Off to the Races with Seven Extraordinary Biotech and Medtech Companies: Jason Napodano

TICKERS: AVH; AVMXY, DND, CTH; CYNAF, NVIV, DRIO, CUR, OPK, ONVO, PBTH

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

Jason Napodano Senior Biotechnology Analyst Jason Napodano of Zacks Investment Research specializes in uncovering small biotech and medtech companies with good prospects for successful drug development. He relishes proven management capable of creative financing to move private equity into the public markets, and he does not shy away from tiny companies outside the U.S. that have extraordinary technology. In this wide-ranging interview with The Life Sciences Report, Napodano presents the elegant growth stories of seven small companies that could return magnificent gains to investors.


Companies Mentioned: Avita Medical Ltd. : Cipher Pharmaceuticals Inc. : Cynapsus Therapeutics Inc. : InVivo Therapeutics Corp. : LabStyle Innovations Corp. : Neuralstem Inc. : OPKO Health Inc. : Organovo Holdings Inc. : Prolor Biotech Inc.

The Life Sciences Report: Jason, you follow some companies that are not domiciled in the U.S. Are there special considerations or concerns that investors should be aware of before investing in stocks outside U.S. markets?

Jason Napodano: I cover a number of Canadian companies, one Switzerland-based company and one Israel-based company. Listing and liquidity are probably the two biggest hurdles that investors need to overcome with regard to companies outside the U.S.

The readers of Zacks Investment Research reports are primarily retail investors. The first hurdle for retail investors is finding a broker who allows them to buy a stock that is trading on the Toronto Stock Exchange (TSX) or the SIX Swiss Exchange.

Then there are tracking stocks. Some are company-sponsored, but others are Pink Sheet stocks not sponsored by the company. Retail investors have to be careful. You might want to buy a foreign-listed company that you can't acquire on its primary exchange, but there may be a U.S. OTC-listed tracking stock available. You have to make sure that you're buying company-sponsored stock—either the American depositary receipt or the tracking stock—and that you're not buying a vehicle set up to follow the stock price that, in the end, may not follow the price at all.

The third issue is differences in accounting and reporting standards. The U.S. is one of the few countries using generally accepted accounting principles (GAAP). There are different standards in Canada and Europe. Some European companies only report twice a year, as opposed to the U.S., which reports quarterly results. Financial statements may look a little different than what you see in the U.S. Investors need to be aware of how and when companies report. Those are nuances investors need to be aware of when they are looking at foreign companies.

TLSR: Are there arbitrage opportunities available when trading international stocks, or is this beyond the scope of the average retail investor? Can hedge funds do it?

JN: Hedge funds probably could do it, because they can get in and out quickly and they have access to information that could allow them to play that arbitrage. However, I'm a pretty big believer in efficient markets. Individual investors may or may not be smart, but the market as a whole is pretty smart. If you have a stock listed on the TSX.V and the last trade there is $5.25, while the U.S. OTC stock is $5.15, you might be able to play some sort of arbitrage. What makes it difficult is the liquidity of the OTC. Can you short a stock on the TSX.V and long the same stock on the OTC? Does your broker allow you to do that? A lot of retail investors are not going to participate in this type of trading, but I wouldn't doubt that there are hedge-fund guys out there who try to capture that arbitrage.

TLSR: Jason, what about a reverse merger? You're familiar with this mechanism. Tell me what it is and why it is done.

JN: In a reverse merger, a private company acquires a public company. In most instances the public company has failed—either filed for bankruptcy or gone out of business—and its assets are substantially gone. But the public company provides the private company with a way to go public via the ticker symbol. When these private companies do become public, they typically change the ticker symbol because they may not have acquired a company in their industry. A reverse merger may involve a failed real estate investment trust (REIT), Internet venture, retailer or oil and gas explorer. The deal is done, and the new company is trading on the OTC.

TLSR: Does the Securities and Exchange Commission (SEC) require an S-1 in a situation like this, when a private company is obviously just acquiring the ticker symbol that it's going to change?

