Dr. June Almenoff

Ohr Pharmaceutical (OTCQB: OHRP)has appointed Dr. June Almenoff to the board of directors.

In a statement, CEO Dr. Irach Taraporewala said Dr. Almenoff offers tremendous expertise in the areas of pharmaceutical research and development, risk management, and product licensing.

“We look forward to working with June as we continue to achieve significant corporate milestones, including the halfway point in enrollment of the Squalamine eye drop Phase 2 clinical trial and our proposed listing on NASDAQ, both expected by mid-year.”

Dr. Almenoff said ”Ohr has a promising pipeline and excellent team, and I look forward to working with the board to help drive the company toward future success.”

Dr. Almenoff is president and CMO of Furiex Pharmaceuticals (NASDAQ:FURX). She also serves as the company’s principal executive officer and a member of the board of directors.

Aradigm has completed Phase 2b clinical trials in BE patients with Pulmaquin and Lipoquin.

Under the accord, the companies have agreed to advance Aradigm’s proprietary inhaled ciprofloxacin formulations into Phase 3 clinical trials in BE, with Grifols responsible for all development and clinical expenses up to a maximum of $65-million for the BE indication.

Aradigm is entitled to receive up to $25-million on achievement of development milestones and is in line for tiered royalties on worldwide sales of products utilizing Aradigm’s proprietary inhaled ciprofloxacin formulations. Grifols also will be granted an option to license Aradigm’s AERx pulmonary drug delivery platform for use with another molecule.

Subject to approval of Aradigm’s shareholders, Grifols will acquire 35% of Aradigm’s common stock on a fully diluted basis at a price of 12.4 cents a share or about $26-million.

Existing Aradigm shareholders, including Tavistock Life Sciences and accounts managed by First Eagle Investment Management, and new investors, Great Point Partners, will co-invest in the transaction and purchase an additional $15.4-million in Aradigm common stock.

Closing is set for the second half of 2013, when Grifols will be entitled to designate two directors to the Aradigm board.

“Grifols is a great fit for us, with their global reach of respiratory physicians treating a number of conditions that could benefit from Pulmaquin,” Igor Gonda, president and CEO of Aradigm said in a statement.

Ramon Riera, president of Global Commercial for Grifols, said Pulmaquin will complement the company’s existing Prolastin-C business. “There is significant overlap between Bronchiectasis and Alpha1 physicians and patients. When launched, Grifols will leverage its existing pulmonary sales team to commercialize Pulmaquin,” he added.

Justin Renz

“There hasn’t been a lot of innovation in the pain space in many years, and we are one of the few companies exploring multiple novel mechanisms to address pain,” Justin Renz, EVP and CFO, says in an interview with BioTuesdays.com. “We have an opportunity for a breakthrough pain medication.”

Mr. Renz explains that Z160 is an oral, state-dependent, N-type calcium channel blocker that is designed to target and modulate only those neurons transmitting pain signals—specifically neurons that are undergoing high frequency firing.

“Preclinical and clinical data to date with Z160 suggest that this state-dependent mechanism of action may avoid the severe psychiatric and neurological side effects of existing drugs and compares well with other drugs used to treat neuropathic pain in terms of efficacy,” he adds.

Specifically, he points out that in animal studies using the spinal nerve ligation model of neuropathic pain, Z160’s efficacy was on par with:  ziconotide, which is sold as Prialt by Jazz Pharmaceuticals; gabapentin, which is sold as Neurontin by Pfizer; and morphine. The company also has established a significant safety database with over 200 people, with no notable dizziness, somnolence or neuropsychiatric side-effects, he adds.

Z160: Compares Well With Drugs Used To Treat Neuropathic Pain

Zalicus’ new formulation of Z160, dosed at 375 mg twice-daily, generated a sixfold improvement in bioavailability and positive pharmacokinetics in Phase 1 clinical testing, compared with an earlier formulation developed by Merck at 1,600 mg a day.  Merck returned the original formulation to a predecessor of Zalicus in 2009 after a Phase 2 trial, even though the compound had no serious adverse events.

Last September, Zalicus initiated a Phase 2 proof-of-concept trial to evaluate the safety and efficacy of Z160 as a treatment in reducing chronic lower back pain, or lumbosacral radiculopathy (LSR). LSR is a common neuropathic pain condition resulting from the compression or irritation of the nerve roots exiting the lumbar region of the spine.

