FENNEC PROVIDES BUSINESS UPDATE AND ANNOUNCES FISCAL YEAR 2018 FINANCIAL RESULTS

On March 13, 2019 Fennec Pharmaceuticals Inc. (NASDAQ:FENC; TSX: FRX), a specialty pharmaceutical company focused on the development of PEDMARKTM (a unique formulation of sodium thiosulfate (STS)) for the prevention of platinum-induced ototoxicity in pediatric patients, reported financial results for the fiscal year ended December 31, 2018 (Press release, Fennec Pharmaceuticals, MAR 13, 2019, View Source [SID1234534311]).

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"Throughout 2018 we were pleased to continue making progress on the advance of PEDMARKTM towards regulatory approval in the U.S. and EU," said Rosty Raykov, chief executive officer of Fennec. "Major accomplishments over the year included approval of our Pediatric Investigation Plan, confirmation of Pediatric Use Marketing Authorization eligibility in the EU and the initiation of our NDA in the U.S. This year, we remain focused on finalizing submissions in both the U.S. and EU and preparations for the potential launch of PEDMARKTM in 2020."

Recent Corporate Highlights and Upcoming Milestones

In December 2018, following a pre-submission meeting with the FDA, Fennec initiated a rolling New Drug Application (NDA) for PEDMARKTM in patients 1 month to <18 years of age with localized, non-metastatic, solid tumors. The NDA submission process is currently well underway. The Company has notified the FDA that the drug substance manufacturer for PEDMARKTM was recently acquired requiring a site transition for the commercial manufacturing site. The new facility of the acquiring company has large scale commercial capabilities and a proven and extensive track record of successful FDA inspections and product launches. As such, full submission is targeted for late 2019 to early 2020. If approved, Fennec expects a first commercial launch for PEDMARKTM in the second half of 2020.
In February 2019, Fennec announced a $12.5 million debt financing with Bridge Bank, which will be funded upon New Drug Application (NDA) approval of PEDMARKTM. The Company anticipates that its cash position of $22.8 million as of December 31, 2018 combined with the $12.5 million debt facility available upon approval of PEDMARKTM will be sufficient to fund the Company’s planned commercial launch of PEDMARKTM.
Fourth Quarter and Year End 2018 Financial Results

Cash Position – Cash and cash equivalents were $22.8 million as of December 31, 2018.
Research & Development (R&D) Expenses – R&D expenses were $1.7 million and $5.0 million, respectively, for the fourth quarter and year ended December 31, 2018, compared to $0.8 million and $1.9 million, respectively, for the same periods in 2017. The increase in R&D expenses were primarily due to the manufacturing and regulatory expenses associated with the preparation for regulatory approval and planned commercialization of PEDMARKTM.
General and administrative (G&A) Expenses – G&A expenses were $1.4 million and $5.4 million, respectively, for the fourth quarter and year ended December 31, 2018, compared to $1.6 million and $5.0 million, respectively for the same periods in 2017. Overall, there was a small decrease in non-cash equity compensation offset by small increases in administrative expenses.
Net Loss – Net losses for the fourth quarter and year ended December 31, 2018 of $3.0 million ($0.15 per share) and $9.9 million ($0.52 per share), respectively, compared to $2.3 million ($0.15 per share) and $7.0 million ($0.47 per share), respectively, for the same period in 2017.
Financial Update

The selected financial data presented below is derived from our unaudited condensed consolidated financial statements which were prepared in accordance with U.S. generally accepted accounting principles. The complete audited condensed consolidated financial statements for the period ended December 31, 2018 and management’s discussion and analysis of financial condition and results of operations will be available via www.sec.gov and www.sedar.com. All values are presented in thousands unless otherwise noted.

