On August 1, 2019 IVERIC bio, Inc. (Nasdaq: ISEE) reported financial and operating results for the second quarter ended June 30, 2019 and provided a business update (Press release, Ophthotech, AUG 1, 2019, View Source [SID1234537986]).

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"We are pleased with the Company’s progress as we achieved several milestones to advance and expand our gene therapy pipeline for orphan inherited retinal diseases, or IRDs," stated Glenn P. Sblendorio, Chief Executive Officer and President of IVERIC bio. "We entered into exclusive global license agreements for rights to gene therapy product candidates for the treatment of BEST1-related IRDs and Leber congenital amaurosis type 10, or LCA10. We are encouraged by new research data from our miniCEP290 collaboration that support our plans to move this program forward in LCA10, and we expanded our gene therapy portfolio with the addition of a minigene research program for USH2A related IRDs, including Usher syndrome type 2A and USH2A-associated nonsyndromatic autosomal recessive retinitis pigmentosa. Manufacturing activities for IC-100 and IC-200, our two lead gene therapy product candidates, are currently ongoing using state-of-the-art manufacturing capabilities of Paragon Gene Therapy, part of Catalent Biologics."

Kourous A. Rezaei, MD, Chief Medical Officer of IVERIC bio added, "With the addition of the USH2A program to our portfolio, IVERIC bio is seeking to treat vision loss associated with what are reportedly the most common form of autosomal dominant retinitis pigmentosa (rhodopsin-mediated) and the most common form of autosomal recessive retinitis pigmentosa (USH2A-related). Retinitis pigmentosa is the most common orphan IRD. We look forward to presenting our gene therapy pipeline and discussing our programs in greater detail with highly-recognized gene therapy scientists and key opinion leaders in retinal diseases at our upcoming Gene Therapy R&D Investor Day."

Second Quarter 2019 and Recent Highlights

Orphan IRD Gene Therapy Programs

IC-100: Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)
Natural history studies and IND-enabling activities for IC-100 are ongoing. The Company expects to initiate a Phase 1/2 clinical trial for IC-100 in the second half of 2020.

IC-200: BEST1-Related IRDs
In April 2019, the Company entered into an exclusive global license agreement with the University of Pennsylvania and the University of Florida Research Foundation for rights to develop and commercialize novel adeno-associated virus (AAV) gene therapy product candidates for the treatment of BEST1-related IRDs, including Best vitelliform macular dystrophy, also known as Best disease. The Company expects to initiate a Phase 1/2 clinical trial for IC-200 in the first half of 2021.

miniCEP290: Leber Congenital Amaurosis Type 10 (LCA10)
Encouraging results from the Company’s collaboration with the University of Massachusetts Medical School (UMass Medical School) in its miniCEP290 program led the Company to exercise its option and, in July 2019, the Company entered into an exclusive global license agreement with the University of Massachusetts for rights to develop and commercialize mutation independent novel AAV minigene therapy product candidates for the treatment of LCA10, which is due to mutations in the CEP290 gene and is the most common type of LCA.

miniUSH2A: USH2A-Related IRDs Including Usher Syndrome Type 2A and USH2A-Associated Nonsyndromatic Autosomal Recessive Retinitis Pigmentosa
In July 2019, the Company entered into a sponsored research agreement with UMass Medical School and an exclusive option agreement with the University of Massachusetts for rights to develop and commercialize novel AAV gene therapy product candidates utilizing a mutation independent minigene therapy approach for the treatment of USH2A-related IRDs. This group of orphan IRDs include Usher syndrome Type 2A and USH2A-associated nonsyndromatic autosomal recessive retinitis pigmentosa.

Gene Therapy Manufacturing
In June 2019, the Company announced that it had entered into a strategic manufacturing relationship with Paragon Gene Therapy, part of Catalent Biologics. This agreement is for production and manufacturing of AAV vectors for pre-clinical activities and planned Phase 1/2 clinical trials for IC-100 and IC-200.

