Cell therapy weekly: KMCAR™ T-cell therapy enters Phase I trial
This week: An Australian biotechnology company has opened its Phase I trial to assess a novel CAR-T therapy for multiple myeloma, while Protara Therapeutics (NY, USA) announced positive 12-month data from its Phase II trial evaluating a cell therapy for non-muscle invasive bladder cancer (NMIBC). Plus, a strategic partnership has been formed to support late-stage manufacturing of an AAV9 gene therapy for a neurodegenerative disorder.
The news highlights:
- Phase I KMCAR™ T-cell trial opened
- Partnership supporting late-phase gene therapy program
- Positive 12-month data for bladder cancer cell therapy
Phase I KMCAR™ T-cell trial opened
Clinical-stage biotechnology company HaemaLogiX (Sydney, Australia) has announced the opening of its KOALA Phase I trial following receipt of Clinical Trial Approval from Australia’s Therapeutic Goods Administration. The KOALA study is a dose-escalation trial assessing the safety and preliminary efficacy of KMCAR™ T-cell therapy, a novel autologous CAR-T therapy targeting the Kappa Myeloma Antigen (KMA) – a tumor-specific receptor found exclusively on myeloma cells and absent from healthy immune cells. This distinguishes it from currently approved BCMA-directed CAR-T therapies.
The therapy is derived from HaemaLogiX’s proprietary KappaMab™ antibody technology, which has been validated in multiple Phase I, IIa and IIb clinical trials, demonstrating a favorable safety profile and durable patient responses. Patient enrollment is now underway at the Peter MacCallum Cancer Centre.
“The commencement of patient screening and enrolment in the KOALA clinical trial represents a significant milestone at a critical time in the evolution of CAR-T therapies for multiple myeloma. Momentum in the field continues to build, highlighted by the Australian Government’s recent decision to provide eligible patients with free access to Carvykti – a BCMA‑targeted CAR‑T cell therapy – reinforcing the growing importance of this treatment class. Our KMCAR™ T-cell therapy is differentiated by its ability to target the tumour‑specific KMA antigen, and we believe it has the potential to further broaden the therapeutic impact of CAR-T approaches in multiple myeloma,” commented Chris Baldwin, CEO of HaemaLogiX.
Partnership supporting late-phase gene therapy program
Elpida Therapeutics (CA, USA) and Catalent (FL, USA) have announced a strategic partnership to support late-phase manufacturing of Elpida’s lead gene therapy program, an AAV9 gene therapy for Spastic Paraplegia Type 50 (SPG50), an ultra-rare neurodegenerative disorder. SPG50 is caused by AP4M1 mutations and, if untreated, can lead to cognitive impairment, epilepsy and progressive paralysis by early adulthood.
“This partnership reflects Catalent’s commitment to applying our broad gene therapy manufacturing expertise and Patient First approach to programs with significant unmet need,” shared David McErlane, Biologics Group President for Catalent. “By leveraging our broad expertise in end-to-end AAV capabilities, we look forward to supporting Elpida’s SPG50 program through late-phase manufacturing so it can advance toward regulatory submission.”
Catalent will use its UpTempo™ AAV manufacturing platform to accelerate production of the research and clinical-grade materials needed for SPG50 process validation and to support Elpida’s Biologics License Application submission.
Positive 12-month data for bladder cancer cell therapy
Protara Therapeutics Inc. has announced positive updated 12-month data from Cohort A of its ongoing Phase II ADVANCED trial evaluating TARA-002 in patients with carcinoma in situ (CIS) (± Ta/T1) NMIBC – specifically Bacillus Calmette-Guérin (BCG)-Naïve NMIBC patients who are receiving their first-line treatment. The results were presented in a poster session at the 2026 American Urological Association Annual Meeting (Washington DC, USA, 15–18 May 2026).
“These data continue to support our conviction that TARA-002 has the potential to make a meaningful difference in the lives of patients with BCG-Naïve NMIBC,” commented Jesse Shefferman, CEO of Protara Therapeutics. “Notably, the durable 12-month complete response (CR) rate observed in the BCG-Naïve cohort continues to perform competitively among approved treatments and investigational therapies in development. We look forward to completing enrollment in the registrational BCG-Unresponsive cohort of ADVANCED-2 and initiating ADVANCED-3, a registrational trial of TARA-002 compared to intravesical chemotherapy in BCG-Naïve and potentially BCG-Exposed patients in the second half of 2026.”
In BCG-naïve patients, TARA-002 achieved a complete response (CR) rate of 72.4% at any time, with CR rates of 66.7% at 6 months and 55% at 12 months. Additionally, 91.7% of responders maintained their CR between 9 and 12 months.