On November 3, 2025 Cellectis (the "Company") (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, reported the acceptance of two abstracts for poster presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2025 annual meeting taking place from December 6 to 9, 2025, in Orlando, FL.

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First poster – Development update on eti-cel

The first poster provides a development update on eti-cel product candidate (UCART20x22), an allogeneic dual CAR-T targeting CD20 and CD22 being developed in Phase 1 of the NATHALI-01 clinical trial, for patients with relapsed/refractory non Hodgkin lymphoma (r/r NHL). In addition, the poster outlines the addition of low dose interleukin-2 (IL-2) to further deepen and extend anti-tumor activity of eti-cel in patients with r/r NHL, supported by compelling preclinical data.

Cellectis unveiled preliminary results on eti-cel, which demonstrate an encouraging overall response rate (ORR) of 86% and a complete response (CR) rate of 57% at the current dose level (n=7), with 4 out of 7 patients achieving a complete response. The preliminary high rate of complete responses underscores the potential of this innovative approach to transform outcomes for r/r NHL patients. Cellectis expects to present the full Phase 1 dataset for eti-cel, including low-dose IL-2 combination cohorts, in 2026.

"We are excited by the progress and evolution of the eti-cel program with the addition of IL-2, which promises to build on the encouraging preliminary response rates observed in the Phase 1 program," said Adrian Kilcoyne, MD, MPH, MBA, Chief Medical Officer at Cellectis. "We look forward to sharing the full Phase 1 dataset including the IL-2 cohorts expected in 2026."

Poster title: Trial in progress: Open-label dose-finding and dose-expansion study to evaluate the safety, expansion, persistence, and clinical activity of UCART20x22 in subjects with relapsed or refractory B-cell non-Hodgkin lymphoma (B-NHL) NATHALI-01

Presenter: Vivian Dai, Senior Director, Clinical Research Scientist at Cellectis

Date/Time: December 7, 2025 at 6:00 PM – 8:00 PM ET

Room: OCCC – West Halls B3-B4

Second poster – Correlation between alemtuzumab exposure and response with lasme-cel

The second poster highlights the correlation between alemtuzumab exposure and depth of response in the difficult-to-treat patients who have received lasme-cel (UCART22) in the course of the Phase 1 of BALLI-01, a clinical trial testing this allogeneic CAR-T product candidate targeting CD22 in relapsed/refractory acute lymphoblastic leukemia (ALL). Additionally, the data identifies a threshold exposure level of alemtuzumab above which achieving a complete response/complete response with incomplete hematologic recovery (CR/CRi) is more likely without any increase in toxicities.

"We strongly believe in the critical role of alemtuzumab in optimizing responses in these heavily pretreated patients," said Adrian Kilcoyne, MD, MPH, MBA, Chief Medical Officer at Cellectis. "These data have confirmed this and demonstrated that we could further enhance the high CR/CRi and minimal residual disease (MRD)-negative rates observed in our Phase 1 program. We look forward to starting enrollment in our pivotal Phase 2 program in Q4 2025."

Poster title: Increased alemtuzumab exposure correlates with improved responses in heavily pretreated R/R ALL patients: Analysis of the BALLI-01 trial

Presenter: Xenia Naj, Ph.D., Director Translational Sciences at Cellectis

Date/Time: December 8, 2025, 6:00 PM – 8:00 PM

Room: OCCC – West Halls B3-B4

(Press release, Cellectis, NOV 3, 2025, View Source [SID1234659247])

On November 3, 2025 Castle Biosciences, Inc. (Nasdaq: CSTL), a company improving health through innovative tests that guide patient care, reported its financial results for the third quarter and nine months ended Sept. 30, 2025.

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"We delivered a strong third quarter, generating $83 million in revenue and 26,841 in total test report volume," said Derek Maetzold, president and chief executive officer of Castle Biosciences. "These strong results are a testament to the workforce culture we have built at Castle and our unwavering commitment to improving patient care.

"Our momentum this quarter reflects the strength of our core dermatologic and gastrointestinal testing franchises, with DecisionDx-Melanoma and TissueCypher each surpassing 10,000 test reports for the first time in a single quarter, significant milestones that underscore the expanding adoption of our core tests. Based on our strong execution, we are raising our full-year 2025 total revenue guidance to $327-335 million from the previously provided range of $310-320 million.

"Additionally, with the launch of AdvanceAD-Tx, our new test designed to guide systemic treatment decision making in patients with moderate-to-severe atopic dermatitis, we are thrilled to provide an additional innovative, first-in-class, proprietary test addressing a significant unmet need in clinical dermatology."

Third Quarter Ended Sept. 30, 2025, Financial and Operational Highlights
•Revenues were $83.0 million, compared to $85.8 million in the third quarter of 2024. Affecting third quarter 2025 revenue was the Novitas local coverage determination (LCD), Genetic Testing in Oncology: Specific Tests, that included DecisionDx-SCC as noncovered, which became effective April 24, 2025, as well as the discontinuation of IDgenetix in May 2025.
•Delivered 26,841 total test reports in the third quarter of 2025, compared to 26,010 in the same period of 2024. Affecting third quarter 2025 test report volume was the Novitas LCD, Genetic Testing in Oncology: Specific Tests, that included DecisionDx-SCC as noncovered, which became effective April 24, 2025, as well as the discontinuation of IDgenetix in May 2025:
◦DecisionDx-Melanoma test reports delivered in the quarter were 10,459, compared to 9,367 in the third quarter of 2024.
◦TissueCypher Barrett’s Esophagus test reports delivered in the quarter were 10,609, compared to 6,073 in the third quarter of 2024.

