Vertex Pharmaceuticals VRTX announced that it has entered into a strategic collaboration with privately-held biotech, Ribometrix, Inc., for an upfront payment of $20 million to develop RNA-targeted treatments for serious diseases. The upfront payment also includes equity investment by Vertex in Ribometrix.
Ribometrix is focused in discovering small molecule drugs, which modulate RNA function by targeting three-dimensional (3D) RNA structures to treat diseases.
Per the terms of the collaboration, Ribometrix is also eligible to receive milestone payments related to research, development, regulatory and commercialization of more than $700 million. Vertex will also pay royalties on future net global sales for products developed under the collaboration.
The collaboration will give Vertex access to Ribometrix’s proprietary discovery platform to discover and develop up to three therapeutic programs. Initially, Ribometrix and Vertex will undertake two discovery programs. Vertex also has the option to add a third program under this collaboration. Vertex can opt to gain exclusive global license for the development and commercialization of novel therapeutic molecules arising from this collaboration.
So far this year, Vertex,a leader in the CF market, has entered into few other collaborations and made acquisitions to boost and diversify its pipeline into non-CF diseases. Vertex collaborated with another privately-held Kymera Therapeutics in May to develop small molecule protein degraders targeting several serious diseases. The company also expanded its collaboration agreement with CRISPR Therapeutics CRSP in June to include gene editing therapies targeting new disease areas — Duchenne muscular dystrophy (“DMD”) and Myotonic dystrophy type 1 (DM1) — using CRISPR’s technology.
In July, Vertex acquired privately-held Exonics Therapeutics, focused on developing gene editing therapies for Duchenne muscular dystrophy and other severe genetic neuromuscular diseases. Vertex acquired all outstanding shares of Exonics and paid an upfront payment of $245 million. The company is likely to transform Exonics into its subsidiary. In September, Vertex inked an all-cash deal to acquire Semma Therapeutics, a privately-held biotech, for $950 million. This acquisition will add pre-clinical cell-based treatment candidates for type I diabetes to Vertex’s pipeline, which is expected to be completed in the fourth quarter.
Meanwhile, Vertex’s non-CF pipeline, though in early stage, looks interesting with treatments being developed for beta thalassemia, sickle cell disease, alpha-1 antitrypsin deficiency, APOL1-mediated kidney diseases and pain.
Meanwhile, the company’s first triple combination CF regimen — next-generation CFTR corrector VX-445 (elexacaftor) plus tezacaftor and Kalydeco — is under review in the United States. The regulatory submission, seeking approval for the combination regimen, was granted priority review by the FDA with a decision from the regulatory body expected on Mar 19, 2020.
Please note that many other companies like AbbVie ABBV and Proteostasis Therapeutics, Inc. PTI are also developing triple CFTR combinations for CF.
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