Alnylam Initiates Phase IIb Clinical Trial of ALN-RSV01 in Adult Lung Transplant Patients Infected with Respiratory Syncytial Virus (RSV) Infection

– Primary Endpoint is Reduction in Incidence of New or Progressive Bronchiolitis Obliterans Syndrome (BOS), a Life-Threatening Complication of RSV Infection in Lung Transplant Patients –

CAMBRIDGE, MA, USA I February 22, 2010 I Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY - News), a leading RNAi therapeutics company, announced today that it has initiated a Phase IIb trial in adult lung transplant patients with ALN-RSV01, an RNAi therapeutic for the treatment of respiratory syncytial virus (RSV) infection. RSV infection in lung transplant patients represents an important unmet medical need; the condition is associated with significant morbidity, including the development of acute lung transplant rejection in 10% to 20% of infected patients. Lung transplant patients infected with RSV are also at risk for an increase in frequency of new or progressive bronchiolitis obliterans syndrome (BOS), a life-threatening complication representing an irreversible disease of the transplanted lung resulting in approximately 50% mortality within three to five years of onset.

“RSV is a major infectious disease in both adult and pediatric populations and there are no effective treatments used widely today. It is an especially dangerous infection in immuno-suppressed patients, such as lung transplant patients,” said Akshay Vaishnaw, M.D., Ph.D., Senior Vice President, Clinical Research at Alnylam. “Alnylam’s new Phase IIb study aims to repeat and extend the results we saw in our previous Phase IIa study in the same patient population. In that small study, treatment with ALN-RSV01 fulfilled the primary study objective of safety and tolerability, and showed preliminary evidence for improved recovery of lung function and a statistically significant reduction in the incidence of new or progressive BOS.”

The Phase IIb trial is a multi-center, global, randomized, double-blind, placebo-controlled study in RSV-infected lung transplant patients with a primary endpoint of a reduction in incidence of new or progressive BOS. Secondary endpoints include assessments for safety and additional measurements of efficacy, including: anti-viral activity; recovery of lung function, as monitored by the proportion of patients with forced expiratory volume in the first second (FEV1), of greater than 80% of their pre-infection baseline value; and, improvement in RSV symptoms as measured by mean cumulative daily total symptom score. The trial is expected to enroll 76 patients who will be randomized in a one to one, drug to placebo ratio. All patients will receive standard of care, and those receiving ALN-RSV01 will have drug administered as a 0.6 mg/kg dose by inhalation using an investigational eFlow Nebulizer System (PARI Pharma) once daily for five days.

“RSV infection represents a significant risk for lung transplant patients, and pulmonologists are clearly in need of an effective RSV therapy in this critical disease area. Morbidities associated with RSV infection in this setting are significant due to the potential for acute and chronic lung rejection and other complications,” said Professor Allan R. Glanville, Director of Thoracic Medicine and Medical Director Lung Transplantation, St. Vincent’s Hospital, Sydney. “I am encouraged by the potential for ALN-RSV01 to treat RSV infection, and I look forward to working with Alnylam in developing this RNAi therapeutic for the treatment of RSV infection in lung transplant patients.”

Results from previous pre-clinical and clinical studies with ALN-RSV01 have documented the drug’s safety profile and anti-viral activity. In 2009, Alnylam completed a Phase IIa study of ALN-RSV01 in RSV-infected adult lung transplant patients. In this small study of 24 patients, ALN-RSV01 was safe and well tolerated, which was the primary study objective. Interpretation of secondary study objectives, including anti-viral activity, was confounded by certain imbalances, for example baseline viral loads, that occurred by chance. By day 14, there was a greater reduction in cumulative symptoms scores in the ALN-RSV01 group. At the 90 day endpoint, ALN-RSV01 treatment was associated with evidence for improved recovery of lung function and a statistically significant reduction in the incidence of new or progressive BOS. In 2008, the anti-viral activity of ALN-RSV01 was demonstrated in the company’s Phase II GEMINI study, which was a randomized, double-blind, placebo-controlled study of intranasal ALN-RSV01 in 88 adult volunteers experimentally infected with RSV. In this study, ALN-RSV01 treatment was associated with a statistically significant decrease in the incidence of RSV infection as compared with placebo. Finally, pre-clinical studies have documented the anti-viral activity of ALN-RSV01 in rodent models (Alvarez et al., Antimicrob Agents Chemother. 53(9):3952-62, 2009).

The ALN-RSV program is partnered with Kyowa Hakko Kirin Co., Ltd. in Asia, and Cubist Pharmaceuticals, Inc. worldwide except Asia. In parallel with Alnylam’s development of ALN-RSV01 for the treatment of RSV in lung transplant patients, Alnylam and Cubist are developing a second-generation compound, ALN-RSV02, which will be focused on the pediatric patient population. Cubist will take the lead in advancing ALN-RSV02 in the pediatric setting in continued collaboration and 50-50 funding with Alnylam, and Cubist retains an opt-in right for ALN-RSV01 in the adult transplant indication.

About RNA Interference (RNAi)

RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and represents one of the most promising and rapidly advancing frontiers in biology and drug discovery today which was awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNAs (siRNAs), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, target the cause of diseases by potently silencing specific mRNAs, thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way.

About Alnylam Pharmaceuticals

Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is applying its therapeutic expertise in RNAi to address significant medical needs, many of which cannot effectively be addressed with small molecules or antibodies, the current major classes of drugs. Alnylam is leading the translation of RNAi as a new class of innovative medicines with peer-reviewed research efforts published in the world’s top scientific journals including Nature, Nature Medicine, and Cell. The company is leveraging these capabilities to build a broad pipeline of RNAi therapeutics; its most advanced program is in Phase II human clinical trials for the treatment of respiratory syncytial virus (RSV) infection. In addition, the company is developing RNAi therapeutics for the treatment of a wide range of disease areas, including liver cancers, TTR-mediated amyloidosis (ATTR), hypercholesterolemia, and Huntington’s disease. The company’s leadership position in fundamental patents, technology, and know-how relating to RNAi has enabled it to form major alliances with leading companies including Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, and Cubist. Alnylam and Isis are joint owners of Regulus Therapeutics Inc., a company focused on the discovery, development, and commercialization of microRNA-based therapeutics. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information, please visit www.alnylam.com.

Alnylam Forward-Looking Statement

Various statements in this release concerning Alnylam’s future expectations, plans and prospects including without limitation statements concerning the timing and scope of clinical trials and studies, the need for novel RSV therapeutics, and its views with respect to the potential for RNAi therapeutics, including ALN-RSV01, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including risks related to: the difficulty drawing conclusions from the small sample size of patients in the Phase IIa study described in this press release; the difficulty drawing conclusions in light of the confounding differences in certain baseline characteristics between those patients taking ALN-RSV01 and those patients taking placebo in the Phase IIa study; the possibility that ALN-RSV01 may not demonstrate the same results in the Phase IIb study as it did in the Phase IIa study; Alnylam’s approach to discover and develop novel drugs, which is unproven and may never lead to marketable products, including ALN-RSV01 or any successor product thereto; obtaining, maintaining, and protecting intellectual property; obtaining regulatory approval for the clinical development and commercialization of products in different jurisdictions; competition from others using technology similar to Alnylam’s and others developing products for similar uses; Alnylam’s dependence on collaborators; and Alnylam’s short operating history; as well as those risks more fully discussed in the “Risk Factors” section of its most recent quarterly report on Form 10-Q on file with the Securities and Exchange Commission. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam does not assume any obligation to update any forward-looking statements.

SOURCE: Alnylam Pharmaceuticals

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