On August 12, 2025 Rakovina Therapeutics Inc. ("Rakovina" or the "Company") (TSX-V: RKV) (FSE: 7JO0), a biopharmaceutical company advancing cancer therapies through artificial intelligence (AI)-powered drug discovery and NanoPalm Ltd., a Saudi-based biotechnology company pioneering advanced patterned lipid nanoparticle (pLNP) delivery systems, reported the signing of a non-binding Letter of Intent (LOI) (Press release, Rakovina Therapeutics, AUG 12, 2025, View Source;utm_medium=rss&utm_campaign=rakovina-therapeutics-and-nanopalm-ltd-sign-letter-of-intent-to-form-joint-venture-leveraging-al-discovered-oncology-therapies-and-novel-lipid-nanoparticle-delivery-technologies [SID1234655206]). The LOI outlines the intention to form a joint venture to co-develop novel small-molecule oncology therapeutics, beginning with loading Rakovina’s dual-function PARP-HDAC inhibitor, kt3283, into Nano Palm’s pLNP technology.

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The collaboration brings together two novel platforms: Rakovina’s proprietary Al-enabled drug discovery engine, powered by an exclusive license to the proprietary Deep Docking platform, is capable of rapidly screening billions of molecules against validated cancer targets and predicting drug-like characteristics in silico, and NanoPalm’s next-generation self-targeting pLNP delivery system, Al-designed to enhance the precision and efficacy of therapeutic delivery. Promising candidates discovered through Rakovina’s platform and formulated with Nanopalm’s pLNP technology will be validated through Rakovina’s in-house pharmacology, toxicology, and in vivo models, creating a seamless path from in silico modeling to formulation to preclinical proof of concept.

kt-3283, the initial development candidate under the proposed joint venture, is a potent small-molecule inhibitor that simultaneously targets PARP and HDAC – two critical cancer survival pathways. Preclinical studies have demonstrated that kt-3283 induces DNA damage, disrupts cell-cycle progression, and produces enhanced cytotoxicity compared to currently approved PARP and HDAC inhibitors. The compound has shown particularly strong activity in Ewing sarcoma, breast, and ovarian cancer models, with data presented at major oncology conferences and published in a peer-reviewed journal.

NanoPalm’s pLNP technology, originally developed for gene therapy, uses Al-assisted formulation design and tunable shape & surface characteristics to improve tissue specificity, intracellular delivery, and systemic tolerability. This collaboration represents an initial oncology application of NanoPalm’s proprietary platform to small-molecule therapeutics, offering the potential to significantly enhance kt-3283’s therapeutic index and tumor-specific delivery. NanoPalm’s pLNP platform has been validated in multiple preclinical gene therapy programs, demonstrating superior delivery precision and biodistribution compared to conventional LNPs.

NanoPalm’s technology is differentiated by its Al-assisted design and modular architecture, enabling tunable surface properties and selective cellular uptake. Originally developed for gene therapy applications, this collaboration represents NanoPalm’s strategic entry into the oncology field.

"This collaboration brings together complementary expertise in drug discovery and delivery technologies," said Jeffrey Bacha, Executive Chairman of Rakovina Therapeutics. "kt-3283 represents the first of what we believe could be multiple novel therapeutics that emerge from this alliance, offering a robust pipeline for development and future commercialization."

"We are excited to partner with Rakovina Therapeutics to bring our precision pLNP delivery technology into oncology for the first time," said Dr. Ali Alhasan, Founder and CEO of NanoPalm. "Our platform is uniquely suited to improve the delivery profile of small-molecule therapies, and the synergy with Rakovina’s innovative discovery capabilities creates an opportunity for the potential joint venture to pursue worldwide development and commercialization opportunities within Saudi Arabia as its operational hub to develop transformative treatments for patients with cancer. This aligns with the Kingdom’s National Biotechnology Strategy, leveraging the local fertile ground for innovating and accelerating local biotech development."

Once finalized, the joint venture will initially focus on advancing kt-3283 through late preclinical development and early clinical studies while evaluating additional Al-derived drug candidates for inclusion in the development pipeline. The partners also plan to explore global licensing and strategic commercialization opportunities as the pipeline matures.

