On December 20, 2016 Prima BioMed Ltd (ASX: PRR; NASDAQ: PBMD), reported that it has received approval from the Competent Authority and Ethics Committee in the UK for its Phase IIb, AIPAC clinical trial of IMP321 (Filing, 6-K, Prima Biomed, DEC 20, 2016, View Source [SID1234517148]). Following conclusion of the safety run in phase, expected in late December, and subject to the dose escalation committee meeting, screening for the larger, randomised phase of the trial is expected to commence in January 2017.
AIPAC (Active Immunotherapy PAClitaxel) is a multi-national, randomised, double-blind, placebo-controlled study of IMP321-plus-paclitaxel in metastatic breast cancer. The safety run-in phase of the first cohort of 15 patients is being conducted across 11 clinical sites in Belgium, The Netherlands and Hungary. Recruitment of the last two patients in this cohort was completed in October. The first safety and pharmacokinetic data from these 15 patients is expected in late December 2016. As previously announced, AIPAC’s expected duration based on forecast recruitment times and patient follow up is approximately three years.
Prima also confirms that interim data from the first cohort of patients in its Phase I, TACTI-mel clinical trial of IMP321 together with KEYTRUDA for metastatic melanoma patients is also expected in late December 2016.

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On December 20 2016 Opsona Therapeutics Ltd (‘Opsona’), the innate immune drug development company focused on novel therapeutic approaches to treat oncology, autoimmune and other inflammatory diseases, reported the preliminary results from its ongoing prospective, open label Phase I/II study being conducted with OPN-305 in secondline lower (Low and intermediate-1) risk myelodysplastic syndrome (MDS) which created interest when presented recently at the 58th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in San Diego by Prof Garcia-Manero from the MD Anderson Cancer Center (Press release, Opsona Therapeutics, DEC 20, 2016, View Source;n=154&a=130 [SID1234517143]).

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OPN-305 is a novel proprietary humanized IgG4 monoclonal antibody (MAb) against Toll-Like Receptor 2 (TLR2), a key target within the innate immune system. Opsona has recently received orphan drug designation from the United States Food and Drug Administration for MDS.

The study in patients with lower risk, red cell transfusion dependent, MDS who have failed hypomethylating agents (HMA) ± an erythropoiesis stimulating agent is ongoing in collaboration with MD Anderson Cancer Center in Houston USA with additional sites now being added in the USA.

As of December 2016, 24 eligible patients have been enrolled, 11 at 5 mg dose and 13 at 10 mg/kg. A total of 15 (75%) patients are evaluable for response. Hematological improvement has been seen in 53% (8/15) with 3 (20%) patients achieving transfusion independence and of these 2/5 (40%) were receiving 10 mg/kg while on OPN-305 monotherapy. 12 patients remain on study.

Median age was 72 years (range 42-87). Nine (43 %) patients were classified as Low risk and 15 (63%) as Intermediate-1 risk by IPSS. Thirteen patients (61%) had diploid cytogenetics, 8 (38%) RAEB,5 (23%) RCMD, 3 (14%) RA, 2 (10%) RARS, and 1 (4%) 5q-, RCMD-RS, CMML.

The median number of prior HMA therapies was 2 (range 1-4) with a median duration of prior therapies from time of diagnosis to enrollment of 22.7 months (range 6.3-56.1). The median number of OPN-305 cycles administered is 5 (2-22) with 5 of 9 (55.5%) patients having received azacitidine add-back after 16 weeks of OPN-305 monotherapy. A total of 5 (29%) patients developed AEs related to OPN-305 all grade 1 with gastrointestinal disorders being the most frequent (23.5%). At this point, no significant drug related toxicity or unexpected infectious complications have been seen and combination with azacitidine has been well tolerated.

To date three (20%) patients were taken off study due to progression to AML and 4 (27%) due to no response all at the 5 mg/kg dose. There is no evidence of treatment related anti-drug antibodies or statistically significant dynamic changes in cytokines in any of the patients.

Myelodysplastic syndromes are a complex and heterogeneous group of bone marrow failure disorders characterized by ineffective hematopoiesis and poor prognosis. There is an urgent need for the development of well tolerated, novel therapies in the treatment of MDS which can delay progression, improve patient survival and quality of life and reduce the social and economic burden of transfusion dependence.

Commenting on today’s announcement Mary Reilly VP Pharmaceutical Development & Operations said "OPN-305 data emerging in this heavily pre-treated group of patients is very encouraging, the unmet need for a safe and tolerable product for this patient population is significant and we are happy to be in collaboration with the MD Anderson Cancer Center one of the leading clinical center’s in this hematological area"

On December 20, 2016 Rosetta Genomics Ltd. (NASDAQ: ROSG), a genomic diagnostics company that improves treatment decisions by providing timely and accurate diagnostic information to physicians, reported financial results for the three and nine months ended September 30, 2016(Filing, Q3, Rosetta Genomics, 2016, DEC 20, 2016, View Source [SID1234517142]).

