On March 10, 2021 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biotechnology company focused on developing and commercializing innovative medicines worldwide, reported that the first patient has been dosed in a Phase 1 clinical trial of BGB-15025, its investigational hematopoietic progenitor kinase 1 (HPK1) inhibitor (Press release, BeiGene, MAR 10, 2021, View Source [SID1234576392]). BGB-15025 is designed to be a potent and highly selective small molecule oral inhibitor of HPK1, a kinase downstream of the T cell receptor (TCR) signaling pathway that is believed to play a key role in T cell activation.

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"We are incredibly proud of BeiGene’s research organization, which now has over 450 people, and its ability to discover not only potentially best-in-class cancer treatments but also investigational agents like BGB-15025, which we believe to be among the first HPK1 inhibitors to enter the clinic and represent a novel immuno-oncology approach targeting T cell activation to fight cancer growth," commented Lai Wang, Ph.D., Senior Vice President, Head of Global Research, Clinical Operations & Biometrics and APAC Clinical Development at BeiGene. "We believe that the unique nature of BGB-15025 and the HPK1 pathway provides us with a compelling scientific rationale for investigating it as a monotherapy and in combination with our anti-PD-1 antibody, tislelizumab. We are excited to advance its clinical development globally."

This first-in-human Phase 1 trial (NCT04649385) will assess the safety, tolerability, pharmacokinetics, and preliminary antitumor activity of BGB-15025 alone and in combination with tislelizumab in patients with advanced solid tumors. This trial will be conducted in multiple countries globally.

About BGB-15025

BGB-15025 is an investigational hematopoietic progenitor kinase 1 (HPK1) inhibitor discovered and being developed by BeiGene. HPK1 is a key negative feedback regulator of T-cell receptor signaling, which is believed to play a key role in antitumor immune response. In preclinical studies, the inhibition of HPK1 enhanced T-cell activation, which is expected to enhance the anti-tumor activity of anti-PD-1 inhibitors such as BeiGene’s tislelizumab.

About Tislelizumab

Tislelizumab (BGB-A317) is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages. In pre-clinical studies, binding to FcγR on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells. Tislelizumab is the first approved medicine from BeiGene’s immuno-oncology biologics program and is being developed globally as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers.

Tislelizumab is approved in China for first-line treatment of patients with advanced squamous non-small cell lung cancer (NSCLC) in combination with chemotherapy. Tislelizumab has also received conditional approval in China for the treatment of patients with classical Hodgkin’s lymphoma (cHL) who received at least two prior therapies, and for the treatment of patients with locally advanced or metastatic urothelial carcinoma (UC) with PD-L1 high expression whose disease progressed during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. Full approval for these indications is contingent upon results from ongoing randomized, controlled confirmatory clinical trials.

In addition, three supplemental Biologics License Applications for tislelizumab have been accepted and are under review in China for first-line treatment of patients with advanced non-squamous NSCLC in combination with

chemotherapy, for the second- or third-line treatment of patients with locally advanced or metastatic NSCLC who progressed on prior platinum-based chemotherapy, and for previously treated unresectable hepatocellular carcinoma.

Currently, 15 potentially registration-enabling clinical trials are being conducted in China and globally, including 12 Phase 3 trials and two pivotal Phase 2 trials.

In January 2021, BeiGene and Novartis entered into a collaboration and license agreement to develop, manufacture, and commercialize tislelizumab in North America, Europe, and Japan.
Tislelizumab is not approved for use outside of China.

On March 10, 2021 Harpoon Therapeutics, Inc. (Nasdaq: HARP), a clinical-stage immunotherapy company developing a novel class of T cell engagers, reported financial results for the fourth quarter and full year ended December 31, 2020 and provided a corporate update (Press release, Harpoon Therapeutics, MAR 10, 2021, View Source [SID1234576391]).

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"This past year was transformative for Harpoon, culminating in the pipeline update we provided in December last year that detailed the strong progress we have made across all our clinical programs," said Jerry McMahon, Ph.D., President and Chief Executive Officer of Harpoon Therapeutics. "We are encouraged by what we have observed in our dose escalation trials including a confirmed partial response for HPN424, our most advanced program. With our successful follow-on equity offering in January 2021, we are in a strong financial position and we expect to generate and present data on all four of our clinical TriTAC programs throughout the course of 2021, including advancement of the portfolio into multiple dose expansion studies."

