EBS Back On Track, BABY On Firm Footing, RARE Rising, GNCA For The Long Haul

(RTTNews) - Shares of Capricor Therapeutics Inc. (CAPR) rose more than 11% on Wednesday as investors cheered the news of FDA's orphan drug designation for the company's cell therapeutic product candidate, CAP-1002, for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy.

The company anticipates filing an IND for CAP-1002 with the FDA in treating Duchenne Muscular Dystrophy-associated cardiomyopathy. in the near future.

A phase I trial of CAP-1002 in patients with advanced heart failure and a a phase I/II clinical trial of CAP-1002 in reducing infarct (scar) size in patients who have suffered a heart attack are already underway.

CAPR closed Wednesday's trading 11.28% higher at $7.50. In after-hours, the stock was up another 5.07% at $7.88.

Emergent BioSolutions Inc. (EBS) has resumed full manufacturing operations of BioThrax after completing its internal manufacturing investigation of foreign particles in two BioThrax lots. The problem came to light on January 28, 2015, during standard quality inspections. Consequently, no BioThrax deliveries were made in the first quarter.

Now that the investigation has been completed and manufacturing activities have resumed, the company has re-affirmed its previous forecast of total revenues of $510 to $540 million, driven by growth in BioThrax sales which are anticipated to be between $270 to $285 million.

For the first quarter of 2015, the company expects total revenues of $62-$64 million and a net loss of $21-$23 million.

EBS closed Wednesday's trading at $29.84, down 0.43%.

Genocea Biosciences Inc.'s (GNCA) phase 2a human challenge study of GEN-004 for the prevention of infection by all serotypes of pneumococcus has completed enrollment with 98 healthy adult subjects at one site in the United Kingdom. The company anticipates reporting top-line data from this study in the fourth quarter of 2015.

Streptococcus pneumoniae, also known as pneumococcus, is a major cause of infectious disease-related death worldwide.

GNCA closed Wednesday's trading at $12.48, down 1.81%.

The new physician reports of clinical effect and safety of GW Pharmaceuticals plc's (GWPH) investigational cannabidiol product candidate, Epidiolex, in the treatment of children and young adults with treatment-resistant epilepsy have revealed promising data.

According to the findings, Epidiolex treatment was associated with meaningful reductions in seizures across a range of childhood epilepsies as well as a sustained response over a 6 month period and a 90% retention rate.

Justin Gover, the company's CEO said, "The findings fully support our decision to advance Epidiolex into Phase 3 placebo-controlled, double-blind trials in Dravet syndrome and Lennox-Gastaut syndrome and we look forward to advancing these trials rapidly during 2015."

GWPH closed Wednesday's trading at $113.04, down 0.04%.

Isis Pharmaceuticals Inc. (ISIS) has announced positive data from an ongoing open-label extension study of ISIS-TTRRx in patients with familial amyloid polyneuropathy.

The familial amyloid polyneuropathy patients completing the ongoing phase 3 study are eligible to enroll in this open-label extension study in which all patients receive ISIS-TTRRx.

An analysis conducted on the first group of patients to reach three months of treatment in the open-label extension study showed a reduction in transthyretin protein levels up to 92 percent with a median reduction of 78 percent compared to patients' baseline TTR levels at entry into the phase 3 study, noted the company.

ISIS closed Wednesday's trading at $66.26, down 0.05%. In after hours, the stock gained 1.42% to $67.20.

Natus Medical Inc. (BABY) has reported better-than-expected results for the first quarter ended March 31, 2015, and increased its revenue and earnings guidance for the full year 2015.

On a non-GAAP basis, the company's net income was $10.3 million or $0.31 per share on revenue of $89.4 million in Q1, 2015. This compares with non-GAAP net income of $8.3 million or $0.26 per share and revenue of $85.6 million in Q1, 2014. Analysts polled by Thomson Reuters expected earnings of $0.29 per share and revenue of $88.93 million.

Looking ahead, the company increased its non-GAAP earnings guidance for the full year 2015 and now expects to report non-GAAP earnings per share of $1.47 to $1.51, an increase from previous guidance of $1.42 to $1.46. Full year 2015 revenue guidance was increased to $376 million to $378 million compared to previous guidance of $373 million to $375 million.

BABY touched a new high of $42.96 on Wednesday before closing the day's trading at $41.64, up 4.28%.

OncoMed Pharmaceuticals Inc. (OMED) has initiated dosing in a phase 2 clinical trial of Demcizumab in patients with first-line metastatic pancreatic cancer. The study, dubbed YOSEMITE, will enroll about 200 patients.

In February of this year, the company initiated dosing in a phase 2 trial of Demcizumab for the treatment of non-small cell lung cancer. This trial, known as DENALI, is also designed to enroll approximately 200 patients.

The safety analysis associated with the two studies - YOSEMITE and DENALI - by an independent data safety monitoring board is expected to occur in late 2015/early 2016. OncoMed is eligible to achieve a $70 million milestone from Celgene Corp. (CELG) for successful completion of an interim safety analysis associated with the two trials.

OMED closed Wednesday's trading at $25.81, up 1.22%.

Ultragenyx Pharmaceutical Inc. (RARE) touched a new high on Wednesday following positive data from an investigator-sponsored trail of Triheptanoin (UX007) for the treatment of movement disorders associated with glucose transporter type-1 deficiency syndrome.

Glucose transporter type-1 deficiency syndrome, or Glut1 DS, is a severely debilitating disease characterized by seizures, developmental delay, and movement disorder.

Based on the study results, Ultragenyx intends to initiate a confirmatory randomized double-blind placebo-controlled study of Triheptanoin in the Glut1 DS movement disorder phenotype and anticipates discussions with the FDA on final study design details in the second half of 2015.

RARE touched a new high of $69.67 on Wednesday before closing the day's trading at $66.96.