On March 27, 2019 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a physician-led biopharmaceutical company focused on dermatological and immuno-inflammatory diseases, reported that management will attend the following conference (Press release, Aclaris Therapeutics, MAR 27, 2019, View Source [SID1234534664]):

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Management will host investor meetings during the William Blair & Company 3rd Annual Late-Stage Therapeutics Conference in New York, NY Thursday, April 4, 2019.

On March 27, 2019 Phio Pharmaceuticals Corp. (NASDAQ: PHIO), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (sd-rxRNA) therapeutic platform, reported its financial results for the fourth quarter and year ended December 31, 2018 and provided a business update (Press release, Phio Pharmaceuticals, MAR 27, 2019, View Source [SID1234534681]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Logo – View Source

"One of the Company’s major accomplishments during the year was the successful transition to focus on developing novel immuno-oncology treatments, a strategic decision made in early 2018," said Dr. Gerrit Dispersyn, President and CEO of Phio Pharmaceuticals. "Based on our internal R&D, and together with our corporate and academic partners, we have been demonstrating the potential of using our self-delivering RNAi platform in immuno-oncology applications. Our results to date suggest that we can improve existing and developing forms of adoptive cell therapies (ACT) based on T-cells and on NK cells. In addition, through direct intratumoral use of self-delivering RNAi compounds, we showed that we have the potential to reprogram the tumor micro-environment. With this latter approach, we hope to lower the barriers that hamper ACT success in solid tumors, a significant clinical unmet need. Based on these successes, we now have three well defined immuno-oncology R&D pipeline programs that may provide compelling alternatives to other therapeutic approaches such as antibody therapy and/or genetic engineering. Our progress laid the groundwork for us to successfully complete an equity offering in October, providing us the capital to confidently execute our R&D pipeline programs towards clinical development and further partnerships."

Year in Review and Recent Corporate Updates

On March 1, 2019, Gerrit Dispersyn, Dr. Med. Sc. became the Company’s President and Chief Executive Officer. Dr. Dispersyn succeeded Geert Cauwenbergh, Dr. Med. Sc., who retired as CEO of the Company and remains as a member of the Company’s Board of Directors.
Effective November 19, 2018, the Company changed its corporate name from RXi Pharmaceuticals Corporation to Phio Pharmaceuticals Corp. This change reflects the Company’s transition from a platform company in dermatology and ophthalmology to one that is focused on developing groundbreaking immuno-oncology therapeutics.
Research conducted using the Company’s self-delivering RNAi platform in the field of immunotherapy to treat cancer was published in a leading peer-reviewed journal, Molecular Therapy. The published paper is titled, "Self-Delivering RNAi (sd-rxRNA) Targeting PD-1 using Adoptive Cell Therapy Approach for the Treatment of Malignant Melanoma."
Presented new data on NK cells at the 16th Annual Discovery on Target Conference and the 33rd Annual Meeting & Pre-Conference Programs of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper).
Data demonstrated potent silencing activity as well as phenotypic effect of NK cells treated with sd-rxRNA compounds targeting checkpoints such as Cbl-b and TIGIT. By treating NK cells ex-vivo with sd-rxRNA compounds the anti-tumor response of these cells can be improved.
Entered into collaborations with industry and academic leaders in immuno-oncology:
The Karolinska Institutet in Sweden is exploring the use of our sd-rxRNA compounds against targets involved in T-cell and NK cell differentiation and/or in the immune cell tumor-induced stress response with the aim of producing anti-tumor adoptive cell therapy grafts with improved functionality and persistence.
Iovance Biotherapeutics, Inc. is evaluating the potential synergies of our novel sd-rxRNA therapeutic compounds with their autologous cell therapy based on tumor-infiltrating lymphocytes for the use in the treatment of cancer.
Completed and reported positive results from the Company’s clinical trials in dermatology and ophthalmology:
Study RXI-109-1501, a Phase 1/2 clinical trial for retinal scarring in subjects with wet age-related macular degeneration with evidence of subretinal fibrosis, successfully met its primary objective as shown by the absence of dose-limiting and serious toxicities. The secondary objective of the study was also met with improved or stable disease in the study eyes of several subjects.
Study RXI-SCP-1502, a Phase 2 clinical trial with Samcyprone for the treatment of cutaneous warts, successfully met its primary effectiveness objectives as shown by high levels of immunotherapeutic and therapeutic response. The immunotherapeutic response rate was 97.7% across all enrolled subjects and from a therapeutic response viewpoint, more than 70% of all warts showed a positive wart response rate. The study results showed furthermore that Samcyprone was safe and well tolerated.
Successfully completed equity offerings in April and October 2018 for total net proceeds of $17.4 million, which the Company expects to provide ample funding for operations into the second half of 2020.
Select Financial Results

