Neoleukin Therapeutics to Participate in Canaccord Genuity 41st Annual Growth Conference

On August 4, 2021 Neoleukin Therapeutics, Inc., "Neoleukin" (NASDAQ:NLTX), a biopharmaceutical company utilizing sophisticated computational methods to design de novo protein therapeutics, reported that Jonathan Drachman, M.D., Chief Executive Officer, will participate in a fireside chat at the virtual Canaccord Genuity 41ST Annual Growth Conference on Wednesday, August 11, 2021 at 8:00 a.m. Eastern Time (Press release, Neoleukin Therapeutics, AUG 4, 2021, View Source [SID1234585921]).

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A live audio webcast of the corporate presentation and question and answer session to follow will be available from the investors section of the Neoleukin website at View Source An archived replay will also be available on the company website for at least 30 days following the event.

G1 Therapeutics Granted New Technology Add-On Payment (NTAP) for COSELA™ (Trilaciclib) by Centers for Medicare & Medicaid Services (CMS)

On August 4, 2021 G1 Therapeutics, Inc. (Nasdaq: GTHX), a commercial-stage oncology company, reported that the Centers for Medicare & Medicaid Services (CMS) has granted a new technology add-on payment (NTAP) for COSELA (trilaciclib) when administered to Medicare beneficiaries in the hospital inpatient setting (Press release, G1 Therapeutics, AUG 4, 2021, View Source [SID1234587577]). It will become effective for provider billing on October 1, 2021. An NTAP provides additional payment to hospitals above the standard Medicare Severity Diagnosis-Related Group (MS-DRG) payment amount.

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This grant follows the receipt of a C Code for pass through hospital outpatient system use (effective July 1, 2021) and a permanent J Code for all sites of care (effective October 1, 2021).

The NTAP will provide hospitals with a payment, in addition to the standard-of-care DRG reimbursement, of up to 65 percent of the average cost of the technology if the cost of the discharge exceeds the full DRG payment. As such, beginning on October 1, 2021, CMS will provide an additional maximum payment of $5,526.30 for COSELA when used in the inpatient hospital setting for fiscal year 2022. Congress created the NTAP program to ensure that Medicare beneficiaries have timely access to innovative therapies while the agency collects data about them to use in future rate-setting.

"CMS’ issuance of an NTAP for COSELA recognizes its potential to address an urgent need for proactive multilineage myeloprotection in patients living with extensive stage small cell lung cancer," said Jack Bailey, Chief Executive Officer of G1 Therapeutics. "We believe the decision to include COSELA in the NTAP program is an important step that will help increase patient access to this important drug. We applaud CMS for their decision; the need for effective tools to help clinicians reduce or prevent myelosuppressive events is critical for extensive stage small cell lung cancer patients undergoing chemotherapy."

About COSELA (trilaciclib) for Injection

COSELA (trilaciclib) was approved by the U.S. Food and Drug Administration on February 12, 2021.

Indication
COSELA (trilaciclib) is indicated to decrease the incidence of chemotherapy-induced myelosuppression in adult patients when administered prior to a platinum/etoposide-containing regimen or topotecan-containing regimen for extensive-stage small cell lung cancer.

Important Safety Information
COSELA is contraindicated in patients with a history of serious hypersensitivity reactions to trilaciclib.

Warnings and precautions include injection-site reactions (including phlebitis and thrombophlebitis), acute drug hypersensitivity reactions, interstitial lung disease (pneumonitis), and embryo-fetal toxicity.

The most common adverse reactions (>10%) were fatigue, hypocalcemia, hypokalemia, hypophosphatemia, aspartate aminotransferase increased, headache, and pneumonia.

This information is not comprehensive. Please click here for full Prescribing Information. View Source

To report suspected adverse reactions, contact G1 Therapeutics at 1-800-790-G1TX or call FDA at 1-800-FDA-1088 or visit www.fda.gov/medwatch.

Charles River Laboratories Announces Second-Quarter 2021 Results

On August 4, 2021 Charles River Laboratories International, Inc. (NYSE: CRL) reported its results for the second quarter of 2021 (Press release, Charles River Laboratories, AUG 4, 2021, View Source [SID1234585671]). For the quarter, revenue was $914.6 million, an increase of 34.0% from $682.6 million in the second quarter of 2020.

