1stOncology™: Cancer Intelligence Service – Page 13119 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Allogene Therapeutics Reports Fourth Quarter and Full Year 2019 Financial Results

On February 27, 2020 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) therapies for cancer, provided a corporate update and reported fourth quarter and full-year 2019 financial results for the periods ended December 31, 2019 (Press release, Allogene, FEB 27, 2020, View Source [SID1234554919]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

Schedule Your 30 min Free Demo!

"2019 was a very successful year for Allogene. We accomplished all of our corporate goals, which includes the establishment of world-class capabilities across multiple new functions and the initiation of our first clinical trials in patients with relapsed or refractory aggressive non-Hodgkin lymphoma and multiple myeloma," said David Chang, M.D., Ph.D., President, Chief Executive Officer and Co-Founder of Allogene. "Our momentum and focus towards making AlloCAR T therapies and their life-saving potential a reality for patients will continue at full speed in 2020. This year will be an important one as we look to demonstrate proof-of-concept data from two key programs and plan for potential pivotal studies."

Recent Highlights

ALLO-501 (anti-CD19 AlloCAR T)
•In 2019, Allogene initiated the ALLO-501 Phase 1 ALPHA trial in patients with relapsed/refractory non-Hodgkin lymphoma (NHL). This study utilizes ALLO-647, Allogene’s anti-CD52 monoclonal antibody (mAb) as a part of the lymphodepletion regimen. Initial data is planned to be presented at a medical meeting in Q2 2020.
•The U.S. Food and Drug Administration cleared the Investigational New Drug Application (IND) for ALLO-501A, the Company’s next generation anti-CD19 AlloCAR T construct devoid of the rituximab off-switch. ALLO-501A, previously referred to as ALLO-501.1, was created to eliminate the rituximab recognition domains in ALLO-501, allowing for use in a broader patient population, including those NHL patients with recent rituximab exposure.
•The Company expects to initiate an abbreviated Phase 1 portion of the ALLO-501A trial (ALPHA2) in the second quarter of the year. The ALPHA2 trial will leverage the findings of the ALPHA trial to finalize the ALLO-501A cell dose and the ALLO-647 based lymphodepletion regimen for a potential pivotal Phase 2 trial.

ALLO-715 (anti-BCMA AlloCAR T)
•The Company has created a robust anti-BCMA strategy centered around ALLO-715 for the treatment of multiple myeloma (MM).
•In 2019, Allogene initiated the ALLO-715 Phase 1 UNIVERSAL trial in patients with relapsed/refractory MM, designed to assess the safety and tolerability of ALLO-715. This trial is also intended to identify the optimal dose of ALLO-647 and includes the potential to evaluate lymphodepletion regimens that do not include fludarabine and cyclophosphamide. UNIVERSAL continues to accrue as planned with initial data anticipated in Q4 2020.
•In January 2020, Allogene entered into a clinical collaboration with SpringWorks Therapeutics to evaluate ALLO-715 in combination with SpringWorks’ investigational gamma secretase inhibitor, nirogacestat, in patients with relapsed/refractory MM. The Company expects to initiate this combination trial in the second half of 2020.
•The Company nominated ALLO-605 as its first TurboCARTM candidate and part of its larger MM strategy. The TurboCAR technology provides the effects of cytokine stimulation to AlloCAR T cells with the goal of enhancing

their potency, expansion and persistence. ALLO-605 is advancing in preclinical development with a potential IND in 2021.

Additional Pipeline Updates
•The Company nominated ALLO-316, an anti-CD70 AlloCAR T, as its next clinical candidate for potential development in acute myeloid leukemia, T-cell malignancies and/or renal cell carcinoma. Submission of the IND for ALLO-316 is expected by year end 2020.

Fourth Quarter Financial Results
•As of December 31, 2019, Allogene had $588.9 million in cash, cash equivalents, and investments.
•Research and development expenses were $49.4 million for the fourth quarter of 2019, which includes $6.4 million of non-cash stock-based compensation expense and $10.0 million in expenses associated with the signing of our collaboration with Notch Therapeutics. For the full year of 2019, research and development expenses were $144.5 million. Research and development expense for the year includes $19.4 million of non-cash stock-based compensation expense.
•General and administrative expenses were $15.2 million for the fourth quarter of 2019, which includes $7.5 million of non-cash stock-based compensation expense. For the full year of 2019, general and administrative expenses were $57.5 million, which includes $26.6 million of non-cash stock-based compensation expense.
•Net loss for the fourth quarter of 2019 was $61.0 million, or $0.58 per share, including non-cash stock-based compensation expense of $13.9 million. For the full year of 2019, our net loss was $184.6 million, or $1.83 per share, including non-cash stock-based compensation expense of $46.1 million.

