FENNEC PHARMACEUTICALS ANNOUNCES $12.5 MILLION DEBT FINANCING WITH BRIDGE BANK

On February 4, 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) to be administered by infusion) for the prevention of ototoxicity induced by cisplatin chemotherapy in pediatric patients with solid tumors, reported a $12.5 million senior debt facility with the Life Sciences Group at Bridge Bank.

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The U.S. operating subsidiary of Fennec Pharmaceuticals Inc. entered into a Loan and Security Agreement with Bridge Bank, securing a $12.5 million debt facility with a maturity date of October 1, 2023, which will be funded upon New Drug Application (NDA) approval of PEDMARKTM in the U.S. The Company intends to use the proceeds from the loan to provide working capital for commercialization activities for PEDMARKTM upon NDA approval.

"We welcome the commitment of Bridge Bank to Fennec as this debt facility provides us with the option to access minimally dilutive capital at an attractive cost upon NDA approval. With this facility, combined with our existing cash position, we believe we have sufficient resources to launch PEDMARKTM," stated Rosty Raykov, Chief Executive Officer of Fennec.

"PEDMARK is the first agent to receive Breakthrough Therapy designation from the U.S Food and Drug Administration for prevention of cisplatin-induced hear loss in pediatric patients," said Justin McDonie, senior vice president of Bridge Bank’s Life Sciences Group. "Bridge Bank welcomes the opportunity to work alongside the Fennec Pharmaceuticals team and to provide flexible debt capital to support the launch of PEDMARK which addresses an unmet orphan condition."

About Bridge Bank

Bridge Bank, a division of Western Alliance Bank, Member FDIC, helps business clients realize their growth ambitions. Founded in 2001 in Silicon Valley, Bridge Bank offers a better way to bank for small-market and middle-market businesses across many industries, as well as emerging technology companies and the private equity community. Geared to serving both venture-backed and non-venture-backed companies, Bridge Bank delivers a broad scope of financial solutions including growth capital, equipment and working capital credit facilities, sustainable energy project finance, venture debt, treasury management, asset-based lending, SBA and commercial real estate loans, ESOP finance and a full line of international products and services. Based in San Jose, Bridge Bank has eight offices in major markets across the country along with Western Alliance Bank’s powerful array of specialized financial services. Western Alliance Bank is the primary subsidiary of Phoenix-based Western Alliance Bancorporation. One of the country’s top-performing banking companies, Western Alliance has ranked in the top 10 on the Forbes "Best Banks in America" list for four consecutive years, 2016-2019. For more information, visit bridgebank.com.

Alexion Reports Fourth Quarter and Full Year 2018 Results

On February 4, 2019 Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) reported financial results for the fourth quarter and full year of 2018 (Press release, Alexion, FEB 4, 2019, View Source [SID1234533046]). Total revenues for the full year of 2018 were $4,131.2 million, a 16 percent increase compared to 2017. The positive impact of foreign currency on total revenues year-over-year was less than one percent, or $5.9 million, inclusive of hedging activities. On a GAAP basis, diluted earnings per share (EPS) for the full year of 2018 was $0.35, an 82 percent decrease versus the prior year, inclusive of $1,183.0 million of expense related to the value of the in-process research and development assets acquired in 2018. Non-GAAP diluted EPS for the full year of 2018 was $7.92, a 35 percent increase versus the prior year.

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Total revenues in the fourth quarter were $1,128.8 million, a 24 percent increase compared to the same period in 2017. The negative impact of foreign currency on total revenues year-over-year was 1 percent, or $5.5 million, inclusive of hedging activities. On a GAAP basis, diluted EPS in the quarter was $(0.20), a 254 percent decrease versus the prior year, inclusive of $379.3 million of expense related to the value of the in-process research and development asset acquired in connection with our acquisition of Syntimmune. Non-GAAP diluted EPS for the fourth quarter of 2018 was $2.14, a 45 percent increase versus the fourth quarter of 2017.

