On May 6, 2026 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the quarter ended March 31, 2026, and provided a business update.

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!

"We began 2026 with continued strong execution across our business, led by sustained growth of ORLADEYO and solid progress across our pipeline," said Charlie Gayer, President and Chief Executive Officer of BioCryst. "ORLADEYO continues to grow because its differentiated oral profile and high level of attack control meet the needs of an increasing number of people living with hereditary angioedema. At the same time, we remain on track with enrollment in our navenibart and BCX17725 pipeline programs. We are also pleased to partner again with Neopharmed Gentili for European rights to navenibart. This progress underscores our strategy to focus on rare diseases where we have deep expertise, execute efficiently, and allocate capital thoughtfully to drive sustainable value for patients and shareholders."

Business & Corporate Updates

New patient prescriptions in Q1 2026 continued to be strong, driving ORLADEYO revenue of $148.3 million (+11% y-o-y; +21% y-o-y on a comparable basis excluding European revenue).
Patient enrollment in ALPHA-ORBIT, the ongoing pivotal study of navenibart in hereditary angioedema, is on track to be completed by the end of this June. The program remains on track to support regulatory filing in the US by the end of 2027. Navenibart is an investigational, long-acting plasma kallikrein inhibitor being studied with every three-month and every six-month subcutaneous dosing.
In May, the company announced that it entered into a licensing agreement with an Irish affiliate of Neopharmed Gentili for exclusive rights to commercialize navenibart in Europe for the prophylaxis of hereditary angioedema. BioCryst will receive upfront consideration of $70M and will be eligible to receive up to $275M in future regulatory and sales milestone payments. BioCryst will also receive tiered royalties on net sales ranging from 18% to 30%. Navenibart is an investigational product that has not yet received regulatory approval in the US or Europe.
The company is studying BCX17725, an investigational KLK5 inhibitor for the treatment of Netherton syndrome, in a Phase 1 trial. The company has begun dosing in Part 4 of this trial, which will enroll up to 12 patients for three months, and expects to report data from this part by the end of 2026.
In Q1 2026, the company ended development of avoralstat, a plasma kallikrein inhibitor for the treatment of diabetic macular edema, to focus the pipeline on rare diseases.
In April, the company appointed Sandeep M. Menon as Chief Research and Development Officer. Dr. Menon brings deep drug development expertise to the leadership team and will lead the company’s R&D efforts with a focused, disciplined, and capital efficient approach.
First Quarter 2026 Financial Results

On January 23, 2026, BioCryst completed the acquisition of Astria Therapeutics, Inc. ("Astria"). The transaction was accounted for as an asset acquisition and as a result, BioCryst recognized a special, non-cash expense of $697.8 million in Q1 2026 related to the acquired in-process research and development asset for navenibart.

The accompanying tables provide GAAP and non-GAAP financial information. Non-GAAP measures include adjustments, as applicable, for the sale of the European ORLADEYO business on October 1, 2025, stock-based compensation, and expenses incurred in connection with the acquisition of Astria, including acquired in-process research and development expense, assembled workforce amortization, severance and retention related costs, and the portion of the Astria stock option payout attributable to post-combination service. Management believes that the presentation of these non-GAAP figures can provide greater transparency into the financial results of core, ongoing operations and improve comparability across reporting periods by excluding items that are non-recurring or other items that may vary significantly from period to period.

BioCryst recorded a GAAP operating loss of $701.6 million for the first quarter of 2026, primarily reflecting the special, non-cash charge related to acquired in-process research and development. On a non-GAAP basis, the company recorded an operating profit of $54.2 million. Additional details on individual adjustments are included in the accompanying financial tables.

Cash, cash equivalents, restricted cash and investments at March 31, 2026, totaled $260.8 million. On a pro-forma basis, including net proceeds of $70 million from the license of European navenibart rights to Neopharmed Gentili after quarter end, cash, cash equivalents, restricted cash and investments at March 31, 2026, totaled $330.8 million.

Financial Outlook for 2026

The company maintained its expectation for full year 2026 global net ORLADEYO revenue to be between $625 million and $645 million and for full year 2026 total revenue, including RAPIVAB (peramivir injection), to be between $635 million and $660 million.

