Arrowhead Pharmaceuticals Inc (ARWR) 2012 Q1 法說會逐字稿

Good afternoon and welcome to the fiscal 2012 first-quarter financial results conference call. All participants will be in a listen only mode. (Operator Instructions) After today's presentation there will be an opportunity to ask questions.

Please note this event is being recorded. I would now like to turn the conference over to Michael Levitan. Please go ahead.

Thank you, operator. Good afternoon, everyone, and thank you for joining us today to discuss Arrowhead's results for its fiscal 2012 first quarter ended December 31, 2011.

With us today from management is our President and CEO, Dr. Christopher Anzalone, and Chief Financial Officer, Ken Myszkowski. Management will provide a brief overview of the quarter and we will then open this call up to your questions.

Before we begin I would like to remind you that the comments made 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 made other than -- 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 management's 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 reports 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 would 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.

The first quarter of our 2012 fiscal year saw a transformational change for Arrowhead. As I have mentioned before, Arrowhead is now a focused and operationally integrated nanomedicine company. With the acquisition of Roche's RNAi assets and facility we now have broad capabilities in small RNAi therapeutics and a facility in Madison, Wisconsin, where we are consolidating our R&D operations.

While these initiatives have increased our cash burn, we still maintain a capital efficient structure and our ability to create value has increased dramatically. We believe that our portfolio of delivery options, our broad RNAi chemistry licenses, and our state-of-the-art facilities and technical talent represent the most complete set of RNAi capabilities in the industry. We are able to rapidly advance our own products and are positioned as an attractive partner for companies interested in RNAi therapeutics.

In addition to these important advances within our RNAi programs, we also made great progress in our obesity program. In the quarter and more recently, we achieved two goals with the publication of nonhuman primate data that received widespread news coverage and clearance to initiate human clinical trials by the FDA.

Our recent major accomplishments include, one, acquiring Roche's RNAi assets and research facility. Two, executing a collaboration and joint licensing agreement with Alnylam that allows Arrowhead to develop a DPC-enabled RNAi therapeutic candidate targeting hepatitis B virus, or HPV, and allows Alnylam to utilize DPC delivery technology for one of their own RNAi therapeutic products.

Three, reporting preclinical results for Adipotide demonstrating substantial weight loss, reduction in body mass index, and abdominal circumference in obese rhesus monkeys. These data were published in the journal Science Translational Medicine and our partner, M.D. Anderson, has received clearance to initiate a Phase I trial in humans. Four, generating cash from financing activity and implementing a 1-for-10 reverse stock split. And five, extending the Company's management team, Board of Directors, and Scientific Advisory Board.

Previously I have commented extensively on our October 2011 acquisition of Roche's RNAi therapeutics group, select licenses, and state-of-the-art research facility in Madison, Wisconsin. Roche invested over $0.5 billion to acquire and develop the technologies necessary to independently create RNAi therapeutics.

We now have the most comprehensive portfolio of nucleic acid delivery platforms, including our proprietary RONDEL system, Dynamic Polyconjugates or DPCs, and Liposomal Nanoparticles or LNPs. We also have licenses to use the three primary RNAi chemistry formats -- canonical, Dicer-substrate, and MIRA duplexes.

With world-class technologies and a world-class team, it is worth emphasizing that Arrowhead is now the most comprehensive RNAi therapeutics company in the world and well-positioned as a partner of choice for large biotech and pharmaceutical companies interested in RNAi therapeutics. We are well underway to complete the integration of our technologies and R&D activities in Madison.

I would like to discuss some of our recent progress in greater depth. In November, primate data with our anti-obesity peptide, Adipotide, was published in science translational medicine. Adipotide specifically targets and kills blood vessels supplying white fat tissue and there is significant interest is in a non-CNS therapy that has the potential to help obese patients.

Published data demonstrated weight loss reduction in body mass index and abdominal circumference and marked improvements in insulin resistance in obese rhesus monkeys after only 28 days of treatment. This was an important achievement as most anti-obesity drug candidates have failed to translate rodent data into positive primate data. We have made this transition in the data we are striking.

