On April 29, 2026 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, reported financial results and updates for the first quarter ended March 31, 2026.

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"Our first-quarter performance reflects strong execution and significant progress against our 2026 strategic objectives," said Brian Goff, Chief Executive Officer, Agios. "The solid early momentum of our U.S. commercial launch of AQVESME in thalassemia highlights both the medicine’s clinical value and strong community reception. Additionally, following collaborative engagement with the FDA, we now plan to submit our mitapivat sNDA for sickle cell disease under the U.S. accelerated approval pathway in the second quarter. 2026 marks an important growth inflection point for Agios as we continue to build a sustainable rare disease company that is rooted in hematology and focused on delivering differentiated medicines that create meaningful long-term value for patients and shareholders."

First Quarter 2026 and Recent Corporate Highlights
Mitapivat (PYRUKYND and AQVESME) Commercial Performance and Update

$18.8 million in U.S. net revenue and $1.9 million in ex-U.S. net revenue in the first quarter of 2026.
U.S. net revenue was driven by the U.S. commercial launch of AQVESME (mitapivat) in thalassemia in late January 2026.
Ex-U.S. net revenue reflected demand for PYRUKYND (mitapivat) in thalassemia in Saudi Arabia.

242 AQVESME prescriptions for thalassemia were written by Risk Evaluation and Mitigation Strategy (REMS)-certified U.S. physicians as of March 31, 2026, driven by motivated prescribers and highly engaged patients.

R&D Highlights

Mitapivat
Thalassemia –
In March 2026, the Emirates Drug Establishment of the United Arab Emirates (UAE) approved PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. With this approval, PYRUKYND becomes the only medicine approved in the UAE for this broad patient population.
Mitapivat is now approved for adults with thalassemia in the U.S., Saudi Arabia, and the UAE. A marketing application for mitapivat in thalassemia is currently under review by the European Commission.
Sickle Cell Disease –
Agios confirmed plans to pursue U.S. accelerated approval for mitapivat in sickle cell disease, following completion of its pre-supplemental New Drug Application (sNDA) meeting with the U.S. Food and Drug Administration (FDA).
The FDA’s accelerated approval pathway expedites the availability of medicines that can fill a medical need for a serious condition, with the requirement of a confirmatory clinical trial to convert to a traditional approval.
The company now plans to submit an sNDA for mitapivat in sickle cell disease in the second quarter of 2026.
Tebapivat
Lower-Risk Myelodysplastic Syndromes (LR-MDS) –
Agios expects to report topline results from its Phase 2b trial in the first half of 2026. Based on findings from the Phase 2a trial, the Phase 2b open-label trial is evaluating three higher daily dose levels of tebapivat (10 mg, 15 mg, and 20 mg) over a 24-week period. The primary endpoint is the proportion of participants achieving transfusion independence, defined as being transfusion-free for at least 8 consecutive weeks during the 24-week period.

Sickle Cell Disease –
Agios expects to report topline results from its Phase 2 trial in the second half of 2026. This double-blind, randomized, placebo-controlled trial is evaluating three daily dose levels of tebapivat (2.5 mg, 5 mg, and 7.5 mg) versus matched placebo over a 12-week period. The primary endpoint is hemoglobin response, defined as a ≥1.0 g/dL increase in average hemoglobin concentration from Week 10 through Week 12 compared with baseline.
First Quarter 2026 Financial Results
For the quarter ended March 31, 2026, net loss was $99.1 million, compared to net loss of $89.3 million for the quarter ended March 31, 2025.

Net product revenue from U.S. sales of mitapivat (PYRUKYND and AQVESME) for the first quarter of 2026 was $18.8 million, compared to $8.7 million for the first quarter of 2025.

Net product revenue from ex-U.S. sales of mitapivat (PYRUKYND) for the first quarter of 2026 was $1.9 million.

Cost of sales for the first quarter of 2026 was $1.3 million.

