Six Oncology-Focused Biotech Growth Engines: Mara Goldstein

TICKERS: AMGN, ARRY, CELG, CLDX, GALE, IMGN, JNJ, NLNK, ONTY, ONXX, VSTM

Source: George S. Mack of The Life Sciences Report  (8/1/13)

Mara Goldstein For Cantor Fitzgerald Senior Biotechnology Analyst Mara Goldstein, the spotlight is always on oncology and the premium revenues commanded by new products in the space. That clarity brings the science, unmet needs and market opportunity into sharp focus for investors. In this interview with The Life Sciences Report, Goldstein makes a winning case for six cancer-focused companies that range from micro caps to genuine large-cap stocks, each with upside potential that could be stunning.


Companies Mentioned: Amgen Inc. : Array BioPharma Inc. : Celgene Corp. : Celldex Therapeutics Inc. : Galena Biopharma Inc. : ImmunoGen Inc. : Johnson & Johnson : NewLink Genetics Corp. : Oncothyreon Inc. : Onyx Pharmaceuticals Inc. : Verastem Inc.

The Life Sciences Report: Mara, we've experienced outperformance in large-cap biotech, and I think this has been going on for about a year. Is that right?

Mara Goldstein: Let me break it out a bit. Year over year (YOY), the large-cap biotechs are up about 59%. In oncology, which is much of a focus for us, mid-caps have appreciated about 33%, while smaller-cap oncology-focused biotechs are up about 25%. Performance on a YOY basis is definitely skewed toward the broad group of large-cap names.

However, on a year-to-date (YTD) basis, performance has been roughly equivalent among the three groups. The group of large caps biotechs that we track—those with market capitalizations greater than $10 billion ($10B)—have risen about 36% YTD. The mid-cap oncology stocks that we track are up about 34%. The small-cap stocks that we track are up about 30%. We track performance on an absolute basis rather than share-weighted, so given merger-and-acquisition (M&A) activity in the sector, we may be underestimating performance. And while we look at universes of mid- and small-cap oncology-focused biotechs, we track all large caps, given that it is a fairly small group.

TLSR: The rally has been broad, but as you say there aren't that many biotech large caps—not a lot of Celgenes (CELG:NASDAQ) and Amgens (AMGN:NASDAQ) in biotech. Focusing for a moment on large-cap outperformance, was that a sign that investors were being conservative, or perhaps concerned about the market? A sign of an unhealthy market, where investors fled to quality? How do you read it?

MG: We don't view it as a sign of conservatism, concern or an unhealthy market. Yes, it's true that investors might view large caps as defensive names, but right now the large-cap biotechs have the drivers in place to experience high growth—those drivers being new product introductions. That is certainly the case for Celgene, a name we follow, which has experienced valuation expansion over the last year. We think the opportunity exists for continued multiple expansion.

On a YTD basis, as well as YOY, we see strong performance among the large-cap biotechs. New product cycles are widely prized among investors, and so it makes sense for companies at the initial stages of new product flow to experience valuation expansion ahead of peers that have more mature product portfolios. Again, we think Celgene is in that camp, but there are a number of other companies as well, such as Gilead Sciences Inc. (GILD:NASDAQ), Regeneron Pharmaceuticals Inc. (REGN:NASDAQ) and Biogen Idec Inc. (BIIB:NASDAQ).

TLSR: Amgen is now—and has been for quite some time—a fully integrated biopharma. It is in need of acquisitions to continue to grow, just like its big pharma brethren. At the very end of June, Onyx Pharmaceuticals Inc. (ONXX:NASDAQ) said that Amgen made an unsolicited bid for the company. You've been quite interested in that offer. Can you tell me why?

