BLA

Ipsen inks a string of deals in expansion effort

Thu, 05 Jun 2008 06:59:58 -0400

France's Ipsen is buying out Tercica in a $404 million deal, one of three new pacts designed to make Ipsen a billion-dollar a year player in the U.S. Ipsen will pay $9 a share for every share of Tercica it doesn't already own. That's more than double Tercica's $4.41 a share close yesterday. Like a lot of developers, Tercica's been losing money. But the biotech recently launched Somatuline for acromegaly, a pituitary gland disorder.

In a separate deal, Ipsen is buying out Vernalis' U.S. subsidiary, gaining the North American rights to Apokyn. And it inked a deal to acquire Octagen's OBI-1, which is being developed to treat hemophilia. The Octagen deal includes $10.5 million upfront and up to $26 million in milestones.

Taken together, Ipsen says the new acquisitions will position Ipsen to become a significant commercial player in the U.S.

- read Ipsen's release for more

Acambis lands $425M smallpox vaccine contract

Wed, 23 Apr 2008 06:59:56 -0400

The U.K.'s Acambis has won a $425 million contract to supply the CDC with its smallpox vaccine for 10 years. Acambis will establish a U.S.-based manufacturing capability for ACAM2000 and deliver a minimum of nine million doses per year of the drug. "The net proceeds will enable us to invest in manufacturing facilities for the U.S. contract, progress our vaccine for hospital-acquired C.difficile infections and further our program of new vaccines against influenza, genital herpes and other significant unmet medical needs," Acambis head Ian Garland told ShareCast.

- see the Acambis release
- read this report from ShareCast

Genzyme gets biosimilar rejection, builds R&D plant

Tue, 22 Apr 2008 06:59:58 -0400

The FDA have given a thumbs down to Genzyme's request for permission to sell its Pompe disease drug Myozyme manufactured in an Allston, MA plant. Genzyme is already manufacturing the drug in a smaller Framingham-based plant and wants to ramp up production at the larger Allston location. But the FDA ruled that the Allston-produced Myozyme must "be classified as [a different product] because of differences in the carbohydrate structures of the molecules." The FDA said Genzyme has to submit a separate BLA to gain approval for Myozyme produced on a larger scale.

This rejection underscores the difficulty biosimilars face in the United States. "The FDA decision...suggests that regulators may be reluctant to approve any generic versions of biologic drugs...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," observes the Boston Globe. Currently, the U.S. has no pathway to quickly approve biosimilar drugs.

As Genzyme points out, Myozyme production has already been scaled up in 40 other countries. "Based on the global clinical experience of nearly 900 patients of all ages currently receiving Myozyme produced at the larger scale...Genzyme believes that Myozyme produced at both the 160L and 2000L scales is clinically effective and safe," the company said in a statement. The company expects the FDA to act on the application by the end of the year.

- see this release from Genzyme
- read the Boston Globe article

ALSO: Genzyme is opening a major new R&D center in Beijing. The facility will house 350 employees and will cost an estimated $90 million. Researchers in the Beijing plant will develop drugs in four focused areas: orthopedics, transplant and immune disease, oncology, endocrinology and cardiovascular disease. According the the release, the R&D facility "is part of Genzyme's ongoing global expansion and commitment to establishing a long-term presence in China." Report

ALSO NOTED: Ipsen files new BLA;Amgen selects Vectibix test; and much more...

