On May 15, 2018 GTx, Inc. (Nasdaq:GTXI) reported financial results for the first quarter ended March 31, 2018 and highlighted recent accomplishments and upcoming milestones (Press release, GTx, MAY 15, 2018, View Source;p=RssLanding&cat=news&id=2349054 [SID1234526616]).

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"GTx is off to a strong start in 2018, with data presented at the SUFU Meeting in March supporting enobosarm’s potential to treat stress urinary incontinence (SUI). The data included additional positive results in a subset of women with both SUI and urge incontinence," said Robert J. Wills, Ph.D., Executive Chairman of GTx. "We look forward to presenting additional data from our open-label, Phase 2 proof-of-concept clinical trial evaluating enobosarm 3 mg in postmenopausal women with SUI at the upcoming AUA meeting in May. In addition, we completed patient enrollment in our placebo-controlled, Phase 2 clinical trial of enobosarm in postmenopausal women with SUI several months ahead of schedule. Enrollment exceeded the 400 patients planned per protocol, and we are eager to report top-line results early in the fourth quarter of 2018."

Clinical Highlights and Anticipated Milestones

Stress Urinary Incontinence (SUI):

Enobosarm, a Selective Androgen Receptor Modulator (SARM), is being evaluated in Phase 2 clinical development for SUI, the Company’s lead indication. Recent and upcoming important milestones are summarized as follows:

At the Society of Urodynamics, Female Pelvic Medicine, & Urogenital Reconstruction (SUFU) Meeting in March, 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. Details of the SUFU presentation can be found here and are summarized below:
At the end of the 12-week treatment period, all 18 enobosarm-treated women demonstrated clinically meaningful (50 percent or greater) reductions in stress urinary incontinence episodes per day, compared to baseline.
The reduction in incontinence episodes was sustained, or durable, well beyond the 12-week treatment period.
Additional positive results in a subset of postmenopausal women suggest a dual treatment effect on both SUI and urge incontinence (UI).
Magnetic resonance imaging (MRI) results showed a statistically significant increase in several important measurements and support the mechanism of action of enobosarm on the pelvic floor.
There were no serious adverse events reported 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.
On May 18, 2018, a podium presentation at the 2018 American Urological Association (AUA) meeting will update results from the Phase 2 POC clinical trial of enobosarm.
The Company has an ongoing randomized, double-blinded, placebo-controlled, Phase 2 trial to assess the efficacy and safety of enobosarm administered orally in post-menopausal women with SUI compared to placebo. More information about the ASTRID (Assessing Enobosarm for Stress Urinary Incontinence Disorder) trial can be found here.
In April, the Company completed patient enrollment in the ASTRID trial several months ahead of schedule, enrolling 493 women at over 60 clinical trial centers across the United States. Top-line results are expected early in the fourth quarter 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.

First Quarter 2018 Financial Results

As of March 31, 2018, cash and short-term investments were $32.1 million compared to $43.9 million at December 31, 2017.
Research and development expenses for the quarter ended March 31, 2018 were $11.0 million compared to $4.2 million for the same period of 2017.
General and administrative expenses for the quarter ended March 31, 2018 were $2.7 million compared to $2.1 million for the same period of 2017.
The net loss for the quarter ended March 31, 2018 was $13.6 million compared to a net loss of $6.3 million for the same period in 2017.
GTx had approximately 22.5 million shares of common stock outstanding as of March 31, 2018. Additionally, there are warrants outstanding to purchase approximately 5.3 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.
About the Phase 2 Proof-of-Concept Clinical Trial

The single-arm, open-label Phase 2 clinical trial is evaluating enobosarm in postmenopausal women with SUI, and is the first clinical trial to evaluate an orally-administered selective androgen receptor modulator (SARM) for SUI. This clinical trial is closed to enrollment; more information about the clinical trial can be found here.

About the Phase 2 ASTRID Clinical Trial

In addition to the Phase 2 proof-of-concept clinical trial being presented at AUA, GTx also has a larger, ongoing, placebo-controlled Phase 2 clinical trial evaluating enobosarm in postmenopausal women with SUI. The study, called ASTRID (Assessing Enobosarm for Stress Urinary Incontinence Disorder), completed enrollment (n=493) recently and is being conducted at over 60 clinical trial centers across the United States. Top-line results are expected early in the fourth quarter of this year. More information about the ASTRID clinical trial can be found here.

