On November 9, 2018 NewLink Genetics Corporation (NASDAQ:NLNK) reported that data from three separate trials are being presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting in Washington D.C (Press release, NewLink Genetics, NOV 9, 2018, View Source [SID1234531173]). Data from a Phase 1a clinical trial of NLG802, a prodrug of indoximod, as well as biomarker data from two Phase 2 studies of indoximod in combination therapy are being presented in poster sessions today and tomorrow, November 9th and 10th, from 8:00AM to 8:00PM ET. These posters with full data sets are provided on the company’s website in the "Posters & Presentations" section under the "Investors & Media" tab.

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"We are delighted to be able to present these data supporting both activity of indoximod within the tumor microenvironment and the higher exposure levels obtained by indoximod prodrug, NLG802, supporting the potential for these drugs to elicit therapeutic responses and improve the lives of patients with cancer," said Charles J. Link, Jr, MD, Chairman and Chief Executive Officer.

Phase 1 Clinical Trial of NLG802, Indoximod Prodrug with Enhanced Pharmacokinetic Properties
Preliminary data from a Phase 1a study of NLG802, a prodrug of indoximod, are being presented today by Olivier Rixe, MD, PhD, Professor of Medicine, University of New Mexico Comprehensive Cancer Center, Albuquerque, NM in a poster session (P331). Co-author, Eugene Kennedy, MD, Chief Medical Officer, NewLink Genetics, will be present Friday, November 9th, 12:45-2:15PM and 6:30-8:00PM, to discuss these data.

NLG802 is being evaluated in an ongoing standard 3+3 dose escalation Phase 1a study in patients with recurrent advanced solid malignancies. The purpose of this trial is to assess the safety, maximum tolerated dose, and pharmacokinetic properties of this drug candidate. As of October 3rd, the cutoff date for these data, 11 patients were enrolled in this study in three separate dose cohorts, 180 mg BID, 363 mg BID, and 726 mg BID.
Key findings from these preliminary data:

NLG802, was well tolerated, with no unexpected safety signals.

At the time of analysis, neither maximum tolerated dose (MTD), nor maximum biologically achievable dose (MBAD) had been reached.

NLG802 produced 4-fold increase in CMAX and AUC after a single dose, and a 4-5.5 fold increases in CMAX and AUC after continuous BID dosing compared with the molar equivalent of indoximod dosing.

The Immunogenic Impact of Indoximod on the Tumor Microenvironment of Patients with Advanced Melanoma
Biopsy data from a Phase 2 study of indoximod plus checkpoint inhibition from patients with advanced melanoma are being presented today by Jiayi Yu, PhD, Senior Scientist, NewLink Genetics, in a poster session (P142). The author will be present Saturday, November 10th, 12:20-1:50PM and 7:00-8:30PM to discuss these data.
In this study, patients with unresectable advanced melanoma underwent pretreatment tumor biopsy followed by a repeat biopsy after cycle 3 of indoximod plus pembrolizumab. Fourteen pairs of tumor specimens (6 patients with objective response and 8 non-responders) underwent RNA sequencing analysis and immunofluorescence staining to
assess immune activity in the tumor microenvironment (TME), to define changes in the tumor genomic profile and gene expression. Baseline samples from the trial were used for predictive biomarker assessment (N = 38).
Key findings from these biopsy data:

Compared to published studies, these biopsy data suggested indoximod exclusively contributed to immunologic and metabolic changes in the TME.

Indoximod in combination therapy may contribute to immunologic and metabolic changes in a different manner than anti-PD-1 alone.

Decreased IDO1 in Ki67- cells supports indoximod’s mechanism of action (MOA).

Patients with high IDO expression showed a trend towards both higher rate of response to treatment and longer progression-free survival (PFS), results which were independent of PD-L1 expression.

