On March 28, 2019 Adaptive Biotechnologies and its collaborators reported that it will present 11 studies, including an oral presentation and 10 poster presentations, at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in Atlanta, GA, March 29 – April 3 (Press release, Adaptive Biotechnologies, MAR 28, 2019, View Source [SID1234534717]).

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New data will demonstrate how immunosequencing at scale and with precision using Adaptive’s immunoSEQ Assay may be used in clinical trials to predict overall survival and response to novel immunotherapies, such as checkpoint inhibitors and cancer vaccines, in solid tumors. Other data will examine the ability of immunoSEQ to quantify and characterize the individual T cell receptors (TCR) of tumor infiltrating lymphocytes (TILs), a type of white blood cell that attacks cancer cells and controls tumor proliferation, to assist in identifying subtypes of colorectal cancer. This information may be used to help determine response to immunotherapy and patient outcomes among subtypes. Collectively the studies also demonstrate a growing use of immunoSEQ by academia and the pharmaceutical industry to help guide clinical research.

"Adaptive’s immune medicine platform is essential to helping our academic and pharmaceutical industry partners decode the information stored in our immune systems to answer translational research questions and discover new prognostic and diagnostic signals in oncology clinical trials," said Chad Robins, CEO and co-founder of Adaptive Biotechnologies. "Making this tool widely available to research partners helps us drive toward our goal of transforming medicine to improve patient lives."

Abstracts are available on AACR (Free AACR Whitepaper) website: View Source!/6812. immunoSEQ presentations of interest include:

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 (abstract #2272/1), poster presentation, April 1, 1:00 – 5:00 pm ET, Exhibit Hall B, Section 20
Tumor infiltrating lymphocytes, immunoSEQ, and CMS classification in the molecular epidemiology of colorectal cancer study, (abstract #2332/1), poster presentation, April 1, 1:00 – 5:00 pm ET, Exhibit Hall B, Section 23
Detection of tumor T-cell clones in mediastinal lymph nodes is associated with lower risk of tumor progression (abstract #3175/28), poster presentation, April 2, 8:00 am – 12:00 pm ET, Exhibit Hall B, Section 21
Spatially resolved immunogenomic analyses reveal diverse sub tumoral microenvironments in the context of melanoma immunotherapy (abstract #3776/9), April 2, 1:00 – 5:00 pm ET, Exhibit Hall B, Section 7
About immunoSEQ Assays

Adaptive’s immunoSEQ Assay helps researchers make discoveries in areas such as oncology, autoimmune disorders, infectious diseases and basic immunology. The immunoSEQ Assay can identify millions of T- and B-cell receptors from a single sample in exquisite detail. Offered as a Service or Kit, the immunoSEQ Assay is used to ask and answer translational research questions and discover new prognostic and diagnostic signals in clinical trials. immunoSEQ Assay provides quantitative, reproducible sequencing results along with access to powerful, easy-to-use analysis tools. The immunoSEQ Assay is for research use only and is not for use in diagnostic procedures

On March 28, 2019 Unum Therapeutics Inc. (NASDAQ: UMRX), a clinical-stage biopharmaceutical company focused on the development of cellular immunotherapies to treat cancer based on its novel T cell technology platforms, reported financial results and provided a corporate update for the full year ended December 31, 2018, and recent activities (Press release, Unum Therapeutics, MAR 28, 2019, View Sourcenews-releases/news-release-details/unum-therapeutics-reports-fourth-quarter-and-full-year-2018" target="_blank" title="View Sourcenews-releases/news-release-details/unum-therapeutics-reports-fourth-quarter-and-full-year-2018" rel="nofollow">View Source [SID1234534736]).