The company also maintained its expectation for full year 2026 non-GAAP operating expenses, excluding stock-based compensation, restructuring, and transaction-related costs, to be between $450 million and $470 million.

Item As of May 6, 2026 As of February 26, 2026
ORLADEYO revenue Unchanged $625 million to $645 million
Total revenue Unchanged $635 million to $660 million
Non-GAAP operating expense Unchanged $450 million to $470 million

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, MAY 6, 2026, View Source [SID1234665184])

On May 6, 2026 BeOne Medicines Ltd. (NASDAQ: ONC; HKEX: 06160; SSE: 688235), a global oncology company, reported financial results and corporate updates from the first quarter of 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!

John V. Oyler, Co-Founder, Chairman, and CEO, BeOne, said:

"These strong first-quarter results reinforce BeOne’s continued growth as a global oncology leader, driven by disciplined commercial execution, and underpinned by our established hematology leadership, and an impressive, rapidly emerging solid tumor pipeline. The sustained competitive advantages of our global superhighway for clinical development and manufacturing are now clear. BRUKINSA has firmly established itself as the foundational, best-in-class BTK inhibitor with unmatched long-term efficacy and safety data for the treatment of CLL and as the only BTKi with proven efficacy superiority over ibrutinib which has resulted in clear global revenue leadership. The fixed-duration combination of sonrotoclax, a foundational, next-generation BCL2 inhibitor, and BRUKINSA represents a potential new standard-of-care in first-line CLL, with BTK CDAC BGB-16673 emerging as a potential first-in-class therapy in the relapsed or refractory setting. With more than 20 abstracts across our hematology and solid tumor pipeline accepted for presentation at ASCO (Free ASCO Whitepaper), BeOne has solidified its position as a leading oncology company."

(Amounts in thousands of U.S. dollars and unaudited)

Three Months Ended

March 31,

2026

2025

% Change

Net product revenues

$

1,487,329

$

1,108,530

34

%

Other revenue

$

26,109

$

8,749

198

%

Total revenue

$

1,513,438

$

1,117,279

35

%

GAAP income from operations

$

249,902

$

11,102

2,151

%

Adjusted income from operations*

$

414,394

$

139,357

197

%

GAAP net income

$

227,357

$

1,270

17,802

%

Adjusted net income*

$

375,042

$

136,137

175

%

GAAP basic EPS per ADS

$

2.05

$

0.01

20,400

%

Adjusted basic EPS per ADS*

$

3.38

$

1.27

166

%

GAAP diluted EPS per ADS

$

1.96

$

0.01

19,500

%

Adjusted diluted EPS per ADS*

$

3.24

$

1.22

166

%

Free Cash Flow*

$

160,547

$

(12,325

)

1,403

%

* For an explanation of our use of non-GAAP financial measures, refer to the "Note Regarding Use of Non-GAAP Financial Measures" section later in this press release and for a reconciliation of each non-GAAP financial measure to the most comparable GAAP measures, see the table at the end of this press release.

First Quarter 2026 Financial Results

Product Revenue totaled $1.5 billion for the first quarter of 2026, representing growth of 34% compared to the prior-year period.

BRUKINSA: Global sales totaled $1.1 billion for the first quarter of 2026, representing growth of 38% compared to the prior-year period; U.S. sales of BRUKINSA totaled $761 million in the first quarter of 2026, representing growth of 35% compared to the prior-year period.
TEVIMBRA (tislelizumab): Global sales totaled $206 million in the first quarter of 2026, representing growth of 20% compared to the prior-year period.
Amgen in-licensed products: Global sales totaled $142 million in the first quarter of 2026, representing growth of 25% compared to the prior-year period.
Gross Margin as a percentage of global product sales for the first quarter of 2026 was 89%, compared to 85% in the prior-year period on a GAAP basis. The gross margin percentage increased due to a proportionally higher sales mix of global BRUKINSA compared to other products in our portfolio. Gross margin also benefited from productivity improvements resulting in lower costs for both BRUKINSA and TEVIMBRA.

