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SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FOR THE QUARTERLY PERIOD ENDED June 30, 2012,
FOR THE TRANSITION PERIOD FROM TO
Commission file number 0-22025
AASTROM BIOSCIENCES, INC.
(Exact name of registrant as specified in its charter)
(Registrants telephone number, including area code)
(Former name, former address and former fiscal year, if changed since last report)
Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes - x No - o
Indicate by check mark whether the registrant has submitted electronically and posted on its corporate Web site, if any, every Interactive Data File required to be submitted and posted pursuant to Rule 405 of Regulation S-T during the preceding 12 months (or for such shorter period that the registrant was required to submit and post such files). Yes - x No - o
Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, or smaller reporting company. See the definitions of large accelerated filer, accelerated filer and smaller reporting company in Rule 12b-2 of the Exchange Act.
Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes - £ No - T
Indicate the number of shares outstanding of each of the issuers classes of common stock as of the latest practicable date.
AASTROM BIOSCIENCES, INC.
QUARTERLY REPORT ON FORM 10-Q
AASTROM BIOSCIENCES, INC.
(a development stage company)
(Unaudited, amounts in thousands)
The accompanying Notes to Condensed Consolidated Financial Statements are an integral part of these statements.
AASTROM BIOSCIENCES, INC.
(a development stage company)
(Unaudited, amounts in thousands except per share amounts)
The accompanying Notes to Condensed Consolidated Financial Statements are an integral part of these statements.
AASTROM BIOSCIENCES, INC.
(a development stage company)
(Unaudited, amounts in thousands)
The accompanying Notes to Condensed Consolidated Financial Statements are an integral part of these statements.
1. Organization and Summary of Significant Accounting Policies
Aastrom Biosciences, Inc. was incorporated in March 1989 (Inception), began employee-based operations in 1991, and is in the development stage. The Company operates its business in one reportable segment research and product development involving the development of patient-specific cell based products for use in regenerative medicine.
Successful future operations are subject to several technical hurdles and risk factors, including satisfactory product development, timely initiation and completion of clinical trials, regulatory approval and market acceptance of the Companys products and the Companys continued ability to obtain future funding.
The Company is subject to certain risks related to the operation of its business and development of its products and product candidates. The Company believes that it will have adequate liquidity to finance its planned operations, including development of its products and product candidates, via cash on hand as of June 30, 2012 of $28,666,000 until at least December 31, 2012. While the Companys budgeted cash usage and operating plan for 2012 does not currently contemplate taking additional actions to reduce the use of cash over the next six months, the Company could, if necessary, delay or forego certain budgeted discretionary expenditures such as anticipated hiring plans or certain non-critical research and development expenditures, as well as slow down or delay certain clinical trial activity (without jeopardizing our Phase 3 clinical trial for CLI). While the Company could sell shares through an At the Market Sales Agreement (ATM) to raise additional capital, there are certain factors, such as volume of trading in our stock and our stock price, which could limit the amount the Company could raise in a short period of time. On a longer-term basis, the Company will need to raise additional funds in order to complete product development programs and complete clinical trials needed to market and commercialize these products. The Company cannot be certain that such funding will be available on favorable terms, if at all. Some of the factors that will impact the Companys ability to raise additional capital and its overall success include: the rate and degree of progress for product development, the rate of regulatory approval to proceed with clinical trial programs, the level of success achieved in clinical trials, the requirements for marketing authorization from regulatory bodies in the United States and other countries, the liquidity and market volatility of the Companys equity securities, regulatory and manufacturing requirements and uncertainties, technological developments by competitors, and other factors. If the Company cannot raise such funds, it may not be able to develop or enhance products, take advantage of future opportunities, or respond to competitive pressures or unanticipated requirements, which would likely have a material adverse impact on the Companys business, financial condition and results of operations. As a result of the need to raise additional capital, there is uncertainty regarding our ability to maintain liquidity sufficient to operate the business effectively over at least the next twelve months, which raises substantial doubt as to our ability to continue as a going concern. The consolidated financial statements do not include any adjustments that might result from the outcome of this uncertainty.
2. Basis of Presentation
The condensed consolidated financial statements included herein have been prepared in accordance with the rules and regulations of the Securities and Exchange Commission (SEC). Certain information and footnote disclosures normally included in financial statements prepared in accordance with generally accepted accounting principles in the United States of America (U.S. GAAP) have been omitted pursuant to such rules and regulations. The financial statements reflect, in the opinion of management, all adjustments (consisting only of normal, recurring adjustments) necessary to state fairly the financial position and results of operations as of and for the periods indicated. The results of operations for the six months ended June 30, 2012, are not necessarily indicative of the results to be expected for the full year or for any other period. The December 31, 2011 condensed consolidated balance sheet data was derived from audited consolidated financial statements, but does not include all disclosures required by U.S. GAAP.
These condensed consolidated financial statements should be read in conjunction with the audited consolidated financial statements and the notes thereto included in our Annual Report on Form 10-K for the period ended December 31, 2011, as filed with the SEC.
