On March 3, 2021 IVERIC bio, Inc. (Nasdaq: ISEE) reported financial and operating results for the fourth quarter and full year ended December 31, 2020 and provided a general business update (Press release, Ophthotech, MAR 3, 2021, View Source [SID1234576009]).

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The Company also announced that patient enrollment and retention for GATHER2, its second Phase 3 clinical trial for Zimura (avacincaptad pegol), a novel complement C5 inhibitor, for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) are progressing well with enrollment ahead of schedule. The Company is accelerating the timeline for when it expects to complete enrollment in GATHER2 to the third quarter of 2021. If the prespecified 12-month results from GATHER2 are positive, the Company plans to file applications with the results from GATHER1 and GATHER2 to the U.S. Food and Drug Administration and the European Medicines Agency for marketing approval of Zimura for GA.

"We start 2021 with significant momentum as we continue to enroll patients into our Zimura GATHER2 clinical trial and now expect to complete enrollment in the third quarter of this year," stated Glenn P. Sblendorio, Chief Executive Officer and President of Iveric Bio. "We are extremely encouraged by the progress of our clinical trial sites to enroll and retain patients. We continue to work with our investigators to provide a safe environment for patients, which we believe increases the patients’ comfort and confidence to participate in the GATHER2 clinical trial. We are committed to continuing patient enrollment and retention aggressively in the GATHER2 clinical trial while prioritizing patient safety."

Pravin U. Dugel, M.D., Chief Strategy and Business Officer of Iveric Bio added, "Although bringing Zimura to patients suffering from geographic atrophy secondary to age-related macular degeneration remains our top priority, in 2021 and thereafter we will continue to explore the potential development of Zimura in earlier stages of age-related macular degeneration as well as neovascular (wet) macular degeneration. Additionally, in our IC-500 program, we are pursuing HtrA1 inhibition as a target in the treatment of GA and potentially earlier stages of AMD. We are focused on advancing our pipeline of both therapeutic and gene therapy product candidates for treating retinal diseases with the potential to create long-term shareholder value."

Therapeutics Programs Targeting Age-Related Macular Degeneration

Zimura (avacincaptad pegol): Complement C5 Inhibitor

In April 2020, the U.S. FDA granted Fast Track designation for Zimura for the treatment of GA secondary to dry AMD.
In June 2020, the Company announced positive 18-month results from GATHER1, its first Phase 3 clinical trial for Zimura for the treatment of GA secondary to AMD. The 18-month data supports the previously announced 12-month data from this trial, at which time point Zimura met the pre-specified primary efficacy endpoint with statistical significance. Zimura was generally well tolerated after 18 months of administration.
In late June 2020, the Company announced that the first patient had been dosed in the GATHER2 clinical trial.
In September 2020, the Company announced that the positive 12-month Phase 3 results from its GATHER1 clinical trial with Zimura were published in Ophthalmology, the Journal of the American Academy of Ophthalmology.
In February 2021, Dr. Dugel presented the positive results from GATHER1 at the Angiogenesis, Exudation, Degeneration 2021 – Virtual Edition meeting.
The Company increased the enrollment target in its ongoing Phase 2b screening clinical trial of Zimura for the treatment of autosomal recessive Stargardt disease. After initially enrolling 95 patients in this trial, the Company plans to enroll approximately 25 additional patients, with the goal of enrolling a total of 120 patients.
IC-500: HtrA1 (high temperature requirement A serine peptidase 1 protein) Inhibitor

The Company recently revised its development plans for IC-500 to include plans to investigate multiple dosing schedules for this product candidate. Based on current timelines, the Company expects to submit an 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-100: Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)
The Company is preparing an IND for IC-100 and plans to meet with regulatory authorities to discuss its selected doses for a first-in-human clinical trial prior to the submission. The Company plans to file an IND for IC-100 with the FDA and begin enrolling patients in a Phase 1/2 clinical trial for IC-100 in the second half of 2021.
IC-200: BEST1-Related IRDs
The Company is completing IND-enabling activities for IC-200 and plans to file an IND for IC-200 with the FDA and begin enrolling patients in a Phase 1/2 clinical trial for IC-200 in the second half of 2021.
Minigene Programs
The Company, in collaboration with the University of Massachusetts Medical School (UMMS), continues to advance its minigene programs for Leber Congenital Amaurosis Type 10 (LCA10), autosomal recessive Stargardt Disease (ABCA4), and USH2A-related IRDs. The Company expects to select a lead construct for its LCA10 minigene program in the second quarter of 2021. The Company expects to obtain additional results from its Stargardt Disease minigene program in the first half of 2021. The Company expects to obtain preliminary results from its USH2A minigene program in the first half 2021.
Corporate Highlights