2020 Financial Guidance
•Allogene expects full year GAAP net losses to be between $260 million and $280 million including estimated non-cash stock-based compensation expense of $70 million to $75 million and excluding any impact from potential business development activities.

Conference Call and Webcast Details
Allogene will host a live conference call and webcast today at 5:30 a.m. Pacific Time / 8:30 a.m. Eastern Time to discuss financial results and provide a business update. To access the live conference all by telephone, please dial 1 (866) 940-5062 (U.S.) or 1 (409) 216-0618 (International). The conference ID number for the live call is 2981059. The webcast will be made available on the Company’s website at www.allogene.com under the Investors tab in the News and Events section. Following the live audio webcast, a replay will be available on the Company’s website for approximately 30 days.

IVERIC bio Reports Fourth Quarter and Year End 2019 Operational Highlights and Financial Results

On February 27, 2020 IVERIC bio, Inc. (Nasdaq: ISEE) reported financial and operating results for the fourth quarter and full year ended December 31, 2019 and provided a general business update (Press release, Ophthotech, FEB 27, 2020, View Source [SID1234554918]).

"We made tremendous progress last year as we build a diversified portfolio in retinal diseases that includes both therapeutics and gene therapy, setting the stage for IVERIC bio to be a leader in developing transformative therapies to treat retinal diseases," stated Glenn P. Sblendorio, Chief Executive Officer and President of IVERIC bio. "We achieved a major milestone with our positive Zimura pivotal clinical trial results in geographic atrophy secondary to dry AMD. Our goal is to continue to build on this momentum. Following the positive data, our team quickly started working on our second Zimura pivotal clinical trial in GA with plans to enroll the first patient next month. Our lead gene therapy programs in rhodopsin mediated adRP and BEST1 related retinal diseases continue to advance towards Phase 1/2 clinical trials and we expect to identify our lead minigene construct for LCA10 later in the year."

Therapeutics Programs

Zimura (avacincaptad pegol): Complement C5 Inhibitor

On October 28, 2019, the Company announced that Zimura (avacincaptad pegol) met its pre-specified primary efficacy endpoint and reached statistical significance in an international, multicenter, randomized, double masked, sham controlled clinical trial in geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD), referred to as the OPH2003 trial. Zimura was generally well tolerated after 12 months of administration. The Company believes that the safety and efficacy results from this trial could meet regulatory agencies’ requirements to serve as one of the two pivotal clinical trials typically required for marketing approval.

January 2020, the Company announced the design of its second pivotal clinical trial of Zimura in GA secondary to dry AMD, ISEE2008. The Company plans to enroll approximately 400 patients in this international, multicenter, double masked, sham controlled clinical trial. Patients will be randomized to receive either monthly administration of Zimura 2mg or sham during the first 12 months of the trial, at which time the primary efficacy analysis of the mean rate of change of GA growth at 12 months will be performed. If the 12 month results are positive, the Company plans to file an application with the U.S. Food and Drug Administration and the European Medicines Agency for marketing approval of Zimura for GA following receipt of that data. At month 12, the Company plans to re-randomize patients in the Zimura 2 mg arm to receive either monthly or every other month administration of Zimura 2 mg. All the patients who

were initially randomized to the sham control arm will continue with monthly administration of sham. The final evaluation will take place at month 24.

The Company plans and is on track to enroll the first patient in the ISEE2008 trial next month.

The Company’s ongoing Phase 2b clinical trial of Zimura for the treatment of autosomal recessive Stargardt disease, an orphan inherited retinal disease, is on track for top-line data to be available during the second half of 2020.

HtrA1 Inhibitor

The Company has identified a lead compound in this preclinical program to address GA secondary to dry AMD and is developing the formulation and manufacturing process with the goal of filing an IND during 2021.

Gene Therapy Programs in Orphan Inherited Retinal Diseases

IC-100: Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)
Natural history studies and IND-enabling activities for IC-100 are ongoing. The Company plans to initiate a Phase 1/2 clinical trial for IC-100 in patients with rhodopsin mediated adRP during the fourth quarter of 2020.

