January 22, 2009

Find selected press releases below

Rheumatoid arthritis - Anti-TNFs defend first-line biologic position despite competition

This report forecasts that total sales for the seven major rheumatoid arthritis markets will grow from $7 billion in 2007 to over $12 billion in 2017. The sustained uptake of anti-TNFs combined with the launch of several new biologic products will drive growth in the short-and mid-term. However, biosimilar entry will cause a small contraction in market value at the end of the forecast period. This report provides:

1. in-depth analysis of the current and future rheumatoid arthritis market across the US, 5EU and Japanese markets, and a 'rest of world' snapshot;
2. rheumatoid arthritis sales forecasts for over 20 key brands to 2017, and total brand figure to benchmark indication-specific sales;
3. detailed brand analysis, including discussion of all anti-TNFs and impact of alternative biologic products like Actemra; and
4. an assess ent of the strategies of several key developers in the rheumatoid arthritis market.

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1. Positive phase I Duchenne’s muscular dystrophy trial of AVI-4658
2. PharmaSurgics enters phase I with peptide product to prevent scarring
3. Neurologix enters phase II gene therapy trial for Parkinson’s disease
4. MHRA clears CTX of stroke triawithof ReNeuron’s neural stem cells
5. Myriad acquires HIV maturation inhibitor from Panacos
6. Positive phase II results of Aradigm’s inhaled liposomal ciprofloxacin
7. Richter-Helm BioLogics and Athera Biotechnologies signed an agreement for the development and manufacturing of a plaque rupture and athero-thrombosis prevention product
8. S*BIO licenses multi-kinase inhibitor to Targanta
9. FDA advises on pivotal trial design of SG’s anti-CD30-drug conjugate

1.- Positive phase I Duchenne’s muscular dystrophy trial of AVI-4658

AVI BioPharma announced results from a Phase 1 trial of its drug candidate AVI-4658 for the treatment of Duchenne muscular dystrophy (DMD) by exon skipping. Biopsy data showed that injection of the drug into the muscles of a series of DMD patients successfully induced dystrophin production in each patient. DMD is an incurable muscle-wasting disease associated with errors in the gene that makes dystrophin, a protein that plays a key structural role in muscle fiber function. The drug candidate AVI-4658 is designed to skip exon 51 of the dystrophin gene, allowing for restoration of the reading frame in the mRNA sequence. Restoration of dystrophin production achieved by skipping this exon may improve or significantly slow the disease process, thus prolonging and improving the quality of life for the affected patient population. Read more

2.- PharmaSurgics enters phase I with peptide product to prevent scarring

PharmaSurgics has received approval from the Swedish medical product agency to proceed to phase I clinical documentation with the novel candidate drug, PXL01, indicated for post-surgical adhesion prevention. Adhesions are bands of scar tissue that connect anatomic structures that should not normally be connected. These develop when the body's repair mechanisms respond to tissue injury as the result of surgical trauma. Adhesions form after almost any type of surgery and are a significant source of post-surgical complications for millions of patients, a major burden for surgeons and a great economic load on the healthcare systems. Currently the problem does not have any satisfying solution and there is a great need for new products. Read more

3.- Neurologix enters phase II gene therapy trial for Parkinson’s disease

Neurologix announced that it has initiated its Phase 2 clinical trial for the treatment of advanced Parkinson’s disease. The first trial participants have undergone surgery at multiple institutions and additional subjects are currently being enrolled. The purpose of the trial is to validate the safety and efficacy of Neurologix’s gene transfer therapy, a novel non-dopaminergic approach to restore motor function in Parkinson’s patients who are sub-optimally responsive to available drug therapy. Neurologix’s approach is to reestablish the production of GABA (gamma-aminobutyric acid), the major brain inhibitory neurotransmitter that helps “quiet” excessive neuronal firing and has been determined to be deficient in patients in the advanced stages of Parkinson’s disease. In Parkinson’s disease, patients lose dopamine-producing brain cells, resulting in substantial reductions in the activity and amount of GABA (gamma-aminobutyric acid). This reduction in GABA causes a dysfunction in brain circuitry responsible for coordinating movement. Read more

4.- MHRA clears CTX of stroke triawithof ReNeuron’s neural stem cells

ReNeuron announced that it has received approval from the UK Medicines and Healthcare Products Regulatory Agency (MHRA) to commence a first-in-man clinical trial for the treatment of patients who have been left disabled by an ischaemic stroke, the most common form of the condition. Stroke is the third largest cause of death and the single largest cause of adult disability in the developed world. ReNeuron's ReN001 cell therapy for stroke consists of a neural stem cell line, designated CTX, which has been generated using the Company's proprietary cell expansion and cell selection technologies and then taken through a full manufacturing scale-up and quality-testing process. As such, ReN001 is a standardised, clinical and commercial-grade cell therapy product capable of treating all eligible patients presenting. ReN001 has been shown to reverse the functional deficits associated with stroke disability when administered several weeks after the stroke event in relevant pre-clinical models of the condition. Extensive pre-clinical testing also indicates that the therapy is safe, with the ReN001 cells eventually cleared from the body with no adverse safety effects arising. Read more

