On August 12, 2025 Autolus Therapeutics plc (Nasdaq: AUTL), an early commercial-stage biopharmaceutical company developing, manufacturing and delivering next-generation programmed T cell therapies, reported its operational and financial results for the second quarter ended June 30, 2025 (Press release, Autolus, AUG 12, 2025, View Source [SID1234655147]).

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"We are encouraged by AUCATZYL’s early launch performance in the U.S., driven by physician enthusiasm for the product profile, unmet need for r/r B-All patients and favorable market access and reimbursement – supported by strong execution on manufacturing and product delivery," said Dr. Christian Itin, Chief Executive Officer of Autolus. "With recent approvals in the EU and UK our focus shifts to market access on a country-by-country basis. In addition to commercial progress in the adult setting, we look forward to reporting clinical data from the pediatric PY1 trial of obe-cel in ALL in the second half of the year and believe there is additional growth opportunity in pediatric ALL."

"Beyond ALL, we believe obe-cel has ‘pipeline-in-a-product’ potential and could deliver improved outcomes in autoimmune disease. We are excited about the recently reported preliminary data from the Phase 1 CARLYSLE study in systemic lupus erythematous (SLE). We look forward to reporting additional Phase 1 data in SLE patients at a medical conference later this year; dosing the first patient in our planned Phase 2 pivotal trial in LN and starting a Phase 1 trial in progressive multiple sclerosis (MS) by year-end."

Key updates and anticipated milestones:

AUCATZYL Launch
Autolus reported Q2 2025 net product sales of $20.9 million.
The Company has 46 centers fully activated in the U.S. as of August 12, 2025.
Patient access to AUCATZYL continues to increase, with coverage secured for greater than 90% of total U.S. medical lives.
On April 25, 2025, the UK Medicines and Healthcare products Regulatory Agency (MHRA) granted conditional marketing authorization for AUCATZYL for the treatment of adult patients age 18+ with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-ALL). Following an initial review and in line with prior practice for CAR T therapies the National Institute for Health and Care Excellence (NICE) issued a preliminary Appraisal Consultation Decision (ACD) recommending against funding for AUCATZYL. Autolus plans to respond to NICE’s questions and will continue to work towards a pathway for patient access to therapy in the UK.
On July 17, 2025, the European Commission (EC) granted conditional marketing authorization for AUCATZYL in adult patients (age 26 and older) with r/r B-ALL. Evaluation of potential pricing and feasibility of market entry opportunities in certain EU countries is ongoing; however, at this time launch in Germany is on hold and the Company does not anticipate any EU sales of AUCATZYL in 2025 and 2026.
Obe-cel data in r/r B-ALL
Autolus presented updated long-term data from the FELIX study in adult patients with r/r B-ALL in an oral presentation at the 2025 European Hematology Association (EHA) (Free EHA Whitepaper) Congress in June. For patients with a response, the updated median duration of response (mDOR) is now 42.5 months. At the updated median follow up of 32.8 months, 38.4% of responders were in ongoing remission without consolidative stem cell therapy or other therapies (versus the previously reported 40% at a median follow-up of 21.5 months). The 24-month probability of Event Free Survival was 43%, and the Overall Survival was 46%, with a further consolidating long-term plateau observed. No new safety signals or Grade ≥3 secondary malignancies were observed at the extended follow-up. These results suggest that obe-cel may be a durable treatment option for some patients with r/r B-ALL.
Obe-cel in lupus nephritis (LN)
Preliminary data from the Phase 1 dose confirmation clinical trial (CARLYSLE) in refractory systemic lupus erythematosus (SLE) patients were reported on April 23, 2025, and support progressing into a planned Phase 2 pivotal study.
The Company has aligned with the U.S. Food and Drug Administration (FDA) on the Phase 2 trial design and potential registrational path to approval and continues to anticipate dosing the first patient in a Phase 2 clinical trial before the end of 2025.
Data with longer term follow-up from the Phase 1 CARLYSLE clinical trial is on track for presentation at a medical conference in the second half of 2025.
Obe-cel in progressive MS
Autolus plans to advance obe-cel into initial clinical development in progressive MS. The Company continues to expect to dose its first patient in a Phase 1 dose escalation study by year-end 2025.
Early-stage pipeline programs and collaborations support longer-term growth
Autolus’ translational programs with UCL continue to fuel its early-stage pipeline, providing a cost-efficient path to development.
Summary of Anticipated News Flow:

ALL: PY01 trial in pediatric ALL first clinical data H2 2025
SLE: Phase 1 CARLYSLE trial presentation at medical conference H2 2025
LN: Expect to dose first patient in Phase 2 trial By year-end 2025
MS: Expect to dose first patient in Phase 1 trial in progressive MS By year-end 2025
ALA: Expect to dose first patient in Phase 1 trial in AL amyloidosis By year-end 2025
ALL: adult lymphoblastic leukemia
SLE: systemic lupus erythematosus
LN: lupus nephritis
MS: multiple sclerosis
ALA: light-chain amyloidosis

Financial Results for the Quarter Ended June 30, 2025

Product revenue, net for the three months ended June 30, 2025 was $20.9 million.

Cost of sales for the three months ended June 30, 2025 totaled $24.4 million. This amount includes the cost of all commercial product delivered to the authorized treatment centers, including product delivered but not yet recorded as product revenue which is captured as deferred revenue. Additionally, cost of sales includes any cancelled orders in the period, patient access program product, and 3rd party royalties for certain technology licenses.

Research and development expenses decreased from $36.6 million to $27.4 million for the three months ended June 30, 2025, compared to the same period in 2024. This change was primarily due to commercial manufacturing-related employee and infrastructure costs shifting to cost of sales and inventory.

Selling, general and administrative expenses increased from $21.9 million to $30.3 million for the three months ended June 30, 2025, compared to the same period in 2024. This increase was primarily due to salaries and other employment-related costs, driven by increased headcount supporting commercialization activities.

Loss from operations for the three months ended June 30, 2025 was $61.2 million, as compared to $58.9 million for the same period in 2024.

Net loss was $47.9 million for the three months ended June 30, 2025, compared to $58.3 million for the same period in 2024. Basic and diluted net loss per ordinary share for the three months ended June 30, 2025, totaled $(0.18), compared to basic and diluted net loss per ordinary share of $(0.22) for the same period in 2024.

Cash, cash equivalents and marketable securities at June 30, 2025, totalled $454.3 million, as compared to $588.0 million at December 31, 2024. The decrease was primarily driven by net cash used in operating activities and impacted by a delayed cash receipt of approximately $21.7 million in R&D tax credit expected from the UK HMRC, which was expected to be received during the six months ended June 30, 2025.

Autolus estimates that, with its current cash and cash equivalents and marketable securities, the Company is well capitalized to drive the launch and commercialization of obe-cel in r/r B-ALL and to obtain data in the LN pivotal trial and MS Phase 1 trial.

Financial Results for the Period Ended June 30, 2025
Selected Consolidated Balance Sheet Data
(In thousands)

June 30, December 31,
2025 2024
Assets
Cash and cash equivalents $ 123,825 $ 227,380
Marketable securities – Available-for-sale debt securities $ 330,454 $ 360,643
Total current assets $ 574,250 $ 660,929
Total assets $ 720,981 $ 782,725
Liabilities and shareholders’ equity
Deferred revenue $ 2,100 $ —
Total current liabilities $ 68,151 $ 60,743
Total liabilities $ 374,517 $ 355,400
Total shareholders’ equity $ 346,464 $ 427,325

Selected Consolidated Statements of Operations and Comprehensive Loss Data
(In thousands, except share and per share amounts)

