On 9/21/09, Neuralstem (AMEX: CUR) ($2.50, +34%, heavy volume) announced that the FDA has approved its Investigational New Drug (IND) application to commence a Phase I trial to treat Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig’s disease) with its spinal cord stem cells. The Company’s patented technology has the ability to produce neural stem cells of the human brain and spinal cord in commercial quantities and the ability to control the differentiation of these cells.
CUR is the first company to commence a stem cell trial to treat ALS, which will evaluate the safety of the Company’s cells, the surgical procedures, and the devices required for multiple injections of these stem cells directly into the grey matter of the spinal cord. This trial will ultimately consist of 18 ALS patients with varying degrees of the disease. The FDA has approved the first stage of the trial, which consists of 12 patients who will receive 5-10 stem cell injections in the lumbar area of the spinal cord. The patients will be examined at regular intervals post-surgery, with final review of the data in about 24 months.
On 5/12/09, Questcor Pharma (NASDAQ: QCOR) ($6.03, -10%, heavy volume) received notice from the FDA that in order for its supplemental New Drug Application (sNDA) seeking approval to market H.P. Acthar Gel (repository corticotropin injection) for the treatment of infantile spasms (IS) to be considered a Complete Response, QCOR must perform additional statistical analyses relating to data from one study within the filing and submit this data to the Agency.
The H.P. Acthar Gel sNDA is a previously submitted application, but on 2/24/09 QCOR announced that the FDA asked them to reformat the sNDA and resubmit before accepting for review to expand use. QCOR resubmitted the sNDA on 3/17/09, but the FDA requested additional statistical analyses relating to data from one secondary study within the filing. On 9/21/09, QCOR announced its plans to submit a sNDA for H. P. Acthar Gel (repository corticotropin injection or Acthar) for the treatment of IS in October. This notification follows the completion by the Company of additional statistical analyses requested by the FDA. These analyses were conducted on data from one supportive study within the Company's filing.
On 9/21/09, Valeant Pharma (NYSE: VRX) ($26.50, unchanged, light volume) announced that along with partner Glaxo (NYSE: GSK) its plans to submit a NDA to the FDA on or before 10/23/09 and a Marketing Authorization Application (MAA) to the EMEA on 10/30/09 for retigabine, which is a first-in-class neuronal potassium channel opener for the adjunctive treatment of partial-onset seizures in adult patients with refractory epilepsy. It is an investigational compound that has a novel mechanism of action (MOA) that is distinct from other antiepileptic drugs (AEDs) currently on the market.
Retigabine has shown robust efficacy and safety as demonstrated in two large completed Phase 3 trials conducted in patients with refractory epilepsy receiving treatment with up to three antiepileptic drugs (AEDs). Valeant also announced that three retigabine modified release technologies will be assessed in a Phase 1 clinical study in September in order to evaluate new formulations to achieve a once- or twice-daily dosing regimen.
On 9/21/09, Impax Labs (NASDAQ: IPXL) ($8.90, unchanged, light volume) reported positive results from a recently completed Phase 2 trial of its late-stage Parkinson’s Disease (PD) drug candidate IPX066. In this cross-over trial, IPX066 reduced subjects’ “off” time during waking hours by 2 hours compared to standard of care treatment, Sinemet (3.8 hours of “off” time for IPX066 vs. 5.8 hours for Sinemet) which was statistically significant. Detailed results from this study will be presented at the World Congress on PD and Related Disorders December 16-19. Impax is currently enrolling patients in the Phase 3 APEX-PD study of IPX066 in levodopa naive PD patients in North America and Europe with plans to commence a second Phase 3 trial of the compound in patients with advanced PD patients by early 2010.
Disclosure: No positions.