On November 7, 2016 SignalRx Pharmaceuticals Inc., a clinical-stage company focused on developing more effective oncology drugs with designed multiple target-selected inhibition profiles, reported the publication of key research supporting the use of its clinical stage drug SF1126 alone and in combination with Sorafinib for the treatment of hepatocellular carcinoma (HCC) (Press release, SignalRx, NOV 7, 2016, http://www.ireachcontent.com/news-releases/signalrx-announces-publication-of-research-results-on-sf1126-as-a-first-in-class-dual-pi3kbrd4-inhibitor-for-treating-hcc-600328031.html [SID1234517420]). The research was published in the November issue of Molecular Cancer Therapeutics journal from the American Association for Cancer Research (AACR) (Free AACR Whitepaper) (Mol Cancer Ther November 1 2016 15 (11) 2553-2562; DOI:10.1158/1535-7163.MCT-15-0976).

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Researchers at the University of California, San Diego School of Medicine and Moores Cancer Center, led by Dr. Donald Durden, Professor and Associate Director of Pediatric Oncology and senior scientific advisor for SignalRx, report results supporting the use of SF1126 as a novel therapeutic agent for the treatment of hepatocellular carcinoma (HCC), the most common kind of liver cancer and second most common cause of cancer death worldwide.

SF1126 is an anticancer agent shown to have an excellent therapeutic window with excellent tolerability and safety in Phase I clinical trials (Clinicaltrials.gov: NCT00907205). While most anti-cancer drugs only interact with a single cancer target, SF1126 inhibits two key cancer signaling molecules in liver cancer cells, phosphatidylinositol 3-kinase (PI3K) and bromodomain-containing 4 (BRD4). SF1126 represents a "first in class" approach to treat liver cancer by hitting two central signaling nodes of the liver cancer cell with only one therapeutic agent. The published work shows that this novel strategy kills liver cancer cells and prevents the growth of liver cancer tumors in mice.

Targeting two pathways with one drug can provide a significant therapeutic advantage since this approach also reduces the risk of severe "off-target" side effects resulting from the combination of side effects associated to each of the multiple drugs used. The treatment of HCC remains a challenge with Sorafenib as the only FDA-approved drug for liver cancer since it prolongs life for an average of only 2-3 months and can have significant side effects. Work from the Durden laboratory shows that using SF1126 with Sorafenib provides a dramatically improved anticancer effect by killing liver cancer cells in synergy.

In HCC, the deregulation of the PI3K/AKT/mTOR, Ras/Raf/MAPK and c-Myc signaling pathways are of prognostic significance. While Sorafenib blocks the Ras/Raf/MAPK pathway, it does not inhibit the PI3K/AKT/mTOR pathway or c-Myc activation. SF1126 controls c-Myc by inhibiting BRD4, which results in blockage of c-Myc production, and by inhibiting PI3K, which leads to enhanced c-Myc degradation. Hence, a combination of SF1126 with Sorafenib offers a new mechanism-driven mode of action to inhibit/treat HCC.

In particular, the research results published in Molecular Cancer Therapeutics demonstrate that:

SF1126 (pan PI3K/BRD4 inhibitor), as a single agent or in combination with Sorafenib, inhibits cancer cell proliferation (Hep 3B, Hep G2, SK-Hep1 and Huh7 HCC cell lines) by effectively inhibiting the PI3K/AKT/mTOR and Ras/Raf/MAPK pathways.
SF1126’s active moiety LY294002 binds to and blocks BRD4 interaction with the acetylated histone-H4 chromatin mark protein and displaces the BRD4 co-activator protein from the transcriptional start site of MYC in Huh7 and SK-Hep-1 HCC cell lines.
SF1126 blocks expression of c-Myc in HCC cells.
SF1126, either alone or in combination with Sorafenib, shows significant antitumor activity in vivo.
These published results establish SF1126 as a dual PI3K/BRD4 inhibitor and the first epigenetic/kinase inhibitor in the clinic. SF1126 has completed a Phase I clinical trial in humans with good safety profile, has received Orphan Drug Designation by the FDA, and is currently in a pediatric Phase I clinical trial in children with neuroblastoma.

Taken together, this published data strongly warrants additional clinical trials of SF1126 in advanced HCC as well as a combination Phase I trial with Sorafenib.

SignalRx is seeking partners for the clinical development of SF1126 as well as the acceleration of the company’s preclinical pipeline with novel and proprietary nM potent small molecules into first-in-man clinical trials.

