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Dew, Isn't it possible that as mentioned earlier on the board that FDA approval will provide the collateral to allow a loan to pay back LFB and avoid the dilution altogether? It gives management 8 months to cook something up that may be to our benefit. I'm sure you have looked at cell genesis recently. They are down the tubes but have lots of money. Go figure. Like Dr. Cox said the inherent risk in a venture like GTCB is much less than that associated with new drug developement. Why is that so so so difficult to see for potential partners? bp
Or what about about they DO repay the loan. They have eight months to raise the cash. It can only come from a few places, a loan ala GE, a new partner, not likely unless there is a new program for a mono clonal which seems doubtful, there is already too much for them to handle or a public stock offering, which seems most likely. All eggs are in a basket called FDA approval. I think Cox is banking on this to raise stock profile to >.50 which will result in a new stock offering to pay back LFB, but then there is the issue of operating expenses. Any sense for what Ovation phase III might bring in? Time lines are so slow I just wish they would tell us what the plan is, its pretty shoestring! bp
If they award themselves cheap future options lets hope we stay in buisness long enough for them to collect. Maybe they are optomistic! bp
Dew, without reading all the fine print, what happens to all those "out of the money" stock options for management? Thanks, bp
Johnbits, unfortunately it does seem that the "hints" and inferences that a significant partnership was being worked on is probably smoke and we have a nearly worthless stock but not a nearly worthless company. That is, the basic premises and programs could be fabulous but are now a dollar short and a day late. And we are stuck. They still have eight months and some very high profile events to go. But as my grandfather famously said, "no money?, no money. " bp
I think you are on to something. Maybe we would have better luck with transgenic mole milk. bp
The loan hasn't been converted to stock (yet) so its still a loan isn't it? It is collateralized by IP so if it is not payed back in June it would seen the company rules would have to to change to allow for >20% ownership. We are becoming diluted to milimolar quantities. bp
Just went back and reviewed Dew's post. It addresses just about everything. We wait for approval. Not much will happen before then. bp
So here is what I think we know so far:
1. Antipicated income from LFB re Atryn, CD 20 program, AAT, FX 7 and 9 (each apparently good and active programs) is not enough alone to keep us afloat or we would not have had the recent "mortgage".
2. Short term-- partnership with Japan--unlikely
3. Short term-- partnership for CD 137 unlikely
4. Atryn not selling at the premium prices set by LEO and will be repriced by LFB but no substantial income to GTC as yet
5. No income for product for ongoing Phase II DIC study since it is on hold.
6. No resolution of 2.8 million for product rendered unusable by US contractor--forgetaboutit
7. Pharmathene income active but not "substantial" (not enough to keep us afloat that is.)
8. Milestones for Atryn approval in US fairly likely but potential income a fraction of yearly operating expenses and no substantial market in the US even for HD once approved (at least not enough to keep us afloat).
9. If we go under LFB owns the IP (and we get...?)
10. If we don't go under, OK now here's the part I am trying to understand better, how is it possible we don't go under? No one will buy us, not even LFB (we have to get a little bit cheaper first and then they own us anyway.)
11. Anything else to add to the list please feel free
12. So the question before the house...even with a high profile approval of Atryn (which you can't eat), unless there is sudden demand for off label indications and unanticipated income, all I can see is a market offering, banking on increased price share due to approval of first tg drug in US (OK, now that has to be worth SOMETHNG!) Anybody see it differently?
