On May 15, 2018 BerGenBio ASA (OSE: BGBIO), a clinical-stage biopharmaceutical company developing novel, selective AXL kinase inhibitors for multiple cancer indications, reported its results for the first quarter 2018 (Press release, BerGenBio, MAY 15, 2018, View Source [SID1234526612]). A presentation of the results by the Company’s management will take place today at 10.00 am CET in Oslo – details below.

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!

Richard Godfrey, Chief Executive Officer of BerGenBio, commented: "We are pleased with the progress made during Q1 2018. Patient recruitment into our global Phase II clinical proof-of-concept trials with bemcentinib is progressing well and we expect to deliver interim read-outs across all studies during 2018. Presentation of these results will be at major clinical congresses, including the annual American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) meeting in June. Coinciding with ASCO (Free ASCO Whitepaper), we will host a satellite event that will allow us to meet with our stakeholders and provide insights from KOLs and clinical experts on our selective AXL inhibitor bemcentinib as a potential cornerstone of cancer combination therapy. We believe that we will be able to demonstrate the significant potential of bemcentinib in cancer therapy by making tumour cells visible to the immune system and more susceptible to treatment with chemotherapy, targeted therapy and immuno-oncology drugs."

Highlights – First Quarter 2018

Good progress advancing bemcentinib’s proof-of-concept clinical development

First efficacy endpoint met in Phase II trial of bemcentinib/TARCEVA (erlotinib) combination in advanced lung cancer (NSCLC) patients
Recruitment completed in first stage of Phase II trial of bemcentinib in combination with KEYTRUDA in advanced breast cancer (TBNC) patients
Bemcentinib shown to be well tolerated in all patients enrolled across three combination trials with KEYTRUDA – data presented at ASCO (Free ASCO Whitepaper)-SITC 2018
Single agent therapy with bemcentinib led to increased immune activity in relapsed / refractory leukaemia (AML & MDS) patients – data presented at ASCO (Free ASCO Whitepaper)-SITC 2018
Post period

Private placement raising gross NOK 187.5 million from international institutional investors including from the USA, specialising in the biotechnology sector
Recruitment completed in the first stage of Phase II trial of bemcentinib in combination with KEYTRUDA in NSCLC patients
Preclinical data highlighting bemcentinib’s potential to reverse tumour immune suppression and enhance immune checkpoint inhibitor efficacy, presented at AACR (Free AACR Whitepaper) annual meeting
Publications describe the role of AXL signalling in, and potential therapeutic effect of selective AXL inhibition to counteract the progression of aggressive fibrosis in lung and liver diseases

Presentation and Webcast Details

A presentation by BerGenBio’s senior management team will take place at 10.00 am CET at:
Felix Konferansesenter, Bryggetorget 3, 0125 Oslo
The presentation will webcast live and the link will be available at www.bergenbio.com in the section Investors/ Financial Reports. A recording will be available shortly after the webcast has finished.

The results report and the presentation will be available at www.bergenbio.com in the section: Investors/ Financial Reports from 7:00 am CET the same day.

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]).

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!

"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]).

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!

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 Bristol-Myers Squibb Company (NYSE:BMY) reported that it will take part in the UBS 2018 Global Healthcare Conference on Tuesday, May 22, 2018, in New York. Charles Bancroft, executive vice president, Chief Financial Officer and Head of Global Business Operations, will answer questions about the company at 11:30 a.m. ET (Press release, Bristol-Myers Squibb, MAY 15, 2018, View Source [SID1234526613]).

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!

Investors and the general public are invited to listen to a live webcast of the session at View Source An archived edition of the session will be available later that day.

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.

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!

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.