During 2020, the Company expanded its Board of Directors and management by adding a number of leading industry experts. Mark S. Blumenkranz, M.D., M.M.S., HJ Smead Professor Emeritus, Department of Ophthalmology, Stanford University joined the Company’s board of directors in July 2020. Pravin U. Dugel, M.D. joined the Company as Chief Strategy and Business Officer in March 2020. Dr. Dugel was previously Managing Partner, Retinal Consultants of Arizona and the Retinal Research Institute; Clinical Professor, USC Eye Institute, Keck School of Medicine, University of Southern California; and Founding Member, Spectra Eye Institute in Sun City, Arizona. Dhaval Desai, PharmD, joined the Company as Chief of Staff in August 2020. Previously, Mr. Desai served as Medical Unit Head at Novartis Eye Care.

In June 2020, the Company raised approximately $150 million in net proceeds in an underwritten public offering of common stock, and pre-funded warrants in lieu of common stock, and a concurrent private placement of common stock.

Fourth Quarter and Year End 2020 Operational Update and 2021 Cash Guidance

As of December 31, 2020, the Company had $210 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 will range between $130 and $140 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.
2020 Financial Highlights

R&D Expenses: Research and development expenses were $17.5 million for the quarter ended December 31, 2020, compared to $11.6 million for the same period in 2019. For the year ended December 31, 2020, research and development expenses were $62.8 million compared to $39.6 million for 2019. Research and development expenses increased primarily due to the initiation of GATHER2 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 $8.0 million for the quarter ended December 31, 2020, compared to $6.3 million for the same period in 2019. For the year ended December 31, 2020 general and administrative expenses were $26.0 million compared to $21.6 million for 2019. General and administration expenses increased primarily due to increases in general consulting costs and professional fees.
Income Tax (Benefit): Income tax benefits of $3.7 million and $0.1 million for the years ended December 31, 2020 and 2019, respectively, were recognized to reflect the favorable settlement of local tax audits.
Net Income: The Company reported a net loss for the quarter ended December 31, 2020 of $25.4 million, or ($0.27) per diluted share, compared to a net loss of $17.5 million, or ($0.39) per diluted share, for the same period in 2019. For the year ended December 31, 2020, the Company reported a net loss of $84.5 million or ($1.14) per diluted share, compared to a net loss of $58.9 million or ($1.39) for the same period in 2019.
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 March 3, 2021 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 888-221-3881 (USA) or 323-794-2590 (International), passcode 9597743. 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 888-203-1112 (USA), passcode 9597743.

On March 3, 2021 bioAffinity Technologies, a privately held company focused on early diagnostics and cancer therapeutics, reported that a scientific paper that explains how porphyrins are selectively incorporated into cancer cells has been published in the Journal of the Federation of Societies for the Study of Experimental Biology (FASEB Journal) (Press release, BioAffinity Technologies, MAR 3, 2021, View Source [SID1234576028]).

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"The research described in the FASEB paper and led by bioAffinity Technologies’ Vice President of Research, David Elzi, Ph.D., resulted in discoveries we have used to design new therapeutic approaches shown to kill cancer cells without harm to normal cells"

The paper, entitled Identification of a novel mechanism for meso‐tetra (4‐carboxyphenyl) porphyrin (TCPP) uptake in cancer cells, identified a novel mechanism by which the porphyrin TCPP, which is used in bioAffinity Technologies’ non-invasive test for the early detection of lung cancer, is incorporated into cancer cells using the cellular receptor CD320, which is involved in the uptake of Vitamin B12. In addition to furthering the understanding of TCPP for use in diagnostics, bioAffinity’s research led to four patent applications protecting multiple novel therapeutic platforms for treating cancer.

"The research described in the FASEB paper and led by bioAffinity Technologies’ Vice President of Research, David Elzi, Ph.D., resulted in discoveries we have used to design new therapeutic approaches shown to kill cancer cells without harm to normal cells," Maria Zannes, bioAffinity President and CEO, said. "We continue to advance this research with a focus on developing therapies for breast, lung and brain cancers."

