For Immediate Release
September 14, 2000
Genzyme Contacts: Diacrin Contact:
Elizabeth Heller (media) Thomas H. Fraser, Ph.D.
617-761-8419 President and CEO
Sally Curley (Genzyme General investors) 617-242-9100
617-591-7140
Kristen Nally (Genzyme Tissue Repair investors)
617-252-7815
Diacrin/Genzyme Joint Venture and FDA Agree to Maximize Product Efficacy Data
in Ongoing NeuroCell(TM)-PD Blinded Phase 2 Clinical Trial
CAMBRIDGE, Mass. and CHARLESTOWN, Mass.-- Genzyme General (Nasdaq:
GENZ) and Diacrin Inc. (Nasdaq: DCRN) today announced that based on discussions
with the U.S. Food and Drug Administration (FDA) and clinical investigators,
they have collectively agreed to extend the blinded NeuroCell-PD phase 2
clinical trial for Parkinson's disease to 18 months from 12 months. The
extension is intended to enable investigators to maximize the amount of
long-term durability data for NeuroCell-PD, and to provide the best efficacy
data for potential product approval. The unblinding of this pivotal trial data
is expected to take place in March 2001.
"We are committed to the NeuroCell program and are very excited about
the potential of NeuroCell to benefit patients who suffer from the devastating
symptoms of Parkinson's disease," said Henri Termeer, president and chief
executive officer, Genzyme Corporation. "The decision by the joint venture, the
FDA, and our clinical investigators to extend the trial illustrates a collective
dedication to ensure the best data possible from the blinded phase 2 study. The
joint venture is near completion in preparing for the confirmatory phase 3 study
and will be ready to begin treating patients once the data is unblinded and a
decision to move forward is made."
Genzyme transferred its interest in the NeuroCell joint venture from
Genzyme Tissue Repair (Nasdaq: GZTR) to Genzyme General in April 1999. A
milestone payment, based on the initiation of a phase 3 clinical trial for
NeuroCell, is part of the terms of the transfer agreement.
To accommodate the extension of the pivotal phase 2 trial, Genzyme
Corporation will propose to its Board of Directors an extension of the first
milestone date for the NeuroCell program to June 30, 2001 from December 31,
2000. This will allow investigators time to complete and review the results of
the phase 2 clinical trial. Genzyme expects that its Board of Directors will
approve the milestone extension at its next regularly scheduled board meeting.
NeuroCell Clinical Trial Background
Preparations for the phase 3 confirmatory trial are moving ahead. The
FDA has reviewed the trial protocol and trial sites and investigators have been
selected. To date, the joint venture has received five Institutional Review
Board (IRB) approvals for the phase 3 trial protocol.
In May 2000, final study results from a phase 1 clinical trial for
NeuroCell-PD were presented and showed that all patients tolerated the treatment
well and a group of patients continued to show improvement 36 months after
surgery. No safety concerns related to treatment have been identified. Safety
and efficacy 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.
In October 1999, the FDA granted NeuroCell-PD Fast Track designation,
making the joint venture eligible to receive expedited review of an application
for NeuroCell-PD product approval.
Background on NeuroCell Transfer
In 1996, Genzyme and Diacrin, Inc. established Diacrin/Genzyme LLC, a joint
venture to develop cell therapies for the treatment of Parkinson's and
Huntington's diseases.
In April 1999, Genzyme Tissue Repair transferred the 50 percent
interest in the joint venture to Genzyme General based on a strategic decision
to focus on products and devices in orthopedics and burn care. Genzyme General
believes that NeuroCell-PD has shown early promise and is an excellent fit with
its specialty therapeutics product pipeline.
Under the terms of the transfer, Genzyme Tissue Repair received $25
million from Genzyme General in 1999, of which $5 million is non-refundable and
$20 million is a pre-payment related to the achievement of two milestones.
The two milestones are now: repayment of $20 million to Genzyme General
if Diacrin/Genzyme LLC does not initiate a phase 3 clinical trial of
NeuroCell-PD by December 31, 2000; repayment of $15 million to Genzyme General
if the phase 3 clinical trial is initiated by December 31, 2000 but
Diacrin/Genzyme LLC does not obtain FDA product approval of NeuroCell-PD prior
to June 30, 2004. Genzyme expects its Board of Directors to extend the December
31, 2000 milestone to June 30, 2001 to accommodate the extension of the phase 2
trial.
In addition to the advance cash payment already received, Genzyme
Tissue Repair will receive royalties of three percent on sales of any products
successfully developed and marketed by the joint venture.
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
development 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-HD(TM) 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.
Genzyme Tissue Repair is a leading developer of biological products and
devices for the treatment of orthopedic injuries such as cartilage damage, and
severe burns.
Genzyme General develops and markets therapeutic products and
diagnostic products and services. Genzyme General has three therapeutic products
on the market and a strong pipeline of products in development focused on the
treatment of rare genetic disorders.
Both Genzyme General and Genzyme Tissue Repair are divisions of the
biotechnology company Genzyme Corporation and have their own common stocks
intended to reflect their value and track their performance.
This press release contains forward-looking statements, including
statements about the expected timing of unblinding of trial data, plans to
propose a milestone deadline extension to the Genzyme Board of Directors, the
expected receipt and timing of approval of the milestone deadline extension by
Genzyme's Board of Directors, the potential benefits and promise of
NeuroCell-PD, the anticipated initiation of a phase 3 clinical trial, and
potential royalties. Actual results may differ materially depending on many
factors, including whether the joint venture decides to initiate a phase 3
clinical trial of NeuroCell-PD, the actual timing and content of a decision by
Genzyme's Board of Directors concerning the milestone deadline, the enrollment
rate for clinical trials, the actual timing and results of clinical trials, the
ability to demonstrate long-term safety and efficacy of the NeuroCell(TM)
products, the timing and content of submissions to and decisions by the FDA and
other regulatory authorities, the ability of Diacrin/Genzyme LLC to manufacture
sufficient quantities of product for clinical trials, the availability of
reimbursement for products, the competitive environment for any products
developed by Diacrin/Genzyme LLC, the continued funding of the joint venture by
Genzyme and Diacrin, and the risks and uncertainties described in Exhibit 99.2
to Genzyme Corporation's Annual Report on Form 10-K for the year ended December
31, 1999, as amended and Diacrin's Annual Report on Form 10-K for the year ended
December 31, 1999.
