Another bit of primary source - a Pharming Press release (edit: since added as a comment, rather than as a blog entry. That's where I'll put such material from now on. Like a footnote.)
Some interesting undercurrents here. Firstly, the competition with Synpac (see next entry) seemed to drive Pharming towards 'talking up' the trials to investors. No wild claims, of course, but perhaps making sure that investors in their stock hung on. Interesting to note that the Belgian government put a large sum towards the building of the plant in Geel. That PR investment obviously paid off!
We began to see though that there was some tension between the needs of Pharming as a business, the principles of the scientists involved and the thirst for knowledge amongst the patient community. There was a substantial overlap between all three, of course - however those tensions would also grow and would need careful management in the years ahead.
There were changes in the Genzyme/Pharming relationship too. We were told that communication would in future be through the local Genzyme representative, rather than through Gerben Moolhuizen at Pharming. This made some sense - it freed up Gerben for the work he was supposed to be doing - but also raised the potential for divide and rule. Plus, as rapidly became clear, our connections with scientists, doctors and indeed within the various companies, meant that the patient representatives were usually better informed about the Pompe project than the local country reps, who had a wide range of responsibilities. We would soon evolve a system where local reps dealt with individual patients and Genzyme HQ dealt with the representative organisations.
For entirely unconnected reasons, Gerben Moolhuizen left to pursue his career elsewhere at the end of May 1999. I, like many others, was sorry to see him go.
Tellingly, his place as a patient representative contact was taken by someone from Genzyme - Andrew Curtis. Another good guy, by the way.
Monday, 21 December 2009
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Pharming Group N.V. Reports Q1 Earnings 1999
ReplyDeletePhase II Clinical Trial for Pompe’s Disease on Track
Leiden, The Netherlands, May 3, 1999 – Pharming Group N.V. (Easdaq:
PHAR) today announced financial results for its first quarter ended March
31, 1999.
Pharming reported that revenues for its first quarter of 1999 increased to
EUR 1.9 million, from EUR 0.1 million in the first quarter 1998. The revenues
comprised payments from corporate partners. Expenses increased in Q1
1999 to EUR 5.6 from EUR 3.7 million in Q1 1998 reflecting, amongst other
factors, the continued development of human alpha-Glucosidase for the
treatment of Pompe’s disease. Net loss for the first quarter amounts to EUR
3.1 million, compared to EUR 3.6 million in the same period in 1998
(excluding exceptional charges in Q1 1998 of EUR 7.1 million for acquired
technologies).
As of March 31, 1999, Pharming had cash and cash equivalents of EUR
67.4 million, primarily invested in term deposits. Pharming believes that its
current cash position will allow it to reach its next phase of development,
namely the market launch of its lead biopharmaceutical product human
alpha-Glucosidase for Pompe’s disease, as well as further developing its
product portfolio.
Highlights Q1
Phase II clinical trial for Pompe’s disease on track
Second US Orphan Drug Designation for human C1 Esterase inhibitor
Cattle transfer from The Netherlands to Finland completed
First patent for transgenic animal technology in Japan
EUR 2.7 million grant from Flanders Government to support
expansion of Pharming’s facilities in Geel, Belgium
Product Development
In the first quarter of 1999, patients were enrolled in the first-ever
clinical
study of human alpha-Glucosidase to treat Pompe’s disease at the Sophia
Children’s Hospital in Rotterdam, the Netherlands. The pilot Phase II trial
includes juvenile and infantile patients. Each patient in the trial will be
treated for six months. The infantile segment of this study is fully underway.