Mon, 17 Mar 2008 07:59:50 -0400

> Ipsen says it has submitted a new BLA for Reloxin, addressing concerns raised by the FDA when the agency declined to file its BLA in January. Release

> Amgen says it will use Lab21's diagnostic test to see which patients are suitable for its cancer drug Vectibix. Report

> NanoBio Corporation reported that a Phase IIb study of NB-001, a topical lotion to treat cold sores, met its primary and secondary endpoints by demonstrating clear efficacy and safety in 482 patients. Release

> The FDA is hustling up new programs designed to shore up confidence in drug safety--and in its own ability to ensure it. Report

> Pay for the CEOs of Wyeth and Pfizer was down in 2007. Report

> The wrestling match in Biovail's boardroom is getting ugly. Founder and former CEO Eugene Melnyk called for a housecleaning on the company's board. Report

> Rumor Mill: Less than 10 days after CEO Jeffrey Kindler pooh-poohed big acquisitions, U.K. markets are awash with rumors that Pfizer is on the hunt for British pharma group Shire. Report

And Finally... A fresh controversy over tainted drugs may be just what the FDA needs to win new federal funding. Article

Experts to weigh in on Amgen's Nplate

Wed, 12 Mar 2008 07:59:55 -0400

Amgen is going before the FDA's Oncologic Drugs Advisory Committee (ODAC) in order to discuss its BLA for Nplate (romiplostim). The drug is a treatment for thrombocytopenia, a disorder that causes the body to attack its own blood clotting cells. The FDA is concerned about adverse reactions noted in clinical trials. These include significantly higher platelet levels, dangerous blood clots, bone marrow growth and malignant tumors in those taking the drug. The experts will advise the FDA whether Nplate's benefits outweigh the risks, and whether it should be given a limited approval.

- check out this release
- and read the AP article for more

ALSO: Here's a peek into the FDA playbook for tomorrow's advisory panel on Amgen and Johnson & Johnson's anemia drugs. According to documents posted on the agency's website, those expert advisors will be asked to consider several limitations on the meds, which have been linked to increased tumor growth and reduced survival among some cancer patients. And the options include yanking FDA approval for that use. Report

Related Article:
Amgen platelet therapy hits endpoint in Phase III. Report

Medicis shares sink as FDA rejects BLA

Thu, 31 Jan 2008 06:59:56 -0500

Medicis Pharmaceutical earned an "incomplete" grade on its latest BLA. The FDA has refused to accept the application for Reloxin--botulinum toxin type A--for a variety of shortfalls, including a gap in how it would live up to its responsibilities as a manufacturer. Another red flag: the application "included letters of authorization supporting a separate BLA submitted by Ipsen." For its part, Medicis, which makes wrinkle-fillers and other products, says it believes its application is strong, adding they will continue to work with the agency. Medicis also noted that the denial was likely to delay any product launch. Investors responded to the fiasco by bidding down company shares by 11 percent.

- check out the AP report

Related Article:
Medicis wins anti-wrinkle drug Reloxin in licensing pact. Report

Emerging Drug Developer: Tolerx

Fri, 26 Oct 2007 06:59:59 -0400

Emerging Drug Developer: Tolerx

Holding promising Phase II data for a monoclonal antibody lined up for a late-stage race to the FDA, Tolerx found itself in the sweet spot of biotech deal making. And GlaxoSmithKline was just one of a number of companies to make a bid for it. For Tolerx CEO Douglas Ringler, the $760 million deal they struck gives the company enough cash to make it all the way through to a BLA. But there's little time for relaxation. Tolerx is engaged in a competitive race to get its CD3 program all the way to the FDA. And it plans to be first. Article

Press Release: Dendreon Corporation's PROVENGE Granted FDA Priority Review

Maureen Martino — Tue, 16 Jan 2007 11:20:58 -0500

Dendreon Corporation's PROVENGE Granted FDA Priority Review For The Treatment Of Asymptomatic, Metastatic, Androgen-Independent Prostate Cancer

SEATTLE, Jan. 16 -- Dendreon Corporation today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing and has assigned priority review status to the Company's Biologics License Application (BLA) for PROVENGE(R) (sipuleucel-T), its investigational active cellular immunotherapy for the treatment of asymptomatic, metastatic, androgen-independent (also known as hormone refractory) prostate cancer.

