Royalty Pharma

Syndax Pharmaceuticals is a clinical-stage biopharmaceutical company based in Waltham, Massachusetts, focused on developing therapies for cancer treatment. The company's lead product, entinostat, is a class I HDAC inhibitor currently in Phase III clinical trials for advanced hormone receptor positive, HER2 negative breast cancer. Additionally, entinostat is being tested in combination with Keytruda and Tecentriq in Phase Ib/II trials for non-small cell lung cancer and metastatic breast cancer. Another significant candidate, SNDX-5613, is a selective inhibitor targeting the Menin-MLL interaction, undergoing Phase I/II trials for acute leukemias. The company is also developing SNDX-6352, a monoclonal antibody for chronic graft versus host disease, currently in Phase I/II trials. Syndax has established collaborations with major pharmaceutical companies and research organizations, enhancing its research and development capabilities. Founded in 2005, Syndax Pharmaceuticals aims to address critical needs in oncology through innovative therapeutic approaches.

Elektrofi is a preclinical-stage company based in Boston, Massachusetts, focused on advancing drug delivery methods to enhance patient experiences. Established in 2016, the company has developed a formulation system capable of processing a diverse array of therapeutic molecules, including large and complex proteins such as monoclonal antibodies, bispecifics, and fusion proteins. Its innovative technology aims to improve subcutaneous administration, allowing drug manufacturers to create breakthrough therapies that empower patients with more control over how they receive essential medications.

Cytokinetics is a late-stage biopharmaceutical company dedicated to the discovery and development of innovative treatments aimed at improving muscle function in patients with debilitating diseases. The company focuses on creating first-in-class muscle activators and next-in-class muscle inhibitors to address conditions characterized by reduced muscle performance, such as amyotrophic lateral sclerosis, heart failure, spinal muscular atrophy, and chronic obstructive pulmonary disease. Utilizing small molecules specifically designed to enhance muscle function and contractility, Cytokinetics aims to advance investigational medicines that have the potential to improve health outcomes for individuals suffering from severe cardiovascular and neuromuscular disorders.

Cytokinetics is a late-stage biopharmaceutical company dedicated to the discovery and development of innovative treatments aimed at improving muscle function in patients with debilitating diseases. The company focuses on creating first-in-class muscle activators and next-in-class muscle inhibitors to address conditions characterized by reduced muscle performance, such as amyotrophic lateral sclerosis, heart failure, spinal muscular atrophy, and chronic obstructive pulmonary disease. Utilizing small molecules specifically designed to enhance muscle function and contractility, Cytokinetics aims to advance investigational medicines that have the potential to improve health outcomes for individuals suffering from severe cardiovascular and neuromuscular disorders.

Cytokinetics is a late-stage biopharmaceutical company dedicated to the discovery and development of innovative treatments aimed at improving muscle function in patients with debilitating diseases. The company focuses on creating first-in-class muscle activators and next-in-class muscle inhibitors to address conditions characterized by reduced muscle performance, such as amyotrophic lateral sclerosis, heart failure, spinal muscular atrophy, and chronic obstructive pulmonary disease. Utilizing small molecules specifically designed to enhance muscle function and contractility, Cytokinetics aims to advance investigational medicines that have the potential to improve health outcomes for individuals suffering from severe cardiovascular and neuromuscular disorders.

Blueprint Medicines is a biopharmaceutical company dedicated to developing targeted therapies for cancers and rare diseases driven by abnormal kinase activation. Founded in 2008 and based in Cambridge, Massachusetts, the company utilizes its proprietary Insights-to-Validation Platform and chemical library to create small molecule kinase inhibitors that address specific genomic drivers. Key drug candidates include avapritinib for systemic mastocytosis and gastrointestinal stromal tumors, BLU-263 for indolent systemic mastocytosis, and pralsetinib for RET-altered cancers such as non-small cell lung cancer and medullary thyroid carcinoma. Additionally, fisogatinib is under investigation for advanced hepatocellular carcinoma, while BLU-782 targets fibrodysplasia ossificans progressiva. Blueprint Medicines collaborates with several pharmaceutical companies to enhance its research and development efforts.

ApiJect Systems is a medical technology company focused on revolutionizing the delivery of injectable medications through its innovative platform for prefilled, single-dose injectors. By utilizing medical-grade plastic resin, ApiJect replaces traditional materials like glass and rubber, streamlining the manufacturing process and significantly reducing environmental impact. The ApiJect Platform enables pharmaceutical and biotech companies to efficiently produce scalable prefilled injectors, either on proprietary Blow-Fill-Seal packaging lines or through partnerships with other manufacturers. This approach not only simplifies the supply chain but also accelerates production times, transforming a multi-step process that typically takes weeks into a rapid, efficient operation. Through these advancements, ApiJect aims to make prefilled injections more affordable and accessible to patients globally.

BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. Oral, once-daily ORLADEYO™ (berotralstat) is approved in the United States for the prevention of HAE attacks in adult and pediatric patients 12 years and older, and under regulatory review for approval in Japan and the European Union. BioCryst has several ongoing development programs including BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases, galidesivir, a potential treatment for COVID-19, Marburg virus disease and Yellow Fever, and BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva. RAPIVAB® (peramivir injection), a viral neuraminidase inhibitor for the treatment of influenza, is BioCryst’s first approved product and has received regulatory approval in the U.S., Canada, Australia, Japan, Taiwan, Korea and the European Union. Post-marketing commitments for RAPIVAB are ongoing.

BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. Oral, once-daily ORLADEYO™ (berotralstat) is approved in the United States for the prevention of HAE attacks in adult and pediatric patients 12 years and older, and under regulatory review for approval in Japan and the European Union. BioCryst has several ongoing development programs including BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases, galidesivir, a potential treatment for COVID-19, Marburg virus disease and Yellow Fever, and BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva. RAPIVAB® (peramivir injection), a viral neuraminidase inhibitor for the treatment of influenza, is BioCryst’s first approved product and has received regulatory approval in the U.S., Canada, Australia, Japan, Taiwan, Korea and the European Union. Post-marketing commitments for RAPIVAB are ongoing.

Biohaven Pharmaceuticals is a clinical-stage biopharmaceutical company dedicated to the discovery, development, and commercialization of innovative treatments for neurological diseases and other therapeutic areas. The company focuses on life-changing therapies targeting conditions such as neuromuscular disorders, metabolic diseases, obsessive-compulsive disorder, and various forms of cancer. Its diverse pipeline includes late-stage product candidates that utilize mechanisms such as calcitonin gene-related peptide receptor antagonists, glutamate modulators, and myeloperoxidase inhibitors. Notable candidates in its pipeline include BHV3000-301, BHV3000-302, and BHV3000-303, among others. Biohaven's commitment to addressing unmet medical needs positions it as a significant player in the biopharmaceutical landscape.

Epizyme, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on the discovery, development, and commercialization of novel epigenetic medicines for cancer and other serious diseases. The company has developed Tazemetostat, an EZH2 inhibitor approved for the treatment of metastatic or locally advanced epithelioid sarcoma in the United States. Additionally, Tazemetostat is being investigated in combination therapies for various cancers, including relapsed follicular lymphoma and high-risk diffuse large B-cell lymphoma, as well as in patients with platinum-resistant solid tumors and castration-resistant prostate cancer. Epizyme also works on other product candidates, such as pinometostat for acute myeloid leukemia and acute lymphoblastic leukemia, along with inhibitors targeting PRMT5 and PRMT1 for different types of cancers. Established in 2007, Epizyme collaborates with several prominent organizations to advance its research and development efforts.