Regards, bp
Comon guys we are talking about an esoteric area of anticoagulation that none of the three of us has DIRECT experience with; although I have in my intern/residency days tried treating DIC with heparin resulting, well nothing. That was 30 years ago and look how much progress we (haven't) made. We are all students, MDs or not and have much to learn about this area. Studies need to go forward that can teach us. I don't know if FFP/heparin helps pump brain but I'd sure like to know if ATIII does...Peace to you both in this moment of impoverishment. There will always be wine. bp
Jesse, come on, not really... Heparin is worthless alone in DIC, heparin resistance etc. When there is no AT III, it can't and doesn't work. And heparin alone certainly doesn't work in HD> bp
Dew, Agreed. I will ask Tom how many countries have approved in EU. I assume LEO has access to intrim data from DIC study and unless recruitment was so low as to provide little data I can only assume they were not impressed with the results of whatever was happening on the DIC front. Weren't AAT and at least one of the other coag factors supposed to be in early clinicals by 09 end? Since GTCB did not allow outright buy by LFB whom I assume are ardent in their persuit, they still seem to be holding out for something. This all gives new meaning to giving away the farm. I drink tonight. bp
OK, OK, my mail box has been flooded with corrections. Goats really are kosher. bp
No it did not work out differently. My understanding is that the option to "buy back" any of the market temporarily given up as collateral for support exists. Am I wrong about this? bp
Why is a malaria grant from Gates/Buffett out of consideration? A few studies in the not too distant past continued to look at vaccine made from antigen made from mouse milk. We have heard little or nothing about this lately. The UN has made a commitment to deal with malaria and so has Gates et al. I still think the goat thing should appeal to a mind like his. If not its Japan or CD 136 but it ain't nothing. I think we are in for an exciting Monday. No way are we in for bad news with potential approval just over the horizon. Ham is traif and so are goats. But goat milk, now thats kosher. Regards, bp
Thanks to go seek and nivasvs, somewhere I thought I read a Nov date for this meeting which seems to be incorrect. Thanks for the additional info. I will (won't we all) watch for it. As to Beerzoid, I think you are quite wrong. Regards, bp
Can anyone verify the date of the expert FDA panel a the web site (or TV) for real time observation of the procedings? I think the future bodes well for 1. An Obama administration that will approach medical cost containment by innovation and support new technologies that do this. 2. A favorable view of Atryn as a very pure drug that does away with the standard risks associated with plasma derived drugs. 3. A public relations campaign that has been paid for in the past by GTCB dollars that could not be brought to bear because of prior dissapointments in the US market. It is waiting to burst free. 4. Pressure for "off label" use in sepsis especially if the phase II in Europe shows no worsening of an already terrible prognosis. And a question. Since we are dealing with a drug and an unmet need. If the experts recommend, what are the chances of an FDA ruling considerably before the Feb 7th date? Regards, bp
Thanks Dew, This would make for very interesting watching, maybe you could arrange for live streaming on ihub? Just kidding. What I was trying to get at was even if the discussions are public would the vote of the panel be public? Would it take place at the conclusion of the hearings? Although exceptions may occur, would a vote to support approval be almost tantamount to approval? Also, (lots of questions here), would the FDA stay composed similiarly to the way it is today come a new administration? Regards, bp
When the expert panel meets in November to make a recommendation to the FDA, is that recommendation made public (that is, before the final FDA February decision date)? bp
Protexia Phase I underway. bp
Thanks Dew, I think this bodes well for some flexibility in pursuit of a policy that does not require an "exact" duplicate of a given drug. bp
Thanks Lewis.
Just how different structurally is Atryn from the naturally occurring human protein? bp
Well, at least we'll know the outcome eventually. bp
Wonder if the current FDA look at Genzyme myozyme for Pompe's disease will give us some insight into their view of biosimiliars. Genz seems to be doing some preemptive damage control.
Press Release Source: Genzyme Corporation
FDA Advisory Panel to Discuss Genzyme's Myozyme BLA on Tuesday
Friday October 17, 1:57 pm ET
Meeting Will Focus on Clinical Data Supporting 2000 L Process
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Genzyme Corporation (Nasdaq: GENZ - News) today announced that its U.S. Biologic License Application (BLA) for Myozyme® (alglucosidase alfa) produced at the 2000 L bioreactor scale will be discussed this Tuesday, October 21st, at a public meeting of the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee in Silver Spring, Maryland. This product is intended for the treatment of Pompe disease, a progressive, debilitating and life-threatening inherited disorder affecting approximately 2,000 people in the United States. The meeting will focus on the clinical outcomes of the Late Onset Treatment Study (LOTS), including statistical analyses of the study results, safety and the indication for alglucosidase alfa.
