On March 10, 2021 Arch Oncology, Inc., a clinical-stage immuno-oncology company focused on the discovery and development of anti-CD47 antibody therapies, reported the presentation of new preclinical data on AO-176 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021 (Press release, Arch Oncology, MAR 10, 2021, View Source;utm_medium=rss&utm_campaign=arch-oncology-announces-two-new-preclinical-data-presentations-on-highly-differentiated-anti-cd47-antibody-ao-176-at-aacr-2021 [SID1234576405]). AO-176 is an anti-CD47 antibody with a potential best-in-class profile that works by blocking the "don’t eat me" signal and also by directly killing tumor cells, with preferential binding to tumor versus normal cells. Currently, AO-176 is being evaluated in Phase 1/2 clinical trials for the treatment of patients with select solid tumors and multiple myeloma, both as monotherapy and in combination with standard therapies.

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"At AACR (Free AACR Whitepaper) this year, we will present two new nonclinical datasets demonstrating AO-176’s strong therapeutic potential in lymphoma and pediatric T-ALL" said Daniel Pereira, Ph.D., Chief Scientific Officer of Arch Oncology. "In lymphoma, we are presenting AO-176’s potent in vivo activity in a lymphoma xenograft, where induction of inflammatory cytokines and immune infiltrates is also observed. Enhanced binding and phagocytosis of lymphoma cells at acidic pH will also be shown in vitro by AO-176 or in combination with rituximab. Finally, AO-176’s unique ability to induce Annexin V positivity and DAMPs that ultimately may aid in inducing immunogenic cell death of the lymphoma cells will also be presented. Additionally, we look forward to sharing new nonclinical data in pediatric T-ALL during a late-breaking poster presentation at AACR (Free AACR Whitepaper) next month. Data continue to support AO-176’s highly-differentiated mechanisms, reinforcing our therapy’s potential to offer an improved efficacy and safety profile among anti-CD47 agents in development for patients with solid tumors and hematologic malignancies."

AACR Annual Meeting 2021

Late-Breaking Poster Presentation Title: The differentiated CD47 monoclonal antibody AO-176 exhibits significant in vivo activity against xenograft models of pediatric acute lymphoblastic leukemia (ALL) (Abstract #LB171)
Session Category: Immunology
Session Title: Therapeutic Antibodies, Including Engineered Antibodies

Poster Presentation Title: AO-176, a highly differentiated clinical stage anti-CD47 antibody, is efficacious in pre-clinical models of lymphoma (Abstract #954)
Session Category: Experimental and Molecular Therapeutics
Session Title: Biological Therapeutic Agents

Information on the abstracts is available on AACR (Free AACR Whitepaper)’s website.

On Saturday, April 10, 2021 at 8:30 am ET when posters are available online for AACR (Free AACR Whitepaper) meeting participants, a copy of the poster presentations will be posted at View Source

About AO-176

AO-176 is a humanized anti-CD47 IgG2 antibody with a potential best-in-class profile. AO-176 is highly differentiated, with the potential to improve upon the safety and efficacy profile relative to other agents in this class of innate checkpoint inhibitors. AO-176 works by blocking the "don’t eat me" signal, the standard mechanism of anti-CD47 antibodies. Beyond blocking this signal, AO-176 has additional mechanisms, including directly killing tumor cells and inducing DAMPs (Damage Associated Molecular Patterns), resulting in Immunogenic Cell Death. Importantly, AO-176 binds preferentially to tumor cells, instead of to normal cells, and binds even more potently to tumors in their acidic microenvironment (low pH). Publications and presentations on AO-176 can be found at View Source

AO-176 is being evaluated in Phase 1/2 clinical trials for the treatment of patients with select solid tumors and multiple myeloma, both as monotherapy and in combination with standard therapies. In a Phase 1 trial in solid tumors, AO-176 demonstrated encouraging safety and evidence of anti-tumor activity when administered as a single agent. Additional information about these trials may be found at www.clinicaltrials.gov using the trial identification number NCT03834948 (solid tumors) or NCT04445701 (multiple myeloma).