MG: First let me say that we do not cover Amgen or Onyx. But we do track this market. While Amgen may have had its reasons for making the bid for Onyx, there has been other M&A activity as well. Broadly speaking, the M&A activity reinforces our notion that access to new sources of product and revenue is of ultimate importance in maintaining growth in revenue and earnings, and can be a tremendous driver of valuation expansion. The fact that Amgen made an offer for Onyx that was just above the historical revenue takeout range usually seen for biotech valuations seems to make a statement that oncology assets are highly valued. The oncology market, broadly, has been a strong growth engine for the industry, as waves of innovation—monoclonal antibodies, personalized therapeutics are examples—change treatment paradigms and create value for companies and shareholders.

TLSR: You've used the bid and Amgen's attributed value for Onyx as something of a proxy for a company that you follow and have a lot of interest in. Could you make that case for me?

MG: We tend to view events and other activities, such as M&A, with a relative lens. That is, if a potential acquirer makes a bid for a target, that valuation can be used to benchmark valuations for other companies with similar attributes. For companies with product-related revenues, the historical takeout valuation range has been 3–9x forward revenue. We can make the case that the upper end of the range is for products experiencing either high rates of growth, or those that have commercial applications in more than one disease indication. We can apply that benchmark to the projected revenue stream of a company and discount it based on any number of risk factors, such as clinical or regulatory risk. Following the disclosure of Amgen's interest in Onyx, we examined what a company like Celldex Therapeutics Inc. (CLDX:NASDAQ) could be worth on a takeout basis using such a benchmark, because Celldex has a two clinical assets in phase 3 development.

TLSR: You were also using the Amgen bid for Onyx, an oncology-focused company, to justify your $142 target price for Celgene. Could you explain why?

MG: We didn't use it to justify our target on Celgene because we think the company's fundamentals—that is, revenue diversification driven by new products and expanding growth rates—are sufficient drivers of price-earnings (P/E) multiple expansion, and we do not view Celgene as expensive based on these fundamentals. But we did look at Amgen's bid for Onyx in the context of how it could be applied to companies that we believe have strategic value.

"It's true that investors might view large caps as defensive names, but right now large-cap biotechs have the drivers in place to experience high growth."

Broadly speaking, we have viewed takeout valuation as the upper limit of what the current and near-term fundamentals of a company could support. A premium is typically paid because the target company has assets that are valuable to the acquiring company. The value could be driven by a need to protect profit and loss from a patent expiration, a buy versus build strategy in an important subsector or for other reasons. Onyx competes with Celgene in the multiple myeloma area, and we think Amgen's bid for the company highlights the unique strategic assets in the Celgene portfolio.

TLSR: Let's stay with Celgene. When we spoke last, for The Life Sciences Report Biotech Watchlist 2013, you liked the company. Its stock has more than doubled over the past 52 weeks. It's up 110% during that time, and year to date it's up 70%. Congratulations on this call. What is the growth story from here on?

MG: When we launched coverage of Celgene in April 2012, our thesis was that Revlimid (lenalidomide), the company's flagship product for multiple myeloma, was a solid performer, but that revenue diversification away from Revlimid would occur, driven by new product launches. We are at the beginning of this dynamic, with the recent launch of Pomalyst (pomalidomide) for multiple myeloma, the pending approval of Abraxane (nab-paclitaxel) for pancreatic cancer and the regulatory filings for apremilast for psoriasis and psoriatic arthritis.

The question, as you rightly ask, is whether these other products are now fairly valued in the share price, given the valuation expansion over the past year. My argument for continued interest in Celgene is that I don't think this has fully played out yet. I believe revenue and earnings growth still have an upward bias, and this is enhanced by the company's cost structure and financial flexibility. There is also a big phase 2 pipeline that holds the potential to drive value into the shares.

TLSR: You've just mentioned a couple of molecules—Abraxane and apremilast—that offer opportunity for growth. I'm looking at Celgene's total revenue for 2012 of $5.5B. Full-year sales of Revlimid were $3.8B, a 17% increase over 2011. Vidaza (azacytidine), a DNA methyltransferase inhibitor used in myelodysplastic syndromes, did $823 million ($823M) in 2012, and that's with a direct competitor, Dacogen (decitabine, Eisai Inc. [ESALF:OTCPK]). You have two drugs combining for 84% of Celgene's top line. Why is this still a compelling story with so much revenue coming from Revlimid?