SignalRx’s novel dual inhibitors have a unique competitive advantage over combining separate agents in cancers where lethality requires simultaneous target inhibition for maximal effect with minimal side-effects. Because it provides a single pharmacodynamics profile the dual inhibition in a single molecule approach provides the optimal way to effect simultaneous target inhibition with significantly less toxicity than combinations of inhibitors.

On November 7, 2016 pSivida Corp. (NASDAQ:PSDV) (ASX:PVA), a leader in the development of sustained release drug products and technologies, reported financial results for its fiscal first quarter ended September 30, 2016 (Press release, pSivida, NOV 7, 2016, View Source [SID1234516355]). In addition, the Company’s new leadership team updated corporate objectives and anticipated product development milestone timeline.

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"Since joining the Company in mid-September, I have been working with our team to assess the impressive clinical and commercial potential of our pipeline that is largely based on pSivida’s proven Durasert sustained drug release technology, the only intraocular sustained release technology with approval of three different products," said Nancy Lurker, President & CEO. "We’ve made significant progress on a number of fronts and I’m even more excited about the potential for pSivida to make a true difference in patients’ lives while we build returns to our shareholders. Our assessment reaffirmed the clear strength and quality of clinical data from studies of our Durasert three-year treatment for posterior segment uveitis (formerly known as Medidur) and we remain focused on preparing our submission for approval of this product candidate in both the European Union and United States during 2017. We’ve also begun a thorough examination of how to most efficiently and effectively launch the Durasert three-year uveitis product in the United States while we actively explore partnership possibilities to address patients with a similar diagnosis in Europe."

"Our review also resulted in our management team deploying more focus on lower risk and nearer term market opportunities as well as a renewed emphasis on potential collaborations for our Durasert technology and implementation of improvements to our product candidate evaluations. Since joining, we have continued to advance our uveitis clinical program and have reprioritized our development programs. These now include a next generation Durasert bio-erodible shorter duration treatment for posterior segment uveitis, increased emphasis on Durasert for severe osteoarthritis (OA) of the knee in conjunction with HSS, and continued work on our Durasert tyrosine kinase inhibitor (TKI) program for Wet AMD. We also continue to pursue our Tethadur platform for large molecules," Ms. Lurker added.

Fiscal First Quarter 2017 Results

Revenue for the first fiscal quarter ended September 30, 2016 totaled $277,000 compared to $466,000 for the prior year quarter. The year-over-year decrease was primarily attributable to $157,000 of non-royalty sublicense consideration earned from Alimera in the prior-year period. Operating expenses for the three months ended September 30, 2016 totaled $7.5 million compared to $5.4 million a year earlier. The increase was primarily attributable to approximately $1.1 million of severance costs, professional fees and stock-based compensation expense related to the September CEO transition, $436,000 of costs for the previously announced U.K. restructuring and approximately $300,000 of CRO and regulatory contractor costs for our Durasert three-year uveitis product candidate. Net loss for the quarter ended September 30, 2016 was $7.2 million, or $0.21 per share, compared to net loss of $4.9 million, or $0.17 per share, for the prior year quarter.

At September 30, 2016, cash, cash equivalents and marketable securities totaled $22.5 million.

Product Candidate Program Update & Anticipated Milestones

Durasert three-year treatment for posterior segment uveitis: The Company met its enrollment target in the second uveitis Phase 3 trial of 150 patients in September. Readout of this second trial, which is required by the U.S. Food & Drug Administration for the Company’s NDA filing, is currently expected by the end of the first half of 2017. The first Phase 3 study met its primary efficacy endpoint with a p value of < 0.001 and safety data that are consistent with the known effect of ocular corticosteroid use. With regard to the planned E.U. submission for marketing authorization, management’s goal remains to submit during the first quarter of 2017. The Company was recently notified that protocol approval for a pediatric study would be required by the European regulatory authority prior to the acceptance of the application for market authorization. The protocol for the pediatric study has been submitted and the timing of its approval could move the acceptance of the market application into the second quarter of 2017.

Next Generation Durasert bio-erodible shorter duration treatment for posterior segment uveitis: The Company has initiated and prioritized a development program for a next generation Durasert bio-erodible for uveitis. The Company is initiating formulation testing now and expects to begin pre-clinical safety and PK studies of this product candidate in the first half of 2017. Management believes this product candidate will provide enhanced benefits to patients and physicians by offering a shorter delivery time period of corticosteroid and providing more flexibility to physicians with multiple Durasert dosing intervals.

