Cabaletta Bio, Inc.
For the period ended Sep 30, 2025. Filed Nov 10, 2025.
View on SEC.govAbout this filing
A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Sep 30, 2025 · Filed Nov 10, 2025
CABA$1.30+1.6%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2025-06-30; filed 2025-08-07.
0000950170-25-104573
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-09-30; filed 2025-11-10.
0001193125-25-273280
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 160 previous paragraphs and 172 current paragraphs.
113 unchanged; 32 reworded; 27 added; 12 removed; 3 previous and 0 current paragraphs rearranged.
Tables are not compared: 5 previous and 5 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (27)
Current report
Based on clinical data presented in October 2025 at the American College of Rheumatology (ACR) Convergence 2025, we are initiating a DM/ASyS registrational cohort within the RESET-Myositis TM trial. There are approximately 60,000 patients with DM in the U.S. who have IVIg as their only FDA-approved treatment option and approximately 15,000 patients with ASyS in the U.S. who have no FDA-approved treatment options. Consistent with the previously announced FDA alignment on registrational cohort design, which is based on several interactions with FDA regarding the registrational trial, including direct review and feedback on the registrational trial protocol in August 2025, we are aligned on a registrational cohort with a 16-week primary endpoint of moderate or major TIS response while off immunomodulators and on no or low-dose steroids.
Current report
The planned size of 14 patients for the registrational cohort is based on the assumed treatment effect of rese-cel in DM/ASyS patients and an estimated background rate. The estimated background rate will be determined from an external myositis patient registry, as aligned with the FDA, and will include patients with similar inclusion criteria as those in the registrational DM/ASyS cohort. The registry protocol and the statistical analysis plan are expected to be submitted to the FDA this year.
Current report
Based on comprehensive literature review to estimate the background rate, the likelihood of an active, refractory myositis patient achieving moderate or major TIS response within 16 weeks and concurrently discontinuing all immunomodulators is expected to be less than 10%. Changes in the assumed background rate would result in a change in the number of rese-cel treated patients who would need to achieve the primary endpoint in the 14-patient registrational cohort. For example, if the background rate is 10%, then 5/14 patients would need to achieve the primary endpoint for a statistically positive study. If the background rate is 25%, then 8/14 patients would need to achieve the primary endpoint for a statistically positive study.
Current report
In October 2025, we announced rese-cel was generally well tolerated across two AChR-positive and two AChR-negative patients (both seronegative; no anti-MuSK or anti-LRP4 antibodies). No CRS (cytokine release syndrome) occurred in three of four patients with grade 2 CRS occurring in AChR-pos-2 that resolved with no sequelae. As of the September 11, 2025 data cut-off, two evaluable patients (AChR-neg-1 and AChR-neg-2) remain off immunomodulatory medication and achieved significant improvements in
Current report
MG-ADL (with AChR-neg-1 achieving Minimal Symptom Expression). AChR-pos-1 is not evaluable due to use of a prohibited cytotoxic medication that may have inhibited CAR T activity and AChR-pos-2 has insufficient follow-up.
Current report
Both cohorts have been fully enrolled. We anticipate aligning with the FDA on the registrational cohort design for studies in MG in the first half of 2026.
Current report
In October 2025, we announced rese-cel exhibited similar CAR T cell expansion and contraction kinetics relative to translational data reported from other RESETTM trials with preconditioning. All three patients experienced substantial depletion of B cells within the first month post-infusion, with patients 2 and 3 achieving complete peripheral B cell depletion. In these two patients, rapid reduction in autoantibodies to desmoglein was observed and the increase in peak B cell activating factor (BAFF) was at the low end of the range of patients dosed with rese-cel plus preconditioning from pre-infusion through the latest follow-up, suggestive of potential deep B cell depletion in the tissue. Additional follow up will be required to determine if the initial clinical effects are durable and to determine if the findings can be replicated in other autoimmune diseases.
Current report
Rese-cel was generally well tolerated with no immune effector cell-associated neurotoxicity syndrome (ICANS) reported. After infusion, patient 1 experienced transient fever (grade 1 cytokine release syndrome). Patient 2 required a course of steroids for a disease flare in the first two weeks following infusion after discontinuing immunomodulators. This steroid course was less intense than a previous course that was administered for a flare prior to infusion where limited impact on disease was observed. The patient has tapered the steroid dose to below the pre-infusion baseline dose at 3 months post-infusion.
Current report
PDAI activity scores have formed the basis for recent regulatory approvals in PV, and total PDAI scores were also reported to be consistent with the PDAI activity scores in the late breaking clinical trial session. PDAI improvements were most significant in the two patients who seemed to experience complete peripheral B cell depletion. All three patients remain off immunomodulators through the data cut-off September 11, 2025.
Current report
ACR Clinical Data
Current report
In October 2025, we presented updated clinical and translational data from the RESETTM clinical trial programs, with a data cut-off of September 11, 2025. As of October 24, 2025, we have enrolled 76 patients at 77 clinical trial sites globally.
