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Merrimack Provides Business Update and Reports 2017 Financial Results

On March 12, 2018 Merrimack Pharmaceuticals, Inc. (Nasdaq: MACK), a clinical-stage oncology company focused on biomarker-defined cancers, reported its fourth quarter and full year 2017 financial results for the period ended December 31, 2017 (Press release, Merrimack, MAR 12, 2018, View Source [SID1234524679]).

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"2017 was a transformative year for Merrimack, in which we reset the company’s foundation to focus on our ten wholly owned clinical and preclinical programs, all targeting biomarker-defined cancers. We are very pleased with the advancements we have made across our pipeline, including today’s announcement to expand enrollment in the SHERLOC study, a randomized Phase 2 trial evaluating MM-121 in non-small cell lung cancer, and our recent dosing of the first patient in the SHERBOC study, a randomized Phase 2 trial evaluating MM-121 in post-menopausal metastatic breast cancer," said Richard Peters, M.D., Ph.D., President and Chief Executive Officer. "We are well-positioned to carry this momentum forward, with three clinical readouts expected in 2018, including randomized Phase 2 data from MM-141 and MM-121 and Phase 1 data from MM-310."

Fourth Quarter and Recent Highlights

Key events from the fourth quarter and more recently include:

As announced separately today, expansion of the enrollment target from 80 to 100 patients in the SHERLOC study, a randomized Phase 2 clinical trial evaluating MM-121 added to standard of care in patients with heregulin-positive non-small cell lung cancer. This augmentation of patient enrollment is driven by the faster than projected enrollment rate seen to date and will result in a strengthened statistical design of the study. Merrimack still expects to report top-line data from this trial in the second half of 2018;

First patient dosed in the SHERBOC study, a randomized, double-blind, placebo-controlled Phase 2 clinical trial evaluating MM-121 added to standard of care in patients with heregulin-positive, hormone-receptor-positive and HER2-negative post-menopausal metastatic breast cancer;

Appointment of George Demetri, M.D., to Merrimack’s Board of Directors. Dr. Demetri is currently a Senior Vice President for Experimental Therapeutics and Director of the Center for Sarcoma and Bone Oncology at Dana-Farber Cancer Institute, as well as a Professor of Medicine at Harvard Medical School, where he is also Co-Director of the Ludwig Center. He is a world-renowned expert in the clinical translation of innovative treatment strategies for cancer, and replaces John Mendelsohn, M.D., who had served on Merrimack’s Board since 2012;
Formation of a new Scientific Advisory Board (SAB) with extensive expertise in precision oncology, bioengineering, drug discovery and clinical development. Members include: Peter Blume-Jensen, M.D., Ph.D.; George Demetri, M.D.; Douglas Lauffenburger, Ph.D.; Peter Sorger, Ph.D.; and Josep Tabernero, M.D., Ph.D. The SAB will work closely with Merrimack’s senior management team to advance the company’s pipeline of targeted cancer therapies; and

Financially, closure of 2017 with $93.4 million in cash and cash equivalents and extinguishment of $60.8 million of convertible debt in the fourth quarter of 2017, resulting in a debt-free balance sheet.
Upcoming Milestones:

Merrimack anticipates the following upcoming clinical milestones:

Top-line results in the first half of 2018 from the CARRIE study, a randomized Phase 2 clinical trial evaluating MM-141, a bispecific antibody targeting the IGF-1 and HER3 receptors, added to standard of care in patients with front-line metastatic pancreatic cancer who have high serum levels of free IGF-1;

Top-line results in the second half of 2018 from the SHERLOC study, a randomized Phase 2 clinical trial evaluating MM-121, a fully human monoclonal antibody targeting the HER3 receptor, added to standard of care in patients with heregulin positive non-small cell lung cancer; and

Safety data and maximum tolerated dose in the second half of 2018 from the Phase 1 clinical study of MM-310, an antibody-directed nanotherapeutic (ADN) targeting the EphA2 receptor, in patients with solid tumors.
Fourth Quarter and Full Year 2017 Financial Results

The following summarizes Merrimack’s financial results for the three months and year ended December 31, 2017:

Research and development expenses from continuing operations for the three months ended December 31, 2017 were $12.4 million, compared to $25.6 million for the three months ended December 31, 2016. Research and development expenses from continuing operations for the year ended December 31, 2017 were $67.3 million, compared to $109.6 million for the year ended December 31, 2016. Research and development spending for the three months and year ended December 31, 2017 was less than expenditures over comparable periods in 2016, primarily due to Merrimack’s refocused clinical and preclinical pipeline;

General and administrative expenses for the three months ended December 31, 2017 from continuing operations were $4.7 million, compared to $11.0 million for the three months ended December 31, 2016. General and administrative expenses for the year ended December 31, 2017 from continuing operations were $28.5 million, compared to $32.1 million for the year ended December 31, 2016. General and administrative spending for the three months and year ended December 31, 2017 was less than expenditures over comparable periods in 2016, primarily due to a decrease in corporate expenses related to reduced headcount levels and stock-based compensation following the asset sale to Ipsen S.A.;

Net loss attributable to Merrimack’s continuing operations for the three months ended December 31, 2017 was $11.8 million, or $0.89 per share, compared to a net loss attributable to Merrimack’s continuing operations of $40.1 million, or $2.93 per share, for the three months

ended December 31, 2016. Net loss attributable to Merrimack’s continuing operations for the year ended December 31, 2017 was $74.8 million, or $5.66 per share, compared to a net loss attributable to Merrimack’s continuing operations of $154.5 million, or $12.33 per share, for the year ended December 31, 2016; and

As of December 31, 2017, Merrimack had 13.3 million shares of common stock, $0.01 par value per share, outstanding.
Financial Outlook

Merrimack continues to believe that its cash and cash equivalents of $93.4 million as of December 31, 2017 and potential net milestone payments anticipated from Shire will be sufficient to fund its planned operations into the second half of 2019.

Conference Call and Webcast

Merrimack will host a live conference call and webcast today, Monday, March 12, 2018 at 8:30 am ET, to provide an update on its operational progress and a summary of these financial results.

Investors and the general public are invited to listen to the call by dialing (877) 564-1301 (domestic) or (224) 357-2394 (international) five minutes prior to the start of the call and providing the passcode 8074029. A listen-only webcast of the call can be accessed in the Investors section of Merrimack’s website, investors.merrimack.com, and a replay of the call will be archived there for six weeks following the call.

Kura Oncology Provides Regulatory Update on Tipifarnib and Reports Fourth Quarter and Full Year 2017 Financial Results

On March 12, 2018 Kura Oncology, Inc. (Nasdaq:KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for oncology, today provided a regulatory update for its lead product candidate, tipifarnib, and reported fourth quarter and full year 2017 financial results (Press release, Kura Oncology, MAR 12, 2018, View Source [SID1234524678]).

"Following a successful end of Phase 2 meeting with the FDA, we plan to initiate a single-arm, registration-directed trial of tipifarnib in at least 59 recurrent or metastatic patients with HRAS mutant squamous cell head and neck cancer (HNSCC) with objective response rate (ORR) as the primary endpoint," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "We expect to initiate this trial, which we are calling the AIM-HN trial, in the second half of 2018. We are encouraged by the feedback we received from the FDA regarding the development path for tipifarnib in HRAS mutant HNSCC, and we look forward to providing more specific information regarding the design and execution of the trial in the months ahead."

Recent Operational Highlights

Feedback from end of Phase 2 clinical meeting with the FDA – Based on feedback from the FDA, Kura is planning for the initiation of its AIM-HN trial of tipifarnib in HRAS mutant HNSCC patients, pending completion of site feasibility activities and submission of the final protocol to the FDA. The AIM-HN trial will be a global, multi-center, single-arm, study of at least 59 recurrent or metastatic patients with measurable disease as determined by RECIST version 1.1 criteria. The primary endpoint will be ORR, as determined by independent radiological review. The FDA indicated in the minutes from the meeting that the AIM-HN trial, as currently designed, may be adequate to support an NDA seeking accelerated approval.

Update on Phase 2 trial of tipifarnib in HRAS mutant HNSCC – In February 2018, Kura reported updated preliminary results from its Phase 2 trial of tipifarnib in patients with HRAS mutant HNSCC at the 2018 Multidisciplinary Head and Neck Cancers Symposium. The update showed that five of the six evaluable patients achieved a confirmed, partial response. Two of these patients achieved durable responses beyond a year and a half. The one evaluable patient who did not achieve a response, based on standard RECIST criteria, experienced prolonged disease stabilization for more than six months. Tipifarnib has been generally well-tolerated with adverse events observed consistent with its known safety profile.

