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Apr-26-2008US Asks for Cholesterol Drug Data(topic overview)
CONTENTS:
- Earnings excluding items are still projected to rise to about $US7.00 per share by 2011, the company said. (More...)
- NEW YORK -- Genzyme Corp reported a slightly better-than-expected first-quarter profit on strong demand for its drugs to treat rare diseases, and its shares rose more than 2 per cent. (More...)
- The setback reduced Genzyme's 2008 non-GAAP earnings guidance to approximately $3.90 a share. (More...)
- The firm's adjusted bottom-line figure still came in above the amount predicted by analysts, bolstered by a rise in revenues. (More...)
- For broader, high-risk populations, the U.S. regulatory agency will require an outcomes study. (More...)
- "We are extremely disappointed in the FDA's decision because it will further delay broad patient access to Myozyme, which is not possible under the MTAP program," said Henri A. Termeer, Genzyme's chairman and chief executive officer. (More...)
- Despite Isis' dramatic drop, Genzyme only slipped 1.3%, or 98 cents, to $72.43. (More...)
- Carlsbad Calif. -based Isis (NASDAQ:ISIS) was down 28.6 percent, or $4.81, on the news, closing at $11.99 in Friday trading. (More...)
- S&P; analyst, S. Kessler, says, "BIDU posts Q1 per-ADS profit of $0.60 vs. $0.32, $0.08 below our forecast. (More...)
- The tech-heavy Nasdaq, suffering from disappointing earnings from Microsoft (nasdaq: MSFT - news - people ), sank 0.3%, or 5.99 points, to 2,422.93, although it still closed up 0.8% for the week. (More...)
- Because the Framingham plant has only limited capacity, it is currently supplying the Allston drug to 140 U.S. Pompe patients free. (More...)
- Mipomersen is an antisense drug that reduces the production of a protein critical to the synthesis and transport of "bad" cholesterol. (More...)
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Earnings excluding items are still projected to rise to about $US7.00 per share by 2011, the company said. Shorter-term however, Genzyme lowered its full-year earnings forecast, excluding items, by 10 cents a share to $US3.90 per share to reflect a delay in FDA approval of larger scale production capacity for its Pompe disease treatment Myozyme. The company cut its full-year Myozyme sales forecast to $US275 million to $US285 million from its prior view of $US320 million to $US330 million due to the regulatory delay. [1] Analysts surveyed by Thomson Financial expected profit of 93 cents per share on revenue of $1.09 billion. Looking ahead, Genzyme lowered its outlook for 2008 adjusted profit to about $3.90 per share from prior guidance of $4 per share, reflecting the delay in FDA approval of Pompe disease treatment Myozyme.[2]
Comparable year-over-year revenue rose 25%, and non-GAAP earnings per share gained a healthy 22%, at $0.95 for the quarter. Guidance took a hit from Monday's FDA rejection of Genzyme's marketing application to manufacture one of its lead drugs, enzyme replacement therapy Myozyme, at a second manufacturing plant. It seems the FDA was worried that the version of Myozyme produced at the second, much larger manufacturing plant wouldn't be sufficiently similar to the one produced at the already-approved plant. According to Genzyme, this delayed approval will cost the company $0.10 a share in earnings for the year, because of Myzozme's unexpectedly crimped supply.[3] The FDA's refusal is obviously bad news for Genzyme, but it also stings potential biosimilar drug manufacturers like Momenta Pharmaceuticals (Nasdaq: MNTA ) and Novartis (NYSE: NVS ). Genzyme hopes its second plant will get the FDA go-ahead by the end of the year. The company reiterated its long-term goal to grow non-GAAP EPS by an average of 20% annually through 2011, with an ultimate goal of $7 per share. Genzyme generally does a good job of detailing all the assumptions behind its near-term annual financial guidance, but after its unexpected Myozyme stumble, let's hope its longer-term outlook becomes a bit more conservative.[3]
Commenting on the FDA guidance, chairman and chief executive officer of Isis, Stan Crooke said, "Conducting an outcome study in parallel with our continued evaluation of the effects of mipomersen on atherogenic lipids will allow us to submit a much stronger NDA for high risk patients. We are confident that mipomersen will bring benefit to patients with high cholesterol and remain committed to its development and commercialization." Genzyme's chairman and chief executive officer, Henri Termeer added, 'We are pleased that the FDA has given clear direction on what will be required for the approval of mipomersen, and has acknowledged its potential to help high risk patients whose needs are not being met by current therapies." The companies said on February 1, that they have received clearance under the Hart-Scott-Rodino Antitrust Improvements Act for their collaboration, which was announced on January 7, 2008. Based on this approval, Genzyme will pay Isis $150 million to purchase five million shares of Isis common stock for $30 per share.[4] Cambridge, Mass. -based Genzyme (Nasdaq: GENZ) announced on Friday that the U.S. Food and Drug Administration has issued guidance on approval requirements for mipomersen. Those terms are crucial as the companies pursue a late-stage human clinical trial using the drug to treat patients with homozygous familiar hypercholesterolemia, a form of bad cholesterol that can create a high risk of premature cardiovascular disease. Genzyme and Isis (Nasdaq: ISIS) inked a strategic alliance earlier this year that calls for Genzyme to develop and commercialize the drug. Genzyme, in fiscal 2008 financial numbers announced this week, said it took an after-tax charge of $56.5 million relating to its strategic investment in Isis.[5]
The ripple effects from Merck and Schering-Plough's Vytorin problem has rocked Isis Pharmceutical's boat. Shares of the Carlsbad, Calif. -based biotech firm were knocked down 28.6%, or $4.81, to $11.99, on Friday, after the U.S. Food and Drug Administration asked Isis (nasdaq: ISIS - news - people ) for new test data regarding cancer for the cholesterol drug mipomersen it's developing with Genzyme (nasdaq: GENZ - news - people ). Such ailments have always been Genzyme's specialty.[6]