Operating Expenses

The following table summarizes operating expenses for the first quarter of 2026:

GAAP

Non-GAAP

(unaudited, in thousands, except percentages)

Q1 2026

Q1 2025

% Change

Q1 2026

Q1 2025

% Change

Research and development

$

541,224

$

481,887

12

%

$

465,904

$

421,195

11

%

Selling, general and administrative

$

555,097

$

459,288

21

%

$

471,993

$

395,511

19

%

Total operating expenses

$

1,096,321

$

941,175

16

%

$

937,897

$

816,706

15

%

Research and Development (R&D) Expenses increased for the first quarter of 2026 compared to the prior-year period on both a GAAP and adjusted basis due to advancing preclinical programs into the clinic and early clinical programs into late stage.

Selling, General and Administrative (SG&A) Expenses increased for the first quarter of 2026 compared to the prior-year period on both a GAAP and adjusted basis due to continued investment to support commercial growth. SG&A expenses as a percentage of product sales were 37% for the first quarter of 2026, compared to 41% in the prior-year period.

Net Income and Basic/Diluted Earnings Per Share

GAAP net income for the first quarter of 2026 was $227 million, an increase of $226 million over the prior-year period, primarily attributable to revenue growth and improved operating leverage.

For the first quarter of 2026, basic and diluted earnings per share were $0.16 and $0.15 per share and $2.05 and $1.96 per American Depositary Share (ADS), compared to basic and diluted earnings per share of $0.00 per share and $0.01 per ADS in the prior-year period.

Free Cash Flow for the first quarter of 2026 was $161 million, representing an increase of $173 million over the prior-year period.

For further details on BeOne’s First Quarter 2026 Financial Statements, please see BeOne’s Quarterly Report on Form 10-Q for the first quarter of 2026 filed with the U.S. Securities and Exchange Commission.

Updated Full Year 2026 Guidance

BeOne’s financial guidance is summarized below:

Prior FY 2026 Guidance

Current FY 2026 Guidance1

Total revenue

$6.2 – $6.4 billion

$6.3 – $6.5 billion

GAAP gross margin %

High-80% range

High-80% range

GAAP operating expenses2

(combined R&D and SG&A)

$4.7 – $4.9 billion

$4.7 – $4.9 billion

GAAP operating income2

$700 – $800 million

$750 – $850 million

Non-GAAP operating income2,3

$1.4 – $1.5 billion

$1.45 – $1.55 billion

1 Assumes May 1, 2026 foreign exchange rates.
2 Does not assume any potential new, material business development activity or unusual/non-recurring items.
3 Non-GAAP operating income is a financial measure that excludes from the corresponding GAAP measure costs related to share-based compensation, depreciation and amortization expense. Guidance assumes that Non-GAAP expenses track overall expense growth.

BeOne’s total revenue guidance for full year 2026 of $6.3 billion to $6.5 billion includes expectations for strong revenue growth driven by BRUKINSA’s leadership position in the U.S. and continued global expansion in both Europe and other important rest of world markets. Gross margin percentage is expected to be in the high-80% range and includes the impact of product mix and a full year of 2026 productivity improvements. Guidance for combined operating expenses on a GAAP basis includes expectations of investment to support growth in both commercial and research at a pace that continues to deliver meaningful operating leverage.

The Company is providing the following additional guidance on items impacting net income and earnings per ADS:

Other income (expense): Estimated range of $25 million to $50 million in expense, includes interest amortization from Royalty Pharma arrangement.
Income tax outlook: Earnings may provide sufficient positive evidence to reverse certain valuation allowances in 2026, resulting in a material tax benefit when recognized; the timing and magnitude of a potential reversal is uncertain; prior to reversal, income tax expense should trend with earnings per historical relationship. See Form 10-Q for additional updates on income tax uncertainties.
Diluted ADS outstanding: The Company expects diluted ADSs outstanding of approximately 118 million.
First Quarter 2026 Business Highlights

Core Marketed Products

BRUKINSA (zanubrutinib)

