On November 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 third quarter ended September 30, 2025.

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"Through three quarters of launch, we are encouraged by our progress to increase the overall market in r/r B-ALL, reaching patients who previously may not have been considered for CAR T therapy. With mounting experience we see physician enthusiasm for AUCATZYL increasing, validated by real world data from the ROCCA Consortium to be presented at the ASH (Free ASH Whitepaper) Annual Meeting in December," said Dr. Christian Itin, Chief Executive Officer of Autolus. "Despite an expected temporary lag in Q3 sales based on the change in CMS reimbursement policy that occurred in Q2, we executed well on new patient starts and project a strong full year of sales."

Dr. Itin continued, "Building on our strong commercial and manufacturing performance, we enter our next phase of growth for obe-cel focused on three key objectives. First, increasing market share within the indicated adult ALL population; second, based on strong Phase 1 data sets we are conducting potential pivotal studies in pediatric ALL and in severe lupus nephritis to broaden the utility and commercial opportunity of obe-cel; and finally, we continue to innovate on manufacturing technology as a foundation for further expansion of access to CAR T therapies."

Product and Pipeline Updates:

AUCATZYL Launch
Autolus reported net product revenue of $21.1 million for the three months ended September 30, 2025 and deferred revenue of $7.6 million as of September 30, 2025.
The Company has 60 centers fully activated in the U.S. as of November 12, 2025, achieving the target of 60 activated centers prior to year-end.
Patient access to AUCATZYL continues to increase, with coverage secured for greater than 90% of total U.S. medical lives.
Data from the ROCCA (Real-World Outcomes Collaborative for CAR T in Adult ALL) database evaluating patient characteristics, toxicity and response after real-world administration of AUCATZYL (obecabtagene autoleucel) and TECARTUS (brexucabtagene autoleucel) for relapsed acute lymphoblastic leukemia with r/r ALL will be presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting.

Obe-cel data in pediatric r/r B-ALL
Data from the ongoing Phase Ib/II CATULUS study evaluating the safety and efficacy of obe-cel in patients under 18 years with CD19-positive r/r B-ALL or B-cell Non-Hodgkin Lymphoma (NHL) will be presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. Data show the safety profile of obe-cel in pediatric patients is consistent with that previously reported in adults, with low rates of high-grade cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS). Overall response rate (ORR) was high at 95% and nearly 90% of responders had ongoing remission at data cut-off. Additional data will be presented in a poster presentation at the ASH (Free ASH Whitepaper) Annual Meeting on December 7, 2025, from 6:00 – 8:00pm ET.
In October 2025, the U.S. Food and Drug Administration (FDA) granted regenerative medicine advanced therapy (RMAT) designation to obe-cel for the treatment of pediatric patients with r/r B-ALL. The RMAT designation is a program created under the 21st Century Cures Act to accelerate development and regulatory review of regenerative medicine therapies, including cell therapies, intended to treat serious or life-threatening diseases.

Obe-cel in lupus nephritis (LN)
Data from the Phase 1 CARLYSLE clinical trial were presented on October 28, 2025, at the American College of Rheumatology (ACR) Convergence 2025. Data in severe refractory systemic lupus erythematosus (srSLE) suggests obe-cel is well tolerated with no ICANS or high-grade CRS. Preliminary efficacy data demonstrate achievement of definition of remission in SLE (DORIS) in 83% (n=5/6) of patients and complete renal response (CRR) in 50% (n=3/6) of patients. All responses and remissions are ongoing with no evidence of disease activity at a median follow up of 8.9 months (range 6-13.8 months).
Additional findings from the ongoing CARLYSLE study will be presented in an oral presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2025 on December 8, 2025, at 11:30am ET. Data show the B-cell reconstitution profiles suggest that obe-cel may induce a reset of pathologic autoimmunity. Updated Phase 1 data with longer follow-up, and data in patients who received 100×106 CAR T-cells will be presented.
The Company has previously aligned with the FDA on the Phase 2 trial design and potential registrational path to approval. Data to date supported progressing into the Phase 2 LUMINA trial. The first patient is expected to be dosed prior to year-end 2025.

Obe-cel in progressive MS
Autolus is advancing obe-cel into initial clinical development in progressive MS. The first patient in the BOBCAT trial was dosed in October 2025. The Phase 1 trial, expected to include up to 18 adult patients, will determine the safety, tolerability, and preliminary efficacy of obe-cel in participants with refractory progressive forms of multiple sclerosis. The primary endpoint is to assess safety and tolerability of obe-cel. Key secondary endpoints include evaluating the preliminary efficacy of obe-cel using change from baseline in standard efficacy measures.

