Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and Molecular Templates, Inc. (Nasdaq: MTEM; “Molecular Templates” or “MTEM”) today announced that the two companies have entered into a strategic research collaboration to discover and develop novel targeted conditioning regimens that may enhance the hematopoietic stem cell transplant process, including transplants conducted as part of treatment with ex vivo CRISPR/Cas9 gene editing therapies such as CTX001. CTX001 is currently being evaluated in two ongoing Phase 1/2 studies in patients with transfusion-dependent beta thalassemia and severe sickle cell disease, where a hematopoietic stem cell transplant is required as part of treatment with CTX001. The collaboration will seek to discover a new conditioning regimen utilizing MTEM’s engineered toxin body (ETB) platform, which is designed to specifically target and remove specific cells to enable successful engraftment of new cells.
“We believe that gene editing holds significant promise in the treatment of severe hemoglobinopathies such as sickle cell disease and beta thalassemia, and Molecular Templates’ unique technology platform could play an important role in creating a targeted conditioning regimen that could replace chemotherapy currently required in conditioning regimens and thus enhance the overall future treatment experience for patients,” said David Altshuler M.D., Ph.D., Executive Vice President and Chief Scientific Officer at Vertex.
“Vertex has a proven history of employing novel technologies to develop innovative medicines for diseases with high unmet medical needs, making them an ideal partner to expand the use of MTEM’s ETB platform for therapeutics outside of oncology,” said Eric Poma, Ph.D., Molecular Templates’ Chief Executive and Scientific Officer. “MTEM is excited to be working with Vertex to focus on discovering and developing the next generation of targeted conditioning agents. This collaboration further validates the potential of our ETB platform and provides meaningful capital to support continued advancement of our own product pipeline.”
Under the collaboration, MTEM will conduct research activities for the use of ETBs for up to two targets selected by Vertex. The initial research will be focused on discovering a novel conditioning regimen using MTEM’s ETB technology platform. In addition, Vertex has an option to select a second target as part of the collaboration. Upon designation of a clinical development candidate that emerges from the research efforts, Vertex has the option to exclusively license molecules against the designated target. Vertex will have exclusive rights to develop molecules that emerge from the research program for any indication.
Vertex will make an up-front payment of $38 million to MTEM, including an equity investment. MTEM is also eligible to receive future development, regulatory and sales milestones and option payments of up to $522 million (across two targets) and tiered royalty payments on future sales.
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