On November 12, 2020 VolitionRx Limited (NYSE AMERICAN: VNRX) ("Volition") reported financial results and a business update for the third quarter ended September 30, 2020 (Press release, VolitionRX, NOV 12, 2020, View Source [SID1234570794]). Volition management will host a conference call tomorrow, November 13 at 8:30 a.m. U.S. Eastern Time to discuss these results. Conference call details may be found below.

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Cameron Reynolds, President and Chief Executive Officer of Volition commented: "During the third quarter, despite the persistence of the COVID-19 pandemic, we have made significant progress on many fronts and are on track to launch our first product, the Nu.Q Vet Cancer Screening Test, on November 30. This is a pivotal moment for Volition, demonstrating that our platform has the reliability and reproducibility to launch in an independent laboratory."

Mr. Reynolds added, "We have also made considerable progress in our human cancer program and, in particular, in blood cancer where we have obtained similar results in both humans and dogs using the same assay. Based on these promising results, we have engaged Diagnostic Oncology CRO LLC as a contract research organization to conduct a U.S. clinical trial for Non-Hodgkin’s Lymphoma. I am proud of the way our team has adapted to the different world we find ourselves in and has kept working at full speed. It is their efforts and tenacity that have made possible these milestones and the many others that we have achieved this quarter and year to date."

Company Highlights

Financial

Cash and cash equivalents as of September 30, 2020 totalled approximately $21 million compared with $17 million as of December 31, 2019.
We continue to manage our expenditures carefully, and as we approach launch and commercialization our burn rate is approximately $1.6 – $1.7 million per month.
Nu.Q Vet Cancer Screening Test Commercial Launch

Launch date of Nu.Q Vet Cancer Screening Test planned for November 30, 2020.
This test will initially be positioned for use in the annual health check of older dogs (those that are seven years and older) and for cases where there is a high suspicion of cancer.
The test will be available from the GI Lab at Texas A&M University to potentially thousands of veterinarians across Texas and the rest of the U.S.
Significant potential market opportunity into the millions of tests per year as there are approximately 77 million dogs in the U.S.
Revenue to Volition is expected to be $45 per test and generate a greater than 85% gross margin.
Pre-launch marketing efforts are underway, including a report entitled "A Look to the Future of Cancer Diagnostics" which compiles contributions from some of the key opinion leaders in the veterinary oncology space. A downloadable copy is available here.
Nu.Q Vet Cancer Screening Test: Clinical Data

The Nu.Q Vet Cancer Screening Test is a simple, low-cost, easy to use ELISA based screening blood test which will help streamline the screening process for up to 1/3 of cancers in dogs including common malignancies such as lymphoma and hemangiosarcoma.
Two abstracts were presented at the Veterinary Cancer Society Virtual Annual Conference in October 2020.
In a study of over 330 dogs conducted by Texas A&M University, the Nu.QTM Vet Cancer Screening Test gave good clinical discrimination with an AUC of 87.3% for lymphoma and 97.6% for hemangiosarcoma.
At 100% Specificity the Nu.Q Vet Cancer Screening Test demonstrated detection rates of 74% of lymphoma and 89% of hemangiosarcoma.
Clinical – New US Regulatory Study

Engaged Diagnostic Oncology CRO LLC, the largest U.S. Contract Research Organization specializing in oncology purposed in-vitro diagnostic device clinical trials, to conduct a U.S. clinical trial for Non-Hodgkin’s Lymphoma (NHL).
The trial is designed to obtain multiple FDA-approved adjunct tests to aid in the diagnosis of the five most common and aggressive forms of NHL.
The trial will enroll up to 1,500 subjects across 10 major U.S. healthcare institutions over 22 months.
This extensive program will cost approximately $2.9 million over two years assuming the completion of numerous projects and includes not only the clinical study but also data analysis and regulatory and reimbursement submission preparation.
Existing data suggests Nu.Q technology will greatly aid physicians in distinguishing NHL from common conditions, fulfilling what we feel is a critical unmet clinical need which represents a major market opportunity.
We expect our first FDA 510K submission will be possible approximately 10-12 months into the trial.
Clinical – NETosis including COVID-19

