PHILADELPHIA, PA — Cabaletta Bio, Inc. (Nasdaq: CABA) is targeting a second-half 2027 Biologics License Application for rese-cel in myositis while preparing another registrational study in systemic sclerosis, advancing its autoimmune cell-therapy program as cash reserves of $225.1 million fund operations into mid-2027.
The company expects to report registrational data from its 17-patient RESET-Myositis cohort in mid-2027. If the results are successful, Cabaletta plans to submit its first BLA for rese-cel later that year.
That filing is expected to include data from 14 adults with dermatomyositis, three adults with antisynthetase syndrome and a juvenile cohort. Cabaletta believes inclusion of the pediatric data could support eligibility for a Priority Review Voucher.
At the European Alliance of Associations for Rheumatology 2026 Congress, the company reported that 80% of 10 evaluable adult dermatomyositis and antisynthetase syndrome patients would have met the primary endpoint planned for the registrational cohort.
All dermatomyositis responders maintained their responses through follow-up of as long as 1.5 years, according to Cabaletta. The first juvenile dermatomyositis patient achieved what the company characterized as an immunomodulator-free moderate Total Improvement Score response at 16 weeks, maintained through 32 weeks.
Those findings remain from early clinical experience and do not establish that the registrational study will reproduce the same results.
Rese-cel is an investigational CAR T-cell therapy designed to reset the immune system in patients with autoimmune diseases. Cabaletta is evaluating the treatment across its RESET clinical program in rheumatology, neurology and dermatology.
The company is also moving systemic sclerosis-associated interstitial lung disease into registrational development. Based on Phase 1/2 data and FDA feedback, Cabaletta plans to begin a single-arm study in approximately 25 patients in the fourth quarter.
That study is expected to use forced vital capacity at 52 weeks as its primary endpoint.
Cabaletta also is modifying its RESET-MG program for generalized myasthenia gravis to explore dosing without preconditioning, based on emerging findings from its lupus and pemphigus vulgaris studies.
The FDA has granted rese-cel Regenerative Medicine Advanced Therapy designation for generalized myasthenia gravis, adding to designations already obtained in myositis, systemic sclerosis and lupus.
The company is pursuing higher-dose, preconditioning-free cohorts in RESET-SLE and RESET-PV after concluding that the lowest dose may represent a threshold dose in both programs.
Chief Executive Officer Steven Nichtberger pointed to the reported safety profile as supporting broader outpatient use of rese-cel.
Across 17 RESET-Myositis Phase 1/2 patients presented at EULAR 2026, all experienced either no cytokine release syndrome or Grade 1 fever, and none experienced immune effector cell-associated neurotoxicity syndrome, according to the company.
Cabaletta is using those findings in part to support expansion of outpatient dosing across the RESET program.
The company is also building manufacturing capacity ahead of potential commercialization. ElevateBio has been selected as a second contract development and manufacturing organization alongside Lonza.
Cabaletta expects both companies to support clinical-to-commercial supply using a substantially closed and partially automated manufacturing process.
The company separately entered a 10-year commercial supply agreement with Cellares in April. Cabaletta expects that arrangement to provide capacity for thousands of rese-cel batches annually with limited capital investment.
At the American Society of Gene & Cell Therapy 2026 meeting, Cabaletta reported that rese-cel manufactured on the Cellares Cell Shuttle platform produced CAR T-cell expansion and B-cell depletion kinetics comparable to its current clinical manufacturing process.
Research and development expenses increased to $44.4 million in the second quarter from $37.6 million a year earlier as Cabaletta advanced multiple clinical programs.
General and administrative expenses declined to $7.6 million from $8.3 million.
The company ended June with approximately $225.1 million in cash, cash equivalents and short-term investments, up from $133.6 million at the end of 2025. The balance includes proceeds from a registered direct offering completed in May.
Cabaletta expects its June 30 cash position to fund its operating plan into mid-2027, leaving the timing of the planned myositis data readout and subsequent BLA submission closely tied to its next financing horizon.
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