Potential biomarkers identified for tipifarnib in hematologic malignancies – In December 2017, Kura presented new findings at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting that identified activation of the CXCL12 pathway and bone marrow homing of myeloid cells as potential biomarkers of tipifarnib’s activity in certain hematologic malignancies, including peripheral T-cell lymphoma (PTCL), myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML) and acute myeloid leukemia (AML). Based on these observations, the company is now prospectively investigating these potential biomarkers in its ongoing Phase 2 trials in various hematologic malignancies.
Upcoming Potential Milestones and Expectations for Clinical Programs

Preclinical data for tipifarnib in HRAS mutant squamous non-small cell lung tumor models at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2018

Preclinical biomarker data from KO-947 in squamous cell carcinomas at AACR (Free AACR Whitepaper) in April 2018

Initiation of the tipifarnib SEQ-HN trial, a screening and outcomes study in HRAS mutant HNSCC in the first half of 2018

Initiation of the tipifarnib AIM-HN trial in HRAS mutant HNSCC in the second half of 2018

Data from Phase 1 dose-escalation trial of KO-947 in the second half of 2018

Additional updates from the ongoing Phase 2 study of tipifarnib in HRAS mutant HNSCC in the second half of 2018

Initiation of a proof-of-concept study of tipifarnib in HRAS mutant squamous non-small cell lung cancer through the Spanish Lung Cancer Group in 2018

Additional clinical data from tipifarnib in hematologic malignancies in the second half of 2018

Submission of an investigational new drug (IND) application for KO-539 in late 2018 or early 2019
Financial Results for the Fourth Quarter and the Full Year 2017

Research and development expenses for the fourth quarter of 2017 were $8.1 million, compared to $5.5 million for the fourth quarter of 2016. Research and development expenses for the full year 2017 were $26.4 million, compared to $20.4 million for the prior year.

General and administrative expenses for the fourth quarter of 2017 were $2.9 million, compared to $2.0 million for the fourth quarter of 2016. General and administrative expenses for the full year 2017 were $9.7 million, compared to $8.0 million for the prior year.

Net loss for the fourth quarter of 2017 was $10.7 million, compared to a net loss of $7.3 million for the fourth quarter of 2016. Net loss for the full year 2017 was $35.4 million, compared to a net loss of $27.6 million for the prior year.

Cash, cash equivalents and short-term investments totaled $93.1 million as of December 31, 2017, compared with $100.8 million as of September 30, 2017 and $67.8 million as of December 31, 2016.

Subsequently, in January 2018, Kura sold an aggregate of approximately 3.1 million shares of its common stock under an ATM facility for net proceeds of $57.4 million.

Management expects that current cash, cash equivalents and short-term investments will be sufficient to fund its current operations into the first half of 2020.
Conference Call and Webcast

Kura’s management will host a webcast and conference call today at 4:30 p.m. ET / 1:30 p.m. PT today, March 12, 2018, to discuss the regulatory update and financial results. The live call may be accessed by dialing (877) 516-3514 for domestic callers and (281) 973-6129 for international callers and using conference ID # 5196505. A live webcast of the call will be available from the Investors and Media section of the company website at www.kuraoncology.com, and will be archived there for 30 days.

Iovance Biotherapeutics Reports Fourth Quarter and Full-Year 2017 Financial Results and Provides Corporate Update

On March 12, 2018 Iovance Biotherapeutics, Inc. (NASDAQ:IOVA), a biotechnology company developing novel cancer immunotherapies based on tumor-infiltrating lymphocyte (TIL) technology, reported its fourth quarter and year-end 2017 (Press release, Iovance Biotherapeutics, MAR 12, 2018, View Source;p=RssLanding&cat=news&id=2337617 [SID1234524677]).