"2018 was a transformational year for Alexion. I am proud of our many accomplishments, including entering rare neurology and making SOLIRIS for gMG Alexion’s best launch ever, completing the Phase 3 program and launching ULTOMIRIS for PNH in the U.S., delivering groundbreaking Phase 3 data and submitting regulatory filings for SOLIRIS in NMOSD and rebuilding our pipeline through disciplined business development, all while continuing to grow our base business," said Ludwig Hantson, Ph.D., Chief Executive Officer of Alexion. "As we look at 2019 and beyond, I am confident that this strong foundation positions us well to achieve our Four Pillars of growth, building durable, blockbuster franchises in PNH/aHUS, metabolics, neurology and FcRn."

Full Year 2018 Financial Highlights

Total net product sales were $4,130.1 million, compared to $3,549.5 million in 2017.
SOLIRIS (eculizumab) net product sales were $3,563.0 million, compared to $3,144.1 million in 2017, representing a 13 percent increase. SOLIRIS volume increased 16 percent year-over-year.
STRENSIQ (asfotase alfa) net product sales were $475.1 million, compared to $339.8 million in 2017, representing a 40 percent increase. STRENSIQ volume increased 47 percent year-over-year.
KANUMA (sebelipase alfa) net product sales were $92.0 million, compared to $65.6 million in 2017, representing a 40 percent increase. KANUMA volume increased 60 percent year-over-year.
GAAP cost of sales was $374.3 million, compared to $454.2 million in 2017. Non-GAAP cost of sales was $352.5 million, compared to $285.8 million in 2017.
GAAP R&D expense was $730.4 million, compared to $878.4 million in 2017. Non-GAAP R&D expense was $646.2 million, compared to $736.3 million in 2017.
GAAP SG&A expense was $1,111.8 million, compared to $1,094.4 million in 2017. Non-GAAP SG&A expense was $953.3 million, compared to $927.8 million in 2017.
GAAP income tax expense was $164.6 million, compared to $104.5 million in 2017. Non-GAAP income tax expense was $310.0 million, compared to $186.7 million in 2017.
GAAP diluted EPS was $0.35, inclusive of $1,183.0 million of expense related to the value of the in-process research and development assets acquired in 2018, compared to $1.97 in 2017. Non-GAAP diluted EPS was $7.92, compared to $5.86 in 2017.
GAAP net income includes total restructuring and related expenses of $50.7 million in 2018, compared to $286.5 million in 2017. GAAP cost of sales includes restructuring related expenses of $5.8 million in 2018, compared to $152.1 million in 2017, GAAP R&D expense includes $0.1 million in 2018, compared to $16.3 million in 2017 and GAAP SG&A expense includes $19.4 million in 2018, compared to $10.9 million in 2017.
Fourth Quarter 2018 Financial Highlights

Total net product sales were $1,128.5 million in the fourth quarter of 2018, compared to $909.4 million in the fourth quarter of 2017.
SOLIRIS net product sales were $976.7 million, compared to $791.9 million in the fourth quarter of 2017, representing a 23 percent increase. SOLIRIS volume increased 28 percent year-over-year.
STRENSIQ net product sales were $126.1 million, compared to $95.6 million in the fourth quarter of 2017, representing a 32 percent increase. STRENSIQ volume increased 43 percent year-over-year.
KANUMA net product sales were $25.7 million, compared to $21.9 million in the fourth quarter of 2017, representing a 17 percent increase. KANUMA volume increased 51 percent year-over-year.
GAAP cost of sales was $96.8 million, compared to $144.6 million in the fourth quarter of 2017. Non-GAAP cost of sales was $93.0 million, compared to $72.5 million in the fourth quarter of 2017.
GAAP R&D expense was $205.6 million, compared to $265.0 million in the fourth quarter of 2017. Non-GAAP R&D expense was $164.0 million, compared to $188.6 million in the fourth quarter of 2017.
GAAP SG&A expense was $318.7 million, compared to $296.4 million in the fourth quarter of 2017. Non-GAAP SG&A expense was $278.0 million, compared to $245.2 million in the fourth quarter of 2017.
GAAP income tax expense was $12.1 million, compared to $59.3 million in the fourth quarter of 2017. Non-GAAP income tax expense was $88.5 million, compared to $46.8 million in the fourth quarter of 2017.
GAAP diluted EPS was $(0.20), inclusive of $379.3 million of expense related to the value of the in-process research and development asset acquired in connection with our acquisition of Syntimmune, compared to $0.13 in the fourth quarter of 2017. Non-GAAP diluted EPS was $2.14, compared to $1.48 in the fourth quarter of 2017.
GAAP net income includes total restructuring and related expenses of $0.5 million in 2018, compared to $95.1 million in 2017. The 2017 statement of operations includes restructuring related expenses of $69.1 million in GAAP cost of sales, $15.3 million in GAAP R&D expense and $4.5 million in GAAP SG&A expense.
Research and Development

