On March 27, 2017 Sophiris Bio Inc. (NASDAQ: SPHS) (the "Company" or "Sophiris"), a clinical late-stage biopharmaceutical company developing topsalysin (PRX302) for the treatment of patients with urological diseases, reported fourth quarter and full year 2016 financial results and key corporate highlights (Press release, Sophiris Bio, MAR 27, 2017, View Source [SID1234518395]).

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Key Corporate Highlights:

Advanced Topsalysin (PRX302) in Clinical Development for Localized Prostate Cancer. During 2016, the Company reported successful results with topsalysin in a completed Phase 2a study for the focal treatment of localized prostate cancer. In 2017, the Company continued the development of topsalysin for the focal treatment of localized prostate cancer with the initiation of a Phase 2b clinical study.

Initiated Phase 2b Localized Prostate Cancer Study. In March 2017, the Company initiated its Phase 2b open-label localized prostate cancer study with investigational sites in both the UK and US. The primary objective of the study is safety and tolerability of an injection of topsalysin and the key efficacy variable is focal ablation of a clinically significant lesion on biopsy after six months. Approximately 40 patients with localized prostate cancer are expected to be enrolled in the study and patient screening has begun

Previously obtained multi-parametric magnetic resonance imaging (mpMRI) of tumor lesions in each patient’s prostate, mapped to real-time three dimensional ultrasound will be used in the study to guide an intrasprostatic injection of topsalysin to treat a single, histological-proven, clinically significant lesion area in each patient’s prostate.

Six months following treatment with topsalysin, a targeted biopsy of the treated area will be conducted. The Company expects to receive the six-month biopsy data for all patients in late 2017 or early 2018. Based upon the results of the 6-month biopsy, the study includes an option to potentially re-treat the targeted lesion area with a second dose of topsalysin, with a targeted biopsy to occur six months following the second dose. In order to be eligible for a second dose, the patient cannot have experienced a significant adverse event attributable to topsalysin or the dosing procedure from the first dose and the patient will need to have had a clinical response from the first dose but still have the presence of a clinically significant lesion area. The Company expects to have final biopsy data on all patients who receive a second dose by the third quarter of 2018.

Reported Successful Results from a Phase 2a Localized Prostate Cancer Study. In June 2016, the Company announced successful results from its completed Phase 2a study of topsalysin in the focal treatment of localized prostate cancer. Biopsy data at six months following treatment in 18 patients showed that topsalysin demonstrated the ability to ablate tumor cells in over half of the patients and two patients experienced complete ablation of their targeted tumor with no histological evidence of any tumor remaining at six months.

Reported Positive Data at Important Urological Meeting. The Company presented positive data from its Phase 3 clinical trial of topsalysin as a treatment for the symptoms of benign prostatic hyperplasia ("BPH") as a late breaking poster at the 111th American Urological Association Annual Meeting. A copy of the poster is available on the Company’s website at www.sophirisbio.com.

Improved Financial Profile to Support Topsalysin Clinical Plans. During 2016, the Company raised net proceeds of $27.4 million and $7.0 million through two financing transactions. As of December 31, 2016, the Company had cash, cash equivalents and securities available-for-sale of $29.0 million and working capital of $27.8 million. The Company expects that its cash, cash equivalents and securities available-for-sale will allow the Company to operate through the end of 2018.
"During 2016, Sophiris has made significant clinical development progress in advancing topsalysin, a novel and first-in-class targeted biologic," said Randall E. Woods, president and CEO of Sophiris. "We believe that topsalysin may be an effective treatment to address significant unmet medical needs in two major commercial markets. Topsalysin could potentially provide a new intermediate treatment that may delay or even obviate the need for more radical treatment approaches in both localized prostate cancer as well as BPH. Topsalysin also has the potential to maintain or improve a patient’s quality of life post-treatment while at the same time remaining attractive to payors."

Financial Results:

At December 31, 2016, the Company had cash, cash equivalents and securities available-for-sale of $29.0 million and working capital of $27.8 million. The Company expects that its cash and cash equivalents will be sufficient to fund its operations through the end of 2018. The Company is currently not planning on pursuing a second Phase 3 trial in BPH, unless the Company can secure a development partner to fund such new clinical trial or the Company obtains other financing.

