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Cafe69, this is the best post of the year. At least to me. Annapurna base camp would be amazing. Are you located in the states?
nothing final yet, and I will report back with all the details if everything pans out.
IGOATOC: i go on a tour of cytogenix
ITMSOTL: i take MS out to lunch
either one would be great.
you guys are just too smart. I am actually planning on climbing Mt. St. Hellens in August, so that is kinda funny.
lol, wow, you are pretty good. I guess my code was pretty weak. You figured it out too fast, so I will have to answer your question later. Once some other secret code things happen.
like: IGOATOC or ITMSOTL
hopefully! fingers crossed.
New 52 week low. Sad day.
ICMST <- secret message
LOL, so true. As long as the 666 doesn't get near my head or arm Ill be okay.
I did see the picture of the property on the info page. Pretty sweet. However, the more I look at that pic, the more I feel sorry for that poor sign I made. It looks as if it knows that its message is but a false hope in the middle of a great big lie. That the field in which it lies will never actually be the home of Cytogenix. Poor sign; goodbye money.
I can only hope that CYGX will suprise everone.
I just noticed that in one of my small initial accounts, I own exactly 666 shares of CYGX. Is it a sign?
I unfortunately will not be able to attend the share holders meeting. This is quite sad due to the fact that I am so close by. I could basically walk there. However, I have a commitment called a job. So, whoever is going, please take good notes. No, actually take great notes. That way you can share with us after the fact what the heck is going on. If possible, record it, either just audio or also video. That would be superb! Thanks a lot!
Cheers to all!
I wish I could have been there holycow. I would love to talk with Malcolm. I am so confused about this company. This is one of those times I wish I had a crystal ball so I could see what Malcolm says behind closed doors.
Nice find Paulness. In the end, Cytogenix will sure have an interesting story whether they crash or whether they fly.
BioMarin Submits New Drug Application for U.S. Marketing Authorization of Kuvan for Phenylketonuria (PKU)
NOVATO, Calif., May 24, 2007 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. announced today that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for Kuvan (sapropterin dihydrochloride), an investigational oral small molecule for the treatment of patients with phenylketonuria (PKU) being developed in partnership with Merck Serono. Merck Serono expects to file a Marketing Authorization Application (MAA) for Kuvan with the European Medicines Agency (EMEA) in the third quarter of 2007.
"This NDA filing represents a significant milestone in our PKU program and our efforts to bring the first treatment option to PKU patients," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "The NDA filing contains data evaluating Kuvan in approximately 650 human subjects in six clinical studies and represents BioMarin's largest and most comprehensive filing to date. The company is now gearing up for the U.S. launch of Kuvan in late 2007 or early 2008."
At a pre-NDA meeting in October 2006, BioMarin met with the FDA to discuss key information on Kuvan to be included in the NDA. Consistent with the discussions at that meeting, the fully electronic NDA filing includes a comprehensive set of preclinical, clinical and manufacturing related data on Kuvan. As part of the NDA submission, BioMarin has requested priority review status. The criteria for assigning priority review status to an application are similar to those for fast track status, which Kuvan has already received. If the FDA accepts the NDA and grants the request for priority review, the FDA is expected to take action on the application within six months of its submission. Kuvan also has orphan drug designation, which allows for seven years of market exclusivity within the United States following the approval of the marketing application.
About Kuvan
Kuvan is an investigational oral small molecule therapeutic for the treatment of PKU. The active ingredient in Kuvan, sapropterin dihydrochloride, is the synthetic form of 6R-BH4 (tetrahydrobiopterin), a naturally occurring enzyme cofactor that works in conjunction with phenylalanine hydroxylase (PAH) to metabolize Phe. Clinical data suggests that Kuvan produces significant reductions in blood Phe levels in BH4-responsive patients. BioMarin and Merck Serono estimate that Kuvan could be a potential treatment option for approximately 30 percent to 50 percent of the estimated 50,000 identified PKU patients in the developed world.
Kuvan has received orphan drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMEA). Upon approval, it will receive seven years of market exclusivity in the United States and 10 years in the European Union for this indication. Additionally, the FDA has granted Kuvan Fast Track designation, which is designed to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.
About PKU
PKU, a genetic disorder affecting approximately 50,000 diagnosed patients in the developed world, is caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH). PAH is required for the metabolism of phenylalanine (Phe), an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and brain, resulting in a variety of complications including severe mental retardation and brain damage, mental illness, seizures and tremors, and cognitive problems. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all PKU patients under the age of 40 in developed countries have been diagnosed at birth. The only treatment currently available for PKU patients is a highly restrictive and expensive medical food diet that most patients fail to adhere to the extent needed for achieving adequate control of blood Phe levels. To learn more about PKU, please visit www.PKU.com. Information on this website is not incorporated by reference into this press release.
