On March 12, 2018 ERYTECH Pharma (Euronext: ERYP – Nasdaq: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, reported its financial results for the year ended December 31, 2017 (Press release, ERYtech Pharma, MAR 12, 2018, View Source [SID1234524674]).

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"2017 was a transformative year for ERYTECH with the achievement of a number of important clinical, regulatory and financial objectives," said Gil Beyen, Chairman and CEO of ERYTECH. "We are extremely pleased with the positive outcome of our Phase 2b trial of eryaspase in second-line metastatic pancreatic cancer and the success of our two financing operations, which generated gross proceeds of approximately $226 million, and our resulting dual listing on Nasdaq and Euronext Paris. With the proceeds of these transactions, we believe we are well capitalized to advance our pipeline programs, most notably the pivotal Phase 3 clinical trial of eryaspase for the treatment of second-line metastatic pancreatic cancer. We met with the FDA to discuss the proposed design of this trial and also obtained similar feedback from the CHMP. We are also exploring the launch of proof of concept studies in first-line pancreatic cancer, and other solid tumor indications, beginning with triple negative breast cancer. Lastly, we resubmitted our MAA for potential approval of eryaspase (GRASPA) for the treatment of relapsed and refractory ALL in Europe. We expect our momentum to continue as we progress towards potential approval in ALL, and anticipate significant advances from other clinical and preclinical research programs in our pipeline."

Full Year and Recent Business Highlights

• In September 2017, ERYTECH reported the full data set from its open-label, multi-center, randomized Phase 2b trial evaluating eryaspase in combination with chemotherapy for the treatment of second-line metastatic pancreatic cancer. In the trial, patients treated with eryaspase achieved significant improvement in both overall survival (OS) and progression-free survival (PFS).

• The company is now preparing for the launch of a pivotal Phase 3 clinical trial in this same indication in the United States and Europe. Feedback on the design of the trial was obtained from the U.S. Food and Drug Administration (FDA) and the Commission for Human Medicinal Products (CHMP) of the European Medicines Agency (EMA). The proposed Phase 3 trial will evaluate eryaspase in combination with standard chemotherapy, compared to standard chemotherapy alone, in approximately 500 patients in the United States and Europe. The primary endpoint will be overall survival (OS). Enrollment of the first patient in this trial is expected in the third quarter of 2018.

• ERYTECH is also broadening the scope of eryaspase to first-line pancreatic cancer, as well as to other solid tumor indications. Recently, the company announced the selection of triple negative breast cancer (TNBC) as the next target indication for expanding the potential treatment scope of eryaspase. ERYTECH is preparing a Phase 2 proof-of-concept clinical trial for this indication and expects to enroll the first patient in this trial in the third quarter of 2018.

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• In October 2017, ERYTECH resubmitted its MAA for eryaspase (GRASPA) for the treatment of relapsed and refractory (R/R) ALL. The MAA resubmission includes the Phase 2/3 clinical trial data from children and adults with R/R ALL as well as additional data to address the outstanding questions of the CHMP. CHMP feedback is expected by the end of 2018.

• In October 2017, the company also identified the recommended dosing from its open-label, dose escalation Phase 1 clinical trial evaluating the safety of eryaspase in combination with chemotherapy for first-line treatment of adult ALL patients, conducted at five clinical sites across the United States. The steering committee reviewed the safety data of the three treatment cohorts and approved further development at a dose level of 100 U/kg. Based on these data and the clinical results obtained in Europe, the company is preparing to discuss next steps for its development in ALL with the FDA in the second quarter of this year.

• In December 2017, the company announced topline results from the Phase 2b clinical trial evaluating eryaspase for the treatment of AML. The open-label, randomized, multi-center clinical trial enrolled a total of 123 patients at 30 European sites. The trial did not meet its primary endpoint of OS. Patient selection is likely the most important factor: the median age of patients in the trial was 78 years, and the median duration of treatment was 5-6 weeks in both treatment arms.

• Throughout 2017, the company also advanced its preclinical pipeline programs:

• In spring 2017, the company presented preclinical data on its erymethionase product candidate at the 2017 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. Based on these preclinical studies, the company believes that erymethionase represents a promising new treatment approach against a broad range of cancers that rely on methionine metabolism.