Highlights for the third quarter of 2016 and recent weeks include:

· Announced the publication of a clinical validation study in support of RosettaGX RevealÔ (Reveal), Rosetta’s first-of-its-kind microRNA classifier for indeterminate thyroid nodules, in the peer-reviewed Journal of Clinical Pathology;
· Reveal was featured on the cover of the October issue of the peer-reviewed journal Cancer Cytopathology;
· Announced that Reveal is now available to be used on ThinPrep samples and reported results from a study confirming that Reveal produces the same high level performance on ThinPrep prepared slides as it does on a direct smear from a thyroid Fine Needle Aspirate ("FNA") biopsy;
· Entered into an exclusive distribution agreement with Rhenium Ltd. for the sales and marketing of Reveal in Israel, where Clalit, (General Sick Fund), the largest organization and the first health insurance institution in Israel, has indicated it will include the Reveal assay in its Sick Fund;
· Closed concurrent registered direct and private placement offerings with one prominent institutional healthcare investor with expected net proceeds of approximately $4.6 million;
· Entered into a research agreement with Sheba Medical Center at Tel HaShomer, Israel, to develop a microRNA-based signature to predict response to nivolumab, an immunotherapy drug marketed as Opdivo, which is approved for the treatment of lung cancer; and
· Fortified its intellectual property portfolio with two new patent allowances for the company’s novel microRNA platform technology as a prognostic for predicting progression of platinum-resistant stage III ovarian cancer and for diagnosing pleural mesothelioma or distinguishing between pleural mesothelioma and other cancers.

Management Commentary

"Throughout 2016 we have been focused on the successful commercial launch of our Reveal assay for the classification of indeterminate thyroid nodules and we have made significant progress advancing this important product offering. We were pleased that two peer-reviewed journal articles supporting the clinical and analytical validation of Reveal were published in prestigious journals, which considerably enhances our sales and marketing efforts. In addition, we presented data from a study confirming that Reveal produces the same high-level performance on ThinPrep prepared slides as it does on a direct smear from a thyroid Fine Needle Aspirate (FNA) biopsy. This opens our market to pathologists who prefer to use ThinPrep slides and we have since seen an uptick in demand," stated Kenneth A. Berlin, President and Chief Executive Officer of Rosetta Genomics.

"We remain committed to advancing our strategy to use Reveal to access new accounts to promote our exceptional thyroid offering as well as our urologic cancer and solid tumor product lines. We believe that opening new relationships with Reveal will allow us to bring our solid tumor and urologic oncology offerings to many of these new accounts, thus further accelerating revenue growth.

"In addition to focusing on our suite of marketed diagnostic assays, we made significant progress on a number of important corporate initiatives that further strengthen Rosetta Genomics. We entered into a research collaboration to identify a microRNA-based biomarker to predict treatment response to Opdivo, fortified our microRNA intellectual property portfolio with two new patent allowances and strengthened our balance sheet by closing financing transactions with expected net proceeds of $4.6 million.

"As we enter 2017, Rosetta Genomics is well-positioned for success and we look forward to achieving a series of value-creating milestones," concluded Mr. Berlin.

Third Quarter Financial Results

· Revenues for the third quarter of 2016 decreased 10% to $2.2 million compared with $2.4 million for the third quarter of 2015, primarily due to lower sales of urologic and solid tumor testing services as the Company refocused its sales force on the Reveal introduction.
· Revenues from urologic cancer testing services for the third quarter of 2016 was $970,000, compared with $1.3 million for the third quarter of 2015, and represented approximately 44% of clinical testing revenues for the third quarter of 2016.
· Revenues from solid tumor testing services for the third quarter of 2016 of $841,000 decreased 26% compared with $1.1 million in the third quarter of 2015, and represented 38% of total clinical testing revenues during the third quarter of 2016.
· Reveal revenues for the third quarter of 2016 were $282,000, a 70% increase compared with $166,000 for the second quarter of 2016. There were no Reveal revenues in 2015 as the assay was launched commercially in the first quarter of 2016. Reveal revenues represented 13% of total clinical testing revenues in the third quarter of 2016.