About BioMarin
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises two approved products and multiple clinical and preclinical product candidates. Approved products include Naglazyme(R) (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin, and Aldurazyme(R) (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation. Investigational product candidates include Kuvan (sapropterin dihydrochloride), a Phase 3 product candidate for the treatment of phenylketonuria (PKU), and 6R-BH4 for cardiovascular indications, which is currently in Phase 2 clinical development for the treatment of peripheral arterial disease and sickle cell disease. For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.
OT: holycow. I have heard this before. One theory is that a higher body temperature relates to an increase in oxidation events which can lead to cancer. It's basic thermodynamics... the hotter it gets the faster reactions occur, etc. Also related to diffusion to some degree. Anyway, calorie restriction reduces these events. Search "calorie restriction lowers body temperature" if you want more info. Oh, btw, resveratrol is theorized to do the same. Having blood work done next week too so hopefully I will see before resveratrol data vs 5 months on resveratrol data. Will report back.
Never heard of chiggers before, but I will be on the lookout... or feelout if they are too small to see. We have ticks all over in Washington, I hate when those things burrow into you.
Unfortunately I did not get a picture of the stakes. I saw quite a few of them, but never really went out to look closely at one. Next time I will.
Those are all the pictures I took. Nothing too exciting or revealing. When/If they break ground, it will be cool to watch it being constructed. And it is really close to my house so I could easily give updates to everyone. Anyways, the whole guard thing made my night exciting so it was worth it. Y'all have a great night!
Now here are the more important photos, however they are not very exciting. Just a bunch of grass.
Grass.
More grass.
Even more grass. The building on the far far far left is Oak Park. In the middle way off in the distance in Spectracell Labs.
These pictures are the Oak Park building... our other neighbor.
The right portion of field in this picture is our lot.
This section of the field is also ours as well, supposedly.
You can see Oak Park in the back.
A sign for Oak Park.
Here are the next set of pictures:
This is some new construction that is taking up a portion of the lot that I had originally thought was CYGX's. Because of the new buildings and the new road, our lot is a lot smaller. But definitely big enough for a good sized building.
Spectracell Laboratories, our neighbor
Well, my mission was a success. I got some pictures, trekked around Cytogenix's new property for a bit, and got a general feel for the area. As everyone probably knows, ground has not been broken. What you will see is a nice grassy green field.
So here is the original satellite image:
Supposedly Cytogenix's land is just north of the bow-tie shaped building (aka: Halliburton)
However, upon arrival, I found out that the area has been developed quite a bit since that satellite photo was taken. This is my rough sketch of the area. The red circles with P1-P11 correlate to the location in which my 11 pictures were taken.
Now before I go on to the actual pictures I want to recount a short story. As I got out of my car to take a picture of Halliburton, I jumped up on a construction road block so I could get a nice view. As soon as I had raised my camera to my eye, two guards ran out from the guard house yelling at me. "Stop stop!! Put that down right now!!," they yelled. One of the guards quickly reached my location and the conversation went on like so:
Gaurd: "Stop, put that camera down right now"
Me: "Am I doing something wrong here?"
G: "You cannot take pictures, now put it down"
Me: "I am not sure what exactly I am doing wrong. Is taking pictures of Halliburtan illegal?"
G: [awkwardly silent for a few seconds] "...well, um... you might use it to break in"
Me: "I am going to break in using a picture of your front door?"
G: [more silence, and a befuddled look] "...uh... what are you doing then?"
I attempted to explain truthfully what I was doing, scoping out the area for cytogenix, blah blah blah
G: "Do you have ID?"
Me: "A drivers license"
G: "Let me see it"
Me: "No" (I said it nicely)
G: [looking at the ground shifting his weight, not really sure what to do] "What is your name?"
Me: "I'd rather not say"
G: [now obviously very uncomfortable and very quiet] ... uh...
Me: "I am just going to take some pictures and be on my way."
G: "...uh.. okay..."
And then he slowly walked away, like a puppy with his tail between his legs. Honestly if I had video taped the whole situation it was very very funny because the poor guard had no idea what to do. And the fact that he sprinted out yelling at me like he was some cop stopping a murder or something... the whole thing still seems like a dream to me because it was so comical and unreal. I have to admit I didnt really know what I was doing either though... I have no idea what laws / rules there are about photographing building. Anyway, here is the picture:
Im bored, so I am going out to the new property to take some pics. Hopefully post them tonight for y'all.
Nice interview today. Kinda vague, like usual, but definitely positive. The only thing that is really bugging me is when I spoke to him a couple months ago he said we were just a couple (2-3) weeks away from having ground breaking. I understand things can take longer than expected, but why does EVERYTHING take longer? When I worked at Genentech they really emphasized setting realistic targets and goals and then following through with those. There comes a point when someone cries wolf so many times that it is hard to believe anything they say. Many of Malcolm's interviews give drastically misleading timelines and expectations. This interview does make me feel better about my investment, but unless things actually come to fruition it doesn't matter how I feel.
