On May 5, 2020 Exicure, Inc. (NASDAQ: XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) constructs, reported that it will present at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting II, occurring June 22 – 24, 2020 (Press release, Exicure, MAY 5, 2020, View Source [SID1234557039]).

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The AACR (Free AACR Whitepaper) poster titled "Phase 1b/2 Study of an Intratumoral TLR9 Agonist Spherical Nucleic Acid (AST-008) and Pembrolizumab: Evidence of Immune Activation" will present updated pharmacodynamic and safety data of AST-008 alone and in combination with pembrolizumab from Exicure’s ongoing Phase 1b/2 study (ClinicalTrials.gov identifier: NCT03684785).

This poster will be presented during the AACR (Free AACR Whitepaper) Virtual Meeting II in the session Late-Breaking Research: Clinical Research 1 / Endocrinology under abstract number LB-140. The abstract will be available for viewing on May 15th and the poster will be available for viewing on June 22nd.

About AST-008

AST-008 is an SNA consisting of toll-like receptor 9 agonists designed for immuno-oncology applications. As of January 31, 2020, we have dosed 17 patients in the Phase 1b stage of the clinical trial. We have observed no treatment related serious adverse events, nor have we observed any dose-limiting toxicity among the treated subjects. The most common reported adverse event was injection site reactions. In December 2019, we received preliminary results from the Phase 1b/2 stage of the clinical trial showing potential signs of anti-tumor activity in patients with Merkel cell carcinoma. In the second quarter of 2020, we plan to initiate a Phase 2 dose expansion for intratumoral AST-008 in combination with approved checkpoint inhibitors to treat two cohorts of patients with advanced or metastatic Merkel cell carcinoma or cutaneous squamous cell carcinoma. Each cohort is expected to enroll up to 29 patients who have failed anti-PD-1/PD-L1, or programmed cell death protein 1/programmed death-ligand 1, therapy. We expect to open a total of up to 15 sites in the United States.

On May 5, 2020 Supernus Pharmaceuticals, Inc. (Nasdaq: SUPN), a pharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, reported financial results for the first quarter of 2020 and associated Company developments (Press release, Supernus, MAY 5, 2020, View Source [SID1234557035]).

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Commercial Update

First quarter 2020 product prescriptions for Trokendi XR and Oxtellar XR, as reported by IQVIA, totaled 203,404, a 2% increase over first quarter 2019.

First quarter 2020 net product sales were $92.5 million, an increase of 11% over $83.1 million in the first quarter of 2019. As previously disclosed, wholesalers, distributors, and pharmacies decreased their inventory levels of the Company’s products in the first quarter of 2019. The Company estimates that this caused net product sales in the first quarter of 2019 to be approximately $10 million lower had inventory levels remained constant, thus favorably impacting year over year net product sales growth in the first quarter of 2020.

Supply of Trokendi XR and Oxtellar XR has not been impacted by COVID-19. The Company has adequate inventory on hand for both products to continue to be available to patients.

Corporate and Product Pipeline Update

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

The Company continues to prepare for the commercial launch of SPN-812 at the end of 2020.
The Company remains engaged with the U.S. Food and Drug Administration (FDA) regarding its New Drug Application (NDA) for SPN-812 for the treatment of ADHD, which has a Prescription Drug User Fee Act (PDUFA) target action date of November 8, 2020.
The Phase III program in adult patients reached approximately 75% of the targeted enrollment before additional enrollment was put on hold in March 2020 due to the impact of COVID-19. The Company is employing virtual efforts to ensure that currently enrolled subjects can progress to completion of treatment. This trial was ahead of schedule prior to the COVID-19 pandemic, with a potential data release in the second half of this year. Depending on when the Company can restart enrollment and complete the study, data from the trial may be pushed into 2021.
CNS portfolio of US WorldMeds

As announced last week, Supernus entered into a definitive Sale and Purchase Agreement to acquire the CNS portfolio of US WorldMeds, a privately-held biopharmaceutical company. The acquisition is expected to close in the second quarter of 2020, subject to certain conditions, including the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act and other customary conditions.
US WorldMeds’ CNS portfolio consists of three marketed products with 2019 net sales and operating earnings of approximately $150 million and $45 million, respectively, and a product candidate with an expected NDA submission in the second half of 2020.
The acquisition expands and strengthens the Company’s neurology portfolio, diversifies its revenue and operating cash flow base and enhances long term growth.
Total consideration consists of an upfront cash payment of $300 million plus regulatory and commercial milestone cash payments up to $230 million. All cash consideration will be funded through existing balance sheet cash.
SPN-820 (NV-5138) – novel first-in-class activator of mTORC1

