On May 15, 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 three months ended March 31, 2017 and provided an overview of recent corporate highlights (Press release, RestorGenex, MAY 15, 2017, View Source [SID1234519163]).

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David Kalergis, Chairman and Chief Executive Officer, stated, "With the completion of our recent private placement, we believe we are now well positioned to advance our clinical development strategy for our lead product candidate, trans sodium crocetinate (TSC). We are in dialogue with the U.S. FDA regarding the design of a Phase 3 trial of TSC in newly diagnosed inoperable GBM patients, and plan to complete a protocol review in the third quarter of 2017. Assuming FDA sign-off on final protocol design, the study is planned to initiate by the end of 2017."

Corporate Highlights

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).

In March 2017, the Company raised aggregate gross proceeds of $25.0 million in an oversubscribed private placement of its Series A convertible preferred stock. At March 31, 2017, the Company had cash and cash equivalents of $12.2 million and a $8.3 million subscription receivable that was received on April 3, 2017.

Three Months Ended March 31, 2017 Financial Results

Research and development expenses were $1.0 million for the three months ended March 31, 2017, compared to $2.4 million for the three months ended March 31, 2016. This decrease in research and development was attributable to a decrease in expenses related to the Company’s pancreatic cancer program and animal toxicology studies.

General and administrative expenses were $1.6 million for the three months ended March 31, 2017, compared to $3.9 million for the three months ended March 31, 2016. The decrease in general and administrative expenses was primarily attributable to a decrease in costs attributable to the reverse merger transaction in January 2016.

We recognized $26.0 million in warrant related expenses (of which $25.6 million was non-cash related and fees netted against gross proceeds) associated with the private placement for the three months ended March 31, 2017, which consisted of the change in fair value of the common stock warrants from issuance,


the excess fair value of the common stock warrants over the gross cash proceeds from the Series A preferred stock offering, and placement agent commissions and other offering costs.

Net loss was $28.6 million for the three months ended March 31, 2017, compared to a net loss of $6.2 million for the three months ended March 31, 2016. For the quarter ended March 31, 2017, the net loss included $26.0 million in warrant related expenses (of which $25.6 million was non-cash related and fees netted against gross proceeds) associated with the private placement. For the three months ended March 31, 2017, net loss attributable to common shares was $28.7 million, which was inclusive of the dividend payable on the Series A convertible preferred stock.

Net cash used in operating activities for the three months ended March 31, 2017 was $3.4 million compared to $4.6 million during the three months ended March 31, 2016.

On May 15, 2017 OncoGenex Pharmaceuticals, Inc. (NASDAQ: OGXI) reported financial results for the first quarter ended March 31, 2017 (Press release, OncoGenex Pharmaceuticals, MAY 15, 2017, View Source [SID1234519151]).

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Recent Events

In January 2017, Achieve Life Science, Inc. (Achieve) and OncoGenex announced they entered into a definitive merger agreement.
In February 2017, OncoGenex announced that apatorsen results from two randomized Phase 2 clinical trials were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2017 Genitourinary Cancers Symposium, held February 16th- 18th in Orlando. Clinical data from trials in bladder and prostate cancers demonstrated apatorsen was well-tolerated and improved patient outcomes when administered in combination with standard-of-care treatments.
In March 2017, Achieve announced a strategic collaboration with the National Center for Complementary and Integrative Health (NCCIH) at the National Institutes of Health (NIH) to conduct non-clinical studies in support of an overall clinical development plan for cytisine as a smoking cessation treatment. As part of the collaboration, Achieve is providing cytisine to the NIH to conduct a series of non-clinical studies required by the U.S. Food and Drug Administration (FDA) to support the submission of an Investigational New Drug (IND) application. The collaboration commenced in March 2015 and results of the studies are expected in the second-quarter of 2017.
In March 2017, the Society for Research in Nicotine and Tobacco (SRNT) held a symposium on cytisine research at its annual conference held in Florence, Italy. The symposium was chaired by Professor Nancy Rigotti, MD, Massachusetts General Hospital/Harvard Medical School, with presentations from Associate Professor Natalie Walker, PhD, National Institute for Health Innovation, University of Auckland, on "Cytisine versus Varenicline for Smoking Cessation: Two Clinical Trials from the Australasian Cytisine Trialist Group," and "The Challenge to Getting Cytisine Licensed For Use Worldwide: Policy Considerations." Dr. Walker was the principal investigator of the 1,310 patient phase 3 CASCAID trial published in the New England Journal of Medicine in December, 2014 titled "Cytisine versus Nicotine for Smoking Cessation".
Financial Results

As of March 31, 2017, the company’s cash, cash equivalents, and short-term investments were $16.5 million compared with $25.5 million as of December 31, 2016. Based on current expectations, OncoGenex believes that its cash, cash equivalents, and short-term investments will be sufficient to fund its currently planned operations for at least the next 12 months.

