Arrowhead Pharmaceuticals Inc (ARWR) 2012 Q3 法說會逐字稿

Good afternoon, and welcome to the Arrowhead Research fiscal 2012 third-quarter 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 been recorded. I would now like to turn the conference over to Mr. Michael Levitan of The Trout Group. 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 third quarter ended June 30, 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 will then open up the call to your questions.

Before we begin, I would like remind you that 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, 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 discussion 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'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. Let me start by mentioning two announcements. First, we will issue a press release before the market opens tomorrow that we have entered into an evaluation agreement with Merck whereby they will assess a novel proprietary antibody against an undisclosed target identified in the human-derived peptide targeting and discovery program which we acquired in the Alvos transaction.

We're very excited about Merck's interest in this program, and we believe there will be many more opportunities to extract value from our portfolio by partnering with BioPharma companies in areas such as this. The timing of this is also very important.

We believe that entering into a collaboration with a company of Merck's stature so soon after acquiring the platform is a tremendous point of validation. We look forward to working with Merck on this and other projects. We also look forward to entering many more collaborations stemming from our targeting library.

The other announcement I would like to touch on is that of our financing. We raised approximately $6.2 million in an offering that was over-subscribed. We believe this is the correct size because it provides us with new capital while limiting dilution for our existing shareholders. We feel comfortable with our current capitalization, given this new financing, the fact that we still have $14 million in our facility with Lincoln Park Capital, and the stage of development of our technology platforms.

Also important in the current financing is the investor group. Approximately half of the funding was provided by current shareholders led by noted investor Jim Mellon and the other half was provided by a large healthcare fund. We believe the quality of these investors speaks well of Arrowhead.

Moving to our business. As you know, last quarter we acquired Alvos Therapeutics and the platform of homing peptides originally developed at the MD Anderson Cancer Center in Houston, Texas. I would like to spend a few moments to talk about what this means for our business and how it has changed the way we position our Company to analysts, investors, and potential partners.

The Alvos acquisition completes Arrowhead's transition into a targeted therapeutics Company. We are focused entirely on bringing drugs to where they can be effective and to nowhere else. The idea of actively targeting drugs is potentially quite powerful. Guiding drugs to their intended site of action offers the promise of increasing the effectiveness of a drug while also increasing safety by limiting off-target effects.

Oncology is a prime example of the potential value of targeting. Most oncology drugs have severe side effects that make patients sick and limits how much can be administered. This is largely a targeting problem because cytotoxics are essentially poisons that can kill healthy tissue as well as cancer cells. In fact, it is thought that only a vanishingly small minority of drugs actually get to tumors while the vast majority goes to healthy tissue.

If we can guide those drugs specifically to tumors, we can increase the effectiveness, decrease side effects, and potentially increase the tolerable dose. The value of this is most obvious in oncology, but similar value could also be seen in other indications as well. This has been a key goal in the pharmaceutical industry for years, and we believe we have something revolutionary in our platform of targeting sequences. Let's review quickly what that is.

Phage display is an established method of discovering novel cell surface receptors and short peptide sequences that are rapidly internalized by various cell types. This has been used in experimental animals for some time, so, while powerful, it is not revolutionary. MD Anderson's breakthrough in the use of this technique to generate data directly in humans.

Under strict ethical guidelines, they have screened end-stage cancer patients to generate the world's largest library of small peptides that are rapidly taken up by a variety of cells in humans. The result is over 42,000 individual peptide sequences that bind to and are internalized by specific cells. What this means is that each of these 42,000 sequences will enter only a certain cell type and no others.

Why should anyone care about this? Because when you link drugs to these peptides, the resulting peptide drug conjugates, or PDCs can specifically bind the intended tissue. Our library is vast. We have peptides capable of targeting virtually any tissue in the body. In addition, we can secure rights to new patient screens so the 42,000 sequences could become 100,000 or 200,000.

We view this as a powerful land grab that can transform therapeutics across almost any indication. This targeting platform opens vast new business opportunities for us, and it also supports and enhances the value of our RNAi capabilities. Remember that after our acquisition of the Roche RNAi business, we now have a portfolio of siRNA delivery systems.

Our two primary systems, RONDEL and dynamic polyconjugates, or DPCs, are both targetable so they may be used with our homing peptides. The result is a unified business that leverages multiple platforms to target and deliver a wide range of therapeutic modalities, including small molecule drugs, peptide drugs, and small RNAs.

