Conference Reports
Piper Jaffray 2008
London, UK, 24th-25th June 2008
The ANdAZ Hotel in the heart of London's bustling financial district was the setting for this year's Piper Jaffray European Conference, bringing together industry executives and investors from within the pharmaceutical industry. From the opening day the event was awash with industry disclosures, partnering and investor discussions, and of course, the occasional light conversation over lunch.
The two-day event featured company presentations from numerous pharmaceutical companies large and small, focused on partnering and investment opportunities. In an informative presentation, Belgian biopharmaceutical company TiGenix reported on ChondroCelect (TGX-001), its autologous chondrocyte-based cartilage regeneration therapy, announcing its availability for licensing. ChondroCelect is currently available through a compassionate use programme, with a BLA filing expected in the second half of 2008. Launches in Germany and the Netherlands expected at the end of the year. Also unveiled was data on preclinical candidate, ChondroCelect 3D (TGX-002), a collaboration between TiGenix and Fidia. By combining Fidia's hyaluronic acid scaffold with TiGenix's ChondroCelect, TGX-002 is less invasive in arthroscopic implantation techniques. TiGenis plans to file an IND for a Phase II trial at the end of 2008.
Acambis presented an update on the development of its prophylactic vaccine against Clostridium difficile, ACAM-CDIFF. Originally conceived as an immunogen to produce a hyperimmune globulin passive vaccine, this CdAB toxin-based vaccine has previously shown promise in a Phase I trial. In 50 healthy adults, ACAM-CDIFF was shown to produce antibody responses in all patients, while being well tolerated. In a separate Phase I trial in 48 patients, it showed a 100% seroconversion rate. A further proof-of-concept Phase I trial is planned for the 4th quarter of 2008.
More news from the world of vaccines came from Swedish pharmaceutical company Bavarian Nordic. The company reported on several of its products including its multi-antigen HIV vaccine for prophylactic and therapeutic use, using the companies MVA-BN technology. The vaccine expresses 8 whole or truncated forms of the HIV virus, with the aim of producing a broad immune response. It is currently in a US Phase I/II safety study in HIV patients. In addition, the company discussed its prophylactic measles vaccine. Also based on the MVA-BN technology, the vaccine expresses 3 measles viral antigens. Preclinical studies showed the vaccine to be safe, and immunogenic in newborn animals. In a South African Phase I trial in 30 healthy adults, it boosted levels of antibodies against the measles virus in nearly all subjects. Following this success, the vaccine is set to be evaluated in a Phase I trial in children in the 2nd half of 2008.
In a similar vane, Scandinavian Biopharmaceutical company, NeuroSearch announced updates to its product pipeline. Included in the presentation was data on ACR-16, a dompaminergic stabilizer for the treatment of Huntington's disease (HD). Able to enhance or depress dopaminergic functions depending on the initial level, this compound holds hope for sufferers of this debilitating neurological disorder. Having previously shown promising results in a Phase II study in HD patients, where it significantly improved voluntary movement and gait function in 60 patients, NeuroSearch has initiated dosing in a European randomized, double-blind, placebo-controlled Phase III trial in 420 patients. The trial will evaluate ACR-16 on the primary endpoint of voluntary motor function, as well as effects on cognition and neuropsychiatric symptoms. In addition, NeuroSearch's partner Astellas is conducting a US placebo-controlled Phase Ib study in schizophrenia patients to evaluate the safety and tolerability of several doses of ACR-16. NeuroSearch plans to launch ACR-16 in 2010. Also presented was data on noradrenaline/5-HT reuptake inhibitor, tesofensine. A Phase II proof-of-concept trial (TIPO-1) in 203 obese patients showed a significant reduction in waist circumference over 24 weeks. In addition, weight loss of more than 8% was reported in 83% of patients. A Phase III trial is under preparation, with a dose of 0.5mg. Tesofensine is also in development for Parkinson's disease (PD) and dyskinesia. It is available for licensing.
Also of particular interest was the completion of Neuropharm's European Phase IIa trial for NPL-2005 for the treatment of fragile X syndrome, a neurological disorder, which leads to mental retardation, and behavioural and emotional problems. Results are expected in the 2nd half of 2008. The FMR-1 gene on the X chromosome, thought to have a role in early development is disabled when the short CGG sequence is repeated more than 230 times. The US orphan-designated NPL-2005 targets and reactivates the FMR-1 gene to relieve the symptoms of fragile X. Neuropharm also has a fenobam formulation, NPL-2009 in Phase II trials for the same disease.
This year's Annual European conference was a great success on all fronts, tailored to appeal to all sectors of the pharmaceutical industry. There can be little doubt that with the international esteem of Piper Jaffray firmly behind it, next year's event shows similar promise.
Jonathan Stephens and Paul D'Souza
Pharmaprojects Analysts