PHASE 3

ULTOMIRIS (ravulizumab-cwvz) – Paroxysmal Nocturnal Hemoglobinuria (PNH): In December 2018, the U.S. Food and Drug Administration (FDA) approved ULTOMIRIS for adults with PNH. Applications for approval in the European Union (EU) and Japan are currently under review. In addition, a Phase 3 study of ULTOMIRIS in children and adolescents with PNH is currently underway.
ULTOMIRIS – Atypical Hemolytic Uremic Syndrome (aHUS): In January 2019, Alexion announced positive topline results from a Phase 3 study of ULTOMIRIS in complement inhibitor naïve patients with aHUS. The study met its primary objective with 53.6 percent of patients demonstrating complete thrombotic microangiopathy (TMA) response. The safety profile of ULTOMIRIS was generally consistent with that seen in Phase 3 studies in PNH. No cases of meningococcal infection were observed. Based on these results, Alexion plans to file for regulatory approval in the U.S. in the first half of 2019 followed by the EU and Japan. In addition, a Phase 3 study of ULTOMIRIS in adolescents and children with aHUS is currently underway.
ULTOMIRIS – Subcutaneous: In late 2018, Alexion initiated a single, PK-based Phase 3 study of ULTOMIRIS delivered subcutaneously once per week to support registration in PNH and aHUS. Data are expected in early 2020.
ULTOMIRIS – Generalized Myasthenia Gravis (gMG): Alexion plans to initiate a Phase 3 study of ULTOMIRIS in gMG in the first quarter of 2019.
ULTOMIRIS – Neuromyelitis Optica Spectrum Disorder (NMOSD): Alexion plans to initiate a Phase 3 study of ULTOMIRIS in NMOSD.
SOLIRIS – NMOSD: Alexion has submitted applications in the U.S. and the EU for the approval of eculizumab for NMOSD. These submissions are based on previously announced results from the Phase 3 PREVENT study, in which 97.9 percent of patients with anti-aquaporin-4 (AQP4) auto antibody-positive NMOSD receiving SOLIRIS on top of stable standard-of-care therapy were free of relapse at 48 weeks compared to 63.2 percent of patients receiving placebo. Alexion also plans to file for regulatory approval in Japan later this year.
ALXN1840 (WTX101) – Wilson Disease: Enrollment is underway in a Phase 3 study of ALXN1840 (WTX101) in Wilson disease, a rare genetic disorder with devastating hepatic and neurological consequences. The study is now powered for superiority versus standard-of-care therapy. ALXN1840 is a first-in-class oral copper-binding agent with a unique mechanism of action to access and bind to serum copper and promote its removal from the liver.
PHASE 1/2

