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Newsroom 2005
- At the annual and special meeting of shareholders, Neurochem Inc. (NASDAQ: NRMX; TSX: NRM) highlighted the recent
advances of its investigational product candidates Fibrillex and Alzhemed and emphasized their further
development as the Company's main priority. Neurochem indicated that the plans to implement the management team
necessary to bring the Company to the next level of product development and commercialization are essentially complete.
During the quarter, the Company's financial position was significantly strengthened, with $89.7 million of cash
available at the end of the quarter.
"Over the past year, we have worked very hard not only to advance the development of our investigational product
candidates, but also to build a strong foundation for our Company with an exceptional management team and a solid
financial base," stated Francesco Bellini, Chairman, President and CEO. "I am very proud to be able to lead
a Company of this calibre and, as CEO and a significant shareholder, I will continue to support Neurochem and work
towards bringing our product candidates to market."
Fibrillex - Results to be Presented to FDA
Neurochem completed the Fibrillex Phase II/III clinical trial in December 2004. Fibrillex is being developed
for the treatment of Amyloid A (AA) Amyloidosis, a serious consequence of chronic inflammatory and infectious
diseases, such as Rheumatoid Arthritis. It is estimated that there are approximately 40,000 people in Europe and the
U.S. diagnosed with the disease.
The objective of the Phase II/III trial was to determine the ability of Fibrillex to delay or stop the progression
of AA Amyloidosis and to assess the safety of Fibrillex compared to a placebo. The trial was initiated in 2000
to demonstrate statistical significance through the composite primary endpoint reaching an absolute difference of 20%
in the number of patients stabilized or improved between the Fibrillex and placebo groups (p value= 0.01).
"The clinical trial for Fibrillex was the first Phase II/III clinical trial and the largest randomized,
placebo-controlled study ever undertaken to test for the efficacy of a drug to treat AA Amyloidosis," said
Denis Garceau, PhD, Neurochem's Senior Vice President, Drug Development. "We have broken new ground in designing
the study and defining endpoints that have never been assessed in a study of that magnitude for this disease."
An assessment of preliminary findings made public on April 18, 2005, indicated that Fibrillex is well tolerated.
The results also indicated that there were 13.4 percent more patients whose condition remained stable or improved
while on Fibrillex therapy, compared to placebo (p value=0.06). While the level of 13.4% is a positive sign, it
did not reach the pre-specified objective of the study of 20%. Preliminary findings on the secondary endpoints also
indicate that Fibrillex does have a consistent and clinically meaningful effect on kidney function. These
preliminary results suggest that Fibrillex delays progression of renal disease in AA amyloidosis patients.
Recognizing the lack of existing specific treatments for the disease, Fibrillex received "orphan drug"
status in both the United States and Europe. Fibrillex also has been accorded "Fast Track Product"
designation by the U.S. Food and Drug Administration (FDA) and was selected for the FDA's Pilot 2 program for
accelerated review. In December 2004 and in advance of completion of the trial, Neurochem (International) Limited, a
wholly owned subsidiary of the Company, concluded a collaboration and distribution agreement for Fibrillex with
Centocor, Inc., a wholly-owned subsidiary of Johnson & Johnson.
Data on the Fibrillex Phase II/III trial is expected to be presented at selected international medical
conferences in the coming months. Presentations at international conferences are scheduled during the European
Renal Association-European Dialysis and Transplant Association (ERA-EDTA) conference to be held in Istanbul, Turkey,
from June 4-7, 2005 and the European League Against Rheumatism (EULAR) conference to be held in Vienna, Austria,
from June 8-12, 2005. The plan is also to publish the data in a peer-reviewed journal. Meanwhile, Neurochem is
continuing to honour its commitment to supply Fibrillex to any patient receiving the drug, through the
on-going open-label extension study.
Neurochem, therefore, intends to continue with plans to seek regulatory approval of Fibrillex. As part of this
process, the Company confirmed a meeting date with the FDA to pursue discussions about its data and to investigate
the various regulatory options to possibly make Fibrillex available to the AA amyloidosis patients.
