Tekmira Pharmaceuticals Corporation (TSX:TKM) announced that it has initiated a Phase 1 human clinical trial for ApoB SNALP. ApoB SNALP, Tekmira’s lead RNAi therapeutic product candidate, is being developed as a treatment for patients with elevated low-density lipoprotein (LDL) cholesterol, or “bad” cholesterol, who are not well served by current therapy. ApoB SNALP is designed to reduce the production of apolipoprotein B (ApoB), a protein produced in the liver that plays a central role in cholesterol metabolism.

Dr. Mark J. Murray, Tekmira’s President and CEO, said “We are very excited to initiate the ApoB SNALP Phase 1 clinical trial representing our first RNAi therapeutic clinical trial and an important milestone for Tekmira. Our preclinical data package supports ApoB as an excellent target for LDL cholesterol lowering and we believe ApoB SNALP is the most advanced RNAi therapeutic targeting a metabolic condition. We expect to complete the Phase 1 clinical trial in early 2010.”

The Phase 1 clinical trial will evaluate the safety, tolerability and pharmacokinetics of escalating single doses of ApoB SNALP in approximately 30 patients with elevated LDL cholesterol. Each dosing cohort will include four patients; three patients will receive ApoB SNALP and one patient will receive a placebo. The trial is also designed to provide preliminary data on the ability of ApoB SNALP to lower serum LDL cholesterol levels. Patients whose LDL cholesterol is reduced by greater than 15% from baseline will be followed until their LDL cholesterol levels return to baseline.

Tekmira’s therapeutic approach is to target ApoB, a protein synthesized in the liver that is essential to the assembly and secretion of very low density lipoprotein (VLDL), a precursor to LDL, both of which are required for the transport and metabolism of cholesterol. ApoB SNALP consists of small interfering RNA (siRNA), designed to silence ApoB, encapsulated in a SNALP formulation. ApoB SNALP is delivered with high efficiency into the liver hepatocytes, the cells which produce ApoB, where the siRNA acts to silence the mRNA coding for ApoB protein resulting in a decrease in circulating VLDL and LDL.

The therapeutic activity of ApoB SNALP has been demonstrated in preclinical models of high cholesterol. Rodents fed a high fat diet demonstrate a 50-100% increase in total cholesterol in the blood. A single ApoB SNALP treatment can overcome such diet-induced high cholesterol, returning blood cholesterol levels to normal. The suppressive effects of a single ApoB SNALP dose lasts for several weeks in preclinical models of high cholesterol.

About RNAi and SNALP

RNAi drugs have the potential to treat human diseases by “switching-off” disease causing genes. The technology, representing one of the most promising and rapidly advancing frontiers in biology and drug discovery, was awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi drugs, such as siRNA, require delivery technology to be administered systemically. In preclinical studies, Tekmira’s SNALP (stable nucleic acid-lipid particles) technology has been shown to be a safe and effective way to deliver RNAi drugs to disease sites. Tekmira believes it has a leading intellectual property position in the field of siRNA delivery.

Source
Tekmira Pharmaceuticals Corporation

WASHINGTON, July 6 — Pemetrexed (Alimta) has been approved for use as maintenance therapy in patients with advanced non-small cell lung cancer who have stable disease after standard chemotherapy, according to the FDA.

Today’s action marks the first time the FDA has approved a drug for maintenance therapy in patients with stable NSCLC.

In a phase III trial, pemetrexed increased overall survival by about three months in patients who had not progressed on platinum-based therapy. Those data were reported in May at the annual meeting of the American Society of Clinical Oncology. (ASCO: Maintenance Pemetrexed Extends NSCLC Survival by Three Months)

But, as in other trials of pemetrexed, no benefit was observed in patients with squamous cell cancer while patients with nonsquamous cell NSCLC survived almost 16 months on average, compared to 10.3 for the placebo group.

Richard Pazdur, MD, director of the Office of Oncology Drug Products in the FDA’s Center for Drug Evaluation and Research, said the drug “represents a new approach in the treatment of advanced non-small cell lung cancer. Typically, patients whose tumors respond to chemotherapy do not receive further treatment after four-to-six chemotherapy cycles.”

Pemetrexed was initially approved in 2004 to treat mesothelioma, but was later approved for treatment of patients with NSCLC whose condition worsened on prior chemotherapy drugs and as an initial therapy for advanced NSCLC.

Reported side effects included damage to blood cells, fatigue, nausea, loss of appetite, tingling or numbness in the hands and feet, and skin rash.

Pemetrexed is marketed by Lilly.

Related Article(s):

• ASCO: Maintenance Pemetrexed Extends NSCLC Survival by Three Months

The Royal Pharmaceutical Society of Great Britain (RPSGB) has received a good performance
review from the Council for Healthcare Regulatory Excellence (CHRE), the health professions’
watchdog.

The independent report showed that the Society successfully met the required level of
performance in all of its regulatory duties during the 2008 /2009 period, which it noted was a
time of significant organisational change.

It also acknowledged the progress being made on the formation of the General Pharmaceutical
Council (GPhC) and said; “We are impressed by the clear evidence we have seen of the
RPSGB learning from the work of other regulators to enable it to improve its own
performance.”

Published today, the CHRE reports on the performance of all nine health professions
regulators in the UK - looking at how each regulator functions and measuring their
performance against agreed standards.

Wendy Harris, the Society’s Deputy Registrar & Director of Regulation, said:

“The Society’s regulatory teams must be congratulated on such a positive report. Although we
have been and continue to work through a period of organisational upheaval, the Society has
stayed committed to maintaining efficient and effective regulatory standards.
“There is still a lot of preparatory and detailed work to be achieved before the GPhC opens its
doors but we will continue to maintain our ‘business as usual’ approach until this transition
occurs.”
In future, the GPhC will be subject to the annual CHRE performance review and not the
RPSGB.
The CHRE Performance Review for 2008 / 2009 is available in full from the CHRE website,
http://www.chre.org.uk.

The Council for Healthcare Regulatory Excellence (CHRE)

CHRE is an independent statutory body covering all of the United Kingdom. It is accountable to
the Westminster parliament. It was established by parliament in 2003 to ensure consistency
and good practice in healthcare regulation.

The CHRE’s primary purpose is to promote the health, safety and wellbeing of patients and the
public. It scrutinises and oversees the health professions regulators, works with them to
identify and promote good practice in regulation, carries out research, develops policy and
gives advice.

The CHRE governs the following nine UK regulatory bodies:

- General Medical Council (GMC)
- Nursing and Midwifery Council (NMC)
- Health Professions Council (HPC)
- General Dental Council (GDC)
- General Optical Council (GOC)
- General Chiropractic Council (GCC)
- General Osteopathic Council (GOsC)
- Royal Pharmaceutical Society of Great Britain (RPSGB)
- Pharmaceutical Society of Northern Ireland (PSNI)

For further information visit the CHRE website, http://www.chre.org.uk.

Source
CHRE