◦DecisionDx-SCC test reports delivered in the quarter were 4,186, compared to 4,195 in the third quarter of 2024. Affecting third quarter test report volume was the Novitas LCD, Genetic Testing in Oncology: Specific Tests, that included DecisionDx-SCC as noncovered, which became effective April 24, 2025.
◦MyPath Melanoma test reports delivered in the quarter were 1,151, compared to 933 in the third quarter of 2024.
◦DecisionDx-UM test reports delivered in the quarter were 436, compared to 397 in the third quarter of 2024.
•Gross margin was 75%, and Adjusted Gross Margin was 77%, compared to 79% and 82%, respectively, for the same periods in 2024.
•Net cash provided by operations was $22.6 million, compared to $23.3 million for the same period in 2024.
•Net loss, which includes non-cash stock-based compensation expense of $12.1 million, was $0.5 million, compared to net income of $2.3 million for the same period in 2024.
•Net loss per share and Adjusted Net Loss per Share, Basic and Diluted, was $0.02, compared to net income per share and Adjusted Net Income per Share, Basic and Diluted, of $0.08, for the same period in 2024.
•Adjusted EBITDA was $9.2 million, compared to $21.6 million for the same period in 2024.

Nine Months Ended Sept. 30, 2025, Financial and Operational Highlights
•Revenues were $257.2 million, compared to $245.8 million during the same period in 2024. Affecting nine months ended September 30, 2025 revenue was the Novitas LCD, Genetic Testing in Oncology: Specific Tests, that included DecisionDx-SCC as noncovered, which became effective April 24, 2025, as well as the discontinuation of IDgenetix in May 2025.
•Delivered 77,817 total test reports in the nine months ended September 30, 2025, compared to 72,000 in the same period of 2024. Affecting nine months ended September 30, 2025 test report volume was the Novitas LCD, Genetic Testing in Oncology: Specific Tests, that included DecisionDx-SCC as noncovered, which became effective April 24, 2025, as well as the discontinuation of IDgenetix in May 2025:
◦DecisionDx-Melanoma test reports delivered in the nine months ended September 30, 2025, were 29,061, compared to 27,336 for the same period in 2024.
◦TissueCypher Barrett’s Esophagus test reports delivered in the nine months ended September 30, 2025, were 27,211, compared to 14,284 for the same period in 2024.
◦DecisionDx-SCC test reports delivered in the nine months ended September 30, 2025, were 13,323, compared to 12,049 for the same period in 2024. Affecting nine months ended September 30, 2025 test report volume was the Novitas LCD, Genetic Testing in Oncology: Specific Tests, that included DecisionDx-SCC as noncovered, which became effective April 24, 2025.
◦MyPath Melanoma test reports delivered in the nine months ended September 30, 2025, were 3,243, compared to 3,030 for the same period in 2024.
◦IDgenetix test reports delivered in the nine months ended September 30, 2025, were 3,605, compared to 14,026 for the same period in 2024. The Company discontinued its IDgenetix test offering effective May 2025.
◦DecisionDx-UM test reports delivered in the nine months ended September 30, 2025, were 1,374, compared to 1,275 for the same period in 2024.
•Gross margin for the nine months ended September 30, 2025, was 67%, and Adjusted Gross Margin was 80%, compared to 79% and 82%, respectively, for the same period in 2024.
•Net cash provided by operations was $37.4 million, compared to $40.5 million for the same period in 2024.
•Net loss, which includes non-cash stock-based compensation expense of $34.5 million, was $21.8 million, compared to net income of $8.7 million for the same period in 2024.
•Net loss per share, Basic and Diluted, was $0.76 and Adjusted Net Loss per Share, Basic and Diluted, was $0.06, compared to net income per share and Adjusted Net Income per Share, Basic and Diluted, of $0.31 and $0.30, respectively, for the same period in 2024.
•Adjusted EBITDA was $32.5 million, compared to $53.7 million for the same period in 2024.
Cash, Cash Equivalents and Marketable Investment Securities
As of Sept. 30, 2025, the Company’s cash, cash equivalents and marketable investment securities totaled $287.5 million.
2025 Outlook
Castle Biosciences is raising its guidance for anticipated total revenue in 2025. The Company now anticipates generating between $327-335 million in total revenue in 2025, compared to the previously provided guidance of between $310-320 million.
Third Quarter and Recent Accomplishments and Highlights