Previously published results from this Phase 2 study of indoximod plus pembrolizumab for patients with advanced melanoma may be found on the company’s website under "Posters & Publications" in the "Investors & Media" section.
Effects of indoximod plus gemcitabine/nab-paclitaxel on tumor microenvironment of patients with metastatic pancreas cancer
Biopsy data from a Phase 2 study of indoximod plus chemotherapy for patients with metastatic pancreatic cancer are being presented today by Jiayi Yu, PhD, Senior Scientist, NewLink Genetics, in a poster session (P706). Co-author, Gabriela Rossi, PhD, Vice President, Biologics Development, NewLink Genetics, will be present Saturday, November 10th, 12:20-1:50PM and 7:00-8:30PM to discuss these data.
In this study, treatment-naïve patients with metastatic pancreatic cancer underwent pre-treatment tumor biopsy with a repeat biopsy on week 8. Sixteen pairs of tumor specimens (8 patients with objective response, and 8 non-responders) underwent RNA sequencing analysis and immunohistochemistry (IHC) staining to assess immune activity in the tumor microenvironment.
Key findings from these biopsy data indicate that indoximod plus standard-of-care (SOC) chemotherapy:

Tumor samples observed to have increased recruitment of intratumoral T cells.

Increased recruitment of innate immune cells (NK cells) in responding patients.

Downregulated Treg population and IDO expression in the TME.

Increased both innate and adaptive immune responses in TME of these patients, supporting indoximod’s MOA.

Previously published results from this Phase 2 study of indoximod plus gemcitabine/nab-paclitaxel for patients with metastatic pancreatic cancer may be found on the company’s website under "Posters & Publications" in the "Investors & Media" section.
About Indoximod
Indoximod is an investigational, orally available small molecule targeting the IDO pathway. The IDO pathway is a key immuno-oncology target, suppressing immune response and allowing for immune escape by degrading tryptophan with the resultant production of kynurenine. Indoximod reverses the immunosuppressive effects of low tryptophan and high kynurenine through mechanisms that include modulation of the AhR-driven transcription of genes that control immune function. This results in increased proliferation of effector T cells, increased differentiation into helper T cells rather than regulatory T cells, and downregulation of IDO expression in dendritic cells. Indoximod is being evaluated in combination with treatment regimens including chemotherapy, radiation, checkpoint blockade and cancer vaccines across multiple indications including recurrent pediatric brain tumors, DIPG, and AML.

About NLG802

NLG802 is a prodrug of indoximod. NLG802 has been shown in preclinical trials to increase bioavailability and exposure to indoximod above the levels achievable by direct administration of indoximod. NLG802 is currently being evaluated in clinical trials.

On November 9, 2018 Exact Sciences Corp. (Nasdaq: EXAS) reported that company management will be presenting at the following investor conference during November and invited investors to participate by webcast (Press release, Exact Sciences, NOV 9, 2018, View Source [SID1234531189]).

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Jefferies 2018 London Healthcare Conference, London
Presentation and Q&A session on Thursday, Nov. 15 at 2:40 p.m. GMT, 9:40 a.m. EST
Evercore ISI HealthconX, Boston
Fireside chat on Tuesday, Nov. 27 at 12:30 p.m. EST

On November 9, 2018 Fortress Biotech, Inc. (NASDAQ: FBIO) ("Fortress"), a biopharmaceutical company dedicated to acquiring, developing and commercializing novel pharmaceutical and biotechnology products, reported financial results and recent corporate highlights for the third quarter ended September 30, 2018 (Press release, Fortress Biotech, NOV 9, 2018, View Source [SID1234531206]).