Gene Therapy R&D Investor Day Scheduled
The Company will host its first "Gene Therapy R&D Investor Day" in New York, NY on Friday, September 13, 2019. The agenda includes updates from IVERIC bio’s management on its gene therapy portfolio in orphan IRDs, and scientific discussions from gene therapy scientists and key opinion leaders in orphan inherited retinal diseases including;

Guangping Gao, PhD, University of Massachusetts Medical School

William A. Beltran, DVM, PhD, University of Pennsylvania

Charles A. Gersbach, Duke University

Hemant Khanna, PhD, University of Massachusetts Medical School

Gustavo D. Aguirre, VMD, PhD, University of Pennsylvania School of Veterinary Medicine

Andreas K. Lauer, MD, Oregon Health & Science University

Bart P. Leroy, MD, PhD, Ghent University and Children’s Hospital of Philadelphia

Alfred S. Lewin, PhD, University of Florida

Therapeutic Programs
The Company expects initial top-line data for its ongoing Phase 2b clinical trial of Zimura (avacincaptad pegol), a C5 complement inhibitor, for the treatment of geographic atrophy secondary to dry age-related macular degeneration to be available in the fourth quarter of 2019. The Company expects initial top-line data for its Phase 2b clinical trial of Zimura for the treatment of autosomal recessive Stargardt disease to be available in the second half of 2020.

Second Quarter 2019 Financial Results

Operational Update
As of June 30, 2019, the Company had $106.9 million in cash and cash equivalents. After taking into account the Company’s recent in-licensing transaction for its miniCEP290 program and sponsored research agreement for its miniUSH2A program, the Company reaffirms its estimate that year-end 2019 cash and cash equivalents will range between $80 million and $85 million. This estimate is based on its current 2019 business plan, including the continuation of its current research and development programs. This estimate does not reflect any additional expenditures, including associated development

costs, in the event the Company in-licenses or acquires any new product candidates or commences any new sponsored research programs.

R&D Expenses: Research and development expenses were $10.0 million for the quarter ended June 30, 2019, compared to $8.5 million for the same period in 2018. For the six months ended June 30, 2019, research and development expenses were $17.7 million compared to $16.2 million for the same period in 2018. Research and development expenses increased primarily due to increases in costs associated with the Company’s gene therapy programs and HtrA1 inhibitor program, offset by decreases in costs associated with the Company’s Zimura programs, personnel expenses and professional and consulting fees.

G&A Expenses: General and administrative expenses were $5.2 million for the quarter ended June 30, 2019, compared to $6.3 million for the same period in 2018. For the six months ended June 30, 2019, general and administrative expenses were $10.7 million compared to $12.0 million for the same period in 2018. General and administrative expenses decreased primarily due to decreases in costs to support the Company’s operations and infrastructure.

Net Income: The Company reported a net loss for the quarter ended June 30, 2019 of $14.4 million, or ($0.35) per diluted share, compared to a net loss of $13.2 million, or ($0.37) per diluted share, for the same period in 2018. For the six months ended June 30, 2019, the Company reported a net loss of $26.9 million or ($0.65) per diluted share, compared to a net loss of $26.3 million or ($0.73) for the same period in 2018.

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 1, 2019 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 800-458-4121 (USA) or 323-794-2597 (International), passcode 3528260. 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 Toll Free), passcode 3528260.

On August 1, 2019 Geron Corporation (Nasdaq: GERN) reported financial results for the second quarter ended June 30, 2019, and recent events (Press release, Geron, AUG 1, 2019, View Source [SID1234538010]). The Company ended the second quarter with $162.3 million in cash and marketable securities.

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During the second quarter, data from the Phase 2 portion of IMerge was reported at the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress that further support Geron’s late-stage development plans for the imetelstat program. Geron continues to plan to open the Phase 3 portion of IMerge in lower risk myelodysplastic syndromes (MDS) for screening and enrollment in August 2019.