On basis of observations from the current Phase II Clinical Study, we expect
to expand the current trial also at other centers in the second quarter. An
interim update with respect to this study can be expected later in the
second quarter of 1999. This study will be followed by pivotal trials expected
to begin in mid 1999 in Europe and the US. This program is the focus of a
joint venture between Pharming and Genzyme General to develop and
commercialize world-wide the enzyme human alpha-Glucosidase as a
treatment for Pompe’s disease, a lethal hereditary muscle disease. Human
alpha-Glucosidase has been designated an Orphan Drug by the US Food
and Drug Administration (FDA). Upon successful completion of the
development program, the Pharming/Genzyme joint-venture will obtain 7
years of market exclusivity in the US once its product receives marketing
approval from the US FDA.
In the first quarter of 1999, Pharming obtained its second Orphan Drug
Designation from the US FDA for recombinant human C1 Esterase inhibitor,
in both prophylaxis and acute treatment of hereditary and acquired
angioedema. Hereditary angioedema (HAE) is an autosomal dominant
disorder that results in a deficiency of functional human C1 Esterase
inhibitor. The deficiency causes a runaway reaction of the immune system
and clinical symptoms include swelling of the skin, abdominal organs, and
upper airway tract. Hereditary angioedema seriously affects quality of life
and can lead to life-threatening complications. The number of patients in the
western world suffering from HAE is estimated to be between 10,000 and
50,000.
Patents
ReplyDeletePharming received its first patent for transgenic animal technology in Japan
from the Japanese patent office, covering the use of casein promoters for the
production of biopharmaceuticals in the milk of transgenic animals. In addition, Pharming’s received its 10th US patent, covering the purification of human Lactoferrin from milk. The total number of patents increased to 22, while at year-end 1998 the number was 19.
Corporate Affairs
Pharming N.V., the Belgium subsidiary of Pharming, received a grant of EUR 2.7 million from the Flanders Government to expand facilities and product development programs in Geel, Belgium, particularly the
construction of a state-of-the-art plant for the purification of biopharmaceuticals from the milk of transgenic rabbits.
AEX Listing
In view of unfavourable stock market conditions for biotechnology companies
world-wide, Pharming postponed its co-listing on the Amsterdam Exchanges until further notice. Pharming has been quoted on the pan-European exchange EASDAQ since July 2, 1998.
"Pharming has had a strong start to the year, and our lead programme, the Phase II clinical trial with human alpha-Glucosidase for Pompe’s disease, is progressing well," said George J. M. Hersbach, President and Chief
Executive Officer of Pharming Group N.V. "We also received our second Orphan Drug Designation for human C1 Esterase inhibitor, which is very good news for our product development portfolio and testimony to the
excellent performance of our Orphan Products business unit. Pharming has a very strong cash position that will allow us to support the market launch of human alpha-Glucosidase, and to further develop our product portfolio."
Pharming focuses on the development, production and world-wide commercialisation of human therapeutic proteins, produced at high levels in the milk of transgenic animals that have been generated using the Pharming’s proprietary technology.
Statements included in this press release which are not historical in nature are intended to be, and are hereby identified as, "forward-looking statements" for purposes of the safe harbour provided by Section 21E of the Securities Exchange Act of 1934, as amended by the Private Securities
Litigation Reform Act of 1995. Forward-looking statements may be identified
by words including "anticipates", "believes", "intends", "estimates",
"expects" and similar expressions. The company cautions readers that
forward-looking statements, including without limitation those relating to the
company's future operations and business prospects, are subject to certain
risks and uncertainties that could cause actual results to differ materially
from those indicated in the forward-looking statements. Factors that could
cause actual results and future operations and business prospects to
materially differ from those anticipated in these forward looking statements
include, but are not limited to, clinical and scientific results and
developments concerning corporate collaborations and the company's
proprietary rights and other known and unknown risks including risk factors
described in the prospectus relating to the company's recent initial public
offering. Pharming Group N.V. cautions readers not to place undue reliance
upon any such forward-looking statements, which speak only as of the date
made. Pharming expressly disclaims any obligation or undertaking to
release publicly any updates or revisions of such statements to reflect any
changes in Pharming’s expectations or any change in events, conditions or
circumstances on which any risk statement is based.