On May 6, 2021 Celldex Therapeutics, Inc. (NASDAQ:CLDX) reported financial results for the first quarter ended March 31, 2021 and provided a corporate update (Press release, Celldex Therapeutics, MAY 6, 2021, View Source [SID1234579435]).

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"We are very encouraged by the recently reported positive interim results from our ongoing Phase 1b study of CDX-0159 in chronic inducible urticaria. These data demonstrated an 80% complete response rate and a well-tolerated safety profile, which we believe is a significant accomplishment in this complex disease setting with limited treatment options," said Anthony Marucci, Co-founder, President and Chief Executive Officer of Celldex Therapeutics. "Based on these exciting results, we recently amended the protocol to add a third cohort in cholinergic urticaria, a debilitating disease affecting mainly young adults, where passive or active sweating induces hives and can severely impair their quality of life. We are nearing completion of patient dosing across both the cold induced and symptomatic dermographism cohorts from this study and look forward to presenting updated results from these cohorts this summer."

Mr. Marucci continued, "We are also making progress advancing our bispecific platform, presenting promising preclinical data at AACR (Free AACR Whitepaper) that supports the development of clinical bispecific candidates that co-target ILT4 and PD-(L)1. We look forward to continuing these efforts while also exploring important targets controlling inflammation and auto-immune pathways."

Recent Pipeline Highlights

CDX-0159 – KIT Inhibitor Program

CDX-0159 is a humanized monoclonal antibody developed by Celldex that binds the KIT receptor with high specificity and potently inhibits its activity. The KIT receptor tyrosine kinase is expressed in a variety of cells, including mast cells, which mediate inflammatory responses such as hypersensitivity and allergic reactions. KIT signaling controls the differentiation, tissue recruitment, survival and activity of mast cells.

Celldex initiated a Phase 1b open label study designed to evaluate the safety of a single dose (3.0 mg/kg) of CDX-0159 administered intravenously in December of 2020. Up to 20 patients with cold contact urticaria (ColdU; n=10) or symptomatic dermographism (SD; n=10) who are refractory to antihistamines are being enrolled. Patients’ symptoms are induced via provocation testing that resembles real life triggering situations. Secondary and exploratory objectives include pharmacokinetic and pharmacodynamic assessments, including changes from baseline provocation thresholds, measurement of tryptase and stem cell factor levels, clinical activity outcomes (impact on urticaria symptoms, disease control, clinical response), quality of life assessments and measurement of tissue mast cells through skin biopsies. The study is being conducted by Dr. Marcus Maurer, Professor of Dermatology and Allergy at Charité – Universitätsmedizin in Berlin.

Interim data from this study were reported in late March. Fifteen out of 20 planned patients with antihistamine refractory CIndU had received a single intravenous infusion of CDX-0159 at 3 mg/kg, including nine patients with ColdU and six patients with SD. Safety results were reported for all 15 patients; activity results were reported for all patients assessed for at least 15 days/2 weeks after treatment (n=10; 7 ColdU and 3 SD). Patients had high disease activity as assessed by provocation threshold testing.

Eight of 10 patients, including all patients with ColdU, (7 ColdU; 1 SD) experienced a complete response (CR) as assessed by provocation threshold testing. One patient experienced a partial response (PR). All patients will continue to be assessed for response through week 12. CDX-0159 was generally well tolerated.
Enrollment is nearing completion in the ColdU and SD cohorts. Based on these compelling results, the study has been expanded to also include 10 patients with cholinergic urticaria and patient screening is expected to begin this month.
Updated results from additional patients with cold induced urticaria and symptomatic dermographism and long term follow up that continues to characterize magnitude and duration of treatment effect and their link to changes in tryptase levels are expected this summer.
Celldex initiated dosing in a Phase 1b multi-center study of CDX-0159 in chronic spontaneous urticaria (CSU) in October. This study is a randomized, double-blind, placebo-controlled clinical trial designed to assess the safety of multiple ascending doses of CDX-0159 in up to 40 patients with CSU who remain symptomatic despite treatment with antihistamines. Secondary and exploratory objectives include pharmacokinetic and pharmacodynamic assessments, including measurement of tryptase and stem cell factor levels and clinical activity outcomes (impact on urticaria symptoms, disease control, clinical response) as well as quality of life assessments. Results from the study are expected by the end of 2021.

