On August 14, 2019 NeuBase Therapeutics, Inc. (NASDAQ: NBSE) ("NeuBase" or the "Company"), a biotechnology company developing next-generation antisense therapies to address genetic diseases, reported financial results for the three and nine month periods ended June 30, 2019, which only include the financial results for Ohr Pharmaceutical prior to the merger and associated financings (Press release, Ohr Pharmaceutical, AUG 14, 2019, View Source [SID1234538799]).

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"Over the past several months, we have positioned the Company with the capital and industry expertise required to advance our strategy to develop antisense oligonucleotide therapeutics by leveraging our PATrOL platform," said Dietrich Stephan, Ph.D., chairman and chief executive officer of NeuBase. "Looking ahead, we are developing next-generation antisense oligonucleotide therapeutics for rare genetic diseases with a goal of transforming patients’ lives. We are currently advancing several preclinical programs, including NT0100 to target Huntington’s Disease and NT0200 to target myotonic dystrophy."

Fiscal Third Quarter of 2019 and Recent Operating Highlights

●Shares of the Company’s common stock commenced trading on the Nasdaq Capital Market under the ticker symbol "NBSE" as of market open on July 15, 2019.
●Completed two financings raising an aggregate of approximately $14 million in gross proceeds, which occurred alongside the Nasdaq listing and included a $5 million investment from Greenlight Capital.
●Appointed four experienced executives from the biotechnology industry to the board of directors: Dr. Dov Goldstein, Dr. Diego Miralles, Dr. Franklyn Prendergast, and Eric Richman.
●Appointed Dr. Danith Ly, the primary inventor of NeuBase’s peptide nucleic acid (PNA) antisense oligonucleotide (PATrOL) platform technology, as the Company’s chief scientific officer.
●Expanded the scientific advisory board with the appointments of Dr. Samuel Broder (former National Cancer Institute Director) and Dr. George Church (Harvard Medical School professor).

Financial Results for the Three Months Ended June 30, 2019:

●For the three months ended June 30, 2019, the Company reported a net loss of approximately $1.1 million, or ($0.40) per share, compared to a net loss of approximately $0.5 million, or ($0.19) per share, in the three months ended June 30, 2018. Loss per share amounts have been retroactively adjusted for the reverse stock split effected on February 4, 2019.
●For the three months ended June 30, 2019, total operating expenses were approximately $1.1 million, consisting of approximately $0.9 million in general and administrative expenses, $0.1 million of research and development expenses, and $0.2 million in depreciation and amortization. This compares to total operating expenses of $1.2 million in the three months ended June 30, 2018, comprised of approximately $0.8 million in general and administrative expenses, $0.1 million in research and development expenses, and $0.3 million in depreciation and amortization.
●At June 30, 2019, the Company had cash and cash equivalents of approximately $1.4 million, compared to cash and equivalents of approximately $3.8 million at September 30, 2018. Subsequent to the end of the quarter, NeuBase completed two financings alongside the closing of the merger in July 2019, raising gross proceeds of approximately $14 million. The Company believes that its current cash balance will provide sufficient capital to fund operations through the end of fiscal 2020.

Financial Results for the Nine Months Ended June 30, 2019:

●For the nine months ended June 30, 2019, the Company reported a net loss of approximately $3.2 million, or ($1.12) per share, compared to a net loss of approximately $6.9 million, or ($3.36) per share, in the same period of 2018. Loss per share amounts have been retroactively adjusted for the reverse stock split effected on February 4, 2019.
●For the nine months ended June 30, 2019, total operating expenses were approximately $3.2 million, consisting of approximately $2.5 million in general and administrative expenses, $0.2 million of research and development expenses, and $0.5 million in depreciation and amortization. This compares to total operating expenses of approximately $7.5 million in the same period of 2018, consisting of approximately $2.9 million in general and administrative expenses, $4.2 million of research and development expenses, $0.8 million in depreciation and amortization, $0.7 million in impairment of goodwill, and $1.2 million in gain on settlement of accounts payable and long term liabilities.

