On May 3, 2022 Orion’s collaboration partner Bayer reported the U.S. Food and Drug Administration (FDA) has accepted a supplemental New Drug Application (sNDA) and granted Priority Review for the oral androgen receptor inhibitor (ARi) darolutamide in combination with docetaxel for the treatment of metastatic hormone-sensitive prostate cancer (mHSPC) (Press release, Orion , MAY 3, 2022, View Source [SID1234613418]).

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The application is being conducted under the FDA Oncology Center of Excellence’s (OCE) Project Orbis initiative, which provides a framework for concurrent submission and review of cancer treatments among participating international health authorities.

The sNDA is based on positive results from the pivotal Phase III ARASENS trial demonstrating a statistically significant improvement in overall survival (OS) for darolutamide plus androgen deprivation therapy (ADT) and docetaxel in men with mHSPC compared to ADT plus docetaxel, which were published in The New England Journal of Medicine. Darolutamide is already approved in more than 60 markets around the world, including the U.S., the European Union (EU), Japan and China, under the brand name Nubeqa, for the treatment of patients with non-metastatic castration-resistant prostate cancer (nmCRPC), who are at high risk of developing metastatic disease.

Darolutamide is developed jointly by Orion and Bayer. Bayer recently submitted applications for additional indication of darolutamide to the European Medicines Agency (EMA), the Ministry of Health, Labor and Welfare (MHLW) in Japan, and China’s Center of Drug Evaluation (CDE). Additional submissions in mHSPC are planned globally by Bayer.

About the ARASENS Trial

The ARASENS trial is a randomized, Phase III, multi-center, double-blind, placebo-controlled trial which was prospectively designed to investigate the efficacy and safety of oral darolutamide, an androgen receptor inhibitor (ARi), plus androgen deprivation therapy (ADT) and the chemotherapy docetaxel in patients with metastatic hormone-sensitive prostate cancer (mHSPC). A total of 1,306 newly diagnosed patients were randomized in a 1:1 ratio to receive 600 mg of darolutamide twice a day or matching placebo, plus ADT and docetaxel.

The primary endpoint of this trial was overall survival (OS). Secondary endpoints included time to castration-resistant prostate cancer (CRPC), time to pain progression, time to first symptomatic skeletal event (SSE), time to initiation of subsequent anticancer therapy, all measured at 12‐week intervals, as well as adverse events (AEs) as a measure of safety and tolerability.

About Metastatic Hormone-Sensitive Prostate Cancer

Prostate cancer is the second most commonly diagnosed malignancy in men worldwide. In 2020, an estimated 1.4 million men were diagnosed with prostate cancer, and about 375,000 died from the disease worldwide.1

At the time of diagnosis, most men have localized prostate cancer, meaning their cancer is confined to the prostate gland and can be treated with curative surgery or radiotherapy. Upon relapse, when the disease will metastasize or spread, androgen deprivation therapy (ADT) is the cornerstone of treatment for this hormone-sensitive disease. Current treatment options for men with metastatic hormone-sensitive prostate cancer (mHSPC) include hormone therapy, such as ADT, androgen receptor pathway inhibitors plus ADT or a combination of docetaxel chemotherapy and ADT. Despite these treatments, a large proportion of men with mHSPC will eventually experience progression to metastatic castration-resistant prostate cancer (mCRPC), a condition with limited survival.

About darolutamide

Darolutamide is an oral androgen receptor inhibitor (ARi) with a distinct chemical structure that binds to the receptor with high affinity and exhibits strong antagonistic activity, thereby inhibiting the receptor function and the growth of prostate cancer cells. The low potential for blood-brain barrier penetration for darolutamide is supported by preclinical models and neuroimaging data in healthy humans. A low blood-brain barrier penetration would explain the overall low incidence of central nervous system (CNS)-related adverse events (AEs) compared to placebo as seen in the ARAMIS Phase III trial and the improved verbal learning and memory observed in the darolutamide arm of the Phase II ODENZA trial.