Supernus and Navitor Pharmaceuticals, Inc., a privately-held company, announced a joint Development and Option Agreement for Navitor’s mTORC1 activator, NV-5138.
Supernus and Navitor will jointly conduct a Phase II clinical program for NV-5138 in treatment-resistant depression. Supernus will pay up to $50 million of the costs of Phase II development, plus certain costs associated with nonclinical development and formulation.
In addition, Navitor has granted Supernus an exclusive, worldwide (excluding Greater China) option to license or acquire NV-5138 prior to initiation of a Phase III clinical program. In exchange for the option to license or acquire NV-5138, Navitor will receive an upfront payment of $25 million, composed of a $10 million option fee and a $15 million equity investment. The equity investment represents an approximately 13% ownership in Navitor. Total payments, exclusive of royalty payments on net sales of NV-5138 and development costs under the agreement, have the potential to reach $410 million to $475 million, which includes the upfront payment, an additional license or acquisition fee depending on whether Supernus ultimately licenses or acquires NV-5138, and subsequent clinical, regulatory and sales milestone payments.
Supernus also will have the first right of refusal for any compound with a similar mechanism of action on mTORC1 as NV-5138 in the central nervous system.
SPN-604 – Novel treatment of bipolar disorder

The Company has reprioritized its research and development (R&D) resources following recent expansion of the product pipeline through mid to late-stage development product candidates. These product candidates include SPN-820 from the Navitor partnership and Apomorphine Infusion Pump from the US WorldMeds transaction. As a result, and given other factors including the estimated timing of a potential launch of SPN-604 and the required investment, the Company is terminating development of SPN-604 for the treatment of bipolar disorder.
Sales and marketing infrastructure

The acquisition of the CNS product portfolio from US WorldMeds includes a sales force of approximately 46 sales representatives that focuses on serving movement disorder specialists in the U.S.
The Company continues to plan on adding salesforce personnel toward the end of 2020 in anticipation of the launch of SPN-812.
"The two business development transactions that we announced over the last couple of weeks strengthen our product portfolio and late-stage pipeline, diversify our revenue base, enhance our long term growth, and strengthen our leadership position in CNS," said Jack Khattar, President and CEO of Supernus. "In addition, they expand our commercial and R&D platforms into the biologics, orphan disease and specialty pharmacy areas."

Operating Expenses

R&D expenses in the first quarter of 2020 were $18.9 million, compared to $15.4 million in the same quarter last year. This increase was primarily driven by enrollment in the SPN-812 Phase III program for adults, initiated in late 2019.

SG&A expenses in the first quarter of 2020 were $42.9 million, compared to $41.0 million in the same quarter last year. This increase was primarily driven by pre-launch activities associated with SPN-812, partially offset by a decrease in marketing expenses for commercial products and a decrease in employee-related expenses.

Operating Earnings and Earnings Per Share

Operating earnings in the first quarter of 2020 were $29.0 million, compared to $25.4 million in the first quarter of 2019. The increase of $3.6 million was primarily due to increased revenue from our two commercial products.

Net earnings (GAAP) in the first quarter of 2020 were $21.5 million, or $0.40 per diluted share, an increase of 18% on a diluted share amount, as compared to $18.3 million, or $0.34 per diluted share, in the same period last year.

Weighted-average diluted common shares outstanding were approximately 53.6 million for the first quarter of 2020, as compared to approximately 54.0 million for the prior year period.

Balance Sheet Highlights

As of March 31, 2020, the Company had $935.6 million in cash, cash equivalents, marketable securities and long term marketable securities, compared to $938.8 million at December 31, 2019. This decrease was primarily attributable to unrealized losses on long term marketable securities resultant from market volatility in the first quarter of 2020.

Financial Guidance

Given the uncertainty caused by the COVID-19 pandemic, the anticipated second quarter acquisition of the CNS portfolio from US WorldMeds and the impact of the partnership with Navitor Pharmaceuticals, the Company is suspending its previously issued full year 2020 financial guidance. The Company expects to update and reinstate full year 2020 guidance no later than the announcement of second quarter 2020 financial results in August.

Conference Call Details

The Company will hold a conference call hosted by Jack Khattar, President and Chief Executive Officer, and Greg Patrick, Senior Vice President and Chief Financial Officer, to discuss these results at 9:00 a.m. Eastern Time, on Wednesday, May 6, 2020.