Defined Responsibilities and Global Scope

Under the proposed joint venture:

Rakovina will contribute kt-3283 drug substance and additional select Al-discovered small-molecule drug candidates, along with supporting in vitro and in vivo validation, pharmacology, toxicology, CMC data, and regulatory expertise.
NanoPalm will contribute its proprietary pLNP platform, biomanufacturing capabilities, delivery science expertise, and preclinical and clinical support infrastructure in Saudi Arabia.
Joint development activities will be coordinated under a mutually agreed governance structure, with initial preclinical validation conducted in Rakovina’s research facilities at the University of British Columbia and manufacturing and biodistribution studies conducted by NanoPalm in Saudi Arabia.
The partners will co-fund the joint venture, pursue third-party investment and non-dilutive grant opportunities, and share equally in ownership, risk, and upside.
Global Reach and Strategic Alignment

The joint venture will be domiciled in Saudi Arabia, with a worldwide development and commercialization mandate. This JV will be the first pLNP-oncology-focused biotech co-founded in Saudi Arabia integrating locally developed nanomedicine with global drug discovery platforms. Development activities will be aligned with international regulatory standards, including those of the FDA, SFDA, and EMA. The parties have committed to a non-exclusive collaboration model, with exclusivity defined for programs combining Rakovina’s compounds with NanoPalm’s delivery technology in oncology and other mutually agreed indications.

Intellectual property arising from the collaboration will be jointly owned, with background IP and technology improvements retained by their respective inventors. Each party will non-exclusively license its IP to the JV for the purpose of fulfilling the venture’s objectives.

On August 12, 2025 Adagene Inc. ("Adagene" or the "Company") (Nasdaq: ADAG), a platform-driven, clinical-stage biotechnology company transforming the discovery and development of novel antibody-based therapies, reported financial results for the six months ended June 30, 2025 and provided corporate updates (Press release, Adagene, AUG 12, 2025, View Source [SID1234655122]).

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"The first half of 2025 was tremendously important for Adagene, as we shared the early overall survival benefit with ADG126 in combination with Merck’s (known as MSD outside of the US and Canada) anti-PD-1 therapy, KEYTRUDA (pembrolizumab) that exceeds standard of care and is highly competitive with data from other products in development. The safety and tolerability data from ADG126 in combination with pembrolizumab in our Phase 1b/2 study in microsatellite stable colorectal cancer contributes to a large body of growing evidence that the power of CTLA-4 inhibition can be harnessed more safely with our approach utilizing conditional activation in the tumor microenvironment. Grade 3 treatment-related adverse events were less than 20%, which is an outstanding achievement given that ADG126 was dosed 10 to 20 times higher than the approved CTLA-4 inhibitors, in order to drive dose-dependent depletion of regulatory T-cells at the desired level inside tumors," said Peter Luo, Ph.D., Chairman, CEO and President of R&D at Adagene. "Now that we have aligned with the FDA on Phase 2 and Phase 3 design elements, which do not require an ADG126 monotherapy arm, we have a clear line of sight into what will be required for regulatory approval. We look forward to initiating the Phase 2 study later this year."

PIPELINE HIGHLIGHTS

ADG126 – Phase 1b/2 data and regulatory update:

As presented at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Poster #248), mOS for the 10 mg/kg cohorts was 19.4 months in microsatellite stable colorectal cancer (MSS CRC) patients free of liver metastasis (NLM), comparing favorably with historical fruquintinib benchmarks of 10.8 months from the FRESCOi study and 12.1 months from the FRESCO-2ii study for the same NLM patient population. Only 1 out of 41 patients in the 10 mg/kg cohorts was censored due to early withdrawal within the first 12 months. Median OS for the 20 mg/kg cohorts has not yet been reached. ADG126 showed a 29% confirmed overall response rate (ORR) in MSS CRC. ADG126 can be dosed at 20 mg/kg Q6W in combination with KEYTRUDA (200 mg, Q3W) with less than 20% Grade 3 adverse events having been observed. All six responders in the 20 mg/kg cohorts remain on treatment, with four patients on study for over one year.

