Arrowhead Pharmaceuticals, Inc. (ARWR) Q2 2012 Earnings Report, Transcript and Summary

Good afternoon. And welcome to the Arrowhead Fiscal 2012 Second Quarter Financial Results Conference Call. All participants will be in listen-only mode. [Operator Instructions] After today’s presentation, there will be an opportunity to ask questions. [Operator Instructions] Please note this event is being recorded. I would now like to turn the conference over to Michael Levitan. Please go ahead, sir.

Thank you, Operator. Good afternoon, everyone. And thank you for joining us today to discuss Arrowhead's results for its fiscal 2012 second quarter ended March 31, 2012. With us today from management are President and CEO, Dr. Christopher Anzalone; and Chief Financial Officer, Ken Myszkowski. Management will provide a brief overview of the quarter and we’ll then open your call up to your questions. Before we begin, I would like to remind you that comments made today’s -- during today's call may contain certain 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. All statements other than statements of historical fact, including without limitation those with respect to Arrowhead’s goals, plans, and strategies, are forward-looking statements. Without limiting the generality of the foregoing words such as may, will, expect, believe, anticipate, intend, could, estimate, or continue, or the negative or other variations thereof or comparable terminology are intended to identify forward-looking statements. In addition, any statements that refer to projections of Arrowhead’s future financial performance, trends in its businesses, or other characterizations of future events or circumstances are forward-looking statements. Forward-looking statements represent managements’ current expectations and are inherently uncertain. You should also refer to the discussions under Risk Factors in Arrowhead’s annual report on Form 10-K and the company’s quarterly report on Form 10-Q for additional matters to be considered in this regard. Thus actual results may differ materially. Arrowhead undertakes no duty to update any of the forward-looking statements discussed on today’s call. With that said, I’d like to turn the call over to Dr. Christopher Anzalone, President and CEO of the company. Chris?

