On March 28, 2019 Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC), currently developing pelareorep, an intravenously delivered immuno-oncolytic virus, reported the publication of an article highlighting the synergistic effect of oncolytic immunotherapy in combination with a proteasome inhibitor in the treatment of multiple myeloma (Press release, Oncolytics Biotech, MAR 28, 2019, View Source [SID1234534709]). The article, entitled, "Oncolytic immunotherapy and bortezomib synergy improves survival of refractory multiple myeloma in a preclinical model," co-authored by Oncolytics’ President and Chief Executive Officer, Dr. Matt Coffey was published in the March 12 edition of Blood Advances.

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The article demonstrates that the combination of reovirus and bortezomib can overcome drug resistance, a major hurdle in the treatment of multiple myeloma. Further, the combination modifies the tumor microenvironment to overcome the immune suppressive environment caused by multiple myeloma cells in the bone marrow. This combination promotes a pro-inflammatory signal within the tumor microenvironment, upregulates the PD-L1/PD-1 axis and enhances immune cell infiltration. Collectively, this suggests the possibility of synergies and increased efficacy with the addition of checkpoint blockade to the combination.

"We’d like to thank our collaborators for this influential work which provides further evidence that the combination of pelareorep with a proteasome inhibitor can synergistically overcome drug resistance and drive enhanced tumor killing and superior overall survival in animal models," said Dr. Rita Laufle, Chief Medical Officer of Oncolytics. "Importantly, the combination engages the immune system in a manner that converts tumor indications that have not been responsive to checkpoint blockade to those that are responsive, by both reversing the immune suppressive aspects of the tumor microenvironment while increasing immune cell trafficking to the tumor and generating new anti-tumor memory T cells. These findings are consistent with our clinical observations that proteasome inhibition, in combination with reovirus, primes the tumor for checkpoint blockade and further validates our combination studies in this and other indications, including our most advanced indication, metastatic breast cancer."

Blood Advances is a semimonthly, peer-reviewed medical journal published by the American Society of Hematology (ASH) (Free ASH Whitepaper). A copy of the paper can be found on our website: View Source

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 March 28, 2019 Marker Therapeutics, Inc. (NASDAQ:MRKR), a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, reported a business and clinical update, as well as an overview of upcoming milestones for 2019 (Press release, TapImmune, MAR 28, 2019, View Source [SID1234534708]).

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"Our MultiTAA T cell therapies have continued to generate positive and compelling clinical data across various indications in several ongoing investigator-sponsored clinical trials led by Baylor College of Medicine (BCM). We plan to advance a Phase 2 Company-sponsored clinical trial in post-transplant acute myeloid leukemia (AML)—a disease area and patient population for which there are limited treatment options. We expect to finalize our clinical trial protocol in AML by the end of the second quarter of 2019 and to submit our IND in the third quarter, with the first patient enrolled by the end of the year," said Peter L. Hoang, President & CEO of Marker Therapeutics.

Continued Mr. Hoang: "While our T cell therapies remain our primary clinical focus, we are also advancing our T cell vaccine candidates, TPIV200 and TPIV100/110, for the treatment of ovarian and breast cancers. Overall, there are two Phase 2 Company-sponsored studies ongoing for TPIV200 in triple-negative breast cancer and ovarian cancer, and we anticipate reporting interim data from the ovarian cancer Phase 2 study in Q4 2019."

PROGRAM HIGHLIGHTS AND CURRENT UPDATES

Multi-Antigen Targeted (MultiTAA) T Cell Therapies

·Acute Myeloid Leukemia Data
oThe Company reported a clinical update from a Phase 1 clinical trial in post-transplant AML in oral and poster presentations at ASH (Free ASH Whitepaper) in December and ASBMT and CIBMTR in February. Results from the BCM-sponsored study showed that the treatment is safe and well-tolerated and has the potential to mediate a meaningful anti-tumor effect, as well as significant in vivo expansion of T cells. Among the highlights from the study, 11 out of 13 patients dosed with MultiTAA T cells as a maintenance therapy after receiving allogeneic stem cell transplant remain alive, ranging from 6 weeks to 2.5 years post-infusion. Nine of these patients have never relapsed after MultiTAA therapy and continue to remain in complete remission (CR). Patients with active disease, overall survival ranged from 4 and 21 months as compared to 4.5 months in historical results after standard of care.