Early-stage pipeline programs and collaborations
In November 2025, Moderna announced that the first patient has been dosed in a Phase 1/2 study of mRNA-2808, an investigational mRNA-based T-cell engager for participants with relapsed or refractory multiple myeloma. mRNA-2808 utilizes an Autolus proprietary binder that was licensed to Moderna in 2022.
Long term follow up from the AUTO8 MCARTY study in multiple myeloma will be presented in a poster presentation at the ASH (Free ASH Whitepaper) Annual Meeting:

Abstract ID: abs25-14286
Title: CAR-T cells co-targeting BCMA and CD19 in RRMM: Results from the UKMRA Phase 1 MCARTY trial in relapsed refractory multiple myeloma
Date: December 7, 06:00 PM – 08:00 PM EST
Location: OCCC – West Halls B3-B4
Presenting Author: Lydia Lee, MBBS, MRCP, MRCPath (Haem), PhD

The first patient in the AUTO8 ALARIC study in AL Amyloidosis is expected to be dosed prior to year-end 2025.
Autolus’ translational programs with UCL continue to fuel its early-stage pipeline, providing a cost-efficient path to development to support long-term growth.

Operational Updates:

Autolus announced leadership team changes to support the next phase of commercial growth, margin improvement and market expansion.

Cintia Piccina was appointed U.S. Chief Commercial Officer and Country General Manager to lead ongoing momentum in AUCTAZYL U.S. launch and drive future growth. Cintia brings to Autolus extensive cellular therapy experience having led teams that successfully launched and commercialized multiple products in Novartis, Bluebird/2seventy bio, Allovir and Adaptimmune, where she was most recently the Chief Commercial Officer.
Miranda Neville was appointed Chief Technical Officer to drive manufacturing optimization, succeeding David Brochu who will continue as an advisor. Ms. Neville joined Autolus in 2018 from the consulting firm AllianceBIO where she spent four years as a Partner and supported several clinical stage and commercial biopharmaceutical companies. Ms. Neville began her career at Human Genome Sciences (HGS). She spent 10 years at HGS in a variety of roles including Manufacturing, Engineering & Program Management, prior to its acquisition by GlaxoSmithKline.
Patrick McIlvenny was appointed Senior Vice President, Finance and Chief Accounting Officer. Before joining Autolus, Mr. McIlvenny served as Senior Vice President, Chief Accounting Officer for Horizon Therapeutics plc, until the acquisition of Horizon by Amgen, and in various finance roles of increasing responsibilities at Ardagh Group S.A and Elan Corporation plc. Prior to joining Elan, Mr. McIlvenny worked with PricewaterhouseCoopers and Deloitte. Mr. McIlvenny is a Fellow of the Institute of Chartered Accountants in England and Wales.

Dr. Itin commented, "Our new team members bring a breadth of leadership experience and will focus on market growth, strategic planning and operational excellence driving growth and efficiency of the ALL business. We are grateful to our prior team members who were instrumental in setting our organization on the right course for a successful launch."

Summary of Anticipated News Flow:

ALL: Initial clinical data from CATULUS trial in pediatric ALL December 7, 2025

SLE: Longer-term follow up data from CARLYSLE trial
December 8, 2025

LN: Expect to dose first patient in Phase 2 LUMINA trial By year-end 2025

ALA: Expect to dose first patient in Phase 1 ALARIC trial in AL amyloidosis By year-end 2025

ALL: acute lymphoblastic leukemia
SLE: systemic lupus erythematosus
LN: lupus nephritis
ALA: light-chain amyloidosis

Financial Results for the Quarter Ended September 30, 2025

Product revenue, net for the three months ended September 30, 2025, was $21.1 million. Deferred revenue balance at September 30, 2025, was $7.6 million, representing product that was shipped and received by centers but not yet dosed.

Cost of sales for the three months ended September 30, 2025, totaled $28.6 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, inventory reserves and write-offs and 3rd party royalties for certain technology licenses.

Research and development expenses decreased from $40.3 million to $27.9 million for the three months ended September 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 $27.3 million to $36.3 million for the three months ended September 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 September 30, 2025, was $71.6 million, as compared to $67.9 million for the same period in 2024.

