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Biotech / Medical : XOMA. Bull or Bear? -- Ignore unavailable to you. Want to Upgrade?

To: opalapril who wrote (8923)	3/3/1999 11:31:00 PM
From: Edscharp	Read Replies (1) \| Respond to of 17367

Xoma's frequently asked questions (updated 2/99) For everyone's convenience I've included the recent updated FAQ's at Xoma's website: xoma.com NEUPREX®: Meningococcemia update 2/99 When will the NEUPREX® Phase III meningococcemia trial complete enrollment? (2/99) We hope to complete enrollment soon. An independent Data Safety Monitoring Board (DSMB) is monitoring the trial to assure that we achieve sufficient statistical power to reliably observe a benefit. The DSMB will meet again in the first quarter to advise us about stopping. Since our objective is to complete a successful trial, we are relying on their guidance as to when to stop enrolling patients. Why didn't the trial end in 1998? (2/99) We previously believed that we would reach a prespecified target total mortality number by the end of 1998. However, when the DSMB met in September 1998, they advised us that we should continue the trial into 1999 in order to reach that figure. What did the DSMB recommend in September 1998? (2/99) They indicated to us that they saw no safety issues and recommended enrollment continue. They also recommended an additional meeting early in 1999 to review the data before concluding enrollment. Was the early 1999 additional review part of the original statistical plan for the trial? (2/99) The DSMB may request additional meetings to review data at timepoints they see as important for the successful conclusion of the trial. This meeting gives the DSMB the opportunity to advise us before we close enrollment. This final review does not involve a formal statistical efficacy analysis. Does this mean NEUPREX® is performing poorly? (2/99) There are no indications from the DSMB that this is the case. They've already performed three safety/futility interim analyses, and there has been no suggestion of a problem with the drug. However, we will not know the actual results until the study is completed and unblinded. What is a DSMB? What do they do? (2/99) A Data Safety Monitoring Board is an independent body of experts who review data from ongoing clinical trials to assure the safety of the patients enrolled. They are not a regulatory body. The DSMB may advise the sponsoring company about study administration, possible safety issues or unexpected efficacy results. How many patients have been enrolled so far? How many have died? (2/99) Patient enrollment has exceeded 360 patients. Because of the sporadic nature of the disease, future enrollment rates are unpredictable. At the request of the DSMB, we have not disclosed the mortality numbers. How many sites are participating in the meningococcemia pivotal trial? (2/99) There are a total of 25 study centers in the United States and the United Kingdom. When are the meningococcemia Phase III results expected? (2/99) After enrollment is concluded, there is a 90-day follow-up period after which the data can be collected, verified and then unblinded. Do you have an estimated date that the NEUPREX® data will go to the FDA for final review? (2/99) Once data are unblinded, we will need to complete the analysis and the final study report before filing with the FDA. If we complete enrollment in the first quarter, and assuming the data are positive, our target is to complete the BLA (Biologics License Application) filing to FDA in autumn 1999. The actual timing will depend on a number of factors which we can't predict at this stage. Does accruing patients in both the US and the UK affect regulatory filings here and there? (2/99) No. Both the US and European regulatory agencies have agreed to a single, multicenter, multinational trial for license application. Once you apply, how long will it take the FDA to approve? (2/99) A drug that offers clinical benefit in a rare and often fatal disease affecting predominately healthy children is expected to receive priority within the FDA and the European regulatory agencies. What is Orphan Drug status? Is this the same as fast-track designation? (2/99) The Orphan Drug Act provides incentives for manufacturers to develop drugs for rare diseases or conditions which have small markets. Orphan Drug status grants seven years of US market exclusivity, provides for certain tax benefits and waives user fees usually paid by the sponsoring company under the Pharmaceutical Developers User Fee Act. It is not a fast-track designation. We have pursued and will continue to pursue fast-track review options. When will you file with the European regulatory agency? (2/99) We plan to file for European approval in parallel with our US filing. Is the pivotal trial placebo-controlled? How many patients will be in the control group? What does "double-blind" mean? (2/99) The meningococcemia Phase III study is double-blinded, randomized and placebo-controlled. Patients are randomly assigned to either receive NEUPREX®, or an inert substance (a "placebo"). Approximately equal numbers of patients will be in each group. Both groups also receive the same standard care including antibiotics, often in an intensive care setting. "Double-blinded" means that neither the treating physician nor the sponsoring company knows which patients receive the drug and which receive placebo until the study is concluded and the blind broken. We will not know which patients received NEUPREX® until after the trial is complete and the data are checked and verified. Even though the meningococcemia pivotal study is double-blinded and placebo-controlled, there must be some positive signs in the overall test group. Are there any signs that the drug is working? (2/99) In double-blinded trials neither the sponsoring company nor the doctors treating patients know who received NEUPREX® or placebo. Why are placebo-controlled trials used? (2/99) Blinded, controlled trials are the accepted scientific approach to demonstrate the efficacy of new drugs while avoiding bias that can result when clinical benefits are erroneously attributed to a drug in the absence of a comparison group who did not receive the drug (AKA the "placebo effect"). Most regulatory authorities and clinicians judge the safety and efficacy of new therapies on the results of placebo-controlled studies. The scientific community and the voting public supports this approach as the accepted way to provide for safe and effective drugs. Given the deadly and mutilating nature of meningococcemia, why is a placebo group considered necessary? (2/99) Our goal is to make the product available in the shortest time possible with the highest possible confidence in its effectiveness. XOMA works closely with the FDA to design trials that meet accepted scientific standards for meaningful and well-controlled studies. Based on our discussions with FDA and medical investigators, a placebo-controlled Phase III study is necessary to objectively demonstrate clinical efficacy. It is important to note that the placebo group receives the same high standard of intensive care as the treatment group (treatment plus standard of care vs. placebo plus standard of care).