On May 5, 2021 Bristol Myers Squibb (NYSE: BMY) reported that the company will participate in a fireside chat at the 2021 Bank of America Securities Virtual Health Care Conference, which will be webcast on Tuesday, May 11, 2021 (Press release, Bristol-Myers Squibb, MAY 5, 2021, View Source [SID1234579211]). Chris Boerner, Ph.D., Executive Vice President, Chief Commercialization Officer will answer questions about the company at 5 p.m. ET.

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Investors and the general public are invited to listen to a live webcast of the session at View Source An archived edition of the session will be available later that day.

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 4, 2021 Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") reported that the European Commission (EC) has approved an additional indication for the oral once-daily therapy XTANDI (enzalutamide) for adult men with metastatic hormone-sensitive prostate cancer (mHSPC, also known as metastatic castration-sensitive prostate cancer or mCSPC) (Press release, Astellas, MAY 4, 2021, View Source [SID1234579030]). Men diagnosed with mHSPC tend to have a poor prognosis, with a median survival of approximately 3-4 years,1 underscoring the need for new treatment options.

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With this indication, enzalutamide is now the only oral treatment approved by the EC to treat three distinct types of advanced prostate cancer — non-metastatic and metastatic castration-resistant prostate cancer (CRPC) and mHSPC.2 The EC approval is based on results from the pivotal Phase 3 ARCHES trial which evaluated enzalutamide in men with mHSPC.3

"Metastatic hormone-sensitive prostate cancer patients have limited options and, unfortunately, there is a poor prognosis for many men," said Andrew Armstrong, M.D., Professor of Medicine, Surgery, Pharmacology and Cancer Biology, Director of Research in the Duke Cancer Institute’s Center for Prostate and Urologic Cancers and lead investigator of ARCHES. "The research supporting this approval provides clinical evidence showing how enzalutamide can help improve outcomes for men with mHSPC, which gives healthcare professionals in Europe the option to offer the treatment across the advanced prostate cancer disease continuum."

Data from the ARCHES trial showed enzalutamide plus androgen deprivation therapy (ADT) significantly reduced the risk of radiographic progression or death by 61% versus placebo plus ADT in men with mHSPC (n=1,150; hazard ratio [HR]=0.39 [95% confidence interval (CI): 0.30-0.50]; P<0.0001).3

"Enzalutamide has been an established standard of care for men with advanced prostate cancer and has been prescribed to more than 610,000 patients worldwide since it was first approved in 2012," said Andrew Krivoshik, M.D., Ph.D., Senior Vice President and Global Therapeutic Area Head, Oncology Development, Astellas. "This new indication for enzalutamide provides men with mHSPC a much-needed, additional therapy option earlier in their treatment journey. We look forward to working with health authorities across Europe to ensure men with mHSPC have access to enzalutamide as soon as possible."

The safety analyses of the ARCHES trial appear consistent with the safety profile of enzalutamide in previous clinical trials in CRPC. In ARCHES, Grade 3 or greater adverse events (AEs) (defined as severe/disabling or life-threatening) were similar for patients receiving both enzalutamide plus ADT and those who received placebo plus ADT (24.3% vs. 25.6%).3

The EC marketing authorization for enzalutamide in men with mHSPC is applicable to European Union (EU) member countries, and is also valid in Iceland, Norway and Liechtenstein.4

This approval will have no impact on the financial forecasts of the current fiscal year ending March 31, 2022.

About metastatic Hormone-Sensitive Prostate Cancer (mHSPC)
In men with prostate cancer, the disease is considered metastatic once the cancer has spread outside of the prostate gland to other parts of the body.5 Men are considered hormone- (or castration-) sensitive if their disease still responds to medical or surgical treatment to lower testosterone levels.6 mHSPC has a median survival of approximately 3-4 years for men starting treatment with ADT.1

About XTANDI (enzalutamide)
Enzalutamide is an androgen receptor signaling inhibitor indicated in the EU for the treatment of adult men with:2

Metastatic hormone-sensitive prostate cancer (mHSPC, also known as metastatic castration-sensitive prostate cancer or mCSPC) in combination with ADT.
High-risk non-metastatic castration-resistant prostate cancer (CRPC).
Metastatic CRPC who are asymptomatic or mildly symptomatic after failure of androgen deprivation therapy in whom chemotherapy is not yet clinically indicated. It is also indicated in adult men with metastatic CRPC whose disease has progressed on or after docetaxel therapy.
Important Safety Information
For important Safety Information for enzalutamide please see the full Summary of Product Characteristics at: View Source

About ARCHES
The company-sponsored, Phase 3, randomized, double-blind, placebo-controlled, multinational ARCHES trial (NCT02677896) enrolled 1,150 patients with mHSPC at sites in the U.S., Canada, Europe, South America, and the Asia-Pacific region. Patients in the trial were randomized to receive enzalutamide 160 mg daily or placebo and continued on a luteinizing hormone-releasing hormone (LHRH) agonist or antagonist or had a history of bilateral orchiectomy. The primary endpoint of the trial was radiographic progression-free survival (rPFS) assessed by blinded independent central review. rPFS was defined as the time from randomization to radiographic disease progression at any time or death within 24 weeks after study drug discontinuation. Radiographic disease progression was defined by identification of two or more new bone lesions on a bone scan with confirmation (Prostate Cancer Working Group 2 criteria) and/or progression in soft tissue disease. Patients were stratified by volume of disease (low vs high) and prior docetaxel therapy for prostate cancer (no prior docetaxel, 1-5 cycles, or 6 prior cycles).3

On May 4, 2021 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported that it will release its first quarter 2021 financial results on Tuesday, May 11, after the close of the U.S. financial markets (Press release, Syndax, MAY 4, 2021, View Source [SID1234579045]).

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In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET on Tuesday, May 11, to discuss the Company’s financial results and provide a general business update.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com. Alternatively, the conference call may be accessed through the following:

For those unable to participate in the conference call or webcast, a replay will be available on the Investors section of the Company’s website, www.syndax.com.