Net loss was $79.1 million for the three months ended September 30, 2025, compared to $82.1 million for the same period in 2024. Basic and diluted net loss per ordinary share for the three months ended September 30, 2025, totaled $(0.30), compared to basic and diluted net loss per ordinary share of $(0.31) for the same period in 2024.

Cash, cash equivalents and marketable securities at September 30, 2025, totaled $367.4 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 $20.1 million in the Company’s 2023 R&D tax credit expected from the UK HMRC.

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

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

September 30
December 31
2025
2024
Assets
Cash and cash equivalents $ 86,124 $ 227,380
Marketable securities – Available-for-sale debt securities $ 281,289 $ 360,643
Total current assets $ 514,577 $ 660,929
Total assets $ 661,947 $ 782,725
Liabilities and shareholders’ equity
Total current liabilities $ 83,071 $ 60,743
Total liabilities $ 396,495 $ 355,400
Total shareholders’ equity $ 265,452 $ 427,325

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

Three Months Ended
September 30, Nine Months Ended
September 30,
2025
2024
2025
2024
Product revenue, net $ 21,144 $ — $ 51,049 $ —
License revenue 50 — 50 10,091
Cost and operating expenses:
Cost of sales (28,643) — (71,039) —
Research and development expenses, net (27,892) (40,323) (82,056) (107,606)
Selling, general and administrative expenses (36,280) (27,330) (96,079) (67,410)
Loss on disposal of property and equipment — (223) (3) (223)
Impairment of operating lease right-of-use assets and related property and equipment — — — (414)
Loss from operations (71,621) (67,876) (198,078) (165,562)
Total other (expenses) income, net (6,965) (14,196) 4,037 (27,428)
Net loss before income tax (78,586) (82,072) (194,041) (192,990)
Income tax expenses (532) (22) (3,155) (66)
Net loss attributable to ordinary shareholders (79,118) (82,094) (197,196) (193,056)
Other comprehensive (loss) income, net of tax (5,782) 27,010 24,254 28,094
Total comprehensive loss $ (84,900) $ (55,084) $ (172,942) $ (164,962)

Basic and diluted net loss per ordinary share $ (0.30) $ (0.31) $ (0.74) $ (0.77)
Weighted-average basic and diluted ordinary
shares 266,141,431 266,084,589 266,136,518 255,480,521

Conference Call
Management will host a conference call and webcast today at 8:30am EST/1:30pm GMT 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

(Press release, Autolus, NOV 12, 2025, View Source [SID1234659806])

On November 12, 2025 Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell immunotherapy, leveraging its novel allogeneic Epstein-Barr virus (EBV) T-cell platform to develop transformative therapies for patients with cancer and autoimmune diseases, reported financial results for the third quarter 2025 and business updates.

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Tabelecleucel (tab-cel or Ebvallo) for Post-Transplant Lymphoproliferative Disease (PTLD)

The U.S. Food and Drug Administration (FDA) has accepted the filing of Atara’s Biologics License Application (BLA) for tabelecleucel (tab-cel) indicated as monotherapy for treatment of adult and pediatric patients two years of age and older with Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) who have received at least one prior therapy. There are no FDA approved therapies in this treatment setting.

The BLA has been granted Priority Review with a Class 2 Resubmission Prescription Drug User Fee Act (PDUFA) target action date of January 10, 2026.

Atara expects to receive an additional $40 million milestone payment from Pierre Fabre Laboratories contingent upon FDA approval of the tab-cel BLA.

In October, Atara completed the transfer of regulatory activities, including BLA sponsorship, to Pierre Fabre Laboratories. Atara will continue to support Pierre Fabre Laboratories, at Pierre Fabre Laboratories expense, with certain regulatory activities related to the BLA. Substantially all operational activities and associated costs related to tab-cel have been transitioned to Pierre Fabre Laboratories.

Corporate Updates

Strategic Alternatives Evaluation: As previously communicated, Atara continues to actively explore and assess potential strategic alternatives with the goal of maximizing shareholder value.

Organizational Restructuring: In October 2025, Atara announced a reduction in its workforce that impacted approximately 29% of its current employees, retaining approximately 15 employees essential to executing on the Company’s strategic priorities.