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Acquisitions contributed 6.0% to consolidated second-quarter revenue growth. The impact of foreign currency translation benefited reported revenue growth by 3.9%. Excluding the effect of these items, organic revenue growth was 24.1%, driven by contributions from all three business segments. The comparison to last year’s COVID-19-related impact increased the reported and organic revenue growth rates in the second quarter of 2021 by 8.6% and 8.0%, respectively, with the greatest impact in the Research Models and Services segment.

On a GAAP basis, second-quarter net income attributable to common shareholders was $88.4 million, an increase of 31.2% from net income of $67.4 million for the same period in 2020. Second-quarter diluted earnings per share on a GAAP basis were $1.72, an increase of 28.4% from $1.34 for the second quarter of 2020. The GAAP net income and earnings per share increases were primarily driven by higher revenue and operating margin improvement, partially offset by higher acquisition-related costs and a higher tax rate. In addition, the gain from the Company’s venture capital and other strategic investments totaled $0.14 per share in the second quarter of 2021, compared to a gain of $0.38 per share for the same period in 2020. The Company’s venture capital and other strategic investment performance has been excluded from non-GAAP results.

On a non-GAAP basis, net income was $133.8 million for the second quarter of 2021, an increase of 68.1% from $79.6 million for the same period in 2020. Second‑quarter diluted earnings per share on a non-GAAP basis were $2.61, an increase of 65.2% from $1.58 per share for the second quarter of 2020. The non-GAAP net income and earnings per share increases were primarily driven by higher revenue and operating margin improvement.

James C. Foster, Chairman, President and Chief Executive Officer, said, "The strength of our leading, non-clinical contract research and manufacturing portfolio was clearly demonstrated in our second-quarter financial performance. Robust industry fundamentals continue to drive unprecedented client demand across most of our businesses, and we are extremely well positioned to succeed in this environment."

"Due to the sustained demand, we are intensely focused on the execution of our strategy, including strengthening our portfolio and strategically adding staff and capacity to support our clients and provide exceptional service to them. We believe the success of this strategy will enable us to achieve our increased 2021 financial guidance, as well as our longer-term strategic and financial goals," Mr. Foster concluded.

Second-Quarter Segment Results

Research Models and Services (RMS)

Revenue for the RMS segment was $176.7 million in the second quarter of 2021, an increase of 51.6% from $116.5 million in the second quarter of 2020. The impact of foreign currency translation contributed 5.2%, and the acquisition of Cellero contributed 1.9% to second-quarter RMS revenue. Organic revenue growth of 44.5% was primarily driven by the year-over-year comparison to the COVID-19-related revenue impact in 2020, which contributed 35.0% on a reported basis and 33.4% on an organic basis to RMS revenue growth. Adjusted for the COVID-19 impact, RMS revenue growth was driven by robust demand for research models across all client segments and geographic regions, particularly in China, as well as higher revenue for research model services.

In the second quarter of 2021, the RMS segment’s GAAP operating margin increased to 24.1% from 3.3% in the second quarter of 2020, and on a non-GAAP basis, the operating margin increased to 27.4% from 9.1%. The GAAP and non-GAAP operating margin increases were driven primarily by operating leverage from higher sales volume of research models, due in part to the favorable comparison to last year’s COVID-19 impact.

Discovery and Safety Assessment (DSA)

Revenue for the DSA segment was $540.1 million in the second quarter of 2021, an increase of 22.0% from $442.6 million in the second quarter of 2020. The impact of foreign currency translation contributed 3.0%, and the acquisitions of Distributed Bio and Retrogenix contributed 0.9% to DSA revenue growth. Organic revenue growth of 18.1% was driven by strong demand in both the Discovery Services and Safety Assessment businesses from biotechnology and global biopharmaceutical clients, as well as a small benefit from the comparison to last year’s COVID-19 impact.

In the second quarter of 2021, the DSA segment’s GAAP operating margin increased to 19.4% from 16.3% in the second quarter of 2020, and on a non-GAAP basis, the operating margin increased to 23.5% from 23.2%. The GAAP and non-GAAP operating margin increases were driven primarily by operating leverage from higher revenue in both the Discovery Services and Safety Assessment businesses, partially offset by foreign currency translation. The impact of foreign currency translation reduced the DSA operating margin by approximately 150 basis points in the second quarter of 2021.