Genzyme GENZ and Isis Pharmaceuticals ISIS, which teamed up in a well-received deal in January, have encountered regulatory turbulence. The pair on Friday announced expected delays in approvals of their cholesterol-lowering product, news that sent shares of Isis down more than 20%. Food and Drug Administration requirements will result in a one-year delay in Mipomersen -- the cholesterol-lowering antisense drug at the heart of the Genzyme/Isis deal -- in its smaller and nearest-term indication and a multiyear delay for a broader, potentially more lucrative indication.[7] The Cambridge, Massachusetts-based biotechnology company posted a net profit of $US145.3 million, or 52 cents per share, compared with a profit of $US158.2 million, or 57 cents a share, a year ago. The net results include an after-tax charge of $US56.5 million related to Genzyme's deal with Isis Pharmaceuticals Inc on mipomersen, its promising experimental cholesterol treatment.[1] Excluding a charge of $56.5 million on Genzyme (nasdaq: GENZ - news - people )'s investment in Isis Pharmaceuticals (nasdaq: ISIS - news - people ), profit grew to 95 cents per share from 78 cents.[2]
The company's net profit fell to $145.3 million or $0.52 per share from the year-ago $158.2 million or $0.57 per share. The company said its recent quarter reflected a charge of $56.5 million related to its strategic investment in Isis Pharmaceuticals.[8]
The biotechnology company posted net income of $145.3 million, or 52 cents a share, down from $158.2 million, or 57 cents a share, a year earlier. Excluding items such as a $56.5 million charge in the latest quarter on Genzyme's investment in Isis Pharmaceuticals Inc. (ISIS), earnings rose to 95 cents a share from 78 cents a share.[9]
Annual sales of Myozyme are now expected to be about $275 million-$285 million, compared to prior projected range of $320 million-$330 million. Genzyme expects Thymoglobulin sales to accelerate in the second-half of this year, as supply levels increase to meet full demand for the product. The company said it continues to focus on its commitment to deliver 20% non-GAAP earnings growth through 2011, with non-GAAP earnings projected to grow to about $7.00 per share by 2011.[8] Sales of chronic kidney disease treatments Renagel and Renvela rose 23% to $168.7 million, edging past Wall Street's expectation for $166 million. Because of the delay in larger-scale Myozyme, the company is now predicting an adjusted profit of $3.90 a share for this year, compared with $4 a share previously expected, and GAAP earnings of $2.65 a share, also down 10 cents from its prior guidance.[10] Seventeen Wall Street analysts expected revenues of $1.09 billion. The company predicted non-GAAP earnings for 2008 of about $3.90 per share, compared to its previous forecast of $4.00 per share. This adjustment, which was previously announced, reflects the delay in FDA approval of 2000L production capacity for Myozyme.[11] The company already has a completed clinical trial -called LOTS - that may form the basis of a new BLA for the 2,000-litre version of Myozyme which should avoid too much of a delay in bringing it to market. It hopes to launch it in the first quarter of 2009, and will forgo an estimated $45m in lost revenues this financial year. At present there is only one copycat version of a biologic drug on the U.S. market - Sandoz' Omnitrope (somatropin) - despite years of wrangling about drawing up a regulatory route for approval of these so-called 'follow-on biologics'. The FDA has made no secret of its concerns about the risk of immunogenic reactions when swapping from an innovator biological product to a follow-on biological product (or vice versa), saying that this should only occur with a physician's consent. The two drugs must also not be considered interchangeable, said Department of Health and Human Services Secretary Michael Leavitt earlier this year when debate on the bipartisan U.S. Biosimilars Act (H.R. 5629) introduced in March.[12] Genzyme just launched Renvela in the U.S. in March and said the company is in active discussions with the Food and Drug Administration to expand the product's labeling to include chronic kidney disease patients with hyperphosphatemia who are not on dialysis. Myozyme, the company's Pompe disease treatment, saw sales surge 78% amid " robust" demand for the drug two years after its launch, driven by the number of new patients starting therapy.[9] In a decision that shows how difficult it is to copy complex, biologic drugs, federal regulators rejected Genzyme Corp.' s request for permission to sell in the United States a version of its Pompe disease drug, called Myozyme, that is made at its Allston manufacturing plant, the company disclosed yesterday afternoon. Though Genzyme already has permission to sell batches of Myozyme manufactured at its smaller Framingham plant, the Food and Drug Administration ruled that Myozyme made at the Allston plant should be considered a different product because of small differences in its chemical structure, Genzyme said.[13]
NEW YORK -- Genzyme Corp reported a slightly better-than-expected first-quarter profit on strong demand for its drugs to treat rare diseases, and its shares rose more than 2 per cent. Excluding items, such as a large charge related to a licensing deal, the biotechnology company earned 95 cents per share, topping analysts' average expectations by 2 cents per share, according to Reuters Estimates. "As they've been able to do, they put up a strong bottom line number and beat consensus," said Matthew Osborne, analyst for Lazard Capital Markets. [1] Wall Street has forecast second-quarter earnings per share of 96 cents, and full-year profit of $3.95 per share. Genzyme reaffirmed its commitment to 20 percent growth in adjusted profit through 2011, totaling about $7 per share.[2]
"We are disappointed by the decision because it will delay access to Myozyme in the United States," said Genzyme Corp. chief executive Henri Termeer in a conference call with analysts. Specifically, Genzyme believes the decision will reduce its revenue by $45 million this year and cut its earnings by 10 cents a share.[13] Looking forward, Genzyme confirmed the full-year earnings projection it cut Monday by a dime to about $3.90 a share, reflecting a delay in FDA approval of the 2000L dose of Myozyme.[9]
"Myozyme is a disappointment that we can't develop the U.S. market at the current time, but there is no doubt in any one of our minds. in our ability to get approval with the FDA," Mr Termeer told analysts. Lazard's Osborne said Myozyme will likely recover from its regulatory delay in the second quarter of 2009, "but demand is still strong." He also forecast significant growth in Genzyme's newer kidney drug, Renvela, once it gets a label expansion approval to include treatment of patients not yet on dialysis.