On August 6, 2026 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 financial results for the second quarter 2026 ending June 30, 2026.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The lasme-cel and eti-cel clinical results presented at EHA (Free EHA Whitepaper) 2026 are promising for patients with relapsed or refractory B-cell malignancies. We are also pleased to have received RMAT designation from the FDA for lasme-cel, which recognizes its potential to address an unmet medical need in B-ALL. We remain focused on advancing new options for people whose disease has returned or stopped responding to available therapies," said André Choulika, Ph.D., Co-Founder and Chief Executive Officer at Cellectis.

Allogeneic CAR-T Pipeline

Lasme-cel in relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) – BALLI-01

The Pivotal Phase 2 BALLI-01 trial is ongoing.

In June 2026, Cellectis received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for lasme-cel for treatment of r/r CD22 positive B-ALL. This designation was granted based on the BALLI-01 clinical data, demonstrating promising efficacy and a manageable safety profile. It reflects the FDA’s recognition of the potential of lasme-cel to address the unmet medical need faced by patients with r/r B-ALL.
In June 2026, Cellectis presented full Phase 1 data from the BALLI-01 trial at an oral presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) 2026 Annual Congress.

45 patients were treated in third line and beyond (3L+), including 15 at the recommended Phase 2 dose (RP2D), and 7 in the target Phase 2 population.

Heavily pretreated population: A median of 5 prior lines of therapy in the target Phase 2 population (range 2–11); 82% had received prior blinatumomab, 56% a CD22-directed antibody drug conjugate (ADC), 53% CD19 CAR-T, and 47% a prior hematopoietic stem cell transplantation (HSCT).

Efficacy Data (target Phase 2 population)

100% overall response rate (ORR) (7/7)
57% complete remission/complete remission with incomplete count recovery (CR/CRi) (4/7), of whom 75% were minimal residual disease (MRD)-negative
All responding patients proceeded to HSCT

Safety Data

The therapy demonstrated a manageable safety profile, with grade ≥ 3 cytokine release syndrome (CRS) and Immune effector cell-associated neurotoxicity syndrome (ICANS), each occurring in 4% of patients.
Immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome (IEC-HS) ≥ grade 3 occurred in 2% of patients.
All events resolved.
In June 2026, the UK Medicines and Healthcare products Regulatory Agency (MHRA) approved the initiation of the Phase 2 study of BALLI-01 in the UK.
In July 2026, enrollments in the Phase 2 BALLI-01 study in France, Italy and Spain have been authorized.

The first interim analysis for the pivotal Phase 2 of the BALLI-01 trial is expected in Q4 2026.

Eti-cel in relapsed or refractory non-Hodgkin lymphoma (r/r NHL) – NATHALI-01

The Phase 1 NATHALI-01 trial is ongoing.

In June 2026, Cellectis presented translational data highlighting the key drivers of response at a poster presentation at the EHA (Free EHA Whitepaper) 2026 annual congress.

As of the February 2026 data cutoff, 14 patients with r/r B-NHL had been treated across three dose levels.

Heavily pretreated population: median of 3 prior lines of therapy; 93% had received prior CD19-directed CAR-T therapy.

Efficacy Data (optimal dose cohort, n=8)

88% ORR
63% complete response (CR) rate
Higher alemtuzumab exposure was associated with a lower inflammatory homeostatic milieu prior to eti-cel infusion, enhanced eti-cel expansion, and higher response rates.
Responders demonstrated sustained low-level interleukin-2 (IL-2) secretion versus non-responders.

These findings support a weight-based alemtuzumab dosing regimen, currently under investigation to optimize lymphodepletion. Subcutaneous low-dose IL-2 is also being evaluated to further enhance eti-cel expansion and response.

Cellectis expects to present the full Phase 1 dataset in Q4 2026.

Partnerships

AstraZeneca – Joint Research and Collaboration Agreement

Activities are continuing under the Joint Research and Collaboration Agreement with AstraZeneca, which leverages Cellectis’ gene editing expertise and manufacturing capabilities to develop up to 10 novel cell and gene therapy products for areas of high unmet medical need, including oncology, immunology and rare genetic disorders.

Servier (through its sublicensee Allogene) – Anti-CD19 CAR-T

In July 2026, Allogene announced that the FDA has granted RMAT and Fast Track designations to cema-cel for the treatment of adult patients with large B-cell lymphoma (LBCL) who, at the completion of first-line (1L) therapy, are in complete or partial response suitable for observation but test positive for minimal residual disease (MRD).

Cema-cel is a product candidate licensed to Servier under the License, Development and Commercialization Agreement signed by and between les Laboratoires Servier and Institut de Recherches Internationales Servier ("Servier") and Cellectis (the "Servier Agreement") and sublicensed by Servier to Allogene in certain territories.