"We ended 2017 having completed significant accomplishments involving both manufacturing and clinical aspects of development of TIL as a viable commercial therapy. We developed a new manufacturing method, lasting 22 days and yielding a cryopreserved product, conducted a clinical study investigating the efficacy of this method, reported preliminary data showing responses from this generation 2 manufacturing method in late-line metastatic melanoma patients and subsequently selected this manufacturing method for all ongoing and future clinical trials for Iovance. We have also expanded our manufacturing capacity in the US, in commercial-ready suites, and built out our capacity in the EU. On the clinical front, we are currently running four studies to evaluate the potential breadth of utility of TIL therapy in multiple indications," said Dr. Maria Fardis, Ph.D., MBA, president and chief executive officer of Iovance Biotherapeutics. "In early 2018, we successfully completed a common stock public offering adding approximately $162 million in net proceeds to the cash reserves. The proceeds from this public offering, combined with the year-end cash balance, put us in a strong position to execute on our upcoming milestones."

2017 Achievements and 2018 Updates

Manufacturing

Completed development of the generation 2 manufacturing method and the associated technology transfer into multiple CMOs in the US and the EU.
Entered into a new three-year Manufacturing Services Agreement (MSA) with PharmaCell B.V., now a subsidiary of Lonza Group Ltd., in the Netherlands to support EU manufacturing. PharmaCell is now able to receive clinical samples and manufacture TIL therapy for patients.
Entered into a new two-year MSA with H. Lee Moffitt Cancer Center and Research Institute (Moffitt).
Commenced a partnership with TrakCel Ltd. to build a scheduling and logistics software tool that automates the supply chain for the company’s TIL therapy.
Clinical

Presented clinical data from the first cohort of the company’s Phase 2 trial investigating LN-144 for the treatment of patients with metastatic melanoma, known as C-144-01, at the 2017 ASCO (Free ASCO Whitepaper) Annual Meeting in June.
Began patient dosing in the second cohort of C-144-01 and reported preliminary data at the SITC (Free SITC Whitepaper) Annual Meeting in November.
Began patient dosing in C-145-03, the company’s Phase 2 trial of LN-145 for the treatment of patients with recurrent and/or metastatic squamous cell carcinoma of the head and neck and reported preliminary data from this study in January 2018.
Began patient dosing in C-145-04, the company’s Phase 2 trial of LN-145 for the treatment of patients with recurrent, metastatic or persistent cervical carcinoma and provided early response data from evaluable patients in early 2018 as well.
Entered into a new clinical grant agreement with Moffitt to provide funding for a clinical study of TIL therapy in non-small cell lung cancer (NSCLC) and Moffitt began patient enrollment in this study in patients with advanced NSCLC cancer combining TIL and nivolumab in patients who have progressed on nivolumab.
First site was activated in the Iovance IOV-LUN-201 study to treat checkpoint naïve patients with NSCLC.
Entered into a multi-year strategic alliance with M.D. Anderson.
First clinical site was activated in Europe for the C-144-01 melanoma study.
Regulatory

Received Fast Track designation in the U.S. for LN-144 for the treatment of advanced melanoma.
Submitted Clinical Trial Applications in multiple countries in Europe in support of the company’s Phase 2 clinical trials and received multiple approvals to commence clinical trials in Europe.
Research

Entered into a collaboration with the Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute to evaluate TILs, marrow infiltrating lymphocytes (MILs), and peripheral-blood associated lymphocytes in acute myeloid leukemia (AML) and chronic lymphocytic leukemia (CLL).
Late-breaking abstract, titled Anti-OX40 agonistic antibody enhances ex vivo CD8+ TIL expansion with increased T-cell effector function, accepted for presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2018. The poster will be available on April 16, 2018.
Corporate

Changed corporate name from Lion Biotechnologies, Inc. to Iovance Biotherapeutics, Inc. and reincorporated from a Nevada corporation to a Delaware corporation.
Appointed Timothy E. Morris as the company’s chief financial officer in August 2017.
Raised approximately $53.7 million in net proceeds, after deducting underwriting discounts and offering expenses, through a public offering that closed in September 2017.
In January 2018, the company closed an underwritten public offering of 15,000,000 shares of its common stock at a public offering price of $11.50 per share, before underwriting discounts. The shares sold at closing included 1,956,521 shares issued upon the exercise in full by the underwriter of its option to purchase additional shares at the public offering price less the underwriting discount. The gross proceeds from the offering, before deducting the underwriting discounts and commissions and other estimated offering expenses payable by the company, were $172.5 million with estimated net proceeds to the company of approximately $161.7 million.
Fourth Quarter and Full-Year 2017 Financial and Operating Results

At December 31, 2017, the company held $145.4 million in cash, cash equivalents and short-term investments, compared to $166.5 million at December 31, 2016. Net cash used in operating activities was $78.7 million during the year ended December 31, 2017.

