On November 6, 2018 Supernus Pharmaceuticals, Inc. (NASDAQ: SUPN), a pharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, reported record financial results for the third quarter of 2018 and related Company developments (Press release, Supernus, NOV 6, 2018, View Source [SID1234530847]).

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!

"Supernus generated another strong quarter of growth, setting a new record for quarterly net product sales of $100 million," said Jack Khattar, President and CEO of Supernus Pharmaceuticals. "Despite the market introduction of new competitive preventive treatments for migraine, Trokendi XR continued to capture a greater portion of the topiramate market. For Oxtellar XR, we continue to prepare for the potential launch of the monotherapy indication for partial seizures."

Progress of Product Pipeline
Given the recently accelerated development timeline for SPN-812 that positions its potential regulatory approval and commercial launch ahead of SPN-810, the Company has directed its resources to prioritize filing of the New Drug Application (NDA) and potential commercial launch of SPN-812 in the United States.

As a result, the following are the updated plans and timelines for both product candidates:

SPN-812 – Novel non-stimulant for the treatment of ADHD

The Phase III program consists of four three-arm, placebo-controlled trials: P301 and P303 trials in patients 6-11 years old and P302 and P304 trials in patients 12-17 years old.
The Company expects to announce top-line data from P301 and P303 pediatric trials concurrently in early December 2018, and from P302, the first adolescent Phase III trial, by the end of December 2018. Top-line data from the second adolescent Phase III trial, P304, are expected by the end of the first quarter of 2019.
The Company expects to submit an NDA for SPN-812 in the second half of 2019, and to launch it, pending U.S. Food and Drug Administration (FDA) approval, in the second half of 2020.
SPN-810 – Treatment of Impulsive Aggression in patients with ADHD

As expected, the first Phase III trial (P301) has reached its original enrollment target. However, given the aforementioned prioritization of SPN-812 and that top-line data from the second Phase III trial (P302) is expected around mid-2019, the Company has decided to keep P301 enrollment active until data from both trials can be released concurrently instead of sequentially. This change does not impact the timing of submission of the NDA for SPN-810, given that the NDA submission is rate-limited by completion of the P302 trial and generation of data in the adolescent patient population.
The Company continues to observe enrollment in the open label extension (OLE) study at 90% or higher. On average, a patient in the OLE study remains on SPN-810 treatment for 9.5 months, which we believe is an encouraging sign of the tolerability and efficacy of SPN-810.
Patient dosing has been initiated in the Phase III trial in adolescent patients.
The Company expects to submit an NDA for SPN-810 in the second half of 2020, and to launch it, pending FDA approval, in the second half of 2021.
SPN-604 (formerly known as Oxtellar XR for Bipolar)

The Company continues to expect initiating pivotal Phase III studies for the treatment of bipolar disorder in the second half of 2019.
"We are pleased to announce the completion of enrollment in the first three Phase III trials for SPN-812," said Jack Khattar. "We look forward to reporting top-line data from these trials during December 2018. If successful, SPN-812 has the potential to be a novel non-stimulant for the treatment of ADHD that compares favorably to existing medications."

Operating Expenses
Research and development expenses in the third quarter of 2018 were $20.4 million, as compared to $13.0 million in the same quarter last year. The increase was due primarily to the initiation of the four Phase III clinical trials for SPN-812 in the second half of 2017 and, to a lesser extent, the OLE trials for SPN-812 and SPN-810.

Selling, general and administrative expenses in the third quarter of 2018 were $40.9 million, essentially unchanged compared to $40.8 million in the same quarter last year.

Operating Earnings and Earnings Per Share
Operating earnings in the third quarter of 2018 were $37.5 million, a 68.2% increase over $22.3 million in the same prior year period. The improvement in operating earnings was primarily due to increased net product sales, partially offset by increased research and development expenses.

GAAP net earnings in the third quarter of 2018 were $28.0 million, or $0.52 per diluted share, as compared to $16.0 million, or $0.29 per diluted share, in the same period last year. In addition to higher operating income, GAAP net earnings and diluted earnings per share for the third quarter of 2018 benefited from the reduction in the statutory U.S. federal income tax rate and, to a lesser extent, from stock option exercises.

Weighted-average diluted common shares outstanding were approximately 54.2 million in the third quarter of 2018, as compared to approximately 53.6 million in the third quarter of 2017.

As of September 30, 2018, the Company had $740.5 million in cash, cash equivalents, marketable securities and long term marketable securities, as compared to $273.7 million at December 31, 2017. This increase reflects net proceeds of $364.9 million from the issuance of convertible senior notes and warrants in March 2018, partially offset by purchases of convertible note hedges, as well as increased cash from operations in the nine months ended September 30, 2018.

Financial Guidance
For full year 2018, the Company is updating its prior guidance as set forth below:

Net product sales in the range of $388 million to $395 million, compared to the previously expected range of $385 million to $400 million.
Research and development expenses of approximately $95 million, including the one-time upfront expense of $15 million in the fourth quarter for the acquisition of Biscayne Neurotherapeutics, Inc.
Operating earnings in the range of $120 million to $125 million, compared to the previously expected range of $115 million to $125 million.
The Company expects an effective tax rate of approximately 23% to 25% for the fourth quarter of 2018.
Supernus ranked number one pharmaceutical company worldwide in Fortune’s "100 Fastest-Growing Companies" list for 2018 and number three in all industries
In August 2018, Fortune ranked qualifying companies based on revenue growth rate, EPS growth rate, and three-year annualized total return for the period ended June 29, 2018. In a review of Supernus and using their methodology, Fortune placed Supernus in the top spot in the pharmaceutical industry worldwide and the third spot across all industries.