Since COR has now reached the $3 mark, could it be possible that a new set of options will become available? If so I would be a pretty happy camper.
Lol, education is definitely what you put into it.
Hopefully this week will be as amazing for BMRN as last week was.
That was a pretty funny comment about the IQ points. The whole presentation was kinda weird, not as professional sounding as previous ones. Good things seem to be coming though and the recent run-up this week confirms that. This next presentation should be good as well:
BioMarin to Present at the Citigroup Global Healthcare Conference
Wednesday May 16, 4:00 pm ET
NOVATO, Calif., May 16 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN - News News) today announced that Jean-Jacques Bienaime, Chief Executive Officer of BioMarin, will present a company update at the Citigroup Global Healthcare Conference in New York City on Wednesday, May 23, 2007 at 3:30 p.m. ET.
Interested parties may access a live audio webcast of the presentation via the investor section of the BioMarin website, www.BMRN.com. A replay of the presentation will be archived on the site for at least one week following the presentation.
thanks. eom
thanks Paulness. Do you know when and where the share holders meeting is? Can you just show up or do you have to make reservations. If I can make it I would love to go. Thanks in advance to anyone that can answer these questions.
OT: Hey everyone! I just got moved into my new place in Houston. Man, I already love this city 10 times more than San Francisco. I do miss Genentech a lot, because it is such a great company, but that city is just horrendous! Houston on the other hand is already wowing me and I am having a great time. Should be a super summer. Tomorrow is my first day for my second internship with BP. Once things cool off a bit I will be more active on this board and I plan on visiting my new neighbor (Cytogenix) shortly. CYGX is actually very close to where I live. Oh, does anyone recall the address of our new property? I want to go take some pics, watch the construction of the new building, etc etc.
NOT OT: Paulness, the most recent news seems very positive to me. Eurogentec is a great collaboration partner. Check out their earnings for the past few years. Great growth, excellence in their arena, other collaborations with some big names.... all looks almost too good to be true. I really hope this collaboration is fruitful and will yield some tangible results. I especially like this:
'Dr. Phillippe Cronet, Director of Eurogentec's Biologics Business Unit, commented, "CytoGenix synDNA(TM) has the potential to make a significant impact as a source of drug substance for a wide array of DNA-based medicine. We are looking forward to establishing a collaborative relationship with CytoGenix."'
Some of the wording in the PR had me a bit concerned at first, but after a reread, it seems like a great news release to me. I haven't had time yet to read this board since I left, but I hope others saw it as a valuable PR as well. Cheers.
BioMarin Pharmaceutical "outperform," estimates raised
Tuesday, May 01, 2007 4:00:10 AM ET
Robert W. Baird
NEW YORK, May 1 (newratings.com) - Analysts at Robert W Baird reiterate their "outperform" rating on BioMarin Pharmaceutical Inc (BMRN.NAS), while raising their estimates for the company. The target price is set to $22.
In a research note published this morning, the analysts mention that the company has posted its 1Q EPS ahead of the estimates as well as the consensus. BioMarin Pharmaceutical has raised its Naglazyme revenue guidance for 2007 from $74-$78 million to $76-$82 million and, hence, has reduced its net loss guidance for the year from $20-$25 million to $18-$23 million. The EPS estimate for 2007 has been raised from -$0.22 to -$0.21.
Great quarter as per Leerink
Strong Quarter - Yesterday after the close, BMRN reported 1Q EPS of ($0.10), a penny ahead of our and consensus estimates of ($0.11). Variance primarily driven by lower R&D expenses. Total revenues of $22.8MM were less than our estimate of $24.1MM and consensus of $23.8MM, due to lower Orapred and Collaboration revenues.
Conference call has been moved up by one day to Tuesday, May 1 at 1pm EDT. Dial in is 1-800-261-3417, password is 47185129.
BMRN raised 2007 Naglazyme guidance to $76-82MM, from a prior range of $74-78MM, and reiterated Aldurazyme sales guidance of $115-125MM. This suggests to us that the Naglazyme launch remains robust even before new markets in Latin America and the Middle East, while relatively flat 1Q performance for Aldurazyme may be due to seasonality.
Kuvan remains on track for NDA submission in 2Q:07 and MAA submission in 3Q:07. BMRN continues to expect US launch in 2007, assuming favorable FDA decision based on priority (6 months) review. FDA has approved Kuvan's expanded access program, and physicians and patients representing more than 70 centers in the U.S. have responded with interest, which should bode well for eventual market uptake.
Phenylase advancing for the remainder of PKU market. Once weekly subcutaneous injections resulted in a sustained decrease in blood Phe to levels within a normal range for a 12-week period. BioMarin plans to file an IND by the end of 2007 and to be in the clinic in early 2008.