On August 14, 2019 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported that the U.S. Patent and Trademark Office has issued U.S. Patent No. 10,370,452 covering compositions and uses of effector T cells expressing a chimeric antigen receptor (CAR), where such T cells are derived from a pluripotent stem cell including an induced pluripotent stem cell (iPSC) (Press release, Fate Therapeutics, AUG 14, 2019, View Source [SID1234538739]). The foundational patent, which expires in 2034, is owned by Memorial Sloan Kettering Cancer Center (MSK) and is licensed exclusively to Fate Therapeutics for all human therapeutic uses.

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"The breadth of this newly-issued patent, which is not restricted by the signaling domain of the CAR construct nor by the antigen to which the CAR targets and binds, covers off-the-shelf, iPSC-derived CAR T cell therapy for the treatment of cancer, infectious disease and immune disorders," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "This patent is a testament to the prescient work of MSK’s Dr. Michel Sadelain in combining CAR and iPSC technologies in an effort to overcome the significant challenges that limit the widespread adoption of patient- and donor-sourced engineered T-cell immunotherapy."

Fate Therapeutics and MSK are conducting research and development of T-cell product candidates derived from iPSCs. These activities include the conduct of IND-enabling studies for FT819, an off-the-shelf CAR T-cell product candidate derived from a clonal master engineered iPSC line with complete elimination of T-cell receptor (TCR) expression and a novel 1XX CAR targeting CD19 inserted into the T-cell receptor alpha constant (TRAC) locus. Fate Therapeutics recently expanded its laboratory operations to New York City to support its research and development of off-the-shelf, iPSC-derived T cells.

Fate Therapeutics has built a dominant intellectual property position broadly covering iPSC technology and iPSC-derived cell products. Its proprietary portfolio includes compositions and methods for generating iPSCs, including engineering their biological properties using CRISPR and other nucleases, and for producing genetically edited cells of the hematopoietic lineage, including NK cells and T cells, from iPSCs. Fate Therapeutics’ iPSC product platform is supported by over 250 issued patents and 150 pending patent applications.

About FT819
FT819 is an off-the-shelf, T-cell receptor (TCR)-less CD19 chimeric antigen receptor (CAR) T-cell product candidate manufactured from a clonal master iPSC line that is undergoing IND-enabling activities in collaboration with Memorial Sloan Kettering Cancer Center (MSK). FT819 is manufactured from a clonal master engineered iPSC line with complete elimination of TCR expression and a novel 1XX CD19-specific CAR inserted into the T-cell receptor alpha constant (TRAC) locus. These synthetic features are intended to mitigate the risk of graft-versus-host disease, a severe life-threatening condition that occurs when donor T cells attack a patient’s healthy tissue, and to regulate CAR expression to enhance the therapeutic potency and durability of CAR T cells. Scientists from the Company and MSK have presented preclinical data demonstrating that FT819 lacks TCR expression, is highly cytotoxic and specific to CD19 antigen and controls tumor progression comparable to peripheral blood CAR19 T cells in a mouse model of acute lymphoblastic leukemia.

About Fate Therapeutics’ iPSC Product Platform
The Company’s proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered in repeat doses to mediate more effective pharmacologic activity, including in combination with cycles of other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Company’s platform is uniquely capable of overcoming numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is fraught with batch-to-batch and cell-to-cell variability that can affect safety and efficacy.

On August 14, 2019 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB) a late-stage clinical biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported financial results for the second quarter of 2019 (Press release, Y-mAbs Therapeutics, AUG 14, 2019, View Source [SID1234538780]).

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"We are very pleased with our second quarter results, highlighted by prudent spending combined with notable progress in the preparation of the initial portions of our rolling BLAs for our two lead product candidates, naxitamab and omburtamab, for submission to the FDA later this year. We continue to solidify our position as a leader in pediatric oncology and as a company focused on rapidly developing therapies to extend and enhance the lives of those living with rare pediatric cancers," stated Thomas Gad, Founder, Chairman, President and Head of Business Development and Strategy.

Dr. Claus Moller, Chief Executive Officer, continued, "We believe our recent pre-BLA meeting with the FDA supports our ambition of initiating submission of a rolling BLA for naxitamab for the treatment of relapsed/refractory high-risk neuroblastoma in the fourth quarter of 2019, with the expectation that the final portion will be submitted in early 2020. We are also excited to initiate the submission of a rolling BLA for omburtamab for the treatment of central nervous system/leptomeningeal metastasis from neuroblastoma by the end of 2019, and expect to submit the two rolling BLAs almost concurrently. These are exciting times for Y-mAbs."