Allogene – Anti-CD70 CAR-T

In July 2026, Allogene announced the publication of complete Phase 1 data from the TRAVERSE study of ALLO-316 in advanced or metastatic renal cell carcinoma (RCC) in the Journal of Clinical Oncology. Allogene announced that ALLO-316 achieved a 31% confirmed response rate with the recommended Phase 2 regimen in patients with Stage IV RCC with high CD70 expression, and that the safety profile was manageable with proactive diagnostic and management strategies effective in mitigating IEC-HS.2

Allogene’s investigational allogeneic CAR-T oncology products utilize Cellectis technologies. The anti-CD70 program is licensed exclusively from Cellectis by Allogene and Allogene holds global development and commercial rights to this program.

Corporate Updates

Annual Shareholders’ Meeting

On June 25, 2026, Cellectis held a Shareholders General Meeting at the Biopark auditorium in Paris, France. At the meeting, during which approximately 56% of voting rights were exercised, resolutions 1 through 29 were adopted, while resolution 30 was rejected, consistent with the recommendations of the Board of Directors. The detailed results of the vote and the resolutions are available on Cellectis’ website: View Source

Financial Results

Cash, cash equivalent and fixed-term deposits: As of June 30, 2026, Cellectis had $169 million in consolidated cash, cash equivalents, restricted cash and fixed-term deposits classified as current financial assets. The Company believes its cash, cash equivalents and fixed-term deposits will be sufficient to fund its operations into Q4 2027.

This compares to $211 million in consolidated cash, cash equivalents, restricted cash and fixed-term deposits classified as current financial assets as of December 31, 2025. The $42 million change was primarily driven by payments to suppliers of $26.9 million, payroll-related payments (salaries, bonuses and social charges) totaling $28.5 million, lease liability payments of $5.4 million, repayments of $2.7 million under the "PGE" loan and capital expenditures of $0.5 million, partially offset by $16.8 million of cash received from customers and $4.9 million of interest received from our financial and cash-equivalent investments.

We currently foresee focusing our cash spending at Cellectis in supporting the development of our pipeline of product candidates, including the manufacturing and clinical trial expenses of lasme-cel, eti-cel and potential new product candidates, and operating our state-of-the-art manufacturing capabilities in Paris (France) and Raleigh (North Carolina).

Revenues and Other Income: Consolidated revenues and other income were $14.5 million for the six-month period ended June 30, 2026, compared to $30.2 million for the six-month period ended June 30, 2025. The $15.8 million decrease between the six-month periods ended June 30, 2025 and 2026 was primarily attributable to a $16.4 million decrease in revenues mainly driven by the level of activities performed under the Research Plans of the AstraZeneca Joint Research Collaboration Agreement in the first half of 2026. It was partially offset by a $0.6 million increase, which was mainly attributable to a higher research tax credit resulting from increased eligible R&D expenses, as well as favorable foreign exchange effects.

R&D Expenses: Consolidated R&D expenses were $52.2 million for the six-month period ended June 30, 2026, compared to $45.0 million for the six-month period ended June 30, 2025. The $7.2 million increase was primarily driven by (i) a $4.5 million increase in personnel expenses reflecting changes in our R&D headcount and higher stock-based compensation expense associated with awards granted in 2026, whose grant-date fair value increased due to a higher underlying share price, and (ii) a $3.7 million increase in purchases and external expenses, primarily attributable to higher clinical development costs related to our BALLI-01 and NATHALI-01 studies, partially offset by (iii) a $1.0 million decrease in depreciation and amortization expenses.

SG&A Expenses: Consolidated SG&A expenses were $11.3 million for the six-month period ended June 30, 2026, compared to $9.8 million for the six-month period ended June 30, 2025. The $1.5 million increase was primarily attributable to a $1.2 million increase in personnel expenses, mainly reflecting higher stock-based compensation expense associated with awards granted in 2026, whose grant-date fair value increased due to a higher underlying share price. Purchases and external expenses increased slightly by $0.2 million, from $4.4 million in 2025 to $4.7 million in 2026.

Net financial gain (loss): The consolidated net financial gain for the six-month period ended June 30, 2026 was $9.2 million, compared to a $18.1 million net financial loss for the six-month period ended June 30, 2025. The $27.3 million difference reflects a $5.0 million increase in financial income and a $22.3 million decrease in financial expenses.

The $5.0 million increase in financial income was primarily attributable to (i) a $7.0 million increase in non-cash gains recognized from fair value measurements, mainly reflecting an $8.7 million gain on the fair value measurement of the Tranche A, B and C warrants issued to the European Investment Bank ("EIB") in the six months ended June 30, 2026, compared with a $1.2 million gain in the same period in 2025, partially offset by (ii) a $1.7 million decrease in interest income earned on cash, cash equivalents and financial assets, and (iii) a $0.4 million decrease in foreign exchange gains.

The $22.3 million decrease in financial expenses was primarily attributable to a $22.8 million decrease in foreign exchange losses mainly resulting from the appreciation of the US dollar against the euro.

Net Loss Attributable to Shareholders of Cellectis: Consolidated net loss attributable to shareholders of Cellectis was $39.6 million (or a $0.39 net loss per share) for the six-month period ended June 30, 2026, compared to a $41.9 million net loss (or a $0.42 net loss per share) for the six-month period ended June 30, 2025. The $2.3 million decrease in net loss was mainly due to (i) a $27.3 million improvement in net financial result, from a net financial loss of $18.1 million as of June 30, 2025 to a net financial gain of $9.2 million as of June 30, 2026, partly offset by (ii) a $24.9 million increase in operating loss.

