On November 10, 2018 Celldex Therapeutics, Inc. (NASDAQ:CLDX) reported data from the Company’s MerTK antibody program in a poster session at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 33rd Annual Meeting(Press release, Celldex Therapeutics, NOV 10, 2018, View Source [SID1234531099]). MerTK is emerging as a promising target for cancer immunotherapy. Its expression in innate immune cells is believed to negatively regulate immune responses and genetic removal of MerTK renders mice resistant to some tumors.

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"MerTK has been described as an immune checkpoint in macrophages, dendritic cells and other immune cells," said Tibor Keler, Ph.D., Executive Vice President and Chief Scientific Officer of Celldex Therapeutics. "Through our significant discovery effort, we have identified two unique antibodies that modulate this pathway resulting in profound levels of cytokine and chemokine production, and importantly we have developed preclinical models that support the premise that antibody modulation of MerTK can lead to antitumor responses."

As detailed in the presentation, Celldex developed a large panel of antibodies to MerTK and investigated their ability to enhance activation of innate immune cells. Two lead candidate human anti-MerTK antibodies were then selected based on their potent induction of cytokines from human macrophages, dendritic cells, and monocytes. Treatment of dendritic cells with the MerTK antibodies led to production of a broad array of pro-inflammatory cytokines and chemokines. Isolated peripheral blood monocytes were found to express high levels of MerTK and were similarly activated by the MerTK antibodies.

Emerging proof of concept data was established in preclinical models. Using a surrogate anti-mouse MerTK​ antibody, similar increases in the levels of cytokines were observed in the blood of mice shortly after treatment with the antibody. The anti-mouse MerTK​ antibody led to increased survival when dosed alone or in combination with a PD-1 inhibitor in a colon cancer model. To test the lead clinical candidates, which bind to human and not mouse MerTK, Celldex generated human MerTK transgenic mice that were shown to appropriately express and regulate human MerTK on macrophages. This will now allow testing of the anti-human MerTK mAbs in inflammation and tumor models. Collectively, the data support that anti-MerTK mAbs can modulate MerTK activity consistent with its role as a negative immune regulator and provide an exciting new approach to enhance innate immune function in cancer.

Celldex is currently completing the preclinical studies for selection of the lead candidate to advance into development activities. These studies include investigating the antitumor effect of combinations with Celldex’s immunotherapy product candidates.

On November 10, 2018 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported that data to be presented today at the 33rd Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) demonstrated preliminary evidence that IPI-549 in combination with Opdivo (nivolumab) is clinically active in indications not expected to respond to Opdivo alone (Press release, Infinity Pharmaceuticals, NOV 10, 2018, View Source [SID1234531169]). In particular, evidence of reversal of resistance to Opdivo included a partial response in a patient with metastatic melanoma who progressed on immediate prior Opdivo therapy. IPI-549 plus Opdivo also resulted in a 26% reduction of tumor target lesions in a patient with chemotherapy-resistant triple negative breast cancer (TNBC), a tumor type intrinsically resistant to checkpoint inhibition. The data also included long-term follow up on sustained partial responses in two patients from combination dose escalation: one with microsatellite stable (MSS) gallbladder cancer and one with adrenocortical carcinoma. The observed early clinical activity from the combination expansion, in addition to findings from the monotherapy cohorts, including associated translational data, support on-mechanism proof of concept for IPI-549. The late-breaking abstract describing these findings will be presented today in a poster presentation at the SITC (Free SITC Whitepaper)’s 33rd Annual Meeting taking place in Washington, D.C., November 7 – 11, 2018.

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"There continues to be a significant need for improvement in the treatment of patients with advanced solid tumors who do not respond to checkpoint inhibitors alone. IPI-549’s potential to improve upon the existing CPIs makes it an important first-in-class drug in development," said David Hong, M.D. Deputy Chair, Department of Investigational Cancer Therapeutics, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX. "The reversal of resistance to checkpoint inhibition is particularly encouraging for the further development of IPI-549, and I look forward to my continued participation in the development of IPI-549."