Approximately 70 LSR patients, at some 20 sites, will receive Z160 twice daily for six weeks, and 70 will receive placebo. The primary endpoint is the change in weekly mean pain score on an 11-point neurologic rating scale. Top-line data from the study is expected in the fourth quarter this year.

In January 2013, Zalicus began enrolling a separate Phase 2 proof-of-concept trial with subjects with postherpetic neuralgia (PHN), a chronic state of neuropathic pain resulting from an outbreak of the herpes zoster virus, which is otherwise known as shingles. Top-line data are due to be released in the fourth quarter this year.

The six-week, double-blind, placebo-controlled study is expected to enroll 140 subjects at over 40 sites in the U.S. The primary objective of the trial is to evaluate the efficacy of Z160, compared with placebo in reducing pain in subjects with PHN as measured by a change in an average weekly pain score.

“Postherpetic neuralgia is an important medical condition for evaluating the activity of Z160 for several important reasons,” Mr. Renz says. “It is a well-recognized standard for establishing clinical proof of concept in neuropathic pain, and, with a prevalence of less than 200,000 patients in the U.S., it has the potential for orphan drug status and could be a feasible first indication to pursue from a commercial perspective.”

“There is a significant unmet medical need for novel, targeted and more efficacious chronic neuropathic pain therapies, with improved safety and tolerability profiles, such as Z160,” he adds.

Few Novel Mechanisms Targeting Pain in Clinical Development

In a new research report, Zacks Investment analyst, Jason Napodano, writes that the two studies “have the potential to dramatically re-value Zalicus,” noting that LSR is an attractive market opportunity.  The prevalence of the condition is high, affecting 3% to 5% of the global population, and to date, there are no drug treatments specifically approved to treat it.

He figures that in LSR alone, Z160 could have a peak sales potential of $500-million or more, with a $250-million opportunity in PHN. Larger indications, such as diabetic peripheral neuropathy and fibromyalgia, may offer significant upside potential, depending on the outcome of pivotal trials, he adds.

Mr. Renz says the mechanism of action for Z160 has been validated by Jazz’s Prialt, which has well- documented clinical efficacy and is the only non-opioid synthetic peptide approved for the management of severe chronic pain in patients who are intolerant of, or refractory to, other treatment options such as morphine.

The problem with Prialt, he notes, is that it must be given through injections directly into the spinal canal so it reaches the cerebrospinal fluid.  The Prialt label also includes a “Black Box” warning that it may cause psychiatric and neurological impairment. Jazz sold only around $21-million of Prialt in 2012.

“If we can prove our oral, small molecule product can hit only those neurons transmitting pain signals, without the side-effects, we’d really have something; that’s what we believe we have with Z160,” Mr. Renz adds.

In addition to Z160, Zalicus is developing a T-type calcium channel blocker, Z944, for chronic or acute pain. It is also discovering Nav 1.7 channel blockers for chronic pain as part of a research collaboration with Hydra Biosciences to identify potential clinical candidates.

Mr. Renz says Z944 demonstrated efficacy in preclinical inflammatory pain models and successfully completed a Phase 1 single- and multiple-ascending dose trial in the U.K. at the end of 2012. “We also know that Z944 penetrates the brain, which is important for a potential pain drug.”

There are no approved T-type calcium channel blockers on the market, and Zacks’ Mr. Napodano describes Z944 as representing a “potentially new and revolutionary way to treat acute pain.”

The company is consulting key opinion leaders in the field for ideas about the best chance of success with the molecule, and it hopes to conduct a proof-of-concept clinical study later this year. “We’re being deliberate to find the right dosing and right indication for this potent molecule,” Mr. Renz says.

Zalicus Pipeline: Focused on Pain and Inflammation

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Nadav Kidron

“We are very pleased to have the FDA clearance to proceed,” CEO Nadav Kidron said in a statement. “The upcoming trial is a major milestone for Oramed and we look forward to continuing to progress ORMD-0801′s clinical development in the U.S.”

