CRISPR Therapeutics AG CRSP, Syndax Pharmaceuticals, Inc. SNDX and KalVista Pharmaceuticals, Inc. KALV were among the early movers in the biopharma space Tuesday.
CRISPR Rises On Gene Editing Therapy Pact With Vertex: CRISPR announced an amended collaboration agreement with Vertex Pharmaceuticals Incorporated VRTX with respect to CTX001, an investigational CRISPR/Cas9-based gene editing therapy that is being developed as a potentially curative therapy for sickle cell disease and transfusion-dependent beta-thalassemia.
The amended agreement provides for Vertex leading the programs and expending 60% of the program costs. In return, the company will receive 60% of profits from future sales, a 10% increase from what was envisaged in the previous agreement.
Meanwhile, CRISPR will assume responsibility for 40% of costs, and receive 40% of profits and a $900 million upfront payment, with potential for a $200 million payment upon first regulatory approval of CTX001.
CRISPR shares were adding 7.14% to $123.25.
KalVista's Study Placed On Hold KalVista faces a setback as the FDA has placed its Phase 2 clinical trial of KVD824 on hold. The investigational asset is an oral product candidate being developed for prophylactic treatment of hereditary angioedema.
The FDA letter did not seek additional studies or new data as a precondition for initiating a Phase 2 study, but has requested further information and analysis related to certain preclinical studies of KVD824, according to KalVista.
The regulatory agency also proposed refinements to the intended KVD824 Phase 2 study protocol.
KalVista shares were sliding 13.49% to $20.
Syndax Moves On Data Readout Syndax announced updated positive data from the Phase 1 dose-escalation portion of the Phase 1/2 study of SNDX-5613 in patients with genetically-defined leukemia.
The company said robust clinical activity, including multiple complete responses with no evidence of minimal residual disease, were observed in heavily pretreated patients.
"Data reported today further support the potential for SNDX-5613 to induce clinically meaningful responses in patients with genetically-defined acute leukemias," said Briggs Morrison, CEO of Syndax.
The company said it has identified the recommended Phase 2 dose and hopes to commence the pivotal Phase 2 study by the end of the second quarter.
The investigational asset was also found to be well tolerated, with the only grade 3 adverse events being QT prolongation, anemia, and differentiation syndrome.
Syndax was seen moving 7.69% to $17.89.
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