On November 12, 2019 Fortress Biotech, Inc. (NASDAQ: FBIO) ("Fortress"), an innovative biopharmaceutical company focused on identifying, in-licensing and developing high-potential marketed and development-stage drugs and drug candidates, reported financial results and recent corporate highlights for the third quarter ended September 30, 2019 (Press release, Fortress Biotech, NOV 12, 2019, View Source [SID1234551047]).

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Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, "We are very pleased with the progress made during the third quarter of 2019 as we continue to focus on generating shareholder value in five distinct ways – through potential revenue and balance sheet growth, monetizations, priority review vouchers and future sales royalties. We recently acquired and launched Ximino, the second prescription oral antibiotic for acne in our marketed dermatology portfolio, which we expect will enable us to continue to grow the commercial side of our business. The last quarter of 2019 is expected to be marked by the achievement of additional milestones, notably the anticipated filing of a New Drug Application ("NDA") for IV tramadol for post-surgical pain management."

Dr. Rosenwald continued, "We are also pleased that MB-107, the lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency ("XSCID"), also known as bubble boy disease, being developed by our partner company Mustang Bio, was granted the Regenerative Medicine Advanced Therapy ("RMAT") designation by the U.S. Food and Drug Administration ("FDA"). We look forward to the presentation of additional clinical data on MB-107 at the upcoming 61st ASH (Free ASH Whitepaper) Annual Meeting and hope to achieve regulatory agreement with the FDA later this year to potentially expedite the development and approval of this critically needed treatment option for XSCID (bubble boy) patients. In addition, positive interim data for cosibelimab, an anti-PD-L1 antibody, were presented at the European Society for Medical Oncology ("ESMO") Congress 2019. The ongoing Phase 1 clinical trial of cosibelimab could support the submission of an initial Biologics License Application ("BLA") to the FDA. We are proud of our achievements during the third quarter and are well positioned to build on the momentum in order to continue to execute on our business plan into 2020 and beyond."

Financial Results:

·As of September 30, 2019, Fortress’ consolidated cash, cash equivalents, short-term investments (certificates of deposit), and restricted cash totaled $156.0 million, compared to $170.5 million as of June 30, 2019, and $99.2 million as of December 31, 2018, a decrease of $14.5 million for the quarter and an increase of $56.8 million year-to-date.
·Fortress’ net revenue totaled $9.8 million for the third quarter of 2019, which includes $9.5 million in net revenue generated from our marketed dermatology products. This compares to a total of $5.2 million in net revenue for the third quarter of 2018.
·Research and development expenses were $14.6 million for the third quarter of 2019, of which $13.9 million was related to Fortress partner companies. This compares to $16.1 million for the third quarter of 2018, of which $15.1 million was related to Fortress partner companies. Non-cash, stock-based compensation expenses included in research and development were $1.2 million for the third quarter of 2019, compared to $1.8 million for the third quarter of 2018.

·Research and development expenses from license acquisitions totaled $0.7 million for the third quarter of 2019, compared to $3.7 million for the third quarter of 2018.
·General and administrative expenses were $14.3 million for the third quarter of 2019, of which $9.3 million was related to Fortress partner companies. This compares to $12.2 million for the third quarter of 2018, of which $7.4 million was related to Fortress partner companies. Non-cash, stock-based compensation expenses included in general and administrative expenses were $2.5 million for the third quarter of 2019, compared to $2.3 million for the third quarter of 2018.
·Net loss attributable to common stockholders was $12.8 million, or $0.22 per share, for the third quarter of 2019, compared to a net loss attributable to common stockholders of $16.6 million, or $0.37 per share, for the third quarter of 2018. For the first nine months of 2019, net loss attributable to common stockholders was $24.5 million or $0.46 per share, compared to $59.3 million or $1.36 per share for the first nine months of 2018.

Recent Corporate Highlights1:

Marketed Dermatology Products

·In the third quarter of 2019, our marketed products generated net revenue of $9.5 million, compared to $5.2 million in the third quarter of 2018.
·Also, in the third quarter of 2019, we launched Ximino, a prescription oral antibiotic for acne.
·We anticipate 2019 annual net pharmaceutical revenues of approximately $30 million.
·We currently have 37 sales representatives dedicated to the dermatology product portfolio.
·We anticipate launching one to two new prescription products in 2020.
·Our dermatology products are marketed by our partner company, Journey Medical Corporation.