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Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, "Fortress and our subsidiaries continued to achieve important corporate and clinical milestones in the third quarter of 2018. Mustang Bio expanded its pipeline into gene therapy by securing an exclusive worldwide license for the development of a potentially first-in-class ex vivo lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency ("X-SCID") from St. Jude Children’s Research Hospital ("St. Jude"). Additionally, Checkpoint Therapeutics announced positive interim safety and efficacy data from its Phase 1/2 clinical trial of CK-101, a third-generation epidermal growth factor receptor ("EGFR") inhibitor being evaluated in advanced non-small cell lung cancer ("NSCLC"). Finally, Cyprium Therapeutics’ product candidate for patients diagnosed with classic Menkes disease, CUTX-101, was granted Fast Track Designation by the U.S. Food and Drug Administration ("FDA"). We plan to continue to acquire and develop compelling and, in some instances, potentially life-saving product candidates, which could lead to maximizing shareholder value."

Financial Results:

· As of September 30, 2018, Fortress’ consolidated cash, cash equivalents, short-term investments (certificates of deposit), cash deposits with clearing organizations and restricted cash totaled $136.1 million, compared to $168.3 million as of December 31, 2017, a decrease of $32.2 million year-to-date.
· Net revenue totaled $63.7 million for the third quarter of 2018, compared to $46.9 million for the third quarter of 2017. Total revenue as of September 30, 2018 includes $5.2 million of Fortress revenue, primarily from the sale of Journey Medical Corporation products, and $58.5 million of revenue from National Holdings Corporation1 ("National Holdings"). Total revenue as of September 30, 2017 included $2.5 million of Fortress revenue and $44.4 million of revenue from National Holdings.
· Research and development expenses were $16.1 million for the third quarter of 2018, of which $15.1 million was related to Fortress Companies. This compares to $15.9 million for the third quarter of 2017, of which $14.2 million was related to Fortress Companies. Non-cash, stock-based compensation expenses included in research and development were $1.8 million for the third quarter of 2018, compared to $1.6 million for the third quarter of 2017.
· Research and development expenses from license acquisitions were $3.7 million for the third quarter of 2018, compared to $0.3 million for the third quarter of 2017.
· General and administrative expenses were $12.2 million for the third quarter of 2018, of which $7.4 million was related to Fortress Companies. This compares to $15.1 million for the third quarter of 2017, of which $10.5 million was related to Fortress Companies. Non-cash, stock-based compensation expenses included in general and administrative expenses were $2.3 million for the third quarter of 2018, compared to $2.6 million for the third quarter of 2017.
· National Holdings’ operating expenses totaled $55.2 million for the third quarter of 2018, compared to $47.7 million for the third quarter of 2017.
· Net loss attributable to common stockholders was $16.6 million, or $0.37 per share, for the third quarter of 2018, compared to a net loss attributable to common stockholders of $27.1 million, or $0.67 per share, for the third quarter of 2017. For the first nine months of 2018, net loss attributable to common stockholders was $59.3 million or $1.36 per share, compared to $56.5 million or $1.39 per share in the first nine months of 2017.

On November 9, 2018 AVEO Oncology (NASDAQ: AVEO) reported financial results for the third quarter ended September 30, 2018 and provided a business update (Press release, AVEO, NOV 9, 2018, View Source [SID1234530993]).

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"The recent announcement of positive topline results from the Phase 3 TIVO-3 study in renal cell carcinoma is transformative for AVEO, and the next step of an ongoing, multi-year effort to ensure tivozanib (FOTIVDA) is available to patients for whom it could deliver its differentiated combination of efficacy and tolerability relative to other commercially available TKIs in RCC," said Michael Bailey, president and chief executive officer of AVEO. "We remain committed to our three-pillar strategy for tivozanib, which includes potential registration in the U.S., the ongoing commercialization of FOTIVDA in Europe with partner EUSA Pharma, and a broad exploration of tivozanib’s potential in combination with immunotherapies in cancer. Our recent achievements with TIVO-3 also provides a strong foundation to realize the potential of our pipeline, which includes two oncology-focused product candidates and three non-oncology product candidates."

