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TRILLIUM THERAPEUTICS REPORTS FIRST QUARTER 2018 FINANCIAL AND OPERATING RESULTS

On May 11, 2018 Trillium Therapeutics Inc. (NASDAQ/TSX: TRIL), a clinical stage immuno-oncology company developing innovative therapies for the treatment of cancer, reported financial and operating results for the three months ended March 31, 2018 (Press release, Trillium Therapeutics, MAY 11, 2018, View Source [SID1234526543]).

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"Following the initial signals of monotherapy responses reported at ASH (Free ASH Whitepaper) last year, we have increasingly focused both our TTI-621 clinical trials on patients with T-cell malignancies throughout the first quarter of 2018" said Dr. Niclas Stiernholm, president and CEO of Trillium Therapeutics. "We have also been preparing to launch our second clinical CD47 program, TTI-622. Having both an IgG1 and an IgG4 SIRPaFc fusion protein in clinical testing should allow us to address important scientific questions related to the impact of the Fc region in various clinical scenarios, including combination therapy."

2018 First Quarter Highlights:

Reported refinements to both our phase 1 trials of TTI-621 to focus near-term efforts on patients with cutaneous T-cell lymphoma (CTCL) and peripheral T-cell lymphoma (PTCL). This action builds on the monotherapy results of TTI-621 presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2017 where weekly infusions of TTI-621 were shown to be well tolerated and intratumoral injection was observed to reduce local lesions in 9 out of 10 patients with mycosis fungoides, a common type of CTCL.
The U.S. Food and Drug Administration granted an Orphan Drug Designation to TTI-621 for the treatment of cutaneous T-cell lymphoma. Orphan Drug Designation qualifies the sponsor of the drug candidate for various development incentives, which include an exemption from fees under the Prescription Drug User Fee Act and a seven-year marketing exclusivity period following approval.
Presented preclinical TTI-622 data at the 2018 AACR (Free AACR Whitepaper) Annual Meeting demonstrating that TTI-622 induces the phagocytosis of a broad panel of tumor cells derived from patients with both hematological and solid tumors. As a monotherapy, TTI-622 treatment resulted in decreased tumor growth and improved survival in a B cell lymphoma xenograft model, as well as enhanced the efficacy of cetuximab (anti-EGFR) and daratumumab (anti-CD38) antibodies in solid and hematological xenograft models, respectively. We expect to enroll the first patient in a Phase 1 clinical trial of TTI-622 in Q2 2018.
First Quarter 2018 Financial Results

As of March 31, 2018, Trillium had cash and cash equivalents and marketable securities, and working capital of $73.9 million and $61.7 million, respectively, compared to $81.8 million and $68.9 million, respectively at December 31, 2017. The decrease in cash and cash equivalents and marketable securities, and working capital was due mainly to cash used in operations of approximately $9.5 million.

Net loss for the three months ended March 31, 2018 of $8.6 million was lower than the loss of $11.5 million for the three months ended March 31, 2017. The net loss was lower due mainly to a net foreign currency gain of $1.6 million for the three months ended March 31, 2018, compared to a net foreign currency loss of $0.4 million in the prior year period. Research and development expenses decreased by $0.9 million in 2018 as a result of lower manufacturing activity for TTI-621 and TTI-622. These decreases were partially offset by higher clinical trial expenses.

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]).

"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]).