IC-200: BEST1-Related IRDs
Natural history studies and IND-enabling activities for IC-200 are ongoing. The Company plans 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)
IVERIC bio, in collaboration with the University of Massachusetts Medical School (UMass Medical School), is continuing to optimize the minigene constructs with the goal of identifying a lead construct by mid-year 2020.

miniABCA4 Program for Stargardt Disease (STGD1)
IVERIC bio, 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 has received preliminary results and expects to receive additional results for the miniABCA4 program during the second half of 2020.

miniUSH2A: USH2A-Related IRDs Including Usher Syndrome Type 2A (Usher 2A) and USH2A-Associated Nonsyndromatic Autosomal Recessive Retinitis Pigmentosa
This research program targets IRDs associated with mutations in the USH2A gene, including Usher 2A and USH2A-associated nonsyndromatic autosomal recessive retinitis pigmentosa. The Company expects to receive preliminary results during the second half of 2020.
Corporate Update
In December 2019, the Company completed an underwritten public offering in which it sold 7,750,000 shares of its common stock at a price of $4.00 per share, and it also sold to certain investors pre-funded warrants to purchase 3,750,000 shares of its common stock at a price of $3.999 per share underlying each warrant. The Company raised approximately $42.6 million in net proceeds from this offering. During the fourth quarter of 2019, IVERIC bio appointed Guangping Gao, PhD as Chief Strategist, Gene Therapy, and Abraham Scaria, PhD as Chief Scientific Officer.

Fourth Quarter and Year End 2019 Financial Results and 2020 Cash Guidance

As of December 31, 2019, the Company had $125.7 million in cash and cash equivalents. The Company estimates that its year-end 2020 cash and cash equivalents will range between $60 million and $70 million. The Company also estimates that its cash and cash equivalents will be sufficient to fund its operations and capital expenditure requirements as currently planned into the beginning of 2022. These estimates are based on the Company’s current business plan, including initiation of the Zimura ISEE2008 trial and the continuation of the Company’s other on-going research and development programs. These estimates do 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.

2019 Financial Highlights

R&D Expenses: Research and development expenses were $11.6 million for the quarter ended December 31, 2019, compared to $16.1 million for the same period in 2018. For the year ended December 31, 2019, research and development expenses were $39.6 million compared to $41.7 million for the same period in 2018. Research and development expenses decreased primarily due to decreased costs associated with the Company’s Zimura OPH2003 clinical trial in GA and OPH2005 clinical trial in Stargardt disease, as earlier-enrolled patients completed those trials, and decreased costs related to the Company’s acquisition of Inception 4 and its HtrA1 inhibitor program during 2018. These decreases were offset by increases in costs associated with the Company’s gene therapy programs.

G&A Expenses: General and administrative expenses were $6.3 million for the quarter ended December 31, 2019, compared to $5.7 million for the same period in 2018. For the year ended December 31, 2019, general and administrative expenses were $21.6 million compared to $23.6 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 (loss): The Company reported a net loss for the quarter ended December 31, 2019 of $17.5 million, or ($0.39) per diluted share, compared to a net income of $104.1 million, or $2.62 per diluted share, for the same period in 2018. In the quarter and year ended December 31, 2018, the Company recognized a gain on extinguishment of a royalty purchase liability of $125 million due to its December 2018 termination of a royalty and sales agreement with Novo Holdings A/S. For the year ended December 31, 2019, the Company reported a net loss of $58.9 million or ($1.39) per diluted share, compared to a net income of $63.1 million or $1.70 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 February 27, 2020 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 800-458-4121 (USA) or 323-794-2598 (International), passcode 6010573. 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 6010573.

Bio-Techne To Present At The Barclays Global Healthcare Conference

On February 27, 2020 Bio-Techne Corporation (NASDAQ:TECH) reported that Chuck Kummeth, President and Chief Executive Officer, will present at the Barclays Global Healthcare Conference on Wednesday, March 11, 2020 at 9:30 a.m. EST (Press release, Bio-Techne, FEB 27, 2020, View Sourcenews/detail/178/bio-techne-to-present-at-the-barclays-global-healthcare-conference" target="_blank" title="View Sourcenews/detail/178/bio-techne-to-present-at-the-barclays-global-healthcare-conference" rel="nofollow">View Source [SID1234554917]). The conference will be held at the Loews Miami Beach Hotel in Miami Beach, FL. A live webcast of the presentation can be accessed via Bio-Techne’s Investor Relations website at View Source or through the following link https://cc.talkpoint.com/barc002/031020a_js/?entity=60_4GXRJYR.

