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 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 28, 2016 Kitov Pharmaceuticals (NASDAQ and TASE: KTOV), reported that TyrNovo Ltd., a company majority-owned by Kitov, will present preclinical data on TyrNovo’s anti-tumor resistance drug candidate NT219 in a poster session at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2017, to be held April 1-5, at the Walter E. Washington Convention Center in Washington, D.C (Press release, Kitov Pharmaceuticals , MAR 27, 2017, View Source [SID1234518293]).

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Dr. Hadas Reuveni, TyrNovo’s founder and chief technology officer, will discuss recent promising results demonstrating NT219’s efficacy in patient-derived xenograft models (PDX) in mice.

NT219 is a small molecule that blocks two feedback pathways highly involved in drug resistance, IRS and STAT3. Combined treatment of NT219 with drugs targeting EGFR, such as Tagrisso and Erbitux, overcame acquired resistance of colon, lung, and head-and-neck cancers in PDX models.

Details on the poster presentations are as follows:

Title: Comprehensive high-throughput screen for combination therapies to block acquired resistance to targeted drugs

Session: PO.ET04.05 – Reversal of Drug Resistance

Location: Section 6

When: Monday, Apr 3, 2017, 8:00 AM – 12:00 PM ET

Poster Board Number: 1190 / 5

About TyrNovo

TyrNovo Ltd. is developer of novel small molecules in the oncology therapeutic field which is majority owned by Kitov Pharmaceuticals (NASDAQ/TASE: KTOV). TyrNovo is developing NT219, a potential oncology combination product. NT219 is a small molecule that presents a new concept in cancer therapy. In combination with various approved oncology drugs, NT219 demonstrated potent anti-tumor effects and increased survival in various cancer models, including sarcoma, melanoma, pancreatic, lung, ovarian, head & neck, prostate and colon cancers. Its mechanism of action is through the prevention of acquired resistance in tumors and by regression of resistant tumors. For more information on TyrNovo please visit View Source

On March 27, 2017 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3 stage biopharmaceutical company focused on discovering and developing novel small molecule drug candidates to treat cancer, with a primary focus on Myelodysplastic Syndromes, reported a corporate update and reported financial results for the year ended December 31, 2016 (Press release, Onconova, MAR 27, 2017, View Source [SID1234518287]).

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Latest on Onconova Therapeutics’ Cancer Pipeline, book your free 1stOncology demo here.

"We are pleased to report that our lead clinical candidate, rigosertib, is positioned for multiple key milestones, following the advancement of our late stage trials for patients with myelodysplastic syndromes (MDS). Enrollment for the targeted INSPIRE pivotal trial for patients with second-line higher-risk (HR) MDS is on track, with trial sites currently active across 17 countries on four continents. The trial eligibility criteria are highly selective and require extensive search to identify appropriate patients meeting the stringent entry criteria. We intend to maintain current momentum, with interim analysis expected in the second half of the year and full enrollment by the first quarter of 2018," said Dr. Ramesh Kumar, President and Chief Executive Officer.

"Development of oral rigosertib in combination with azacitidine for first line HR MDS patients is advancing as planned, with the protocol for a pivotal Phase 3 trial expected to be ready for submission to FDA for a Special Protocol Assessment during the second or third quarter of this year. MDS represents a growing and underserved market, with more than 10,000 patients diagnosed with HR-MDS annually in the U.S. No new therapy has been approved in over ten years and none has ever been approved for patients after the failure of hypomethylating agents."

"We will continue to focus on our lead programs and will look for partnering or other opportunities for our other pipeline and clinical programs. We will be presenting non-clinical data on pipeline compounds at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) conference," added Dr. Kumar.

Recent Business Highlights:

Enrollment on Track for INSPIRE Pivotal Trial of IV Rigosertib in 2nd line HR-MDS

The global Phase 3 INSPIRE trial of IV rigosertib in patients who have failed to respond to or progressed following hypomethylating agent (HMA) therapy is active in sites across four continents in 16 countries, which is in line with the Company’s plan. Our partner, SymBio Pharmaceuticals, has opened an additional 33 sites in Japan in collaboration on the INSPIRE protocol. The trial is expected to be active in all 19 countries in the second quarter of 2017 following the activation of sites in Switzerland and the Netherlands.
Pre-planned interim analysis for the global INSPIRE trial is currently on track and will be conducted after 88 death events have occurred.
Progress on Oral Rigosertib Combination with Azacitidine for 1st-line HR-MDS