BMRN is amending BH4 protocol for the ongoing Phase II PAD trial to take advantage of post-hoc subgroup analyses showing a greater effect in more severe patients. Measures of endothelial dysfunction and oxidative stress will be added to the protocol at several sites, and vitamin C will be added to half of patients in the study to see if synergy is derived. Phase II trial for sickle cell disease and PAH are both expected to begin this quarter.
Reiterate Outperform - With a strong enzyme replacement franchise, emerging PKU franchise, and longer term potential for BH4 remaining in treating chronic cardiovascular diseases, BMRN is an attractive investment, in our opinion. BMRN is on the Leerink Swann Focus List.
We derive a $21 per share mid-2007 fair value based on 8x 2008E revenue share of $267MM discounted at 20% for half a period.
[thanks pharmer10 at yahoo message board]
Yes I am indeed back. I wrote a nice long post about my trip earlier this week but when I tried to post it my browser crashed and lost the whole thing. Unfortunately I have been too busy to retype that. My last day at Biomarin is tomorrow so I have a huge presentation to give and then I pack up and leave for Houston on Friday. Somewhere in there also falls a week long vacation to Mexico. So for the next three weeks I will pretty much be absent. As for the trip to Whitney, oh man it was a blast! Unfortunately I did not get to summit due to two major snow storms back to back. Got nuked on very hard, about 28 inches, and there were 75 mph winds during one of the storms... we will just leave it at that for now. Pictures and a more complete story may follow within a month.
As far as the PR today, looks very nice. I definitely believe that I am either going to lose all my money and look like a fool with this investment, or I am going to really strengthen my patience, build my character, and make a good return on my money once synDNA really truly makes it big. For now it is hard to say which way the dice are going to land. The PR increased my confidence... for this week. Once I get to Houston I want to meet up with CYGX, maybe take a tour, and snap a few photos of the plot of land where the new building will be built. If I tried to get a lunch meeting with MS, would anyone be in the area to join me or be willing to come? Just curious.
Unfortunately I am going to be very busy the next two/three weeks so I will have to do more updates on this board then. Initial reaction for me is positive for the quarterly results. It beat the estimate. Other things in the work seem to be looking good too like BR3 trials and Kuvan. I am expecting BMRN to inlicense a drug or possibly buy out another company with all the money it has (almost half a billion!!). We shall see....
Cheers to all and I will be more responsive in a month or so.
BioMarin Pharmaceutical Inc. Earnings Conference Call (Q1 2007)
Scheduled to start Tue, May 1, 2007, 12:00 pm Eastern
http://biz.yahoo.com/cc/7/78487.html
BioMarin Announces First Quarter 2007 Financial Results
Monday April 30, 5:00 pm ET
Q1 2007 Results Driven by Improved Naglazyme Sales - Raising Guidance
Conference Call and Webcast to Be Held May 1 at 12:00 p.m. ET (18:00 CET)
NOVATO, Calif., April 30 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN - News News) today announced financial results for its first quarter ended March 31, 2007. The net loss was $9.3 million ($0.10 per share) for the first quarter of 2007, compared to a net loss of $9.8 million ($0.13 per share) for the first quarter of 2006. The net loss during the first quarter of 2007 includes $3.6 million of non-cash stock compensation expense, compared to $1.7 million of non-cash stock compensation expense during the first quarter of 2006.
As of March 31, 2007, BioMarin had cash, cash equivalents, and short-term investments totaling $273.3 million. Including the proceeds from the public offering of senior subordinated convertible notes in April 2007, on a pro- forma basis, BioMarin had cash, cash equivalents, and short term investments totaling approximately $590 million.
"Strong Naglazyme sales are driving better than expected bottom-line results and fueling the development of our pipeline. Based on the upside to first quarter sales, we are increasing our Naglazyme sales expectations for 2007. We are also improving our net loss guidance due to higher interest income and slightly higher Naglazyme sales, partially offset by higher spending on preclinical and clinical development programs, as well as Kuvan launch preparation," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin.
Mr. Bienaime continued, "We remain on track to submit the NDA for Kuvan by the end of this quarter and, assuming that we receive priority review, anticipate potential approval by the end of the year. We are also pleased that our expanded access program for Kuvan has been approved by the FDA, and physicians and patients representing more than 70 centers in the U.S. have responded with interest, which we believe bodes well for the potential launch of Kuvan. We also remain encouraged regarding the possible application of 6R- BH4 in treating cardiovascular disease and plan to conduct additional studies to further characterize the effect of BH4 in patients with endothelial dysfunction. Additionally, our recent convertible note offering has provided us with the financial flexibility to pursue growth opportunities that will further augment our pipeline and longer-term growth prospects."