CDX-0159 development is being expanded into prurigo nodularis (PN), a chronic skin disease characterized by the development of hard, intensely itchy (pruritic) nodules on the skin. Initiation of this study is planned for the fourth quarter of 2021.

Manufacturing activities are also progressing as planned to support the introduction of the CDX-0159 subcutaneous formulation into the clinical program in the third quarter of 2021.
CDX-1140 – CD40 Agonist Program

CDX-1140 is a potent CD40 human agonist antibody developed by Celldex that the Company believes has the potential to successfully balance systemic doses for good tissue and tumor penetration with an acceptable safety profile.

In the Phase 1 study of CDX-1140 in patients with recurrent, locally advanced or metastatic solid tumors and B cell lymphomas, both the monotherapy and combination with CDX-301 portions of the trial are complete. Expansion cohorts are actively recruiting including:

CDX-1140 with KEYTRUDA (pembrolizumab) in patients with squamous cell head and neck cancer and non small cell lung cancer who have progressed on checkpoint therapy; and,
CDX-1140 with standard of care chemotherapy in first line metastatic pancreatic cancer.

Updated data from this program are expected to be presented later this year.
CDX-527 – Bispecific Antibody Program

CDX-527 is the first candidate developed by Celldex from its bispecific platform which utilizes the Company’s proprietary highly active anti-PD-L1 and CD27 human antibodies to couple CD27 co-stimulation with blockade of the PD-L1/PD-1 pathway.

In August 2020, Celldex initiated a Phase 1 dose-escalation study in up to ~40 patients with advanced or metastatic solid tumors that have progressed during or after standard of care therapy to be followed by tumor-specific expansion cohorts. The study is designed to determine the MTD during a dose-escalation phase and to recommend a dose level for further study in the subsequent expansion phase. The expansion is designed to further evaluate the tolerability, and biologic and anti-tumor effects of selected dose level(s) of CDX-527 in specific tumor types. Initial data from the Phase 1 study focused on pharmacokinetic and pharmacodynamic properties have been accepted for presentation at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting to be held June 4th-8th.
Preclinical Programs

In April 2021, Celldex presented promising data from the Company’s growing bispecific platform at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. The Company described the discovery and characterization of ILT4 inhibitory monoclonal antibodies (mAbs) for engineering bispecific antibodies (bsAbs) that revert myeloid cell suppression by antagonizing ILT4 and activate T-cell responses through PD-(L)1 inhibition. Based on the results, Celldex is developing clinical bispecific candidates that co-target ILT4 and PD-(L)1. Celldex is also exploring important targets controlling inflammation and auto-immune pathways.

In direct support of our bispecific platform, Celldex has entered into a research and collaboration agreement with Biosion, Inc., a global biotechnology company focused on the discovery and clinical development of innovative biologicals for unmet medical needs, to construct and develop bispecific antibodies that combine undisclosed components from both Celldex and Biosion discoveries.
While Celldex’s clinical development programs have not been significantly, negatively impacted by COVID-19 to date, the Company continues to carefully monitor the evolving situation closely across all development programs and work to minimize potential impact/disruptions.

First Quarter 2021 Financial Highlights and 2021 Guidance

Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2021 were $176.1 million compared to $194.4 million as of December 31, 2020. The decrease was primarily driven by first quarter cash used in operating activities of $18.1 million. At March 31, 2021, Celldex had 39.6 million shares outstanding.

Revenues: Total revenue was $0.7 million in the first quarter of 2021 compared to $2.7 million for the comparable period in 2020. The decrease in revenue was primarily due to the $1.8 million milestone payment received from Rockefeller University in the first quarter of 2020 related to Celldex’s manufacturing and development services agreement.

R&D Expenses: Research and development (R&D) expenses were $12.7 million in the first quarter of 2021 compared to $11.7 million for the comparable period in 2020. The increase in R&D expenses was primarily due to an increase in contract research and clinical trial expenses.

G&A Expenses: General and administrative (G&A) expenses were $4.1 million in the first quarter of 2021 compared to $3.7 million for the comparable period in 2020. The increase in G&A expenses was primarily due to higher stock-based compensation expense.

