On March 16, 2017 Fortress Biotech, Inc. (NASDAQ: FBIO) ("Fortress"), a biopharmaceutical company dedicated to acquiring, developing and commercializing novel pharmaceutical and biotechnology products, reported financial results and recent corporate highlights for the fourth quarter and full year ended December 31, 2016 (Press release, Fortress Biotech, MAR 15, 2017, View Source [SID1234518161]).

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Dr. Lindsay A. Rosenwald, Fortress’ Chairman, President and Chief Executive Officer, said, "Fortress had another productive year in 2016 and early 2017, with the launch of three additional Fortress Company subsidiaries, Cellvation, Caelum Biosciences, and Cyprium Therapeutics, which broaden our pipeline in cellular therapeutics and rare disease. In addition, we completed a tender offer purchasing a majority of National Holdings Corporation, a full service investment banking and advisory firm, which has a significant presence in biotechnology and the life sciences. National presents multiple opportunities for synergies with our core biotech drug development business. At the same time, our established Fortress Companies have continued to achieve significant milestones, including the publication of a case study in The New England Journal of Medicine in which Mustang Bio’s MB-101 CAR T therapy achieved an unprecedented complete response in a glioblastoma patient. In 2017, we plan to continue to work with our Fortress Companies to advance their pipelines in and toward clinical development, and explore opportunities to strengthen our subsidiary company portfolio."

Financial Results:
As of December 31, 2016, Fortress’ consolidated cash and cash equivalents totaled $88.3 million, compared to $82.5 million as of September 30, 2016, and $98.2 million as of December 31, 2015, an increase of $5.8 million for the fourth quarter and a decrease of $9.9 million year-to-date. These totals exclude restricted cash of $15.9 million as of December 31, 2016 and September 30, 2016, and restricted cash of $14.6 million as of December 31, 2015.

Revenue totaled $16.5 million as of December 31, 2016, compared to $0.9 million as of December 31, 2015. Total revenue as of December 31, 2016 includes $6.2 million of Fortress revenue and $10.3 million of revenue from National Holdings Corporation (includes revenue from the date of the close of the acquisition, September 9, 2016 through September 30, 2016).

Research and development expenses were $29.6 million for the year ended December 31, 2016, of which $22.6 million was related to Fortress Companies. This compares to $18.4 million for 2015, of which $8.4 million was related to Fortress Companies. Noncash stock-based compensation expenses included in research and development were $4.7 million for the year ended December 31, 2016, and $5.8 million for 2015.

Research and development expenses from license acquisitions totaled $5.5 million for the year ended December 31, 2016, compared to $11.4 million for the year ended December 31, 2015.

General and administrative expenses were $34.0 million for the year ended December 31, 2016, of which $15.4 million was related to Fortress Companies. This compares to $21.6 million for 2015, of which $6.7 million was related to Fortress Companies. Noncash stock-based compensation expenses included in general and administrative expenses were $7.4 million for the year ended December 31, 2016, and $8.5 million for 2015.

Net loss attributable to common stockholders was $55.1 million, or $1.38 per share, for the year ended December 31, 2016, compared to a net loss attributable to common stockholders of $48.4 million, or $1.24 per share, for 2015.

Recent Fortress Biotech and Fortress Company Highlights:

Fortress Biotech, Inc.
Phase 1/2 data demonstrating CNDO-109-activated allogeneic natural-killer cells are safe and well‐ tolerated, and potentially capable of extending complete remissions in high‐risk acute myeloid leukemia patients were presented in May at the Innate Killer Summit 2016.

In September 2016, Fortress, through its subsidiary FBIO Acquisition, Inc., purchased approximately 56 percent of National Holdings Corporation (NASDAQ: NHLD) common stock at a purchase price of $3.25 per share in cash, for an aggregate purchase price of approximately $22.9 million.

Fortress recently launched three new Fortress Companies: Cellvation, to develop cellular therapeutics for the treatment of traumatic brain injury, Caelum Biosciences, to develop therapies for amyloid light chain ("AL") amyloidosis and Cyprium Therapeutics, to develop novel therapies for the treatment of Menkes disease and related copper metabolism disorders.

Avenue Therapeutics, Inc.

