20-F – Annual and transition report of foreign private issuers [Sections 13 or 15(d)]

(Filing, Annual, Nymox, 2016, MAR 31, 2017, View Source [SID1234518382])

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Diffusion Pharmaceuticals Provides Corporate Highlights and Reports 2016 Financial Results

On March 31, 2017 Diffusion Pharmaceuticals Inc. (NASDAQ:DFFN), a clinical stage biotechnology company focused on the development of novel small molecule therapeutics for cancer and other hypoxia-related diseases, reported financial results for the full year of 2016 and provided an overview of recent operational highlights (Press release, Diffusion Pharmaceuticals, MAR 31, 2017, View Source [SID1234518358]).

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David Kalergis, Chairman and Chief Executive Officer, stated: "The recently received proceeds from our private placement provide Diffusion with resources to move forward with our Phase 3 clinical program testing of trans sodium crocetinate (TSC) in newly diagnosed GBM patients, as well as advance its use in other indications. Diffusion staff, expert consultants and key opinion leaders continue to work together to craft the most cost-effective programs with optimal chances for success."

Corporate Highlights

In November 2016, our common stock was approved for listing, and commenced trading on the NASDAQ Capital Market;

In February 2017, data from our Phase 1/2 clinical trial evaluating the safety and efficacy of TSC in newly diagnosed glioblastoma multiforme was published in the print edition of the peer-reviewed Journal of Neurosurgery;

In March 2017, we completed an initial closing of our Series A Convertible Preferred Stock offering to accredited investors in private placement;

In March 2017, U.S. Patent 9,604,899 entitled "Bipolar Trans Carotenoid Salts and Their Uses" was granted by the United States Patent and Trademark Office. This patent expands the coverage of the therapeutic use of TSC and other related compounds to five hypoxia-related conditions including congestive heart failure, chronic renal failure, acute lung injury (ALI), chronic obstructive pulmonary disease (COPD) and respiratory distress syndrome (RDS).
Year End 2016 Results

Research and development expenses were $7.3 million during the year ended December 31, 2016, compared to $3.9 million during the year ended December 31, 2015. This increase was primarily a result of an additional $1.2 million in expenses related to animal toxicology studies, an increase of $1.0 million in active product ingredient manufacturing costs and an additional $0.5 million in costs related to the TSC pancreatic cancer program. Additionally, a $1.0 million noncash impairment charge was recognized for the abandonment of future development efforts related to the RES-440 IPR&D asset. Salaries and wages expense and stock compensation expense increased by $0.3 million and $0.4 million, respectively, due to an increase in headcount. The overall increase in research and development expense was offset by a $0.9 million decrease in spend related to GBM trials.

General and administrative expenses were $11.1 million for the year ended December 31, 2016, compared to $2.5 million for the year ended December 31, 2015. The increase was primarily attributable to $4.1 million in professional fees incurred in connection with preparing to operate as a public company, merger and transaction related fees and fees related to investment bank advisory services. There was also a $2.5 million noncash charge recognized upon settlement of a litigation matter. In addition, insurance expense increased by $0.8 million due to an increase in directors and officer’s insurance and salaries and wages and stock compensation expense increased by $0.4 million and $0.4 million, respectively, due to an increase in headcount.

Net loss was $18.0 million for the year ended December 31, 2016, compared to a net loss of $6.7 million for the year ended December 31, 2015. The increase in the net loss was primarily due to higher expenses associated with research and development and general and administrative costs.

Cash and cash equivalents were $1.6 million as of December 31, 2016, compared to $2.0 million as of December 31, 2015.

Kite Completes Submission of U.S. Biologics License Application (BLA) for Axicabtagene Ciloleucel as the First CAR-T Therapy for the Treatment of Patients With Aggressive Non-Hodgkin Lymphoma (NHL)

On March 31, 2017 Kite Pharma, Inc. (Nasdaq:KITE) reported that it has completed the rolling submission with the U.S. Food and Drug Administration (FDA) of the Biologics License Application (BLA) for axicabtagene ciloleucel (previously known as KTE-C19) as a treatment for patients with relapsed or refractory aggressive non-Hodgkin lymphoma (NHL) who are ineligible for autologous stem cell transplant (ASCT) (Press release, Kite Pharma, MAR 31, 2017, View Source [SID1234518361]).

