- Treated first patients in phase I/II clinical study of DTX101 for hemophilia B -
- Expect to file an IND and initiate dosing for DTX301 for OTC deficiency in second half 2016 -
- Selected candidate for GSDIa and expect to file an IND in second half 2016 -
- Three new inherited metabolic disease (IMD) programs expected to be named this year -
CAMBRIDGE, Mass., March 24, 2016 (GLOBE NEWSWIRE) -- Dimension Therapeutics, Inc. (NASDAQ:DMTX), a biopharmaceutical company advancing novel, adeno-associated virus (AAV) gene therapies targeting the liver, a central organ for devastating rare diseases, today reported financial results for the full year ended December 31, 2015 and provided an update on the company’s recent corporate progress.
“2015 was a remarkable year for Dimension, during which we initiated our first clinical trial for our lead candidate for hemophilia B and further progressed key inherited metabolic disease programs in our product pipeline,” said Annalisa Jenkins, MBBS, MRCP, Chief Executive Officer of Dimension. “In the second half of this year, we expect to submit investigational new drug (IND) applications for the most advanced AAV gene therapies for the treatment of ornithine transcarbamylase (OTC) deficiency and GSDIa. We are proud to be translating leading science into potential new options for patients who are living everyday with the devastating consequences of their genetic disease.”
Recent Highlights and Upcoming Milestones
- Initiated multi-center Phase 1/2 study for DTX101, Dimension’s lead product candidate designed to deliver Factor IX, or FIX, gene expression, in adult patients with moderate/severe to severe hemophilia B; first dosing of patients in January 2016. Recent highlights and upcoming milestones include:
- Cohort data from the Phase 1/2 trial expected in the second half of 2016.
- In February 2016, received notification on the Clinical Trial Application (CTA) from the Medicines & Healthcare products Regulatory Agency (MHRA), allowing Dimension to proceed with our Phase 1/2 clinical trial in the UK.
- Further advanced the company’s product development pipeline, including product candidates focused on inherited metabolic diseases (IMD) – DTX301 for OTC deficiency and DTX401 for GSDIa – and DTX201 for hemophilia A via Dimension’s collaboration with Bayer. Recent highlights and upcoming milestones include:
- DTX301 for OTC deficiency: IND filing and initiation of patient dosing anticipated in 2H 2016.
- DTX401 for GSDIa: Selected a candidate in late 2015 via our cooperative research and development agreement, or CRADA, with Janice Chou, Ph.D. and the Eunice Kennedy Shriver National Institute of Child Health and Human Development, or NICHD, an institute of the U.S. National Institutes of Health; anticipate an IND filing in 2H 2016.
- DTX201 for hemophilia A: Selected a candidate in 2015 and expect IND-enabling studies in 2016.
- Further, Dimension expects to name three additional IMD programs this year.
- Received orphan drug designation for DTX301
- Announced in January 2016 that the U.S. Food and Drug Administration (FDA) granted orphan drug designation for DTX301 for the treatment of OTC deficiency.
- Announced in March 2016 that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion recommending DTX301 for designation as an orphan medicinal product for the treatment of OTC deficiency.
- Opening of Woburn, MA, facility on track and anticipated occupancy for April 2016, operational by mid-2016; includes office space and state-of-the-art laboratories to produce non-GMP material in support of advanced preclinical and IND-enabling activities.
Full Year 2015 Financial Results
- Cash Position: Cash and cash equivalents as of December 31, 2015 were $127.0 million, compared with $17.9 million on December 31, 2014. Cash and cash equivalents as of December 31, 2015 included total net proceeds of $64.6 million from the company’s initial public offering of common stock in October 2015 and the underwriters’ exercise of their over-allotment option in November 2015. Based on its current operating plan to fund seven programs, the company expects its existing cash and cash equivalents, payments received in connection with its collaboration agreement with Bayer and borrowing capacity under its loan and security agreement with Silicon Valley Bank will enable it to fund its operating expenses and capital expenditure requirements through Q4 2017.
- Revenue: For the year ended December 31, 2015, Dimension recognized $7.8 million of revenue associated with our collaboration agreement with Bayer, compared to $2.8 million for the same period in 2014.
