ADARx bags USD 75 Million to advance its RNA tech pipeline

ADARx Pharmaceuticals, a biotechnology company developing RNA targeting therapeutics, announced the completion of a USD 75 million Series B financing to progress its drug development pipeline. SR One Capital Management and OrbiMed Advisors co-led Series B. Sirona Capital united this financing as well as existing investors OrbiMed Advisors and Lilly Asia Ventures.

Simeon George, M.D., CEO of SR One, said that the ADARx team focuses on expeditiously translating their insights around RNA biology into novel medicines to benefit patients. The team has made tremendous progress in advancing its unique, multi-faceted RNA platform in the short time since the Company’s launch, and they look forward to helping build the pipeline with this financing.

Carl Gordon, Ph.D., CFA, Founding Partner and Co-Head of Global Private Equity at OrbiMed Advisors, told OrbiMed is pleased to continue backing Zhen Li and her team in creating an exciting oligonucleotide-based drug company, which is developing new treatment modalities.

The proceeds from the Series B financing will be utilized to progress ADARx’s proprietary RNA platform technologies for base editing, inhibition, degradation, and delivery. The outcomes will also be utilized to transition ADARx’s growing pipeline of research phase products into the clinic, initiating Phase 1 clinical trials for ADARx’s lead program.

Vor Biopharma’s AML drug secures Fast Track Designation 

Vor Biopharma has declared that the FDA has awarded their acute myeloid leukemia (AML) engineered hematopoietic stem cell therapy (eHSC), VOR33, a Fast Track Designation.

VOR33 comprises CRISPR genome-edited hematopoietic stem and progenitor cells, which have been engineered to exclude CD33. Once infused, VOR33 is developed to protect patients’ healthy cells from anti-CD33 therapies, such as VCAR33 or Mylotarg (gemtuzumab ozogamicin).

VOR33 is intended to substitute the standard of care transplants for AML patients at high risk of relapse and seamlessly integrate them into current transplant settings.

VOR33 was created through Vor’s genome eHSC platform combined with multiple therapeutic modalities to create treatment-resistant transplants that can improve the potential of targeted cancer therapies through cell therapy and gene editing.

Vor said in a statement that it is actively enrolling in its Phase I/II clinical trial for AML patients who currently have restricted treatment options.

Ixaka declares positive interim Phase 3 clinical trial data for its lead cell therapy candidate REX-001

Ixaka Ltd is an integrated cell and gene therapy company, declares positive interim results of its lead REX-001 drug candidate from a Phase 3 clinical trial, which assessed the safety and efficacy results 12 months after treatment with REX-001 or placebo in patients diagnosed with chronic limb-threatening ischemia (CLTI) and diabetes. After evaluating the interim efficacy and safety data, the independent Data Monitoring Committee (DMC) recommended sustaining the trial unchanged.

Following unblinded pre-established statistical analysis of safety and efficacy results from the first 30% of enrolled patients, the DMC recommended continuing the study as planned in the protocol, confirming that the anticipated effect of REX-001 compared to placebo in the trial is on track and that the benefit-risk was acceptable. There were no tremendous safety concerns.

This positive interim analysis backs the hypothesis of REX-001 being an effective and safe treatment for CLTI and is compatible with the effect observed in earlier clinical trials. REX-001 treatment resulted in full ulcer healing in over 70% of patients. The Phase 3 trial with REX-001 comprises two planned interim evaluations after 30% and 50% of patients enrolled have reached the 12-month follow-up visit. Ixaka remains blinded to the data until the execution of the trial.

REX-001 is an MCT product in clinical development as patient-specific cellular immunotherapy. The product is developed using cells extracted from a patient’s bone marrow and enriched for white blood cells. Re-administration into the affected limb proffers multiple immune and progenitor cells squarely to the diseased vessels to meet the complex disease processes, which lead to the clinical progression of CLTI (plaque deposition, inflammation, ischemia, ulcer formation, vessel degeneration).

FDA awards Breakthrough Device Designation to the NovoTTF-200T™ System for Advanced Liver Cancer

Novocure has been offered a breakthrough designation from the FDA for the company’s devices zeroed in on aggressive liver cancer.

Following final results in July from a phase 2 pilot study, Novocure intends to move its therapy into an additional phase 2 trial as a first-line option for unresectable or metastatic tumors when given in combination with Tecentriq (atezolizumab) and Avastin (bevacizumab).

It is worn as a patch over the skin; the electric fields hamper the division and reproduction of cancer cells of a particular size while decreasing their effects on healthy cells.

Novocure CEO Asaf Danziger said in a statement that their data hints (Tumor Treating Fields) have the potential to extend survival in this, particularly aggressive disease. They are working closely with trial investigators and intend to start a randomized controlled trial studying TTFields in combination with atezolizumab and bevacizumab as soon as possible.

The company’s finished phase 2 pilot study of liver cancer patients combined its devices with sorafenib. Among the 11 patients who completed 12 weeks of treatment, 10 showed disease control, while two participants saw their tumors contract.

Novocure is also evaluating the therapy in non-small cell lung cancer. This past April, the company was given the green light from the FDA to move ahead with a phase 2 clinical trial combining the electric fields with Keytruda (pembrolizumab) in the first-line setting. The study has at least five sites actively enrolling participants.