Changes in Fair Value Remeasurement of Contingent Consideration: During the quarter ended March 31, 2021, the Company recorded a $0.5 million loss on fair value remeasurement of contingent consideration primarily due to changes in discount rates and the passage of time.

Net Loss: Net loss was $16.5 million, or ($0.42) per share, for the first quarter of 2021 compared to a net loss of $12.6 million, or ($0.73) per share, for the comparable period in 2020.

Financial Guidance: Celldex believes that the cash, cash equivalents and marketable securities at March 31, 2021 are sufficient to meet estimated working capital requirements and fund planned operations through 2023.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ USA.

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On May 6, 2021 Clarity Pharmaceuticals, a clinical stage radiopharmaceutical company focused on the treatment of serious disease, reported that the U.S. Patent and Trademark Office (USPTO) has granted the patent application for Clarity’s SAR-bisPSMA compound and its variants (Press release, Clarity Pharmaceuticals, MAY 6, 2021, View Source [SID1234579259]). The grant of the patent application bolsters Clarity’s strong Intellectual Property (IP) position on the Prostate Specific Membrane Antigen (PSMA) targeting agent for imaging and treatment of prostate cancer.

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SAR-bisPSMA is the result of Clarity’s optimisation of the well characterised PSMA targeting peptide, which has been in many thousands of patients to date, by combining two PSMA targeting peptides with Clarity’s proprietary SAR Technology and the theranostic isotopes of copper, copper-64 for imaging and copper-67 for therapy. The copper pairing enables diagnosis, staging and subsequent treatment of prostate cancers that express PSMA. The optimisation of the targeting peptide has resulted in superior targeting of, and retention in, tumours compared to a singular PSMA targeting peptide in preclinical models (Zia et al., 2019)1. The product was developed through the long-standing collaboration between Clarity and Professor Paul Donnelly at the University of Melbourne.

Clarity’s Executive Chairman, Dr Alan Taylor, commented: "The grant of the SAR-bisPSMA patent application is an example of the effectiveness of Clarity’s broad patent strategy around its SAR Technology, which allows the filing of additional patents with a wide range of new disease targeting agents."

The news from the USPTO come shortly after Clarity has completed the assignment of its key patent portfolio from the University of Melbourne, providing Clarity with the full rights and ownership of the patents.

"The grant of the SAR-bisPSMA patent application also comes soon after Clarity received a response from the U.S. Food and Drug Administration on its an Investigational New Drug (IND) application for a theranostic trial of 64Cu/67Cu SAR-bisPSMA in the U.S. (NCT04868604)2 that the study may proceed. With our diagnostic trial of 64Cu SAR-bisPSMA in previously diagnosed but yet untreated patients prior to surgical removal of the prostate in the start-up phase (NCT04839367)3, we are now ready to embark on two clinical trials of SAR-bisPSMA in the next few months," said Dr Taylor.

Prostate cancer is the second most common cancer diagnosed in men globally and the fifth leading cause of death worldwide4. The American Cancer Society estimates in 2021 there will be 248,530 new cases of prostate cancer in the U.S. and around 34,130 deaths from the disease5. For metastatic prostate cancer, the 5-year relative survival rate is 30%, indicating a high unmet need for early detection and better treatment options for mCRPC. Annually, there are around ~34,000 men in the U.S. who are diagnosed with mCRCP5, ~90% of whom have tumours which express PSMA6.

Clarity’s Executive Chairman, Dr Alan Taylor, commented, "The theranostic SAR-bisPSMA product has blockbuster market potential for prostate cancer. The product has ideal characteristics for a radiopharmaceutical and numerous advantages associated with the optimisation of the targeting peptide and using the copper pairing, including increased targeting and retention of the product in tumours, centralised manufacture, reaching more treatment sites and patients around the world, as well as diagnostic, dosimetry and therapeutic benefits. We are excited to progress the development of this proprietary asset into the clinic for the treatment of men with prostate cancer as part of our ultimate goal of improving treatment outcomes for kids and adults with cancer. We are pleased to have received the patent grant to reinforce Clarity’s IP position around our PSMA compounds, giving us confidence in the commercialisation process moving forward."