IV Tramadol

·In October 2019, an eAbstract was presented at ANESTHESIOLOGY 2019, the Annual Meeting of the American Society of Anesthesiologists in Orlando, FL, highlighting the Phase 3 data for IV tramadol in the management of post-surgical pain in patients undergoing bunionectomy, an orthopedic model. As previously announced, IV tramadol 50 mg met the primary endpoint, as well as all of the key secondary endpoints. We expect an NDA for IV tramadol to be filed with the FDA by year-end 2019.
·We anticipate that the Avenue Therapeutics merger with InvaGen Pharmaceuticals Inc. will be completed in 2021, if the conditions of the Stock Purchase and Merger Agreement are met, resulting in a potential net distribution to Fortress of approximately $48 million plus potential future product royalties.
·Also, in October 2019, IV tramadol Phase 1 clinical data were published in the peer-reviewed journal Clinical Pharmacology in Drug Development. The paper titled "Comparing the Pharmacokinetics of 2 Novel Intravenous Tramadol Dosing Regimens to Oral Tramadol: A Randomized 3-Arm Crossover Study", can be accessed here.
·IV tramadol is currently in development at our partner company, Avenue Therapeutics, Inc.

MB-107 (Lentiviral Gene Therapy for XSCID)

·In August 2019, MB-107, a lentiviral gene therapy for the treatment of XSCID, also known as bubble boy disease, was granted RMAT designation by the FDA.
·Also in August 2019, we entered into a license agreement with CSL Behring for the Cytegrity stable producer cell line, which will be used to produce the viral vector for the MB-107 lentiviral gene therapy program for the treatment of XSCID.
·Updated Phase 1/2 clinical data for MB-107 have been selected for oral and poster presentations at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, which will be held December 7-10, 2019, at the Orange County Convention Center in Orlando, FL.
·MB-107 is currently in development at our partner company, Mustang Bio, Inc.

1 Includes product candidates in development at Fortress, majority-owned and controlled partners and partners in which Fortress holds significant minority ownership positions. As used herein, the words "we," "us" and "our" may refer to Fortress individually or together with our affiliates and/or partners, as dictated by context.

CAEL-101

·In October 2019, the European Commission granted orphan drug designation to CAEL-101 for the treatment of AL amyloidosis. The U.S. Food and Drug Administration (FDA) had previously granted two orphan drug designations to CAEL-101 for the use of CAEL-101 as a therapeutic agent for patients with AL amyloidosis, and the use of CAEL-101 as a radio-imaging agent in amyloidosis.
·We received feedback from the FDA that supports our initiating a pivotal Phase 2/3 program stratified by Mayo stage beginning in 1H2020.
·CAEL-101 is currently in development at Caelum Biosciences, Inc., with its partner Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN).

Cosibelimab (formerly CK-301, anti-PD-L1 antibody)

·In September 2019, positive interim results for cosibelimab were presented at the ESMO (Free ESMO Whitepaper) Congress 2019 in Barcelona, Spain. The poster presentation provided updated interim efficacy and safety results from the ongoing multicenter Phase 1 clinical trial of cosibelimab, including expansion cohorts in cutaneous squamous cell carcinoma ("CSCC") and non-small cell lung cancer ("NSCLC"). A 50% objective response rate was observed in CSCC, and a 40% objective response rate was observed in NSCLC. Cosibelimab appeared to be safe and well-tolerated with a potentially favorable safety profile as compared to the currently available anti-PD-1 therapies.
·CSCC enrollment to support a potential initial BLA submission for cosibelimab is ongoing.
·In November 2019, we announced that pharmacokinetic and target occupancy modeling data for cosibelimab were presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 34th Annual Meeting. The poster, entitled, "Semi-mechanistic PK and target-occupancy modeling to support dose justification for anti-PD-L1 clinical candidate CK-301 (TG-1501) in oncology patients," compares pharmacokinetic and tumor target occupancy data at steady state under various dosing regimens of cosibelimab to those of three marketed anti-PD-L1 monoclonal antibodies, atezolizumab, durvalumab and avelumab. The results demonstrate that cosibelimab dosed at 800 mg and 1200 mg once every two weeks or every three weeks is expected to achieve over 99% PD-L1 target occupancy throughout the dosing interval, which is comparable to atezolizumab and durvalumab, and higher than avelumab, at approved doses.
·Cosibelimab is currently in development at our partner company, Checkpoint Therapeutics, Inc.