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"2018 was an important year for Unum as we brought the company public and continued to innovate in cell therapy with the advancement of our ACTR platform and the development of our new BOXR platform," said Chuck Wilson, President and CEO of Unum. "We have demonstrated proof of concept with the ACTR approach and see a clear path to further evaluating differentiated, potentially best-in-class product candidates designed to address continued patient needs. We are building out our solid tumor pipeline and have initiated our first solid tumor clinical trial, ATTCK-34-01, and selected BOXR1030 as our first product candidate from the BOXR platform. In 2019, we look forward to reporting data from all of our ongoing trials that will help determine next steps for our efforts in non-Hodgkin lymphoma, multiple myeloma, and solid tumors."

Recent Highlights

Continuing Dose Escalation in ATTCK-20-03 Phase I Trial: At the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December, Unum presented preliminary results from the first two dose levels of its ongoing Phase 1 study of ACTR707 in combination with rituximab in patients with relapsed/refractory CD20+ B cell non-Hodgkin lymphoma (r/r NHL). These data demonstrated that three of the six patients treated at Dose Level 1 (25 MM ACTR+ T cells) and one of three patients treated at Dose Level 2 (40 MM ACTR+ T cells) achieved a complete response. Enrollment and dose-limiting toxicity (DLT) assessments at Dose Level 3 (55 MM ACTR+ T cells) have since been completed, and enrollment at Dose Level 4 (80 MM ACTR+ T cells) has initiated. Through the first three cohorts, no DLTs and no severe adverse events of cytokine release syndrome (CRS) or neurologic events have been observed. Following completion of the dose escalation phase of the trial, Unum plans to initiate a cohort expansion at the preliminary recommended Phase 2 dose of ACTR707 in the second half of 2019.

Completed Enrollment of ATTCK-20-2 Phase I Trial: Following the decision in 2018 to prioritize ACTR707 for future development in r/r NHL, Unum has completed enrollment in the ATTCK-20-2 study, a Phase I clinical trial evaluating safety and anti-lymphoma activity of ACTR087 in combination with rituximab in patients with r/r NHL. Unum plans to report data on all enrolled patients from ATTCK-20-2 at the end of 2019. These data will inform other ACTR programs, specifically ATTCK-17-01, a Phase I trial of ACTR087 in combination with SEA-BCMA.

Continuing Dose Escalation with ATTCK-17-01 Phase I Trial: At the 2018 ASH (Free ASH Whitepaper) Annual Meeting in December, Unum presented data from the initial cohorts of the ongoing Phase 1 ATTCK-17-01 study, testing ACTR087 in combination with SEA-BCMA (an investigational monoclonal antibody targeting B cell maturation antigen, or BCMA) in patients with relapsed/refractory multiple myeloma (r/r MM). In the first three cohorts, no DLTs and no severe adverse events of CRS or neurologic events were observed. Having cleared cohorts with very low levels of SEA-BCMA antibody administered, dose escalation is continuing now at doses of SEA-BCMA that may be expected to have pharmacological activity based upon preclinical studies. Enrollment at Dose Level 4 (30 MM ACTR+ T cells and 2.0 mg/kg SEA-BCMA) is ongoing. In subsequent dose cohorts, the dose of both ACTR087 and SEA-BCMA will be explored. Unum expects to continue to enroll and dose patients through the dose escalation phase of the trial and to report data from multiple dose cohorts in the second half of 2019.

Initiated ATTCK-34-01 Phase I Trial in Solid Tumors: Unum announced that it has initiated ATTCK-34-01, a Phase 1, multicenter, single-arm, open-label dose escalation study evaluating ACTR T cells in combination with trastuzumab for the treatment of patients with HER2+ advanced cancers. This is Unum’s first ACTR T cell study in solid tumors. The primary study objectives are to assess the safety and tolerability of the combination, and to define dose recommendations for further development. Additional objectives include assessment of anti-tumor activity, ACTR T cell persistence, and trastuzumab pharmacokinetics. Unum plans to report initial clinical data from the ongoing dose escalation at the end of 2019.