Cash Position

At December 31, 2018, the Company had cash of $14.9 million as compared with $3.6 million at December 31, 2017.

Revenues

Revenues for the year ended December 31, 2018 were $138,000, as compared with $15,000 for the year ended December 31, 2017. Revenues for the years ended 2018 and 2017 related to the work performed by the Company as a sub-awardee under the government grant awarded to BioAxone Biosciences, Inc. for the development of a novel sd-rxRNA compound, BA-434, that targets PTEN for the treatment of spinal cord injury.

Research and Development Expenses

Research and development expenses for the year ended December 31, 2018 were $4.3 million as compared with $5.4 million for the year ended December 31, 2017. The decrease was primarily due to the completion of the work in the Company’s dermatology and ophthalmology programs, including clinical trial-related and manufacturing-related expenses, and a decrease in payroll expenses due to a reduction in headcount as compared with the prior year period.

Acquired In-process Research and Development Expenses

The Company recorded acquired in-process research and development expense of $4.7 million during the year ended December 31, 2017. The expense related to the fair value of consideration given, which includes transaction costs, liabilities assumed and cancellation of notes receivable, and the deferred tax impact of the Company’s acquisition of MirImmune. The Company had no such expense during the year ended December 31, 2018.

General and Administrative Expenses

General and administrative expenses for the year ended December 31, 2018 were $3.2 million as compared with $4.0 million for the year ended December 31, 2017. The decrease was primarily due to a decrease in payroll expenses due to a reduction in headcount as compared with the prior year period, as well as a decrease in professional fees for legal-related services.

Income Tax

The Company had no income tax benefit or expense for the year ended December 31, 2018. The Company recognized an income tax benefit of $1.6 million for the year ended December 31, 2017 due to the tax-related impact of the Company’s acquisition of MirImmune.

Net Loss

Net loss for the year ended December 31, 2018 was $7.4 million, or $1.04 per share, compared with $12.5 million, or $5.52 per share, for the year ended December 31, 2017. The decrease was primarily due to a decrease in acquired in-process research and development expense and the changes in operating expenses, as discussed above.

On March 27, 2019 XenTech SAS, a company specialized in the development and sale of preclinical research services to foster the development of oncology drugs, reported a strategic collaboration with Gustave Roussy, Europe’s leading cancer center (Press release, XENTECH, MAR 27, 2019, View Source [SID1234553821]). This co-operation, the first of its kind for XenTech, will focus on the development of a collection of tumor explant models from patients who developed acquired resistance to targeted therapies following initial response. These PDX models will be used in Gustave Roussy’s oncology R&D programs, as well as being added to XenTech’s existing PDX platform for translational oncology research projects for academic and industry customers.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Patients with tumors that harbor specific driver molecular alterations benefit from targeted therapies, but responses are generally short-lived due to the emergence of adaptive/secondary resistance. Between 2015 and 2020 the MATCH-R trial led by Gustave Roussy (NCT02517892) will have enrolled 600 patients treated with targeted therapies. Biopsies will be used to generate PDX models, obtained from 300 patients who have developed resistance following initial response. The MATCH-R PDX platform will be regularly upgraded with new models, providing a unique resource in unravelling the mechanisms involved in acquired resistance to targeted therapies and testing novel therapeutic strategies to circumvent or delay the emergence of resistance.