The consolidated financial statements include the accounts of Aastrom and its wholly-owned subsidiaries, Aastrom Biosciences GmbH, located in Berlin, Germany and Aastrom Biosciences, SL, located in Barcelona, Spain
(collectively, the Company). All inter-company transactions and accounts have been eliminated in consolidation. The subsidiaries are not a significant component of the consolidated financial statements as each has ceased operations and had limited operations historically.
3. Fair Value Measurements
The Company measures certain assets and liabilities at fair value on a recurring basis. Fair value represents the amount that would be received upon the sale of an asset or paid to transfer a liability in an orderly transaction between market participants. As such, fair value is determined based upon assumptions that market participants would use in pricing an asset or liability. As a basis for considering such assumptions, a three-tier fair value hierarchy, which prioritizes the inputs used in measuring fair value as follows:
· Level 1 inputs: Quoted prices (unadjusted) for identical assets or liabilities in active markets;
· Level 2 inputs: Inputs, other than quoted prices included in Level 1 that are observable either directly or indirectly; and
· Level 3 inputs: Unobservable inputs for which there is little or no market data, which require the reporting entity to develop its own assumptions.
In many cases, a valuation technique used to measure fair value includes inputs from multiple levels of the fair value hierarchy described above. The lowest level of significant input determines the placement of the entire fair value measurement in the hierarchy.
See Note 5 for disclosures related to the fair value of the Companys warrants. The Company does not have any other assets or liabilities on the balance sheet as of June 30, 2012 that are measured at fair value.
4. Stock-Based Compensation
The Company issues nonqualified and incentive stock options as well as other equity awards pursuant to its 2009 Omnibus Incentive Plan, as amended (Option Plan). Such awards pursuant to the Option Plan may be granted by the Companys Board of Directors to certain of the Companys employees, directors and consultants.
During the six months ended June 30, 2012, the Company granted 4,054,445 service-based options to purchase common stock. These options were granted with exercise prices equal to or greater than the fair market value of the Companys stock at the grant date, vest over four years and expire after ten years. The weighted average grant-date fair value of service-based options granted under the Companys Option Plan during the six months ended June 30, 2011 and 2012 was $1.55 and $1.34, respectively.
The net compensation costs recorded for the service-based stock options related to employees and directors were $1,224,000 and $2,039,000 for the quarter and six months ended June 30, 2012, respectively, compared to $1,232,000 and $1,848,000 for the corresponding periods ended June 30, 2011.
The fair value of each service-based stock option grant for the reported periods is estimated on the date of the grant using the Black-Scholes option-pricing model using the weighted average assumptions noted in the following table.
The following table summarizes the activity for service-based stock options for the indicated periods:
As of June 30, 2012 there was $8,308,000 of total unrecognized compensation cost related to non-vested service-based stock options granted under the Option Plan. That cost is expected to be recognized over a weighted-average period of 3.3 years.
The total fair value of options vested during the six months ended June 30, 2011 and 2012 was $924,000 and $1,362,000, respectively.
5. Stock Purchase Warrants
The Company has historically issued warrants to purchase shares of the Companys common stock in connection with certain of its common stock offerings. The following warrants were outstanding at June 30, 2012, and include provisions that could require cash settlement of the warrants or have anti-dilution price protection provisions requiring each to be recorded as liabilities of the Company at the estimated fair value at the date of issuance, with changes in estimated fair value recorded as non-cash income or expense in the Companys statement of operations in each subsequent period:
(i) warrants to purchase an aggregate of 740,131 shares of the Companys common stock, issued on October 17, 2007 in connection with the Companys registered direct offering, exercisable from April 18, 2008 through April 17, 2013 at an exercise price of $12.72 per share, all of which remained outstanding as of June 30, 2012;
(ii) Class A warrants to purchase an aggregate of 4,882,228 shares of the Companys common stock, issued on January 21, 2010 in connection with the Companys registered public offering, exercisable for a five year period commencing on July 21, 2010 at an exercise price of $2.49 per share (as adjusted from $2.52 per share for the anti-dilution provision triggered in the December 2010 warrant exchange outlined below), 4,525,978 of which remained outstanding as of June 30, 2012; and
(iii) warrants to purchase an aggregate of 10,000,000 shares of the Companys common stock, issued on December 15, 2010 in connection with the Companys registered public offering, exercisable for a five year period commencing on December 15, 2010 at an exercise price of $3.22 per share (the December 2010 warrants). On June 27, 2012, the Company announced a common stock exchange for the December 2010 warrants. As part of this effort, the Company entered into separate warrant exchange agreements with certain warrant holders, pursuant to which the Company issued one share of common stock for every two warrants exchanged, issuing 3,833,334 shares of common stock in exchange for 7,666,666 warrants. As of June 30, 2012, 2,333,334 warrants from this issuance remained outstanding and were subject to an offer to exchange at the same terms as described above. As of the expiration of the exchange offer on July 27, 2012, 2,025,234 warrants were exchanged for 1,012,615 shares of common stock, leaving 308,100 December 2010 warrants outstanding.