Dermatology- Skin Cancer
•DecisionDx-Melanoma: The Company presented new data demonstrating DecisionDx-Melanoma stratifies risk across histological subtypes at the 25th Annual Fall Clinical Dermatology Conference, which was held Oct. 23–26, 2025, in Las Vegas, Nevada. Specifically, cutaneous melanoma (CM) subtypes, such as superficial spreading and nodular melanoma, vary in how often they occur and in their outcomes. Even among patients with the same subtype, differences in tumor biology can lead to very different prognoses. In a real-world cohort of 13,560 patients with stage I–III CM from Castle’s ongoing collaboration with the National Cancer Institute’s Surveillance, Epidemiology and End Results (NCI’s SEER) Program Registries, DecisionDx-Melanoma stratified melanoma-specific survival (MSS) across different tumor subtypes. For example, five-year MSS in nodular melanoma was 98.5% for patients with Class 1A (lowest risk) test results versus 82.3% for patients with Class 2B (highest risk) test results; similar stratification was observed across superficial spreading, lentigo maligna and unspecified subtypes. These results suggest that DecisionDx-Melanoma provides clarity in overall risk beyond histology, supporting more informed treatment planning and potentially improved outcomes. See the Company’s news release from October 24, 2025, for more information.
•DecisionDx-SCC: Two new studies were published supporting the clinical utility of DecisionDx-SCC in patients with high-risk cutaneous squamous cell carcinoma (SCC). The first study represented a new validation milestone, establishing DecisionDx-SCC as a significant predictor of local recurrence (LR) in patients classified as high-risk by National Comprehensive Cancer Network (NCCN) guidelines, thereby adding a third utility to the test’s existing capabilities. The test has now been validated to predict individual risk of metastasis, benefit from adjuvant radiation therapy (ART) and risk of LR, providing comprehensive results to support tailored post-surgical management and treatment pathway recommendations for patients with SCC. The second publication shared results from a clinical impact study, affirming the impact of the test’s results in guiding these recommendations, specifically the use of ART and surveillance imaging, by providing actionable decision points based on individual patient risk. See the Company’s news release from August 25, 2025, for more information.
Gastroenterology
•The Company announced new data demonstrating the personalized risk stratification provided by its TissueCypher Barrett’s Esophagus (BE) test at the American Foregut Society’s (AFS) 2025 Annual Meeting. Specifically, this study evaluated TissueCypher’s ability to stratify risk in 85 patients diagnosed with non-dysplastic Barrett’s esophagus (NDBE) who received test results from four surgical practices. Patients with NDBE are generally considered to have the lowest risk of cancer progression, and current guidelines recommend surveillance every three to five years. However, while 85% of patients in the study received low-risk TissueCypher results, 15% were classified as intermediate- or high-risk by the TissueCypher test, indicating a significantly higher likelihood of progressing to high-grade dysplasia (HGD) or esophageal adenocarcinoma (EAC) than their pathology results suggested. Patients with intermediate-risk scores had a median five-year progression probability of 9%, and those with high-risk scores had a 16% probability. Both groups exceeded the 8.5% five-year risk of progression associated with expert-confirmed low-grade dysplasia (LGD) based on population estimates, which is the threshold at which guidelines recommend escalating to endoscopic eradication therapy (EET) or more frequent surveillance every six to twelve months. These findings show that TissueCypher can deliver clinically meaningful risk insights that can help physicians better tailor care for patients with BE. Notably, patients in the study with intermediate- and high-risk scores had similar or greater predicted progression risk than patients diagnosed with LGD, despite having a NDBE diagnosis. By identifying low-risk patients whose care can follow guideline-based surveillance intervals and intermediate- and high-risk patients who may benefit from earlier intervention, TissueCypher can potentially support more precise, risk-aligned management aimed at preventing disease progression. See the Company’s news release from September 9, 2025, for more information.

Dermatology- Atopic Dermatitis
•AdvanceAD-Tx: The Company announced the launch of AdvanceAD-Tx, a gene expression profile (GEP) test designed to guide systemic treatment decision making in patients ages 12 and older with moderate-to-severe atopic dermatitis (AD). This innovative 487-GEP test is designed to identify patients with a Janus kinase (JAK) inhibitor responder profile who are more likely to achieve an Eczema Area and Severity Index improvement of 90% (EASI-90), more quickly and with reduction of flares and itch by three months, when treated with a JAK inhibitor than those treated with a T helper type 2 (Th2)-targeted therapy. See the Company’s news release from November 3, 2025, for more information.
Corporate
•The Company announced that its founder, president and chief executive officer Derek Maetzold was named CEO of the Year by The CEO Magazine. The Executive of the Year Awards program recognizes senior executives driving measurable impact, innovation and inspiration. See the Company’s news release from October 1, 2025, for more information.
•The Company announced it was recognized as a Greater Pittsburgh Top Workplace by The Pittsburgh Post-Gazette. The designation is based exclusively on anonymous employee feedback gathered through a third-party survey. The confidential survey measures several aspects of workplace culture designed to be indicative of employee satisfaction and engagement, including feeling respected and supported, enabled to grow and empowered to execute. Castle was among just 89 companies honored with a Greater Pittsburgh Top Workplaces award in 2025. See the Company’s news release from September 23, 2025, for more information.
•The Company announced it was included in the inaugural 2025 America’s Greatest Companies list, published by Newsweek. The ranking honors 650 U.S. companies demonstrating strong performance across four key pillars: financial strength, workforce dedication, innovation, and commitment to environmental sustainability and corporate ethics. Evaluations were based on company reviews, filings with the U.S. Securities and Exchange Commission (SEC) and Patent and Trademark Office (USPTO), and third-party data sources to provide an objective measure of corporate performance. See the Company’s news release from September 17, 2025, for more information.

