Recent Breakthrough Therapy Designations
Sponsor	Product	Indication	Supporting Data	Status
Vtesse	VTS-270, a formulation of (2-hydroxypropyl)-beta-cyclodextrin	Rare disease - Niemann-Pick Type C1 disease	Phase I trial, conducted by National Institutes of Health, in which post hoc analyses showed preliminary evidence of overall disease stabilization and improvement in several disease domains compared to a natural history study patient cohort.	Clinical - Phase IIb/III trial underway
Rhythm	Setmelanotide (RM-493), a melanocortin-4 receptor (MC4R) agonist	Rare disease - Pro-opiomelanocortin (POMC) deficiency obesity	Not disclosed.	Clinical - Phase II trial enrolling
AbbVie and Genentech (Roche)	Venetoclax, an inhibitor of the B-cell lymphoma-2 (BCL-2) protein	Cancer - In combination with Rituxan (rituximab) for treatment of relapsed/refractory chronic lymphocytic leukemia	Results from the M13-365 study	Clinical - Combination of venetoclax and rituximab for relapsed/refractory CLL is being further evaluated in the ongoing MURANO (GO28667) study
AstraZeneca	Lynparza (olaparib), an oral poly ADP-ribose polymerase (PARP) inhibitor	Cancer - Monotherapy treatment of BRCA1/2 or ATM gene mutated metastatic castration resistant prostate cancer (mCRPC) in patients who have received a prior taxane-based chemotherapy and at least one newer hormonal agent (abiraterone or enzalutamide)	Results of the TOPARP-A Phase II trial, which found that olaparib monotherapy in mCPRPC may offer substantial improvement over available therapies	Clinical
AbbVie and Genentech (Roche)	Venetoclax, an inhibitor of the B-cell lymphoma-2 (BCL-2) protein	Cancer - In combination with hypomethylating agents for patients with treatment-naïve acute myeloid leukemia (AML) who are ineligible to receive standard induction therapy	Data from untreated patients, 65 years or older with AML and reported in one investigational study	Clinical
Allergan	Rapastinel (GLYX-13), intravenous formulation of a novel NMDA receptor partial agonist	Psychiatric - Adjunctive treatment of major depressive disorder (MDD)	Phase II evidence demonstrating a rapid onset of efficacy (within 1 day) in MDD patients who had an inadequate response to one or more antidepressants	Clinical - Phase III trials expected to begin in 2016
Immunomedics	Sacituzumab govitecan (IMMU-132), an antibody-drug conjugate	Cancer - Treatment of patients with triple-negative breast cancer (TNBC) who have failed at least 2 prior therapies for metastatic disease	Phase II study in patients with metastatic TNBC who had received a median of 5 prior therapies	Clinical - FDA Special Protocol Assessment in place for an international, randomized, controlled trial
Adaptimmune Therapeutics	NY-ESO, an affinity-enhanced T-cell targeting therapy	Cancer - HLA-A201, HLA-A205 or HLA-A*206 allele-positive patients with inoperable or metastatic synovial sarcoma who have received prior chemotherapy and whose tumor expresses the NY-ESO-1 tumor antigen	Data from a Phase I/II trial in patients with unresectable, metastatic or recurrent synovial sarcoma who have received prior chemotherapy. In the primary efficacy analysis, 60% of the 10 patients receiving the target dose of cells responded, and there was a 50% overall response rate in the 12 patients receiving any dose of cells.	Clinical - Pivotal trials expected to begin by end of 2016.
Genentech (Roche)	Ocrevus (ocrelizumab), a humanized monoclonal antibody designed to selectively target CD20-positive B cells	Neurodegenerative - Treatment of primary progressive multiple sclerosis (PPMS)	Results from Phase III ORATORIO study that showed ocrelizumab significantly reduced disability progression and other markers of disease activity compared with placebo.	Clinical - BLA submissions in PPMS and relapsing multiple sclerosis planned for first half 2016
AstraZeneca	Durvalumab (MEDI4736), a PD-L1 inhibitor	Cancer - Treatment of patients with PD-L1 positive inoperable or metastatic urothelial bladder cancer whose tumor has progressed during or after one standard platinum-based regimen	Data from a Phase I trial (Study 1108)	Clinical
Novartis	Midostaurin (PKC412), an oral, multi-targeted kinase inhibitor	Cancer - Adults with newly diagnosed, FLT3-mutated acute myeloid leukemia (AML) who are eligible for standard induction and consolidation chemotherapy	Results from the Phase III RATIFY (CALGB 10603) trial, in which patients who received midostaurin and standard induction and consolidation chemotherapy had a significant 23% improvement in overall survival compared to standard therapy alone; median OS in the midostaurin group was 74.7 months vs. 25.6 months in the control arm.	Clinical - Global regulatory submissions to begin in first half 2016

Breakthrough Pace Shows No Signs Of Slowing

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