Research and Development (R&D) Expenses were $81.1 million for the first quarter of 2026, compared to $72.7 million for the first quarter of 2025, due to workforce-related expenses supporting pipeline advancement efforts, as well as increased mitapivat process development expenses.

Selling, General and Administrative (SG&A) Expenses were $48.3 million for the first quarter of 2026, compared to $41.5 million for the first quarter of 2025, due to an increase in activities to support the U.S. commercial launch of AQVESME in thalassemia, as well as an increase in stock compensation expense.

Cash, cash equivalents and marketable securities were $1.0 billion as of March 31, 2026, compared to $1.2 billion as of December 31, 2025. Agios expects that its cash, cash equivalents and marketable securities, together with anticipated product revenue and interest income, will provide the financial independence to execute the U.S. commercial launch of AQVESME in thalassemia, prepare for the potential U.S. commercial launch of mitapivat in sickle cell disease, advance the company’s existing clinical programs, and opportunistically expand its pipeline through both internally- and externally-discovered assets.

First Quarter 2026 Conference Call Information
Agios will host a conference call and live webcast today at 8:00 a.m. ET to discuss the company’s first quarter 2026 financial results and recent business highlights. The live webcast will be accessible on the Investors section of the company’s website (www.agios.com) under the "Events & Presentations" tab. A replay of the webcast will be available on the company’s website approximately two hours after the event.

(Press release, Agios Pharmaceuticals, APR 29, 2026, View Source [SID1234664892])

On April 29, 2026 Caris Life Sciences (NASDAQ: CAI), a leading, patient-centric, next-generation AI TechBio company and precision medicine pioneer, reported the development and peer-reviewed validation of a new predictive signature to inform therapy selection in glioblastoma (GBM) patients. The study, published in Neuro-Oncology Advances, describes the development and evaluation of the model in a cohort of more than 5,800 GBM patients. The Caris AI Insights in Glioblastoma is featured on the Caris Molecular Tumor Board Report, an innovative profiling report that provides additional insight into tumor biology and is available upon request with no additional tissue required when ordering MI Cancer Seek.

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Caris Life Sciences continues to advance precision oncology by integrating multimodal real-world datasets, available with its proprietary CodeAI platform, enabling the creation of Caris AI Insights, an engine that utilizes Whole Exome Sequencing (WES), Whole Transcriptome Sequencing (WTS), and clinical data to generate deeper biological understanding and more actionable insights for clinicians and researchers.

GBM is the most common and aggressive brain cancer, with patients typically surviving around 12 months despite treatment. Current treatments include surgery to remove the tumor, radiation and chemotherapy with temozolomide (TMZ). Unfortunately, nearly half of GBM patients do not respond to TMZ and many develop resistance that leads to recurrence. Determining whether TMZ will benefit the patient is a critical step in treatment and provides insight for developing new therapies for patients who do not respond favorably.

The novel signature, developed by Caris using multimodal molecular and clinical data, is an AI-derived model designed to infer O6-methylguanine-DNA methyltransferase (MGMT) promoter methylation status from NGS data. MGMT promoter methylation is an established biomarker associated with response to TMZ therapy in GBM patients that is commonly assessed using pyrosequencing. In the peer-reviewed validation study, the signature demonstrated high concordance with pyrosequencing-based MGMT assessment and improved discrimination of overall survival outcomes across MGMT-defined patient subgroups, while also addressing variability in MGMT classification associated with traditional testing methods. These findings show that Caris’ proprietary AI signature can complement existing testing approaches and provide additional clinical insight for isocitrate dehydrogenase (IDH)-wild type GBM patients treated with TMZ.

"The Caris AI Insights signature for GBM showcases Caris’ advanced AI capabilities in our pursuit of improving cancer patient outcomes," said Caris President David Spetzler, MS, PhD, MBA. "With peer-reviewed validation demonstrating strong concordance with traditional testing approaches and clearer prognostic stratification, we believe that this signature can complement existing testing methods to improve clinical insight for glioblastoma patients treated with TMZ."

