On May 15, 2018 Exicure, Inc., the pioneer in gene regulatory and immunotherapeutic drugs utilizing three-dimensional, spherical nucleic acid (SNA) constructs, reported financial results for the first quarter ended March 31, 2018, and provided an update on corporate progress (Press release, Exicure, MAY 15, 2018, View Source;p=RssLanding&cat=news&id=2349297 [SID1234526643]).

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"Exicure continues to drive forward our SNA technology through ongoing clinical development. In the fourth quarter of 2017, we launched a Phase 1 clinical trial of AST-008, our TLR9 agonist developed for immuno-oncology applications. We expect to report results from this trial in the third quarter of 2018," said Dr. David Giljohann, Chief Executive Officer of Exicure. "We are also expanding our efforts in neurology, where pre-clinical results have suggested our spherical nucleic acid platform has advantages over existing technology. We look forward to presenting animal data later this summer."

Corporate Progress

Launched Phase 1 clinical trial of AST-008, a TLR9 agonist for immuno-oncology applications. Received authorization from Medicines and Healthcare products Regulatory Agency (MHRA) in the United Kingdom to conduct a Phase 1 clinical trial of AST-008 in the United Kingdom and began dosing healthy subjects during the fourth quarter of 2017. Our current plan anticipates preparing and commencing a Phase 1b/2 clinical trial for AST-008 late this year.
Began dosing patients in the Phase 1 clinical trial of XCUR17 in early April 2018. This trial is designed to test safety and efficacy of the drug. Twenty-five patients will be enrolled and dosed over a period of 26 days. We expect trial data during the third quarter of this year.
Generated pre-clinical data utilizing an SNA designed to stimulate the production of SMN2 mRNA for application in spinal muscular atrophy (SMA). These data suggest that the SNA design may potentially have superior pharmacodynamics properties compared to other nucleic acid therapeutic designs. We are currently collecting in vivo data in SMA mouse models.
Informed by our sponsoring market maker that FINRA has cleared our Form 211. This important step in our path toward trading on the OTCQB has been completed. We are now addressing final administrative items and expect to announce beginning of trading in the near future.
Strengthened management team with the appointment of Matthias Schroff as Chief Operating Officer. Dr. Schroff brings to Exicure a proven track record in clinical development and deep experience in the immuno-oncology, RNAi and gene expression, and TLR9 biology.
Pipeline Updates

AST-008: AST-008 is an SNA consisting of toll-like receptor 9, or TLR9 agonists designed for immuno-oncology applications. The Phase 1 clinical trial of AST-008 evaluates the safety, tolerability, pharmacokinetics, and pharmacodynamics of AST-008 by subcutaneous administration in healthy volunteers. Our current plan anticipates preparing and commencing a Phase 1b/2 clinical trial for AST-008 late this year. The Company ultimately plans to clinically advance AST-008 in combination with checkpoint inhibitors.

XCUR17: XCUR17 is an antisense SNA that targets the mRNA encoding IL-17RA, a protein that is considered essential in the initiation and maintenance of psoriasis. Our Phase 1 trial of XCUR17 is a microplaque study in patients with mild to moderate psoriasis.

AST-005: AST-005 is an SNA containing TNF antisense oligonucleotides and is intended to be applied in a gel to psoriatic lesions. AST-005 is the subject of our collaboration with Purdue Pharma L.P. Purdue Pharma has notified Exicure it has declined to exercise its option to develop AST-005 at this time, but that it also intends to retain rights relating to the TNF target, and Purdue reserves its right to continue joint development, with Exicure, of new anti-TNF drug candidates and to retain its exclusivity and other rights to AST-005.

First Quarter 2018 Financial Results and Financial Guidance

Cash Position: As of March 31, 2018, Exicure had cash and cash equivalents of $21.1 million compared to $25.8 million as of December 31, 2017.