The product is approved under the brand name Nubeqa in more than 60 markets around the world, including the U.S., EU, Japan, China, for the treatment of patients with non-metastatic castration-resistant prostate cancer (nmCRPC), who are at high risk of developing metastatic disease. The compound is also being investigated in further studies across various stages of prostate cancer, including in the ARANOTE Phase III trial evaluating darolutamide plus androgen deprivation therapy (ADT) versus ADT alone for metastatic hormone-sensitive prostate cancer as well as the Australian and New Zealand Urogenital and Prostate Cancer Trials Group (ANZUP)-led international co-operative group Phase III trial, evaluating darolutamide as an adjuvant treatment for localized prostate cancer with very high risk of recurrence (DASL-HiCaP, ANZUP1801). Information about these trials can be found at www.clinicaltrials.gov.

On May 3, 2022 Sapience Therapeutics, Inc., a biotechnology company focused on the discovery and development of peptide therapeutics to address difficult-to-treat cancers, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead program, ST101, for the treatment of advanced melanoma for patients in stages IIB through IV (Press release, Sapience Therapeutics, MAY 3, 2022, View Source [SID1234613417]). This is the third orphan drug designation received for the ST101 program, following orphan designations by the FDA for the treatment of AML in April 2018 and for glioma in June 2020.

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ST101 is a first-in-class peptide antagonist of C/EBPβ currently being evaluated in the Phase 2 portion of an ongoing Phase 1-2 clinical study in patients with advanced unresectable and metastatic solid tumors that are refractory to standard therapy (NCT04478279). This study includes expansion cohorts that are currently dosing and enrolling patients with GBM, cutaneous melanoma, locally advanced or metastatic hormone-receptor positive breast cancer and castration-resistant prostate cancer.

Dr. Barry Kappel, Sapience’s CEO and President, commented, "Patients with advanced stage melanoma have very poor prognosis with a median survival of less than two years from diagnosis. The available treatments are limited, and many patients are refractory to targeted approaches or immunotherapy agents. With its unique mechanism of action targeting a key transcription factor C/EBPβ, we have a significant opportunity to deliver a novel therapeutic option with ST101. Our clinical plans remain on track, and we look forward to completing enrollment in all four expansion cohorts in Phase 2."

Dr. Gina Capiaux, Sapience’s Vice President, Regulatory Affairs added, "Receiving our third Orphan Drug Designation is another important regulatory achievement that reinforces the FDA’s recognition of the potential of ST101 to improve clinical outcomes in patients with advanced melanoma. We look forward to quickly advancing the development of ST101 for patients in need."

Orphan Drug Designation is granted to drugs or biological products for the treatment of rare diseases or conditions that impact fewer than 200,000 people in the United States. Incentives that come with the designation include eligibility for federal grants, research and development tax credits, waiver of filing fees, and the potential for a 7-year marketing exclusivity period. The designation does not alter the standard regulatory requirements and process for obtaining marketing approval. ST101 also previously received Fast Track Designation for recurrent GBM and advanced cutaneous melanoma in patients who have disease progression on or after anti-PD-1/anti-PD-L1 therapy.

About ST101
ST101, a first-in-class antagonist of C/EBPβ, is currently being evaluated in the Phase 2 portion of an ongoing Phase 1-2 clinical study in patients with advanced unresectable and metastatic solid tumors (NCT04478279). ST101-101 is an open-label, two-part, Phase 1-2 dose-finding study designed to determine the safety, tolerability, PK, PD, and proof-of-concept efficacy of ST101 in patients with advanced solid tumors. The study consists of two phases: a Phase 1 dose escalation/regimen exploration phase and a Phase 2 expansion phase. In the ongoing dose escalation study, ST101 has demonstrated clinical proof-of-concept with a RECIST 1.1-confirmed partial response (PR) in a patient with cutaneous melanoma and evidence of long-lasting stable disease in several additional patients. In the ongoing Phase 2 dose expansion part of the study, Sapience has initiated enrollment in patients with GBM, metastatic cutaneous melanoma, locally advanced or metastatic hormone-receptor positive breast cancer and castration-resistant prostate cancer. ST101 has been granted Fast Track designation for recurrent GBM and advanced cutaneous melanoma in patients who have disease progression on or after anti-PD-1/anti-PD-L1 therapy, as well as orphan designations from the FDA for advanced melanoma, glioma and AML, and from the European Commission for the treatment of glioma.

