On February 27, 2019 Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC), currently developing pelareorep, an intravenously delivered immuno-oncolytic virus, reported the publication of an abstract demonstrating a biomarker for predicting clinical response in patients treated with pelareorep (Press release, Oncolytics Biotech, FEB 27, 2019, View Source [SID1234533792]). This analysis was conducted in patient samples from REO 024; a study of pelareorep and Keytruda in combination with chemotherapy in patients with second-line pancreatic cancer. Detailed results will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting taking place March 30 through April 3 in Atlanta, Georgia.

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”With a simple blood draw, this biomarker data allows physicians to understand which patients are likely to respond to treatment, allowing for the design of clinical studies that are cheaper, faster and more likely to succeed,” said Dr. Matt Coffey, President and CEO of Oncolytics Biotech. ”Our biomarker data that we will present at AACR (Free AACR Whitepaper) is immediately applicable to future clinical studies. Investigators should now be able to predict which patients will respond to pelareorep in combination with a checkpoint inhibitor. This biomarker will be evaluated in our clinical studies with checkpoint inhibitors, including both of our multiple myeloma studies, the AWARE-1 breast cancer study, and importantly, in the same setting this data was produced, our phase two second-line pancreatic study in combination with Keytruda.”

"Using patient blood samples from our REO 024 study in second line pancreatic cancer, T cell receptor sequencing was performed with Adaptive Biotechnologies’ immunoSEQ Assay to measure the diversity or clonality of the T cell population,” said Dr. Rita Laeufle, CMO of Oncolytics Biotech. ”Results from this analysis demonstrate that higher clonality after one three-week cycle of treatment can identify patients likely to respond to combination treatment of pelareorep and a checkpoint inhibitor.”

The results suggest that those patients with a statistically significant change in their T cell population demonstrate a clinical benefit from pelareorep treatment. High T cell clonality correlates with progression free survival at baseline (HR=0.05, p=0.01). Moreover, high clonality correlates with overall survival at both baseline (HR=0.124, p=0.01) and after one cycle of treatment (HR=0.08, p=0.01). This research highlights the potential utility of measuring T cell clonality as a predictive and prognostic biomarker of pelareorep therapy.

The abstract, authored by Dr. Grey Wilkinson, a translational scientist at Oncolytics Biotech, and his colleagues, in collaboration with Northwestern University, UT Health San Antonio and Adaptive Biotechnologies, can be found online at View Source!/6812/presentation/4866. Full details from the poster presentation will be announced after it is presented.

Poster Board Number: 1
Presentation Number: 2272
Title: Exploratory analysis of T cell repertoire dynamics upon systemic treatment with the oncolytic virus pelareorep in combination with pembrolizumab and chemotherapy in patients with advanced pancreatic adenocarcinoma
Date: Monday, April 1
Lecture Time: 1:00 p.m. ET – 5:00 p.m. ET
Location: Georgia World Congress Center, Exhibit Hall B, Poster Section 20
Speakers: Grey Wilkinson
Session Category: Clinical Research
Session: Current Developments in Non-invasive Biomarkers for Assessment of Cancer 3

About Pelareorep

Pelareorep is a non-pathogenic, proprietary isolate of the unmodified reovirus: a first-in-class intravenously delivered immuno-oncolytic virus for the treatment of solid tumors and hematological malignancies. The compound induces selective tumor lysis and promotes an inflamed tumor phenotype through innate and adaptive immune responses to treat a variety of cancers and has been demonstrated to be able to escape neutralizing antibodies found in patients.

On February 26, 2019 ImaginAb, Inc., a clinical stage immuno-oncology imaging company, reported the appointment of Ian Wilson as Chief Executive Officer while Martyn Coombs steps down for family reasons, effective immediately (Press release, ImaginAb, FEB 27, 2019, View Source [SID1234534236]). In addition, Tim Irish has joined ImaginAb’s Board of Directors as an independent non-executive Director and Jeffrey Evelhoch, Ph.D. has joined the Company’s Scientific Advisory Board.