Alzhemed - North American Phase III Clinical Trial on Track
Progress throughout 2004 and into the first quarter of 2005 on the development of the investigational product
candidate, Alzhemed, for the treatment of Alzheimer's Disease (AD) is on-track and encouraging. This first-in-class
product candidate has a unique mechanism of action, which may be capable of stopping the disease in its early stages
by interfering with the formation of the toxic beta-amyloid complex and has been shown by many scientists to be
neurotoxic. Data from a Phase II clinical trial and interim results from a follow-on open-label extension study
over a 28-month period indicate that this orally administered investigational product candidate is safe and appears
to stop the progression of the disease, particularly in milder AD patients.
During 2004, Alzhemed was highlighted at the 8th International Montreal/Springfield Symposium on Advances in
Alzheimer Therapy and at the U.S. Alzheimer's Association at its annual international conference as a promising new
treatment in development for AD.
Neurochem initiated a large-scale Phase III clinical trial in North America in June 2004. This double-blind,
placebo-controlled study will include some 950 mild-to-moderate AD patients at 68 clinical centers across North
America, who will be receiving medication over the trial period of 18 months. Approximately 1,100 patients have
been screened and close to 800 are already on study medication. Recruitment in North America is therefore
proceeding on schedule and the study is on track for completion as planned.
The Company plans to initiate patient recruitment for a similar Phase III clinical trial in Europe this fall.
Neurochem announced in April 2005 that its Independent Safety Review Board (ISRB) has issued a recommendation to continue
the Company's North American Phase III clinical trial for Alzhemed. The recommendation by the members of the ISRB
was based on their recent review of the safety data from 562 patients who had been monitored in the trial for an
average of 12 weeks to that point.
Cerebril - A Phase IIb Clinical Trial Under Preparation
Cerebril, Neurochem's lead product candidate for the treatment of Hemorrhagic Stroke due to Cerebral Amyloid
Angiopathy (HS-CAA), has successfully completed a Phase IIa clinical trial to assess safety conducted at five
centers in the U.S.. There were no concerns regarding safety, and the pharmacokinetic profile of the product
candidate was well characterized.
A Phase IIb trial is currently being designed to test the efficacy and safety of Cerebril in preventing
recurring strokes caused by amyloid. Currently, no treatment exists for this disease.
Senior Management Team - Additions and Reassignment of Responsibilities
Neurochem also announced senior management appointments and reassignment of responsibilities: Francesco Bellini,
Chairman, President and CEO, committed to remain with the Company in an executive capacity for at least three
more years. Andreas Orfanos, M.B.B.Ch, MBA, was named Executive Vice President - Strategic Planning and
Scientific Affairs, Shona McDiarmid, PhD, LLB, was appointed Vice President - Intellectual Property, Daniel
Delorme, PhD, was named Vice President - Research, Mariano Rodriguez was appointed Vice President, Finance
and Chief Financial Officer, Christine Lennon was named Vice President, Business Development and Judith Paquin
was appointed Vice President, Human Resources. Philippe Calais, Pharm. D., became President - Global Business
and Denis Garceau, PhD, was appointed Senior Vice President - Drug Development.
Recent Developments
Neurochem announced in March 2005 that it had completed a public offering of 4 million common shares. The common
shares issued from treasury were sold in the United States and Canada and resulted in total gross proceeds to the
Company of approximately US$61.2 million. Neurochem Inc. and its subsidiaries intend to use the net proceeds from
the offering to fund the clinical trials of their investigational product candidates, primarily Alzhemed, to
advance Fibrillex, to further complete pre-clinical and research and development programs and for working
capital and general corporate purposes.
First Quarter Results - Consolidated Financial Results Highlights
The following information should be read in conjunction with the unaudited consolidated financial statements for the
three-month period ended March 31, 2005, as well as the audited consolidated financial statements for the year ended
December 31, 2004. For discussion regarding related-party transactions, contractual obligations and commercial
commitments, critical accounting policies, recent accounting pronouncements, and risks and uncertainties, refer to
the Management's Discussion and Analysis of Consolidated Financial Condition and Results of Operations for the year
ended December 31, 2004. All dollar figures are Canadian dollars, unless specified otherwise.
