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Ohr Pharmaceutical Reports Third Quarter 2016 Financial and Business Results

On August 9, 2016 Ohr Pharmaceutical, Inc. (NasdaqCM: OHRP), an ophthalmology research and development company, reported results for its third quarter ended June 30, 2016 (Filing, Q2, Ohr Pharmaceutical, 2016, AUG 12, 2016, View Source [SID:1234514538]).

"The start of the Phase 3 registration program for Squalamine during the quarter marked a significant milestone in our overall development program for our lead drug candidate," said Jason Slakter, MD, Chief Executive Officer of Ohr. "There remains a significant need for new treatment options that can enhance visual acuity gains in wet AMD and provide a non-invasive treatment option. We believe that Squalamine, when administered as part of a combination therapy, meets these needs and has the potential to set a new standard of care. We look forward to an exciting second half of calendar 2016."

Third Quarter Highlights
· Commenced enrollment in the Phase 3 clinical development program to investigate Squalamine lactate ophthalmic solution, 0.2% ("Squalamine", also known as OHR-102) as a treatment to improve visual acuity for patients with wet AMD.
o The Phase 3 program includes two clinical trials designed as double-masked, placebo-controlled, multicenter, international studies of Squalamine administered topically twice a day in patients with newly diagnosed wet AMD, in combination with Lucentis injections.
o The primary endpoint in both studies is a measurement of visual acuity gain at nine months, which is the most clinically meaningful endpoint for wet AMD patients. Subjects will be followed to two years for safety.
· Appointed David M. Brown, MD to serve as the chair of the Steering Committee for the Phase 3 clinical program of Squalamine in wet-AMD.
o Dr. Brown is Clinical Professor of Ophthalmology at Baylor College of Medicine, vice-chair for research at the Blanton Eye Institute, Houston Methodist Hospital, and partner at Retina Consultants of Houston.
· Completed an in vivo study demonstrating sustained pharmacological anti-angiogenic activity of OHR3031, an angiogenesis inhibitor
o Single intravitreal injection of microparticles containing OHR3031 produced clinically meaningful and statistically significant efficacy six weeks after dose administration in a rabbit model of laser-induced CNV.
o Dose response was observed in the reduction of average CNV lesion areas with OHR3031 compared to vehicle treatment, with the highest dose exhibiting a statistically significant effect at Week 6.
o Magnitude of difference in average CNV lesion size for the high dose of OHR-3031 compared to vehicle treatment at 6 weeks was comparable to that seen at 2 weeks with a currently approved anti-VEGF agent conducted in a previous study.
o OHR3031 was developed using SKS sustained release technology
· Presented two posters on the Squalamine Phase 2 IMPACT study and OHR3031 in vivo studies at the Association for Research in Vision and Ophthalmology (ARVO) Conference in May.

Financial Results for Third Quarter ended June 30, 2016
· For the third quarter ended June 30, 2016, the Company reported a net loss of approximately $7.7 million, or ($0.24) per share, compared to a net loss of approximately $3.3 million, or ($0.11) per share in the same period of 2015.
· For the third quarter ended June 30, 2016, total operating expenses were approximately $7.6 million, consisting of approximately $1.7 million in general and administrative expenses, $5.6 million in research and development expenses, and $0.3 million in depreciation and amortization. This compares to total operating expenses in the same period of 2015 of approximately $3.3 million, consisting of $1.6 million in general and administrative expenses, $1.5 million in research and development expenses, and $0.3 million in depreciation and amortization.
· At June 30, 2016, the Company had cash and cash equivalents of approximately $17.6 million. This compares to cash and equivalents of approximately $28.7 million at September 30, 2015.

Financial Results for the Nine-Months ended March 31, 2016
· For the nine months ended June 30, 2016, the Company reported a net loss of approximately $19.1 million, or ($0.61) per share, compared to a net loss of approximately $11.3 million, or ($0.41) per share in the same period of 2015.
· For the nine months ended June 30, 2016, total operating expenses were approximately $17.8 million, consisting of $5.8 million in general and administrative expenses, $11.8 million of research and development expenses, and $0.9 million in depreciation and amortization. This compares to total operating expenses of $14.3 million in the same period of 2015, comprised of approximately $5.7 million in general and administrative expenses, $7.4 million in research and development expenses, and $0.9 million in depreciation and amortization.

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ProNAi Therapeutics Reports Second Quarter 2016 Results

On August 12, 2016 ProNAi Therapeutics, Inc. (NASDAQ: DNAI), a drug development company focused on advancing targeted therapeutics for the treatment of patients with cancer, today reported its financial and operational results for the second quarter of 2016 (Filing, Q2, ProNAi Therapeutics, 2016, AUG 12, 2016, View Source [SID:1234514537]).

