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IVERIC bio Reports Third Quarter 2020 Operational Highlights and Financial Results

On November 2, 2020 IVERIC bio, Inc. (Nasdaq: ISEE) reported financial and operating results for the fiscal quarter ended September 30, 2020 and provided a general business update (Press release, Ophthotech, NOV 2, 2020, View Source [SID1234569666]).

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"It has been a landmark year as we have achieved several major milestones for Zimura," stated Glenn P. Sblendorio, Chief Executive Officer and President of IVERIC bio. "Over the past year, we announced positive 12-month and 18-month results from our GATHER1 Phase 3 clinical trial of Zimura for the treatment of geographic atrophy secondary to age-related macular degeneration. We initiated patient enrollment in GATHER2, our second Phase 3 clinical trial for the treatment of GA secondary to AMD. Additionally, we strengthened our balance sheet, advanced our two lead gene therapy product candidates to be on track to enter into the clinic next year and identified a lead compound for our HtrA1 inhibitor program."

Pravin U. Dugel, M.D., Chief Strategy and Business Officer of IVERIC bio added, "We are excited about the momentum generated with Zimura as a potential treatment for GA and, based on scientific data, Zimura‘s potential impact in wet (neovascular) AMD and in earlier stages of dry AMD. We are also encouraged by our HtrA1 inhibitor, IC-500, which we believe could be another important target in the treatment of AMD. Looking forward, our goal is to expand our footprint in multiple stages and types of AMD, dry and wet. We intend to do this by potentially studying Zimura in additional indications and by advancing the development of IC-500. We expect to make great strides in 2021, as we continue to move our pipeline of therapeutics and gene therapy product candidates forward."

Therapeutics Programs Targeting Age-Related Macular Degeneration

Zimura (avacincaptad pegol): Complement C5 Inhibitor
In September 2020, the Company announced that the positive Phase 3 results from its GATHER1 clinical trial with Zimura were published in Ophthalmology, the Journal of the American Academy of Ophthalmology. The published article is available online at View Source(20)30845-9/fulltext.
Today, the Company announced that the positive GATHER1 data from the first Zimura Phase 3 clinical trial will be presented at the 2020 American Academy of Ophthalmology – Retina Subspecialty Day Virtual Annual Meeting, November 13, 2020 at 6:10pm ET by Dr. Donald J. D’Amico, Professor and Chairman of Ophthalmology at Weill Cornell Medical College and Ophthalmologist-in-Chief at the New York Presbyterian Hospital.
The GATHER1 data were recently presented at the following virtual conferences:

Virtual EURETINA Annual Meeting, by Dr. Frank G. Holz, Professor and Chair of the Department of Ophthalmology, University of Bonn, Germany;
Retina Society Annual Meeting, by Dr. Carl D. Regillo, Chief, Retina Services at Wills Eye Hospital;
Meeting of the Club Jules Gonin, by Dr. Glenn J. Jaffe, Robert Machemer Professor of Ophthalmology and a member of the Vitreoretinal Faculty at Duke University Eye; and
American Society of Retina Specialists Annual Meeting, by Dr. Baruch D. Kuppermann, Chairman of the Department of Ophthalmology at UC Irvine.
In late June 2020, the Company announced that the first patient had been dosed in GATHER2, its second Phase 3 clinical trial for Zimura for the treatment of GA secondary to AMD. If 12-month results from GATHER2 are positive, the Company plans to file applications with the U.S. Food and Drug Administration and the European Medicines Agency for marketing approval of Zimura for GA.
IC-500: HtrA1 (high temperature requirement A serine peptidase 1 protein) Inhibitor Program

The Company selected a lead product candidate from its HtrA1 inhibitor program, which it will refer to as IC-500. Based on current timelines, the Company is planning to submit an IND to the FDA for IC-500 in GA secondary to AMD in the second half of 2021.
Gene Therapy Programs in Orphan Inherited Retinal Diseases (IRDs)