On May 3, 2022 Oncotelic Therapeutics, Inc. ("Oncotelic" or the "Company" or "We") (OTCQB:OTLC), reported the appointment of Dr. Fatih Uckun as its Chief Medical Officer (Press release, Oncotelic, MAY 3, 2022, View Source [SID1234613416]). Dr. Uckun will be responsible for execution of internal company led registrational trials.

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"The addition of Dr. Uckun to our executive team, comes at a critical time for Oncotelic. We have made significant inroads toward our goal of developing OT-101 as treatment for difficult to treat cancers," said Dr. Vuong Trieu, CEO and Chairman. "Dr. Uckun is one of the highest caliber physician scientists. His deep knowledge in clinical oncology and vast experience in translational research for successful applications of immunotherapy in oncology, especially for difficult to treat cancer subtypes will be invaluable, as we further advance OT-101 and the rest of our product portfolio."

"With the completion of the joint venture with Dragon Overseas Capital Limited for OT-101, we will be initiating OT-101 registration trials against difficult to treat cancers such as DIPG and pancreatic cancers", said Dr. Fatih Uckun, CMO. "I look forward to engaging the global oncology community in bringing OT-101 to these patients."

About Dr. Uckun

Dr. Uckun is an elected Member of the American Society for Clinical Investigation (ASCI), an honor society for physician-scientists, and an active member of several professional organizations. He received numerous awards for his work on monoclonal antibodies, recombinant cytokines and fusion proteins, radiation sensitizers, kinase inhibitors and targeted therapeutics for difficult-to-treat cancers, including the Stohlman Memorial Award of the Leukemia Society of America, the highest honor given to a Leukemia Society Scholar.

Prior to joining Oncotelic, Dr. Uckun served as Chief Medical Officer and Chief Scientific Officer of Reven Pharmaceuticals (from 2020 to 2022), Chief Medical Officer of Ares Pharmaceuticals (from 2017 to 2022), Chief Clinical Advisor of Aptevo Therapeutics (2021), Vice President and Clinical Strategy Lead, Oncology-Hematology of Worldwide Clinical Trials (2020), Chief Medical Officer of Mateon and Oncotelic (2019-2020), and Executive Medical Director and Strategy Lead in Global Oncology and Hematology at Syneos Health (from 2017 to 2018). Prior to this, he was Vice President of Research and Clinical Development at Nantkwest, Chief Scientific Officer of Jupiter Research Institute and, before that, held senior-level scientific and research positions at Parker Hughes Institute and its cancer center, Paradigm Pharmaceuticals, and the Children’s Cancer Study Group.

Dr. Uckun earned his doctoral degrees at the University of Heidelberg, Germany where he also served as an active member of the autologous bone marrow transplant and peripheral stem cell transplant clinical research teams of the Tumor Center. Dr. Uckun completed his residency training in pediatrics, clinical fellowship training in Hematology/Oncology/Blood and Bone Marrow Stem Cell Transplantation as well as postdoctoral research training in immunology at the University of Minnesota. Dr. Uckun has more than 30 years of professional experience in developmental therapeutics with a special emphasis on targeted therapeutics/precision medicines and biopharmaceuticals. He has published more than 500 peer-reviewed papers, authored numerous review articles and book chapters and and is an inventor on numerous patents.

Dr. Uckun worked as a Professor of Therapeutic Radiology-Radiation Oncology, Pharmacology, and Pediatrics as well as Director of the Biotherapy Institute at the University of Minnesota (1986-1997), where he became the first recipient of the Endowed Hughes Chair in Biotherapy, and as a Professor of Pediatrics and Head of Translational Research in Leukemia and Lymphoma of the Children’s Center for Cancer and Blood Diseases at the University of Southern California (2009-2015). From 2012-2015, Dr. Uckun served as chair of the Biotargeting Working Group and a Member of the Coordination and Governance Committee of the NCI Alliance for Nanotechnology in Cancer.

On May 3, 2022 Veracyte, Inc. (Nasdaq: VCYT) reported financial results for the first quarter ended March 31, 2022 (Press release, Veracyte, MAY 3, 2022, View Source [SID1234613409]).

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"I am extremely pleased with our team’s strong results in the first quarter," said Marc Stapley, Veracyte’s chief executive officer. "Last year we assembled the critical elements needed to drive near and long-term growth, including our acquisitions of Decipher Biosciences and HalioDx, complementing our prior acquisition of the nCounter diagnostic platform. We opened this year with the organization re-aligned to fully execute our strategic plan initiatives. With our innovative lab-based business and international reach, enabled by the automated nCounter platform, we are positioned to achieve our vision of improving outcomes for patients all over the world."