Three Months Ended June 30, Six Months Ended June 30,
2025 2024 2025 2024
Product revenue, net $ 20,923 $ — $ 29,905 $ —
License revenue — — — 10,091
Operating expenses:
Cost of sales (24,445 ) — (42,396 ) —
Research and development expenses, net (27,430 ) (36,612 ) (54,164 ) (67,283 )
Selling, general and administrative expenses (30,265 ) (21,903 ) (59,799 ) (40,080 )
Loss on disposal of property and equipment — — (3 ) —
Impairment of operating lease right-of-use assets and related property and equipment — (414 ) — (414 )
Loss from operations (61,217 ) (58,929 ) (126,457 ) (97,686 )
Foreign exchange gains (losses), net 1,634 1,226 2,942 (379 )
Interest income (expenses), net 12,063 (518 ) 8,057 (12,854 )
Total other income (expenses), net 13,697 708 10,999 (13,233 )
Net loss before income tax (47,520 ) (58,221 ) (115,458 ) (110,919 )
Income tax expense (397 ) (51 ) (2,623 ) (43 )
Net loss attributable to ordinary shareholders (47,917 ) (58,272 ) (118,081 ) (110,962 )
Other comprehensive income, net of tax 18,968 1,026 30,036 1,084
Total comprehensive loss $ (28,949 ) $ (57,246 ) $ (88,045 ) $ (109,878 )

Basic and diluted net loss per ordinary share $ (0.18 ) $ (0.22 ) $ (0.44 ) $ (0.43 )
Weighted-average basic and diluted ordinary shares 266,141,411 265,025,783 266,134,021 255,131,873

Conference Call
Management will host a conference call and webcast today at 8:30am EDT/13:30pm BST to discuss the company’s financial results. Conference call participants should pre-register using this link to receive the dial-in numbers and a personal PIN, which are required to access the conference call. A simultaneous audio webcast and replay will be accessible on the events section of Autolus’ website at View Source

On August 12, 2025 Monopar Therapeutics Inc. ("Monopar" or the "Company") (Nasdaq: MNPR), a clinical‐stage biopharmaceutical company focused on developing innovative treatments for patients with unmet medical needs, reported second quarter 2025 financial results and recent developments (Press release, Monopar Therapeutics, AUG 12, 2025, View Source [SID1234655146]).

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Recent Developments

ALXN1840 for Wilson Disease

On June 6, 2025, Alexion Pharmaceuticals officially transferred sponsorship of the investigational new drug ("IND") application for ALXN1840 to Monopar. The U.S. Food and Drug Administration ("FDA") acknowledged this change on July 29, 2025, confirming that the transfer was effective as of June 6, 2025. Monopar is now fully responsible for the program, including its commercial advancement and compliance with all applicable federal regulations.

Monopar is preparing to submit a New Drug Application ("NDA") to the FDA in early 2026.

MNPR‐101 for Radiopharmaceutical Use

The Company’s MNPR-101-Zr Phase 1 (imaging and dosimetry) and MNPR-101-Lu (therapeutic) Phase 1a clinical trials in advanced cancers are active and enrolling in Australia, and the Company’s Expanded Access Program (also referred to as compassionate use) for MNPR-101-Zr and MNPR-101-Lu is active and enrolling in the U. S. Monopar continues its preclinical work with MNPR-101-Ac (therapeutic) with plans to enter the clinic in the future.

Financial Results for the Second Quarter Ended June 30, 2025, Compared to the Second Quarter Ended June 30, 2024

Cash and Net Loss

Cash, cash equivalents and investments as of June 30, 2025, were $53.3 million. Monopar expects that its current funds will be sufficient to continue operations at least through December 31, 2026, in order to: (1) assemble a regulatory package and file an NDA for ALXN1840; (2) continue to conduct and conclude its first-in-human imaging and dosimetry clinical trial with MNPR-101-Zr; (3) continue to conduct its first-in-human therapeutic clinical trial of MNPR-101-Lu; (4) advance its preclinical MNPR-101-Ac program into the clinic; and (5) invest in internal research and development projects to expand its radiopharmaceutical and rare disease pipeline.

Net loss for the second quarter of 2025 was $2.5 million or $0.35 per share compared to net loss of $1.7 million or $0.49 per share for the second quarter of 2024.

Research and Development ("R&D") Expenses

R&D expenses for the second quarter of 2025 were $1,730,000, compared to $1,130,978 for the second quarter of 2024. This represents an increase of $599,023 attributed to (1) a $636,300 increase in R&D personnel expenses including stock-based compensation, partially offset by (2) a net decrease of $37,277 in other R&D expenses.