In addition to its therapeutic research, bioAffinity Technologies is developing CyPath, a platform technology to diagnose cancers, including lung, prostate, bladder and cervical cancers, and diseases of the lung such as asthma and COPD. The Company’s first product is CyPath Lung, a non-invasive and cost-effective test for the early detection of lung cancer that allows patients to collect their sample at home.

bioAffinity Technologies recently completed a test validation trial of CyPath Lung evaluating sputum from people at high risk for lung cancer, including patients with the disease and others who were cancer-free. The trial resulted in 92% sensitivity and 87% specificity in high-risk patients who had nodules smaller than 2 cm or no nodules in the lung.

CyPath Lung uses flow cytometry, a method able to interrogate individual cells in a fraction of seconds, and automated analysis to identify parameters indicative of cancer. Unlike genomic or other molecular markers used in liquid biopsies, bioAffinity’s CyPath technology does not collect genetic material for evaluation. CyPath uses flow cytometry to investigate the micro-environment by identifying cell populations that are preferentially labeled by the porphyrin TCPP and other indices of cancer.

"The porphyrin TCPP is selectively taken up by cancer and cancer-associated cells and fluoresces. We use TCPP in CyPath Lung as a cellular label to help diagnose cancer in people at high risk for the disease," said Vivienne Rebel, M.D., Ph.D., Executive Vice President and Science and Medical Officer of bioAffinity Technologies. "Dr. Elzi led a team that investigated how TCPP enters cancer cells with an initial goal of better understanding the biological basis for the phenomenon we witness in CyPath Lung; that is, the labeling by TCPP of populations of cells in people with lung cancer. Dr. Elzi’s research has opened wholly new, exciting avenues for cancer therapeutics and diagnostics."

On March 3, 2021 Century Therapeutics, a leading cell therapy company developing induced pluripotent stem cell (iPSC)-derived cell therapies in immuno-oncology, reported the closing of a $160 million Series C financing (Press release, Century Therapeutics, MAR 3, 2021, View Source [SID1234576045]). The financing was led by Casdin Capital and brought together a syndicate of new investors including Fidelity Management & Research LLC, the Federated Hermes Kauffmann Funds, RA Capital, Logos Capital, OrbiMed, Marshall Wace, Qatar Investment Authority, Avidity Partners, and Octagon Capital. Founding investors Versant Ventures and Leaps by Bayer also participated. Eli Casdin, Founder and Chief Investment Officer of Casdin Capital will be joining the Century Therapeutics Board of Directors.

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"We are fortunate to be surrounded by such a top-tier group of investors, whose support will enable the acceleration of Century’s technology platform into the clinic," said Lalo Flores, Chief Executive Officer, Century Therapeutics. "With this new investor partnership, we are well-positioned to capitalize on the tremendous potential of our integrated iPSC, cell engineering and manufacturing capabilities to develop safer, more effective and more affordable next generation allogeneic cancer therapies."

Funds raised will help advance Century’s rich pre-clinical pipeline, which includes multiple iPSC-derived CAR-iT and CAR-iNK cell products. These products are designed to resist host rejection, enhance cell persistence, and allow repeat dosing to provide durable responses in all patients. The team anticipates beginning clinical testing of its first products in 2022, as well as generating multiple INDs annually in the coming years.

"It’s a remarkable and transformative time in the field, with the ability to engineer cells for therapeutic impact now a commercial reality. At the same time, iPSC technology has matured and is now leading the transition from bespoke autologous products to off-the-shelf allogeneic ones." said Eli Casdin CIO of Casdin Capital. "We could not be more excited to partner with the expert team at Century Therapeutics and do our part to help them leverage and accelerate their deep technical expertise in cellular reprogramming, differentiation, genetic engineering and manufacturing to deliver on this next phase of cellular therapy."

On March 3, 2021 Caribou Biosciences, Inc., a leading clinical-stage CRISPR genome editing biotechnology company, reported the successful completion of an oversubscribed $115 million Series C financing (Press release, Caribou Biosciences, MAR 3, 2021, View Source [SID1234575993]). Proceeds from the financing will be used to further develop the Company’s proprietary, next-generation CRISPR technology platform and to advance the Company’s pipeline of wholly-owned allogeneic immune cell therapies for oncology with best-in-class potential.