On May 6, 2021 CRISPR Therapeutics (NASDAQ: CRSP), a biopharmaceutical company focused on developing transformative gene-based medicines for serious diseases, and Nkarta, Inc. (NASDAQ: NKTX), a biopharmaceutical company developing engineered NK cell therapies to treat cancer, reported a strategic partnership to research, develop, and commercialize CRISPR/Cas9 gene-edited cell therapies for cancer (Press release, Nkarta, MAY 6, 2021, View Source [SID1234579318]).

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Under the agreement, the companies will co-develop and co-commercialize two CAR NK cell product candidates, one targeting the CD70 tumor antigen and the other target to be determined. In addition, the companies will bring together their complementary cell therapy engineering and manufacturing capabilities to advance the development of a novel NK+T product candidate harnessing the synergies of the adaptive and innate immune systems. Finally, Nkarta obtains a license to CRISPR gene editing technology to edit five gene targets in an unlimited number of its own NK cell therapy products.

CRISPR Therapeutics and Nkarta will equally share all research and development costs and profits worldwide related to the collaboration products. For each non-collaboration product candidate incorporating a gene editing target licensed from CRISPR Therapeutics, Nkarta will retain worldwide rights and pay CRISPR Therapeutics milestones and royalties on net sales. The agreement includes a three-year exclusivity period between CRISPR Therapeutics and Nkarta covering the research, development, and commercialization of allogeneic, gene-edited, donor-derived NK cells and NK+T cells.

"By bringing together CRISPR Therapeutics’ and Nkarta’s highly complementary expertise and proprietary platforms we plan to accelerate the development of potentially groundbreaking genome engineered NK cell therapies," said Samarth Kulkarni, Ph.D., Chief Executive Officer at CRISPR Therapeutics. "This collaboration broadens the scope of our efforts in oncology cell therapy, and expands our efforts to discover and develop novel cancer therapies for patients."

"Uniting the best-in-class gene editing solution and allogeneic T cell therapy expertise of CRISPR with Nkarta’s best-in-class CAR NK cell therapy platform will be a major advantage to advancing the next wave of transformative cancer cell therapies," said Paul J. Hastings, President and Chief Executive Officer of Nkarta. "With this partnership, Nkarta can systematically apply world-class gene editing across our entire pre-clinical pipeline going forward. CRISPR’s deep understanding of CD70 biology and experience in allogeneic T cell clinical development can accelerate the development of early-stage Nkarta programs, to deliver innovative treatments to patients that much faster."

Nkarta Conference Call Details

Nkarta management will host a conference call to discuss the collaboration today at 4:30 p.m. Eastern Time (ET). The event will be simultaneously webcast and available for replay from the Nkarta website at www.nkartatx.com, under the Investors section. Investors may also participate in the conference call by calling 877-876-9174 (domestic) or +1-785-424-1669 (international). The conference ID is NKARTA.

On May 6, 2021 NantHealth, Inc. (NASDAQ-GS: NH), a provider of enterprise solutions that help businesses transform complex data into actionable insights, reported financial results for its first quarter ended March 31, 2021 (Press release, NantHealth, MAY 6, 2021, View Source [SID1234579347]).

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"Last month, we successfully completed a $137.5 million financing transaction and will use a portion of the proceeds to retire our existing convertible notes," said Ron Louks, Chief Operating Officer, NantHealth. "We intend to use the balance of the proceeds to, among other things, support our growth initiatives, which include further developing our existing SaaS offerings, expanding our recently acquired OpenNMS business and investing in our data services and cloud capabilities.

"Turning to our 2021 first-quarter financial results, as expected revenues declined largely due to the accounting for amortization of services related to expired or renewed contracts at the end of the 2020 fourth quarter. While we remain committed to managing our overhead costs, we have continued the ramp up of our investment in research and development to improve and expand the innovative solutions we bring to our customers.

"Looking ahead, we see growing opportunities to expand and diversify our business within and beyond healthcare. Earlier this week, we launched Eviti Connect for autoimmune diseases, extending our proven payer solution beyond oncology, and announced plans to further develop the Eviti platform to allow us to expand into additional disease states, over time. In addition, we are expanding OpenNMS offerings to include a new cloud-based platform to supplement our already robust on-premise platform, which will both broaden the solutions we offer to our healthcare customers and attract new customers across a variety of industries."