Audited Condensed Consolidated
Statement of Operations:
(U.S. Dollars in thousands except per share amounts)
Three Months Ended Twelve Months Ended
December 31, December 31, December 31, December 31,
2018 2017 2018 2017

Revenue $ - $ - $ - $ -

Operating expenses:
Research and development 1,723 886 5,008 1,936
General and administrative 1,382 1,629 5,401 5,015

Loss from operations (3,105 ) (2,515 ) (10,409 ) (6,951 )

Other (expense)/income
Unrealized gain/(loss) on derivatives - 206 167 (134 )
Other loss 6 (4 ) 6 (8 )
Net interest income 115 23 348 47
Total other (expense)/income, net 121 225 521 (95 )

Net income/(loss) $ (2,984 ) $ (2,290 ) $ (9,888 ) $ (7,046 )

Basic net income/(loss) per common share $ (0.15 ) $ (0.15 ) $ (0.52 ) $ (0.47 )

Diluted net income/(loss) per common share $ (0.15 ) $ (0.15 ) $ (0.52 ) $ (0.47 )

Fennec Pharmaceuticals Inc.
Balance Sheets
(U.S. Dollars in thousands)

December 31, 2018 December 31, 2017
Assets
Cash and cash equivalents $ 22,781 $ 28,260
Other current assets 169 141
Total Assets $ 22,950 $ 28,401

Liabilities and stockholders’ equity
Current liabilities $ 1,637 $ 1,477
Derivative liabilities - 167
Total stockholders’ equity 21,313 26,757
Total liabilities and stockholders’ equity $ 22,950 $ 28,401

Working Capital Fiscal Year Ended
Selected Asset and Liability Data: December 31, 2018 December 31, 2017
(U.S. Dollars in thousands)
Cash and cash equivalents $ 22,781 $ 28,260
Other current assets 169 141
Current liabilities excluding derivative liability (1,637 ) (1,477 )
Working capital $ 21,313 $ 26,924

Selected Equity:
Common stock & APIC $ 151,326 $ 146,882
Accumulated deficit (131,256 ) (121,368 )
Stockholders’ equity 21,313 26,757

About PEDMARKTM (sodium thiosulfate/STS)

Cisplatin and other platinum compounds are essential chemotherapeutic components for many pediatric malignancies. Unfortunately, platinum-based therapies cause ototoxicity in many patients, and are particularly harmful to the survivors of pediatric cancer.

Each year in the U.S. and Europe there is estimated that over 10,000 children with solid tumors are treated with platinum agents. The vast majority of these newly diagnosed tumors are localized and classified as low to intermediate risk in nature. These localized cancers may have overall survival rates of greater than 80%, further emphasizing the importance of quality of life after treatment. The incidence of hearing loss in these children depends upon the dose and duration of chemotherapy, and many of these children require lifelong hearing aids. There is currently no established preventive agent for this hearing loss and only expensive, technically difficult and sub-optimal cochlear (inner ear) implants have been shown to provide some benefit. Infants and young children at critical stages of development lack speech language development and literacy, and older children and adolescents lack social-emotional development and educational achievement.

STS has been studied by cooperative groups in two Phase 3 clinical studies of survival and reduction of ototoxicity: COG ACCL0431 and SIOPEL 6. Both studies are closed to recruitment. COG ACCL0431 enrolled one of five childhood cancers typically treated with intensive cisplatin therapy for localized and disseminated disease, including newly diagnosed hepatoblastoma, germ cell tumor, osteosarcoma, neuroblastoma, and medulloblastoma. SIOPEL 6 enrolled only hepatoblastoma patients with localized tumors. COG ACCL0431 final results were published in the Lancet Oncology. SIOPEL 6 final results were published in the New England Journal of Medicine.

Triumvira Announces Addition of Veteran VP of Regulatory Affairs, Presentations at CellCAN and TRI-CON Conferences, and Attendance at Bio-Europe Partnering

On March 13, 2019 Triumvira Immunologics, Inc., (Triumvira) a privately held biopharmaceutical company developing a novel platform for engineering T-cells to attack cancers, reported the appointment of Cynthia Molina as Vice President of Regulatory Affairs (Press release, Triumvira Immunologics, MAR 13, 2019, View Source [SID1234534313]). In this position, Cynthia will be responsible for global regulatory strategic development, oversight of regulatory submissions, and interactions with US and international governmental health authorities.