For the three month-period ended March 31, 2005, the net loss amounted to $16,970,000 ($0.54 per share), compared to
$9,164,000 ($0.31 per share) for the comparative period last year.
Revenues from collaboration agreement amounted to $1,205,000 for the current quarter and consist of the revenue
earned under the agreement with Centocor, Inc. (Centocor) in respect of Fibrillex. Revenue recognized during
the quarter is in respect of the non-refundable upfront payment received from Centocor, which is being amortized
over the remaining estimated period to obtaining the regulatory approvals of the investigational drug product. The
period to obtain the regulatory approvals is estimated and is subject to change based on additional information that
the Company may receive from time to time.
Reimbursable costs revenue amounted to $444,000 for the current quarter and consists of costs reimbursable by
Centocor in respect of Fibrillex-related activities. The Company earns no margin on these reimbursable costs.
Research and development expenses, before research tax credits and grants, amounted to $11,965,000 for the
current quarter, compared to $5,577,000 for the same period last year. The increase is mainly due to expenses
incurred in relation to the development of Alzhemed; more specifically for the Phase III North American clinical
trial and preparation for the Phase III European clinical trial. In the quarter ended March 31, 2005, research and
development expenses also included costs incurred to support the on-going Fibrillex open-label extension study, as
well as on-going drug discovery programs.
Research tax credits amounted to $409,000 this quarter, compared to $312,000 for the comparable quarter
last year. Research tax credits represent refundable tax credits earned under the Quebec Scientific Research and
Experimental Development Program. The increase is due to higher eligible expenses during the current period.
General and administrative expenses for the quarter totalled $5,165,000, compared to $3,965,000 for the same
quarter last year. The increase is attributable to the expansion of the corporate infrastructure to support growth
and the overall activity levels at the Company. More specifically, the increase is due to operating costs related
to the facilities acquired during the second quarter of 2004 and higher legal fees incurred in relation to the
Immtech litigation, and to other corporate agreements and matters.
Reimbursable costs amounted to $444,000 for the current quarter and consist of costs incurred on behalf of
Centocor in respect of Fibrillex related activities and reimbursable by Centocor.
Stock-based compensation amounted to $770,000 for the current quarter, compared to $424,000 for the comparable
quarter last year. This expense relates to employee stock options, whereby compensation cost is measured at fair value
at the date of grant and is expensed over the award's vesting period. The increase is due to new stock options
granted during the past year.
Depreciation and amortization for the current quarter increased to $570,000 from $368,000 for the comparable
quarter last year. The increase reflects the depreciation and amortization associated with the acquisition during
the past year of additional property and equipment, including the facilities acquired in the second quarter of
2004, and the increase in patent costs.
Interest and bank charges amounted to $121,000 for the current quarter, compared to $24,000 for the
comparable quarter last year. The increase is due to interest expense on the $10,500,000 revolving decreasing term
credit facility entered into by the Company to finance the acquisition of the facilities last year.
Interest income amounted to $251,000, compared to $322,000 for the comparable quarter last year. The
decrease results from lower average cash balances in the current period, compared to the same period last year.
Foreign exchange gains amounted to $226,000 for the current quarter, compared to $441,000 for the same quarter
last year. The decrease is attributable to lower monetary assets denominated in US dollars during the period, compared
to the comparable period in 2004. Gains were recorded as a result of an increase in the value of the US dollar versus
the Canadian dollar during the respective periods.
