On May 19, 2016 Rosetta Genomics Ltd. (NASDAQ: ROSG), a leading developer and provider of microRNA-based and other molecular diagnostics, reported financial results for the three months ended March 31, 2016 (Filing, Q1, Rosetta Genomics, 2016, MAY 19, 2016, View Source [SID:1234512620]).

Recent developments include:

· Expanded molecular diagnostics test menu with the launch of three new product offerings in common hematologic cancers and solid tumors;
· Received conditional approval status from the New York State Department of Health (NYSDOH) for RosettaGX Reveal, the Company’s novel microRNA classifier for the diagnosis of indeterminate thyroid Fine Needle Aspirate (FNA) smears;
· Entered into an agreement that establishes health insurance access to Rosetta’s entire suite of diagnostic tests and services with America’s Choice Provider Network (ACPN), an independent multispecialty national provider network; and
· Granted U.S. patent allowance for use of gene expression signature for classification of kidney tumors and granted European patent allowance for use of microRNA molecules for the treatment of liver cancer.

Management Commentary

"We are especially pleased to report record quarterly clinical testing revenues as it demonstrates the progress we have made in expanding our molecular diagnostics test menu, selling our clinical testing products and improving collections," said Kenneth A. Berlin, President and Chief Executive Officer of Rosetta Genomics. "Throughout the first quarter we completed the revamping of our sales force and invested in our billing and collections department. The results are reflected in our growing revenue and expanding customer base, as well as in improved collections. Further, these changes position us to drive revenue growth throughout the balance of the year and beyond.

"The commercial launch of RosettaGX Reveal continues to be a prime focus for our team. We expect the positive performance data from our blinded validation study to be published in a peer-reviewed journal in the coming weeks. These data demonstrate exceptional performance and we anticipate that a journal publication will strongly support our reimbursement and sales efforts. In addition, our revamped sales team has been able to use RosettaGX Reveal to access new accounts to promote not only our exceptional thyroid offering, but also to promote our urologic cancer and solid tumor product lines. Since the beginning of the year, these promotional efforts resulted in the acquisition of over 30 thyroid customer accounts and over 60 new customer accounts for our urology and solid tumor businesses.

"Our work for the balance of the year will continue to focus on driving revenue growth in both our base business as well as with our new products, such as RosettaGX Reveal, expanding reimbursement, improving collections and advancing our clinical development programs, which should position us to achieve a number of important milestones that will enhance shareholder value," concluded Mr. Berlin.

First Quarter Financial Results

Please note that the pro forma comparisons below are meant to provide a comparison as if the PersonalizeDx acquisition occurred on January 1, 2015. The actual acquisition date was April 13, 2015.

· Revenues for the first quarter of 2016 increased 711% to $2.6 million compared with revenues of $321,000 for the first quarter of 2015, and increased 27% compared with pro forma revenues of $2.1 million for the first quarter of 2015.
· Revenues from urologic cancer testing services in the first quarter of 2016 were $1.4 million, an increase of 7% compared with pro forma revenues of $1.3 million for the first quarter of 2015, and represented approximately 54% of clinical testing revenues for the quarter.
· Revenues from solid tumor testing services in the first quarter of 2016 increased 272% to $1.2 million compared with revenues of $321,000 for the first quarter of 2015, and increased 58% compared with pro forma revenues of $0.8 million in the first quarter of 2015. Solid tumor testing services represented nearly 46% of total clinical testing revenues during the first quarter of 2016, with the balance coming from RosettaGX Reveal.
· Cost of revenues for the first quarter of 2016 increased to $1.7 million compared with $352,000 for the first quarter of 2015, due to the acquisition of PersonalizeDx leading to a higher volume of processed samples, as well as to increases in personnel and infrastructure.
· Research and development expenses for the first quarter of 2016 increased to $842,000 from $748,000 for the first quarter of 2015, primarily due to increased activities in Thyroid and other areas.
· Sales, marketing and business development expenses for the first quarter of 2016 increased to $1.9 million from $1.6 million in the prior-year period due to a larger commercial footprint as a result of the acquisition of PersonalizeDx.
· General and administrative expenses for the first quarter of 2016 increased to $2.2 million compared with $1.4 million for the same period in 2015, with the increase primarily due to increased personnel and activities related to the acquisition of PersonalizeDx.
· The operating loss for the first quarter of 2016 was $4.0 million, which included $230,000 of non-cash stock-based compensation expense, compared with an operating loss of $3.8 million for the first quarter of 2015, which included $276,000 of non-cash stock-based compensation expense.

