Trevor Arsenault, VP of business development and alliance management

With franchises in dermatology, wound care and oral health, closely-held Klox Technologies has three near-term milestones this year: launching its oral care business with Colgate Palmolive; expanding the Dermatology franchise; and seeking a marketing partner for its wound business care.

“We are the only fluorescence-based, topical technology on the market for skin and soft tissue disorders,” Trevor Arsenault, VP of business development and alliance management, says in an interview with BioTuesdays. “The technology has been validated in that two of our three programs are fully partnered.”

Mr. Arsenault explains that Klox’s BioPhotonics platform consists of a multi-LED lamp and a topical photo converter media, such as a gel, which includes light-capturing molecules. Once the gel is illuminated, different molecules in the gel produce different wavelengths of light, which penetrate the skin to different depths, he points out.

“This fluorescence biomodulation can stimulate cellular responses along with controlling bacteria, modulating inflammatory mediators, stimulating fibroblasts for collagen synthesis/deposition, modulating vascular endothelial growth factors, and myofibroblast formation,” he contends.

“And these hyper-pulsed fluorescence systems are modulated to individual indications,” he says, adding that current clinical programs have shown a high degree of safety and efficacy. “A point of differentiation is that this treatment is non-systemic.”

Klox was founded in 2007 by Dr. Francesco Bellini, a biotech entrepreneur and co-founder of BioChem Pharma, which discovered the antiviral, 3TC, a cornerstone for HIV/AIDS therapy.

The BioPhotonics platform, which can be used in combination with existing therapies or on its own, has CE Mark designations in Europe for dermatology, oral health and acute chronic wounds. In Canada, Klox has approval to use its technology in dermatology for the treatment of moderate-to-severe acne.

Mr. Arsenault says the Klox and LEO Pharma of Denmark have been collaborating since 2014 to develop Klox’s dermatology business and in March of this year, the business was spun out into a new company, FB Dermatology, which is jointly owned by both companies.

FB Dermatology is commercializing Klox’s BioPhotonic technology in dermatology under the brand name, Kleresca for acne and Kleresca for skin rejuvenation.

FB Dermatology is commercializing Klox’s BioPhotonic technology in dermatology under the brand name, Kleresca for acne and Kleresca for skin rejuvenation

“Down the road, we are looking to expand in other dermatology indications, such as eczema and rosacea,” he adds. FB Dermatology is currently operating in seven countries in Europe and Australia, with plans to be in 12 countries by the first half of 2018.

In oral health, which is partnered with Colgate Palmolive, Mr. Arsenault says Klox has completed a proof-of-concept study, which showed statistical significance on clinical attachment after one treatment of its PERIO-1 gel as an adjunct to scaling and root planning in periodontal patients.

Periodontitis is the number one cause of tooth loss in adults and represents a $14-billion market.

In addition, he says Klox is focusing its business development activities to partner its wound care business, which has CE Mark approval for chronic and post-surgical wounds as well as burns in Europe.

In May, Klox reported positive results of a 100-patient EUREKA study as the European Wound Management Association in Amsterdam, with its LumiHeal gel for the treatment of hard-to-heal, non-responsive, chronic wounds.

the EUREKA study demonstrated a high rate of wound closure with LumiHeal, pain reduction, improved quality of life, control of bacteria and reduction of inflammation

The data confirmed an interim analysis reported in September 2016 on 33 patients with Venus leg ulcers (VLUs), diabetic food ulcers (DFUs) and pressure ulcers.

Among other things, the EUREKA study demonstrated a high rate of wound closure with LumiHeal, pain reduction, improved quality of life, control of bacteria and reduction of inflammation.

In addition, 96% of wounds that closed completely, remained closed throughout the follow up period, and 69.2% of VLUs and 68.8% of DFUs were ready for skin graft, according to the clinician’s opinion. A study with patients suffering from pressure ulcers is ongoing.

Mr. Arsenault says that the final results of the EUREKA study show, that taken together with efficacy, treatment ease of use, safety, and a positive impact on pain reduction, “LumiHeal has the ability to become a leading option for the treatment and management of hard-to-heal, chronic wounds.”

Klox also has an ongoing study in post-surgical scarring with 42 patients undergoing bilateral breast reductions, with full data set to be reported in the current quarter. Interim data demonstrated lower rates of hyper pigmentation and rates of red scars and normal scar height, compared with silicone sheets.

Regarding its R&D pipeline, Mr. Arsenault says the company is working in animal health, including indications, such as pyoderma and wound care, and has established strong preliminary data; burns; additional consumer products; and rare keratin and collagen-based genetic diseases, which is at the preclinical stage.

