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|Dimension Therapeutics Reports First Quarter 2017 Financial Results and Provides Corporate Update|
Initial data from Phase 1/2 clinical trial of DTX301, Dimension’s lead AAV8 vector IMD product candidate for OTC Deficiency, expected 2H 2017
The company announced its decision to discontinue the development of DTX101, an investigational AAVrh10-based gene therapy product in development for the treatment of moderate/severe-to-severe hemophilia B. The decision followed the review of the emerging DTX101 Phase 1/2 clinical study data, including the data as of the beginning of
“We are disappointed with the outcome of our DTX101 program, addressing an important disease with significant unmet need; however, our Phase 1/2 open-label clinical study did not demonstrate an ability to achieve a minimum target product profile for continued development or future commercialization,” said
Dr. Jenkins continued, “We remain excited about the opportunities around our IMD portfolio, which, unlike DTX101, utilizes the AAV8 capsid, and look forward to initial data later this year with DTX301 for OTC deficiency.” The company does not believe the outcome of the DTX101 program will affect the ongoing Phase 1/2 clinical development of DTX301, Dimension’s lead AAV8-based gene therapy, in OTC deficiency, or current vector design for the company’s other investigational AAV therapeutic programs in development. While the company remains focused on the development of our IMD programs testing AAV8 based vectors, it will be undertaking a comprehensive portfolio prioritization review to thoroughly examine resources and the opportunities to focus efforts, which review is expected to be completed by the end of the second quarter of 2017.
Recent Highlights and Upcoming Milestones
- Inherited Metabolic Disease (IMD) Programs -
• Continued to advance robust portfolio of IMD candidates utilizing the capsid serotype AAV8 - DTX301 for OTC deficiency, DTX401 for GSDIa, DTX501 for PKU, DTX701 for Wilson disease, and DTX601 for citrullinemia type I:
- Hemophilia A -
• DTX201: Currently in IND-enabling studies in collaboration with Bayer for the treatment of moderate/severe to severe hemophilia A.
First Quarter 2017 Financial Results
Cautionary Note Regarding Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the potential productivity of Dimension’s ongoing collaborations, timing and likelihood of achievement of Dimension’s upcoming development milestones, including timing of disclosure of data, the expected progress of Dimension's portfolio and programs, timing and likelihood of regulatory filings and approvals, and our ability to develop and advance product candidates into, and successfully complete, clinical studies. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include the risks that Dimension’s product candidates, including its candidate, DTX301, will not achieve development milestones, including patient enrollment, dosing of patients, release of initial data, or regulatory filings; and the risks described under the caption "Risk Factors" in Dimension Therapeutics’ Quarterly Report on Form 10-Q for the period ended
This selected financial information should be read in conjunction with the unaudited, condensed consolidated financial statements and notes included in the Company's Quarterly Report on Form 10-Q for the quarter ended