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Genzyme’s LOTS study was a randomized, double-blind, placebo-controlled clinical trial that enrolled 90 patients. The co-primary efficacy endpoints were the Six-Minute Walk Test (6MWT) and percent-predicted Forced Vital Capacity (FVC). These co-primary endpoints were prospectively agreed upon with the FDA and are considered the most clinically relevant measures of walking ability and pulmonary function, respectively, for patients with late-onset Pompe disease. The co-primary endpoints achieved statistical significance according to the statistical analysis plan for the primary efficacy endpoints.
The results showed that, at 18 months, patients treated with alglucosidase alfa increased their distance walked in six minutes by an average of 28 meters, as compared with the placebo group, which did not show any improvement from baseline (ANCOVA; p<0.035). Percent-predicted FVC, the co-primary endpoint, increased in the treated group by one percentage point at 18 months. In contrast, FVC declined by approximately three percentage points in the placebo group over the same period (ANCOVA; p<0.0055). The results for both efficacy endpoints were consistent across various prospectively defined subgroups.
Earlier this week, Genzyme learned that the FDA will use a sensitivity analysis as the primary analysis for the 6MWT for discussion at the panel meeting. Genzyme believes that the application of this sensitivity analysis is not appropriate for this trial. Genzyme believes that the pre-specified primary analysis demonstrates the efficacy of the 2000 L product.
Additionally, the LOTS safety data demonstrate that alglucosidase alfa produced at the 2000 L scale has an acceptable safety profile in late-onset patients. The number of patients with serious and treatment-emergent non-serious adverse events was similar in the alglucosidase alfa and placebo groups. Twenty-eight percent of patients in the treatment group compared to 23 percent in the placebo group experienced infusion-associated reactions. Allergic symptoms were more frequent during infusion-associated reactions in the treatment group. Similar to the 160 L experience, five percent of patients experienced anaphylaxis and two thirds of these were able to continue treatment. There was one death in the Myozyme group unrelated to treatment.
“Genzyme believes that the LOTS safety and efficacy data, in combination with additional clinical and post-marketing safety data, support a full approval of alglucosidase alfa produced at the 2000 L scale for patients with late-onset Pompe disease,” said Genzyme Senior Vice President Alison Lawton. “We look forward to a productive discussion of these data at the advisory committee to facilitate a successful first-cycle approval for the 2000 L process and thereby assure a continued supply of alglucosidase alfa, the only treatment for patients with Pompe disease.”
Genzyme currently has U.S. approval to sell Myozyme, manufactured at the 160 L scale, and the company has been seeking clearance from the FDA for 2000 L-scale production. Genzyme submitted a separate BLA for alglucosidase alfa produced by the 2000 L manufacturing process on May 30th, following a determination by the FDA that alglucosidase alfa produced at the 160 L and 2000 L scales should be considered as two separate products because of comparability differences.
Genzyme is proposing the following indication for the product produced at the 2000 L scale: “Alglucosidase alfa is indicated for long-term use in patients with late-onset Pompe disease (GAA deficiency). Alglucosidase alfa has been shown to improve distance walked and stabilize pulmonary function in patients with late-onset Pompe disease.” Late-onset patients are currently defined for the clinical studies as patients who develop clinical manifestations of the disease after two years of age, and who have no cardiac involvement.
Formal FDA action is expected on Genzyme’s BLA by November 29, 2008. Genzyme anticipates that the FDA review process will culminate in the availability of two commercial versions of alglucosidase alfa in the United States: one produced at the 160 L scale and the other produced at the 2000 L scale. Production of Myozyme at the 2000 L scale has been approved for the use in all patients with Pompe disease in more than 40 countries. Approximately 1000 patients worldwide are on treatment.