Thanks, Michael. Good afternoon, everyone, and thank you for joining us on our call today. Arrowhead made substantial progress on multiple fronts during the second quarter of our 2012 fiscal year and soon thereafter. We made progress in our existing programs, we made moves that maximize the value of our platforms, and we expanded our assets and capabilities in the manner that strengthens our core programs and provides new business opportunities. These are important value drivers. Our recent major accomplishments include the following. One, received FDA clearance to initiative Adipotide Phase I clinical trial. Two, executed a collaboration and joint licensing agreement with Alnylam, a leading RNAi therapeutics company which allows Arrowhead to develop a Dynamic Polyconjugate or DPC enabled RNAi therapeutic candidate targeting hepatitis B virus or HBV, and allows Alnylam to utilize DPC delivery technology for one RNAi therapeutic product. Three, received a patent from the U.S. Patent and Trademark Office covering siRNA Inhibitors of HIF-2a, a target treating renal cell carcinoma. Four, entered into a strategic alliance and master services agreement for RNAi therapeutics with Axolabs GmbH, a custom research organization offering preclinical solutions and consultancy in the field of oligonucleotide therapeutics. Five, released the white paper on HBV and potential RNAi treatment, including Arrowhead DPC enabled RNAi therapeutic and development. And sixth, acquired Alvos Therapeutics formerly known as Mercator Therapeutics Incorporated, a pioneering in targeting for tumors and tumor vascular cell in humans, including what we believe is the world largest proprietary library of human-derived targeting peptides. Each accomplishment makes Arrowhead a stronger company and drives long-term value for our shareholders. As such, let’s consider some of them more closely. In January, the IND for our obesity drug candidate Adipotide was accepted by the FDA allowing the initiation of a clinical trial that has the safety of the compound. Adipotide is a new class of treatment for obesity, design to specifically kill blood vessels supplying white fat tissue. This is the first and what we believe to be powerful suite of drug candidates that not only may help to combat obesity, but may also help to reverse symptoms associated with Type 2 diabetes. The Phase I clinical trial will be conducted by The University of Texas MD Anderson Cancer Center which will bear all the direct costs associated with this trial. This is an important platform for us, because of the powerful animal data that have been generated across multiple species, the unique mode of action and the enormity of the health problem. In fact in analysis of the obesity epidemic in the United States was presented just yesterday at the Centers for Disease Control and Prevention, and published online in the American Journal of Preventive Medicine. It predicts that by the year 2030 42% of Americans will be obese. These numbers are staggering because it means 32 million more obese people within 2 decades and this is on top of the almost 78 million people who were reportedly obese in 2010. This is not a cosmetic problem but rather represents a public health cliff from which we cannot fall without incurring crippling economic and social costs. Obesity is potentially the largest pharmaceutical market in the industrialized world and we believe that our technology puts Arrowhead at the leading edge. Also in January, we executed a collaboration and joint licensing agreement with Alnylam. As part of that agreement, we licensed to Alnylam, the use of our DPC to deliver siRNA against a single target. This includes active collaboration to ensure that the DPC and siRNA chemistries are optimized for this target. These collaborations have begun and they represent an important step for us to become a partner to additional companies in RNAi therapeutics. We believe these partnerships suggest validation for the DPC platform, particularly because we were able to execute it only 2 short months after acquiring the DPC technology. In addition, we are confident that this has a potential to lead to a larger partnership with Alnylam, as its scientists learn more about the broad power of DPC-enabled delivery. The other side of the Alnylam deal provided us with a license to develop and commercialize in RNAi therapeutic, targeting HBV, the DPC-enabled RNAi therapeutic candidate targeting HBV was one of the more advanced RNAi programs at Roche and we have continued this work. We now have a large data set that includes impressive results with multiple sequences across multiple animal models. This will be our first DPC-enabled candidate in the clinic. We have completed work, validating the specific type of DPC, siRNA sequences and chemical modifications we will use. This data will be presented over the coming months and weeks at scientific conferences and will be submitted for publication. We plan to file an IND late in the second calendar quarter of 2013, the white paper we published which is available on our website, describes the substantial unmet need for 350 million HBV carriers worldwide and how RNAi and DPCs in particular are well suited to address the deficiencies of current treatment options. There is currently no cure for HBV and our treatment goal is to provide that cure rather than offering chronic treatments. We are exploring the potential to conduct this trial in Asia, where we believe, we may be able to conduct the Phase 1 in HBV infected volunteers. Such a trial would be designed to generate an efficacy readout during Phase I, enabling relatively early partnering opportunities for these candidates. Hepatitis B candidates have created a huge amount of value for many biotech and pharmaceutical companies recently, and we believe that HBV represents a similar but less crowded market that can grow a substantial interest. We are well-positioned to benefit from this interest. Part of the final development of the siRNA chemistry used in our HBV candidate was done with Axolabs as part of our strategic alliance. Axolab's core team and facilities were part of Roche in Germany and before that they were the world’s first RNAi company. This team has helped to round out our capabilities and we believe that will make our partnering value proposition even stronger. Under Roche, the DPC technology was largely embargoed from publicity. We are committed to publishing some of these data to unveil the power and flexibility of this platform. We are currently preparing and submitting multiple reports to high impact peer-reviewed journals and expect publications this year. In addition, Arrowhead scientists will present DPC enabled siRNA delivery data including our RNAi therapeutic for HBV at 6 scientific conferences in May and June alone. We are excited about these presentation opportunities at conferences around the world, which showcase years of efforts by our scientists to bring DPC-enabled therapeutics to market. Our first clinical candidate using RONDEL delivery system, CALAA-01 is currently being studied in humans in the Phase Ib trial that we expect to be complete in early summer. We continued to be pleased with what this has taught us about the RONDEL system. We still have not seen any SAEs that we believe are attributable to the delivery platform. This coupled with the clinical data we reported in the journal Nature 2 years ago, demonstrating mRNA and protein knockdown in biopsy tumors, as well as dose-dependent accumulation of our nano particles in tumor cells give us comfort that RONDEL is a powerful solution to systemic delivery of small RNAs. Finally, we announced last month our acquisition of Alvos Therapeutics, formerly known as Mercator Therapeutics. Alvos has licensed a large platform of proprietary human-derived homing peptides from MD Anderson Cancer Center that we believe should be the world’s largest human-derived targeting library. This technology is designed to direct therapeutic agents to primary and metastatic tumors, and associated vasculature, and then shuttle those agents directly into targets cells. The platform is powerful because of its breadth and how it was derived. Regarding latter, MD Anderson employed phage display technology in end-stage cancer patients to identify peptides sequences that will bind and be rapidly taken up by specific cell types. To our knowledge, only MD Anderson has been able to screen patients -- human patients in this way. The result is a library of approximately 42,000 sequences that will bind specifically to certain cell types. This should be immediately relevant therapeutically because they were derived from human patients rather than animal models. While the goal was to produce sequences that could be conjugated to cancer drugs, in order to deliver those drugs preferentially to cancer cells, the library has substantial value beyond oncology as well. In fact, we have sequences that will specifically bind virtually any tissue in the body, enabling us to target a wide variety of drugs to their preferred destination and nowhere else. As large and broad as this is, we also have the opportunity to work with MD Anderson to screen additional patients to grow the library further. This acquisition strengthens our RNAi program because it is highly synergistic. We intend to apply the Alvos technology for targeting our proprietary siRNA delivery vehicles. Remember that our 2 primary siRNA delivery systems, DPCs and RONDEL are highly valuable in part because they are targetable. We now have a huge proprietary library of targeting peptides for linking 2 of these vehicles, providing our program with a powerful new source of flexibility and value in oncology and beyond, where we use the library to create our own targeted RNAi therapeutics and we believe that this will be attractive to partners as well. The new library is also valuable in creating a new class of therapeutics, peptide-drug conjugates or PDCs. We see this as a significant new value driver and new source of non-dilutive capital. Our model with this program is to create new proprietary drugs by linking our homing peptides to generic drugs. This is designed to create “super generics” that have the advantage of years of clinical data, but are now targeted to accumulate preferentially at specific cells, while being invisible to other cells. These holds the promise of decrease in side effect and toxicities, while increasing efficacy. Our program is also focused on working with partners to target their drugs and thereby enhancing their efficacy and limiting side effects. This could be used to rescue failed compounds that may have been discontinued due to unacceptable off target effects. It could also be used as a powerful patent extension strategy by helping partners create advanced new drugs with those that are about to lose patent protection. Even though, they would be using existing APIs, these would be transformational new drugs that are actively guided to target cells. We are very excited by this business and believe we will have substantial traction with large pharmaceutical companies, most of which are facing a steep patent cliff. The Alvos acquisition also serves to unify our programs. We are truly a targeted therapeutics company now. We are leveraging a collection of RNAi platforms, much of which was assembled by Roche for over $500 million to create targeted RNAi therapeutics. We are also using or targeting platform to create targeted non-RNA drugs and Adipotide is our first clinical candidate. With that update, I’d now like to turn the call over to our CFO, Ken Myszkowski, to review our financials for the period. Ken.