Fourth Quarter 2020 Business Highlights and Other Recent Developments

In January 2021, Harpoon announced that the the first patient had been dosed in a Phase 1/2 clinical trial for HPN328, the company’s fourth TriTAC in clinical development. HPN328 targets delta-like ligand 3 (DLL3) for the treatment of small cell lung cancer (SCLC) and other DLL3-expressing tumors.

In January 2021, Harpoon completed a successful follow-on public offering of its common stock resulting in net proceeds of approximately $108.1 million, after deducting underwriting discounts and commissions and other offering costs.

In December, Harpoon provided a corporate pipeline update and reported a confirmed partial response based on RECIST v1.1 criteria for its most advanced program, HPN424 for the treatment of metastatic castration-resistant prostate cancer (mCRPC). As of December 1, 2020, the the highest fixed dose cohort at 160ng/kg, demonstrated that one patient has achieved a confirmed partial response in the ongoing study. In addition, 3 of the 7 patients enrolled in this cohort, had serum PSA reductions, including one with a reduction of 50% (PSA50). Presentation of Phase 1 data and initiation of an expansion cohort is planned for the first half of 2021 with interim data from this expansion cohort anticipated by the end of 2021.

In December, Harpoon also provided an update on dose escalation trials for HPN536 and HPN217. For the HPN536 Phase 1/2a clinical trial for the treatment of ovarian and pancreatic cancers and other mesothelin-expressing solid tumors, dosing has occurred across 9 fixed-dose cohorts of 6 to 280ng/kg and 1 step dose cohort up to 600ng/kg. Initiation of an expansion cohort is anticipated in the second half of 2021, with a presentation of Phase 1 clinical data expected by year-end 2021. Additionally, the Phase 1 dose escalation for HPN217 (a BCMA-targeting TriTAC) is progressing well, and as of December 1, 2020, 6 relapsed/refractory multiple myeloma patients had been treated in fixed dose cohorts ranging from 5 to 810µg, reflecting a more than 100-fold increase in dose in 8 months. A presentation of interim data is anticipated in 2021, and initiation of a dose expansion cohort is expected in the second half of 2021.

In January 2021, Harpoon was granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for HPN217 for the treatment of multiple myeloma. HPN217 targets B-cell maturation antigen (BCMA).

Harpoon nominated its first ProTriTAC product candidate, HPN601, which is currently in preclinical development and targets epithelial cell adhesion molecule (EpCAM), and is applicable to a wide array of solid tumors. ProTriTACs have the potential for additional tumor specificity and enhanced safety profiles due to limited interaction with their molecular targets in healthy tissue, which enables targeting tumor-associated antigens that may be more broadly expressed. Harpoon presented encouraging preclinical data for HPN601 at the 35th Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) annual meeting on November 12, 2020.
Fourth Quarter and Full Year 2020 Financial Results

Harpoon ended the fourth quarter of 2020 with $150.0 million in cash, cash equivalents, and marketable securities compared to $155.1 million as of December 31, 2019. The cash balance at the end of the year does not include the follow-on financing that closed on January 11, 2021 resulting in net proceeds of approximately $108.1 million.

Revenue for the quarter ended December 31, 2020 was $7.5 million compared to $2.2 million for the quarter ended December 31, 2020. Revenue for the year ended December 31, 2020 was $17.4 million, compared to $5.8 million for the prior year. During both the three month and full year periods, the increase in revenue was primarily due to revenue recognized from the development and option agreement with AbbVie, signed in November 2019.

Research and development (R&D) expense for the quarter ended December 31, 2020 was $15.1 million compared to $12.7 million for the quarter ended December 31, 2019. R&D expense for the year ended December 31, 2020 was $52.6 million, compared to $41.6 million for the prior year. The increase for both periods primarily arose from higher clinical development and personnel-related expense, which included conducting preclinical studies and the continuation and preparation of the clinical trials for HPN424, HPN536, HPN217 and HPN328. These higher expenses were offset by a decrease in manufacturing costs due to a scale up of manufacturing activities in 2019 compared to 2020 to support our four TriTAC product candidates in various stages of development.

General and administrative (G&A) expense for the quarter ended December 31, 2020 was $3.9 million compared to $4.3 million for the quarter ended December 31, 2019. General and administrative expenses for the year ended December 31, 2020 were $16.2 million, compared to $22.4 million for the prior year. The decrease was primarily attributable to lower legal fees associated with the Maverick litigation incurred in 2020, partially offset by an increase in personnel expenses related to an increase in headcount and other professional services to support our operations as a public company.

