« June 2004 | Main | August 2004 »
July 31, 2004
"Father Of Gene Therapy" Arrested
This is sad and terrible news. I'll let the news article tell the story:
"Gene therapy pioneer Dr. William French Anderson was arrested Friday on charges of sexually molesting a girl over a four-year period, authorities said.
Anderson, 67 -- an internationally prominent University of Southern California scientist known as "the father of gene therapy" -- was arrested about 8:30 a.m. at his San Marino home one day after prosecutors filed a criminal complaint alleging six felony counts of assault, said Sandi Gibbons, spokeswoman for the Los Angeles County district attorney's office." ...
"The six felony counts against Anderson include one count of continuous sexual abuse against a person under 14 and five counts for lewd acts with a child, Gibbons said.
If convicted on all counts, Anderson could receive up to 56 years in state prison, Gibbons said.
Authorities said the girl, now 17, was about 10 when the alleged abuse started."
From the Guardian, some background on the Doctor:
In 1979, Anderson led a team of scientists with the National Institutes of Health in curing a genetic flaw in the cells of mice. By 1990, his laboratory had performed the first gene therapy experiment on a 4-year-old child with a rare genetic disease that prevents babies from developing their own immune systems.
Posted by Dave at 08:58 PM | Comments (0) | TrackBack
July 30, 2004
Francis Crick R.I.P.
"It is difficult to imagine a problem that would not be solved in 25 years.”
That is what Francis Crick once said. Why do I mention a "Francis Crick"? well...he is one of the two (some say three) people who discovered (recognized) the double-helix structure of DNA. This discovery eventually let to the discovery of the Huntington's gene and the Human Genome Project.
It would be hard to overestimate his importance in the eventual cure for Huntington's Disease. Go here to read a wonderfully written article by a truly gifted writer. Here are some excerpts to tempt you:
The evening in 1953 that Crick and his friend, American scientist James Watson, had the idea that DNA had a double helical structure, was probably not particularly different from any other. There couldn’t have been fire in Cambridge’s dry, grey April sky. Over a pint of beer at The Eagle, a popular Cambridge watering hole near Freeschool Lane, Crick had his moment — probably drowned out by the thronging din from drunk Cambridge academics...
Crick bewildered his contemporaries for disappearing beneath mountains of data, to emerge, usually weeks later, with distilled information that would, in the words of one Stanford scientist, “make rooms of intellectuals gasp”. He was bizarrely patient with his inquiries into the structure of DNA — the tempestuous arguments he had with Watson at the now legendary Eagle tavern in Cambridge are now the stuff of legend.
Crick once said, “It is difficult to imagine a problem that would not be solved in 25 years.” The human genome project has begun. And as that other great scientist Albert Einstein famously said, “Time has all the answers.”
Posted by Dave at 07:19 PM | Comments (0) | TrackBack
Criticism of Ron Reagan
I've debated whether to post this or not, but I believe there is good information in the article. I do believe that all sides should be presented.
Michael Fumento has an article in the conservative National Review on Ron Reagan's speech. With one quibble, he is accurate on the facts. The quibble? He says "embryonic stem cells have never been tested on a human", that isn't true. (Such as this reference to research on Huntington's Disease patients.) He also goes a little heavy handed in his criticism of Ron Reagan.
However, he makes several points that are worth repeating here:
Far from blocking federal embryonic-stem-cell research funding, Bush specifically authorized it so long as it used existing lines of embryonic cells. ... "Adult stem cells" can be extracted from various places in the human body as well as blood in umbilical cords and placentas. They were first used to treat human illness in 1957.
By the 1980s, adult stem cells were literally curing a variety of cancers and other diseases; embryonic stem cells have never been tested on a human. Adult stem cells now treat about 80 different diseases; again embryonic stem cells have treated no one. Adult stem cells obviously aren't rejected when taken from a patient's own body, though they may be from an unmatched donor; embryonic stem cells have surface proteins that often cause rejection. Implanted embryonic stem cells also have a nasty tendency to multiply uncontrollably, a process called "cancer." Oops.
The only potential advantage embryonic stem cells ever had was the belief that only they could be coaxed into becoming all the different cells of the body. We don't even know whether that's true. Conversely, three different labs have now discovered it may be true of certain adult stem cells.
Further, perhaps we have no need for "one size fits all." In recent years researchers have found that they can tease various adult stem cells into far more types of mature tissue than was previously thought possible. Moreover, they seem to find adult stem cells essentially wherever they look — including blood, bone marrow, skin, brains, spinal cords, dental pulp, muscles, blood vessels, corneas, retinas, livers, pancreases, fat, hair follicles, placentas, umbilical cords, and amniotic fluid. We may need all sizes, but we don't need them from one type of stem cell.
Posted by Dave at 07:03 PM | Comments (2) | TrackBack
July 29, 2004
A Novena For HD
I got this request from a reader, Anne-Marie. It's the kind of request that is hard to refuse. She requested that I post this request, and while I'm not Catholic, I appreciate the importance of the request.
It's for a wonderful cause...Huntington's Disease. Here's her request:
Below is a letter that I have sent out to my family and friends. If you are drawn to prayer, please consider joining us. My belief in prayer has grown deeper as I continue to live a full life with stage 4 breast cancer. My fourth of five cancers since I was 21 years old (43 years old this month). I started wearing the Miraculous Medal 3.5 years ago and believe many prayers are being answered on my behalf. Let’s storm heaven and get God’s help today.
Annie B
Dear Family and Friends,
I am writing to invite you to join me in praying a novena for the following prayer intentions: a cure/treatment for Huntington’s disease, for breast cancer and for melanoma along with the personal prayer intentions that rest on the heart of each person joining me in this Novena.
On December 8, 2004, the Catholic Church will celebrate the 150th Anniversary of the Dogma of the Immaculate Conception of Mary, the mother of God along with the approval of the Miraculous Medal as a sacramental medal.
Many miracles have been attributed to the Miraculous Medal. My hope is that by praying to Mary of the Immaculate Conception during this anniversary year, her promise of grace to those who pray and wear this medal will be fulfilled.
December 8th also marks the one-year anniversary of our Dad’s death from Huntington’s Disease. Dad has seen the face of God and I believe that he joins us in petitioning God for help. My plan is to pray the novena twice this year on the following dates, each time ending on a feast day of Our Lady:
August 4th –August 15th, The Feast of the Assumption
November 30th – December 8th, The Feast of the Immaculate Conception and the 150th Anniversary of its promulgation
Please consider joining me in praying this Novena either individually or as a family. I believe many graces await our petitions.
The following web sites provide the story of the Miraculous Medal, a place to request individual medals and the Novena. Peace be with you.
http://www.amm.org/medalp.htm Story of Miraculous Medal and free medal.
Love, Annie B
Posted by Dave at 07:35 PM | Comments (0) | TrackBack
Rasagiline
This was forwarded to me by a reader (thanks!)...
There is an article in the Israeli paper Haaretz that talks about a Rasagiline, a drug that is showing effectiveness in treating Parkinson's disease. A key phrase from the article:
"...hopes it will prove not only to slow Parkinson's, but to help nerve cells grow anew, something no other drug in the world can do. "
Is this drug a possible treatment for Huntington's? I don't know, but there aren't any published studies on it's use with HD. I did find several medical studies showing that Rasagiline is effective against cell death induced by apoptosis, which happens to be what the defective HD gene induces in cells.
It's definitely worth a look for researchers.
Posted by Dave at 07:25 PM | Comments (0) | TrackBack
Amarin Update
Amarin also released their quarterly financials and they lost $3.2 million the last three months as they didn't have ANY income. (Wait, another pharmaceutical that isn't raking in the dough?) Amarin, a company that has been under disasterous management until recently, has the rights to Miraxion (LAX-101) which in going into another round of phase III drug trials for treating Hunginton's Disease.
Key phrase from the press release:
Announcement of corporate strategy - Amarin will develop its late-stage development pipeline initially focusing on Huntington's disease and treatment unresponsive depression...
The future of their company depends on Miraxion. Here's the press release:
Amarin Corporation Reports Second Quarter 2004 Financial Results
Thursday July 29, 8:18 am ET
LONDON, July 29 - Amarin Corporation plc (NASDAQ: AMRN - News) today reported a net loss for the quarter, including discontinued activities, of $3.2 million or $0.18 per American Depositary Share (ADS), compared with a net loss of $7.0 million or $0.39 per ADS in the second quarter of 2003.