General and Administrative ("G&A") Expenses

G&A expenses for the second quarter of 2025 were $1,504,295, compared to $657,806 for the second quarter of 2024. This represents an increase of $846,489 primarily attributed to (1) a $370,103 increase in Board compensation resulting from the grant of stock options in March 2025 (no stock options were granted to the Board in 2024), (2) a $255,650 increase in G&A personnel expenses including stock-based compensation, (3) a $114,322 increase in legal fees, (4) a $63,200 increase in state franchise taxes, (5) a $41,416 increase in insurance expenses and (6) a net increase of $1,798 in other G&A expenses.

Interest Income

Interest income for the three months ended June 30, 2025, increased by $707,294 compared to the same period in 2024. The increase is attributed to interest earned on U.S. Treasury securities and higher bank balances in 2025, resulting from over $55 million of funds raised in the fourth quarter of 2024.

On August 12, 2025 INOVIO (NASDAQ: INO), a biotechnology company focused on developing and commercializing DNA medicines to help treat and protect people from HPV-related diseases, cancer, and infectious diseases, reported its financial results for the second quarter of 2025 and provided an update on recent company developments (Press release, Inovio, AUG 12, 2025, View Source [SID1234655145]).

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"With device DV testing complete, we remain on track to submit our BLA for INO-3107 in the second half of this year, with the goal of having FDA acceptance of the file by year end. Utilizing our breakthrough therapy designation, we’ve requested rolling submission and expect to be able to immediately provide the clinical and non-clinical modules for review, while we complete work on the device-related sections and update our Investigational New Drug (IND) Application for our confirmatory trial," said Dr. Jacqueline Shea, INOVIO’s President and Chief Executive Officer. "We believe that INO-3107 could become the preferred treatment option for Recurrent Respiratory Papillomatosis (RRP) patients and their physicians—a treatment option with the potential to change the trajectory of this disease. I look forward to building on the significant progress of this past quarter and providing updates as we work toward a potential approval date in mid-2026."

Operational Highlights

INO-3107 – Recurrent Respiratory Papillomatosis (RRP)
INOVIO completed the DV testing for the CELLECTRA 5PSP device and requested in July 2025 that the FDA allow it to begin submitting its BLA on a rolling basis based on the Breakthrough Therapy designation previously granted to INO-3107. INOVIO anticipates completing its submission over the next several months and requesting a priority review. FDA inspection of INOVIO as clinical sponsor of the Phase 1/2 trial was successfully completed. The company is working on the device-related sections for its BLA and updating its active IND so it can begin enrolling patients into its placebo-controlled, randomized confirmatory trial, which will include 100 patients and be conducted at approximately 20 sites across the United States.

Data from a retrospective study (RRP-002) investigating the long-term clinical efficacy of patients treated with INO-3107 have been published in a peer-reviewed journal, The Laryngoscope. The data demonstrate that INO-3107 provided significant clinical benefit to RRP patients, as measured by reduction in surgery, that continued to improve in years two and three following initial treatment. Highlights from the paper include:

Patients experiencing a 50-100% reduction in surgeries (Overall Response Rate) increased from 72% at the end of the initial 12-month Phase 1/2 trial (Year 1) to 86% at the end of the second 12-month period (Year 2)
Patients experiencing a Complete Response (CR – 0 surgeries per year) increased from 28% for Year 1 to 50% for Year 2
Mean number of surgeries was reduced from 4.1 in the pre-treatment period (the 52 weeks prior to beginning treatment with INO-3107) to 1.7 for Year 1 to 0.9 for Year 2
Partial data into the third 12-month period (Year 3 – median follow up 2.8 years following initial treatment) continued the trend of improvement and a reduced number of surgeries
INO-3107 was well tolerated, with no serious adverse events or long-term safety concerns identified
Next Generation DNA Medicine Candidates
INOVIO presented data on its next generation DNA medicine technology at the Orphan Drug Summit in July. Data included key insights from an ongoing Phase 1 proof-of-concept trial evaluating DNA-encoded monoclonal antibodies (DMAbs) for COVID-19 (preprint of manuscript available on Research Square) as well as an overview of DNA-encoded protein technology (DPROT) that targets long-term protein expression and aims to address some of the shortcomings of conventional therapeutic protein replacement treatments.