Net loss for the quarter ended December 31, 2020 was $11.4 million compared to $14.3 million for the quarter ended December 31, 2019. Net loss for the year ended December 31, 2020 was $49.9 million, compared to $55.6 million for the prior year.
Anticipated 2021 Milestones

HPN424 – in the first half of 2021, present interim data from the dose escalation phase of our Phase 1/2a trial and initiate the dose expansion cohort
HPN536 – in the second half of 2021, initiate the dose expansion cohort of our Phase 1/2a trial and, by year end 2021, present interim Phase 1 data from the dose escalation phase of the trial
HPN217 – in the second half of 2021, initiate the dose expansion cohort of our Phase 1/2 trial, and, in 2021, present interim data from the dose escalation phase of the trial
HPN328 – in the second half of 2021, present interim data from the dose escalation phase of our Phase 1/2 trial
2021 Financial Guidance

For 2021, management estimates cash used in operating activities of approximately $85 to $95 million.

COVID-19 Business Update

In response to the ongoing COVID-19 pandemic, Harpoon has established testing and other protocols for personnel access to its headquarter offices and laboratory although the majority of the company’s employees continue to telecommute. Harpoon is currently continuing its clinical trials, and has not yet experienced any material delays or impacts as a result of the COVID-19 pandemic. In addition, Harpoon’s third-party contract manufacturers continue to operate at or near normal levels. Harpoon continues to assess the potential impact of the COVID-19 pandemic on its business and operations, including its programs, expected timelines, expenses, manufacturing activities and preclinical and clinical trials. The full extent to which the COVID-19 pandemic may have a negative impact on Harpoon’s business, assets, results of operations and financial condition will depend on future developments that are highly uncertain and cannot be accurately predicted.

Conference Call and Webcast

Harpoon’s management will host a webcast and conference call at 4:30 p.m. ET / 1:30 p.m. PT today, March 10, 2021, to discuss the financial results for the fourth quarter and full year 2020 and provide a corporate update. The live call may be accessed by dialing 866-951-6894 for domestic callers and 409-216-0624 for international callers and entering the conference code: 4785099. A live webcast and archive of the call will be available online from the investor relations section of the company website at View Source

On March 10, 2021 Protagonist Therapeutics, Inc. ("Protagonist" or the "Company") (Nasdaq: PTGX) reported financial results and provided a corporate update for the fourth quarter and full year ended December 31, 2020 (Press release, Protagonist, MAR 10, 2021, View Source [SID1234576390]).

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"The year 2020 marked a significant period of growth for Protagonist as we focused on expanding the scope of our pipeline and advancing different development molecules in multiple clinical programs and indications," commented Dinesh V. Patel, Ph.D., Protagonist President and Chief Executive Officer. "In December, we released compelling interim data from our ongoing Phase 2 trial with our most advanced candidate, rusfertide, which has received orphan drug designation and Fast Track designation for the treatment of polycythemia vera, a rare disease that affects about 100,000 treated patients in the U.S. alone. In the first half of 2021, we expect to complete our planned regulatory interactions and finalize the registrational clinical development plan, which represents an important turning point for the Company as rusfertide has the potential to become a new therapeutic treatment option for polycythemia vera patients. Continuing with the momentum of building our rusfertide portfolio, we expect to announce initial results of the hereditary hemochromatosis proof-of-concept trial in the second half of 2021 and select an additional indication for this candidate in 2021."

Dr. Patel added, "This past year, we continued to make progress with our ongoing 150-patient, Phase 2 IDEAL trial in ulcerative colitis with the gut-restricted alpha-4-beta-7 integrin blocker PN-943 and expect to complete this study in 2022. During the year, we also added two new clinical development stage assets to the ongoing oral IL-23 receptor antagonist program with our partner, Janssen. With a strong cash position through mid-2024, we look forward to focusing our capital to continue building value across our portfolio."

PRODUCT DEVELOPMENT AND CORPORATE UPDATE

Disorders of Red Blood Cells and Iron Regulation

Rusfertide (PTG-300)
Investigational, injectable, hepcidin mimetic discovered through our peptide technology platform. Hepcidin regulates iron homeostasis and controls the absorption, storage, and distribution of iron in the body. Rusfertide is currently being evaluated for disorders associated with iron overload and excessive erythrocytosis (red blood cell production).

