REDONDO BEACH, CA / ACCESSWIRE / October 19, 2015 / It's with good reason that there is a growing choir of voices heralding the science of targeted therapies as innovative approaches to improving medical care. Advancements in biology and technology underscore discovering specific receptors in cells that can be targeted for meaningful benefits in hard to treat diseases and conditions. There is also a significant diagnostic component in identifying which patients may benefit from certain therapies, while giving direction to avoid other treatments that likely would be ineffective. Combined, these streamline the treatment process and improve outcomes, equating to better quality of life for patients and a quantifiable savings to overburdened healthcare systems.

Targeted therapies are becoming prominent in the cancer space, including the work of cell therapy company Caladrius Biosciences (CLBS), which late in September announced an award from the National Cancer Institute to fund research to optimize its therapeutic agent targeting tumor-initiating cells. The company's lead product candidate is CLBS20, which is being used in an ongoing Phase 3 trial for metastatic melanoma under Fast Track and Orphan Drug designations with Special Protocol Assessment granted by the FDA. New data on CLBS20 is being reported at the popular Alliance for Regenerative Medicine's Stem Cell Meeting on the Mesa Scientific Symposium on October 9, 2015.

Loxo Oncology (LOXO) is making headway with its oral cancer drug candidate LOXO-101, the only selective tropomyosin receptor kinase (TRK) inhibitor in clinical development. LOXO-101 is currently in a Phase 1 dose escalation trial at the University of Colorado Cancer Center for patients with advanced solid tumors. Late in July, the peer-reviewed journal Cancer Discovery published a research brief describing a female patient in the trial with advanced soft tissue sarcoma widely metastatic to the lungs that responded to LOXO-101 after failing other courses of treatment.

Also contributing to the brief was molecular information company Foundation Medicine (NASDAQ:FMI), the developer and marketer of the clinical assays FoundationOne for solid tumors and FoundationOne Heme for hematologic malignancies and sarcomas. The assays deliver a genomic profile that identifies molecular alterations in a specific patient's cancer and matches that profile with relevant targeted therapies and clinical trials. In the Loxo trial, FoundationOne Heme was used to determine that the patient's cancer harbored a TRK gene fusion.

While targeted therapies are seeing a growing importance for cancer, cardiovascular disease, the leading cause of death in the U.S. in 2013, remains relatively unattended regarding the new approach. That is, with the exception of ARCA biopharma (ABIO), who is developing Genarco, potentially the first genetically-targeted treatment for atrial fibrillation (AF), under a Fast Track designation from the FDA. AF is an abnormal heart rhythm where the atria beats irregularly, allowing blood to slow down and potentially pool that affected some 5 million Americans in 2010, according to GlobalData. As published by the American Heart Association, an estimated 33.5 million people around the world have AF. Because poor blood movement increases the chances of a clot, ischemic stroke is a real concern, with about 15-20 percent of all stroke patients annually having AF. AF is now considered an epidemic cardiovascular disease because of the increasing incidence in the U.S. and industrial countries, which makes ARCA's efforts in developing a genetically-targeted treatment for AF an important potential advancement for patients living with the disease or at risk for developing it.

Historically, vitamin K antagonists like warfarin have been widely used to try and avoid stroke in AF patients, however, there is still an unmet need for safer antiarrhythmic drugs to treat AF. Amiodarone has common and sometimes serious side effects and contraindications, yet the FDA approved it in 1985 to treat arrhythmias. In 2009, the FDA approved Sanofi's (NYSE:SNY) MULTAQ (dronedarone). All of the antiarrhythmic drugs approved today have one thing in common: they are not for AF patients with heart failure with reduced ejection fraction (HFREF). Beta-blockers approved for HFREF patients are often used off-label for AF, but there is evidence that they provide little therapeutic benefit for this patient population.

Gencaro, a beta-blocker/vasodilator, has been extensively studied, including a Phase 3 trial of 2,708 patients that showed a 74 percent reduction in incidence of new onset AF in patients with the genotype most responsive to Gencaro. Although it is a beta-blocker at its core, this targeted therapy is different as Gencaro has additional properties versus other Beta-blockers, specifically inverse agonism and lowering noepinephrine. The compound's mechanism of action involves targeting cardiac myocytes to reduce adverse Beta-1 adrenergic signaling that causes cardiac chamber remodeling. The fact that Gencaro is a Beta-blocker is actually a benefit to ARCA because this class of drugs has been studied for decades with a well-characterized safety profile, which de-risks some of the research by ARCA.

The decision to focus on Beta-blockers was spearheaded by Dr. Michael Bristow, President and CEO of ARCA. Dr. Bristow has been a long-time scientific and academic thought leader in developing Beta-blockers. It was Bristow who led the movement to use Beta-blockers on heart failure patients, when they were historically labeled as contraindicated. Today, Beta-blockers are the first-line treatment for heart failure patients. Dr. Bristow was also the founder of Myogen, a company acquired by Gilead Sciences (NASDAQ:GILD) in 2006 for $2.5 billion, to gain control of ambrisentan, an oral endothelin receptor antagonist that was eventually approved under the brand name Letairis for treatment of pulmonary arterial hypertension.

ARCA was founded with a focus in the targeted therapy arena. Its scientists identified a specific genotype where Gencaro is more effective. While multiple genotypes can benefit, clinical research has determined that the most responsive genotype is ADRB1 Arg389Arg, which is present in 50 percent of the U.S. population. Through a collaboration with Laboratory Corporation of America (NYSE:LH), the two companies developed a simple blood diagnostic test to identify the genotypes. ARCA retains the intellectual property and commercialization rights to the diagnostic test.

Moreover, clinical data from the 2,708 patient Phase 3 trial (the "BEST" trial) of Gencaro suggest that Gencaro may provide a clinical benefit to cardiovascular patients with the ADRB1 Arg389Arg genotype, including controlling and preventing heart failure events, ventricular tachycardia/ventricular fibrillation and other indications.

The Phase 2B/3 trial of Genarco, dubbed "GENETIC-AF," is an adaptive design clinical trial, slated to enroll a total of 620 AF patients with HFREF with the ADRB1 Arg389Arg genotype. Medical technology giant Medtronic (NYSE:MDT) is a collaborator in the trial, providing the implantable continuous monitoring devices to measure and analyze AF burden in patients. The Phase 2b portion will enroll about 200 patients, with enrollment expected to be completed by the end of 2016. Upon successful evaluation of data by the trial's Data Safety Monitoring Board, ARCA could move the trial forward into Phase 3 , meaning this could be a pivotal trial to support an NDA with the FDA for commercialization of Genarco in the United States. ARCA currently owns world-wide rights for Gencaro.

Biotechs broadly took a hit in September, with the iShares Nasdaq Biotechnology ETF posting its worst month in more than a decade. A good place to look for value during a retracement is in companies addressing areas of unmet need with innovative therapies. The targeted therapeutic space certainly fits that bill, especially for cardiovascular conditions where there is very limited competition.

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SOURCE: Emerging Growth LLC