"We are delighted to collaborate with Gustave Roussy on this unique and ambitious program," said Jean-Gabriel Judde, CSO and president of XenTech. "These new models will expand XenTech’s bank of PDX models with focus on the advanced drug-resistant setting. The MATCH-R PDX platform will enable a better understanding of acquired resistance to last-generation targeted therapies, providing clinically relevant models to perform preclinical POC studies, translating into increased patient survival. Full clinical and molecular annotation will enable model selection for testing innovative therapies, investigating new and existing pathways, and identifying biomarkers."

"Understanding the mechanisms of acquired resistance to novel targeting agents is crucial in providing optimal care to cancer patients," said Benjamin Besse, head of the department of medical oncology at Gustave Roussy and principal investigator of the MATCH-R clinical trial. "This strategic collaboration with XenTech, a renowned expert in PDX development and in vivo pharmacological studies, is a major asset for Gustave Roussy’s precision medicine program. There is no doubt that these clinically relevant models will speed-up the development of novel therapeutic agents leading to extended clinical benefit for metastatic cancer patients."

XenTech will participate in American Association Cancer Research Annual Meeting, Friday, March 29 – Wednesday, April 3, 2019, in Atlanta, GA.

On March 26, 2019 Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company discovering and developing novel small molecule therapeutics to treat unmet needs in autoimmunity and cancer, reported its fourth quarter and full year 2018 financial results and business highlights (Press release, Kezar Life Sciences, MAR 26, 2019, View Source [SID1234534625]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"This past year was pivotal for Kezar, and I’m proud of the tremendous progress our team has accomplished," said John Fowler, Chief Executive Officer. "We successfully completed our IPO, creating a strong capital base, and we are rapidly advancing KZR-616, our first-in-class selective immunoproteasome inhibitor for patients living with severe and underserved autoimmune diseases. We look forward to sharing results from the initial two cohorts from the Phase 1b portion of our trial in lupus patients and initiating our Phase 2 trial in lupus nephritis (LN) in the second quarter of 2019. We are also pleased to announce the planned expansion of our KZR-616 clinical program with additional Phase 2 trials commencing in up to four new indications later this year, including dermatomyositis and polymyositis. Lastly, our ongoing discovery efforts in protein secretion are progressing well, and we anticipate nominating our first oncology clinical candidate before the end of the year."

Fourth Quarter and Recent Clinical and Business Highlights

Our systemic lupus erythematosus (SLE) and LN program is advancing with enrollment continuing in the open-label dose escalation Phase 1b portion in SLE patients. We plan to initiate the Phase 2 portion of the trial in patients with active, proliferative LN and expect to report data from the first two cohorts of the Phase 1b portion in the second quarter of 2019.

We initiated a Phase 1, randomized, double-blind, placebo controlled, single and multiple ascending dose trial to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and immunomodulatory activity of a new lyophilized formulation of KZR-616. This formulation of KZR-616 has the potential to improve the ease of drug administration, transportation and storage, which we believe may result in increased patient adoption in a commercial setting. Additionally, data from this trial may support development and potential regulatory approval.

Clinical development efforts are underway to initiate trials in up to four additional autoimmune diseases of high unmet need beginning in the second half of 2019, with the first two diseases being dermatomyositis and polymyositis.

The protein secretion program (Sec61 translocon modulation) also advanced during the fourth quarter which will be showcased in two poster presentations at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) in Atlanta, GA on April 2, 2019. We remain on track to nominate a first clinical candidate in oncology this year.

Earlier this month, we announced the appointment of Celia Economides as Senior Vice President of Strategy and External Affairs. Ms. Economides will serve as a member of the management team and executive committee leading Kezar’s overall investor relations, communications, patient advocacy and strategic efforts.

