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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, reported its financial and operational results for the second quarter of 2016 (Press release, ProNAi Therapeutics, AUG 12, 2016, View Source [SID:1234514526]).

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"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.

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.

Soligenix Announces Recent Accomplishments and
Second Quarter 2016 Financial Results

On August 11, 2016 Soligenix, Inc. (OTCQB: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, reported its recent accomplishments and financial results for the second quarter ended June 30, 2016 (Filing, Q2, Soligenix, 2016, AUG 11, 2016, View Source [SID:1234514528]).

Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix stated, "We continue to be very encouraged with the positive results demonstrated in our Phase 2 oral mucositis trial with SGX942 (dusquetide) and expect to report long-term safety data during the fourth quarter of this year. We have engaged the regulatory authorities in both the US and Europe, and anticipate having a pivotal Phase 2b/3 study design of SGX942 during the first half of next year. We also continue to actively enroll patients in our pivotal Phase 3 study in cutaneous T-cell lymphoma with SGX301 (synthetic hypericin)."

Schaber continued, "We continue to advance the development of RiVax and OrbeShield in our biodefense business segment, and are pleased with the government agencies additional non-dilutive funding of over $8 million through newly awarded funding and the exercise of options in support of these programs. The continued support of these programs demonstrates the productive collaboration between the Company and the government agencies."

Soligenix Recent Accomplishments:

· On July 25, 2016, the Company announced that the Biomedical Advanced Research and Development Authority (BARDA) and the National Institute of Allergy and Infectious Diseases (NIAID) have each provided additional funding to advance preclinical development of OrbeShield (oral beclomethasone 17,21-dipropionate or oral BDP) as a medical countermeasure for civilian and military use in the treatment of gastrointestinal acute radiation syndrome (GI ARS). The additional funding totaled $634,000.

· On July 18, 2016, the Company announced positive preliminary proof-of-concept results from its collaboration with Axel Lehrer, PhD of the Department of Tropical Medicine, John A. Burns School of Medicine, University of Hawaiʻi and Hawaii Biotech, Inc. to develop a heat stable subunit Ebola vaccine. The Company evaluated its proprietary vaccine thermostabilization technology, ThermoVax, licensed from the University of Colorado, to stabilize components of the vaccine.

· On June 25, 2016, the Company presented preliminary results from its SGX942 Phase 2 clinical trial in oral mucositis at the Multinational Association for Supportive Care in Cancer (MASCC) conference in Adelaide, Australia. The reduction in duration of severe oral mucositis ranged from 50% in the overall population to 67% in the population with the most severe disease.

· On May 25, 2016, the Company announced that NIAID exercised an option for the evaluation of RiVax to fund animal efficacy and toxicology studies. The exercised option will provide an additional $3.2M in funding.

· On May 5, 2016, the Company announced that NIAID exercised an option for RiVax bulk drug substance and finished drug product process scale-up and technology transfer that will support preclinical studies and manufacturing in accordance with current good manufacturing practices. The exercised option will provide an additional $4.3M in funding.

Financial Results – Second Quarter Ended June 30, 2016

Soligenix’s revenues for the quarter ended June 30, 2016 were $3.2 million as compared to $1.1 million for the same period for the prior year. Revenues included contracts with BARDA and NIAID in support of OrbeShield development in the treatment of GI ARS and advanced development of the Company’s thermostabilization technology, ThermoVax, combined with its ricin toxin vaccine RiVax, as a medical countermeasure to prevent the effects of ricin exposure.

Soligenix’s basic net loss was $0.1 million, or $0.00 per share, as compared to $4.0 million, or $0.15 per share, for the quarters ended June 30, 2016 and 2015, respectively. Included in the net loss for quarters ended June 30, 2016 and 2015 is non-cash income of $0.9 million and a non-cash expense of $1.9 million, respectively. This non-cash item reflects the change in fair value of the liability related to warrants issued in the Company’s June 2013 registered public offering and is included in other income/(expense).

Research and development expenses, were $0.8 million as compared to $1.4 million for the quarters ended June 30, 2016 and 2015, respectively. The decrease was related to completion of patient enrollment and treatment in the Phase 2 trial of SGX942 for the treatment of oral mucositis in head and neck cancer. The long-term follow-up safety data from this trial continues to be collected, with completion expected during the second half of 2016.