Tivozanib TIVO-3 Study North America Update

Positive Topline Results from Phase 3 TIVO-3 Trial of Tivozanib in Renal Cell Carcinoma, with Goal to Submit NDA in Approximately Six Months. On Monday, November 5, 2018, AVEO announced positive topline results from the primary analysis of the TIVO-3 trial, the Company’s Phase 3 randomized, controlled, multi-center, open-label study to compare tivozanib to sorafenib in 351 subjects with refractory advanced or metastatic renal cell carcinoma (RCC). The trial met its primary endpoint of demonstrating a statistically significant benefit in progression-free survival (PFS). Patients receiving tivozanib demonstrated a 44% improvement in median PFS and a 26% reduction in risk of progression or death (hazard ratio (HR)=0.74; p=0.02). Preliminary overall survival (OS) at the time of the final PFS analysis was immature (only 46% of events reported) and showed no statistically significant difference (HR=1.06, p=0.69). 149 patients remain on treatment or in long term follow-up.

Tivozanib was generally well-tolerated, with adverse events consistent with those observed in previous tivozanib trials, including the Phase 3 TIVO-1 trial in front-line RCC. The most common adverse event in patients receiving tivozanib was hypertension, an on-target event known to reflect effective VEGF pathway inhibition and one managed by anti-hypertensive medication. Detailed results of the trial will be submitted for presentation at an upcoming major medical meeting.

Based on results from the TIVO-3 trial, together with the previously completed TIVO-1

trial of tivozanib in the first-line treatment of RCC, the Company plans to submit a potential New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) in approximately six months.

Tivozanib TiNivo Study Update

Updated Phase 2 Results from the TiNivo Trial of Tivozanib and Nivolumab (OPDIVO) in RCC Presented at the 2018 ESMO (Free ESMO Whitepaper) Annual Meeting. Updated Phase 2 data from the Phase 1b/2 TiNivo study of tivozanib in combination with nivolumab (OPDIVO, Bristol-Myers Squibb), an immune checkpoint, or PD-1, inhibitor, were presented at the 2018 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Annual Meeting in Munich. As of the date of the presentation, the Phase 1b/2 study had enrolled a total of 28 patients, and the Phase 2 portion of the study (n=22) was designed to assess the safety, tolerability, and anti-tumor activity of the full dose and schedule of oral tivozanib as established in the Phase 1b portion of the study.

The data demonstrated that the tivozanib-nivolumab combination continued to exhibit synergistic efficacy and favorable tolerability. Treatment-related Grade 3/4 adverse events occurred in 60% of patients, the most common of which was hypertension. Preliminary efficacy was assessed in all 25 patients, who were treated with the full dose and schedule of oral tivozanib in combination with intravenous nivolumab. Of these patients, 13 (52%) had received at least one prior systemic therapy, including 2 (8%) that had received prior PD-1 therapy, and 12 (48%) were treatment naïve. An overall response rate was observed in 14 patients (56%) (complete response plus partial response), including 1 patient (4%) achieving a complete response, and a disease control rate (complete response plus partial response plus stable disease) was observed in 24 patients (96%). The 2 patients (8%) who received prior PD-1 therapy both achieved a partial response. At the time of data collection, 13 patients (52%) remained on study and 18 patients (72%) had tumor shrinkage of at least 25%, with a majority of patients having disease control for at least 48 weeks.

Tivozanib (FOTIVDA) European Union Updates

Tivozanib (FOTIVDA) Launched in Sweden, the Netherlands and Scotland for the Treatment of RCC. Since the beginning of the third quarter of 2018, FOTIVDA was launched in Sweden, the Netherlands and Scotland for the first-line treatment of adult patients with RCC, following price and reimbursement approvals within each country. FOTIVDA is now available in Germany, the U.K., Austria, the Netherlands and Sweden. FOTIVDA was granted European Commission approval in August 2017 for the treatment of adult patients with RCC in the European Union plus Norway and Iceland.