Current report
In the RESET-Myositis TM trial, we presented complete adult Phase 1/2 clinical data from 6 patients in the combined DM/ASyS (4 DM and 2 ASyS syndrome) cohort and 6 patients in the IMNM cohort, in addition to 1 patient in the JIIM cohort. Regarding safety, 4 of 13 patients experienced fever, or grade 1 CRS, and no ICANS was observed.
Current report
All 4 DM/ASyS patients who met the key inclusion criteria for the registrational cohort with sufficient follow-up achieved immunomodulatory-free TIS responses of moderate or major improvement at week 16. Based on these clinical data, we are initiating a DM/ASyS registrational cohort within the RESET-Myositis TM trial. There are approximately 60,000 patients with DM in the U.S. who have IVIg as their only FDA-approved treatment option and approximately 15,000 patients with ASyS in the U.S. who have no FDA-approved treatment options. Consistent with the previously announced FDA alignment on registrational cohort design, we expect to enroll 14 patients in the registrational cohort with a 16-week primary endpoint of moderate or major TIS response while off immunomodulators and on no or low-dose steroids. We remain on track to initiate enrollment in the registrational DM/ASyS cohort in the fourth quarter of 2025.
Current report
Two of 4 IMNM patients with sufficient follow-up achieved immunomodulatory-free TIS responses at week 24. In a subset of ASyS and IMNM patients with limited durability or response, rese-cel achieved complete B cell elimination and an apparent B cell reset, but did not lead to antibody clearance, suggesting CD19 long-lived plasma cells may be a clinically meaningful source for potentially pathogenic autoantibodies in these patients. Prior to the potential initiation of a registrational IMNM cohort, additional patients will be enrolled in the Phase 1/2 cohort with refined entry criteria and existing patients will be followed to further evaluate efficacy and durability in this patient population.
Current report
In the RESET-SSc TM trial, we presented preliminary Phase 1/2 clinical data from 6 patients, including 3 in the severe skin (SSc-Skin) cohort and 3 in the organ (SSc-Organ) cohort. Three of these 6 patients experienced low-grade CRS (grade 1 or 2) and one ICANS event was observed (grade 3, previously reported in March 2025).
Current report
All 4 patients with at least 3 months of follow-up achieved an rCRISS-25 response off immunomodulators and steroids. These initial data suggest the potential for rese-cel to reset the immune system in systemic sclerosis, allowing patients to achieve transformative clinical responses off all immunomodulators and glucocorticoids. We anticipate FDA alignment on the registrational cohort design in the fourth quarter of 2025.
Current report
In the RESET-SLETM trial, we presented preliminary Phase 1/2 clinical data from 9 patients, including 5 patients in the non-renal SLE cohort and 4 patients in the LN cohort. Six of 9 patients experienced no CRS (grade 1 events were reported in 3 patients) and 8 of 9 patients experienced no ICANS (grade 4 in 1 patient, previously reported in August 2024). Three of 4 SLE patients with at least 3 months of follow-up achieved DORIS (definition of remission in SLE), and the fourth patient with pure class V LN achieved a complete renal response. Three of 4 LN patients with at least 3 months of follow-up showed renal response. All 9 patients were off all immunomodulators through the data cut-off of September 11, 2025. Patients across both cohorts achieved a median 8-point reduction in SLEDAI-2K and a significant reduction in anti-dsDNA antibodies was observed.
Current report
Based on the clinical responses observed in lupus following complete B cell depletion after administration of rese-cel with preconditioning, and with the initial data from 3 patients in RESET-PV™ showing that potentially complete B cell depletion is possible with a single, weight-based dose of rese-cel without the use of a fludarabine and cyclophosphamide lymphodepleting regimen, we are expanding this approach into lupus, which predominantly affects women of child-bearing potential. We are incorporating this new dose-escalation cohort into the RESET-SLETM trial with initial clinical data anticipated in 2026.
Current report
Other Income
Current report
Other income, net primarily consists of foreign currency gains and losses and proceeds received from the sale of our Pennsylvania research and development tax credits.
Current report
General and administrative expenses were $6.8 million for the three months ended September 30, 2025 compared to $6.8 million for the three months ended September 30, 2024. General and administrative expenses were comparable to the prior quarter.
Current report
Interest Income
Current report
Other Income, net increased $0.5 million for the three months ended September 30, 2025 as compared to the three months ended September 30, 2024, primarily due to proceeds received from the sale of our Pennsylvania research and development tax credits.
Current report
Results of Operations for the nine months ended September 30, 2025 and 2024
Current report
Interest Income
Current report
Other Income, net increased $0.4 million for the nine months ended September 30, 2025 as compared to the nine months ended September 30, 2024, primarily due to proceeds received from the sale of our Pennsylvania research and development tax credits.