“Our positioning is the R&D of the BioPhotonics technology, then using partnerships to go to the market,” he points out. “We will continue to push and expand where the platform can be used.”

via BioTuesdays Features - BioTuesdays http://ift.tt/2tQYq7D

Marc Wayne, president and CEO

As president and CEO of newly formed Canopy Health Innovations, a spin off of Canopy Group (TSX:WEED), a world leading diversified cannabis company, Marc Wayne is a pioneer of the medical cannabis sector. He was a co-founder of Bedrocan Canada, one of Canada’s first licensed producers of medical cannabis, and founding chairman of the Cannabis Canada Association, a trade group with 17 licensed producers. Mr. Wayne also was instrumental in developing the Canadian Consortium for the Investigation of Cannabinoids, a global innovator in education and research on medical cannabis. In this interview with BioTuesdays, Mr. Wayne discusses the potential of cannabis-based treatments and how they can offer alternatives to traditional medicines, avoid negative side effects and be disruptive to the pharmaceuticals sector.

Why was Canopy Health created?

We launched Canopy Health last December as a R&D-based biopharmaceutical company to focus on developing and researching clinically-ready, whole-plant cannabis drug formulations and dose delivery systems for human and companion animal health applications. The ultimate goal is validating diversified treatment options for Canadians and for patients and consumers in other jurisdictions, where treatment with cannabis is federally lawful. We plan to undertake a combination of exploratory studies and early-stage clinical testing to identify, develop and validate products aimed at disrupting the market for a variety of existing pharmaceutical product categories, and then commercializing those products through licensing or other arrangements. Our goal is to provide cannabinoid-based medicines as a safer and effective alternative to established therapies. So, by putting these products through clinical testing and establishing validated health claims, doctors will be more comfortable with the data behind them.

What’s your connection with Canopy Growth?

Canopy Growth, which has a market cap of about $1.2-billion, is the largest but not the controlling shareholder of Canopy Health. We have preferential access to Canopy Growth’s supply and licenses and Canopy Growth has first right to commercialize any IP and/or products we develop.

The Canopy-Health Research Model

Can you explain cannabis medicines as disruptive to the pharma sector?

I believe there is a market for natural, validated and improved cannabis products that will replace or be used as adjunct therapies to current medicines on the market for specific indications. We are beginning to see cannabis oils replacing dried flowers in the medical field because they provide the standardized reproducibility and dosage form, which researchers need and doctors are comfortable prescribing. There was a study in the U.S., looking at Medicaid expenditures in states where medical cannabis is legal, which found an 11% reduction in spending for traditional pharmaceuticals, or some $4-billion. So, in areas where medical cannabis is available, there is some disruptive effect on people using traditional medications. And we see that anecdotally from people registered in the medical marijuana program in Canada, claiming that when they use cannabis, they use less of their other medications. So we think there is an opportunity to develop and distribute cannabis products that are clinically tested and have validated health claims. And our pharmacy system is actively looking to distribute cannabis products and has applied to Health Canada to do so, which makes them a natural outlet for these products.

What’s the regulatory difference in the U.S. and Canada?

Research is difficult to do in the U.S. because cannabis is categorized as a Schedule 1 drug by the DEA, which implies that it has no acceptable medical value. That hinders the ability to access product for research and get protocols approved for research. By and large, the focus in the U.S. is on showing the harm of cannabis and it has been difficult for researcher’s to get FDA approval for protocols aiming to show efficacy of cannabis for medical purposes. However, in Canada, we have a much more favorable environment and it’s a great time to seize this opportunity. For example, the current cannabis medical program in Canada allows us to do observational and exploratory research straight in humans because we can already legally and medically access the product, even though it is a controlled substance. Part of our work is to do these early studies to find product signaling in different disease indications.

What’s the regulation state in Europe?

The European market is evolving rapidly in terms of medical cannabis. Holland, of course, has been leading regulation for many years, having supplied cannabis through their pharmacies since 2002. Germany, a much larger market, recently approved medical cannabis use and some Canadian companies, including Canopy Growth, are already exporting dried flowers in patient-ready containers to Germany. I believe these products are being sold in German pharmacies. In addition, we understand that research data in Germany will help facilitate insurance coverage. And clinical research is a big focus in the Czech Republic, which also has a federal medical cannabis program.

What is Canopy Health working on?

canopy-health positioning 

We’re not disclosing what we’re working on specifically, but in general, we’re targeting whole-plant, multiple compound formulations towards specific medical indications. We have a strong focus on intellectual property creation and we are targeting both human and companion animal health. We spend time characterizing strains and products that we want to work with, studying technologies and delivery systems. We are looking to research a variety of different products with different delivery systems, form factors, depending on the specific indication. For example, it makes sense to deliver an arthritis product as a topical lotion, while a treatment for sleep or anxiety might best be delivered orally.