For the three months ended December 31, 2016

The Company reported a net loss of $0.5 million ($0.02 per share) for the three months ended December 31, 2016 compared to a net loss of $2.5 million ($0.15 per share) for the three months ended December 31, 2015.

Research and development expenses were $1.0 million for the three months ended December 31, 2016, compared to $1.7 million for the three months ended December 31, 2015. The decrease in research and development expenses were primarily attributable to a decrease in the costs associated with the Company’s Phase 3 PLUS-1 clinical trial which was completed in November 2015 and its Phase 2a proof of concept clinical trial for localized prostate cancer which was completed in June 2016.

General and administrative expenses were $1.2 million for the three months ended December 31, 2016 compared to $0.7 million for the three months ended December 31, 2015. The increase in general and administrative expenses was primarily due to an increase in personnel related costs.

Gain on revaluation of the warrant liability was $1.6 million for the three months ended December 31, 2016. This non-cash gain was associated with the change in the fair value of the Company’s warrant liability from September 30, 2016 to December 31, 2016.

For the 12 months ended December 31, 2016

The Company reported a net loss of $11.2 million ($0.49 per share) for the twelve months ended December 31, 2016 compared to a net loss of $14.2 million ($0.84 per share) for the twelve months ended December 31, 2015.

Research and development expenses were $3.5 million for the twelve months ended December 31, 2016 compared to $9.9 million for the twelve months ended December 31, 2015. The decrease in research and development costs were primarily attributable to a decrease of $5.9 million in the costs associated with the Company’s completed Phase 3 PLUS-1 clinical trial of topsalysin for the treatment of BPH and to a lesser extent a decrease in costs associated with the Company’s Phase 2a proof of concept clinical trial for localized prostate cancer which commenced in May 2015 and was completed in June 2016.

General and administrative expenses were $6.8 million for the twelve months ended December 31, 2016 compared to $3.6 million for the twelve months ended December 31, 2015. The increase is primarily due to the inclusion of $1.6 million in offering costs which were allocated to the warrants issued in connection with the Company’s offerings which closed in May and August of 2016. The increase, to a lesser extent, is due to an increase in personnel related costs of $0.8 million and legal, accounting, consulting and professional fees of $0.7 million.

Loss on revaluation of the warrant liability was $0.3 million for the twelve months ended December 31, 2016. The non-cash loss was associated with the change in the fair value of the Company’s warrant liability.

Loss on early extinguishment of debt was $0.2 million for the twelve months ended December 31, 2016. This consists of the final payment and a prepayment fee which was offset by the Company’s unamortized debt premium resulting from the payoff of its loan with Oxford.

On March 27, 2017 Xenetic Biosciences, Inc. (NASDAQ:XBIO) ("Xenetic" or the "Company"), a clinical-stage biopharmaceutical company focused on the discovery, research and development of next-generation biologic drugs and novel orphan oncology therapeutics, reported an update to the progress of its patent portfolio development (Press release, Xenetic Biosciences, MAR 27, 2017, View Source [SID1234537803]).

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Since 2014, the Company has broadened its patent portfolio geographically, into key markets including areas of Europe, Asia and North America, including the United States. Current patents include the manufacturing and conjugation chemistry of Xenetic’s PolyXen platform technology for creating proprietary, next-generation protein therapeutics by attaching polysialic acid ("PSA"), a biodegradable polymer found in living systems, to existing protein or peptide therapeutics, which can improve their pharmacological properties. Xenetic has also successfully obtained patent coverage for its proprietary polysialated protein therapeutics in the United States and globally.

M Scott Maguire, Xenetic’s Chief Executive Officer, stated, "Our focus remains on delivering shareholder value by leveraging our PolyXen platform technology with additional collaborations like our deal with Shire PLC on their SHP656 program, and advancing our lead product in clinical development, XBIO-101, for the treatment of endometrial cancer and triple negative breast cancer. We have worked diligently to continue to build our robust patent estate which includes 200 issued patents in order to provide substantial coverage for potentially safe and well tolerated therapy options for patients across a variety of indications."