oMarker will pursue post-transplant AML as the lead indication of its T cell therapy program. Based on findings from various dose cohorts in the Phase 1 BCM-sponsored trial, Marker has made a strategic decision to focus on post-transplant AML, and plans to initiate pre-IND discussions with the U.S. FDA in the second quarter of 2019, with an IND submission for the Company-sponsored potentially pivotal Phase 2 study in the third quarter. The multicenter study will evaluate clinical efficacy of MultiTAA specific T cells in patients with AML or myelodysplastic syndromes (MDS) in both the adjuvant and active disease setting, following an allogeneic hematopoietic stem cell transplant (HSCT). The dose administered will be the maximum tolerated dose from the BCM-sponsored Phase 1 trial. In the adjuvant setting, patients will be randomized 2:1 to either MultiTAA therapy at approximately 90 days post-transplant versus standard of care observation, while the active disease patients will receive MultiTAA T cells as part of a single-arm group.

·Lymphoma Data
As reported in January, 2019:

oTo date, no relapses have been observed for any patient entering a complete response (CR);
oPatients with active disease are now between 1 and 5+ years in CR after infusion of MultiTAA cells (ongoing);
oSeveral patients with stable disease show potential durable disease stabilization, with two patients experiencing stable disease for over 9 months and 24 months, respectively;
oResponses in all six patients who entered CR were associated with an expansion of infused T cells, as well as induction of antigen spreading.

·Acute Lymphoblastic Leukemia (ALL) Data
As reported at ASBMT and CIBMTR in February:

oPatients are now up to 28 months in continued complete remission (CCR);
oThe one patient who experienced relapse displayed mixed donor/recipient chimerism after transplant, but remained in CCR for 6 months;
oPatients who remain in CCR have been durable for between 4 to 28 months, with a median of 16 months.

·Multiple Myeloma Data
As the Company reported in January, 2019, ten patients with active disease have been treated, including:

oOne patient with a CR durable for approximately 29 months before relapse, was subsequently given a second treatment infusion of MultiTAA T cells, resulting in stable disease for 3 months (ongoing) after the second treatment;
oTwo patients achieved partial responses (PR) of between 14 and 22 months (ongoing) as of last follow-up;
oAll seven remaining patients experienced stabilization of disease following infusion of MultiTAA cells initially. Three patients developed transient disease stabilization of between 3-7 months with subsequent progression, and four patients have ongoing stable disease.
oEight patients were treated in remission, with a median follow-up of 21 months. Only one patient has relapsed to date;
oCorrelative studies show significant expansion of MultiTAA T cells, as well as significant evidence of epitope spreading with expansion of endogenous T cells specific for tumor-associated antigens that were not targeted by the MultiTAA product.

Overall, across all indications, MultiTAA therapy appears to be safe and well-tolerated, with no incidence of cytokine release syndrome, neurotoxicity or any other serious adverse events related to the therapy.

T Cell Based Vaccines

·Ovarian Cancer Data
·As the Company reported in January, 2019, it has completed enrollment in its Phase 2 study in ovarian cancer using TPIV200 as a maintenance therapy for patients in their first remission after surgery and platinum-based chemotherapy. To date, Marker has enrolled, randomized, and treated 120 patients at 17 clinical sites. The study completed enrollment six months faster than anticipated and the Company expects to reach its planned interim analysis trigger of 55 patients who have progressed before the end of 2019.

·Triple Negative Breast Cancer Data
·The Company also reported initial findings from its interim analysis of its dose-finding study in triple negative breast cancer, using TPIV200 as a maintenance therapy for patients in remission following first-line therapy. The four-arm study included low- and high-dose TPIV200 with or without cyclophosphamide. Of 27 patients evaluated to date for immunogenicity, 26 showed significant immune response to the vaccine treatment. Of 80 patients treated at 11 clinical sites, 11 have shown disease progression to date following treatment with TPIV200.