The model was trained to predict MGMT promoter methylation status, as measured by a pyrosequencing assay, using tumor profiling data from the MI Cancer Seek NGS assay, which also has FDA-approved CDx indications. As reported in the study, the signature was developed using a clinico-genomic dataset of 5,841 patients and further evaluated in a prospective cohort of more than 3,400 cases. The model stratified patients into distinct survival groups based on the signature score, with higher scores associated with significantly longer overall survival in TMZ-treated patients.

(Press release, Caris Life Sciences, APR 29, 2026, View Source [SID1234664912])

On April 29, 2026 Crescent Biopharma, Inc. ("Crescent" or the "Company") (Nasdaq: CBIO), a clinical-stage biotechnology company dedicated to rapidly advancing the next wave of therapies for cancer patients, reported financial results for the first quarter ended March 31, 2026 and recent business highlights.

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"Crescent’s execution on the advancement of our pipeline this quarter positions us for meaningful clinical data readouts in 2027 from CR-001, our potentially best-in-class PD-1 x VEGF bispecific antibody, as well as our differentiated ADC programs. The innovative design of our Phase 1/2 ASCEND study of CR-001 enables us to quickly generate comprehensive data for this next generation immuno-oncology backbone both as monotherapy and in combination with standard of care chemotherapy in multiple tumor types, and we’re also working with our partner, Kelun-Biotech, to deliver ADC combination data," said Joshua Brumm, chief executive officer of Crescent. "We are pleased that both our global ASCEND trial and the Phase 1/2 trial of CR-003 in China are progressing well. We are on track to initiate two more trials during the second half of 2026, with the first ADC combination study for CR-001 and planned clinic entry for CR-002. This continued momentum underscores our commitment to delivering transformative therapies for people living with cancer."

Recent Business Highlights & Anticipated Milestones

CR-001, PD-1 x VEGF bispecific antibody
•CR-001 is an investigational tetravalent bispecific antibody that combines two complementary, validated mechanisms in oncology via a blockade of PD-1 and VEGF. Enrollment is ongoing in ASCEND, a global, open-label Phase 1/2 clinical trial evaluating CR-001 in multiple solid tumor types, including non-small cell lung cancer (NSCLC) and various gastrointestinal and gynecological cancers, in both treatment-naïve and previously treated patients.
•A trial in progress abstract of the ASCEND study design has been accepted for poster presentation during the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting being held May 29-June 2, 2026 in Chicago: "ASCEND: A phase 1/2, dose-escalation, optimization, and dose-expansion study to evaluate the safety and antitumor activity of CR-001 in adults with locally advanced or metastatic solid tumors."

•Under its strategic collaboration, Crescent granted Sichuan Kelun-Biotech Biopharmaceutical Co., Ltd., ("Kelun-Biotech") exclusive rights to research, develop, and commercialize CR-001 (also known as SKB118) in Greater China. Kelun-Biotech plans to initiate a Phase 1/2 trial of CR-001 (SKB118) in China in the first half of 2026.