Upcoming Presentations
INOVIO will present data on INO-3107 and other promising DNA medicine candidates at the following upcoming events:

American Academy of Otolaryngology – October 10-13
World Vaccine Congress Europe – October 13-16
European Society for Medical Oncology – October 17-21
37th International Papillomavirus Society Conference – October 23-26
World Orphan Drug Congress – October 27-29
ISV Congress – October 28-30
General Corporate
INOVIO remains focused on financial discipline and directing resources to support the INO-3107 program. The company strengthened its balance sheet with an underwritten public offering of common stock and warrants in July 2025. Net proceeds from the offering, after deducting underwriting discounts, commissions and offering expenses, were approximately $22.5 million.

Second Quarter 2025 Financial Results

Research and Development (R&D) Expenses: R&D expenses for the three months ended June 30, 2025, decreased to $14.5 million from $23.1 million for the same period in 2024. The decrease was primarily the result of lower drug manufacturing, clinical study and other expenses related to INO-3107, and lower contract labor, among other variances.
General and Administrative (G&A) Expenses: G&A expenses decreased to $8.6 million for the three months ended June 30, 2025, from $10.2 million for the same period in 2024. The decrease was primarily related to a decrease in employee and consultant stock-based compensation, lower outside services and lower contract labor, among other variances.
Total Operating Expenses: Total operating expenses decreased to $23.1 million for the three months ended June 30, 2025, from $33.3 million for the same period in 2024.
Net Loss: Net loss for the three months ended June 30, 2025, decreased to $23.5 million, or $0.61 per basic and diluted share, from a net loss of $32.2 million, or $1.19 per basic and diluted share, for the three months ended June 30, 2024.
Shares Outstanding: As of June 30, 2025, INOVIO had 36.7 million common shares outstanding and 51.7 million common shares outstanding on a fully diluted basis, after giving effect to the exercise, vesting, and conversion, as applicable, of its outstanding common stock warrants, including pre-funded warrants and stock options, restricted stock units and convertible preferred stock.
Cash, Cash Equivalents and Short-term Investments: As of June 30, 2025, cash, cash equivalents and short-term investments were $47.5 million (excluding net proceeds from the July 2025 offering of $22.5 million), compared to $94.1 million as of December 31, 2024.
INOVIO’s balance sheet and statement of operations are provided below. Additional information is included in INOVIO’s quarterly report on Form 10-Q for the quarter ended June 30, 2025, which can be accessed at: View Source

Cash Guidance

INOVIO estimates its current cash, cash equivalents and short-term investments balances, including the net proceeds from the July 2025 offering, will support the company’s operations into the second quarter of 2026. This projection includes an operational net cash burn estimate of approximately $22 million for the third quarter of 2025. These projections do not include any further capital-raising activities that INOVIO may undertake.

Conference Call / Webcast Information
INOVIO’s management will host a live conference call and webcast with slides at 4:30 p.m. ET today to discuss INOVIO’s financial results and provide a general business update. The live webcast and replay may be accessed by visiting INOVIO’s website at View Source

On August 12, 2025 Bayer and Kumquat Biosciences Inc., a clinical-stage biotech company founded by pioneers of the KRAS pathway, reported that they have entered into an exclusive global license and collaboration to develop and commercialize Kumquat’s KRAS G12D inhibitor (Press release, Kumquat Biosciences, AUG 12, 2025, View Source [SID1234655142]). Under the agreement, Kumquat is responsible for the initiation and completion of the Phase Ia study, while Bayer will complete development and commercial activities.

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Kumquat received U.S. Food and Drug Administration (FDA) clearance of the investigational new drug (IND) for its KRAS G12D inhibitor in July 2025. Under the terms of the agreement, Kumquat will receive up to $1.3 billion, including upfront, clinical and commercial milestones, and additional tiered royalties on net sales. Kumquat retains an exclusive option to negotiate for participating in profit-loss sharing in the US.