· The net loss for the first quarter of 2016 was $4.0 million, or $0.20 per ordinary share on 20.7 million weighted average shares outstanding, compared with a net loss for the first quarter of 2015 of $3.9 million, or $0.30 per ordinary share on 12.8 million weighted average shares outstanding.
· On a non-GAAP basis, excluding $230,000 of non-cash stock-based compensation expense, the net loss for the first quarter of 2016 was $3.8 million, or $0.18 per ordinary share on 20.7 million weighted average shares outstanding. For the first quarter of 2015, excluding the $276,000 of non-cash stock-based compensation expense, the non-GAAP net loss was $3.6 million, or $0.28 per ordinary share on 12.8 million weighted average share outstanding.

Balance Sheet Highlights

As of March 31, 2016, Rosetta Genomics had cash, cash equivalents, restricted cash and short-term bank deposits of $12.6 million compared with $13.6 million as of December 31, 2015. The Company used approximately $2.6 million in cash to fund operations during the first quarter of 2016, and collected approximately $2.7 million in cash from its clinical testing services in addition to $1.6 million in cash receipts from a licensing deal signed in December 2015. Based on the Company’s current operations and plans, which include a cost-reduction plan should it be unable to raise sufficient additional capital, if necessary, Rosetta Genomics expects its current cash position will fund operations for at least the next 12 months.

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On May 19, 2016 Celator Pharmaceuticals, Inc. (Nasdaq:CPXX) reported that Phase 3 clinical trial data for VYXEOS (cytarabine:daunorubicin) Liposome for Injection (also known as CPX-351), its lead product candidate, will be presented at upcoming medical conferences (Press release, Celator Pharmaceuticals, MAY 19, 2016, View Source [SID:1234512619]).

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Presentation at ASCO (Free ASCO Whitepaper) Annual Meeting in Chicago, June 3-7, 2016:

Date & Track Time:
Saturday, June 4, 2016 – 3:00pm to 6:00pm CT

Track:
Hematologic Malignancies – Leukemia, Myelodysplastic Syndromes, and Allotransplant

Presentation Title:

Final results of a phase III randomized trial of CPX-351 versus 7+3 in older patients with newly diagnosed high risk (secondary) AML

Presenter:
Jeffrey E. Lancet, M.D., H. Lee Moffitt Cancer Center & Research Institute

Abstract #:
7000

Presentation Time:
3:00pm to 3:12pm CT

Location:
Arie Crown Theatre

Presentation at EHA (Free EHA Whitepaper) Annual Congress in Copenhagen, June 9-12, 2016:

Date:
Saturday, June 11, 2016

Topic:
Acute Myeloid Leukemia – Clinical Session

Title:
New Compounds in AML Treatment

Presentation Title:
CPX-351 treatment of previously untreated older AML patients with high-risk AML markedly increases the response rate over 7+3 in patients with FLT3 mutations

Presenter:
Bruno C. Medeiros, M.D., Stanford University

Abstract Code:
S502

Presentation Time:
4:15pm to 4:30pm CET

Location:
Hall A3

The ASCO (Free ASCO Whitepaper) abstracts became available at 5:00pm ET on May 18th (abstracts.asco.org) and the EHA (Free EHA Whitepaper) abstracts at 12:00pm CET on May 19th (EHA Learning Center – Official eLearning Portal of the European Hematology Association (EHA) (Free EHA Whitepaper)).

Analyst and Investor Meeting on VYXEOS

Celator will host an Analyst and Investor meeting on Saturday, June 4, 2016 at the Chicago Marriott Downtown Magnificent Mile starting at 7:00pm CT. The meeting will discuss data from the Phase 3 clinical trial in high-risk AML patients. Seating is limited, please contact the Trout Group, Brooks Rahmer ([email protected]), regarding attendance. The meeting will be webcast and available on Celator’s website (www.celatorpharma.com).