On May 5, 2020 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ: APTO, TSX: APS), a clinical-stage company developing highly differentiated agents that target the underlying mechanisms of cancer, reported financial results and corporate update for the three months ended March 31, 2020 and reported on corporate developments (Press release, Aptose Biosciences, MAY 5, 2020, View Source [SID1234557034]).

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The net loss for the quarter ended March 31, 2020 was $11.5 million ($0.15 per share) compared with $5.5 million ($0.14 per share) for the quarter ended March 31, 2019. Total cash and cash equivalents and investments as of March 31, 2020 were $90.0 million. Based on current operations, we expect that cash on hand and available capital provide the Company with sufficient resources to fund all planned Company operations including research and development into 2022.

Key Corporate Highlights

Aptose COVID-19 Update — Because treatment of critically ill patients with hematologic cancers is not elective, Aptose’s Phase 1 clinical trials for CG-806 and APTO-253 have continued to enroll and treat patients, despite recent events. The company is continually adapting to COVID-19-driven disruptions at clinical sites, but thus far experienced no material delays. Although headwinds may be felt in the coming days, Aptose has introduced adaptive and precautionary measures and safeguards such as virtual monitoring for the safety of the patients and clinical teams as a top priority.

CG-806 Phase 1 a/b B-cell Malignancy Clinical Study –- During the quarter, Aptose successfully completed dose level three (450 mg dose cohort) of the CG-806 trial. The safety review monitoring committee then reviewed all data and unanimously agreed to advance to the 600 mg dose level. Currently, patient treatment has ongoing in this fourth dose cohort. To date, CG-806 continues to be well-tolerated, and initial indications of desired pharmacologic activity have been observed starting at the second dose level, including strong inhibition of several oncogenic driver kinases and a robust increase in peripheral blood lymphocytes – or lymphocytosis – classically ascribed as a response to the inhibition of BTK. Currently, 21 U.S. sites are open for screening and enrolling patients for the study, with additional sites scheduled to come on board. More information is available at www.clinicaltrials.gov.

CG-806 Proposed AML Study — CG-806 is the only BTK inhibitor that also possesses strong FLT3 inhibitory activity, giving it broad therapeutic potential across hematology in both lymphoid and myeloid malignancies. Based on safety, pharmacokinetic and pharmacodynamic data from patients in the ongoing Phase 1 a/b study in B cell malignancies, we now have identified what we believe may be a therapeutic starting dose for the treatment of AML patients, and are finalizing our efforts to submit the IND application to proceed into a parallel study in this new indication. In due course, this initiative will be designed to treat AML and MDS patients with CG-806 as a stand alone treatment and in combination with other agents.

APTO-253 Phase 1b Clinical Study –- APTO-253 is the only known clinical-stage molecule that can directly target and inhibit expression of the MYC oncogene, shown to reprogram survival signaling pathways and contribute to drug resistance in many malignancies, including AML and B cell malignancies. In the ongoing Phase 1b trial, Aptose has observed MYC suppression at all dose levels to date, and plans to continue dose escalation to higher dose levels that may deliver responses for AML and MDS patients and will inform decisions on how to move forward with development of the molecule. Other therapies directed at MYC suppression are limited by severe toxicities, drug resistance and myelosuppression of the healthy bone marrow, which has not been observed in extensive preclinical testing of APTO-253 and is being borne out in clinical testing to date. More information is available at www.clinicaltrials.gov.

A summary of the results of operations for the three-month periods ended March 31, 2020 and 2019 is presented below:

The net loss for the three-month period ended March 31, 2020 increased by $6.0 million to $11.5 million as compared with $5.5 million for the comparable period in 2019 primarily as a result of an increase of $3.7 million in stock-based compensation in the current period, a combined increased in program costs and related labor costs of approximately $1.9 million on our CG-806 and APTO-253 development programs, and higher cash-based general and administrative expenses of $569 thousand. These expenses were partially offset by higher net finance income of $308 thousand in the current period, which increased by $214 thousand compared to the comparative period, mostly as a result of higher interest earned on larger balances of cash equivalents and investments held during the three-month period ended March 31, 2020.