[1] "Conducting an outcome study in parallel with our continued evaluation of the effects of mipomersen on atherogenic lipids will allow us to submit a much stronger NDA for high risk patients. We are confident that mipomersen will bring benefit to patients with high cholesterol and remain committed to its development and commercialization." "We are pleased that the FDA has given clear direction on what will be required for the approval of mipomersen, and has acknowledged its potential to help high risk patients whose needs are not being met by current therapies," said Henri A. Termeer, Genzyme's chairman and chief executive officer. "Having outcome data earlier on in the development process will be important to patients and serve to enhance the value of this treatment. We plan to engage in discussions with regulatory agencies in Europe and the rest of the world, and look forward to receiving their feedback." In early 2008, Isis and Genzyme announced that they had entered into a strategic alliance in which Genzyme will develop and commercialize mipomersen. Final contracts are still being negotiated and are expected to be completed this quarter.[14] NEW YORK -- Biotechnology companies Isis Pharmaceuticals Inc. and Genzyme Corp. said Friday approval of their cholesterol drug candidate mipomersen will face at least a one-year delay, now that the Food and Drug Administration has asked for more study data.[15] NEW YORK (Thomson Financial) - Isis Pharmaceuticals Inc. and Genzyme Corp. Friday said the Food and Drug Administration indicated that reduction of LDL cholesterol is an acceptable surrogate endpoint for accelerated approval of cholesterol treatment mipomersen for use in patients with homozygous familial hypercholesterolemia (hoFH).[16]
ISIS has been climbing for most of the past year. Isis Pharmaceuticals shares have been plunging on news that U.S. health regulators have asked for data from two preclinical carcinogenic studies of its cholesterol drug, Mipomersen, to be included in the final late-stage study results.[17] BANGALORE, April 25 (Reuters) - Isis Pharmaceuticals (ISIS.O: Quote, Profile, Research ) will delay submitting final results from a late-stage study of its cholesterol drug after U.S. health regulators asked for additional cancer-related data from two preclinical trials, sending its shares tumbling 30 percent.[18] The health-care sector wasn't immune to bad news. Isis Pharmaceuticals (nasdaq: ISIS - news - people ) announced it will delay submitting final results from a late-stage study of its cholesterol drug after U.S. health regulators asked for additional cancer-related data from two preclinical trials.[19]
Isis and co-developer Genzyme (nasdaq: GENZ - news - people ) expect to submit final data from the ongoing late-stage trial of the drug, mipomersen, in 2010.[19] Isis and co-developer Genzyme Corp (GENZ.O: Quote, Profile, Research ) expect to submit final data from the ongoing late-stage trial of the drug, mipomersen, in 2010.[18]
The data Genzyme provided to the FDA for approval showed differences in the carbohydrate component of the drug, which is involved in uptake of the enzyme into muscle tissue. The FDA considered these differences significant enough to consider the 2000-litre-batch material a new drug, requiring its own clinical trial data.[20] Genzyme has already completed a clinical trial using the larger batch material, so anticipates receiving approval by the end of 2008, following a priority review by the FDA. However, the decision has sparked speculation about the regulator's likely stand on generic copies of biologic drugs.[20]
Currently, Genzyme has U.S. approval to sell Myozyme manufactured at the 160L scale, and the company has been seeking clearance from the FDA for Myozyme produced at the 2000L scale. Production at this larger scale has already been approved in more than 40 countries. Based on the global clinical experience of nearly 900 patients of all ages currently receiving Myozyme produced at the larger scale, including patients who participated in the Late-Onset Treatment Study (LOTS), Genzyme believes that Myozyme produced at both the 160L and 2000L scales is clinically effective and safe.[21] 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 2000L scale free of charge to approximately 140 patients.[21] The agency proposed that Genzyme initiate a rolling BLA review process by submitting results from the LOTS study. Genzyme expects the FDA to give the BLA priority review and to act on the application by the end of this year. The LOTS study, which met its co-primary efficacy endpoints, was undertaken to evaluate the safety and efficacy of Myozyme in juvenile and adult patients with Pompe disease. Genzyme had already been preparing to submit results from this study to the FDA to fulfill a post-marketing commitment. Genzyme anticipates that this process will culminate in the availability of two commercial versions of Myozyme in the United States: one produced at the 160L scale and the other produced at the 2000L scale.[21]
The FDA has informed Cambridge, Mass. -base Genzyme Corporation that the company will be required to file two separate biologics license applications (BLA) for its two formulations of Myozyme, potentially delaying patient access to the first-ever approved treatment for Pompe disease, an inherited muscle disorder.[22]
The ruling will limit production of the Pompe disease treatment, leading Genzyme to cut projected 2008 sales from the drug to a range of $275 million to $285 million from $320 million to $330 million.[9] Revenue climbed 25 percent to $1.10 billion from $883.2 million a year ago, led by 15 percent growth in sales of Gaucher disease drug Cerezyme to $304.3 million and 23 percent growth in kidney treatments Renagel and Renvela to $168.7 million.[2] Sales of injected Gaucher disease drug Cerezyme rose 15% to $304 million and sales of Fabry disease treatment Fabrazyme increased 16% to $117 million year over year, vs. the consensus targets of $301 million and $118 million, respectively.[10]
Fabrazyme, which gained over two-thirds share of the overseas market for Fabry disease treatment, generated sales of $116.5 million, up 16% from $100.7 million a year ago.[8]
The company plans to file midyear for U.S. and European approval of Mozobil for stem-cell transplantation in lymphoma and multiple myeloma patients. It expects to launch the product in the United States early next year and forecast eventual peak annual sales of $400 million for the product.[1] Sales of Myozyme grew 78% to $67.3 million from $37.9 million last year, despite the delay in U.S. approval for 2000-liter production capacity.[8]
The delayed production of the larger-scale version will affect 2008 sales of the drug. Genzyme now expects Myozyme sales of roughly $275 million to $285 million in 2008, down from its previous estimate of $320 million to $330 million.[10] The target price has been reduced from $90 to $88. In a research note published yesterday, the analysts mention that the company has announced the requirement of a separate filing for Myozyme on account of marginal differences in the materials generated at its 2,000-litre facility. Genzyme has therefore reduced its 2008 Myozyme sales guidance from $320 million-$330 million to $275 million-$285 million and its EPS guidance from $4.00 to $3.90.[23]