IC-100: Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa (RHO-adRP) and IC-200: BEST1-Related IRDs
IND-enabling activities for IC-100 and IC-200, and natural history studies for IC-200, are ongoing. The Company plans to file an IND for IC-100 with the FDA in early 2021 and begin enrolling patients in a Phase 1/2 clinical trial for IC-100 during the first half of 2021. The Company plans to file an IND for IC-200 with the FDA in the middle of 2021 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, 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 fourth quarter of 2020 or early 2021. The Company expects to obtain additional results from its Stargardt Disease minigene program in early 2021. The Company expects to obtain preliminary results from its USH2A minigene program in early 2021.
Corporate Update

In July 2020, Mark S. Blumenkranz, M.D., M.M.S., joined the Company’s board of directors. Dr. Blumenkranz is a biotechnology industry leader and internationally known Vitreo-Retinal Specialist with notable expertise in pharmaceuticals for age-related macular degeneration and ocular gene therapy.

Third Quarter 2020 Operational Update and Cash Guidance

As of September 30, 2020, the Company had $231.1 million in cash, cash equivalents and available for sale securities. The Company has revised its estimated year-end 2020 cash, cash equivalents and available for sale securities to range between $210 million and $215 million, reflecting the impact of a fourth quarter $6.0 million milestone payment to Archemix Corp. 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, through at least mid-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 Q3 Financial Highlights

R&D Expenses: Research and development expenses were $18.8 million for the quarter ended September 30, 2020, compared to $10.4 million for the same period in 2019. For the nine months ended September 30, 2020, research and development expenses were $45.3 million compared to $28.1 million for the same period in 2019. Research and development expenses increased primarily due to increased manufacturing and preclinical development activities associated with the Company’s IC-100 and IC-200 gene therapy programs, the completion of its GATHER1 clinical trial, the initiation of its GATHER2 clinical trial and the progression of its IC-500 development program.
G&A Expenses: General and administrative expenses were $6.6 million for the quarter ended September 30, 2020, compared to $4.7 million for the same period in 2019. For the nine months ended September 30, 2020, general and administrative expenses were $17.9 million compared to $15.4 million for the same period in 2019. General and administration expenses increased primarily due to increases in general consulting costs and professional fees.
Net Income: The Company reported a net loss for the quarter ended September 30, 2020 of $25.5 million, or ($0.27) per diluted share, compared to net loss of $14.4 million, or ($0.35) per diluted share, for the same period in 2019. For the nine months ended September 30, 2020, the Company reported a net loss of $59.1 million or ($0.87) per diluted share, compared to a net loss of $41.4 million or ($1.00) 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 November 2, 2020 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 866-575-6539 (USA) or 323-794-2575 (International), passcode 6339331. 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 6339331.

Bio-Techne To Present At The 29th Annual Credit Suisse Virtual Healthcare Conference

On November 2, 2020 Bio-Techne Corporation (NASDAQ:TECH) reported that Jim Hippel, Chief Financial Officer, will present at the 29th Annual Credit Suisse Virtual Healthcare Conference on Wednesday, November 11, 2020 at 4:15 p.m. EST (Press release, Bio-Techne, NOV 2, 2020, View Source [SID1234569665]). A live webcast of the presentation can be accessed via the IR Calendar page of Bio-Techne’s Investor Relations website at View Source

Fate Therapeutics to Webcast Conference Call Reporting Third Quarter 2020 Financial Results

On November 2, 2020 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported that the Company will host a conference call and live audio webcast on Thursday, November 5, 2020 at 5:00 p.m. ET to report its third quarter 2020 financial results and provide a corporate update (Press release, Fate Therapeutics, NOV 2, 2020, https://ir.fatetherapeutics.com/news-releases/news-release-details/fate-therapeutics-webcast-conference-call-reporting-third-6 [SID1234569664]).

In order to participate in the conference call, please dial (877) 303-6235 (domestic) or (631) 291-4837 (international) and refer to conference ID 1060606. To listen only, please dial (877) 638-9571 (domestic) or (760) 666-3586 (international) and refer to conference ID 1060606. The live webcast can be accessed under "Events & Presentations" in the Investors and Media section of the Company’s website at www.fatetherapeutics.com. The archived webcast will be available on the Company’s website beginning approximately two hours after the event.