The fair value of the Class A warrants and the December 2010 warrants are measured using the Monte Carlo valuation model, while the October 2007 warrants are measured using the Black-Scholes valuation model. Both of
the methodologies are based, in part, upon inputs for which there is little or no observable market data, requiring the Company to develop its own assumptions. The assumptions used in calculating the estimated fair value of the warrants represent the Companys best estimates, however these estimates involve inherent uncertainties and the application of management judgment. As a result, if factors change and different assumptions are used, the warrant liabilities and the change in estimated fair value of the warrants could be materially different.
Inherent in both the Monte Carlo and Black-Scholes valuation models are assumptions related to expected stock-price volatility, expected life, risk-free interest rate and dividend yield. The Company estimates the volatility of its common stock based on historical volatility that matches the expected remaining life of the warrants. The risk-free interest rate is based on the U.S. Treasury zero-coupon yield curve on the grant date for a maturity similar to the expected remaining life of the warrants. The expected life of the warrants is assumed to be equivalent to their remaining contractual term. The dividend rate is based on the historical rate, which the Company anticipates to remain at zero.
The Monte Carlo model is used for the Class A warrants and the December 2010 warrants to value the potential future exercise price adjustments triggered by the anti-dilution provisions as well as the value of the put feature of the December 2010 warrants. These both require Level 3 inputs which are based on the Companys estimates of the probability and timing of potential future financings and fundamental transactions. The other assumptions used by the Company are summarized in the following tables:
The following table summarizes the change in the estimated fair value of the Companys warrant liabilities (in thousands):
6. Series B Convertible Preferred Stock
On March 9, 2012, the Company completed the sale of 12,308 shares of Series B-1 Non-Voting Convertible Preferred Stock (Series B-1 Preferred Stock) at an offering price of $3,250 per share. The Company received
$37,620,000 in net proceeds from the sale of the shares of Series B-1 Preferred Stock, after offering expenses. In addition to the Series B-1 Preferred Stock, which was issued at the closing, the Company also authorized Series B-2 Voting Convertible Preferred Stock (Series B-2 Preferred Stock). The Series B-1 Preferred Stock and Series B-2 Preferred Stock collectively are referred to as the Series B Preferred Stock. The Series B-1 Preferred Stock is not entitled to vote on matters on which the common shareholders are generally entitled to vote. The Series B-2 Preferred Stock will be entitled to vote with the holders of the common stock as a single class, with each share of Series B-2 Preferred Stock having the number of votes equal to the number of shares of common stock issuable upon conversion of such Series B-2 Preferred Stock. On May 3, 2012, shareholder approval was obtained in accordance with Nasdaq Marketplace Rule 5635(b), which allowed the holder of Series B-1 Preferred Stock to exchange all of the then outstanding shares for shares of Series B-2 Preferred Stock on a one-for-one basis. The Series B Preferred Stock will, with respect to dividend rights and rights on liquidation, winding-up and dissolution, rank on parity with any other class or series of the Company capital stock that the Company may issue in the future which is designated as being on parity with the Series B Preferred Stock, and rank senior to our common stock and Series A preferred stock. The Series B Preferred Stock is convertible, at the option of the holder thereof at any time after the five year anniversary of the closing of the offering, into shares of our common stock at a conversion price of $3.25 per share of common stock. At any time after the five year anniversary of issuance, the Company may elect to convert any or all outstanding shares of Series B Preferred Stock into shares of our common stock, subject to certain limitations. Dividends on the Series B Preferred Stock will be cumulative and compound daily, at a rate of 11.5% per annum, payable upon conversion, liquidation, redemption or other similar events, and payable in cash or Series B-1 Preferred Stock until the five year anniversary of issuance. Following the five year anniversary of issuance and until the earlier of the tenth anniversary of the issuance and the date no Series B Preferred Stock remain outstanding, dividends will accrue at a rate of 8% per annum and will be payable in cash or Series B-1 Preferred Stock, at our option. Unless prohibited by Michigan law governing distributions to shareholders, the Series B-1 Preferred Stock shall be redeemable at the option of holder of the Series B-1 Preferred Stock commencing at any time after the five year anniversary of issuance, liquidation, winding up, dissolution or other similar events, subject to certain terms and limitations.
The Series B Preferred Stock does not, in its entirety, require liability classification and has been evaluated for embedded features to determine if those features require bifurcation and separate classification as derivative liabilities. The Series B Preferred Stock host contract was evaluated for equity or mezzanine classification based upon the nature of the redemption and conversion features. Generally, any feature that could require cash redemption for matters not within the Companys control, irrespective of probability of the event occurring, requires classification outside of stockholders equity. The Series B Preferred Stock has been recorded in the mezzanine section and will be accreted to its redemption value through charges to stockholders equity (deficit) using the effective interest method.
The carrying value of the Series B-1 Preferred Stock presented as mezzanine equity in the consolidated financial statements is $1,450,000, as of June 30, 2012. As of June 30, 2012, there are no outstanding shares of Series B-1 Preferred Stock; however, as the Series B-1 Preferred Stock dividends can be redeemed for cash, the Company accretes the carrying value of the Series B-1 Preferred Stock to its redemption value of $31,102,000 by charges to accumulated deficit using the effective interest method. Such accretion for the six months ended June 30, 2012 amounted to $1,450,000. The redemption value is determined by the greater of the conversion price of the Series B-1 Preferred Stock or the common stock price, multiplied by the total accumulated dividends at the five year anniversary of issuance.