Because our platform supports the development of varied projects, we can stay operationally focused while becoming strategically diversified. This enables us to have many shots on goal without falling victim to the challenges of lack of focus.

We are positioned to address the following four opportunities. One, pharmaceutical companies want to make their APIs better. We have over 42,000 targeting peptides capable of homing specifically to virtually any tissue in the body and deliver therapeutic agents to where they can be effective. Two, pharmaceutical companies want enrichment strategies. Our targeting library can be used to create companion diagnostics to de-risk clinical trials by identifying those patient populations most likely to respond to treatment.

Three, we want to make generics better. Taking generics with years of clinical history and targeting them to increase effectiveness and decrease toxicity is a compelling business model. It presents the opportunity to create a proprietary drug with unique qualities and price flexibility without creating a new API. And four, pharmaceutical companies need reliable ways to deliver siRNA. We have non-lipid, targeted delivery platforms that have been validated in rodents, primates, and humans.

We now have multiple target therapeutic candidates in development including ARC-520 for treatment of Hepatitis B virus, or HBV, Adipotide for obesity, and CALAA-01 for cancer. We also have early pre-clinical oncology programs using generics and RNAi as well as active programs to enable partners to build therapeutics on our platforms.

Since our last conference call, we have made important progress on several fronts. Our major recent accomplishments include the following. One, as I mentioned, we acquired the homing peptide platform through our Alvos Therapeutics acquisition. Two, presented data for our EPC siRNA delivery platform and HBV program at six scientific conferences. Three, began dosing patients in a Phase 1 clinical trial of our anti-obesity drug candidate, Adipotide.

Four, dosed additional patients in the Phase 1B study of CALAA-01. Five, completed all internal pre-clinical requirements and selected ARC-520 as our clinical candidate for HBV. Six, initiated the final IND-enabling steps for ARC-520, including GMP manufacturing and GLP toxicology studies, and we recently filed our pre-IND data package with the US FDA.

Seven, announced the publication of a new study in the Journal of the American Diabetes Association demonstrating rapid improvement in pro-diabetic markers when obese mice are treated with Adipotide, and eight, we strengthened our balance sheet.

Each of these accomplishments represents a critical step toward our goal of developing a product pipeline that can drive long-term value for our shareholders. I'd like to highlight a few of them in more detail. Last month, we announced that patient dosing had begun in a Phase 1 clinical trial for Adipotide. The first patient has now completed his one-month treatment. This is a significant step forward, and we are eager to review the data as we accrue more patients.

The population being studied is obese, castrate-resistant, prostate cancer patients because long-term hormone therapy leads to obesity in many of these patients. The study is intended to identify a maximum tolerated dose, assess pharmacokinetics, measure the change in weight, and monitor disease progression in addition to other secondary outcome measures.

Since fat tissue is known to produce substances that can promote the growth of some tumors, investigators at MD Anderson, who are bearing all the costs of the trial, also want to learn if decreasing white fat can slow the growth of prostate cancer. Although we are not currently testing Adipotide in obese patients without prostate cancer, we believe the safety profile established in this Phase 1, should it prove acceptable, will be applicable to a Phase 1B or a Phase 2 trial in a wider obese patient population. The current trial enables us to potentially follow up with one or multiple Phase 2s in cancer, obesity, or diabetes.

As you know, Adipotide is a peptide drug that targets a protein on the surface of blood vessels that support white fat tissue. The targeting sequence of the drug is one of the 42,000 in our library. Once Adipotide is internalized into the cell, it induces apoptosis, or programmed cell death, disrupting the blood supply to fat cells and causing them to be metabolized.

Further, most of the weight loss observed in rodent and non-human primate studies was due to decreased food intake. This suggests that A, we are seeing a CNS effect without the drug even entering the CNS, and B, that animals appear to have a diminished appetite while they are losing weight, which is the opposite of what generally occurs.

The former is important from a safety standpoint because we appear to be leveraging the body's own feedback system to decrease appetite rather than directly affecting brain chemistry. The latter is important because the possibility that people may be less hungry while they are losing weight is compelling from a market standpoint.

We also recently announced the publication of a paper in the Journal of the American Diabetes Association, which reported that obese mice treated with Adipotide displayed significantly improved insulin sensitivity, improved glucose tolerance, and a reduction in serum triglycerides after only two to three days of treatment. These effects occurred independent of and prior to Adipotide-induced weight loss.