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Ian Wilson, CEO of ImaginAb said, "I am honored to take this leadership role at ImaginAb, and continue to build on the many successes we have achieved, most notably in the last two years. Our ImaginAb team extends a warm welcome to Tim and Jeff as they embark on their new Board roles. We look forward to leveraging their extensive experience within global pharmaceutical companies as we continue to expand our strategic relationships and our clinical programs."

With over 25 years of experience in the commercial development of in vivo medical diagnostics and imaging medical devices, Ian originally joined ImaginAb in early 2018 as Chief Operations Officer, driving clinical execution and R&D. Prior to that, he was CEO and Chief Technology Officer of Edinburgh Molecular Imaging Ltd., leading the development of novel optical imaging agents. Ian has also held positions at Xstrahl Ltd., as CTO and COO, and numerous roles at GE Healthcare including Portfolio and Strategy Manager and Head of Biology.

"I am delighted to be joining the ImaginAb Board of Directors, as the team forges its exciting path towards developing much-needed tools that can measure CD8 T Cells in patients. This vital technology will aid the whole immuno-oncology area in patient selection and management and drive us toward improved outcomes," said Mr. Tim Irish.

Tim Irish has worked in the life sciences industry for thirty years with a career spanning global health technology companies across Europe and North America, including GSK, GE and Philips. Between 2007 and 2015 Tim served on ten Boards, five as Chair, where he successfully executed two trade sales and raised significant equity financing, including an IPO. Since 2015 his governance portfolio covers Life Sciences and Healthcare, both public and private, including board roles as Senior Independent Director and Non-Executive Director at NICE, four European NED roles, and Professor of Practice at King’s College London’s School of Management and Business.

"ImaginAb’s application of precision imaging has the potential to increase our understanding of cancer immunology and may fundamentally change how cancer is treated," said Jeff Evelhoch, Ph.D. "I am excited to work with the other esteemed members of the ImaginAb Scientific Advisory Board to help advance this novel technology."

Jeff Evelhoch, Ph.D., is responsible for development and qualification of novel biomarkers, use of biomarkers to inform pipeline decisions and the development and deployment of companion diagnostics at Merck. Jeff joined Merck Research Laboratories in 2008 as Vice President and Head of Imaging and was named Vice President and Head of Translational Biomarkers in 2015. He joined Merck after four years at Amgen as Executive Director and Head of Imaging Sciences, which followed 2 years at Pfizer Global Research & Development and Pharmacia as Director of Structural Imaging.

On February 27, 2019 Idera Pharmaceuticals, Inc. (NASDAQ: IDRA) reported completion of enrollment into the ongoing phase 2 expansion of the ILLUMINATE-204 trial investigating tilsotolimod, Idera’s intratumorally-delivered toll-like receptor 9 (TLR9) agonist, in combination with ipilimumab (Yervoy*) in patients with unresectable or metastatic melanoma following failure of PD-1 inhibitor treatment (Press release, Idera Pharmaceuticals, FEB 27, 2019, View Source [SID1234533745]).

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52 patients have been dosed in the trial which included two separate patient groups. The company completed the targeted enrollment of 40 patients in the primary enrollment population, constituting patients who are naïve to prior ipilimumab treatment in the metastatic setting. This patient population mirrors the enrollment for the ILLUMINATE-301 phase 3 pivotal trial. The secondary exploratory enrollment population targeted up to 20 patients who had prior ipilimumab treatment in the metastatic setting.

The ILLUMINATE-204 trial is a multi-center trial being conducted at 10 sites throughout the United States.

The company is currently enrolling ILLUMINATE-301, a global, multi-center, randomized Phase 3 trial that compares effectiveness and safety between two treatment groups: tilsotolimod combined with ipilimumab versus ipilimumab given alone. This trial is being conducted at up to 100 centers with a target enrollment of 308 patients. Enrollment remains on track and is expected to be completed in the 4th quarter of 2019.

The company also recently received notice from the U.S. Food and Drug Administration that the company can proceed to implement the ILLUMINATE-206 clinical trial under a new Investigational New Drug (IND) Application. The ILLUMINATE-206 trial will test the safety and effectiveness of tilsotolimod in combination with ipilimumab and nivolumab in treating patients with Squamous Cell Carcinoma of the Head and Neck (SCCHN) and Microsatellite Stable Colorectal Cancer (MSS-CRC). The ILLUMINATE-206 trial is expected to initiate in the second quarter of 2019.