Second Quarter Highlights
Laxdale acquisition - signature of a definitive agreement to acquire Laxdale Limited ("Laxdale"), a privately owned, neuroscience development company based in Stirling, Scotland. This transaction consolidates Amarin's position in neuroscience and represents an initial step in the implementation of its strategy to emerge as a leader in the development and commercialization of novel drugs for the treatment of neurological disorders affecting the central nervous system. In addition, the acquisition broadens Amarin's development pipeline to include rights to Miraxion for all central nervous system disorders, including Huntington's disease and treatment unresponsive depression, in the U.S., E.U. and Japan together with existing licensee relationships for the major European markets and Japan.
Miraxion progress - recent discussions with the United States Food and Drug Administration ("U.S. FDA") and with the European Medicines Evaluation Agency ("EMEA") have provided valuable information which will be used in designing the protocol for the planned phase III trials with Miraxion.
Additional positive clinical data analysis - additional analysis of the clinical data from the initial pivotal study identified a sub-set of Huntington's disease patients (with a specific gene variant) that responded to Miraxion with statistical significance at 6 months and at 12 months. This sub-set of patients represents a significant majority of the Huntington's disease patient population.
Announcement of corporate strategy - Amarin will develop its late-stage development pipeline initially focusing on Huntington's disease and treatment unresponsive depression, for which a development partner will be sought. Amarin will seek to directly commercialize its neurology products in the U.S. and out-license or partner them in Europe and Japan. Amarin will also out-license or partner its pipeline globally for indications outside neurology. Amarin intends to leverage its development capabilities by supplementing its internal development pipeline through acquiring and/or in-licensing products for direct marketing by Amarin in its core U.S. market and selected field of neuroscience.
Financial results - operating loss from continuing activities for the second quarter of 2004 of $1.6 million, compared with an operating loss of $1.9 million in the second quarter of 2003.
Commenting on the results and progress made by Amarin in the second quarter, Rick Stewart, Amarin's Chief Executive Officer said, "The completion of the Laxdale acquisition will be a major milestone for Amarin and represents a refocusing of Amarin's resources to progress Miraxion through phase III clinical trials in Huntington's disease. We are encouraged by the additional data from the initial Huntington's disease pivotal study and look forward to commencement of the phase III studies. Amarin now has a substantial development pipeline and will be seeking development partners for certain compounds outside our selected therapeutic area of neuroscience or geographic reach."
The results for the second quarter and for the six months ended June 30, 2004 are analyzed between continuing and discontinued activities and are set out in further detail in the three pages of financial tables attached. The Laxdale acquisition is contingent upon Amarin shareholder approval, completion by Amarin of a $15 million financing and other customary conditions. Amarin's reported results for the second quarter and six months ended June 30, 2004 do not include the results of Laxdale. The results of Laxdale will be consolidated with Amarin, if and when the acquisition closes later this year.
Continuing Activities
The operating loss from continuing activities for the second quarter of 2004 was $1.6 million, compared with an operating loss of $1.9 million in the second quarter of 2003. For the six months ended June 30, 2004, the operating loss from continuing activities was $3.3 million, compared with an operating loss of $3.8 million for the same period in 2003. This operating loss represents head office operating expenses, business and corporate development costs and Miraxion product rights amortization.
Discontinued Activities
The operating loss on discontinued activities for the second quarter of 2004 of $1.4 million (compared with an operating loss of $4.9 million in the second quarter of 2003) represents the cost of conducting safety studies on Zelapar. Following the sale of the majority of Amarin's U.S. operations to Valeant Pharmaceuticals International ("Valeant") in the first quarter of 2004, Amarin remains responsible for undertaking safety studies on Zelapar and is liable for up to $2.5 million of development costs. It is expected that the remaining development cost obligation of $1.1 million will be incurred in the third quarter.
Under the terms of the sale to Valeant, Amarin will be paid a contingent milestone of $3 million if the safety studies referred to above are successfully completed. Of the $3 million, Amarin would receive a net amount of $1.6 million after accounting for contingent obligations of Amarin that only arise if this milestone is earned. In addition, Amarin will be paid a further contingent milestone of $5 million if Zelapar is approved by the U.S. FDA. As discussed below, Elan Corporation plc. ("Elan") has the option to seek early repayment of its outstanding $5 million loan if Amarin receives this $5 million approval milestone from Valeant. Neither of these milestones was earned in the second quarter and will be recognized as income if and when they are achieved.
As set out in Amarin's 2003 Annual Report filed with the Securities and Exchange Commission under Form 20-F, following the sale of the majority of Amarin's U.S. operations to Valeant, Amarin and Valeant are disputing the responsibility for incremental product inventory of approximately $6 million held at wholesalers. No provision has been made with respect to this matter. It is our view that the additional inventory should not impact the consideration payable to Amarin, whether as a result of a purchase price adjustment or otherwise. We cannot predict how this matter will be resolved. The Company intends to take all appropriate action to protect its interests in the event any claims should be asserted against it. This matter and the closing balance sheet continue to be discussed between the two parties.
The operating loss on discontinued activities for the second quarter of 2003 includes the results of the U.S. operations sold to Valeant in the first quarter of 2004 and the results of Amarin Development AB sold to Watson Pharmaceuticals, Inc. in the fourth quarter of 2003.
For the six months ended June 30, 2004, Amarin earned a profit before interest and tax of $21.4 million on discontinued activities (compared with a loss of $6.3 million for the same period in 2003) which includes an exceptional loss of $2.4 million on disposal of the majority of its U.S. operations and certain products and an exceptional gain of $25.6 million on the renegotiation of debt obligations to Elan during the first quarter. A deferred tax charge of $7.5 million arose on this exceptional gain.
The loss before interest and tax of $6.3 million on discontinued activities for the six months ended June 30, 2003 included charges totaling $7.3 million relating to Permax inventory and deductions and a credit of $7.5 million relating to a waiver of part of Amarin's debt obligations.
Balance Sheet
In conjunction with the acquisition of Laxdale, Amarin agreed a loan facility of up to StgGBP0.95 million to Laxdale. This loan facility is secured by a floating charge against Laxdale's assets. At June 30, 2004, Laxdale had drawn down StgGBP0.3 million and currently has drawn down a total of StgGBP0.5 million. At the time of the original licensing of Miraxion from Laxdale in 2000, Amarin recorded transaction consideration as an intangible fixed asset. The amortized historical cost at June 30, 2004 was $3.8 million. No provision has been made against carrying value of the loan to Laxdale or the Miraxion intangible asset. The carrying value of the loan to Laxdale and the Miraxion intangible asset are potentially dependent upon the completion by Amarin of the contemplated financing (described below) and the subsequent closing of the acquisition of Laxdale.
Liquidity
At June 30, 2004 Amarin had cash of $7.2 million. This is expected to be sufficient for Amarin to continue in operation until mid 2005 based on estimated head office operating expenses, expected business and corporate development costs, remaining Zelapar development obligations and working capital movements.
Pursuant to the Share Purchase Agreement for Laxdale, Amarin intends raising $15-20 million in an equity financing to fund the combined entity through the end of 2005, including the planned phase III trials for Miraxion in Huntington's disease.
At June 30, 2004, Amarin had a $5 million 5-year loan note owing to Elan with capital repayments of $1.5 million due in January 2006 and July 2007 and $2 million due in January 2009. At Elan's option, the loan note can be repaid from proceeds that Amarin receives from a $5 million milestone payable by Valeant on the NDA approval of Zelapar by the US FDA. The loan note is redeemable by Amarin at any time and carries an interest rate of 8%.
Rick Stewart continued, "Amarin's strategy is to incrementally add differentiated neuroscience compounds to our development pipeline with the ultimate objective of commercializing using an Amarin sales and marketing infrastructure in the U.S.. We will seek collaborators to maximize the return on the products outside the U.S. and also where the U.S. opportunity extends into therapeutic markets requiring a substantial sales force."
About Amarin Corporation
Amarin Corporation is a neuroscience company focused on the development and commercialisation of novel drugs for the treatment of neurological disorders affecting the central nervous system.
For press releases and other corporate information, visit our website at http://www.amarincorp.com.
Statements in this press release that are not historical facts are forward-looking statements that involve risks and uncertainties which may cause the Company's actual results in future periods to be materially different from any performance suggested herein. Such risks and uncertainties include, without limitation, the uncertainty of entering into and consummating a definitive agreement on terms acceptable to the parties, the inherent uncertainty of pharmaceutical research, product development and commercialisation, the impact of competitive products and patents, as well as other risks and uncertainties detailed from time to time in periodic reports. For more information, please refer to Amarin Corporation's Annual Report for 2003 on Form 20-F and its Form 6-Ks as filed with the U.S. Securities and Exchange Commission. The company assumes no obligation to update information on its expectations.