Received Orphan Drug Designation in Japan for the treatment of adult patients with relapsed or refractory (R/R) marginal zone lymphoma (MZL).
Submitted New Drug Application in Japan for R/R MZL and tablet formulation.
Sonrotoclax (BCL2 inhibitor)

Launched and commercially available in China for the treatment of adult patients with R/R mantle cell lymphoma (MCL) and R/R chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL).
Included in the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) guidelines as a recommended third-line treatment for R/R MCL patients.
TEVIMBRA (tislelizumab)

Received acceptance of a Supplemental Biologics License Application (sBLA) by the U.S. Food and Drug Administration (FDA) with Priority Review for the treatment of adult patients with first-line HER2-positive gastroesophageal adenocarcinoma (GEA) in combination with ZIIHERA (zanidatamab) and chemotherapy, based on results of the HERIZON-GEA-01 trial which demonstrated statistically significant and clinically meaningful improvement in overall survival versus trastuzumab plus chemotherapy.
Received acceptance of sBLA by the Center for Drug Evaluation (CDE) in China for the treatment of adult patients with first-line HER2-positive GEA in combination with ZIIHERA and chemotherapy.
ZIIHERA (zanidatamab)

Received acceptance of sBLA by the CDE in China for the treatment of adult patients with first-line HER2-positive GEA in combination with chemotherapy, with or without TEVIMBRA.
Select Clinical-Stage Programs

Hematology

BGB-16673 (BTK CDAC): Initiated Phase 2 cohorts in R/R MZL and Richter’s Transformation.
Breast and Gynecological Cancers

BGB-43395 (CDK4 inhibitor): Received acceptance of Phase 1 study data as a poster presentation at ASCO (Free ASCO Whitepaper).
BG-C9074 (B7-H4 ADC): Received acceptance of Phase 1 study data as a rapid oral presentation at ASCO (Free ASCO Whitepaper).
Gastrointestinal Cancers

BGB-B2033 (GPC3x41BB bispecific antibody):
Received FDA Orphan Drug Designation for hepatocellular carcinoma (HCC).
Initiated potentially registrational study in patients with HCC.
Received acceptance of Phase 1 study data as a rapid oral presentation at ASCO (Free ASCO Whitepaper).
Lung Cancer

BG-C0979 (ADAM9-targeting ADC): Initiated first-in-human study.
Inflammation and Immunology

BG-A3004 (KLRG1 mAb): Initiated first-in-human study.
Anticipated R&D Milestones

Programs

Milestones
Timing

BRUKINSA

•

Interim analysis in the Phase 3 MANGROVE study data in combination with rituximab versus bendamustine plus rituximab for the treatment of adult patients with first-line MCL.
1H 2026

•

Japan regulatory action for the treatment of adult patients with first-line gastric cancer.
1H 2026

•

U.S. FDA regulatory action for the treatment of adult patients with first-line HER2-positive GEA in combination with ZIIHERA.
2H 2026

TEVIMBRA

•

China regulatory action for the treatment of adult patients with first-line HER2-positive GEA in combination with ZIIHERA.
1H 2027

Hematology

•

Sonrotoclax (BCL2 inhibitor):

◦

FDA regulatory action on New Drug Application as monotherapy treatment of adult patients with R/R MCL.

1H 2026

◦

Phase 3 study initiation for the treatment of adult patients with R/R multiple myeloma t(11;14).

2H 2026

•

BGB-16673 (BTK CDAC):

◦

Phase 2 potential accelerated approval submission (if data support) for the treatment of adult patients with R/R CLL.

2H 2026

Breast/Gynecologic

•

BGB-43395 (CDK4 inhibitor):
Cancers

◦

Phase 3 study initiation for the treatment of adult patients with first-line HR-positive, HER2-negative metastatic breast cancer.

1H 2026

Lung Cancer

•

BON-110 (PD-1xVEGF-AxCTLA-4 trispecific antibody):

First-in-human study initiation.

1H 2026

Gastrointestinal

•

BGB-B2033 (GPC3x41BB bispecific antibody):

Cancers

◦

Pivotal Phase 3 study initiation.