Product Sales
Net sales of Naglazyme® (galsulfase), an enzyme replacement therapy for mucopolysaccharidosis VI (MPS VI), were $18.4 million for the first quarter of 2007, compared to $16.3 million for the fourth quarter of 2006, representing a sequential increase of approximately 12.9 percent. Naglazyme net sales for the three months ended March 31, 2006 were $7.0 million. BioMarin is commercializing Naglazyme in the United States, Europe, and Latin America, and through distributors in other international markets.
Net sales of Aldurazyme® (laronidase), an enzyme replacement therapy for mucopolysaccharidosis I, (MPS I) by BioMarin/Genzyme LLC increased by approximately 25.8 percent to $26.8 million for the first quarter of 2007, compared to $21.3 million in the first quarter of 2006. Aldurazyme net sales for the three months ended December 31, 2006 were $26.5 million. BioMarin's share of the profit of BioMarin/Genzyme LLC was $6.2 million for the first quarter of 2007, compared to a profit of $3.8 million for the first quarter of 2006.
Royalty and License Revenues
Royalty and license revenues for the first quarter of 2007 were $0.4 million, and include royalties on net product sales of the Orapred product line, including Orapred® (prednisolone sodium phosphate oral solution) and Orapred ODT(TM) (prednisolone sodium phosphate orally disintegrating tablets). BioMarin expects to receive an additional milestone payment of $4.0 million on the first anniversary of FDA approval of Orapred ODT in June 2007.
Updated Financial Guidance
2007 Projected Net Product Sales
BioMarin estimates 2007 net sales of Naglazyme to be in the range of $76 million to $82 million from a previous range of $74 million to $78 million and 2007 sales of Aldurazyme by the joint venture to be in a range of $115 million to $125 million.
2007 Projected Net Loss
BioMarin estimates its GAAP net loss for the fiscal year ending December 31, 2007 to be in the range of $18 million to $23 million from a previous range of $20 million to $25 million. This includes approximately $16 million to $18 million in non-cash stock compensation expense. Additionally, the 2007 projected net loss includes the $4 million Orapred ODT milestone payment and a $15 million milestone payment from Merck Serono in the second half of 2007 for the acceptance of the MAA filing for Kuvan.
Update on 6R-BH4 Program for Cardiovascular Indications
Post-hoc sub-group analyses of the Phase 2 trial for uncontrolled hypertension revealed a greater effect of BH4 in patients with higher systolic blood pressure of >150 mm Hg than in patients with systolic blood pressure <150 mm Hg, with a within group p-value of 0.004. The placebo group did not show a difference when comparing placebo patients with greater or less than 150 mm Hg in systolic blood pressure. When comparing 6R-BH4 to placebo in patients with a mean systolic blood pressure of >150 mm Hg, the BH4 group had a mean systolic blood pressure reduction of 14.1 mm Hg, compared to a mean systolic blood pressure reduction of 5.9 mm Hg in the placebo group, a difference of 8.2 mm Hg that did not reach statistical significance with a p- value trend of 0.142. These are post-hoc analyses and can only help provide insights that will need to be confirmed in prospectively designed studies. In an effort to better understand the role of BH4 in treating endothelial dysfunction and cardiovascular disease, BioMarin plans to initiate two small exploratory studies in the second half of 2007:
* A double-blind placebo-controlled study to assess the effect of 6R-BH4
on endothelial dysfunction in approximately 40 subjects with more
severe systolic hypertension (SBP >145 mm Hg) and with confirmed and
significantly impaired endothelial function.
* A clinical study to assess the effect of co-administration of Vitamin C
with 6R-BH4 on 6R-BH4 pharmacokinetics and efficacy in treating
endothelial dysfunction in approximately 40 subjects with more severe
hypertension (SBP >150 mm Hg) and significantly impaired endothelial
function. Vitamin C can have a direct oxidation protective effect on
BH4, and the level of Vitamin C in the formulation from the prior
hypertension study was much higher than in the current 6R-BH4
formulation. We will assess whether this protective effect is
meaningful in patients with cardiovascular disease.
After reviewing the results of the hypertension study in greater detail, a few additions have been made to the protocol for the ongoing Phase 2 trial for peripheral arterial disease. Measures of endothelial dysfunction and oxidative stress will be added to the protocol at several sites, and given the potential that increased Vitamin C might protect BH4 from oxidation in patients, co-administration of Vitamin C with BH4 or placebo will be added to half of the patients in the study.
A Phase 2 trial for sickle cell disease and an investigator-sponsored trial for pulmonary arterial hypertension are both expected to begin in the second quarter of 2007.
Update on Phenylase Program
Preclinical data demonstrates that Phenylase administered subcutaneously once weekly in PKU mice resulted in a sustained decrease in blood Phe to levels within a normal range for a 12-week period. BioMarin plans to file an IND by the end of 2007 and to be in the clinic in early 2008.