Revenue for the three months ended March 31, 2017 decreased to zero from $2.9 million for three months ended March 31, 2016. Total operating expenses for the three months ended March 31, 2017 were $3.3 million compared to $7.4 million for the same period in 2016. Net loss for the three months ended March 31, 2017 was $3.3 million compared to $3.7 million for the same period in 2016.

As of May 15, 2017 OncoGenex had 30,087,485 shares outstanding.

On May 15, 2017 Kura Oncology, Inc., (NASDAQ:KURA) a clinical stage biopharmaceutical company focused on the development of precision medicines for oncology, reported first quarter 2017 financial results and provided a corporate update (Press release, Kura Oncology, MAY 15, 2017, View Source [SID1234519150]).

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"We remain focused on operational execution, delivering on our portfolio and key milestones," said Troy Wilson, Ph.D., J.D., President and CEO of Kura Oncology. "With tipifarnib, we are working to build upon the encouraging clinical activity we observed in our Phase 2 trial in HRAS mutant squamous cell head and neck cancers. We are also seeking to validate the potential novel biomarkers we identified in our Phase 2 trial in patients with PTCL, which we believe are associated with the initial activity we observed. We recently advanced KO-947, our second product candidate, into clinical testing, and we believe its profile and a substantial body of preclinical data make it a compelling therapeutic candidate. I’m pleased with our progress to advance multiple targeted programs aimed at addressing the urgent needs of cancer patients facing a poor prognosis and limited treatment options."

Recent Operational Highlights

First patient dosed in Phase 1 trial of KO-947 – In April, Kura began clinical testing of KO-947 as a potential treatment for cancers in which the mitogen activated protein kinase (MAPK) pathway is dysregulated. The trial design includes a dose escalation, maximum tolerated dose expansion and one or more tumor-specific extension cohorts.

Composition of matter patent issued for KO-947 – In April, the U.S. Patent and Trademark Office issued a patent that covers KO-947, a drug candidate targeting extracellular-signal-regulated kinases (ERK), and structurally-related compounds, as well as methods of using the compounds for the treatment of diseases including cancer.

Preclinical data presentation for KO-947 at AACR (Free AACR Whitepaper) – In April, Kura presented preclinical data for KO-947 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2017. The data showed that KO-947 demonstrated prolonged inhibition of the MAPK pathway and durable tumor regression was observed in preclinical cell line and patient-derived xenograft models, including KRAS- and BRAF-mutant adenocarcinomas and squamous cell carcinomas lacking BRAF/RAS mutations.

Preclinical data presentation for KO-539 at AACR (Free AACR Whitepaper) – In April, Kura presented preclinical data for KO-539, its development candidate targeting the menin-MLL interaction. The data showed that KO-539 demonstrated robust, sustained tumor regressions in multiple aggressive models of MLL-rearranged leukemias that correlated with modulation of target gene expression.
Upcoming Potential Milestones and Expectations for Clinical and Preclinical Programs

A trial-in-progress poster presentation for the Phase 2 trial of tipifarnib in HRAS mutant squamous cell carcinomas of the head and neck (SCCHN), including supporting rationale from patient-derived xenograft models at the ASCO (Free ASCO Whitepaper) Annual Meeting on June 2-6, 2017.

Poster presentation with data from the first and second stages of the Phase 2 trial of tipifarnib in peripheral T-cell lymphomas (PTCL) and associated biomarkers at the European Hematology Association (EHA) (Free EHA Whitepaper) conference on June 22-25, 2017.

Additional data from the Phase 2 trial of tipifarnib in HRAS mutant SCCHN in the second half of 2017.

Additional preclinical and clinical data for tipifarnib in PTCL, in the second half of 2017.

Data from the Phase 2 tipifarnib trials in lower risk myelodysplastic syndromes (MDS) and in chronic myelomonocytic leukemia (CMML) in the first half of 2018.

Data from KO-947 Phase 1 in 2018.
Financial Results for the First Quarter 2017

Cash, cash equivalents and short-term investments totaled $59.2 million as of March 31, 2017, compared with $67.8 million as of December 31, 2016. Management expects that current cash, cash equivalents and short-term investments will be sufficient to fund current operations into the second half of 2018.

Research and development expenses for the first quarter of 2017 were $5.5 million, compared to $4.6 million for the first quarter of 2016.

General and administrative expenses for the first quarter of 2017 were $2.1 million, compared to $2.4 million for the first quarter of 2016.

Net loss for the first quarter of 2017 was $7.5 million, or $0.39 per share, compared to a net loss of $6.6 million, or $0.36 per share, for the first quarter of 2016.

