Roche's Risdiplam Meets Primary Endpoint In Infants Study

Roche Holdings (OTC:RHHBY) announced positive top-line results on pipeline candidate, risdiplam, for the treatment of spinal muscular atrophy (SMA) from part 2 of the FIREFISH study.

The study is evaluating risdiplam in infants aged one to seven months with type 1 SMA. Risdiplam is an investigational survival motor neuron-2 (SMN2) splicing modifier, designed to increase and sustain SMN protein levels throughout the central nervous system and in peripheral tissues.

The primary outcome measure of the study was the proportion of infants sitting without support for at least five seconds at month 12 of treatment, assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III).

Risdiplam met the primary endpoint by demonstrating statistically significant and medically meaningful motor milestone improvement in infants with Type 1 SMA.

FIREFISH is a two-part, open-label, pivotal study in infants aged one to seven months with type 1 SMA. Part 1 (n=21) assessed the safety profile of the drug and determined the dose for Part 2. Part 2 (n=41) assessed efficacy as measured by the proportion of infants sitting without support after 12 months of treatment or longer.

We remind investors that Roche leads the clinical development of risdiplam as part of a collaboration with the SMA Foundation and PTC Therapeutics (NASDAQ:PTCT) .

The drug is being evaluated in a broad clinical trial program in newborn babies to 60-year olds with SMA. The study also includes patients previously treated with SMA-targeting therapies.

Apart from FIREFISH, the company has three ongoing studies — SUNFISH, JEWELFISH and RAINBOWFISH.

In November 2019, Roche announced positive results from Part 2 of SUNFISH, evaluating the efficacy and safety of risdiplam in patients aged two to 25 years with Type 2 or 3 SMA.

In the same month, the FDA granted priority review to the drug. A decision for approval is expected by May 24, 2020.

The successful development of risdiplam will significantly benefit Roche. However, competition is stiff in the SMA market from the likes of Biogen’s (NASDAQ:BIIB) Spinraza and Novartis’ (NYSE:NVS) gene therapy, Zolgensma.

Roche’s shares have gained 32.8% in the past year compared with the industry's 17.3% growth.

The company’s efforts to develop its portfolio beyond oncology are encouraging. The company has more than a dozen investigational medicines in clinical development for diseases like multiple sclerosis, SMA, neuromyelitis optica spectrum disorder, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease, Duchenne muscular dystrophy and autism.

The stock currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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