On March 10, 2021 Medidata, a Dassault Systèmes company and the global leader in creating end-to-end solutions to support the entire clinical development process, reported its expanded partnership with Karyopharm Therapeutics (NASDAQ: KPTI) (Press release, Karyopharm, MAR 10, 2021, View Source [SID1234576421]). In 2014, Karyopharm selected one Medidata technology solution for a single study. Today, the company is leveraging 10 solutions in Medidata Rave Clinical Cloud in more than 15 clinical studies focused on hematologic malignancies and solid tumors.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"Medidata is pleased to play a key role in supporting Karyopharm’s mission of bringing novel therapies to market, providing hope for patients," said Glen de Vries, co-founder and co-CEO, Medidata. "This agreement is a clear demonstration of our shared mission to advance analytics and technology to make a difference in health care."

With Medidata solutions, Karyopharm is able to:

Centralize operations, eliminate manual data entry, and operate with a clear view of all cross-application data in one place
Simplify and customize reporting within or across studies and leverage over 30 standard reports
Reduce complexity by standardizing and improving data quality with powerful artificial intelligence and machine learning algorithms that automatically manage the complexity of clinical data
"Medidata continues to be an important strategic partner for Karyopharm by providing a cutting-edge technological infrastructure that helps us reach our clinical trial goals," said Kristan Gallitano, senior vice president, Development Operations at Karyopharm. "Medidata provides us the flexible, scalable technology and support we need to meet the evolving challenges of drug development."

Medidata is a wholly owned subsidiary of Dassault Systèmes, which with its 3DEXPERIENCE platform is positioned to lead the digital transformation of life sciences in the age of personalized medicine with the first end-to-end scientific and business platform, from research to commercialization.

On March 10, 2021 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer, reported that an abstract for CA-4948, a small molecule IRAK4 inhibitor, has been accepted for poster presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, which will be held virtually from April 10-15, 2021 (Press release, Curis, MAR 10, 2021, View Source [SID1234576439]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Details of the presentation are as follows:

Poster Presentation

Title: Identification of NF-kappaB phospho-p50 as a Potential Predictive Biomarker for IRAK4 inhibitor CA-4948 in Patients with Non-Hodgkin’s Lymphoma
Session Name: Biomarkers Predictive of Therapeutic Benefit
Session Date: Saturday, April 10, 2021
Additional meeting information can be found on the AACR (Free AACR Whitepaper) website View Source The presentation will also be available under "Events and Presentations" in the Investors section of the Company’s website at www.curis.com.

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 Veracyte, Inc. (Nasdaq: VCYT) reported the U.S. Preventive Services Task Force (USPSTF) for its new, revised recommendations that expand eligibility for lung cancer screening (Press release, Veracyte, MAR 10, 2021, View Source [SID1234576406]). The updated recommendations lower the age for current and former smokers to begin screening from 55 to 50 years and reduces smoking intensity – from a 30 "pack-year" history to 20. The independent expert panel’s final recommendations appear online in the Journal of the American Medical Association and are expected to increase the number of people in the United States who are eligible for annual screening with low dose CT (LDCT) scans to nearly 15 million.

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"We commend the USPSTF for their new lung cancer screening recommendations, which will help ensure that more lives are saved through early detection," said Bonnie Anderson, Veracyte’s chairman and chief executive officer. "Lung cancer is the leading cause of cancer death in the United States because too often the disease is found after it has spread and is less treatable. Expanding screening eligibility – as well as the number of people who actually undergo screening – will be key to saving lives.

"Much work remains in the fight against lung cancer, including ensuring that physicians have optimal tools to help guide patient care when potentially cancerous lung nodules are found through screening. We are proud to be part of the healthcare sector that is utilizing novel genomic science to improve lung cancer early detection, diagnosis and treatment."

Veracyte’s Percepta Genomic Sequencing Classifier is available to improve the diagnosis of potentially cancerous lung nodules found on CT scans. The company is developing a first-of-its-kind, noninvasive nasal swab test to help determine which patients with lung nodules should undergo additional diagnostic procedures and which can simply be monitored. Additionally, the company is developing a comprehensive genomic profiling test to inform treatment decisions at the time of diagnosis. Both tests are scheduled for introduction in the second half of 2021.

About Lung Cancer

Lung cancer is the deadliest cancer globally, killing more than 1.75 million people worldwide each year, according to the World Health Organization. Early detection is key, with a five-year survival rate of nearly 60 percent when the cancer is found early, compared to six percent when it is found at a later stage, according to the American Lung Association. Lung nodules are typically the first sign of lung cancer. While the vast majority of lung nodules ultimately prove to be benign, physicians currently lack clear diagnostic tools to determine which patients have cancer and which do not. This can lead to unnecessary invasive biopsies, which are costly and risky, as well as to delayed diagnosis and treatment.