Current report
On August 7, 2025, we filed a Registration Statement (File No. 333-289339) with the Securities and Exchange Commission, or the SEC, which was declared effective on August 15, 2025, or the 2025 Shelf Registration Statement, in relation to the registration of common stock, preferred stock, debt securities, warrants and/or units of any combination thereof for the purposes of selling, from time to time, our common stock, debt securities or other equity securities in one or more offerings. We also simultaneously entered into a Sales Agreement with TD Cowen to provide for the offering, issuance and sale of up to an aggregate amount of $150.0 million of our common stock from time to time in “at-the-market” offerings, or the 2025 ATM Program, under the 2025 Shelf Registration Statement and subject to the limitations thereof, or the 2025 Sales Agreement. No shares have been sold pursuant to the 2025 ATM Program as of the date of this Quarterly Report on Form 10-Q.
Removed paragraphs (12)
Previous report
EULAR Clinical Data
Previous report
In February 2025, we announced updated clinical and translational data from the first 10 patients in the RESETTM clinical trial program, with a data cut off of January 8, 2025. We are presenting new clinical and translational data from 18 evaluable patients who were dosed with rese-cel across the RESET-Myositis TM, RESET-SLETM and RESET-SSc TM trials at the EULAR 2025 Congress in three oral presentations, as of the data cut-off dates of May 6, 2025, for the RESET-Myositis TM and RESET-SSc TM trials and June 2, 2025, for the RESET-SLETM trial.
Previous report
In the RESET-Myositis TM trial, 7 out of 8 patients achieved a clinical response off all immunomodulators and off, or while actively tapering, steroids. Clinical responses were sustained throughout the follow-up period in all 7 responding patients. All 4 ASyS and DM patients achieved a clinically meaningful TIS response, with 3 of these patients having achieved a major TIS response as of the most recent follow-up. Clinically meaningful drug-free TIS responses were observed in 3 of 4 refractory patients with IMNM, which are consistent with published data. These findings continue to reflect the more modest TIS responses in IMNM compared to other myositis subtypes. Regarding safety findings, 4 of 8 patients experienced Grade 1 CRS (fever) and no ICANS was observed.
Previous report
In the RESET-SLETM trial, 7 out of 7 patients with sufficient follow-up to evaluate efficacy achieved substantial SLEDAI reductions off all immunomodulators and glucocorticoids. All 3 of the SLE patients without nephropathy achieved definition of remission in SLE, or DORIS, as of the latest follow-up, and the first LN patient achieved DORIS and a complete renal response. In the 8 patients with safety follow-up, 2 of 8 patients experienced Grade 1 CRS (fever), and 1 ICANS event was observed (Grade 4, previously reported).
Previous report
In the RESET-SSc TM trial, both patients in the severe skin cohort had meaningful modified Rodnan Skin Score (mRSS) improvements after discontinuing immunomodulatory drugs and steroids, which was sustained out to 6 months in the first patient. The first patient also met the revised Composite Response Index in Systemic Sclerosis (CRISS) criteria starting at 3 months, highlighting the potential of rese-cel to provide a drug-free, clinical response in patients with SSc. 1 patient experienced transient Grade 2 CRS, and 1 ICANS event was observed (Grade 3, previously reported)
Previous report
Overall, in the 18 evaluable patients who were dosed with rese-cel with at least 4 weeks of follow-up across the RESET-Myositis TM, RESET-SLETM and RESET-SSc TM trials, 94% had either no CRS or Grade 1 CRS (transient fever) and 89% had no ICANS
Previous report
(2 patients with ICANS events previously reported). Peak rese-cel expansion was observed within approximately two weeks across myositis, lupus, and SSc TM patients. B cells were rapidly and transiently reduced in peripheral blood within the first month, and their repopulation was observed beginning approximately two months after infusion, generally expressing a transitional, naïve phenotype. Tissue-resident B cell depletion was confirmed via a lymph node biopsy in the first systemic sclerosis patient.
Previous report
As of July 31, 2025, five 6-patient Phase 1/2 disease cohorts are fully enrolled across the RESETTM clinical development program.
Previous report
Other Expense
Previous report
Other expense consists primarily of expenses associated with foreign currency gains and losses.