MB-101 (IL13Rα2-targeted CAR T cell therapy)

In October 2019, we announced that City of Hope received $4.1 million in grant awards for a clinical trial of MB-101 (IL13Rα2-targeted CAR T cell therapy) in combination with nivolumab (commercial name: Opdivo) and ipilimumab (commercial name: Yervoy) in patients with recurrent malignant glioma. The trial, which is now enrolling patients, is the first human study to combine IL13Rα2-targeted CAR T cell therapy with checkpoint inhibitors, as well as the first to locally deliver CAR T cells with systemic nivolumab combination treatment.

·MB-101 is currently in development at our partner company, Mustang Bio, Inc.

MB-103 (HER2-targeted CAR T cell therapy)

In August 2019, we announced that the California Institute for Regenerative Medicine ("CIRM") granted City of Hope $9.28 million to fund an ongoing Phase 1 clinical trial of MB-103 (HER2-targeted CAR T cell therapy) for the treatment of HER2-positive breast cancer with brain metastases.

·MB-103 is currently in development at our partner company, Mustang Bio, Inc.

MB-105 (Prostate Stem Cell Antigen (PSCA)-targeted CAR T cell therapy)

In September 2019, we announced that City of Hope opened and has begun to treat its first patients in a Phase 1 clinical trial of MB-105 (PSCA-targeted CAR T cell therapy) for the treatment of prostate cancer.

·MB-105 is currently in development at our partner company, Mustang Bio, Inc.

MB-106 (CD20-targeted CAR T cell therapy)

Fred Hutchinson Cancer Research Center will present a poster about the design of the ongoing Phase 1/2 clinical trial investigating the safety and efficacy of MB-106 (CD20-targeted CAR T cell therapy) for high-risk B-cell non-Hodgkin lymphomas at the 61st ASH (Free ASH Whitepaper) Annual Meeting, which will be held December 7-10, 2019, at the Orange County Convention Center in Orlando, FL. The abstract is available here.

·MB-106 is currently in development at our partner company, Mustang Bio, Inc.

MB-108 (Oncolytic Virus C134)

·In October 2019, we announced that the first participant was dosed in a Phase 1 clinical trial to determine the safety and efficacy of MB-108 (oncolytic virus C134), an attenuated herpes simplex virus type 1, in recurrent glioblastoma multiforme.
·MB-108 is currently in development at our partner company, Mustang Bio, Inc.

General Corporate

·In August 2019, we announced the appointment of Kevin L. Lorenz, J.D., to our Board of Directors.

On November 12, 2019 TG Therapeutics, Inc. (NASDAQ: TGTX) reported its financial results for the third quarter ended September 30, 2019 and recent company developments (Press release, TG Therapeutics, NOV 12, 2019, View Source [SID1234551046]).

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Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer, stated, "In the third quarter our team continued to execute on our core mission of developing combination therapies for patients with B-cell diseases. With the announcement of positive marginal zone lymphoma data earlier this year, and positive follicular lymphoma data only a few weeks ago, we have our first two foundational datasets for which we believe approval is in our reach. Adding on top of this, will be our UNITY-CLL Phase 3 study, which we are targeting a PFS readout around year-end or into the first quarter of next year. With these core pieces in place, our broader combination approach should come into focus, as we execute on a strategy to position U2 as an important stand-alone treatment as well as one that can improve outcomes when combined with other available therapies. We believe our recent ASH (Free ASH Whitepaper) abstracts contain some initial insights into the future of U2." Mr. Weiss continued, "With a pro forma cash position of approximately $100 million at the end of the third quarter, we believe we have sufficient capital resources through our next two major pivotal data releases including UNITY-CLL and the ULTIMATE MS Phase 3 trials."