Iovance anticipates cash, cash equivalents and investments to be between $190 million and $210 million at December 31, 2018.

The company is providing both GAAP and non-GAAP financial information. All non-GAAP information excludes amounts related to stock-based compensation. See "Use of Non-GAAP Financial Measures" below for a description of the company’s non-GAAP Financial Measures. Reconciliation between certain GAAP and non-GAAP measures is provided at the end of this press release.

GAAP and Non-GAAP Net Loss Attributable to Common Stockholders

GAAP net loss attributable to common stockholders for the quarter ended December 31, 2017 was $25.9 million, or $0.36 per share, compared to GAAP net loss of $15.7 million or $0.25 per share for the quarter ended December 31, 2016.

Non-GAAP net loss attributable to common stockholders for the quarter ended December 31, 2017 was $23.1 million, or $0.32 per share, compared to $12.6 million, or $0.20 per share for the quarter ended December 31, 2016. The non-GAAP net loss for the quarters ended December 31, 2017 and 2016 excludes $2.8 million and $3.1 million of non-cash stock-based compensation, respectively.

GAAP net loss attributable to common stockholders for the year ended December 31, 2017 was $92.1 million, or $1.41 per share, compared to $102.3 million or $1.85 per share for the year ended December 31, 2016. The 2016 GAAP net loss attributable to common stockholders included a one-time deemed dividend related to a charge of $49.5 million incurred because of the conversion feature of the Series B convertible preferred stock. Non-GAAP net loss for the year ended December 31, 2017 was $80.1 million, or $1.23 per share, compared to non-GAAP net loss of $34.0 million or $0.62 per share for the year ended December 31, 2016. The non-GAAP net loss for the years ended December 31, 2017 and 2016 excludes $12.0 million and $18.9 million of non-cash stock-based compensation, respectively. The 2016 non-GAAP net loss also excludes the one-time charge of $49.5 million related to the deemed dividend.

GAAP and Non-GAAP Expenses

GAAP research and development (R&D) expenses were $20.7 million for the quarter ended December 31, 2017, an increase of $10.6 million compared to $10.1 million for the quarter ended December 31, 2016. The increase in R&D expenses is due to increased spending on clinical activities and manufacturing. R&D associated stock-based compensation expense was $0.9 million for the three months ended December 31, 2017 and $1.5 million for the three months ended December 31, 2016. Non-GAAP R&D expenses were $19.8 million for the quarter ended December 31, 2017, an increase of $11.1 million, compared to $8.7 million for the quarter ended December 31, 2016.

GAAP general and administrative (G&A) expenses were $5.4 million for the quarter ended December 31, 2017, a decrease of $0.5 million compared to $5.8 million for the quarter ended December 31, 2016. G&A associated stock-based compensation expense was $1.8 million for the three months ended December 31, 2017 and $1.7 million for the three months ended December 31, 2016. Non-GAAP G&A expenses were $3.5 million for the quarter ended December 31, 2017, a decrease of $0.6 million, compared to $4.1 million for the quarter ended December 31, 2016.

Use of Non-GAAP Financial Measures

This press release contains non-GAAP financial measures, including expenses adjusted to exclude certain non-cash expenses. These measures are not in accordance with, or an alternative to, generally accepted accounting principles (GAAP), and may be different from non-GAAP financial measures used by other companies. The items included in GAAP presentations but excluded for purposes of determining non-GAAP financial measures for the periods presented in this press release are: (i) the non-cash stock-based compensation expense which may fluctuate from period-to-period based on factors including the timing and accounting of grants for stock options and changes in the company’s stock price which impacts the fair value of options granted, and (ii) the one-time non-cash deemed dividend related to the conversion feature of the Series B Preferred Stock. The company believes the presentation of non-GAAP financial measures provides useful information to management and investors regarding various financial and business trends relating to the company’s financial condition and results of operations. When GAAP financial measures are viewed in conjunction with non-GAAP financial measures, investors are provided with a more meaningful understanding of Iovance’s ongoing operating performance. In addition, these non-GAAP financial measures are among those indicators the company uses as a basis for evaluating operational performance, allocating resources and planning and forecasting future periods. Non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. To the extent this release contains historical or future non-GAAP financial measures, the company has also provided corresponding GAAP financial measures for comparative purposes. Reconciliation between certain GAAP and non-GAAP measures is provided at the end of this press release. Beginning in 2018, Iovance will no longer report non-GAAP expenses or non-GAAP net loss per share.

