On August 13, 2018 ImmunoCellular Therapeutics, Ltd. ("ImmunoCellular") (NYSE American: IMUC), a biotechnology company developing immunotherapies for the treatment of cancer based on its Stem-to-T-Cell research program, reported financial results for the second quarter ended June 30, 2018 (Press release, ImmunoCellular Therapeutics, AUG 13, 2018, View Source [SID1234529109]).

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Corporate Update

Stem-to-T-Cell Research Program: As previously disclosed, the Company through its research was able to verify a successful transfer of the selected T cell receptor genetic material into human hematopoietic stem cells. The Company is currently producing the transfected human hematopoietic stem cells that are intended for use in performing preclinical experiments. In this preclinical testing, the transfected human hematopoietic stem cells will be injected into animals and the maturation of the stem cells and integration into bone marrow will be monitored. The Company’s academic collaboration has progressed and a manuscript intended for publication in a scientific journal describing the results of that work is being prepared.

Strategic Alternatives Exploration: The Company has been actively engaged in a broad range of conversations with potential strategic partners to explore strategic alternatives, which may include a potential merger, consolidation, reorganization or other business combination, as well as the sale of the Company or the Company’s assets. These conversations have included the exchange of detailed information to determine the potential for an alignment of programs and strategies, as well as possible options for continuing to fund operation. The Company plans to continue this exploratory process with the assistance of its external strategic financial advisor, but cannot guarantee that any actions will be taken as a direct result of this review.

Clinical-Stage Programs: The ICT-107 (phase 3-ready for glioblastoma), ICT-121 (phase 1 completed for recurrent glioblastoma) and ICT-140 (phase 1/2-ready for ovarian cancer), are patient-specific dendritic cell-based immunotherapies targeting solid tumors. The Company continues to pursue opportunities for partnerships, licensing or sale of these anticancer assets.

Litigation Settlement: The Company reached a tentative, mutually acceptable agreement to settle the class action suit. The tentative agreement, which is subject to final documentation and Court approval, provides in part for a settlement payment of $1.15 million in exchange for mutual releases and the dismissal of all claims against the Company and its officers and directors in connection with the securities class action suit. The $1.15 million settlement payment will be fully funded by the Company’s insurance carrier.

Liquidity and Capital Resources: The Company has implemented an aggressive plan to reduce expenditures while remaining actively focused on operations and executing on key initiatives. As a result, the second quarter operating loss was reduced by 97%, or $11.0 million, to $307,090, compared to the second quarter of 2017. Working capital at the end of the second quarter was $3.4 million and the Company had cash of $2.9 million and no debt, as of June 30, 2018.

Anthony J. Gringeri, PhD, President and Chief Executive Officer commented: "During the second quarter and the first half of 2018, we continued to make significant progress on our strategies to advance our Stem-to-T-Cell program and explore strategic alternatives while also implementing actions to reduce our operating expenses to strengthen the financial condition of the company. Additionally, we are actively engaged with our strategic financial advisor to explore strategic opportunities for enhancing shareholder value. This remains a top priority for the ImmunoCellular management team and the board of directors."

Continued Dr. Gringeri: "We believe our Stem-to-T-Cell research program has the potential to be a game-changing treatment for cancer by utilizing the patient’s immune system to fight cancer. In April we were able to verify successful transfer of the selected T cell receptor genetic material into human hematopoietic stem cells. This milestone represents an important step in validating the Stem-to-T-Cell approach and is a key component of the proof-of-concept work for this technology which lays the groundwork for undertaking planning for preclinical testing. We are producing the transfected human hematopoietic stem cells that will be used for the preclinical phase of this program."

"We have streamlined our operations to manage our business in a fiscally responsible manner. Looking forward, we plan to remain focused on advancing our Stem-to-T-Cell research program, pursuing partnering, licensing or sale of our clinical-stage dendritic cell-based immunotherapy programs and enhancing shareholder value," concluded Dr. Gringeri.