Research and Development

The research and development expenses for the three-month periods ended March 31, 2020 and 2019 were as follows:

Research and development expenses for the three-month period ended March 31, 2020 were $5.9 million as compared with $3.3 million for the comparative period in 2019, an increase of approximately $2.6 million. Changes to the components of our research and development expenses presented in the table above were primarily as a result of the following events:

In the three-month period ended March 31, 2020, program costs for our CG-806 program consisted mostly of manufacturing costs, including costs to scale up manufacturing and research costs associated with optimizing the formulation, and clinical trial costs. In the three-month period ended March 31, 2019, program costs for our CG-806 program consisted mostly of costs to complete the preclinical studies and prepare regulatory filings in support of an IND filing and the manufacturing of drug product for the Phase I clinical trial.

In the three-month period ended March 31, 2020, program costs for our APTO-253 program consisted mostly of costs related to the Phase Ib clinical trial, and manufacturing costs for a second GMP. In the comparative period in 2019, the Company completed production of a GMP batch of drug product, and initiated necessary studies to present to the FDA in support of removing the clinical hold.

An increase in personnel expenses mostly related to seven new positions, including a Chief Medical Officer, hired since the second quarter of 2019 to support two Phase I clinical trials.

Stock-based compensation increased by approximately $682 thousand in the three months ended March 31, 2020, compared with the three months ended March 31, 2019 mostly related to an increase in the number of options granted in the current period and a higher grant date fair value of options. In the current period, 1,322,500 stock options were granted to employees working in research and development with an average grant date fair value of $4.40. In the comparative period, 390,050 stock options were granted to employees in research and development with a grant date fair value of $1.29.

General and Administrative

The general and administrative expenses for the three-month periods ending March 31, 2020 and 2019 were as follows:

General and administrative expenses for the three-month period ended March 31, 2020 were $5.9 million as compared with $2.3 million for the comparative period, an increase of approximately $3.6 million. The increase was primarily as a result of the following:

General and administrative expenses, other than stock-based compensation and depreciation of equipment, increased by approximately $569 thousand in the three months ended March 31, 2020, primarily as a result of higher personnel related costs mostly related to two additional hires, including a Chief Business Officer, in the second quarter of 2019, higher insurance and professional and regulatory costs, and higher office administrative costs.

Stock-based compensation increased by approximately $3.1 million in the three months ended March 31, 2020, compared with the three months ended March 31, 2019 mostly related to an increase in the number of options granted in the current period, and a higher grant date fair value of options. In the current period, 4,786,334 stock options were granted to directors, executive offices and general and administrative employees with an average grant date fair value of $4.66 and we issued 645,000 restricted stock units (RSUs) with an average grant date fair value of $7.32. In the comparative period, 1,024,000 stock options were granted to directors, executive officers and general and administrative employees with a grant date fair value of $1.29.

Conference Call and Webcast

Aptose will host a conference call to discuss results for the quarter ended March 31, 2020 today, Tuesday, May 5, 2020 at 5:00 PM ET. Participants can access the conference call by dialing 1-844-882-7834 (North American toll free number) and 1-574-990-9707 (international/toll number) and using conference ID # 6470926. The conference call can be accessed here and will also be available through a link on the Investor Relations section of Aptose’s website at View Source An archived version of the webcast along with a transcript will be available on the Company’s website for 30 days. An audio replay of the webcast will be available approximately two hours after the conclusion of the call for seven days by dialing 1-855-859-2056 (toll free number) and 1-404-537-3406 (international/toll number), using the conference ID # 6470926.

On May 5, 2020 NuCana plc (NASDAQ: NCNA) reported it has re-commenced enrollment of new patients in NuTide:121, its ongoing global Phase III study of Acelarin (NUC-1031) plus cisplatin in patients with biliary tract cancer (Press release, Nucana BioPharmaceuticals, MAY 5, 2020, View Source [SID1234557033]). The re-opening of NuTide:121 has begun in certain geographies, including Australia, Canada, South Korea, Taiwan, Ukraine and the United Kingdom. NuCana will work closely with the clinicians involved in NuTide:121 to re-open all of the clinical sites to new patient enrollment as soon as practicable.

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"We will continue to monitor the impact of the COVID-19 pandemic, but re-opening this study is important for patients with biliary tract cancer who need treatment" said Hugh S. Griffith, NuCana’s Founder and Chief Executive Officer. Mr. Griffith added "The COVID-19 pandemic has had a dramatic impact on the global healthcare delivery system and on cancer patient care, and it is vital that the development of new cancer treatments, such as Acelarin, is resumed as quickly as possible.