Software and Services Highlights:

Clinical Decision Support (Eviti):
In May, launched Eviti Connect for autoimmune diseases, bringing the Company’s payer solution for oncology to a disease type that affects more than 50 million in the U.S. alone. The Company developed a new, flexible platform that will enable it to expand into disease areas beyond oncology and autoimmune
Launched Eviti Connect 8.4, which included:
New functionality enables drug customization per payer or line of business providing more flexibility to meet diverse policies within the payer organization
New payer communication capability that reduces delays in treatment plan reviews
In May, Eviti Connect won the MedTech Breakthrough award for "Best MedTech Overall Software," recognized for the platform’s innovative ability to break through digital health and technology markets
Payer Engagement (NaviNet and Population Health Management):
Continued customer renewals, securing three multi-year agreements in Q1 with over $700K of annual contract value
AllPayer, the Company’s direct-to-provider solution, had its seventh consecutive quarter of growth
Enhanced NaviNet essential workflows to support automation and promote greater usability
Open Authorizations now allows health plans to configure rules defining which services do not require prior authorization. This increases transparency for providers and saves time by eliminating unnecessary requests
New Referral Submission API enables providers to automate management of referral requests through NaviNet, making it easier to guide patients to optimal quality and cost referral sites
Announced Population Health Management, a new product offering that integrates multiple data sources into one platform, providing a more complete view of the patient. This enables payers and providers to engage proactively with patients, increasing the quality of care while reducing costs
Network Monitoring and Management (OpenNMS):
Deployed a solution for a global energy customer that scales the monitoring and analysis of network communications by aggregating the data streams (netflow) to greatly improve scalability and usability of the solution
Initiated a Zero-touch Appliance service pilot with a large national retailer
Business and Financial Highlights

For the 2021 first quarter:

Total net revenue was $16.2 million compared with $18.2 million in Q1 of 2020. Within total revenue, total software-related revenue was $16.2 million compared with $18.1 million in prior-year first quarter.
Gross profit was $9.1 million, or 56% of total net revenue, compared with $11.0 million, or 60% of total net revenue, for the prior-year period.
Selling, general and administrative (SG&A) expenses increased to $12.5 million from $12.4 million in the 2020 first quarter.
Research and development (R&D) expenses increased to $5.0 million from $3.6 million.
Net loss from continuing operations attributable to NantHealth, net of tax, was $15.4 million, or $0.14 per share, compared with $8.9 million, or $0.08 per share, in the 2020 first quarter.
Non-GAAP net loss from continuing operations attributable to NantHealth was $9.6 million, or $0.09 per share, compared with $6.1 million, or $0.06 per share, for the first quarter of 2020.
At March 31, 2021, cash and cash equivalents totaled $10.8 million.
Conference Call Information and Forward-Looking Statements

Later today, the company will host a conference call at 1:30 p.m. PT (4:30 p.m. ET) to review its results of operations for the first quarter ended March 31, 2021. The conference call will be available to interested parties by dialing 844-309-3709 from the U.S. or Canada, or 281-962-4864 from international locations, passcode 8364209. The call will be broadcast via the Internet at www.nanthealth.com. Listeners are encouraged to visit the website at least 10 minutes prior to the start of the scheduled presentation to register, download and install any necessary audio software. A playback of the call will be archived and accessible on the same website for at least three months.

Discussion during the conference call may include forward-looking statements regarding topics such as the company’s financial status and performance, regulatory and operational developments, and other comments the company may make about its future plans or prospects in response to questions from participants on the conference call.

Use of Non-GAAP Financial Measures

This news release contains references to Non-GAAP financial measures, including adjusted net loss and adjusted net loss per share, which are financial measures that are not prepared in conformity with United States generally accepted accounting principles (U.S. GAAP). The Company’s management believes that the presentation of Non-GAAP financial measures provides useful supplementary information regarding operational performance, because it enhances an investor’s overall understanding of the financial results for the Company’s core business. Additionally, it provides a basis for the comparison of the financial results for the Company’s core business between current, past and future periods. Other companies may define these measures in different ways. Non-GAAP financial measures should be considered only as a supplement to, and not as a substitute for or as a superior measure to, financial measures prepared in accordance with U.S. GAAP. Non-GAAP per share numbers are calculated based on one class of common stock and do not incorporate the effects, if any, of using the two-class method.