7. Net Loss Per Common Share
Basic earnings (loss) per share is calculated using the two-class method, which is an earnings allocation formula that determines earnings (loss) per share for the holders of the Companys common shares and holders of the Series B Preferred Stock. The Series B Preferred Stock shares contain participation rights in undistributed earnings, but do not share in the losses of the Company. Therefore, in the event of a loss from continuing operations, the Series B Preferred Stock is not considered in the calculation of basic loss per share.
Net loss per common share is computed using the weighted-average number of common shares outstanding during the period. Common equivalent shares are not included in the diluted per share calculation where the effect of their inclusion would be anti-dilutive. The aggregate number of common equivalent shares (related to options,
warrants and preferred stock) that have been excluded from the computations of diluted net loss per common share at June 30, 2011 and 2012 were 23,210,000 and 31,355,000, respectively.
We were incorporated in 1989 and are a regenerative medicine company focused on the development of innovative cell therapies to repair or regenerate damaged or diseased tissues. We are developing patient-specific, expanded multicellular therapies for use in the treatment of severe, chronic ischemic cardiovascular diseases. We believe ixmyelocel-T (the generic name for our product approved by the U.S. Food and Drug Administration (FDA) and United States Adopted Names (USAN) Council in March 2011 for our multicellular therapy) is a disease modifying therapy with multi-functional properties including: tissue remodeling, immuno-modulation and the promotion of angiogenesis. Our proprietary cell-manufacturing technology enables the manufacture of multicellular therapies, expanded from an adult patients own bone marrow, and delivered directly to damaged tissues. Preclinical and clinical data suggest that ixmyelocel-T may be effective in treating patients with severe, chronic ischemic cardiovascular diseases such as critical limb ischemia (CLI), the most severe form of peripheral arterial disease (PAD). Preliminary data utilizing ixmyelocel-T in dilated cardiomyopathy (DCM) have provided indications of both efficacy and safety. Nearly 200 patients have been treated in recent clinical trials using ixmyelocel-T (over 400 patients safely treated since our inception). In November 2011, we released positive Phase 2b data from our RESTORE-CLI clinical trial and launched our pivotal Phase 3 REVIVE trial in CLI in February 2012. We also plan to start a randomized, placebo-controlled, double-blinded Phase 2b trial in DCM in the second half of 2012.
Ixmyelocel-T is a patient-specific, expanded multicellular therapy developed using our proprietary, automated processing system. Ixmyelocel-T is a product derived from an adult patients own bone marrow but it is significantly enhanced compared with the original bone marrow. Our process enhances the patients bone marrow mononuclear cells by expanding the mesenchymal stromal cells and M2 anti-inflammatory macrophages while retaining many of the hematopoietic cells. The manufacture of our patient-specific, expanded multicellular therapies is done under current Good Manufacturing Practices (cGMP) and current Good Tissue Practices (cGTP) guidelines required by the FDA.
Our therapy has several features that we believe are primarily responsible for success in treating adult patients with severe, chronic cardiovascular diseases:
Patient-specific (autologous) we start with the patients own cells, which are accepted by the patients immune system allowing the cells to integrate into existing functional tissues. This characteristic of our therapy, we believe, eliminates both the risk of rejection and of having to use immunosuppressive therapy pre- or post-therapy. Our data also suggests that ixmyelocel-T provides the potential for long-term engraftment and tissue repair.
Expanded we begin with a small amount of bone marrow from the patient (up to 60 ml) and significantly expand the number of certain cell types, primarily CD90+ (mesenchymal stromal cells or MSCs) and CD14+autofluorescent+ (M2 anti-inflammatory macrophages) to far more than are present in the patients own bone marrow (up to 200 times the number of certain cell types compared with the starting bone marrow). Ixmyelocel-T is derived from the patients own bone marrow but it is significantly enhanced compared with the starting bone marrow.
Multicellular we believe the multiple cell types in ixmyelocel-T, which are normally found in bone marrow but in smaller quantities, possess the key functions required for reducing chronic inflammation, immuno-modulation, and the promotion of angiogenesis.
Minimally invasive our procedure for taking bone marrow (an aspirate) can be performed in an out-patient setting and takes approximately 15 minutes. For diseases such as CLI, the administration of ixmyelocel-T
is performed in an out-patient setting (e.g. a physicians office) in a one-time, approximately 20 minute procedure.
Safe bone marrow and bone marrow-derived therapies have been used safely and efficaciously in medicine for over three decades. Our product, ixmyelocel-T, a bone marrow-derived, patient-specific, expanded multicellular therapy leverages this body of scientific study and medical experience and appears well tolerated in patients treated to date.
Our therapy is produced at our cell manufacturing facility in the United States, located at our headquarters in Ann Arbor, Michigan.