Financial Update:

Third Quarter 2025 Financial Results:

Cash, cash equivalents and short-term investments as of September 30, 2025, totaled $13.7 million, as compared to $22.3 million as of June 30, 2025.
Net cash used in operating activities was $9.8 million for the third quarter 2025, as compared to $4.0 million in the same period in 2024. Net cash used in operating activities increased by $5.8 million year-over-year, primarily driven by a decrease in cash receipts from Pierre Fabre in the third quarter 2025 after the BLA acceptance milestone and a sale of tab-cel intermediates inventory were completed in the same period in 2024, this was partially offset by a decrease in operating expenses in the third quarter 2025.
Atara reported net loss of $4.3 million, or $0.32 per share for the third quarter 2025 as compared to $21.9 million or $2.93 per share for the same period in 2024.
Total revenues were $3.5 million for the third quarter 2025, as compared to $40.2 million for the same period in 2024. Total revenues decreased by $36.7 million year-over-year, primarily due to the accelerated recognition of deferred revenue and additional upfront and milestone payments received in the same period 2024 as a result of the A&R Commercialization Agreement, effective December 2023. In addition, the decrease is due to the accelerated recognition of deferred revenue in the first and second quarters 2025 following the transition of manufacturing, development and safety responsibilities to Pierre Fabre Laboratories.
Total costs and operating expenses include non-cash stock-based compensation, depreciation and amortization expenses of $1.3 million for the third quarter 2025, as compared to $7.6 million for the same period in 2024.
Research and development expenses were $2.9 million for the third quarter 2025, as compared to $43.9 million for the same period in 2024.
Research and development expenses include $0.3 million of non-cash stock-based compensation expenses for the third quarter 2025, as compared to $2.9 million for the same period in 2024.
General and administrative expenses were $4.0 million for the third quarter 2025, as compared to $10.4 million for the same period in 2024.
General and administrative expenses include $0.9 million of non-cash stock-based compensation expenses for the third quarter 2025, as compared to $3.5 million for the same period in 2024.
2025 Outlook and Cash Runway:

Under its commercialization agreement with Pierre Fabre Medicament, Atara is eligible to receive a $40 million milestone payment upon FDA approval of the tab-cel BLA. In addition, Atara will be eligible to receive double-digit tiered royalties as a percentage of net sales and milestones related to commercial sales of EBVALLO.
We anticipate the full-year 2025 operating expenses will decrease by at least 60% compared to 2024, driven by the transition of substantially all tab-cel activities and associated costs to Pierre Fabre Laboratories as well as the implementation of operational efficiencies in the first half of the year.
Atara projects that cash, cash equivalents and short-term investments as of September 30, 2025, combined with the net proceeds of the milestone payment upon tab-cel BLA approval under its commercialization agreement with Pierre Fabre Medicament, will provide significant cash runway and flexibility for the company to execute on its strategic priorities.

(Press release, Atara Biotherapeutics, NOV 12, 2025, View Source [SID1234659805])

On November 12, 2025 Aprea Therapeutics, Inc. (Nasdaq: APRE) ("Aprea", or the "Company"), a clinical-stage biopharmaceutical company developing innovative treatments that exploit specific cancer cell vulnerabilities while minimizing damage to healthy cells, reported financial results for the third quarter ended September 30, 2025, and provided a business update. The Company reported continued clinical progress across both its WEE1 and ATR inhibitor programs and has cash runway into the fourth quarter of 2026.

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"We are pleased with the continued progress in our development programs, as the emerging data on both of our clinical assets demonstrate evidence of activity," said Oren Gilad, Ph.D., President and Chief Executive Officer of Aprea. "For APR-1051, our WEE1 kinase inhibitor, we’re encouraged by early signs of anti-tumor activity to date in the ongoing ACESOT-1051 trial, including 3 out of 4 patients with stable disease in the 100 mg once daily cohort. We have recently advanced into the 150 mg once daily cohort as dose escalation in this trial continues. For ATR-119, identifying the recommended Phase 2 dose for the once daily (QD) dosing provides a solid foundation for next-stage development, and we are now considering potential combination strategies, with radiation or checkpoint inhibitors, that could expand its clinical impact. These developments reinforce Aprea’s differentiated DDR approach and our commitment to helping patients with value creating clinical catalysts anticipated in 2026."