· The Company also had $107,000 of revenues from Hematological FISH testing (HEME FISH), a new line of business this quarter, which represented 5% of total clinical testing revenues in the third quarter of 2016.
· On a non-GAAP basis, gross billings for Reveal during the third quarter of 2016 were $930,000 compared with $511,000 for the second quarter of 2016, representing growth of 82%. Gross billings are the aggregate amounts invoiced to customers.
· Cost of revenues for the third quarter of 2016 decreased to $1.8 million from $2.2 million for the third quarter of 2015.
· Research and development expenses for the third quarter of 2016 increased to $879,000 from $586,000 for the third quarter of 2015, primarily due to timing of acquisition of clinical samples.
· Sales, marketing and business development expenses were $1.7 million for the third quarters of 2016 and 2015.
· General and administrative expenses for the third quarter of 2016 decreased to $1.8 million compared with $1.9 million for the same period in 2015 primarily due to financing-related expenses in 2015.
· The operating loss for the third quarter of 2016 was $3.9 million, which included $238,000 of non-cash stock-based compensation expense, compared with an operating loss of $3.9 million for the third quarter of 2015, which included $211,000 of non-cash stock-based compensation expense.
· The net loss for the third quarter of 2016 was $4.0 million, or $0.19 per ordinary share on 20.9 million weighted average shares outstanding, compared with a net loss for the third quarter of 2015 of $3.9 million, or $0.27 per ordinary share on 14.8 million weighted average shares outstanding.
· On a non-GAAP basis, excluding the 238,000 of non-cash stock-based compensation expense, the net loss for the third quarter of 2016 would have been $3.7 million, or $0.18 per ordinary share.
· For 2015, on a non-GAAP basis, excluding the 211,000 of non-cash stock-based compensation expense, the net loss for the third quarter of 2015 would have been $3.7 million, or $0.25 per ordinary share.

Nine Month Financial Results

· Revenues for the first nine months of 2016 increased 53% to $7.2 million compared with $4.7 million for the first nine months of 2015. On a pro forma basis (as if the PersonalizeDx acquisition occurred on January 1, 2015 instead of the actual acquisition date of April 13, 2015), revenues for the first nine months of 2016 increased 9% compared with pro forma revenues of $6.6 million for the first nine months of 2015.
· Cost of revenues for the nine-month period ended September 30, 2016 was $5.4 million compared with $4.4 million for the same period of 2015.
· Total operating expenses for the first nine months of 2016 of $13.1 million compared with $10.9 million for the first nine months of 2015, which included a gain of $2.4 million on bargain purchase related to the acquisition of PersonalizeDx.
· The operating loss for the first nine months of 2016 was $11.3 million, which included $697,000 of non-cash stock-based compensation expense, compared with an operating loss for the first nine months of 2015 of $10.6 million, which included $755,000 of non-cash stock-based compensation expense as well as a gain of $2.4 million on bargain purchase related to the acquisition of PersonalizeDx.

· The net loss for the first nine months of 2016 was $11.4 million, or $0.55 per ordinary share on 20.8 million weighted average shares outstanding, compared with a net loss for the first nine months of 2015 of $10.6 million, or $0.76 per ordinary share.

Balance Sheet Highlights

As of September 30, 2016, Rosetta Genomics had cash, cash equivalents, restricted cash and short-term bank deposits of $6.1 million, compared with $13.6 million as of December 31, 2015. The Company used approximately $9.1 million in cash to fund operations during the first nine months of 2016, and collected approximately $6.9 million in cash from its clinical testing services as well as $1.6 million from a licensing deal signed in December 2015. Following the close of the quarter, Rosetta Genomics closed concurrent registered direct and private placement offerings with one institutional healthcare investor for expected net proceeds of approximately $4.6 million. Rosetta Genomics believes that its current cash, together with its existing operating plan, which includes cost-reduction plan should it be unable to raise additional capital, should provide sufficient liquidity resources for the Company and its subsidiaries through the third quarter of 2017.

2017 Revenue and Unit Guidance for RosettaGX Reveal

Rosetta Genomics also today introduces 2017 revenue and unit guidance for RosettaGX Reveal. For 2017, Rosetta Genomics expects Reveal revenue to be between $4.0 million and $5.0 million, and expects to process between 2,500 and 3,500 Reveal units throughout the year.

ThinPrep is a registered trademark of Hologic, Inc. and Opdivo is a registered trademark of Bristol-Myers Squibb.

Use of Non-GAAP Financial Measures
This press release contains certain non-GAAP financial measures. A "non-GAAP financial measure" refers to a numerical measure of historical or future financial performance, financial position or cash flows that excludes (or includes) amounts that are included in (or excluded from) the most directly comparable measure calculated and presented in accordance with GAAP in the financial statements. In this release, Rosetta provides non-GAAP gross billings, non-GAAP net loss and non-GAAP net loss per share data as additional information relating to its operating results. The presentation of this additional information is not meant to be considered in isolation or as a substitute for revenues, net loss or net loss per share prepared in accordance with GAAP.

Pursuant to the requirements of Regulation G promulgated by the SEC, the Company has provided a reconciliation of each non-GAAP financial measure used in this earnings release and related conference call or webcast to the most directly comparable financial measure prepared in accordance with GAAP. This reconciliation is presented in the tables below under the heading "Reconciliation of GAAP to Non-GAAP Consolidated Statement of Operation." Investors are encouraged to review these reconciliations to ensure they have a thorough understanding of the reported non-GAAP financial measures and their most directly comparable GAAP financial measures.

Management uses these non-GAAP measures for internal reporting and forecasting purposes. The Company has provided these non-GAAP financial measures in addition to GAAP financial results because it believes that these non-GAAP financial measures provide useful information to certain investors and financial analysts for comparison across accounting periods not influenced by certain non-cash items that are not used by management when evaluating the Company’s historical and prospective financial performance.

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