Rare Disease Report

Roche Buys Small French Company to Develop Drug for Spinal Muscular Dystrophy

JANUARY 20, 2015
James Radke

Swiss Pharma Company Roche will acquire the French privately-held biotech company Trophos to develop olesoxime (TRO19622),  a drug being developed fro spinal muscular atrophy (SMA).
SMA is a highly disabling genetic disease characterized by progressive muscle weakness and loss of motor function. Typically, SMA presents in early childhood and is a common genetic cause of infant mortality.

In a pivotal phase II clinical trial,  olesoxime showed a beneficial effect on the maintenance of neuromuscular function in individuals with Type 2 and non-ambulatory Type 3 SMA.

The pivotal study was conducted in seven European countries. It was a double-blind, placebo-controlled study in 165 type 2  and non-ambulatory type 3 SMA patients, ranging in age from 3 to 25 years old. Patients were randomized to treatment (10 mg/kg olesoxime dosed daily as a liquid oral suspension or matching placebo in a 2:1 ratio).

Results from this pivotal study showed a progressive loss of motor function in the placebo arm, as described for the typical disease progression and similarly documented in other observational and interventional trials. This loss of function, assessed as the primary endpoint, was prevented for two years in olesoxime-treated patients, with fewer disease-related adverse events. Other secondary endpoints were consistent with these effects.

Sandra Horning, M.D., Chief Medical Officer and Head of Global Product Development at Roche  said:

 “We will build on the work done by Trophos and the French Muscular Dystrophy Association to advance the development of olesoxime and to bring it to people who live with this devastating condition as quickly as possible.”

Christine Placet, Chief Executive Officer of Trophos  added:

“SMA is a grievous disease with a huge impact on the daily life of patients and their families, who are currently left only with supportive care. We are proud to see the development of this medicine evolving, with the ultimate goal of a potential first medicine for SMA.”

Under the terms of the agreement, Trophos’s shareholders will receive an upfront cash payment of EUR 120 million, plus additional contingent payments of up to EUR 350 million based on achievement of certain predetermined milestones.

About Spinal Muscular Atrophy (SMA)

SMA is a life-limiting and highly disabling genetic disease characterised by progressive muscle weakness and loss of motor function. SMA affects the motor neurons of the voluntary muscles used for activities such as crawling, walking, head and neck control and swallowing.

Patients with SMA are usually categorized by having one of the four types of the disease, based on severity, the highest level of motor functioning achieved and time of onset:
Type 1: The most severe form of SMA. Symptoms usually emerge within the first six months of life. Affected infants have low muscle tone, profound muscle weakness and impaired ability to move. Babies with type 1 SMA never sit. Simple tasks, such as holding up their heads, feeding and swallowing, can be very difficult. Progressive weakness of chest muscles increases the risk of respiratory infections and poor lung growth. Babies with type I SMA are at very high risk of irreversible decline in respiratory capacity. Type 1 SMA carries a high mortality rate, with more than half of all affected children not surviving beyond two years of age.
Type 2: Intermediate form of SMA. Symptoms usually emerge between six and 18 months of age. Individuals with Type 2 SMA typically are able to sit, but cannot walk, have severe and progressive motor disability and often require care 24 hours a day for their whole life. Individuals with Type 2 often develop severe curvature of the spine (scoliosis) and weakness of the chest muscles leading to high risk of severe respiratory infections. The severity and progression of the disease varies from person to person, life expectancy ranges from early childhood to adulthood.
Type 3: Symptoms can emerge anywhere between 18 months and early adulthood and include difficulty walking, muscle weakness and an increased risk of respiratory infections. A significant number of people with Type 3 SMA lose the ability to walk and can also develop severe scoliosis and other orthopaedic problems. Many patients become non-ambulatory and are wheelchair bound at the age of 40.
Type 4: Adult form of SMA. A less common form of SMA, this type affects adults and is characterised by a slower progression of symptoms that mainly affect the ability to walk. Symptoms typically emerg after the age of 35 and patients have a normal life expectancy.

Stay informed on the latest rare disease news and developments by signing up for our newsletter.
Copyright © RareDR 2013-2018 Rare Disease Communications. All Rights Reserved.