Manufacturing Solutions (Manufacturing)

Revenue for the Manufacturing segment was $197.8 million in the second quarter of 2021, an increase of 60.2% from $123.5 million in the second quarter of 2020. The impact of foreign currency translation contributed 5.4%, and the acquisition of Cognate BioServices (Cognate) contributed 28.2% to second-quarter Manufacturing revenue. Organic revenue growth of 26.6%, was driven primarily by robust demand in the Biologics Testing Solutions and Microbial Solutions businesses.

In the second quarter of 2021, the Manufacturing segment’s GAAP operating margin decreased to 28.7% from 34.8% in the second quarter of 2020, and on a non-GAAP basis, the operating margin decreased to 33.2% from 37.4%. The GAAP and non-GAAP operating margin decreases were driven primarily by the addition of Cognate, as well as higher production costs in the Microbial Solutions business. In addition, the GAAP operating margin declined due to higher amortization costs associated with Cognate.

Increases 2021 Guidance

The Company is increasing its 2021 financial guidance, which was previously provided on May 4, 2021, primarily as a result of the stronger-than-expected second-quarter financial performance and an expectation that robust client demand trends will continue for the remainder of the year.

The Company’s increased guidance for revenue growth, earnings per share, and free cash flow is as follows:

Footnotes to Guidance Table:

(1) The contribution from acquisitions reflects only those acquisitions that have been completed.
(2) Organic revenue growth is defined as reported revenue growth adjusted for acquisitions and foreign currency translation.
(3) Acquisition-related amortization includes an estimate of $0.05-$0.10 for the impact of the Vigene acquisition because the preliminary purchase price allocation has not been completed.
(4) These adjustments are related to the evaluation and integration of acquisitions, and primarily include transaction, advisory, and certain third-party integration costs, as well as certain costs associated with acquisition-related efficiency initiatives.
(5) These items primarily relate to charges of a) approximately $0.30 associated with U.S. and international tax legislation, and b) approximately $0.40 associated with debt extinguishment costs and the write-off of deferred financing costs related to debt refinancing.
(6) Venture capital and other strategic investment performance only includes recognized gains or losses. The Company does not forecast the future performance of these investments.
(7) Reconciliation of the current 2021 free cash flow guidance is as follows: Cash flow from operating activities of approximately $720 million, less capital expenditures of approximately $220 million, equates to free cash flow of approximately $500 million.

Webcast

Charles River has scheduled a live webcast on Wednesday, August 4, at 9:30 a.m. ET to discuss matters relating to this press release. To participate, please go to ir.criver.com and select the webcast link. You can also find the associated slide presentation and reconciliations of GAAP financial measures to non-GAAP financial measures on the website.

Non-GAAP Reconciliations

The Company reports non-GAAP results in this press release, which exclude often-one-time charges and other items that are outside of normal operations. A reconciliation of GAAP to non-GAAP results is provided in the schedules at the end of this press release.

Use of Non-GAAP Financial Measures

This press release contains non-GAAP financial measures, such as non-GAAP earnings per diluted share, which exclude the amortization of intangible assets, and other charges related to our acquisitions; expenses associated with evaluating and integrating acquisitions and divestitures, as well as fair value adjustments associated with contingent consideration; charges, gains, and losses attributable to businesses or properties we plan to close, consolidate, or divest; severance and other costs associated with our efficiency initiatives; the impact of the termination of the Company’s U.S. pension plan; the write-off of deferred financing costs and fees related to debt financing; third-party costs associated with the remediation of unauthorized access into our information systems detected in March 2019; investment gains or losses associated with our venture capital and other strategic equity investments; and adjustments related to the recognition of deferred tax assets expected to be utilized as a result of changes to the our international financing structure and the revaluation of deferred tax liabilities as a result of foreign tax legislation. This press release also refers to our revenue in both a GAAP and non-GAAP basis: "organic revenue growth," which we define as reported revenue growth adjusted for foreign currency translation, acquisitions, and divestitures. We exclude these items from the non-GAAP financial measures because they are outside our normal operations. There are limitations in using non-GAAP financial measures, as they are not presented in accordance with generally accepted accounting principles, and may be different than non-GAAP financial measures used by other companies. In particular, we believe that the inclusion of supplementary non-GAAP financial measures in this press release helps investors to gain a meaningful understanding of our core operating results and future prospects without the effect of these often-one-time charges, and is consistent with how management measures and forecasts the Company’s performance, especially when comparing such results to prior periods or forecasts. We believe that the financial impact of our acquisitions and divestitures (and in certain cases, the evaluation of such acquisitions and divestitures, whether or not ultimately consummated) is often large relative to our overall financial performance, which can adversely affect the comparability of our results on a period-to-period basis. In addition, certain activities and their underlying associated costs, such as business acquisitions, generally occur periodically but on an unpredictable basis. We calculate non-GAAP integration costs to include third-party integration costs incurred post-acquisition. Presenting revenue on an organic basis allows investors to measure our revenue growth exclusive of acquisitions, divestitures, and foreign currency exchange fluctuations more clearly. Non-GAAP results also allow investors to compare the Company’s operations against the financial results of other companies in the industry who similarly provide non-GAAP results. The non-GAAP financial measures included in this press release are not meant to be considered superior to or a substitute for results of operations presented in accordance with GAAP. The Company intends to continue to assess the potential value of reporting non-GAAP results consistent with applicable rules and regulations. Reconciliations of the non-GAAP financial measures used in this press release to the most directly comparable GAAP financial measures are set forth in this press release, and can also be found on the Company’s website at ir.criver.com.

