Rubius Therapeutics Reports Fourth Quarter and Full-Year 2018 Financial Results with Business Updates
Investigational New Drug Application Cleared for First-Ever Red Cell Therapeutic™, RTX-134, for Treatment of Phenylketonuria
Data from Company’s Oncology Pipeline to be Presented at AACR 2019 Annual Meeting
“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
Recent Business Highlights and Anticipated Upcoming Milestones
March 2019, Rubius announced that the U.S. FDAcleared 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) 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 Hollesto its board of directors and Greg Whiteheadas 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, RImanufacturing 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, RIand 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 millioncrossover round, a $254.3 millioninitial 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
In the fourth quarter of 2018, Rubius invested
G&A expenses were
Full Year Financial Results
Net loss for the full year 2018 was
For the full year 2018, Rubius invested
G&A expenses were
Cash Flow Highlights
About Rubius Therapeutics
Rubius Therapeutics is a clinical-stage biopharmaceutical company developing a new class of medicines called Red Cell Therapeutics™. The Company’s proprietary RED PLATFORM® was designed to genetically engineer and culture Red Cell Therapeutics™ that are selective, potent and off-the-shelf allogeneic cellular therapies for the potential treatment of several diseases across multiple therapeutic areas. Rubius’ initial focus is to advance RCT™ product candidates for the treatment of rare diseases, cancer and autoimmune diseases by leveraging three distinct therapeutic modalities — cellular shielding, potent cell-cell interaction and tolerance induction. For more information, visit www.rubiustx.com, or follow us on Twitter and LinkedIn.
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the planned timing, enrollment and results for our preclinical and clinical activities, including the Phase 1b clinical trial for RTX-134 for the treatment of PKU, our ability to fund the further development of our Red Cell Therapeutic product candidates, statements regarding the renovation and operation of our manufacturing facility, our expectations regarding the therapeutic potential of our Red Cell Therapeutics, including RTX-134 for the treatment of PKU, the timelines for us to file additional INDs, and our strategy, business plans and focus. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those risks and uncertainties related to the development of our Red Cell Therapeutic product candidates and their therapeutic potential and other risks identified in our SEC filings, including our Annual Report on Form 10-K for the year ended
Condensed Consolidated Statement of Operations
(in thousands, except share and per share data)
|For the three months
ended December 31,
|For the year ended
|Research and development||16,539||6,598||51,769||21,226|
|General and administrative||12,583||10,875||39,894||22,038|
|Total operating expenses||29,122||17,473||91,663||43,264|
|Loss from operations||(29,122||)||(17,473||)||(91,663||)||(43,264||)|
|Other income (expense), net||1,939||(44||)||2,468||(583||)|
|Accretion of Series A redeemable convertible preferred stock to redemption value||-||-||-||(656||)|
|Net loss attributable to common stockholders||$||(27,183||)||$||(17,517||)||$||(89,195||)||$||(44,503||)|
|Net loss per share attributable to common stockholders, basic and diluted||$||(0.35||)||$||(2.07||)||$||(2.27||)||$||(5.55||)|
|Weighted average common shares outstanding, basic and diluted:||76,747,827||8,478,687||39,285,468||8,023,785|
Condensed Consolidated Balance Sheet Data
|December 31,||December 31,|
|Cash, cash equivalents and investments||$||404,051||$||104,288|
|Total stockholders' deficit and convertible preferred stock||393,008||96,103|
Source: Rubius Therapeutics