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Biopharmaceuticals Neurology FDA

 News Release - July 31, 2007

Accentia Biopharmaceuticals Files Pre-Investigational New Drug (IND) Application with FDA for Pivotal Phase 3 Clinical Trial of Revimmune for Refractory Multiple Sclerosis

First Therapy to Propose Restoration of Neurologic Function as the primary endpoint and the First to Offer the Potential for Elimination of the Autoimmunity Responsible for Multiple Sclerosis

TAMPA, Fla.--(HSMN NewsFeed)--Accentia Biopharmaceuticals Inc. (NASDAQ:ABPI ) has filed a preliminary Investigational New Drug (pre-IND) application with the U.S. Food and Drug Administration (FDA) for Revimmune(TM) in the treatment of refractory Multiple Sclerosis (MS). The Company has requested a meeting with the FDA within the next 60 days to discuss a proposed pivotal Phase 3 clinical trial involving approximately 270 patients with relapsing/remitting MS with a primary endpoint of improvement in function (reversal of disability). Unlike Revimmune, no other approved therapy for MS has shown a recovery of neurologic function and all others have been approved on the more limited basis of reducing the risk of disease exacerbation or progression. Accentia's proposal to use the restoration of neurologic function as the endpoint will be the first filed with the FDA.

Based on a clinical study at the Johns Hopkins University School of Medicine, which showed an unprecedented 42% average improvement in function, the Company believes that Revimmune holds the potential to restore function in many patients who have acute deficits due to MS. In addition, the Company believes that the long-term follow-up with patients in the proposed 48-week trial will demonstrate that Revimmune induces not only a reduction of the risk of exacerbations, but potentially also long-lasting remissions and/or cures in MS patients.

Dr. Howard Weiner, an internationally recognized physician and Professor of Neurology at the Harvard Medical School and Co-Director of the Center for Neurologic Diseases at the Brigham & Women's Hospital, recently conducted a satellite media tour and participated in interviews with television and radio stations across the U.S. to highlight the benefits of using Revimmune to treat MS patients. Key interviews from the tour can be found at www.accentia.net.

The Principal Investigator for the ongoing MS study with Revimmune at Johns Hopkins University School of Medicine is Dr. Douglas Kerr, Associate Professor of Neurology. The co-Principal Investigators on this study are Dr. Daniel Drachman, Dr. Robert Brodsky, and Dr. Adam Kaplin. The National Multiple Sclerosis Society has supported the clinical protocol at Johns Hopkins University.

Developed by Dr. Richard Jones, Dr. Robert Brodsky, and colleagues at the Johns Hopkins University School of Medicine, Revimmune temporarily eliminates peripheral immune cells, including the immune cells causing the autoimmunity, while selectively sparing hematopoeitic stem cells in the bone marrow. Investigators at Johns Hopkins discovered that stem cells uniquely have high levels of a particular protective enzyme that can be measured in advance of therapy, which makes them impervious to Revimmune, and allows the surviving stem cells to give rise to a new immune system over 2 to 3 weeks. The newly reconstituted peripheral immune system typically lacks the misdirected immunity to self-antigens, which is characteristic of autoimmune diseases.

Revimmune can be administered as an inpatient or outpatient infusion for 4 hours per day for 4 consecutive days. The treatment is intended to allow patients to recover at home while their immune system reconstitutes itself over a 2 to 3 week period. Revimmune includes a risk management program to enhance patient safety by ensuring appropriate patient selection, supportive care, and tracking of outcomes data.

BACKGROUND ON REVIMMUNE CLINICAL RESULTS

Studies at Johns Hopkins University School of Medicine by Dr. Jones, Dr. Brodsky, and colleagues have demonstrated the potential benefits of Revimmune in a variety of autoimmune diseases.

