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.

PsiOxus Therapeutics Announces First-in-Human Dosing of their Second Gene Therapy Cancer Treatment.

On March 13, 2019 PsiOxus Therapeutics, Ltd. (PsiOxus), the gene therapy for cancer company, reported that it has started dosing NG-350A, an antibody based cancer gene therapy, to cancer patients (Press release, PsiOxus Therapeutics, MAR 13, 2019, View Source [SID1234534308]). The Phase 1 FORTITUDE study is being conducted at multiple cancer centers in the United States and will assess the safety, tolerability and preliminary antitumor activity of NG-350A in subjects with solid tumors.

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"Our approach is to systemically deliver a gene therapy vector to turn tumor cells into drug factories," stated Dr Brian Champion, the Chief Scientific Officer of PsiOxus. "The patient’s own tumor cells are used to produce the therapeutic antibody directly in the tumor micro-environment to treat their cancer." The monoclonal antibody delivered by NG-350A is a CD40 agonist, a potentially powerful activator of a patient’s immuno-inflammatory response. When delivered systemically, CD40 agonists have produced adverse events that may limit their use. NG-350A directs the production of the antibody locally within the tumor and PsiOxus is developing this agent to improve the potential for a tolerable and effective treatment. John Beadle, M.D., Chief Executive Officer of PsiOxus stated "PsiOxus is delighted to have our second cancer gene therapy in clinical development. We look forward to generating clinical data on this exciting new product to treat and benefit cancer patients."

FORTITUDE is an open-label, dose expansion, multicenter, Phase 1 study expected to enroll up to 125 patients across multiple clinical study sites in the United States. Phase 1a of the study will assess the safety, tolerability and dose of NG-350A and will enroll patients at study sites in the United States, led by Dr Aung Naing of the MD Anderson Cancer Center. Phase 1b of the study will evaluate NG-350A in expansion cohorts in subjects with specific metastatic or advanced tumors. The ClinicalTrials.gov identifier for the NG-350A study is: NCT03852511.

PsiOxus’ proprietary T-SIGn platform uses the enadenotucirev oncolytic virus as a vector to deliver combinations of therapeutic transgenes to carcinomas to fight cancer. All T-SIGn products are administered intravenously and are designed to selectively infect and replicate only in tumor cells. NG-348, the first T-SIGn virus to enter clinical trials, is licensed to Bristol-Myers Squibb.

CASI PHARMACEUTICALS ANNOUNCES EXCLUSIVE DISTRIBUTION PARTNER FOR MELPHALAN HYDROCHLORIDE FOR INJECTION (EVOMELA®) IN CHINA

On March 13, 2019 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. based pharmaceutical company with a platform to develop and accelerate the launch of pharmaceutical products and innovative therapeutics in China, U.S., and throughout the world, reported that the National Medical Products Administration (NMPA) has approved the Company’s Clinical Trial Application (CTA) allowing for a registration clinical trial to evaluate the efficacy and safety of vincristine sulfate LIPOSOME injection (MARQIBO) (Press release, CASI Pharmaceuticals, MAR 13, 2019, View Source [SID1234534306]).

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MARQIBO is a U.S. Food and Drug Administration (FDA)-approved product currently marketed in the U.S. by Spectrum Pharmaceuticals, Inc. (Spectrum), for the treatment of adult patients with Philadelphia chromosome–negative (Ph‒) acute lymphoblastic leukemia (ALL) in second or greater relapse or whose disease has progressed following two or more anti-leukemia therapies. CASI acquired greater China rights to this drug from Spectrum.

The Company is currently reviewing certain requirements provided by the Center for Drug Evaluation (CDE), a division of the NMPA, and upon satisfying those requirements, the Company will commence the registration trial for MARQIBO.

