Friday March 16, 8:03 am Eastern Time
Press Release
SOURCE: Genzyme General
Genzyme and Diacrin Report Preliminary Results
From Phase 2 Trial of NeuroCell(TM)-PD
CAMBRIDGE, Mass. and CHARLESTOWN, Mass., March 16 /PRNewswire/ --
Genzyme General (Nasdaq: GENZ - news), a division of Genzyme Corp., and Diacrin Inc.
(Nasdaq: DCRN - news) announced today that they have completed a preliminary analysis
of results from the Phase 2 clinical trial of NeuroCell(TM)-PD. This double- blind,
randomized, placebo-controlled trial evaluated the safety and efficacy of NeuroCell-PD for
use in treating patients with Parkinson's disease. NeuroCell-PD is a cellular approach that
involves the transplantation of porcine fetal neural cells to attempt to replace cells lost to
disease.
The clinical trial enrolled 18 patients with severe Parkinson's disease at three medical centers
in the United States. Ten patients received NeuroCell- PD transplants and eight patients
received placebo surgery. Each of the treated patients received a total of approximately 48
million cells transplanted on both sides of the brain. Treated patients were given cyclosporine
to suppress the immune system and attempt to prevent rejection of the cells.
Patients were evaluated at baseline and at three-month intervals to at least 18 months
following surgery using a broad range of clinical measurements. The trial's primary endpoint
was the difference between the treated group and the control group in the change in total
score on the Unified Parkinson's Disease Rating Scale (UPDRS) in the off state (more than
12 hours since last medication) obtained pre-operatively and 18 months after surgery. The
UPDRS is a validated clinical instrument that has been used widely in clinical trials in
Parkinson's disease, including those involving neurotransplantation. Both the treatment group
and the control group, on average, showed improvement from baseline. However, there was
no difference between the treatment and the control group.
Four secondary end points were measured. At 12 months, patients in the treated group
showed a reduction in the percentage of waking hours spent with dyskinesias when
compared with the control group. On average, no significant differences were observed
between the groups on clinical measurements of motor skills, investigator global evaluations,
and percentage of waking hours spent in the off state. An analysis of results for the trial's
tertiary endpoints is ongoing.
Four out of ten patients in the treatment group experienced serious adverse events, as did five out of eight patients in the control
group. The analysis conducted to date shows that there was a higher incidence of adverse events associated with
immunosuppressants (such as hair growth, elevated creatinine, and hypertension) among treated patients. These events are
consistent with those reported for other patients on immunosuppression, such as patients undergoing solid organ transplants. A
higher incidence of cancer among immunosuppressed patients has been reported in the clinical literature. In the Phase 2 trial of
NeuroCell-PD, occurrences of cancer were reported less frequently among treated patients (2 out of 10) than among placebo
patients (4 out of 8). To date, all patients have tested negative for porcine endogenous retrovirus, and there were no reports of
other treatment- related infections. No serious adverse events that involved disabling dyskinesia were reported.
``Although we are encouraged by the improvement of some patients in this trial who received NeuroCell-PD, we need to better
understand the basis for these improvements so that they can be extended to future trials of cell transplantation for Parkinson's
disease,'' said Thomas H. Fraser, Ph.D., president and chief executive officer of Diacrin.
Genzyme and Diacrin expect to present detailed results from the Phase 2 clinical trial of NeuroCell-PD at a forthcoming
scientific meeting and to submit the results for publication in a peer-reviewed journal. The companies expect to complete
additional analysis of the Phase 2 data as quickly as possible and to reach a decision within a few months whether to proceed
with additional clinical studies of NeuroCell-PD.
``We were hopeful that this trial would provide clear evidence that we had a product that could help Parkinson's patients,'' said
Henri A. Termeer, chairman and chief executive officer of Genzyme Corp. ``We are proud of the way this trial was designed
and conducted and grateful to those who participated in it. We will continue to review and evaluate the results to determine
whether we can expect to further develop a product that meets the standards we have established for all of our products.''