Anticipated Upcoming Milestones
* Q207 -- NDA filing for Kuvan
* Q207 -- Initiation of Phase 2 Trial of 6R-BH4 in sickle cell disease
* Q207 -- Initiation of investigator-sponsored trial of 6R-BH4 in
pulmonary arterial hypertension
* June 2007 -- Initiation of Expanded Access Program for Kuvan for
approximately 500 PKU patients in the U.S.
* Q307 -- MAA filing for Kuvan by Merck Serono
* Q407 -- IND filing for Phenylase
Upcoming Investor Conferences
* May 8-10: Baird Growth Stock Conference -- Chicago
* May 14-15: Rodman & Renshaw 4th Annual Global Healthcare Conference --
Monte Carlo
* May 22-24: Citigroup Healthcare Conference -- New York City
* May 30-June 1: Bank of America Healthcare Conference -- Las Vegas
BioMarin will host a conference call and webcast to discuss first quarter 2007 financial results tomorrow, Tuesday, May 1, at 12:00 p.m. ET (18:00 CET). This event can be accessed on the investor section of the BioMarin website at www.BMRN.com.
Date: May 1, 2007
Time: 12:00 p.m. ET (18:00 CET)
U.S. & Canada Toll-free Dial in #: 800.261.3417
International Dial in #: 617.614.3673
Participant Code: 47185129
Replay Toll-free Dial in #: 888.286.8010
Replay International Dial in #: 617.801.6888
Replay Code: 96143665
About BioMarin
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio is comprised of two approved products and multiple clinical and preclinical product candidates. Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin, and Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation. Investigational product candidates include Kuvan(TM) (sapropterin dihydrochloride), a Phase 3 product candidate for the treatment of phenylketonuria (PKU), and 6R-BH4 for cardiovascular indications, which is currently in Phase 2 clinical development for the treatment of peripheral arterial disease. For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.
Forward-Looking Statement
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: the sales expectations of BioMarin's product Naglazyme and BioMarin/Genzyme LLC's product Aldurazyme; Alliant Pharmaceuticals' commercialization of Orapred ODT; the financial performance of the BioMarin as a whole; the timing of BioMarin's clinical trials of 6R-BH4 for other indications; the continued clinical development and commercialization of Aldurazyme, Naglazyme, Kuvan, and 6R-BH4 for other indications; actions by regulatory authorities, including actions related to Kuvan, and 6R-BH4 for other indications; and expectations regarding actions by Merck Serono related to filing the marketing authorization application for Kuvan. These forward- looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: our success in continuing commercialization of Naglazyme; our joint venture partner's success in continuing the commercialization of Aldurazyme; Alliant Pharmaceuticals' success in commercializing Orapred ODT; results and timing of current and planned preclinical studies and clinical trials; our ability to successfully manufacture our products and product candidates; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities concerning each of the described products and product candidates; the market for each of these products and particularly Aldurazyme, Naglazyme and Orapred ODT; actual sales of Aldurazyme, Naglazyme and Orapred ODT; Merck Serono's activities related to Kuvan; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's 2006 Annual Report on Form 10-K, as amended, and the factors contained in BioMarin's reports on Form 10-Q and Form 8-K. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.
NOTE: BioMarin® and Naglazyme® are a registered trademarks of BioMarin Pharmaceutical Inc.
Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.
Orapred® is a registered trademark of Medicis Pediatrics, Inc. and is
used under license.
Contact:
Investors Media
Eugenia Shen Susan Berg
BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc.
(415) 506-6570 (415) 506-6594
BioMarin Pharmaceutical Inc. and BioMarin/Genzyme LLC
Net Product Sales of Aldurazyme and Naglazyme
For the Three Months Ended March 31, 2006 and 2007
(In millions, unaudited)
Three Months Ended
March 31,
2006 2007
Aldurazyme (as reported by BioMarin/Genzyme LLC) (1) $21.3 $26.8
Naglazyme 7.0 18.4
(1) The company recognizes its 50% share of the net income of BioMarin/Genzyme LLC as Equity in the income of BioMarin/Genzyme LLC in the company's consolidated statements of operations.