Research and Development (R&D) Expenses: Research and development expenses were $3.3 million for the quarter ended March 31, 2018, compared to $3.5 million for the quarter ended March 31, 2017. The decrease in research and development expense of $0.2 million was primarily due to a net decrease in costs related to our clinical development programs of $0.6 million, partially offset by higher employee-related expenses of $0.2 million and higher platform and discovery-related expense of $0.2 million.

General and Administrative (G&A) Expenses: General and administrative expenses were $2.0 million for the quarter ended March 31, 2018, compared to $1.4 million for the quarter ended March 31, 2017. The increase in general and administrative expenses of $0.6 million was primarily due to higher legal costs associated with preparation and filing of form S-1 to register the shares of common stock sold last year in connection with our merger and private placement. Also contributing to the increase versus the prior quarter were expenses associated with being a public company and salary increases and new hires.

Net Loss: Net loss was $5.5 million for the quarter ended March 31, 2018, compared to net loss of $2.7 million for the quarter ended March 31, 2017. The $2.9 million increase in net loss is due principally to a $2.4 million decrease in non-cash collaboration revenue in addition to the net increase in operating expenses of $0.4 million discussed above. The quarter ended March 31, 2017 included $2.4 million of collaboration revenue which represented the amortization of deferred revenue associated with the upfront cash payment of $10.0 million received in December of 2016 connected with the Purdue collaboration. On January 1, 2018, we adopted ASC 606 and recorded any remaining unamortized deferred revenue under the Purdue collaboration to the beginning balance of accumulated deficit at January 1, 2018.

Cash Runway Guidance: Exicure believes that, based on its current operating plans and estimates of expenses, as of the date of this press release, its existing cash and cash equivalents as of March 31, 2018, will be sufficient to meet its anticipated cash requirements through March 31, 2019.

On May 15, 2018 Savara Inc. (NASDAQ:SVRA), an orphan lung disease company, reported that the Company’s Chief Executive Officer, Rob Neville, will present at the Bank of America Merrill Lynch 2018 Healthcare Conference on Tuesday May 15th, 2018 at 1:55 p.m. Pacific Time at the Encore Hotel in Las Vegas (Press release, Savara, MAY 15, 2018, View Source [SID1234526660]).

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Interested parties can access a live audio webcast on the Savara website at www.savarapharma.com. An archived presentation will be available on the website for 30 days.

On May 15, 2018 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a biopharmaceutical company dedicated to the development and delivery of high quality, cost-effective pharmaceutical products and innovative therapeutics to patients in the U.S., China and throughout the world, reported financial results for the first quarter and provided a review of recent accomplishments and anticipated upcoming milestones (Press release, CASI Pharmaceuticals, MAY 15, 2018, View Source [SID1234526614]).

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Ken K. Ren, Ph.D., CASI’s Chief Executive Officer, commented, "We have made significant progress in 2018, having acquired a portfolio of 25 US FDA-approved ANDAs from Sandoz, which has substantially broadened our product pipeline. The acquisition squarely enhances our strategic focus of capitalizing on the evolving CFDA landscape, which is expected to streamline and accelerate the approval process for high-quality generic products."

Dr. Ren continued, "The capital raise announced in March leaves us well-positioned to drive forward with our EVOMELA commercialization plans, and given the positive tenor of the April 2018 Center for Drug Evaluation (CDE) Expert Advisory Committee meeting, where no questions were posed and no new materials were requested, we remain encouraged that we will receive additional feedback from the CDE/CFDA regarding the EVOMELA application in the coming weeks. We remain committed to developing a strong and robust product portfolio through licensing opportunities as well as seeking other advantageous relationships that will further expand our footprint in both the U.S. and in China."

CASI Pharmaceuticals, Inc. / 9620 Medical Center Drive / Suite 300 / Rockville, MD 20850
Phone 240.864.2600 / Fax 301.315.2437

First Quarter and Recent Business Highlights

Company prepares for commercial launch of EVOMELA in China – In March 2018, CASI hired Thomas Zhang as General Manager of its Sales & Marketing team in China and is currently building out a robust and experienced team in China to support commercial operations there. Mr. Zhang has over 20 years of commercial experience in China, and prior to joining CASI was responsible for the successful launch and commercialization of Herceptin and Xeloda at Shanghai Roche Pharmaceuticals Co., Ltd. A team of seasoned sales and marketing professionals are being hired in preparation for the launch of EVOMELA following anticipated marketing authorization.

