GTC Biotherapeutics (fka GTCB)

[DewDiligence]

PARI and Kamada Announce Positive Intermediate Phase-1 Results for Inhaled Alpha-1 Antitrypsin

[Given that hereditary AAT deficiency affects the lungs, administering replacement AAT by inhalation sounds like a good idea. However, the PARI/Kamada inhaled-AAT program only recently entered phase-1 and we don’t yet know if inhalation will prove to be a safe and effective route of administration. This PR doesn’t offer much help as there are no data to speak of. (For background on Kamada’s injectable AAT program, which is in phase-3, see #msg-10073726.)

Please note that the source material used by PARI/Kamada is plasma rather than recombinant AAT, and hence this drug entails the risks of any plasma-derived protein.]

http://biz.yahoo.com/prnews/070531/aqth128.html?.v=5

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Thursday May 31, 12:33 pm ET

MONTEREY, Calif., May 31 /PRNewswire-FirstCall/ -- The first of two stages of the Phase I clinical trial show positive results for Kamada's Alpha-1 Antitrypsin (AAT) liquid drug candidate for inhalation delivered with PARI's eFlow electronic nebulizer for the treatment of patients suffering from AAT deficiency. Dosage levels studied resulted in good safety profiles and patient tolerability, leading the way to continued drug development of aerosolized AAT delivered with an optimized eFlow.

The study, which was approved by the European Medicines Agency, involved 24 patients who received various doses of inhaled AAT. AAT, also known as Alpha-1 Proteinase Inhibitor (API), is used for chronic replacement therapy in individuals who lack AAT and have an inherited form of panacinar emphysema.

Lack of AAT leads to various health problems including significant reduction in lung function, lung inflammation, shortness of breath, and recurrent exacerbations. This leads to emphysema, which is marked by damage to the walls of the air sacs in the lungs resulting in inefficient breathing and shortness of breath.

"In November 2006, we signed a strategic agreement with Kamada to develop inhaled AAT using our advanced aerosol delivery platform, eFlow [#msg-14954548]. Today, we are pleased that this collaboration is showing good initial results and are optimistic that this partnership will benefit patients in the long term," said Dr. Martin Knoch, President of PARI Pharma GmbH. "By developing a targeted therapy that can be administered directly to the lungs, we believe this could be a substantial improvement and a great example of how the advancements in eFlow are helping to improve drug delivery to the lungs in the future."

AAT is currently used for replacement therapy in the form of weekly intravenous infusions that distribute the medication throughout the bloodstream in order to reach the lungs. An inhaled treatment would offer a more targeted therapy by delivering medication directly to the lungs. Kamada is developing inhaled AAT in partnership with PARI. Kamada also produces and markets an intravenous AAT in several countries and is undergoing Phase III trials in the United States with its intravenous AAT.

Inhaled AAT was designated, both in Europe and in the USA, as an orphan drug for the treatment of congenital emphysema and cystic fibrosis.

About eFlow

eFlow, an electronic, portable nebulizer, enables extremely efficient aerosolization of liquid medications via a vibrating, perforated membrane. Compared to other nebulizer systems, eFlow can produce aerosols with a very high density of active drug, a precisely defined droplet size, and a high proportion of respirable droplets delivered in the shortest possible period of time. Combined with its silent mode of operation, small size (it fits in the palm of your hand), light weight, and battery use, eFlow helps reduce the burden of taking daily inhaled treatments. For additional information please see http://www.paripharma.com.

About Kamada

Kamada is a biopharmaceutical company engaged in development, production, and marketing of high quality, ready to use, plasma therapeutics. Kamada, Ltd. is based at Kiryat Weizmann Science Park, Ness Ziona, Israel. Online at http://www.kamada.com

About PARI Pharma

PARI Pharma focuses on the development of aerosol delivery devices and therapies. Based on PARI's 100-year history working with aerosols, PARI Pharma develops treatments for pulmonary and nasal administration optimized to advanced delivery platforms, such as eFlow.

PARI Pharma provides comprehensive inhalation drug development, including nebulizer formulation development and optimization, CMC, analytics, aerosol characterization, clinical protocol development, and regulatory guidance. PARI Pharma has several clinical development programs ongoing, either partnered or on its own. PARI Pharma, a PARI Medical Holding company, is located in Munich, Germany with a major presence in the United States. Online at http://www.paripharma.com
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