On March 28, 2016 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a biopharmaceutical company dedicated to innovative therapeutics addressing cancer and other unmet medical needs, reported financial results for the three and 12 months ended December 31, 2015 (Filing, Q4/Annual, EntreMed, 2015, MAR 28, 2016, View Source [SID:1234510065]).

The Company reported a net loss of ($1.7 million), or ($0.05) per share, for the three months ended December 31, 2015. This compares with a net loss of ($1.6 million), or ($0.05) per share for the fourth quarter of 2014. The increase in net loss can be attributed to costs associated with enrolling patients in our Phase 2 Trial for ENMD-2076 in fibrolamellar carcinoma (FLC) during the fourth quarter of 2015, as well as increasing costs associated with expanding our China operations.

The net loss for the year ended December 31, 2015 was ($7.2 million), or ($0.22) per share, compared with a net loss of ($26.2) million or ($0.92) per share for 2014. The reported net loss for 2014 included a non-cash expense of $19.7 million for acquired in-process research and development associated with the September 2014 successful in-license of the rights for greater China of MARQIBO, ZEVALIN and EVOMELA from Spectrum Pharmaceuticals. The Company secured these rights primarily with equity and no cash up front. Excluding this non-cash expense, the net loss for 2014 would have been ($6.5 million), or ($0.23) per share.

As of December 31, 2015, CASI had cash and cash equivalents of $5.1 million. In January 2016, the Company completed an initial closing of a strategic financing and received net proceeds of $10.2 million.

Sara B. Capitelli, CASI’s Vice President, Finance and Principal Accounting Officer, commented, "Our research and development expenses for the fourth quarter increased over the prior year due to clinical trial costs associated with the initiation of our FLC trial in November 2015 and higher costs associated with our growing China operations during 2015. The decrease in general and administrative expenses compared with the previous year primarily reflects higher costs incurred in 2014 associated with our in-license of Spectrum products in 2014. We are continuing to execute our clinical development plans in the U.S. and China, and expect operating expenses to increase in 2016."

Further information regarding the Company, including its Annual Report on Form 10-K for the year ended December 31, 2015, can be found at www.casipharmaceuticals.com.

Ken K. Ren, Ph.D., Chief Executive Officer, commented, "Our financial results for the fourth quarter and year ended December 31, 2015 were as anticipated. In January 2016, we received $10.2 million net proceeds in an initial closing of a strategic financing which included common stock priced at $1.19 per share, the proceeds of which will support our product pipeline and advance our clinical and regulatory activities. Our import drug registration activities for MARQIBO, ZEVALIN and EVOMELA in China are ongoing, including the filing of our import registration trial application with CFDA for MARQIBO in January 2016. Our partner, Spectrum Pharmaceuticals, recently received FDA approval for EVOMELA which now allows us to advance the CFDA registration process towards an import registration trial and market approval in China. We continue to advance the clinical development of our lead proprietary drug candidate, ENMD-2076. Our Phase 2 trial in FLC is progressing well with 60% of patients already recruited for stage one of our 2-stage trial, and our Phase 2 trials in ovarian clear cell carcinoma, triple negative breast cancer and soft tissue sarcoma continue to progress along with correlative biomarker analysis. We continue to carefully manage our expenses, while achieving interim milestones towards our mission to become a fully-integrated pharmaceutical company with a rich product pipeline."

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DNA structural lesions are prevalent in sporadic colorectal cancer. Therefore, we proposed that gene variants that predispose to DNA double-strand breaks (DSBs) would be found in patients with familial colorectal carcinomas of an undefined genetic basis (UFCRC).
We collected primary T cells from 25 patients with UFCRC and matched patients without colorectal cancer (controls) and assayed for DSBs. We performed exome sequence analyses of germline DNA from 20 patients with UFCRC and 5 undiagnosed patients with polyposis. The prevalence of identified variants in genes linked to DNA integrity was compared with that of individuals without a family history of cancer. The effects of representative variants found to be associated with UFCRC was confirmed in functional assays with HCT116 cells.
Primary T cells from most patients with UFCRC had increased levels of the DSB marker γ(phosphorylated)histone2AX (γH2AX) after treatment with DNA damaging agents, compared with T cells from controls (P < .001). Exome sequence analysis identified a mean 1.4 rare variants per patient that were predicted to disrupt functions of genes relevant to DSBs. Controls (from public databases) had a much lower frequency of variants in the same genes (P < .001). Knockdown of representative variant genes in HCT116 CRC cells increased γH2AX. A detailed analysis of immortalized patient-derived B cells that contained variants in the Werner syndrome, RecQ helicase-like gene (WRN, encoding T705I), and excision repair cross-complementation group 6 (ERCC6, encoding N180Y) showed reduced levels of these proteins and increased DSBs, compared with B cells from controls. This phenotype was rescued by exogenous expression of WRN or ERCC6. Direct analysis of the recombinant variant proteins confirmed defective enzymatic activities.
These results provide evidence that defects in suppression of DSBs underlie some cases of UFCRC; these can be identified by assays of circulating lymphocytes. We specifically associated UFCRC with variants in WRN and ERCC6 that reduce the capacity for repair of DNA DSBs. These observations could lead to a simple screening strategy for UFCRC, and provide insight into the pathogenic mechanisms of colorectal carcinogenesis.
Copyright © 2015 AGA Institute. Published by Elsevier Inc. All rights reserved.