2H 2026

Inflammation and

•

BGB-16673 (BTK CDAC):
Immunology

Phase 2 study initiation for the treatment of adult patients with chronic spontaneous urticaria.

2H 2026

Corporate Updates

Entered into an exclusive option with Huahui Health to license worldwide rights to HH160 (BON-110), a novel trispecific antibody targeting PD-1, VEGF-A and CTLA-4.
BeOne’s Earnings Results Webcast

The Company’s earnings conference call for the first quarter 2026 will be broadcast via webcast at 8:00 a.m. ET on Wednesday, May 6, 2026, and will be accessible through the Investors section of BeOne’s website at www.beonemedicines.com. Supplemental information in the form of a slide presentation, transcript of prepared remarks, and a replay of the webcast will also be available.

(Press release, BeOne Medicines, MAY 6, 2026, View Source [SID1234665183])

On May 5, 2026 Pfizer reported Strong First-Quarter Results And Reaffirms 2026 Guidance.

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!

(Press release, Pfizer, MAY 5, 2026, View Source [SID1234665407])

On May 5, 2026 Verrica Pharmaceuticals Inc. ("Verrica") (Nasdaq: VRCA), a therapeutics company developing and commercializing medications for the treatment of dermatological diseases, including skin cancers, reported the presentation of Phase 2 clinical data highlighting the potential abscopal effects of the Company’s novel oncolytic peptide, VP-315 (ruxotemitide), in the treatment of basal cell carcinoma (BCC) at the 2026 Society for Investigative Dermatology (SID) Annual Meeting, taking place from May 13-16, 2026, in Chicago, Illinois. Patients with BCC frequently present with multiple tumors, and approximately one-third will develop additional primary tumors over their lifetime, highlighting the need for therapies that address diseases such as BCC with multifocal 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!

"The peripheral anticancer activity we are observing well beyond the injection site suggests that VP-315 could be exerting an abscopal effect for the treatment of basal cell carcinoma," said Jayson Rieger, PhD, MBA, President and Chief Executive Officer of Verrica Pharmaceuticals. "Such activity may indicate a broader immune activation within the tumor microenvironment of non-target lesions. If this is demonstrated in further pivotal testing, it could suggest that VP-315 has a unique potential to not only effectively treat primary BCC lesions but could also address the unmet need in patients with more than one lesion. It could also benefit patients with difficult to detect, residual disease that can sometimes be left behind after surgical excision of the tumor," Dr. Rieger continued.

The poster, titled "VP-315 Demonstrates a Potential Abscopal Effect in Untreated Non-Target Basal Cell Carcinoma (BCC) Lesions" will be presented by Noah Rosenberg MD, Verrica’s Chief Medical Officer. Dr. Rosenberg commented "We are excited about the potential of VP-315 to provide a primary and/or neoadjuvant treatment option for patients with BCC, whether it be their first lesion or multiple lesions after a long history of BCC. While the best outcome for patients is to completely eliminate the tumor, which we have observed in many patients in our Phase 2 study, overall tumor size was reduced on average by 86%, which is clinically meaningful. This highlights the potential for VP-315 to improve the patient experience by reducing the size and potential complexity of future procedures, even where surgical excision is ultimately required," Dr. Rosenberg concluded.

Presentation Details:

Poster Number: LB1190
Category: Non-Melanoma Cancers and UV Biology/Injury
Poster Session Dates and Time:
Friday, May 15, 2026 (4:30 pm – 6:00 pm)
Location: (Salons B, C, D – Lower Level, Hilton Chicago)

Key Findings:

Background

VP-315 (ruxotemitide) is a potential first-in-class oncolytic chemotherapeutic peptide immunotherapy administered directly into a tumor to induce immunogenic cell death and thereby unleashing a broad spectrum of tumor antigens for T-cell responses, which may offer a non-surgical option for patients suffering from skin cancer. Verrica holds an exclusive worldwide license to develop and commercialize VP-315 for certain dermatologic oncology indications, including non-metastatic melanoma and non-metastatic merkel cell carcinoma, and intends to focus initially on basal cell and squamous cell carcinomas as the lead indications for development. VP-315 has demonstrated positive tumor-specific immune cell responses in multi-indication Phase 1/2 oncology trials.