Can you discuss your development timelines?

For the balance of this year and into early 2018, we plan to conduct exploratory observational studies, begin IP protection and settle on formulation and trial design, with our first clinical trials beginning in the spring next year. The plan is to begin the drug approval process with our first product in 2019.

CANOPY-HEALTH TIMELINE

What’s your business model?

Canopy Health owns all of our IP. We partner for supply primarily with Canopy Growth or if needed in licensing deals with other biopharmaceutical, nutraceutical, technical device and/or medical cannabis companies as required, all with the objective of receiving out-licensing and commercialization fees on products we create. As mentioned, Canopy Growth is our first call customer.

Can you give us an overview of Canopy Animal Health?

It is a wholly-owned subsidiary of Canopy Health and will focus on creating cannabis-based healthcare products for companion dogs and cats in order to maintain, improve or extend the quality of life of family pets. Our focus is on palliative care, anxiety, appetite stimulation and pain management. We are working with contract research organizations with veterinary drug development experience. We believe there is a gap and need for a company looking seriously at natural cannabinoid drug development for our little friends who share many of the same ailments. We want Canopy Animal Health to be a leader in this area.

How are you financing product development?

We raised $7-million in a seed round last year and we are now actively in the process of rasing an additional $7-million to $10-million in an A round, which should take us well into our development plan over the next 3-5 years. Proceeds will be used to advance research and product development, pursue IP protection and commercialization, and for working capital.

canopy-health pipeline roadmap

via BioTuesdays Features - BioTuesdays http://ift.tt/2sOddD3

Albert Friesen, CEO and chairman

Medicure (TSXV:MPH) intends to leverage its unique sales and marketing model, which has fueled the growth of its Aggrastat IV platelet inhibitor, as it brings new generic products into its cardiovascular portfolio.

Aggrastat has strong efficacy data and is priced to provide considerable savings to hospital budgets. In early clinical testing, Aggrastat demonstrated a 41% reduction in death and myocardial infarction in high-risk cardiovascular patients.

“We tried several models after we acquired the U.S. rights to Aggrastat from Merck in 2006, but frankly they didn’t work,” Albert Friesen, CEO and chairman, says in an interview with BioTuesdays. “They were costly and we didn’t gain the traction with hospitals and doctors that we were looking for.”

So the company went back to the drawing board. It hired one sales executive and one PhD, who were experts in Aggrastat, and gradually expanded its team. Currently, it stands at 53 people, including 40 in sales and marketing, and 13 PhDs. They all work out of the company’s head office in Winnipeg, Canada.

“Every day at 4 pm the sales team meets, in what is called ‘the huddle’, where the team exchanges experiences they’ve had with clients,” Dr. Friesen says. “Everybody on the team is highly trained to promote and explain the quality and benefits of using Aggrastat and we’ve developed a total service and relationship approach with our clients.”

Aggrastat is indicated for the treatment of acute coronary syndrome

Sales staff spend about 50% of their time servicing hospitals and doctors in the U.S. and “the program is highly efficient and less costly than earlier models we tried,” he adds. “We have an infrastructure in place and now we are going to leverage it as we call on the same hospitals with additional products.”

Dr. Friesen also notes that Aggrastat, which is indicated for the treatment of acute coronary syndrome, is priced below two competing products in the hospital market. Aggrastat costs at least 25% less than Integrilin, and over 70% less than ReoPro.

The strategy seems to be working. Medicure has grown yearly sales of Aggrastat from a low of $2-million in 2009, to $30-million in 2016. Dr. Friesen says the drug is tracking to continued growth this year.

In addition, Aggrastat has grown its patient market share from 2% a few years ago to almost 50% now.  

Medicure made a big push converting hospitals to Aggrastat with a high-dose bolus regimen, which was approved by the FDA in 2013 and became the recommended dose for a reduction of thrombotic cardiovascular events in patients with non-ST elevated acute coronary syndrome.

Last September, the FDA also approved a new bolus vial format of Aggrastat. Dr. Friesen says Aggrastat is the only product of the three in its market, which enables doctors to use a short infusion that positions the product favorably in the contemporary setting of coronary intervention.

“Development of the bolus vial was in response to feedback we received from interventional cardiologists and catheterization lab nurses across the U.S.,” he adds. “We believe this new product format will have a positive impact on hospital utilization of Aggrastat.”

