CBMG To Keep On Your Radar, CEMP Gets Funding, MACK On Fast Track

(RTTNews) - Bio Blast Pharma Ltd.'s (ORPN) lead product candidate Cabaletta has been granted Orphan Drug Designation by FDA for the treatment of Spinocerebellar Ataxia Type 3 (commonly known as SCA3 and Machado Joseph disease).

Cabaletta is under phase II study in Spinocerebellar Ataxia Type 3 in Israel. A phase 2/3 study of Cabaletta in treatment of Oculopharyngeal Muscular Dystrophy is also ongoing. Cabaletta also has orphan drug designation for Occulopharyngeal Muscular Dystrophy.

Bio Blast went public on the NASDAQ Global Market on July 31, 2014, priced at $11.00 per share.

ORPN closed Wednesday's trading at $4.65, down 1.27%.

Cellular Biomedicine Group Inc. (CBMG) is a stock to keep on your radar as the company has a couple of catalysts coming its way.

The company expects to present twelve month follow-up data analysis from its phase I/IIa clinical trial of lead regenerative medicine product candidate ReJoin for Knee Osteoarthritis on December 5, 2014 at the World Stem Cell Summit in San Antonio, Texas.

The six-month follow-up data analysis, announced in June of this year, concluded that ReJoin cell therapy for Knee Osteoarthritis patients is safe and revealed an increase in cartilage volume as early as three months after the therapy.

A multi-center phase IIb clinical trial for ReJoin is underway, with interim data scheduled for release in Q1 2015.

CBMG closed Wednesday's trading at $15.60, down 0.57%.

Cempra Inc. (CEMP) is all set to receive a funding of $16.0 million under a contract with Biomedical Advanced Research and Development Authority (BARDA) to cover the cost of a phase I study and start-up activities of a phase 2/3 study of Solithromycin in pediatric patients.

CEMP closed Wednesday's trading at $13.22, down 5.10%.

Clovis Oncology Inc.'s (CLVS) initial results from a phase II trial of Rucaparib, an investigational drug for the treatment of platinum-sensitive ovarian cancer, has demonstrated encouraging clinical activity and safety in both BRCA mutant and BRCAness patients.

In the trial, dubbed ARIEL2, the overall response rate was 70% in BRCA-mutant patients and 40% in BRCAness patients. (DNA repair deficiencies beyond BRCA are commonly referred to as "BRCA-like" or "BRCAness").

Rucaparib has also proven to be well-tolerated, with no drug discontinuations due to treatment-related adverse events in the trial, noted the company.

CLVS closed Wednesday's trading at $48.28, down 10.23%.

Immunovaccine Inc. (IMV.V) has received clearance from Health Canada to conduct a phase II clinical study of DPX-Survivac in patients with diffuse large B cell lymphoma.

The trial will enroll up to 24 patients, and the first patient is expected to be enrolled in the study in early 2015.

Merrimack Pharmaceuticals Inc.'s (MACK) investigational nanotherapeutic MM-398, also known as ''nal-IRI,'' has been granted Fast Track designation by FDA for the treatment of patients with metastatic adenocarcinoma of the pancreas who have been previously treated with gemcitabine-based therapy.

MM-398 recently completed a phase 3 trial in post-gemcitabine pancreatic cancer, and the company expects to initiate the NDA submission for MM-398 in 2014 with the goal of completing the NDA submission late in the first quarter or early in the second quarter of 2015.

MACK closed Wednesday's trading at $8.72, down 2.46%.

Synergy Pharmaceuticals Inc.'s (SGYP) phase II trial of SP-333 in patients with opioid-induced constipation has met the primary endpoint and demonstrated statistically significant improvement in mean change from baseline in the number of spontaneous bowel movements.

In the phase II trial, once-daily oral SP-333 was evaluated in three doses of 1.0, 3.0 and 6.0 mg against placebo in 289 patients.

SGYP closed Wednesday's trading at $2.84, down 4.05%.