On March 28, 2016 Diffusion Pharmaceuticals Inc. (OTCQX:DFFN) a clinical stage biotechnology company focused on the development of novel small molecule therapeutics for cancer, reported financial results for the year ended December 31, 2015 and provided an overview of recent operational highlights (Press release, Diffusion Pharmaceuticals, MAR 28, 2016, View Source [SID:1234510058]).

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David Kalergis, Chairman and Chief Executive Officer of Diffusion Pharmaceuticals, said, "2015 was a transformational year for Diffusion. In May, we completed the Phase 2 trial of our lead drug trans sodium crocetinate (TSC) in glioblastoma (GBM) brain cancer with positive top-line results. In August, we received the FDA’s agreement on a Phase 3 clinical trial plan, which could support registration of TSC based on a single pivotal trial. In December, we entered into a merger agreement with RestorGenex Corporation (now known as Diffusion Pharmaceuticals Inc.) and, upon the merger’s closing on January 8, 2016, became a public company. Looking further ahead into this coming year, we have assembled a clinical trial advisory committee of global experts to guide us through our upcoming discussions with the FDA regarding expansion of the use of TSC from GBM into pancreatic cancer, one of the most devastating of all the cancers."

Operational Highlights

On January 8, 2016, Diffusion Pharmaceuticals LLC completed a reverse merger with RestorGenex Corporation in an all-stock transaction. Following the close of the reverse merger, RestorGenex was renamed Diffusion Pharmaceuticals Inc. and the Company’s ticker symbol was changed to "DFFN". In conjunction with the merger, holders of outstanding units of Diffusion Pharmaceuticals LLC were issued approximately 83.0 million shares of common stock in the renamed publicly traded company, bringing the total number of shares outstanding to approximately 101.6 million. The issuance of the 83.0 million new shares thus allowed the Company to add an FDA Phase 3-ready asset to what had previously been a preclinical drug development pipeline.
As a result of the new stock issuance, former equity holders of Diffusion Pharmaceuticals LLC owned approximately 84.1% of the Common Stock and the shareholders of former RestorGenex owned approximately 15.9% of the Common Stock immediately following the merger’s closing on a fully-diluted basis (subject to certain exceptions and adjustments). The 83.0 million newly issued shares of Diffusion Pharmaceuticals Inc., now belonging to former Diffusion Pharmaceuticals LLC holders, have not been registered with the SEC and therefore are not eligible for public trading at this time. The Company has agreed to file a registration statement covering the resale of these shares within 270 days of the merger closing.

Lead Candidate: Diffusion is continuing to advance the clinical development of its lead candidate TSC. TSC has demonstrated positive results in a Phase 2 clinical trial in patients with newly diagnosed GBM. The U.S. Food and Drug Administration (FDA) has granted orphan drug designation for TSC for the treatment of GBM. At the End-of-Phase-2 meeting with the FDA in August 2015, Diffusion reached an agreement with the agency on the design of a single Phase 3 study which, if successful, would be sufficient to support registration. The Company intends to commence the Phase 3 trial within the next 12 months, contingent on the availability of financial resources and the completion of certain manufacturing and animal toxicology guidelines mandated by the FDA agreement.

Pancreatic Cancer Indication: Diffusion is currently in discussions with the FDA regarding a planned Phase 2/3 clinical trial for TSC in pancreatic cancer. The Company has assembled a clinical advisory committee of key opinion leaders in the field of pancreatic cancer to facilitate the development of the program. The Company anticipates commencing the trial in the first half of 2017, assuming the availability of financial resources.

Metastatic Brain Cancer Indication: The Company is also planning a Phase 2/3 clinical trial program with TSC in metastatic brain cancer, an indication for which it has also received orphan drug designation.

Year End 2015 Results
The financial statements for the year ended December 31, 2015 filed Friday, March 25, 2016 via Form 10- K pertain solely to legacy RestorGenex’s operations. Financial statements filed Friday, March 25, 2016 via Form 8-K/A pertain to legacy Diffusion Pharmaceuticals LLC’s operations as of and for the year ended December 31, 2015 and pre-date the closing of the reverse merger on January 8, 2016. The financial results discussed below pertain to legacy Diffusion Pharmaceuticals LLC’s 2015 operations, as reported in the Form 8-K/A, which also includes pro-forma financial statements for the combined company.