Key Business Updates and Potential Upcoming Key Milestones

ACESOT-1051: A Biomarker Focused, Phase 1 Trial of Oral WEE1 inhibitor, APR-1051

APR-1051 is a potent and selective small molecule WEE1 inhibitor designed to potentially solve tolerability challenges of the WEE1 class and has the potential to achieve improved clinical activity than other programs currently in development. APR-1051 is being advanced as a monotherapy in biomarker-defined cancers likely to respond to WEE1 inhibition. Among these, cancers over-expressing Cyclin E represent a high unmet medical need. Patients with Cyclin E over-expression have poor prognosis and, currently, lack effective therapies options.
APR-1051 is currently evaluated in the ongoing Phase 1 ACESOT-1051 (A Multi-Center Evaluation of WEE1 Inhibitor in Patients with Advanced Solid Tumors, APR-1051). Results from Dose Level 6 (100 mg once daily), show that 3 out of 4 patients achieved stable disease, per RECIST v1.1 in heavily pretreated gastrointestinal and gynecologic malignancies. Disease stabilization was observed in patients with FBXW7, CCNE1, and KRASG12V + TP53 alterations, molecular profiles known to drive replication stress and WEE1 dependency.
Following successful clearance of the 100 mg cohort, dose escalation has progressed to Dose Level 7 (150 mg once daily) with the goal of identifying doses that maximize therapeutic benefit while maintaining an acceptable safety profile. APR-1051 was manageable with mostly Grade 1 or 2 adverse events, which were mainly gastrointestinal events and fatigue.
A poster titled Early safety and efficacy of APR-1051, a novel WEE1 inhibitor, in patients with cancer-associated gene alterations: Updated data from ACESOT-1051 Phase 1 trial was presented on October 24, 2025at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper). This poster summarizes preliminary results from the trial with a cutoff date of September 17, 2025. A copy is available on the Aprea corporate website here.
Pending additional data, future studies of ACESOT-1051 may evaluate APR-1051 in combination with checkpoint inhibitors to address unmet medical needs across distinct patient populations.
For more information, refer to ClinicalTrials.gov NCT06260514.
ABOYA-119: Ongoing Clinical Trial Evaluating ATR inhibitor, ATRN-119

ATRN-119 is a potent and highly selective first-in-class macrocyclic ATR inhibitor, designed and developed to be used in patients with tumors harboring mutations in DDR-related genes. Cancers with mutations in DDR-related genes represent a high unmet medical need. These patients often have a poor prognosis and currently lack effective therapeutics options.
ATRN-119 is being evaluated in the open-label Phase 1/2a clinical trial (ABOYA-119) as monotherapy in patients with advanced solid tumors having at least one mutation in a defined panel of DDR-related genes. As of September 8, 2025, 43 patients with advanced solid tumors have been enrolled.
Based on results to date, Aprea has determined the recommended Phase 2 dose (RP2D) to be 1,100 mg for the once daily dosing for ATRN-119.
Following RP2D determination Aprea is strategically pausing further enrollment in both once daily and twice daily monotherapy dosing arms of the ABOYA-119 trial to consider combination studies aimed at maximizing therapeutic benefits. Aprea is currently in discussions with leading academic centers to explore combining ATRN-119 with radiation in patients with HPV+ head and neck cancer, an indication where synergistic anti-tumor effects have been observed in preclinical data. Additional investigator-led studies evaluating ATRN-119 in combination with I/O agents and antibody-drug conjugates are also being explored.
A poster titled Updated data from ABOYA-119: A phase 1/2a trial of ATRN-119, a novel macrocyclic ATR inhibitor, in patients with advanced solid tumors harboring DNA damage trial was presented on October 24, 2025 at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper). This poster summarizes preliminary results from the trial with a cutoff date of September 8, 2025. A copy can be found here.
For more information on ABOYA-119, please refer to clinicaltrials.gov NCT04905914.
Select Financial Results for the Third quarter Ended September 30, 2025

As of September 30, 2025, the Company reported cash and cash equivalents of $13.7 million compared to $22.8 million as of December 31, 2024. The Company believes its cash and cash equivalents as of September 30, 2025, will be sufficient to meet its currently projected operating expenses and capital expenditure requirements into the fourth quarter of 2026.
For the third quarter ended September 30, 2025, the Company reported an operating loss of $3.1 million, compared to an operating loss of $4.1 million in the third quarter of 2024.
Research and Development (R&D) expenses were $1.6 million for the quarter ended September 30, 2025, compared to $2.8 million for the third quarter of 2024. The decrease in R&D expenses was primarily related to higher expenses in 2024 related to study start up activities in preparation for enrollment of the first patient into ACESOT-105, our Phase 1 dose-escalation study of APR-1051, and lower expenses in 2025 related to the ABOYA-119 clinical trial to evaluate ATRN-119, our clinical-stage oral small molecule inhibitor of ATR.
General and Administrative (G&A) expenses were $1.5 million for the quarter ended September 30, 2025, compared to $1.6 million for the third quarter of 2024. The decrease in G&A expenses was primarily related to a decrease in insurance costs.
The Company reported a net loss of $3.0 million ($0.47 per basic share) on approximately 6.4 million weighted average common shares outstanding for the quarter ended September 30, 2025, compared to a net loss of $3.8 million ($0.64 per basic share) on approximately 5.9 million weighted average common shares outstanding for the comparable period in 2024.