Priority Review is granted to products that, if approved, would provide a significant improvement in the safety or effectiveness of the treatment, diagnosis or prevention of a serious or life-threatening disease. The goal for reviewing a product with Priority Review status is six months from the filing date. The Prescription Drug User Fee Act (PDUFA) date for completion of review by the FDA of the PROVENGE BLA is May 15, 2007.

"Clinical trials have shown that PROVENGE increases survival and is generally well tolerated in men with late-stage prostate cancer, a highly prevalent disease for which there are currently few available treatment options," said Mitchell H. Gold, president and chief executive officer of Dendreon. "We are extremely pleased the FDA has granted priority review to PROVENGE, which may represent an important new treatment option for men suffering from prostate cancer."

Prostate cancer is the most common non-skin cancer in the United States and the third most common cancer worldwide. More than one million men in the United States have prostate cancer, with an estimated 232,000 new cases of prostate cancer diagnosed each year. More than 30,000 men die each year of the disease.

The BLA submission is based primarily on an improvement in overall survival observed in Study D9901, a multi-center, randomized, double-blind, placebo-controlled Phase 3 Study, the results of which were published in the July issue of the Journal of Clinical Oncology.

About PROVENGE

PROVENGE (sipuleucel-T) is an investigational product that may represent the first in a new class of active cellular immunotherapies (ACIs) that are uniquely designed to stimulate a patient's own immune system. PROVENGE is in late-stage clinical development for the treatment of patients with advanced prostate cancer. In clinical studies, patients typically received three infusions over a one-month period as a complete course of therapy.

About Dendreon

Dendreon Corporation is a biotechnology company whose mission is to target cancer and transform lives through the discovery, development and commercialization of novel therapeutics that harness the immune system to fight cancer. The Company applies its expertise in antigen identification, engineering and cell processing to produce active cellular immunotherapy product candidates designed to stimulate an immune response. Active cellular immunotherapy holds promise because it may provide patients with a meaningful clinical benefit, such as survival, combined with low toxicity. The Company has headquarters in Seattle and is traded on the Nasdaq Global Market under the symbol DNDN. For more information about the Company and its programs, visit www.dendreon.com.

Except for historical information contained herein, this news release contains forward-looking statements that are subject to risks and uncertainties surrounding the efficacy of PROVENGE to treat men suffering from prostate cancer, risks and uncertainties surrounding the presentation of data to the FDA and approval of product applications by the FDA and risks and uncertainties inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics. Factors that may cause such differences include risks related to our limited operating history, risks associated with completing our clinical trials, the risk that the safety and/or efficacy results of a clinical trial for PROVENGE will not support an application for a biologics license, the risk that the FDA may interpret data differently than we do or require more data or a more rigorous analysis of data than expected, the risk that the FDA will not approve a product for which a biologics license has been applied, the risk that the results of a clinical trial for PROVENGE or other product may not be indicative of results obtained in a later clinical trial, risks that we may lack the financial resources and access to capital to fund required clinical trials or commercialization of PROVENGE, our dependence on the efforts of third parties, and our dependence on intellectual property. Further information on the factors and risks that could affect Dendreon's business, financial condition and results of operations are contained in Dendreon's public disclosure filings with the U.S. Securities and Exchange Commission, which are available at www.sec.gov.

Press Release: Auxilium Pharmaceuticals Amends Agreement with Cobra Biologics

Maureen Martino — Tue, 28 Nov 2006 12:47:26 -0500

Auxilium Pharmaceuticals Amends Agreement with Cobra Biologics Ltd.