•Crescent also plans to evaluate CR-001 in combination with multiple ADCs, including CR-002 and CR-003. Initiation of the first Phase 1/2 combination trial of CR-001 with a Kelun-Biotech ADC is expected in the second half of 2026 in China.
•Crescent anticipates reporting:
•Proof-of-concept clinical data from the ASCEND trial of CR-001 in the first quarter of 2027, including initial safety, pharmacokinetics, pharmacodynamics and preliminary antitumor activity from dose escalation and backfill cohorts in first-line and previously treated patients in multiple solid tumor types. A backfill cohort of first-line NSCLC patients is planned as part of this readout.
•Initial data of CR-001 in combination with standard of care chemotherapy in first-line and previously treated patients by mid-2027 (Q2/Q3) utilizing the dose expansion part of the ASCEND trial.
•Initial data from the Phase 1/2 trial in China of CR-001 in combination with a Kelun-Biotech ADC in mid-2027 (Q2/Q3).
CR-002, topoisomerase inhibitor ADC targeting PD-L1
•CR-002 is a topoisomerase inhibitor ADC directed to PD-L1, a validated target known to have high expression in multiple solid tumors. CR-002 incorporates a PD-L1 antibody selected for high internalization to facilitate payload release in target cells and a linker designed for intracellular cleavage and high stability in circulation.
•Crescent is on track to submit an Investigational New Drug (IND) application to the FDA for CR-002 in mid-2026 to support the initiation of a Phase 1/2 trial in solid tumors in the second half of 2026, with proof-of-concept data expected in the second half of 2027.
CR-003, topoisomerase inhibitor ADC targeting integrin beta-6 (ITGB6)
•CR-003 is an investigational topoisomerase inhibitor ADC directed to ITGB6, which is overexpressed in many solid tumors with minimal expression in most normal tissues. CR-003 consists of an anti-ITGB6 fully human IgG1 monoclonal antibody conjugated via a stable, clinically validated cleavable linker.
•A Phase 1/2 trial of CR-003 (also known as SKB105) in participants with advanced solid tumors led by Kelun-Biotech in China is ongoing and proof-of-concept data are expected in the first quarter of 2027. A Phase 1/2 combination trial of CR-003 and CR-001 is expected to initiate in the first half of 2027, with initial data anticipated by year-end 2027. Under its strategic collaboration, Kelun-Biotech granted Crescent exclusive rights to research, develop, and commercialize CR-003 (SKB105) in the United States, Europe and all markets outside of Greater China.

First Quarter 2026 Financial Results

Cash position: Cash and cash equivalents were $189.2 million as of March 31, 2026, which is anticipated to fund operations into 2028.

Revenue: Revenue for the three months ended March 31, 2026 was $1.0 million compared to no revenue in 2025. The revenue recognized in 2026 is the result of the $20.0 million upfront payment received from Kelun-Biotech pursuant to the license agreement for CR-001.

Research and development (R&D) expenses: R&D expenses were $17.9 million and $10.6 million for the three months ended March 31, 2026 and 2025, respectively. The increase in R&D expenses is the result of continued development of CR-001 and CR-002.

General and administrative (G&A) expenses: G&A expenses were $7.9 million and $3.6 million for the three months ended March 31, 2026 and 2025, respectively. The increase in G&A expenses is the result of personnel costs, including share-based compensation, and professional services associated with operating as a public company.
Net loss: Net loss was $23.3 million and $15.1 million, or $0.70 and $19.63 per basic and diluted share, for the three months ended March 31, 2026 and 2025, respectively.

Shares outstanding: As of March 31, 2026, Crescent had approximately 33.4 million shares of the Company’s ordinary shares and ordinary share equivalents issued and outstanding, including ordinary shares underlying pre-funded warrants and non-voting convertible preferred stock.

(Press release, Crescent Biopharma, APR 29, 2026, View Source [SID1234664893])

On April 29, 2026 Assertio Holdings, Inc. ("Assertio" or the "Company") (Nasdaq: ASRT), reported that it has reached a mutual agreement with Garda Therapeutics, Inc. ("Garda") to extend the deadline to commence the previously announced tender offer to acquire all outstanding shares of Assertio to May 4, 2026.

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As previously announced on April 8, 2026, Assertio has entered into a definitive agreement (the "Garda Agreement") to be acquired by Garda for $18.00 per share in cash, or a total cash consideration of $125.1 million, plus a contingent value right. The Garda Agreement includes a 20-day "window-shop" period. Under the terms of the window-shop provision, Assertio is free to engage with other parties who may provide superior value to shareholders. In the event the Board terminates the Garda Agreement in favor of a superior bid during the window-shop period, a reduced breakup fee would apply.

(Press release, Assertio Holdings, APR 29, 2026, View Source [SID1234664913])

On April 29, 2026 AstraZeneca reported first quarter 2026 results.

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(Presentation, AstraZeneca, APR 29, 2026, View Source [SID1234665031])