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Before joining Triumvira, Ms. Molina was most recently VP of Global Regulatory Affairs at Cell Medica, Inc in Houston, TX, for 6 years and accumulated 30 years of experience in multiple facets of product development for pharmaceutical, biotechnology, biologic, and in vitro diagnostic products, with a primary focus on regulatory affairs. In addition to regulatory affairs, her experience includes clinical and preclinical strategic planning, quality assurance, process development and manufacturing for oncology and infectious disease products. Ms. Molina has experience leading preparation of a variety of global regulatory submissions, including INDs, NDAs, BLAs, MAAs, global CTAs, 510Ks, PMAs, orphan and Fast Track submissions, and clinical study reports. She has held management positions at both large and small corporations, including Abbott Laboratories.

Paul Lammers, MD, MSc., President and CEO of Triumvira commented, "We are very pleased to welcome Cynthia to our management team. Her background and experience in regulatory affairs will enhance our interactions with regulatory agencies supporting our plan to bring multiple TAC product candidates into clinical development alone, as well as in collaboration with our business partners."

Additionally, several Triumvira executives are presenting at upcoming conferences. Triumvira Chief Technology Officer Donna Rill and Senior Vice President of R&D Dr. Andreas Bader will present at CellCAN’s Second Annual, Pan-Canadian Strategic Forum on Regenerative Medicine and Cell Therapy, to be held March 13-15, 2019, in Toronto, ON. Director of R&D and Head of Platform Development Dr. Christopher Helsen will present at the 26th International Molecular Med Tri-Conference on March 14, 2019, in San Francisco, CA. Topics of their presentations will be:

Characterizing products for GMP manufacturing: What, how and when? (Ms. D. Rill)

Optimizing cell and gene therapy manufacturing, Planning for Success – the path to commercialization (Ms. D. Rill)

TAC technology, its unique and novel MoA, preclinical data and considerations for biomarker analysis in our Phase I/II trial (Dr. A. Bader)

Development of a CD19-TAC therapy for a first in human phase I study (Dr. C. Helsen)

Triumvira also announces Joshua Carle, Vice President of Business Development, will attend the Bio-Europe Spring Partnering conference in Vienna, Austria from March 25–27, 2019. Mr. Carle and Triumvira are seeking to find out-licensing partners to accelerate our TAC pipeline development, collaborators with novel cancer cell surface antigens looking to expand into engineered T-cells, and to identify immuno-oncology assets that are complementary to our TAC T-cell platform.

CTI BioPharma Reports Fourth Quarter and Full Year 2018 Financial Results and Recent Highlights

On March 13, 2019 CTI BioPharma Corp. (Nasdaq: CTIC) reported financial results for the fourth quarter and full year ended December 31, 2018 (Press release, Idera Pharmaceuticals, FEB 27, 2019, View Source [SID1234533745]).

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"The Company is focused on advancing its development program of pacritinib for the treatment for myelofibrosis patients with severe thrombocytopenia in the United States and Europe in 2019," commented Adam R. Craig, M.D., Ph.D., President and Chief Executive Officer of CTI BioPharma. "Following the previously-announced completion of full enrollment in the U.S. Phase 2 PAC203 study of pacritinib in myelofibrosis patients with severe thrombocytopenia, the Company expects to meet with the U.S. Food and Drug Administration and report the determination of an optimal dose of pacritinib in mid-2019, with a Phase 3 registration study targeted to commence enrollment shortly thereafter. The Company further expects to report topline efficacy and safety data from the Phase 2 study by the end of 2019."