Research and development expenses were $3.9 million for the year ended December 31, 2015, compared to $2.0 million in the prior year. The increase was attributable primarily to the clinical development activities for the TSC Phase 2 clinical trial in GBM that was completed in the second quarter 2015.

General and administrative expenses were $2.5 million for the year ended December 31, 2015, compared to $1.3 million for the year ended December 31, 2014. The increase was attributed primarily to costs associated with the merger transaction.

Net loss was $6.7 million for the year ended December 31, 2015, compared to a net loss of $3.5 million for the year ended December 31, 2014.

Cash, cash equivalents, and certificates of deposit were $2.0 million as of December 31, 2015, compared to $4.8 million as of December 31, 2014. On a pro forma combined basis in conjunction with the closing of the merger with RestorGenex, cash, cash equivalents, and certificates of deposit were $14.0 million for the year ended December 31, 2015.

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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Despite progress in the treatment of chemotherapy/radiotherapy-induced nausea and vomiting (CINV/RINV), their management remains insufficient.
In order to evaluate the incidence and impact of CINV/RINV on the quality of life perceived by patients and estimated by clinicians, a declarative, cross-sectional survey was conducted in France through an online questionnaire.
This survey included 187 participants: 75 oncologists, 35 oncology nurses and 77 patients. Clinicians over-estimated the incidence of CINV/RINV, but underestimated their impact on the quality of life of patients. The sub-optimal prescription of anti-emetic treatments was more prominent when the therapy administered had low or medium emetogenic potential. Only 30% of patients rated their nausea and vomiting as controlled from the start. A major proportion of patients (68%) declared poor compliance with their anti-emetic regimen. The acceptance of CINV/RINV as normal side effects of the chemotherapy/radiotherapy (51%) led the patients not to report them, thus limiting their active management. The number of drugs to absorb, and the fear that the action of swallowing the pill would induce nausea or vomiting were also quoted by the patients as compliance-limiting factors.
The perceptual gap between clinicians and patients regarding the incidence and impact of CINV/RINV contributes to a sub-optimal level of anti-emetic cover and control. The anti-emetic regimen needs to be regularly assessed and adapted to the patient in order to improve CINV/RINV management.
Copyright © 2016 Société Française du Cancer. Published by Elsevier Masson SAS. All rights reserved.

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Patient-reported outcomes have been associated with survival in numerous studies across cancer types, including breast cancer. However, the Brief Pain Inventory-Short Form (BPI-SF) and the Rotterdam Symptom Checklist (RSCL) have rarely been investigated in this regard in breast cancer.
Here we describe a post hoc analysis of the prognostic effect of baseline scores of these instruments on survival in a phase III trial of patients with advanced breast cancer who received gemcitabine plus paclitaxel or paclitaxel alone after anthracycline-based adjuvant or neoadjuvant therapy. The variables for this analysis were baseline BPI-SF "worst pain" and BPI-SF "pain interference" scores, and four RSCL subscales (each transformed to 0-100). Univariate and multivariate Cox models were used, the latter in the presence of 11 demographic/clinical variables. Kaplan-Meier curves and log-rank tests were used to compare survival for patients by BPI-SF or RSCL scores.
Of 529 randomized patients, 286 provided BPI-SF data and 336 provided RSCL data at baseline. Univariate analyses identified BPI-SF worst pain and pain interference (both hazard ratios [HR], 1.07 for a 1-point increase; both p ≤ 0.0061) and three of four RSCL subscales [activity level, physical distress, and health-related quality of life (HRQOL) (HR, 0.86-0.91 for 10-point increase all p ≤ 0.0104)], to have significant prognostic effect for survival. BPI-SF worst pain (p = 0.0342) and RSCL activity level (p = 0.0004) were prognostic in the multivariate analysis. Median survival for patients categorized by BPI-SF worst pain score was 23.8 (n = 91), 17.9 (n = 94) and 14.6 (n = 94) months for scores 0, 1-4, and 5-10, respectively (log-rank p = 0.0065). Median survival was 23.8 and 14.6 months for patients (n = 330) with above- and below-median RSCL activity level scores respectively (log-rank p < 0.0001).
Pretreatment BPI-SF worst pain and RSCL activity scores provide distinct prognostic information for survival in patients receiving paclitaxel or gemcitabine plus paclitaxel for advanced breast cancer even after controlling for multiple demographic and clinical factors.
Clinicaltrials.gov, NCT00006459 .

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