Conference Call and Webcast Details
Castle Biosciences will hold a conference call on Monday, November 3, 2025, at 4:30 p.m. Eastern time to discuss its third quarter 2025 results and provide a corporate update.
A live webcast of the conference call can be accessed here: View Source or via the webcast link on the Investor Relations page of the Company’s website, View Source Please access the webcast at least 10 minutes before the conference call start time. An archive of the webcast will be available on the Company’s website until November 24, 2025.
To access the live conference call via phone, please dial 833-470-1428 from the United States, or global dial-in numbers are available here: View Source, at least 10 minutes prior to the start of the call, using the conference ID 735311.
There will be a brief Question & Answer session following management commentary.

(Press release, Castle Biosciences, NOV 3, 2025, View Source [SID1234659246])

On November 3, 2025 Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, reported positive results from its ongoing ANTLER phase 1 clinical trial evaluating vispacabtagene regedleucel (vispa-cel; formerly CB-010), an allogeneic anti-CD19 CAR-T cell therapy, in patients with relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL).

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"The ANTLER data mark an exciting advancement for the field of cellular immunotherapy," said Mehdi Hamadani, MD, professor of medicine and section chief of hematologic malignancies at the Medical College of Wisconsin and an investigator on the ANTLER trial. "This clinical dataset demonstrates vispa-cel’s efficacy and durability are comparable to autologous CAR-T therapies, yet its off-the-shelf availability and favorable tolerability profile make it well suited for outpatient administration at both large academic centers and sophisticated community hospitals. This combination of robust clinical activity and accessibility could significantly broaden patient access to transformative CAR-T cell treatments, particularly for those who cannot wait or are ineligible for transplantation or autologous CAR-T cell therapies."

As of the September 2, 2025, safety data cutoff date, the ANTLER trial has enrolled 84 patients, including a confirmatory cohort of 22 CD19-naïve second-line (2L) large B cell lymphoma (LBCL) patients. The confirmatory cohort was designed to prospectively confirm the positive outcomes of partial HLA matching (≥4 matched HLA alleles) observed in earlier retrospective analyses. Patients in the confirmatory cohort received vispa-cel at the recommended phase 2 dose (RP2D; 80×10⁶ CAR-T cells), and the data from this cohort, as of an efficacy data cutoff date of September 29, 2025, confirmed that a single dose of partially matched (≥4 matched HLA alleles) vispa-cel results in efficacy that is on par with approved autologous CAR-T cell therapies including an 82% overall response rate (ORR), a 64% complete response (CR) rate, and 51% progression-free survival (PFS) at 12 months (Table 1). Median follow-up for the confirmatory cohort is 6.0 months.

Table 1. ANTLER phase 1 trial endpoints
Endpoints
Confirmatory cohort1
N=22
Optimized profile2
N=35
ORR
82%
86%
CR rate
64%
63%
Median PFS
(95% CI)
NR
(2.0, NE)
NR
(2.8, NE)
12-month PFS
(95% CI)
51%
(28, 70)
53%
(34, 69)
Median DoR
(95% CI)
NR
(1.7, NE)
NR
(2.1, NE)

12L LBCL 4+ HLA matched, dosed with 80M vispa-cel CAR-T cells
22L (N=32) and 3L+ (N=3) LBCL patients treated with 40M, 80M, or 120M vispa-cel CAR-T cells optimized for multiple factors, including 2+ HLA matching and young donor-derived
CR: complete response; DoR: duration of response; HLA: human leukocyte antigen; NE: not evaluable; NR: not reached; ORR: overall response rate; PFS: progression-free survival
Data cutoff date for efficacy: September 29, 2025

The Company leveraged its large allogeneic CAR-T cell clinical data set (>140 patients dosed across multiple clinical trials) to identify key factors linked to successful patient outcomes. Two of those factors are donor age (young donors drive enhanced outcomes relative to older donors) and partial HLA matching (matching 2 or more [2+] alleles correlates with outcomes on par with autologous CAR-T cell therapies). Of the 84 patients dosed with vispa-cel, there are 35 CD19-naïve LBCL patients who received vispa-cel with an optimized profile (32 of these patients were 2L and 3 of these patients were 3L+). The optimized profile vispa-cel was manufactured from young donor-derived T cells, and the 35 patients matched a minimum of 2 HLA alleles with the T cell donor. Twenty of the 35 patients in the optimized profile cohort were enrolled in the confirmatory cohort, and the remaining 15 patients were enrolled in dose escalation or expansion.

Data from this 35-patient cohort further confirmed that the efficacy and durability of vispa-cel are on par with the autologous CAR-T cell therapies. Median follow-up for the optimized profile cohort was 11.8 months, and the longest responding patient, who completed the 2-year ANTLER trial and enrolled in the long-term follow-up study, is in complete response at 3 years post infusion. As of the September 29, 2025, efficacy data cutoff date, the results for the 35-patient optimized profile cohort included an 86% ORR, a 63% CR rate, and 53% PFS at 12 months (Table 1).

In all patients treated in ANTLER (N=84), vispa-cel has demonstrated a generally well-tolerated safety profile. As of the September 2, 2025, safety data cutoff date, treatment emergent adverse events at any grade in ≥25% of all patients who received vispa-cel were thrombocytopenia (62%), cytokine release syndrome (CRS; 55%), anemia (52%), neutropenia (39%), hypokalemia (26%), and leukopenia (26%). In the confirmatory and optimized profile cohorts, there were no cases of graft-versus-host disease (GvHD) or ≥grade 3 immune effector cell-associated neurotoxicity syndrome (ICANS), <5% patients experienced ≥grade 3 CRS, and there were manageable rates of infections and prolonged cytopenias (Table 2). The safety profile of vispa-cel allows for use in an outpatient setting.