"We made tremendous progress with the clinical development of tilsotolimod in the second half of 2018, and enrollment in the phase 3 study in patients with melanoma progressing on PD-1 therapy is tracking to complete by the end of 2019." stated Joanna Horobin, M.B. Ch.B, Idera’s Chief Medical Officer. "Oncologists are enthusiastic to extend the use of tilsotolimod to potentially improve the outcome of immuno-therapy for other patient populations, such as head and neck cancer, and in microsatellite stable CRC where so far immunotherapy outcomes have not been optimal."

As a reminder, the company provided an interim data update from the ILLUMINATE-204 trial in December 2018.

Summary of ILLUMINATE-204 Key Findings from the December 2018 Update:

37 patients dosed with 8 mg of tilsotolimod in combination with ipilimumab were evaluated for the December 2018 update;
All 37 patients were evaluable for safety;
The combination regimen continues to be generally well tolerated. 9/37 subjects (24.3%) had immune-related toxicities indicating that tilsotolimod + ipilimumab does not appear to add immune-related toxicity versus ipilimumab alone;
Injection-related toxicities were grade 1-2 transient fever and flu-like symptoms lasting <48 hours;
34 patients were evaluable for efficacy;
Responses, including 3 Complete Responses (CR), were observed in 11 of the 34 evaluable patients (32.4%);
Duration of response ranges from > 1 month to > 30 months, with 36% of responses ongoing at the time of the report;
Per RECIST v1.1, the Overall Response Rate (ORR) is 29.4%; one patient with an unconfirmed Partial Response (uPR) at the end of treatment assessment progressed due to a new lesion at the 3-month follow-up disease assessment;
Overall, 26 patients out of 34 evaluable for efficacy (76.5%) experienced disease control (CR, PR, or Stable Disease [SD]);
Analysis of spider plots show tumor shrinkage in both injected and uninjected lesions, indicating an abscopal effect;
Responding subjects include one patient with mucosal melanoma and one patient with acral melanoma, two forms of melanoma that are particularly difficult to treat; and
Importantly, 2 of 5 patients with prior ipilimumab experience achieved responses, further demonstrating a signal that tilsotolimod has the potential to help overcome prior ipilimumab resistance.
Additionally:

A RECIST v1.1 PR of > 2.5 years is ongoing in 1 patient treated with tilsotolimod 4 mg in combination with ipilimumab; and
A RECIST v1.1 CR of > 1 year is ongoing in 1 patient treated with tilsotolimod 16 mg in combination with pembrolizumab.
About Tilsotolimod (IMO-2125)
Tilsotolimod is a TLR 9 agonist that received Fast Track Designation from the US Food and Drug Administration (FDA) in 2017 for the treatment of anti-PD-1 refractory melanoma, in combination with ipilimumab as well as orphan drug designation from the FDA for the treatment of melanoma Stages IIb to IV. It signals the immune system to create and activate cancer-fighting cells (T-cells) to target solid tumors. Currently approved immuno-oncology treatments, specifically check-point inhibitors, work for some but not all, as many patients’ immune response is missing or weak and thus they do not benefit from the checkpoint therapy. Intratumoral injections with tilsotolimod are designed to selectively enable the tumor-specific T-cells to recognize and attack cancers that remained elusive and unrecognized by the immune system exposed to checkpoint inhibitors alone, while limiting toxicity or impact on healthy cells in the body.

About ILLUMINATE-301
The ILLUMINATE-301 study (2125-MEL-301) is for patients who have metastatic melanoma for whom treatment with an anti-PD-1 drug like Keytruda (pembrolizumab) or Opdivo (nivolumab) has failed. ILLUMINATE-301 is a global, multi-center, randomized Phase 3 study that compares the effectiveness and safety between two treatment groups: IMO-2125 combined with ipilimumab (Yervoy) versus ipilimumab given alone.

For additional details about ILLUMINATE-301, please go to clinicaltrials.gov and search for study identifier NCT03445533.