The research was conducted by a team led by renowned obesity expert, Dr. Randy Seeley, Director of the Cincinnati Diabetes and Obesity Center. This study suggest that Adipotide may also be a powerful agent against Type II diabetes. This is important because diabetes is the sister epidemic to obesity, and it may provide an additional regulatory pathway for Adipotide.

Also during the quarter, Arrowhead scientists presented data at a series of scientific conferences highlighting our DPC siRNA delivery system and our DPC-enabled, HBV RNAi therapeutic in development. We believe that our HBV program has the potential to be more effective than current treatments because of its ability to reduce the expression of viral proteins, including HBV surface antigens.

This is important because there is currently no cure for HBV, and there is a widely held theory that knocking down viral replication and viral proteins can lead to a cure, in part by enabling the patient's immune system to rebound and clear the rest of the infection. We are very excited about the program and believe that it is uniquely positioned as a potentially powerful new therapeutic. We have seen multi-log reductions in viral protein expression, translating to greater than 99% knockdown in multiple animal models.

These results are stunning for their implications to the HBV treatment, and they demonstrate the power of our targeted DPC delivery system more broadly. In a world where companies get excited about 75% or 80% knockdown, what we have demonstrated is remarkable. We anticipate publication of these results in peer-reviewed scientific journals in coming months.

In June, we announced that we selected ARC-520 as our HBV clinical candidate. Promoting it as a clinical candidate so soon after acquiring the Roche RNAi assets represents an important milestone for Arrowhead. Having completed all internal pre-clinical requirements and initiated the final IND-enabling steps, including GMP manufacturing, GLP toxicology, and we recently filed the pre-IND data package with the US FDA.

We have put in place an aggressive development timeline for ARC-520 and anticipate filing an IND or foreign equivalent at the end of Q2, 2013 and conducting a Phase 1 clinical trial in chronic HBV carriers designed to provide early proof of concept. There are thought to be more than 350 million carriers of HBV worldwide and no cure. So needless to say, this is an attractive market.

ARC-520 includes two siRNA sequences targeting two different regions of the HBV genome as a strategy to minimize the potential development of resistance in individual patients. The RNA sequences were part of a large-scale screening program initiated by Roche prior to the acquisition by Arrowhead.

Consistent with the Company's overarching strategy, the DPCs used in ARC-520 employ active ligand-mediated targeting specific to a receptor on hepatocytes. This approach results in high-potency knockdown of target genes validated in mice, rats, and non-human primates and has demonstrated a low toxicity profile in primates enabling long-term dosing.

Since we acquired Alvos in April, we have been working toward the integration of that platform into our existing operations and have begun outreach to potential partners. The initial responses have been positive because we have something that is unique and addresses targeting goals that many companies already have. The Merck collaboration is an important validation of this platform, and we are confident that multiple deals will follow.

During the previous 12 months and particularly in the last quarter, we worked hard to bring together the necessary resources, expertise, facilities, strategy, and technology platforms to become a unified, targeted therapeutics Company. Looking toward the future, we are committed to sharpening our focus and making rapid progress in our clinical development programs and executing on our business strategies.

Importantly, this includes setting aggressive goals for ourselves and for our product candidates and ensuring that we can meet them. Over the next 12 to 18 months, we expect to reach several important milestones that have the potential to be value-creating events for our shareholders. We will provide updates periodically on our progress toward meeting these milestones, which include the following.

One, complete the CALAA-01 Phase 1B by the end of August which is just a couple of weeks away. Two, publication of data on our DPC delivery vehicle in our HBV program anticipated for the end of 2012 to the first half of 2013. Three, complete IND-enabling steps for ARC-520, including GLP toxicology, GMP manufacturing of our clinical supply and final Phase 1 protocol development. These have various completion times between Q4, 2012 and Q2, 2013.

Four, file an IND, or a foreign equivalent, for ARC-520 in mid-2013. Five, release interim results from the Adipotide Phase 1. Six, screen targeting peptides and select a PDC, or peptide drug conjugate candidate, for internal development. Seven, select an additional RNAi candidate for internal development, and eight, assign homing peptides and RNAi collaborations and partnerships. With that update, I would now like to turn the call over to our CFO, Ken Myszkowski, to review our financials for the period.