In January we announced that the investigational new drug application, or IND, for Adipotide was accepted by the FDA, thereby providing clearance to initiate a Phase I clinical trial to test the safety of the compound in human patients. Our partner, M.D. Anderson Cancer Center in Houston, plans to begin treating obese prostate cancer patients in the Phase I study in the coming weeks and is some bearing all of the direct costs associated with this trial.

Up to 39 patients are expected to take part in this Phase I trial; up to five dose levels of the drug will be tested. Three participants will be enrolled at each dose level with each group of participants receiving the lowest dose level by injection under the skin once per day for 28 days and each new group receiving a higher dose than the group before it if no intolerable side effects are seen. This will continue until the highest tolerable dose is found.

Initiation of this trial will be a significant milestone for our rapidly advancing obesity program. We are very excited about the potential for Adipotide platform and believe we have a very powerful suite of drug candidates that not only may help combat obesity but also may help to reverse symptoms associated with Type 2 diabetes.

There are a number of reasons that the Phase I is enrolling obese prostate cancer patients rather than focusing exclusively on obesity. One, prostate cancer patients on long-term hormone therapy become obese over time due to chemical castration. Two, obesity complicates dosing regimes so decreasing a patient's BMI could make ongoing cancer therapy simpler and more effective.

Three, there is increasing data that some tumor types may have become less aggressive if fat load is decreased because fat acts as an endocrine organ. Therefore, in addition to increasing quality of life for prostate cancer patients, weight loss may be helpful in management of the disease. M.D. Anderson is a leader in prostate cancer treatment so we expect it to be able to enroll patients rapidly.

If the Phase I is successful and we establish a safe dosing schedule, we will have the ability to do one or multiple Phase II trials. These could include more prostate cancer patients and/or otherwise healthy obese patients and/or Type 2 diabetes patients. Phase I data will help us understand Adipotide's safety profile and also help us choose an optimal patient population that can benefit from this therapy.

In January, we executed a collaboration and joint licensing agreement with Alnylam allowing Arrowhead to develop DPC-enabled RNAi therapeutic candidate targeting hepatitis B virus, or HPV, and Alnylam to utilize DPC delivery technology for one of its own RNAi therapeutic products. Both companies are eligible to receive milestone payments and royalties on sales of product resulting from the licenses.

This deal is important to Arrowhead for several reasons. To license this technology to a leading RNAi company like Alnylam so soon after we acquired the technology from Roche is a strong validation of the potential for the RNAi delivery assets that we acquired. We licensed the DPC technology to Alnylam for a single product only, allowing us to demonstrate what our technology can do without giving up much in terms of rights.

As we work in close collaboration with Alnylam we are certainly open to further partnerships that would provide Alnylam with access to the DPCs for additional targets.

More importantly, in December I said I would provide more guidance on our first independent lead compound coming out of the DPC program after the first of the year. I am pleased to say that the license we now have from Alnylam we are now able to expand our pipeline to include our first DPC-enabled RNAi therapeutic candidate targeting HPV.

With over 350 million carriers worldwide, HPV represents a large underserved medical need and one that RNAi and DPCs are well-suited to address. We are very pleased with the preclinical data generated by Roche and are excited to be able to develop a proprietary DPC-enabled RNAi therapeutic targeting HPV.

Of course, all this progress with our new RNAi platforms does not suggest that we are backing away from RONDEL and our first clinical candidate, CALAA-01. To the contrary, we have a team in Madison that is focused on continual optimization of RONDEL and actively working to broaden its use. The CALAA-01 Phase Ib is treating patients and we expect this to be complete by summer.

With that update I would now like to turn the call over to our CFO, Ken Myszkowski, to review our financials for the periods. Ken?

Thanks, Chris, and good afternoon, everyone. As we reported earlier today, our net loss attributable to Arrowhead for the fiscal 2012 first quarter was $2.5 million, or $0.25 per share, based on 10.1 million weighted average shares outstanding. This compares with a net loss attributable to Arrowhead of $1.4 million, or $0.20 per share, based on 7.2 million weighted average shares outstanding for the quarter ended December 31, 2010.

On a consolidated basis net cash used in operating activities for the fiscal first quarter and fiscal 2012 was $2.7 million compared with $900,000 in the prior-year period. For the quarter ended December 31, 2011, revenues were $23,000 compared to revenues of $296,000 for the quarter ended December 31, 2010.