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The Series C financing was co-led by new premier healthcare investors Farallon Capital Management, PFM Health Sciences, and Ridgeback Capital Investments. Additional new investors include AbbVie Ventures, Adage Capital Partners LP, Avego Bioscience Capital, Avidity Partners, Invus, Janus Henderson Investors, LifeSci Venture Partners, The Leukemia & Lymphoma Society Therapy Acceleration Program (LLS TAP), Monashee Investment Management, LLC, Point72, and funds managed by Tekla Capital Management LLC. Existing investors participating in the round included Heritage Medical Systems, Maverick Ventures, and Pontifax Global Food and Agriculture Technology Fund (Pontifax AgTech). Santhosh Palani, Ph.D., Partner at PFM Health Sciences, and Jeffrey Long-McGie, Managing Director at Ridgeback Capital Investments, are joining Caribou’s Board of Directors.

"Caribou has successfully leveraged its next-generation CRISPR technology platform to create a promising clinical-stage therapeutic and a pipeline of pre-clinical allogeneic CAR-T and CAR-NK cell therapies that are potentially transformative for patients with unmet medical needs," said Jennifer Doudna, Ph.D., co-founder of Caribou. "Given its pioneering and selective approach in the field, Caribou’s CRISPR technology platform should continue to serve as a powerful engine for therapeutic development."

"We are excited to have the support of such an impressive group of new investors and proud of the continued commitment from current investors," said Rachel Haurwitz, Ph.D., Caribou’s President and Chief Executive Officer. "This funding will help fuel our continued clinical advancement and support our goal of bringing genome-edited immune cell therapies to patients as rapidly as possible."

Caribou has developed a next-generation CRISPR technology platform with substantial advantages in genome editing specificity and efficiency. The Company’s technology platform has fueled a pipeline of allogeneic cell therapies for oncology with best-in-class potential including enhanced persistence of its off-the-shelf cell therapies that is expected to drive the clinical durability of effect in multiple malignancies.

CB-010, Caribou’s lead allogeneic CAR-T cell program, targets CD19 and is being evaluated in a Phase 1 clinical trial for patients with relapsed/refractory B cell non-Hodgkin lymphoma. It is the first clinical-stage allogeneic CAR-T cell therapy in which PD-1 was genetically disrupted in the CAR-T genome, leading to more durable anti-tumor activity in pre-clinical studies. CB-011, Caribou’s second allogeneic CAR-T cell therapy, targets BCMA for the treatment of relapsed/refractory multiple myeloma and is immunologically cloaked for enhanced persistence. CB-012, Caribou’s third allogeneic CAR-T cell therapy, targets CD371 for the treatment of relapsed/refractory acute myeloid leukemia. Caribou is also developing iPSC-derived allogeneic natural killer (NK) cell therapies for solid tumor indications. Last month, Caribou announced a collaboration and license agreement with AbbVie for the research and development of two additional, unnamed CAR-T cell therapies.

"Caribou has built a remarkable and highly differentiated technology platform along with a pipeline of novel therapeutic candidates which hold breakthrough potential," said Dr. Palani. "We are delighted to support Caribou’s management team in the continued growth of the organization and the development of the first clinical candidates from its CAR-T cell program."

SVB Leerink acted as exclusive financial advisor for the Series C financing. Reed Smith LLP represented Caribou in the transaction.

About Caribou’s Pioneering Next-Generation CRISPR Platform

CRISPR genome editing uses easily designed, modular biological tools to make DNA changes in living cells. There are two basic components of CRISPR systems: the nuclease protein(s) that cut DNA and the RNA molecule(s) that guide the nuclease to generate a site-specific, double-stranded break, leading to editing at the targeted genomic site. CRISPR systems occasionally edit unintended genomic sites, known as off-target editing, which may lead to harmful effects on cellular function.

In response to this challenge, Caribou has developed chRDNAs (pronounced "chardonnays"), highly specific RNA-DNA hybrid guides that direct substantially more precise genome editing than all RNA guides. chRDNAs drive highly specific, multiplex genome editing including gene insertion. Caribou uses chRDNA guides in concert with various CRISPR enzymes to develop complex immune cell therapies.

Caribou is deploying chRDNAs to power the development of its CRISPR-edited therapies by guiding cellular editing with the highest level of fidelity.

On March 3, 2021 Merck (NYSE: MRK), known as MSD outside the United States and Canada, reported that Michael T. Nally, chief marketing officer, is scheduled to participate in the Barclays Global Healthcare Conference on March 9, 2021 at 1:50 p.m. EST (Press release, Merck & Co, MAR 3, 2021, View Source [SID1234576010]).

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Investors, analysts, members of the media and the general public are invited to watch a live video webcast of the presentations at View Source