MG: For us, it's all about revenue diversification. Today, much of the company's top line is concentrated in Revlimid sales. The product has a strong hold in the market and has yet to be fully launched in Europe, so we are looking for additional growth there.

"M&A activity reinforces our notion that access to new sources of product and revenue is of ultimate importance in maintaining growth in revenue and earnings, and can be tremendous drivers of valuation expansion."

However Revlimid, as a percent of sales, will decline because of new sources of revenue. And the new revenues from Pomalyst, Abraxane and apremilast should grow at a much higher rate than Revlimid. At the end of 2012, Revlimid accounted for about 70% of Celgene's revenue, and we anticipate that will fall to about 50% in five years, but sales will still continue to grow. We think the transition is particularly compelling because it is driven by more than one product, and this diversifies risk.

Revlimid has essentially become a backbone treatment for multiple myeloma, and if you look across the broad spectrum of trials conducted in multiple myeloma, Revlimid is broadly incorporated into new combinations. We think this creates stability for the franchise. We want to see Revlimid's predominance in Celgene's top line lessen, but we don't want it to be driven by declining sales. We’d rather see that movement driven by inclusion of new sources of revenue.

TLSR: You mentioned apremilast for psoriatic arthritis. In a research note you said, "Ironically, one of apremilast's most compelling features may be is that it is not a traditional biologic." Certainly, if I were taking any of the anti-TNF (tumor necrosis factor) biologics, such as Enbrel (etanercept; Amgen), Humira (adalimumab; AbbVie Inc. [ABBV:NYSE]) or Remicade (infliximab; Johnson & Johnson [JNJ:NYSE])‎, I would love to be able to wind down to a pill. Is that going to be the big selling point for apremilast?

MG: In the spectrum of indications such as psoriatic arthritis and psoriasis, there are several small molecular agents that are widely used. These include methotrexate, cyclosporine and retinoids. But these carry a high side-effect burden, particularly when used for long periods of time, including birth defects, liver damage, kidney problems, anemia and hypertension. For patients with disease unresponsive to currently used topical medications, oral medications and phototherapy, biological agents such as Enbrel and Humira are the next treatment option.

Thoughout its clinical trial program, apremilast was thought to be seated behind the biological agents, but data generated in the phase 3 program suggests a rationale for apremilast to be seated in front of biologicals—and maybe even as a replacement, in the case of psoriasis, for methotrexate or cyclosporine. That, to us, is very compelling because that is a huge market.

TLSR: What are the catalysts for Celgene?

MG: The company has a couple of catalysts ahead. For Revlimid, the European submission for newly diagnosed disease should occur by year-end, based on the recent positive readout of a study known as MM-020. We also think that approval of Abraxane in pancreatic cancer, with a late-September PDUFA (Prescription Drug User Fee Act) date, is a catalyst.

Apremilast regulatory actions will also be a catalyst. A new drug application (NDA) for psoriatic arthritis was filed in the U.S. earlier this year, and a separate NDA will be filed for psoriasis this year, as well as a combined psoriasis/psoriatic arthritis filing in Europe. We look forward to more data coming out of the apremilast clinical trial programs, as well as product approval. And a regulatory decision in Europe for Pomalyst, for relapsed or refractory multiple myeloma, is expected this year. The company has a big, earlier-stage pipeline that doesn't have a lot of visibility, so there's opportunity from that as well.

TLSR: What is the attraction of Abraxane? This is an old cytotoxic agent, paclitaxel, reformulated.

MG: Abraxane's initial launches were based on clinical results that did not provide signficant overall differentiation versus taxol, with the exception of in certain subpopulations of non-small cell lung cancer (NSCLC) patients. But with the release of positive data from the MPACT study, Abraxane now has a role in the pancreatic cancer market, where there has been little advance and uniformly poor patient outcomes. This is the core driver behind our enthusiasm for the drug.