In the first quarter this year, Medicure experienced its highest level of Aggrastat hospital demand in the history of owning the product. Hospital demand exceeded wholesale demand due to reductions in inventory levels within the wholesale channel. 

In the first quarter this year, Medicure experienced its highest level of Aggrastat hospital demand in the history of owning the product

Net revenue from the sale of Aggrastat in the first quarter of 2017 was $8.7 million, compared with $6.1 million for the year-earlier quarter, an increase of 43%.

Dr. Friesen recalls that when Medicure began looking to expand its acute care cardiovascular portfolio, it asked doctors using Aggrastat for a list of the five top products they used in their practice with the same patients. “Rather than purchase any of these products, we decided to develop some generic versions of them,” he adds.

While the company was looking for one of these products, it came across a company called Apicore LLC, which was one of two companies developing a product Medicure was interested in.

In 2014, Medicure acquired a minority stake in Apicore, climbing to 61% last December on a fully diluted basis. Medicure has an option to raise its stake to 100% in July 2017. Apicore’s founders currently own about 35% of the company.

Apicore is a New Jersey-based developer and manufacturer of specialty active pharmaceutical ingredients (APIs) and generic pharmaceuticals, with more than 15 abbreviated new drug applications (ANDAs). The company also manufactures more than 100 different API's, including more than 35 for which Drug Master Files have been submitted to the FDA and 12 that are approved for commercial sale in the U.S. by Apicore’s customers.

Apicore is a developer and manufacturer of specialty APIs and generic pharmaceuticals, with more than 15 ANDAs

Sales for Apicore were about $24 million in 2016, with continued sales growth expected in 2017.

In March the FDA approved Apicore’s ANDA for tetrabenazine to treat involuntary movements of Huntington’s disease. TAGI Pharma holds the commercial rights to tetrabenazine, while the branded product, Xenazine, is sold by Valeant Pharmaceuticals.

Last December, Medicure filed an ANDA with the FDA for an intravenous generic product for an acute cardiovascular indication, which is partnered with Apicore. Medicure holds all the commercial rights to the product, as well as for two other generics under development with other companies.

“In 2018, we should be in the market with our first generic and by the end of 2019, we should be selling the other two, for a total of four products, including Aggrastat,” he adds. “In five years, I’d like our portfolio to have more than five products.”

via BioTuesdays Features - BioTuesdays http://ift.tt/2sXge0D

Fahar Merchant, chairman, president and CEO

Medicenna Therapeutics (TSXV:MDNA) is advancing a targeted dual-action immunotherapeutic, MDNA55, that consists of an engineered version of interleukin-4 (IL-4) plus a tumor-killing cytotoxin for the treatment of recurrent glioblastoma, the most common and uniformly fatal form of brain cancer.

“Cancer cells proliferate with the help of IL-4, so our approach is to trick the cell to ingest our toxin-laden IL-4 Empowered Cytokines, which makes MDNA55 a powerful Trojan horse,” Fahar Merchant, chairman, president and CEO, says in an interview with BioTuesdays.

“Because MDNA55 is so targeted and so potent, we don’t need to use much of the drug,” he adds, noting that one-gram of MDNA55 is enough to treat 10,000 patients.

Medicenna, which is derived by combining the word, medicine, with Avicenna, the father of evidence-based medicine, is currently enrolling patients in a Phase 2b study, which if successful, could lead to an application for accelerated approval of MDNA55 for patients with recurrent glioblastoma.

Dr. Merchant explains that MDNA55 enters a cancer cell by binding to the IL-4 receptor on the cell surface and, after entering the cell, initiates apoptosis, or cell death.

According to Dr. Merchant, the IL-4 receptor is over expressed in about 20 different cancers, affecting more than one million cancer patients every year, but is not generally associated with normal brain cells or healthy tissue.

according to merchant, “our drug candidate has a two-prong approach, targeting tumor cells and the immunosuppressive tumor micro-environment”

Based on an analysis of biopsies, the IL-4 receptor is widely seen in many other CNS cancers, including newly diagnosed glioblastoma, metastatic brain cancer, meningioma, diffuse intrinsic pontine glioma, CNS lymphoma, pediatric brain tumors and pituitary adenomas.

Recurrent glioblastoma has an annual incidence of 33,300 in the U.S., EU and Japan, representing a market opportunity of $650-million based on current pricing assumptions.

Medicenna also is developing MDNA55 as a potential treatment for metastatic brain cancer and pediatric glioma, which have a total annual incidence of 95,300 patients and a market opportunity of more than $1.3-billion.