"We believe that with these results, Caribou has achieved what the field has long sought — strong evidence that an allogeneic CAR-T cell therapy can be on par with the efficacy and durability of autologous treatments and broaden access with a safety profile that allows for outpatient use," said Rachel Haurwitz, PhD, Caribou’s president and CEO. "This milestone positions vispa-cel as a potentially best-in-class allogeneic CAR-T cell therapy for patients with large B cell lymphoma. We believe we have a straightforward regulatory path toward full registration by following the FDA’s recommendation for a randomized, controlled phase 3 trial in second-line large B cell lymphoma, and we plan to refine our pivotal trial design in the coming months through continued engagement with the FDA."

Table 2. ANTLER phase 1 trial safety data
Events, n (%)
All treated
N=84
Confirmatory cohort
N=221
Optimized profile
N=352
All grade
≥Grade 3
All grade
≥Grade 3
All grade
≥Grade 3
ICANS
12 (14)
4 (5)
1 (5)
0 (0)
1 (3)
0 (0)
CRS
46 (55)
1 (1)
13 (59)
1 (5)
19 (54)
1 (3)
Infections
43 (51)
21 (25)
9 (41)
4 (18)
20 (57)
6 (17)
Prolonged cytopenias3
NA
22/80 (28)
NA
5/19 (26)
NA
7/32 (22)
IEC-HS4
2 (2)
2 (2)
1 (5)
1 (5)
1 (3)
1 (3)

12L LBCL 4+ HLA matched, dosed with 80M vispa-cel CAR-T cells
22L (N=32) and 3L+ (N=3) LBCL patients treated with 40M, 80M, or 120M vispa-cel CAR-T cells optimized for multiple factors, including 2+ HLA matching and young donor-derived
3Prolonged cytopenias are defined as Grade 3 or 4 neutropenia, thrombocytopenia, or anemia ongoing at day 28 (+/- 5 days) post CAR-infusion, based on laboratory data, distinct from investigator-reported clinical adverse events.
4Includes one vispa-cel-related grade 5 IEC-HS that occurred on day 25 post-infusion.
CRS: cytokine release syndrome; IEC-HS: immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome; NA: not applicable
Data cutoff date for safety: September 2, 2025

A cohort of third-line or later LBCL patients with prior exposure to CD19-targeted therapy enrolled 5 patients as of the September 2, 2025, safety data cutoff date. Enrollment in this cohort has been paused to focus on CD19-naïve patients.

Pivotal phase 3 trial for 2L LBCL for full approval
In recent interactions, the FDA has recommended the Company conduct a randomized, controlled trial in 2L LBCL CD19-naive patients who are ineligible for transplant and autologous CAR-T cell therapy, and the Company intends to follow this approach with its planned pivotal phase 3 clinical trial design, evaluating approximately 250 patients. Patients randomized to the study arm would receive a single dose of vispa-cel at the recommended phase 2 dose of 80×10⁶ CAR-T cells following lymphodepletion with cyclophosphamide and fludarabine, and patients randomized to the comparator arm would be treated with the investigator’s choice of standard of care immunochemotherapy agents. The primary endpoint is PFS, and an interim analysis of PFS is planned. Secondary endpoints would include ORR, complete response rate (CRR), duration of response (DoR), duration of complete response (DoCR), overall survival (OS), quality of life (QoL), and safety. This expected trial design reflects Caribou’s internal analysis and interactions with the FDA to date. The Company intends to further refine the pivotal trial design through continued engagement with the FDA prior to initiation. Caribou plans to bring vispa-cel closer to where patients live by leveraging community and academic sites within the U.S. and globally.

Webcast conference call today at 8:00 am ET
Caribou will host a live conference call and webcast on Monday, November 3 at 8:00 am ET to discuss the ANTLER trial data and the pivotal phase 3 clinical trial design, as well as the CaMMouflage phase 1 clinical trial for r/r multiple myeloma. The presenters will include:
•Mehdi Hamadani, MD, professor of medicine, section chief of hematologic malignancies at Medical College of Wisconsin, and investigator on the ANTLER trial
•Joseph McGuirk, DO, professor of hematology/oncology and division director for hematologic malignancies and cellular therapeutics at University of Kansas Cancer Center
•Adriana Rossi, MD, director of CAR-T and stem cell transplant clinical program at the center of excellence for multiple myeloma at Mt Sinai, and investigator on the CaMMouflage trial
•Rachel Haurwitz, PhD, president and chief executive officer, Caribou Biosciences
•Tina Albertson, MD, PhD, chief medical officer, Caribou Biosciences

A live webcast of the presentation will be accessible via Caribou’s website on the Events page. The archived webcast will be available on the Caribou website for 30 days after the event.

About vispacabtagene regedleucel
Vispacabtagene regedleucel (vispa-cel; formerly known as CB-010) is an allogeneic anti-CD19 CAR-T cell therapy being evaluated in patients with relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL). To Caribou’s knowledge, vispa-cel is the first allogeneic CAR-T cell therapy in the clinic with a PD-1 knockout, a genome-editing strategy designed to enhance CAR-T cell activity by limiting premature CAR-T cell exhaustion. The FDA granted vispa-cel Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, and Fast Track designations for B-NHL.

