-- Demonstrated successful delivery of MRNA-046, a survivin-specific UsiRNA formulated in our proprietary DiLA2 liposomal delivery technology, via intravenous administration in mouse orthotopic and subcutaneous liver cancer models and a mouse orthotopic bladder cancer model. -- Knockdown ( > 60%) of survivin mRNA in the orthotopic liver cancer model with MRNA-046 was associated with a significant (~65%) decrease in tumor weight at study termination; this decrease was comparable to tumor weight reduction with Avastin® (bevacizumab)-treated mice. -- Knockdown ( > 60%) of survivin mRNA with MRNA-046 in the subcutaneously implanted liver tumor model was likewise associated with a significant (~65%) decrease in tumor weight at study termination. -- MRNA-046 achieved up to 90% knockdown of survivin mRNA with local (intravesical) delivery in a mouse orthotopic bladder cancer model. There was a dose-dependent decrease in bioluminescence of up to 90% at 1 mg/kg in MRNA-046-treated mice, indicating significant reduction in tumor volume. -- Demonstrated potent anti-tumor activity with a UsiRNA targeting PLK1 (Polo-like Kinase 1) in a mouse orthotopic bladder cancer model. Data following local (intravesical) administration of the PLK1 UsiRNA in a DiLA2 liposome formulation demonstrated a dose-dependent decrease in bioluminescence of greater than 90% at a dose of 1 mg/kg, indicating significant reduction in tumor volume. -- Demonstrated tumor reduction when two UsiRNAs, one targeting survivin and the second targeting PLK1, were combined within a single formulation. Both UsiRNAs were encapsulated in a DiLA2 liposomal delivery formulation, and delivered directly to the bladder in an orthotopic bladder cancer model. At an equivalent total dose, tumor bioluminescence with the combination was significantly lower (~30%), when compared to MRNA-046 alone. -- Extended and expanded the collaboration with the Vancouver Prostate Centre's (VPC) world-renowned clinical cancer research laboratory in the area of bladder cancer. The program will continue to evaluate UsiRNAs and DiLA2 delivery technologies in the Centre's well-validated bladder cancer models.Advanced RNAi Drug Discovery Platform:
-- Demonstrated that strategic placement of UNA (unlocked nucleobase analogs) within short-interfering RNAs, termed UsiRNAs, results in greater strand-specific activity and minimizes off-target activity when compared to standard siRNAs. -- Observed significant knockdown of a liver-specific target in non-human primates following a single systemic dose of 0.3 mg/kg. -- Demonstrated exceptional stability of our lead delivery system formulation (DiLA2) over a one year period under a variety of conditions with no observed loss in potency or change in particle characteristics.Advanced R&D Collaboration Opportunities:
-- Announced an early collaborative effort with AstraZeneca Investment (China) Company, Ltd. in March 2010 focused on our proprietary DiLA2 delivery system for systemic delivery in hepatocellular carcinoma (HCC). -- Announced an early collaborative effort in January 2010 with a major international pharmaceutical company to utilize the broad capabilities of our proprietary drug discovery engine for RNAi therapeutics, bringing the total number of early collaborative efforts, including the AstraZeneca effort, to three.Advanced Intellectual Property Portfolio:
-- Received a Notification of Granting Patent Rights in China for the use of nucleic acids, such as an siRNA, for the treatment of cancer. This particular patent describes the modulation of claudins, which are proteins implicated in tumor progression and metastasis. -- Received Notice of Allowance covering methods for the delivery of a broad array of compounds with pharmacological (biological) activity, including siRNAs, using targeting peptides from the Company's proprietary Trp cage Phage Display library, which have preferential binding affinity for lung tissue.Presented at Numerous Scientific and Investor Meetings and Conferences:
-- Presented at Informa Life Science 10th Annual Conference, EuroTIDES, and Innovations in Oncology Drug Discovery, Development and Delivery Webinar Series. -- Presented at BioPartnering Europe, BIOInvestor, BIOEurope, OneMedPlace, BIO CEO & Investor and Roth OC Growth Conference.Conference Call and Webcast Information Management will host a conference call to provide a business update and to review financial results for the quarter and year ended December 31, 2009. The call is scheduled for Tuesday, March 23, at 4:30 pm Eastern Time (1:30 pm Pacific Time). To participate in the live conference call, U.S. residents should dial 800-573-4754 and international callers should dial 617-224-4325. The participant code for the live conference call is 37051807. To access the 24-hour telephone replay, U.S. residents should dial 888-286-8010 and international callers should dial 617-801-6888. The participant code for the replay is 99112425. Alternatively, to access the live audio webcast for this conference call, please go to MDRNA's Web site at http://www.mdrnainc.com approximately 15 minutes prior to the conference call in order to register and download any necessary software. A replay of the webcast will be available for 30 days following the event. About MDRNA, Inc. MDRNA is a biotechnology company focused on the development and commercialization of therapeutic products based on RNA interference (RNAi). Our goal is to improve human health through the development of RNAi-based compounds and drug delivery technologies that together provide superior therapeutic options for patients. Over the past decade, we have developed substantial capabilities in molecular biology, cellular biology, amino acid chemistry, peptide chemistry, pharmacology and bioinformatics, which we are applying to a wide range of RNAi technologies and delivery approaches. These capabilities plus the in-licensing of key RNAi-related intellectual property have rapidly enabled us to become a leading RNAi-based therapeutics company with a pre-clinical pipeline in oncology. Through our capabilities, expertise and know-how, we are incorporating multiple RNAi technologies as well as peptide- and liposome-based delivery approaches into a single integrated drug discovery platform that will be the engine for our clinical pipeline as well as a versatile platform for establishing broad therapeutic partnerships with biotechnology and pharmaceutical companies. We are also investing in new technologies that we expect to lead to safer and more effective RNAi-based therapeutics while aggressively building upon our broad and extensive intellectual property estate. By combining broad expertise in siRNA science with proven delivery platforms and a strong IP position, MDRNA is well positioned as a leading RNAi-based drug discovery and development company. Additional information about MDRNA, Inc. is available at http://www.mdrnainc.com. MDRNA Forward-Looking Statements Statements made in this news release may be forward-looking statements within the meaning of Federal Securities laws that are subject to certain risks and uncertainties and involve factors that may cause actual results to differ materially from those projected or suggested. Factors that could cause actual results to differ materially from those in forward-looking statements include, but are not limited to: (i) the ability of MDRNA to obtain additional funding; (ii) the ability of MDRNA to attract and/or maintain manufacturing, research, development and commercialization partners; (iii) the ability of MDRNA and/or a partner to successfully complete product research and development, including preclinical and clinical studies and commercialization; (iv) the ability of MDRNA and/or a partner to obtain required governmental approvals; and (v) the ability of MDRNA and/or a partner to develop and commercialize products that can compete favorably with those of competitors. Additional factors that could cause actual results to differ materially from those projected or suggested in any forward-looking statements are contained in MDRNA's most recent periodic reports on Form 10-K and Form 10-Q that are filed with the Securities and Exchange Commission. MDRNA assumes no obligation to update and supplement forward-looking statements because of subsequent events.
MDRNA, INC. AND SUBSIDIARIES
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(In Thousands, Except Per Share Data)
Three Months Ended Year Ended
December 31, December 31,
2008 2009 2008 2009
--------- --------- --------- ---------
(Unaudited)
Revenue
License and other revenue $ 147 $ 208 $ 1,360 $ 14,643
Product revenue --- --- 972 70
Government grants 40 --- 277 19
--------- --------- --------- ---------
Total revenue 187 208 2,609 14,732
--------- --------- --------- ---------
Operating expenses:
Cost of product revenue --- --- 2,906 ---
Research and development 9,749 3,107 36,771 14,882
Selling, general and
administrative 2,488 2,252 13,617 10,088
Restructuring 173 26 8,257 455
--------- --------- --------- ---------
Total operating expenses 12,410 5,385 61,551 25,425
--------- --------- --------- ---------
Loss from operations (12,223) (5,177) (58,942) (10,693)
Other income (expense):
Interest income 22 1 519 5
Interest expense (100) (227) (797) (538)
Net gain on settlement of
liabilities --- --- --- 654
Change in fair value price
adjustable warrants --- 4,618 --- 2,526
--------- --------- --------- ---------
Net loss $ (12,301) $ (785) $ (59,220) $ (8,046)
========= ========= ========= =========
Basic and diluted net loss per
share:
Net loss per common share --
basic and diluted $ (0.39) $ (0.02) $ (2.01) $ (0.21)
========= ========= ========= =========
Shares used in computing net
loss per share -- basic and
diluted 31,147 40,783 29,529 37,457
========= ========= ========= =========
December 31, December 31,
Selected Balance Sheet Data (In Thousands) 2008 2009
----------- -----------
(Unaudited) (Unaudited)
Cash and Cash Equivalents
(includes restricted cash of $2,268 and $998,
respectively) $ 3,352 $ 1,746
Accounts Receivable, net 32 211
Property and Equipment, Inventories and Other
Assets 9,753 5,272
----------- -----------
Total Assets 13,137 7,229
=========== ===========
Fair Value Liability for Price Adjustable
Warrants --- 7,243
Other Liabilities 16,396 6,872
----------- -----------
Total Liabilities 16,396 14,115
=========== ===========
Accumulated Deficit (254,085) (263,017)
=========== ===========
Contact Information: Contacts: MDRNA, Inc.: Peter Garcia Chief Financial Officer (425) 908-3603 pgarcia@mdrnainc.com Westwicke Partners (Investors): Stefan Loren, Ph.D., (443) 213-0507 sloren@westwicke.com John Woolford, (443) 213-0506 john.woolford@westwicke.com McKinney|Chicago (Media): Alan Zachary, (312) 944-6784 x 316 or (708) 707-6834 azachary@mckinneychicago.com