On May 5, 2021 Castle Biosciences, Inc. (Nasdaq: CSTL), a skin cancer diagnostics company providing personalized genomic information to improve cancer treatment decisions, reported data on its 15-gene expression profile (15-GEP) test, DecisionDx-UM, at the Association for Research in Vision and Ophthalmology (ARVO) 2021: Revolutionary Eye and Vision Research Meeting (Press release, Castle Biosciences, MAY 5, 2021, View Source [SID1234579228]).

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The virtual poster is entitled "Uveal Melanoma Patient Attitudes Towards Prognostic Testing Using Gene Expression Profiling."

DecisionDx-UM, the test highlighted in the poster, is Castle’s prognostic 15-GEP test for patients with uveal melanoma, a rare cancer of the eye that carries a high risk of spreading (metastasizing). The DecisionDx-UM test is designed to accurately identify patients who are at low risk (Class 1) or high risk (Class 2) of metastasis based on the unique biology of their primary tumor and is the current standard of care in the management of uveal melanoma at the majority of U.S. ocular oncology practices.

"Up to half of patients diagnosed with uveal melanoma will experience metastatic disease, and prior studies show that newly diagnosed patients have overwhelmingly been in favor of learning their prognoses," said first author Basil K. Williams, M.D., assistant professor and director of Ocular Oncology at the University of Cincinnati College of Medicine. "This study demonstrated that uveal melanoma patients were satisfied with their decisions to pursue prognostic information through GEP testing, and they found particular value in DecisionDx-UM’s ability to help them understand their individual metastatic risk."

Study methods and findings:

The objective of the patient-based study was to understand uveal melanoma patients’ experiences following testing with DecisionDx-UM compared to patients with alternative or no prognostic testing.
An online questionnaire was distributed by the Melanoma Research Foundation’s CURE OM (Ocular Melanoma) initiative that captured de-identified information regarding patient-reported experiences. Patients were asked questions regarding the decision to undergo prognostic testing and the extent to which they felt regret about their decisions.
Of the 177 survey participants, 159 (90%) reported wanting prognostic information at diagnosis.
Of patients tested with DecisionDx-UM, the vast majority (80/81 respondents, 99%) reported gaining value from their test result, including:
Increased knowledge and understanding
More personalized treatment options
Information relevant to life planning
A sense of relief from uncertainty about the future
Of the patients who received prognostic testing with DecisionDx-UM, decision regret levels did not differ depending on whether they received a low or high-risk test result (Kruskal-Wallis; n=28, 23, 30 for 1A, 1B, 2; p=0.13).
Patients who received prognostic testing experienced lower levels of decision regret than those who opted out of testing, independent of which prognostic tests were used (Wilcoxon Rank-Sum tests: DecisionDx-UM vs. alternative tests: p=0.89, DecisionDx-UM vs. opt-out: p=0.0002, alternative tests vs. opt-out: p=0.003).

On May 5, 2021 IVERIC bio, Inc. (Nasdaq: ISEE) reported financial and operating results for the quarter ended March 31, 2021 and provided a general business update (Press release, Ophthotech, MAY 5, 2021, View Source [SID1234579255]).

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"Iveric Bio is entering an important period as we remain focused on the execution of our ongoing Zimura GATHER2 clinical trial, which is our second Phase 3 clinical trial for Zimura for the treatment of geographic atrophy secondary to age-related macular degeneration. We are committed to completing recruitment for the GATHER2 trial in the third quarter of this year," stated Glenn P. Sblendorio, Chief Executive Officer of Iveric Bio. "To date, both the recruitment and retention of patients in GATHER2 have exceeded our expectations. We are on track for initial, topline data from GATHER2 to be available approximately one year after the recruitment of the last patient in the GATHER2 clinical trial, plus the time needed for database closure and analysis of the initial, topline data."

"A key goal of ours is to expand and advance our footprint in multiple stages and types of AMD," stated Pravin U. Dugel, M.D., President of Iveric Bio. "We are excited by the opportunity to potentially expand the reach of Zimura beyond GA and to continue the development of IC-500, our HtrA1 inhibitor, which we expect could be complementary to Zimura in treating AMD patients. We remain committed to developing safe and effective therapeutic and gene therapy treatment options for retinal diseases with significant unmet medical needs."

