On May 15, 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 medicine reported first quarter 2019 financial results and provided an overview of operational progress (Press release, Rubius Therapeutics, MAY 15, 2019, View Source [SID1234536305]). In addition, Rubius announced that company president, Torben Straight Nissen, Ph.D., will be leaving the Company at the end of July 2019, to join Flagship Pioneering where he has held a venture partner role since joining Rubius in 2016.

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"Rubius has made significant progress this year, including the clearance of our first Investigational New Drug application for RTX-134 for the treatment of patients with phenylketonuria," said Pablo J. Cagnoni, M.D., chief executive officer of Rubius Therapeutics. "We plan to begin enrolling patients in the Phase 1b trial during the second quarter with initial clinical data expected during the second half of the year. We presented important preclinical data from our emerging oncology portfolio at AACR (Free AACR Whitepaper), demonstrating promising anti-tumor activity with no observed toxicities for our lead product candidates, RTX-240 and RTX-224, for the treatment of solid tumors and RTX-aAPC for the treatment of HPV-positive tumors. The year is off to a great start as we continue to advance our Red Cell Therapeutics towards our ultimate goal of treating patients who are underserved by available therapies."

"We are grateful to Torben for all his contributions towards building a world-class organization and for remaining with Rubius since I joined the company to continue supporting the scale-up of our manufacturing capabilities," continued Dr. Cagnoni. "During his tenure, Torben also took part in executing key financings, made important leadership hires and helped drive the formation and advancement of our portfolio of programs that target rare diseases, cancer and autoimmune diseases. We wish him the best going forward."

"I am very proud of the hard work, dedication and innovative spirit of the Rubius team that has led the company to where it is today," said Dr. Straight Nissen. "I believe Rubius’ Red Cell Therapeutics approach has the potential to transform the future of cellular medicine, and I look forward to following the company as they work towards bringing these important therapies to patients. I wish all my talented colleagues at Rubius continued success as I transition to focus on earlier stage platform companies within the Flagship Pioneering ecosystem."

Dr. Straight Nissen will continue to provide consulting services to Rubius into 2020. With his departure, Spencer Fisk, senior vice president of technical operations, and Thomas Wickham, Ph.D., senior vice president of discovery, will report directly to Dr. Cagnoni.

First Quarter Program Highlights and Updates

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

oThe Company expects to enroll up to 10 adult patients with PKU in the Phase 1b trial, with enrollment expected to begin during the second quarter of 2019 and initial clinical data anticipated in the second half of 2019, including:

Preliminary safety;

Longevity of RTX-134 cells in circulation;

Proof-of-mechanism as measured by production of trans-cinammic acid, the metabolite of phenylalanine when degraded by phenylalanine ammonia lyase (PAL); and

Selection of a preliminary dose and schedule.

On April 2, 2019, Rubius presented preclinical data from its emerging oncology pipeline at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.

oRTX-240 (4-1BBL and 1L-15TP) and RTX-224 (4-1BBL and IL-12) are allogeneic cellular therapy product candidates that are engineered to replicate the human immune system by stimulating the adaptive and innate immune systems to induce an immune response.

RTX-240 and RTX-224 each demonstrated potent activation of the immune system, anti-tumor activity and a potentially broad therapeutic window when compared to agonists antibodies or recombinant cytokines.

Additionally, RTX-224 in combination with an anti-PD-1 antibody demonstrated substantial tumor shrinkage in a difficult-to-treat MC38 colon cancer model.

oRed Cell Therapeutic (RCT) artificial antigen presenting cells, or RTX-aAPCs, are engineered to induce a tumor-specific response by expanding tumor-specific T cells against a target antigen.

RTX-aAPCs activated and significantly expanded antigen-specific T cells to promote substantial tumor regressions, leading to increased survival with no observed toxicity, in an OVA-positive murine model.

The Company continued to strengthen its leadership by appointing Natalie Holles to its board of directors and Greg Whitehead as senior vice president and chief quality officer, while bolstering internal capabilities in discovery, platform development and technical operations.

Rubius remains on track to submit a total of four to five INDs across 2019 and 2020, while continuing to progress additional pipeline programs in rare diseases, cancer and autoimmune diseases.

First Quarter Financial Results

Net loss for the first quarter of 2019 was $32.6 million or $0.42 per common share, compared to $14.4 million or $1.72 per common share in the first quarter of 2018.

In the first quarter of 2019, Rubius invested $20.9 million in research and development (R&D) related to its novel RED PLATFORM and towards expanding and advancing its product pipeline, compared to $9.5 million in the first quarter of 2018. The year-over-year increase was due to an additional $3.6 million of costs incurred in preparation for the Phase 1b clinical trial for RTX-134, and $9.3 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 across 2019 and 2020. These increases were offset by a decrease in external manufacturing and research costs that were not related to programs.

G&A expenses were $13.5 million during the first quarter of 2019, as compared to $5.1 million for the first quarter of 2018. The higher costs were primarily driven by a $7.4 million increase in personnel costs, stock-based compensation and facility costs, due to increased headcount in our general and administrative function, as well as increases in professional fees and infrastructure costs to support the Company’s growth and to operate as a public company.

Cash Position

As of March 31, 2019, cash, cash equivalents and investments were $378.9 million as compared to $404.1 million as of December 31, 2018, providing Rubius with a cash runway into 2021. During the quarter, the Company used $22.9 million of cash to fund operations and $4.2 million to fund capital expenditures.