Recent Developments and Highlights

ASH Presentations: Two triple therapy data abstracts were accepted for presentation at the upcoming 61stAmerican Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting, including an oral presentation for the triple combination of U2 (umbralisib and ublituximab) plus venetoclax, and Phase 1 data for TG-1701, the Company’s novel BTK inhibitor, monotherapy and in combination with U2.
Positive Interim Data from FL Cohort of UNITY-NHL Trial: Positive interim data from the follicular lymphoma (FL) cohort of the UNITY-NHL trial was announced, with results meeting the Company’s prespecified ORR target. The Company plans to present the data at a future medical conference as well as discuss the data with the U.S. Food and Drug Administration (FDA).
GENUINE Progression Free Survival (PFS): Final long-term results from the Phase 3 GENUINE study demonstrated that ublituximab in combination with ibrutinib improved progression-free survival (PFS), as determined by Independent Review Committee (IRC).
U2 Published in Blood: Phase I/Ib combination trial of U2 was published in Blood, the Journal of the American Society of Hematology (ASH) (Free ASH Whitepaper).
Ublituximab Data in Multiple Sclerosis: Updated Phase 2 extension trial data for ublituximab in relapsing forms of multiple sclerosis (RMS), as well as the ULTIMATE I & II Phase 3 RMS program study design and demographic data was presented at the 35th Annual Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS).
Remaining 2019 and Early 2020 Milestones

Initiate a rolling New Drug Application (NDA) submission for umbralisib to treat adult patients with previously treated marginal zone lymphoma (MZL).
Report potential top-line PFS results from the Phase 3 UNITY-CLL trial evaluating U2 in patients with frontline and previously treated CLL.
Share results from the Phase 2b UNITY-NHL FL cohort with the FDA to determine potential filing opportunities.
Financial Results for the Three and Nine Months Ended September 30, 2019

R&D Expenses: Other research and development (R&D) expense (not including non-cash compensation and non-cash in-licensing expense) was $56.5 million and $118.8 million for the three and nine months ended September 30, 2019, respectively, compared to $32.8 million and $98.7 million for the three and nine months ended September 30, 2018, respectively. The increase in R&D expense is primarily attributable to an increase in manufacturing expenses for Phase 3 clinical trials and potential commercialization (incurred during 2019) of $27 million and $31.6 million during the three and nine months ended September 30, 2019, respectively, as compared to prior periods. This was partially offset by a decrease in clinical trial expense of $3.8 million and $14.2 million for the three and nine months ended September 30, 2019, respectively, as compared to prior periods. We expect our other R&D expenses to decrease during the remainder of 2019 and into 2020 as our clinical trial expenses continue to decrease and the bulk of our CMC expenditures have been incurred during 2019.

G&A Expenses: Other general and administrative (G&A) expense (not including non-cash compensation) was $2.3 million and $6.6 million for the three and nine months ended September 30, 2019, respectively, as compared to $1.8 million and $6.2 million for the three and nine months ended September 30, 2018, respectively. Other G&A expenses remained consistent with the prior period, and we expect Other G&A expenses to increase modestly through the remainder of 2019.

Net Loss: Net loss was $61.9 million and $133.3 million for the three and nine months ended September 30, 2019, respectively, compared to a net loss of $34.0 million and $119.6 million for the three and nine months ended September 30, 2018, respectively. Excluding non-cash items, the net loss for the three and nine months ended September 30, 2019 was approximately $59.9 million and $127.5 million. Cash used in operations for the three months ended September 30, 2019 was approximately $33 million, as the payments of much of the increase in manufacturing expenses were deferred 12 months, having little impact on the quarter’s cash utilization.

Cash Position and Financial Guidance: Cash, cash equivalents and investment securities were $72.5 million as of September 30, 2019. Pro forma cash, cash equivalents and investment securities as of September 30, 2019 (excluding our fourth quarter 2019 operations) are approximately $96.3 million, after giving effect to $23.8 million of net proceeds from the utilization of the Company’s at-the-market ("ATM") sales facility during the fourth quarter of 2019. The Company believes its cash, cash equivalents and investment securities on hand as of September 30, 2019, inclusive of the proceeds raised from the ATM facility subsequent to the third quarter, as well as future availability under the ATM facility, will be sufficient to fund the Company’s planned operations into the fourth quarter of 2020.
Conference Call Information

The Company will host a conference call today, November 12, 2019, at 8:30 am ET, to discuss the Company’s third quarter 2019 financial results and provide a business outlook for the remainder of 2019.

