On May 11, 2018 Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel cell and gene therapies for life-threatening rare genetic diseases, reported financial results for the first quarter of 2018 (Press release, Abeona Therapeutics, MAY 11, 2018, View Source;p=RssLanding&cat=news&id=2348683 [SID1234526535]). The Company will host a call to update investors on recent clinical developments and quarter financial results on Monday, May 14th at 10:00 am (Eastern). Interested parties are invited to participate in the call by dialing 877-407-9210 (toll free domestic) or 201-689-8049 (International) or via webcast View Source

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"Abeona has made significant progress across multiple fronts, including various regulatory achievements, additional enrollments in our MPS IIIA trial, and the reporting of encouraging initial results for our MPS IIIB trial," stated Carsten Thiel, Ph.D., Abeona’s CEO. "Notably, both our lead clinical programs, EB-101 and ABO-102, were recently granted RMAT designation by the FDA, supporting the strong safety and biopotency evidence from these programs, and their potential to address the unmet medical need for RDEB and MPS IIIA patients."

1st Quarter Summary Financial Results:

– Cash position: Cash and cash equivalents as of March 31, 2018 were $132 million, compared to $137.8 million as of December 31, 2017.
– Revenues: Revenues were $2.6 million for the first quarter of 2018, compared to $186 thousand in the first quarter of 2017. A large portion of the increased quarterly revenues consisted of the recognition of Foundation grants that were announced during the 4th quarter of 2017. A portion of the grants were received in the 4th quarter of 2017 and in the 1st quarter of 2018, and the amount recognized is matched against corresponding expenditures for drug manufacture and clinical readiness. Additional revenues consisted of royalties from marketed products, specifically MuGard. In the quarter, Abeona adopted ASC 606 pertaining to revenue recognition, and therefore there will no longer be any recognition of deferred revenues related to upfront payments from earlier license agreements.
– Loss per share: Loss per share was $0.18 for the first quarter of 2018, compared to a loss per share of $0.13 in the comparable period in 2017.

Abeona Recent Highlights:

April 23, 2018: Announced FDA Grants RMAT Designation to ABO-102 Gene Therapy in MPS IIIA
April 20, 2018: Announced EMA Grants Orphan Drug Designation in the European Union for ABO-202 Gene Therapy Program in Batten Disease
April 2, 2018: Announced Appointment of Carsten Thiel, Ph.D., as Chief Executive Officer
March 15, 2018: Announced FDA Grants Rare Pediatric Disease Designation for ABO-202 Gene Therapy Program in CLN1 Disease
February 12, 2018: Announced FDA Grants Orphan Drug Designation for ABO-202 Gene Therapy Program in Infantile Batten Disease
February 8, 2018: Reported Top-Line Data from Phase 1/2 Gene Therapy Trial in MPS IIIA
— ABO-102 results presented at WORLDSymposium for Lysosomal Diseases show significant time- and dose-dependent reduction of underlying disease pathology, including decreased CSF and urine GAGs (HS fragments) and diminished liver volumes
— Evidence of cognitive benefit at six months post treatment in Cohort 2 and at one year in Cohort 1
— Company receives FDA allowance to lower enrollment age to six months
February 7, 2018: Reported on Initial Safety and Biopotency Signals in MPS IIIB Gene Therapy Clinical Trial
— ABO-101 is well tolerated and demonstrates early biopotency signals with significant disease-specific heparan sulfate (HS) reductions in cerebral spinal fluid, urine, and plasma and greater than 300-fold increase in NAGLU enzyme activity observed in first subject at 30 days post injection
January 29, 2018: Announced FDA Grants Regenerative Medicine Advanced Therapy Designation for EB-101 Gene Therapy in Epidermolysis Bullosa

"2018 continues to be a year of execution for Abeona. With the expanded leadership, we look to a number of milestones in the year including the commencement of our pivotal Phase 3 trial in EB, the completion of enrollment in our Phase 1/2 trial in MPS IIIA, and significant progress on our in-house GMP manufacturing facility," stated Steven H. Rouhandeh, Abeona’s Executive Chairman. "With the team focusing their efforts and leading the charge on clinical development, we look forward to our continued dialogue with the regulatory bodies and advancing our gene therapies to rare disease patients."

On May 11, 2018 Paul Changelian, Ph.D., Vice President, Biology, Aclaris Therapeutics, Inc. (NASDAQ:ACRS), reported that it will speak at the 2018 American Hair Research Summit on "Preclinical to Clinical Translation of JAK Kinase Inhibitors" on May 15 (Press release, Aclaris Therapeutics, MAY 11, 2018, View Source [SID1234526536]). The meeting is organized by the American Hair Research Society (AHRS) and will take place May 14-16 in Orlando, Florida.