JN: Yes, it does. There are some regulations, but fewer than in the typical initial public offering (IPO) process. You asked why a company would do this. It's a heck of a lot faster and less expensive. If you're a small, private biotech company and you don't have the capital—or maybe the trial data or the awareness—to do a fully marketed IPO with a big-name investment bank, you can get a reverse merger done in three to four months at significantly lower cost. An IPO may take 6–9 months or longer, and costs more money.

When a company goes public via a reverse merger, management typically controls the process and doesn't give up ownership to the public markets immediately. The reverse merger allows management to bring the company public and then decide the terms and timing of the S-1 filing and the initial financings, as opposed to coming public via an IPO, where ownership of the company is offered on day one. Many private companies go public via a reverse merger with the founders, management and bankers owning 90% of the shares; they only float whatever they need to acquire the company, which may be less than 10% of the shares. Typically what happens is the stock begins to trade, and then management files an S-1, registers the rest of the shares and raises capital.

TLSR: I surmise that these reverse merger deals are vehicles that private equity people use to create shareholder value more quickly in the public markets. Is that indeed the case?

JN: Absolutely. Sometimes reverse mergers get a bad rap because there is not a lot of information available to investors, and not a lot of liquidity. I sift through the murky water that surrounds reverse mergers and try to pick out the gems. Investors may have to look at 20 before they find one worth investing in, but if they can find one, they can get in early on what is potentially an enormous opportunity.

TLSR: Let's go ahead and talk about some of your ideas. Go ahead.

JN: Since we're talking about reverse mergers, I'll talk about two that have been successful. I'll start with InVivo Therapeutics Corp. (NVIV:OTCBB), which I started covering in Q3/11 at $0.64. It is now about $5.50/share. InVivo has a biodegradable scaffolding for patients who have experienced acute spinal cord injury. The company came public without a lot of fanfare. It had private equity backing, and it had a lead investor. I got involved with the story and loved it.

TLSR: The reverse merger, in this case, saved time and was capital-efficient. Is that part of the story here? What made InVivo a candidate for reverse merger?

JN: The reverse merger was a value creator here. InVivo was a good candidate for the technique because it had preclinical data but didn't have a data package that would generate interest among big investment banks for an IPO. The company needed to come public to raise money more efficiently. After its initial financing, InVivo was able to generate additional data and control that process better. New data have come out since the merger, and that has sent the stock up six-fold. A lot of investors would shy away from a situation like InVivo's, but there was incredible value and opportunity here, and it's proved out a winner.

TLSR: InVivo Therapeutics' technology platform is a biodegradable polymer scaffold that you can seed with human neural stem cells. You could also put medication, like a corticosteroid, into the scaffold. Where are we now in this project's development, and what are the catalysts?

JN: The company just received U.S. Food and Drug Administration (FDA) approval to begin its first human clinical study with the device alone. It will not be seeding the scaffold with cells, and no drug is eluting off it.

"If you're a small, private biotech company and you don't have the capital to do an IPO with a big-name investment bank, you can get a reverse merger done in three to four months at significantly lower cost."

This trial should start sometime in Q3/13. It will be a small pilot study—a humanitarian use device (HUD) study, which means that if the company can generate good safety and feasibility data, it could get its device on the market with just that study. Acute spinal cord injury patients are a small population, and nothing is available for them. If the study goes well, the company can file a humanitarian device exemption (HDE), which is essentially the medical device equivalent of an orphan drug designation.

For medical devices, you don't do phase 1/2/3 studies, as you would in drug development. You do what's known as an investigational device exemption (IDE) or a pilot study, which is the equivalent of a phase 1. Next is a premarket approval (PMA) study, which is essentially the equivalent of a phase 3 trial. InVivo is doing the equivalent of an IDE; it's called an HDE because of the orphan indication. The company could get to market with the scaffold platform for the orphan indication if results are positive. To expand the label into much larger populations, InVivo would have to go back and do a PMA trial.

TLSR: With this HDE, there has to be some clinical benefit derived from the scaffold, even without seeded cells or a drug-eluting process. What possible efficacy could come out of just the scaffold alone?

JN: Under the HDE guidelines, you must demonstrate safety, no harm. There's not a lot to demonstrate in terms of efficacy.