Type of Study:
Open-Label Phase II trial

Methods

Subjects received <8 mg (in 0.5mL) intratumoral VP-315 injections in up to 2 treated lesions (TLs). Up to 3 non-treated lesions (NTLs) per subject were monitored over 12 weeks. Changes in NTL size were assessed histologically after excision on digital images in relation to tumor bed size, histologic subtype and NTL location relative to TLs.

Results:

14 untreated NTLs in 9 subjects demonstrated an overall 67% reduction in lesion size following treatment of TLs. Reductions ranged from 50-100% (superficial) and 25-100% (nodular). Complete histologic clearance was observed in 21% of NTLs (3 /14). Reductions in tumor size were observed in all NTL locations including proximal, distal and contralateral to TLs. No skip lesions were observed. Non-serious local skin reactions were reported in 1 NTL.

Conclusions

The therapeutic effects of VP-315 extended beyond treated lesions. Reductions in untreated NTLs suggest a potential abscopal effect consistent with broader immune activation within the tumor microenvironment of remote NTLs.

About Basal Cell Carcinoma

Basal cell carcinoma is the most common form of cancer in the U.S., and incidence is rising worldwide. There are approximately 3.6 million diagnoses of basal cell carcinomas in the U.S. each year, with a high unmet need for new treatment options. Basal cell carcinoma is generally treated with invasive surgery to remove the tumor, which can cause pain, infection, bleeding and scarring.

(Press release, Verrica Pharmaceuticals, MAY 5, 2026, View Source [SID1234665158])

On May 5, 2026 Compass Therapeutics, Inc. (Nasdaq: CMPX), a clinical-stage, oncology-focused biopharmaceutical company developing proprietary antibody-based therapeutics to treat multiple human diseases, reported first quarter 2026 financial results and provided a business update.

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!

"We recently announced positive data from our Phase 2/3 study of tovecimig and look forward to meeting with the FDA before filing a BLA later this year. Most patients with BTC have no approved therapeutic option in the second line setting. Tovecimig, with its strong response rate, striking progression benefit and impact on overall survival would be a compelling treatment alternative for these patients," said Thomas Schuetz, MD, PhD, Chief Executive Officer and Vice Chairman of the Board of Directors."

"In the post-checkpoint inhibitor setting where treatment alternatives are also critically needed, we have ongoing studies with very two promising candidates. Our novel PD-1 x PD-L1 checkpoint inhibitor CTX-8371 continues to demonstrate strong and durable clinical activity and we look forward to presenting dose-escalation and early expansion cohort data at ASCO (Free ASCO Whitepaper). CTX-10726, our differentiated PD-1 x VEGF-A bispecific antibody, is also in a Phase 1 study and we expect to share initial data later this year."

Pipeline Updates:

Tovecimig (DLL4 and VEGF-A bispecific antibody)

In April 2026, the Company announced data from its Phase 2/3 study of tovecimig, which it plans to include in a BLA submission, to treat patients with biliary tract cancer in the second line setting:

Overall Response Rate (primary endpoint): 17.1% for tovecimig combination (n=111), including one complete response, compared to 5.3% for paclitaxel alone (n=57)(p=0.031).
Progression-Free Survival (secondary endpoint): 4.7 months for tovecimig combination compared to 2.6 months for paclitaxel alone (HR=0.44, p<0.0001).
Overall Survival (secondary endpoint): Analysis was confounded by high crossover from the control arm (n=31) and markedly prolonged survival of these crossover patients after receiving tovecimig. The OS of the patients randomized to the tovecimig combination arm (n=111), which does not include the OS of these crossover patients later treated with tovecimig, was a median of 8.9 months.
PFS Before / After Crossover (secondary endpoint): Patients treated with tovecimig after crossing from the control arm progressed after a median 3.5 months (PFS2) in the third line setting. These same 31 patients, when initially randomized to paclitaxel alone (PFS1), had progressed more quickly, with a median of 1.9 months in the second line setting (HR=0.36, p=0.0016).
OS Crossover vs. Non-Crossover (post hoc subset analysis): In an analysis of OS in all patients initially randomized to the paclitaxel control arm (n=57), crossover patients who subsequently received tovecimig demonstrated a statistically significant improvement in median OS of 12.8 months compared to 6.1 months for non-crossover patients who received only paclitaxel (n=26)(HR=0.54, p=0.04).
Pooled OS of All Patients Treated with Tovecimig (post hoc subset analysis): For all patients treated with tovecimig, including both crossover patients and patients initially randomized to the tovecimig combination arm (n=142), the pooled median OS was 9.8 months. The median OS for patients randomized to the paclitaxel alone who did not crossover (n=26) was 6.1 months.
Safety: Tovecimig was generally well tolerated and the safety profile was consistent with prior studies, with no new safety signals identified.