5.- Myriad acquires HIV maturation inhibitor from Panacos

Myriad Pharmaceuticals announced that it has acquired all rights to bevirimat from Panacos Pharmaceuticals. Bevirimat has demonstrated potent activity against a broad range of HIV strains, and laboratory studies have shown bevirimat to be an inhibitor of HIV isolates that are resistant to currently approved HIV drugs. Bevirimat is currently in Phase 2b clinical studies using a new tablet formulation that has an oral bioavailability and pharmacokinetic profile comparable to the previous solution formulation. Read more

6.- Positive phase II results of Aradigm’s inhaled liposomal ciprofloxacin

Aradigm announced positive top-line results from an open-label study of efficacy, safety and tolerability with its once daily inhaled liposomal ciprofloxacin hydrochloride (ILCH) in patients with non-cystic fibrosis bronchiectasis. This orphan drug condition is a chronic severe respiratory disease and there is currently no drug specifically approved for its treatment in the US. The primary efficacy endpoint was the change from baseline in the sputum Pseudomonas Aeruginosa colony forming units (CFU), the standard objective measure of the reduction in pulmonary bacterial load. The two evaluated doses of ILCH in the evaluable patient population demonstrated significant mean decreases against baseline in the Pseudomonas Aeruginosa CFU over the treatment period of 3.5 log and 4.0 log units, respectively. Bronchiectasis is a chronic condition characterized by abnormal dilatation of the bronchi and bronchioles associated with chronic infection. It is frequently observed in patients with cystic fibrosis. CF is a genetic disease that causes thick, sticky mucus to form in the lungs, pancreas and other organs. In the lungs, the mucus tends to block the airways, causing lung damage and making these patients highly susceptible to lung infections. Ciprofloxacin is a widely prescribed antibiotic to treat infections of the lung frequently experienced by cystic fibrosis patients. Read more

7.- Richter-Helm BioLogics and Athera Biotechnologies signed an agreement for the development and manufacturing of a plaque rupture and athero-thrombosis prevention product

Richter-Helm BioLogics and Athera Biotechnologies have signed an agreement for the development and manufacturing of Athera's novel product for prevention of plaque rupture and athero-thrombosis through binding of the protein, Annexin A5, to endothelium. The recombinant protein Annexin A5 is intended for the treatment of patients with Acute Coronary Syndrome and who are at imminent risk for Myocardial Infarction. This agreement secures a highly cost efficient long-term development and manufacturing plan for Athera, including possible future large volume commercial production. Annexin A5 is a biotechnology product produced using Richter-Helm's proprietary E. coli based expression system. Read more

8.- S*BIO licenses multi-kinase inhibitor to Targanta

S*BIO and Tragara Pharmaceuticals announced that S*BIO has granted a worldwide exclusive license to Tragara to develop and commercialize its novel multi-kinase inhibitor SB1317. SB1317 is a novel orally-available, multi-kinase inhibitor with excellent pharmacological and pharmaceutical properties. SB1317 development will be initially focused on the treatment of hematologic malignancies and Tragara will also explore the therapeutic potential of the compound's activity in solid tumors. Read more

9.- FDA advises on pivotal trial design of SG’s anti-CD30-drug conjugate

Seattle Genetics announced that it has reached agreement with the U.S. Food and Drug Administration (FDA) on a Special Protocol Assessment (SPA) for a pivotal trial of SGN-35, an antibody-drug conjugate (ADC), for patients with relapsed or refractory Hodgkin lymphoma. The SPA provides an agreement between the FDA and Seattle Genetics regarding the design, including size and clinical endpoints, of the pivotal trial to support an efficacy claim in a New Drug Application (NDA). The single-arm pivotal trial will assess efficacy and safety of single-agent SGN-35 in patients with relapsed or refractory Hodgkin lymphoma who previously received autologous stem cell transplant. SGN-35 is an ADC comprising an anti-CD30 antibody attached by an enzyme cleavable linker to a potent, synthetic drug payload, monomethyl auristatin E (MMAE), using Seattle Genetics’ proprietary technology. The ADC is designed to be stable in the bloodstream, but to release MMAE upon internalization into CD30-expressing tumor cells, resulting in a targeted cell-killing effect. Read more

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