Adjusted Net Loss Attributable to Shareholders of Cellectis: Consolidated adjusted net loss attributable to shareholders of Cellectis was $35.6 million (or a $0.35 net loss per share) for the six-month period ended June 30, 2026, compared to a net loss of $39.6 million (or a $0.40 net loss per share) for the six-month period ended June 30, 2025.

The interim condensed consolidated financial statements of Cellectis have been prepared in accordance with International Financial Reporting Standards, as issued by the International Accounting Standards Board ("IFRS").

Please see "Note Regarding Use of Non-IFRS Financial Measures" for reconciliation of GAAP net income (loss) attributable to shareholders of Cellectis to adjusted net income (loss) attributable to shareholders of Cellectis.

CELLECTIS S.A.
UNAUDITED INTERIM CONDENSED STATEMENTS OF CONSOLIDATED FINANCIAL POSITION
($ in thousands)

As of
December 31, 2025 June 30, 2026
ASSETS
Non-current assets
Intangible assets 535 1,117
Property, plant, and equipment 38,788 34,797
Right-of-use assets 23,658 19,196
Non-current financial assets 5,088 4,723
Other non-current assets 20,025 22,734
Deferred tax assets 382 382
Total non-current assets 88,476 82,949
Current assets
Trade receivables 14,398 5,075
Subsidies receivables 7,800 7,525
Other current assets 5,383 4,970
Cash, cash equivalents and current financial assets 208,663 166,847
Total current assets 236,244 184,417
TOTAL ASSETS 324,720 267,365
LIABILITIES
Shareholders’ equity
Share capital 5,903 5,924
Premiums related to the share capital 437,445 371,749
Currency translation adjustment (33,316 ) (32,679 )
Retained earnings (deficit) (266,538 ) (264,344 )
Net income (loss) (67,593 ) (39,584 )
Total shareholders’ equity 75,901 41,067
Non-current liabilities
Non-current financial liabilities 74,013 66,185
Non-current lease debts 27,725 23,823
Non-current provisions 1,329 1,332
Total non-current liabilities 103,067 91,340
Current liabilities
Current financial liabilities 10,460 7,500
Current lease debts 7,701 6,774
Trade payables 17,277 18,202
Deferred income and contract liabilities 96,803 90,918
Current provisions 1,169 917
Other current liabilities 12,342 10,647
Total current liabilities 145,752 134,958
TOTAL LIABILITIES AND SHAREHOLDERS’ EQUITY 324,720 267,365

Cellectis S.A.
UNAUDITED INTERIM CONDENSED STATEMENTS OF CONSOLIDATED OPERATIONS
For the six-month period ended June 30, 2026
($ in thousands, except share and per share amounts)

For the six-month period ended June 30,
2025 2026

Revenues and other income
Revenues 27,380 11,006
Other income 2,842 3,446
Total revenues and other income 30,222 14,452
Operating expenses
Research and development expenses (45,012 ) (52,165 )
Selling, general and administrative expenses (9,780 ) (11,329 )
Other operating income 804 353
Total operating expenses (53,988 ) (63,140 )
Operating loss (23,766 ) (48,688 )
Net Financial gain (loss) (18,098 ) 9,176
Income tax - (72 )
Net loss (41,863 ) (39,584 )
Basic and diluted net loss per share attributable to shareholders of Cellectis ($/share) (0.42 ) (0.39 )
Number of shares used for computing (basic and diluted) 100,231,292 100,587,696

UNAUDITED INTERIM CONDENSED STATEMENTS OF CONSOLIDATED OPERATIONS
For the three-month period ended June 30, 2026
($ in thousands, except share and per share amounts)

For the three-month period ended June 30,
2025 2026

Revenues and other income
Revenues 16,725 5,229
Other income 1,469 1,675
Total revenues and other income 18,193 6,904
Operating expenses
Research and development expenses (23,080 ) (24,976 )
Selling, general and administrative expenses (5,078 ) (5,739 )
Other operating income 378 290
Total operating expenses and other operating income (27,779 ) (30,425 )
Operating loss (9,586 ) (23,521 )
Net Financial gain (loss) (14,150 ) 1,727
Income tax - (25 )
Net loss (23,736 ) (21,819 )
Basic and diluted net loss per share attributable to shareholders of Cellectis ($/share) (0.24 ) (0.22 )
Number of shares used for computing (basic and diluted) 100,305,204 100,647,451

(Press release, Cellectis, AUG 6, 2026, View Source [SID1234669789])

On August 6, 2026 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported financial results for the second quarter ended June 30, 2026.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"This quarter, we executed strongly across our portfolio while rapidly integrating Amicus into BioMarin’s operations and advancing plans to accelerate growth for GALAFOLD and POMBILITI + OPFOLDA, and extending the benefit of these medicines to more patients worldwide," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "Strong global demand led us to increase full-year VOXZOGO revenue guidance to at least $1 billion in 2026. Adding to this momentum is the opportunity to advance our second potential indication with VOXZOGO, hypochondroplasia, based on recent pivotal data that exceeded our expectations." Mr. Hardy added, "With our larger, more diversified commercial portfolio of innovative medicines, we are positioned to deliver additional growth and increased profitability. We expect strong execution through the remainder of 2026, bringing together our expanded portfolio, scale and disciplined integration efforts to reach more patients living with serious genetic conditions around the world."