At the American Society of Hematology (ASH) (Free ASH Whitepaper) conference this past December, Protagonist presented updated data for 18 patients with polycythemia vera ("PV") treated with rusfertide in the ongoing Phase 2 study, which demonstrated dramatic decreases in the need for therapeutic phlebotomy by maintaining control over blood hematocrit levels.
By mid-2021, Protagonist expects to complete enrollment of 50 patients for the ongoing Phase 2 study of rusfertide in low-risk and high-risk PV patients requiring frequent phlebotomy treatment.
The Company is consulting with regulatory authorities on the registrational clinical development plan for rusfertide in PV in the first half of 2021.
In December 2020, Protagonist released findings from a large-scale independent analysis of real-world data, which demonstrated that hematocrit levels are not adequately managed for a majority of PV patients on currently available treatment options, across broad categories, including both high-risk and low-risk patient groups.
In December 2020, the U.S. Food and Drug Administration ("FDA") granted Fast Track designation to rusfertide for PV. In October 2020, the European Medicines Agency ("EMA") granted orphan drug designation to this candidate in the same indication. The FDA previously awarded rusfertide orphan drug designation in the U.S.
Protagonist is expecting to announce preliminary results in the second half of 2021 from the ongoing Phase 2 open-label proof-of-concept study of rusfertide in patients with hereditary hemochromatosis ("HH"); a disease affecting over a million people in the U.S. and with no approved therapies.
Beyond PV and HH, the Company expects to select a third indication for rusfertide in 2021.
During the first quarter of 2021, Protagonist initiated a new open-label Phase 2 study for rusfertide in PV patients with routinely elevated hematocrit levels (>48%).
Inflammatory Bowel Diseases

PN-943
Investigational, orally delivered, gut-restricted alpha-4-beta-7 specific integrin antagonist for inflammatory bowel diseases.

The 150-patient Phase 2 study (the "IDEAL" study) evaluating the safety, tolerability and efficacy of PN-943 in patients with moderate to severe ulcerative colitis is underway and completion is expected in 2022.
Oral IL-23 Receptor Antagonists

PTG-200; PN-235; PN-232
Investigational, orally delivered, IL-23 receptor antagonists.

In October 2020, Protagonist and Janssen announced two new, second-generation oral candidates, PN-235 and PN-232, to be developed as part of our joint oral IL-23 pathway blocker portfolio strategy. A Phase 1 study of PN-235 and a Phase 1 study of PN-232 are expected to be completed in the second half of 2021.
The Phase 2A proof-of-concept study (the "PRISM" study) with the first-generation candidate PTG-200 for patients with moderate to severe Crohn’s disease is continuing to enroll in 2021.
Financial Update

During the fourth quarter of 2020, the Company raised approximately $115.0 million through an underwritten public offering of common stock where 5,476,189 shares were sold at a price to the public of $21.00 per share.
The Company sold an additional 918,000 shares through its At-the-Market ("ATM") program during October 2020, raising $18.9 million at an average net price of $20.56 per share.
Throughout 2020, Protagonist raised $255 million in capital, net expenses, through two public offerings and its ATM program.
Financial Results

Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities as of December 31, 2020 were $307.8 million. The Company expects current cash, cash equivalents and marketable securities and access to its debt facility to be sufficient to fund its planned operating and capital expenditures through first half of 2024.
License and Collaboration Revenue: License and collaboration revenue was $5.7 million for the fourth quarter of 2020 compared to $2.7 million for the same period of 2019. The increase was primarily due to the additional services provided to Janssen under the collaboration agreement during 2020 related to PN-232 and PN-235. License and collaboration revenue for the full year 2020 was $28.6 million compared to $0.2 million for 2019. The increase that occurred in Protagonist’s year over year revenue under the Janssen collaboration included: completion of additional services during 2020, primarily related to PN-232 and PN-235; an update to the forecast for remaining services to be completed under the collaboration, accelerating our overall percentage completion under the accounting performance obligation; and the previously reported 2019 one-time cumulative adjustment related to the application of revenue recognition principles following the May 2019 amendment of the Janssen collaboration agreement, which previously reduced revenue by $9.4 million during 2019.
Research and Development ("R&D") Expenses: R&D expenses for the fourth quarter and full year 2020 were $19.5 million and $74.5 million respectively, as compared to $15.9 million and $65.0 million, respectively, for the same periods of 2019. The increases were primarily due to advancing our clinical trials with our pipeline assets rusfertide and PN-943, as well as all three of our IL-23 receptor antagonist assets under the Janssen collaboration (PTG-200, PN-235 and PN-232).
General and Administrative ("G&A") Expenses: G&A expenses for the fourth quarter and full year 2020 were $5.0 million and $18.6 million, respectively, as compared to $4.1 million and $15.7 million for the same periods of 2019. The increases were primarily related to professional fees, insurance costs and employee compensation related expenses supporting the growth in our operations.
Net Loss: The fourth quarter net loss was $18.9 million, or a net loss of $0.48 per share, and the full year 2020 net loss was $66.2 million, or a net loss of $1.92 per share, compared to the fourth quarter of 2019 net loss of $17.5 million, or a net loss of $0.63 per share, and the full year 2019 net loss was $77.2 million, or a net loss of $2.98 per share.
Conference Call and Webcast Information

