On November 6, 2018 Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX), a clinical stage biopharmaceutical company focused on addressing the large unmet need in transthyretin (TTR) amyloidosis (ATTR), reported its financial results for the quarter ended September 30, 2018 and provided an update on the Company’s recent achievements (Press release, Eidos Therapeutics, NOV 6, 2018, View Source [SID1234576272]).

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!

"We are working to advance the AG10 clinical development program," said Neil Kumar PhD, chief executive officer of Eidos. "The Phase 1 data demonstrated that AG10 was well tolerated at blood concentrations resulting in near-complete TTR stabilization in healthy volunteers. We are announcing the results from the Phase 2 study in ATTR cardiomyopathy patients at the 2018 American Heart Association Annual Scientific Sessions on November 10, 2018."

Recent Achievements and Upcoming Milestones

Eidos presented Phase 1 data at the 2018 Annual Scientific Meeting of the Heart Failure Society of America, demonstrating that AG10 was well tolerated and establishing clinical proof-of-concept in healthy adult volunteers.

The U.S Food & Drug Administration granted Orphan Drug Designation to AG10 for the treatment of ATTR.

The European Medicines Agency adopted a positive opinion for the designation of AG10 as an orphan medicinal product for the treatment of ATTR.

The Journal of Medicinal Chemistry published the design and preclinical characterization of AG10, demonstrating that AG10’s potentially superior stabilizing activity is driven by the unique ability to mimic the disease-protective T119M mutation and its selectivity for TTR.

The Phase 2 study of AG10 in ATTR cardiomyopathy (ATTR-CM) wild-type and mutant patients with symptomatic heart failure (NYHA Class II-III) concluded and eligible subjects entered a long term, open label extension study.

Eidos will present the Phase 2 data for AG10 in ATTR-CM at the Annual Scientific Sessions of the American Heart Association (AHA) in a late-breaking Featured Science oral presentation on November 10, 2018 at 10am EST. Eidos will also host a conference call and webcast on November 12, 2018 at 8am EST to discuss the results of the Phase 2 trial. Details for the conference call can be found at www.eidostx.com.
Financial Results for the Third Quarter 2018

Cash and cash equivalents totaled $166.6 million at September 30, 2018 compared with $5.5 million at December 31, 2017.

Research and development expenses were $7.9 million for the third quarter of 2018, compared to $2.3 million for the same period of 2017, an increase of $5.6 million. The increase was primarily due to increased expenses for contract consultants, contract manufacturing and other activities for AG10 clinical trials and increases in headcount and related salaries and expenses.

General and administrative expenses were $2.6 million for the third quarter of 2018 compared to $0.5 million for the same period in 2017, an increase of $2.1 million. The increase was primarily due to increased salaries and employee-related expenses and increases in professional fees and services in connection with becoming a public company.

Net loss for the quarter ended September 30, 2018 was $10.2 million or $0.29 per common share, compared to a net loss of $2.8 million or $0.74 per common share for the same period in 2017.

About AG10
AG10 is an orally administered small molecule designed to potently stabilize tetrameric transthyretin, or TTR, thereby halting at its outset the series of molecular events that give rise to amyloidosis, or ATTR. AG10 has completed a Phase 2 clinical trial in patients with ATTR cardiomyopathy and symptomatic heart failure. Results from this trial will be presented on November 10, 2018 at the American Heart Association’s Annual Scientific Sessions.

AG10 was designed to mimic a naturally-occurring variant of the TTR gene (T119M) that is considered a "rescue mutation" because it has been shown to prevent ATTR in individuals also carrying a pathogenic, or disease-causing, mutation in their other copy of the TTR gene. To our knowledge, AG10 is the only TTR stabilizer in development that has been observed to mimic the "super-stabilizing" properties of this rescue mutation that have been well described.

On November 6, 2018 Exicure, Inc. (OTCQB:XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing three-dimensional, spherical nucleic acid (SNA) constructs, reported financial results for the third quarter ended September 30, 2018, and provided an update on corporate progress (Press release, Exicure, NOV 6, 2018, View Source;p=RssLanding&cat=news&id=2375500 [SID1234530761]).

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!

"During the quarter, we made progress in our clinical program in immuno-oncology while expanding our preclinical data in a growing number of disease models," said Dr. David Giljohann, Chief Executive Officer of Exicure. "The FDA informed us that our Phase1b/2 clinical trial in immuno-oncology may proceed. We also completed patient dosing in a Phase1 trial for Exicure’s lead anti-inflammatory compound. Additionally, the Exicure team continues to develop promising preclinical data in neurology, as well as demonstrated the preclinical delivery of our platform in the gastrointestinal tract. All of this work supports a core strategic objective of developing our digital medicines platform in oncology, genetically defined disorders, and inflammatory disease," added Dr. Giljohann.

