-Management to Host Conference Call Today at
"We continue to advance our pipeline of rare disease and oncology
programs, most recently with the announcement of robust non-human
primate data using our conjugate platform for subcutaneous delivery of
RNAi therapeutics," stated
Subcutaneous delivery to the liver with DsiRNA-EX Conjugates: DsiRNA-EX Conjugate technology is a proprietary delivery platform that is designed to enable convenient subcutaneous delivery for Dicerna's emerging pipeline of liver-targeted RNAi therapies. These conjugates do not involve lipid nanoparticles and are built on the DsiRNA-EX platform, using an extension to one end of the double-stranded DsiRNA molecule.
Rare Disease Program Update
DCR-PH1: DCR-PH1 is a DsiRNA-EX-based therapeutic candidate for Primary Hyperoxaluria Type 1 (PH1), a rare, inherited disorder of liver metabolism that often results in life-threatening damage to the kidneys. In a genetic mouse model of PH1, Dicerna has shown that DCR-PH1 knocks down the activity of the gene transcript that encodes for the enzyme glycolate oxidase (GO), thereby significantly reducing the production of oxalate, the key disease pathology of PH1. Similar results, if obtained in PH1 patients, may have significant clinical benefit.
In addition to PH1, Dicerna is deploying the DsiRNA-EX Conjugate technology to generate subcutaneously-injectable RNA-based therapeutics against multiple rare disease targets expressed in the liver.
Oncology Program Update
DCR-MYC: DCR-MYC is a potent and specific inhibitor of MYC, an oncogene frequently amplified or overexpressed in a wide variety of cancer tumor types, including hepatocellular carcinoma (HCC). DCR-MYC is a DsiRNA-based therapeutic formulated in Dicerna's EnCore lipid nanoparticle for delivery to solid tumors. The MYC oncogene encodes a small intracellular protein that lacks a good binding site, which makes it a challenging target for traditional pharmaceutical approaches that seek to use small molecules or monoclonal antibodies to inhibit protein activity. Dicerna's RNAi-based approach, which targets gene transcripts before they are translated into proteins, avoids these difficulties. This novel approach has yielded encouraging results in pre-clinical studies, in which Dicerna has shown that DCR-MYC knocks down MYC transcript activity and significantly reduces tumor volume in multiple mouse tumor models, including models of HCC.
Phase 1 DCR-MYC Trials in Solid Tumors and Hepatocellular Carcinoma (HCC)
More detailed financial information and analysis may be found in the
Company's Quarterly Report on Form 10-Q, which was filed with the
Based on our current cash position and operating plan, the Company re-iterates its expectation that it has sufficient cash to fund operations through 2016. This estimate assumes no additional funding from new partnership agreements or debt or equity financing events.
Management will conduct a conference call at
Dicerna is a biopharmaceutical company focused on the discovery and development of innovative treatments for rare, inherited diseases involving the liver and for cancers that are genetically defined. The Company is using its proprietary RNA interference (RNAi) technology platform to build a broad pipeline in these therapeutic areas. In both rare diseases and oncology, Dicerna is pursuing targets that have historically been difficult to inhibit using conventional approaches, but where connections between targets and diseases are well understood and documented. The Company intends to discover, develop and commercialize novel therapeutics either on its own or in collaboration with pharmaceutical partners. For more information, please visit www.dicerna.com.
Cautionary Note on Forward-Looking Statements
This press release includes forward-looking statements. Such
forward-looking statements are subject to risks and uncertainties that
could cause actual results to differ materially from those expressed or
implied in such statements. Applicable risks and uncertainties include
those relating to our preclinical research and other risks identified
under the heading "Risk Factors" included in our most recent Form 10-K
filing and in other future filings with the
Consolidated Balance Sheets (unaudited)
|Cash and cash equivalents||$||22,635||$||26,067|
|Total stockholders' equity||$||86,729||$||98,340|
Consolidated Statements of Operations (unaudited)
(In thousands, except share and per share data)
For the Three Months
|Research and development||$||8,692||$||5,251|
|General and administrative||5,445||2,841|
|Total operating expenses||14,137||8,092|
|Loss from operations||(14,137||)||(8,092||)|
|Preferred stock warrant remeasurement||-||(2,559||)|
|Other income (expense), net||53||(153||)|
Less: Accretion and dividends on
Net loss attributable to common
Net loss per share allocable to common stockholders
Weighted average shares outstanding -
|GAAP to Non-GAAP Reconciliation: Net Loss and Net Loss Per Share|
|(unaudited, in thousands, except share and per share amounts)|
|For the Three Months|
|NET LOSS PER SHARE|
|GAAP net loss per share attributable to common stockholders - basic and diluted||$||(0.79)||$||(1.02)|
|Adjustments to net loss (as detailed below)||0.13||0.46|
|Non-GAAP loss per share - basic and diluted||$||(0.66)||$||(0.56)|
|An itemized reconciliation between net loss on a GAAP basis and net loss on a non-GAAP basis is as follows:|
|GAAP net loss attributable to common stockholders||$||(14,084)||$||(11,008)|
|Accretion and dividends on redeemable convertible preferred stock||-||204|
|Preferred stock warrant remeasurement||-||2,559|
|Milestone Payment on License Agreement||-||-|
|R&D: Stock-based compensation||847||1,726|
|G&A: Stock-based compensation||1,473||486|
|Non-GAAP net loss||$||(11,764)||$||(6,033)|
|Weighted Average Shares Outstanding -basic and diluted||17,815,515||10,822,325|
|Numbers may not foot due to rounding|
Use of Non-GAAP Financial Measures
We supplement our consolidated financial statements presented on a GAAP
basis by providing additional measures which may be considered
"non-GAAP" financial measures under applicable
These non-GAAP financial measures are not in accordance with generally
accepted accounting principles in
1. Stock-based compensation expense recorded in accordance with the accounting standard for share-based payments.
We believe that excluding the accounting impact of share-based payments, for both employees and non-employees, better reflects the recurring economic characteristics of our business. Share-based payments to non-employees are measured at each reporting date and recognized as services are rendered or vesting occurs.
2. Warrant remeasurement in accordance with accounting standards for derivative instruments.
We believe that excluding preferred stock warrant remeasurement better reflects the recurring economics of our business. Upon our IPO, the warrants were reclassified to additional paid-in-capital and are no longer marked to market.
3. Milestone Payment on License Agreement.
We believe that excluding the payment to Tekmira for the license to their LNP delivery technology for use in our DCR-PH1 development program better reflects the recurring nature of our business. Based on our current drug development programs and recent advances in our technology platform, we do not expect to enter into similar licensing arrangements.
4. Other items.
We evaluate other items on an individual basis, and consider both the quantitative and qualitative aspects of the item, including (i) its size and nature, (ii) whether or not it relates to our ongoing business operations, and (iii) whether or not we expect it to occur as part of our normal business on a regular basis.
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