BIOMARIN PHARMACEUTICAL INC. AND SUBSIDIARIES
CONSOLIDATED STATEMENTS OF OPERATIONS
For the Three Months Ended March 31, 2006 and 2007
(In thousands, except for per share data, unaudited)
Three Months Ended
March 31,
2006 2007
Revenues:
Net product sales $8,979 18,334
Collaborative agreement revenues 4,514 4,147
Royalty and license revenues 319 357
Total revenues 13,812 22,838
Operating expenses:
Cost of sales (excludes amortization of
developed product technology) 1,722 4,117
Research and development 12,279 18,159
Selling, general and administrative 10,893 16,284
Amortization of acquired intangible assets 373 1,093
Total operating expenses 25,267 39,653
Loss from operations (11,455) (16,815)
Equity in the income of BioMarin/Genzyme LLC 3,800 6,163
Interest income 699 3,694
Interest expense (2,824) (2,335)
Net loss $(9,780) (9,293)
Net loss per share, basic and diluted $(0.13) (0.10)
Weighted average common shares outstanding,
basic and diluted 74,963 94,557
BIOMARIN PHARMACEUTICAL INC. AND SUBSIDIARIES
CONSOLIDATED BALANCE SHEETS
December 31, 2006 and March 31, 2007
(In thousands, except for share and per share data)
December 31, March 31,
2006 2007
(unaudited)
ASSETS
Current assets:
Cash and cash equivalents $89,162 $98,331
Short-term investments 199,685 174,976
Accounts receivable, net 14,670 14,525
Advances to BioMarin/Genzyme LLC 1,596 814
Inventory 25,075 27,438
Other current assets 4,036 4,561
Total current assets 334,224 320,645
Investment in BioMarin/Genzyme LLC 31,457 31,619
Property, plant and equipment, net 55,466 56,487
Acquired intangible assets, net 11,655 10,563
Goodwill 21,262 21,262
Restricted cash 1,731 2,617
Other assets 7,641 6,571
Total assets $463,436 $449,764
LIABILITIES AND STOCKHOLDERS' EQUITY
Current liabilities:
Accounts payable and accrued liabilities $32,166 $24,889
Current portion of acquisition obligation,
net of discount 6,787 6,786
Current portion of deferred revenue 7,092 7,154
Total current liabilities 46,045 38,829
Convertible debt 223,940 172,500
Long-term portion of acquisition obligation,
net of discount 68,548 67,945
Deferred revenue, net of current portion 5,023 3,297
Other long-term liabilities 2,078 2,479
Total liabilities 345,634 285,050
Stockholders' equity:
Common stock, $0.001 par value: 150,000,000
shares authorized; 91,725,528 and 95,607,422
shares issued and outstanding at December 31,
2006 and March 31, 2007, respectively 92 96
Additional paid-in capital 709,359 765,540
Accumulated other comprehensive loss (25) (5)
Accumulated deficit (591,624) (600,917)
Total stockholders' equity 117,802 164,714
Total liabilities and stockholders' equity $463,436 $449,764
Source: BioMarin Pharmaceutical Inc.
You know, this is a really good point. Quarterly numbers are very important here, compared to year over year. Recent events have led me to be pretty nervous with BMRN, but in the long run I really dont see any problems. Short term, I could see us going lower especially if the conference call does not show a bright side of things. Well, actually, long term there are some problems as well... mainly a very limited pipeline.
My two main questions are: what is the cash for? what happened to sickle cell trial?
Hopefully the answers to both of these relieve my short and long term worries. I would definitely like to see the $20's again soon.
Citadel LP Raises Stake in BioMarin (BMRN) to 5.52%
04-26-2007 11:27:02 AM
Citadel LP disclosed a 5.52% stake (5.575 million shares) in Biomarin. This is up from the 43,657 share stake the firm held at the quarter ended 12/31/06.
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio is comprised of two approved products and multiple clinical and preclinical product candidates.
Started by market-guru Kenneth Griffin in his Harvard dorm room in 1987, Citadel has become one of the world's largest hedge funds.
Cramera says:
BioMarin Pharmaceutical
"I like [it]. I know people were disappointed with that last quarter. I think it's a mistake. BioMarin's a good biotech stock - and a takeover target."
Five Firms Big Pharma Should Buy Next
Wednesday April 25, 7:00 am ET
By Karen Andersen
Pharmaceutical companies still see riches in biotechnology. AstraZeneca's (NYSE:AZN - News) $15.6 billion deal to acquire MedImmune (NasdaqGS:MEDI - News) is only the latest example of their continuing willingness to pay high premiums (in this case, a 53% premium to where MedImmune was trading before it put itself up for sale) for future growth.
At Morningstar, we've seen various themes to these drug stock take-outs over the past year. In some cases, the larger firm in a collaboration takes out the smaller partner, in an effort to claim full rights to future profits; Amgen's (NasdaqGS:AMGN - News) acquisition of antibody expert Abgenix and Lilly's (NYSE:LLY - News) acquisition of Cialis-driven ICOS are both good examples of this behavior. Sometimes a firm looks to expand into new therapeutic areas, such as when HIV expert Gilead (NasdaqGS:GILD - News) bought cardiopulmonary firm Myogen, Merck (NYSE:MRK - News) bought Sirna Therapeutics for its innovative new RNA interference technology, or when Abbott (NYSE:ABT - News) bought Kos Pharmaceuticals for its cholesterol drug pipeline. In other cases, firms attempt to ward off slowing growth by injecting new products into established businesses, like Genzyme's (NasdaqGS:GENZ - News) purchase of AnorMED to supplement a sluggish transplant segment, or Shire's (Other OTC:SHPGF.PK - News) purchase of New River to fend off generic competition to its attention deficit franchise.