ALXN1830 (SYNT001): In the fourth quarter of 2018, Alexion announced the completion of its acquisition of Syntimmune. The acquisition added anti-FcRn antibody ALXN1830 (SYNT001) to the company’s clinical pipeline. ALXN1830 is currently in Phase 1b/2a development in patients with warm autoimmune hemolytic anemia (WAIHA) and in patients with pemphigus vulgaris (PV) or pemphigus foliaceus (PF). In 2019, the company plans to initiate two pivotal trials – one in WAIHA following successful completion of the current Phase 1b/2a study and one in gMG.
ALXN1810 – Subcutaneous: A Phase 1 study of subcutaneous ALXN1210 co-administered with Halozyme’s ENHANZE drug-delivery technology, PH20, is underway. Pending co-formulation data, this next-generation subcutaneous formulation will be called ALXN1810 and has the potential to further extend the dosing interval to once every two weeks or once per month.
ULTOMIRIS – Amyotrophic Lateral Sclerosis (ALS): Alexion plans to initiate a proof-of-concept study for ULTOMIRIS in ALS.
ULTOMIRIS – Primary Progressive Multiple Sclerosis (PPMS): Alexion plans to initiate a proof-of-concept study for ULTOMIRIS in PPMS.
Caelum Biosciences – CAEL101 – Light Chain (AL) Amyloidosis: In January 2019, Alexion entered into a collaboration with Caelum Biosciences to develop CAEL101 for AL amyloidosis, a rare systemic disorder that causes misfolded immunoglobulin light chain protein to build up in and around tissues, resulting in progressive and widespread organ damage. CAEL101 is a first-in-class amyloid fibril targeted therapy designed to improve organ function by reducing or eliminating amyloid deposits in patients with AL amyloidosis. In a Phase 1a/1b study, CAEL101 demonstrated improved organ function, including cardiac and renal function, in patients with relapsed and refractory AL amyloidosis.
PRE-CLINICAL

Dicerna – GalXC: Alexion is collaborating with Dicerna Pharmaceuticals to jointly discover and develop up to four subcutaneously delivered GalXC RNA interference (RNAi) candidates, currently in pre-clinical development, for the treatment of complement-mediated diseases.
Complement Pharma – CP010: Alexion is collaborating with Complement Pharma to co-develop CP010, a pre-clinical C6 inhibitor that has the potential to treat multiple neurological disorders.

2019 financial guidance assumes a GAAP effective tax rate of 13 to 15 percent and non-GAAP effective tax rate of 14 to 16 percent.

Alexion’s financial guidance is based on current foreign exchange rates net of hedging activities and does not include the effect of acquisitions, license and collaboration agreements, intangible asset impairments, litigation charges, changes in fair value of contingent consideration or restructuring and related activity outside of the previously announced activities that may occur after the issuance of this press release.

Conference Call/Webcast Information:

Alexion will host a conference call/audio webcast to discuss the fourth quarter and full year 2018 results today at 8:00 a.m. Eastern Time. To participate in the call, dial 866-762-3111 (USA) or 210-874-7712 (International), conference ID 5972194 shortly before 8:00 a.m. Eastern Time. A replay of the call will be available for a limited period following the call. The audio webcast can be accessed on the Investor page of Alexion’s website at: View Source

Syntrix Wins $3.4M NIH Grant to Conduct Phase 1/2 Trial of SX-682 in Myelodysplastic Syndrome

On February 4, 2019 Syntrix Pharmaceuticals reported that it has been awarded a three-year grant worth $3.4 million from the National Heart Lung and Blood Institute of the National Institutes of Health to assess its investigational CXCR1/2 inhibitor SX-682 in patients with low- and high-risk myelodysplastic syndrome (MDS) who had progression or were intolerant to prior therapy (Press release, Syntrix, FEB 4, 2019, View Source [SID1234553882]). The phase 1/2 clinical trial will be carried out in collaboration with researchers at the Moffitt Cancer Center led by Dr. Rami Kamrokji.

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"Patients with low-risk MDS have an expected median survival measured in years, but suffer from hematologic deficits and related symptoms that lead to transfusion dependence," said Syntrix’s President John Zebala. "Patients with high-risk MDS have debilitating cytopenias and borderline leukemia, and can have expected survival measured in months."

Only three drugs have received regulatory approval for MDS treatment, all with suboptimal response rates (<50%) and of limited durability (1-2 years). Once these agents fail in patients, there is no second-line treatment. Prognosis after failure is dismal, with median survival estimated at <6 months for higher-risk patients, and <18 months for lower-risk patients.

The Phase 1/2 clinical trial of SX-682 builds on groundbreaking discoveries by investigators at the Moffitt Cancer Center and the Albert Einstein College of Medicine who showed CXCR1/2 is pivotal in MDS and that its inhibition is a therapeutic strategy against the disease.