Second Quarter 2019 and Recent Corporate Developments

Subsequent to the end of the second quarter, on July 8, 2019, Y-mAbs announced that it has completed a successful pre-BLA meeting with the FDA regarding a potential pathway for FDA approval of naxitamab for the treatment of relapsed/refractory high-risk neuroblastoma. During the meeting, the Company reached alignment with the FDA on an Accelerated Approval Pathway for naxitamab along with a rolling BLA submission. The Company expects to submit the Clinical/Safety portion and the non-Clinical portion of the BLA in November 2019. For the Chemistry, Manufacturing and Controls (CMC) portion, the Company believes it will have sufficient data from the Process Performance Qualification (PPQ) batches to complete the CMC portion in early 2020. Y-mAbs continues to evaluate potential avenues to accelerate the submission of the CMC portion, and hopes to comply with FDA requirements at an earlier time.

Also subsequent to the end of the second quarter, on July 1, 2019, Y-mAbs announced the status of patient recruitment for the Company’s two pivotal phase II trials, one for omburtamab for the treatment of central nervous system/leptomeningeal metastasis (CNS/LM) from neuroblastoma and the other for naxitamab for the treatment of relapsed/refractory high-risk neuroblastoma. As of June 30, 2019, all of the 18 planned omburtamab patients had been enrolled in the study. The Company believes it remains on track to start submission of a rolling BLA in 2019 under the Breakthrough Therapy Designation that the Company previously received from the FDA. For naxitamab, more than 30 patients of a planned total of 37 patients in the Company’s Study 201 had been enrolled in the study as of June 30, 2019.

After the close of the second quarter, on July 1, 2019, Y-mAbs announced that the Company has entered into a development, manufacturing and supply agreement with SpectronRx in South Bend, Indiana, to secure access to clinical and commercial scale radiolabeling capacity for omburtamab. Under the terms of the agreement, SpectronRx has agreed to establish a manufacturing unit designated for Y-mAbs within its existing facilities, at which Y-mAbs believes both clinical and commercial supply of radiolabeled omburtamab can be produced.

On June 26, 2019, Y-mAbs announced the acceptance of two oral presentations and five poster presentations at the International Society of Pediatric Oncology (SIOP) Annual Congress for its two lead product candidates, naxitamab and omburtamab.

On May 15, 2019, Y-mAbs announced the acceptance of three poster presentations at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting for its two lead product candidates, naxitamab and omburtamab.

On May 14, 2019, Y-mAbs announced an update on its GD2-GD3 Vaccine program for relapsed high-risk neuroblastoma. A total of 230 patients have received the GD2-GD3 Vaccine to date and Y-mAbs plans to begin using a newly manufactured Current Good Manufacturing Practice (cGMP) drug product in the fourth quarter of 2019.
Second Quarter 2019 Financial Results

Y-mAbs reported a net loss of $18.0 million, or $0.53 per basic and diluted share, for the three months ended June 30, 2019, compared to a net loss of $10.3 million, or $0.39 per basic and diluted share, for the three months ended June 30, 2018.

For the six months ended June 30, 2019, Y-mAbs reported a net loss of $34.0 million, or $0.99 per basic and diluted share, compared to a net loss of $17.8 million, or $0.66 per basic and diluted share, reported for the six months ended June 30, 2018.

Operating Expenses

Research and Development
Research and development expenses were $14.5 million for the three months ended June 30, 2019, compared to $8.3 million for the three months ended June 30, 2018, an increase of $6.2 million. The increase in research and development expenses primarily reflects the following:

$2.9 million increase in outsourced manufacturing for our two lead product candidates, naxitamab and omburtamab;
$2.2 million increase in outsourced research and supplies to support expanding development activities; and
$0.7 million increase in personnel costs.
Research and development expenses were $27.0 million for the six months ended June 30, 2019, compared to $14.5 million for the six months ended June 30, 2018, an increase of $12.5 million. The increase in research and development expenses primarily reflects the following:

$7.2 million increase in outsourced manufacturing for our two lead product candidates, naxitamab and omburtamab;
$3.5 million increase in outsourced research and supplies to support expanding development activities; and
$1.4 million increase in personnel costs.
General and Administration
General and administrative expenses were $4.1 million for the three months ended June 30, 2019, compared to $2.0 million for the three months ended June 30, 2018, an increase of $2.1 million. Such increase in general and administrative expenses primarily reflects the following:

$1.4 million increase in personnel costs; and
$0.5 million increase in commercial infrastructure.
General and administrative expenses were $7.9 million for the six months ended June 30, 2019, compared to $3.2 million for the six months ended June 30, 2018, an increase of $4.7 million. Such increase in general and administrative expenses primarily reflects the following:

$2.8 million increase in personnel costs; and
$0.8 million increase in commercial infrastructure costs.
Cash and Cash Equivalents
The Company had approximately $120.2 million in cash and cash equivalents as of June 30, 2019, compared to $147.8 million as of December 31, 2018. The decrease of $27.6 million was primarily attributable to the increased costs of operation as the Company prepares for its submission of rolling BLAs for naxitamab and omburtamab and the build-up of the Company’s commercial infrastructure.

Webcast and Conference Call

The Company will host a conference call today at 4:30 pm Eastern Time. To participate in the call, please dial (877) 407-0792 (domestic) or (201) 689-8263 (international) and reference the access code 13693605. A webcast will be available at: View Source

On August 14, 2019 Cleveland BioLabs, Inc. (NASDAQ:CBLI) reported financial results and development progress for the second quarter ended June 30, 2019 (Press release, Cleveland BioLabs, AUG 14, 2019, View Source [SID1234538834]).

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Cleveland BioLabs reported a net loss of $(0.6) million, excluding minority interests, for the second quarter of 2019, or $(0.05) per share, compared to a net loss, excluding minority interests, of $(0.9) million, or $(0.08) per share, for the same period in 2018. The decrease in net loss was primarily due to reduced research and development expenses related to the oncology applications of the entolimod family of compounds, a decrease in General and Administrative costs, and a decrease in the non-cash adjustment to our warrant liabilities, partially offset by reduced revenues.

As of June 30, 2019, the Company had $2.6 million in cash, cash equivalents and short-term investments, which, based on the Company’s current operational plan, is expected to fund operations into June 2020.

Yakov Kogan, Ph.D., MBA, Chief Executive Officer, stated, "The pursuit of regulatory approval and commercialization for entolimod as a medical radiation countermeasure remains our main priority and focus."

Further Financial Results

Revenue for the second quarter of 2019 decreased to $0.28 million compared to $0.39 million for the second quarter of 2018. The net decrease was primarily attributable to decreased revenue from our Joint Warfighter Medical Research Program ("JWMRP") contract from the Department of Defense ("DoD") for the continued development of the entolimod as a medical radiation countermeasure and by decreased revenue from our service contract with Incuron.

Research and development costs for the second quarter of 2019 decreased to $0.60 million compared to $0.91 million for the second quarter of 2018. The reduction in research and development costs is due to a $0.36 million decrease in expenses related to the oncology applications of the entolimod family of compounds, partially offset by a $0.05 million increase in spending for biodefense applications of entolimod.

General and administrative costs for the second quarter of 2019 decreased to $0.45 million compared to $0.55 million for the second quarter of 2018. This decrease was primarily attributable to a $0.03 million decrease in CBLI’s legal and professional fees, a $0.03 million decrease in travel expense, and a $0.03 million decrease in personnel and subcontractor expenses relating to outsourced assistance.

On August 13, 2019 DelMar Pharmaceuticals, Inc. (NASDAQ: DMPI) ("DelMar" or the "Company"), a biopharmaceutical company focused on the development of novel cancer therapies, reported an update on the first 20 patients enrolled in its ongoing Phase 2 clinical study investigating the first-line treatment of VAL-083 in combination with radiation therapy in newly-diagnosed, MGMT-unmethylated GBM (Press release, DelMar Pharmaceuticals, AUG 13, 2019, View Source [SID1234538649]).

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As of August 1, 2019, 17 of the first 20 enrolled patients have received at least their first assessment (two patients have not been enrolled long enough to receive their first assessment and one patient died before their first assessment). "Best Overall Response" for these patients per investigator assessment were:

Nine have been assessed as having achieved a complete response (CR) (9/17, or 53%)
Seven have been assessed with stable disease (SD), (7/17, or 41%)
One has been assessed as disease progression (PD) (1/17, or 6%)
Importantly, 16 of the 20 patients enrolled (80%) were still alive as of the August 1, 2019 data cut-off date. The Company recently announced the enrollment of the 20th patient into this trial.