Genzyme reported $145.3 million in net income during the quarter, compared to $158.2 million in net income during the fiscal 2007 first quarter. The company said the income dip reflects an after-tax charge of $56.5 million relating to its strategic investment in Isis. Genzyme also said it continues to use part of its operating cash flow in a three-year program designed to repurchase shares.[24] Genzyme reported quarterly profit of $145 million, or 52 cents a share, in the first quarter, vs. $158.2 million, or 57 cents a share, in the year-ago quarter.[10] On an adjusted basis, profit increased to $260 million, or 95 cents a share, from $210 million, or 78 cents a share, in the 2007 quarter.[10]
In the same period last year, the company had a profit of $210.7 million or $0.78 per share.[11] Net income dipped to $145.3 million, or 52 cents per share, from $158.2 million, or 57 cents per share, a year ago.[2] Genzyme reported first-quarter GAAP net income of $145.3 million or $0.52 per share, lower than $158.2 million or $0.57 per share in the prior-year quarter.[11] As per the deal, Genzyme was to pay Isis $150 million to purchase five million shares of Isis common stock for $30 per share upon Hart-Scott-Rodino clearance.[4]
Excluding certain items, the company's non-GAAP net income increased to $260.9 million or $0.95 per share.[11] Looking ahead, for the second quarter, the company said it expects non-GAAP earnings per share to be in the mid $0.90s.[8] On average, 16 analysts polled by First Call/Thomson Financial expected the company to report earnings of $0.93 per share.[11]
"We continue to focus on our commitment to deliver 20 percent non-GAAP earnings growth through 2011, while building the company to ensure that we sustain our growth over the longer term." Fellow biotech Biogen Idec BIIB also aired its earnings, beating by 4 cents a share on an adjusted basis. Sales of Biogen's MS drug Avonex and Biogen and Elan's ELN MS and Crohn's disease drug Tysabri both beat analyst views.[10] Biotech company Genzyme Corp. said Wednesday its first-quarter earnings slipped 8 percent as an investment charge offset strong sales growth, and revised its full-year outlook to reflect delayed approval of the biotech company's Myozyme drug.[2] Genzyme also said Wednesday that it expects second-quarter adjusted earnings to be in the mid-90-cent range, reflecting ongoing investments to drive future growth, as well as constraints on U.S. Myozyme sales.[9]
"We continue to focus on our commitment to deliver 20 percent non-GAAP earnings growth through 2011, while building the company to ensure that we sustain our growth over the longer term." Genzyme continues to make strong progress in advancing programs within its late-stage development pipeline. These programs have significant potential to drive the company's growth beyond 2011. These statements are subject to risks and uncertainties that could cause actual results to differ materially from those forecasted. These statements speak only as of today's date and Genzyme undertakes no obligation to update or revise the statements. Genzyme(R), Myozyme(R), Fabrazyme(R), Cerezyme(R), Thyrogen(R), Renagel(R), Renvela(R), Thymoglobulin(R), Synvisc(R), Campath(R) and Clolar(R) are registered trademarks of and Mozobil(TM) and Synvisc-ONE(TM) are unregistered trademarks of Genzyme or its subsidiaries.[25] Henri Termeer, Genzyme's chairman and chief executive officer, said in a press release, "We continue to focus on our commitment to deliver 20 percent non-GAAP earnings growth through 2011, while building the company to ensure that we sustain our growth over the longer term."[11]
CAMBRIDGE, Mass., April 23 /PRNewswire-FirstCall/ -- Genzyme Corporation (Nasdaq: GENZ) today reported results for the first quarter of 2008, which featured excellent revenue growth, continued operating leverage, a significant increase in non-GAAP profit, and strong progress across the company.[25] The biotech company beats on profit, but revenue falls shy of Wall Street views for the first quarter.[7]
Genzyme's first quarter revenue grew by 25 percent compared to the same period the prior year, from $883 million to $1.1 billion.[26] Cambridge-based biotechnology company Genzyme Corp. reported first quarter income of $145 million compared to $158 million for the same period last year.[26]
Since the program began a year ago, Genzyme has repurchased 4.5 million shares, including 1 million shares repurchased during the first quarter.[24]
The Street is looking for earnings of $0.96 a share for the next quarter. Genzyme said this forecast reflects ongoing investments, including the U.S. launch of Renvela and the expanded European introduction of Clolar.[8] For the second quarter, the company predicted non-GAAP earnings in the mid-$0.90 range. Genzyme said this forecast reflected ongoing investments, including those in the U.S. launch of Renvela and the expanded European introduction of Clolar.[11]
The setback reduced Genzyme's 2008 non-GAAP earnings guidance to approximately $3.90 a share. [3] Wall Street analysts were looking for earnings of 93 cents a share on revenue of $1.085 billion.[10] The latest mean estimates of analysts polled by Thomson Reuters were earnings of 93 cents a share and revenue of $1.09 billion.[9]
For the second quarter the company sees earnings per share in the mid 90s cents range, excluding items.[1] The company said it expects 20 percent growth in earnings per share by 2011.[26]
Analysts were looking for the firm to post a second-quarter profit of $0.96 per share.[11] For the second quarter, adjusted profit is expected to be in the mid-90 cents per share range.[2]
Sales of Cerezyme for Gaucher disease rose 15 per cent to $US304.3 million for the quarter, while sales of Fabrazyme for Fabry disease jumped 16 per cent to $US116.5 million.[1] The genetics business saw sales rise 12 per cent to $US74.3 million, fuelled by growth in reproductive diagnostic testing.[1] Kidney treatments Renagel and Renvela registered a growth of 23% in sales to $168.7 million from $137.4 million last year.[8]
First-quarter sales of Pompe disease drug Myozyme rose 78% to $67.3 million, just shy of Wall Street's target of $68 million.[10]
Genzyme's kidney disease medicines, Renagel and Renvela, grew a combined 23 per cent to $US168.7 million.[1] Under terms of that agreement, Genzyme is responsible for development and commercialization of the product, with a transition period of two years. Isis was able to cap its costs during the transition period at $75 million.[27] Genzyme's press releases and other company information are available at http://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. Any statement describing Isis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement, including those statements that are described as Isis' goals. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such products. These and other risks concerning Isis' programs are described in additional detail in Isis' annual report on Form 10-K for the year ended December 31, 2007, which is on file with the SEC. Copies of this and other documents are available from the Company.[14]