Therapeutics Programs Targeting Geographic Atrophy Secondary to Age-Related Macular Degeneration

Zimura (avacincaptad pegol): Complement C5 Inhibitor

Enrollment and retention for GATHER2, the Company’s pivotal clinical trial of Zimura in development for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD), are progressing well with enrollment on target. In March 2021, the Company announced it accelerated the timeline for when it expects to complete enrollment in GATHER2 to the third quarter of 2021.
The Phase 2b screening clinical trial of Zimura for the treatment of autosomal recessive Stargardt disease, referred to as the STAR trial, is ongoing with the goal of enrolling approximately 120 patients.
IC-500: HtrA1 (high temperature requirement A serine peptidase 1 protein) Inhibitor

During the first quarter of 2021, the Company revised its development plans for IC-500 to include plans to investigate multiple dosing schedules for this product candidate. In April 2021, the Company commenced its first preclinical tolerability study for IC-500 and is currently planning additional preclinical studies, including pharmacokinetic and target engagement studies. Formulation optimization and other manufacturing activities are also ongoing. The Company expects to submit an IND to the FDA for IC-500 in GA secondary to AMD in the second half of 2022.
Iveric Bio to Host Dry AMD Virtual Symposium for Investors/Analysts

The Company will host a dry AMD Virtual Symposium for investors and analysts on Friday, June 18, 2021 from 10:00am – 12:00pm Eastern Time. The event will include presentations and discussions with retinal specialists and key opinion leaders on the dry AMD landscape, Zimura pivotal program in GA and highlights from the Company’s IC-500 program in AMD. The event will be accessible via webcast on the Iveric Bio website at www.ivericbio.com. For more information, please contact Kathy Galante at [email protected].

Gene Therapy Programs in Orphan Inherited Retinal Diseases (IRDs)

IC-200: BEST1-Related IRDs
The Company is completing a preclinical efficacy and toxicology study for IC-200, in the naturally occurring canine model of Best disease. Published data have demonstrated long-term rescue in this model following a single sub-retinal injection. The Company is on track to release the recently manufactured cGMP batch of IC-200 in preparation for the planned IND filing and plans to move IC-200 into the clinic, in a Phase 1/2 trial in the second half of 2021.
IC-100: Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa (RHO-adRP).
The Company continues to evaluate the results of preclinical toxicology studies for IC-100. In the Company’s preclinical efficacy and toxicology study in a naturally occurring canine model of RHO-adRP, efficacy was demonstrated at all three doses tested. The Company also tested the same three doses in a GLP toxicology study in non-human primates. Ocular inflammation on clinical exam was observed in the high dose group in canines and to varying degrees at different dosing levels tested in non-human primates. Due to the different findings in the two different species, and the Company’s high commitment to the safety of its patients, the Company is planning to discuss with regulators the design of its planned first-in-human clinical trial for IC-100 prior to submitting an IND. The Company now believes that IC-100 will likely be delayed from entering into a Phase 1/2 clinical trial this year.
Minigene Programs
The Company, in its minigene collaboration with the University of Massachusetts Medical School, has identified a lead construct for its Leber Congenital Amaurosis Type 10 (LCA10) program and is currently considering development plans for this program. The Company expects to obtain additional results from its Stargardt Disease (ABCA4) program in the second quarter of 2021, and expects to obtain preliminary results from its USH2A-related inherited retinal diseases program in the second half of 2021.
The Company announced today the formation of its Gene Therapy Inherited Retina Disease Scientific Advisory Committee that will work closely with senior management as the Company advances its gene therapy inherited retinal disease programs. The members of the advisory committee include:

Elias Traboulsi, MD, MEd
Head of the Department of Pediatric Ophthalmology
Director of the Center for Genetic Eye Diseases
Cole Eye Institute
Professor of Ophthalmology, Cleveland Clinic Lerner College of Medicine,
Cleveland Clinic
Andreas K. Lauer, MD
Chair, Department of Ophthalmology, Casey Eye Institute
Professor of Ophthalmology, School of Medicine
Bart P. Leroy, MD, PhD
Head, Department of Ophthalmology, Ghent University Hospital
Senior Staff Member, Center for Medical Genetics Ghent, Ghent University Hospital
Professor of Ophthalmology & Ophthalmic Genetics, Ghent University
Director of the Retinal Degenerations Clinic Children’s Hospital of Philadelphia
Mark Pennesi, MD, PhD
Division Chief, Ophthalmic Genetics
Associate Professor in Ophthalmology, Oregon Health & Science University
Eleonora Lad, MD, PhD
Director of Grading, Duke Reading Center
Associate Professor of Ophthalmology, Duke University Medical Center
Board of Directors and Management

Today the Company announced the promotions of Pravin U. Dugel, MD, to President, and Kathy Galante to Senior Vice President, Investor Relations, both effective as of May 1.
In April 2021, the Company announced that David R. Guyer, MD, was stepping down from the Iveric Bio Board of Directors after 14 years, effective following Iveric Bio’s 2021 Annual Stockholder Meeting scheduled to be held on May 19, 2021.
First Quarter Financial Results and 2021 Cash Guidance

As of March 31, 2021, the Company had $180.2 million in cash, cash equivalents and available for sale securities.
The Company estimates its year-end 2021 cash, cash equivalents and available for sale securities to range between $125 and $135 million. The Company also estimates that its cash, cash equivalents and available for sale securities will be sufficient to fund its planned capital expenditure requirements and operating expenses, excluding any potential approval or sales milestones payable to Archemix Corp. or any commercialization expenses for Zimura, into 2024. These estimates are based on the Company’s current business plan, including the continuation of its ongoing clinical development programs for Zimura, the progression of its IC-100 and IC-200 programs into the clinic, and the advancement of its IC-500 development program. These estimates also assume that the Company will enroll approximately 400 patients in the GATHER2 trial. These estimates do not reflect any additional expenditures related to potentially studying Zimura in other indications or resulting from the potential in-licensing or acquisition of additional product candidates or technologies or commencement of new sponsored research programs, and any associated development the Company may pursue.
2021 Q1 Financial Highlights

R&D Expenses: Research and development expenses were $18.5 million for the quarter ended March 31, 2021, compared to $13.8 million for the same period in 2020. Research and development expenses increased primarily due to the initiation of our GATHER2 trial and commencement of patient enrollment and increased manufacturing activities for Zimura, increased manufacturing and preclinical development activities associated with the Company’s IC-100 and IC-200 gene therapy programs and the progression of its IC-500 development program.
G&A Expenses: General and administrative expenses were $8.3 million for the quarter ended March 31, 2021, compared to $5.0 million for the same period in 2020. General and administration expenses increased primarily due to legal costs associated with ongoing litigation.
Income Tax Benefit: For the quarter ended March 31, 2021, the Company recorded no income tax benefit. An income tax benefit of $3.3 million was recognized in the quarter ended March 31, 2020 to reflect a favorable settlement of a state corporate income tax audit.
Net Loss: The Company reported a net loss for the quarter ended March 31, 2021 of $26.8 million, or ($0.29) per diluted share, compared to a net loss of $15.1 million, or $(0.28) per diluted share, for the same period in 2020.
Conference Call/Web Cast Information

Iveric Bio will host a conference call/webcast to discuss the Company’s financial and operating results and provide a business update. The call is scheduled for May 5, 2021 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 888-317-6003 (USA) or 412-317-6061 (International), passcode 5841649. A live, listen-only audio webcast of the conference call can be accessed on the Investors section of the Iveric Bio website at www.ivericbio.com. A replay will be available approximately two hours following the live call for two weeks. The replay number is 877-344-7529 (USA) or 412-317-0088, passcode 10153477.

On May 5, 2021 Eli Lilly and Company (NYSE: LLY) reported that it will participate in the Bank of America Securities 2021 Health Care Conference on Tuesday, May 11, 2021 (Press release, Eli Lilly, MAY 5, 2021, View Source [SID1234579176]). Michael Mason, senior vice president, president of Lilly Diabetes, will participate in a virtual fireside chat at 9:30 a.m., Eastern Time.

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A live audio webcast will be available on the "Webcasts & Presentations" section of Lilly’s Investor website at View Source A replay of the presentation will be available on this same website for approximately 90 days.