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"Last month, we announced positive results from our ZUMA-1 pivotal trial with axicabtagene ciloleucel," said Arie Belldegrun, M.D., FACS, Chairman, President, and Chief Executive Officer of Kite. "We look forward to working closely with the FDA during the review of axicabtagene ciloleucel and the possibility of bringing this therapy to patients with aggressive NHL whose outlook is dismal with current therapy."

In December 2015 axicabtagene ciloleucel received Breakthrough Therapy Designation (BTD) by the FDA for diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL). If approved, Kite plans to commercially launch axicabtagene ciloleucel in 2017. Kite is also planning a regulatory submission to the European Medicines Agency (EMA) for axicabtagene ciloleucel in 2017.

"The Leukemia & Lymphoma Society (LLS) applauds Kite for achieving this significant milestone and bringing this promising therapy closer to patients with lymphoma who desperately need new options," said Louis J. DeGennaro, Ph.D., LLS President and Chief Executive Officer. "LLS has supported companies that are working to dramatically change cancer treatment through the development of immunotherapy for the past two decades, and we immediately recognized the great opportunity to support Kite’s CAR-T program in 2015 through our Therapy Acceleration Program (TAP). Partnerships created through TAP, now in its tenth year, have the potential to bring several breakthrough therapies, such as axicabtagene ciloleucel, to patients in the coming year."

The ZUMA-1 pivotal trial for axicabtagene ciloleucel for the treatment of patients with aggressive NHL was supported in part by funding from LLS’ TAP.

About axicabtagene ciloleucel

Kite’s lead product candidate, axicabtagene ciloleucel, is an investigational therapy in which a patient’s T cells are engineered to express a chimeric antigen receptor (CAR) to target the antigen CD19, a protein expressed on the cell surface of B-cell lymphomas and leukemias, and redirect the T cells to kill cancer cells. Axicabtagene ciloleucel has been granted Breakthrough Therapy Designation status for diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL) by the U.S. Food and Drug Administration (FDA) and Priority Medicines (PRIME) regulatory support for DLBCL in the EU.

Loxo Oncology Announces Proof of Concept Clinical Data for Larotrectinib in TRK Fusion Glioblastoma Presented at the AACR Annual Meeting 2017

On March 31, 2017 Loxo Oncology, Inc. (Nasdaq:LOXO), a biopharmaceutical company innovating the development of highly selective medicines for patients with genetically defined cancers, reported that larotrectinib (LOXO-101) data will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2017 Annual Meeting taking place April 1 – 5, 2017, in Washington, DC (Press release, Loxo Oncology, MAR 31, 2017, View Source [SID1234518362]).

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The abstract and poster describe initial clinical data across the larotrectinib program for all patients with TRK fusion primary CNS cancers. The cases include three patients with glioblastoma: one patient treated under an expanded access protocol and two patients treated in the ongoing Phase 2 NAVIGATE trial. In the cases described, larotrectinib showed preliminary evidence of anti-tumor activity. The expanded access patient, described in detail in the abstract, had progressed through prior radiation, temozolomide and bevacizumab and demonstrated a brief mixed radiographic response on larotrectinib in the context of a molecularly complex tumor (select regions of the tumor harbored a TRK fusion, while others did not). The two patients treated in NAVIGATE, described in the poster, were enrolled following progression on chemoradiation and temozolomide (both cases) and bevacizumab (one case). The NAVIGATE patients remain on therapy at four months with radiographic evidence of treatment effect, as of a March 13, 2017 data cut-off date. Glioblastomas are highly aggressive CNS tumors, particularly in the post-bevacizumab setting where median overall survival is typically three to six months. Global regulatory discussions have established that primary CNS tumors, including glioblastoma, will not be included in the primary efficacy analysis dataset intended to support initial drug approval, though they are being enrolled in a dedicated treatment arm of NAVIGATE.