To view the full list of Fortune’s 100 Fastest-Growing Companies go to: View Source

"I am so proud of our employees. They deserve all the recognition Supernus has received over the past few years, from making the Deloitte Technology Fast 500 list three years in a row to being ranked as the number one Fortune 100 fastest growing pharmaceutical company in the world," said Jack Khattar. "Their hard work and commitment to excellence and to our patients are second to none, and I am very fortunate to be working with such an incredible organization."

Conference Call Details
The Company will hold a conference call hosted by Jack Khattar, President and Chief Executive Officer, and Greg Patrick, Vice President and Chief Financial Officer, to discuss these results at 9:00 a.m. Eastern Time, on Wednesday, November 7, 2018. An accompanying webcast also will be provided.

Please refer to the information below for conference call dial-in information and webcast registration. Callers should dial in approximately 10 minutes prior to the start of the call.

Conference dial-in:
International dial-in:
Conference ID:
Conference Call Name:
(877) 288-1043
(970) 315-0267
2697616
Supernus Pharmaceuticals Third Quarter 2018 Earnings Conference Call

Following the live call, a replay will be available on the Company’s website, www.supernus.com, under "Investor Relations".

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 Oncoceutics, Inc. reported that it will host an investigator meeting and that initial efficacy data of its lead compound ONC201 in patients with H3 K27M-mutant gliomas will be presented at the 2018 Annual Meeting of the Society for Neuro-Oncology in New Orleans, on November 15th to November 18th (Press release, Oncoceutics, NOV 6, 2018, View Source [SID1234558365]).

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, along with scientific and medical collaborators, will give 10 presentations that describe several aspects of ONC201, including clinical efficacy and safety, mediated via highly specific interaction with dopamine receptor 2 (DRD2), unique molecular characteristics, and novel insights on dopamine receptor dysregulation in glioma. Moreover, presentations will include the preclinical effectiveness and mechanism of action of ONC206, another molecule from this newly established class of compounds, called imipridones. One of the highlights of the event will be an oral presentation entitled, "Integrated clinical experience with ONC201 in previously-treated H3 K27M-mutant glioma patients," which scored highly in the review process of the SNO conference and will be one of three selected abstracts to be presented by a discussant separate from the oral presentation. The data set that will be disclosed will focus on radiographic and clinical responses in H3 K27M-mutant glioma patients from several clinical programs with ONC201.

"The reported findings are very meaningful for the medical community, and we look forward to sharing our experience with ONC201 in this first group of patients," said Yazmin Odia, MD, Lead Physician of Medical Neuro-Oncology, Miami Cancer Institute. "Patients with H3 K27M-mutant gliomas have a dismal prognosis, and there is currently no known effective drug for these patients."

Additionally, Oncoceutics will hold an investigator meeting for physicians involved in ongoing and future clinical trials with ONC201.

"We are conducting a number of clinical trials to translate these compelling findings into robust clinical data sets," said Wolfgang Oster, MD, PhD, and CEO of Oncoceutics. "We are excited about the progress in our development programs and hope that our findings will make a difference for patients with unmet medical need. We are grateful to the Society for Neuro-Oncology and its members for their interest and collaboration."

More Information: Imipridone-related SNO 2018 Abstracts

ONC201 glioma clinical experience (3 abstracts)

ACTR-34 (Talk): Integrated clinical experience with ONC201 in previously-treated H3 K27M-mutant glioma patients, Chi et al, Sunday November 18th, 8:50-9:00am
ACTR-33 (Talk): Tumor Tissue Penetration and Pharmacodynamics of ONC201 in Adult Recurrent Glioblastoma Patients, Arrillaga-Romany et al, Friday November 16th, 2:40-2:45pm
PDCT-06 (Talk): Phase I clinical trial of ONC201 in pediatric H3 K27M-mutant glioma or newly diagnosed DIPG, Gardner et al, Friday November 16th, 8:22-8:26pm
DRD2 dysregulation in glioma and ONC201 preclinical efficacy (4 abstracts)

DRES-10 (Talk): DRD5 is a modulator of glioma susceptibility to DRD2 antagonism by ONC201, Prabhu et al, Sunday November 18th, 11:25-11:30am
EXTH-26 (Poster): Molecular determinant of clinical response to ONC201, an inhibitor of dopamine receptor 2 (DRD2) signaling, in glioblastoma patients, Li et al, Saturday November 17th
DDIS-12 (Poster): ONC201: The first selective, non-competitive DRD2/3 antagonist for clinical neuro-oncology, Prabhu et al, Saturday November 17th
DDIS-16 (Poster): ONC201 in combination with radiation exhibits synergistic efficacy in high grade gliomas and other advanced cancers, Tarapore et al, Saturday November 17th
Utility of imipridone scaffold in neuro-oncology (3 abstracts)

EXTH-64 (Talk): Imipridones Cause Metabolic Reprogramming and Elicit Unique Vulnerabilities in Glioblastoma, Ishida et al, Friday November 16th, 8:06-8:10pm
EXTH-17 (Talk): Selective, non-competitive DRD2/3 antagonism by imipridone ONC206 is effective in tumors with dopamine receptor dysregulation, Prabhu et al, Friday November 16th, 8:02-8:06pm
EXTH-58 (Poster): ONC206, an imipridone family member, suppresses glioblastoma cells via blocking cancer stemness pathways, Jung et al, Saturday November 17th

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.