Dr. David Reardon, clinical director of the Center for Neuro-Oncology at the Dana-Farber Cancer Institute and Professor of Medicine at the Harvard Medical School, and Dr. John de Groot, Professor and Chairman ad interim in the Department of Neuro-Oncology at The University of Texas MD Anderson Cancer Center (MDACC), are considered key opinion leaders in the brain cancer field and currently serve as DelMar Scientific Advisory Board members. Dr. Reardon commented, "Both John and I agree that we desperately need something better to offer our patients and we feel that VAL-083 has some promise and potential."

Dr. Reardon added, "For a tumor such as GBM, which is intrinsically infiltrative and destructive in the brain, stabilization of disease is an important achievement."

Professor Zhong-ping Chen, Founder Chairman of the Department of Neurosurgery/Neuro-oncology at Sun Yat-sen University Cancer Center (SYSUCC), who is the study’s principal investigator, stated, "We are pleased to be leading this Phase 2 trial for first-line GBM treatment with DelMar, and are encouraged by the enhanced levels of tumor shrinkage and the complete responses we are observing after treatment with VAL-083 in combination with radiation. These preliminary data appear to support the premise that VAL-083 has the potential to provide a valuable treatment option for these patients. We look forward to completing full enrollment in the study as soon as possible."

This Phase 2 trial which is being conducted at SYSUCC in Guangzhou, China, and in collaboration with Guangxi Wuzhou Pharmaceutical Company, is designed to enroll up to 30 patients to determine whether first-line therapy with VAL-083 treatment improves progression free survival (PFS). The current standard of care is first-line temozolomide (TMZ) with radiation. Of the 20 patients enrolled, 17 (85%) have received their two-month (post-third cycle) MRI and investigator assessment, 13 (65%) have received their five-month MRI and investigator assessment, and seven (35%) have received their eight-month MRI and investigator assessment. Two patients (10%) have not been on the study long enough to reach their first assessment, and one patient (5%) died before their first assessment. Assessments are based on the trial investigator’s clinical and radiologic assessment, according to the Response Assessment in NeuroOncology (RANO) criteria.

Saiid Zarrabian, DelMar’s president and chief executive officer, noted, "We continue to be encouraged by these early results and look forward to the completion of enrollment for our two, late-stage, Phase 2 studies. These results support our optimism that VAL-083 may provide a better treatment option than currently available treatments."

This Phase 2 trial is a single-arm, open-label study testing VAL-083 in combination with standard radiotherapy in GBM patients who have an unmethylated promoter of the methylguanine DNA-methyltransferase (MGMT) gene. The clinical trial in newly-diagnosed GBM patients is designed to determine if first-line treatment with VAL-083 plus radiotherapy can provide improvements over the historical efficacy of standard of care TMZ plus radiotherapy. Efficacy will be measured based on tumor response to treatment, progression-free survival, progression-free survival at six months, and overall survival compared to historical results in the target population.

The Company also recently announced the initiation of an adjuvant arm to the MDACC study to provide early disease data on VAL-083. This arm will enroll up to 24 newly-diagnosed patients who have undergone surgery and chemoradiation with TMZ but will now receive VAL-083 in place of standard of care TMZ for adjuvant therapy. This arm is in addition to a trial arm treating patients with recurrent disease, administering VAL-083 in patients who have been heavily pre-treated with TMZ prior to disease recurrence. The recurrent arm will allow a total of 83 patients to be enrolled, and both arms are being conducted at MDACC.

About VAL-083

VAL-083 (dianhydrogalactitol) is a "first-in-class" bifunctional DNA-targeting agent that introduces inter-strand DNA cross-links at the N7-position of guanine leading to DNA double-strand breaks and cancer cell death. VAL-083 has demonstrated clinical activity against a range of cancers, including GBM and ovarian cancer in historical clinical trials sponsored by the U.S. National Cancer Institute (NCI). DelMar has demonstrated that VAL-083’s anti-tumor activity is unaffected by common mechanisms of chemoresistance, including MGMT, in cancer cell models and animal studies. Further details regarding these studies can be found at:

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