Caution Concerning Forward-Looking Statements

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by the use of words such as "anticipate," "believe," "expect," "intend," "will," "would," "may," "estimate," "plan," "outlook," and "project," and other similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These statements also include statements regarding the impact of the COVID-19 pandemic; the projected future financial performance of Charles River and our specific businesses; the future demand for drug discovery and development products and services, including our expectations for future revenue trends; our expectations with respect to the impact of acquisitions completed in 2020 and 2021 on the Company, our service offerings, client perception, strategic relationships, revenue, revenue growth rates, and earnings; the development and performance of our services and products, including our investments in our portfolio; market and industry conditions including the outsourcing of services and spending trends by our clients; and Charles River’s future performance as delineated in our revised forward-looking guidance, and particularly our expectations with respect to revenue, the impact of foreign exchange, enhanced efficiency initiatives, and the assumptions surrounding the COVID-19 pandemic that form the basis for our revised annual guidance. Forward-looking statements are based on Charles River’s current expectations and beliefs, and involve a number of risks and uncertainties that are difficult to predict and that could cause actual results to differ materially from those stated or implied by the forward-looking statements. Those risks and uncertainties include, but are not limited to: the COVID-19 pandemic, its duration, its impact on our business, results of operations, financial condition, liquidity, business practices, operations, suppliers, third party service providers, clients, employees, industry, ability to meet future performance obligations, ability to efficiently implement advisable safety precautions, and internal controls over financial reporting; the COVID-19 pandemic’s impact on client demand, the global economy and financial markets; the ability to successfully integrate businesses we acquire (including Cognate BioServices and Vigene Biosciences, and risks and uncertainties associated with Cognate’s and Vigene’s products and services, which are in areas that the Company did not previously operate); the timing and magnitude of our share repurchases; negative trends in research and development spending, negative trends in the level of outsourced services, or other cost reduction actions by our clients; the ability to convert backlog to revenue; special interest groups; contaminations; industry trends; new displacement technologies; USDA and FDA regulations; changes in law; the impact of Brexit; continued availability of products and supplies; loss of key personnel; interest rate and foreign currency exchange rate fluctuations; changes in tax regulation and laws; changes in generally accepted accounting principles; and any changes in business, political, or economic conditions due to the threat of future terrorist activity in the U.S. and other parts of the world, and related U.S. military action overseas. A further description of these risks, uncertainties, and other matters can be found in the Risk Factors detailed in Charles River’s Annual Report on Form 10-K as filed on February 17, 2021 and the Quarterly Report on Form 10-Q as filed on May 4, 2021, as well as other filings we make with the Securities and Exchange Commission. Because forward-looking statements involve risks and uncertainties, actual results and events may differ materially from results and events currently expected by Charles River, and Charles River assumes no obligation and expressly disclaims any duty to update information contained in this press release except as required by law.