We have made great progress on the research program for the use of our Nu.Q technology in NETosis and in particular in monitoring disease progression of COVID-19.
Several studies have either been collected or are being negotiated in Europe and we anticipate the next results will be reported before the end of this year.
We are also negotiating a large FDA trial for use of our assays in neutrophil extracellular traps (NETs) for COVID-19 and influenza in the U.S. and will announce the full details once they have been finalized.
We have filed a novel patent for this application and plan to utilise results of these trials and other ongoing studies to further our aim of developing a clinically useful product to help in the battle against the COVID-19 pandemic and potentially other diseases such as influenza and sepsis.
Expansion

We are in the final paperwork stages for "Silver One" the production hub for our products and components close to our Lab in Belgium.
Facility will be the production hub of all of our products and components, to both secure our own supply at a lower cost, and to drive reagent revenue, building on our purchase of Octamer GmbH (now called Volition Germany) earlier this year.
We plan to achieve full ISO certification next year.
Our plan is to produce, at large scale, raw materials such as recombinant nucleosomes, which act as the calibrant to our Nu.Q assays, in addition to antibodies that are key elements to our branded products. We plan to manufacture our full diagnostic kits once finalized.
We expect to offer all elements, including a service lab provision, for both commercial sale and for clinical trial purposes and CE-marked products for Europe and beyond.
To drive revenue, we have appointed our first Sales Manager who starts in December.
We have also opened a small, shared laboratory at California State University in San Marcos, California where we will focus on blue-sky innovation and discovery research.
Assay Development and Clinical Studies (Cancer)

In various ways our "Marquee trials" have now been affected by the continued pandemic either by slower or paused collection, or a host of other supply chain or travel and communication issues. We believe we have successfully managed those areas under our direct control (such as assay development and running samples – both on track with our milestones) but many issues are not within our control.
We have now successfully completed 12 Nu.Q discovery grade assays on subsets of both of our National Taiwan University studies – colorectal and lung cancers – and are working on data analysis.
An abstract has been accepted regarding Nu.Q performance in lung cancer detection for presentation at the upcoming IASLC conference in January 2021. We expect to submit the colorectal cancer data to upcoming conferences.
Upcoming Milestones

Volition expects to achieve the following milestones during 2020 and beyond:

Launch of the Nu.Q Vet Cancer Screening Test in the U.S.
Complete the purchase and fit out of "Silver One", to serve as our manufacturing hub and service lab in Belgium.
Focus on driving revenue in the coming quarters, where possible during the pandemic, in four key areas:
– Nu.Q Vet products
– Disease monitoring tests (e.g. COVID-19)
– Reagent sales
– Licensing of our technology for others to commercialize.

Continue to advance our previously announced large-scale colorectal and lung cancer trials in Europe, Asia and the U.S.
Publish several abstracts and peer-reviewed scientific papers with clinical results as well as showing the robustness and utility of our Nu.Q platform.
Advance the development of Nu.Q Capture.
Announce patient data demonstrating the wide utility of our epigenetic toolbox.
Continue to file patents to expand and extend our Intellectual Patent portfolio.
VolitionRx Limited Third Quarter 2020 Earnings and Business Update Conference Call

Cameron Reynolds, President and Chief Executive Officer of Volition, will host the call along with David Vanston, Chief Financial Officer and Scott Powell, Executive Vice President, Investor Relations.

A live audio webcast of the conference call will also be available on the investor relations page of Volition’s corporate website at View Source

In addition, a telephone replay of the call will be available until November 27, 2020. The replay dial-in numbers are 1-844-512-2921 (toll-free) in the U.S. and Canada and 1-412-317-6671 (toll) internationally. Please use replay pin number 13713126.

On November 12, 2020 Relay Therapeutics, Inc. (Nasdaq: RLAY), a clinical-stage precision medicine company transforming the drug discovery process by leveraging unparalleled insights into protein motion, reported third quarter 2020 financial results (Press release, Relay Therapeutics, NOV 12, 2020, View Source [SID1234570793]).

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"In our first quarter as a public company, I am proud of our team’s unwavering focus on bringing our medicines to patients," said Sanjiv Patel, M.D., president and chief executive officer of Relay Therapeutics. "In July, we completed and closed our initial public offering. In September, we dosed the first patient with our second targeted medicine, RLY-4008, the only selective small molecule inhibitor of FGFR2 in clinical development. We look forward to providing updates across our pipeline in 2021."

Second Quarter Financial Highlights

Cash and Cash Equivalents: Cash, cash equivalents and investments totaled approximately $713 million as of September 30, 2020, which includes the net proceeds of $425 million from the Company’s initial public offering, compared to $356 million as of December 31, 2019. The Company expects its current cash and cash equivalents will be sufficient to fund its current operating plan into 2023.