Marker continues to advance the development of its proprietary PolyStart platform, a nucleic acid-based technology with the potential to increase the potency of our vaccines by conferring a four-fold increase in expression of target-cell-specific, naturally processed antigenic epitopes on a cell’s surface. This approach boosts helper and/or long-lived killer T cells, enabling their potential to effectively seek out and destroy target cells.

CASH POSITION AND GUIDANCE

Marker reported cash and cash equivalents totaling $61.7 million as of December 31, 2018. Based on current operating plans, Marker expects that current cash resources will be sufficient to meet operating requirements into Q4 of 2020.

UPCOMING NEAR-TERM POTENTIAL MILESTONES

·Pre-IND discussions for AML with the U.S. FDA in Q2;
·First update in solid tumor program in Q2 concurrently with a major medical meeting;
·IND submission for Company-sponsored Phase 2 AML study in Q3, with first patient enrolled by end of 2019;
·Interim analysis readout in the TPIV200 ovarian trial in Q4;
·Overall update on ongoing clinical trials in cell therapy at end of 2019.

Business Update Call and Webcast

Marker management will host business update conference call and webcast today at 5:00 p.m. EDT. To access the call, participants should dial 1-855-238-2333 (domestic) or 1-412-317-5215 (international) and refer to the "Marker Therapeutics, Inc. call." The webcast will be accessible in the Investors section of the Company’s website at www.markertherapeutics.com. The archived webcast will be available for replay on the Marker website approximately two hours after the event.

On March 28, 2019 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported publication of abstracts on neratinib for the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019 (Press release, Puma Biotechnology, MAR 28, 2019, View Source [SID1234534707]). The AACR (Free AACR Whitepaper) Annual Meeting will be held at the Georgia World Congress Center in Atlanta, Georgia from March 29 to April 3. Posters will be available on Puma’s website following presentation.

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Full abstracts of the following presentations are available online at www.aacr.org :

March 31, 2019, 3:35 – 3:50 p.m. EDT – Abstract 1724 (Oral): Natural history and clinical characteristics of ERBB2 mutant hormone receptor-positive breast cancers: Results from the AACR (Free AACR Whitepaper) Project GENIE Registry.
Michele LeNoue-Newton et al, Vanderbilt-Ingram Cancer Center, TN.

March 31, 2019, 3:50 – 4:05 p.m. EDT – Abstract 929 (Oral): Paired tumor and cfDNA in patients with HER2-mutant solid tumors treated with neratinib reveals convergence of multiple ontarget resistance mechanisms: Results from the SUMMIT ‘Basket’ Trial.
Helen H. Won et al, Memorial Sloan Kettering Cancer Center, New York.

April 2, 2019, 3:20 – 3:35 p.m. EDT – Abstract 4459 (Oral): Morphologic and genomic characterization of circulating tumor cells in patients with ERBB2 mutant HER2 non-amplified metastatic breast cancer treated with neratinib.
Stephanie Nicole Shishido et al, USC, Los Angeles.

April 2, 2019, 4:35 – 4:50 p.m. EDT – Abstract 4527 (Oral): Patient-derived organoids and xenografts identify neratinib plus HER2 antibody drug conjugate as a synergistic drug combination for HER2 mutated, non-amplified metastatic breast cancer.
Shunqiang Li et al, Washington University School of Medicine, Saint Louis, MO.

March 31, 2019, 1:00 – 5:00 p.m. EDT – Abstract 328 (Poster): ADAM17-induced activation of HER receptors mediate resistance to trastuzumab in a subset of moderate HER2-expressing breast cancer cells.
Katharina Feldinger et al, University of Birmingham, UK.

March 31, 2019, 1:00 – 5:00 p.m. EDT – Abstract 329 (Poster): Hyperactivation of mTORC1 drives acquired resistance to the pan-HER tyrosine kinase inhibitor neratinib in HER2-mutant cancers.
Dhivya R. Sudhan et al, UT Southwestern Medical Center, Dallas, TX.