Initiated Preclinical Development of the First Product Candidate from the BOXR Platform: In November 2018, Unum announced its new Bolt-On Chimeric Receptor technology platform (BOXR), which improves T cell functionality by countering immunosuppression in solid tumor cancers. Unum presented preclinical data at the 2018 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) meeting demonstrating the ability to significantly improve the activity of both ACTR- and CAR-targeted engineered T cells in immunosuppressive solid tumors. Unum has subsequently nominated BOXR1030 as the first product candidate from this platform and has initiated IND-enabling preclinical studies and plans to present additional information regarding BOXR1030 in the second half of 2019. BOXR1030 consists of a T cell co-expressing a glypican-3 (GPC3)-directed CAR and an undisclosed metabolism-enhancing bolt-on transgene. GPC3 is a well-known oncofetal antigen selectively expressed in a variety of tumor types including certain liver and lung cancers.
Fourth Quarter 2018 Financial Results

Collaboration Revenue: Collaboration revenue recognized during the fourth quarter ended December 31, 2018 was $3.8 million, compared to $2.1 million in the same period of 2017. The increase reflects the recognition of a portion of the $25.0 million upfront payment received from Seattle Genetics under Unum’s collaboration agreement as well as reimbursements of research and development costs attributed to the ATTCK-17-01 study.

R&D Expenses: Research and development expenses were $10.8 million for the fourth quarter ended December 31, 2018, compared to $7.6 million for the same period of 2017. The increase reflects higher clinical trial costs for the active Phase I clinical trials, as well as increased personnel-related costs, materials and facility-related costs related to scaling manufacturing processes, and increased consultant costs to support these activities.

G&A Expenses: General and administrative expenses for the fourth quarter ended December 31, 2018, were $2.0 million, compared to $1.4 million for the same period of 2017. The increase is primarily due to expenses around operating as a public company and higher personnel related costs.

Net Loss: Net loss attributable to common stockholders was $8.6 million, or $0.29 per share, for the fourth quarter ended December 31, 2018, and $6.7 million, or $0.66 per share, for the same period of 2017.

Cash, Cash Equivalents and Marketable Securities: As of December 31, 2018, Unum had cash, cash equivalents, and marketable securities of $78.6 million. Total cash burn for 2018, net of IPO related costs, was $36.3 million. Today, Unum updated its cash runway and now anticipates that its existing cash, cash equivalents, and marketable securities will fund operating expenses and capital expenditure requirements into early 2021.
Investor Call and Webcast Information

Unum will host a live conference call and webcast today, March 28, 2019, at 8:00 a.m. ET, to discuss these financial results and company updates. To access the call, please dial 866-300-3411 (domestic) or 636-812-6658 (international) and refer to conference ID number 8169027. A webcast will be available at View Source at least 10 minutes before the event begins. The archived webcast will be available at the same location approximately two hours after the event and will be archived for 90 days.

On March 28, 2019 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology,reported its financial results for the year ended December 31, 2018 and provided a corporate update (Press release, BioLineRx, MAR 28, 2019, View Source;p=RssLanding&cat=news&id=2392600 [SID1234534702]).

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Highlights and achievements during the fourth quarter 2018 and subsequent period:

Presented data from Phase 2a COMBAT/KEYNOTE-202 pancreatic cancer study in collaboration with Merck at the ESMO (Free ESMO Whitepaper) 2018 Congress demonstrating that BL-8040 in combination with KEYTRUDA (pembrolizumab) showed encouraging disease control and overall survival in patients with metastatic pancreatic cancer; compelling pharmacodynamic data also demonstrated T-cell infiltration into tumors and a reduction of the tumor immuno-suppressive microenvironment;
Initiated a triple combination arm of COMBAT/KEYNOTE-202 evaluating the safety, tolerability and efficacy of BL-8040 in combination with KEYTRUDA and chemotherapy;
Entered into agreement with Biokine Therapeutics to increase the Company’s economic stake in BL-8040 to 80% from the previous level of 60%;
Initiated Phase 1/2a multicenter, open-label clinical study in the UK and Israel for AGI-134, a novel immunotherapy evoking a direct anti-tumor response and vaccine effect for the treatment of solid tumors;
Announced FDA Orphan Drug Designation for BL-8040, for the treatment of pancreatic cancer. This is in addition to prior orphan drug designations received for BL-8040 in AML and stem cell mobilization;
Announced FDA Biological Product Designation for AGI-134, providing the Company with eligibility to obtain 12 years of market exclusivity upon approval of the product for commercial use by the FDA; and
Completed an underwritten public offering for gross proceeds of $15.4 million.
"During the fourth quarter, we continued to advance our novel pipeline of promising anti-cancer therapies toward significant and potentially value-creating milestones, and this progress was a key driver in our previously announced decision to acquire an additional 20% economic stake in BL-8040 from Biokine Therapeutics," said Philip Serlin, Chief Executive Officer of BioLineRx. "In cancer immunotherapy, following the encouraging results we announced from the dual combination arm, we initiated the triple combination arm of the COMBAT/KEYNOTE-202 study evaluating our lead therapeutic candidate, BL-8040, in combination with Merck’s KEYTRUDA and chemotherapy for the treatment of metastatic pancreatic cancer, an indication for which we also recently received FDA Orphan Drug Designation."

"In stem-cell mobilization, our most advanced indication, we continue to move forward with the Phase 3 GENESIS study in the randomized placebo-controlled phase of the trial, and we hope that we will be able to replicate the compelling results observed in the lead-in portion of the trial. Concurrently, in relapsed/refractory AML, we are evaluating our future development plan, and anticipate meeting with the regulatory authorities to discuss the plan during the second half of this year. In consolidation AML, we hope to announce interim data from the large, randomized placebo-controlled Phase 2b BLAST study by the end of this year as well."

"Finally, our second oncology program, the cancer immunotherapy vaccine AGI-134, is also progressing as planned, with initial safety data from the ongoing Phase 1/2a study expected later this year. As we progress through 2019, we are rapidly approaching important data readouts that we believe can create significant shareholder value and additional partnering interest, and we look forward to providing future updates throughout the year."

Expected significant milestones through end of 2019 and early 2020:

Top-line results from the Phase 2 triple combo pancreatic cancer trial of BL-8040, KEYTRUDA and chemotherapy under collaboration with Merck toward the end of 2019;
Potential interim results from Phase 2 AML consolidation study in the second half of 2019;
Initial safety results from part 1 of Phase 1/2a trial for AGI-134 in second half of 2019;
Top-line results from one or more of the solid tumor trials under collaboration with Genentech, potentially by end of 2019 or early 2020.
Financial Results for the Year Ended December 31, 2018

Research and development expenses for the year ended December 31, 2018 were $19.8 million, an increase of $0.3 million, or 1.5%, compared to $19.5 million for the year ended December 31, 2017. The small increase resulted primarily from an increase in share-based compensation.

Sales and marketing expenses for the year ended December 31, 2018 were $1.4 million, a decrease of $0.3 million, or 19.6%, compared to $1.7 million for the year ended December 31, 2017. The decrease resulted primarily from one-time legal fees related to AGI-134, as well as market research for BL-8040 and AGI-134, incurred in the 2017 period.

General and administrative expenses for the year ended December 31, 2018 were $4.4 million, an increase of $0.4 million, or 9.9% compared to $4.0 million for the year ended December 31, 2017. The increase resulted primarily from an increase in share-based compensation.

The Company’s operating loss for the year ended December 31, 2018 amounted to $25.6 million, compared with an operating loss of $25.2 million for the year ended December 31, 2017.