"During the second quarter, we successfully licensed our first new asset, PNT141, a potent, selective and orally bioavailable small molecule inhibitor of the Cdc7 kinase," said Dr. Nick Glover, President and CEO of ProNAi Therapeutics. "Our goal is to build a broad pipeline consisting primarily of assets that leverage discoveries on the leading edge of cancer biology. PNT141 highlights this strategy as Cdc7 has a central function in both DNA replication and DNA damage response, two mechanisms that are increasingly recognized as having critical roles in driving cancer. In addition to toxicology and manufacturing work, we are conducting a robust preclinical assessment of PNT141 aimed at further informing our clinical development plans and patient selection strategies as we prepare this product candidate for clinical trials."

In June 2016, the company reported that it had suspended development of the cancer drug PNT2258. This decision was informed by a review of the interim data from the Wolverine Phase 2 trial of PNT2258. Although modest efficacy was observed from PNT2258 in this interim analysis, the company did not view these data as robust enough to justify continued development of the drug. The company continues to review these data in order to determine next steps with the asset and technology; however, no further investment in PNT2258 or the underlying DNAi platform by ProNAi is contemplated and the company subsequently has closed its research facility based in Plymouth, Michigan, which supported these programs.

Second Quarter 2016 Financial Results (all amounts reported in U.S. currency)
Total operating expenses for the three months ended June 30, 2016 were $12.9 million compared to $6.6 million for the three months ended June 30, 2015. Total operating expenses for the six months ended June 30, 2016 were $23.6 million compared to $13.3 million for the six months ended June 30, 2015. Total operating expenses included non-cash stock based compensation of $1.3 million and $2.7 million for the three and six months ended June 30, 2016 and of $0.4 and $0.6 for the three and six months ended June 30, 2015, respectively.

Research and development expenses increased to $9.1 million for the three months ended June 30, 2016 from $4.7 million for the three months ended June 30, 2015. Research and development expenses increased to $15.8 million for the six months ended June 30, 2016 from $10.0 million for the six months ended June 30, 2015. These increases were primarily due to a non-recurring $2.8 million restructuring charge related to estimated close-out expenses for PNT2258, a $0.9 million upfront payment for the exclusive license of PNT141 and an increase in personnel-related costs. These increased costs were partially offset by a decrease in third-party manufacturing costs.

General and administrative expenses increased to $3.8 million for the three months ended June 30, 2016 from $1.9 million for the three months ended June 30, 2015. General and administrative expenses increased to $7.8 million for the six months ended June 30, 2016 from $3.3 million for the six months ended June 30, 2015. These increases were primarily due to a $0.3 million non-recurring restructuring charge, increased personnel-related costs and fees incurred in support of activities as a public company and corporate growth and costs pertaining to business development activities.

For the three months ended June 30, 2016, ProNAi incurred a net loss of $12.9 million compared to a net loss of $15.2 million for the three months ended June 30, 2015. For the six months ended June 30, 2016, ProNAi incurred a net loss of $23.4 million compared to a net loss of $23.3 million for the six months ended June 30, 2015. During the three and six months ended June 30, 2015, net loss included a non-cash charge related to the change in fair value of preferred stock warrants of $8.6 million and $10.0 million.

At June 30, 2016, ProNAi had $130.6 million in cash and cash equivalents compared to $150.2 million in cash and cash equivalents at December 31, 2015.
At June 30, 2016, there were 30,220,083 shares of common stock issued and outstanding and stock options to purchase 4,409,724 shares of common stock issued and outstanding.

ProNAi Therapeutics Reports Second Quarter 2016 Results

On August 12, 2016 ProNAi Therapeutics, Inc. (NASDAQ: DNAI), a drug development company focused on advancing targeted therapeutics for the treatment of patients with cancer, reported its financial and operational results for the second quarter of 2016 (Press release, ProNAi Therapeutics, AUG 12, 2016, View Source [SID:1234514526]).

At June 30, 2016, ProNAi had $130.6 million in cash and cash equivalents compared to $150.2 million in cash and cash equivalents at December 31, 2015.

At June 30, 2016, there were 30,220,083 shares of common stock issued and outstanding and stock options to purchase 4,409,724 shares of common stock issued and outstanding.

Vedanta Biosciences Announces Collaboration with the NYU Langone Medical Center to Develop Microbiome-Derived Immunotherapies for Cancer

On August 11, 2016 Vedanta Biosciences, pioneering the development of a novel class of therapies designed to modulate pathways of interaction between the human microbiome and the host immune system reported that it has entered into a translational collaboration with the NYU Langone Medical Center focused on developing novel microbiome-derived immunotherapies for cancer patients being treated with checkpoint inhibitors (Press release, Vedanta Biosciences, AUG 11, 2016, View Source [SID:SID1234515735]).