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Dr. Changelian will review the history of drug discovery targeting Janus kinase (JAK) enzymes. JAK inhibitors initially were conceived as drugs to reduce organ transplant rejection but gradually were found to have utility in autoimmune diseases and oncology. Harnessing the benefits of JAK inhibition for dermatological conditions will require minimizing the potential for over suppression of the immune system and subsequent infections, through strategies such as identifying molecules highly selective for JAK1 and JAK3 inhibition or developing topical formulations with high skin permeability while minimizing systemic absorption. Current efforts to expand the range of diseases that could be targeted with JAK inhibitors will be discussed with emphasis on hair disorders, including alopecia areata and androgenetic alopecia.

Dr. Changelian is known for his key role in the discovery and development of tofacitinib, a pan-JAK inhibitor, while working as a scientist at Pfizer. In 1993, he first heard about the National Institutes of Health’s discovery of an enzyme in the Janus family of kinases involving the immune function. Seven years after initiating the discovery program, Dr. Changelian and the Pfizer immunology team selected tofacitinib for clinical development. Tofacitinib became the first JAK inhibitor approved by the FDA for an autoimmune disease. Dr. Changelian joined Aclaris in 2017 when Aclaris acquired Confluence Life Sciences, Inc., a company founded by former Pfizer scientists.

On May 11, 2018 Adamis Pharmaceuticals Corporation (NASDAQ: ADMP) reported financial results and a business update for the first quarter ended March 31, 2018 (Press release, Adamis Pharmaceuticals, MAY 11, 2018, View Source [SID1234526537]).

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Dr. Dennis J. Carlo, President and Chief Executive Officer of Adamis Pharmaceuticals, said, "We have made a substantial amount of progress on several fronts since the beginning of the year. The development of our product pipeline continues to move forward and we have several target milestones that we hope to reach later this year. We believe the completion of these milestones will increase shareholder value. As for our progress on Symjepi , we continue to be pleased with developments regarding our discussions with potential commercialization partners. We believe that we are closer to concluding the process of naming our licensing partner and we remain focused on bringing this product to market."

Company Highlights and Product Updates

Some of the company’s product updates and accomplishments since the beginning of 2018 include the following:

Symjepi (epinephrine) Injection 0.15mg – The FDA determined that the company’s NDA for Symjepi (epinephrine) Injection 0.15mg was sufficiently complete to permit a substantive review and indicated that no potential review issues were identified as of the date of the agency’s communication in February. Approval is expected in the second half of this year.

Symjepi human factors data – Adamis presented human factors data for Symjepi at the American Academy of Allergy Asthma and Immunology joint congress with the World Allergy Organization, and another human factors study was published in the Annals of Allergy, Asthma and Immunology.

APC-1000 (Beclomethasone HFA) – Our product candidate received approval from the FDA to proceed with Phase 3 clinical studies. We continue to make progress in this area.

APC-6000 (Naloxone PFS) – We are working toward filing a New Drug Application (NDA) later this year and have completed pharmacokinetic (PK) studies.

Sales of several products sold by our U.S. Compounding, Inc. subsidiary have been increasing including Adamis’ unique compound to manage ulcers in horses. One horse that was receiving U.S. Compounding’s product recently came in first in the 2018 Kentucky Derby.

Second Quarter Financial Results

Revenues were approximately $3.2 million and $3.0 million for the three months ended March 31, 2018 and 2017, respectively. The increase in revenues (4.6%) for the three months ended March 31, 2018 compared to the comparable period of 2017 reflected an increase in the volume of sales of USC’s compounded pharmaceutical formulations resulting in part from increased sales and marketing personnel and efforts. The company’s revenues for the first quarter of 2018 increased approximately 11.9% compared to the revenues for the fourth quarter of 2017.

At March 31, 2018, the Company had cash and cash equivalents of $10.1 million. Net cash used in operating activities for the three months ended March 31, 2018 and 2017, was approximately $7.9 million and $3.5 million, respectively. Net cash used in operating activities increased primarily due to the increase in operating losses; increase in accounts receivable, inventories and prepaid expenses; and a decrease in accounts payable and accrued expenses as compared to 2017.

Selling, general and administrative expenses ("SG&A") for the three months ended March 31, 2018 and 2017 were approximately $6.5 million and $5.6 million, respectively. The increase in SG&A expenses was primarily due to new hires, compensation and stock option expenses, increases in accounting, audit and other professional fees, patent expenses, selling expenses and market research expenses related to Symjepi (epinephrine) and our APC-6000 product candidate.

