Sign Up NowThis Month's Tiny Gems - February 2014

Amarin Corp. (AMRN)
Discovery Laboratories, Inc. (DSCO)
Kreisler Manufacturing Corp. (KRSL)
Stemline Therapeutics, Inc. (STML)

 

 

 

Amarin Corp. (AMRN)

 

Amarin Corp. (AMRN) is engaged in commercializing and developing therapeutics to improve cardiovascular health. Vascepa(R) is Amarin's first FDA-approved product as an adjunct to diet to reduce triglyceride levels in adult patients with severe hypertriglyceridemia. Amarin refers to this as the MARINE indication.

 

In its first year of commercial operations, Vascepa recorded (non-GAAP) revenues of $28.1 million, as compared to no revenues in 2012. This figure is comprised of $26.4 million in recognized revenue and recorded deferred revenue of $1.7 million at December 31, 2013. The company recorded (non-GAAP) adjusted net loss per share of $1.26 for 2013, as compared to an adjusted net loss of $0.87 for the same period in 2012.

 

Amarin also increased its patents issued or allowed in the United States to 40, and halved the number of worldwide staff to reduce costs and better match the operational size of the company. Furthermore, Amarin made significant progress throughout multiple areas of the Vascepa commercialization plan, growing the Vascepa prescriber base to more than 16,000 physicians.

 

Moving forward, Amarin CEO John F. Thero says the company will focus on expanding Vascepa to improve treatment options for patients with mixed dyslipidemias, which is the combination of elevated LDL cholesterol and triglyceride and decreased high-density liproprotein (HDL).

 

Vascepa was given three years of marketing exclusivity by the U.S. Food and Drug Administration (FDA) in connection with the MARINE indication, and the company expects to see continued sales growth throughout 2014. Plans for this growth include the launch of multichannel awareness campaigns, including medical education and promotional educational programs, disease state and other direct personal campaigns, managed care education, and presence at key medical association and scientific meetings.

 

 

 

Discovery Laboratories, Inc. (DSCO)

 

Discovery Laboratories, Inc. (DSCO) is a specialty biotechnology company dedicated to advancing a new standard of respiratory critical care. The company’s technology platforms include a novel proprietary KL4 surfactant and proprietary drug delivery technologies being developed to enable efficient delivery of aerosolized KL4 surfactant and other inhaled therapies.

 

Discovery Labs' strategy is to initially focus on neonatology and improve the management of RDS in premature infants. In support of this mission, the company believes that its RDS product portfolio has the potential to become the new standard of care for respiratory distress syndrome (RDS) and, over time, enable the treatment of a significantly greater number of premature infants.

 

Discovery Labs’ proprietary capillary aerosol generator (CAG) is initially being developed as part of the AEROSURF® program. AEROSURF is a novel investigational drug-device combination product being developed to deliver Discovery Labs' KL4 surfactant in aerosolized form to premature infants with RDS. The appeal of AEROSURF is its potential use for the administration of KL4 surfactant to premature infants without invasive endotracheal intubation.

 

 

 

Kreisler Manufacturing Corp. (KRSL)

 

Kreisler Manufacturing Corp. (KRSL), through its wholly owned subsidiary Kreisler Industrial Corporation, manufactures precision metal components and manifold and tube assemblies for use in military and commercial aircraft engines and industrial gas turbines.

 

With design and manufacturing facilities in North America, Kreisler has established a global customer base that includes Rolls-Royce, Siemens, Pratt & Whitney, General Electric, Mitsubishi, Honeywell, Volvo, and more.

 

The company’s ISO 9000 registered quality system and aerospace-certified manufacturing processes complement its in-house design, engineering, and manufacturing capabilities by ensuring that processes are under statistical control and that products meet mil spec and other quality standards.

 

Kreisler has earned NADCAP accreditation for internal manufacturing processes, including welding, nickel plating, as well as special chemical processes such as passivation and titanium cleaning.

 

 

 

Stemline Therapeutics, Inc. (STML)

 

Stemline Therapeutics, Inc. (STML) is engaged in the development of oncology products that target both tumor bulk and cancer stem cells (CSCs). Whereas most cancer therapies target only the tumorm mass, leaving CSCs unaffected and leading to a recurrence of the tumor, Stemline's therapies are specifically designed to attack the tumor and CSCs to induce response and prolong survival.

 

The company's development portfolio includes candidates SL-401 and SL-701, which have demonstrated clinical activity, including durable complete responses (CRs), in phase 1/2 studies of patients with advanced hematologic and brain cancer, respectively.

 

SL-401 is a novel targeted therapy that attacks both tumor bulk cells and CSCs of multiple hematologic cancers, including blastic plasmacytoid dendritic cell neoplasm (BPDCN) and acute myeloid leukemia (AML). SL-401 targets the interleukin-3 receptor (IL-3R), which is over-expressed on tumor cells and CSCs but only minimally expressed on healthy cells. This specific activity leads to an improved safety profile with minimal off-target effects. In the phase 1/2 trial of single agent SL-401, five of six BPDCN patients achieved tumor shrinkage (83% overall response rate), and there were three CRs. In addition to BPDCN, SL-401 has shown very promising single agent activity in patients suffering from relapsed/refractory AML.

 

SL-701, an off-the-shelf, subcutaneously injected peptide vaccine comprised of several synthetic peptides, is being advanced into later stage trials of adults with second-line glioblastoma multiforme (GBM), the most common and aggressive type of brain tumor, and children with brainstem and non-brainstem glioma. The candidate is designed to stimulate the immune system in a very targeted manner to cross the blood-brain barrier and attack tumor cells located anywhere in the brain. In phase 1/2 clinical trials, SL-701 was shown to be safe and well-tolerated, with injection site reactions and fever the only notable adverse events. These trials also showed single agent efficacy, with an overall response and disease stabilization rate of 59% (13/22) in adult patients with recurrent/refractory high-grade gliomas, including recurrent/refractory GBM. The overall response and disease stabilization rate in pediatric patients with gliomas was 86% (19/22).