About VYXEOS

VYXEOS (cytarabine:daunorubicin) Liposome for Injection, also known as CPX-351, is a nano-scale co-formulation of cytarabine and daunorubicin at a synergistic 5:1 molar ration. VYXEOS represents a novel approach to developing combinations of drugs in which molar ratios of two drugs with synergistic anti-tumor activity are encapsulated in a nano-scale liposome in order to maintain the desired ratio following administration. The FDA granted Breakthrough Therapy designation to VYXEOS for the treatment of adults with therapy-related AML (t-AML) or AML with myelodysplasia-related changes (AML-MRC. VYXEOS was granted orphan drug status for the treatment of AML by the FDA and the European Commission. VYXEOS was also granted Fast Track designation for the treatment of elderly patients with secondary AML by the FDA.

In a Phase 3 trial in patients with high-risk (secondary) AML, the median overall survival for patients treated with VYXEOS in the study was 9.56 months compared to 5.95 months for patients receiving the standard of care regimen of cytarabine and daunorubicin known as 7+3, representing a 3.61-month improvement in favor of VYXEOS. The hazard ratio (HR) was 0.69 (p=0.005), which represents a 31% reduction in the risk of death versus 7+3. The percentage of patients alive 12 months after randomization was 41.5% on the VYXEOS arm compared to 27.6% on the 7+3 arm. The percentage of patients alive 24 months after randomization was 31.1% on the VYXEOS arm compared to 12.3% on the 7+3 arm.

Sixty-day all-cause mortality was 13.7% versus 21.2%, in favor of patients treated with VYXEOS. No substantial difference in Grade 3 or higher adverse events was observed between VYXEOS and 7+3. In the intent-to-treat population, Grade 3-5, hematologic adverse events were similar for overall infections, febrile neutropenia, and bleeding events. In the intent-to-treat population, Grade 3-5, non-hematologic adverse events were similar across all organ systems, including cardiac, gastrointestinal, general systems, metabolic disorders, musculoskeletal, nervous system, respiratory, skin and renal.

Celator published results from two randomized, controlled, Phase 2 trials with VYXEOS. The first trial was conducted in newly diagnosed elderly AML patients and the second trial was conducted in patients with AML in first relapse

On May 19, 2016 Rosetta Genomics Ltd. (NASDAQ: ROSG), a leading developer and provider of microRNA-based and other molecular diagnostics, reported financial results for the three months ended March 31, 2016.

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Recent developments include:
Expanded molecular diagnostics test menu with the launch of three new product offerings in common hematologic cancers and solid tumors;

Received conditional approval status from the New York State Department of Health (NYSDOH) for RosettaGX Reveal, the Company’s novel microRNA classifier for the diagnosis of indeterminate thyroid Fine Needle Aspirate (FNA) smears;
Entered into an agreement that establishes health insurance access to Rosetta’s entire suite of diagnostic tests and services with America’s Choice Provider Network (ACPN), an independent multispecialty national provider network; and

Granted U.S. patent allowance for use of gene expression signature for classification of kidney tumors and granted European patent allowance for use of microRNA molecules for the treatment of liver cancer.

Management Commentary
"We are especially pleased to report record quarterly clinical testing revenues as it demonstrates the progress we have made in expanding our molecular diagnostics test menu, selling our clinical testing products and improving collections," said Kenneth A. Berlin, President and Chief Executive Officer of Rosetta Genomics. "Throughout the first quarter we completed the revamping of our sales force and invested in our billing and collections department. The results are reflected in our growing revenue and expanding customer base, as well as in improved collections. Further, these changes position us to drive revenue growth throughout the balance of the year and beyond.

"The commercial launch of RosettaGX Reveal continues to be a prime focus for our team. We expect the positive performance data from our blinded validation study to be published in a peer-reviewed journal in the coming weeks. These data demonstrate exceptional performance and we anticipate that a journal publication will strongly support our reimbursement and sales efforts. In addition, our revamped sales team has been able to use RosettaGX Reveal to access new accounts to promote not only our exceptional thyroid offering, but also to promote our urologic cancer and solid tumor product lines. Since the beginning of the year, these promotional efforts resulted in the acquisition of over 30 thyroid customer accounts and over 60 new customer accounts for our urology and solid tumor businesses.