Under its agreement with EUSA Pharma, licensee for tivozanib in the territories of Europe (excluding Russia, Ukraine and the Commonwealth of Independent States), Latin America (excluding Mexico), Africa and Australasia for all diseases and conditions in humans, excluding non-oncologic diseases or conditions of the eye, AVEO is entitled to double-digit royalty payments on net sales of FOTIVDA in Europe, up to $8.0 million in

milestone payments for potential reimbursement approvals for Germany, France, Italy, and Spain and a potential $20.0 million R&D reimbursement payment for access to TIVO-3 trial data in addition to other regulatory and commercial milestone payments.

Ficlatuzumab Update

Trials in Progress Poster for Phase 2 Study of Ficlatuzumab in Combination with Cetuximab in HNSCC Presented at the 2018 ESMO (Free ESMO Whitepaper) Annual Meeting. An investigator-sponsored Phase 2 trial of ficlatuzumab and cetuximab (ERBITUX), an EGFR-targeted antibody, in patients with cetuximab-resistant, metastatic head and neck squamous cell carcinoma (HNSCC) was presented as a trials in progress poster at the 2018 ESMO (Free ESMO Whitepaper) Annual Meeting. This randomized multi-center study, which is being conducted under the direction of Julie E. Bauman, MD, MPH, Professor of Medicine, Chief, Division of Hematology/Oncology, Associate Director of Translational Research, University of Arizona Cancer Center, is expected to enroll approximately 60 patients randomized to receive either ficlatuzumab alone or ficlatuzumab and cetuximab.

CAN017 (AV-203) Update

CANbridge IND for CAN017 (AV-203) Trial in Esophageal Squamous Cell Cancer Accepted in China. In August 2018, AVEO announced that the China National Drug Administration (CNDA) accepted CANbridge Life Sciences’ Investigational New Drug (IND) Application for a Phase 1b/3 clinical trial of CAN017 (AV-203), AVEO’s clinical-stage ErbB3 (HER3) inhibitory antibody candidate, in esophageal squamous cell cancer (ESCC). Under the terms of a March 2016 agreement, the acceptance of the IND triggered a $2.0 million milestone payment to AVEO from CANbridge Life Sciences. CANbridge licensed worldwide rights, excluding the United States, Canada, and Mexico, to AV-203 from AVEO, and AVEO is eligible to receive up to $40.0 million in potential additional development and regulatory milestone payments and up to $90.0 million in potential commercial milestone payments, assuming the successful achievement of specified development, regulatory and commercialization objectives.

Financial Update

Secured Additional Funding through Offering of Common Stock. In August 2018, AVEO announced an underwritten public offering of 2,500,000 shares of common stock at a price of $2.26 per share, resulting in aggregate gross proceeds of $5.7 million. In October and November 2018, the Company sold 3,781,389 shares of its common stock in at-the market transactions pursuant to its sales agreement with Leerink Partners LLC (Leerink) and received approximately $8.4 million in net proceeds.

Third Quarter 2018 Financial Highlights

AVEO ended Q3 2018 with $20.4 million in cash, cash equivalents and marketable securities as compared with $33.5 million at December 31, 2017.

Total revenue for Q3 2018 was approximately $2.5 million compared with $4.6 million for Q3 2017.

Research and development expense for Q3 2018 was $5.2 million compared with $4.7 million for Q3 2017.

General and administrative expense for Q3 2018 was $2.7 million compared with $2.1 million for Q3 2017.

Net loss for Q3 2018 was $22.2 million, or a loss of $0.18 per basic and diluted share, compared with net loss of $26.4 million for Q3 2017, or a loss of $0.22 per basic and diluted share. Approximately $16.2 million of Q3 2018 net loss was a non-cash loss attributable to the increase in the fair value of the 2016 private placement warrant liability that principally resulted from the increase in the stock price that occurred within the quarter. In Q3 2017, the non-cash loss attributable to the increase in the fair value of such warrant liability was $23.5 million.