The MPACT trial evaluated Abraxane combined with standard-of-care therapy (gemcitabine) and showed an overall survival benefit that approached six weeks. Given limited treatment options and overall poor outcomes for the disease, there is strong rationale to use Abraxane in combination with gemcitabine. MPACT was conducted in patients with metastatic disease, and the company has yet to fully test Abraxane in earlier stages of pancreatic cancer, so there may be additional upside.

In addition to pancreatic cancer, Abraxane has shown compelling phase 2 data in malignant melanoma, and mature phase 3 overall survival data is expected this year. We think of Abraxane as an older product with a new lease on growth. And because the manufacturing infrastructure is already in place, we expect this drug to generate beneficial economy of scale for Celgene.

TLSR: Pomalyst is a second-line therapy being labeled for refractory multiple myeloma. When and if Revlimid quits working, patients go to Pomalyst. How is physician uptake?

MG: We have only had one quarter's worth of sales so far for Pomalyst, which was about $29M. We think the numbers are pretty good. Pomalyst would also be a second-line to Velcade (bortezomib; Millennium Pharmaceuticals Inc./Takeda Pharmaceutical Co. Ltd. [TKPYY:OTCPK]). Patients tend to be retreated with different cocktails of the same drugs, and this allows Celgene to get a bigger piece of the market that it is already in. There's a lot of leverage in that.

TLSR: You are following Galena Biopharma Inc. (GALE:NASDAQ). We've discussed it before. Tell me your growth theory.

MG: Galena's clinical program is immunotherapeutic, and the technology is peptide-based vaccines. It is developing NeuVax (nelipepimut-S or E75) to prevent recurrence of breast cancer in women who have been treated for early-stage disease and are at risk for recurrence. The company opened a 700-patient, phase 3 trial called PRESENT last year, and is enrolling patients now. Some interesting data have been generated around this vaccine from earlier studies, but obviously, it needs to demonstrate efficacy in the phase 3.

Galena has another peptide-based vaccine, folate-binding protein E39, which is in early development for ovarian and endometrial cancers that overexpress folate-binding protein (FBP). FBP is significantly overexpressed in the vast majority of ovarian and endometrial cancers, and to a lesser degree in breast, lung, colorectal and renal cell carcinomas. Because of limited distribution of FBP in healthy tissues, it is an interesting target.

Most recently, Galena purchased product rights to Abstral (fentanyl; sublingual tablets) from Orexo AB (ORX:STO). Abstral's target market is palliative care of patients with breakthrough cancer pain in the U.S. This provides Galena with the opportunity build a commercial organization, develop a marketing plan and begin to build relationships with oncologists, purchasers and payers.

TLSR: The sublingual narcotic Abstral doesn't really fit Galena's scientific model, but it fits the business model, with sales personnel set to get into the oncology office or clinic. Does this imply that the company wants to market NeuVax when and if the product is approved?

MG: I believe that Galena wants to capture as much value from NeuVax as possible for its shareholders. I wouldn't rule anything out at this point in time.

TLSR: The patients here are women who've had a brush with breast cancer, and you can imagine they are very motivated to prevent recurrence. I would presume that a great demand for NeuVax would exist if the product is approved. The immunization will occur in multiple doses. How much will it cost patients, or payers?

MG: Potential pricing for NeuVax has not been disclosed, and I imagine that Galena has and will continue to perform sensitivity analysis on pricing. But I would expect that if NeuVax is able to prevent cancer recurrence, particularly in distant spread of disease, it would alleviate a considerable burden on the healthcare system.

"The oncology market, broadly, has been a strong growth engine for the industry."