About Enobosarm and SUI

Enobosarm (GTx-024), a selective androgen receptor modulator (SARM), has been evaluated in 25 completed or ongoing clinical trials enrolling over 2,100 subjects, in which approximately 1,500 subjects were treated with enobosarm at doses ranging from 0.1 mg to 100 mg. At all evaluated dose levels, enobosarm was observed to be generally safe and well tolerated. The rationale for evaluating enobosarm as a treatment for SUI is supported by preclinical in vivo data demonstrating increases in pelvic floor muscle mass following treatment with GTx’s SARM compounds, including enobosarm, and the proof-of-concept Phase 2 clinical trial of enobosarm 3 mg for the treatment of postmenopausal women with SUI.

About Stress Urinary Incontinence

Stress urinary incontinence (SUI) refers to the unintentional leakage of urine during activities that increase abdominal pressure such as coughing, sneezing or physical exercise. SUI, the most common type of incontinence suffered by women, affects up to 35 percent of adult women. There are a variety of treatments that are used to treat SUI in women, such as behavioral modification and pelvic floor physical therapy, especially as initial treatment options. As the condition worsens however, bulking agents and surgical procedures are often the most widely used treatments

On May 15, 2018 Cleveland BioLabs, Inc. (NASDAQ:CBLI) reported financial results and development progress for the first quarter ended March 31, 2018 (Press release, Cleveland BioLabs, MAY 15, 2018, View Source [SID1234526615]).

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Cleveland BioLabs reported a net loss of $(1.2) million, excluding minority interests, for the first quarter of 2018, or $(0.11) per share, compared to a net loss, excluding minority interests, of $(1.7) million, or $(0.15) per share, for the same period in 2017. The decrease in net loss was primarily due to a non-cash adjustment to our warrant liabilities and reduced operating costs partially offset by reduced revenue.

As of March 31, 2018, the Company had $7.7 million in cash, cash equivalents and short-term investments, which, based on the Company’s current operational plan, is estimated to fund operations for at least one year beyond the filing date of our Form 10-Q.

Yakov Kogan, Ph.D., MBA, Chief Executive Officer, stated, "The past quarter was one of important progress for the company and our Entolimod program. We continued our pursuit of a pre- Emergency Use Authorization ("pre-EUA") with the U.S. Food and Drug Administration ("FDA") and a Marketing Authorization Application ("MAA") with the European Medicines Agency ("EMA") for entolimod as a medical radiation countermeasure."

"As previously reported, the company has received Day 120 review questions from EMA regarding the MAA for entolimod and a formal notification that responses to these questions should be submitted to the agency by September 14, 2018. Consistent with the FDA review of the company’s pre-EUA application, several of the EMA review questions focused on the comparability between the entolimod formulation used in prior safety and efficacy studies and the formulation proposed for commercialization. The analytical analysis of the specimens collected during an in-vivo biocomparability study in non-human primates to address these comparability questions is ongoing," added Dr. Kogan. "Other Day 120 questions from the EMA are generally similar to those discussed in the past with the FDA, and include questions on validation of various aspects of manufacturing, the animal-to-human dose-conversion strategy, and the human safety database."

Further Financial Results

Revenue for the first quarter of 2018 decreased to $0.2 million compared to $0.6 million for the first quarter of 2017. The decrease was primarily due to decreased revenue from our Joint Warfighter Medical Research Program contract from the Department of Defense for the continued development of the entolimod as a medical radiation countermeasure.

Research and development costs for the first quarter of 2018 decreased to $1.3 million compared to $1.4 million for the first quarter of 2017. The reduction in research and development costs is primarily due to reductions in entolimod for biodefense applications partially offset by increases in the entolimod family of compounds for oncology.

General and administrative costs for the first quarter of 2018 decreased to $0.7 million compared to $0.8 million for the first quarter of 2017. This decrease was primarily attributable to reductions in personnel and other operating costs in connection with cost savings efforts to streamline operations.