Update on advisory committee meeting notice from China Center for Drug Evaluation (CDE) – On April 26, 2018, the Center for Drug Evaluation (CDE), a group within the China FDA in charge of technical review, held a meeting of the Expert Advisory Anti-Tumor (Oncology) Drugs Committee (the "Advisory Committee") to review the EVOMELA application. In preparation for the Advisory Committee meeting, no additional questions were posed and the CDE requested no new analysis or materials. The Advisory Committee meeting did not include sponsor or public participants and the Advisory Committee will provide its recommendations directly to the CDE. CASI anticipates receiving feedback from the CDE/CFDA on the EVOMELA application in the coming weeks.

Acquired portfolio of 25 US FDA-approved ANDAs from Sandoz Inc. – In January 2018, the Company announced the acquisition of a portfolio of 25 US FDA-approved ANDAs, plus four pipeline ANDA’s that are pending FDA approval. CASI intends to select and commercialize certain products from the portfolio that have a unique market opportunity and cost-effective manufacturing in China and/or in the U.S.

Announced $50 million private placement to new and existing investors – In March 2018, the Company announced a $50 million private placement (common stock with accompanying warrants). Existing stockholders or their affiliates, including IDG-Accel China Growth Fund III, ETP Global Fund LP, and new stockholders including Robert W. Duggan, former Chairman and CEO of Pharmacyclics Inc., led the financing.

Upcoming presentations – CASI will provide a corporate update at the 2018 BIO International Convention (Boston, MA June 4-7).

Financial Results for the Quarter ended March 31, 2018

Cash Position: As of March 31, 2018, CASI had cash and cash equivalents of $49.9 million.

R&D Expenses: R&D expenses for the quarter ended March 31, 2018 were $1.7 million compared to $1.0 million in 2017, an increase of $0.7 million. The increase in R&D expenses primarily reflects costs associated with the technology transfer activities and regulatory support associated with the recently acquired ANDA portfolio, including non-cash intangible amortization expense of $0.3 million.

G&A Expenses: G&A expenses for the quarter ended March 31, 2018 were $1.3 million compared to $0.6 million in 2017. The increase in G&A over the prior year is primarily attributed to an increase in salary, benefits and recruitment expense in China, largely related to sales and marketing efforts to prepare for the anticipated launch of the Company’s first commercial product in China, as well as other general and administrative functions. There were also increased costs associated with business development, investor and public relations activities, and an increase in legal and other professional services fees during the 2018 period.

Net Loss: The Company reported a net loss of ($3.6 million), or ($0.05) per share, for the quarter ended March 31, 2018. This compares with a net loss of ($1.7 million), or ($0.03) per share for the first quarter of 2017. The increase in net loss is primarily due to costs associated the technology transfer activities and regulatory support for our ANDA portfolio, the write-off of approximately $0.7 million due to acquired in-process R&D primarily related to ANDAs not approved by the FDA, and increased costs associated with G&A functions, including employment costs for sales and marketing efforts, increased business development and investor relations activities, as well as other professional service fees.

On May 15, 2018 X4 Pharmaceuticals, a clinical stage biotechnology company developing novel CXCR4 antagonists to improve immune cell trafficking to treat cancer and rare disease, reported results from a pilot study of X4P-001-IO in combination with Opdivo (nivolumab) in patients with clear cell renal cell carcinoma (ccRCC) who are non-responsive to the anti-PD-1 checkpoint inhibitor Opdivo alone (Press release, X4 Pharmaceuticals, 15 15, 2018, View Source [SID1234526644]). The data were presented at the 16th Annual Meeting of the Association for Cancer Immunotherapy (CIMT) (Free CIMT Whitepaper), taking place May 15-17 in Mainz, Germany.