The investigator sponsored trial (IST) of tovecimig in combination with the current first-line, standard-of-care regimen of gemcitabine, cisplatin, and durvalumab in patients with BTC (NCT05506943) is ongoing. The Company is evaluating multiple additional studies for tovecimig in other indications, including both ISTs and Company-sponsored studies.

CTX-8371 (PD-1 x PD-L1 bispecific antibody)

Cohort expansions for CTX-8371 have been initiated in patients with triple-negative breast cancer (TNBC, n=28), non-small cell lung cancer (NSCLC, n=28), and Hodgkin lymphoma (HL, n=12) in the post-checkpoint inhibitor setting. These indications were selected based on the deep and durable responses observed in these indications in the dose escalation portion of the study. Half of the patients with each tumor type will be dosed at 3.0 mg/kg and half will be dosed at 10.0 mg/kg.
Initial data from these cohort expansions, as well as available data from the Phase 1 dose-escalation portion of the study, will be presented at ASCO (Free ASCO Whitepaper) 2026. Additional data from the cohort expansions are expected in the fourth quarter of 2026.

CTX-10726 (PD-1 x VEGF-A bispecific antibody)

The Phase 1 study has been initiated with clinical data expected in the fourth quarter of 2026.
The Phase 1 multiple ascending dose-escalation study will include four doses (0.3, 1.0, 3.0, and 10.0 mg/kg) in a 3+3 dose-escalation design. The multi-center study will enroll patients with a prioritized set of solid tumor indications, including patients with locally advanced, unresectable or metastatic renal cell carcinoma, gastroesophageal cancer, hepatocellular carcinoma, and endometrial cancer, in whom standard of care therapies have failed.
CTX-10726 is a tetravalent PD-1 x VEGF-A bispecific antibody discovered and engineered by the Company. CTX-10726 exhibits more potent PD-1 blockade compared with data reported for other drugs in the class and the Company believes it has a unique understanding of aspects of its mechanism of action that will guide development.

CTX-471 (CD137 or 4-1BB agonist antibody)

Initiation of the Phase 2 trial of CTX-471 in patients with tumors expressing NCAM (CD56) is expected in the second half of 2026.

Financial Results

Net loss for the quarter ended March 31, 2026, was $18.3 million or $0.10 per common share, compared to $16.6 million or $0.12 per common share for the same period in 2025.

Research and Development (R&D) Expenses

R&D expenses were $13.4 million for the quarter ended March 31, 2026, as compared to $13.1 million for the same period in 2025, an increase of $0.3 million or 3%.

General and Administrative (G&A) Expenses

G&A expenses were $6.9 million for the quarter ended March 31, 2026, as compared to $4.9 million for the same period in 2025, an increase of $2.0 million or 41%. The increase was primarily driven by pre-commercialization expenses of $1.0 million and higher stock compensation (excluding stock compensation related to pre-commercialization) of $1.4 million.

Cash Position

As of March 31, 2026, cash and marketable securities were $195 million as compared to $209 million as of December 31, 2025, a decrease of $14 million, with an anticipated cash runway into 2028. During the first quarter of 2026, $18 million of net cash was used in operating activities, and this was partially offset by proceeds from exercise of common stock of $4 million.

(Press release, Compass Therapeutics, MAY 5, 2026, View Source [SID1234665157])