To access the live call, dial 1-844-515-9178 (U.S./Canada) or 1-614-999-9313 (International) and refer to conference ID #: 7756175. A live webcast of the call will also be accessible on the Investors section of the Company’s website at www.protagonist-inc.com. The replay will be available on the company’s website approximately two hours after the call and will remain available for 60 days.

On March 10, 2021 ChromaDex Corp. (NASDAQ:CDXC) reported fourth quarter and full year 2020 financial results (Press release, ChromaDex, MAR 10, 2021, View Source [SID1234576389]).

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Fourth Quarter 2020 and Recent Highlights

Total net sales of $15.4 million, up 18% from $13.1 million in the year ago quarter.
Tru Niagen net sales of $12.3 million, a 21% increase from the year ago quarter.
Gross margin of 61.0%, a 400 basis point increase from the year ago quarter.
Net loss was ($6.1) million or ($0.10) per share, an improvement of $0.05 per share year-over-year.
Adjusted EBITDA excluding total legal expense, a non-GAAP measure, was a loss of ($1.1) million, a $1.0 million improvement year-over-year.
Growing body of clinical research suggests that nicotinamide riboside ("NR") may support important areas of human health.
Phase 2 clinical study showed a reduction in liver fat and inflammatory markers in patients with non-alcoholic fatty liver disease (NAFLD) when receiving a nutritional protocol that included NR.
Phase 3 clinical study on 300 patients in Turkey with mild-to-moderate COVID-19 showed a 3.5 day reduction in recovery time when adding a daily nutritional protocol that included NR to the local standard of care.
Tru Niagen received the ‘Most Favourite Brand’ award by Watsons Hong Kong loyalty members for the second consecutive year.
Full Year 2020 Highlights

Delivered on financial outlook to investors across all metrics. Slightly better than target on selling and marketing and G&A expense.
Total net sales of $59.3 million, up 28% from $46.3 million in the prior year.
Tru Niagen net sales of $47.1 million, a 31% increase from $36.1 million in the prior year.
Gross margins of 59.5%, a 390 basis point increase from the prior year.
Lower selling and marketing expense as a percentage of net sales (35.4% in 2020 versus 39.4% in 2019).
Net loss was ($19.9) million or ($0.33) per share, an improvement of $0.24 per share year-over-year.
Adjusted EBITDA excluding total legal expense, a non-GAAP measure, was a loss of ($1.0) million for FY 2020, a $7.8 million improvement year-over-year.
Signed 225th material transfer agreement through ChromaDex External Research Program (CERP), which has resulted in 60 published studies to date, including 11 published clinical studies, on Niagen.
Expanded in Europe and Australia, following regulatory approval for Tru Niagen.
Nestlé Health Science’s new Celltrient Cellular Energy products featuring Tru Niagen launched in the United States, following the launch of Tru Niagen capsules on Persona, a Nestlé Health Science company and leading personalized vitamin subscription program, earlier this year.
ChromaDex Chief Scientific Advisor, Dr. Charles Brenner, Receives 2020 National Scientific Achievement Award from the American Society for Nutrition.
"ChromaDex’s philosophy of focusing on business fundamentals with a science-based strategic approach continued in 2020," said ChromaDex CEO Rob Fried. "In 2021, we will further our position as the world’s leading NAD+ company by growing the Tru Niagen brand, extending partnerships and furthering scientific advancements with world-leading scientists."

Results of operations for the three months ended December 31, 2020

For the three months ended December 31, 2020 ("Q4 2020"), ChromaDex reported net sales of $15.4 million, up 18% compared to $13.1 million in the fourth quarter of 2019 ("Q4 2019"). The increase in Q4 2020 revenues was driven by growth in sales of Tru Niagen and Niagen ingredient revenues.