Thanks, Chris, and good afternoon, everyone. As we reported earlier today, our net loss attributable to Arrowhead for the quarter ended June 30, 2012 was $8 million, or $0.71 per share, based on 11.2 million weighted average shares outstanding. This compares with a net loss attributable to Arrowhead of $1.8 million, or $0.25 per share, based on 7.2 million weighted average shares outstanding for the quarter ended June 30, 2011.

Total operating expenses for the quarter ended June 30, 2012 were $6.9 million, compared with $1.8 million for the quarter ended June 30, 2011. The increase in operating expenses includes a $2 million non-cash charge to record a reserve against a receivable from Nanotope. The balance of the increase primarily relates to costs associated with our R&D facility acquired in October of 2011, its associated technical staff, and our research programs.

Net cash used in operating activities for the first nine months of fiscal 2012 was $10.8 million compared with $6 million for the prior-year period. The increase in cash used on our operating activity primarily relates to our costs associated with our new R&D facility.

At June 30, 2012, we had cash resources of $3.3 million, including a cash balance of $2.3 million and an amount due from a previous financing of $1 million. As we reported earlier today, we also closed a registered direct offering. Gross proceeds will be approximately $6.2 million prior to commissions and fees.

Our cash at September 30, 2011 was $7.5 million. Cash outlays for R&D and G&A were $10.9 million during the first nine months of the fiscal year, and expenditures for capital equipment was $400,000 while cash inflows consisted of revenues of $200,000, cash from the sale of investments of $500,000, and proceeds from the sale of equity securities of $5.4 million.

Our shares outstanding at June 30, 2012 were 11.3 million, up 2.7 million from 8.6 million shares at September 30, 2011. The increase in our shares outstanding is primarily due to shares issued for the Roche Madison acquisition, as well as related financings. With that brief review, I will now turn the call over to Chris for concluding remarks.

Thank you, Ken. The last few months have been important in the obesity and RNAi therapeutics markets. We believe the recent FDA approvals for Arena and Vivus -- the first in 13 years -- signal an easing in the regulatory stance, an increase in acknowledgement by the FDA that obesity is one of the great healthcare challenges of our times. We see that as a positive signal for Arrowhead, our shareholders, and the millions of patients that struggle with obesity and potentially could benefit from treatment with Adipotide.

On the RNAi front, Alnylam, which is an Arrowhead partner, released some encouraging clinical data on their ALN-TTR02 candidate that sparked a lot of investor interest. The last couple of years have been difficult for developers of RNAi therapeutics, but market reaction to the Alnylam data suggests a renewed hunger for good RNAi data, even if it is early. This is encouraging, and we believe that we are well-positioned to benefit from an increased demand, particularly as ARC-520 moves toward the clinic.

During the final few minutes of the call, let's take a look at where we have come strategically and operationally. Less than 10 months ago, we were a Company with a single solution for siRNA delivery and a promising pre-clinical obesity program. Both programs were dependent on outsourcing for further development.

We are now a fundamentally different Company that provides our shareholders with a more focused business, broader upside potential, increased value to partners, and enhanced ability to rapidly innovate. Through the Roche and Alvos acquisitions, we are now a targeted therapeutics Company with deeper value drivers than just 10 months ago.

They include -- a Phase 1 clinical trial on obesity that is fully funded by MD Anderson. Completion of the Phase 1B oncology trial in the next two weeks. A very promising program in HBV that will reach the clinic next year. The world's largest human-derived peptide-targeting library that can be used for targeting drugs and the creation of companion diagnostics.

A comprehensive set of our RNAi licenses providing broad freedom to operate. A portfolio of siRNA delivery solutions, including two non-lipid platforms that are targetable and validated. State-of-the-art scientific infrastructure assembled by Big Pharma that a small biotech company could not afford to build. Complete experimental animal facilities to rapidly cycle drug development internally and a technical team of over 40 scientists that is second to none.

These are some of our tools, and we believe that we are focused on the right areas strategically. The pharmaceutical world is increasingly interested in targeting strategies, and we believe that we have the world's most powerful platform to provide this. There is a clear need for siRNA delivery, and we believe we have the most powerful platforms to solve this challenge.

From a therapeutics standpoint, obesity is garnering tremendous attention, and we are treating patients with the candidates and underlying platform that demonstrate a stand-out pre-clinical data and the unique mode of action.

Hepatitis B remains a large, underserved market worldwide that we see as a major focus for the pharmaceutical industry on the heels of tremendous value creation surrounding Hepatitis C therapies. We have a candidate that has demonstrated great promise and will be in the clinic next year. From a strategic and operational standpoint, we believe we are on strong footing. With that, thank you very much for your interest. And, I would now like to open the call to questions. Operator?