R&D Expenses: Research and development expenses were $24 million for the third quarter of 2020, as compared to $18 million for the third quarter of 2019. This increase was primarily due to higher personnel costs, including an increase of $4.6 million in non-cash stock-based compensation expenses related to the accounting treatment of certain options granted in the first quarter of 2020, as well as an increase in costs to support our clinical trials, offset in part by a decrease in outside and consulting services for our pre-clinical candidates.

G&A Expenses: General and administrative expenses were $12 million for the third quarter of 2020, as compared to $4 million for the third quarter of 2019. This increase was primarily due to higher personnel costs, including an increase of $6.0 million in non-cash stock-based compensation expenses related to the accounting treatment of certain options granted in the first quarter of 2020, to support our infrastructure.

Net Loss: Net loss was $36 million for the third quarter of 2020, or a net loss per share of $3.00, as compared to a net loss of $20 million for the third quarter of 2019, or a net loss per share of $5.53.

On November 12, 2020 Brickell Biotech, Inc. ("Brickell" or the "Company") (Nasdaq: BBI), a clinical-stage pharmaceutical company focused on developing innovative and differentiated prescription therapeutics for the treatment of debilitating skin diseases, reported financial results for the third quarter ended September 30, 2020 and provided a corporate update (Press release, Vical, NOV 12, 2020, View Source [SID1234570792]).

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"I am proud of the focus, drive and determination that the Brickell team has shown in 2020, which has allowed us to execute against our corporate goals. As a result, we are now well-positioned to continue executing our strategy to develop sofpironium bromide as a potentially best-in-class treatment option for the more than 10 million people in the U.S. suffering with primary axillary hyperhidrosis," commented Robert Brown, Chief Executive Officer of Brickell. "Last month, we initiated the Cardigan I registration trial and completed an equity financing, which provided us with additional capital needed to initiate the Cardigan II second registration trial later this year and is expected to allow us to complete the U.S. pivotal Phase 3 program, with topline results anticipated in the fourth quarter of 2021."

"The third quarter was a productive period for our Japanese development partner, Kaken, who recently received regulatory approval for ECCLOCK (sofpironium bromide gel, 5%) in Japan for the treatment of primary axillary hyperhidrosis. Japan is the first country to approve sofpironium bromide for any indication and Kaken remains on target for commercial launch later this year. We are thrilled with the progress that both Brickell and Kaken have made and look forward to providing updates on the advancement of our U.S. pivotal Phase 3 program, as well as Kaken’s launch efforts in Japan, over the coming months," concluded Mr. Brown.

Business and Recent Developments

Completed an equity financing in October 2020 resulting in net proceeds of approximately $13.7 million that strengthened the Company’s balance sheet and is expected to fully fund its operations through topline results of the U.S. pivotal Phase 3 program.

Initiated the U.S. Phase 3 Cardigan I clinical trial, a multicenter, randomized, double-blinded, vehicle (placebo)-controlled study to evaluate the safety and efficacy of topically applied sofpironium bromide gel, 15% for the treatment of primary axillary (underarm) hyperhidrosis. Subjects will apply sofpironium bromide or vehicle once daily at bedtime to their underarms for six consecutive weeks, with a two-week post-treatment follow-up. The study is expected to enroll up to 350 subjects aged nine years and older with primary axillary hyperhidrosis.

Japanese development partner, Kaken Pharmaceutical, Co., Ltd. ("Kaken"), received regulatory approval in Japan to manufacture and market sofpironium bromide gel, 5% for the treatment of primary axillary hyperhidrosis. This approval was based on the results of Kaken’s Japanese pivotal Phase 3 registration study of sofpironium bromide gel, 5% in 281 patients with primary axillary hyperhidrosis, in which all primary and secondary efficacy endpoints demonstrated statistically significant differences between sofpironium bromide gel and vehicle. In addition, sofpironium bromide gel, 5% was observed to be safe and generally well tolerated in this study, as well as in the accompanying 52-week long-term safety extension study with 185 patients in Japan.

Kaken and Brickell were granted by the Japanese Patent Office a composition of matter patent with claims directed to the novel polymorphic, or crystalline, forms of sofpironium bromide that is expected to provide additional protection for these newly developed and distinct forms in Japan through 2040.