About the ANTLER phase 1 clinical trial
The ANTLER clinical trial is a multicenter, open-label phase 1 trial evaluating vispacabtagene regedleucel (vispa-cel; formerly CB-010) in adults with relapsed or refractory B cell non-Hodgkin lymphoma (B-NHL). Eighty-four patients have been treated in the ANTLER clinical trial as of September 2, 2025. Using a 3+3 enrollment strategy, safety and efficacy were assessed in 16 patients in dose escalation evaluating 40×106, 80×106, and 120×106 CAR-T cell dose levels with a lymphodepletion regimen of cyclophosphamide at 60 mg/kg/day for 2 days followed by fludarabine at 25 mg/m2/day for 5 days. Forty-one patients with 2L LBCL were enrolled in the dose expansion portion, and 80×106 CAR-T cells was selected as the recommended phase 2 dose (RP2D). An additional 22 patients with 2L LBCL were enrolled in the confirmatory cohort, which prospectively evaluated the Company’s partial HLA matching strategy. Five patients were enrolled in a cohort of third-line or later LBCL patients with prior exposure to CD19-targeted therapy. Additional information on the ANTLER trial (NCT04637763) can be found at clinicaltrials.gov.

(Press release, Caribou Biosciences, NOV 3, 2025, View Source [SID1234659245])

On November 3, 2025 BioNTech SE (Nasdaq: BNTX, "BioNTech" or "the Company") reported financial results for the three and nine months ended September 30, 2025 and provided an update on its corporate progress.

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"In the third quarter, we made substantial progress in executing against our oncology strategy. We advanced our priority pan-tumor programs, mRNA cancer immunotherapies and pumitamig. Simultaneously, we further broadened these programs to include evaluation of novel combinations with the aim to deliver differentiated or best-in-class therapeutic profiles," said Prof. Ugur Sahin, M.D., Chief Executive Officer and Co-Founder of BioNTech. "Our collaboration with Bristol Myers Squibb on pumitamig is already demonstrating the strength of this partnership, with multiple additional pivotal trials in preparation with pumitamig planned to start this and next year. This illustrates our commitment to delivering truly transformative options for patients in need."

Financial Review for Third Quarter and Year-to-Date 2025

in millions €,
except per share data Third Quarter 2025 Third Quarter 2024 Year-to-date
2025 Year-to-date
2024
Revenues 1,518.9 1,244.8 1,962.5 1,561.1
Net profit / (loss) (28.7) 198.1 (831.1) (924.8)
Basic earnings / (loss) per share (0.12) 0.82 (3.45) (3.83)
Diluted earnings / (loss) per share (0.12) 0.81 (3.45) (3.83)
Revenues for the three months ended September 30, 2025, were €1,518.9 million, compared to €1,244.8 million for the comparative prior year period. For the nine months ended September 30, 2025, revenues were €1,962.5 million, compared to €1,561.1 million for the comparative prior year period. The increases in both quarterly and year-to-date revenues compared to the prior year were primarily driven by revenues related to BioNTech’s collaboration with BMS that were recognized in the third quarter of 2025. This increase was partially offset by lower sales volumes of BioNTech’s COVID-19 vaccines.

Research and development ("R&D") expenses were €564.8 million for the three months ended September 30, 2025, compared to €550.3 million for the comparative prior year period. For the nine months ended September 30, 2025, R&D expenses were €1,599.5 million, compared to €1,642.4 million for the comparative prior year period. Year-to-date R&D expenses were mainly driven by the start of late-stage trials for immuno-oncology ("IO") and antibody-drug conjugate ("ADC") development programs and partly offset by cost savings resulting from active portfolio management.

Sales, general and administrative ("SG&A") expenses, in total, amounted to €148.5 million for the three months ended September 30, 2025, compared to €150.5 million for the comparative prior year period. For the nine months ended September 30, 2025, SG&A expenses were €406.5 million, compared to €466.9 million for the comparative prior year period. The year-to-date and quarter-to-quarter decreases were mainly driven by lower external costs, partially compensated by an ongoing commercial build-up.

Other operating result amounted to negative €704.2 million during the three months ended September 30, 2025, compared to negative €354.6 million for the comparative prior year period. For the nine months ended September 30, 2025, other operating result amounted to negative €730.1 million compared to negative €616.9 million for the prior year period. The increase in other operating expenses compared to the third quarter of 2024 was primarily influenced by the settlement of a contractual dispute.

Net loss was €28.7 million for the three months ended September 30, 2025, compared to a net income of €198.1 million for the comparative prior year period. For the nine months ended September 30, 2025, net loss was €831.1 million, compared to a net loss of €924.8 million for the comparative prior year period.

Basic and diluted loss per share was €0.12 for the three months ended September 30, 2025, compared to a basic earnings per share of €0.82 and diluted earnings per share of €0.81 for the comparative prior year period. For the nine months ended September 30, 2025, basic and diluted loss per share was €3.45, compared to a basic and diluted loss per share of €3.83 for the comparative prior year period.

Cash and cash equivalents plus security investments as of September 30, 2025, reached €16,704.9 million, comprising €10,092.9 million in cash and cash equivalents, €4,275.6 million in current security investments and €2,336.4 million in non-current security investments.