Non-operating income amounted to $2.4 million for the year ended December 31, 2018, compared with non-operating expenses of $0.3 million for the year ended December 31, 2017. Non-operating income for the year ended December 31, 2018 primarily relates to fair-value adjustments of warrant liabilities and a capital gain from realization of the investment in iPharma. Non-operating expenses for the year ended December 31, 2017 primarily relate to fair-value adjustments of warrant liabilities.

Net financial income amounted to $0.2 million for the year ended December 31, 2018 compared to net financial income of $1.1 million for the year ended December 31, 2017. Net financial income for the year ended December 31, 2018 primarily relates to investment income earned on bank deposits, offset by interest paid on loans. Net financial income for the year ended December 31, 2017 relates primarily to gains recorded on foreign currency hedging transactions and investment income earned on bank deposits.

The Company’s net loss for the year ended December 31, 2018 amounted to $23.0 million, compared with a net loss of $24.4 million for the year ended December 31, 2017.

The Company held $30.2 million in cash, cash equivalents and short-term bank deposits as of December 31, 2018. Subsequent to year end, the Company raised $15.4 million of gross proceeds from an underwritten public offering.

Net cash used in operating activities for the year ended December 31, 2018 was $24.2 million, compared to $20.5 million for the year ended December 31, 2017. The $3.7 million increase in 2018 was the result of a decrease in accounts payable and an increase in prepaid expenses and other receivables.

Net cash provided by investing activities for the year ended December 31, 2018 was $9.6 million, compared to net cash used in investing activities of $15.9 million for the year ended December 31, 2017. The changes in cash flows from investing activities relate primarily to investments in, and maturities of, short-term bank deposits during both periods, the acquisition of Agalimmune in 2017, and the acquisition of an additional 20% of BL-8040 sublicense receipts, as well as realization of the investment in iPharma, during 2018.

Net cash provided by financing activities for the year ended December 31, 2018 was $13.1 million, compared to $38.7 million for the year ended December 31, 2017. The cash flows in 2018 primarily reflect the net proceeds of the loan from Kreos Capital, as well as net proceeds from the ATM program. The cash flows in 2017 primarily reflect the underwritten public offering of our ADSs in March 2017 and the direct placement of ADSs and warrants to BVF Partners in July 2017.

Conference Call and Webcast Information

BioLineRx will hold a conference call today, March 28, 2019 at 10:00 a.m. EDT. To access the conference call, please dial +1-866-229-7198 from the U.S. or +972-3-918-0664 internationally. The call will also be available via webcast and can be accessed through the Investor Relations page of BioLineRx’s website. Please allow extra time prior to the call to visit the site and download any necessary software to listen to the live broadcast.

A replay of the conference call will be available approximately two hours after completion of the live conference call on the Investor Relations page of BioLineRx’s website. A dial-in replay of the call will be available until March 30, 2019; please dial +1-888-295-2634 from the U.S. or +972-3-925-5938 internationally

On March 28, 2019 Odonate Therapeutics, Inc. (NASDAQ: ODT), a pharmaceutical company dedicated to the development of best-in-class therapeutics that improve and extend the lives of patients with cancer, reported the initiation of CONTESSA TRIO, a multi-cohort, multicenter, Phase 2 study of tesetaxel, Odonate’s investigational, orally administered taxane (Press release, Odonate Therapeutics, MAR 28, 2019, View Source [SID1234534718]).