March 31, 2019, 1:00 – 5:00 p.m. EDT – Abstract 389 (Poster): Role of HER3 signaling pathways in ER+ and HER2+ breast cancers.
Rosalin Mishra et al, University of Cincinnati College of Pharmacy, Cincinnati, OH.

March 31, 2019, 1:00 – 5:00 p.m. EDT – Abstract 395 (Poster): Aberrant HER2 signaling is a therapeutic target in a subset of castration-resistant prostate cancer.
Joshua W. Russo et al, Beth Israel Deaconess Medical Center, Boston, MA.

April 1, 2019, 1:00 – 5:00 p.m. EDT – Abstract 1923 (Poster): KRAS-mutant (mt) colorectal cancer (CRC) organoid models generated from patient-derived xenografts (PDX) show response to combination of trametinib (Tm), neratinib (N), and trastuzumab (Tz).
Rekha Pal et al, NSABP Foundation, Inc., Pittsburgh, PA.

April 2, 2019, 1:00 – 5:00 p.m. EDT – Abstract 3705 (Poster): Identification of frequent HER2 activating mutations in canine primary pulmonary adenocarcinoma.
Gwendolen Lorch et al, Ohio State University College of Veterinary Med., Columbus, OH.

April 3, 2019, 8:00 a.m. – 12:00 p.m. EDT – Abstract 4827 (Poster): The therapeutic superiority of neratinib in combination with trastuzumab compared to pertuzumab plus trastuzumab in HER2-positive in vivo breast cancer models.
Jamunarani Veeraraghavan et al, Baylor College of Medicine, Houston, TX.

April 3, 2019, 8:00 a.m. – 12:00 p.m. EDT – Abstract 4832 (Poster): Preclinical evaluation of neratinib plus T-DM1 in orthotopic PDX models of HER2-positive breast cancer brain metastases.
Jing Ni et al, Dana Farber Cancer Institute, Boston, MA.

On March 28, 2019 AIVITA Biomedical, Inc., a biotech company specializing in innovative stem cell applications, reported that the Company will be presenting new data at the 2019 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in Atlanta, Georgia (Press release, AIVITA Biomedical, MAR 28, 2019, View Source [SID1234534704]). Chief Medical Officer Robert O. Dillman, M.D. will give a presentation concerning prognostic and predictive markers of efficacy for patients receiving AIVITA’s next-generation immunotherapy targeting tumor-initiating stem cells.

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The American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting is a six-day meeting covering the latest discoveries in cancer research. The presentation details are as follows:

Title: Soluble programmed cell death molecule-1 (sPD-1) as a prognostic and predictive biomarker in patients with metastatic melanoma randomized for treatment with autologous dendritic cell or tumor cell vaccines.
Session Category: Clinical Research
Session Title: Circulating and Cell-free Biomarkers for Diagnosis and Monitoring of Cancer 2
Session Date and Time: Monday Apr 1, 2019 8:00 AM – 12:00 PM
Location: Georgia World Congress Center, Exhibit Hall B, Poster Section 18
Poster Board Number: 26
Permanent Abstract Number: 1257

AIVITA is currently conducting three clinical studies investigating its platform ROOT OF CANCER therapy in patients with ovarian cancer, glioblastoma and melanoma. AIVITA uses 100% of proceeds from the sale of its ROOT of SKIN skincare line to support the treatment of women with ovarian cancer.

About ROOT OF CANCER

OVARIAN CANCER

AIVITA’s treatment is a platform technology applicable to most solid tumor types and consists of autologous dendritic cells loaded with autologous tumor antigens from purified autologous self-renewing tumor-initiating cells.

AIVITA’s ovarian Phase 2 double-blind study is active and enrolling approximately 99 patients who are being randomized in a 2:1 ratio to receive either the autologous cancer stem cell-targeting immunotherapy or autologous monocytes as a comparator.

Patients eligible for randomization and treatment will be those (1) who have undergone debulking surgery, (2) for whom a cell line has been established, (3) who have undergone leukapheresis from which sufficient monocytes were obtained, (4) have an ECOG performance grade of 0 or 1 (Karnofsky score of 70-100%), and (5) who have completed primary therapy. The trial is not open to patients with recurrent ovarian cancer.