Xenetic’s IP for its PolyXen technology platform provides protection on average into 2027 – 2029.

About PolyXen

PolyXen is a patent-protected platform technology for creating proprietary, next-generation protein therapeutics by attaching polysialic acid ("PSA"), a biodegradable polymer found in living systems, to existing protein or peptide therapeutics, which can improve their pharmacological properties.

Attachment of PSA ("polysialylation") to a therapeutic increases its apparent size, which reduces systemic clearance rates, while shielding the protein from other degradation pathways. The PolyXen platform permits optimization of a target therapeutic’s pharmacological properties, by controlling the amount, size, and sites of attachment of the PSA polymers.

In clinical and preclinical settings, therapeutic proteins polysialylated with the PolyXen platform have been shown to have extended circulating half-life, improved thermodynamic stability and resistance to proteases, while retaining pharmacological activity. Numerous human clinical trials to date have shown no evidence of PSA- induced immunogenicity.

On March 27, 2017 TESARO, Inc. (NASDAQ: TSRO), an oncology-focused biopharmaceutical company, reported that the U.S. Food and Drug Administration (FDA) has approved ZEJULA (niraparib), an oral, once-daily poly(ADP-ribose) polymerase (PARP) inhibitor, for the maintenance treatment of women with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response (CR or PR) to platinumbased chemotherapy (Press release, TESARO, MAR 27, 2017, View Source [SID1234518315]). ZEJULA is the first PARP inhibitor to be approved by the FDA that does not require BRCA mutation or other biomarker testing. TESARO anticipates launching ZEJULA in the United States in late April.

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Multimedia materials accompanying this release are available at: View Source

ZEJULA is the only PARP inhibitor that has demonstrated a clinically meaningful increase in progression-free survival (PFS) in women with recurrent ovarian cancer, regardless of BRCA mutation or biomarker status, in a randomized, prospectively designed Phase 3 clinical trial. ZEJULA offers oral, once-daily dosing, enabling convenient administration for maintenance treatment. FDA approval of ZEJULA is based upon data from the international Phase 3 ENGOTOV16/NOVA trial, a double-blind, placebo-controlled study that enrolled 553 patients with recurrent ovarian cancer who had achieved either a PR or CR to their most recent platinum-based chemotherapy. Approximately two-thirds of study participants did not have germline BRCA mutations. Progression in the NOVA study was determined by robust, unbiased, blinded central review, to be the earlier of radiographic or clinical progression. ZEJULA significantly increased PFS in patients with and without germline BRCA mutations as compared to control. Treatment with ZEJULA reduced the risk of disease progression or death by 74% in patients with germline BRCA mutations (HR 0.26) and by 55% in patients without germline BRCA mutations (HR 0.45). The magnitude of benefit was similar for patients entering the trial with a PR or a CR.

The most common grade 3/4 adverse reactions to ZEJULA in the NOVA trial included thrombocytopenia (29%), anemia (25%), neutropenia (20%), and hypertension (9%). The majority of hematologic adverse events were successfully managed via dose modification, and discontinuation of therapy due to thrombocytopenia, neutropenia and anemia occurred in 3.3%, 1.9% and 1.4% of patients, respectively. Following dose adjustment based on individual tolerability, the incidence of grade 3/4 thrombocytopenia was low; approximately

On March 27, 2017 EUSA Pharma (EUSA), a specialty pharmaceutical company with a focus on oncology and oncology supportive care, and Apeiron Biologics reported that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the approval of dinutuximab beta for use in the treatment of high-risk neuroblastoma (Press release, EUSA Pharma, MAR 27, 2017, View Source [SID1234527665]).

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The marketing authorisation application included data developed from multiple clinical trials across Europe that included over 1,000 patients receiving dinutuximab beta. The clinical development was led by the SIOPEN academic neuroblastoma group and supported by Apron Biologics2. EUSA Pharma holds the exclusive global rights to dinutuximab beta.