"We are constantly evaluating innovative approaches to improve outcomes for patients, focusing on areas of high unmet medical need," said Juergen Eckhardt, M.D., Head of Business Development and Licensing at Bayer’s Pharmaceuticals Division. "We look forward to collaborating with Kumquat, an accomplished team of experts with deep KRAS insights. Our intent is to explore the development of a potential new treatment option for patients, while further complementing Bayer’s robust early precision oncology pipeline."

Oncogenic driver mutations, such as KRAS mutations, are changes in the DNA of genes that drive the development and growth of cancer. These mutations are often identified as key targets for cancer treatment, and their identification offers the opportunity to develop target-specific drugs. KRAS G12D mutations are found most frequently in 37 percent of pancreatic ductal adenocarcinoma (PDAC), 13 percent of colorectal cancer and 4 percent of non-small cell lung cancers. PDAC is the most common type of pancreatic cancer (accounting for 85 percent of cases) and remains one of the most difficult tumors to treat, with patients having few treatment options beyond chemotherapy and the five-year survival rate being less than 10 percent. Pancreatic cancer is the sixth leading cause of cancer-related death worldwide. The incidence continues to rise annually, with projections indicating a 95.4 percent increase in new cases by 2050, potentially reaching a total of 998,663 new cases globally.4

"KRAS mutations are crucial for cancer development and can be targeted with specific therapies in a more selective manner," said Dominik Ruettinger, M.D., Ph.D., Global Head of Research and Early Development for Oncology at Bayer’s Pharmaceuticals Division. "KRAS mutations occur in nearly 25 percent of human cancers, yet the most prevalent and oncogenic KRAS (G12D) variant still lacks effective treatment options. We look forward to exploring the investigational KRAS G12D inhibitor, which targets a highly relevant signaling pathway that promotes tumor growth and survival."

"Since pioneering the direct targeting of KRAS G12C mutation over a decade ago, we have continued to discover innovative strategies to target other KRAS mutants, including KRAS G12D," said Yi Liu, Chief Executive Officer of Kumquat. "Advancing our novel KRAS G12D asset into the clinic reflects our commitment to delivering durable therapies for KRAS patients suffering from deadly malignancies such as pancreatic, lung and colorectal cancers. This collaboration with Bayer validates the strength of our platform and the potential of our KRAS G12D candidate to address long-standing unmet needs in oncology. We are thrilled to collaborate with Bayer, who shares our vision and strategy for realizing the benefit of small molecule-based transformative treatments. While advancing optimally our KRAS G12D program through the clinic, this collaboration provides Kumquat the financial resources to accelerate its broader clinical pipeline for long-term value, and position Kumquat to deliver life-changing medicines and achieve sustained growth in the coming years."

On August 12, 2025 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived off-the-shelf cellular immunotherapies to patients, reported business highlights and financial results for the second quarter ended June 30, 2025 (Press release, Fate Therapeutics, AUG 12, 2025, View Source [SID1234655134]).

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"We begin the second half of the year with meaningful progress across our clinical programs as we continue our mission to make cell therapies accessible to all. Our priority remains focused on driving patient enrollment to demonstrate both the therapeutic differentiation and unique on-demand availability of FT819 in autoimmune diseases. We remain encouraged by the promising FT819 data in SLE and LN we reported this past quarter, showing significant disease improvement with less-intensive or no conditioning, and have made strides in expanding our trial sites and accelerating enrollment. Building on this momentum, we are also working closely with the FDA under our RMAT designation with the goal of commencing our registrational study for FT819 in SLE and LN in 2026," said Bob Valamehr, Ph.D., MBA, President and Chief Executive Officer of Fate Therapeutics. "Additionally, we continue to strengthen our broader pipeline programs with an extended partnership with Ono Pharmaceuticals, and advancements in bringing our next-generation, off-the-shelf CAR T cells with Sword and Shield technology toward the clinic. Operationally, we have taken proactive steps to optimize our resource allocation and extend our cash runway, positioning us well to continue executing across our pipeline, working to bring transformative off-the-shelf cellular immunotherapies to patients with unmet needs."