Completed the 12-month Phase 3 open-label long-term safety study evaluating sofpironium bromide gel, 5% and 15% in 300 subjects nine years and older with primary axillary hyperhidrosis. The study results confirmed that sofpironium bromide gel, at both concentrations, was safe and generally well tolerated, which was consistent with the earlier Phase 2 clinical trial results. No treatment-related serious adverse events were observed. The Company expects to release additional details at an upcoming scientific forum.

Entered into a collaboration agreement with AnGes, Inc ("AnGes") through which the Company has certain rights to develop AnGes’ proprietary investigational adjuvanted plasmid DNA vaccine intended to prevent SARS-CoV-2 (COVID-19) in the U.S., South America and certain other emerging markets. AnGes is currently conducting Phase 1/2 clinical studies with its vaccine candidate in Japan.
Upcoming Milestones

Plan to initiate the Cardigan II study, the second U.S. pivotal Phase 3 clinical trial, prior to year end. The Cardigan II study will evaluate the safety and efficacy of sofpironium bromide gel, 15% versus vehicle in approximately 350 subjects aged nine years and older with primary axillary hyperhidrosis.

Expect Kaken to launch ECCLOCK commercially in Japan by the end of 2020. Under our agreement with Kaken, Brickell is entitled to receive commercial milestone payments, as well as tiered royalties based on a percentage of net sales in Japan.

Expect to report topline data from both the Cardigan I and II U.S. pivotal Phase 3 clinical studies by the end of 2021.

Expect to receive results from the Phase 1/2 clinical studies with AnGes’ COVID-19 vaccine candidate in Japan through the first quarter of 2021. The results from these studies will guide AnGes’ and Brickell’s global development efforts of this novel vaccine candidate.
Financial Results

The Company reported cash and cash equivalents and marketable securities of $20.2 million as of September 30, 2020 compared to $11.7 million as of December 31, 2019. In addition, Brickell has prepaid $4.2 million to third-party clinical research organizations as part of conducting the U.S. pivotal Phase 3 clinical trials of sofpironium bromide. Subsequent to the end of the third quarter, the Company completed a public equity offering resulting in net proceeds of approximately $13.7 million.

Revenue was $0.1 million for the third quarter of 2020 compared to $1.2 million for the third quarter of 2019. Revenue in both periods was driven by research and development activities related to the agreement with Kaken pursuant to which Kaken provided research and development funding to Brickell. The decrease in revenue recognized was attributable to Brickell’s Phase 3 open-label long-term safety study of sofpironium bromide gel and other ancillary clinical studies that were ongoing in 2019 but were concluded or winding down by the end of the first quarter of 2020. Conducting these studies is the basis for revenue recognition of a $15.6 million research and development payment received from Kaken in the second quarter of 2018.

Research and development expenses were $1.3 million for the third quarter of 2020 compared to $3.3 million for the third quarter of 2019. This decrease was primarily due to reduced clinical and other related regulatory and compliance costs of the Phase 3 open-label long-term safety study of sofpironium bromide gel and other ancillary clinical studies that were concluded or winding down by the end of the first quarter of 2020.

General and administrative expenses were $3.2 million for the third quarter of 2020 compared to $3.9 million for the third quarter of 2019. This decrease was primarily due to lower costs of $1.1 million for professional-related fees associated with the merger with Vical Incorporated that occurred in the third quarter of 2019 and $0.4 million for other miscellaneous fees, partially offset by higher costs of $0.6 million for stock and other compensation expense that was driven by increased headcount and $0.2 million for directors’ and officers’ liability insurance due to becoming a public company.

Brickell’s net loss was $4.3 million for the third quarter of 2020 compared to $4.8 million for the third quarter of 2019.

Conference Call and Webcast Information

Brickell’s management will host a conference call today at 4:30 p.m. ET to discuss the financial results and recent corporate developments. The dial-in number for the conference call is 1-877-705-6003 for domestic participants and 1-201-493-6725 for international participants, with Conference ID #13708850. A live webcast of the conference call can be accessed through the "Investors" tab on the Brickell Biotech website at View Source A replay will be available on this website shortly after conclusion of the event for 90 days.