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Results from the nine patients with advanced ccRCC enrolled in the pilot study as of February 26, 2018 were presented at the CIMT (Free CIMT Whitepaper) meeting in a poster session on May 15th and an oral presentation on May 17th. All patients in the study were non-responsive to single agent Opdivo with either stable or progressive disease. Enrolled patients continued to receive standard bi-weekly Opdivo therapy and X4P-001-IO (400 mg, oral, once daily). Median duration of treatment with the combination was 3.7 months (range 1-10 months).

Highlights of the data presented at CIMT (Free CIMT Whitepaper) include:

X4P-001-IO in combination with Opdivo had acceptable toxicity. The most frequent adverse events were diarrhea, nasal congestion, dry eye, headache and cough. No grade 4 or 5 adverse events occurred. All Grade 3/serious adverse events were manageable with appropriate intervention.
Combination therapy with X4P-001-IO and Opdivo exhibited anti-tumor activity in some patients with advanced ccRCC who were previously non-responsive to single agent Opdivo therapy.
Four patients who had progressed on prior Opdivo monotherapy had a best response of stable disease with additional X4P-001-IO treatment.
Of the five patients who were stable on prior Opdivo monotherapy, one had a partial response with combination therapy.
"These data demonstrate that the combination with X4P-001-IO and nivolumab has the potential to augment responses in patients who previously received the anti-PD-1 checkpoint inhibitor nivolumab alone," said David F. McDermott, M.D., Beth Israel Deaconess Medical Center, Harvard Medical School and lead investigator of the study. "This preliminary data requires validation in larger studies as we continue to seek treatments to address the larger population of cancer patients who do not adequately respond to checkpoint inhibitors."

"These findings contribute to our growing understanding of combining CXCR4 antagonists with other agents such as checkpoint inhibitors," said Sudha Parasuraman, M.D., Chief Medical Officer of X4 Pharmaceuticals. "Because the mechanisms of CXCR4 antagonism and check point inhibition act at different points in the tumor immunity cycle, it is reasonable to consider the potential for synergistic activity."

About X4P-001-IO in Cancer

X4P-001-IO is an investigational selective, oral, small molecule antagonist of C-X-C receptor type 4 (CXCR4). CXCR4 is a chemokine receptor present in abundance on certain immune cells and cancer cells and it plays a critical role in immune cell trafficking, infiltration and activation in the tumor microenvironment. CXCR4 signaling is disrupted in a broad range of cancers, facilitating tumor growth by allowing cancer cells to evade immune detection and creating a pro-tumor microenvironment. X4P-001-IO has the ability to help restore immunity within the tumor microenvironment and has the potential to enhance the anti-tumor activity of approved and emerging oncology agents, such as checkpoint inhibitors and targeted therapies. X4P-001-IO is being investigated in several clinical studies in solid tumors.

On May 15, 2018 Nordic Nanovector ASA (OSE: NANO) reported its first quarter 2018 results on Wednesday, 30 May 2018 (Press release, Nordic Nanovector, MAY 15, 2018, View Source [SID1234553504]).

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First Quarter 2018 Results Presentation and Webcast

A presentation by Nordic Nanovector’s senior management team will take place at 8:30 am CEST on 30 May at:

Thon Hotel Vika Atrium, Munkedamsveien 45, 0250 Oslo

Meeting Room: NYLAND

The presentation will be recorded as a webcast and will be available at www.nordicnanovector.com in the section: Investors & Media

The results report and the presentation will be available at www.nordicnanovector.com in the section: Investors & Media/Reports and Presentation/Interim Reports/2018 from 7:00 am CEST the same day.

Results presentation in Norwegian

As announced in April, a separate presentation of the results in Norwegian, to be hosted by Nordic Nanovector’s CFO and Interim CEO, and its VP IR & Corporate Communications, will take place on Thursday, 31 May 2018 at 8:30 am CEST at:

Thon Hotel Vika Atrium, Munkedamsveien 45, 0250 Oslo

Meeting Room: VIPPETANGEN

To attend the meeting please email – [email protected]

The presentation will NOT be recorded as a webcast