Clinical Development Programs
Our clinical development programs are focused on addressing areas of high unmet medical needs in severe, chronic ischemic cardiovascular diseases. We have completed a successful Phase 2b clinical trial in CLI. We have reached agreement with the FDA on CMC which has allowed us to launch our pivotal Phase 3 REVIVE clinical trial in the first quarter of 2012 with a protocol approved by the FDA through the Special Protocol Assessment (SPA) process. Our CLI development program has also received Fast Track Designation from the FDA. We have completed our Phase 1/2 clinical trials in DCM and plan to begin a randomized, placebo-controlled, double-blinded Phase 2b trial in the second half of 2012. Our DCM development program has received Orphan Disease Designation from the FDA.
Results to date in our clinical trials may not be indicative of results obtained from subsequent patients enrolled in those trials or from future clinical trials. Further, our future clinical trials may not be successful or we may not be able to obtain the required Biologic License Application (BLA) approval to commercialize our products in the United States in a timely fashion, or at all. See Risk Factors in Item 1A of our Annual Report on Form 10-K for the fiscal year ended December 31, 2011.
Critical Limb Ischemia
CLI is the most serious and advanced stage of peripheral arterial disease (PAD) resulting from chronic inflammation and lipid accumulation. PAD is a chronic atherosclerotic disease that progressively restricts blood flow in the limbs and can lead to serious medical complications. This disease is often associated with other serious clinical conditions including hypertension, cardiovascular disease, dyslipidemia, diabetes, obesity and stroke. CLI is used to describe patients with the most severe form of PAD: those with chronic ischemia-induced pain (even at rest) or tissue loss (ulcers or gangrene) in the limbs, often leading to amputation and death. Many CLI patients are considered no option patients as they have exhausted all other treatment options with the exception of amputation. The one-year and four-year mortality rates for no option CLI patients that progress to amputation are approximately 25% and 70%, respectively. Ixmyelocel-T, our disease modifying therapy with multiple functions, has shown significant promise in the treatment of CLI patients with existing tissue loss and no option for revascularization. Currently, there are an estimated 250,000 no option CLI patients in the U.S.
Phase 2b Clinical Program RESTORE CLI
Our U.S. Phase 2b RESTORE-CLI program was a multi-center, randomized, double-blind, placebo-controlled clinical trial. This clinical trial was designed to evaluate the safety and efficacy of ixmyelocel-T in the treatment of patients with CLI and no option for revascularization. It was the largest multi-center, randomized, double-blind, placebo-controlled cellular therapy study ever conducted in no option CLI patients. We completed enrollment of this trial in February 2010 with a total of 86 patients at 18 sites across the United States, with the last patient treated in March 2010. These patients were followed for a period of 12 months after treatment. In addition to assessing the safety of our product, efficacy endpoints included time to first occurrence of treatment failure the trials primary efficacy end-point (defined as major amputation, all-cause mortality, doubling in wound surface area and de novo gangrene), amputation-free survival (defined as major amputation and all-cause mortality), major amputation rates, level of amputation, wound healing, patient quality of life and pain scores. The primary purpose of the trial was to assess performance of our therapy and, if positive, prepare for a Phase 3 program.
Final results of the Phase 2b RESTORE-CLI clinical trial were presented at the American Heart Association Scientific Sessions in November 2011 and published in the peer-reviewed journal Molecular Therapy in April 2012. Patients in the treatment arm showed a 62% reduction in risk relative to placebo in the primary efficacy endpoint of time to first occurrence of treatment failure (p=0.0032). While the study was not powered to show statistical significance in the secondary endpoint of amputation free survival, results from a subgroup of 45 patients with wounds at baseline (the approximate profile of the Phase 3 patient population) showed a 61% reduction in risk (21% ixmyelocel-T treated versus 44% control event rate; p=0.0802). The study also met the primary safety endpoint with no meaningful differences between the treated and control groups.
Phase 3 Clinical Program REVIVE
In February 2012, several principal investigators participating in the pivotal Phase 3 REVIVE clinical trial for patients with CLI and no option for revascularization began screening patients. The first patient was randomized and aspirated in May 2012. We had previously received Fast Track Designation from the FDA for use of ixmyelocel-T for CLI in October 2010 and reached agreement with the FDA on a Special Protocol Assessment (SPA) in July 2011. The Phase 3 REVIVE No Option Trial that we agreed to with the FDA under the SPA process includes 594 no option CLI patients with tissue loss (ulcers and gangrene) at baseline. Patients will be randomized 1:1 and followed for 12 months for the primary efficacy endpoint of amputation-free survival. Patients will be followed for an additional 6 months for safety. We anticipate that enrollment will occur at approximately 80 sites across the U.S.
DCM is a severe, chronic cardiovascular disease that leads to enlargement of the heart, reducing the pumping function of the heart to the point that blood circulation is impaired. Patients with DCM typically present with symptoms of congestive heart failure, including limitations in physical activity and shortness of breath. DCM is now the third leading cause of heart failure in the U.S. There are two types of DCM: ischemic and non-ischemic. Ischemic DCM, the most common form representing an estimated 60% of all DCM patients, is associated with atherosclerotic cardiovascular disease. We estimate that there are approximately 125,000 ischemic DCM patients in the U.S., our target patient population for further clinical development. Patient prognosis depends on the stage and cause of the disease but is typically characterized by a very poor quality of life and a high mortality rate.