On May 15, 2018 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a biopharmaceutical company dedicated to the development and delivery of high quality, cost-effective pharmaceutical products and innovative therapeutics to patients in the U.S., China and throughout the world, reported financial results for the first quarter and provided a review of recent accomplishments and anticipated upcoming milestones (Press release, CASI Pharmaceuticals, MAY 15, 2018, View Source [SID1234526614]).

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Ken K. Ren, Ph.D., CASI’s Chief Executive Officer, commented, "We have made significant progress in 2018, having acquired a portfolio of 25 US FDA-approved ANDAs from Sandoz, which has substantially broadened our product pipeline. The acquisition squarely enhances our strategic focus of capitalizing on the evolving CFDA landscape, which is expected to streamline and accelerate the approval process for high-quality generic products."

Dr. Ren continued, "The capital raise announced in March leaves us well-positioned to drive forward with our EVOMELA commercialization plans, and given the positive tenor of the April 2018 Center for Drug Evaluation (CDE) Expert Advisory Committee meeting, where no questions were posed and no new materials were requested, we remain encouraged that we will receive additional feedback from the CDE/CFDA regarding the EVOMELA application in the coming weeks. We remain committed to developing a strong and robust product portfolio through licensing opportunities as well as seeking other advantageous relationships that will further expand our footprint in both the U.S. and in China."

CASI Pharmaceuticals, Inc. / 9620 Medical Center Drive / Suite 300 / Rockville, MD 20850
Phone 240.864.2600 / Fax 301.315.2437

First Quarter and Recent Business Highlights

Company prepares for commercial launch of EVOMELA in China – In March 2018, CASI hired Thomas Zhang as General Manager of its Sales & Marketing team in China and is currently building out a robust and experienced team in China to support commercial operations there. Mr. Zhang has over 20 years of commercial experience in China, and prior to joining CASI was responsible for the successful launch and commercialization of Herceptin and Xeloda at Shanghai Roche Pharmaceuticals Co., Ltd. A team of seasoned sales and marketing professionals are being hired in preparation for the launch of EVOMELA following anticipated marketing authorization.

Update on advisory committee meeting notice from China Center for Drug Evaluation (CDE) – On April 26, 2018, the Center for Drug Evaluation (CDE), a group within the China FDA in charge of technical review, held a meeting of the Expert Advisory Anti-Tumor (Oncology) Drugs Committee (the "Advisory Committee") to review the EVOMELA application. In preparation for the Advisory Committee meeting, no additional questions were posed and the CDE requested no new analysis or materials. The Advisory Committee meeting did not include sponsor or public participants and the Advisory Committee will provide its recommendations directly to the CDE. CASI anticipates receiving feedback from the CDE/CFDA on the EVOMELA application in the coming weeks.

Acquired portfolio of 25 US FDA-approved ANDAs from Sandoz Inc. – In January 2018, the Company announced the acquisition of a portfolio of 25 US FDA-approved ANDAs, plus four pipeline ANDA’s that are pending FDA approval. CASI intends to select and commercialize certain products from the portfolio that have a unique market opportunity and cost-effective manufacturing in China and/or in the U.S.

Announced $50 million private placement to new and existing investors – In March 2018, the Company announced a $50 million private placement (common stock with accompanying warrants). Existing stockholders or their affiliates, including IDG-Accel China Growth Fund III, ETP Global Fund LP, and new stockholders including Robert W. Duggan, former Chairman and CEO of Pharmacyclics Inc., led the financing.

Upcoming presentations – CASI will provide a corporate update at the 2018 BIO International Convention (Boston, MA June 4-7).

Financial Results for the Quarter ended March 31, 2018

Cash Position: As of March 31, 2018, CASI had cash and cash equivalents of $49.9 million.

R&D Expenses: R&D expenses for the quarter ended March 31, 2018 were $1.7 million compared to $1.0 million in 2017, an increase of $0.7 million. The increase in R&D expenses primarily reflects costs associated with the technology transfer activities and regulatory support associated with the recently acquired ANDA portfolio, including non-cash intangible amortization expense of $0.3 million.

G&A Expenses: G&A expenses for the quarter ended March 31, 2018 were $1.3 million compared to $0.6 million in 2017. The increase in G&A over the prior year is primarily attributed to an increase in salary, benefits and recruitment expense in China, largely related to sales and marketing efforts to prepare for the anticipated launch of the Company’s first commercial product in China, as well as other general and administrative functions. There were also increased costs associated with business development, investor and public relations activities, and an increase in legal and other professional services fees during the 2018 period.