In terms of what the device can do by itself, the company talks about the body's response to acute spinal cord injury, inflammation, which gives rise to scarring. This can push on the spine, causing long-term damage and paralysis. InVivo's procedure involves stabilizing the spinal column with pedicle screws, and then the customized scaffold is cut and placed over the wound in the spinal cord. Cutting and placing the scaffold takes only a few minutes and is done by the spinal surgeon during the stabilization procedure. It's very efficient, and so far looks to be very safe.

The device is designed to act as a shield and protect the spinal cord from the body's own inflammatory process. As the body's inflammatory response subsides, the scaffold degrades, and you don't get that secondary scarring and injury that InVivo believes is the cause of the majority of the paralysis.

TLSR: Who will be the patients? What is their status coming into this study?

JN: Patients have what's known as an AISA-A (American Spinal Injury Association classification) injury—complete paralysis with no motor or sensory function at or below the injury. Safety will be the primary endpoint, with ASIA impairment score data as secondary endpoints. If paralysis and loss of sensation are complete, any improvement should be attributable to the device, because typically these patients do not progress. If the company can say, "We protected that patient from further damage," or is able to show touch sensation on pinprick testing, improved bladder function, or improved lung function, it will be attributable to the scaffold device. That will have to be confirmed in PMA pivotal trials, but the orphan indication allows the company to expand on that initial ASIA-A population by selling the product on the market. One final thing worth noting: The trial is with only five patients and open-label in design, so we should start to see data shortly after the trial starts.

TLSR: Jason, there was another reverse merger company that you wanted to talk about. What is it?

JN: It's Organovo Holdings Inc. (ONVO:OTCPK). The reason I like to talk about InVivo and Organovo together is because the same private equity team, banking team and lead investor brought both companies public.

Organovo is developing a three-dimensional (3-D) bioprinter. It came public in early 2012, which is when I started coverage, and it was trading at $1.55/share. Today, it's more than $6.30. The business development team has another home run, a fourfold win, with Organovo. On July 12 the company uplisted from the OTC to the NYSE.MKT. This listing on a major exchange allows for greater visibility and credibility with institutional investors. The stock nearly doubled on this news. This is the ultimate kind of success for a reverse merger.

TLSR: Anywhere you turn today, you hear about 3-D printing. What is Organovo doing with it?

JN: Organovo is developing a bioprinter to create 3-D biological structures for use as bioassays. Other companies are collaborating to see what can be accomplished with these 3-D bioassay structures.

TLSR: The company is developing a 3-D liver model as a hepatic toxicity screen, presumably for drug developers. Is that its lead development project? Is the idea that a 3-D liver structure will have more of a liver organ phenotype than hepatic cells floating in suspension or in a dish?

JN: Yes. When you plate what's known as two-dimensional (2-D) liver cells in a dish, they begin to stop functioning as liver cells within 24 hours. You start with a dome-shaped cell in the dish, and within 24 hours it begins to flatten out and lose interaction. If you're a large pharmaceutical company and want to test a drug for liver toxicity, you only have the ability to test on viable liver cells for those 24 hours. With Organovo's 3-D liver cell assay, you can build structures that are 20–25 cells high. Because of this more phenotypic shape, the liver cells still behave like in vivo liver cells at five days. Because they are surrounded by each other, they build connective junctions and work in concert to produce enzymes and perform other normal functions of liver cells.

TLSR: This sounds like a premium-priced product line. What is the value proposition for the pharma customer?

JN: Organovo's process is to create a simple assay kit built in tiny microwell plates, ranging from 24, 48 or 96 wells per kit. Each microwell will contain little 3-D livers, which it then sells to pharmaceutical companies. Drug developers can use Organovo's product to get up to 144 hours of data, and not just the 24 hours that 2-D assays allow.

"When a company goes public via a reverse merger, management typically controls the process and doesn't give up ownership to the public markets immediately."

Using Organovo's product, a pharma could protect itself from spending a lot of money to bring a drug to phase 3 only to have the drug fail because of liver toxicity—or worse, bring a drug to market and then have it pulled off the shelf because of toxicity. From a pharmaceutical company's standpoint, it's a risk-reward calculation. It may cost more to buy these assays from Organovo, but one phase 3 trial could cost $25–50 million ($50M). There is a real value proposition here, and I think Organovo can start generating revenues as soon as next year.

TLSR: What about using different cell types to build other organ models as toxicity screens?