Wednesday November 29, 7:30 am ET

Horsham Facility to Become Primary Commercial Source of AA4500 Development Relationship with Cobra Continues

MALVERN, Pa., Nov. 29 -- Auxilium Pharmaceuticals, Inc. (Nasdaq: AUXL - News), a specialty biopharmaceutical company, today announced that it has amended its manufacturing agreement with UK-based Cobra Biologics Ltd. Under the amended agreement, Cobra will complete only one BLA (Biologics License Application) batch of the active ingredient for AA4500, the Company's injectable enzyme. The amendment reflects Auxilium's decision to move forward with its facility in Horsham, Pennsylvania to support the BLA submission and as a primary source of the commercial supply of AA4500. As previously announced, Auxilium will use the materials produced by Cobra in its ongoing and potential future clinical trials for AA4500 for the treatment of Dupuytren's contracture and other indications. The companies agreed to continue their collaboration related to the technology transfer, ongoing stability programs and other services provided by Cobra.

"This amendment allows our Manufacturing, Quality Assurance and Development teams to focus on getting the Horsham facility ready for the BLA submission expected at the end of 2007," said Mr. Armando Anido, Auxilium's Chief Executive Officer and President. "The amendment also ensures that Cobra will continue to provide technology support and be involved as a development partner for this critical asset. We look forward to continuing our collaboration with the Cobra team."

About Auxilium

Auxilium Pharmaceuticals, Inc. is a specialty biopharmaceutical company with a focus on developing and marketing to urologists, endocrinologists, orthopedists and select primary care physicians. Auxilium markets Testim® 1%, a topical testosterone gel, for the treatment of hypogonadism through its approximately 180-person sales and marketing team. Auxilium has four projects in clinical development. Auxilium believes that AA4500, an injectable enzyme, is in phase III of development for the treatment of Dupuytren's contracture and is in phase II of development for the treatment of Peyronie's disease and Frozen Shoulder Syndrome (Adhesive Capsulitis). Auxilium's transmucosal film product candidate for the treatment of overactive bladder (AA4010) is in phase I of development. The Company is currently seeking a partner to further develop this product candidate. Auxilium has two pain products using its transmucosal film delivery system in pre-clinical development. Auxilium has rights to six additional pain products and products for hormone replacement and urologic disease using its transmucosal film delivery system, and options to all indications using AA4500 for non-topical formulations. For additional information, visit http://www.auxilium.com .

Safe Harbor Statement

This release contains "forward-looking-statements" within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the quality of the AA4500 produced and the work performed pursuant to the amended agreement with Cobra, the performance of Cobra under the amended agreement, the timing of the filing of the BLA submission for AA4500 for the treatment of Dupuytren's contracture, the timing for the commencement of various clinical trials for Auxilium's product candidates, including AA4500. All statements other than statements of historical facts contained in this release, including but not limited to, statements regarding future expectations, plans and prospects for the Company, financial guidance and other statements containing the words "believe," "may," "could," "will," "estimate," "continue," "anticipate," "intend," "should," "plan," "expect," and similar expressions, as they relate to the Company, constitute forward-looking statements. Actual results may differ materially from those reflected in these forward-looking statements due to various factors, including general financial, economic, regulatory and political conditions affecting the biotechnology and pharmaceutical industries and those discussed in the Company's Annual Report on Form 10-K for the period ended December 31, 2005 and the Company's Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2006 under the heading "Risk Factors", which is on file with the Securities and Exchange Commission (the "SEC") and may be accessed electronically by means of the SEC's home page on the Internet at http://www.sec.gov or by means of the Company's home page on the Internet at http://www.auxilium.com under the heading "Investor Relations -- SEC Filings." There may be additional risks that the Company does not presently know or that the Company currently believes are immaterial which could also cause actual results to differ from those contained in the forward-looking statements. Given these risks and uncertainties, any or all of these forward-looking statements may prove to be incorrect. Therefore, you should not rely on any such factors or forward-looking statements.

In addition, forward-looking statements provide the Company's expectations, plans or forecasts of future events and views as of the date of this release. The Company anticipates that subsequent events and developments will cause the Company's assessments to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's assessments as of any date subsequent to the date of this release.

