for the quarter ended December 31, 2015 was ($43.6) million, or ($0.19) per share, basic, compared to ($58.0) million, or ($0.30) per share, basic, for the comparable period in 2014. Net loss for the year ended December 31, 2015 was ($169.7) million, or ($0.81) per share, basic, compared to ($268.5) million, or $(1.38) per share, basic, for the comparable period in 2014. The decreases in net loss for both the quarter and year were primarily due to decreases in research and development expenses and an increase in net revenues, partially offset by an increase in selling, general and administrative expenses.
Cash and cash equivalents, short- and long-term investments and short- and long-term restricted cash and investments totaled $253.3 million at December 31, 2015 compared to $242.8 million at December 31, 2014.

On February 29, 2016 Intrexon Corporation (NYSE: XON), a leader in synthetic biology, reported its fourth quarter and full year results for 2015 (Filing, Q4/Annual, Intrexon, 2015, FEB 29, 2016, View Source [SID:1234509288]).

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Business Highlights and Recent Developments:

• Currently engaging with agencies of numerous governments and non-governmental organizations concerning the potential use of Oxitec’s OX513A to reduce or eradicate populations of the Aedes aegypti mosquito, the primary vector for dengue, chikungunya, and Zika virus;

• Announced expansion of Oxitec’s ‘Friendly Aedes aegypti Project’ in Piracicaba, Brazil to an area covering 35,000 to 60,000 residents following strong results demonstrating 82% reduction in wild Ae. aegypti larvae. In preparation of this growing program and to meet increasing demand for its proprietary vector control solution, Oxitec is initiating a new mosquito production facility in Piracicaba that will have capacity to protect over 300,000 people;

• Entered into research collaboration with Janssen Pharmaceutica NV, one of the Janssen Pharmaceutical Companies of Johnson & Johnson, to discover and develop ActoBiotics therapies directed against selected targets to treat Type 2 diabetes (T2D), obesity and/or metabolic disorders related to energy dysregulation. The ActoBiotics oral delivery system for biological effectors is well suited to tackle multiple aspects of T2D;

• In addition to Janssen, signed three additional health collaborations in 2015 utilizing the ActoBiotics platform: Oragenics (NYSE MKT: OGEN) for treatment of diseases of the oral cavity, Synthetic Biologics (NYSE MKT: SYN) for treatment of the metabolic disorder phenylketonuria, and ZIOPHARM Oncology (NASDAQ: ZIOP) for treatment of graft-versus-host-disease;

• Expanded relationship with Fibrocell Science via a new Exclusive Channel Collaboration (ECC) for the development of genetically-modified fibroblasts to treat chronic inflammatory and degenerative diseases of the joint, including arthritis and related conditions, with cell-based therapeutics that have been modified to express one or more proteins at sites of joint inflammation helping overcome the limitations of existing treatment approaches;

• Entered into a multi-year collaboration with the Harvest Intrexon Enterprise Fund, sponsored by Harvest Capital Strategies, LLC. The fund, which raised $245 million, is dedicated to the inventions and discoveries of Intrexon suitable for pursuit by a startup;

• Announced first ECC with a startup, Thrive Agrobiotics, Inc., backed by the Harvest Intrexon Enterprise Fund, utilizing the ActoBiotics platform for expression of nutritive proteins to improve the overall growth and feed efficiency in weaning piglets;

• Established Intrexon Energy Partners II, a joint venture with a select group of external investors employing the Company’s proprietary gas-to-liquids bioconversion platform utilizing methanotrophs to produce 1,4-butanediol (BDO), a key chemical intermediate with a global market value exceeding $5 billion used to manufacture spandex, polyurethane, plastics, as well as polyester;
• Announced exclusive agreement between Intrexon Energy Partners (IEP) and Dominion Energy, a subsidiary of Dominion Resources (NYSE: D), to explore the potential for commercial-scale biological conversion of natural gas to isobutanol, a drop-in fuel with numerous advantages over other clean burning gasoline blendstocks. Dominion will be the exclusive partner to construct, own, operate, and maintain the production facilities in the Marcellus and Utica Shale Basins via potential long-term services agreements with IEP;