2026 Business and Pipeline Highlights
Innovation
•BioMarin recently submitted its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the approval of VOXZOGO for the treatment of hypochondroplasia. If approved, VOXZOGO would be the first targeted therapy for the treatment of hypochondroplasia, with a potential 2027 launch. The company plans to provide an update on the application status as part of its third quarter earnings update.

•In May, the company announced that the Phase 3 CANOPY-HCH-3 study of VOXZOGO in children with hypochondroplasia met its primary endpoint, with a statistically significant increase in annualized growth velocity (AGV) at week 52 versus placebo (LS mean difference +2.33 cm/yr, p<0.0001), along with significant improvements in standing height, height Z-score, and the key secondary endpoint of arm span. The full Phase 3 dataset will be shared in a late-breaking oral presentation at the European Society for Paediatric Endocrinology Annual Meeting in September.
•In June, at the Endocrine Society Annual Meeting (ENDO 2026), a Phase 2 investigator-sponsored three-year extension study of VOXZOGO in 13 children with hypochondroplasia showed sustained improvements in growth with a favorable safety profile. Mean AGV increased from 4.27 cm/year at baseline to 7.24 cm/year at year one (p<0.001) and remained above baseline through year three, with mean height standard deviation score (SDS) improving 0.72 over the three years.
•Also at ENDO 2026, the company presented Phase 1 data for BMN 333, BioMarin’s long-acting C-type natriuretic peptide (CNP) for achondroplasia. In a single-ascending-dose study in healthy adults, BMN 333 demonstrated sustained exposure supporting weekly dosing and was well tolerated, with free CNP exposure at the highest dose more than 13-fold that of another long-acting CNP agent, reflecting its potential to become a new standard of care in achondroplasia. The Phase 2/3 study is enrolling, with a data update expected in 2027.
•In July, BioMarin announced that the FDA accepted its sNDA for full approval of VOXZOGO in children with achondroplasia, with a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2027.
•In the second quarter, the European Commission approved PALYNZIQ for adolescents 12 years and older with phenylketonuria (PKU). PALYNZIQ is the only therapy that enables people with PKU to reach physiologic Phe levels while reducing dietary restrictions, regardless of severity.
•During the quarter, BioMarin added BMN 820 (formerly DMX-200) to its portfolio, a first-in-class oral CCR2 inhibitor for focal segmental glomerulosclerosis (FSGS) for which BioMarin holds exclusive U.S. commercialization rights. BMN 820 has the potential to treat a broad FSGS population, regardless of nephrotic syndrome status, and represents a U.S. total addressable patient population of approximately 30,000. The Phase 3 ACTION 3 trial is ongoing, with pivotal data expected in 2028.
•BMN 351, BioMarin’s Phase 1/2 candidate for Duchenne muscular dystrophy, continued in development. The company expects to provide a program update by year-end.
•Following the pivotal ENERGY 3 trial results, previously announced in May, in which BMN 401 did not meet one of its two co-primary endpoints for the treatment of ENPP1 deficiency, BioMarin has now made the decision to discontinue development of BMN 401 across all indications.
•In July, BioMarin and the n-Lorem Foundation entered a collaboration and global exclusive license agreement to develop a first-in-disease antisense oligonucleotide (ASO) medicine for ReNU syndrome, a serious, rare neurodevelopmental condition with no approved targeted therapies. ReNU syndrome has an expected global population of approximately 100,000.
Growth
•BioMarin expects peak revenue for GALAFOLD to be approximately $1.4 billion by the mid-2030s and for POMBILITI + OPFOLDA to be approximately $1.2 billion by the mid-to-late-2030s. BioMarin expects these high growth therapies to benefit from its global scale and proven commercial capabilities.
•Metabolic Conditions (formerly Enzyme Therapies) revenue grew 25% Y/Y in the second quarter of 2026, driven by the additions of GALAFOLD and POMBILITI + OPFOLDA and continued strength from PALYNZIQ. The number of patients on therapy grew across all BioMarin-marketed therapies, both Y/Y and sequentially.
•Strong U.S. and global demand led to increased full-year 2026 VOXZOGO revenue guidance to a low end of $1 billion. The number of children being treated with VOXZOGO globally increased by more than 20% Y/Y in the second quarter. In the U.S., the majority of new patient starts were under two years of age, and the region drove approximately 25% of total VOXZOGO revenue during the quarter.