ORMD-0801 is an orally ingestible insulin capsule indicated for the early stages of Type 2 diabetes, when it can still slow the rate of degeneration of the disease by providing additional insulin to the body and allowing pancreatic respite.

Orally administered insulin also has the potential benefit of enhanced patient compliance as well as the advantage of mimicking insulin’s natural location and gradients in the body by first passing through the liver before entering the bloodstream.

The study, conducted at Indiana University, clearly indicates that the majority of patients treated by Medigus’ endoscopic system SRS were able to stop taking daily PPl (drugs that have a long-lasting reduction effect of gastric acid production) in the third year after the procedure.

The three-year study follows five-year results of a pilot study of the SRS endoscopic system for the treatment of gastroesophageal reflux disease (GERD). The pilot study had been conducted at the Deenanath Mangeshkar Hospital and Research Center in India in 2007.

According to lead investigator of the pilot study, Dr. Amol Bapaye, the five-year follow-up results are similar to those produced by a laparoscopic-surgical procedure for the treatment of GERD in the same timeframe.

“Medigus has developed an innovative solution to treat GERD and we’re excited to present the excellent supportive data from our three-year study and five-year follow-up,” CEO Dr. Elazar Sonnenschein said in a statement.

“It is rewarding to watch patients that no longer need to rely on daily use of drugs to support their routine,” Dr. Sonnenschein said. “We strongly believe that our minimal invasive solution will replace many of the unnecessary surgeries which are performed today.”

The complete SRS™ system including the console, the endoscope and standard monitors (not included)

The endoscopic system SRS performs a minimally-invasive treatment of GERD, one of the most common chronic diseases globally. The system, which includes a disposable surgical endoscope, performs an anterior fundoplication in lieu of the current surgical procedure, without incisions and opening the abdominal cavity.

The SRS offers a treatment by an independent device that doesn’t require any additional components during the procedure. The operation is rather intuitive and can be performed by one surgeon or GI.  In addition, the procedure can be conducted in the endoscopy suite to ensure the saving of operation room time.

“We are very optimistic about the [near-term] and [long-term] potential of the technology as the system is already both FDA-approved and CLIA-waived, allowing it to be placed directly into ophthalmology and optometry practices,” writes analyst Drew Jones.

He said TearLab’s Osmolarity system also does not face any immediate competition. “Taking into account the company’s commercialization strategy (focused on ophthalmologists) and what we believe is a conservative approach to sizing the market, we believe the market opportunity for TearLab is around $576-million today.”

Longer term, he expects TEAR to expand its sales effort to include optometrists that would “easily push the market opportunity for TearLab north of $1.4-billion.”

He said the stock’s valuation is “compelling despite a recent run up.”

This conference brings together brings together U.S., Canadian and international investors who are interested in the latest developments in the Canadian healthcare sector.

A few of us from BioTuesdays will also be attending.  If you would like to meet there with a particular Canadian company CEO that we interviewed this year, or you are a C-suite executive that would like to introduce your company’s story to us at BioTuesdays, please let us know.

The stock was changing hands at $9.03, up 13%, after touching a 52-week high of $9.16 on Tuesday morning.

Feltl analyst Ben Haynor upped his price target to $11 from $9.50, writing that he has increased his estimate for 2013 system orders to 1,263 from 1,120, citing the Master’s program placement. He said management noted on a conference call that they expect approximately half of their placements to be part of that program for the next couple of years.

Craig-Hallum analyst Steven Crowley raised his price target to $10 from $8.50, writing that TearLab is in the “midst of capturing a large and very lucrative market with an already FDA-approved, CLIA- waived, and CMS-reimbursed point-of-care test that is likely to become standard-of-care within an especially attractive medical specialty” for the quantitative diagnosis of dry eye disease.

“We think investors need to own this first mover with a novel point-of-care diagnostic platform play in the very attractive eye care market,” Mr. Crowley added.

Canaccord analyst Jeff Frelick also moved his target to $10 from $8, writing that he expects the “strong ramp to continue through 2013.”

“TearLab crushed our first quarter bookings estimate, driven by strong growth in its Master’s program with large ophthalmology practices,” writes Mr. Frelick.

TearLab booked 388 orders in the quarter for its osmolarity device, which is used to diagnose and manage dry eye disease, well ahead of Canaccord’s estimate of 234. TearLab also placed 324 instruments in the quarter, well ahead of Canaccord estimate of 114.

