Strategische herpositionering.
BERKELEY, Calif., Aug 15, 2006 (BUSINESS WIRE) -- XOMA Ltd. (Nasdaq:XOMA) today unveiled its strategy and provided an update on the status of its efforts to advance NEUPREX(R) (opebacan) toward regulatory approval in Europe and the US in multiple disease indications.
"Our strategy to find clear approval opportunities for NEUPREX(R) is beginning to demonstrate some meaningful progress. We have ongoing activities in a number of different indications based on NEUPREX(R)'s established activity as a potent neutralizer of bacterial endotoxin," said Jack Castello, XOMA's chairman of the board, president and chief executive officer. "With recent accomplishments here and in Europe, we are pleased to provide our shareholders with an update on our strategy for the approval of NEUPREX(R)."
European Strategy
In spite of the availability of improved vaccines, meningococcemia remains an ongoing life-threatening problem with no meaningful advances in therapy in over a decade. In Europe, XOMA is implementing a two-part regulatory strategy that leverages the large body of clinical data, including phase III studies, that already exists for NEUPREX(R) in meningococcemia. The two elements of this strategy are pursuit of an Orphan Drug Designation, and a marketing authorization of NEUPREX(R) in meningococcemia under the "exceptional circumstances" mechanism established by the European Medicines Agency (EMEA).
Orphan Medicinal Product Designation
On July 14, 2006, the EMEA's Committee for Orphan Medicinal Products (COMP) issued a positive opinion that opebacan (NEUPREX(R)) be granted Orphan Medicinal Product designation in meningococcal disease. XOMA expects the European Commission formally to designate opebacan (NEUPREX(R)) as an orphan medicine in the next two to three months. A number of important benefits would accrue to XOMA under an orphan designation for NEUPREX(R):
-- Free formal scientific advice through the Scientific Advice Working Party
-- Reduced marketing authorization fees
-- Ten year market exclusivity upon marketing authorization
Approval Under Exceptional Circumstances
In parallel with XOMA's efforts to obtain an Orphan Medicinal Product designation, XOMA has held discussions with the EMEA concerning NEUPREX(R)'s potential approvability for meningococcemia under exceptional circumstances. The exceptional circumstances mechanism provides an approval pathway for marketing authorization applications where the applicant is unable to provide comprehensive data on the efficacy and safety under normal conditions of use. Approval under exceptional circumstances may include post-approval commitments to allow for periodic reassessment of the safety of the product.
The EMEA has indicated that the information already available about NEUPREX(R) and the circumstances for obviating extensive additional clinical trials in this rare disease fit within the intent of this European legislation. Based on this understanding, XOMA is now completing its regulatory assessment for this approval pathway for NEUPREX(R) in Europe. XOMA has also held meetings with key opinion leaders and confirmed their continuing interest in the clinical use of opebacan based on its extensive safety database, the results from the phase III clinical trial in meningococcemia and the ongoing unmet therapeutic need for this life-threatening disease.
The next step in the exceptional circumstances regulatory process is to obtain formal scientific advice from EMEA concerning the suitability of the existing NEUPREX(R) data in supporting a marketing authorization application. XOMA plans to complete its regulatory assessment for Europe in the first quarter of 2007.
US Strategy
Because US regulatory pathways differ from Europe's, XOMA's NEUPREX(R) strategy in the US is based on the identification of acute clinical indications in which: 1) endotoxin has been documented in the disease process, 2) the time from disease onset to administration of NEUPREX(R) is known and 3) administration of NEUPREX(R) can occur in time to have a meaningful clinical impact. XOMA has elected to proceed with a strategy that requires only a limited financial investment until convincing clinical benefit has been observed. In keeping with these criteria, XOMA currently is supporting two US investigator-initiated clinical trials in pediatric open heart surgery and severe burns, and will soon start a XOMA-sponsored trial at prominent academic medical centers in allogeneic hematopoietic stem cell transplantation (initially in donor bone marrow transplantation), pending approval of the Investigational Review Boards (IRB) at these institutions.
"Conducting clinical trials through these means allows XOMA to make progress in multiple indications for only a fraction of the cost of conventional studies," said Mr. Castello. "At the same time, we also gain valuable clinical data, which may provide sufficient rationale to justify larger XOMA-initiated follow-on studies and potential marketing licenses for NEUPREX(R)."
Pediatric Open Heart Surgery
Infants undergoing open heart surgery are at risk for endotoxin leakage from the gastrointestinal tract into the blood stream ("endotoxemia") due to their underlying heart disease and cardiac bypass during surgery. Endotoxemia has been documented to occur before, during and after surgery and is associated with increased post-operative complications and prolonged hospital stays for these infants. NEUPREX(R) is being evaluated for both safety and its ability to prevent endotoxin-induced post-operative complications.
The current Phase I/II trial, being conducted at Children's Hospital in Dallas, Texas, is expected to be completed in mid-2007. The design and nature of follow-on trials will be determined by the results of this trial.
Severe Burns
Severely burned patients suffer many complications, including late infections and death. Research at Parkland Burn Center in Dallas has indicated that many of these complications result from endotoxemia and early changes in the patient's immune status, both of which may be corrected by early administration of NEUPREX(R). A trial to assess NEUPREX(R)'s ability to prevent endotoxin-related complications has been initiated at Parkland Burn Center with the dosing of the first patient occurring in July of 2006. XOMA expects this trial to complete enrollment in 2007.
Hematopoietic Stem Cell Transplantation (HSCT)
Prior to receiving a transplant, candidates for donor bone marrow or other stem cell transplantation receive large doses of radiation or chemotherapy to prepare them for the transplant. This regimen disables their immune system and gastrointestinal tract for several weeks and leaves them susceptible to many complications, including endotoxemia and infections. In addition, endotoxemia has been shown to induce or worsen a complication called acute graft vs. host disease (aGvHD), which occurs in approximately 40% of donor stem cell transplant patients. Research indicates that NEUPREX(R) may help prevent or reduce the severity of aGvHD, as well as prevent or reduce infectious complications during the period when these patients are immuno-suppressed from their radiation or chemotherapy regimens.
XOMA is working with major HSCT centers in the US that have expressed interest in participating in this clinical trial and expects to start enrollment before the e