About ILLUMINATE-204
The ILLUMINATE-204 study (2125-204) is for patients who have metastatic melanoma for whom treatment with an anti-PD-1 drug like Keytruda** (pembrolizumab) or Opdivo* (nivolumab) has failed. ILLUMINATE-204 is a multi-center, two-arm Phase 1/2 study that tests the safety and effectiveness of tilsotolimod in combination with either ipilimumab (Yervoy) or pembrolizumab (Keytruda) for the treatment of patients with anti-PD-1 refractory metastatic melanoma.

For additional details about ILLUMINATE-204, please go to clinicaltrials.gov and search for study identifier NCT02644967.

About ILLUMINATE-206
The ILLUMINATE-206 study (2125-206) is a Phase 2 multi-center, multi-cohort study of intratumoral tilsotolimod in combination with nivolumab and ipilimumab in patients with Squamous Cell Carcinoma of the Head and Neck (SCCHN) who are either immunotherapy naïve and immunotherapy refractory and immunotherapy naïve patients with Microsatellite Stable Colorectal Cancer (MSS-CRC). The trial is expected to open for enrollment in the second quarter of 2019.

About Metastatic Melanoma
Although melanoma is a rare form of skin cancer, it comprises over 75% of skin cancer deaths. The American Cancer Society estimates that there were approximately 76,000 new invasive melanoma cases and 10,000 deaths from the disease in the USA in 2016. Additionally, according to the World Health Organization, about 132,000 new cases of melanoma are diagnosed around the world every year.

On February 27, 2019 CytomX Therapeutics, Inc. (Nasdaq: CTMX), a clinical-stage oncology-focused biopharmaceutical company pioneering a novel class of investigational antibody therapeutics based on its Probody therapeutic technology platform, reported full-year 2018 financial results (Press release, CytomX Therapeutics, FEB 27, 2019, View Source/news-releases/news-release-details/cytomx-therapeutics-announces-full-year-2018-financial-results" target="_blank" title="View Source/news-releases/news-release-details/cytomx-therapeutics-announces-full-year-2018-financial-results" rel="nofollow">View Source [SID1234533793]). As part of its 2019 Research and Development Day held yesterday in New York, CytomX provided an operational update on the company.

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As of December 31, 2018, CytomX had cash, cash equivalents and short-term investments of $436.1 million, sufficient capital to fund its operating expenses and capital requirement into 2021.

"Over the last year, we have generated meaningful clinical proof of concept data for the Probody platform across both of our lead, wholly-owned programs," said Sean McCarthy, D.Phil., president, chief executive officer and chairman of CytomX Therapeutics. "As we showed at our inaugural Research and Development Day yesterday, our PD-L1 Probody therapeutic, CX-072, is active across a wide range of tumors and has a potentially differentiated safety profile as monotherapy and in combination. CX-2009, our first-in-class CD166 Probody Drug Conjugate, is well tolerated and has demonstrated anti-tumor activity across multiple tumor types. In 2019, we will continue to explore the full potential of these innovative product candidates as we maintain our intense focus on discovery, development and ultimate commercialization of a new generation of differentiated cancer therapeutics."

2018 Business Highlights and Recent Developments

PROCLAIM-CX-072 (PD-L1 Probody Therapeutic) Clinical Program

CX-072 is a Probody therapeutic targeting PD-L1, a clinically- and commercially-validated anti-cancer target.
Enrollment began in January 2017 in PROCLAIM-CX-072, a Phase 1/2 clinical trial evaluating CX-072 as monotherapy and in combination with YERVOY (ipilimumab) or Zelboraf (vemurafenib) in patients with cancer.
Enrollment is complete with follow-up continuing in the monotherapy dose escalation arm evaluating CX-072 in patients with advanced unresectable solid tumors or lymphomas (Part A) and in the monotherapy dose escalation arm in patients with PD-L1-positive tumors (Part A2).
Enrollment and follow-up are ongoing in the monotherapy expansion cohorts of CX-072 at 10 mg/kg in multiple indications (Part D).
Data from Parts A, A2 and D was presented most recently at CytomX’s 2019 Research and Development Day.
Additional data from Part D is expected in 2019.
Enrollment of the dose escalation arm combining CX-072 plus Yervoy (ipilimumab) in patients with advanced unresectable solid tumors or lymphomas (Part B) is complete and was presented most recently at CytomX’s 2019 Research and Development Day.
Enrollment is ongoing in the dose escalation combination arm of CX-072 plus Zelboraf (vemurafenib) in patients with V600E BRAF-positive melanoma (Part C).
PROCLAIM-CX-2009 (CD166 Probody Drug Conjugate) Clinical Program