"This past quarter, data presented at EHA (Free EHA Whitepaper) for the Phase 2 portion of IMerge reported higher efficacy responses from prior reported data for both 8-week and 24-week RBC-TI rates, highlighting the meaningful and durable transfusion independence potentially achievable with imetelstat treatment in heavily transfusion dependent lower risk MDS patients. Accordingly, we’re very much looking forward to the Phase 3 portion of IMerge opening for screening and enrollment later this month," said John A. Scarlett, M.D., Chairman and Chief Executive Officer. "In addition, a second EHA (Free EHA Whitepaper) presentation reported results of statistical analyses in which the months of median overall survival were more than double for imetelstat-treated relapsed/refractory MF patients in IMbark, compared to real-world data from closely matched patients treated with best available therapy. This provides additional support for our ongoing strategic evaluation of potential late-stage development approaches and regulatory scenarios in relapsed/refractory MF as we prepare for an End of Phase 2 meeting with the FDA, which we’re planning to conduct by the end of the first quarter of 2020."

Phase 3 Portion of IMerge Phase 2/3 Clinical Trial – Initial Site Initiations Complete and Trial to Open for Patient Screening and Enrollment in August 2019

In May, Geron completed the transfer of the imetelstat investigational new drug (IND) sponsorship from Janssen Biotech, Inc. (Janssen), and assumed complete development responsibility for imetelstat. Site initiations for the Phase 3 portion of IMerge were completed for several clinical sites in July 2019. Geron is planning for the trial to open for patient screening and enrollment later this month.

IMerge is a two-part Phase 2/3 clinical trial of imetelstat in transfusion dependent patients with lower risk MDS who are relapsed/refractory to erythroid stimulating agents (ESAs). The primary efficacy endpoint is the rate of red blood cell transfusion independence (RBC-TI) lasting at least eight weeks, or 8-week RBC-TI rate, which is defined as the proportion of patients achieving RBC-TI during any consecutive eight weeks since entry into the trial. Key secondary endpoints include the rate of RBC-TI lasting at least 24 weeks, or 24-week RBC-TI rate, durability of transfusion independence and the amount and relative change in RBC transfusions.

The Phase 3 portion of IMerge is designed as a randomized, double-blind, placebo-controlled clinical trial to test the hypothesis that imetelstat improves the rate of RBC-TI compared to placebo.

Approximately 170 patients are planned to be enrolled and randomized in a 2:1 ratio to receive either imetelstat or placebo. Many key aspects from the Phase 2 portion of IMerge remain the same for the Phase 3 portion. A target patient population of non-del(5q) lower risk MDS patients who were naïve to treatment with hypomethylating agents (HMAs) and lenalidomide was identified from the Phase 2 portion and will be enrolled in the Phase 3. In addition, the primary and secondary endpoints, the dose and schedule of imetelstat administration and many of the clinical sites remain the same as in the Phase 2. The Company is planning for the trial to be conducted at multiple medical centers globally, including North America, Europe, Middle East and Asia.

Second Quarter and Year-to-Date 2019 Results

For the second quarter of 2019, the Company reported a net loss of $14.2 million, or $0.08 per share, compared to $6.9 million, or $0.04 per share, for the comparable 2018 period. Net loss for the first six months of 2019 was $24.3 million, or $0.13 per share, compared to $14.1 million, or $0.08 per share, for the comparable 2018 period.

Revenues for the three and six months ended June 30, 2019 were $101,000 and $158,000, respectively, compared to $208,000 and $526,000 for the comparable 2018 periods. Revenues for the three and six months ended June 30, 2019 and 2018 included royalty and license fee revenues under various non-imetelstat license agreements. The decline in revenues reflects a reduction in the number of active research license agreements in 2019 related to the Company’s human telomerase reverse transcriptase, or hTERT, technology as a result of patent expirations on the underlying technology.

Total operating expenses for the three and six months ended June 30, 2019 were $15.3 million and $26.7 million, respectively, compared to $7.5 million and $15.2 million for the comparable 2018 periods. Research and development expenses for the three and six months ended June 30, 2019 were $10.1 million and $16.0 million, respectively, compared to $3.2 million and $5.6 million for the comparable 2018 periods. The increase in research and development expenses, compared to the same periods in 2018, primarily reflects costs for the transition of the imetelstat program, including resuming sponsorship of the ongoing imetelstat clinical trials; expenses for start-up activities for the Phase 3 portion of IMerge; and higher personnel-related costs for the expanding development team. General and administrative expenses for the three and six months ended June 30, 2019 were $5.2 million and $10.6 million, respectively, compared to $4.2 million and $9.6 million for the comparable 2018 periods. The increase in general and administrative expenses, compared to the same periods in 2018, primarily reflects higher corporate and patent legal costs and increased personnel-related expenses for additional headcount to support the development organization.