• Strengthened the Company’s immuno-oncology efforts via a collaboration with the biopharmaceutical business of Merck KGaA, Darmstadt, Germany including advancement of the first two chimeric antigen receptor (CAR) T-cell targets of interest, a Cooperative Research and Development Agreement with the National Cancer Institute, as well as through an exclusive licensing agreement with The University of Texas MD Anderson Cancer Center for pursuit of non-viral adoptive cellular therapies in conjunction with ZIOPHARM Oncology;

• Declared a special stock dividend of 17,830,305 shares of ZIOPHARM common stock owned by Intrexon to its shareholders;

• In the food sector where Intrexon’s strategy centers on responsibly harnessing the power of biology and technology to produce nutritious food that is more appetizing and convenient to consumers, two of its subsidiaries received landmark approvals in 2015 – the AquAdvantage Salmon by the U.S. Food and Drug Administration and Arctic Apples by the U.S. Department of Agriculture Animal and Plant Health Inspection Service and Health Canada; and

• Completed the acquisitions of Oxitec Ltd., a company that has pioneered a targeted and innovative approach to control mosquitoes that spread disease and insect pests that damage crops; Okanagan Specialty Fruits, the pioneering agricultural company behind the Arctic apple; and ActoGeniX, the biopharmaceutical company forging a new frontier in cellular therapeutics.
Fourth Quarter Financial Highlights:

• Total revenues of $41.5 million, an increase of 34% over the fourth quarter of 2014;

• Net loss of $32.7 million attributable to Intrexon, or $(0.28) per basic share, including non-cash charges of $19.4 million;

• Adjusted EBITDA of $9.4 million, or $0.08 per basic share;

• Cash consideration received for reimbursement of research and development services covered 64% of cash operating expenses (exclusive of operating expenses of consolidated subsidiaries); and

• Cash, cash equivalents, and short-term and long-term investments totaled $343.8 million, and the value of equity securities totaled $83.7 million at December 31, 2015.
Full Year Financial Highlights:

• Total revenues of $173.6 million, an increase of 141% over the year ended December 31, 2014;

• Net loss of $84.5 million attributable to Intrexon, or $(0.76) per basic share, including non-cash charges of $61.6 million;

• Adjusted EBITDA of $53.4 million, or $0.48 per basic share;

• Distributed as a dividend to Intrexon’s shareholders equity securities having a market value of $172.4 million at the time of distribution;

• Total consideration received for technology access fees and reimbursement of research and development services covered 167% of cash operating expenses (exclusive of operating expenses of consolidated subsidiaries); and

• Total consideration received for technology access fees, reimbursement of research and development services and products and services revenues covered 133% of consolidated cash operating expenses.

"Our essential strategy is accomplished when our team executes well, and in 2015 our team executed beautifully its mission to advance its leadership position in the rapidly emergent field of engineered biology," commented Randal J. Kirk, Chairman and Chief Executive Officer of Intrexon. "Our model has as its chief objective the attainment of a large portfolio of significant economic interests in products across multiple industries while maintaining the discipline of living within our means, those being provided chiefly from our partners with whom we are working to develop world-changing products."

Mr. Kirk concluded, "We are well positioned and excited as we look forward to the remainder of 2016, a year in which we intend to make progress along three principal axes. First, extending the trend that we showed in 2015 over 2014, we expect to continue our financial growth trajectory. Second, we expect to deliver to the world in this year several examples of projects that were initiated within our company, and these may include specimens from our methanotrophic bioconversion platform and clinical stage therapeutic candidates. Finally, we expect to partner some of our mature stage assets with companies that add real value toward their maximized commercial realizations."

Fourth Quarter 2015 Financial Results Compared to Prior Year Period
Total revenues were $41.5 million for the quarter ended December 31, 2015 compared to $31.1 million for the quarter ended December 31, 2014, an increase of $10.4 million, or 34%. Collaboration and licensing revenues increased $8.2 million over the three months ended December 31, 2014 due to (i) the recognition of deferred revenue for upfront payments received from the Company’s license and collaboration agreement with the biopharmaceutical business of Merck KGaA, which became effective in May 2015, and from other collaborations signed by Intrexon in 2015 and (ii) increased research and development services for both new collaborations and for the expansion or addition of new programs with previously existing collaborators, including ZIOPHARM Oncology, Inc. (ZIOPHARM) and Intrexon’s joint venture with Intrexon Energy Partners, LLC (Intrexon Energy Partners). Product revenues were $9.2 million for the three months ended December 31, 2015 compared to $7.4 million for the three months ended December 31, 2014, an increase of $1.8 million, or 25%. The increase in product revenues during the three months ended December 31, 2015 primarily relates to an increase in the quantity of pregnant cows and weaned calves sold due to higher customer demand. Gross margin on product revenues declined for the period due to a decline in the average sales price and gross margins for sales of livestock previously used in production. Service revenues and gross margins remained consistent period over period.