About Sofpironium Bromide

Sofpironium bromide is a proprietary investigational new chemical entity that belongs to a class of medications called anticholinergics. Anticholinergics block the action of acetylcholine, a chemical that transmits signals within the nervous system that are responsible for a range of bodily functions, including activation of the sweat glands. Sofpironium bromide was retrometabolically designed. Retrometabolic drugs are designed to exert their action topically and are potentially rapidly metabolized into a less active metabolite once absorbed into the blood. Sofpironium bromide was discovered at Bodor Laboratories, Inc. by Dr. Nicholas Bodor D.Sc., d.h.c. (multi), HoF, Graduate Research Professor Emeritus, University of Florida.

About Hyperhidrosis

Hyperhidrosis is a life-altering medical condition where a person sweats more than the body requires to regulate its temperature. More than 15 million people, or 4.8% of the population of the United States, and 12.76% of the population in Japan, are believed to suffer from hyperhidrosis1,2. Primary axillary (underarm) hyperhidrosis is the targeted first indication for sofpironium bromide and is the most common site of occurrence of hyperhidrosis, affecting an estimated 65% of patients with hyperhidrosis in the United States. Additional information can be found on the International Hyperhidrosis Society website: View Source

On November 12, 2020 Nkarta, Inc. (Nasdaq: NKTX), a clinical-stage biopharmaceutical company developing engineered natural killer (NK) cell therapies to treat cancer, reported financial results for the third quarter ended September 30, 2020, and highlighted recent corporate accomplishments (Press release, Nkarta, NOV 12, 2020, View Source [SID1234570791]).

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"We’re excited by the continued progress made at Nkarta this quarter and the ability of our teams to advance Nkarta’s allogeneic, off-the-shelf cell therapy programs," said Paul J. Hastings, President and Chief Executive Officer of Nkarta. "We have dosed the first patient in our first clinical trial of NKX101, continue to prepare our in-house clinical GMP capabilities for the production of NKX019, and remain on track to file Nkarta’s second IND in the first quarter of 2021 for NKX019. With patients always foremost in mind, we remain focused on advancing Nkarta’s NK cell platform as the next foundation in anti-cancer cell therapy."

Recent Developments

First patient dosed in the Phase 1 clinical trial of NKX101, a first-in-class investigational NK cell cancer immunotherapy engineered to express a chimeric antigen receptor (CAR) targeting NKG2D ligand, for the treatment of relapsed/refractory acute myeloid leukemia (AML) and higher risk myelodysplastic syndromes (MDS).
Nadir Mahmood, Ph.D. appointed to the expanded role of Chief Financial and Business Officer, having previously served as Nkarta’s Chief Business Officer. He succeeded Matthew Plunkett, Ph.D., who stepped down as Chief Financial Officer in October 2020.
Alicia J. Hager, J.D., Ph.D. joined Nkarta as its Chief Legal Officer.
Anticipated Near-term Clinical Milestones

In 1Q 2021, Nkarta expects to file an Investigational New Drug (IND) Application for NKX019, an investigational NK cell therapy engineered to target tumors expressing CD19 antigen for the treatment of B-cell malignancies.
Third Quarter 2020 Financial Highlights

Cash and Cash Equivalents: As of September 30, 2020, Nkarta had cash, cash equivalents, restricted cash and short-term investments of $330.2 million, which includes proceeds from the Company’s July 2020 IPO of $265.1 million, net of underwriting discounts and commissions and other offering costs.

R&D Expenses: Research & development expenses were $9.8 million for the third quarter of 2020, which includes $0.7 million of non-cash stock-based compensation expense.

G&A Expenses: General and administrative expenses were $3.9 million for the third quarter of 2020, which includes $0.9 million of non-cash stock-based compensation expense.

Net Loss. Net loss was $13.7 million, or $0.44 per basic and diluted share, for the quarter ended September 30, 2020.
Financial Guidance

Nkarta expects its current cash and cash equivalents will be sufficient to fund its current operating plan into at least the second half of 2023. The company expects cash and cash equivalents at December 31, 2020 to be in the range of $300 million to $310 million.
About NKX101
NKX101 is an investigational, off-the-shelf cancer immunotherapy that uses natural killer (NK) cells derived from the peripheral blood of healthy donors and engineered with membrane-bound IL15 and a chimeric antigen receptor (CAR) targeting NKG2D ligands on tumor cells. NKG2D, a key activating receptor found on naturally occurring NK cells, induces a cell-killing immune response through the detection of stress ligands that are widely expressed on cancer cells. By engineering NKX101 with the proprietary NKG2D-based CAR, the ability of NK cells to recognize and kill tumor cells in pre-clinical models is increased significantly compared to non-engineered NK cells. The addition of membrane-bound IL15, a proprietary version of a cytokine for activating NK cell growth, has been shown in pre-clinical models to enhance the proliferation, persistence and sustained activity of NK cells. A multi-center Phase 1 clinical trial of NKX101 in patients with relapsed/refractory acute myeloid leukemia (AML) or higher risk myelodysplastic syndromes (MDS) is currently enrolling. Additional information about the clinical trial is available on ClinicalTrials.gov, identifier NCT04623944.