FT819 iPSC-derived off-the-shelf CAR T-cell program in autoimmune disease

In discussion with the FDA on potential registrational study design in moderate-to-severe SLE and refractory LN. In August, the Company met with the U.S. Food and Drug Administration (FDA) under its Regenerative Medicine Advanced Therapy (RMAT) designation for FT819 to seek preliminary feedback on a proposed registrational study design to support regulatory approval in moderate-to-severe SLE and refractory LN. In April 2025, the Company was granted RMAT designation by the FDA for FT819 to treat moderate-to-severe SLE, including LN. Established under the 21st Century Cures Act, the RMAT designation program was created to expedite the development and review of regenerative medicine therapies for serious or life-threatening diseases or conditions.
Interim Phase 1 SLE data using fludarabine-free conditioning regimen presented at EULAR congress and patient enrollment ongoing. The Phase 1 clinical trial of FT819 for the treatment of patients with moderate-to-severe SLE, including patients with LN and with extrarenal lupus (NCT06308978), continues enrolling patients at two dose levels – a single dose of 360 million cells and a single dose of 900 million cells. The Company intends to identify a recommended dose for a registration enabling study and continues to expand clinical site activation in the U.S. and entry into European Union and United Kingdom to broaden geographic reach. At the European Alliance of Associations for Rheumatology (EULAR) 2025 Congress in June, interim Phase 1 data from patients with moderate-to-severe SLE with or without LN using a fludarabine-free conditioning regimen was presented. Three patients with refractory active LN (median prior therapies = 8 [7-8]) were treated with a single dose of FT819 at 360 million cells following a fludarabine (flu)-free conditioning regimen. As of the data cut-off date of May 15, 2025, all three patients achieved an objective renal response. The first LN patient achieved DORIS as well as complete renal response at 6 months, which was also noted at 12-month follow up. Additionally, one patient with refractory extrarenal lupus (prior therapies = 6, including cyclophosphamide; SLEDAI-2K = 18) was treated with a single dose of FT819 at 900 million cells and a single dose of cyclophosphamide. The patient was evaluable for 1-month follow-up, demonstrating improvement across multiple disease-specific scores including an 8-point reduction in SLEDAI-2K from baseline and a 1-point reduction in physician’s global assessment (PGA).
First SLE patient treated as add-on to standard-of-care maintenance therapy. The Company’s Phase 1 SLE study is also designed to assess the safety, pharmacokinetics, and anti-B cell activity of FT819 as an add-on to maintenance therapy without conditioning chemotherapy. At the EULAR Congress in June, the Company reported that the first patient treated while on maintenance therapy, a stable dose of mycophenolate mofetil and steroids for the treatment of refractory extrarenal lupus, received a single dose of FT819 at 360 million cells as an add-on to maintenance therapy (prior therapies = 5). As of the data cut-off date of May 15, 2025, the patient achieved low lupus disease activity state (LLDAS) at 3- and 6-months following administration of FT819 in the absence of conditioning. The patient also experienced a reduction in SLEDAI-2K to 2 from 8 at baseline and in PGA to 0.5 from 2 at baseline, with tapering of steroid dose to less than 5 mg / day. Patient enrollment is ongoing with the aim of investigating patient outcome with single- or multiple-doses of FT819 within a treatment cycle.
Phase 1 SLE study amended to include additional B cell-mediated autoimmune diseases. The Company has expanded its current Phase 1 clinical trial of FT819 to include clinical investigation of multiple B cell-mediated autoimmune diseases, with plans to initiate independent dose-expansion cohorts in the second half of 2025 for the treatment of anti-neutrophil cytoplasmic antibody-associated vasculitis (AAV), idiopathic inflammatory myositis (IIM), and systemic sclerosis (SSc).
FT825 / ONO-8250 iPSC-derived off-the-shelf CAR T-cell Program in Solid Tumors