Financial Guidance

AVEO believes that its $20.4 million in cash, cash equivalents, and marketable securities at the end of Q3 2018 and the additional $8.4 million raised from sales under its sales agreement with Leerink in October and November 2018 would allow it to fund planned operations into the second quarter of 2019. This estimate assumes no receipt of additional milestones from AVEO’s partners, no additional funding from new partnership agreements, no additional equity or debt financings, and no sales of equity through the exercise of outstanding warrants issued in connection with the 2016 private placement or outstanding warrants issued in connection with the recent settlement of the securities class action litigation.

On November 9, 2018 BioXcel Therapeutics, Inc. ("BTI") (Nasdaq: BTAI), a clinical stage biopharmaceutical development company utilizing novel artificial intelligence approaches to identify the next wave of medicines across neuroscience and immuno-oncology, reported quarterly results for the third quarter ended September 30, 2018 and provided an update on key strategic and operational initiatives (Press release, BioXcel Therapeutics, NOV 9, 2018, View Source [SID1234531025]).

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During the third quarter of 2018, the Company made several advances in the development of its two lead clinical programs, BXCL501, a proprietary sublingual thin film formulation of dexmedetomidine (Dex), and BXCL701, an orally-available systemic innate-immune activator.

Third Quarter 2018 and Recent Highlights:

(BXCL501)-Neuroscience Program-

A first-in-human pharmacokinetic (bioavailability) and safety study for the sublingual thin film formulation of Dex is expected to be initiated by the end of 2018 following approval of the investigational new drug (IND) application;

Received valuable feedback and guidance on further development of BXCL501 during a pre-investigational new drug meeting with FDA;

Appointed a clinical research organization (CRO) to support the company in conducting and managing clinical studies;

Completed manufacturing of Company’s proprietary sublingual thin film formulation of Dex, and the drug is available for clinical studies;

Data readout from the pharmacokinetic and safety study of BXCL501 is expected in the first half of 2019;

Expect data readouts from intravenous (IV) Dex studies supporting BXCL501 development in acute agitation in patients with schizophrenia and senile dementia of the Alzheimer’s type (SDAT) in coming weeks;

Established an industry leading neuro advisory board to support global development of BXCL501 and emerging neuroscience programs.

(BXCL701)-Immuno-Oncology Program-

Received FDA acceptance of IND application for Phase 1b/2 clinical study to evaluate BXCL701 in combination with pembrolizumab (Keytruda) in treatment emergent neuroendocrine prostate cancer (tNEPC); trial initiation is expected in fourth quarter of 2018;

Completed manufacturing of BXCL701 drug product, available for clinical studies;

Selected a leading CRO to support the Company in conducting and managing clinical studies;

Data from the pharmacokinetic, safety and efficacy study of BXCL701 in tNEPC expected to be available throughout 2019;

Entered a clinical immuno-oncology (IO) partnership with Nektar Therapeutics to develop combination of BXCL701, Nektar Therapeutics’ NKTR‑214 and a checkpoint inhibitor as a potential treatment for pancreatic cancer; companies will be sharing the cost of the trial;

Established an industry leading IO clinical advisory board to support global development of BXCL701 and emerging programs.

Emerging Programs-

Continued the use of the artificial intelligence platform to select and prioritize additional pipeline opportunities to augment the current neuroscience and IO portfolio.

Business & Operations-

Strengthened management team with drug development experts including:

Chetan Lathia, Ph.D. as Senior Vice President and Head of Translational Medicine, Clinical Pharmacology and Regulatory Affairs, to support advancement of the pipeline programs.

David Hanley, Ph.D. as Vice President and Head of Global Pharmaceutical Development and Operations, to lead the pharmaceutical development activities and operational efforts.

Vimal Mehta, Ph.D., President and Chief Executive Officer of BTI, commented, "We have made tremendous progress during the past quarter that we believe is truly transformational for BTI. We are well positioned to execute on our clinical programs across our two primary areas of focus."