Previous report
Results of Operations for the six months ended June 30, 2025 and 2024
Previous report
General and administrative expenses were $16.4 million for the six months ended June 30, 2025 compared to $12.9 million for the six months ended June 30, 2024. The increase of $3.5 million in our general and administrative expenses include:
Reworded paragraphs (32)
Previous wording
Following a Type C meeting with the FDA and receipt of meeting minutes in April 2025, we are planning to implement the following design for two single-arm, sub-type disease-specific registrational cohorts in the ongoing RESET-Myositis TM trial, either of which, if successful, enable a future Biologics License Application, or BLA, submission for rese-cel in myositis. One cohort will evaluate approximately 15 patients with either DM or ASyS, and one cohort will evaluate approximately 15 patients with IMNM. Inclusion and exclusion criteria will remain highly similar to the Phase 1/2 cohorts. While a BLA for either cohort can be submitted independently, we believe that together, these two cohorts have the potential to support a broad label to address many of the approximately 80,000 myositis patients in the U.S., including those with or without a history of IVIg use. The registrational cohorts will evaluate the same single, weight-based infusion of rese-cel at 1 x 106 cells/kg as used in the Phase 1/2 myositis cohorts. The primary endpoint for the registrational cohorts is based on the total improvement score, or TIS, to evaluate patient outcomes within 26 weeks of rese-cel infusion. The FDA supported the use of pooled rese-cel safety data from across the entire RESETTM clinical trial program to supplement myositis specific safety data for the BLA submission in myositis. In the recent Type C meeting, we aligned with the FDA on the size of the required safety database, which is expected to be approximately 100 autoimmune disease patients treated with the same single weight-based dose. We also aligned with the FDA on combining the Phase 1/2 DM & ASyS cohorts into a single cohort designed to treat six patients with either DM or ASyS. We plan to initiate registrational cohort enrollment in the second half of 2025 with expected BLA submission in 2027.
Current wording
The registrational cohorts will evaluate the same single, weight-based infusion of rese-cel at 1 x 106 cells/kg as used in the Phase 1/2 myositis cohorts. The FDA supported the use of pooled rese-cel safety data from across the entire RESETTM clinical trial program to supplement myositis specific safety data for the BLA submission in myositis and we aligned with the FDA on the size of the required safety database, which is expected to be approximately 100 autoimmune disease patients treated with the same single weight-based dose. We remain on track to initiate enrollment in the registrational DM/ASyS cohort in the fourth quarter of 2025 with expected BLA submission in 2027.
Previous wording
The RESET-SLETM Phase 1/2 clinical trial, which is actively enrolling patients, is designed to treat six SLE patients with active LN, and in a separate parallel cohort, six patients with active SLE without renal involvement, with a single weight-based dose of 1.0 x 106 cells/kg. In May 2023, we announced the FDA granted Fast Track Designation for rese-cel in patients with SLE and LN. In March 2024, Health Canada issued a No Objection Letter in response to a Clinical Trial Application, or CTA, for the RESET-SLETM trial, and in October 2024, the European Medicines Agency allowed a CTA submitted by Cabaletta for the RESET-SLETM trial to proceed, enabling us to begin the process of activating clinical trial sites and pursuing patient enrollment in these geographies. We anticipate aligning with the FDA on the registrational cohort design for studies in SLE/LN in 3Q25.
Current wording
The RESET-SLETM Phase 1/2 clinical trial, which is actively enrolling patients, is designed to treat six SLE patients with active LN, and in a separate parallel cohort, six patients with active SLE without renal involvement, with a single weight-based dose of 1.0 x 106 cells/kg. In May 2023, we announced the FDA granted Fast Track Designation for rese-cel in patients with SLE and LN. In March 2024, Health Canada issued a No Objection Letter in response to a Clinical Trial Application, or CTA, for the RESET-SLETM trial, and in October 2024, the European Medicines Agency allowed a CTA submitted by Cabaletta for the RESET-SLETM trial to proceed, enabling us to begin the process of activating clinical trial sites and pursuing patient enrollment in these geographies. We anticipate aligning with the FDA on the registrational cohort design for studies in SLE/LN in the fourth quarter of 2025.
Previous wording
The actively enrolling RESET-SSc TM Phase 1/2 clinical trial of rese-cel is designed to treat six patients with severe skin manifestations and six patients with severe organ involvement associated with SSc, each in separate parallel cohorts, with a single weight-based dose of 1.0 x 106 cells/kg. We announced the FDA granted Fast Track Designation for rese-cel for SSc patients and Orphan Drug Designation for rese-cel for SSc in January and March 2024, respectively. We anticipate aligning with the FDA on the registrational cohort design for studies in SSc in 4Q25.
Current wording
The actively enrolling RESET-SSc TM Phase 1/2 clinical trial of rese-cel is designed to treat six patients with severe skin manifestations and six patients with severe organ involvement associated with SSc, each in separate parallel cohorts, with a single weight-based dose of 1.0 x 106 cells/kg. We announced the FDA granted Fast Track Designation for rese-cel for SSc patients and Orphan Drug Designation for rese-cel for SSc in January and March 2024, respectively. We anticipate aligning with the FDA on the registrational cohort design for studies in SSc in the fourth quarter of 2025.
Previous wording
The actively enrolling RESET-MGTM Phase 1/2 clinical trial of rese-cel is designed to treat six patients with AChR-positive gMG and six patients with AChR-negative gMG, each in separate parallel cohorts, with a single weight-based dose of 1.0 x 106 cells/kg. We anticipate aligning with the FDA on the registrational cohort design for studies in MG in 1H26.
