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 TESARO, Inc. (NASDAQ:TSRO), an oncology-focused biopharmaceutical company, reported a substantial expansion of its niraparib clinical development program (Press release, TESARO, MAR 27, 2017, View Source [SID1234518284]). Following the landmark results of the Phase 3 NOVA trial of niraparib, a comprehensive portfolio review, and the FDA approval of ZEJULA (niraparib) for patients with recurrent ovarian cancer, TESARO is implementing its plans to initiate registration strategies in the settings of metastatic ovarian, breast and lung cancers.

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"Based on the unprecedented results of the NOVA trial in women with recurrent ovarian cancer, we previously announced the expansion and refinement of our PRIMA and QUADRA trials to include a broad patient population, and in the case of PRIMA, eliminated the enrollment requirement for a biomarker selected tumor. With the approval of ZEJULA in hand, we will now begin to execute on our plans to pursue potentially transformational applications of niraparib in a broad range of metastatic cancer indications," said Mary Lynne Hedley, Ph.D., President and COO of TESARO. "We plan to expand our first-line ovarian cancer strategy to include a combination study that assesses the potential benefit of niraparib plus an anti-PD-1 antibody in the maintenance setting and initiate a clinical study of niraparib in combination with bevacizumab in patients with a first recurrence of ovarian cancer, with an intent to replace chemotherapy in this setting. We remain strongly committed to studying niraparib in the breast cancer setting and also expect to initiate a new trial of niraparib in combination with an anti-PD-1 antibody in women with metastatic triple-negative breast cancer. Finally, our goal to move niraparib into indications beyond ovarian and breast cancers encompasses plans to initiate a registration strategy for the first-line treatment of patients with metastatic non-small cell lung cancer that includes a phase 2 trial of niraparib in combination with an anti-PD-1 antibody in patients, regardless of PDL-1 tumor expression, and a phase 3 trial of niraparib in combination with an anti-PD-1 antibody in patients with high levels of PDL-1 tumor expression."

Niraparib is the only PARP inhibitor approved in the U.S. for the maintenance treatment of women with recurrent ovarian, fallopian or primary peritoneal cancers. This approval was based upon the results of a randomized, prospectively designed Phase 3 clinical trial where niraparib demonstrated a clinically meaningful increase in progression-free survival (PFS) in women with recurrent ovarian cancer following a response to platinum-based chemotherapy.

The BRAVO study is assessing niraparib in patients with breast cancer who are germline BRCA mutation carriers. This study is sponsored by TESARO and is being conducted by Breast International Group (BIG) and the European Organisation for Research and Treatment of Cancer (EORTC). Following a recent interim analysis of data by the independent data monitoring committee (IDMC), TESARO believes the BRAVO study is unlikely to produce data that is interpretable and therefore suitable for registration in this indication. A large number of patients in the chemotherapy control arm did not continue in the trial long enough to receive their first radiological scan, which is required to assess disease progression, resulting in an unusually high rate of censoring in the control arm. At this time, TESARO believes this is likely associated with the desire of patients who carry germline BRCA mutations to be treated with a PARP inhibitor rather than chemotherapy and the increased availability of PARP inhibitors. A final determination as to whether the planned enrollment in BRAVO should be completed will be made by the Steering Committee in the near term. No safety concerns have been noted by the IDMC with respect to niraparib. Approximately 5-10% of women with breast cancer are germline BRCA mutation carriers. TESARO expects the results and experience gained from the BRAVO trial to be supportive of the planned trial of niraparib in combination with an anti-PD-1 antibody in women with metastatic triple-negative breast cancer. Approximately 15-20% of women with breast cancer have triple negative breast cancer.

The expanded niraparib clinical development program now includes the following:

Ovarian Cancer

OvCa 3000-03-003: A Phase 3 clinical trial of niraparib in combination with an anti-PD-1 antibody in comparison to niraparib in first-line maintenance treatment of patients with advanced ovarian cancer who have responded to platinum induction therapy.

OvCa 3000-03-002: A Phase 3 clinical trial of niraparib in combination with bevacizumab in comparison to standard of care in patients with a first recurrence of ovarian cancer.

PRIMA: A Phase 3 clinical trial of niraparib in patients with advanced ovarian cancer who have responded to platinum induction therapy.

TOPACIO: A Phase 2 trial to evaluate the preliminary safety and efficacy of niraparib plus KEYTRUDA in patients with triple negative breast cancer and in patients with platinum resistant recurrent ovarian cancer being conducted by TESARO in collaboration with Merck.

QUADRA: A registration trial of niraparib for the treatment of patients with recurrent ovarian cancer who have received three or four regimens of therapy.

AVANOVA: An NSGO (Nordic Society of Gynaecological Oncology) Phase 1/2 trial (in collaboration with ENGOT) evaluating niraparib plus bevacizumab in patients with recurrent ovarian cancer.

