Marina Biotech and MiNA Therapeutics Announce License Agreement for the Development of saRNA-Based Therapeutics - Seite 2

About SMARTICLES Clinical Experience

Clinical achievements with SMARTICLES represent the combined experiences (a total of approximately 100 patients) of licensees ProNAi Therapeutics, Inc., Plymouth, MI and Mirna Therapeutics, Inc., Austin, TX. 

PNT2258, from ProNAi Therapeutics, is a first-in-class, 24-base, single-stranded, chemically-unmodified DNA oligonucleotide drug targeting BCL2. Data from an ongoing pilot Phase II trial of PNT2258 were reported recently at the 56th Annual Meeting of the American Society of Hematology (ASH). The investigators for the study concluded that:

• PNT2258 treatment results in significant, durable responses in patients with relapsed or refractory non-Hodgkin's Lymphoma (r/r NHL) with eleven of the thirteen (11/13) patients treated achieving clinical benefit, with ongoing Progression Free Survival (PFS) extending to 18 months and beyond.
  
  
• In particular, all four of the patients (4/4) with DLBCL responded to PNT2258, with three patients achieving complete responses (CR) and one patient achieving a partial response (PR), with durations extending to greater than 500 days.
  
  
• Durable and clinically meaningful CR's and PR's were achieved in subjects with aggressive disease, such as Richter's transformation and Burkitt's-like DLBCLPNT2258 therapy is safe and very well tolerated with dosing periods up to and exceeding 18 months.
  
  

Mirna Therapeutics' clinical compound, MRX34, is a double-stranded microRNA "mimic" of the naturally occurring tumor suppressor miR-34, which inhibits cell cycle progression and induces cancer cell death. Data from an ongoing Phase 1 clinical trial shows that MRX34 has a manageable safety profile in patients with advanced primary liver cancer (hepatocellular carcinoma), other solid tumors with liver metastasis, and hematological malignancies. A maximum tolerated dose (MTD) was established at 110 mg/m2 for MRX34 administered twice weekly for three weeks followed by one week off. Dose escalation is on-going for a second dosing regimen wherein MRX34 is administered daily for five consecutive days followed by two weeks off. While this Phase 1 study is intended to investigate safety, tolerability, pharmacokinetics, and dosing regimens, treatment with MRX34 has provided early signals of clinical activity in advanced cancer patients with primary liver, neuroendocrine, colorectal and small cell lung cancers, as well as diffuse large B-cell lymphoma.