In 2016, Avenue completed an end of Phase 2 meeting with the U.S. Food and Drug Administration regarding its lead candidate, intravenous (IV) tramadol, for the management of post-operative pain. Based on the outcome, Avenue anticipates its Phase 3 study will consist of three trials: an efficacy and safety study in an orthopedic model, an efficacy and safety study in a soft tissue model and an openlabel safety study. Initiation of the Phase 3 study is planned for 2017.

In December 2016, Avenue received Notices of Allowance from the U.S. Patent and Trademark Office for two continuation patent applications covering methods of administration for IV Tramadol; issuance of both patents occurred in February 2017.

Caelum Biosciences, Inc.

In January 2017, Caelum entered into an agreement with Columbia University ("Columbia") to secure exclusive worldwide license rights to CAEL-101, a chimeric fibril-reactive monoclonal antibody.

Interim data from the ongoing Phase 1a/1b study of CAEL-101 were presented by Columbia in December 2016 at the American Society of Hematology (ASH) (Free ASH Whitepaper)’s 58th Annual Meeting. These data demonstrate CAEL-101 is safe and well-tolerated, and 67 percent of patients with measurable disease burden showed organ response. Full Phase 1a/1b data are expected mid-2017.

Cellvation, Inc.

On October 31, 2016, Cellvation secured exclusive worldwide rights to three programs for traumatic brain injury (TBI) from The University of Texas Health Science Center at Houston: two Phase 2 programs evaluating CEVA101 cell therapy in adult and pediatric TBI patients, and CEVA-D, a next-generation bioreactor that enhances the anti-inflammatory potency of bone marrow-derived cells without genetic manipulation. Data from the Phase 2 CEVA101 studies are expected in 2019.

Phase 1 data demonstrating CEVA101 is safe and effective in modulating the neuroinflammatory response and reducing secondary injury in adults with TBI were published online in November 2016 in STEM CELLS.

Checkpoint Therapeutics, Inc.

In January 2016, Checkpoint announced it signed a license agreement with Teva Pharmaceutical Industries Ltd. for the exclusive worldwide rights to develop and commercialize CK-102, an oral poly (ADP-ribose) polymerase (PARP) inhibitor in early clinical development for solid tumors. A Phase 1b study is planned to commence in the next 12 months.

In May 2016, Checkpoint entered into an exclusive worldwide license agreement with Jubilant Biosys Ltd. to develop and commercialize novel compounds that inhibit the BET protein BRD4. In connection therewith, Checkpoint sublicensed development and commercialization rights to the compounds in hematological malignancies to TG Therapeutics, Inc. Checkpoint retains development and commercialization rights in solid tumors. Checkpoint plans to submit an Investigational New Drug ("IND") application for a Phase 1 study in the second half of 2017.

In September 2016, following approval of the IND, the first patient was dosed in the Phase 1 doseescalation portion of a Phase 1/2 study of epidermal growth-factor receptor ("EGFR") inhibitor CK-101. Checkpoint expects to initiate the Phase 2 safety and efficacy portion of the study in patients with EGFR T790M mutation‐positive non‐small cell lung cancer in the second half of 2017.

Cyprium Therapeutics, Inc.

In March 2017, Cyprium entered into a Cooperative Research and Development Agreement (CRADA) with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), part of the NIH, to advance the clinical development of Phase 3 candidate CUTX-101, a Copper Histidinate injection, for the treatment of Menkes disease. Also effective in March 2017, Cyprium and the NICHD entered into a worldwide, exclusive license agreement to develop and commercialize adenoassociated virus (AAV)-based gene therapy, called AAV-ATP7A, to deliver working copies of the copper transporter that is defective in Menkes patients and to be used in combination with CUTX-101.

Helocyte, Inc.
In February and March 2016, respectively, Helocyte entered into investigator-initiated clinical research support agreements with City of Hope National Medical Center to support two Phase 2 studies of Helocyte’s immunotherapies Triplex and PepVax for cytomegalovirus control in allogeneic hematopoietic stem-cell transplant recipients ("HSCT"). The ongoing Phase 2 studies are also supported by grants from the National Cancer Institute.

From June to November 2016, Helocyte closed on sales of convertible promissory notes raising aggregate gross proceeds of approximately $4.4 million.

