Silicon Investor (SI) -- The First Internet Community

STOCKTALK

We've detected that you're using an ad content blocking browser plug-in or feature. Ads provide a critical source of revenue to the continued operation of Silicon Investor. We ask that you disable ad blocking while on Silicon Investor in the best interests of our community. If you are not using an ad blocker but are still receiving this message, make sure your browser's tracking protection is set to the 'standard' level.

Biotech / Medical : Pharmion -- Ignore unavailable to you. Want to Upgrade?

To: tuck who wrote (1)	11/17/2003 3:53:00 PM
From: tuck	Read Replies (1) \| Respond to of 22

From the S-1: >>In addition to the efficacy data from the study, several quality of life parameters were also studied and showed statistically significant improvement in the azacitidine patient group compared to patients receiving best supportive care. In particular, azacitidine patients showed statistically significant improvement over time in fatigue, physical functioning and shortness of breath. In patients receiving best supportive care who showed stable or worsening quality of life prior to crossover to azacitidine, statistically significant improvement occurred in fatigue, physical functioning, shortness of breath and general well-being after crossover to azacitidine. In , we plan to submit an NDA for Vidaza, under Subpart H of the New Drug Application regulations, which allows for conditional approval of a drug to treat serious or life-threatening diseases based on a surrogate endpoint, a more easily measured indicator of the effect of a drug on the course of a disease, as long as the drug provides a meaningful therapeutic benefit over existing treatments. In addition, under Subpart H a drug can be approved on the basis of one Phase I or Phase II study, conditioned upon the sponsor agreeing to complete a confirmatory Phase IV study. Since there are currently no drugs approved for the treatment of MDS and best supportive care is the only existing treatment, the Subpart H approval option is available. The E.U. has a set of rules for drugs that target unmet medical needs where the target condition is rare, enabling the regulatory authorities to authorize an application for such drugs on an “exceptional circumstances” basis. Our planned submission is based upon the previously cited Silverman study for the treatment of MDS that was conducted by the Cancer and Leukemia Group B, or CALGB, and the NCI and two supportive Phase II CALGB/NCI studies. Due to the fact that the Phase III CALGB study was not prospectively designed as a registration study, over the last 24 months we have engaged in a lengthy process of collecting and analyzing data from the CALGB studies to confirm their published results and to compile the information needed to submit an NDA for Vidaza in MDS. This activity involved visiting nearly 75 treatment centers and accessing and auditing nearly 400 patient records from the CALGB studies. In addition, working with third party manufacturers, we have successfully developed a fully scaled up manufacturing process for the active ingredient and finished product. As required under Subpart H in the U.S., we are initiating a comparative confirmatory clinical trial that will examine survival outcomes and other secondary end points, using a multicenter, randomized, open-label, parallel group format. The aim of this study is to compare the effect of Vidaza plus best supportive care against conventional care regimens plus best supportive care on survival in MDS patients. As a result of the fact that this study is global in nature and MDS treatment practices vary among countries, there will be three comparative conventional care treatments: best supportive care only; low dose cytarabine, plus best supportive care; or standard chemotherapy, plus best supportive care. This design takes into account the actual “conventional care” used to treat MDS patients in each country targeted for trial participation and should also help to enhance timely enrollment. The study will recruit over 330 patients and will be one of the largest studies to date in this disease. The primary objective of this confirmatory study is to look at survival in these MDS patients. All other relevant endpoints, such as time to transformation to AML, time to relapse after complete remission or partial remission, disease progression, hematological status (peripheral blood counts, need for platelet and red blood cell transfusions and hematological response), episodes of infections requiring intravenous antibiotics and safety parameters will be assessed. Pharmacia has a one-time option that expires 75 days after our filing of our NDA for Vidaza with the FDA under which Pharmacia can obtain the right to participate with us in both the development of the product and its commercialization on a 50:50 basis. Were it to exercise this right, we would negotiate a mutually acceptable joint development and promotion agreement and Pharmacia would be obligated to reimburse us for fifty percent of the development costs that we incurred in connection with Vidaza through the date of the execution of that agreement.<< Cheers, Tuck