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Rocket Pharmaceuticals Reports First Quarter 2018 Financial Results and Operational Highlights

On May 11, 2018 Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) ("Rocket"), a leading U.S.-based multi-platform gene therapy company, reported financial results for the quarter ended March 31, 2018, and provided an update on the Company’s recent achievements, as well as upcoming milestones (Press release, Rocket Pharmaceuticals, MAY 11, 2018, View Source;p=irol-newsArticle&ID=2348660 [SID1234526542]).

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"2018 is off to a great start for Rocket. During the first quarter, we continued to make excellent progress advancing our pipeline of lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy programs towards key value inflection points. We look forward to presenting updated patient data from the Fanconi Anemia program later this month, and to disclosing our AAV-based gene therapy program by the end of the year," said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. "We remain on track for multiple regulatory filings across our pipeline over the next 12 months. As a result, there is the potential for four programs to be in the clinic in 2019, with clinical data for up to two programs."

Dr. Shah continued, "While near-term milestones are certainly important, Rocket’s long-term strategy is based on a sustainable and integrated multi-platform approach. We remain focused on our mission of continued innovation to deliver best-in-class curative cell and gene therapies to patients living with devastating diseases."

Anticipated Milestones Over the Next 12 -18 Months

Fanconi Anemia (FA): Updated patient data from the ongoing Phase 1/2 study of RP-L102 conducted with CIEMAT is expected over the next 12 months. The next update will be provided at the American Society of Gene and Cell Therapy’s (ASGCT) (Free ASGCT Whitepaper) 21st Annual Meeting in Chicago. The oral presentation is scheduled during the Presidential Symposium on Friday, May 18, 2018, at 2:45 p.m. Central Time.
Leukocyte Adhesion Deficiency-I (LAD-I): An Investigational Medicinal Product Dossier (IMPD) is expected to be filed in the fourth quarter of 2018. Clinical data is expected over the next 12-18 months.
Pyruvate Kinase Deficiency (PKD): An IMPD application is expected to be filed in early 2019.
Infantile Malignant Osteopetrosis (IMO): Preclinical studies continue to advance in support of filing a first-in-human clinical study.
AAV: Disclosure of the disease indication and preclinical data is planned for the second half of 2018.
Upcoming Conferences

Bank of America Merrill Lynch Health Care Conference 2018. Rocket is scheduled to present on Tuesday, May 15, at 9:20 a.m. Pacific Time.
UBS Global Healthcare Conference. Rocket is scheduled to present on Wednesday, May 23, at 9:30 a.m. Eastern Time.
Jefferies Global Healthcare Conference. Rocket is scheduled to present on Thursday, June 7, at 2:00 p.m. Eastern Time.
First Quarter 2018 Financial Results

Cash position. Cash, cash equivalents and short-term investments as of March 31, 2018, were $182.7 million, which includes a $52.0 million fully convertible debenture which expires in 2021.
R&D expenses. Research and development expenses were $5.7 million for the quarter ended March 31, 2018, compared to $2.3 million for the quarter ended March 31, 2017.
G&A expenses. General and administrative expenses were $8.7 million for the quarter ended March 31, 2018, compared to $0.6 million for the quarter ended March 31, 2017. The increase in G&A expenses was primarily due to one-time merger related expenses of $5.3 million.
Net loss. Net loss was $15.3 million or $(0.42) per share (basic and diluted) for the quarter ended March 31, 2018, compared to $2.7 million or $(0.39) per share (basic and diluted) for the quarter ended March 31, 2017.
Shares outstanding. Approximately 39.4 million shares of common stock were outstanding as of March 31, 2018.
Financial Guidance

Cash position. Based on its current operating plan, Rocket expects its cash, cash equivalents and short-term investments as of March 31, 2018, will be sufficient to run its operations into 2020.

Ligand to Participate in Upcoming Investor Conferences

On May 11, 2018 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported that company executives are scheduled to participate in the following upcoming investor conferences (Press release, Ligand, MAY 11, 2018, View Source [SID1234526541]):

• Berenberg Conference USA in Tarrytown, New York. Presentation takes place on Thursday, May 24, 2018 at 11:45 a.m. Eastern time (8:45 a.m. Pacific time). Matt Korenberg, CFO will attend for Ligand.

• 15th Annual Craig-Hallum Institutional Investor Conference in Minneapolis. Conference takes place on Wednesday, May 30, 2018 with one-on-one meetings only. John Higgins, CEO, Matt Foehr, COO and Matt Korenberg, CFO will attend for Ligand.

• Jefferies Healthcare Conference in New York City. Presentation takes place on Wednesday, June 6, 2018 at 8:00 a.m. Eastern time (5:00 a.m. Pacific time). Matt Korenberg, CFO will attend for Ligand.

A live webcast of the Jefferies conference presentation will be available on Ligand’s website at www.ligand.com. A replay of the presentations will be archived on the website for 30 days.

Galectin Therapeutics Reports 2018 First Quarter Financial

Results and Provides Business Update

On May 11, 2018 Galectin Therapeutics Inc. (NASDAQ: GALT), the leading developer of therapeutics that target galectin proteins, reported financial results for the three months ended March 31, 2018, and provided a business update (Press release, Galectin Therapeutics, MAY 11, 2018, View Source [SID1234526540]). These results are included in the Company’s Quarterly Report on Form 10-Q, which has been filed with the U.S. Securities and Exchange Commission and is available at www.sec.gov.