Phase 1 data demonstrating Triplex is safe, well-tolerated and highly immunogenic at multiple dose levels in healthy volunteers were published in December 2016 online in Blood. These data supported the initiation of the Phase 2 study of Triplex in HSCT patients, which is expected to be fully enrolled by the second half of 2017.
Journey Medical Corporation (JMC)

In January 2016, JMC entered into two licensing agreements with third parties for the distribution of the first two products in the company’s dermatology franchise: Luxamend, a prescription wound cream, and Ceracade, an emollient for the treatment of various types of dermatitis. Sales commenced for Luxamend and Ceracade in April 2016 and June 2016, respectively.

U.S. sales commenced in October 2016 for JMC’s Targadox brand of oral antibiotic, indicated for the treatment of severe acne.

Mustang Bio, Inc.

A case study demonstrating Mustang’s MB-101 (IL13Rα2-specific CAR T cells) achieved a complete response in a glioblastoma patient enrolled in the Phase 1 study was published in the December 29 edition of The New England Journal of Medicine. Additional preclinical and Phase 1 data on MB-101 were presented at the American Society of Gene and Cell Therapy 19th Annual Meeting in May 2016, and the 21st Annual Meeting and Education Day of the Society for Neuro-Oncology in November 2016.

From October 2016 to January 2017, Mustang closed on a total of approximately $94.5 million in a private placement financing, prior to fees and expenses.

On March 15, 2017 Aeterna Zentaris Inc. (NASDAQ, TSX: AEZS) (the "Company"), a specialty biopharmaceutical company engaged in developing and commercializing novel treatments in oncology and endocrinology, reported financial and operating results for the fourth quarter and year ended December 31, 2016 (Press release, AEterna Zentaris, MAR 15, 2017, View Source [SID1234518148]).

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Commenting on recent key developments, David A. Dodd, President and Chief Executive Officer of the Company, stated, "During the fourth quarter and the first few weeks of 2017, we made substantial progress with our development programs. On January 30, 2017 we announced the conclusion of the clinical phase of our development of Zoptrex. The following day we had a successful pre-NDA meeting with the FDA. We anticipate reporting top-line results in April. This is a very exciting and anxious time for us, as we approach the culmination of highly dedicated and successful work by many throughout our Company. I would like to thank our R&D team for their hard work in bringing Zoptrex to this point."

Mr. Dodd continued his commentary with an update on the development of Macrilen. "On January 4, 2017, we reported the top-line results from our confirmatory Phase 3 study of Macrilen for the evaluation of adult growth hormone deficiency ("AGHD"). We reported that the top-line results indicated that macimorelin did not meet one of the pre-defined criteria required to demonstrate equivalence to the Insulin Tolerance Test ("ITT") as a means of diagnosing AGHD. Following this announcement, we conducted a thorough evaluation of the study data, including external statistical expertise and independent review by leading endocrinologists in both the U.S. and Europe. We were highly encouraged by the results and input received from these experts. As we announced on February 13, 2017, we concluded that Macrilen demonstrated performance supportive of achieving registration with the U.S. Food and Drug Administration, despite its failure to meet one of the pre-defined equivalence criteria. We explained the reasons for our conclusion in our February 13 release. Briefly, we concluded that Macrilen demonstrated more consistent and reproducible results than the ITT. Moreover, Macrilen stimulated the pituitary gland more powerfully than the ITT and demonstrated good specificity and sensitivity in this study, thus reproducing the results of our previous study. We demonstrated that Macrilen achieves a high degree of correlation with the ITT, which could be further optimized when a higher cut-off point, such as the ITT cut-off point, is used for the Macrilen test. We believe that such an increased cut-off point would be justified by the more powerful stimulation of Macrilen as compared to the ITT. We are scheduled to meet with the FDA at the end of Q1 to discuss our rationale for proceeding with Macrilen."

