The specialty drug pipeline is coming off a strong year. A number of FDA approvals in 2019 were either the first new therapies or the first in class approved for certain conditions, and the specialty pipeline is continuing to grow at a rapid pace.1
These recent developments for specialty pharmacy were discussed during a session of the National Association of Specialty Pharmacy (NASP) 2020 Annual Meeting & Expo Virtual Experience. During the session, presenter Ray Tancredi, MBA, RPh, CSP, divisional vice president of specialty pharmacy development and brand Rx/vaccine purchasing at Walgreens, said 27 of the 49 new drugs and biologics approved by the FDA in 2019 were considered specialty products.1
“We saw 59 in 2017 and 63 new drugs in 2018, and there are no signs of slowing. We’re going to see an even greater explosion of drugs over the next few years,” Tancredi said.1
Medications approved in 2019 provided new therapeutic options for hard-to-treat diseases. These first-in-class drugs included darolutamide (Nubeqa; Bayer) for men with nonmetastatic castration-resistant prostate cancer and the oral medication selinexor (Xpovio; Karyopharm Therapeutics), which is indicated in combination with the corticosteroid dexamethasone for treating adult patients with relapsed/refractory multiple myeloma who have received at least 4 prior therapies and whose disease is resistant to several other forms of treatment.1,2
In his presentation, Tancredi also discussed drug products in the oncology pharmacy pipeline for various forms of cancer, such as breast cancer, acute myeloid leukemia (AML), non—small cell lung cancer (NSCLC), and biliary cancer.
“The pipeline is rich with potential oncolytics,” he said.
These investigational products include the following:
- Guadecitabine (Otsuka Pharmaceutical Co, Ltd), a DNA methyltransferase inhibitor for AML and myelodysplastic syndrome with a subcutaneous injection delivery. The drug is in phase 3 development for these indications as well as phase 2 trial stage for ovarian cancer, according to Tancredi.
- Napabucasin (Sumitomo Dainippon Pharma Co, Ltd), an oral, first-in-class STAT3 inhibitor for colorectal cancer. “It targets malignant cancer stem cells that are responsible for malignancy metastatic growth and recurrence,” Tancredi said. “Phase 3 trials are ongoing.”
- Relatlimab (Bristol Myers Squibb), an antilymphocyte activation gene 3 injection for melanoma and renal cell cancer. In phase 3, this drug is also being investigated with other therapies for potential additional oncology indications, Tancredi said.
- Tipifarnib (Kura Oncology), a farnesyltransferase inhibitor for head and neck cancers.1 This investigational drug has been granted fast track designations by the FDA for HRAS-mutant head and neck squamous cell carcinomas and for the treatment of adult patients with relapsed/refractory angioimmunoblastic T-cell lymphoma, follicular T-cell lymphoma, and nodal peripheral T-cell lymphoma with T follicular helper phenotype.3 “Phase 2 trials are ongoing in evaluating the efficacy of head and neck cancers,” said Tancredi.1
- Cerdulatinib (Portola Pharmaceuticals, Inc), an oral, dual-spleen tyrosine kinase and Janus kinase inhibitor4 for non-Hodgkin lymphomas. “Researchers feel that cerdulatinib should be able to induce significantly greater death of cancer cells than any other therapy,” Tancredi said.
- Sitravatinib (Mirati Therapeutics, Inc), an oral multitargeted tyrosine kinase inhibitor in phase 2 for NSCLC. Additionally, sitravatinib is being evaluated in a phase 2 study in combination with nivolumab (Opdivo; Bristol Myers Squibb) for treatment of advanced or metastatic urothelial carcinoma, according to Mirati Therapeutics.5
- Savolitinib (AstraZeneca), an oral, phase 3, MET-tyrosine kinase inhibitor for renal cell carcinoma. In some studies, savolitinib has demonstrated antitumor activity in patients with MET-driven papillary renal cell carcinoma.6
- Zalifrelimab (Agenus Inc), an experimental injection drug for urinary tract cancer that also is being investigated in combination with balstilimab for cervical cancer. “In our ongoing clinical trials, zalifrelimab and the combination of zalifrelimab plus balstilimab (AGEN2034, anti—PD-1 antibody) have delivered durable as well as curative responses in patients across a broad range of tumors,” said Garo H. Armen, PhD, chairman and CEO of Agenus, in a prepared statement.7
- Motixafortide (BL-8040; BioLineRx Ltd), an injection targeting CXCR4 in phase 3 for multiple myeloma. A short synthetic peptide used as a platform for cancer immunotherapy, motixafortide has been shown to affect multiple modes of action in “cold” tumors, including immune cell trafficking, tumor infiltration by immune effector T cells, and reduction in immunosuppressive cells (such as bone marrow—derived suppressor cells) within the tumor niche—turning these cold tumors “hot,” sensitizing them to immune checkpoint inhibitors and chemotherapy.8
In addition to oncology drugs, Tancredi’s presentation at the NASP meeting included a look at some investigational drugs also in the pipeline for rare and orphan conditions, specialty pharmacy, and gene therapy.
- Tancredi R. Specialty pharmaceutical pipeline updates. Presented at: National Association of Specialty Pharmacy 2020 Annual Meeting & Expo Virtual Experience; September 17, 2020.
- FDA approves new treatment for refractory multiple myeloma. News release. FDA. July 3, 2019. Accessed September 18, 2020. https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-refractory-multiple-myeloma
- Kura Oncology receives fast track designation for tipifarnib in T-cell lymphomas. News release. Kura Oncology Inc. March 3, 2020. Accessed October 13, 2020. https://www.globenewswire.com/news-release/2020/03/03/1994222/0/en/Kura-Oncology-Receives-Fast-Track-Designation-for-Tipifarnib-in-T-Cell-Lymphomas.html
- Challenging the status quo. Portola. Accessed October 13, 2020. https://www.portola.com/research/
- Mirati Therapeutics presents phase 2 data on sitravatinib in combination with nivolumab in urothelial cancer at ESMO Virtual Congress. News release. Mirati Therapeutics Inc. September 18, 2020. Accessed October 13, 2020. https://ir.mirati.com/news-releases/news-details/2020/Mirati-Therapeutics-Presents-Phase-2-Data-On-Sitravatinib-In-Combination-With-Nivolumab-In-Urothelial-Cancer-At-ESMO-Virtual-Congress/default.aspx
- Choueiri TK, Heng DYC, Lee JL, et al. Efficacy of savolitinib vs sunitinib in patients with MET-driven papillary renal cell carcinoma: the SAVOIR phase 3 randomized clinical trial. JAMA Oncol. 2020;6(8):1247-1255. doi:10.1001/jamaoncol.2020.2218
- Agenus receives $10M from UroGen Pharma for rights to zalifrelimab (CTLA-4) for intravesical treatment of urinary tract cancers. News release. Agenus Inc. November 11, 2019. Accessed October 13, 2020. https://www.prnewswire.com/news-releases/agenus-receives-10m-from-urogen-pharma-for-rights-to-zalifrelimab-ctla-4-for-intravesical-treatment-of-urinary-tract-cancers-300955231.html
- Motixafortide (BL-8040). BioLineRx. Accessed October 13, 2020. https://www.biolinerx.com/pipeline/bl-8040/overview