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]).

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"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.

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]).

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"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

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]).

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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.

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.

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.

1

• 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

3

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)