Wei-Wu He, Ph.D., CASI’s Executive Chairman commented, "Ph negative ALL is a rare but aggressive disease and while patient outcomes have vastly improved over the last three decades, patients continue to relapse and current salvage therapies are inadequate, particularly among the aging Chinese patient population. MARQIBO is the first and only liposome-encapsulated vincristine approved and marketed in the U.S. for second line treatment of adult Philadelphia chromosome-negative acute lymphoblastic leukemia and has been safely administered in patients since its U.S. approval in 2012. Receiving NMPA approval to conduct the registration trial in China with MARQIBO is an important milestone for CASI. This approval, along with the recent CTA approval for ZEVALIN and the fast track market approval of EVOMELA, further demonstrates CASI’s regulatory strength in working with the NMPA to advance products through the approval process."

Crinetics Pharmaceuticals Reports Fourth Quarter and Full Year 2018 Financial Results and Provides Corporate Update

On March 13, 2019 Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), a clinical stage pharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors, reported financial results for the quarter and full year ended December 31, 2018 and provided an update on its corporate activities and product pipeline (Press release, Crinetics Pharmaceuticals, MAR 13, 2019, View Source [SID1234534304]).

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"2018 was a transformative year for Crinetics, highlighted by our successful initial public offering in July and the progress of our pipeline programs for rare endocrine diseases," said Scott Struthers, Ph.D., Founder and Chief Executive Officer of Crinetics. "We have initiated our Phase 2 EVOLVE and EDGE clinical trials for our lead product candidate, CRN00808, in acromegaly, and will initiate our Phase 1 proof-of-concept trial for CRN01941 aimed at neuroendocrine tumors during the first half of the year."

Anticipated 2019 Activities

Continued enrollment of ACROBAT EVOLVE and ACROBAT EDGE Phase 2 clinical trials for CRN00808 in acromegaly. The EVOLVE trial is a double-blind, placebo-controlled, randomized withdrawal study designed to evaluate CRN00808 in patients with acromegaly that respond to somatostatin analog monotherapy. The EDGE trial is an open label exploratory study designed to evaluate CRN00808 in patients with acromegaly that do not respond completely to somatostatin analog monotherapy.

Initiation of Phase 1 clinical trial for CRN01941 for neuroendocrine tumors with topline data expected in late 2019 or early 2020.

Continued advancement of our pipeline programs for congenital hyperinsulinism and Cushing’s Disease.

Full Year 2018 Highlights

Filed IND with the FDA. In August 2018, Crinetics filed its Investigational New Drug (IND) application for CRN00808 in acromegaly.

Completed initial public offering. In July 2018, Crinetics closed its initial public offering of 6,900,000 shares of common stock at a public offering price of $17.00 per share. Net proceeds were approximately $106.5 million, after deducting underwriting discounts, commissions, and offering expenses.

Awarded up to $3.2 million in SBIR grants for congenital hyperinsulinism and acromegaly. In June 2018, Crinetics was awarded up to approximately $3.2 million in Small Business Innovation Research (SBIR) grants from the National Institute of Diabetes and Digestive and Kidney Diseases of the National Institutes of Health (NIH) to fund the continued research and development of its nonpeptide, oral somatostatin agonists for congenital hyperinsulinemias (CHI) and acromegaly. Crinetics will be eligible to receive funding for up to approximately $1.9 million for CHI and $1.3 million for acromegaly.

Fourth Quarter and Full Year 2018 Financial Results

Research and development expenses were $7.7 million and $24.5 million for the three months and full year ended December 31, 2018, respectively, compared to $2.6 million and $9.2 million for the same periods in 2017. The increases were primarily attributable to development and manufacturing activities for CRN00808 as well as the company’s clinical and preclinical programs and personnel costs.

General and administrative expenses were $2.6 million and $6.7 million for the three months and full year ended December 31, 2018, compared to $0.5 million and $1.9 million for the same periods in 2017. The increases were primarily due to costs to operate as a public company, as well as personnel costs to support the company’s growth.