Assessment of COVID-19 Impact in 2020

In this press release, the Company has provided its assessment for the impact from the COVID-19 pandemic in 2020, including on the Company’s revenue. This assessment was determined using methodologies, assumptions, and estimates that vary depending on the specific reporting segment and situation. For the Research Models and Services segment, the assessment was primarily based on comparisons to daily historical research model sales volumes prior to the COVID-19 pandemic and the subsequent reduction in research model order activity associated with our clients’ COVID-19 pandemic-related site closures and/or their reduced on-site activity, as well as our discussions with clients, particularly of our research model services and HemaCare businesses, with regard to revenue expectations and operational impacts from the COVID-19 pandemic. For the Discovery and Safety Assessment segment, the assessment was based on multiple factors including, but not limited to, discussions with clients with regard to the cause of delays to discovery projects and safety assessment studies, location-specific actions to ensure employee safety in our facilities, the impact of remote versus in-person activities and services, and supply chain delays and other resource constraints. For the Manufacturing Solutions segment, the assessment was based on multiple factors including, but not limited to, analysis of the sales impact due to the COVID-19 pandemic, assessments of idle instruments and the related revenue streams due to the inability to access clients’ sites, as well as discussions with clients with regard to their revenue expectations and operations. The estimated revenue loss related to COVID-19 was also expected to be partially offset by incremental work on clients’ COVID-19 programs. Because this assessment involves risks and uncertainties, actual events and results may differ materially from these estimates and assumptions, and Charles River assumes no obligation and expressly disclaims any duty to update them.

Certara to Host Inaugural Symposium Dedicated to Advancing Pediatric Drug Development

On August 4, 2021 Certara, the global leader in biosimulation, reported it will host the inaugural New Horizons in Pediatric Drug Development Symposium taking place October 28-29, 2021 (Press release, Certara, AUG 4, 2021, View Source [SID1234585706]). The two-day virtual symposium is being held to bring together thought leaders and innovators in pediatric drug development to share new developments in the field and to collaborate on new ideas to advance pediatric drug development into a new era.

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"We are thrilled to have such an amazing group of speakers and renowned experts to support the advancement of drug development in pediatrics," said Patrick Smith, PharmD., Senior Vice President of Integrated Drug Development at Certara and chair of the organizing committee. "Pediatric drug development is rapidly evolving, with exciting new tools and emerging technologies, which address some of the inherent complexities associated with developing drugs for children. We look forward to the exciting conversations this event will bring forth."

The symposium is spearheaded by an organizing committee comprised of regulatory and drug development experts in the biopharmaceutical industry. Keynote speaker, Lynne Yao, M.D., Director at the FDA Division of Pediatric and Maternal Health, Center for Drug Evaluation and Research, will discuss challenges and opportunities to accelerate global pediatric drug development. Additionally, the agenda features speakers who will speak about the regulatory environment, clinical trial innovation, and the changing landscape with the recent passing of the US FDA’s Research to Accelerate Cures and Equity for Children Act to expand and encourage pediatric drug development, specifically in oncology.

"Being passionate about paediatric development, I am very proud and honoured to be part of this symposium. This is the opportunity to bring together distinguished experts to share their views and experience, which will certainly benefit all involved," said Solange Corriol-Rohou, M.D., Senior Global Regulatory Policy Director at AstraZeneca. "It is amazing to see how paediatric development has evolved and been optimised these last few years. Multi-stakeholder collaboration has increased with the uptake of new tools and methods, such as digital technologies, extrapolation or quantitative approaches, for the benefit of children in need."

Iveric Bio Reports Second Quarter 2021 Operational Highlights and Financial Results

On August 4, 2021 IVERIC bio, Inc. (Nasdaq: ISEE) reported financial and operating results for the quarter ended June 30, 2021 and provided a general business update (Press release, Ophthotech, AUG 4, 2021, View Source [SID1234585722]).

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"This is an exciting time for the Company as we have delivered on several transformational milestones, particularly with regard to the execution of our Zimura pivotal program," stated Glenn P. Sblendorio, Chief Executive Officer of Iveric Bio. "For GATHER2, our second Zimura Phase 3 clinical trial for geographic atrophy secondary to age-related macular degeneration, we received a Special Protocol Assessment agreement from the FDA, we completed enrollment four months ahead of schedule, and patient retention, as measured by the injection fidelity rate, continues to exceed expectations and we are targeting the 12-month rate to be greater than 90%. In addition, the results of post-hoc analyses from GATHER1, our first Zimura Phase 3 clinical trial in GA secondary to AMD, indicate that Zimura may have a therapeutic benefit in earlier stages of GA."