JN: Liver toxicity is the No. 2 largest reason for drug failure. The No. 1 cause is cardiovascular toxicity. But this first development project at Organovo makes sense, because liver tissues can be grown, and liver is the only organ that can regenerate itself. Starting with liver, the company can map out the process and gain traction with its first product offering. Then it makes sense to create 3-D lung, kidney and cardiac assays.

In recent conversations, the company has talked about building oncology models as well. It has been expanding its research capabilities in this area, having signed collaborations with ZenBio Inc., the Knight Cancer Institute at Oregon Health and Science University, and even software maker Autodesk Inc. I can't tell you what the next product will be, but I know Organovo wants to create a suite of biological toxicity and oncology assays that it can take to large pharma. It will definitely expand once it qualifies the process.

TLSR: Your next thought?

JN: I have mentioned the banking team, the private equity group and the investors who brought Organovo and InVivo public. What I didn't mention is that this team brought another company public, Prolor Biotech Inc. (PBTH:NYSE), which I do not cover. Just to give you a sense, Prolor was less than $1/share in 2009, and it was recently acquired by OPKO Health Inc. (OPK:NYSE) for more than $6/share, more than $500M. These guys know what they're doing. I've found a banking team and a private equity group that is three-for-three in grand slams. Now that team, headed up by Adam Stern at SternAegis Ventures, has brought a fourth company public, called LabStyle Innovations Corp. (DRIO:OTCBB), which I want to talk about.

TLSR: Clearly you love the pedigree. Tell me about the company.

JN: Diabetics must carry around a glucometer, a lancing mechanism, test strips and usually a needle and insulin. If you're female, you might just throw it all in your bag. If you're a guy, you might put it all in a little satchel. LabStyle has developed a product called Dario, which is the size of a stick of gum or a lighter and includes three mechanisms in one—the test strips, the glucometer and the lancing mechanism. The Dario ties into your smartphone. There are other products on the market that are similar, but they connect through the phone's charging dock, and different smartphone brands have different charging docks. The Dario hooks in through the headphone jack, which is universal. It's truly an elegant design, and LabStyle looks like another Prolor, InVivo and Organovo.

TLSR: Where is Dario in development? What is the regulatory pathway?

JN: LabStyle is an Israel-based company, so it started overseas. It filed for approval and is currently awaiting the CE mark, which is clearance for sale in the European Union. The company says it is on track for that approval in the next couple of months. Approval is about nine months or so behind that in the U.S., and I would expect the company's 510(k) clearance for the U.S. market to come at some point next year. I encourage people to go to mydario.com and check out this device. The company just raised money, and is signing distribution partners. It is putting the pieces into place so that, as soon as it gets the CE mark, it is off to the races in Europe. With 510(k) approval next year, it's off to the races here in the U.S. I think this Dario product is a winner. It's a very clean story in a huge market.

TLSR: We talked about foreign investment earlier, and you seem very comfortable with it. I know you follow Canadian companies too. You've done quite well with one called Cipher Pharmaceuticals, Inc. (DND:TSX), haven't you?

JN: Yes. Cipher is based in Mississauga, Ontario, and I initiated coverage on it at $0.96 two years ago. The stock is at around $6.60 today. I'm up almost 650% on this small Canadian pharmaceutical company. I'm the typical U.S. investor—skeptical of any company that is not in the U.S.—but I dug into this name and found it sitting on a potential gold mine.

Cipher has an improved formulation of isotretinoin, which is the most commonly used prescription acne medication. It licensed the drug to Ranbaxy Laboratories Ltd. (RBXLY:OTCMKTS), which markets the product as Absorica. Cipher markets the product as Epuris in Canada. The sales are unbelievable, and it has only been on the market for a couple of months. Every time I see the IMS Health numbers, they exceed my expectations. I just put out a note to Zacks investors and titled it, "Yes, I'm Raising my Estimates on Cipher Again." It has been a wonderful growth story—a true diamond in the rough.

TLSR: Jason, you just recently initiated coverage on a couple of companies. Could you talk about them?