(Press release, Aprea, NOV 12, 2025, View Source [SID1234659804])

On November 12, 2025 AN2 Therapeutics, Inc. (Nasdaq: ANTX), a biopharmaceutical company focused on discovering and developing novel small molecule therapeutics derived from its boron chemistry platform, reported financial results for the third quarter ended September 30, 2025.

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"This quarter, we continued to execute across a diverse pipeline spanning multiple, potentially high impact projects in infectious diseases and oncology, leveraging our boron chemistry platform to address areas of high unmet need. These programs reflect our commitment to delivering transformative therapies, each having potential to meaningfully add shareholder value within our cash runway," said Eric Easom, Co-Founder, Chairman, President and CEO of AN2 Therapeutics. "Our program studying oral AN2-502998 for Chagas disease is progressing through a Phase 1 first-in-human clinical trial, and planning for a subsequent Phase 2 proof-of-concept study is underway with our collaborators at Drugs for Neglected Diseases initiative (DNDi). In oncology, we expect our two lead programs to enter development in 2026, each with the potential to meaningfully impact solid-tumor cancers. We’ve also entered into a collaboration with GSK focused on TB, where our work is supported by a third year of funding from the Gates Foundation. This initiative reflects our commitment to applying boron chemistry to a disease that continues to impact a quarter of the world’s population."

Third Quarter & Recent Business Updates:

Chagas Disease

Advancing Phase 1 first-in-human clinical trial of oral AN2-502998

The Company is progressing through a Phase 1 first-in-human clinical trial evaluating the safety, tolerability, and pharmacokinetics of oral AN2-502998 in healthy volunteers. Oral AN2-502998 is under development for chronic Chagas disease, an infectious disease caused by the parasite Trypanosoma cruzi (T. cruzi), which affects an estimated 6-7 million people worldwide, including approximately 300,000 in the U.S. and over 100,000 in Europe. AN2-502998 is the only compound of which the Company is aware to have demonstrated curative activity in preclinical studies across multiple species, including in nonhuman primates (NHP) with long-term, naturally acquired chronic infections of diverse T. cruzi genetic types. Because NHP infections are naturally acquired in the environment, this efficacy data may be more predictive of efficacy in human clinical trials than other animal models. The Company anticipates Phase 1 data in the first quarter of 2026 and initiation of a Phase 2 proof-of-concept study in patients with chronic Chagas disease in 2026, depending upon the outcome and timing of completion of the Phase 1 study. Through the Company’s collaboration with DNDi, the company will leverage DNDi’s extensive clinical trial network and expertise in Chagas disease to rapidly advance the clinical development of AN2-502998. There are no FDA approved treatments for adults with chronic Chagas disease.
Boron Chemistry Pipeline

Continuing to advance boron chemistry compounds in oncology

The Company is pursuing a number of oncology targets where we believe boron chemistry offers a competitive advantage in terms of binding-site differentiation, pharmacodynamics, drug-like properties and IP, including initially ENPP1 and PI3Kα. The unique binding modes of boron-containing compounds enable the discovery of inhibitors with high ligand efficiency against targets considered undruggable or difficult to access with traditional chemistry approaches. Boron chemistry has produced first-in-class molecules against a number of targets including CPSF3 (AN2-502998 and acoziborole) and LeuRS (epetraborole, ganfeborole and tavaborole). The Company has discovered preclinical compounds with profiles that are sub-nanomolar, highly selective and with strong oral pharmacokinetics. The Company anticipates advancing the first oncology compound into development in early 2026 with potential clinical proof-of-concept data within the Company’s current cash runway. The Company expects to advance its second oncology compound into development in mid-2026.
Nontuberculous Mycobacteria (NTM) Lung Disease Caused by M. abscessus

