Cell therapy weekly: Promising Phase I results for Parkinson’s disease cell therapy
This week: A US$110 million deal to develop and commercialize ex vivo T-cell therapies is signed and BlueRock Therapeutics (MA, USA) announces positive data from its Phase I clinical trial for its investigational cell therapy for Parkinson’s disease. Plus, CARVYKTI® demonstrates significant survival benefits in patients with multiple myeloma.
The news highlights:
- Research collaboration to develop and commercialize ex vivo T-cell therapies
- Positive Phase I data for investigational Parkinson’s disease cell therapy
- CARVYKTI demonstrates significant survival benefits in patients with multiple myeloma
Research collaboration to develop and commercialize ex vivo T-cell therapies
In a US$110 million deal, Prime Medicine (Prime; MA, USA) and Bristol Myers Squibb (NJ, USA) have entered an agreement to develop next-generation T-cell therapies. Through this partnership, Prime’s precise, multiple gene editing capabilities, such as its Prime Assisted Site-Specific Integrase Gene Editing (PASSIGE™) technology, will be used to support Bristol Myer’s Squibb in developing, manufacturing and commercializing next-generation cell therapies.
Prime Medicine’s PASSIGE technology utilizes prime editing with integrase or recombinase to insert large genetic sequences, enabling stable expression. This non-viral process avoids DNA breaks and off-target edits, allowing for more precise genetic modification.
Prime could earn more than US$3.5 billion in milestone payments, including up to US$1.4 billion for development and over US$2.1 billion for commercialization, as well as royalties on net sales.
“We are excited to collaborate with Bristol Myers Squibb, a global leader in cell therapy for hematology, immunology and oncology. Through this effort, we will apply our Prime Editing technology beyond the rare genetic diseases in our internal pipeline, potentially unlocking opportunities in areas of high unmet needs in immunological diseases and cancer,” said Keith Gottesdiener, President and CEO of Prime Medicine. “We are particularly excited that efforts under this collaboration will leverage our PASSIGE technology, [which] we believe will advance our one-step, non-viral, multi-kilobase-size gene editing approach into the clinic. There is tremendous opportunity for PASSIGE and Prime Editing to revolutionize the field of cell therapy and we look forward to expanding our reach over time through both internal and partnered efforts.”
Positive Phase I data for investigational Parkinson’s disease cell therapy
At the International Congress of Parkinson’s Disease and Movement Disorders (27 September – 1 October 2024; PA, USA), BlueRock Therapeutics, a subsidiary of Bayer AG (Leverkusen, Germany), announced positive 24-month data from its Phase I clinical trial, exPDite, assessing BRT-DA01. BRT-DA01, also known as bemdaneprocel, is an investigational cell therapy that uses stem cell-derived dopamine-producing neurons to replace those damaged by Parkinson’s disease, potentially restoring motor function.
At 24 months, the data demonstrated that patients tolerated bemdaneprocel well, with no reported adverse events. Additionally, transplanted cells have continued to survive in the brain after discontinuing immunosuppression at 12 months. Positive trends in motor symptoms, particularly in the high-dose cohort, suggest that bemdaneprocel may provide sustained benefits for movement impairments in Parkinson’s disease, which were assessed by the Movement Disorder Society-Unified Parkinson’s Disease Rating Scale Part II and III and the Hauser Parkinson’s Disease Diary.
“We are very excited to share the 24-month data from the exPDite trial, which shows that bemdaneprocel could be a potentially meaningful treatment option for individuals living with Parkinson’s disease,” said Amit Rakhit, Chief Development and Medical Officer at BlueRock Therapeutics. “The completion of this study marks an important milestone for bemdaneprocel and sets the stage for the next phase of clinical development.”
CARVYKTI demonstrates significant survival benefits in patients with multiple myeloma
At the 21st International Myeloma Society Annual Meeting (Rio de Janeiro, Brazil; 25-28 September 2024), Johnson & Johnson (NJ, USA) announced long-term results from its Phase III CARTITUDE-4 study, which demonstrated how a single infusion of CARVYKTI significantly extended overall survival when compared with standard therapies. In patients with relapsed or lenalidomide-refractory multiple myeloma who have previously received at least one line of the treatment, including a proteasome inhibitor, the risk of death was reduced by 45% compared to standard therapies. This positive long-term data positions CARVYKTI as the first and only cell therapy to improve the overall survival rate for patients with lenalidomide-refractory multiple myeloma as early as the second line.
“The three-year follow-up data from the Phase III CARTITUDE-4 study show a statistically significant and clinically meaningful improvement in overall survival and quality-of-life measures with CARVYKTI versus standard therapies – meaningful results that have the potential to transform the multiple myeloma treatment landscape,” said Binod Dhakal, Associate Professor of Medicine at the Medical College of Wisconsin (USA), Division of Hematology and study investigator. “This adds to the growing body of data reinforcing the promise of a single infusion of CARVYKTI, which, in addition to demonstrating a significant overall survival benefit, also offers patients the opportunity of a period free from multiple myeloma treatment as early as second line.”