On May 5, 2021 Quanterix Corporation (NASDAQ: QTRX), a company helping to transform healthcare with digital protein biomarker technology enabling precision health, reported financial results for the three months ending March 31, 2021 (Press release, Quanterix, MAY 5, 2021, View Source [SID1234579201]).

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"We made productive advances on our strategic and operational priorities during the first quarter 2021," said Chairman, Chief Executive Officer and President, Quanterix, Kevin Hrusovsky. "The precision health ecosystem continues to catalyze interest and adoption from healthcare payors, government agencies, academia, and pharmaceutical companies as they make tangible advances in developing drug therapies for Alzheimer’s, Parkinson’s, Multiple Sclerosis, traumatic brain injury, COVID-19, infectious diseases and cancers. We are rapidly innovating our technology and expanding our assay menu with a priority focus on early detection plasma biomarkers for Alzheimer’s and neuro-degeneration to keep pace with today’s research demands and deliver on the promise of non-invasive biomarkers to fuel breakthroughs in diagnostics and drug development."

First Quarter 2021 Financial Highlights

Key financial results for the first quarter of 2021 are shown below:

Q1 GAAP total revenue, which includes grant revenue of $2.3M, was $27.2M versus prior year Q1 of $15.7M;
Q1 non-GAAP total revenue was $24.9M versus prior year Q1 of $15.7M, an increase of 58%;
Q1 GAAP product revenue was $18.2M versus prior year Q1 of $9.8M, an increase of 86%;
Q1 GAAP service and other revenue was $6.4M versus prior year Q1 of $5.8M, an increase of 11%
Q1 GAAP gross margin was 60.1%; Q1 non-GAAP gross margin was 58.5% versus prior year Q1 of 48.5%, an increase of 1,000 bps.
For additional information on the non-GAAP financial measures included in this press release, please see "Use of Non-GAAP Financial Measures" and "Reconciliation of Non-GAAP Financials" below.

First Quarter Business Highlights

Received Emergency Use Authorization (EUA) from the U.S. Food and Drug Administration (FDA) for Quanterix’ Simoa SARS-CoV-2 N Protein Antigen Test that can be run on the Simoa HD-X Analyzer.
Released the SARS-CoV-2 N Protein Antigen Advantage Assay tailored for Research Use, enabling scientists to measure viral load and immune response in a variety of sample matrices, including blood obtained by minimally invasive capillary (finger prick) sample.
Record Instrument placement revenue of $7.0M, an increase of 86%, and consumables revenue of $11.3m, an increase of 86%.
Simoa played a key role in research to detect the COVID-19 virus in serum/plasma, dried blood microsamples (DBS) and saliva samples, including a Nature Communications study validating non-invasive antigen detection methods as an important complement to current PCR-based nasal and nasopharyngeal (NP) swab samples.
Continued to experience significant demand for phosphorylated tau at threonine 181 (pTau181) assays for the study of Alzheimer’s Disease (AD) pathology and Neuro Multiplex assays associated with many neurodegenerative diseases.
Simoa neurology assays and technologies continue to support various Alzheimer’s drug development programs globally, focusing on detecting signs of precognitive impairment early in the disease cascade with blood biomarkers.
Established a joint Simoa laboratory with WuXi AppTec in China to improve access to Quanterix’ industry leading biomarker testing and development offerings across China and the APAC market.
Successfully raised approximately $270M in net proceeds through a follow-on offering in February, further strengthening our balance sheet with growth capital.
Participated in important industry events aimed at addressing public health challenges created by the COVID-19 pandemic, including the Precision Medicine World Conference (PMWC) 2021 COVID-19 Conference and the Precision Medicine Leadership Summit hosted in connection with Powering Precision Health.
Invited to discuss the Powering Precision Health movement, rebuilding public trust in science and leadership of the industry-wide proteomics revitalization disrupting healthcare on Health Care Rounds Podcast and Mendelspod.
Welcomed Laurie Olson, a seasoned industry executive and former Pfizer executive leadership team member, to our board as we continue to scale the business and capitalize on the market opportunity ahead.
Simoa technology was highlighted in 110 new publications in the first quarter 2021, bringing total Simoa-specific inclusions to more than 1,200 publications.
Conference Call

In conjunction with this announcement, Quanterix Corporation will host a conference call on May 5 at 4:30 p.m., EDT. Individuals interested in listening to the conference call may do so by dialing (800) 697-5978 for domestic callers, or (630) 691-2750 for international callers. Please reference the following conference ID: 9220 612#. A live webcast will also be available at: View Source

The webcast will be available on the Company’s website, View Source, for one year following completion of the call.