Durasert implant for severe osteoarthritis (OA) of the knee: On August 1, 2016, the Hospital for Special Surgery in New York, NY and pSivida announced the opening of an IND in support of an investigator-sponsored clinical study of a Durasert implant to treat severe OA of the knee. Management believes severe OA of the knee is a large and growing condition with continued high unmet medical needs. The implant is designed to provide long-term pain relief for this condition, which, if effective, could potentially result in the delay of knee replacement surgery. The study is an open-label, single dose, safety and tolerability study of the screw implant to deliver dexamethasone, a corticosteroid previously proven to provide pain relief in knee OA. Six patients will each receive the implant in one knee. While a safety and tolerability study, change from baseline in weekly mean of pain intensity scored at rest, during activity and at night will be assessed through 24 weeks. To date two patients have received the implant and HSS expects to have all six patients implanted over the next few months.

Durasert bio-erodible TKI for Wet AMD: Management believes that pSivida’s TKI program could represent a valuable advancement to the treatment of Wet AMD. As part of the new leadership’s program assessment, it has been determined that further evaluation of additional TKIs is needed to optimize candidate selection, and management is actively pursuing a lead candidate for the clinic.

Tethadur for large molecules: The Tethadur program applies proprietary technology to achieve the sustained release of large molecules such as biologics. Recently, management narrowed its focus to silica-based technology from the earlier silicon-based technology in an effort to advance the program in a cost-effective way. Pre-clinical activities on this program are continuing.

"During the past few weeks I have had the pleasure of getting to know the people that developed pSivida’s terrific technology. With the recent additions of Dr. Dario Paggiarino and Deb Jorn, we have a talented and committed team with a singular focus to successfully bring pSivida’s products to patients and deliver greater shareholder returns," concluded Ms. Lurker.

On November 7, 2016 Ignyta, Inc. (Nasdaq: RXDX), a biotechnology company focused on precision medicine in oncology, reported company highlights and financial results for the third quarter ended September 30, 2016 (Press release, Ignyta, NOV 7, 2016, View Source [SID1234516390]).

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"During the third quarter, we advanced our lead program, entrectinib, with continued successful execution of STARTRK-2, our global pivotal Phase 2 clinical trial now open at more than 100 sites in 12 countries," said Jonathan Lim, M.D., Chairman and CEO of Ignyta. "This global clinical footprint helps ensure that entrectinib, a novel, orally available, CNS-penetrant tyrosine kinase inhibitor targeting tumors that harbor TRK, ROS1, or ALK fusions, is made available to patients for clinical development on a worldwide basis. Further, by expanding our collaboration with the European Organisation for Research and Treatment of Cancer, or EORTC, we hope to identify additional patients across Europe who may be eligible for STARTRK-2."

Ignyta will not be conducting a conference call in conjunction with this release, but will summarize company highlights for the third quarter as part of its presentation at the Credit Suisse 25th Annual Healthcare Conference on Tuesday, November 8, 2016, at 10:30 a.m. Mountain time (9:30 a.m. Pacific time) in Scottsdale, AZ. A webcast of the presentation will be available during the presentation in the Investors section of the company’s website at View Source, and will be archived and available at that site for 14 days.

Company Highlights

Announced Approval of an IDE for Trailblaze Pharos

In August 2016, the U.S. Food and Drug Administration (FDA) approved an investigational device exemption (IDE) for the company’s RNA-based companion diagnostic, next-generation sequencing (NGS) assay known as Trailblaze Pharos. The Trailblaze Pharos assay is intended for use in identifying patients, including those who are treatment-naïve, who have solid tumors with NTRK1/2/3, ROS1, or ALK gene rearrangements leading to fusion proteins, to determine eligibility for enrollment into the global STARTRK-2 trial.

Expanded Collaborative Agreement with EORTC

In September 2016, we expanded our collaborative agreement with EORTC. Under this collaboration, Ignyta will serve as a diagnostic laboratory performing fusion testing for EORTC’s SPECTA (Screening Patients for Efficient Clinical Trial Access) initiative, which may help us identify patients with a variety of tumor histologies across Europe who could potentially be enrolled in our STARTRK-2 clinical trial.

Third Quarter 2016 Financial Results

For the third quarter of 2016, net loss was $23.3 million, or $0.56 per share, compared with $14.6 million, or $0.49 per share, for the third quarter of 2015.

Ignyta did not record any revenue for the three months ended September 30, 2016, or for the three months ended September 30, 2015.

Research and development expenses for the third quarter of 2016 were $16.6 million, compared with $10.4 million for the third quarter of 2015. This increase was primarily due to the $4.3 million increase in the external development costs associated with entrectinib, taladegib and other product candidates, coupled with personnel expenses related to hiring and engaging additional employees and consultants to help advance the company’s product candidates.