CX-2009 is a Probody drug conjugate (PDC) that targets CD166, an antigen that is broadly and highly expressed in many types of cancer, and is conjugated with DM4, a clinically-validated toxin licensed from ImmunoGen.
Enrollment began in June 2017 in PROCLAIM-CX-2009, a Phase 1/2 clinical trial, evaluating CX-2009 as monotherapy in a subset of seven cancer types (Part A) and in patients selected for high level of CD166 expression (Part A2).
Preliminary data from Parts A and A2 was presented at CytomX’s 2019 Research and Development Day.
BMS-986249 (CTLA-4 Probody Therapeutic) Clinical Program

Bristol-Myers Squibb (BMS), continues enrollment in a Phase 1/2 clinical trial evaluating BMS-986249 alone and in combination with OPDIVO (nivolumab) in solid tumors that are advanced and have spread.
BMS has stated that they anticipate preliminary data from this trial in 2019.
CX-2029 (CD71 Probody Drug Conjugate) Clinical Program

CytomX, in collaboration with AbbVie, is advancing CX-2029, a CD71-directed Probody Drug Conjugate.
CD71, also known as the transferrin receptor 1 (TfR1), is highly expressed in a number of solid and hematologic cancers and has particularly attractive molecular properties for efficient delivery of cytotoxic payloads to tumor cells.
Enrollment began in late June 2018 in PROCLAIM-CX-2029, a Phase 1/2 clinical trial evaluating CX-2029 as monotherapy in patients with solid tumors or lymphomas and is ongoing.
CX-188 (PD-1 Probody Therapeutic) Program

CX-188 is a PD-1-directed Probody therapeutic.
PD-1 is the receptor for the PD-L1 ligand responsible for inhibiting T-cell activation in a variety of cancers and is a clinically- and commercially-validated anti-cancer target.
In October 2018, CytomX filed an Investigational New Drug Application (IND) with the Food and Drug Administration (FDA) on CX-188. The CX-188 IND was cleared by the FDA in November 2018. Due to a recent program and portfolio prioritization, the company has decided to indefinitely postpone the CX-188 clinical trials. The company may elect to initiate clinical trials of CX-188 in the future.
CytomX Technology Acquisition from Agensys, Inc.

In January 2019, CytomX announced the acquisition of drug conjugate linker-toxin and CD3-based bispecific technologies from Agensys, Inc., an affiliate of Astellas Pharma Inc.
CytomX Therapeutics 2019 Research and Development Day

CytomX hosted a Research and Development Day on Tuesday, February 26, 2019. A replay of the webcasted event is available under the "Investors & News" section of www.CytomX.com.

Full Year Financial Results

Cash, cash equivalents and short-term investments totaled $436.1 million as of December 31, 2018, compared to $374.1 million as of December 31, 2017.

Revenue was $59.5 million for the year ended December 31, 2018, compared to $71.6 million for the year ended December 31, 2017. The $12.1 million decrease is largely attributable to the recognition of $14.0 million in milestone revenue in 2017 from AbbVie (net of a $1.0 million license fee paid to SGEN) as a result of completion of certain milestones under the CD71 Agreement, the recognition of a $10 million milestone in 2017 from BMS related to the IND filing for BMS-986249, the recognition of $6.5 million in revenue in 2017 related to the delivery of the ImmunoGen 2017 License to ImmunoGen in connection with the ImmunoGen Research Agreement, a decrease of $4.5 million in the amortization of deferred revenue under the ImmunoGen Research Agreement which concluded on June 30, 2018, and a decrease of $0.5 million in revenues from Pfizer as a result of Pfizer terminating our Research Collaboration, Option and License Agreement in March 2018. These decreases were partially offset by the recognition of $11.7 million of revenue in 2018 related to the milestone payment of $21.0 million from AbbVie (net of the associated $4.0 million sublicense fee to SGEN) for the achievement of the IND filing success criteria under the CD71 Agreement, an increase of $7.9 million in amortization of deferred revenue in 2018 related to the $200.0 million upfront payment received in the second quarter of 2017 as a result of the extension of the BMS collaboration, and an increase of $3.6 million of revenue from Amgen under the Amgen Agreement entered into in September 2017.