Research and development expenses were approximately $2.2 million and $1.5 million for the three months ended March 31, 2018 and 2017, respectively. The increase in research and development expenses for the three months ended March 31, 2018, compared to the comparable period of the prior year was due in part to an increase in development costs of our product candidates and compensation and stock option expenses, in part due to new hires.

Net loss for the first quarter of 2018 was approximately $7.6 million, compared to net loss of approximately $5.8 million for the same period in 2017.

Future Milestones

Some of the company’s goals for the 2018 year include the following:

Finalizing and announcing the commercialization strategy for Symjepi (epinephrine) Injection 0.3mg;

FDA approval for Symjepi TM (epinephrine) Injection 0.15mg;

Initiate pivotal Phase 3 studies of APC-1000 in asthmatics;

Complete a "proof of concept" study with dry powder inhaler platform using fluticasone;

Filing an NDA for Naloxone injection;

Increase sales of compounded medications from our U.S. Compounding, Inc. subsidiary by at least 30%.

On May 11, 2018 Celsion Corporation (NASDAQ: CLSN), an oncology drug development company, reported financial results for the quarter ended March 31, 2018 and provided an update on its development programs for ThermoDox, its proprietary heat-activated liposomal encapsulation of doxorubicin, and GEN-1, an IL-12 DNA plasmid vector encased in a nanoparticle delivery system, which enables cell transfection followed by persistent, local secretion of the IL-12 protein (Press release, Celsion, MAY 11, 2018, View Source [SID1234526538]). The Company’s lead program is ThermoDox, which is currently in Phase III development for the treatment of primary liver cancer. The Company’s immunotherapy candidate, GEN-1, is currently in Phase I/II development for the localized treatment of ovarian cancer.

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"Celsion continues to make significant progress with our two ongoing clinical development programs for ThermoDox and GEN-1. With sound fundamentals and a strong balance sheet, we are well positioned to complete enrollment in our ongoing 550 patient global, pivotal Phase III OPTIMA Study in primary liver cancer and to initiate our 130 patient Phase I/II randomized OVATION II Study in newly diagnosed patients with ovarian cancer in June 2018 and reporting clinical findings from the Phase I cohort of 12 patients of the OVATION II by the end of 2018," said Michael H. Tardugno, Celsion’s chairman, president and chief executive officer. "Our GEN-1 immunotherapy has generated impressive results to-date. We expect to report final progression-free survival data from our Phase IB clinical trial (the OVATION I Study) by the end of the second quarter of 2018. These data will provide additional insights into GEN-1’s clinical and safety profile and reinforce our confidence in GEN-1’s potential to serve as a highly effective first-line therapy in newly diagnosed patients with ovarian cancer."

Recent Developments

ThermoDox

ThermoDox Highlighted at the International Liver Congress 2018 Symposium. On April 12, 2018, the Company announced a presentation and discussion of ThermoDox and the evolving treatment landscape for primary liver cancer or hepatocellular carcinoma (HCC), as part of a company-sponsored symposium at the International Liver Congress 2018, in Paris, France. The symposium titled, "Emerging Horizons in HCC: From Palliation to Cure," featured presentations by co-chairs and HCC experts, Ghassan Abou-Alfa, M.D., a board-certified medical oncologist at Memorial Sloan Kettering Cancer Center in New York City, and Riccardo Lencioni, M.D., FSIR, EBIR, professor at the University of Pisa School of Medicine.

Dr. Abou-Alfa’s presentation, "New Developments in Targeted Therapies for HCC: The Mounting Wave of Immuno-Oncology," discussed recent developments in treating HCC, including the role of tyrosine kinase inhibitors (TKIs), immuno-oncology and CAR-T therapies, as well as advancements in chemotherapy and combination treatment with local therapy. Prof. Lencioni’s presentation, "Rethinking Our Approach to Intermediate-Size HCC" focused on the increasing incidence and burden of HCC globally, the limited overall survival benefit with current therapies at later stages of disease progression, and the potential for ThermoDox to provide enhanced survival benefit with standardized radiofrequency ablation. The slides from Prof. Lencioni’s presentation are available on Celsion’s corporate website at www.celsion.com.