Total operating expenses were $75.9 million for the quarter ended December 31, 2015 compared to $50.1 million for the quarter ended December 31, 2014, an increase of $25.8 million, or 51%. Research and development expenses were $26.2 million for the three months ended December 31, 2015 compared to $17.6 million for the three months ended December 31, 2014, an increase of $8.6 million, or 49 percent. Salaries, benefits and other personnel costs increased $3.6 million due to (i) an increase in research and development employees necessary to support new and expanded collaborations, (ii) the additional costs of stock options and performance-based bonus awards for all research and development employees and (iii) the cost of new employees assumed as a result of Intrexon’s various acquisitions. Lab supplies and contract research organizations expenses increased $2.7 million as a result of (i) the progression of programs into the preclinical phase with certain of Intrexon’s collaborators; (ii) the increased level of research and development services provided to the Company’s collaborators; and (iii) costs incurred as a result of the Company’s various acquisitions. Depreciation and amortization increased $1.8 million primarily from the acquisition of property and equipment and intangible assets assumed as a result of Intrexon’s various acquisitions. Selling, general and administrative expenses were $34.7 million for the three months ended December 31, 2015 compared to $19.8 million for the three months ended December 31, 2014, an increase of $14.9 million, or 75 percent. Salaries, benefits and other personnel costs increased $10.5 million due to (i) an increase in selling, general and administrative employees necessary to support Intrexon’s expanding operations, (ii) the additional costs of stock options and performance-based bonus awards, including those paid under the 2015 annual executive bonus plan, for all selling, general and administrative employees; (iii) the cost of new employees assumed as a result of the Company’s various acquisitions. Legal and professional expenses increased $2.9 million primarily due to (i) incremental legal and professional fees related to the ongoing operations of acquired subsidiaries; and (ii) increased professional and business development expenses related to certain collaborations entered into during the three months ended December 31, 2015.
Total other income, net, was $3.7 million for the quarter ended December 31, 2015 compared to $38.5 million for the quarter ended December 31, 2014, a decrease of $34.8 million, or 90%. This decrease was primarily related to the changes in the value of Intrexon’s securities portfolio.

Full Year 2015 Financial Results Compared to Prior Year Period
Total revenues were $173.6 million for the year ended December 31, 2015 compared to $71.9 million for the year ended December 31, 2014, an increase of $101.7 million, or 141%. For the year ended December 31, 2015, total product revenues of $41.9 million were derived primarily from the sale of pregnant cows, live calves and livestock used in production and service revenues of $42.9 million were derived primarily from the provision of in vitro fertilization and embryo transfer services. For the year ended December 31, 2014, product and service revenues were $11.5 million and $14.8 million, respectively. The increases relate primarily to the inclusion of a full year of results for Trans Ova in 2015 versus approximately five months of results for 2014. Collaboration and licensing revenues increased $42.6 million over the year ended December 31, 2014 due to (i) the recognition of deferred revenue for upfront payments received from Intrexon’s license and collaboration agreement with the biopharmaceutical business of Merck KGaA, which became effective in May 2015, and from other collaborations signed by the Company in 2015; (ii) increased research and development services performed for both for new collaborations and for the expansion or addition of new programs with previously existing collaborators, including primarily ZIOPHARM, Fibrocell Science, Inc., Genopaver, LLC, and Intrexon Energy Partners and (iii) the recognition of previously deferred revenue related to collaboration agreements for which Intrexon satisfied all of its obligations or which were terminated during in 2015.