• In September 2017, ERYTECH presented early preclinical data on its eryminase and erymethionase programs at the 13th International Congress of Inborn Errors of Metabolism (ICIEM). The findings from the research on eryminase, consisting of arginine deiminase encapsulated in red blood cells, showed a decrease in arginine levels in a disease model of arginase-1 deficiency, supporting a potential treatment approach for hyperargininemia. This study was conducted in collaboration with Queen’s University in Canada. The company’s preclinical data involving erymethionase, which is methionine- g-lyase encapsulated in red blood cells, showed lower homocysteine levels, supporting a potential treatment approach for homocystinuria. This study was conducted through a research collaboration with the Fox Chase Cancer Center (FCCC).

• ERYTECH also continues to explore its ERYMMUNE program, in which it intends to use its proprietary ERYCAPS platform to encapsulate tumor antigens within red blood cells as a potentially innovative approach to cancer immunotherapy. Preclinical proof-of-concept studies of ERYMMUNE are ongoing.
Full Year 2017 Financial Results

• ERYTECH’s key financial figures for the full year of 2017 compared with the same period of the previous year are summarized below:

In thousands of euros FY 2017 FY 2016
Revenues

— —
Other income

3,364 4,138

Total operating income

3,364 4,138

Research and development

(25,463 ) (19,720 )
General and administrative

(8,791 ) (6,808 )
Total operating expenses

(34,254 ) (26,528 )

Total operating loss

(30,889 ) (22,390 )

Financial income

539 558
Financial expenses

(3,183 ) (70 )
Financial income (loss)

(2,644 ) 488

Loss before tax

(33,533 ) (21,902 )

Income tax

3 (10 )

Net loss

(33,530 ) (21,913 )

Net loss for the full year 2017 was €33.5 million, compared to €21.9 million in 2016. The €11.6 million increase was primarily attributable to the increase in clinical and regulatory development expenses, related to the company’s ongoing clinical programs in ALL, AML and pancreatic cancer, the continuation of its regulatory initiatives in Europe and preparatory work related to additional clinical programs. R&D expenses also comprise pre-clinical developments on additional product candidates and the broadening of the ERYCAPS platform to include the potential development of immune therapies and enzyme-related therapies.

R&D expenses increased by €5.7 million. The increase included additional expenses in external provider services in relation with the company’s intensified clinical and regulatory activities, as well as the additional staffing for preclinical research and clinical development.

G&A expenses increased by €2.0 million, as a result of infrastructure developments to sustain the company’s growth.

Operating income decreased by €0.8 million, reflecting primarily a decrease in research tax credits.

The €2.6 million financial loss in 2017 was impacted by a €3.0 million currency exchange variation on the company’s cash position denominated in U.S. dollars and consolidated in euros.

•In April 2017, ERYTECH completed a €70.5 million ($82 million) private placement in which it issued 3,000,000 new ordinary shares.

•In November 2017, ERYTECH completed a global offering of its ordinary shares (including in the form of American Depositary Shares or ADSs) in the United States and Europe, with gross proceeds of approximately €124 million ($144 million). The offering resulted in the issuance of a total of 5,374,033 new ordinary shares, comprising 4,686,106 ADSs, at an offering price of $23.26 per ADS in the United States and 687,927 ordinary shares through a concurrent private placement in Europe and other countries outside of the United States and Canada at a price of €20.00 per ordinary share. Each ADS represents the right to receive one ordinary share. The underwriters exercised their overallotment option in full to purchase 702,915 additional ADSs and 103,189 additional ordinary shares in the global offering. Upon the consummation of the global offering, ERYTECH’s ADSs began trading on the Nasdaq Global Select Market on November 10, 2017.

•As of December 31, 2017, ERYTECH had cash and cash equivalents totaling €185.5 million (approximately $223 million), compared with €37.6 million on December 31, 2016. The net cash increase of €147.9 million was primarily the result of €177.4 million in net proceeds from the company’s financing activities in April and November 2017. Excluding the financing rounds, total cash utilization in 2017 was €26.4 million, comprised of a €24.7 million net cash utilization in operating activities and €1.7 million in capital expenditures.
Key News Flow and Milestones Expected over Next 12 Months

•Meeting with the FDA to discuss next steps in ALL

•Initiation of a pivotal Phase 3 clinical trial in second-line pancreatic cancer in Europe and the United States

•Initiation of a Phase 2 proof-of-concept clinical trial in first-line pancreatic cancer

•Initiation of a Phase 2 proof-of-concept clinical trial in TNBC

•Advance U.S. registration-directed activities for ALL

•CHMP feedback on MAA resubmission for GRASPA in R/R ALL

•Initiation of Phase 1 clinical trial with erymethionase

•Updates on preclinical pipeline programs

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Full Year Results 2017 Conference Call Details

As a reminder, ERYTECH management will hold a conference call and webcast on Tuesday, March 13th, 2018 at 01:30pm CET / 08:30am EDT to business highlights and full year 2017 financials. Gil Beyen, Chairman and CEO, Eric Soyer, CFO and COO and Iman El-Hariry, CMO will deliver a brief presentation, followed by a Q&A session.