In order to participate in the conference call, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), Conference Title: TG Therapeutics Third Quarter 2019 Business Update Call. A live audio webcast will be available on the Events page, located within the Investors & Media section, of the Company’s website at View Source An audio recording of the conference call will also be available for replay at www.tgtherapeutics.com, for a period of 30 days after the call.

On November 12, 2019 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported that Troy Wilson, Ph.D., J.D., President and Chief Executive Officer, is scheduled to present at the Stifel Healthcare Conference on Tuesday, November 19, 2019 at 1:15 p.m. ET / 10:15 a.m. PT (Press release, Kura Oncology, NOV 12, 2019, View Source [SID1234551045]). The conference will be held November 19-20, 2019 in New York City.

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A live audio webcast of the presentation will be available in the Investors section of Kura’s website at www.kuraoncology.com, with an archived replay available for 30 days following the event.

On November 12, 2019 Precision BioSciences, Inc. (Nasdaq: DTIL) ("Precision"), a genome editing company dedicated to improving life through the application of its pioneering, proprietary ARCUS platform, reported financial results for the third quarter ended September 30, 2019 and provided a corporate update (Press release, Precision Biosciences, NOV 12, 2019, View Source [SID1234551044]).

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Key Highlights

After quarter end, announced publication of an abstract accepted for presentation at the upcoming 61st Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in Orlando, Florida, December 7-10, 2019, supporting the safety and clinical activity of Precision BioSciences’s lead CD19-targeted off-the-shelf (allogeneic) chimeric antigen receptor (CAR) T product candidate, PBCAR0191. Abstract reports initial data from first three patients treated at Dose Level 1 as of August 1, 2019. Trial is ongoing and updated data, including from patients treated at Dose Level 2, will be presented during the ASH (Free ASH Whitepaper) annual meeting on December 9, 2019 at 6:00 pm ET.

Announced an investigator update event during ASH (Free ASH Whitepaper) meeting to discuss the PBCAR0191 data presented, starting at 8:15pm ET on December 9, 2019, with accompanying live webcast.

Received acceptance from US Food and Drug Administration (FDA) of investigational new drug (IND) application for second and wholly-owned allogeneic CAR T therapy candidate PBCAR20A, targeting CD20. Phase 1/2a clinical trial to begin in the fourth quarter of 2019.

Further enhanced senior leadership team with appointment of David Thomson, PhD, as Chief Operating Officer, and Nicholas Riddle, MD, PhD, as Vice President, Financial Strategy and Investor Relations.

Ended the quarter with $206.3 million in cash and cash equivalents, which is expected to fund operating expenses and capital expenditure requirements into 2021.

"We reached a transformative moment for Precision BioSciences this quarter; it is very exciting to report initial data from our first clinical trial with a product candidate that leverages our unique approach to allogeneic CAR T therapy," commented Matt Kane, Chief Executive Officer and Co-Founder of Precision BioSciences. "These data bring the reality of a true off-the-shelf CAR T therapy a step closer for patients in need of new and improved treatment options. While preliminary and from a limited number of patients, the safety profile, in vivo cell expansion and early evidence of clinical activity we have demonstrated with PBCAR0191 in the absence of biologic lymphodepletion is very encouraging and gives us confidence in the approach we have taken to allogeneic CAR T. We are looking forward to sharing updated results from patients treated at both Dose Level 1 and Dose Level 2 at ASH (Free ASH Whitepaper). The team at Precision is committed to advancing our pipeline of differentiated CAR T product candidates as rapidly as possible to bring these potentially transformative therapies to patients."