Current treatments for DCM patients include both heart transplantation and placement of left ventricular assist devices (LVADs). There are less than 2,500 heart transplantations in the U.S. each year, because many DCM patients are not eligible and transplants are expensive at an estimated cost of over $1 million. LVADs are also expensive at an estimated cost of over $175,000 and have a mortality rate of 50% at two years.
In February 2007, the FDA granted Orphan Drug Designation to ixmyelocel-T for the treatment of DCM. Our DCM development program is currently in Phase 2. We recently completed follow up on two U.S. Phase 1/2 trials investigating surgical and catheter-based delivery for our product in the treatment of DCM. The final results from these Phase 1/2 clinical trials were presented at the Society for Cardiovascular Angiography and Interventions (SCAI) meeting on May 10, 2012. We plan to initiate a randomized, placebo-controlled, double-blinded Phase 2b trial using catheter delivery in approximately 100 ischemic DCM patients in the U.S. in the second half of 2012.
Surgical Trial Program DCM
We completed enrollment of 40 DCM patients in the IMPACT-DCM clinical trial in January 2010 and the final patient was treated in March 2010. Participants in the IMPACT-DCM clinical trial were required to have New York Heart Association (NYHA) functional class III or IV heart failure, a left ventricular ejection fraction (LVEF) of less than or equal to 30% (60-75% is typical for a healthy person), and meet other eligibility criteria, including optimized medical therapy. Patients were randomized in an approximate 3:1 ratio of treatment to control group. Patients in the treatment group received our therapy through direct injection into the heart muscle during minimally invasive-surgery (involving a chest incision of approximately 2 inches). The primary objective of this study was to assess the
safety of ixmyelocel-T in patients with DCM. Efficacy measures include cardiac dimensions and tissue mass, cardiac function (e.g. cardiac output, LVEF, cardiopulmonary exercise testing parameters), cardiac perfusion and viability, as well as other efficacy endpoints. NYHA functional class and quality of life are also assessed. Patients were followed for 12 months after treatment.
Catheter Trial Program DCM
The Catheter-DCM clinical trial was designed to explore catheter-based direct injection delivery of ixmyelocel-T to treat DCM patients. This multi-center, randomized, controlled, prospective, open-label, Phase 2 study enrolled patients DCM at clinical sites across the United States.
We reported final 12-month results from the Catheter-DCM Phase 2 trial at the SCAI 2012 Scientific Sessions on May 10, 2012. The trial included 22 ischemic DCM (IDCM) and non-ischemic DCM (NIDCM) patients with a New York Heart Association (NYHA) heart failure class of III or IV, or moderate to severe heart failure, and a left ventricular ejection fraction of 30 percent or less, which is a measure of how much blood leaves the heart with each pump. Patients were randomized to receive an injection of the treatment into their heart muscles or to a control group, and were followed at three, six and 12 months. After 12 months, no procedural complications and no difference in adverse events were reported among patients who received the treatment and the control group. IDCM patients who received the cell treatment had a lower mean number of major adverse cardiac events (MACE) (0.22 compared to 1.67 in the control group). IDCM patients who received the treatment were more likely to see improvement in NYHA class, six-minute walking distance and ejection fraction, compared to those in the control group. No consistent trends were noted in NIDCM patients. We plan to launch a randomized, placebo-controlled, double-blind Phase 2b trial in the second half of 2012 for up to 100 ischemic DCM patients in the U.S. and using catheter administration.
Results of Operations
Research and development expenses increased to $7,069,000 for the quarter ended June 30, 2012 from $5,304,000 for the quarter ended June 30, 2011. For the six months ended June 30, 2012, research and development expense increased to $13,865,000 from $9,676,000 during the same period a year ago. The increases are due to advanced preparation and initial enrollment for the Phase 3 clinical program for ixmyelocel-T, including increased employee costs, clinical site identification and initiation, as well as certain regulatory expenses. These amounts include non-cash stock-based compensation expense of $755,000 for the quarter ended June 30, 2012, compared to expense of $674,000 for the quarter ended June 30, 2011. For the six months ended June 30, 2012, non-cash stock-based compensation expense increased to $1,199,000 from $1,035,000 for the same period a year ago. The increases in stock-based compensation expense are due primarily to an increase in the number of stock options granted which is partially offset by the decrease in the fair value per stock option for such grants.
Our major ongoing research and development programs are focused on the clinical development of ixmyelocel-T for treatment of severe, chronic cardiovascular diseases. The following table summarizes the approximate allocation of cost for our research and development projects (in thousands):
Selling, general and administrative expenses remained flat at $2,231,000 for the quarter ended June 30, 2012 compared to $2,203,000 for the quarter ended June 30, 2011. For the six months ended June 30, 2012, selling, general and administrative expenses decreased to $3,993,000 from $4,098,000 during the same period a year ago. Non-cash stock-based compensation expense included in selling, general and administrative expenses decreased to $469,000 for the quarter ended June 30, 2012 from $558,000 for the quarter ended June 30, 2011. For the six months ended June 30, 2012, non-cash
stock-based compensation expense remained flat at $840,000 compared to $813,000 for the same period a year ago.