General and administrative expenses were $6.1 million for third quarter of 2016, compared with $3.9 million for third quarter of 2015. This increase was driven by higher personnel and share-based compensation costs, higher facilities-related expenses resulting from the increase of our leased facilities space, and increases in consulting fees and depreciation expense.

At September 30, 2016, the company had cash, cash equivalents and available-for-sale securities totaling $152.5 million and current and long-term debt of $32.0 million. At December 31, 2015, the company had cash, cash equivalents and available-for-sale securities totaling $172.1 million and current and long-term debt of $31.0 million.

On November 7, 2016 Xenetic Biosciences, Inc. (NASDAQ:XBIO) ("Xenetic" or the "Company"), a clinical-stage biopharmaceutical company focused on discovery, research and development of next-generation biologic drugs and novel orphan oncology therapeutics, reported the closing of its public offering of an aggregate of 2,424,242 units, consisting of (i) 484,849 units, consisting of one share of Convertible Series B Preferred Stock and a Class A Warrant to purchase one share of common stock and (ii) 1,939,393 units consisting of one share of Convertible Series B Preferred Stock and a Class B Warrant to purchase one share of common stock, at a public offering price of $4.125 per unit (Press release, Xenetic Biosciences, NOV 7, 2016, View Source [SID1234537810]). OPKO Health, Inc. (Nasdaq:OPK) along with other healthcare institutional investors participated in the offering.

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As previously announced, in connection with the closing of its public offering, the Company commenced trading of its common stock today on The Nasdaq Capital Market under the symbol "XBIO."

Ladenburg Thalmann & Co. Inc. acted as the sole book running manager for the offering.

The total gross proceeds of the public offering are approximately $10 million before the underwriter’s discount and expenses. The net proceeds from this offering will be used to fund the research and development of Xenetic’s product candidates, including Virexxa, as well as future development programs, potential in licensing of products or technology, potential in licensing of products or technology, capital expenditures, working capital, repayment of existing indebtedness, and other general corporate purposes.

The shares and warrants described above were offered by Xenetic pursuant to a registration statement on Form S-1 previously filed with, and subsequently declared effective by the Securities and Exchange Commission ("SEC"). A final prospectus supplement relating to the offering was filed with the SEC and is available, along with the accompanying base prospectus, on the SEC’s website at View Source or from Ladenburg Thalmann & Co. Inc., Prospectus Department, 570 Lexington Avenue, 11th Floor, New York, New York 10022, by calling (212) 409-2000.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale is not permitted.

On November 7, 2016 ArQule, Inc. (Nasdaq: ARQL) reported its financial results for the third quarter of 2016 (Press release, ArQule, NOV 7, 2016, View Source [SID1234516358]).

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For the quarter ended September 30, 2016, the Company reported a net loss of $5,817,000 or $0.08 per share, compared to a net loss of $2,354,000 or $0.04 per share, for the third quarter of 2015. For the nine-month period ended September 30, 2016, the Company reported a net loss of $15,898,000 or $0.23 per share, compared to a net loss of $10,922,000 or $0.17 per share for the nine-month period ended September 30, 2015.

At September 30, 2016, the Company had a total of $37,659,000 in cash, equivalents and marketable securities.