Financial Results

Cash, cash equivalents and marketable securities totaled $107.4 million as of December 31, 2018, compared to $51.0 million as of December 31, 2017. The increase in cash, cash equivalents and marketable securities was primarily attributable to IPO proceeds, net of cash used by the Company in operations to advance its clinical stage programs as well as preclinical research and development.

Research and development expenses for the fourth quarter of 2018 increased by $3.0 million to $4.6 million from $1.6 million in the fourth quarter of 2017. Full year R&D expenses increased by $11.7 million in 2018 compared to 2017. This increase was primarily related to advancing both the KZR-616 clinical program across indications and the protein secretion preclinical program in addition to personnel and facility-related expenses.

General and administrative expenses for the fourth quarter of 2018 increased by $1.0 million to $1.8 million from $0.8 million in the fourth quarter of 2017. Full year G&A expenses increased by $4.3 million in 2018 compared to 2017. The increase was primarily due to an increase in stock-based compensation, personnel expenses, consulting and professional fees related to operating as a public company, and facility-related expenses due to the move to our new corporate office.

Net loss for the fourth quarter of 2018 was $5.8 million, or $0.30 per basic and diluted common share, compared to a net loss of $2.3 million, or $3.34 per basic and diluted common share, for the fourth quarter of 2017. Net loss for 2018 was $23.2 million, or $2.26 per basic and diluted common share, compared to $8.5 million, or $14.21 per basic and diluted common share, in 2017.

Total shares outstanding were 19.1 million as of December 31, 2018. Additionally, there were 2.2 million outstanding options granted to purchase common stock at a $3.80 weighted average exercise price as of December 31, 2018.

On March 26, 2019 IMV Inc. (Nasdaq: IMV; TSX: IMV), a clinical stage immuno-oncology corporation, reported preliminary data from the phase 2 cohort of the DECIDE clinical study (Press release, IMV, MAR 26, 2019, View Source [SID1234534641]). Six patients receiving DPX-Survivac monotherapy with intermittent low-dose cyclophosphamide (mCPA) have reached the first CT scan assessment with key related findings as follows:

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

83% of the subjects (5 of 6) show stable disease (SD), including two tumor regressions
80% (4 of 5) with stable disease are in subjects with a lower baseline tumor burden (BTB), which also includes the two tumor regressions
"This initial phase 2 data confirms the earlier trends we saw in the phase 1b portion of the study," said Frederic Ors, Chief Executive Officer. "It supports the potential of DPX-Survivac as a monotherapy and the use of our patient selection strategy. We are encouraged by these early initial results and are committed to advancing this program quickly with the goal of providing an additional treatment option to patients with advanced ovarian cancer."

Importantly, in earlier stages of this trial, durable clinical responses occurred after 140 days, and have now lasted for 20 months or more. Additional data at the 140 day mark of this cohort will be available by the end of the first half of 2019.

This amended phase 2 study evaluates the safety and efficacy of DPX-Survivac monotherapy with mCPA in patients with advanced recurrent ovarian cancer. As of the March 25, 2019 data cut-off date, 13 patients have been enrolled in the phase 2 portion of the trial in addition to the 53 enrolled in the phase 1b cohort. Five patients were randomized into the DPX-Survivac monotherapy cohort. Seven patients had been randomized into DPX-Survivac/mCPA in combination with epacadostat before the phase 2 protocol was amended to stop enrollment in the combination arm. One of the patients in the combination arm elected to switch to the monotherapy arm of the trial. Positive data from the phase 1b portion of the trial led IMV to amend the study to monotherapy in patients with lower tumor burden.

The amended phase 2 cohort of the DECIDE trial is targeting an enrollment of at least additional 16 patients in the population with a lower tumor burden. Enrollment is ongoing at multiple sites in the U.S. and Canada.