NuTide:121 is a global, multi-center, randomized Phase III study of Acelarin, NuCana’s ProTide transformation of gemcitabine, that will enroll up to 828 patients in approximately 120 sites across North America, Europe, Asia and Australia. Patients are being randomized 1:1 and treated with either a combination of Acelarin (725 mg/m2) plus cisplatin (25 mg/m2) or the current standard of care regimen, gemcitabine (1,000 mg/m2) plus cisplatin (25 mg/m2).

On May 5, 2020 Quanterix Corporation (NASDAQ:QTRX), a company digitizing biomarker analysis to advance the science of precision health, reported financial results for the three months ended March 31, 2020 (Press release, Quanterix, MAY 5, 2020, View Source [SID1234557032]).

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"Q1 2020 was yet another strong quarter for Quanterix underscored by strong execution and revenue growth, particularly given the COVID-19 pandemic," said Kevin Hrusovsky, President, Chairman and Chief Executive Officer, Quanterix. "We are making continuous advances in many neurology, immunity and COVID-19 applications. We are also utilizing our Accelerator Laboratory to run trials and studies for customers experiencing disruptions in their laboratories. Despite the present near-term challenges, we remain positive and committed to the unprecedented market opportunity."

First Quarter 2020 Financial Highlights

Key financial results for the first quarter of 2020 are shown below:

·Q1 revenue was $15.7M versus prior year Q1 of $12.3M, an increase of 27%;
·Q1 product revenue was $9.8M versus prior year Q1 of $9.5M, an increase of 3%;
·Q1 service and other revenue was $5.8M versus prior year Q1 of $2.8M, an increase of 107%;
·Q1 GAAP gross margin was 43.3% versus prior year Q1 of 48.7%; Q1 non-GAAP gross margin was 48.5% versus prior year Q1 of 48.7%. Q1 2020 gross margin includes 200 bps of adverse impact from our successful HD-X trade-in program.

For additional information on the non-GAAP financial measures included in this press release, please see "Use of Non-GAAP Financial Measures" and "Reconciliation of Non-GAAP Financials" below.

First Quarter 2020 Business Highlights

·Early in the progression of COVID-19, we took aggressive steps to protect our employees and their families, while rapidly implementing a resilience plan to maintain our operations and support our global customer base. As a result, we have successfully maintained our operations and have expanded our Accelerator capacity to support customer disruptions and sustain clinical trials.
·To support the global public health response, we are engaging closely with researchers and KOLs around the world. We have implemented a set of important and differentiated tools for COVID-19 investigators to immediately deploy to study disease progression, life-threatening cytokine release syndrome and patient-treatment response. Researchers are leveraging Simoa’s extreme sensitivity and specificity of cytokine panels to measure levels and profiles in non-critical and critical COVID-19 patients to identify ways to combat the virus and help prevent cytokine storm. We are also working toward developing a SARS-COV-2 quantitative IgG assay, developing an antigen early detection assay in blood and a high-definition SARS-COV-2 assay to enable research pursuits.

·Sponsored a webinar with Powering Precision Health, "Highly Sensitive and Accurate Multiplex Test to Monitor the Immune Response to Viral Infection," which featured insights from several researchers actively treating COVID-19 patients in Europe and China. Specifically, the webinar discussed capabilities to conduct highly sensitive and accurate multiplexed tests for cytokines and chemokines in serum and plasma.
·Reinforced the growing utility of Simoa in neurology research, including results published in JAMA Network Open, demonstrating the promise of blood biomarkers to serve as clinical tools for objectively identifying and monitoring sports-related concussions (SRCs) and mild traumatic brain injuries (mTBIs), marking a notable advance in the pursuit of a concussion diagnostic test. Conducted as part of the CARE Consortium, and funded by the US Department of Defense (DOD) and the National Collegiate Athletic Association (NCAA), the study used Simoa to evaluate a total of 504 collegiate athletes in what is considered the largest athletic cohort for a biomarker concussion study to date.
·Quanterix’ Simoa technology was highlighted in 53 new publications, bringing total Simoa-specific inclusions to over 789 publications.

Conference Call

In conjunction with this announcement, Quanterix Corporation will host a conference call on May 5, 2020, at 4:30 p.m., EDT to discuss the Company’s financial results and business outlook. To access this call, dial (833) 686-9351 for domestic callers, or (612) 979-9890 for international callers. Please reference the following conference ID: 4994882.

A live webcast will be accessible on the Investors section of Quanterix’ website: View Source The webcast will be available on the Company’s website following completion of the call.