About NKX019
NKX019 is an investigational, off-the-shelf cancer immunotherapy that uses natural killer (NK) cells derived from the peripheral blood of healthy donors and engineered with a chimeric antigen receptor (CAR) targeting the CD19 antigen and membrane-bound IL15. CD19 antigen is a B-cell marker and validated target for B cell cancer therapies. NKX019 uses the CAR to target and bind to CD19, leading to an immune response that eliminates CD19-expressing cells in preclinical studies. The addition of membrane-bound IL15, a proprietary version of a cytokine for activating NK cell growth, has been shown in preclinical models to enhance the proliferation, persistence and activity of NK cells. Nkarta plans to file an IND application with the FDA in the first quarter of 2021. A Phase 1 clinical trial of NKX019 in patients with advanced relapsed/refractory B cell malignancies is planned to initiate in 2021.

About Nkarta’s NK Cell Technologies
Nkarta has pioneered a novel discovery and development platform for the engineering and efficient production of allogeneic, off-the-shelf natural killer (NK) cell therapy candidates. The approach harnesses the innate ability of NK cells to recognize and kill tumor cells, and builds upon the important advances in cellular immunotherapy and chimeric antigen receptor (CAR) biology. To enhance the intrinsic activity of NK cells, Nkarta genetically engineers the cells with a CAR that consists of a targeting receptor designed to recognize and bind to specific proteins on the surface of cancerous cells. This receptor is fused to co-stimulatory and signaling domains to amplify cell signaling and NK cell cytotoxicity. Upon binding the target, NK cells become activated and release cytokines that enhance the immune response and cytotoxic granules that lead to killing of the target cell. All of Nkarta’s NK cell therapy candidates are engineered with a membrane-bound IL15, a proprietary version of a cytokine known for activating NK cell growth, to enhance the persistence and activity of the NK cells.

Nkarta’s manufacturing process generates an abundant supply of NK cells that, at commercial scale, is expected to be significantly lower in cost than other current allogeneic and autologous cell therapies. Key to this efficiency is the rapid expansion of donor-derived NK cells using a proprietary NKSTIM cell line, leading to the production of hundreds of individual doses from a single manufacturing run. The platform also features the ability to freeze and store CAR NK cells for an extended period of time and is designed to enable immediate, off-the-shelf administration to patients at the point of care.

On November 12, 2020 Achieve Life Sciences, Inc. (Nasdaq: ACHV), a clinical-stage pharmaceutical company committed to the global development and commercialization of cytisinicline for smoking cessation and nicotine addiction, reported third quarter 2020 financial results and provided an update on the cytisinicline clinical development program (Press release, OncoGenex Pharmaceuticals, NOV 12, 2020, View Source [SID1234570790]).

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Recent Events & Highlights

Initiated the Phase 3 ORCA-2 clinical trial evaluating the efficacy and safety of 3 mg cytisinicline dosed 3 times daily compared to placebo in 750 adult smokers at 15 clinical sites in the United States
Promoted John Bencich to Chief Executive Officer and Dr. Cindy Jacobs to President
Presented successful results from the RAUORA, head-to-head non-inferiority clinical trial comparing cytisinicline and Chantix (varenicline), at the Society for Research on Nicotine and Tobacco European (SRNT-E) Annual Meeting
Presented mechanism of action data comparing 5-HT3 receptor activity of cytisinicline vs. Chantix at SRNT-E, providing rationale for differentiated side effect profile of smoking cessation treatments
Announced smoking cessation Key Opinion Leader Virtual Roundtable to be held on November 17, 2020
"With the initiation of the Phase 3 ORCA-2 trial and the breakthrough evidence, both mechanistically and clinically, in support of cytisinicline in comparison to Chantix, the third quarter of 2020 has undeniably been the most pivotal in the history of our company," commented John Bencich, Chief Executive Officer of Achieve. "Our primary focus in the coming months will be enrollment and execution of ORCA-2, while continuing to explore opportunities for commercialization and expansion into new therapeutic indications, digital health technologies, and audiences who may benefit from cytisinicline, specifically, users of vapes or e-cigarettes."