This Phase 1/2 trial in MDS patients will test the hypothesis that targeting CXCR1/2 with SX-682 will be efficacious in the disease by eliminating the MDS stem cells and bone marrow MDSCs. The FDA approved the protocol for the study in an IND sponsored by Syntrix.

ABOUT SX-682: SX-682 is a clinical-stage oral allosteric small-molecule inhibitor of CXCR1 and CXCR2 (CXCR1/2). Inhibiting both human receptors is believed essential. CXCR1/2 are a combined "master switch" of the immunosuppressive tumor microenvironment. Clinical studies in melanoma, breast, ovarian, prostate and colon cancer have shown a direct correlation between serum levels of CXCR1/2 ligands and disease progression. SX-682 has been validated in all major solid tumor models, where it exhibits mono-agent anti-tumor activity, blocks metastasis, depletes immunosuppressive myeloid cells, activates tumor killing by effector cells, reverses chemo-resistance, and potently synergizes with anti-CTLA-4 and anti-PD1. SX-682 is also being evaluated in solid tumors supported by the National Cancer Institute.

GeneQuantum Healthcare and MITRO Biotech Enter into Strategic Cooperation to Develop Next Generation Radionuclide Conjugates

On February 3, 2019 GeneQuantum Healthcare of Suzhou and MITRO Biotech of Nanjing reported that signed the "Strategic Cooperation Agreement on Next Generation Radionuclide Conjugates Development" in Nanjing. Dr. Gang Qin, President of GeneQuantum and Dr. Xinping Li, CEO of MITRO, attended the signing ceremony with their teams (Press release, GeneQuantum Healthcare, FEB 3, 2019, View Source [SID1234553991]).

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In pursuit of achieving mutually beneficial win-win cooperation, the two parties intend to integrate the leading bioconjugation platform of GeneQuantum and the unique advantages of MITRO in radiolabeling and molecular imaging technique to jointly develop next generation radionuclide conjugates for rapid and efficient companion diagnosis and precision target therapy on tumors.

"Since its establishment, GeneQuantum has been committed to building a differentiated innovation platform to solve the key challenges of the biopharmaceutical industry. Based on our proprietary ligase dependent conjugation technology (LDC), GeneQuantum has gradually established intelligent automatic conjugation system (iLDC) with concurrent fully integrated manufacturing process; combined with our comprehensive quality system which meets global standard, GeneQuantum can provide a holistic solution for the efficient development and manufacturing of any kinds of site-specific bioconjugates. MITRO, on the other hand, has a unique radionuclide production and labeling technology platform," said Dr. Gang Qin. "We are pleased to work with a distinctive and leading technology partner such as MITRO to develop next generation radionuclide conjugates for both diagnostic and therapeutic purposes, meeting the unmet clinical needs of cancer patients worldwide."

"MITRO focuses on the development and application of isotope labeling and molecular imaging technology, and is committed to establishing a biomedical innovation and transformation research platform, using the advanced isotope labeling and molecular imaging technology to develop new drugs," said Dr. Xinping Li. "We are glad to work with GeneQuantum Healthcare based on our mutual interest of advanced medicine. This cooperation will fully integrate the technical and resource advantages of GeneQuantum in site-specific bioconjugation and MITRO in isotope labeling and non-invasive molecular imaging technology. The collaboration will create an internationally competitive platform of radionuclide bioconjugates for medical use. This will contribute to the most efficient non-invasive diagnosis of cancer and precise targeted therapy, as well as a systemic solution of novel biomedical drug development."

AskAt Granted Use Patent for EP4 Receptor Antagonist for the Treatment of Cancer by the China National Intellectual Property Administration

On February 3, 2019 AskAt reported that received a Notice of Allowance dated January 18, 2019 from the China National Intellectual Property Administration (CNIPA) in connection with Application No. 201510982848.3, a use patent for AskAt’s EP4 receptor antagonist for the treatment of cancer (Press release, AskAt, FEB 3, 2019, View Source [SID1234535036]).

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