General and administrative expenses were $1.0 million as compared to $0.9 million for the quarters ended June 30, 2016 and 2015, respectively.
As of June 30, 2016, the Company’s cash position was $3.2 million.

RXi Pharmaceuticals Reports Second Quarter 2016 Financial Results and Highlights Recent Corporate Developments

On August 11, 2016 RXi Pharmaceuticals Corporation (NASDAQ: RXII), a clinical-stage RNAi company developing innovative therapeutics that address significant unmet medical needs, reported its financial results for the second quarter ended June 30, 2016, and provided a business update (Press release, RXi Pharmaceuticals, AUG 11, 2016, View Source [SID:1234514527]).

"First of all, I am proud to report that careful execution of RXi’s clinical programs by our team has resulted in the complete enrollment of our Phase 2 study RXI-109-1402 for hypertrophic scars, and has exceeded our expected enrollment target for the full year in our Phase1/2 study, RXI-109-1501 in retinal scarring, allowing us to adhere to our originally projected readout timelines. I am also pleased to say that we have been able to do that while maintaining our quarterly burn rate at about $2 million," said Dr. Geert Cauwenbergh, President and CEO of RXi Pharmaceuticals. He further added, "We also continue to aggressively pursue strategic transactions that will enhance RXi’s pipeline for long-term success and increase shareholder value. We are satisfied with the traction we have experienced thus far in that process."

The Company will host a conference call today at 4:30 p.m. EDT to discuss financial results and provide an update on the Company. The webcast link will be available under the "Investors – Event Calendar" section of the Company’s website, www.rxipharma.com. The event may also be accessed by dialing toll-free in the United States and Canada: +1 888-669-0684.

International participants may access the event by dialing: +1 862-225-5361. An archive of the webcast will be available on the Company’s website approximately two hours after the presentation.
Select Second Quarter 2016 Financial Highlights
Cash Position
At June 30, 2016, the Company had cash, cash equivalents and short-term investments of approximately $5.8 million, compared with $10.6 million at December 31, 2015.
Per the terms of the Company’s public offering in June 2015, the Company could not sell shares of the Company’s common stock under the purchase agreement with Lincoln Park Capital Fund,
LLC ("LPC") until the expiration of the 13-month overallotment purchase rights ("Purchase Rights"). On July 2, 2016, with the expiration of the Purchase Rights, the Company’s restriction on selling common stock under the purchase agreement was lifted. Under the purchase agreement, the Company has the right to sell LPC up to $10.8 million shares of the Company’s common stock. To date, the Company has sold a total of 40,000 shares of common stock to LPC for net proceeds of $146,000.
Net Revenue
Net revenue for the quarter ended June 30, 2016 was $9,000 as compared with no revenues for the quarter ended June 30, 2015. Net revenue during the second quarter of 2016 was due to the value of the common stock and warrants issued by Thera Neuropharma, Inc. to the Company per the terms of the exclusive licensing agreement for RXi’s proprietary sd-rxRNA platform to develop therapeutics for neurodegenerative diseases.
Research and Development Expenses
Research and development expenses for the quarter ended June 30, 2016 was $1.3 million, compared with $1.4 million for the quarter ended June 30, 2015. Research and development expenses decreased from the prior quarter primarily related to a decrease in stock-based compensation expense due to the full vesting of stock options granted in 2012 offset by increases in research and development expenses due to manufacturing costs for Samcyprone and clinical trial expenses for Study 1502, the Company’s common warts trial, which commenced in December 2015.
General and Administrative Expenses
General and administrative expenses for the quarter ended June 30, 2016 was $0.9 million as compared with $0.8 million for the quarter ended June 30, 2015. The increase in general and administrative expenses was primarily due to an increase in legal and proxy-related fees for the Company’s special meeting and reverse stock split in April 2016 and the use of professional service providers due to the Company’s focus on business development activities in line with its key corporate initiatives offset by a decrease in stock-based compensation expense due to the full vesting of stock options granted in 2012.
Net Loss
Net loss for the quarter ended June 30, 2016 was $2.2 million, compared with $2.2 million for the quarter ended June 30, 2015. Net loss was consistent quarter over quarter with fluctuations in operating expenses discussed above.
NASDAQ Compliance
On April 18, 2016, the Company effected a one-for-ten reverse stock split of the Company’s common stock, resulting in total common shares issued and outstanding of 6.5 million. All share
and per share amounts in the financial statements have been retroactively adjusted for all periods presented to give effect to the reverse stock split. On May 2, 2016, the Company received written notice from the Nasdaq Stock Market, LLC notifying the Company that it had regained compliance with the minimum bid price requirement for continued listing on The NASDAQ Capital Market.