Following a productive end of Phase 2 meeting with the Food and Drug Administration (FDA) in the third quarter of 2016, a protocol for a pivotal Phase 3 trial is being developed. The Company expects to submit this protocol for review by regulatory agencies in the US and Europe in the second or third quarter of 2017.
The pivotal trial will be designed as a 1:1 randomized placebo controlled trial of oral rigosertib plus azacitidine, compared to azacitidine plus placebo. The Company plans to use a full dose of azacitidine, as defined in the product insert. The patient population studied on this trial will be HMA naïve HR MDS patients. The primary endpoint for assessment of efficacy will be Response Rate of complete remission (CR) + partial remission (PR,) as per the International Working Group (IWG) 2006 Response criteria.
Formal FDA review will be sought via the Special Protocol Assessment (SPA) mechanism.
Further details, including sample size and other criteria will be available post regulatory review, anticipated in the second half of 2017. While the pivotal trial is being designed, we plan to expand the trial cohort with the view of further dose optimization for the planned pivotal Phase 3 Trial.
Upcoming Events and Conferences

Sachs Cancer Bio Partnering & Investor Forum, New York City: March 28, 2017
American Association for Cancer Research (AACR) (Free AACR Whitepaper), Washington, D.C.: April 1-5, 2017
2016 Financial Results

Cash and cash equivalents as of December 31, 2016, totaled $21.4 million, compared to $19.8 million as of December 31, 2015. Onconova believes that its current cash and cash equivalents will be sufficient to fund its ongoing trials and operations into the fourth quarter of 2017.
Net loss was $19.7 million for the year ended December 31, 2016, compared to $24.0 million for the year ended December 31, 2015, primarily due to the change in fair value of warrant liability in the 2016 period related to warrants issued in the July 2016 rights offering.
Research and development expenses were $20.1 million for the year ended December 31, 2016, compared to $25.9 million for the year ended December 31, 2015.
General and administrative expenses were $9.2 million for the year ended December 31, 2016, compared to $9.5 million for the year ended December 31, 2015.
"During 2016, we sharpened our focus and were able to reduce costs while making progress on our lead programs. We are encouraged with the interest from potential partners and importantly, look forward to advancing our clinical programs," concluded Dr. Kumar.

On March 27, 2017 Kyowa Hakko Kirin Co., Ltd. (Tokyo: 4151, President and CEO: Nobuo Hanai, "Kyowa Hakko Kirin") reported that the top-line results of a Japanese Phase 3 study of ARQ 197 (generic name: tivantinib) did not meet its primary endpoint (Press release, Kyowa Hakko Kirin, MAR 27, 2017, View Source [SID1234518280]).

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JET-HCC is a randomized, double-blind placebo-controlled study in Japan, to evaluate the efficacy and safety of tivantinib in the patients with c-Met diagnostic-high inoperable hepatocellular carcinoma with a history of prior sorafenib therapy.

The primary endpoint is progression-free survival (PFS), and the top-line results did not show a significant difference in PFS, between the tivantinib group and the placebo group. In terms of safety, the results showed that the safety profiles were similar as previously observed, and a novel safety issue was not found.

The details of the study results will be presented at upcoming scientific congresses and/or in scientific journals.

"I sincerely thank all of the patients, their families, and all of the personnel who contributed to completing this Japanese study of tivantinib." said Mitsuo Satoh, Executive Officer, Vice President, Head of R&D Division of Kyowa Hakko Kirin. "The result was disappointing, but Kyowa Hakko Kirin has gained great experiences in oncology fields through this study."

The Kyowa Hakko Kirin Group companies strive to contribute to the health and well-being of people around the world by creating new value through the pursuit of advances in life sciences and technologies.

＜Outline of the study design＞ Target disease Patients with c-Met diagnostic-high inoperable hepatocellular carcinoma with a history of prior sorafenib therapy,
Design Multi-center, randomized, double-blind, placebo-controlled study
Dose One tablet of the study drug (tivantinib 120 mg, or placebo) is daily administered to the subjects of each group twice in a day
Subject Number approximately 190
Primary endpoint Progression-free survival
Study Location Japan

About ARQ197 (tivantinib)
Tivantinib, an oral agent whose molecular target is c-MET, was discovered by ArQule, Inc. (NASDAQ: ARQL). Kyowa Hakko Kirin signed a license agreement with ArQule for the exclusive rights to the development and sales of tivantinib in Japan and some parts of Asia (China, Korea, and Taiwan) on April 27th, 2007. News Release

About c-Met
c-Met is receptor tyrosine kinase. When abnormally activated, the c-Met receptor tyrosine kinase plays multiple roles in aspects of human cancer, including cancer cell growth, survival, angiogenesis, invasion and metastasis.