Total operating expenses were $320.5 million for the year ended December 31, 2015 compared to $141.9 million for the year ended December 31, 2014, an increase of $178.6 million, or 126%. Research and development expenses were $147.5 million for the year ended December 31, 2015 compared to $59.0 million for the year ended December 31, 2014, an increase of $88.5 million, or 150%. In January 2015, Intrexon paid $59.6 million in common stock for an exclusive license to certain technologies owned by the University of Texas MD Anderson Cancer Center, or MD Anderson. Salaries, benefits and other personnel costs increased $12.5 million due to (i) an increase in research and development employees necessary to support new and expanded collaborations, (ii) the additional costs of stock options and performance-based bonus awards for all research and development employees; and (iii) the cost of new employees assumed as a result of Intrexon’s various acquisitions. Lab supplies and contract research organization expenses increased $8.2 million as a result of (i) the progression of programs into the preclinical phase with certain of Intrexon’s collaborators; (ii) the increased level of research and development services provided to the Company’s collaborators; and (iii) costs incurred as a result of the Company’s various acquisitions. Depreciation and amortization increased $4.0 million primarily from the acquisition of property and equipment and intangible assets assumed as a result of Intrexon’s various acquisitions. Selling, general and administrative expenses were $109.1 million for the year ended December 31, 2015 compared to $63.6 million for the year ended December 31, 2014, an increase of $45.5 million, or 72%. Salaries, benefits and other personnel costs increased $28.0 million due to (i) the inclusion of selling, general and administrative employees of Trans Ova for a full year in 2015 compared to approximately five months in 2014; (ii) an increase in selling, general and administrative employees necessary to support Intrexon’s expanding operations, (iii) the additional costs of stock options and performance-based bonus awards, including those paid under the 2015 annual executive bonus plan, for all selling, general and administrative employees; and (iv) the cost of new employees assumed as a result of the Company’s various acquisitions. Legal and professional expenses increased $7.0 million primarily due to costs associated with Intrexon’s various 2015 acquisitions, the license agreement with MD Anderson, a full year of legal and professional costs for Trans Ova, Intrexon’s 2015 public offerings, and other business development activity. Other selling, general and administrative expenses, including rent, utilities and depreciation and amortization, have increased in 2015 as a result of (i) the expansion of Intrexon’s operations, including through the Company’s various acquisitions, and (ii) a full year of Trans Ova expenses compared to approximately five months in 2014. Total operating expenses for the year ended December 31, 2015 also include $63.9 million of products and services costs which primarily consist of employee compensation costs, livestock, feed, drug supplies, recipient costs and facility charges related to the production of such products and services; this amount was $19.3 million for the year ended December 31, 2014. The increase relates primarily the inclusion of a full year of results for Trans Ova in 2015 versus five months of results for 2014.

Total other income, net, was $68.8 million for the year ended December 31, 2015 compared to total other expense, net, of $10.5 million for the year ended December 31, 2014, an increase of $79.3 million. This increase was primarily related to the $81.4 million realized gain recognized upon the special stock dividend of all of Intrexon’s shares of ZIOPHARM to Intrexon’s shareholders in June 2015.

On February 29, 2016 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and reported financial results for the fourth quarter and full year ending December 31, 2015 (Filing, Q4/Annual, PTC Therapeutics, 2015, FEB 29, 2016, View Source [SID:1234509287]).

"We are shocked and disappointed to have received a Refuse to File (RTF) letter from the FDA regarding our NDA for Translarna, and we are engaging in dialogue with the FDA to determine a path forward," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "Despite this recent setback, we achieved a number of key milestones in 2015 as we continue to build PTC into a global, commercial biotechnology company. Translarna had a landmark first year launch in nonsense mutation Duchenne muscular dystrophy and we are currently under regulatory review in the EU to remove the condition to the existing marketing authorization as well as expand our label to include nonsense mutation cystic fibrosis. Our Phase 3 ACT CF clinical trial is fully enrolled and our clinical stage SMA and cancer stem cell assets continue to progress. I am proud of what we accomplished in 2015. Resilience has been one of our key values since I founded PTC in 1998 and our team continues to work diligently to bring Translarna to patients globally."

Key Fourth Quarter, Full Year 2015 and other Corporate Highlights:

· Refuse to File letter received from the FDA regarding Translarna for nonsense mutation Duchenne muscular dystrophy (nmDMD). The letter from the U.S. Food and Drug Administration (FDA) received on February 22, 2016 stated that the new drug application (NDA) for Translarna was not sufficiently complete to permit a substantive review. Specifically, PTC was notified in the letter that, in the view of the FDA, both the Phase 2b and ACT DMD trials were negative and do not provide substantial evidence of effectiveness. The FDA also characterized certain of the company’s adjustments to the ACT DMD study as post hoc and therefore not supportive of effectiveness. In addition, the FDA noted that the NDA did not contain adequate information regarding the abuse potential of Translarna, a requirement for new molecules that cross the blood-brain barrier. PTC is engaging in dialogue with the FDA to discuss and clarify the matters set forth in the letter and to determine the best path forward.