Multiple Sclerosis:

According to information from the National Multiple Sclerosis Society (http://nationalmssociety.org/), there are approximately 400,000 people in the U.S. with Multiple Sclerosis. For the clinical course, 85% of patients are in the category of relapsing-remitting. Based upon a paper by D. Hirtz et al. "How common are the 'common' neurologic disorders?," the annual incidence of Multiple Sclerosis in the U.S. was approximately 4.2 new cases per 100,000 population in 2005.

Revimmune treatment of 20 Multiple Sclerosis patients has resulted in the following successful outcomes in 2 published studies from C. Krishnan, D. Kerr et al. and D. Gladstone et al.:
  • All patients have had a reduction or elimination of new and enhancing lesions on the MRI
  • No patient has had a clinical exacerbation following treatment and most patients have had a reduction in EDSS and an improvement in the MSFC following treatment
  • During follow-up, no patients increased their baseline EDSS scores by more than 1.0
  • No patient had a new lesion on brain magnetic resonance imaging; no patient showed any enhancing lesions
Six medicines are currently approved for the treatment of RRMS: interferon (beta)-1b (Betaseron®), interferon (beta)-1a (Avonex® and Rebif®) glatiramer acetate (Copaxone®), mitoxantrone (Novantrone®) and natalizumab (TYSABRI®). A systematic review of 21 studies failed to reveal a difference among the interferons and glatirimer acetate in reducing the frequency of relapses (Galetta, Markowitz et al., 2002). Interferons were found to be more potent than glatiramer acetate in reducing gadolinium enhancement on MRI, with an 82-89% reduction from pretreatment levels. Disability progression was found to decrease between 12 and 37% in all the pivotal Phase 3 trials compared to placebo treated patients. The vast majority of patients continued to accrue disability as defined by EDSS, MSFC, neuro-cognitive studies, and brain parenchymal fraction (BPF).

Systemic Lupus:

Investigators have treated 40 severe systemic lupus erythematosus (SLE) patients in clinical studies with Revimmune. A significant improvement in the SLE diseases activity index was observed. There were 5 durable complete responses. Among severe, refractory cases, approximately 80% of patients treated had a complete or partial response when treated.

Myasthenia Gravis:

Using Revimmune, investigators have treated 11 patients with myasthenia gravis refractory to conventional immunosuppressive therapy. Nine of the subjects in the study markedly improved, and have returned to full activity.

Aplastic Anemia:

Acquired severe aplastic anemia (SAA) is a severe, life-threatening autoimmune disease wherein a patients' immune system mistakenly attacks their own stem cells in their bone marrow. Most SAA patients will die within a year of diagnosis. Investigators have treated 75 SAA patients with Revimmune and the majority of patients have achieved a complete remission without the need of other immunosuppressive agents.

Autoimmune Hemolytic Anemia:

Investigators have treated 10 patients with refractory autoimmune hemolytic anemia. After Revimmune treatment, all patients responded and became transfusion independent. There were 6 patients that achieved complete remission and 3 patients that achieved partial remission. There were no relapses at a median follow-up of 15 months and 7 of the 9 patients were able to discontinue steroids.

Experience with other autoimmune diseases:

Investigators have reported favorable case experience with refractory scleroderma, acquired pemphigus, rheumatoid arthritis, and Crohn's Disease.

References:

"How common are the 'common' neurologic disorders?;" D. Hirtz, D. J. Thurman, K. Gwinn-Hardy, M. Mohamed, A. R. Chaudhuri, and R. Zalutsky; Neurology 2007 68: 326-337.

"High-Dose Cyclophosphamide in the Treatment of Aggressive Multiple Sclerosis;" Chitra Krishnan, Daniel Drachman, Justin McArthur, David Irani, Avindra Nath, Carlos Pardo-Villamizar, David Yousem, Robert Brodsky, Peter Calabresi, Douglas A. Kerr, Baltimore, MD. AAN Abstract (P01.072); April 4, 2006.

