Iveric Bio Reports Fourth Quarter and Year End 2020 Operational Highlights and Financial Results

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.

Exicure Granted Orphan Drug Designation by the U.S. Food and Drug Administration for Cavrotolimod

On March 3, 2021 Exicure, Inc. (NASDAQ: XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) technology, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for its clinical product candidate, cavrotolimod (AST-008), for the treatment of patients with Merkel cell carcinoma (MCC) (Press release, Exicure, MAR 3, 2021, View Source [SID1234576008]).

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"We are excited to have been granted Orphan Drug Designation by the FDA for cavrotolimod for MCC," said Dr. Douglas Feltner, Chief Medical Officer of Exicure. "This designation marks significant progress toward our goal of fulfilling the unmet medical need and finding a treatment for Merkel cell carcinoma patients."

Exicure previously announced that the FDA granted two Fast Track designations for cavrotolimod (AST-008) for MCC and cutaneous squamous cell carcinoma (CSCC), both in the advanced/metastatic setting after progression on anti-PD-1/PD-L1 antibodies.

About Orphan Drug Designation

Orphan Drug Designation is a designation granted by the FDA which provides orphan status to drugs or biologics which are intended to treat rare diseases or disorders that affect fewer than 200,000 people in the United States. This designation provides the sponsor of the drug certain incentives, including tax credits for qualified clinical trials and fee waivers. Orphan Drug Designation confers eligibility for seven years of market exclusivity to an orphan drug post-approval, subject to a receipt by the FDA of marketing approval.

About Cavrotolimod (AST-008)

Cavrotolimod (AST-008) is an SNA consisting of toll-like receptor 9 agonists designed for immuno-oncology applications. In December 2019, Exicure announced preliminary results from the Phase 1b stage of the clinical trial including potential signs of anti-tumor activity with cavrotolimod in combination with pembrolizumab in cancer patients, including those with MCC. To date, 20 patients in the Phase 1b stage of the clinical trial have been dosed, and no cavrotolimod-related serious adverse event or dose-limiting toxicity has been reported. The most commonly reported adverse events were injection site reactions and flu-like symptoms. In the second quarter of 2020, Exicure initiated Phase 2 dose expansion cohorts of intratumoral cavrotolimod in combination with approved checkpoint inhibitors to treat two cohorts of patients with advanced or metastatic MCC or CSCC. Each cohort is expected to enroll up to 29 patients whose tumors have progressed on anti-PD-1/PD-L1, or programmed cell death protein 1/programmed death-ligand 1, antibody monotherapy.

Salarius Announces Proposed Public Offering of Common Stock

On March 3, 2021 Salarius Pharmaceuticals, Inc. (Nasdaq: SLRX), a clinical-stage biopharmaceutical company developing potential new medicines for patients with pediatric cancers, solid tumors, and other cancers, reported that it has commenced an underwritten public offering of shares of its common stock (Press release, Salarius Pharmaceuticals, MAR 3, 2021, View Source [SID1234576007]). The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

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Ladenburg Thalmann & Co. Inc. is acting as sole book-running manager in connection with the public offering.
A shelf registration statement on Form S-3 (File No. 333-231010) relating to the shares was filed with the Securities and Exchange Commission (the "SEC") and was declared effective by the SEC on May 17, 2019. A copy of the preliminary prospectus supplement and accompanying prospectus relating to the offering, when available, may be obtained be obtained at the SEC’s website at www.sec.gov or from Ladenburg Thalmann & Co. Inc., Attn: Prospectus Department, 640 Fifth Avenue, New York, NY 10019 or by email at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

PMV Pharmaceuticals Reports Fourth Quarter and Full Year 2020 Financial Results and Corporate Highlights

On March 3, 2021 PMV Pharmaceuticals, Inc. (Nasdaq: PMVP), a precision oncology company pioneering the discovery and development of small molecule, tumor-agnostic therapies targeting p53 mutants, reported financial results for the fourth quarter and full year ended December 31, 2020 and provided corporate highlights (Press release, PMV Pharma, MAR 3, 2021, View Source [SID1234576006]).

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"2020 was a transformative year for PMV Pharma, marked by our entry into the clinic, a strengthened management team and successful completion of our Nasdaq initial public offering," said David Mack, Ph.D., President and Chief Executive Officer. "The treatment of the first patient with PC14586, our tumor agnostic small molecule targeting the p53 Y220C mutant, was an important step forward in precision oncology. Our strong leadership and balance sheet position us to further advance our discovery pipeline of small molecule, tumor-agnostic precision medicine products that specifically target p53 mutants."