The income (expense) related to the non-cash change in fair value of warrants was $1,948,000 for the quarter ended June 30, 2012 compared to ($2,465,000) for the quarter ended June 30, 2011. For the six months ended June 30, 2012, the non-cash change in the fair value of warrants was $1,048,000 compared to ($1,210,000) for the same period a year ago. The changes are due partly to the June 27, 2012 warrant exchange for the December 2010 warrants. Fluctuations in the fair value of warrants in future periods could result in significant non-cash adjustments to the condensed consolidated financial statements, however any income or expense recorded will not impact our cash and cash equivalents, operating expenses or cash flows.
Our net loss attributable to common shareholders was $8,566,000, or $0.22 per share, for the quarter ended June 30, 2012 compared to $9,957,000, or $0.26 per share, for the quarter ended June 30, 2011. For the six months ended June 30, 2012, our net loss was $18,310,000, or $0.47 per share, compared to $14,945,000, or $0.39 per share, for the same period a year ago. The changes in net loss are primarily due to the non-cash change in the fair value of warrants, increases in research and development expenses, and the increase in selling, general and administrative expenses as described above.
Liquidity and Capital Resources
We are currently focused on utilizing our technology to produce patient specific cell-based therapies for use in severe chronic ischemic cardiovascular diseases. At such time as we satisfy, if at all, applicable regulatory approval requirements, we expect the sales of our cell-based therapies to constitute nearly all of our product sales revenues.
We do not expect to generate positive cash flows from our consolidated operations for at least the next several years and then only if we achieve significant product sales. Until that time, we expect that our revenue sources from our current activities will consist of only minor sales of our cell products and manufacturing supplies to our academic collaborators, grant revenue, research funding and potential licensing fees or other financial support from potential future corporate collaborators.
We expect that we will need to raise significant additional funds or pursue strategic transactions or other strategic alternatives in order to complete our product development programs, complete clinical trials needed to market our products, and commercialize our products. To date, we have financed our operations primarily through public and private sales of our equity securities, and we expect to continue to seek to obtain the required capital in a similar manner. As a development stage company, we have never been profitable and do not anticipate having net income unless significant product sales commence. With respect to our current activities, such sales are not likely to occur until we obtain additional funding, complete the required clinical trials for regulatory approvals, and receive the necessary approvals to market our products. Through June 30, 2012, we had accumulated a net loss attributable to common shareholders of approximately $260,158,000. We cannot provide any assurance that we will be able to achieve profitability on a sustained basis, if at all, obtain the required funding, obtain the required regulatory approvals, commence product sales or complete additional corporate partnering or acquisition transactions.
We have also, but to a lesser degree, financed our operations through grant funding, payments received under research agreements and collaborations, interest earned on cash, cash equivalents, and short-term investments, stock option and warrant exercises and funding under equipment leasing agreements. These financing sources, in addition to our public and private sales of our equity securities, have generally allowed us to maintain adequate levels of cash and other liquid investments.
Our cash and cash equivalents totaled $28,666,000 at June 30, 2012, an increase of $23,136,000 from December 31, 2011. During the six months ended June 30, 2012, the primary uses of cash and cash equivalents included $14,553,000 for our operations and working capital requirements in preparation for the Phase 3 clinical program for ixmyelocel-T. As of June 30, 2012 we had $26,100,000 of cash deposited into an Insured Cash Sweep (ICS) program which is administered by Bank of New York Mellon. This program maximizes our Federal Deposit Insurance Company (FDIC) coverage by dividing our ICS funds into amounts under the standard FDIC maximum
and places these amounts with other ICS Network member banks (each an FDIC-insured institute). These funds are placed in savings accounts at the member banks earning interest while still maintaining insurance coverage.
Our future cash requirements will depend on many factors, including continued scientific progress in our research and development programs, the scope and results of clinical trials, the time and costs involved in obtaining regulatory approvals, the costs involved in filing, prosecuting and enforcing patents, competing technological and market developments, costs of possible acquisition or development of complementary business activities and the cost of product commercialization. We do not expect to generate positive cash flows from operations for at least the next several years due to the expected spending for research and development programs and the cost of commercializing our product candidates. We intend to seek additional funding through research and development agreements or grants, distribution and marketing agreements and through public or private debt or equity financing transactions. Successful future operations are subject to several technical and risk factors, including our continued ability to obtain future funding, satisfactory product development, obtaining required regulatory approvals and market acceptance for our products.