Amarin Corporation plc
Period Ended 30 June 2004 Selected Data (UK GAAP - UNAUDITED)
--------------------------------------------------
Three months ended 30 June
--------------------------------------------------
2004 2003
---------------------------------------- ---------
Pre Exceptional Exceptional Total Total
$'000 $'000 $'000 $'000
Revenue:
Revenue from
continuing activities - - - -
Revenues from
discontinued activities (29) - (29) 2,595
---------------------------------------- ---------
Total revenues (29) - (29) 2,595
---------------------------------------- ---------
---------------------------------------- ---------
Cost of sales:
Direct costs - - - 1,070
---------------------------------------- ---------
Cost of sales from
discontinued activities - - - 1,070
---------------------------------------- ---------
---------------------------------------- ---------
Gross (loss)/profit:
Continuing activities - - - -
Discontinued activities (29) - (29) 1,525
---------------------------------------- ---------
Total gross (loss)/profit (29) - (29) 1,525
---------------------------------------- ---------
---------------------------------------- ---------
Operating expenses/(income):
Selling, General &
Administrative 1,496 - 1,496 1,792
Amortisation of
intangible assets 144 - 144 125
---------------------------------------- ---------
Operating expenses
from continuing
activities 1,640 - 1,640 1,917
Selling, General &
Administrative - - - 3,537
Amortisation of
intangible assets - - - 1,223
Loss/(gain) on
renegotiation of
Elan debt - 14 14 -
---------------------------------------- ---------
Selling, General &
Administrative from
discontinued
activities - 14 14 4,760
Research &
development from
discontinued
activities 1,383 - 1,383 1,632
---------------------------------------- ---------
Operating
expenses/(income)
from discontinuing
activities 1,383 14 1,397 6,392
Total selling,
general &
administrative 1,640 14 1,654 6,677
Total research &
development 1,383 - 1,383 1,632
---------------------------------------- ---------
Total operating
expenses/(income) 3,023 14 3,037 8,309
---------------------------------------- ---------
---------------------------------------- ---------
Operating (loss)
from continuing
activities (1,640) - (1,640) (1,917)
Operating
(loss)/profit on
discontinued
activities (1,412) (14) (1,426) (4,867)
Total operating
(loss)/profit (3,052) (14) (3,066) (6,784)
Exceptional
income/(expense) -
discountinued
activities
Escrow proceeds of
Q4 2003 Swedish
disposal - -
Loss on disposal of
US operations and
certain products (62) -
(Loss)/profit on
ordinary activities
before interest
Continuing
activities (1,640) (1,917)
Discontinued
activities (1,488) (4,867)
-------- --------
(3,128) (6,784)
Net interest payable (68) (192)
-------- --------
(Loss)/income before taxes (3,196) (6,976)
Income tax (expense) - (41)
Dividends payable - -
-------- --------
Net (loss)/income
for the period (3,196) (7,017)
-------- --------
-------- --------
Weighted average
shares - basic 17,940 17,932
Weighted average
shares - diluted 17,940 18,791
(Loss)/income per share:
Basic (0.18) (0.39)
Diluted (0.18) (0.39)
--------------------------------------------------
Six months ended 30 June
--------------------------------------------------
2004 2003
---------------------------------------- ---------
Pre Exceptional Exceptional Total Total
$'000 $'000 $'000 $'000
Revenue:
Revenue from
continuing activities - - - -
Revenues from
discontinued activities 1,017 - 1,017 5,862
---------------------------------------- ---------
Total revenues 1,017 - 1,017 5,862
---------------------------------------- ---------
---------------------------------------- ---------
Cost of sales:
Direct costs 107 - 107 6,470
---------------------------------------- ---------
Cost of sales from
discontinued activities 107 - 107 6,470
---------------------------------------- ---------
---------------------------------------- ---------
Gross (loss)/profit:
Continuing activities - - - -
Discontinued activities 910 - 910 (608)
---------------------------------------- ---------
Total gross (loss)/profit 910 - 910 (608)
---------------------------------------- ---------
---------------------------------------- ---------
- - -
Operating expenses/(income): - - -
Selling, General &
Administrative 2,962 - 2,962 3,533
Amortisation of
intangible assets 288 - 288 250
---------------------------------------- ---------
Operating expenses
from continuing
activities 3,250 - 3,250 3,783
-
Selling, General &
Administrative 1,575 - 1,575 7,662
Amortisation of
intangible assets - - - 2,446
Loss/(gain) on
renegotiation of
Elan debt - (25,572) (25,572) (7,500)
---------------------------------------- ---------
Selling, General &
Administrative from
discontinued
activities 1,575 (25,572) (23,997) 2,608
Research &
development from
discontinued
activities 1,383 - 1,383 3,120
---------------------------------------- ---------
Operating
expenses/(income)
from discontinuing
activities 2,958 (25,572) (22,614) 5,728
Total selling,
general &
administrative 4,825 (25,572) (20,747) 6,391
Total research &
development 1,383 - 1,383 3,120
---------------------------------------- ---------
Total operating
expenses/(income) 6,208 (25,572) (19,364) 9,511
---------------------------------------- ---------
---------------------------------------- ---------
Operating (loss)
from continuing
activities (3,250) - (3,250) (3,783)
Operating
(loss)/profit on
discontinued
activities (2,048) 25,572 23,524 (6,336)
Total operating
(loss)/profit (5,298) 25,572 20,274 (10,119)
Exceptional
income/(expense) -
discountinued
activities
Escrow proceeds of
Q4 2003 Swedish
disposal 350 -
Loss on disposal of
US operations and
certain products (2,438) -
(Loss)/profit on
ordinary activities
before interest
Continuing
activities (3,250) (3,783)
Discontinued
activities 21,436 (6,336)
-------- --------
18,186 (10,119)
Net interest payable (97) (447)
-------- --------
(Loss)/income before taxes 18,089 (10,566)
- -
Income tax (expense) (7,500) (143)
Dividends payable - (24)
-------- --------
Net (loss)/income
for the period 10,589 (10,733)
-------- --------
-------- --------
-
Weighted average
shares - basic 17,940 16,250
Weighted average
shares - diluted 17,940 17,109
-
(Loss)/income per share: -
Basic 0.59 (0.66)
Diluted 0.59 (0.66)
As at 30 June
------------------------
------------------------
2004 2003
-------- --------
$'000 $'000
1. Select Balance Sheet Data
Net current assets/(liabilities) 5,230 (9,714)
- see note 4
Cash 7,211 11,232
Total assets - see note 5 12,140 68,311
Long term creditors and provisions
- see note 6 (5,000) (34,356)
Called up share capital
(ordinary shares) and capital
redemption reserve 29,088 29,076
Total shareholders' funds 4,246 2,982
-------- --------
Three months ended 30 June
----------------------------
----------------------------
2004 2003
-------- --------
2. EBITDA $'000 $'000
(Loss)/profit for period (3,196) (7,017)
amortisation 144 1,348
interest 68 192
taxation - 41
EBITDA (2,984) (5,436)
3. The selected financial data set out above should be read in
conjunction with Amarin's 2003 Annual Report filed with the Securities
and Exchange Commission under Form 20-F.
4. In conjunction with the acquisition of Laxdale, Amarin agreed a loan
facility of up to StgGBP0.95 million to Laxdale. This loan facility is
secured by a floating charge against Laxdale's assets. At June 30,
2004, Laxdale had drawn down StgGBP0.3million and currently has drawn
down a total of StgGBP0.5 million. The recoverability of this loan is
potentially dependent upon the completion by Amarin of a financing of
$15 million or more and the subsequent closing of the acquisition of
Laxdale.
5. At the time of the original licensing of Miraxion from Laxdale in
2000, Amarin recorded transaction consideration as an intangible fixed
asset. The amortized historical cost at June 30, 2004 was $3.8
million. The carrying value of the Miraxion intangible asset is
potentially dependent upon the completion by Amarin of a financing of
$15 million or more and the subsequent closing of the acquisition of
Laxdale.
6. At June 30, 2004, Amarin had a $5 million 5-year loan note owing to
Elan with capital repayments of $1.5 million due in January 2006 and
July 2007 and $2 million due in January 2009. At Elan's option, the
loan note can be repaid from proceeds that Amarin receives from a $5
million milestone payable by Valeant on the NDA approval of Zelapar by
the US FDA.
7. Shareholders' Equity 30-Jun-04
---------
$'m
UK GAAP 4.2
Lax-101 product rights (3.8)
Income recognition (0.6)
Preference dividends 0.5
---------
US GAAP 0.3
---------
---------
8. Basis of preparation - Going Concern
These selected financial data have been prepared on a going concern
basis, consistent with the basis of preparation of the Group's current
annual financial statements, filed with the SEC under form 20-F.
At June 30, 2004 Amarin had cash of $7.2 million. This is expected to
be sufficient for Amarin to continue in operation until mid 2005 based
on estimated head office operating expenses, expected business and
corporate development costs, remaining Zelapar development obligations
and working capital movements.