Current wording
The actively enrolling RESET-MGTM Phase 1/2 clinical trial of rese-cel is designed to treat six patients with AChR-positive gMG and six patients with AChR-negative gMG, each in separate parallel cohorts, with a single weight-based dose of 1.0 x 106 cells/kg.
Previous wording
The ongoing RESET-PVTM trial is designed to evaluate rese-cel as a monotherapy without preconditioning in patients with mucosal pemphigus vulgaris, or mPV, and mucocutaneous pemphigus vulgaris, or mcPV. We anticipate sharing initial dose data from the RESET-PVTM trial in 2H25.
Current wording
The ongoing RESET-PVTM trial is designed to evaluate rese-cel as a monotherapy without preconditioning in patients with mucosal pemphigus vulgaris, or mPV, and mucocutaneous pemphigus vulgaris, or mcPV.
Previous wording
We were incorporated in April 2017 and started principal operations in August 2018. Our operations to date have been financed primarily by proceeds from the sale of convertible notes and convertible preferred stock prior to our initial public offering, or IPO, and proceeds from the sale of our common stock in public equity offerings, including our IPO, “at-the-market” offerings and follow-on offerings of shares of our common stock and pre-funded warrants. As of June 30, 2025, we had $194.7 million in cash, cash equivalents and investments.
Current wording
We were incorporated in April 2017 and started principal operations in August 2018. Our operations to date have been financed primarily by proceeds from the sale of convertible notes and convertible preferred stock prior to our initial public offering, or IPO, and proceeds from the sale of our common stock in public equity offerings, including our IPO, “at-the-market” offerings and follow-on offerings of shares of our common stock and pre-funded warrants. As of September 30, 2025, we had $159.9 million in cash, cash equivalents and investments.
Previous wording
The Minaris Agreement is scheduled to expire upon completion of Minaris' services related to MuSK-CAART and rese-cel. In August 2023, as amended in August 2024, we entered into an agreement with Minaris to serve as one of our manufacturing partners for the global clinical development of rese-cel in multiple indications, including potential late-stage clinical trials and commercial readiness activities for rese-cel. Under the August 2023 work orders, Minaris converted our non-dedicated suite to a dedicated suite for GMP manufacturing for our rese-cel and MuSK-CAART programs, or the Dedicated Suite, for an initial term of 18 months with two 18 month extensions at our sole option on six months' notice prior to the end of the term. In August 2024, we notified Minaris that we would extend the initial term by 18 months through August 2026. In addition, we agreed to certain monthly minimum runs. In August 2024, the 2023 work order related to GMP manufacturing was amended to reduce the minimum monthly runs through the end of 2024. In lieu of the original $1.5 million termination fee under the terms of the Minaris Agreement, we would incur up to a $1.08 million termination fee if we terminate both the rese-cel and MuSK-CAART work orders for any reason. We may terminate for convenience the Minaris Agreement or any work order with six months' prior written notice, however, we may not terminate the Dedicated Suite without terminating both the MuSK-CAART and rese-cel GMP run work orders. Minaris may terminate the Minaris Agreement or any work order for convenience on 18 months' prior written notice, but such notice may not be effective prior to February 2028.
Current wording
In January 2021, we entered into a Development and Manufacturing Services Agreement, or the Minaris Agreement, with Minaris to serve as an additional cell processing manufacturing partner for the MuSK-CAART Phase 1 clinical trial, or MusCAARTes TM trial. The Minaris Agreement is scheduled to expire upon completion of Minaris' services related to MuSK-CAART and rese-cel. In August 2023, as amended in August 2024, we entered into an agreement with Minaris to serve as one of our manufacturing partners for the global clinical development of rese-cel in multiple indications, including potential late-stage clinical trials and commercial readiness activities for rese-cel. Under the August 2023 work orders, Minaris converted our non-dedicated suite to a dedicated suite for GMP manufacturing for our rese-cel and MuSK-CAART programs, or the Dedicated Suite, for an initial term of 18 months with two 18 month extensions at our sole option on six months' notice prior to the end of the term. In August 2024, we notified Minaris that we would extend the initial term by 18 months through August 2026. In addition, we agreed to certain monthly minimum runs. In August 2024, the 2023 work order related to GMP manufacturing was amended to reduce the minimum monthly runs through the end of 2024. In lieu of the original $1.5 million termination fee under the terms of the Minaris Agreement, we would incur up to a $1.08 million termination fee if we terminate both the rese-cel and MuSK-CAART work orders for any reason. We may terminate for convenience the Minaris Agreement or any work order with six months' prior written notice, however, we may not terminate the Dedicated Suite without terminating both the MuSK-CAART and rese-cel GMP run work orders. Minaris may terminate the Minaris Agreement or any work order for convenience on 18 months' prior written notice, but such notice may not be effective prior to February 2028.