For additional information about AIVITA’s AVOVA-1 trial patients can visit: www.clinicaltrials.gov/ct2/show/NCT02033616

GLIOBLASTOMA

AIVITA’s glioblastoma Phase 2 single-arm study is active and is enrolling approximately 55 patients to receive the cancer stem cell-targeting immunotherapy.

Patients eligible for treatment will be those (1) who have recovered from surgery such that they are about to begin concurrent chemotherapy and radiation therapy (CT/RT), (2) for whom an autologous tumor cell line has been established, (3) have a Karnofsky Performance Status of > 70 and (4) have undergone successful leukapheresis from which peripheral blood mononuclear cells (PBMC) were obtained that can be used to generate dendritic cells (DC). The trial is not open to patients with recurrent glioblastoma.

For additional information about AIVITA’s AV-GBM-1 trial please visit: www.clinicaltrials.gov/ct2/show/NCT03400917

MELANOMA

AIVITA’s melanoma Phase 1B open-label, single-arm study will establish the safety of administering anti-PD1 monoclonal antibodies in combination with the cancer stem cell-targeting immunotherapy in patients with measurable metastatic melanoma. The study will also track efficacy of the treatment for the estimated 14 to 20 patients. This trial is not yet open for enrollment.

Patients eligible for treatment will be those (1) for whom a cell line has been established, (2) who have undergone leukapheresis from which sufficient monocytes were obtained, (3) have an ECOG performance grade of 0 or 1 (Karnofsky score of 70-100%), (4) who have either never received treatment for metastatic melanoma or were previously treated with enzymatic inhibitors of the BRAF/MEK pathway because of BRAF600E/K mutations and (5) are about to initiate anti-PD1 monotherapy.

For additional information about AIVITA’s AV-MEL-1 trial please visit: www.clinicaltrials.gov/ct2/show/NCT03743298

On March 28, 2019 Rubius Therapeutics, Inc. (Nasdaq:RUBY), a clinical-stage biopharmaceutical company that is generating red blood cells and bioengineering them into an entirely new class of cellular medicines, reported fourth quarter and full-year 2018 financial results (Press release, Rubius Therapeutics, MAR 28, 2019, View Source [SID1234534703]).

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"2018 was a transformative year for Rubius as we transitioned from a private, preclinical company into a public company on the brink of entering the clinic with our first program, RTX-134, for the treatment of patients with phenylketonuria," said Pablo J. Cagnoni, M.D., chief executive officer. "With our first Investigational New Drug application for RTX-134 now cleared by the U.S. Food and Drug Administration, we plan to begin treating patients in our Phase 1b trial during the second quarter with initial clinical data expected during the second half of the year. We will also be presenting preclinical data at the AACR (Free AACR Whitepaper) 2019 Annual Meeting, supporting our growing oncology pipeline. We stand well capitalized and well positioned to achieve our goal of filing four to five IND’s by the end of 2020, as we work to advance this new class of cellular medicines on behalf of patients."

Recent Business Highlights and Anticipated Upcoming Milestones

· In March 2019, Rubius announced that the U.S. FDA cleared the Company’s Investigational New Drug (IND) application for RTX-134, an allogeneic, off-the-shelf cellular therapy for the potential treatment of patients with phenylketonuria (PKU), an inherited metabolic disorder that is characterized by the body’s inability to metabolize the essential dietary amino acid, phenylalanine.

· Rubius expects to begin treating patients in a Phase 1b clinical trial in the second quarter of 2019 and plans to report initial clinical data from the trial during the second half of 2019.

·The primary objectives of the Phase 1b study are to evaluate preliminary safety, longevity of the RTX-134 cells in circulation, to obtain proof-of-mechanism as measured by production of trans-cinnamic acid (the byproduct of PAL) and select a preliminary dose and schedule.

·At this year’s American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, being held March 29-April 3, Rubius will present preclinical data from its oncology pipeline, including its lead oncology candidates RTX-240 (formerly RTX-212) and RTX-224 for the

treatment of solid tumors and RTX-aAPC (HPV+), an artificial antigen presenting cell, for the treatment of HPV+ tumors, including head and neck cancers.