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In Cohort 1, approximately 90 patients (with potential expansion to up to 150 patients) with locally advanced or metastatic TNBC who have not received prior chemotherapy for advanced disease will be randomized 1:1:1 to receive tesetaxel plus either: (1) nivolumab; (2) pembrolizumab; or (3) atezolizumab. Nivolumab and pembrolizumab (PD-1 inhibitors) and atezolizumab (a PD-L1 inhibitor) are IO agents approved for the treatment of multiple types of cancer. One of these agents, atezolizumab, in combination with the intravenously delivered taxane, nab-paclitaxel, was recently approved by the U.S. Food and Drug Administration (FDA) as a first-line treatment for patients with metastatic TNBC. The dual primary endpoints for Cohort 1 are objective response rate (ORR) and progression-free survival (PFS). Secondary endpoints include duration of response (DoR) and overall survival (OS). Efficacy results for each of the three PD-(L)1 inhibitor combinations will be assessed for correlation with the results of each of the three approved PD-L1 diagnostic assays.
In Cohort 2, approximately 40 elderly patients (with potential expansion to up to 60 patients) with HER2 negative MBC who have not received prior chemotherapy for advanced disease will receive tesetaxel monotherapy. The primary endpoint for Cohort 2 is ORR. Secondary endpoints include PFS, DoR and OS.
"Taxane-IO combinations hold great promise for patients living with TNBC," said Sara Tolaney, M.D., M.P.H., Associate Director, Susan F. Smith Center for Women’s Cancers, Director, Clinical Trials, Breast Oncology at Dana-Farber Cancer Institute and Principal Investigator of CONTESSA TRIO. "This study will investigate the safety and antitumor activity of tesetaxel, an orally administered taxane with a distinct tolerability and pharmacokinetic profile, in combination with three approved PD-(L)1 inhibitors. CONTESSA TRIO also will investigate tesetaxel monotherapy in elderly patients with MBC, a patient population in need of easier-to-take and better tolerated therapies."

About Tesetaxel

Tesetaxel is an investigational, orally administered chemotherapy agent that belongs to a class of drugs known as taxanes, which are widely used in the treatment of cancer. Tesetaxel has several pharmacologic properties that make it unique among taxanes, including: oral administration with a low pill burden; a long (~8-day) terminal plasma half-life in humans, enabling the maintenance of adequate drug levels with relatively infrequent dosing; no history of hypersensitivity (allergic) reactions; and significant activity against chemotherapy-resistant tumors. In patients with metastatic breast cancer, tesetaxel was shown to have significant, single-agent antitumor activity in two multicenter, Phase 2 studies.

About CONTESSA TRIO

CONTESSA TRIO is a multi-cohort, multicenter, Phase 2 study of tesetaxel, an investigational, orally administered taxane, in patients with locally advanced or metastatic breast cancer (MBC). In Cohort 1, approximately 90 patients (with potential expansion to up to 150 patients) with locally advanced or metastatic triple-negative breast cancer (TNBC) who have not received prior chemotherapy for advanced disease will be randomized 1:1:1 to receive tesetaxel dosed orally at 27 mg/m2 on the first day of each 21-day cycle plus either: (1) nivolumab at 360 mg by intravenous infusion on the first day of each 21-day cycle; (2) pembrolizumab at 200 mg by intravenous infusion on the first day of each 21-day cycle; or (3) atezolizumab at 1,200 mg by intravenous infusion on the first day of each 21-day cycle. Nivolumab and pembrolizumab (PD-1 inhibitors) and atezolizumab (a PD-L1 inhibitor) are immuno-oncology (IO) agents approved for the treatment of multiple types of cancer. One of these agents, atezolizumab, in combination with the intravenously delivered taxane, nab-paclitaxel, was recently approved by the U.S. Food and Drug Administration (FDA) as a first-line treatment for patients with metastatic TNBC. The dual primary endpoints for Cohort 1 are objective response rate (ORR) and progression-free survival (PFS). Secondary endpoints include duration of response (DoR) and overall survival (OS). Efficacy results for each of the three PD-(L)1 inhibitor combinations will be assessed for correlation with the results of each of the three approved PD-L1 diagnostic assays. In Cohort 2, approximately 40 elderly patients (with potential expansion to up to 60 patients) with human epidermal growth factor receptor 2 (HER2) negative MBC will receive tesetaxel monotherapy dosed orally at 27 mg/m2 on the first day of each 21-day cycle. The primary endpoint for Cohort 2 is ORR. Secondary endpoints include PFS, DoR and OS. Patients with central nervous system metastases are eligible for both cohorts

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.