Corporate Progress

Announced launch of a Phase1b/2 trial for AST-008, Exicure’s TLR9 agonist drug candidate designed for immuno-oncology applications. The IND for AST-008 has been opened by the FDA and we have been informed by the FDA that our proposed Phase1b/2 trial may proceed. We expect patient dosing to begin late this year.
Completed the Phase 1 clinical trial of AST-008 which demonstrated the desired highly potent immune system activation without serious adverse events or dose limiting toxicity.
Completed patient dosing in the Phase 1 clinical trial of XCUR17, our clinical stage anti-inflammatory drug candidate being tested in psoriasis. We expect topline results in 2018.
Presented data at the Oligonucleotide Therapeutics Society meeting showing the distribution and therapeutic efficacy of Exicure’s technology through oral delivery to the GI tract.
Developed preclinical bio-distribution data of Exicure’s platform after intrathecal injection, demonstrating a favorable distribution profile in the CNS, and enabling the potential expansion of the Exicure platform into the central nervous system.
Raised approximately $22.0 million in gross proceeds at a per share price of $4.50 in a private placement offering.
Clinical Updates

AST-008: AST-008 is an SNA consisting of toll-like receptor 9 (TLR9) agonists designed for immuno-oncology applications. The Phase 1 clinical trial of AST-008 was completed during the quarter and demonstrated our desired highly potent immune system activation without serious adverse events or dose limiting toxicity. The IND for AST-008 has been opened by the FDA and we have been informed that our proposed Phase1b/2 trial may proceed. We expect to dose the first patient late this year.

XCUR17: XCUR17 is an antisense SNA that is designed to target the mRNA encoding IL-17RA. In our Phase 1 trial, XCUR17 is being tested in a microplaque study in patients with mild to moderate psoriasis. We have completed patient dosing and expect to report topline results late in 2018.

Third Quarter 2018 Financial Results and Financial Guidance

Cash Position: As of September 30, 2018, Exicure had cash and cash equivalents of $32.4 million compared to $25.8 million as of December 31, 2017.

Research and Development (R&D) Expenses: R&D expense was $4.0 million for the three months ended September 30, 2018 and $3.1 million for the three months ended September 30, 2017, an increase of $0.9 million, or 28%. The increase in R&D expense of $0.9 million was primarily due to higher employee-related expenses of $0.4 million, higher clinical development programs expense of $0.3 million, and higher platform and discovery-related expense of $0.2 million.

General and Administrative (G&A) Expenses: General and administrative expense was $1.9 million for the three months ended September 30, 2018 and $1.3 million for the three months ended September 30, 2017, an increase of $0.6 million, or 51%. This increase was due to higher costs associated with being a public company of $0.3 million, including higher expense for investor and public relations, director and officer insurance, and transfer agent, over the counter and other regulatory compliance related matters, as well as higher legal costs of $0.2 million mostly related to 2018 financing activities, and higher compensation and related expense of $0.1 million associated with salary increases.

Net Loss: Net loss was $5.3 million for the quarter ended September 30, 2018, compared to net loss of $1.9 million for the quarter ended September 30, 2017. The $3.4 million increase in net loss was due principally to a decrease in revenue of $2.4 million that mostly reflects the absence of revenue recognized in the prior period related to the amortization of the upfront payment, and certain reimbursable R&D activities pursuant to a research collaboration, option and license agreement. In addition, we had an increase in Other income of $0.5 million principally due to the fair value adjustment of our common stock warrant liability offsetting the increases in R&D and G&A discussed above.

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 September 30, 2018, will be sufficient to meet its anticipated cash requirements into the first quarter of 2020.

On November 6, 2018 Portola Pharmaceuticals, Inc. (Nasdaq: PTLA) reported that the Company will participate in the following investor conferences in November and December (Press release, Portola Pharmaceuticals, NOV 6, 2018, View Source;p=RssLanding&cat=news&id=2375709 [SID1234530782]):

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!

Credit Suisse’s 27th Annual Healthcare Conference on Tuesday, November 13, 2018, at 2:50 p.m. Mountain Time in Scottsdale, AZ. The Company will participate in a fireside chat at the conference, which will be webcast live and available for replay from the Investor Relations section of Portola’s website at www.portola.com.

Citi’s Global Healthcare Conference on Thursday, December 6, 2018, in New York, NY. The Company will conduct one-on-one meetings with institutional investors at this conference.

On November 6, 2018 RedHill Biopharma Ltd. (Nasdaq: RDHL) (Tel-Aviv Stock Exchange: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company primarily focused on proprietary drugs for gastrointestinal diseases, reported that it will report its third quarter 2018 financial results on Tuesday, November 13, 2018 (Press release, RedHill Biopharma, NOV 6, 2018, View Source [SID1234530864]).

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!

The Company will host a conference call on Tuesday, November 13, 2018 at 8:30 a.m. EST to review the financial results and business highlights.

To participate in the conference call, please dial one of the following numbers 15 minutes prior to the start of the call: United States: +1-866-575-6539; International: +1-929-477-0402; and Israel: +972-3-376-1315. The access code for the call is: 2166581.

The conference call will be broadcast live and will be available for replay for 30 days on the Company’s website, View Source Please access the Company’s website at least 15 minutes ahead of the conference call to register.

On November 6, 2018 Jounce Therapeutics, Inc. (NASDAQ: JNCE), a clinical stage company focused on the discovery and development of novel cancer immunotherapies and predictive biomarkers, reported that it will report third quarter 2018 financial results and provide a corporate update before market open on Tuesday, November 13, 2018 (Press release, Jounce Therapeutics, NOV 6, 2018, View Source;p=RssLanding&cat=news&id=2375531 [SID1234530885]). Jounce Therapeutics’ management team will host a live conference call and webcast at 8:00 a.m. ET.

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!

Conference Call and Webcast
To access the conference call, please dial (866) 916-3380 (domestic) or (210) 874-7772 (international) and refer to conference ID 4689666. The live webcast can be accessed under "Events & Presentations" in the Investors and Media section of the company’s website at www.jouncetx.com. The webcast will be archived and made available for replay on the company’s website approximately two hours after the call and will be available for 30 days thereafter.