Merck to Present at the Cowen Health Care Conference

On February 27, 2020 Merck (NYSE: MRK), known as MSD outside the United States and Canada, reported Robert M. Davis, chief financial officer and executive vice president, Merck Global Services, is scheduled to present during a fireside chat at the Cowen and Company 40th Annual Health Care Conference in Boston on March 3 at 12:00 p.m. EST (Press release, Merck & Co, FEB 27, 2020, View Source [SID1234554916]).

Investors, analysts, members of the media and the general public are invited to listen to a live audio webcast of the presentation at View Source

Tocagen Reports Fourth Quarter and Full Year 2019 Financial Results

On February 27, 2020 Tocagen Inc. (Nasdaq: TOCA), a clinical-stage, cancer-selective gene therapy company, reported financial results and business highlights for the fourth quarter and full year ended December 31, 2019 (Press release, Tocagen, FEB 27, 2020, View Source [SID1234554914]).

"Following our extensive review of strategic alternatives, we are excited about the recently announced merger between Tocagen and Forte Biosciences. The proposed merger will create a dermatology company with established clinical proof of concept for their lead asset and an anticipated cash runway to reach a data readout in mid-2021 for its planned randomized Phase 2 trial in patients with atopic dermatitis," said Marty J. Duvall, Chief Executive Officer of Tocagen. "We believe that the proposed merger with Forte has the potential to deliver immediate and long-term value and the Forte leadership team has our full support."

Fourth Quarter 2019 Financial Results

Research and Development (R&D) Expenses: R&D expenses were $7.6 million for the quarter ended December 31, 2019, compared to $15.6 million for the quarter ended December 31, 2018. The reduction in R&D expenses was primarily driven by a reduction in manufacturing and clinical development costs due to the completion of our Toca 5 trial in September 2019.

General and Administrative (G&A) Expenses: G&A expenses were $3.1 million for the quarter ended December 31, 2019, compared to $3.5 million for the quarter ended December 31, 2018. The decrease in G&A expenses was primarily due to lower personnel related costs.

Net Loss: Net loss was $10.6 million, or $0.44 per common share (basic and diluted), for the quarter ended December 31, 2019, compared to a net loss of $19.6 million, or $0.96 per common share (basic and diluted), for the quarter ended December 31, 2018. The 2019 calculation is based on 23.9 million average common shares outstanding for the fourth quarter of 2019, compared to 20.5 million average common shares outstanding for the fourth quarter of 2018.

2019 Twelve-Month Results

License Revenue: License revenue was less than $0.1 million for the 12 months ended December 31, 2019, compared to $18.0 million for the 12 months ended December 31, 2018. The 2018 revenue was associated with a $16.0 million upfront payment and a $2.0 million development milestone earned upon

completion of enrollment in the Toca 5 clinical study, both recognized under Tocagen’s license agreement with ApolloBio.

R&D Expenses: R&D expenses were $45.3 million for the 12 months ended December 31, 2019, compared to $51.1 million for the 12 months ended December 31, 2018. The decrease in R&D expenses primarily reflects decreased costs in clinical development and manufacturing related to the wind down and suspension of the Company’s research and development activities.

G&A Expenses: G&A expenses were $16.2 million for the 12 months ended December 31, 2019, compared to $12.8 million for the 12 months ended December 31, 2018, with the increase primarily driven by commercial readiness activities incurred in the first half of 2019 in anticipation of a potential commercial launch following the completion of our Phase III clinical trial. In September 2019, upon missing our primary endpoint in our Toca 5 clinical trial, all commercial readiness activities were discontinued.

Net Loss: Net loss was $63.5 million, or $2.69 per common share (basic and diluted), for the 12 months ended December 31, 2019, compared to a net loss of $49.0 million, or $2.44 per common share (basic and diluted), for the 12 months ended December 31, 2018. The 2019 calculation is based on 23.6 million average common shares outstanding for the 12 months ended December 31, 2019, compared to 20.1 million average common shares outstanding for the prior year.

Cash Position

Cash, cash equivalents and marketable securities were $21.8 million at December 31, 2019 compared to $96.1 million at December 31, 2018.