Shares outstanding as of September 30, 2025, were 240,455,450, excluding 8,096,750 shares held in treasury.

"The receipt of $1.5 billion from our partnership with Bristol Myers Squibb further underscores the strategic value of our collaborations not only in the long but also in the short term," said Ramón Zapata, Chief Financial Officer at BioNTech. "We are increasing our 2025 full year revenue guidance to €2.6-2.8 billion. At the same time, we continue to optimize our cost base to support a sustainable development trajectory and ensure operational efficiency."

2025 Financial Year Guidance5:

FY Guidance March 2025 FY Guidance November 2025
Revenues for the 2025 financial year €1,700 – €2,200 million €2,600 – €2,800 million
BioNTech has increased its previous revenue guidance and now expects its revenues for the full 2025 financial year to be in the range of €2,600 – €2,800 million. With regards to COVID-19 vaccine franchise, the guidance reflects the following assumptions: relatively stable COVID-19 vaccine pricing and market share as compared to 2024; inventory write-downs and other charges estimated to be approximately 15% of BioNTech’s share of gross profit from COVID-19 vaccine sales in Pfizer Inc.’s ("Pfizer") territory; and anticipated revenues from a pandemic preparedness contract with the German government. Current and potential further developments in law, global public policy, international trade, and public sentiment as they continue to evolve could further impact the anticipated COVID-19 vaccine revenues and expenses. The revenue guidance also includes anticipated revenues from collaborations, and from the BioNTech Group service businesses.

Planned 2025 Financial Year Expenses and Capex:

FY Guidance March 2025 FY Guidance November 2025
R&D expenses €2,600 – €2,800 million €2,000 – €2,200 million
SG&A expenses €650 – €750 million €550 – €650 million
Capital expenditures for operating activities €250 – €350 million €200 – €250 million
BioNTech has lowered expense guidance ranges for R&D, SG&A and capital expenditures for operating activities for the 2025 financial year.

The Company expects to continuously focus investments on R&D and scaling the business for late-stage development and commercial readiness in oncology, while remaining cost-disciplined. Strategic capital allocation will continue to be a key driver of the Company’s trajectory. As part of BioNTech’s strategy, the Company may continue to evaluate appropriate corporate development opportunities with the aim of driving sustainable long-term growth and creating future value.

The full interim unaudited condensed consolidated financial statements can be found in BioNTech’s Report on Form 6-K for the period ended September 30, 2025, filed today with the United States Securities and Exchange Commission ("SEC") and available at www.sec.gov.

(Press release, BioNTech, NOV 3, 2025, View Source [SID1234659244])

On November 3, 2025 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the third quarter ended September 30, 2025, and provided a business update.

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"BioCryst’s excellent performance this quarter was driven by the continued momentum of ORLADEYO, which delivered impressive growth. We’ve recently made two strategic moves that are transformative for our company: the sale of our European ORLADEYO business, which strengthened our margins and financial position and enabled us to pay off our debt, and the proposed acquisition of Astria, which we believe gives us the incredible opportunity to help even more HAE patients and drive sustainable growth and profitability well into the next decade," said Jon Stonehouse, Chief Executive Officer of BioCryst.

"It has been an incredible honor to lead the employees of BioCryst over these past nearly 19 years. I couldn’t be more confident in the exceptional team we have built and am excited to see them take BioCryst forward and continue to deliver innovative treatments for patients living with rare diseases."

ORLADEYO (berotralstat): Oral, Once-daily Treatment for Prevention of Hereditary Angioedema (HAE) Attacks

ORLADEYO net revenue in the third quarter of 2025 was $159.1 million (+37 percent year-over-year (y-o-y)).
New patient prescriptions in the third quarter were strong, slightly beating those in Q3 2024 despite the recent launches of new prophylactic competitors.
The number of new prescribers of ORLADEYO in the U.S. in the third quarter was 64, exceeding our two-year quarterly average.
Patient retention rates remained consistent with long-term trends.
Sales from the U.S. contributed 89 percent of global ORLADEYO net revenues in the third quarter.
"ORLADEYO demand remained strong in the third quarter, with new patient adds and prescriber confidence driving continued growth. Even with the market entry of new prophylactic therapies, ORLADEYO remains the most differentiated option for patients and our commercial results are strong evidence of this. We’re not resting on our success though: we’re excited about the possibility to bring ORLADEYO granules to kids with HAE in the near-term as well as potentially expanding our HAE portfolio with navenibart. Our team’s continuing focus on execution and innovation is improving our ability to help more patients in the HAE community, now and for years to come," said Charlie Gayer, President and Chief Commercial Officer of BioCryst.