· Successful first year Translarna launch with 2015 revenues of $33.7M. PTC has established a strong global commercial footprint launching the first approved therapy in Duchenne muscular dystrophy (DMD), with sales generated in 23 countries including most recently Argentina, the Czech Republic, Hungary, Portugal and Singapore. PTC is targeting to expand access to Translarna to over 35 countries by the end of 2016. Market access discussions regarding funding on a country-by-country basis are ongoing. In the UK, PTC has had constructive discussions with the National Health Services (NHS) England regarding a managed access agreement for Translarna with a decision from the National Institute for Health and Care Excellence (NICE) expected in the coming months. In Germany, PTC has had multiple discussions with the German Federal Association of the Statutory Health Insurances (GKV-SV) over the last several months to come to agreement on pricing and reimbursement. Recently, these discussions transitioned into an arbitration process, which did not lead to an acceptable agreement. As a result, PTC expects to delist Translarna from the German pharmacy ordering system. Under these circumstances, patients and healthcare professionals may be able to

access Translarna through a reimbursed importation pathway possible under German law, thus minimizing any access issues for existing and new German patients.

· Actively pursuing regulatory approvals for Translarna in DMD globally. In early January 2016, PTC submitted the ACT DMD Phase 3 results to the European Medicines Agency (EMA) in fulfillment of the principal condition of the EMA marketing authorization for the treatment of nmDMD in ambulatory patients aged five and over. The submission to the EMA, called a type II variation, seeks to remove the condition to the existing marketing authorization. PTC anticipates the Committee for Medicinal Products for Human Use (CHMP) will issue its recommendation regarding this request in mid-2016. Translarna also received regulatory approvals in both Israel and South Korea in 2015. In September 2015, Health Canada initiated an expedited review of Translarna for potential approval for nmDMD in the first half of 2016.

· ACT CF Phase 3 clinical trial on track for completion by year-end 2016 with top-line results expected early 2017. In November 2015, PTC announced that it had completed enrollment for ACT CF, the company’s second Phase 3 clinical trial of Translarna for patients with nonsense mutation cystic fibrosis (nmCF). ACT CF is a 48-week placebo-controlled Phase 3 clinical trial designed to evaluate the effect of Translarna in patients six years of age or older with nmCF not receiving chronic inhaled aminoglycosides. During the third quarter, PTC submitted a variation to its marketing authorization requesting EMA approval of Translarna for the treatment of nmCF based on the company’s previous Phase 3 study. PTC anticipates the CHMP will issue its recommendation regarding this submission in mid-2016.

· As part of 10 by ‘20 strategy, four additional indications in development for Translarna. Given its mechanism of action, Translarna has the potential to address numerous genetic disorders caused by a nonsense mutation to address significant unmet need across a spectrum of many rare diseases. In addition to its advanced DMD and CF programs, PTC is pursuing indications in mucopolysaccharidosis type I (MPS I), aniridia, and two genetically defined epilepsy disorders, Dravet syndrome and CDKL5. PTC’s goal is to investigate Translarna’s activity in a minimum of ten indications beyond DMD

and CF by 2020 in order to deliver on its commitment to patients and maximize the potential of Translarna as both a product and a pipeline.

· Internally developed pipeline continues to progress. In January 2016, clinical development of the spinal muscular atrophy (SMA) program, a collaboration with Roche and the SMA Foundation, resumed with a second compound, RG7916, beginning a Phase 1 study in healthy volunteers. In addition, PTC’s cancer stem cell program in oncology continues to advance with Phase 1 data expected in 2016. PTC’s discovery group is focused on the advancement of novel programs for rare and neglected disorders including next generation nonsense read-through, Huntington’s disease and familial dysautonomia.

· Maintained strong balance sheet with approximately $339 million in cash and cash equivalents. PTC completed a successful $150 million offering of 3.00% convertible senior notes due 2022 in August 2015, raising net proceeds of approximately $145 million. PTC finished 2015 with approximately $339 million in cash and cash equivalents.