MDNA55 has Orphan Drug status from the FDA and European Medicines Agency, and Fast Track designation from the FDA. More than 40 issued and filed patents cover Medicenna’s IP portfolio.

Dr. Merchant points out that the IL-4 receptor is also found on non-malignant cells that protect the tumor from being attacked by the immune system. This immunosuppressive tumor micro-environment comprises some 40% of a glioblastoma tumor mass.

“So our drug candidate has a two-prong approach, targeting tumor cells and the immunosuppressive tumor micro-environment,” he adds.

Pointing to potential advantages of MDNA55, Dr. Merchant suggests that 55% of glioblastomas are resistant to chemotherapy, while MDNA55 targets these same chemo-resistant cells.

“We are looking to treat patients with glioblastoma that relapse after surgery, radiotherapy and chemotherapy,” Dr. Merchant explains, adding that 75% of these relapsed patients are not candidates for another bout of surgery.

In addition, MDNA55 is delivered by direct injection to the tumor in the brain using a minimally invasive image-guided catheter, enabling high drug doses without systemic exposure. The delivery system also by-passes the blood-brain barrier, which would otherwise block transport of therapeutics to the brain.

Seventy-two patients have been treated with MDNA55 in three early-stage clinical trials in the U.S. and Germany. The drug was well tolerated with no adverse systemic side effects.

In one study with 25 patients who were not candidates for surgery, Dr. Merchant says five patients had a complete response and nine had a partial response, where the tumor shrunk by more than 50%, after one treatment with MDNA55. The objective response rate in the study was 56%.

He also notes that the 14 responders to MDNA55 had a median survival of 379 days, while the 11 non-responders had a median survival of 98 days.

Seventy-two patients have been treated with MDNA55 in three early-stage clinical trials in the U.S. and Germany. The drug was well tolerated with no adverse systemic side effects.

In addition, 20% of the 57 patients in two of the three early studies, including those that had and those that didn’t have surgery, achieved long-term survival of three-to-four years, which Dr. Merchant says is typical of “what we see with today’s checkpoint inhibitors approved for other types of cancer.”

He also points out that in clinical trials that cleared Roche’s Avastin to treat recurrent glioblastoma, all patients died after 14-to-18 months. “Despite an inferior patient population, compared with the Avastin studies, MDNA55’s response and survival rates are compelling,” he adds.

In April, Medicenna treated the first of the planned 43 patients, who have progressed or recurred following previous therapy, in a Phase 2b clinical trial at nine sites in the U.S., including Duke, UCSF, Cleveland Clinic and Weill Cornell - Sloan Kettering.

Patients will receive MDNA55 with a second-generation image-guided catheter in order to bypass the blood-brain barrier as well as an imaging agent for real-time monitoring of drug distribution.

The primary endpoint is overall response rate, with secondary outcome measures including, progression-free survival, overall survival, and exploratory predictors of outcome assessed by IL4 receptor expression in archived tumor biopsies.

“With those nine clinical sites, we expect to complete enrolment before the end of 2017, with top-line results anticipated in the first quarter of 2018, and an end-of-Phase 2 meeting with the FDA in the second quarter,” Dr. Merchant suggests.

If the company succeeds in obtaining accelerated approval of MDNA55 for the treatment of recurrent glioblastoma, it would likely move into a Phase 3 study with patients having newly-diagnosed glioblastoma.

MDNA55 AND MDNA109: product pipeline

In the second half this year, Medicenna plans to begin a Phase 2 study with MDNA55 in patients with metastatic brain cancer, with data available towards the end of 2018.

Medicenna also is collaborating with MD Anderson Cancer Center in Houston to develop next generation fusion protein therapeutics targeting the IL-4 receptor. The multi-year collaboration will focus on advancing preclinical development of IL-4 Empowered Cytokines and a Superkine platform, which Medicenna licensed from Stanford University.

Dr. Merchant points out that analysis of patient biopsies consistently shows over-expression of the IL-4 receptor in many solid cancers outside the brain as well as leukemia and lymphoma. “We hope to have a candidate ready for IND-enabling studies ready by the end of 2018.”

Medicenna has been funding its recurrent glioblastoma clinical trial and next-generation IL-4 Empowered Cytokine programs with a $14.1-million non-dilutive grant from the Cancer Prevention & Research Institute of Texas (CPRIT) along with an additional $14-million (Canadian) of private capital raised prior to going public early this year. In return, CPRIT is eligible for low single-digit royalties following commercial launch of MDNA55 to a maximum of four times the original grant.

“The CPRIT grant provides solid third-party validation of our platform,” Dr. Merchant contends.

via BioTuesdays Features - BioTuesdays http://ift.tt/2rwtIlc