Pursuant to the Share Purchase Agreement for Laxdale, Amarin intends
raising $15-20 million in an equity financing to fund the combined
entity through the end of 2005, including the planned phase III trials
for Miraxion in Huntington's disease.
These selected financial data do not reflect the potential adjustments
that would be necessary should the Group cease to be a going concern.
Selected Income Statement Data - extract of continuing activities
----------------------- --------------------
Three months Six months
ended 30 June ended 30 June
----------------------- --------------------
2004 2003 2004 2003
------- ------- ------- -------
Total Total Total Total
$'000 $'000 $'000 $'000
Revenue:
Revenue from
continuing
activities - - - -
------- ------- ------- -------
- - - -
Operating
expenses/(income):
Selling, General &
Administrative 1,496 1,792 2,962 3,533
Amortisation of
intangible assets 144 125 288 250
------- ------- ------- -------
Operating expenses
from continuing
activities 1,640 1,917 3,250 3,783
Operating (loss)
from continuing
activities (1,640) (1,917) (3,250) (3,783)
------- ------- ------- -------
Selected Income Statement Data - extract of discontinued activities
----------------------- --------------------
Three months Six months
ended 30 June ended 30 June
----------------------- --------------------
2004 2003 2004 2003
------- ------- ------- -------
Total Total Total Total
$'000 $'000 $'000 $'000
Revenue:
Revenues from
discontinued
activities (29) 2,595 1,017 5,862
------- ------- ------- -------
Cost of sales:
Cost of sales from
discontinued
activities - 1,070 107 6,470
------- ------- ------- -------
------- ------- ------- -------
Gross (loss)/profit
Discontinued
activities (29) 1,525 910 (608)
------- ------- ------- -------
Total gross
(loss)/profit (29) 1,525 910 (608)
------- ------- ------- -------
------- ------- ------- -------
Operating
expenses/(income):
Selling, General &
Administrative - 3,537 1,575 7,662
Amortisation of
intangible assets - 1,223 - 2,446
------- ------- ------- -------
Selling,General &
Administrative
pre-exceptional item - 4,760 1,575 10,108
Exceptional
loss/(gain) on
renegotiation of
Elan debt 14 - (25,572) (7,500)
------- ------- ------- -------
Total selling,
general &
administrative from
discontinued
activities 14 4,760 (23,997) 2,608
Research &
development from
discontinued
activities 1,383 1,632 1,383 3,120
------- ------- ------- -------
Total operating
expenses/(income)
from discontinued
activities 1,397 6,392 (22,614) 5,728
------- ------- ------- -------
------- ------- ------- -------
Total operating
(loss)/profit on
discontinued
activities (1,426) (4,867) 23,524 (6,336)
Exceptional
income/(expense) -
discountinued
activities
Escrow proceeds of
Q4 2003 Swedish
disposal - - 350 -
Loss on disposal of
US operations and
certain products (62) - (2,438) -
------- ------- ------- -------
(Loss)/profit on
ordinary activities
before interest
Discontinued
activities (1,488) (4,867) 21,436 (6,336)
------- ------- ------- -------
Posted by Dave at 07:04 PM | Comments (0) | TrackBack
Sirna Update
Sirna Therapeutics, the company with an inside track to someday marketing an extremely promising "cure" for Huntington's Disease has announced their last quarter's results.
For the last quarter, Sirna lost $6.3 million in the last quarter. Their expenses were $6.8 million. (I thought all pharmaceutical companies were making a fortune!) Good thing they have $41 million in the bank, eh? Key excerpt from the article (available below):
Howard Robin, President and Chief Executive Officer of the Company commented, ... "We also announced that we are evaluating a new program in central nervous system diseases, specifically Huntington's disease, which includes a collaboration with Dr. Beverly Davidson at the University of Iowa."
Sirna Therapeutics Reports Second Quarter Financial Results
Thursday July 29, 7:32 am ET
BOULDER, Colo., July 29 - Sirna Therapeutics, Inc. (Nasdaq: RNAI - News) today reported financial results for the quarter ended June 30, 2004.
For the quarter ended June 30, 2004, the Company reported a net loss applicable to common stock of $6.3 million, or $0.18 per share, compared to a net loss applicable to common stock of $7.8 million, or $0.28 per share, for the same period in 2003.
Contract revenues for the second quarter of 2004 were $454,000 compared with $3.0 million for the same quarter in 2003. The 2004 second quarter revenues include contract revenues from Eli Lilly & Company (Lilly), as well as contract manufacturing revenues from Archemix Corp., a privately held company. Contract revenues for the second quarter of 2003 included $2.7 million for the completion of Sirna's manufacturing contract with Geron.
Total expenses for the second quarter of 2004 were $6.8 million compared to $10.8 million for the second quarter of 2003. Second quarter 2003 expenses included $5.3 million for a patent cost write-off. Overall, the Company's research and development expenses were $5.6 million for the second quarter of 2004 compared to $4.3 million incurred during the second quarter 2003, an increase of $1.3 million. The increase is primarily attributable to the scale- up of research and development activities, as well as an increase in expenses related to the advancement towards the clinic of Sirna-027, the Company's RNAi drug candidate targeting age-related macular degeneration (AMD).
The Company's cash, cash equivalents and securities available for sale were $41.6 million at June 30, 2004.
Howard Robin, President and Chief Executive Officer of the Company commented, "Sirna made important progress during the second quarter, including further preparations to file our first siRNA Investigational New Drug application with the FDA later this year for AMD. We also announced that we are evaluating a new program in central nervous system diseases, specifically Huntington's disease, which includes a collaboration with Dr. Beverly Davidson at the University of Iowa."
About Sirna Therapeutics
Sirna Therapeutics is using its proprietary technology and expertise in nucleic acids to develop a new class of nucleic acid-based therapeutics involving RNA interference (RNAi). RNAi is a mechanism used by cells to regulate the expression of genes and replication of viruses. The RNAi mechanism uses short interfering RNA (siRNA) to induce the destruction of target RNA using naturally occurring cellular protein machinery. Harnessing the natural phenomenon of RNAi holds potential for the development of a new class of drugs with specificity towards a wide range of diseases that result from undesirable protein production or viral replication. More information on Sirna Therapeutics is available on the Company's web site at http://www.sirna.com.
Statements in this press release which are not strictly historical are "forward-looking" statements which should be considered as subject to many risks and uncertainties, including early stage of development and short operating history, ability to achieve and maintain profitability, ability to obtain and protect patents, risk of third-party patent infringement claims, ability to engage collaborators, ability to obtain regulatory approval for products, concentration of stock ownership, and availability of materials for product manufacturing. These and additional risk factors are identified in the Sirna's Securities and Exchange Commission filings, including the Forms 10-K and 10-Q and in other SEC filings. Sirna undertakes no obligation to revise or update any forward-looking statements in order to reflect events or circumstances that may arise after the date of this release.
SIRNA THERAPEUTICS, INC.
CONDENSED BALANCE SHEET
(in thousands)
June 30, December 31,
2004 2003
(unaudited)
Assets
Cash, cash equivalents and
securities available-for-sale $ 41,592 $ 36,624
Accounts receivable 217 156
Property, plant and equipment, net 3,018 3,402
Other assets, net 2,364 2,502
Total assets $ 47,191 $ 42,684
Liabilities and stockholders' equity
Current liabilities $ 6,240 $ 4,042
Long-term liabilities 389 3,868
Stockholders' equity 40,562 34,774
Total liabilities & stockholders' equity $ 47,191 $ 42,684
SIRNA THERAPEUTICS, INC.
CONDENSED STATEMENTS OF OPERATIONS
(Unaudited)
(in thousands, except share amounts)
Three months ended Six months ended
June 30, June 30,
2004 2003 2004 2003
Revenue
Contract revenues $ 450 $2,877 $768 $3,262
Contract revenues-joint venture -- -- -- 2
Contract revenues-related
parties 4 105 4 208
Total revenues 454 2,982 772 3,472
Expenses
Research and development 5,629 4,276 10,735 8,053
General and administrative 1,147 1,225 2,292 2,615
Write-off of patent costs -- 5,344 -- 5,344
Total expenses 6,776 10,845 13,027 16,012
Operating loss (6,322) (7,863) (12,255) (12,540)
Other income (expense)
Interest income,
expense and other expense 41 105 36 --
Equity in loss of
unconsolidated affiliate -- (77) -- (209)
Total other income (expense) 41 28 36 (209)
Net loss (6,281) (7,835) (12,219) (12,749)
Accretion of dividends on
preferred stock -- -- -- 562
Net loss applicable to
common stock $(6,281) $(7,835) $(12,219) $(13,311)
Net loss per share
(basic and diluted) $(0.18) $(0.28) $(0.36) $(0.84)
Shares used in computing
net loss per share 35,523,434 28,273,448 33,647,086 15,895,980
Posted by Dave at 06:56 PM | Comments (0) | TrackBack
A Star For HD
The late John Ritter's son, Jason Ritter, is working on behalf of Huntington's Disease. No, it isn't in the Ritter family. His girlfriend and a friend both have it in their families.
