Agios Pharmaceuticals Inc., of Cambridge, Mass., and New York’s Royalty Pharma said they sold Agios’ tiered, sales-based royalty rights on worldwide net sales of New York-based Bristol Myers Squibb Co.’s (BMS) Idhifa (enasidenib), as well as its rights to receive up to $55 million in outstanding regulatory milestone payments from BMS to Royalty for $255 million. Agios will continue to co-promote Idhifa and receive reimbursement from BMS under its 2010 collaboration agreement with Celgene Inc., a wholly owned subsidiary of BMS. Agios also retains the right to receive a $25 million payment upon achieving a specified ex-U.S. commercial milestone event. Idhifa is an FDA-approved oral therapy for treating adults with relapsed or refractory acute myeloid leukemia with an isocitrate dehydrogenase-2 mutation.

In initial experiments by Dcprime BV, of Leiden, the Netherlands, and the University of Bergen in developing a humanized immunocompetent mouse model to study the relapse vaccine DCP-001, results showed individual responses were somewhat heterogeneous, the company said. DCP-001 is generated by transforming a leukemic cell, DCOne, into a whole cell-based cancer vaccine. Dcprime said an overall beneficial effect of DCP-001 on DCOneluc tumor volumes compared to saline was observed. In an additional experiment, optimizing both preconditioning of NSGS mice (n=9) and dosing strategies with DCP-001, two doses of DCP-001 (2x106 and 2x105 cells) were compared to saline-treated mice. Again, a clear effect of DCP-001 in reducing DCOneluc tumor volumes, as compared to saline was seen, the researchers said. To establish a humanized mouse model, different mice strains were tested, and NSGS mice provided the best conditions for both human immune reconstitution and growth of a DCOne leukemia reporter cell line DCOneluc. The therapeutic effect of DCP-001 vaccination on the reporter leukemia cell line DCOneluc was evaluated in a cohort of NSGS mice (n=8) either treated with saline or with two consecutive doses of 2x106 DCP-001 cells. DCP-001 is being evaluated proof-of-concept ADVANCE-II study in acute myeloid leukemia patients in complete remission but with persistent measurable residual disease.

Revive Therapeutics Ltd., of Toronto, said it expanded its partnership with the University of Wisconsin-Madison to evaluate novel formulations of psilocybin in a phase I clinical study investigating the therapeutic application of psilocybin for an undisclosed addiction use disorder. R&D at the university focuses on tannin-chitosan composites in the form of thin films, hydrogels and 3D foams. The research will include development of composite formulations, physiochemical characterization of composite materials and rate of psilocybin release from composites. Final formulations will be investigated in preclinical and clinical studies in various diseases and disorders. Revive said it identified tannin-chitosan composite thin films as the lead candidate for the development of a unique delivery platform for therapeutic doses (1mg-20 mg) of psilocybin.

Scopus Biopharma Inc., of New York, said it signed an exclusive, worldwide license to a potentially first-in-class, targeted immuno-oncology gene therapy drug developed at City of Hope, of Los Angeles. The licensed gene therapy drug, CpG-STAT3siRNA, is a highly selective and targeted therapy that silences activity of the STAT3 gene by way of RNA interference while stimulating the TLR9 receptors to activate the body's immune defense to recognize and kill cancer cells, Scopus said. In preclinical testing, the STAT3 inhibitor has reduced growth and metastasis of various preclinical tumor models, including melanoma, colon and bladder cancers, as well as leukemia and lymphoma. A first-in-human phase I trial in B-cell lymphoma patients is expected to begin in the second half of 2020 at City of Hope.

Tonix Pharmaceuticals Holding Corp., of New York, said it will acquire the migraine and pain treatment technologies of Trigemina Inc., of Moraga, Calif., and will assume the license for some of the technologies from Stanford University. The lead asset, TNX-1900 (formerly TI-001, oxytocin solution for intranasal delivery), is a formulation of nasal oxytocin with demonstrated activity in several nonclinical studies in pain, including migraine prophylaxis and neuropsychiatric models and with safety data from non-U.S. studies, Tonix said. TNX-1900 is being studied for prophylaxis of chronic migraine and is based on a formulation of oxytocin, a naturally occurring human hormone that acts as a neurotransmitter in the brain.

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