Thanks, Chris, and good afternoon, everyone. As we reported earlier today, our net loss attributable to Arrowhead for the quarter ended March 31, 2012 was $5.3 million or $0.50 per share, based on 10.7 million weighted average shares outstanding. This compares to a net loss attributable to Arrowhead of $2.9 million or $0.40 per share based on 7.2 million weighted average shares outstanding for the quarter ended March 31, 2011. Total operating expenses for the quarter ended March 31, 2012 were $4.9 million, compared with $1.8 million for the quarter ended March 31, 2011. The increase in operating expenses is due to the cost associated with the Madison research facility acquired from Roche and the associated technical staff and research programs. Net cash used in operating activities for the first 6 months of fiscal 2012 was $6.9 million compared with $5 million in the prior year period. Turning to our balance sheet, at March 31, 2012, our total cash resources were $6.3 million comprised of cash and cash equivalents of $3.6 million and $2.7 million due to us from a recent financing. Cash outlays for R&D and G&A were $7 million during the first half of the fiscal year. While cash inflows consisted of revenues of $200,000, cash from the sale of investments of $300,000 and proceeds from the sale of equity securities of $2.6 million. Our shares outstanding at March 31, 2012 were 10.8 million, up from 2.2 million -- up 2.2 million from 8.6 million shares at September 30, 2011. The increase in shares outstanding is primarily due to shares issued to Roche for the Madison acquisition as well as related financings. With that brief overview, I’ll turn the call back to Chris for concluding remarks.

Thank you, Ken. Our accomplishments over the past few months have been important and that they touch on our key value drivers. When you consider these in future accomplishments, think about them in a framework of how we view long-term value creation. There are 5 broad categories that we believe are important in creating shareholder value. So as we grow this company, evaluate our progress based on how our accomplishments fit into these 5 groups, they are one, all the science. Shareholder capital is precious and we will only invest -- and we only invested in world class science. Two, be transformational. Incrementalism is not a viable strategy for us. If we are going to put shareholder capital at risk, it must be for something that is potentially transformational in medicine. Three, do not play the binary game. Many small biotech companies are focused on a single drug candidates and this often leads to a binary outcome. We view this as a bet not a business. We look to engineer multiple shots on goal and thereby provide our shareholders with a more muted downside risk and broader upside potential. Four, rely on leverage. We operate under the strategy of building something once and selling them multiple times. This means focusing on multiple scalable platforms on which we may build multiple drugs. In addition, these platforms should be usable together, thereby providing another layer of combinatorial leverage that could lead to a virtually limitless set of potential drugs at a manageable incremental cost. And five, enable non-dilutive funding. Ensure that the platforms we’re developing have value to larger pharmaceutical and biotech companies. These companies may therefore provide non-dilutive capital to help fund our internal programs in return for access to these platforms for their own drug development. We are all motivated by an overarching appreciation for opportunity, opportunity to cure disease, opportunity to alleviate suffering, and opportunity to build value for our shareholders. However, as a biotech company, we must be governed by risk and more appropriately mitigating risk. We believe that the simple framework I just outlined helps us to maximize our opportunities and limit our risk exposure. Further, we believe that our current programs and more specifically our recent accomplishments fit neatly into this framework. Thank you for your interest. And I would now like to open the call out to your questions.