Fourth Quarter and Full Year Financial Highlights
Revenues
Sales commission and other were $94,000 and $414,000 for the three and twelve months ended December 31, 2016, respectively, and $41,000 and $297,000, for the same periods in 2015, respectively. The quarter-over-quarter and year-over-year increases were attributable to our sales team exceeding pre-established unit sales baseline thresholds under our co-promotion agreements to sell Saizen and to our promotion of APIFINY, which did not begin until the first quarter of 2016. In the corresponding periods of 2015, sales commission and other revenues were mainly related to EstroGel, which we no longer promote.
License fees were $210,000 and $497,000 for the three and twelve months ended December 31, 2016, respectively, as compared to $61,000 and $248,000 for the same periods in 2015. The increase is explained by the out-licensing agreements that we entered into in 2016 for Zoptrex with respect to certain territories outside our core areas of interest.

Research and Development ("R&D") costs
R&D costs were $4.6 million and $16.5 million for the three and twelve months ended December 31, 2016, respectively, compared to $4.2 million and $17.2 million for the same periods in 2015. The increase in our R&D costs for the three months ended December 31, 2016, as compared to the same period in 2015, was mainly attributable to higher comparative third-party costs in connection with the confirmatory Phase 3 clinical trial of Macrilen, which was initiated late in 2015 with the enrollment of the first patient in the fourth quarter of 2015. Patient recruitment was completed in the fourth quarter of 2016. The decrease in our R&D costs for the twelve months ended December 31, 2016, as compared to the same period in 2015, was mainly attributable to the realization of cost savings in connection with our ongoing efforts to streamline our R&D activities and to increase our commercial operations and flexibility by reducing our R&D staff, which was started in 2014.
General and Administrative ("G&A") Expenses

G&A expenses were $1.8 million and $7.1 million for the three and twelve months ended December 31, 2016, respectively, as compared to $4.0 million and $11.3 million for the same periods in 2015. The decrease in our G&A expenses for the three months and twelve months ended December 31, 2016, as compared to the same periods in 2015, is mainly due to the recording in the fourth quarter of 2015 of a provision related to the restructuring of our finance and accounting function and the closure of our office in Quebec City, as well as the realization of cost savings in connection with the restructuring. The comparative decrease for the twelve-month period is also explained by certain transaction costs allocated to warrants in connection with the completion of share issuances in March and December 2015.

Selling Expenses
Selling expenses were $1.5 million and $6.7 million for the three and twelve months ended December 31, 2016, respectively, as compared to $1.8 million and $6.9 million for the same periods in 2015. Selling expenses for the three and twelve months ended December 31, 2016 and 2015 represent mainly the costs of our contracted sales force related to our co-promotion activities as well as our internal sales management team. Selling expenses remained relatively stable during 2016.

Net Finance (Costs) Income
Net finance (costs) income were $(622,000) and $4.5 million for the three and twelve months ended December 31, 2016, as compared to $(185,000) and $(15.3) million, for the same periods in 2015. The increases in finance income or decreases in finance costs were mainly attributable to the change in fair value recorded in connection with our warrant liability. Such change in fair value results from the periodic "mark-to-market" revaluation, via the application of option pricing models, of outstanding share purchase warrants. During 2016, the "mark-to-market" warrant valuation was impacted by the expiration of the remaining Series B Warrants. During 2015, the change in assumptions that were applied to determine the fair value of the alternate cashless feature included in the Series B Warrants significantly impacted the "mark-to-market" valuation. Furthermore, the closing price of our common shares, which, on the NASDAQ, fluctuated from $3.25 to $4.94 during the three-month period and $2.67 to $4.94 during the twelve-month period ended December 31, 2016, respectively, compared to $4.00 to $11.43 and $4.00 to $84.20 during the same periods in 2015, also had a direct impact on the change in fair value of warrant liability. In addition, with specific reference to 2015, finance costs were also impacted by the warrant exercise inducement fee paid to certain holders of the Series B Warrants.

Net Loss
Net loss for the three and twelve months ended December 31, 2016 was $(8.2) million and $(25.0) million, or $(0.71) and $(2.41) per basic and diluted share, as compared to a net loss of $(10.0) million and $(50.1) million, or $(1.46) and $(18.14) per basic and diluted share, for the same periods in 2015. The decrease in net loss for the three months ended December 31, 2016, as compared to the same period in 2015, is due largely to lower G&A expenses, as presented above. The decrease in net loss for the twelve months ended December 31, 2016, as compared to the same period in 2015, is due largely to lower operating expenses and higher comparative net finance income, as presented above.