Expected 2019 Milestones

Determination of the optimal dose of pacritinib – mid-2019
Commence enrollment in Phase 3 study of pacritinib in myelofibrosis patients with severe thrombocytopenia (platelet count of less than 50,000 per microliter) – 3Q 2019
Reporting of top-line efficacy and safety data from PAC203 at a major medical meeting by the end of 2019
Fourth Quarter Financial Results

Total revenues for the three months and year ended December 31, 2018 were $14.1 million and $26.3 million, respectively, compared to $0.5 million and $25.1 million for the respective periods in 2017. The increase in total revenues for the fourth quarter in 2018 compared to the same period in 2017 is primarily due to the recognition of license and contract revenue in 2018 related to the achievement of a regulatory milestone under the license and collaboration agreement for PIXUVRI with Servier as well as the attainment of a worldwide net sales milestone of TRISENOX under the agreement with Teva Pharmaceutical Industries Ltd.

GAAP operating income was $0.2 million for the fourth quarter of 2018 and GAAP operating loss was $32.9 million for the year ended December 31, 2018, compared to GAAP operating loss of $13.7 million and $39.5 million for the respective periods in 2017. Operating income in the fourth quarter of 2018 as compared to operating loss in the same period in 2017 resulted primarily from the increase in license and contract revenue as mentioned above. Operating loss for the year ended December 31, 2018 as compared to 2017 resulted primarily from the decrease in selling, general and administrative expenses.

Net income attributable to common stockholders for the fourth quarter of 2018 was $0.8 million, or $0.01 for basic and diluted earnings per share, compared to net loss attributable to common stockholders of $14.3 million, or $(0.33) per share, for the same period in 2017. Net loss attributable to common stockholders for the twelve months ended December 31, 2018, was $29.4 million, or $(0.52) per share, compared to a net loss of $45.0 million, or $(1.24) per share, for the same period in 2017.

As of December 31, 2018, cash, cash equivalents and short-term investments totaled $67.0 million, compared to $43.2 million as of December 31, 2017.

Transgene – NEOVIVA project supporting the development of myvac™ awarded a €5.2 million grant from Bpifrance’s “Investments for the Future” programme

On March 13, 2019 Transgene (Euronext Paris:TNG), a biotech company that designs and develops virus-based immunotherapies against cancers and infectious diseases, reported that the NEOVIVA project was selected by the "Investments for the Future" (IFP) Programme (Programme d’Investissements d’Avenir) operated by Bpifrance for the development of an industrial sector focused on Transgene’s individualized immunotherapy platform myvac (Press release, Transgene, MAR 13, 2019, View Source [SID1234534268]). Under the agreement, the NEOVIVA project will receive €5.2 million over the five-year duration of the program from Bpifrance, of which Transgene will receive €2.6 million.

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Transgene holds the intellectual property of the myvac platform and is actively working to further develop this innovative technology in collaboration with its three French partners participating in the NEOVIVA project: HalioDx in Marseille, Traaser in Evry and the Curie Institute in Paris. The NEOVIVA project complements the already existing collaboration between Transgene and the Japanese company NEC focused on the clinical validation of an individualized immunotherapy based on artificial intelligence.

The goal of the NEOVIVA project is to develop and validate a manufacturing approach that would provide all solutions needed for the development of individualized immunotherapies. These are designed to stimulate the patient’s immune system to recognize and kill cancer cells by using their own genetic mutations. Two proof of concept clinical trials are being prepared in Europe and US for the treatment of HPV-negative head and neck cancers and ovarian cancer. These are expected to start in H2 2019.

"We would like to thank Bpifrance for its financial support which will accelerate the development of myvac. This funding is a further recognition of the myvac platform and reinforces our ambition to stand as a world leader in the development of individualized viral-vector (MVA) based immunotherapies. We believe the synergies among our partners and their respective teams are highly complementary and harness creativity which will lead to the success of our project. We are delighted to have access to our partners’ expertise through the NEOVIVA project," said Philippe Archinard, CEO and Chairman of Transgene.