Variable interest entities
As previously reported, on January 1, 2005, the Company adopted the recommendations of the CICA Accounting Guideline
15 - Consolidation of Variable Interest Entities (AcG-15), which provide guidance for determining when an enterprise
consolidates the assets, liabilities and results of activities of entities that are subject to control on a basis other
than ownership of voting interests (a "variable interest entity" (VIE)). This guideline requires the Company
to identify VIEs in which it has an interest, to determine whether it is the primary beneficiary of such entities
and, if so, to consolidate the VIEs. A primary beneficiary is an enterprise that will absorb a majority of the
VIE's expected losses, receive a majority of its expected residual return, or both. It was determined that the
Company's investment in a holding company that owns Innodia's shares (Innodia Holding) meets the criteria for being
a VIE and that the Company is the primary beneficiary of Innodia Holding. The implementation of AcG-15 resulted in
the consolidation of the Company's interest in Innodia Holding starting January 1, 2005. The effect of the
implementation of this accounting guideline was to adjust the net carrying value of the long-term investment and the
opening deficit by $2,501,000 at January 1, 2005. The revised carrying amounts of the long-term investment and
non-controlling interest at January 1, 2005, were $3,359,000 and $1,439,000, respectively. The implementation of
this accounting guideline in the first quarter of 2005 resulted in the consolidation of a share of loss in a
company subject to significant influence of $755,000 and non-controlling interest of $225,000, in the Consolidated
Statement of Operations.
Litigation
Regarding the ongoing dispute with Immtech International, Inc. (Immtech) concerning a 2002 agreement, the Company
continues to vigorously defend against the claims brought by Immtech in its legal proceedings filed on August 12, 2003,
with the Federal District Court for the Southern District of New York, U.S.A. The dispute is now before an arbitral
tribunal convened in accordance with the rules of the International Court of Arbitration.
A hearing has been scheduled by the arbitral tribunal for mid-September 2005. Immtech has claimed monetary damages
which, to date, it has estimated at between a total of US$18 million and US$42 million, which includes an estimated
valuation for equitable relief. The Company has counterclaimed damages which, to date, it has estimated at no less
than US$3.5 million, which includes an estimated valuation for equitable relief. The outcome of this matter, or the
likelihood and the amount of loss, if any, is not determinable. No provision for possible loss has been recorded by
the Company in connection with this matter.
Liquidity and capital resources
As at March 31, 2005, the Company had cash, cash equivalents and marketable securities of $89,703,000, compared to
$29,173,000 at December 31, 2004. The increase is primarily due to proceeds received from the issue of additional
share capital during the quarter (as described below), net of funds used in operations and in investing activities.
On March 9, 2005, the Company completed a public offering of its common shares in the United States and in Canada.
The Company issued four million common shares at a price of US$15.30 per share. Total proceeds from the offering were
$74,495,000 (US$61,200,000) and the issue costs totaled $4,955,000. Certain funds raised from the share issuance were
denominated in U.S. dollars. The Company maintains a significant U.S. dollars position to serve as a natural hedge
of exchange rate fluctuations with respect to planned U.S. dollar denominated R&D expenditures primarily relating to
its phase III clinical programs. Net proceeds will be used to fund the clinical trials of the Company's product
candidates (primarily Alzhemed), other research and development programs, advancing Fibrillex, working
capital and general corporate purposes.
As at April 30, 2005, the Company had 34,569,581 common shares outstanding, 2,287,614 options granted under the employee
stock option plan and 4,000,000 warrants issued and outstanding.
Neurochem Inc.