(Operator Instructions)

Donald Hutchinson, Safe Harbor Financial Management.

It has been quite a while since they announced the dosing of the first patient for Adipotide. Has the second one started?

We are not going to announce when every patient gets dosed. We announced when the first one was dosed. That was a little bit over a month ago. And, as I mentioned in my remarks, that patient has completed his treatment. But, it is not going to be our policy to give -- .

Let's just make it a general question. Are there more than one?

We announced that the first patient has been dosed, and we are actively recruiting other patients. And again, we are not going to provide granular information as we dose individual patients. But, I'll tell you this. We have seen a tremendous amount of interest in joining the trial. MD Anderson has a large patient population to draw from, and we are confident that we can accrue these patients fairly rapidly.

Okay. I, for one, am disappointed about the dilution with the stock offering. I'm curious as to why -- anyone could have figured out when the cash was going to run down to a point where new financing was necessary and could have ascertained that months ago -- why this offering was not done six months ago when the stock was about double where it is right now? Something of a mystery to me. Could you explain why the decision was to hold off?

Sure. So, timing of financing is never perfect. We were working on the Alvos acquisition for some time, and we really couldn't do anything until that was complete. Even though that was not a cash acquisition, it was material -- we thought -- to our business.

We are really building our business around this targeted therapeutics concept, and so it was going to be large part of our business going forward. We needed to get through that before we could start any discussions with potential investors. So, that pushed us off. And after that, it just takes time to speak with the right investors, and here we are. Of course, you like to raise money when you are at a higher stock price. But, you don't always have control over that timing.

Well, to be honest with you, your Company basically has been a stock-issuing machine and then a reverse-split kind of a situation. So, anybody who has owned this thing for a long time is severely underwater, and it is largely due to the constant dilution of the share base. Let me ask you a question about that other than registering my displeasure with this policy. What is the likelihood that we will have another share dilution over the next couple of years?

Let me address that a couple of ways. So first, with the question -- the possibility of additional financings in the future is certainly there. We are a biotechnology company, and biotechnology companies require capital. Toward those ends, I would submit that as a Biotech company, we have a manageable burn rate, and in fact over the last several years, we have had a fairly light burn rate compared to other biotech companies.

Now, let's look at where we are now versus where we have been in the past. We have substantially focused this business. Two years ago we were in a number of different areas, and now we are a focused biotech company. Two years ago in the biotech space, we had a single piece of technology that we think was powerful, but it was a single piece of technology in siRNA delivery.

Where we are now is we have two very large platforms. One was the result of acquiring Roche's RNAi business. Roche spent over $0.5 billion developing that program, and we acquired that. Alvos Therapeutics also has had a very attractive and very powerful platform, and we acquired that.

So, when you look at where we are, given the dilution -- no one likes dilution. If you look at where we are and the assets we have underneath us, I would submit that we have done a good job putting together the assets that we need to really grow this.

The last few years have been a very difficult time in the capital markets for small companies, particularly small biotech companies, and I think we did a very good thing a couple of years ago. And, we reacted very quickly to the damaged capital markets by decreasing our burn rate substantially. I think we acted more quickly than many of our peers, and we're still around because of that.

As we brought down on our burn rate, we were still able to do move our programs forward. And again, many other biotech companies were not able to do that. So, while again, no one likes dilution, I think that we managed that very trying time fairly well. We are still able to assemble a set of assets that I think our world-class.

Well, what disturbs me is that there was about $6 million that was raised. And about a similar amount -- slightly more, I guess, burned off in one quarter. That does not look like good tidings for the near future to me. The amounts of money may not be spectacular, but it does not look like what was raised now is going to last very long.

As I mentioned in my prepared remarks, we think the size of this raise was right, given our facility with Lincoln Park Capital, which has [another] $14 million on it, the state of our technology development, and our stock price. It's interesting -- you are concerned about the dilution which we all are. But, you are also suggesting that maybe we did not raise enough money.

It would have been better to raise money in some of their manner if there was a choice is my point.

Okay.

Continual share dilution impoverishes all shareholders.

Okay, I appreciate your comments.

Thank you.

(Operator Instructions)

Todd Aldrich.