"Our work for the balance of the year will continue to focus on driving revenue growth in both our base business as well as with our new products, such as RosettaGX Reveal, expanding reimbursement, improving collections and advancing our clinical development programs, which should position us to achieve a number of important milestones that will enhance shareholder value," concluded Mr. Berlin.

First Quarter Financial
Results Please note that the pro forma comparisons below are meant to provide a comparison as if the PersonalizeDx acquisition occurred on January 1, 2015. The actual acquisition date was April 13, 2015.

Revenues for the first quarter of 2016 increased 711% to $2.6 million compared with revenues of $321,000 for the first quarter of 2015, and increased 27% compared with pro forma revenues of $2.1 million for the first quarter of 2015.
Revenues from urologic cancer testing services in the first quarter of 2016 were $1.4 million, an increase of 7% compared with pro forma revenues of $1.3 million for the first quarter of 2015, and represented approximately 54% of clinical testing revenues for the quarter.

Revenues from solid tumor testing services in the first quarter of 2016 increased 272% to $1.2 million compared with revenues of $321,000 for the first quarter of 2015, and increased 58% compared with pro forma revenues of $0.8 million in the first quarter of 2015. Solid tumor testing services represented nearly 46% of total clinical testing revenues during the first quarter of 2016, with the balance coming from RosettaGX Reveal.

Cost of revenues for the first quarter of 2016 increased to $1.7 million compared with $352,000 for the first quarter of 2015, due to the acquisition of PersonalizeDx leading to a higher volume of processed samples, as well as to increases in personnel and infrastructure.

Research and development expenses for the first quarter of 2016 increased to $842,000 from $748,000 for the first quarter of 2015, primarily due to increased activities in Thyroid and other areas.

Sales, marketing and business development expenses for the first quarter of 2016 increased to $1.9 million from $1.6 million in the prior-year period due to a larger commercial footprint as a result of the acquisition of PersonalizeDx.

General and administrative expenses for the first quarter of 2016 increased to $2.2 million compared with $1.4 million for the same period in 2015, with the increase primarily due to increased personnel and activities related to the acquisition of PersonalizeDx.
The operating loss for the first quarter of 2016 was $4.0 million, which included $230,000 of non-cash stock-based compensation expense, compared with an operating loss of $3.8 million for the first quarter of 2015, which included $276,000 of non-cash stock-based compensation expense.

The net loss for the first quarter of 2016 was $4.0 million, or $0.20 per ordinary share on 20.7 million weighted average shares outstanding, compared with a net loss for the first quarter of 2015 of $3.9 million, or $0.30 per ordinary share on 12.8 million weighted average shares outstanding.

On a non-GAAP basis, excluding $230,000 of non-cash stock-based compensation expense, the net loss for the first quarter of 2016 was $3.8 million, or $0.18 per ordinary share on 20.7 million weighted average shares outstanding. For the first quarter of 2015, excluding the $276,000 of non-cash stock-based compensation expense, the non-GAAP net loss was $3.6 million, or $0.28 per ordinary share on 12.8 million weighted average share outstanding.

Balance Sheet Highlights
As of March 31, 2016, Rosetta Genomics had cash, cash equivalents, restricted cash and shortterm bank deposits of $12.6 million compared with $13.6 million as of December 31, 2015. The Company used approximately $2.6 million in cash to fund operations during the first quarter of 2016, and collected approximately $2.7 million in cash from its clinical testing services in addition to $1.6 million in cash receipts from a licensing deal signed in December 2015. Based on the Company’s current operations and plans, which include a cost-reduction plan should it be unable to raise sufficient additional capital, if necessary, Rosetta Genomics expects its current cash position will fund operations for at least the next 12 months.

On May 19, 2016 AstraZeneca and its global biologics research and development arm, MedImmune, reported that they will provide an update on their extensive oncology pipeline at the annual meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) in Chicago, USA, on 3-7 June 2016 (Press release, AstraZeneca, MAY 19, 2016, View Source [SID:1234512562]).

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Highlights will include new data demonstrating the strength and versatility of AstraZeneca’s industry-leading line of DNA damage response (DDR) medicines in multiple types of cancer. New data will highlight the continued momentum behind AstraZeneca’s numerous immuno-oncology (IO) programmes, and showcase small-molecule developments including Tagrisso (osimertinib) in leptomeningeal (brain) disease and the highly-selective Bruton’s tyrosine kinase (BTK) inhibitor, acalabrutinib, in chronic lymphocytic leukaemia (CLL).