Webcast and Conference Call

Iovance will host a conference call today at 4:30 p.m. ET to discuss these fourth quarter and full-year 2017 results and provide a corporate update. The conference call dial-in numbers are: 1-844-646-4465 (domestic) or 1-615-247-0257 (international). The conference ID access number for the call is 1497629. The live webcast can be accessed under "News & Events" in the "Investors" section of the company’s website at View Source or you may use the link: View Source

A replay of the call will be available from March 12, 2018 at 7:30 p.m. ET to April 18, 2018 at 8:30 p.m. ET. To access the replay, please dial 1-855-859-2056 (domestic) or 1-404-537-3406 (international). The conference ID number for the replay is 1497629. The archived webcast will be available for thirty days in the Investors section of Iovance Biotherapeutics’ website at View Source

Inovio Pharmaceuticals to Report Fourth Quarter and Year End 2017 Financial Results on March 14, 2018

On March 12, 2018 Inovio Pharmaceuticals, Inc. (NASDAQ:INO) reported that it will host a conference call and live webcast to report its 2017 fourth quarter and year end financial results on Wednesday, March 14, 2018 at 4:30 p.m. ET (Press release, Inovio, MAR 12, 2018, View Source [SID1234524676]).

A live and archived version of the audio presentation will be available online at View Source This is a listen-only event but will include a live Q&A with analysts.

A replay of the conference call will be accessible two hours after the call at 877-481-4010 (domestic) or 919-882-2331 (international) using replay ID 26416.

GTx Provides Corporate Update and Reports Fourth Quarter and Full Year 2017 Financial Results

On March 12, 2018 GTx, Inc. (Nasdaq:GTXI) reported financial results for the fourth quarter and year ended December 31, 2017 and highlighted recent accomplishments and upcoming milestones (Press release, GTx, MAR 12, 2018, View Source;p=RssLanding&cat=news&id=2337621 [SID1234524675]).

"2017 was a transformational year for GTx, marked by positive results from our Phase 2 proof-of-concept clinical trial in post-menopausal women with stress urinary incontinence and the initiation of the ASTRID trial, a Phase 2 placebo-controlled clinical trial of enobosarm for the treatment of SUI," said Robert J. Wills, Ph.D., Executive Chairman of GTx. "We believe that there is a significant opportunity for an effective oral therapy to treat SUI and look forward to results from the ASTRID trial in the second half of 2018."

Clinical Highlights and Anticipated Milestones

Stress Urinary Incontinence (SUI) Phase 2 Proof-of-Concept Trial:

Last week at the Society of Urodynamics, Female Pelvic Medicine, & Urogenital Reconstruction (SUFU) Meeting and at the International Continence Society (ICS) annual meeting in 2017, positive results were presented from the Company’s Phase 2 proof-of-concept (POC) clinical trial of enobosarm 3 mg administered orally in post-menopausal women with SUI. The results, including additional positive results presented at SUFU in a subset of women with both urge and stress incontinence, are summarized as follows:

At the end of the 12-week treatment period, all of the 18 enobosarm-treated women showed a clinically meaningful reduction in stress urinary incontinence episodes per day (the primary endpoint of the trial).

Mean stress leaks decreased by 81 percent from baseline;
Stress leaks decreased from a mean of 5.17 leaks/day at baseline to 1.00 leak/day;
All 18 patients demonstrated clinically meaningful reductions in stress urinary incontinence episodes per day, compared to baseline, of at least 50 percent; and
Median Medical, Epidemiologic and Social Aspects of Aging (MESA) scores for SUI decreased from 79.5 percent to 44.5 percent.
The reduction in incontinence episodes was sustained, or durable, well beyond the 12-week treatment period.

