On March 30, 2016 Amal Therapeutics (Amal) reported that they have completed CHF 3 million (EUR 2.75 million) Series A financing round with Boehringer Ingelheim Venture Fund (BIVF) as cornerstone investors (Press release, Boehringer Ingelheim, MAR 29, 2016, View Source [SID:1234510134]). VI Partners and High-Tech Gründerfonds also participated in the round which will help progress Amal’s cancer vaccines.

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Amal Therapeutics is a Swiss biotech company developing and progressing therapeutic cancer vaccines. The company will use the funds to progress the preclinical development of its lead vaccine (ATP124) for colorectal cancer and to further develop its KISIMA technology platform for therapeutic tumor vaccination. This novel technology is able to generate potent long lasting anti-tumor immunity and avoid tumor immune escape.

Dr. Frank Kalkbrenner from the Boehringer Ingelheim Venture Fund and Dr. Frank Hensel from the High-Tech Gruenderfonds will join the Board as Board Observers. Dr. Diego Braguglia from VI Partners will also become a member of Amal’s Supervisory Board.

Dr. Madiha Derouazi, CEO and founder of Amal Therapeutics said: "This Series A investment recognizes the potential of the KISIMA technology platform and the value of our scientific assets. We are now in a position to rapidly progress our lead vaccine, ATP124, for colorectal cancer into the clinic and continue to develop our pipeline in other cancer indications. I look forward to working with the Board to make Amal Therapeutics a leading innovator in the field of immunotherapies".

Dr. Knut Elbers, Boehringer Ingelheim Venture Fund representative on Amal’s Board added: "Dr. Derouazi’s dedicated leadership and scientific expertise allowed us to assemble a group of outstanding scientists to show proof-of-principle in a series of pre-clinical tumor studies. We are confident that Amal’s KISIMA technology could revolutionize the peptide-based cancer vaccine field – bringing an exciting new technology to the patient".

Dr. Braguglia, VI Partners: "We believe that the KISIMA technology is superior to many other tumor vaccine technologies, both as a stand-alone treatment and in combination with other vaccines or immuno-oncology treatment modalities. I’m pleased to be joining the current seasoned investors and support the team of Amal in moving its technology into the clinic".

Amal’s vaccines combine a Cell Penetrating Peptide (CPP) with a multi-antigenic chimeric cargo with various CD8+ and CD4+ epitopes and a constitutive activator of dendritic cells, enabling them to simultaneously stimulate multi-epitopic cytotoxic T cell-mediated immunity, induce helper T (Th) cells and promote immunological memory.

About Amal Therapeutics SA

Chronic hepatitis B infection (CHB) is characterized by sub-optimal T cell responses to viral antigens. A therapeutic vaccine capable of restoring these immune responses could potentially improve HBsAg seroconversion rates in the setting of direct acting antiviral therapies. A yeast-based immunotherapy (Tarmogen) platform was used to make a vaccine candidate expressing hepatitis B virus (HBV) X, surface (S), and Core antigens (X-S-Core). Murine and human immunogenicity models were used to evaluate the type and magnitude of HBV-Ag specific T cell responses elicited by the vaccine. C57BL/6J, BALB/c, and HLA-A*0201 transgenic mice immunized with yeast expressing X-S-Core showed T cell responses to X, S and Core when evaluated by lymphocyte proliferation assay, ELISpot, intracellular cytokine staining (ICS), or tumor challenge assays. Both CD4+ and CD8+ T cell responses were observed. Human T cells transduced with HBc18-27 and HBs183-91 specific T cell receptors (TCRs) produced interferon gamma (IFNγ following incubation with X-S-Core-pulsed dendritic cells (DCs). Furthermore, stimulation of peripheral blood mononuclear cells (PBMCs) isolated from CHB patients or from HBV vaccine recipients with autologous DCs pulsed with X-S-Core or a related product (S-Core) resulted in pronounced expansions of HBV Ag-specific T cells possessing a cytolytic phenotype. These data indicate that X-S-Core-expressing yeast elicit functional adaptive immune responses and supports the ongoing evaluation of this therapeutic vaccine in patients with CHB to enhance the induction of HBV-specific T cell responses.

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Leuprorelin acetate, a luteinizing hormone-releasing hormone agonist, is used worldwide in premenopausal women with hormone receptor-positive breast cancer. This study was conducted to assess the non-inferiority of the 6-month depot formulation, TAP-144-SR (6M) 22.5 mg to the 3-month depot formulation, TAP-144-SR (3M) 11.25 mg in postoperative, premenopausal patients with hormone receptor-positive breast cancer.
This was a 96-week phase III, randomized, open-label, parallel-group comparative study. All patients concomitantly received oral tamoxifen (20 mg daily). The primary endpoint was the suppression rate of serum estradiol (E2) to the menopausal level (≤30 pg/mL) from Week 4 through Week 48.
In total, 167 patients were randomized to receive TAP-144-SR (6M) (n = 83) or TAP-144-SR (3M) (n = 84) and the E2 suppression rate was 97.6 and 96.4 %, respectively. The estimated between-group difference was 1.2 % (95 % confidence interval -5.2 to 7.8). The non-inferiority of TAP-144-SR (6M) to TAP-144-SR (3M) for E2 suppression was confirmed. As for safety, common adverse events were hot flush and injection site reactions including induration, pain, and erythema in both treatment groups, which were of ≤Grade 2 in severity and not serious. No significant between-group differences in safety profiles and tolerability were observed.
TAP-144-SR (6M) was not inferior to TAP-144-SR (3M) for its suppressive effect on serum E2. TAP-144-SR (6M) was also as well tolerated as TAP-144-SR (3M).

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Control of serum uric acid (sUA) levels is very important during chemotherapy in patients with malignant tumors, as the risks of tumor lysis syndrome (TLS) and renal events are increased with increasing levels of sUA. We investigated the efficacy and safety of febuxostat, a potent non-purine xanthine oxidase inhibitor, compared with allopurinol for prevention of hyperuricemia in patients with malignant tumors, including solid tumors, receiving chemotherapy in Japan.
An allopurinol-controlled multicenter, open-label, randomized, parallel-group comparative study was carried out. Patients with malignant tumors receiving chemotherapy, who had an intermediate risk of TLS or a high risk of TLS and were not scheduled to be treated with rasburicase, were enrolled and then randomized to febuxostat (60 mg/day) or allopurinol (300 or 200 mg/day). All patients started to take the study drug 24 h before chemotherapy. The primary objective was to confirm the non-inferiority of febuxostat to allopurinol based on the area under the curve (AUC) of sUA for a 6-day treatment period.
Forty-nine and 51 patients took febuxostat and allopurinol, respectively. sUA decreased over time after initiation of study treatment. The least squares mean difference of the AUC of sUA between the treatment groups was -33.61 mg h/dL, and the 95 % confidence interval was -70.67 to 3.45, demonstrating the non-inferiority of febuxostat to allopurinol. No differences were noted in safety outcomes between the treatment groups.
Febuxostat demonstrated an efficacy and safety similar to allopurinol in patients with malignant tumors receiving chemotherapy.
View Source ; Identifier: JapicCTI-132398.

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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.