Gross margin percentage improved by 400 basis points to 61.0% in Q4 2020 compared to 57.0% in Q4 2019. The improvement in gross margin percentage was driven by the positive impact of increased Tru Niagen consumer product sales and product cost savings initiatives.

Operating expenses decreased by $0.8 million to $15.5 million in Q4 2020, compared to $16.3 million in Q4 2019. The decrease in operating expenses was driven by a decrease of $1.9 million in general and administrative expense, and a decrease of $0.1 million of R&D expense, partially offset by $1.2 million of higher selling and marketing expenses. The decrease in general and administrative expense was driven by the absence of $2.2 million of bad debt expense related to the write-off of an Elysium receivable in Q4 2019.

The net loss for Q4 2020 was ($6.1) million or ($0.10) per share as reported compared to a net loss of ($8.9) million or ($0.15) per share for Q4 2019 as reported. Non-GAAP net loss per share in Q4 2019 was ($0.11) excluding a $2.2 million, or $0.04 per share, non-cash charge related to the write-off of the Elysium receivable as bad debt.

Adjusted EBITDA excluding total legal expense, a non-GAAP measure, was a loss of ($1.1) million for Q4 2020, compared to a loss of ($2.1) million for Q4 2019, a $1.0 million improvement.

ChromaDex defines Adjusted EBITDA excluding total legal expense as net income or (loss) which is adjusted for interest, income tax, depreciation, amortization, non-cash stock compensation costs, severance and restructuring expense, bad debt expense related to Elysium Health and total legal expense.

For Q4 2020, the net cash flow from operating activities was at break-even, versus ($0.6) million used in Q4 2019.

Results of operations for the year ended December 31, 2020

For the full year ended December 31, 2020 ("FY 2020"), ChromaDex reported net sales of $59.3 million, up 28% compared to $46.3 million in the full year ended December 31, 2019 ("FY 2019"). The increase in FY 2019 revenues was driven by growth in sales of Tru Niagen.

Gross margin percentage improved by 390 basis points to 59.5% in FY 2020 compared to 55.7% in FY 2019. The improvement in gross margin percentage was driven by the positive impact of increased Tru Niagen consumer product sales and product cost savings initiatives.

Operating expenses decreased by $1.9 million to $55.1 million in FY 2020, compared to $57.1 million in FY 2019. The decrease in operating expenses was driven by a decrease of $3.9 million in general and administrative expense, and a decrease of $0.7 million of R&D expense, partially offset by $2.7 million of higher selling and marketing expense. The decrease in general and administrative expense was driven by lower legal costs of $2.7 million and the absence of $2.2 million of bad debt expense related to the write-off of an Elysium receivable in 2019.

The net loss for FY 2020 was ($19.9) million or ($0.33) per share as reported compared to a net loss of ($32.1) million or ($0.56) per share for FY 2019 as reported. For FY 2020, the reported loss was negatively impacted by a non-cash charge of $6.9 million related to stock-based compensation.

Adjusted EBITDA excluding total legal expense, a non-GAAP measure, was a loss of ($1.0) million for FY 2020, compared to a loss of ($8.8) million for FY 2019, a $7.8 million improvement. The $7.8 million improvement was primarily driven by higher sales and gross margins, partially offset by higher marketing and selling expense.

For full year 2020, the net cash used in operating activities was ($10.6) million versus ($20.4) million for the same period in the prior year. The Company ended the year of 2020 with cash of $16.7 million.

2021 Outlook

Looking forward, the Company expects continued, steady revenue growth driven by its global ecommerce business, as well as growth with existing and new strategic partners. The Company expects continued gross margin improvement to slightly better than 60%, and roughly flat selling and marketing and R&D expense as a percentage of net sales year-over-year. The Company expects slightly higher general and administrative expense, excluding severance, restructuring and legal expense. The Company plans to increase investments and resources to drive brand awareness and accelerate its R&D pipeline to capitalize on growth in the NAD+ market globally.

Investor Conference Call

ChromaDex management will host an investor conference call to discuss the fourth quarter results and provide a general business update on Wednesday, March 10, at 4:30pm ET.

On March 10, 2021 DiaMedica Therapeutics Inc. (Nasdaq: DMAC), a clinical-stage biopharmaceutical company focused on developing novel treatments for neurological disorders and kidney diseases, reported a business update and financial results for the year ended December 31, 2020 (Press release, DiaMedica, MAR 10, 2021, View Source [SID1234576388]). DiaMedica will host a conference call Thursday, March 11, 2021, at 7:00 a.m. Central Time to discuss its business update and full year financial results.