"Three additional analyses conducted on the results of the NASH-CX trial since we published initial, top-line trial results further support our belief that GR-MD-02 is the first compound to demonstrate clinically meaningful positive effects in patients with NASH cirrhosis without esophageal varices. First, a statistically significant correlation was identified between the decrease in portal pressure (HVPG, or hepatic venous pressure gradient) and the improvement in hepatocyte ballooning treatment with GR-MD-02 at 2 mg/Kg. Second, an ad hoc analysis examining the PK-PD (pharmacokinetics-pharmacodynamics) correlation between human data and mouse NASH model showed that the apparent lack of a dose response in the 8 mg/Kg dose group (GR8) may be due to very high levels of GR-MD-02 in the bloodstream. Finally, results from the 13C-Methacetin Breath Test, a measure of liver function, conducted as part of the trial found that results for patients without baseline varices mirrored the results for changes in HVPG. This additional analysis further supports our original report of the NASH-CX findings which demonstrated a significant improvement in HVPG in patients without varices and has been provided to the FDA as part of our proposed plan for a Phase 3 trial," said Dr. Peter Traber, CEO and Chief Medical Officer of Galectin Therapeutics.

"About half of the total population of patients with NASH cirrhosis do not have esophageal varices. In addition, endoscopy to evaluate for varices is part of the standard of care for patients with newly diagnosed NASH cirrhosis. Consequently, the sub-group of NASH patients that may

benefit from our compound is clear at initial diagnosis. Because the sub-group that had a statistically significant response to GR-MD-02 is routinely identified, we believe there is sound logic to pursue further investigation."

Summary of Key Development Programs and Updates

Since reporting the initial NASH-CX trial results in December 2017, continued analysis of the data has led to three additional findings:

The findings from the 13C-Methacetin Breath Test conducted as part of the trial found that results for patients without baseline varices mirrored the results for changes in HVPG. This further supports our findings as well as represents a significant finding in the search to discover an effective non-invasive test for liver function and NASH.

Reinforcing the positive effects of GR-MD-02, a statistically significant (p=0.04) correlation was identified between the decrease in portal pressure (HVPG) and the improvement in hepatocyte ballooning (viz., representing a decrease in liver cell death) upon treatment with GR-MD-02 at 2 mg/Kg. This suggests an important pathophysiological link between the improvement in liver biopsy and reductions in HVPG. To our knowledge, this is the first time that such a correlation has been demonstrated in a human clinical trial in patients with NASH cirrhosis.

An ad hoc analysis examining the PK-PD correlation between human data and mouse NASH model showed that the apparent lack of a dose response in the 8 mg/Kg dose group (GR8) may be due to very high levels of GR-MD-02, where excessive levels of GR-MD-02 are less effective. This was supported when a statistically significant difference was observed between the GR8 patient group with high serum drug levels (> 12,000 µg*hr/mL) and those with lower (< 12,000 µg*hr/mL) serum drug levels, where those with the lower serum drug levels had a positive response on HVPG.

The Company made an oral presentation at the International Liver Meeting in April 2018 in Paris. Dr. Naga Chalasani, one of the principal investigators on the NASH-CX clinical trial, led a session entitled, "A multicenter, randomized, double-blind, PLB-controlled trial of Galectin-3 inhibitor (GR-MD-02) in patients with NASH cirrhosis and portal hypertension."

The company continues to enroll cohort 3 (GR-MD-02 8 mg/kg) of the pembrolizumab (KEYTRUDA) combination immunotherapy clinical trial, which will include at least 10 patients with melanoma, to provide a larger group of patients to evaluate. It is hoped additional data can be reported in mid-2018, when we anticipate a decision on progressing to phase 2.
Financial Results

For the three months ended March 31, 2018, the Company reported a net loss applicable to common stockholders of $4.5 million, or $0.12 per share, compared with a net loss applicable to common stockholders of $5.2 million, or $0.15 per share, for the three months ended March 31, 2017. The decrease is largely due to lower research and development expenses as our Phase 2 clinical program is winding down, somewhat offset by higher non-cash stock-based compensation expense in the three months ended March 31, 2018.

Research and development expense for the three months ended March 31, 2018, was $2.3 million, compared with $3.8 million for first quarter of 2017. The decrease primarily relates to the winding down of the NASH-CX Phase 2b clinical trial.

General and administrative expense for the three months ended March 31, 2018, was $1.9 million, compared with $1.2 million for first quarter of 2017, with the increase primarily due to an increase in non-cash stock-based compensation expense and an increase in business development and investor relations expenses.

As of March 31, 2018, the Company had $4.0 million of non-restricted cash and cash equivalents in addition to a $10 million line of credit which has not yet been used. The Company believes it has sufficient cash to fund currently planned operations and research and development activities through at least March 31, 2019.

INVESTOR PRESENTATION

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]).

Celsion Corporation Reports First Quarter 2018 Financial Results

and Provides Business Update

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.

"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.