Select Business and Corporate Highlights
Since inception in 2012, RXi has achieved each one of its projected corporate goals on time and within budget. In January of this year, the Company outlined its 2016 corporate goals at the Biotech Showcase conference. We have made significant progress towards successfully completing these goals and remain on track to fully execute against each goal before the end of 2016.

Business Development
The Company has been actively pursuing multiple business development opportunities to drive growth, innovation and shareholder value. As shown with the completed licensing deals with both MirImmune, Inc. and Thera Neuropharma, Inc., RXi’s robust pipeline and extensive patent portfolio provides for a broad spectrum of possibilities including technology and research collaborations, strategic partnerships and out-licensing opportunities.

Recently, the Company announced that it is in the process of exploring strategic options including a range of potential M&A and business development opportunities that are complementary with current RXi activities and may significantly advance our clinical pipeline. These efforts are actively progressing and the Company remains committed to enhancing its growth through this type of transaction.

Therapeutic Development
The Company’s ongoing Phase 2 clinical trial, RXI-109-1402, is evaluating its first clinical candidate RXI-109, an sd-rxRNA compound targeting connective tissue growth factor (CTGF) to reduce scar formation of in the skin following scar revision surgery. Results from a blinded panel and an Investigator review, released in October 2015, showed that incision sites treated with RXI-109 after scar revision surgery achieved better scores as compared to control incision sites in the same subjects, three months post scar revision surgery. The adaptive protocol in place has allowed the Company to apply early learnings from all trials to date, to optimize the best treatment regimen necessary to advance into the next phase of development. As such, the Company has already identified the best time to start treatment as well as the optimal dose level, and is now evaluating the best treatment length (Cohorts 3 and 4). This study is now fully enrolled and the Company anticipates it will provide preliminary data in the second half of 2016 with a full interim analysis, for Cohorts 3 and 4, mid-2017.

As in dermal scarring, CTGF is known to play a role in retinal scarring. Reduction of CTGF in the eye by RXI-109 treatment may reduce the formation of retinal fibrosis that often accompanies
late stage AMD and contributes to vision loss. Enrollment in the first of three cohorts in the Company’s Phase 1/2 trial, RXI-109-1501, is complete. As there were no safety issues in the first cohort, enrollment into the second cohort at the next higher dose lever is ongoing.

Samcyprone, the Company’s second clinical candidate, is a topical immunotherapy currently being evaluated in a Phase 2a clinical trial. RXI-SCP-1502 is a multi-center, multi-dose trial conducted in subjects with at least one cutaneous, plantar or periungual wart present for at least four weeks. The Company recently presented data, from a pilot study conducted by Hapten Pharmaceuticals, which led to the acquisition of Samcyprone, a proprietary formulation of Diphenylcyclopropenone (DPCP), and the initiation of its ongoing Phase 2 clinical trial. This trial is expected to be fully enrolled by the end of 2016 and the Company expects to share early read-outs H2 2016.

Consumer Health Program
RXi’s consumer health compounds, RXI-231 and RXI-185, are in development and are intended to affect the appearance of the skin. Both of these sd-rxRNA compounds are part of RXi’s partnering and business growth initiatives providing multiple development opportunities for non-therapeutic skin health. Evaluation of topical delivery options for these compounds is underway and the Company expects to explore one of these candidates in consumer testing by the end of 2016.

Intellectual Property Estate
The Company actively broadens and protects its valuable intellectual property estate. Several new patent applications have been filed to protect our current pipeline and future developments providing a competitive advantage to the Company for numerous strategic partnering opportunities.