- Advanced lead IMD candidates - DTX301 for OTC deficiency and DTX401 for GSDIa, DTX601 for citrullinemia type I, DTX501 for PKU, and DTX701 for Wilson disease :
- DTX301: Completed IND-enabling studies; IND filing and initiation of the global, multi-center Phase 1/2 clinical trial anticipated by the end of 2016.
- DTX401: Lead candidate advancing in IND-enabling studies.
- Granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for DTX401 for the treatment of GSDIa.
- DTX601, DTX501, DTX701: Ongoing preclinical translational research activities with the University of Pennsylvania (PENN) to support selection of development candidates for citrullinemia type 1, phenylketonuria (PKU), and Wilson disease.
- DTX101: Conducting multi-center Phase 1/2 study for DTX101 , Dimension's lead product candidate for adult patients with moderate/severe to severe hemophilia B.
- Completed cohort 1 and dosing cohort 2; expect to disclose clinical data in January 2017.
- In October, the Data Safety Monitoring Committee (DSMC) accepted Dimension's recommendation to dose escalate to 5x10 12 GC/kg for the second cohort.
- DTX201: Currently in IND-enabling studies in collaboration with Bayer for treatment of moderate/severe to severe hemophilia A.
- Presented 3 posters highlighting advances in mammalian AAV manufacturing technology during the European Society of Gene and Cell Therapy (ESGCT) congress.
- Dimension researchers reported on new technologies to support current and next generation HELA 2.0 AAV manufacturing platform.
- Cash Position: Cash, cash equivalents and marketable securities as of September 30, 2016, were $93.8 million, compared with $127.0 million on December 31, 2015. Based on its current operating plan to fund seven programs, the company expects its existing cash, cash equivalents and marketable securities, 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 Q1 2018.
- Revenue: For the quarter ended September 30, 2016, Dimension recognized $2.8 million of revenue associated with its collaboration agreement with Bayer, compared to $2.1 million for the same period in 2015. The increase was due to services performed in connection with its performance obligations under the collaboration agreement with Bayer.
- R&D Expenses: Research and development expenses for the quarter ended September 30, 2016, were approximately $13.8 million, compared to $10.4 million for the same period in 2015. The increase was primarily due to increased expenditures in preparation of an IND filing and Phase 1/2 clinical for DTX301, manufacturing and IND-enabling studies for DTX401, and manufacturing process development activity in Dimension's Woburn facility, offset primarily by decreased expenditures with DTX101 as we conduct our Phase 1/2 study for DTX101.
- G&A Expenses: General and administrative expenses were $3.3 million for the quarter ended September 30, 2016, compared to $2.3 million for the same period in 2015. The increase was primarily due to increased personnel-related costs (including stock-based compensation), as a result of hiring additional full-time and contractor employees to support growth in operations, finance and human resources.
- Net Loss: For the quarter ended September 30, 2016, the company reported a net loss of $(14.4) million, or $(0.58) per share, compared to a net loss of $(10.7) million, or $(2.73) per share, for the same period in 2015.
For more information, please visit http://www.dimensiontx.com.Cautionary Note Regarding Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the clinical and developmental progress of Dimension's portfolio and lead programs, including upcoming milestones, the potential productivity of Dimension's ongoing collaborations, and the regulatory progress of Dimension's product candidates and programs. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include the risks that Dimension's product candidates, including its lead candidate, DTX101, will not successfully be developed or commercialized; and the risks described under the caption "Risk Factors" in Dimension Therapeutics' Quarterly Report on Form 10-Q for the quarter ended September 30, 2016, which is on file with the Securities and Exchange Commission, as well as other risks detailed in Dimension Therapeutics' additional filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Dimension Therapeutics undertakes no duty to update this information unless required by law. DIMENSION THERAPEUTICS, INC. CONDENSED CONSOLIDATED BALANCE SHEETS (In thousands, except share and per share amounts) (Unaudited)
|September 30,||December 31,|
|Cash and cash equivalents||$||74,943||$||127,047|
|Prepaid expenses and other current assets||2,900||2,740|
|Total current assets||98,459||129,930|
|Property and equipment, net||8,718||3,339|
|Liabilities and Stockholders' Equity|
|Accrued expenses and other current liabilities||6,968||3,715|
|Amounts due to related parties||—||522|
|Total current liabilities||18,125||13,201|
|Deferred revenue, net of current portion||11,770||13,670|
|Notes payable, net of discount and current portion||4,751||759|
|Commitments and contingencies (Note 10)|
|Preferred stock, $0.0001 par value; 5,000,000 shares authorized at September 30, 2016 and December 31, 2015; zero shares issued or outstanding at September 30, 2016 and December 31, 2015.||—||—|
|Common stock, $0.0001 par value; 150,000,000 shares authorized as of September 30, 2016 and December 31, 2015; 25,043,506 and 25,008,227 shares issued and outstanding as of September 30, 2016 and December 31, 2015, respectively.||2||2|
|Additional paid-in capital||159,207||156,775|
|Accumulated other comprehensive loss||(1||)||—|
|Total stockholders' equity||72,038||105,583|
|Total liabilities and stockholders' equity||$||107,177||$||133,269|
|Three Months Ended||Nine Months Ended|
|September 30,||September 30,|
|Research and development||13,796||10,372||33,841||25,238|
|General and administrative||3,305||2,334||9,482||6,115|
|Total operating expenses||17,101||12,706||43,323||31,353|
|Loss from operations||(14,339||)||(10,645||)||(35,984||)||(25,956||)|
|Interest income (expense), net||(26||)||(22||)||8||(72||)|
|Accretion of convertible preferred stock to redemption value||—||—||—||(23||)|
|Net loss attributable to common stockholders||$||(14,365||)||$||(10,667||)||$||(35,976||)||$||(26,051||)|
|Net loss per share attributable to common stockholders — basic and diluted||$||(0.58||)||$||(2.73||)||$||(1.44||)||$||(6.71||)|
|Weighted average common shares outstanding — basic and diluted||24,930,348||3,907,196||24,900,773||3,880,779|
|Other comprehensive gain (loss):|
|Unrealized gain (loss) on marketable securities||(1||)||—||(1||)||—|
|Total other comprehensive gain (loss)||(1||)||—||(1||)||—|
|Total comprehensive loss||$||(14,366||)||$||(10,667||)||$||(35,977||)||$||(26,028||)|
CONTACT:Jean FranchiChief Financial OfficerDimension Therapeutics617firstname.lastname@example.orgBurns McClellan, on behalf of Dimension TherapeuticsMedia: Justin Jackson212-213-0006, email@example.com