Recent Developments and Upcoming Milestones

Program updates

On November 6, 2019, Precision announced that initial results from the ongoing Phase 1/2a trial of its lead investigational allogeneic CAR T cell therapy candidate, PBCAR0191, will be presented during the 61st Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in Orlando, Florida, December 7-10, 2019. PBCAR0191, which is being developed in collaboration with Servier, is Precision’s first allogeneic CAR T therapy candidate in clinical trials and targets the well characterized cancer cell surface protein CD19. The Phase 1/2a trial includes adult patients with relapsed or refractory (R/R) non-Hodgkin’s lymphoma (NHL) or R/R B-cell precursor acute lymphoblastic leukemia (B-ALL). The abstract outlining initial data from patients treated with PBCAR0191 at Dose Level 1 can be accessed on the ASH (Free ASH Whitepaper) conference website. Data in the abstract include results as of the cutoff date of August 1, 2019 for three patients with advanced NHL treated at Dose Level 1. No significant toxicities were observed, including no serious adverse events and no dose-limiting toxicities. All patients had a minimum follow-up of 28 days (median 60 days). Two of the three patients experienced an objective tumor response by Lugano criteria, at day 14 and day 28, respectively. The third patient, who had previously progressed following treatment with axicabtagene ciloleucel (Yescarta), an approved anti-CD19 autologous CAR T therapy, had not met the definition of response, but demonstrated evidence of central necrosis, decreased tumor size, and decreased PET-avidity at day 28, in the context of post-infusion tumor site pain and mild CRS symptoms. Peripheral blood analysis for CAR T cell expansion has identified preliminary evidence of cell expansion.

The PBCAR0191 Phase 1/2a trial is ongoing and updated results from patients treated at Dose Level 1 and Dose Level 2 will be presented at the ASH (Free ASH Whitepaper) annual meeting on December 9, 2019 starting at 6:00 p.m. ET. Precision will host a live webcast of an investigator update event during the ASH (Free ASH Whitepaper) meeting to discuss the presented data, beginning at 8:15 p.m. ET on December 9, 2019. The webcast will be accessible from the "Events & Presentations" page within the Investors & Media section of the Precision website and a replay will be available for 30 days following the call.

On September 16, 2019, Precision announced that the FDA accepted its IND application for PBCAR20A. Wholly-owned by Precision, PBCAR20A is an allogeneic anti-CD20 CAR T therapy candidate in development for the treatment of patients with NHL, chronic lymphocytic leukemia (CLL), and small lymphocytic lymphoma (SLL). Precision plans to initiate a Phase 1/2a clinical trial in the fourth quarter of 2019, with initial data expected in 2020.

Senior leadership appointments

On September 30, 2019, Precision announced that Nicholas Riddle, MD, PhD, joined as Vice President, Financial Strategy and Investor Relations. Dr. Riddle joined Precision from J.P. Morgan where he was an Executive Director in the global healthcare investment banking group.

On September 23, 2019, Precision appointed David Thomson, PhD, to the position of Chief Operating Officer. Dr. Thomson previously served as Precision’s Chief Development Officer since 2017.

Upcoming Corporate Presentations

Precision’s senior management team will be presenting and meeting with investors at the following upcoming conferences:

Barclays Gene Editing and Gene Therapy Summit, New York, NY, November 13, 2019

Stifel Healthcare Conference, New York, NY, November 20, 2019

Jefferies London Healthcare Conference, London, UK, November 20, 2019

Piper Jaffray Annual Health Care Conference, New York, NY, December 3–5, 2019

J.P. Morgan Healthcare Conference, San Francisco, CA, January 13-16, 2020

Third Quarter 2019 Financial Results

Cash and Cash Equivalents: As of September 30, 2019, Precision had approximately $206.3 million in cash and cash equivalents. We expect that existing cash and cash equivalents will be sufficient to fund operating expenses and capital expenditure requirements into 2021.

Revenues: Total revenues for the quarter ended September 30, 2019 were $4.9 million, compared to $2.5 million for the quarter ended September 30, 2018. This increase was primarily due to research funding from a collaboration partner, offset by a decrease in license fees.

Research and Development Expenses: Research and development expenses were $19.8 million for the quarter ended September 30, 2019, as compared to $9.7 million for the same period in 2018. This increase of $10.1 million was primarily due to platform development and early-stage research expenses.

General and Administrative Expenses: General and administrative expenses were $7.1 million for the quarter ended September 30, 2019, as compared to $3.3 million for the same period in 2018. The increase of $3.8 million was primarily due to increased employee-related costs for additional personnel and facility costs associated with our growing infrastructure needs.

Net Loss: Net loss was $20.7 million, or $(0.41) per share, for the quarter ended September 30, 2019, compared to a net loss of $9.8 million, or $(0.62) per share, for the same period in 2018.

