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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 8:30 a.m. Eastern Time
CAMBRIDGE, Mass., March 09, 2017 (GLOBE NEWSWIRE) -- Dimension Therapeutics, Inc. (NASDAQ:DMTX), a biopharmaceutical company advancing novel, adeno-associated virus (AAV) gene therapies targeting the liver, a key organ for human metabolism, today provided updated interim results from the Company’s Phase 1/2 clinical program of DTX101 for the treatment of adult patients with moderate/severe to severe hemophilia B. In addition, Dimension today reported financial results for the full year ended December 31, 2016 and provided an update on the Company’s recent corporate progress across its portfolio of inherited metabolic disease (IMD) programs.
“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 Annalisa Jenkins, MBBS, FRCP, Chief Executive Officer of Dimension. “We also continue to explore options for DTX101 as our data mature, while remaining laser-focused on maximizing the value of our portfolio. We are dedicated to advancing these and other key programs in order to bring hope to patients and their families living with rare genetic diseases associated with the liver.”
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 January 2017, the Company completed dosing in the first two cohorts (Cohort 1: N=3, 1.6 x 1012 GC/kg; Cohort 2: N=3, 5 x 1012 GC/kg) and reported interim topline data. All patients in both cohorts improved from moderate/severe-to-severe to either moderate or mild range in terms of factor levels based on World Federation of Hemophilia (WFH) criteria. In addition, none of the patients in cohort 2 have required prophylactic or on-demand recombinant FIX transfusion for spontaneous bleeds post-dosing. As required by the clinical trial protocol, the Company reported the ALT levels for patient 3 in cohort 2 to the Data Safety Monitoring Committee (DSMC), the U.S. Food and Drug Administration (FDA), and other appropriate regulatory authorities, and remains in ongoing communications. Dimension will await feedback prior to initiating dosing of Cohort 3.
“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. Steven Pipe, Director of the Division of Pediatric Hematology and Oncology and Pediatric Medical Director of the Hemophilia and Coagulation Disorders Program at the C.S. Mott Children’s Hospital of the University of Michigan, and the lead investigator on the clinical trial. “I look forward to the further progress of the clinical program, which has demonstrated the potential for patients to avoid bleeding and to have options that potentially minimize or eliminate the need for infusions of factor concentrates.”
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
Dimension Therapeutics will host a live conference call and webcast today, March 9, 2017 at 8:30 a.m. Eastern Time. Members of the Company’s management team will also be joined by Dr. Steven Pipe, Director of the Division of Pediatric Hematology and Oncology and Pediatric Medical Director of the Hemophilia and Coagulation Disorders Program at the C.S. Mott Children’s Hospital of the University of Michigan.
The live webcast can be accessed by visiting the investor relations section of the Dimension Therapeutics website at www.dimensiontx.com. Please connect to the Company's website at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast. Alternatively, please call 844-804-8784 (U.S.) or 210-229-8833 (International) to listen to the conference call. The conference ID number for the live call will be 84164743. An archive of the webcast will be available until March 23, 2017 on the investor relations section of the Company’s website.
About Dimension Therapeutics, Inc.
Dimension Therapeutics, Inc. (NASDAQ:DMTX) is a leader in discovering and developing new therapeutic products for people living with devastating rare and metabolic diseases associated with the liver, based on the most advanced mammalian adeno-associated virus (AAV) gene delivery technology. Dimension is actively progressing its broad pipeline, which features programs addressing unmet needs for patients suffering from inherited metabolic diseases, including OTC deficiency, GSDIa, citrullinemia type 1, PKU, Wilson disease, a collaboration with Bayer in hemophilia A, and a wholly owned clinical program in hemophilia B. Dimension has two phase 1/2 clinical trials for the treatment of hemophilia B and OTC deficiency. The company targets diseases with readily identifiable patient populations, highly predictive preclinical models, and well-described, and often clinically validated, biomarkers. Founded in 2013, Dimension maintains headquarters in Cambridge, Massachusetts.
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 December 31, 2016, which is on file with the Securities and Exchange Commission, as well as other risks detailed in Dimension Therapeutics’ additional filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Dimension Therapeutics undertakes no duty to update this information unless required by law.
|DIMENSION THERAPEUTICS, INC.|
|CONSOLIDATED BALANCE SHEETS|
|(In thousands, except share and per share amounts)|
|Cash and cash equivalents||$||30,234||$||127,047|
|Prepaid expenses and other current assets||5,484||2,740|
|Total current assets||85,318||129,930|
|Property and equipment, net||8,402||3,339|
|Deferred offering costs||145||—|
|Liabilities, Convertible Preferred Stock and Stockholders’ Equity|
|Accrued expenses and other current liabilities||7,247||3,715|
|Amounts due to related parties||—||522|
|Total current liabilities||20,639||13,201|
|Deferred revenue, net of current portion||8,663||13,670|
|Notes payable, net of discount and current portion||4,169||759|
|Commitments and contingencies [(Note 15)]|
|Preferred stock, $0.0001 par value; 5,000,000 shares authorized at|
|December 31, 2016 and 2015; zero shares issued or outstanding|
|at December 31, 2016 and 2015.||—||—|
|Common stock, $0.0001 par value; 150,000,000 shares authorized as of|
|December 31, 2016 and 2015; 25,043,506 and 25,008,227 shares issued|
|and outstanding as of December 31, 2016 and 2015, respectively.||2||2|
|Additional paid-in capital||160,185||156,775|
|Accumulated other comprehensive loss||(51||)||—|
|Total stockholders’ equity||59,941||105,583|
|Total liabilities and stockholders’ equity||$||93,865||$||133,269|
|DIMENSION THERAPEUTICS, INC.|
|CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS|
|(In thousands, except share and per share amounts)|
|Year Ended December 31,|
|Research and development||47,741||33,992||12,974|
|General and administrative||12,780||8,723||2,727|
|Total operating expenses||60,521||42,715||15,701|
|Loss from operations||(49,050||)||(34,965||)||(12,951||)|
|Interest income (expense), net||49||(91||)||(17||)|
|Accretion of convertible preferred stock to redemption value||—||(23||)||(71||)|
|Net loss attributable to common stockholders||$||(49,001||)||$||(35,079||)||$||(13,039||)|
|Net loss per share attributable to common stockholders — basic and diluted||$||(1.97||)||$||(4.41||)||$||(3.61||)|
|Weighted average common shares outstanding — basic and diluted||24,915,887||7,949,670||3,610,592|
|Other comprehensive loss|
|Unrealized loss on marketable securities||(51||)||—||—|
|Total other comprehensive gain (loss)||(51||)||—||—|
|Total comprehensive loss||$||(49,052||)||$||(35,056||)||$||(12,968||)|
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 December 31, 2016.
Contacts: Jean Franchi Chief Financial Officer Dimension Therapeutics 617-714-0709 firstname.lastname@example.org Burns McClellan, on behalf of Dimension Therapeutics Media: Justin Jackson 212-213-0006, ext.327 email@example.comNEXT ARTICLE
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