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|Dimension Therapeutics Announces Full Year 2016 Financial Results and Recent Updates Across Rare and Metabolic Disease Portfolio and in HeLa Manufacturing|
In December, initiated Phase 1/2 clinical trial of DTX301, Dimension’s lead IMD product candidate for OTC Deficiency; Initial data expected 2H 2017
Updates interim results from Phase 1/2 clinical program for DTX101 in hemophilia B
Conference call today at
“During 2016, we further advanced the science and manufacturing of AAV-based gene therapy including the continued development of our next generation HeLa 2.0 platform and initiated our global Phase 1/2 clinical trial for DTX301, our lead IMD product candidate for the treatment of ornithine transcarbamylase (OTC) deficiency,” said
DTX101 Phase 1/2 Clinical Program
DTX101 is designed to deliver stable expression of blood clotting Factor IX (FIX) in patients with hemophilia B, a rare genetic bleeding disorder resulting from a deficiency in FIX. In the Phase 1/2 open-label study, patients received a capsid serotype AAVrh10 vector with a codon-optimized FIX gene expressing wild-type FIX protein. In
“The encouraging clinical data from Dimension’s lead program are supported by what we have seen in our patients who participated in the study, many of whom have anecdotally related the positive impact of therapy on their well-being. These data add to the growing body of evidence that restoration of clotting function may be achieved by gene therapy,” said Dr.
Other Recent Highlights and Upcoming Milestones
Full Year 2016 Financial Results
About the Hemophilia B Phase 1/2 Program
Dimension’s phase 1/2 clinical trial of DTX101 is a single arm, open-label, multi-center study, designed to evaluate the safety, dose, and early efficacy of DTX101 in adult patients with moderate/severe to severe hemophilia B. Patients enrolled in Cohorts 1 and 2 range in age from 28 to 70 years, demonstrating baseline FIX expression of ≤2% that requires either prophylactic or on demand recombinant FIX transfusion. Dimension is continuing to explore underlying demographics and patient characteristics to optimize dosing of DTX101.
Additional information about Dimension’s Phase 1/2 study of DTX101 may be found at ClinicalTrials.gov, using Identifier NCT: NCT02618915.
Conference Call Information
The live webcast can be accessed by visiting the investor relations section of the
For more information, please visit www.dimensiontx.com.
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 development, preclinical and clinical results, upcoming milestones, the potential productivity of Dimension’s ongoing collaborations and the continued progress of Dimension's portfolio and programs, including the initiation, timing, scope, or 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 candidates, DTX101 and DTX301, will not successfully be developed or commercialized in the times indicated or at all; the impact of the observed data in Cohorts 1 and 2, including the adverse event described above and any later safety event on timing, dosing, regulatory action or patient enrollment with respect to DTX101 and DTX301; and the risks described under the caption "Risk Factors" in Dimension Therapeutics’ Annual Report on Form 10-K for the year ended
This selected financial information should be read in conjunction with the audited, condensed consolidated financial statements and notes included in the Company's Annual Report on Form 10-K for the year ended