Sean Bohen, Executive Vice President, Global Medicines Development and Chief Medical Officer at AstraZeneca, said: "Oncology is a strategic priority for AstraZeneca because of the potential of our broad pipeline to offer transformational therapies in cancer care. At ASCO (Free ASCO Whitepaper), we will update on our next-generation portfolio focusing on DNA damage response as a breakthrough paradigm in cancer treatment, including new long-term overall survival data for Lynparza. Our increased commitment to DDR therapies complements developments in our exciting immuno-oncology pipeline, from which we are expecting clinical results over the coming year."

DDR: A promising scientific platform, a leading position for AstraZeneca

DDR is a term describing the network of cellular pathways that minimise the daily impact of DNA damage. Currently, many cancers are known to have defects in DDR pathways, which makes them dependent on and therefore, highly sensitive to inhibition of the remaining DDR pathways. Targeting DDR deficiencies to preferentially kill cancer cells, while minimising the impact on normal cells, has potential for more selective, better tolerated therapies to improve survival in multiple cancers.

AstraZeneca is developing a comprehensive pipeline of compounds that target molecular pathways across the DDR system. These include the PARP inhibitor Lynparza (olaparib); Wee1 inhibitor AZD1775, ATM inhibitor AZD0156; ATR inhibitor AZD6738, and Aurora B Kinase inhibitor AZD2811. These compounds act on different cell-cycle points to prevent tumour cells from reproducing.

At the ASCO (Free ASCO Whitepaper) congress, DDR presentations will highlight:

The potential for maintenance of DDR therapies as shown by Lynparza overall survival data from Study 19 in ovarian cancer (Abstract # 5501). This abstract has been selected as a "Best of ASCO (Free ASCO Whitepaper)" abstract.
Opportunities for combination approaches with DDR and immuno-oncology therapies as shown in a Phase I study of the PD-L1 inhibitor, durvalumab, in combination with Lynparza or a VEGFR inhibitor, cediranib, in women’s cancers (Abstract # 3015)
The importance of selecting patients with a DDR pathway defect using the right diagnostic tool (abstract #4041)
The potential of DDR therapies against multiple biological DDR targets in different tumour types, with studies of the highly-selective WEE1 inhibitor, AZD1775, in advanced high-grade serous ovarian cancer (Abstract # TPS5610), squamous cell carcinoma of the head and neck (SCCHN) (Abstract # TPS6106), advanced solid tumours (Abstract # TPS2608) and glioblastoma (GBM) (Abstract # 2008)
Immuno-Oncology: Robust development momentum on track for read-outs in H1 2017

AstraZeneca is leading in a number of first-line studies with its IO strategy, where combined PD-L1 and CTLA-4 blockade – through the combination of durvalumab and tremelimumab – may address a significant unmet medical need for cancer patients who may not benefit from PD-1 pathway drugs in monotherapy.

Key updates include presentations covering pre-clinical data, late-stage trials and biomarker research:

Early study results of durvalumab monotherapy in urothelial bladder cancer from Phase Ib Study 1108 (Abstract # 4502)
Final results from a Phase III study of tremelimumab in mesothelioma (Abstract # 8502)
New study results on safety and clinical activity of durvalumab as first-line treatment in non-small cell lung cancer (NSCLC) (Abstract # 9029)
Ongoing investigation of the potential synergistic effects of durvalumab and the CTLA-4 inhibitor, tremelimumab, in bladder cancer (DANUBE trial – Abstract # TPS4574) and SCCHN (KESTREL trial – Abstract # TPS6101)
Enhanced understanding of PD-L1 biomarker expression in relation to primary versus metastatic tumours and sample age (Abstract # 3025)
Tagrisso in brain metastasis; acalabrutinib in CLL

AstraZeneca’s strong heritage in developing innovative targeted small molecules was underscored by the recent approval of Tagrisso as the first indicated treatment for EGFR T790M mutation-positive metastatic NSCLC in the US, EU and Japan. At ASCO (Free ASCO Whitepaper), new data will highlight the importance of Tagrisso activity in leptomeningeal disease through its ability to penetrate the blood-brain barrier. Further presentations will show the growing role of circulating tumour DNA (ctDNA) testing for diagnosis and treatment monitoring.