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Clinical Developments

DM199 for the Treatment of Acute Ischemic Stroke

Productive Type B Meeting with the FDA

Initiation of Phase 2/3 Trial in AIS

The U.S. Food and Drug Administration (FDA) accepted DiaMedica’s request for a Type B Pre-IND meeting request related to its development plan for its product candidate, DM199, in the treatment of acute ischemic stroke (AIS). In written responses to the questions submitted by DiaMedica, the FDA agreed with DiaMedica’s proposals regarding key elements of a Phase 2/3 trial for DM199 in patients with AIS. These plans include an adaptive trial design with a primary endpoint based upon the modified Rankin Scale (mRS) at day 90.

The FDA also acknowledged that, provided the study results qualify, a single trial may support a Biologics License Application (BLA) submission. Additionally, based upon the clinical and preclinical testing performed to date and currently in process, the FDA did not require any additional studies in preparation for an investigational new drug (IND) submission and initiation of the Company’s planned Phase 2/3 trial.

DiaMedica believes that the feedback received from the FDA provided a well-defined regulatory pathway and is proceeding with the preparation of an IND submission to initiate a Phase 2/3 adaptive trial design which will include an interim analysis to allow for an increase in the sample size, if necessary, to improve powering.

DiaMedica plans to submit the IND later this month for its proposed Phase 2/3 AIS trial and initiate it this summer. DiaMedica expects the trial will be a double blinded, placebo controlled, randomized study of approximately 350 participants, based on a 90% powering for statistical significance on the primary endpoint of mRS at day 90. Secondary endpoints will include stroke recurrence, mRS shift, NIHSS and Barthel Index, deaths, safety and tolerability measures, and biomarkers relating to KLK1. The Company also plans to discuss with the FDA the addition of a sub-study for proof of efficacy in reducing stroke recurrence based upon the statistically significant reduction in severe recurrent strokes observed in its ReMEDy Phase 2 AIS trial.

The results of the Company’s ReMEDy Phase 2 trial in AIS will be the subject of oral and poster presentations at the upcoming International Stroke Conference 2021 being held virtually on March 17-19, 2021.

The Company will also be hosting a Stroke KOL webinar on the Treatment of Acute Ischemic Stroke on March 19th at noon Eastern Time. To register for the webinar, please click here.

"We are very pleased with the progress we’ve made in recent weeks advancing our DM199 program for AIS patients," said Rick Pauls, President and Chief Executive Officer of DiaMedica. "Our Phase 2/3 trial builds on statistically and clinically significant results from our ReMEDy trial, and we’re committed to designing and executing a study that meets the qualifications for and could provide evidence to support a BLA submission."

DM199 for the Treatment of Chronic Kidney Disease

Completion of Enrollment in CKD Caused by Type II Diabetes Cohort; Data Read-out in Q2 2021

Enrollment Continues in IgA Nephropathy and CKD in African Americans with Hypertension

DiaMedica’s Phase 2 REDUX (Latin for restore) trial is a multi-center, open-label, investigation to assess the safety and efficacy of multiple doses of DM199, administered over 90 days, in participants with chronic kidney disease (CKD) (Stage 2 or 3) targeting enrollment of approximately 90 participants in three equal Cohorts. Cohort 1 of the study is focused on non-diabetic, African Americans with hypertension, a group that is at greater risk for CKD than Caucasians. Additionally, the study is designed to identify African American participants with the APOL1 gene mutation as an exploratory biomarker as these individuals have an even higher risk of developing CKD. Cohort 2 of the study is focused on participants with IgA Nephropathy (IgAN) and Cohort 3 includes participants with diabetic kidney disease (DKD).

As of March 1, 2020, DiaMedica had enrolled a total of 68 subjects, including a fully enrolled DKD Cohort, and approximately 70% of the IgAN and 50% of the African American Cohorts. The Company has continued to experience slower than expected enrollment in the first two Cohorts of the REDUX trial due to the COVID-19 pandemic. However, with the significant declines in new COVID-19 cases and the anticipated availability and effectiveness of vaccines, the Company currently anticipates completion of Cohort 1 and Cohort 2 in the second half of 2021. Preliminary topline results from the DKD Cohort are expected to be available in the second quarter of 2021.