Data Monitoring Committee Unanimously Recommended Continuation of the OPTIMA Study in Primary Liver after its Planned Safety and Data Review from 411 Patients; Enrollment Now at 85%. On April 9, 2018, the Company announced that the independent Data Monitoring Committee for the Company’s 550-patient, pivotal Phase III clinical study of ThermoDox in combination with radiofrequency ablation for primary liver cancer (the OPTIMA Study), unanimously recommended that the study continue according to protocol to its data readout. The DMC’s recommendation was based on the Committee’s assessment of safety and data integrity of the first 75% of patients randomized in the trial as of February 5, 2018 and concluded that the integrity of the study is intact and that ThermoDox is safe for continued enrollment of newly diagnosed, intermediate-stage patients. An analysis of blinded data from the intent-to-treat population, consolidated for both arms, indicated that median progression free survival (PFS) was 20.8 months. This compares favorably to the HEAT Study median PFS of 13.8 months and is consistent with the hypothesis-generating estimates from the HEAT Study manuscript published in the October 2017 issue of the peer-reviewed medical journal, ‘Clinical Cancer Research.’ The OPTIMA Study’s design and statistical plan incorporates two pre-planned interim efficacy analyses by the DMC with the intent of evaluating safety, efficacy and futility to determine if there is overwhelming evidence of clinical benefit or a low probability of treatment success to continue, modify or terminate the study.

The DMC analysis in April 2018 was the last planned interim analysis prior to enrollment completion in the third quarter of 2018 with results from the first interim efficacy analysis available in the first half of 2019.

Presentation of ThermoDox HEAT Study Manuscript by Lead Author, Dr. Won Young Tak, at Korean Liver Cancer Association’s 12th Annual Scientific Meeting. On Feb. 12, 2018, the Company announced that an abstract discussing the Company’s Phase III HEAT study evaluating ThermoDox in combination with radiofrequency ablation was one of six selected for presentation as part of the lecture of the Presidential Selection at the Korean Liver Cancer Association’s 12th Annual Scientific Meeting in Seoul, South Korea. Dr. Tak’s presentation highlighted learnings from the Company’s 701 patient HEAT Study and included results from simulation studies and findings from the post hoc subgroup analysis. Dr. Tak noted that key findings from the study and analyses of ThermoDox plus RFA suggested that the therapeutic effect of ThermoDox plus RFA may be improved when the RFA dwell time for solitary lesions is greater than or equal to 45 minutes.

Dr. Tak’s presentation explored the hypothesis prompted by these findings: ThermoDox, when used in combination with RFA standardized to a minimum dwell time of 45 minutes (sRFA ≥ 45 min), may increase the overall survival (OS) of patients with hepatocellular carcinoma. The final OS analysis from the HEAT Study demonstrated that in a large, well bounded, subgroup of patients (n=285 patients, 41% of the previous 701 patient HEAT Study), treatment with a combination of ThermoDox and standardized RFA provided an average 58% improvement in OS compared to standardized RFA alone. The Hazard Ratio (HR) was 0.63 (95% CI 0.43 – 0.93) with a p-value of 0.0198. In this large subgroup, median OS for the ThermoDox plus standardized RFA group translated into a 25.4-month (more than 2.1 years) survival benefit over the standardized RFA-only group – totaling approximately 80 months (6-1/2 years, which is considered a curative treatment for HCC) for the ThermoDox plus standardized RFA group versus 53 months for the standardized RFA-only group.

GEN-1 Immunotherapy

Presentation of GEN-1 Clinical Development Program and Recent Clinical and Translation Research Data by Ovarian Cancer Expert at Oppenheimer & Co. Investor Event. On March 5, 2018, the Company announced that Premal H. Thaker, M.D., M.S., a nationally recognized expert in gynecologic oncology, Associate Professor of Obstetrics and Gynecology at the Siteman Cancer Center at the Washington University School of Medicine in St. Louis, and investigator in Celsion’s GEN-1 development program presented, "Ovarian Cancer: New Horizons and Treatments" at an investor event hosted by Oppenheimer & Co. in New York City on March 1, 2018.

Dr. Thaker’s presentation highlighted the following:

GEN-1 is a novel new approach that is designed to deploy the anti-cancer mechanism of the potent, broad-spectrum immunotherapy, IL-12, without the toxicities associated with the recombinant IL-12 protein.

In a Phase I study of GEN-1, 14 newly diagnosed patients with Stage III/IV ovarian cancer were intraperitoneally administered GEN-1 plus neoadjuvant chemotherapy. Results from the study demonstrated immunological changes consistent with the ability of GEN-1 to increase local (peritoneal) levels of IL-12 and its downstream anti-cancer cytokines and reduction in vascular endothelial growth factor (VEGF; potent angiogenic factor that contributes to tumor angiogenesis) levels with little change in systemic circulation.