Aïcha Douhou, Leader of the Health sector at the innovation department, Bpifrance commented: "We are delighted to support Transgene and its partners HalioDX, Traaser and the Curie Institute in their innovative approach for the design and development of individualized immunotherapy against solid tumors. We expect this project to further boost the development of Transgene bioproduction manufacturing unit for this individualized immunotherapy."

This consortium which combined bio-engineering, bioIT and a recognized know-how in viral vectorisation will be led by Transgene and each partner will contribute to the project as follows:

Transgene will be responsible for the project’s strategy and the clinical development plan. The product design for each patient will be generated in collaboration with NEC which will provide bioIT predictions needed to finalise the immunotherapy. Transgene will also manufacture the myvacTM clinical batches in its pilot manufacturing unit on the short timelines required for the treatment;
Traaser will be in charge of the interpretation of the genomic profiles determined by DNA sequencing. This will be used to facilitate, guide and secure the study of the individualized virotherapy artificial intelligence algorithms results;
HalioDx will study biomarkers to monitor and maximize the clinical efficacy of myvacTM with Immunogram, a high-tech clinical research platform that includes a suite of proprietary tests including Immunosign and the Immunoscore assay suite;
The Curie Institute (Immunotherapy Cancer Centre, led by Dr Amigorena) will contribute to the project in generating translational data and characterizing the therapy’s mechanism of action.

X4 Pharmaceuticals Completes Merger with Arsanis

On March 13, 2019 X4 Pharmaceuticals, Inc. (Nasdaq: XFOR, as of March 14, 2019), a clinical-stage biopharmaceutical company focused on the development of novel therapeutics for the treatment of rare diseases, reported the completion of its merger with Arsanis, Inc. (Nasdaq: ASNS, through March 13, 2019), effective as of March 13, 2019 (Press release, X4 Pharmaceuticals, MAR 13, 2019, View Source [SID1234534287]).

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"With the acquisition of synergistic R&D capabilities, X4 intends to establish a globally-recognized Center of Research Excellence to build upon our core science and technology, and to create long-term growth for a sustainable global rare disease business."

The holders of shares of X4 capital stock outstanding immediately prior to the merger received 0.5702 shares of Arsanis common stock in exchange for each share of X4 capital stock in the merger. On March 13, 2019, Arsanis is effecting a six-for-one reverse stock split. As a result of the reverse stock split, every six shares of Arsanis common stock outstanding following the merger, including the shares issued to the holders of shares of X4 capital stock in the merger, will be combined and reclassified into one share of Arsanis common stock. No fractional shares will be issued in connection with the reverse stock split. Instead, cash, based on the average closing price per share of Arsanis common stock on the Nasdaq Global Market on the 10 consecutive trading days prior to March 13, 2019, will be paid in lieu of fractions of shares.

Following the merger and the reverse stock split, the combined organization is expected to have approximately 6.7 million shares outstanding.

In connection with the merger, Arsanis will change its name to X4 Pharmaceuticals, Inc. The combined organization will commence trading on March 14, 2019 on the Nasdaq Capital Market under the symbol "XFOR".

The combined organization will operate under the leadership of X4’s management team prior to the merger, including Paula Ragan, Ph.D., President and Chief Executive Officer, and Adam S. Mostafa, Chief Financial Officer. The board of directors of the combined organization is comprised of six directors: four directors from the former X4 board, Michael S. Wyzga, Isaac Blech, Gary J. Bridger, Ph.D. and Dr. Ragan, and two directors from the former Arsanis board, David McGirr and René Russo, Pharm.D., BCPS. Mr. Wyzga is the new chairman of the board. The corporate headquarters of the combined organization is located in Cambridge, Massachusetts.

About WHIM Syndrome

WHIM syndrome is a primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene resulting in susceptibility to certain types of infections. WHIM is an abbreviation for the characteristic clinical symptoms of the syndrome: Warts, Hypogammaglobulinemia, Infections, and Myelokathexis. There is no approved therapy for the treatment of WHIM syndrome.