Biotech FDA and Clinical Trial Calendar Update

by: Mike Havrilla

Below is a summary of updates to the FDA decision calendar since my last article, which includes a total of 90 entries through Sunday:

1.) Nuvo Research (OTCPK:NRIFF) NDA re-submission for Pennsaid - A topical NSAID for arthritis pain/inflammation - along with Paladin Labs.

2.) Hemispherx Biopharma (NYSEMKT:HEB) Ampligen (Poly I: Poly C12U) NDA (three month PDUFA date delay announced 2/18/09 as additional data was submitted within three months of original decision date. A decision is now expected by 5/25/08 for the Company's resubmission of its Ampligen NDA, which is designated as an Orphan Drug for the treatment of chronic fatigue syndrome, which has no FDA-approved treatments on the market. Click here for a review article on the decades-long development of Ampligen and HEB will be presenting new clinical data at a medical conference on March 13.

3.) Schering-Plough (SGP): Saphris (asenapine) sublingual tablets response submitted 2/20/09 to the complete response letter from January 2009 for the acute treatment of schizophrenia in adults and for the acute treatment of manic or mixed episodes associated with bipolar I disorder in adults as mono-therapy. SGP gave guidance for finalizing labeling discussions with FDA for approval, but no timeline was given for a final decision by the agency.

4.) Takeda Pharma (OTC:TKPHY) received FDA approval for the first new gout treatment in decades, which will be marketed as Uloric (febuxostat).

5.) Savient Pharma (OTC:SVNT) announced a three month delay in the review of its gout drug candidate Krystexxa (pegloticase) (formerly Puricase) with a new PDUFA date of 7/30/09 and a FDA Advisory Panel scheduled for 3/5/09. SVNT submitted additional data to the agency to improve its chances for approval, including a risk management plan, a bi-weekly dosing request, and more details on the cardiovascular side effects of the drug, which remain as the major overhang on the drug and focus of the upcoming advisory panel meeting.

6.) Genzyme (GENZ) expects to receive FDA approval by the end of next week for Myozyme produced at the 2,000 liter bio-reactor scale, which will be marketed in the U.S. as Lumizume.

Below is a summary of updates to the Clinical Trial Results Calendar and pending FDA New Filings or Re-Submission with a total of 127 entries through Sunday:

1.) Gilead Sciences (NASDAQ:GILD): This past week, the FDA reiterated its stance on that GILD must conduct additional clinical trials before re-submitting the Aztreonam Lysine (inhaled formulation) NDA and there are currently two ongoing studies for the treatment of an opportunistic infection (P. aeruginosa) in cystic fibrosis patients. GILD expects to provide clarity on timelines for NDA re-submission at a later date.

2.) Cell Therapeutics (NASDAQ:CTIC) and Spectrum Pharma (NASDAQ:SPPI): After the market close on Friday, CTIC announced that it would sell its stake in the 50/50 joint venture with SPPI (RTI Oncology) for $18M cash. The joint venture was for cancer drug Zevalin, which has a priority review PDUFA date of 4/2/09 to expand its label to include consolidation therapy for follicular B-cell non-Hodgkin's lymphoma if a response is achieved with first-line therapy to increase the market size from 1,000 to 18,000 patients for SPPI.

The move by CTIC provides a capital lifeline (a recent SEC filing showed only enough cash to operate through the end of this month) and reduces cash burn by $15M annually to around $4M per month. CTIC will now focus on the development of lead cancer drug candidates pixantrone and Opaxio (which is under review for potential 2H09 approval in Europe and has pending Phase 3 data expected 2H09 for ovarian cancer).

CTIC expects to begin submission of a rolling NDA with a priority review (six month) request during the current quarter for pixantrone and reported additional Phase 3 clinical trial data this past week. Last November, pixantrone achieved its primary efficacy endpoints in a Phase 3 trial and the new data includes a rapid response time and favorable safety profile compared to standard chemo and anthracyclines (a class of cancer drugs which result in chronic toxicities such as heart damage which limit their use).

CTIC is also evaluating strategic alternatives for its Italian R&D facility, which would further reduce its burn rate. Agreements with Novartis (NYSE:NVS) for pixantrone and Opaxio provide for $17.5M ($7.5M licensing option for NVS + approval milestone payment for pixantrone) and $25M (Opaxio approval milestone in Europe).