- R&D Expenses: Research and development expenses for the year ended December 31, 2015, were approximately $34.0 million, compared to $13.0 million for the same period in 2014. The increase was largely due to expenditures in advancing preclinical development of the company’s pipeline, preparing for the initiation of the company’s DTX101 clinical trial, licensing fees from our agreements with REGENXBIO, personnel costs associated with the growth of the company (including non-cash stock-based compensation).
- G&A Expenses: General and administrative expenses were $8.7 million for the year ended December 31, 2015, compared to $2.7 million for the same period in 2014. The increase was largely due to expenditures in personnel associated with the growth of the company (including non-cash stock-based compensation), increased professional fees associated with the costs of operating as a public company and costs related to general operations.
- Net Loss: The Company reported a net loss of approximately $(35.1) million for the year ended December 31, 2015, compared to a net loss of $(13.0) million for the year ended December 31, 2014.
About Dimension Therapeutics
Dimension Therapeutics, Inc. (NASDAQ:DMTX) is a leader in discovering and developing new therapeutic products for people living with devastating rare 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 and GSDIa, a collaboration with Bayer in hemophilia A, and a wholly owned clinical program in hemophilia B. 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 progress of Dimension's portfolio and lead programs, the build-out and occupation of Dimension's new facilities, and the regulatory progress of Dimension's product candidates and programs. 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 lead candidate, DTX101, will not successfully be developed or commercialized; and the risks described under the caption "Risk Factors" in Dimension Therapeutics’ Annual Report on Form 10-K for the year ended December 31, 2015, 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)|
|December 31,||December 31,|
|Cash and cash equivalents||$||127,047||$||17,913|
|Prepaid expenses and other current assets||2,740||404|
|Total current assets||129,930||20,291|
|Property and equipment, net||3,339||1,780|
|Liabilities, Convertible Preferred Stock and Stockholders’ Equity (Deficit)|
|Accrued expenses and other current liabilities||3,715||1,080|
|Amounts due to related parties||522||750|
|Total current liabilities||13,201||7,992|
|Deferred revenue, net of current portion||13,670||17,513|
|Notes payable, net of discount and current portion||759||1,166|
|Commitments and contingencies (Note 14)|
|Convertible preferred stock (Series A and B), $0.0001 par value; zero shares and|
45,000,000 shares authorized as of December 31, 2015 and 2014, respectively;
zero shares and 10,000,000 shares issued and outstanding as of December 31, 2015
and 2014, respectively; aggregate liquidation preference of $0 and $10,000
as of December 31, 2015 and 2014, respectively.
|Stockholders’ equity (deficit):|
|Preferred stock, $0.0001 par value; 5,000,000 and zero shares authorized at|
December 31, 2015 and 2014, respectively; zero shares issued or outstanding
at December 31, 2015 and 2014, respectively
|Common stock, $0.0001 par value; 150,000,000 and 65,700,000 shares authorized|
as of December 31, 2015 and 2014 respectively; 25,008,227 and 4,056,818
shares issued and outstanding as of December 31, 2015 and 2014, respectively.
|Additional paid-in capital||156,775||1,947|
|Total stockholders’ equity (deficit)||105,583||(14,191||)|
|Total liabilities, convertible preferred stock and stockholders’ equity (deficit)||$||133,269||$||22,133|
|DIMENSION THERAPEUTICS, INC.|
|CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS|
|(In thousands, except share and per share amounts)|
|Year Ended December 31,|
|Research and development||33,992||12,974|
|General and administrative||8,723||2,727|
|Total operating expenses||42,715||15,701|
|Loss from operations||(34,965||)||(12,951||)|
|Interest income (expense), net||(91||)||(17||)|
|Net loss and comprehensive loss||(35,056||)||(12,968||)|
|Accretion of convertible preferred stock to redemption value||(23||)||(71||)|
|Net loss attributable to common stockholders||$||(35,079||)||$||(13,039||)|
|Net loss per share attributable to common stockholders — basic and diluted||$||(4.41||)||$||(3.61||)|
|Weighted average common shares outstanding — basic and diluted||7,949,670||3,610,592|
Chief Financial Officer
Burns McClellan, on behalf of Dimension Therapeutics
Media: Justin Jackson