As recently reported, Adagene has now gained alignment with the FDA on Phase 2 and Phase 3 trial design elements for ADG126, and the Company plans to begin enrollment in 2H 2025. In addition, Adagene has initiated evaluation of ADG126 plus pembrolizumab in combination with standard of care in MSS CRC patients, as approved by the Merck/Adagene joint development committee and supported by the 2021 supply agreement between the two partners.

MAJOR COLLABORATIONS

Sanofi: In July, Sanofi agreed to make a strategic investment of up to US$25 million in Adagene. The Company plans to use the proceeds to fund its research and development activities, including clinical development of ADG126, through a randomized Phase 2 trial in MSS CRC. To further explore the clinical potential of ADG126, Adagene will supply Sanofi with ADG126 to evaluate the safety, efficacy, pharmacokinetics and biomarker data in combination with other anticancer therapies in over 100 patients in a Phase 1b/2 clinical trial in advanced solid tumors. Adagene continues to own worldwide commercial rights to ADG126.

Sanofi has also exercised its option to select a third SAFEbody discovery program, utilizing Adagene’s proprietary masking technology and antibody engineering expertise. The bispecific therapeutic, with undisclosed targets, will be engineered by Adagene and induces an option exercise fee, as well as milestones and royalties as per the 2022 partnership agreement with Adagene.

Exelixis: Including upfront and other milestone payments, Adagene has received over US$18 million in total from Exelixis to date, under a technology license agreement to develop novel masked antibody-drug conjugate candidates.

ConjugateBio: In July 2025, Adagene entered into a partnering agreement with ConjugateBio to develop novel antibody drug conjugates. Adagene will provide ConjugateBio with a proprietary antibody for use in partner companies’ bispecific ADC development programs.

CORPORATE UPDATES

In April, Adagene appointed John Maraganore, Ph.D. as Executive Advisor to provide strategic guidance, and to contribute to Adagene’s growth, value creation and benefit for patients.

In May, Mickael Chane-Du joined Adagene as Chief Strategy Officer to promote and advance Adagene’s financing, internal strategic planning and external business development efforts.

FINANCIAL HIGHLIGHTS

Cash and Cash Equivalents:

Cash and cash equivalents were US$62.8 million as of June 30, 2025, compared to US$85.2 million as of December 31, 2024. Total borrowings from commercial banks in China (denominated in RMB) decreased to US$6.6 million as of June 30, 2025 from US$18.2 million as of December 31, 2024. Further, the cash balance as of June 30, 2025 does not include any equity proceed received from Sanofi in July 2025.

Research and Development (R&D) Expenses:

R&D expenses were US$12.0 million for the six months ended June 30, 2025, compared to US$14.7 million for the same period in 2024. The decrease of approximately 18% in R&D expenses reflects clinical focus on and prioritization of the company’s masked, anti-CTLA-4 SAFEbody ADG126.

Administrative Expenses:

Administrative expenses were US$3.7 million for the six months ended June 30, 2025, compared to US$3.6 million for the same period in 2024.

Net Loss:

Net loss attributable to Adagene Inc.’s shareholders was US$13.5 million for the six months ended June 30, 2025, compared to US$17.0 million for the same period in 2024.

Ordinary Shares Outstanding:

As of June 30, 2025, there were 58,914,087 ordinary shares issued and outstanding. Each American depository share, or ADS, represents one and one quarter (1.25) ordinary shares of the company.

Non-GAAP Net Loss:

Non-GAAP net loss, which is defined as net loss attributable to ordinary shareholders for the period after excluding share-based compensation expenses, was US$11.4 million for the six months ended June 30, 2025, compared to US$14.5 million for the same period in 2024. Please refer to the section in this press release titled "Reconciliation of GAAP and Non-GAAP Results" for details.

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 ADC Therapeutics SA (NYSE: ADCT), a commercial-stage global leader and pioneer in the field of antibody drug conjugates (ADCs), reported financial results for the second quarter ended June 30, 2025, and provided operational updates (Press release, ADC Therapeutics, AUG 12, 2025, View Source [SID1234655123]).

