Amgen And UCB Announce U.S. FDA Acceptance Of Biologics License Application For Romosozumab

THOUSAND OAKS, Calif. and BRUSSELS, Sept. 26, 2016 /PRNewswire/ -- Amgen (NASDAQ:AMGN) and UCB (Euronext Brussels: UCB) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for romosozumab, an investigational monoclonal antibody for the treatment of osteoporosis in postmenopausal women at increased risk of fracture. Romosozumab works by binding and inhibiting the activity of the protein sclerostin, a protein naturally occurring in the bone, thereby increasing bone formation and decreasing bone resorption.

"We believe romosozumab could serve as an important therapeutic option for osteoporosis patients with an increased risk of fracture," said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. "We look forward to potentially providing a new therapy to address a critical unmet medical need and further supporting patients worldwide."

The BLA, submitted on July 19, 2016, is based on data from the pivotal Phase 3 placebo-controlled FRActure study in postmenopausal woMen with ostEoporosis (FRAME) in approximately 7,200 patients.

"Once a patient suffers a fragility fracture, the burden of osteoporosis can have a tremendous impact on a patient's life,"^1,2 said Dr. Pascale Richetta, head of bone and executive vice president, UCB. "We are pleased with the FDA's acceptance of the BLA filing for review and hope to continue the regulatory process towards U.S. approval to introduce a potential therapy to reduce the risk of fractures for this patient population."

The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of July 19, 2017, for romosozumab.

About Romosozumab
Romosozumab is an investigational bone-forming monoclonal agent and is not approved by any regulatory authority for the treatment of osteoporosis. It is designed to work by inhibiting the activity of the protein sclerostin, and has a dual effect on bone, both increasing bone formation and decreasing bone breakdown. Romosozumab is being studied for its potential to reduce the risk of fractures in an extensive global Phase 3 program. This program includes two large fracture trials comparing romosozumab to either placebo or active comparator in more than 10,000 postmenopausal women with osteoporosis. Amgen and UCB are co-developing romosozumab.

About the FRAME study
FRAME is a multicenter, international, randomized, double-blind, placebo-controlled, parallel-group study in postmenopausal women with osteoporosis, defined as low bone mineral density at the total hip or femoral neck. The study evaluated the effectiveness of romosozumab treatment, compared with placebo, in reducing the risk of new vertebral fractures through 12 months. The study also further evaluated if romosozumab treatment for 12 months followed by denosumab treatment for 12 months, compared with placebo followed by denosumab treatment, was effective in reducing the risk of new vertebral fractures through 24 months. In addition, clinical fracture (a composite endpoint which encompasses all symptomatic fractures, both non-vertebral and painful vertebral fractures) risk reduction, non-vertebral fracture (fractures outside of the spine, excluding sites that are not considered osteoporotic, fractures due to high trauma or pathologic fractures) risk reduction and other endpoints were assessed at 12 and 24 months.

7,180 patients were randomized 1:1 to receive either 210 mg romosozumab subcutaneous (SC) monthly (QM) or placebo SC QM for the 12-month double-blind study period. After the placebo-controlled study period, patients entered the open-label phase where all patients received 60 mg denosumab SC every six months (Q6M) for 12 months, while remaining blinded to initial treatment. An additional 12 month extension period of open-label 60 mg denosumab SC Q6M is currently ongoing.

About Amgen
Amgen is committed to unlocking the potential of biology for patients suffering from serious illnesses by discovering, developing, manufacturing and delivering innovative human therapeutics. This approach begins by using tools like advanced human genetics to unravel the complexities of disease and understand the fundamentals of human biology.

Amgen focuses on areas of high unmet medical need and leverages its expertise to strive for solutions that improve health outcomes and dramatically improve people's lives. A biotechnology pioneer since 1980, Amgen has grown to be one of the world's leading independent biotechnology companies, has reached millions of patients around the world and is developing a pipeline of medicines with breakaway potential.

For more information, visit www.amgen.com and follow us on www.twitter.com/amgen.

About UCB
UCB, Brussels, Belgium (www.ucb.com) is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. With more than 7,700 people in approximately 40 countries, the company generated revenue of € 3.9 billion in 2015. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCB_news

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Moreover, sales may be impacted by international and domestic trends toward managed care and health care cost containment and the reimbursement policies imposed by third-party payers as well as legislation affecting biopharmaceutical pricing and reimbursement.

CONTACT: Amgen, Thousand Oaks 
Kristen Davis, 805-447-3008 (media) 
Kristen Neese, 805-313-8267 (media) 
Arvind Sood, 805-447-1060 (investors)  

CONTACT: UCB, Brussels   
France Nivelle, Global Communications, UCB   
T +32.2.559.9178, [email protected]   
Laurent Schots, Media Relations, UCB   
T+32.2.559.92.64, [email protected]    
Antje Witte, Investor Relations, UCB   
T +32.2.559.94.14, [email protected] 
Isabelle Ghellynck, Investor Relations, UCB 
T+32.2.559.9588, [email protected]  

References:
¹ International Osteoporosis Foundation. The Global Burden of Osteoporosis. What you need to know. Available at:  http://www.iofbonehealth.org/data-publications/fact-sheets/what-you-need-know-about-osteoporosis. Accessed July 14, 2016.
²International Osteoporosis Foundation. Who's at Risk? 2015. Available at: http://www.iofbonehealth.org/whos-risk. Accessed July 15, 2016.

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