In order to sell the Allston version of the drug in the United States, Genzyme said it will have to file another application with new data showing the drug is safe and effective in large numbers of patients. Because it was already preparing to submit such data anyway, Genzyme said it believes it probably will be able to start selling the Allston-made drug early next year.[13] Per the new requirement, the companies have sped up plans for an outcomes trial in high-risk patients that will begin in 2009. Traditional outcome studies include 10,000-plus patients and take five to 10 years to complete, but the companies said they expect their trial can be smaller and shorter. They didn't provide specifics on a Friday morning conference call. "Having outcome data earlier on in the development process will be important to patients and serve to enhance the value of this treatment," said Genzyme CEO Henri Termeer. "We plan to engage in discussions with regulatory agencies in Europe and the rest of the world, and look forward to receiving their feedback."[7]
A Phase III study of mipomersen in hoFH patients is currently enrolling study subjects. The companies, which are co-developing the drug, said they plan to conduct an outcome trial to support full approval of mipomersen for hoFH and to expand its indication to include other patients with high cholesterol who are at high risk for cardiovascular events.[16] The FDA is now asking for data from two ongoing preclinical carcinogenicity studies in order for the drug to be filed in homozygous familial hypercholesterolemia, a severe form of high cholesterol that creates an increased risk of premature cardiovascular disease. The companies now expect to file for this indication in 2010, a one-year delay.[7] The FDA will require data from two ongoing preclinical studies for the drug's cancer risk to be included in the hoFH filing, which is now anticipated to take place in 2010, the companies said.[16]
The company said it had been preparing to give the FDA positive data collected from 900 patients who are already taking the Allston-made drug.[13] The company said it would have to submit a BLA to get U.S. commercial approval to produce Myoszyme at the 2000-liter scale. The FDA is expected to act on the application by the end of this year.[8] The material approved by the FDA was made in 160 litre batches. The company says it is now unable to satisfy demand for the drug by making it at this scale, so has requested FDA approval for the same drug made on a 2000 litre scale.[20]
Earlier in the week Genzyme said that the FDA will require it to submit a separate application for the 2000L production of the drug, considering it a different product than the 160L scale, which is already approved.[10] Currently Myozyme is made in a 160 litre-scale bioprocessing facility but rising demand for the drug led Genzyme to ask for approval to make it in a much-larger, 2,000-litre bioreactor. Somewhat bizarrely, this will likely lead to a situation where there will be two versions of the same product on the market, differentiated by their production process, said Genzyme in a conference call, It pointed out that production at this larger scale has already been approved in more than 40 countries.[12]
Genzyme Corporation recently announced that the FDA has informed the company of its opinion that Myozyme® (alglucosidase alfa) produced at the 160L bioreactor scale and Myozyme produced at the 2000L scale should be classified as two different products because of differences in the carbohydrate structures of the molecules.[21] The FDA will require Genzyme to submit a separate biologics license application (BLA) to gain approval for Myozyme produced at the 2000L scale.[21]
CARLSBAD, Calif. and CAMBRIDGE, Mass., April 25, 2008 /PRNewswire/ -- Isis Pharmaceuticals, Inc. and Genzyme Corp. announced today that the FDA has provided guidance regarding approval requirements for mipomersen.[14] In response to the FDA's guidance, the companies are revising the development plan for mipomersen to accelerate plans for an outcome study. Isis and Genzyme plan to communicate further details of the revised development plan as they are finalized. "Because our development plan, and our joint plan with Genzyme, has always included outcome studies to maximize the profile and commercial potential of mipomersen, this FDA guidance accelerates these planned studies and simplifies the overall development path for mipomersen," said Dr Stan Crooke, Chairman and Chief Executive Officer of Isis.[14] A phase 3 study of mipomersen in hoFH is currently enrolling patients. In response to the FDA's guidance, the companies are revising the development plan for mipomersen to accelerate plans for an outcome study and planning to communicate the revised development plan when they are finalized.[4]
Mipomersen is being developed to treat a condition called homozygous familial hypercholesterolemia, which is a severe form of high cholesterol. In a statement, the companies said they will speed up plans for an outcomes trial based on FDA guidance.[15] The companies noted that the FDA has indicated that reduction of LDL-cholesterol is an acceptable surrogate endpoint for accelerated approval of mipomersen for use in patients with homozygous familial hypercholesterolemia, or hoFH.[4]
'We have considerable clinical experience, with hundreds of patients already on 2000 litre material around the world. Because of carbohydrate differences, the FDA has decided that these are two separate products. 'This decision gives an idea of the amount of work companies will have to do to simply move their own product from one site to another - so its going to be at least as difficult for new companies to make a biosimilar,' UK-based biopharmaceuticals consultant Malcolm Rhodes told Chemistry World. Lincoln Tsang, who chairs the UK BioIndustry Association's regulatory advisory committee, thinks that the FDA's handling of new versions of biologics produced by different processes will not necessarily inform its stance on biosimilar generics. He adds that U.S., European and Japanese regulators have a shared set of technical guidelines on how to assess product comparability as a result of process change. 'The technical guidelines have been put in place quite recently,' he says, 'and before that regulators applied their own guidelines.[20] While Myozyme (alglucosidase alfa) made at another plant in Framingham is already registered on the U.S. market as a treatment for Pompe disease, the FDA has ruled thatthe Allstron versionmust be considered a different product because of differences in the biological signature of the active molecule. In essence, the agency is concerned about how the protein made at is glycosylated - the pattern of carbohydrate side chains that can have an impact on its behaviour in the body.[12]