Acambis shares plunge after U.S. nixes contract

Mon, 13 Nov 2006 19:01:38 -0500

Shares of Acambis took a beating after the company announced it was no longer in the competition for a U.S. contract to provide smallpox vaccine. Its stock plunged 39 percent after company officials said it was no longer being considered. Acambis said that it would arrange a meeting with government officials to "seek clarification." "We are surprised that the U.S. government would eliminate Acambis," CEO Gordon Cameron said. "We believe that our proposal would have met the requirements."

- here's the International Herald Tribune report on Acambis

Related Articles:
Acambis completes vaccine BLA. Report
Acambis researching single-shot solution to flu. Report

SPOTLIGHT: Acambis completes vaccine BLA

Tue, 18 Apr 2006 20:01:33 -0400

Acambis has completed its BLA to the FDA for its experimental smallpox vaccine, ACAM2000. The drug was given fast-track status in late 2004. Report

UCB submits BLA for Cimzia

Wed, 01 Mar 2006 19:01:35 -0500

Belgium's UCB has submitted a BLA to the FDA for Cimzia as a treatment for Crohn's disease. And the developer says it plans to file a European submission in a matter of weeks. If approved, Cimzia would be the first-ever biologic utilizing subcutaneous injection for the treatment of Crohn's disease.

"Our experience in the PRECiSE clinical trials program has shown Cimzia to be a well-tolerated and effective treatment," said William Sandborn, M.D., professor of medicine at the Mayo Clinic College of Medicine and a leading investigator in the Cimzia program. "In addition, subcutaneous administration is a welcomed attribute for patients and can offer greater convenience."

- read the report from AFX

Anti-anemia therapy from Roche passes multiple Phase III tests

Thu, 15 Dec 2005 19:01:39 -0500

Roche says that its anti-anemia drug CERA successfully completed four late-stage trials, laying the groundwork for a BLA and an eventual new drug that could garner more than a billion dollars in annual sales. Amgen has filed a lawsuit against Roche, claiming it infringed on six of its patents. But Roche insists that the litigation will not impede its marketing plans.

"The completion of the four CERA phase III maintenance studies is an important milestone in CERA's history, as we move forward with the Biologics License Application with expected filing with the US Food and Drug Administration in 2006," said Richard Hinson, vice president, operations, Hoffmann-La Roche. "CERA's unique ability to provide up to once-monthly dosing may potentially offer a convenient and efficacious treatment option for patients with anemia in all stages of chronic kidney disease."

- read this release for more information

SPOTLIGHT: BLA filed for Rituxan

Tue, 30 Aug 2005 20:01:33 -0400

Biogen Idec and Genentech have filed a BLA with federal regulators for the use of Rituxan in patients with rheumatoid arthritis. Release

SPOTLIGHT: Isolagen reports mixed Phase III data

Sun, 31 Jul 2005 20:01:33 -0400

Isolagen has produced mixed results for two Phase III studies of treating contour deformities with the Isolagen Process. The Exton, Pennsylvania-based biotech said it hit three out of four primary endpoints. Researchers say they'll launch a new study in November that can be used to support a BLA application in 2006. Release

Therion pockets $30M in new round

Thu, 16 Jun 2005 20:01:36 -0400

Cambridge, Massachusetts-based Therion Biologics has rounded up $30 million in its fourth round of venture capital. The round was led by Hans-Werner Hector, who helped found the software company SAP. Much of the money will fund the development of its cancer therapy, PanVac-VF and ProstVaC-VF. Phase II and Phase III data are expected in 2006.

"Therion is poised to make a significant impact on the treatment of cancer with our products designed to selectively seek out and destroy malignant cells without the serious side effects associated with cytotoxic chemotherapy," said Mark Leuchtenberger, president and CEO of Therion. "This financing reflects the ongoing interest among our investors for our programs and strategy as we move closer to a potential BLA filing for marketing approval of PANVAC-VF and the initiation of registration studies for PROSTVAC-VF in 2006."

- read this story from Mass High Tech for more