JN: One is an Australian biotech, a regenerative medicine company. The other is a small Canadian biotech. The first is Avita Medical Ltd. (AVH:ASX; AVMXY:OTCQX), listed in Australia and the United Kingdom. It has a regenerative medicine device product called ReCell Spray-On Skin (autologous cell suspension), a treatment for severe skin defects, burns, scalds, scars, vitiligo (discoloration of the skin when melanin is lost), acne scarring and what I call the holy grail of indications, chronic wounds. I like this product because it makes sense for burns and scalds, especially in children. It's a difficult story to communicate—the pictures at the company's website are worth a thousand words.

"Investors may have to look at 20 reverse mergers before they find one worth investing in, but if they can find one, they can get in early on what is potentially an enormous opportunity."

Avita's ReCell product is fantastic because all that is needed to generate the suspension is skin from a donor site about the size of a postage stamp, instead of a huge harvest that creates another large wound, and two painful sites that need to heal. With ReCell, a skin biopsy is put in the ReCell kit, which separates and isolates the patient's skin cells through an easy-to-follow, six-step process. The cells can then be sprayed onto the wound and a new layer of healthy skin grows.

TLSR: This product already has an approval, doesn't it?

JN: It's approved in Europe, but things in Europe are a little different with respect to marketing medical devices. Companies file for approval of a medical device without specific use claims. Once approved, the European Medicines Agency (EMA) asks for post-marketing studies to clarify specific uses of the approved device. These post-marketing studies then serve as the basis for gaining insurance reimbursement. In the U.S., ReCell is essentially in phase 3, and we'll get data from that trial next year. Then Avita will file for approval, and ReCell should be on the U.S. market in 2015.

From a burns and scalds standpoint, ReCell is going to dominate the market. From an acne scarring or vitiligo standpoint, I think it can capture significant market share. For chronic wounds, venous stasis ulcers, diabetic foot ulcers and similar conditions, I need to see more data on how ReCell fares compared to the market-leading products, such as Dermagraft (human fibroblasts on a dissolvable mesh material; Shire Plc (SHPGY:NASDAQ; SHP:LSE), Apligraf (graft skin; Organogenesis Inc. [private]) or EpiFix (biologic human amniotic membrane; MiMedx Group, Inc. (MDXG:OTCBB)).

TLSR: You have to obtain cells from the biopsy, but you can't macerate the tissue when you are processing it because you must have vital cells in suspension to spray onto the wound. How are the ReCell's harvested cells processed?

JN: The cells must be aggregated to break them up, but you have to be gentle enough not to kill them. With ReCell, the process is done with a mini-medical device right on site, and takes about a half hour. The kit comes with all the necessary buffers and reagents. No harmful chemicals or growth hormones are added to the cell suspension. The suspension contains all of the skin cells necessary to promote healthy skin growth: skin stem cells, keratinocyte cells that form the basis of healthy skin, fibroblasts that produce collagen-providing texture and fullness, melanocytes that produce skin color (melanin).

The cells in the ReCell spray have shown viability in studies that compares to that of non-disrupted cells. The product has shown healing rates comparable to allogeneic (same species donor) products. I like ReCell because it's autologous, meaning that it comes directly from the patient. If a patient has vitiligo, a clinician can obtain a biopsy from right below the vitiligo, process and spray. The same with a burn. And there are no supply issues.

TLSR: What is the business model? Is there a license fee paid to Avita for every use of the processing device?

JN: Almost all medical devices now follow the razor-razor blade model. You buy the device, a "lab-in-a-box" modular system, from Avita, and then you buy the single-use consumable sets. When you run out of spray bottles or kits, you go back to Avita and buy more consumables as needed.

TLSR: I'm noting this 106-patient burn trial (NCT01138917) says that pivotal data completion will be accomplished by mid-2014. When can we expect to see those data?

JN: I would say pretty close to that time. The trial was 85–90% enrolled in March 2013. My guess is that it's fully enrolled by now, and that investigators are waiting on patients to heal and get the evaluations. As soon as that last patient hits the endpoint, the company should be able to package and present data pretty quickly. I expect data by the middle of 2014, and then an application, at least for burns and scalds, will go to the FDA shortly after.