U.S. regulators say Myozyme produced at the 160-liter bioreactor scale and the same drug made at the 2,000-liter scale should be classified as two different products.[10]
The drug is approved in more than 40 nations but not yet in the U.S. on a wide scale, and Genzyme needs FDA clearance for a 2,000-liter manufacturing process to make it more widely available.[2] The first-quarter results the drug developer released yesterday included lowered 2008 guidance, following a negative surprise from the FDA earlier in the week. Genzyme's first-quarter numbers weren't bad at all, despite its dimmer outlook.[3] Cowen and Co. analyst Eric Schmidt suggested the postponement and FDA guidance could impact financial terms of the deal between Isis and Genzyme. The partners are expected to finalize their partnership later in the quarter. He downgraded shares of Isis to "Neutral" from "Outperform", adding that the postponement and a lack of milestones make for a less optimistic outlook on the stock's near-term performance.[15] The recent quarter includes a charge of $56 million related to Genzyme's deal with Isis Pharmaceutical ISIS.[10] The company said the net income dip was primarily the result an after-tax charge of $56.5 million related to the company's $150 million investment in Isis Pharmaceuticals Inc.[26]
Isis Pharmaceuticals is a registered trademark of Isis Pharmaceuticals, Inc. Ibis Biosciences and Ibis T5000 are trademarks of Ibis Biosciences, Inc. Regulus Therapeutics is a trademark of Regulus Therapeutics LLC. These statements are subject to risks and uncertainties that could cause actual results to differ materially from those forecasted. These statements speak only as of today's date and Genzyme undertakes no obligation to update or revise the statements.[14] It looks like Genzyme Corp.'s partnership with Isis Pharmaceuticals Inc. to develop a new drug that lowers bad cholesterol is starting to bear fruit.[5]
As part of the strategic relationship, Genzyme would also have preferred access to future Isis drugs for CNS and certain rare diseases. Earlier in October, Isis had announced safety results from its ongoing Phase 2 open label study of drug candidate ISIS 301012 in patients with familial hypercholesterolemia or FH and said the drug continues to be well tolerated by these patients who were treated for five or more months.[4] In January, Isis and entered a major strategic alliance, under which Genzyme would develop and commercialize Mipomersen, Isis' lipid-lowering treatment for high risk cardiovascular patients that utilizes novel antisense technology.[4] Upon completion of final contracts, Genzyme would pay Isis a $175 million up-front mipomersen license fee. In addition to this initial $325 million, Isis also has the opportunity to receive from Genzyme up to $825 million in development and regulatory milestone payments plus up to $750 million in commercial milestone payments.[4] The deal also accounted for potential milestone payments of more than $1.5 billion for Isis, but $825 million of that money is tied to development and approvals, while $750 million depends on commercial success.[27]
ISIS closed Thursday's regular trading session at $16.80, up $0.05 on a volume of 0.85 million shares.[4] Shares of the Cambridge, Mass. -based company were up $1.58, or 2.2%, at $73.13 in recent trading Wedneday.[10]
The Cambridge, Mass. -based company (Nasdaq: GENZ) reported $1.1 billion in revenue during the quarter ending March 31, up from $883.2 million over the same period in 2007.[24] Revenue climbed 25% to $1.1 billion, from $883.2 million in the first quarter of 2007.[10] Stronger sales helped Genzyme Corp. grow revenue more than 25 percent in its fiscal 2008 first quarter.[24] Sales of the biotech company's Avonex exceed Wall Street targets in the first quarter.[10]
The company expects to begin providing U.S. patients with commercial 2000L Myozyme during the first quarter of 2009.[21]
"We had a very strong first quarter to start the year," said Henri A. Termeer, Genzyme's chairman and chief executive officer.[25] "We had a very strong first quarter to start the year," said Genzyme CEO Henri Termeer in a release on Wednesday.[10]
Genzyme will host a conference call today at 11:00 a.m. Eastern to discuss results for the first quarter of 2008.[25]
The firm's adjusted bottom-line figure still came in above the amount predicted by analysts, bolstered by a rise in revenues. The company also provided guidance for the second quarter, with results to be impacted by some investments it is making in expanding its product offerings. [11] We are lowering our FY 08 (Jun) revenue growth estimate to 5.5% from 8% on concerns about the U.S. economy, pricing pressures in host bus adaptors and mezzanine cards, and a possible decline in demand after a strong quarter for new products in the embedded storage products line.[28]
The agreement also allows for profit sharing, which could garner the 30 percent of the product's sales revenues for Isis upon mipomersen's introduction to the market.[27] Genzyme said the revenue jump came from increased sales of a number of treatments.[24] Genzyme Corp.' s (GENZ) first-quarter net income dropped 8.2% on charges as revenue jumped amid strong growth of treatments for Gaucher and renal diseases.[9] Sales for the quarter registered a double-digit increase on strong growth in sales of treatments for lysosomal storage disorders and renal disease.[8] We have concerns about margins, hurt by growing traffic acquisition costs and notable spending on sales and R&D.; We are cutting our '08 per-ADS earnings projection to $3.68 from $3.91, but raising our '09 estimate to $6.30 from $5.88 on moderating investment growth.[28] RIO posts Q1 earnings per ADS of $0.41 vs. $0.46 on a 4.6% sales gain, shy of our $0.51 estimate because of foreign exchange losses.[28]
Within the company's transplant business, quarterly sales of Thymoglobulin and Lymphoglobuline increased 11% to $43.7 million, as worldwide demand for Thymoglobulin remained strong.[8] Aldurazyme sales rose 38% to $37.0 million, while Thyrogen sales grew 28% to $33.8 million.[8]
Total revenue for the quarter grew 25% to $1.1 billion from $883.2 million in same period a year ago.[11] Revenue for the quarter rose 25 per cent to $US1.1 billion, exceeding Wall Street forecasts of $US1.09 billion.[1]
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.[14]
Separately, the company announced plans for construction of a $90 million plant in China.[22]