Phase 3 ORCA-2 Trial Initiation
Achieve’s first Phase 3 ORCA-2 trial was initiated in October 2020 and will randomize 750 U.S. smokers to one of three study arms to determine the efficacy and safety of cytisinicline administered for either 6 or 12 weeks, compared to placebo. The primary endpoint is biochemically verified continuous abstinence during the last 4 weeks of treatment in the 6 and 12-week cytisinicline treatment arms compared to placebo. Each treatment arm will be compared independently to the placebo arm and the trial will be determined to be successful if either or both of the cytisinicline treatment arms show a statistical benefit compared to placebo.

Management Team Update
In September, the Company announced the promotion of John Bencich to Chief Executive Officer and Dr. Cindy Jacobs to President. Mr. Bencich has been serving as Achieve’s Chief Financial and Operating Officer since 2017 and will join the Board of Directors in his role. Dr. Jacobs has been serving as the Chief Medical Officer of Achieve since 2017 and will continue in her role leading the regulatory and clinical development efforts for cytisinicline. Rick Stewart will remain with Achieve as the Executive Chairman of the Board of Directors and Dr. Anthony Clark will remain in his role as Chief Scientific Officer.

RAUORA: Significantly Fewer Adverse Events and Higher Quits Rates for Cytisinicline vs Chantix
Final results from the New Zealand RAUORA Phase 3 non-inferiority clinical trial comparing cytisinicline to varenicline (Chantix) in Māori (indigenous New Zealanders) and whānau (family) of Māori were presented at the SRNT-E Annual Meeting in September 2020. Results showed that cytisinicline met the pre-specified non-inferiority endpoint and was trending towards superiority demonstrating a 4.29% improvement in quit rates in favor of cytisinicline. Subjects in the cytisinicline arm were approximately one and a half times more likely to have quit smoking at 6 months compared to subjects who received varenicline. Importantly, significantly fewer overall adverse events (AEs) were reported in cytisinicline-treated subjects. Of the subjects who experienced AEs (111 in the cytisinicline arm compared to 138 in the varenicline arm), there was significantly less nausea and vivid dreams.

MOA Data Explaining Reduced Nausea and Vomiting with Cytisinicline Presented at SRNT-E
Data presented at the SRNT-E Annual Meeting in September 2020 provides a rationale based on detailed receptor pharmacology to explain why the incidence of nausea and vomiting associated with cytisinicline appears to be consistently lower than that seen with Chantix. The preclinical study, conducted at the University of Cambridge Department of Biochemistry, was designed to examine the in vitro binding characteristics of cytisinicline compared to varenicline at the human 5-HT3 receptor. The study reported an IC50 of 0.50 mM for cytisinicline and 0.25 µM for varenicline, representing a 2000-greater fold agonist binding affinity to the 5-HT3 receptor for varenicline compared to cytisinicline.

Virtual Smoking Cessation KOL Roundtable to be Held November 17, 2020
Achieve announced plans to host a virtual roundtable on cytisinicline and smoking cessation on Tuesday, November 17, 2020, at 12:00PM EST. Five esteemed experts in the field of smoking cessation will discuss the ongoing ORCA-2 trial, review recent cytisinicline data, and discuss the importance of smoking cessation in the midst of the COVID-19 pandemic. Visit View Source for additional information and click here to register for the event.

Financial Results
As of September 30, 2020, the company’s cash, cash equivalents, and restricted cash was $22.4 million. Total operating expenses for the three and nine months ended September 30, 2020 were $3.8 million and $10.0 million, respectively. Total net loss for the three and nine months ended September 30, 2020 was $3.8 million and $10.0 million, respectively.

As of November 12, 2020, Achieve had 3,617,664 shares outstanding.

Conference Call Details
Achieve will host a conference call at 4:30pm Eastern time today, Thursday, November 12, 2020. To access the webcast, log on to the investor relations page of the Achieve website at View Source. Alternatively, access to the live conference call is available by dialing (877) 472-9809 (U.S. & Canada) or (629) 228-0791 (International) and referencing conference ID 8047128. A webcast replay will be available approximately two hours after the call and will be archived on the website for 90 days.