Interest and other income for the three and six months ended June 30, 2019 was $1.1 million and $2.3 million, respectively, compared to $717,000 and $1.1 million for the comparable 2018 periods. The increase in interest and other income, compared to the same periods in 2018, primarily reflects higher yields on the Company’s increased marketable securities portfolio.

The Company ended the second quarter of 2019 with $162.3 million in cash and marketable securities. Since May 2019, the Company has raised cumulative net cash proceeds of approximately $2.6 million from the sales of an aggregate of 1,893,091 shares of common stock under an At Market Issuance Sales Agreement, after deducting sales commissions and other offering expenses payable by Geron. The Company expects these net cash proceeds to provide additional financial flexibility as it advances the imetelstat development program. The funds will support future development costs, including conducting the Phase 3 portion of IMerge.

2019 Financial Guidance Reaffirmed

For fiscal year 2019, the Company expects total operating expenses to range from $80 to $85 million, of which approximately $20 to $25 million represents one-time costs that include imetelstat program transition activities from Janssen to Geron and purchase of drug product, drug substance and raw materials from Janssen to supply the Phase 3 portion of IMerge and prepare for new drug manufacturing. The Company continues to expect transition of the imetelstat program from Janssen to be completed by the end of the third quarter of 2019.

As of July 31, 2019, the Company had 38 employees, and plans to grow to a total of approximately 45 to 50 employees by year-end 2019, of whom half will be research and development personnel.

Conference Call

Geron will host a conference call to discuss second quarter and year-to-date financial results and recent events at 4:30 p.m. ET on Thursday, August 1, 2019.

Participants may access the conference call live via telephone by dialing domestically +1 (877) 303-9139 or internationally +1 (760) 536-5195. The conference ID is 2379646. A live, listen-only webcast will also be available on the Company’s website at www.geron.com/investors/events. If you are unable to listen to the live call, an archived webcast will be available on the Company’s website for 30 days.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic myeloid malignancies. Early clinical data suggest imetelstat may have disease-modifying activity through the suppression of malignant progenitor cell clone proliferation, which allows potential recovery of normal hematopoiesis. Ongoing clinical studies of imetelstat consist of IMerge, a Phase 2/3 trial in lower risk myelodysplastic syndromes (MDS) and IMbark, a Phase 2 trial in Intermediate-2 or High-risk myelofibrosis. Imetelstat has been granted Fast Track designation by the United States Food and Drug Administration for the treatment of patients with transfusion dependent anemia due to non-del(5q) lower risk MDS who are refractory or resistant to an erythroid stimulating agent.

On August 1, 2019 Bicycle Therapeutics plc, a biotechnology company pioneering a new class of therapeutics based on its proprietary bicyclic peptide (Bicycle) technology, reported that management will present at the Canaccord Genuity 39th Annual Growth Conference on Wednesday, August 7, 2019 at 1:30 p.m. EDT in Boston, MA (Press release, Bicycle Therapeutics, AUG 1, 2019, View Source [SID1234538042]).

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A live webcast of the presentation can be accessed in the Investors & Media section of Bicycle’s website at www.bicycletherapeutics.com. An archived replay of the webcast will be available for 90 days following the presentation date.

On August 1, 2019 ARCA biopharma, Inc. (Nasdaq: ABIO), a biopharmaceutical company applying a precision medicine approach to developing genetically-targeted therapies for cardiovascular diseases, reported financial results for the quarter ended June 30, 2019 and provided a corporate update (Press release, Arca biopharma, AUG 1, 2019, View Source [SID1234538067]).