It's wonderful to see celebrity support for Huntington's Disease. So many celebrities choose the "in" charity, but not one that needs a major celebrity (such as HD). I'm now a big fan. Here's the relevant excerpt from the article:
Ritter lives with his girlfriend, stage actress Marianna Palka, whom he met in New York City. And after becoming friends with 8 Simple Rules guest star Billy Brown (who played Kyle), Ritter started speaking on behalf of research to help cure Huntington's disease - which has affected Brown's and Palka's families. It is a genetic disease that causes degeneration of brain cells
"We want to get awareness out there and get stem-cell research," Ritter said.
We may want to let him know there is stem cell research and that there is other research that is VERY promising.
Posted by Dave at 06:46 PM | Comments (0) | TrackBack
July 28, 2004
Democratic Convention - Part 2
Well, Senator Diana Degette spoke earlier about stem cell research. Unfortunately, the transcript is not yet available.
Ron Reagan's speech was better than I expected. He spoke specifically about "embryonic" stem cell research. Of course there was no mention of adult stem cell research or that embryonic stem cell research is legal, just not federally funded. So it was a bit misleading in what it left out.
Not much else to report from yesterday in regards to health care and medical research. There is starting to be some discussion amoung the "chattering classes" on when Kerry will divulge the details of his plan to reduce the cost of health care. At this point I doubt we'll see much more detail before election day.
Tonight the vice-presidential candidate, John Edwards, will be speaking. It should be interesting to see if he makes reference to lawyers and medical malpractice.
Posted by Dave at 04:48 PM | Comments (0) | TrackBack
July 27, 2004
(Sigh) The "F" word and the "E" Word
I'm afraid to see how Ron Reagan's speech is covered by journalists in tomorrow's paper. Based on the following it isn't going to be pretty and it is certainly hard to have an intelligent debate on such an important subject when the facts aren't even reported correctly.
The blog 'Fight Aging', has a roundup of Stem Cell Politics (via InstaPundit)...
In it, you'll see some of the misleading arguments that are being made on stem cell research. Some examples:
First item, taken from USA Today frames the issue properly: "The Bush administration has limited federal funding of research on stem cells that results in the destruction of a living human embryo."
The second item is taken from the Courier-Post Online that, embarrassingly, goes the whole article without mentioning "federal funding" or "embryonic" and gives the impression that opposition is to all stem cell research. Truly bad journalism.
The next piece from Chris Mooney does mention "embryonic" and also makes the accurate point that that embryonic stem cells are unlikely to cure Alzheimers. There is room for debate on this, but I don't agree that embryonic stem cells could "yield a wealth of information about Alzheimers". But I also wouldn't rule out that stem cells, of either type, could help repair the brain from the damage done by Alzheimer's after a treatment is found.
The following piece concerning NIH and adult stem cells, also by Chris Mooney, is also accurate on the details of the debate.
A piece by Chris Mooney, again, quotes a LA Times story saying "federal restrictions on stem-cell research". (It really doesn't take that much ink to say "federal funding" and "embryonic research"!)
In the next one references a New York Times article that seems to believe that the only stem cells that exist are those that come from embryos. Here's the quote from the article "To cultivate the cell lines necessary for research, human embryos have to be destroyed." The article also doesn't mention that the federal limitation is on (lets say it together) "federal funding of embryonic stem cell research"
The final item mentions a study by the "non-profit and non-partisan" Results For America that shows support for stem cell research. I didn't look to see how the survey questions were worded but the article the results came from did manage to say "federal funding for embryonic stem cell research". Though they also managed to preface that with "major new restriction" which isn't accurate because there was no federal funding when the decision was announced by the Bush administration. What was new was what WAS allowed that wasn't allowed before then.
I really do hope that journalists start to get this right. Whether it is by ignorance or by choice, the question being debated (federal funding of embryonic stem cell research) should not be framed so inaccurately.
Posted by Dave at 07:38 PM | Comments (0) | TrackBack
Democratic Convention - Part 1
It's been a little more than a day into the Democratic Convention and this is what has been said on health care so far:
Senate candidate Nancy Farmer basically said American's need more access to health care.
Senate candidate Barack Obama talked about people stuggling with health care bills.
Hillary Clinton said that Kerry would "Solve a health care crisis, not ignore it"
So, not much to say about health care and medical research other than "it's too expensive." As for tonight...
Arizona Governor Janet Napolitano is to talk about health care issues.
Part of Teresa Heinz Kerry's speech will be about health care. Howard Dean, the Democratic candidate who had the strongest health care policy in the primaries, will speak.
The top speaker tonight will be Ron Reagan who will speak on funding embryonic stem cell research.
Posted by Dave at 06:35 PM | Comments (0) | TrackBack
Lithium
The Journal 'Nature' has an interesting article on Lithium and Alzhiemers. The study they report on indicates that Lithium slows the progression of Alzheimers.
One of the ways it does this, is of interest to those in the HD community: It reduced the amyloids ('tangles') in the brain. Amyloids are key feature of Huntington's Disease. It's possible this 'simple salt' could delay the onset of HD.
Researchers now think that the simple salt could slow the progress of degenerative brain disorders, such as Huntington's and Alzheimer's disease.
Paula Nunes and colleagues from the University of São Paulo, Brazil, studied 74 elderly people with bipolar disorder. Four percent of those taking lithium had Alzheimer's disease, compared with 21% of patients who were not taking the drug.
The researchers conclude that lithium therapy may lower the risk of developing Alzheimer's disease. ...
The team think that the drug works by blocking the action of an enzyme called GSK-3. In a brain with Alzheimer's, GSK-3 seems to prompt the addition of phosphate molecules to the tau protein, causing tangles to form. Lithium may prevent this from happening.
Posted by Dave at 06:06 PM | Comments (0) | TrackBack
July 26, 2004
pHD In The Olympics!
Yep! British oarswoman Sarah Winckless will be in Athens next month and she has a real chance for a medal.
Winckless will again compete in the double in Athens, this time with Elise Laverick. On the sidelines will sit her mother -- a reminder of what the future holds.
Winckless's mother was diagnosed with Huntington's disease in 1996 and her daughter took the test shortly after.
"I was very calm when I found out," Winckless said. "It was something mum had had diagnosed about six months earlier and I was fully prepared that the test could go either way.
"To be honest I've just got on with my life."...
Winckless says the fact she carries the gene does not affect her preparation for the Games but, with a Cambridge University degree and a marketing career, she has several other options to consider when she returns from Athens in September.
"I don't think anyone knows what the future holds," she said.
"I think the very nature of sport is you take it a year at a time and I don't think the fact that I've got the gene makes my goal-setting or my attitude any different from anyone else in the squad."
She sounds like an incredible woman. I'll be one person on this side of the 'pond' cheering for her!
Posted by Dave at 10:26 PM | Comments (0) | TrackBack
July 25, 2004
Huntington's Surprising Effect On Other Cells
Do yourself a favor and read this on the HD Lighthouse website. Dr. Marsha Miller has some excellent comments on this new, and important, study.
The crux of the study...HD affects other cells of the body also, not just the striatum. However, the other's don't die as a result of the defective gene. This gives researchers valuable clues about the disease and hints for potential treatments.
Dr. LaVonne Goodman, with HDDW, was kind enough to send along these comments on this important study:
I believe the Isacson article to be a pivotal one, and so might deserve a second comment from this “contributing editor”.
Huntington’s disease is caused by one gene. But this gene product, the abnormal Huntingtin protein affects many cellular systems. More than twenty such systems are described. And now we learn from Isacson that these adverse effects also occur in brain cells outside the striatum; And in skin cells. But while these cells have similar Huntingtin effect, they don’t get severely damaged and die.
Isacson studied and described three major cellular systems that are affected in all these cells: (1) mitochondria energy problems, (2) decreased neurotrophic factors, and (3) ineffective proteosome systems (which clear defective proteins). All these other surviving cells express the same problems: But they don’t get severely damaged and die.
It is becoming clear that the damage to striatum comes from many different cellular directions. No longer should there be competing arguments about which system is most important. They probably all are.
She goes on to discuss the importance of testing combinations of 'safe' drugs, which is something that HDDW is focused on. (Thankfully.)