"Glioblastomas have historically defied rational targeted therapy approaches, so we are encouraged that larotrectinib may have a role in treating TRK fusion presentations of this devastating disease. We hope these early data lead to increased molecular profiling and referrals to appropriate clinical trials," said Josh Bilenker, M.D., chief executive officer of Loxo Oncology. "We knew it would be necessary to evaluate these patients separately from our primary efficacy dataset, which relies on a RECIST overall response rate primary endpoint. Given the limitations around response assessment in neuro-oncology, randomized survival trials remain the gold standard for evaluating new drugs in primary CNS cancers. However, patients with systemic tumors that have metastasized to the brain are included in our primary efficacy dataset, though they have been exceedingly uncommon. As illustrated in our adult Phase 1 dataset and this AACR (Free AACR Whitepaper) poster, we are pleased with larotrectinib’s ability to enter the CNS in a tolerated fashion and address TRK fusion tumors."

Abstracts from the AACR (Free AACR Whitepaper) Annual Meeting 2017 are available online on the conference website.

The details of the poster presentation are as follows:

Date: April 5, 2017, 8:00am – 12:00pm ET
Title: Potential role of larotrectinib (LOXO-101), a selective pan-TRK inhibitor, in NTRK fusion-positive recurrent glioblastoma
Session: Late-Breaking Research: Experimental and Molecular Therapeutics II
Abstract Code: LB-302
Poster Board Number: 10
Session Location: Poster Section 34

About Larotrectinib (LOXO-101)
Larotrectinib (LOXO-101) is a potent, oral and selective investigational new drug in clinical development for the treatment of patients with cancers that harbor abnormalities involving the tropomyosin receptor kinases (TRKs). Growing research suggests that the NTRK genes, which encode for TRKs, can become abnormally fused to other genes, resulting in growth signals that can lead to cancer in many sites of the body. In an ongoing Phase 1 clinical trial, larotrectinib has demonstrated encouraging preliminary efficacy. Larotrectinib is also being evaluated in the NAVIGATE global Phase 2 multi-center basket trial in patients with solid tumors that harbor TRK gene fusions, and the SCOUT Phase 1/2 trial in pediatric patients, including patients with advanced cancer, TRK gene fusions and infantile fibrosarcoma. Larotrectinib has been granted Breakthrough Therapy Designation and Rare Pediatric Disease Designation by the U.S. FDA. For additional information about the larotrectinib clinical trials, please refer to www.clinicaltrials.gov. Interested patients and physicians can contact the Loxo Oncology Physician and Patient Clinical Trial Hotline at 1-855-NTRK-123 or visit www.loxooncologytrials.com.

Phase II Copanlisib Data Show Durable Tumor Response in Indolent Non-Hodgkin’s Lymphoma

On March 31, 2017 Bayer reported positive data on its investigational compound copanlisib, an intravenous pan-class I phosphatidylinositol-3-kinase (PI3K) inhibitor with predominant inhibitory activity against PI3K-α and PI3K-δ isoforms (Press release, Bayer, MAR 31, 2017, View Source;dcp=Phase-II-Copanlisib-Data-Show-Durable-Tumor-Response-in-Indolent-Non-Hodgkins-Lymphoma&ccm= [SID1234518413]). The Phase II CHRONOS-1 trial, an open-label, single-arm study of copanlisib evaluating patients with relapsed or refractory indolent non-Hodgkin’s lymphoma (iNHL), met its primary endpoint of a pre-specified objective response rate (ORR). The results across all patient groups show an ORR of 59.2%, with a 12% complete response (CR) rate and a median duration of response (DOR) of more than 98 weeks (687 days). These data will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2017 Annual Meeting in Washington, D.C., USA in the Novel Agent and Intervention Clinical Trials session on April 4, 2017 from 3:05 PM – 3:20 PM (ET).

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"Based on the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) guidelines, inhibition of the PI3K pathway has been shown to be an effective therapeutic strategy in treating indolent lymphomas, like follicular lymphoma; however, concerns exist about the safety of available oral PI3K inhibitors, highlighting the need for new approaches," said Martin Dreyling, Professor of Medicine at the University of Munich Hospital in Grosshadern. "The results of CHRONOS-1 demonstrate that intermittent intravenous administration of copanlisib achieved durable efficacy with a manageable safety profile in this difficult-to-treat patient population."