Patients are being followed for up to seven months post-treatment to assess enobosarm’s duration of effect, and to date no patient, including nine patients who have reached seven months, has returned to her baseline level of SUI episodes.
Additional positive results in subset of postmenopausal women suggest dual treatment effect on both urge incontinence and stress urinary incontinence.

While all of the women in the trial had predominant SUI, some also experienced urge incontinence (UI). Eleven of the 18 women completing 12 weeks of treatment were determined to have both SUI and UI at baseline, also known as mixed incontinence.

Mean urge leaks decreased by 68 percent from baseline;
Urge leaks decreased from a mean of 1.41 leaks/day at baseline, to 0.45 leaks/day;
9 of 11 women demonstrated a reduction in their number of UI leaks, compared to baseline, with 8 of 11 demonstrating a clinically meaningful reduction in their UI episodes per day of at least 50 percent; and
Median Medical, Epidemiologic and Social Aspects of Aging (MESA) scores for UI decreased from 56 percent to 22 percent.
Magnetic resonance imaging (MRI) was used to quantitatively measure muscle in the pelvic floor of 17 women at 12 weeks compared to their baseline. The results showed a statistically significant increase in several important measurements and support the mechanism of action of enobosarm on the pelvic floor.

At week 12, mean levator ani muscle thickness increased 1.15 mm (p=0.006) from a baseline measurement of 4.79 mm;
At week 12, mean inner urethral muscle diameter increased 0.7 mm (p=0.002) from a baseline measurement of 10.7 mm; and
At week 12, mean outer urethral muscle diameter increased 0.7 mm (p=0.0003) from a baseline measurement of 15.4 mm.
There were no serious adverse events reported during the trial and reported adverse events were minimal and included headaches, nausea, fatigue, hot flashes, insomnia, muscle weakness and acne. Mild transient elevations in liver enzymes that were within normal limits were observed, except for one patient with levels greater than 1.5 times the upper limit of normal which returned to normal following her 12-week treatment period. Reductions in total cholesterol, LDL-C, HDL-C and triglycerides were also observed.

SUI Phase 2 Placebo-Controlled ASTRID Clinical Trial:

Based on the positive results from the Phase 2 POC trial, the Company initiated a randomized, double-blinded, placebo-controlled, Phase 2 trial to assess the efficacy and safety of enobosarm administered orally in approximately 400 post-menopausal women with SUI compared to placebo. More information about the ASTRID (Assessing Enobosarm for Stress Urinary Incontinence Disorder) trial, which is ongoing, can be found here. Top-line results from the Phase 2 placebo-controlled clinical trial are expected in the second half of 2018.

Prostate Cancer:

The Company has a Selective Androgen Receptor Degrader (SARD) preclinical program to evaluate its novel SARD technology in castration-resistant prostate cancer (CRPC). The Company has ongoing mechanistic preclinical studies designed to select the most appropriate compound to potentially advance into a first-in-human clinical trial.

Fourth Quarter and Year-End 2017 Financial Results

As of December 31, 2017, cash and short-term investments were $43.9 million compared to $21.9 million at December 31, 2016.
Research and development expenses for the quarter ended December 31, 2017 were $6.9 million compared to $4.6 million for the same period of 2016. Research and development expenses for the year ended December 31, 2017 were $21.5 million compared to $17.2 million for the year ended December 31, 2016.
General and administrative expenses for the quarter ended December 31, 2017 were $2.5 million compared to $2.3 million for the same period of 2016. General and administrative expenses for the year ended December 31, 2017 were $9.2 million compared to $8.7 million for the year ended December 31, 2016.
The net loss for the quarter ended December 31, 2017 was $9.3 million compared to a net loss of $6.9 million for the same period in 2016.
The net loss for the year ended December 31, 2017 was $30.4 million compared to a net loss of $17.7 million for the year ended December 31, 2016. The net loss for the year ended December 31, 2016 included a non-cash gain of $8.2 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.
GTx had approximately 21.5 million shares of common stock outstanding as of December 31, 2017. Additionally, there are warrants outstanding to purchase approximately 6.4 million shares of GTx common stock at an exercise price of $8.50 per share and approximately 3.3 million shares of GTx common stock at an exercise price of $9.02.