M. abscessus program advancing through investigator-initiated trial (IIT)

Building on the microbiological and safety data from the Company’s prior NTM study, the Company believes that epetraborole has the potential to address a critical unmet need in M. abscessus lung disease, one of the most difficult to treat NTM infections for which no FDA-approved therapies exist. The Company is supporting the design of an IIT expected to begin enrollment in early 2026, pending finalization of the trial protocol and regulatory allowance to proceed. The Company anticipates that data from this potential study, if positive, could provide the clinical evidence of human proof of concept in M. abscessus and inform the design of a subsequent pivotal trial. NTM lung disease represents a growing global health concern. It is estimated that approximately 120,000–150,000 people in the U.S. are living with NTM lung disease, of which 10–15% are caused by M. abscessus.
Melioidosis

Preparing for epetraborole’s next phase of development in acute melioidosis, a highly lethal bacterial infection and recognized biothreat

AN2 is developing epetraborole for the treatment of acute melioidosis, a highly lethal bacterial infection and recognized biothreat. The Company completed enrollment in a 200-patient observational trial (non-epetraborole treatment) in October 2024, and in June 2025 announced key insights from the trial, which reinforce the high mortality of the disease, despite standard of care. This study provided critical data which will allow the Company to optimize the design of upcoming clinical studies. Discussions are underway with the U.S. government to fund Phase 2 development of epetraborole in acute melioidosis. Melioidosis has a 90-day mortality rate approaching 40%, despite standard of care (SOC) drugs, including ceftazidime or meropenem. The aim of the program is to meaningfully lower the expected mortality rate by dosing epetraborole on top of standard of care. If approved for the treatment of melioidosis, the Company plans to seek a priority review voucher and could generate revenue from U.S. and other governmental stockpiling, as well as from use as treatment in disease-endemic countries, including the U.S.
Global Health

Research collaboration with GSK to advance boron-based LeuRS-inhibitors targeting TB; AN2 awarded third year funding from Gates Foundation

In November 2025, the Company announced a collaboration with the global biopharma company GSK to develop new therapies for TB. As part of this effort, the Gates Foundation will provide a third year of funding to support AN2’s work within the collaboration. TB continues to pose a major global health challenge, affecting more than a quarter of the world’s population and causing over 1.25 million deaths annually.
Selected Third Quarter Financial Results

Research and Development (R&D) Expenses: R&D expenses for the third quarter of 2025 were $7.0 million, compared to $8.3 million for the same period during 2024 due to decreased clinical trial expenses, personnel-related expenses, chemistry manufacturing and controls expenses, and consulting and outside services, primarily related to termination of the EBO-301 clinical study and corporate restructuring activities in August 2024. These decreases were partially offset by increases in preclinical and research studies and expenses related to start-up activities of the Phase 1 trial in Chagas disease and facility and other expenses.
General and Administrative (G&A) Expenses: G&A expenses for the third quarter of 2025 were $3.0 million, compared to $3.5 million for the same period during 2024 due to decreased personnel-related expenses, professional and outside services expenses, and other expenses.
Restructuring Charges: There were no restructuring charges in the third quarter of 2025. Restructuring charges for the third quarter of 2024 were $2.2 million due to severance payments and other employee termination-related expenses.
Interest Income: Interest income for the third quarter of 2025 was $0.7 million, compared to $1.3 million for the same period in 2024 due to lower cash, cash equivalents and investment balances and lower interest rates in 2025 as compared to 2024.
Net Loss: Net loss for the third quarter of 2025 was $9.4 million, compared to $12.7 million for the same period during 2024.
Cash Position: The Company had cash, cash equivalents and investments of $65.1 million at September 30, 2025. The Company projects that existing cash, cash equivalents and investments will sustain operations into 2028 under the current operating plan.

(Press release, AN2 Therapeutics, NOV 12, 2025, View Source [SID1234659803])

On November 12, 2025 Abeona Therapeutics Inc. (Nasdaq: ABEO) reported financial results and business highlights for the third quarter of 2025 and shared recent operational progress.

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"We are scaling the ZEVASKYN launch to meet patient needs," said Vish Seshadri, Chief Executive Officer of Abeona. "We have strong and growing patient demand. Despite a one-quarter shift in patient starts, we remain steadfast in our 2026 launch goals. Our conviction is built on our expanding treatment site network and powerful momentum from the patient and caregiver community."