Outside of burns and scalds, we recently got information from the company on the initiation of a 65-patient trial studying ReCell's effectiveness in treating chronic venous leg ulcers. The trial, called RESTORE, will take place in Europe and should help expand use and reimbursement of the device. If you look at the company's preliminary data on ReCell in venous leg ulcers and diabetic foot ulcers, it is very encouraging. Chronic wounds represent a very large market opportunity for Avita. I am pleased to see clinical activity moving forward with ReCell.

TLSR: What about that other company that you just recently initiated on?

JN: Cynapsus Therapeutics Inc. (CYNAF:OTCQX; CTH:TSX.V) is a small Canadian company developing a sublingual formulation of a drug called apomorphine, which is a rescue medication for Parkinson's disease patients who experience something called "off time," a rigid or frozen state that occurs when their levodopa + carbidopa medication has worn off. This typically happens in the morning, because the Parkinson's patient takes his or her last dose of medication before bed, and it has worn off by the time they awaken.

Apomorphine is a rapid rescue medication for that "off time" state. An injectable formulation of this product, called Apokyn (U.S. WorldMeds [private]), is on the market. It is absolutely the right drug because it works. But Apokyn is completely wrong for a patient who has dyskinesia (uncontrollable movements), because the patient can't administer his or her own drug subcutaneously with shaking hands. Neither is it feasible for patients in "off" episodes—who are frozen—to give themselves a shot. Oral tablet formulations of apomorphine have failed because they get deactivated by the liver on first-pass metabolism. Cynapsus' sublingual film formulation of apomorphine is put under the tongue and dissolves in a very short period of time. Patients with dyskinesia can take it themselves. It just makes sense.

The regulatory route for this reformulated drug is through the FDA's 505(b)2 pathway because the drug is already on the market, so Cynapsus doesn't have to generate a lot of efficacy data. It has go to the FDA with similar blood plasma bioequivalence and biomarker data as for the injectable apomorphine, to show the new formulation gets the drug into the blood at the right concentration. The company also has to demonstrate long-term safety, but we see this as a relatively low hurdle given the history of Apokyn.

TLSR: If this sublingual formulation of apomorphine is effective as a rapid rescue, how much could it be worth?

JN: I think it could be a $500M or more opportunity. Parkinson's is an enormous indication, and almost all Parkinson's patients experience this on/off phenomenon. Apokyn was an orphan drug, protected for seven years, so no one has been able to develop an additional formulation. As soon as Apokyn came off orphan protection, Cynapsus got the patent for the sublingual formulation. This is a very clean, low-risk story, and I love the way Cynapsus has pulled this off, being the first to patent this delivery.

If this were a U.S. stock, it would be four or five times the price it is now, but because it's in Canada, no one knows about it. Cynapsus reminds me a lot of Cipher when it was trading at $0.64 and no one was looking at it. All of a sudden, people started to take notice. That's where Cynapsus is right now.

TLSR: When could Cynapsus' product be on the market?

JN: It could be on the market as early as 2016.

TLSR: This is a $17M market cap company. That would scare most people away, but you're clearly very passionate about it. Data are expected from a phase 1 trial in healthy human subjects in Q3/13. If data from the phase 1 study show the right levels of apomorphine in the serum, will that move these shares?

JN: I think so, yes. I don't think Cynapsus has a drug problem. I think it just has an awareness problem. The company is financed.

TLSR: You have written some quite detailed work on Neuralstem Inc. (CUR:NYSE.MKT) recently. Everyone thinks of it as a stem cell company, but you are very excited about another side of its technology, aren't you?

JN: Yes. Everybody knows Neuralstem as the company treating amyotrophic lateral sclerosis (ALS) patients with neural stem cells, directly injecting them into the spinal cord. It has just gotten an approval to use those same exact cells in a spinal cord injury trial. The lesser known part of the story is Neuralstem's phase 1 small molecule program with NSI-189, which is addressing a completely new mechanism of action for depression and other neurological disorders.

What excites me is that new discoveries of mechanisms of action and new targets for central nervous system (CNS) disease don't come around very often. Do I know that Neuralstem's phase 1 trial is going to work? No. . .but the fact that the company is working on something new in CNS, and also has interesting animal data with preclinical proof of concept, leads me to believe that if Neuralstem can show that this molecule is safe and if there are some signs of efficacy, there is going to be a lot of interest from big pharma. I don't want to oversell this, but if this NSI-189 molecule hits, it could be a tenbagger for the company.