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Controversy exists as to whether erectile response to phosphodiesterase type 5 inhibitors is compromised in men with low total testosterone (TT) levels. This is amplified by reports of improved response to phosphodiesterase type 5 inhibitor therapy after coadministration of testosterone replacement therapy in hypogonadal men unresponsive to phosphodiesterase type 5 inhibitors.
To determine whether TT and luteinizing hormone levels influence efficacy of tadalafil for erectile dysfunction in men with concomitant lower urinary tract symptoms and benign prostatic hyperplasia.
This integrated analysis included 1,075 men randomized to once-daily tadalafil 5 mg (n = 540) or placebo (n = 535) for 12 weeks in three prospective clinical trials who had not received concomitant testosterone replacement therapy. Subjects were categorized at baseline by low vs normal TT levels (n = 1,049; <300 vs ≥300 ng/dL) and normal vs high luteinizing hormone levels (n = 1,058; ≤9.4 vs >9.4 mIU/mL). Treatment-group differences in International Index of Erectile Function (IIEF) by hormone subgroups were assessed using analysis of covariance.
Changes in IIEF erectile function domain and other domain scores.
The overall study population was comprised primarily of white men (>86%) with a mean age range of 64 to 70 years. Median baseline TT level in the integrated population was 355 ng/dL; levels were lower than 300 ng/dL (cutoff for normal) in 32.4% of men. Men with low TT levels reported diabetes (21.8%), cardiovascular disease (54.1%), and hypertension (49.1%) numerically more often than men with normal TT levels (10.6%, 43.2%, and 36.7%, respectively). Low TT and high luteinizing hormone levels were associated with numerically, but not statistically significantly, lower 12-week IIEF domain scores compared with those with normal levels. Changes in most 12-week IIEF domain scores showed that tadalafil was significantly more effective than placebo (P < .02).
Low TT levels at baseline did not negatively influence response to tadalafil in men of advancing age with concomitant lower urinary tract symptoms and benign prostatic hyperplasia and erectile dysfunction.
Copyright © 2016 International Society for Sexual Medicine. Published by Elsevier Inc. All rights reserved.

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Reported here is the application of silver nanoparticle-based surface-enhanced Raman spectroscopy (SERS) as a label-free, non-invasive technique for detection of oral squamous cell cancer (OSCC) using saliva and desquamated oral cells. A total of 180 SERS spectra were acquired from saliva and 120 SERS spectra from oral cells collected from normal healthy individuals and from confirmed oropharyngeal cancer patients. Notable biochemical peaks in the SERS spectra were tentatively assigned to various components. Data were subjected to multivariate statistical techniques including principal component analysis and linear discriminate analysis (PCA-LDA) revealing a sensitivity of 89% and 68% and a diagnostic accuracy of 73% and 60% for saliva and oral cells, respectively. The results from this study demonstrate the potential of saliva and oral cell SERS combined with PCA-LDA diagnostic algorithms as a promising clinical adjunct for the non-invasive detection of oral cancer.
Copyright © 2015. Published by Elsevier Inc.

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To determine the feasibility and clinical utility of using comprehensive genomic profiling (CGP) in the course of clinical care to identify clinically relevant tumor genomic alterations for patients with either rare or refractory gynecologic cancers to facilitate point-of-care management. Use of an expert, multidisciplinary, institutional molecular tumor board (MTB) assessment is discussed regarding input on putative targeted options for individualized therapy.
A prospective clinical trial is ongoing. We report on the initial 69 patients with gynecologic cancers that were either rare or refractory to standard therapy. CGP was performed by Foundation Medicine, Inc. Genomic alterations were reviewed by members of an MTB. Consensus recommendations on genomically targeted, FDA-approved, on- and off-label therapies and clinical trials were sent to the treating physician, and decisions and outcomes were assessed.
Study outcomes were available for 64 patients. The mean number of genes altered per tumor was 4.97 (median=4; range, 1-26), and the average turnaround time from testing laboratory report to generation of formal recommendations was approximately three weeks. Evaluation of genomic and clinical data by the MTB led to generation of targeted treatment options in all 64 patients, and the percentage of patients for whom one or more of these recommendations were implemented by the treating physician was 39%. Sixty-four percent of the patients receiving targeted therapy based on a CGP result experienced radiologic response or showed evidence of clinical benefit or stable disease.
These data suggest that an institutional MTB is a feasible venue for reviewing tumor genomic profiling results and generating clinical recommendations. These data also support the need for further studies and guidelines on clinical decision making with greater availability of broad genomically based diagnostics.
Copyright © 2016 Elsevier Inc. All rights reserved.

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