Second Quarter 2018 Financial Results

For the quarter ended June 30, 2018, ImmunoCellular incurred a net loss of $306,704, or $(0.01) per basic and diluted share, compared to a net loss of $4.1 million or $(1.14) per basic and diluted share, for the quarter ended June 30, 2017. The decrease in the net loss is primarily due to the suspension of the ICT-107 phase 3 trial in June of 2017 and reductions in the Company’s other research and development programs along with reductions in general and administrative expenses.

Research and development expenses for the three months ended June 30, 2018 were $58,981 compared to $10,353,601 in the same period in 2017. During the quarter ended June 30, 2018, the Company’s trial related expenses were primarily limited to costs associated with its Stem-to-T-cell program. During the quarter ended June 30, 2017, the Company wrote off remaining supply inventories and expensed costs associated to wind down the phase 3 trial of ICT-107.

General and administrative expenses for the three months ended June 30, 2018 and 2017 were $670,203 and $988,266 respectively. This decrease was primarily due to reductions in compensation expense, professional fees, the number of members of the board of directors, board member compensation and the downsizing of corporate offices.

ImmunoCellular reported $3.8 million of cash used in operations during the six months period ended June 30, 2018, compared to $9.5 million in the same period in 2017. No warrants were exercised during 2018; accordingly, there were no financing proceeds. As of June 30, 2018, the Company had working capital of $3,427,092, compared to working capital of $4,647,903 as of December 31, 2017. The Company had no long-term debt obligations, no capital lease obligations, or other similar long-term liabilities, as of June 30, 2018, and the Company had approximately $2.9 million of cash and 41.9 million shares of common stock outstanding.

In light of ongoing research and exploratory strategic activities, ImmunoCellular is not holding a conference call to discuss second quarter 2018 financial results at this time. The Company plans to provide relevant updates at an appropriate time in the future.

On August 13, 2018 Diffusion Pharmaceuticals Inc. (Nasdaq: DFFN) ("Diffusion" or "the Company"), a clinical-stage biotechnology company focused on improving patient outcomes in unmet medical needs using its novel small molecule trans sodium crocetinate (TSC), reported its financial results for the three months ended June 30, 2018 and provides a business update (Press release, Diffusion Pharmaceuticals, AUG 13, 2018, View Source [SID1234529170]).

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Commenting on the second quarter, David Kalergis, Chairman and Chief Executive Officer, said, "We have continued to screen and enroll patients with inoperable glioblastoma multiforme (GBM) into our INTACT Phase 3 pivotal trial. Following the dose escalation run-in portion, this planned 236-patient study will enroll half the patients in the treatment arm consisting of standard-of-care radiation and chemotherapy plus TSC, and half in the control arm, which is standard-of-care alone. We designed INTACT based on our Phase 2 study that demonstrated a nearly four-fold increase in overall survival at two years in inoperable GBM patients."

Mr. Kalergis continued, "During the quarter, U.S. Patent 9,950,067 issued relating to methods of treating cancer using bipolar trans carotenoids including TSC. European patent 2575487 was validated for oral formulations of bipolar trans carotenoids and includes novel compositions in tablet, pill or capsule form. Further, US Patent 10,016,384, also relating to oral formulations of bipolar trans carotenoids, issued on July 10, 2018. We believe TSC holds great promise in treating a number of diseases in addition to cancer and are pleased to be able to protect a patient-friendly oral formulation suitable for more chronic uses. Our intellectual property protection further supports the value of TSC as we discuss partnership opportunities in various indications and geographies."

Diffusion is continuing preparations for a Phase 2 randomized, double-blind, placebo-controlled trial with TSC in acute stroke in cooperation with UCLA and the University of Virginia, and is in discussions with potential partners. Financing permitting, we expect to begin enrolling patients in early 2019 with data in about 18 months thereafter. The study calls for the administration of TSC by specially-trained Emergency Medical Technicians to ambulance-transported patients within two hours of the onset of a suspected acute stroke. In-ambulance administration could overcome the current severe timing delay in administering therapy to stroke victims, serving a market of up to 800,000 patients a year who suffer acute stroke.