Chris, I had a follow-up question on the previous investor's comment. That really is the ability to forecast your business. Continued goal orientation you have set out. I think part of that post-Roche asset acquisition was the ability to raise non-dilutive capital faster than what the cash burn would be. I assume by today's headline that hasn't been the case. So, to what degree can we take confidence again that we won't see further dilutions like this versus just non-dilutive capital potentially coming in?

We continue to feel confident that we have what we need to bring in non-dilutive capital. We have been -- the market for RNAi therapeutics has been difficult for everybody over the last two years. We have been disappointed at the rate of partnerships that could come out of that. We are not alone in that. If you look at Alynylam, if you look at RXi, if you look at Marina, if you look at Silence -- if you look at all those in the space, it has been a very difficult time to do partnerships.

It feels as though that market is finally turning. Given the Alynylam data that were just reported, they were good data. And, their market cap went up substantially. It feels as though the market is turning a bit, and we are hopeful of that. And when it does, we believe that we are well-positioned as a complete player with RNAi chemistry freedom to operate and a portfolio of delivery systems.

But again, to harken back to how we have been funding the Company, let's look at our competitors in the field. Let's look at Marina and RXi and Silence and others. I think that we have reacted well to the changing capital market environments. And, at the same time, have been able to bolster our underlying assets as such that when that RNAi market does turn, we are well positioned for it. Even look at Alynylam, they have always had a large cash position, but until recently, didn't trade for much of a premium on that cash. And, that is a reflection of the RNAi market.

There has been substantial interest in the market for some time. But then, there was a real concern that delivery was going to kill the field. I think the market is coming around. They have seen that is not the case, and I think that, again, we are well positioned to take advantage of that.

And, on top of that, what we have been able to do with expanding our business without substantially expanding our costs is the target. This fits very well with our RNAi assets and enables us to get into whole new markets internally as well as with partners. I think this announcement about this Merck collaboration is a good example of that. We just acquired this targeting platform quite recently, but we were able to get into this collaboration fairly quickly. We see that as a good sign that the market is going to value this platform, and the partners are going to value the platform.

I would also just ask is -- it seemed like with your June slide presentation up on the Company website in terms of goal orientation, and I think you had at least one RNAi collaboration at least potentially. I assume that's CALAA-01, or RONDEL, I think. Q4 of this year through Q1 of '13. I think you had at least two targeted for the other part of the RNAi platform there. Running from, I think, Q3 of this year again through Q1 of next year. I guess we can possibly assume the those collaborations may potentially bring in some non-dilutive capital to keep running the business?

Yes, I think that is a good assumption. I think those collaborations can take multiple structures. The least of which would be collaborations where a partner would provide financing of a development program and thereby decrease our burn rate. But, there are also certainly the likelihood of good collaborations and partnerships done where there is economics on top of that, yes.

And also, quick question just on the data for the Phase 1B. I guess that is going to be wrapped up in August. Is this going to basically be results by scientific journal? Or, is there a chance the Company could basically release those -- or self-release that?

We have not yet made that decision.

Would potential partners that would want to partner on RONDEL where that Phase 1B would be critical? Would they basically have the opportunity through maybe signing a disclosure agreement to get a look at those results? Or, would it only be after those results came out either self-released or in a journal?

Certainly as we are in discussions with partners -- once those discussions turn confidential, those data would be available, yes.

Thank you.

Lee Alper, Hammock Investors.

I got on a little late so I may have missed something. But, could you remind me again what are the terms of the Lincoln Park financing?

The Lincoln Park facility enables us to take down as much as $500,000 a week at our discretion, and there's a formula to determine what the cost of that capital is. There is a look-back period of a fairly short period of time, and then the average close of that period goes into a formula to figure out what the price is. And then, to be clear, the current financing was not a Lincoln Park financing, it was outside of that facility.

I understand that. But, are you saying you could have not gotten the Lincoln Park financing cheaper than what we paid for this dilution?

The Lincoln Park facility is not a financing strategy, at least the way I view it. The Lincoln Park facility provides us flexibility that I don't believe should be relied upon solely to operate a business. So, while we can draw down -- and, in fact, we did draw down in the third fiscal quarter as we mentioned, I would not rely solely on that facility for development. Even though that can be cheap capital. I think your point is a good one. On a spot basis, if we draw down, it is, relatively speaking, cheap capital.

All right. I think it may have been a lot cheaper than this. And, along those lines, is there any financing involved in them Merck collaboration that you're doing? Is there any funds coming from Merck?