Breast Cancer

TNBC 3000-03-004: A Phase 3 clinical trial of niraparib in combination with anti- PD-1 antibody in comparison to standard of care in patients with advanced triple negative breast cancer.

TOPACIO: A Phase 2 clinical trial to evaluate the safety and efficacy of niraparib plus KEYTRUDA in patients with triple negative breast cancer and patients with platinum resistant recurrent ovarian cancer being conducted by TESARO in collaboration with Merck.

Lung Cancer

Lung 3000-02-001: A Phase 2 clinical trial of niraparib in combination with an anti-PD-1 antibody in patients with advanced NSCLC and niraparib alone in patients with advanced squamous cell carcinoma of the lung.

Lung 3000-03-001: A Phase 3 clinical trial of niraparib in combination with an anti-PD-1 antibody in comparison to anti-PD-1 alone in patients with advanced NSCLC and high levels of PDL-1 tumor expression.

Prostate Cancer

Janssen Biotech has licensed rights to develop and commercialize niraparib specifically for patients with prostate cancer worldwide, except in Japan.

TESARO Investor Conference Call and Webcast
TESARO will webcast a conference call with investors and analysts today, March 27, 2017 at 4:30 PM ET. Investors and analysts may access this call by dialing (877) 853-5334 (U.S. and Canada) or (970) 315-0307 (international); no passcode is necessary. During this conference call, TESARO management will review the approval of ZEJULA and expanded niraparib development program, as well as answer questions from investors and analysts. This event will be webcast live and archived for 30 days, and may be accessed from the TESARO Investor Events and Presentations webpage at www.tesarobio.com.

About ZEJULA (Niraparib)
ZEJULA (niraparib) is an oral, once-daily poly(ADP-ribose) polymerase (PARP) 1/2 inhibitor that is indicated in the U.S. for the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy. In preclinical studies, ZEJULA concentrates in the tumor relative to plasma, delivering greater than 90% durable inhibition of PARP 1/2 and a persistent antitumor effect.

Select Important Safety Information
Myelodysplastic Syndrome/Acute Myeloid Leukemia (MDS/AML) was reported in patients treated with ZEJULA in all clinical studies. Discontinue ZEJULA if MDS/AML is confirmed.

Hematologic adverse reactions (thrombocytopenia, anemia and neutropenia) have been reported in patients treated with ZEJULA. Do not start ZEJULA until patients have recovered from hematological toxicity caused by previous chemotherapy (≤ Grade 1). Monitor complete blood counts weekly for the first month, monthly for the next 11 months of treatment, and periodically after this time.

Hypertension and hypertensive crisis have been reported in patients treated with ZEJULA. Monitor blood pressure and heart rate monthly for the first year and periodically thereafter during treatment with ZEJULA. Closely monitor patients with cardiovascular disorders, especially coronary insufficiency, cardiac arrhythmias, and hypertension.

Based on its mechanism of action, ZEJULA can cause fetal harm. Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment and for six months after receiving the final dose. Because of the potential for serious adverse reactions in breastfed infants from ZEJULA, advise a lactating woman not to breastfeed during treatment with ZEJULA and for one month after receiving the final dose.

In clinical studies, the most common adverse reactions included: thrombocytopenia, anemia, neutropenia, nausea, constipation, vomiting, abdominal pain/distension, mucositis/stomatitis, diarrhea, fatigue/asthenia, decreased appetite, headache, insomnia, nasopharyngitis, dyspnea, rash and hypertension.

Please see full Prescribing Information for additional Safety Information.

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 23, 2017 GlobeImmune, Inc. reported it has entered into a definitive purchase agreement for the sale of 12,835,490 shares of its common stock to NantCell, Inc., a member of the NantWorks ecosystem of companies (Press release, GlobeImmune, MAR 23, 2017, View Source [SID1234528328]). In connection with the sale of its common stock, NantCell has agreed to pay GlobeImmune $100,000 in cash and issue to GlobeImmune 200,000 shares, an estimated $2.0 million in value, of NantCell’s common stock.

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"We are pleased to enter into this strategic agreement with NantCell," said Timothy C. Rodell, M.D., Chairman of GlobeImmune. "We believe that, with NantCell’s resources, this combination could leverage GlobeImmune’s Tarmogen technology platform to accelerate development of our programs in oncology and infectious disease," continued Dr. Rodell.

"GlobeImmune has continued to impact the oncology industry by putting innovative drug candidates forward to treat cancer and infectious diseases," said Dr. Patrick Soon-Shiong, founder of NantCell. "In our mission to win the war against cancer, our team welcomes opportunities to engage with companies who are revolutionizing cancer care and treatments. We are looking forward to leveraging the expertise of GlobeImmune."