TLSR: When are the phase 1 data expected?

JN: In Q3/13 or Q4/13. It's tough to peg the exact time. We're waiting for patients to enroll.

TLSR: I find it very interesting that Neuralstem's phase 1 trial with NSI-189 is only a 24-patient study, yet it is double-blind. Also, the idea with this drug is to stimulate neo-neurogenesis in the hippocampus. This is a tall order, isn't it?

JN: The belief is that hippocampal atrophy starts the major depressive disorder—or Alzheimer's disease, schizophrenia, bipolar disorder, anxiety disorder. It's the destruction of the hippocampus that leads to those indications.

For Alzheimer's disease, a lot of researchers have spent a lot of time looking at beta amyloid and plaque formation. We are starting to get the sense, in some parts of the scientific community, that beta amyloid and plaque are a result of the disease, not a precursor to the disease. The precursor to the disease could be hippocampal atrophy. Neuralstem's NSI-189 is designed to protect the hippocampus, stabilize it and even grow it, so that patients never get to the point where they have major depressive disorder, Alzheimer's, anxiety or schizophrenia. Big pharma may come to Neuralstem and say, "Thanks for the phase 1 depression data, but we're going to take the next steps into anxiety, Alzheimer's or schizophrenia."

With regard to the size and design of the phase 1 trial—24 patients and double-blind—Neuralstem specifically ran a larger-than-usual phase 1 program so that it could generate enough data to attract a big pharma partner. The goal was to show the mechanism works. Where the pharma companies take it remains to be seen.

TLSR: Jason, this has been terrific. Thank you.

JN: I appreciate that. Thanks a lot, George.

Jason Napodano is senior biotechnology analyst for Zacks Investment Research. In 2009, Napodano was promoted to managing director of research for Zacks' Small-Cap Research division, which focuses on writing high-quality institutional research on underfollowed or undervalued small-cap stocks. Prior to his tenure at Zacks, Napodano spent three years on the buyside with Eastover Capital in Charlotte, N.C., where he focused on large-cap equities and specialized in healthcare, energy and technology. Prior to joining Eastover, Napodano worked as a research scientist for TechLab Inc., a biotechnology company focused on developing diagnostic kits and vaccines for infectious diseases. He also spent a year working in a lab at the Fralin Biotechnology Center, and a year working for a cancer researcher in Virginia. Napodano has a bachelor's degree in biochemistry from Virginia Tech, with an additional bachelor's degree in chemistry and a minor in math. He has a master's degree in business administration and finance, with a concentration in securities analysis, from Wake Forest University. Napodano is also a Chartered Financial Analyst (CFA).

Want to read more Life Sciences Report interviews like this? Sign up for our free e-newsletter, and you'll learn when new articles have been published. To see a list of recent interviews with industry analysts and commentators, visit our Streetwise Interviews page.

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.
2) The following companies mentioned in the interview are sponsors of The Life Sciences Report: Neuralstem Inc., Cynapsus Therapeutics Inc., Avita Medical Ltd., OPKO Health Inc. Streetwise Reports does not accept stock in exchange for its services or as sponsorship payment.
3) Jason Napodano: 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. Zacks has or seeks to have a financial relationship with the following companies mentioned in this interview: InVivo Therapeutics Holdings Corp., Organovo Holdings Inc., Prolor Biotech Inc., OPKO Health Inc., LabStyle Innovations Corp., Cipher Pharmaceuticals Inc., Avita Medical Ltd., Cynapsus Therapeutics Inc., Neuralstem Inc., Shire Plc, MiMedx Group Inc., Ranbaxy Laboratories Ltd. 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.
4) Interviews are edited for clarity. Streetwise Reports does not make editorial comments or change experts' statements without their consent.
5) The interview does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility. By opening this page, each reader accepts and agrees to Streetwise Reports' terms of use and full legal disclaimer.
6) From time to time, Streetwise Reports LLC and its directors, officers, employees or members of their families, as well as persons interviewed for articles and interviews on the site, may have a long or short position in securities mentioned and may make purchases and/or sales of those securities in the open market or otherwise.




The Life Sciences Report The Life Sciences Report