Posted by Dave at 10:32 PM | Comments (0) | TrackBack
July 24, 2004
Stem Cell Research - The Real Story
There's going to be some good news for the HD community next week. One of the topics coming out of the Democratic convention will be medical research.
We certainly need more discussion on this. The bad news is that the discussion is going to be limited to stem cell research, specifically embryonic stem cell research. The speaker on this will be Ron Reagan, the son of the former president.
Ordinarily, I would consider this all good news but the game here is all political, not medical, and the facts are going to be discarded. That's not new really, since getting the facts on stem cell research has been a challenge. Both sides are guilty of playing fast and loose with them.
I've been studying this topic for the last few months and this is what I've found out...
Too many news articles and advocates do not use the word "embryonic" when discussion the politics of stem cells. There are two types of stem cells - 'adult' and 'embryonic'. Adult stem cells can be found in the blood, bone marrow, fat, organs, placenta, umbilical cord, etc. Embryonic stem cells come from human embryos. The source of these cells are reason for the heated debate.
What makes stem cells different from other cells is their ability to change into other forms of cells (called transdifferentiation). Researchers are trying to use stem cells to create other types of cells the body needs. This type of research is of special interest to those who have diseases that involve the loss of cells, Huntington's and Alzheimers are just two of many.
So, you might be asking, why not just use the adult cells and not worry about using embryonic stem cells? Well, as it turns out, they do not behave the same. And there lies the rub. Embryonic stem cells are unique and are uniquely fascinating to many researchers. They have more questions than answers and some believe that answering these questions will greatly help medicine.
However, this is where many advocates tend to 'stretch' the truth to make the case for their side. First, adult stem cells DO transdifferentiate and researchers are learning more and getting better at it all the time. Those who say that only embryonic stem cells are capable are either ill-informed or lying. On the other hand, those who say that there is nothing worthwhile in researching embryonic stem cells (ethical issues aside) are also wrong.
Other 'inaccuracies'...stem cell research is not banned or even limited (except for certain cloning activies). There isn't even any limitation on embryonic stem cell research. Yes, you heard me, researchers are FREE to do embryonic stem cell research. What you are hearing about is FEDERAL FUNDING of embryonic stem cell research. Even that isn't banned, its just limited to the stem cell lines that were in existence at the time of the decision (numbers vary on how many there are, but 73 is a common number used).
But...it is told there are only (12, 17, 19,21...the reported number varies) stem cells truly available and usable. This is true, what you won't hear is that it takes time to make a line available and the number available is steadily increasing.
But...it is told these lines aren't usable for treatments on humans. Actually, this is true, or mostly so. There are issues with many of the lines. That doesn't mean they aren't useful for research, it's just that many are useable on humans. What isn't said in all this is that they are a VERY LONG WAY from use in humans and one of the biggest reasons has to do with the unique nature of the embryonic stem cells...they are relatively unstable and controllable (at this point). Treatments with these cells have a high occurance of tumors. (There is also research in using these cells to treat tumors).
But...adult stem cells are not useful for treatment. This is not true, all successful stem cell treatments today involve adult stem cells. Bone marrow transplants are really just stem cell transplants as it is the adult stem cells that do the work. This, however, does not mean the embryonic don't have any potential for future treatments.
Heard...nobody is investing in embryonic stem cell research because there is no future in it. That isn't true, well...at least not exactly true. Private research companies, as a rule, are NOT investing in embryonic stem cell research. However, there is funding coming from many foundations including the Juvenile Diabetes Research Foundation, Stem Cell Research Foundation, Michael J. Fox Foundation, etc.
In fact, there is far more funding for embryonic stem cell research than there is for Huntington's Disease research. There is also a lot of money going into this research in other countries, not to mention the federal funding of approved cell lines in the US.
So what is the big deal then?...Well, NIH funding accounts for a huge percentage of all medical research. Full federal funding opens up a huge new source of cash. It also makes more researchers available (who are now involved in federally funded projects).
So what is the potential of stem cells in treatments, especially Huntington's Disease? There appears to be potential, for both adult and stem cells for use in many medical treaments. Parkinson's & paralysis are two such candidates for this type of treatment. It does appear that treatments involving adult stem cells will arrive sooner (for a variety of reasons).
As for their use in Huntington's Disesase? I'm more of skeptic on this one. At this stage, I consider it the equivalent of adding air to flat tire without patching the hole. The mutated huntingtin protein will continue to kill cells and the surgical insertion of stem cells (embryonic or adult) into the brain has its own problems and limitations. However, when a cure does come, there is a chance that this type of treament could be helpful in restoring brains that were damaged. Of course there is always the possiblity of delivering this type of treatment in a less-invasive manner.
There is an argument that could be made that a stem cell treatment for HD could create some type of cell that would be effective in helping protect the brain from the mutated huntingtin protein. This might be possible, however, I believe that there are other potential treatments with a greater likelihood for success and in a sooner timeframe.
Keep this in mind as you follow the convention next week. If we are lucky this will lead to a broader discussion on medical research. I'm somewhat doubtful. I suspect we'll see more 'heat' than 'light' on the subject.
Perhaps this will lead the Bush campaign to release more information on any medical research proposals they may have. So far they are presenting less information than Kerry on the subject, even though the current administration has done quite a bit of good so far(esp. the last 18 months). but time will tell.
Posted by Dave at 07:46 PM | Comments (1) | TrackBack
July 23, 2004
Vaccine Study Showing Success
Not for HD (see article from last year), but for Alzheimers. These vaccines are designed to identify specific 'bad' proteins and then use the body's own immune system to destroy them. Researchers are looking at doing the same for Huntington's Disease.
The results of the lastest Alzheimer's vaccine test are very promising...
Patients who received the vaccine scored better on memory tests a year later than those who were not given the vaccine; there was no perceptible difference in the patients' day-to-day functioning. In addition, an examination of the brains of four patients who died found a marked reduction in the plaque-like substance that typically fills space between brain cells in Alzheimer's.
Posted by Dave at 05:38 PM | Comments (0) | TrackBack
July 22, 2004
40+ CAG Counts & Early Cell Death
There has been an explosion of information on the huntingtin protein this year and here's the latest...
Researchers at Tufts University, with the help of an $1,000,000 from NIH have identified something unique that happens when a cell has 40 or more CAG repeats such as is the case in Huntington's Disease. The money quote:
They found that the proteins that signal that DNA damage is present are activated when a cell contains an expanded CAG repeat sequence.
"We know this because when we used cells that were defective in the checkpoint proteins, there was a large increase in chromosome breakage at the expanded repeat. This means that cells with expanded repeats are particularly vulnerable and if their surveillance mechanism fails for any reason they will probably die. This is important because cell death is a hallmark of most of the CAG expansion diseases, leading to neurodegeneration in Huntington's disease and muscle degeneration in myotonic dystrophy."
Here's the press release from Tufts:
Study by Tufts biologist provides window into progression of some degenerative diseases
Findings could lead to advances in tackling Huntington's disease, muscular dystrophy
MEDFORD/SOMERVILLE, Mass. – A Tufts University study has shed light on how some inherited diseases such as Huntington's and muscular dystrophy develop in humans.
"Our findings show a possible reason that cells with a certain type of mutation (expansion of repetitive DNA) die prematurely," said Catherine Freudenreich, assistant professor of biology at the School of Arts and Sciences at Tufts. "We may be able to use this information to stop or slow the development of some of these degenerative diseases that affect thousands of people every year."
She and her colleagues – post-doctoral fellow Mayurika Lahiri and former Tufts undergraduate researchers Tanya Gustafson and Elizabeth Majors – published their findings, "Expanded CAG repeats activate the DNA damage checkpoint pathway" in the July 23 issue of the journal Molecular Cell.
Freudenreich, a molecular biologist, studies the unstable elements in the human genome, particularly the type of unstable element called "trinucleotide repeat sequences," whose expansion causes numerous human genetic diseases such as Huntington's disease (a degenerative neurological disease) and myotonic dystrophy (a type of muscular dystrophy). There are more than 15 repeat expansion diseases, all of which are of special interest because they are caused by a highly unusual DNA mutation, one in which a repetitive DNA sequence expands from a small number of copies to a larger number. For example, 20 copies of a DNA sequence (such as CAG) could expand to 70 or 100 copies to cause disease.
With a grant for more than $1 million from the National Institutes of Health, Freudenreich's team investigated whether the presence of expanded repeats in a cell is recognized by the cell as damage and, if so, whether the cell activates the surveillance system that facilitates repair (called the "DNA damage checkpoint pathway"). They found that the proteins that signal that DNA damage is present are activated when a cell contains an expanded CAG repeat sequence.