That's a great question. Unfortunately, there are no economics disclosed. As you can imagine, anything that we say about this in the press release that will come out tomorrow morning all has to be approved by Merck, and so no economics were disclosed.

Because it's confusing if you're getting money from Merck, and you could have gotten money from Lincoln Park, why this financing was necessary at this time is a concern of a lot of people.

I appreciate that. But, also take a look at the financing in another light. We are a heavily retail stock, which is fine. But, I think it is important for us to also start to build an institutional share base. And, we brought in, I think, a very strong institution into this financing, and I think that is part of our maturation as a biotech company. So to that extent, I think that is another important aspect of the financing.

A year ago, or more maybe two -- you spun off a piece of the Company to [Weiss], I think -- the Korean company?

Yes, sir, it was a year ago -- about a year and a half ago.

Any updates on that? Are we seeing anything from that?

That is a good question. We don't have any progress updates on them. As you are likely alluding to, we do have milestone payments associated with revenue bolts from Wisepower. We do not have an update on where we are vis-a-vis those milestone payments. However, in January, we do have a $2.5 million note coming due that they owe to us as part of the acquisition. So, that note matures and will provide us, again, with $2.5 million in January.

Is there a reason why we don't have an update 18 months after we have given them the product to try to do some work with?

We have periodic updates from them. But, nothing terribly granular. I have nothing to report on when any of those milestone payments might be triggered.

The last question is, we have another collaboration -- I'm drawing a blank on the name of it -- with another one of our products with another company. Are there any updates on that?

We have a development collaboration with Alnylam, and we have no update on that. We continue to work with Alnylam on an undisclosed target of theirs.

I got to tell you, you are were leaving us pretty much in the dark on a lot of the stuff that we thought we had. A lot of potential.

I appreciate your position there. I will tell you this. Again, our internal programs continue to move forward. Our platforms continue to move forward. My hands are a bit tied on what I can tell you about development programs with other companies.

Alnylam is a publicly traded company, and they have their own considerations. They did not want to disclose what target we are helping them to deliver. When that changes, we can certainly give you an update. But right now, that is their prerogative to keep that confidential. The same thing with the new Merck collaborations. We're certainly hopeful that at some point we can update you on that progress. But right now, we cannot.

Tom [Birchfell].

I spoke with you about a year ago, I guess, and we talked about efficacy in CALAA-01. I know it was a Phase 1. But, you said that things looked awfully good at that time. And so, we have the 1B now. That is wrapping up. I am just wondering, is there any reason to think that we don't have the same type of successful efficacy indicators? Understanding it is a Phase 1, but there is no reason to think that well, this is a shot in the dark, and it didn't work?

I don't know how to react to that. As you say, it is the Phase 1 so it is not powered for efficacy. In addition, the target, RM2, or ribonucleotide reductase subunit M2, is likely not a single agent drug. There is interesting data to suggest that it will have synergistic effects with radiation or other cytotoxics, but as a stand-alone drug -- it likely is not intended to be a stand-alone drug.

Now, what I can tell you is this. When we look at the Phase 1, what we can determine on an efficacy standpoint is the efficacy of RONDEL, if you will, or the ability of RONDEL to do what we want it to do. And, as with published in Nature, we think that we have shown that it can do what we want it to do. We saw a dose-dependent accumulation of RONDEL in biopsied tumor cells and not in surrounding tissue. We saw RNA knockdown. We saw protein knockdown.

So, the way I view that Phase 1 is in two ways. One, how good is the drug? Or, how safe is the drug? And two, how good and how safe is the delivery system? It is easier to answer the latter than the former. And again, I think that the data that has been shown thus far suggests that RONDEL is a well-tolerated and potentially effective delivery system for siRNA.

Seeing we are going to finish that up roughly the end of August. And, I think you mentioned to another caller that you didn't yet determine how that is going to be announced -- the results? We have been waiting for a couple of years on this. I am away under water on my investment. I know that you roll the dice. You take your chances. But, I am anxious to hear, and I know other companies -- they sometimes even show their incremental results that are biotechs. Surely, we can get an expectation of when we are going to have the results that we have been waiting on all this time?

So again, we have not made a determination on how to provide that data. We will wrap up the Phase 1B in the next couple of weeks, and then we can make that determination and make that announcement.

Okay. Thanks.

(Operator Instructions)

Todd Aldrich.

May I ask if you feel as encouraged about RONDEL as you have been in the past based on this wrapping up at the end of August?