Lee Morley, EUSA Pharma’s Chief Executive Officer, said, "This positive CHMP opinion is an important milestone for EUSA as we work to bring dinutuximab beta to children suffering from the high risk form of the devastating disease, neuroblastoma. This cancer is responsible for up to 10% of childhood tumors, and with treatment options limited we are working hard to make this life saving therapy available to children around the world. Following this positive opinion in Europe, we plan to submit dinutuximab beta for approval in the United States in the coming year."

Dr. Hans Loibner, Apeiron Biologic’s Chief Executive Officer, said, "We are delighted with the CHMP positive opinion for dinutuximab beta, which follows our extensive development work with a number of partners, in particular the SIOPEN group. Dinutuximab beta represents an important potential treatment in an area of significant unmet need, and we look forward to working with EUSA to make this product available around the world."

Dinutuximab beta is currently used in Europe under a managed access program as part of treatment regimens for high-risk neuroblastoma. Following the CHMP positive opinion, the European Commission will now issue a formal decision on approval, and if approved dinutuximab beta will be indicated for use in the 28 countries of the European Union in children aged 12 months and above, who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as patients with history of relapsed or refractory neuroblastoma, with or without residual disease. In patients with a history of relapsed/refractory disease and in patients who have not achieved a complete response after first line therapy, dinutuximab beta should be combined with interleukin-2 (IL-2). Prior to the treatment of relapsed neuroblastoma, any actively progressing disease should be stabilised by other suitable measures.

– Ends –

About dinutuximab beta and neuroblastoma
Neuroblastoma is an orphan oncology condition with significant unmet medical need. It accounts for up to 10% of childhood tumors and affects approximately 1,200 children in Europe each year. Dinutuximab beta is currently used extensively across Europe under a managed access scheme and is included in a number of treatment protocols for high-risk neuroblastoma.

Dinutuximab beta is an anti-GD2 monoclonal antibody that significantly improves event-free and overall survival in children with high-risk neuroblastoma, with a favorable safety profile compared to other immunotherapies. Dinutuximab beta forms an important part of treatment regimens for high-risk neuroblastoma and dinutuximab beta’s novel features offer the potential for further development to expand its current role. Dinutuximab beta has orphan drug designation in the US and EU, and EUSA plans to file the product for approval in the United States in 2017.

On March 27, 2017 ArQule, Inc. (Nasdaq: ARQL) reported that its partner, Kyowa Hakko Kirin, reported top-line results of the JET-HCC Phase 3 trial of tivantinib in Japan, and that the trial did not meet its primary endpoint of progression free survival (PFS) (Press release, ArQule, MAR 27, 2017, View Source [SID1234518273]).

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JET-HCC is a randomized, double-blind placebo-controlled study that enrolled approximately 190 Japanese patients with c-Met diagnostic-high inoperable hepatocellular carcinoma (HCC) with a history of prior sorafenib therapy, to evaluate the efficacy and safety of tivantinib.

The primary endpoint of the trial is PFS, and the top-line results did not show a significant difference in PFS between the tivantinib group and the placebo group. There were no new safety issues observed in the trial.

The details of the study results will be presented in an upcoming scientific forum.

"I would like to thank our partner, Kyowa Hakko Kirin, and all the participants in their study," said Paolo Pucci, Chief Executive Officer of ArQule. "The results are disappointing as there is a need for a second-line HCC therapy in Japan."

About Hepatocellular Carcinoma (HCC)

Liver cancer is the sixth most common cancer globally with 782,000 new cases in 2012 and is the second most common cause of cancer-related death with 745,000 deaths in 2012.1 HCC accounts for about 90 percent of primary liver cancers.2 Cirrhosis, chronic hepatitis B and C and smoking are recognized worldwide as factors increasing the risk of HCC.2

About Tivantinib (ARQ 197)

Tivantinib (ARQ 197) is an orally administered, small molecule inhibitor of the c-Met receptor tyrosine kinase ("MET") and its biological pathway. Kyowa Hakko Kirin and ArQule entered into a license agreement for exclusive rights to the development and sale of tivantinib in Japan and certain parts of Asia (China, Korea, and Taiwan) on April 12, 2007.