FDA’s Ok to Roche’s Oral SMA Therapy; Roche’s Etrolizumab Mixed Results in Ulcerative Colitis; Secura receives rights to Copiktra from Verastem

Roche Set to Compete With Biogen and Novartis in the SMA Market With its First Oral Therapy 

The US FDA has recommended the use of Evrysdi (risdiplam), a candidate of Genentech, a Roche company, for spinal muscular atrophy (SMA), an inherited neuromuscular disease resulting in muscle atrophy, in adults and children two months of age and older.

Evrysdi is orally administered and was evaluated in a broad clinical trial program with patients range from birth to 60 years old, including difficult-to-treat populations whose disease had progressed. It is a survival motor neuron-2 (SMN-2) splicing modifier, designed to increase and sustain SMN protein levels throughout the central nervous system and peripheral tissues. It is under investigation in two clinical studies for Types 1, 2, and 3 SMA. The drug showed improvement in both the trials. The drug improved survival without permanent ventilation at 12 and 23 months compared to natural history.

Before Evrysdi, Biogen’s Spinraza and Novartis’ Zolgensma are already approved and commercially available in the Spinal muscular atrophy market; however, it’s comparatively lower price and oral administration route will benefit the drug to expand in the SMA market. 

Roche’s Gut Disease Drug, Etrolizumab Presents Muddled Results in Ulcerative Colitis Trials 

Genentech has been running a major clinical trial program, studying its first-ever investigational therapy, Etrolizumab, to treat Ulcerative Colitis. The results from the trials evaluating Etrolizumab as induction therapy and as maintenance therapy posted by the company appeared mixed.

The results from the Phase III HIBISCUS I induction study, etrolizumab met the primary endpoint in patients without prior anti-tumor necrosis factor (anti-TNF) treatment; however, in the HIBISCUS II induction study, which also included people without prior anti-TNF treatment, etrolizumab failed to meet its primary endpoint.

In the HICKORY study, in people with prior anti-TNF treatment, etrolizumab managed to meet the primary endpoint at induction, but at maintenance, it failed. In the LAUREL maintenance study in people without previous anti-TNF treatment also etrolizumab was unable to meet its primary endpoint.

Etrolizumab is an antibody engineered to block two members of a family of proteins called integrins to stop immune overreaction by preventing the WBCs to enter into the gut.  

Secura Bio Secures the Global Commercial and Development Rights to Copiktra from Verastem

Verastem, a biopharma company advancing cancer treatments, has signed a definitive agreement with Secura Bio to sell its global commercial and development rights to COPIKTRA (duvelisib). Moreover, Secura also has received rights to Verastem’s oral inhibitor of phosphoinositide 3-kinase (PI3K), and the first FDA-approved dual inhibitor of PI3K-delta and PI3K-gamma. 

Under the terms of the agreement, Verastem will receive an up-front amount of USD 70 Million, with a total deal value of up to USD 311 Million and royalties on sales. For smoother transactions, Secura has taken a USD 70 Million financing by  Athyrium Capital Management. Secura will fund the ongoing trials and projects of Verastem to develop VS-6766 and Defactinib in Low-Grade Serous Ovarian Cancer and KRAS Mutant Non-Small Cell Lung Cancer at least until 2024. 

Secura plans to reinforce the development of Copiktra in the treatment of T-cell lymphomas, and exploit PI3K and HDAC inhibitors for additional value in the trial outcomes.