"We know this because when we used cells that were defective in the checkpoint proteins, there was a large increase in chromosome breakage at the expanded repeat. This means that cells with expanded repeats are particularly vulnerable and if their surveillance mechanism fails for any reason they will probably die. This is important because cell death is a hallmark of most of the CAG expansion diseases, leading to neurodegeneration in Huntington's disease and muscle degeneration in myotonic dystrophy."
Although some of the reasons for the degeneration are understood, activation of the checkpoint pathway is a possible contributor that wasn't recognized before.
In the study, the researchers also found that when some checkpoint proteins were absent, the CAG repeat became very instable, contracting at an increased frequency. This means the checkpoint status of a cell can influence repeat stability.
"This is interesting because it still isn't understood why the repeat doesn't change size in some cell types, but is very unstable in others," Freudenreich said.
For example, in Huntington's disease the repeat is prone to expansion during sperm development, which leads to inheritance of even longer repeats in the resulting children and a worsening of the disease in the next generation. The CAG repeat also expands further in the affected brain cells of patients, which could explain why those brain cells die first. So identification of factors that limit expansion, such as these checkpoint proteins, could be very useful in controlling the inheritance and severity of the repeat expansion diseases.
"Freudenreich's study is an example of the innovative genetic research being done at Tufts and it will have long-term impact on future understanding of the mechanisms responsible for genomic instability and their relationship with certain inherited diseases," said Susan Ernst, dean of the School of Arts and Sciences at Tufts and a professor of biology.
###
EDITORS NOTE: Here is a link to Freudenreich's web page at Tufts: http://ase.tufts.edu/BIOLOGY/faculty/bios/freudenreich/freudenreich.html
Tufts University, located on three Massachusetts campuses in Boston, Medford/Somerville, and Grafton, and in Talloires, France, is recognized among the premier research universities in the United States. Tufts enjoys a global reputation for academic excellence and for the preparation of students as leaders in a wide range of professions. A growing number of innovative teaching and research initiatives span all Tufts campuses, and collaboration among the faculty and students in the undergraduate, graduate and professional programs across the University's eight schools is widely encouraged.
Posted by Dave at 05:29 PM | Comments (0) | TrackBack
July 21, 2004
Guthrie Song Spoof
Ok, ok...it get's a little serious around here at times. It's time for some humor.
JibJab.com has a hilarious spoof of Woodie Guthrie's "This Land is Your Land" satirizing the 2004 Presidential campaign. It's a Flash movie to the famous Guthrie tune 'starring' Bush & Kerry. Before you go to their website to see it, you need to know this:
It's about a PG-13.
It's bi-partisan, it makes fun of both presidential candidates (and others)
It's bound to offend some
If you're on a modem I hope you're patient. It's 3.7mb
It is funny
At least for now, it is available off of JibJab's front page. (If that fails, try this.)
Best thing about JibJab? They have their own blog.
Posted by Dave at 07:25 PM | Comments (0) | TrackBack
July 20, 2004
Ethics, Genetic Testing, and Eugenics
I see this discussion crop up from time to time and I think it is a good one to have...How far does society go in prenatal testing? At what point does it become Eugenics?
The Kalamazoo Gazette reports on the 'Genetic Town Hall' put on by Johns Hopkins University. Kalamazoo is the third stop on a multi-city tour. I'm not sure if Johns Hopkins is 'preachin', 'listenin', or 'discussin' but this is a topic dear to the heart of the Huntington's Disease community. And it is certainly worth discussing.
My question, and there is no answer..."Do you chose to have a child that would be at risk for Huntington's if you believe a cure will happen before the child would become a teenager?" We used to believe that Huntington's was a death sentence. That may not be the case for our generation. So what do we do now?
Neither the questions or the answers are easy on this one and there is no simple answer for any parent. I just hope that we, as a community, do not treat those with the HD gene as 'less' than the rest of of us. The consequences would be tragic (and wrong).
Posted by Dave at 06:39 PM | Comments (0) | TrackBack
MethylGene Update
As you know, MethylGene and EnVivo Pharmaceuticals are collaborating on using MethylGene's HDAC inhibitors as a treatment for Huntington's and other diseases. HDAC inhibitors show great promise in slowing down the progression of Huntington's Disease.
Today MethylGene issued their quarterly update. Based on the numbers given, it looks like the recent IPO has put them on solid financial ground. In addition, their EnVivo partnership looks like a great arrangement for both companies. This may be a company to keep an eye on. Here's the press release:
MethylGene Announces Second Quarter 2004 Results
Company Achieves U.S. $500,000 Milestone Payment for Initiating HDAC Cancer Clinical Trials
MONTREAL--July 20, 2004--MethylGene Inc. (TSX:MYG) a biopharmaceutical company, today announced operational and financial results for the second quarter ended June 30th, 2004. During the quarter, MethylGene successfully completed its initial public offering (IPO) and began trading its shares on the Toronto Stock Exchange under the symbol "MYG".
"The successful closing of MethylGene's initial public offering and concurrent private placement, raising a total of approximately $21.4 million, has solidified our financial position and will allow us to aggressively pursue our near term scientific and business objectives," said Donald F. Corcoran, President and CEO of MethylGene. "Our first reportable quarter as a public company was an active one, particularly with respect to our HDAC programs. We expect to continue this high level of activity for our preclinical, clinical and business development programs moving forward."
The quarter was highlighted by a number of events including:
- Commencement of two Phase I MGCD0103 dose escalation monotherapy
clinical trials, at sites in Canada and the United States, in
patients with advanced solid malignancies refractory to currently
available therapies. MGCD0103 is an orally active, isotypic
selective, small molecule HDAC inhibitor that inhibits a specific
subset of cancer-related HDAC enzymes, resulting in an activity
profile that is genotypically, phenotypically and functionally
distinct from indiscriminate HDAC inhibitors. Management
anticipates completing enrollment for these trials in the next 12
months and initiating a Phase I monotherapy trial in haematological
tumors this year.
- Achievement of a U.S. $500,000 milestone payment from research and
development partner Taiho for advancement of MGCD0103 into human
cancer clinical trials.
- Granting of U.S. patent #6,541,661 "Inhibition of Histone
Deacetylases", which provides protection for compounds and methods
for inhibiting histone deacetylase (HDAC) enzymes.
- Continued enrollment in a dose escalating monotherapy Phase I trial
for MG98, the Company's DNA methyltransferase inhibitor, in
patients with solid tumors. A randomized two-step Phase II trial
in metastatic renal cell cancer in combination with interferon
alpha is expected to commence in the third quarter of 2004.
- Continued advancement of the Company's beta-lactamase program for
generating small molecule inhibitors to overcome antibiotic
resistance. This program is partnered with Merck with the goal of
identifying a clinical candidate by the end of 2005.
- The exercise of an option by EnVivo Pharmaceuticals, a private U.S.
biotechnology company based in Boston, to enter into an exclusive
research, collaboration and license agreement with MethylGene to
exploit MethylGene's isotypic selective HDAC inhibitor technology
in neurodegenerative diseases such as Alzheimer's, Parkinson's and
Huntington's disease.
Financial Results
Total revenues for the second quarter ended June 30, 2004 were $3.5 million compared to $755,000 for the same period last year. Total revenues for the first six months of 2004 were $5.0 million compared to $1.5 million for the first six months of the corresponding period last year. For the second quarter of 2004, revenue consisted primarily of contract research payments and license fees of $3.3 million generated from Taiho, Merck and EnVivo collaborations, compared to revenues of $524,000 generated in the same period a year earlier from Vernalis, MGI Pharma and Merck collaborations.
Total expenditures for the second quarter ended June 30, 2004 were $4.6 million compared to $2.9 million for the same period last year. Total expenditures for the first six months of 2004 were $9.2 million compared to $6.4 million for the first six months of the corresponding period last year.
Gross research and development expenses were $3.9 million for the second quarter and $8.0 million for the first six months of 2004, up 37% from $2.8 million for the second quarter of 2003 and up 39% from $5.8 million for the first six months of last year, respectively. These increases were primarily due to increased clinical trial related spending and to higher salaries and benefit costs primarily due to expensing of non-cash stock option costs and increased headcounts.
General and Administrative expenses for the second quarter were $1.2 million and for the six months were $2.2 million, up 54% from $784,000 and 46% from $1.5 million respectively for the prior year, primarily due to increased salary and benefit costs relating to expensing non-cash stock options costs.
Net loss for the second quarter ended June 30, 2004 was $1.3 million and for the first six months was $4.3 million, down from $2.2 million and $5.0 million, respectively for the same periods last year.
On June 30, 2004 the Company had cash, cash equivalents and short-term investments totaling $45.9 million compared to $30 million at December 31, 2003. On June 29th, 2004 the Company completed an initial public offering and a concurrent private placement for total gross proceeds of approximately $21.4 million.