Recent Developments

ZEVASKYN (prademagene zamikeracel)

● Product release assay optimized; patient treatment anticipated to start in 4Q’25: During the third quarter, Abeona manufactured a full batch of drug product following patient biopsy collection in August 2025. Although the Company produced bonafide drug product, it could not be released because a rapid sterility assay, mandated by the FDA as a release assay during the final stage of the Biologics License Application (BLA) review, initially yielded a false positive result for sterility. The Company immediately implemented a temporary pause on collecting patient biopsies and worked diligently to optimize the release assay to ensure reliable results and avoid any unnecessary future lot rejections. Upon completion of assay optimization and the necessary regulatory submission for its implementation, Abeona resumed biopsy collection in November 2025. The Company now anticipates commercial product treatment starting in the fourth quarter of 2025.
● Patients at the first two Qualified Treatment Centers (QTCs) are actively advancing through process for ZEVASKYN treatment; Patient demand growing, reflecting unmet need for ZEVASKYN: Of the more than a dozen initial patients identified at the first two QTCs, Abeona has already received ZEVASKYN product order forms (ZPOFs) for 12 patients and they are in the process of scheduling treatments. ZEVASKYN demand has more than doubled at QTCs, with approximately 30 eligible patients now identified.
● Strategic expansion of the QTC network with three activated centers and others progressing through onboarding: With the recent activation of Children’s Hospital Colorado (CHCO), a highly recognized epidermolysis bullosa (EB) center, there are now three activated ZEVASKYN treatment centers along with Lurie Children’s Hospital of Chicago and Lucile Packard Children’s Hospital Stanford. Several additional centers across the US are in various stages of site onboarding.

● Strong early access momentum; focused on driving continued payer engagement and provider readiness: Following ZEVASKYN approval, there has been a steady cadence of coverage decisions from commercial health plans that account for approximately 60 percent of all RDEB patients. Policies covering ZEVASKYN have been published by all major commercial payers including United Healthcare, Cigna, Aetna, Anthem, and most Blue Cross Blue Shield plans, that account for 80 percent of lives covered by commercial insurance, signaling broad and early market acceptance. In addition, the Centers for Medicare and Medicaid Services (CMS) has established a permanent Healthcare Common Procedure Coding System (HCPCS) J-code for ZEVASKYN, J3389 (Topical administration, prademagene zamikeracel, per treatment), effective January 1, 2026.

Other corporate updates

● Pipeline program ABO-503 selected for FDA Rare Disease Endpoint Advancement (RDEA) Pilot Program: ABO-503 gene therapy for X-linked retinoschisis (XLRS) has been selected to participate in the FDA RDEA Pilot Program. As part of the RDEA program, Abeona will have opportunities for enhanced communication and collaboration with the FDA, including frequent advice and regular ad-hoc conversations to accelerate the development and validation of product-specific novel efficacy endpoints for Abeona’s XLRS program.
● Strengthened management team with appointment of Head of Clinical Development & Medical Affairs: Abeona appointed James A. Gow, MD, MBA, MS, MHCM, as the Senior Vice President, Head of Clinical Development & Medical Affairs. Dr. Gow has over 20 years of industry experience in clinical development and medical affairs and is a recognized expert in gene therapy, especially in ophthalmology.

Financial Results

Cash, cash equivalents, restricted cash and short-term investments totaled $207.5 million as of September 30, 2025. The current cash position, without accounting for anticipated revenue from ZEVASKYN, is expected to be sufficient to fund current and planned operations for over two years.

Research and development (R&D) spending for the three months ended September 30, 2025 was $4.2 million, compared to $8.9 million for the same period of 2024. The reduction in R&D expense was primarily due to costs capitalized into inventory and select costs, such as engineering runs and other production costs, reclassified as selling, general, and administrative (SG&A) following FDA approval of ZEVASKYN. SG&A expenses were $19.3 million for the three months ended September 30, 2025, compared to $6.4 million for the same period of 2024. In addition to the reclassification of select R&D expenses to SG&A, the increase in SG&A reflects increased headcount and professional costs associated with the commercial launch of ZEVASKYN.

Net loss was $(5.2) million for the third quarter of 2025, or $(0.10) per basic and diluted common share. Net loss in the third quarter of 2024 was $(30.3) million, or $(0.63) per basic and diluted common share.

Conference Call Details

The Company will host a conference call and webcast on Wednesday, November 12, 2025, at 8:30 a.m. ET, to discuss the financial results and corporate progress. To access the call, dial 877-545-0523 (U.S. toll-free) or 973-528-0016 (international) and Entry Code: 922481 five minutes prior to the start of the call. A live, listen-only webcast and archived replay of the call can be accessed on the Investors & Media section of Abeona’s website at View Source The archived webcast replay will be available for 30 days following the call.

(Press release, Abeona Therapeutics, NOV 12, 2025, View Source [SID1234659802])