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KPTI vs QURE

Karyopharm Therapeutics Inc. and uniQure N.V.

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Karyopharm Therapeutics Inc.

Karyopharm Therapeutics Inc., a commercial-stage pharmaceutical company, discovers, develops, and commercializes drugs directed against nuclear export for the treatment of cancer and other diseases in the United States. The company discovers, develops, and commercializes novel and small molecule Selective Inhibitor of Nuclear Export (SINE) compounds that inhibit the nuclear export protein exportin 1 (XPO1). Its lead compound, include XPOVIO in combination with bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma; in combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma; and for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL). It also developing Selinexor for treating hematological and solid tumor malignancies, including multiple myeloma, endometrial cancer, myelofibrosis, and DLBCL; and ELTANEXOR for treating Myelodysplastic Neoplasms, as well as verdinexor, KPT-9274, and IL-12 compounds. In addition, the company has license agreement with Menarini Group to develop and commercialize selinexor for human oncology indications; license agreement with Antengene Therapeutics Limited to develop and commercialize selinexor, eltanexor, and KPT-9274 for the treatment and/or prevention of human oncology indications, as well as verdinexor for the diagnosis, treatment, and/or prevention of human non-oncology indications; and distribution agreement for the commercialization of XPOVIO with FORUS Therapeutics Inc. Further, the company has a collaboration with Bristol Myers Squibb company to evaluate novel cereblon E3 ligase modulator agent mezigdomide in combination with Selinexor in patients with relapsed/refractory multiple myeloma. Karyopharm Therapeutics Inc. was incorporated in 2008 and is headquartered in Newton, Massachusetts.

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uniQure N.V.

uniQure N.V. develops treatments for patients suffering from rare and other devastating diseases in the United States. The company offers HEMGENIX that allows people living with hemophilia B to produce factor IX, which can lower the risk of bleeding. Its lead product candidate is AMT-130, a gene therapy candidate, which is in Phase I/II clinical study for the treatment of Huntington's disease. The company also develops AMT-260, which is in Phase I/IIa clinical trial for the treatment of mesial temporal lobe epilepsy; AMT-162, which is in Phase I/IIa clinical trial to treat superoxide dismutase enzyme-amyotrophic lateral sclerosis; and AMT-191, an investigational gene therapy candidate which is in phase I/IIa clinical trial for the treatment of fabry disease. It has a licensing agreement with Apic Bio to develop, manufacture, and commercialize intrathecally administered investigational gene therapy for ALS caused by mutations in SOD-1; and development and commercial supply agreement with CLS Bhering. uniQure N.V. was founded in 1998 and is headquartered in Amsterdam, the Netherlands.

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