Roth analyst Matt Dolan raised his price target to $9 from $7.50, pointing out that the most important component to his thesis is the implied revenue that will be generated by each system in the field.

He said that on the conference call, management reiterated its belief that each TearLab system can generate $20,000 annually, implying that its current U.S. order base of 1,101 units would generate $22-million in annual card revenue.

“If the company’s quarterly order rate remains strong (as we saw in the first quarter), then we believe this revenue run rate could accelerate significantly over the next several quarters,” Mr. Dolan added.

Finally, Sidoti & Co. upgraded TearLab to “buy” from “neutral,” based on higher growth estimates for 2013 and 2014, and a roll out of 2015 profit estimates.

Mark J. Ahn, PhD

“Abstral is the latest and best-in-class drug for this indication,” president and CEO Mark Ahn says in an interview with BioTuesdays.com, noting that the company is on track for a fourth quarter launch.

Abstral was approved by the FDA in January 2011 for inadequately controlled BTcP in patients who are already receiving, and who are tolerant to, opioid therapy for their persistent baseline cancer pain. BTcP affects 40% to 80% of cancer patients, with reported episodes of four per day and a median duration of 30 minutes. Abstral delivers the analgesic power and increased bioavailability of micronized fentanyl in a sublingual tablet, which dissolves under the tongue within seconds, relieves pain in minutes and lasts for the entire 30-to-60-minute pain episode.

Galena acquired the U.S. rights to Abstral from Sweden’s Orexo AB in mid-March for $10-million upfront and $5-million within the first 12 months of closing, plus low double-digit royalties and one-time milestone payments based on pre-specified net sales.

Abstral is the transmucosal immediate-release fentanyl (TIRF) market leader in Europe, where it achieved sales in 2012 of $54-million by Orexo’s partner, ProStrakan/Kyowa Hakko Kirin. It is marketed in Canada by Paladin Labs and has been filed for approval in Japan by Kyowa Hakko Kirin. In 2012, the U.S. market for TIRFs was approximately $400-million.

“Abstral diversifies and strengthens our pipeline, gives us an FDA-approved product that will become a cornerstone of our commercial strategy and will generate revenues in 2014 to support the development of our pipeline,” Dr. Ahn contends.

He says Abstral is a perfect fit with Galena, which is currently conducting a global Phase 3 clinical trial with its NeuVax vaccine for the prevention of breast cancer recurrence in early-stage, low-to-intermediate HER2 breast cancer patients. “Abstral will allow us to build a call-point with oncologists who will hopefully prescribe NeuVax in about three years’ time.”

Dr. Ahn explains that the majority of patients who use fentanyl tablets or lollipops for BTcP have solid tumors and that by far, the largest users are breast cancer patients who, with disease recurrence, suffer very painful bone pain.

“Of course, if we are able to prevent disease recurrence in breast cancer patients with NeuVax, that would be even better,” he says. “But from a business perspective, Abstral is a straight line to the future customers of our entire pipeline.”

Based on 2012 data, the market leaders in BTcP were Fentora, a fentanyl-buccal tablet, with sales of $161-million, or an approximately 40% market share, and Actiq, a fentanyl lollipop, with sales of $30-million, or an approximately 7.5% market share. Both products are sold by Cephalon/Teva Pharmaceutical Industries.

“With the first and only sublingual tablet, we know we have the best and most convenient product, as well as the best commercial team,” Dr. Ahn says of Abstral.

He says the company has a commercialization management team in place and is currently deciding whether to contract a sales force, with an option to acquire the team, or build its own sales force to distribute Abstral.

Abstral’s advantages include: rapid absorption into the oral mucosa; rapid onset of action compared with other TRIF’s; the fact that the drug can be titrated quickly by physicians to meet the pain needs of patients; convenient administration under the tongue; and a favorable side-effect profile.

Joseph Pantginis, an analyst with Roth Capital Partners, raised his price target on Galena to $7 from $5 at the end of March, saying “we believe the acquisition [of Abstral] is encouraging as it could potentially mitigate Galena’s burn rate in a significant way, as well as build early relationships with the cancer physicians who will ultimately prescribe NeuVax, if successful.”

