March 21, 2016
By Mark Terry, BioSpace.com Breaking News Staff
Recent positive regulatory decisions or clinical trial data releases have been good news for four biopharmaceutical companies. Here’s a look at who they are and where they stand.
Salisbury, UK-based GW Pharmaceuticals (GWPH) recently announced positive data from its Phase III study of Epidiolex (cannabidiol) to treat Dravet syndrome. The drug met the primary endpoint of a statistically significant drop in convulsive seizures compared to placebo. Dravet syndrome is a rare type of epilepsy with no currently approved treatments in the U.S.
“These data demonstrate that Epidiolex delivers clinically important reductions in seizure frequency together with an acceptable safety and tolerability profile,” said Orrin Devinsky, of New York University Langone Medical Center’s Comprehensive Epilepsy Center, in a statement, “providing the epilepsy community with the prospect of an appropriately standardized and tested pharmaceutical formulation of cannabidiol being made available by prescription in the future.”
GW Pharmaceuticals popped from $38.46 on Mar. 11 to $84.71 on Mar. 14 at the news. Shares are currently trading for $77.42.
Celator Pharmaceuticals (CPXX), with headquarters in Ewing, N.J. and Vancouver, British Columbia, announced on Mar. 14, positive results from its Phase III trial of Vyxeos (daunorubicin) in patients with high-risk (secondary) acute myeloid leukemia (AML) compared to the standard care regimen of cytabarine and daunorubicin, also known as 7+3. The trial’s primary endpoint was a statistically significant improvement in overall survival.
Celator jumped from $1.68 on Mar. 14 to $9.38 on Mar. 16. Shares are currently trading for $9.57.
New Haven, Conn.-based BioBlast Pharma (ORPN) announced on Mar. 16 that its HOPEMD Phase II trial in patients with oculopharyngeal muscular dystrophy (OPMD), which evaluated the safety and tolerability of trehalose, was shown to be safe and well-tolerated. The drug also showed improvements in dysphagia and muscle strength and function as secondary efficacy endpoints.
On Mar. 17, the company announced it had entered into definitive securities purchase agreements with two healthcare-focused institutional investors. They plan to issue about 2.16 million ordinary shares at a purchase price per share of $3.10 in a registered direct offer.
BioBlast traded at $2.64 on Mar. 15, jumped to $4.08 on Mar. 16, and are currently trading for $2.77.
Washington, Penn.-based Vitae Pharmaceuticals (VTAE) announced on Mar. 16 positive topline results from its Phase IIa proof-of-concept trial of VTP-43742 in patients with psoriasis. The drug showed clear efficacy, with patients receiving 350 mg dose achieving a 24 percent decrease in Psoriasis Area Severity Index (PASI) score compared to placebo. The patients in the 700 mg dose group had a 30 percent PASI score reduction.
“We believe these data validate ROR?t as an exciting and novel therapeutic target for the treatment of psoriasis and other autoimmune disorders,” said Jeff Hatfield, Vitae’s president and chief executive officer, in a statement. “While the autoimmune market is currently dominated by injectable antibody therapy, we believe VTP-43742 has the potential to expand utilization of oral therapy in a variety of autoimmune disorders, such as psoriasis, psoriatic arthritis, rheumatoid arthritis, multiple sclerosis and inflammatory bowel disease with an effective, safe and well tolerated, once-a-day agent.”
Vitae has shown some recent volatility. Shares traded on Mar. 2 for $10.85, dropped to $4.69 on Mar. 4, then dropped further to $4.11 on Mar. 16. Shares increased to $6.79 on Mar. 17, and are currently trading for $7.71.