"We are extremely proud of the momentum that we have created," stated Pravin U. Dugel, President of Iveric Bio. "During July 2021, we strengthened our balance sheet with a public offering. We believe this enables us to further support the impressive recruitment with superb retention in our Zimura GATHER2 Phase 3 trial, prepare and potentially file a New Drug Application for Zimura in GA secondary to AMD, begin preparations for a potential commercial launch of Zimura in GA secondary to AMD, initiate a drusen clinical development program, and invest in sustained release delivery technologies for Zimura. We are committed to execute on our strategy to develop and deliver treatments for diseases of the retina through our Zimura, HtrA1 inhibitor and gene therapy programs, with the potential to create long-term shareholder value."

Therapeutics Programs Targeting Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)

Zimura (avacincaptad pegol): Complement C5 Inhibitor

Special Protocol Assessment (SPA) for GATHER2
The Company announced on July 6, 2021, that it received written agreement from the U.S. Food and Drug Administration (FDA) under a Special Protocol Assessment (SPA) for the overall design of GATHER2, the Company’s second pivotal clinical trial of Zimura in development for the treatment of GA secondary to AMD. The agreement further solidifies the Company’s plans to file an application with the FDA for marketing approval of Zimura for GA secondary to AMD, if the ongoing GATHER2 clinical trial meets its primary endpoint at 12 months. Zimura met its pre-specified primary efficacy endpoint at 12 months and reached statistical significance in the previously completed GATHER1 pivotal clinical trial.

In parallel discussions with those for the GATHER2 SPA, the FDA indicated to the Company that, as part of a future NDA submission for Zimura, the GATHER1 results will be considered using the original prespecified primary efficacy endpoint analysis, together with a post-hoc analysis using the same FDA preferred method that will be used for the GATHER2 trial (mean rate of growth (slope) estimated based on GA area measured by fundus autofluorescence (FAF) in the relevant timepoints). The GATHER 1 results as analyzed by the FDA preferred analysis is highly consistent with and strongly supportive of the results from the original prespecified analysis. The complete results of both analyses for GATHER1 have been presented in the Company’s July 6, 2021 press release and Form 8-K filing.
GATHER2 Enrollment, Retention, and Injection Fidelity Rate
On July 26, 2021, the Company announced completion of enrollment in GATHER2, four months ahead of the Company’s original schedule. Based on this timeline, the Company expects topline GATHER2 data to be available in the second half of 2022, approximately one year after the enrollment of the last patient plus the time needed for database lock and analysis.

In June 2021, the Company announced that it is targeting patient retention for the GATHER2 trial, as measured by injection fidelity rate through month 12, of greater than 90%. The injection fidelity rate continues to exceed the Company’s expectations. Injection fidelity is calculated by dividing the total number of actual injections by the total number of expected injections based on the number of enrolled patients. The Company considers injection fidelity to be the most important and stringent measure of patient retention because it reflects the timely administration of the drug into the patient’s eye.
Earlier Stages of Dry AMD Prior to Geographic Atrophy
In June 2021, Vas Sadda, MD, of Doheny Eye Institute at UCLA, presented new post-hoc analyses from the GATHER1 trial on progression of drusen and nascent GA (iRORA/cRORA), which are earlier forms of dry AMD, in patients treated with Zimura 2 mg as compared to patients in the sham group. These post-hoc analyses suggest that Zimura may have the potential to impact AMD even before atrophy occurs, thereby changing the natural course of the disease. The Company expects to initiate a drusen clinical development program in 2022.
Autosomal Recessive Stargardt Disease
Patient enrollment in 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 25 new patients for a total of approximately 120 patients.
IC-500: HtrA1 (high temperature requirement A serine peptidase 1 protein) Inhibitor

During the second quarter of 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 investigational new drug application (IND) to the FDA for IC-500 in GA secondary to AMD in the second half of 2022.