Under the terms of the agreement, Vedanta will collaborate with a group of oncologists led by Jeffrey S. Weber, M.D., Ph.D., deputy director of the Laura and Isaac Perlmutter Cancer Center at NYU Langone and a renowned melanoma and immunotherapy expert, on clinical studies to support the identification of new microbiome immunotherapies for cancer. The studies will also explore mechanisms by which the gut microbiome influences the efficacy of checkpoint inhibitors in cancer patients. Recent research published in Cell by Vedanta co-founder Dr. Kenya Honda at Keio University, has suggested that human-dwelling bacterial strains can activate immune cells in the gut that could be harnessed for immunotherapies. Vedanta has a worldwide, exclusive license to IP covering Dr. Honda’s discovery. Other findings in the field indicate that gut bacteria can potentially modulate the therapeutic responses to checkpoint blockades, as well as other classes of cancer therapeutics.

"Dr. Weber is a pioneer in translational research, particularly in immunotherapy and the development of checkpoint inhibitors," said Dr. Bruce Roberts, Chief Scientific Officer of Vedanta. "We look forward to working with Dr. Weber to expand Vedanta’s portfolio of immune activating microbial cocktails for use in standalone immunotherapy and in combination with checkpoint inhibitors."

"Checkpoint inhibitors are a major advance in cancer therapy, but many patients do not respond to therapy, and some patients who respond will eventually relapse," said Dr. Weber. "Recent data suggest an important role for the microbiome in the anti-tumor activity of immunotherapy, and our other studies of the microbiome will offer interesting new clinical insights into how and why these treatments work. Further understanding of the role of the microbiome in immunotherapeutic responses against cancer may also lead to new and improved therapies."

CAR T-cells Targeting the CD4 Protein Granted Orphan Drug Designation for the Treatment of Peripheral T-cell Lymphoma (PTCL)

On August 11, 2016 iCell Gene Therapeutics reported that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation for its chimeric antigen receptor engineered T-cells directed against the target protein CD4 (CD4CAR) for the treatment of peripheral T-cell lymphoma (PTCL) (Press release, iCell Gene Therapeutics, AUG 11, 2016, View Source [SID:1234514558]). The Orphan Drug Designation program provides orphan status, and associated development incentives, to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US.

Yupo Ma, MD, PhD, Professor of Pathology at Stony Brook University & Chairman and Chief Scientific Officer at iCell Gene Therapeutics, said: "CD4CAR could significantly enhance currently available treatment options for these patients. The Orphan Drug Designation is an important achievement as we advance our development plans for this promising treatment in T-cell hematologic cancers."

About CAR T-cell Technology
A "chimeric antigen receptor" (CAR) engineered T-cell is a patient’s T-cell (a component of the immune system) that has been genetically modified to express a protein on its surface with the capability to bind to a target protein on another cell. Upon binding, the CAR protein will send a signal across the cell membrane to the interior of the T-cell to set in motion mechanisms to selectively kill the targeted cell.

About PTCL
Although there are clinical development programs ongoing with CAR T-cells for CD19+ cell hematological malignancies, CD4+ peripheral T-cell lymphomas (PTCLs) have not been targeted by a CAR therapy in a human trial. PTCLs account for 10–15% of all non-Hodgkin’s lymphomas (NHLs) and are more difficult to treat in comparison to B-cell NHLs. Furthermore, and with few exceptions, T-cell NHLs have poorer outcomes, lower response rates, shorter times to progression, and shorter median survival in comparison to B-cell NHLs. As a result, the standard of care for PTCLs is not well-established and the only potential curative regimen is Bone Marrow Transplant (BMT). Not only is BMT poorly-tolerated, but is not an option for a significant subset of patients with resistant disease. This leaves many patients with no curative options.

William Tse, MD, FACP and Chief of the Blood and Marrow Transplantation Division, Department of Medicine at University of Louisville School of Medicine, said "we are very excited to have this opportunity to partner with the iCell Gene Therapeutics and to lead this cutting-edge immunotherapy into first-in-human clinical trial for patients suffering this extremely difficult to treat T cell lymphoma."

About CD4CAR
CD4CAR is in development for CD4+ T-cell malignancies. The novel CD4-specific chimeric antigen receptor engineered T-cells are properly-matched allogeneic human T-cells engineered to express an anti-CD4scFV antibody domain. An initial Phase I clinical study is being planned through collaboration between iCell Gene Therapeutics, the National Institutes of Health, Indiana Clinical and Translational Sciences Institute, Stony Brook Hospital, the Blood and Marrow Transplantation Division and the Clinic Trial Research Unit at James Graham Brown Cancer Center at University of Louisville.