On November 12, 2019 IVERIC bio, Inc. (Nasdaq: ISEE) reported further details of the clinical trial results for the Company’s Zimura (avacincaptad pegol) program in geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD), reviewed the Company’s financial results for the third quarter 2019 and provided a general business update (Press release, Ophthotech, NOV 12, 2019, View Source [SID1234551043]).

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Zimura (avacincaptad pegol) Highlights

On October 28, 2019, IVERIC bio provided topline data confirming that Zimura (avacincaptad pegol), the Company’s complement factor C5 inhibitor, met its prespecified primary endpoint in reducing the mean rate of geographic atrophy (GA) growth in patients with dry age-related macular degeneration (AMD). Today, the Company provided further clinical details and the development strategy for Zimura in GA secondary to AMD. These announcements will be discussed during today’s conference call/webcast (also see the second press release issued earlier today and the press release issued on October 28, 2019.)

"We are excited to have achieved a major milestone with our recent Zimura clinical trial results in geographic atrophy secondary to dry AMD and we look forward to start enrolling patients in a second pivotal clinical trial in the first quarter of 2020. We believe these events have the potential to be a catalyst for our company," stated Glenn P. Sblendorio, Chief Executive Officer and President of IVERIC bio. "IVERIC bio is now in a strong position, with a diversified, retina-focused portfolio, including both a late stage clinical program for a large market retinal disease and early stage gene therapy programs in inherited retinal diseases. We are committed to efficiently progressing these programs with the goal of continuing to create value for our shareholders."

Gene Therapy 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 on track. The Company expects to initiate a Phase 1/2 clinical trial for IC-100 in patients with rhodopsin mediated adRP in the second half of 2020.

IC-200: BEST1-Related IRDs
Natural history studies and IND-enabling activities for IC-200 are on track.

The Company expects to initiate a Phase 1/2 clinical trial for IC-200 in patients with BEST1 related retinal diseases during 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. The Company plans to provide an update on the lead minigene construct in early 2020.

miniABCA4 Program for Stargardt Disease (STGD1)
The Company, through its collaborative sponsored research agreement with UMass Medical School, is evaluating several ABCA4 minigene constructs in both in vitro and in vivo experiments. The Company expects to have results from the miniABCA4 program in 2020.

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.

On October 29, 2019, Abraham Scaria, PhD was appointed to the position of Chief Scientific Officer. Dr. Scaria will lead the Company’s research and pre-clinical gene therapy activities. Dr. Scaria’s extensive experience includes positions at Genzyme, Sanofi and most recently at Casebia Therapeutics, leading multiple ocular gene therapy programs.

Second Quarter 2019 Financial Results
Operational Update
As of September 30, 2019, the Company had $94.9 million in cash and cash equivalents. The Company reaffirms its estimate that year-end 2019 cash and cash equivalents will range between $80 million and $85 million. With the initiation of enrollment for the Company’s second pivotal clinical trial for Zimura in GA planned for the first quarter of 2020, the Company estimates that its cash and cash equivalents will be sufficient to fund its operations and capital expenditure requirements as currently planned through the first half of 2021. These estimates are based on the Company’s current business plan, including the continuation of its current research and development programs. This estimate does not reflect any additional expenditures in the event the Company were to in-license or acquire any new product candidates or commences any new sponsored research programs.

R&D Expenses: Research and development expenses were $10.4 million for the quarter ended September 30, 2019, compared to $9.4 million for the same period in 2018. For the nine months ended September 30, 2019, research and development expenses were $28.1 million compared to $25.6 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.

G&A Expenses: General and administrative expenses were $4.7 million for the quarter ended September 30, 2019, compared to $6.0 million for the same period in 2018. For the nine months ended September 30, 2019, general and administrative expenses were $15.4 million compared to $17.9 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 September 30, 2019 of $14.4 million, or ($0.35) per diluted share, compared to a net loss of $14.7 million, or ($0.41) per diluted share, for the same period in 2018. For the nine months ended September 30, 2019, the Company reported a net loss of $41.4 million or ($1.00) per diluted share, compared to a net loss of $41.0 million or ($1.13) 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 November 12, 2019 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 888-208-1711 (USA) or 323-994-2082 (International), passcode 5526863. 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 5526863.