Gene Therapy Programs in Orphan Inherited Retinal Diseases (IRDs)

IC-200: BEST1-Related IRDs
The Company has completed an IND-enabling preclinical toxicology study of IC-200, in a naturally occurring canine disease model of Best disease. Subject to regulatory review, the Company plans to initiate a Phase 1/2 clinical trial of IC-200 during the fourth quarter of 2021. The first IC-200 clinical trial will focus on patients with the autosomal recessive form of the disease, autosomal recessive bestrophinopathy.
IC-100: Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)
As previously disclosed, the Company was planning to discuss with the FDA the results from its toxicology studies of IC-100, and the design of its first-in-human clinical trial, before submitting an IND. The FDA advised, in lieu of this meeting, additional discussion should be conducted during the 30-day IND review period following IND submission. The Company is currently considering its development options for this product candidate.
Minigene Programs
The Company, through its minigene collaboration with the University of Massachusetts Medical School (UMMS), has identified a lead construct for its Leber Congenital Amaurosis Type 10 (LCA10) miniCEP290 program and is currently planning preclinical development for this program. The Company is evaluating preclinical data from its Stargardt Disease (ABCA4) program and expects to obtain preliminary results from its USH2A-related inherited retinal diseases (USH2A) program in the first half of 2022.

The Company recently hired four individuals who were previously at UMMS, including Hemant Khanna, Ph.D., the principal investigator for the Company’s miniCEP290, miniABCA4 and miniUSH2A sponsored research programs. Dr. Khanna joined the Company as Vice President, Pre-Clinical Ocular Research. The Company is working to transition the research and preclinical development activities for these programs from UMMS to the Company. The Company is also preparing to establish laboratory space for these employees to continue working on these programs and other preclinical research and development activities for the Company.
Management Updates

In August 2021, Christopher Simms joined Iveric Bio as Senior Vice President and Chief Commercial Officer. Mr. Simms is an accomplished healthcare leader with more than 20 years of diverse commercial leadership experience at Johnson & Johnson, Genentech, and Novartis, including focused experience in retina, ophthalmology, and optometry.
Second Quarter Financial Results and 2021 Cash Guidance

As of June 30, 2021, the Company had $159.9 million in cash and cash equivalents.
In July 2021, the Company raised approximately $108 million in net proceeds in an underwritten public offering of common stock. The Company sold 13,397,500 shares of its common stock in this public offering.
The Company now estimates its year-end 2021 cash, cash equivalents and available for sale securities to range between $215 million and $225 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 through at least mid-year 2024. These estimates are based on the Company’s current business plan including the continuation of its ongoing clinical development programs for Zimura, preparation and potential filing of a New Drug Application and a Marketing Authorization Application for Zimura in GA secondary to AMD, beginning preparations for a potential commercial launch of Zimura in GA secondary to AMD, initiating a drusen clinical development program, and investing in sustained release delivery technologies for Zimura, the progression of its IC-200 and IC-100 programs into the clinic, and the advancement of its IC-500 development program. Excluded from these estimates are any potential approval or sales milestones payable to Archemix Corp. and potential expenses for actual commercial launch of Zimura, any additional expenditures related to potentially studying Zimura in indications outside of GA and drusen or resulting from the potential in-licensing or acquisition of additional product candidates or technologies, and any associated development the Company may pursue.
2021 Q2 Financial Highlights

R&D Expenses: Research and development expenses were $23.5 million for the quarter ended June 30, 2021, compared to $12.7 million for the same period in 2020. For the six months ended June 30, 2021, research and development expenses were $42.0 million compared to $26.5 million for the same period in 2020. Research and development expenses increased primarily due to the commencement of patient enrollment and ongoing progress of the GATHER2 clinical trial, increased manufacturing activities for Zimura and increases in research and development personnel.
G&A Expenses: General and administrative expenses were $6.7 million for the quarter ended June 30, 2021, compared to $6.3 million for the same period in 2020. For the six months ended June 30, 2021, general and administration expenses were $15.0 million compared to $11.3 million for the same period in 2020. General and administration expenses increased primarily due to legal costs associated with continued litigation efforts.
Income Tax Benefit: The Company recorded no income tax benefit for the three and six months ended June 30, 2021. Income tax benefits of $0.4 million and $3.7 million for the three and six months ended June 30, 2020, respectively, were recognized to reflect a favorable settlement of a state corporate income tax audit.
Net Loss: The Company reported a net loss for the quarter ended June 30, 2021 of $30.1 million, or ($0.32) per diluted share, compared to a net loss of $18.6 million, or $(0.32) per diluted share, for the same period in 2020. For the six months ended June 30, 2021, the Company reported a net loss of $56.9 million or ($0.61) per diluted share, compared to a net loss of $33.7 million or ($0.61) 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 August 4, 2021 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 1-888-317-6003 (USA) or 1-412-317-6061 (International), passcode 7257034 . 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 1-877-344-7529 (USA) or 1-412-317-0088 , passcode 10158843.