Cell therapy weekly: Gilead Sciences acquires Arcellx for $7.8 billion
This week: Ray Therapeutics (CA, USA) received Priority Medicines (PRIME) designation for its gene therapy for retinitis pigmentosa, the US Food and Drug Administration (FDA; MD, USA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to Orca Bio (CA, USA) for its allogeneic T-cell therapy for high-risk hematologic malignancies, Gilead Sciences acquired Arcellx (both CA, USA) for US$7.8 billion, and Cellares (CA, USA) and Cabaletta Bio (PA, USA) entered into a 10-year commercial supply agreement for an autoimmune CAR-T therapy.
The news highlights:
Retinitis pigmentosa gene therapy gets PRIME designation
European Medicines Agency (EMA) has granted PRIME designation to Ray Therapeutics’ lead gene therapy program, RTx-015, for the treatment of retinitis pigmentosa (RP). RTx-015 also received RMAT designation from the US Food and Drug Administration (FDA; MD, USA) earlier this month.
“Receiving PRIME designation for RTx-015 in RP from the EMA, alongside our recent RMAT designation from the FDA, is a powerful validation of our science and clinical outcomes, and the urgent need we are addressing,” said Paul Bresge, CEO and co-founder of Ray Therapeutics. “There are more than a million people living with retinitis pigmentosa who have no treatment options to augment their vision and improve their quality of life. These designations accelerate our ability to advance RTx-015 globally and deliver on our mission to restore vision for patients living with blinding diseases.”
The PRIME designation was granted following positive preliminary safety and efficacy results from the ongoing Phase I/II study of RTx-015 in patients with advanced retinitis pigmentosa.
RMAT designation for T-cell therapy
Orca Bio received RMAT designation from the FDA for its second-generation investigational allogeneic T-cell immunotherapy for the treatment of high-risk hematologic malignancies, Orca-Q. This designation was based on data from the ongoing Phase I clinical trial evaluating the therapy for the treatment of hematologic malignancies in six different treatment cohorts, with clinical findings demonstrating durability and positive outcomes across several key metrics.
“The FDA’s RMAT designation for Orca-Q recognizes the significant unmet need for patients with serious hematologic malignancies and validates the promising clinical findings from our ongoing Phase 1 study,” said Nate Fernhoff, co-founder and CEO at Orca Bio. “As this is our second cell therapy candidate to receive RMAT status, we have experienced firsthand how this program can support accelerated development and enhanced communication with the FDA. With our newly expanded Phase I study continuing to enroll patients and additional data expected later this year, we remain focused on advancing our high-precision approach to deliver an important new treatment to patients with blood cancer.”
Gilead Sciences acquires Arcellx
Gilead Sciences has completed its acquisition of Arcellx for US$115 per share in cash, plus one non‑transferable contingent value right of US$5 per share, representing a total implied equity value of approximately US$7.8 billion. This acquisition expands upon the existing partnership between Kite (a subsidiary of Gilead) and Arcellx, giving Gilead complete ownership and authority over anitocabtagene autoleucel (anito-cel), an investigational BCMA‑directed CAR T‑cell therapy for multiple myeloma.
“With the Arcellx acquisition, our focus turns to executing with speed and discipline as we prepare to bring anito‑cel to patients,” said Cindy Perettie, Executive Vice President and Global Head of Kite. “I want to thank the Arcellx team for their scientific leadership, close collaboration to date and deep expertise they bring as we advance anito‑cel. With this acquisition, anito‑cel and the differentiated D‑Domain BCMA binder will advance within Kite, combining this science with our global manufacturing, regulatory and commercial capabilities to unlock the full value of this potentially transformative therapy for people living with multiple myeloma.”
Autoimmune disease CAR-T therapy commercial supply agreement
Cellares and Cabaletta Bio announced a 10-year commercial supply agreement for the manufacturing of resecabtagene autoleucel (rese-cel), Cabaletta’s investigational CAR-T therapy for autoimmune diseases using Cellares’ Cell Shuttle and Cell Q platforms.
“The first two doses of rese-cel manufactured on the Cell Shuttle met all release specifications and were delivered and infused on time to patients in the RESET clinical program, validating the automated manufacturing model and supply chain logistics we committed to in 2023. For a company developing potentially curative therapies for autoimmune diseases, where the addressable patient population can far exceed that in oncology, scalability, reliability, and cost efficiency are important considerations. This 10-year commercial agreement with Cellares provides the cost-efficient foundation we need to expand access to rese-cel while building a profitable business,” said Steven Nichtberger, CEO of Cabaletta Bio.