The number of shares outstanding as of July 16, 2004 were 21,340,189 common shares.
About MethylGene
MethylGene is a biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutics in cancer and infectious disease. Two cancer product candidates, MG98, partnered with MGI Pharma in North America and MGCD010, partnered with Taiho Pharmaceutical for certain Asian countries, are currently in clinical trials. MG98 is expected to enter a randomized two-step Phase II combination trial with interferon alpha in metastatic renal cell cancer in Q3 2004. MGCD0103 is currently in two Phase I dose-escalation monotherapy trials against solid tumors. In collaboration with Merck, MethylGene is developing small molecule beta-lactamase inhibitors to overcome antibiotic resistance. MethylGene has a portfolio of research programs including kinases and HDAC inhibitors for non-oncology indications such as neurodegenerative diseases to be exploited in collaboration with EnVivo Pharmaceuticals. Please visit our website at www.methylgene.com.
Except for historical information, this press release may contain forward-looking statements, which reflect the Company's current expectation regarding future events. These forward-looking statements involve risk and uncertainties,(which can be found in the Company's prospectus dated June 18, 2004 and can be found on www.sedar.com) which may cause but are not limited to, changing market conditions, the successful and timely completion of clinical studies, the establishment of corporate alliances, the impact of competitive products and pricing, new product development, uncertainties related to the regulatory approval process and other risks detailed from time to time in the Company's ongoing quarterly and annual reporting.
BALANCE SHEETS
(unaudited)
As at
June 30, December 31,
2004 2003
$ $
---------------------------------------------------------------------
ASSETS
Current
Cash and cash equivalents 19,055,017 2,664
Short-term investments 26,833,112 30,812,249
Research and development tax
credits receivable 2,205,000 3,760,000
Technology Partnership Canada
receivable 26,319 35,384
Receivables under collaboration
agreements 217,806 652,796
Other current assets 948,211 1,046,606
---------------------------------------------------------------------
Total current assets 49,285,465 36,309,699
Property, plant and equipment 4,297,268 4,202,369
Intangible assets 1,889,222 1,890,951
---------------------------------------------------------------------
55,471,955 42,403,019
---------------------------------------------------------------------
---------------------------------------------------------------------
LIABILITIES AND SHAREHOLDERS'
EQUITY
Current
Bank indebtedness - 825,522
Accounts payable and accrued
liabilities 2,476,755 2,604,257
Current portion of unearned
revenue 3,145,379 2,935,040
Current portion of long-term debt 6,579 13,160
Current portion on obligations
under capital leases 19,890 19,318
Total current liabilities 5,648,603 6,397,297
Unearned revenue 2,322,033 4,079,809
Long-term debt - -
Obligations under capital leases 16,665 26,756
---------------------------------------------------------------------
Total liabilities 7,987,301 10,503,862
---------------------------------------------------------------------
Shareholders' equity
Capital stock 72,981,459 54,079,575
Contributed surplus 4,139,140 3,195,272
Deficit (29,635,945) (25,375,690)
---------------------------------------------------------------------
Total shareholders' equity 47,484,654 31,899,157
---------------------------------------------------------------------
55,471,955 42,403,019
---------------------------------------------------------------------
---------------------------------------------------------------------
STATEMENTS OF OPERATIONS AND DEFICIT
(unaudited)
For the periods ended
Three-month periods Six-month periods
ended June 30, ended June 30,
---------------------------------------------------------------------
2004 2003 2004 2003
$ $ $ $
--------------------------------------------------------------------
REVENUES
Contract revenues
and license fees 3,284,992 524,490 4,723,528 1,017,156
Interest income 178,018 230,336 302,703 456,866
---------------------------------------------------------------------
3,463,010 754,826 5,026,231 1,474,022
---------------------------------------------------------------------
EXPENSES
Research and
development 3,893,408 2,846,144 8,021,130 5,773,795
Government
assistance (430,266) (1,072,323) (911,066) (1,603,176)
---------------------------------------------------------------------
Net research and
development 3,463,142 1,773,821 7,110,064 4,170,619
General and
administrative 1,206,390 784,035 2,226,101 1,529,085
Amortization of
intangible assets 28,166 24,759 55,534 48,680
Amortization of
property, plant
and equipment 13,421 11,400 26,601 22,379
Write-down of
patents 157,433 - 157,433 -
Bank charges and
interest 4,931 4,736 18,641 7,926
Foreign exchange
loss (gain) (225,338) 346,072 (376,058) 656,327
4,648,145 2,944,823 9,218,316 6,435,016
Net loss before
income taxes (1,185,135) (2,189,997) (4,192,085) (4,960,994)
Income taxes 68,170 - 68,170 -
Net loss for the
period (1,253,305) (2,189,997) (4,260,255) (4,960,994)
Deficit,
beginning of
period (28,382,640) (22,671,790) (25,375,690) (19,900,793)
---------------------------------------------------------------------
Deficit, end of
period (29,635,945) (24,861,787) (29,635,945) (24,861,787)
---------------------------------------------------------------------
---------------------------------------------------------------------
Basic and diluted
loss per share (0.08) (0.13) (0.26) (0.30)
---------------------------------------------------------------------
Weighted average
number common of
shares 16,417,248 16,464,706 16,361,927 16,464,706
---------------------------------------------------------------------
STATEMENTS OF CASH FLOWS
(unaudited)
For the periods ended
Three-month periods Six-month periods
ended June 30, ended June 30,
---------------------------------------------------------------------
2004 2003 2004 2003
$ $ $ $
---------------------------------------------------------------------
OPERATING
ACTIVITIES
Net loss for the
period (1,253,305) (2,189,997) (4,260,255) (4,960,994)
Item not affecting
cash:
Amortization of
property, plant
and equipment 289,780 241,934 565,068 464,118
Amortization of
intangible assets 28,166 24,759 55,534 48,680
Unrealized foreign
exchange (gain)
losses 157,923 (40,225) 176,482 95,337
Write-down of
patents 157,433 - 157,433 -
Loss on disposal
of property, plant
and equipment - 1,662 - 6,944
Non-cash
compensation
expense 498,814 - 943,868 -
---------------------------------------------------------------------
(121,189) (1,961,867) (2,361,870) (4,345,915)
Changes in
non-cash working
capital balances
relating to
operations 1,889,842 (379,526) 1,754,391 (1,817,798)
Changes in
long-term portion
of unearned
revenues (782,136) (234,355) (1,757,776) (468,710)
---------------------------------------------------------------------
Cash flows related
to operating
activities 986,517 (2,575,748) (2,365,255) (6,632,423)
---------------------------------------------------------------------
INVESTING
ACTIVITIES
Acquisitions of
property, plant
and equipment, net
of government
assistance (349,847) (295,440) (659,967) (557,906)
Acquisitions of
intangible assets (199,075) (52,002) (211,241) (122,609)
Purchases of
short-term
investments (40,648,110) - (63,311,240) (14,403,661)
Proceeds from
maturities of
short-term
investments 39,541,393 8,123,508 67,113,900 27,750,465
Proceeds from
disposition of
property, plant
and equipment - 12,002 - 12,827
---------------------------------------------------------------------
Cash flows related
to investing
activities (1,655,639) 7,788,068 2,931,452 12,679,116
---------------------------------------------------------------------
FINANCING
ACTIVITIES
Issuance of common
shares 21,392,700 - 21,392,700 -
Redemption of
common shares (28) - (28) -
Share issue costs (2,064,894) - (2,064,894) -
Repayment of
obligation under
capital leases (4,794) (4,472) (9,519) (4,472)
Repayment of
long-term debt (3,290) (6,206) (6,581) (12,414)
---------------------------------------------------------------------
Cash flows related
to financing
activities 19,319,694 (10,678) 19,311,678 (16,886)
---------------------------------------------------------------------
Increase in cash,
cash equivalents
and bank
indebtedness 18,650,572 5,201,642 19,877,875 6,029,807
Cash, cash
equivalents and
bank indebtedness,
beginning of
period 404,445 213,295 (822,858) (614,870)
---------------------------------------------------------------------
Cash, cash
equivalents and
bank indebtedness,
end of period 19,055,017 5,414,937 19,055,017 5,414,937
---------------------------------------------------------------------
---------------------------------------------------------------------
Cash and cash
equivalent consist
of:
Cash 1,216,828 501,600 1,216,828 501,600
Cash equivalent 17,838,189 4,913,337 17,838,189 4,913,337
---------------------------------------------------------------------
Supplemental
information
Interest paid 648 1,329 11,567 1,915
Income tax paid 68,170 - 68,170 -
---------------------------------------------------------------------
---------------------------------------------------------------------
Posted by Dave at 06:20 PM | Comments (0) | TrackBack