Previous wording
Research and development expenses were $37.6 million for the three months ended June 30, 2025 compared to $23.4 million for the three months ended June 30, 2024. The table below summarizes our research and development expenses:
Current wording
Research and development expenses were $39.8 million for the three months ended September 30, 2025 compared to $26.3 million for the three months ended September 30, 2024. The table below summarizes our research and development expenses:
Previous wording
$7.8 million increase in manufacturing costs primarily due to expanded cell processing capabilities, increase in patient enrollment and related activities, including those for commercial readiness;
Current wording
$8.2 million increase in manufacturing costs primarily due to expanded cell processing capabilities, increase in patient enrollment and related activities, including those for commercial readiness;
Previous wording
$3.8 million increase in clinical trial costs primarily due to higher enrollment across multiple clinical studies for rese-cel;
Current wording
$4.6 million increase in clinical trial costs primarily due to higher enrollment and clinical trial sites across multiple clinical studies for rese-cel;
Previous wording
$2.6 million increase in personnel costs primarily driven by an increase in headcount to support overall growth related to our rese-cel program, including an increase of $0.7 million in stock-based compensation expense;
Current wording
$1.4 million increase in personnel costs primarily driven by an increase in headcount to support overall growth related to our rese-cel program, including an increase of $0.5 million in stock-based compensation expense;
Previous wording
Interest income decreased by $1.3 million for the three months ended June 30, 2025 compared to the three months ended June 30, 2024, primarily due to lower cash, cash equivalents and investment balances earning interest, following the use of proceeds from the May 2023 financing and sales of common stock under the 2023 ATM Program in late 2023 and early 2024 and the timing of the June 2025 financing, which closed in mid-June 2025.
Current wording
Interest income decreased by $0.6 million for the three months ended September 30, 2025 compared to the three months ended September 30, 2024, primarily due to lower cash, cash equivalents and investment balances earning interest, following the use of proceeds from the May 2023 financing and sales of common stock under the 2023 ATM Program in late 2023 and early 2024 and the timing of the June 2025 financing, which closed in mid-June 2025.
Previous wording
The following sets forth our results of operations for the six months ended June 30, 2025 and 2024:
Current wording
The following sets forth our results of operations for the nine months ended September 30, 2025 and 2024:
Previous wording
Research and development expenses were $66.7 million for the six months ended June 30, 2025 compared to $45.4 million for the six months ended June 30, 2024. The table below summarizes our research and development expenses:
Current wording
Research and development expenses were $106.5 million for the nine months ended September 30, 2025 compared to $71.7 million for the nine months ended September 30, 2024. The table below summarizes our research and development expenses:
Previous wording
$9.3 million increase in manufacturing costs primarily due to expanded cell processing capabilities, increase in patient enrollment and related activities, including those for commercial readiness;
Current wording
$17.5 million increase in manufacturing costs primarily due to expanded cell processing capabilities, increase in patient enrollment and related activities, including those for commercial readiness;
Previous wording
$7.9 million increase in clinical trial costs primarily due to higher enrollment across multiple clinical studies for rese-cel;
Current wording
$12.5 million increase in clinical trial costs primarily due to higher enrollment and clinical trial sites across multiple clinical studies for rese-cel;
Previous wording
$6.2 million increase in personnel costs primarily driven by an increase in headcount to support overall growth related to our rese-cel program, including an increase of $1.4 million in stock-based compensation expense; partially offset by a
Current wording
$7.6 million increase in personnel costs primarily driven by an increase in headcount to support overall growth related to our rese-cel program, including an increase of $1.9 million in stock-based compensation expense; partially offset by a
Previous wording
$0.7 million decrease in development services due to lower spend on lab consumables and external research activities.
Current wording
$1.5 million decrease in development services due to lower spend on lab consumables and external research activities.
Previous wording
General and administrative expenses were $8.3 million for the three months ended June 30, 2025 compared to $6.9 million for the three months ended June 30, 2024. The increase of $1.4 million in our general and administrative expenses include:
Current wording
General and administrative expenses were $23.1 million for the nine months ended September 30, 2025 compared to $19.7 million for the nine months ended September 30, 2024. The increase of $3.4 million in our general and administrative expenses include:
Previous wording
$3.1 million of additional personnel costs, primarily driven by an increase in headcount, including our international operations, an increase of $0.7 million in stock-based compensation expense and $0.4 million of severance costs; and
Current wording
$2.9 million of additional personnel costs, primarily driven by an increase in headcount, including our international operations, an increase of $0.7 million in stock-based compensation expense and $0.4 million of severance costs; and
Previous wording
$0.3 million higher administrative costs, including legal, information technology and travel as well as other administrative costs.
Current wording
$0.5 million higher administrative costs, including legal, information technology and travel as well as other administrative costs.
Previous wording
Interest income decreased by $2.8 million for the six months ended June 30, 2025 compared to the six months ended June 30, 2024, primarily due to lower cash, cash equivalents and investment balances earning interest, following the use of proceeds from the May 2023 financing and sales of common stock under the 2023 ATM Program in late 2023 and early 2024 and the timing of the June 2025 financing, which closed in mid-June 2025.