Consolidated Financial Information1
($'000 CDN, except per share data)
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Three-month period
ended March 31
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Consolidated Statements
of Operations
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2005
(unaudited)
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2004
(unaudited)
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Revenues:
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Collaboration agreement
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1,205
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-
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Reimbursable costs
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444
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-
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1,649
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-
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Expenses:
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Research
and development
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11,965
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5,577
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Research tax credits
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(409)
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(312)
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Research grants
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(9)
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(119)
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General
and administrative
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5,165
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3,965
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Reimbursable costs
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444
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-
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Stock-based
compensation
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770
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424
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Depreciation
and amortization
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570
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368
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Interest and bank charges
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121
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24
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18,617
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9,927
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Net loss before
undernoted items:
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(16,968)
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(9,927)
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Interest income
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251
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322
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Foreign exchange gain
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226
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441
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Other income
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51
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-
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Share of loss in a
company subject to a
significant influence
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(755)
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-
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Non-controlling interest
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225
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-
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Net loss
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(16,970)
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(9,164)
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Net loss per share:
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Basic
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(0.54)
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(0.31)
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Diluted
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(0.54)
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(0.31)
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Weighted average number
of common shares
outstanding:
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Basic
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31,401,858
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29,863,793
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Diluted
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34,568,931
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34,817,965
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Consolidated
Balance Sheets
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At March 31
2005
(unaudited)
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At December 31
2004
(unaudited)
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Cash, cash equivalents
and marketable securities
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89,703
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29,173
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Other current assets
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6,363
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20,410
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Total current assets
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96,066
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49.583
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Restricted cash
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7,258
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-
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Long-term prepaid
expenses and deposits
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1,815
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1,361
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Long-term investment
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2,604
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4,421
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Capital assets
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20,711
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21,083
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Total assets
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128,454
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76,448
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Current liabilities
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15,297
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16,406
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Deferred revenue
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10,101
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9,772
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Long-term
accrued liabilities
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121
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126
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Long-term debt
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9,275
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9,450
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Non-controlling interest
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1,214
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-
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Shareholders' equity
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92,446
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40,694
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Total liabilities and
shareholders' equity
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128,454
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76,448
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1 Condensed from the Company's unaudited consolidated financial statements.
About Neurochem
Neurochem is focused on the development and commercialization of innovative therapeutics for neurological disorders. The
Company's pipeline of proprietary, disease-modifying oral products addresses critical unmet medical needs.
1,3-propanedisulfonate (1,3PDS; Fibrillex) is designated as an orphan drug and a Fast Track Product candidate
and is also part of an FDA Continuous Marketing Applications Pilot 2 program. The Phase II/III clinical trial of
Fibrillex for the treatment of AA Amyloidosis was recently concluded and preliminary results were disclosed
in April 2005. 3-amino-1-propanesulfonic acid (3APS; Alzhemed), for the treatment of Alzheimer's Disease, is
in a Phase III clinical trial and 3APS (Cerebril), for the prevention of Hemorrhagic Stroke caused by Cerebral
Amyloid Angiopathy, has completed a Phase IIa clinical trial.
To Contact Neurochem
For additional information on Neurochem and its drug development programs, please call the North American toll-free number
1 877 680-4500 or visit our Web Site at: www.neurochem.com.
Certain statements contained in this presentation, other than statements of fact that are independently verifiable
at the date hereof, may constitute forward-looking statements. Examples of such forward-looking statements include
those regarding Fibrillex, as well those regarding continuing and further development efforts. Such statements,
based as they are on the current analysis and expectations of management, inherently involve numerous risks and
uncertainties, known and unknown, many of which are beyond Neurochem's control. Such risks include but are not
limited to: the impact of general economic conditions, general conditions in the pharmaceutical industry, changes
in the regulatory environment in the jurisdictions in which Neurochem does business, stock market volatility,
fluctuations in costs, and changes to the competitive environment due to consolidation, as well as other risks
included in public filings of Neurochem. Consequently, actual future results may differ materially from the
anticipated results expressed in the forward-looking statements. In the case of forward-looking statements
regarding Fibrillex and continuing and further development efforts, specific risks which could cause actual results
to differ materially from Neurochem's current analysis and expectations include: failure to demonstrate the safety,
tolerability and efficacy of our product, the expense and uncertainty of obtaining regulatory approval, including
from the FDA, and the possibility of having to conduct additional clinical trials. Additionally, even if regulatory
approval is obtained, therapeutic products are generally subject to: stringent on-going governmental regulation,
challenges in gaining market acceptance, and competition. The reader should not place undue reliance on the
forward-looking statements included in this presentation. These statements speak only as of the date made and
Neurochem is under no obligation and disavows any intention to update or revise such statements as a result of
any event, circumstances or otherwise.
For further Information, please contact:
Dr. Lise Hébert
Vice President, Corporate Communications
lhebert@neurochem.com
275 Armand-Frappier
Laval (Quebec)
H7V 4A7
Tel: (450) 680-4500
Fax: (450) 680-4501
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