We believe that we have adequate liquidity to finance our operations, including development of our products and product candidates, via our cash and cash equivalents on hand as of June 30, 2012 until at least December 31, 2012. While our budgeted cash usage and operating plan for 2012 does not currently contemplate taking additional actions to reduce the use of cash over the next six months, we could, if necessary, delay or forego certain budgeted discretionary expenditures such as anticipated hiring plans or certain non-critical research and development expenditures, as well as slow down or delay certain clinical trial activity (without jeopardizing our Phase 3 clinical trial for CLI). While we could sell shares through an At the Market Sales Agreement (ATM) in order to raise additional capital, there are certain factors, such as volume of trading in our stock and our stock price, which could limit the amount we could raise in a short period of time. On a longer-term basis, we will need to raise additional funds in order to complete product development programs and complete clinical trials needed to market and commercialize these products. We cannot be certain that such funding will be available on favorable terms, if at all. Some of the factors that will impact our ability to raise additional capital and our overall success include: the rate and degree of progress for our product development, the rate of regulatory approval to proceed with clinical trial programs, the level of success achieved in clinical trials, the requirements for marketing authorization from regulatory bodies in the United States and other countries, the liquidity and market volatility of our equity securities, regulatory and manufacturing requirements and uncertainties, technological developments by competitors, and other factors. If we cannot raise such funds, we will not be able to develop or enhance products, take advantage of future opportunities, or respond to competitive pressures or unanticipated requirements, which would have a material adverse impact on our business, financial condition and results of operations. As a result of the need to raise additional capital, there is uncertainty regarding our ability to maintain liquidity sufficient to operate our business effectively over at least the next twelve months, which raises substantial doubt as to our ability to continue as a going concern. The consolidated financial statements do not include any adjustments that might result from the outcome of this uncertainty.
Off-Balance Sheet Arrangements
At June 30, 2012, we were not party to any off-balance sheet arrangements.
This report, including the documents that we incorporate by reference, contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act). Any statements about our expectations, beliefs, plans, objectives, assumptions or future events or performance are not historical facts and may be forward-looking. These statements are often, but are not always, made through the use of words or phrases such as anticipates, estimates, plans, projects, trends, opportunity, comfortable, current, intention, position, assume, potential, outlook, remain, continue, maintain, sustain, seek, achieve, continuing, ongoing, expects, management believes, we believe, we intend and similar words or phrases, or future or conditional verbs such as will, would, should, could, may, or similar expressions. Accordingly, these statements involve estimates, assumptions and uncertainties which could cause actual results to differ materially from those expressed in them. The factors described in Part I, Item 1A, Risk Factors, in our Annual Report on Form 10-K for the fiscal
year ended December 31, 2011, among others, could have a material adverse effect upon our business, results of operations and financial conditions.
Because the factors referred to in the preceding paragraph could cause actual results or outcomes to differ materially from those expressed in any forward-looking statements we make, you should not place undue reliance on any such forward-looking statements. Further, any forward-looking statement speaks only as of the date on which it is made, and we undertake no obligation to update any forward-looking statement or statements to reflect events or circumstances after the date on which such statement is made or to reflect the occurrence of unanticipated events. New factors emerge from time to time, and it is not possible for us to predict which factors will arise. In addition, we cannot assess the impact of each factor on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. These forward-looking statements include statements regarding:
· potential strategic collaborations with others;
· future capital needs and financing sources;
· adequacy of existing capital to support operations for a specified time;
· product development and marketing plan;
· features and successes of our cellular therapies;
· manufacturing and facility capabilities;
· clinical trial plans and anticipated results;
· anticipation of future losses;
· commercialization plans; and
· revenue expectations and operating results.
As of June 30, 2012, we would not expect our operating results or cash flows to be affected to any significant degree by the effect of a sudden change in market interest rates or credit conditions on our securities portfolio.
Our vendors in countries outside the United States are typically paid in Euro. However, such expenditures have not been significant to date. Accordingly, we are not directly exposed to significant market risks from currency exchange rate fluctuations. We believe that the interest rate risk related to our accounts receivable is not significant. We manage the risk associated with these accounts through periodic reviews of the carrying value for non-collectability and establishment of appropriate allowances. We do not enter into hedging transactions and do not purchase derivative instruments.
Disclosure Controls and Procedures
Our management, with the participation of our Chief Executive Officer and Chief Accounting Officer, evaluated the effectiveness of our disclosure controls and procedures as of June 30, 2012. The term disclosure controls and procedures is defined in Rules 13a-15(e) and 15d-15(e) under the Exchange Act. Based on their evaluation, our management, including our Chief Executive Office and Chief Accounting Officer, concluded that our disclosure controls and procedures were effective.
Changes in Internal Control Over Financial Reporting
During the quarter ended June 30, 2012, there were no changes made in our internal control over financial reporting (as such term is defined in Rules 13a-15(f) and 15d-15(f) of the Exchange Act) that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.
From time to time we receive threats or may be subject to litigation matters incidental to our business. However, we are not currently a party to any material pending legal proceedings.
Information regarding risk factors of the Company is set forth in Part 1, Item 1A, Risk Factors, of our Annual Report on Form 10-K for the fiscal year ended December 31, 2011, which was filed with the Securities and Exchange Commission on March 15, 2012. There have been no material changes in our risk factors from those disclosed in Part 1, Item 1A., Risk Factors of our Annual Report on Form 10-K for the fiscal year ended December 31, 2011.
The Exhibits listed in the Exhibit Index immediately following the Signature, are filed as a part of this Quarterly Report on Form 10-Q.
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.