VBL Therapeutics to Report Third Quarter 2020 Financial Results on November 16

On November 2, 2020 VBL Therapeutics (Nasdaq: VBLT), a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class treatments for cancer, reported that it will host a conference call and live audio webcast on Monday, November 16, 2020 at 8:30am Eastern Time to report third quarter ended September 30, 2020 financial results and to provide a corporate update (Press release, VBL Therapeutics, NOV 2, 2020, View Source [SID1234569663]).

Jounce Therapeutics Announces Update on Vopratelimab Program

On November 2, 2020 Jounce Therapeutics, Inc. (NASDAQ: JNCE), a clinical-stage company focused on the discovery and development of novel cancer immunotherapies and predictive biomarkers, reported an update on its vopratelimab (vopra) program (Press release, Jounce Therapeutics, NOV 2, 2020, View Source [SID1234569662]). The intent of the EMERGE Phase 2 trial was to induce ICOS hi CD4 T cells with ipilimumab, and then administer vopra with the goal of increasing proliferation and expansion of these ICOS hi CD4 T cells, which were associated with durable clinical benefit in the ICONIC trial of vopra alone and in combination with a PD-1 inhibitor. Early evaluation of the data from the EMERGE trial of vopra in combination with ipilimumab in PD-(L)1 inhibitor experienced non-small cell lung cancer (NSCLC) patients indicates the trial will not meet pre-specified interim criteria for continuation of enrollment. Therefore, the EMERGE trial will not be expanded. The company also announced the first patient dosed in the SELECT Phase 2 trial, supporting vopra’s potential in combination with JTX-4014 in immunotherapy naïve biomarker-selected NSCLC patients.

"We are disappointed that an early look at the EMERGE data indicates that we will not meet our pre-specified interim criteria for continued enrollment," said Beth Trehu, M.D., Chief Medical Officer at Jounce Therapeutics. "Less than 50% of the EMERGE patients had emergence of ICOS hi CD4 T cells after ipilimumab treatment, indicating that CD4 T cell activation by CTLA-4 inhibitors may be impaired in PD-(L)1 inhibitor resistant patients. Patients who have progressed on or after PD-1 inhibitor therapies represent a large and growing unmet need and new therapeutic mechanisms may be needed to more effectively treat these patients. We would like to thank the patients, investigators and study teams for their participation in the EMERGE trial."

"The PD-(L)1 experienced or resistant population continues to prove difficult to treat. To bring necessary benefit to these patients it is becoming clearer that novel approaches beyond T cells may be needed as part of the solution," said Richard Murray, Ph.D., chief executive officer and president of Jounce Therapeutics. "Our pipeline beyond vopra is focused on these novel approaches and we believe our existing programs, particularly, JTX-8064, our lead macrophage program targeting LILRB2 (ILT4), and other programs from our discovery platform, are poised to make meaningful contributions to both the PD-(L)1 naïve and resistant populations."

EMERGE Enrollment, Dosing and Safety
Fifty-nine patients were enrolled, 50 of whom are evaluable based on pre-specified criteria which required at least one dose of each drug and at least one CT scan for response assessment or clinical progression without a CT. Vopra continued to be safe and the combination with ipilimumab was well tolerated. The type and frequency of adverse events were similar to those seen with ipilimumab.