Upcoming Events:

PTC will participate in the following upcoming conferences:

· Cowen and Company’s 36th Annual Health Care Conference on March 8 at 11:20 a.m. (ET) in Boston, MA
· Barclay’s Global Healthcare Conference on March 15 at 1:35 p.m. (ET) in Miami, FL
· Deutsche Bank’s 41st Annual Healthcare Conference on May 4-5 in Boston, MA

The presentations will be webcast live on the Events and Presentations page under the investor relations section of PTC’s website at www.ptcbio.com and will be archived for two weeks following the presentation. PTC’s current investor presentation is available at the same website location.

Fourth Quarter and Full Year 2015 Financial Highlights:

· Translarna net product sales were $12.7 million for the fourth quarter of 2015, representing 30% sequential growth versus $9.8 million in the third quarter of 2015. For the full year 2015, Translarna generated $33.7 million in net product sales compared to $0.7 million in the prior year.

· Total revenues for the fourth quarter of 2015 were $12.7 million compared to $12.7 million in the same period of 2014. Total revenues for 2015 were $36.8 million compared to $25.2 million for the same period of 2014. The change in total revenue was a result of the expanded commercial launch of Translarna during 2015, which received marketing authorization from the EMA in August 2014, offset by lower grant revenue.

· Non-GAAP R&D expenses were $31.4 million for the fourth quarter of 2015, excluding $3.7 million in non-cash, stock-based compensation expense, compared to $23.7 million for the fourth quarter of 2014, excluding $3.2 million in non-cash, stock-based compensation expense. GAAP R&D expenses were $35.0 million for the fourth quarter of 2015 compared to $26.9 million for the fourth quarter of 2014. For the full year 2015, non-GAAP R&D expenses were $105.7 million, excluding $16.1 million in non-cash, stock-based compensation expense, compared to $70.1 million for 2014, excluding $9.7 million in non-cash, stock-based compensation expense. For the full year 2015, GAAP R&D expenses were $121.8 million compared to $79.8 million in the prior year period. The increase in R&D expense for the fourth quarter and year ended December 31, 2015, as compared to the prior year periods was primarily due to expansion of our clinical development activities including late-stage studies and extension programs in both Duchenne muscular dystrophy and cystic fibrosis.

· Non-GAAP SG&A expenses were $21.7 million for the fourth quarter of 2015, excluding $4.2 million in non-cash, stock-based compensation expense, compared to $14.5 million for the fourth quarter of 2014, excluding $3.5 million in non-cash, stock-based compensation expense. GAAP SG&A expenses were $25.9 million for the fourth quarter of 2015 compared to $18.0 million for the fourth quarter of 2014. For the full year 2015,

non-GAAP SG&A expenses were $64.2 million, excluding $17.8 million in non-cash, stock-based compensation expense, compared to $35.2 million for 2014, excluding $9.6 million in non-cash, stock-based compensation expense. GAAP full-year 2015 SG&A expenses were $82.1 million compared to $44.8 million in 2014. The increase in SG&A expense for the fourth quarter and year ended December 31, 2015, as compared to the prior year periods primarily resulted from additional costs associated with commercial activities in support of the launch of Translarna for DMD.

· Net loss for the fourth quarter of 2015 was $50.9 million compared to a net loss of $27.3 million for the same period in 2014. Net loss for the full year 2015 was $170.4 million compared to $93.8 million for the same period in 2014.

· Cash, cash equivalents, and marketable securities totaled approximately $339 million at December 31, 2015 compared to approximately $315 million at December 31, 2014. This increase includes net proceeds of approximately $145 million from a $150 million convertible debt offering completed in the third quarter of 2015.

· Shares issued and outstanding as of December 31, 2015 were 34.3 million, which includes 0.3 million shares of unvested restricted stock.

2016 Guidance:

· Total ex-U.S. Translarna nmDMD revenues for 2016 are anticipated to be between $65 and $85 million. This guidance assumes current exchange rates and the continued commercial expansion for Translarna in nmDMD outside of the U.S.

· Operating expenses for 2016 are currently under review as a result of the Refuse to File letter recently received from the FDA.

Non-GAAP Financial Measures

In this press release, PTC’s financial results and financial guidance are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, non-GAAP financial results exclude stock-based compensation expense. These results are provided as a complement to results reported in GAAP because management believes these non-GAAP financial measures are the best indication of the company’s business.

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