For example, take a drug like Kadcyla (ado-trastuzumab emtansine), which is sold by Roche and incorporates ImmunoGen Inc.'s (IMGN:NASDAQ) monoclonal antibody drug conjugate technology using Herceptin (trastuzumab) and a cytotoxic payload. Kadcyla was approved earlier this year for previously treated metastatic disease in women who have already received treatment with Herceptin. On an annual basis, the cost of Kadcyla is about $9,800/month, or more than $90,000/year. I think there's a big range of pricing that could occur with NeuVax, and it's a function of many factors.

TLSR: The phase 2 study of NeuVax did not show statistical significance. I understand that the phase 3 PRESENT study is designed a little differently, specifically to select out for the higher-risk patients where we should see some benefit if the product works. Could you talk about that just briefly?

MG: The standard of care for HER2-positive cancer changed during the NeuVax phase 2 study—namely, the use of Herceptin for the treatment of women with early-stage disease. Early studies also showed a waning immunological response over time. The program was amended to include booster dosing in women who were more than six months from completion of the initial vaccination schedule. Of the original group of patients receiving the vaccine in the phase 2 study, roughly half received booster doses at six-month intervals.

The work done so far does suggest there is activity with NeuVax, but a phase 3 trial is necessary to tease this out. There may be a level of risk that isn't quite the same as seen in other clinical programs, given the changing protocol. But we find the immunological response to NeuVax quite interesting, as well as data that seems to point in the right direction.

TLSR: When could we be looking at approval of NeuVax. Does 2017 sound reasonable to you?

MG: It depends on the pace of enrollment and when the data roll out. 2017 might be the earliest, if enough events occur to get the trial to read out. That would be a best-case scenario.

TLSR: Investors have been focused on that NeuVax phase 3 trial, but since that study doesn't have a near-term catalyst, do you think that FBP or perhaps even the investigator-sponsored trial with NeuVax + Herceptin could move shares?

MG: The phase 2 NeuVax + Herceptin trial has an opportunity to read out sooner than the PRESENT study, and could be a catalyst for investors.

But we also think that enrollment updates for the NeuVax PRESENT trial, as well as for the FBP trial, are catalysts for Galena. Another piece is that the company is planning to relaunch Abstral in Q4/13, which provides a different sort of catalyst—one pegged to revenue generation.

TLSR: Can we move to another name? Pick one.

MG: We like NewLink Genetics Corp. (NLNK:NASDAQ.GM). It has a very interesting platform technology, and we like platforms because they hold the promise to be broadly applicable in the treatment of cancer.

NewLink is likely to have a milestone-type event in H2/13, with the passage of the first interim analysis for the phase 3 clinical trial for HyperAcute Pancreas (algenpantucel-L). Our expectation is that the trial will continue, though there is early-stopping criteria incorporated into the program. NewLink is also developing the HyperAcute platform for NSCLC, and a phase 2b/3 trial with an adaptive trial design is getting underway. In addition, there is a HyperAcute melanoma program.

NewLink also has a small molecule program in development, exploring inhibitors of indoleamine (2,3)-dioxygenase (IDO). IDO is an enzyme that plays a role in regulating immune response by suppressing T-cell function, and is one way by which cancer grows undetected by the immune system. IDO is considered a checkpoint inhibitor.

Many different tumor types overexpress IDO, including prostate, colorectal, pancreatic, cervical, endometrial, lung and bladder, among others. The company had a lead molecule, indoximod, in early studies at the completion of its initial public offering, but we think this molecule and the broader IDO program have been largely overlooked until recently. NewLink is currently conducting a phase 2 study of indoximod in combination with docetaxel for the treatment of advanced breast cancer, and is in a phase 2 investigator-sponsored study in combination with Dendreon's (DNDN:NASDAQ) Provenge for metastatic prostate cancer. The company has a second IDO inhibitor compound, NLG 919, for which an investigational new drug application (IND) could be filed before the end of 2013. We like to think about these as competitive small molecule immunotherapies.

TLSR: When you and I spoke back in July 2012, you mentioned a smaller name, Oncothyreon Inc (ONTY:NASDAQ), which plunged back in mid-December when its NSCLC vaccine failed to meet its endpoint. Could you address it?