"In September, we received FDA IND clearance for our first-in-human Phase 1b/2 trial to evaluate BXCL701 in combination with Keytruda as a potential therapy for tNEPC. We anticipate initiating this clinical study prior to year-end. In addition, we expanded our research collaboration with Nektar Therapeutics into a new clinical partnership for further development of the triple combination of BXCL701, Nektar Therapeutics’ NKTR‑214 and a checkpoint inhibitor. Today, we are presenting encouraging preclinical data on the triple combination across multiple tumor models at the 2018 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting. This study demonstrated robust anti-tumor activity resulting in complete tumor regression in certain in vivo models and generation of functional immunological memory, supporting the triple combination as a potential therapy for pancreatic cancer and other tumors."

"Following our productive interactions with the FDA on BXCL501, we plan to initiate our first-in-human pharmacokinetic (bioavailability) and safety trial once our IND is approved. The aim of this trial is to define the optimal dose of BXCL501 and collect sufficient clinical data to proceed to a Phase 3 registration trial in 2019. We have previously reported positive data from our trial of IV Dex, which were essential in determining the ideal dosing strategy. We now expect that data from the IV Dex studies in patients with schizophrenia and SDAT will be available by the end of 2018."

"We are de-risking clinical development of our lead candidates through patient selection optimization, translational research and predictive biomarker discovery utilizing artificial intelligence approaches. Further, we continue to identify additional opportunities for BXCL501 and BXCL701 and plan to pursue their development. We anticipate filing both INDs and clinical trial applications for additional indications across multiple locations, in an effort to establish our global footprint and leverage the significant value of our lead clinical programs."

Dr. Mehta concluded, "We remain firmly committed to our goal of providing patients with transformative therapies while also creating value for our shareholders. We are very excited by

what the future holds for our company and look forward to delivering on the anticipated milestones."

Third Quarter 2018 Financial Results

BTI reported a net loss of $4.9 million for the third quarter of 2018, compared to a net loss of $0.9 million for the same period in 2017.

Research and development expenses were $3.8 million for the third quarter of 2018, as compared to $0.6 million for the same period in 2017. The increase was primarily due to an expansion of research and development activities, including increased personnel costs, professional fees, clinical trials, and manufacturing costs associated with BTI’s two lead drug candidates.

General and administrative expenses were $1.3 million for the third quarter of 2018, as compared to $0.3 million for the same period in 2017. The increase was primarily due to additional payroll and payroll-related expenses, professional fees and costs associated with operating as a public company.

As of September 30, 2018, cash and cash equivalents totaled $47.1 million.

Upcoming investor conferences:

·

Jefferies Global Healthcare Conference, November 14‑15, 2018, London

·

2018 Prescriptions for Success Healthcare Conference, December 12, 2018, New York

·

Investor access event at the J.P. Morgan Healthcare Conference, January 7‑10, 2019, San Francisco

About BXCL501:

BXCL501 is a first in class, sublingual film of dexmedetomidine, a selective alpha 2a receptor agonist for the treatment of acute agitation. BTI believes that BXCL501 directly targets a causal agitation mechanism and has demonstrated anti-agitation effects in preclinical and clinical studies. It has a well-established regulatory and reimbursement path in schizophrenia and bipolar disorder, as demonstrated by a previously-approved drug, Adasuve.

About BXCL701:

BXCL701 is a first in class oral immunotherapy with dual mechanisms of action, with an established safety profile from 700 healthy subjects and cancer patients. Designed to stimulate both the innate and acquired immune systems, BXCL701 works by inhibiting dipeptidyl peptidase (DPP) 8/9 and blocking immune evasion by targeting fibroblast activation protein (FAP). Preclinical combination data evaluating BXCL701, a checkpoint inhibitor and other IO agents has demonstrated encouraging anti-tumor activity in multiple tumor types and formation of functional immunological memory. It is under development for tNEPC and pancreatic cancer.