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"Entering the second half of 2025, we have streamlined our strategic focus and strengthened our financial foundation, which now allows us to pursue multiple promising opportunities to expand ZYNLONTA into earlier lines of therapy in DLBCL and indolent lymphomas," said Ameet Mallik, Chief Executive Officer of ADC Therapeutics. "We recently shared impressive efficacy data from our LOTIS-7 study of ZYNLONTA plus glofitamab in patients with relapsed or refractory DLBCL and have additional key clinical milestones anticipated through 2026. These milestones include LOTIS-5 achieving the prespecified PFS event target this year and a ZYNLONTA sBLA filing anticipated in 2026, in addition to ongoing Phase 2 investigator-initiated trials in indolent lymphomas. We remain committed to executing our strategy with discipline as we pursue the substantially larger therapeutic opportunity for ZYNLONTA."

Second Quarter 2025 Operational Updates & Recent Highlights

•Completed private investment in public equity (PIPE) financing extending expected cash runway to 2028. The Company entered into a securities purchase agreement for the sale of its equity securities to certain institutional investors in a $100 million PIPE financing, of which the net proceeds of $93.1 million are anticipated to fund multiple catalysts supporting ZYNLONTA’s clinical development and commercialization activities.
•LOTIS-7 data presentations at the European Hematology Association (EHA) (Free EHA Whitepaper) 2025 Congress (EHA2025) and the 18th International Conference on Malignant Lymphoma (ICML) highlighted high response rates and manageable safety and tolerability of ZYNLONTA plus glofitamab (COLUMVI) in patients with relapsed/refractory (r/r) diffuse large B-cell lymphoma (DLBCL). As of the April 2025 cutoff, data from the Phase 1b clinical trial showed an overall response rate (ORR) of 93.3% and a complete response (CR) of 86.7% among the 30 efficacy evaluable patients enrolled in the study. Among the 41 safety evaluable patients, the combination was generally well tolerated with a manageable safety profile and no dose-limiting toxicities across dose levels. The Company expects to engage with the U.S. Food and Drug Administration (FDA) and provide an update on the LOTIS-7 trial in the second half of 2025. Once sufficient data with longer follow-up is available, the Company plans to pursue publication and compendia inclusion in the first half of 2027.

•LOTIS-5 remains on track to reach prespecified progression-free survival (PFS) events by the end of 2025. After the prespecified number of PFS events is reached and data are available, the Company expects to provide topline data on the Phase 3 confirmatory trial evaluating ZYNLONTA in combination with rituximab in patients with 2L+ DLBCL. A potential supplemental Biologics License Application (sBLA) submission to regulatory authorities is anticipated in the first half of 2026, with potential confirmatory approval in 2L+ DLBCL and publication and compendia inclusion in the first half of 2027.
•Updated data from the investigator-initiated trial presented at ICML demonstrated the potential of ZYNLONTA as a monotherapy in r/r marginal zone lymphoma. The updated data presented by Izidore S. Lossos, MD, Chief, Division of Hematology Lymphoma Section, at Sylvester Comprehensive Cancer Center, part of the University of Miami Miller School of Medicine, demonstrated an ORR of 84.6% (22/26) and a CR of 69.2% (18/26) with a manageable safety profile. The Phase 2, single-arm, open-label, multicenter trial is being conducted at the Sylvester Comprehensive Cancer Center, City of Hope, Emory Winship Cancer Institute and Vanderbilt-Ingram Cancer Center. The Company plans to assess a potential regulatory pathway. In addition, once sufficient data is available, a potential publication and compendia inclusion is anticipated in the first half of 2027.
•IND-enabling activities advancing for PSMA-targeting ADC. IND-enabling activities are underway for the Company’s exatecan-based, prostate-specific membrane antigen (PSMA)-targeting ADC, which has been selected for advancement. Completion of these activities is expected by the end of 2025.
•Announced strategic restructuring and prioritization plan, discontinuing early development efforts for the remaining preclinical programs in solid tumors and focusing on ZYNLONTA. As research and development efforts and related programs are closed out, the Company plans to shut down its UK facility and reduce the global workforce across functions by approximately 30%, which is expected to be substantially completed by September 30, 2025.