Net Loss: The Company reported a net loss of ($3.6 million), or ($0.05) per share, for the quarter ended March 31, 2018. This compares with a net loss of ($1.7 million), or ($0.03) per share for the first quarter of 2017. The increase in net loss is primarily due to costs associated the technology transfer activities and regulatory support for our ANDA portfolio, the write-off of approximately $0.7 million due to acquired in-process R&D primarily related to ANDAs not approved by the FDA, and increased costs associated with G&A functions, including employment costs for sales and marketing efforts, increased business development and investor relations activities, as well as other professional service fees.

On May 15, 2018 Bristol-Myers Squibb Company (NYSE:BMY) reported that it will take part in the UBS 2018 Global Healthcare Conference on Tuesday, May 22, 2018, in New York. Charles Bancroft, executive vice president, Chief Financial Officer and Head of Global Business Operations, will answer questions about the company at 11:30 a.m. ET (Press release, Bristol-Myers Squibb, MAY 15, 2018, View Source [SID1234526613]).

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Investors and the general public are invited to listen to a live webcast of the session at View Source An archived edition of the session will be available later that day.

On May 15, 2018 BerGenBio ASA (OSE: BGBIO), a clinical-stage biopharmaceutical company developing novel, selective AXL kinase inhibitors for multiple cancer indications, reported its results for the first quarter 2018 (Press release, BerGenBio, MAY 15, 2018, View Source [SID1234526612]). A presentation of the results by the Company’s management will take place today at 10.00 am CET in Oslo – details below.

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Richard Godfrey, Chief Executive Officer of BerGenBio, commented: "We are pleased with the progress made during Q1 2018. Patient recruitment into our global Phase II clinical proof-of-concept trials with bemcentinib is progressing well and we expect to deliver interim read-outs across all studies during 2018. Presentation of these results will be at major clinical congresses, including the annual American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) meeting in June. Coinciding with ASCO (Free ASCO Whitepaper), we will host a satellite event that will allow us to meet with our stakeholders and provide insights from KOLs and clinical experts on our selective AXL inhibitor bemcentinib as a potential cornerstone of cancer combination therapy. We believe that we will be able to demonstrate the significant potential of bemcentinib in cancer therapy by making tumour cells visible to the immune system and more susceptible to treatment with chemotherapy, targeted therapy and immuno-oncology drugs."

Highlights – First Quarter 2018

Good progress advancing bemcentinib’s proof-of-concept clinical development

First efficacy endpoint met in Phase II trial of bemcentinib/TARCEVA (erlotinib) combination in advanced lung cancer (NSCLC) patients
Recruitment completed in first stage of Phase II trial of bemcentinib in combination with KEYTRUDA in advanced breast cancer (TBNC) patients
Bemcentinib shown to be well tolerated in all patients enrolled across three combination trials with KEYTRUDA – data presented at ASCO (Free ASCO Whitepaper)-SITC 2018
Single agent therapy with bemcentinib led to increased immune activity in relapsed / refractory leukaemia (AML & MDS) patients – data presented at ASCO (Free ASCO Whitepaper)-SITC 2018
Post period

Private placement raising gross NOK 187.5 million from international institutional investors including from the USA, specialising in the biotechnology sector
Recruitment completed in the first stage of Phase II trial of bemcentinib in combination with KEYTRUDA in NSCLC patients
Preclinical data highlighting bemcentinib’s potential to reverse tumour immune suppression and enhance immune checkpoint inhibitor efficacy, presented at AACR (Free AACR Whitepaper) annual meeting
Publications describe the role of AXL signalling in, and potential therapeutic effect of selective AXL inhibition to counteract the progression of aggressive fibrosis in lung and liver diseases

Presentation and Webcast Details

A presentation by BerGenBio’s senior management team will take place at 10.00 am CET at:
Felix Konferansesenter, Bryggetorget 3, 0125 Oslo
The presentation will webcast live and the link will be available at www.bergenbio.com in the section Investors/ Financial Reports. A recording will be available shortly after the webcast has finished.

The results report and the presentation will be available at www.bergenbio.com in the section: Investors/ Financial Reports from 7:00 am CET the same day.