Summary of EMERGE Preliminary Efficacy Data

0.10 mg/kg vopratelimab + 3 mg/kg ipilimumab 0.03 mg/kg vopratelimab + 3 mg/kg ipilimumab 0.01 mg/kg* vopratelimab + 3 mg/kg ipilimumab All doses combined
Evaluable patients: n 22 18 10 50
Patients with tumor reduction: n (%) 12 (54.5) 3 (16.7) 4 (40.0) 19 (38.0)
Confirmed ORR: n (%) 1 (4.5) 0 (0.0) 0 (0.0) 1 (2.0)
OS: Median (95% CI**) NE*** (5.3, NE) NE (4.5, NE) 8.8 (1.7, NE) 11.6 (6.0, NE)
Patients remaining
on study 8 1 0 9
* Dose not protocol-specified, due to dosing error at a single study site; ** Confidence interval; *** Not estimable; Ipilimumab was dosed for a maximum of 4 doses. Data as of October 26, 2020

The data for all doses combined, with or without including the 10 subjects dosed at .01 mg/kg, did not meet the pre-specified criteria for continuation of enrollment in the trial, with tumor reduction in 38% of patients and one confirmed RECIST 1.1 response. In the .10 mg/kg cohort, 55% of patients had tumor reduction and confirmed overall response rate (ORR) is 4.5%. Median overall survival (OS) for all dose groups combined was 11.6 months and has not been reached in the .10 and .03 mg/kg cohorts. Nine patients remain on study, including four patients continuing to benefit on vopra alone after completion of up to four ipilimumab doses. The biomarker analysis is not yet complete.

EMERGE Interim Analysis Clinical Criteria to Expand Trial

Tumor reduction in ≥ 50% of patients,
Median overall survival tracking to ≥ 13 months, and
Overall response rate ≥ 10%
Interim analysis criteria were selected to provide preliminary evidence that a combination of vopra and ipilimumab could potentially be superior to docetaxel in a randomized Phase 3 trial with an overall survival endpoint. Early data evaluation indicates, across all EMERGE doses, these interim criteria cannot be met and EMERGE will not be expanded beyond current enrollment.

SELECT
SELECT and EMERGE were developed to address distinct hypotheses for the potential of vopra and ICOS hi CD4 cells to improve patient outcomes. These trials are also addressing different patient populations, PD-(L)1 inhibitor resistant versus PD-(L)1 inhibitor naïve and biomarker selected.

The randomized Phase 2 SELECT trial of vopra in combination with Jounce’s PD-1 inhibitor, JTX-4014 began enrollment in October 2020. The trial compares vopra plus JTX-4014 to JTX-4014 alone in immunotherapy naïve NSCLC patients who have been pre-selected with the TISvopra predictive biomarker. SELECT will enroll approximately 75 patients. TISvopra is an 18 gene RNA tumor inflammation signature which was optimized to predict the emergence of ICOS hi CD4 T cells and associated clinical benefit. SELECT is powered to demonstrate the statistical superiority of the combination of vopratelimab plus JTX-4014 compared to JTX-4014 alone. Preliminary efficacy data from the SELECT trial is expected in 2021.

Conference Call and Webcast Information:
Jounce Therapeutics will host a conference call and webcast today at 8:00 a.m. ET. To access the conference call, please dial (866) 916-3380 (domestic) or (210) 874-7772 (international) and refer to conference ID 8186260. The webcast can be accessed under "Events & Presentations" in the Investors and Media section of the Jounce website at www.jouncetx.com. The webcast will be archived and made available for replay on Jounce’s website approximately two hours after the call and will be available for 30 days.

About Vopratelimab
Vopratelimab is a clinical-stage monoclonal antibody that binds to and activates ICOS, the Inducible T cell CO-Stimulator, a protein on the surface of certain T cells commonly found in many solid tumors. Vopratelimab is currently being assessed in the SELECT Phase 2 clinical trial in combination with Jounce’s internal investigational PD-1 inhibitor, JTX-4014, compared to JTX-4014 alone. The SELECT trial is currently enrolling approximately 75 immunotherapy naïve NSCLC patients who have been pre-selected with the TISvopra predictive biomarker, an 18 gene RNA tumor inflammation signature which predicted the emergence of ICOS hi CD4 T cells and clinical benefit in the ICONIC trial of vopratelimab alone and in combination with a PD-1 inhibitor. SELECT is powered to demonstrate the statistical superiority of the combination of vopratelimab plus JTX-4014 compared to JTX-4014.