The news prompted analysts at Cowen & Company LLC to downgrade Isis to neutral. Uncertainty about when the companies will finalize their mipomersen partnership, or whether they will revise the terms of their agreement still abounds. Final contracts for the deal could be completed this quarter.[27] The U.S. Food and Drug Administration said that reduction of low-density-lipoprotein (LDL)-cholesterol is an acceptable "surrogate endpoint" for accelerated approval of mipomersen in patients with homozygous FH, Isis said.[18] The Food and Drug Administration determined that a bigger batch size of Genzyme Corp.' s (GENZ) Myozyme must be classified separately from a lower batch size, which already has manufacturing approval.[9] Genzyme said it will have to reapply with the Food and Drug Administration for Myozyme approval at a larger manufacturing capacity, delaying a decision by up to six months.[2]
The U.S. Food and Drug Administration has rejected an application from biotechnology firm Genzyme to make its already-approved, protein-based drug Myozyme in larger batches.[20]
Analysts, on average, were expecting 96 cents a share. Genzyme lowered some of its 2008 expectations following an unfavorable Food and Drug Administration ruling.[9]
For broader, high-risk populations, the U.S. regulatory agency will require an outcomes study. "This appears to represent a shift in FDA thinking away from surrogate marker approvals for drugs that treat hypercholesterolemia," wrote Cowen and Co. analyst Eric Schmidt in a note to investors. [7] The FDA did not a return a call seeking comment. Congress is currently considering at least two bills to create a process to let drug makers market generic versions of biologic drugs, such as those made by Genzyme and many other biotechs. Lobbyists disagree about how long brand-name drug makers should be entitled to exclusive rights to their drugs before facing generic competition, as well as how difficult the approval process should be.[13] The FDA decision also suggests that regulators may be reluctant to approve any generic versions of biologic drugs - called biogenerics or biosimilars - without clinical data proving the drugs are at least as safe and effective as the originals if there are even slight differences in the compounds. "It sends a very loud message and sets a very high bar," said Alison Lawton, Genzyme's senior vice president for regulatory affairs.[13] Unlike traditional, small molecule-based drugs, it is often difficult to prove that a biologic generic has an identical make-up to the original drug. 'Clearly the FDA is setting a bar here,' said Alison Lawton, head of regulatory affairs at Genzyme, when asked what the regulator's decision meant for it's likely stance on biosimilars.[20]
"We are extremely disappointed in the FDA's decision because it will further delay broad patient access to Myozyme, which is not possible under the MTAP program," said Henri A. Termeer, Genzyme's chairman and chief executive officer. [21] 'We're disappointed by the FDA's decision,' says Henri Termeer, Genzyme's chief executive officer. The decision 'does indicate a clear difference to how the EMEA, and the Japanese authorities, look at these matters of similarity.[20]
Genzyme said the FDA was concerned about slight differences in the carbohydrate structures of the molecules.[13]
The drug candidate is in late-stage development, but the FDA said it will require data from two ongoing preclinical studies testing whether or not mipomersen causes cancer.[15] Currently in phase 3 development, mipomersen has been shown in phase 2 trials to reduce cholesterol and other atherogenic lipids more than 40% beyond reductions achieved with current standard lipid-lowering drugs, enabling more patients to achieve lipid targets.[4] The company said the forecast reflects investments in product launches or expansions, as well as late-stage trials of an experimental multiple sclerosis drug.[1] The company's products and services are focused on rare inherited disorders, kidney disease, orthopaedics, cancer, transplant, and diagnostic testing. Genzyme's commitment to innovation continues today with a substantial development program focused on these fields, as well as immune disease, infectious disease, and other areas of unmet medical need.[14] Mr Termeer said the company continues to look for other deals that fit Genzyme's focus on rare, difficult to treat diseases.[1]
NEW YORK, April 23 (newratings.com) - Analysts at Lazard Capital maintain their "buy" rating on Genzyme Corporation (GENZ), while reducing their estimates for the company.[23] Genzyme will now be forced to file a new Biologics License Application (BLA) to sell Myozyme made at the plant, holding back the increase in production capacity by six months or more.[12] Currently, Genzyme has U.S. approval to sell Myozyme manufactured at the 160 liter.[22] The agency determined that a bigger batch size of Genzyme's Myozyme must be classified separately from a lower batch size, which already has manufacturing approval, and get separate approval.[9]
Despite Isis' dramatic drop, Genzyme only slipped 1.3%, or 98 cents, to $72.43. [6] Genzyme (NASDAQ:GENZ) fared better, dropping only 85 cents, to end the day at $72.49.[27]
Carlsbad Calif. -based Isis (NASDAQ:ISIS) was down 28.6 percent, or $4.81, on the news, closing at $11.99 in Friday trading. [27] ABOUT ISIS PHARMACEUTICALS, INC. Isis is exploiting its expertise in RNA to discover and develop novel drugs for its product pipeline and for its partners.[14] According to the FDA Omnitrope is not officially called a generic because it is not rated as therapeutically equivalent to any of the other approved human growth hormone (hGH) products. It is however "sufficiently similar" to them for approval. It was cleared under existing legislation - the 505(b)(2) pathway of the Hatch-Waxman Act - which is only really suitable for approving small, simple follow-on biologics.[12] "The landscape for cholesterol has changed and the FDA is reassessing the most appropriate path for drug approval," Monane said.[6]
The companies are studying the drug in patients with a rare genetic disorder, known as familial hypercholesterolemia (FH), characterized by high "bad" cholesterol.[18] Mipomersen is currently in phase 3 development for patients with homozygous familial hypercholesterolemia, a disease which creates a greatly increased risk of premature cardiovascular disease (CVD) and CVD-related death.[14] A weekly injectable therapeutic, mipomersen is being developed primarily for patients at significant cardiovascular risk who are unable to achieve target cholesterol levels with statins alone or who are intolerant of statins.[4]
"Conducting an outcome study in parallel with our continued evaluation of the effects of mipomersen on atherogenic lipids will allow us to submit a much stronger NDA (application) for high risk patients," said Isis Chairman and Chief Executive Dr. Stan Crooke, in a statement.[15]