"High-Dose Cyclophosphamide for Moderate to Severe Refractory Multiple Sclerosis;" Douglas E. Gladstone, MD; Kenneth W. Zamkoff, MD; Lauren Krupp, MD; Robert Peyster, MD; Patrick Sibony, MD; Christopher Christodoulou, PhD; Emily Locher, RN; Patricia K. Coyle, MD Arch Neurol/Vol 63, Published Online August 14, 2006.

"Immunomodulatory agents for the treatment of relapsing multiple sclerosis: a systematic review." Galetta, S. L., C. Markowitz, et al. (2002). Arch Intern Med 162(19): 2161-9.

About Accentia Biopharmaceuticals

Accentia Biopharmaceuticals, Inc. and its subsidiaries (collectively referred to as the "Company" or "Accentia") is a vertically integrated biopharmaceutical company focused on the development and commercialization of drug candidates that are in late-stage clinical development and typically are based on active pharmaceutical ingredients that have been previously approved by the FDA for other indications. Usually these drug candidates can access the accelerated 505(b)(2) regulatory approval pathway, which is generally less time-consuming and less expensive than the typical 505(b)(1) pathway that must be used for new chemical entities. The Company's lead product candidate is SinuNase(TM), a novel application and formulation of a known therapeutic to treat chronic rhinosinusitis. SinuNase has been granted Fast Track status by the FDA and it is currently in a pivotal Phase 3 clinical trial. During this fiscal year, the Company also plans to file an Investigative New Drug (IND) for a pivotal Phase 3 clinical trial of Revimmune, to treat numerous autoimmune diseases with an initial indication targeting refractory relapsing-remitting Multiple Sclerosis. Revimmune is based on pulsed, ultra-high dosing of a well-known chemotherapeutic agent under a risk management program. Additionally, through an investment strategy, the Company has acquired the majority ownership interest in Biovest International, Inc. ("Biovest"), (BVTI.OB) and a royalty interest in Biovest's lead drug candidate, BiovaxID(TM) and any other biologic products developed by Biovest. Biovest is currently conducting a pivotal Phase 3 clinical trial for BiovaxID which is a patient-specific anti-cancer vaccine focusing on the treatment of follicular non-Hodgkin's lymphoma. BiovaxID has been granted Fast Track status by the FDA. In addition to these product candidates, the Company has a specialty pharmaceutical business, which markets products focused on respiratory disease and an analytical consulting business that serves customers in the biopharmaceutical industry. For further information, visit the Company Web site at www.accentia.net.

Forward-Looking Statements

Statements in this release that are not strictly historical in nature constitute "forward-looking statements." Such statements include, but are not limited to, statements about Revimmune(TM) SinuNase(TM), BiovaxID(TM), AutovaxID(TM), CRSFungal Profile(TM) and any other statements relating to products, product candidates, product development programs the FDA or clinical study process including the commencement, process or completion of clinical trials or the regulatory process. Such statements may include, without limitation, statements with respect to the Company's plans, objectives, expectations and intentions and other statements identified by words such as "may," "could," "would," "should," "believes," "expects," "anticipates," "estimates," "intends," "plans" or similar expressions. Such forward-looking statements involve known and unknown risks, uncertainties, and other factors that may cause the actual results of Accentia to be materially different from historical results or from any results expressed or implied by such forward-looking statements. These factors include, but are not limited to, risks and uncertainties related to the progress, timing, cost, and results of clinical trials and product development programs; difficulties or delays in obtaining regulatory approval for product candidates; competition from other pharmaceutical or biotechnology companies; and the additional risks discussed in filings with the Securities and Exchange Commission. All forward-looking statements are qualified in their entirety by this cautionary statement, and Accentia undertakes no obligation to revise or update this news release to reflect events or circumstances after the date hereof. The product names used in this statement are for identification purposes only. All trademarks and registered trademarks are the property of their respective owners.


Source: Accentia Biopharmaceuticals

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