[Operator Instructions] Our first question and partner pronunciation comes from Milton Aronowitz from Wells Fargo Advisors.

Milton Aronowitz, shareholder, and follow your company pretty closely. January 4th, you had a release. FDA clears JV clearance to enunciate Clinical I trials for your obesity drug and you’re first quarter were a part of February 9, you said you talked about the progress and you’ve been cleared to begin the human clinical trials. When is this going to happen? It’s been 4 months since you were first given clearance?

Yes. Thanks very much for that question. It’s a good question. So indeed we weren’t given clearance by the FDA to move forward then we had a bit of a delay, because we decided to change the protocol a bit. There are few minor changes. The one, primary change had to do with taking blood samples from patients. In the regional protocol, it was an option and we decided to make that mandatory, just to make sure, we could generate the data that we needed and that took a little of time to go back to bureaucracy of MD Anderson to get IRB approval, that is finished. And so we believe, we should be dosing patients anytime now. Now, also keep in mind that we are not paying for that Phase 1 trial. The good news to that of course, that we are I think receiving a large amount of value for no additional capital. The bad news is, I have limited control over how much I can push on them to move quickly. I believe that we should be dosing that patient literally any day now. But this is of course a bit later than I was hoping, given that we could have moved into the trial in January.

[Operator Instructions] Our next question comes from Don Hutchinson of Safe Harbor Financial Management.

I am curious to know what at least rough plans might be for raising additional capital as it appears the amount that you have on hand right now is apparently not going to last too awfully long.

We have not given any guidance on our plans but here is what I can tell you. We have a facility from Lincoln Park Capital that we took out must have been in October of 2011, just prior to the Roche acquisition. And that facility gives us the ability to draw down $15 million at our own discretion. So we’ve added something that gives us an off of lot of flexibility about how and when to bring in outside capital. So that helps us sleep at night if you will. So we’ve got that. We are also as you know, actually looking to partner parts of our RNAi program as well as this new targeting program. And so between Lincoln Park facility, our partnering endeavors and potential outside capital raise, we feel comfortable that we have access to the capital that we need. Again, we have not giving guidance on what our short-term strategy is on that. But again, we’re comfortable that we have access to the capital we need to keep our programs moving forward.

Could you just refresh me I do remember now the -- about this facility of Lincoln. Just tell us how that actually can occur in the terms?

Sure. We think it’s a really nice facility actually, because it gives us an awful lot of flexibility about how much money to raise and when to raise it. Ken, would you like to discuss the parameters of that.

Sure. So we have the ability at our discretion to request up to $0.5 million from the facility. And we could pull that down once every 4 days. The pricing is based upon the average share price looking back over the past 12 days. So there is a mechanism that comes into play that calculates what the shares will be based at and we will know what that is, when we decide to pull the funds off of that facility. Like Chris said, it’s $15 million facility. It has a 3 year term and it’s available to us at our discretion. And just -- one more one thing on that, there is no warrant associated with that, and so the cost of that capital we believe is fairly low relative to other capital.

[Operator Instructions] It appears we have a follow-up from Milton Aronowitz from Wells Fargo Advisors.

Chris, what's your timetable for Calando to wrap up Ib and have you -- are you exploring actively any partnerships in regard to that?

We will -- we intend to wrap up the Phase Ib in early summer. We feel like we’re on track to do that and feel comfortable that’s going to happen. We are actively looking to partner the RONDEL platform. The way we are doing that now is a bit different than we have approached companies in the past. As you know, you have been a long-term investor, as you know, that well -- we were focus only on Calando until October and now we have a fundamentally different value proposition. We’ve got a much larger set of platforms that we can use and we can help companies get into RNAi and develop their own RNAi therapeutics. And so we approach them in a slightly different way. We view RONDEL as a very important piece of that, but it’s no longer the only piece. And so we generally speak with companies -- with the menu of options and now RONDEL is one of those options. Now, it’s attractive one of course, because it’s got clinical data and because we -- the clinical data to us at least suggests that it is a well-tolerated system. And so it’s an important part of our platform but it’s only one part.

Would you expect to go into Phase II?

No. We haven’t given any guidance on that, yet. We have finished the Phase Ib, release the data, and then consider what our options are for that program.

At this time, we will conclude the question-and-answer session. I would like to turn the call back over to Christopher Anzalone for any final remarks, the management may have.

Thanks very much for your interest. And we look forward to talking to you next quarter.

The conference has now concluded. And we thank you for attending today’s presentation. You may now disconnect your lines and have a wonderful day.