Based on these results and because additional analysis is required, a Phase 3 clinical study of NeuroCell-PD will not be initiated
before June 30, 2001. Under the terms of a Genzyme transfer agreement, Genzyme Biosurgery (Nasdaq: GZBX - news) will
be required to return a $20 million milestone pre-payment plus interest to Genzyme General within 90 days following June 30.
The payment may be made using cash or designated shares of Genzyme Biosurgery stock.
In April 1999, Genzyme Corp. transferred its 50 percent interest in the NeuroCell joint venture with Diacrin to Genzyme
General from Genzyme Tissue Repair, which is now part of Genzyme Biosurgery. In exchange, Genzyme Tissue Repair
received $25 million from Genzyme General, of which $5 million was non- refundable and $20 million was a prepayment
related to the initiation of a Phase 3 trial of NeuroCell-PD by June 30, 2001.
NeuroCell Program Background
Prior to conducting the Phase 2 trial, Genzyme and Diacrin completed an open-label Phase 1 clinical trial of NeuroCell-PD in
12 patients. No specific safety concerns related to treatment have been identified. Safety and clinical data will continue to be
collected on these patients for up to five years. Additionally, all patients enrolled in the study will be followed for safety in a
life-long registry.
Genzyme and Diacrin established a joint venture in 1996 to develop cell therapies for the treatment of Parkinson's and
Huntington's diseases.
Genzyme General develops and markets therapeutic products and diagnostic products and services. Genzyme General has four
therapeutic products on the market and a strong pipeline of products in development focused on the treatment of genetic
disorders and other chronic debilitating diseases with well-defined patient populations. Genzyme General is a division of the
biotechnology company Genzyme Corporation.
Diacrin is developing cell transplantation technology for treating human diseases that are characterized by cell dysfunction or cell
death and for which current therapies are either inadequate or nonexistent. Products under investigation include: NeuroCell- PD
for Parkinson's disease; porcine neural cells for stroke, focal epilepsy and intractable pain; porcine spinal cord cells for spinal
cord injury; NeuroCell(TM)-HD for Huntington's disease; porcine liver cells for acute liver failure; human liver cells for
cirrhosis; human muscle cells for cardiac disease; and porcine retinal pigment epithelial cells for macular degeneration. Diacrin is
developing NeuroCell-PD and NeuroCell-HD in a joint venture with Genzyme Corporation.
This press release contains forward-looking statements based on current expectations, including statements concerning: the
preliminary results of the phase 2 trial of NeuroCell(TM)-PD; the potential use of trial data in future trials for Parkinson's
disease; plans to conduct additional analysis of the trial data; plans to present the trial results and submit the results for
publication; and the expected timing of the final analysis of the phase 2 trial data and a determination as to whether to proceed
with additional clinical studies; the refund of $20 million plus interest by Genzyme Biosurgery to Genzyme General; and the
potential election by Genzyme Biosurgery to use designated shares of Genzyme Biosurgery Division Common Stock to make
such payment. Actual results may differ materially depending on many factors, including without limitation: the actual timing and
results of the final analysis of phase 2 trial safety and efficacy data; the feasibility of further development activities; the continued
funding of the joint venture; decisions by the FDA and other regulatory authorities; and the risks and uncertainties described in
the reports filed by Diacrin and Genzyme with the Securities and Exchange Commission under the Securities Exchange Act of
1934, as amended, including without limitation Diacrin's 2000 Annual Report on Form 10-K and Exhibit 99.2 to Genzyme's
1999 Annual Report on Form 10-K, as amended. Genzyme General Division Common Stock and Genzyme Biosurgery
Division Common Stock are both series of common stock of Genzyme Corporation. Therefore, holders of Genzyme General
Division Common Stock and Genzyme Biosurgery Division Common Stock are subject to all of the risks and uncertainties
described in the aforementioned reports filed by Genzyme.
Genzyme® is a registered trademark of Genzyme Corporation. NeuroCell(TM) is a trademark of Diacrin/Genzyme LLC. All
rights reserved.
Genzyme's releases are on the World Wide Web at genzyme.com. They are also available from Genzyme's
fax-on-demand service at 1-800-436-1443 in the United States and 1-201-521-1080 elsewhere.
SOURCE: Genzyme General |