Financial Results for the Three Months Ended June 30, 2018

We had cash and cash equivalents of $12.9 million as of June 30, 2018. We believe that our cash and cash equivalents will enable us to fund our obligated operating expenses and capital expenditure requirements into September 2019.

We recognized $1.4 million in research and development expenses during the three months ended June 30, 2018, compared with $1.2 million during the three months ended June 30, 2017. The increase was mainly attributable to a $0.8 million increase in expenses related to our Phase 3 GBM trial, offset by a $0.6 million decrease in expense associated with manufacturing costs.

General and administrative expenses for the three months ended June 30, 2018 were $1.7 million, compared with $1.8 million for the three months ended June 30, 2017. The decrease in general and administrative expense was primarily due to a $0.3 million decrease in professional fees, partially offset by an increase in salary and wages expense of $0.2 million.

Net cash used in operating activities for the first half of 2018 was $5.8 million, compared with $6.2 million during the same period in the prior year.

On August 13, 2018 Stemline Therapeutics, Inc. (Nasdaq: STML), a clinical-stage biopharmaceutical company developing novel oncology therapeutics, reported that the U.S. Food and Drug Administration (FDA) has accepted for filing the Company’s Biologics License Application (BLA) for ELZONRISTM (tagraxofusp; SL-401) for the treatment of patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN) (Press release, Stemline Therapeutics, AUG 13, 2018, View Source [SID1234532233]). The FDA also granted Priority Review for the BLA and has set a target action date of February 21, 2019, under the Prescription Drug User Fee Act (PDUFA).

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The FDA grants Priority Review to product applications that, if approved, would provide significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.

ELZONRIS has also been granted Breakthrough Therapy Designation (BTD) and Orphan Drug Designation (ODD) by the FDA.

Ivan Bergstein, M.D., Stemline’s CEO, commented, "The acceptance of our BLA for filing and grant of Priority Review represent tremendous milestones for Stemline and the BPDCN patient community. We would like to thank the patients and their families who participated in our clinical trials, as well as recognize the tireless work of our investigators and entire Stemline team. Given both Priority and Breakthrough status, our commercial organization is positioning itself to rapidly launch ELZONRIS, if approved, to ensure this important new treatment reaches patients as quickly as possible."

Conference Call and Webcast
Stemline Therapeutics will host a conference call and audio webcast on Monday, August 13, 2018 at 8:30 AM ET. Interested participants and investors may access the conference call by dialing 844-389-8660 (U.S./Canada) or 478-219-0408 (International) and referencing conference ID: 4762319. An audio webcast can also be accessed via the Investor Relations tab of the Stemline Therapeutics website at View Source

About ELZONRISTM (tagraxofusp; SL-401)
ELZONRISTM (tagraxofusp; SL-401) is a novel targeted investigational therapy directed to CD123, a cell surface receptor expressed on a range of malignancies. ELZONRIS successfully completed a pivotal trial in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), and a Biologics License Application (BLA) in this indication has been accepted for filing and been granted Priority Review by the U.S. Food and Drug Administration (FDA). ELZONRIS has also been granted Breakthrough Therapy Designation (BTD) and Orphan Drug Designation by the FDA. ELZONRIS is also being evaluated in clinical trials in additional indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), and others.

On August 13, 2018 PharmaMar (MSE:PHM) reported that The International Association for the Study of Lung Cancer (IASLC) announced on Friday August 10th the titles for the presentations at the 19th World Conference on Lung cancer which will take place September 23-26 in Toronto, Canada (Press release, PharmaMar, AUG 13, 2018, View Source [SID1234528928]). PharmaMar will present a poster titled "Overall survival with lurbinectedin plus doxorubicin in relapsed SCLC. Results from an expansion cohort of a phase Ib trial"

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On August 13, 2018 Sutro Biopharma, Inc.,and TheLeukemia & Lymphoma Society , or LLS, reported that they are partnering to develop STRO-001, Sutro’s CD74-targeting antibody-drug conjugate, to treat relapsed and/or refractory multiple myeloma and non-Hodgkin’s lymphoma (Press release, Sutro Biopharma, AUG 13, 2018, View Source [SID1234529232]).