Infinity is evaluating IPI-549 in MARIO-1, a Phase 1b study in approximately 200 patients with advanced solid tumors. Additionally, Infinity is planning to initiate MARIO-275, a global, randomized Phase 2 study to evaluate the effect of adding IPI-549 to Opdivo in approximately 150 checkpoint-inhibitor naïve advanced urothelial cancer patients who have progressed or recurred following treatment with platinum-based chemotherapy.

"The rigorous design of our Phase 1b study sets a high bar for the determination of proof of concept, given that the goals of our study are to see clinical activity both in settings where patients are not expected to respond to Opdivo alone, and in indications where there are high levels of immunosuppressive macrophages," said Dr. Sam Agresta, Chief Medical Officer of Infinity Pharmaceuticals. "We do this with the intention of re-sensitizing patients to Opdivo to overcome the limiting effects of macrophages to checkpoint inhibitor therapy, and we are excited that the data we shared today demonstrate clinical activity in this patient population. We look forward to the maturation of MARIO-1 and the advancement of the IPI-549 clinical development program in the checkpoint-inhibitor naïve setting with MARIO-275."

"Today’s data validate our scientific rationale for the development of IPI-549 as a potentially first-in-class therapeutic alternative for patients lacking better treatment options," said Adelene Perkins, Chief Executive Officer of Infinity Pharmaceuticals. "We will be expanding our melanoma cohort, with patients refractory to immediate prior anti-PD1 therapy, to 40 patients based on promising early signals and have triggered expansion of the TNBC cohort. We will also be initiating our Phase 2 MARIO‑275 trial in urothelial cancer with an emphasis on patients with high myeloid derived suppressor cell levels, given that MDSCs further promote immunosuppression. We look forward to providing an update on these efforts."

Details of Today’s Late-Breaking Presentation

Infinity will present a poster entitled "The first clinical/translational data from the expansion cohorts of a Ph1/1b Study of IPI-549, a tumor macrophage-reprogramming small molecule, in combination with nivolumab in advanced solid tumors" (Poster P716). The data reported today from an October 14, 2018 data cutoff included 82 patients treated with IPI-549 at 40 mg QD and Opdivo at 240mg IV once every two weeks.

Summary of Data

Combination expansion data demonstrated that IPI-549 in combination with Opdivo is well tolerated and is associated with a favorable safety profile. Among 82 patients evaluable for safety, the majority of side effects reported were Grade 1 or Grade 2, with 3 (4%) patients discontinuing the study due to treatment-related toxicities. There were no treatment-related deaths. These 82 patients have been treated in seven distinct cohorts: non-small cell lung cancer (n=8), melanoma (n=15), head and neck cancer (n=12), triple negative breast cancer (n=17), adrenocortical cancer (n=5), mesothelioma (n=11), and a biomarker defined baseline MDSC high cohort (n=14). The majority of the study population is 4th line and resistant to anti-PD1/PDL1 therapy. Among the 44 patients evaluable for activity (approximately one-third of the planned 129 patients in the combination expansion cohorts), 15 patients showed a best response of stable disease or better, including one partial response in an advanced melanoma patient who progressed on immediate prior Opdivo therapy. In addition, a patient with chemotherapy-resistant triple negative breast cancer showed 26% reduction in tumor target lesions at the first assessment. Reductions in elevated baseline levels of MDSCs as well as corresponding increases in the proliferative fraction of previously exhausted memory cytotoxic T-cells were seen in these patients. Another partial response was observed in a patient with advanced mesothelioma. Twenty-five patients remain on study and were not evaluable for activity as of the data cutoff. The data included long-term follow up on additional partial responses in two patients from the combination dose escalation: one with microsatellite stable gallbladder cancer and one with adrenocortical carcinoma and these two partial responses were maintained for over 12 and 17 months, respectively. These patients also demonstrated sustained inhibition of MDSCs during the period in which the partial response was maintained. Enrollment is ongoing.