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"We continue to make important progress on our lead development program Gencaro building on our GENETIC-AF Phase 2B clinical trial results, which were published in the Journal of the American College of Cardiology: Heart Failure," commented Dr. Michael Bristow, ARCA’s President and Chief Executive Officer. "The U.S. FDA recently agreed to amend the Special Protocol Assessment agreement for our Phase 3 trial to include heart failure patients with preserved ejection fraction (HFpEF) and a left ventricular ejection fraction (LVEF) up to 55%, the upper boundary of the LVEF range in GENETIC-AF for which Gencaro exhibited effectiveness for preventing atrial fibrillation/flutter. The revised PRECISION-AF target population, patients with recent atrial fibrillation and heart failure with mid-range and preserved ejection fractions, is an underserved population and we believe our Phase 2B data indicates that Gencaro may have a positive impact for these patients."

Pipeline Update

GencaroTM (bucindolol hydrochloride) – a pharmacologically unique beta-blocker and mild vasodilator being developed as a potential genetically-targeted treatment for atrial fibrillation (AF) in patients with heart failure (HF).

In April 2019, GENETIC-AF Phase 2B clinical trial results were published in the Journal of the American College of Cardiology: Heart Failure in the paper "GENETIC-AF: Bucindolol for the Maintenance of Sinus Rhythm in a Genotype-Defined Heart Failure Population".
In July 2019, the U.S. Food and Drug Administration (FDA) agreed to amend ARCA’s Special Protocol Assessment (SPA) agreement for the Phase 3 PRECISION-AF clinical trial to expand the target trial population to include heart failure patients with left ventricular injection fractions (LVEF) > 40% and < 55%. Subject to securing additional financing, ARCA anticipates initiating PRECISION-AF in the first quarter of 2020.
AB171 – a thiol-substituted isosorbide mononitrate being developed as a potential genetically-targeted treatment for heart failure (HF) and peripheral arterial disease (PAD).

Chemistry, manufacturing and controls (CMC) activities continued in the second quarter.
Subject to securing additional financing, IND-enabling non-clinical studies are anticipated to begin in the fourth quarter of 2019, and an IND submission is anticipated in the second half of 2020.
Second Quarter 2019 Summary Financial Results

Cash and cash equivalents were $9.97 million as of June 30, 2019, compared to $6.6 million as of December 31, 2018. ARCA believes that its current cash and cash equivalents will be sufficient to fund its operations, at its current cost structure, through the first quarter of 2020.

Research and development (R&D) expenses for the three months ended June 30, 2019 were $0.4 million compared to $1.2 million for the corresponding period of 2018. The decrease in R&D expenses was primarily due to decreased clinical expenses following the completion of the GENETIC-AF clinical trial in 2018.

General and administrative (G&A) expenses for the three months ended June 30, 2019 were $1.1 million, compared to $1.0 million in the second quarter of 2018. The Company expects G&A expenses in 2019 to be consistent with those in 2018 as it maintains administrative activities to support its ongoing operations.

Total operating expenses for the three months ended June 30, 2019 were $1.5 million compared to $2.2 million for the corresponding period of 2018. The decrease in total operating expenses was primarily attributable to the decrease in R&D expense due to the completion of the GENETIC-AF clinical trial in 2018.

Net loss was $1.4 million, or $1.14 per share, for the second quarter of 2019 compared to $2.1 million, or $2.74 per share, for the second quarter of 2018.

The Company will need to raise additional capital, and/or complete a partnership or other possible strategic transaction, to fund future operations and develop Gencaro or any other product candidates.

On August 1, 2019 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a physician-led biopharmaceutical company focused on immuno-inflammatory and dermatological diseases, reported it will report financial results for the second quarter 2019, Thursday, August 8th, after U.S. financial markets close (Press release, Aclaris Therapeutics, AUG 1, 2019, View Source [SID1234537994]).

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Management will conduct a conference call at 5:00 PM ET to discuss Aclaris’ financial results and provide a general business update. The conference call will be webcast live over the Internet and can be accessed by logging on to the "Investors" page of the Aclaris Therapeutics website, www.aclaristx.com, prior to the event. A replay of the webcast will be archived on the Aclaris Therapeutics website for 30 days following the call.

To participate on the live call, please dial (844) 776-7782 (domestic) or (661) 378-9535 (international), and reference conference ID 3391498 prior to the start of the call.