Current wording
Interest income decreased by $3.4 million for the nine months ended September 30, 2025 compared to the nine months ended September 30, 2024, primarily due to lower cash, cash equivalents and investment balances earning interest, following the use of proceeds from the May 2023 financing and sales of common stock under the 2023 ATM Program in late 2023 and early 2024 and the timing of the June 2025 financing, which closed in mid-June 2025.
Previous wording
Interest expense increased $0.9 million for the six months ended June 30, 2025 as compared to the six months ended June 30, 2024, primarily due to finance lease arrangements related to embedded leases within our manufacturing agreements with Minaris and Lonza.
Current wording
Interest expense increased $1.4 million for the nine months ended September 30, 2025 as compared to the nine months ended September 30, 2024, primarily due to finance lease arrangements related to embedded leases within our manufacturing agreements with Minaris and Lonza.
Previous wording
From our inception in April 2017 to the time of our initial public offering, or IPO, our operations were financed by proceeds of $86.4 million from the sale of convertible notes and our convertible preferred stock and proceeds of $71.0 million from the sale of common stock in our IPO. Since our IPO, we have generated cash from public offerings of our common stock and pre-funded warrants to purchase our common stock resulting in aggregate net proceeds of approximately $374.0 million. As of June 30, 2025, we had $194.7 million in cash, cash equivalents and investments which should enable us to fund our operations and capital expenditures into the second half of 2026. Cash in excess of immediate requirements is invested in accordance with our investment policy, primarily with a view to liquidity and capital preservation.
Current wording
From our inception in April 2017 to the time of our initial public offering, or IPO, our operations were financed by proceeds of $86.4 million from the sale of convertible notes and our convertible preferred stock and proceeds of $71.0 million from the sale of common stock in our IPO. Since our IPO, we have generated cash from public offerings of our common stock and pre-funded warrants to purchase our common stock resulting in aggregate net proceeds of approximately $374.0 million. As of September 30, 2025, we had $159.9 million in cash, cash equivalents and investments which should enable us to fund our operations and capital expenditures into the second half of 2026. Cash in excess of immediate requirements is invested in accordance with our investment policy, primarily with a view to liquidity and capital preservation.
Previous wording
We have incurred losses since our inception and, as of June 30, 2025, we had an accumulated deficit of $430.2 million. Our primary use of cash is to fund operating expenses, which consist primarily of research and development expenditures, and to a lesser extent, general and administrative expenditures. Cash used to fund operating expenses is impacted by the timing of when we pay these expenses, as reflected in the change in our outstanding prepaid expenses and other current assets, accounts payable and accrued expenses.
Current wording
We have incurred losses since our inception and, as of September 30, 2025, we had an accumulated deficit of $475.0 million. Our primary use of cash is to fund operating expenses, which consist primarily of research and development expenditures, and to a lesser extent, general and administrative expenditures. Cash used to fund operating expenses is impacted by the timing of when we pay these expenses, as reflected in the change in our outstanding prepaid expenses and other current assets, accounts payable and accrued expenses.
Previous wording
In June 2025, we issued (i) 39,200,000 shares of our common stock and accompanying warrants to purchase an aggregate of 39,200,000 shares of common stock (or pre-funded warrants in lieu thereof) and (ii) in lieu of common stock, to certain investors, pre-funded warrants to purchase an aggregate of up to 10,800,000 shares of our common stock and accompanying warrants to purchase an aggregate of 10,800,000 shares of common stock (or pre-funded warrants in lieu thereof), at an exercise price of $0.00001 per pre-funded warrant. The combined offering price of each share of common stock and accompanying common stock warrant was $2.00. The combined offering price of each pre-funded warrant and accompanying common stock warrant was $1.99999. The pre-funded warrants were exercisable immediately. The common stock and pre-funded warrants were sold in combination with an accompanying common stock warrant to purchase one share of common stock (or a pre-funded warrant in lieu thereof) for each share of common stock or pre-funded warrant sold. Each common stock warrant has an exercise price per share of $2.50. The common stock warrants are immediately exercisable from the date of issuance and will expire fifteen months from the date of issuance. Aggregate net proceeds were $93.6 million after deducting underwriting discounts and commissions and offering expenses. As of June 30, 2025, no pre-funded warrants or common stock warrants had been exercised.
