-Management to Host Conference Call Today at
"We continue to advance our pipeline of rare disease and oncology
development programs. During the third quarter we presented new
preclinical data showing advances in delivery potency with DsiRNA-EX
Conjugates that we believe puts us at the cutting edge of RNAi delivery
to the liver," 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 investigational 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. These extensions are unique to Dicerna technology, enabling a differentiated and independent approach to subcutaneous delivery of RNAi-inducing therapeutics.
Rare Disease Program Update
DCR-PH1: DCR-PH1 is a DsiRNA-EX-based therapeutic candidate for Primary Hyperoxaluria Type 1 (PH1), a severe, rare genetic disease of liver metabolism that often results in life-threatening damage to the kidneys. In a genetic mouse model of PH1, DCR-PH1 knocked down the activity of the HAO1 gene transcript that encodes for the enzyme glycolate oxidase, 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 non-human primate studies, a single dose of DCR-PH1 can lead to an average of 84% knockdown of the HAO1 gene transcript with up to 93% knockdown observed.
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, seeks to avoid these difficulties. This novel approach has yielded encouraging results in pre-clinical studies, where DCR-MYC knocked down MYC transcript activity and significantly reduced 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 expects that it has sufficient cash to fund operations for at least the next twelve months. 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 of investigational treatments 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, including, for
example, our expected timeline of development and licensing plans. 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 clinical and preclinical research and other risks
identified under the heading "Risk Factors" included in our most recent
Form 10-Q filing and in other future filings with the
|Consolidated Balance Sheets (unaudited)|
|Cash and cash equivalents||$||52,943||$||26,067|
|Total stockholders' equity||
|Consolidated Statements of Operations (unaudited)|
|(In thousands, except share and per share data)|
|For the Three Months Ended||For the Nine Months Ended|
|Research and development||12,142||7,489||32,708||19,546|
|General and administrative||4,857||3,725||14,822||10,938|
|Total operating expenses||16,999||11,214||47,530||30,484|
|Loss from operations||(16,999||)||(11,214||)||(47,346||)||(30,484||)|
|Preferred stock warrant remeasurement||-||-||-||(2,559||)|
|Other income (expense), net||55||21||142||(306||)|
|Less: Accretion and dividends on redeemable convertible preferred stock||-||-||-||204|
|Net loss attributable to common stockholders||(16,944||)||(11,193||)||(47,204||)||(33,553||)|
|Net loss per share allocable to common stockholders - basic and diluted||$||(0.82||)||$||(0.63||)||$||(2.47||)||$||(2.17||)|
|Weighted average shares outstanding - basic and diluted||20,592,840||17,706,645||19,097,230||15,438,699|
|GAAP to Non-GAAP Reconciliation: Net Loss and Net Loss Per Share|
|(unaudited, in thousands, except per share amounts)|
|For the Three Months||For the Nine Months|
|NET LOSS PER SHARE|
|GAAP net loss per share attributable to common stockholders - basic and diluted||$||(0.82||)||$||(0.63||)||$||(2.47||)||$||(2.17||)|
|Adjustments to net loss (as detailed below)||0.11||0.12||0.38||0.61|
|Non-GAAP loss per share - basic and diluted||$||(0.71||)||$||(0.51||)||$||(2.09||)||$||(1.56||)|
|An itemized reconciliation between net loss on a GAAP basis and net loss on a non-GAAP basis is as follows:|
|GAAP net loss per share attributable to common stockholders||$||(16,944||)||$||(11,193||)||$||(47,204||)||$||(33,553||)|
|Accretion and dividends on redeemable convertible preferred stock||-||-||-||204|
|Preferred stock warrant remeasurement||-||-||-||2,559|
|Milestone Payment on License Agreement||-||500||-||500|
|R&D: Stock-based compensation||871||726||3,038||3,338|
|G&A: Stock-based compensation||1,522||881||4,336||2,886|
|Non-GAAP net loss||$||(14,551||)||$||(9,086||)||$||(39,830||)||$||(24,066||)|
|Weighted Average Shares Outstanding -basic and diluted||20,592,840||17,706,645||19,097,230||15,438,699|
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
Our "Non-GAAP net loss" and "Non-GAAP net loss per share - basic and diluted" financial measures exclude the following items from GAAP net loss and net loss per share:
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 Arbutus for the license to their LNP delivery technology for use in our PH1 development program better reflects the recurring nature of our business. Based on our current drug development program 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.
News Provided by Acquire Media