Value Commitment

•As part of the acquisition of Amicus, which closed on April 27, 2026, the company identified approximately $280 million of cost reductions on a GAAP basis, and approximately $220 million of cost reductions on a Non-GAAP basis, expected to be fully realized in 2028, representing an approximately 50% reduction from Amicus-reported 2025 GAAP and Non-GAAP operating expenses, respectively. Synergies reflect a reduction of Amicus’ legacy labor costs and external spend and are expected to be largely driven by general and administrative functions, with the large majority of sales and marketing capabilities retained to support continued commercial growth.
•GALAFOLD and POMBILITI + OPFOLDA, combined, are expected to reach over 60% Non-GAAP Operating Margin by 2030.
•The company is targeting gross leverage below 2.5 times by mid-year 2027, an acceleration by approximately one year of prior timeline guidance provided at deal announcement, supported by profitability growth of the combined company.
Second Quarter 2026 Financial Highlights
•Total Revenues for the second quarter of 2026 were $990 million, an increase of $165 million compared to the same period in 2025, primarily driven by revenues from GALAFOLD and POMBILITI + OPFOLDA, which were acquired from Amicus on April 27, 2026, as well as new patients initiating VOXZOGO therapy across all regions and growth in U.S. patients treated with PALYNZIQ. These increases were partially offset by lower VIMIZIM revenue due to the timing of large government orders outside the U.S. and lower ALDURAZYME sales volume due to the timing of order fulfillment to Sanofi.

•GAAP Net Income for the second quarter of 2026 decreased to $45 million compared to $241 million for the same period in 2025. The decrease was primarily driven by the acquisition of Amicus, including integration and restructuring costs, intangible asset amortization, interest expense from debt issued to finance a portion of the transaction, and amortization of inventory fair value step-up. Other drivers included higher sales and marketing spend to support newly acquired products and global expansion of VOXZOGO and higher Research and Development (R&D) spend related to BMN 401, which was acquired in the third quarter of 2025, partially offset by higher gross profit driven by revenue growth as described above.

•Non-GAAP Income for the second quarter of 2026 decreased to $236 million compared to $282 million for the same period in 2025. The decrease was primarily driven by higher interest expense, higher sales and marketing spend to support newly acquired products and global expansion of VOXZOGO, and higher R&D spend related to BMN 401, partially offset by higher gross profit driven by revenue growth as described above.

Financial Highlights (in millions of U.S. dollars, except per share data, unaudited)
Three Months Ended
June 30, Six Months Ended
June 30,
2026 2025 % Change 2026 2025 % Change
Total Revenues $990 $825 20% $1,756 $1,571 12%
Net Product Revenues by Product:
VOXZOGO $253 $221 14% $472 $435 9%
Metabolic Conditions:
VIMIZIM $194 $215 (10)% $405 $404 —%
NAGLAZYME
135 129 5% 265 243 9%
PALYNZIQ 135 106 27% 225 199 13%
GALAFOLD #N/A #N/A NM #N/A #N/A NM
BRINEURA
51 49 4% 98 89 10%
ALDURAZYME 44 56 (21)% 80 105 (24)%
POMBILITI + OPFOLDA #N/A #N/A NM #N/A #N/A NM
Total Metabolic Conditions Revenue #N/A #N/A #N/A #N/A #N/A #N/A
KUVAN
$24 $27 (11)% $48 $52 (8)%
ROCTAVIAN(1)
$12 $9 33% $14 $20 (30)%
GAAP Net Income
$45 $241 (81)% $150 $426 (65)%
Non-GAAP Income (2)
$236 $282 (16)% $385 $502 (23)%
GAAP Operating Margin % (3)
11.2% 33.5% 13.7% 31.9%
Non-GAAP Operating Margin % (2)
36.4% 39.9% 31.0% 37.9%
GAAP Diluted EPS
$0.23 $1.23 (81)% $0.77 $2.19 (65)%
Non-GAAP Diluted EPS (2)
$1.20 $1.44 (17)% $1.96 $2.57 (24)%

Updated 2026 Full-Year Financial Guidance (in millions, except EPS amounts)

•Total Revenues, VOXZOGO, and Non-GAAP Diluted EPS guidance raised, reflecting strong first-half 2026 performance and second-half 2026 revenue expectations for both Metabolic Conditions and VOXZOGO.
•Guidance reflects post-close contributions from Amicus beginning April 27, 2026.
•BioMarin will continue to include interest expense related to the Amicus financing in both GAAP and Non‑GAAP financial results. Based on current rates, interest expense associated with the financing is estimated at approximately $200 million on an annualized basis, with Term Loans and Senior Notes scheduled to mature after 2030.
Item Provided on May 4, 2026
Updated August 6, 2026
Midpoint Growth (Y/Y)
Total Revenues $3,825 to $3,925 $3,875
to
$3,925 21%
Metabolic Conditions $2,725 to $2,775 Unchanged 31%
VOXZOGO $975 to $1,025 $1,000 to $1,050 11%
Other Revenues(1)
$100 to $125 Unchanged
Non-GAAP Diluted EPS (2)(3)(4)
$4.85 to $5.05 $4.90 to $5.10 59%

BioMarin will host a conference call and webcast to discuss second quarter 2026 financial results today, Thursday, August 6, 2026, at 4:30 p.m. ET. This event can be accessed through this link or on the investor section of the BioMarin website at www.biomarin.com.
U.S./Canada Dial-in Number: 800-715-9871 Replay Dial-in Number: 800-770-2030
International Dial-in Number: 646-307-1963
Replay International Dial-in Number: 609-800-9909
Conference ID: 3551298
Conference ID: 3551298