Corporate Highlights:

Initiated Phase 1/2 study of PC14586 in patients with advanced solid tumors that have a p53 Y220C mutation. The Phase 1/2 study will enroll up to 130 patients with a p53 Y220C mutation as determined by next generation sequencing. Phase 1 dose escalation will assess the safety, tolerability, pharmacokinetics, and preliminary anti-tumor activity of PC14586. Phase 2 will assess the overall response rate and duration of response at the PC14586 dose identified in Phase 1.
For information on the Phase 1/2 trial, please visit www.clinicaltrials.gov (NCT study identifier NCT04585750).
In October, the FDA granted PMV Pharma Fast Track designation to PC14586 for the treatment of patients with locally advanced or metastatic solid tumors that have a p53 Y220C mutation.
Appointment of Deepika Jalota, Pharm. D. to Chief Regulatory and Quality Officer, who joined PMV Pharma in June 2019 as Senior Vice President, Head of Regulatory Affairs and Quality Assurance. Dr. Jalota will continue to lead global regulatory strategy and execution as well as the quality assurance function. Dr. Jalota is a strategic regulatory leader with more than 20 years of regulatory affairs and drug development experience in oncology and other therapeutic areas. Prior to joining PMV Pharma, she served as Vice President, Global Regulatory Strategy, Oncology at Bayer HealthCare Pharmaceuticals where she was responsible for driving the development of global regulatory strategies for multiple early and late-stage oncology projects, including Xofigo, Vitrakvii and Nubeqa.
Expanded Scientific Advisory Board with the appointment of p53 pioneer Dr. Guillermina (Gigi) Lozano, Professor and Chair, Department of Genetics, MD Anderson Cancer Center.
Fourth Quarter 2020 Financial Results

PMV Pharma ended the fourth quarter with $361.4 million in cash, cash equivalents, and marketable securities, compared to $101.5 million as of December 31, 2019. Net cash used in operations was $32.7 million for the twelve months ended December 31, 2020 compared to $22.1 million for the twelve months ended December 31, 2019.
Net loss for the year ended December 31, 2020 was $34.4 million compared to $25.4 million for the year ended December 31, 2019.
Research and development (R&D) expenses were $23.9 million for the year ended December 31, 2020 compared to $20.8 million for the year ended December 31, 2019. The increase in R&D expenses was primarily related to increased headcount and clinical expenses related to advancing research on PC14586, the Company’s lead drug candidate.
General and administrative (G&A) expenses were $11.0 million for the year ended December 31, 2020, compared to $5.9 million for the year ended December 31, 2019. The increase in G&A expenses was primarily due to expanding the infrastructure necessary for operating as a public company.
About p53

p53 plays a pivotal role in preventing abnormal cells from becoming a tumor by inducing programmed cell death. Mutant p53 takes on oncogenic properties that endow cancer cells with a growth advantage and resistance to anti-cancer therapy. The p53 Y220C mutation is associated with many cancers, including but not limited to breast, non-small cell lung cancer, colorectal, pancreatic, and ovarian cancers.

About PC14586

PC14586 is a first-in-class, small molecule, p53 reactivator designed to selectively bind to the crevice created by the p53 Y220C mutant protein, hence, restoring the wild-type, or normal, p53 protein structure and tumor suppressing function. PC14586 is being developed for the treatment of patients with locally advanced or metastatic solid tumors that have a p53 Y220C mutation.

Avid Bioservices Declares Quarterly Dividend on Its Series E Convertible Preferred Stock

On March 3, 2021 Avid Bioservices, Inc. (NASDAQ:CDMO) (NASDAQ:CDMOP), a dedicated biologics contract development and manufacturing organization (CDMO) working to improve patient lives by providing high quality development and manufacturing services to biotechnology and pharmaceutical companies, reported that its Board of Directors has declared a quarterly cash dividend payment on the Company’s 10.50% Series E Convertible Preferred Stock (the "Series E Preferred Stock") (Press release, Avid Bioservices, MAR 3, 2021, View Source [SID1234576005]).

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The quarterly dividend on the Series E Preferred Stock is payable on April 1, 2021 to holders of record at the close of business on March 15, 2021.

The quarterly dividend payment on the Series E Preferred Stock will be $0.65625 per share, which is equivalent to an annualized 10.50% per share, based on the $25.00 per share stated liquidation preference, accruing from January 1, 2021 through March 31, 2021. The Series E Preferred Stock is listed on the NASDAQ Capital Market and trades under the ticker symbol "CDMOP".