To ensure that severely affected adults with Pompe disease in the United States have access to treatment, Genzyme, in collaboration with the FDA, created the Myozyme Temporary Access Program (MTAP) in May 2007. Through this program, the company is currently providing Myozyme produced at the 2000 L scale free of charge to approximately 160 patients. U.S. demand for alglucosidase alfa has now increased to the extent that it is no longer feasible for Genzyme to supply product to additional adult patients under the MTAP program. FDA approval of 2000 L production scale is needed to provide broader access to treatment for adult patients in the United States. There are approximately 50 – 100 patients identified who are waiting for access to treatment.
FDA and Genzyme briefing documents for Tuesday’s advisory committee meeting are available on the FDA’s Website.
Prior to the public advisory committee meeting, Genzyme and the FDA will meet with the advisory panel members in closed session to discuss proprietary manufacturing information. During this morning session, the discussion will focus around the biochemical characteristics of alglucosidase alfa produced at the 2000 L scale and its comparability to product produced at the 160 L scale.
About Pompe Disease
Pompe disease manifests as a broad spectrum of clinical symptoms. All patients typically experience progressive muscle weakness and breathing difficulty, but the rate of disease progression can vary widely depending on the age of onset and the extent of organ involvement. When symptoms appear within a few months of birth, babies frequently display a markedly enlarged heart and die within the first year of life. When symptoms appear during childhood, adolescence or adulthood, patients may experience steadily progressive debilitation and premature mortality due to respiratory failure. They often require mechanical ventilation to assist with breathing and wheelchairs to assist with mobility.
About Myozyme
Myozyme (alglucosidase alfa) is indicated for use in patients with Pompe disease (GAA deficiency). The U.S. product label includes a boxed warning with information on the potential risk of hypersensitivity reaction. Life-threatening anaphylactic reactions, including anaphylactic shock, have been observed in patients during Myozyme infusion. Because of the potential for severe infusion reactions, appropriate medical support measures should be readily available when Myozyme is administered. Full prescribing information for the product, including a complete list of the most common adverse reactions, is available on myozyme.com.
About Genzyme
One of the world's leading biotechnology companies, Genzyme is dedicated to making a major positive impact on the lives of people with serious diseases. Since 1981, the company has grown from a small start-up to a diversified enterprise with more than 10,000 employees in locations spanning the globe and 2007 revenues of $3.8 billion. In 2007, Genzyme was chosen to receive the National Medal of Technology, the highest honor awarded by the President of the United States for technological innovation.
With many established products and services helping patients in nearly 90 countries, Genzyme is a leader in the effort to develop and apply the most advanced technologies in the life sciences. The company's products and services are focused on rare inherited disorders, kidney disease, orthopaedics, cancer, transplant and immune disease, and diagnostic testing. Genzyme's commitment to innovation continues today with a substantial development program focused on these fields, as well as cardiovascular disease, neurodegenerative diseases, and other areas of unmet medical need.
Genzyme’s press releases and other company information are available at www.genzyme.com and by calling Genzyme’s investor information line at 1-800-905-4369 within the United States or 1-678-999-4572 outside the United States.
This press release contains forward-looking statements regarding Genzyme’s business plans and strategies, including without limitation: its expectations regarding receipt of FDA approval of the alglucosidase alpha 2000 L process BLA and the timing thereof; its expectations regarding the content of the discussions at the Advisory Committee meeting, including that no vote on whether to recommend approval of the BLA will be taken; and its expectation that both the 160 L scale and 2000 L scale materials will be commercially available in the US. These statements are subject to risks and uncertainties that could cause actual results to differ materially from those forecasted. These risks and uncertainties include, among others: Genzyme's ability to actually obtain FDA approval of the alglucosidase alpha 2000 L process in November 2008; that the Advisory Committee meeting is not positive or focuses on content different than what the Company anticipates; and the risks and uncertainties described in Genzyme's SEC reports filed under the Securities Exchange Act of 1934, including the factors discussed under the caption "Risk Factors" in Genzyme's Quarterly Report on Form 10Q for the quarter ended June 30, 2008. Genzyme cautions investors not to place substantial reliance on the forward-looking statements contained in this press release. These statements speak only as of today’s date and Genzyme undertakes no obligation to update or revise the statements.