Yes. We still think that RONDEL is a good delivery system. Our value proposition, though, has changed as we said in the past. RONDEL is good, but now it is one of several arrows. We have another system -- the DPCs, or Dynamic Polyconjugates, that are also good. And, we also have chemistries that we have to offer. And so, I think our value as an RNAi player is just broader than it used to be. Which again is not to say that we don't believe in RONDEL, it just means that we have additional assets around it to support RONDEL and to support the overall programs.

The other part of this, too, is you had mentioned I think about a year or so back that the goal posts had moved in terms of trying to partner anything with BioPharma. That predominately being you needed good solid clinical results. It seems like to me, as you look at the potential of DPC, it is preclinical. We have nothing in human. RONDEL is the furthest along. So, it would seem that most of the focus or encouragement, I would assume, would be on RONDEL, just given how mature it is. And then, I guess, seemingly being the most ripe for partnering.

Yes, and so not to take anything away from RONDEL, but to talk DPCs up -- here's what we have with DPCs. We have a much more complete data set on the DPCs preclinically than we did with RONDEL. We have a substantial amount of non-human primate data across multiple iterations of the DPCs. We have got substantial data in various rodent models across various iterations of DPCs. So, I think the DPCs now even though it is preclinical, has a broader data set than RONDEL at the same time. And so, I think that partners are interested in DPCs as well. Even though we don't yet have clinical data on it.

You'd mentioned the ability to have a very powerful platform and/or partnering opportunity. But, I would think, too, a lot of these partners would want a viable Company that has survivability. Just my opinion, but without the ability to get significant non-dilutive capital, i.e., partnerships and funding in the door -- to what extent do you think a lot of BioPharma partners would really be that encouraged to partner that deeply without knowing or feeling that partner, meaning Arrowhead, would likely be around in 12, 18, 24 months?

I can't speak for any other company but my own. But, I have not seen that as a concern thus far.

James Cash, [Poudrehead] Pharma.

Yes, gentlemen. Could you tell me when was the first public announcement of this financing?

It was the press release this morning. I think it went on the wire -- I want to say -- at 9.15 AM or thereabouts Eastern Time.

It certainly seems like there was some anticipation of this easily a couple of days -- toward the end of last week. This really causes me dismay when it seems like the playing field is not quite level as far as who gets the information and when.

I really appreciate your comments, sir. And, I share the concern always. I can tell you that it was a confidentially marketed deal, and we did everything we could to make sure that confidentiality was honored. But, I do appreciate your concerns, and you tend to see that sometimes in financing. But, I can tell you that we did everything we could to make sure that our interactions were confidential.

Well, what was the formula by which you priced it at this? And, did you realize when you priced it at this that the stock would probably fall to that price?

I'm sorry, say that again?

What was the formula by which you priced the private placement at this $2.76? And, did you understand that when you placed it at that price the stock would fall to that price?

The lead investor dictated the terms.

(laughter) Okay. Gentlemen.

Todd Aldrich.

Did management invest in this round of financing to the extent you can answer that? And with the stock down at this level, I guess by your estimation, being a very powerful and potential value-creative proposition long-term, any chance management would be investing any time soon and put their own money to work at this level?

Management did not participate in this round. But in the past, management has invested [and backed] multiple rounds as well as in subsidiaries. I have invested substantial amounts of my own cash into the Company in the past. I did not invest in this round, but I have in the past.

Any chance management would be buying any more stock sometime soon with the stock at such a discount with such great value-add potential as you describe it?

It is certainly possible, sure.

Then, last question. Would you say the -- or could you probability-[weight] to the extent you can the opportunity for non-dilutive capital to [get them into] these partnerships between now and the first of 2013 first quarter of next year? Thanks.

I'm not going to weight that. We're speaking with companies on a number of levels, and we believe that we will bring in partnerships and collaborations. What the economics of those will look like, it is just speculation.

I guess the concern is, as you had mentioned before after the Roche asset acquisition, that there was a chance to bring in non-dilutive capital. That would likely or potentially run ahead of cash burn. That has not been the case. The collaborations that you have had so far, and seemingly, the one tomorrow we're going to hear about with Merck had no capital inflows. Thus my concern of any future collaborations from here through next year?

We do believe that capital will come in from partnerships, yes.

This will conclude our question-and-answer session. I would now like to turn the conference back over to Chris Anzalone for any closing remarks.

Thanks very much for your interest and attention today. And, we will talk to you next quarter.

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