The study, which will be conducted in very high-risk patients, likely will begin in early 2009. In a conference call with investors, Isis representatives said they expect the trial to be smaller than traditional outcome studies of 10,000 patients, and much shorter than typical outcome trials, which can last from five to 10 years.[27]
A webcast replay will be available for a limited time. CONTACT: Investors, Kristina Lemonidis, +1-760-603-2490, or Media, AmyBlackley, Ph.D., +1-760-603-2772, both of Isis Pharmaceuticals; orInvestors, Patrick Flanigan, +1-617-768-6563, or Media, Erin Emlock,+1-617-768-6923, both of Genzyme Corp.[14] 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.[25]
Infants and children with Pompe disease in the United States continue to receive commercially approved Myozyme produced at the 160L scale.[21] Myozyme is an enzyme-replacement therapy, the first drug developed to treat Pompe disease, a rare genetic disorder that causes muscle degeneration, ultimately leading to death.[20]
Isis' drug development programs are focused on treating cardiovascular and metabolic diseases.[14] Isis' partners are developing antisense drugs invented by Isis to treat a wide variety of diseases.[14]
Mipomersen, formerly ISIS 301012, is a lipid-lowering drug targeting apolipoprotein B-100.[4] As an innovator in RNA-based drug discovery and development, Isis is the owner or exclusive licensee of over 1,500 issued patents worldwide.[14] Earlier, submission was expected in 2009, Isis spokeswoman Amy Blackley said. "It is clear that the time line has shifted significantly to the right and development risk and expense have increased," Leerink Swann analyst Joseph Schwartz said in a note to clients.[18]
S&P; analyst, S. Kessler, says, "BIDU posts Q1 per-ADS profit of $0.60 vs. $0.32, $0.08 below our forecast. [28] In addition to the up-front payment, once the product is launched, the two companies will share profits.[4] 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.[14] Genzyme said it has submitted data only from a very small number of patients, making it hard to tell whether the molecular differences were significant.[13] To make sure the differences weren't important, executives said the FDA demanded data from larger numbers of patients that proves the version made in the larger plant is safe and effective.[13] The FDA concerns about Myozyme centered on carbohydrate differences in Myozyme produced at the 160 liter scale and 2000 liter scale.[22]
The data on the larger-scale version of Myozyme has already been seen by the European Medicines Agency (EMEA), who approved that drug for use in Europe in March 2006.[20] For more complex applications a brand new regulatory route will have to be mapped out. In this respect the U.S. is lagging well behind the EU, which has already approved the first handful of biosimilars in order to provide lower-cost alternatives to brandname biologic drugs and alleviate some of the burden on cash-strapped healthcare systems of prescribing these drugs.[12] The Company has successfully commercialized the world's first antisense drug and has 18 drugs in development.[14]
Barr Pharmaceuticals is an active Stock Advisor pick. Fool contributor Brian Lawler does not own shares of any company mentioned in this article.[3]
The tech-heavy Nasdaq, suffering from disappointing earnings from Microsoft (nasdaq: MSFT - news - people ), sank 0.3%, or 5.99 points, to 2,422.93, although it still closed up 0.8% for the week. [19] Much for accurate guidance. at least for Genzyme (Nasdaq: GENZ ).[3]
Updated from 9:50 a.m. EDT Genzyme GENZ posted first-quarter financial results Wednesday morning that beat Wall Street estimates on the top and bottom lines.[10] Genzyme will host a conference call on July 23, 2008 at 11:00 a.m. Eastern to discuss financial results for the second quarter of 2008.[25]
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.[14]
Because the Framingham plant has only limited capacity, it is currently supplying the Allston drug to 140 U.S. Pompe patients free. [13] Unlike in Europe, the U.S. is yet to establish a framework by which generic drug makers can seek approval to sell copies of off-patent biologic drugs, or 'biosimilars'.[20]
Mipomersen is an antisense drug that reduces the production of a protein critical to the synthesis and transport of "bad" cholesterol. [17] In phase 2 studies, mipomersen, a weekly injectable therapeutic, was observed to reduce cholesterol and other atherogenic lipids beyond reductions achieved with standard lipid-lowering drugs.[14] Phase II studies showed that mipomersen reduced cholesterol and other atherogenic lipids beyond reductions achieved with standard lipid-lowering drugs.[27]
SOURCES
1. Business Spectator - Genzyme Q1 profit tops expectations
2. Genzyme 1Q profit slips on charge, but tops estimate - Forbes.com
3. FDA Stings Genzyme Earnings
4. FDA Guides Isis On Development Path For Mipomersen; Stock Down 30% - Update [ISIS] - RTTNews, Today's Top Stories, Global Newswires, ToDay's Top News,Global Business news .
5. Genzyme and partner, Isis, get guidelines for drug approval - Boston Business Journal:
6. Isis Is Delayed - Forbes.com
7. FDA Delays Hit Genzyme, Isis JV Deal | Biotech | ALNY GENZ GSK ISIS - TheStreet.com
8. Genzyme Q1 Profit Falls On Isis Investment; Sees Q2 Non-GAAP EPS In Mid $0.90s - Update 2 [GENZ] - RTTNews, Today's Top Stories, Global Newswires, ToDay's Top News,Global Business news .
9. UPDATE: Genzyme 1Q Net Drops 8.2% Amid Investment Charge
10. Genzyme Beats Earnings Targets | Biotech | AMGN BIIB ELN GENZ IMCL ISIS - TheStreet.com
11. Genzyme Q1 Profit Slips On Charges [GENZ] - RTTNews, Today's Top Stories, Global Newswires, ToDay's Top News,Global Business news .
12. Genzyme knockback has implications for biosimilars
13. Treatment and Health News (Orphan Disease Portal)
14. PharmaLive: FDA Provides Clarity to Isis Regarding the Development Path for Mipomersen
15. Isis, Genzyme expect 1-year delay on cholesterol drug | Chron.com - Houston Chronicle
16. Isis/Genzyme: FDA provides guidance on approval requirements for mipomersen - Forbes.com
17. Isis Pharmaceuticals (ISIS) NewsBite - FDA Wants More Data on Isis' Cholesterol Drug
18. UPDATE 2-Isis to delay cholesterol drug data; shares drop | Markets | Markets News | Reuters
19. U.S. Stocks Finish Strong - Forbes.com
20. FDA takes tough line on biologic drug
21. Genzyme And FDA At Odds Over Myozyme® Manufacturing - Pharmaceutical Processing
22. Therapeutics Daily
23. Genzyme "buy," target price reduced - newratings.com
24. One-time charge hits Genzyme net income - Boston Business Journal:
25. Genzyme Reports Strong First-Quarter Growth
26. www.wbjournal.com - Genzyme Earnings Dip As Revenue Grows
27. Isis Shares Slip on Need for Mipomersen Data
28. StreetInsider.com
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