OREANDA-NEWS. Dimension Therapeutics, Inc. (NASDAQ:DMTX), a biopharmaceutical company advancing novel, adeno-associated virus (AAV) gene therapies for devastating rare diseases associated with the liver, a key organ for human metabolism, today reported financial results for the first quarter ended March 31, 2016, and provided an update on the company’s recent corporate progress.

“We have reinforced our leadership position in gene therapy for liver-associated rare diseases, expanding our inherited metabolic disease portfolio and preparing for two IND filings anticipated in the second half of this year,” said Dr. Annalisa Jenkins, MBBS, FRCP, Chief Executive Officer of Dimension. “This is in addition to our ongoing clinical program in hemophilia B, which is expected to report initial data later this year. We eagerly anticipate these and other key milestones and are committed to transforming medicine and the treatment of patients with devastating genetic diseases.”

Recent Highlights and Upcoming Milestones

- Inherited Metabolic Disease (IMD) Programs -

  • Expanded IMD portfolio through comprehensive research collaboration and license agreement with the University of Pennsylvania (PENN), signed earlier this month. Exclusive programs under the collaboration will address citrullinemia type 1, phenylketonuria (PKU), and Wilson disease.
    • PENN will be responsible for all preclinical translational research to support candidate selection and IND filings in each disease area.
    • Dimension will hold the IND and be responsible for the manufacture of vectors for IND-enabling studies, advancement into clinical testing, regulatory strategy, and commercialization. Dimension has exclusive worldwide rights to REGENXBIO’s proprietary NAV® Technology Platform in each disease area.
  • Further advanced lead IMD programs, DTX301 for OTC deficiency and DTX401 for GSDIa. Recent highlights and upcoming milestones include:
    • DTX301: Received US FDA orphan drug designation for OTC deficiency (January 2016) and positive opinion from European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) recommending orphan medicinal product designation (March 2016).
      • IND filing and initiation of patient dosing anticipated in 2H 2016.
    • DTX401: Lead candidate advancing through 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 (NICHD).
      • IND filing anticipated in 2H 2016.

- Hemophilia B & A Programs -

  • 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.
    • Trial commenced January 2016.
    • Received notification (February 2016) on the Clinical Trial Application (CTA) from the Medicines & Healthcare products Regulatory Agency (MHRA) and Bulgarian Drug Agency (BDA), allowing Dimension to proceed with our Phase 1/2 clinical trial in the UK and Bulgaria.
      • Initial data from the Phase 1/2 trial expected in the second half of 2016.
  • DTX201 is in development for the treatment of moderate/severe to severe hemophilia A.
    • Currently in IND-enabling studies through collaboration with Bayer.
  • Earlier this month, presentations at the American Society for Gene and Cell Therapy (ASGCT) Annual Meeting highlighted preclinical data for both DTX101 and DTX201.

- Expanded facilities -

  • Occupancy of Woburn, MA, state-of-the-art laboratories and office space in April 2016 on track to be operational by mid-2016.
    • Facilities to produce non-GMP material at larger scale in support of advanced preclinical and IND-enabling activities, enabling seamless technology transfer to CMO partners.

First Quarter 2016 Financial Results

  • Cash Position: Cash and cash equivalents as of March 31, 2016, were $117.0 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 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 quarter ended March 31, 2016, Dimension recognized $2.2 million of revenue associated with our collaboration agreement with Bayer, compared to $1.6 million for the same period in 2015.
  • R&D Expenses: Research and development expenses for the quarter ended March 31, 2016, were approximately $8.8 million, compared to $5.5 million for the same period in 2015. The increase was largely due to expenditures in advancing preclinical development of DTX301 and other programs in the company’s pipeline, DTX101-related expenses as the program transitioned from preclinical studies to clinical studies and R&D personnel costs associated with the growth of the company (including non-cash stock-based compensation). 
  • G&A Expenses: General and administrative expenses were $2.9 million for the quarter ended March 31, 2016, compared to $1.5 million for the same period in 2015.  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: For the quarter ended March 31, 2016, the company reported a net loss of $(9.5) million, or $(0.38) per share, compared to a net loss of $(5.4) million, or $(1.40) per share, for the same period in 2015.

About Dimension Therapeutics, Inc.

Dimension Therapeutics, Inc. (NASDAQ:DMTX) is the 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, GSDIa, citrullinemia type 1, PKU, Wilson disease, 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.

 
DIMENSION THERAPEUTICS, INC.
CONDENSED CONSOLIDATED BALANCE SHEETS
(In thousands, except share and per share amounts)
(Unaudited)
             
    March 31,     December 31,  
    2016     2015  
Assets                
Current assets:                
Cash and cash equivalents   $ 117,027     $ 127,047  
Accounts receivable     1,264       143  
Prepaid expenses and other current assets     2,644       2,740  
Total current assets     120,935       129,930  
Property and equipment, net     6,245       3,339  
Total assets   $ 127,180     $ 133,269  
Liabilities and Stockholders’ Equity                
Current liabilities:                
Accounts payable   $ 2,238     $ 1,555  
Accrued expenses and other current liabilities     5,024       3,715  
Amounts due to related parties     —       522  
Deferred revenue     7,114       6,835  
Notes payable     574       574  
Total current liabilities     14,950       13,201  
Deferred revenue, net of current portion     14,465       13,670  
Notes payable, net of discount and current portion     616       759  
Other liabilities     538       56  
Total liabilities     30,569       27,686  
                 
Stockholders’ equity:                
Preferred stock, $0.0001 par value; 5,000,000 shares authorized at March 31, 2016
  and December 31, 2015; zero shares issued or outstanding at March 31, 2016
  and December 31, 2015.		     —       —  
Common stock, $0.0001 par value; 150,000,000 shares authorized as of
  March 31, 2016 and December 31, 2015; 25,020,727 and 25,008,227 shares issued
  and outstanding as of March 31, 2016 and  December 31, 2015, respectively.		     2       2  
Additional paid-in capital     157,315       156,775  
Accumulated deficit     (60,706 )     (51,194 )
Total stockholders’ equity     96,611       105,583  
Total liabilities and stockholders’ equity   $ 127,180     $ 133,269  
                 
DIMENSION THERAPEUTICS, INC.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(In thousands, except share and per share amounts)
(Unaudited)
     
  Three Months Ended  
  March 31,  
  2016     2015  
Revenue $ 2,206     $ 1,594  
Operating expenses:              
Research and development   8,805       5,506  
General and administrative   2,941       1,453  
Total operating expenses   11,746       6,959  
Loss from operations   (9,540 )     (5,365 )
Interest income (expense), net   28       (24 )
Net loss and comprehensive loss   (9,512 )     (5,389 )
Accretion of convertible preferred stock to redemption value   —       (19 )
Net loss attributable to common stockholders $ (9,512 )   $ (5,408 )
Net loss per share attributable to common stockholders — basic and diluted $ (0.38 )   $ (1.40 )
Weighted average common shares outstanding — basic and diluted   24,851,933       3,853,262