MG: Oncothyreon is a name that we have struggled with. The company's pipeline has not advanced, and that is the primary reason behind the decline in valuation. The phase 3 START trial, examining Stimuvax (now L-BLP25) in NSCLC, did not meet its clinical endpoint, and the company's studies for PX-866 (an inhibitor of the PI3 kinase [PI3K]/PTEN/AKT signaling pathway) have, thus far, not generated phase 2 data that warrant further clinical trials.

There are still additional studies to read out for PX-866, and while START did not meet overall signficance, it did produce some interesting subgroup analysis based on stratifications prespecified in the trial. We have yet to learn what Merck KGaA (MKGAY:OTCPK), the licensee of that product, will do with this candidate.

With those two programs devalued, Oncothyreon licensed a small molecule candidate, ARRY-380, from Array BioPharma Inc. (ARRY:NASDAQ), which allows the company to redirect the resources originally intended for the PI3K inhibitor program to ARRY-380, an orally active, reversible and selective small-molecule HER2 inhibitor, for the treatment of HER2-positive breast cancer.

Oncothyreon is a less straightforward story. Its valuation is particularly inexpensive with a $105M market cap, and that's what keeps us interested.

TLSR: You have initiated on Verastem Inc. (VSTM:NASDAQ), which is targeting cancer stem cells (CSCs). Can you tell me your growth theory here?

MG: We like Verastem for a couple of reasons. It has underpinnings of amazing technology and executive management. The scientific foundation of Verastem is based on collaborative work performed by Dr. Robert Weinberg, Dr. Eric Lander and Dr. Piyush Gupta. Of the three, Dr. Weinberg is probably the most well known to the investment community, having been credited for the discoveries of the first human oncogene, the ras gene and the first tumor suppressor gene, the Rb gene. Collectively, doctors Weinberg, Lander and Gupta discovered a biological process that endows cancer cells with properties that facilitate resistance, invasion and mobility, transforming them in to cancer stem cells, or CSCs.

Additionally, the company's scientific advisory board is an interdisciplinary group of experts from the broad reaches of oncology drug development, including basic science, translational medicine and medical therapy.

The company went public in 2012 with a pipeline of preclinical compounds, which is a rarity in the biotechnology sector. Typically, issues will have candidates in human clinical trials. We think Verastem's ability to make the transition from private to public without a product candidate in human clinical trials is a testament to the science and personnel at the company. The ability to finance shows the strength of the company's scientific and technical base.

But the company then did something that changed the development timeline—in the summer of 2012, Verastem acquired a compound that has activity against a cancer stem cell target, and this action accelerated the company's development pipeline into a phase 2 study. This study, targeting the focal adhesion kinase (FAK) for the treatment of pleural mesothelioma, appears to be sufficient to be considered a registration study based on its design and endpoints. While we love the scientific underpinnings of Verastem, we also feel that a large part of the investment appeal is an underappreciated commercialization opportunity resident within the company.

TLSR: What about the catalysts? What and when?

MG: The next catalyst for Verastem will be the dosing of its first patients with VS-6063, now called defactinib, for treatment of mesothelioma. This will be a phase 2, randomized, double-blind, placebo-controlled, multicenter study, and its start will certainly be a catalyst for Verastem. The primary endpoint will be overall survival, and the trial incorporates an adaptive design based on biomarker analysis.

In addition, Verastem plans to conduct a bridging study in Japanese mesothelioma patients in Q3/13, and initiate a phase 2 program with defactinib in K-RAS mutant NSCLC as well. We expect the first update—a function of the adaptive trial design in mesothelioma—to occur in H1/14, as well as data from the bridging study in Japan from the NSCLC phase 2 study in 2014.

The company has just initiated a phase 1 study of a second compound, VS-4718, which is also an inhibitor of FAK. It is being tested in solid tumors and data could be available in 2014. The company has another candidate, VS-5584, which is a PI3K/mTOR inhibitor, and plans to initiate a pilot study with this inhibitor in H2/13. These will be very focused clinical milestones, but along the way we would anticipate other events, such as publications, presentations and the like.