Net loss for the three months ended December 31, 2018 was $8.5 million, compared to a net loss of $2.5 million for the three months ended December 31, 2017. For the full year ended December 31, 2018, the company’s net loss was $27.1 million compared to a net loss of $9.2 million for the full year ended December 31, 2017.

Cash, cash equivalents and short-term investments totaled $163.9 million as of December 31, 2018, compared with $14.2 million as of December 31, 2017.

As of February 28, 2018, the company had 24,095,485 common shares outstanding.

Financial Guidance

Crinetics expects that its cash, cash equivalents and investments will fund its current operating plan at least through the end of 2020.

Sophiris Bio Reports Fourth Quarter 2018 and Year-end Financial Results and Recent Corporate Highlights

On March 13, 2019 Sophiris Bio Inc. (NASDAQ: SPHS) (the "Company," "We" or "Sophiris"), a biopharmaceutical company developing topsalysin (PRX302), a first-in-class, pore-forming protein, in late-stage clinical trials for the treatment of patients with urological diseases, today reported financial results for the fourth quarter and full year 2018 and recent corporate highlights (Press release, Sophiris Bio, MAR 13, 2019, View Source [SID1234534299]).

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"During the past year, Sophiris has made important progress in determining the ideal method for topsalysin administration as a focal treatment for localized prostate cancer," said Randall E. Woods, president and CEO of Sophiris. "The Phase 2b study provided a compelling look at this potential, showing that a single administration of topsalysin led to a clinical response in 27% of patients, including a complete ablation of tumor in 16% of patients. Our investigators have also noted that a drug capable of delaying or obviating the need for radical therapy in nearly a third of the patient population with the potential safety profile that we have seen to date could be very attractive for patients with localized intermediate risk prostate cancer."

Allison Hulme, Ph.D., chief operating officer and head of R&D for Sophiris, added: "In recent weeks we have made significant progress on a key next step for the development of topsalysin: the development of a Phase 3 protocol for localized prostate cancer. Based on both the efficacy and the encouraging safety data from our Phase 2 program and the invaluable input from our Scientific Advisory Board who are supportive of our continuing development of topsalysin as a focal therapy, we have finalized a proposed Phase 3 design and initiated the process of obtaining formal scientific advice from the European Medicines Agency (EMA). We are on track to obtain feedback from the EMA in the first half of this year."

Woods added, "In addition to working with regulatory authorities to determine the potential path to market, we have been actively pursuing options to move topsalysin into the final stages of clinical development, and we currently believe that the ideal funding option will either be a potential development partnership or other strategic transaction. We have also re-prioritized some development activities enabling us to extend our cash runway through the third quarter of this year."

Recent Corporate Highlights:

Completion of Phase 2b trial in localized prostate cancer. In December, we provided top-line data from patients who received a second administration of topsalysin in the trial. Eleven of the 37 patients evaluated six months after receiving a single administration of topsalysin went on to receive a second administration. It was determined that both the first and the second administration of topsalysin continue to appear safe and well-tolerated by patients. There were no adverse events considered related to topsalysin that were experienced by more than one patient following the second administration. Importantly, a total of 27% of patients (10/37) demonstrated a clinical response six months following the first administration of topsalysin. Six of the ten clinical responders experienced a complete ablation of their tumor with no remaining tumor detected following a targeted biopsy of the treated area.
Preparations for Phase 3 trial in localized prostate cancer. We, along with our Scientific Advisory Board and our other scientific advisors, believe that the data generated in the single-administration portion of the Phase 2b prostate cancer study supports the advancement of the program into a single Phase 3 pivotal trial. Currently, we have initiated formal scientific advice with EMA and in the coming weeks plan to initiate dialog with the U.S. Food and Drug Administration on the single confirmatory Phase 3 design.
Completion of topsalysin drug substance manufacturing. We recently completed the manufacture of a batch of topsalysin drug substance, which is planned for use in the upcoming Phase 3 confirmatory study in localized prostate cancer.
Funding of future development of topsalysin. The management team remains focused on determining the best path forward for funding future clinical development for topsalysin and continues to engage in strategic discussions as part of this effort.
Financial Results:

At December 31, 2018, the Company had cash, cash equivalents and securities available-for-sale of $12.5 million and working capital of $8.2 million. The Company expects that its existing cash and cash equivalents will be sufficient to fund its operations through September 2019, assuming no new clinical trials are initiated and the Company continues operating as a going concern. The Company will require significant additional funding to advance topsalysin in clinical development. As of December 31, 2018, the outstanding principal balance of the Company’s term loan was $7 million on which the Company is currently making monthly interest only payments and is scheduled to begin making principal payments in April 2019.

For the three months ended December 31, 2018

The Company reported a net income of $5.5 million or $0.18 per share for the three months ended December 31, 2018, compared to net loss of $4.0 million or ($0.13) per share for the three months ended December 31, 2017. The net income for the three months ended December 31, 2018 was driven by a non-cash gain related to the revaluation of the Company’s warrant liability.

Research and development expenses

Research and development expenses were $2.0 million for the three months ended December 31, 2018, compared to $1.9 million for the three months ended December 31, 2017. The increase in research and development costs was primarily attributable to increases in the costs associated with manufacturing activities for topsalysin offset in part by a decrease in personnel related costs.

General and administrative expenses

General and administrative expenses were $0.9 million for the three months ended December 31, 2018, compared to $1.3 million for the three months ended December 31, 2017. The decrease in general and administrative expense was primarily due to decreases in personnel related expenses and marketing research activities.

Gain (loss) on revaluation of the warrant liability

Gain on revaluation of the warrant liability was $8.5 million for the three months ended December 31, 2018, compared to a loss on the revaluation of the warrant liability of $0.6 million for the three months ended December 31, 2017. The Company’s outstanding warrants may require it to pay the warrant holder cash under certain provisions of the warrant therefore the Company accounts for these warrants as a liability, and the Company is required to calculate the fair value of these warrants each reporting date. The non-cash gain reported for the three months ended December 31, 2018, was associated with a decrease in the fair value of the Company’s warrant liability from September 30, 2018 to December 31, 2018, which is calculated using a Black-Scholes pricing model. The decrease in the fair market value of the Company’s warrant liability was directly related to a decrease in the Company’s stock price from September 30, 2018 to December 31, 2018. Certain inputs utilized in the Company’s Black-Scholes fair value calculation may fluctuate in future periods based upon factors which are outside of the Company’s control. A significant change in one or more of these inputs used in the calculation of the fair value may cause a significant change to the fair value of the Company’s warrant liability, which could also result in a material non-cash gain or loss being reported in the Company’s consolidated statement of operations and comprehensive loss.

For the year ended December 31, 2018

The Company reported a net loss of $6.8 million or ($0.23) per share for the year ended December 31, 2018 compared to a net loss of $8.6 million or ($0.29) per share for the year ended December 31, 2017.

Research and development expenses

Research and development expenses were $10.7 million for the year ended December 31, 2018 compared to $6.2 million for the year ended December 31, 2017. The increase in research and development costs was primarily attributable to increases in the costs associated with manufacturing activities for topsalysin.

General and administrative expenses

General and administrative expenses were $4.4 million for the year ended December 31, 2018 compared to $5.7 million for the year ended December 31, 2017. The decrease in general and administrative expense was primarily due to decreases in non-cash stock-based compensation expense, marketing research activities and its personnel related costs.

Gain on revaluation of the warrant liability

Gain on revaluation of the warrant liability was $8.7 million for the year ended December 31, 2018 as compared to a gain of $3.3 million for the year ended December 31, 2017. The non-cash gain is associated with the change in the fair value of our warrant liability which was calculated using a Black-Scholes pricing model.