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.

On May 15, 2017 Zymeworks Inc. ("Zymeworks") (NYSE: ZYME; TSX: ZYME) a clinical-stage biopharmaceutical company dedicated to the discovery, development and commercialization of next-generation multifunctional biotherapeutics, initially focused on the treatment of cancer, reported financial results for the first quarter ended March 31, 2017 (Press release, Zymeworks, MAY 15, 2017, View Source [SID1234519134]).

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"During the first quarter, among other highlights, we received top-line data from the dose escalation stage of our on-going Phase 1 clinical trial of our lead product candidate, ZW25," said Ali Tehrani, Ph.D., Zymeworks’ President & CEO. "ZW25 has demonstrated preliminary anti-tumor activity across multiple cancer types in HER2 expressing patients who have progressed after several lines of treatment with HER2-targeted therapies."
First Quarter Highlights:
Preliminary data for ZW25 phase 1 clinical trial received
Orphan drug designation for ZW25 in gastric cancer received
Second research milestone with collaborator Eli Lilly announced
Two industry veterans added to Board of Directors
State-of-the-art lab facility opened

On May 3, 2017, subsequent to the first quarter, Zymeworks completed its initial public offering and sold 4,500,000 common shares at a price of $13.00 per share. In addition, Zymeworks has granted the underwriters an option, exercisable within 30 days of the date of its final prospectus relating to the IPO, to purchase up to an additional 675,000 common shares.

First Quarter Ended March 31, 2017 Financial Results
All amounts are in U.S. dollars. Zymeworks’ unaudited condensed consolidated financial statements are prepared in accordance with accounting principals generally accepted in the United States ("U.S. GAAP").

Revenues for the first quarter ended March 31, 2017 were $0.2 million compared to $0.3 million for the same period of 2016. The decrease in collaboration revenue of $0.1 million is due to a $0.3 million decrease in research support payments from Merck, which was partially offset by the increase in research support payments of $0.2 million from Daiichi.

Research and development expenditures for the first quarter ended March 31, 2017 were $9.1 million, compared to $7.9 million for the same period in 2016. The increase of $1.2 million, was primarily due to increased activities associated with our therapeutic platforms and early-stage research and discovery programs, recorded in other research activities.

General and administrative expenses in the first quarter ended March 31, 2017 were $6.3 million compared to $2.1 million for the same period in 2016. General and administrative expenses increased by $4.2 million, compared to the same period in 2016, primarily due to an increase in professional fees and compensation costs. The compensation cost increase was the result of higher share-based compensation expenses due to reclassification under U.S. GAAP of certain awards from equity to liability for accounting purposes as well as certain new hires. The increase in professional fees over the same period in 2016 was associated with consulting services as well as legal, intellectual property, assurance and taxation services.

Net loss for the three months ended March 31, 2017 was $15.9 million. Zymeworks expects that over the next several years, research and development expenditures will increase in connection with the ongoing development of product candidates and other clinical, preclinical and regulatory activities.

As of March 31, 2017, Zymeworks had $26.8 million in cash and cash equivalents and short-term investments, as well as $1.9 million in SR&ED and IRAP receivables. On May 3, 2017, subsequent to the first quarter, Zymeworks completed its initial public offering for a total of $58.5 million in aggregate gross proceeds.