Key Highlights

ARQ 087, our proprietary FGFR inhibitor, is approaching completion of enrollment in the phase 2 portion of the phase 1/2 trial in intrahepatic cholangiocarcinoma (iCCA). Discussions with regulatory agencies in the U.S. and Europe are nearing completion for the design of a potential pivotal trial in iCCA. We plan to finalize the trial design by year end pending final trial results.
ARQ 531, our proprietary reversible BTK inhibitor, continues to demonstrate best-in-class potential with preclinical data to be presented at the 2016 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. The data, to be presented by The Ohio State University, demonstrate that ARQ 531 effectively inhibits C481S mutant BTK in patient derived cells and in a TCL1 mouse model shows efficacy superior to that of ibrutinib in Chronic Lymphocytic Leukemia (CLL). The company plans to complete preclinical studies and file an Investigational New Drug (IND) application in early 2017 to begin clinical trials with an initial focus on the fast-to-market, ibrutinib resistant C481S mutant BTK CLL population.
ARQ 092, our lead proprietary AKT inhibitor, continues to demonstrate the potential utility of targeting AKT in rare non-oncological indications and will be the focus of an oral presentation at the ASH (Free ASH Whitepaper) Annual Meeting. The data, to be presented by The University of Illinois College of Medicine, demonstrate that in neutrophils and platelets from Sickle Cell Disease (SCD) patients in vitro and cell-cell interactions in a mouse model of SCD, ARQ 092 attenuates neutrophil-platelet interactions. The study provides evidence that ARQ 092 could be a novel therapy in treating and preventing acute vaso-occlusive complications in SCD. The data warrants further studies of ARQ 092 in SCD.
ARQ 092 phase 1 trial for Proteus syndrome continues to enroll and our collaborator, the National Institutes of Health (NIH), is in the final stages of implementing an updated enrollment protocol that will facilitate logistics for patients and their families. To date, the drug has been well tolerated, and we are looking forward to assessing full data from the initial two cohorts in the early part of next year.
ARQ 092 clinical research to be expanded into PROS (PIK3CA-Related Overgrowth Spectrum) family of rare diseases. The company received approval of its IND application from the Food and Drug Administration (FDA) in the PROS family of rare diseases, including Proteus syndrome, for a potential clinical trial.
Tivantinib METIV-HCC phase 3 trial for hepatocellular carcinoma (HCC) is scheduled to conclude in early 2017. Top-line data is expected in the first quarter of 2017.
"It is exciting to see our proprietary pipeline being pursued independently and through collaborations with the top researchers in the fields of oncology, hematology and selected rare diseases," said Paolo Pucci, Chief Executive Officer of ArQule. "We are encouraged that several projects have attracted the interest of leading scientific institutions including ARQ 531, our BTK inhibitor, which is in preclinical testing with our collaborators at The Ohio State University. ARQ 092 continues to progress in Proteus syndrome through a program that includes a phase 1 trial conducted by the NIH. In addition, we now have preclinical data for ARQ 092 in Sickle Cell Disease through the work of The University of Illinois College of Medicine. Lastly, we are pleased to have moved closer to a potential pivotal trial in an attractive fast to market opportunity for ARQ 087 in iCCA."

"Our lead proprietary drug candidate, ARQ 087, is nearing completion of the phase 2 iCCA trial," said Dr. Brian Schwartz, M.D., Head of Research and Development and Chief Medical Officer at ArQule. "Encouraging discussions and positive feedback from the regulatory agencies, combined with the totality of the efficacy data we have observed in the clinical trials, has moved us closer to defining a pivotal trial design for ARQ 087 in this indication."

Revenues and Expenses

Revenues for the quarter ended September 30, 2016, were $1,223,000 compared with revenues of $2,653,000 for the quarter ended September 30, 2015. Revenues in the nine-months ended September 30, 2016 were $3,522,000 compared with revenues of $8,442,000 in the nine-months ended September 30, 2015. Revenue in the three and nine-month periods of 2016 and 2015 is comprised of revenue from the Daiichi Sankyo tivantinib development agreement and the Kyowa Hakko Kirin exclusive license agreement.

The revenue decreases in the quarter ended September 30, 2016 of $0.5 million from our Daiichi Sankyo METIV-HCC trial and $1.0 million from our Kyowa Hakko Kirin JET-HCC trial were principally due to the March 2016 extension of the development period through December 31, 2016 for both programs. The revenue decreases in the nine months ended September 30, 2016 of $2.0 million from our Daiichi Sankyo METIV-HCC trial and $2.9 million from our Kyowa Hakko Kirin JET-HCC trial were also principally due to the extension of the development period through December 31, 2016.

Research and development expense in the third quarter of 2016 was $5,265,000 compared with $3,180,000 for the third quarter of 2015. The $2.1 million increase in research and development expense in the third quarter of 2016 was principally due to increased outsourced clinical and product development costs of $1.9 million and professional fees of $0.2 million.

Research and development expense in the nine-months ended September 30, 2016 was $13,800,000 compared with $11,920,000 in the nine-months ended September 30, 2015. The $1.9 million increase in research and development expense in the nine-months ended September 30, 2016 was primarily due to increased outsourced clinical and product development costs of $2.6 million and professional fees of $0.2 million, partially offset by decreased labor and related costs of $0.4 million and facility costs of $0.5 million.

General and administrative expense was $1,824,000 in the third quarter of 2016 compared with $1,839,000 in the third quarter 2015.

General and administrative expense was $5,755,000 in the nine-months ended September 30, 2016 compared with $7,802,000 in the nine-months ended September 30, 2015. General and administrative expense decreased by $2.0 million in the nine-months ended September 30, 2016 primarily due to lower facility costs of $1.6 million, labor related costs of $0.2 million and professional fees of $0.2 million.