Given the current interest in scooping up biotechs at healthy prices, we've decided to use clues from past acquisitions to provide a list of firms that represent potential future targets. To start, we've created a screen that filters out any biotechs with market capitalizations of less than $250 million (firms that tend to be extremely volatile and speculative) and greater than $15 billion (firms that are difficult for even large pharmaceutical companies to afford). We've also screened for biotechs that we don't think are overvalued by the market--that is, firms that have received a Morningstar Rating of at least 3 stars. We were left with 26 firms, which we've qualitatively culled into a list of what we see as the five most likely future targets, as shown in the table below.
To see the related table, click here:
http://news.morningstar.com/article/article.asp?id=190410
As we discussed in our most recent quarterly review of the health-care sector, we don't typically factor acquisition speculation into our fair value estimates, but instead focus on what we think the firm's products and pipeline are really worth in the long run. Below, we've highlighted why we think these five firms could create value for prospective buyers, including excerpts from company reports.
...
BioMarin Pharmaceutical (NasdaqGM:BMRN - News)
BioMarin's rare disease expertise has earned it a narrow economic moat, and strong prospects for current products and its late-stage pipeline could make it a desirable target for partner Genzyme or other firms looking to gain exposure to this niche. While share prices fell in February due to poor hypertension data for lead pipeline candidate Phenoptin, we still think the drug has excellent prospects to treat the rare disease phenylketonuria, and it could be on the market by the end of the year. As I mention in my analysis, "BioMarin should enjoy steady sales growth from its innovative, life-saving treatments, and we expect it is just a matter of time before this biotech turns a profit."
Quick note:
I highly recommend everyone listen to this one Wednesday. Hopefully we hear some good things. Particularly I am very curious about the new debt, the unannounced sickle cell trials, the new insight on the failed BR3 trial, Kuvan progress, and of course financial for Q1. Honestly I have been a bit nervous lately due to some of the strange events taking place lately, but hopefully this webcast will clear things up.
BioMarin to Host First Quarter 2007 Financial Results Conference Call and Webcast on Wednesday, May 2 at 5:00 p.m. ET (23:00 CET)
BioMarin Pharmaceutical Inc. today announced that Jean-Jacques Bienaime, Chief Executive Officer of BioMarin, will host a conference call and webcast on Wednesday, May 2, at 5:00 p.m. ET (23:00 CET) to discuss first quarter 2007 financial results.
What: BioMarin First Quarter 2007 Financial Results Conference Call and
Webcast
When: Wednesday, May 2, 2007 at 5:00 p.m. ET (23:00 CET)
U.S. / Canada Dial-in Number: 800.261.3417
International Dial-in Number: 617.614.3673
Participant Code: 47185129
Replay Dial-in Number: 888.286.8010
Replay International Dial-in Number: 617.801.6888
Replay Code: 96143665
Interested parties may access a live audio webcast of the conference call via the investor section of the BioMarin website, www.BMRN.com. A replay of the call will be archived on the site for at least one week following the call.
BioMarin Announces Closing of $324,875,000 Public Offering of Senior Subordinated Convertible Notes
Monday April 23, 4:00 pm ET
NOVATO, Calif.
BioMarin Pharmaceutical Inc. announced today that it closed the sale of $324,875,000 aggregate principal amount of its 1.875% Senior Subordinated Convertible Notes due 2017 (including $42,375,000 aggregate principal amount of Notes purchased by the underwriter pursuant to its over-allotment option).
The maturity date of the Notes is April 23, 2017. Holders of the Notes may convert, at any time before maturity, any outstanding Notes into shares of the Company's common stock at an initial conversion rate of 49.1171 shares per $1,000 principal amount of the Notes, which represents a conversion price of approximately $20.36 per share, subject to adjustment under certain circumstances.
The Company has received approximately $316.4 million from the sale of the Notes after deducting the underwriting discount and estimated offering expenses. The Company intends to use the net proceeds of the offering to fund future business development transactions and for general corporate purposes, including working capital. These business development transactions may include acquisitions or licenses of complementary businesses, technologies or products. The Company has no material agreements or commitments with respect to any such acquisitions, license or investment and reserves the right, at the sole discretion of its board of directors, to reallocate the use of proceeds in response to these and other factors. Until the net proceeds are used, the Company intends to invest the funds in investment-grade, interest-bearing securities.
Merrill Lynch & Co. acted as sole book-running manager of the public offering. A shelf registration statement relating to these securities has been filed with the Securities and Exchange Commission and has become effective. This communication shall not constitute an offer to sell or the solicitation of an offer to buy nor shall there be any sale of these securities in any state in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.