Liquidity
Cash and cash equivalents were $22.0 million as at December 31, 2016, as compared to $41.5 million as at December 31, 2015. The decrease in cash and cash equivalents as at December 31, 2016, as compared to December 31, 2015, is mainly due to the net cash used in operating activities. The decrease was partially offset by the net proceeds generated by the sale and issuance of common shares and warrants during 2016.

On March 15, 2017 Prima BioMed Ltd (ASX: PRR; NASDAQ: PBMD) ("Prima" or the "Company") reported that the second cohort comprising six patients has now been fully recruited for its TACTI-mel (Two ACTive Immunotherapeutics in melanoma) clinical trial being conducted in Australia (Press release, Prima Biomed, MAR 15, 2017, View Source [SID1234518142]). Patients with unresectable or metastatic melanoma that have had a suboptimal response to KEYTRUDA were dosed with the higher 6 mg dose of IMP321 in combination with KEYTRUDA.

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Prima’s Chief Medical & Scientific Officer, Dr Frédéric Triebel, will be presenting the TACTI-mel clinical trial at the Immune Checkpoint Inhibitors conference at the Sheraton Hotel Boston, Massachusetts, held on March 15-16, 2017.
The presentation will be delivered at 1:30pm on Thursday 16 March, 2017 EDT.
A copy of these presentation slides will be made available on the Prima BioMed website.
Further information on the conference can be found at http://immune-checkpoint.com/about/about

About IMP321
IMP321, a first-in-class Antigen Presenting Cell (APC) activator based on the immune checkpoint LAG-3, represents one of the first proposed active immunotherapy drugs in which the patient’s own immune system is harnessed to respond to tumour antigenic debris created by chemotherapy. As an APC activator IMP321 boosts the network of dendritic cells in the body that can respond to tumour antigens for a better anti-tumour CD8 T cell response.

On March 15, 2017 GTx, Inc. (Nasdaq: GTXI) reported financial results for the fourth quarter and year ended December 31, 2016, and highlighted recent accomplishments and upcoming milestones (Press release, GTx, MAR 15, 2017, View Source [SID1234518139]). The Company has two ongoing clinical trials of enobosarm (GTx-024) in women with advanced breast cancer and one ongoing trial with enobosarm as a potential treatment for stress urinary incontinence (SUI) in postmenopausal women. The Company has identified lead compounds from its selective androgen receptor degrader (SARD) portfolio and has preclinical studies underway that are required prior to initiating a clinical trial in men with castration-resistant prostate cancer (CRPC) during the second half of 2017.

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"In 2016, we broadened our clinical development efforts for enobosarm beyond breast cancer with the initiation of our clinical trial in SUI in postmenopausal women. Early results in this study are very promising, and we have added additional clinical sites and expect to receive top-line results from this trial in the third quarter. Later this year, we also expect to initiate a first in human clinical trial of a SARD in men with advanced prostate cancer," said Robert J. Wills, Ph.D., Executive Chairman of GTx. "There is a great deal of interest in our SARD program based on data we have generated that demonstrate our SARD compounds degrade and inhibit multiple forms of the androgen receptor, including AR splice variants, and may therefore potentially treat CRPC in men who are non-responsive to current androgen therapy."

Corporate Highlights and Anticipated Milestones

Enobosarm in Breast Cancer: The Company’s lead product candidate, enobosarm, a selective androgen receptor modulator (SARM), is being developed as a targeted treatment for two advanced breast cancer indications: (i) estrogen receptor positive (ER+) and androgen receptor positive (AR+) breast cancer, and (ii) AR+ triple negative breast cancer (TNBC). For both clinical trials, the primary efficacy endpoint is a determination of clinical benefit (CB), which is defined as a complete response, partial response or stable disease.