Second Quarter and First Half 2025 Financial Results

•Product Revenues: Net product revenues were $18.1 million for the second quarter ended June 30, 2025, and $35.5 million for the first six months of 2025 as compared to $17.0 million and $34.9 million for the same periods in 2024. The period-over-period changes were primarily driven by higher sales price and variability in sales volume.
•Research and Development (R&D) Expense: R&D expense was $30.1 million for the three months ended June 30, 2025, and $59.0 million for the six months ended June 30, 2025, as compared to $24.3 million and $50.0 million for the same periods in 2024. The increases in R&D costs were driven by timing and enrollment of our ZYNLONTA clinical trials LOTIS-5 and LOTIS-7, and an increase in IND-enabling activities for our PSMA-targeting ADC. These increases were partially offset by a reduction in spending on discontinued programs.
•Selling and Marketing (S&M) Expense: S&M expense was $10.1 million and $20.7 million for the three and six months ended June 30, 2025, respectively, compared to $10.7 million and $22.1 million for the same periods in 2024. The period-over-period decreases were primarily due to a reduction in marketing and advertising expenses.
•General & Administrative (G&A) Expense: G&A expense was $8.8 million and $18.8 million for the three and six months ended June 30, 2025, respectively, compared to $10.2 million and $22.7 million for the same periods in 2024. The reductions in G&A expense were primarily due to lower external professional fees.
•Restructuring, impairment and other related costs: In connection with the strategic reprioritization and restructuring plan announced in June 2025, the Company incurred $13.1 million in restructuring and impairment costs for the three and six months ended June 30, 2025, which consisted of $6.7 million in employee severance and related benefit costs, and $6.4 million in non-cash impairment of assets in connection with the close down of the UK facility.
•Net Loss: Net loss for the quarter ended June 30, 2025, was $56.6 million, or a net loss of $0.50 per basic and diluted share, as compared to a net loss of $36.5 million, or a net loss of $0.38 per basic and diluted share, for the same period in 2024. Net loss for the six months ended June 30, 2025 was $95.2 million, or a net loss of $0.86 per basic and diluted share, as compared to a net loss of $83.2 million, or a net loss of $0.93 per basic and diluted share for the six months ended June 30, 2024. The higher net loss of the three- and six-month periods are primarily due to the increase in R&D expense and the restructuring, impairment and related costs incurred in connection with the strategic reprioritization and restructuring plan.
•Adjusted Net Loss: Adjusted net loss, which is a non-GAAP financial measure, was $28.7 million, or an adjusted net loss of $0.25 per basic and diluted share for the quarter ended June 30, 2025, as compared to adjusted net loss of $24.4 million, or $0.25 per basic and diluted share, for the same period in 2024. Adjusted net loss for the six months ended June 30, 2025, was $52.6 million, or an adjusted net loss of $0.48 per basic and diluted share, as compared to net loss of $55.5 million, or an adjusted net loss of $0.62 per basic and diluted share for the six months ended June 30, 2024. The increase in adjusted net loss for the three-month period is due to higher R&D costs. The decrease in adjusted net loss per share for the six-month period is primarily attributable to a higher number of weighted average shares outstanding.
•Cash and cash equivalents: As of June 30, 2025, cash and cash equivalents were $264.6 million, compared to $250.9 million as of December 31, 2024. In June 2025, the Company entered into securities purchase agreements for the sale of its equity securities to certain institutional investors in a $100.0 million PIPE financing, which resulted in net proceeds of $93.1 million, extending the expected cash runway into 2028.

Conference Call Details

ADC Therapeutics management will host a conference call and live audio webcast to discuss second quarter 2025 financial results and provide a company update today at 8:30 a.m. Eastern Time. To access the conference call, please register here. Registrants will receive the dial-in number and unique PIN. It is recommended that you join 10 minutes before the event, though you may pre-register at any time. A live webcast of the call will be available under "Events & Presentations" in the Investors section of the ADC Therapeutics website at ir.adctherapeutics.com. The archived webcast will be available for 30 days following the call.

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