TLSR: Are all three of the compounds targeting cancer stem cells?

MG: They all target cancer stem cells, but via different pathways. Decatinib is a FAK inhibitor, as is VS-4718. VS-5584 is an inhibitor of the PI3K/mTOR pathway, which also is a known cancer stem cell pathway.

TLSR: Mara, is there anything else that you wanted to talk about?

MG: I would like to talk about Celldex, which I mentioned earlier. You and I also discussed this company when we were focusing on catalysts for The Life Sciences Report Watchlist 2013.

Celldex shares have experienced valuation expansion such that the shares can now be considered mid cap as opposed to small cap. With such a transition come new investors who might not otherwise have been able to invest in a smaller-cap company. We think the fundamentals of an evolving late-stage pipeline support the attractiveness of the shares.

TLSR: What might be a catalyst, per se?

MG: Celldex has a fair number of clinical catalysts through the second half of this year. We think an important inflection point could from rindopepimut, a cancer vaccine against a particular form of glioblastoma multiforme, a kind of brain cancer. While there is a phase 3 trial underway in newly diagnosed disease, called ACT IV, the company is also conducting a phase 2 program called ReACT, looking at patients who have had a recurrence of disease.

What is significant about ReACT is that, with a positive outcome, Celldex could use the trial as a registration study, and that could change the near-term commercialization picture. Recurrent disease is difficult to treat, so we have not incorporated success in our valuation model. We think there is upside to a positive trial outcome.

The second important clinical catalyst for Celldex is CDX-1127. This compound is in phase 1. CDX-1127 is particularly interesting because there is some thought that it has downstream effects on CD40. CD40 is involved in the regulation of immune response, and is implicated in antitumor immunity. It is a very interesting molecule, and we think little attention is being paid because it is a phase 1 compound.

We also think that the dosing of triple-negative breast cancer patients in the CDX-011 phase 3 trial, expected sometime in H2/13, will be a catalyst.

The last catalyst, which is a potentially big one, is for CDX-1135, for use in dense deposit disease (also called DDD), a very rare orphan drug indication. Dense deposit disease refers to the dense changes that occur in the glomerular basement membrane of the kidney that prevent proper waste filtering of blood. These deposits cause disruption in kidney function, and ultimately lead to kidney failure, requiring transplantation and dialysis. These deposits can also occur in the eye. Because of the nature of the disease and the small number of patients that are affected—two or three per million, the company could have data on one or two patients by the end of 2013. This could demonstrate the potential viability of this candidate.

TLSR: I've enjoyed this, Mara. Best wishes to you.

MG: Thank you, George.

Mara Goldstein joined Cantor Fitzgerald & Co. from Thomson Reuters, where she served as director of research for Reuters Insight. Goldstein was initially responsible for the firm's healthcare research practice, and later assumed responsibility for all research activities and sectors. Prior to that, Goldstein was an executive director and senior pharmaceutical analyst at CIBC World Markets. At Cantor, Goldstein covers the biotechnology sector. Goldstein also worked at Alex Brown & Sons and CS First Boston. She holds a bachelor's degree in economics from Purdue University.

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DISCLOSURE:
1) George S. Mack conducted this interview for The Life Sciences Report and provides services to The Life Sciences Report as an independent contractor. He or his family own shares of the following companies mentioned in this interview: None.
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3) Mara Goldstein: I or my family own shares of the following companies mentioned in this interview: None. I personally am or my family is paid by the following companies mentioned in this interview: None. My company has a financial relationship with the following companies mentioned in this interview: Read the Cantor Fitzgerald legal statement. I was not paid by Streetwise Reports for participating in this interview. Comments and opinions expressed are my own comments and opinions. I had the opportunity to review the interview for accuracy as of the date of the interview and am responsible for the content of the interview.
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