On May 15, 2017 ImmunoCellular Therapeutics, Ltd. ("ImmunoCellular") (NYSE MKT: IMUC) reported financial results for the first quarter 2017 and a re-evaluation of its financing and development strategies for ICT-107, its patient-specific, dendritic cell-based immunotherapy for patients with newly diagnosed glioblastoma (Press release, ImmunoCellular Therapeutics, MAY 15, 2017, View Source [SID1234519149]). Despite having made meaningful progress in executing the ongoing phase 3 registration trial of ICT-107, it has become clear that ImmunoCellular’s ability to access sufficient additional financial resources needed to complete the trial and continue its ongoing operations are limited. As of March 31, 2017 , the Company had $5.3 million in cash.

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Accordingly, ImmunoCellular’s board of directors has undertaken a strategic review to determine the feasibility of continuing to execute this trial independently or completing development through a partnership or acquisition of the asset. Given its limited financing options, ImmunoCellular is considering restructuring its business if a partner or acquirer for ICT-107 is not identified in the near term, but in any event, not later than 30 days. While this review is in progress, ImmunoCellular also intends to evaluate strategies to refocus and reallocate its available resources on its promising Stem-to-T-Cell research programs.

In light of the uncertainties surrounding these strategic pursuits, the Company is deferring a business update conference call in conjunction with reporting first quarter 2017 financial results until a later date.

First Quarter 2017 Financial Results

For the quarter ended March 31, 2017, ImmunoCellular incurred a net loss of $5.8 million, or $1.64 per basic and diluted share, compared to a net loss of $5.6 million, or $2.47 per basic and diluted share, for the quarter ended March 31, 2016.

For each of the quarters ended March 31, 2017 and March 31, 2016, the Company incurred approximately $4.7 million of research and development expenses. During the quarter ended March 31, 2017, the Company incurred expenses related to the Phase 3 trial of ICT-107 as the Company increased the number of sites participating in the trial and as treated more patients. During the quarter ended March 31, 2016, the Company incurred significant initial expenses to the North American and European cooperative groups for their support for ICT-107. During the most recent quarter, the Company also incurred expenses related to its Stem-to-T-cell immunotherapies.

The Company also reported that cash used in operations in the first quarter of 2017 was $6.1 million compared to $5.4 million in the prior year quarter. The increase primarily reflects a reduction in accounts payable and accrued expenses as of March 31, 2017. As of March 31, 2017, the Company had $5.3 million in cash and 3.5 million shares of common stock outstanding.

On May 15, 2017 Novocure (NASDAQ:NVCR) reported that United States Food and Drug Administration (FDA) has designated it’s Tumor Treating Fields (TTFields) delivery system as a Humanitarian Use Device (HUD) for the treatment of pleural mesothelioma (Press release, NovoCure, MAY 15, 2017, View Source [SID1234519135]).

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HUD designation covers devices that treat rare, orphan diseases or conditions. The HUD designation is the first step in obtaining a Humanitarian Device Exemption (HDE) for the treatment of pleural mesothelioma with TTFields. Novocure enrolled the last patient in its STELLAR trial, a phase 2 pilot trial in pleural mesothelioma, in March 2017 and, with final data collection in 2018, is evaluating its options to file an HDE application with the STELLAR data. An approved HDE would allow Novocure to market TTFields in combination with standard of care chemotherapy as a treatment for pleural mesothelioma in the United States.

"Due to the clinical and pathological course of mesothelioma, standard treatment methods rarely lead to a complete cure and new effective treatments are desperately needed. Our interim data from the STELLAR trial give us hope that TTFields used in combination with standard of care chemotherapies may increase survival for patients with mesothelioma without significantly increasing side effects," said Dr. Eilon Kirson, Novocure’s Chief Science Officer and Head of Research and Development. "We are pleased that the FDA has recognized orphan status for mesothelioma and acknowledges the urgent demand for new treatment options."

Novocure’s ongoing STELLAR trial includes 80 patients with unresectable, previously untreated malignant pleural mesothelioma. STELLAR’s primary endpoint is overall survival and secondary endpoints are progression free survival, response rate, and treatment-emergent toxicity. Interim results presented in December 2016 at the International Association for the Study of Lung Cancer (IASLC) 17thWorld Conference on Lung Cancer suggest improvements in progression free survival and one-year survival rates when TTFields is added to standard chemotherapy. Forty-two STELLAR patients who received treatment with TTFields plus pemetrexed and cisplatin or carboplatin experienced a 12-month survival rate of 79.7 percent and median progression free survival of 7.3 months. Median survival had not been reached at the time of presentation due to limited follow-up time, with an average follow-up time of 11.5 months. No device-related serious adverse events were reported.

"We believe that treatment with TTFIelds affects fundamental aspects of cell division and may have broad applicability across a variety of solid tumors," said Asaf Danziger, Chief Executive Officer of Novocure. "We are pleased to receive HUD designation as it is a major step towards a second regulatory approval for TTFields in the United States."

Treatment with TTFields is not approved for the treatment of mesothelioma by the U.S. Food and Drug Administration. The safety and effectiveness of TTFields therapy for mesothelioma has not been established.