Key Business Highlights:

Increased first quarter total revenue by 85% to $67.8 million, compared to the first quarter of 2021;
Grew test volumes to 23,245, an increase of 61% compared to the first quarter of 2021;
Announced that new, expanded data will be presented at the upcoming American Thoracic Society (ATS) 2022 International Conference on the Percepta Nasal Swab test’s performance in current and former smokers with lung nodules, regardless of their smoking history burden;
Announced that three abstracts will be presented at the upcoming American Urological Association conference highlighting new data on the genomic underpinnings of prostate and bladder cancers, derived from analyses of the Decipher GRID database;
Introduced the Veracyte Biopharma Atlas, a unique pan-cancer database of comprehensive biomarkers linked to clinical outcome data leveraging Veracyte’s unique multi-omic and immuno-oncology capabilities, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting in April;
Announced that an updated clinical practice guideline, as well as a review article and separate commentary, were published in journals of the American Thoracic Society and highlight the role of the Envisia Genomic Classifier in the diagnosis of idiopathic pulmonary fibrosis (IPF); and
Ended the first quarter of 2022 with cash and cash equivalents of $163.6 million.
First Quarter 2022 Financial Results

Total revenue for the first quarter of 2022 was $67.8 million, compared to $36.7 million in the first quarter of 2021. Testing revenue was $56.0 million, an increase of 69% compared to $33.1 million in the first quarter of 2021 driven by the strong performance of our Decipher urology portfolio. Product revenue was $3.0 million, a decrease of 3% compared to $3.1 million in the first quarter of 2021 as volume growth was offset by an approximately 5% currency headwind due to a decline in exchange rates. Biopharmaceutical and other revenue was $8.8 million, an increase of $8.2 million compared to $0.6 million in the first quarter of 2021, driven primarily by the contribution of the HalioDx acquisition.

Total gross margin for the first quarter of 2022, including the amortization of acquired intangible assets, was 58%, compared to 62% in the first quarter of 2021. Non-GAAP gross margin, excluding the amortization of acquired intangible assets and other acquisition related expenses was 65%, compared to 66% in the first quarter of 2021.

Operating expenses, excluding cost of revenue, were $54.4 million, a decrease of 20% compared to the first quarter of 2021. Non-GAAP operating expenses, excluding the cost of revenue, amortization of acquired intangible assets and other acquisition related expenses, were $49.1 million compared to $31.9 million in the first quarter of 2021.

First quarter 2022 net loss was $14.5 million, an improvement of 65% compared to the first quarter of 2021. Basic and diluted net loss per common share was $0.20, an improvement of 70% compared to the first quarter of 2021. Net cash used by operating activities in the first quarter of 2022 was $8.9 million, an improvement of $31.7 million compared to the first quarter of 2021.

A reconciliation of GAAP to non-GAAP financial measures has been provided in the tables included in this press release. An explanation of these measures is also included below under the heading "Note Regarding Use of Non-GAAP Financial Measures."

2022 Financial Outlook

The company is currently projecting full year 2022 total revenue of $265 million to $275 million, representing year-over-year growth of 21% to 25% compared to the prior year. This compares to our prior 2022 total revenue guidance of $260 million to $275 million.

Conference Call and Webcast Details

Veracyte will host a conference call and webcast today at 4:30 p.m. Eastern Time to discuss the company’s financial results and provide a general business update. The conference call will be webcast live from the company’s website and will be available via the following link: View Source The webcast should be accessed 10 minutes prior to the conference call start time. A replay of the webcast will be available for one year following the conclusion of the live broadcast and will be accessible on the company’s website at View Source

On May 3, 2022 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported that it will report first quarter 2022 financial results on Tuesday, May 10, 2022, prior to the open of the market (Press release, UroGen Pharma, MAY 3, 2022, View Source [SID1234613408]). The announcement will be followed by a live audio webcast and conference call at 10:00 AM Eastern Time.

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Audio Webcast

The webcast will be made available on the Investors section of the Company’s website at View Source Following the live audio webcast, a replay will be available on the Company’s website for approximately 30 days.