Genzyme® and Myozyme® are registered trademarks of Genzyme or its subsidiaries. All rights reserved.
Contact:
Genzyme Corporation
Media Contact:
Lori Gorski, 617-768-9344
lori.gorski@genzyme.com
or
Investor Contact:
Patrick Flanigan, 617-768-6563
Dew, do you think this will have impact on the uvitis trial? Very dissapointing re RA. Local rheumatologist LOVED it in his patients. bp
Thanks Dew, I was hoping that since this was laboratory based that alternative sources might be used to address cost/availability issues. Regards, bp
Anyway to ascertain the source for the AAT? Human plasma derived, transgenic? Thanks, bp
Hmmm. Waltham is right next door to the farm. The hidden hand of GTCB PR?? bp
Doo (same pronounciation, different spelling), If these words are yours and are original I congratulate you for an excellent definition of group think. If these thoughts were conceptualized by someone else, I did not see an acknowlegement. Surely the irony regarding those remain interested in this stock and continue to post and share ideas (and perhaps offer support during a turbulent time) on this board can not be lost on you. The "group" has long departed, selling their shares and markedly depressing the share price. Those who remain interested in this company and its future are a distinct minority, but if you insist, still a "group". Most of the posters seem pretty independent minded and not reluctant to criticize, or as I perfer to think of it, analyze critically. Many as am I, are fascinated by the potential of this technology and the promise for medical treatments as yet unrealized. Many look for opportunity during a difficult time and are looking for specific signs as to whether the company may or may not be successful. I acknowlege that money is tight but see plenty of reason for existing and hopefully new partners to keep the goats afloat. If you think any of the points I made are untrue so say, but please without the suggestion that the observations were made by a mindless unquestioning automaton. Regards, bp
Fellow goatherders, As we all try to deal with the current oppressive psychological atmosphere together there are some things worth remembering. We have not been led to believe by management we are in danger of going under any time soon, survival through February FDA approval process, sucessful one hopes, is apparent as is survival through the presidential election which will help when people realize cost efficiency for medication production matters putting pressure on FOB legislation. Presentations have been scheduled months into the future and as far as anyone can tell operations continue as they have. Jesse's covert ops survellience of the parking lot shows normal daily activity. No employee gtcb malcontents have shown up on this board. WE HAVE PASSIONATE LEADERSHIP. Cox and Meade are not bored and I never have the sense they are doing it just for the money. They are committed and working for us. People have come through tough times before and new technologies and ideas have been pissed on until someone recognizes their value. OldBerkley, pass the water, it sure is dry out here in the Biotech Dessert. regards, bp
I agree. Likelihood very high a panel of experts will be called upon to opine re the safety and purity of drugs made with this the new technology. It sets a standard and may allow future applications to move faster once the technology is vetted and endorsed. bp
FYI Revaroxaban (oral anticoagulant) approved in Europe (Bayer). bp
Got it. Thank you. bp
Dew, But he has bought other shares (not the big stuff representing LFB) but smaller but substantial amounts that I presume are for his personal account. I have interpreted this as a continued vote of confidence on his part. Anyway when a non open market acquisition is made is the stock just valued at the market on a particular date? Thanks, bp
When someone like Christian Bechon buys "non open market" what does that mean? Thanks, bp
Recently announced is funding for malaria eradication by the UN 168 million some from the Gates foundation for vaccine research. bp
It appears they divested themselves of any plasma derived products they used to have also. Their web site is not too impressive, nor is their product line but don't miss the stuff re "the NABI way"...let the chanting begin. bp
Jesse, Wow, I guess I never took the discussions you suggested I review seriously until now nor did I think much about the Cox NABI connection. Interesting. It does look like NABI doesn't have much going for it with its intrinsic pipeline. And in spite of that they are buying back shares. Did I read that they have a 40 mil loan outstanding? Anyway I'll go back and check. Thanks, bp
Isn't there a loan in there someplace too?