Research and development expenses increased by $11.6 million during the year ended December 31, 2018 compared to the corresponding period in 2017. The increase was primarily attributed to an increase in lab services of $10.0 million and $12.3 million in clinical trial expenses related to CX-072, CX-2009 and CX-2029 Phase 1/2 clinical development. There was also an increase of $10.5 million in personnel related expenses; $1.2 million increase in allocation of information technology and facilities related expenses; $0.9 million in lab supplies and $0.7 in consulting expenses. These amounts were largely offset by the recognition, during the third quarter of 2017, of $10.7 million of non-cash in-process research and development expense; $12.1 million of sublicense fees payable to the University of California, Santa Barbara, as a result of both the $200 million upfront payment made by BMS in connection with the expanded collaboration and the Amgen Agreement and a $1.0 million sublicense fee to ImmunoGen upon commencement of enrollment of Phase 1/2 and first patient dosing in the clinical trial for CX-2009 in the second quarter of 2017.

General and administrative expenses increased by $7.9 million during the year ended December 31, 2018 compared to the corresponding period in 2017. This increase was largely attributed to an increase of $5.6 million in personnel related expenses due to increases in headcount, and $2.3 million for various consulting and other services.

Teleconference Scheduled Today at 5:00 p.m. ET
Conference Call/Webcast Information

CytomX management will host a conference call today at 5:00 p.m. ET. Interested parties may access the live audio webcast of the teleconference through the "Investor & News" section of CytomX’s website at View Source or by dialing 1-877-809-6037 (U.S. and Canada) or 1-615-247-0221 (International) and using the passcode 3748238. An archive of the webcast will be available on the CytomX website from February 27, 2019, until March 6, 2019.

On February 27, 2019 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, 2018 (Press release, Tocagen, FEB 27, 2019, View Source [SID1234533808]).

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"We had a very strong close to the year, accomplishing all of our 2018 business goals. In particular, NRG Oncology’s selection of Toca 511 & Toca FC for a Phase 2/3 trial in newly diagnosed glioblastoma was a major accomplishment for our team and we look forward to initiating this trial in late 2019. Expansion of our late-stage cancer-selective gene therapy program regimen into the front-line indication could pave the way to having a greater impact on more patients in the years ahead," said Marty Duvall, chief executive officer of Tocagen. "Furthermore, having ended the year with approximately $96 million in cash, we have a favorable financial position as we look towards the results in 2019 from our Phase 3 Toca 5 trial in recurrent high grade glioma and build for success going into 2020."

Fourth Quarter 2018 Highlights

NRG Oncology, a member of the National Cancer Institute’s National Clinical Trial Network, announced in November 2018 plans to conduct and sponsor a clinical trial to evaluate Toca 511 & Toca FC for the treatment of patients with newly diagnosed glioblastoma (GBM). The proposed NRG-BN006 Phase 2/3 trial will be the first clinical trial of the Toca 511 & Toca FC regimen in the newly diagnosed GBM setting, and is expected to commence in late 2019. NRG Oncology conducts practice-defining, multi-institutional trials funded primarily by the NCI.

Under Tocagen’s PRIME (PRIority MEdicines) designation for Toca 511 & Toca FC, the European Medicines Agency indicated that the statistical analyses, seamless design and use of overall survival as the primary endpoint in the ongoing Phase 3 Toca 5 clinical trial are appropriate for a potential marketing authorization applications for Toca 511 & Toca FC in Europe.