Phase 1 study ongoing for advanced solid tumors. Under its collaboration with Ono Pharmaceutical Co., Ltd. (Ono), the Company is conducting a multi-center, Phase 1 study to assess the safety, pharmacokinetics, and activity of FT825 / ONO-8250, a multiplex-engineered CAR T-cell product candidate targeting human epidermal growth factor receptor 2 (HER2), in patients with advanced solid tumors (NCT06241456). The study now includes fresh-biopsy testing for HER2 expression to ensure patient stratification and eligibility based on HER2 status. Dose escalation is currently ongoing at the third dose level of 900 million cells, with each patient administered conditioning chemotherapy and a single dose of FT825 / ONO-8250 either as monotherapy or in combination with epidermal growth factor receptor (EGFR)-targeted monoclonal antibody therapy. FT825 / ONO-8250 has demonstrated a favorable safety profile with no dose-limiting toxicities (DLTs) to date.
Next-generation iPSC-derived off-the-shelf CAR T-cell Programs with Novel Sword & Shield Technology Designed to Reduce or Eliminate the Need for Conditioning Chemotherapy

IND allowance by FDA for FT836 MICA/B-targeted CAR T-cell program. In July, the FDA allowed the Company’s investigational new drug (IND) application to initiate Phase 1 clinical testing of FT836, a multiplex-engineered CAR T-cell product candidate uniquely targeting major histocompatibility complex (MHC) proteins A (MICA) and B (MICB) which are expressed on many types of cancer cells with limited detection on healthy tissue. The Phase 1 study is designed to assess the safety and activity of FT836 without administration of conditioning chemotherapy for the treatment of advanced solid tumors. The development of FT836 is supported by a $4 million award from the California Institute of Regenerative Medicine (CIRM).
Creation of master iPSC bank for FT839 dual-CAR T-cell program. FT839 is a CD19/CD38 dual CAR T-cell product candidate designed to target an array of aberrant immune cells. At the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting in May, the Company presented preclinical data demonstrating robust eradication of aberrant CD19+ B cells, CD38+ plasma cells, and CD38+ activated T cells by FT839 using unmatched peripheral blood mononuclear cells sourced from a patient with autoimmune disease. The Company has generated a master iPSC bank for conduct of further preclinical and IND-enabling studies, and is currently evaluating opportunities for clinical investigation of FT839 in hematological malignancies and autoimmunity, beginning in 2026.
Other Corporate Updates

Extension of Ono collaboration for second solid tumor CAR T-cell product candidate. Under its collaboration with Ono, the Company is conducting preclinical development of a second iPSC-derived CAR T-cell candidate targeting an undisclosed solid tumor antigen. Based on a review of the preclinical data package for the collaboration candidate in June, the Company and Ono agreed to extend the collaboration’s research term and continue further preclinical development of the candidate. The Company expects to continue to receive co-funding from Ono in connection with its preclinical development activities under the joint research plan through at least June 2026.
Operating runway extension. The Company has implemented a tactical operations plan that is expected to extend funding of its operations through the end of 2027, which is intended to enable the achievement of key clinical and collaboration milestones while maintaining sufficient funds to support ongoing operations beyond those milestones. The cash runway extension includes the pipeline prioritization of its iPSC-derived CAR T-cell programs, a 12% reduction in its current employee headcount, and cost saving measures across the organization.
Second Quarter 2025 Financial Results

Cash & Investment Position: Cash, cash equivalents, and investments as of June 30, 2025 were $248.9 million.
Total Revenue: Revenue was $1.9 million for the second quarter of 2025, which was derived from the conduct of preclinical development activities for a second collaboration candidate targeting an undisclosed solid tumor antigen under the Company’s collaboration with Ono Pharmaceutical.
Total Operating Expenses: Total operating expenses were $38.9 million for the second quarter of 2025, including research and development expenses of $27.4 million and general and administrative expenses of $11.4 million. Such amount included $7.2 million of non-cash stock-based compensation expense.
Shares Outstanding: As of June 30, 2025, common shares outstanding were 114.7 million, pre-funded warrants outstanding were 3.9 million, and preferred shares outstanding were 2.8 million. Each preferred share is convertible into five common shares.