Infinity Investor/Analyst Reception and Webcast
In conjunction with SITC (Free SITC Whitepaper)’s 33rd Annual Meeting, Infinity will host a reception and webcast for investors and analysts today, November 10, 2018, beginning at 6:30 a.m. ET to discuss the clinical development of IPI-549, including a review of data from the Phase 1b clinical study.

The event will feature David Hong, M.D., Deputy Chair, Department of Investigational Cancer Therapeutics, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX.

The webcast and accompanying slides can be accessed in the "investors/media" section of the company’s website, www.infi.com. A replay of the event will also be available.

About IPI-549 and the Ongoing MARIO-1 Phase 1/1b Study
IPI-549 is an investigational first-in-class, oral, immuno-oncology product candidate targeting tumor-associated myeloid cells through selective phosphoinositide-3-kinase-gamma (PI3K-gamma) inhibition, thereby reducing pro-tumor macrophage function and increasing anti-tumor macrophage function. In preclinical studies, IPI-549 demonstrated the ability to reprogram macrophages from a pro-tumor (M2), immune suppressive function, to an anti-tumor (M1) immune activating function and enhance the activity of, and overcome resistance to, checkpoint inhibitors.i ii As such, IPI-549 may have the potential to treat a broad range of solid tumors and represents a potentially additive or synergistic approach to restoring anti-tumor immunity in combination with other immunotherapies such as checkpoint inhibitors.

The ongoing Phase 1/1b study being conducted by Infinity is designed to evaluate the safety, tolerability, activity, pharmacokinetics and pharmacodynamics of IPI-549 as a monotherapy and in combination with nivolumab (Opdivo) in approximately 200 patients with advanced solid tumors.iii The study includes monotherapy and combination dose-escalation components, in addition to monotherapy expansion and combination expansion components. The monotherapy dose-escalation and expansion components are complete. The combination dose-escalation component is also complete, and combination expansion cohorts are enrolling.

The combination expansion component of the study includes multiple cohorts designed to evaluate IPI-549 in patients with specific types of cancer, including patients with non-small cell lung cancer, melanoma and head and neck cancer whose tumors show initial resistance or initially respond to but subsequently develop resistance to immune checkpoint blockade therapy. The combination expansion component also includes a cohort of patients with triple negative breast cancer who have not been previously treated with immune checkpoint blockade therapy, a cohort of patients with mesothelioma, a cohort of patients with adrenocortical carcinoma and a cohort of patients with high baseline blood levels of MDSCs.

About the Planned MARIO-275 Phase 2 Study
Infinity is planning to conduct MARIO-275: MAcrophage Reprogramming in Immuno-Oncology, a global, randomized Phase 2 study to evaluate the effect of adding IPI-549 to Opdivo in checkpoint-naïve advanced urothelial cancer patients who have progressed or recurred following treatment with platinum-based chemotherapy. Approximately 150 patients will be randomized between combination therapy and Opdivo monotherapy. The primary endpoint of the trial will be overall response rate, which will be assessed in the overall population as well as in subsets of patients with different baseline levels of myeloid derived suppressor cells (MDSCs). Opdivo is approved for use by the FDA as a single agent in patients with locally advanced or metastatic urothelial cancer who have progressed or recurred following treatment with platinum-based chemotherapy or who have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. In exploratory analyses of the CheckMate-275 data, high levels of MDSCs were associated with shorter overall survival in patients treated with Opdivo2. In Infinity’s MARIO-1study, MDSCs were reduced in the majority of patients treated with IPI-549 monotherapy.3 IPI-549 in combination with Opdivo has been administered to over 80 patients and demonstrated early evidence of clinical activity with translational studies demonstrating evidence of on-mechanism IPI-549-mediated effects.4

IPI-549 is an investigational compound, and its safety and efficacy has not been evaluated by the U.S. Food and Drug Administration or any other health authority.

On November 10, 2018 Dendrogenix a company developing a innovative therapies, reported a first funding round of €3.6M, including €1.2M in capital and €2.4M in grants and recoverable advances from walloon region (Press release, Dendrogenix, NOV 10, 2018, View Source [SID1234539438]).

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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.