·Rubius strengthened its leadership by appointing Natalie Holles to its board of directors and Greg Whitehead as senior vice president and chief quality officer.

·The Company plans to continue investing in its pipeline and expects to file its first oncology IND for RTX-240 for the treatment of solid tumors by early 2020, with two to three additional IND filings following in 2020.

·Rubius intends to continue renovating and hiring key personnel for its Smithfield, RI manufacturing facility with the goal of being operational in 1000L bioreactors by the end of 2020.

Successful Execution on Key Priorities in 2018

·Purchased 135,000 sq. ft. manufacturing facility in Smithfield, RI and initiated renovations

·Scaled manufacturing of RCTs from 50L to 200L bioreactors

· Transferred RTX-134 manufacturing process to contract manufacturing organization

·Executed three successful financings, including a $101.0 million crossover round, a $254.3 million initial public offering after expenses, and a recent debt financing of up to $75.0 million, which further extends cash runway into 2021

·Increased RCT storage stability from 28 days to 42 days with additional studies ongoing to explore extending RCT storage stability even further

·Continued to strengthen the board of directors, build a leading scientific team and attract experienced leadership to deliver against Rubius’ objectives

·Strengthened internal capabilities in discovery, platform development, technical operations and manufacturing

Fourth Quarter Financial Results

Net loss for the fourth quarter of 2018 was $27.2 million or $0.35 per common share, compared to $17.5 million or $2.07 per common share in the fourth quarter of 2017.

In the fourth quarter of 2018, Rubius invested $16.5 million in research and development (R&D) related to its novel RED PLATFORM and towards expanding and advancing its product pipeline, compared to $6.6 million in the fourth quarter of 2017. The year-over-year increase was due to an additional $2.4 million of costs incurred in preparation for the Phase 1b clinical trial for RTX-134, and $6.7 million was associated with personnel costs, stock-based compensation, facility and laboratory costs driven by increases in R&D headcount and expanded research activities to support Rubius’ goal of delivering four to five IND’s during 2019 and 2020.

G&A expenses were $12.6 million during the fourth quarter of 2018, as compared to $10.9 million for the fourth quarter of 2017. The higher costs were primarily driven by increases in

professional fees and infrastructure costs to support the Company’s growth and to operate as a public company.

Full Year Financial Results

Net loss for the full year 2018 was $89.2 million or $2.27 per common share, compared to $44.5 million or $5.55 per common share for the full year 2017.

For the full year 2018, Rubius invested $51.8 million in R&D related to its novel RED PLATFORM and towards expanding and advancing its product pipeline, compared to $21.2 million for the full year 2017. The year-over-year increase was primarily driven by an increase of $8.3 million of costs incurred in preparation for the Phase 1b clinical trial for RTX-134, and $19.1 million in personnel costs, stock-based compensation, facility and laboratory costs driven by increases in R&D headcount and expanded research activities to support Rubius’ goal of delivering four to five IND’s during 2019 and 2020.

G&A expenses were $39.9 million during the twelve months of 2018, as compared to $22.0 million for the same period in 2017. The higher costs were primarily driven by an $7.6 million increase in stock-based compensation and $10.3 million increase in personnel costs, facility and professional fees to support the Company’s growth and to operate as a public company.

Cash Flow Highlights

As of December 31, 2018, cash, cash equivalents and investments were $404.1 million as compared to $104.3 million as of December 31, 2017, providing Rubius with a cash runway into 2021. The year-end cash balance reflects $101.0 million of net proceeds received from its Series C preferred stock financing during the first quarter of 2018, $254.3 million of net proceeds from the Company’s initial public offering during the third quarter of 2018, and, in the fourth quarter, $25.0 million from a debt financing. During the fourth quarter, the Company used $19.7 million of cash in operations and used $3.3 million for capital purchases. During the full year, the Company used $58.3 million of cash in operations and $15.0 million for capital purchases, including $8.0 million to acquire the manufacturing facility in Smithfield, Rhode Island.