Total operating expenses for the quarter ended December 31, 2011, were $4.1 million compared with $3.6 million during the quarter ended December 31, 2010. The increase in operating expenses was primarily due to new costs associated with the research facility we acquired from Roche, the associated technical staff, and research programs.

Turning to our balance sheet, as of December 31, 2011, we had cash and cash equivalents of $6.8 million as well as $3.9 million in subscriptions receivable from completed financing transactions. Our cash balance at September 30, 2011, was $7.5 million. The decrease in our cash balance was the result of operating spending somewhat offset by in-flows from financing and investing activities.

With that brief overview I will turn the call back to Chris for concluding remarks.

Thank you, Ken. As you can see we are well underway in our efforts to make Arrowhead a focused and operationally integrated nanomedicine company with multiple products and technologies. Arrowhead's unmatched assets and capabilities in small RNAi therapeutics position us as a partner of choice in this area. And we also have our own proprietary programs in oncology and HPV.

Our platform anti-obesity technology is potentially revolutionary and we expect that the Phase I trial for Adipotide at M. D. Anderson will commence in the coming weeks. With a strong management team, state-of-the-art R&D facilities, and a strengthened balance sheet and capital resources we are very excited about the rest of this year.

Thank you for your interest and I would now like to open up the call for questions. Operator?

(Operator Instructions) Donald Hutchinson, Safe Harbor Financial Management.

Good afternoon. I wonder if you could give an update about what might be happening with Nanotope and Leonardo. Then, if you could, could you construct a potential calendar of events that might occur during the year?

Sure. Thanks for that question. First with Nanotope and Leonardo, we have not spoken about them too much of late because they are minority holdings for us. They continue to move forward.

Leonardo, as you know, is leveraging the great team and resources that Mauro Ferrari has assembled in Houston, Texas. It is a multi-staged drug delivery platform that we think is usable for small RNAs as well as for small molecules. We are very bullish on this platform and it is moving along.

We own less than 10% of that company right now so we don't talk about that too much. It is potentially synergistic with what we have in Madison regarding the DPCs and RONDEL, and so we are certainly exploring ways that we may be able to work together with that multistage platform because we do think that it has significant long-term value.

With Nanotope, again we own a minority position in that so we have not really spoken about it, but that platform continues to move forward as well on a few fronts. Cartilage regeneration, spinal cord regeneration are two major focus areas and they continue to develop those areas.

Now regarding a timeline or a roadmap for what we can expect later this year, we are having our annual meeting next week and I will be discussing in a bit more detail what our plans are for the rest of the year. And so I can give more color to that next week. I will have a presentation that will be webcast after the meeting is over, so stay tuned for that.

(Operator Instructions) George Santana, Ascendiant Securities.

Thanks for taking my question. Can you comment about your financing strategy going forward?

Sure, I can tell you this. When we acquired the Roche assets we increased our burn rate substantially compared to where it was prior to that. We were okay with that because we were also increasing, we thought and we continue to think, our ability to finance that burn rate by bringing in those assets and those capabilities.

As we have discussed in the past and as I mentioned on the call today, we think we now have the most complete capabilities in small RNA therapeutics in the industry. We have a portfolio of delivery vehicles, we have very broad freedom to operate within the RNAi chemistry, and we have second-to-none facilities that includes animal facilities as well as state-of-the-art chemistry and biology facilities.

So with all of those we believe that we can present to potential partners a very compelling case that we should be a partner of choice, and so our forward financing strategy incorporates that. We think that we can bring in deals that will bring in nondilutive capital so we are not solely reliant upon the capital markets for operating capital.

So that is the basic strategy. Beyond that what our strategy is for the next few quarters in a more granular way, we have not provided guidance on that other than, again, the fact that we believe we are now positioned to rely less on the capital markets going forward.

Okay, thank you.

(Operator Instructions) Having no further questions this will conclude our question-and-answer session. I would like to turn the conference back over to Chris Anzalone. Please go ahead.

Thanks very much for listening today and please tune in for our annual meeting next week. As I mentioned, we will be providing a presentation overview in the Company and what our strategy is for the next year. I think it might be helpful for all of those who are interested in the Company. So thanks very much. Bye-bye.

The conference is now concluded. Thank you for attending today's presentation. You may now disconnect.