(Press release, BioMarin, AUG 6, 2026, View Source [SID1234669788])

On August 6, 2026 Barinthus Biotherapeutics plc (NASDAQ: BRNS) ("Barinthus Bio," or the "Company"), reported its financial results for the quarter ended June 30, 2026, and provided an overview of the Company’s corporate developments. Barinthus Bio is an immunology and inflammation ("I&I") company focused on developing therapies that promote immune tolerance with curative potential.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"During the second quarter, our primary focus remained advancing the proposed combination with Clywedog and progressing the clinical development of VTP-1000, our lead asset for celiac disease," said Bill Enright, Chief Executive Officer of Barinthus Bio. "We are pleased to announce the completion of enrollment in the multiple ascending dose portion of the Phase 1 AVALON trial and remain on track to report topline data in the fourth quarter of 2026. We believe VTP-1000 has the potential to address a significant unmet need in celiac disease and to be an important driver of long-term value for shareholders of the combined company."

Recent Corporate Developments

•During the third quarter of 2026, Barinthus Bio completed enrollment in all cohorts in the MAD portion of the Phase 1 AVALON clinical trial.
•On June 30, 2026, Barinthus Bio received a notice (the "Extension Notice") from the Nasdaq Stock Market ("Nasdaq") informing the Company that Nasdaq had granted the Company an additional 180 calendar days, or until December 28, 2026, to regain compliance with the Bid Price Requirement for continued listing on the Nasdaq Capital Market under Nasdaq Listing Rule 5550(a)(2). In connection with the Extension Notice, the listing of the Company’s American Depositary Shares (the "ADSs") was transferred from the Nasdaq Global Market to the Nasdaq Capital Market, effective as of July 2, 2026. The Extension Notice has no other immediate effect on the listing of the ADSs.

Upcoming Milestones

Celiac Disease (VTP-1000):
•Data from the MAD portion of the Phase 1 AVALON clinical trial, which includes a gluten challenge following three doses of test medication, is expected in the fourth quarter of 2026.
•An abstract outlining Phase 1 Single Ascending Dose data for VTP-1000 has been selected for poster presentation at the American College of Gastroenterology ("ACG") Annual Scientific Meeting, to be held October 9–14, 2026 in Nashville, Tennessee. The poster will be presented by Dr. Adam Bledsoe, from the Division of Gastroenterology and Hepatology at Mayo Clinic.

Corporate:
•Barinthus Bio expects to complete the merger with Clywedog in the second half of 2026 which will result in a differentiated biopharmaceutical company focusing on metabolic and autoimmune diseases with three potentially disease-modifying clinical stage therapies.
•At closing, the combined company will be renamed "Clywedog Therapeutics Holdings, Inc." and is expected to trade on the NASDAQ under the new ticker symbol "CLYD." The combined company’s estimated cash runway is expected to extend through 2027, supported by existing cash and additional investments by OrbiMed and Torrey Pines Investments, LLC, both existing shareholders in Clywedog, and new investors.

Second Quarter 2026 Financial Highlights

•Cash: As at June 30, 2026, cash, cash equivalents and restricted cash were $59.6 million, compared to $67.2 million as of March 31, 2026. The $7.6 million decrease was a result of the net cash used in operating activities of which $8.1 million was used for the development of the Company’s pipeline and general corporate expenses, and a $0.5 million translational gain from the conversion of balances in pound sterling denominated entities to the United States dollar reporting currency. Based on standalone research and development plans, the Company expects its available resources to fund its operating expenses and capital expenditure requirements for at least the next 12 months from the date of issuance of the financial statements.
•Research and Development Expenses: Research and development expenses were $3.9 million in the second quarter of 2026 compared to $3.6 million for the first quarter of 2026. The increase was primarily attributable to an increase in VTP-1000 Celiac program cost in second quarter of 2026 offset by reduced activity in the Barinthus legacy asset clinical programs and the reduction in workforce. The quarter-on-quarter research and development expenses are outlined in the following table, with the expense primarily attributable to the continued progression of the Phase 1 AVALON clinical trial of VTP-1000 in celiac disease, and the reduced activity on legacy assets in infectious disease and oncology. It is anticipated that research and development expenses related to the Barinthus legacy assets in infectious disease and oncology will continue to decrease going forward as the clinical trials complete, and that research and development expenses related to the autoimmune program will continue at current levels or increase, as the clinical development continues.
Three months ended June 30, 2026 Three months ended March 31, 2026 Change
$000
$000
$000
Direct research and development expenses by program:
VTP-1000 Celiac $ 2,936 $ 1,422 $ 1,514
Barinthus legacy assets1
506 1,480 (974)
Total direct research and development expenses 3,442 2,902 540
Indirect research and development expenses:
Personnel-related (including share-based compensation) 440 503 (63)
Facility related 15 87 (72)
Other indirect costs 26 101 (75)
Total indirect research and development expenses 481 691 (210)
Total research and development expense $ 3,923 $ 3,593 $ 330

