Rubius Therapeutics Reports Second Quarter 2019 Financial Results and Operational Progress
On Track to Report Initial Clinical Data from Phase 1b Clinical Trial of RTX-134 for Phenylketonuria by Year-End
“During the second quarter, we continued to advance our promising pipeline of Red Cell Therapeutics™ for the potential treatment of rare diseases, cancer and autoimmune diseases,” said
Second Quarter Highlights and Upcoming Milestones
- Rubius is working closely with its contract manufacturer to improve its ability to consistently produce product in 200-liter bioreactors in order for Rubius to begin enrolling patients in its Phase 1b clinical trial for patients with phenylketonuria (PKU) in the near term.
- Rubius plans to report initial clinical data from the first patients enrolled in the trial by year-end, including:
- Preliminary safety;
- Longevity of RTX-134 cells in circulation; and
- Proof-of-mechanism as measured by production of trans-cinnamic acid, the metabolite of phenylalanine when degraded by phenylalanine ammonia lyase, or PAL.
- The Company continued to strengthen its leadership by appointing
Kris Elverum as senior vice president of business development and strategy to oversee corporate strategy and create and execute potential new business development opportunities. - On
June 5, 2019 , Rubius announced the issuance of three patents from the U.S. Patent and Trademark Office related to its rare disease and oncology programs and platform.- Two of the issued patents cover composition of matter and methods of use for the Company’s lead investigational cellular therapy, RTX-134, for the treatment of patients with PKU.
- The third patent, the Company’s first issued composition of matter patent in immuno-oncology, relates to human cells having a checkpoint inhibitor linked to the cell surface.
- Today, in total, Rubius Therapeutics has 30 patent families and more than 130 pending patent applications worldwide.
Second Quarter Financial Results
Net loss for the second quarter of 2019 was
In the second quarter of 2019, Rubius invested
G&A expenses were
Six Month Financial Results
Net loss for the first six months of 2019 was
In the six months ended
G&A expenses were
Cash Position
As of
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, recruitment, 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 further develop our RCT product candidates, our manufacturing process, our expectations regarding the therapeutic potential of our RCTs, our expectations regarding new members of our leadership team 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 RCT product candidates and their therapeutic potential and other risks identified in our SEC filings, including our Quarterly Report on Form 10-Q for the quarter ended June 30, 2019, and subsequent filings with the SEC. We caution you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. We disclaim any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent our views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. We explicitly disclaim any obligation to update any forward-looking statements.
Condensed Consolidated Statement of Operations
(in thousands, except share and per share data)
(unaudited)
| For the three months ended June 30, |
For the six months ended June 30, |
||||||||||||||||
| 2019 | 2018 | 2019 | 2018 | ||||||||||||||
| Revenue | $ | $ | $ | $ | |||||||||||||
| Operating expenses: | |||||||||||||||||
| Research and development | 27,518 | 11,361 | 48,389 | 20,867 | |||||||||||||
| General and administrative | 13,767 | 9,023 | 27,302 | 14,120 | |||||||||||||
| Total operating expenses | 41,285 | 20,384 | 75,691 | 34,987 | |||||||||||||
| Loss from operations | (41,285 | ) | (20,384 | ) | (75,691 | ) | (34,987 | ) | |||||||||
| Other income (expense), net | 1,895 | (855 | ) | 3,720 | (663 | ) | |||||||||||
| Net loss | $ | (39,390 | ) | $ | (21,239 | ) | $ | (71,971 | ) | $ | (35,650 | ) | |||||
| Net loss per share, basic and diluted | $ | (0.50 | ) | $ | (2.43 | ) | $ | (0.92 | ) | $ | (4.17 | ) | |||||
| Weighted average common shares outstanding, basic and diluted: |
78,396,714 | 8,727,392 | 77,972,757 | 8,542,362 | |||||||||||||
Condensed Consolidated Balance Sheet Data
(in thousands)
(unaudited)
| June 30, | December 31, | ||||
| 2019 | 2018 | ||||
| Cash, cash equivalents and investments | $ | 362,330 | $ | 404,051 | |
| Total assets | 438,032 | 479,109 | |||
| Total liabilities | 94,388 | 86,101 | |||
| Total stockholders’ equity and convertible preferred stock | 343,644 | 393,008 | |||
Contacts:
Lori Melançon
Vice President, Corporate Communications and Investor Relations
+1 (617) 949-5296
lori.melancon@rubiustx.com
Media Contact:
1AB
+1 (973) 271-6085
dan@1abmedia.com
Source: Rubius Therapeutics