ER+/AR+ Breast Cancer: The Company has an ongoing open-label, multi-center Phase 2 clinical trial of enobosarm in women with advanced, ER+, AR+ breast cancer. Patients receive orally-administered enobosarm (9 mg or 18 mg) daily for up to 24 months. The first stage of evaluation was assessed among the first 18 evaluable patients for each cohort. At least 3 of 18 patients per cohort achieved CB at week 24, and the trial has proceeded to the second stage of enrollment. In Stage 2, if at least 9 of 44 evaluable patients achieve CB at week 24, the trial will have successfully demonstrated its primary endpoint, and those patients achieving CB at 24 weeks will be able to continue treatment for a total of up to 24 months. The two dose cohorts in the trial are being treated independently for the purpose of assessing efficacy. To date, in this ongoing clinical trial:

CB has been demonstrated in both the 9 mg and 18 mg dose cohorts in Stage 1 of the trial, allowing both cohorts to advance to Stage 2 of the trial.
A sufficient number of evaluable patients have already demonstrated CB at week 24 in the 9 mg dose group for the study to achieve the pre-specified primary efficacy endpoint in this ongoing clinical trial. Of the 40 patients in the 9 mg dose cohort whose AR status has been confirmed AR+, 10 patients have demonstrated CB at week 24, 23 patients have discontinued either at or prior to week 24, and 7 patients remain on study and have not yet reached week 24. There are another 5 patients who have been enrolled to the 9 mg cohort whose AR status has not yet been confirmed. Of the 10 evaluable patients achieving CB, 2 had a partial response and 8 had stable disease. The majority of adverse events are grade 1 and 2, and the most common adverse events reported (occurring in ≥10% of patients) include nausea (31%), fatigue (18%), and arthralgias (13%). Elevations in transaminases (ALT and AST) during enobosarm treatment were mild with the majority being grade 1 or 2.
An abstract submitted by Dr. Beth Overmoyer, the lead principal investigator for the clinical trial and a medical oncologist with the Dana-Farber Cancer Center Institute, detailing data from Stage 1 of the 9 mg cohort, has been accepted for the "poster walk" at the European Society for Medical Oncology IMPAKT Breast Cancer Conference, which is being held May 4-6, 2017, in Brussels, Belgium.

Enobosarm appears to be safe and generally well tolerated. The independent Safety Monitoring Committee met in December 2016, and recommended that the clinical trial continue as planned. The trial will continue with a daily dose of either enobosarm 9 mg or 18 mg until 44 evaluable patients in each cohort have completed treatment in order to better characterize the CB response. The Company expects to report top-line results from this study in the third quarter of 2017.

AR+ TNBC: The Company also has an ongoing open-label, multi-center Phase 2 clinical trial to evaluate the efficacy and safety of orally-administered enobosarm in up to 55 women with advanced, AR+ TNBC. Patients receive 18 mg of enobosarm once daily for up to 12 months. Stage 1 of the trial is being assessed among the first 21 evaluable patients, and if at least 2 of 21 patients achieve CB at week 16, then the trial can proceed to Stage 2 of enrollment of up to a total of 41 evaluable patients. The primary efficacy objective of the trial is CB response following 16 weeks of treatment in 41 evaluable patients.

The Company expects to have sufficient data from Stage 1 of the trial by the second quarter of 2017 to determine if patient enrollment should continue into Stage 2 of the trial.
SARMs in Non-Oncologic Indications: The Company also is developing SARMs as potential treatments for both stress urinary incontinence (SUI) in postmenopausal women and Duchenne muscular dystrophy (DMD), a rare disease characterized by progressive muscle degeneration and weakness.

Stress Urinary Incontinence: Enrollment continues in the Company’s ongoing Phase 2 proof-of-concept clinical trial of 3 mg of enobosarm in postmenopausal women with SUI. The Company expects to have top-line results from the trial in the third quarter of 2017.

Duchenne Muscular Dystrophy: Utilizing data developed from its preclinical development efforts, the Company is pursuing a potential strategic collaboration with biopharma companies experienced in orphan drug development to continue the development of a SARM for the treatment of DMD.

SARDs in Prostate Cancer: the Company’s selective androgen receptor degrader (SARD) technology is being evaluated as a potentially novel treatment for men with castration-resistant prostate cancer (CRPC), including those who do not respond or are resistant to currently approved therapies. The Company believes that its SARD compounds will degrade multiple forms of the androgen receptor, including AR splice variants, such as AR-V7, along with mutant versions of the receptor.

Castration-Resistant Prostate Cancer: The Company has screened dozens of compounds from its extensive patented SARD portfolio and has now selected lead compounds that are undergoing further preclinical studies required for a first in human clinical trial, including toxicology studies. It is anticipated that a first in human clinical trial of a SARD will be initiated during the second half of 2017.