1 In January 2025, we announced a strategic focus on developing a pipeline in I&I, and the deprioritization of our programs in infectious disease and oncology. The following programs were previously presented separately and have been grouped collectively as "Barinthus Legacy Assets" for both years presented: VTP-300 HBV, VTP-850 Prostate Cancer, VTP-200 HPV, VTP-600 NSCLC, VTP-500 MERS and other and earlier stage programs.
•General and Administrative Expenses: General and administrative expenses were $7.1 million in the second quarter of 2026, compared to $2.5 million for the first quarter of 2026. The increase of $4.6 million related primarily to an increase in unrealized losses on foreign exchange driven mainly by translation of United States dollar balances in pound sterling denominated entities and an increase in professional fees.
•Net Loss: For the second quarter of 2026, the Company generated a net loss attributable to its shareholders of $10.6 million or $(0.26) per share on both basic and fully diluted bases, compared to a net loss attributable to its shareholders of $5.5 million, or $(0.14) per share on both basic and fully diluted bases for the first quarter of 2026.

(Press release, Barinthus Biotherapeutics, AUG 6, 2026, View Source [SID1234669787])

On August 6, 2026 Agenus Inc. (Nasdaq: AGEN), a leader in immuno-oncology innovation, reported financial results for the second quarter ended June 30, 2026, and provided an update on its financing-supported strategy to advance botensilimab (BOT), a multifunctional, Fc-enhanced anti-CTLA-4 antibody, plus balstilimab (BAL), an anti-PD-1 antibody, in a curative-intent treatment setting before surgery in high-risk, resectable microsatellite-stable (MSS) colon cancer.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

As previously announced, Agenus completed an oversubscribed private placement in July 2026 structured to support ROBBIN, its planned global registrational Phase 3 trial of neoadjuvant (before surgery) BOT+BAL in patients with high-risk Stage II and Stage III microsatellite-stable (MSS) colon cancer. MSS disease accounts for approximately 85% of early-stage colorectal cancers. In the population targeted by ROBBIN, treatment remains centered on surgery followed by chemotherapy or observation, with no new curative-intent therapies approved in more than 20 years.,

The decision to accelerate ROBBIN is grounded in previously reported findings from the independent NEST and UNICORN studies evaluating neoadjuvant BOT+BAL treatment in patients with Stage II/III MSS colorectal cancer. Treatment before surgery produced deep pathologic responses, while recent advanced-disease updates provide complementary evidence of durable BOT+BAL immune activity.

ROBBIN addresses an estimated 38,000 newly diagnosed patients annually in the United States and more than 200,000 worldwide. Agenus estimates that this population represents an annual addressable sales opportunity of more than $7 billion.

"The financing completed in July gives us a clear path to act on the clinical evidence supporting BOT+BAL in a large, underserved patient population," said Garo H. Armen, Ph.D., Chairman and Chief Executive Officer of Agenus. "The previously reported neoadjuvant findings support testing whether BOT+BAL before surgery can reduce recurrence and improve the potential for cure. With capital aligned to ROBBIN’s development milestones, we are positioned to pursue that opportunity with focus and urgency."

Previously Announced Financing Supports ROBBIN Execution
The private placement, announced and completed in July, provided approximately $85 million in upfront gross proceeds and included milestone-aligned warrants that could provide up to approximately $255 million in additional gross proceeds if fully exercised. The warrant structure aligns potential additional capital with planned ROBBIN milestones.

Based on the company’s current operating plan, the upfront proceeds are expected to support ROBBIN initiation, regulatory alignment and company operations through Q3 2027. Assuming full exercise of the warrants, the financing is expected to support the planned ROBBIN program and company operations through year-end 2031.

Agenus continues to implement disciplined cost-management measures and is directing internal resources toward ROBBIN execution, clinical and access-program supply, regulatory activities and supporting data generation.

Clinical Evidence Reinforces BOT+BAL’s Differentiated Profile
Previously reported findings from the independent NEST and UNICORN studies provide the direct neoadjuvant clinical rationale for ROBBIN in Stage II and Stage III MSS colon cancer.

Among 38 BOT+BAL-treated patients, approximately 30% achieved a pathologic complete response (pCR; no viable tumor found at surgery), and approximately 40% achieved a major pathologic response (MPR; 10% or less viable tumor remaining). At the applicable data cutoffs, no disease recurrences had been reported. Manuscripts with longer-term follow-up from both studies are anticipated in the second half of 2026.

Recent advanced-disease updates further reinforced the durability of BOT+BAL activity. At ESMO (Free ESMO Whitepaper) Gastrointestinal Cancers Congress 2026, follow-up from the fully enrolled 123-patient Phase 1b cohort in refractory MSS metastatic colorectal cancer without active liver metastases showed median overall survival of 21.2 months and three-year overall survival of 33%. At last follow-up, 17% of patients were alive and off all systemic cancer therapy. No new safety signals or treatment-related deaths were reported.

During the quarter, durable BOT+BAL activity was also reported in checkpoint-refractory melanoma and post-immunotherapy hepatocellular carcinoma, further supporting the combination’s activity across tumors that had resisted prior immunotherapy or multiple lines of treatment.