The study showed no serious systemic toxicities. These clinical findings, including a partial or complete response in 86% of patients, R0 resections in 100% of patients treated at the highest dose cohort and recently reported progression-free survival (PFS) of over 21 months compared to historical controls for PFS of approximately 12 months, support further evaluation of GEN-1’s safety and efficacy in patients with Stage III/IV ovarian cancer.

Corporate Development

Corporate Presentations at Two Investor Conferences. In March 2018, the Company presented at two investor healthcare conferences:

The B. Riley FBR Inaugural China Healthcare Investing & Partnering Symposium. The conference was held March 15-17, 2018 at The InterContinental Hotel Hangzhou, China.

The Oppenheimer 28th Annual Healthcare Conference. The conference was held March 20-21, 2018 at The Westin New York Grand Central in New York City.

The webcast of Celsion’s presentation at the Oppenheimer conference has been archived on the "News & Investors" section of Celsion’s corporate website at www.celsion.com.

Raised $27.5 Million in Gross Proceeds During the Fourth Quarter of 2017, and an Additional $1.3 Million in Gross Proceeds During the First Quarter of 2018. Recent minimally dilutive equity offerings totaling approximately $28.8 million in gross proceeds during the fourth quarter of 2017 through January 2018 have strengthened the Company’s balance sheet and will be used to support the Company’s development efforts and potentially significant clinical milestones for ThermoDox and GEN-1 clinical programs into the third quarter of 2019.

The Company raised $17.0 million in gross proceeds through the exercise of outstanding common stock warrants in early October 2017.

In October 2017, the Company completed an underwritten equity offering of shares of common stock and warrants to purchase common stock with Oppenheimer & Co. The gross proceeds of the offering were approximately $6.6 million.

In November 2017 and January 2018, the Company raised $5.2 million in gross proceeds off its ATM Equity Facility with Cantor Fitzgerald.

Financial Results

For the quarter ended March 31, 2018, Celsion reported a net loss of $4.5 million, or a loss of $0.25 per share, compared to a net loss of $5.2 million, or a loss of $3.09 per share, in the same period of 2017.

Net cash used for operating activities was $4.7 million for the quarter ended March 31, 2018, compared to $3.1 million for the same period of 2017. Cash, cash equivalents, short-term investments and interest receivable at March 31, 2018 was $20.8 million. Cash provided by financing activities was approximately $1.2 million during the quarter ended March 31, 2018.

Research and development costs were $2.7 million for the quarter ended March 31, 2018, compared to $3.5 million for the same period of 2017. Clinical development costs for the Phase III OPTIMA Study were $1.3 million for the quarter ended March 31, 2018 compared to $1.6 million for the same period of 2017. R&D costs for other development programs were lower because of the Company’s tighter clinical development focus around the pivotal Phase III OPTIMA Study for the treatment of primary liver cancer and the clinical development program for GEN-1 IL-12 immunotherapy for the localized treatment of ovarian cancer.

General and administrative expenses were $1.7 million for the quarter ended March 31, 2018, compared to $1.5 million for the same period of 2017. This modest increase was due to higher professional fees and an increase in non-cash stock option compensation expense.

As the Company paid off its Venture Debt Facility with Hercules Technology Growth Capital, Inc. during 2017, the Company did not have any interest expense in the first quarter of 2018. Interest expense was $0.1 million for the first quarter of 2017.

Quarterly Conference Call

The Company is hosting a conference call to provide a business update and discuss its first quarter 2018 financial results at 11:00 a.m. EDT on Friday May 11, 2018. To participate in the call, interested parties may dial 1-888-298-3457 (Toll-Free/North America) or 1-719-325-4917 (International/Toll) and ask for the Celsion Corporation First Quarter 2018 Earnings Call (Conference Code: 6550185) to register ten minutes before the call is scheduled to begin. The call will also be broadcast live on the internet at www.celsion.com.

The call will be archived for replay on Friday, May 11, 2018 and will remain available until Friday, May 25, 2018. The replay can be accessed at 1-719-457-0820 or 1-888-203-1112 (Toll-Free/USA) or 1-719-457-0820 (International/Toll) using Conference ID: 6550185. An audio replay of the call will also be available on the Company’s website, www.celsion.com, for 90 days after 2:00 p.m. EDT on Friday, May 11, 2018.

On May 11, 2018, DelMar Pharmaceuticals, Inc. (the "Company") presented the presentations to describe its business and preclinical program (Presentation, DelMar Pharmaceuticals, MAY 11, 2018, View Source [SID1234526539]).

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