The full analysis set comprised 142 patients, of which 141 patients had iNHL. At the time of analysis, median duration of treatment was 22 weeks and 46 patients remained on treatment. In the follicular lymphoma (FL) subset of CHRONOS-1 (n=104), copanlisib treatment resulted in an ORR of 58.7%, including a CR of 14.4% and a median DOR of more than 52 weeks (370 days). The safety and tolerability were consistent with previously published data on copanlisib. The most common treatment-related adverse events were transient hyperglycemia (all grades: 49%/Grade ≥3: 40%), which did not show severity above Grade 4 and hypertension (all grades: 29%/Grade ≥3: 23%), which did not show severity above Grade 3.

"NHL is the tenth most common cancer worldwide and one of the most common cancers in the U.S. Despite treatment advances, most indolent NHL patients relapse after, or are refractory to, current therapies," said Robert LaCaze, Executive Vice President and Head of the Oncology Strategic Business Unit at Bayer. "The positive results from CHRONOS-1 are an important milestone and reflect the potential clinical utility of copanlisib in addressing the unmet medical need in patients with malignant lymphoma."

Other copanlisib data to be presented at AACR (Free AACR Whitepaper) 2017 include preclinical analysis of copanlisib activity in B-cell lymphomas as a single agent or in combination with conventional and targeted agents and a study on the binding affinity of copanlisib.

Bayer is in discussion with the U.S. Food and Drug Administration (FDA) with respect to a New Drug Application (NDA), seeking accelerated approval of copanlisib for the treatment of relapsed or refractory FL who have received at least two prior therapies. The company has been granted Fast Track Designation by the FDA for copanlisib for this indication. Fast Track is a program designed to facilitate the development, and expedite the review of drugs to address unmet medical need in the treatment of a serious or life-threatening condition.

Copanlisib was also granted Orphan Drug Designation (ODD) by the FDA Office of Orphan Products Development in the U.S. in February 2015 for the treatment of FL and in February 2017 for the treatment of splenic, nodal, and extranodal subtypes of marginal zone lymphoma (MZL). The ODD program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases and disorders. The FDA regards any disease that affects less than 200,000 patients in the U.S. as rare.

About CHRONOS-1
CHRONOS-1 is an open-label, single-arm Phase II study (ClinicalTrials.gov Identifier: NCT01660451) evaluating copanlisib as a monotherapy in non-Hodgkin’s lymphoma patients. CHRONOS-1 was designed to evaluate the efficacy and safety of copanlisib in patients with relapsed or refractory indolent NHL, including follicular lymphoma (FL), who received at least two prior therapies. The primary endpoint of CHRONOS-1 is the objective tumor response rate, with duration of response, overall survival, progression-free survival, quality of life, and safety serving as secondary endpoints.

About Non-Hodgkin’s Lymphoma
Non-Hodgkin’s Lymphoma (NHL) is the most common hematologic malignancy and the tenth most common cancer worldwide, with nearly 386,000 new cases diagnosed in 2012. It accounts for nearly 200,000 deaths per year worldwide. NHL comprises a highly heterogeneous group of diseases that can be indolent or aggressive with a poor prognosis. Follicular lymphoma is the most common histological subtype of indolent NHL, for which there is a need to improve treatment options.

About Copanlisib
Copanlisib is a novel pan-class I PI3K inhibitor with predominant inhibitory activity against PI3K-α and PI3K-δ isoforms, being developed by Bayer. The PI3K pathway is involved in cell growth, survival and metabolism, and its dysregulation plays an important role in non-Hodgkin’s lymphoma (NHL). Copanlisib is administered as a 1-hour infusion on an intermittent weekly basis (3 weeks on/1 week off).

Copanlisib has shown promising clinical activity in Phase I and Phase II studies in heavily pretreated patients with recurrent indolent and aggressive NHL. The broad clinical development program also includes Phase III studies in indolent NHL patients who have relapsed or are refractory to prior therapies. Information about these trials can be found at www.clinicaltrials.gov and www.chronostrials.com.

Copanlisib is not approved by the U.S. Food and Drug Administration, the European Medicines Agency or any other health authority.