The call is accessible via the below teleconferencing numbers, followed by the Conference ID#: 7997444#:

USA: +1 8338186807 United-Kingdom: +44 02031070289
Switzerland: +080 0836508 Germany: +49 06922224728
France: +33 0176748988 Belgium: +32 024003547
Sweden: +46 0856619361 Finland: +358 0972519310
Netherlands: +31 0207075547 Spain: +34 914142503
The webcast can be followed live online via the link: View Source

An archived replay of the call will be available for 7 days by dialing (US & Canada): +1 833 818 6807, (UK): +44(0) 203 107 0289, (France): +33(0)1 726 74 89 88, (Spain): +34 91412503, Conference ID # 7997444#

An archive of the webcast will be available on ERYTECH’s website, under the "Investors" section at investors.erytech.com

2018 Financial Calendar:

General Assembly Meeting of Shareholders: Friday, June 22, 2018 at 10:00am CET in Paris

Quarterly financial updates:

Business Update and Financial Highlights for the 1st quarter of 2018: May 14, 2018 (after U.S. market close), followed by a conference call and webcast on May 15, 2018 (2:30pm CET/8:30am ET)

Business Update and Financial Highlights for the 2nd quarter and first-half of 2018: September 17, 2018 (after U.S. market close), followed by a conference call and webcast on September 18, 2018 (2:30pm CET/8:30am ET)

On March 12, 2018 Immutep Limited (ASX: IMM; NASDAQ: IMMP) ("Immutep" or the "Company") has reported that it has entered into a clinical trial collaboration and supply agreement with Merck & Co., Inc., Kenilworth, NJ, USA (known as MSD outside the United States and Canada), through a subsidiary, to evaluate the combination of Immutep’s lead immunotherapy product candidate eftilagimod alpha ("efti" or "IMP321") with MSD’s anti-PD-1 therapy KEYTRUDA (pembrolizumab) in a new clinical trial that will evaluate the combination in several different solid tumours (Press release, Immutep, MAR 12, 2018, View Source [SID1234524702]).

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The planned Phase II clinical trial, referred to as TACTI-002 (Two ACTive Immunotherapies), will evaluate the safety and efficacy of this novel immunotherapy combination in patients with non-small cell lung cancer ("NSCLC"), head and neck cancer, or ovarian cancer. The TACTI-002 clinical trial will be a Phase II, Simon two-stage, non-comparative, open-label, single-arm, multicentre clinical study. Up to 120 patients across the three indications are planned to be treated in medical centres in Europe and the United States with the trial expected to commence in the second half of 2018.

"We are extremely pleased to be collaborating with MSD, one of the world’s leading immuno-oncology companies" said Marc Voigt, CEO of Immutep. "This clinical trial will evaluate a novel combination of two complementary immuno-oncology treatments in three cancer indications simultaneously, which could lead to more rapid drug development subject to successful outcomes. The data generated thus far from our ongoing TACTI-mel clinical trial has supported our hypothesis that there is a compelling therapeutic synergy in administering efti in combination with another immuno-oncology treatment. This new Phase II clinical trial significantly builds on the momentum we are delivering in the evaluation of efti in cancer, with two Phase I clinical trials and now two Phase II clinical trials in our program for 2018."

The trial combines two immuno-oncology treatments with complementary mechanisms of action, analogous to releasing the brakes and pushing the accelerator of the body’s immune system at two different positions in the cancer immunity cycle. Immutep’s efti is a first-in-class antigen presenting cell ("APC") activator which stimulates cancer-fighting T cells, while KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells.

Hutchison China MediTech has filed a 20-F – Annual and transition report of foreign private issuers [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 20-F, Hutchison China MediTech, 2018, MAR 12, 2018, View Source [SID1234524655]).

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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]).

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"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.

Aclaris Therapeutics has filed a 10-K – Annual report [Section 13 and 15(d), not S-K Item 405] with the U.S. Securities and Exchange Commission (Filing, 10-K, Aclaris Therapeutics, 2018, MAR 12, 2018, View Source [SID1234524656]).

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