The preclinical studies supporting the SARD program have been accepted for presentation and received recognition of merit at several upcoming international meetings, including the Endocrine Society’s annual meeting, ENDO 2017, from April 1-4, 2017, and the annual meeting of the European Association of Urology being held from March 24-28, 2017.

Fourth Quarter and Year-End 2016 Financial Results

As of December 31, 2016, cash and short-term investments were $21.9 million compared to $29.3 million at December 31, 2015.
Research and development expenses for the quarter ended December 31, 2016 were $4.6 million compared to $3.9 million for the same period of 2015. Research and development expenses for the year ended December 31, 2016 were $17.2 million compared to $13.6 million for the year ended December 31, 2015.
General and administrative expenses for the quarter ended December 31, 2016 were $2.3 million compared to $2.1 million for the same period of 2015. General and administrative expenses for the year ended December 31, 2016 were $8.7 million compared to $8.2 million for the year ended December 31, 2015.
The net loss for the quarter ended December 31, 2016 was $6.9 million compared to a net loss of $3.2 million for the same period in 2015. The net loss for the quarter ended December 31, 2015 included a non-cash gain of $2.7 million related to the change in the fair value of the Company’s warrant liability. During the first quarter of 2016, the Company modified its outstanding warrants with no further adjustment to the fair value of these warrants being required subsequent to the first quarter of 2016.
The net loss for the year ended December 31, 2016 was $17.7 million compared to a net loss of $18.7 million for the year ended December 31, 2015. The net loss for the years ended December 31, 2016 and December 31, 2015 included a non-cash gain of $8.2 million and $3.1 million, respectively, related to the change in the fair value of the Company’s warrant liability.
GTx had approximately 15.9 million shares of common stock outstanding as of December 31, 2016. Additionally, there remain warrants outstanding to purchase approximately 6.4 million shares of GTx common stock at an exercise price of $8.50 per share.

On March 15, 2017 CytRx Corporation (NASDAQ: CYTR), a biopharmaceutical research and development company specializing in oncology, reported financial results for the year ended December 31, 2016, and provided an overview of recent accomplishments and upcoming milestones for its research and development programs (Press release, CytRx, MAR 15, 2017, View Source [SID1234518133]).

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"2016 was a pivotal year for CytRx. We achieved several key milestones, including reporting results from our pivotal Phase 3 trial evaluating aldoxorubicin as a treatment for patients with relapsed or refractory soft tissue sarcomas (STS)," said Steven A. Kriegsman, CytRx’s Chairman and CEO. "We look forward to sharing the results of this trial with the U.S. Food and Drug Administration (FDA) this quarter and discussing the regulatory path for a NDA submission for alddoxorubicin. We also hope to present the Phase 3 trial results to the medical community in a peer-reviewed forum in the coming months."

Mr. Kriegsman continued, "Beyond STS, we are also evaluating aldoxorubicin in a Phase 2b trial versus topotecan in patients with second-line small cell lung cancer (SCLC), and we remain on track to report top-line results from that trial by the end of the second quarter of the year."

Upcoming Milestones

Hold Type B pre-NDA meeting with the FDA in March 2017 to obtain input on the Company’s planned NDA filing strategy for aldoxorubicin as a new treatment for patients with STS. Subject to the outcome of this meeting, CytRx intends to file a NDA with the FDA or consider other alternatives which could include strategic alliances or partnerships.
Provide an update of the meeting following official receipt of regulatory guidance from the FDA.
Report top-line results from the global Phase 2b clinical trial evaluating aldoxorubicin versus topotecan in patients with second-line SCLC by the end of the second quarter of 2017.
Present results from the Company’s pivotal Phase 3 clinical trial evaluating aldoxorubicin compared to investigator’s choice in patients with relapsed or refractory STS at a major medical meeting during 2017.
Report overall survival results, a secondary endpoint, from the pivotal Phase 3 clinical trial evaluating aldoxorubicin compared to investigator’s choice in patients with relapsed or refractory STS during 2017.
Present updated data from the ongoing Phase 1b/2 clinical trial of aldoxorubicin in combination with ifosfamide/mesna in patients with advanced sarcomas during 2017.
Select the first internally developed ultra-high potency drug conjugate candidate for future development during 2017.
Fourth Quarter 2016 and Recent Highlights

Granted Type B Pre-NDA Meeting with FDA for Aldoxorubicin. In January 2017, CytRx announced that, in response to a request from the Company, the FDA has agreed to a Type B pre-NDA meeting at which the Company will seek input on its plans for a NDA for aldoxorubicin as a treatment for patients with STS.