Key updates will also include presentation on the potential of our potent, highly-selective BTK inhibitor, acalabrutinib, in chronic lymphocytic leukaemia (CLL):

Data from the BLOOM study of Tagrisso in patients with leptomeningeal disease as a complication of EGFRm-metastatic NSCLC (Abstract # 9002)
Intensive plasma ctDNA profiling in experimental trials to identify markers of acquired drug resistance (Abstract # 11530)
Acalabrutinib – preliminary results from a first-line study as first-line therapy in CLL (Abstract # 7521) and in a Phase II study in combination with pembrolizumab in metastatic pancreatic cancer (Abstract # 4130)

On May 19, 2016 Syros Pharmaceuticals reported that preclinical data on its lead program, SY-1425, in genomically defined subsets of patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) will be highlighted in an oral presentation at the 21st Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) taking place June 9-12 in Copenhagen, Denmark (Press release, Syros Pharmaceuticals, MAY 19, 2016, View Source [SID:1234512614]). The Company will also present new preclinical data on its first-in-class selective cyclin-dependent kinase 7 (CDK7) inhibitor, SY-1365, in acute leukemia.

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SY-1425 in Novel Genomically Defined Subset of AML and MDS Patients Using its gene control platform, Syros identified a subset of AML and MDS patients whose tumors have a highly specialized regulatory region of non-coding DNA, known as a superenhancer, associated with the RARA gene. The super-enhancer associated with RARA is believed to lead to over-production of the RARα transcription factor, locking cells in an immature, undifferentiated and proliferative state. Treatment with SY-1425, an oral, potent and selective agonist of RARα, appears to promote differentiation of cancer cells with the RARA-associated super-enhancer, inhibiting the cancer’s growth. The oral presentation at EHA (Free EHA Whitepaper) will detail SY-1425’s mechanism of action as well as in vitro and in vivo data showing that a biomarker for the RARA super-enhancer discovered by Syros is predictive of response to treatment with SY-1425 in models of AML, including a survival benefit observed in the mice with the RARA biomarker when treated with SY-1425. Syros is on track to advance SY-1425 into a Phase 2 trial in mid-2016 in subsets of AML and MDS patients whose tumors are positive for the RARA biomarker.

Date & Time: Sunday, June 12, from 9-9:15 a.m. CEST Presentation Title: Super-Enhancer Analysis Defines Novel AML and MDS Sub-Types Sensitive to SY-1425, a Potent and Selective RARα Agonist Session Title: AML Biology – Novel Targeted Therapies Presenter: Michael R. McKeown, Ph.D., Senior Scientist, Syros Pharmaceuticals Abstract Number: S807 Location: Bella Center, Auditorium 2

CDK7 Inhibition as a Novel Treatment Strategy for Acute Leukemia
Certain cancers, including AML and acute lymphoblastic leukemia (ALL), are dependent on high and constant expression of transcription factors for their growth and survival and have been shown to be particularly responsive to selective inhibition of the transcriptional kinase CDK7. The poster presentation at EHA (Free EHA Whitepaper) details preclinical data demonstrating that SY-1365, the Company’s first-in-class selective and potent CDK7 inhibitor, preferentially kills cancer cells by inducing robust and dose-dependent apoptosis in acute leukemia cell lines while not inducing apoptosis in non-cancerous cells. The data also show that SY-1365 produces a significant survival benefit in patient-derived xenograft models of AML. Syros expects to advance SY-1365 into a Phase 1/2 trial in the first half of 2017 in patients with acute leukemia, including AML and ALL.

Date & Time: Saturday, June 11, from 5:30-7 p.m. CEST Presentation Title: First-in-Class CDK7 Inhibitor Induces Robust Apoptosis in Acute Myeloid Leukemia and Demonstrates Durable In Vivo Efficacy Session Title: Acute Myeloid Leukemia – Biology 3 Presenter: Yoon J. Choi, Ph.D., Senior Scientist, Syros Pharmaceuticals Abstract Number: P558 Location: Bella Center, Hall H, Poster Area