Business Updates

The company has selected Ron Dullinger to become its next Chief Commercial Officer, effective January 1, 2026. With decades of commercial leadership experience across rare disease, oncology, and vaccine markets, Mr. Dullinger has a proven track record of building and leading high-performing teams and delivering exceptional results. He joined the BioCryst commercial organization in 2019 as Vice President of U.S. Sales and has served most recently as Senior Vice President and General Manager of the Americas region, leading the commercialization of ORLADEYO.
The Prescription Drug User Fee Act goal date for the company’s new drug application for ORLADEYO granules in children with HAE aged 2 to <12 is December 12, 2025. ORLADEYO would be the first targeted oral prophylactic therapy for children with HAE.
In October, the company entered into a definitive agreement to acquire Astria Therapeutics. The proposed transaction will add Astria’s lead product candidate, navenibart, to BioCryst’s HAE portfolio. Navenibart is an injectable, long-acting, monoclonal antibody inhibitor of plasma kallikrein currently in Phase 3 clinical development for HAE prophylaxis. The transaction is expected to close in the first quarter of 2026.
A Phase 1 trial of BCX17725, an investigational KLK5 inhibitor for the treatment of Netherton syndrome, is enrolling in healthy volunteers and patients. The company expects initial data in patients from this program by the end of the first quarter of 2026.
A Phase 1 trial of avoralstat, an investigational plasma kallikrein inhibitor for the treatment of diabetic macular edema (DME), is enrolling in patients. The company expects initial data from this program by the end of the year and plans to seek a strategic partner for development beyond Phase 1.
Third Quarter 2025 Financial Results

For the three months ended September 30, 2025, total revenues were $159.4 million, compared to $117.1 million in the third quarter of 2024 (+36 percent y-o-y). The increase was primarily due to $159.1 million in ORLADEYO net revenue in the third quarter of 2025, compared to $116.3 million in ORLADEYO net revenue in the third quarter of 2024 (+37 percent y-o-y).

Research and development expenses for the third quarter of 2025 increased to $44.6 million from $41.1 million in the third quarter of 2024 (+9 percent y-o-y), primarily due to advancement of BCX17725 into the clinic and investigational new drug (IND)-enabling activities for pre-clinical programs. These increases were partially offset by the discontinuation and close-out of the Factor D program, a decrease in stock-based compensation, and ORLADEYO-related regulatory, safety, quality, and manufacturing expenses, previously recorded in research and development, that are now recorded in selling, general, and administrative to reflect the program’s commercial progression.

Selling, general and administrative expenses for the third quarter of 2025 increased to $83.0 million, compared to $65.1 million in the third quarter of 2024 (+27 percent y-o-y). Approximately $6.9 million of the increase was driven by transaction-related costs and stock-based compensation. Approximately $4.7 million was driven by ORLADEYO-related regulatory, safety, quality, and manufacturing expenses, previously recorded in research and development, that are now recorded in selling, general, and administrative to reflect the program’s commercial progression. The remainder was driven by the growth of ORLADEYO and general and administrative expenses.

Operating income for the third quarter of 2025 was $29.6 million, compared to $7.7 million for the third quarter of 2024. Non-GAAP operating income, excluding stock-based compensation expense and transaction-related costs, was $51.7 million for the third quarter of 2025, compared to $24.9 million for the third quarter of 2024.

Interest expense was $19.7 million in the third quarter of 2025, compared to $24.8 million in the third quarter of 2024 (-21 percent y-o-y). The decrease was primarily the result of the $125 million in partial prepayments on the outstanding principal amount under the Pharmakon Term Loan made in 2025, and the decrease in the effective interest rate related to the Pharmakon Loan Agreement.

Net income for the third quarter of 2025 was $12.9 million, or $0.06 per share, compared to a net loss of $14.0 million, or $0.07 per share, for the third quarter of 2024. Non-GAAP net income, excluding stock-based compensation expense, transaction-related costs, and loss on extinguishment of debt was $35.6 million, or $0.17 per share, for the third quarter of 2025, compared to $3.2 million, or $0.02 per share, for the third quarter of 2024.

Cash, cash equivalents, restricted cash and investments totaled $269.4 million at September 30, 2025, of which $14.8 million of cash and cash equivalents are held within the company’s European business and is reflected in current assets held for sale, compared to $351.7 million at September 30, 2024. Net cash utilization for the third quarter of 2025 was $17.8 million, which was driven by the $50 million Pharmakon prepayment made in July 2025. Excluding this prepayment, there was $32.2 million of cash generated during the quarter, primarily driven by ORLADEYO sales.

In October, the company prepaid the remaining outstanding amount under the Pharmakon term loan of $198.7 million following the closing of the sale of its European ORLADEYO business. The pro forma cash balance at September 30, 2025 was $294 million, which includes the impacts of net proceeds from the European sale and payment of associated expenses, transfer of cash from European entities, and prepayment of the remaining balance of the Pharmakon term loan and associated expenses.

Financial Outlook for 2025
The company is raising its outlook for full year 2025 global net ORLADEYO revenue to between $590 million and $600 million and lowering its outlook for 2025 non-GAAP operating expenses, excluding stock-based compensation expense and transaction-related costs, to between $430 million and $440 million. These figures exclude revenue and expenses associated with the European ORLADEYO business in the fourth quarter of 2025 as the sale of this business has been completed.

The company remains on track to deliver net income and positive cash flows for full year 2025. Positive cash flow refers to the improvement in cash, cash equivalents, restricted cash and investments from year end 2024 to year end 2025, not including the impact of debt prepayments or the sale of the European ORLADEYO business.

Conference Call and Webcast
BioCryst management will host a conference call and webcast at 8:30 a.m. ET today to discuss the financial results and provide a corporate update. The live call may be accessed by dialing 1-844-481-2942 for domestic callers and 1-412-317-1866 for international callers. A live webcast and replay of the call will be available online in the investors section of the company website at www.biocryst.com.

(Press release, BioCryst Pharmaceuticals, NOV 3, 2025, View Source [SID1234659243])