Strengthened the Balance Sheet with $8.1 Million in Financing. In December 2016, CytRx announced its entry into securities purchase agreements with existing institutional investors to purchase shares of its common and preferred stock at a purchase price of $0.42 per share in a registered direct offering with gross proceeds of $8.1 million.

Chief Medical Officer Dr. Dan Levitt was Appointed Chief Operating Officer and Dr. Earl W. Brian Joined the Board of Directors. In December 2016, CytRx appointed Chief Medical Officer Daniel Levitt, MD, PhD, to the additional role of Chief Operating Officer. The Company also appointed Earl Warren Brien, MD, a noted sarcoma surgeon, industry consultant, and private healthcare investor, to its Board of Directors.

Reported Updated Results from Pivotal Phase 3 Trial of Aldoxorubicin in Second-Line STS In November 2016. CytRx announced positive updated results from its pivotal Phase 3 clinical trial evaluating aldoxorubicin compared to investigator’s choice in patients with relapsed or refractory STS. The study, which enrolled 433 patients, demonstrated a statistically significant improvement in progression-free survival (PFS) between aldoxorubicin and investigator’s choice therapy in 246 patients with leiomyosarcoma and liposarcoma, (p=0.007). Aldoxorubicin also demonstrated a statistically significant improvement in PFS over investigator’s choice in 312 patients treated in North America (p=0.028). Aldoxorubicin performed better than investigator’s choice for the entire study population and narrowly missed statistical significance (p=0.12). Adverse events were as expected and comparable to prior trials. Patients continue to be followed for overall survival (OS), a secondary endpoint, and CytRx expects the OS data to be available in 2017. CytRx plans to submit the results of this clinical trial for presentation at an upcoming major scientific meeting in 2017.

Presented Positive Aldoxorubicin Combination Clinical Trial Results at ESMO (Free ESMO Whitepaper) 2016 and CTOS 2016. In October 2016, CytRx presented a poster highlighting interim results from its ongoing Phase 1b/2 clinical trial of aldoxorubicin in combination with ifosfamide/mesna in patients with advanced sarcomas at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2016 Congress. In November 2016, the Company presented a poster highlighting updated interim results from the same Phase 1b/2 trial. Of 36 evaluable patients, 35 (95%) achieved clinical benefit (13 (36%) with a partial response and 22 (61%) with stable disease). While the combination did have certain toxicities, none were treatment-limiting.

Full Year 2016 Results

CytRx reported cash, cash equivalents and short-term investments of $57.0 million as of December 31, 2016.

Net loss for the year ended December 31, 2016, was $50.8 million, or $(0.63) per share, compared with a net loss of $58.6 million, or $(0.97) per share, for the year ended December 31, 2015. In 2016, the Company recognized a non-cash gain of $3.8 million on the fair value adjustment of warrant derivative liability primarily related to warrants issued in July 2016, compared to a non-cash gain of $4.4 million in 2015 related to warrants in issued in August 2011, which expired in August 2016. The Company reported licensing revenue of $200,000 in 2016, compared to $100,000 in 2015.

Research and development (R&D) expenses were $35.9 million for 2016, and included development expenses of $27.0 million for aldoxorubicin, approximately $2.3 million for pre-clinical development of new albumin-binding cancer drugs (German lab), and approximately $4.8 million for general operation of our clinical programs. R&D expenses were $43.4 million for 2015.

General and administrative (G&A) expenses were $16.0 million for 2016, compared with $19.7 million for 2015. G&A expenses decreased by approximately 19% primarily due to a significant decrease in legal fees of $4.2 million offset by pre-commercialization costs incurred in the first half of 2016. They also included non-cash stock-compensation expense of $4.9 million and $5.8 million for 2016 and 2015, respectively.