Current wording
In June 2025, we issued (i) 39,200,000 shares of our common stock and accompanying warrants to purchase an aggregate of 39,200,000 shares of common stock (or pre-funded warrants in lieu thereof) and (ii) in lieu of common stock, to certain investors, pre-funded warrants to purchase an aggregate of up to 10,800,000 shares of our common stock and accompanying warrants to purchase an aggregate of 10,800,000 shares of common stock (or pre-funded warrants in lieu thereof), at an exercise price of $0.00001 per pre-funded warrant. The combined offering price of each share of common stock and accompanying common stock warrant was $2.00. The combined offering price of each pre-funded warrant and accompanying common stock warrant was $1.99999. The pre-funded warrants were exercisable immediately. The common stock and pre-funded warrants were sold in combination with an accompanying common stock warrant to purchase one share of common stock (or a pre-funded warrant in lieu thereof) for each share of common stock or pre-funded warrant sold. Each common stock warrant has an exercise price per share of $2.50. The common stock warrants are immediately exercisable from the date of issuance and will expire fifteen months from the date of issuance. Aggregate net proceeds were $93.6 million after deducting underwriting discounts and commissions and offering expenses. As of September 30, 2025, 4,800,000 pre-funded warrants had been exercised and 6,000,000 remain outstanding. As of September 30, 2025, no common stock warrants had been exercised.
Previous wording
During the six months ended June 30, 2025, cash used in operating activities of $61.2 million was attributable to our net loss of $81.1 million, partially offset by non-cash charges of $18.8 million for stock-based compensation charges, amortization of finance lease, non-cash operating lease expense, depreciation, accretion of operating lease liabilities and foreign currency exchange rates and a net change of $1.0 million in our net operating assets and liabilities.
Current wording
During the nine months ended September 30, 2025, cash used in operating activities of $95.7 million was attributable to our net loss of $125.9 million, partially offset by non-cash charges of $29.0 million for stock-based compensation charges, amortization of finance lease, non-cash operating lease expense, depreciation, accretion of operating lease liabilities and foreign currency exchange rates and a net change of $1.2 million in our net operating assets and liabilities.
Previous wording
During the six months ended June 30, 2024, cash used in operating activities of $44.8 million was attributable to a net loss of $52.7 million and a net change of $4.2 million in our net operating assets and liabilities, partially offset by non-cash charges of $12.0 million primarily from stock-based compensation, non-cash lease expense, accretion of lease liabilities and depreciation.
Current wording
During the nine months ended September 30, 2024, cash used in operating activities of $65.1 million was attributable to a net loss of $83.3 million and a net change of $1.3 million in our net operating assets and liabilities, partially offset by non-cash charges of $19.5 million primarily from stock-based compensation, non-cash lease expense and accretion of lease liabilities and depreciation.
Previous wording
During the six months ended June 30, 2025, cash used in investing activities of $50.0 million was attributable $49.0 million of purchases of investments and $1.0 million of purchases of property and equipment.
Current wording
During the nine months ended September 30, 2025, cash used in investing activities of $100.0 million was attributable to $99.0 million of purchases of investments and $1.0 million of purchases of property and equipment.
Previous wording
During the six months ended June 30, 2024, cash provided by investing activities of $22.9 million was attributable to $24.5 million from the maturity of short-term investments, partially offset by purchases of $1.6 million of property and equipment.
Current wording
During the nine months ended September 30, 2024, cash provided by investing activities of $35.2 million was attributable to $37.0 million from the maturity of short term investments, partially offset by purchases of $1.8 million of property and equipment.
Previous wording
During the six months ended June 30, 2025, cash provided by financing activities of $92.8 million was attributable to $93.6 million in sales of common stock, warrants and pre-funded warrants to purchase common stock, net of issuance costs paid, $2.6 million in sales of common stock from our ATM offering, net of sales agent commission and fees and $0.2 million from the purchases of shares under our 2019 Employee Stock Purchase Plan, or 2019 ESPP, offset by $3.6 million in principal payments on finance leases.
Current wording
During the nine months ended September 30, 2025, cash provided by financing activities of $92.0 million was attributable to $93.5 million in sales of common stock, warrants and pre-funded warrants to purchase common stock, net of issuance costs paid, $2.6 million in sales of common stock from our ATM offering, net of sales agent commission and fees and $0.2 million from the purchases of shares under our 2019 Employee Stock Purchase Plan, or 2019 ESPP, offset by $4.3 million in principal payments on finance leases.
Previous wording
During the six months ended June 30, 2024, cash provided by financing activities of $7.2 million was from $5.7 million in sales of common stock, net of issuance costs paid, and $1.5 million from the exercise of employee stock options and purchases of shares under our 2019 ESPP.
Current wording
During the nine months ended September 30, 2024, cash provided by financing activities of $7.3 million was from $5.7 million in sales of common stock, net of issuance costs paid, and $1.6 million from the exercise of employee stock options and purchases of shares under our 2019 ESPP.
Rearranged previous paragraphs (3)
Previous report — wording carried elsewhere
In January 2021, we entered into a Development and Manufacturing Services Agreement, or the Minaris Agreement, with Minaris to serve as an additional cell processing manufacturing partner for the MuSK-CAART Phase 1 clinical trial, or MusCAARTes TM trial.
Previous report — wording carried elsewhere
$1.1 million of additional personnel costs, primarily driven by an increase in headcount, including for our international operations and $0.4 million of severance costs; and
Previous report — wording carried elsewhere
$0.4 million higher administrative costs, including legal, information technology and travel as well as other administrative costs.