"We look forward to reporting upcoming topline results from our Phase 2 REDUX trial," commented Dr. Harry Alcorn, Jr., DiaMedica’s Chief Medical Officer. "While we intend to follow the data from all three Cohorts, we anticipate that for CKD, we will have the opportunity to initially position D199 in patients with IgA Nephropathy, an orphan drug indication, and in the longer term provide a treatment option to the overall CKD and DKD patient populations."

Strengthening the Balance Sheet

During 2020, the Company completed two underwritten public offerings of its common shares receiving gross proceeds of $31.5 million, and net proceeds of $28.8 million, after deducting the underwriting discount and estimated offering expenses. DiaMedica ended 2020 with $27.5 million in cash, cash equivalents and marketable securities which should provide sufficient capital to fund the Company’s operations through mid-2022 and which the Company believes will allow it to complete the REDUX trial and initiate its Phase 2/3 trial in AIS.

Financial Results

Research and development (R&D) expenses were $8.3 million for the year ended December 31, 2020 compared to $7.9 million for the year ended December 31, 2019, an increase of $0.4 million. The increase was primarily due to a combination of costs incurred for the REDUX Phase 2 CKD study initiated late in 2019, driven in particular by the addition of Cohort 3 targeting participants with DKD, which fully enrolled during the fourth quarter of 2020, and increased non-cash share-based compensation costs. These increases were partially offset by decreases in clinical study costs incurred for the ReMEDy stroke study, which wound down in the first half of 2020, and non-recurring costs of the Phase 1b CKD study which was started and completed in the prior year period. Additionally, there was a year over year net decrease in drug manufacturing and development costs.

General and administrative (G&A) expenses were $4.4 million and $3.7 million for the years ended December 31, 2020 and 2019, respectively. This $0.7 million increase was primarily due to increased non-cash share-based compensation costs, outside professional services and directors and officers’ liability insurance. The increase in 2020 was partially offset by reduced travel and meeting costs primarily due to the COVID-19 pandemic.

Total other income, net was $0.4 million for the year ended December 31, 2020 compared to $1.0 million for 2019. This decrease was driven primarily by reduced R&D incentives receivable from the Australian Government, paid for qualifying research work performed by DiaMedica’s Australian subsidiary during 2020, related to the decreased ReMEDy stroke study costs. This decrease was partially offset by increased foreign currency transaction gains recognized during 2020.

Balance Sheet and Cash Flow

The Company had cash, cash equivalents and marketable securities of $27.5 million, current liabilities of $2.0 million and working capital of $25.9 million as of December 31, 2020, compared to $7.9 million in cash, cash equivalents and marketable securities, $1.3 million in current liabilities and $7.5 million in working capital as of December 31, 2019. The increases in the Company’s combined cash, cash equivalents and marketable securities and in its working capital are due primarily to the Company’s February and August 2020 public offerings.

Net cash used in operating activities was $9.2 million for the year ended December 31, 2020, compared to $9.1 million for the year ended December 31, 2019. This slight increase relates primarily to the combination of the increase in the net loss, partially offset by an increase in non-cash share-based compensation.

Conference Call Information

DiaMedica Management will host a conference call to discuss its 2020 financial results and business update on Thursday, March 11 2021, at 7:00 a.m. Central Time:

Interested parties may access the conference call by dialing in or listening to the simultaneous webcast. Listeners should log on to the website or dial in 15 minutes prior to the call. The webcast will remain available for play back on DiaMedica’s website, under investor events and presentations, following the earnings call and for 12 months thereafter. A telephonic replay of the conference call will be available until March 18, 2021, by dialing (855) 859-2056 (US Toll Free), (404) 537-3406 (International), replay passcode 9297319.

About DM199

DM199 is a recombinant (synthetic) form of human tissue kallikrein-1 (KLK1). KLK1 is a serine protease (protein) that plays an important role in the regulation of diverse physiological processes including blood flow, inflammation, fibrosis, oxidative stress and neurogenesis via a molecular mechanism that increases production of nitric oxide and prostaglandin. KLK1 deficiency may play a role in multiple vascular and fibrotic diseases such as chronic kidney disease, retinopathy, stroke, vascular dementia, and resistant hypertension where current treatment options are limited or ineffective. DiaMedica is the first company to have developed a recombinant form of the KLK1 protein. The KLK1 protein, produced from porcine pancreas and human urine, has been used to treat patients in Japan, China and Korea for decades. DM199 is currently being studied in patients with chronic kidney disease and patients with acute ischemic stroke.