Tocagen presented clinical and preclinical data at congresses hosted by the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) and Society for Neuro-Oncology (SNO). We identified immunologic biomarkers in our clinical trials that may help predict patient outcomes following treatment with Toca 511 & Toca FC, and preclinical studies demonstrated the additive benefits of combining Toca 511 & Toca FC with chemotherapy.

In December 2018, Tocagen raised $30.0 million of gross proceeds in an underwritten public offering of its common stock.

2019 Milestones

Toca 5 second interim analysis expected in the second quarter of 2019

Toca 5 final analysis planned for the fourth quarter of 2019

Prepare to initiate a rolling biologics license application (BLA) for Toca 511 & Toca FC in recurrent high grade glioma

Build U.S. commercial team for potential launch of Toca 511 & Toca FC

NRG Oncology to initiate NRG-BN006 clinical trial evaluating Toca 511 & Toca FC in newly diagnosed GBM in late 2019

Report updated Toca 6 safety and immune activity data in advanced solid tumors

Fourth Quarter 2018 Financial Results

Research and Development (R&D) Expenses: R&D expenses were $15.6 million for the quarter ended December 31, 2018, compared to $8.3 million for the quarter ended December 31, 2017. The R&D expenses in both periods were primarily driven by costs to support the Toca 5 trial and to support the manufacturing of drug product. The increase in 2018 reflects primarily higher manufacturing spend as compared to the same period in 2017.

General and Administrative (G&A) Expenses: G&A expenses were $3.5 million for the quarter ended December 31, 2018, compared to $2.4 million for the quarter ended December 31, 2017. The increase in G&A expenses was primarily due to higher costs to support increased operations activity.

Net Loss: Net loss was $19.6 million, or $0.96 per common share (basic and diluted), for the quarter ended December 31, 2018, compared to a net loss of $10.8 million, or $0.55 per common share (basic and diluted), for the quarter ended December 31, 2017. The 2018 calculation is based on 20.5 million average common shares outstanding for the fourth quarter of 2018, compared to 19.8 million average common shares outstanding for the fourth quarter of 2017.

2018 Twelve-Month Results

License Revenue: License revenue was $18.0 million for the 12 months ended December 31, 2018, compared to less than $0.1 million for the 12 months ended December 31, 2017. 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 $51.1 million for the 12 months ended December 31, 2018, compared to $29.1 million for the 12 months ended December 31, 2017. The increase in R&D expenses primarily reflects increased costs in manufacturing and our Toca 5 clinical trial.

G&A Expenses: G&A expenses were $12.8 million for the 12 months ended December 31, 2018, compared to $8.6 million for the 12 months ended December 31, 2017, with the increase primarily driven by higher personnel costs and costs to support increased operations activity.

Net Loss: Net loss was $49.0 million, or $2.44 per common share (basic and diluted), for the 12 months ended December 31, 2018, compared to a net loss of $38.9 million, or $2.66 per common share (basic and diluted), for the 12 months ended December 31, 2017. The 2018 calculation is based on 20.1 million average common shares outstanding for the 12 months ended December 31, 2018, compared to 14.6 million average common shares outstanding for the prior year.

Cash Position and Guidance

Cash, cash equivalents and marketable securities were $96.1 million at December 31, 2018 compared to $88.7 million at December 31, 2017. Total gross cash burn to support operations was $51.3 million for the year ended December 31, 2018. Based on current operating plans, Tocagen estimates the total cash used in 2019 to fund operations and capital expenditures will be approximately $65 million.

About Toca 511 & Toca FC

Tocagen’s lead product candidate is a two-part cancer-selective immunotherapy comprising an investigational biologic, Toca 511 (vocimagene amiretrorepvec), and an investigational small molecule, Toca FC (flucytosine, extended-release). Toca 511 is a retroviral replicating vector (RRV) that selectively infects cancer cells and delivers a gene for the enzyme, cytosine deaminase (CD). Through this targeted delivery, infected cancer cells carry the CD gene and produce CD. Toca FC is an orally administered prodrug, 5-fluorocytosine (5-FC), which is converted into an anti-cancer drug, 5-fluorouracil (5-FU), when it encounters CD. 5-FU kills cancer cells and immune-suppressive myeloid cells resulting in anti-cancer immune activation and subsequent tumor killing.