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CD74 is a protein highly expressed in B-cell malignancies such as myeloma and lymphoma.

LLS has agreed to contribute clinical development funding for STRO-001 — Sutro’s first internally-developed product candidate — through its Therapy Acceleration Program, which forges collaborations with biotechnology companies to help bring innovative therapies to patients faster.

Sutro intends to use the funding for a Phase 1 study, initiated in April of this year, to evaluate STRO-001 for treating multiple myeloma, diffuse large B-cell lymphoma, mantle cell lymphoma andindolent lymphomas, such asfollicular lymphoma.

The clinical trial is currently open and enrolling patients at City of Hope Comprehensive Cancer Center, Duarte, CA; Medical College of Wisconsin, Milwaukee; Texas Oncology, Austin; Rocky Mountain Cancer Centers, Aurora, CO; and Virginia Cancer Specialists in Fairfax. Sutro plans to add more sites later this year.

The study’s primary outcome measures are safety and tolerability of STRO-001 in dose escalation, and preliminary anti-tumor activity in dose expansion. This is the first clinical trial on a product candidate created with cell-free protein synthesis.

Sutro is obligated to make payments to The Leukemia & Lymphoma Society based on pre-specified late-stage clinical development, regulatory and commercialization milestones.

Financial terms of the partnership have not been disclosed.

"LLS is committed to advancing therapies to address critical unmet need," said Lee Greenberger, Ph.D., LLS chief scientific officer. "New options for therapies are vital for patients with non-Hodgkin’s lymphoma and myeloma who do not respond to available treatments. Sutro’s approach offers a promising option for these patients."

Sutro CEO Bill Newell said: "Despite treatment advances, many multiple myeloma and non-Hodgkin’s lymphoma patients have treatment-resistant disease or continue to suffer relapses, and their physicians have limited options, underscoring the need fornovel targeted therapies.

"Our collaboration with The Leukemia & Lymphoma Society will facilitate expansion of clinical research on STRO-001 for patients with progressive disease following standard of care therapies."

Added Dr. Arturo Molina, a medical oncologist and Sutro’s chief medical officer: "It’s difficult to find well-tolerated treatments that effectively target tumors in relapsed and refractory lymphoma and multiple myeloma. This partnership will enable us to examine if STRO-001 can be a potent new option for targeting these tumors."

Preclinical research findings presented by Sutro at the American Society of Hematology (ASH) (Free ASH Whitepaper)’s 2017 annual meeting and at other scientific meetings last year highlighted the specificity of STRO-001’s anti-CD74 antibody component, the high prevalence of CD74 expression in myeloma and lymphoma tumor samples, STRO-001’s potent in vitrocytotoxicity in multiple B-cell tumor cell lines and its anti-tumor activity in multiple myeloma and lymphoma xenograft models.

Sutro’s Proprietary Cell-Free Platform

STRO-001 was developed with Sutro’s XpressCFTMand XpressCF+TM proprietary cell-free protein synthesis and site-specific conjugation platforms, which enable rapid evaluation of a wide variety of protein structures and design and manufacturing of a highly-optimized single molecular species, rather than the usual mixture of imprecisely conjugated antibodies that comprise an antibody drug conjugate made by conventional cell-based manufacturing.

This cell-free technology should allow Sutro to move optimized proteins seamlessly through every stage of development — from discovery through commercial-stage production, without needing to generate individual cell lines for protein production.

Sutro’s manufacturing center in San Carlos, California, is built to maximize the speed and efficiency of protein production and is the first and only current cGMP-compliant, scalable cell-free manufacturing facility.

Added Dr. Trevor Hallam, Sutro’s chief scientific officer: "With XpressCF+TM, we incorporate non-natural amino acids into specific positions on the generated antibody for site-specific conjugation of cytotoxins with a linker and warhead to enable consistent, stable, pinpoint placement of STRO-001’s toxic payload. This leads to highly efficient delivery of the cytotoxin to tumor cells. By contrast, earlier generations of ADCs can have unpredictable pharmacologic properties, resulting in the potential for sub-optimal stability, compromised efficacy and poor tolerability for patients."