Expanding Patient Access and Supporting Treatment Continuity
Agenus broadened authorized access to BOT+BAL during the second quarter through France’s national Autorisation d’Accès Compassionnel (AAC) program and physician-led paid named-patient programs in additional countries.

The programs now span a broader network of countries, treating institutions and healthcare professionals. Agenus recognized $6.4 million in pre-commercial product revenue from authorized access programs during the second quarter, compared with $4.6 million in the first quarter of 2026.

As Agenus concentrates its development resources on ROBBIN, the access programs enable the company to continue supporting eligible patients with advanced disease, sustain engagement with experienced treating physicians and advance its neoadjuvant registrational strategy.

As part of prioritizing resources toward the ROBBIN trial, Agenus discontinued its planned future funding commitment to BATTMAN, the Phase 3 study sponsored by the Canadian Cancer Trials Group (CCTG) evaluating BOT+BAL in refractory MSS metastatic colorectal cancer. Agenus was one of the study’s funding sources and supplied BOT+BAL, while CCTG sponsored and conducted the trial. Following Agenus’s funding decision, CCTG formally terminated the study. The decision reflected financing and development priorities and was not driven by enrollment performance, efficacy or safety findings.

Agenus remains committed to supporting continued BOT+BAL treatment for patients already enrolled in BATTMAN when medically appropriate and permitted under applicable requirements. In France, eligible patients may continue to access BOT+BAL through the established national AAC program. Agenus has also established physician-led compassionate-access pathways in Canada, Australia and New Zealand, the other countries in which BATTMAN had been planned to enroll patients. The pathways in Canada, Australia and New Zealand will remain open to new physician requests through December 31, 2026.

Second Quarter 2026 Financial Results
Revenue

Total revenue for the second quarter of 2026 was $34.5 million, compared with $25.7 million a year earlier. This included $6.4 million in pre-commercial BOT+BAL product revenue from authorized patient-access programs and $28.1 million in non-cash royalty revenue, up from $24.8 million. Non-cash royalty revenue relates to royalty interests Agenus previously monetized and does not provide cash to the company.

Total revenue for the first six months of 2026 was $68.3 million, compared with $49.8 million a year earlier, including $11.0 million in pre-commercial product revenue and $57.3 million in non-cash royalty revenue.

Near-Term Milestones

•
Manuscripts with longer-term follow-up from NEST and UNICORN anticipated in the second half of 2026
•
Investigator-sponsored BOT+BAL presentations at ESMO (Free ESMO Whitepaper) 2026
•
ROBBIN initiation and first patient dosing anticipated in the first quarter of 2027
Corporate Webcast Information

Agenus will host a corporate strategy webcast, including a live question-and-answer session, on Thursday, September 10, 2026, at 4:30 p.m. ET. Following the company’s recent financing webcast, the September event will provide a more comprehensive discussion of Agenus’s corporate priorities, including the acceleration of BOT+BAL in neoadjuvant colon cancer through ROBBIN, upcoming clinical and data milestones, and ongoing patient-access efforts. The webcast was previously anticipated for late August; the revised timing allows for broader speaker participation and a more substantive strategic discussion. Additional details, including the agenda and access information, will be announced prior to the event.

(Press release, Agenus, AUG 6, 2026, View Source [SID1234669786])

On August 6, 2026 Circio Holding ASA (OSE: CRNA), a biotechnology company developing novel circular RNA expression technology for gene and cell therapy, reported that it has been selected for an oral presentation at the European Society of Gene and Cell Therapy annual meeting in Hamburg, Germany, 27-30 October 2026.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The ESGCT Annual Meeting is the leading gene and cell therapy conference in Europe and the second largest globally, recognized as one of the most prestigious scientific meetings in the field. The meeting is widely attended by pharma industry professionals, academics, patient advocacy organizations and life science media. Only a small number of submitted abstracts are selected for oral presentation, underscoring the scientific quality and relevance of the work presented.

"ESGCT is one of the most important international meetings in the gene and cell therapy field, and it is very important for Circio that the organizing committee has selected one of Circio´s abstracts for an oral presentation," said Dr. Thomas B Hansen, CTO of Circio. "Presenting our circVec platform at ESGCT provides an excellent opportunity to showcase how our proprietary DNA vector technology has the potential to significantly enhance next-generation gene and cell therapies to a highly relevant audience of life science and industry leaders. This meeting also provides an important forum to engage with existing and prospective collaborators and partners as we continue to advance our R&D programs and expand commercial opportunities."

Circio has submitted three abstracts for ESGCT 2026, of which one has been selected for oral presentation and two for poster presentation. Further information about the Circio presentations and the full abstract texts will be released on the general abstract publication date, 27 October 2026.

Presentation title: circVec: A DNA Vector Platform Harnessing Intracellular Circular RNA Biogenesis for Next-Generation T Cell Therapies

Session: Non-viral vectors/nanotechnology and RNA therapeutics I

Time & location: Wednesday 28 October at 10:00am – Hamburg, Germany

(Press release, Circio, AUG 6, 2026, View Source [SID1234669740])