NeuVax is an immunotherapy that stimulates the immune system to actively seek out and selectively kill cancer cells. NeuVax directs “killer” T-cells to target and destroy cancer cells that express HER2/neu, a protein associated with epithelial tumors in breast, ovarian, pancreatic, colon, bladder and prostate cancers.

NeuVax Mechanism of Action: Active Specific Immunotherapy (ASI)

When discussing NeuVax, the first breast cancer vaccine in Phase 3 clinical trials, Dr. Ahn frames the novel treatment into three numbers: 25, 0, and 6.

He says that 25% of node-positive breast cancer patients, who achieve remission after standard-of-care treatment, will have recurrence within 36 months. In Galena’s Phase 2 trial, patients receiving NeuVax at the 36-month time point had a recurrence rate of 0%. And $6-billion is the current market size of Herceptin, the market leader for patients with HER2-positive breast cancer.

Herceptin is prescribed only to patients with an immunohistochemistry (IHC) score of 3+, which indicates the highest expression of HER2 receptor protein on the surface of the cancer cells and represents 20% to 30% of the patient population. In addition to Roche/Genentech’s Herceptin, Roche also markets Perjeta for HER2-positive breast cancer. GlaxoSmithKline also has a drug in the space called Tykerb.

Galena’s NeuVax is targeting HER2 IHC 1+/2+ breast cancer patients, who are not eligible for Herceptin. “Low-to-intermediate HER2 expression is a significant unmet medical need and isn’t addressed by current therapies,” Dr. Ahn points out. “It’s about double the size of the Herceptin market.”

NeuVax v. Herceptin Target Therapy for HER2+ Breast Cancers

The annual addressable node-positive population for NeuVax would represent 30,000-to-40,000 patients in the U.S. and 50,000-to-80,000 in Europe, he adds.

Based on a successful Phase 2 trial which achieved its primary endpoint of disease-free survival, the FDA, in 2012, agreed that the design and planned analysis of the Phase 3 study adequately address the objectives necessary to support an acceptable regulatory submission for marketing approval.

The company’s Phase 3 PRESENT trial with NeuVax, which has a special protocol assessment from the FDA, is enrolling 700 patients, who have successfully been treated with surgery, radiation and/or chemotherapy. There are over 100 hospitals participating in the study, of which about 45 are in North America and the remainder in Europe and Israel.

Dr. Ahn says the company hopes to complete enrollment at the end of 2013, with an interim analysis around the beginning of next year or at 70 events.

In addition to the PRESENT trial, Galena and Genentech/Roche are collaborating with the Henry Jackson Foundation on a Phase 2b study that is comparing NeuVax in combination with Herceptin against Herceptin alone. The trial is enrolling 300 node-positive breast cancer patients with HER2 1+ and 2+ expressing tumors, which are disease-free after standard-of-care therapy.

In an earlier study with 62 patients, Dr. Ahn says 32 patients on Herceptin alone had a 12.5% recurrence rate after 24 months, while 30 patients on NeuVax plus Herceptin had a recurrence rate of 0%.

Asked about its partnering plans, Dr. Ahn says the company is actively looking at distribution partnerships for NeuVax outside of North America. “But we plan to distribute NeuVax in North America on our own, which is why the Abstral acquisition was so important. Our intention is to build a valuable oncology company that makes a meaningful difference in advancing cancer care, and that’s exactly what we’re doing.”

GALE Pipeline

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A total of 388 orders for TearLab Osmolarity systems were booked in the latest quarter. Of those, 162 systems were under the company’s new “Masters Multi Unit Program,” 195 were through its “3/15″ and “3/24″ access programs, 11 were direct purchases and 20 were purchased outside of the U.S.

“We started 2013 with strong system orders and a solid launch of our new Masters program,” CEO Elias Vamvakas said in a statement “While our revenue growth highlights our continuing success in building a recurring revenue base, like in previous quarters, our strong sales performance was not fully reflected in our first quarter financial results due to the traditional lag between access program contract signing and product shipment, and to our current system manufacturing back-order position.”

He said that moving forward, “driven by the success of our sales and marketing programs, and by strong showings at key events like ASCRS, we expect to see this positive momentum continue through the year.”