On May 4, 2021 Clarity Pharmaceuticals, a clinical stage radiopharmaceutical company focused on the treatment of serious disease, reported that it has received a response from the U.S. Food and Drug Administration (FDA) on its Investigational New Drug (IND) application that the study for selection and treatment of Prostate-Specific Membrane Antigen (PSMA) positive prostate cancers using 64Cu-SAR-bisPSMA and 67Cu-SAR-bisPSMA may proceed (Press release, Clarity Pharmaceuticals, MAY 4, 2021, View Source [SID1234579036]).

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The SECuRE trial (Systemic Cu theranostics in prostate cancer) is a Phase I/IIa study for identification and treatment of PSMA-expressing metastatic castrate resistant prostate cancer (mCRPC), which will be conducted in the U.S. (NCT04868604)1. It is a theranostic multi-centre, single arm, dose escalation study with a cohort expansion planned for up to 44 patients. The trial employs diagnostic Positron Emission Tomography imaging with 64Cu-SAR-bisPSMA for selection of patients suitable for therapy cycles with 67Cu-SAR-bis-PSMA.

Prostate cancer is the second most common cancer diagnosed in men globally and the fifth leading cause of death worldwide2. 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 disease3. 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 mCRCP3, ~90% of whom have tumours which express PSMA4.

Although there are some new therapeutic radiopharmaceutical agents for prostate cancer in late phase clinical trials, given the large patient population, product supply for therapeutic radiopharmaceuticals presents a constraint as they rely on the production of therapeutic isotopes from a small number of nuclear reactors, with reactor shutdowns often causing isotope shortages around the globe.

Clarity’s SAR-bisPSMA product utilises two isotopes of copper, which do not have the same constraints:

The therapeutic product utilises copper-67, which is being produced domestically in the USA on electron accelerators, avoiding the issues commonly associated with the production of isotopes on nuclear reactors.
The diagnostic product utilises copper-64, which is regularly produced in significant volumes on cyclotrons in the U.S., and has a half-life of 12.7 hours, avoiding the short half-life issues of other diagnostic isotopes.
The diagnostic and therapeutic products can be centrally manufactured and shipped as finished product direct to the treatment centres, which removes the need for dedicated radiopharmacy facilities at treatment centres.
Dr Alan Taylor, Clarity’s Executive Chairman, commented on the IND approval, "The FDA response suggests not only the importance of developing novel treatments for men with late-stage prostate cancer, whose prognosis is currently very poor, but also validates Clarity’s copper pairing paradigm and the centralised manufacturing concept, which differentiates it from the competitor products and enables product supply to the levels suitable for use in large patient indications."

"We are very excited to commence the SECuRE trial in mCRCP patients and have engaged a world class group of key opinion leaders in the prostate cancer space to support the development of 64/67Cu SAR-bisPSMA. Clarity’s Global Clinical Development Group has unrivalled experience in the commercialisation of the only currently approved radiotherapeutic for prostate cancer. The FDA response is a crucial milestone in the development of SAR-bisPSMA theranostics and we are looking forward to progressing this trial at some of the leading cancer centres in the U.S. as part of our ultimate goal of developing better treatments for children and adults with cancer," said Dr Taylor.

Reference List
National Institute of Health, U.S. National Library of Medicine, View Source
World Cancer Research Fund, Prostate Cancer Statistics, View Source
American Cancer Society, Cancer Statistics Center, View Source!/cancer-site/Prostate
A. Silver, I. Pellicer, W. R. Fair, W. D. Heston and C. Cordon-Cardo 1997. "Prostate-specific membrane antigen expression in normal and malignant human tissues." Clinical Cancer Research. vol. 3, 81-85, January 1997